San Diego researchers have discovered that an existing drug that may help slow Huntington’s Disease and its symptoms, giving hope for those suffering from a disease with no cure available at the moment.
The compound KD3010, previously tested for Diabetes, could hold the key for slowing the disease, researchers at UC San Diego School of Medicine have found, and they hope to launch a clinical trial within 18 months.
There is no treatment or cure for Huntington’s disease right now, a neurodegenerative, genetic disease that progressively diminishes physical and mental abilities.
"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 at UC San Diego School of Medicine, in a statement. "It improved motor function, reduced neurodegeneration and increased survival in a mouse model of Huntington's disease and reduced toxicity in neurons generated from human HD stem cells."
The drug started as a diabetes drug from the now-defunct San Diego biotech company Kalypsys. Because the drug has previously gone through safety studies for its diabetes clinical trial, it could be in human clinical trials in as soon as 18 months or less.
In a mouse test, where mice had a type of Huntington’s that produces symptoms like those in people, the animals saw longer lives, better balance, coordination, and didn’t lose as much weight.
"We saw significant improvements in terms of neurodegeneration," La Spade said. "It also extended their lives by 16 percent."
The drug works to increase the amount of protein produced by the body for the mitochondria, which powers cells.
The drug also helped mice get rid of misfolded proteins, which have been linked to Alzheimer’s, Parkinson’s and other neurodegenerative disorders. La Spada said he hoped the drug could be tested in connection with other related neurological diseases.
The study was published in the December 7 online issue of Nature Medicine.