Researchers at the University, Oregon Health and Science University (OHSU) and Harvard University recently received a grant to study Fanconi anemia (FA), a rare and fatal genetic disorder.
The inter-institutional team will research new and existing drugs and compounds that may prevent the complications associated with FA. The research also has broad relevance for people with cancer.
The team of researchers consists of Markus Grompe from OHSU, Grover Bagby from OHSU and John Postlethwait from the University. Co-investigators are Alan D’Andrea from Harvard and Susan Olson from OHSU.
FA can lead to bone marrow failure and cancer, according to the Fanconi Anemia Research Fund’s Web site. FA is extremely rare; therefore, it is difficult to test for and diagnose. An estimated one in 350,000 people worldwide have FA.
“This is a terrible disease with no good treatment available,” D’Andrea said. “Even though this is a rare disease, we believe the kinds of treatments we’re researching will be of great benefit to people with anemia and people who are prone to develop cancer in the general population.”
The inter-institutional team received the prestigious $10.7 million grant from the National Heart Lung and Blood Institute, a branch of the National Health Institute (NIH).
Some of the researchers have collaborated together in the past, and this new grant from NIH is a sign toward further progress. Grompe is leading the investigation team after the original head investigator retired. Postlethwait is new to the team.
Research on FA is important to breast cancer, ovarian cancer and prostate cancer treatments because one of the FA genes is involved in all of those cancers. “This work has broad relevance,” D’Andrea said.
The team will research thousands of drug candidates in mice at OHSU, zebrafish at the University and human cell lines at Harvard, according to the University’s May 26 press release. One thousand of the drug candidates who will be screened have been approved by the Food and Drug Administration to treat other medical conditions. Another 10,000 molecules with similar properties will be screened for possible therapeutic benefits, according to the University’s press release. Some of the drug candidates were identified at a FA Research Fund workshop in Portland. The fund, founded by former University President Dave Frohnmayer and his wife Lynn, provided about S650,000 to pursue ideas created at the workshop, according to the University press release. Some of the potential drug candidates were first identified at a Fanconi Anemia Research Fund workshop in Portland.
Two of the Frohnmayer children, Kirsten and Katie, died from the disorder. Their third daughter, Amy, is 23 years old and is getting her Master’s degree from Stanford this month. She has FA.
“If you pass her on the street, you wouldn’t know she has this lethal disease,” Frohnmayer said.
Amy is at high risk for bone marrow failure and cancer because of her FA. The research grant is great news for the Frohnmayers.
“It’s so heartening and important to see this multi-institutional grant,” Frohnmayer said. “These things extend hope in a tangible way.”
Bagby, one of the principal researchers, is the chair of the scientific advisory board of the FA Research Fund and friends with the Frohnmayers. “There’s a personal side to this (research) too,” he said.
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Researchers to study fatal genetic disorder
Daily Emerald
June 3, 2010
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