Researchers discover key mutation in acute myeloid leukemia
A gene mutation discovery may lead to treatment changes in acute myeloid leukemia (AML), a cancer of the blood.
Balintfy: Researchers have discovered mutations in a particular gene that affect the treatment prognosis for some patients with acute myeloid leukemia or AML. AML is an aggressive blood cancer.
Ley: It is a very bad disease to get.
Balintfy: That's Dr. Tim Ley at the Washington University School of Medicine in St. Louis. He says there are 13,000 new AML patients in America annually.
Ley: 13,000 news cases a year and 9,000 deaths.
Balintfy: He explains that in this disease, the cancer cells or tumors crowd out the normal cells in bone marrow, where the elements of blood are made in the human body. Dr. Ley adds that even with the best therapies available, the vast majority of patients with this disease die from it.
Ley: So the patients die of the complications of basically a kind of bone marrow failure where the tumor is taking over the normal marrow space.
Balintfy: Now researchers using targeted DNA sequencing on nearly 300 AML patient samples have confirmed that mutations discovered in a single gene, appear responsible for treatment failure in a significant number of AML patients. Dr. Ley says the mutations predict poor overall survival.
Ley: And what that usually means is that patients relapse earlier, and when you have relapse to AML, you’re very, very lucky to go on to have a long term survival because the first relapse is usually the beginning of the end for patients with AML.
Balintfy: Patients with the mutation survived for a median of just over a year, compared to the median survival of nearly 3.5 years among those without the mutation. But Dr. Ley explains that identifying this gene mutation could be an important tool for classifying patients early to know whether or not to intensify their treatment up front.
Ley: What that generally means is that if the patient has a donor for an allogenic bone marrow transplant available, in first remission, what we do in poor risk patients is we transplant them immediately because we know that without a transplant they’re not likely to do very well.
Balintfy: The five-year survival rate for adults with AML is about 20 percent. But researchers are optimistic that the findings, once verified in larger studies, will not only lead to a change in medical care, but may provide a molecular target against which to develop new drugs. For more information on AML and this study published in the New England Journal of Medicine, visit www.cancer.gov. This is Joe Balintfy, National Institutes of Health, Bethesda, Maryland.
About This Audio Report
Reporter: Joe Balintfy
Sound Bite: Dr. Tim Ley
Topic: acute myeloid leukemia, AML, blood, blood cancer, cancer, tumor, bone marrow
Additional Info: Researchers discover key mutation in acute myeloid leukemia