A phase 3 clinical trial has found that a chemotherapy-free treatment pairing produced markedly better two-year survival rates in adults with a hard-to-treat form of leukaemia.
The New England Journal of Medicine has published full results from the GIMEMA ALL2820 trial, which tested a new approach to treating Ph-positive acute lymphoblastic leukaemia (ALL) — a type of blood cancer where a specific genetic fault, called the Philadelphia chromosome, makes the disease more aggressive and harder to treat. The trial compared two treatment strategies in adults with the condition.
Patients receiving the combination of ponatinib (a targeted drug that blocks the faulty signals driving the cancer) and blinatumomab (an immunotherapy that directs the body’s own immune cells to attack cancer cells) had a 24-month event-free survival rate of approximately 87–90%. Those treated with imatinib — the previous standard targeted drug — alongside conventional chemotherapy had a rate of approximately 71–74%.
That gap — approximately 16 percentage points at the two-year mark — is the headline finding from a randomised phase 3 trial, the type of large, controlled study considered the most reliable basis for changing clinical practice.
The ponatinib–blinatumomab approach is notable because it removes chemotherapy from the treatment entirely, relying instead on two targeted agents. Chemotherapy carries well-documented side effects, so a regimen that matches or exceeds its results without it has been a long-standing goal in blood cancer research.
The findings were posted to social media by the New England Journal of Medicine, which published the full trial data.
Ph-positive ALL accounts for around a quarter of adult ALL cases and has historically carried a poorer outlook than other forms of the disease. Treatments have improved markedly since imatinib became standard care, but relapse remains a serious risk.
Anyone concerned about symptoms of leukaemia — which can include persistent tiredness, unexplained bruising, frequent infections, and night sweats — should contact their GP or call NHS 111.
Source: @NEJM