The EU has given the green light to a new project that focuses on the most common and debilitating form of muscular dystrophy, known as Duchenne Muscular Dystrophy (DMD). The project, called MYOAMP, has identified a gap in the current body of knowledge concerning stem cell therapies for DMD, and aims to pool existing studies into a single knowledge base that can be used as a synthesised resource in a final preparatory stage leading to clinical studies. MYOAMP is unique in that the project consortium is not only comprised of research institutions and SMEs, but includes patient organisations adding invaluable insight to the project.
DMD affects over 30 000 people in Europe, a number equal to the population of Monaco, where the MYOAMP consortium gathered recently to officially launch the three year project funded under FP6.
|MYOAMP will apply a novel approach
to existing studies by influencing protein production via mRNA.
MYOAMP is basing their research on a newly designed technique known as ‘exon-skipping'.
An exon is a portion of a gene that codes for a particular protein, and when
the gene is responsible for proteins harmful to the organism, as is the case
in DMD, experts have developed a technique to ‘skip' the exon, leaving them with
a new protein offering potential therapeutic qualities.
The project brings together experts from across Europe to define best practice procedures for transferring human muscle stem cells in Good Manufacturing Practice (GMP) conditions to be used in exon-skipping to treat DMD.
Currently, little is known about growing human cells in GMP conditions, which certifies that they are suitable for clinical trials. Several studies have been carried out on animals, but never in a systematic manner, making comparisons difficult. MYOAMP aims to change that through the dedicated study of the amplification of quality myogenic (muscle) stem cells used in cell therapy techniques.
The expected result of the project is the definition of protocols in obtaining amplified human stem cells allowing for optimised efficiency in clinical trials. Additionally, MYOAMP will provide guidelines and standard operative procedures for growing these cells that will address technical, ethical and safety issues under GMP conditions.
To maximise the impact of the project, MYOAMP has chosen from the beginning to
include patient associations throughout the development of the project. International
patient associations such as the Duchenne Parent Project in France and the UK,
the Association Monégasque Contre les Myopathies, and the Association Française
contre les Myopathies are all participating. As such, the end-users of the research
will be the first to learn of the progress of the project. Participants feel
this is the best way to ensure public health officials understand the necessity
of supporting clinical trials.