The Food & Drug Administration (FDA) has given the green light to start a clinical trial of gene therapy for a rare immune deficiency, the Wiskott-Aldrich syndrome (WAS), the United States. Généthon has made the pre-clinical trials and provide lots of drug delivery. After it out in Paris and London, this trial will now start Boston. This is one of the first international clinical trials for the treatment a rare disease by gene therapy.

Earlier this year (Cf. CP of January 5, 2011) Généthon, the laboratory of Biotherapy, non- organization funded by the AFM thanks to donations from the Telethon and the Children's Hospital Boston announced their partnership to conduct a clinical trial of gene therapy for the Wiskott-Aldrich Syndrome (WAS), a severe immune deficiency that causes death before adulthood. Promoter of the European trial conducted in parallel to the Great Ormond Street Hospital in London and the Hospital Necker-Enfants Malades in Paris (See the CP February 24, 2010) Généthon also provide lots of drugs used vectors for this trial in the United States.

In total, the test will include five patients, five per site, which will be treated in 2013-2014. Sites in London and Paris have already begun to address patients. Performed simultaneously in London, Paris and Boston, this protocol represents a unique collaboration to accelerate the evaluation of new advanced therapies for rare diseases.

The trial in Boston is funded by the National Heart, Lung and Blood - the National Heart, Lung and Blood Institute (NHLBI). The main Investigators at Children's Hospital Boston is the Dr. Sung-Yun Pai (blood- oncologist), Luigi Notarangelo, programme manager for research and diagnostic molecular primary immunodeficiencies, and Dr David Williams, head of Service Haematology and Oncology and director of translational research. The treatment of the cells of patients will be conducted in the premises of the centre Connell O'Reilly at the Dana-Farber Cancer Institute, in collaboration with the Centre for Therapy Cell of the Harvard Medical School. The WAS is a rare immune deficiency resulting in heavy bleeding due to low platelets and numerous serious infections. Most patients also suffer from mild to severe eczema and have a higher risk of developing autoimmune diseases and cancer as lymphoma. The clinical trial is the culmination of a program Research initiated in 2002 by the team of Anne Galy Genethon laboratory UMR951/Généthon, University of Evry Val d'Essonne, EPHE).

The researcher developed an ex vivo using a lentiviral vector derived from HIV to transfer genes into hematopoietic stem cells CD34 + autologous patient suffering from Wiskott-Aldrich syndrome. “This approval represents a new milestone for the project initiated in WAS 2002 Genethon by Anne Galy and his team. It is also a recognition International quality of work of Genethon. Without donations from the Telethon, this test International gene therapy could not exist,” says Laurence Tiennot- Herment, president of the AFM and Généthon. “We are very pleased to have received FDA approval for the extension of WAS clinical trial in the United States. This demonstrates, again, the ability to Généthon to bring its research to the clinic in collaboration with the best clinical teams around the world for the benefit of patients. This authorization highlights also our strong expertise in the areas of translational research, the biomanufacturing, preparation and implementation of clinical trials” explains Frédéric Revah, managing director of Genethon.

“At Children's Hospital in Boston, we are determined to use therapies cellular and molecular advanced to treat devastating diseases in children. The test on the WAS is particularly noteworthy because it represents an ongoing effort to transatlantic collaboration of scientific and clinical research in gene therapy and a huge multidisciplinary team at Children's Hospital” says Dr David A Williams, Principal investigator of the study.

Created by the AFM and funded almost exclusively by donations from the Telethon, Généthon mission to make available to patients innovative treatments in gene therapy.

The AFM is an association of patients and parents of patients. Through donations from the Telethon (90 million in 2010), it has become a major player in biomedical research for rare diseases in France and worldwide.

The Department of Paediatric Oncology and Haematology at Children's Hospital Boston, internationally renowned treats young children and adolescents against cancer and blood diseases. The haematology department treats children with minor ailments of blood.