FINDING THE LIGHT OF HOPE
IN A DARK PLACE
Families living with muscular dystrophy and rare diseases are often left feeling hopeless. Through 14 years of experience we have found a solution we can all believe in.
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.
Types of Stem Cells
MESENCHYMAL
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.
HOW DOES ADULT STEM CELL THERAPY WORK?
Ryan’s disease, Duchenne, 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.
For Ryan, these newly injected mesenchymal stem cells hone in on areas of inflammation and identify the cells in his body which lack the Dystrophin protein. At this point the new mesenchymal stem cells repair the cells negatively impacted by his disease and allow them to produce Dystrophin which allows his body to fight back against the disease for roughly 3-4 months.
After 3 months, the newly injected stem cells are essentially “used up” which means at this point the improvements plateau for a couple weeks and then eventually are all overtaken by the disease once again.
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. Because dystrophin is necessary to protect muscle cells, without it, muscle cells are vulnerable to atrophy as there is now a higher concentration of an enzyme that destroys the muscle cells.
HOw Stem cells help DMD
Ryan’s disease, Duchenne, 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.
For Ryan, these newly injected mesenchymal stem cells hone in on areas of inflammation and identify the cells in his body which lack the Dystrophin protein. At this point the new mesenchymal stem cells repair the cells negatively impacted by his disease and allow them to produce Dystrophin which allows his body to fight back against the disease for roughly 3-4 months.
After 3 months, the newly injected stem cells are essentially “used up” which means at this point the improvements plateau for a couple weeks and then eventually are all overtaken by the disease once again.
The Science
Dr. Neil Riordan explains an in-depth analysis of his research and provides multiple perspectives to help answer any questions and concerns regarding this new form of regenerative medicine.
Join Us
Your support has the potential to have a huge impact on the DMD community and those with rare diseases. Join our cause in helping to make treatment more accessible.