In an unusual move, the nonprofit organization Blood Cancer United announced Thursday that it will purchase the remaining supply of the discontinued investigational cancer drug Luberta.
As part of the deal, Blood Cancer United, formerly known as the Leukemia and Lymphoma Society, will also receive Investigational New Drug designation, manage a compassionate use program for children with rare forms of blood cancer, and distribute the drug free to patients while supplies last.
Luberta (rubertamab tazevibulin) is an antibody-drug conjugate that targets the folate receptor (FOLR1) and was originally developed to target lung and ovarian tumors in adults, but it serves as a bridge to stem cell transplantation in a small subset of patients with acute myeloid leukemia. San Francisco-based Sutro Biopharma, which was developing Ruberta, decided to halt development in March 2025 to prioritize funding for other medicines. As a result, the Compassionate Youth Program was also discontinued.
“Families face a maze of operational hurdles, small trial populations, and commercial calculations that could wipe out a promising drug,” said E. Anders Kolb, president and CEO of Blood Cancer United. “We have acquired the remaining supplies of rubertamab tazevibulin to maintain current access to treatment, while promoting clearer regulatory pathways, shared trial infrastructure, and shared incentives to ensure these treatments are studied and continue to serve children in need.”
Rare disease drug development is often at the mercy of economic realities when determining whether a drug is viable, even with the best intentions for patients, when it is unlikely that the drug company will be able to sell the drug in large quantities. Federal programs, including the Orphan Drug Act, focus on providing financial support for research and development of drugs to treat rare diseases, but even with this designation, not all drugs will be collected and manufactured.
Things get even more complicated with pediatric medicines, as many medicines originate from indications for adults and are only used “off-label” in children. Clinical trials for these drugs typically focus on adults, and pediatric applications are often not a priority for drug developers. Even if the drug shows potential for use in children, pediatric use becomes collateral damage when adult use dries up.
rare disease
Learn more about rare diseases
“Unfortunately, the story of drugs being left behind is one that we hear far too often,” Crystal McCall, a physician specializing in pediatric hematology and oncology at Stanford University, told STAT. “It’s people with rare diseases who are losing out, and unfortunately childhood cancer is a prime example of that.”
Blood Cancer United is acquiring Luberta as part of its Dare to Dream project, which prioritizes spending on innovative treatments and care opportunities. While other nonprofits have previously partnered with pharmaceutical companies to help develop promising medicines, as Blood Cancer United did with Sutro Biopharma through the early development of Luberta, it is unusual for nonprofits to pursue compassionate use programs and distribute medicines on their own.
“This is a great model for pediatric cancer drug development because it reduces the risk of the drug and there’s a lot of investment in this drug in terms of research and development,” said Nancy Goodman, patient advocate and founder of Kids v Cancer. “I hope this is a model we can replicate.”
aspen peck and familyProvided by the Peck family
One of the first patients treated at Luberta was Aspen Peck, who was diagnosed a few days after her first birthday. Her father, Troy Peck, told STAT that Aspen would not be able to survive without access to Luberta. Aspen’s case is being used to guide the treatment of other patients with similar symptoms.
About 20 new patients a year could use Luberta, so Blood Cancer United’s Kolb said he expects there will be enough supply for several years. Notably, the current supply has an expiration date of 2028, but Kolb said the organization is planning stability testing for potential shelf life extensions. At this time, he said, getting the drug to patients who need it is the top priority and the company will continue to evaluate the feasibility of commercialization pathways.
Blood Cancer United is committed to expanding access to Ruberta to patients who need it, and Dr. Kolb noted how important this drug is to children and their families. “I don’t think it can be overstated the fact that there are children alive today because of this drug. If we can get this drug to everyone who needs it, we’ll have a thousand birthdays that might not have happened otherwise.”
After Aspen graduates from kindergarten next week, Aspen and his family plan to enjoy the usual “kid’s games” like summer camp and swimming without his diagnosis getting in the way. “This is bigger than us, bigger than Aspen,” Troy Peck said. “This is not a numbers game where you save other children’s lives.”
