Blood Cancer United Acquires Investigational Drug Supply to Preserve Access for Children with Rare, Fatal Leukemia

First-of-its-kind nonprofit intervention enables continued compassionate use of a promising treatment for children facing a 15% to 30% survival rate

WASHINGTON, June 11, 2026 /PRNewswire/ -- In an unprecedented intervention, Blood Cancer United announced that it has acquired the remaining supply of a promising investigational therapy to keep it available for children with an ultra-rare and frequently fatal form of acute myeloid leukemia (AML).

Luveltamab tazevibulin, an antibody-drug-conjugate targeting the Folate Receptor-1 (FOLR1), no longer had a path to development for its primary indication in adult cancer. As a consequence, a compassionate use program for children was discontinued.

The therapy has shown early promise for a subtype of AML known as CBFA2T3-GLIS2-rearranged AML, which is diagnosed in an estimated 17 children in the U.S. each year. This form of leukemia is resistant to conventional chemotherapy and has a survival rate of only 15 to 30%. Blood Cancer United acquired the existing drug supply and the rights to make it available through the FDA's compassionate use pathway for this specific pediatric use at no cost to patients.

The challenge of developing treatments for rare diseases means that promising therapies are often discontinued for commercial reasons, a situation that leaves pediatric cancer patients with few options.

"Families face a maze of operational hurdles, small trial populations and commercial calculations that can make a promising drug vanish," said E. Anders Kolb, M.D., president and chief executive officer of Blood Cancer United. "We acquired the remaining supply of luveltamab tazevibulin to keep treatment available now, while we push for clearer regulatory paths, shared trial infrastructure and incentives so these therapies can be studied and sustained for the children who need them."

Blood Cancer United's role is limited to preserving access to the existing supply. The organization will not commercialize the drug.

"Because this disease is so rare, we don't have a lot of data but so far the investigational therapy has shown meaningful benefit, in some cases enabling deep remissions and allowing kids to proceed to bone marrow transplant, currently the only potentially curative option," said Gwen Nichols, M.D., chief medical officer of Blood Cancer United. "When access to a promising therapy disappears, it is not an abstract policy issue — it is a life-or-death moment for families. Preserving this access is both medically urgent and morally necessary."

This intervention is critical for families like Aspen Peck's. Diagnosed with CBFA2T3-GLIS2-rearranged AML around her first birthday, Aspen's parents were told her chances of survival were extremely low. After relapsing following intensive chemotherapy and a bone marrow transplant, she received luveltamab tazevibulin through compassionate use and has remained in remission since early 2022.

"When Aspen was diagnosed, we were told it would be a miracle if she made it to kindergarten — and she'll be finishing kindergarten next week," said Troy Peck, Aspen's father. "This drug gave our daughter another chance — and every family facing this diagnosis deserves that chance."

This effort is funded through Dare to Dream, a Blood Cancer United project dedicated to transforming pediatric cancer treatment and care, including a gift from The Andrew McDonough B+ Foundation.

"For nearly 20 years, our foundation has worked in my son Andrew's memory to help children with cancer, and this milestone reflects that commitment," said Joe McDonough, co-founder of The Andrew McDonough B+ Foundation. "We are proud to help support this effort to preserve access to a promising therapy that could make a lifesaving difference for children with this rare leukemia."

Blood Cancer United has invested more than $2 billion in research since 1949, with ongoing investments in pediatric research, including:

• Support for foundational work by James Downing, M.D., of St. Jude Children's Hospital, that contributed to the identification of CBFA2T3-GLIS2 re-arrangement as a driver of this rare childhood AML.
• Multiple grants totaling several million dollars to Soheil Meshinchi, M.D., Ph.D., of Fred Hutchinson Cancer Center in Seattle, which led to the identification of FOLR1 as a therapeutic target in CBFA2T3-GLIS2 AML, and work that continues to advance understanding of the disease and development of targeted therapies, including an ongoing clinical trial of FOLR1-directed CAR T-cell therapy.

If you are a healthcare provider or caregiver interested in learning more about how to access this treatment, email [email protected].

About Blood Cancer United^®
Blood Cancer United® (formerly The Leukemia & Lymphoma Society) is the largest global nonprofit focused on blood cancer patient support, research, and advocacy. Since their founding in 1949, the organization has consistently evolved to better serve people affected by all 100-plus types of blood cancer. Blood Cancer United funds innovative research, offers free resources and personalized support, and advocates at state and national levels for more accessible and affordable healthcare for all patients. To learn more, visit www.BloodCancerUnited.org.

For support and to learn more, visit www.BloodCancerUnited.org. Patients can contact blood cancer information specialists at (800) 955-4572, Monday through Friday, 9 a.m. to 9 p.m. ET. Connect with the organization on Facebook, X, Instagram, LinkedIn, TikTok and Threads.

Media Contact:
Sandra Salviejo
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SOURCE Blood Cancer United® formerly The Leukemia & Lymphoma Society