Chesterfield family call for life saving drug to be made available on NHS

Little Grace Catt, from Tupton in Chesterfield, was diagnosed at four months with spinal muscular atrophy (SMA)type one – a genetic condition that causes muscle weakness and can leave children with respiratory difficulties.

Grace was offered a trial of a new drug called Spinraza.

There is currently no cure for SMA, but Spinraza has helped Grace with her condition.

Luke Catt, Grace’s dad, said: “If Grace hadn’t started the trial, she wouldn’t be here now. “Grace was in intensive care at Sheffield Children’s hospital on Christmas Day, and we were told she may not make it. Spinraza has increased Grace’s motion range, she was floppy like a newborn before. She can now hold her head up, and has even started to taste food.”However, although the drug has helped Grace, it is not available on the NHS.

The National Institute for Health and Care Excellence (NICE), the agency that decides on the cost-effectiveness of therapies for inclusion in the NHS, recommended against Spinraza, also known as nusinersen, being offered to treat people with SMA in august 2018. At an estimated £450,000 for a first year of treatment and £225,000 for subsequent years, the NICE report states, ‘the cost of nusinersen is too high for it to be considered a cost-effective use of NHS resources. Because of this, nusinersen is not recommended.”

Although Grace will continue on the trial of the drug for the foreseeable future, other families of youngsters with SMA are not so fortunate. Luke added: “We have met families locally who missed the cut off point for the trial, and their children died. It’s hard enough for us, but at least Grace has been given the opportunity to have Spinraza.”

Luke and wife Gemma are part of TreatSMA, an organisation that represents the voice of those affected by SMA and their parents and carers.TreatSMA held a protest against the policy on March 6, outside the NICE office in Manchester.