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FDA Approves New Formulation of Important Childhood Leukemia Drug


Rye Brook, N.Y., March 8, 2023 – The U.S. Food and Drug Administration yesterday approved nelarabine injection, a critical treatment for T-cell acute lymphoblastic leukemia (T-ALL), an aggressive blood cancer that is most common in children. This is an important approval because it provides a much-needed option to the only other approved form of nelarabine, which has been affected by long-term shortages.

“The nelarabine drug shortage has jeopardized the lives of children with cancer, so we at LLS are extremely grateful that patients will have another source of this essential drug, which provides a significant boost in remissions and disease-free survival rates for T-ALL without adding any new toxicity,” said Gwen Nichols, M.D., LLS Chief Medical Officer. 

“It is notable on the heels of International Women’s Day that the company behind today’s approval, Shorla Oncology, is female led,” added Dr. Nichols. According to the company, its focus is on developing innovative, high-barrier-to-entry pharmaceutical therapies with a particular interest in women’s and pediatric health.

In addition to treating this aggressive form of leukemia, nelarabine is also approved to treat T-cell lymphoblastic lymphoma (T-LBL). T-LBL is a rare form of non-Hodgkin lymphoma that also primarily affects children but can be either aggressive or indolent (slow growing). It is generally treated the same way as T-ALL.

LLS supported a clinical trial in collaboration with the National Institutes of Health and the National Cancer Institute evaluating nelarabine in combination with a drug called a CXCR4 inhibitor, which is thought to enhance activity against advanced forms of T-ALL. The trial was completed recently; results are still being evaluated. 

LLS Dare to Dream Project is transforming treatment and care for kids with blood cancers

The challenges faced by children with blood cancer and their families are not limited to drug supply shortages. Kids are different and should be treated differently for their blood cancers. The LLS Dare to Dream project is working to improve the quality of life for every child with blood cancer. 

Dare to Dream is seeking to raise $175 million over the next five years to support global, collaborative research into unique treatments for children’s blood cancer. The LLS PedAL Master Clinical Trial is already underway, matching children with aggressive forms of leukemia to treatments targeted to their unique tumor biology. 

Dare to Dream also offers access to the full range of LLS education, personalized support services and financial assistance. LLS is also a staunch advocate on behalf of all children and their families because children need safer, less toxic and more effective treatment.

“The PedAL clinical trial continues our rich history at LLS of bringing together the best minds across the world to create innovative research to improve cancer care,” said Dr. Nichols. “LLS works across all fronts—helping to develop treatments and then ensuring access to the best possible medicines for children with all types of blood cancer.”