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Researchers from Purdue University in the United States developed a series of new compounds to treat acute myeloid leukemia and tested them in a preclinical study.
According to the Canadian Cancer Society, over 1,300 Canadians were diagnosed with this aggressive leukemia in 2013, with nearly a thousand Canadians died from it. It is the most common form of acute leukemia that affects adults.
Acute myeloid leukemia is an aggressive type of leukemia that has too many lymphoblasts or lymphocytes in the bone marrow and peripheral blood. It can spread to the lymph nodes, spleen, liver, central nervous system, and many other organs. Without treatment, acute myeloid leukemia progresses quickly and can be life-threatening. can cause weakness or fatigue, bruises or bleeds easily, shortness of breath, joint pain, severe weight loss, and frequent infection. Risk factors of this aggressive leukemia include smoking, exposure to industrial chemicals and radiations, and history of certain blood disorders.
Acute myeloid leukemia is difficult to treat. The goal of existing treatment method is to remove leukemia symptoms that are present and to destroy leukemia forming cells. The five-year survival rate for this disease is about 30%, but for elderly patients over 65 years, the five-year survival rate is below 10%.
In a recent study published in EBioMedicine by The Lancet, Purdue University researchers developed a series of new FLT3-inhibiting compounds that have high potential in treating the aggressive leukemia. Using human acute myeloid leukemia cell lines and mouse models, the researchers observed that these compounds are highly effective against both common FLT3 mutations and those mutations which confer drug resistance. More excitingly, these new orally administered compounds have shown no signs of toxicity so far, and do not appear to cause weight loss or other health problems.
This discovery opens new doors to reshape the future therapy of acute myeloid leukemia, especially for those who no longer respond to first- or second-generation FLT3 inhibitors. Further thorough research on safety and efficacy is still needed before we can conclude that these new compounds are safe and effective in humans.
Written by Man-tik Choy, Ph.D.
Reference: Naganna et al. Amino alkynylisoquinoline and alkynylnaphthyridine compounds potently inhibit acute myeloid leukemia proliferation in mice. EBioMedicine, 2019: Article in Press. DOI: 10.1016/j.ebiom.2019.01.012.