Projects we fund

Overview

Diffuse intrinsic pontine glioma (DIPG) is a childhood brain cancer with no effective treatment. Currently, more than 90% of children with DIPG die within 18 months of it being diagnosed. Professor Chris Jones and colleagues at the Institute of Cancer Research recently discovered that the tumour cells in around a quarter of DIPG patients have mutations in a gene known as ACVR1. These mutations in ACVR1 have never been seen in any other form of cancer, but are the same as those found in every cell of the body in patients with a condition called fibrodysplasia ossificans progressiva (FOP). This is a debilitating condition where the soft tissue turns to bone during childhood. The link between FOP and DIPG has never been reported before and highlights the important overlap between processes which control normal tissue development and those which control cancer. The hope is that ACVR1 represents a new target for developing better treatments for children with DIPG.

What difference will this project make?

We don’t have any effective treatment at the moment, and this project is the strongest hope we’ve had for a new treatment for some time. Professor Chris and his team will investigate whether treating DIPG cells with specific drugs designed to inhibit the action of ACVR1 will also stop the cancer cells growing. They’ll test this both ‘in vitro’, (in laboratory culture dishes), and ‘in vivo’ (meaning in a living body), using mice. They’ll test a collection of DIPG cells with and without the ACVR1 mutation, and try several potential new drugs. They’ll also use the ACVR1 mutated cells in a very large scale screening exercise to look for other ways to kill them. Finally, the team will aim to breed mice with the mutated ACVR1 gene, and study them to help them better understand how the tumours develop. This knowledge will give them better understanding of how we could go about treating them. By the end of the study, the team hopes to have enough data to show which drugs work best. If they’ve worked well enough, this could lead to a clinical trial of new drugs for children with DIPG.