CHOP doctor to lead cancer research funded by Alex's Lemonade Stand

A research team led by a cancer doctor at the Children's Hospital of Philadelphia has received a $4.97 million grant from the Alex's Lemonade Stand Foundation to develop a treatment for neuroblastoma — a rare cancer mostly commonly found in young children. 

Dr. Yael Mossé's project was one of four selected to receive funding from a pool of more than 80 applicants. Her research team will use the funds to create drugs that target MYCN, an "undruggable" protein that drives several aggressive pediatric cancers, including neuroblastoma and medulloblastoma, a type of brain cancer. 

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"One of the holy grails of pediatric cancer drug development is finding a way to directly target MYCN in patient tumors," Mossé said in an email to PhillyVoice. "On behalf of my project team, I'd like to thank Alex's Lemonade Stand Foundation for the opportunity to work with my fellow peers to accelerate breaththroughs in pediatric cancer research."

Researchers have struggled to drug MYCN because it is among a group of proteins essential to normal cell processes. 

"To date, virtually all of the strategies targeting MYCN haven't really led to any responses in kids because all have been indirect," Mossé said. "We are now poised to deliver on the holy grail of pediatric cancer and that is to develop a drug that will allow for MYCN to degrade in a cancer cell and directly impact patients with MYCN-driven childhood cancers."

The researchers will use a technology called targeted degradation to design a drug that will trick cancer cells into dissolving MYCN, leading to their death. The protein fuels tumor growth, but the cancer can't grow without it. 

Mossé said her goal is to create a MYCN-direct drug in four years and prepare to launch a clinical trial after the grant is completed. 

Two other CHOP researchers will be among those on her multidisciplinary team — Dr. John Maris, a pediatric oncologist, and Ophir Shalem, a scientist whose research has focused on neurodegenerative diseases. 

The research team also will include scientists from St. Jude Children's Research Hospital, the University of California, San Francisco, the University of Würzburg and Nurix Therapeutics. 

Alex's Lemonade Stand Foundation awarded the grant through its Crazy 8 Initiative, an effort that brings together cross-disciplinary scientists, like neurologists with biologists, to accelerate treatments and cures for pediatric cancers.