Marina Cavazzana, MD, PhD, Paris Descartes University, France and Adrian J. Thrasher, MD, PhD, University College London Institute of Child Health, UK, have been honored with the Pioneer Award for basic and clinical gene therapy for immunodeficiency disorders. Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers, is commemorating its 25th anniversary by bestowing this honor on the leading 12 Pioneers in the field of cell and gene therapy selected by a blue ribbon panel and publishing a Pioneer Perspective by the award recipients
Dr. Cavazzana has been at the forefront of advances in treating life-threatening inherited diseases of the immune system with gene therapy, using a patient's own modified stem cells. She describes the translation of this work to the clinic and its ongoing advances and novel applications in the article "Hematopoetic Stem Cell Gene Therapy: Progress on the Clinical Front." The article by Dr. Cavazzana is available free on the Human Gene Therapy website at http://online.liebertpub.com/doi/full/10.1089/hum.2014.2504.
A pioneer of gene therapy in the UK, Dr. Thrasher has been at the leading edge of basic science research on the function of therapeutic genes for inherited disorders and the development of viral vectors to deliver them to affected patients. He has collaborated on gene therapy clinical trials targeting immunodeficiency disorders with groups in Europe and the USA.
"Cell therapy and gene therapy are advancing together to improve patient care," says Dr. Cavazzana. "We can expect to be able to rebuild a new immune system not only in primary immunodeficiencies but also in severe acquired clinical conditions (such as those in HIV-1-infected patients)."
"I've seen some very exciting times in the field, from the first evidence that biochemical defects can be corrected in vitro, to some remarkable clinical successes in patients with devastating diseases. I look forward with huge enthusiasm to the exciting developments on the horizon, which are likely to impact on more patients with an even wider range of disorders," says Dr. Thrasher.
"These pioneers contributed to the first real clinical successes of gene therapy through their work in inherited immune deficiency disorders," says James M. Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia.