We’ve come a long way since our founding in 1949, when blood cancer was uniformly fatal and poorly understood.
Today, our growing understanding of the mechanisms of cancer and in particular of the genetic basis of many blood (and other) cancers has allowed us to personalize treatment for many patients, improving survival rates and the quality of life for patients with many forms of cancer.
Most people with Hodgkin lymphoma and children with acute lymphoblastic leukemia are cured today. People with chronic myeloid leukemia and chronic lymphocytic leukemia (CLL) often have long-term remissions, and some may even achieve “functional cures” with no or very limited treatment for many years.
These are some big wins. We are working tirelessly to improve outcomes for even more people with blood cancer.
Today, Blood Cancer United has two main goals for the research we fund. The search for cures for every form of blood cancer continues. But with so many patients living years, decades or even a lifetime with blood cancer, we also want treatment to meet patients where they want to be, improving their quality of life during and after treatment.
ASH 2025: A chance to reflect on how far we’ve come, and see where we’re going
Every December, thousands of blood cancer scientists and experts come together for the American Society of Hematology (ASH) annual meeting. With Blood Cancer United being the largest nonprofit funder of blood cancer research, this is a once-a-year opportunity to showcase our work on master trials and with many partners from the nonprofit and for-profit sector. It’s also a great opportunity to talk with other scientists about cutting-edge research around the world.
Here are five takeaways from this year’s ASH meeting about how treatment options will continue to improve outcomes and quality of life for patients and their families.
New frontline therapies will give patients and their doctors more choices
For decades, chemotherapy was the first treatment for nearly everyone with blood cancer. While chemo still has a place for many patients, immunotherapy and targeted therapies are the main types of new treatments being approved today.
We expect many new frontline treatments for a range of blood cancers in the coming years. One example discussed at ASH is a chemo-free combination of a tyrosine kinase engager (ponatinib) and a bispecific T-cell engager (blinatumomab) that could become the standard of care for patients with Philadelphia chromosome-positive acute lymphoblastic leukemia.
We also heard more about the effectiveness and very good safety profile of the relatively new non-covalent BTK inhibitor called pirtobrutinib for first-line treatment for chronic lymphocytic leukemia. This could be a game changer, especially for older and frail patients who are generally less tolerant of side effects.
With more types of drugs than ever available to treat CLL, the other question will be in which order to use them. If a patient starts with pirtobrutinib, will the other BTK “-tinib” type drugs be effective later? Scientists are working on those answers.
Another big leap could be menin inhibitor combinations for people with acute myeloid leukemia. Menin inhibitors are already approved as monotherapy for people whose AML has relapsed or not responded to initial treatment. Combinations are being tested that could become first-line treatment of choice for some types of this very aggressive and fast-moving cancer that still leads to poor outcomes for many patients.
Shorter treatment times could offer efficacy with fewer side effects and less cost than continuous treatment
Another exciting theme emerging from ASH is more exploration of time-limited treatments. Chemotherapy is nearly always used for a limited time, but that’s because of its long-term toxicity.
Newer targeted treatments usually come with fewer side effects, and they are sometimes given continuously, stopping only if the cancer starts progressing again.
But fewer side effects don’t mean no side effects. Being able to stop treatment to reduce side effects while maintaining efficacy would be everyone’s first choice.
We heard new data at ASH about bispecific antibodies (more on that next) being used for 6-12 months versus forever in people with multiple myeloma. These time-limited treatments, particularly when they consist of combinations of different drugs, appear to offer the same sustained remissions as continuous therapy.
Another important relatively new area of research are so-called ‘de-escalation’ trials that study whether we can use less of an approved treatment, a shorter duration or less frequent dosing and achieve the same activity against cancer but reduce side effects (and potentially the cost of treatment).
Opti-AML, a study that is part of our Beat AML® master trial, is looking at whether a reduction in venetoclax treatment, used in combination with azacitidine to treat acute myeloid leukemia, can be just as effective but with fewer side effects. This could mean fewer dosing interruptions, treatment-related infections, transfusions, and hospital stays for people facing AML.
Data from another study presented at ASH is expected to be practice-changing for AML. The study showed that the combination of azacitidine and venetoclax, which is currently the standard of care for elderly, unfit patients, is also highly effective in younger patients compared with their standard treatment, intensive chemotherapy. Importantly, azacitidine plus venetoclax was associated with fewer serious infections, better quality of life, and less time in the hospital.
Bi- and tri-specific treatments continue to gain traction
Bispecific and tri-specific therapies work by engaging two (bi) or three (tri) genetic targets to help patients’ immune systems recognize and kill cancer cells. We’re seeing “rapid uptake and steady growth” of bispecifics for multiple myeloma, according to a report from Oncology News Central.
These therapies come with clear benefits. They can be used “off the shelf” and don’t require the complex and often time-consuming manufacturing process that’s required for CAR T. That’s time some patients don’t have. Bispecific therapies can also be administered in many types of hospital settings, while CAR T therapy typically requires patients to travel to one of the limited numbers of highly specialized cancer centers.
New combination treatments aim to improve effectiveness
Step one for any new treatment in development is to test it as monotherapy. This is the only way to understand its specific effectiveness and safety. But once those questions are answered, clinician scientists test combinations of the new drug with others. Combination therapies are often more powerful because combinations can attack cancer cells in several ways simultaneously.
At ASH we heard about several studies that are examples of this approach. A clinical trial called Majestic-3 is testing a combination of two immunotherapies (teclistamab and daratumumab) in people whose disease returned after their initial treatment for multiple myeloma. The two-drug combination shows strikingly improved progression-free survival compared to standard-of-care, and early data suggest that it might also improve overall survival.
In November, the FDA approved the bispecific antibody epcoritamab in combination with rituximab and lenalidomide for people with follicular lymphoma that has returned or worsened despite prior treatment. Study results presented at ASH demonstrated that this triple combination was superior to rituximab plus lenalidomide alone in reducing the risk of disease progression or death by almost 80%. Importantly, this new treatment option can be given in an outpatient setting.
Another example of an effective combination treatment was presented for CLL. A large clinical study demonstrated that a fixed-duration treatment with a combination containing venetoclax was as effective as continuous treatment with ibrutinib alone. This finding supports another treatment option for patients who prefer not to be on a generally well tolerated, but indefinite treatment.
New medications will speed up and improve access to treatment
Just six months ago we heard exciting news about long-term remissions and even the possibility of a cure for some people with multiple myeloma who received a one-time infusion with CAR T-immunotherapy. This personalized immunotherapy has been a game changer for some, but not everyone benefits.
The CAR T manufacturing process can take months. T-cells need to be removed from a patient’s blood, sent to labs for modification, grown for a time, and then reinfused. People with advanced or aggressive cancer may not be able to wait.
Now a new type of CAR treatment may change this. It’s called “in vivo,” meaning the “manufacturing” happens inside a patient’s body instead of in the lab (“‘n vitro”). A specialized “vector” allows the treatment to find, enter, and modify T cells inside the body to make them more effective killers of cancer cells. Very early results in four patients with advanced multiple myeloma showed that all responded favorably to the treatment with limited side effects.
Research is the key to maintaining momentum for more patients
Many exciting advances are already changing how we treat blood cancers. But more research is needed to extend these benefits to all people with blood cancer. Our continued investment in preclinical and clinical research is critical to maintain this great positive momentum and ultimately achieve cures for all blood cancers.
About the author
Lore Gruenbaum, Ph.D., is the chief scientific officer of Blood Cancer United, a global leader in blood cancer research and support.
Dr. Gruenbaum and her team set the Blood Cancer United research investment strategy. Their aim is to improve survival and quality of life for patients with every form of blood cancer.
Dr. Gruenbaum joined in 2020 as vice president for the Therapy Acceleration Program (TAP), a strategic venture philanthropy initiative that seeks to accelerate the development of innovative blood cancer therapeutics and change the standard of care, while also generating a return on investment for Blood Cancer United’s mission. In 2023, Dr. Gruenbaum took on oversight of the Beat AML® Master Clinical Trial and implementation of a new strategy to reach additional patient populations.
Dr. Gruenbaum received her Ph.D. in biochemistry (summa cum laude) in her native Germany, moving to the United States more than 25 years ago to complete her postdoctoral research at Yale University.
