Pitt and UPMC Partner to De-Risk Disease Therapies

Pitt and UPMC, through its Translational Sciences division, are once again collaborating to provide funding support for therapeutics projects that need further de-risking experiments to determine their clinical and commercial potential. We are featuring two projects that received funding from the first request for proposals from 2022.

Ed Burton and Donna Huryn have teamed to explore a drug discovery project seeking to advance a therapeutic molecule to treat progressive supranuclear palsy (PSP).

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As a clinician, Ed Burton operates a pre-eminent clinic for sufferers of progressive supranuclear palsy (PSP), a debilitating and fatal neurodegenerative disease with no effective treatments. And as principal investigator for the Pittsburgh Institute for Neurodegenerative Diseases at the University of Pittsburgh, he is working tirelessly to change that.

Dr. Burton, Professor in the Department of Neurology, has formed a cross-disciplinary collaboration with Donna Huryn, Professor in the Department of Pharmaceutical Sciences at the School of Pharmacy, to create a class of molecules that combat the damage to neurons caused by the disease.

The drug discovery collaboration is fueled by sponsored research from the Translational Sciences team at UPMC Enterprises, the innovation, commercialization, and venture capital arm of UPMC. Last fall, Enterprises' Translation Sciences group partnered with the Pitt Office of Industry and Economic Partnerships in the Office of Innovation and Entrepreneurship, to conduct a request for proposals for research with commercial potential that needed funding to conduct proof of concept experiments.

“When I started my PSP clinic in 2016, I didn’t advertise it, but relied on word of mouth. I was shocked when the clinic was at full capacity by 2017, underscoring the need for PSP care in the US,” said Burton, Professor of Microbiology and Molecular Genetics and UPMC Endowed Chair in Movement Disorders, with a note of frustration in his voice. “Sadly, by the end of 2021, all of the people in the initial cohort had died. Our whole focus on this project is clinical translation. We eventually want to have something that we can test in patients to slow the progression of symptoms and help these people.”

Key to Burton’s research are proprietary experimental models that mimic hallmark features of PSP, including neuroinflammation and loss of synaptic markers. Simultaneously, his lab developed a method of analyzing potential therapeutic benefits of novel interventions, so that when the lab initiated chemical screens of potential therapeutic molecules, they could directly determine if those molecules conferred functional benefits in the context of PSP.

“Over time, we found several mechanisms that prolong survival and protect mobility,” Burton said.

With targets in mind, Burton teamed up with Huryn to solve the problem of finding the right molecules that could work in humans.

“We have a good starting point, and now the goal is to develop therapies that are appropriate for people,” Huryn said. “We are working on compounds that get into the brain, are not metabolized by the liver and do not have toxic off-target effects.”

Burton said the funding from UPMC Enterprises is critical for conducting experiments to test the therapeutic candidates that Huryn’s lab generates.

“We are thrilled to collaborate so closely with a group of world-class investigators and the Innovation Institute at Pitt to advance our understanding of PSP,” said Matthias Kleinz, Executive Vice President of Translational Sciences at UPMC Enterprises. “Very few physicians and scientists in the world understand PSP as comprehensively as Dr. Burton, who for years has been studying both the underlying pathophysiology and the clinical impact PSP has on patients battling this disease. At the same time, Dr. Huryn’s invaluable expertise in medicinal chemistry and drug discovery will enable the development and validation of optimal therapeutic candidates. We’re very eager to support Ed and Donna in their pursuit of a transformative therapy benefitting PSP patients and their families.”

Pitt and UPMC Enterprises Translational Sciences are supporting a new funding opportunity for transformative therapeutics. This request for proposals focuses on the following areas. Awards will be announced later in 2023: 

• Precision Cardiovascular Disease
• Precision Metabolic Disease
• Chronic Kidney Disease
• Next-Generation RNA Therapies