Current drug development and trial designs in neuro-oncology: report from the first American Society of Clinical Oncology and Society for Neuro-Oncology Clinical Trials Conference.

Successful drug development for people with cancers of the CNS has been challenging. There are multiple barriers to successful drug development including biological factors, rarity of the disease, and ineffective use of clinical trials. Based upon a series of presentations at the First Central Nervous System Clinical Trials Conference hosted by the American Society of Clinical Oncology and the Society for Neuro-Oncology, we provide an overview on drug development and novel trial designs in neuro-oncology. This Review discusses the challenges of therapeutic development in neuro-oncology and proposes strategies to improve the drug discovery process by enriching the pipeline of promising therapies, optimising trial design, incorporating biomarkers, using external data, and maximising efficacy and reproducibility of clinical trials.

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Declaration of interests RR has received research support from Project Data Sphere and personal fees from St Lucia Consulting, outside the submitted work;and has been supported by the Joint Center for Radiation Therapy Foundation Grant. CKA has received research support from Puma Biotechnology, Lilly, Merck, Seattle Genetics, Nektar, Tesaro, G1-Therapeutics, Zion Pharmaceuticals, Novartis Pharmaceuticals, Pfizer, AstraZeneca, and Elucida; served as consultant for Genentech, Eisai, IPSEN, Seattle Genetics, AstraZeneca, Novartis Pharmaceuticals, Immunomedics, Elucida, and Athenex; and received royalties from UpToDate, Jones & Bartlett. HAT received personal fees from Novartis Pharmaceuticals; grants and personal fees from Bristol Myers Squibb, Roche, and Genentech; and grants from Merck and Celegene, outside the submitted work. MM reports personal fees from KaryoPharm, Mevio, Zapprx, Sapience, and Xoft outside the submitted work. He reports participation in the board of directors for Oncoceutics and stock ownership in Chimerix. PYW has received research support from AstraZeneca, BeiGene, Celgene, Chimerix, Eli Lily, Genentech, Roche, Kazia, MediciNova, Merck, Novartis Pharmaceuticals, Nuvation Bio, Puma, Servier, Vascular Biogenics, and Variation Biotechnologies Vaccines; and is on advisory boards for AstraZeneca, Bayer, Black Diamond, Boehringer Ingelheim, Boston Pharmaceuticals, Celularity, Chimerix, Day One Bio, Genenta, GlaxoSmithKline, Karyopharm, Merck, Mundipharma, Novartis Pharmaceuticals, Novocure, Nuvation Bio, Prelude Therapeutics, Sapience, Servier, Sagimet, Vascular Biogenics, and VBI Vaccines. JdG reports grant or research support from Sanofi-Aventis, AstraZeneca, EMD-Serono, Eli Lilly, Novartis, Deciphera Pharmaceuticals, and Mundipharma; paid consultancies from Celldex, Deciphera Pharmaceuticals, AbbVie, FivePrime Therapeutics, GW Pharmaceuticals, Carthera, Eli Lilly, Kadmon, Boston Biomedical, Taiho Pharmaceuticals, Kairos Venture Investments, Syneos Health, Monteris, Agios, Mundipharma Research, GenomiCare, Blue Earth Diagnostics, Del Mar Pharmaceuticals, Insightec, Voyager Therapeutics, Merck, Tocagen, Bioasis Technologies, and ResTORbio; advisory board membership for Genentech, Celldex, Foundation Medicine, Novogen, Deciphera, AstraZeneca, Insys Therapeutics, Merck, Eli Lilly, Novella Clinical, Karyopharm Therapeutics, Blue Earth Diagnostics, Kiyatec, Vanquish Oncology, Orsenix, Insightec, Prelude Therapeutics, Debiopharm Therapeutics, and Janssen Global Services; data safety monitoring board membership for VBL Therapeutics (VB111; glioblastoma), Novella (ICT-107; glioblastoma), and VBI Vaccines (VBI-1901; glioblastoma); stock ownership in Ziopharm Oncology and Gilead; and company employment (spouse) in Ziopharm Oncology. EG has received honoraria for advisory board participation from Kiyatec (personal compensation), and Karyopharm Therapetuics for data safety and monitoring board participation (compensation to employer). Her institution has received grant funding from Servier Pharmaceuticals, Celgene, MedImmune, and Tracon Pharmaceuticals; and she has been supported by the National Cancer Institute of the National Institutes of Health (NIH) under award numbers UG1CA189823 (Alliance for Clinical Trials in Oncology), R01 CA258239, U19 CA264362, and The Ben and Catherine Ivy Foundation. MK reports research funding paid to their institution from BMS, AbbVie, Biontech, Astellas, Celldex, Daiichi Sankyo, and CNS pharmaceuticals; and honoraria from George Clinical, Voyager Therapeutics, Johnson & Johnson, and the Jax lab for genomic research; and is supported by NIH grants (U01-NS090284-05, U19-CA264385-01, P50CA190991-08, P01-CA225622-04, and R01-CA235612-03). M-YCP has been supported by the National Cancer Institute of the NIH under award number U10 CA180822 (NRG Oncology—Statistics and Data Management Center). All other authors declare no competing interests.