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.
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Current AML Research and Clinical Trials
- Epigenetics is based on the idea that certain genes become silenced (or turned off), which contributes to causing or maintaining cancer. Drugs that can reverse the silencing process are being studied in clinical trials, either alone or in combination with other drugs. Two gene-silencing processes scientists are trying to reverse include:
- Methylation. Researchers are investigating two drugs that block methylation: azacitidine (Vidaza®) and decitabine (Dacogen®). These hypomethylating agents are FDA approved in the treatment of MDS. Both are being studied as single agents or in combination with other drugs to treat newly diagnosed and relapsed/ refractory AML.
- Histone deacetylation inhibition. Histone deacetylase inhibitors under study in clinical trials include valproic acid, suberoylanilide hydroxamic acid (SAHA) and entinostat. These drugs are being studied in combination with azacitidine or decitabine.
- Sapacitabine, a nucleoside analog, has shown promising results in trials for the treatment of older patients with AML.
- Clofarabine, a purine analog, related to cladribine has shown results as a single agent and has also shown improved rates of remission in studies, when combined with low-dose cytarabine in the treatment of older AML patients.
- CPX-351 is a liposomal carrier (containing cytarabine and daunorubicin) and is being studied in the treatment of relapsed AML patients.
- Gemtuzumab ozogamicin is an antibody-drug conjugate that pairs the antitumor antibiotic calicheamicin to an anti-CD33 antibody. This drug was FDA approved in 2000 based on its success treating older patients with relapsed AML but was later taken off the market when studies indicated it did not offer long-term benefits. It is once again under study as it has shown results in selected patients.
- Vaccine therapy that will boost the immune reaction against AML cells is another avenue of research. For instance, in one vaccine study, certain types of white blood cells are removed and exposed to a protein found on many AML cells, called Wilms tumor 1 protein or WT1. These cells are then reinfused to the patient and help other immune cells attach the leukemia cells. An early study of this vaccine has shown promising results but more research is needed.
- CAR T-cell therapy removes T cells from the patient’s blood and modifies them in the lab so that they have specific substances known as “chimeric antigen receptors” (CARS) that will help them attach to leukemia cells. Then, the cells are infused back into the patient where they can target the leukemia cells. This technique has shown very promising clinical trial results in the treatment of certain types of lymphocytic leukemias and CAR T-cell therapy is now being studied for use in AML treatment.
- Another concept called “differentiation therapy” involves studying the use of all-trans retinoic acid (ATRA), which is approved to treat APL, and some types of histone deacetylase inhibitor drugs to promote the growth and differentiation of immature leukemic blast cells.