Patients and families dealing with FA benefit greatly by seeking out an FA center that offers not only multi-specialty care and transplants but also access to emerging treatments such as gene therapy. The Fanconi Anemia Center provides exactly this type of research-driven approach. Our goal is to create safer and more effective therapies for FA.
Several clinical studies open to FA patients are described here with links to more detailed information about eligibility.
Gene Therapy for FA
As an alternative to transplantation, our researchers have developed a new method to remove some of the FA patient’s own marrow or stem cells, transfer a normal FA gene into these cells, and then give these “gene-corrected” cells back to the patient. This new form of FA gene correction is called Lentivirus Vector-Based Gene Therapy. In late 2011, we became the first FA treatment center in the world to offer lentiviral vector gene therapy to FA patients. Our study is limited to patients in FA complementation group A and without an HLA-matched sibling donor. The main purpose of this ground-breaking study is to measure safety. That’s why only adults (>18 years of age) are being enrolled initially. Expansion to include children may occur later. Families, patients, or physicians should contact the FA Center at Seattle Children’s for study details and updates. Click here for more information.
Improved Transplant Methods for FA Patients
Easier Low-Dose Chemo Preparation for Transplantation in FA
Recognizing the hypersensitivity of all FA patients to pre-transplant conditioning, we helped develop the low-dose cyclophosphamide regimen that is now a standard of care.
Alternative Donors for FA Patients with No Matched Sibling
We recently created special transplant protocols for the many FA children who lack matched donors. For example, one current study allows haploidentical (half-matched) parents and siblings of FA patients to donate marrow. Click here for more information: FHCRC-2064
Preparing for FA Transplant Without Radiation
We are partnering with other FA Centers to eliminate transplant-related radiation; this study, open to FA patients without related donors, aims to limit the risks that radiation therapy may have for FA patients. More information here FHCRC-2598.
Other Studies for FA Patients
Many of the other clinical studies being conducted by researchers at the Seattle Cancer Care Alliance—including Seattle Children’s, the University of Washington Medical Center, and the Fred Hutchinson Cancer Research Center—may also be appropriate for FA patients. General descriptions of these lines of research are provided here.
Families, patients, or physicians wanting information about specific clinical studies should contact the Fanconi Anemia Center at (206) 288-7222.
- Several trials are underway to boost the number of active stem cells harvested from umbilical cord blood, a method that could eventually improve options for FA patients without matched donors.
- Researchers at the Hutchinson Center are conducting several studies to prevent or treat one of the major dangerous complications seen in transplanted FA patients: graft versus host disease (a condition where the transplanted immune cells attack the patient’s body).
- Hematologists and oncologists at SCCA have developed many new treatments for the leukemias and the solid head and neck tumors that tend to occur in FA patients (with or without transplant).