The gold standard for treating Parkinson’s disease is far from perfect. Within five years of initiating treatment with the popular drug Sinemet or other L-dopa drugs, as many as 80 percent of patients develop the painful movement disorder known as dyskinesia — visible as tremors and involuntary limb jerking. As a result, many Parkinson’s patients delay treatment and suffer through early-stage motor difficulties.
Now, a promising alternative without the side effect of dyskinesia might make it to market, thanks in large part to support from the innovative Coulter-Drexel Translational Research Partnership Program at Drexel University.
In its ninth year, the program helps researchers at select universities commercialize discoveries to improve human health. So far, more than 40 Drexel projects — devices, diagnostics and drugs — have received money totaling about $5.5 million. Of those, a dozen have been licensed, meaning management teams are raising venture capital for additional pivotal FDA studies.
Think of it as a kinder, gentler version of the reality TV show “Shark Tank.” Researchers submit applications to an oversight committee at their university. Those who advance must make a 20-minute oral presentation and prepare for pointed questions about market size and competition. Winners receive grants and guidance to help commercialize their ideas.
“I think of the Coulter Program as the angel investor,” says its director, Davood Tashayyod.
Sandhya Kortagere, an assistant professor of microbiology and immunology in Drexel’s College of Medicine, applied to the Coulter Program after tests of her novel Parkinson’s drug in rodent models showed good results. (Her initial work was supported with a $100,000 grant from Drexel Ventures, a University-run tech transfer organization that funds innovative faculty ideas.)
Called PCT-3010, the drug modulates both dopamine — too little causes Parkinson’s — and norepinephrine in the brain to reduce motor disability and other symptoms, at least in rodents. It would give physicians an option for treating early symptoms of the disease, which affects 6 million older people worldwide, 1.5 million of them in the United States alone.
Kortagere’s experiments also showed that PCT-3010 has the potential to improve mild cognitive impairment associated with Parkinson’s disease. L-dopa and most other therapies can worsen cognitive effects.
“My drug, when fully developed, will help fill the existing treatment gaps,” says Kortagere, who has focused on Parkinson’s, in part, because an aunt suffered from the disease.
PCT-3010 solves the shortcomings of current therapies by essentially having more selective signaling properties. It activates the dopamine D3 receptors without causing desensitization and inhibits the norepinephrine transporters in the brain.
“This dual-acting drug is novel with a proven mechanism of action that is distinct from all known PD therapeutics in the market or in pipeline,” she wrote in her statement for the oversight committee.
The $100,000 award from the Coulter Program allows Kortagere to run a “killer experiment” that will either prove the concept or invalidate (kill) it through additional testing on animals. Before that, tests will take place for toxicity profiling, as well.
Funders for these essential but costly studies are hard to come by.
“Having somebody like Coulter to fund me is really important to help us cross the barrier of preclinical studies,” Kortagere said.
Established by the Wallace H. Coulter Foundation, the program carries on the legacy of health care innovation that biomedical engineer and inventor Wallace Coulter prized.
Only 16 universities around the country (Duke, Stanford and Michigan are among them) have Coulter programs and Drexel is just one of seven that the foundation has endowed. The foundation granted initial annual awards to the universities and then established endowments of $10 million, equally matched by the institutions.
Nationally, the Coulter partnerships have funded 280 projects to the tune of $70 million, according to Mara Neal, director of research awards for the Miami-based foundation.
“We don’t believe in basic research, research in search of knowledge,” she says. “Translational research is research in search of a product ... We promote the concept of serial innovators — those professors who stay at the University and do what they do best … teach, research and innovate.”
Drexel’s approach, says President John A. Fry, “is to maximize the real-world impact of our activities, with a strong focus on co-operative education, translational research and civic engagement. Drexel is committed to this program and I encourage all faculty to apply.”
For Kortagere, the program’s guidance on business aspects, including a business adviser with experience in the pharmaceutical industry, is as important as the grant.
“As an academic researcher, we often lack the ability to package and promote our products” she says. “I’m very appreciative that Coulter brought in people and educated us on several aspects of the therapeutics development and commercialization. I wish other [grant-awarding] agencies funding translational research adopted this model.
“Ultimately,” Kortagere says, “I felt like Coulter team wanted me to succeed. That was the message I got from the whole experience.”