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FDA Expands Use of Leukemia Treatment to Include Children

LLS supported the very first antibody-drug conjugate gemtuzumab ozogamicin, which targets CD33, and was approved in 2000 to treat acute myeloid leukemia.

Rye Brook, N.Y., March 18, 2023 – The U.S. Food and Drug Administration recently approved inotuzumab ozogamicin (Besponsa®) for the treatment of pediatric patients 1 year and older with CD22-positive B-cell acute lymphoblastic leukemia that has returned or worsened despite earlier treatment. The treatment was first approved for adults in 2017.

“LLS support has played a major role in development of antibody-drug conjugates (ADCs) like inotuzumab ozogamicin,” says Lee Greenberger, Ph.D., LLS Chief Scientific Officer. “ADCs have been called “biological missiles,” because they have the ability to find very specific cancer cells, in this case leukemia cells with a protein called CD22, and then to directly deliver a highly potent drug to kill them.”

LLS supported the very first antibody-drug conjugate gemtuzumab ozogamicin, which targets CD33, and was approved in 2000 to treat acute myeloid leukemia. LLS also supported the development of ADCs that target CD22 as far back as 2008. 

Today, there are well over a dozen ADCs to treat six types of blood, plus additional non-blood cancers. LLS has millions of research dollars currently committed to developing even more ADCs through research funding grants and its unique venture philanthropy initiative, the LLS Therapy Acceleration Program.

LLS Dare to Dream: Transforming Treatment and Care for Kids with Blood Cancer

In addition to supporting research that advances all blood cancer treatments, LLS supports and leads research focused exclusively on improving the quality of life and outcomes for every child with blood cancer.

LLS PedAL, a pillar of the LLS Dare to Dream Project, is the first integrated, global, acute leukemia master clinical trial testing new, safer therapies in children. The trial matches children to treatments based on their unique tumor biology. 

The idea is to use treatments that zero in on cancer cells while having less effect on healthy cells to provide effective treatment with fewer short-term side effects and avoid the long-term effects childhood cancer survivors can have for years, decades or even a lifetime.

The goal of PedAL is to radically change the landscape of how children with acute leukemia are treated and to provide a clinical trial framework that will accelerate research into better treatments for these patients. 

“Far too often, progress for children with blood cancer has fallen behind adults as evidenced by the original approval in 2017 of inotuzumab,” says Dr. Greenberger. “We are in a new era of cancer treatments and know more than ever about individual genetic traits that drive blood cancers, allowing us to move away from one-size-fits-all treatment to treatments personalized to each patient. What’s even more exciting is that the pace of our new knowledge is speeding up all the time.”

If you or a loved one need personalized disease, treatment or support information, you can contact one of our Information Specialists:

Media Contact:
Ryan McDonald