Cell therapy weekly: MSCs show promise to treat rare pediatric heart condition

This week: Longeveron treats first patient with MSCs, Duchenne muscular dystrophy improved in mouse model and new funding for development of synthetic ‘gene circuits.

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Mar 01, 2018
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The news highlights:

Infant with hypoplastic left heart syndrome treated with MSCs
Study demonstrates cell therapy improves Duchenne muscular dystrophy in a mouse model
Could cell therapies be improved with synthetic gene circuits?

Infant with hypoplastic left heart syndrome treated with MSCs

Longeveron (FL, USA) has commenced a new clinical trial to treat infants with a rare heart condition with allogeneic mesenchymal stem cells with the treatment of the first patient. The trial, a partnership between Longeveron, the University of Maryland and Johns Hopkins University (both MD, USA) will treat 30 infants with the aim of improving ventricular function.

"The hope is the stem cell therapy available through this clinical trial will be a game-changer for these infants, an effective way to improve their outcomes and allow them to live longer lives," commented Sunjay Kaushal, Director of Pediatric Cardiac Surgery at the University of Maryland, who performed the procedure.

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Study demonstrates cell therapy improves Duchenne muscular dystrophy in a mouse model

In research published in Stem Cell Reports, cardiosphere-derived cells improved cardiac muscle function, walking ability and overall survival when administered to a mouse model of Duchenne muscular dystrophy. CAP-1002, developed by Capricor Therapeutics (CA, USA), was demonstrated to stimulate tissue repair and regeneration by reducing inflammation though exosome secretion. CAP-1002 will now be delivered to approximately 84 boys and young men with Duchenne muscular dystrophy as part of the HOPE-2 trial.

“We look forward to initiating HOPE-2 so we can see in a double-blind, randomized, placebo-controlled trial whether the effects seen in the mice and in the first human study are validated,” explained Linda Marbán, Capricor president and chief executive officer. “While gene therapy offers tremendous potential in restoring dystrophin expression and sustaining muscle function, there will still be significant inflammation and fibrosis which can offset the restorative effects. We believe that CAP-1002 can work synergistically with the emerging disease modifying therapies to control those pathological aspects of Duchenne muscular dystrophy.”

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Could cell therapies be improved with synthetic gene circuits?

US$53 million of funding has been awarded to Senti Biosciences (CA, USA) to further develop their platform to designing adaptive therapies to fix genetic errors. Adaptive therapies powered by ‘genetic circuits’ could act locally, respond to a range of disease conditions, be controlled externally, and implement therapeutics with multiple steps and components.

"Synthetic biology has advanced significantly over the last several years, and the team we have assembled at Senti is uniquely capable of capitalizing on its progress to turn adaptive therapies into commercial reality,” commented Tim Lu, co-founder and CEO of Senti Biosciences. “This funding round will accelerate the scaling of our genetic circuit programming platform and its translation into clinical treatments." 

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For more weekly cell therapy news, read previous editions of the cell therapy weekly.

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