Glucocorticoid Corticosteroids
In conclusion, corticosteroids are a class of medications with wide ranging clinical uses and well studied effects on the immune system. ALS progression is associated with changes in immune system function, with the early disease states associated with anti inflammatory immune markers and the advanced disease states associated withpro inflammatory immune markers. Although treatment with […]
Vitamin C
Vitamin C is safe and inexpensive. As an antioxidant, it has a plausible mechanism for influencing the course of neurodegenerative diseases. Two flawed preclinical studies by the same group showed benefits in a mouse model of familial ALS. There are two case reports in which it was associated with improvement. However, there are multiple possible […]
Azathioprine
As an immunosuppressant drug, AZA has a plausible mechanism for slowing the progression of ALS. However, there is no pre-clinical data to support its use and two clinical trials did not support efficacy. There are 2 published cases in whichALS reversals occurred on AZA, but it is not clear to us that the AZA actually […]
L-Carnitine
There are good theoretical mechanisms for carnitines, some preclinical evidence forLC and ALCAR, and a single clinical trial that suggested ALCAR could slow disease progression in PALS. All three carnitines appear to be well-tolerated, generally safe, and inexpensive. We believe that there is a need for future clinical trials of carnitines in PALS to further […]
Vitamin E
Vitamin E (a-tocopherol) is perhaps the most studied supplement in the history of ALS and was taken by one of the most famous ALS patients. Vitamin E has mechanistic potential in ALS as an antioxidant but appears in the SOD1 mutant mouse model to only have an effect on delaying disease onset. This bears out […]
Glutathione
As an ALS treatment, glutathione and cysteine-containing supplements that increase glutathione appear reasonably safe, and they have a plausible mechanism, positive preclinical data and 2 interesting case reports. Unfortunately small clinical trials of glutathione itself and of acetylcysteine showed no significant benefit. Given these negative clinical trials, we do not advise PALS to take glutathione […]
Perampanel
Perampanel is a drug currently used to treat seizures which has a mechanism of action that theoretically could be useful in treating patients with ALS. A single flawed study in a mouse model of ALS showed some benefits of perampanel, but data from humans with ALS is quite limited. Due to the lack of data […]
Penicillin G/Hydrocortisone
In our opinion, there is no convincing evidence that GABA overload plays a role in ALS progression. PNG/HC has other theoretical mechanisms by which it could slow ALS progression, but previous human trials involving steroids and a trial of a b lactam with more potent effects on glutamate did not help. We have not been […]