A study sponsored by Stem Cells Persia is investigating the safety and efficacy of autologous marrow-derived stem cell therapy in children and adults with Duchenne muscular dystrophy.

Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is an X-linked hereditary disorder that primarily affects boys and is characterized by intensifying muscle weakness.   The condition is caused by the hereditary absence of dystrophin protein which is essential for the integrity associated with muscle cells. Symptoms typically manifest at a young age (as early as 3-5 years old) and usually start with a weakness of the pelvic and lower limb muscle groups, spreading progressively to the upper extremities and trunk. When the affected person is in their teens, the muscles of breathing and the heart are also affected.

There is no cure for DMD. Current treatment can be symptomatic, consisting of braces, physical therapy, and corrective surgical procedure. Pharmacologic treatment can include steroids, anticonvulsants, and immunosuppressants to manage symptoms. Advanced medical care allows boys with DMD to reside into their early 30s and there are reports of success into the 40s and 50s.

Originate Cell Therapy for Duchenne Muscular Dystrophy

There are several exciting treatment strategies involving gene treatment and gene repair that are being investigated to find a cure intended for Duchenne muscular dystrophy. Stem-cell-based therapies are a promising method of treatment for this debilitating condition. There are two essential approaches to cell therapies for DMD. One is to genetically alter cells from a patient with DMD so that dystrophin expression is restored. These repaired cells are after that re-implanted into the patient through an autologous stem cell hair transplant. An allogeneic stem cell transplant, on the other hand, involves the transfer of functional dystrophin-producing cells from a healthy person.

New Study for Duchenne Muscle Dystrophy

Researchers have designed the single-arm single-center clinical trial to test the safety plus efficacy of mesenchymal stem cell treatment for DMD. Marrow-derived autologous mesenchymal stem cells will be administered with this open-label single group interventional study and outcomes is going to be measured in terms of improved muscle strength measured by MMT or kinetic muscle testing 12 months after the infusion. The particular Brooke and Vignos Scale will also be measured. The study desires to enroll 20 patients aged between 4 and quarter of a century old. Healthy people are not being accepted into the research as volunteers. The results are expected in March 2020.

References:

  1. https://clinicaltrials.gov/ct2/show/NCT03067831?term=stem+cells&sfpd_s=01%2F01%2F2017&sfpd_e=10%2F19%2F2017&draw=1&rank=4
  2. https://www.mda.org/disease/duchenne-muscular-dystrophy

Mira Swave, MD

Contributor at Regenerative Medicine Right now

Mira Swave, M. G. is a specialist in the field of Regenerative Medicine.

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