George Kumar: Breakthrough in Pediatric Brain Cancer Research
George Kumar, Senior Director at AstraZeneca, shared on LinkedIn:
“Breakthrough in Pediatric Brain Cancer Research
NRCAM = neuronal cell adhesion molecule -a variant defined by microexon skipping is a targetable cell surface proteoform in high-grade gliomas
Researchers at Children’s Hospital of Philadelphia (CHOP) have identified missing fragments of messenger RNA – called microexons – in pediatric high-grade gliomas, one of the most aggressive childhood brain tumors.
These tiny RNA segments are present in normal brain cells but consistently skipped in tumor cells, creating a cancer-specific version of proteins like NRCAM that fuel tumor growth and invasion.
Importantly, this tumor-specific protein variant can be recognized by the immune system, opening the door to new immunotherapy approaches such as monoclonal antibodies and CAR T-cell strategies that selectively target cancer cells while sparing healthy brain tissue.
This discovery offers a promising new direction for treating hard-to-treat pediatric brain cancers and may extend to other tumors where microexon skipping occurs.
Figure Courtesy: Cell Reports
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Title: NRCAM variant defined by microexon skipping is a targetable cell surface proteoform in high-grade gliomas
Authors: Priyanka Sehgal, Ammar S. Naqvi, Makenna Higgins, Jiageng Liu, Kyra Harvey, Julien Jarroux, Taewoo Kim, Berk Mankaliye, Pamela Mishra, Grace Watterson, Justyn Fine, Jacinta Davis, Katharina E. Hayer, Annette Castro, Adanna Mogbo, Charles Drummer 4th, Daniel Martinez, Mateusz P. Koptyra, Zhiwei Ang, Kai Wang, Alvin Farrel, Mathieu Quesnel-Vallieres, Yoseph Barash, Jamie B. Spangler, Jo Lynne Rokita, Adam C. Resnick, Hagen U. Tilgner, Thomas De Raedt, Daniel J. Powell Jr., Andrei Thomas-Tikhonenko
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