CMTRF investigates a gene therapy program for CMT1A

CMT Research Foundation Partners with Cyprus Institute for Neurology and Genetics’ Kleopas Kleopa to Launch Study of Possible Gene Therapy for CMT1A

This is the second major investment by CMTRF.

ATLANTA (January 16, 2019) The newly launched CMT Research Foundation (CMTRF), a nonprofit focused solely on delivering treatments and cures for Charcot-Marie-Tooth, today announced it has partnered with Dr. Kleopas Kleopa at the Cyprus Institute for Neurology and Genetics to launch a two-year study of the application of gene therapy for CMT1A.

Nearly 3 million individuals of every age, race and ethnicity have a form of CMT, a debilitating, genetic disease of which there are many variants. Because CMT is progressive, patients continue to lose function as they age, creating a greater degree of disability. Currently, there is no cure or any effective treatments for CMT. CMT1A is the most common type of CMT and is caused by a duplication of PMP22 gene.

People normally have two copies of the PMP22 gene. Those with CMT1A have three copies resulting in excessive gene dosage. Dr. Kleopa and his team will use a gene therapy approach to try to reduce the excessive amount of PMP22 in a pre-clinical model of the disease. It is hoped that this treatment could stop the progression of the CMT1A and perhaps even reverse some of the damage it causes. The research team will utilize viral vectors that have been used in clinical trials for other disorders, increasing the potential for translation in the clinic.

“Just four months after starting the CMTRF, we have research programs in place to attack two types of CMT which effect roughly 60% of people with CMT,” says Susan Ruediger, CEO of CMTRF. “Dr. Kleopa is a renowned neurologist and highly regarded gene therapy expert whose approach is proven in other genetic neurological diseases. This particular strategy has never before been applied to CMT1A and has the potential to deliver treatment options.”

“We are excited to work on this project for the development of gene therapy for CMT1A. We hope that this work will provide a clinically translatable approach to help patients,” says Dr. Kleopa. “We thank the CMTRF for supporting our efforts and for their enthusiasm and commitment to expedite therapies for CMT patients worldwide.”

Dr. Kleopa is a Professor at the Cyprus School of Molecular Medicine and Coordinator of the Neuroscience MSc/PhD Program. He completed his medical and doctoral studies at the University of Wuerzburg, Germany in 1994, and a Neurology residency at Drexel University in Philadelphia in 1999, followed by a 2-year fellowship in neuromuscular disorders and electromyography at the University of Pennsylvania. He was appointed Clinical Instructor in Neurology at the Faculty of the Hospital of the University of Pennsylvania in 2001 before joining the Cyprus Institute of Neurology and Genetics in 2002, where he was appointed Senior Consultant Neurologist. He established specialized neuromuscular and electromyography clinics, as well as the Neuroscience Research Laboratory focusing on translational studies in demyelinating and neuromuscular disorders. He has been the principal investigator in at least 20 successful externally funded research projects in the last 18 years, including gene therapy applications for inherited neuropathies. He has published more than 80 peer reviewed papers and 15 book chapters and has received several national and international awards for his work.

Established in 1990 as a non-profit institution, The Cyprus Institute of Neurology & Genetics is an internationally recognized Centre of Excellence offering high level specialized services as a tertiary referral center for neurological disorders in the region, as well as innovative translational research programs and postgraduate education in the areas of neurology, genetics, biomedical and medical sciences. The ultimate goal of CING is to improve the quality of life of people at the national and international level. The Cyprus School of Molecular Medicine is the postgraduate school of The Cyprus Institute of Neurology and Genetics.

The CMT Research Foundation (CMTRF) is focused solely on delivering treatments and cures for CMT. Founded by two patients who are driven to expedite drug delivery to people who live with CMT globally, the organization funds research and drug development. The 501(c)(3) federal tax-exempt organization is supported by personal and corporate financial gifts.