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Posts tagged Stem Cells

Retinal Regeneration: Releasing Your Inner Salamander

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For someone with a retinal disease such as retinitis pigmentosa or macular degeneration, their vision loss is caused by photoreceptor degeneration. Photoreceptors are the retinal cells that capture light and convert it into electrical signals, which are sent back to the brain where they are used to create the images we see.
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ARVO 2018: Dr. Henry Klassen Provides Update on jCyte Stem Cell Trials

Dr. Henry Klassen, jCyte co-founder and investigator at UC Irvine, provides an update in the video below on the clinical trials for an RP therapy derived from stem cells.

Vision Improvements Reported in Early Stem Cell Trial for Wet AMD

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Two patients with advanced wet age-related macular degeneration (AMD) in a Phase I clinical trial demonstrated improved visual acuity sustained for one year after a sheet of retinal pigment epithelial (RPE) cells derived from embryonic stem cells was transplanted under their retinas. Each patient had one eye treated. Vision improvement for one patient was 29 letters or about 6 lines on an eye chart. The other had a gain of 21 letters or about 4 lines.
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FFB’s Investments Are Filling the Pipeline for Vision-Saving Therapies

GXM_7140With five gene-therapy clinical trials underway or soon to begin, Applied Genetic Technologies Corporation (AGTC) is generating tremendous excitement for the potential to overcome vision loss from several inherited retinal diseases.

At the Foundation’s Investing in Cures Summit on September 16 in Chicago, Sue Washer, AGTC’s chief executive officer, emphasized FFB’s crucial role in moving the company and its projects forward. “We as an organization would not be here today without FFB,” she said. “And that all started with the work that was funded by the Foundation in Bill Hauswirth’s lab at the University of Florida.” Bill Hauswirth, PhD, is one of AGTC’s scientific co-founders, and his groundbreaking gene-therapy research has been funded by FFB for 20 years.
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jCyte Stem-Cell Therapy Moves into Phase IIb Clinical Trial for RP

These are retinal progenitors.

These are retinal progenitors.

The stem-cell therapy company jCyte is launching a Phase IIb clinical trial of its therapy for people with retinitis pigmentosa (RP). The trial is taking place at University of California, Irvine, and Retina-Vitreous Associates Medical Group in Los Angeles. The 70-participant study is being led by Henry Klassen, MD, PhD. Participant enrollment is scheduled to begin this month.

The treatment involves intravitreal injection of retinal progenitor cells (RPCs), which are stem cells that have partially developed into the retinal cells that make vision possible. Based on lab studies, researchers believe the treatment can preserve and potentially rescue the patient’s existing photoreceptors, thereby saving and possibly restoring vision.
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Unregulated Stem-Cell Therapy Causes Severe Vision Loss for Three Florida Women

A report today in The New England Journal of Medicine (NEJM) describes the cases of three women with age-related macular degeneration (AMD) who lost much of their eyesight after receiving ocular injections of stem cells derived from their own fat tissue. All of the women had good enough eyesight to drive before the procedures. Each paid $5,000 to receive the injections from a private clinic in Sunrise, Florida. The New York Times and other major media outlets have published news stories on the NEJM report.
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Stem-Cell Therapy for Retinitis Pigmentosa Safe Thus Far in Early Human Study

Dr. Klassen in his labAn emerging stem-cell-derived treatment designed to preserve and potentially restore vision in people with retinitis pigmentosa (RP) has demonstrated a favorable safety profile in an ongoing Phase I/II clinical trial at the University of California, Irvine. The therapy is being developed by the regenerative medicine company jCyte with trial funding from the California Institute for Regenerative Medicine. Earlier research funded by the Foundation Fighting Blindness helped advance this therapeutic approach toward a human study.

Given this trial is one of the first-ever for a stem-cell-derived therapy for RP, this safety report is good news and an important step in the right direction. We at the Foundation look forward to additional reports from this study in the coming years as the trial advances.

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A Stem-Cells Video for Kids (and Newbies of All Ages)

Animated videoGiven that inherited retinal diseases are often diagnosed in childhood, it’s only fitting that kids get the opportunity to learn about the promising research and emerging treatments under development to save and restore their vision. Of course, the biology and genetics can be difficult to understand for even an educated adult, so when something kid-friendly is produced, especially a video, we are eager to share it.
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Stem Cells Derived from Patient’s Skin Provide Insights into AMD

Dr. Stephen TsangInduced pluripotent stem cells (iPSC) — stem cells derived by genetically tweaking a small sample of a person’s skin or blood — are again demonstrating their power for helping researchers fight retinal diseases.

In this latest development, Stephen Tsang, M.D., Ph.D., a Foundation-funded researcher at Columbia University, used them to create a human model of age-related macular degeneration (AMD). The advancement not only gives us a better understanding of how AMD occurs; it provides a new, and potentially better, platform for testing vision-saving therapies. Results of the study were published in Human Molecular Genetics.

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Have Scientists Found a Better Way to Make Stem Cells?

Stem cell cultures, courtesy of the NIHCould soaking a patient’s blood cells in a liquid with the acidity of vinegar be a safer and more effective way to develop stem cells for vision-restoring, retinal-disease treatments? Based on a study recently published in Nature, it might be. The research has a long way to go before it is ready for prime time — i.e., evaluation in humans — but the results thus far are intriguing.
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