$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.
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
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
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.
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
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.