BrainStorm's NurOwn Cell Therapy Receives Orphan Drug Designation in the European Union for ALS

NEW YORK, NY and PETAH TIKVA, ISRAEL--(Marketwired - Jul 29, 2013) - BrainStorm Cell Therapeutics (OTCQB: BCLI), a leading developer of adult stem cell technologies for neurodegenerative diseases, today announced that the European Commission has granted Orphan Drug Designation for NurOwn, the Company's stem cell therapy consisting of autologous bone marrow-derived mesenchymal stromal cells secreting neurotrophic factors, for the treatment of Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's Disease.NurOwn received Orphan Drug Designation from the Food and Drug Administration (FDA) in 2011.

Orphan drugs benefit from 10 years market exclusivity in the European Union (EU) after marketing approval. Additional benefits for sponsor companies include reduced fees for various centralized activities including applications for marketing authorization, inspections and protocol assistance, as well as possible eligibility for EU grants and other R&D-supporting initiatives.

BrainStorm is currently conducting a Phase IIa dose-escalating trial with 12 ALS patients at the Hadassah Medical Center in Jerusalem, Israel. The company anticipates launching a Phase II multi-center trial at three leading institutions in the United States towards the end of 2013, pending FDA approval.

About BrainStorm Cell Therapeutics, Inc.BrainStorm Cell Therapeutics Inc. is a biotechnology company engaged in the development of first-of-its-kind adult stem cell therapies derived from autologous bone marrow cells for the treatment of neurodegenerative diseases. The Company holds the rights to develop and commercialize its NurOwn technology through an exclusive, worldwide licensing agreement with Ramot, the technology transfer company of Tel Aviv University. For more information, visit the company's website at http://www.brainstorm-cell.com.

Safe Harbor Statement - Statements in this announcement other than historical data and information constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements.The potential risks and uncertainties include, without limitation, risks associated with BrainStorm's limited operating history, history of losses; minimal working capital, dependence on its license to Ramot's technology; ability to adequately protect the technology; dependence on key executives and on its scientific consultants; ability to obtain required regulatory approvals; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at http://www.sec.gov.These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements.The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

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BrainStorm's NurOwn Cell Therapy Receives Orphan Drug Designation in the European Union for ALS

Bank on it? A $60,000 bet on stem-cell cures

Forget about cash-start banking your body.

Using a technology that echoes science fiction movies, a French company is offering people a "bank" to store their own stem cells for years in a bet that those cells may be used to grow replacement organs and possibly save their lives someday.

"It's a personal deposit of your cell," said Dr. Andr Choulika, CEO of the biotech company Cellectis, which is launching the stem-cell bank known as Scil this month.

Given the rate of developments in stem-cell research and their potential for curbing health costs, Choulika thinks Scil's model of creating and storing stem cells one day will become so cheap that it will become mandatory for people with medical insurance.

"This pace of science currently goes so fast, probably in a few years, less than 10 years, everyone will have their own cell backup, and it will be a requirement," said Choulika, whose company is named after the Gaelic word for "story," and is pronounced "sail" by Choulika.

"We believe that regenerative medicine is the future," he said.

In the present, though, it is anything but cheap. Scil's services cost $60,000, Choulika said, adding that it is "a one-time payment, for your life."

(Read more: Hot biotech IPOs )

Choulika said he expects enthusiastic adoption by wealthy clients, as has been seen with other new technologies. Then, system improvements and competition could drive the initial price down considerably.

"It's expensive, or it seems expensive, for one reason," Choulika said. "We don't know how many people are going to sign up, and it's a very complex and sophisticated technology," he said. The number of people now who could both afford the process and be interested in trying it might top 1 million.

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Bank on it? A $60,000 bet on stem-cell cures

Big bet on future stem-cell cures

Forget about cashstart banking your body.

Using a technology that echoes science fiction movies, a French company is offering people a "bank" to store their own stem cells for years in a bet that those cells may be used to grow replacement organs and possibly save their lives someday.

"It's a personal deposit of your cell," said Dr. Andr Choulika, CEO of the biotech company Cellectis, which is launching the stem-cell bank known as Scil this month.

Given the rate of developments in stem-cell research and their potential for curbing health costs, Choulika thinks Scil's model of creating and storing stem cells one day will become so cheap that it will become mandatory for people with medical insurance.

"This pace of science currently goes so fast, probably in a few years, less than 10 years, everyone will have their own cell backup, and it will be a requirement," said Choulika, whose company is named after the Gaelic word for "story," and is pronounced "sail" by Choulika.

"We believe that regenerative medicine is the future," he said.

In the present, though, it is anything but cheap. Scil's services cost $60,000, Choulika said, adding that it is "a one-time payment, for your life."

(Read more: Hot biotech IPOs)

Choulika said he expects enthusiastic adoption by wealthy clients, as has been seen with other new technologies. Then, system improvements and competition could drive the initial price down considerably.

"It's expensive, or it seems expensive, for one reason," Choulika said. "We don't know how many people are going to sign up, and it's a very complex and sophisticated technology," he said. The number of people now who could both afford the process and be interested in trying it might top 1 million.

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Big bet on future stem-cell cures

Christiano fights for life after operation

WA Derby winner Alta Christiano is fighting for his life after suffering an adverse reaction to stem-cell treatment.

The Gary Hall Sr-trained star has been closely monitored at the Lark Hill Veterinary Clinic since the procedure two weeks ago.

Connections opted for the treatment to repair Alta Christiano's near fore suspensory ligament, which he tore after a Byford trial win in May.

The three-year-old is unbeaten from three starts in WA after a syndicate of Hall's clients bought an 80 per cent share in him from New Zealand owners.

He scored a brilliant win over Macha in the $200,000 WA Derby (2536m) at Gloucester Park in April.

"We don't know if he'll pull through, but we're hoping," Hall said.

"He's still under constant treatment. We're not sure if he picked up an infection or had a bad reaction to the stem-cell treatment."

Hall is hoping for a change of fortune at Gloucester Park tonight.

He has Real Hammer and Heez Orl Black taking on smart three-year-old Condrieu in the feature race, the $35,000 John Higgins Memorial (2130m).

The Greg and Skye Bond-trained Condrieu, a winner of three of his past five starts, is the one to beat from barrier three.

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Christiano fights for life after operation

Epilepsy in a Dish

Stem cell research reveals clues to disease's origins and possible treatment.

Epilepsy in a dish: Stem cell research reveals clues to disease's origins and possible treatment

U-M-led study of neurons created from skin of patients with Dravet syndrome

A new stem cell-based approach to studying epilepsy has yielded a surprising discovery about what causes one form of the disease, and may help in the search for better medicines to treat all kinds of seizure disorders.

The findings, reported by a team of scientists from the University of Michigan Medical School and colleagues, use a technique that could be called "epilepsy in a dish".

By turning skin cells of epilepsy patients into stem cells, and then turning those stem cells into neurons, or brain nerve cells, the team created a miniature testing ground for epilepsy. They could even measure the signals that the cells were sending to one another, through tiny portals called sodium channels.

In neurons derived from the cells of children who have a severe, rare genetic form of epilepsy called Dravet syndrome, the researchers report abnormally high levels of sodium current activity. They saw spontaneous bursts of communication and "hyperexcitability" that could potentially set off seizures. Neurons made from the skin cells of people without epilepsy showed none of this abnormal activity.

They report their results online in the Annals of Neurology, and have further work in progress to create induced pluripotent stem cell lines from the cells of patients with other genetic forms of epilepsy. The work is funded by the National Institutes of Health, the American Epilepsy Society, the Epilepsy Foundation and U-M.

The new findings differs from what other scientists have seen in mice -- demonstrating the importance of studying cells made from human epilepsy patients. Because the cells came from patients, they contained the hallmark seen in most patients with Dravet syndrome: a new mutation in SCN1A, the gene that encodes the crucial sodium channel protein called Nav1.1. That mutation reduces the number of channels to half the normal number in patients' brains.

"With this technique, we can study cells that closely resemble the patient's own brain cells, without doing a brain biopsy," says senior author and team leader Jack M. Parent, M.D., professor of neurology at U-M and a researcher at the VA Ann Arbor Healthcare System. "It appears that the cells are overcompensating for the loss of channels due to the mutation. These patient-specific induced neurons hold great promise for modeling seizure disorders, and potentially screening medications."

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Epilepsy in a Dish

California Stem Cell Submits FDA Phase III Clinical Trial Protocol for the Treatment of Metastatic Melanoma

IRVINE, Calif.--(BUSINESS WIRE)--

California Stem Cell, Inc. (CSC) announced today the submission of a Phase III protocol to the U.S. Food and Drug Administration (FDA) for a randomized, double-blind study in metastatic melanoma. The multicenter trial will examine the effectiveness of CSCs patient specific cancer immunotherapy in patients with recurrent stage III or stage IV metastatic melanoma.

The study, led by Chief Medical Officer Robert Dillman, M.D., will explore the efficacy of subcutaneous injections of autologous dendritic cells that have been loaded with irradiated autologous tumor cells suspended in GM-CSF (DC-TC). The protocol calls for approximately 250 adult patients to be randomized in a 2:1 ratio to receive either the DC-TC or a control treatment. Doses will be administered weekly for three consecutive weeks, then monthly for the following five months. The primary and sole efficacy endpoint will be overall survival.

CSCs submission of the Phase III protocol comes on the heels of two earlier Phase II clinical studies in which the combined median 5-year survival in patients with stage IV melanoma was a remarkably high 51%.

This therapy has been proven to be safe and appears to prolong survival in late stage melanoma. We are very excited to be advancing this program into Phase III trials, so that we can further evaluate what we believe to be a very promising option for cancer patients, said Robert Dillman, M.D., Chief Medical Officer at CSC.

About California Stem Cell

California Stem Cell Inc. (CSC) is an Irvine, CA-based company focused on the development of stem cell-based therapies for metastatic cancers, spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS, or Lou Gehrigs Disease).

CSC has proprietary methods to generate human stem cell lines, expand them to clinically and commercially useful numbers, and differentiate them at extremely high purity using fully-defined, proprietary media and GMP processes.

Follow us on Twitter: http://twitter.com/castemcell

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California Stem Cell Submits FDA Phase III Clinical Trial Protocol for the Treatment of Metastatic Melanoma

Stem Cell Clinical Trials Expert to Lead Cardiology Division at Cedars-Sinai Heart Institute

Newswise LOS ANGELES (July 24, 2013) Timothy D. Henry, MD, an expert known for his innovative work in developing stem cell treatments for advanced heart disease patients, has joined the Cedars-Sinai Heart Institute as director of Cardiology.

Henry comes to Cedars-Sinai from Minneapolis, where he was director of research for the Minneapolis Heart Institute Foundation and professor of medicine at the University of Minnesota School of Medicine.

As Henry begins his tenure, Prediman K. Shah, MD, the renowned cardiologist who has led the Division of Cardiology for nearly 20 years, is stepping down to focus on patient care and on basic and clinical research, including his quest to develop a vaccine and mutant-gene-based therapies that would thwart heart attacks by preventing and possibly reversing arterial cholesterol buildup. Shah, the Shapell and Webb Family Chair in Clinical Cardiology, director of Oppenheimer Atherosclerosis Research Center and director of the Atherosclerosis Prevention and Treatment Center, also will continue treating patients and teaching medical residents and cardiology trainees.

Dr. Shah has been a wonderful colleague and a highly respected national leader, accelerating the pre-eminence of our Cardiology Division, and Dr. Henry will be starting his new position from a robust platform built over many decades of outstanding service, said Shlomo Melmed, MD, senior vice president of Academic Affairs, dean of the Cedars-Sinai medical faculty and the Helene A. and Philip E. Hixon Chair in Investigative Medicine. We are delighted that Dr. Shah will be remaining on as a senior cardiologist in our Heart Institute.

This is a milestone moment, said Eduardo Marbn, MD, PhD, director of the Cedars-Sinai Heart Institute. At the same time we celebrate Dr. Shahs achievements and his valuable contributions to the Heart Institute and to overall heart health, we are welcoming Dr. Henry and beginning a new era during which we foresee the next wave of leading-edge stem cell clinical trials for heart disease patients.

For several years, Henry has been collaborating with Marbn on stem cell clinical trials. The first such collaboration follows upon work at Cedars-Sinai in which heart attack patients received infusions of cardiac stem cells. This study, published in February 2012 in The Lancet, showed that patients who underwent the stem cell procedure experienced a significant reduction in the size of the scar left behind by a heart attack. Patients also experienced a sizable increase in healthy heart muscle following the experimental stem cell treatments.

The new study is being directed by Henry and by Raj Makkar, MD, director of Interventional Cardiology and associate director of the Cedars-Sinai Heart Institute. The national trial called ALLSTAR uses heart cells from unrelated donors in an effort to reverse lasting tissue damage after a heart attack.

My goal in joining the Cedars-Sinai Heart Institute is to help make stem cells a regular treatment option for heart disease, Henry said. Right now, many patients with advanced heart disease have limited treatment options. Stem cells are the next frontier.

A native of Mohall, N.D., Henry earned his bachelors degree at the University of North Dakota and his medical degree at the University of California, San Francisco. Following an internship, residency and cardiology research fellowship at University of Colorado Health Sciences Center in Denver, Henry moved to the University of Minnesota and the Minneapolis Heart Institute, where he has focused on interventional cardiology, treatment of patients with acute heart attacks and novel therapy for patients with advanced heart disease who are not candidates for bypass surgery or stenting.

He has authored hundreds of studies published in peer-reviewed medical journals. He currently serves on the Advisory Committee for the AHA Mission: Lifeline Program, the AHA Acute Cardiac Care Committee of the Council on Clinical Cardiology and the Board of the Society of Chest Pain Centers. He has served as national principal investigator of multiple large, multicenter trials in acute coronary syndromes, myocardial infarction and angiogenesis including several ongoing cardiovascular stem cell trials, including RENEW, ALLSTAR and ATHENA. He is also principal investigator for one of seven NIH Clinical Cardiovascular Stem Cell Centers. As a visiting professor, Henry has lectured throughout the world on systems of care for acute myocardial infarction, refractory angina and stem cell therapy for cardiovascular disease. The process to grow cardiac-derived stem cells was developed by Dr. Marbn when he was on the faculty of Johns Hopkins University. Johns Hopkins has filed for a patent on that intellectual property and has licensed it to Capricor, a company in which Cedars-Sinai and Marbn have a financial interest. Capricor is providing funds for the ALLSTAR clinical study at Cedars-Sinai.

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Stem Cell Clinical Trials Expert to Lead Cardiology Division at Cedars-Sinai Heart Institute

Stem cell transplant gives hope to leukemia patient

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On Thursday night, the 39-year-old woman from Newfoundland will undergo a stem cell transplant from an anonymous donor in Halifax. Julia Wong/Global News

HALIFAX Janice Davidson is getting a new lease on life.

On Thursday night, the 39-year-old leukemia patient will undergo a stem cell transplant from an anonymous donor in Halifax.

You cant believe there is someone out there in the world who you dont know who is willing to give you a gift that is like no other really, the mother of three said.

Davidson was diagnosed with leukemia five years ago. Since then, she has tried several different treatment options and relapsed twice. A stem cell transplant is now her best option to beat the disease.

Its not going to be an easy path but we know were on the right path, she said. We know what we need to do and we are very hopeful for the future.

The latest figures from One Match, the national program for blood stem cell donors and recipients, show that there are about 750 Canadians waiting to receive a stem cell transplant. Eight of those are in Nova Scotia.

Factors such as genetics, ethnic background and treatment affect wait time in receiving a transplant so Davidson is extremely thankful that she found a match. It is especially fortunate because her three siblings were not matches and there are only 317,810 donors registered in Canada. Of those, 10,337 live in Nova Scotia.

Husband Ian says he cannot express enough how thankful he is to the donor, adding that he or she is now like another member of their family.

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Stem cell transplant gives hope to leukemia patient

New stem cell gene therapy gives hope to prevent inherited neurological disease

Public release date: 24-Jul-2013 [ | E-mail | Share ]

Contact: Alison Barbuti alison.barbuti@manchester.ac.uk 44-016-127-58383 University of Manchester

Scientists from The University of Manchester have used stem cell gene therapy to treat a fatal genetic brain disease in mice for the first time.

The method was used to treat Sanfilippo a fatal inherited condition which causes progressive dementia in children but could also benefit several neurological, genetic diseases.

Researchers behind the study, published in the journal Molecular Therapy this month, are now hoping to bring a treatment to trial in patients within two years.

Sanfilippo, a currently untreatable mucopolysaccharide (MPS) disease, affects one in 89,000 children in the United Kingdom, with sufferers usually dying in their mid-twenties. It is caused by the lack of SGSH enzyme in the body which helps to breakdown and recycle long chain sugars, such as heparan sulphate (HS). Children with the condition build up and store excess HS throughout their body from birth which affects their brain and results in progressive dementia and hyperactivity, followed by losing the ability to walk and swallow.

Dr Brian Bigger, from the University of Manchester's Institute of Human Development who led the research, said bone marrow transplants had been used to correct similar HS storage diseases, such as Hurler syndrome, by transplanting normal cells with the missing enzyme but the technique did not work with Sanfilippo disease. This is because monocytes, a type of white blood cell, from the bone marrow, did not produce enough enzyme to correct the levels in the brain.

Dr Bigger said: "To increase SGSH enzyme from bone marrow transplants, and to target it to the cells that traffic into the brain, we have developed a stem cell gene therapy which overproduces the SGSH enzyme specifically in bone marrow white blood cells.

"We have shown that mice treated by this method produce five times the normal SGSH enzyme levels in the bone marrow and 11 per cent of normal levels in the brain.

"The enzyme is taken up by affected brain cells and is enough to correct brain HS storage and neuro inflammation to near normal levels and completely corrects the hyperactive behaviour in mice with Sanfilippo.

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New stem cell gene therapy gives hope to prevent inherited neurological disease

Stem cell retina therapy treatment might provide miraculous 'cure' for blindness

The three blind mice might get the chance to see because of groundbreaking stem cell research being done in Britain.

Scientists in the UK took stem cells from mice embryos, put them in a petri dish and coaxed them into becoming photoreceptors, the cells in the retina that catch light.

After collecting 200,000 of the stem cells turned photoreceptors the scientists then injected the cells into the eyes of blind mice, and some of the cells integrated into the host retina and restored sight.

The rodents were then run through a maze and examined by optometry to confirm that they did indeed respond to light.

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The groundbreaking work was published by Britain's Medical Research Council and in the science journal "Nature Biotechnology."

While the work is still years away from helping humans, it is an extremely promising start to curing blindness caused by photoreceptor loss like retinitis pigmentosa and age-related macular degeneration.

The biggest breakthrough that the team in England was able to achieve was turning the finicky stem cells into stable photoreceptors, instead of deadly cancer cells.

Additionally, researchers in the past could turn stem cells into rod cells which are a component of the complex network of nerves in the retina but now they are able to create photoreceptors that comprise all the different nerves needed for sight.

"Over recent years, scientists have become pretty good at working with stem cells and coaxing them to develop into different types of adult cells and tissues," said lead researcher Robin Ali at the University College London Institute of Ophthalmology and Moorfields Eye Hospital.

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Stem cell retina therapy treatment might provide miraculous 'cure' for blindness