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.
ALS TDI scientists turned their attention to relcovaptan as a potential therapy for ALS due to its reported ability to turn down the production of aquaporin4. Aquaporin4, which controls the salt/water balance in the brain, appears to be upregulated in people with the disease. These findings however could not be reproduced and therefore, the drug is no longer being pursued.
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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