Therapies That Can Arrest DMD
Several emerging therapies are significantly changing the way DMD is treated. Stem cell therapy, stem cell mobilization therapy, and (potentially) gene editing. RaceMD® is concentrating on intermediate therapies that may be applicable now. RaceMD® is in collaborations with some of the stem cell doctors who are applying current therapies.
Stem Cell Therapy
Stem cells are the building blocks of all cells in the human body. Every cell in our bodies begins as a stem cell and, through a series of genetic signalling, transform into the various cells that make up the human body. In Duchenne, a dysfuntion in the stem cell process prevents some of these stem cells from transforming into effective muscle.
Stem cell transplantation offers two types of therapies; allogeneic and autologous.
Allogeneic stem cell transplants are stem cells derived from a donor, via bone marrow, muscle fibers, fat tissue, and some times umbilical cord blood. The donor’s blood must be matched to the recipient to ensure there is no rejection of the stem cells post-transplant.
Autologous stem cell transplants take the patient’s own stem cells and reintroduce them into the body. This type of transplant does not pose a risk of rejection since the cells are derived from the patient themself. Both of these procedures have been performed in Duchenne patients and both types of stem cells (from donors and from patient) have shown promise in the treatment of Duchenne.
For a synopsis of potential stem cell therapies please read Rudnicki, 2016. Extensive stem cell research efforts in India at the Neurogen Brain and Spine Institute have yielded positive results. Dr. Alok Sharma, executive director of Neurogen and a global leader in stem cell therapy and Duchenne, has successfully treated over 400 Duchenne patients with stem cell therapy and over 800 patients with other dystrophies or other similar illnesses. In Ukraine, approximately 50 DMD patients were also treated with stem cell transplant therapy in clinical trials. France researchers conducted a clinical trial on stem cell transplant therapy for 10 throat muscular dystrophy patients. All clinical trials showed positive results.
Stem Cell Mobilizing Therapy
Along with stem cell transplantation, granulocyte-colony stimulating factor (G-CSF) has demonstrated promise as another therapeutic option for DMD. Commonly used prior to stem cell extraction, G-CSF has been shown to be therapeutic on its own as well. G-CSF therapy involves the mobilization of a patient’s own stem cells via intramuscular injection of the compound without the need for a stem cell transplant. G-CSF stimulates the release of stem cells from bone marrow. In animal models this allowed more stem cells to reach maturation, resulting in increases in skeletal muscle mass, strength, respiratory function and more satellite cells. Japan first demonstrated the efficacy of G-CSF therapy for DMD. Poland has initiated clinical trials. Studies in the United States have long-explored the safety of G-CSF for a variety of diseases and conclude that G-CSF can be a safe and effective therapy option for both adult and pediatric use.
Gene therapy is rapidly advancing the ability to treat Duchenne at a genetic level. DMD is caused in part by a mutation of the gene which produces dystrophin, the protien responsible for building healthy muscle. Researchers in the US, Canada and Japan have recently found a way to alter the genetic code through exon skipping in order to signal the body to produce funtional dystrophin. By skipping the mutated genes, a percentage of DMD patients are able to produce the dystrophin they lack. Seperate researchers have used gene editing techniques known as as CRISPR and Falon to correct the genetic errors in animal models of the disease.
For more complete explanations of the research and clinical trial basis for stem cell therapy, please visit www.duchennestemcellforum.org.