Key Information
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 able to confirm the diagnoses, nor the improvements described in the case series of 3 PALS taking PNG/HC; however, even if these treatment effects were real, these improvements were transient and quickly became unresponsive to treatment. Unless the ongoing placebo-controlled trial shows objective and sustained clinical improvements, we do not recommend that PALS take this expensive and risky combination of penicillin G and hydrocortisone.
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