Florian Thomas, M.D., Ph.D.
Director of the Hereditary Neuropathy Foundation Center of Excellence, Chair of the Department of Neurology at Hackensack University Medical Center and Hackensack Meridian School of Medicine at Seton Hall University
Updated: May 7, 2019
Florian Thomas, M.D., Ph.D., director of the Hereditary Neuropathy Foundation Center of Excellence and chair of the Department of Neurology at Hackensack University Medical Center and Hackensack Meridian School of Medicine at Seton Hall University, will be recognized for his scientific achievements during the 71st American Academy of Neurology (AAN) Annual Meeting in Philadelphia May 4-10.
A leader in the field of Charcot-Marie-Tooth neuropathy (CMT), Dr. Thomas will present a successful Phase 2 study and results from a Phase III clinical study for which he served as principal investigator at the 2019 AAN Annual Meeting.
Dr. Thomas’ Phase 2 Study
Dr. Thomas’ abstract titled “Results of the dose-escalation portion of a Phase 2 Study of ACE-083, a Local Muscle Therapeutic in Patients with Charcot-Marie-Tooth (CMT) Disease” was selected as a 2019 Abstract of Distinction and is scheduled for May 10.
Dr. Thomas’ Phase 3 Study
In addition, Dr. Thomas’ Emerging Science abstract, titled “Efficacy and safety of PXT3003 in patients with Charcot-Marie-Tooth type 1A (CMT1A): results of PLEO-CMT, an international pivotal Phase 3 trial,” was one of 11 abstracts selected for dual presentations—both platform and poster—at the AAN meeting on May 7.
Results from the Studies
Patients with CMT suffer from muscle shrinkage and weakness as well as sensory loss; this affects walking, activities of daily living and quality of life. Until now no treatment has been available to stabilize or reverse the disease. In the phase 2 study, Dr. Thomas and his co-investigators show that the study drug, when injected into the leg muscle that lifts the foot, increases muscle volume. In the phase 3 study, results generated by this clinical trial, found the study drug as potentially the first treatment for CMT1A, the most common subtype of CMT, to be safe, well-tolerated, and effective at improving disability.
More about Dr. Thomas’ CMT Research
Dr. Thomas has engaged in CMT research and advocacy for more than 30 years. He has published on several subtypes, identified, with an international team of collaborators, a novel CMT gene and spearheaded in the United States two of the first treatment trials for the most common subtype of CMT. Dr. Thomas and his team work collaboratively to evaluate, diagnose, and treat patients living with CMT.