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Art Sullivan, a 38-year-old Ironman athlete, and Josh Lehman, 48, who works at the University of Rochester, are connected by an unlikely foe: a type of brain tumor called an astrocytoma that tends to afflict younger adults. They are also benefiting from the first new treatment for this cancer in decades—and it's changing their lives.

"The new drug has been mentally transformative, " said Lehman, who was first diagnosed with brain cancer 15 years ago, went into a long remission, and then saw it return in 2024.

"I was prepared in a positive way to go down the chemo-radiation path, " Lehman said, explaining that in the past the only treatment he required was surgery. "But this was such a blessing. What are the chances? I felt so fortunate, and although it was a new drug, I had a lot of trust because of my care team at Wilmot." Lehman serves as director of human resources operations for the university.

The U.S Food and Drug Administration approved vorasidenib to treat astrocytoma in August 2024. The drug is a daily pill that targets a pathway in the brain where this cancer grows. The pill has few or no side effects for most people, aside from fertility concerns.

Leadership in advancing brain cancer care

Wilmot Cancer Institute neuro-oncologist Nimish Mohile, MD, has 30 patients using the new medication, including Lehman and Sullivan.

In 2021, Mohile was part of the expert panel who wrote the first-ever national guidelines for treating brain cancer, and this year Mohile and the national group published new guidance in the Journal of Clinical Oncology about the practice-changing research supporting vorasidenib for some types of brain tumors.

Astrocytoma tumors can range from low-grade types—which is what Lehman and Sullivan have–to aggressive, high-grade gliomas, such as glioblastoma suffered by Senator John McCain and Beau Biden, son of the former president. People can live many years with low-grade tumors. That's why the new drug with fewer side effects than chemotherapy and radiation is important: It could make survivorship easier.

"When radiation becomes a core part of brain cancer treatment, eventually it causes cognitive issues—memory and emotions, for example—which interferes with work and relationships, and in rare cases puts patients at risk for later cancers, " Mohile said. "So, whatever we can do to delay or avoid radiation therapy to the brain, especially in younger people, is a good thing."

Brain cancer symptoms and treatment

Art Sullivan's only symptom of brain cancer was a seizure that came unexpectedly in December of 2023, while he was watching cartoons with his two little boys. An ambulance rushed him to the hospital. After stressful days of waiting for test results, he got official confirmation of a grade 2 astrocytoma, which surgeons quickly removed.

A longtime endurance athlete, Sullivan was fit prior to surgery and ran the Buffalo Marathon in May 2024, four months to the day after his craniotomy.

Turning the emotional corner has been more difficult, he said, but being under the care of Mohile and receiving a next-generation treatment has helped. He has been taking vorasidenib for a year with no side effects.

"It's pretty surreal coming to the cancer center the first couple of times, " Sullivan said. "Big change. Big adjustment, and I'm still working on it. But I'm finally getting my feet under me."

FDA approval of vorasidenib for individuals aged 12 years or older came after 331 patients took part in a clinical trial that measured how long they remained stable before needing to start more traditional chemotherapy and radiation. Results showed that compared to a placebo, vorasidenib lengthened the period before cancer returned and extended the time until patients needed to switch to a new therapy.

Aside from this, most brain cancer treatments used today were developed in the 1990s.

The slow progress, Mohile said, is primarily because brain cancer of all types is rare. The number of patients who get rare cancers is so small that it's difficult to obtain the data necessary for scientific advances and drug approvals.

Community awareness of brain cancer treatment options

Wilmot serves a 27-county region across western and central New York, which is larger than Vermont and New Hampshire combined. One of the challenges, particularly in smaller towns and rural areas, is making sure that community doctors and patients know about the latest cancer treatments available. The nationally published guidelines can be a useful tool.

"We have huge neuro-oncology deserts, " Mohile said. "If a medical oncologist sees 200 new patients, maybe one of them will have brain cancer, and so they are not necessarily up to date on the latest surgical techniques or the most modern treatments. That's why guidelines are important. When they have that patient in front of them, they can get quick access to knowing: what are my options?"

"Information on new treatments also empowers patients to have more understanding and to ask their doctors about all treatments, " he said.

Although there is excitement around vorasidenib, it is not a cure. "It's a big step forward in a rare disease that's hard to study, " said Mohile, who is also the Ann Aresty Camhi Professor in Neurology at the University of Rochester.

Brain cancer research

Scientists are continually working to develop options for patients. In addition to vorasidenib, more good news came this month when the FDA approved a second new treatment for another rare type of brain tumor: the drug dordaviprone treats gliomas that have a very specific gene mutation.

Several Wilmot adult patients and children participated in clinical trials in past years that evaluated this drug, dordaviprone—giving them access to a groundbreaking treatment years before it was widely available. David Korones, MD, led those studies. Mohile also noted that a person from the United Kingdom came to Wilmot to get access to the drug through the clinical study, and a local patient has been taking it for years with success.

"We're starting to see progress with targeted therapies in the field of neuro-oncology, " Mohile said.

Another important initiative is to expand the currently available cellular treatment called CAR T-cell therapy. Oncologists use it mostly to treat blood cancers; CAR T-cell therapy harnesses a patient's own healthy immune cells to attack cancer cells in diseases such as leukemia and lymphoma.

But now researchers are investigating CAR T-cell therapy for solid tumors such as brain, breast, and lung cancer—and Wilmot recently joined with four other research institutions in New York state to advance this effort more quickly.

Kerry O'Banion, M.D., Ph.D., a professor at the UR Del Monte Institute for Neuroscience and a Wilmot member, is also investigating how to protect the brain during radiation therapy for cancer. He focuses on stopping damage to the nerve cells during radiation exposure as the brain attempts to rewire itself.

In the past five years, Mohile added, scientists have gained a clearer picture of molecular targets that could tamp down brain cancer. This allows oncologists to classify brain tumors by their mutations and then try to personalize treatment accordingly.

People with astrocytoma can live up to 20 years, and therefore the goal is to find a new intervention when it's needed.

"You constantly want to be moving ahead, " Mohile said.

More information: Jaishri Blakeley et al, Therapy for Diffuse Astrocytic and Oligodendroglial Tumors in Adults: ASCO-SNO Guideline Rapid Recommendation Update, Journal of Clinical Oncology (2025). DOI: 10.1200/JCO-25-00250  Journal information: Journal of Clinical Oncology