Duchenne’s Muscular Dystrophy Patient Receives First Umbilical Cord Stem Cell Treatment in the USA

Ryan Benton, now 31 years old, is Duchenne's Muscular Dystrophy patient who receives first umbilical cord stem cell treatment approved by FDA, in the USA.

The approval of this individual compassionate treatment represents the achievement of 5 years of hard work, personal involvement and persuasive communication. But all these were supported by the medical records of his successful previous stem cell treatments performed in Panama since 2009, as most important argument.

As a result, since September 2014, Ryan could benefit by the stem cell treatment 2 times per year until, in his hometown, Wichita from Kansas.

Administration of stem cell treatment is done under the legal form of an approved clinical study: “Allogeneic Human Umbilical Cord Mesenchymal Stem Cells for a Single Male Patient With Duchenne Muscular Dystrophy (DMD)”, lead by Maurice HV Strickland, MD as principal investigator.

In January 2016, another important milestone was achieved by his family, treating physicians and all their friends from local muscular dystrophy community.

FDA has granted a third stem cell treatment per year for Ryan and also, approved the stem cell treatment for a second child, 6 years old who also had a positive history of previous treatments done in Panama.

In 2016 this second patient received his first treatment performed in USA.

Stem cell treatment an innovative therapy for Duchenne Muscular Dystrophy?

Stem cell treatment is an innovative experimental therapy for Duchenne Muscular Dystrophy. Based on preclinical studies it is assumed that stem cell might promote muscular regeneration and decrease the inflammation, as means of increasing muscular strength and alleviate symptoms.

Ryan is one of the firsts patients to whom were proposed stem cell intramuscular injections, as therapy which might have the potential to reverse the progression of disease.

By the end of 2007, at age 22, shortly after the first round of injections he noticed an increase in muscle strength which he never experienced before.  He didn’t experience any side effect.

After the second round of stem cell injections he underwent a muscular biopsy. The result showed an increase of muscular dystrophin level up to 50-100, which are the normal value for this muscular protein. In comparison at age 3 when Ryan was diagnosed with DMD the dystrophin level found at muscular biopsy was 0 to 5, which is characteristic for this type of disease.

During next year, Ryan stopped stem cells injections and as a result he experienced a decline in his muscular strength due to progression of muscular deterioration.

In the next 5 years, when he was able to travel and financial support only once a year basis therapy he noticed a pattern.  Shortly after stem cell treatment a major increase of muscular strength occurred for about 4 months and then a slowly, but progressive decline.

The conclusion he comes to was that most probably for a sustained and constant therapeutic effect the stem cell injections would have to be repeated at 3-4 months.

In what consists of the stem cell treatment for Duchenne Muscular Dystrophy?

During the clinical trial Ryan receives 4 intramuscular stem cell injections, on consecutive days, during 1 therapeutic round. For the first 2 years of the clinical trial were approved 2 therapeutic rounds (1 at 6 months) and for the last year, an additional 3rd round(1 round at 4 months).

Previously when the treatment was performed in Panama by Dr. Neil Riordan, the therapeutic rounds took about one week and were repeated at 2-3 months intervals in the first year.

The source of stem cells used for the treatment is donated umbilical cord tissue and the type of cells is adult mesenchymal stem cells derived from umbilical cord tissue.

The stem cell technology utilized in the experimental stem cell treatments performed in Panama and also, in the clinical trial approved by FDA was developed by Dr. Neil H. Riordan, PhD.

Why is necessary more than 1 round of  umbilical cord tissue derived stem cells injections?

In Ryan’s case a significant improvements have been noticed both by the patient, the treating doctors and his family. Following each round of stem cell injections it could be seen an increase on overall health, stamina, physical strength, muscle mass and ease in ability to breathe.

But these positive outcomes were limited only for 3-4 months, followed by decline. As a result there is necessary for periodically administration of stem cells in order to achieve a sustained positive outcome and more effectively control the disease.

This might happen because:

  • Umbilical cord derived stem cells from a donor usually at an average of 3-6 months are removed from the patient’s body by his/her immune system which recognize them as “foreigner cells”
  • Umbilical cord derived mesenchymal stem cells promotes antiinflamatory effect and muscular regeneration, most probably by means of  active factors (e.g. cytokines or growth factors) secreted by cells (so called paracrine mechanism) . These effects might overcome the underlying mechanisms responsible for Duchenne Muscular Dystrophy clinical symptoms but can’t correct the primary defect responsible for this disease: the X chromosome defect. This might be done through gene therapy or other methods, but these are in the early stages of research.

Therefore it’s very important to understand the intimate mechanism by which stem cells might exert their therapeutic effect in order to set the right expectations.

Ryan's story could be one of the cases which might support the use of stem cell treatment, as new therapeutic approach which seems to offer at least a part of therapeutic solution for increasing the quality of life and extend the life expectancy for patients with DMD.

But still this is a very early stage of using stem cells for this kind of conditions.

Further clinical studies are needed before setting if, and which might be the therapeutic outcomes, which source of stem cells is the safest and more effectiveness and which would be the best treatment algorithm.

You can watch Ryan's story told by himself.

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