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news.The ALS Association, which is dedicated to uncovering the cause of amyotrophic lateral sclerosis, has agreed to fund a study into the disease using monkeys to test the ability of an Isis Pharmaceuticals drug to lower production of a mutant protein linked to some inherited cases of the syndrome.
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Based on findings in rats, researchers believe this drug has the potential to be an effective treatment for inherited amyotrophic lateral sclerosis (ALS, also called Lou Gehrig’s disease).
Researchers hope this antisense drug will also prove useful in other ALS patients as well as for targeting new genes that are found linked to the disease.
In some patients with inherited ALS, the protein copper-zinc superoxide dismutase (SOD1) is changed, or mutated. While the exact mechanism of toxicity is not yet documented, death of motor neurons is undoubtedly the result. Researchers have shown that antisense treatment aimed at stopping production of mutant SOD1 in rats prolongs survival.
Time of treatment for the rats was near onset of symptoms, reflecting the scenario for actual patients who often have definite and even advanced signs of motor neuron loss by the time of ALS diagnosis.
For the new study investigators will contract with an outside facility to study 20 rhesus monkeys who will receive the antisense candidate delivered into the fluid surrounding the spinal cord (cerebrospinal fluid or CSF). The study will use the same equipment appropriate for human use. Delivery directly into the CSF is also the approach the investigators envision for human trials.