“These results support the importance of the immune system, and more specifically T-reg populations, in modulating the clinical course of a patient’s disease,” Appel said. “Thus, enhancing the number of T-regs and their anti-inflammatory functions in ALS patients could have important therapeutic benefits in slowing the rate of disease progression and stabilizing ALS patients for longer periods of time than currently available therapies.”
Some 27 percent of patients receiving the higher dose had no progression of the disease, about 2.5 times as many as were seen in a placebo group, a post-hoc analysis showed.
Results of the first stage showed that receiving dexpramipexole appeared to slow the progression of symptoms measured both by the ALS Functional Rating Scale and by pulmonary capacity. The second stage had similar results, with slower disease progression and a reduced risk of death in participants receiving the higher dosage.