A REDLYNCH man with a progressive and life-threatening disease says a new gene silencing treatment is giving “hope” to sufferers.

Vince Nicholas has hereditary transthyretin-mediated (hATTR) amyloidosis, which causes a build-up of abnormal proteins called amyloid in the body.

The disease has had a devastating impact on his family, his mother Lillian died from the disease in 1991 and her five siblings also died from it. Vince’s twin brother, Keith, died from it in 2015 and he has another sibling with the disease.

The National Institute for Health and Care Excellence has now recommended the use of patisiran on the NHS in England to treat the disease. Prior to this there have been few treatment options.

Vince underwent a liver transplant in 2010 as a way to slow the progression of the disease as he says “there was no other option”.

He says this announcement is a “miracle”, adding: “This is going to mean the next generation, or even closer, they won’t have to go through hell and make critical choices in their lives like having liver transplants, having to retire early and having to rely solely on your wife or family because you can’t do anything anymore. It is amazing, absolutely wonderful.”

He added: “It is a momentous day for all of us who have been struggling with amyloidosis, my whole family for the last generation really. It is fantastic. We really weren’t expecting it, we knew it was being done but we didn’t know it would be coming through in our lifetime or certainly my time. It is huge.”

The 59-year-old was diagnosed with the amyloidosis gene when he was 30 after the death of his mother but he did not start getting symptoms until about 12 years ago.

“It is like the furring of a kettle, the body can’t get rid of all of it,” said Vince, “It furs up and attaches itself to the nervous system and around the heart and main organs. It eventually gets so much that everything starts shutting down. You get pins and needles, numbness in your legs, you can’t feel things with your hands. You can’t eat properly, you can’t walk, and you can’t do things. Your whole life changes, you have to get other people to do things for you.”

But, he says since he has been taking the drug he has been able to do more things than before, adding: “The main thing is it has given me hope that I haven’t got a major terminal illness which I would have had. If I hadn’t had a liver transplant I wouldn’t be here today. Before I was on the drug I knew I would probably have at least five years or less before I die.”

Patisiran helps in reducing the production of an abnormal protein that damages nerves and organs across the body.

Professor Philip Hawkins, the head of the National Amyloidosis Centre at the Royal Free Hospital, London, said the decision marked an “important step forward” in the treatment of a disease that is “life-threatening to patients and devastating to families”. He added: “Patisiran has shown in its main clinical study that it can halt or even improve potentially debilitating symptoms of this disease in the majority of patients. This means we now have a real possibility of preserving quality of life for eligible patients for longer than has so far been possible. Gene-silencing is a promising area of medicine and it is heartening to see this science translating into treatments that can potentially help those suffering from serious illnesses like hATTR amyloidosis.”