Researchers at the University of Cincinnati Gardner Neuroscience Institute’s Brain Tumor Center reported on May 4 that they are the first in the world to achieve complete remission of a rare pituitary cancer using a novel immunotherapy treatment.
The development is significant because aggressive pituitary tumors, known as pituitary carcinomas and aggressive pituitary adenomas, have historically responded poorly to standard treatments such as surgery, radiation, and chemotherapy. The UC team is now urging neurosurgical and neuro-oncology communities worldwide to use tumor genomic testing for these cancers to guide consideration for immunotherapy as a potential first-line treatment.
The findings were published on May 1 in Surgical Neurology International. Jonathan Forbes, MD, associate professor and residency program director in the Department of Neurosurgery at UC’s College of Medicine, said: “To date, immunotherapy has revolutionized the care of patients with metastatic melanoma and non-small cell lung cancer, but until recently, they hadn’t been available for patients with aggressive pituitary cancer.”
In their case study, Forbes and colleagues described a patient who initially presented with visual loss due to a pituitary tumor. After surgical removal restored vision completely, pathology revealed an aggressive tumor type. Despite post-operative radiation therapy keeping the patient tumor-free for about a year, recurrence occurred. Standard chemotherapy failed to control further growth. As symptoms worsened and hospice was considered, genomic testing identified a mismatch repair mutation that made the patient eligible for an appropriate clinical trial involving immunotherapy.
Forbes said: “Following a thorough review of tumor genomics, we were able to get the patient signed up for the right clinical trial. Using that scientifically-informed regimen, he was able to clear the cancer. The double vision completely resolved on the immunotherapy regimen. It was the first in the world to have a complete response, meaning the cancer completely vanished and did not return after medication was stopped.”
Since publishing their initial report on this case, three more patients worldwide with similar tumors have also achieved complete responses after receiving immunotherapy guided by genomic profiling. Forbes said it should become standard practice to test each pituitary tumor’s DNA so that those with certain mutations can access these medications immediately: “If the tumor DNA fits a particular profile,” he said,“patients should be permitted to have expanded access to these potentially life-saving medications immediately.”
Looking ahead, UC researchers plan trials using this approach—combining genomics-driven selection with immunotherapies—for glioblastoma treatment as well. A planned Phase 1 trial will incorporate navigated focused ultrasound technology designed to help immune therapies cross into brain tissue more effectively.
“This case report reinforces that there are major discoveries happening here at our Cancer Center in Cincinnati,” Forbes said.“It is incredibly important for patients in our region to have unencumbered access to innovative and scientifically informed clinical trials.”
