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Gene Therapy Briefs

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Apr 1, 2012 (Vol. 32, No. 7)

Gene Therapy Briefs

    • European Medicines Agency (EMA) published a reflection paper of regulatory considerations for specific gene therapy medicinal products (GTMPs) whose characteristics have been changed at various stages during clinical development, based on scientific discussions within EMA committees/working parties. Among areas discussed by the nine-page paper are changes of splice donor/acceptor sequences, E4 substitutions in chimeric nonhuman adenoviral vectors, changes of selection marker in plasmid vector, promoter exchanges in genetically modified encapsulated cells, AVV vector serotype changes, and RV to RV-SIN in monogenic inherited diseases. While already-established efficacy and/or safety profiles of GTMPs and their potential risk for patients should be evaluated, “the developers are strongly advised to seek product-specific scientific advice from the regulatory authorities,” EMA concluded.
    • A California clinic has become the first in the state licensed to carry out a controversial chronic pain treatment that has sparked a two-year-old FDA lawsuit. Health Link Medical Center in Oceanside, CA, is approved for the Regenexx stem cell treatment developed by Regenerative Sciences. The company is being sued by FDA over use of Regenexx for chronic pain. FDA contends that stem cell treatments constitute medication, which falls under its jurisdiction, and that Regenexx safety has not been sufficiently tested, while inspections found facilities offering the treatment do not meet its standards. Regenerative Sciences counters that because stem cells are produced by the body, they are not drugs, and thus do not need FDA approval.
    • StemCells will initiate a Phase I/II clinical trial of its HuCNS-SC® product candidate (purified human neural stem cells) in dry age-related macular degeneration (AMD) following authorization from FDA. The trial will evaluate safety and preliminary efficacy of HuCNS-SC cells for dry AMD. The trial will be an open-label dose-escalation study, expected to enroll a total of 16 patients. HuCNS-SC cells will be administered by a single injection into the space beneath the retina. Patients’ vision will be evaluated using conventional methods of ophthalmological assessment at predetermined intervals over a one-year period. Patients will then be followed for an additional four years in a separate observational study.
    • Cedars-Sinai Heart Institute researchers published clinical trial results in The Lancet showing that treating heart attack patients with an infusion of their own heart-derived cells helps damaged hearts re-grow healthy muscle. One year after receiving the stem cell treatment, scar size was reduced from 24% to 12% of the heart in the 17 patients treated with cells. Eight patients in the control group, who did not receive stem cells, saw no reduction in their heart attack scars. The clinical trial, named Caduceus, was part of a Phase I investigative study of 25 patients (average age=53), treated at Cedars-Sinai Heart Institute and at The Johns Hopkins Hospital.
    • Three adults previously treated in one eye with gene therapy for Leber congenital amaurosis (LCA) received the same treatment in their other eye, resulting in further vision improvement. All three became better able to see in dim light, while two were able to navigate obstacles in low-light situations, with no adverse effects. Scientists from The Children’s Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania led the study, published in Science Translational Medicine. The three adults were among 12 patients, four of them children, with LCA who underwent a clinical trial of the gene therapy reported in 2009. Six patients improved enough to no longer be classified as legally blind.
  • New This Year: Human Gene Therapy Methods

    The field of gene therapy is rapidly evolving and holds great promise for its applications in treating human diseases. Human Gene Therapy, the premier journal in the field, launched 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.

    www.liebertpub.com/hum



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