Largest-ever drug trial launched in Huntington’s disease
Huntington's Clinical Research: Steve Hersch, MD
Development of a new drug is an arduous and complicated process. It is even more challenging if the drug is a nutritional substance, such as creatine, that cannot be patented and therefore lacks significant backing from a pharmaceutical company. Add an orphan disease like Huntington’s to the endeavor and the hurdles are enormous.
However, dedication and a compelling scientific foundation have enabled the launch of the largest therapeutic trial to date for Huntington’s disease (HD), which is being led by MIND investigators Steven Hersch, MD, PhD, and Diana Rosas, MD. This study, a definitive test of whether high- dose creatine can slow the progression of HD, is in collaboration with the Huntington’s Study Group and funded by the National Institutes of Health, the National Center for Complementary and Alternative Medicine (NCCAM) and the U.S. Food and Drug Administration (FDA) Orphan Products division.
Creatine is a natural substance that can help supply energy to cells and also reduces oxidative stress to brain cells. In HD, brain cells run out of energy, hastening their degeneration. Earlier studies using creatine with HD mouse models as well as smaller pilot clinical trials led by Drs. Hersch and Rosas demonstrated impressive results.
“Large amounts of creatine have to be ingested in order for therapeutic levels to reach the brain, so safety was a big concern,” says Dr. Hersch. “However, our Phase II trial with only 10 patients showed that high levels were safe and reached the brain. We were also amazed to see that a blood biomarker that detects brain degeneration was normalized, and brain imaging showed a slowing of brain shrinkage.”
Now, 650 patients from 44 sites around the world will test whether creatine monohydrate can slow the progression of HD in comparison to a placebo. The trial will take five years and millions of dollars to determine whether the biological effects that were observed in the small trial will translate to significantly slowing down disease progression and maintaining the quality of life for patients with HD.