The next wave: Exploring diverse research platforms to bring transformational options to patients with blood disorders

Hematologic malignances are a complex and diverse group of diseases, with more than 100 distinct types. Each requires a nuanced approach to patient care and treatment based upon where it forms in the body or how quickly it grows. 

Scientific advances in hematology over the past few decades - many of them pioneered by researchers and scientists at BMS - have changed survival expectations for people with certain blood cancers like multiple myeloma and have reduced the burden from blood transfusions for people with anemia. Still, many blood disorders cannot be cured or have not seen new treatment options for years and place a debilitating burden on a patient’s quality of life.

We’ve asked Ridwaan Jhetam, senior vice president of WW Medical Affairs Hematology, and Rosanna Ricafort, vice president, head of Late Development Hematology and Cell Therapy about the range of novel modalities and research platforms BMS is exploring through its robust pipeline while working to deliver the “next wave” of advances in hematology and cell therapy.

How does BMS prioritize the development of treatment options across hematologic disease states?

Ridwaan: What’s fascinating to me about blood cancers is not just the breadth of the diseases themselves, but the distinct patient needs within each one. For example, there are more than a dozen subtypes of acute myeloid leukemia each based on genetic abnormalities or the percentage of immature white blood cells (myeloblasts) in the bone marrow and blood. With certain types of leukemia or lymphoma, the primary goal of treatment may be delaying progression. On the other hand, treatment in multiple myeloma may be concentrated on extending a patient’s survival following a relapse. We are focusing our science and attention on where we can address whatever those most urgent needs for patients are, to make a transformational impact with our novel medicines or improved technologies. This often means prioritizing areas that may have been without innovation or seen little progress in years, as we did to address anemia in patients with diseases such as myelodysplastic syndrome (MDS) or beta thalassemia.

Rosanna: The only way we can keep a pulse on evolving patient needs is by keeping the patient voice at the center of all that we do. Throughout our clinical development process, we stay closely connected with our patient advocacy team, researchers, and physicians in the field, to better understand what people living with blood cancer are experiencing and where they need most support. We take those learnings, combine them with the decades of scientific and clinical expertise we’ve gained over the years in hematology and work to develop medicines that can help improve outcomes and quality of life for even more patients.

Tell us more about some of the key research platforms BMS is exploring.

Rosanna: We have a robust hematology pipeline with more than 25 programs in clinical development. More than half of these are in late-stage trials, covering multiple research platforms and combinations, particularly in cell therapy. I have been focused in the field of cell therapy for more than two decades, and it’s been amazing to be part of the transformative progress in this area. But we’re still just scratching the surface of the full potential of cell therapies and looking to move beyond blood cancers into areas such as severe lupus and multiple sclerosis and help even more patients with this technology.

Ridwaan: I’m particularly excited about our research in targeted protein degradation and its potential in the treatment of anemia in several disease areas. We’ve seen promising activity with our investigational, novel CELMoD™ agents in multiple myeloma and types of non-Hodgkin lymphoma both in the newly diagnosed and relapsed or refractory settings, with more areas of focus on the way. We’re encouraged by the growing body of evidence for the assets in this platform, which were purposefully designed for improved potency and combinability. We continue to evaluate the potential of our first-in-class erythroid maturation agent in additional areas like myelofibrosis, where anemia places a significant burden on patients who may require frequent blood transfusions. We continue to evaluate the potential of our first-in class erythroid maturation agent in additional areas like myelofibrosis, where anemia places a significant burden on patients who may require frequent blood transfusions.

What does the future of hematology and cell therapy innovation look like to you?

Ridwaan: We have delivered many firsts in hematology and cell therapy, but our eyes are set on what’s next and what has the potential to be best for patients, such as evaluating specific biomarkers and precision medicine approaches to drive more personalized care. We also have an integrated framework to improve recruitment of diverse patients in our clinical trials, to help ensure that participation is more reflective of the real-world population living with these diseases. We’re continuing to explore ways to leverage the latest technologies in artificial intelligence and machine learning, to understand how these can play a more meaningful role in the drug discovery, development and treatment decision-making process for patients.

Rosanna: We know that this vital work cannot be done alone: we are partnering across a complex and interconnected ecosystem that includes researchers, treaters and the advocacy community, all for the purpose of reaching patients and their families. Together, we are invested in propelling the science forward and envision a future where novel therapies and approaches can help create longer, better lives for patients with blood diseases.