As we round out our first year of marriage and our first year of battling cancer, I recently asked my husband Brad how he would describe this past year. “Disrupted.” Before cancer, we had plans: big, extravagant, travel-the-world kind of plans. After two years of a long-distance relationship, we were finally going to live in the same city. We were going to get settled in our brand-new home. We were going to make new Florida friends. We were excited about the opportunities we had in our careers. We were going to capitalize on the “newlywed phase” to the fullest extent! Then, we learned that cancer doesn’t care if you’re newly married with plans in place; it can rock your world in an instant.
Five days – that’s how long we had been married when we heard the most unexpected, gut-wrenching words: “it’s cancer.” Just five days before, Brad was in a black tux. I was in the most beautiful wedding gown. We were surrounded by our biggest support system and celebrating the vows we had just made to each other – to love each other for better or worse, for richer or poorer, in sickness and in health.
Our “Meet the Researcher” series on The LLS Blog shares what our outstanding LLS-funded researchers are working on, the incredible impact they’re making in the fight against blood cancer, and what inspires their efforts to find better treatments and cures. Dr. Maria “Ken” Figueroa, Associate Professor and Co-Leader of the Cancer Epigenetics Research Program at Sylvester Comprehensive Cancer Center, part of the University of Miami Health System, is focused on an exciting area of research called epigenetics. Epigenetics refers to chemical modifications that regulate (switch on and off) gene activity. With support from LLS, Dr. Figueroa is studying how the epigenetic changes that occur as people age may contribute to acute myeloid leukemia (AML) and other blood cancers. Advancing age is the most important risk factor for cancer overall, according to the National Cancer Institute. Dr. Figueroa’s cutting-edge work offers new clues toward understanding the factors associated with cellular aging and cancer risk.
Why were you drawn to blood cancer research?
As an attending hematologist back in Argentina, it quickly became apparent that continuing to treat patients with broad chemotherapies that were highly toxic and resulted in frequent relapse was ineffective and unsustainable beyond a first relapse. The initial discoveries by Dr. Brian Druker on the efficacy of targeted therapies in chronic myeloid leukemia (CML) in the early 2000s led me to believe that similar approaches could be implemented for other blood cancers.[1]It was clear that a better understanding of the mechanisms driving the origin of cancers would be required to improve treatment.
What is the focus of your research?
My team is studying how blood forms in the bone marrow and how the normal instructions that govern this process become corrupted – either during normal aging or during malignant transformation. Our hope is that by understanding the underlying changes associated with abnormal blood production, we will be able to identify ways to prevent or treat AML and other blood cancers.
How can studying the epigenetic changes that occur as we age help inform our understanding of blood cancer risk and how to treat these diseases?
Our studies into the epigenetic changes of aging hematopoietic (blood-forming) cells have helped identify many cancer-predisposing changes that occur with normal aging. These changes don't result in cancer per se but can put us in a more susceptible state, in which any additional insult may push the cells in the wrong direction. As we learn more about these changes, we will be better positioned to identify effective preventive interventions or diagnostic strategies that would allow for early therapies to be given. Finally, these unique contributing mechanisms could be harnessed themselves for the development of novel treatments. All of these approaches are currently being explored in my lab.
How will blood cancer patients benefit from your work?
Our primary focus is to identify ways to improve the diagnosis and treatment of blood cancers. We seek to identify laboratory markers that can be used both for early diagnosis and prognostication of disease. This way, we will be able to better classify patients based on their risk of rapid disease progression and their likelihood to respond to therapies. In addition, by better understanding the mechanisms that drive malignant transformation, we aim to identify how we can precisely target the malignant cells with novel therapies, while at the same time preserving normal cells. This type of highly selective therapy is predicted to be more effective and better tolerated than currently available chemotherapies.
How has LLS helped advance your research career?
LLS has been instrumental in supporting my career. From an early Special Fellow Award that was the first grant to support my transition to an independent research position to continued support for several of my projects, funding from LLS has been essential in my ability to pursue original research, especially in areas that the National Institutes of Health (NIH) would not support.
What makes you want to get out of bed in the morning?
I live in a state of constant excitement about the next discovery. I am fortunate to have recruited an incredibly talented group of investigators to my lab, and I deeply enjoy my discussions with them. Every day, they surprise me with new and exciting findings that bring us closer to our goals. As a mentor of basic scientists, I always try to have my trainees spend some time in the clinic with our physicians. This gives my trainees the opportunity to appreciate the experience of blood cancer patients and how our research is working toward improving that experience. I feel that it is important for them to also feel this sense of excitement for new discoveries.
[1] LLS has provided more than $27.4 million in funding since the 1990s to bolster the visionary research of Brian Druker, MD, Director of The Knight Cancer Institute at Oregon Health & Science University. With LLS support, Dr. Druker pioneered targeted therapy, a cornerstone of precision medicine. His work led to the development of imatinib, a breakthrough in CML treatment that was first approved in 2001 and has significantly improved outcomes for patients across the globe. Today, Dr. Druker is driving innovation in precision medicine through our Beat AML Master Clinical Trial.
Learn more about Dr. Figueroa and some of her recent breakthrough work by reading our Research Spotlight story here or following her on Twitter @KenFigueroaLab.
We caught up with Gwen Nichols, MD, Chief Medical Officer at The Leukemia & Lymphoma Society (LLS), to share the progress we’re making to bring cures and better care to children with blood cancer through The LLS Children’s Initiative.
Tell us about The LLS Children’s Initiative and why it’s so important to you and LLS.
When I joined LLS in the spring of 2017, I knew my first order of business would be advancing our work for pediatric blood cancers. As a physician and researcher, I had seen first-hand the limitations of current therapies and the devastating long-term effects of treatment experienced by childhood cancer survivors. I could not stand by any longer. Together with our community of volunteers, patients, families, researchers, and partners, we realized it would take unprecedented collaboration – and our signature grit – to transform pediatric blood cancer treatment and care. Last year, we launched The LLS Children’s Initiative, a $100 million, multi-year endeavor to take on childhood blood cancer from every direction, including research, education and support, and advocacy. Our goal is to help children not only survive their cancer but thrive in their lives after treatment. With the pandemic impacting patients’ access to treatment, it’s more important than ever to put patients and families at the center of their care. That’s exactly what LLS is doing.
As a longtime champion of precision medicine and breakthrough research, how is LLS working to bring the promise of precision medicine to pediatric blood cancer patients?
Children have been treated with a one-size-fits-all approach for too long, despite cancer behaving differently in children than in adults. For many childhood cancers, chemotherapies developed decades ago remain the standard of care and simply don’t work well enough. In this era of precision medicine – giving the right treatment to the right patient at the right time – it’s critical for children to receive new therapies designed just for them. At the heart of The LLS Children’s Initiative is LLS PedAL, the first global precision medicine clinical trial for pediatric acute leukemia. LLS PedAL will test multiple novel therapies simultaneously at more than 200 clinical sites worldwide to bring safer, more effective treatments to children and young adults with relapsed acute leukemia. Through LLS PedAL, we will identify and validate underlying molecular drivers of disease and match patients with the most promising treatments based on their unique tumor biology. Ultimately, every child who enters the trial will receive screening to determine their specific biological markers.
When will the LLS PedAL trial open, and what progress has been made so far?
We anticipate opening LLS PedAL by summer 2021. Until then, we’re pressing forward. Thankfully, we have brilliant physicians, scientists, volunteers, patient organizations, partners, and pharma/biotech companies in our corner. Earlier this summer, LLS and The Children’s Oncology Group, a national clinical trials network supported by the National Cancer Institute (NCI) with more than 9,000 clinical experts in childhood cancer at top children’s hospitals and institutions, joined forces. Approximately 200 COG sites across the U.S., Canada, Australia, and New Zealand will now be eligible to participate in LLS PedAL. We’re also collaborating with researchers and regulators in the U.K. and EU. By tapping into established infrastructures, we’ll be able to bring better treatments to children with relapsed acute leukemia rather than requiring families to travel to hospitals far from home. What’s more, we’ve partnered with PRA Health Sciences to help us manage the trial. We’re also in discussions with several pharma companies. And we’re working closely with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) on the trial protocol. All of these milestones are powered by a shared drive to cure pediatric blood cancers.