Disease Information & Support


The Word:


Taking part in a clinical trial may be the best treatment choice for some acute myeloid leukemia (AML) patients. Clinical trials are under way for patients at every treatment stage and for patients in remission. Today's standard treatments for cancer are based on earlier clinical trials. The Leukemia & Lymphoma Society continues to invest funds in AML research.

Clinical trials can involve new drugs, new treatments and new uses for approved drugs or therapies to treat AML. Clinical trials are conducted worldwide under rigorous guidelines to help doctors find out whether new cancer treatments are safe and effective or better than the standard treatment.

Current AML Research and Clinical Trials

Epigenetics Research

Epigenetics is a natural process in our bodies that turns genes on or off. Researchers suspect that when certain genes are mistakenly turned off ("silenced") it may contribute to the cause of cancer or its growth. They're studying drugs that can reverse gene silencing, either alone or in combination with other drugs.

Two gene-silencing processes scientists are trying to reverse include:

  • Methylation. A small molecule in our bodies called a methyl group sometimes attaches itself to other molecules, silencing them and causing methylation. Researchers are investigating two drugs that block methylation: azacitidine (Vidaza®) and decitabine (Dacogen®).
  • Histone deacetylation inhibition. Enzymes called histone deacetylases can silence genes that normally prevent cancer from developing. Histone deacetylase inhibitors prevent the process. Inhibitors being studied include valproic acid, suberoylanilide hydroxamic acid (SAHA) and entinostat. These drugs are combined with Vidaza or Dacogen.

Novel Drugs

Scientists are trying to create new drugs or find them from natural sources. Specifically, they're studying some novel drugs that kill cells by triggering new pathways that cause cell death and thereby overcome resistance. The novel drugs can be combined with standard AML drugs such as ara-C and daunorubicin (Cerubidine®).

Some novel drugs currently being tested to treat AML are:

  • clofarabine (Clolar®) , which is approved to treat acute lymphoblastic leukemia
  • tipifarnib (Zarnestra®)
  • vosaroxin (which is being studied in combination with cytarabine for relapsed/refractory AML)
  • flavopiridol
  • interleukin-2 (IL-2) with histamine dihydrochloride (Ceplene®)
  • a class of drugs called antisense molecules

New drugs that target FLT-3-ITD include midostaurin, sorafenib (Nexavar®) and AC220. These drugs are being combined with other chemotherapy drugs. CPX-351 is a treatment currently being studied in newly diagnosed older adults and in relapsed/refractory adults.

Differentiation Therapy

Differentiation therapy involves studying the use of all-trans retinoic acid (ATRA), which is approved to treat acute promyelocytic leukemia (APL) and some types of histone deacetylase inhibitor drugs to convert immature leukemic blast cells into fully functioning cells.

Maintenance Therapy

Researchers are focusing on less intensive chemotherapy, called maintenance therapy, using drugs such as 6-thioguanine or cytarabine. Some studies have resulted in improved disease-free survival, although most studies showed no improvement in overall survival. Other clinical trials are examining the role of hypomethylating agents, such as azacitidine and decitabine as maintenance therapy.

Donor Lymphocyte Infusion

Researchers are studying the effects of transferring lymphocytes from the blood of a healthy donor to an AML patient. Called a donor lymphocyte infusion, the procedure is done to destroy any cancer cells remaining after stem cell transplantation, vaccine therapy or other immunotherapies.

Go back to:
last updated on Thursday, May 17, 2012

Get Information
& Support

Contact an Information Specialist.

Newly Diagnosed?