Thursday, August 02, 2012

Stem Cells in Ophthalmology Update 22: A Stargardt’s Clinical Trial Patient’s Story – In Her Own Words

Back a few months ago, I got an inquiry about my tables of clinical trials published in my online Journal. I responded to the woman inquirer and found out that she was considering participating in a clinical trial. The inquirer was Maurie Hill. She has Stargardt’s disease.

We began corresponding and she told me that she was planning on going to the Wills Eye Institute in Philadelphia to be screened for the Advanced Cell Technology’s ongoing stem cell clinical trial. She was screened and accepted into the program and became the sixth patient treated, and the first of the second cohort, treated with 100,000 RPE (retinal pigment epithelial) cells derived from human embryonic stem cells (hESCs).

A little information about Maurie. She is 52, married, has a 9 year-old daughter, and lives in Vermont. She has an Associates degree in Electronics and Engineering Technology and works (and writes) for a company, Ai Squared,  involved in providing technology for the visually disabled.

Interestingly, she is one of six siblings, two others of whom also have Stargardt’s disease.

When Maurie told me that she had been accepted into the clinical trial, I suggested that she keep notes and write about her experiences. She thought that would be a good idea and here, in her own words, is the first entry in her diary.

I'm in the ACT Stem Cell Trial for Stargardt’s Disease!

by: Maurie Hill on July 31, 2012

Picture of Maurie after the surgery

My last article highlighted my experience at the Visions 2012 conference and the closing session's three main speakers. They spoke about some of the ongoing human clinical trials exhibiting great promise for battling retinal degenerative diseases. One of those speakers, Matthew Vincent, Director of Business Development at Advanced Cell Technology (ACT), spoke effectively about the current stem cell trials for both Stargardt disease and AMD. And even more recently, ACT announced that they treated their fourth (U.S) Stargardt patient, the first patient to receive a higher RPE cell concentration in their worst eye. I am excited to inform you that I am this patient! I was treated on July 11th at Wills Eye Institute in Philadelphia. It was an exciting experience that I'll never forget; physically joining the team within the many teams that have worked so hard to bring us to this point in medical history.

Well there's a lot to catch you up on but first I will tell you that the surgery itself, performed by Dr. Carl Regillo, went according to plan. Within a week, the doctors could no longer tell that my eye had been physically intruded upon using a common procedure called a vitrectomy. This procedure was used to remove vitreous fluid and replace it with something unusual - RPE cells derived from just one donated human embryonic stem cell (hESC). After two weeks, my vision appears to be the same as it was before surgery, which is expected at this point.

Let's back up a bit.  After seeing interviews of the first two patients in this trial on the CBS Morning News and listening to them on NPR, I wanted to know more. The Stargardt patient, who understandably wished to remain anonymous, could now read five letters on the ETDRS visual acuity chart. But what did she see in those letters beforehand? Could she see part of the letters clearly using a small remaining portion of central vision? Did the bold letters appear gray because her vision was cloudy, or did the letters appear to be completely missing from the page, as if she were looking at a white sheet of paper?

Why does that matter? Since Stargardt’s Disease is a slowly progressive disease, I wondered how many years ago was it that she could actually distinguish those letters. In other words, how many years did this procedure effectively reverse? Since these new healthy RPE cells theoretically take over the job for your current genetically deprived RPE cells in the eye, they should be "munching up" the debris that naturally flakes off your photoreceptor cells every day,.so what functional aspect of vision comes back first? Does what was lost last come back first? Though I still had nearly 20/20 vision at age 20, my ability to scan across text quickly (aka speed read) was noticeably poor even at age 10. And my ability to quickly adapt from dark to light and recognize faces was compromised by age 30 although my vision was still better than 20/100.

It seemed the only way to get the answers was to try this myself. Easier said than done; only 12 participants would be included in this Phase I/II trial. I really had no illusions that I would be accepted into this trial but wanted to go through the motions and see how far it went in order to blog about the process.

The first thing I did was visit and search for all the current trials for Stargardt Disease. I found the ACT trial and read the eligibility, inclusion, and exclusion criteria.  I didn't want to waste anyone's time but I didn't see anything that would exclude me. I showed the study to my ophthalmologist at my yearly visit and he agreed. I had the closest participating institute fax the required form to his office. There was a lot more to it, but that got the ball rolling. Through a series of luck, timing, and hard work, the stars aligned perfectly, and I overcame what appeared at first to be the logistical nightmare of my life.

But a nightmare it was not. I am a procrastinator at heart, but untrue to form, I was totally ready and prepared for this a full week or so before it all happened. Though in retrospect, I think I had been preparing for this since 1995 when I first read about the probable cause of my failing vision - poorly equipped RPE cells in the RPE layer.

I received twice as many RPE cells (100,000 to be exact) as the first three Stargardt participants.  Amateur logic tells me that I should experience improvement in half the time. But then I remembered there are millions of photoreceptor cells that take residence in the RPE layer - will 50,000 extra fresh RPE cells really make a difference, or could the excess baggage actually have a negative effect? My positively foolish self is truly looking forward to this adventure of not knowing what's next. Just like when I ran high school cross country, I never liked to run the race course beforehand because I didn't want to know how far I had left. This time around is no different; I'm going to experience every turn for the first time. The only difference is this time, I'm taking you along for the ride.

As you can see, I’m a risk taker. And as such, I have purchased a small amount of ACT stock- but it’s my vision I’m betting on and would never let greed override my responsibility to tell the truth about my clinical trial experience. I don’t have a clue as to whether this is a good investment or not, even if my vision does improve, as this is a very early stage of the clinical trial process.

Editor’s Note: As this blog entry was in preparation, Advanced Cell Technology announced that their eleventh clinical trial patient had been treated, this one the fourth patient in their dry AMD clinical trail, and also the first of the second cohort (with 100K RPE cells) in that trial. The patient was treated by Dr. Regillo at Wills Eye Institute.


At 5:57 PM, Blogger DMSOB said...

What a great story!

Thank you both, Irv and Maurie!

At 1:12 AM, Blogger KyleD said...

Please keep sharing everything you learn and will be the eyes for so many of us! Thank you for all you are doing.

At 1:13 AM, Blogger KyleD said...

Please continue to share your experiences and are the eyes for so many of us! Thank you and God Bless You and the clinicians!

At 8:03 AM, Anonymous Anonymous said...

Best wishes.

Being a trial patient and a share holder of the very company that treat your eye, does it conflict of interest?


At 3:57 PM, Anonymous Anonymous said...

You are courageous to venture into the unknown. People like myself, with a 38 year daughter with Stargardt's, will be forever grateful for those of you who took the first step and had the injections. I too owe the stock, and hope and pray that my investment will pay for my daughters treatment made possible by people like you, and the scientists at ACTC and the doctors doing the procedures. Here's hoping for sight for you.

At 7:54 AM, Anonymous Anonymous said...

The beginning of the stem cell revolution .... Simply amazing. Let's hope it I for real this time.

At 8:50 AM, Anonymous Anonymous said...

Thanks for sharing your story. I have had Stargardts for over 15 years, (54 years old, late diagnosis) & would love to be a participant in the clinical trial. When I fist heard about ACTC & the upcoming trials, I also purchased some of the ACTC stock. We can only hope & pray that this will benefit millions with their work on Stargardts, AMD & other visual diseases. I pray your sight is restored.

At 10:22 AM, Anonymous Anonymous said...

I too own the stock and am hoping for success with that, but more importantly, I am hoping and praying that you see better again! Vison loss must be very troubling for a vibrant mother of a 9 year old. Here's to your good vision. Thank you so much for sharing your story with us.

At 7:04 PM, Anonymous Anonymous said...

Wonderful information stream. This gene therapy is very, very important to first of all prove that it is a safe treatment. It is ground breaking, in that it will open the door a bit quicker & wider for many other genetic diseases. I also look forward to the gene silencing work to go to clinicals by Irish firm Genable, in their attempt to attack adRP.


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