James Niblock, Director, Early and Post-Trial Access Programs at Thermo Fisher Scientific, shared on LinkedIn:
“This was a really interesting read, building on the news from earlier this summer that Blood Cancer United (formerly The Leukemia and Lymphoma Society) had acquired the rights and remaining supply of luveltamab tazevibulin, an investigational drug originally being developed in adult cancers, including ovarian cancer.
Through an FDA expanded access program, the drug had shown encouraging results in children with a rare and aggressive form of AML, with data reported in 25 patients.
When Sutro discontinued development of the drug in 2025, the pediatric program and the patients relying on continued access were also at risk of being impacted.
Blood Cancer United’s decision to step in and take over the EAP is the first example I’m aware of where a nonprofit has taken on this kind of responsibility for an existing expanded access program.
Two things stood out:
- The importance of continuity planning and exploring all of the ‘What If?’ questions during program planning. What happens if development is discontinued? Who assumes responsibility for the program, the IND, the drug supply, and most importantly the patients already receiving treatment?
- A reinforcement of the potential value of expanded/early/managed access in identifying signals of benefit in patient populations outside the original development plan. It’s never a substitute for clinical research, but particularly in rare diseases and small patient populations, experience gained through these programs can generate insights that might otherwise never emerge.
The collaboration between Blood Cancer United with Sutro Biopharma is an inspiring example of finding a path forward when development decisions create a potential treatment gap, and of doing everything possible to preserve access for patients in need.”
Title: How One Nonprofit Is Providing Access to a Discontinued Investigational Drug for Children With a Rare Form of AML
Author: Jo Cavallo, E. Anders Kolb
This might be interesting:
Acute Myeloid Leukemia (AML) Survival Rates: Why Prognosis Differs Across Genetic Subtypes
