November 1, 2011 (Vol. 31, No. 19)

  • Genevolve Vision Diagnostics developed a noninvasive molecular diagnostic for all major forms of congenital color blindness, for use by ophthalmologists and optometrists. The test, marketed under the Eyedox™ brand, involves using a noninvasive buccal epithelial cell collector to pick up skin cells sloughed daily from the lining of the mouth.The DNA goes to a CLIA-certified laboratory where the color genes are identified and classified for inherited color vision deficiency, and a treatment plan and family history are developed. While gene therapy has been shown to cure some forms of congenital blindness, better diagnostics will be critical for identifying patients for current and future gene therapies.
  • The U.K. CF Gene Therapy Consortium, funded by the Cystic Fibrosis Trust, seeks to raise £6 million for a gene therapy trial. As of October 6, supporters raised £700,000 toward funding the multidose clinical trial, which calls for more than 100 people with cystic fibrosis in Edinburgh and London to be given either a healthy gene, the gene transfer agent, and the promoter or a placebo, once a month for a year. If patient lung function shows improvement, the consortium has said it will pursue a pharmaceutical partner to take the product into a Phase III clinical trial starting in 2015.
  • University of Pennsylvania researchers reported sustained remissions of up to a year among a small group of advanced chronic lymphocytic leukemia patients treated with genetically engineered versions of their own T cells.Carl June, M.D., led a team from the Abramson Cancer Center and Perelman School of Medicine in removing patients’ cells, modifying them in UPenn’s vaccine production facility, then infusing the new cells back into patients’ bodies after chemotherapy.The findings, published simultaneously in the New England Journal of Medicine and Science Translational Medicine, are the first demonstration of gene transfer therapy to create “serial killer” T cells aimed at cancerous tumors, a possible roadmap for treating other cancers.
  • U.S. and British researchers identified a highly undifferentiated stem-cell-like T cell within a patient’s immune system that can continually refresh itself for long periods of time, possibly permanently.Writing in Nature Medicine, the researchers—from NCI, NIAID, NIH, UPenn, and Cardiff University School of Medicine—discussed how they isolated a tiny fraction of highly undifferentiated T cells showing the first changes in gene expression that occur when T cells “remember” antigens they have seen previously, and prepare to react quickly. NCI’s Nicholas P. Restifo, M.D., and his team hope to regenerate younger T cells from older ones by manipulating certain genes.
  • An FDA advisory panel identified three challenges to development of cellular and gene therapies for retinal disorders in adult and pediatric populations. FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee pinpointed the challenges as: 1)Selection of efficacy endpoints for clinical trials, especially for trials intended to study rare retinal disorders and disorders in very young children; 2)Treatment of the contralateral eye and repeat administration of the product; and 3)Identifying methods to confirm accurate delivery of the intended dose into the target site, in both preclinical and clinical studies.No cell or gene therapy products are licensed for ophthalmic indications in the U.S., but several such products are in development.
  • Christopher Reid and his family are celebrating 10 years since he became the first child in England to receive gene replacement therapy for severe combined immuno-deficiency (SCID), a genetic condition that meant he had no natural immune system and was forced to live in a protective “bubble.”He was diagnosed with SCID at eight months of age, and given three months to live. But in December 2001, Christopher underwent an operation in which an artificially created gene was mixed with his bone marrow, and injected into his body. Gene therapy is now considered a cure for SCID, as reported in Science Translational Medicine in August.

Launching in 2012: Human Gene Therapy Methods

The field of gene therapy is evolving and and holds great promise for its applications in treating human disease. Human Gene Therapy, the premier in the field, is launching Human Gene Therapy Methods to complement the flagship publication. HGT Methods answers the growing need for a central forum for nurturing, promoting, and advancing new technologies and methods that will ultimately pave the way for product development. The Editor-in-Chief is James M. Wilson, M.D., Ph.D.

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