New UPMC test promises lower cost, more effective treatment for brain cancer
February 8, 2016 12:00 AM
Marina Nikiforova demonstrates testing a brain cancer sample in the Next Generation Sequencing Lab at UPMC. The new test she helped develop predicts how aggressive a tumor will be and which chemotherapeutic agents will work best. UPMC is considering commercializing Dr. Nikiforova’s test.
Dr. Nikiforova holds an Ion torrent PGM 3/8th chip in the lab.
By Kris B. Mamula / Pittsburgh Post-Gazette
UPMC is exploring the possibility of creating a for-profit business to capitalize on the health system’s expertise in cancer diagnostics, part of a strategy to diversify revenue in an area of growing interest among research institutions nationwide.
The Pittsburgh health system recently developed and trademarked a test that can guide doctors in the treatment of brain cancers based on a tumor’s genetic makeup. Marina Nikiforova, director of UPMC’s three-year-old Molecular & Genomic Pathology Laboratory in Oakland, led a team that developed the test given the trademarked name “GlioSeq.”
“The test can be done on a very small tissue sample,” she said. “It provides such a wealth of information. This is a personalized approach.”
Brain cancers can be among the deadliest forms of the disease and physicians have historically used a one-size-fits-all treatment regimen of surgery, chemotherapy and radiation. With the new test, doctors can predict the aggressiveness of the tumor and what kind of chemotherapy will be most effective in treatment.
GlioSeq also promises to identify a variety of genetic mutations that cause brain cancer, eliminating the time and expense of individual tests that had been necessary in the past.
As part of a National Institutes of Health-funded study published in December, GlioSeq was tested on 54 adult and pediatric tumor samples for genetic abnormalities that had already been identified through other means. GlioSeq correctly identified all previously known gene aberrations, as well as many additional gene markers in the tumors.
An estimated 77,670 new cases of brain and central nervous system cancers are expected to be diagnosed in 2016, according to the Central Brain Tumor Registry of the United States, a Hinsdale, Ill.-based nonprofit, and most of the lesions will be non-malignant. But tumor cells that look the same under the microscope can respond very differently to treatment, depending on individual genetic malformations.
In addition, more than 25 percent of the so called “novel new” drugs approved last year by the Food and Drug Administration were either targeted to the individual patient’s molecular information or were new therapeutic biologics. Of the 45 new novel drugs approved, 13, or more than 25 percent, were personalized medicines, according to the FDA.
UPMC is working with the University of Pittsburgh’s Innovation Institute, the university’s commercialization arm, to develop a business model for the new test.
One option is simply to license the technology to other laboratories; another is to create a for-profit lab to do the testing, which is now being offered to research institutions around the country, Dr. Nikiforova said.
The size of the market and other issues related to commercialization are still being worked out, but universities nationwide are increasingly looking to licensing royalties as a way to make up for stagnant federal research funding.
Stanford University is an example of what’s possible: the university received more than $95 million in gross royalty revenue during the 2014-2015 year from 695 technologies, according to the Palo Alto, Calif.-based university. Eight inventions generated $1 million or more, for a total $1.5 billion over the past 45 years.
Faculty members at universities nationwide have debated the ethics of commercializing faculty research, but Stanford has aggressively pursued the strategy with what it calls ”socially responsible licensing,” which includes waiving royalties for sales in developing countries for some medical innovations.
With husband Yuri Nikiforov, who is director of the division of molecular and genomic pathology at the University of Pittsburgh, Dr. Nikiforova also helped develop a test of thyroid cancer tissue, called ThyroSeq, which can distinguish between more and less aggressive tumors.
That’s valuable information considering 20 percent to 30 percent of thyroid tissue samples yield uncertain results about the presence of cancer. UPMC has been offering ThyroSeq to other research institutions for a couple years.
Historically, treating cancer was much like firing a shotgun in the dark in a one-size-fits-all approach; some patients benefited, others did not. All that’s changing with a new focus on developing testing and treatments tailored to the individual, based on genetic mutations.
Danville, Pa.-based Geisinger Health System, for example, has been working with biopharmaceutical company Regeneron since 2014 to recruit 250,000 volunteers to donate a blood sample, which will be used to identify genetic connections to diseases.
Such testing will become a routine part of patient care, according to David Ledbetter, Geisinger’s chief scientific officer. Regeneron will use the results to identify disease links, which might be helped by new pharmaceuticals. Geisinger’s aim is simply to improve patient care.
Disease prevention or early intervention will ultimately reduce the health care costs, experts say. Similar efforts are underway at other health care institutions, where a patient’s genetic fingerprints are playing a bigger role in treatment decisions and the advice that doctors give about prevention.
“We knew that when you sequence healthy adults, you will find mutations in 2 percent of the people that puts them at increased risk for cancer or heart disease,” Dr. Ledbetter said. “We identify who’s going to be at higher risk, then determine what we can do to try to avoid disease.”
“This will become a routine part of clinical care.”
Kris B. Mamula:firstname.lastname@example.org, 412-263-1699
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