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Research & Innovation

Our physicians and researchers work closely together to bring groundbreaking new treatments to patients. We have several new therapies in development, including new treatments for Fanconi anemia.

Global Gene Therapy Clinical Trial for Fanconi Anemia Type A (FA-A) now enrolling

What is Fanconi anemia (FA)?

FA is a rare genetic disorder affecting DNA repair. Approximately two-thirds of FA cases are caused by genetic defects in the FANCA gene, which causes the FA subtype known as FA Complementation Group A (FA-A). FA patients may develop bone marrow failure (very low blood counts) and cancers of the blood or other cancers.

How does this gene therapy work?

Blood stem cells collected from the FA-A patient are genetically modified to introduce an intact copy of the FANCA gene using a virus that has been changed in the laboratory so that it cannot grow or spread to cause an infection. The genetically modified cells are then returned back into the patient.

Who is eligible to participate in the gene therapy clinical trial?

FA-A patients ages 1 through 12, who have not developed severe bone marrow failure, do not have an HLA-matched sibling donor for bone marrow transplant and are not on other experimental therapies.

What does participation in the gene therapy clinical trial involve?

Before you begin the study, you will have to have several tests to determine whether you are eligible to join the trial. These tests can take up to one month to complete.

After the doctors conducting the study confirm that you are eligible for the trial, participating in the study will involve:

  • Stem cell collection: Stem cells have the potential to make the different blood cells in your body. You will receive medications that make stem cells in your bone marrow enter your blood where they will be collected from your vein (most likely using a catheter) during a procedure called apheresis.
  • Infusion of genetically modified cells: Your stem cells will be genetically changed in a laboratory to introduce the intact copy of the FANCA You will then receive an infusion of the gene-modified cells through the catheter. There is no conditioning chemotherapy before the infusion.
  • Follow up after gene therapy: Patients will need to return for follow-up visits, including blood and bone marrow tests, over the next three years. In addition, patients will have long-term follow-up with their home physician approximately 1-2 times per year for another 12 years.

To learn more about this trial, please:

Lucile Packard Children’s Hospital Stanford is the only hospital in the United States to offer this clinical trial.

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