A drug used to combat malaria has shown great promise in the treatment of ALS (Lou Gehrig's disease). The drug, pyrimethamine (Daraprim), has been used to treat malaria since 1953, but has recently been given a new look for curbing the levels of a toxic protein created by the gene mutation prevalent in ALS (amyotrophic lateral sclerosis) sufferers.
The gene mutation creates a protein termed SOD1, and pyrimethamine reduced the level of SOD1 in test patients with inherited ALS, according to a new study conducted by investigators at Hospital for Special Surgery (HSS) in New York, N.Y. Their results were published in the online journal Annals of Neurology.
"Our multicenter, international study found that pyrimethamine reduced levels of SOD1 in the cerebrospinal fluid of patients with familial ALS, and the amount of lowering was related to the dose of pyrimethamine," said Dale J. Lange, neurologist and principal researcher at HSS. "There is currently no cure for this devastating disease, but our study represents the first time a drug lowered a protein known to be relevant to disease progression; as such, a slowing of disease progression would be expected."
Although specific reduction levels were not noted, Dr. Lange and his team were excited about the results.
"This is the first study in humans with ALS that targeted and achieved a significant reduction of a disease-relevant biomarker in the cerebrospinal fluid. We found that pyrimethamine was safe and well tolerated in patients with ALS caused by different SOD1 mutations," Lange noted. "Although not proven by this study, a slowing of disease progression was observed. A larger study is needed, and is being planned, to determine if pyrimethamine does indeed influence the disease course in ALS patients."
Since patients had varying levels of disease severity, some outcomes were affected differently, researchers noted. And some with the worst symptoms were more likely to drop out, according to the investigators.
The study was supported by a grant from the Muscular Dystrophy Association of America. A total of 32 ALS patients participated, and each underwent lumbar punctures, blood studies, and clinical assessments of strength, motor function, quality of life, and potential adverse effects. Out of the patients enrolled, 24 completed six visits over 18 weeks, and 21 completed all study visits.
ALS progressively attacks nerve cells in the brain and in the spinal cord. Over time patients lose the ability to initiate and control muscle movement, and the ability to speak, walk, breathe and swallow become affected, culminating in paralysis. ALS is often fatal within five years. In addition to Lou Gehrig, acclaimed theoretical physicist and cosmologist Stephen Hawking is afflicted with ALS.
According to the ALS Association website, there are about 6,000 new cases each year, with about 20,000 individuals suffering each year. The average survival rate is three years, prompting active research worldwide to find a cure.
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