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One MS patient's 'starting line' for stem cell therapy

Posted: October 14, 2014 at 9:40 pm

By Richard M. Cohen

image courtesy Richard Cohen

I am one of twenty struggling every day with multiple sclerosis to be included in an innovative, phase one stem cell clinical trial at the Tisch MS Research Center of New York. Now theres a mouthful. Please let me explain. Many of us read tidbits about cell therapy and think it simply is space-age medicine that will be launched in the future.

In fact, we are at the starting line now, and the race has begun. A phase one trial tests safety. The group is small, and all are treated with the real thing. No placebos, sugar pills. The trial tests autologous cells, which mean our own. That eliminates rejection and alters risk. No new medical procedure comes risk-free, but the dangers are minimal. The stem cells are pulled from bone marrow harvested from our breast bones. Sounds hideous. It is not.

In this trial, the stem cells are infused directly into the spinal column. Nope. Not painful at all. Then we watch and wait. Results, if there are to be any, can take many months to show themselves. This particular procedure has never been used before. I was the first in the group to be treated, making me the first in the world to have this done. For more than forty years, I have lived with an illness that left no room for hope. Suddenly, that has changed, though change does not necessarily come easily.

The expectation game is dangerous. No one really knows what to expect from this experiment. My doctor makes that point over and over. Yet it is hard to control the fantasies that inevitably pop into my head. The possibility of restoring at least some vision when I have been legally blind for years is enticing, to say the least. I used to run and race or simply hike up country hills. Now I hobble on a cane. I am lucky if I can stay on my feet walking two city blocks. The possibility of restored mobility takes my breath away.

I know better than to go too far down these roads in my mind, but that visual journey is unavoidable. Maybe that is okay. Hope is a funny thing. We need something to hope for. Any doctor will tell you attitude is an important factor in fighting a disease. I have learned the power of remaining positive. We need fuel to keep the engine running. Those flights of fancy, imagining we can be better than we are, to some extent can become self-fulfilling prophecies.

This is an exciting period in the history of medicine. That probably has been said throughout the ages. Science does not stand still. No one can see around the bend. That may be what makes hope possible, the idea that there is something just out of sight that is revolutionary and good, just waiting for us to get there.

Richard M. Cohen writes Journey Man, an independent blog, also carried by The Huffington Post. Cohen is the author of Blindsided, published in 2004, which chronicled his battles with multiple sclerosis and cancer, and Strong at the Broken Places in 2008, both New York Times Best Sellers. Cohens latest book, I Want to Kill the Dog, was published in 2012. Cohen is married to journalist, Meredith Vieira, with whom he has three grown children.

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One MS patient's 'starting line' for stem cell therapy

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Stem Cell Therapy For ALS Gets FDA's Fast Track Designation

Posted: October 14, 2014 at 9:40 pm

By C. Rajan, contributing writer

The U.S. FDA has just granted BrainStorm Cell Therapeutics novel stem cell therapy, NurOwn, Fast Track status for the treatment of amyotrophic lateral sclerosis (ALS), the company announced via press release.

"We are pleased that the FDA has granted Fast Track status for NurOwn as this will allow us greater and more frequent dialogue with the Agency as we continue the development of this ground-breaking cell therapy for the treatment of ALS," said Tony Fiorino, MD, PhD, CEO of BrainStorm. "We expect Fast Track designation, which recognizes the potential of NurOwn as to address an unmet medical need in ALS, to help speed and improve our development program."

Israeli biotech company BrainStorm is developing novel adult stem cell technologies for neurodegenerative diseases, such as ALS. The company licensed the exclusive rights to the NurOwn technology from Ramot, the technology transfer company of Tel Aviv University.

NurOwn is a personalized stem cell product made from autologous mesenchymal stem cells. These adult stem cells are obtained from the patients bone marrow and are induced to secrete neurotrophic factors, which are growth factors that can stimulate the survival and maintenance of neurons that degenerate in neurologic disorders.

NurOwn is currently being studied in randomized, double-blind, placebo-controlled phase 2 clinical trials in ALS patients in both Israel and the U.S. Reuters reports that the last patient visit has been completed in the phase 2a clinical trial in Jerusalem. The company expects to release final results of the study by the end of this year. The U.S. arm of the Phase 2 study is being conducted at three sites in the U.S., and is expected to be wrapped up in early 2015.

The FDA's Fast Track program aims to speed up the development of new drugs and biologics in order to get them to patients suffering from serious, unmet medical needs. The Fast Track designation will allow BrainStorm Cell to submit an NDA on a rolling basis and will grant the company more communication and support from FDA during the development process.

ALS, also known as Lou Gehrig's disease, is a rapidly progressive neurological disease that results in death within 2 to 5 years of diagnosis in most cases, and less than 20 percent of patients live more than 5 years after onset of symptoms. The relatively rare condition affects about 2 persons in every 100,000, with approximately 5,600 new cases diagnosed every year in the U.S, according to the ALS Association.

There is no cure for the disease to date, although the only approved ALS drug, Riluzole, has demonstrated its ability to extend survival by at least a few months.

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Stem cells improve vision enough for horse riding

Posted: October 14, 2014 at 9:40 pm

Seeing is definitely believing when it comes to stem cell therapy. A blind man has recovered enough sight to ride his horse. A woman who could see no letters at all on a standard eye test chart can now read the letters on the top four lines. Others have recovered the ability to see colour. All have had injections of specialised retinal cells in their eyes to replace ones lost through age or disease.

A trial in 18 people with degenerative eye conditions is being hailed as the most promising yet for a treatment based on human embryonic stem cells.

"We've been hearing about their potential for more than a decade, but the results have always been in mice and rats, and no one has shown they're safe or effective in humans long term," says Robert Lanza of Advanced Cell Technology in Marlborough, Massachusetts, the company that carried out the stem cell intervention. "Now, we've shown both that they're safe and that there's a real chance these cells can help people."

Ten years ago, the team at Advanced Cell Technology announced that it had successfully converted human embryonic stem cells into retinal pigment epithelial cells. These cells help keep the eyes' light-detecting rods and cones healthy. But when retinal pigment epithelial cells deteriorate, blindness can occur. This happens in age-related macular degeneration and Stargardt's macular dystrophy.

In a bid to reverse this, Lanza's team injected retinal cells into one of each of the 18participants' eyes, half of whom had age-related macular degeneration and half had Stargardt's. A year later, 10people's eyes had improved, and the eyes of the others had stabilised. Untreated eyes had continued to deteriorate.

"On average, we're seeing three lines [on an eye test chart] of visual improvement in our patients," says Lanza.

There were no serious side effects and no sign of tumours, which can be a potential risk in stem cell therapies.

Lanza says the aim of the study was to halt further deterioration, so the improvements in sight were an unexpected bonus. He speculates that the improvements might be the result of rods and cones that had become dormant when the native retinal pigment epithelial cells died, resuming their function when the fresh cells were added.

"The results are highly encouraging," says Pete Coffey of University College London, who heads a project to treat people with age-related macular degeneration using tiny patches of retinal pigment epithelial cells made from human embryonic stem cells.

Advanced Cell Technology is now planning a larger trial, first in 100 people with Stargardt's, then in people with macular degeneration.

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Stem cells improve vision enough for horse riding

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Stem Cells Could Produce Insulin – Video

Posted: October 14, 2014 at 8:40 pm


Stem Cells Could Produce Insulin
A team of scientists at Harvard seems to have taken a crucial step toward eventually curing diabetes by creating insulin-producing beta cells. Follow Jasmine Bailey: http://www.twitter.com/jasmine...

By: Newsy Science

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Stem Cells Could Produce Insulin - Video

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Therapeutic Potential of Adult Stem Cells in the Congenital Muscular Dystrophies – Video

Posted: October 14, 2014 at 8:40 pm


Therapeutic Potential of Adult Stem Cells in the Congenital Muscular Dystrophies
Dr. Olga Igoucheva from Thomas Jefferson University shares an update on her research with adult stem cells in Collagen VI and LAMA2 Muscular Dystrophies.

By: CureCMD

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Therapeutic Potential of Adult Stem Cells in the Congenital Muscular Dystrophies - Video

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Healthy recipes with plant-based foods are weapons against cancer

Posted: October 14, 2014 at 11:52 am

Pink is the official color of Breast Cancer Awareness Month, but the foods recommended for breast cancer prevention are green, white, red, yellow and brown.

Theyre all - no surprise - the foods of the earth: fruits, vegetables, herbs, spices, nuts, seeds. October is, conveniently, also Vegetarian Awareness Month. This is not a matter of overbooking; health and plant-based food go together.

Behold, your cancer-fighting all-stars:

Broccoli: A 2011 University of Michigan study found the plant sterols in broccoli reduce breast cancer stem cells. Broccolis just coming into season here, so buy local and eat up.

Coffee: Java junkies and cold-brew fiends, this ones for you. A 2011 Breast Cancer Research report shows the antioxidants in 2 cups a day protects cells from cancer growth.

Parsley: This ubiquitous garnish is high in vitamin C and apigenin, another phytonutrient we never knew about before. Apigenin may be cancers WMD, according to findings by the University of Missouri. Celebrate with some parsley-rich tabbouli.

Pomegranate: In season now, these sweet-tart beauties can arrest cancer cell growth and destroy existing cancer cells, according to a University of California study. The fine print: You need to consume a lot of pomegranate, about 3 cups a day, for the goodness to kick in.

Soy: Organic whole soy, the way they eat it in Asia - tempeh, tofu, edamame and miso - has received the blessing from the American Institute for Cancer Research. Choose that rather than the GMO soy isolates present in much American processed food.

Turmeric: Gold dust. Antiseptic, anti-inflammatory and antioxidant, this wonderful warming spice seems to inhibit or erase cancer cell growth, according to a 2011 Cancer Prevention Research study.

Walnuts: High in omega-3s, the same awesome anti-inflammatory amino acid in salmon. A joint study by the journal Nutrition and Cancer and the American Institute for Cancer Research found walnut consumption reduces breast cancer risk and retards cancer cell growth.

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Healthy recipes with plant-based foods are weapons against cancer

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Researchers Develop New Cells Meant to Form Blood Vessels, Treat Peripheral Artery Disease

Posted: October 14, 2014 at 11:52 am

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Newswise INDIANAPOLIS -- Researchers have developed a technique to jump-start the body's systems for creating blood vessels, opening the door for potential new treatments for diseases whose impacts include amputation and blindness.

The international team, led by scientists at the Indiana University School of Medicine, is targeting new therapies for illnesses such as peripheral artery disease, a painful leg condition caused by poor blood circulation. The disease can lead to skin problems, gangrene and sometimes amputation.

While the body has cells that specialize in repairing blood vessels and creating new ones, called endothelial colony-forming cells, these cells can lose their ability to proliferate into new blood vessels as patients age or develop diseases like peripheral arterial disease, said Mervin C. Yoder Jr., M.D., Richard and Pauline Klingler Professor of Pediatrics at IU and leader of the research team.

Peripheral artery disease patients can be given medication to improve blood flow, but if the blood vessels to carry that improved flow are reduced in number or function, the benefits are minimal. If "younger," more "enthusiastic" endothelial colony forming cells could be injected into the affected tissues, they might jump-start the process of creating new blood vessels. Gathering those cells would not be easy however -- they are relatively difficult to find in adults, especially in those with peripheral arterial disease. However, they are present in large numbers in umbilical cord blood.

Reporting their work in the journal Nature Biotechnology, the researchers said they had developed a potential therapy through the use of patient-specific induced pluripotent stem cells, which are normal adult cells that have been "coaxed" via laboratory techniques into reverting into the more primitive stem cells that can produce most types of bodily tissue. So, in one of the significant discoveries reported in the Nature Biotechnology paper, the research team developed a novel methodology to mature the induced pluripotent stem cells into cells with the characteristics of the endothelial colony-forming cells that are found in umbilical cord blood. Those laboratory-created endothelial colony-forming cells were injected into mice, where they were able to proliferate into human blood vessels and restore blood flow to damaged tissues in mouse retinas and limbs.

Overcoming another hurdle that has been faced by scientists in the field, the research team found that the cord-blood-like endothelial colony-forming cells grown in laboratory tissue culture expanded dramatically, creating 100 million new cells for each original cell in a little less than three months.

"This is one of the first studies using induced pluripotent stem cells that has been able to produce new cells in clinically relevant numbers -- enough to enable a clinical trial," Dr. Yoder said. The next steps, he said, include reaching an agreement with a facility approved to produce cells for use in human testing. In addition to peripheral artery disease, the researchers are evaluating the potential uses of the derived cells to treat diseases of the eye and lungs that involve blood flow problems.

A short video explaining the research is available here: http://youtu.be/nyPk_5bLdzs

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Diabetes researchers growing insulin-control cells by the billions

Posted: October 14, 2014 at 11:51 am

Good news: A scientific breakthrough offers the hope of new treatments for diabetics such as Ryan Buhlman, of Warrnambool, who uses an insulin pump. Photo: Damian White

Harvard researchers have pioneered a technique to grow by the billions the insulin-producing cells diabetics lack, a breakthrough that might create new ways to treat the disease.

The breakthrough comes after 15 years of seeking a bulk recipe for making beta cells, which sense the level of sugar in the blood and keep it in a healthy range by making precise amounts of insulin, according to Harvard scientists led by Douglas Melton, who have published their work in the journal Cell. The process begins with human stem cells, which have the ability to become any type of tissue or organ.

The technique is an important step towards understanding and treating diabetes, a condition in which the pancreas's beta cells are insufficient or dead. Diabetes affects 347 million people worldwide, and its chronic high blood-sugar levels can injure hearts, eyes, kidneys, the nervous system and other tissues.

"This is part of the holy grail of regenerative medicine or tissue engineering, trying to make an unlimited source of cells or tissues or organs that you can use in a patient to correct a disease," said Albert Hwa, director of discovery science at JDRF, a New York-based diabetes advocacy group that funded Dr Melton's work.

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The procedure for making mature, insulin-secreting beta cells had taken years of painstaking research that led to a 30-day, six-step recipe, Dr Melton said. Laboratories would be able to use the cells to test drugs and learn more about how diabetes occurs, he said.

"They had to go through an awful lot of trial and error to get to this," said Jeanne Loring, director of the Scripps Research Institute's Centre for Regenerative Medicine in La Jolla, California. "The proof will be in how well this protocol works for people in other laboratories."

People with type 2 diabetes, in which the body loses its ability to produce insulin over time, usually take drugs that boost its production. About 15 per cent of patients with type 2 diabetes could not make enough of the hormone, even with drug treatment, and had tohave daily injections to replace it, Dr Melton said.

Type 1 diabetes destroys beta cells, and patients must carefully monitor their food and exercise while injecting appropriate doses of insulin to keep blood-sugar levels in a healthy range. While self-treatment technology had improved, nothing could replace human beta cells for controlling blood sugar, Dr Melton said.

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Spinal cord injury victims may benefit from stem cell transplantation studies

Posted: October 14, 2014 at 11:46 am

PUBLIC RELEASE DATE:

13-Oct-2014

Contact: Robert Miranda cogcomm@aol.com Cell Transplantation Center of Excellence for Aging and Brain Repair

Putnam Valley, NY. (Oct. 13, 2014) Two studies recently published in Cell Transplantation reveal that cell transplantation may be an effective treatment for spinal cord injury (SCI), a major cause of disability and paralysis with no current restorative therapies.

Using laboratory rats modeled with SCI, researchers in Spain found in laboratory tests on cells harvested from rats - specifically ependymal progenitor cells (epSPCs), multipotent stem cells found in adult tissues surrounding the ependymal canal of the spinal cord - responded to a variety of compounds through the activation of purinergic receptors P2X4, P2X7, P2Y1 and P2Y4. In addition, the epSPCs responded to adenosine triphosphate (ATP) through this activation. ATP, a chemical produced by a wide variety of enzymes that works to transport energy within cells, is known to accumulate at the sites of spinal cord injury and cooperate with growth factors that induce remodeling and repair.

"The aim of our study was to analyze the expression profile of receptors in ependymal-derived neurospheres and to determine which receptors were functional by analysis of intercellular Ca2+ concentration," said study co-author Dr. Rosa Gomez-Villafuertes of the Department of Biochemistry at the Veterinary School at the University of Complutense in Madrid, Spain. "We demonstrated for the first time that epSPCs express functional ionotropic P2X4 and P2X7 and metabotropic P2Y1 and P2Y4 receptors that are able to respond to ATP, ADP and other nucleotide compounds."

When they compared the epSPCs from healthy rats to epSPCs from rats modeled with SCI, they found that a downregulation of P2Y1 and an upregulation of P2Y4 had occurred in the epSPCs in the SCI group.

"This finding opens an important avenue for potential therapeutic alternatives in SCI treatments based on purinergic receptor modulation," the researchers concluded.

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The study will be published in a future issue of Cell Transplantation and is currently freely available on-line as an unedited early e-pub at: http://www.ingentaconnect.com/content/cog/ct/pre-prints/content-CT-1257_Gomez_Villafuertes.

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Stem cell physical

Posted: October 13, 2014 at 8:49 pm

12 hours ago Stem cells show auxeticity; the nucleus expands, rather than thins, when it's stretched. Credit: Effigos AG

Looking at stem cells through physicists' eyes is challenging some of our basic assumptions about the body's master cells.

One of the many mysteries surrounding stem cells is how the constantly regenerating cells in adults, such as those in skin, are able to achieve the delicate balance between self-renewal and differentiation in other words, both maintaining their numbers and producing cells that are more specialised to replace those that are used up or damaged.

"What all of us want to understand is how stem cells decide to make and maintain a body plan," said Dr Kevin Chalut, a Cambridge physicist who moved his lab to the University's Wellcome Trust-MRC Cambridge Stem Cell Institute two years ago. "How do they decide whether they're going to differentiate or stay a stem cell in order to replenish tissue? We have discovered a lot about stem cells, but at this point nobody can tell you exactly how they maintain that balance."

To unravel this mystery, both Chalut and another physicist, Professor Ben Simons, are bringing a fresh perspective to the biologists' work. Looking at problems through the lens of a physicist helps them untangle many of the complex datasets associated with stem cell research. It also, they say, makes them unafraid to ask questions that some biologists might consider 'heretical', such as whether a few simple rules describe stem cells. "As physicists, we're very used to the idea that complex systems have emergent behaviour that may be described by simple rules," explained Simons.

What they have discovered is challenging some of the basic assumptions we have about stem cells.

One of those assumptions is that once a stem cell has been 'fated' for differentiation, there's no going back. "In fact, it appears that stem cells are much more adaptable than previously thought," said Simons.

By using fluorescent markers and live imaging to track a stem cell's progression, Simons' group has found that they can move backwards and forwards between states biased towards renewal and differentiation, depending on their physical position in the their host environment, known as the stem cell niche.

For example, some have argued that mammals, from elephants to mice, require just a few hundred blood stem cells to maintain sufficient levels of blood in the body. "Which sounds crazy," said Simons. "But if the self-renewal potential of cells may vary reversibly, the number of cells that retain stem cell potential may be much higher. Just because a certain cell may have a low chance of self-renewal today doesn't mean that it will still be low tomorrow or next week!"

Chalut's group is also looking at the way in which stem cells interact with their environment, specifically at the role that their physical and mechanical properties might play in how they make their fate decisions. It's a little-studied area, but one that could play a key role in understanding how stem cells work.

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