Stem Cells: from basic research to clinical experimentation
DIRECTOR: Giulio Cossu

Aim of this program is the development and integration of research projects studying stem cell biology and their ability to restore tissue integrity in pre-clinical models of genetic and acquired diseases. Its final goal is to promote clinical translation of a number of these projects optimizing chance of efficacy and minimizing risks for patients.
This is an exciting period for research in stem cell biology and translational medicine in general: many research projects have been developed in pre-clinical models for the previous years and are now facing the challenge of entering clinical experimentation.
It has also become apparent that the whole process, starting with gene discovery and continuing with the understanding of the pathogenesis and the creation and use of animal (mainly mouse) models, is now much rapid than it was only ten years ago.
At the same time it is perceived by the community that clinical translation is a critical step and must be taken with all possible reliable predictions of efficacy and low risks for patients. We have learned in recent years that one serious adverse events (Hacein-Bey-Abina et al. 2008), even if unpredictable on the basis of thorough and rigorous pre-clinical work, may hamper the whole field for years. It is probably worth to stress that the International Society for Stem Cell Research (ISSCR) has recently published “Guidelines for clinical translation of stem cell research” (available at isscr.org). These Guidelines were drafted by an international task force (Hyun et al., 2008) “to help facilitate the responsible and timely development of clinically useful stem cell-based therapies and to help minimize the potential harms to research subjects and patients”.
Within this overall scenario, our Institution has a prominent international role, mainly due to pioneering work of congenital immune deficiencies (Aiuti et al. 2002), and many highly promising projects ready for or already entering clinical trials. Moreover there are a large number of basic or clinical research groups that are either directly involved in stem cell research, but at an earlier stage of development, or provide invaluable expertise in cell and molecular biology, in immunology and in imaging, just to name a few. These groups may be interested and involved in a large Institutional program like this one, by contributing their expertise and, at the same time, benefiting of the previous experience of those groups that are ahead towards clinical experimentation.

For this reason the program suffers of certain heterogeneity and will require a continuous effort by all participants to progressively focus and refine the objectives. At the same time it offers a great opportunity to put together many of the leading expertise of our Institution and create synergies to achieve faster and safer clinical translation of our research.

As usual in this area, the program is divided in three main workpackages, basic, pre-clinical and clinical, as well as in a number of milestones and deliverables that will have to be reached at the end of its three years duration. Moreover, because of its nature, a first overall evaluation of its progress will be carried out one year after its start.