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 today 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 either of 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 more fully 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.
“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.