Bioethics Blogs

Hitting the Right Target? Lab Studies Suggest Epigenetic Drug May Fight Childhood Brain Cancer

Caption: Remembering a few of the many children who’ve died of DIPG; Left, Lyla Nsouli and parents; upper right, Andrew Smith and mom; lower right, Alexis Agin and parents.
Credits: Nsouli, Smith, and Agin families

Every year in the United States, several hundred children and their families receive a devastating diagnosis: diffuse intrinsic pontine glioma (DIPG). Sadly, this inoperable tumor of the brain stem, little known by the public, is almost always fatal, and efforts to develop life-saving treatments have been hampered by a lack of molecular data to identify agents that might specifically target DIPG. In fact, more than 200 clinical trials of potential drugs have been conducted in DIPG patients without any success.

Now, using cell lines and mouse models created with tumor tissue donated by 16 DIPG patients, an international research coalition has gained a deeper understanding of this childhood brain cancer at the molecular level. These new preclinical tools have also opened the door to identifying more precise targets for DIPG therapy, including the exciting possibility of using a drug already approved for another type of cancer.

The latest work was performed at 13 institutions in the United States, France, Canada, and The Netherlands. In addition to its groundbreaking science, this coalition is to be commended for the creative way in which it pulled together researchers from a wide range of disciplines, along with financial support from both the public and non-profit sectors. Besides NIH, the coalition received funding from the National Science Foundation, Department of Defense, Lyla Nsouli Foundation, Children’s Oncology Group, DIPG Collaborative, Accelerate Brain Cancer Cures Foundation, CureSearch for Childhood Cancer, Team Julian Foundation, and many other non-profit organizations.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.