Blood Cancer United buys drug stockpile for compassionate use
Blood Cancer United, a nonprofit organization formerly known as the Leukemia & Lymphoma Society, made a groundbreaking announcement on Thursday. They revealed their decision to purchase the remaining supplies of Luvelta, an investigational cancer drug that had been discontinued. In addition to acquiring the drug, Blood Cancer United also obtained the investigational new drug designation and took charge of the compassionate-use program for children with a rare form of blood cancer. They will be distributing the medication to patients at no cost while the supplies last.
Luvelta, also known as Luveltamab Tazevibulin, is an antibody-drug-conjugate designed to target a folate receptor (FOLR1). Originally developed to combat lung and ovarian tumors in adults, Luvelta has shown potential in a small subset of patients with acute myeloid leukemia as a bridge to stem cell transplantation. The drug was being developed by San Francisco-based Sutro Biopharma, but the company decided to halt its development in March 2025 to focus on funding other drugs, leading to the discontinuation of the compassionate-use program.
E. Anders Kolb, the president and CEO of Blood Cancer United, expressed the organization’s commitment to ensuring that promising drugs like Luvelta remain available to patients in need. He highlighted the challenges faced by drug developers in the rare disease space, where economic realities often dictate the feasibility of bringing a medication to market, especially if the patient population is small. Despite federal programs like the Orphan Drug Act providing financial support for rare disease research, not all drugs receive the necessary backing for manufacturing.
The plight of pediatric medications is further complicated by the fact that many drugs initially developed for adult use end up being used off-label for children. Clinical trials often prioritize adult populations, leaving pediatric applications as an afterthought. Crystal Mackall, a pediatric hematology and oncology specialist at Stanford University, lamented the fact that children with rare diseases, especially those with cancer, are often overlooked in drug development.
Blood Cancer United’s purchase of Luvelta is part of their Dare to Dream Project, which focuses on investing in transformative treatment and care opportunities. While nonprofit organizations have previously collaborated with pharmaceutical companies to support drug development, it is rare for them to independently manage compassionate-use programs and distribute medications. Nancy Goodman, a patient advocate and founder of Kids v Cancer, lauded this model and hoped it could be replicated in the future.
One of the first patients to benefit from Luvelta was Aspen Peck, whose life was saved thanks to the drug. Aspen’s case has served as a guide for treating other patients with similar forms of the disease. With approximately 20 new patients potentially using Luvelta each year, Blood Cancer United expects to have enough supplies for a few years. They are exploring options to extend the drug’s shelf life beyond its current expiry in 2028.
As Blood Cancer United works to expand access to Luvelta, Kolb emphasized the critical importance of the medication to children and their families. He underscored the impact of the drug in saving lives and enabling children like Aspen to enjoy a normal childhood. Aspen and her family are looking forward to a summer filled with camping, swimming, and other activities, free from the burden of her diagnosis. Troy Peck, Aspen’s father, emphasized the significance of saving lives beyond their own, highlighting the profound impact of Luvelta on the lives of children with cancer.
