RANKINE FAMILY FELLOWSHIP IN NF1
At a time when funding for discovery science is increasingly difficult to secure, your support provides something invaluable: the ability to keep pursuing promising ideas and building the evidence needed to bring others alongside us.
Sinai Health's Lunenfeld-Tanenbaum Research Institute (LTRI), scientists are tackling some of the most important questions in NF1-associated cancer, from identifying vulnerabilities that could point to new treatment strategies to understanding why some NF1- altered cells become cancerous while so many others do not. This year, both areas of research are moving into important new phases, with large-scale studies underway and promising early findings beginning to emerge.
Your generosity is powering progress in NF1 research
Dr. Daniel Schramek’s team is advancing a genome-wide screen to identify new treatment targets for NF1-associated cancers and related pre-cancerous changes. Dr. Chris Lowden, Rankine Family Fellow in NF1, is part of the team advancing this important work.
The team is about 70 per cent through generating and analyzing data from more than 300 mice, with several promising early findings already identified.
Once complete, the strongest candidates will be validated to determine whether they could lead to new drug targets involving NF1 and the related MAPK pathway.
A second major screen is also examining vulnerabilities in NF1-mutant breast cancer.
Early signals are encouraging, but careful analysis and testing are still underway. Support from the Duck & Dodge community makes this work possible at scale and helps attract new partners and investment to NF1 research.
Uncovering why some NF1-altered cells become cancerous
Dr. Rod Bremner’s team is investigating why some NF1-altered cells become cancerous while most do not. Although the loss of NF1 can increase the activity of RAS genes that drive cell growth, only a small number of affected cells form tumours.
Using single-cell sequencing data from mouse and human samples, the researchers found that cancer-susceptible cells have more active genes and greater variability in gene activity, which may create rare combinations that trigger cancer.
Dr. Renzhi Yao, Rankine Family Fellow in NF1, has been instrumental in advancing this work. He has shown that reducing the activity of genes linked to this variability can block cancerous transformation in laboratory models.
The team will now test these findings in living mouse models through a large-scale screen with Dr. Daniel Schramek’s lab. If confirmed, the research could reveal new ways to prevent NF1-associated cancers before tumours develop. Support from the Duck & Dodge community gives Dr. Yao the opportunity to pursue this promising direction.
