The Science

What are Adult Stem Cells?

Stem cells are very powerful and can help people with or without Duchenne’s. We all have them. When you’re sick or hurt, these cells identify and repair the damaged tissue. In a lab setting, these cells have the potential to multiply indefinitely.

In the body, adult stem cells may lose their capacity and their speed to multiply as we age. In certain people with degenerative conditions, such as Muscular Dystrophy, stem cells may not be supplied quickly enough to fully repair damaged tissue.

How Does Adult Stem Cell Therapy Work?

Stem cells are unique in their ability to differentiate into various cell types and promote tissue repair and regeneration. In the case of MSCs, these cells can be administered through minimally invasive methods, such as intravenous (IV) infusion. Once in the body, MSCs can migrate to sites of injury or inflammation, where they can exert their therapeutic effects.

These effects include reducing inflammation, promoting tissue repair, and modulating the immune system. While the exact mechanisms of action are still being studied, the regenerative properties of MSCs hold immense promise for treating a wide range of conditions.

Why is Stem Cell Therapy Needed?

During youth, the multiplication of stem cells is highly efficient. As we age, this ability to multiply naturally decreases. While in the womb, one stem cell multiplies into one billion new cells every 30 days. By the time you reach your twenties, a single stem cell will produce 32,000 cells in 30 days; at 65 years a single stem cell will only produce 200 new cells.

For those with debilitating injuries or diseases, the naturally decreasing stem cells cannot multiply fast enough to keep up with the physical deterioration. Adult stem cell therapies can help people overcome these conditions by replenishing their dying stem cells and allowing their body to fight back.

Types of Stem Cells

MESENCHYMAL (MSCs)

Proven to be the most effective type of adult stem cell & are found on every single blood vessel in the human body.

While there are many types of stem cells, Coming Together for a Cure is only focused on helping advance research and access to Mesenchymal stem cells. These cells are key for keeping our bodies alive; each person has an innumerable amount throughout their body. Some tissues are particularly rich in MSCs, such as bone marrow and fat, and in tissues that are discarded after a healthy birth, such as the umbilical cord, placenta, and amniotic tissue.

In short, we understand that MSCs control inflammation, modulate the immune system, stimulate regression, and decrease fibrosis and scarring. We believe the advancements of research and the proven effectiveness of MSCs will help to find a cure with many diseases.

Hematopoietic

Found in the blood vessels & have the ability to become blood cell or platelets.

EMBRYONIC

A special type of cell that allow a baby to grow organs when in the womb.

INDUCED PLURIPOTENT

Cells grown in a lab and programmed for a specific task.

What diseases and conditions have stem cells shown to positively affect?

These are a few of the most common, many other conditions and diseases are continuing to undergo clinical trials and new discoveries are being made on a regular basis.

  • Muscular Dystrophy

  • Multiple Sclerosis

  • Rheumatoid Arthritis

  • Cerebral Palsy

  • Autism

  • Heart Failure

  • Osteoarthritis

  • Spinal Cord Injury

  • Autoimmune Diseases

Demystifying Stem Cell Therapy

Stem cell therapy has captured the attention of the medical community and the public alike, offering hope for individuals battling various rare diseases and conditions. However, misconceptions surrounding this groundbreaking field have led to confusion and skepticism. We aim to demystify stem cell therapy, clarify common misunderstandings, and shed light on its potential for medicine’s future.

  • One of the most prevalent misconceptions about stem cells is that they are solely derived from embryos. While embryonic stem cells have been a topic of ethical debate, it is essential to note that they are not the only source of stem cells. Adult stem cells, found in various tissues throughout the body, and induced pluripotent stem cells (iPSCs), reprogrammed from adult cells, have shown great promise in regenerative medicine. CTFAC only advocates for the research and development of mesenchymal stem cells (MSCs), which are not derived from embryonic stem cells and pose no ethical concerns.

  • Stem cell therapy has the potential to revolutionize the treatment of rare diseases and improve the quality of life for countless individuals. While the field is continuously advancing, it is crucial to distinguish between FDA-approved stem cell treatments and therapies that have yet to receive FDA approval. Umbilical cord derived mesenchymal stem cell therapy is currently not FDA-approved. This lack of FDA approval limits the reach and accessibility of this potentially life-saving treatment. We firmly believe in the need to begin FDA sanctioned clinical trials so the safety and efficacy of MSCs can be evaluated and verified. To make this treatment more widely available to those who need it most, we are actively advocating for clinical trials that will pave the way for FDA approval. By obtaining FDA approval, we can ensure that MSC stem cell therapy reaches a broader population and provides hope to individuals and families battling rare diseases. Our commitment to bringing this transformative treatment to the forefront of medical care drives us to tirelessly work towards making it accessible to all who can benefit from its potential.

  • Researchers worldwide are actively investigating the potential applications of stem cell therapy for various diseases and conditions. Promising results have been observed in preclinical studies and early-stage clinical trials for neurodegenerative diseases, autoimmune disorders, cardiovascular diseases, and orthopedic conditions. However, it is essential to acknowledge that further research and well-designed clinical trials are necessary to understand stem cell treatments’ complete safety and efficacy. Coming Together for a Cure is committed to supporting and advancing this research to bring hope and healing to those affected by rare diseases.

  • As we look to the future, the potential of stem cell therapy is vast and exciting. With continued research and technological advancements, we envision a world where personalized stem cell treatments become a reality, offering targeted and effective solutions for various medical conditions. However, we recognize challenges to overcome, such as regulatory hurdles and the need for long-term safety and efficacy data. Coming Together for a Cure is dedicated to collaborating with researchers, healthcare professionals, and regulatory bodies to responsibly advance stem cell therapies and ensure their accessibility to those in need.

  • Often times, we notice that patients experience similar outcomes, although it is important to note that any specific outcomes are not guaranteed and each patient will have a different experience, as there are a variety of contributing factors that are unique to all patients, i.e.; level of disease progression, age, weight, daily lifestyle activities, etc.

Stem Cell FAQs

  • No patients who have received this therapy have experienced negative effects. Dr. Riordan’s team at the stem cell institute has administered these stem cells thousands of times and there has never been a single instance of rejection. To prove this point, MSC stem cells are approved to treat graft vs host disease in Canada and New Zealand.

  • Adult stem cells should not be confused with embryonic stem cells. Both the Catholic and Southern Baptist churches support the use of adult stem cells as a means of medical therapy.

  • In May of 2017 “Charlie’s Law” was passed into law in Texas through HB-810. For the first  time ever in the United States it is now legal for patients to receive medical therapy with expanded adult stem cells such as MSCs.

  • For allogeneic stem cell therapy, eventually the number of newly introduced active stem cells would decline. This decline begins to take place after three months or so as the cells become recognizable to the immune system and are cleared by the body. This specific time frame and type of decline does not apply to all types of stem cells.

  • The most common method for providing stem cell therapy is through intravenous infusions. No anesthesia is required. Protocols vary by the clinic or clinical trial in question. On average, patients receive three to four infusions total over the course of four to five days. IV infusions typically take no longer than 30 minutes.

  • All donated cords are the by-products of normal healthy births. Donors are required to sign a consent form, complete a detailed questionnaire and give a blood sample. The mother’s blood and cord blood undergo extensive testing for HIV, STD’s, hepatitis, etc. Each cord is carefully screened for sterility and infectious disease under the international blood bank standards.

  • Aside from initial pain at the injection site, patients will not experience any discomfort. In rare cases patients experience minor fever, headache, or nausea. Although these side effects usually subside within 24 hours. No long-term negative side effects have been reported.

  • Currently, no insurance companies cover adult stem cell therapy.

  • Umbilical cord-derived MSCs proliferate/differentiate more efficiently and “older” cells, such as those found in the fat and therefore, they are considered to be more “potent”.

  • The current regulatory environment in the United States has strict restrictions on the isolation and expansion of cells. Within the United States, pure MSC products are not able to be given to patients as no pure MSC cell product has been approved by the FDA at this time. Currently, the only stem cell products that are FDA-approved for use in the United States consist of blood-forming stem cells that are derived from umbilical cord blood. These products are approved for use in patients with disorders that affect the production of blood but they are not approved for other uses. 

    https://www.fda.gov/vaccines-blood-biologics/consumers-biologics/important-patient-and-consumer-information-about-regenerative-medicine-therapies

How do Stem Cells Help DMD?

Duchenne, the disease Ryan had, is caused by the absence of a functional protein on the mutated gene, called Dystrophin.

Dystrophin is necessary to break down and repair muscle tissue – without it the cells are unable to hold their shape causing fibrous tissue to form in the muscle and increased inflammation throughout the body and immune system.

With Ryan’s therapy, newly injected mesenchymal stem cells honed in on areas of inflammation and identified the cells in his body which lacked the Dystrophin protein. The new mesenchymal stem cells would then repair the cells negatively impacted by his disease and allow them to produce Dystrophin, allowing his body to fight back against the disease for roughly 3-4 months.

After 3 months, the newly injected stem cells would essentially become “used up” causing his improvements to plateau for a couple weeks and then eventually be overtaken by the disease once again.