With a new $300,000 grant from the Muscular Dystrophy Association, a researcher with the University of Oklahoma Health Sciences Center hopes to advance the understanding and treatment of a neuromuscular disease called Friedreich ataxia.
With ataxia, parts of the nervous system that control movement and balance are affected, triggering problems with movement and coordination. Friedreich ataxia is the most prevalent inherited ataxia. It typically starts in the early adolescent years with unsteadiness in limb movement and walking. It then progresses to a point where the muscles are so severely uncoordinated that patients must depend upon a wheelchair to get around.
“There is no effective therapy currently available for Friedreich ataxia, and it often results in premature death, usually when the patient is in his or her 30s or 40s,” said principal researcher Dr. Sanjay Bidichandani, who is the CMRI Claire Gordon Duncan Chair in Genetics and a professor of Pediatrics at the OU College of Medicine.
With the new three-year grant, Bidichandani plans to evaluate the effectiveness of a promising new class of drugs known as HDAC inhibitors in the treatment of Friedreich ataxia. HDAC inhibitors essentially help genes that are packaged too tightly to loosen up.
“It’s been 20 years since we helped find the gene for Friedreich ataxia, and now we recognize that the gene is packaged too tightly in the cell in people with this condition,” he said. “It’s what we geneticists call an epigenetic defect.”
As a result, the gene cannot be read effectively, and a key protein is not produced in sufficient quantities. This eventually leads to neurodegeneration and cardiac dysfunction. HDAC inhibitors help reverse this cell “packaging” problem.
“More efficient versions of the currently available HDAC inhibitors are being developed by our collaborators. So we are in the process of testing these, as well as existing HDAC inhibitors, to identify which are most efficient at reversing the specific epigenetic defect in Friedreich ataxia,” Bidichandani said, adding that they hope to identify one or several drugs that might eventually become an approved therapy for the disease in the near future.
This year, the Muscular Dystrophy Association received 350 grant applications, the largest number in its 65-year history of funding basic, translation and clinical neuromuscular research. Bidichandani’s grant was one of only 36 new grants funded this year and the only one in Oklahoma.
“We are extremely proud to be part of the effort to help find a cure for Friedreich ataxia,” Bidichandani said.
Powered by its big-picture perspective to accelerate treatments and cures across the broad spectrum of neuromuscular diseases, the Muscular Dystrophy Association recently awarded $10 million in new research grants to researchers worldwide.
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“These new grants are potential game-changers, a signal of our passionate resolve to helping kids and adults fighting neuromuscular diseases live longer and grow stronger,” said Valerie Cwik, M.D., MDA’s Executive Vice President and Chief Medical & Scientific Officer. “There’s more new drug discovery underway than ever before, and we’re determined to double the number of promising human clinical trials in the next five years.”
