iBET

IBET – ITQB SEMINAR, ITQB Room: 2.13 July 18th – 11H00 – 12H00

15.07.2013

Prof. Marc Peschanski

Scientific Director

"Harnessing
pluripotent stem cells for pharmacology"

Affiliation: I-STEM (INSERM/UEVE 861,
AFM), Evry - FRANCE

Pluripotent stem cells, because they can be amplified at will and
differentiated into any human cell phenotype, are potentially perfect platform
to evaluate in vitro efficacy and toxicity of chemical compounds that may be
used to treat diseases. I will illustrate those capacities using examples taken
from ongoing programs at I-Stem that concern myotonic dystrophy type I,
progeria, Huntington disease and statin-induced myopathic toxicity.

Short CV

Scientific
Director I-STEM (INSERM/UEVE 861, AFM) Marc Peschanski, founder and scientific
director of I-Stem is a medical doctor in Neurosciences. Entered at INSERM in
1982, he first worked on the neurophysiology and anatomy of pain in Paris and
San Francisco. From 1985 his work was oriented towards the study of
neuroplasticity and transplantation of foetal neurons, with which his team
conducted the first clinical studies in France, starting in 1991 in patients
with Parkinson’s disease, and the first world trial in Huntington’s patients
from 1996, followed by a European study of Phase II in a hundred patients,
currently in 4 countries.

Co-founder
of the Clinical Investigation Centre at the Henri-Mondor Hospital and to its
associated Biotherapy branch, he was also a founder of the European
neurotransplantation Network (NECTAR) and its first chairman in 1991-92. He has
coordinated several European research networks for gene therapy and cell
therapy of neurodegenerative diseases, as well in most recent years pluripotent
stem cells. He currently coordinates the Scr&Tox network of FP7.

The
Institute for Stem cell Therapy and Exploration of Monogenic diseases (I-Stem),
has been created the first of January 2005. It is a centre for research and
development, dedicated to the development of treatments based on the potential
offered by Stem cells and applicable to rare diseases of genetic origin.

Defined
by three key words, "therapeutics”, "monogenic diseases” and "stem cells”, the
activity of I-Stem extends from basic biological research and pathological
mechanisms up to the transfer of new therapies to clinical research.

The
objective of I-Stem is the development of treatments intended for monogenic
diseases, founded on the strong potential of stem cells for substitutive and
regenerative therapies. A second original objective of I-Stem is the
development of cell models representative of pathologies on the basis of human
embryonic stem cell lines each carrying a mutant gene associated with a given
disease. These should help elucidate mechanisms of pathogenesis, and
consequently, reveal possible therapeutic targets. These models could also be
used as a basis for screening compounds libraries in order to discover new
potential drugs.

Host: Paula
Alves

ITQB Room: 2.13

July 18th – 11H00 – 12H00