GENE THERAPY R&D CENTERS FUNDED WITH $18 MIL

GENE THERAPY R&D CENTERS FUNDED WITH $18 MIL over the next five years are being backed at the University of California-San Francisco, the University of Pennsylvania and other academic sites. Funding for "select" studies at gene therapy "core" centers at UCSF, U. Penn and elsewhere will be provided by the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) and the Cystic Fibrosis Foundation. The gene therapy core center at UCSF, directed by Division of Molecular Medicine and Diagnostics chief Y. W. Kan, will "make and apply new discoveries to treat cystic fibrosis, sickle cell anemia and other inherited genetic disorders by correcting genetic defects through gene therapy," UCSF said Oct. 12. NIDDK funding for the center will be $2.25 mil. over a five-year period. The UCSF center will be separated into four clinical core facilities focusing on: development of transgenic animals; study of the behavior and properties of cells; analysis of genes; and complications arising from the use of modified viruses. Following the development of new treatments they will be tested in experimental clinical trials at UCSF's General Clinical Research Centers, Pediatric Clinical Research Center and Cystic Fibrosis Center. U. Penn announced Oct. 21 that James Wilson, MD/PhD, will direct similar efforts at the university, which also received an NIDDK five-year $1.25 mil. grant. Wilson, formerly chief of Division of Molecular Medicine & Genetics at the University of Michigan Medical Center and reknowned geneticist and gene therapy researcher, led the research team which employed gene replacement therapy to cure human cystic fibrosis cells in a laboratory culture which is now used to treat patients with the disease. The university also expects to receive funding from the Cystic Fibrosis Foundation. A third gene therapy core center that NIDDK plans to fund will be led by Dusty Miller, University of Washington-Seattle. Although the award announcement has not been made pending finalization of the NIH budget, NIDDK ultimately expects to appropriate $2.5 mil. each year for all three centers. The Cystic Fibrosis Foundation will supplement NIDDK's support by funding pilot and feasibility studies at the three centers. In addition, the foundation will support studies at six gene therapy centers announced by the National Heart, Lung & Blood Institute Oct. 14. In total, CFF will provide roughly $10 mil. over a five- year period to conduct 40 studies at the nine centers. NHLBI said the six centers will "emphasize the basic, preclinical and clinical research in cystic fibrosis, but will also explore investigations applicable to other heart, lung, and blood diseases, including hypercholesterolemia, atherosclerosis and hemophilia." The centers will receive $4 mil. annually over five years. Principal investigators at the centers include: Michael Welsh, University of Iowa; Richard Boucher, University of North Carolina in Chapel Hill; Arthur Beaudet, Baylor College of Medicine; Jeffrey Whitsett, Children's Hospital Medical Center in Cincinnati; William Guggino, Johns Hopkins University; and Ronald Crystal, Cornell University Medical College in New York City.