Spain leads research in advanced therapies with almost 120 clinical trials

One of the health priorities in Spain for the Congressional Reconstruction Commission and for the Shock Plan for Science is the development of advanced therapies, the cornerstone for curing diseases such as cancer. This area accounts for 62% of the clinical trials which Spain has managed to position itself as a leader and benchmark country, with almost 120 trials in progress, most of them in the public sector.

Advanced therapies represents a new generation of innovative medicines based on genes, cells or tissues. These disruptive and innovative therapies have great therapeutic potential by allowing the treatment of degenerative and genetic diseases, and even certain types of cancer, which may not be treated by other approaches. However, the vast majority of advanced therapies of Spanish origin are in very early stages and it has not been possible to make progress in the industrial development of drugs, reachable to patients.

In addition to clinical trials, Spain also leads the production of scientific knowledge in biotechnology, being the 9th world leader which growth has been nearly 30% in the last decade, reaching 1,085 publications per year. We also rank fifth in the number of scientific publications in the field of cell therapy with respect to GDP, ahead of countries such as South Korea, the United Kingdom and Italy.

One of the major milestones achieved in advanced therapies in Spain was the approval of Alofisel as the first cell therapy from a donor by European Union in 2018. This was the result of years of research by the CSIC and the Autonomous University and the launch of the spin-off Cellerix. Currently, the company leading manufacturing processes is Takeda and supplies the therapy from its plant in Tres Cantos (Madrid) to 19 European countries, consolidating its position as a world leader in the commercial manufacture of this type of medicine. "Since Alofisel was approved, only four advanced therapies have been approved in the EU. It is an area that is very slowly moving towards full development in Europe, but there are more and more therapeutic areas where they offer new hope for patients," says Maria del Pilar Redondo, head of Takeda's Tigenix.

At the forefront of many projects about immunotherapy strategies to achieve new targets against cancer is Dr. Ignacio Melero, researcher at the Department of Immunology of the Clinic Universidad de Navarra. "The framework is one of intense collaboration with industry, both with large pharmaceutical companies and biotech companies. We combine these projects with a significant number of clinical trials that we lead as sponsors or in which we participate. We hope that some of these agents or strategies will be a break-through in terms of efficacy for our patients," says Dr Melero.

VIVEbiotech is dedicated to the industrial production of lentiviral vectors for clinical trials of gene therapies aimed at oncohematological diseases curing. This organization is committed to the internationalization of its 34 projects amongst US, Europe, Asia and Oceania, in order to face the multiple challenges in this area of activity.

The challenges of advanced therapies

According to Gurutz Linazasoro, CEO of VIVEbiotech and coordinator of AseBio's advanced therapies working group, one of the priority challenges is regulation. "Therapies that move on the frontier of knowledge and the Agencies must regulate for the first time previously unknown aspects, such as the use of genetic, embryonic, viral material or new biomaterials. For this reason, they are almost always lagging. Harmonisation of all regulatory legislation worldwide would be desirable," the expert stresses.

However, Dr Ignacio Melero attributes the lack of competitive public funding as one of the "major obstacles to the death valley between discoveries and the patient". Therefore, lowering the costs of advanced therapies to reduce their price could be a solution.

Linazasoro thinks: "it is true that they are curative or highly effective therapies in pathologies with a very poor prognosis. However, in the medium term they are unaffordable for private finances and public health services. One way to reduce costs is through greater efficiency in production processes through digitalisation, automation, standardisation, and industrialisation, and through public-private collaboration.

Health authorities were supported by all industries with equipment and knowledge, which has undoubtedly been the great lesson of this pandemic. "Everyone has stepped up to the plate. Nobody thought that a vaccine would be achieved in 9 months, the most optimistic forecast was 18 months. The question is, why not do a similar exercise in times of health calm to combat diseases with high mortality rates?”, Linazasoro asks.

Despite of being leaders in scientific production and clinical trials in advanced therapies, Spain is not capable of turning this scientific knowledge into products and services with a high benefit. "We need to take advantage of this reality and generate a thriving network of start-ups that are well financed and well advised. And this is where public-private collaboration takes on special relevance and where AseBio can play a magnificent bridging role. Biospain is the showcase for Spanish biotechnology, both internally and externally. It is the ideal time and instrument to emphasise the three great challenges facing Spanish biotechnology: growth, becoming more competitive and digitalization," concludes Linazasoro.

Biospain, a benchmark event in the Spanish biotechnology sector and on an international scale, will be held from September 27 to October 1 in hybrid format, both virtually and face-to-face, at the Palacio de Congresos y Auditorio de Navarra-Baluarte in Pamplona-Iruña in collaboration with the Government of Navarra and Sodena, Navarra's development company.