More than 1.4 million people live with malignant brain tumors. Glioblastoma – the cancer that killed Senators John McCain and Ted Kennedy – is one of the deadliest. Nine of 10 diagnosed die within five years.

Glioblastoma knows no social, economic, or demographic barriers. It originates in brain tissue and rapidly disrupts brain function. With a median survival of 14 months, it claims the lives of most patients who are diagnosed. Despite years of coordinated efforts by the scientific community, experimental drugs continue to be ineffective.

These grim realities are the result of a drought in drug development for this patient population. Since 1982, only five drugs have been approved for brain cancer patients. In comparison, for lung cancer patients, this number exceeds 50. New drug development is costly, exceeding a $2 billion price tag from bench to bedside. Attaining FDA approval can take decades and, today, a new drug has less than an 8% chance of making it to market.

The funding and time needed for brain tumor drug development is ill-suited for today’s patients. As a neurosurgeon at the Barrow Neurological Institute and a clinical trialist at the Ivy Brain Tumor Center, these are some of the realities that my colleagues and I grapple with every day.

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Accelerated Phase 0/2 Clinical Trials
Despite the odds, the answers are out there, waiting for breakthroughs like we have seen with immunotherapy for melanoma and triple-therapy for HIV. The paucity of new drugs requires a bold, “high-risk, high-reward” approach to rapidly identify effective new experimental therapies, increase life expectancy, and pursue a cure.

Enabled by a $50 million grant from the Ben & Catherine Ivy Foundation, the Ivy Brain Tumor Center in Phoenix, Arizona aims to level the playing field and put time back on the side of patients in the fight of their lives. Unlike traditional clinical trials, accelerated Phase 0/2 clinical trials test new, experimental drug combinations on a patient-by-patient basis. Initial results are available in as few as seven days, allowing patients to expediently continue onto an advanced trial or switch to a different treatment.

We are focused on progress – not process – so we can quickly bring promising therapeutic options to patients living with brain tumors around the world. Because for us, this work is personal.

[1] Sanai N, Li J, Boerner J, Stark K, Wu J, Kim S, Derogatis A,
Phase 0 Trial of AZD1775 in First-Recurrence Glioblastoma Patients. Clin Cancer Res. 2018 Aug 15;24(16):3820-3828. doi: 10.1158/1078-0432.CCR-17-3348. Epub 2018 May 24. [Pubmed][Article]
[2]Bao X, Wu J, Sanai N, Li J. Determination of total and unbound ribociclib in human plasma and brain tumor tissues using liquid chromatography coupled with tandem mass spectrometry. J Pharm Biomed Anal. 2019 Mar 20;166:197-204. doi: 10.1016/j.jpba.2019.01.017. Epub 2019 Jan 11.[Pubmed][Article]