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From poster syncnode ICELL forum,
Dr. Steven Schwartz LA Times Interview/Comments
http://www.latimes.com/health/boostershots/la-heb-stem-cell-trial-ucla-20110714,0,4920946.story
By Daniela Hernandez, Los Angeles Times/For the Booster Shots blog
July 14, 2011, 2:01 p.m.
After more than 20 years of research, doctors at UCLA’s Jules Stein Eye Institute have begun treating the first patients in clinical trials for two progressive eye diseases that cause blindness: dry age-related macular degeration and Stargardt’s macular dystrophy.
The patients were given an injection of specialized eye cells that were derived from embryonic stem cells. Dr. Steven Schwartz, who is leading the trial at UCLA, performed both stem cell transplant surgeries Tuesday. The two patients are said to be recovering without complications.
According to Dr. Robert Lanza, chief scientific officer at Advanced Cell Technologies Inc., which developed the cells and is sponsoring the trials, “you could feel the excitement in the air and that history was being made.”
Surgery began for the first of two patients, a 77-year old woman with dry macular degeneration, around 9:30 a.m. Tuesday and took about half an hour. In the afternoon, a 27-year old woman with Stardgart’s macular dystrophy underwent the same procedure. Both patients are legally blind.
Doctors will monitor these two patients over the coming weeks. Another set of surgeries is scheduled to start in August.
Schwartz explained how these trials will help patients and what they mean for regenerative medicine. His comments were edited for space and clarity.
What are dry age-related macular degeneration and Stargardt’s macular dystrophy?
Twenty percent of age-related macular degeneration is wet macular degeneration and it is treatable. The other 80% of people have an untreatable, progressive visual loss leading to legal blindness called dry, or atrophic, macular degeneration.
The retinal cells, the rods and cones, and the underlying retinal pigment epithelium atrophy. As they atrophy, there is a long period of time when they are compromised and then they’re gone.
Stargardt’s is a genetic disorder and it strikes earlier in life. Patients start to notice visual changes as early as their teens and as late as their forties. There are a number of known genetic abnormalities in the photoreceptors that are toxic over time.
In this trial, what cells in the eye are you replacing with stem cells?
Advanced Cell Technologies Inc. has been able to take [embryonic] stem cells and differentiate them into highly functional retinal pigment epithelium that do everything they’re supposed to do. Our strategy of giving the eye brand-new, ready-to-go retinal pigment epithelium is designed for areas that are compromised, not for the areas that are gone. So we need to catch it early enough for this treatment to work.
Will the patients regain vision?
The patients’ central vision is already gone. Not rescuable. So the patients we’re enrolling in this trial know they will not be getting their central vision back.
If not to restore vision, what is the goal?
This is a safety trial. It’s not designed to improve vision. It may; and if we see a signal, that would be great news and we’re hoping we will. It’s plausible biologically, but that’s not what we’re looking for.
What results are you hoping to see?
I hope what happens is that we find this is safe and that we can optimize the dosing, and that allows us to move into eyes that are earlier in disease. That could have a real visual upside.
How long is the surgery?
Under an hour. It’s an outpatient procedure done with local anesthesia. It’s a surgery that we’ve done before – not with the injection of these stem cells, but we’ve accessed the eye before, and that’s one of the things that I’ve had a lot to do with surgically.
What does this trial mean for medicine?
We’re super-privileged to be taking this first step. It’s the unknown. These patients are doing a service for mankind. It’s inspirational.
Replies to a few questions,
"when is next financial filing due?"
Accelerated Filers:
10-Q: for Quarterly Period Ended 06/30/11 due Tuesday, August 09, 2011
"When are the next patients allowed in?"
The 2 patients that received injections on July 12 will be reviewed by DSMB at 6 weeks. How long review takes I don't know. DSMB review should take place around August 24.
"Patients will be enrolled sequentially, and within each cohort of 3 patients, each patient's clinical course over the first 6 weeks following cell transplantation will be reviewed by an independent (DSMB) before enrollment is opened for the next 2 patients"
http://clinicaltrials.gov/ct2/show/NCT01345006?term=%22Advanced+Cell+Technology%22&rank=1
" rock, I don't get what was injected. The MA09 RPE's are embryonic, right?"
No, terminally differentiated RPE's DERIVED from MA09 hESC cell line.
The paragraph from Caldwell below best defines what took place, the RPE's injected would be the "butterfly". Hope that helps you, it did me.
We develop a stem cell line. In the current example of the derived RPE cell line, the embryonic cell line utilized our single blastomere technology. It was one of the first lines we developed. We utilize Good Manufacturing Practices to develop what is called a “Master Bank” of human embryonic stem cells. These cells are characterized and tested to insure they are truly hESCs. You should know that one of the scientific tests to determine that the cells are hESCs, is whether they can produce a teratoma or benign tumor. ALL hESCs must be able to do this. We then develop a “working bank” of these cells. They in turn are differentiated into a particular cell type. In this case, RPE cells. We develop a “master bank” of RPE cells, again utilizing Good Manufacturing Practices in our lab in Worcester. That Master Bank of RPE cells is characterized, tested for purity and is worked to the point where it is “terminally differentiated”. Meaning that they can NO LONGER revert back to an embryonic state. The best example I can think of it that: It is now a butterfly and NOT a caterpillar. We test the RPE cells for purity and to insure that NO residual embryonic stem cells still exist. They must be 99+% pure with no lingering hESC’s, period!"
http://investorshub.advfn.com/boards/read_msg.aspx?message_id=53531326
mail ?,
"rock, how did Geron's pps react when they announced injection last year?"
Oct 13, 2010 6.31 6.40 5.96 6.04 5,054,300 6.04
Oct 12, 2010 5.88 6.38 5.78 6.37 9,169,300 6.37
Oct 11, 2010 5.52 5.74 5.50 5.67 3,349,100 5.67
Oct 08, 2010 5.27 5.37 5.22 5.33 583,600 5.33
http://finance.yahoo.com/q/hp?s=GERN&a=08&b=11&c=2010&d=10&e=11&f=2010&g=d
Geron anoounced injection on Oct,11,2010. In the 3 days of higher volume(in 3 days traded 18MM which was volume for entire month prior) not quite a 20% pps increase before decline. The $6.37 close is as high as GERN has traded since.
If by some miracle ACT would move identical we would need approx. 100MM in volume for 3 trading sessions including today and would top off at approx. .22. Would I count on this analogy? NO, I would hope for small gains that we can sustain which to date has been a problem.
As of May 31, 2011.....1,545,269,200 per S-8 filing on 6-20-2011
http://www.sec.gov/Archives/edgar/data/1140098/000114420411036482/v226244_s8.htm
ACT Announces First Patients Undergo Embryonic Stem Cell Transplantation Treatment for Stargardt's Disease and Macular Degeneration at UCLA's Jules Stein Eye Institute
Press Release Source: Advanced Cell Technology, Inc. On Thursday July 14, 2011, 8:30 am
MARLBOROUGH, Mass., July 14, 2011 /PRNewswire/ -- Advanced Cell Technology, Inc. ("ACT"; OTCBB: ACTC), a leader in the field of regenerative medicine, today announced the dosing of the first patients in each of its two Phase 1/2 clinical trials for Stargardt's macular dystrophy and dry age-related macular degeneration (dry AMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). The patients were treated Tuesday (July 12) by Steven Schwartz, M.D., Ahmanson Professor of Ophthalmology at the David Geffen School of Medicine at UCLA and retina division chief at UCLA's Jules Stein Eye Institute. Robert Lanza, M.D., chief scientific officer of ACT, attended the procedures. Both patients successfully underwent the outpatient transplantation surgeries and are recovering uneventfully.
Both the Stargardt's trial and the dry AMD trial will enroll 12 patients each, with cohorts of three patients each in an ascending dosage format. Both trials are prospective, open-label studies designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation into patients with Stargardt's and dry AMD at 12 months, the studies' primary endpoint.
"This first treatment milestone is welcomed by scientists, stem cell advocates and patients hoping for cures," said Gary Rabin, interim chairman and chief executive officer of ACT. "The two trials could not have started any smoother, and we are very pleased to announce that the procedures went well. The dosing of the first patients represents an important milestone for ACT and opens the doors to a potentially significant new therapeutic approach to treating the many forms of macular degeneration. We believe that these procedures represent a key step forward in therapeutic stem cell research, and the capacity to treat a variety of devastating diseases."
Dr. Schwartz, the studies' principal investigator, explained, "One patient in each clinical trial, the Stargardt's trial and the dry AMD trial, has undergone surgical transplantation of a relatively small dose (50,000 cells) of fully-differentiated retinal pigment epithelial (RPE) cells derived from human embryonic stem cells. Early indications are that the patients tolerated the surgical procedures well. The primary objective of these Phase 1/2 studies is to assess the safety and tolerability of these stem cell-derived transplants. We will be carefully monitoring our patients over the course of the trials. We are privileged to be collaborating with ACT and honored to be working with these pioneering patients."
Dry AMD, the most common form of macular degeneration, Stargardt's and other forms of atrophy-related macular degeneration are usually untreatable. Safe and effective therapies are greatly needed for the treatment of these common forms of blindness. Disease progression of both Stargardt's and dry AMD includes thinning of the layer of RPE cells in the patient's macula, the central portion of the retina and the anatomic location of central vision. With RPE cell death comes the loss of macular photoreceptors and loss of central vision. Currently both conditions are untreatable and often lead to legal blindness over a multi-year course. ACT's Stargardt's and dry AMD therapies treat these conditions by transplanting RPE cells in the patient's eyes before the RPE population is lost.
"Today –13 years after the discovery of human embryonic stem cells – the great promise of these cells is finally being put to the test," said Dr. Lanza. "The initiation of these two clinical trials marks an important turning point for the field. While we will continue writing research papers and carrying out more research, it's time to start moving these exciting new stem cell therapies out of the laboratory and into the clinic. Tens of thousands of people continue to die every day from diseases that could potentially be treated using stem cells. In the meantime, we intend to accelerate our efforts to translate new embryonic stem cell (ES) and induced pluripotent stem (iPS) cell therapies into the clinic. It has taken years of extensive research to get to this point. Our research and preclinical studies have demonstrated the safety and effectiveness of such therapies. We hope these cells may provide a treatment option not only for degenerative eye diseases, but for a wide spectrum of other debilitating conditions, ranging from diabetes to vascular and autoimmune diseases. Our team remains committed to moving the field of regenerative medicine forward from bench to bedside."
Further information about patient eligibility for the Stargardt's macular dystrophy and the dry AMD studies is also available on www.clinicaltrials.gov.
About hESC-RPE Cells
The retinal pigment epithelium (RPE) is a highly specialized tissue located between the choroids and the neural retina. RPE cells support, protect and provide nutrition for the light-sensitive photoreceptors. Human embryonic stem cells differentiate into any cell type, including RPE cells, -- and have a similar expression of RPE-specific genes compared to human RPE cells and demonstrate the full transition from the hESC state.
About SMD, Dry AMD and Degenerative Diseases of the Retina
Stargardt's macular dystrophy (SMD) is one of the most common forms of macular degeneration in the world. Stargardt's causes progressive vision loss, usually starting between 10 to 20 years of age. Eventually, blindness results from photoreceptor loss associated with degeneration in the pigmented layer of the retina, called the retinal pigment epithelium or RPE cell layer.
Degenerative diseases of the retina are among the most common causes of untreatable blindness in the world. As many as 30 million people in the United States and Europe suffer from macular degeneration, which represents a $25-30 billion worldwide market that has yet to be effectively addressed. Approximately 10% of people ages 66 to 74 will have symptoms of macular degeneration, the vast majority suffering from the "dry" form of AMD – which is currently untreatable. The prevalence increases to 30% in patients 75 to 85 years of age.
About Advanced Cell Technology, Inc.
Advanced Cell Technology, Inc. is a biotechnology company applying cellular technology in the field of regenerative medicine. For more information, visit http://www.advancedcell.com.
About the Jules Stein Eye Institute
Established in 1966, the Jules Stein Eye Institute represents the culmination of a dream shared by ophthalmologist, businessman and philanthropist Dr. Jules Stein and his wife, Doris, of creating a world-renowned center dedicated to the preservation of vision and the prevention of blindness. The Institute's comprehensive programs for the care of patients with eye disorders, research in the vision sciences, education in the field of ophthalmology and outreach to the community, coupled with its state-of-the art facilities, have brought national and international recognition to UCLA and the Institute, as it continues its mission to advance ophthalmology worldwide.
Forward-Looking Statements
Statements in this news release regarding future financial and operating results, future growth in research and development programs, potential applications of our technology, opportunities for the company and any other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words "will," "believes," "plans," "anticipates," "expects," "estimates," and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements, including: limited operating history, need for future capital, risks inherent in the development and commercialization of potential products, protection of our intellectual property, and economic conditions generally. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in the company's periodic reports, including the report on Form 10-K for the year ended December 31, 2010. Forward-looking statements are based on the beliefs, opinions, and expectations of the company's management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. Forward-looking statements are based on the beliefs, opinions, and expectations of the company's management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. There can be no assurance that the Company's clinical trials will be successful.
Contact:
Investors:
CEOcast, Inc., James Young, 212-732-4300
Press:
Russo Partners, Martina Schwarzkopf, Ph.D., martina.schwarzkopf@russopartnersllc.com, 212-845-4292 or 347-591-8785
Jules Stein Eye Institute, Elaine Schmidt, eschmidt@mednet.ucla.edu, 310-795-2272
RECEO,
I guess it all comes down to do you think they will RS and dilute in order to get to the finish line if the trials are successful?
That question goes way beyond a yes or no answer. "Finish line" to me is commercializing the product. ACT is hoping to partner up at some time during the clinical trials to take on the long and expensive route to market. What form or when a partnership will occur will be largely dependent on the level of clinical results needed for a partner to step up. Right now our cash on hand should pay operating costs and trial costs through year end. If no partner exists by then I would assume the $21MM available from Socius would be tapped. The RS scenario, imo, will only be used to move to large exchange.
elk,
When I read the PR from Roslin on the agreement, I came away with the following.
http://www.roslincells.com/sitepix/downloads/RC%20Pfizer%20PSC%20Line%20Evaluation_13Jul11.pdf
First, Pfizer is no stranger to the UK, they have many things going on there. Roslin Cell is proving itself to be a leader in this process and is even taking steps towards standardization which will be paramount going forward, imo, so Pfizers interest is warranted.
The agreement is for Roslin's Cell lines which are already being used in research programs. They also state in PR "Commercial terms are confidential and not disclosed."
The ACT lines will have to be processed and verified by Roslin as stated here, which will take some time.
http://www.roslincells.com/quality_control.asp
In summary, ACT will be involved with any commercialization of single blastomere lines. While Pfizer may show interest when they are available, this PR, at least for now is all Roslin/Pfizer..imo
RECEO,
The following link was posted recently as to my thoughts 16 months ago, not much has changed for me.
http://investorshub.advfn.com/boards/read_msg.aspx?message_id=47339667
I believe ACTC only as about 200M dilatable shares left
Actually, they have about HALF that many left to issue.
The following would be the common ways to correct the structuring:
1) buy back of shares...IMO, I do not see that happening.
2) increase the AS# and remain on the OTC:BB
3) reverse and increase AS# at the same time(hopefully only done if large exchange wqas the goal)
pps increase to $3-$5 at some point in time(imo won't happen prior to needing AS# increased) and move to large exchange
magtv and elk,
I believe all Roslin cells developed to date are hESC's?
http://www.roslincells.com/cell.asp
Secondly, they are pluripotent as will be the single blastomere lines developed through ACT. If the single blastomere lines NED 1-4 were not pluripotent why would the NIH be trying to include them in this definition?
Therefore, the NIH proposes replacing the current definition of hESCs in
Section II with the following: ‘‘For the purpose of these Guidelines, ‘human embryonic stem cells (hESCs)’ are pluripotent cells that are derived from early stage human embryos, up to and including the blastocyst stage, are capable of dividing without differentiating for a prolonged period in culture, and are known to develop into cells and tissues of the three primary germ layers.’’
Post from Kukkido,,ICELL,
Cha Biotech Meeting July 11, 2011
I met with Dr. Kwang Yul Cha, President and Chairman of Cha Biotech on July , 2011 with his assistant Ms. Yuna Lee at Chaum Medical in Gangnam-gu South Korea. Here are the questions and answers for our interview.
1. Is CHA using Frozen Embryos given to it from ACTC for its clinical trials.
Yes
2. Have they been delivered yet?
Yes
3. When will the injections start?
In two weeks.
4. When will there be initial results?
In 6 weeks if not sooner, we have to be very careful as the KFDA wants to proceed with Caution.
5. Why, does this have to do with the prior scandals dealing with the cloning results of Hwang Woo-suk. Yes, since these are the first results for Hesc everyone wants positive results and credible and honest trial documentation.
6. Are your safety and efficacy end points the same as ACTC?
Yes
7. Is Cha waiting for KFDA approval or has it only been approved by the Bio Ethics Committee.
Yes, at the same time.
8. Can Cha use the data from the US Trials to Help with trials in Korea?
Yes, but the KFDA wants its own data.
9. Can Cha get compassionate use in Korea?
Yes
10. If and when trial results are exceptional can Cha terminate and go to an open Phase III in Combination with Commercialization.
We believe we will have positive results and hope that we can move quickly through Phase I/II to provide important information so KFDA will allow us to move ahead to Phase III.
11. How long will that take, can you give us a timeline?
initial procedure will take 2 weeks to start injections. immunosuppresants are being administered now, the injections and initial results will be in about 6 weeks. After the 6 weeks we will have to reevaluate the stregnths and dosages to determine exactly what we are going to move ahead with. ie. larger amount of stem cells for the procedure per patient.
12. Will Cha apply for and NPP f0r Orphan Status and a compassionate use program.
Yes, we are talking to the KFDA about the ORphan Drug Status and will give there opinion on it by next week.
13. What about Off Label once approved?
That is our main thrust to get off the trials and move into compassionate use
http://investorstemcell.com/forum/advanced-cell-technology/3621-2.htm#post18205
interstate,
for some reason this topic always draws confusion. We were trading on the OTC:BB, we were delinquent in SEC filings. We obtained the "E" giving us time to file SEC documents. ACT did not file within the grace period and was not allowed to TRADE on the BB. Therefore they TRADED on the pinks until they became SEC compliant again and refiled to once again trade on the BB. At no time during this process were they a pinksheet stock, they always remained a SEC reporting stock. You cannot just quit being a SEC reporting company unless you file paperwork that meet exemptions from being SEC reporting.
Bottom line, ACT traded on the pinks during delinquency...that is it.
What is the difference between OTC, other-OTC and OTC Bulletin Board (OTCBB)? And where do the Pink Sheets fit in?
An over-the-counter (OTC) security is generally considered to be any equity security that is not listed on NASDAQ, NYSE or Amex. The OTCBB and the Pink Sheets are both quotation services for OTC securities. NASDAQ operates the OTCBB service and permits FINRA members to quote any OTC security that is current in certain required regulatory filings (see Listing Requirements). The Pink Sheets is a privately owned company that permits FINRA members to quote any OTC security and does not maintain regulatory filing requirements.
http://www.otcbb.com/faqs/otcbb_faq.stm
elichen,
What are your objections to a RS?
I don't have any objections. On the contrary, I was one of the first to support the idea of such on this board 16 months ago as link below will show. Many here do not support the idea. BTW, ACT is not a pinksheet stock and never has been.
http://investorshub.advfn.com/boards/read_msg.aspx?message_id=47339667
fordwill,
Question: can someone describe the process of such an injection for me?
I did find more info on the surgery process ford,
(from Edmund Mickunas, vice president of Advanced Cell Technology’s regulatory division)
"Treatment involves subretinal injection of RPE cells generated from human embryonic stem cells. Patients will first undergo a partial pars plana vitrectomy."
“Then, a very small needle is introduced to puncture the retina at a certain point,” Mr. Mickunas said. “A small hole is created. Then, a cannula is introduced and a bleb is created subretinally. Once that is done, then the cells are injected into that bleb.”
http://www.osnsupersite.com/view.aspx?rid=80795
________________________________________________________________________
partial pars plana vitrectomy
http://www.ehow.com/about_5182980_pars-plana-vitrectomy_.html
cannula
small flexible tube
bleb
blister-like fluid collection
thanks rumit, it is pretty well spelled out here if reading the scientific papers doesn't interest some.
Slide 15 and 17
http://www.advancedcell.com/documents/0000/0305/ACT_World_Stem_Cell_and_Regenerative_Medicine_Congress_2011.pdf
The following statement you just made ends the discussion for me because I know better. I don't want to play anymore.
My position has always been that unless someone with the expertise could show how we could realistically get our share price up to the lofty numbers folks were throwing around and thus get on a better exchange, even with good clinical news, given our number of OS, then a rs split seemed inevitable and necessary.
My position here on restructuring at the right time was documented long before your first post here. I have not changed my mind whatsoever.
So, what has changed your mind in the last 6 months? Were you not opposed to a reverse split then and just a couple months ago changed direction?
"My point is we're not going very far price wise, imo, as we hopefully mature to a company with a proven technology, if you will, without institutional following, ownership, sponsorship and all the benefits that come as a result of that."
btw, 1.2 Billion was volume for a month
spetty, we will have volume again but not sure as to what extent in comparison to 6 months ago. Clinical trials are a slow process, time will tell. We managed this kind of volume when we were in default, I expected better is all I am saying.
nmbr1stckpckr: Ok, I got it, it's all manipulation games AGAIN and we just wait for the $1-$10 push....no problem
farviewhill This stock has never had institutional buying to speak of and yet we did 1.2 B in volume in Dec, 6 months ago. I don't see your point?
I would like to cite again, my well worn mantra of the financial manipulative scum that are out there preying on these young biotechs through their manipulative action, including naked shorting.
This has been all well documented by reputable sources and there have been many convictions of guilty parties in the process, but no where enough, imo
That has been the endless mantra here for 5 years now. We could dilute another 1 Billion shares and it would still be the reason? It's a very small part of what is going on, imo...if there is a question to be asked at this time I feel the following is more appropriate...Where is the volume? Are the dirty scum buckets controlling that too? Nothing will happen here worth discussing until volume increases significantly, then the question will be as it always has been, will it sustain..Institutional interest isn't happening and won't under our current circumstances. If you folks think all those 100's of millions of discounted shares are all sold into the market, I would say think again. We have mega shares, we are on the OTC, we deal with it.
To those mailing about the "what's up with pps" questions, here is a post of mine from a week or so ago on icell that imo applies..
"This may be as good a time as any for my views on the pps issue. The next 2 weeks will mark the
2nd anniversary of our last Authorized share increase. In less than 2 years we have added 1 Billion
shares into the float with the majority of them discounted and many with major discounts. Think about that.
Example, we added about 9 times more shares than our competitor Gern has outstanding? No Company
I am aware of can dilute in those numbers and not have the same affect as ACT's pps. I view the duilution
as 90% of pps problem the last 5 years and the very reason sustaining increases was next to impossible
given the holders of debt shares would pounce everytime a pps pop leveled off.
The recent cleansing of CD debt has and will make a difference going forward. We chewed through many shares
on the last rise but it isn't over, imo, and the next pop or two will see pressure again. The second problem currently
is volume. Volume precedes price. About 6 months ago we had a December volume of 1.2 Billion shares,
http://www.otcbb.com/asp/tradeact_mv.asp?SearchBy=issue&Issue=ACTC&SortBy=volume&Month=12-1-2010&IMAGE1.x=21&IMAGE1.y=9
And on Dec 8, 2010 had a one day volume of 143MM shares
http://finance.yahoo.com/q/hp?s=ACTC.OB&a=11&b=1&c=2010&d=11&e=31&f=2010&g=d
So when we have 3-5MM share volume days I really don't understand what folks think is suppose to happen?
I envision the OS#/float to be a problem for some time. I know many think with Oct. progress reports we can
substantiate a pps high enough to make an exchange move, I am not in that camp and I hope if we can't make
the initial listing pps requirement that ACT restructures ACT in such a way we can move because I feel the OTC
and mega shares will continue to alienate the very institutions we need to get behind this stock.
a couple Phase1/2 definitions that may help,
1) Phase 1-2 includes both phase 1 and phase 2 procedures into a single trial. These studies are initiated when there is an expectation of less than usual short-term side effects and risks associated with the drug or biologic such as vaccine trials. Once the metabolic and pharmacologic actions of the drug and the side effects are determined then the trial moves directly into determining the effectiveness of the agent. As with Phase I and II studies, the participants are closely monitored and as with phase II trials involve several hundred participants.
http://www.genome.gov/Pages/Research...review_app.pdf
2) Phase I/II Trials: Phase I/II trials combine a Phase I and a Phase II trial of the same treatment into a single protocol. First the Phase I part of the trial is done, to determine the MTD. Then, more patients are treated at the MTD in the Phase II part, which follows immediately afterwards. The Phase I and Phase II parts are basically ordinary Phase I or Phase II trials, so you can analyze the trial as either a simple Phase I or Phase II trial, depending whether they've reached the Phase II part of the trial or are still in the Phase I part. The only way to find out is to talk to the investigators.
http://cancerguide.org/trials_phase1.html
Phase 1 is always first..SAFETY.... any early results will come from that Phase. One of the major reasons they chose Stargardt first.
ford,
Happy 4th of July weekend to all. In one year, we will have the data in from the 2 Phase I/II trials. I'm investing in this company like this therapy works. The Company will be either gearing up for a phase III trial or in a phase III
Rabin is hoping to start Phase 2 in early 2012 and you are saying Phase 2 completed by July next year and into Phase 3????
yep, 750K shares acquired through stock option..eom
nmbr1,
Wow! You're excitement actually showed thru in your post!
sorry about that..:) Another thought I posted on other board probably explains part of it..
"A side note to my last post and something I find "refreshing". ACT was mentioned only twice, as the sponsor(important) and in regards to where cells were manufactured. It signifies that ACT has handed in their term paper after 10 years of hard work from all their scientific people and now a group of independent professional individuals are basically in charge to make sure the best possible stage is set to evaluate many years of extensive studies and research. Hearing what the elite in the eye industry say as we move forward is the refreshing part for me as compared to just what an ACT PR says. The term paper is close to being graded, and that in itself is a major accomplishment."
dianne,
while the article contained a lot of info, I walked away with the following.
JSEI is utilizing all resources at their disposal understanding these trials can have a tremendous impact on future clinical eye regenerative medicine progress. To have Dr. Holland, founder of the Research Center,
and the Center operations person Dr. Gary Holland, monitoring the trials at all site locations signifies to me how important and how serious JSEI views these trials. JSEI, world-renowned clinic with exceptional resources at hand and most importantly all of them, even the higher ups from different divisions, advising each other and working as a team and admitting in this article "Everything just clicked. And it worked just the way universities are ideally supposed to work" is very important not only for the trials and results but should be a major assurance to all invested here that everything possible is being done to promote success. I applaud Dr. Steven Schwartz's recognition of all involved at JSEI and his disclosure of applying all resources available.
Article posted by Ceasar on ICELL,
UCLA Article Elaborates on SMD/Dry AMD Patient Prep and Trials
Jules Stein researchers begin first stem cell trials targeting eye diseases / UCLA Today
http://www.today.ucla.edu/portal/ut/jules-stein-researchers-begin-209350.aspx
Jun 29, 2011 By Cynthia Lee
Jules Stein researchers begin first stem cell trials targeting eye diseases
Researchers at the Jules Stein Eye Institute(JSEI) have begun two clinical trials to determine the safety of stem cell therapy and patients’ ability to tolerate it for the treatment of two common, currently untreatable degenerative eye diseases. This FDA-approved study is only the second in the United States to use human embryonic-derived stem cells in patients and the first to address eye diseases.
Twelve patients with the dry form of age-related macular degeneration (dry AMD) will be enrolled for one trial, and 12 with Stargardt’s macular dystrophy will be signed up for the other. While these patients still have some vision, all are legally blind. Currently, early participants are being screened by a battery of tests to ensure they don’t have other conditions, including cancer.
"There are substantial screening and health issues that have to be looked at before these patients undergo the surgery," said principal investigator Dr. Steven Schwartz, JSEI’s retinal division chief and Ahmanson Professor of Ophthalmology at the David Geffen School of Medicine. "It’s major eye surgery in which we will be putting retinal pigment epithelial (RPE) cells derived from human embryonic stem cells into a special place in the eye, called the subretinal space. It’s our hope that the procedure and the therapy turn out to be safe and that we can continue to move forward to learn more about this new therapy."
Researchers are hoping that one day stem cell therapy will transform the way doctors treat diseases — by replacing diseased cells with stem cells that have the singular ability to turn into heart cells, eye cells or any other that might be needed. Schwartz made clear, however, that no one, including the patients, has any expectations of significant visual improvement, although preclinical studies showed that eyesight among test subjects did improve substantially.
"Patients understand that there may be little or no benefit to them directly in terms of this procedure," Schwartz said, adding that there are risks, known and unknown, associated with any new therapy. "But these patients are all excited to do it because they want to open the door for this emerging field. They are true pioneers. The patients are the real heroes here." The study, which began last April, is slated to be completed by September 2013.
Help can’t come too soon for those suffering from these eye diseases. The dry form of macular degeneration is the most common form of the disease and the leading cause of blindness in the developed world, especially among people over the age of 55. The number of people affected by this disease is expected to double over the next 20 years as the population ages. In contrast, Stargardt’s Macular Dystrophy can affect those at the other end of the age spectrum, usually starting in children between 10-20 years old.
In both cases, the layer of RPE cells located beneath the retina deteriorates and atrophies. Over time, the death of these cells and the eventual loss of photoreceptors, or light-sensitive cells, in the eye can lead to blindness as central vision is gradually destroyed.
Surgeons will inject 50,000-plus new RPE cells into the patient’s subretinal space, the space where native RPE cells are found and an "immunoprivileged" part of the eye where an immune system attack is less likely. These factors, natural immune privilege and pinpointing the anatomic location, theoretically improve the cells’ chances of implanting and functioning. "This is well-studied, and we are taking a conservative, biologically plausible first step that is hopefully optimized for success." Schwartz said.
The eye is also a good candidate for stem cell therapy because researchers will be readily able to see what the RPE cells are doing and measure whether the transplant has an effect on eye functions, Schwartz explained.
The results of Schwartz’s research could have a profound impact on the future of the therapy. "Key learnings from this study may potentially lay the foundation for clinical regenerative medicine in the eye for decades to come," he said.
The sponsor of the tests, Advanced Cell Technology Inc. (ACT) of Marlborough, Mass., has been working on developing the therapy for the last decade. JSEI’s Clinical Research Center, conceived by Institute Chairman Dr. Bartly Mondino and run by Dr. Gary Holland, will monitor the trials taking place at UCLA and at four or five other collaborating eye institutes. The Casey Eye Institute at the Oregon Health and Science University in Portland, Ore., is currently enrolling patients, and other collaborating partners will be named later.
A look at photoreceptor cells, stained green, in the control group (right) which didn't receive the RPE treatment and the treated group (left). In preclinical trials, researchers found that the treatment slowed the progression of retinal dystrophy by helping the photoreceptors survive. (Courtesy of Advanced Cell Technology, Inc.)
"It’s because Jules Stein Eye Institute has the Clinical Research Center, thanks to the vision of Dr. Mondino, that’s allowing us to do this work," Schwartz said. "Another big factor is the research excellence of UCLA, which uniquely positions us to undertake these trials."
"We have great hopes for this," said Mondino, who added that no one can predict what will happen because this has never been tried before. "The Clinical Research Center has the infrastructure to support such a trial to optimize the possibility of success. We have the expertise of Dr. Schwartz and his team in the retinal division. We have the cells from ACT. I think we have the right combination here to ensure the best possible outcome."
Schwartz also applauded Dr. Owen Witte, head of UCLA’s regenerative medicine group, director of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Researchand the President's Chair of Developmental Immunology, and Dr. Donald Kohn, who runs UCLA’s GMP (good manufacturing practices) laboratory, for their assistance.
"They were both inspirational," Schwartz said. "Their leadership and advice have been invaluable."
The GMP laboratory is where the RPE cells created at ACT will go for final preparation before being given to patients, explained Kohn, whose own research involves using bone marrow stem cells in the treatment of certain blood diseases. "Since we have such a facility up and running, it allows UCLA to immediately move into this kind of pioneering trial," he said.
The trials’ high profile meant that the Clinical Research Center coordinated with not only the regenerative medicine company and the FDA, but with UCLA’s research infrastructure, including the Office of Research Administration, the Institutional Review Board as well as the Office of Contracts and Grants Administration.
"We were able to bring together vice chancellors and deans from the different programs, the University of California Office of the President, clinical administrators and coordinators in a productive, open, strategic and far-sighted way," Schwartz said. "Everything just clicked. And it worked just the way universities are ideally supposed to work.
"It’s an honor and a privilege to be doing this research and leading a team at a great university of this caliber. We just want to get it right, respect the patients and hopefully come up with key findings so that science and society can go forward," he said.
What you posted is for wetAMD. The Stargardt trial they have going on can be found here.
http://clinicaltrials.gov/ct2/show/NCT01367444?term=Stargardt&rank=2
June 28, 2011
Posted by Admin
Quick Update and Major Upcoming Milestones
Greetings,
We are very pleased to update you on our latest progress and some significant upcoming milestones.
As you may know, we recently announced that ACT was granted EU orphan medicinal product designation for our retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs) for the treatment of Stargardt’s Macular Dystrophy (SMD).
Additionally, we also announced that the first patients had been enrolled at the Jules Stein Eye Institute at UCLA for our two upcoming Phase 1/2 clinical trials using hESC-derived RPE cells for Stargardt’s disease and Dry Age-Related Macular Degeneration (Dry AMD). They are finishing the testing and pre-surgical preparations on these patients currently, and the surgeries are scheduled for the second week in July.
We hope you will continue to check in with us at this exciting time to learn more about ACT’s latest developments and our clinical progress.
Thank you.
Gary Rabin
Interim Chairman and CEO
Advanced Cell Technology, Inc.
rumit,
not sure.
I did find the MOU agreement which is basically a rehash of PR...
there is a download PDF you can click at this link
http://www.currentagreements.com/q/3MYv95gWkJvjrkeczVlIUU
Agreement has made it to the Roslin Cell website. Nice paragraph by Paul De Sosa, CSO of Roslin Cell.
(single blastomere lines: potential for more stable properties once expanded, genetically uniform and robust)
http://www.roslincells.com/sitepix/downloads/Press%20Release_Roslin_ACT_Blastomere_21Jun11.pdf
Paul De Sousa (Chief Scientific Officer)
Dr. Paul De Sousa has trained as a developmental and reproductive biologist, with a specific interest in mechanisms controlling the creation of developmentally competent eggs and embryos and cell regeneration. In 1998 he joined the Roslin Institute in Scotland where he began applying his basic research interests to animal biotechnology. In 2002 his group began focusing on the derivation of new human embryonic stem cells in accordance with emerging regulatory standards for therapeutic use. In 2005 he joined the University of Edinburgh as a Senior Research Fellow establishing a laboratory focused on the use of human embryonic stem cells in regenerative medicine and underpinning egg and embryo biotechnology. In concert with this he co-founded Roslin Cells Ltd.
louisa,
thanks, if others care to read the discussion on Orphan Status you can take a look here.
http://investorstemcell.com/forum/advanced-cell-technology/3662.htm
rumit,
good job no doubt..:) now where is the 8K for Roslin swami?
Orphan Status Granted in EU For Stargardt Disease,
ACT Granted EU Orphan Medicinal Product Designation for hESC-Derived RPE Cells for Treatment of Stargardt's Disease
http://www.prnewswire.com/news-releases/act-granted-eu-orphan-medicinal-product-designation-for-hesc-derived-rpe-cells-for-treatment-of-stargardts-disease-124587868.html
To All,
With all due respect, let's make this the final post on me. ACT has a hundred differnt things going on and this topic is a waste of time.
I am invested here because I have always believed in the Science of ACT. I was considered a basher here for years because I posted factual info on the horrific financing, court cases, OS# issue, gifted shares and many other things. I post ALL info relevant to ACT, that includes the good, the bad and the ugly. It is all important when you put your hard earned money down. I have never told anyone to buy, sell or hold although I have been asked a million times. A ton of info is posted here and other places so the decision what to do is yours, I honestly don't care. I have spent more hours than I am willing to admit providing as much pertinent info as I can. I have probably read and reread each filing many many times going back to 2005. So, I have a handle on things but certainly do not know it all, not by a longshot. I have never met any players at ACT and most certainly do not work for them and even the idea does not interest me. I am here because I see potential and have no problem sharing any info I find with you folks, good or bad. If some feel I have a secret agenda, so be it, but they could not be more wrong. There are many good folks here and at ICELL that I hope are rewarded by ACT's success. The best to all of you here and I really hope this issue is terminated..thanks
Jon, You asked if I believe the Roslin "definitive collaboration agreement" should 8K'd? IMO, yes, based on the following. If not under the 1.01 provision then under something like "other events". Agreements like this have always been disclosed to my knowledge..
Item 1.01 Entry into a Material Definitive Agreement.
(a)
If the registrant has entered into a material definitive agreement not made in the ordinary course of business of the registrant, or into any amendment of such agreement that is material to the registrant, disclose the following information:
(1)
the date on which the agreement was entered into or amended, the identity of the parties to the agreement or amendment and a brief description of any material relationship between the registrant or its affiliates and any of the parties, other than in respect of the material definitive agreement or amendment; and
(2) a brief description of the terms and conditions of the agreement or amendment that are material to the registrant.
(b)
For purposes of this Item 1.01, a material definitive agreement means an agreement that provides for obligations that are material to and enforceable against the registrant, or rights that are material to the registrant and enforceable by the registrant against one or more other parties to the agreement, in each case whether or not subject to conditions.
Instructions.
1.
Any material definitive agreement of the registrant not made in the ordinary course of the registrant’s business must be disclosed under this Item 1.01. An agreement is deemed to be not made in the ordinary course of a registrant’s business even if the agreement is such as ordinarily accompanies the kind of business conducted by the registrant if it involves the subject matter identified in Item 601(b)(10)(ii)(A) - (D) of Regulation S-K (17 CFR 229.601(b)(10)(ii)(A) - (D)). An agreement involving the subject matter identified in Item 601(b)(10)(iii)(A) or (B) need not be disclosed under this Item.
2.
A registrant must provide disclosure under this Item 1.01 if the registrant succeeds as a party to the agreement or amendment to the agreement by assumption or assignment (other than in connection with a merger or acquisition or similar transaction).
3.
With respect to asset-backed securities, as defined in Item 1101 of Regulation AB (17 CFR 229.1101), disclosure is required under this Item 1.01 regarding the entry into or an amendment to a definitive agreement that is material to the asset-backed securities transaction, even if the registrant is not a party to such agreement (e.g., a servicing agreement with a servicer contemplated by Item 1108(a)(3) of Regulation AB (17 CFR 229.1108(a)(3)).
"The retinal cells may have an advantage when it comes to immune rejection. The space beneath the retina where the cells are injected is generally free of the body's patrolling immune sentries. But in the patients in the trial, the RPE cells have been so damaged by disease that it's not clear whether they continue to maintain their immune-protected cocoon. So just to be safe, the volunteers will be taking drugs to suppress their immune system, in much the same way that patients receiving organ transplants from unrelated donors do."
http://investorshub.advfn.com/boards/read_msg.aspx?message_id=64342186
ford,
The following link has several videos that may put the subretinal injection into perspective. While I am sure these don't portray exactly what will transpire in our clinical trials I believe it won't be far from some of the procedures you can watch of several videos.
http://www.eyemoviepedia.com/videos/4166636950/67
rumit,
Your scenario is as good as any. I suspect it may be Pr'd tomorrow but who knows. Thank you for taking the time to post the Time Magazine photos. I have enjoyed working with you in several different areas of this investment and look forward to more of the same. Thanks again..
Hi Louisa,
In orignial post I had the wrong link to the following info, here is the corrected version. The site is a register of Orphan Products and lists the day it was designated such. I would assume a PR will be forthcoming...thanks
7th from bottom
EU/3/11/874
Human embryonic stem-cell-derived retinal pigment epithelial cells
Treatment of Stargardt’s disease
TMC Pharma Services Ltd
21/06/2011
http://ec.europa.eu/health/documents/community-register/html/orphreg.htm
ford,
I honestly do not know much more at this time regarding procedural steps...thanks