Published October 15, 2014

For the first time, researchers have created functioning human lung cells from stem cells.

The longest-running trial of stem cells derived from a human embryo found that the cells caused patients none of the problems scientists feared, such as forming tumors, and reversed partial blindness in about half the eyes receiving transplants, researchers reported on Tuesday.

The results, published in The Lancet, could help re-invigorate the controversial quest to harness stem cells, which have the ability to turn into any of the 200 kinds of human cells, to treat diseases.

In an accompanying commentary, Dr. Anthony Atala of the Wake Forest Institute for Regenerative Medicine called the work "a major accomplishment."

After intense excitement among scientists and the public about the promise of stem cells and ethical debates about destroying human embryos to obtain them, the field stumbled when a high-profile trial for spinal cord injury was halted by Geron Corp in 2011 and the interest of other companies waned.

The small study's main goal was assessing the safety of the transplanted cells. Called retinal pigment epithelial cells, they were created by taking stem cells from a days-old embryo created in a fertility clinic and inducing them to differentiate into the specialized cells.

The study "provides the first evidence, in humans with any disease, of the long-term safety and possible biologic activity" of cells derived from embryos, said co-author Dr. Robert Lanza, chief scientific officer of Advanced Cell Technology, which produced the cells and funded the study.

Nine patients with Stargardt's disease (which causes macular degeneration in childhood) and nine with dry age-related macular degeneration (a leading cause of adult blindness) received implants of the retinal cells in one eye. The other eye served as a control.

Four eyes developed cataracts and two became inflamed, probably due to the patients' age (median: 77) or the use of immune-supressing transplant drugs.

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Stem cells from human embryos prove safe, improve vision, study says

Antonio Regalado for MIT Technology Review 2014-10-15 19:15:44 UTC

When stem cells were first culled from human embryos sixteen years ago, scientists imagined they would soon be treating diabetes, heart disease, stroke, and many other diseases with cells manufactured in the lab.

It's all taken longer than they thought. But now, a Massachusetts biotech firm has reported results from the largest, and longest, human test of a treatment based on embryonic stem cells, saying it appears safe and may have partly restored vision to patients going blind from degenerative diseases.

Results of three-year study were described Tuesday in the Lancet by Advanced Cell Technology and collaborating eye specialists at the Jules Stein Eye Institute in Los Angeles who transplanted lab-grown cells into the eyes of nine people with macular degeneration and nine with Stargardt's macular dystrophy.

The idea behind Advanced Cell's treatment is to replace retinal pigment epithelium cells, known as RPE cells, a type of caretaker tissue without which a person's photoreceptors also die, with supplies grown in laboratory. It uses embryonic stem cells as a starting point, coaxing them to generate millions of specialized retina cells. In the study, each patient received a transplant of between 50,000 and 150,000 of those cells into one eye.

The main objective of the study was to prove the cells were safe. Beyond seeing no worrisome side effects, the researchers also noted some improvements in the patients. According to the researchers half of them improved enough to read two to three extra lines on an eye exam chart, results Robert Lanza, chief scientific officer of Advanced Cell, called remarkable.

"We have people saying things no one would make up, like 'Oh I can see the pattern on my furniture, or now I drive to the airport," he says. "Clearly there is something going on here."

Lanza stressed the need for a larger study, which he said the company hoped to launch later this year in Stargardt's patients. But if the vision results seen so far continue, Lanza says "this would be a therapy."

Some eye specialists said it's too soon to say whether the vision improvements were real. The patients weren't examined by independent specialists, they said, and eyesight in patients with low vision is notoriously difficult to measure. That leaves plenty of room for placebo effects or unconscious bias on the part of doctors.

"When someone gets a treatment, they try really hard to read the eye chart," says Stephen Tsang, a doctor at Columbia University who sees patients losing their vision to both diseases. It's common for patients to show quick improvements, he says, although typically not as large as what Advanced Cell is reporting.

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Stem Cell Eye Treatment May Restore Vision

Posted: Tuesday, October 14, 2014, 7:00 PM

TUESDAY, Oct. 14, 2014 (HealthDay News) -- A new study is the first to show the long-term safety of embryonic stem cell transplants to treat human disease.

The research involved 18 people who received the transplants to treat forms of macular degeneration, a leading cause of vision loss.

The transplants, which restored some sight in more than half of the patients, appeared safe up to three years after the procedure.

The study, funded by a U.S.-based company called Advanced Cell Technology, was published Oct. 14 in The Lancet.

"Embryonic stem cells have the potential to become any cell type in the body, but transplantation has been complicated by problems," lead author Dr. Robert Lanza, chief scientific officer at Advanced Cell Technology, said in a journal news release. Those problems include the rejection of the transplanted cells by the patient's immune system, as well as the danger that the cells might spur certain types of cancers called teratomas.

A teratoma is a type of cancer that occurs when stem cells develop into multiple types of cells and form incompatible tissues that can include teeth and hair.

As Lanza explained, because of these issues, scientists interested in embryonic stem cell therapy have tended to focused on sites in the body that typically do not produce a strong immune response. The eye is one such spot.

In the new study, human embryonic stem cells were first prompted to develop into eye cells called retinal pigment epithelial cells. They were then transplanted into nine people with Stargardt's macular dystrophy, and another nine with dry atrophic age-related macular degeneration.

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Embryonic Stem Cell Therapy Shows Long-Term Effectiveness, Safety

NEW YORK, July 7, 2014 /PRNewswire/ --Advanced Cell Technology, Inc. (OTCQB: ACTC) is a biotechnology company focused on developing and commercializing human pluripotent stem cell technology in the field of regenerative medicine. The company is currently conducting clinical trials for treating dry age-related macular degeneration (AMD) and Stargardt's macular degeneration (SMD), as well as several clinical and preclinical programs for other ocular therapies. Outside of ophthalmology, ACTC also has a preclinical development pipeline focused on autoimmune diseases, inflammatory diseases and wound healing. The company's intellectual property portfolio includes pluripotent human embryonic stem cells (hESCs), induced pluripotent stem cells (iPSCs), and other cell therapy research programs.

As the worldwide population has continued to age, so too has the need for regenerative medicine. In fact, by 2050, the number of people in the world over the age of 65 is expected to rise to 1.5 billion nearly triple the amount today. Unsurprisingly, as this demographic shift occurs over the next 35 years, health care expenditures are projected to increase rapidly as well. For example, in the US, the share of GDP devoted to healthcare is estimated to reach 34% by 2040 from about 18% just a few years ago. Considering the majority of treatments for chronic and/or life-threatening diseases that are available today only treat symptoms rather than offer a cure for the underlying cause, regenerative medicine such as the stem cell therapies being developed by ACTC are aimed at addressing this unmet and growing need.

Macular degeneration (i.e. age-related macular degeneration, or AMD) is a medical condition that results in a loss of vision in the center of the visual field (the macula) because of damage to the retina. This indication is the leading cause of blindness and visual impairment in adults over fifty years of age. Currently, it is estimated that there are approximately 30 million people worldwide who suffer from AMD ranging from early-stage to late-stage (i.e. legal blindness), with an estimated market size of around $30 billion. Further, in an article in the journal, Lancet projected that the number of people globally with AMD will be 196 million in 2020, growing to 288 million by 2040.

A full in-depth analyst report on ACTC that includes risk factors, industry review, financial position, potential revenues, review of current business model, competition breakdown, analyst summary, and recommendation can be viewed by using the following link at no cost:

http://bit.ly/-ACTC-AnalystReport

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FORWARD-LOOKING DISCLAIMER

This report may contain certain forward-looking statements and information, as defined within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, and is subject to the Safe Harbor created by those sections. This material contains statements about expected future events and/or financial results that are forward-looking in nature and subject to risks and uncertainties. Such forward- looking statements by definition involve risks, uncertainties and other factors, which may cause the actual results, performance or achievements of mentioned company to be materially different from the statements made herein.

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Content is researched, written and reviewed on a best-effort basis. Research report provided for informational purposes. This document, article or report is written and authored by Michael Maggi, Chartered Financial Analyst. However, we are only human and are prone to make mistakes. If you notice any errors or omissions, please notify us below.

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Advanced Cell Technology Analyst Report; Shareholder Value Likely to Continue to Erode for the Foreseeable Future by ...

MARLBOROUGH, Mass.--(BUSINESS WIRE)--

Advanced Cell Technology, Inc. (ACT; OTCBB: ACTC), a leader in the field of regenerative medicine, today announced treatment of the second patient in its Phase 1/2 clinical trial for Stargardts macular dystrophy (SMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). The surgery was performed on Friday, June 29 at Moorfields Eye Hospital in London, the same site as the first patient treatment in January, by a team of surgeons led by Professor James Bainbridge, consultant surgeon at Moorfields and Chair of Retinal Studies at University College London. The procedure was successfully performed without any complications. ACT and Moorfields Eye Hospital recently received clearance from the Data and Safety Monitoring Board (DSMB) to treat the final two patients in the first cohort of this clinical trial.

We are very pleased to continue our forward momentum with both our U.S. trials and our European trial, commented Gary Rabin, chairman and CEO. It was less than a month ago that we received DSMB approval to treat the second and third patients in our E.U. trial, and it is very gratifying to have already completed dosing of the second. It is a pleasure to be working with Professor Bainbridge and the rest of his team at Moorfields Eye Hospital, and we continue to be encouraged by the steady progress of the trial thus far.

The Phase 1/2 trial is designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation in patients with SMD at 12 months, the studys primary endpoint. It will involve a total of 12 patients, with cohorts of three patients each in an ascending dosage format. It is similar in design to the U.S. trial for SMD that was initiated in July 2011.

The European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP) has officially designated ACT's human embryonic stem cell (hESC)-derived retinal pigment epithelial (RPE) cells as an orphan medicinal product for the treatment of Stargardt's Macular Dystrophy (SMD).

More information on the status of the companys clinical trials will be posted today on Mr. Rabins Chairmans blog.

About Stargardts Disease Stargardts disease or Stargardts Macular Dystrophy is a genetic disease that causes progressive vision loss, usually starting in children 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, which is the site of damage that the company believes the hESC-derived RPE may be able to target for repair after administration.

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.

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 companys periodic reports, including the report on Form 10-K for the year ended December 31, 2011. Forward-looking statements are based on the beliefs, opinions, and expectations of the companys 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 companys 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 Companys clinical trials will be successful.

Excerpt from:
ACT Announces Second Patient with Stargardt’s Disease Treated in EU Clinical Trial

MARLBOROUGH, Mass.--(BUSINESS WIRE)--

Advanced Cell Technology, Inc. (ACT; OTCBB: ACTC), a leader in the field of regenerative medicine, announced today the dosing of the third patient in its Phase I/II trial for dry age-related macular degeneration (dry AMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). The outpatient transplantation surgery was performed successfully, and the patient is recovering uneventfully.

Gary Rabin, chairman and CEO of ACT, commented, The completion of enrollment of the first cohort of patients in our dry AMD clinical trial is a significant step forward in our RPE clinical program. The first six patients in the U.S. trials have all been treated at UCLA, and as we have recently announced, the trials should soon expand to additional sites. As we have built our clinical team, we have been fortunate to have attracted the attention of some of the highest-caliber ophthalmologists and related institutions in the U.S. and Europe and recognize the huge value that their expertise provides us as we plan for the future of our therapeutic programs. With their guidance, we have also worked with the FDA to successfully expand the criteria of eligibility for patients to participate in our dry AMD trial.

The procedures at UCLA were all conducted by the team led 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.

The six patients treated at UCLA to date have tolerated the surgical procedure well. commented Dr. Schwartz. There have been no complications in the procedure, nor any issues relating to the safety of the injected stem cell-derived RPE cells in any of the patients. We continue to regularly evaluate all patients in the trial, and while still preliminary, I am encouraged by the patients progress and the relative straightforwardness of the surgical procedure.

We are extremely pleased with the progress being made in all three of our clinical trials here in the U.S. and the U.K., commented Robert Lanza, M.D., ACTs chief scientific officer. The data we are reviewing seems to be pointing in the appropriate direction, With the treatment of the latest two dry AMD patients, we look forward to having more significant points of reference to understand the progress of the trial and consider the endpoint design for the next phase. Both Stargardts disease and dry AMD are progressive diseases that result vision loss and blindness due to the thinning of the layer of RPE cells in the patient's macula, the central portion of the retina responsible for central vision. We still have many patients left to treat during the course of these trials, but our team remains hopeful that stem cell-derived RPE cells may someday provide a new therapeutic approach for the treatment of many forms of macular degeneration. We hear from patients who suffer from these diseases on nearly a daily basis, and appreciate the huge responsibility we have to them.

ACT is conducting three clinical trials in the U.S. and Europe using hESC-derived RPE cells to treat forms of macular degeneration. Each trial will enroll a total of 12 patients, with cohorts of three patients each in an ascending dosage format. These 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 dry-AMD or Stargardt's macular dystrophy (SMD) at 12 months, the studys primary endpoint. Preliminary results relating to both early safety and biological function for the first two patients in the United States, one SMD patient and one dry AMD patient, were recently reported in The Lancet. On January 20, 2012, the first SMD patient to be enrolled in the Companys U.K. clinical trial was treated at Moorfields Eye Hospital in London. The final patient of the first cohort in the companys SMD trial in the U.S. was treated on February 13, 2012.

Further information about patient eligibility for the dry AMD study and the concurrent study on SMD is also available on http://www.clinicaltrials.gov; ClinicalTrials.gov Identifiers: NCT01345006 , NCT01469832 and NCT01344993.

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.

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ACT Announces Third Dry AMD Patient Treated in Clinical Trial

SILVER SPRING, Md.--(BUSINESS WIRE)--

Nuvilex, Inc. (OTCQB:NVLX), an emerging biotechnology provider of cell and gene therapy solutions, today pointed out the potential for substantial partnership and licensing opportunities using the companys cell encapsulation technology for applications in stem cell research and medicine. Migration of implanted cells away from the target site and host rejection have been recognized as fundamental challenges faced by the stem cell community regarding their use in therapy, which the companys technology overcomes.

The technology being acquired from associate SG Austria is used to place live stem cells into strong, flexible and permeable capsules. These capsules can then be implanted into animals or humans for specific therapies. Stem cells can then exist at the desired location inside the capsules, prevented from migrating and protected from the immune system that aims to eliminate such foreign cells from the body.

Stem cell therapy is being used by clinicians throughout the world for treating such diverse diseases as spinal cord injury, amyotrophic lateral sclerosis, burns, glioma, multiple myeloma, arthritis, heart disease, stroke, Stargardt's Macular Dystrophy, and age-related macular degeneration, among others, most of which can be found at ClinicalTrials.gov.

Historically, researchers have faced numerous difficulties in succeeding with certain stem cell treatments, because of the problems associated with keeping stem cells alive for significant periods of time, stopping rejection and destruction by the recipients immune system, and keeping stem cells from migrating away from the desired sites. Cells encapsulated in SG Austrias porous beads have been shown to remain alive for long periods of time in humans, surviving intact for at least two years. Once encapsulated, cells are protected from the bodys immune system. Furthermore, encapsulated cells remain within the beads and are unable to migrate to other sites in the body.

In the February 29, 2012 research report, Goldman Small Cap Research stated, The Cell-in-a-Box approach could significantly advance the implementation and utilization of stem cells for a host of debilitating diseases and conditions, making it a uniquely valuable commodity. We believe that by partnering with leading players in the field, Nuvilex could find that companies with deep pockets would be happy to collaborate or license the delivery system and engage in further research which could result in meaningful development and licensing revenue.

Dr. Robert Ryan, Chief Executive Officer of Nuvilex, discussed the value for licensing the companys stem cell therapy, adding, By overcoming traditional barriers to effective stem cell therapy, namely viability, migration, and host rejection, we believe these new advances in medical science utilizing stem cells and encapsulation will enable us to take quantum leaps forward now and in the future. As a result of challenges SG Austria has overcome, new advances will be surprisingly close at hand and are part of the driving force behind our desire to work with a number of companies in this endeavor. Our primary goal has been and remains to use our technology to bring life changing treatments to patients on an expedited basis.

About Nuvilex

Nuvilex, Inc. (OTCQB:NVLX) is an emerging international biotechnology provider of clinically useful therapeutic live encapsulated cells and services for encapsulating live cells for the research and medical communities. Through our effort, all aspects of our corporate activities alone, and especially in concert with SG Austria, are rapidly moving toward completion, including closing our agreement. One of our planned offerings will include cancer treatments using the companys industry-leading live-cell encapsulation technology.

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Nuvilex Forecasts Vast Partnership Opportunities Using Breakthrough Stem Cell Technology

Advanced Cell Technology, which has unsuccessfully sought funding several times from the $3 billion California stem cell agency, drew some attention today in a piece in a Massachusetts newspaper.

The Worcester Telegram took a look at the firm, headquartered in Santa Monica, Ca., with labs in Marlboro, Mass., in the wake of Geron's departure from hESC research. The move left ACT as the only firm in the country with an hESC trial and perhaps with a better shot at CIRM funding.

Reporter Lisa Eckelbecker wrote,

"Advanced Cell, publicly traded since 2005, has spent years developing its technologies. The company brings in little revenue and has an accumulated deficit of $180.9 million. About 1.6 billion shares of Advanced Cell common stock is outstanding, a result of numerous financings over the years. It trades for about 10 cents a share on the Over-the-Counter Bulletin Board, an electronic exchange for small companies. No analysts from major Wall Street banks report on the company.

"The company's treatment for Stargardt's macular dystrophy and dry age-related macular degeneration — the treatment that required (a) mountain of paperwork before the FDA — first went into the eyes of patients in July in Los Angeles. The retinal pigment epithelial cells, generated from embryonic stem cells, were developed to slow the progression of the eye disorders, which can lead to blindness."

ACT moved its headquarters to California following the passage of Prop. 71 in 2004, the ballot initiative that created the California stem cell agency. The company said at the time it expected to "gain significant momentum by being able to take advantage of a favorable environment for funding."

ACT initially landed in Alameda, Ca., but has since moved to Southern California. Its official opening in 2006 in Alameda was attended by the state treasurer and at least one CIRM official, according to the company. The firm has never secured funding from the stem cell agency, which does not release the names of rejected applicants. However, the California Stem Cell Report carried an item in 2008 that pointed out that a researcher for ACT complained publicly about a reviewer's conflict of interest in connection with an ACT application(see here and here). At the time, Robert Klein, then CIRM chairman, brushed off the complaint. The journal Nature has also reported that ACT has applied unsuccessfully several times for CIRM awards.

It is a fair bet that ACT was an initial applicant in the round that provided funding to Geron last spring. However, by the time Geron's application went to the full CIRM board, the other applicants had withdrawn – the first time such an event had occurred at CIRM.

Since Geron pulled out of the hESC business last month, it is likely that ACT and CIRM have opened fresh discussions, given their mutual interest in producing a stem cell therapy. CIRM also has a new chairman who is familiar with ACT. After Geron was awarded its $25 million loan from CIRM last May, the agency's board elected as chairman a Los Angeles bond financier, Jonathan Thomas, who led an early round of financing for ACT in 2000. Thomas last summer sold his remaining 17,046 shares in ACT for $3,239. Thomas said he had a "significant loss" on the sale but did not disclose the amount.

Geron's flight from hESC and ACT's perserverance come as the stem cell agency is pushing aggressively to drive research into the clinic. Plus CIRM needs tangible results that voters can understand if CIRM is win ballot-box approval for continued funding in the next few years. The agency will run out of cash in about 2017 and is considering mounting a campaign for another multibillion bond issue.

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