Pediatric Cancer and Blood Disease Research

Testing approaches to make stem cell transplantation more accessible and safer

Stanford’s Alice Bertaina, MD, PhD, is a pioneer in alpha/beta T-cell depleted haploidentical stem cell transplantation, which greatly increases the number of patients who can receive transplants. This approach makes it possible to receive a transplant from a donor who is only a partial match while reducing the risk of graft-versus-host disease (GvHD) by removing some of the donor’s immune cells before transplant.

Dr. Bertaina is using this approach to transplant patients with blood cancers and combining it with kidney transplant to treat patients with rare diseases like Schimke immune-osseous dysplasia (SIOD) and focal segmental glomerulosclerosis (FSGS).

Harnessing the immune system to make stem cell transplants safer

A new clinical trial only available at Stanford is testing whether cell therapy can make these alpha/beta T-cell depleted haploidentical stem cell transplants even safer and more effective. In the clinical trial, patients receive one of these unique stem cell transplants and then are given a cell therapy called T-allo10. This cell therapy is made from the cells of the same person who donated stem cells for the transplant. The cell therapy contains T regulatory cells that tell the transplant recipient’s immune system to leave the transplanted stem cells alone. We are testing whether this approach can reduce GvHD even further and help the recipient’s immune system rebuild itself after transplant.

Attacking disease at the genetic level

We are proud to offer clinical trials testing the effectiveness of gene editing, or replacing a mutated or defective gene with a working copy, for several conditions, including Fanconi anemia, pyruvate kinase deficiency, and IPEX syndrome.

Bringing research from the lab to our patients

Stanford’s Center for Definitive and Curative Medicine (CDCM) unites top researchers and doctors working to bring potential cures from the lab to patients at Lucile Packard Children’s Hospital Stanford. Many of the innovative trials described above were developed with the support of the CDCM, giving your child access to the most advanced care, faster.