Généthon, the not-for-profit biotherapy laboratory operated by the French
Muscular Dystrophy Association (AFM) with funding from the country's annual
Telethon*, has just obtained approval from the French and British health
authorities for a Phase I/II clinical trial of gene therapy for a rare
immunodeficiency, Wiskott-Aldrich syndrome (WAS). The Généthon-sponsored
trial will be performed both in France (led by Professors Alain Fischer (1) and Marina
Cavazzana-Calvo (2) at Necker Children's Hospital, Paris) and in the UK (led by Professor
Adrian Thrasher at London's Great Ormond Street Hospital). With just a few days to
go to Rare Disease Day 2010* (February 28th, with a focus on "Bridging Patients
and Researchers”), initiation of this trial marks a new step towards curing rare
diseases with innovative biotherapies.
Wiskott Aldrich syndrome (WAS) is a severe, X-linked immunodeficiency which results in
hemorrhage, recurrent infection and eczema. It is almost always fatal in affected
children. Although a bone marrow transplant can reestablish the patient's immune
system, this operation requires a compatible donor and carries a non-negligible risk of
failure. Gene therapy (which has already enabled the treatment of other hereditary
immune deficits) therefore represents a major source of hope for patients waiting for
therapy.
The forthcoming clinical trial is the end result of research initiated in 2002 by Anne Galy's
group (UMR951) (3) in the Généthon laboratory. The lab's researchers have developed an
ex vivo approach that uses an HIV-derived lentiviral vector to transfer genes into
autologous CD34+ hematopoietic stem cells from the patient. Généthon has developed,
produced and quality-controlled the trial's batches of gene-based medicines under GMP
conditions.
The trial also demonstrates the extent of the AFM lab's know-how, ranging from
preclinical research and the GMP production of gene-based drugs through to trial logistics
and regulatory approval. The preparatory work took eight years and involved a €10
million investment.
"This trial is enabling the Généthon laboratory (funded almost exclusively by donations
from France's annual Telethon) to demonstrates its world-class expertise in the
production of gene-based medicines and the preparation and implementation of clinical
trials. It confirms Généthon's ability to become a major international player in
demonstrating the efficacy of gene therapy for rare diseases" emphasized AFM and
Généthon President Laurence Tiennot-Herment.
About Généthon
Généthon is a not-for-profit research centre that was created by the French Muscular
Dystrophy Association (AFM) and is now funded almost exclusively by donations from
France's annual Telethon. Its goal is to deliver gene therapies to patients with rare
diseases in general and neuromuscular diseases in particular. With over 200 scientists,
physicians, engineers and regulatory affairs specialists, Généthon is one of the world's
leading centers for preclinical and clinical research and development in the field of gene
therapy. Généthon also has a biomanufacturing platform for clinical-grade vectors and is
involved in building the world's largest facility for pre-industrial pilot production. A 5000
m2 facility will open in Evry in early 2011. www.genethon.fr
About the AFM
The French Muscular Dystrophy Association (AFM) federates patients with neuromuscular
diseases (genetic diseases that kill muscle after muscle) and their parents. Thanks in
great part to donations from France's annual Telethon (€90 million in 2009), the AFM has
become a major player in biomedical research into rare diseases in France and
worldwide. It is currently funding 34 clinical trials on 30 different genetic diseases
affecting the eyes, the blood, the brain, the immune system, the muscle... Thanks to its
Généthon research lab, the AFM stands out through its unique ability to produce and trial
its own gene-based medicines.
