Illustration of a degenerative disease in brain

$1M, two-year grant from Dr. Ralph and Marian Falk Medical Research Trust to advance CWRU research on therapies for Huntington’s disease

A $1 million, two-year grant from the Dr. Ralph and Marian Falk Medical Research Trust will allow researchers from the Case Western Reserve University School of Medicine to further their work on developing drugs to treat Huntington’s disease (HD) and other neurological disorders.

Xin Qi, an associate professor of physiology and biophysics at the School of Medicine, leads a research team that has been working for four years to develop drug treatments for HD, an inherited, chronic neurological disorder that causes brain cells to die. HD typically surfaces at age 40 and progresses until fatal 10 to 20 years after diagnosis. There is no known therapy to prevent or slow the disease.

With the new round of funding from the trust’s Transformational Award Program, Qi and her team—including Drew Adams, an assistant professor of genetics and genome sciences and co-principal investigator, and consultant William Harte, the chief translational research officer at the university—will improve on novel compounds they’ve recently identified by screening for similar but safer and more effective potential medicines.

“We will synthesize and evaluate a series of analogs of the chemical leads and assess their effectiveness in treating HD in animal models and patient cells,” she said. “That will allow us to identify and focus on further drug development toward novel therapeutics for HD and a wider range of neurological disorders.”

The Dr. Ralph and Marian Falk Medical Research Trust, based in Providence, Rhode Island, supports biomedical research to improve treatments and find cures for diseases in which none is known. The trust supports such research with two programs: the Catalyst Research Award Program that provides seed funding to develop promising concepts, and the two-year Transformational Research Award to help researchers advance these concepts toward commercial development.

With this new grant, researchers at the School of Medicine have now been awarded five grants totaling nearly $3 million from the Falk Medical Research Trust for their work since 2014. All these projects received initial support from the School of Medicine’s therapeutics Accelerator program, the Council to Advance Human Health. One of the projects, an antivirulent agent, has already been licensed by Q2Pharma Ltd. for potential use in treating antibiotic-resistant infections.

Qi’s new therapeutics target mitochondria, which are membrane-bound organelles (tiny cell-like structures within cells) that generate most of the chemical energy to power a cell’s biochemical reactions. Mitochondrial dysfunction is a prominent early feature in patients with neurodegenerative diseases such as HD, Alzheimer’s and Parkinson’s.

With the previous Catalyst Award from the Falk Trust, Qi and her team were able to identify initial compounds that reversed disease activity in mouse models. With the new grant, these compounds will be further developed to use in patient trials.

For more information, contact Bill Lubinger at