A diagnosis of acute myeloid leukemia (AML) can be terrifying, with the disease claiming over 10,000 lives each year. The Leukemia & Lymphoma Society (LLS), however, is working with world-renowned researchers and medical centers, multiple pharmaceutical companies, a clinical research organization, a genomics provider and the FDA, in hopes of improving outcomes for patients.

Groundbreaking approach

“The Beat AML Master Trial is the first ever precision medicine clinical trial in a blood cancer,” explains Amy Burd, Ph.D., and vice president of LLS Research Strategy. “This trial has the potential to change the paradigm of treatment for AML, one of the most deadly blood cancers,” she adds, “And one that has not seen a change in the standard of care in 40 years.”

The groundbreaking trial, sponsored by LLS, a nonprofit, will test multiple novel, targeted drugs simultaneously throughout the country. Newly diagnosed AML patients over age 60 will have a genetic analysis performed, and be directed to a study arm of the trial testing a drug deemed most appropriate.

Technology is employed to identify the genetic mutations causing the cancer, so that patients can be treated with a therapy or combination of therapies designed to inhibit the genetic mutation and kill the cancer cells while sparing the healthy cells.

Burd clarifies: “These treatments are often far less harmful to surrounding healthy cell tissue than standard chemotherapy treatments."

A cutting-edge therapy

Another emerging precision medicine approach for cancer patients is CAR T-cell therapy. T-cells, a type of white blood cell, are part of the normal cells found within everyone’s immune system. Their normal job is to assist the immune system and eliminate foreign material that can enter the body.

“CAR T-cell therapy is an approach that harnesses the patient’s own immune system to fight the cancer,” says Lee Greenberger, Ph.D., chief scientific officer within LLS. “The patient’s immune cells are purified, and then genetically engineered in the laboratory. These genetically engineered cells are re-injected into the patient. Then, they find and kill the cancer cells in the body.

"Certain types of T-cells can kill other cells. In CAR-T, these cytotoxic T-cells are extracted from the patient’s blood and then genetically engineered to produce special receptors on their surface that allow them to recognize a unique protein on the cancer cell. This allows the genetically engineered T-cell to home-in on the cancer cell and kill it.

"Not only does the T-cell receptor have a homing device, it also contains special programming that allows it to rapidly divide and multiply when the tumor cell is encountered.”

“This trial has the potential to change the paradigm of treatment for AML…”

Seeing results

In 2010, retired chemist Douglas Olson became one of the first patients to undergo CAR T-cell therapy. Having been diagnosed in 1996 with chronic lymphocytic leukemia, he eventually underwent chemotherapy, followed by five years of remission, but didn't respond to treatment when the cancer returned. A bone marrow transplant seemed to be his only hope.

“Three weeks after undergoing CAR T-cell therapy, Olson’s doctor told him 19 percent of his white cells were killing cancer cells and they were multiplying,” remembers Greenberger. “The next week, his doctor told him they could not find a single cancer cell in his body. Less than four weeks after the first infusion of the modified T-cells, the clinical trial saved his life.”

Moving forward

Although blood cancers remain the third-leading cause of cancer deaths in the United States, Greenberger is optimistic about the future of research.

"CAR T-cell therapy is one of a number of different emerging immunotherapy approaches that are showing great promise in treating blood cancer patients, as well as patients with other types of cancers,” he says. “Clinical trials of patients with relapsed or refractory acute lymphoblastic leukemia, chronic lymphocytic leukemia, lymphoma and myeloma are showing remarkable results for some of the patients.

“Advances in technology and in our understanding of the underpinnings that cause cancer, as well as new emerging immunotherapies and precision medicine-targeted agents, are all rapidly converging to give us hope that we are closer than ever to finding cures.”