On the challenges in developing competitive technologies

Jorge Vega, José Buxadó
The search for new molecular entities (NME) to satisfy unmet medical needs is a very expensive task with a very low rate of success. The discovery of a useful molecule is, by nature, an event that depends on the complexity of the disease under investigation, research planning and management, time and other less predictable dependent variables. Drug development, from the laboratory to regulatory approval for commercialization, usually takes more than 7 years and costs hundreds of millions of
more » ... of millions of dollars. The low productivity and high risk of research and development (R&D) in the biotechnology industry suggests that more attention must be paid to innovation on new pharmaceutical formulations, more efficient and economic technologies, and new medical applications for existing products with a well-known efficacy and generally recognized as safe. Results of these innovative efforts will be found in the Abstracts, following this paper, which were presented at the 15th edition of the CIGB's Scientific Workshop, held in June 2004. These Abstracts describe the kinetics and scaling up of plantibody production and the consistency of several manufacturing processes. They also show the downstream process for manufacturing a DNA molecule, such as a potentially active pharmaceutical ingredient, for gene therapy and recent innovations in the upstream process by using microbial and mammalian cells. Recent advances in the development of new technologies and medical applications at the CIGB have been recognized by the mainstream journals. Prestigious scientists from the Leices-ter Royal Infirmary (United Kingdom), the Imperial College Faculty of Medicine (United Kingdom) and the CIGB conducted a well-designed clinical trial on mild-to-moderate left-sided ul-cerative colitis with the Epidermal Growth Factor (EGF) manufactured by the Cuban institution. Remission was achieved in more than 80% of the patients and no side effects were reported by any of the patients taking EGF [1]. CIGB's scientists and engineers published the expression and scaling-up of a monoclonal antibody (MAb) by using tobacco plants as bioreactors. A praiseworthy work involving more than 30 highly skilled professionals was performed to obtain large amounts of the antibody. Among other advantages, lower production costs, a virtually unlimited scale-up potential, and no known susceptibility to human or animal diseases make the plant system a very attractive alternative [2]. The large-scale synthesis, at the CIGB production plant, the pharmaceutical development and clinical trials of the first synthetic vaccine have been published by Science Magazine [3]. These results were described as a sweet victory for Cuban science by two editorial comments of Nature and Science [4, 5]. Penetrating the most demanding markets with new products and overcoming regulatory barriers by developing safer and more economic technologies are major challenges for biotechnologists participating in this Scientific Workshop. Producing biomolecules in transgenic plants represents an attractive alternative for substituting microorganism and mammalian cells in the biotechnology industry, while CIGB's production staff still has the challenge of maintaining statistically consistent manufacturing processes in compliance with current Good Manufacturing Practices. Improving the recovery and lowering the costs of manufacturing processes were also focused in round tables, presentations and posters in this workshop. The discussions were basically on technologies of products that are currently in the market, but that will probably have an impact in the development of NME that could be eventually discovered by the CIGB research staff.