Nonprofit buys experimental cancer drug to maintain patient access

In a rare move, nonprofit organization Blood Cancer United announced Thursday it was buying the remaining supplies of Luvelta, a discontinued investigational cancer drug.

As part of the transaction, Blood Cancer United, previously known as the Leukemia & Lymphoma Society, also will acquire the investigational new drug designation and manage the compassionate-use program for children with a rare form of blood cancer, distributing the medication to patients at no cost while supplies last.

Luvelta (Luveltamab Tazevibulin) is an antibody-drug-conjugate targeting a folate receptor (FOLR1) — originally developed to target lung and ovarian tumors in adults — that can serve 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 Luvelta, decided to discontinue development in March 2025 to prioritize funding other drugs. And along with that, the compassionate-use program was eliminated.

“Families face a maze of operational hurdles, small trial populations and commercial calculations that can make a promising drug vanish,” said E. Anders Kolb, 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.”

In rare disease drug development, even with the best intentions for patients, the drugmakers are often at the mercy of economic realities when deciding whether it’s feasible to develop a medication but when it is unlikely the company will be able to sell large quantities of the drug. Federal programs including the Orphan Drug Act focus on providing financial aid for research and development of drugs for rare diseases, however, not all drugs are picked up and manufactured even with this designation.

This is made even more complicated for pediatric medications, as many drugs originate with adult indications and are used only “off label” for children. Typically, clinical trials for these medications are often in adult populations, and pediatric applications are often not a priority for drug developers. Should the adult-use cases dry up, the pediatric-use cases are the collateral damage, even if the drug showed potential for use in kids.

“It is unfortunately one of those stories that we hear too often, of drugs kind of being left behind,” Crystal Mackall, a doctor at Stanford University who specializes in pediatric hematology and oncology, told 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 is purchasing Luvelta as part of its Dare to Dream Project, which prioritizes spending on transformative treatment and care opportunities. While other nonprofit groups have previously partnered with pharmaceutical companies to support development of promising medications — something Blood Cancer United did with Sutro Biopharma throughout Luvelta’s early development — it is uncommon for nonprofits to facilitate compassionate-use programs and distribute medications independently.

“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.”

Aspen Peck and family Courtesy Peck family

One of the first patients treated with Luvelta was Aspen Peck, who was diagnosed just days after her first birthday. Her father, Troy Peck, told STAT that Aspen would not be alive without access to Luvelta. Aspen’s case has been used as a guide for treatment of other patients with similar presentations of the disease.

With approximately 20 new patients who may use Luvelta annually, Blood Cancer United’s Kolb  said they anticipate having enough supplies for a few years. Notably, the current supply has an expiry of 2028, however Kolb said the organization is planning to pursue stability testing for potential shelf-life extension. At this time, he said the first priority is getting medication to the patients who need it, and they will continue to evaluate feasibility of commercialization pathways as they arise.

As Blood Cancer United focuses on expanding access of Luvelta to patients in need, Kolb noted how critical this medication is 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.”

After graduating from kindergarten next week, Aspen and her family will be spending the summer camping, swimming, and enjoying the normal “kid stuff,” unencumb…