Stem Cell Transplant | CHOP Research Institute

Can a Small Molecule Drug Improve Cell Fitness for Transplantation?

Published on

Oct 20, 2023

Snapshot Science: Can a Drug Improve Cells for Transplantation?

Researchers found exposing cells to a small molecule drug improves cell fitness during hematopoietic stem cell transplants, which could improve ex vivo gene therapy.

Stem Cell Transplant for Sickle Cell Disease or Thalassemia Major

The study will enroll male and female patients between 2 years to 25 years of age with sickle cell disease or beta thalassemia major, who do not have a matched related donor, and their doctor feels their condition should be treated with hematopoietic stem cell transplantation (HSCT).

MICRO Study

The purpose of this study is to collect and store data samples, such as stool samples, mouth swabs and saliva specimens for research about the microbiome.

17-SIBS Study

The purpose of this research study is to understand the impact of transplant on the quality of life of the entire family including sibling donors, other siblings and parents.

Comprehensive Center for the Cure of Sickle Cell Disease and Other Red Blood Cell Disorders

Pioneering new approaches to diagnosis, care, and treatment for patients with red blood cell disorders with novel gene therapies and stem-cell-based treatments that aim to reduce symptoms and prolong life.

Jason L. Freedman, MD, MSCE

Dr. Freedman has a particular interest in epidemiologic and translational research within pediatric supportive oncology. He aims to advance the understanding and treatment of symptoms, infections, and organ toxicities in pediatric cancer and hematopoietic stem cell transplant patients.

E-mail:

freedmanj [at] chop.edu