UNDERSTANDING DYSFERLINOPATHY
Important Points Regarding Stem Cell Therapy
UNDERSTANDING DYSFERLINOPATHY
Important Points Regarding Stem Cell Therapy
What are stem cells and what is stem cell therapy?
Stem cells are remarkable because they are capable of self-renewal and because they can multiply and divide to develop into many different cell types. There are several types of human stem cells: embryonic and adult stem cells. Embryonic stem cells are only found in developing embryos and these cells can develop into any cell type in the human body. Adult stem cells are more limited but can still become a variety of different cell types. For example, stem cells in the bone marrow give rise to red blood cells that carry oxygen throughout your body and white blood cells that fight infection.
Current stem cell technology is not advanced enough to provide a solid foundation for the development of a therapy for dysferlinopathy
Stem cell therapy involves introducing stem cell populations into patients to replace damaged tissues and researchers believe that the use of these cells may someday be able to treat diseases like cancer, Parkinson’s disease, and muscle diseases. Most of these treatments are still going to take many years to develop, however, because there are still a number of big hurdles to overcome.
One of the biggest challenges in using stem cells therapeutically is making sure that the cells which are injected become the type of cells the researcher wants them to be. This can be very difficult because stem cells need to be told what to do by the cells around them. Until we know what signals stem cells need from the environment to develop into the type of cell desired and how to make sure the stem cells get these signals, cells used in stem cell therapies likely will not have any benefits and could possibly cause harm.
Unsafe stem cell therapies
Because stem cell therapy has the potential to cure many debilitating diseases, there is much excitement surrounding the development of these procedures. Unfortunately, this excitement has also led to the exploitation of patients’ hopes.
Many stem cell therapies are currently being directly advertised to the public that have not yet been proven to treat any disease.
Many stem cell therapies are currently being directly advertised to the public that have not yet been proven to treat any disease. These treatments are very dangerous because they often do not meet the minimum ethical, scientific, and medical standards that clinical trials and approved medical treatments require. Treatments that have not yet been tested and proven to be beneficial are unlikely to help patients and may actually endanger patient lives.
Patients are advised to be highly skeptical of any therapies that base their claims on patient testimonials claim that there is no risk, claim that they can treat more than one disease with the same cells, or do not clearly document where the cells are from and how the treatment will be performed. Most importantly, patients should be skeptical of any treatment that costs a large amount of money because unproven treatments like clinical trials are generally free for patients.
No stem cell therapies have been developed yet for dysferlinopathy (LGMD2B/LGMDR2 and Miyoshi Myopathy 1). We at the Jain Foundation are dedicated to finding a cure for dysferlinopathy and are currently pursing all avenues that we think will lead to a treatment in the future. Unfortunately, current stem cell technology is not advanced enough to provide a solid foundation for the development of a therapy for dysferlinopathy. We have therefore decided to concentrate our efforts on treatments that can be advanced into the clinic more rapidly than any stem cell therapy can.
If you would like to learn more about stem cell therapies, please visit the websites below: