'The adult mammal lacks the ability to regenerate neurons lost to retinal damage or disease in a meaningful capacity. However, previous studies from this laboratory have demonstrated that PNU-282987, an α7 nicotinic acetylcholine receptor agonist, elicits a robust neurogenic response in the adult murine retina. With eye drop application of PNU-282987, Müller glia cells re-enter the cell cycle and produce progenitor-like cells that can differentiate into various types of retinal neurons. In this study, we analyzed the regenerative capability of PNU-282987 in two retinal disease models and identified the source of newly regenerated neurons. ... These results demonstrate that PNU-282987 can induce retinal regeneration in adult mice following onset of retinal damage. The ability of PNU-282987 to regenerate retinal neurons in a robust manner offers a new direction for developing novel and potentially transformative treatments to combat neurodegenerative disease."
This is why we need our Usher 1F community to help us raise funds for research for a cure. We cannot rely on big pharma and investors to fund it for us.
"The most common causes of vision impairment in developed nations are abnormalities of the posterior eye tissues. Poor drug distribution to lesions in patient's eyes is a key barrier to ocular disease therapy. The existence of barriers, such as the corneal barrier, aqueous barrier, and inner and outer blood-retinal barriers, severely limits medication accessibility in these locations. Because of its anatomical peculiarities, the posterior portion is particularly difficult to access for medications. The use of several new strategies for drug delivery is therefore a viable option for enhanced therapy of ocular disorders since recent advances in ocular drug delivery systems research have brought fresh insights into drug development.
"This article provides an overview of several aspects of ocular medication administration to the posterior region, with a focus on nano carrier-based approaches, suprachoroidal drug delivery system and ophthalmic devices, including the structure of the eye, its barriers, delivery routes, and the present status of drugs/devices."
"Transplantation of retinal pigment epithelial (RPE) cells derived from embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs) have shown promising results in improving retinal function in various preclinical models of retinal degeneration and clinical studies without any severe side effects. ... This review summarizes the preclinical and clinical evidence of stem cell therapy and current limitations in utilizing stem cells for retinal degeneration."
"The therapy can be administered in a medical office setting without the need for goggles or other interventions. ...Based on preliminary evidence from the company's Phase 1/2a study, MCO-010 is potentially applicable not only for RP but other degenerative diseases of the eye irrespective of gene mutations."
"Our data demonstrate that pluripotent stem cell derived cone precursors can engraft into the host retina in close apposition to the host bipolar cells. Half of the transplanted mice exhibited visual behaviour and of these, 33% showed binocular light sensitivity. The majority of retinal ganglion cells (RGCs) exhibited contrast‐sensitive light responses and motion sensitivity, however quite a few exhibited unusual responses reflecting remodelling of the neural retina. Together these data suggest that pluripotent stem cell‐derived cone precursors can elicit conventional and unusual light responsiveness at advanced degeneration stages."
"Although there currently are no options available to treat retinitis pigmentosa (RP), a novel approach has been evaluated in a phase 2 clinical trial that determined the clinical effect of the injection of human retinal progenitor cells to improve vision and visual fields in this patient population by activating and preserving retinal cells to treat RP.
"The trial’s findings indicated that this treatment may be able to enhance vision and visual fields in specific patients with RP whose living cells can be rejuvenated, according to David S. Boyer, MD, an adjunct clinical professor of ophthalmology at the Keck School of Medicine of USC in Los Angeles, California.
"The phase 3 trial will begin in the third quarter of 2022."
The Phase 2b trial which starts in June, will be a randomized, double-blind, sham-controlled, multi-center trial in the USA enrolling patients with advanced RP. The treatment will involve a single intravitreal injection of MCO-010 to confirm its ability to improve the patients' vision. MCO has received orphan drug designations for RP and Stargardt disease from the FDA.
Mark Pennesi, PhD, the Kenneth C Swan Associate Professor of Ophthalmology at the Casey Eye Institute in Portland, who will lead the Phase I/IIa studies, said: “I’m eager to begin clinical proof-of-concept studies to establish whether Endogena’s approach could offer a new treatment paradigm in retinitis pigmentosa.
“This novel method of tissue repair holds great promise for patients with this devastating degenerative eye condition.”
"Researchers at the National Eye Institute (NEI) have determined how certain short protein fragments, called peptides, can protect neuronal cells found in the light-sensing retina layer at the back of the eye. The peptides might someday be used to treat degenerative retinal diseases."