Grounded in Science
A balance of research news and well-being for the Usher syndrome community.
March is a busy month here at the Usher Syndrome Coalition. A group of our U.S.-based USH Champions are preparing to meet with their Members of Congress to advocate for increased federal funding for Usher syndrome research. We will share how it went next month!
USH Connections Conference | July 19-20, Rochester, NY + Online
Hosted in partnership with the National Technical Institute for the Deaf
In other news, can you believe there are only 4 months until the 2024 USH Connections Conference? We look forward to meeting you there or online! Register here.
Have you joined the Usher Syndrome Coalition Discord Community Server? It’s a safe place for the community to connect with each other. Join here: https://discord.gg/czwHGaDu7W
Research Spotlight
jCyte and Phase 3 U.S. Pivotal Trial
On February 21, 2024, jCyte released this exciting press release: “jCyte Inc. Announces Positive Pre-Phase 3 FDA Type B Meeting and Outlines Plans to Start Pivotal Trial of jCell® for Retinitis Pigmentosa in The Second Half of 2024.”
jCyte is a California based biotechnology company working to develop an innovative therapy to treat degenerative retinal disorders, including retinitis pigmentosa (RP), and has received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for its “first-in-class allogeneic cell therapy, jCell.” jCells are similar to stem cells that haven’t yet fully developed into mature photoreceptors and they work to preserve vision by releasing substances that reduce photoreceptor cell death and promote the function of surviving photoreceptors. Because cone survival is dependent on a substance released by rods, this cell therapy acts as a gene agnostic vision loss prevention treatment.
jCyte’s phase 2/3 clinical trial design was approved by the FDA and, jCyte states that, “Enrollment is expected to begin the second half of this year and more information will be shared when appropriate."
What this means for Usher syndrome: jCell, this first-in-class allogeneic cell therapy, moving forward to Phase 3 brings us one step closer to a potential treatment for those with various forms of RP, including those living with Usher syndrome.
Check out our Current USH Research page specific to USH subtype as well as gene-independent therapeutic approaches.
In Case You Missed It: Science News Feature
Human Neural Progenitors Expressing GDNF Enhance Retinal Protection in a Rodent Model of Retinal Degeneration
October 3, 2023: Many modalities are being evaluated for the treatment of retinal degenerative diseases such as retinitis pigmentosa (RP), including stem cell therapy where dead or damaged cells are replaced by healthy ones. In a previous study, subretinal injections of human neural progenitor cells (hNPCs) and an engineered version that expresses a nerve growth factor (hNPCGDNF) successfully preserved visual function. However, these experiments were performed using mouse models showing early stages of retinal degeneration and did not translate to clinical patients in later stages of the disease.
In this study, two groups of Royal College of Surgeon (RCS) rats, which are a well-established model for RP, received subretinal injections of hNPC and/or hNPCGDNF in one eye at different time points (21-23 days and 60 days) to assess their effect on early and late stages of retinal degeneration. The remaining eye was used as a control. At 90 days post-treatment, vision was measured and then all rats were sacrificed, and retinas were harvested and processed. Results show that subretinal injection of human neural progenitors expressing GDNF enables better visual and cellular preservation than just hNPC at both early and late stages of disease. Treatment with hNPCGDNF yielded multiple benefits, including activation of specific cell-survival pathways, reduction of oxidative stress, and promotion of cell regulation processes such as phagocytosis which improves cell health.
What this means for Usher syndrome: Previous preclinical studies demonstrated the potential of hNPC in treating RP, which resulted in an ongoing Phase 1/2a clinical trial. This new study revealed the addition of GDNF to hNPC outperformed hNPC alone for the treatment of early and late-stage RP and may become a potential therapeutic option one day.
For more science news, check out our Science News page, organized by treatment approach and type of Usher syndrome.
DISCLAIMER: The Usher Syndrome Coalition does not provide medical advice nor promote treatment methods. USH Science News is intended to help summarize more complex literature for the community to use at their own discretion.
On Well-Being: Humor
Humor has been proven to be a valuable resource in stress management. If this is something you struggle with, don't worry, you're not alone! Fortunately, humor is a skill that can be learned, with practice...
USH Life Hack of the Month
(Send your USH life hacks to info@usher-syndrome.org.)
The Be My Eyes app can also be used for generating Image Descriptions of a photo. If a friend texts you a photo and you aren't quite sure what you're looking at, click on share photo then scroll down to “Describe with Be My Eyes” for a detailed Image Description! -USH3A iPhone user.
