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ALS, amyotrophic lateral sclerosis or Lou Gehrig’s disease, is a disease of the motor neurons — the nerves that control muscles. As the motor neurons are lost, the muscles that they control become weaker, eventually resulting in paralysis. People with ALS usually do not survive more than five years past diagnosis, although about 10 percent live much longer.
Research Pipeline
In the past 50 years, the field of research into treatments and cures for ALS has been enormous. A total of 159 clinical trials have been performed, but the result has been only one approved treatment, Riluzole, that affects the progression of the disease. This drug only increases survival by a few months. The rate of success reflects the fact that researchers don't understand the true cause of the disease, or why it progresses.
Basic research and genomics technologies are now beginning to offer some clues as to the mechanism of the disease. In January 2007, through an $18 million grant, MDA was instrumental in helping to set up the ALS Therapy Development Institute, a non-profit research organization focused on ALS research. ALS TDI and other MDA-sponsored researchers have made huge steps forward in understanding the nature of the disease, and some of these will lead to potential new therapies.
Perhaps even more importantly, in 2008, ALS TDI published a seminal paper explaining why so many compounds that had been shown to affect the progression of ALS in mouse models failed to work in clinical trials. This work will allow drug development in ALS to be performed in a much more efficient manner.
Research Strategies
In 2008, 62 companies have a product in development for ALS. Strategies for treatments include:
- Stem cell treatments designed to replace lost nerves, or maintain existing nerve cells
- Growth factors to help maintain muscles and/or nerves
- Neuroprotective compounds to preserve nerves
- Therapies designed to modulate the immune response, preventing breakdown of nerves and muscles
- Glutamate inhibitors, which are drugs related to riluzole.
- Anti-apoptotic compounds that prevent cell death
- Strategies designed to remove the mutant protein that causes the inherited form of ALS
- Various strategies based on specific hypotheses as to the cause of the disease, such as production of proteins that re-fold misfolded proteins
Many of the drugs in development for ALS may also have uses in other diseases. For example, the growth factor treatment that MDA is funding for a Phase II trial in myotonic dystrophy is also being tested in ALS patients. Other drugs in development are being tested for use in Friedreich’s ataxia, Charcot-Marie Tooth disease, multiple sclerosis, Parkinson’s disease etc, in addition to ALS.
MDA’s Translational Research Program is supporting Washington University to plan and run a Phase I study for a treatment designed to remove or "block" the mutated protein which causes the most common inherited form of ALS. MDA has also provided $200,000 to California Stem Cell, Inc., to develop pilot data in support of a stem cell trial for ALS.
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