Thalidomide may slow Lou Gehrig’s disease

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Treatment with thalidomide or its analog lenalidomide prolongs life in mice with amyotrophic lateral sclerosis (ALS), scientists report.

Lead investigator Dr. Mahmoud Kiaei told Reuters Health that small clinical trials are already underway in the US and in Germany “and we hope to set up a larger clinical trial in New York to examine the efficacy of these drugs in ALS patients.”

ALS, also known as Lou Gehrig’s disease, is an incurable progressive degenerative neurologic disorder in which nerve cells in the brain and spinal cord die, leading to muscle wasting and total paralysis.

In the Journal of Neuroscience, Dr. Kiaei of Weill Cornell Medical Center, New York and colleagues note that there is increased activity of inflammatory mediators including TNF-alpha in the spinal cords of patients with ALS and in ALS mice.

The researchers found that early treatment of such mice with thalidomide or lenalidomide, which attenuate TNF-alpha and like proteins, significantly increased mean survival from 130 days to more than 150 days.

Use of either agent also attenuated weight loss, enhanced motor performance and decreased motor neuron cell death.

The study of these immune-modulating drugs “showed for the first time they were effective in ALS transgenic mice,” Kiaei said. “Due to a high percentage of survival extension, it does provide a high level of merit for these drugs to be taken into clinical trial.”

SOURCE: Journal of Neuroscience, March 2006

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