Pediatric Cancer and Blood Disease Research

Your child deserves the very best care, and for pediatric cancers and blood diseases, the very best care is often a clinical trial. Clinical trials are built on decades of promising research in the laboratory, and the goal of the trial is usually to make sure the treatment is safe and measure how well it works.

To find out more about our current trials, see our list of clinical trials or call (844) 760-9177.

Pioneering immunotherapy to find and kill cancer cells

Our physicians and researchers are working at the forefront of cancer immunotherapy, a promising field of cancer treatment. One type of immunotherapy, CAR T-cell therapy, uses a patient’s own genetically modified immune cells to track down and attack leukemia cells.

CAR T-cell therapy brings lasting remission after long fight

Jesus’ multi-year battle against leukemia included stem cell transplantation and immunotherapy.

Read Jesus' story

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.

Stem cell and kidney transplants help Shriya beat SIOD

A revolutionary approach combines a stem cell transplant from a parent followed by a kidney from the same parent, giving Shriya a life free of immunosuppressive drugs.

Read her story