The California Institute for Regenerative Medicine (CIRM) has awarded $6 million to Neurobiology and Behavior & Psychiatry and Human Behavior Chancellor’s Professor Leslie Thompson. The award will help Professor Thompson and her team advance their new Huntington’s disease (HD) stem cell therapy through the late stage testing needed to apply to the Food and Drug Administration (FDA). Once approved by the FDA, the therapy can begin early clinical trials in patients.
Professor Thompson’s lab is at the forefront of using stem cells to better understand and treat HD, a fatal genetic disorder that breaks down nerve cells and erodes the ability to control body movement and speech. The team’s stem cell therapy involves transplanting neural stem cells into the body. Once transplanted, the neural stem cells can secrete a molecule called brain-derived neurotrophic factor, which has been shown to improve the function of the existing neurons. While not a cure, the aim of the treatment is to slow the progression of the disease by strengthening the body’s existing neurons.
“Huntington’s disease affects around 30,000 people in the U.S., and children born to parents with HD have a 50/50 chance of getting the disease themselves,” says Dr. Maria T. Millan, the President and CEO of CIRM. “We have supported Dr. Thompson’s work for a number of years, reflecting our commitment to helping the best science advance.”