update from Dr. Ceradini- Parent Project Italy

I would like to update you on the stem cells progress for DMD.
As you know Giulio Cossu is working hard on it and we have good news since the first trial, conducted at the Scientific Institute San Raffaele in Milan and leaded by Giulio, started on march.

This study was funded by Parent Project onlus together with other funding agencies and represents the first attempt of an allograft transplantation of stem cells (mesoangioblasts) from a donor immunocompatibile (a brother or sister) in a DMD patient. This is a phase I-II trial aimed at establishing the safety and feasibility.

A first step of the trial, completed at the end of 2010, was based on a series of detailed measurements of strength and muscle function and the analysis of the clinical course of each patient in a period of 18 months. 28 DMD patients have participated in this first step and the recruitment was made through the Italian Registry of Patients DMD/BMD.
Of these 28 patients, 3 children (aged 6-9 years) have been selected for the heterologous transplantation of mesoangioblasts. The stem cells, obtained by muscle biopsy from the brother or sister, are injected into the blood of DMD patients by four different doses. To date, all 3 children have already received the first dose, at the moment the boys are doing well and do not show side effects. Obviously we have to wait to see if the results are those in which we hope.

In addition to the clinical trial Giulio Cossu, in collaboration with a Japanese group of Tottori University, is working on an innovative and ambitious project based on an human artificial chromosome. (HAC). The project is funded by Parent Project onlus. The aim of this study is to develop a system for an autologous stem cell transplantation in DMD patients, thus bypassing the need for a donor immunocompatibile and immunosuppressive therapy

Autologous mesoangioblasts will be engineered with a HAC vector containing the entire 2.4 megabases of the human dystrophin locus and a number of additional cDNAs needed to optimize the process. Episomal HACs (Ren et al., 2006) have many advantages over conventional gene therapy vectors such as episomal maintenance (thus avoiding insertional mutagenesis) and the ability to carry the whole gene with its own regulatory elements; furthermore they can be engineered to express additional beneficial functions.

Engraftment, dystrophin production, amelioration of the pathology and functional recovery will be tested in transplanted mice. If successful, this project will set the stage for a cell therapy of DMD.

kind regards,



Responsabile scientifico
Parent Project Onlus

Numero verde: 800.943.333
tel. 06 66182811
Fax: 06 60513295
Email: f.ceradini@parentproject.it

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Comment by Ambrish Kapadia on September 11, 2011 at 12:30am

Dear Dr Francesca , What is the difference between Myoblasts and mesoangioblasts.


Myoblasts as I understand have not been so successful due to their short life and their easy recognition and destruction  by patient's immune system . You must be aware that  Dr Trembley has been working on the "myoblast transfer therapy" for more than 20 yrs without much success.


Will this therapy of Dr Cossu involve immunesuppression of the host ?

If the DMD boys do not have any immunocompatibile brothers or sisters can any 6/6 HLA compatible donor ( which can be found from NMDP or other similar bone marrow donor registries or even in patient's network , can be voluntary donor of the muscle ?


Will lookforward to yr comments at earliest


Rgds Ambrish

Comment by Andrew Kerr on May 28, 2011 at 12:26am
Is there any hope of finding a non-sibling that is immunocompatibile?  In our case, we just have the two boys, both with DMD.
Comment by amit gupta on May 27, 2011 at 1:49pm

This is very encouraging, Pat.

Comment by RAKTIM SINGH on May 27, 2011 at 8:09am
 Thanks Pat for the update. This is really wonderful news. 

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