Speech of Dr Gamez on the 30th Spanish Conference on Neuromuscular Diseases

27/10/2015 18:39

Dr. Josep Gámez, head of the Peripheral Nervous System group at Vall d’Hebron Institute of Research (VHIR) and coordinator of the Muscular and Rare Diseases Unit at Vall d’Hebron University Hospital, gave the keynote speech of the 30th Spanish Conference on Neuromuscular Diseases, hosted in Vigo the 23rd and 24th of October. Over 200 patients and families listened to their speech, entitled “From bench to bed: translational medicine applied to neuromuscular diseases”, in which he talked about the benefits of the translational medicine, and the leadership of Vall d’Hebron in terms of patient care and research on neuromuscular diseases in Spain.

Research in these diseases is led at VHIR by Dr. Gámez’s group; the Pediatrics Neurology group, headed by Dr. Alfons Macaya and Dr. Francina Munell; and the Neuromuscular and Mitochondrial Pathology group, led by Dr. Ramon Martí. The three groups work jointly in the research of neuromuscular diseases and are carrying out clinical trials based on gene therapy in diseases such as spinal muscular atrophy and the familial amyloidotic polyneuropathy (Andrade’s disease).

Dr. Gámez presented the case of Vall d’Hebron as a success of translational medicine. At the beginning of the conference, he said that despite the knowledge gained over the last years by basic researchers, the impact on the clinical practice has been minor. He added that the growth in R+D for the development of new molecules neither has been followed by an increase of the number of commercialized drugs.

It is in this context, where he reported that it is needed the translational research, whose aim is to enable the transition of basic research into clinical applications for the benefit of patients. This is the 4B medicine: “from Bench to Bed and Back, and Beyond”, based on the interconnection of clinical and basic researchers, other professionals such as experts in bioinformatics and biostatistics, and new agents such as biotech companies or patients associations.

“Vall d’Hebron has the perfect scenario and staff for the development of Translational Medicine”, reported Dr. Gámez. “We share the same location; we are leaders in Spain in clinical trials; and give multidisciplinary health care assistance to patients of all the ages”.

Dr. Gámez is the head of the Multidisciplinary Unit of Amyotrophic Lateral Sclerosis (ALS), which offers to patients all the specialties that they need. Thus, these patients, who usually have limited mobility and require complex medical processes, can visit all their doctors in a single visit to the hospital.

The congress was organized by the Spanish federation of neuromuscular diseases (ASEM), with the presence of the responsible of health in the Xunta de Galicia, Rocío Mosquera, the major of Vigo, Abel Caballero, and the president of the federation, Cristina Fuster, among other authorities from the Diputación de Pontevedra.