My lab’s vision for the next five years is to apply what we have learned to develop molecules that can alter FA protein behavior. These could be used to treat cancer or alter gene editing outcomes.
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Blanche P. Alter
Senior Clinician | MD, MPH | Bethesda, MD
Dr. Alter developed an interdisciplinary clinical research program, conducting systematic investigations of cancer in the inherited bone marrow failure syndromes (http://marrowfailure.cancer.gov/). The prototype disorder for this program is Fanconi anemia, in which there is a large excess of unusual cancers that occur at atypically early ages, including myelodysplastic syndrome, acute myeloid leukemia, and cancers of the oral cavity and oropharynx, esophagus, cervix and vulva, and liver.
This project involves active, protocol-driven clinical research. Investigations include surveys to determine the incidence and characteristics of cancer in each of the syndromes; case-control studies to identify additional risk factors and perhaps new cancer pathways; genotype/phenotype correlations with reference to cancer susceptibility; heterozygote surveys with regard to the possible relation between the carrier state and cancer; and biologic studies of the tumors to determine their resemblance to their counterparts in the general population. The patients who have these syndromes comprise a group at high risk of cancer which may be suitable for studies of cancer screening and prevention modalities.
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In a world where every day presents new challenges, my 11-year-old son, Omar, from Oman, stands out for his resilience and eagerness to raise awareness about Fanconi anemia. Diagnosed at just five years old, we truly believe his journey is a testament to hope and the strength of the human spirit.
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That's why we're thrilled to announce the launch of the Fanconi Cancer Foundation-AACR NextGen Grant for Transformative Cancer Research, a flagship funding opportunity in partnership with the American Association for Cancer Research (AACR).