May 23, 2019
Hackensack Meridian Health Hackensack University Medical Center is pleased to announce that Florian Thomas, M.D., Ph.D., director of the Hereditary Neuropathy Foundation Center of Excellence and chair of the Department of Neurology at Hackensack Meridian Health Hackensack University Medical Center and Hackensack Meridian School of Medicine at Seton Hall University, has been recognized for scientific achievements at 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 presented results from a Phase III clinical study, for which he served as national principal investigator, at the 2019 AAN Annual Meeting.
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 selected for dual presentations at this year’s AAN meeting. CMT is a hereditary neuropathy that affects one in 2,500 people. Patients 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. Based on the results generated by this clinical trial, sponsored by Pharnext, Dr. Thomas and his co-investigators 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.
The AAN Science Committee selected Dr. Thomas’ Emerging Science presentation as one of 11 abstracts identified for both platform and poster presentations.
“A renowned neurologist and scientist, Dr. Thomas models our mission to transform health care and serve as a leader of positive change,” said Mark D. Sparta, FACHE, president and chief hospital executive, Hackensack University Medical Center. “We are proud of Dr. Thomas’ most recent achievements in service of our patients, their families, and our community.”
More broadly, 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.
In describing their philosophy and approach, Dr. Thomas said, “We provide patient-centered care to address the physical and emotional needs associated with chronic neurological illnesses and to empower people to be knowledgeable about their conditions, engage in their own care, optimize health behavior, avoid complications, advocate effectively for themselves, and live their lives to the best of their potential. It has been very frustrating for our patients that until now no treatment options existed to improve the long term prognosis. It is very rewarding that, through this and other research, potential treatment options are starting to appear on the horizon.”