Currently, there is no treatment to halt the progression of Huntington’s disease (HD), a fatal genetic disorder that slowly robs sufferers of their physical and mental abilities. In a new collaboration between SBP’s Conrad Prebys Center for Chemical Genomics (Prebys Center) and the University of California, San Diego School of Medicine, researchers have discovered that an existing compound, previously tested in humans for diabetes, offers hope for slowing HD and its symptoms.
“Our work at the Prebys Center is really a story that begins with Albert La Spada’s truly ground-breaking research on HD. Our expertise in chemical design, synthesis and dosing, have helped put his discoveries—and this compound—on a clinical trajectory. We hope our work leads toward significant benefit to a population in need of better treatment,” said Gavin Magnuson, project manager at the Prebys Center.
“We’re very excited by our pre-clinical testing of this compound (KD3010),” said Albert La Spada, MD, PhD, professor of pediatrics, cellular and molecular medicine and neurosciences in the UC San Diego School of Medicine. “It improved motor function, reduced neurodegeneration and increased survival in a mouse model of HD and reduced toxicity in neurons generated from human HD stem cells.”
La Spada said these findings are particularly notable because the drug was well-tolerated by participants in a Phase Ib clinical trial for diabetes, conducted in 2006, by a now-defunct biotech company.
“We have a drug that was FDA approved for human use in a clinical trial. It did not produce any significant side effects. This means it has a good safety profile and so likely can be moved into testing in HD patients much more quickly than compounds that have not been tested in humans. This is important since right now there is zero that can be done to alter the progression of this devastating disease.”
“This collaboration is an example of the Prebys Center’s goal to accelerate the pace of new drug discovery and development” said Anthony Pinkerton, PhD, director of chemistry at the Prebys Center. “We look forward to remaining actively engaged with Dr. La Spada to advance KD3010 toward phase II clinical development.”
The study was published in the December 7, 2015 online issue of Nature Medicine.
The PPAR delta paper also got to be the cover of the Nature Medicine Issue.