Stem cell-derived retinal pigmented epithelial cells. Cell borders are green and nuclei are red. (Photo Credit: Dennis Clegg, UCSB Center for Stem Cell Biology and Engineering)
Two UK patients suffering from vision loss caused by age-related macular degeneration (AMD) have regained their sight thanks to a stem cell-based retinal patch developed by researchers from UC Santa Barbara (UCSB). The preliminary results of this promising Phase 1 clinical study were published yesterday in the journal Nature Biotechnology.
AMD is one of the leading causes of blindness and affects over six million people around the world. The disease causes the blurring or complete loss of central vision because of damage to an area of the retina called the macula. There are different stages (early, intermediate, late) and forms of AMD (wet and dry). The most common form is dry AMD which occurs in 90% of patients and is characterized by a slow progression of the disease.
Patching Up Vision Loss
In the current study, UCSB researchers engineered a retinal patch from human embryonic stem cells. These stem cells were matured into a layer of cells at the back of the eye, called the retinal pigment epithelium (RPE), that are damaged in AMD patients. The RPE layer was placed on a synthetic patch that is implanted under the patient’s retina to replace the damaged cells and hopefully improve the patient’s vision.
The stem cell-based eyepatches are being implanted in patients with severe vision loss caused by the wet form of AMD in a Phase 1 clinical trial at the Moorfields Eye Hospital NHS Foundation Trust in London, England. The trial was initiated by the London Project to Cure Blindness, which was born from a collaboration between UCSB Professor Peter Coffey and Moorsfields retinal surgeon Lyndon da Cruz. Coffey is a CIRM grantee and credited a CIRM Research Leadership award as one of the grants that supported this current study.
The trial treated a total of 10 patients with the engineered patches and reported 12-month data for two of these patients (a woman in her 60s and a man in his 80s) in the Nature Biotech study. All patients were given local immunosuppression to prevent the rejection of the implanted retinal patches. The study reported “three serious adverse events” that required patients to be readmitted to the hospital, but all were successfully treated. 12-months after treatment, the two patients experienced a significant improvement in their vision and went from not being able to read at all to reading 60-80 words per minute using normal reading glasses.
Successfully Restoring Sight
Douglas Waters, the male patient reported on, was diagnosed with wet AMD in July 2015 and received the treatment in his right eye a few months later. He spoke about the remarkable improvement in his vision following the trial in a news release:
“In the months before the operation my sight was really poor, and I couldn’t see anything out of my right eye. I was struggling to see things clearly, even when up-close. After the surgery my eyesight improved to the point where I can now read the newspaper and help my wife out with the gardening. It’s brilliant what the team have done, and I feel so lucky to have been given my sight back.”
This treatment is “the first description of a complete engineered tissue that has been successfully used in this way.” It’s exciting not only that both patients had a dramatic improvement in their vision, but also that the engineered patches were successful at treating an advanced stage of AMD.
The team will continue to monitor the patients in this trial for the next five years to make sure that the treatment is safe and doesn’t cause tumors or other adverse effects. Peter Coffey highlighted the significance of this study and what it means for patients suffering from AMD in a UCSB news release:
Peter Coffey
“This study represents real progress in regenerative medicine and opens the door to new treatment options for people with age-related macular degeneration. We hope this will lead to an affordable ‘off-the-shelf’ therapy that could be made available to NHS patients within the next five years.”
The Stem Cellar 
My daughter is 32 was diagnosed with stargardts at age 10. The defective genes have been identified. How can we find out more.
You can learn more about this clinical trial ongoing in the UK by visiting the London Project to Cure Blindness website: http://thelondonproject.org. Here is a link to the original press release about this trial including contact information: http://thelondonproject.org/?q=latest-news/press-release-patients-regain-sight-after-being-first-receive-retinal-tissue-engineered
This is great news. How does one know which type of MD one have ?. Is this the same as RP ?
Hi Willie. Age-related macular degeneration (AMD) and retinitis pigmentosa (RP) both affect the retina of the eye and cause vision loss, but they are different diseases. AMD affects the cells in the macula of the retina, which causes blurriness or loss of central vision. Patients with RP don’t experience damage to the macula but to a different part of the retina that causes them to lose their peripheral vision. The best way to find out what type of AMD you have is to go to a doctor and get diagnosed.
My mother underwent a radiotherapy for remival of a tumour and her optic nerve has damaged. She had blurred vision for many years and last year she developed Occlusion. Due to this she cannot see at all. Would this treatment be of any use to her? Please suggest. Thanks in advance.
Hi Ketki, we are very sorry to hear about your mom’s condition. We suggest that you contact the researchers who developed this technology as they will be able to best answer your questions.
Is there a trial going on in the US? My dad has wet MD in one eye and dry in the other. He is now totally blind. He would jump at the chance to be part of a trial.
I am not aware of a trial testing this treatment in the US. I would contact the London Project to Cure Blindness to inquire about future trials outside of the UK.
Great news for amd suffers my dad is 89 years old and has it. It has impeded on every aspect of his life to the point that he is depressed has lost his confidence he has heard about this treatment and it has given him hope even though he thinks it might be to late for him but hopefully it will help others as there seems to be so many affected by it
I WISH YOUR WEB SITE WAS MORE READABLE FOR ME DUE TO THE FACT THAT I HAVE AMD IN BOTH EYES, BOLD TYPE WOULD BE A PLUS AND NO BLACK BACKGROUND IN YOUR WEB WOULD ALSO HELP ME, AND I WOIULD LOVE IT IF YOU PASS MY EMAIL ADDRESS TO WHOEVER IS DOING CLINICAL TRIALS ON DRY AMD. I JUST TURNED 70 OCT 23 , 2018 AND I AFRAID THAT IF I LOOSE MY EYESIGHT I WILL CERTAINLY TAKE THE POSITION THAT LIFE WOULD NOT BE WORTH LIVING WITHOUT SEEING. MY EMAIL IS SHEPBANK@GMAIL.COM OR SHEPBANK@HOTMAIL.COM…ANY ASSISTANCE IS MOST GREATFULLY APPRECIATED. SINCERELY LOUIS C. SHEPTIN
I have amd wet. is London doing stem cell treatment regularly ? can I go to London or California to get a treatment ? what would the cost be ?
Dear Maurice, there are several treatments already approved by the US FDA for the wet form of AMD. There’s no need to go to London for them, you can get them here in the US. Here’s a link to more information about them:
https://www.brightfocus.org/macular/wet-macular-degeneration/treatments
I hope that helps.
Yours truly,
Kevin
So I have a mother that is 91 and has macular degeneration. She is vibrant woman otherwise with excellent health.
I also have a significant other that is 68, which also has dry macular degeneration. He is an avid reader of the different studies on the subject. A person with lots of knowledge as a machinist but because of the degeneration it is difficult to continue his work and so he is retired. He lives near Chicago, a known city in the USA , so is there anything that he can do to be a part of any trials with this research?
slmh2@aol.com
Hello Sherry Lyn, sorry to hear about your mother and partner with MD. I can well imagine how challenging that must be. As a state agency we fund stem cell research but we don’t do any. To find out how you can be part of a clinical trial you have to contact the researchers/company directly. Only they can decide if you are eligible and able to take part.
Please send me he name of the research company. I have dry and wet macular degeneration.
Here are links to two clinical trials that might be appropriate for you. I do hope they help
https://clinicaltrials.gov/ct2/show/NCT02286089?
https://clinicaltrials.gov/ct2/show/NCT03178149?
My father would like to volunteer to have this treatment done to one of his eyes. How ever this process is done we are willing to try.
Please call me, my name is Laura, my cellphone number is 702 539 3488
Hi Laura, I’m afraid that clinical trial is not accepting patients right now, but this one might be:
https://clinicaltrials.gov/ct2/show/NCT04339764 If you just paste that link into your browser it will take you to a page on the http://www.clinicaltrials.gov website that has information about a trial that might be appropriate for your father.
Good luck.
I was diagnoised with maculate degeneration both eyes . pleasw help me,
I’m so sorry to hear about your diagnosis. I can only imagine how distressing that must be. I found one clinical trial for macular degeneration. Here’s a link to the page that has information about it. I do hope this helps: https://clinicaltrials.gov/ct2/show/NCT04339764
My mother suffers from sight loss with MD. She is 93 could she be in the trial for stem cell patch. I am sure she would be willing.
Hi Kathy, I’m afraid your mother would be considered too old for this trial. But there may be others in the future that she could be a candidate for.
My husband is 67 and has wet AMD that is progressing on treatment with aylvia and avastin. Will there be a stem cell trial opening in the US?
hello Beth, there are no clinical trials using stem cells for wet AMD that I know of. Most of the AMD clinical trials using stem cells are for the dry kind. I believe that’s because wet AMD already has some treatments that have been approved.