Join Our Boys mum weighs options after trial delays

By Rebekah O'Reilly

The family of Roscommon brothers George and Isaac Naughton have been forced to consider alternative treatments after delays to the life-saving cardiac trial which the boys were preparing to embark on.

George and Isaac suffer from Duchenne Muscular Dystrophy (DMD), a progressive neuromuscular disease that destroys muscle tissue and is currently fatal.

The hurdle comes after the family met their fundraising target of €650,000 in just weeks in February of this year. The delays in the trial have come due to recent policy changes in the US.

George and Isaac's mum, Paula Naughton, spoke to the Westmeath Independent about the severity of the situation.

“After Duchenne, time is our biggest enemy. For our family, we have already lost a son. The fear is that we would lose one of our boys, or both of them. We are willing to do anything to prevent that.”

Both George and Isaac played a huge role in the fundraising campaign for the cardiac trial, and Paula has noted that their brother Archie had been the driving force behind Join Our Boys Trust since the beginning.

“Before Archie passed, all he was interested in was finding treatment options for his brothers. From the age of seven, he spent his life fundraising for research and treatments, and trying to find answers. He always had so much hope, and he still plays such a huge role.”

While the cardiac trial is still set to go ahead, the lack of a timeline for the project has forced the family to begin seeking alternative options.

"Of course, it is getting to the stage that we don't know when the project will go ahead, and so we have started to look at other options. At the moment, the only other option is gene therapy, which is also only available in the US.

“The drug is called Elevidys by a company called Sarepta Therapeutics. We are speaking with two centres in the US that offer that treatment. It's one treatment, and it's a viral vector. There are a lot of considerations. There is the possibility that it will improve some function but you just don't know because every child is different.

“We are non-US citizens, so we will have to pay for it. The cost is up to $3.3million for each patient. This would mean we would have to raise over $6.6million, which is such an enormous ask from the public who have already been so generous. It's a huge moral and ethical dilemma.”

A viral vector is a type of virus that has been modified to safely deliver genetic material into cells. Scientists remove the harmful parts of the virus and use it as a delivery system to insert new or corrected genes into the body.

In the case of Elevidys, the viral vector is used to deliver a working version of a gene that helps produce dystrophin, a protein that is missing or faulty in children with Duchenne Muscular Dystrophy (DMD). This treatment aims to slow down the disease and improve muscle function, though results can vary from patient to patient.

Despite the setback, Isaac and George's mum Paula wants to reassure donors that all the money raised will go towards treatment for the brothers.

“We never thought we could raise so much for the treatment in the US in such a short amount of time, and I just want to reassure people that the funding is exclusively for George and Isaac's treatment.

“One thing we want people in our community to know is that the funds donated to us are ring-fenced, and they will not be spent on anything other than their treatment.”

As for the impact that Duchenne Muscular Dystrophy has had on the Naughton family, Paula said: “It's been very difficult, the reality of trying to do this, and raising funds to save your children's lives when you've already lost a child. It increases the urgency, and it is very challenging.

“What people don't see is the time and effort that goes into coordinating the fundraising. They don't see the begging, all the emails, and how challenging it is to have to ask people for help. It takes time away from your family, and takes me away from spending time with my boys.”

The Join Our Boys Trust has now been approached by The SuperPower Podcast with Gian Power, which focuses on the extraordinary stories and challenges that people live through. It's aim to to educate, support and inspire others.

The family will talk about the challenges they have faced as they battle Duchenne Muscular Dystrophy, and further highlight their fundraising efforts.

A number of fundraisers and events are set to take place over the coming months, as the Join Our Boys Trust and the Naughton family face the decision on whether to wait for the cardiac trial, or to move forward with fundraising for gene therapy.

One such event is the 'Cherry Blossoms at Kilgar Gardens' fundraiser which is taking place on April 12 from 11am to 1pm and 2pm to 4pm. To book, contact Attracta Doran on 0838453046.

The Naughton family would like to extend their sincere thanks to Paula Byrne and Attracta for their support on this event.

To keep up to date with their story, search Join Our Boys on Facebook and Instagram, or go to: https://joinourboys.org/

To donate towards their fundraising efforts, go to: https://www.gofundme.com/f/save-george-isaacs-hearts