FDA Rejects BLA for NurOwn, Simvastatin Futile as DMT for Parkinson, Music Potentially Improves Insomnia Symptoms

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Months after the company claimed it was planning on submitting a biologics license application (BLA) for its NurOwn technology platform for the treatment of patients with ALS, BrainStorm Cell Therapeutics announced that it received a refusal to file letter from the FDA. The FDA indicated that the company can request a Type A meeting to discuss the content of the refusal to file letter. In March 2021, following a review of the pivotal phase 3 trial of NurOwn, the agency concluded that the current level of data did not cross the threshold of substantial evidence to support a BLA. Original results showed that NurOwn did not meet its primary end point of statistical significance, as 33% and 28% of those on MSC-NTF and placebo, respectively, showed a change in disease progression of at least 1.25 points on ALS Functional Rating Scale after 28 weeks of treatment.More than a year later, in August 2022, BrainStorm announced new clinical analyses that strengthened the findings of NurOwn. The erratum included several changes, which prompted the company to submit the BLA, a decision that came more than a year after the FDA recommended against it.

Findings from a randomized, double-blind, placebo-controlled study showed that simvastatin, a statin used to treat high cholesterol and triglyceride levels, was futile as a disease-modifying therapy for patients with moderate forms of Parkinson disease (PD).The prespecified primary outcome, 24-month change in Movement Disorder Society Unified Parkinson Disease Rating Scale (MDS-UPDRS) part III score, on average, worsened by an additional 1.52 points for those on simvastatin compared with placebo. According to the study investigators, these findings provide no evidence to support a phase 3 trial. In the simvastatin arm, participants entered a 1-month phase of 40 mg daily dosing, following by 23 months of 80 mg daily doses, before a 2-month washout period. A total of 216 patients progressed to the 80-mg dose, and 178 of them were included in the primary outcome analysis. At 24 months, the mean change in MDS-UPDRS part III scores was 2.4 (SD, 11.2) in the placebo group and 4.5 (SD, 12.2) in the simvastatin group. Above all, the test of the futility hypothesis indicated that simvastatin was futile as a treatment for PD (P = .006).

In an updated review of randomized controlled trials, music showed to potentially be an effective approach for the improvement of subjective sleep quality for adults with insomnia symptoms.1 The review made it more apparent that listening to music could also be nonpharmacological intervention treatment for improving adults with insomnia as it has been currently used as a sleeping aid. In comparison with no intervention or treatment as usual (TAU), results showed moderate-certainty evidence for improved sleep quality in those in music groups as measured by the Pittsburgh Sleep Quality Index. In contrast, the review demonstrated very low‐certainty evidence for a difference of the effects between the groups on insomnia severity.Lead investigator Kira Jespersen, PhD, MSc, and colleagues noted that, “music probably facilitates a large improvement in the quality of sleep compared to no treatment or treatment as usual. We do not know if listening to music has an effect on the severity of insomnia (difficulty in falling or staying asleep) or the number of times a person wakes up (broken sleep) compared to no treatment or treatment as usual.”

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