BRUSSELS - A new treatment protocol of 9 repurposed drugs for recurrent glioblastoma appears to be safe with good tolerability. These preliminary results are an important milestone in brain tumor research, internationally presented this weekend at the Annual Meeting of the Society for Neuro-Oncology in New Orleans, USA.

Trial outside the box

Current standard treatment of glioblastoma consist of surgery, radiation, and chemotherapy with temozolomide. Few are cured with this. Only 15-20% of patients are still alive 5 years after the first treatment. For decades multiple new experimental therapies have all failed to improve the outcome of patients when glioblastoma recurs, as it does in 99% of the patients.

In 2013, German neurosurgeon Marc-Eric Halatsch, in collaboration with U.S. psychiatrist Richard Kast, had the non-conventional idea to add nine drugs to the treatment. These drugs were already marketed but used for illnesses other than cancer. They noted that these added nine drugs had attributes that experimental data indicated might inhibit glioblastoma’s growth. The added nine drugs had low side effects and were well-known to most doctors. Maintenance of good quality of life was the highest importance in Drs. Halatsch and Kast’s thought in choosing the repurposed drugs pathway.

Five years ago, Kast and Halatsch applied for funding at the Anticancer Fund to bring their innovative CUSP9 idea (Coordinated Undermining of Survival Paths by 9 Repurposed Drugs) to a clinical trial. Today, the version 3 of the protocol (CUSP9v3) has 10 patients included in its trial coordinated by the Ulm University Hospital in Germany, financially supported by the Anticancer Fund.

“We are aware that combinating nine drugs with standard treatment is highly unusual. We strongly believe drug repurposing has high potential, as do combination therapies. We support this research because we believe that ideas like that of Halatsch and Kast can work. You get results when you push the limit and explore potential treatments outside the box”, explains medical director Guy Buyens.

Preliminary results

After regulatory approval, between 2016 and October 2018, ten patients with glioblastoma recurrence were included in the CUSP9 proof-of-concept trial. The first results at 12-week indicated little toxicity, maintenance of good quality of life, and initial hints of effectiveness, although full results will only be available in 2019.

“Drug repurposing is a valuable approach for speeding up cancer research. Existing non-cancer drugs have the advantage of being well-documented, easily available, affordable, and having a well-known safety profile. The downside is that they are mostly generic and therefore lack a commercial incentive for the pharma industry to pursue them for cancer treatments. Non-profit support and public funding for this type of research is crucial in exploring these potential treatment advances”, explains Lydie Meheus, CEO of the Anticancer Fund.

The Anticancer Fund promotes the research of cancer treatment options where needs are high. Drug repurposing is one of the focus areas of the clinical research the fund facilitates and supports. Gauthier Bouche, M.D., director of clinical research at the Anticancer Fund, is co-author of the results published at the SNO congress in New Orleans.

More info on CUSP9 can be found here.

Read more on drug repurposing here.