Cancer patients living with a rare disease which attacks the body's own organs following a life-saving stem cell transplant are set to benefit from a pioneering new medicine on the NHS.
Belumosudil (Rezurock ®) is an innovative treatment for chronic graft versus host disease, which develops in around a third of patients who have undergone stem cell or bone marrow transplants for blood cancers such as leukaemia and lymphoma.
Around 1,200 patients undergo a donor stem cell or bone marrow transplant each year in England, but around 400 recipients will develop a serious complication known as graft versus host disease, where white blood cells in the donated tissue begin to attack the recipient's organs and weaken their immune system.
The condition - which typically occurs within the first year after a transplant - is usually mild but can be fatal in one in ten cases. The disease causes inflammation and damage to vital organs such as the lungs and liver and weakened immune systems mean patients are more vulnerable to infections.
For around half of patients with chronic graft versus host disease, their condition will not respond to currently available treatments such as pentostatin (a chemotherapy drug), rituximab (antibody treatment) and imatinib (a targeted therapy).
Around 200 patients living with the most severe cases of the disease and who have not had success with two previous therapies are now set to benefit from belumosudil over the next three years, after the NHS fast-tracked funding for the treatment following clinical guidance from the National Institute of Health and Care Excellence (NICE).
The drug is given once a day as a tablet and works by inhibiting the proteins which are responsible for the potentially life-threatening inflammatory response caused by chronic graft versus host disease. It can be taken at home, reducing the number of hospital appointments for some patients.
In clinical trials using belumosudil to treat chronic graft versus host disease, up to three quarters of patients responded to therapy and this was most effective for treating disease affecting the mouth, intestine or joints.
The NHS England announcement comes on Rare Disease Day (29 February), which aims to highlight the more than 7,000 rare diseases that around 3.5 million people across the UK live with.
Dan Yeates, a 31-year-old from South West London, was diagnosed with acute lymphoblastic leukaemia in January 2020, and following a stem cell transplant in June 2020, developed chronic graft versus host disease.
He said:
“I have chronic graft versus host disease in my eyes, skin, gut and lungs, and it severely impacts my quality of life. After my stem cell transplant, it has been an incredibly hard journey, both physically and mentally.
“This announcement signals a massive quality of life improvement for many people like me. Now, it is such a relief to know that if my disease progresses, there will be a treatment available to me to take at home.”
NHS England's Medical Director for Specialised Services, James Palmer, said:
“This rollout is a real breakthrough for patients with graft versus host disease and it will help people living with this debilitating condition effectively manage their symptoms and enhance their quality of life and daily functioning.
“It's great news that the NHS has been able to fast track funding for a treatment which will have a significant impact for hundreds of patients over the coming years.”
The treatment is made available through the NHS England's Innovative Medicines Fund, which fast-tracks the most promising non-cancer medicines to patients.
It is the fourth treatment that has been made available to patients earlier through the Innovative Medicines Fund. Last September, the NHS used the fund to rapidly rollout Sebelipase alfa, a treatment for Wolman's Disease - a rare genetic and fatal condition which presents in babies and children.
Professor Adrian Bloor, a Consultant Haematologist at The Christie NHS Foundation Trust, said:
“The rapid rollout of belumosudil provides hope for patients living with chronic graft versus host disease, and signals a major step forward in addressing the often-debilitating consequences of the disease. Although belumosudil has only been available for a short time, it is already demonstrating a transformative benefit for patients under my care.”
Dr Robert Danby, Chief Medical and Scientific Officer at Anthony Nolan, the stem cell transplant charity, said:
“We know from our patients only too well the devastating effect chronic graft versus host disease can have on a person's physical and mental health.
“This is welcome news for stem cell transplant recipients across England and Wales who struggle with this debilitating, life-threatening condition. We are investing in vital research to help even more patients survive and thrive after a stem cell transplant.”
Belumosudil is the latest innovative medicine that has been secured for NHS patients following recent rollout of wearable Parkinson's treatment, foslevodopa-foscarbidopa and preventative breast cancer drug, anastrozole.