What happens when a $2 million gene therapy is not enough

investing.com:

By Deena Beasley

LOS ANGELES (Reuters) — Baby Ben Kutschke was diagnosed at three months with spinal muscular atrophy, a rare inherited disorder which is the leading genetic cause of death in infancy globally. It leaves children too weak to walk, talk, swallow or even breathe.

So when in 2021 his parents heard about Zolgensma – a one-time therapy costing millions of dollars that promises to replace genes needed for the body to control muscles – they had high hopes.

They were disappointed.

After treatment with the $2.25 million therapy at almost eight months old, Ben was able to hold his head up for a few seconds – a significant milestone, his mother Elizabeth Kutschke told Reuters. But he did not advance to rolling over or sitting up, and after a few weeks doctors recommended the family add another drug to help him.

«I just started to worry,» she said from their home in Berwyn, Illinois. «He wasn't getting worse, but the gradual progress we had seen… was no longer happening.»

Ben is one of a growing number of patients with spinal muscular atrophy (SMA) whose doctors are turning to additional drugs on top of the gene therapy, six top U.S. neurologists told Reuters.

Their experience raises broader questions around other high-cost gene therapies coming to market, sometimes after accelerated regulatory approvals, drug pricing experts said.

Zolgensma, launched in 2019 by Swiss-based healthcare group Novartis as a «potential cure» for SMA, was the most expensive drug in the world at the time.

Gene therapies work by replacing genes – the body's blueprint for its development. The gene Zolgensma delivers instructs the body to make a protein vital for muscle control.

Other SMA therapies need to be taken continuously, but