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UCLA Researchers Have Broken Ground in DMD Stem Cell Therapy

Posted by Douglas W. Stoddard MD, M Sp Med, Dip Sport Med, ES on 2 January 2018
UCLA Researchers Have Broken Ground in DMD Stem Cell Therapy

Duchenne muscular dystrophy (DMD) is a genetic disorder effecting 1 in 5,000 adolescent males in the U.S.

As the most common fatal childhood genetic disease, DMD is best explained as a progressive muscle degeneration and weakness. An absence of dystrophin - a protein keeping muscle cells intact - causes DMD. It's found mostly in boys, starting between ages 3-5.

Initially, DMD attacks hip, pelvic, thigh, and shoulder muscles. As it progresses, it spreads to the skeletal (voluntary) muscles in the arms, legs and trunk, often enlarging the calves. The heart and respiratory muscles are weakened during the early teens, leading to low life expectancies in those afflicted.

While those with DMD usually don't survive beyond their teens, researchers from UCLA have found a way to isolate, mature, and transplant skeletal muscle cells created from human pluripotent stem cells.  It can produce all cell types of the body and pave the way for a stem cell replacement therapy for muscle diseases, including Duchenne Muscular Dystrophy.

The Problems of the Past

Human pluripotent stem cells are self-replicating, derived from human embryos or fetal tissue, and develop into cells and tissues of the three primary germ layers.

Scientists were already utilizing human pluripotent stem cells to generate skeletal muscle cells able to function in living muscle, while regenerating dystrophin-producing muscle fibers.

But UCLA's research team found that current methods can only produce immature cells incapable of producing a cell replacement therapy for DMD. In layman's terms, lab-produced skeletal muscles aren't fully functional.

A Future Solution?

The healing potential of cells generated from human pluripotent stem cells versus the muscle stem cells found during the development process is yet to be fully understood.

However, the UCLA researchers found a regenerative fetal skeletal muscle cell, containing two new cell surface markers (ERBB3 and NGFR), enabling the precise isolation of muscle cells from human tissue. The cells can be separated from many cell types, to regenerate using human pluripotent stem cells.

Matured cells helped construct uniformed muscle cells with fibers smaller than cells in real human muscle. UCLA discovered a specific cell-signaling pathway - called TGF Beta - that needs to be deactivated to enable generation of protein-rich skeletal muscle fibers aiding in muscle contraction.

Beyond the Lab

April Pyle is an associate professor, and a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. Pyle believes the results have provided the exact information researchers wanted.

"This is the first study to demonstrate that functional muscle cells can be created in a laboratory and restore dystrophin in animal models of Duchenne using the human development process as a guide," claims Pyle.

There have been many other stem cell research initiatives with the same goal in mind, but the UCLA study is the most successful to date, although it's still in the stages of infancy.

Hopefully, with further research and funding, those with DMD could be blessed with added decades to their lives, and a chance to push beyond the restrictions of the debilitating condition.

The healing potential of stem cells is still largely untapped. And while RegnerVate isn't performing DMD Stem Cell Therapy, we do provide various PRP and stem cell injection therapies that can effectively treat chronic conditions afflicting your muscles and joints.

If you're suffering from conditions such as osteoarthritis, tendonitis, or plantar fasciitis (to name a few), contact us to see if you can alleviate your pain in a non-invasive way.

Author: Douglas W. Stoddard MD, M Sp Med, Dip Sport Med, ES
About: Dr. Stoddard is a sport medicine and injection physician in Toronto and is the Medical Director of RegenerVate. After receiving his medical degree from the University of Toronto, he trained in Australia at the Australian Institute of Sport in Canberra, obtaining his Master Degree in Sport Medicine. His injection training, including ultrasound, PRP and Prolotherapy, was primarily done in the USA. He is a diplomat of the Canadian Academy of Sport and Exercise Medicine (CASEM), is married and a proud father of two boys. He is an avid triathlete and occasional guitar player.
Tags: Stem Cell Research

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