Drug found to lower levels of biomarker linked to ALS

A decades-old medication used to treat malaria is capable of targeting genetic mutation in ALS patients.

By Amy Wallace

June 12 (UPI) -- Researchers found pyrimethamine, a drug to treat malaria, safely and effectively lowers levels of a biomarker linked to amyotrophic lateral sclerosis, or ALS.

ALS or Lou Gehrig's Disease, progressively attacks nerve cells in the brain and spinal cord causing patients to lose the ability to initiate and control muscle movement, speech and patients eventually become paralyzed. ALS patients are often on ventilators and the disease is fatal within three to five years of diagnosis.


Some people with an inherited form of ALS have a mutation on the SOD1 gene. Researchers tested to see if pyrimethamine, a malaria drug, could safely and effectively lower levels of the toxic protein produced by the gene mutation.

"Our multi-center 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," Dr. Dale J. Lange, principal investigator and neurologist-in-chief at the Hospital for Special Surgery, said in a press release. "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."


Researchers enrolled 32 patients with various SOD1 genetic mutations linked to ALS who underwent three lumbar punctures, blood studies and a clinical assessment of strength, motor function, quality of life and possible negative effects.

Over the 18-week study period, 24 participants completed six visits and 21 completed all study visits.

"To our knowledge, 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," Lange said. "We found that pyrimethamine was safe and well tolerated in patients with ALS caused by different SOD1 mutations."

"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."

The study was published June 9 in the Annals of Neurology.

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