A research team has reason to rejoice after the Food and Drug Administration granted it Friday approval to begin a clinical trial on a new pediatric brain cancer drug that could have been forgotten by drug companies.
The team's lead researcher, Dr. Teresa Purzner, has already beaten impossible odds. The neurosurgeon and his mother of three were able to get approval in record time and with little money thanks to the help of a team of scientific altruists called SPARK.
The development of new drugs in the United States is driven by pharmaceutical companies; University researchers rarely bring their findings to the bedside. According to the National Institutes of Health, out of a total of 10,000 new potential drugs stored in the country's laboratories, only one will reach patients in need. Why? Because the process can last 10 to 15 years and cost more than $ 1 billion per drug.
The exterior of the headquarters of the Food and Drug Administration is visible in White Oak, Maryland on November 9, 2015.
As a result, the number of new drugs approved by the FDA has remained stagnant at around 31 per year over the past 10 years. The majority of these drugs are similar to those already existing and many targeted diseases for which there are large markets – such as hypertension and hypercholesterolemia – and therefore a return on investment.
Enter SPARK, a non-profit program created in partnership between Stanford University and volunteers from the biotechnology, pharmacy and investment sectors, helps university researchers share their discoveries to patients. Since its inception, SPARK has paid particular attention to projects generally neglected by pharmaceutical companies, including rare diseases and diseases affecting children.
Purzner has paused his neurosurgical practice to study medulloblastoma, a type of brain cancer in children. Compared to diseases such as hypertension and hypercholesterolemia, which affect millions of Americans, medulloblastoma is rare and affects only 250 to 500 children each year.
A variety of pills and capsules are described in this undated stock image.
"It's particularly moving to see children – beautiful, wonderful and innocent things – and see the impact of the therapies we give them. Drugs and radiation therapies have an impact on their cognition, quality of life and ability to function as independent adults in the future, "Purzner said in an interview with ABC News.
Purzner had a clear goal: to find a targeted treatment that could block the basic biochemical pathway responsible for the development of this cancer, and she did it. She has tested the potential drug in mice with good results and she has just received FDA approval to test it in clinical trials, which she will do through the Pediatric Consortium for Tumors brain. She did it all in five years and for a price of $ 500,000.
"To go from my initial discoveries in the laboratory to the point that the Pediatric Consortium for Brain Tumors has selected for clinical trials, this would have never been possible without the help of SPARK (…) they gave me a clear path and made me believe that it was possible, "Purzner says.
Each year, SPARK provides 10 teams with expert funding and mentorship to promote the development of effective and cost-effective medicines.
"It is a case of scientific altruism, of scientific advisors who give of their time (…), these people are remarkably generous," said Dr. Kevin Grimes, professor of chemical and systemic biology at Stanford University, practicing physician and co-director of SPARK.
For the founder of SPARK, Daria Mochly-Rosen, professor of chemical biology and systems at Stanford, the mission of the organization is personal.
"SPARK has been the best part of my life," Mochly-Rosen told ABC News.
A variety of pills and capsules are seen in this undated photo.
And like the story of Purzner, there are many. Sixty percent of SPARK projects have already started clinical trials or have been licensed to companies.
"SPARK is tackling a black hole that neither NIH nor biopharmaceuticals can fill," Dr. Roham Zamanian, Associate Professor of Medicine and Director of the Canadian Pulmonary Arterial Hypertension Program, told ABC News. Stanford University. It allows researchers to advance drugs that would otherwise have no way, addressing the high cost of developing drugs and bringing precision drugs to patients, Zamanian said.
Zamanian's treatment for rare lung disease, called pulmonary hypertension, will be the subject of a global clinical trial this year.
The SPARK model has been replicated in 12 academic institutions around the world.
"It may be a way of having an impact on the health Zamanian said: "While bringing a precision and personalized medicine that maximizes benefits and decreases toxicity for patients."
Dr. Edith Bracho-Sanchez is a board-certified pediatrician, a Stanford Global Health and Journalism Scholar and a member of the ABC News Medical Unit.