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Intellectual Property on Pediatric Cancer is Dedicated to the Public

April 2, 2015

Intellectual property resulting from the discovery of specific DNA mutations linked to a rare and often deadly type of adolescent liver cancer, fibrolamellar hepatocellular carcinoma, has been dedicated to the public by the institutions that made the discovery, The Rockefeller University (“Rockefeller”) and the New York Genome Center (“NYGC”). The institutions made this unusual move in the hope of accelerating progress toward the delivery of diagnostics and therapies for the devastating disease.

The intellectual property was described in an international PCT patent application, “Fusion Proteins and Methods of Use Thereof,” [PCT/US2014/057580] and is based on research described in the journal Science in February 2014. With today’s dedication, scientists worldwide, whether working in academia or industry, are free to use the technology described in the patent application without proprietary constraints.

The research was initiated by Sanford Simon, professor and head of the Laboratory of Cellular Biophysics at Rockefeller. The researchers sequenced DNA and RNA at NYGC from tumors that had been surgically removed from 15 people with fibrolamellar. NYGC computational biologists and members of the Simon laboratory at Rockefeller discussed the possibility of using a series of computer algorithms to search for sequence differences between the tumor samples and samples of healthy liver tissue. One abnormality that was quite evident, and was present in all 15 patients was a piece of DNA that had been broken and incorrectly rejoined, creating a mutated gene that had the potential to wreak havoc in the bodies of individuals with the gene. This translocation, the abnormal protein that results from it, and any diagnostics and therapeutics derived from the information are the subject of the intellectual property being dedicated to the public today.

The work, to which researchers from Memorial Sloan Kettering Cancer Center also significantly contributed, was accomplished using tissue samples donated for research by young people who had undergone surgery for fibrolamellar hepatocellular cancer. Financial support was provided by families and friends of young people whose lives had been touched by the disease, and by generous research funding from the Fibrolamellar Cancer Foundation. Additional support was also provided by theHoward Hughes Medical Institute, the New York Genome Center, The Rockefeller University Center for Clinical and Translational Science and by a gift to The Rockefeller University by an anonymous donor.

“The finding of this previously undiscovered mutation represents the best of what can be accomplished when scientists cross institutional boundaries and join forces to generate genomic discovery,” says Russ Carson, chair of The Rockefeller University’s Board and co-chair of the New York Genome Center’s Board.
“The institutions who today donate this intellectual property to the public firmly believe that making the technology freely available is in the best interests of the patients, families, and philanthropists whose support made its discovery possible.”

About The Rockefeller University
The Rockefeller University is one of the world’s foremost biomedical research institutes and is dedicated to conducting innovative, high-quality research to improve the understanding of life for the benefit of humanity.
Founded in 1901, The Rockefeller Institute for Medical Research was the country’s first institution devoted exclusively to biomedical research. In the 1950s, the Institute expanded its mission to include graduate education and began training new generations of scientists to become research leaders around the world. In 1965, it was renamed The Rockefeller University. Its more than 70 laboratories conduct biological and biomedical research and a community of over 2,000 faculty, students, postdocs, technicians, clinicians and administrative personnel work at the University’s 14-acre campus.
Rockefeller’s unique approach to science has led to some of the world’s most revolutionary contributions to biology and medicine. During Rockefeller’s history, 24 of its scientists have won Nobel Prizes, 21 have won Albert Lasker Medical Research Awards and 20 have garnered the National Medal of Science, the highest science award given by the United States. For more information, go to

About the New York Genome Center
The New York Genome Center (NYGC) is an independent, nonprofit at the forefront of transforming biomedical research and clinical care with the mission of saving lives. As a consortium of renowned academic, medical and industry leaders across the globe, NYGC focuses on translating genomic research into clinical solutions for serious disease. Our member organizations and partners are united in this unprecedented collaboration of technology, science, and medicine. We harness the power of innovation and discoveries to improve people’s lives — ethically, equitably, and urgently. Member institutions include: Albert Einstein College of Medicine, American Museum of Natural History, Cold Spring Harbor Laboratory, Columbia University College of Physicians and Surgeons, Cornell University/Weill Cornell Medical College, Hospital for Special Surgery, The Jackson Laboratory, Memorial Sloan Kettering Cancer Center, Icahn School of Medicine at Mount Sinai, NewYork-Presbyterian Hospital, The New York Stem Cell Foundation, New York University, North Shore-LIJ, The Rockefeller University, Roswell Park Cancer Institute, Stony Brook University and IBM. For more information, visit:

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