Stem cell therapy for sidelined star Smoko

Magnifisio dashed home strongly over 1400m to win Saturdays Lee-Steere Stakes at Ascot. Picture: Westernracepix

Sprinter Smoko will have stem cell therapy at Murdoch Veterinary Hospital to a strained suspensory ligament in his off-foreleg.

Vets found Smoko had strained the ligament when he pulled up sore following his shock sixth as a $2 favourite to Shining Knight in last Tuesday's Colonel Reeves Stakes (1100m) at Ascot.

Co-trainer Ross Price said Smoko would be sidelined for months.

"He will go to Murdoch where they will look at him and see about stem cell therapy," he said.

"In about 10 days we will take him up there and see what they can do. It is then going to be five months off and hoping."

Smoko was a $6.50 chance in Saturday week's Winterbottom Stakes (1200m) before he was scratched. WA's hopes of winning back the Group 1 weight-for-age hinge on Magnifisio, Shining Knight and Testamezzo, with Barakey in doubt after struggling to recover from a virus.

"He is still feeling flat and I will have to wait and see if he improves over the next few days," trainer Jim Taylor said.

Magnifisio firmed from $12 into $8 on the TAB yesterday following her strong win at her debut over 1400m in Saturday's Group 2 Lee-Steere Stakes at Ascot.

Melbourne sprinters Angelic Light, Moment Of Change and reigning champion Buffering dominate betting at $4.30, $6.50 and $7.50.

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Stem cell therapy for sidelined star Smoko

Production of human motor neurons from stem cells is gaining speed

PUBLIC RELEASE DATE:

10-Nov-2014

Contact: Ccile Martinat CMARTINAT@istem.fr 33-603-855-477 INSERM (Institut national de la sant et de la recherche mdicale) @inserm

This news release is available in French.

The motor neurons that innervate muscle fibres are essential for motor activity. Their degeneration in many diseases causes paralysis and often death among patients. Researchers at the Institute for Stem Cell Therapy and Exploration of Monogenic Diseases (I-Stem - Inserm/AFM/UEVE), in collaboration with CNRS and Paris Descartes University, have recently developed a new approach to better control the differentiation of human pluripotent stem cells, and thus produce different populations of motor neurons from these cells in only 14 days. This discovery, published in Nature Biotechnology, will make it possible to expand the production process for these neurons, leading to more rapid progress in understanding diseases of the motor system, such as infantile spinal amyotrophy or amyotrophic lateral sclerosis (ALS).

Human pluripotent stem cells have the ability to give rise to every cell in the body. To understand and control their potential for differentiation in vitro is to offer unprecedented opportunities for regenerative medicine and for advancing the study of physiopathological mechanisms and the quest for therapeutic strategies. However, the development and realisation of these clinical applications is often limited by the inability to obtain specialised cells such as motor neurons from human pluripotent stem cells in an efficient and targeted manner. This inefficiency is partly due to a poor understanding of the molecular mechanisms controlling the differentiation of these cells.

Inserm researchers at the Institute for Stem Cell Therapy and Exploration of Monogenic Diseases (I-Stem - Inserm/French Muscular Dystrophy Association [AFM]/University of vry Val d'Essonne [UEVE]), in collaboration with CNRS and Paris-Descartes University, have developed an innovative approach to study the differentiation of human stem cells and thus produce many types of cells in an optimal manner.

"The targeted differentiation of human pluripotent stem cells is often a long and rather inefficient process. This is the case when obtaining motor neurons, although these are affected in many diseases. Today, we obtain these neurons with our approach in only 14 days, nearly twice as fast as before, and with a homogeneity rarely achieved," explains Ccile Martinat, an Inserm Research Fellow at I-Stem.

To achieve this result, the researchers studied the interactions between some molecules that control embryonic development. These studies have made it possible to both better understand the mechanisms governing the generation of these neurons during development, and develop an optimal "recipe" for producing them efficiently and rapidly.

"We are now able to produce and hence study different populations of neurons affected to various degrees in diseases that cause the degeneration of motor neurons. We plan to study why some neurons are affected and why others are preserved," adds Stphane Nedelec, an Inserm researcher in Ccile Martinat's team.

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Production of human motor neurons from stem cells is gaining speed

Family's desperate bet on a diabetes cure

The day Olivia Cox was diagnosed with Type 1 diabetes at age 16, her mother vowed to find a cure.

"I said to her, "there's someone walking this Earth who has been cured of diabetes, and I'm going to find him," Ruth Cox said.

Cox's search started with a call to Harvard University and ended with a family trip to Lima, Peru. It was at a clinic there that now 18-year-old Olivia and her father, Jeff, 54, who also has diabetes, received an infusion of stem cells designed to wipe out diabetes in their bodies or, at the very least, lessen its impact. The treatment illegal in the United States cost $70,000 for both father and daughter. Two months later, the Niskayuna family is waiting for a transformation and wondering if, in their desperation for a cure, they were snookered by false promises.

Because stem cells can be programmed to become anything from heart muscle to toenails, stem cell therapy can hypothetically be used to treat anything, from baldness to Lou Gehrig's Disease. But the study of regenerative medicine is still nascent in the United States, where it is restricted to procedures that use the patient's own cells, and it has been primarily used in treating cancer a procedure that saved Ruth Cox 13 years ago, when she had breast cancer.

Stem cell treatment using donor cells is more common elsewhere in the world, but with varying results and none that could be described as a cure. An executive order from President Barack Obama opened up funding for stem cell research and there are now more than 4,000 clinical trials under way, some on animals and some recruiting people with various ailments.

The American Diabetes Association strongly supports stem cell research, according to a statement posted on its website, which reads in part:

"Scientists from across the United States and throughout the world, including those involved with the American Diabetes Association believe that stem cell research, especially embryonic stem cell research, holds great promise in the search for a cure and better treatments for diabetes."

Jeff Cox, diagnosed with Type 1 diabetes when he was 11, has suffered none of the complications that often come with the disease neuropathy, loss of vision and heart disease. But Cox said living with diabetes is hell. He pricks his finger at least a dozen times a day to check his blood sugar level, because it is a more precise reading than the glucose monitor he wears. He also wears a pump that he programs to inject him with insulin automatically based on his diet and exercise each day. All the therapies used to treat diabetes are designed to intervene where the pancreas has gone awry.

In Type 1 diabetes, the pancreas doesn't produce insulin due to an autoimmune attack against the beta cell that produces insulin the hormone that converts glucose into energy our bodies need to survive. The Coxes didn't want their daughter to face a lifetime of managing her diabetes. They wanted a cure, and they were willing to take a risk to find it.

In order to treat diabetes with stem cell therapy, pancreatic stem cells isolated from umbilical cord blood that are programmed to produce insulin, plus autologous mesenchymal stem cells from the patient's bone marrow, are injected. Once in the pancreas, the cells are supposed to replicate themselves, gradually replacing the non-insulin producing cells in the host's pancreas. The treatment is conducted in Peru, China, Russia and India and elsewhere, but Zubin Master, a bioethicist at Albany Medical College, said the risks of traveling abroad for stem cell therapy range from paying for an expensive treatment that doesn't work, to cancer and death.

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Family's desperate bet on a diabetes cure

Production of human motor neurons from stem cells gaining speed

4 hours ago Neurons (green) are detected by TuJI whereas motoneurons are revealed in red by the visicular transporter of acetylcholine. Credit: Inserm/Martinat, Ccile

The motor neurons that innervate muscle fibres are essential for motor activity. Their degeneration in many diseases causes paralysis and often death among patients. Researchers at the Institute for Stem Cell Therapy and Exploration of Monogenic Diseases (I-Stem - Inserm/AFM/UEVE), in collaboration with CNRS and Paris Descartes University, have recently developed a new approach to better control the differentiation of human pluripotent stem cells, and thus produce different populations of motor neurons from these cells in only 14 days. This discovery, published in Nature Biotechnology, will make it possible to expand the production process for these neurons, leading to more rapid progress in understanding diseases of the motor system, such as infantile spinal amyotrophy or amyotrophic lateral sclerosis (ALS).

Human pluripotent stem cells have the ability to give rise to every cell in the body. To understand and control their potential for differentiation in vitro is to offer unprecedented opportunities for regenerative medicine and for advancing the study of physiopathological mechanisms and the quest for therapeutic strategies. However, the development and realisation of these clinical applications is often limited by the inability to obtain specialised cells such as motor neurons from human pluripotent stem cells in an efficient and targeted manner. This inefficiency is partly due to a poor understanding of the molecular mechanisms controlling the differentiation of these cells.

Inserm researchers at the Institute for Stem Cell Therapy and Exploration of Monogenic Diseases (I-Stem - Inserm/French Muscular Dystrophy Association [AFM]/University of vry Val d'Essonne [UEVE]), in collaboration with CNRS and Paris-Descartes University, have developed an innovative approach to study the differentiation of human stem cells and thus produce many types of cells in an optimal manner.

"The targeted differentiation of human pluripotent stem cells is often a long and rather inefficient process. This is the case when obtaining motor neurons, although these are affected in many diseases. Today, we obtain these neurons with our approach in only 14 days, nearly twice as fast as before, and with a homogeneity rarely achieved," explains Ccile Martinat, an Inserm Research Fellow at I-Stem.

To achieve this result, the researchers studied the interactions between some molecules that control embryonic development. These studies have made it possible to both better understand the mechanisms governing the generation of these neurons during development, and develop an optimal "recipe" for producing them efficiently and rapidly.

"We are now able to produce and hence study different populations of neurons affected to various degrees in diseases that cause the degeneration of motor neurons. We plan to study why some neurons are affected and why others are preserved," adds Stphane Nedelec, an Inserm researcher in Ccile Martinat's team.

In the medium term, the approach should contribute to the development of treatments for paralytic diseases such as infantile spinal muscular amyotrophy or amyotrophic lateral sclerosis. "Rapid access to large quantities of neurons will be useful for testing a significant number of pharmacological drugs in order to identify those capable of preventing the death of motor neurons," concludes Ccile Martinat.

Explore further: Team finds a better way to grow motor neurons from stem cells

More information: Combinatorial analysis of developmental cues efficiently converts human pluripotent stem cells into multiple neuronal subtypes, Nature Biotechnology, 17 Nov 2014. DOI: 10.1038/nbt.3049

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Production of human motor neurons from stem cells gaining speed

Clinical Trials: Advanced Cell Technology – Stem Cell Therapy – Video


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...

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Clinical Trials: Advanced Cell Technology - Stem Cell Therapy - Video

Parkinson's stem cell therapy works in rats

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

BioEden the specialist tooth stem cell bank announce record sales up 45%

(PRWEB UK) 9 November 2014

Every day stem cell treatments are in the news, often bringing the last hope for many patients with serious conditions that have defied traditional medicine.

BioEden's Group CEO Tony Veverka says, 'the increase in popularity in banking a persons own cells is as a result of two main things; 1. The likelihood of needing stem cell therapy is high, and 2. You need a stem cell match - your own cells are the perfect match'.

BioEden's tooth stem cell banking service is the only way you can access your own cells without the need for medical intervention. More and more parents are choosing to bank their children's stem cells as the baby tooth is shed naturally. However, stem cells can be banked from a healthy adult molar, that perhaps is being removed for orthodontic reasons.

Obtaining stem cells from other sources such as bone marrow is not only invasive but costly. Taking the decision to bank ones own cells is a sensible consideration, and costs from just 295.

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BioEden the specialist tooth stem cell bank announce record sales up 45%

Researchers create stem cell model of Parkinsons disease in a dish

Published November 07, 2014

A team of stem cell scientists has identified the biological mechanisms of Parkinsons disease and recreated a model of the disease in a dish.

Researchers at The New York Stem Cell Foundation (NYSCF) Research Institute studied a pair of identical twins one with Parkinsons and one without as well as another unrelated Parkinsons patient and four healthy control subjects to observe key characteristics of the disease. After comparing the individuals biological factors, they noticed differences in the patients neurons ability to produce dopamine. Dopamine production is deficient in Parkinsons disease.

"The unique scenario of identical twins, one with this disease and one without, allowed our scientists an unprecedented look into the mechanisms of Parkinson's disease," Susan L. Solomon, NYSCF chief executive officer, said in a news release. "Advanced stem cell research techniques allow us to push the boundaries of science and see what actually goes wrong at the cellular level, step by step during the disease process."

Parkinsons disease affects an estimated 500,000 people in the United States, according to the National Institutes of Health (NIH). The average age of onset is 60, and the risk of developing it increases with age. Symptoms of Parkinsons include tremor, shaking in the hands, arms, legs, jaw or head; impaired balance or postural instability; slowness of movement; and stiffness of the limbs and trunk.

There is currently no cure for Parkinsons.

While the disease is moderately hereditary, scientists have yet to fully understand the mechanisms of inheritance. The researchers note the DNA mutations that produce the enzyme glucocerebrosidase (GBA) have been linked to a five-fold increased risk of developing Parkinsons, but only 30 percent of people with this mutation have been shown to get the disease by age 80. This suggests that genetic and non-genetic factors cause Parkinsons. In studying the identical twins, scientists were able to analyze these mechanisms.

The scientists made induced pluripotent stem (iPS) cells from skin samples from both twins to generate a cellular model of Parkinsons in a dish, recreating the outstanding features of the disease specifically the dopamine and a-synuclein deficiency.

Scientists saw that the neurons from the twin affected by Parkinsons produced less dopamine and had higher levels of an enzyme called monomine oxidase B (MAO-B), as well as a poorer ability to connect with each other, compared to the twin that did not have the disease.

The findings suggest a possible therapy for Parkinsons: treating neurons with molecules that reduce the activity of MAO-B and GBA, while normalizing -synuclein and dopamine levels.

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Researchers create stem cell model of Parkinsons disease in a dish

Scientists create Parkinson's disease in a dish

PUBLIC RELEASE DATE:

6-Nov-2014

Contact: David McKeon dmckeon@nyscf.org 212-365-7440 New York Stem Cell Foundation @nyscf

New York, NY (November 6, 2014) - A team of scientists led by The New York Stem Cell Foundation (NYSCF) Research Institute successfully created a human stem cell disease model of Parkinson's disease in a dish. Studying a pair of identical (monozygotic) twins, one affected and one unaffected with Parkinson's disease, another unrelated Parkinson's patient, and four healthy control subjects, the scientists were able to observe key features of the disease in the laboratory, specifically differences in the patients' neurons' ability to produce dopamine, the molecule that is deficient in Parkinson's disease. In addition, the scientists also identified a potential strategy for developing novel therapies for Parkinson's disease.

Attributed to a combination of genetic and nongenetic factors, Parkinson's disease has no completely effective therapy or cure. Parkinson's disease is moderately heritable, but the mechanisms of this inheritance are not well understood. While genetic forms of the disease exist, sporadic forms are far more common.

"The unique scenario of identical twins, one with this disease and one without, allowed our scientists an unprecedented look into the mechanisms of Parkinson's disease," said Susan L. Solomon, NYSCF Chief Executive Officer. "Advanced stem cell research techniques allow us to push the boundaries of science and see what actually goes wrong at the cellular level, step by step during the disease process."

DNA mutations resulting in the production of a specific enzyme called glucocerebrosidase (GBA) have been linked to a five-fold greater risk of developing Parkinson's disease; however, only 30% of individuals with this mutation have been shown to develop Parkinson's disease by the age of 80. This discordance suggests that multiple factors contribute to the development of Parkinson's disease, including both genetic and non-genetic factors. To date, there has been no appropriate model to identify and test multiple triggers leading to the onset of the disease.

In this study, published today in Cell Reports, a set of identical twins, both with a GBA mutation, provided a unique opportunity to evaluate and dissect the genetic and non-genetic contributions to the development of Parkinson's disease in one twin, and the lack of disease in the other. The scientists made induced pluripotent stem (iPS) cells from skin samples from both twins to generate a cellular model of Parkinson's in a dish, recapitulating key features of the disease, specifically the accumulation of -synuclein and dopamine deficiency.

Upon analyzing the cell models, the scientists found that the dopamine-producing neurons from both twins had reduced GBA enzymatic activity, elevated -synuclein protein levels, and a reduced capacity to synthesize and release dopamine. In comparison to his unaffected brother, the neurons generated from the affected twin produced less dopamine, had higher levels of an enzyme called monoamine oxidase B (MAO-B), and poor ability to connect with each other. Treating the neurons with molecules that lowered the activity of MAO-B together with overexpressed GBA normalized -synuclein and dopamine levels in the cell models. This suggests that a combination therapy for the affected twin may be possible by simultaneously targeting these two enzymes.

"The subject of Parkinson's disease discordant twins gave us an incredible opportunity to utilize stem cell models of disease in a dish to unlock some of the biological mechanisms of disease," said Dr. Scott Noggle, NYSCF Vice President, Stem Cell Research and The NYSCF - Charles Evans Senior Research Fellow for Alzheimer's Disease. "Working with these various different groups and scientists added to the depth and value of the research and we hope our findings will be applicable to other Parkinson's disease patients and other neurodegenerative disorders."

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Scientists create Parkinson's disease in a dish

Stem cell transplants may help reduce seizures, study says

New research from McLean Hospital and the Harvard Stem Cell Institute has shown that stem cell therapy reduces seizures in mice.

Researchers used an animal model to transplant seizure-inhibiting, human embryonic stem cell-derived neurons into the brains of mice that had a common form of epilepsy. Half of the mice that received the transplanted neurons no longer had seizures, while the other half experienced a significant drop in seizure frequency.

The transplanted neurons integrated into the mouse brains and began to receive neuronal activity. The neurons then released GABA, an inhibitory response that reversed the electrical hyperactivity that causes seizure.

Previous studies showed increasing inhibition in the epileptic brain can help control the seizure and also a lot of anti-epilepsy drugs are mimicking this GABA, so many of them worked by binding to the GABA receptors, researcher Sangmi Chung, assistant professor of psychiatry at Harvard, told FoxNews.com.

Researchers initially set out to test the functionality of human neurons, but later decided to test their effect on epilepsy because it is such a devastating disease. About 30 percent of people do not respond to seizure drugs and one out of 26 people will be affected by seizures in their lifetime, Chung said.

Over 65 million people worldwide are affected by epileptic seizures, which can cause convulsions, loss of consciousness and other neurological symptoms. Patients are treated with anti-seizure drugs, and may choose to have a portion of their brain removed.

Because mouse cells mature more quickly than human cells within weeks instead of years it was unclear how long a stem cell transplant in a human would take before becoming effective, Chung noted.

If we compare it with the mouse [model], we believe it will be years, not weeks, she said.

However, the study found that, even without full maturation, the cells integrated into the epileptic mouse brains, receive signals and release GABA, therefore preventing seizures.

I think its really good news in terms of transplantation even maturing, not fully mature [cells] still work, Chung said.

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Stem cell transplants may help reduce seizures, study says

Direct Generation of Neural Stem Cells Could Enable Transplantation Therapy

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Newswise CAMBRIDGE, Mass. (November 6, 2014) Induced neural stem cells (iNSCs) created from adult cells hold promise for therapeutic transplantation, but their potential in this capacity has been limited by failed efforts to maintain such cells in the desirable multi-potent NSC state without continuous expression of the transcription factors used initially to reprogram them.

Now, Whitehead Institute scientists have created iNSCs that remain in the multi-potent state without ongoing expression of reprogramming factors. This allows the iNSCs to divide repeatedly to generate cells in quantities sufficient for therapy.

Therapeutically, its important to make neural stem cells because they can self-renew and make lots of cells, says Whitehead Institute Founding Member Rudolf Jaenisch, who is also a professor of biology at MIT. If you just make mature neurons, which has been done by others, you never get enough cells.

To make iNSCs via direct lineage conversion researchers use viruses to insert a cocktail of transcription factors into the genome of mouse adult skin cells. A drug triggers these transcription factors to turn on genes active in neural stem cells. This direct conversion, known as transdifferentiation, bypasses the step of pushing the cells first through an embryonic stem-cell-like state.

In previous research, iNSCs remained addicted to the drug and reprogramming transcription factors; if either the drug or the factors was removed, the cells reverted to skin cells or spontaneously differentiated.

If the reprogramming factors are still active, its horrible for the cells, says John Cassady, a scientist in Jaenischs lab. The cells would be unable to differentiate and the resulting cells would not be therapeutically useful.

In a paper published online this week in the current issue of the journal Stem Cell Reports, Cassady and other Whitehead scientists describe how they preserve the cells properties without keeping the reprogramming factors active. First, the cells were grown in a special medium that selects for neural stem cells. Then, the drug is removed. Instead of spontaneously differentiating, the iNSCs remain in a multi-potent state that can differentiate into neurons and glia cells on cue. Cassady also refined the reprogramming cocktail to contain eight transcription factors, which produces iNSCs that are transcriptionally and epigenetically similar to mouse neural stem cells.

Cassady notes that a random sample of skin cells can contain neural crest cells, which may more easily make the transition to iNSCs. To eliminate the possibility that his method might actually rely on cells having this sort of head start, Cassady converted fully mature immune system cells called B-lymphocytes, which have a very specific genetic marker, to iNSCs. The resulting cells had the profile of their new identity as iNSCs, yet retained their B-lymphocyte genetic marker, showing that Cassadys method could indeed convert non-neural cells to iNSCs.

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Direct Generation of Neural Stem Cells Could Enable Transplantation Therapy

Transplant of stem-cell-derived dopamine neurons shows promise for Parkinson's disease

PUBLIC RELEASE DATE:

6-Nov-2014

Contact: Mary Beth O'Leary moleary@cell.com 617-397-2802 Cell Press @CellPressNews

Parkinson's disease is an incurable movement disorder that affects millions of people around the world, but current treatment options can cause severe side effects and lose effectiveness over time. In a study published by Cell Press November 6th in Cell Stem Cell, researchers showed that transplantation of neurons derived from human embryonic stem cells (hESCs) can restore motor function in a rat model of Parkinson's disease, paving the way for the use of cell replacement therapy in human clinical trials.

"Our study represents an important milestone in the preclinical assessment of hESC-derived dopamine neurons and provides essential support for their usefulness in treating Parkinson's disease," says senior study author Malin Parmar of Lund University.

Parkinson's disease is caused, in part, by the death of neurons that release a brain chemical called dopamine, leading to the progressive loss of control over dexterity and the speed of movement. Currently available drug and surgical treatment options can lose effectiveness over time and cause serious side effects such as involuntary movements and psychiatric problems. Meanwhile, another approach involving the transplantation of human fetal cells has produced long-lasting clinical benefits; however, the positive effects were only seen in some individuals and can also cause involuntary movements driven by the graft itself. Moreover, the use of tissue from aborted human fetuses presents logistical issues such as the limited availability of cells, hampering the effective translation of fetal tissue transplantation as a realistic therapeutic option.

To rigorously assess an alternative hESC-based treatment approach, Parmar and lead study author Shane Grealish of Lund University transplanted hESC-derived dopamine neurons into brain regions that control movement in a rat model of Parkinson's disease. The transplanted cells survived the procedure, restored dopamine levels back to normal within five months, and established the correct pattern of long-distance connections in the brain. As a result, this therapy restored normal motor function in the animals. Importantly, the hESC-derived neurons show efficacy and potency similar to fetal neurons when transplanted in the rat model of Parkinson's disease, suggesting that the hESC-based approach may be a viable alternative to the approaches that have already been established with fetal cells in Parkinson's patients.

In a related Forum article published in the same issue, Roger Barker of Addenbrooke's Hospital and the University of Cambridge laid out the roadmap for taking stem-cell-derived dopamine neurons to the clinic for treating Parkinson's disease. "This involves understanding the history of the whole field of cell-based therapies for Parkinson's disease and some of the mistakes that have happened," he says. "It also requires a knowledge of what the final product should look like and the need to get there in a collaborative way without being tempted to take shortcuts, because a premature clinical trial could impact negatively on the whole field of regenerative medicine."

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Cell Stem Cell, Grealish et al.: "Human ESC-derived dopamine neurons show preclinical efficacy and potency similar to fetal neurons when grafted in a rat model of Parkinson's disease."

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Transplant of stem-cell-derived dopamine neurons shows promise for Parkinson's disease

FDA Clears ISCO's Parthenogenetic Stem Cells For Investigational Clinical Use

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