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mvecho,
The NIH "redefinition" would allow 5 single blastomere lines to be eligible for FED funding. Thsi proposal has been bogged down with lawsuit issues and still remains in limbo. Article below describes quite well.
WASHINGTON - The National Institute of Health's proposal to revise its definition of human embryonic stem cells (hESC) to permit earlier stage embryos may at first glance appear to be a minor technical change. But to firms like Advanced Cell Technology Inc., which has sunk a "fortune" into developing the cell lines, it is "hugely important," said Robert Lanza, chief scientific officer for the Worcester, Mass.-based biotech.
The NIH late Friday afternoon proposed changing the definition of hESCs issued in guidelines last July from "cells that are derived from the inner cell mass of blastocyst stage human embryos" to "pluripotent cells that are derived from early stage human embryos, up to and including the blastocyst stage." (See BioWorld Today, July 7, 2009, and Feb. 22, 2010.)
That change would allow ACT's five single-blastomere lines currently under review at the NIH to receive federal funding for research, if approved by the agency. ACT currently has an investigational new drug application (IND) under review at the FDA for a Phase I/II trial using its MA09 single-blastomere line to treat Stargardt disease, a genetic condition and the leading cause of juvenile blindness in the U.S., Lanza told BioWorld Today.
The IND currently is on hold while ACT addresses some of the FDA's questions, but Lanza said the company is hoping to initiate the study by the end of the third quarter.
The hESC definition change also would affect ACT's four other NED, or "no embryo destruction," lines awaiting placement on the NIH registry: NED1, NED2, NED3 and NED4.
ACT's approach, Lanza said, is "simply plucking one cell out of the embryo in a way that doesn't harm the embryo and then you can create a line from that cell."
The MA09 line is the one the firm has been using for its "master bank" for its clinical trials, he noted. "What we have found, interestingly, is that these lines have opportunities superior to the other embryonic stem cell lines that we have been studying," he said. ACT's five lines affected by the NIH's proposed change were generated under good manufacturing practice conditions, "so that they are suitable for use in patients," Lanza said. "It turns out that when we look at embryonic stem cells side by side, that our best performers, the cells that do some of these tricks most efficiently, actually were the single-cell derived embryos," he said. "So if we want to make various endothelial cells or vascular cells to repair damage, we find that the single blastomere lines, at least one or two of them, are five times greater in efficiently generating the cells. That is huge when you talking about having to expand cells into very large numbers for medical therapy."
Lanza insisted that "it shouldn't really matter what cells" hESC lines are derived from.
"These are coming from the spirit of the law that these are leftover embryos with the proper consent forms that were discarded from IVF clinics or from leftover embryos that were not being used," he said. "So whether or not you generate your lines from this or that cell from the embryo really shouldn't matter," Lanza said.
And because the single blastomere method does not require the destruction of the embryo, "it could be strongly argued that these lines are far more ethical," he said.
Given the "many years" of data ACT has collected, including lifetime animal studies to show the cells do not form teratomas, it would have been "a real shame" if the NIH had ignored the pleas from the company and other researchers to revise the guidelines, Lanza said.
"You can imagine changing the official guidelines is not trivial," he said. "So my hat is off to them for correcting this. They are doing the right thing here. We are very pleased that they were willing to make this correction."
Lanza noted that his firm and its collaborators have applied for several NIH grants, which currently are on hold until ACT's lines are approved.
The company's efforts in winning federal funding, he said, "got caught" in President Bush's 2001 order that restricted federal funding for hESC research, which was lifted last March by President Obama. (See BioWorld Today, March 10, 2009, and March 11, 2009.)
The firm had a solicitation at the Department of Defense for $5.6 million to create universal blood, but none of the Bush lines were O negative, and ACT was unable to get the White House to sign off on permitting the use of the company's single blastomere technique.
"They let the clock run out on it," Lanza said. If the NIH revises it guidelines and approves ACT's five single-blastomere lines, it would open the possibility that the firm could potentially get the DoD to reconsider, he added.
Meanwhile, the company is in discussions with the Foundation for Fighting Blindness for appropriated funding from DoD to back ACT's clinical trial.
"There are many, many people who could greatly benefit from this technology," Lanza said. "It would be very sad for us not to be able to move ahead and try to help as many people as possible."
The NIH proposal is open for public comment for 30 days
102nd Meeting of the Advisory Committee to the Director (ACD)
National Institutes of Health (NIH)
http://acd.od.nih.gov/ACDJune2011Agenda.pdf
As reported yesterday, no "redefinition" discussion this time around.
what options can be exercised?..eom
E-mailed this morning on whether redefinition will be part of June 9-10 Advisory Meeting, it will not be.
Gretchen S. Wood
Immediate Office of the Director
National Institutes of Health
One Center Drive, Room 114
Bethesda, Maryland 20892-0147
NIH - About NIH - Advisory Committee to the Director
Dear Rocky
I am hoping to finalize this today and have it posted on the ACD website. While there is a Stem Cell Working Group report on the agenda for Day 1, there will not be any discussion on the redefinition. I am sure you have already visited our stem cell site, but here is the link should you need it: NIH Stem Cell Information Home Page .
Best wishes,
Gretchen
equity,
pay attention to insider buys
That will be easy, there has never been any.
SEC Form 4 Filed,
Rabin 750K sell @ .2007 on May 20. He seems to be having a difficult time filing on time.
http://www.sec.gov/Archives/edgar/data/1140098/000114420411033994/xslF345X03/v224879_ex.xml
elk,
yep, that was what rumit was getting at.
http://investorshub.advfn.com/boards/read_msg.aspx?message_id=63735701
Patent issued right on time,
doubt it will make a PR and it is not a material event.
http://investorshub.advfn.com/boards/read_msg.aspx?message_id=63116416
United States Patent & Trademark Office
application number 10/374,512
GYNOGENETIC OR ANDROGENETIC PRODUCTION OF PLURIPOTENT CELLS AND CELL LINES, AND USE THEREOF TO PRODUCE DIFFERENTIATED CELLS AND TISSUES
Patent Issue Date Used in PTA Calculation 5-31-2011
RECEO,
Let me know if this is what you were asking and if it answers your question..welcome to the board
(slide 11 at link below)
Established GMP-compliant process for the Reproducible Differentiation
and Purification of RPE cells.
– Virtually unlimited supply of cells
– Can be derived under GMP conditions pathogen-free
– Can be produced with minimal batch-to-batch variation
– Can be thoroughly characterized to ensure optimal performance
– Molecular characterization studies reveal similar expression of RPE-specific genes to controls
and demonstrates the full transition from the hESC state.
Manufacturing
Ideal Cell Therapy Product
• Centralized Manufacturing
• Small Doses that can be Frozen and Shipped
• Ease-of-Handling by Doctor
http://www.advancedcell.com/documents/0000/0305/ACT_World_Stem_Cell_and_Regenerative_Medicine_Congress_2011.pdf
copper,
It seems to me that at that juncture -- when you have an approved treatment, we would need the support of a major pharma to get the treatment out on a scale commensurate with demand
NO doubt about that.
(recent PharmaTV Interview)
Gary Rabin:
"Well right now we obviously are deficit funding ourselves through our own capital, ultimately we enforce plan to commercialize this product probably in partnership with a large pharma or a biotech that has penetration into the ophthalmology surgeon market and the therapy that we are doing is the subretinal injection 50 to 200,000 RPE cells suspended in ceiling 150 micrometer of ceiling, so this is a very easily scalable, transportable model that looks very much like a traditional pharma product or a biologic."
http://investorshub.advfn.com/boards/read_msg.aspx?message_id=62474597
(conference call)
I just returned from Europe where we had some great meetings. As far as the RPE program goes, it would be highly premature to seek partners early in the phase 1 study as we are hopeful that even though we are dealing with late stage patients, we will soon have data on retinal engraftment of our cells, and thier impact on photorecptor function. If we see no safety issues, and are able to detect some engraftment of our cells to the photoreceptor / Bruch's membrane, we will obviously be dealing from a much stronger valuation and negotiating position.
copper thorn,
Welcome to the board.
You asked,
"How much training and equipment would an opthamologist need to administer this treatment?"
In the scheme of the clinical trial, I view this as probably the least to be concerned with. Below are a few blurbs from the past.
"Finally, the mechanism of application is a needle. Unlike most appliance applicators, it does NOT have to be approved by the FDA. A needle is recognized by the FDA as a standard mechanism of application and even more importantly every eye surgeon utilizes a needle in surgical procedures dealing with the eye. Therefore, you don’t have to teach the “old dog new tricks” which is a huge barrier when normally introducing new therapies utilizing a delivery device."
http://investorshub.advfn.com/boards/read_msg.aspx?message_id=41946204
In our particular situation, we are inserting a needle into the eye- which is something that is done all the time, by the way, this isn’t something that’s foreign to the practitioner that does the application- but our application takes place only once or possibly twice over the life of the patient. It is our expectation that the therapy which we’ll apply will have an impact on either slowing down or arresting the progression of the disease. We’ve seen that in our animal models. There have been some very dramatic results when we’ve applied it into animals and we are extremely hopeful that we will see the same types of results when we apply it into humans.
http://investorshub.advfn.com/boards/read_msg.aspx?message_id=57868481
interstate,
good post. Minor correction, it is 6 weeks after 1st patient and 4 weeks after 3rd patient. Thanks again
dryAMD:
"Six weeks after the first patient in each dose cohort receives the cell transplant, the DSMB will review the clinical data and recommend if the next two patients in the dose cohort may be treated. Each cohort of 3 patients will be reviewed by the DSMB when the 3rd patient completes 4 weeks of follow-up.
SMD:
"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. A full safety assessment of all 3 patients in each cohort will be made by the DSMB when the 3rd patient in each cohort completes 4 weeks of follow-up, and before the first patient in the next cohort receives a cell transplant."
Twaro and all,
You are absolutely correct Twaro in that the e-mail discussion I had with Maureen from OHSU did not in any fashion represent a delay in the start of trials. If by chance a delay would happen, it certainly isn't because of what she said. Jules stein came on board for both trials 18 days prior to OHSU. Jules Stein would have needed training meetings also with the same group that needs to go to OHSU. Thinking that both clinical trial sites would be in exactly the same stage of the process would be a mistake. If anything was to be recognized(as new) from e-mail exchange I had, I felt it was the fact that OHSU is seeking IRB approval and dealing with contract to become a trial site for dryAMD. That was my original question to her and was glad to hear the response. I followed up with questions about the recruitment for Stargardt and was not surprised at the stage they were in given they have only been approved and on clinicaltrials.gov for 10 days.
To grasp at any straws from that exchange is unwarranted,imo. There is much more to these trials than most think. I have not heard anything from Jules Stein or anyone else to indicate a problem or delay, fwiw.
euro,
I will respond the same as I did on investorcell board..
"I envision all clinical trials as being akin to watching a snail race. If something happens quicker then I am pleasantly surprised. Yes, patience before patients."
UPDATE: Casey Eye Involvement With Both Trials plus Recruitment info
I had several e-mail exchanges with Maureen Toomey , Study Coordinator for Casey Eye. They will be in on the dryAMD trial also. Below is a quick snapshot followed by e-mail responses.
[B]Stargardt Trial[/B]
1) recruitment has not started yet
2) ACT needs to travel to Casey for training meeting
3) Mid June contemplated for training meeting
4) After meeting and after stem cells received, recruitment can begin
[B]DRY AMD[/B]
1) ACT asked them to be a site a couple weeks ago
2) In the process of IRB approval and negotiating contract
3) Probably a couple months before recruitment begins
____________________________________________________________________________ On 05/26/2011 10:16 AM, Maureen Toomey wrote:
Dear Rocky,
Thank you for your email. About a couple of weeks ago, Advanced Cell Technology asked us to be a site in the Dry AMD study. We are currently in the process of obtaining institutional approval to begin recruitment and negotiating the contract. I expect it will be another couple months or so before we are ready to begin recruiting patients.
Hope this helps.
Maureen
Maureen Toomey, BS, CCRP
Study Coordinator
Casey Eye Institute
3375 SW Terwilliger Blvd.
Portland, OR 97239
Ph: 503-494-3795
Fax: 503-418-3964
Rocky,
We have not yet started to recruit patients for the Stargardt’s study. ACT still needs to travel to our site for a training meeting. This will probably occur in the middle of June. Shortly after the site visit and after we receive the stem cells, we should be ready to recruit patients.
Thanks,
Maureen
louisa,
possible first injection in June as stated prior.
Roslin..no idea
Orphan desig in EU? psssible in June according to TMC Pharma's e-mail
http://investorstemcell.com/forum/advanced-cell-technology/2490.htm
EU CTA(clinical trial application for Stargardt) possible by end of June according to ACT PR dated April 20
"During the CTA review process, which requires a minimum of 60 days, the reviewers decide if an applicant is permitted to proceed with its proposed clinical trial."
http://www.advancedcell.com/news-and-media/press-releases/act-files-european-clinical-trial-application-for-phase-12-study-using-embryonic-stem-cells-to-treat-/
Korea? ACT hasn't mentioned it. Remember CHA acquired exclusive rights to RPE technology for Korea. CHA has access to almost everything ACT has done with FDA here and ACT per contract is to help them through the Korean process. This is not a JV as SCRMI is.
louisa,
NIH has an Advisory meeting June 9–10, 2011. Not sure they will be addressing "redefinition" as court case is still ongoing as far as I know.
http://acd.od.nih.gov/meetings.asp
June 9 - 10, 2011
Advisory Committee to the Director
National Institutes of Health
Bethesda, MD
The agenda include: NIH Director's Report, NIH updates, and other committee business.
http://advocacy.cancer.gov/activities/meetings
dianne,
Isn't June the time frame they have given?
(Rabin statement from CC)
"Based on our extensive calendar review, I therefore expect that we will begin both trials relatively contemporaneously, in late May or early to mid June."
only to say todays volume was about 141 million shares less than volume on Dec 8,2010,(less than 6 months ago)..:)
Biowonk,
first off, the post you linked to was a Form 4 filed by CEO Rabin...not Lanza. Secondly, In 2010 Lanza's compensation was 3,142,298(salary of $350k, Bonus $50K, and stock awards $2,717,298) plus he holds a total of 28,442,180 shares as of May 10,2011, most of which were gifted to him in 2010. Lanza also sold about 3.6MM shares under a pre-arranged plan last year.
http://www.sec.gov/Archives/edgar/data/1140098/000101376210000926/form8k.htm
So nothing really "paltry" about his compensation or gifted shares.
Better to keep your ears close to the ground regarding the on-going trials
Gee, I will attempt to do just that.
I hear ya,
"Penalties. Any late Form 4 filings must be disclosed in the proxy statement and a box must be checked on the cover page of the issuer’s Form 10-K indicating that late filings were made. Additionally, the Sarbanes-Oxley Act gives the SEC broad authority to seek any equitable relief it finds appropriate or necessary to benefit investors for any violations of the new two business day filing deadline."
http://www.levettrockwood.com/00129012.pdf
Securities Exchange Act of 1934
http://taft.law.uc.edu/CCL/34Act/sec16.html
Form 4 filed,(thanks rumit),
http://www.sec.gov/Archives/edgar/data/1140098/000114420411032278/xslF345X03/v224105_ex.xml
demmo,
He only stated he would provide financial "results".
Actually, the bolded statement below implies much more. "Current Information" upgrade requires much more.
http://investorshub.advfn.com/boards/read_msg.aspx?message_id=62955541
International Drug & Explosives Detection Company IDenta Corp. to Become a Reporting Company
IDenta Corp. (PINKSHEETS: IDTA) CEO Yaacov Shoham announced today that the company will begin releasing financial results starting with the first quarter of 2011 and results will be released each quarter thereafter. Mr. Shoham commented, "Because of the healthy situation of all aspects of our company, we are going to move IDenta from a designation of 'No Information' to one of 'Current Information.' This will allow the investing community and others a chance to see the successes and continued growth of our company. We are very pleased to be able to begin reporting results."
ford,
are you referring to patient screening?
investing,
Not real sure you or anyone else will know when those shares are sold. Form 4's are no longer required and they hold less than 10% of OS# so reporting is a non-issue the way I understand it all.
dianne,
Hello,
It really isn't unusual. I know of one company you are familiar with where the CEO booked $8.86MM in 2010 and he had NEVER bought shares on the open market either. For that matter, no BOD or management ever has. When you can issue shares to yourself and others, why buy? I agree with what you are saying 100%, just saying.....
rangois,
Place and time for annual meeting is set. Whether you attend or not is a personal thing. There is nothing on the agenda that I would travel 20 miles to attend. Probably those that do go are interested in meeting management and being involved in the "question/answer" session.
"You are cordially invited to attend the 2011 Annual Meeting of Stockholders (the “Annual Meeting”) of Advanced Cell Technology, Inc., which will be held at the Hyatt Regency Suites, Palm Springs, 285 N. Palm Canyon Dr., Palm Springs, CA 92262 on Thursday, June 9, 2011, at 9:00 a.m. local time. Details of the business to be conducted at the Annual Meeting are provided in the attached Notice of Annual Meeting and Proxy Statement."
http://www.sec.gov/Archives/edgar/data/1140098/000114420411027813/v221776_def14a.htm
harlem.
Everyone has their own opinions on this topic. I find the Rabin statement below in line with my thoughts. You could see a "player" get involved with dollars or options to pursue if positive results continue. So many avenues of possibles.
An example:
Oxford BioMedica will receive committed funding of up to US$24 million over the initial phase of development. Oxford BioMedica granted sanofi-aventis a license to develop the products and an option for further development, manufacture and commercialisation on a worldwide basis.
(Rabin from CC)
"I just returned from Europe where we had some great meetings. As far as the RPE program goes, it would be highly premature to seek partners early in the phase 1 study as we are hopeful that even though we are dealing with late stage patients, we will soon have data on retinal engraftment of our cells, and thier impact on photorecptor function. If we see no safety issues, and are able to detect some engraftment of our cells to the photoreceptor / Bruch's membrane, we will obviously be dealing from a much stronger valuation and negotiating position. It is quite possible that within about 12 weeks of commencement of the trials, we may have a pretty interesting set of data to evaluate and discuss, covering an early look at a total of 6 patients between the two trials."
Fern,
Yeah, that is Myoblast Phase 2. We have been looking for a JV for years on that program but I don't think that is what Harlem was getting at..thanks
harlem,
I am seeing the "JV" thing everywhere. I am not real sure what you or others are meaning. A joint venture for what? Meaning a Pharma partner to go the distance? or something else?
Secondly, why would initial data on engraftment or other early signs prompt a JV "in the next few months" early in the safety phase?
I can respond to your question better if you respond to these..thank you Harlem
Twaro,
Investors will not experience the exponential gains seen after positive Phase III results this year, that is a almost a given.
Phase 3 results?
Karin,
AXM (Pink Sheets: AXMP) is the parent company of AXM Shenyang. The company was founded in 2004 at Shenyang, China. Its mission is to become a premier China-based US GMP pharmaceutical manufacturer and distributor targeting and penetrating the markets of China. The company currently owns licenses and is capable to produce both prescription and nonprescription drugs. AXM Shenyang has the ability to produce 30,000,000 tubes for ointment, 500,000,000 tablets and 250,000,000 capsules annually. The company has six buildings totaling over 200,000 square feet that are built on more than 13 acres of campus. It has the technology of microbial fermentation which is used to produce testosaterone and testosaterone undecanoate. In 2007, AXM Shenyang was appointed to sell GM1 as exclusive distributor for Brazil. Currently AXM Shenyang has more than 200 people in marketing and distribution serving more than 20 provinces in China.
http://www.cordblood-america.com/cbai-news/129-china-cord-blood-america-otc-bb-cbai-to-operate-stem-cell-business
fordwill,
excellent post. While we have made progressional steps(all good), we have a long row to hoe yet...
hopeful,
since you first came to this site you keep saying Geron split and we have discussed this before. Geron has never split.
Also, you speak of Lanza being on Geron's BOD's at some time..I very much doubt that. If you have a link, please provide...thanks
I will pass on the offer Louisa, had a few e-mails with the author and will seek a source with first hand knowledge. Thank you anyway.
rumit, a cell line with an O negative blood type is huge, as you implied. If I were to bet 20 cents, I would say they don't have one. I will continue to prod at times, maybe they will respond at some point.
No responses whatsoever on that topic rumit. One would think a yes or no would be in order, but not the case.
yes, I saw that and thanks. All of these "clarifications" go back to the reaming ACT received from the Senate. Not hard to figure why confusion exists.
http://investorshub.advfn.com/boards/read_msg.aspx?message_id=34923915&
rumit and all,
here is the report on the actual development of 2 single blastomere cell lines ACT created. MA01, and MA09
10 experiments took place and the Table on slide 1 at link below tells what happened to all ten embryos.
"The zona pellucida was disrupted using either acidic Tyrode’s solution (experiments 1–5) or multiple piezo-pulses (experiments 6–10). After biopsying, the embryos were not allowed to continue
developing and were discarded."
(Nov 2006 Nature)
Human embryonic stem cell lines derived from single
blastomeres
http://cord.rutgers.edu/stemcellcourse/documents/hESC_from_PGD_Nature_07.pdf