A Quest for the BEST Ewing Sarcoma Therapy

By Jo Avelar

May 29, 2024
5 min read

Jo Anne Avelar
(626) 739-2716

LOS ANGELES, CA (May 30, 2024) – A generous $1 million donation from an anonymous donor in 2019 allowed St. Baldrick’s to put out a call for fresh ideas to tackle Ewing sarcoma, emphasizing the urgency of improving patient outcomes. This gift established the Martha’s Better Ewing Sarcoma Treatment (BEST) Grant for All, named after a remarkable teenager who was fighting Ewing sarcoma and passed away in 2022.

More than 44 researchers responded, and after a vigorous scientific peer review process, Martha’s BEST Grant was awarded to Dr. Poul Sorensen for research to target Ewing sarcoma tumors.

Now as the grant wraps up, Dr. Sorensen and his team are particularly thrilled about their findings concerning a surface protein called IL1RAP, which they discovered is highly expressed on the surface of Ewing sarcoma cells. With this grant, they crafted an antibody designed to bind onto IL1RAP specifically on these tumor cells. Next steps involved combining this antibody with drugs to form highly specific therapeutic antibody-drug conjugates that target and kill Ewing sarcoma cells.

“We have an overwhelming desire to see this transition into clinical trials for kids with Ewing sarcoma,” said Dr. Sorensen. “A clinical trial represents the culmination of Martha’s family’s hopes with this grant, and holds strong potential to improve outcomes for these children.”

This research is so promising that a number of funding partners have come together to support the final steps necessary to push this potential new treatment over the finish line, with the goal of opening a clinical trial.

Here’s what these partners – D-Feet Cancer, The Faris Foundation, The Shohet Family Fund for Ewing Sarcoma Research, and Martha’s family – have to say about their motivation in supporting this work:

“Dalton was always ready with a smile, a hug, and a quick wit. Before he passed away from Ewing Sarcoma at 12 years old, his parents, Jen and Will promised Dalton they would continue to fight this disease for kids like him. “D-Feet Cancer, the Dalton Fox Foundation, was formed to help make good on this promise and we are proud to support Dr. Sorensen’s Better Ewing Sarcoma Therapies (BEST) research.”

– Jen and Will

“Dr. Sorensen’s research has unveiled a very provocative and novel approach to understanding metastasis in Ewing sarcoma, an elusive malady. Ewing sarcoma needs a creative approach to better understand its challenging and daunting outcome with metastatic disease. Our foundation carefully vets original, bold, and transformative research “as if our child’s life depends on it.” This is THAT research — full of promise and hope. We are very excited and optimistic about supporting his rigorous science and the knowledge that will come forth from this investigation.”

– Dr. Asha Virani, The Faris Foundation Executive Director.

“Noah desperately wanted to help others, to give back, and to make a positive difference in the world. We fund this research for Noah in hopes that it can help Noah’s and our wish for a better outcomes in Ewing sarcoma come true. This research has the potential to transform the treatment of Ewing sarcoma for all who are affected in the future. This is the possibly life-changing work we are desperately hoping for.”

– Felice Adler-Shohet, The Shohet Family Fund for Ewing Sarcoma Research.

“Our family is extremely hopeful that the continued funding of the Martha’s BEST (for all) grant will someday provide a cure for Ewing sarcoma, giving kids like Martha a chance at life after cancer.”

– Ned Riedel, father of Martha.

With this new infusion of funding from the original anonymous donor and the partners above, Dr. Sorensen and his team will use the new BEST grant to wrap up the following studies necessary before launching a clinical trial:

  1. Expand on previous studies that showed their IL1RAP antibody drug conjugate is able to block Ewing sarcoma primary tumor growth and metastasis in models. These models will allow researchers to see how the immune system affects their findings.

  2. Assess the effectiveness of the IL1RAP antibody drug conjugate in combination with current standard of care chemotherapy. Because initial clinical trials will likely involve patients with advanced disease undergoing chemotherapy treatment, this will be necessary to determine appropriate dose levels.

  3. Evaluate how the drug moves through and out of the body in a safe way (the pharmacokinetic profile of the IL1RAP antibody drug conjugate) – a necessary step before phase 1 clinical trials.

“I’ve learned that making strides in science requires teamwork, not just individual effort. Seeing a group of funders come together to support this critical last step is fantastic. It shows how collaboration outside the lab is just as crucial as collaboration within it. This chance for us to bring meaningful deliverables back to kids with Ewing sarcoma wouldn’t be possible without these passionate donors and partners,” says Dr. Sorensen.

Donate now and help support research into better treatments for kids with cancer. 

About St. Baldrick’s Foundation
Every 2 minutes, a child somewhere in the world is diagnosed with cancer. In the U.S., 1 in 5 will not survive. The St. Baldrick’s Foundation, the largest charity funder of childhood cancer research grants, has awarded more than $342 million to researchers to Conquer Kids’ Cancer. When you give to St. Baldrick’s, you don’t just give to one hospital – you support every institution with the expertise to treat kids with cancer across the U.S. St. Baldrick’s ensures that children fighting cancer now — and those diagnosed in the future — will have access to the most cutting-edge treatment, by supporting every stage of research, from new ideas in the lab to the training of the next generation of researchers, to lifesaving clinical trials. St. Baldrick’s has played a role in virtually every advancement in the field over the past 25 years and remains essential in advancing progress and fostering innovation in childhood cancer research. Visit StBaldricks.org and help #ConquerKidsCancer.