Positive steps towards an MND drug therapy

23 Sept 2022

There is hope on the horizon for people with motor neurone disease as a first ever therapy to slow progression has found positive outcomes in clinical trials.

Tofersen, developed by Biogen, has been especially designed to target a subset of people living with motor neurone disease (MND), also known as amyotrophic lateral sclerosis (ALS). In between 8-23% of cases, the disease is thought to be caused by a mutation of the protein SDO1 (Muller 2022). It is this protein that Tofersen targets.

The outcomes of VALOR, the phase 3 clinical trial for Tofersen were published in the New England Journal of Medicine, and whilst it did not achieve its clinical endpoint, demonstrated some potentially positive findings.

An open label extension has found that people with MND on the therapy demonstrate reduced levels of SDO1 and a slowing of their MND progression over a twelve month period.

Additionally, researchers found a corresponding reduction in levels of neurofilament light chains, a potential marker of nerve damage which is used in monitoring other neurological conditions such as multiple sclerosis (MS).

This is the first drug found which may impact MND disease progression, and provides hope for a future with viable therapies to support people with MND.

'This new data gives us hope that effective therapies for MND are now a reality and that with more investment, more research and continued interest from pharmaceutical companies, additional therapies that will benefit all people with MND are closer than ever before.'

MNDA Research Blog 6 June 2022

To find out more, visit MND Association's research blog, read Biogen's announcementor see the initial VALOR paper published in the New England Journal of Medicine.

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