Pilot study tests ALS drug to treat chemo brain in cancer patients

First U.S. clinical trial to explore riluzole for cancer-related cognitive impairment

Orange, Calif. – The UCI Health Chao Family Comprehensive Cancer Center has launched the first U.S. clinical trial to investigate the impact of riluzole therapy on circulating levels of a key brain protein in cancer patients who have experienced treatment-related cognitive impairment, also known as “chemo brain” or “brain fog.”

The phase 2a clinical trial is also designed to evaluate the effect of riluzole on cognitive function in these patients over time, said principal investigator Alexandre Chan, PharmD, MPH, founding chair and professor of the UC Irvine Department of Clinical Pharmacy Practice. He expects to enroll 39 patients in the randomized, double-blinded, placebo-controlled pilot study, which was originally focused on breast cancer survivors.

Riluzole was originally approved by the FDA in 1995 to treat patients with amyotrophic lateral sclerosis (ALS) patients based on studies showing the drug increased circulating levels of brain-derived neuropathic factor, or BDNF. Produced in the brain and nervous system, BDNF regulates the survival and growth of neurons and processes related to learning and memory.

Conventional cancer treatments have been linked to significant declines in BDNF levels, prompting Chan’s interest in studying riluzole’s potential to boost BDNF production and mitigate chemo brain.

“Our hypothesis is that if we can elevate BDNF and restore neuronal function in our patients, it would also help these patients recover from cognitive changes they may have experienced from their cancer treatment,” he said.

In 2023, Chan and neuroscientist Munjal Acharya, PhD, an associate professor of anatomy and neurobiology at the UC Irvine School of Medicine, were awarded a five-year, $2.4 million grant by the National Cancer Institute to study whether augmenting BDNF could alleviate cancer-related cognitive impairment.

For the current clinical trial, Chan received seed funding from the School of Pharmacy & Pharmaceutical Sciences, the School of Medicine and the UC Irvine Anti-Cancer Challenge, the cancer center’s annual fundraiser whose proceeds go directly to support promising pilot projects.

The study is open to cancer patients ages 18 and older who have been treated with chemotherapy, radiotherapy, surgery and/or other interventions, and who experienced worsened cognitive function during or after treatment.

Participants take a pill orally twice a day for eight weeks. Neither the investigators nor the participants know who is receiving the drug or a placebo until after the clinical trial concludes and the results are studied.

“Chemo brain is a common thing I see in clinic,” said Chan. “I work very closely with our medical oncologists, and as an oncology pharmacist, I provide patients with advice on how to manage the physical and cognitive side effects of their treatment. My focus is on helping patients feel better.

‘It’s not just about curing their cancer. If we want them to really survive, it’s also about bringing them back to where they were, to recover from these cognitive changes.”

For more information or to participate in the clinical trial, email ucstudy@hs.uci.edu or call 1-877-UC-Study (827-8839).

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The UCI Health Chao Family Comprehensive Cancer Center is one of only 57 National Cancer Institute-designated U.S. comprehensive cancer centers and the only one based in Orange County, the sixth most populous county in the nation. A research powerhouse, the cancer center brings together basic and translational scientists with clinician investigators to drive discoveries through the pipeline into the clinical arena. The center treats more patients with cancer — and more complex cases — than any other healthcare provider in the region, with more than 80,000 outpatient visits and 65,000 infusion treatments annually at practice sites in Orange, Irvine, Yorba Linda, Newport Beach, Costa Mesa and Laguna Hills. The center also offers more than 500 clinical trials, including the region’s largest portfolio of early-phase and investigator-initiated trials. It has the only adult bone marrow transplant program in Orange County, performing more than 100 transplants a year and enabling critically ill patients to receive life-saving care close to home. It is also one of the few U.S. programs to provide transplants for debilitating autoimmune diseases such as multiple sclerosis. With the opening of its second clinical hub in Irvine in July 2024, the cancer center has tripled its space to treat cancer patients and conduct novel studies aimed at bringing an end to cancer.

UCI Health, one of California’s largest academic health systems, is the clinical enterprise of the University of California, Irvine. The 1,317-bed system comprises its main campus UCI Medical Center, its flagship hospital in Orange, Calif., the UCI Health — Irvine medical campus, four hospitals and affiliated physicians of the UCI Health Community Network in Orange and Los Angeles counties and a network of ambulatory care centers across the region. UCI Medical Center provides tertiary and quaternary care and is home to the only Orange County-based National Cancer Institute-designated comprehensive cancer center, high-risk perinatal/neonatal program and American College of Surgeons-verified Level I adult and Level II pediatric trauma center, gold level 1 geriatric emergency department and regional burn center. Powered by UC Irvine, UCI Health serves nearly 4 million people in Orange County, western Riverside County and southeast Los Angeles County. Follow us on Facebook, Instagram, LinkedIn and X (formerly Twitter).

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