Rapamycin may boost autophagy, clearing protein aggregates from motor nerves which buildup during the course of the disease. Originally developed as an immunosuppressant, rapamycin is routinely administered to kidney transplant patients to prevent organ rejection.
Aquaporin4, which controls the salt/water balance in the brain, appears to be upregulated in people with ALS. Relcovaptan, a drug reported to reduce Aquaporin 4, may therefore be helpful to people with the disease.
Retigabine may protect motor neurons in people with ALS by reducing hyperexcitability – a potential early step in the disease. A KCNQ2/3 potassium channel opener, the drug increases the levels of potassium (M-current) in nerve cells– a key regulator of the excitability in the nervous system. Retigabine is currently used to control seizures in people with epilepsy who do not respond to other medications.
Riluzole may protect motor nerves from further deterioration by reducing glutamate levels in the brain and spinal cord by blocking its release from nerve terminals.
Some scientists suspect that a subset of ALS cases may be due to retroviral infection. The antiviral ritonavir, originally developed to fight the retrovirus HIV, was therefore evaluated as a potential treatment for the disease. In a placebo-controlled trial of 24 people with ALS, ritonavir appeared to be ineffective in slowing the progression of the disease.
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