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Hopes for a new medicine to assist people afflicted with ALS were dashed by negative results from a large clinical trial reported Tuesday.

The experimental drug, tirasemtiv, failed to slow the decline in lung function any better than a placebo, said Cytokinetics (CYTK), the drug’s maker. The phase 3 clinical trial enrolled more than 700 people with ALS, the degenerative nerve disorder that robs people of the ability to move or breathe.

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  • It seems that progress on ALS drugs is elusive. Maybe targeting an aspect of the disease process or mechanism is the wrong approach. Maybe we need to go upstream, and target what triggers the disease process or mechanism. This likely varies by the individual. Just as we don’t have one but many breast cancers, we have not one but many combinations of gene variants, cumulative exposures, and epigenetic and phenotypic changes that lead to the devastating disease of ALS.

    There are a small number of physicians treating chronic diseases this way, in which each patient becomes an N of 1, and their particular disease triggers are examined, tested and treated to strengthen their immune and neurological systems, and arrest and partially reverse the disease process.

    At a medical conference in September, I heard a retired construction executive tell the story of his recovery from an 80% diagnosis of ALS. The executive wrote an inspiring short self-published book, An Unexpected Journey: Searching for a cause and finding hope in the battle against ALS. He wants his story to get out, to perhaps help others who otherwise have no hope. You can order it from his blog here:

    The point of anecdotes and patient stories is not to give false hope, but to raise new angles and avenues for exploration, for research and clinical validation. Perhaps ALS has multiple causes and some are reversible, or perhaps there are conditions that mimic ALS, and some patients can benefit by being screened and treated for those. Perhaps more physicians will go upstream and look for causes. Perhaps more pharma companies will begin to research, test and validate some of these approaches. Will they, if there is not a patent prospect at the end of the rainbow? If not them, who?

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