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A Cure in Progress!!!


If you’re reading this blog for the first time, we who have Stargardt’s Disease are experiencing a loss of vision due to macular degeneration.

I don’t usually post about clinical trials because I really value the importance of adapting and coping with our vision loss. I do realize that we can’t ignore science and the hope for a cure, but I value overall wellness in the meantime. I think it’s only right that I include some information about clinical trial on this site. Last week there was a study update on last year’s publication. It isn’t a new publication, but it lets us know that people are still working hard to find a cure and it’s important to stay educated on their findings. So here’s the breakdown of the study. As soon as they release another publication, I’ll be sure to post about it. But as always, live life to the fullest and enjoy the vision you have and how special it makes you! Keep hope alive but don’t stress over a cure because you are awesome either way!

This study, which began in 2011 is expected to present result in December 2015!! Only one onth away! Click here to read the 2014 publication. Scroll down to read my summary.

Here’s the thing about embryonic stem cells: they are our source of hope for replacement cells as a regenerative medicine! At the same time, when stem cells are placed in a new environment, they have the potential to change and renew themselves which could be unsafe. The damage could be formation of a tumor, development into unwanted cells or rejection by your body’s immune system. It may sound a little scary but we are so fortunate to have brilliant people dedicating all their time to determining the safety of our cure (human embryonic stem cells hESC)!

Here’s how they are going about this: They have two studies to assess the primary concern of safety and tolerability of stem cell transplants in nine patients with Stargardt’s (age >18) and nine patients with Age-related Macular Degeneration (age >55). They organized three dosage groups for each disorder (50,000, 100,000, and 150,000 cells). The follow up was for about 22 months with a series of examinations. I believe the update includes a group of four better vision (20/100) SMD patients at 100,000 cells and also a group of three SMD patients at 200,000 cells. Don’t forget to read the full article if you want more details!

Here’s what they found!!!!: there was no evidence of harmful increase in cells, rejection, or safety issues related to the transplanted tissue. That is not to say that there were no adverse events. The harmful events were associated with the eye surgery and suppression of he immune system to allow the survival of the transplanted stem cells. Here’s what they reported: 13 (72%) of 18 patients had patches of increasing subretinal pigmentation consistent with transplanted retinal pigment epithelium. Best-corrected visual acuity, monitored as part of the safety protocol, improved in ten eyes, improved or remained the same in seven eyes, and decreased by more than ten letters in one eye, whereas the untreated fellow eyes did not show similar improvements in visual acuity. Vision-related quality-of-life measures increased for general and peripheral vision, and near and distance activities, improving by 16-25 points 3-12 months after transplantation in patients with atrophic age-related macular degeneration and 8-20 points in patients with Stargardt's macular dystrophy.

Here’s what it all means: The results of this study provide the first evidence of the medium-term to long-term safety, transplant survival, and possible biological activity of stem cell progeny that is able to develop into more than one cell type in individuals with any disease. The results suggest that hESC-derived cells could provide a potentially safe new source of cells for the treatment of various unmet medical disorders requiring tissue repair or replacement. (STARGARDT’S!)

Thank you to:

Prof Steven D Schwartz, MD, Prof Carl D Regillo, MD, Prof Byron L Lam, MD, Dean Eliott, MD, Prof Philip J Rosenfeld, MD, Ninel Z Gregori, MD, Jean-Pierre Hubschman, MD, Prof Janet L Davis, MD, Gad Heilwell, MD, Marc Spirn, MD, Joseph Maguire, MD, Roger Gay, PhD, Jane Bateman, RN, Rosaleen M Ostrick, MPH, Debra Morris, MPH, Matthew Vincent, PhD, Eddy Anglade, MD, Prof Lucian V Del Priore, MD, Prof Robert Lanza, MD!

And here's the link where I saw the update:

https://clinicaltrials.gov/ct2/show/NCT01345006

"No object is mysterious. The mystery is your eye." - Elizabeth Bowen

#research #stargardts

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