Phase 3 Trial for CMT 1A Underway at Hackensack University Medical Center

Rare genetic disease currently has limited treatment options to counter nerve degeneration

Florian P. Thomas, M.D., Ph.D., chair of the Neuroscience Institute and Department of Neurology at Hackensack University Medical Center and founding chair and professor of Neurology at Hackensack Meridian School of Medicine, is leading a Phase 3 study on a new treatment for Charcot-Marie-Tooth 1A (CMT 1A), a rare genetic disease with limited treatment options. 

The degenerative nerve disorder process begins in utero, but may not show symptoms until childhood, adolescence or even late adulthood. It often starts with neuropathy in the fingers or two, which over time spreads up the lower portions of the limbs. It can affect the ability to grasp thin objects and cause gait issues, including foot drop. CMT 1A’s genetic factors cause an imbalance in protein production which affects nerve function.

Dr. Thomas has led multiple clinical trials for CMT, which will hopefully lead to the first available drug treatments. In the Phase 3 trial he’s currently leading, the drug is designed to reduce excess protein expressed by duplicate genes. The regimen consists of low doses of active ingredients already used in clinical practice for more than three decades, boosting Dr. Thomas’ confidence in their safety. The trial is fully enrolled with 13 patients in his center alone and currently collecting data.

Hackensack University Medical Center is the largest center in New Jersey set up to address the needs of CMT patients, with efforts led by Dr. Thomas. The range of services available to patients includes psychological counseling, genetic counseling, orthopedic surgery, physical and occupational therapy, neurology – and access to clinical trials.

Learn more about neurological innovations at Hackensack University Medical Center.