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Therapeutics for Rare and Neglected Diseases

Use of Retinal Progenitor Cells for the Treatment of Retinitis Pigmentosa

Retinitis pigmentosa (RP) is a severe form of hereditary blindness characterized by progressive damage to and loss of the light-sensing cells of the retina. Most patients have night blindness in their early teens, typically progressing to legal blindness by age 40. There are no approved treatments for RP. The lead collaborator has identified an innovative approach to save the light-sensing cells of the eye. The purpose of this project is to support the development of these retina-derived cells as a transplantable treatment to stop the cellular damage that leads to blindness in RP patients.

Scientific Synopsis

RP is a rare genetic disease of the eye characterized clinically by the loss of rod photoreceptor cells, followed by cone-cell degeneration. As rod cell function degenerates, impaired night vision typically appears as an initial symptom. When rod photoreceptors die, cone photoreceptors are destabilized, resulting in progressive loss of visual acuity and color/central vision. The principal investigators have developed a cell-based therapeutic approach to stop retinal cell degeneration.

Work in animal models has demonstrated the ability of grafted retinal-derived, lineage-restricted progenitor cells (RPCs) to promote survival and function of dystrophic host photoreceptors. The principal investigators and others have demonstrated that injected donor RPCs survive in the vitreous and migrate to the retina, where they integrate with the host cellular architecture. As lineage-restricted progenitor cells, these engrafted RPCs are able to differentiate further into rod photoreceptors, providing additional support to the dependent cone cells. The therapeutic hypothesis is that the release of trophic factors and engraftment by donor RPCs should rescue host cone cells, improving existing vision and delaying or preventing total blindness. In a pre-Investigational New Drug (IND) meeting, the Food and Drug Administration (FDA) identified additional studies needed to support submission of a full IND package.

Lead Collaborator

University of California, Irvine
Henry Klassen, M.D., Ph.D.

Public Health Impact

There are no approved treatments for retinitis pigmentosa. The disease typically results in legal blindness by age 40.

Outcomes

TRND researchers and the collaborator performed IND-enabling studies to support submission of a full IND package. TRND researchers completed a biodistribution study of the formulated RPCs (jCell) in pig eyes. The results of these studies were incorporated into an IND filing by the collaborator’s start-up company, jCyte, Inc. The FDA cleared the IND to begin human clinical trials. jCyte will now be able to continue development of jCell using internal resources. This TRND project is now complete.

Last updated: 09-06-2017
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