Precision Medicine for Wilms Tumor Patients

Precision medicine for Wilms tumor patients Each year in the U.S., about 650 children are diagnosed with Wilms tumor, the most common pediatric kidney cancer. The vast majority of patients respond well to the current standard of care involving a combination of surgery, chemotherapy and radiation. However, approximately 20% of patients with “favorable histology” Wilms tumor experience recurrence. Previously, researchers discovered that loss of heterozygosity (LOH) on chromosomes 1 and 16 is associated with a significantly increased risk of relapse. A Children’s Oncology Group (COG) research team led by Jeffrey Dome, M.D., Ph.D., sought to determine whether an augmented chemotherapy regimen can overcome the negative effect of LOH.

Extracting meaningful data from CRISPR screens Wei Li, Ph.D., a principal investigator in the Center for Genetic Medicine Research, received $2.23 million from the NIH to develop statistical and computational methods that sidestep existing hurdles that currently complicate genome-wide CRISPR/Cas9 screening. Fighting lymphoma with targeted T-cells To help improve outcomes to allogenic hematopoietic stem cell transplants for patients with Epstein Barr virus (EBV)-associated lymphomas, researchers are testing transplants of immune cells specifically trained to fight cells infected with EBV. “Liquid biopsies” to track diffuse midline gliomas A multi-institutional team led by researchers at Children’s National showed that quantifying the amount of circulating tumor DNA possessing key mutations characteristic of diffuse midline gliomas could reliably predict the tumors’ response to radiotherapy. TAA-Ts as therapy for tumors Recently, a team led by Holly Meany, M.D., uncovered tumor-associated antigen cytotoxic T cells (TAA-Ts) that represent a new and potentially effective nontoxic therapeutic approach for patients with relapsed or refractory solid tumors. Fighting lethal cancer with a one-two punch New research could lead to a promising way to treat neuroblastoma by enabling the immune system to recognize these tumors in order to spark an anti-tumor immune response. Anthony D. Sandler, M.D., and colleagues reported on these results using an experimental model of the disease. Fighting neuroblastoma with TGFB receptor Catherine Bollard, M.B.Ch.B., M.D., and her team showed the potential efficacy of a novel cell therapy for treatment of pediatric patients with relapsed/refractory neuroblastoma. Their new approach allows them to engineer NK cells, expand and reinfuse them within a patient to enable them to fight neuroblastoma.

Upcoming Events American Academy of Pediatrics National Conference and Exhibition October 25-29, 2019 New Orleans, Louisiana Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR February 19-23, 2020 Orlando, Florida Annual Meeting of the European Society for Blood and Marrow Transplantation March 22-25, 2020 Madrid, Spain Pediatric Academic Societies Meeting April 29-May 1, 2020 Philadelphia, Pennsylvania American Society of Pediatric Hematology/Oncology May 6-9, 2020 Fort Worth, Texas Copyright © 2019, All rights reserved. Our mailing address is: Children's National 111 Michigan Ave NW Washington, District of Columbia 20010