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By Ashley Sumrall, MD, FACP

When it comes to brain tumors, H3 K27M mutant glioma is the worst of the worst. It wraps around the brain like a spider web, rarely responds to chemotherapy and is considered universally fatal. But an experimental drug, called ONC201, is showing unprecedented results: Six of the first 14 clinical trial participants saw their tumors regress, including one woman whose tumor shrank by 96 percent.

Levine Cancer Institute (LCI) is the only site in the Southeast where H3 K27M glioma patients can get access to this remarkable therapy.

Killing Brain Tumor Cells Without Side Effects

ONC201 targets the DRD2 dopamine receptor, which is overexpressed in glioblastoma cells. The drug aims to induce cell death – without harming healthy cells – by shutting down signaling pathways that help tumors grow.

ONC201 is being applied to several cancers, and Massachusetts General Hospital and Dana-Farber Cancer Institute launched clinical trials for recurrent glioblastoma in 2016.

Remarkable Regression for H3K27M Glioma

An abstract presented at the 2018 ASCO Annual Meeting revealed unprecedented results in adults and children with gliomas that tested positive for the H3 K27M mutation. Imaging showed that six of the first 14 clinical trial participants saw their tumors regress. These patients had few – if any – side effects from ONC201.

Five of those six patients had thalamic glioma. Two patients in this subgroup saw complete regressions of their thalamic lesions while another had a 30 percent regression.

Another patient started taking ONC201 after repeat surgery to remove the tumor – and remained disease-free 14 months later. And then there’s the woman with recurrent midline H3 K27M glioma whose tumor almost completely shrank away (96 percent) after nearly two years of ONC201.

Launching a Trial in Just 10 Days

When a man with H3 K27M glioma came to LCI in 2017, his tumor kept growing despite chemotherapy and radiation. It would’ve been easy to transition him to hospice. Instead, we brought ONC201 here as fast as we could.

Thanks to the FDA’s compassionate use program, we opened a single-patient clinical trial in just 10 days. Unfortunately, ONC201 didn’t help this patient. But his willingness to pursue clinical trials opened the door for us to become the second site in the ONC013 trial in the US, which offers ONC201 to adults with recurrent, H3 K27M glioma.

LCI can enroll up to 17 patients in ONC013 and we signed up our second patient in June 2018 – a young woman with a large tumor wrapped around the junction of the brain and spinal cord. A month later, her tumor had stopped growing. We’re hopeful that the tumor will start shrinking – it usually takes four to six months for ONC201 to trigger regression.

The LCI Difference

ONC013 is just the latest example of LCI’s commitment to delivering every opportunity for a good outcome, even when patients are considered terminally ill.

We see more patients with primary brain tumors than almost any other center in the Southeast, and our volumes are growing as more patients travel here from across the region. These patients are drawn by our renowned brain cancer team, and by our team approach that brings together neuro-oncologists like myself, medical oncologists, radiation oncologists, surgeons and other key specialists to evaluate each patient and develop the best possible treatment plan, and give them the best chance at a longer, healthier life.

For more information or to refer a patient, please call our general line at 980-442-2358 or reach a trial nurse at 980-442-2358.

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