The LLS Ground-Breaking Precision Medicine Approach Master Trial  BEAT AML Releases Third Year Test Results


A recent posting by the Leukemia and Lymphoma Society references the Beat AML Master Trial. The trial evaluates targeted therapies to treat acute myeloid leukemia (AML) and was launched in October 2016. A recent article in Nature announced the first published study results.

The announcement also mentioned that the researchers created a collection of data that can be accessed through the National Institute of Health and other agencies. They believe that making their findings public will lead to the development of new drugs to treat AML.

About The Leukemia and Lymphoma Society (LLS) Trial

LLS has the distinction of being the largest voluntary agency in the world related to blood cancer. It supports research for blood cancer all over the world. Funding by LLS for acute myeloid leukemia began over sixty-six years ago. LLS has supported AML research by investing almost $100 million in the last five years with a goal of developing improved lifesaving therapies.

LLS has taken the lead for the Beat AML Master Trial. This is a “first” as until now no other non-profit agency has participated in a study together with pharma and biotech sponsors.

Permission to lead the trial was given to LLS by the FDA who also worked with LLS regarding trial protocol. Three internationally renowned scientists who will lead the Beat AML trial are the Knight Cancer Institute’s Dr. Brian Druker, Dr. John Byrd of Ohio State University, and Dr. Ross Levine of Memorial Sloan Kettering. Ten arms of the trial, which are testing several novel therapies for AML, will be led by other prominent researchers at their respective institutions.

LLS anticipates a total participation of five hundred patients and eventually between fifteen to twenty research sites that are currently testing targeted therapies.

To date, 672 tumor biopsies that were taken from 562 patients have been analyzed by researchers who discovered mutations that had not previously been identified.

Unlike many other trials that use placebos for comparison, in the Beat AML Master Trial, every participant receives appropriate therapy. If they do not have a specific marker, they will be treated with broad therapy.

Rather than “one size fits all” this trial involves the testing of novel therapies that are paired with specific molecular mutations in AML. The trial is considered an “umbrella trial” because it consists of multiple collaborators as well as multiple sponsors who are working together to develop precision therapies in accordance with a patient’s gene profile (interaction of a person’s genes).

The “umbrella” concept sets the Beat AML Master Trial apart from most other trials because the newly-diagnosed subjects are selected after being screened for mutations that are known to be the cause of their disease. They will then be treated with a targeted therapy developed for that specific mutation.

About Acute Myeloid Leukemia (AML)

AML mostly affects the elderly who generally cannot withstand the standard, toxic chemotherapy regimen. It is perhaps one of the most common forms of leukemia yet one of the most deadly, accounting for about ten thousand deaths every year.

There has not been any significant advancement in standard treatment for AML patients in over forty years. However, the FDA has recently approved a variety of drugs for specific subsets.

Developing targeted therapies has been difficult for scientists due to the complexity of AML’s mutational patterns. To date, more than eleven types of genetic classes have been identified. In addition, over twenty subsets of AML may be assigned when diagnosing leukemic blasts (cancers).

A Model For Future Clinical Trials

The final results of the Beat AML Master Trial will provide a pathway for future clinical trials and offer a wide range of treatment options. Researchers believe that the novel collaborative approach of the trial will be used in the future as a model for other clinical trials.

Read more about this story here.


Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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