Progress in new cancer treatments is accelerating so rapidly that the standard of care for many cancer patients is changing right before our very eyes.
Since 2017, the U.S. Food and Drug Administration (FDA) has approved a remarkable 53 therapies just to treat patients with blood cancers, and The Leukemia & Lymphoma Society (LLS) has helped advance 46 of these treatments.
I have no reason to believe the next few years won’t be as productive and groundbreaking as the last few. With that, here are some of my predictions for 2020:
CAR T-cell Immunotherapy Continues to Amaze
Revolutionary advances in harnessing the body’s immune system to seek out and destroy cancer cells, is creating excitement about chimeric antigen receptor (CAR) T-cell immunotherapy, a treatment that engineers the patient’s own cells to fight cancer. While the treatment is currently FDA approved for two types of cancer - acute lymphoblastic leukemia (ALL) and large B-cell lymphoma - I predict we will see CAR-T approved this year for patients with mantle cell lymphoma and multiple myeloma. Compelling data for both of these blood cancers was presented at the American Society of Hematology meeting in December. I’m especially excited about so called “off-the-shelf immunotherapy” that doesn’t require engineering individual patients’ T cells, making manufacture of these cells less costly and time consuming.
More Precision Medicine/Less Chemotherapy
Precision medicine - giving patients a drug based on their molecular profile rather than taking a one-size-fits-all approach - is showing great promise in acute myeloid leukemia (AML) and chronic lymphocytic leukemia (CLL). While chemotherapy, drugs that directly kill cells, remains an important component of many treatment regimens, I predict we will see less reliance on these types of drugs as more targeted therapies, drugs that work by interfering with molecules that help drive cancer growth, gain approval.
More Help for Children with Cancer
For too long we’ve been treating children with acute leukemia with the same protocols developed more than 30 years ago. While most children with ALL survive with treatment, the harsh chemotherapy combinations leave many with lasting side effects. We can and will do better for these children. With the success of our Beat AML Master Clinical Trial, a precision medicine study for adults with AML, we are now planning a global precision medicine trial for children with AML. To develop new therapies for the 40% of children and young adults who don’t respond to treatment, we aim to launch our trial – LLS PedAL – in the summer of 2020.
As well, we’ve more than doubled our commitment to pediatric research grants, new services for children with cancer and their families, including our best-in-class Information Resource Center and Clinical Trials Support services, with trained medical specialists ready to help patients, families and caregivers navigate a cancer diagnosis and treatment, including enrolling in an appropriate clinical trial.
More Collaborations to Help More Patients
LLS is a global leader in the fight against cancer but we can’t do it alone. Nonprofit, government, industry and the business sector all must work together to solve big problems.
LLS is a trusted convener, and we will continue to facilitate collaboration across partners such as consumer companies such as Walgreens and Burlington, pharma and bio companies such as Bristol-Myers Squibb, Pfizer and Johnson & Johnson, professional organizations such as The American Society of Hematology (ASH) and Milken Faster Cures, and the Paul G. Allen Frontiers Group and The Mark Foundation for Cancer Research, to fund cutting edge research to advance more breakthrough, novel cancer treatments in 2020.
Improving Access to Treatments and Care
All of the scientific progress doesn’t mean a thing if patients can’t access the treatments. One of LLS’s priorities is increasing participation in clinical trials and we will continue to approach this challenge from many directions in 2020.
Our collaboration with ASH will help inform more patients about appropriate clinical trials. As well, we will be awarding grants under a new program called IMPACT (Impactful Medicine Providing Access to Clinical Trials), to expand access to high-quality clinical trials to patients served by community-based sites of care, particularly rural, minority, and/or economically disadvantaged blood cancer patients.
We will continue to collaborate with all of the players in the healthcare ecosystem to attack the high cost of cancer care for patients. Financial toxicity and quality of life will be the focus of new research with patients at the center.
We aim to get two policies on the president’s desk by late May - a cap on out-of-pocket costs to patients in Medicare Part D, along with an initiative known as “smoothing,” which would spread out the cost to patients in Medicare so they are not hit with a very high upfront payment.
On the state level, we are fighting back against “junk plans” in 13 states. These insurance plans don’t adequately provide coverage, particularly for patients with preexisting conditions. We will be issuing a study in February showing the impact of these plans.
As you can see, we’ve got our work cut out for us in 2020 and we’ll all need to stay focused as we deliver our mission for patients and families.
As the 61st Annual ASH (American Society of Hematology) Meeting and Exhibition winds down here in Orlando, here are some thoughts on where things stand in the world of blood cancers.
New targeted therapies approved in just the past two years alone are making precision medicine more of a reality for acute myeloid leukemia (AML). As I noted in a previous blog our own Amy Burd, Ph.D., LLS vice president of research strategy, presented compelling data that precision medicine, giving a patient a drug based on their molecular profile rather than one-size fits all, is bringing a paradigm change in how AML patients will be treated moving forward.
For patients with chronic lymphocytic leukemia (CLL), we’re seeing a decided shift toward chemo-free combinations of targeted medicines, opening a debate about whether the old standard, chemotherapy plus an antibody, will be replaced by a new regimen. Studies are showing that combinations of targeted therapies that work by blocking the activity of bad-acting genes, proteins or cellular pathways are quickly pushing their way to the front of the line for treatment of CLL. One study presented at the meeting showed that two targeted oral therapies given together, venetoclax and ibrutinib, resulted in deep remissions for CLL patients, with 75% having high rates of undetectable minimal residual disease, meaning no traces of cancer cells could be found with highly sensitive tests. LLS didn’t fund this study, but has invested millions over two decades to advance both of these therapies.
Further, with the advent of CAR T-cell immunotherapy and bispecific antibodies, both once the stuff of science fiction but now increasingly becoming mainstays of treatment with remarkable results, it’s clear we are experiencing a seismic shift in the standard of care for many patients with leukemia, lymphoma and myeloma.
Since coming on the scene, CAR-T has emerged as a miracle treatment that has brought patients from the brink of death to enduring remissions. But CAR-T doesn’t work for everyone and even many patients who initially respond, can relapse.
Enter mosunetuzumab. This new investigational drug is considered so game-changing for patients whose lymphoma has returned following treatment that it was featured during the plenary session, held in the biggest hall in the conference center to accommodate thousands of attendees. Mosunetuzumab is a bispecific antibody, meaning it binds two proteins, one on the surface of tumor cells (CD20) and the other on the surface of the recipient’s T cells (CD3).
The data presented in the plenary showed that of 124 patients with fast moving, aggressive lymphomas, 46 patients (37%) saw a decrease in the amount of cancer, with 24 (19%) achieving complete remission. Those with less aggressive disease still saw results; a reduction of disease was seen in 42 patients (63%), with 29 (43%) achieving complete remission as well. Among 18 patients whose lymphoma progressed after treatment with CAR-T, 22% experienced complete remissions. The remissions for these patients appear to be long-lasting.
The Leukemia & Lymphoma Society (LLS) launched our Beat AML® master clinical trial in 2016 to revolutionize how we treat patients with acute myeloid leukemia. After forty years of basically doing the same thing and treating AML patients with a one-size-fits-all approach despite it being a disease of multiple subtypes, it was time to try something new. Our study is testing multiple drugs simultaneously at 16 cancer centers around the U.S., using genomic technology to give AML patients the option to try a therapy that has a better chance of working for the specific mutation driving their diagnosis.
We are here in Orlando at the 61st ASH Annual Meeting, and yesterday Amy Burd, Ph.D., vice president of research strategy for LLS, presented an update from the Beat AML trial. The key takeaway: patients on the trial generally do better than patients who opt for standard chemotherapy.
According to Dr. Burd: “We have demonstrated that a precision medicine approach to assigning treatment within seven days is feasible and safe. Patients who elected the assigned treatment had a lower early death rate and superior overall survival compared to patients who elected standard of care. The data suggests that this umbrella approach has the potential to improve both short and long term outcomes in AM, and sets the stage for a precision medicine approach in other blood cancers.”
More AML News
Much discussion here at #ASH19 is focused on a drug called venetoclax, considered by many to be a game-changer for many forms of blood cancer. LLS has supported advances in venetoclax over the past two decades, helping to lead to its approval for chronic lymphocytic leukemia. The U.S. Food and Drug Administration approved it in 2018 for AML. Multiple studies presented at the meeting examine venetoclax in combination with other therapies for a variety of blood cancers, including AML.
One study by a team from University of Colorado, including Craig Jordan, MD, who leads a $5 million Specialized Center of Research grant from LLS, looks at why some patients with AML are resistant to venetoclax. Recent clinical trials report that more than 70% of previously untreated older AML patients respond to the combination of venetoclax with the chemotherapy azaciticine, but a subset of AML patients do not respond. The team’s study shows that AML patients with a prevalence of a type of white blood cell called a monocyte do not respond to this combination and clearly new strategies are needed for this subset of patients.
After a 40 year drought in new therapies for AML, several new drugs that target specific mutations were approved by the FDA from 2017 to 2019, potentially improving outcomes for certain subsets of AML patients. However, more novel targets are still needed. LLS-funded researcher, Xiangguo Shi, Ph.D., of Baylor College of Medicine, is presenting a study Monday at the ASH meeting discussing the role of a gene called NMNAT1 (nicotinamide nucleotide adenylyltransferase 1) in AML. The study shows that developing drugs that inhibit this gene’s activity warrant further consideration.