Psychology Associate Professor Jeff Carroll’s work to better understand Huntington’s disease got a boost with a recent $100,000 grant from Ionis Pharmaceuticals, which is developing a drug that has shown promising results in early trials.
Carroll’s lab creates tools and techniques to investigate the normal role of the HD gene. Mutations in the gene cause Huntington’s disease, a fatal degenerative brain disorder.
Ionis and partner Roche Pharmaceuticals recently completed a safety study of the experimental drug, IONIS-HTTRx, which reduces the levels of the protein connected to the gene mutation. Reducing that protein, called huntingtin, can relieve symptoms of Huntington’s disease.
The completion of the safety study is “the most exciting clinical advance in HD so far,” Carroll says. “The demonstration that increasing doses of the drug lead to decreasing levels of the mutant huntingtin protein in the spinal fluid is a remarkable vindication of Ionis' many years of hard work and dedication to this project.”
Carroll, who carries the HD mutation himself, is among many in the HD community watching the progression of the drug trials very closely. He and Ed Wild of University College London run HD Buzz, a patient advocacy project that shares scientific news on HD research with HD families.
“The HD community is justifiably excited about and ready to participate in the pivotal study which will be required to demonstrate if the drug has an impact on HD symptoms, thanks to the observed reductions in the huntingtin protein,” Carroll says.