Class of 2016: Progress from the first St. Baldrick’s Robert J. Arceci Innovation Awardees

by St. Baldrick's Foundation
December 16, 2020

The volunteers who make up the St. Baldrick’s Foundation certainly “think different” when raising money for childhood cancer research. And thinking outside the box was a trademark of the late great Dr. Bob Arceci – a St. Baldrick’s shavee, board member and world-renowned researcher. 

Dr. Arceci

In his memory, the St. Baldrick’s Robert J. Arceci Innovation Awards were created. The goal was to give doctors the freedom and flexibility to think outside the box – to explore avenues that may have been left unexplored otherwise. The grants are unrestricted, making them unique in cancer research. And they are big awards, at $250,000 a year for 3 years. 

The first class of researchers to receive these innovation awards began in 2016.  Dr. Charles Mullighan, from St. Jude Children’s Research Hospital received the North America award, and Dr. Sam Behjati, from Wellcome Sanger Institute in the U.K., received the International award. Read on to find out where they are now: 

Getting More Precise With Leukemia 

Dr. Charles Mullighan

Upon receiving this exploratory funding Dr. Mullighan set out to explain genetic missteps in different types of leukemia, and to evaluate the potential for epigenetic targeted therapy in Acute Lymphoblastic Leukemia (ALL).  

“The work supported by this grant has allowed me to take multiple risks and pursue many new lab techniques, including studying new types of leukemia, developing new experimental approaches that examine genes in single human cells, and building new experimental models of leukemia,” Mullighan said. 

Many patients undergo genomic sequencing to classify the disease and accurately diagnose the type of leukemia. These sequencing results have important implications for how the disease is treated. Dr. Mullighan’s work has resulted in a new classification of B-Cell Acute Lymphoblastic Leukemia (B-ALL) – the most common ALL subtype – that has been translated into the clinic. This classification tool improves accurate diagnosis of B-ALL. According to Dr. Mullighan, this grant has significantly propelled his goal of “making basic science discoveries and moving these to better diagnostic and treatment approaches.” His work has also identified new genetic drivers of treatment failure and relapse in ALL.

Not one to stop asking questions, Dr. Mullighan continues his work with support from federal and foundation sources. He plans to research the genomics of response to immunotherapy, and relapse in acute lymphoblastic leukemia and related disorders. We look forward to hearing more about his accomplishments. 

Groundbreaking Work on Precursors to a Childhood Cancer 

Dr. Behjati in his lab

With his innovation award funding, Dr. Behjati zeroed in on Wilms tumor, which is the most common type of kidney cancer in children, with around 500 cases diagnosed annually. Dr. Behjati and colleagues were the first to compare healthy kidney tissue with Wilms tumor tissue, using comparative genome analysis to figure out what causes the disease.  

Doctors homed in on a specific “rogue cell” that appears normal in both cancerous and non-cancerous tissue, but suppresses the H19 gene, which is meant to ensure the healthy growth of cells. With this gene turned off, cancer cells can grow uninhibited. 

Dr. Behjati explains it like this: “As cells divide, they acquire mutations, so we sought to know the ‘post code’ — to find out where does it come from and to sequence that post code. At the root of the tumor is a patch that looks normal genetically. But since it’s pre-cancer, the DNA changes.” 

Dr. Behjati continued: “In adult cancers, you have polyps in the colon that can be a precursor to bowel cancer. But, these types of pre-cursor cells are not seen in children, which is what makes this study groundbreaking.”  

Dr. Behjati is currently expanding his research into the causes of different childhood cancers, with an emphasis on pediatric solid tumors. We are excited to see where this research takes him. 

Freedom to take innovative ideas and run with them, to devote the time to work with a team of researchers to study whether those ideas have merit, is one of the hallmarks of the Arceci Award. Work like Dr. Behjati’s and Dr. Mullighan’s would not have been possible without you – the St. Baldrick’s supporters, fundraisers, and donors throughout the world.

Help Ensure This Important Work Continues by Donating Today.


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