ACHIEVING THE IMPOSSIBLE: WHAT’S AHEAD IN 2018?
By Dr. Louis J. DeGennaro, LLS President and CEO | January, 2018
We’ve just concluded one of the most historic years on record in terms of new therapy approvals for blood cancers. In all, the U.S. Food and Drug Administration (FDA) approved 18 therapies to treat patients with blood cancers, including some entirely new agents and some new uses for already approved drugs.
Among these approvals were the first new therapies — four to be precise — for acute myeloid leukemia (AML) after a 40-year drought in treatment advances for this deadly blood cancer. And two revolutionary CAR (chimeric antigen receptor) T-cell immunotherapies, which reprogram the body’s own T cells to find and kill cancer cells were approved for patients with leukemia and lymphoma. I’m proud to say that The Leukemia & Lymphoma Society (LLS) played a role in all but three of these advancements.
These achievements demonstrate that we’ve reached a new era in cancer treatment, but these new therapies can’t save lives if patients don’t have access to them. Consider the impact of natural disasters such as Hurricanes Harvey, Irma and Maria on blood cancer patients, who faced added distress from the loss of property and medical care to due flooding and not being able to reach their treatment centers. As a patients first organization, LLS committed up to $1 million to provide direct support to impacted communities in 2017, helping more than 1,500 patients and their families during their time of great need.
Looking ahead, I expect the rapid pace of progress to accelerate this year, and LLS will continue to lead the charge to advance cutting-edge research to find cures and ensure patients have access to these therapies.
Here are my predictions for what we can expect this year.
1. CAR T-cell and other immunotherapies will continue to make headlines. As I noted, we saw two remarkable therapies approved that deliver genetically engineered immune cells to seek out and destroy cancer cells. The therapies are approved for young acute lymphoblastic leukemia (ALL) patients who have relapsed or did not respond to their treatment, and diffuse large B-cell lymphoma (DLBCL) patients who also relapsed or were nonresponsive to therapy. There will likely be more FDA approvals for these therapies in 2018. We might even see consideration of moving this therapy from last resort to earlier in the course of treatment, perhaps for high-risk patients. I expect to see significant progress in CAR T-cell therapies for the treatment of patients with myeloma. Clinical trial data presented at the recent American Society of Hematology (ASH) annual meeting showed high response rates for patients with myeloma, and we will see more pivotal data for myeloma patients in 2018. LLS recently announced its commitment of $46 million to new research grants, including support for the next phase of CAR T-cell immunotherapy — understanding why some patients respond and others don’t, making the process more powerful, safer, available to more patients with other types of cancers, including myeloma, and less cumbersome to manufacture. Currently, LLS has eight grant programs open for CAR T-cell therapies and 12 for other types of T-cell immunotherapies. I also anticipate that these approaches to therapy will also impact the treatment of other cancers, beyond the blood cancers.
2. The attack against AML will intensify. LLS continues to push the envelope in AML research. We are one year into our Beat AML Master Clinical Trial, and anticipate unveiling some results toward the end of the year. We launched the trial to change the paradigm of how this deadly blood cancer is treated. Advances in genomic technology have led to a greater understanding of genetic drivers of AML. We now know that it is not one disease, but a complex constellation of subtypes of the disease, and the one-size-fits-all model wasn’t working. With this trial, we’re taking a precision medicine approach, giving patients a targeted therapy appropriate for their subtype. This is a major collaboration: We now have more than 170 patients enrolled at seven leading cancer centers with more joining soon as well as six pharmaceutical companies and nine study arms on board.
3. Combination therapies will unlock powerful new treatment options. I expect to see more clinical trial data, and possibly more approvals, in combination therapies, for diseases like AML and chronic lymphocytic leukemia (CLL). Doctors have significantly more therapies in their arsenals to treat these cancers, but now they are trying to figure out the most effective cocktails to produce lasting remissions, maybe even cures.
4. Unique collaborations will continue to drive breakthroughs. We continue to partner with The Sarah Cannon Fund at The HCA Foundation to fight another deadly blood cancer, mantle cell lymphoma. With support from Sarah Cannon, we’ve awarded $5 million to two multidisciplinary, cutting-edge research teams studying combination therapies for this subtype of lymphoma. Read more about the collaboration here. In 2018, with our Beat AML trial as a model, we will also pursue a collaborative initiative to get new agents approved for pediatric cancer patients.
5. Patient support will evolve in new ways. We are redoubling our efforts to help more patients sooner and ensure they can afford their treatments. It’s all about connectivity. Our goal is to reach blood cancer patients within 90 days of their diagnosis through our Information Resource Center, a toll-free call center staffed by healthcare professionals armed with the most up-to-date information about blood cancers, treatment options and other critical resources for patients and their families. Additionally, we’re expanding our Clinical Trial Support Center — staffed with registered nurses with expertise in blood cancers, who work one-on-one with patients and caregivers to search for appropriate clinical trials. We are also pursuing initiatives to increase our outreach to and support of cancer survivors. One way we’re doing this is by enhancing LLS Community, an interactive online network enabling blood cancer patients to connect with one another and share information. The data collected through LLS Community can serve as a resource for public health/outcomes research on important issues such as late effects of treatment, prevention research, or patient reported outcomes.
6. Your voice will be more important than ever. Our policy and advocacy team in Washington D.C. and across the U.S. continue their work to influence state and federal laws and regulations to ensure patients can access the care and treatments they need at an affordable price. We are committed to defending vital insurance protections that cancer patients rely on to access their treatments at a reasonable cost, and advocating for laws and rules to lower patient out of pocket costs for expensive but potentially lifesaving therapies. For example, we’ve helped get “oral parity” laws passed in 43 status plus Washington D.C. — ensuring patients who get their cancer therapies from a pharmacy and take them orally receive equitable insurance coverage with patients who receive their cancer treatments intravenously in a clinical setting. We are setting our sights on four more states in 2018. Whether you are a patient, caregiver or proud supporter of LLS, your voice can make all the difference in bringing about change.
7. Vital conversations will take place to address the cost of cancer care. In 2017 LLS issued a position statement on the cost of cancer care, calling upon all stakeholders in the healthcare ecosystem — drug, diagnostic and device manufacturers, healthcare providers, hospitals, pharmacy benefit managers, patient organizations and federal and state regulators — to work together to put patients first by enacting reforms and adopting policies to reduce costs and financial distress for cancer patients. In 2018, we intend to continue leading this conversation, and have been convening meetings with representatives of the different stakeholder groups to assess areas of consensus we intend to pursue to reduce system costs.
We are hopeful that 2018 will be just as impactful, if not more so, for the blood cancer community and beyond as last year. Follow The LLS Blog for more updates throughout the year and know that none of this progress would be possible without you. Please consider making a donation to LLS to help us achieve our goal of a world without blood cancers.