Research progress is often described through its most visible moments: a published finding, a clinical trial or a new treatment. But those moments depend on years of less-visible work: researchers must gather evidence, develop the right tools, test promising ideas and determine what is safe before a new approach can reach people.

For those affected by Fanconi anemia, this groundwork is especially important. People with FA face an extraordinarily high risk of cancer and can be particularly vulnerable to the harmful effects of standard treatments.

Three recent FCF investments show how laboratory research and clinical experience work together to enable us to pursue better options.

Exploring whether gene therapy could prevent oral cancer

People with FA face a substantially increased risk of head and neck cancer, particularly cancer in the mouth. Researchers are asking whether gene therapy might one day help reduce that risk before cancer develops.

With FCF support, Markus Grompe and his team at Oregon Health & Science University are testing this idea in FA mouse models. The researchers are studying whether gene therapy can be delivered to tissues lining the mouth and evaluating its safety and potential effectiveness.

This work is preclinical, meaning more laboratory research will be needed before researchers can determine whether this approach should advance toward study in people.

Identifying drugs that may be safer for people with FA

Cancer prevention and treatment both present a central challenge in FA: A drug must not only work against cancer, but also be safe for people with underlying DNA-repair vulnerabilities.

Markus Grompe and Craig Dorrell at Oregon Health & Science University are testing potential cancer-prevention compounds and screening chemotherapy drugs in FA mouse models.

The goal is to build a stronger body of evidence about which compounds may prevent or treat cancer without causing unacceptable harm in FA. This research is also preclinical, with the idea that it will help researchers prioritize the most promising approaches for further study.

Turning shared clinical experience into evidence

Because FA-associated cancers are rare, important information can remain scattered across individual cases and institutions. FCF’s Virtual Tumor Board brings clinicians and researchers together to discuss complex cancer cases and share their expertise.

Now, FCF is funding Agata Smogorzewska and Tamar Berger at The Rockefeller University to take the next step: gathering and analyzing medical information from individuals whose cases were presented through the Virtual Tumor Board.

Many individuals and families have already consented to participate, and information has been shared with the research team for analysis. The project is expected to produce two scientific publications, including the first published analysis of a range of FA-associated cancers discussed through an FA-specific tumor board.

This work will provide the first comprehensive analysis of FA-associated cancers presented through a virtual tumor board housed outside an academic institution. The findings will give clinicians and researchers valuable information about the diagnosis, treatment and outcomes of FA-associated cancers and help influence future cancer care guidance.

Connecting research at every stage

Each project addresses a different part of the same urgent challenge:

• Testing whether oral gene therapy could reduce cancer risk
• Screening prevention and treatment drugs for effectiveness and FA-specific safety
• Turning clinical experience into evidence the field can use

Together, they show why laboratory and clinical research can’t happen in isolation. Questions and experiences from cancer care can help guide research priorities, while laboratory studies can identify approaches that may eventually warrant further clinical investigation.

Across these three projects, FCF has committed $631,164. This is what sustained research investment makes possible: connecting work across different stages so that evidence, tools and promising ideas can continue moving toward safer cancer care.