Research Report Winter 2022: Four New Grants Funded

In early 2022, four new grants were awarded to advance research in cancer preclinical models, Fanconi Anemia Neurological Syndrome (FANS), and mental health.

If you’d like to submit a research grant proposal, please contact Isis Sroka, Scientific Director.

If you’d like to support FA research by making a gift or hosting a fundraiser, please visit here.

Stay tuned for further updates on these projects and other research initiatives.

See the full list of FARF-funded research grants here.

New Grant: Mental Health

Psychosocial Experiences of Adults with Fanconi Anemia: A Participatory Mixed-Methods Research Study

Kathleen Bogart and Megan Voss
Oregon State University and University of Minnesota

The issue: research has shown that adults living with rare diseases face a variety of psychosocial challenges, including anxiety, depression, stigma, isolation, disease self-management, and mortality concerns. However, little is known about the mental health issues specific to adults living with FA.

The project:The aim of this grant is to partner with the FA community to design a quality-of-life study that considers the physical, mental, emotional, and spiritual needs of adults living with FA. The research team will administer a quantitative survey to 100 adults living with FA and conduct semi-structured follow-up interviews with 15 adults living with FA. Results from this grant will help FARF and clinicians around the world develop strategies to improve the mental health of those living with FA.

Amount funded: $98,495

New Grants: Cancer Preclinical Models

Development and Characterization of FA-HNSCC PDX Models

Jenny Grandis and Daniel Johnson
University of California San Francisco

The issue:young adults with FA who are diagnosed with squamous cell carcinoma face limitations with treatment options due to DNA repair defects associated with the disease. Preclinical models that can be used to identify non-DNA damaging FA cancer therapies are desperately needed.

The project: the research team will generate preclinical patient-derived xenograft models from FA head and neck squamous cell carcinoma tumors, characterize tumor-specific genetic alterations, and perform drug screening studies.

Amount funded: $100,000

Modeling the Role of Environmental Responses in Fanconi Anemia Epithelial Stem and Progenitor Cells in FA Squamous Cell Carcinoma

Ken Weinberg, Hiro Nakagawa, Frank Ondrey, Daria Mochley-Rosen, and Craig Emelts
Stanford University, Columbia University, University of Minnesota, and University of Alabama at Birmingham

The issue: identifying safe and effective therapies for FA cancers requires preclinical models that faithfully recapitulate the natural progression of disease.

The project: this grant will develop models to better understand squamous cell carcinoma in individuals with FA using animal models and organoids created from FA patient cancer cells. Developing these models is significant because they will enable researchers to better understand the pathways that result in cancer and determine the best prevention and therapeutic strategies to eliminate FA cancers.

Amount Funded: $500,000

New Grant: Fanconi Anemia Neurological Syndrome

FANS – a Search for a Cause with Advanced Technologies

Prashanth Ramachandran and Michael Wilson
University of California San Francisco

The issue: Fanconi Anemia Associated Neurological Syndrome (FANS) is a rare condition that affects a subset of people with FA. FANS results in brain lesions that can lead to weakness, seizures, and cognitive issues, but the cause is currently unknown.

The project: the aim of this grant is to examine the immune profiles of individuals with FA who have been diagnosed with FANS to find the underlying cause and an appropriate therapy to treat this condition.

Amount Funded: $71,118

Grants that concluded this winter:

Chemoprevention of Cancer in Fanconi Anemia

Markus Grompe
Oregon Health Sciences University

Issue: preclinical models that closely follow the natural progression of FA cancers – as observed in humans – are needed to better understand how to prevent and treat these cancers.

The project: the research team established a mouse model that develops squamous cell carcinoma by incorporating specific Fanconi gene mutations into the animals. The team will now use this model to test drugs that have the potential to prevent or delay tumor formation in Fanconi patients.

Categories: Announcements

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