The Scottish Medicines Consortium (SMC) has approved two new drugs for blood cancer treatment on the NHS in Scotland, offering hope to patients with aggressive forms of these diseases. Axicabtagene ciloleucel (axi-cel), known as Yescarta, a CAR-T therapy, has been approved for adults with diffuse large B-cell lymphoma (DLBCL) and high-grade B-cell lymphoma (HGBL). Additionally, quizartinib (Vanflyta) has been recommended for newly diagnosed FLT3-ITD-positive acute myeloid leukemia (AML). These approvals mark a significant step forward in blood cancer treatment in Scotland, aligning it with standards in England and Wales.
Yescarta for DLBCL and HGBL
Yescarta is a CAR-T therapy that involves modifying a patient's own T cells to target and destroy cancer cells. DLBCL is an aggressive type of non-Hodgkin lymphoma, often treated with stem-cell transplants. The SMC's approval of Yescarta follows a resubmission from Gilead, the pharmaceutical company behind the drug, after initial concerns about cost-effectiveness. The approval now allows Yescarta to be used as a second-line treatment for patients whose cancer has returned within a year of initial treatment or does not respond to first-line therapies.
Kirsty Stewart, a 31-year-old from Aberdeen living with DLBCL, expressed her relief at the news, stating that Yescarta offers "fresh hope" and a "better chance of finding a cure and living a more normal life." She highlighted the challenges of living with DLBCL, including constant fatigue and the fear of relapse.
Josh Hill, Blood Cancer UK's Scottish Policy Officer, emphasized the importance of this approval, noting that blood cancer survival rates in the UK lag behind other wealthy nations. He stated that earlier access to CAR-T therapies like Yescarta is crucial for improving outcomes for those with aggressive blood cancers.
Quizartinib for FLT3-ITD-Positive AML
Quizartinib (Vanflyta), developed by Daiichi Sankyo, has also been approved for the treatment of newly diagnosed FLT3-ITD-positive AML. AML is a fast-growing blood cancer requiring urgent treatment. Quizartinib, a tyrosine kinase inhibitor, will be used in combination with standard cytarabine and anthracycline induction, and standard cytarabine consolidation chemotherapy.
In Scotland, approximately 150 people are diagnosed with AML each year, with FLT3-ITD mutations present in about 25% of cases. Before this approval, there were no targeted treatments available in Scotland for this specific mutation. The addition of quizartinib to the treatment regimen is expected to significantly improve outcomes for these patients.
Aileen Lamb, from Edinburgh, who had acute myeloid leukemia and is now in remission, underscored the psychological impact of a blood cancer diagnosis. She noted that the approval of quizartinib provides not only a potential new road to recovery but also much-needed hope for patients facing this challenging disease.
