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ALS platform trial to put neuroinflammation hypothesis to the test

The idea that neuroinflammation drives ALS will get its most rigorous test to date with the Healey ALS platform trial.

The largest test of the idea that ALS is driven by neuroinflammation will come from the first collaborative platform trial in the disease, which could blow open the range of pathways and targets in play for a disease that has remained stubbornly intractable.

The Healey ALS trial was launched on Sept. 18 by Massachusetts General Hospital and will test five different agents. The trial is exceptional not only for how quickly companies signed on, but for the bolus of data it could provide within the next 18 months. The trial is set to treat the first patients in early 2020 and the primary endpoint in the trial is assessed at six months, putting the first possible data read out near YE20 or early 2021 (see “Lessons from Healey ALS”).

Three of the five candidates will test the hypothesis with programs aiming to tamp down inflammatory pathways -- from the complement cascade to pro-inflammatory cytokine release to immune-driven oxidative stress.

And the candidates in the trial aren't just the leftovers of discontinued development programs from unrelated indications. Rather, most are backed by at least some evidence to support a role in reducing neurodegeneration. At least one of the programs has shown clinical benefit in ALS patients.

While inflammation-targeted agents have been tested in the past and failed, companies with these new therapies are banking on a combination

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