Clinical Trials: Advanced Cell Technology - Stem Cell Therapy
Last month (October 2014) in The Lancet, Advanced Cell Technology (ACT) published their preliminary phase 1 clinical data for their Stem Cell therapy trials for Stargardt #39;s Macular Dystrophy...
By: Essceejulies
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Clinical Trials: Advanced Cell Technology - Stem Cell Therapy - Video
Dopamine-making neurons derived from human embryonic stem cells.
A rat model of Parkinson's disease has been successfully treated with neurons derived from human embryonic stem cells, according to a study led by Swedish scientists. Its a promising sign for scientists at The Scripps Research Institute and Scripps Health who hope to perform similar therapy on Parkinsons patients, using artificial embryonic stem cells.
In rats and people, neurons that make the neurotransmitter dopamine are essential for normal movement. The cells are destroyed in Parkinson's, leading to the difficulty in movement that characterizes the disease.
Researchers transplanted dopamine-producing cells grown from human embryonic stem cells into the brains of rats whose own dopamine-making neurons had been destroyed. The rats were immune-suppressed so they would not reject the cells. Within five months, the transplanted cells boosted dopamine production to normal levels, restoring normal movement in the rats.
The study was published Thursday in the journal Cell Stem Cell. The senior author was Malin Parmar of Lund University in Lund, Sweden.
The results support the Scripps approach of using the artificial embryonic stem cells, called induced pluripotent stem cells, said Jeanne Loring, who heads the Center for Regenerative Medicine at The Scripps Research Institute in La Jolla. Loring is part of a group called Summit 4 Stem Cell that's raising funds to treat eight Parkinson's patients with their own IPS cells.
Particularly significant is the study's comparison of the effects of dopamine-making neurons derived from fetal cells to that of embryonic stem cells, Loring said by email.
"In the 1980s and 1990s, there were several clinical trials that showed that grafts of fetal brain containing the precursors of dopamine neurons could reverse the effects of Parkinson's disease in some patients," Loring said. "We, and the others developing stem cell therapies, based our plans on the results of those studies, but no one had ever directly compared fetal tissue and human pluripotent stem cell-derived dopamine neurons in an animal model of PD."
Induced pluripotent stem cells appear to have much the same capacity as human embryonic stem cells to generate different tissues and organs.
There has been uncertainty about how similar they are to each other, specifically whether the IPS process produces mutations. But recent studies have found the cell types are extremely similar, including a study also published in Cell Stem Cell on Thursday. That study compared IPS cells with embryonic stem cells produced by SCNT, or somatic cell nuclear transfer, the same process used to create Dolly the sheep.
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Parkinson's stem cell therapy works in rats
Stem Cells Help Victim of Spinal Cord Injury to Walk
A young man that was paralyzed after a gunshot wound to the spine, and after 4 weeks of stem cell treatment he regained use of his legs. We look at video of his recovery and speak with his...
By: TheLipTV
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Stem Cells Help Victim of Spinal Cord Injury to Walk - Video
(Ivanhoe Newswire) CLEVELAND, Ohio -- In 1990 the Human Genome Project started. It was a massive scientific undertaking that aimed to identify and map out the body's complete set of DNA. This research has paved the way for new genetic discoveries; one of those has allowed scientists to study how to fix bad chromosomes.
Our bodies contain 23 pairs of them, 46 total. But if chromosomes are damaged, they can cause birth defects, disabilities, growth problems, even death.
Case Western scientist Anthony Wynshaw-Boris is studying how to repair damaged chromosomes with the help of a recent discovery. He's taking skin cells and reprogramming them to work like embryonic stem cells, which can grow into different cell types.
You're taking adult or a child's skin cells. You're not causing any loss of an embryo, and you're taking those skin cells to make a stem cell. Anthony Wynshaw-Boris, M.D., PhD, of Case Western Reserve University, School of Medicine told Ivanhoe.
Scientists studied patients with a specific defective chromosome that was shaped like a ring. They took the patients' skin cells and reprogrammed them into embryonic-like cells in the lab. They found this process caused the damaged ring chromosomes to be replaced by normal chromosomes.
It at least raises the possibility that ring chromosomes will be lost in stem cells, said Dr. Wynshaw-Boris.
While this research was only conducted in lab cultures on the rare ring-shaped chromosomes, scientists hope it will work in patients with common abnormalities like Down syndrome.
What we're hoping happens is we might be able to use, modify, what we did, to rescue cell lines from any patient that has any severe chromosome defect, Dr. Wynshaw-Boris explained.
It's research that could one day repair faulty chromosomes and stop genetic diseases in their tracks.
The reprogramming technique that transforms skin cells to stem cells was so ground-breaking that a Japanese physician won the Nobel Prize in medicine in 2012 for developing it.
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Stem cells to repair broken chromosomes
Stem Cell Therapy for Multiple Sclerosis: Ron McGill
Ron McGill suffers from relapsing-remitting multiple sclerosis. He was started experiencing symptoms in 2009 but was not diagnosed with MS until January of 2013. He received several infusion...
By: http://www.cellmedicine.com
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Stem Cell Therapy for Multiple Sclerosis: Ron McGill - Video
For more than 30 years, stem cells have been the great hope of medical science. Given their remarkable ability to turn into any type of cell in the body, researchers have theorized that they could be used to treat and perhaps even cure all sorts of diseases and conditions from spinal cord injury to baldness.
Progress has been painfully slow for most areas of research but this week researchers in Sweden are reporting a major advancein a possible stem cell treatment for Parkinson's. While the treatment has only been tried in rats, the scientists -- led byMalin Parmar, an associate professor of regenerative neurobiology at the Lund University -- said they believe the results are promising enoughto move to clinical trials in humans within a few years.
A degenerative condition of the central nervous system, Parkinson's affects an estimated 7 to 10 million people worldwide. Actor Michael J. Fox has Parkinson's and Google co-founder Sergey Brin has a gene that makes him susceptible to the disease. Both have not only raised awareness of the disease through their celebrity but have contributed millions of dollars to advance research.
Parkinson's is caused by the loss of dopamine-producing cells in the brain that help regulate things like movement and emotions. The scientistsat the Lund University found that when they turned human embryonic stem cells into neurons that produce dopamine and injected them into the brains of rats, something remarkable happened. The damage from the disease seemed to reverse.
The scientists wrote that while they believe their research was "rigorous," they pointed out that "a number of crucial issues" still need to be addressed before the treatment can be tested in humans. For instance, they need to make sure the cells continue to work the way they are supposed to over longer time periods.
Read more:
Parkinsons disease and depression often go hand in hand
Stem cell study shows promising results for severe stroke patients
Growing stem cells using cloning techniques reopens ethics debate
Dance for Parkinson's: movement as medicine
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New hope for Parkinsons patients in stem cell treatment
By Cyndi Root
International Stem Cell Corporation is now approved to manufacture human parthenogenetic stem cells. The Food and Drug Administration (FDA) cleared the cells for investigational clinical use. The company announced the approval in a press release, stating that it improves its chance for approval of its Parkinsons disease treatment and provides an avenue for using the cells in other indications such as stroke or traumatic brain injury.
Dr. Ruslan Semechkin, ISCO's Chief Scientific Officer, said, "Many stem cell lines can never be used to develop commercial therapeutic products because they don't meet the FDA's ethical and quality standards. With this clearance from the FDA, the Company has removed any uncertainty in the potential clinical use of human parthenogenetic stem cells."
FDA Action
Like all manufacturing to FDA standards, stem cells must be produced in good manufacturing practice (GMP) conditions. The cells must be grown under repeatable conditions and be identical, so that patients receive standardized stem cell therapy. In addition, the federal agency seeks to reduce the risk of an infectious disease. ISCO provided the FDA assurances relating to the original egg donor's risk of infectious diseases, the testing of the master cell bank, and the genetic stability of the stem cell line. ISCO intends to produce the stem cells at its facility in Oceanside, CA and will provide an update on the first batch later.
Parthenogenetic Stem Cells
ISCO states that its parthenogenetic stem cells (hpSCs) are a new class of stem cells with the best characteristics of other stem cells. The company creates the cells by stimulating the donors oocytes (eggs), which are not fertilized and are not viable embryos. Stimulating the oocytes begins the process of cell division. This method creates cells that are histocompatiblethey do not depend on the target patient. Immunomatching and using unfertilized oocytes provides an ethical advantage and a reliable source for cell-based therapy.
Parkinson's Disease Submission
Dr. Semechkin stated the FDA manufacturing approval provides a boost to its Parkinson's disease submission, which the company intends to submit by the end of 2014. ISCO provided an update on the program in October 2014, stating that none of the preclinical pharmacology and toxicology studies have shown adverse events or pathological reactions. ISCO intends to present the results of those studies at the Society for Neuroscience annual meeting.
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FDA Clears ISCO's Parthenogenetic Stem Cells For Investigational Clinical Use
McLean Hospital and Harvard Stem Cell Institute scientists have new evidence that stem cell transplantation could be a worthwhile strategy to help epileptics who do not respond to anti-seizure drugs.
As reported in Cell Stem Cell, the laboratory of McLean Associate Neurobiologist Sangmi Chung, PhD, transplanted seizure-inhibiting, human embryonic stem cell-derived neurons into the brains of mice with a common form of epilepsy. Half of the mice who received the transplanted neurons no longer had seizures, while the other half experienced a significant drop in seizure frequency.
"After the transplantation we observed that the human neurons integrate into the epileptic brain," said Chung, who is also a Harvard Stem Cell Institute affiliated faculty member and an assistant professor at Harvard Medical School. "The transplanted neurons begin to receive excitatory input from host neurons and in turn generate inhibitory responses that reverse the electrical hyperactivity that cause seizures."
The recovery seen after the human stem cell-derived neuron transplants, which were done while the cells were still maturing into their full-grown form, is similar to that published in a 2013 study by University of California, San Francisco, scientists who transplanted fetal mouse brain cells into epileptic mice.
While encouraging, Chung noted that further primate studies and a process to purify the neurons, so only those known to inhibit seizures are transplanted (called interneurons), would need to be completed before a treatment in humans could be considered.
"Because embryonic stem cells can differentiate into many different cell types, even when we drive them into neurons, there are always other cell types," she said. "For clinical purposes, we need to make sure the cells are safe, without any contaminant. Currently we are working on a different method to specifically isolate interneurons."
Over 65 million people worldwide are affected by epileptic seizures, which can cause convulsions, loss of consciousness and other neurological symptoms. The exact cause of the condition is unknown, but it is hypothesized that diminished populations of interneurons is a contributor.
Most epileptic patients can be treated with anti-seizure drugs, which contain molecules that can inhibit electrical symptoms, similar to the normal function of interneurons. But about one-third do not benefit from existing medication. Patients may opt to have a portion of their brain cut out to control symptoms.
"This seems to be an area that needs a novel therapy," Chung said. "Before starting this project, I was a stem cell biologist mostly interested in the development of neural stem cells, but as I've come to know about epilepsy, I've become motivated to continue this research."
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Human stem cell-derived neuron transplants reduce seizures in mice
After four surgeries, nine kinds of chemotherapy, stem cell transplants, 14 radiation treatments and antibody treatments, Katie Hawley beat cancer. Twice.
Katie, then 9, learned she had cancer in 2009, when doctors found an egg-sized, stage 3 tumor in her stomach, the cause of her pain and nausea. The rare tumor strikes fewer than 5 of every 1 million children each year, according to the National Institute of Health.
In my heart, I hoped and believed she would beat this and live to 100, said Mary Kay Hawley, Katies mom. Yet, that quiet voice would whisper the nightmarish words, She may not make it to her 10th birthday. With that, I became her court jester I wanted to give her the world.
After she endured a yearlong battle with ganglio neuroblastoma, doctors found no evidence of cancer cells left in her body. For more than two years, the Ladera Ranch girl lived like a normal kid again aside from scans every three months.
Until the day before Valentines Day last year, when one of her routine CT scans showed the cancer had returned with a vengeance, spreading to her skull, hips and legs.
I was terrified that I was going to lose her, Hawley said. She fell to her knees and begged God to spare her daughter. I prayed until I had peace.
Katie, now a freshman at San Juan Hills High, was sent to undergo an experimental, strong chemotherapy treatment in San Francisco. She had a 33 percent chance of improvement, a 33 percent chance the disease would stay the same and 33 percent chance it would get worse. There was a 1 percent chance the treatment would kill the cancer cells, Hawley said.
Katie turned out to be the 1 percent and had a scan clear of cancer in June last year.
KEEPING THE CANCER AT BAY
To stop the cancer from returning, Katie was put on an aggressive, 10-month, in-home chemotherapy with Accutane treatments, making her extremely sick, tired and depressed, Hawley said. She took 14 pills a day, went to the hospital twice a week for blood tests and checkups and continued with CT scans every three months to check that the cancer hadnt returned.
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Experimental treatments planned in teen's miraculous cancer fight
8 hours ago In this multiple confocal analysis of transverse sections from transgenic zebrafish embryos, vasculature is labeled by red fluorescence, NF-kB protein complex that regulates inflammation by green fluorescence and nuclei by blue fluorescence. The arrowhead indicates a potential hematopoietic stem cell emerging in the dorsal aorta with high expression of NF-kB. The image at bottom right combines all channels. Credit: UC San Diego School of Medicine
Hematopoietic stem cells (HSCs) give rise to all blood and immune cells throughout the life of vertebrate organisms, from zebrafish to humans. But details of their genesis remain elusive, hindering efforts to develop induced pluripotent stem cell (iPSC) replacements that might address a host of blood disorders.
In a paper published Nov. 20 in the journal Cell, researchers at the University of California, San Diego School of Medicine describe the surprising and crucial involvement of a pro-inflammatory signaling protein in the creation of HSCs during embryonic development, a finding that could help scientists to finally reproduce HSCs for therapeutic use.
"The recent breakthrough of induced pluripotency has made the concept of patient-specific regenerative medicine a reality," said principal investigator David Traver, PhD, professor in the Department of Cellular and Molecular Medicine. "The development of some mature cell lineages from iPSCs, such as cardiac and neural, has been reasonably straightforward, but not with HSCs. This is likely due, at least in part, to not fully understanding all of the factors used by the embryo to generate HSCs. We believe the discovery that pro-inflammatory cues are important in vivo will help us recapitulate instruction of HSC fate in vitro from iPSCs."
Traver and colleagues specifically looked at the role of a cytokine (a type of cell signaling protein) called tumor necrosis factor alpha or TNF, which plays a pivotal role in regulating systemic inflammation and immunity. The work extended previous research by Spanish biologist Victoriano Mulero, who had reported that TNF was important in the function of the embryonic vascular system and that in animal models where TNF function was absent, blood defects resulted.
The Cell paper's first author Raquel Espin-Palazon, a postdoctoral researcher in Traver's lab and a former colleague of Mulero's, determined that TNF was required for the emergence of hematopoietic stem cells during embryogenesis in zebrafish a common animal model.
Traver said the finding was completely unexpected because HSCs emerge relatively early in embryonic formation when the developing organism is considered to be largely sterile and devoid of infection.
"Thus, there was no expectation that pro-inflammatory signaling would be active at this time or in the blood-forming regions," Traver said. "Equally surprising, we found that a population of embryonic myeloid cells, which are transient cells produced before HSCs arise, are the producers of the TNF needed to establish HSC fate. So it turns out that a small subset of myeloid cells that persist for only a few days in development are necessary to help generate the lineal precursors of the entire adult blood-forming system."
The newly discovered role of TNF in HSC development mirrors a parallel discovery regarding interferon gamma (INFg), another cytokine and major mediator of pro-inflammatory signaling, highlighting multiple inputs for inflammatory signaling in HSC emergence. Traver said the crucial roles of TNF and INFg in HSC emergence are likely similar in humans because of the highly conserved nature of HSC development across vertebrate evolution.
Explore further: New blood: Tracing the beginnings of hematopoietic stem cells
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Before there will be blood: Pro-inflammatory signaling plays surprising role in creation of hematopoietic stem cells