Clinical trial suggests new drug candidate for spinocerebellar ataxia may offer certain therapeutic benefits (L-arginine)

Medical Xpress / Niigata University / eClinicalMedicine ^ | Dec. 4, 2024 | Tomohiko Ishihara et al

[ConservativeMind]

A team has conducted a trial on the efficacy and safety of L-arginine in treating spinocerebellar ataxia type 6 (SCA6).

Spinocerebellar ataxia (SCA) is a neurodegenerative disorder affecting the cerebellum, a part of the brain responsible for coordinating movement.

Many hereditary SCAs are classified as polyglutamine diseases, a group of disorders caused by proteins with abnormal long polyglutamine (polyQ) tracts.

Professor Osamu Onodera and Professor Yoshitaka Nagai identified L-arginine, an amino acid, as a promising compound that inhibits polyQ protein aggregation. In animal models of polyglutamine disease, L-arginine has shown therapeutic potential. Since L-arginine is already an approved medication, our team decided to explore its safety and efficacy as a potential treatment for polyglutamine diseases through a clinical trial.

The study (AJA030-002, jRCT2031200135) was designed as a multicenter, placebo-controlled, double-blind, randomized Phase II trial to evaluate L-arginine's efficacy and safety in SCA6, a subtype of SCA with relatively high prevalence and uniform symptom presentation in Japan.

Forty patients with SCA6 were recruited and randomly assigned to receive either L-arginine or a placebo.

For 48 weeks, 20 patients received L-arginine while the other 20 received placebo. This Phase II trial was exploratory, with a primary aim to observe safety and preliminary efficacy in a relatively small sample. The trial assessed treatment efficacy using the Scale for the Assessment and Rating of Ataxia (SARA), a standardized tool for evaluating ataxia severity, especially in cerebellar diseases.

After 48 weeks, the L-arginine group showed an improvement of 0.96 ± 0.55 points in mean SARA scores, indicating a mild reduction in ataxia symptoms. In contrast, the placebo group experienced a mean worsening of 0.56 ± 0.55 points in SARA scores. This translates to an approximate 1.5-point treatment effect over one year.

The study suggests that L-arginine may offer certain therapeutic benefits for patients with SCA6.

[ConservativeMind]

L-arginine is an available supplement and showed indications of helping with polyglutamine diseases like spinocerebellar ataxia.

[ConservativeMind]

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[Hazwaste]

Thank you for posting this! SCA17 runs in my family. While this is specific to SCA6, it’s worth a try.

[sauropod]

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