Reacción a "A blood-based cell therapy slows muscle deterioration in children and young people with Duchenne muscular dystrophy, according to a phase III trial"

HOPE-3 is the phase 3 clinical trial for Deramiocel. It is a systemic allogeneic cell therapy (based on cells derived from cardiospheres, or CDCs) that has been evaluated in participants with Duchenne muscular dystrophy (DMD) who are in the late ambulatory stage or non-ambulatory.

This is a very important finding, as few clinical trials target this population of patients with advanced Duchenne muscular dystrophy who are non-ambulatory or in the late stages of ambulation.

As demonstrated in this article, Deramiocel offers significant therapeutic benefits for both skeletal and cardiac muscle in advanced DMD, with manageable safety and tolerability.

In the context of DMD, Deramiocel does not correct the dystrophin deficiency in these patients, but it is capable of slowing the progression of the disease.

The therapeutic effects of Deramiocel are very encouraging:

• A 54% reduction in the mean progression of skeletal muscle disease over 12 months.
• With regard to the effects on the progression of cardiomyopathy, there was a 119 per cent slowdown in the average progression of cardiac disease, representing a stabilisation of cardiomyopathy progression not seen with other cardiac treatments for DMD.
• The preservation of upper limb function (the primary therapeutic objective in HOPE-3) yielded significant clinical benefits in advanced DMD. The indicators show an improvement in quality of life, taking into account the ability to eat independently (as reported by DMD patients) following treatment with Deramiocel at 12 months, which suggests a significant potential benefit in maintaining independence.

There are no approved specific therapies for cardiomyopathy in DMD, which is the leading cause of death in patients with this condition. The loss of dystrophin leads to inflammation and myocardial damage, which eventually results in the death of cardiomyocytes and their replacement by fibroadipose tissue. The stabilisation observed following treatment reflects the preservation of viable myocardium, resulting in the maintenance of overall contractile function.

Deramiocel significantly slows the progression of DMD with a favourable safety profile. Neither the preservation of upper limb function nor the attenuation of cardiomyopathy had previously been demonstrated in the population with advanced DMD. Another very important factor is that the study included DMD patients with various mutations; as this is a therapy that does not depend on the patient’s specific mutation, it is widely applicable.

These findings reinforce Deramiocel’s status as a safe, effective and promising therapy for people living with DMD, although longer-term follow-up is needed to establish its durability and long-term safety.