Tag Archives: U.S. Food and Drug Administration


DxNA LLC opens Valley Fever multicenter clinical trial

DxNA LLC has started a multi-center clinical study of DxNA’s molecular diagnostic test for the detection of Valley Fever (coccidioidomycosis), using its proprietary diagnostic platform, The GeneSTAT® System.

DxNA intends in 2015 to submit its 510(k) application to the U.S. Food and Drug Administration for clearance of the test and the GeneSTAT System.

Valley Fever is a fungal infection caused by Coccidioides organisms that typically enter the body through the lungs.  While the most people who are infected do not develop significant symptoms, a portion of infected patients develop symptoms that can be highly debilitating, such as cough, fever and fatigue. Creating an early, definitive diagnostic is critical to patient health, since these symptoms are similar to other respiratory diseases caused by bacteria or virus. As a result, Valley Fever is often misdiagnosed and mistreated.

The Valley Fever assay on the GeneSTAT System has been developed based on intellectual property exclusively licensed from the Phoenix-based non-profit Translational Genetics Research Institute (TGen), and will provide for the rapid detection of the fungus in patients.

“Valley Fever is Arizona’s disease and therefore has always been a critical target for TGen,” said Dr. Paul Keim, Director of TGen’s Pathogen Genomics Division in Flagstaff, Ariz. — TGen North. “This is an exciting step towards the realization of our Valley Fever diagnostic test being able to help clinicians better diagnose and respond to patients.”

Currently definitive testing is done by culture to grow the fungus in a laboratory, which is time consuming, up to 21 days, and potentially exposes laboratory personnel to the highly infective fungus.  The highly sensitive DxNA test is performed directly on the patient specimen, reducing the time to a definitive diagnosis and appropriate care.

The study will be carried out at three sites to encompass samples from the endemic regions for Valley Fever: central and southern Arizona, and the San Joaquin Valley in California.  In addition to the these sites, The Valley Fever Center for Excellence, under the direction of Dr. John Galgiani, has been instrumental in working with DxNA in the process of developing the assay and in setting up the clinical trial.


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.

The FYI on PAP Testing

The FYI On PAP Testing To Fight Against HPV, Cervical Cancer

According to the American Cancer Society, there will be more than 12,000 new cases of cervical cancer diagnosed this year in the United States alone, making it the third most common cancer in women. The disease forms slowly, but has very few symptoms in early stages.

The No. 1 risk factor in developing cervical cancer — the human papilloma virus, or HPV.

This virus, which can also cause genital warts, comes in more than 100 different strains of varying degree. About 40 of these strains can affect the genital area, potentially leading to cervical cancer.

The bad news?

Genital HPV infection is very common among sexually active people. It is so common, in fact, that some estimate that half of all men and three quarters of all women have been infected with HPV at some point in their lifetime.

The good news?

Most HPV infections will go away on their own without treatment within one to two years. However, some will continue to “hide” in the body for many years before they cause problems. This makes it nearly impossible to determine when patients became infected with the disease, how long they’ve had it and who gave it to them.

Thankfully, women have two powerful tools in the fight against HPV and cervical cancer — PAP tests and vaccination.

PAP Tests

All women should have PAP testing done to detect early precancerous changes on the cervix.

A PAP test, which is not painful to the patient, is done by a clinician inserting a metal or plastic speculum into the vagina, which is opened slowly to allow the clinician to visualize the cervix — the opening to the uterus. The clinician then uses a small brush and/or spatula to gently collect cells from the cervix. The cells are then sent to the laboratory to be tested for abnormalities. The lab test does not test for HPV itself, but rather looks for cellular changes that may be caused by HPV.

In the United States alone, the death rate from cervical cancer declined by 70 percent between 1955 and 1992 because of the initiation of the PAP test. The current recommendation for PAP testing is that it begins at the age of 21, regardless of when women first started having sexual intercourse. PAP testing prior to the age of 21 often will lead to continued unnecessary testing or procedures.

HPV Vaccine

Many women ask what they can do to protect themselves against HPV infection and cervical cancer. If one is between the ages of nine and 26, the U.S. Food and Drug Administration has approved a vaccination against certain strains of HPV that are more likely to cause genital warts and cervical cancer. This vaccination is given in a series of three injections over a six-month period.

Other ways to reduce one’s risk include:

  • Practicing abstinence
  • Having only one sexual partner who has no other intimate partners
  • Having sexual contact that does not involve anal or vaginal intercourse
  • Using condoms every time

Be sure to contact your healthcare provider, public health department or local Planned Parenthood health center for more information about cervical cancer and the importance of a proactive screening strategy and PAP testing.