Manchester health campaigners have welcomed the news that two city hospitals are among just a handful of UK centres which will be providing a life-changing new gene therapy for adults and older children.
Manchester Royal Infirmary and the Royal Manchester Children’s Hospital are on a list of just seven UK centres approved by the NHS to deliver a pioneering gene therapy to those living with severe thalassaemia, a rare and life-limiting blood disorder.
The NHS has negotiated a deal with manufacturer Vertex that will see the treatment enter its Innovative Medicines Fund (IMF), providing NHS patients with fast-tracked access while further evidence on its benefits to patients is gathered over the next five years.
Thalassaemia is a genetic condition that affects the body’s ability to produce haemoglobin. People living with the condition can experience a wide range of health problems including anaemia and excess iron, caused in part by the need for lifelong blood transfusions, which in turn can lead to conditions such as diabetes, heart problems and cirrhosis.
The two Manchester hospitals will now treat people from all across the North of England, saving them journeys of hundreds of miles to other centres in the Midlands and the South.
Manchester-based charities the Caribbean and African Health Network (CAHN) and the North of England Bone Marrow and Thalassaemia Association (NEBATA) described the news as a “breakthrough”.
Charles Kwaku-Odoi, Chief Executive of CAHN, added: “We are delighted with the introduction of Casgevy, a pioneering therapy that brings renewed hope for individuals living with thalassaemia as it represents a significant advancement in gene-editing therapy treatment.
“Access to such innovative care funded through the NHS is positive development, with the potential to transform lives by not only treatment but also the possibility of a cure. We look forward to supporting our community as they benefit from this healthcare breakthrough.”
Dr Sajida Rasul, Vice Chairperson of NEBATA, said: “NEBATA and our members welcome this announcement after decades of tireless advocacy for equality of access to cutting edge treatments and for the rights of people affected by thalassaemia to live a full, and unencumbered life. This crucial treatment offers the promise of healthy and free futures for people with thalassaemia.
“The burden of transfusions every two to four weeks on the health and day-to-day lives of people with thalassaemia is heavy and long-lasting. They have to take heavy drugs with many side effects to preserve organs from iron overload, have dips in strength and energy throughout their transfusion schedule and often struggle with fertility.
“By reducing or eliminating transfusion dependence, Casgevy can really open doors in people’s choices – where they live, if and where they work, how and when they travel – all things that most of us take for granted.
“It is heartening to see NICE and NHS England have worked with the manufacturer and heard the voices of thalassaemia patients and their families and given meaning to commitments to eradicating health inequalities.
“We are especially proud to support MFT, as a specialist centre, in delivering the treatment and have every confidence in the excellent haematology team.”
Professor Bola Owolabi, Director of the National Healthcare Inequalities Improvement Programme at NHS England, said:
“This is an incredibly exciting step forward in the treatment of thalassaemia and could drastically change the lives of those living with what can be an extremely painful condition.
“We are committed to reducing healthcare inequalities by rolling out new and pioneering treatments on the NHS for conditions, such as thalassaemia, which disproportionally affects people from some minority ethnic backgrounds.
“We hope this ground-breaking treatment enables people with beta thalassaemia to live longer, more independent and higher quality lives – without the need for regular treatments and hospital appointments and without pain, fatigue and other side effects that can come with this severe and life-limiting condition.”
Mark Cubbon, Chief Executive of Manchester University NHS Foundation Trust (MFT), which runs both hospitals, also welcomed the news.
Mark said: “Working in partnership, the hospitals of MFT have delivered a number of clinical firsts for the communities which we serve. Offering this life-changing treatment represents a lifeline to older children and adults who were facing significant challenges and hundreds of blood transfusions.
“We are very pleased to have been chosen to offer this pioneering care to improve the health and quality of life of our diverse communities not just in Greater Manchester, but across the North of England.”
Notes for Editors:
- For more information about thalassaemia and Casgevy please click here.
- The NHS is one of the first health services in the world to offer this life-altering treatment, which will be manufactured in the UK. This means that patients in the North of England will have early access to this life-changing therapy.
- Until now, the only curative treatment for thalassaemia was a stem cell transplant, but this option was available to only a few patients due to difficulties in finding compatible donors. Casgevy changes the game by offering a new path to health and freedom, without the challenges of donor matching or transplant rejection.
- Casgevy is the first approved therapy using the Nobel Prize-winning CRISPR gene-editing technology, offering hope where traditional treatments fall short. Clinical trials showed remarkable success, with 93% of patients no longer needing blood transfusions for at least a year after treatment.
- The therapy involves removing a patient’s blood stem cells, editing them using CRISPR to produce functioning haemoglobin, and reinfusing them back into the body. This once-in-a-lifetime treatment could allow patients to live free from the constant need for transfusions, offering a potential cure.