The French Institute I-Stem Realizes First Innovative Screens Using Stem Cells to Identify Drugs for Myotonic Dystrophy, DM1 Disease

EVRY, France - Four research teams of I-STEM[*] have joined forces in a collaborative
project that has just achieved a first pilot therapy-oriented screen of
compounds and RNA interference aiming at reversing the altered phenotypes
observed in human embryonic stem cells carrying the mutant gene for myotonic
dystrophy type1. This assay inaugurates a series of R&D planned in 2009.

Human embryonic stem (hES) cells lines carrying the mutant
gene responsible for diseases may replicate associated molecular defects
associated and be used, therefore, to analyse pathological mechanisms and
search for treatments. I-STEM teams have shown that hES cell lines carrying
the mutant gene responsible for myotonic dystrophy type1 (DM1) -the most
frequent myopathy in adult- present known cellular and molecular
abnormalities. hES capacity of self-renewal and pluripotency provides an
unlimited and highly versatile cell resource, relevant for large-scale
analyses. In order to exploit fully these potentials of hES cell lines within
the framework of its exploration of therapeutics for monogenic diseases,
I-STEM has set up a screening department through a close partnership with the
companies Velocity11, Discngine and Prestwick Chemical. I-STEM has installed
at its site, in Evry-Genopole, a powerful automation platform using the
innovative Velocity11 BioCel1800(R) technology, coupled to a specific data
management system designed by Discngine. The Conseil Régional d’Ile-de-France
and the Association Française contre les Myopathies (thanks to the French
Telethon donations) co-funded this platform[**]. The investments to build
this facility assays have been developed in order to screen the “FDA
approved” Prestwick Chemical library and a subset of the in house designed
siRNA (small interferent RNA) library.

Using this screening platform, the I-STEM teams have looked
for compounds and siRNA that would provoke the disruption of abnormal
aggregation seen in the nucleus of human embryonic stem cells carrying the
DM1 mutation. Several of the 1120 compounds and 50 siRNA assayed were
identified as candidates.

I-STEM intends to perform five to ten similar screening
campaigns per year on other genetic diseases, using its library of human stem
cell lines carrying genetic mutations[***].

About I-STEM

The Institute for Stem Cells in the Treatment and Study of
Monogenic Diseases- is a laboratory which has set out to explore the
therapeutic potential of stem cells in the treatment of rare genetic
diseases. Headed by Marc Peschanski (an INSERM Research Director), I-STEM was
in early 2005, the first lab in France to be allowed to work on (imported)
human embryonic stem cell lines. Then, in June 2006, it was authorized by the
French Agency for Biomedicine to set up a library of mutated cell lines that
can serve as models in the study of monogenic diseases. For more information:
www.istem.eu

About Velocity11

Visit www.velocity11.com

About Prestwick Chemical

Visit: www.prestwickchemical.com

About Discngine S.A.

Visit: www.discngine.com

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[*] High Throughput Screening (Marc Lechuga), Neuromuscular
diseases (Dr Geneviève Piétu), Functional Genomics (Dr Sandrine
Baghdoyan), Motoneuronal diseases (Dr Cécile Martinat).

[**] Other main funders of I-STEM are INSERM, Genopole® and the
Conseil Général de l’Essonne.

[***] Human ES cell lines are obtained, in particular, through
collaborations with Pr Stéphane Viville (Centre DPI, Strasbourg)
and Pr Karen Sermon (AZ-VUB, Brussels).

Source: AFM-Association Francaise contre les Myopathies

Contact: AFM Press department/Delphine Carvalho, Stéphanie Bardon, Géraldine Broudin - Tel : +33-1-69-47-28-28 - presse at afm.genethon.fr . About Velocity11 contact: Dr Bill Bradbury, Primetek Solutions (Kingston, UK), tel. +44-208-546-0869, email: info at primetek-solutions.com. About Prestwick Chemical contact: Dr. Marie-Louise Jung, tel. +33-6-81-34-17-84, marielouise.jung at prestwickchemical.fr. About Discngine S.A. contact: Jean-Philippe Laval, tel. +33-6-14-12-54-66, jean.philippe.laval at discngine.com.