Promising research needs continued support to answer the next question. A laboratory finding may need testing in more models. A screening study may need years of follow-up. An approach ready for a clinical study needs the resources to evaluate safety and learn from participants’ experiences.
On Oct. 1, the Fanconi Cancer Foundation’s Board of Directors approved nine research awards totaling approximately $2.35 million. The awards will catalyze research on cancer prevention, earlier detection, treatment discovery, blood and marrow research, and the shared information needed to improve care.
Test familiar drugs for cancer prevention
Earlier FCF support helped bring a preclinical study of naproxen and metformin to the point of clinical testing in a cancer prevention study. A renewed $250,000 award to Dr. Agata Smogorzewska at The Rockefeller University and Dr. Rachel Uppgaard at the University of Minnesota will enroll 48 participants with FA.
The study will test the drugs during separate intervention periods, assessing safety, tolerability, oral lesions and biological changes associated with cancer development. The question is whether these drugs can safely alter changes that precede oral cancer in FA. Their use for other conditions does not establish that they can prevent cancer in people with FA.
Build stronger evidence for earlier detection
A $249,850 award supports Dr. Lisa McReynolds’ comprehensive cancer screening program at the National Cancer Institute at NIH. With more than 100 people enrolled, the program combines screening with the collection of samples over time. Continued support will help researchers evaluate potential cancer warning signs and prepare information for shared research through FRIENDS.
A $240,881 award to Drs. Martial Guillaud and Denise Laronde at BC Cancer, funded by Fanconi Canada, will advance oral cancer biomarker research. The team will study abnormal DNA content in brushed oral cells and additional biological markers, following changes over time. The aim is to help identify which lesions warrant closer evaluation.
Find treatments that spare vulnerable healthy cells
Earlier FCF-funded research helped establish development of lab-grown organoids, models of healthy, precancerous and cancerous tissue from people with FA. That work has already identified drugs that preferentially kill cancer cells, providing candidates for further investigation.
A renewed $329,690 award to Dr. Hiroshi Nakagawa at Columbia University and Dr. Kenneth Weinberg at Stanford University will help the team assess the most promising drug candidates for prevention or treatment of FA cancer. The researchers will examine the selectivity of the drugs and whether they cause DNA damage. The goal will be to identify drugs that are suitable for eventual clinical testing. Testing healthy FA tissue alongside cancer is central to that work.
Reduce damage before it leads to disease
Two new awards investigate ways to limit DNA damage caused by aldehydes, substances produced during normal metabolism that FA cells can have difficulty managing.
Dr. Grant Rowe at Boston Children’s Hospital will receive $250,000 to investigate ways to boost an enzyme called ALDH9A1, which helps break down aldehydes. The work grew from an observation in blood-forming cells from a person with FA who had not developed marrow failure. Laboratory studies suggested that a variant in the enzyme could improve removal of damaging aldehyde byproducts. The new project will look for drugs that activate the enzyme and test their protective potential.
Dr. Meng Wang at Cornell University will receive $249,995 to test approaches that intercept formaldehyde before it damages DNA. The work includes investigating whether sustained drug treatment can protect against marrow failure and leukemia in FA models. Both projects remain in laboratory and animal research, where effectiveness and safety must be established before studies in people.
Explore new ways to address blood and marrow disease
A $250,000 award to Drs. Branden Moriarity and John Wagner at the University of Minnesota will support development of a gene correction approach in FA blood-forming cells and mouse models. The technique seeks to insert a functioning gene, potentially allowing correction directly in the body in the future.
A separate $200,000 award to Dr. Agnieszka Czechowicz at Stanford University and Dr. Tippi Mackenzie at the University of California, San Francisco, supports initiation of an early clinical study of prenatal stem cell transplantation for FA. The study will use maternal blood-forming stem cells delivered through the fetal umbilical circulation.
The prenatal study will investigate an approach intended to prevent blood and marrow disease. Its potential benefits remain unproven, and maternal and fetal safety, lasting establishment of the transplanted cells and long-term outcomes are essential questions. Neither project establishes a way to correct all tissues affected by FA or prevent solid tumors.
Connect information across institutions
A renewed $330,000 award to Dr. Samuel Volchenboum and collaborators at the University of Chicago will support FRIENDS, the 19-institution consortium building a shared data resource for FA research.
The next steps include compiling clinical data from the 19 participating institutions and piloting contributions of electronic health records from individuals. This resource will enable research on cancer, transplant effects, fertility and other aspects of FA care across a larger community. The award includes $80,000 for external institutional work to prepare records for shared analysis.
Continue the work earlier support made possible
These nine awards reflect different stages of research, from discovering how cells protect themselves to testing an intervention in people. Each supports a defined next step, with further evidence needed before an approach can become part of care.
For donors, continued funding allows researchers to build on earlier contributions rather than stop at an encouraging result. For people affected by FA, it supports work toward more informed care, earlier detection and safer options. We’re grateful to the individuals, families, donors and partners helping make these next steps possible.
