Good news! Cancer is history (soon)!
"An experimental immunotherapy has staved off brain tumours that would usually be incurable in four children. The therapy trains immune cells in a person’s blood to target three protein markers found in paediatric brain cancers.
The tumour-associated antigen T-cell therapy was given to 33 children and young adults with brain tumours that are usually fatal.
One patient with DIPG, a type of cancer that is usually fatal within a year, is alive two years post therapy.
Three others with other types of aggressive or recurrent brain cancers remain disease-free between two and five years on.
It is unclear why the treatment worked in some and not others. The small safety trial doesn’t deliver a cure yet, but the therapy doesn’t require genetic engineering, can target multiple proteins and might work better for solid tumours than chimeric antigen receptor (CAR)-T-cell therapy."
"Key takeaways:
- The multi-target design may help address tumor heterogeneity, one of the major barriers to successful treatment of aggressive childhood cancers.
- The trial successfully established a feasible manufacturing process, identified a maximum tolerated dose and defined an early safety profile – key milestones needed to advance the therapy into future Phase 2 studies.
- Researchers analyzed patients with DIPG or relapsed brain cancers, showing both responses and prolonged disease control, with some patients remaining disease-free years after treatment.
..."
From the abstract:
"Central nervous system (CNS) tumors are the deadliest cancers in children, highlighting the need for new therapies. The tumor-associated antigens (TAAs) WT1, PRAME and survivin are widely expressed by these tumors, and a manufacturing technique has been developed to target these intracellular TAAs using autologous, nongenetically engineered T cells.
Here we therefore conducted ReMIND, an open-label, phase 1 adaptive dose-finding study to determine the safety/feasibility of autologous, systemically administered trivalent T cells targeting WT1, PRAME and survivin in children with CNS tumors.
Eligible patients had newly diagnosed diffuse intrinsic pontine glioma without lymphodepletion (arm A, n = 16 enrolled, n = 11 infused) and relapsed/recurrent nonbrainstem CNS malignancies without (arm B, n = 28 enrolled, n = 18 infused) or with (arm C, n = 7 enrolled, n = 4 infused) lymphodepletion.
Primary end points were safety, feasibility and maximum tolerated dose determination; secondary end points included preliminary efficacy and immunobiological correlates, including in vivo TAA-T persistence and systemic immune activation. Dose level 3 (8 × 107 cells per m2 per dose) was determined as the maximum tolerated dose.
Treatment was well tolerated with fatigue and headache being the most common adverse events, although two possibly related serious adverse events of tumor swelling occurred. One grade 5 event in a patient with diffuse intrinsic pontine glioma with hydrocephalus, tumor edema and respiratory failure was categorized as a dose-limiting toxicity.
Median overall survival for arm A was 13.7 months from diagnosis (range, 6.2–32.0) and median progression-free survival for arms B/C was 5.0 months from infusion (range, 0.5–51.6).
Three patients in arms B/C are alive without disease at 31.8, 41.2 and 51.6 months without further treatment, including one complete response.
This trial met safety/feasibility primary end points with some preliminary signals of efficacy. ..."
Nature Briefing: Translational Research
Magnetic resonance imaging scan of a vertical section of the head of a child with a glioma brain cancer (dark red, centre)
