Blood cancers pose a significant threat to humanity, claiming numerous lives relentlessly. In our recent Q&A with Lore Gruenbaum, VP of the Research Therapy Acceleration Program at The Leukemia & Lymphoma Society, we delved into the multifaceted response to this global crisis. We discussed advancements in treatment and envisioned future possibilities. Lore emphasized The Leukemia & Lymphoma Society's pivotal role in driving research, having supported, in some way, nearly every major FDA-approved breakthrough in blood cancer research over the past two decades with substantial investments.

#LBS: To what extent are governments, medical organizations, and other stakeholders adequately responding to the global challenge posed by blood cancers, a fatal disease that claims countless lives annually?

Lore Gruenbaum: Blood cancer is not just one disease but rather a myriad of different diseases grouped in broad categories including leukemia, lymphoma, myeloma and other less common forms of the disease. Until about ten years ago, treatment options for these diseases were very limited with chemotherapy and, for some indications, radiotherapy as the only options.

Fortunately, this picture is changing.  Since 2017, a wave of new drug approvals, including precision medicines, antibody-based therapies, and cell therapies have dramatically increased survival rates and, in some cases, turned previously deadly cancers into manageable chronic diseases. Most famously, Chimeric Antigen Receptor (CAR) T-cell therapies have achieved cures for subsets of patients, and these therapies are now being explored in additional patient populations and diseases other than blood cancer.

Close interactions between academic researchers, biopharma, regulators, medical and patient organization have been critical to support the fast progression of clinical candidates through the different stages of development to approval. Funding from a range of organizations, including governments, private foundations and public charities has played a critical role for the advancement of very innovative approaches, that were initially viewed as too risky by professional investors and pharma stakeholders.

Nevertheless, more work remains to be done. Some blood cancers still lack satisfactory treatment options. Many of them are extremely rare and therefore not attractive to large commercial drug developers. And many patients still relapse after initially achieving responses to treatment. We need to continue to grow our understanding of the mechanisms of disease and relapse and our arsenal of treatment options. RNA-based therapeutics and gene/base editing approaches are just two new approaches that are being actively pursued by academic researchers, biotech, and pharma companies.

Moreover, we need to educate physicians and patients on novel treatment options and create the infrastructures to bring these therapies to the patients who need them. In many countries, innovative drugs are not available or not affordable for patients.

Continuous extensive work at the global as well as the local level—including ongoing collaboration between governments, medical organizations and other stakeholders—will be required to ensure that patients can obtain the treatments they need.

#LBS: How has The Leukemia & Lymphoma Society contributed to advancing treatment options for blood cancers, particularly in transitioning from clinical stages to approval? Could you provide some examples of your success stories and elaborate on the potential impact these achievements have had?

Lore Gruenbaum: LLS has invested more than $1.7 billion in scientific research over almost 75 years, supporting in some way, almost every major breakthrough in blood cancer research including 70% of the blood cancer treatments approved by the U.S. Food and Drug Administration (FDA) over the last two decades.

Our investment in all stages of research empowers the scientists and biotech companies we support to take risks, think big and test bold ideas. To give an example, LLS recognized the promise of CAR T-immunotherapy already in the 1990s, and through long-term and multi-faceted investments, supported the work leading all the way to the first approval of this revolutionary therapy that is saving lives today.

LLS continues to provide funding for immunotherapy, with more than $79 million committed to research into next generation of immunotherapy treatments. We hope to find ways to make these therapies safer and more effective, all while lowering costs and other barriers to access.

More than one-quarter of LLS’s research funding is dedicated to acute myeloid leukemia (AML), a hard-to-treat and aggressive blood cancer. LLS’s strategic focus on AML is helping researchers and companies uncover new precision treatments that can be matched to different types of the disease, and to uncover the root causes of these types of cancer. An example of LLS’s longstanding investment in AML research is support leading to the discovery of FLT3-ITD, a genetic mutation found in about 25% of AML patients. This research was foundational for the 2023 FDA approval of quizartinib, the first drug designed to target this mutation. Clinical trial participants who received quizartinib plus chemotherapy lived longer compared to those who received traditional chemotherapy treatment.

Our venture philanthropy initiative, the Therapy Acceleration Program (TAP), makes investments and forms partnerships with biotech companies that are developing innovative drugs to change the standard of care for patients with blood cancers. Since 2017, four LLS TAP-supported therapies have been approved by the FDA or included in the National Comprehensive Cancer Network guidelines, all leading to prolonged survival and improved response rates. TAP-supported therapies are being studied in over 20 active clinical trials, including several registration-enabling studies that may soon lead to additional FDA approvals.

With multiple funding programs designed to support all stages of research, LLS makes investments over the long term to shepherd early ideas all the way through to drug approvals. This sustained scientific investment has led to cures and to patients living longer, healthier lives. Survival rates for many blood cancer patients have doubled, tripled, or even quadrupled since the 1960s—but we at LLS aren’t done yet.

#LBS: As someone engaged in the research and development of blood cancer treatments, could you provide insights into the clinical progress and the future landscape of treatments options for this group of frequently fatal diseases? Please talk about the challenges and potential solutions?

Lore Gruenbaum: After a series of major breakthroughs in recent years, researchers and biotech companies, including those funded by LLS, are working hard to refine and expand their use so more patients can benefit. In 2023 alone, 16 drugs received FDA approvals in blood cancers with the promise of many more in the years to come.

To accelerate more breakthroughs and FDA approvals, LLS has committed more than $240 million to innovative research by nearly 300 top scientific investigators at prestigious cancer institutions around the world. In addition to these grants, TAP currently has $40 million invested in over 20 active partnerships with biotech companies, among then several in Europe, to support development of novel treatment platform technologies and first-in-class drugs for unmet medical needs, including for rare blood cancers.

We are making meaningful progress in all types of blood cancer and working through the challenges that arise from the recent and promising clinical progress with solutions dedicated to improving patients’ lives. Here are several examples of what’s coming next:

  • Very exciting are new data for a new class of drugs called menin inhibitors. Patients with AML with the so-called ‘KMT2A fusion’, who have among the worst AML outcomes, show improved overall survival and complete hematological responses after treatment with this type of drug candidate. The first ever menin inhibitor could get approved in the United States later in 2024.
  • After years of having nothing but supportive care to offer for patients with myelofibrosis, there are now several ongoing late-stage drug trials. These trials aim to not only improve symptoms but also change the course of the disease itself. Research has shown that combining the kinase inhibitor ruxolitinib with a novel BET inhibitor called pelabresib significantly improves all hallmarks of myelofibrosis. We will be watching for a possible drug approval in the U.S. for pelabresib in myelofibrosis over the next couple of years.
  • The LLS Beat AML Master Clinical Trial, the first collaborative precision medicine clinical trial in blood cancer, uses advanced technology for genomic analysis to match patients to precision treatment within seven days.  Beat AML has demonstrated improved survival and better quality of life for patients receiving precision medicine compared to patients receiving standard-of-care chemotherapy. Beat AML is currently investigating whether a shorter treatment course of an FDA-approved drug combination in older patients can maintain efficacy while lowering toxicity, which can often limit treatment in older patients.
  • Rare cancers like chronic myelomonocytic leukemia (CMML) are especially difficult to study. There is a major effort underway, led by LLS and supported by a generous $17 million donation from the Segal Family Foundation, to test new treatments and dig deeper into understanding how CMML forms and attacks the body. LLS is also supporting education programs for patients and caregivers, and other activities to increase biotech’s understanding and interest in this disease and accelerate patients’ enrollment in CMML clinical trials. Without committed disease support for a rare blood cancer like CMML, researchers have difficulty driving breakthroughs to impact patients.
  • Through the Dare to Dream project, LLS is powering innovative research, new safer treatments, support services and advocacy for children to improve access to care and outcomes. The global LLS PedAL Master Clinical Trial was launched in June 2022 to find safer and more effective treatments for children with relapsed or refractory acute leukemia by matching each child to treatment based on their unique tumor biology. This approach will fundamentally change how children with acute leukemia are treated and—it is hoped—lessen the long-term, and often life-long, effects of pediatric cancer treatment.  

#LBS: The London Biotechnology Show 2024 serves as a pivotal platform for stakeholders in the biotechnology domain to converge, exchange knowledge, and expand the influence of biotech solutions. What role do events of this nature play in shaping the significance of advancements in the healthcare sector?

Lore Gruenbaum: These types of events are critical for bringing together diverse groups of people and organizations that are driving innovation in the biotech and healthcare space. These environments foster collaboration among innovative thinkers, which helps all of us see new possibilities and think about new ways to approach research challenges.

It is also critical that these types of gathering include a range of professionals. Key innovations originate from all over the world. Forums such as the London Biotechnology Show can foster critical information sharing, collaboration and forward momentum in the UK and beyond and serve to connect European stakeholders to key partners worldwide to ultimately provide better solutions for patients.