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FDA Approves First Targeted Treatment for Newly Diagnosed Leukemia Subtype

LLS supported critical research in the development of the treatment, called ponatinib. 

Rye Brook, N.Y., March 25, 2024 – The U.S. Food and Drug Administration (FDA) last week approved ponatinib (Iclusig®) in combination with chemotherapy to treat adults with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia (PH+ ALL).  

About one in four adults with ALL have this subtype and require a different treatment approach. The main treatment for ALL is long-term chemotherapy, but the one in four whose cancer harbors the PH+ gene need an additional treatment like ponatinib that specifically targets the gene. 

“Finding unique genetic features on cancer cells, like the Philadelphia chromosome, and then designing drugs to target them is one of the biggest advances in cancer research in the 21st century,” says Lee Greenberger, Ph.D., LLS Chief Scientific Officer. “LLS supported the first major breakthrough in targeted treatment and also supported the latest one, with LLS-funded researchers discovering critically important information needed to design ponatinib.” 

LLS supported research led to the 2001 FDA approval of imatinib (Gleevec®) for the treatment of PH+ chronic myeloid leukemia (CML). Today, a patient who is in remission from PH+ CML after two years of treatment with imatinib has the same life expectancy as someone who doesn’t have cancer.  

The advent of targeted treatments for ALL has not only improved survival rates, but it has made treatment more tolerable. This is essential because most people who get ALL are older and are the very people less likely to be able to tolerate the treatment they need to survive. 

“PH+ ALL is an aggressive form of leukemia associated with poor outcomes,” says Dr. Greenberger. “Ponatinib will offer newly diagnosed patients with PH+ ALL a new option that could make treatment easier and more effective for them.” 

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Media Contact: 
Ryan McDonald 
Senior Manager, Medical and Science Communications