Advances in understanding and drug development for ALK2/ACVR1 gain of function mutations in Diffuse Intrinsic Pontine Glioma (DIPG)
Wong, Jong Fu;
Brennan, Paul E;
Edwards, Aled M;
Bullock, Alex N
A poster outlining the program being undertaken within the Structural Genomics Consortium to co-develop drugs for both Diffuse Intrinsic Pontine Glioma (DIPG) and Fibrodysplasia Ossificans Progressiva (FOP).
The following topics are covered: prevalency of ALK2/ACVR1 mutations in DIPG, the crystal structure of the ALK2-FKBP12 complex, the increased BMP pathway activity in ALK2 mutants, the M4K Pharma ALK2 inhibitor development workflow and the M4K mission statement "Affordable Meds 4 Kids".
Funding Acknowledgment: The SGC is a registered charity (number 1097737) that receives funds from AbbVie, Bayer Pharma AG, Boehringer Ingelheim, Canada Foundation for Innovation, Eshelman Institute for Innovation, Genome Canada through Ontario Genomics Institute [OGI-055], Innovative Medicines Initiative (EU/EFPIA) [ULTRA-DD grant no. 115766], Janssen, Merck KGaA, Darmstadt, Germany, MSD, Novartis Pharma AG, Ontario Ministry of Research, Innovation and Science (MRIS), Pfizer, São Paulo Research Foundation-FAPESP, Takeda, and Wellcome.