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

CMT Research Foundation Announces New Research to Study Inflammation as Potential Treatment Target for Charcot-Marie-Tooth Disease

by | Oct 6, 2020 | , ,

The CMT Research Foundation is proud to announce a new research collaboration with renowned nerve disease expert Dr. Rudolf Martini of University Hospital Würzburg to investigate whether inhibiting inflammation in the peripheral nerves could reduce symptoms and improve outcomes for patients with Charcot-Marie-Tooth disease Type 1B. Read more about this project and see the four stages in the project.

WATCH: Power of the Patients: How We Change the Pace of Drug Development

by | Sep 24, 2020 | ,

The CMT Research Foundation is asking and answering the most pressing questions patients have about the need for treatments and cures. In episode 2, CMT Research Foundation CEO Susan Ruediger interviews Cure SMA’s Chief Scientific Officer Dr. Jill Jarecki, DTx Pharma Scientist Dr. Raffaella Gesuete and patient advocate Gary Donaldson to understand what patients can do today to influence the drug development process and speed progress. {HINT: Patients have the power to change everything!}

A Family’s Fight to Change Their Daughter’s Future

by | Sep 15, 2020 |

Eight-year-old Leonor struggles to walk, sit up straight and hold a glass of water. Other kids make fun of her because she has to wear leg braces. When Leonor was diagnosed with CMT, doctors said she’d ben in a wheelchair for life. Her family refuses to accept that fate, and so do we. There’s always something we can do. Learn how you can take action to end CMT.