Stem cell transplantation clinical trials
Our doctors and researchers are constantly working to make stem cell transplantation safer and available to more people—especially patients suffering from conditions without a cure. After years of testing in the laboratory, we can bring potential treatments to our patients for testing in clinical trials.
Some of our unique clinical trials include:
- A new approach to stem cell transplantation that removes the immune system’s “fighter cells” from the donor’s cells before they are transplanted into the recipient. This approach aims to reduce the risk of graft-versus-host disease and enable patients without a fully matched donor to still have access to a potentially curative stem cell transplant. This approach is currently available for patients with:
- Blood cancers.
- Crohn’s disease.
- When combined with a kidney transplant from the same donor, patients with Schimke immuno-osseous dysplasia (SIOD), autoimmune focal segmental glomerulosclerosis (FGGS), systemic lupus erythematosus, and cystinosis.
- Another related trial also adds 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 recipient’s immune system to leave the transplanted stem cells alone. We are testing whether this approach can help the recipient’s immune system rebuild itself after transplant. This trial is only available at Stanford.
- New approaches to stem cell transplantation for patients with Fanconi anemia.
- For some patients with high-risk leukemias, cancer can return even after a stem cell transplant. A new clinical trial is testing the safety of QUAIL-100 (also known as LGNA-100), a novel investigational living bacterial therapeutic that has been significantly weakened so that it cannot grow or persist. These weakened bacteria are designed to be unable to cause an infection, though patients may still experience temporary side effects from the immune response. The temporary side effects are expected and are likely necessary to activate the immune system like a vaccine. This immune response is expected to attack cancer cells in new ways. QUAIL-100 is given after patients receive a half-matched, or haploidentical, stem cell transplant and the donor cells have successfully settled into the recipient’s body. Lab research shows that QUAIL-100 can help expand and activate the immune system, specifically different kinds of immune cells that are being studied for their ability to find and target cells that cause blood cancers like acute lymphocytic leukemia, acute myelogenous leukemia, and myelodysplastic syndromes. This is a Phase 1 study, meaning its main purpose is to test safety. It is sponsored by Laguna Biotherapeutics and led by researchers at Lucile Packard Children’s Hospital Stanford.

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