President's Letter

Slowly but steadily gene and cell therapies have been advancing during the last three decades and now their value is unquestionable. Glybera, an AAV vector carrying lipoprotein lipase gene, has been approved for the treatment of the deficiency in this enzyme. The superiority of gene therapy compared to other treatments for congenital immunodeficiencies is evident and its application at world-wide level is getting ready. In other diseases such as retinopathies, beta-thalassemia, and hemophilia B, clinical efficacy is also evident. In cancer, where each tumor and patient is different, the genetic modification of T lymphocytes is revolutionizing leukemia treatment. At last the society begins to learn that these genetic treatments are changing the way genetic diseases are tackled and the debate has moved to the financial issues of its application. The main actor of this advance has been the vector. New vectors derived from lentivirus for in vitro and from AAV for in vivo applications, show enough safety and efficiency to convince clinicians and regulators that these therapies are the best option for the patient. Still, vectors and gene editing tools are evolving to be more precise, effective, and safe. The correction of genetic defects combined with the potential to induce stem cells from differentiated cells, offers new possibilities to regenerate corrected tissues and organs. This combination of gene and cell therapy is changing transplantation-based medicine. Participating in this medical revolution is an exciting adventure. Beyond papers and impact factors, the motivation of researchers in this field is to translate scientific knowledge into patient benefit. This intermediary role may come at the expense of the prestige of basic knowledge-finding, but it drives the advance of society and we are proud of it. Although Spanish gene and cell therapy is still short on budget and organization compared to other countries, numerous groups are trying to compensate such deficiencies with imagination. The slogan that applied research supports industrial development is trendy, but we have not been able to capitalize on this idea. Gene and Cell Therapy is by definition applied research. The goal is a product to treat a disease. If such a disease is an unmet need affecting a considerable number of patients, if the product is novel, scientifically sound, and simple enough to be scaled up, then investing on such a gene therapy project is a smart choice for public and private investors. Besides more investment, Spanish gene and cell therapy needs a greater involvement of clinical leaders with a pioneering spirit. We have an excellent public health system with fantastic reference hospitals with leaders in multiple pathologies. To contribute to the progress of gene and cell therapy we need to involve these clinical opinion leaders when such therapies are at the conceptual stage. Partnering with pioneering clinicians of reference for a disease ensures success sooner or later. Public funding and the contribution of these pioneers are crucial at the initial stage when vectors and therapeutic protocols are improved until results are attractive enough for industry. In Spain, pharmaceutical companies have begun to invest in gene and cell therapy, and several biotechnology companies startups have been created. Gene and cell therapy generates wealth and jobs for the excellent students trained in our scientific, medical and biotechnological universities.

From the Spanish Society of Gene and Cell Therapy we aim to support the development of these therapies at a national and international level. We offer information on research groups working on gene and cell therapy and we invite you to become a member of our Society  and benefit from free access to “Human Gene Therapy”, the journal of the European Society of Gene and Cell Therapy, and to participate in our web page and biannual meeting. Welcome to this exciting adventure.

 

Ramon Alemany

President of the SETGyC