If you haven't already heard, results from the phase I trial using human embryonic stem cells for patients with Stargardt's Disease and Macular Degeneration have been published in the Lancet, and the results are very promising!
Nine patients were treated from each disease which involved surgical implantation of the stem cells into the retina of one affected eye. The main aim of phase I trials is to test the safety of the intervention, so whether there were an adverse events or complications from having the stem cells implanted, and in this case whether the stem cells 'stuck' to the eye. The investigators also looked at whether there was any improvement in vision.
Excitingly, the results were very promising - there was no major adverse events (although some patients with Stargardt's developed cataracts but this was treated surgically and didn't affect vision after treatment), no rejection of the implanted cells, no abnormal tumour growth and it looked as though the cells remained functioning at follow-up (which was up to two years after initial implantation). To top it off, there was also objectively reported improvement in vision in the treated eye!
To put it in perspective, this is still early days and we won't be running out to get stem cells injected tomorrow. Although it is definitely looking like a very possible treatment in the future and in our lifetime! The next step is to proceed to a phase II trial which will include a larger number of patients and find the best dose (number of cells to be injected) to gain the best results. Further evidence of the effectiveness will be developed to show how much vision can be restored, if it is permanent, whether the implantation needs to be done in the early stages of the disease and many other questions.
This is extremely exciting and shows how amazing modern medical science can be. It can give us all a little more hope that potentially in the future we may be able to see better, and if not us, the generations to come who will unluckily have to experience the hurdles we have faced.
For more information here are the links to the results and news reports:
Wall Street Journal
The Lancet
Good news indeed, but unfortunately we are pretty far from a real cure .
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This is promising... personally, I'm thinking the "Bionic Eye" from Second Sight has a better chance of being a help first. Can you imagine Google Glass... built in?
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DeleteI think this is very informative article which you have posted!! Most of the people are not aware about the eye disease as me. Through your post we will not be aware only but also be able to care for our best eyes.
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DeleteThat is so exciting that the stem cell research for your disease has improved so much! I cannot imagine what it would be like to have any kind of rare retinal disease, so congratulations for being such a warrior. We want to keep hearing updates about how all of the trials are going. Hopefully you can be a pioneer in this field.
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Thank you for taking an interest! I hope I can be too :)
Deletehello , my name is Florence . I have officially been diagnosed with Satgardts I am 12 and live in Italy , This is my blog
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I am hopefully going to be on a clinical trial to here in Rome .
Thanks Florence and I hope you are doing ok with SD! I will put a link to your blog on mine. Keep me posted on how you're going :)
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Yeah dear, you are right dear, there are many eye disorders which are bizarre also and I think heterochromia disorder is also one of them. One of my friends is also affected with this problem.
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