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Precision Medicine for AML

Published: 2019-05-10 |

Source: The Leukemia & Lymphoma Society

Precision medicine centers on giving the right treatment to the right patient at the right time based on their genetic information and other characteristics. We are beginning to crack the code to apply precision medicine to acute myeloid leukemia (AML), one of the most daunting challenges in the blood cancers.

Each year, about 20,000 Americans are diagnosed with AML, and half of these patients are expected to have exceptionally poor outcomes. Standard treatment has typically involved intensive chemotherapy, followed by a stem cell transplant in some cases. But this approach fails most patients, with the majority of whom are older adults who cannot tolerate the intensity of treatment. With more than one-quarter of our research budget invested in AML, LLS is leading the charge to address this urgent, unmet need. Our work is paying off. Researchers are now able to home in on specific genetic drivers of the disease--opening the door to a more personalized approach to treatment.

In 2018 alone, the FDA approved five new treatments for AML, and LLS helped advance all of these somewhere along their development journey:

* ivosidenid (Tibsovo), a new targeted therapy, was approved for patients with relapsed or refractory AML who have certain genetic mutation called IDH1.

* arsenic trioxide (Trisenox), an existing drug, was approved in combination with chemtherapy for newly diagnosed adult patients with low-risk acute promyelocytic leukemia (APL).

* glasdegegib (Daurismo), a new therapy, has been approved in combination with chemotherapy for the treatment of newly diagnosed adult patients with AML who are age 75 or older or who have other chronic health conditions or diseases that might prelude the use of intensive chemotherapy. The therapy targets a cell signaling pathway called Hedgehog, which is crtical for the development of immature cells into cells with more specialized functions.

* venetoclax (Venclexta), an existing therapy, is now approved in combinationwith chemotherapy, for the treatment of newly diagnosed adult patients with AML who are age 75 or older or who have other chronic healh conditions or diseases that might prelude the use of intensive chemotherapy.

* gilteritinib (Xospata), a new therapy, was approved for patients with relapsed or refractory AML, who have a genetic mutation called FLT3, which is seen in about one-third of AML patients.

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