Physician at Hackensack University Medical Center Leads the first U.S. Clinical Trial of a Promising New Drug to TreatCharcot-Marie-Tooth Disease

November 1, 2018

Hackensack Meridian Health Hackensack University Medical Center announced that an international phase 3 study of a novel treatment for Charcot-Marie-Tooth (CMT) hereditary neuropathy, which in the United States was led by Florian P. Thomas, M.D., Ph.D, chair of the Neuroscience Institute and the Department of Neurology, and director of the Hereditary Neuropathy Foundation Center of Excellence at Hackensack University Medical Center, yielded positive results.

CMT disease is a chronic, often severe and debilitating inherited condition affecting some 125,000 people in the United States, of which approximately 50 percent have a particular subtype called Charcot-Marie-Tooth type 1A disease (CMT1A). It causes progressive weakness and impairs sensation resulting, among many other symptoms, in weakness, joint deformities and difficulty walking.

“While CMT is not life-threatening in most cases, it does lead to significant disability and comorbities,” explains Dr. Thomas. “We are excited at the prospect of taking our findings to the Food and Drug Administration and applying for approval of this important new treatment option for our patients at the Hereditary Neuropathy Center.”

For this trial Pharnext, a pharmaceutical company based in France, had developed a novel therapy, PXT3003, designed for patients with mild to moderate CMT1A. The study assessed the efficacy and safety of PXT3003 compared to placebo in over 300 participants in Europe, the USA and Canada.

“While in our institution, child and adult neurologists, geneticists, genetic counselors, rehab specialists and counseling psychologists work together to help patients with safe ambulation, hand function, disease adjustment and family planning,” adds Dr. Thomas. “Until now we had no options to slow disease progression. Now we have reasons for optimism that the FDA will approve the first treatment designed to directly affect the disease process.”

In 2016, Hackensack University Medical Center partnered with the Hereditary Neuropathy Foundation (HNF) to address CMT hereditary neuropathies, serving as a hub for clinical care, community engagement, research, training of health care professionals and education for the CMT community.

“Hackensack University Medical Center is one of HNF’s leading Centers of Excellence supporting patients through their interprofessional approach for patients and families with CMT,” said Allison Moore, founder and CEO of the Hereditary Neuropathy Foundation. “Dr. Thomas’ empathic approach in evaluating and treating patients is unique. His mission is to improve patient outcomes and quality of life by pursuing clinical trials of novel medications that can help people with CMT.”

Allison, who has CMT, often consults with Dr. Thomas on approaches to HNF’s research initiatives, clinical study designs and for her own medical care.

About The Neuroscience Institute at Hackensack Meridian Health Hackensack University Medical Center
The Neuroscience Institute team at Hackensack University Medical Center works collaboratively to evaluate, diagnose and treat patients living with hereditary neuropathies (CMT). The Institute is led by Florian P. Thomas, M.D., Ph.D., a leader in the field of hereditary neuropathy and multiple sclerosis. Dr. Thomas has engaged in CMT research for more than 25 years and has published on several subtypes, identified, with an international team of collaborators a novel CMT gene, and spearheaded, in the United States, the first two treatment trials for some of the most common subtypes of CMT.

About Hackensack Meridian Health Hackensack University Medical Center
Hackensack Meridian Health Hackensack University Medical Center, a 781-bed nonprofit teaching and research hospital located in Bergen County, NJ, is the largest provider of inpatient and outpatient services in the state. Founded in 1888 as the county’s first hospital, it is now part of one of the largest networks in the state comprised of 33,000 team members and more than 6,500 physicians. Hackensack University Medical Center was listed as one of the top two hospitals in New Jersey in U.S. News & World Report’s 2018-19 Best Hospital rankings. It was also named one of the top five New York Metro Area hospitals. Hackensack University Medical Center is one of only five major academic medical centers in the nation to receive Healthgrades America’s 50 Best Hospitals Award for five or more years in a row. Becker’s Hospital Review recognized Hackensack University Medical Center as one of the 100 Great Hospitals in America 2018. The medical center is one of the top 25 green hospitals in the country according to Practice Greenhealth, and received 25 Gold Seals of Approval™ by The Joint Commission – more than any other hospital in the country. It was the first hospital in New Jersey and second in the nation to become a Magnet® recognized hospital for nursing excellence; receiving its fifth consecutive designation in 2014. Hackensack University Medical Center has created an entire campus of award-winning care, including: the John Theurer Cancer Center; the Heart & Vascular Hospital; and the Sarkis and Siran Gabrellian Women’s and Children’s Pavilion, which houses the Joseph M. Sanzari Children’s Hospital and Donna A. Sanzari Women’s Hospital, which was designed with The Deirdre Imus Environmental Health Center® and listed on the Green Guide’s list of Top 10 Green Hospitals in the U.S. Hackensack University Medical Center is the Hometown Hospital of the New York Giants and the New York Red Bulls and is Official Medical Services Provider to THE NORTHERN TRUST PGA Golf Tournament. It remains committed to its community through fundraising and community events especially the Tackle Kids Cancer Campaign providing much needed research at the Children’s Cancer Institute housed at the Joseph M. Sanzari Children’s Hospital. To learn more, visit www.HackensackUMC.org.