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ACT Announces First Stargardt Patient Treated With Higher Dosage of Embryonic Stem Cell-Derived Retinal Pigment …

Posted: July 13, 2012 at 3:12 am

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

Advanced Cell Technology, Inc. (ACT; OTCBB: ACTC), a leader in the field of regenerative medicine, today announced treatment of the fourth patient, the first in the second patient cohort, in its U.S. clinical trial for Stargardts Macular Dystrophy (SMD). The surgery was performed on Wednesday, July 11 at Wills Eye Institute in Philadelphia, by a surgical team lead by Carl D. Regillo, MD, FACS, director of the Wills Eye Clinical Retina Research Unit, attending surgeon in the Wills Eye Retina Service at the Wills Eye Institute, and professor of ophthalmology at Thomas Jefferson University. In keeping with trial protocol, the patient was injected with 100,000 human embryonic stem cell-derived retinal pigment epithelial (RPE) cells, as compared with the 50,000 cell dose used in the three patients of the first cohort. The outpatient transplantation surgery was performed successfully and the patient is recovering uneventfully.

It is very gratifying to have second cohort, higher-dosage patient treatment underway in our U.S. clinical trial for SMD, commented Gary Rabin, chairman and CEO of ACT. We are also pleased to be working with Dr. Regillo and his team at Wills Eye Institute, a truly first-class institution that is ranked as one of the best ophthalmology hospitals in the country byU.S. News & World Report.

Initiated in July of last year, the Phase I/II 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. As part of its RPE clinical program, the company is concurrently conducting a clinical trial for dry age-related macular degeneration and second trial for SMD in the United Kingdom.

Doubling the cell dosage marks an important milestone in our clinical programs, said Robert Lanza, MD, ACTs chief scientific officer. We look forward to continued progress and safety findings in the coming months, in both our U.S. and European trials.

Further information about patient eligibility for ACTs SMD study and the concurrent studies in the U.S. and Europe (for dry age-related macular degeneration and SMD, respectively) are available at http://www.clinicaltrials.gov, with the following Identifiers: NCT01345006 (U.S. SMD), NCT01344993 (dry AMD), and NCT01469832 (E.U. SMD).

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.

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Cells derived from debrided burn tissue may be useful for tissue engineering

Posted: July 12, 2012 at 7:19 am

Public release date: 11-Jul-2012 [ | E-mail | Share ]

Contact: David Eve celltransplantation@gmail.com Cell Transplantation Center of Excellence for Aging and Brain Repair

Putnam Valley, NY. (July 11, 2012) A research team in the Netherlands has found that cells from burn eschar, the non-viable tissue remaining after burn injury and normally removed to prevent infection, can be a source of mesenchymal cells that may be used for tissue engineering. Their study compared the efficacy of those cells to adipose (fat)-derived stem cells and dermal fibroblasts in conforming to multipotent mesenchymal stromal cell (MSC) criteria.

Their study is published in the current issue of Cell Transplantation (21:5), now freely available on-line at http://www.ingentaconnect.com/content/cog/ct/.

"In this study we used mouse models to investigate whether eschar-derived cells fulfill all the criteria for multipotent mesenchymal stromal cells as formulated by the International Society for Cellular Therapy (ISCT)," said study co-author Dr. Magda M.W. Ulrich of the Association of Dutch Burn Centres, The Netherlands. "The study also assessed the differentiation potential of MSCs isolated from normal skin tissue and adipose tissue and compared them to cells derived from burn eschar."

According to the researchers, advances in burn treatment have meant that the percentage of patients surviving severe burn injuries is increasing. This escalating survival rate has also increased the number of people who are left with burn scars, which lead to functional problems with the skin, such as contracture, and the social and psychological aspects of disfigurement.

Tissue engineering to rebuild the skin is the most promising approach to solving these problems. However, two problems arise with tissue engineering the source of the cells and the design of the scaffold aimed at creating a microenvironment to guide cells toward tissue regeneration.

"The choice of cells for skin tissue engineering is vital to the outcome of the healing process," said Dr. Ulrich. "This study used mouse models and eschar tissues excised between 11 and 26 days after burn injury. The delay allowed time for partial thickness burns to heal, a process that is a regular treatment option in the Netherlands and rest of Europe."

The researchers speculated that during this time the severely damaged tissues could attract stem cells from the surrounding tissues, as elevated levels of MSCs have been detected in the blood of burn victims.

"MSCs can only be beneficial to tissue regeneration if they differentiate into types locally required in the wound environment," noted Dr. Ulrich. "We concluded that eschar-derived MSCs represent a population of multipotent stem cells. The origin of the cells remains unclear, but their resemblance to adipose-derived stem cells could be cause for speculation that in deep burns the subcutaneous adipose tissue might be an important stem cell source for wound healing."

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Verastem Acquires Clinical-Stage FAK Inhibitor from Pfizer

Posted: July 12, 2012 at 7:19 am

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

Verastem, Inc., (VSTM) a biopharmaceutical company focused on discovering and developing drugs to treat breast and other cancers by targeting cancer stem cells, announced an agreement with Pfizer for the exclusive in-license of worldwide commercial rights for VS-6063 (formerly PF-04554878), a focal adhesion kinase (FAK) inhibitor that has completed a Phase 1 clinical study in advanced solid tumors.

FAK is a non-receptor tyrosine kinase that regulates tumor cell proliferation and invasion. The targeted disruption of this pathway in preclinical models of cancer reduces cancer stem cells, primary tumor mass and metastasis.

Verastem has identified the FAK pathway as a critical regulator of the survival of cancer stem cells, which are an underlying cause of cancer recurrence and metastasis, said Robert Weinberg, Ph.D., Verastem co-founder and chair of the Scientific Advisory Board.

VS-6063 is being developed for the treatment of solid tumors. According to data presented at ASCO 2011 from a Phase 1 safety study of VS-6063 in 36 patients conducted by Pfizer, VS-6063 was well-tolerated and demonstrated signs of clinical activity to support further development. Verastem anticipates conducting clinical trials targeting solid tumor indications with VS-6063.

Like Pfizer, Verastem is committed to bringing innovative treatments to patients with cancer, said Garry Nicholson, President and General Manager of Pfizer Oncology. Verastems specific focus on targeting cancer stem cells makes them the ideal company to continue the development of this compound.

Under the terms of the agreement, Verastem will assume sole responsibility for global product development of VS-6063. Pfizer will receive an upfront payment in cash and Verastem equity, development milestones and royalties and milestones on future sales of VS-6063.

VS-6063 accelerates Verastems FAK inhibitor program with a clinical, Phase 2-ready product candidate targeting this key regulatory pathway for cancer stem cells, said Christoph Westphal, M.D., Ph.D., Chairman and Chief Executive Officer of Verastem. We believe our focus on identifying patients with a high cancer stem cell burden for treatment with our targeted therapies uniquely positions Verastem to lead the next wave of therapeutics in cancer.

Conference Call and Webcast Information Verastem will discuss the acquisition during the Research and Development Day to be held tomorrow, July 12, at 9:00am ET. A live webcast of the event can be accessed by visiting the investors section of the Companys website at http://www.verastem.com. A replay will be available for two weeks from the date of the event.

A live, listen-only conference call of the event can be accessed by dialing 1-866-700-7173 five minutes prior to the start of the event and providing the passcode 73322380.

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Verastem Acquires Clinical-Stage FAK Inhibitor from Pfizer

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Stem cell cures bring challenges

Posted: July 12, 2012 at 7:19 am

A WORLD-RENOWNED Australian stem cell expert says it's crucial that any potential cures using the technology are available globally, particularly in developing countries.

Professor Alan Trounson heads the California Institute of Regenerative Medicine, which is advancing towards a cure for conditions including HIV and diabetes.

Prof Trounson, the former director of Monash Immunology and Stem Cell Laboratories in Melbourne, said the research institute's work towards a HIV cure was about to enter human trials.

He said if the trials were successful further research would be needed to modify the technology so it could be affordably used where it is needed most, in Africa.

"I want the HIV work to go globally because it shouldn't be restricted to patients in Western (countries)," Prof Trounson told AAP.

"We're going to have to modify some of that further research to get it into a suitable treatment that we can use in Africa.

"I'm very hopeful that the industry will do that.

"We have to try and make these as available to people as possible."

Prof Trounson said the HIV research uses blood stem cells to mimic a gene mutation found in a small proportion of the population who are immune to the virus.

The approach is based on the case of an American, known as the Berlin patient, who was cured of HIV after receiving a life-saving bone marrow transplant to treat leukaemia from a donor who carried the HIV-immune mutation.

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Cells derived from debrided burn tissue may be useful for tissue engineering

Posted: July 12, 2012 at 4:15 am

Public release date: 11-Jul-2012 [ | E-mail | Share ]

Contact: David Eve celltransplantation@gmail.com Cell Transplantation Center of Excellence for Aging and Brain Repair

Putnam Valley, NY. (July 11, 2012) A research team in the Netherlands has found that cells from burn eschar, the non-viable tissue remaining after burn injury and normally removed to prevent infection, can be a source of mesenchymal cells that may be used for tissue engineering. Their study compared the efficacy of those cells to adipose (fat)-derived stem cells and dermal fibroblasts in conforming to multipotent mesenchymal stromal cell (MSC) criteria.

Their study is published in the current issue of Cell Transplantation (21:5), now freely available on-line at http://www.ingentaconnect.com/content/cog/ct/.

"In this study we used mouse models to investigate whether eschar-derived cells fulfill all the criteria for multipotent mesenchymal stromal cells as formulated by the International Society for Cellular Therapy (ISCT)," said study co-author Dr. Magda M.W. Ulrich of the Association of Dutch Burn Centres, The Netherlands. "The study also assessed the differentiation potential of MSCs isolated from normal skin tissue and adipose tissue and compared them to cells derived from burn eschar."

According to the researchers, advances in burn treatment have meant that the percentage of patients surviving severe burn injuries is increasing. This escalating survival rate has also increased the number of people who are left with burn scars, which lead to functional problems with the skin, such as contracture, and the social and psychological aspects of disfigurement.

Tissue engineering to rebuild the skin is the most promising approach to solving these problems. However, two problems arise with tissue engineering the source of the cells and the design of the scaffold aimed at creating a microenvironment to guide cells toward tissue regeneration.

"The choice of cells for skin tissue engineering is vital to the outcome of the healing process," said Dr. Ulrich. "This study used mouse models and eschar tissues excised between 11 and 26 days after burn injury. The delay allowed time for partial thickness burns to heal, a process that is a regular treatment option in the Netherlands and rest of Europe."

The researchers speculated that during this time the severely damaged tissues could attract stem cells from the surrounding tissues, as elevated levels of MSCs have been detected in the blood of burn victims.

"MSCs can only be beneficial to tissue regeneration if they differentiate into types locally required in the wound environment," noted Dr. Ulrich. "We concluded that eschar-derived MSCs represent a population of multipotent stem cells. The origin of the cells remains unclear, but their resemblance to adipose-derived stem cells could be cause for speculation that in deep burns the subcutaneous adipose tissue might be an important stem cell source for wound healing."

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“Master Molecule” May Help Heart Treatment

Posted: July 12, 2012 at 4:15 am

Johns Hopkins researchers have discovered that a single protein molecule may hold the key to turning cardiac stem cells into blood vessels or muscle tissue, according to a release from the university. This finding may lead to better ways to treat heart attack patients.

Human heart tissue typically forms scars rather than healing well after an attack. However, stem cells have been shown improve the repair process by turning into the cells that make up healthy heart tissue, including heart muscle and blood vessels. The recent discovery of a master molecule that guides the destiny of these stem cells has the potential to result in even more effective treatments for heart patients, the Johns Hopkins researchers say.

In a study published in the June 5 online edition of journal Science Signaling, the Johns Hopkins team reported that tinkering with a protein molecule called p190RhoGAP shaped the development of cardiac stem cells and prodded them to become the building blocks for either blood vessels or heart muscle. The scientists said that by altering levels of this protein, they were able to affect the future of these stem cells. In biology, finding a central regulator like this is like finding a pot of gold, said Andre Levchenko, a biomedical engineering professor and member of the Johns Hopkins Institute for Cell Engineering, who supervised the research effort.

The lead author of the journal article, Kshitiz, a postdoctoral fellow who uses only his first name, said, Our findings greatly enhance our understanding of stem cell biology and suggest innovative new ways to control the behavior of cardiac stem cells before and after they are transplanted into a patient. This discovery could significantly change the way stem cell therapy is administered in heart patients.

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"Master Molecule" May Help Heart Treatment

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Verastem Acquires Clinical-Stage FAK Inhibitor from Pfizer

Posted: July 12, 2012 at 4:15 am

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

Verastem, Inc., (VSTM) a biopharmaceutical company focused on discovering and developing drugs to treat breast and other cancers by targeting cancer stem cells, announced an agreement with Pfizer for the exclusive in-license of worldwide commercial rights for VS-6063 (formerly PF-04554878), a focal adhesion kinase (FAK) inhibitor that has completed a Phase 1 clinical study in advanced solid tumors.

FAK is a non-receptor tyrosine kinase that regulates tumor cell proliferation and invasion. The targeted disruption of this pathway in preclinical models of cancer reduces cancer stem cells, primary tumor mass and metastasis.

Verastem has identified the FAK pathway as a critical regulator of the survival of cancer stem cells, which are an underlying cause of cancer recurrence and metastasis, said Robert Weinberg, Ph.D., Verastem co-founder and chair of the Scientific Advisory Board.

VS-6063 is being developed for the treatment of solid tumors. According to data presented at ASCO 2011 from a Phase 1 safety study of VS-6063 in 36 patients conducted by Pfizer, VS-6063 was well-tolerated and demonstrated signs of clinical activity to support further development. Verastem anticipates conducting clinical trials targeting solid tumor indications with VS-6063.

Like Pfizer, Verastem is committed to bringing innovative treatments to patients with cancer, said Garry Nicholson, President and General Manager of Pfizer Oncology. Verastems specific focus on targeting cancer stem cells makes them the ideal company to continue the development of this compound.

Under the terms of the agreement, Verastem will assume sole responsibility for global product development of VS-6063. Pfizer will receive an upfront payment in cash and Verastem equity, development milestones and royalties and milestones on future sales of VS-6063.

VS-6063 accelerates Verastems FAK inhibitor program with a clinical, Phase 2-ready product candidate targeting this key regulatory pathway for cancer stem cells, said Christoph Westphal, M.D., Ph.D., Chairman and Chief Executive Officer of Verastem. We believe our focus on identifying patients with a high cancer stem cell burden for treatment with our targeted therapies uniquely positions Verastem to lead the next wave of therapeutics in cancer.

Conference Call and Webcast Information Verastem will discuss the acquisition during the Research and Development Day to be held tomorrow, July 12, at 9:00am ET. A live webcast of the event can be accessed by visiting the investors section of the Companys website at http://www.verastem.com. A replay will be available for two weeks from the date of the event.

A live, listen-only conference call of the event can be accessed by dialing 1-866-700-7173 five minutes prior to the start of the event and providing the passcode 73322380.

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Verastem Acquires Clinical-Stage FAK Inhibitor from Pfizer

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"Master Molecule" May Help Heart Treatment

Posted: July 11, 2012 at 6:14 pm

Johns Hopkins researchers have discovered that a single protein molecule may hold the key to turning cardiac stem cells into blood vessels or muscle tissue, according to a release from the university. This finding may lead to better ways to treat heart attack patients.

Human heart tissue typically forms scars rather than healing well after an attack. However, stem cells have been shown improve the repair process by turning into the cells that make up healthy heart tissue, including heart muscle and blood vessels. The recent discovery of a master molecule that guides the destiny of these stem cells has the potential to result in even more effective treatments for heart patients, the Johns Hopkins researchers say.

In a study published in the June 5 online edition of journal Science Signaling, the Johns Hopkins team reported that tinkering with a protein molecule called p190RhoGAP shaped the development of cardiac stem cells and prodded them to become the building blocks for either blood vessels or heart muscle. The scientists said that by altering levels of this protein, they were able to affect the future of these stem cells. In biology, finding a central regulator like this is like finding a pot of gold, said Andre Levchenko, a biomedical engineering professor and member of the Johns Hopkins Institute for Cell Engineering, who supervised the research effort.

The lead author of the journal article, Kshitiz, a postdoctoral fellow who uses only his first name, said, Our findings greatly enhance our understanding of stem cell biology and suggest innovative new ways to control the behavior of cardiac stem cells before and after they are transplanted into a patient. This discovery could significantly change the way stem cell therapy is administered in heart patients.

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Cord Blood Stem Cells Restore Toddler's Hearing

Posted: July 11, 2012 at 6:14 pm

A virus infection Stephanie Connor acquired during pregnancy put her unborn daughter at significant risk for brain damage and lifelong hearing loss.

"It was traumatic," said Connor, of LaBelle, Fl, after learning about her daughter's condition. "It was like mourning the loss of a child."

At age 1, baby Madeleine was completely deaf in her right ear and her hearing was severely lost in the left, said Connor. While a hearing aid helped to amplify some sounds for Madeleine, it would never fully repair the damage in her ear.

But a simple experimental procedure that Connor enrolled in for Madeleine may have restored her hearing and reversed her condition.

In January 2012, Madeleine, 2, became the first child to undergo an experimental hearing loss treatment through an FDA-approved trial at Memorial Hermann-Texas Medical Center that infused stem cells from her own banked cord blood into her damaged inner ear.

Within the last six months, Connor says she's seen a dramatic improvement in Madeleine's ability to hear.

"Before, when she would hear something she would look all around," Connor said. "But now we notice that she turns in the right direction of the sound."

Madeleine was also able to speak for the first time, Connor said.

For more than two decades, umbilical cord blood transplantation -- either by a baby's own cord blood or another's, depending on the type of procedure -- has been used to treat otherwise fatal diseases including blood disorders, immune diseases, and some types of cancers.

Infusing cord blood stem cells into the body may also have the potential to heal and regenerate damaged cells and tissues.

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Stem Cells Restore Toddler's Hearing

Posted: July 11, 2012 at 6:14 pm

A virus infection Stephanie Connor acquired during pregnancy put her unborn daughter at significant risk for brain damage and lifelong hearing loss.

"It was traumatic," said Connor, of LaBelle, Fl, after learning about her daughter's condition. "It was like mourning the loss of a child."

At age 1, baby Madeleine was completely deaf in her right ear and her hearing was severely lost in the left, said Connor. While a hearing aid helped to amplify some sounds for Madeleine, it would never fully repair the damage in her ear.

But a simple experimental procedure that Connor enrolled in for Madeleine may have restored her hearing and reversed her condition.

In January 2012, Madeleine, 2, became the first child to undergo an experimental hearing loss treatment through an FDA-approved trial at Memorial Hermann-Texas Medical Center that infused stem cells from her own banked cord blood into her damaged inner ear.

Within the last six months, Connor says she's seen a dramatic improvement in Madeleine's ability to hear.

"Before, when she would hear something she would look all around," Connor said. "But now we notice that she turns in the right direction of the sound."

Madeleine was also able to speak for the first time, Connor said.

For more than two decades, umbilical cord blood transplantation -- either by a baby's own cord blood or another's, depending on the type of procedure -- has been used to treat otherwise fatal diseases including blood disorders, immune diseases, and some types of cancers.

Courtesy Stephanie Connor

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