Promises and Problems in Brain Cancer Therapy: What’s Hot Right Now?

Brain cancer offers a unique challenge for treatments, as the nervous system can often be harder to direct treatment towards, though there are novel drugs that hold a lot of promise, according to John Henson, MD.

During a site visit to the Georgia Cancer Center in Augusta, GA, CancerNetwork® spoke with Henson about brain cancers, among other topics. Currently, there is [significant] excitement surrounding novel therapies, specifically about the ability to alter the genes inside the tumor cells with a viral delivery mechanism. Additionally, there are drugs that alter the metabolic processes in the tumor cells that are exciting, with Henson specifically citing the recent FDA approval of vorasidenib (Voranigo) for grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation.1

Notably, he also discussed the difficulty developers/investigators have had employing immunotherapy in patients with malignant gliomas, as the nervous system is more troublesome for the immune system to reach when targeting tumor cells. Surgery and radiation chemotherapy, when given up-front, also pose a challenge for immunotherapy’s efficacy, as sometimes the treatments themselves can reduce the efficacy of immunotherapy. For this reason, investigators and researchers are pushing to move immunotherapy to the beginning of treatment.

Henson is the director of the Hereditary Brain Tumor Clinic and the Brain Tumor Program at Georgia Cancer Center.

Transcript:

CancerNetwork: What novel research or developments in the neuro-oncology field hold promise to improve outcomes among different brain cancer populations?

We are facing some interesting situations in terms of new therapies. We have an emerging ability to alter the genes that are inside the tumor cells, typically using a viral delivery mechanism. There are novel drug therapies that are coming out that are going to hold a lot of promise.

Some of them have not delivered that promise yet. For example, with immunotherapy, which has been important in many other kinds of cancers, we have not found the way to employ that in the most useful manner for patients with malignant gliomas. So that’s an example where there’s this whole burgeoning field in oncology that’s been so helpful to many different types of tumors, that we have not been able to take advantage of. Then we have interesting drugs that are being developed right now for altering the metabolic processes that are inside the tumor cells. I am thinking specifically of the recently approved IDH1 and IDH2 inhibitors; we now have a pill where we can block gliomas that are formed based on IDH mutations. These are examples of things that are literally pouring out of the pipeline. We are very excited about that.

Why has immunotherapy been harder to develop for brain cancers vs other disease states?

We are not entirely sure why that is, but there are a number of theories. One is that, although you can ramp the immune system up with immunotherapy to try to attack the tumor, the nervous system is a special space in terms of the ability of the immune system to get there. In addition, we have typically employed those treatments at a time after the patient has gotten their surgery and their initial radiation chemotherapy treatment, and there’s reason to suspect that those treatments, in and of themselves, may create a difficult environment for the immune system in which the immune system could try to attack the tumors. That somehow the treatment itself is making it more difficult for the immune system to attack. One of the things that is currently a fairly large topic in the immunotherapy of malignant gliomas is trying to move that treatment far up in the beginning of therapy. Not waiting until after the patient finishes their radiation chemotherapy for the first 6 months of treatment, but moving it up to the very beginning.

Reference

FDA approves vorasidenib for grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation. News release. FDA. August 6, 2024. Accessed November 20, 2025. https://tinyurl.com/yc55dwex