New Compounds Developed for Acute Myeloid Leukemia Treatment
Background on Acute Myeloid Leukemia
Researchers from Purdue University in the United States have created a series of novel compounds aimed at treating acute myeloid leukemia (AML) and tested them in a preclinical study. The Canadian Cancer Society reported that in 2013, over 1,300 Canadians were diagnosed with this aggressive form of leukemia, resulting in nearly a thousand fatalities. AML is the most prevalent type of acute leukemia among adults.
Understanding Acute Myeloid Leukemia
Acute myeloid leukemia is characterized by an excessive number of lymphoblasts or lymphocytes in the bone marrow and peripheral blood. The disease can metastasize to lymph nodes, the spleen, liver, central nervous system, and other organs. Without timely intervention, AML progresses rapidly and poses a significant threat to life. Common symptoms include weakness or fatigue, easy bruising or bleeding, shortness of breath, joint pain, severe weight loss, and frequent infections. Risk factors associated with this aggressive leukemia include smoking, exposure to industrial chemicals and radiation, and a history of specific blood disorders.
Current Treatment Challenges
Treating acute myeloid leukemia remains challenging. Existing therapies primarily aim to alleviate symptoms and eliminate leukemia-forming cells. The five-year survival rate for AML is approximately 30%. However, for elderly patients aged over 65, this rate drops to below 10%.
Recent Developments in Research
A recent study published in EBioMedicine by The Lancet details Purdue University’s development of new FLT3-inhibiting compounds, which show promising potential in treating this aggressive leukemia. The researchers utilized human AML cell lines and mouse models, discovering that these compounds effectively target both common FLT3 mutations and those mutations that confer drug resistance. Notably, these new orally administered compounds have demonstrated no signs of toxicity and do not appear to induce weight loss or other health issues.
Implications for Future Treatments
This discovery could significantly alter the landscape of therapy for acute myeloid leukemia, particularly for patients who do not respond to first- or second-generation FLT3 inhibitors. However, further comprehensive research on the safety and efficacy of these compounds is necessary before confirming their suitability for human use.
Author and Reference
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.