The Fanconi Cancer Foundation is proud to partner with organizations around the world that are working to improve awareness, care, and support for people living with rare diseases, including Fanconi anemia. One of these partners, the Ivan & Joan Foundation, based in Tanzania, continues to advance important advocacy efforts in its country through leadership and community engagement.

Recently, Winlady Boniface, founder of the Ivan & Joan Foundation, was invited to serve as a panelist at a national rare diseases symposium. The event brought together representatives from key government departments, health leaders, and advocates to discuss challenges and opportunities in rare disease care. Dr. Grace Magembe, Chief Medical Officer at the Ministry of Health, served as the guest of honor.

During the symposium, Winlady spoke on behalf of families of children living with rare diseases and highlighted several critical challenges affecting access to care:

• Delayed diagnosis of rare diseases
• Limited availability of orphan drugs
• Lack of health insurance coverage for costly treatments, including bone marrow transplants
• The absence of a dedicated unit within the Ministry of Health that provides accurate information about rare disease treatment

In response to these challenges, she presented several recommendations aimed at strengthening national support for rare disease communities:

• Establish a national rare disease database
• Create a specialized Center of Excellence for rare diseases to provide diagnostic tools, access to orphan drugs, and psychosocial support
• Include provisions for rare diseases within health insurance policies so that a portion of treatment costs, such as 50 percent, can be covered
• Develop a dedicated rare disease department within the Ministry of Health
• Integrate rare diseases into national health strategic plans

The symposium created an important opportunity for dialogue with government leaders and decision makers. According to Winlady, the discussions were productive, and officials from key departments responded to the issues raised and committed to exploring potential solutions.

Advocacy efforts like these play an essential role in building stronger systems of care for rare diseases. By elevating the experiences of families and engaging policymakers, organizations such as the Ivan & Joan Foundation are helping move rare disease awareness and care forward in their communities.

The Fanconi Cancer Foundation is honored to support and collaborate with partners around the world who are working to improve the lives of individuals and families affected by Fanconi anemia and other rare conditions.

To help community members explore advocacy opportunities, FCF offers coaching to support them in shaping their story and identifying a clear call to action. Please contact us if you’re interested!

The post Speaking Up for Rare Disease Communities appeared first on Fanconi Cancer Foundation.

Before my diagnosis, I just didn’t feel good for a while. I had frequent nosebleeds, bruised easily, and was tired a lot. Eventually, those symptoms led to testing and my FA diagnosis. At the time, I didn’t fully understand what was happening, but looking back, I can see how much my body was going through even at such a young age.

Today, I get to live a pretty normal life. Most of my day-to-day focus is on routine checkups, and for the most part, life feels typical. Still, there’s always a small, constant worry in the back of my mind that something could change. Living with FA means learning to hold both things at once: gratitude for feeling well and awareness that FA doesn’t simply disappear.

One thing I wish more people understood is that FA doesn’t go away after transplant. Even when things look normal from the outside, it’s something you live with for life. That reality shapes how you think about your health, your future, and the care you take with your body.

A defining moment for me came as I got older and started to understand how much I went through, even though I don’t remember all of it. Realizing that my life could have turned out very differently made me appreciate my health and where I am now in a deeper way. It changed how I see my past and how I approach my future.

I’m a high school junior and also a concurrent college student. I’m involved in school leadership, and I hope to work in health care someday. My goal is to become a CRNA and work in pediatrics, giving back in a meaningful way to the kind of care that made such a difference in my own life.

Looking ahead, I hope to stay healthy and see continued progress in FA research. In 2026, I’ll be celebrating several milestones: 10 years post-transplant, four years post spinal fusion, and turning 18. Those moments feel significant, not just as anniversaries, but as reminders of how far I’ve come.

To the donors who make research possible, thank you. The research and care you support truly change lives. You’ve helped make a future like mine possible.

FA is part of my life, but it’s not who I am.

The post A Part of My Life, But Not Who I Am appeared first on Fanconi Cancer Foundation.

My name is Carly, and I’m sharing our story as the parent of a child with Fanconi anemia (FA). My daughter, Paige, was diagnosed when she was 11 years old. Like many families, we had never heard of FA before it became part of our lives.

Paige’s diagnosis came when she presented at the hospital with bone marrow failure. Everything happened quickly, and suddenly we were trying to understand a rare, lifelong condition while also making decisions no family ever expects to face. It was overwhelming, frightening, and isolating.

In May 2015, Paige received a bone marrow transplant. Her sibling wasn’t a match, so we were incredibly grateful to learn that a donor overseas was. Finding that match was a defining moment for our family. It brought hope during a time when we were holding our breath, waiting for answers. At the same time, we had to have our other daughter tested, and the months of waiting to learn whether she might also have FA were some of the hardest we’ve lived through.

Daily life since then has been shaped by recovery and care. Paige is still recovering from her transplant, and supporting her through long periods of isolation and ongoing medical procedures has been challenging for our whole family. Living with FA means being constantly mindful. It’s a lifelong condition, and Paige will need to live a very healthy lifestyle to protect her health as much as possible.

One thing I wish more people understood about Fanconi anemia is exactly that: it doesn’t end with a single treatment or milestone. It’s something she will carry with her throughout her life, even as she grows, dreams, and plans for the future.

Paige is so much more than FA. She has a deep love of bugs and spiders and dreams of becoming an entomologist or arachnologist one day. She was also passionate about gymnastics and loved being active, until treatment made it no longer possible. Seeing her adapt and keep her curiosity and joy, even when so much was taken away, has been both heartbreaking and inspiring.

What I hope for most is a cure. And until then, I hope for Paige and others with FA to live lives as close to normal as possible, filled with opportunity, independence, and happiness.

To the donors who make research possible, thank you. Truly. Your support means hope for families like ours, and it reminds us that we’re not facing this journey alone.

The post Walking Beside My Daughter Through Fanconi Anemia appeared first on Fanconi Cancer Foundation.

Tara (center right) with her sisters Natalie and Brenna, and mom Mary Eilleen

Fear and sadness are embedded with Fanconi anemia (FA), yet I have always tried to have a hopeful tone to my essays thinking of the effect on the families who read it. This one is no different. I lost my gutsy 24-year-old daughter Tara in September. Her siblings nicknamed her “Puce” because she had amazing red hair. If anyone acted like a smart Alek, they were acting “pucey.” You get the idea. With her small frame, I called her the “perfect huggable human,” and I meant it. With her dry sense of humor and big personality, she also was the perfect youngest child of four. I told her that while I had those first three kids in less than four years, I waited, but knew I needed a fourth – I needed her.

She was created by God for purpose. I always told her she brought out the best in people. As the brain lesions associated with Fanconi began to multiply and her abilities began to be whittled away, all the ways we connected, and all the ways we reinforced faith in something bigger than this earth, better than a physical body that was failing, became eternally important. I would assure her that she was not her body – she was much more. She had a soul. 

Tara; and with her siblings Natalie, Brenna and Connor at Brenna’s wedding

The summer of 2025 was horrific. There were multiple aspirations and multiple hospitalizations that included an intubation. I was always hopeful when we got her home. While she was incredibly thin after continually losing weight and muscle mass, I would be encouraged by each bite she took. She could stand and take some steps with considerable help, but I told myself she was getting stronger. Then a week or so after being dismissed from the hospital, she slumped and couldn’t speak. We had dealt with so much with all the brain lesions, the seizures, the ebbing and flowing that sometimes gave a whisper of hope to her devastated family. I thought maybe the bladder infection they had been treating her for had reoccurred. We brought her into the emergency department of our local hospital in Grand Rapids, Michigan – a hospital whose doctors almost routinely admitted they knew nothing about FA, much less Fanconi Anemia Neurological Syndrome (FANS). I asked for a brain MRI as well, and they refused saying we couldn’t get one in the ER. I knew that wasn’t correct, but I justified the decision thinking they must believe it was the bladder infection, which can cause neurological symptoms in people prone to such things. We took her home.

Tara as her bright, colorful self; with her beloved dog Lily; and with her nephew Warren in early 2025, who locked right onto her!

Two days later, we were back insisting we needed a brain MRI. We succeeded in getting one, and it showed the largest lesion Tara ever had. She had what some doctors had called “speckle brain,” a lot of tiny lesions throughout the brain. This one was different – it took her speech and then within a few weeks, her life. 

Tara definitely fought the good fight. She rarely gave in to despair but often expressed confusion about what was happening to her. She said in her last year, “My body keeps breaking. I tell myself to stop breaking.” What was breaking was all our hearts. She didn’t deserve any of the heartache that FA and FANS delivered. None of the people with FA do. The added trial of FA is that one never knows which body part it will strike and with what. 

Little Tara on the way to her Make-A-Wish trip

So, how do we nurture hope? By grabbing gratitude with both hands. My hope and prayers for a cure for Tara went unanswered, but we are pulling for those still in the fight! I hurt so much for the life Tara didn’t get and the one she did, but I immerse myself in gratitude as much as I can. Gratitude: for family – so many prayed and supported; for friends – countless showed up for the visitation and funeral; for teachers from high school and even elementary school – they connected with Tara and they came to comfort us; for doctors, nurses, and physical therapists – many called me, more wrote, some sent plants and flowers and several paid their respects in person. Unbelievable. As a Catholic, I’m also grateful for the priest who showed up at our house the day she died to pray with us and then commended her soul to God at her funeral Mass. It’s comforting to understand how in her short life, she touched so many. 

The much adored little sister of the family

For those still running in the FA race we collectively hate, you have amazing, dedicated researchers and physicians who work in a lab and with patients and families, and countless others involved in the fight. We have the Fanconi Cancer Foundation with professionals of all sorts working in the trenches, serving those of us who suffer. 

Thanks to Tara for showing us an incredible example of faith, grace and courage, and thank you to everyone who aids in the war against FA. 

Gratitude may be cloaked in tears for now, but it lives. 

The post Tara Fought FANS with Grace and Courage; She Finished the Race appeared first on Fanconi Cancer Foundation.

Advancing a Natural History of FA-Associated Cancer

Investigators: Neelam Giri, MD, and Lisa McReynolds, MD, PhD
Institution: National Cancer Institute, NIH
Funding Amount: $249,950

Drs. Giri and McReynolds will continue their work developing an evidence-based cancer screening program for individuals with FA through supplemental funding to their existing NIH Center Comprehensive Program. Their project studies the natural history of oral potentially malignant lesions (OPMLs) and investigates biomarkers of carcinogenesis.

The NIH cancer screening program has already enrolled more than 80 participants and collected thousands of biospecimens. By collaborating with other FACC investigators, the team is analyzing DNA damage in these samples, offering valuable insight into how cancer develops in FA. Their work will help establish effective early detection strategies and create a robust biorepository for future cancer research.

Developing a DNA Ploidy Analysis Platform

Investigators: Martial Guillaud, PhD, and Denise Laronde, PhD
Institution: BC Cancer
Funding Amount: $95,787 — Funded by Fanconi Canada

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.

Reducing the Burden of Squamous Cell Carcinoma in FA

Investigators: Eunike Velleuer-Carlberg, MD, and Christine Krieg
Institution: University of Düsseldorf; German Fanconi Anemia Family Support Group and Research Fund
Funding Amount: $74,366

This project continues the long-running “Reducing the Burden of SCC in Fanconi Anemia” initiative, first funded by FCF in 2006. Dr. Velleuer-Carlberg and Ms. Krieg lead efforts to advance oral cancer screening through education, outreach, and research.

Their work has shown the effectiveness of brush biopsy screening and continues to promote awareness among individuals with FA, clinicians, and dentists. Ongoing DNA ploidy analysis of pre-malignant cells will improve early detection and expand understanding of cancer progression in FA.

Testing Chemoprevention Approaches for Oral Cancer

Investigators: Agata Smogorzewska, MD, PhD, and Rachel Uppgaard, DDS
Institution: Rockefeller University; University of Minnesota
Funding Amount: $250,000

With a 500–700 times greater risk of head and neck squamous cell carcinoma, individuals with FA face a pressing need for prevention strategies. Building on findings from FCF’s collaboration with Stand Up to Cancer, this multi-institute clinical trial evaluates naproxen and metformin as potential chemopreventive drugs for oral cancer. The study will assess both safety and biological markers of cancer prevention, establishing the framework for future chemoprevention trials in FA.

A Shared Commitment to Progress

The Fanconi Anemia Cancer Consortium brings together scientists, clinicians, and advocates from around the world who share a mission to reduce the burden of FA-associated cancers. FCF’s continued investment in this work—and the collaboration of dedicated partners like Fanconi Canada—helps drive meaningful progress toward earlier detection, improved care, and ultimately, prevention.

The post Fanconi Cancer Foundation Expands Cancer Consortium Research with New and Continued Grants appeared first on Fanconi Cancer Foundation.

A Global Commitment to Care

Born in Zanzibar and now living in New York, Fatma has dedicated herself to improving how healthcare is experienced, not just delivered.

She currently works as a medical scribe at an OB-GYN clinic, supporting patients through deeply personal moments such as high-risk pregnancies and cancer screenings. She also serves on the Patient and Family Advisory Council at the Meyer Cancer Center through Weill Cornell and NewYork-Presbyterian, helping to make hospital systems more compassionate and inclusive.

“Healing extends beyond treatment plans,” Fatma shared. “It lies in creating spaces where patients feel safe, seen, and empowered, even in the face of uncertainty.”

In her hometown of Zanzibar, Fatma has volunteered in rural clinics, offering encouragement to people who often face limited access to care. “A simple act of kindness,” she said, “can radically shift a patient’s experience.”

Leading Through Empathy

Fatma’s leadership isn’t loud; it’s deeply human. She leads by listening: to patients, to researchers, and to peers in the FA community. Fatma received many nominators for this award who describe her as “a bridge between the patient world and the scientific world,” someone whose voice and empathy “improves how we conduct research, deliver care, and reminds us why we do this work.”

Her brother, Hisham, sees that same strength at home.

“Fatma often leads by example. Throughout her life, she holds others to the same standards she holds herself which is often inspiring. She is always transparent, empathetic, and leads from within.”

Whether in a research setting, a hospital, or a community meeting, Fatma’s steady presence brings people together and reminds them that care begins with compassion.

A Voice for Advocacy and Hope

Advocacy has become a defining part of Fatma’s journey. Through her work with FCF and the broader rare disease community, she’s used her voice to raise awareness and build connection.

“Having been a patient myself, I know how heavy those moments can feel,” Fatma shared in her acceptance speech. “It means a lot to ease that weight, even in small ways.”

She also reflected on the people who have guided her:

“I wouldn’t be standing here without the people who have stood beside me. You’ve shown me what resilience looks like and reminded me why it’s worth continuing to speak up and fight for change.”

Her humility, gratitude, and clarity of purpose inspire everyone around her, a reflection of the FA community’s strength and shared hope.

Looking Ahead

For Fatma, this recognition is not an ending but a beginning. She plans to attend medical school and continue working toward her dream of transforming healthcare for underserved communities around the world.

Her mother, Zaitoon, summed it up perfectly:

“Fatma demonstrates leadership in empowering others and showing them that Fanconi anemia is not a reason to let your dreams drift away. Anything is possible if you truly set your mind to it.”

As she moves forward, Fatma carries the legacy of Amy Frohnmayer Winn and Christopher Byrd, two leaders who lived with courage, joy, and purpose. She continues that legacy with grace, empathy, and a belief in the power of human connection.

Congratulations, Fatma. Your compassion, strength, and vision inspire us all!

Louise Dalgleish Receives Second Place

Louise strives to shift perceptions of adults with Fanconi anemia beyond their medical conditions and works alongside clinicians and researchers to improve patient experiences. She’s passionate about highlighting the ambitions and everyday challenges faced by individuals with FA, advocating for understanding, equity, and support within the medical community.

Louise is also deeply committed to increasing accessibility and inclusion for LGBTQ+ individuals in healthcare. Through her recent collaboration with Pfizer’s OUTpatients campaign, she shared her experience as an LGBTQ+ person living with a rare disease, bringing visibility and awareness to an often-overlooked intersection of identities.

A dedicated member of FCF’s FAdult Council and Advocacy Program, as well as a spokesperson for Fanconi Hope, Louise uses her voice to uplift others and inspire change. Congratulations, Louise! Your courage and advocacy strengthen the entire FA community.

The post Celebrating Fatma Issak, 2025 Winn/Byrd Award Recipient appeared first on Fanconi Cancer Foundation.

At the 2025 Fanconi Cancer Foundation Scientific Symposium in Minneapolis, the Foundation presented its Lifetime Achievement Award to Dr. John E. Wagner, MD, in recognition of his decades of groundbreaking work and unwavering commitment to improving the lives of people with Fanconi anemia (FA).

The award was introduced by Lynn Frohnmayer, co-founder of the Fanconi Cancer Foundation, who reflected on Dr. Wagner’s remarkable influence on the field, and on her own family’s journey. “I recently wrote to John telling him I’d be introducing him for this award,” she said. “He replied, ‘Ha ha. This makes sense. You got me into this—and what a journey it has been.’”

A Legacy of Compassion and Discovery

Dr. Wagner serves as Professor of Pediatrics, Co-Director of the Center for Translational Medicine, and Founding Director of the Institute for Cell, Gene and Immunotherapies at the University of Minnesota. His pioneering research in cellular therapies has transformed the landscape for children and adults with FA.

Lynn recounted how, in the 1980s, families affected by FA faced devastating barriers to care. “If you had a child with FA and no matched donor, you could not find a transplant center willing to help,” she said. “One huge concern was graft-versus-host disease, a potentially lethal complication.”

At that same time, Dr. Wagner, then at Johns Hopkins, was developing an innovative method called counterflow elutriation, which removed specific immune cells from donor marrow to reduce the risk of graft-versus-host disease.

In 1994, when Lynn and her husband, David, learned that their daughter Kirsten had developed leukemia, they turned to Dr. Wagner for help. He agreed to try the new elutriation approach with her, the first time it had been used in an FA transplant. “He was a magnificent physician,” Lynn said. “Brilliant, deeply caring, creative, and forever willing to try a new approach when faced with a challenge.”

Left: Dr. Wagner accepting the Lifetime Achievement Award from FCF Co-Founder Lynn Frohnmayer (L) and Board President Lisa Mingo (R)

Right: Dr. Wagner with his family

Transforming Transplantation for FA

Dr. Wagner went on to pioneer the use of umbilical cord blood as a source of stem cells for transplant, a breakthrough that has since saved countless lives. His work also advanced the idea of embryo selection, allowing parents to conceive a healthy, genetically matched sibling who could serve as a stem cell donor. This innovation led to the birth of FA patient Molly Nash’s brother, Adam, in 2000, an event that captured global attention and opened new ethical and scientific conversations.

Another of Dr. Wagner’s lasting contributions came through his 1998 Fanconi Anemia Research Fund grant to test the safety and effectiveness of a then-novel drug, fludarabine. The results were transformative: transplant survival rates for individuals with FA rose from 15–20 percent to more than 60 percent, and later to over 90 percent today. “Literally overnight,” Lynn said, “we went from near-hopeless odds to outcomes we could only dream of.”

A Journey of Bold Ideas

In his acceptance remarks, Dr. Wagner reflected on that journey with humility and humor. He shared stories from his early days at Johns Hopkins and the many families, scientists, and clinicians who shaped his work. “As every parent would say,” he noted, “we don’t have enough time. We have to have bold ideas. We have to take risks.”

He revisited the controversies and ethical challenges surrounding embryo selection, recalling debates with religious leaders and global media attention that led to threats to his own safety. However, he emphasized the value of open, respectful dialogue between science, ethics, and faith, a point shared with him by Pope Benedict when he was invited to the Vatican in 2011.

Dr. Wagner closed his remarks by honoring the individuals with FA whose courage has guided every advance in his career. “We have to take chances because we might make a difference,” he said. He recognized his colleagues, his wife, Lisa, and the many families who trusted him with their care. “These are the true pioneers,” he told the audience, inviting all who had participated in FA research and treatment to stand together.

Before concluding, he offered a message to clinicians and researchers carrying the work forward: “Engage the public. Tell the truth, even when it’s hard or not well received. Think deeply about what should be done, not only what can be done. Collaborate with ethicists and always keep your promises to the people you serve. Even when an experiment doesn’t achieve what you hoped, learn from it, because that learning will move the field forward.”

Continuing the Work

Today, Dr. Wagner’s commitment to the FA community continues through his service on the Fanconi Cancer Foundation’s Board of Directors and Scientific Advisory Board, and through his focus on one of the greatest challenges ahead: understanding and preventing FA-related cancers. His leadership and collaboration across disciplines are helping to bridge the fields of immunotherapy, gene editing, and cancer biology, advances that will shape the next generation of care for people with FA.

As Dr. Wagner reminded the community, progress requires courage. The work is not finished. To answer the next unanswered questions, researchers and advocates must continue to take bold risks, ask hard questions, and invest in ideas that have the power to transform what’s possible once again.

Through decades of scientific innovation and personal dedication, Dr. Wagner has helped transform the outlook for individuals with Fanconi anemia, from a disease once defined by childhood loss to one where longer, fuller lives are possible. The Fanconi Cancer Foundation is honored to celebrate his extraordinary contributions and his enduring partnership with the FA community.

The post Honoring Dr. John E. Wagner for a Lifetime of Impact appeared first on Fanconi Cancer Foundation.

Nearly 300 members of the global Fanconi anemia community gathered in Minneapolis for the 2025 Scientific Symposium and Retreat for Adults with FA, three days that blended science, lived experience, and hope.

Attendees joined from 18 countries across six continents, representing Argentina, Australia, Canada, Denmark, the Dominican Republic, Germany, Kenya, Mexico, the Netherlands, Pakistan, South Africa, the United Kingdom, the United States, France, Spain, Egypt, Israel, Brazil, Poland, and Portugal.

Community Voices Lead the Way

The meetings opened with a joint town hall that united adults with FA, caregivers, clinicians, and researchers. In heartfelt discussions, individuals with FA shared the realities of daily life with the condition, reminding all in attendance that every scientific advancement begins with real, human experiences.

At the Retreat for Adults with FA, sessions focused on quality of life and access to care. A standout mental health discussion emphasized the need for accessible, FA-informed support and highlighted emerging efforts to connect community members with specialized providers.

Cancer prevention was another central theme. Researchers from the NIH, Germany’s Cancer Awareness Team, and The Rockefeller University shared updates on cancer screening initiatives, while Dr. Agata Smogorzewska previewed the upcoming cancer prevention study. Many attendees participated in on-site oral screenings, underscoring the event’s practical and preventive focus.

“Science doesn’t move forward without community, and community is stronger because of the science.” – attendee 

Members of the FCF Patient Advocacy Program also made history this year, presenting posters for the first time that explored advocacy’s impact on health and daily life. Advocates led panels and spoke alongside researchers, strengthening the bridge between lived experience and scientific discovery.

And of course, no global gathering is complete without a little celebration. The after-party hosted by the German Team was a hit! It was complete with traditional foods and drinks, a lively photo backdrop (featuring some legendary poses), spontaneous singing, and plenty of dancing. 

Shaping the Future of FA Research

At the Scientific Symposium, collaboration took center stage. And behind the scenes, the Scientific Advisory Board (SAB) met to refine FCF’s scientific strategy, focusing on the FA Cancer Consortium and Gene Editing Initiative.

The first in-person FRIENDS Executive Committee meeting marked another milestone. The group’s discussions advanced data harmonization and helped identify top priorities for the FA Research Data Commons, ensuring a unified, global approach to FA research.

International Summit

This year’s International Summit brought together researchers, clinicians, and patient support group leaders from across Europe, Africa, North and South America, and beyond. The exchange of ideas across borders continues to be one of the greatest strengths of this community, and it was inspiring to see collaborations forming in real time.

Discussions spanned topics from cancer prevention to emerging genetic therapies, reflecting a shared commitment to ensuring that progress in FA research benefits individuals and families everywhere. The energy and openness of the Summit embodied what makes the FA community truly global.

Dive Deeper into the Science

Two keynote speakers showcased groundbreaking research shaping the future of cancer and FA biology.

Dr. Siyuan (Steven) Wang, PhD presented “Chromatin Tracing and the Evolution of Single-Cell 3D Genomes in Kras-Driven Cancer.” His work revealed stage-specific changes in 3D genome organization as cancer progresses, offering new pathways to potential diagnostic biomarkers.

Dr. Mike Spencer Chapman, PhD shared insights on “Clonal Dynamics of Stem Cell Transplantation.” His study uses somatic mutations as clonal markers to track donor and recipient cell behavior over time—findings that may guide improved transplant strategies.

Poster Award Highlights

• Best Basic Poster – Cecilia Ayala-Zambrano: Loss of FANCA modifies the 3D organization of the genome in squamous carcinoma cells. FANCA deficiency was shown to alter genome architecture, increasing translocations and new gene fusions.
• Best Translational Poster – Colette Rogers: In vivo correction of a genetically humanized FA murine model using digital editing technologies. Demonstrated proof-of-concept for in vivo gene editing in bone marrow stem cells for FA.
• Best Clinical Poster – Luitpold Distel: Radiosensitivity in patients with breast cancer risk genes in comparison to Fanconi anemia. Found that radiation sensitivity among individuals with certain breast cancer risk genes was modestly higher than average but variable.

Recognizing Excellence and Commitment

The meetings also honored outstanding contributions across the FA community:

• Lifetime Achievement Award: Dr. John Wagner—Professor of Pediatrics and Founding Director of the Institute for Cell, Gene and Immunotherapies at the University of Minnesota—for his pioneering leadership in FA research and cell therapy.
• Winn/Byrd Award for Adults with FA: Fatma Isaak, with Louise Dalgleish as first runner-up.
• Award of Appreciation: Kidz1stFund, recognized for its extraordinary commitment to FA research. Every dollar raised supports research at the University of Minnesota, totaling more than $13.6 million, while 7,700 individuals have joined the national marrow registry through its drives.
• David B. Frohnmayer Early Investigator Award: Dr. Lisa McReynolds for her promising work advancing FA science.

Looking Ahead

The 2025 meetings captured what defines the Fanconi Cancer Foundation community: collaboration, compassion, and shared purpose.

From deeply personal discussions about mental health to forward-looking conversations on data sharing and gene editing, the Symposium and Retreat demonstrate how community and science move forward together.

We look forward to seeing you in Phoenix, Arizona, October 1-3, 2026!

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EUGENE, OREGON – In 2015, Phil and Penny Knight made a bold ten-year pledge of $10 million to the Fanconi Cancer Foundation (FCF) to address the extraordinarily high risk of life-threatening cancer in people with Fanconi anemia (FA), a rare genetic disorder that often leads to highly aggressive malignancies at a young age. Their investment in research focused on FA cancer has provided new knowledge on understanding of DNA-repair biology and its effects on cancer. Research supported by the Knights has shaped best practices in prevention, early-detection, and treatment strategies for cancer, benefiting people with FA and providing far-reaching benefits for the broader population.

Katherine De Los Santos’ lived experience shows the impact Phil and Penny Knight have made. A few years ago, persistent chest pain led her to enroll in the FCF-funded cancer screening trial at the National Institutes of Health, supported by the Knights’ gift. Her esophageal cancer was found early, allowing for swift treatment and recovery. Today, Katherine is cancer-free, continuing her studies toward her dream of becoming a pediatric hematologist-oncologist, and is living proof that early detection saves lives.

“If not for this research,” Katherine shares, “not only would I not be here today, but I wouldn’t have a second chance to pursue my dreams and enjoy the small pleasures of life. Since being in remission this year, I have graduated Mercer University with my master’s degree and gotten engaged to the love of my life thanks to this research!” 

A Decade of Breakthroughs

Over the last ten years, the Knights’ support has enabled research that would never have happened without private philanthropy. Their gift has been instrumental in:

• Advancing early cancer detection: Funding worldwide efforts focused on using brush biopsies, a noninvasive screening tool now showing promise for cancer detection in the broader population.
• Driving innovation in cancer prevention trials: Laying the groundwork for therapies that can prevent cancer, leading to an upcoming clinical trial. 
• Pursuing targeted cancer treatments: Supporting the first head and neck cancer clinical trial for advanced oral cancer in FA, aiming for more effective and less toxic treatment options.
• Advancing gene editing approaches: Supporting research into innovative techniques like base and prime editing to fix the mutations in DNA that cause FA. By correcting these mutations in FA cells, scientists are developing a precise, less risky alternative to bone marrow transplantation and cancer therapy. The ultimate cure for FA will entail gene therapy, which has the potential to fix the underlying genetic cause of this disease. 
• Expanding understanding of the BRCA (breast cancer)–DNA repair pathway: Illuminating mechanisms that cause tumor formation and informing new strategies to prevent and treat cancers affected by faulty DNA repair in all people.

Inspired by Friendship and Shared Vision

The Knights have been loyal supporters of the Fanconi Cancer Foundation for decades. In 2015 they became champions for the FA community with their commitment of a $10 million pledge focused on addressing the urgent cancer risk in FA. By then, two of Lynn and David Frohnmayer’s three daughters had passed away from complications of the disease, and David himself died in 2015 from cancer. Their third daughter, Amy, died of leukemia the following year. 

“The deep sadness of losing three remarkable daughters to FA and the loss of my terrific husband to cancer is with me daily,” shares Lynn Frohnmayer, FCF Co-Founder. “It is mitigated only by the knowledge and belief that the generosity of others, and most especially the transformative gifts of the Knights, is preventing early deaths, greatly extending life expectancy, and has given hope to FA families around the world. Words cannot express the enormity of my gratitude.”

Impact Beyond FA

Although FA is a rare disease, the genes involved are part of a critical DNA repair pathway present in everyone, meaning the Knights’ investment in FA research has had a truly global impact. Our understanding of FA and FA cancer is advancing the broader field of cancer biology, influencing studies on breast, ovarian, and head and neck cancers, and informing prevention strategies that could help countless people.

“When our organization was founded in 1989, physicians considered this a pediatric disease, killing almost every patient in the first or second decade of life, writes Fanconi Cancer Foundation CEO Isis Sroka, PhD. “Today, most patients live into their late 20s, 30s 40s and even later. The game-changing gifts from the Knights are largely responsible for this remarkable expansion in life expectancy. Knowledge about the causes, prevention and non-toxic treatment of cancer developed because of their generosity has applicability for the population in general.”

John E. Wagner, MD, professor of pediatrics and co-leader of the Transplant and Cellular Therapy Program at the Masonic Cancer Center, University of Minnesota, underscores the impact of this research on patients: “Thanks to sustained FA research, survival after blood and marrow transplant now exceeds 90 percent, gene therapy is restoring marrow function in children, and earlier detection and new immunotherapies are changing what’s possible for people with FA. The Knights’ support helped turn discoveries in DNA repair and stem cell biology into today’s life-saving care.”

The Power of Philanthropy in Scientific Research

In today’s climate, with national research budgets under pressure, private donors like Phil and Penny Knight are vital. Their decade-long commitment is transformational, bringing hope to families and changing the trajectory of cancer research.

The discoveries made possible by the Knights’ gift will continue to shape the future of FA and cancer research for years to come. Their legacy shows how vision and generosity can spark remarkable progress, and reminds us that transformative change often starts with a single bold act. 

Learn more: The FA and Cancer Story

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About Fanconi Cancer Foundation
The Fanconi Cancer Foundation (FCF) is the leading organization worldwide dedicated to advancing research, advocacy, and support for people affected by Fanconi anemia (FA) and associated cancers. FA is a rare inherited disease characterized by bone marrow failure, leukemia, and a very high risk of cancer. FA stems from mutations in one of 23 genes, including notable breast cancer susceptibility genes BRCA1 and BRCA2. 

Founded in 1989 by parents Lynn and David Frohnmayer, FCF’s contributions have been instrumental in improving treatments such as hematopoietic cell transplant, significantly enhancing success rates and improving patient outcomes and life expectancy. Studying FA and its connection to cancer not only provides crucial insights into the disease itself but also contributes to a deeper understanding of the complex interplay between DNA repair mechanisms and cancer development. This research has the potential to inform the development of novel diagnostics, preventive strategies, and therapies for various types of cancer. By fostering collaboration and innovation, FCF continues to lead the charge toward effective treatments and ultimately a cure for Fanconi anemia and associated cancers.

The post Phil and Penny Knight’s $10 Million, Decade-Long Gift Transforms Rare Disease and Cancer Research appeared first on Fanconi Cancer Foundation.

My daughter Emily was just six years old when we learned she had Fanconi anemia (FA). After Emily had a series of nosebleeds and unexplained bruises, we took her for routine bloodwork right after Christmas 2021. A day later, her pediatrician called us urgently to say Emily’s blood counts were dangerously low and we needed to bring her to the hospital immediately. While there, she had a bone marrow biopsy and was referred to hematology. After weeks of testing to rule out other conditions, we received confirmation in January 2022—Emily had FA. We were stunned.

Emily had a bone marrow transplant in May 2022. While that procedure was life-saving, it isn’t a cure. This was a turning point for us. It showed me not only what she was dealing with but also her incredible strength at such a young age. It also reminded us that just because she doesn’t look sick, doesn’t mean there isn’t something lying under the surface. She may act like a “normal” kid, but she’s constantly going to doctor appointments and carries an underlying worry of getting sick.

We’re constantly worried about symptoms and about avoiding anything that could increase Emily’s cancer risk. Because of her experiences with emergency hospital visits, Emily is now very afraid of doctors. Any illness or injury can feel frightening for her and for us.

And yet, Emily is much more than her diagnosis. She’s an athlete and an artist who loves playing soccer and basketball. She creates paper dragons for her friends, enjoys drawing, and is a proud Swiftie.

I hope that research continues to expand options and extend life for people with FA. Research brings hope and longevity for those who deal with FA and their families

To the FCF donors, thank you, thank you, thank you for all you do! The FA community is nothing without your support. The Fanconi Cancer Foundation has been such a source of information and support. We appreciate this organization more than you will ever know.

The post When Childhood Meets FA: Emily’s Story of Courage appeared first on Fanconi Cancer Foundation.