Regenerative medicine for retinitis pigmentosa appears promising

Amarantus BioScience Holdings, Inc., announced results of a study showing that mesencephalic-astrocyte-derived neurotrophic factor (MANF) can prevent vision loss in an animal model of retinitis pigmentosa.

MANF, a naturally-occurring protein that reduces and prevents apoptosis in response to injury or disease, has demonstrated efficacy in three animal models of retinitis pigmentosa. 

In the latest study, animals received an intravitreal administration of MANF after the onset of retinal degeneration on day 31, which resulted in a statistically significant protective effect on visual acuity on day 38 and day 45 compared with vehicle-treated animals.

This is the first observation of MANF providing a functional improvement in vision in a model of retinitis pigmentosa.

"These promising data provide a compelling basis to continue the further development of MANF in RP towards first-in-man studies," said Gerald E. Commissiong, president & CEO of Amarantus. “Ultimately, we believe proof of concept for MANF in human studies in ocular diseases will support MANF development in other therapeutic areas where significant compelling preclinical efficacy data has been published and confirmed by independent groups, as well as the company's own datasets. We firmly believe MANF has blockbuster potential in ophthalmology, neurology, cardiology and metabolic disorders, including diabetes.”