Researchers in the U.K. have overthrown the orthodox view that childhood cancers have a low mutation burden, opening up new drug targets and opportunities for repurposing existing therapies. In particular, a high mutation rate is associated with a response to cancer immunotherapy. But although PD-1 checkpoint inhibitors are approved for treating pediatric cancers with a high level of microsatellite instability mutations, in general it is thought childhood tumors are not amenable to immunotherapy.

Researchers in the U.K. have overthrown the orthodox view that childhood cancers have a low mutation burden, opening up new drug targets and opportunities for repurposing existing therapies. In particular, a high mutation rate is associated with a response to cancer immunotherapy. But although PD-1 checkpoint inhibitors are approved for treating pediatric cancers with a high level of microsatellite instability mutations, in general it is thought childhood tumors are not amenable to immunotherapy.

A study has demonstrated the potential of a novel ligand-based CAR T-cell therapy for targeting CD7-positive T-cell malignancies, including T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphomas. The receptor CD7 is a prominent target antigen, being expressed in around 95% of T-ALL, 50% of peripheral T-cell lymphomas and 10% of acute myeloid leukemias.

Allogenica SAS has been awarded a €2.5 million (US$2.7 million) grant under the French government’s France 2030 program to help advance its universal CAR T candidate, XL-001, for CD19-positive hematologic cancers.

The absent, small or homeotic-like 1 (ASH1L) protein regulates the expression of HOXA genes, which are critical for the development of MLL1 translocations frequently found in leukemia patients. Knockdown of ASH1L leads to growth arrest and apoptosis of leukemia cells, thereby inhibiting leukemia progression suggesting that ASH1L can be considered as a therapeutic target in this setting.