CureWorks will be a ‘game changer’ for Riley kids with cancer



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The unique collaboration among elite children’s hospitals means Riley oncologists and IU School of Medicine researchers will have greater access to cutting-edge immunotherapy trials for pediatric oncology patients.

By Maureen Gilmer, IU Health senior journalist

Kids with aggressive, life-threatening cancers and their families in the Midwest have a new reason to feel hopeful, thanks to a collaboration among elite children’s hospitals that includes Riley Hospital for Children at IU Health.

“Game changer” is how Dr. Jamie Renbarger describes the news that Riley has been selected as the only CureWorks member hospital in the Midwest.

The unique collaboration among five academic children’s hospitals from Seattle to Washington, D.C., means Riley oncologists and Indiana University School of Medicine researchers will have greater access to cutting-edge immunotherapy trials for pediatric oncology patients.

“Being part of this collaborative group from a T-cell therapy and a cellular therapy perspective really puts us in a different ballgame,” said Dr. Renbarger, division chief of Riley’s hematology/oncology program.

“It feels like a game changer in terms of where we are in the field. But beyond that, for Riley patients and families, it will mean bringing clinical trial options for patients who have no good options left,” she said.

Instead of being forced to travel long distances for clinical trials, as is the case for a few Riley patients who are enrolled in trials at Seattle Children’s, patients will soon be able to get more advanced care closer to home.

Member hospitals are supported in launching and participating in exclusive clinical trials. CureWorks streamlines immunotherapy production, clinical trial enrollment and the trial coordination process.

“This opportunity to work with esteemed partners will allow more rapid development of the new frontier of care to help children fight cancer,” said Riley chief medical officer Dr. Elaine Cox.


Riley is a Midwest destination for pediatric and adolescent/young adult cancer care as the No. 15-ranked pediatric oncology program in the country (U.S. News & World Report).

“We are thrilled that Riley Children’s Health at Indiana University Health is joining CureWorks to be a key part of our collaborative effort to accelerate the discovery of new therapies for pediatric cancer,” said Dr. Michael Jensen, executive director of CureWorks and director of the Ben Towne Center for Childhood Cancer Research at Seattle Children's Research Institute.

“Through this collaboration and by extending the reach of promising clinical trials, our goal is to more quickly develop treatments with fewer side effects and better remission rates and, ultimately, enable more kids with cancer to grow up and realize their full potential.”

It likely will be late in the first quarter of 2020 or sometime in the second quarter before patients will be accepted into the first trial for acute lymphoblastic leukemia (ALL) therapies at Riley.

Dr. Jodi Skiles, director of pediatric stem cell transplant and cellular therapy at Riley, said the current CAR T-cell therapy available for ALL patients will continue to be available at Riley, but new therapies hopefully will be more successful at not only getting kids into remission but sustaining that remission.

One of the trials coming down the pike through CureWorks is a similar CAR T product that also targets a marker on the surface of the leukemia cell called CD19, but through monthly “boosts of energy” to those cells, researchers hope to see a sustainable remission. Six monthly infusions after the initial CAR T-cell infusion appears to provide protection against relapse for ALL, Dr. Skiles said.

A newer generation of products coming out through these studies will also target CD19, as well as other markers that exist on the surface of cancer cells, giving two different targets for the CAR T-cell therapy to go after, making it less likely there will be a relapse, Dr. Skiles said.

Beyond ALL, there are a number of CAR T products that will be available through CureWorks for solid tumors as well, she said.

“We are super excited,” Dr. Skiles said. “It’s exciting for us because it gives us the opportunity to be in the game and in the science of making CAR T therapy a reality for kids with the hardest-to-treat cancers. For patients it’s a homerun because they will have access to potentially lifesaving therapy closer to their home community.”

To be the only hospital in the Midwest accepted into the CureWorks network means Riley clinicians and IU scientists will be on the front lines when it comes to developing cures for the most stubborn cancers and learning in real time with colleagues, rather than waiting for years for a paper to be published on the treatment.


Along with the clinical trials at Riley will come a bigger patient population, which will require increased staff, Dr. Skiles and Dr. Renbarger acknowledge.

“From a staffing perspective, we see our group growing,” Dr. Renbarger said. “We anticipate the number of patients we care for each year will increase substantially over the next couple of years, so we need to be prepared for that.”

The transplant and CAR T program has doubled over the past year, from about 30 patients to 60. In 2020, the number could grow to 70 or 80, both physicians expect.

Clinical trials at Riley will mean “pushing the envelope on optimizing cellular therapy options or products for kids with really aggressive life-threatening leukemias,” Dr. Renbarger said.

“I think the other piece we’re excited about is that they are rolling out CAR T trials, immunotherapy trials, for kids with other types of malignancies – brain tumors, sarcomas, neuroblastoma,” she said.

“We’ll be able to offer treatments that I think have a good chance of revolutionizing the field and moving toward FDA approval of these products for kids with really bad cancers.”

Besides Riley and Seattle Children’s, other hospitals that are part of the CureWorks network are Children’s Hospital Los Angeles, Children’s National (Washington, D.C.) and BC Children’s Hospital (Vancouver, British Columbia).