DMD: Your Guide to Stem Cell Treatment.

Free Download: Stem Cell Therapy for Duchenne Muscular Dystrophy

Disclaimer: 

The information provided by R3 Stem Cell is for educational purposes and is not a substitute for professional medical advice, diagnosis, or treatment. Individual results may vary and are not guaranteed. The FDA considers stem cell therapy experimental at this point.

 

Any claims made on this website refer to procedures performed OUTSIDE of the USA. R3 Stem Cell has clinics in Mexico, Philippines, South Africa, Turkey, India, Pakistan.

Please fill out the form below to receive the guide

Consumer Guide to Stem Cell Treatment for Duchenne Muscular Dystrophy

Every day, R3 Stem Cell receives inquiries worldwide from individuals asking if stem cell therapy can help with Duchenne’s Muscular Dystrophy (DMD). Spoiler alert: It can help a lot! In this guide, we’ll go through the basics of how stem cells work for DMD, the latest research, and what to expect with a regenerative procedure.

A Significant Global Issue

Duchenne muscular dystrophy (DMD) is a progressive degeneration of the striated muscles of the body and has a fatal prognosis. It primarily affects the skeletal and cardiac muscles of male children with the occurrence of 1 in 3500. It is one of the deadliest muscular disorders with no definite cure available currently. Since 90% of the male sufferers die by the age of 21 years due to cardiorespiratory failure, it has become a global cause of concern. The age of onset of DMD is early childhood but most children become wheelchair bound by the age of 10-12 years.

 

The disease is caused by mutation, deletion, or duplication of the dystrophin gene, leading to synthesis of functionally impotent dystrophin. Dystrophin is a protein essential to maintaining the integrity of the exoskeleton of the muscle cells. Dystrophin is also a structural component of neurons, glial cells, and Schwann cells. 

The disease manifests as progressive weakness of the muscles, leading to loss of function. Children typically exhibit loss of ambulation in the second decade of life, followed by premature death. Treatment of muscular dystrophy consists of medical, rehabilitation, and surgical management aiming to preserve the function of the individual and prolong their independence. These treatments contribute very little towards altering the pathology of the disease.

Hope for DMD: See if stem cell therapy can help. Call (844) GET-STEM.

Traditional Treatments for Duchenne’s Muscular Dystrophy

DMD remains an incurable disease, although several medicines, including corticosteroids, are used to delay the process of muscle degeneration. Glucocorticoids are widely used for its anti-inflammatory property, however there are significant side effects including weight gain, diabetes, fragile bones, increased risk of fractures, reduced growth and suppressed immune system.

 

Physiotherapy for DMD patients such as stretching and mobility exercises can aid in temporary muscle flexibility. However, these treatments lose effectiveness in later stages of the disease. Therefore, there is a great need to develop an effective clinical intervention that is capable of arresting the disease progression.

Stem Cell Therapy for Duchenne’s Muscular Dystrophy

If a new technology such as mesenchymal stem cell and exosome therapy could provide a long term solution for Duchenne’s Muscular Dystrophy, it would and should become first line therapy.

 

A regenerative therapy that can improve a child’s function that is a safe, nonsurgical option should receive consideration.

Case Report

In a 2014 Case Report, researchers treated A 9-year-old boy who had a history of difficulty in getting up from the floor, climbing stairs, and frequent falls since the age of 4 years. The increasing weakness in the lower limbs led to toe walking and loss of ambulation by the age of 8 years. The weakness then progressed to the upper extremities and performing overhead activities was difficult. Diagnosis of DMD was confirmed based on the clinical features, elevated serum creatinine phosphokinase (CPK) levels (5460 IU/L), and genetic analysis with multiplex polymerase chain reaction for dystrophin gene showing deletion of exons 51 and 52.

Treatment Approach

The boy was treated with serial autologous bone marrow mononuclear cell transplantations followed by multidisciplinary rehabilitation. Brooke-Vignos score was 10 and he was wheelchair bound. Over 36 months, gradual progressive improvement was noticed in muscle strength, ambulation with assistive devices, fine motor movements, Brooke-Vignos score, and functional independence measure score. Nine months after the transplantation, electromyography findings showed the development of new normal motor unit potentials of the vastus medialis muscle.

A total of 33 million bone marrow mononuclear cells were administered both intrathecal (spinal cord) along with intramuscular at various points. The patient underwent serial cellular transplantations at 9, 21, and 31 months after the first transplantation with the same regimen.

Role of Satellite Cells in Muscle Repair

Repair and regeneration of these cells is engineered by local stem cells and infiltrating inflammatory cells. These local stem cells are termed “satellite” cells. There is, however, only a limited pool of satellite cells in the muscles; therefore, in DMD it cannot meet the increasing demands of accelerated cell necrosis. In the presence of increased necrotic tissue, the extracellular environment also becomes conducive to necrosis.

In our patient we used BMMNC transplantation followed by rigorous rehabilitation. It has been observed that physical activity facilitates the effectiveness of the stem cell transplantation in muscular dystrophy.

A 2015 Study: Umbilical Cord Stem Cell Therapy in DMD Patients

A 2015 study from India evaluated 14 participants with DMD. Nine received umbilical cord mesenchymal stem cells (UC-MSCs), with five control patients not receiving cells. The participants in Group A (Control) were not treated with UC-MSCs and were monitored over a period of 3 years, while the participants in Group B were injected with MSCs monthly for 4 months.

For each monthly SCT (Stem cell therapy), approximately 2 million UC-MSCs/kg body weight dissolved in normal saline were prepared for each patient. 1 million UC-MSC/kg bodyweight were injected slowly intravenously into the circulatory system of the patient, while 1 million UCMSC/ kg were injected intramuscularly, keeping a watch on vital parameters of the patient. The UCMSCs for intramuscular injection was divided into equal aliquots to be injected into the major muscles such as the quadriceps, hamstring muscles, calf muscles, deltoid muscle, gluteus major and minor, and paraspinal group of muscles.

Findings: Stability in Muscle Strength After Stem Cell Therapy

There was stability in muscle strength in DMD patients after MSCs transplantation. After 1 year (N=9), 4/9 subjects had a 1 unit increase in strength (mean=3.11 (0.78)). In comparison, control patients had shown progressive decline in muscle strength (3.2 ± 0.4472), after 1 year 3/5 subjects had 1 unit decrease in strength, (mean=2.6 (0.54)).

Overall, the researchers noted stability in the strength measurements in the treatment groups versus the control group, who experience declines. Overall this study establishes the clinical safety for allogenic UC -MSCs transplants in DMD patients. Combined case studies on all the DMD patients in this study clearly establish a beneficial role of MSCs in the treatment of DMD patients.

UC-MSCs administration to the DMD patients did not result into any deleterious effects on such patients and thus considered to be a safe and effective cellular therapy for DMD patients in age group 5 to 18 years. This can be used as alternative to glucocorticoid therapy to control decline in muscle power in DMD patients. However, patients need repeated booster sessions of SCT to maintain muscle power.

A 2021 USA Case Report on MSC and Exosome Therapy

A USA Case Report from 2021 evaluated treatment of an 11 year old male with DMD. The treatment protocol was administered over a 90- day period. Three consecutive treatments were performed. The first infusion was given on day 0, the second on day 30, and the third on day 60. MSCs and exosomes were mixed with processed platelet-rich plasma (PRP), then infused via peripheral IV over a 60-120 minute interval. Pretreatment with IV diphenhydramine (Benadryl), methylprednisolone, and cephalexin were infused 20-60 minutes before the infusion of MSCs/ exosomes.

Patient Outcomes

The patient showed promising improvement in several of the evaluated categories. As demonstrated in Table 1, the distance patient was able to walk in 15 seconds increased, the number of claps patient was able to make in 15 seconds increased, and the distance for standing long jump of both the right and left leg significantly increased.

The patient tolerated the infusions well with no adverse reactions. Both the patient and his parents reported positive improvement in his overall balance and dexterity from their daily observations throughout the entire protocol.

A 2021 Compassionate Use Study in Poland

A compassionate use study out of Poland was published in 2021 evaluating Wharton’s Jelly Umbilical Cord mesenchymal stem cells for muscular dystrophy. Treatment consisted of one to five intravenous injections per one treatment course of advanced therapy medicinal product containing WJMSCs. The first administration was always intravenous for safety reasons; the following injections were administered intravenously or intrathecally. The study group included 22 patients: 11 men and 11 women.

Results

In general, patients tolerated administrations well. Only one patient experienced transient headache and lower back pain after the last administration. Overall, the individual response to treatment was heterogeneous. In the most successful case, the patient began moving without a crutch, stopped rehabilitation, and rejoined a full-time job.

Inflammatory Processes in DMD and the Role of Stem Cells

The genetic component of muscular dystrophies cannot be resolved, but the consequences of the involved mutation may be ameliorated. A potential mode of action may be related to the antiinflammatory properties of these cells 57-60 because inflammatory. processes are involved in the pathogenesis of muscular dystrophies.

 

For instance, the tumor necrosis factor-α serum concentration in patients with DMD was increased eight times compared with healthy boys of the same age, and interleukin-6 was increased twice.

Our study showed objective and significant improvement in muscle strength in 12 patients (54.5%). This amelioration resulted in improved gait in three patients and improved results in a motor scale in another three patients; altogether, in 6 of 22 (27.3%) patients the benefit from the therapy was significant enough to ameliorate their clinical parameters.

2015 and 2018 Studies on WJ-MSCs for DMD Treatment

In 2015, Rajput et al described the administration of WJ-MSCs in 11 children with DMD and reported stabilization compared with a small (n = 5) control group. In 2018, Dai et al described an improvement in electromyography in nine patients with DMD after intra-arterial administration of UC-MSCs.

A 2015 Chinese Study on hUC-MSCs for DMD

A 2015 Chinese study evaluated human umbilical cord mesenchymal stem cell (hUC-MSCs) therapy on 3 patients. The transplantation of the hUC MSCs was performed by infusion with an intravenous drip over a 30 min period, and the patients were evaluated at 1, 3, 4 and 12 weeks following the procedure.

 

The evaluation was based on physical characteristics, as well as on molecular testing for serum creatine kinase (CK) and lactate dehydrogenase (LDH) levels and a histological examination of muscle biopsies.

Outcomes

The patients suffered no adverse reactions in response to the transplantation of the hUC MSCs. At 1 week following transplantation, all 3 patients showed improvement in the muscle force of the limbs, muscle size and daily activity. The walking gait had improved by 1 week post-transplantation and reached a normal status by 12 weeks. Serum CK and LDH levels were decreased relative to the baseline levels. 

 

A histological examination of muscle biopsies displayed no obvious tissue regeneration. In conclusion, the treatment of patients with BMD using hUC-MSCs was safe and of therapeutic benefit that lasted for up to 12 weeks. hUC-MSCs are, therefore, a potential cell therapy-based treatment option for patients with muscular dystrophies.

A 2018 Indian Study on Stem Cell Therapy in 150 Patients

A 2018 study out of India was published that evaluated 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: Neurological and Functional Improvements

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. 

 

In R3’s experience, 75% of patients with Duchenne’s Muscular Dystrophy achieve success with stem cell and exosome therapy. It’s an exciting option for patients!

Is Stem Cell Therapy Right for Your Child’s DMD? Call (844) GET-STEM to discuss.

Why Doesn’t R3 Stem Cell Use A Person’s Own Stem Cells for Duchenne’s Muscular Dystrophy?

R3 used to perform autologous therapies, where a patient’s own bone marrow or adipose stem cells were used. However, a lot of stem cells in one’s body are as old as that person is, and hence not very active. Their ability to successfully increase sufficient blood flow and allow for muscle repair is inferior to umbilical cord stem cells.

 

Specifically, the therapeutic potential of autologous bone marrow or adipose stem cells in the treatment of older patients is impaired by a number of age-related factors such as oxidative stress, telomere length, DNA damage, disease, and long-term use of some medications.

 

This is in stark contrast to the youthful genotype and phenotype of neonatal tissue-derived stem cells, such as from the umbilical cord. They are better at facilitating repair and regeneration of tissue damage, creating new blood flow with superior antiinflammatory and immunomodulatory efficacy compared to mature stem cells from one’s adipose or bone marrow.

 

As a result of the inferiority of autologous stem cells due to the reasons above and better results being seen with umbilical cord stem cells, R3 only uses the donor stem cells today

Tailored treatments await you. Contact (844) GET-STEM today!

How do the Stem Cells and Exosomes Work for Duchenne’s Muscular Dystrophy?

Stem cells and exosomes act in the body through several mechanisms. They do NOT become part of a patient’s DNA, which means they do not engraft into the person’s existing cells. The predominant method of action is thought to be through paracrine mechanisms, which means “cell to cell” interaction.

They act through

Angiogenesis

provokes formation of new blood vessels.

Reduce inflammation

Duchenne’s Muscular Dystrophy is associated with significant inflammation, and the regenerative biologics reduce it nicely

Immune system modulation

the stem cells and exosomes modulate the immune system very differently than steroids. Instead of blanketly suppressing the immune system, the regenerative biologics tamp down the harmful processes while amping up the beneficial ones. This includes ramping up production of several helpful growth factors and cytokines, while tamping down harmful ones.

Cellular signaling

the biologics are able to perform “cell to cell” communication. This promotes recipient cells to proliferate their growth factor production, protein production and regenerate nerve tissues that are damaged.

Prevent cell death

most cells have a timed death, where they are only allowed to live a certain length of time. This is called apoptosis. The regenerative biologics allow normally functioning cells (i.e. neuron cells) to live longer, and spare them from the pre-programmed death.

Preventing scar tissue

Duchenne’s Muscular Dystrophy patients may experience significant scarring throughout the course of the disease. Once that scar tissue forms, it becomes nonfunctional. Stem Cells and exosomes are great at preventing scar tissue (anti-fibrosis).

Stem Cells can also release a huge variety of molecules into the extracellular environment. These molecules, which include extracellular vesicles, lipids, free nucleic acids, and soluble proteins, exert crucial roles in repairing damaged tissue.

Along with offering MSCs for treatment of Duchenne’s Muscular Dystrophy, R3 Stem Cell often includes stem cell exosomes, which are a type of extracellular vesicle participating in extensive cell to cell communication for new blood flow creation.

Seeking a Different Approach to DMD? Discover stem cell therapy. (844) GET-STEM

Where do the stem cells and exosomes come from?

R3 Stem Cell’s regenerative biologics originate from umbilical cord tissue that has been donated after a scheduled c-section. No baby (or mother) is harmed during the c-section procedure. The umbilical cord tissue is normally discarded, but if the mother passes screening tests then the umbilical cord is immediately sent to the lab. The screening tests are extremely rigorous, and mandated by the USA FDA.

 

The lab carefully processes the umbilical cord to generate large amounts of stem cells and exosomes that are of the highest quality possible. The lab team consists of multiple PhD’s working in ISO Certified, cGMP compliant clean rooms to ensure quality assurance that exceeds USA FDA standards. The proprietary production process combines the highest potency, safety and affordability for providers to confidently offer exosome procedures.

 

Millions of dollars have been invested into the pharmaceutical grade production of the biologics including first rate clean rooms, bioreactors, nano-particle tracking analyzers, cytometers, PCR, tangential flow machines and real-time environmental monitoring. The quality assurance testing complies with screening and testing stan¬dards consistent with the American Association of Tissue Banks, cGMP standards, FDA regulations and the highest level of any regulatory agency globally.

Stem Cell Derived Exosomes

R3 Stem Cell’s Centers of Excellence globally include umbilical cord stem cell derived exosomes with umbilical cord stem cells to provide enhanced results. Exosomes are lipid bound vesicles (acellular) produced by cells that contain a plethora of growth factors, cytokines, mRNA and other proteins.


They are exceptionally helpful in cell to cell communication, and very effective for reducing inflammation when they become ingested by their recipient cell. They act as shuttles to send nucleic acids and proteins to other cells, in this way, allowing cell-to-cell communication and transporting molecules among both close and distant cells. In general, these released proteins are important regulators of intracellular information.

Exosomes could be the mediators of many stem cell-associated therapeutic activities. We have seen them to be “faster acting” than stem cells, so R3 frequently uses them in conjunction to provide a “1-2 punch” for patient outcomes.

Is stem cell therapy safe?

After a decade of performing over 24,000 stem cell procedures worldwide, R3 knows that the regenerative procedures are safe. The quality control employed during the stem cell production is second to none, and the side effects R3 sees are usually mild to moderate and temporary.

 

They may include itching, dizziness, lightheadedness, low grade fever, chills, headache, nausea. These are typically temporary. If a patient has an allergic reaction to the multivitamin or a preservative, all of R3’s Centers have the medications to resolve it quickly.

 

One of the questions we get asked a lot is, “Will the stem cells get rejected?” The answer is NO.

 

MSCs do not express major histocompatibility complex (MHC) antigens of the class II subtype and contain low levels of MHC molecules of the class I subtype. MSCs also lack the co-stimulatory molecules essential for immune detection, including CD40, CD80, and CD86.

 

Therefore, MSCs generally have low immunogenicity and can avoid immune rejection by the recipient, which serves as the foundation for their successful application without needing to match the donor to the recipient. Scientists call this being “immunologically privileged”.

 

Another question often asked is “Is there a chance of a tumor forming?” Current research has concluded that the answer is NO. The mesenchymal stem cells and exosomes used during treatment have never been shown to have tumor forming potential. In fact, they have been shown to be anti-tumor forming.

Want to know if stem cell therapy could benefit your child’s DMD? Call (844) GET-STEM

For the past decade, R3 has been successfully treating Duchenne’s Muscular Dystrophy with stem cell and exosome injections. The procedures are performed both intravenous and intrathecal.

 

Each patient receives a combination of the mesenchymal stem cells along with exosomes, and patients are given a multivitamin IV drip too. R3’s providers use approximately two million stem cells per kilogram for the procedure. As you can see in the research described above, typically treatments are necessary twice a year.

Similar to the research mentioned above, R3 Stem Cell’s outcomes for Duchenne’s Muscular Dystrophy have been exceptional! The patient satisfaction rate is 85% year over year. Patients typically experience improved muscle function and improved quality of life. Keep in mind results cannot be guaranteed and will vary between individuals.

 

It may take a couple of months to see all the improvements, as it can take that long to build up new muscle. It should be noted, again, that stem cell therapy is not a cure, and may need to be repeated every six months or so for continued benefit with DMD.

Because stem cell therapy for Duchenne’s Muscular Dystrophy is not a permanent cure, it’s important to make it affordable. Repeat therapies can help maintenance and/or achieve additional improvements for musculoskeletal health. So a lot of patients seek additional treatments at R3 Stem Cell every six months.

 

R3 Stem Cell’s fees are less than half what comparable (and reputable) regenerative clinics charge. Be wary of clinics trying to pass off PRP as a stem cell therapy. If they mention only taking your blood for the treatment, it is NOT a stem cell treatment!

R3’s Experience

For the past decade, R3 Stem Cell’s Centers globally have performed over 24,000 regenerative procedures in seven countries. Over fifty have been for Duchenne’s Muscular Dystrophy. Patient satisfaction across all conditions treated is 85%!

 

R3 combines safety, effectiveness and affordability for the therapies. Internationally, the Intellicell is used, which is culturing the most active mesenchymal stem cells to create the “smartest” stem cell in the world!

 

R3 Stem Cell offers free consultations for individuals to discuss whether regenerative therapy is indicated for their Duchenne’s Muscular Dystrophy. Simply call +1 (844) GET-STEM to schedule yours!

New to Stem Cell Therapy for DMD? Start with a free consultation: (844) GET-STEM

Disclaimer: 

This guide’s education does not constitute medical advice. The USA FDA considers stem cell therapy experimental. Any claims made in this Guide refer to procedures performed outside of the USA.

References

About R3 Stem Cell

David Greene, MD, PhD, MBA, Founder/CEO

 

R3 Stem Cell offers treatments that bring patients hope and options. Hope that surgery can be avoided, and tissue injury can be repaired with patients being able to get back to desired activities.

 

Founder and CEO David Greene, MD, PhD, MBA writes extensively on regenerative medicine and gives many seminars worldwide on a regular basis. With over forty Centers of Excellence globally, R3 is at the forefront of regenerative therapies.

 

R3’s Centers have successfully performed over 24,000 regenerative procedures to date. Call today for your free consultation (844) GET-STEM!

No portion of this Document may be reproduced without the Express Written Consent of R3 Stem Cell.

 

Disclaimer: This guide’s education does not constitute medical advice. The USA FDA considers stem cell therapy experimental. Any claims made in the Guide refer to procedures performed outside the USA.

Brand Ambassador Gallery

The USA stem cell leader offers procedures in

7 Countries including:

SUCCESS STORIES

*Outcomes will vary between individuals. No claims are being made with regenerative therapies. The FDA considers stem cell therapy experimental. See our THERAPY COMMITMENT HERE.
service_icon1

R3 STEM CELL MASTER CLASS

Learn everything you need to know about the ever expanding field of regenerative medicine in this 8 part series that includes over four hours of entertaining content!

service_icon2

R3 STEM CELL INTERNATIONAL

R3 Stem Cell International includes 45 clinics in 7 countries. These Centers of Excellence treat all types of conditions with safe, effective protocols by expert stem cell physicians.

service_icon3

FREE STEM CELL CONSULTATION

R3 Stem Cell offers a no cost consultation to see if you or a loved one is a candidate for regenerative cell therapies including cytokines, growth factors, exosomes, and stem cells.

service_icon4

PROVIDER PARTNERSHIP

The R3 Partnership Program offers providers an all-in-one regenerative practice program including marketing, consultations and booked procedures!

FREE WEBINAR: AVOID SURGERY WITH STEM CELL THERAPY

The links below provide national city resource information about stem cell therapy. To find a clinic near you click here

Disclaimer: The city links above provide general information on stem cell treatment. To find an R3 Stem Cell clinic near you click here

The USA stem cell leader offers procedures in

7 Countries including: