Stem cells are cells that have the unique ability to self-renew and to transform into many specialized cell types. They can be isolated either from embryos or from adult tissue. Adult stem cells are being used for various medical therapies in which they are grown and differentiated into different cell types, and then introduced into patients to repair or replace damaged tissue. Bone marrow transplantation, for instance, is a form of stem cell therapy.
Research is underway to develop and apply stem cell treatments to various diseases and conditions, including muscular dystrophy. One line of research involves removing stem cells from patients, using gene editing technologies to correct the disease-causing mutation, and transplanting these corrected cells back into muscles. C3 recently awarded a grant to Dr. Rita Perlingeiro, Professor of Medicine at University of Minnesota, to investigate this therapeutic strategy for LGMD2A. However, these treatments will still take years to develop.
Unfortunately, there are numerous stem cell therapies that are currently being advertised to patients that have not been proven to treat any disease. The clinics advertising these therapies are exploiting the hopes of patients and their families. Not only are the treatments unproven and unlikely to be beneficial, but they can even harm patients. Additionally, patients who undergo these procedures may be ineligible to participate in future clinical trials.
On August 28, the Food and Drug Administration (FDA) announced a crackdown on ‘unscrupulous’ stem cell clinics, as well as policy that will ease the path for the development of legitimate treatments in this field. The full FDA statement can be accessed here.
No stem cell therapies have been developed yet for calpainopathy/LGMD2A. We at C3 are dedicated to funding research to find a cure for this disease.