"This LLS award will improve our ability to protect the hearts of pediatric AML patients from the harmful effects of therapy, which threatens long-term cardiovascular health. This study will augment our ability to identify children at highest risk for heart failure to allow timely initiation of medications to preserve heart function." - Kasey Leger, M.D., Seattle Children’s Hospital
The Leukemia & Lymphoma Society Doubles its Funding of Pediatric Cancer Research Grants with $25 Million Commitment, Part of Bold Effort to Change the Course of Treatment for Children.
Blood Cancer Awareness Month in September provides an opportunity to remind the public about the urgent need to support research to find cures, and The Leukemia & Lymphoma Society (LLS) is marking the occasion with a reinvigorated focus on helping children with cancer live better, longer lives.
LLS announced that it has more than doubled its funding of research focused specifically on childhood blood cancers, adding 20 new research grants valued at more than $13.8 million to its research portfolio in 2019. With these new grants, LLS now has committed more than $25 million over a five-year period to change fundamentally how children with blood cancers are treated. Further, LLS also supports grants that are relevant to adolescent and young adult cancer patients.
The new pediatric grants are part of The LLS Children's Initiative, a $50 million comprehensive attack on children's cancer from every angle, from new research investment to advance novel therapies and bolster clinical trials, to enhanced services and support for children and their families, to renewed policy efforts. LLS is also planning an unprecedented global precision medicine clinical trial for children with acute leukemia as part of the initiative.
The Urgent Need
The two most common leukemias in children are acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML), where the five-year survival rates are approximately 90 percent and 60 percent, respectively. While significant progress has evolved in the treatment of ALL over the 70 years since LLS was founded, advances in treating AML are more recent, after many decades with little change. This means that approximately 700 children will die of leukemias in the United States every year. Many of the children who do survive these blood cancers experience long-term complications, typically from treatments they receive. Further, while scores of targeted therapies have been approved for adult cancer patients, only four cancer treatments have been approved for first use in children since the 1980s.
As we now understand more fully the molecular basis of these leukemias, and have new technologies and an arsenal of molecularly targeted drugs and novel immunotherapies for blood cancers, the stage is set to make significant progress for children with these diseases.
Since its founding 70 years ago, LLS, the world's largest nonprofit dedicated to blood cancer research and patient services, has invested nearly $1.3 billion in cancer research, resulting in game-changing advances. The new pediatric cancer grants announced today are part of a larger $44 million comprehensive package of new grants targeting all the blood cancers - leukemia, lymphoma, myeloma and other rare blood cancers – that afflict both children and adults. In all, LLS now is funding more than 292 research projects around the world, a $179 million investment propelling innovations in immunotherapy and precision medicine.
To learn more about all of LLS's new research grants click here.
"September is Blood Cancer Awareness Month, and it is also Childhood Cancer Awareness Month, making this an important time to talk about the need to take a bold new approach to helping children with cancer," said Gwen Nichols, M.D., LLS chief medical officer. "Children are not little adults. They need better, less toxic treatments designed just for them. Our goal is a wholesale shift in the standard of care for pediatric patients, moving from toxic chemotherapies that leave survivors with lifelong health challenges, to effective, safe treatments that target cancer precisely, without harming the rest of the child's body."
Among the new pediatric research grants are:
A project to improve young patients' quality of life
Toxic chemotherapies frequently cause long-term side effects. Kasey Leger, M.D., Seattle Children's Hospital, is trying to help prevent children from suffering heart damage due to treatment with chemotherapy.
New immunotherapeutic approaches for children with blood cancers
Several of the researchers, including Terry Fry, M.D., University of Colorado, Soheil Meshinchi, M.D., Fred Hutchinson Cancer Research Center, and Ryotaro Nakamura, M.D., City of Hope, are all developing next-generation chimeric antigen receptor (CAR) T-cell immunotherapy, a revolutionary approach that reprograms a patient's T cells to find and kill cancer cells. CAR-T is approved to treat children with acute lymphoblastic leukemia but these researchers are testing new approaches to make it even more effective for more children with acute leukemias. Meshinchi is also developing other approaches to harness the immune system, including a treatment called bi-specific T-cell engagers and another employing antibodies.
Jatinder Lamba, M.D., University of Florida, is investigating personalized antibody treatments targeting CD33, a protein commonly expressed on the surface of leukemia cells. He is focused on identifying biomarkers that will predict which children with acute myeloid leukemia will respond to agents that target CD33.
Addressing high-risk forms of acute leukemia with very poor prognoses
Robert Albero Gallego, M.D., of Columbia University, and Iannis Aifantis, M.D., of New York University, both received funding to tackle T-cell acute lymphoblastic leukemia, which is a devastating diagnosis for children. Another diagnosis with very poor outcomes for children is called CRLF2-rearranged ALL.
Charles Mullighan, MS.c, M.D., of St Jude Children's' Research Hospital, received funding to develop two novel approaches to treat this disease. One method will use a targeted therapy to degrade and destroy abnormally active proteins in cancer cells, and the other will use the gene editing process called CRISPR to identify which genes are essential for the growth of CRLF2-rearranged leukemic cells.
Better monitoring of Hodgkin lymphoma treatment
Hodgkin lymphoma is a blood cancer that makes up about 7 percent of the cancers that afflict children, adolescents and young adults under the age of 20. While survival rates for children with Hodgkin lymphoma are quite high, the treatments are harsh and often cause long-term side effects.
Davide Rossi, M.D., Ph.D., Foundation for the Institute of Oncology Research in Switzerland, is looking at using liquid biopsy, a highly sensitive test that looks for pieces of DNA from tumor cells circulating in the blood, to improve monitoring of patients early in their treatment. This minimally invasive test that can be repeated with a simple blood draw (versus a PET or CAT scan) can help determine early on during therapy if patients who respond well to treatment can receive less chemotherapy, while those who are more resistant would benefit from higher doses.
"This critical funding from LLS will help us advance targeted and immunotherapeutic treatments for babies under age five with acute leukemia subtypes who do not respond to standard chemo. We hope that this work will lead to therapies that can cure this aggressive type of leukemia without harming the infants’ growing bodies." - Soheil Meshinchi, M.D., Fred Hutchinson Cancer Research Center
Founded in 1949 by a grieving couple who lost their teen-aged son to leukemia, LLS has dedicated its 70th year to the patients, caregivers, survivors, volunteers, healthcare professionals and researchers who share our relentless drive to find cancer cures. Whether they have benefited from our lifesaving work or contributed to our successes, they have shared their stories of being part of #GenerationLLS – by adding their story to the Generation LLS Family Tree.
"We were founded by a family for families, and we've remained true to our founders' vision, the belief that cancers are curable," said Louis J. DeGennaro, Ph.D., president and CEO of LLS. "We've helped usher in a new era of cancer therapies that centers on giving the right treatment to the right patient at the right time, and we will be relentless until we achieve our goal of eradicating these diseases. The LLS Children's Initiative continues our founders' legacy of helping children and families throughout their entire cancer experience."
LLS PedAL: Precision Medicine for Children with Leukemia
Through the LLS PedAL master clinical trial, The Leukemia & Lymphoma Society (LLS) is setting out to change fundamentally how children with pediatric acute leukemia, including acute myeloid leukemia and other high-risk leukemias, are treated.
LLS is working with a team of pediatric oncologists to lay the groundwork for a global precision medicine master clinical trial to match children with relapsed acute leukemia to a targeted therapy based on the specific abnormalities driving their cancer. The goal of the trial is to test multiple targeted therapies simultaneously at up to 200 clinical sites worldwide. LLS anticipates treating the first patient in the summer of 2020.
In addition to the clinical trial, LLS PedAL seeks to break down silos that prevent researchers from sharing data about pediatric cancer patients. The PedAL team is working to consolidate pediatric cancer data from multiple institutions into a single data set, establish a common language to define and analyze the data and make that data available to researchers worldwide.
Gwen Nichols, MD, Chief Medical Officer of The Leukemia & Lymphoma Society, heads a team of preeminent leaders in pediatric acute leukemia to conceive, develop and implement LLS PedAL:
E. Anders Kolb, MD, Nemours Center for Cancer and Blood Disorders, Co-Chair, PedAL Initiative;
Soheil Meshinchi, MD, PhD, Fred Hutchinson Cancer Research Center, Target and Biomarker Lead;
Todd Cooper, DO, Seattle Children's Hospital, Clinical Trial Lead;
Todd Alonzo, PhD, Children's Oncology Group, Group Statistician;
Kimberly Stegmaier, MD, Dana-Farber Cancer Institute, Preclinical Discovery Lead;
Samuel L. Volchenboum, MD, PhD, University of Chicago, Bioinformatics Lead;
Laura Di Laurenzio, PhD, The Leukemia & Lymphoma Society, Project Leader;
Julie Guillot, AML Parent and Partnership Outreach Chair.
About The Leukemia & Lymphoma Society
The Leukemia & Lymphoma Society® (LLS) is the world's largest voluntary health agency dedicated to blood cancer. The LLS mission: Cure leukemia, lymphoma, Hodgkin's disease and myeloma, and improve the quality of life of patients and their families. LLS funds lifesaving blood cancer research around the world, provides free information and support services, and is the voice for all blood cancer patients seeking access to quality, affordable, coordinated care. Founded in 1949 and headquartered in Rye Brook, NY, LLS has chapters throughout the United States and Canada.
To learn more and support The Leukemia & Lymphoma Society, visit www.LLS.org