Landmark NHS approval of cancer drugs offers new hope for children and adults with rare blood cancers

Infants and young children are among hundreds of patients set to receive new life-saving drugs for rare blood cancers.

Around 200 patients a year will benefit from the two new cutting-edge treatments approved by NHS England, offering fresh hope to families when other therapies have failed.

Dabrafenib is a tablet that treats 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 will also mean patients can take the drug at home instead of spending time in hospital, causing less disruption to their daily lives than more invasive therapies.

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. But for between 10% and 30% 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, binding to a protein on the surface of the cancer cell, 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.

Improving the treatment for rare cancers is a key ambition of the new 10 Year Cancer Plan, and the NHS is committed to ensuring more patients have access to advance, safe and effective treatments, which can save or transform their life.

The rollout of these new treatments from today is thanks to NHS England's Clinical Priorities Advisory Group (CPAG), which expertly assesses dozens of specialist medicines, medical devices and treatments each year, according to their benefit for patients, clinical effectiveness, and value for money.

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.

CPAG's experts noted how the therapeutic landscape for these disorders has significantly evolved, with the discovery of specific genetic drivers, many patients can now benefit from targeted therapies, such as BRAF and MEK inhibitors.

The new NHS precision treatments should offer better outcomes and fewer side effects for patients, shifting the standard of care toward personalised, mutation-specific approaches for those who do not respond to or cannot tolerate conventional therapies.

One person who has already benefited from one of the drug treatments which will now be available on the NHS is Lesley Coombs

Lesley, a 69-year-old woman from Cambridgeshire received dabrafenib four years ago through a compassionate access programme offered by the drug's manufacturer. Lesley had been undergoing treatment for follicular lymphoma alongside histiocytosis with the BRAFV600E mutation, which she describes as a 1 in 60 million combination.

Following several rounds of chemotherapy that had successfully treated the lymphoma, the small lump on her neck had not shrunk as hoped before the tumour started to grow again. Radiotherapy was then organised in the hope it might prove effective, but before that was started Lesley was provided access to dabrafenib.

Without the medicine, in addition to radiotherapy Lesley may have also required a stem cell transplant that would have led to a long recovery, even if successful.

Lesley said: I started the dabrafenib 10 days before my planned radiotherapy, but within three days of starting the drug the tumour had rapidly started to shrink. My family and I were amazed - the radiotherapy was put on hold and to this day I continue to be in remission.

Thanks to the drug I have been able to continue living a very active lifestyle - I regularly cycle 40 miles, walk up to 10 miles, and enjoy holidays full of activities. Most important of all is the absolute pleasure and enjoyment of spending time with my grandchildren, enjoying the activities we do together.

Access to the drug and the care of incredible staff at both Hinchingbrooke and Norfolk & Norwich University Hospitals allowed me to pick up my life again after cancer and I'm delighted many more people will now be able to benefit from this medicine too.

The main symptoms of Hodgkin lymphoma are a painless lump or swelling, usually in the neck, armpit or groin; pain in the glands in your neck, armpit or groin when some drinks alcohol; a high temperature; night sweats; shortness of breath; itchy skin; and sudden weight loss.

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."

For malignant histiocytosis, non-specific progressive symptoms include one or more lumps that rapidly grow, localised pain, numbness, tingling and persistent fever.

Lynn Jackson, Executive Director at the charity, Histio UK said: This announcement is excellent news for all our patients with BRAFV600E mutation positive histiocytic neoplasms and for our Histio Champions and the team at Histio UK who have worked so hard to achieve this amazing outcome.

NHS access to dabrafenib for those who are eligible will change lives and outcomes now and in the future.

The CPAG process looks at drugs and treatments that are not being assessed by NICE, for example where they are not licenced for the clinical indication in question.

Public Health Minister Sharon Hodgson said: For people living with rare blood cancers, every new medicine can make a real difference. These innovative treatments are an important step, giving hundreds of children and adults access to cutting-edge care on the NHS, offering fresh hope to patients and their families.

"Through our National Cancer Pln, we are committed to improving cancer care and delivering better outcomes for all those diagnosed with it. By backing innovation and working closely with the NHS to bring promising medicines into routine care, we are helping ensure more patients can benefit from the latest breakthroughs, while giving families greater confidence that world-class treatment is available closer to home.

This latest package of new cancer treatments follows the CPAG decision at the start of this year to fund prostate cancer drug, abiraterone for thousands of men with prostate cancer that had not spread.