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

Rifaquat Rahman; Mei-Yin C Polley; Laura Alder; Priscilla K Brastianos; Carey K Anders; Hussein A Tawbi; Minesh Mehta; Patrick Y Wen; Susan Geyer; John de Groot; Gelareh Zadeh; Steven Piantadosi; Evanthia Galanis; Mustafa Khasraw

doi:10.1016/S1470-2045(23)00005-0

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

Lancet Oncol. 2023 Apr;24(4):e161-e171. doi: 10.1016/S1470-2045(23)00005-0.

Authors

Affiliations

¹ Department of Radiation Oncology, Dana-Farber Cancer Institute, Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA. Electronic address: rrahman@bwh.harvard.edu.
² Department of Public Health Sciences, University of Chicago, Chicago, IL, USA.
³ Duke Cancer Institute, Duke University Medical Center, Durham, NC, USA.
⁴ Massachusetts General Hospital, Dana-Farber Cancer Institute, Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA; Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
⁵ MD Anderson Cancer Center, Houston, TX, USA.
⁶ Miami Cancer Institute, Baptist Hospital, Miami, FL, USA.
⁷ Centre for Neuro-Oncology, Dana-Farber Cancer Institute, Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA; Center for Neuro-Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA, USA.
⁸ Department of Quantitative Health Sciences, Mayo Clinic, Rochester, MN, USA.
⁹ University of California San Francisco Brain Tumor Center, San Francisco, CA, USA.
¹⁰ Department of Neurological Surgery University of Toronto, Toronto, ON, Canada.
¹¹ Department of Surgery, Dana-Farber Cancer Institute, Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA.
¹² Department of Oncology, Mayo Clinic Comprehensive Cancer Center, Mayo Clinic, Rochester, MN, USA.

Abstract

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.

Publication types

Review
Research Support, N.I.H., Extramural

MeSH terms

Drug Development
Humans
Medical Oncology
Neoplasms* / drug therapy
Reproducibility of Results
Societies, Medical

Abstract

Publication types

MeSH terms

Grants and funding