Bioethics Blogs

Breakthrough Immunotherapies Seem Like a Dream Come True for Children with Leukemia

Guest Post: Nancy Jecker, Aaron Wightman, Abby Rosenberg, Doug Diekema

Paper: From protection to entitlement: selecting research subjects for early phase clinical trials involving breakthrough therapies

A breakthrough therapy to cure cancer in children suffering from acute lymphoblastic leukemia (ALL) is a dream for many families.  New immunotherapies appear to make this dream a reality. Such therapies use a person’s own immune cells to recognize and combat their disease. In the largest study to date of ALL patients treated with a form of immunotherapy known as Chimeric Antigen Receptor (CAR) T-Cell therapy, a 93% remission rate was reported. Such results are a glimmer of hope for those whose prognoses were previously considered very poor.

However, the good news is tempered by the fact this potentially lifesaving experimental therapy may not be available to everyone who might benefit. And demand is growing as word spreads. Since CAR T-cell therapy for ALL is available only through clinical trials, do patients have a right to participate? How should we choose among medically suitable candidates?

We have faced these questions before. Most recently, with ZMapp to treat Ebola Virus Disease, azidothymidine (AZT) to treat HIV and AIDS, and Immunitab (Gleevac) to treat Chronic Myleogenous Leukemia. Are patients suffering from devastating, life-threatening diseases entitled to breakthrough therapies?

In a recent paper, we argue that benefit is a continuum, from the complete uncertainty associated with standard research, to an intermediate stage where evidence of benefit mounts and reaches a peak, to a final stage of clearly demonstrated benefit that is sufficient to gain approval for clinical applications.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.