NF Preclinical Consortium

The NF Preclinical Consortium (NFPC)

In 2013, NTAP partnered with the Children’s Tumor Foundation (CTF) to support a network of laboratories that evaluate therapeutic agents in relevant animal models. These included testing the efficacy, tolerability, pharmacokinetics and pharmacodynamics of agents already developed or in development for other conditions in genetically engineered mouse models (GEMMs) with NF1. The laboratories in this consortium were the Clapp laboratory at Indiana University, and the Ratner laboratory at Cincinnati Children’s Hospital. (Read their paper: A Collaborative Model for Accelerating the Discovery and Translation of Cancer Therapies)

Drs. Clapp and Ratner have developed complementary GEMMs with NF1-associated plexiform neurofibroma. Each laboratory had unique and complementary reproducible measures of efficacy for plexiform neurofibromas. Many of these studies performed across the Clapp and Ratner laboratories formed the foundation of the later clinical successes with MEK inhibitors and multi-tyrosine kinase inhibitors.

Concurrently, the laboratories of Drs. Shannon (UCSF) and Cichowski (Brigham and Womens) focused on  juvenile myelomonocytic leukemia and malignant peripheral nerve sheath tumors with support from CTF. Through the life of the NFPC, all stakeholders were focused on sharing techniques, reagents, ideas and networks to test the most relevant therapies in the most relevant models as efficiently and accurately as possible.

During the seven years of the NFPC, 95 preclinical trials of 38 drugs or combinations were completed with significant collaboration across 18 pharmaceutical companies. This diligent and expert consortium yielded much of the critical data leading to the revolutions in clinical translational research for NF1 tumors the field is experiencing today.