Tag Archives: translational genomics research institute

bioscience

TGen's Zenhausern is named an NAI fellow

Dr. Frederic Zenhausern, a Professor at the Translational Genomics Research Institute (TGen) and developer of a rapid DNA processor, has been named to the Fellows of the National Academy of Inventors (NAI).

Zenhausern, Ph.D., MBA, will be inducted into the NAI Fellows by Deputy U.S. Commissioner for Patents Andy Faile, from the U.S. Patent and Trademark Office, during the 3rd Annual Conference of the National Academy of Inventors, March 7, 2014, in Alexandria, Va.  Fellows will be presented with a special trophy and a rosette pin.

Zenhausern also is a Professor and Director of the Center for Applied Nanobioscience and Medicine at the University of Arizona College of Medicine – Phoenix. The center uniquely applies a combination of advances in nanoscience, microelectromechanical systems, molecular biology, and genomics to a new generation of biological tools and sensors based on nano and microscale technologies.

Applying interdisciplinary science approaches to medicine, Zenhausern’s work is aimed at early diagnostics of human diseases, in particular, integrating bioassay chemistries with several biomarkers and targets developed by researchers at TGen.. Over the years, his team has collaborated on multiple projects at TGen for designing bioanalytical  platform technologies to translate molecular analysis into clinical tools and, ultimately, promoting adoption of novel technologies for point-of-care diagnostics applications.

The Microfluidic DNA Analysis System (MiDAS), a desktop printer-sized box that is described as robust and user-friendly, is one of Zenhausern’s innovations. The integrated DNA analyzer can be transported directly to a point-of-care or deployed in a mobile setting, eliminating some of the issues that arise when collecting and shipping a specimen from a remote site to a centralized laboratory for molecular testing. The core technology is also enabling the rapid automation of preparation of a biological sample for interfacing with various high-resolution analytical instrumentations, such as Next Generation Sequencing. These emerging diagnostic tools in personalized medicine are being used the team of clinicians at TGen Clinical Translational Research Division.

A similar platform was configured for genomic assays ready for implementations in medical countermeasures against radiological and nuclear disasters, and also applicable in clinical settings for predicting which patients are most sensitive to radiation in guiding personalized treatment, and preventing the development of toxicities that may result from radiotherapy. These innovations are described in multiple patents with TGen co-inventors, which led to significant federal funding of Arizona academic institutions, and to generating commercial interest and licensing from multiple U.S. companies, also contributing to Arizona’s economic development.

Zenhausern has co-authored more than 70 scientific publications and is named on many pending and more than a dozen issued U.S. patents in various domains ranging from DNA sequencing to optical data storage. Zenhausern’s responsibilities also include leading clinical research at the personalized medicine research laboratory at Scottsdale Healthcare Research Institute and serving on several corporate scientific boards and international consortia in life sciences.

Election to NAI Fellow status is a high professional distinction accorded to academic inventors who have demonstrated a prolific spirit of innovation in creating or facilitating outstanding inventions that have made a tangible impact on quality of life, economic development, and the welfare of society.

The 143 innovators elected to NAI Fellow status represent 94 universities and governmental and non-profit research institutes.  Together, they hold more than 5,600 U.S. patents.

Included in the 2013 class are 26 presidents and senior leadership of research universities and non-profit research institutes, 69 members of the National Academies, five inductees of the National Inventors Hall of Fame, six recipients of the U.S. National Medal of Technology and Innovation, and nine Nobel Laureates among other major awards and distinctions.

Academic inventors and innovators elected to the rank of NAI Fellow were nominated by their peers for outstanding contributions to innovation in areas such as patents and licensing, innovative discovery and technology, significant impact on society, and support and enhancement of innovation.

brain

TGen-Barrow-PCH study brain injuries

In an effort to lower medical costs, identify patients at risk for injury, and speed patient recovery, scientists will attempt to identify a molecular signal that indicates severity of brain-injury during a $4 million, five-year federal grant to Barrow Neurological Institute at St. Joseph’s Hospital and Medical Center, Phoenix Children’s Hospital and the Translational Genomics Research Institute (TGen).

The molecular profile — comprised of RNA, the body’s relay messenger between DNA and protein — could help identify which patients are most at risk for vasospasm after hemorrhagic stroke.  Hemorrhagic stroke can occur as:

•    Subarachnoid hemorrhage, or the bleeding into the area between the brain and a thin membrane that covers it.
•    Ruptured brain aneurysm, which is an abnormal bulge or ballooning in the wall of an artery within the brain.

By identifying this RNA molecular marker, a new standard of individualized care could be established, enabling medical teams to respond more rapidly to quickly changing health conditions, and allowing earlier intervention to prevent a secondary injury from occurring.

“We hope this study will lead to less injury, less testing and cost, and shorter stays in the hospital,” said Dr. Yashar Kalani, M.D. and Ph.D., a resident physician in Neurological Surgery and assistant professor at the Barrow Neurological Institute and one of the study’s principal investigators. Additional investigators at Barrow include Drs. Robert Spetzler, Peter Nakaji, Felipe Albuquerque and Cameron McDougall.

Vasospasms are characterized by bleeding in the brain that causes irritation and nearby blood vessels to spasm and narrow. This decreases blood flow to the brain, which can result in damage or even death to parts of the brain.

Only about half of patients with brain-aneurysm ruptures survive, and those who do survive often are severely disabled for life. In the 10 days following such ruptures, blood vessels can narrow, leading to loss of oxygen, strokes and brain damage.

“If we knew what is happening during this period, we might be able to intervene and prevent the secondary injury,” Dr. Kalani said.

Barrow will provide patient care and collect blood and spinal fluid samples that will be analyzed by TGen. A recent TGen study showed spinal fluid could be sequenced for RNA biomarkers. Samples will be checked daily to compare and identify changes.

Another part of the study will be conducted at Barrow’s partnership with Phoenix Children’s Hospital, where researchers will investigate the effects of intraventricular hemorrhage — another form of bleeding in the brain — in newborn babies. Intraventricular hemorrhage in newborns occurs secondary to diminished blood flow and oxygen delivery to the brain. Intraventricular hemorrhage is associated with the development of hydrocephalus and damage to the brain that can result in cerebral palsy or other types of motor and cognitive delays.

“This study will get us one step closer to learning what is unique in pediatric stroke so we can provide the best quality care and improve the long term outcomes for these children,” said Dr. P. David Adelson, one of the principal investigators of the study at Barrow Neurological Institute at Phoenix Children’s Hospital.

“In addition, as this study progresses, we want to know how to identify children at risk, and how they differ from adults with similar conditions, this will not only help us to be more accurate at providing current treatments but to develop new ones.” said Dr. Jorge Arango, an investigator affiliated with Barrow Neurological Institute at Phoenix Children’s Hospital and with the University of Arizona College of Medicine-Phoenix.

In the study of both adults and children, TGen researchers will use state-of-the-art sequencing — to analyze RNA transcripts, searching for biomarkers that could identify at-risk patients.

RNAs are cell molecules made from DNA that help create proteins.

“There has been an explosion over the last several years in our understanding of the functional and regulatory mechanisms modulated by RNA” said Dr. Kendall Van Keuren-Jensen, Ph.D., an Assistant Professor in TGen’s Neurogenomics Division and also a principal investigator in the study funded by the National Institutes of Health (NIH).

“We are very excited about the potential for extracellular RNAs to provide us with accessible information about the mechanism of disease, and in doing so, provide us with pre-symptomatic markers of disease,” said Dr. Matt Huentelman, Ph.D., an Associate Professor in TGen’s Neurogenomics Division and also a principal investigator on the project. “In the best-case scenario, these markers can be coupled with an improved clinical management of the disease, too. In a nutshell, that is what we are exploring under this new grant award.”

This type of study is now possible because of continuing improvements in optics and computer speed that enables TGen’s cutting-edge technology to sequence at ever-faster rates and at ever-lower costs. While it took 13 years and $2.7 billion to spell out the first human genome, such sequencing can now be done in a matter of days and for less than $5,000.

Additional partners in the study include: University of California, San Francisco; and Stanford University.

stem.cell

TGen-led study finds link to Parkinson’s disease

The absence of a protein called SMG1 could be a contributing factor in the development of Parkinson’s disease and other related neurological disorders, according to a study led by the Translational Genomics Research Institute (TGen).

The study screened 711 human kinases (key regulators of cellular functions) and 206 phosphatases (key regulators of metabolic processes) to determine which might have the greatest relationship to the aggregation of a protein known as alpha-synuclein, which has been previously implicated in Parkinson’s disease. Previous studies have shown that hyperphosphorylation of the α-synuclein protein on serine 129 is related to this aggregation.

“Identifying the kinases and phosphates that regulate this critical phosphorylation event may ultimately prove beneficial in the development of new drugs that could prevent synuclein dysfunction and toxicity in Parkinson’s disease and other synucleinopathies,” said Dr. Travis Dunckley, a TGen Assistant Professor and senior author of the study.

Synucleinopathies are neurodegenerative disorders characterized by aggregates of α-synuclein protein. They include Parkinson’s, various forms of dementia and multiple systems atrophy (MSA).

The study — SMG1 Identified as a Regulator of Parkinson’s disease-associated alpha-Synuclein Through siRNA Screening — was published today in the journal PLOS ONE.

By using the latest in genomic technologies, Dr. Dunckley and collaborators found that expression of the protein SMG1 was “significantly reduced” in tissue samples of patients with Parkinson’s and dementia.

“These results suggest that reduced SMG1 expression may be a contributor to α-synuclein pathology in these diseases,” Dr. Dunckley said.

TGen collaborators in this study included researchers from Banner Sun Health Institute and Mayo Clinic Scottsdale.

Tissue samples were provided by the Banner Brain and Body Donation Program. The study was funded by the Arizona Parkinson’s Disease Consortium, which includes Mayo Clinic Scottsdale, Sun Health Research Institute, Barrow Neurologic Institute, Banner Good Samaritan Medical Center, Arizona State University, and TGen.

The study is available at: http://dx.plos.org/10.1371/journal.pone.0077711.

quayle

Quayle elected to TGen Foundation Board

Former U.S. Vice President Dan Quayle, who served under President George H.W. Bush from 1989-93, was elected today to the Board of Directors of the TGen Foundation, the philanthropic arm of the non-profit Translational Genomics Research Institute (TGen).

Vice President Quayle attended grade school and high school in Phoenix and Scottsdale and he and his wife, Marilyn, are now residents of Paradise Valley. He brings to the TGen Foundation his vast career experience in politics and financial investment.

“It is my honor to be selected to be a part of TGen and the phenomenal work this biomedical institute is doing to find better treatments for the most serious diseases affecting humanity,” said Quayle, who also is a former U.S. representative and senator from Indiana, and who today is chairman of Cerberus Global Investments, a private equity company with $25 billion under management.

TGen Foundation Board Chairman Bennett Dorrance welcomed Quayle, noting that he is instantly one of the best known among a cadre of top-flight Arizonans who serve on the non-profit panel.

“Today we welcome Vice President Dan Quayle to our TGen family with high expectations and confidence that he will further enhance our philanthropic reach across the nation and help fuel TGen’s genomic research of the world’s most pressing diseases,” Dorrance said. “We welcome his involvement, his extraordinary relationships and his business acumen.”

Dr. Jeffrey Trent, TGen President and Research Director, also welcomed Quayle, whose term as Vice President (1989-1993) coincided with Dr. Trent serving as Scientific Director of the National Human Genome Research Institute at the National Institutes of Health (NIH) in Bethesda, Maryland.

“In 1990, the NIH and the Department of Energy joined with international partners in a quest to sequence all 3 billion letters in the human genome. Vice President Quayle is acutely aware of the importance of this public effort, and remains an advocate for genomic research and what is means for our patients,” Dr. Trent said.

TGen Foundation President Michael Bassoff said that the addition of Quayle to the TGen Foundation Board of Directors would undoubtedly be of huge importance to the future of the institute.

“He brings a powerful internationally recognized voice to advance TGen’s scientific research,” Bassoff said.

Quayle graduated from DePauw University in 1969, and received his law degree from Indiana University in 1974.

He was first elected to the U.S. House of Representatives in 1976 at age 29; to the U.S. Senate in 1980 at age 33; and Vice President of the United States in 1988 at age 41, during which he made official visits to 47 nations and served as chairman of the National Space Council. He has authored three books, including Standing Firm, a vice-presidential memoir, which was on The New York Times best-seller list for 15 weeks.

Quayle also was a distinguished visiting professor of international studies at Thunderbird, The American Graduate School of International Management in Glendale, Arizona.

At Cerberus, one of the world’s leading investment firms, he has been actively involved in new business sourcing and marketing in North America, Europe and Asia. His extensive global network of public and private sector decision-makers, combined with his investment expertise, has significantly contributed to the growth of Cerberus.

bioscience

17th U.S. Surgeon General joins TGen advisory panel

The 17th Surgeon General of the United States and President of Canyon Ranch Institute Richard H. Carmona, M.D., M.P.H., FACS, today joined the Translational Genomics Research Institute (TGen) advisory committee on rare childhood disorders.

Dr. Carmona’s role on the National Advisory Committee for TGen’s Center for Rare Childhood Disorders (the Center) will be to help incorporate genomic technologies into the standard of care.

The Center uses rapid genomic tests to discover the genetic source of childhood ailments. Many of these rare disorders have no name, and often are just a collection of symptoms with no apparent cause.

Dr. Carmona is the keynote speaker today (Oct. 15) as TGen celebrates the opening of its clinic for the Center at 3330 N. 2nd Street, Suite 402, Phoenix, Ariz.

“Modern scientific advances are uncovering the nature and causes of disease like no other point in history,” said Dr. Carmona. “I am honored to be invited to help guide TGen’s development of this unique Center — focused on children — that uses the latest DNA technology to understand and diagnose rare disorders at the molecular level and to develop treatments for patients who previously had few, if any, options for care.

“Throughout my career, I’ve had the privilege of studying and applying genetic advances to improve the lives of my patients, and as Surgeon General of the United States, my team and I developed the successful ‘Surgeon General’s Family History Initiative’ with colleagues throughout the U.S. Department of Health and Human Services to help more people connect the concept of family health history and genetics to their own lives.”

The Center’s National Advisory Committee (NAC) is a core group of advisors and supporters with a passion for helping children and families affected by rare and neglected pediatric diseases and disorders.

Dr. Carmona will serve on the NAC’s Scientific-Medical Advisory Sub-Committee, which focuses on helping geneticists and health care professionals to collaborate in order to incorporate genomic technologies into the standard of care.

“Dr. Carmona brings to TGen a wealth of experience, forged through first-hand treatment of patients and by driving innovative public health programming at the individual, national, and global levels,” said NAC Co-Chair David Harbour.

“We welcome Dr. Carmona as an internationally recognized physician and health policy expert, whose knowledge and experience will help the Center bring hope and answers to our young patients and their families,” said NAC Co-Chair Jacquie Dorrance.

Born to a poor Hispanic family in New York City, Dr. Carmona experienced homelessness, hunger, and health disparities during his youth. The experiences greatly sensitized him to the relationships among culture, health, education and economic status and shaped his future.

After dropping out of high school, Dr. Carmona enlisted in the U.S. Army in 1967. While serving, he earned his General Equivalency Diploma and went on to become a combat-decorated Special Forces Vietnam veteran. After leaving active duty, he attended Bronx Community College of the City University of New York through an open enrollment program for veterans. He received an associate of arts degree. He then attended the University of California, San Francisco, where he received a bachelor of science degree (1977) and medical degree (1979). At the University of California Medical School, Dr. Carmona was awarded the prestigious gold-headed cane as the top graduate.

Trained in general and vascular surgery, Dr. Carmona also completed a National Institutes of Health-sponsored fellowship in trauma, burns, and critical care. A Fellow of the American College of Surgeons, Dr. Carmona was recruited jointly by the Tucson Medical Center and the University of Arizona to start and direct Southern Arizona’s first regional trauma care system. Dr. Carmona would later become chairman of the State of Arizona Southern Regional Emergency Medical System, a professor of surgery, public health, and family and community medicine at the University of Arizona, and the Pima County Sheriff’s Department Surgeon.

In 2002, Dr. Carmona was nominated by the president and unanimously confirmed by the U.S. Senate to become the nation’s 17th Surgeon General. Dr. Carmona was selected because of his extensive experience in public health, clinical sciences, health care management, preparedness, and his commitment to prevention as an effective means to improve public health and reduce health care costs while improving the quality and quantity of life.

As Surgeon General, Dr. Carmona focused on prevention, preparedness, health disparities, health literacy, and global health to include health diplomacy. He also issued many landmark Surgeon General communications during his tenure, including the definitive Surgeon General’s Report about the dangers of second-hand smoke.

Dr. Carmona has published extensively and received numerous awards, decorations, and local and national recognitions for his achievements. A strong supporter of community service, he has served on community and national boards and provided leadership to many diverse organizations.

In 2006, Dr. Carmona became Vice Chairman of Tucson-based Canyon Ranch, a leader in the health and wellness field and President of Canyon Ranch Institute, a 501(c)3 non-profit public charity dedicated to catalyzing the possibility of optimal health for all people by translating the best practices of Canyon Ranch and its partners to help educate, inspire, and empower every person to prevent disease and embrace a life of wellness.

Dr. Carmona is also the first Distinguished Professor of Public Health at the University of Arizona’s Mel and Enid Zuckerman College of Public Health and the first Dean’s Distinguished Professor of Health Promotion and Entrepreneurship at The Ohio State University College of Nursing.

stem.cell

TGen identifies genes linked to unhealthy liver function

A groundbreaking study of nearly 2,300 extremely obese diabetes patients, led by the Translational Genomics Research Institute (TGen), has identified genes associated with unhealthy liver function.

This is believed to be the nation’s first large-scale genome-wide association study in overweight patients with diabetes.

Results of the study, done in conjunction with the Geisinger Health System, will be presented at the 64th annual meeting of the American Association for the Study of Liver Diseases Nov. 1-5 at the Walter E. Washington Convention Center in Washington, D.C.

The study — Genome-wide analysis identifies loci associated with total bilirubin levels, steatosis, and mild fibrosis in nonalcoholic fatty liver disease — looked at how genomic factors affect the development of non-alcoholic fatty liver disease. It was selected for presentation from among a record 3,139 submittals from around the world proposed for what also is known as The Liver Meeting 2013.

“These genetic factors could help us identify patients who are most at risk of developing non-alcoholic forms of fatty-liver disease (NAFLD), and which patients may be more likely to progress to severe forms of NAFLD, such as steatohepatitis (NASH),” said Dr. Johanna DiStefano, the study’s principal investigator and lead author. Dr. DiStefano is Director of TGen’s Diabetes, Cardiovascular and Metabolic Diseases Division.

NAFLD is the build up of extra fat in liver cells, not caused by alcohol. It is one of the most common causes of chronic liver disease. NASH is liver inflammation and damage caused by a buildup of fat in the liver, not caused by alcohol.

“Our results showed evidence for new genetic loci that may play a role in the biological mechanisms of NAFLD and NASH,” said Dr. Glenn S. Gerhard, a faculty member of the Geisinger Obesity Institute and a co-investigator of the study.

“We discovered genes that may help identify those patients most at risk for the types of liver disease so severe that they could require transplants,” said Dr. Gerhard, Administrative Director for the Institute for Personalized Medicine at Penn State University-Hershey.

Patients included in this study were those with extreme obesity enrolled in a bariatric surgery program.

The study identified evidence for association with markers in the neurocan gene (NCAN) on chromosome 19p12, and rs2501843 on chromosome 1.

The American Association for the Study of Liver Diseases (AASLD) is the leading organization of scientists and healthcare professionals committed to preventing and curing liver disease. AASLD was founded in 1950 by a small group of leading liver specialists to bring together those who had contributed to the field of hepatology.

AASLD has grown to an international society responsible for all aspects of hepatology. Its annual meeting, The Liver Meeting, has grown in attendance from 12 to more than 9,500 physicians, surgeons, researchers, and allied health professionals from around the world.

stem.cell

TGen researchers uncover root of myeloma relapse

Researchers have discovered why multiple myeloma, a difficult to cure cancer of the bone marrow, frequently recurs after an initially effective treatment that can keep the disease at bay for up to several years.

Working in collaboration with colleagues at Princess Margaret Hospital in Toronto, researchers from Mayo Clinic in Arizona and the Translational Genomics Research Institute (TGen) in Phoenix were part of the team that conducted the study published in the Sept. 9 issue of Cancer Cell.

The research team initially analyzed 7,500 genes in multiple myeloma cells to identify genes which when suppressed made cancer cells resistant to a common class of drugs called proteasome inhibitors such as bortezomib or carfilzomib. Then, the team studied bone marrow biopsies from patients to further understand their results. The process identified two genes (IRE1 and XBP1) that control response to the proteasome inhibitor and the mechanism underlying the drug resistance that is the barrier to cure.

The findings showed recurrence was due to an intrinsic resistance found in immature tumor progenitor (mother) cells is the root cause of the disease and also spawns relapse. The research demonstrates that although the visible cancer cells that make up most of the tumor are sensitive to the proteasome inhibitor drug, the underlying progenitor cells are untouched by this therapy. These progenitor cells then proliferate and mature to reboot the disease process, even in patients who appeared to be in complete remission.

“Our findings reveal a way forward toward a cure for multiple myeloma, which involves targeting both the progenitor cells and the plasma cells at the same time,” says Rodger Tiedemann, M.D., a hematologist specializing in multiple myeloma and lymphoma at Princess Margaret. “Now that we know that progenitor cells persist and lead to relapse after treatment, we can move quickly into clinical trials, measure this residual disease in patients, and attempt to target it with new drugs or with drugs that may already exist.”

“Some myeloma cells are too immature to be caught by the drugs and thus hide underground only to reemerge later,” says Keith Stewart, M.B., Ch.B., Dean for Research at Mayo Clinic in Arizona and contributor to the study. “This study has wide implications in the search for a cure of this common blood cancer as this ‘progenitor cell’ will have to be targeted.”

Jonathan Keats, Ph.D., head of TGen’s Multiple Myeloma Research Laboratory, said: “This study, which leverages data generated at TGen as part of the Multiple Myeloma Genomics Initiative, shows how mutations acquired by multiple myeloma tumors can make a tumor resistant to specific therapies and highlights the importance of TGen’s precision medicine approaches.”

Dr. Tiedemann says: “If you think of multiple myeloma as a weed, then proteasome inhibitors are like a goat that eats the mature foliage above ground, producing a remission, but doesn’t eat the roots, so that one day the weed returns.”

The study — Xbp1s-Negative Tumor B Cells and Pre-Plasmablasts Mediate Therapeutic Proteasome Inhibitor Resistance in Multiple Myeloma — was funded by the National Cancer Institute, Multiple Myeloma Research Foundation, Leukemla and Lymphoma Society and Canadian Cancer Society, the Arthur Macaulay Cushing Estate and The Princess Margaret Cancer Foundation.

Dr. Tiedemann is the Molly and David Bloom Chair in Multiple Myeloma Research, at the University of Toronto, Dr. Stewart is the Anna Maria and Vasek Pollack Professor of Cancer Research at Mayo Clinic. Dr. Keats is an Assistant Professor in TGen’s Integrated Cancer Genomics Division.

medical.research

TGen Foundation plans two major fundraising events

The Translational Genomics Research Institute (TGen) Foundation plans two climate-appropriate fundraising events — “Shop for a Cause” while it’s still hot, and “stepNout” when it cools off this fall.

Mark your calendars for TGen’s 8th annual stepNout Run/Walk/Dash set for Nov. 3 at Kiwanis Park, 6111 S. All-America Way, in Tempe. Nearly 1,000 participants are expected.

The TGen Foundation this year is looking for corporate sponsors that might help push stepNout’s total fundraising to $1 million. Opportunities to showcase sponsors’ support are available through TGen’s website, Facebook, race-day t-shirts and other media.

In its previous seven years, stepNout has generated nearly $600,000 for TGen’s groundbreaking research of pancreatic cancer, the nation’s fourth leading cause of cancer death.

More information and registration for stepNout is available at tgenfoundation.org/step. Sponsorships are available by contacting Andrea Kobielski, Development Associate for TGen Cancer Programs, at akobielski@tgen.org or 602-343-8572.

Meantime, Shop for a Cause is Macy’s 8th annual discount shopping event, providing customers with a fabulous shopping experience while fostering worthy non-profit charities, such as TGen.

For a $5 donation to TGen, shoppers may take up to 25 percent off most purchases during a one-day sale on Aug. 24. (Some exclusions apply.)

Get your savings pass at tgen.org/tgen-foundation. Click on Shop for a Cause. Deadline is noon Aug. 23. All — 100 percent — of your $5 donation will go to TGen research.

“Giving back is a key component of Macy’s culture. We are honored to offer our customers an easy and convenient way to make a positive difference in their communities and in the lives of others, while enjoying great savings at Macy’s,” said Martine Reardon, Chief Marketing Officer for Macy’s, which since 2006 has raised $46 million for charities nationwide.

StemCellSciCamp08_5619

$20,000 APS grant funds TGen education initiative

A $20,000 grant from the APS Foundation will help the Translational Genomics Research Institute (TGen) expand its TGen2School initiative by providing science kits and instruction in Science, Technology, Engineering and Math (STEM) education.

The kits and accompanying instruction for teachers are part of the TGen2School initiative at TGen’s Pathogen Genomics Division – TGen North – in Flagstaff, where some of the world’s top experts in disease-causing microorganisms study everything from valley fever to MRSA and even anthrax and plague.

TGen North’s Bio-SEEK: Bio-Science Education Enrichments Kits Program provides five different types of bioscience education kits for teachers and their students. The goal is improved overall scientific literacy, and a better-prepared bioscience workforce.

The program includes instructional sessions to help educators use the kits to teach such concepts as infectious disease and genomic testing methods, biosafety procedures, bioinformatics, and how DNA is used in forensics, public health and other life sciences.

“These are ideal tools that teachers can use to convey complex concepts in ways students can easily absorb, and it lessens the burden on the pocketbooks of teachers,” said Zsuzsi Kovacs, TGen North’s STEM Education Coordinator. “These kits are built on next-generation science standards and bioscience basics that students need to succeed in the genome-age.”

TGen will provide instruction for teachers during professional development days at TGen North, 3051 W. Shamrell Blvd., southeast of Interstate 17 and the exit to the Flagstaff Pulliam Airport.

“Commercial bioscience kits often contain limited directions, making teaching concepts challenging when teachers already have so much on their plate,” Kovacs said. “With professional development and teacher-friendly directions, educators will be able to adapt them in a way that is best for their students.”

Thanks to the APS Foundation’s grant, the newly developed kits will be provided at no charge through a checkout system available to teachers who have attended the professional training.

TGen2School initiative aligns with the goals of David Engelthaler, TGen North’s Director of Programs and Operations, one of the leaders in STEM education in Flagstaff, which in 2012 became the nation’s first STEM City.

“With initial funding from the Flagstaff Community Foundation (FCF) and others, we have placed a concerted effort into our TGen2School program,” said Engelthaler, a former State Epidemiologist for the State of Arizona. “We are so excited that the APS Foundation has decided to help us. Their grant will allow us to grow and expand our program in a direction that better meets the needs of our teachers.”

The grant to TGen North was one of 15, totaling more than $500,000, made by the Foundation to non-profit organizations throughout Arizona and New Mexico. Of the 30 fastest growing occupations projected through 2016, more than half will require mastery of STEM subjects, according to the U.S. Bureau of Labor Statistics.

“We at the APS Foundation applaud the efforts of all the organizations who received the grants,” said Julie Coleman, Executive Director of the APS Foundation. “We are pleased to be able to help support and encourage non-profits who engage in promoting STEM education, and other educational efforts, to increase student achievement. Success in education will result in a healthy society, strong economy and robust Arizona.”

Krauss Honored For Science

Scientific internship helps build Arizona’s biomedical workforce

Arizona’s future leaders in biology and medicine graduated today from one of the nation’s premier scientific internship programs, sponsored by Helios Education Foundation and the Translational Genomics Research Institute (TGen).

The 45 interns in the 2013 Helios Scholars at TGen summer internship program completed eight weeks of biomedical investigations and presented their findings at a daylong scientific symposium July 26 at the Renaissance Phoenix Downtown Hotel.
Under the mentorship of TGen researchers – who provide one-on-one instruction – Helios Scholars use cutting-edge technology to help discover the genetic causes of diseases such as diabetes, Alzheimer’s disease and cancer.

This is the seventh 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 is one of the premier examples of how students can become immersed in the sciences and see a real connection between the work they’re doing in labs and future career opportunities in the field,” said Helios Education Foundation President and CEO Paul Luna. “At Helios Education Foundation, we believe in the transformational power of education. The Helios Scholars at TGen program is helping prepare students for academic success while potentially making scientific breakthroughs that could improve the lives of future generations.”

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

“Our partnership with the Helios Education Foundation is helping prepare a whole new generation of biomedical investigators for Arizona,” said Dr. Jeffrey Trent, TGen’s President and Research Director. “We help them explore the biosciences beyond the classroom, honing their skills through participating in potentially life-changing research projects.”

The internships help students from diverse backgrounds – selected from a competitive pool of nearly 500 applicants – sharpen their research skills as they prepare for careers in science and medicine.

In addition to technical skills, students participate in professional development seminars that broaden their knowledge and skills in science communication, networking, career development, business etiquette and public speaking. The ultimate goal of the seminars is to produce savvy, polished future scientists and physicians.

“Many of this year’s Helios Scholars were born after the start of the Human Genome Project,” said Brandy Wells, TGen’s Manager of Science Education and Outreach. “These students of the genome-age are torchbearers for future medical discoveries, based on a precise understanding of the genetic underpinnings of human disease.”

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

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TGen-TD2-Scottsdale Healthcare study benefits patients

The Side-Out Foundation’s breast cancer pilot study, led by the Translational Genomics Research Institute (TGen), Translational Drug Development (TD2) and Scottsdale Healthcare, has shown that cancer patients do better when their treatment is guided by molecular profiling.

Specifically, 52 percent of patients with advanced breast cancer received clinical benefit – meaning their disease was controlled for a longer time – when their cancer was treated based on addressing the abnormal proteins in their tumor, according to the study conducted at the Virginia G. Piper Cancer Center Clinical Trials, a partnership of Scottsdale Healthcare and TGen.

Each patient’s treatment was “personalized,” meaning that the therapy they received was based on their individual tumor biology.

“This study demonstrates the feasibility of personalized cancer treatment, and shows that this approach merits further investigation in future studies,” said Gayle Jameson, Nurse Practitioner at Scottsdale Healthcare’s Virginia G. Piper Cancer Center Clinical Trials and the study’s Principal Investigator.

“The success of this pilot study will lead to a larger study and hopefully greater clinical benefit for more patients with advanced breast cancer,” said Jameson, who presented the results of the study in June at the 2013 American Society of Clinical Oncology (ASCO) in Chicago.

Due to the overwhelmingly positive results, a new study incorporating additional technology for tumor analysis, Side-Out II, will open at the Virginia G. Piper Cancer Center Clinical Trials in the near future for patients with advanced breast cancer.

“The success of our pilot proof-of-concept study has established a firm launching pad for the upcoming Side-Out II study, which involves a more in-depth investigation of tumor biology with an expanded repertoire of tests to direct personalized treatment,” said Dr. Jasgit Sachdev, M.D., a breast cancer specialist and Associate Professor at the Virginia G. Piper Cancer Center Clinical Trials.

“By showing the significant advantages of molecular profiling, this pilot study has enabled us to move forward with a project that should strengthen the evidence for using this approach in routine clinical care.”

The recent pilot study built on previous studies by Scottsdale Healthcare and TGen that showed the value of guiding treatment based on molecular profiling, in which each patient’s tumor was analyzed for protein abnormalities that may “drive” the cancer’s growth. The results pointed investigators toward specific genetic changes that might be addressed by specific medications.

Beyond molecular profiling, the pilot study also included mapping proteomic pathways within the tumor tissue so each patient could receive a highly targeted regimen designed to impede their cancer growth.

All of the patients in the recent study had advanced breast cancer that had progressed following multiple previous chemotherapy treatments. Of the 25 patients, 13 received clinical benefit as a result of molecular profiling. For all 25 patients, the therapy selected based on their tumor analysis was different than what they would have received in their next planned treatment, if they had not participated in the study.

The Virginia G. Piper Cancer Center at Scottsdale Healthcare was the lead site in the 2-½ year pilot study. In addition, patients in the study were treated at Virginia Cancer Specialists, US Oncology, in Fairfax, Vir.; and at Evergreen Hematology & Oncology in Spokane, Wash.

Translational Drug Development (TD2), a TGen company, managed the pilot clinical trial, and will also oversee the follow-on study, Side-Out II.

“This was an exciting study for TD2,” said Linda Vocila, BSN, RN, Director of Clinical Operations at TD2 and co-author of the study. “It demonstrates that close collaboration between physicians and scientists leads to greater clinical benefit for patients with cancer.”

Two labs analyzed tissue: the Center for Applied Proteomics and Molecular Medicine (CAPMM) at George Mason University in Manassas, Vir.; and Caris Life Sciences in Phoenix.

The Side-Out Foundation of Fairfax, Vir., sponsored the study.

To participate in a clinical trial at the Virginia G. Piper Cancer Center, please contact Patient Care Coordinator Joyce Schaffer at 480-323-1339 or joschaffer@shc.org.

medical.research

TGen, Ventana announce research collaboration

The Translational Genomics Research Institute (TGen) and Ventana Medical Systems, Inc. (Ventana), a member of the Roche Group, today announced a collaborative research agreement to discover and develop diagnostic markers for treating cancer.

The two Arizona-based institutions will leverage each other’s expertise in discovery and diagnostic product development, bringing innovative cancer diagnostic tests to patients.

The first project under the umbrella research agreement will focus on diagnostic, prognostic and drug biomarkers for pancreatic cancer, the fourth leading cause of death from cancer in the United States. This year, an estimated 45,000 people will be diagnosed and more than 38,000 patients will die from the disease. Worldwide, more than 213,000 are diagnosed with pancreatic cancer each year, and the numbers are growing. Fewer than 1 in 4 pancreatic cancer patients survive more than a year, and fewer than 6 percent survive more than five years – the worst survival rate of any cancer.

This dismal picture of pancreatic cancer is mainly due to the lack of tools for early detection and the ineffectiveness of current therapeutics. This is why new diagnostic markers and more efficacious therapies are desperately needed.

“TGen is on the cutting edge of translational research, where investigators discover the genetic components of disease,” says Jeffrey Trent, Ph.D., President and Research Director of TGen. “Our goal is to rapidly translate basic research findings into actionable targets. Partnering with Ventana we hope will accelerate our goal to deliver meaningful discoveries to cancer patients today.”

“When a patient is faced with cancer, getting an accurate diagnosis quickly is the most important part of their treatment,” says Ventana President and CEO Mara G. Aspinall. “As the global leader in tissue-based cancer diagnostics, our strength is moving research into the clinic in order to improve the lives of all patients afflicted with cancer. We are thrilled to be able to pursue this with a partner right in our Arizona backyard.”

prevention trial - brain scan images

Ivy Foundation Grants Over $9M for Brain Cancer Research

The Ben & Catherine Ivy Foundation (Ivy Foundation) announced its 2012 grant recipients, which total more than $9 million in funding for brain cancer research. The Ivy Foundation is the largest privately funded brain cancer research foundation in North America. Catherine Ivy is the founder and president of the Ivy Foundation, which has a research funding focus on glioblastoma multiforme (GBM), the most common and deadliest of malignant primary brain tumors in adults.

The Ivy Foundation awarded the following grants and/or provided funding in 2012:

· $2,500,000 over three years:  Principal Investigator, Greg D. Foltz, M.D., Director, The Ben & Catherine Ivy Center for Advanced Brain Tumor Treatment, Swedish Medical Center
· $5,000,000 over five years:  Principal Investigators, John Carpten, Ph.D. and David Craig, Ph.D., Translational Genomics Research Institute (TGen) – a collaborative effort with University of California, San Francisco; University of California, Los Angeles; Memorial Sloan-Kettering Cancer Center; Massachusetts General Hospital; Dana Farber/Harvard Cancer Center; MD Anderson; and University of Utah
· $45,000 annually: Principal Investigator, Brandy Wells, Translational Genomics Research Institute (TGen), for the Ivy Neurological Sciences Internship program
· Over $2 million paid out in 2012 for previously committed multi-year brain cancer research grants

“We are encouraged and remain strongly committed to moving the progress forward for patients diagnosed with brain cancer,” said Ivy. “The 2012 Ivy Foundation grant recipients are important strategic partners in our objective to double the life expectancy of people diagnosed with GBM within the next seven years.”

head.injury

TGen and Riddell Announce Partnership

Head protection plays a vital role in the health and safety of any athlete participating in helmeted sports.  In a move that could help revolutionize football player safety, the Translational Genomics Research Institute (TGen), and Easton-Bell Sports through its Riddell brand, announced today it would work together on a study designed to advance athlete concussion detection and treatment.  Information gathered through the study will also be used to develop new football headgear and further refine updates to player monitoring technology.

“TGen welcomes this remarkable opportunity to join Riddell in a major research study with the goal of helping to objectively monitor a player on the field (with microelectronics combined with nucleic acid sequencing),” said Dr. Jeffrey Trent, TGen President and Research Director. “TGen’s work over the past several years in the area of head trauma is accelerating new insights to the critical study of concussion injury.”

The genesis of this potentially groundbreaking study is to merge a player’s genetic information with real-time microelectronic information captured by Riddell’s Sideline Response System (SRS). A highly sophisticated, data-intensive system, Riddell SRS provides researchers, athletic staff and players with a wide range of valuable information on the number and severity of head impacts a player receives during games and practices.  Employed since 2003 by several well-respected research institutions, Riddell SRS has captured 1.8 million impacts from youth to elite football competition, and its data has led to impactful changes to rules, how the game is played and coached, and has informed new helmet designs.

“As the industry leader in football head protection, Riddell has the unique opportunity to advance TGen’s groundbreaking medical research into the brain as we work together towards identifying a way to accurately and quickly diagnose concussions in football players,” said Dan Arment, President of Riddell. “With Riddell’s commitment to player protection and history of innovation, we are hopeful that our collaboration with TGen will help us better protect athletes and lead us to meaningful advancements in helmet technology that move the game of football forward.”

A key question the study seeks to answer is: are the effects of sub-concussive hits identifiable through blood-based molecular information? “Based on our current information, we believe this study will have the unique ability to provide a molecular ‘risk’ and ‘recovery’ score, enabling physicians to better identify when a player might be expected to recover from the effects of the concussion and get back on the field,” said Dr. Kendall Van Keuren-Jensen, TGen Assistant Professor, whose technique for studying molecular information at a micro level will drive the research.

While the joint study will begin with football, the Riddell-TGen partnership has the potential to improve sports equipment manufactured by brands in the broader Easton-Bell Sports portfolio, including headgear for hockey, baseball, cycling, snowsports, and powersports. “As the awareness of head injury grows across all sports, supporting science like this will help us offer a more protective helmet solution to the athlete,” said Arment.

Local Institutes and Advocate to Join Study

As part of the study, TGen will work with the Barrow Neurological Institute 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.

“Combining our neurological expertise and the information from our B.R.A.I.N.S. program, with TGen’s genomic knowledge and Riddell’s helmet technology, will provide great insight into how we measure concussions and how they affect the human brain,” said Dr. Javier Cárdenas, a neurologist and brain injury expert with Barrow Neurological Institute. “The genomic data could aid in the treatment process and will greatly add to the growing body of knowledge we’re acquiring about head injury patients.”

Joining Barrow will be athletic trainers from A.T. Still University and SAFE Football, which teaches alternative game-play techniques that reduce the number of head impacts while increasing competitiveness.

“Our partnerships with Barrow Neurological Institute, A.T. Still University, and Safe Football provide a multifaceted approach to identifying athletes in need of medical attention, to educating athletes on concussion and brain injury, to reducing the risk of injury through development of better techniques, and to improving treatment outcomes,” said Dr. Matt Huentelman, TGen Associate Professor and a co-investigator on the study.

Bioscience helix

Ivy Foundation Renews Support for TGen Program

The Arizona-based Ben & Catherine Ivy Foundation will fund a second year of the Ivy Neurological Science Internship Program at the Translational Genomics Research Institute (TGen).

The internship program offers hands-on biomedical research experience for high school, undergraduate and aspiring medical school students pursuing careers in brain tumor research, neuroscience and neurogenomics.

Through the program, world-class scientific investigators at TGen guide interns in the translational process of moving laboratory discoveries along the pipeline into new treatments for patients in clinical trials.

“Based upon the success of the 2012 pilot year, we believe the Ivy Neurological Science Internship Program at TGen will inspire a new generation of leaders in this field,” said Catherine Ivy, President of The Ben & Catherine Ivy Foundation. “There is an urgent and continuing need to encourage research into the intricate workings of brain cancer.”

TGen will select seven students for the program this year. Starting in June, two high-school students will participate in a 10-week summer program. Four undergraduate students will spend the fall semester at TGen, and one student planning to attend medical school will participate for a full academic year, beginning in the fall.

“Development of a local, knowledge-based workforce depends on educating and training talented students in the latest aspects of biomedical research and medicine,” said TGen President Dr. Jeffrey Trent. “The continued support from the Ivy program greatly enhances our efforts to provide hands-on experience in the area of translational research.”

In addition to brain tumor and neurological sciences research experience, Ivy interns will participate in a clinical training module that will engage them with the ultimate focus of these studies – the patient.

“TGen recognizes that we must invest in the development of the next generation of researchers and physicians; we need to prepare today’s students for the complex and challenging work awaiting them in the areas of brain tumor and neurological sciences research,” said Brandy Wells, Manager of TGen’s Education and Outreach.

For more information, please contact Brandy Wells at bwells@tgen.org or 602-343-8655.

prevention trial - brain scan images

TGen scientist launches innovative online research project

A scientific researcher at Phoenix-based Translational Genomics Research Institute (TGen) has launched a first-of-its-kind online memory test to help better understand human cognition and how it might relate to Alzheimer’s disease and other brain disorders.

Dubbed “MindCrowd”, the study seeks to attract 1 million individuals, aged 18 to 80, willing to complete the 10-minute online memory test at mindcrowd.org. Researchers will use the test results to build a base of data for further study on how cognition and memory changes as people age.

Eventually, the researchers want to leverage this newly-gained biological insight into therapeutic application — treatment. The hope is for the online test to go viral with friends, families and colleagues challenging one another to take the test and compare the results.

MindCrowd is the brainchild of TGen Associate Professor Dr. Matt Huentelman who believes understanding how the brain works in healthy individuals will foster the development of new medicines and therapies for those with brain disorders. Dr. Huentelman’s TGen lab studies the genomics of human neurological traits and diseases with a specific focus on learning, memory and Alzheimer’s.

“MindCrowd is the first research project of its kind,” said Huentelman, an expert in genomics as it relates to memory. “By harnessing the power of the Internet, we can study a million – or more – individuals to help bring us closer to a cure for Alzheimer’s disease and other brain disorders. Combining our knowledge of human genetics and neuroscience with an online research study like MindCrowd is a revolutionary approach to understanding our differences in brain performance and how it may influence risk for disease. We expect to add significantly to our understanding of cognition and how genetic factors impact our memory as we age.”

The MindCrowd project has two phases: Phase I involves memory testing of 1 million or more study participants. Following an in-depth analysis of Phase I test results, researchers will then solicit a subset of Phase I participants willing to donate a DNA saliva sample and undergo an additional round of online testing.

Participation is encouraged from a broad range of ages, backgrounds and cognitive abilities. Those taking the test are free to remain anonymous, although it is encouraged that people share basic data to help the project succeed. The test does not predict or diagnose any condition, rather it provides data on one type of memory and how these processes change as people age and have varied life experiences.

MindCrowd is a collaborative effort among leading scientific research institutions and organizations including TGen, the University of Arizona, Banner Alzheimer’s Prevention Initiative and others.

Visit www.mindcrowd.org to take the test.

medical.research

Foundation donates $500,000 for TGen research

The Seena Magowitz Foundation has donated $500,000 from two charity golf tournaments dedicated to supporting pancreatic cancer research at the Translational Genomics Research Institute (TGen).

Past donations from the Seena Magowitz Foundation have helped fund significant scientific research that is making a difference in the lives of pancreatic cancer patients and their families.

In January, TGen Physician-In-Chief Dr. Daniel Von Hoff presented study results at a San Francisco cancer symposium, showing that the drug Abraxane (nab-paclitaxel) when combined with gemcitabine, significantly extended the survival of pancreatic cancer patients. The Seena Magowitz Foundation helped fund the clinical trials that led to this advance.

“A decade of advocacy and fundraising is really paying off with concrete results that are actually helping pancreatic patients survive longer,” said Roger Magowitz, President and Co-Founder of the Seena Magowitz Foundation. “Our supporters deserve to take a bow, because the research behind these advances might not be possible without their help.”

Pancreatic cancer is the fourth-leading cause of cancer death in the U.S. and annually takes the lives of more than 38,000 Americans. A staggering 75 percent of those diagnosed die within the first year, and only 6 percent survive more than five years.

The most recent $500,000 donation to TGen from the Seena Magowitz Foundation represented funds raised during the 10th annual Seena Magowitz Golf Classic on Dec. 8 at the Arizona Biltmore Resort, and also from the 3rd annual Atlanta Golf Classic, organized Oct. 1 by pancreatic cancer survivor Howard Young, President of General Wholesale Beer Company. Young is a TGen Foundation Board Member and Chairman of TGen’s National Advisory Council for Pancreatic Cancer Research.

“TGen could not have made the progress we have against this disease without the unflagging dedication of special people like Roger Magowitz and Howard Young, and the hundreds of supporters they have inspired over the past decade,” said TGen Foundation President Michael Bassoff.

Magowitz informed the TGen Foundation of the $500,000 donation during the March 28 Evening on the Diamond, an annual fundraising event hosted by the Arizona Diamondbacks at Chase Field.

“We are experiencing a remarkable confluence of forces between the Seena Magowitz Foundation, Major League Baseball and TGen in the fight against this most aggressive and deadly of cancers,” Magowitz said. “It is this kind of concerted effort, backed by prominent individuals and organizations, that eventually will lead to a cure for pancreatic cancer.”

Evening on the Diamond included the presentation of the Arizona Diamondbacks Foundation Community Leadership Award, now named in memory of Lee Hanley, the late TGen Foundation Board Member who passed away in 2012 from pancreatic cancer. The newly named Lee T. Hanley Community Leadership Award was presented to former Arizona Sen. Jon Kyl for his 26 years of service in the U.S. Congress. Past winners include TGen Board Chairman Bill Post, TGen Foundation Board Member Karl Eller, and TGen President and Research Director Dr. Jeffrey Trent.

Singer-songwriter and multi-instrumentalist Alyssa Bonagura, who performed Dec. 8 during the 10th annual Seena Magowitz Golf Classic, also performed during Evening on the Diamond.

In March, Brian Cashman, Senior Vice President and General Manager of the New York Yankees, at the invitation of D-backs President and CEO Derrick Hall, joined TGen’s National Advisory Council for Pancreatic Cancer Research. Both MLB officials’ fathers died from the disease, and Hall was master of ceremonies at the Seena Magowitz Golf Classic.

Major sponsors of the golf tournament include: Leggett & Platt, Mattress Firm, Sealy, Mattress Discounters, Sleep Inc., Serta, Comfort Revolution, Ellman Family Vineyards, General Wholesale Beer Company, Morley Company, Simmons, Raymond James, Customatic, and the Arizona Diamondbacks.

Roger Magowitz founded the Seena Magowitz Foundation in honor of his mother, Seena, who died of pancreatic cancer in 2001.

clinical research advantage - vaccines

TGen-Scottsdale Healthcare battle tumor growth

The safety and preliminary efficacy of a new class of tumor fighting drugs were reported by Scottsdale Healthcare’s Virginia G. Piper Cancer Center Clinical Trials and the Translational Genomics Research Institute (TGen).

Early results from the phase I, first in-human study of an RNA interface (RNAi) drug were announced during the American Association for Cancer Research (AACR) Annual Meeting 2013, April 6-10, in Washington, D.C. The drug, TKM-080301 (also known as TKM-PLK1) is being developed by Tekmira Pharmaceuticals Corporation.

The study was conducted at Virginia G. Piper Cancer Center Clinical Trials at Scottsdale Healthcare, a partnership with TGen. It found that the RNAi drug acts by silencing the PLK1 gene involved in tumor growth and can be safely administered in humans.  Most patients tolerated the drug well; some showed therapeutic benefit.

“RNAi therapies are a unique approach to cancer treatment as they have the potential to ‘turn off’ the genes’ coding for proteins involved in cancer cell division,” said Dr. Ramesh K. Ramanathan, Medical Director of Virginia G. Piper Cancer Center Clinical Trials at Scottsdale Healthcare and deputy director of the Clinical Translational Research Division of TGen. “Using a lipid nanoparticle, the RNAi drug can be delivered to a cancer cell to block the expression of specific proteins involved in tumor growth.”

TKM-080301 targets a specific gene called polo-like kinase 1 (PLK1), which codes for a protein involved in tumor cell growth. Prior research has shown that high levels of PLK1 are present in many types of cancer, including many of the more aggressive forms.

“Our preclinical results have shown that by decreasing PLK1 levels in cancer cells, we can stop tumor growth and kill the cancer cells,” Dr. Ramanathan said.

He and his colleagues have been enrolling patients with advanced solid tumors or lymphoma into the ongoing multicenter, open-label, dose-escalation study. Sequential cohorts of three to six patients have been assigned to escalating doses of TKM-080301 as a 30-minute intravenous infusion. To date, the researchers have assigned 23 patients to the drug at doses ranging from 0.15 mg/kg per week to 0.9 mg/kg per week.

The most common drug-related adverse events have been mild to moderate and include fever, chills, nausea, vomiting and fatigue. Dose-limiting toxicities were observed at the 0.9 mg/kg per-week dose. One patient with a history of asthma experienced shortness of breath and hypoxia; another patient had thrombocytopenia. The researchers subsequently reduced the maximum dose to 0.75 mg/kg per week.

Two patients have been assigned to TKM-080301 for more than six months and have shown no evidence of cumulative toxicity. One of these patients has stable disease and the other has a durable confirmed partial response.

“RNAi therapies, such as the one used in our study, have the potential to make a significant and broad impact on how we treat cancer because we have the ability to target virtually any protein involved in the disease,” Ramanathan said. “This approach has the potential to augment the currently available cancer treatments to improve outcomes for the patient.”

medical.research

Medical miracle girl raises funds for TGen

Shelby Valint, the 12-year-old Phoenix girl whose sequenced genome led her from a wheelchair to walking, is raising funds for the non-profit Translational Genomics Research Institute (TGen).

The “Shelby Valint Inspiration Fundraiser” will generate needed research dollars for TGen’s Center for Rare Childhood Disorders (C4RCD). It was research through this innovative unit at TGen that helped enable Shelby to go from a wheelchair to walking.

“TGen has done so much for me,” Shelby said. “Now, I want to do something for TGen so they can continue to help other children like me with rare medical disorders.”

The fundraiser is being organized by Shelby’s mother, Renee Valint, and by one of Shelby’s 7th Grade teachers, Tracy Livingston, whose husband – the Honorable Rep. David Livingston – is a freshman member of the Arizona House of Representatives, representing the north Valley’s District 22.

“In October, TGen launched their Center for Rare Childhood Disorders, which is helping parents in Arizona find answers and treatment for their children,” said Rep. Livingston, who has invited Gov. Jan Brewer and members of the Arizona Legislature to the fundraiser at the home of Shelby’s parents, Renee and Scott Valint – 1-5 p.m. April 6 at 1517 E. Red Range Way, about a mile south of Carefree Highway, just east of 14th Street.

“In my recent tour of TGen’s facilities, I saw first-hand the cutting-edge research, tools and technology being used to help children like Shelby,” Rep. Livingston said. “My wife, Tracy … has personally seen Shelby’s amazing transformation.”

By sequencing, or spelling out, the nearly 3 billion letters in Shelby’s DNA, TGen researchers found a gene that prevented Shelby from producing sufficient amounts of a brain chemical called dopamine, which is needed for balance and muscle control.

Using a combination of drugs usually given to older persons for treatment of Parkinson’s disease, Shelby was able within several weeks to abandon her wheelchair. She was able to more easily walk, talk, eat and even breathe, generally restoring her to a normal functioning child.

“Before TGen’s discovery, we had been through an enormous amount of despair with all the doctor visits and tests, and I had watched helplessly as Shelby was poked and prodded with a heart-wrenching number of needles and IVs,” Renee Valint said. “Shelby’s newfound ability to walk and talk, and generally lead a normal life, is a testament to the unwavering dedication to helping patients exhibited by the scientists at TGen.”

To see Shelby’s amazing transformation from a girl who was unable to walk, talk and eat to a girl who dances across the room, watch this recent story from CBS 5 News.

 

medical.research

FDA approved thyroid cancer drug tested by TGen

The FDA has approved a thyroid cancer drug successfully tested at Virginia G. Piper Cancer Center Clinical Trials, a partnership of Scottsdale Healthcare and the Translational Genomics Research Institute (TGen).

The U.S. Food and Drug Administration approved cabozantinib for the treatment of progressive, metastatic medullary thyroid cancer (MTC), a rare endocrine gland cancer affecting the thyroid. Previously, MTC patients had limited treatment options.

“This was a really exciting trial. We have a drug that blocks the gene that causes a rare type of cancer,” said Dr. Michael Demeure, the Site Principal Investigator on the Phase III clinical trial and endocrine surgeon at Scottsdale Healthcare. “We’re trying to do more tests like this to try to bring innovative and exciting new cancer drugs for our patients.”

More than 56,000 Americans will be diagnosed with thyroid cancer this year, and 1,780 will die from the disease. About 4 percent of thyroid cancers are medullary, a form of carcinoma that originates from the parafollicular, or C, cells, which produce the hormone calcitonin. Physicians are able to confirm a diagnosis of MTC by detecting elevated levels of calcitonin in the blood.

MTC often is not detected until its late stages. And unlike 90 percent of differentiated thyroid cancers, MTC does not respond to the most common treatments, making it difficult to manage.

The RET gene, which is part of the family of tyrosine kinase proteins, plays a role in the development of MTC. Cabozantinib is an inhibitor of multiple receptor tyrosine kinases, including RET, MET and VEGFR2.

“Cabozantinib targets tyrosine kinases that are important in medullary thyroid cancer, including RET, MET and VEGFR2. When such tyrosine kinases — which are known to play a role in tumor growth and metastasis — are also the drug target, that gives you hope that you can impact favorably on the disease,” said Dr. Demeure, contrasting MTC with other cancers, such as pancreatic cancer, where the precise genetic source of the cancer remains unconfirmed or unknown.

The Virginia G. Piper Cancer Center at Scottsdale Healthcare enrolled two patients with MTC as part of an international randomized clinical trial of more than 300 patients.

FDA’s approval on Nov. 29 was based on demonstrating improved progression-free survival (PFS). The estimated median PFS was 11.2 months for patients taking cabozantinib, compared to 4 months for patients taking placebo. The drug is sold as COMETRIQ and marketed by South San Francisco-based Exelixis, Inc.

One patient who continues to benefit from clinical trial treatments at Scottsdale Healthcare’s Virginia G. Piper Cancer Center Clinical Trials is Gordon Hunt, 68, a retired life-insurance salesman from Phoenix.

Hunt said he started noticing discomfort in his neck several years ago. After seeing a series of specialists, a calcitonin test finally confirmed that he had an advanced case of MTC.

Hunt endured several surgeries that included the removal of his thyroid and lymph nodes in his neck and chest. Following his most recent surgeries more than two years ago, performed by Dr. Demeure, Hunt’s calcitonin levels dropped from a one-time high of 3,300 picograms per milliliter, when he was first diagnosed, to about 500 pg/ml.

After receiving cabozantinib since February 2011, Hunt’s calcitonin levels are down to about 250 pg/ml, indicating that the cancer might still be in his system, but he has had no detectable tumors.

“I feel like he saved my life,” Hunt said of Dr. Demeure, who suggested he take part in the cabozantinib clinical trial.

“I’m just thankful for it, because I’m sure I’d be probably ready for another surgery of some sort if I hadn’t been on the medication,” said Hunt, who also expressed gratitude to the entire staff of the Virginia G. Piper Cancer Center at Scottsdale Healthcare. “They’ve been responsive to my every need.”

Hunt said he at first suffered side effects, including vomiting, diarrhea, stomach pains, weight loss and constipation. But by lowering his dosage, the side effects eventually diminished, he said. Dose reduction was required in 79 percent of clinical trial patients, according to the FDA.

Hunt receives monthly doses of the drug along with tests for calcitonin, as well as quarterly scans for tumors.

Between doses, he and his wife Nancy, a retired schoolteacher, travel extensively, including trips in the past year to California, Texas, Missouri and Australia.

“We’re still active, so that’s a good thing,” said Hunt, noting that the couple, who have lived 47 years in Phoenix, still go regularly to the gym and are active in their church.

“I’m excited. I played a part in making it (FDA approval) happen,” Hunt said. “I thank God that I was chosen to take part in obtaining the approval of the medication. If it works for me, it’s going to work for other people, and that’s good.”

In addition to treating MTC, cabozantinib is being explored as a therapy for numerous tumor types, including prostate, ovarian, brain, melanoma, breast, and non-small cell lung cancers.

medical.research

BIO5-TGen collaboration targets Alzheimer’s disease

BIO5 Oro Valley today announced a collaboration with the Translational Genomics Research Institute (TGen) to develop new therapies for the treatment of Alzheimer’s and other neurodegenerative diseases.

BIO5 Oro Valley co-Director and University of Arizona College of Pharmacy medicinal chemist Dr. Christopher Hulme’s collaborative effort with TGen Assistant Professor Dr. Travis Dunckley will focus on the development of novel, small molecule inhibitors of dual-specificity tyrosine phosphorylation-regulated kinase 1A (DYRK1A). Upregulation of this kinase is implicated in promoting memory deficits associated with Down syndrome and neurodegenerative pathologies, particularly Alzheimer’s disease.

“DYRK1A is a well-validated, recently discovered target, ready for translational efforts to deliver an oral medication to patients suffering from this insidious disease,” said Dr. Hulme. “Indeed, coupled with the advanced small molecules in-hand that target DYRK1A, further efforts are underway that will broaden our therapeutic presence in the Alzheimer’s arena to other Arizona-based biological discoveries.”

Statistics from the National Institutes of Health indicate that 5.1 million older Americans – or 1-in-8 – suffer from Alzheimer’s, which makes it the sixth leading cause of death in the United States and the only cause of death among the top 10 in the United States that cannot be prevented, cured or even slowed. Estimated to effect 45 million people worldwide by 2020, dementia is currently a leading, major unmet medical need and a costly burden on public health. Seventy percent of these cases have been attributed to Alzheimer’s, a neurodegenerative pathology characterized by a progressive decline in cognitive functions.

“This collaborative partnership is a critical step in advancing discoveries of the role DYRK1A plays to developing therapeutics that could alter the course of Alzheimer’s disease,” said Dr. Dunckley.

Drs. Hulme and Dunckley will focus on providing a significant alternative to common approaches that focus on small molecules that inhibit the production of neurotoxic fragments of amyloid proteins and antibody immunization approaches targeting the build up of these fragments.

The joint effort will explore the decrease of DYRK1A activity in the brain with proprietary small-molecule inhibitors. This approach could lead to new therapeutic strategies to alleviate cognitive deficits associated with Alzheimer’s and Down syndrome.

Tony Pena, Brian Cashman

Yankees’ GM supports TGen Research

A top official of the New York Yankees whose father passed from pancreatic cancer has joined a prestigious national panel organized by the Translational Genomics Research Institute (TGen) to fight this aggressive disease.

Brian Cashman, Senior Vice President and General Manager of the vaunted Yankees Major League Baseball franchise, has joined TGen’s National Advisory Council for Pancreatic Cancer Research.

TGen’s National Advisory Council leads a critically needed funding effort and promotes a deeper public understanding of pancreatic cancer, the nation’s fourth-leading cause of cancer death, which in 2012 took the lives of nearly 44,000 in the U.S. and nearly 235,000 worldwide.

Cashman lost his father, John, in September after a 10-month battle with pancreatic cancer. He had wanted his Yankees to reach the World Series as one last gift to his father.

“My father loved the Yankees. There are a lot of people who face these kinds of challenges, and they look to the Yankees to provide positive inspiration. For my father, the Yankees were always something he could look forward to,” he said. “I welcome the responsibilities and challenges of my role in the fight against pancreatic cancer. I have a personal experience to draw from, and coupled with my unique standing within the fabric of baseball, I’d like to believe I can make the type of contribution my father would be proud of.”

Cashman was invited to join TGen’s National Advisory Council by another council member, Arizona Diamondbacks President and CEO Derrick Hall, who in 2011 lost his father, Larry, to pancreatic cancer, even as Derrick was fighting his own battle with prostate cancer.

The Yankees and Diamondbacks played one of the game’s iconic 7-game World Series in 2001.

In addition to Cashman and Hall, another MLB official, David Dombrowski – President, CEO and General Manager of the Detroit Tigers – also is a member of the National Advisory Council for Pancreatic Cancer Research.

Other members of TGen’s National Advisory Council are: Raymond Bojanowski, Co-founder and Co-chairman of the Seena Magowitz Foundation; Karl Glassman, Chief Operating Officer and Executive Vice President of Leggett & Platt Inc.; Diane Halle,
President of the Bruce T. Halle Family Foundation and the Herbert K. Cummings Charitable Trust; Steve Hilton, Chairman and Chief Executive Officer of Meritage Homes Corp.; David Lane, President of the Lane Affiliated Companies; Roger Magowitz, President and Founder of the Seena Magowitz Foundation; Vincent McBeth, President of the The McBeth Group International and a retired U.S. Navy Commander; Larry Rogers, President and CEO of the Sealy Corp.; Steve Stagner, President and CEO of the Mattress Firm; Louis A. “Chip” Weil III, retired Chairman, President and CEO of Central Newspapers Inc.; and Howard Young, President of the General Wholesale Company.

“Brian Cashman is a powerful addition to TGen’s National Advisory Council. His personal experience, championship reputation, and national visibility will be a huge boost to TGen’s fight against pancreatic cancer,” said Michael Bassoff, TGen Foundation President.

Cashman joined the Yankees as a 19-year-old intern and now commands one of the most demanding jobs in sports. During 25 seasons, he has earned five World Series rings. At age 30, he became the youngest GM to win a World Series. And during 1998-2000 he became the only GM in Baseball history to win World Series titles in each of his first three seasons.

pharmaceuticals

Arizona bioscience job growth outpaces nation

Arizona’s bioscience sector added jobs at nearly four times the national rate over the past decade and experienced double-digit job growth during the economic recovery, a new report shows.

Since Arizona’s Bioscience Roadmap was launched in 2002, Arizona’s bioscience jobs have increased by 45 percent to 99,018 in 2011. Nationally, the growth rate during this time was 12 percent. While hospitals dominate Arizona’s bioscience jobs, the state’s non-hospital subsectors grew 14 percent in 2011 alone.  During the economic recovery years of 2009-11, the state’s bioscience jobs increased 11 percent while there was no gain across the state’s private sector.

The new performance analysis of Arizona’s bioscience sector, commissioned by the Flinn Foundation, also found that the number of bioscience establishments in Arizona continues to grow faster than the national average and bioscience wages in the state are outpacing those in other private-sector industries.

The 10th-annual study, released Feb. 5 by the Battelle Technology Partnership Practice, did reveal funding challenges for the state.  In 2012, Arizona fell to its lowest venture capital investment level since 2009 and suffered a drop in National Institutes of Health funding while the top-10 funded states advanced.

“Arizona’s bioscience sector continues to significantly outperform the nation in terms of job and establishment growth and has made impressive gains in building a more concentrated industry base,” said Walter Plosila, senior advisor to the Battelle Technology Partnership Practice.  “However, more attention must be paid to academic research performance and venture capital investment to continue the trend in years to come.”

Plosila added that progress has been made over the past decade on all 19 actions recommended by Battelle in 2002, including substantial progress on nine.

The Roadmap was launched in 2002 as a long-range plan to make the state’s bioscience sector globally competitive. The Roadmap was commissioned by the Phoenix-based Flinn Foundation, which committed to 10 years of major funding of Arizona biosciences and formed a network of committees involving statewide experts to implement its recommendations.

There was also a major increase in bioscience establishments, rising 31 percent since 2002 to 892 firms, which is above the national growth rate of 23 percent.

Bioscience jobs in Arizona pay an average salary of $56,328, or 28 percent higher than the $44,098 for all private-sector industries. Since 2002, bioscience salaries have increased 44 percent.

“After 10 years, Arizona has carved a niche in the highly lucrative and competitive biosciences field,” said Martin Shultz, chair of Arizona’s Bioscience Roadmap Steering Committee. “We’re one of the nation’s top emerging bioscience states, and our growth in high-wage jobs continued during both good economic times and bad.”

In terms of research dollars, NIH funding in 2012 was $174 million, or 19 percent greater, than the 2002 figure. This is a decrease from $184 million in 2011. While NIH funding, the gold standard for biomedical research funding, did increase slightly faster than the national average of 18 percent over the past decade, Arizona is no longer meeting its goal of obtaining funding at a growth rate higher than the top-10 funded states. In addition, its share of the funding pool remains nearly the same as it was in 2002.

The latest data also shows:
•    The largest non-hospital bioscience subsector continues to be research, testing and medical laboratories. This group now boasts about 8,900 workers across 466 establishments, roughly a 60 percent increase in both employees and firms since 2002. The other subsectors are drugs, pharmaceuticals and diagnostics; hospitals; medical devices and equipment; and agricultural feedstock and chemicals.
•    Venture capital investment was $22 million in 2012, which is the lowest figure since 2009. This was a drop of 68 percent from 2011, compared with a national decline of 49 percent.
•    Bioscience-related academic research and development expenditures at Arizona’s universities reached a record $452 million in 2011, a 55 percent increase since 2002. Arizona’s growth had outpaced the nation until 2009, but now trails the overall U.S. growth rate of 74 percent.
•    Arizona universities spun out seven bioscience companies in 2012. University discoveries have now led to 67 new bioscience startups since 2002 as well as 180 bioscience patents.

There were a number of major developments in 2012 that showed the collaborative nature of Arizona biosciences, including the completion of major projects, the approval of future pursuits, and an emphasis on education.

The University of Arizona opened its new Health Sciences Education Building on the Phoenix Biomedical Campus that enabled the UA College of Medicine-Phoenix to increase enrollment and for Northern Arizona University to begin Phoenix-based physician assistant and physical therapy programs. In addition, final approval was granted by the Arizona Board of Regents for the UA Cancer Center-Phoenix to be built on the same campus in partnership with St. Joseph’s Hospital and Medical Center.

A number of incubators and accelerators opened or expanded with more in the planning stages. BioInspire, an incubator for medical-device startups, opened in Peoria; GateWay Community College in Phoenix opened the Center for Entrepreneurial Innovation; the Arizona Center for Innovation at the UA Science and Technology Park in Tucson opened upgraded facilities and launched new programming; Flagstaff received funding for a planned accelerator; and the statewide Arizona Furnace accelerator began awarding seed money and access to incubation space.

Among other major developments, the inaugural Arizona SciTech Festival attracted 200,000 participants from across the state during February and March 2012, making it one of the largest in the nation; Banner Alzheimer’s Institute launched a $100 million trial to prevent or delay Alzheimer’s disease; a new skin-cancer drug first tested by Translational Genomics Research Institute and Scottsdale Healthcare received expedited approval from the Food and Drug Administration; Arizona State University began leading the first national algae biofuel testbed; Mayo Clinic announced plans for a new cancer center on its north Phoenix campus; and Banner MD Anderson Cancer Center in Gilbert announced a $63 million expansion.

On Dec. 4, 2012, the Flinn Foundation and bioscience leaders from across Arizona came together at the Arizona Biltmore to celebrate the 10th anniversary of the launching of Arizona’s Bioscience Roadmap. The Foundation announced it has committed to continue funding Arizona biosciences and coordinating the Roadmap as it enters its next chapter.

“We recognize this is a long-term pursuit,” said Jack Jewett, president and CEO of the Flinn Foundation. “We will continue to strive to improve the lives of Arizonans today and tomorrow through new medical discoveries, access to clinical trials and the recruitment of top researchers, while also attracting high-wage jobs that will strengthen Arizona’s economy.”

The Flinn Foundation is a Phoenix-based, private, nonprofit philanthropic endowment. It was established by Dr. and Mrs. Robert S. Flinn in 1965 with the mission of improving the quality of life in Arizona to benefit future generations. The nonprofit philanthropy supports the advancement of Arizona’s bioscience sector, the Flinn Scholars program, arts and culture, and the Arizona Center for Civic Leadership.

medical.research

Barrett-Jackson Auction, raffle net $129,000 for TGen research

The sale of a 2008 Shelby GT Barrett-Jackson Edition (Lot #3009) and the raffle of a 40th Anniversary Corvette generated $129,000 for cancer research at the Translational Genomics Research Institute (TGen) during Scottsdale’s 42nd Annual Barrett-Jackson Collector Car Auction.

Proceeds went to the Barrett-Jackson Cancer Research Fund at TGen, in Memory of Russ and Brian Jackson. The fund, established in 2010, is a salute to auction Chairman and CEO Craig Jackson’s father, Russ, and brother, Brian, whose lives were cut short by colon cancer.

The 2008 Shelby GT Barrett-Jackson Edition (Lot #3009) was auctioned Jan. 18 for $100,000 and sold to Rick Hendrick of Hendrick Motorsports in Charlotte, N.C.  Ele Chesney, a good friend of Nellie Jackson, the mother of Craig Jackson, donated this red and black convertible — upgraded by TMS Autosports — to raise funds for TGen.

The 1993 40th Anniversary Edition Chevrolet Corvette coupe was won by Roy Moreno of Allan, Texas (near Corpus Christi). The raffle raised $29,000 for TGen’s research. The ruby red Corvette, donated by Phoenix businessman David Harbour, is one of the 40th Anniversary models of this legendary sports car, which was first built in 1953.

Although the raffle was held during the Jan. 13-20 auction at Westworld of Scottsdale, the TGen Foundation conducted the raffle. Neither Barrett-Jackson Auction Co. LLC nor any of its affiliates were responsible for conducting this promotion.

“We are extremely grateful to Craig Jackson and his team at Barrett-Jackson for everything they do to raise funds for TGen research, and for spreading the word about TGen’s pursuit of personalized medicine; treating each patient based on their unique genetic profile,” said Michael Bassoff, President of the TGen Foundation.

More than 140,000 Americans, men and women, were diagnosed last year with colon cancer, which in 2012 killed nearly 52,000 patients, the third-leading cause of cancer death in the U.S.

An additional 241,000 American men were diagnosed last year with prostate cancer, which in 2012 killed nearly 28,000 patients, the second-leading cause of cancer death among men in the U.S.

In addition to its annual Scottsdale event, Barrett-Jackson also will conduct collector car auctions this year in: Palm Beach, Fla. (April 4-6), Las Vegas, Nev. (Sept. 26-28), and — announced Jan. 20 — a new auction in Reno, Nev. (Aug. 8-10).

The 2012 Barrett-Jackson Scottsdale auction helped raise nearly $5.9 million for local and national charities.