Targeted Therapy for Leukemia

Clinical trials

Roswell Park researchers are working continually to develop new targeted therapies, and to bring the latest options from around the world here for our patients. These are offered through clinical trials.

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Targeted therapy is also a systemic treatment in that it circulates through the whole body. But instead of finding rapidly dividing cells like chemotherapy, targeted drugs find the cancer cells by focusing in on specific or unique characteristics of the cancer cells, such as a protein or genetic mutation. 

Even though two patients have been diagnosed with the same type of leukemia, they may not respond in the same way to the same treatment. Advanced testing, such as genetic and molecular testing, reveal that the underlying biology of cancer cells can be very different in those two patients. For example, specific abnormal proteins and genes may be identified in cancer cells in some patients but not in others.

The unique genetic and molecular profile of your cancer cells will determine which type of targeted therapy may fight your cancer. Targeted therapies may be used alone or in combination with chemotherapy.

Targeted therapies for leukemia include:

  • Tyrosine kinase inhibitors
  • GD33 targeting drug that is linked to a chemotherapy drug
  • Core binding factor
  • Drugs that target the genetic mutations FLT3, IDH1, IDH2 and the KMT2A rearrangement

Roswell Park researchers are working continually to develop new targeted therapies, and to bring the latest options from around the world here for our patients. These are offered through clinical trials.

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