Gene treatment trial for seriously ill Hartlepool boy

Luke Malcolmson, who has a rare disease, is looking forward to a treat when Rough Diamond perform for him. Luke is pictured with his mam Jayne.

Luke Malcolmson, who has a rare disease, is looking forward to a treat when Rough Diamond perform for him. Luke is pictured with his mam Jayne.

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A BRAVE little Hartlepool boy - who is living with a rare medical condition - is a step away from qualifying for clinical trials which could drastically change his life.

Five-year-old Luke Malcolmson suffers from a muscular condition called Duchenne muscular dystrophy. It causes muscles to progressively weaken because the cells break down and are gradually lost.

His family has been told that he is suitable to take part in new gene therapy clinical trials which start next month in Newcastle. The question is whether Luke, who is a pupil at Sacred Heart Primary School in Hartlepool, will be one of the people chosen.

His mum Jayne Malcolmson, 42, explained the difference that the trial could make. “The theory is that this changes what he has from being really devastating to a less aggressive form of muscular dystrophy.”

Duchenne muscular dystrophy affects just one in 3,500 males born in the UK.

It could leave the youngster unable to walk by the time he is just 10 years old. It is a currently incurable disorder which causes muscle degeneration and eventual death.

But the gene therapy trial could affect both his lifespan and how long he can walk for - meaning he could still be walking into his 40s.

The trial involves a process called exon skipping. Exons are part of our DNA but some of the exons are deleted in people with Duchenne muscular dystrophy. Scientists have come up with a method in which some people can receive treatment where the missing exons can be skipped over, in an effect compared to a sticking plaster.

This leads to the production of dystrophin in the muscles, which can hopefully improve muscle function.

“It’s like a jigsaw puzzle,” said Jayne. “Luke is suitable for the new clinical trial but he has not been enrolled yet. We hope that he might get into the trial because the potential is huge. He would be one of the first children in the UK to be trialled.”

In the meantime, Luke’s aunt Gemma Hanna, 33, will head to Iceland for an eight-day fundraising mission in October to raise £5,000 for a charity called Action Duchenne.

To support her, visit the Action Duchenne muscular dystrophy website at www.actionduchenne.org to make a donation.