Lymphopenia concern with MS therapies

Cladribine has shown good efficacy and safety in real world treatment of relapsing multiple sclerosis but lymphopenia and herpes virus infections appear more common than seen in clinical trials, according to German researchers.

In a study involving 270 patients treated with cladribine, they observed a profound reduction of both relapses and new or enlarging MRI lesions. However severe lymphopenia was common in patients who had received previous treatment with dimethyl fumarate and was associated with increased rates of herpes virus.

“A decision towards cladribine as escalation treatment should be weighted carefully in those patients,” the researchers suggested in Multiple Sclerosis Journal.

Neuroscience unit wins reprieve

The Australian National University’s neuroscience research capability will survive in reduced form after the university dropped a plan to axe the Eccles Institute of Neuroscience.

The ANU had proposed scrapping the institute as part of a wide-ranging cost-cutting plan that would cut 15 positions, provoking a public outcry, according to the Canberra Times.

However under a new plan that involves voluntary redundancies, the institute will stay within the John Curtin School of Medical Research with cellular and circuit neuroscience areas retained, as part of a new interdisciplinary Eccles Institute of Neuroscience and Brain Sciences.

Gene replacement therapy shows promise in SMA

The gene replacement therapy onasemnogene abeparvovec has shown ‘sustained and durable efficacy’ in a phase 1 trial in infants with spinal muscular atrophy (SMA).

In the START clinical trial, 13 of 15 children who were treated with a single intravenous dose of onasemnogene abeparvovec as infants continued to show a favourable benefit to risk ratio for up to 6.2 years after dosing, with no treatment-related serious adverse events reported during long-term follow-up.

Writing in JAMA Neurology, the study investigators said the results of the ongoing trial suggested the treatment improved motor function and extended survival, with a median age of 30.8 months for the low-dose cohort of patients and 25.7 months for the therapeutic-dose cohort.

Efficacy would be confirmed in phase 3 studies of the therapy, they said.