ALS Research

Amyotrophic Lateral Sclerosis Research: ALS (Lou Gehrig's disease) involves a cascade of cellular events leading to the death of neurons (called apotosis). Research is keying in on several clues, including the role of excess glutamate (a chemical needed for nerve signal transmission), faulty mitochondria (the part of the cell that provides energy), nerve cell transport problems (signals and growth factors transported by proteins), and the possible role of viruses.  Here is a sample of the work being done to treat ALS:

Replacing damaged nerve cells is a tantalizing and hugely promising idea, although it's not clear that new cells would resist the source of damage that caused ALS in the first place.  It does appear, however, that stem cells can take on the identity of motor neurons and form function-restoring contacts with muscle. Thhis could be done because the stem cells act as vehicles for neurotrophic factors, which are proteins found in the brain and muscle that support growth and survival of neurons. Until large-scale tests of four factors failed to improve ALS symptoms, this area was a major source of hope in the research community. Still, scientists are working with factors and new ways to deliver them.

For example, a clinical trial of Insulin-like Growth Factor-1 (IGF-1, or myotrophin) began in 2003 to see if the drug slows the progression of weakness in ALS. IGF-1 is essential for normal development of the nervous system and appears to protect motor neurons in animal models.  It is thought to block cell death pathways and promote muscle reinnervation and axonal growth and regeneration.

Umbilical cord blood/stem cells: Animal research suggests a clinical role for intravenous umbilical cord blood/stem cells, and indeed in some clinics these cells are already being provided to people with ALS. There have been no conclusive, long-term safety tests or beneficial results reported. Some people have told the ALS Association of their improved strength and function after the infusion. The door is open for further study.

Drug cocktails: recent mouse model studies of ALS showed dramatic benefits using a combination of drugs, including riluzole (the only drug now approved by the FDA to treat ALS), nimodipine (a calciaum channel blocker used in the treatment of acute stroke and migrane headache) and minocycline (an antibiotic that may block inflammation). The compounds given together appear to delay cell death, prevent nerve cell loss and reduce inflammation. Scientists are looking at other drugsthat help nerve cells survive, including protease inhibitors that prevent cell death and antiviral drugs. People with HIV and ALS showed improvement after taking an "AIDS cocktail" that targets viruses. This suggests that a viral mechanism might be related to some forms of ALS.

Drugs approved by the FDA for other reasons are being tested in ALS, including buspar (anxiety) and Celebrex (arthritis). Both have shown a positive effect on life expectancy for mice with ALS-like symptoms. The breast cancer drug tamoxifen (Nolvadex) has also shown benefit in tests with mice; clinical trials are ongoing.

A drug called AVP-923 (dextromethorphan/quinidine) is in clincal trials for the treatment of emotional lability (unwanted laughing and crying) in people with ALS. Trials are also continuing for creatine, a naturally occuring chemical involved in the energy metabolism of muscle, which may help people with ALS.