Marks the First-Ever Global Pediatric Trial for Menin Inhibitors
The PedAL (Pediatric Acute Leukemia) Master Trial is at the heart of LLS’s Dare to Dream Project to transform treatment and care for kids with blood cancer
WASHINGTON, APRIL 21, 2025 – The Leukemia & Lymphoma Society (LLS) announced today the first pediatric patient has received treatment in a new subtrial of its Pediatric Acute Leukemia (PedAL) Master Clinical Trial (NCT04726241). PedAL is a first-of-its-kind integrated worldwide master trial that tests multiple targeted drugs simultaneously to accelerate treatments for leukemia, the most common childhood cancer.
Infants and children with specific types of relapsed/refractory leukemia (KMT2A-rearranged, NUP98-rearranged, or NPM1-mutant acute leukemia) are now eligible to enroll in the subtrial, which is investigating a combination of chemotherapy and a menin inhibitor.
This subtrial involves pediatric patients with some of the most common and hardest-to-treat leukemias. In fact, 80% of infants with acute lymphoblastic leukemia have a genetic rearrangement of the KMT2A gene. About 40% of infants with acute myeloid leukemia have one of the three specific types of leukemia that the trial’s treatment targets1.
Menin inhibitors are a new and promising class of drugs that block the interaction between other proteins and the menin protein. Research has shown that the menin protein is responsible for driving progression of certain types of leukemia.
The new subtrial is open in North America at Children’s Hospital of Philadelphia, Cincinnati Children’s Hospital, St. Jude Children’s Research Hospital, Memorial Sloan Kettering Cancer Center, Lurie Children’s Hospital of Chicago and SickKids - The Hospital for Sick Children in Toronto. Global trial sites are open in Spain, the Netherlands, France and Austria.
“It is rare for a trial to be so ambitious and forward thinking about the needs of children while studies in adults are still ongoing. The new subtrial is evaluating the safety, efficacy and dosing for the treatment of certain acute leukemias in pediatric patients,” says E. Anders Kolb, M.D. President and Chief Executive Officer of The Leukemia & Lymphoma Society. “Historically, cancer treatments have been evaluated in adults first and if successful, then children. This has resulted in a nearly decade-long delay between when therapies are first available for adults compared to children.”
The new subtrial brings LLS one step closer to achieving its bold goal: By 2040, LLS will enable patients with blood cancer to gain more than one million years of life.
New trial expands on PedAL, builds on success of LLS’s Dare to Dream Project
PedAL launched in 2022 with one treatment subtrial investigating venetoclax. That trial is now open at approximately 80 clinical sites in 19 countries across the globe.
Enrollment in the treatment trials begins with the robust PedAL screening trial (NCT04726241), open in 179 sites in the United States, Canada, Australia and New Zealand. To date, the screening trial has enrolled almost 450 children to identify their unique tumor biology and help match them to any targeted treatment clinical trial they are eligible for, whether in or outside of PedAL.
Because blood cancers are more common in adults, there is a larger incentive for new treatments to be developed in that population, and progress for pediatric acute leukemia has fallen behind. PedAL is changing this paradigm.
A delay in research and the development of therapies for children with aggressive forms of cancer threatens their survival. Today, leukemia is the second leading cause of cancer deaths in children. And for children who survive, 80% develop life-altering and chronic health issues from their treatment.
“More children are being diagnosed with leukemia today than ever before,” says Kim Schuetz, an oncology nurse, an LLS Dare to Dream Ambassador and mother to Austin, who was diagnosed with acute lymphoblastic leukemia in 2011. “We need to study more treatment options for these children. Through Dare to Dream, we’re striving to transform care, so they don’t experience a laundry list of life-long health issues and the possibility of developing secondary cancers because of treatment from standard therapies, such as harmful chemotherapies.”
LLS has supported the development of menin inhibitors over two decades
In the early 2000s, LLS provided funding to a group of bright scientific investigators, which led them to discover the menin protein inside cancer cells and its role in the devastating effects of certain forms of leukemia.
Over the next several years, LLS provided more than $6 million through its venture philanthropy program, the Therapy Acceleration Program (TAP), to scientists at the University of Michigan. They discovered small molecule inhibitors of the interaction of two proteins, menin and KMT2A/MLL, that, when fused together, drive progression of AML. With TAP funding, the scientists demonstrated the activity of their compounds in the laboratory setting. To facilitate translation of the scientists’ progress in the laboratory to the clinic, LLS introduced the scientists to a clinical-stage biopharmaceutical company.
Helping children with blood cancer survive and thrive
Dare to Dream is transforming treatment and care for kids with blood cancer, so they not only survive but thrive after treatment. PedAL is an integral part of LLS’s Dare to Dream Project along with support services, advocacy efforts and funding for blood cancer research:
- Research: With 35 active academic research grants totaling more than $31 million in funding commitments to pediatric and adolescent and young adult research, LLS-funded investigators are conducting groundbreaking research including exploring the origins of pediatric blood cancers and investigating increased incidence and better treatment options for leukemia in children with Down Syndrome.
- Advocacy: To ensure that more kids with blood cancer have better access to care, LLS advocates for meaningful changes at the state and federal level. A recent victory was the reauthorization of the Childhood Cancer Survivorship, Treatment, Access and Research (STAR) Act, which expanded childhood cancer research and created plans to enhance the tracking and reporting of pediatric cancer cases and treatment outcomes.
- Education & Support Services: LLS continues to expand its free patient education and personalized support services for pediatric patients and families to provide guidance, information and comfort during treatment and beyond.
“The successes of the Dare to Dream Project, now and in the future, positions us well to reach our bold goal of enabling blood cancer patients to gain more than one million years of life by 2040,” says Gwen Nichols, M.D., Chief Medical Officer of LLS. “Advances in treatment and care, spearheaded by Dare to Dream, will give even more children a chance to grow and thrive into adulthood.”
People can donate to LLS at any time. From May 12-16, all donations made to LLS will go directly to The Dare to Dream Project. Interested in donating to The Dare to Dream Project? Visit this link for more information.
About The Leukemia & Lymphoma Society
The Leukemia & Lymphoma Society® (LLS) is the global leader in the fight against blood cancer. The LLS mission: Cure blood cancer and improve the quality of life of all patients and their families. LLS funds lifesaving blood cancer research around the world, provides free information and support services, and is the voice for all blood cancer patients seeking access to quality, affordable, coordinated care.
Founded in 1949, LLS has regions throughout the United States and Canada. To learn more, visit www.LLS.org. Patients should contact the Information Resource Center at (800) 955-4572, Monday through Friday, 9 a.m. to 9 p.m. ET.
For additional information, visit lls.org/lls-newsnetwork. Follow us on Facebook, X, Instagram, LinkedIn and TikTok.
About The Dare to Dream Project
Dare to Dream exists to transform treatment and care for kids with blood cancer. Because kids are different and need to be treated differently, Dare to Dream funds groundbreaking research and the LLS PedAL Master Clinical Trial as well as expands LLS support services and drives advocacy efforts to help all kids with blood cancers get accessible, affordable, quality healthcare.
LLS proudly and gratefully acknowledges all those contributing to the LLS mission, including corporate partners Walgreens, Valvoline™ Global Operations, The Wawa Foundation, and Subaru of America, Inc. and its retailers who have each pledged over $1 million to Dare to Dream. LLS also recognizes Sarah Asma and the Moore family; The Don & Lorraine Freeberg Foundation; The Harry T. Mangurian Jr. Foundation; Norcross Foundation, Inc.; Joan and Paul Rubschlager; and The Bobby Zahurak Pediatric AML Research and Patient Support Fund for their outstanding contributions to Dare to Dream.
For more information about Dare to Dream, including resources for patients and their families, trial details, and eligibility, visit: lls.org/dare-to-dream.
About LLS PedAL
As part of LLS’s Dare to Dream Project, the Pediatric Acute Leukemia Master Trial (PedAL) is the first-of-its-kind global master clinical trial for pediatric acute leukemia patients that will fundamentally change how children are treated. Prior to enrolling in therapeutic trials, patients enroll in the PedAL Screening Trial (NCT04726241) to identify the unique tumor biology of each child’s cancer and help them to match with the most promising treatment. The Screening Trial is currently open at multiple sites in the United States, Canada, Australia, and New Zealand.
At this time, two PedAL therapeutic trials are open and actively enrolling patients in the U.S., Canada, Australia, New Zealand, Japan, and Europe, with more therapeutic trials planned for global execution. To learn more about PedAL, visit lls.org/dare-to-dream.
PedAL would not be possible without the support of major foundation donors such as Gateway for Cancer Research, which pledged $1.5 million over three years to support genomic sequencing and flow cytometry, which is being conducted through the PedAL screening trial, and the Lisa Dean Moseley Foundation, which has committed $1.25 million over five years to support the PedAL Principal Investigators Fellowship Program, whose members are leading the PedAL screening trial and therapeutic trials.
Media Contact:
Sandra Salviejo
Senior Director, Communications
Sandra.salviejo@lls.org
Reference:
- Juul-Dam KL et al. European Journal of Medical Genetics 2023