Fanconi anemia patients have a very high predisposition to head, neck, and anogenital squamous cell carcinoma (SCC). To understand how these cancers develop, Dr. Smogorzewska and team are cataloging genetic changes that are present in the cancers. They are using the latest DNA sequencing technologies...
Supported Research
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There is a great deal of excitement about the potential of CRISPR genome editing to treat disorders such as Fanconi anemia. But the mutations that cause FA also reduce the effectiveness of genome editing. Dr. Corn and colleagues will search for potential ways to re-activate...
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Grant description: Beyond surgical resection, there is no effective therapy nowadays to treat FA patients that develop solid tumors, so a drug able to kill tumor cells but nontoxic for FA patients is of critical relevance. Gefitinib and afatinib candidates are approved drugs and are...
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Oral cancer in Fanconi patients is a severe disease requiring innovative treatment and prevention measures. The potential use of FDA-approved diabetes drugs is a practical strategy for Fanconi patients for oral cancer prevention or treatment studies, once these preliminary studies are conducted. There is an...
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Grant description: Persons with FA, as well as their family members, voice concern about being underweight and unable to build muscle mass. These concerns have an obvious physical impact, not only on the person's appearance to others and themselves and the negative impact on immune...
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FA confers a high risk of developing leukemia at a young age, and patients with FA who develop leukemia are difficult to treat with have poor long-term outcomes. Leukemia develops as a consequence of the acquisition of genetic mutations within blood stem cells in the...
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Head and neck cancer is a major issue for the FA population. There is no suitable preclinical model to study potential strategies for preventing or delaying squamous cell carcinoma in FA. Using a cancer-prone mouse model developed by a lab in the United Kingdom, researchers...
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Grant description: Treatment of blood complications in FA kids using bone marrow transplantation has seen great improvement over the past decade. However, after a successful bone marrow transplantation, those kids affected with FA must now face nonblood malignancies in subsequence adolescent life which defy conventional...
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Grant description Bone marrow transplantation has been used for 60+ years to treat >1,000,000 patients suffering from many types of blood or immune diseases, and this therapy is the best current treatment for FA patients experiencing blood problems. This procedure relies on replacing sick blood-forming...
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Fanconi Anemia (FA) is characterized by fragile bone marrow and the inability to repair DNA damage which accumulates in repopulating stem cells, leading to marrow failure. Current treatments often involve bone marrow transplantation however the majority of patients will not have an appropriately matched sibling...
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“If I had waited another six months, my story wouldn’t be the same.” Katherine was born into a world shaped by loss. Her older sister, Gracie, was diagnosed with Fanconi anemia (FA) shortly after the family moved to the United...
Institution: Laboratorio de Citogenetica, Instituto Nacional de Pediatría, Mexico City, México Area of expertise: Medical genetics, dysmorphology. My work: I am a medical geneticist from Mexico City, introduced to the field of Fanconi anemia (FA) by my mentor, Dr. Sara Frias, whom...
At the Fanconi Cancer Foundation (FCF), we understand the critical importance of addressing mental health alongside physical health for individuals with Fanconi anemia (FA) and their family caregivers. Recent research on the mental health challenges faced by adults with FA...