1) MADC, the Mid-Atlantic DIPG Consortium, brought three geographically tied institutions together to fight DIPG. Johns Hopkins, Children's National Medical Center and the National Institutes of Health-Pediatric Oncology Branch agreed to share DIPG tumor material that had been obtained through timely autopsy donation.
2) From a fresh tissue specimen, the researchers were able to establish a cell line- JHH-DIPG1. The cell line was evaluated for the Notch pathway as this is known to occur in other aggressive brain tumors. JHH-DIPG1 showed a high level of Notch pathway activation.
3) From the cell line, a xenograft animal model was also developed.
So.... what makes this significant? Well,
First, it is very difficult for any one institution to have enough DIPG tissue for research. I see these type of collaborative efforts key to being able to crack the code of DIPG.
Secondly, it continues to show that parents will donate tumor after death to try to make a difference in this horrendous disease. It shows that Stanford was not a fluke. Cell lines and animal models can be developed for DIPG.
Thirdly, it brings DIPG more on par with other brain tumor and cancer researchers. We now have cell lines and animal models (not only at Hopkins but also elsewhere). Without these, research seemed at a standstill.
Finally, there are actual Notch agents that are available! The pediatric phase 1 with the gamma-secretase inhibitor RO4929097 has already closed because it has reached its enrollment. We are now just waiting for results. The research here could be a basis for starting a clinical trial for DIPG with an agent- or at least being able to do more pre-clinical work with an appropriate model.
AACR Notch Abstract-
Gamma-secretase inhibitor RO4929097 in treating young patients with relapsed or refractory solid tumors, CNS tumors, lymphoma or T-Cell Leukemia