UC San Diego Health System announces human testing of stem cell therapies

SAN DIEGO (CNS) - UC San Diego Health System announced Monday that human testing of injected neural stem cell therapies are underway at its Sanford Stem Cell Clinical Center.

Researchers are conducting three different trials -- one on a 26-year-old woman paralyzed after a traffic crash, and others on diabetes and leukemia patients.

"What we are seeing after years of work is the rubber hitting the road," said Lawrence Goldstein, director of the UC San Diego Stem Cell program and Sanford Stem Cell Clinical Center.

"These are three very ambitious and innovative trials," he said. "Each followed a different development path -- each addresses a very different disease or condition. It speaks to the maturation of stem cell science that we've gotten to the point of testing these very real medical applications in people."

The first tests are being made with low doses in order to ensure the safety of the patients, Goldstein said.

Working with Maryland-based Neuralstem Inc., neural stem cells were injected into the site of the paralyzed woman's spinal cord injury on Sept. 30, and she is recovering at home without complications or adverse effects, said Dr. Joseph Ciacci, a neurosurgeon at UC San Diego Health System. Her name was not released.

The researchers hope that the transplanted cells will develop into neurons that bridge the gap created by the injury, replace severed or lost nerve connections and restore at least some motor and sensory function. According to UCSD, testing in laboratory rats with spinal cord injuries were promising.

A two-year trial on about 40 Type 1 diabetes patients will involve implanting cells under the skin that were derived from embryonic stem cells, with the hope they will safely mature into pancreatic beta and other cells able to produce a continuous supply of needed insulin and other substances, according to the researchers. The first procedure is expected to take place sometime this month, according to UCSD.

Type 1 diabetes, which usually onsets during childhood and has no cure, causes the pancreas to produce little or no insulin. Patients have to inject insulin daily and rigorously manage their diet and lifestyle.

The third trial will involve a potential drug to fight chronic lymphocytic leukemia, the most common form of blood cancer in adults. Patients in the test will receive the drug via an intravenous infusion every 14 days at the UCSD Moores Cancer Center.

See the original post:
UC San Diego Health System announces human testing of stem cell therapies

UCSD Stem Cell program begins human testing

SAN DIEGO (CNS) - UC San Diego Health System announced Monday that human testing of injected neural stem cell therapies are underway at its Sanford Stem Cell Clinical Center.

Researchers are conducting three different trials -- one on a 26-year-old woman paralyzed after a traffic crash, and others on diabetes and leukemia patients.

"What we are seeing after years of work is the rubber hitting the road," said Lawrence Goldstein, director of the UC San Diego Stem Cell program and Sanford Stem Cell Clinical Center.

"These are three very ambitious and innovative trials," he said. "Each followed a different development path -- each addresses a very different disease or condition. It speaks to the maturation of stem cell science that we've gotten to the point of testing these very real medical applications in people."

The first tests are being made with low doses in order to ensure the safety of the patients, Goldstein said.

Working with Maryland-based Neuralstem Inc., neural stem cells were injected into the site of the paralyzed woman's spinal cord injury on Sept. 30, and she is recovering at home without complications or adverse effects, said Dr. Joseph Ciacci, a neurosurgeon at UC San Diego Health System. Her name was not released.

The researchers hope that the transplanted cells will develop into neurons that bridge the gap created by the injury, replace severed or lost nerve connections and restore at least some motor and sensory function. According to UCSD, testing in laboratory rats with spinal cord injuries were promising.

A two-year trial on about 40 Type 1 diabetes patients will involve implanting cells under the skin that were derived from embryonic stem cells, with the hope they will safely mature into pancreatic beta and other cells able to produce a continuous supply of needed insulin and other substances, according to the researchers. The first procedure is expected to take place sometime this month, according to UCSD.

Type 1 diabetes, which usually onsets during childhood and has no cure, causes the pancreas to produce little or no insulin. Patients have to inject insulin daily and rigorously manage their diet and lifestyle.

The third trial will involve a potential drug to fight chronic lymphocytic leukemia, the most common form of blood cancer in adults. Patients in the test will receive the drug via an intravenous infusion every 14 days at the UCSD Moores Cancer Center.

Here is the original post:
UCSD Stem Cell program begins human testing

Scientists Grow, Implant Human Intestinal Tissue in Mice

SUNDAY, Oct. 19, 2014 (HealthDay News) -- New stem cell-based research could improve understanding of intestinal diseases and eventually lead to new treatments, a new study suggests.

Scientists used stem cells to grow "organoids" of functioning human intestinal tissue in a lab dish. They then transplanted the organoids into mice, creating a new model for studying intestinal disorders, according to the researchers.

"This provides a new way to study the many diseases and conditions that can cause intestinal failure, from genetic disorders appearing at birth to conditions that strike later in life, such as cancer and Crohn's disease," lead investigator Dr. Michael Helmrath, surgical director of the Intestinal Rehabilitation Program at the Cincinnati Children's Hospital Medical Center, said in a center news release.

"These studies also advance the longer-term goal of growing tissues that can replace damaged human intestine," he added.

Further research could eventually lead to the ability to create personalized human intestinal tissue to treat gastrointestinal diseases, according to the researchers.

"These studies support the concept that patient-specific cells can be used to grow intestine," Helmrath explained.

The research was published online Oct. 19 in the journal Nature Medicine.

The intestinal organoids were created using so-called pluripotent stem cells, which can become any type of tissue in the body. The scientists created these "blank" stem cells by reprogramming adult cells taken from skin and blood samples.

The stem cells were placed in lab dishes with a specific molecular mixture that prompted the cells to grow into intestinal organoids, which developed into fully mature, functioning human intestinal tissue after being transplanted into mice.

The mice were genetically engineered so that their immune systems would not reject the human tissue, the study authors noted.

Read more here:
Scientists Grow, Implant Human Intestinal Tissue in Mice

UCSD Announces Human Testing Of Stem Cell Treatment Is Underway

UC San Diego Health System announced Monday that human testing of injected neural stem cell therapies are underway at its Sanford Stem Cell Clinical Center.

Researchers are conducting three different trials one on a 26-year-old woman paralyzed after a traffic crash, and others on diabetes and leukemia patients.

"What we are seeing after years of work is the rubber hitting the road," said Lawrence Goldstein, director of the UC San Diego Stem Cell program and Sanford Stem Cell Clinical Center.

"These are three very ambitious and innovative trials," he said. "Each followed a different development path each addresses a very different disease or condition. It speaks to the maturation of stem cell science that we've gotten to the point of testing these very real medical applications in people."

The first tests are being made with low doses in order to ensure the safety of the patients, Goldstein said.

Working with Maryland-based Neuralstem Inc., neural stem cells were injected into the site of the paralyzed woman's spinal cord injury on Sept. 30, and she is recovering at home without complications or adverse effects, said Dr. Joseph Ciacci, a neurosurgeon at UC San Diego Health System. Her name was not released.

The researchers hope that the transplanted cells will develop into neurons that bridge the gap created by the injury, replace severed or lost nerve connections and restore at least some motor and sensory function. According to UCSD, testing in laboratory rats with spinal cord injuries were promising.

A two-year trial on about 40 Type 1 diabetes patients will involve implanting cells under the skin that were derived from embryonic stem cells, with the hope they will safely mature into pancreatic beta and other cells able to produce a continuous supply of needed insulin and other substances, according to the researchers. The first procedure is expected to take place sometime this month, according to UCSD.

Type 1 diabetes, which usually onsets during childhood and has no cure, causes the pancreas to produce little or no insulin. Patients have to inject insulin daily and rigorously manage their diet and lifestyle.

The third trial will involve a potential drug to fight chronic lymphocytic leukemia, the most common form of blood cancer in adults. Patients in the test will receive the drug via an intravenous infusion every 14 days at the UCSD Moores Cancer Center.

View original post here:
UCSD Announces Human Testing Of Stem Cell Treatment Is Underway

Promise Put to the Test

Contact Information

Available for logged-in reporters only

Newswise A 26-year-old woman paralyzed after a motor vehicle accident a year ago has successfully undergone a first-in-human experimental procedure to test whether neural stem cells injected at the site of a spinal cord injury is safe and could be an effective treatment.

The procedure, conducted on Sept. 30 under the auspices of the Sanford Stem Cell Clinical Center at UC San Diego Health System and in collaboration with Neuralstem, Inc., a Maryland-based biotechnology firm, is the first of four in the Phase I clinical trial. Post safety testing, its hoped that the transplanted neural stem cells will develop into new neurons that bridge the gap created by the injury, replace severed or lost nerve connections and restore at least some motor and sensory function.

The patient, whose identity remains confidential for privacy reasons, has been discharged and is recovering without complication or adverse effects at home, said Joseph Ciacci, MD, principal investigator and neurosurgeon at UC San Diego Health System.

The spinal cord injury trial is one of three recent ground-breaking stem cell efforts at UC San Diego, supported by the Sanford Stem Cell Clinical Center, to make the significant leap from laboratory to first-in-human clinical trials.

Last month, researchers at UC San Diego Moores Cancer Center and the Sanford Stem Cell Clinical Center launched a novel Phase I trial to assess the safety of a monoclonal antibody treatment that targets cancer stem cells in patients with chronic lymphocytic leukemia, the most common form of blood cancer.

And later this month, the first patient is scheduled to receive an unprecedented stem cell-based therapy designed to treat type 1diabetes in another Phase I clinical trial at UC San Diego.

What we are seeing after years of work is the rubber hitting the road, said Lawrence Goldstein, PhD, director of the UC San Diego Stem Cell program and Sanford Stem Cell Clinical Center at UC San Diego Health System. These are three very ambitious and innovative trials. Each followed a different development path; each addresses a very different disease or condition. It speaks to the maturation of stem cell science that weve gotten to the point of testing these very real medical applications in people.

To be sure, Goldstein said, the number of patients involved in these first trials is small. The initial focus is upon treatment with low doses to assess safety, but also with hope of patient benefit. As these trials progress and additional trials are launched Goldstein predicts greater numbers of patients will be enrolled at UC San Diego and the Sanford Stem Cell Clinical Center and elsewhere.

Continue reading here:
Promise Put to the Test

Margot Martini's family launch awareness day

The family of little Margot Martini has announced the launch of a day to promote the need for potential stem cell donors to join UK and worldwide registries.

A year ago today the brave youngster was diagnosed with both acute lymphoblastic leukaemia (ALL) and acute myeloid leukaemia (AML). She was just 14 months old at the time.

A worldwide hunt was launched to find a perfect stem cell match, leading to more than 50,000 people coming forward to be tested under the Swab4Margot campaign.

Finally in February a suitable donor was found and she underwent a bone marrow transplant. The following month doctors declared she was 99 per cent certain to be disease free, but tests in June revealed a small amount of ALL had returned.

Margot's parents Yaser and Vicki made the difficult decision in July to stop her treatment after medical experts told them her chances of being able to live a normal life after further treatment were slim.

Now two-year-old, Margot is living out the rest of her days at home with her loving family, but throughout her brave fight Team Margot - the group formed to support her battle - has remained committed to promoting awareness around the need for more potential stem cell donors to come forward. As a result of the group's efforts in the UK, more than 500 people will now receive potentially life saving bone marrow transplants.

Booked to take place on October 7, 2015, the inaugural Team Margot Stem Cell and Bone Marrow Awareness Day will give particular encouragement to those of mixed race to register, as this is an under-represented stem bank.

There are 37,000 people worldwide who are desperately seeking a stem cell match and only 60 per cent of transplant recipients currently find a perfect donor.

Of those from a black, Asian or ethnic minority background, a little more than 20 per cent of transplant recipients ever find their perfect match.

Yaser Martini said: "Team Margot is committed to raising global awareness around the need for more stem cell donor recruits, hence the launch of this annual international awareness day to help promote greater understanding and to encourage more people to register as potential stem cell /bone marrow donors.

Read more here:
Margot Martini's family launch awareness day

My stem cells could help save the life of man Ive never met

A HEALTHCARE worker at Royal Bournemouth Hospital has donated stem cells in a bid to save the life of an unknown man.

Claire Waugh, pictured, who has always been a regular blood donor, decided to join the Anthony Nolan stem cell register after her father was diagnosed with prostate cancer three years ago.

The healthcare assistant co-ordinator was later identified as a possible match for a man needing life-saving treatment.

Following rigorous testing Claire was visited by nurses from the blood cancer charity, who gave her three injections every day for three days to stimulate her bone marrow to produce stem cells.

On the fourth day she travelled to Kings College Hospital in London to receive a final set of injections and undergo a stem cell collection in a simple five-hour outpatient procedure, which is similar to giving blood.

Claire said: I couldnt move or bend my arm due to the fairly heavy duty needle, but I was looked after really well so in the end the time went very quickly.

After donating, Claires stem cells were rushed to the recipient within the required 72 hours. A volunteer from Anthony Nolan told me that if he doesnt survive, there is nothing else on this earth that would have cured him, so this was this persons last chance, added Claire.

When my dad was poorly it made me think that if he needed this kind of help, I would be praying every night that someone would help him.

By doing this, it meant that I could give that chance to someone else and their family.

Royal Bournemouth Hospital granted special leave to Claire for the donation with the charity covering all of her and her husbands travel expenses.

Originally posted here:
My stem cells could help save the life of man Ive never met

Retinal stem cell study shows promise for therapy

SAN DIEGO (KUSI) - Macular degeneration is the leading cause of vision loss for people over the age of 50. Scientists have discovered a new therapy that may actually restore sight in those affected.

Scientists are excited not only because it worked, and helped some people see clearly again, but also because this study puts a focus on an new kind of stem cell therapy, using skin cells.

Macular degeneration causes a blurry or black area in the middle of your field of vision that grows over time, causing more sight loss.

There is no cure, but a new study published this week in the journal The Lancet, is giving patients hope.

Embryonic stem cells were turned into retinal cells and implanted into the eyes of 18 patients.

Vision improved for about half of them.

Dr. Andreas said, "This study was primarily to see if these cells would be safe, and the bonus was that some people started to see better."

Dr. Andreas Bratyy-Layal and Dr. Suzanne Peterson are stem cell scientists with the Scripps Research Institute.

They see this as a major breakthrough.

Although this sight study did do that, Dr. Peterson says labs around the country, including here in San Diego, are moving away from the practice.

Follow this link:
Retinal stem cell study shows promise for therapy

Researchers in Berlin and Bath Identify Nave-Like Human Stem Cells

16.10.2014 - (idw) Max-Delbrck-Centrum fr Molekulare Medizin (MDC) Berlin-Buch

In their search for the earliest possible stage of development of human embryonic stem cells (hESCs) that still have the potential to develop into any types of body cells and tissue, researchers from the Max Delbrck Center for Molecular Medicine (MDC) Berlin-Buch, Germany, and the University of Bath, United Kingdom, have apparently been successful. Jichang Wang, Gangcai Xie, and Dr. Zsuzsanna Izsvk (MDC), together with Professor Laurence D. Hurst (University of Bath), report the discovery of a subtype of cells in culture dishes with hESCs and human induced pluripotent stem cells (hiPSCs) that resemble this very early, pluripotent or nave state (Nature, doi:10.1038/nature13804)*. They also discovered the mechanism that turns human ES cells into nave-like human stem cells. While this has potential implications for medicine and for understanding early human development, an evolutionary enigma still remains unsolved.

Human embryonic stem cells (hESCs) differ considerably from those of mice. Mouse nave cultures resemble the inner cell mass which gives rise to the embryo, while none of the cultured hESC lines do. Nave ESCs of mice are easy to maintain, but not human ESCs isolated from pre-implantation embryos. The hESC lines, researchers work with in their laboratories are considered to be less nave, and have limited differentiation potential. Researchers hypothesize that they have partially lost their pluripotency. Why this is so remains unclear.

What properties characterize human nave stem cells? Can they be identified and proliferated in the laboratory and retained in culture? Researchers in Europe, Asia and the USA are trying to find the answers to these questions in order to be able to use these cells for therapy in the future.

Evolution pointed the way It was evolution that showed the researchers in Bath and Berlin the way to the successful approach. They pinpointed one particular class of ancient viruses called HERVH (human endogenous retrovirus H). HERVH integrated into our DNA millions of years ago, and although it does not function as a virus any longer, it is not silent.

HERVH-derived sequences appear at a very early stage in human embryos, that is, HERVH is highly expressed at just the right time and place in human embryos where one would expect to see nave stem cells. This was also observed by Professor Kazutoshi Takahashi (Kyoto University, Kyoto, Japan), almost at the same time when Dr. Izsvk and Professor Hurst made their discovery.**

Dr. Izsvk and Professor Hurst succeeded in going one step further. They were able to identify the switch that regulates HERVH. In hESC cultures they identified a transcription factor called LBP9 as being central to the activity of HERVH in early embryos. Using a reporter system that made cells expressing HERVH via LBP9 glow green, the Berlin and Bath team found that they had purified human ESCs that showed all the hallmarks of nave mouse stem cells.

This transcription factor was not previously known to be important to human stem cells. However, unknown to them at the time, the same transcription factor was shown by Austin Smiths group (University of Cambridge, UK) to have a role in mouse nave cells***.

Our human nave-like cells look remarkably like the mouse ones, and are close to human inner cell mass (ICM), said Jichang Wang (PhD student, MDC), first author of the Nature publication. With our HERVH-based reporter system we can easily isolate nave-like human ESCs from any human ESC culture. These cells grow like the mouse nave stem cells and express many of the same genes such as NANOG, KLF4 and OCT4 that are associated with murine navet. When we knockdown LBP9 or HERVH, these cells no longer resemble nave-like human stem cells, he added.

To explore a potential role in stem cell-based therapeutics, the next task will be to keep these isolated human nave-like stem cells in culture and proliferate them. HERVH would also be particularly useful in identifying optimal conditions for long-term culturing. As HERVH inhibits differentiation, its expression should be transient, otherwise it might be detrimental to normal embryo development. What factors keep this delicate process in balance is yet to be determined.

Continued here:
Researchers in Berlin and Bath Identify Nave-Like Human Stem Cells