A Clinical Study Shows Safety and Efficacy of Autologous Bone Marrow Mononuclear Cell Therapy to Improve Quality of Life in Muscular Dystrophy Patients

Abstract

Muscular dystrophy is a genetic disorder with no definite cure. A study was carried out on 150 patients diagnosed with muscular dystrophy. These included Duchenne muscular dystrophy, limb-girdle muscular dystrophy, and Becker muscular dystrophy variants. They were administered autologous bone marrow-derived mononuclear cells intrathecally and intramuscularly at the motor points of the antigravity weak muscles followed by vigorous rehabilitation therapy. No significant adverse events were noted. Assessment after transplantation showed neurological improvements in trunk muscle strength, limb strength on manual muscle testing, gait improvements, and a favorable shift on assessment scales such as the Functional Independence Measure and the Brooke and Vignos Scales. Furthermore, imaging and electrophysiological studies also showed significant changes in selective cases. On a mean follow-up of 12 ± 1 months, overall 86.67% cases showed symptomatic and functional improvements, with six patients showing changes with respect to muscle regeneration and a decrease in fatty infiltration on musculoskeletal magnetic resonance imaging and nine showing improved muscle electrical activity on electromyography. Fifty-three percent of the cases showed an increase in trunk muscle strength, 48% showed an increase in upper limb strength, 59% showed an increase in lower limb strength, and approximately 10% showed improved gait. These data were statistically analyzed using Student's paired t test and found to be significant. The results show that this treatment is safe and efficacious and also improves the quality of life of patients having muscular dystrophy. This manuscript is published as part of the International Association of Neurorestoratology (IANR) supplement issue of Cell Transplantation.

A Case of Autism Showing Clinical Improvements after Cellular Therapy along with PET CT Evidence

“…Their safety and efficacy has already been established in other neurological disorders. [19][20][21][22][23][24][25][26] These cells are a mixture of various hematopoietic and non hematopoietic cells which are more potent in combination as compared to their individual fractions. On transplantation, they migrate to the damaged areas and initiate repair.…”

Investigational treatments and therapeutic targets in Becker muscular dystrophy

“…First, autologous bone marrow mononuclear cells, which have myogenic potential, were infused, and provided evidence for the putative benefits of cellular therapy in altering the disease progression [55,56]. Second, boosting of NO-cGMP signaling is a target for BMD treatment, because nNOS is deregulated in BMD [57].…”

Neuroregenerative Rehabilitation Therapy with long-term Lithium in a Male Amyotrophic Lateral Sclerosis Patient: A Case Report.

“…Being autologous, these cells are a safe treatment modality; and, because these are adult stem cells, they are not prone to developing tumorigenic attributes. Multiple clinical trials, in a variety of neurological ailments, have demonstrated the safety and efficacy of adult autologous BMMNCs as a therapy [12][13][14]41 . None of the studies have shown any major or irreversible adverse events post transplantation.…”

“…Effective therapies for halting disease progression and improving quality of life are the need of the hour. Various studies worldwide advocate the efficacy and safety of autologous bone marrow-derived mononuclear cells (autologous BMMNCs) as a therapeutic intervention for neurological disorders [12][13][14] , including for ALS [15][16][17] . Lithium not only increases the survival and potency of these transplanted cells 18 , but is also safe when administered to ALS patients 19,20 .…”