ADMINISTER cells capable of “repairing the muscles” to slow Duchenne’s disease. This is the path attempted by researchers led by Craign McDonald of the University of California at Davis, for a degenerative pathology that currently has no resolutive therapies.
Duchenne is a genetically based disease that affects muscles from an early age, causing muscle weakness and inflammation, hindering movement or preventing it altogether. But the heart and respiratory muscles are also affected, so that these patients also suffer from cardiomyopathies and breathing difficulties. There are several strategies being studied, especially on the front of advanced therapies. One of these is based on the administration of cells capable of exerting a repairing action and slowing down muscle degeneration, with appreciable effects both in the movements of the arms and in the health of the heart. A hope, as the clinical trials that tested it are called, HOPE.
We talk about it on the pages of Lancet, with the publication of the HOPE-2 study, which tested the efficacy of this cell therapy on a group of patients older than 10 years, walking with difficulty and non-ambulatory. Twenty in all, divided into two groups: one received therapy (eight), the other (twelve) a placebo.
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What does cell therapy consist of
The therapy in question is made from cardiac cells coming from donors (and for this reason allogeneic, called CDC from Cardiosphere-Derived Cells) to which various properties are attributed. In fact, preclinical and clinical studies have suggested how the presence of these cells can have an immuno-modulating, anti-inflammatory, and regenerative effect.
The researchers gave CDCs intravenously to some patients for a year, with infusions three months apart. And they then measured whether, how much and how cell therapy improved movement, heart and respiratory function. To do this they used a scale that measures the motor skills of the upper limbs in the Duchenne (the Performance of Upper Limb scale), carried out magnetic resonance analysis and performed spirometric tests. The results showed that yes, CDCs appear to be beneficial by reducing the loss of muscle function in the upper limbs and inducing improvements in cardiac structure and function. On the other hand, no significant differences were observed in lung function. Not surprisingly, the authors comment: too many confounding factors for people with non-ambulatory Duchenne, starting with scoliosis. In general, with the exception of hypersensitivity problems, the therapy was also well tolerated.
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The significance of the first results
“HOPE-2 is the first therapeutic trial in Duchenne dystrophy to show stabilization in upper limb function and improve cardiac structure and function compared to placebo,” the researchers write. These benefits, the experts explain, would be attributable to the actions of the CDC, and in particular to the particles released by these cells: exosomes, containing substances capable of decreasing inflammation and promoting tissue regeneration. And these are not marginal improvements: slowing the loss of muscle function in these patients, who are highly dependent on the motility of the arms to move, eat and move objects, is essential in order to preserve their independence as much as possible.
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The next steps
The research will now continue expanding the study to more patients and lengthening the time to monitor the long-term effects of the therapy, in the HOPE 3 trial. The aim of the ongoing and future studies is to understand what benefits cell therapy can really bring to Duchenne. both in younger and more ambulatory patients, and in non-ambulatory people, on which historically less experiments have been made.