Related Experiment Videos
A Collaborative Model for Accelerating the Discovery and Translation of Cancer Therapies
Ophélia Maertens1,2,3, Mila E McCurrach4,5, Benjamin S Braun6
1Genetics Division, Department of Medicine, Brigham and Women's Hospital, Boston, Massachusetts.
Abstract:
Preclinical studies using genetically engineered mouse models (GEMM) have the potential to expedite the development of effective new therapies; however, they are not routinely integrated into drug development pipelines. GEMMs may be particularly valuable for investigating treatments for less common cancers, which frequently lack alternative faithful models. Here, we describe a multicenter cooperative group that has successfully leveraged the expertise and resources from philanthropic foundations, academia, and industry to advance therapeutic discovery and translation using GEMMs as a preclinical platform. This effort, known as the Neurofibromatosis Preclinical Consortium (NFPC), was established to accelerate new treatments for tumors associated with neurofibromatosis type 1 (NF1). At its inception, there were no effective treatments for NF1 and few promising approaches on the horizon. Since 2008, participating laboratories have conducted 95 preclinical trials of 38 drugs or combinations through collaborations with 18 pharmaceutical companies. Importantly, these studies have identified 13 therapeutic targets, which have inspired 16 clinical trials. This review outlines the opportunities and challenges of building this type of consortium and highlights how it can accelerate clinical translation. We believe that this strategy of foundation-academic-industry partnering is generally applicable to many diseases and has the potential to markedly improve the success of therapeutic development. Cancer Res; 77(21); 5706-11. ©2017 AACR.
Insights
Genetically engineered mouse models (GEMM) accelerate cancer therapy development. A consortium model involving foundations, academia, and industry successfully identified therapeutic targets for neurofibromatosis type 1 (NF1) tumors, leading to clinical trials.
Area of Science:
- Oncology
- Translational Medicine
- Preclinical Research
Background:
- Genetically engineered mouse models (GEMM) are valuable preclinical tools, especially for rare cancers, but are underutilized in drug development.
- Neurofibromatosis type 1 (NF1) and its associated tumors historically lacked effective treatments and robust preclinical models.
Purpose of the Study:
- To describe the establishment and success of the Neurofibromatosis Preclinical Consortium (NFPC) as a model for therapeutic development using GEMMs.
- To highlight the collaborative foundation-academic-industry approach to accelerate drug discovery and translation for NF1.
Main Methods:
- Established a multicenter cooperative group (NFPC) integrating expertise from philanthropic foundations, academia, and industry.
- Conducted extensive preclinical trials of drugs and drug combinations using GEMMs as a platform.
- Facilitated collaborations with pharmaceutical companies for drug testing and target identification.
Main Results:
- The NFPC conducted 95 preclinical trials involving 38 drugs/combinations since 2008.
- Identified 13 novel therapeutic targets for NF1-associated tumors.
- These findings have directly inspired 16 clinical trials.
Conclusions:
- The foundation-academic-industry consortium model effectively accelerates therapeutic discovery and clinical translation for rare diseases like NF1.
- This collaborative strategy holds significant potential for improving the success rate of drug development across various diseases.
- Leveraging GEMMs within a structured consortium is a powerful approach to advance oncology treatments.