Hundreds of patients, including infants and young children, are set to receive new NHS blood cancer treatments after other therapies have failed.
Around 200 patients a year will benefit from the two new treatments approved by NHS England, offering fresh hope to families when other options have not worked.
Dabrafenib is a tablet used to treat histiocytic neoplasms, rare and potentially deadly blood cancers. Without effective treatment, around one in 10 children with high-risk disease die within a year of diagnosis, while seven in 10 adults die within five years.
The potentially lifesaving treatment works by blocking proteins that help cancer cells grow, slowing or stopping the disease.
Its approval means patients can take the drug at home instead of spending time in hospital, causing less disruption to their daily lives than more invasive therapies.
Dr Rubina Ahmed, director of research, policy and services at Blood Cancer UK, said: “Today’s announcement is welcome news for people affected by some of the rarest blood cancers.
“Blood cancer is the UK’s third biggest cancer killer, and some of the rarest forms can be particularly hard to treat, with few options available if the disease comes back or stops responding to treatment. Every new treatment offers renewed hope for patients and their families.
“We’re entering a remarkable era for blood cancer treatment, with research transforming what’s possible for patients. It’s vital that people can benefit from these advances as quickly as possible, so we welcome more innovative treatments becoming available on the NHS.”
Children as young as eight with Hodgkin lymphoma that has returned or not responded to initial treatment will also benefit from a new drug combination.
Hodgkin lymphoma is a rare cancer that affects around 1,800 people in England each year. For between 10 per cent and 30 per cent of patients, around 180 to 540 people, the disease comes back or does not respond to standard treatment.
A new combination of brentuximab, vedotin and bendamustine targets cancer cells more precisely by binding to a protein on the surface of the cancer cell and delivering a cancer-killing drug directly into it.
Patients will receive the treatment through a drip into a vein. The combination has been shown to achieve high remission rates, is generally well tolerated and can help patients become well enough to receive a potentially life-saving stem cell transplant.
Professor Peter Johnson, NHS national clinical director for cancer, said: “This is a landmark moment for people with histiocytic neoplasms and Hodgkin lymphoma, giving them access to new treatment options they might not otherwise have had.
“For people living with the uncertainty of these rare cancers, these innovative therapies could offer something that can be hard to find – renewed hope – while allowing many patients to take their treatment at home instead of in hospital, so they can spend more time living their lives.”
Professor James Palmer, national medical director for specialised services at NHS England, said: “These new treatments could be life-changing, and will offer hope to hundreds of children and adults affected by rare blood cancers who have not had success with other treatment options.
“I want to thank the many clinical, commercial and patient experts that have helped NHS England arrive at this point because their expertise and support has been invaluable.
“The rollout of these new treatments are excellent examples of the NHS’s commitment to offering access to innovative medicines for those living with rare conditions.”
