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Immunotherapy Promising for Slowing Progression of ALS

MONDAY, June 4, 2018 -- A new immunotherapy appears to be well tolerated and possibly of benefit in patients with amyotrophic lateral sclerosis (ALS), according to a phase 1, first-in-human study published online May 18 in Neurology: Neuroimmunology & Neuroinflammation.

Jason R. Thonhoff, M.D., Ph.D., from the Houston Methodist Neurological Institute, and colleagues assessed the safety and tolerability of autologous infusions of expanded regulatory T lymphocytes (Tregs) in three patients with ALS but no family history. The patients underwent leukapheresis, and Tregs were isolated and expanded ex vivo. Tregs were administered at early stages of the disease in four doses over two months and at later stages in four doses over four months. Concomitant interleukin-2 was also administered throughout the study period.

The researchers found that infusions of Tregs were safe and well-tolerated in all patients. After each infusion, Treg numbers and suppressive function increased. During both early and later stages of disease, the infusions slowed progression rates. Increased Treg suppressive function was correlated with slowing of disease progression per the Appel ALS scale for each patient. During Treg infusions, measures of maximal inspiratory pressure also stabilized, in two patients in particular.

"These results demonstrate the safety and potential benefit of expanded autologous Treg infusions, warranting further clinical trials in patients with ALS," the authors write.

Several authors disclosed financial ties to the pharmaceutical industry.

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Posted: June 2018