In an uncommon decision, Blood Cancer United, a nonprofit organization, revealed on Thursday that it is purchasing the remaining stock of Luvelta, an investigational cancer drug that has been discontinued.
In this agreement, Blood Cancer United, formerly the Leukemia & Lymphoma Society, will also obtain the investigational new drug designation and oversee the compassionate-use program. This program aims to provide children with a rare type of blood cancer access to the medication at no cost, as long as supplies last.
Luvelta (Luveltamab Tazevibulin) is an antibody-drug conjugate that targets a folate receptor (FOLR1). It was initially developed for lung and ovarian tumors in adults but can be used as a bridge to stem cell transplantation for a small subset of patients with acute myeloid leukemia. Sutro Biopharma, based in San Francisco, was developing Luvelta but halted its development in March 2025 to focus on other drugs, which led to the end of the compassionate-use program.
“Families face a maze of operational hurdles, small trial populations, and commercial calculations that can make a promising drug vanish,” stated E. Anders Kolb, president and CEO of Blood Cancer United. “We acquired the remaining supply of Luveltamab Tazevibulin to ensure treatment is available now, while we advocate for clearer regulatory paths, shared trial infrastructure, and incentives so these therapies can be studied and sustained for the children who need them.”
In the development of drugs for rare diseases, despite good intentions for patients, economic factors often dictate whether a medication can be feasibly developed, especially if the drug cannot be sold in large quantities. Federal initiatives like the Orphan Drug Act provide financial support for the research and development of drugs for rare diseases, but not all are manufactured even with this designation.
This complexity increases for pediatric medications, as many drugs originate with adult indications and are only used “off label” for children. Clinical trials often focus on adult populations, and pediatric uses are typically not prioritized. If adult-use cases decline, pediatric applications suffer, even if the drug showed promise for children.
“It is unfortunately one of those stories that we hear too often, of drugs kind of being left behind,” said Crystal Mackall, a doctor at Stanford University specializing in pediatric hematology and oncology, in an interview with STAT. “The people who are losing are those individuals with rare diseases, and unfortunately children’s cancer is the poster child for this.”
Blood Cancer United’s acquisition of Luvelta is part of its Dare to Dream Project, which focuses on funding transformative treatment and care opportunities. While some nonprofit groups have partnered with pharmaceutical companies to support the development of promising drugs, Blood Cancer United’s independent facilitation of compassionate-use programs and distribution of medications is rare.
“This is just a terrific model for pediatric cancer drug development because the drug has been de-risked and so much has been invested in the 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.”
One of the initial patients to receive Luvelta was Aspen Peck, diagnosed shortly after her first birthday. Her father, Troy Peck, shared with STAT that without access to Luvelta, Aspen would not have survived. Her case has informed treatment for other patients with similar conditions.
Blood Cancer United’s Kolb estimates that around 20 new patients may use Luvelta each year, and they expect to have enough supply for several years. While the current stock is set to expire in 2028, Kolb mentioned plans to explore stability testing to potentially extend its shelf life. The immediate focus is on providing the medication to those in need, with future evaluations of commercialization possibilities.
As Blood Cancer United works to broaden access to Luvelta, Kolb emphasized the drug’s importance to children and their families. “I don’t think we can overstate the fact that there are children alive today because of this drug,” Kolb said. “If we can get this drug to everybody who needs it, it’s a thousand birthdays that may not have happened otherwise.”
Following her upcoming kindergarten graduation, Aspen and her family plan to spend the summer enjoying activities like camping and swimming, free from the burden of her diagnosis. “This is bigger than us, bigger than Aspen,” Troy Peck expressed. “This is not a numbers game, saving other kids’ lives.”
