NIH launches first US clinical trial of patient-derived stem cell therapy to replace dying cells in retina
NEI-led study to test safety of treatment for a form of age-related macular degeneration that currently lacks treatment.
Researchers at the National Eye Institute (NEI) are launching a clinical trial to test the safety of a novel patient-specific stem cell-based therapy to treat geographic atrophy, the advanced “dry” form of age-related macular degeneration (AMD), a leading cause of vision loss among people age 65 and older. The geographic atrophy form of AMD currently has no treatment.
“The protocol, which prevented blindness in animal models, is the first clinical trial in the US to use replacement tissues from patient-derived induced pluripotent stem cells (iPSC),” said Kapil Bharti, a senior investigator and head of the NEI Ocular and Stem Cell Translational Research Section. The NEI is part of the National Institutes of Health.
The therapy involves taking a patient’s blood cells and, in a lab, converting them into iPS cells, which have the potential to form any type of cell in the body. The iPS cells are programmed to become retinal pigment epithelial (RPE) cells, the type of cell that dies early in the geographic atrophy stage of macular degeneration. RPE cells nurture photoreceptors, the light-sensing cells in the retina. In geographic atrophy, once RPE cells die, photoreceptors eventually also die, resulting in blindness. The therapy is an attempt to shore up the health of remaining photoreceptors by replacing dying RPE with iPSC-derived RPE.
Before they are transplanted, the iPSC-derived RPE are grown in sheets one cell thick, replicating their natural structure within the eye. This monolayer of iPSC-derived RPE is grown on a biodegradable scaffold designed to promote the integration of the cells within the retina. Surgeons position the patch between the RPE and the photoreceptors using a surgical tool designed specifically for that purpose.
Under the Phase I/IIa clinical trial protocol 12 patients with advanced-stage geographic atrophy will receive the iPSC-derived RPE implant in one of their eyes and be closely monitored for a period of at least one year to confirm safety.
A concern with any stem cell-based therapy is its oncogenic potential: the ability for cells to multiply uncontrollably and form tumours. In animal models, the researchers genetically analysed the iPSC-derived RPE cells and found no mutations linked to potential tumour growth.
Furthermore, the use of an individual’s autologous (own) blood cells is expected to minimize the risk of the body rejecting the implant.
Should early safety be confirmed, later study phases will include more patients to assess the efficacy of the implant to prevent blindness and restore vision in patients with geographic atrophy.
An FDA requirement for moving forward with the clinical trial was the establishment of GMP protocols to ensure that the iPSC-derived RPE are a clinical-grade product. GMP protocols are key for making the therapy reproducible and for scaling up production should the therapy receive FDA approval.
The preclinical research for the trial was supported by the NEI Intramural Research Program and by an NIH Common Fund Therapeutic Challenge Award. The trial is being conducted at the NIH Clinical Center in Bethesda, MD.
Related News
-
News BioNTech to begin mRNA vaccine manufacturing in Rwanda by 2025
German biotechnology company BioNTech has stated their intentions to begin production at their mRNA vaccine factory in Rwanda by 2025, which will mark the first foreign mRNA vaccine manufacturing site on the continent of Africa. -
News Identifying Alzheimer’s Disease biomarker proteins with whole blood tests
A University of Manchester spin-out pharmaceutical company, PharmaKure, has reported successful study results for the quantification of Alzheimer’s Disease biomarker proteins with a whole blood test. -
News Bill & Melinda Gates Foundation to boost mRNA vaccine initiatives in Africa with USD $40m
To address vaccine inequality and accessibility issues, the Bill & Melinda Gates Foundation aims to deliver USD $40m to various biotech companies and vaccine manufacturers in support of mRNA vaccine development. -
News CPHI Podcast Series: Exploring neurological frontiers in Alzheimer's and beyond
The next episode of the CPHI Podcast Series delves into the science and background behind some recent developments in the field of Alzheimer's disease and neurological disorders. -
News Is patient centricity the future of pharmaceutical manufacturing?
In this interview with Sandra Sánchez y Oldenhage, President of PharmAdvice, she speaks to the importance of considering patients in the manufacturing stages of the pharmaceutical supply chain, and how it can redefine healthcare. -
News CPHI Podcast Series: How to leverage AI for Drug Discovery
Artificial intelligence is the topic of debate in the latest episode from the CPHI Podcast Series, where Digital Editor Lucy Chard speaks with Bill Whitford of DPS Group about the integration of AI in healthcare. -
News Pfizer forges ahead with blood cancer therapy after approval from FDA
Pfizer gains accelerated approval from the US FDA for their new bispecific antibody therapy for multiple myeloma, set to address an unmet need for patients. -
News Alzheimer's drug donanemab deemed effective in landmark clinical trial
Results from the TRAILBLAZER-ALZ 2 Randomised Clinical Trial into the use of donanemab to treat early symptoms of Alzheimer’s disease have been analysed.
Position your company at the heart of the global Pharma industry with a CPHI Online membership
-
Your products and solutions visible to thousands of visitors within the largest Pharma marketplace
-
Generate high-quality, engaged leads for your business, all year round
-
Promote your business as the industry’s thought-leader by hosting your reports, brochures and videos within your profile
-
Your company’s profile boosted at all participating CPHI events
-
An easy-to-use platform with a detailed dashboard showing your leads and performance