Phase 1/2a study on Lisavanbulin Published in Journal Cell Reports Medicine

In June 2025, Cell Reports Medicine published the results of an international clinical trial investigating Lisavanbulin, a new experimental pill for people with glioblastoma and other high-grade gliomas. Glioblastoma is an aggressive brain cancer that almost always returns after initial treatment with the current standard of care, comprised of surgery, radiation, and chemotherapy. Once it recurs, treatment options are limited and outcomes are often poor. This study set out to determine the safest daily dose of Lisavanbulin, to see whether it could help control tumor growth. 

Lisavanbulin is taken by mouth and converted inside the body to its active form, Avanbulin. Avanbulin works by disrupting microtubules, tiny structures that act like scaffolding inside cells and are essential for cell division. When microtubules are disturbed, cancer cells make fatal mistakes as they try to divide, ultimately leading to their death. A particularly important feature of Lisavanbulin is that it can cross the blood–brain barrier, the natural defense system that blocks many drugs from reaching brain tumors. This gives it a potential advantage over many existing chemotherapies for glioblastoma.

The study found that patients with glioblastoma or other high-grade gliomas could tolerate up to 30 milligrams per day. When looking at how well the drug worked, the results were mixed. In the first phase of the trial, which included glioblastoma patients regardless of tumor biology, one person had a complete response, meaning their tumor disappeared on scans. Another had a partial response, with the tumor shrinking by more than 90 percent. Seven others experienced stable disease for a time. Two patients remained on Lisavanbulin for more than four years with ongoing benefit—an exceptional outcome in this disease. However, most patients did not have significant tumor shrinkage and eventually saw their cancer progress.

For patients, the takeaway is that Lisavanbulin appears safe at the recommended dose and can lead to remarkable, long-lasting benefit in a small number of cases. The challenge is finding those patients ahead of time. In an important development for the future of this therapy, the Glioblastoma Foundation recently acquired Lisavanbulin. This means that further research and development will be driven by an organization fully dedicated to improving outcomes for glioblastoma patients. Future studies will focus on finding a gene signature for patients who will respond to Lisavanbulin.  If validated, it could allow doctors to offer Lisavanbulin only to patients most likely to respond, making treatment an effective option for those that might respond.

While Lisavanbulin is not a cure and will not work for everyone, the trial has shown that with the right testing, it may become a powerful tool for certain patients. As ongoing studies progress, there is hope that this drug could offer longer, better-quality lives for some patients facing this challenging disease.

Lisavanbulin is a promising drug therapy being developed by the Glioblastoma Foundation for a subset of glioblastoma patients. You can support the continued research and development of Lisavanbulin by making a donation to the Glioblastoma Foundation. Your contribution helps accelerate progress toward more personalized and effective treatments for glioblastoma—bringing hope to patients and families affected by this devastating disease.

Donations to the Glioblastoma Foundation go directly to fund the development of novel drug therapies for glioblastoma. We’re committed to funding new, more effective therapies to extend survival for patients diagnosed with glioblastoma and improve the standard of care for glioblastoma.

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The current standard of care for glioblastoma consisting of radiation and chemotherapy is ineffective.

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