Tag Archives: tgen

stroke

TGen, Phoenix Children’s Hospital look for new ways to store blood

The Translational Genomics Research Institute (TGen) and Phoenix Children’s Hospital are developing new economical methods of preserving, storing and transporting high-quality blood plasma proteins for use in diagnosing and treating disease.

Under a $698,502 three-year grant from the National Institutes of Health, the Phoenix Children’s Hospital Biorepository and TGen’s Proteomics Division are using special filter papers to devise a test that is highly accurate, yet simple enough that patients could provide their own blood samples from home, even under a variety of room temperatures.

Leading the project are Dr. David F. Carpentieri, M.D., Pediatric Pathologist and Biorepository Director at Phoenix Children’s Hospital; Dr. Patrick Pirrotte, Ph.D., Technical Director of TGen’s Center for Proteomics; Lizzie K. Neylon, MBA, Biorepository Coordinator at Phoenix Children’s Hospital; and Dr. Kostas Petritis, Ph.D., Principal Investigator at Phoenix Children’s Hospital.

In the initial phase of the study, researchers seek to reduce, and even eliminate, both immediate and long-term protein degradation by selecting the best methods of collecting, stabilizing and storing blood proteins. This combined effort holds the promise of significantly improving existing protocols with a simple and cost-effective method suitable for highly sensitive mass-spectrometry proteomic analysis.

Preliminary work has already been presented at meetings of Human Proteomics (HUPO) and the International Society of Environmental and Biological Repositories (ISBER). Researchers began collecting blood samples on May 2 from 20 consenting volunteers.

According to Dr. Carpentieri, this project is critical for the future of cost-effective proteomics research as suggested by similar DNA and RNA protocols that quickly stabilize molecules at ambient, or room-temperature, conditions.

“It is the vision of this working group, that this study will substantially contribute to the reduction of pre-analytical laboratory errors and will lead the way to reliable discoveries in the field of proteomics and pediatric diseases,” Dr. Carpentieri said.

Dr. Pirrotte said a key to the project is the use of dried blood-derived plasma proteins for the early detection of disease biomarkers. By storing dried blood on pre-treated filter paper, protein storage costs could be alleviated. Furthermore, samples would not need to be kept cold during shipment.

“We could collect samples without a clinician or trained personnel even being present. We could envision a skin-prick test that patients could conduct themselves, the sample could then be mailed to laboratories for analysis,” Dr. Pirrotte said. “Such a method would empower the patient, and increase the number potential medical follow-ups, while reducing costs. This could ultimately lead to better long-term monitoring of the patient’s health.”

In a second phase of the project, expected to start sometime in 2016, Phoenix Children’s Hospital and TGen would apply the best methods towards a study of 20 leukemia patients.

pharmaceuticals

TGen and Dell expand pediatric cancer fight

Dell has announced its extended partnership with the Translational Genomics Research Institute (TGen) to help clinical researchers and doctors globally expand the reach and impact of the world’s first Food and Drug Administration (FDA)-approved precision medicine trial for pediatric cancer.

The renewed commitment includes an additional $3 million Dell grant to support continued collaboration with TGen and support the Neuroblastoma and Medulloblastoma Translational Research Consortium’s (NMTRC) expanded pediatric cancer clinical trials in EMEA, starting with sites in France and Lebanon. This is the second grant Dell has provided TGen to accelerate treatment of pediatric cancer, bringing its total contributions to more than $15 million since 2011.

The grant will also allow TGen to use Dell technology to bring genomic sequencing to point of diagnosis and enable TGen to extend its capabilities past pediatric cancer to support sequencing for other medical conditions affecting children including rare childhood diseases. By leveraging the capacity of Dell’s technology infrastructure, TGen is able to redirect some of its attention and resources to research rare disease sequences and help families get answers more quickly.

With most large hospitals lacking the time and budget to research pediatric cancer treatments, TGen’s aim is to reach and treat as many children as possible. However, when looking to support patients globally, it realized that it could no longer manage all of its data processing from the US. With time running out for many of its patients, TGen needed a solution that it could trust and that would reduce the amount of time needed to find and sequence genomic data. Based on a successful partnership in the US, TGen and Dell decided to extend its partnership and expand its support to EMEA.

“Time is of the essence in our line of work so we’re constantly undergoing vendor evaluations to try to find the right tool for the job. Dell understands what we’re trying to accomplish – not an easy claim in the world of quick-fire genome sequencing – and it has the partnerships and hardware to help us do it,” said James Lowey, TGen Vice President of Technology.

TGen’s extended partnership with Dell will help it optimize a high-performance computing infrastructure to enable researchers to analyze and store massive amounts of genetic data more quickly and reach more patients than ever before. To date, TGen has been able to increase the number of computational hours by 376 percent and reduce the time it takes to analyze a patient’s molecular data — a process that used to take ten days — to six hours. These results will now be replicated in EMEA, as the infrastructure scales easily to handle the increased number of patients across the new sites.

“We are proud to help TGen in EMEA gain the speed and efficiency it needs to ensure that more children can benefit from timely, local and effective treatment.  Pediatric cancer is an issue that affects too many lives and we are committed to delivering benchmark solutions and support to ensure that the team at TGen are able to focus on this most important of work,” said Aongus Hegarty, President, Dell EMEA.

medical.research

TGen study may help stop spread of Staph

Staphylococcus aureus — better known as Staph — is a common inhabitant of the human nose, and people who carry it are at increased risk for dangerous Staph infections. However, it may be possible to exclude these unwelcome guests using other more benign bacteria, according to a new study led by scientists representing the Translational Genomics Research Institute (TGen), the Statens Serum Institut, and Milken Institute School of Public Health (SPH) at the George Washington University.

The study, published today in the AAAS journal Science Advances, suggests that a person’s environment is more important than their genes in determining the bacteria that inhabit their noses. The study also suggests that some common nasal bacteria may prevent Staph colonization.

“This study is important because it suggests that the bacteria in the nose are not defined by our genes and that we may be able to introduce good bacteria to knock out bad bugs like Staph,” said Dr. Lance B. Price, Director of TGen’s Center for Microbiomics and Human Health and a Professor of Environmental and Occupational Health at the Milken Institute. “Using probiotics to promote gut health has become common in our culture. Now we’re looking to use these same strategies to prevent the spread of superbugs.”

The multi-center research team looked at data taken from 46 identical twins and 43 fraternal twins in the Danish Twin Registry, one of the oldest registries of twins in the world. “We showed that there is no genetically inherent cause for specific bacteria in the nasal microbiome,” said senior author Dr. Paal Skytt Andersen. Dr. Andersen is head of the Laboratory for Microbial Pathogenesis and Host Susceptibility in the Department of Microbiology and Infection Control at the Statens Serum Institut and an Adjunct Professor at the University of Copenhagen.

The so-called nasal microbiome is the collection of microbes living deep within the nasal cavity. This research might ultimately lead to interventions that could route Staph from the nose and thus prevent dangerous infections, including those caused by antibiotic resistant Staph, the authors say. Studies suggest drug-resistant Staph infections kill more than 18,000 people in the United States every year.

The researchers also looked for possible gender differences and found that contrary to past studies that showed that men are at higher risk for Staph nasal colonization. This study, using DNA sequencing, found that there is no difference between men and woman in the likelihood of nasal colonization by Staph.

“This was a surprising finding. I felt like I was one of the MythBusters guys. For years, most scientists agreed that men were more likely to be colonized by Staph than women. But now we see that that was probably just an artifact of using old methods, and that men just tend to have more bacteria in their noses, which makes them easier to culture,” said Dr. Cindy Liu, a Pathology resident at Johns Hopkins School of Medicine, a TGen research affiliate, and the study’s lead author.

Importantly, the study found evidence that other types of organisms can disrupt Staph. A prime example is Corynebacterium, a mostly harmless bacterium that is commonly found on the skin. The study found that having high amounts of Corynebacterium in the nose was predictive of having low amounts of Staph and vise versa.

“We believe this study provides the early evidence that the introduction of probiotics could work to prevent or knock out Staph from the nose,” said Dr. Liu.

The next step will be to prove out the findings of the study’s models in a laboratory setting.

breast.cancer

TGen breast cancer study is journal’s most cited

At its Annual Meeting, the American Association for Cancer Research (AACR) announced that a scientific paper describing potential drug targets following the unprecedented genomic sequencing of 14 metastatic triple-negative breast cancer patients was the most cited study in 2013 of any published that year by AACR’s journal Molecular Cancer Therapeutics.

In the study, genomic sequencing – spelling out the billions of pieces of genetic information in an individual patient’s DNA – revealed therapeutic drug targets for difficult-to-treat, metastatic triple-negative breast cancer (TNBC).

The Translational Genomics Research Institue (TGen), Baylor University Medical Center and US Oncology Research investigators found significant recurring mutations and other changes in more than a dozen genes in patients treated at Baylor University Medical Center at Dallas. In addition, the investigators identified mutations previously unseen in metastatic TNBC and took the sequencing data into account in selection of therapeutic protocols specific to each patient’s genetic profile.

“The nature of this disease cried out for innovative research techniques such as whole genome sequencing coupled with new tools for data analysis,” said Dr. David Craig, Ph.D., TGen’s Deputy Director of Bioinformatics, one of the study’s co-lead authors.

The most frequently mutated gene among the tumors was the TP53 tumor suppressor, and aberrations were observed in additional tumor suppressor genes including CTNNA1, which was detected in two of six African American patients. Alterations were also seen in the ERBB4 gene, known to be involved in mammary-gland maturation during pregnancy and lactation, but not previously linked to metastatic TNBC.

Each tumor was genomically unique, but the majority contained alterations in one or both of the RAS/RAF/MEK/ERK and PI3K/AKT/MTOR pathways. Targeted therapeutic intervention aimed at these pathways achieved antitumor responses in several cases.

“The fact that this study has been cited more often than any other in Molecular Cancer Therapeutics that year is a testament to strong interest the oncology community has in understanding the molecular basis of metastatic TNBC, and in the hypothesis that targeting specific mutations in patients’ TNBCs will improve patients’ outcomes,” said Joyce O’Shaughnessy, M.D., the study’s other co-lead author. Dr. O’Shaughnessy is the Celebrating Women Chair of Breast Cancer Research at Baylor University Medical Center at Dallas, Chair of the US Oncology Network’s Breast Cancer Program, and a practicing oncologist with Texas Oncology.

Metastatic TNBC is a highly aggressive form of breast cancer that does not express the estrogen receptor, progesterone receptor or HER-2, the biomarkers successfully targeted in most breast cancers.

Metastatic TNBC has a poor prognosis with a median survival rate among metastatic patients of only one year. While TNBC accounts for only about 15 percent of all breast cancers, its more aggressive biology makes it responsible for nearly one in four deaths related to this disease.

“This study continues to stand as a great example of molecular medicine in practice. The results have provided novel and interesting clues into breast cancer biology and into the promise and challenges of precision medicine,” said Dr. John Carpten, Ph.D., TGen’s Deputy Director of Basic Science, Director of TGen’s Integrated Cancer Genomics Division, and the study’s senior author.

The study, “Genome and transcriptome sequencing in prospective triple negative breast cancer uncovers therapeutic vulnerabilities,” was conducted by the Translational Genomics Research Institute (TGen) and US Oncology Research with support from Life Technologies Corporation.

Molecular Cancer Therapeutics is one of several peer-reviewed scientific journals published by the 35,000-member American Association for Cancer Research (AACR), the oldest and largest scientific organization in the world focused on every aspect of high-quality, innovative cancer research. The programs and services of the AACR foster the exchange of knowledge and new ideas among scientists dedicated to cancer research, provide training opportunities for the next generation of cancer researchers, and increase public understanding of cancer.

bioscience

Scottsdale schools, TGen launch bioscience initiative

The Scottsdale Unified School District (SUSD) Foundation is teaming with the Phoenix-based Translational Genomics Research Institute (TGen) to create an Excellence in Bioscience Teacher Initiative.

Scheduled to take place in July 2015 at TGen, the new pilot program will benefit teachers and students within the Scottsdale Unified School District.

The Excellence in Bioscience Teacher Initiative will provide teachers in science, technology, engineering and math (STEM) with a strong fluency in cutting-edge biomedical research. An intensive, thee-day agenda will immerse them in a series of presentations, interactive discussions and practical experiences led by TGen bio-medical experts.

“Following our induction of TGen president — and Arcadia High School graduate — Dr. Jeffrey Trent into the SUSD Foundation’s Hall of Excellence in 2013, we have been working to expand our relationship with TGen to benefit the District,” said Price Nosky, chair of the SUSD Foundation.  “With SUSD Superintendent Dr. David Peterson’s encouragement, and through the work of SUSD staff members, we are pleased to announce the formation of this new partnership and initiative.”

The SUSD Foundation will fund the $26,500 initiative as part of the annual contributions it makes to support SUSD, reflective of its goal, and that of its sponsors, to support educational opportunities that directly impact students and teachers in the classroom.

TGen is a leading biomedical research institute dedicated to translating laboratory breakthroughs into new clinical diagnostics and therapeutic treatments for patients with cancer, neurological conditions and other serious maladies.

“We are pleased to work with the SUSD Foundation to create this educational opportunity for the SUSD,” said Dr. Trent.  “TGen recognizes that to fully achieve its mission we must invest in local schools to help build the pipeline of translationally-minded future scientists and physicians that will elevate Arizona in terms of health care and economic competitiveness.”

“We believe that increasing the content-knowledge, depth of understanding and insight of just one educator can benefit an exponential number of students over time,” said Brandy Wells, TGen Director of Education and Public Affairs.  “We hope this initiative can be used as a model for industry-education partnerships in the future.”

According to Dr. Peterson, a select number of teachers from Saguaro High School’s Math & Science Academy will be chosen to participate in the initiative.

“We will identify select teachers to participate in this initiative who will also work to share the information gained with fellow educators and students in the District,” said Dr. Peterson.  “This initiative of the SUSD Foundation will further showcase SUSD as a respected educational leader in developing collaborative, innovative programs that provide development opportunities for our teachers, and in turn, enables them to develop rigorous academic experiences for our students.”

Wells said that retaining and developing effective teachers is an ongoing need also recognized by TGen and one solution supported by the Institute is a visible and celebrated elevation of, and investment in, individual teachers.

“TGen’s interest in education and outreach is to help train and inspire Arizona’s next generation of researchers and physicians,” said Wells, a former junior high school teacher.  “Positively impacting teachers has been an ongoing interest area for me personally.  This program represents an opportunity I would have benefited from and sincerely enjoyed as a teacher.”

The pilot program was praised by Scottsdale Mayor W.J. “Jim” Lane, who has overseen an expansion of bioscience efforts through the city’s Cure Corridor, a stretch of Shea Boulevard that includes hospitals, clinics and other innovative businesses dedicated to bioscience and medicine.

“This new partnership is exciting and potentially powerful,” said Mayor Lane. “Combining cutting-edge research, innovation and technology with education can benefit everyone, particularly the students within the Scottsdale Unified School District.  As a Mayor active within Arizona’s Bioscience industry, I see this as the perfect collaboration.”

brain

TGen brain-tumor study guides new treatments

Led by the Translational Genomics Research Institute (TGen) and UC San Francisco (UCSF), a comprehensive genetic review of treatment strategies for glioblastoma brain tumors was published May 1 in the Oxford University Press journal Neuro-Oncology.

The study, Towards Precision Medicine in Glioblastoma: The Promise and The Challenges, covers how these highly invasive and almost-always-deadly brain cancers may be treated, reviews the continuing challenges faced by researchers and clinicians, and presents the hope for better treatments by harnessing the power of the human genome.

The study also describes a pioneering clinical trial underway for 15 patients at UCSF, guided by TGen research, in which an individual patient’s genomic profile is used to offer treatment recommendations to an expert, multidisciplinary panel. 

“This study thoroughly explores how we arrived at the current standard-of-care, and how — through cutting-edge genomic technologies — we might find better answers for these patients who need our help today,” said Dr. Jeffrey Trent, TGen President and Research Director and the study’s senior author.

Funded by The Ben & Catherine Ivy Foundation, the study is one of several simultaneous and coordinated efforts seeking to uncover the molecular source of this deadly brain cancer with the goal of prolonging survival of glioblastoma patients. 

“Despite pivotal advances in the characterization of genomic mutation in glioblastoma, targeted drug agents have so far shown minimal effect in clinical trials, and patient survival remains poor,” said Dr. Michael D. Prados, the Charles B. Wilson, MD, Endowed Chair in Neurological Surgery at UCSF, and one of the study’s co-lead authors.

One of the major difficulties in treating brain tumors is finding drugs that can penetrate the blood-brain barrier, which buffers the brain from the rest of the body’s blood-circulatory system. Located along capillaries, the blood-brain barrier protects the brain from rapid changes in the body’s metabolic conditions and minimizes exposure to molecules that are toxic to neurons in the brain.

“This study outlines strategies for overcoming past failures, primarily by applying targeted combination therapies that match the tumors’ genetic changes with drug compounds that can reach the central nervous system,” said Dr. Sara Byron, Research Assistant Professor in TGen’s Center for Translational Innovation, and the study’s other co-lead author.

Another major challenge in treating glioblastoma is its intrusive penetration into adjoining tissues, which prevents the complete surgical removal of the tumors from the brain, even with follow-up radiation and chemotherapy: “It is this invasive, infiltrative disease component that is the ultimate cause of recurrence, resistance and death,” the study says.

“All patients will continue to show tumor growth and progression because of rapidly proliferating infiltrative disease remaining after surgery,” according to the study. “Effective treatment for glioblastoma remains an unmet need.”

The only FDA-approved drugs to treat glioblastoma are temozolomide, nitrosoureas, and bevacizumab.

In the clinical trial begun at UCSF, multiple biopsies are performed on each patient at the time of surgery in different regions of the brain tumor. That is followed by extensive genome-wide profiling, leading to a selection of drugs that would target the brain cancer and diffuse regions of the lesion that cannot be removed by surgery.

Drug selection is individualized, and multiple FDA-approved agents (up to four) allowed. “Rules” for drug selection are implemented, using the specialized drug pharmacopeia designed for this trial. The drugs are chosen carefully, considered with knowledge about the ability of the drug to reach the brain and the patient’s past treatment history and concomitant therapies, with the assistance of multi-specialty, multi-institutional molecular tumor board that drafts a report to the treating physician.

In addition, “Small, informative, tissue-based clinical trials that take into account the individual molecular features of patients and provide early ‘go’ or ‘no go’ decisions are needed and should be prioritized over unselected, large, population-based strategies,” the study recommends.

A separate clinical trial that follows this path, also guided by TGen genomic research, is underway at Barrow Neurological Institute. This clinical trial also is funded by The Ben & Catherine Ivy Foundation. For more about this clinical trial, go to: www.tgen.org/home/news and click on March 10, 2015.

“These studies, and their associated clinical trials, have the potential to lift our knowledge of glioblastoma to an unprecedented new level,” said Catherine Ivy, President of The Ben & Catherine Ivy Foundation. “Developing drug compounds that breach the blood-brain barrier and are effective against tumors would fulfill one of the medical community’s most critical unmet needs, and boost the hopes of brain tumor patients everywhere.”

Contributing to the study published today were all three TGen Deputy Directors:  Dr. John Carpten, Deputy Director of Basic Science; Dr. Michael Berens, Deputy Director for Research Resources; and Dr. David Craig, Deputy Director of Bioinformatics.

86547298

Cycle for the Cure supports TGen cancer research

Participants in the 5th annual Cycle for the Cure — supporting cancer research at the Translational Genomics Research Institute (TGen) — have more than cycling to choose from this year.

Kinesis resistance exercise, yoga and fitness classes will join stationary cycling, or spinning, as part of Cycle for the Cure’s record-breaking efforts to support TGen. 

For spinning enthusiasts, three exhilarating 2-hour indoor cycling sessions are set for May 3, with one already sold out:

•    8-10 a.m. — Gainey Village Health Club & Spa, 7477 E. Doubletree Ranch Road, Scottsdale.
•    10 a.m.-noon — DC Ranch Village Health Club & Spa, 18501 N. Thompson Peak Parkway, Scottsdale.
•    3-5 p.m. — Camelback Village Racquet & Health Club, 4444 E. Camelback Road, Phoenix. SOLD OUT.

Besides spinning, the Camelback Village location also will offer two Kinesis classes and a Yoga class on May 3.

On May 2, the Gainey Village club will offer a variety of fitness classes as part of their “Village for a Cure” alternative for spinning classes.

The Village Health Clubs welcome non-members to participate. Bikes can be reserved with a $200 tax-deductible donation. For details and to register, please go to www.tgenfoundation.org/cycle.

“This year, our Cycle for the Cure event provides even more fun ways to support TGen’s amazing cancer research,” said event Co-Chair Vicki Vaughn. “We are so energized and proud of the dedicated support we continue to receive from our friends and families.”

More than 200 men and women are expected to participate, aiming to raise a record $175,000 for TGen cancer research. Last year, Cycle for the Cure raised a record $154,000, exceeding last year’s goal by more than 20 percent.


A Post-Ride Party, with food, drinks and music by Nate Nathan and the MacDaddy-O’s Band, is planned for 5 p.m. May 3 at Camelback Village. The party is free for riders; $20 donation for guests.

“Spinning participants should look forward to a fast-paced two hours of upbeat music and positive energy, led by some of the best instructors in the business,” said Robyn DeBell, the event’s other Co-Chair. “Please join us to help fund TGen’s cancer fighting research.”

Dr. Jeffrey Trent, TGen President and Research Director, will be among TGen’s renowned scientists participating in Cycle for the Cure

Using genomic sequencing, TGen helps doctors match the appropriate therapy to each patient’s DNA profile, producing the greatest patient benefit.
 


“Year after year, these dedicated volunteers find new ways to energize Cycle for the Cure, raising awareness and essential funding for TGen’s research,” said TGen Foundation President Michael Bassoff. “Cycle for the Cure helps deliver hope and answers for patients and their families. The dollars raised stay right here in Arizona, where TGen can really make a difference.”

cancer

TGen begins Zebrafish pancreatic cancer studies

For more than a decade, a glassy striped fish smaller than a door key has proved an important model organism in scientific research. Named for the uniform horizontal stripes on the side of its body, the zebrafish is a tiny creature that packs a punch in terms of biological similarities with the human.

Today, scientists at the Translational Genomics Research Institute (TGen) are using zebrafish to accelerate investigations of pancreatic cancer, the nation’s fourth-leading cause of cancer-related death.

TGen researchers believe the zebrafish can aid in the search for therapeutics that could help slow down, and even reverse, the growth and spread of cancer in pancreatic cancer patients.

Amazingly, this tiny fish shares about 70 percent of the genetic code of humans, has genetic similarities for the overwhelming majority of genes that cause human disease, grows to maturity in a matter of weeks, and are relatively inexpensive to manage.

Importantly, because of their transparent scales, they can provide a window into the real-time development of cancer tumors.

“With a microscope, you can see what is wrong without having to dissect it,” said Dr. Haiyong Han, a TGen Associate Professor, head of TGen’s Pancreatic Cancer Research Unit. “You can see the tumor just by looking into the fish.”

Zebrafish are vertebrates, so they have organs similar to people, including a pancreas, an organ near the stomach that produces digestive juices and several key hormones.

Genes are responsible for creating proteins. An altered gene disrupts the associated proteins, including those that lead to cancer. By studying altered genes in zebrafish, TGen researchers can monitor the initiation, growth and fatal effects of pancreatic tumors, including their spread to other organs.

Even though they are small, zebrafish are relatively complex organisms, allowing researchers to use them to mimic problems, and solutions, in people. And because zebrafish achieve maturity within about 3 months, researchers can compress the time otherwise needed to study tumor development.

“Where it would take months or years in a human, it only takes days or weeks to see the tumor growth in zebrafish,” said Dr. Daniel D. Von Hoff, TGen’s Distinguished Professor and Physician-In-Chief. “This is going to be of tremendous benefit for our scientific investigations, and ultimately for our patients.”

Care of the zebrafish is overseen by an Institutional Animal Care and Use Committee, or IACUC, which reviews all testing procedures and ensures the humane treatment of the fish throughout their lifecycle.

This year, nearly 49,000 Americans will be diagnosed with pancreatic cancer, and more than 40,000 will die from this disease. Median survival for patients with advanced disease is less than 6 months following diagnosis, and the 5-year survival rate is less than 6 percent for all patients.  

Pancreatic cancer’s lethal nature stems from its propensity to rapidly spread to distant organs. Because there is no early screening test, it usually is not diagnosed until its late stages, often when surgery is no longer an option, making it difficult to treat.

Dr. Han will monitor the growth and dissemination of tumors in the zebrafish and, specifically, look at the epithelial–mesenchymal transition (EMT), in which epithelial cells gain migratory and invasive characteristics.

This should help TGen investigators to better understand how pancreatic cancer invades local tissue, causing pain, and how it spreads to other organs — especially to the liver and lungs — which often is the actual cause of death in pancreatic cancer patients.

And because of low costs associated with maintaining zebrafish, investigators can conduct multiple studies in relatively short periods of time.

TGen’s zebrafish project is funded by the Seena Magowitz Foundation.

Healthcare Leadership Awards 2015_38

Az Business honors winners of Healthcare Leadership Awards

Az Business magazine honored winners of the 2015 Healthcare Leadership Awards Thursday in front of a packed house at the Arizona Grand Resort.

“The Healthcare Leadership Awards honor the women, men and institutions whose passion and innovation are saving lives, extending lives, and improving the quality of our lives,” said Cheryl Green, publisher for AZ Big Media, which publishes Az Business magazine.

Sponsors included Blue Cross Blue Shield of Arizona, Arizona Central Credit Union and CodeRed-I.

Here are the winners of the 2015 Healthcare Leadership Awards:

Healthcare advocate/educator of the year: Catherine Ivy, Ben & Catherine Ivy Foundation

Behavioral health company of the year: Southwest Behavioral Health Services

Bioscience company of the year: VisionGate

Insurance provider of the year: Blue Cross Blue Shield of Arizona

Legal advocate of the year: Martin L. Shultz, Brownstein Hyatt Farber Schreck

Medical company of the year: SynCardia

Medical research company of the year: Barrow Neurological Institute

Researcher of the year: Dr. Daniel D. Von Hoff, TGen and HonorHealth

Physician of the year: Robert J. Arceci, M.D., Phoenix Children’s Hospital

Healthcare executive of the year: Tim Bricker, Chandler Regional and Mercy Gilbert medical centers

Medical center or hospital of the year: Cancer Treatment Centers of America at Western Regional Medical Center

Lifetime achievement award: Peter Fine, Banner Health

“The word visionary is thrown around often, but it’s not often that it really fits,” said Michael Gossie, editor in chief for Az Business magazine. “But that’s not the case with Peter Fine. He is a visionary. And it took a visionary to take two healthcare systems with very different histories and grow it into a healthcare company that generates more than $5 billion in annual revenue, operates 28 acute-care hospitals across seven states, and employs more than 45,000 employees in Arizona.”


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research

Clinical trials change Arizona’s bioscience, business sectors

Last November, Arizona voters resoundingly passed Prop. 303 making it legal in Arizona for companies and physicians to provide terminally ill patients the “right to try” investigational drugs or therapies outside an FDA approved clinical trial.    While it sounded good in the short description provided to voters, in reality, it is unlikely to provide the outcomes one might expect since the manufacturers, physicians, pharmacists, and hospitals are required to follow the federal processes that govern these investigational treatments.  Proposition 303 did not change that.

What Are Clinical Trials?

The clinical trials process is an important step in the discovery, development and delivery pathway that leads to new life saving and live enhancing innovations.  Clinical trials are research studies that explore whether a medical strategy, treatment, or device is safe and effective for patients. These studies also may show which medical approaches work best for certain illnesses or groups of people. Clinical trials produce the best data available for health care decision making and the studies follow strict scientific standards. These standards protect patients and help produce reliable study results.

Today, in Arizona, there are 1,380 ongoing clinical trials according to ClincialTrials.gov which is the national database provided by the U.S. National Institutes of Health.  These studies cover a wide range of therapies and conditions.  Through the dedicated work of innovators, healthcare professionals, and patients, we are learning more about the safety and effectiveness of future treatments.

ClinicalTrials.gov  is a registry and results database of publicly and privately supported clinical studies of human participants conducted around the world and currently lists 187,600 studies with locations in all 50 states and in 189 countries.

How can we speed the path to innovation and give more patients access to life changing innovations?

On April 29th, members of Arizona’s life science industry and members of the community at large will gather the Sheraton Phoenix Downtown for the 2015 AZBio Expo which will focus on clinical trials in Arizona. Event details and ticket information for the 2015 AZBio Expo on April 29, 2015 at the Sheraton Phoenix Downtown are available at AZBioExpo.com.

Thanks to the support of leaders in Arizona’s life science community, practicing physicians, patients, and caregivers are invited to register for the full day conference free of charge with discount code “AZBusiness.”

The program is designed to provide an update on what Arizona’s clinical trial landscape looks like today and what is could grow to be in the future.  The program includes:

• An Introduction to The Clinical Trials Process by Mark Slater, PhD, Vice President, Research at HonorHealth Research Institute

• A keynote presentation by Matthew Huentelman, PhD, Associate Professor, Neurogenomics Division and Head of the Neurobehavioral Research Unit at the Translational Genomics Research Institute (TGen)

• A keynote presentation by Glen Weiss, MD, MBA, Director of Clinical Research & Medical Oncologist, Western Regional Medical Center, Cancer Treatment Centers of America

• A View of Arizona’s Clinical Trials Landscape by Joan Koerber-Walker, President and CEO of the Arizona Bioindustry Association and Chairman of the AdvaMed State Medical Technology Alliance in Washington, DC.

• A Discussion on Funding Clinical Trials led by Joan Koerber-Walker with Terry Urbine, PhD of the  UA College of Pharmacy, Jeremy Shefner, MD, PhD of the Barrow Neurological Institute, and Teresa Bartels from Gateway for Cancer Research.

• A Discussion on Engaging Patients in the Process led by Greg Vigdor, President & CEO, Arizona Hospital and Healthcare Association with  Brian Browne of Banner Research, Barbara Kavanaugh of the Arizona Myeloma Network, and Marcia K. Horn of the International Cancer Advocacy Network

• A Discussion on Growing Arizona’s Clinical Trials Base led by Nazneen Aziz, PhD,  Chief Research Officer and Senior Vice President, Phoenix Children’s Hospital with  Joan Rankin Shapiro, PhD of the UA College of Medicine Phoenix), and Linda Vocila, BSN, RN of TD2.

• Rapid Fire Presentations featuring Arizona companies with active clinical trials here in Arizona and around the world including:  Cancer Prevention Pharmaceuticals, Inc.,  Capstone Therapeutics,  the Center For Sustainable Health at the Biodesign Institute at Arizona State University,  Cord Blood Registry,  Insys Therapeutics, Inc., and  NuvOx Pharma.

By focusing on clinical trials together, we can help find answers for the people who matter most, the patients.

healthcare

Az Business names Healthcare Leadership Awards finalists

Each year, AZ Business magazine hosts the Healthcare Leadership Awards to honor the women, men and institutions that bring excellence and innovation to Arizona’s healthcare sector.

Az Business is proud to announce the 2015 Healthcare Leadership Awards finalists, who were chosen by a panel of industry experts and will be recognized at the Healthcare Leadership Awards dinner and awards ceremony on April 9  at the Arizona Grand Resort. The finalists, in alphabetical order, are:

Abrazo Health — Arrowhead Hospital

Abrazo Health — Michele Finney

Affiliated Urologists — Dr. Mark Hong

Banner Health, Cardon Children’s Medical Center — Rachel Calendo

Banner Health — Peter Fine

Ben & Catherine Ivy Foundation — Catherine Ivy

Blue Cross Blue Shield of Arizona

Brownstein Hyatt Farber Schreck — Martin L. Shultz

Cancer Treatment Centers of America at Western Regional Medical Center

CTCA — Dr. Glen Weiss

Dedicated Health Solutions

Dignity Health — Barrow Neurological Institute

Dignity Health, Chandler Regional and Mercy Gilbert medical centers — Tim Bricker

Dignity Health — St. Joseph’s Hospital and Medical Center

HonorHealth and TGen — Dr. Daniel D. Von Hoff

HonorHealth – Virginia G. Piper Cancer Center

IASIS Healthcare — Tony Marinello

Insys Therapeutics

Magellan Health

Maricopa Integrated Health System — Dr. David Wisinger

Medtronic

Midwestern University — Kathleen Goeppinger

Quarles & Brady — Roger Morris

Phoenix Children’s Hospital – Dr. Robert J. Arceci

Radiant Research

Remuda Ranch

Snell and Wilmer – Richard Mallery

Sonora Quest Laboratories

Southwest Behavioral Health Services

SynCardia Systems

The CORE Institute — Dr. David Jacofsky

UnitedHealthcare of Arizona

University of Arizona Cancer Center

VisionGate

chromosome

TGen uses X chromosome to uncover disorders in girls

Using a basic genetic difference between men and women, the Translational Genomics Research Institute (TGen) has uncovered a way to track down the source of a neurological disorder in a young girl.

TGen’s discovery relies on a simple genetic fact: Men have one X and one Y chromosome, while women have two X chromosomes. This women-only factor was leveraged by TGen investigators to develop a highly accurate method of tracking down a previously unrecognized disorder of the X-chromosome.

The study of a pre-teen girl, who went years with an undiagnosed neurobehavioral condition, was published today in the scientific journal PLOS ONE.

TGen’s findings were made within its Dorrance Center for Rare Childhood Disorders, where investigators and clinicians apply the latest tools of genomic medicine to provide answers for parents seeking to identify the disease or disorder affecting their child.

The scientists sequenced, or spelled out in order, the complete genetic codes of DNA and RNA of the girl. Because girls inherit an X chromosome from each of their parents (boys inherit a Y chromosome from their father), they also sequenced her mother and father. On average, about half of all X chromosomes active in a female come from the mother and the other half from the father.

“We now have the tools to significantly accelerate the diagnostic process, reducing the need for children to undergo multiple tests that can be emotionally and physically taxing for the entire family,” said Dr. David Craig, TGen’s Deputy Director of Bioinformatics, Co-Director of the Dorrance Center and the paper’s senior author.

Sequencing would reveal the proportion of X chromosomes, and if disproportionate, whether the more abundant of the two were damaged in some way, which often leads to X-linked genetic conditions.

“At the time of enrollment, we suspected the girl had a complex neurobehavioral condition, based on her attention deficit, and delays in development and learning,” said Dr. Vinodh Narayanan, Medical Director of the Dorrance Center. “Her brain MRI scans were normal. We needed to find out more — at the genetic level — about what might be causing her disorder.”

By sequencing the DNA and RNA, TGen investigators were able to precisely identify which cells contained active X chromosomes from the girl’s mother, which contained active X chromosomes from the father, in what proportions, and whether they were associated with any known disorders.

They discovered that the X chromosome from the father contained a segment shown to be associated with neurobehavioral conditions. Interestingly, however, the proportion of X chromosomes active in the girl’s cells skewed toward the normal X inherited from her mother. This skewing may have led to a milder, harder to diagnose condition undetected by conventional methods.

“This study shows the power sequencing holds when scanning for potential disease causing and disease-modifying genetic variations,” said Dr. Matt Huentelman, the other Co-Director of the Dorrance Center and an author of the PLOS ONE paper. “I’m most excited to see the pace at which TGen has pushed the genome sequencing technology to where it can help patients — today.”

TGen Research Associate Szabolcs Szelinger, the paper’s lead author, said: “With just a small bio sample, we are now able to provide a comprehensive evaluation of the effects that genetic variation has on patients, leading to highly personalized treatment options, while at the same time providing researchers with insights into the underlying molecular processes.”

Since opening in October 2013, TGen’s Dorrance Center for Rare Childhood Disorders Dorrance Center has enrolled nearly 300 families. And with the rapid decrease in sequencing costs and improved analytical methods, comprehensive, integrative sequencing approaches will likely be used more in the future. Information on the Dorrance Center is available at www.c4rcd.org.

Funding for the study came from the State of Arizona, the Stardust Foundation, and donations to the TGen Foundation.

stk99406cor

TGen and NAU pandemic flu test patent approved

The federal government has awarded a patent to the Translational Genomics Research Institute (TGen) and Northern Arizona University (NAU) for a test that can detect — and assist in the treatment of — the H1N1 pandemic flu strain.

TGen and NAU initially developed this precise, genomics-based test during a significant global swine flu outbreak in 2009.

The newly-patented test, developed at TGen’s Pathogen Genomics Division (TGen North) in Flagstaff, can not only detect influenza — as some tests do now — but also can quickly inform doctors about what strain of flu it is, and whether it is resistant to oseltamivir (sold by Roche under the brand name Tamiflu), the primary anti-viral drug on the market to treat H1N1.

As with other influenza strains, H1N1 flu can over time be expected to show signs of resistance to oseltamivir, and new treatments will be needed to respond to future pandemics.

“The problem with influenza is that it can become resistant to the antiviral drugs that are out there,” said Dr. Paul Keim, Director of TGen North, a Regents Professor of Biology at NAU and one of the test’s inventors. “Because it is a virus, it easily mutates and becomes resistant.”

David Engelthaler, Director of Programs and Operations for TGen North and another of the test’s inventors, said this flu detection and susceptibility test uses a molecular technique that rapidly makes exact copies of specific components of H1N1’s genetic material.

“Many people, including physicians, don’t realize that the pandemic swine flu strain from 2009 is still the most important flu strain out there. This assay is very effective with detecting and characterizing this dominant strain in the U.S. and around the world,” said Engelthaler, the former State Epidemiologist for Arizona, and former State of Arizona Biodefense Coordinator.

The third inventor of the test is TGen North Lab Manager Elizabeth Driebe.

Previously, only the U.S. Centers for Disease Control Prevention (CDC) and a few select labs could look for resistance, using time-intensive technology.

“This new test puts the power in the hands of the clinician to determine if their drugs will work or not. This is really important moving forward as we discover new strains that are resistant to antivirals,” Engelthaler said.

The World Health Organization (WHO) has identified dozens of instances in which H1N1 was resistant to Tamiflu.

At most doctors’ offices, there is no readily available test for H1N1. Such tests generally are conducted by state and federal health agencies, and usually for those patients who require hospitalization and appear at high risk because they have a suppressed immune system or they have a chronic disease.

“Our test measures minute amounts of virus and minute changes to the virus. Not only does it detect when resistance is occurring, but it also detects it at the earliest onset possible,” Engelthaler said.

This new patent — No. US 8,808,993 B2, issued Aug. 19 by the U.S. Patent and Trademark Office — could be licensed for development of test kits or for development of a testing service.

Earlier this year, TGen-NAU celebrated its first joint patent for a genomics-based test that can identify most of the world’s fungal infections that threaten human health.

childrens hospital

TGen, Dell help children with cancer

Dell, Terascala and the Translational Genomics Research Institute (TGen) are installing state-of-the-art computing and programing specialized for human genome investigations at the National Cancer Institute (NCI).

As part of a formal collaboration, Dell is providing the Dell Genomic Data Analysis Platform, designed to deliver fast analysis of the billions of data points required when sequencing, or decoding, aspects of the human DNA and other genomic data.

TGen is providing NCI with high performance computing and bioinformatics support, as well as specialized software and tools developed over the last three years as TGen and Dell have partnered in support of pediatric cancer research programs globally, including a groundbreaking personalized medicine trial for pediatric cancer conducted by the Neuroblastoma and Medulloblastoma Translational Research Consortium (NMTRC).

NCI is providing TGen with access to more than 800 sequenced child-cancer genomes, which will be used in TGen’s ongoing pediatric cancer research.

“This is an enormous opportunity for the government to work with top private and research organizations to share information and expertise that will help guide physicians and medical benefit for children around the world. A system like this is critical in expediting patient care,” said James Lowey, TGen’s Vice President of Technology.

The mission of the Oncogenomics Section at the NCI is to harness the power of high throughput genomic and proteomic methods to improve the outcome of children with high-risk metastatic, refractory and recurrent cancers.

The new system at NCI will be used for four purposes:

1. House genomics data of well over 800 pediatric cancers, in a user friendly database.
2. RNAseq and immunohistochemistry database for the Stand Up to Cancer Pediatric Dream team.
3. Computational server for a precision therapy trial to be conducted with the Children’s Oncology Group.
4. Computational server for a precision therapy trial to be conducted with the Center for Cancer Research, NCI.

The research goals are to integrate the data, decipher the biology of these cancers and to identify and validate biomarkers and novel therapeutic targets and to rapidly translate findings to the clinic. This new system will host the largest collection of clinically annotated pediatric cancer genomic data ever to be released to the scientific community. Not only will this system enable state-of-the-art precision therapy trials for children and adults with lethal cancers, but also provide clinically annotated high-resolution genomics data for basic science research.

“With the advent of faster, low-cost genome sequencing technology, researchers now have a critical gateway to understanding the underlying molecular pathways for diseases. And for some diseases, every hour closer to discovery and result can mean the difference between life and death,” said Walker Stemple, Dell’s High Performance Computing Product Manager.

Terascala technology is present in several key areas of the new Dell computer cluster: TeraOS improves the reliability, ease of use, and performance of the Lustre storage in the solution; Terascala gateways enable a direct interface for the genome sequencers to the Lustre storage; and Terascala Support delivers optimized technical support across the entire storage appliance.

“We are very excited to be a contributor to this significant HPC advancement at the NCI,” said Steve Butler, CEO of Terascala. “The result is a much faster and more efficient workflow for NCI researchers. Our hope and belief is that NCI researchers will be able to accelerate their work resulting in a faster timeline in breakthroughs in the battle against cancer.”

When cancer has spread beyond the site of origin it becomes hard to cure. In addition, children with relapsed or refractory cancers have about a 20 percent chance of survival. Neuroblastoma, a rare cancer that strikes one in 100,000 children annually usually before the age of 5, is so deadly that it is responsible for one in seven pediatric cancer deaths. The cancer comes from the sympathetic nervous system, which controls heart rate, blood pressure and digestion, with aggressive tumors that are unique to each child.


To overcome these challenges, parents and physicians and scientists from the Neuroblastoma and Medulloblastoma Translational Research Consortium (NMTRC; https://nmtrc.org) and TGen have teamed to launch a groundbreaking personalized medicine clinical trial investigation for pediatric cancer. The trial is based on research from a group of collaborating investigators who are developing a personalized medicine process that is intended to permit near “real time” processing of information on patient tumors and prediction of best drugs for a specific patient.

This process generates more than 200 billion measurements per patient that must be analyzed, shared and stored. The computation and analysis of this information can take weeks, even months, to process. The Dell solution and TGen software decreased RNA-Seq data analysis time from 7 days to 4 hours.

TGen fundraising event moves to Scottsdale

The 9th annual stepNout Run, Walk Dash, a major fundraising event for pancreatic cancer research at the Translational Genomics Research Institute (TGen), is moving to the Scottsdale Sports Complex.

More than 1,000 participants have attended stepNout in each of the past few years, and even more are expected to attend this year’s morning-long event on Nov. 2 in Scottsdale, featuring fun, competitive races for all ages and abilities, including the event’s signature 5K run.

“We are thrilled to announce that our enormously successful stepNout event will come to the City of Scottsdale. By partnering in this new way with the City of Scottsdale, TGen is generating answers and hope in the search for new treatments for patients with pancreatic cancer,” said TGen Foundation President Michael Bassoff.

Vowing to “fight pancreatic cancer, one step at a time,” stepNout organizers plan to raise more than $150,000 this year on the way to eventually surpass the $1 million mark in fundraising. Participants have donated more than $750,000 since the event started in 2006 at Kiwanis Park in Tempe.

One of TGen’s goals is to develop a method of early detection for pancreatic cancer. Currently, there are no tests to catch this disease in its early stages. As a result, it often is not diagnosed until its late stages, making it more difficult to treat.

Pancreatic cancer this year will take the lives of nearly 40,000 Americans, the nation’s fourth-leading cause of cancer-related death.

TGen’s pancreatic cancer research is led by Dr. Daniel D. Von Hoff, TGen’s Distinguished Professor and Physician-In-Chief, and Chief Scientific Officer for the Virginia G. Piper Cancer Center Clinical Trials at Scottsdale Healthcare, a partnership with TGen.

Dr. Von Hoff is recognized as one of the world’s leading authorities on pancreatic cancer. He and his team have helped develop three different treatment regimens to improve survival for people with advanced pancreatic cancer. If applied earlier, these regimens have the potential to make an even more powerful impact against the disease.

“We are proud to add stepNout to the calendar of exciting and meaningful events that call Scottsdale home. TGen is one of the most significant contributors to Scottsdale’s Cure Corridor of research and medical facilities, offering world-class healthcare opportunities to residents of Scottsdale and all Arizona citizens,” said Scottsdale Mayor W.J. “Jim” Lane.

Scottsdale Sports Complex, 8081 E. Princess Drive, is a state-of the-art, 71-acre competitive sport facility offering tournament level playing conditions. The facility accommodates a variety of flat field sports such as soccer, lacrosse, football, Ultimate Frisbee and rugby. In addition to sports fields, the complex has a lighted basketball court, a shaded playground, multi-use paths, open park space and two restroom facilities.

If you go to stepNout


What: TGen’s 9th annual stepNout Run/Walk/Dash for pancreatic cancer research.
Where: Scottsdale Sports Complex, 8081 E. Princess Drive, northeast of Hayden and Bell roads, between Loop 101 and Frank Lloyd Wright Boulevard.
When: 7-11 a.m. Sunday, Nov. 2. Registration starts at 7 a.m.; races begin at 9 a.m.; an awards ceremony is set for 10 a.m.; and a kids’ dash is planned for 10:30 a.m.
Cost: Registration fees range from $15 to $35, depending on age and competition. Children ages 4 and under are free.

Registration: Register at the event, or register online by Oct. 28 by visiting www.tgenfoundation.org/step.
Parking: Free.

brain

TGen-Ivy Foundation brain cancer trial approved

In 2012, The Ben & Catherine Ivy Foundation awarded $10 million in grants for two groundbreaking brain cancer research projects at the Translational Genomics Research Institute (TGen). One of those projects has officially received the final regulatory approval from University of California, San Francisco, which means patient enrollment for the trial can begin.

In the $5-million-project, “Genomics Enabled Medicine in Glioblastoma Trial,” TGen and its clinical partners will lead first-in-patient clinical trial studies that will test promising new drugs that might extend the survival of GBM patients. This multi-part study will take place in clinics across the country and TGen laboratories.

“GBM is one of the top three fastest-killing cancers out there and it affects people of all ages,” said Catherine (Bracken) Ivy, founder and president of The Ben & Catherine Ivy Foundation. “It is critical that we fund research that will help patients live longer so we can study and treat brain cancer.”

The project begins with a pilot study of 15 patients, using whole genome sequencing to study their tumor samples to help physicians determine what drugs might be most beneficial.

To support molecularly informed clinical decisions, TGen labs also will examine genomic data from at least 536 past cases of glioblastoma, as well as tumor samples from new cases, developing tools that will produce more insight into how glioblastoma tumors grow and survive. TGen also will conduct a series of pioneering lab tests to measure cell-by-cell responses to various drugs.

“GBM is a disease that needs answers now, and we strongly believe those answers will be found in the genome,” said Dr. David Craig, TGen’s Deputy Director of Bioinformatics, Director of TGen’s Neurogenomics Division, and one of the projects principal investigators. “Identifying the genes that contribute to the survival of glioblastoma will provide valuable information on how to treat it, and may also lead to an improved understanding of what drives other cancers as well.”

To get new treatments to patients as quickly as possible, this five-year study will include a feasibility study involving up to 30 patients, followed by Phase II clinical trials with as many as 70 patients. TGen is teaming with the Ivy Early Phase Clinical Trials Consortium that includes: University of California, San Francisco; University of California, Los Angeles; the MD Anderson Cancer Center; Memorial Sloan Kettering Cancer Center; University of Utah; and the Dana-Farber/Harvard Cancer Center.

The results of these clinical trials should not only help the patients who join them, but also provide the data needed for FDA approval and availability of new drugs that could benefit tens of thousands of brain cancer patients in the future.

“Working with physicians, the project will aim to understand treatment in the context of the tumor’s molecular profile. We will have the opportunity to determine when combinations of drugs might be more effective than using a single drug, quickly identify which therapies don’t work, and accelerate discovery of ones that might prove promising for future development,” said Dr. John Carpten, TGen’s Deputy Director of Basic Science, Director of TGen’s Integrated Cancer Genomics Division, and another of the project’s principal investigators.

In addition to helping patients as quickly as possible, the project should significantly expand Arizona’s network of brain cancer experts.

brain

‘Get Your Jersey On’ funds TGen concussion study

Kyrene de las Brisas Elementary School students and teachers will wear their favorite sports team jersey or t-shirt to class today, the first organization to participate in “Get Your Jersey On,” a fun way to help promote and fund concussion research at the Translational Genomics Research Institute (TGen).

The Chandler school is the first of what is expected to be many organizations this fall that will help fund TGen’s collaboration with the Arizona State University Sun Devil football team to help find new ways to protect athletes from serious injuries caused by head trauma.

“Our school is just a few miles south of ASU, and what better — and fun — way to show our support for the teams and athletes than to help fund a program that will ultimately help protect their health. Concussions affect not just athletes, but people of all ages. We are proud to partner with TGen to help raise the awareness of this important research,” said Dino Katsiris, Assistant Principal at Kyrene de las Brisas Elementary School.

Teachers and parents of students participating in “Get Your Jersey On” are encouraged to make small donations of $5 or $10 to TGen. If you would like your organization to participate, contact Dean Ballard, Assistant Director of Development for the non-profit TGen Foundation, at dballard@tgen.org, or 602-343-8543.

Student-athletes at ASU wear football helmets made by Riddell, a leader is sports helmet technology, with sensors that record the number, direction and intensity of impacts during games and practices.

TGen researchers, working with Barrow Neurological Institute and A.T. Still University, are attempting to connect data about the helmet impacts with biological changes that could be detected in the players’ blood, urine or saliva samples.

The goal is to discover a biomarker — some change in the student-athlete’s genetic makeup — that would objectively indicate when they are too injured to continue playing, and when they are fit enough to return to the game.

Representatives from the Sun Devil medical team and TGen will collect the molecular samples from the participating athletes, all of whom volunteered to partake in the study.

“It is so exciting to have the children of Kyrene de las Brisas Elementary School join us in this important work,” said Michael Bassoff, President of the TGen Foundation. “We welcome the participation of Brisas Elementary School and other businesses and organizations who want to turn their love of sports into a way to help protect the athletes they admire.”

For more information about the TGen-led concussion study, please visit tgen.org.

Conquering Concussions

ASU, TGen Team Up for Concussion Research

Riddell, the leader in football helmet technology and innovation, and the Translational Genomics Research Institute (TGen), a leader in cutting-edge genomic research, today announced that the Pac-12’s Arizona State University and its Sun Devil football program will again participate in a genetic research study designed to advance athlete concussion detection and treatment.

Now in its second year, the joint research project will combine molecular information and head impact data from Sun Devil football student-athletes to identify whether the effects of sub-concussive hits are identifiable. The researchers will monitor the players’ changing molecular information throughout a season of typical head impact exposure associated with football practice and games. Representatives from the Sun Devil medical team and TGen will collect the molecular samples from the participating athletes, all of whom volunteered to partake in the study.

“This partnership represents another dynamic and innovative step toward ensuring that the health and well-being of our student-athletes remains our most important goal,” Vice President for Arizona State University Athletics Ray Anderson said. “Sun Devil Athletics continues to serve as a pioneering force in this important issue and is proud to participate in this world-class research study for the second consecutive year with two outstanding industry trendsetters in Riddell and TGen.”

Arizona State’s preferred helmet and protective equipment provider, Riddell, has again deployed its Sideline Response System (SRS) to obtain real-time head impact data from Arizona State football student-athletes. Riddell SRS provides researchers with a wide range of valuable information on the frequency and severity of head impacts a player receives during games and practices. Data gathered from the system will be combined with genetic information from players that experience concussion, with the objective of helping physicians diagnose concussion and better identify when a player might be expected to recover and return to the field.

“Player protection has become an essential part of football, and this cutting-edge partnership sets ASU apart from not only the rest of the conference, but every collegiate football program in the nation,” ASU Head Coach Todd Graham said. “We are not only looking out for our student-athletes while they are enrolled at ASU, but for the rest of their lives. You become a part of the brotherhood once you put on the maroon and gold, and that doesn’t end at graduation.”

Riddell will also utilize the player head impact data collected from the ASU and TGen research partnership to inform the development of new football helmets and further refine updates to smart helmet technologies like Riddell SRS and its recently launched Riddell InSite Impact Response System.

“We’re impressed by the enthusiasm exhibited by our partners, Arizona State University and TGen, as we enter the second season of our important research collaboration,” President of Riddell Dan Arment said. “They have matched our level of passion for football, and we are all committed to better protecting those that play the sport we love. We are left encouraged following the first year of our project and look forward to continuing on the path towards advancing concussion detection and treatment of athletes.”

The researchers at TGen are exploring whether the effects of sub-concussive hits are identifiable through blood-based molecular information. Their findings could prove pivotal to the game of football and other sports. Similar to last season, during this phase of the study the TGen faculty and staff are on the sidelines collecting samples and data. A baseline sample was collected from all participating players prior to their pre-season workouts. Since then, the researchers have followed the team through their daily workouts and will continue throughout the season.

Through the collection of samples over various points in time and the data generated by Riddell SRS, the goal is to identify the genomic changes in athletes exposed to routine head impacts during practice and games, athletes with diagnosed concussions that recover on both a routine time scale, and athletes with persistent symptoms following concussion that require additional treatment.

“As the mother of a young son who has played football, I’m keenly aware of the need to improve the current standards in place today for dealing with this issue,” said TGen Associate Professor Dr. Kendall Van Keuren-Jensen, whose technique for studying the collected samples drives this unique partnership. “As a researcher whose daily work looks for ways to determine the early warning signs of head injury, I get to see first hand how committed Arizona State University and Riddell are to student-athlete safety, and their determination to improve the game at all levels.”

Following the season long campaign, the researchers will gather post-season data and begin the analysis process with their colleagues at Barrow Neurological Institute and A.T. Still University. During this process, TGen will work closely with Barrow, whose B.R.A.I.N.S. (Barrow Resource for Acquired Injury to the Nervous System) program treats patients who have sustained a traumatic brain or spinal cord injury. The Barrow data will provide the researchers with additional concussion data and allow for comparison between data sets.

Medical Technology - AZ Business Magazine January/February 2012

NIH awards BAI, Mayo $8.3 million

The National Institutes of Health (NIH) renewed funding for the Banner Alzheimer’s Institute (BAI) and Mayo Clinic, Phoenix, longitudinal study of the earliest changes associated with the risk of developing Alzheimer’s disease at older ages. The award, an estimated $8.3 million over the next five years, continues NIH’s long-term support of the investigation.

The study, which began two decades ago, has been examining the subtle brain imaging, memory and thinking changes that occur in healthy late-middle-aged and older adults who have inherited from their parents either one, two or no copies of the apolipoprotein E (APOE4) gene, the major genetic risk factor for developing late-onset Alzheimer’s. Each additional copy of the gene significantly increases a person’s chance of developing the disease.

“We are extremely grateful to the NIH and our wonderful research volunteers for their support,” said Dr. Eric M. Reiman, BAI Executive Director and one of the study’s principal investigators. “From the beginning, this study has been driven by our interest in finding treatments to prevent or end Alzheimer’s as quickly as possible, and to provide the information and tools needed to do just that.”

By studying individuals at three levels of genetic risk, researchers have been able to get a sneak peek at the changes associated with the risk of Alzheimer’s. As study participants begin to reach older ages, researchers hope to further clarify the extent to which characteristic brain imaging and other biological changes are associated with subsequent clinical decline. Additionally, researchers hope to further clarify the number of at-risk persons needed to conduct prevention trials, as well as share this valuable resource with other researchers and further develop the methods needed to test the range of promising treatments as quickly as possible.

This longitudinal study began in 1994, soon after researchers discovered the APOE4 gene’s contribution to the risk of developing Alzheimer’s. They have been following approximately 200 healthy volunteers with varying copies of the APOE4 gene, starting between the ages of about 50-65. Every two years, participants are monitored using an extensive battery of brain imaging, memory and thinking tests. A growing number of participants have also been providing cerebrospinal fluid samples. As many of the volunteers reach older ages, a growing number are now at risk for developing mild cognitive impairment (MCI) and dementia. This disease progression will give researchers the opportunity to characterize the extent of change in various biomarker and cognitive measurements. Data will be used to evaluate potential treatments that could combat amyloid plaques, which are strongly associated with Alzheimer’s, as well as help inform the design of future prevention trials.

“Like Dr. Reiman, I am excited about the opportunity we have been given to help advance the study of preclinical Alzheimer’s,” said Dr. Richard J. Caselli, Professor of Neurology at Mayo Clinic in Arizona and the study’s other principal investigator. “We also look forward to the chance to share our data and samples with other researchers to help advance the scientific fight against this terrible disease.”

The study has had a profound impact on Alzheimer’s prevention efforts. It has helped shape the field’s understanding of the progressive brain changes that precede the clinical onset of Alzheimer’s by almost two decades. It has also served as the foundation for the Alzheimer’s Prevention Initiative, an international collaborative formed to accelerate the evaluation of promising but unproven therapies. Data from this longitudinal study has also contributed to the development of the National Institute on Aging and Alzheimer’s Association research criteria for pre-clinical Alzheimer’s. It has also provided key information for the first reconceptualization of Alzheimer’s as a sequence of biological changes that progress over a person’s lifetime.

“By providing insights into the earliest Alzheimer’s-related changes to brain function and structure, this study is contributing to the National Plan to Address Alzheimer’ Disease goal of finding effective interventions by 2025,” said Dr. Neil Buckholtz, of the National Institute on Aging, which leads the NIH research program on Alzheimer’s.

This work also includes researchers from Arizona State University, University of Arizona and the Translational Genomics Research Institute, organizations that are partners in the Arizona Alzheimer’s Consortium. Dr. Eric M. Reiman of Banner Alzheimer’s Institute and Dr. Richard J. Caselli of Mayo Clinic are the two principal investigators.

Alzheimer’s is a debilitating and incurable disease that affects as many as 5 million Americans age 65 and older, according to a number of estimates. Without the discovery of successful prevention therapies, the number of U.S. cases is projected to nearly triple by 2050.

bioscience

Helios Scholars at TGen featured at symposium

The 45 interns in the 2014 Helios Scholars at TGen summer internship program graduated today, following a daylong scientific symposium at the Sheraton Phoenix Downtown Hotel.

Arizona’s future leaders in biology and medicine worked for eight weeks in one of the nation’s premier scientific internship programs, sponsored by the Helios Education Foundation in partnership with the Translational Genomics Research Institute (TGen).

At today’s symposium, students presented scientific posters and oral presentations about their biomedical investigations, which were conducted under the one-on-one guidance and mentorship of TGen researchers. Like their mentors, Helios Scholars use cutting-edge technology to help discover the genetic causes of diseases such as diabetes, Alzheimer’s disease, infectious disease and many types of cancer.

This is the eighth class of Helios Scholars at TGen, funded for 25 years by Helios Education Foundation. Helios is focused on creating opportunities for individuals to succeed in postsecondary education by advancing the academic preparedness of all students and fostering a high-expectations, college-going culture in Arizona and Florida.

“TGen’s summer intern program enables students to learn first-hand what it is like to work in a professional scientific environment, and helps them discover the skills they will need to make important contributions in science and medicine,” said Helios Education Foundation President and CEO Paul Luna. “The Helios Scholars at TGen program is helping prepare students for further academic success and for meaningful careers that not only benefit them, but will improve people’s lives through breakthrough medical and scientific research.”

The program is open to Arizona high school, undergraduate and graduate level students, including those in medical school.

“Our partnership with the Helios Education Foundation helps prepare a new generation of biomedical investigators for Arizona,” said Dr. Jeffrey Trent, TGen’s President and Research Director. “As we help them explore the biosciences beyond the classroom, TGen provides them with opportunities to participate in potentially life-changing research that can benefit actual patients.”

Helios Scholars also participate in professional development programs in science communication, public speaking, and basic business etiquette. This year’s interns were selected from among more than 500 applications.

“Our students arrive here with a passion for science and medicine,” said Julie Euber, TGen’s Education and Outreach Specialist and supervisor of the Helios Scholars at TGen. “Participating in authentic research projects helps shape their skills and abilities, preparing them for a lifetime of discovery and achievement in the biosciences.”

The program application opens in January of each year for the following summer at www.tgen.org/intern.

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TGen study provides new insights on HIV

A new study led by the Translational Genomics Research Institute (TGen) provides insights into the interplay among bacteria, viruses and the immune system during HIV infection.

Currently, doctors measure HIV-positive men’s infectivity — their potential to infect others — based on their blood viral load. However, some men produce large amounts of virus in their semen despite having low levels in their blood. Researchers call this “compartmentalization,” where different levels of the virus can be found in different parts of the body; in this case, in the semen, versus the blood.

Because of the importance of semen in HIV transmission — in both homosexual and heterosexual populations — researchers who conducted the study published today in the journal PLOS Pathogens sought to understand how HIV could be localized, or compartmentalized, in the semen.

Significantly, the study revealed a link between higher levels of HIV and higher levels of both bacteria and cytokines, biochemicals that can be described as the immune system’s alarm bells.

“Our study is trying to tackle an important problem in HIV research,” said Dr. Lance Price, Director of TGen’s Center for Microbiomics and Human Health, and one of the study’s senior authors. “We found that HIV infection affects the relationship between semen bacteria and immune system, and both are linked to semen HIV levels.”

These findings could point to new ways to control the spread of HIV, said Dr. Price. “Our data suggest that semen bacteria may play a role in localized inflammation and HIV viral load in the semen, which is an important target for reducing HIV transmission.”

While HIV can be found in many body fluids (semen, vaginal secretions, blood and breast milk), it is most commonly transmitted via semen in both homosexual and heterosexual sex.

Homosexual men were the focus of this study because they continue to be the population most at risk for HIV in North America. The study included 27 homosexual men infected with HIV, and 22 homosexual men who were uninfected.

The 27 infected individuals were examined before treatment, and at both one-month and six-month intervals following treatment with anti-retroviral therapy (ART). Samples from the 22 uninfected participants in the study served as controls.

“By comparing the semen bacteria in both uninfected and infected men, we found that HIV can cause an imbalance in the semen microbiome,” said Dr. Cindy Liu, the study’s lead author, a clinical pathology resident at the Johns Hopkins School of Medicine, and an adjunct professor at TGen at the time of the study. “This imbalance can be corrected by HIV treatment. This suggests that there are complex host-microbe interactions in the semen.”

“We have taken the first step to show that bacteria, HIV, and immune response in the semen may be connected,” said Dr. Rupert Kaul, an immunologist from the University of Toronto, and another senior author of the study. “What we need to better understand next is the precise relationship between these three factors — whether the bacterial imbalance is the trigger or the result of the localized immune response.”

The authors of the study, The Semen Microbiome and Its Relationship with Local Immunology and Viral Load in HIV Infection, are particularly excited about what this research may mean for another important patient population.

“Even though we have focused on men whose semen viral load can be controlled with HIV treatment in this study,” said Dr. Kaul, “we need to study men who continue to have high HIV levels in their semen despite being on treatment. This will be important to improving how we treat patients and control the spread of HIV.”

medical.research

TGen study finds origin of lung fungus

Cryptococcus gattii, a virulent fungus that has invaded the Pacific Northwest is highly adaptive and warrants global “public health vigilance,” according to a study by an international team led by the Translational Genomics Research Institute (TGen).

C. gattii, which likely originated in Brazil, is responsible for dozens of deaths in recent years since it was first found in 1999 on Vancouver Island, British Columbia, Canada, well outside its usual tropical habitats.

“We identified several genes that may make the outbreak strains more capable of surviving colder environments and that make it more harmful in the lungs,” said David Engelthaler, Director of Programs and Operations for TGen’s Pathogen Genomics Division (TGen North) and lead author of the study published today in the scientific journal mBio.

This study should form the basis of additional investigations about how and why C. gattii disperses and emerges. It identified several new genomic targets for diagnostic tests, and possible new targets for therapeutic drugs and preventative vaccines.

“By closely analyzing the genomes of dozens of outbreak strains, as well as globally diverse strains, we were able to closely compare and determine the genomic differences that may cause their clinical and ecological changes,” said Dr. Paul Keim, one of the study’s senior authors. Dr. Keim also is Director of TGen North, and Director of the Microbial Genetics and Genomics Center at Northern Arizona University (NAU).

TGen, working with the U.S. Centers for Disease Control and Prevention and others, conducted one of the largest global fungal genome analyses of a specific species to understand its emergence in new environments. The collaborative team included 24 researchers from 13 institutions in seven nations who sequenced 115 genomes of C. gattii collected from 15 countries.

“By thinking globally, we were able to better understand what was happening locally,” Engelthaler said.

C. gattii was typically a tropical fungus before it was discovered in the temperate environs of Vancouver Island. It soon evolved into a new, more virulent, pulmonary disease that quickly spread to mainland Canada and south into the state of Washington. That was followed by an outbreak in Oregon of another new strain of C. gattii, which also displayed increased lethality and similarly spread throughout the Pacific Northwest.

C. gattii previously was associated with neurological disease in strains found elsewhere in the world. But the strains discovered in the Pacific Northwest not only establish a new environmental niche, but also display increased virulence and produce severe lung infections.

“We provide evidence that the Pacific Northwest strains originated from South America, and identified numerous genes potentially related to habitat adaptation, virulence expression and to clinical presentation,” said Dr. Wieland Meyer, the study’s other senior author.

“Further elucidation and characterization of these genetic features may lead to improved diagnostics and therapies for infections caused by this continually evolving fungus,” said Dr. Meyer, who is affiliated with: Sydney Medical School-Westmead Hospital; the University of Sydney; and the Westmead Millennium Institute for Medical Research.

This study concludes that: “Public health vigilance is warranted for emergence in
regions where C. gattii is not thought to be endemic.”

New tests developed for this study by TGen are making it easier to detect this and other fungi, and could lead to better monitoring and treatments. The same tools used in this study also were used to investigate the cause of a fungal meningitis outbreak associated with steroid back injections, and the recent outbreak of Valley Fever in the state of Washington.

The journal, mBio, is published by the American Society for Microbiology.

Collaborators in this study include researchers at the University of California-Davis, and in the United Kingdom, Australia, New Caledonia, Thailand, Columbia, and Brazil.

The study, “Cryptococcus gattii in North American Pacific Northwest: whole population genome analysis provides insights into species evolution and dispersal,” was supported by a grants from the National Institutes of Health (NIH) and the Medical Research Council (MRC) of South Africa.

Research reported in this publication was supported by the National Institute of Allergy and Infectious Diseases of the NIH under Award Number R21AI098059. The content is solely the responsibility of the authors and does not necessarily represent the official views of the NIH.

bioscience

TGen teams up with Ceres Nanosciences

The Translational Genomics Research Institute (TGen) and Ceres Nanosciences, Inc. (Ceres) today announced a development collaboration to benefit patients with cancer, infectious diseases, and other life-threatening illnesses.

TGen, at the heart of Phoenix’s Biomedical Campus, is recognized as a pioneer in studying genomics, or DNA, to uncover the underlying molecular causes of disease, as well as developing automated workflows to enable molecular medicine. Ceres, a biotechnology company located in northern Virginia, has developed and commercialized a novel nanotechnology, the “Nanotrap®”, which provides powerful sample processing capabilities for a wide array of diagnostic applications and sample handling needs.

The Nanotrap technology was invented at George Mason University (Mason) under funding from the National Institutes of Health (NIH) for biomarker discovery applications, and currently is being developed into commercial grade products by Ceres with continuing support from NIH, Defense Advanced Research Projects Agency (DARPA), Department of Homeland Security (DHS), and the Commonwealth of Virginia.

The combination of Ceres’ Nanotrap technology and TGen’s world-class research capabilities and resources will lead to significant improvements in high sensitivity and high throughput clinical diagnostics, providing substantial patient benefit with newer, more accurate, and more reliable testing methods for a range of life-threatening diseases.

The collaboration has already produced data that demonstrates the Nanotrap particles’ performance in automated high-throughput biomarker discovery applications. Ceres, TGen and Mason will use the exciting preliminary results to seek additional collaborations and support to develop an array of new sample processing and diagnostic solutions.

“There is great patient demand and clinical utility for new, groundbreaking diagnostic tests for the early detection of cancer and other life-threatening diseases,” said Ross Dunlap, CEO of Ceres. “The biggest challenge to delivering these tests is building the scale and efficiency required to make these new tests cost-effective. Speed and throughput coupled with even higher analytical sensitivities than have ever been achieved before is the key. The collaboration between Ceres and TGen is already addressing this challenge by integrating advanced automation solutions, analytical platforms, and sample processing technologies to deliver groundbreaking protein and DNA based clinical tests.”

“TGen’s collaboration with Ceres enables additional research to expand our work into other disease settings,” said Dr. Michael Berens, TGen Deputy Director for Research Resources, “and Nanotrap allows us to do so by functioning at the fastest and most-sensitive levels while using only minimally- or non-invasive patient samples.”

medical.research

TGen, SHC begin clinical trial for anti-tumor drug

The Virginia G. Piper Cancer Center at Scottsdale Healthcare and the Translational Genomics Research Institute (TGen) are studying the safety and effectiveness of a new drug, AG-120, for treatment of patients with solid tumors, especially those with brain tumors and gallbladder bile duct cancer.

“AG-120 is designed specifically for those patients who carry the IDH1 gene mutation,” said Dr. Daniel D. Von Hoff, Distinguished Professor and Physician-In-Chief at TGen, and Chief Scientific Officer for the Virginia G. Piper Cancer Center Clinical Trials at Scottsdale Healthcare, a partnership between Scottsdale Healthcare and TGen that delivers new treatments to cancer patients based on precision medicine.

The IDH1 gene mutation is most commonly found in gliomas, which make up the largest group of “primary” brain tumors, those that start in the brain. Gliomas include all tumors arising from the gluey or supportive tissue of the brain. They represent about 30 percent of all brain tumors, and about 80 percent of all malignant brain tumors.

As with all brain cancers, gliomas are difficult to treat and many grow back after surgery, radiation and standard of care chemotherapy. Many drugs cannot get to the brain because of a filtering mechanism in the body called the blood-brain barrier.

“There is a great need for more effective treatments for patients with gliomas and other solid tumors, such as gallbladder bile duct cancer,” said Dr. Von Hoff, who is the Principal Investigator for AG-120 clinical trial. This study will enroll as many as 50 patients. Researchers will use precision medicine to match treatments to patient’s specific genomic, or molecular, makeups.

Mutations in isocitrate dehydrogenase (IDH) 1 and 2, originally discovered in 2008, occur in the vast majority of low-grade gliomas and secondary high-grade gliomas. IDH mutations are oncogenic, meaning they have the potential to cause cancer. These mutations occur early in the formation of gliomas and in gallbladder bile duct cancer.

AG-120 is produced by Agios Pharmaceuticals Inc., based in Cambridge, Mass.

IDH1 is a metabolic enzyme identified by Agios as a protein that is mutated in a wide range of malignant tumors. Agios and its collaborators recently demonstrated that IDH1 mutations initiate and drive cancer growth by blocking differentiation, or maturation, of primitive cells. According to Agios, the inhibition of these mutated proteins may lead to clinical benefit for those cancer patients whose tumors carry them.