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Duchenne Dystrophy, CNR “Identified new mechanism”

For the most frequent hereditary muscle disease still no cure is available, new hope is arriving from CNR of Pozzuoli.

November 16, 2018
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Duchenne Muscular Dystrophy is the most common hereditary muscle disease. The most affected are mainly male children.

The onset is early, and, besides the skeletal muscles, also many organs, like the heart, the lungs and the brain are affected. Today, there is still no cure available for this pathology.

The Study of the Institute of biomolecular chemistry of Cnr-Icb

Good news arrives from a study published in Nature Communications , realized by the institute of biomolecular chemistry of the National Council of Research in Pozzuoli.

The team guided by Vincenzo Di Marzo, discovered how in a model animal it is possible to significantly slow down the course of the disease and to recuperate the lost motor functions, by means of medical drugs capable of regulating the function of cannabinoids produced by our own organism.

“Together with our researchers we have been engaged for years now in studying the ever growing number of molecules  which make up the system of endocannabinoids and the receptors and enzymes connected with it, from a chemical-structural and functional point of view” - Di Marzo tells us -  “In many cases our studies demonstrate how the alterations of this system are associated with various pathologies of the neurological kind, like dementias, epilepsy, acute and chronic pain, and various forms of tumours. For some of them, poorly treatable or not treatable at all from a clinical point of view, medical drugs which act, amongst others, by regulating the function of endocannabinoids, are yielding positive results”.

The alterations of the endocannabinoid system in muscle pathologies remain however still little known.

“Our study addresses this very aspect and for the first time has found important alterations of the endocannabinoids present in skeletal muscles affected by Duchenne Muscular Dystrophy, and in particular hyperactivity of the CB1 receptor in the striated muscle tissue, both in man and in an animal models, explains Fabio Arturo Iannotti of Cnr-Icb who planned and conducted the research project.  

“We additionally demonstrated, how, with the repeated administration of medical drugs capable of mitigating this hyperactivity, a partial but significant recovery of the motor functions is obtained, as well as a reduction of inflammation in the animal model”.
Other collaborators in the research were the Institute of Genetics and Biophysics of the National Council of research of Naples, the Pharmacy Department of the Federico II University, the Santa Lucia Foundation in Rome, the Telethon institute of genetics and medicine of Pozzuoli and the Paediatric Hospital Gaslini of Genoa.  

What do the current therapies consist of?

Today, therapy is based mostly on the consumption of strong anti-inflammatory drugs with which it is possible to contain the symptomatology in a partial manner and with which it is difficult to conduct a risk-benefit evaluation.

“Of particular interest – Di Marzo states -  is the discovery that the antagonists of the CB1 receptor, promote the maturation of muscle stem cells, of which the dysfunction renders regeneration of muscle fibres ineffective, and at the same time contrast the initiation of inflammation and the muscle degeneration typical of the pathology. The latter effect is similar to the one we found in another study in which we utilized some natural and non-psychotropic isolated cannabinoids of  ‘Cannabis Sativa’”.
                                                                  
“Even though it is always necessary to interpret the results obtained in experimental models, with much caution - Di  Marzo concludes - the auspice is that a similar scientific discovery paves the way to new and more effective therapies for muscle pathologies and can offer a different life perspective to patients affected by Duchenne Muscular Dystrophy”.

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