Research Grant Archives - Fanconi Cancer Foundation

Developing a DNA Ploidy Analysis Platform (2025)

Sherri Van Ravenhorst — Tue, 25 Nov 2025 17:45:46 +0000

This project builds on ongoing collaborations among BC Cancer, the NIH, and the University of Düsseldorf. Drs. Guillaud and Laronde are developing an automated DNA ploidy analysis platform for oral brush samples from individuals with FA.

This innovative, noninvasive method enables frequent monitoring of cellular changes over time, providing critical insight into how precancerous lesions progress. Earlier detection and intervention can lead to better outcomes and a deeper understanding of FA-related cancer development. FCF extends sincere gratitude to Fanconi Canada for their continued partnership and support of this research.

This project is a part of the FA Cancer Consortium.

Thank you to project sponsor Fanconi Canada.

The post Developing a DNA Ploidy Analysis Platform (2025) appeared first on Fanconi Cancer Foundation.

Development of translational read-through-inducing drugs for Fanconi Anemia caused by nonsense mutations

Sherri Van Ravenhorst — Fri, 07 Nov 2025 23:32:36 +0000

Project Overview: This project addresses an urgent need for alternative therapies for individuals with FA aimed at preserving stem cell function, reducing genome instability, and delaying cancer onset. By developing novel therapeutic strategies utilizing translational-read-through-inducing drugs (TRIDs), nonsense-mediated decay (NMD) inhibitors, and anticodon-engineered tRNAs (ACE-tRNAs) Drs. Cipolli and Bezzerri will evaluate the restorative ability of these drugs and their potential as future therapies for individuals with FA who have nonsense mutations (20-30% of individuals with FA).

Impact Statement: This groundbreaking work aims to restore functional FANC proteins by combining TRIDs, ACE-tRNAs and NMD inhibitors in immortalized FA patient derived cells. This therapeutic strategy for nonsense mutations will guide treatments for a significant proportion of FA individuals and may be utilized in the future for other bone marrow failure syndromes.

The post Development of translational read-through-inducing drugs for Fanconi Anemia caused by nonsense mutations appeared first on Fanconi Cancer Foundation.

Cancer Prevention in Fanconi Anemia through Alleviating Formaldehyde Genotoxicity

Brettany Frederick — Thu, 23 Jan 2025 20:36:54 +0000

In collaboration with the American Association for Cancer Research, a global leader in cancer research, we’ve presented Dr. Wang with the 2024 Fanconi Cancer Foundation-AACR NextGen Grant for Transformative Cancer Research. Through this groundbreaking work, Dr. Wang seeks to prevent cancer in patients with Fanconi anemia (FA) by targeting specific nutritional and metabolic pathways.

The accumulation of DNA damage and genetic mutations ultimately causes cancer. This process is greatly accelerated in children and young adults with FA. A recent advancement in the field is the discovery that our bodies produce high quantities of reactive chemicals called aldehydes, which cause DNA damage in FA patients. In this project, Dr. Wang will leverage mouse models and FA patient samples to study the nutritional and metabolic pathways that regulate aldehyde production, with the aim of targeting these pathways as novel cancer prevention strategies for FA patients.

Researchers: Meng Wang, MD, PHD

The post Cancer Prevention in Fanconi Anemia through Alleviating Formaldehyde Genotoxicity appeared first on Fanconi Cancer Foundation.

Oral Cavity Gene Therapy

Sherri Van Ravenhorst — Mon, 16 Dec 2024 19:25:04 +0000

This supplemental funding builds on the groundwork laid by the 2022 grant awarded to Drs. Markus Grompe and Raymond Monnat, titled Oral Mucosal Gene Therapy as a Prevention for FA-Associated Cancers. The proposed research focuses on reducing the risk of squamous cell carcinoma (SCC) in individuals with Fanconi anemia (FA) by correcting the FA genetic defect in K14-positive basal stem cells, which are the origin of SCC.

People with FA are at much higher risk of developing SCC because their K14 basal stem cells in the squamous epithelium are particularly vulnerable. Gene therapy that corrects this genetic defect directly in these cells offers a potentially transformative approach to cancer prevention. This research seeks to determine if gene-corrected oral stem cells have a selective advantage, allowing healthy cells to outcompete those with mutations. The results will help assess the viability of targeted gene therapies for FA, advancing a new and highly promising strategy to reduce cancer risk in this population.

The post Oral Cavity Gene Therapy appeared first on Fanconi Cancer Foundation.

Small Molecule Screening in Fanconi Anemia Mice

Sherri Van Ravenhorst — Mon, 16 Dec 2024 19:22:35 +0000

This supplemental funding builds on the work from the 2020 Fanconi Cancer Foundation grant awarded to Dr. Grompe, titled Chemoprevention of Cancer in Fanconi Anemia, and additional research supported by the Stand Up To Cancer head and neck cancer team in 2022. Chemoprevention studies are crucial because people with FA have an extremely high risk of developing squamous cell carcinoma (SCC), and standard cancer treatments are often too toxic for them. This project uses advanced animal models to identify compounds that may delay or prevent cancer from developing. It addresses a critical need in FA care by identifying chemopreventive agents that are both safe and effective for this vulnerable population. These findings will help lay the groundwork for future clinical trials, aiming to improve cancer prevention and treatment outcomes for people with FA.

The post Small Molecule Screening in Fanconi Anemia Mice appeared first on Fanconi Cancer Foundation.

Extended Funding: Development of In Utero Therapies for Fanconi Anemia

Sherri Van Ravenhorst — Mon, 16 Dec 2024 18:42:38 +0000

This is an extension of a project funded in 2023. Correcting FA mutations in all cells of the body may prevent issues such as bone marrow failure and cancer in people with FA. Since mutations in FA genes start during the gestational process, the ideal time to correct genes may be in utero. The goal of this study is to use laboratory-based experiments to determine whether gene editing in utero (during gestation) can correct FA gene variants in various tissues of the body.

The post Extended Funding: Development of In Utero Therapies for Fanconi Anemia appeared first on Fanconi Cancer Foundation.

Spatial Analysis of FA Tumors for Detection of Their Immune Repertoire and Potential Actionable Targets

Sherri Van Ravenhorst — Wed, 04 Sep 2024 17:47:18 +0000

Fanconi anemia (FA) patients show an increased predisposition to squamous cell carcinomas (SCC). Tumors, being complex cellular ecosystems, are composed of a myriad of different cell types including malignant cells, infiltrating immune cells, fibroblasts, and endothelial cells. Interactions among these cell types can determine the prognosis and clinical management of the patient. Infiltrating immune cells have demonstrated to be promising actionable targets in novel therapeutic strategies through the enhancement of their anti- tumoral response. These insights, nonetheless, remain unknown in FA SCC.

In this work we propose to perform spatial analysis of SCC from patients with FA. The technologies that we propose to use (t-CycIF and GeoMX) work with FFPE samples stored in the pathological archives of hospitals. These technologies allow identification of tumor cell types with very high resolution. We will identify markers for patient’s stratification, prognostic markers, and very importantly to propose therapeutic targets for the treatment of patients with cancer, mainly immunotherapy. This will allow us to learn from the past and project improvements for the future of patients with FA.

The post Spatial Analysis of FA Tumors for Detection of Their Immune Repertoire and Potential Actionable Targets appeared first on Fanconi Cancer Foundation.

Transforming Treatment of Inherited Bone Marrow Failure in Fanconi Anemia by Precise In Vivo Genome Editing

Sherri Van Ravenhorst — Mon, 01 Apr 2024 20:07:47 +0000

Fanconi anemia (FA) is a rare inherited bone marrow failure syndrome characterized by chromosomal abnormalities and increased susceptibility to cancer. Allogeneic bone marrow transplantation is currently the only treatment for bone marrow failure, but its use is limited by the need for a suitable donor and potential side effects.

Base and prime editing will be implemented to target frequent mutations described in individuals with FA and will be combined with optimized viral and non-viral delivery systems to target HSCs in vivo to provide a promising avenue for the treatment of this inherited bone marrow failure syndrome.

Development of In Utero Therapies for Fanconi Anemia

admin — Wed, 20 Mar 2024 12:40:00 +0000

Correcting FA mutations in all cells of the body may prevent issues such as bone marrow failure and cancer in people with FA. Since mutations in FA genes start during the gestational process, the ideal time to correct genes may be in utero. The goal of this study is to use laboratory-based experiments to determine whether gene editing in utero (during gestation) can correct FA gene variants in various tissues of the body.

The post Development of In Utero Therapies for Fanconi Anemia appeared first on Fanconi Cancer Foundation.

Building a Fanconi Anemia Data Commons

admin — Wed, 20 Mar 2024 12:38:38 +0000

Studying a rare disease such as Fanconi anemia is challenging: data must be captured from as many treatment locations as possible. No single institution can have a breakthrough; it requires sharing as much data as possible from as many individuals as possible. Since 2015, Data for the Common Good (D4CG) has been building data commons based on the latest technological breakthroughs and leading international efforts to improve pediatric cancer and rare disease research through better data collection and storage. Through this grant funding, D4CG – in partnership with FARF and other worldwide stakeholders – will establish the world’s first Fanconi anemia data commons by collecting data from registries in North America, Europe, and other parts of the world and harmonizing those data to a new international consensus standard, allowing the information to be combined and shared for research and discovery.

The post Building a Fanconi Anemia Data Commons appeared first on Fanconi Cancer Foundation.