New blood cancer treatments approved for UK patients - EMJ

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UK Lymphoma and Leukaemia Patients Access New Treatments

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Key Summary:

  • NICE has approved new first-line treatment options for eligible patients with specific leukaemia and lymphoma.
  • The BTK inhibitor drugs have shown improved outcomes in clinical trials compared with standard approaches.
  • These approvals expand the role of targeted therapies in blood cancer care.

NEW TREATMENT options will now be available to patients in the UK with specific types of lymphoma and leukaemia.

The National Institute of Health and Care Excellence (NICE) ruled last week that eligible adults in England and Wales with mantle cell lymphoma (MCL) and chronic lymphocytic leukaemia (CLL) will now be able to access the drugs on the NHS.

For patients with CLL, it has introduced the first and only all-oral regime incorporating a second-generation BTK inhibitor. For those with MCL, it now offers a targeted BTK inhibitor as part of the first-line treatment pathway for those eligible.

BTK Inhibitors

BTK Inhibitors work by blocking the Bruton’s tyrosine kinase enzyme, which is responsible for driving the growth and survival of B cells. By shutting down this signalling pathway, these medications can be used in certain blood cancers to stop abnormal white blood cells from multiplying.

Patients diagnosed with CLL who have received no previous treatment will now have access to acalabrutinib plus venetoclax, a drug that was found to reduce disease progression and death by 35% compared with standard-of-care chemoimmunotherapy. 1

Research into the medication also showed after three years of treatment with acalabrutinib plus venetoclax, 77% of patients were progression-free, compared to 67% of their peers receiving chemoimmunotherapy1.

Acalabrutinib in combination with bendamustine and rituximab is the new option NICE is recommending for patients with MCL. In trials, the drug significantly improved progression free survival compared with placebo plus bendamustine and rituximab. 2

Median progression-free survival was 66.4 months for patients given the acalabrutinib combination compared to 49.6 months for those on the placebo plus bendamustine and rituximab. 2

Welcome Recommendation

Several health care professionals have endorsed NICE’s recommendations for the new treatments, which will be produced by AstraZeneca.

Dr Toby Eyre, Consultant Haematologist at Oxford University Hospitals NHS Foundation Trust, said: “We welcome NICE’s recommendation of acalabrutinib plus venetoclax for people living with chronic lymphocytic leukaemia in England.

“This fixed-duration treatment option provides eligible patients with access to an innovative regimen that offers the flexibility of planned time off treatment.

“Expanding treatment choice through the NHS is an important step forward and may help reduce the burden of therapy while supporting patients to live well with their disease.”

Dr David Lewis, Plymouth Hospitals NHS Trust, said: “NICE’s recommendation of acalabrutinib in combination with bendamustine and rituximab as a first-line treatment for mantle cell lymphoma is an important step for patients in England.

“As an incurable disease that mainly affects older people, optimising treatment from the outset is critical. This NHS access decision gives eligible patients the opportunity to benefit from the only targeted treatment currently available in the first-line setting and could help establish a new standard of care for transplant-ineligible patients.”

These approvals expand the role of targeted therapies in blood cancer care. By using medicines that block cancer-driving pathways, these treatments provide additional options for patients and clinicians.

References

1 Lymphoma Action. Mantle cell lymphoma. Available at: https://lymphoma-action.org.uk/information-and-support/types-lymphoma/non-hodgkin-lymphoma/mantle-cell-lymphoma

2 Brown JR, Seymour JF, Jurczak W, et al. Fixed-Duration Acalabrutinib Combinations in Untreated Chronic Lymphocytic Leukemia. New England Journal of Medicine. 2025;392(8):748-762. Available at: https://doi.org/10.1056/NEJMoa2409804

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