Taskforce launched to drive access to treatments

The Motor Neurone Disease Association has convened a taskforce of experts from a range of disciplines to focus on access for people with MND to new and emerging treatments.

The announcement came in the same week that Tofersen received US Food and Drug Administration approval, under the name Qalsody, as a therapy for people with MND who have the SOD1 gene mutation. The approval marks a step forward in the fight against MND and paves the way for similar applications to be made in Europe and the UK.

With further positive signals around trials involving Interleukin-2, a repurposed cancer drug, the MND Association has now pledged a significant investment and recruited to a new post to form a taskforce, which includes people with MND, focused on ensuring people living with the disease can access potential treatments at the earliest opportunity.

It can take many years for a potential treatment to progress from the laboratory to the clinic and there are a number of complex processes to navigate involving multiple groups, from researchers to pharmaceutical companies, regulators to pharmacists.

Yet people with MND don’t have time to wait. A third of those diagnosed with the disease die within a year and half within two years.

MND Association Chief Executive Tanya Curry said:

"People with MND desperately need some hope. Right now, researchers are talking about being on the cusp of really meaningful breakthroughs and we need to be ready to act as those happen.

"Recognising the need for urgency, and in line with our Promise to the MND community that we will not rest until MND is treatable and ultimately curable, we have committed to the creation of this Taskforce. Already we have engaged with experts from pharmaceuticals, leading neurologists, manufacturers and specialists in the field of market access. We are seeking to pool knowledge, expertise and resources to do everything possible to accelerate access to effective treatments as and when they become available."

Following its inaugural meeting earlier this month, the Taskforce is currently focused on identification of the barriers to access and how they can be overcome.

While research into the causes and potential treatments for MND has progressed significantly in recent years, in the UK there have been no new drugs licensed since Riluzole, 25 years ago, which has shown to extend life.

At the end of 2022, the MND Association had a research portfolio worth £20 million, and is the largest charitable funder of MND research in the UK, with much of that work focused on translational research – taking potential treatments from the laboratory into the clinic.

We will continue to update the MND community as this work progresses.