CIRM funded trial for AMD shows promising results

This upcoming July is healthy vision month, a time to remember the importance of making vision and eye health a priority. It’s also a time to think about the approximately 12 million people, 40 and over in the United States, that have a vision impairment. Vision can be something that many of us take for granted, but losing even a portion of it can have a profound impact on our everyday life. It can impact your ability to do everyday things, from basic hygiene routines and driving to hobbies such as reading, writing, or watching a film.

It is because of this that CIRM has made vision related problems a priority, providing over $69 million in funding for six clinical trials related to vision loss. There is reason to be hopeful as these trials have demonstrated promising results. One of these trials, conducted by Regenerative Patch Technologies LLC (RPT), announced today results from its CIRM funded clinical trial ($16.3 million) for advanced, dry age-related macular degeneration (AMD).

AMD is a progressive disease resulting in death of the retinal pigment epithelium (RPE), an area of the eye that plays a key role in maintaining vision. Damage to the RPE causes distortion to central vision and eventually leads to legal blindness. Thanks to CIRM funding, RPT and scientists at the University of Southern California (USC) and UC Santa Barbara (UCSB) are growing specialized RPE cells from human embryonic stem cells (hESCs), placing them on a single layer scaffold, and implanting the combination device in the back of the eye to try to reverse the blindness caused by AMD.

One of the trial participants is Anna Kuehl, a USC alumna and avid nature lover. She was diagnosed with AMD in her mid 30s and gradually began losing the central vision in her left eye. Although her peripheral vision remained intact, she could no longer make out people’s faces clearly, drive a car, or read the time on her watch. This also meant she would have much more difficulty going on the nature hikes she enjoys so much. After receiving treatment, she noticed improvements in her vision.

Anna was not alone in these improvements post treatment. The implant, known as CPCB-RPE1, was delivered to the worst eye of 15 patients with AMD. All treated eyes were legally-blind having a best corrected visual acuity (BCVA) of 20/200 or worse (20/20 indicates perfect vision).

Patients in the clinical trial were assessed for visual function and the results were as follows:

  • At an average of 34 months post-implantation (range 12-48 months), 27% (4/15) showed a greater than 5 letter improvement in BCVA and 33% (5/15) remained stable with a BCVA within 5 letters of baseline value. The improvements ranged from 7-15 letters or 1-3 lines on an eye chart.
  • In contrast, BCVA in the fellow, untreated eye declined by more than 5 letters (range 8-21 letters or 1-4 lines on an eye chart) in 80% (12/15) of subjects. There was no improvement in BCVA in the untreated eye of any subject. 
  • The implant was delivered safely and remained stably in place throughout the trial.
  • Refinements to the implantation procedure during the trial further improved its efficiency and safety profile.

In a news release from RPT, Mark Humayun, M.D., Ph.D., founder and co-owner of RPT, Director of the USC Ginsburg Institute for Biomedical Therapeutics and Co-Director of the USC Roski Eye Institute, Keck Medicine of USC, had this to say about the trial results.

“The improvements in best corrected visual acuity observed in some eyes receiving the implant are very promising, especially considering the very late stage of their disease. Improvements in visual acuity are exceedingly rare in geographic atrophy as demonstrated by the large decline in vision in many of the untreated eyes which also had disease. There are currently no approved therapies for this level of advanced dry age-related macular degeneration”. 

The full presentation can be found on RPT’s website linked here.

Watch the video below to learn more about Anna’s story.

13 thoughts on “CIRM funded trial for AMD shows promising results

  1. I’m AQP4 positive, NMO and lost my left eye vision in February 2021 as well as part of my right eye.

    Just like Candace Coffee, is there any steem cell or other experimental trial going on at the moment, if so I would gladly be a volunteer.

    Thank you for any info
    Josée Ouellet
    Québec, Canada

    • Dear Josee I’m so sorry to hear about your vision problems. I can only imagine how challenging that must be for you. I’m afraid I don’t know of any stem cell therapies that would be able to help you. I’m sorry I don’t have better news.

      • Thanks for taking the time to answer me. If you ever hear about any type of research/treatment please get in touch with me. I am open to try anything and travel the world to get a chance to recover part of my vision. Thanks again 🙂

  2. I have AMD in both eyes, dry in my left and wet in my right. I’ve been getting injections in my right eye since 2015 and I’m about blind in it. The left eye I still have good corrective vision but feel I only have 2 years at the most before I lose sight in it also. Any help for me?

    • Hey Matthew, there’s a lot of really encouraging research taking place right now, hopefully something will come along soon that can help you.

  3. My father is an active healthy 90 year old who has had for the past 5 years AMD in his left eye. No doctor can give us any information to get him into trials to due to his age.
    Any hope to get him into a trial study because age should not be an issue.

    • Hello Karen, I can only imagine how frustrating it must be not to be able to get your dad into a clinical trial because of his age. It seems like lots of clinical trials set age boundaries that really limit what people over the age of 80, or younger sometimes, can take part in. Hopefully if these therapies prove effective and safe those boundaries will be lifted.

  4. Hi, I have tried to contact via email & communication on posts & haven’t any luck so far.
    I’m hoping to find a way for my son for stem cell
    Hello again! I’m hoping to find stem cell research/trials/ cure for my son who has SC which is Serpigenious Choridopathy.
    Are there any trials for stem cell that would be willing to help with SC?
    We live in Florida however, my son will be in California around the 18th for an appointment at UCLA for stem cell.
    Maybe an appointment for possible stem cell trials would be available while my son is in California ?
    We look forward to your reply, thank you, Debbie Kelso mother for Chris Cardman.

      • Thank you so much for your reply! PLEASE remember my son should something come up. Trials, stem cell etc! Thank you, Chris’s mother Debbie Kelso 

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