Breakthrough Life Science Prize Awarded to Baby-Saving Drug

Scientists behind a baby-saving drug have won the most prestigious award in their category at the Breakthrough Prize ceremony.

Earlier this week, those in science, maths, and physics were honoured at the award event, with a total of $22 million (£16.87 million) awarded for the winners.

The 2019 Breakthrough Prize in Life Sciences was given to C Frank Bennett and Adrian R Krainer for their gene therapy drug Spinraza, as well as Angelika Amon, Xiaowei Zhuang, and Zhijan ‘James’ Chen.

Spinraza has been designed for infants suffering from Spinal Muscular Atrophy (SMA), which is fatal in babies.

Cori Bargmann, chair of the selection committee, said: “The winners of the Breakthrough Prize in Life Science show us all how it’s done. Through creativity, innovation, persistence, and skill, each of them brought about an advance that was previously unimaginable.”

SMA sufferers do not typically live beyond the age of two. However, the scientists put their skills and experience together to come up with a successful therapy so the condition does not have to be a death sentence.

Bennett and Krainer’s drug targets a closely related gene to encourage it to produce a motor neuron protein. This strengthens and extends the life of motor neurons in the body, meaning signals from the brain to the muscles continue to work.

Speaking with The Guardian, Professor Kevin Talbot, head of clinical neurology at the University of Oxford, said the therapy “is demonstrated to have a dramatic effect in children”.

However, he noted the expense of the drug, which is why winning awards such as this helps to launch therapies and maintain research into them.

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