In 1969, cancer advocates published a full-page advertisement in The New York Times calling on President Richard Nixon to make curing cancer a national priority. Three years later, Congress passed the National Cancer Act, substantially increasing funding for cancer research.
In the five decades since, advances in diagnosing and treating cancer have helped people to survive and live longer than before. One particularly impressive example is multiple myeloma, which has seen five-year survival rates nearly double from 27% in 1975 to 51% in 2011.
Researchers, however, still have significant room for improvement for many blood cancers. While 90% of patients with Hodgkin’s lymphoma in 2011 lived five years or longer after diagnosis, only 28% of patients with AML did the same, making it one of the deadliest blood cancers today.
Those still left behind in the war on cancer are top of mind as we recognize Blood Cancer Awareness Month. The entire month of September provides advocates such as Louis DeGennaro, Ph.D., President and CEO of The Leukemia & Lymphoma Society (LLS), an opportunity to highlight the unmet needs of the estimated 1.4 million people in the U.S. who are either living with or are in remission from blood cancer.
“I’m glad we can acknowledge we have had great success in many blood cancers,” DeGennaro said. “We truly have made remarkable progress. But some of the remaining challenges we face are due to the vast complexity of the diseases.”
Those complexities are now forcing advocates like him into new roles in developing new treatment options for patients.
More Complex Than We Thought
While the advocates who petitioned President Nixon 50 years ago suggested that cancer could be conquered within a matter of years, the disease proved to be a far more formidable foe.
Blood cancer is a complex family of many diseases—including leukemia, lymphoma, multiple myeloma, myelodysplastic syndromes (MDS) and myeloproliferative neoplasms (MPNs)—each with its own subtypes.
As advances in molecular profiling offer new information, researchers continue to classify new subtypes based on their genetic, genomic and molecular characteristics. A big breakthrough came in 1996 when researchers discovered the overexpression of FLT3 in acute myeloid leukemia (AML). Similarly, in 2008 researchers sequenced the genome of tumor cells in a woman with AML, revealing new mutations not previously associated with the disease, such as IDH1 and IDH2. Scientists now believe that AML comprises at least 11 genetically distinct diseases.
Today, the World Health Organization recognizes more than 100 different classifications of blood cancer. While many of these subtypes may present themselves similarly in terms of symptoms, the cells affected and underlying biology can be very different.
“It’s not surprising then that the one-size-fits-all treatment approaches of the past don’t work for the majority of patients diagnosed with blood cancer,” DeGennaro said. “Understanding the underlying complexity of each subtype of blood cancer has been the fundamental challenge.”
Personalized Blood Cancer Treatment
As usual, researchers haven’t been intimidated by these challenges. They are continuing to unravel the complexities of the disease and pinpoint some of the genetic, genomic and molecular drivers. Those discoveries have led to the development of targeted treatments and the promise of an era of precision medicine in the near future.
Sometimes medicine takes time to catch up to scientific discoveries, DeGennaro laments. For instance, while researchers found in 1974 that tumor cells in many patients with MDS are missing part of chromosome 5, and researchers were unable to manage this until 2006.
Advocacy organizations like the LLS have played a major role in closing this gap. The LLS was founded 70 years ago by a couple who lost their son to leukemia and wanted to support research. Since then, the LLS has invested $1.3 billion in research focused on advancing treatments for all blood cancers.
That funding has been fruitful. In 2017 and 2018 alone, the U.S. Food and Drug Administration approved 34 blood cancer treatments, with the development of most supported by LLS in one way or another, according to DeGennaro. “That’s an incredible sign of the impact advocates have had on cancer,” DeGennaro said.
As a result of progress in blood cancer research over the decades, we’re beginning to imagine using the word cure more and more often.
Bigger Roles for Blood Cancer Advocates
With all this progress, the challenge for modern doctors is now choosing which treatment to use when and potentially in what combinations.
Solving this emerging challenge requires a fundamental shift in how researchers evaluate new treatments, away from traditional models and toward more adaptive trials, according to DeGennaro. Adaptive studies test multiple treatments across multiple cancers to determine which patient subsets may benefit the most from which treatments.
With adaptive trials, advocacy organizations find themselves moving from simply funding research projects to being at the center of these collaborations between industry and clinical research centers. In 2016, the LLS launched one such trial – the Beat AML® Master Clinical Trial, a multi-treatment, multi-site study to answer questions about targeted treatments for AML. The ultimate goal is to facilitate FDA approval of new AML therapies by exploring the feasibility of assigning a personalized therapy based upon genomics within a seven day period in a safe manner.
“As a result of all this progress in blood cancer research over the decades, we’re beginning to imagine using the word cure more and more often,” DeGennaro said. “And for the first time, I can see a research pathway that might be able to take us to prevention. Not tomorrow or in 10 years, but I can see that pathway and how we might begin to address blood cancer prevention. We have some exciting days ahead of us.”
To learn more about how a deeper understanding of disease differences may lead to more targeted treatments, read “Do Lymphoma Subtypes Make a Difference in Treatment Decisions?”