JUDY WOODRUFF: Next, to the 2012 Nobel Prizes. The first was awarded today for groundbreaking work in reprogramming cells in the body.

Ray Suarez looks at those achievements.

MAN: The Nobel Assembly at Karolinska Institute has today decided to award the Nobel Prize in Physiology or Medicine,2012 jointly to John B. Gurdon and Shinya Yamanaka.

RAY SUAREZ: The two scientists are from two different generations and celebrated today's announcement half-a-world apart.

But today they were celebrated together for their research that led to a groundbreaking understanding of how cells work.

Sir John Gurdon of CambridgeUniversity was awarded for his work in 1962. He was able to use specialized cells of frogs, like skin or intestinal cells, to generate new tadpoles and show DNA could drive the formation of all cells in the body.

Forty years later, Dr. Yamanaka built on that and went further. He was able to turn mature cells back into their earliest form as primitive cells. Those cells are in many ways the equivalent of embryonic stem cells, because they have the potential to develop into specialized cells for heart, liver and other organs.

Dr. Shinya Yamanaka is currently working at KyotoUniversity. Embryonic stem cells have had to be harvested from human embryos, a source of debate and considerable controversy.

For Gurdon, the prize had special meaning. At a news conference in London, he recalled one schoolteacher's reaction to his desire to study science.

JOHN GURDON, co-winner, Nobel Prize For Medicine or Physiology: It was a completely ridiculous idea because there was no hope whatever of my doing science, and any time spent on it would be a total waste of time, both on my part and the part of the person having to teach him. So that terminated my completely -- completely terminated my science at school.

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Stem Cell Scientists Gurdon and Yamanaka Win Nobel Prize in Medicine

John Gurdon (left) and Shinya Yamanaka showed how to reprogram cells into their embryonic states.

J. Player/Rex Features; Aflo/Rex Features

The discovery that cells can be reprogrammed to an embryonic-like state has won this years Nobel Prize in Physiology or Medicine for two leading lights of stem-cell research: John Gurdon and Shinya Yamanaka.

Reprogrammed cells regain pluripotency, the potential to differentiate into many mature cell types. Many researchers hope that cells created in this way will eventually be used in regenerative medicine, providing replacement tissue for damaged or diseased organs. The field has become one of the hottest in biology, but the prizewinners discoveries were not without controversy when they were made.

Gurdon, who is based at the Gurdon Institute in Cambridge, UK, was the first person to demonstrate that cells could be reprogrammed, in work published 50years ago1. At the time, scientists believed that cellular specialization was a one-way process that could not be reversed. Gurdon overturned that dogma by removing the nucleus from a frog egg cell and replacing it with the nucleus from a tadpoles intestinal cell. Remarkably, the process was able to turn back the cellular clock of the substitute nucleus. Although it had already committed to specialization, inside the egg cell it acted like an eggs nucleus and directed the development of a normal tadpole.

Gurdon was a graduate student at the University of Oxford, UK, when he did the work. He received his doctorate in 1960 and went on to do a postdoc at the California Institute of Technology in Pasadena, leaving his frogs in Europe. He did not publish the research until two years after he got his PhD, once he was sure that the animals had matured healthily. I was a graduate student flying in the face of [established] knowledge, he says. There was a lot of scepticism.

Mammalian cells did not prove as amenable to this process, known as cloning by nuclear transfer, as frog cells. It was nearly 35years before the first cloned mammal Dolly the sheep was born, in 1996. Dolly was the only live birth from 277 attempts, and mammalian cloning remained a hit-and-miss affair.

Scientists were desperate to improve the efficiency of the system and to understand the exact molecular process involved. That is where Shinya Yamanaka of Kyoto University, Japan, made his mark. Yamanaka who was born the year that Gurdon published his formative paper used cultured mouse cells to identify the genes that kept embryonic cells immature, and then tested whether any of these genes could reprogram mature cells to make them pluripotent.

In the mid-2000s, the stem-cell community knew that Yamanaka was close. I remember when he presented the data at a 2006 Keystone symposium, says Cdric Blanpain, a stem-cell biologist at the Free University of Brussels. At that time he didnt name them and everyone was betting what these magic factors could be.

A few months later, attendees at the 2006 meeting of the International Society for Stem Cell Research in Toronto, Canada, packed out Yamanakas lecture. The audience waited in silence before he announced his surprisingly simple recipe: activating just four genes was enough to turn adult cells called fibroblasts back into pluripotent stem cells2. Such induced pluripotent stem (iPS) cells could then be coaxed into different types of mature cell types, including nerve and heart cells.

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Cell rewind wins medicine Nobel

10/5/2012 6:38 AM ET (RTTNews) - Amyotrophic Lateral Sclerosis is a progressive neurodegenerative disease that attacks nerve cells in the brain and spinal cord, leading to complete paralysis, and eventually, death. Also known as Lou Gehrig's disease, Amyotrophic Lateral Sclerosis, or ALS, is said to affect as many as 30,000 Americans, with 5,600 new cases being diagnosed each year.

Currently, there are two FDA-approved drugs to treat ALS namely, Sanofi-Aventis' (SNY:Quote) Riluzole, which prolongs life by 2-3 months, and Avanir Pharmaceuticals Inc.'s (AVNR:Quote) Nuedexta, which treats emotional instability that accompanies this disease.

Developing a neural stem cell therapy for ALS is Rockville, Maryland-based biotechnology company Neuralstem Inc. (CUR:Quote).

For readers who are new to Neuralstem, here's a brief overview of the company's pipeline and the upcoming events to watch out for...

The company is testing its cell product - NSI-566 human spinal cord stem cells, via transplantation technique, in the treatment of ALS symptoms. The phase I NSI-566 study was completed as recently as August of this year. This groundbreaking trial, the first to be approved by the FDA to test neural stem cells in patients with ALS, began in January 2010.

The trial was designed to enroll up to 18 patients, the last of which was treated in August of this year. The entire trial concludes six months after the final surgery.

The interim data on the NSI-566 ALS trial will be updated on October 8, 2012, according to the company.

NSI-566 will also be evaluated in treating motor deficits due to ischemic stroke. The company has received approval to commence a combined phase I/II ischemic stroke trial with NSI-566 in China, and it is expected to begin early next year.

The trial is designed to test up to 118 patients who have suffered an ischemic stroke with chronic residual motor disorder with NSI-566 cell line, 4-24 months post-stroke. The duration of the combined trial, including patient monitoring and data collection, is approximately two years.

Ischemic strokes, the most common type of stroke, occur as a result of an obstruction within a blood vessel supplying blood to the brain. After a stroke, many patients suffer from paralysis in arms and legs, which can be permanent.

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Walkthrough With Neuralstem

LOS ANGELES and LA JOLLA, Calif., Oct. 5, 2012 /PRNewswire/ -- A senior research neuroscientist from The Salk Institute,Mohamedi Kagalwala PhD, has been recruited to head NeuroGeneration's new laboratories in La Jolla, California. Dr. Kagalwala, an expert on neural stem cells, will become director of the new Division of Biotherapeutics and Drug Discovery.

"I am extremely pleased to lead NeuroGeneration's new Division and expand its technology of adult neural stem cells for Parkinson's disease. It will allow us to develop personalized iPS cell therapies for degenerative brain disorders," said Dr. Kagalwala. "In addition, by investigating intrinsic neurogenesis and brain repair mechanisms, our team will be able to modify discrete molecular mechanisms during aging and neurodegenerative changes. We will then be in a better position to influence the environment, either with drugs or cellular therapies, to prevent the progression of disease and facilitate brain repair."

This new Division will complement the neural stem cell therapy studies for Parkinson's disease and other Atypical Parkinsonism led by Dr. Michel Levesque, NeuroGeneration's scientific founder.

Within the new facility providing core state of the art technologies, NeuroGeneration will expand its bioinformatic platforms to include personalized neurogenomic, analysis for drug target discovery for aging, Parkinson's disease, Stroke, Amyotrophic Lateral Sclerosis, Alzheimer's disease, Multiple Sclerosis, Epilepsy, Depression and Schizophrenia.

ABOUT NEUROGENERATION:

NeuroGeneration is a life science company designing new cellular therapies and biological modulators for the prevention and treatment of neurodegenerative disorders. The company has completed a Phase I clinical trial for Parkinson's disease using adult-derived autologous neural stem cells. It intends to complete a Phase II study for the treatment of Parkinson's disease as soon as it receives final approval from the FDA. NeuroGeneration's Division of Biotherapeutics and Drug Discovery offers molecular products using its drug discovery platforms to target neuroprotective and endogenous repair mechanisms.

FOR MORE INFORMATION CONTACT:

NeuroGeneration Laboratories Division of Biotherapeutics and Drug Discovery 3210 Merryfield Row San Diego, CA92121

Patricia Eastman NeuroGeneration,Inc 8670 Wilshire Blvd, Suite 201 Los Angeles, CA 90211 USA Tel.:1-310-659-3880 Email: info@neurogeneration.com http://www.neurogeneration.com

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NeuroGeneration Recruits Top Scientist To Direct New Division of Biotherapeutics and Drug Discovery In La Jolla, CA

Reaching a long-sought milestone, Japanese researchers have demonstrated in mice that eggs and sperm can be grown from stem cells and combined to produce healthy offspring, pointing to new treatments for infertility.

If the achievement can be repeated in humans and experts said they are optimistic that such efforts will ultimately succeed the technique could make it easier for women in their 30s or 40s to become mothers. It could also help men and women whose reproductive organs have been damaged by cancer treatments or other causes.

About one in 10 American women of childbearing age have trouble becoming or staying pregnant, and more than one-third of infertile couples must contend with a medical problem related to the prospective father, according to the national Centers for Disease Control and Prevention in Atlanta.

Using current technology, only about one-third of attempts at assisted reproduction result in live births, CDC data show. Scientists, doctors and patients would like to boost that percentage.

"These studies provide that next level of evidence that in the future fertility could be managed with stem cell intervention," said Teresa Woodruff, chief of fertility preservation at Northwestern University Feinberg School of Medicine.

The prospect of using stem cells to grow new eggs is particularly tantalizing, since women are born with a set number and don't make more once they are gone. In a sense, the therapy would allow them to turn back their biological clocks, said Stanford stem cell researcher Renee A. Reijo Pera, who studies reproduction.

"This is a get-them-back strategy," she said.

Dr. Mitinori Saitou and colleagues at Kyoto University detailed how they generated the functional mouse eggs in a report published online Thursday by the journal Science. Last year, the researchers reported in the journal Cell that they had done the same thing with mouse sperm.

In both cases, the team started with embryonic stem cells, which have the potential to develop into all of the different types of cells in the body.

The scientists exposed the embryonic stem cells to stimuli that coaxed them to become egg and sperm precursors.

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Mouse stem cells used to produce eggs, Japanese scientists say

Public release date: 17-Sep-2012 [ | E-mail | Share ]

Contact: Nicole White nicole.white@cshs.org 310-423-5215 Cedars-Sinai Medical Center

LOS ANGELES Sept. 17, 2012 Leading scientists and clinicians from across the nation will discuss the latest findings on potential stem cell treatments for diabetes and eye diseases at the second Cedars-Sinai Regenerative Medicine Scientific Symposium.

WHO: Stem cell scientists, clinicians and industry leaders.

The symposium is being hosted by the Cedars-Sinai Regenerative Medicine Institute, led by Clive Svendsen, PhD. The institute brings together basic scientists with specialist clinicians, physician scientists and translational scientists across multiple medical specialties to convert fundamental stem cell studies to therapeutic regenerative medicine.

FEATURED RESEARCH: The symposium's morning session will feature an overview of the current state of stem cells and diabetes, including efforts to start the first clinical trials with stem cells for the treatment of diabetes. Other research to be presented includes an update on regenerative medicine approaches to treating macular degeneration, a progressive deterioration of the eye that causes gradual loss of vision. This will include an update from Gad Heilweil , MD, on a key, stem-cell clinical trial on macular degeneration at the University of California Los Angeles.

WHEN: Sept. 21, 2012 8:30 a.m. to 6 p.m. Thomson's lecture begins at 8:40 a.m.

WHERE: Harvey Morse Auditorium Cedars-Sinai Medical Center 8700 Beverly Boulevard Los Angeles, CA 90048

How to register: http://www.cedars-sinai.edu/RMI

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Leading stem cell scientists to focus on diabetes, eye diseases at Cedars-Sinai symposium

ScienceDaily (Sep. 11, 2012) UCLA stem cell researchers have found that a gene therapy regimen can safely restore immune systems to children with so-called "Bubble Boy" disease, a life threatening condition that if left untreated can be fatal within one to two years.

In the 11-year study, researchers were able to test two therapy regimens for 10 children with ADA-deficient severe combined immunodeficiency (SCID). During the study, they refined their approach to include a light dose of chemotherapy to help remove many of the blood stem cells in the bone marrow that are not creating an enzyme called adenosine deaminase (ADA), which is critical for the production and survival of healthy white blood cells, said study senior Dr. Donald Kohn, a professor of pediatrics and of microbiology, immunology, and molecular genetics in Life Sciences and a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.

The refined gene therapy and chemotherapy regimen proved superior to the other method tested in the study, restoring immune function to three of the six children who received it, Kohn said. Going forward, an even further refined regimen using a different type of virus delivery system will be studied in the next phase of the study, which already has enrolled eight of the 10 patients needed.

The study appears Aug. 30 in the advance online issue of the peer-reviewed journal Blood.

"We were very happy that in the human trials we were able to see a benefit in the patients after we modified the protocol," Kohn said. "Doctors treating ADA-deficient SCID have had too few options for too long, and we hope this will provide them with an efficient and effective treatment for this devastating disease."

Children born with SCID, an inherited immunodeficiency, are generally diagnosed at about six months. They are extremely vulnerable to infectious diseases and don't grow well. Chronic diarrhea, ear infections, recurrent pneumonia and profuse oral candidiasis commonly occur in these children. SCID cases occur in about 1 of 100,000 births

Currently, the only treatment for ADA-deficient SCID calls for injecting the patients twice a week with the necessary enzyme, Kohn said, a life-long process that is very expensive and often doesn't return the immune system to optimal levels. These patients also can undergo bone marrow transplants from matched siblings, but matches can be very rare.

About 15 percent of all SCID patients are ADA-deficient. Kohn and his team used a virus delivery system that he had developed in his lab in the 1990s to restore the gene that produces the missing enzyme necessary for a healthy immune system. To date, about 40 children with SCID have received gene therapy in clinical trials around the world, Kohn said.

Two slightly different viral vectors were tested in the study, each modified to deliver healthy ADA genes into the bone marrow cells of the patients so the needed enzyme could be produced and make up for the cells that don't have the gene. Four of the 10 patients in the study remained on their enzyme replacement therapy during the gene therapy study. There were no side effects, but their immune systems were not sufficiently restored, Kohn said.

In the next six patients, the enzyme therapy was stopped and a small dose of chemotherapy was given before starting the gene therapy to deplete the ADA-deficient stem cells in their bone marrow. Of those patients, half had their immune systems restored. The human findings confirmed another study, also published recently in Blood by Kohn and UCLA colleague Dr. Denise Carbonaro-Sarracino, which tested the techniques in parallel, using a mouse model of ADA-deficient SCID.

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Stem cell researchers use gene therapy to restore immune systems in 'Bubble Boy' disease

Featured Article Academic Journal Main Category: Bones / Orthopedics Also Included In: Stem Cell Research Article Date: 23 Aug 2012 - 11:00 PDT

Current ratings for: Osteoporosis Clue Found In Stem Cell Signalling Protein

1 (1 votes)

These are the implications of a new study led by Harvard Medical School (HMS) that was published online in The Journal of Clinical Investigation on 13 August.

Senior author Bjorn Olsen, Hersey Professor of Cell Biology at HMS, told the press about what they found:

"It shifts the thinking about what controls the differentiation of stem cells to bone cells instead of fat cells, and how to make sure this mechanism stays active with aging."

Bone is not a dead material: it is living tissue that is changing all the time, as it is continuously formed and reabsorbed.

Osteoporosis is a common bone disease where bone tissue becomes progressively thinner, resulting in higher risk of fracture. It affects about 1 in 5 American women and is thought to be caused by stem cells that normally differentiate into bone-forming cells becoming fat cells instead over time.

For the study, Olsen, who is professor of developmental biology and dean for research at Harvard School of Dental Medicine, and colleagues, decided to investigate the role of vascular endothelial growth factor, or VEGF, a common signalling protein that plays a key role in the development of blood vessels that are important in early bone growth and skeletal maintenance in mammals. The protein works by activating receptors on the surface of cells.

Soon after they were born, the mice's skeletons began to show osteoporosis-like qualities, such as reduced bone tissue and a build up of fat in the bone marrow.

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Osteoporosis Clue Found In Stem Cell Signalling Protein

Peter Aldhous, San Francisco bureau chief

It's official: stem cells are drugs. At least, that's the opinion of the US district court in Washington DC, which has ruled that the Food and Drug Administration (FDA) has the authority to regulate clinics offering controversial stem cell therapies.

Treatments in which stem cells are harvested from bone marrow and injected straight back into the same patient are deemed part of routine medical practice - not regulated by the US government. But if the cells are subjected to more than "minimal manipulation", the FDA maintains that the therapy becomes a "drug", which must be specifically approved for use.

It was on this basis that in 2008 the FDA began moves to shut downRegenerative Sciences, a clinic in Broomfield, Colorado, that treats orthopaedic problems using a stem cell therapy called Regenexx.

Regenerative Sciences challenged the FDA's authority to regulate its activities, setting the stage for a legal fight. In 2010, the FDA sought an injunction to take Regenexx off the market. This has now been granted in the court's ruling.

Christopher Centeno, medical director of Regenerative Sciences, vows to appeal. "This is really round one," he says. "Our position remains that a patient's cells are not drugs."

Regenexx consists of mesenchymal stem cells, which give rise to tissues including bone and cartilage, taken from a patient's bone marrow and grown in culture for about two weeks. Centeno has published a series of case reports describing its use to treat joint problems - but no controlled clinical trials.

"I think it's a good ruling, and I'm glad to see that that the FDA has exercised its muscle on the case," says Christopher Scott, who heads the Program on Stem Cells in Society at Stanford University in California.

Scott hopes that the FDA will now step up its efforts to regulate other clinics offering unproven stem cell therapies. These include Celltex of Sugar Land, Texas, which rose to prominence after Texas governor Rick Perry was injected with stem cells supplied by the company to aid his recovery from back surgery.

Last month, the Houston Chronicle revealed that FDA inspectors had found multiple violations of good manufacturing practice at Celltex.

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Ruling frees FDA to crack down on stem cell clinics

A clinical trial of ALS patients conducted by BrainStorm Cell Therapeutics shows its adult stem cell therapy is well-tolerated, appears to be safe and does not present undue risk, according to an interim safety review.

Moreover, in some patients signs of stabilization of the disease were detected.

Israel-based BrainStorm is developing NurOwn for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's Disease, a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord.

"It's very uncommon to give at such an early point in a clinical study efficacy data, but we cannot ignore the fact on an individual basis we could see improvement in many of the patients involved, each one in different areas," Moshe Neuman, CEO of Biomedical Research Design, which serves as a contract research organization for the trial, said.

In some patients breathing improved, in others it was muscle strength and in others it was speech, he told Reuters.

Neuman said a final report was expected by the end of the year after each patient has been observed for nine months.

BrainStorm President Chaim Lebovits said the preliminary results demonstrate that the stem cells have the potential not only to stop deterioration but perhaps even cure ALS.

"The coming phases in the trial will have to prove this, but these results also reaffirm our belief that we have an enormous potential of being successful with less severe indications such as multiple sclerosis and Parkinson's," he said.

Patients in the trial were transplanted with stem cells derived from their own bone marrow and treated with the NurOwn stem cell technology.

The Phase I/II trial, designed to evaluate the safety and preliminary efficacy of BrainStorm's therapy, is being conducted at Jerusalem's Hadassah Medical Center. The company submitted the interim safety report to Israel's Health Ministry.

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Israel adult stem cell trials - hope for ALS patients?

Tuesday, July 03 16:25:12

Ireland has the capacity to be an international centre for commercialisation in the field of regenerative medicine, delegates at an international stem cell conference in NUI Galway heard today.

Reflecting this potential, new Irish company Orbsen Therapeutics is developing proprietary technologies designed to isolate stem cells. The NUI Galway spin-out is targeting the rapidly maturing and expanding regenerative medicine market, which is expected to grow to $118 billion next year.

Frank Barry is Professor of Cellular Therapy at NUI Galway, Director of Orbsen Therapeutics, and organiser of the Mesenchymal Stem Cell Conference, which opened yesterday.

Mesenchymal stem cells (MSCs) are a type of adult stem cell, and this event brings together the world's leading scientists in the field to discuss their latest ideas and findings. This is the first major stem cell conference to take place in Ireland, and is looking at all aspects of adult stem cells, from basic biology to manufacturing to clinical trials and therapeutics.

Stem cells hold great promise as an alternative to drugs and surgical procedures for treating a wide range of medical conditions including heart disease, arterial disease of the limbs, diabetes complications, arthritis and other inflammatory conditions. The treatment potential of stem cells is linked to their natural capacity to dampen inflammation and promote healing, repair and regeneration of damaged tissues.

According to Professor Barry: "Ireland has a strong research base in adult stem cell therapy and has the capcacity for advanced stem cell bioprocessing. There is huge potential in this market and we anticipate that there will be extraordinary growth over the next 5-10 years. There are currently over 400 regenerative medicine products on the market with many more in development." Orbsen Therapeutics has developed a clear pipeline of clinical indications which they hope, using their proprietary technologies, to bring through to clinical trial over the coming years. These include osteoarthritis, acute lung injury syndrome, diabetic foot ulcer, critical limb ischemia and others."

"Combining the utility, novelty and the value of its technologies, Orbsen is well placed to take advantage of the many opportunities in this fast moving and important emerging market", said Brian Molloy, CEO of Orbsen Theraepeutics."

Orbsen Therapeutics Limited was formed as a spin out company to develop and commercialise new intellectual property built up by researchers at the SFI-funded Regenerative Medicine Institute (REMEDI) at NUI Galway.

Scientists at NUI Galway are investigating how adult stems cells might be used to develop new treatments for vascular disease, osteoarthritis and lung injury. The University has become a leading centre of translational research in adult stem cells involving its National Centre for Biomedical Engineering Science (NCBES) and REMEDI.

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Ireland could be stem cell research hub

Public release date: 2-Jul-2012 [ | E-mail | Share ]

Contact: Sarah Jackson press_releases@the-jci.org Journal of Clinical Investigation

In Parkinson's disease, the loss of dopamine-producing cells in the midbrain causes well-characterized motor symptoms. Though embryonic stem cells could potentially be used to replace dopaminergic (DA) neurons in Parkinson's disease patients, such cell therapy options must still overcome technical obstacles before the approach is ready for the clinic. Embryonic stem cell-based transplantation regimens carry a risk of introducing inappropriate cells or even cancer-prone cells. To develop cell purification strategies to minimize these risks, Dr. Lorenza Studer and colleagues at Memorial Sloan Kettering Cancer Center in New York developed three different mouse lines to fluorescently label dopaminergic neurons at early, mid, and late stages of differentiation. Their data suggest that mouse embryonic stem cells induced to the mid stage of neuronal differentiation are best suited for transplantation to replace dopaminergic neurons. Further, their work identified new genes associated with each stage of neuronal differentiation. Their results in the mouse model system help define the differentiation stage and specific attributes of embryonic stem cell-derived, dopamine-generating cells that hold promise for cell therapy applications.

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TITLE:

Identification of embryonic stem cellderived midbrain dopaminergic neurons for engraftment

AUTHOR CONTACT:

Lorenz Studer

Memorial Sloan Kettering Cancer Center, New York, NY, USA

Phone: 212.639.6126; E-mail: studerl@mskcc.org

Original post:
Generating dopamine via cell therapy for Parkinson's disease

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.

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

Public release date: 2-Jul-2012 [ | E-mail | Share ]

Contact: Kim Newman sciencenews@einstein.yu.edu 718-430-3101 Albert Einstein College of Medicine

July 2, 2012 -- (Bronx, NY) -- Researchers at Albert Einstein College of Medicine of Yeshiva University have found that abnormal bone marrow stem cells drive the development of myelodysplastic syndromes (MDS), serious blood diseases that are common among the elderly and that can progress to acute leukemia. The findings could lead to targeted therapies against MDS and prevent MDS-related cancers. The study is published today in the online edition of the journal Blood.

"Researchers have suspected that MDS is a 'stem cell disease,' and now we finally have proof," said co-senior author Amit Verma, M.B.B.S., associate professor of medicine and of developmental and molecular biology at Einstein and attending physician in oncology at Montefiore Einstein Center for Cancer Care. "Equally important, we found that even after MDS standard treatment, abnormal stem cells persist in the bone marrow. So, although the patient may be in remission, those stem cells don't die and the disease will inevitably return. Based on our findings, it's clear that we need to wipe out the abnormal stem cells in order to improve cure rates."

MDS are a diverse group of incurable diseases that affect the bone marrow and lead to low numbers of blood cells. While some forms of MDS are mild and easily managed, some 25 to 30 percent of cases develop into an aggressive disease called acute myeloid leukemia. Each year, about 10,000 to 15,000 people in the U.S. are diagnosed with MDS, according to the National Marrow Donor Program.

Most cases of MDS occur in people over age 60, but the disease can affect people of any age and is more common in men than women. Symptoms vary widely, ranging from anemia to infections, fever and bleeding. Treatment usually involves chemotherapy to destroy abnormal blood cells plus supportive care such as blood transfusions.

In the current study, lead author Britta Will, Ph.D., research associate in the department of cell biology, and her colleagues analyzed bone marrow stem cells and progenitor cells (i.e., cells formed by stem cells) from 16 patients with various types of MDS and 17 healthy controls. The stem and progenitor cells were isolated from bone marrow using novel cell-sorting methods developed in the laboratory of co-senior author Ulrich Steidl, M.D., Ph.D., assistant professor of cell biology and of medicine and the Diane and Arthur B. Belfer Faculty Scholar in Cancer Research at Einstein.

Genome-wide analysis revealed widespread genetic and epigenetic alterations in stem and progenitor cells taken from MDS patients, in comparison to cells taken from healthy controls. The abnormalities were more pronounced in patients with types of MDS likely to prove fatal than in patients with lower-risk types.

"Our study offers new hope that MDS can be more effectively treated, with therapies that specifically target genes that are deregulated in early stem and progenitor cells," said Dr. Steidl. "In addition, our findings could help to detect minimal residual disease in patients in remission, allowing for more individualized treatment strategies that permanently eradicate the disease."

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Myelodysplastic syndromes (MDS) linked to abnormal stem cells

The laboratory grew and stored human stem cells, which are capable of becoming any cell in the body, and made them available to scientists nationwide for use in experiments to study diseases such as diabetes and spinal cord injuries. When it is dismantled, several thousand vials of stem cellswill be sent back to the research centers where they originated, and the equipment will be given to other UMass labs.

Susan Windham-Bannister, president of the Massachusetts Life Sciences Center, a quasi-public agency that oversees the $1 billion life sciences initiative, defended the decision to initially fund the stem cell bank. She said there are many examples of technology that in hindsight are unnecessary, but at the time it was conceived, when the investment was made, it was absolutely state of the art. The center, she said, was one of them.

Originally, the bank was seen as a repository for embryonic stem cell lines that were being created but were not eligible for federal funding under Bush-era restrictions. The field has evolved significantly since then, with President Obamas loosening of restrictions on federal funding and the development of new technologies for making stem cells.

Still, stem cell banks are seen as useful by some. The California Institute for Regenerative Medicine, for example, is preparing to invest $10 million in its own stem cell banking initiative, and another $20 million to underwrite the creation of stem cells from patients with specific diseases.

Massachusetts Senate minority leader Bruce Tarr, Republican of Gloucester, said he was concerned that lawmakers had not been told the bank would close.

Given the fact that this is a resource that was created by an act of the Legislature, I would hope anyone seeking to change its status would consult with the Legislature, he said. The notion has always been we have been working hard to make Massachusetts a leader in stem cell research, and I dont know how ceasing the operations of the stem cell bank advances that goal.

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UMass stem cell lab to close

WWL-TVs Sally-Ann Roberts talks about her sisters health battle. Watch Richard Ford on The Colbert Report. TV tweet of the day so far.

TV Linkzilla Daily for 6/27/12 starts now.

When Good Morning America anchor Robin Roberts announced June 11 that shed been diagnosed with a rare form of bone-marrow cancer MDS, or myelodysplastic syndrome -- and would undergo chemotherapy and a bone-marrow transplant, her sister, WWL-TV anchor Sally-Ann Roberts, was cast in a key recovery role.

Sally-Ann Roberts, it turned out, was a perfect match to be a bone-marrow-cell donor for her sister. The New Orleans anchors medical contribution wont come for weeks or perhaps months, but shes already begun efforts to raise awareness of the need for donors.

Her station has launched a Perfect Match Supporting Sally-Ann & Robin page on its website, which now holds several stories on the topic, including a Wednesday (June 27) piece in which WWL staffers sign up to join a bone-marrow registry.

An informational and registration phone-bank, staffed by volunteers from organ- and bone-marrow-donation organizations, will operate from 6-9 p.m. Thursday (June 28), in coordination with the stations morning news block.

In a recent interview, Sally-Ann Roberts said her match is a real blessing, because only 25 percent of people who need a bone-marrow transplant actually find a match among their siblings.

She continued:

The majority of people who need a bone marrow donor have to go outside of their family in order to find one. Sometimes it's like a needle in a haystack, and that's why Robin wants to use this challenge that she's facing right now to try to bring attention to the national narrow donor registry. Millions of people are part of it. If a person -- man woman or child -- is in search of a bone marrow donor they can go to this registry and have a chance to find one. There are many, many people who have used the registry successfully.

The only problem is that minorities are underrepresented in the donor registry. Unlike organ donations, where it really doesn't matter what the ethnic background of an individual is for an organ donation, bone marrow for stem cells has to be aligned with the person's genetic makeup. And that's why if you're African American you will find a match with another African American. Native Americans, the same thing. So that's why every racial group needs to be represented in the marrow-donor registry. That's what were trying to do. Were trying to direct people to BeTheMatch.org. If they do, they'll get a packet in the mail and will be able to do a swab, just the inside of their cheek, and will get a self-addressed stamped envelope. They mail it back with the required information. They may get a phone call, they may never get a phone call. But people who have done so -- I've gone online to listen to some of their stories -- they feel so grateful that they were able to reach out and help another individual in need.

Original post:
WWL-TV and Sally-Ann Roberts rally support for bone-marrow donor registry

A mother with three daughters who have Fanconi anemia sued the federal government for the right to compensate bone marrow donors. The U.S. Attorney General will not pursue the case with the Supreme Court, thus making a lower court's ruling law. That means bone marrow donors may now receive vouchers worth up to $3,000. NBC's Dr. Nancy Snyderman reports.

By JoNel Aleccia

Certain bone marrow donors could soon be compensated for their life-saving stem cells after federal officials declined to take the matter to the U.S. Supreme Court, allowing a lower court order to become law.

At least one agency, MoreMarrowDonors.org, hopes to begin a pilot program offering up to $3,000 in scholarships, housing vouchers or charity donations -- but not cash -- in exchange for matching donations of marrow cells derived from blood.

This decision is a total game-changer, said Jeff Rowes, a senior attorney with the Institute for Justice, which filed the lawsuit three years ago on behalf of cancer victims and others seeking bone marrow matches. Any donor, any doctor, any patient across the country can use compensation in order to get bone marrow donors.

That may be the effect of the decision by U.S. Attorney General Eric Holder to forgo a high court review of a 9th U.S. Circuit Court of Appeals ruling that certain kinds of bone marrow donations are exempt from federal rules banning compensation.

Under the ruling, donors who provide marrow cells through a process similar to blood donation, called peripheral blood stem cell apheresis, can be compensated because those cells are no longer regarded as organs or organ parts as defined in the National Organ Transplant Act.

The ruling does not apply, however, to bone marrow obtained through traditional techniques that use a needle to aspirate the cells from the hip.

Although it applies only to nine states covered by the 9th Circuit Court, Rowes expects the effects to be felt nationwide.

The move met with praise from Doreen Flynn, 36, of Lewiston, Maine, the lawsuits namesake and the single mother of three daughters with an incurable blood disorder called Fanconi anemia.

The rest is here:
Bone marrow donors soon may be compensated

The Sugar Land company involved in Gov. Rick Perry's unlicensed adult stem-cell procedure is rife with basic manufacturing problems, according to the U.S. Food and Drug Administration.

In a report one expert called a blow to the entire adult stem-cell industry, the FDA found that Celltex Therapeutics Corp. cannot guarantee the sterility, uniformity and integrity of stem cells it takes from people and then stores and grows for eventual therapeutic reinjection.

"You have not performed a validation of your banking and thawing process to assure viability" of the stem cells, reads the April 27 report, meaning that the company cannot verify the cells are alive.

The FDA report, which followed an April 16-27 inspection of Celltex, was released under the Freedom of Information Act Monday to the Houston Chronicle and a University of Minnesota bioethicist who complained in February that Celltex is a potential danger to patients and not in compliance with federal law.

The report, partially redacted, was not accompanied by a warning letter.

A former FDA official who read it, however, said the deficiencies - 79 in all, from incorrectly labeled products to failed sterility tests - are so serious that Celltex risks being shut down if it does not remedy the problems quickly. The former official asked not to be identified.

Adult stem cells are cells in the body that multiply to replenish dying cells. Long used to treat leukemia and other cancers, they have shown promise for tissue repair in many other diseases in the last decade, although most scientists in the field consider them not ready for mainstream use.

Rules take effect Friday

Celltex has been in the public eye since it was revealed that Perry's Houston doctor treated him with his own stem cells during back surgery last July and in follow-up appointments. His stem cells were stored and grown at Celltex.

Perry subsequently called for Texas to become the nation's leader of adult stem cell medicine, which he touts as an ethical alternative to embryonic stem cells. Perry worked with his Houston doctor and a state representative to write legislation intended to commercialize the therapy in Texas.

Originally posted here:
FDA report faults Houston-area stem-cell company

23-06-2012 01:57 I have become so worried about my health because without health I will never get to experience the happiness of falling in love. I now do a Roberg test after every gym workout along with a couple of other tests. I also found out my cousin may be having his pacemaker removed from his heart because there is a surgery to correct his heart without the need for this implanted machine. He has had a pacemaker since his early 20s. It is a possibility I could have what my cousin has and that could possibly explain some of my symptoms. I do feel many doctors in Colombia will do a better job at diagnosing and treating someone with my symptoms. I am seriously considering going for examinations in Colombia and may even seek out treatment there or in another country. I do believe a radical and aggressive approach to both my physical and mental health will definitely enable me to find love in life and make massive amount of friends to share my activities with. I get so jealous of seeing guys in the gym with their girlfriend and friends. I just want to be happy and loved. I basically believe I can still be salvaged!

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Romberg Test/ Cardiac Pacemaker Removal?/Finding Love in Life/Stem Cell Treatment - Video

Stem cell therapy has gone to the dogs. The technology aimed at giving ailing pets a new lease on life has arrived in Hawaii.

13-year-old Kumba is still a bit dazed, coming out of general anesthesia. The veterinarian at Surf Paws Animal Hospital just extracted about two tablespoons of fat tissue from the dog. Stem cells from that fat tissue will then be used to help him with his arthritis.

"Once we get the stem cells then we do some extra processing steps to wake them up so that they're very active. At the end of that, the veterinarian will inject the stem cells into the areas of damage," says Carol Spangler Vaughn of Medivet America.

A company called MediVet America is bringing the technology to animal hospitals in Hawaii. This is a first for Oahu. The company says the procedure works on other animals with different types of ailments.

"So the nice thing about this we're not gonna give you a puppy back but we'll give you some nice quality time with your animal. You won't have to put them down because of their arthritis," Vaughn said.

Kumba's arthritis had gotten worse in the past five years, and his owners were wondering whether it was best to end his life to stop him from suffering.

'When we start saying things like oh we don't know how much longer, poor Kumba, he must be in a lot of pain. That kind of stuff really hits home especially since he's been with us for so long," said Rumi Hospodar Kumba's owner.

But with this new procedure, they're counting on Kumba to be pain free in a few weeks and are looking forward to get backdoing some of the things Kumba enjoyed, like swimming.

"He can't do that now since his joints are so bad, and he's getting so old so that's one of the many things I'm looking forward to," Kelsea Hopsodar, his other owner said.

The cost of the procedure runs from 24 to 28 hundred dollars, and it's covered by most pet insurance policies.

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Stem cell therapy gives dog new lease on life