New patient-centered initiative aims to accelerate pediatric AML research and treatments

Blood Cancer United^® today announced the launch of Dare to Dream REALworld: AML, a patient-centered initiative co-founded with Rally Foundation for Childhood Cancer Research. The initiative will engage current and former pediatric acute myeloid leukemia (AML) patients and their families to build real-world evidence that can inform research and future clinical trials, helping accelerate the development of new treatments.

For children with rare diseases like AML, time matters. Because patient populations are small, clinical trials can be difficult to conduct, and research can move more slowly than families can afford to wait. REALworld: AML gives children and families an opportunity to contribute directly to research while helping us build the evidence researchers need to design more efficient studies and advance better treatment options."

Gwen Nichols, M.D., chief medical officer, Blood Cancer United

REAL stands for Real-world Evidence Accelerating New Treatments. REALworld: AML was co-founded by Blood Cancer United and Rally Foundation for Childhood Cancer Research, whose early support helped make the initiative possible. Tempus (NASDAQ: TEM), is providing technology to support the initiative, including Olivia, its personal health concierge app.

"Families confronting pediatric AML urgently need research approaches designed around the realities of rare childhood cancers," said Dean Crowe, Founder and CEO, Rally Foundation for Childhood Cancer Research. "We are proud to have helped co-found REALworld: AML and support an initiative that puts families at the center of research. By building high-quality evidence from the experiences of children with AML, we have an opportunity to advance treatment development while creating a model that could ultimately benefit children facing other rare cancers."

Families who participate can contribute directly to research while gaining access to Olivia, which brings a patient's health information together in one place, helping families access, organize and better understand their records. Through Olivia, participants will also receive educational information from Blood Cancer United based on their child's diagnosis, treatment, and support needs.

"The promise of Olivia is to ensure every patient receives the full benefit of what medicine offers, while also accelerating scientific discovery to improve those benefits," said Chris Scotto DiVetta, SVP of Olivia at Tempus. "Olivia puts meaningful health and educational resources directly into the hands of families, while creating new opportunities for them to play an active role in advancing pediatric AML research. We're thrilled to be part of the Dare to Dream Project and contribute to an initiative that has the potential to make a meaningful difference for children and families."

Building evidence to advance pediatric treatment and care

REALworld: AML aims to create a rigorous dataset that researchers can use to better understand pediatric AML and improve the design of future clinical trials. Over time, the data may also support external control arms, allowing researchers, when scientifically and regulatorily appropriate, to compare patients receiving an investigational treatment with carefully validated information from similar patients who previously received standard treatment. This approach could allow more children in a trial to receive investigational therapy and help researchers evaluate promising treatments more efficiently.

The initiative comes as the U.S. Food and Drug Administration continues to advance guidance on the use of real-world data in clinical research and regulatory decision-making. The approach has particular potential in pediatric cancers and other rare diseases, where small patient populations can make traditional randomized trials difficult.

Helping the next family

For Abby Breyfogle, the promise of REALworld: AML is deeply personal. Her twin daughters, Kenedi and Kendal, were both diagnosed with AML shortly after birth. After about six months in the hospital undergoing chemotherapy, both girls went into remission. Kendal later relapsed and died in September 2017. Kenedi remains in remission today.

Abby is turning her family's experience into an opportunity to help others. She has shared Kenedi's health records and plans to share Kendal's, so both of her daughters' experiences can contribute to pediatric AML research.

Families can currently contribute to this research by sharing electronic health records from children who are living with or in remission from pediatric AML. The project also recognizes the importance of learning from children who have died from the disease and expects to be able to include their records in the near future.

"When you've lived through pediatric AML, you understand how urgently families need better options. Knowing that researchers can learn from both Kenedi's and Kendal's experiences means so much to our family. Our hope is that what we've been through can help the next family who hears the words 'your child has cancer.'"

Families interested in participating in REALworld: AML can learn more here: Dare to Dream REALworld: AML | Blood Cancer United

Transforming treatment and care

REALworld: AML is part of Dare to Dream, Blood Cancer United's initiative to transform treatment and care for children with blood cancer through biomedical research, patient support, advocacy, and clinical research innovation.

At the heart of Dare to Dream is PedAL, the first pediatric acute leukemia Master Clinical Trial. Through the PedAL screening trial, children receive comprehensive genomic screening to identify the unique biology of their leukemia and help determine whether they may be eligible for a PedAL treatment subtrial or another appropriate clinical trial. To date, PedAL has screened more than 660 children at more than 100 medical centers worldwide and currently includes two treatment arms: one evaluating the use of venetoclax and another evaluating ziftomenib, a menin inhibitor being studied for certain forms of acute leukemia.

Through Dare to Dream, Blood Cancer United also recently preserved access to an investigational therapy for children with a rare and aggressive form of AML by acquiring the existing drug supply and making the treatment available to eligible patients through a compassionate use program. Dare to Dream also expands access to education, personalized support, financial assistance resources, and supports advocacy efforts for families facing blood cancer.