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.
Dr. FRANCESCA CERADINI
Parent Project Onlus
Numero verde: 800.943.333
tel. 06 66182811
Fax: 06 60513295