One of FARF’s guiding principles is to gather researchers and clinicians in the FA community every year to share updates in research and care. In September 2019, we held the 31st Fanconi Anemia Scientific Symposium in Chicago, Ill. Forty-four presenters gave talks and 58 presented posters. Presentations addressed a number of topics relevant to the understanding and treatment of Fanconi anemia. Topics included the FA pathway, gene editing and gene therapy, hematopoietic stem cell biology, central nervous system abnormalities, and cancer in FA.
Read research updates presented during scientific sessions:
Dr. Stella Davies (Cincinnati Children’s Medical Center) opened the 2019 FARF Scientific Symposium with a session entitled “It is not in the stars to hold our destiny but in ourselves”. The appropriately-titled panel featured talks from four different stakeholders in the FA community:
Jack Timperley, adult with Fanconi anemia
Dr. Lindsey Romick-Rosendale, Cincinnati Children’s Medical Center, FA researcher
Dr. Margaret MacMillan, University of Minnesota, FA clinician
Rutger Boerema, parent of two children with Fanconi anemia
Fifty-eight posters were presented covering a range of topics, including bone marrow failure, patient registries and natural history studies, cancer in FA, and the FA pathway. Two poster receptions allow for presenters to share their work with other researchers, doctors, and FA family members.
Each year, a panel of expert FA researchers and clinicians determine the best three abstracts/posters. Each winner is presented with an award and a $500 check. Congratulations to the 2019 abstract/poster award winners:
Best Clinical – Melody Mazon for “Molecular chaperone binding profiles reflect cellular phenotypes of Fanconi anemia missense mutations”
Best Translational – Jose Casado for “NKG2D – ligands expression in hematopoietic stem cells from FA patients: implications in Bone Marrow Failure”
Best Basic – Ivan Rosado for “A novel endogenous source of DNA damage in Fanconi anemia”
FARF Tank made a splash for the second year! The contest allows up and coming researchers an opportunity to pitch their innovative ideas to the scientific community and win a $10,000 grant. Eight contestants had only five minutes each to pitch their ideas to the audience and our panel of judges. At the end, the audience voted for the project they thought most deserved the $10,000 grant. The judges also chose a winner. Congratulations to this year’s winners:
Sylvie Van Twest (people’s choice): Expanding the Fanconi Anemia research and diagnostic toolkit: Developing alpaca nano-antibodies
Adam Nelson (judges’ choice): Viral Specific T cells for HPV-associated SCC
Jack, 20, lives in Park Ridge, Illinois, where he is a student of philosophy and business administration at Northeastern Illinois University. As a member of the Fanconi Anemia Adult Council, Jack serves as a voice for other adults and teens living with FA. He works with the FARF staff and board of directors as an advisor and advocate for the needs of the adult FA population. In this role, he has also spoken at several fundraisers all over the country, telling his story and expressing to supporters just how crucial their role is to advancing research. After he graduates, Jack hopes to pursue a PhD in a philosophy-related discipline and eventually start his own research and technology company.
Dr. Grant Rowe of Boston Children’s Hospital became the third recipient of the David B. Frohnmayer Award for Early Investigators. The award was inaugurated in 2015 to honor the memory of FARF co-founder David Frohnmayer, who passed away earlier that year. To honor David’s commitment to mentorship and to young investigators, the award is meant to acknowledge an early investigator who has significantly impacted FA science and contributed to the FA community at large.
The FA Research Fund has been around for more than 30 years, and in that time, few researchers can say they’ve dedicated their career to studying Fanconi anemia. Dr. Alan D’Andrea is one of those researchers. He first received funding from FARF in 1998, and has continued to make tremendous advances over the following 20+ years. We are immensely grateful for the work of Dr. D’Andrea and his team.
Thank you to the 2019 FARF Symposium sponsors, Rocket Pharma, Foresee Pharmaceuticals, and Colombia Bank.