Therapeutics

Drug discovery:

• Through a collaboration with the NIH, we tested 1040 FDA drugs and nutritionals against an ALS micro-assay and found more than a dozen that had an effect.
• Jean-Pierre Julien discovered a three-drug cocktail, which significantly slowed the effects of ALS on SOD1 mice.

We’re taking these drugs to animal and human trials rapidly in a unprecedented collaboration with ALSA and Project ALS.

Gene therapy:

• In collaboration with Project ALS, Dr. Rothstein has personally dedicated his time to furthering work in a novel gene therapy, which is designed to extend the life of specific motor neurons that affect breathing.
  This gene therapy has already shown significant benefit to SOD1 mouse.

Basic Research

Basic research continues to be a priority for the Packard Center as it is the only way to gather new facts about how ALS works in the human body. As we learn more, we can be even more effective about designing specific therapies to the many ways ALS damages the body. This year’s discoveries were important, not just to us, but to other research bodies around the world. Among the most important were:

• ALS damage begins in the glial cells that surround motor neurons; if we can find a way to keep glial cells healthy, we may be able to protect motor neurons.
• Mitochondria are damaged in ALS patients; if we can find a way to slow down their damage, we may be able to slow down ALS.
• The immune system may play a significant role in ALS and be a possible target for therapy.

Genetics Research

These two important projects help us understand better the role of genetics in ALS:

• Philip Wong has created the first animal model using ALS2 gene, one associated with yet another form of inherited ALS. This animal is of great importance to scientists in helping to understand the similarities and differences of the ALS disease pathways from ALS2 and SOD1.
• Jonathan Glass is working with deCode Genetics and Center investigators at various universities to isolate genes associated with sporadic ALS. This intense nationwide collaboration to collect DNA aims to find markers for the disease, in a first step to isolate new genes.

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