RESEARCH OTHER THAN GENE THERAPY
MDA is continuing research in DMD that isn't gene therapy.
As discussed in Chapter 4, the steroid drug prednisone has been found effective in preserving and even in some cases increasing muscle strength, but with major side effects.
Research into the mechanism by which prednisone and similar drugs increase muscle strength is a priority. If the mechanism can be worked out, it's possible that better drugs with fewer side effects could be developed that would achieve the same ends.
MDA researchers are also on the trail of what happens to calcium in the muscle fibers of boys with DMD. If there is in fact some kind of signal that goes wrong in Duchenne, so that too many calcium channels stay open, or a few stay open too long, or calcium is released improperly from inside the cell, it's possible that problem could be corrected. (Researchers are also looking into the possibility that prednisone works by limiting calcium-related damage to muscle fibers.)
Certain enzymes, such as nitric oxide and calpain, may play a role in muscle degeneration, and MDA-funded scientists are working on understanding this kind of mechanism with an aim to stopping it.
The process of inflammation, a reaction that often occurs in the body when tissue is degenerating for any reason, could be contributing to muscle degeneration in DMD. MDA scientists are also working on this problem to see if there is harmful inflammation and if it can be stopped.
The anabolic steroid oxandrolone (Oxandrin) is being tested in MDA-supported clinical trials. This drug or others like it may play a role in preserving muscle strength in Duchenne. Understanding the mechanism of these drugs may also lead to other ideas for manipulation of the muscle degeneration-regeneration cycle.
Muscle differentiation factors are proteins that influence muscle development. These are being studied to further understand muscle generation and regeneration.
Similarly, there are proteins that act as muscle cell death regulators. These are also being studied. Understanding how such proteins operate could lead to manipulating them for therapeutic purposes.
Help MDA Help You
MDA IS HERE TO HELP
There are many ways you can help in the fight against neuromuscular disease. Through their donations and volunteer activities, concerned individuals, families, companies and organizations help make it possible for MDA to continue its worldwide research effort and nationwide programs to aid those with neuromuscular disorders and their families.
MDA accepts tax-deductible donations and pledges of financial help year-round, particularly at the time of the Jerry Lewis MDA Labor Day Telethon. In addition, families of those with muscular dystrophy are among MDA's local and national volunteer leaders, and are active in assisting with fund-raising and educational events, which take place throughout the year.
Many local, regional and national businesses and organizations help MDA through sponsorship programs. MDA also has an active planned giving program, offering a number of ways for people to benefit the Association through their retirement or estate plans.
Call the district director at your local MDA office for information about assisting MDA as a sponsor or volunteer. |
Next... Chapter 6: Your Child's Emotional Needs  |
