New Drug with Potential to Treat Amyotrophic Lateral Sclerosis
Based on findings in rats, researchers believe an antisense drug has potential to be an effective treatment for some familial types of ALS.
"The potential of this therapeutic approach is very exciting for ALS patients carrying the SOD1 mutation," said Lucie Bruijn, Ph.D., ALSA science director and vice president.
In some patients with inherited ALS, the protein copper-zinc superoxide dismutase (SOD1) is mutated. The exact mechanism of toxicity is not yet documented, the result of toxicity by all indications is the degeneration of motor neurons.
Timothy Miller, M.D., Ph.D., and Don Cleveland, Ph.D., of the Ludwig Institute, University of California, San Diego, and Richard Smith, M.D., of the Center for Neurologic Study have shown that antisense treatment has prolonged life in rats that are affected by these familial types of ALS.
The rats began treatment at the onset of ALS related symptoms, similar to the time that
people are first diagnosed with ALS, when their motor neurons have already degenerated.
The doctors from the Center for Neurologic Study will contract with an outside facility to study 20 monkeys over 90 days to test the antisense drug, currently named ISIS 333611.
The study is required by FDA regulations. The FDA enforces monkey trials before any human drug trials will be permitted.
The monkeys will be injected with ISIS 333611 directly into the fluid surrounding their spinal cord.
The study will use the same equipment appropriate for human use and will test dose tolerance and safety.
The spinal cord delivery was chosen to reflect the same procedure expected to be used on patients with ALS, should a human drug trial occur in the future.
Contact:
Gary Wosk,
818-587-2241
gwosk (at) alsa-national.org
"The potential of this therapeutic approach is very exciting for ALS patients carrying the SOD1 mutation," said Lucie Bruijn, Ph.D., ALSA science director and vice president.
In some patients with inherited ALS, the protein copper-zinc superoxide dismutase (SOD1) is mutated. The exact mechanism of toxicity is not yet documented, the result of toxicity by all indications is the degeneration of motor neurons.
Timothy Miller, M.D., Ph.D., and Don Cleveland, Ph.D., of the Ludwig Institute, University of California, San Diego, and Richard Smith, M.D., of the Center for Neurologic Study have shown that antisense treatment has prolonged life in rats that are affected by these familial types of ALS.
The rats began treatment at the onset of ALS related symptoms, similar to the time that
people are first diagnosed with ALS, when their motor neurons have already degenerated.
The doctors from the Center for Neurologic Study will contract with an outside facility to study 20 monkeys over 90 days to test the antisense drug, currently named ISIS 333611.
The study is required by FDA regulations. The FDA enforces monkey trials before any human drug trials will be permitted.
The monkeys will be injected with ISIS 333611 directly into the fluid surrounding their spinal cord.
The study will use the same equipment appropriate for human use and will test dose tolerance and safety.
The spinal cord delivery was chosen to reflect the same procedure expected to be used on patients with ALS, should a human drug trial occur in the future.
Contact:
Gary Wosk,
818-587-2241
gwosk (at) alsa-national.org
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