On July 19, 2017, the Leukemia & Lymphoma Society, or LLS, proclaimed that they would be expanding their initiative to beat acute myeloid leukemia. Referred to as "Beat AML Master Trial", the project aims to test new and innovative treatments for those suffering from AML. For more information regarding the trial, please visit www.lls.org/beataml.
Necessity for "Beat AML"
In the US, blood cancer is the third leading type of cancer death. Specifically, over 20,000 Americans suffer from acute myeloid leukemia resulting in over 10,000 deaths every year. An AML diagnosis can be devastating, especially for those over sixty, where survival rate drops to under 20 percent.
Some blood cancers have made major advances in their treatments; however, in the last forty years, the progress made for AML specifically has been slim. The treatment remains a mix of toxic chemotherapies, but some researchers believe a better therapy for patients is out there. This is why LLS has decided it is their responsibility to step up their research to find an investigational treatment to combat the deadly blood cancer.
Dr. Louis J. DeGennaro, Ph.D., is the president and CEO of the Leukemia & Lymphoma Society. He explains their goals for "the Beat AML Master Trial", “as the world’s leading non-profit dedicated to fighting blood cancers, LLS is uniquely able to convene the key stakeholders in cancer research to make the Beat AML Master Trial possible. Working closely with the U.S. Food and Drug Administration (FDA), renowned cancer researchers, medical centers and the pharmaceutical industry, we are seeking to change the way cancer research is conducted.”
Response to the trial
Response to LLS's master trial was overwhelmingly successful. Over seventy patients enrolled in the trial, along with six of the most nationally respected cancer centers, with a steady influx of new entrants. Four influential biopharmaceutical companies also got on board with the mission, seeing its vital importance, and three more are planning to join them.
OSUCCC, MSK, OHSU Knight Cancer Institute, University of Colorado Cancer Center, University of Texas Southwestern, and University of Chicago Comprehensive Cancer Center are currently in the process of enrolling patients. Celgene, Gileade Sciences, Alexion, and Boehringer Ingelheim have provided drugs to the study, including investigational drugs samalizumab/ALXN6000 (anti-CD200); BI 836858 (anti-CD33); enasidenib/AG-221/CC-90007 (IDH2 inhibitor); entospletinib (SYK inhibitor).
DeGennaro explains his gratitude toward the extremely positive response to the trial, “we are very pleased with the patient enrollment to date and would like to offer our deepest thanks to our Beat AML Master Trial collaborators for their hard work and commitment to helping patients affected by this challenging disease. It takes a team to find cures and, together, we believe we can change the treatment paradigm for AML and bring new and better treatments to patients who urgently need them.”
How the trial is administered
Patients enrolled in the trials receive therapies dependent on their genetic markers, which is a specific trait of this approach. DeGennaro explains why this personalization is a necessity, “LLS is a patients first organization, and cancer patients have waited too long for better treatments for AML. We are very grateful to those patients who have enrolled in the Beat AML Master Trial; the learnings and treatments resulting from this innovative trial will help tackle these diseases, by delivering the right drug to the right patient at the right time.”
The trial utilizes advanced genomic technology that is administered by Foundation Medicine. By this, they gain the ability to identify genomic mutations that are promote cancer in patients who have been recently diagnosed and are 60 years old, or older. Once this identification has been made, researchers can best match them with an investigational drug based on their subtype of acute myeloid leukemia. The process takes exactly a week to complete, which is far faster than any preceding genomic technology.
Desired results of the trial
Researchers have specific questions they want answers to that drive the trial itself, but the main question is whether or not a personalized therapy that is largely centered around genomics be administered to a patient with acute myeloid leukemia within only seven days safely?
The trial is already reaching and surpassing their goals, because more than 70 patients have been treated within 7 days, whereas initially the objective was only 20.
The first of its kind
Not only is the Beat AML Master Trial innovative in its own right due to the technology it is utilizing, but it is the first ever cancer clinical trial that has been run by a non-profit organization. A major accolade, the LLS has been awarded the Investigational New Drug (IND) approval by the FDA. They continue to habitually collaborate and converse with the FDA in regard to their trial.
In good hands
Physician scientists involved in the Beat AML Master Trial are some of the most revered in the industry, including Brian Druker, M.D., OHSU Knight Cancer Institute and Ross Levine, M.D., MSK. John Byrd, M.D., OSUCCC is also involved, who was previously granted with LLS's Return of the Child Award, which is bestowed on someone who has given a key and durable scientific accomplishment to the better knowledge, control, and treatment of blood cancers.
Future of the trial
Four more cancer centers and three more biopharmaceutical companies are expected to join the Beat AML Master Trial. The number of enrolled patients is projected to eventually hit 500. The trial will continue for two more years at minimum, dispersed between 15 and 20 clinical sites.
Beyond the trial
Although clearly impressive and innovative in many rights, the Beat AML Master Trial is not the Leukemia & Lymphoma Society's only ongoing initiative to assist patients of blood cancer. A whopping 26 percent of their research budget is dedicated to AML research and they have spent over $100 million only in the last five years. They also donate many of their resources to initiatives that enable patients via education and support services.
One way in which LLS benefited the AML community was in 2009 when they partnered with Celator Pharmaceuticals in order to assist the progression of their investigational drug CPX-351 (Vyxeos ®). The drug was a combination of two existing therapies that LLS thought would be promising in the treatment of AML. The drug's promise showed when it performed more strongly than the traditional therapy used for AML in a Phase 3 trial done on patients who had secondary AML, which is a high-risk subset of the disease. Despite the fact that this work began all the way back in 2009, the drug is currently under FDA review. If approved, it will be the second treatment passed in the past 40 years to treat AML patients. The FDA's decision is set to be announced within the month. From teaming with pharmaceutical companies to starting their own clinical trial, LLS has proven to constantly be on the lookout for ways to assist those with blood cancer.
DeGenarro explains, “Throughout our 68-year history, LLS has time and again taken a leadership role when it has recognized a challenge for patients. We’re doing it again with the Beat AML initiative, working together to advance new therapies and improve outcomes for these patients who desperately need new and better options."