Novel Stem Cell Treatment can be a Ray of Hope for Children
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Researchers put forward a safe and efficient new stem cell therapy for regenerating cardiac function in pediatric patients

In a new study, scientists at Okayama University isolated cardiac stem cells and assessed their potential use as regenerative therapy in young patients with cardiac defects. They confirmed the safety and effectiveness of their proposed treatment in early-phase trials and even identified the mechanism through which the stem cells improved cardiac function.

Dilated cardiomyopathy (DCM) is a condition caused by the weakening of the heart muscle, affecting the ventricles. If allowed to progress unchecked, DCM can lead to heart failure and death, especially in children. The only cure, at present, is a heart transplant, which comes with its own challenges: long waiting times to secure a suitable donor heart, the possibility of organ rejection, long hospitalizations and recovery times, among others.

In recent decades, stem cells have become the cornerstone of regenerative medicine, allowing medical professionals to treat damaged organs and reverse the course of several diseases. Scientists have turned to “cardiosphere-derived cells” (CDCs), a type of cardiac stem cells known to have beneficial effects in adults suffering from specific heart conditions. By developing (“differentiating”) into heart tissue, CDCs can reverse the damage inflicted by diseases. However, little is known about their safety and therapeutic benefit in children.

In a study published in Science Translational Medicine, the team not only showed the effectiveness of CDCs in replenishing damaged tissues in DCM but also revealed how this happens.

--The researchers tested their phase 1 trial involved 5 young patients suffering from DCM. The scientists now had a better idea of the suitable dose of CDCs to give their young patients, thanks to the pre-clinical trials in animals.
--1 year after injection, the patients showed no sign of severe side effects from the treatment, but most importantly, there were encouraging signs of improved heart function. However, they are satisfied that CDC treatment appears sufficiently safe and effective to progress to a larger clinical trial.

--Another important finding was the mechanism through which CDCs actually lead to improved cardiac function. Indeed, their analyses revealed that transplanted cells secrete small vesicles called “exosomes,” which are enriched with proteins called “microRNAs” that initiate a whole cascade of molecular interactions.
--These microRNA-enriched exosomes have two effects.
1) It blocks the damage-inducing cells from causing further harm to the heart tissue.
2) It induces the differentiation of stem cells into fully functioning cardiac cells (“cardiomyocytes”), starting the regenerative process.
--This generates hope that injecting these exosomes alone might be enough to reverse this type of heart damage in patients, bypassing the need for CDCs in the first place.

Looking back on their research, the scientists are hopeful that a phase 2 trial will confirm their suspicions, and what this could mean for future patients. Prospective transplant patients sometimes wait for years for a donor heart to become available. This type of therapy could allow them to live relatively normal lives, and even prevent the need for a transplant altogether for patients who have not yet reached such a critical stage.

Source: http://www.okayama-u.ac.jp/eng/research_highlights/index_id121.html
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