More treatments for more patients and the growing impact of AI on health: A report from recent medical conferences

Blood Cancer United was front and center at two major medical conferences recently, the American Society of Clinical Oncology (ASCO) and the European Hematology Association (EHA). These yearly meetings are an opportunity for our team to share and learn from others about advances that can benefit patients with blood cancer and their families.  

We have seen a range of new treatments emerge in recent years giving many patients and their healthcare providers more treatment options and even the ability to pick a therapy that aligns with each patient’s treatment goals and personal life priorities.  

We are also seeing an increased focus on therapies that are better tolerated, more convenient to take, and that provide better quality of life. And the potential to achieve cures is on the horizon for many more blood cancers. 

Here are a few takeaways from the conferences. 

Immunotherapies are changing the therapeutic landscape for multiple myeloma and many types of lymphoma.  

Our understanding of immunotherapy has grown immensely since the first FDA immunotherapy approvals, which were only for patients who had exhausted all other available treatment options. We now know that a significant percentage of patients who receive CAR-T cell therapy, a personalized type of immunotherapy, can be cured. We are also now able to use these therapies safely at earlier times in a patient’s treatment history.  

Early work on CAR-T helped in the development dual-targeting bispecific antibodies called “bispecifics.” We currently have nine FDA-approved bispecifics for blood cancers, and these approved drugs are now being tested for even more patients at earlier stages of disease, including in many cases for newly diagnosed patients.  

Researchers are also looking at combining bispecifics with each other or with other therapies. In clinical studies for myeloma and lymphoma, these combinations are leading to very high response rates that are also “deep,” which means very few or no cancer cells can be detected after treatment, including for patients with higher-risk forms of disease. 

One last exciting development around immunotherapy: ‘in vivo CAR’. These CAR-T cells are generated directly in the patient’s body – without the need for the multi-week, complex CAR T-cell manufacturing process. It’s still early days, but the data from several ongoing, small clinical trials is very encouraging with high response rates and so far, good safety.  

New targeted therapies are becoming available even for the hardest to treat blood cancers.  

Excitement continues around Menin inhibitors, a new treatment option for people whose acute myeloid leukemia (AML) has molecular changes in genes called NPM1 and KMT2. Blood Cancer United provided years of funding support for menin research, including direct support for early work towards a recently approved menin inhibitor treatment called ziftomenib.  

We are continuing to drive progress in this field through our Master Clinical Trials (Beat AML in adults and PedAL in children), which are testing menin inhibitors for types of patients who were not included in the initial FDA approvals.  

We are seeing a similar picture here as with immunotherapies: initial FDA approvals were for patients with advanced disease and extensive prior treatments, but the value of these therapies, in particular in combination with approved therapies, will likely be much broader including for newly diagnosed patients, patients who had bone marrow transplants, and patients with other blood cancers.  

AI is having a positive impact on both basic research and clinical development.  

AI is being used to make sense of the big data sets we are now able to acquire. AI models can help identify new drug targets, design new drugs that work on those targets, and accelerate the early stages of research that precede drug evaluation in patients.  

At both ASCO and EHA, we also saw how AI is being used to improve cancer diagnostics and predict a patient’s course of disease – ultimately as a tool to support physicians and patients as they are making treatment decisions. It is of the utmost importance that we use AI responsibly, and as our medical director has written, “ensure that AI-driven health care remains ethically sound, equitable, and patient-centric.” 

We also heard many promising examples of how AI can help with clinical trials: patient education & recruitment, communication with trial sites, data management, and interactions with regulators. Our use of AI in our Beat AML Master Trial, which was supported by a recent grant from Amazon Web Services, is saving time and money, allowing us to communicate faster and more effectively with FDA, and ultimately move our studies forward more quickly. 

Now is the time to redouble our efforts.

We need a strong, continued investment in basic research and clinical trials to keep this progress going. We also need to keep fighting for better access for all cancer patients, which will turn more blood cancers into chronic conditions and ultimately lead to cures even for patients with aggressive and advanced stage disease. Blood Cancer United will be here, investing in research and the next generation of scientific leaders so that the many promising efforts we see today can continue and improve more patient lives.