‘Geographics’ impacting on amyloidosis drug access across Ireland
A group set up to raise awareness of Amyloidosis has told how it is ‘heartbreaking to think that due to geographics or living beyond the border in Ireland’ that access to a life saving drug cannot be granted.
ATTR Amyloidosis All Ireland Support Group formed on May 1, 2020 and came together with the aim of helping and supporting those who suffer from the disease as well as their families.
One of the founders, Rosaline Callaghan, is currently receiving the ground-breaking, gene-silencing drug Patisiran, by infusions every three weeks, administered by a specialist nurse in her Derry home.
However, the drug is not available across the border in the South and the group has now urged elected representatives and the Minister for Health to reverse this decision.
Rosaline, who founded the group with James Green, Jennifer Doherty, Bernie Cannon and Evelyn Cunningham, told the Journal how the group has been working hard behind the scenes and campaigning for treatment to be made available in Ireland, as over the last few years, treatments have emerged and some exciting new trials are being carried out. “It is heartbreaking to think that due to geographics or living beyond the border in Ireland that access to this drug cannot be granted. It is frustrating as we know of people suffering from the disease that are in dire need of treatment soon. There are at least 30 people on the island of Ireland needing access to this drug before it is too late for them. The HSE are currently in talks with the pharmaceutical company over pricing but hopes were dashed recently when an agreement was not met and now the process has to be restarted. Time is not on the side of those suffering from this disease.
“We urge Irish politicians and the powers that be to keep fighting for treatment for these patients. It is just not acceptable, particularly in the case of the Donegal mutation, that there is no treatment available in Ireland for what is a very Irish disease.
“We want people to know that Hereditary Amyloidosis Thr60Ala is a rapidly progressive, ultimately fatal disease in the absence of treatment.”
Rosaline’s father spent the last 22 months of his life in the Foyle Hospice and he died before any treatments were available. The gene-silencing treatment has been available in the North of Ireland since October 2019. There are around 12 Hereditary Amyloidosis patients in the north receiving this drug, which is ‘a treatment and not a cure’, and comes with its own set of challenges, but it does halt or slow down the progression.
“For patients similarly afflicted in Ireland to not have access to this game- changer is cruel and inhumane. One member of our group, living in Kildare, does not have access to it and we have seen him decline steadily over the past months. He is currently in hospital.”
Amyloidosis is a rare disorder and is caused by a buildup of abnormal or misfolded proteins in the body called amyloid. These are deposited into various organs and nerves which can lead to permanent damage and organ malfunction. Symptoms usually present as tingling/numbness or pins and needles in the limbs, severe gastro-intestinal issues and cardiac problems. Patients are often misdiagnosed as having carpal tunnel syndrome, arthritis, IBS, colitis, enlarged heart and so on. The Donegal mutation (thr60ala) is hereditary, a diagnosis that not only affects the patient but also their immediate and wider family circles as a parent has a 50/50 per cent chance of passing it onto their children.
The group recently undertook a Go Fund Me event which involved walking three million steps in March. They would like to thank everyone for their support so far and anyone wishing to make a donation can do so through www.gofundme.com/manage/3-million-steps-for-hattr-amyloidosis.
You can join the support group on Facebook - at ATTR Amyloidosis All Ireland Support Group, the page All Ireland AttrAmyloidosis Awareness or email Rosaline at [email protected]