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Stem Cells in Ophthalmology Update 13: Advanced Cell Technology Update

Posted Jan 10 2012 5:28pm
In the wake of the 60 Minutes expose of illegitimate stem cell activities, I thought I would bring you good news about a couple of  legitimate, government approved clinical trials using stem cells.

As part of the Biotech Showcase 2012 conference program, being held in San Francisco, ACT company chairman and CEO, Gary Rabin will present talks on his company’s progress as part of two panels at the Regenerative Medicine State of the Industry Briefing. In advance of his two talks, the company released a statement about the ongoing clinical trials, results and timing, on his From the Chairman company blog.

In his statement, reproduced below, Rabin commented on the progress of two of the three government approved clinical trials currently underway at UCLA’s Jules Stein Eye Institute on treating Stargardt’s Macular Dystrophy and the dry form of age-related macular degeneration (Dry AMD). (See Update 8 and Update 9 for additional information.) (The other clinical trial, also for treating Stargardt’s, is taking place in the UK at Moorfields Eye Hospital in London.[ Update 10 ])
As I reported last July, in Update 9 , the first two patients in each of the UCLA trials were treated on July 12th. Since these are safety studies, the investigators are carefully watching the first patients response to the treatment before treating other patients in the twelve patient trials.
Rabin stated, “As you are no doubt aware, the trials at UCLA are being conducted by Dr. Steven Schwartz of JSEI and overseen by our chief scientific officer, Dr. Robert Lanza. Each patient has received an injection of 50,000 hESC-derived RPE cells in one eye. Both trials will involve twelve patients, and are designed to evaluate the safety and tolerability of the injected RPE cells. Based on the results of the first patient in each study, we are authorized by the Data and Safety Monitoring Board (DSMB) to move forward with the next two patients in the studies, each of whom will also be treated with 50,000 RPE cells.” (Emphasis added by editor.)
Rabin went on to say, “I am delighted to inform you that we are currently scheduled to treat our first patient in the UK at the end of next week or early the following week, and that we will be treating four additional US patients beginning that same following week.”
As for publishing the results of the studies, he said that the company would take the appropriate approach of “publishing it in the form of a paper in a prestigious, peer-reviewed medical journal. However, the peer-review process takes time. The process typically takes several months, so I am actually quite thrilled that we are now moving toward the final stages of completing it, less than six months after the first patients were treated. We are far enough along that at this point the additional waiting time will be measured only in weeks, not months.”
So, this is good news for the cell stem treatment of retinal diseases.
I have also just learned about several other clinical studies now underway in this field and will discuss them in a followup report ( Update 14 ) to be published either later today or early tomorrow.
Now, here is the complete statement from Mr. Rabin, as taken from his company’s website:

January 10, 2012
Clinical Trials, Results, and Timing
Greetings,
For many of us, the New Year is a time not just for looking forward but for reflecting on events, achievements and lessons learned over the past year. I anticipate an amazing year ahead for ACT, and at the same time I also cannot help but reflect with pride on how far the company has come with its clinical programs over the past year.
IND Filing and Clinical Trials
I will never forget the moment I learned that the company's Investigational New Drug Application (IND) for its human clinical trial for Stargardt's Macular Dystrophy (SMD) had been approved by the FDA. It was clear then that ACT truly was on the road to potentially making medical history. So much has happened in the interim that it is hard to believe that happened only a bit over one year ago, in late November, 2010!
Shortly after that, our IND for Dry Age Related Macular Degeneration (Dry AMD) was also approved. The brief time since then has been a whirlwind of activity in preparation for the clinical trials, and we were enormously pleased and proud to start them in July, at the first site, UCLA's Jules Stein Eye Institute (JSEI).
As you are no doubt aware, the trials at UCLA are being conducted by Dr. Steven Schwartz of JSEI and overseen by our chief scientific officer, Dr. Robert Lanza. Each patient has received an injection of 50,000 hESC-derived RPE cells in one eye. Both trials will involve twelve patients, and are designed to evaluate the safety and tolerability of the injected RPE cells. Based on the results of the first patient in each study, we are authorized by the Data and Safety Monitoring Board (DSMB) to move forward with the next two patients in the studies, each of whom will also be treated with 50,000 RPE cells.
Part of what makes research and development in the regenerative medicine sector so exciting is that it involves sailing into largely uncharted waters. ACT's two trials are the only ongoing human embryonic stem cell-based trials, period. We are quite literally creating a new area of medicine. This means there is tremendous pressure on us to "get it right." The responsibility to provide the first-ever validation for this enormously promising new sector rests entirely on our shoulders.
I hope, then, that our many fans and followers can understand why this process takes some time. All eyes are on us, both from the standpoint of support and scrutiny. Should our trials succeed, it could provide the validation that the regenerative medicine sector has been in need of for some time. This is why, in every stage, we are bending over backwards to make sure we cross all our t's and dot all our i's. As the saying goes, Rome wasn't built in a day. If we can successfully complete these trials and bring these therapies to market, though, the potential benefits would be manifold:
*  The potential to at least partially restore sight to millions of people suffering from Dry AMD, the most common cause of blindness for people over age 55.*  Provide a much-needed validation to the entire regenerative medicine sector.*  Provide an enormously useful base of scientific knowledge on which we and others can develop other treatments and cures.*  Last but not least, reward our investors for their patience and support with a return on their investment as befits a company with the only approved treatment for Dry AMD, which has a potential market size of $25-30 Billion in the US and Europe alone, as well as for SMD.
For many years, I was an equity market investment manager. That is an industry where you can evaluate results on a daily basis. Just because you can do that, though, does not mean that you should. The best investors, by far, look at long-term investment opportunities. Running a biotech company is not the same. I am well aware that we have many shareholders who want to know why we don't just treat patients and release results as fast we can, and as fast as available. To do so would be beyond foolhardy for a company like ACT. If there is anything this industry has had some issues with, it is credibility in the mainstream healthcare world. We plan to change that. But to do that, it can't happen overnight. Patient selection, clinical site selection, the timing of patient treatment, and unexpected non-ocular conditions of patients found in health screenings are among the major factors that impact patient treatment. Another factor in the timing of treating these new patients is that we will have at our disposal a new kind of three-dimensional retinal imaging technique, which has not previously been available. Believe me, the timing of patient surgeries has nothing to do with safety, efficacy or availability of suitable patients.
I know that many investors want us to go as fast as possible in treating patients. But there is a tortoise/hare effect here that I simply won't discuss now. I know that it is hard to be patient, but we are making every decision for the best interest of the company in the long run. We don't get bonus points for finishing the trial a few months earlier as compared to making it a truly game-changing medical opportunity. I know that many of you don't know me from Adam, but I'm a very methodical person. Look who we recently added to the Board - one of the leading scientists in the world; one of the best entrepreneurs in the world (founder of Life Alert and eFax), and the CFO of a highly-regarded biotech company considered to have made excellent, value-preserving large bio/pharma partnering deals. We have this under control.
One ill-conceived decision could set the company on a downward path (ACT has been there). Highly prestigious peer-reviewed medical journals do substantial review and due diligence. Top-rated eye hospitals and surgeons are very process-oriented and sometimes bureaucratic. Pushing them harder to move faster doesn't earn you any credibility or success.
Nevertheless, I am delighted to inform you that we are currently scheduled to treat our first patient in the UK at the end of next week or early the following week, and that we will be treating four additional US patients beginning that same following week.
Publishing Data
The question that inevitably comes up asks when we are going to publish the initial data from the trials. We are eagerly anticipating doing so but we are not going to just post it in raw form. This trial has the potential to make medical history and we want to share the initial results with the world in a strategic way.
The only appropriate approach with data this significant is publishing it in the form of a paper in a prestigious, peer-reviewed medical journal. However, the peer-review process takes time. The process typically takes several months, so I am actually quite thrilled that we are now moving toward the final stages of completing it, less than six months after the first patients were treated. We are far enough along that at this point the additional waiting time will be measured only in weeks, not months. We are coordinating the scientific publication with a general mainstream media release strategy (see below). When the paper is published, rest assured that we plan to leverage it to make sure it is very broadly known, not only in medicine, but in the broader medical and scientific community, as well as the investment community.
We sincerely appreciate everyone's patience as we continue this process. We are keenly aware that our investors, fans and other followers are anxious to see the data.
Thank you for your patience and thank you, as always, for your interest and support.
Gary RabinChairman and CEOAdvanced Cell Technology, Inc.
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