Cell Therapies For CMT
Cell therapies involve the transplantation of human cells into an individual in order to slow or halt progression of disease. A number of different cell types can be used — including various types of stem cells. If the therapeutic cells are taken from and returned to the same individual, they are considered autologous. If they instead originate from a donor, they are allogeneic.
In some cases, a hybrid approach is used that combines elements of both cell and gene therapy. Cells may be removed from a patient, genetically modified to include a new gene, and then returned to the patient from which they were taken.
The goal in cell therapy is for the transplanted cells to counteract the effects of missing or dysfunctional proteins. With the restoration of normal protein function, it may be possible to preserve, maintain or restore physical function.
CMT Research Foundation is leading the charge to ensure safe and effective therapies for all forms of CMT make it to the market to help those living with CMT today. We are working every day to support the families, the foundations, the academics, the scientists, the biotechs and the pharma companies who share our goal and who are working to make it happen.
Related News & Research
Is there a connection between CMT1X and Multiple Sclerosis?
A recent study by Georgios Koutis et al at the National and Kapodistrian University of Athens recently published a paper in The Journal of Neurology, Neurosurgery and Psychiatry suggesting emerging evidence of a connection between CMTX1 and Multiple Sclerosis (MS)....
CMT Research Foundation Adds Dr. Charles Abrams and Jeff Ellman To The Team
CMT Research Foundation (CMTRF), focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease, added Jeff Ellman to its Board of Directors and Charles Abrams MD, Ph.D, to its Scientific Advisory Board. CMT is a progressive genetic nerve disease...
Two papers were recently published regarding CMT1B and Axonal Neuregulin 1 Type III (Nrg1TIII), a protein which builds myelin (the insulation) on the nerves.
In summary, there is a molecule which blocks the Nrg1TIII in CMT1B, causing the demyelination of the nerves. Both papers found that if the strength or amount of Nrg1TIII can be controlled, the nerves affected by CMT may be restored. When more of the protein is added...
Why FDA acceptance of gene therapy in SMA matters to CMT
Novartis's announcement about FDA filing acceptance could translate to CMT On Monday, Novartis, the parent company of AveXis, announced that a license application for their gene therapy approach to treat Spinal Muscle Atrophy (SMA) was accepted by the FDA. This is a...
Fontan Surgery for Heart Defects May Not Be Advisable for CMT1A Patients, Case Study Finds
Charcot-Marie-Tooth News reports the findings of a study where patients with Charcot-Marie-Tooth disease (CMT) who undergo Fontan surgery to correct a heart defect in childhood may later develop difficulties with the diaphragm — the muscle that allows us to breath —...
Where We Started, How Far We Have Come and Where We are Going
The CMT Research Foundation offers hope to those living with CMT that a treatment and cure are on their way!
$1,000,000 pledge to CMT research
CMTRF Receives $1 Million Research Grant from Grandview Steers Foundation The pace of research for CMT just got faster. Atlanta (November 8, 2018) The newly launched CMT Research Foundation (CMTRF), the only 501(c)(3) not-for-profit organization focused solely on...
Key CMT Researchers added to Scientific Advisory Board
Gene Therapy, Adult Stem Cell and Clinical Experts are added to the CMT Research Foundation’s Scientific Advisory Board
Charcot Marie Tooth (CMT) Experts Launch National Nonprofit to Fund Only Treatments and Cures
CMT Research Foundation to speed dollars to most promising research Atlanta (September 19, 2018) Two experts in Charcot-Marie-Tooth (CMT), who also have the condition, today launched the CMT Research Foundation (CMTRF) as the only 501(c)(3) not-for-profit organization...
