Leukemia and Lymphoma: What Have We Learned?
Summary
- Sylvester Comprehensive Cancer Center is at the forefront of new leukemia research, drug development and clinical trials.
- The leukemia program at Sylvester has led more than 50 clinical trials in the past decade.
- The work done at Sylvester has led to the FDA approval of three new drugs to treat leukemia or related conditions in the past two years.
Over the past two decades, breakthroughs in cancer genetics and new cancer medicines have transformed how hematologists treat blood cancers. With one of the most extensive leukemia programs in the country, Sylvester Comprehensive Cancer Center, part of the University of Miami Miller School of Medicine, is at the forefront of new research, drug development and clinical trials.
“Clinical trials produce paradigm shifting research creating the new standards of patient care, where researchers, clinicians and patients come together to turn discovery into progress and bring the future of cancer care within reach,” said Jonathan Trent, M.D., Ph.D., associate director for clinical research at Sylvester and a Miller School professor of medical oncology.
Dr. Trent added the future of cancer care is being rewritten every day with precision medicine, immunotherapy and data-driven discovery transforming how physician-scientists prevent, detect and treat cancer.
“We have one of the best early-phase clinical trial programs in leukemia in the world,” said Mikkael Sekeres, M.D., M.S., Sylvester’s chief of the Division of Hematology. “Because we’re an NCI-designated cancer center, we have access to clinical trials that others don’t. And we have internationally renowned investigators leading them.”
The Danger of Blood Cancers
Cancers like leukemia, lymphoma myelodysplastic syndromes (MDS) and myeloproliferative neoplasms start when blood cells grow out of control. They can affect people of all ages and are particularly deadly. Many blood cancers relapse after treatment or do not respond to treatment at all.
Acute myeloid leukemia (AML), for instance, has a five-year survival rate of only about 30%. Half of patients relapse within five years.
“AML is a devastating disease. Thankfully, there have been over 10 new drug approvals in the last decade. The clinical trials and translational work at Sylvester has been instrumental in that effort,” said Justin Watts, M.D., Sylvester’s chief of the leukemia section in the Division of Hematology. “It is an exciting time to be here at Sylvester. We’re doing cutting-edge research that’s changing lives for patients with leukemia.”
The leukemia program at Sylvester has led more than 50 clinical trials in the past decade. Sylvester’s research is improving patients’ lives and finding cures.
“We are the only program in the region that is offering these early-phase clinical trials and bringing access to these new lines of treatment to our community,” said Maria “Ken” Figueroa, M.D., Sylvester’s associate director of translational research and a professor of biochemistry and molecular biology at the Miller School. “New treatment options are essential for patients that have failed more than one line of care or have rare diseases that don’t have established treatments.”
In an enduring effort to advance translational hematology research, Sylvester holds a leukemia symposium every two years and is set to open the new Griffin Cancer Research Building. This new building brings cancer research, from basic research labs to clinical trials and patient care, into one building.
“The work that we’ve done here at Sylvester has led to the FDA approval of three new drugs to treat leukemia or related conditions in the past two years,” Dr. Sekeres said. “Patients at Sylvester enrolled in those clinical trials have actually already had access to these new drugs for years.”
Dr. Watts led the development of olutasidenib, which treats patients with relapsed or refractory AML. It targets a genetic mutation called IDH1 in the leukemia cells, slowing their growth and restoring normal blood cell function. It is especially important for older adults with AML who have not responded to other treatments.
“As we’ve become better at understanding the genetics of cancer, we’ve also become better at identifying therapies that take advantage of those gene differences,” said Dr. Sekeres. “This has led to the development of targeted therapies.”
Another new targeted therapy, quizartinib, targets the mutation FLT3-ITD found in many AML patients. These mutations are linked to more aggressive disease and worse outcomes. These patients often have limited and less effective conventional treatment options, said Dr. Sekeres, who led its development.
The third, imetelstat, is a first-of-its-kind treatment that blocks telomerase, an enzyme that helps leukemia cells survive and grow. It is a promising new option for patients with MDS, a common blood cancer in older adults that can lead to AML.
Many other clinical trials at Sylvester have led to new standards of AML treatment. With six clinical investigators, Sylvester researchers typically have 20 to 30 trials for leukemia and MDS going at once, and several physician-scientists focused on translational work, bringing research discoveries from the laboratory to patients.
“Translation is not linear, even though we use the term bench to bedside. It’s really bench to bedside to the community and back,” Dr. Figueroa said. “Everything we do is through different stages of that lens. Some projects are years away from patients, and others are on the cusp of a clinical trial, or they are a clinical trial. Some are testing how new treatments perform in the real world.”
Hematologists from Sylvester and around the world will present at the 4th Biennial Miami Leukemia Symposium in April, highlighting the most significant developments in leukemia research.
“It’s all the leukemia experts in one place for great discussion, insight and new collaborations,” Dr. Watts said.
The Next Generation of Sylvester Cancer Research
One of the most promising areas of research, Dr. Watts said, is projects using triplet therapies. Triplet therapies are combinations of three treatments that act on different pathways to attack the cancer more effectively. Dr. Watts hopes triplet therapies can prevent or delay resistance so that treatments work the first time and stay effective for a long time.
Other trials focus on cutting-edge immunotherapies, including bispecific T-cell engagers or BiTE antibodies. These innovative molecules are designed to bind cancer-killing immune cells to tumor cells, prompting the T cell to kill the cancer.
Sylvester is investing in these new cancer treatments and driving the next generation of clinical trials. This work will happen at the Griffin Cancer Research Building, opening in fall 2025.
“This large, new, state-of-the-art facility is built specifically for clinical research. That is what you need to make breakthroughs,” Dr. Watts said. “It brings clinical, translational and basic scientists all in one place, getting us together as a community and sparking innovations.”
The building will provide everything patients and researchers in clinical trials need, including biopsies, imaging and labs. It’s all housed in one structure.
“At Sylvester and in the Griffin Cancer Research Building, we’re leading the research that is creating the treatments of tomorrow,” Dr. Sekeres said. “This is incredibly exciting for physicians and gives us new hope for patients.”
Tags: Acute Myeloid Leukemia, blood cancers, cancer research, clinical trials, Dr. Jonathan Trent, Dr. Justin Watts, Dr. Maria Figueroa, Dr. Mikkael Sekeres, myelodysplastic syndromes, Sylvester Comprehensive Cancer Center