New Research, Treatment Helps Kids Fight Cancer

In 2017, University of Wisconsin Department of Pediatrics faculty made great strides to advance research and improve treatments for children with cancer.

Inga Hofmann, MD, received a one-year, $200,000 grant from Evans MDS to analyze mechanisms of expression in GATA2, a gene whose mutations can cause myelodysplastic syndrome and acute myeloid leukemia. Her team aims to identify prognostic markers that will help predict the risk of disease progression and inform the timing of stem-cell transplantation – work that dovetails with her role as medical director for the new Program for Advanced Cellular Therapy (PACT), a collaboration of the University of Wisconsin School of Medicine and Public Health and UW Carbone Cancer Center.

Dr. Hofmann also received a $50,000 infrastructure grant from the St. Baldrick’s Foundation to build staff capacity for clinical research on advanced cell therapy for pediatric cancer.

Kenneth DeSantes, MD, launched a new clinical trial to evaluate MGA271, a genetically modified antibody, for the treatment of certain types of relapsed childhood cancers that express a molecule known as B7-H3. It’s the first trial from researchers in the Pediatric Cancer Dream Team, an eight-institution collaboration first established in 2013 and led at UW by Paul Sondel, MD, PhD. Support for the Dream Team, which comes from the St. Baldrick’s Foundation, Stand Up to Cancer, and the American Association for Cancer Research, was also renewed in 2017 for four more years.

With a three-year, $75,000 grant from the Cannonball Foundation, Mario Otto, MD, PhD, continued a single-institution, Phase I clinical trial that evaluates a novel graft engineering process for hematopoietic stem-cell transplantation in children with solid-tumor or hematologic cancers that do not respond to standard treatment or that recur. The trial will also evaluate the safety of administering zoledronate, a medication that has been shown to activate the immune cells infused in the graft, after transplantation.

In addition, Dr. Otto received approval from the Food and Drug Administration (FDA) to conduct a Phase I clinical trial to evaluate the use to CLR131, a novel radiopharmaceutical, to treat children with relapsed solid tumors and central-nervous system cancers.

Finally, American Family Children’s Hospital was one of a handful of centers nationwide to receive FDA approval to provide chimeric antigen receptor (CAR)-T cell therapy, Kymriah (tisagenlecleucel), for the treatment of acute lymphoblastic leukemia (ALL) that has resisted other treatment or has relapsed a second time. Christian Capitini, MD, who led clinical trials for the therapy, treated the first patient in Wisconsin with Kymriah in early 2018.