$3 million 'Breakthrough' prize for Developers Behind Spinraza

18 October 2018

The article in The Guardian, published yesterday, states:

"The 2019 Breakthrough prize will see seven winning discoveries each celebrated with a $3m award for those behind the research to share...

Four of the seven top awards are going to academics in the life sciences, among them C Frank Bennett, senior vice president of research at Ionis Pharmaceuticals, and Adrian Krainer of Cold Spring Harbor Laboratory, who developed the drug [Spinraza] to treat the condition spinal muscular atrophy (SMA).

Bennett and Krainer developed the first drug to treat the disease, which works by targeting not the problem gene but another closely related gene. In doing so, it boosts the ability of this secondary gene to produce the all-important survival motor neuron protein..."

The article also quotes Prof Kevin Talbot, head of clinical neurology at the University of Oxford:

Prof Talbot said Bennett and Krainer’s award was well deserved, adding that the discovery represents a landmark in how neurological diseases are treated.

"Spinal muscular atrophy, a hitherto completely fatal disorder of children, is now resulting in children who are surviving, and that is a triumph,” said Talbot. But, he added, more needs to be done to make it available.

“This is a treatment that has been available for the last 18 months, roughly, and is demonstrated to have a dramatic effect in children,” he said. “It is quite clearly something that works. The problem is that it is phenomenally expensive.”

While Talbot notes the drug, called nusinersen, is available in almost all developed countries, including the US, Canada and Scotland, it has not been approved by the health watchdog, Nice, in England and Wales. “Nice don’t seem to have a way to deal with these rapid moving developments in medicine,” he said. “It is a major problem.'

Read the full article