Just like with basic research, the past few years have seen important developments in translational research on DIPG. As recently as 2008, translational research was not conducted on DIPG cells because researchers lacked access to DIPG tissue. Instead, this research was conducted on cells from adult glioblastoma tumors, which recent research has shown is considerably different from DIPG. In 2009, using post-mortem tissue donated by families of DIPG patients, researchers first developed cell lines of DIPG tissue that could be used for testing. The following year, in 2010, researchers successfully inserted DIPG cells into the brainstems of mice, which allowed animal testing on DIPG tumors for the first time. As a result, researchers can now investigate potential DIPG therapies on actual DIPG cells both grown in petri dish and in animal models.
The ability to conduct translational research using DIPG cells has already produced great results. In May 2015, researchers released the results of a study conducted on the DIPG cell lines and DIPG-infected mice. This study looked at 83 different potential chemotherapy drugs, and identified one of them – panobinostat – as potentially effective in treating DIPG. Panobinostat is a “histone deacetylase inhibitor,” which means that it affects that way histone-wrapped DNA is modified reversing some of the abnormal gene expression pattern of DIPG cells. Given that most DIPG tumors also have other mutations and abnormalities, panobinostat is not expected to cure any DIPG patients by itself, but doctors and researchers are optimistic that it could provide an effective treatment when used in combination with other drugs. There is currently an one clinical trial using panobinostat, but it is limited to patients whose tumors are progressing. There is not yet a clinical trial using panobinstat for newly diagnosed patients.