Advanced therapies: real opportunities for many diseases

On October 26th, the conference “Advanced therapies: clinical practice and quality in the production” took place in Milan.  The event, organized by Bioskills, was coordinated with the contribution of the Hospital San Gerardo, the Lombardy Region, the University of Milan Bicocca and the Tettamanti Foundation. The scientific board of the conference included Doctor Giuseppe Gaipa and professors Ettore Biagi and Andrea Biondi.

We asked Dr. Giuseppe Dastoli, R&D Director ” CIK -CAR Program ” Tettamanti Foundation Monza, to comment on some of the most significant passages of the meeting.

What impression did you get from the conference?

I think it offered an excellent opportunity to highlight the excellence of Italian research in the field of advanced cell therapy and of the high quality the academic facilities achieved in implementing the Good Manufacturing Practice (GMP) standards for the purpose of clinic experimentation of such products.

The regulatory aspect plays a decisive role for the future of cellular therapies. Do you think that the current legislation provides reasonable assurance of safety and effectiveness of these therapeutic tools?

The security levels required under Italian legislation for the GMP production of advanced therapies, are sure among the highest in Europe and internationally. As emerged during the conference , it could be useful to consider whether the required quality standards at an early stage of experimentation academic, should be maintained at the same levels as in the process of industrial production , or if , while ensuring high levels of safety , may be It adopted a more flexible so as to accelerate the development of innovative academic origin.

What future can you foresee for cancer treatment based on mesenchymal stromal cells?

Mesenchymal stem cells have different applications for their differentiation capacity of adipose, chondroblastic and osteoblastic type. Mesenchymal cells are also used for their immunomodulatory capacity. These characteristics open up new opportunities for a large number of clinical applications, which are beginning to take their first positive steps in clinical research.

What are in your opinion the options of cell therapy in thalassemia?

The data concerning the first treated patients are extremely attractive and, if confirmed, cell therapy could certainly be an important therapeutic option for patients with the most severe forms of the disease.

The cytokine-induced killer seems to envisage innovative solutions and are particularly effective in the treatment of patients with leukemia relapse after transplant. How do you assess the results of the first studies in this direction?

The adoptive immunotherapy is an important therapeutic weapon for the management of serious conditions, including the later stages, after other therapies have failed. In the last few years, the use of the CAR (Chimeric Antigen Receptor) has reached the clinical phase of testing. The CARs, after being inserted inside of T lymphocytes, both with viral gene and non-viral gene methodology, allow to strengthen the cytotoxic activity against specific cancer cells. In the case of relapsed lymphoblastic leukaemia after transplantation, the results reached so far are promising and have attracted the attention of many research groups worldwide and of a number of major pharmaceutical companies, interested in providing relevant investments on these products.

The transition of innovative therapies from academic research to the industrialization is a key factor to enlarge the audience of patients eligible for this new treatment. What is your perspective on this?

The intervention of the pharmaceutical industry may certainly cause a change of gear in the prospects of being able to spread the use of advanced therapies to a broad population. Some problems of production and logistics, that can complicate the diffusion of these treatments, are still to be overcome. Most likely a synergy between industry and academia should also be evaluated to find solutions in favour of the hospitals with GMP production systems of cell therapies. These centres could ease the relocation of a part the production, especially in the case of manipulation of the patient’s cells. It could be interesting to analyse these aspects to assess their feasibility.