Ushering In a New Era of Pediatric Blood Cancer Treatment and Care


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.

You’re incredibly passionate about the importance of data sharing to accelerate cures. Can you tell us more about the data commons project and what this will accomplish?

As we teach kids, sharing is caring. We want to ensure pediatric cancer researchers everywhere have a wealth of genomic and clinical information at their fingertips. Typically, the way data is collected and stored varies from institution to institution and country to country, making it challenging for researchers to compare and validate their findings. A team at the University of Chicago Pediatric Cancer Data Commons (PCDC), led by Samuel L. Volchenboum, MD, PhD, MS, is working to break down these data siloes. As part of LLS PedAL, a pioneering project is underway to gather pediatric acute leukemia data from multiple institutions, establish a common language to define and analyze the data and make that data available in a single data set to researchers worldwide. Researchers will then be able to identify patterns and benchmarks for treatment, assess findings, and determine new areas of study in real-time.

In addition to leading LLS PedAL, LLS is funding more pediatric blood cancer research. What’s the focus of this research, and what do you find most exciting?

LLS is funding more than 30 research grants focused on deepening our understanding of the unique genetic causes and molecular biomarkers of pediatric blood cancer and how best to target them. The goal is to accelerate more curative therapies with fewer side effects for children and young adults. Among the most promising treatment approaches is harnessing the power of the immune system to fight cancer. With LLS support, renowned researchers are developing next-generation chimeric antigen receptor (CAR) T-cell immunotherapies, which genetically reprogram a patient’s own T cells to find and kill cancer cells. Our more than 20-year investment in CAR T-cell immunotherapy led to the approval of a game-changing CAR-T for children and young adults with ALL in 2017. Researchers are now working to make this approach even more effective for more children and explore other emerging strategies like bi-specific T-cell engagers. We’re also investing in research to address the long-term effects of treatment, such as heart damage, infertility and cognitive impairments.

Watch this powerful story from the Breyfogle family who is dedicated to realizing cures and better care for childhood blood cancer.

What’s the latest on LLS’s work in the pediatric advocacy community?

The reality is the best treatments in the world can’t help patients who don’t have access to them. Our Office of Public Policy in D.C., alongside more than 50,000 online volunteers, is focused on driving policies to break down barriers to care and accelerate the development of new treatments. As co-chair of the Policy Committee of the Alliance for Childhood Cancer, LLS played an instrumental role in the enactment of The Childhood Cancer Survivorship, Treatment, Access and Research (STAR) Act, the most comprehensive childhood cancer legislation ever introduced. We’ve recently been mobilizing our advocates to ensure the act remains fully funded for a third straight year. What’s more, LLS helped advance the Research to Accelerate Cures and Equity (RACE) for Children Act to increase drug company investment in precision medicine trials for children with cancer. The law – which went into effect in August – requires that drugs being tested for adult cancers also be evaluated in children if the molecular targets are relevant to childhood cancer. The RACE Act will encourage more investment in LLS’s PedAL trial. And of course, LLS continues to work at both the federal and state level to strengthen Medicaid and the Children’s Health Insurance Program (CHIP), which together provide health coverage to more than half of our nation’s kids. I encourage you to sign up to be an advocate and join our lifesaving work today.

LLS is expanding its free education and support for children, caregivers, families, and healthcare professionals. Can you tell us about some of the new resources available?

LLS is dedicated to helping patients and families navigate treatments and access quality care. Our free, one-on-one support is making all the difference right now. I encourage you to reach out to LLS Information Specialists at (800) 955-4572 or by live chat/email. We also offer in-depth clinical trial navigation and support through our Clinical Trial Support Center as well as one-on-one nutrition consultations with a registered dietitian. Several of our master’s level oncology professionals and registered nurses on staff have expertise in pediatric blood cancers. And all of our trusted education and support can be accessed from the comfort of your home. Among the many new resources are our Caring for Kids and Adolescents caregiver workbook, a booklet to guide your child's return to school after a cancer diagnosis, and a digital coloring app for children. We also offer online chats, financial support, a peer-to-peer support program, webcasts, and so much more! You can find more resources on this page, and we encourage you to check often as we update throughout the year. At the same time, we’re helping healthcare and school professionals stay informed on childhood, adolescent, and young adult cancers. For example, our Staying Connected online continuing education (CE) program educates school and college personnel on improving patients’ learning experience during and after treatment. The program can be completed at your own convenience, and parents are welcome, too. Along with this, we offer a podcast for healthcare professionals and robust CE/CME programs.

Find more free information, education, and support from LLS.

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