A Milestone for Research: Shir for Life Funds Breakthrough Immunotherapy Research at CHOP

March, 2025 - Shir for Life completed its second major payment to support an innovative research project at Children’s Hospital of Philadelphia (CHOP), bringing our total funding to $180,000. This contribution is helping drive the development of a first-in-class targeted immunotherapy for children with neuroblastoma—a cancer that remains one of the deadliest in pediatrics.

A New Frontier: Targeting ALK in Neuroblastoma

Led by Dr. Yaël Mossé, CHOP’s research team is developing a precision therapy that targets ALK, a protein found on the surface of most neuroblastoma cells and absent from healthy tissue. This makes ALK an ideal candidate for antibody-based treatments, offering the potential to kill cancer cells while sparing healthy ones.

“The Shir for Life Foundation provided critical funding to create and optimize a new precision immunotherapy which will catalyze our ability to test this new drug in children with high-risk neuroblastoma,” said Dr. Mossé. “We think that this new therapy has the potential to be a game changer and be applicable to most if not all children with neuroblastoma as a safe and effective treatment.”

What’s Being Developed

The project focuses on creating a humanized ALK-targeted Antibody Drug Conjugate (ADC)—a therapy that links a monoclonal antibody to a potent cancer-killing payload. This strategy has shown success in blood cancers and is now being tested in solid tumors like neuroblastoma.

Shir for Life’s funding is now being used to optimize and test multiple ADC candidates in vitro and in vivo, with the goal of selecting the best therapy to advance to clinical trials.

Why It’s Urgent

Relapsed neuroblastoma currently has survival rates below 10%. The treatments available are aggressive and often leave children with lifelong complications. This new immunotherapy has the potential to be safer, more targeted, and effective against resistant disease.

The research may also extend beyond neuroblastoma. ALK expression is now being identified in other pediatric cancers like rhabdomyosarcoma, gliomas, and medulloblastoma—offering hope to even more children.

What’s Next

Dr. Mossé and her team are working to complete preclinical studies and initiate an Investigational New Drug (IND) application to begin Phase 1 clinical trials. With additional funding, Shir for Life aims to continue supporting this journey from lab bench to bedside.

This work represents the kind of high-impact, science-driven progress that Shir for Life is proud to champion—thanks to the generosity of our donors and supporters.

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