VIDEO: Promising advancements in brain cancer treatment

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It's been really hard to treat brain tumors, but recently, there have been some important advances. In other cancers, there's been a lot of progress in targeted therapies, and for brain tumors, the advances have lagged behind, but in the past couple of years, there have been real improvements. In low-grade gliomas with IDH mutation, in 2023, the phase 3 INDIGO trial showed that vorasidenib [Servier] was very active in increasing progression-free survival for these tumors, and this drug is likely to be approved for grade 2 IDH-mutated tumors later this year. There have also been advances with targeted therapies for upper low-grade tumors, especially in the pediatric population. Tovorafenib [DAY101, Day One Biopharmaceuticals], a type 2 RAF inhibitor, has shown very high response rates in a trial of recurrent BRAF-altered low-grade gliomas in children, especially those with BRAF-KIAA fusions, and this drug also will likely be approved some time this year. There have also been advances using the combination of dabrafenib and trametinib for BRAF 600E-mutated gliomas, both in adults and children, and this has also led to regulatory approval.

Another area where there has been advances — although not a regulatory approval — has been in the area of immunotherapies. There's been particular interest in [chimeric antigen receptor (CAR)] T-cell therapy, both for diffuse midline gliomas with H3 K27M mutations, as well as for glioblastomas, and responses have been seen with some of these therapies, although the responses have not been as durable as we would like. There's also been a lot of interest in viral therapies for glioblastoma, both to directly kill tumor cells and also to augment the antitumor response. And in Japan, there's been a study with a herpesvirus where multiple injections of the virus has significantly improved survival in recurrent glioblastomas, and similar studies with multiple injections are going on in the U.S.

And then there are two other types of tumor where there has been important advances. Brain metastases, which has traditionally been a difficult area to treat, has seen multiple advances, both with targeted therapies and immunotherapies, and then even most recently with antibody-drug conjugates. This is due in part to the fact that the blood-brain barrier is disrupted in brain metastases, so many of these agents that don't cross the blood-brain barrier very well can still cross sufficiently to have a therapeutic effect. And then, finally, primary [central nervous system (CNS)] lymphoma, that really has had no significant advance in treatment since the introduction of methotrexate has started to see really encouraging results with immunotherapies and especially with CAR T-cell therapy. So, for multiple brain tumors, there really has been important progress over the past 2 years.