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Highlights
02/02/2004
At Ospedale Maggiore of Milan, “Centro Dino Ferrari”,
the first transplantation of human stem cells in DuchenneMuscolar
Dystrophy
Disease incidence is one out of 3500 male newborn.
Milan, 2004 February 2nd – On 2003 November 11th the “Istituto Superiore
di Sanità” (an Italian National Regulatory Health Agency) and the
Ethic Committee of I.R.C.C.S. Ospedale Maggiore - Policlinico in Milan authorised
the group directed by Prof. Nereo Bresolin, Chief of the Neurological Institute
of Ospedale Maggiore - Policlinico and of “Centro Dino Ferrari” to
perform the first human stem cell transplantation in few children affected by
Duchenne Muscular Dystrophy (DMD). This group of patients will include an affected
child son of a medical doctor who personally participated to this fundamental
experiment.
The aim of this project was presented in the press conference given today at
Ospedale Maggiore - Policlinico in Milan. The speakers, introduced by Dott. Anna
Parravicini, chief of the Communication Office of Ospedale Maggiore - Policlinico
in Milan, were: Prof. Girolamo Sirchia, Minister of Health; Prof. Guido Coggi,
Dean of the Faculty of Medicine and Surgery, University of Milan; Dr. Piero Ferrari,
Honorary President of Associazione Amici del “centro Dino Ferrari”;
Prof. Ferruccio Bonino, Scientific Director of Ospedale Maggiore – Policlinico;
Prof. Nereo Bresolin, Director of the Neurology Unit, Ospedale Maggiore – Policlinico
and Scientific Director of “Centro Dino Ferrari”; Dr. Yvan Torrente,
coordinator of the Stem Cell Laboratory of the Neurological Institute, University
of Milan; Rep. Dr.. Domenico Galbiati, President of IRCCS Eugenio Medea and Prof.
J. Andoni Urtizberea, Delegué a la Miologie, representing the Association
Française contre les Myopathies – French Telethon, which financed
this project together with the Italian Health Minister and the Associazione Amici
del “Centro Dino Ferrari”.
The initial step of this project aims to assess stem cells safety. Stem cells
from a dystrophic muscle will be directly injected in a short hand muscle of
the same patient and eventual inflammation and/or side effects will be evaluated.
This step is expected to last one year to evaluate growth anomalies eventually
induced by the treatment needed to test muscle strength.
Therefore the experimental protocol will be a safety trial. However it will be
a first step and will open the way to further studies aimed to the characterise
the isolation of stem cells from dystrophic muscles, define their proliferative
behaviour and transduce them with vectors carrying the lacking gene of dystrophin.
A source of healthy muscle proliferating cells will be thus available.
This project has become a reality thanks to “Centro Dino Ferrari” which
has been involved since twenty-five years in developing research on muscular
dystrophies by scientific programs, important publications and a widespread network
in Italy and abroad.
Duchenne Muscular Dystrophy (DMD) is a severe muscle disease characterised by
a inexorable and devastating course with dramatic social consequences. Disease
incidence is one affected out of 3500 male newborn. The progressive muscle weakness
causes children to loose their movement capability. They become unable to walk
and are wheelchair-bound by the age of ten. The vast majority of them die in
late adolescence because of respiratory and cardiac failure.
This project resulted from the collaboration between “Centro Dino Ferrari”,
particularly Dott. Yvan Torrente, coordinator of the Stem Cell Laboratory, Prof.
Maurizio Moggio, chief of the Morphology Lab “Centro Dino Ferrari”,
co workers of Prof. Nereo Bresolin, and several Centres in order to reach a common
goal. Among them we would like to mention: the team of Dott. Paolo Rebulla, chief
of the Cell Factory Lab of IRCCS Ospedale Maggiore – Policlinico in Milan
which guarantees a safety environment where cells have to be handled; I.R.C.C.S.
Eugenio Medea, Associazione La Nostra Famiglia, where children from the whole
Italy undergo rehabilitation; Prof. Giulio Cossu, chief of the Stem Cell Research
Institute (SCRI) at Ospedale San Raffaele; Prof. Roberto Bottinelli from IRCCS
University of Pavia, who will verify eventual changes in muscle strength after
transplantation.
“
Actually – says Prof. Nereo Bresolin, Director of “Centro Dino Ferrari” – today
evidences come from a many years’ work, often powerless toward the inexorable
disease outcome. The present results can not address whether stem cells therapy
of muscular dystrophy is a dream or a true possibility, but new therapeutic strategies
will be certainly foreseen and lead us to definitive answers”.
“Enzo Ferrari, my beloved father – Piero Ferrari remembered – strongly
supported this research team, grown around Prof. Guglielmo Scarlato. Substained
by Associazione Amici “Centro Dino Ferrari” this group will be more
and more able to relieve suffering related to these severe pathologies”.
Several centres are dedicated to support and rehabilitate children affected by
muscular dystrophy. Only few of them in Italy can offer families a qualified
attempt of therapeutics for this hopeless disease.
“
Centro Dino Ferrari” was founded in 1984 thanks to Ing. Enzo Ferrari and
was dedicated to the loving memory of his son Dino affected by muscular dystrophy.
Since then, “Centro Dino Ferrari” has contributed to scientific research
by mean of important programs and publications. Identification of new strategies
towards the repair of muscle injury is one of the keys in gene therapy. Pharmacological
approaches have been ineffective so far in reducing damages and promoting muscle
regeneration. By advanced technical tools and methods, we can isolate stem cells
deriving from different kind of tissues, muscle included.
A method to isolate stem cells from dystrophic muscle in order to use them in
the donor himself was developed by Prof. Nereo Bresolin’s team. The rationale
for using stem cells obtained by dystrophic patients derives from the observation
that other elements than the lack of dystrophin are involved in the opathogenesis
of Duchenne dystrophy.
Today we are making the first step towards understanding stem cells efficacy
in treating muscular diseases. |
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