Clinical Perspectives

Novel Treatment for Brain Cancer Using Immune Checkpoint Inhibitors

February 23, 2021

Brain Cancer Immunotherapy

Brain metastasis

When it comes to brain cancer treatment, a personalized medicine approach is key and understanding who will benefit from new treatments and who should avoid them if they are going to have a bad side effect. And, immunotherapy is at the forefront of novel therapy for patients with brain cancer.

Scientists at Moffitt Cancer Center are working to understand how to turn on the immune system and focus it on the brain. Our researchers found that combination therapy with ipilimumab and nivolumab in patients with brain metastases from melanoma was extremely effective in some patients. Many of whom are alive, doing extremely well and are enjoying life five years post-treatment.  

Dr. Peter Forsyth
Dr. Peter Forsyth

For the first time in decades, we could see what was possible in brain tumors and how effective treatments could be if we could just figure out the best treatment.

It’s the little things like timing or sequencing. In our experiments with test tubes and mice, immunotherapies and targeted therapies such as BRAF MEK inhibitors are performing very well. Somehow the immunotherapy helped to first set the stage for targeted therapies to work better. For example, the Phase IB Study of Avelumab with Radiotherapy in Patients with Leptomeningeal Disease (MCC 19648), the primary goal to establish the safety of the combination of Avelumab and WBRT in patients with LMDz and perform exploratory analysis of the patient survival rate.

"It is very helpful for us to understand the clinical setting and provide a direct and fast pipeline to bring these discoveries to the clinic to make a difference right away," said Dr. Peter Forsyth.  


Dr. Solmaz Sahebjam
Dr. Solmaz Sahebjam

In gliomas brain metastasis (GBM), using a single immune checkpoint inhibitor is probably not very effective and the reasons for this are not clear. Although it’s likely immunosuppressive cells in the tumor microenvironment that make a tumor "cold" and less likely to respond to the treatment. Dr. Solmaz Sahebjam, a medical oncologist and head of the Early Phase Drug Development Program at Moffitt Cancer Center, is tackling this problem head-on. "We know that using a single immunotherapy in GBMs is not going to be enough to attack and kill these tumors. Therefore, we are combing several immunotherapies approaches together. We think the early results are very exciting and are much better than expected,” said Dr. Sahebjam. 

Dr. Michael Vogelbaum
Dr. Michael Vogelbaum

Our neurosurgeon, Dr. Michael Vogelbaum is leading a study to examine the role of a special kind of cell in GBMs called a MDSC that gets into the tumor and keeps it "cold." "We think that these cells are really keeping GBMs 'cold and quiet' so that they are below the body's radar detection system. We are confident that we can find drugs to deliver into the GBM to silence these cells and allow the body’s own defense systems to finally recognize and kill the GBM cells," said Dr. Vogelbaum, MD, PhD, program leader and chief of neurosurgery of Neuro-Oncology.

"Seeing patients doing so well changed my life as well as the lives of patients. It’s amazing to bring together a group of world-renown scientists to develop novel therapies for brain cancer and see tremendous results," said Dr. Forsyth.