It would seem the beautiful foxglove plant has more uses than just the garden.

A novel drug screen in liver-like cells shows that cardiac glycosides, which are found in the leaves of the digitalis or foxglove plant, could reduce low-density lipoprotein (LDL) cholesterol differently than statins, potentially providing a new treatment for patients.

The foxglove plant in bloom on MUSC's campus.

These findings were reported by the Medical University of South Carolina researcher Stephen A. Duncan, D.Phil., SmartState Chair of Regenerative Medicine at MUSC, and colleagues in the April 6 issue of Cell Stem Cell.

Duncan said the glycosides were identified through a stem cell screen for compounds that could be used off-label for the treatment of high cholesterol. The nice thing about finding new uses for drugs already on the market is that they can be used relatively quickly in patients because most of the needed safety trials have already been completed.

Not everyone with high LDL cholesterol responds to statins. Statins increase levels of a cell surface receptor that removes LDL cholesterol from the bloodstream. However, statins do not work in patients with familial hypercholesterolemia (FH), who have a rare mutation in that receptor. It is an inherited disorder that leads to aggressive and premature cardiovascular disease. FH patients have very high cholesterol and can die of cardiovascular disease by their forties. The existing drugs for FH can cause fatty liver disease, and the best treatment is a liver transplant.

Duncan and his graduate student Max Cayo, who is finishing his M.D. at the Medical College of Wisconsin, developed a drug screen to identify an alternative to statins. Apolipoprotein B (apoB) is a molecule that liver cells use to make LDL. Drugs that decreased apoB could potentially lower cholesterol independently of the LDL receptor in FH patients and also in patients with other forms of high cholesterol.

FH was a perfect model for testing alternatives to statins. Yet the rarity of FH meant these liver cells were scarce. Duncans group made induced pluripotent stem cells out of skin fibroblasts taken from a single patient with FH. Stem cells continually double their numbers while in culture. This meant that a sample of converted skin cells from a single patient with FH provided a renewable source of liver-like cells that retained the mutation.

The group tested these liver-like cells with the SPECTRUM library, a collection of 2,300 pharmaceuticals, many of which have reached clinical trials. Surprisingly, all nine cardiac glycosides in the collection, some widely prescribed for heart failure, reduced apoB in liver-like cells from the patient with FH. In further tests, they also lowered apoB in human hepatocytes and in mice engineered to grow normal human livers without the FH mutation.

Next, the team combed through more than five thousand medical records of patients prescribed cardiac glycosides for heart failure who also had LDL cholesterol records. Similar drops in LDL levels were observed in these patients as in a matching group of patients prescribed statins.

This study provides the first evidence that cardiac glycosides could potentially reduce LDL cholesterol independently of the LDL receptor, where statins act, by reducing apoB.

The cardiac glycosides are always prescribed with care, as they are known to be toxic at high doses. However, they could offer inexpensive life-saving options for patients with FH. Additionally, a cardiac glycoside in a low dose could conceivably provide an added benefit to patients already taking a statin. Duncan is exploring plans for a clinical trial that would determine the correct dose in hypercholesterolemia patients.

Using patient stem cells to screen drugs that are already on the market is a great way to investigate treatments for liver diseases.

There are so few livers available for transplant, Duncan said. Having the stem cell model where we make liver cells in the culture dish opens up a possibility of using this not only to investigate a disease, but also as a way to discover drugs that could fix a disease.

This article has been republished frommaterialsprovided by theMedical University of South Carolina. Note: material may have been edited for length and content. For further information, please contact the cited source.

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SPECTRUM Drug Screen Reveals Fox Gloves Can Treat High Cholesterol - Technology Networks

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It will be several weeks before Jonathan Pitre finds out if his second stem-cell transplant was successful. Tina Boileau / -

The perilous wait now begins for Jonathan Pitre.

Pitre, 16, was transfused with blood and marrow drawn from his mothers hip late Thursday afternoon. The stem-cell rich material holds the power to alter the course of Pitres aggressive skin disease, epidermolysis bullosa (EB), and change his life.

So far, so good, saidPitres mother, Tina Boileau.

It will be several weeks before Pitre finds out whether the transplant has worked its magic.

While waiting for that answer, theRussell teenager will have to travel the most difficult part of his medical journey: a time when his immune system is at its lowest ebb, and when he feels the full effects of high-dose chemotherapy and radiation.

His physician, Dr. Jakub Tolar, has warned that the period represents the highest risk for complications, the most common of which are infections and graft-versus-host disease (GVHD). It is a potentially life-threatening situation in which the implanted stem cells produce T-cells that attack normal cells.

In about two weeks time, doctors will start to look for signs that Boileaus stem cells have successfully established themselves in Pitres bone marrow.The presence of white blood cells is one of the earliest signs of stem-cell growth; an improvement in the condition of Pitres skin could also signal that the stem cells have started to work.

Last year, after his first stem-cell transplant, Pitre and his mother were thrilled when doctors discovered new white cells in his bloodstream. But their hopes were crushed when tests showed Pitres own stem cells had recolonized his bone marrow, and were producing the cells.

This time, Boileau said, they will wait to see more lab results before getting their hopes too high.

I think we will have that uncertainly until we know for sure through skin and bone marrow biopsies that the engraftment worked, she said.

Boileau went into surgery early Thursday morning to have blood and bone marrow drawn from her hip. She was at her sons bedside later in the afternoon to watch as the stem cells dripped through an intravenous tube connected to the right atrium of her sons heart.

If the transplant works, Boileaus stem cells will establish themselves in her sons bone marrow, grow, divide and make new blood cells equipped with the power to provide Pitre with the key protein he needs to rebuild his damaged skin.

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His stem-cell transplant complete, the wait begins for Jonathan Pitre - Ottawa Citizen

Pennsylvania state trooper Matt Uram was talking with his wife at a July Fourth party in 2009 when a misjudged spray of gasoline burst through a nearby bonfire and set him alight. Flames covered the entire right side of his body, and after he fell to the ground to smother them, his wife beat his head with her bare hands to put out his burning hair. It was only on the way to the ER, as the shock and adrenaline began to wear off, that the pain set in. It was intense, he says. If you can imagine what pins and needles feel like, then replace those needles with matches.

From the hospital, Uram was transferred to the Mercy Burn Center in Pittsburgh, where doctors removed all of the burned skin and dressed his wounds. It was on the border between a second- and third-degree burn, and he was told to prepare for months of pain and permanent disfigurement. Not long after this assessment, however, a doctor asked Uram if he would be willing to take part in an experimental trial of a new device.

The treatment, developed by German researcher Dr. Jrg Gerlach, was the worlds first to use a patients stem cells to directly heal the skin. If successful, the device would mend Urams wounds using his bodys ability to regenerate fully functioning skin. Uram agreed to the procedure without hesitation.

Five days after the accident, surgeons removed a small section of undamaged skin from Urams right thighabout the size of a postage stampand used it to create a liquid suspension of his stem cells that was sprayed in a fine mist onto the damaged skin. Three days later, when it was time to remove the bandages and re-dress the wounds, his doctor was amazed by what he saw. The burns were almost completely healed, and any risk of infection or scarring was gone.

Pennsylvania State Trooper Matt Uram's arm eventually healed without scarring. RenovaCare

A study subsequently published in the scientific journal Burns described how the spray was able to regrow the skin across the burn by spreading thousands of tiny regenerative islands, rather than forcing the wound to heal from its edge to the inside. The technique meant reducing the healing time and minimizing complications, with aesthetically and functionally satisfying outcomes, the paper stated.

Dozens more burn victims in Germany and the U.S. were successfully treated with the spray following Urams procedure, and in 2014 Gerlach sold the technology to RenovaCare. The medical technology startup has now transformed the proof-of-concept device from a complicated prototype into a user-friendly product called a SkinGun, which it hopes clinicians will be able to use outside of an experimental setting. For that to happen, RenovaCare is preparing clinical studies for later this year, with the aim of Food and Drug Administration approval for the SkinGun.

Once these obstacles are overcome, RenovaCare CEO Thomas Bold believes, the SkinGun can compete with, or even replace, todays standard of care.

Current treatment of severe burns involves transplanting healthy skin from one area of the body and stitching it to another in a process called skin grafting or mesh skin grafting. It is a painful procedure that creates an additional wound at the donor site and can cause restricted joint movement because the transplanted skin is unable to grow with the patient. It is able to cover an area only two to three times as large as the harvested patch. The current standard of care is just horrible, says Bold. We are part of regenerative medicineit is the medicine of the future and will be life-changing for patients.

RenovaCare's SkinGun sprays a liquid suspension of a patient's stem cells onto a burn or wound in order to regrow the skin without scars. RenovaCare

Beyond regulatory matters, there are also limitations to the technology that make it unsuitable for competing with treatments of third-degree burns, which involve damage to muscle and other tissue below the skin. Still, stem cell researcher Sarthak Sinha believes that while the SkinGun may not be that advanced yet, it shows the vast potential of this form of regenerative medicine. What I see as the future of burn treatment is not skin repair but rather functional regeneration of skin and its appendagessuch as hair follicles, glands and fat, says Sinha. This could be achieved by engaging deeper layers of skin and its resident stem cells to partake in tissue regeneration.

Research is already underway at RenovaCare to enable treatment of third-degree burns, which Bold describes as definitely within the range of possibility. Bold claims the adaptations to the SkinGun would allow it to treat other damaged organs using a patients stem cells, but for now the company is focusing solely on burns and wounds to skinthe largest organ of the human body.

Urams burns are now completely unnoticeable. There is no scar tissue or even pigment discoloration, and the regenerated skin even tans. If I show someone where I was burnt, I bet $100,000 they couldnt tell, he says. Theres no scars, no residual pain; its like the burn never happened. Its a miracle.

Uram is frustrated that the treatment is not available to other burn victims, particularly children. I want to see the FDA get off their butts and approve this, he says. A grown man like me to be scarred is OK, but think about the kids that have to live the rest of their lives with pain and scarring. Thats not OK.

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Spray-On Skin: 'Miracle' Stem Cell Treatment Heals Burns Without ... - Newsweek

Over recent years, I have seen a growing interest in stem cells and a particular preparation called Platelet Rich Plasma (PRP). Many famous athletes including Tiger Woods have received PRP for various musculoskeletal problems and some have credited it with their accelerated healing and more rapid return to play.

PRP is plasma, the liquid part of blood, concentrated with many more platelets than typically found there. Platelets are known for their importance in clotting blood, but they also contain hundreds of proteins called growth factors. These are responsible for the cascade of events naturally involved in tissue repair. Your own innate stem cells are attracted to the site of injury and play a critical role in the healing process.

Typically, PRP is isolated from your own blood, drawn in the office while you wait. The highly concentrated growth factors are then delivered back into the body at the site of interest. PRP injections have been used for musculoskeletal problems such as sprained knees, osteoarthritis, and chronic tendon injuries. Previously, these types of conditions were treated with medications, physical therapy, and surgery, but PRP recipients commonly report less pain and stronger, more stable joints. It may even promote new cartilage formation in aging joints enabling you to put off joint replacement surgery.

PRP can also be very effective in treating chronic tendon injuries, especially tennis elbow, a common injury of the tendons on the outside of the elbow. Previously, cortisone injections were commonly used, but we know steroids will ultimately weaken tendons and promote rupture. In contrast, now PRP treatments lead to stronger tendons.

Promoting healing after tendon surgery is another use for PRP. For example, an athlete with a completely torn heel cord may require surgery to repair the tendon. Healing of the torn tendon can potentially be improved by treating the injured area with PRP during surgery. With a shorter recovery time, less chronic pain and stronger tissue, you can see why athletes love PRP!

More recently, PRP is being used extensively in aesthetic medicine to keep us looking younger and to promote hair growth. In the same ways the growth factors in PRP facilitate tissue repair from injury or surgery, they also regenerate aging skin. PRP injected into the facial skin has been called the vampire facial made famous by some Hollywood stars.

Today we use a more advanced technique called micro-needling. The PRP is layered across your face and delivered to the skin using a handheld device called a Micropen. This device has 12 tiny micro-needles that drive the PRP in, calling in the tissue repair team to get to work! The result is accelerated collagen production with new, thicker, stronger collagen. The procedure is well tolerated and done in the office while you are awake. It takes less than a couple of hours to complete and usually two to three treatments are recommended spaced four to six weeks apart. The collagen repair process can take four to six weeks, we expect to see the full results blossom over the course of months and continue to improve over a year.

The best thing about PRP Micropen Facelift is that theres not serious downtime like you get with laser resurfacing or surgery. Plus, unlike dermal fillers, which will fade in months, these results will continue to improve over the year. Most commonly we treat faces, but the procedure is safe to use all over the body including necks, chests, hands, and even eye lids. It is also quite helpful for minimizing and fading stretch marks.

If you have any questions or want to learn more about PRP for musculoskeletal or skin rejuvenation, please plan to attend our free interactive community lecture on this topic at the Coronado Library Winn Room from 6-7PM on Thursday 04/06/2017!

Lauren Mathewson, ND is Board certified in naturopathic medicine and Patrick Yassini, MD is board certified in family medicine, integrative and holistic medicine. They practice at Peak Health Group, 131 Orange Avenue, #100, Coronado, Calif.; the office number is (619) 522-4005.

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Now You Can Harness Your Own Stem Cells - Coronado Eagle ... - Coronado Eagle and Journal

As a kid, I was always intrigued with potions and products. My father worked as a scientist, whose specialty was chemistry as well as business. For many years he worked as the Director of Research and Development for the Mennen Company. Perhaps this is where my love of products and researching products began.

Like many women, my skin can be difficult at times. I have eczema which makes it intermittently sensitive, so I have to be careful of the products I use. While researching these products, I also looked into the science supporting them.

As fate would have it while exploring some interesting articles on my Twitter feed recently, I came across an intriguing tweet I just couldnt ignore. It was a tweet by a glamorous NYC dermatologist who was talking about how excited she was to receive her Lifeline Skin Care products in the mail. Her excitement was so infectious; I decided to look into these products for myself, and looking into them, ultimately led me to buy them and to contact the company to see if we could collaborate together, so I could spread the word.

While researching Lifeline Skin Cares products, I also looked into the science supporting them. Lifeline Skin Care products use something I had never heard of before; they use human, non-embryonic stem cells as one of the main ingredients to help tone and reduce the signs of aging.

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As a therapist, I not only look for products that work well and that I believe in, but also look at the philosophy of the company. Lifeline Skin Care was a socially conscious company and fit that standard.

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The original goal for these researchers was to find a cure for diabetes and Parkinsons disease. These scientists created the first non-embryonic human stem cells. This discovery made finding cures for Parkinsons disease and corneal disease more promising. Currently, some of ISCOs most promising research is in the field of Parkinsons disease.

Parkinsons disease (PD) is a long-term degenerative disease of the central nervous system. It mainly affects the motor system and its symptoms usually have a slow-onset. In early stages, the disease is characterized by shaking, slow movement, difficulty in walking, and rigidity. In time, thinking and behavioral problems may occur. Advanced stages of the disease bring dementia.

istock jm1366

International Stem Cell Corporation (ISCO), is the parent company of Lifeline Skin Care and has devoted many years of research to improve this terrible disease. The company has developed a unique method of creating human neural stem cells which when introduced into the brain, promote the recovery of dopaminergic neurons, the brain cells that are originally affected and cause the disease symptoms. ISCOs preclinical studies showed that the administration of these neural stem cells were safe and improved motor symptoms. To date, 3 of the planned total of 12 patients, have entered the clinical trial and have received neural stem cells. At this point in time, all patients have been discharged from their hospital settings and are observed to be meeting clinical expectations.

Lifeline Skin care (LSC) - a subsidiary of ISCO - uses the extracts from human stem cells, (produced by ISCO), and developed for the skin in order to improve the signs of ageing. The latest technology being used to advance a cure for PD is now available for the skin in a line of products produced by LSC. The profits from the sale of these skin care products go directly to ISCO in order to fund the development of a therapy for PD.

From a skincare perspective, not only did Lifeline Skin Cares products feel good on my face, but I started to notice that my skin appeared brighter and less wrinkles, especially around my eyes (love that!).

From a psychological perspective, the younger we look and feel, the more optimistic and hopeful we tend to be about life and future options. I like the idea of feeling young, looking forever fabulous and most of all, being healthy.

Fortunately, Lifeline Skin Care found a way to help women and men look and feel their very best while scientists from their parent company work toward eradicating illness by using their special non-embryonic stem cell technology. Beauty is more than skin-deep; beauty can be on a mission, too.

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The International Stem Cell Corporation, a Company Dedicated to Curing Parkinson's Disease - Huffington Post

Mouse heart section showing human progenitor cells that formed functional human blood vessels. Purple color signifies human blood vessels, red staining signifies the blood vessels of the mouse that received the human cell implants. Credit: UIC

Researchers from the University of Illinois at Chicago have identified a molecular switch that converts skin cells into cells that make up blood vessels, which could ultimately be used to repair damaged vessels in patients with heart disease or to engineer new vasculature in the lab. The technique, which boosts levels of an enzyme that keeps cells young, may also circumvent the usual aging that cells undergo during the culturing process. Their findings are reported in the journal Circulation.

Scientists have many ways to convert one type of cell into another. One technique involves turning a mature cell into a pluripotent stem cell one that has the ability to become any type of cell and then using chemical cocktails to coax it into maturing into the desired cell type. Other methods reprogram a cell so that it directly assumes a new identity, bypassing the stem-cell state.

In the last few years, scientists have begun to explore another method, a middle way, that can turn back the clock on skin cells so that they lose some of their mature cell identity and become more stem-like.

They dont revert all the way back to a pluripotent stem cell, but instead turn into intermediate progenitor cells, says Dr. Jalees Rehman, associate professor of medicine and pharmacology at UIC, who led the team of researchers. Progenitor cells can be grown in large quantities sufficient for regenerative therapies. And unlike pluripotent stem cells, progenitor cells can only differentiate into a few different cell types. Rehman calls this method to produce new cells partial de-differentiation.

Other groups have used this technique to produce progenitor cells that become blood vessel cells. But until now, researchers had not fully understood how the method worked, Rehman said.

Without understanding the molecular processes, it is difficult for us to control or enhance the process in order to efficiently build new blood vessels, he said.

His group discovered that the progenitors could be converted into blood vessel cells or into red blood cells, depending on the level of a gene transcription factor called SOX17.

The researchers measured the levels of several genes important for blood vessel formation. They saw that as progenitor cells were differentiating into blood vessel cells, levels of the transcription factor SOX17 became elevated.

When they increased levels of SOX17 even more in the progenitor cells, they saw that differentiation into blood vessel cells was enhanced about five-fold. When they suppressed SOX17, the progenitor cells produced fewer endothelial cells and instead generated red blood cells.

It makes a lot of sense that SOX17 is involved because it is abundant in developing embryos when blood vessels are forming, Rehman said.

When the researchers embedded the human progenitor cells into a gel and implanted the gels in mice, the cells organized into functional human blood vessels. Skin cells that had not undergone a conversion did not form blood vessels when similarly implanted.

When they implanted the progenitor cells into mice that had sustained heart damage from a heart attack, the implanted cells formed functional human blood vessels in the mouse hearts and even connected with existing mouse blood vessels to significantly improve heart function.

The human adult skin cells used by Rehmans team can easily be obtained by a simple skin biopsy.

This means that one could generate patient-specific blood vessels or red blood cells for any individual person, Rehman said. Using such personalized cells reduces the risk of rejection, he said, because the implanted blood vessels would have the same genetic makeup as the recipient.

Rehman and his colleagues noticed something else about the progenitor cells they had elevated levels of telomerase the anti-aging enzyme that adds a cap, or telomere, to the ends of chromosomes. As the caps wear away a little bit each time a cell divides, they are believed to contribute to aging in cells, whether in the body or growing in culture in the laboratory.

The increase in telomerase we see in the progenitor cells could be an added benefit of using this partial de-differentiation technique for the production of new blood vessels for patients with cardiac disease, especially for older patients, Rehman said. Their cells may already have shortened telomeres due to their advanced age. The process of converting and expanding these cells in the lab could make them age even further and impair their long-term function. But if the cells have elevated levels of telomerase, the cells are at lower risk of premature aging.

While telomerase has benefits, the enzyme is also found in extremely high levels in cancer cells, where it keeps cell division in overdrive.

We were concerned about the risk of tumor formation, Rehman said, but the researchers didnt observe any in these experiments. But to truly determine the efficacy and safety of these cells for humans, one needs to study them over even longer time periods in larger animals.

Reference:

Zhang, L., Jambusaria, A., Hong, Z., Marsboom, G., Toth, P. T., Herbert, B., . . . Rehman, J. (2017). SOX17 Regulates Conversion of Human Fibroblasts into Endothelial Cells and Erythroblasts via De-Differentiation into CD34 Progenitor Cells. Circulation. doi:10.1161/circulationaha.116.025722

This article has been republished frommaterialsprovided by the University of Illinois at Chicago. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Partial De-differentiation Converts Skin Cells into Blood Vessel Cells ... - Technology Networks

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Turning skin cells into blood vessel cells while keeping them young Science Daily Other methods reprogram a cell so that it directly assumes a new identity, bypassing the stem-cell state. In the last few years, scientists have begun to explore another method, a middle way, that can turn back the clock on skin cells so that they lose ...

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M

ice that walk straight and fluidly dont usually make scientists exult, but these did: The lab rodents all had a mouse version of Parkinsons disease and only weeks before had barely been able to lurch and shuffle around their cages.

Using a trick from stem-cell science, researchers managed to restore the kind of brain cells whose death causes Parkinsons. And the mice walked almost normally.The same technique turned human brain cells, growing in a lab dish, into the dopamine-producing neurons that are AWOL in Parkinsons, scientists at Swedens Karolinska Institute reportedon Monday in Nature Biotechnology.

Success in lab mice and human cells is many difficult steps away from success in patients. The study nevertheless injected new life into a promising approach to Parkinsons that has suffered setback after setback replacing the dopamine neurons that are lost in the disease, crippling movement and eventually impairing mental function.

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This is not going to happen in five years or possibly even 10, but Im excited about the potential of this kind of cell replacement therapy, said James Beck, chief scientific officer of the Parkinsons Foundation, which was not involved in the study. It could really give life back to someone with Parkinsons disease.

There is no cure for Parkinsons, a neurodegenerative disease that affects an estimated 10 million people worldwide, most prominently actor Michael J. Fox. Drugs that enable the brain to make dopamine help only somewhat, often causing movement abnormalities called dyskinesia as well as bizarre side effects such as a compulsion to gamble; they do nothing to stop the neurodegeneration.

As Parkinsons patients wait, Fox Foundation and scientist feud over drug trial

Rather than replacing the missing dopamine, scientists led by Karolinskas Ernest Arenas tried to replace dopamine neurons but not in the way that researchers have been trying since the late 1980s. In that approach, scientists obtained tissue containing dopamine neurons from first-trimester aborted fetuses and implanted it intopatients brains.Although a 2001clinical trialfound that the transplants partly alleviated the rigidity and tremors of Parkinsons, the procedure caused serious dyskinesia in about 20 percent of patients, Beck said. More problematic is that fetal issue raises ethical concerns and is in short supply.

It was clear that usable fragments of brain tissue were extremely difficult to recover, said Dr. Curt Freed, of the University of Colorado, who pioneered that work.

Instead, several labs have therefore used stem cells to produce dopamine neurons in dishes. Transplanted into the brains of lab rats with Parkinsons, the neurons reduced rigidity, tremor, and other symptoms. Human studies are expected to begin in the US and Japan this year or next, Beck said.

In the Karolinska approach, there is no need to search for donor cells and no cell transplantation or [need for] immunosuppression to prevent rejection, Arenas told STAT. Instead, he and his team exploited one of the most startling recent discoveries in cell biology: that certain molecules can cause one kind of specialized cell, such as a skin cell, to pull a Benjamin Button, aging in reverse until they become like the embryonic cells called stem cells. Those can be induced to morph into any kind of cell heart, skin, muscle, and more in the body.

Muhammad Ali and Parkinsons disease: Was boxing to blame?

Arenas and his team filled harmless lentiviruses with a cocktail of four such molecules. Injected into the brains of mice with Parkinsons-like damage, the viruses infected plentifulbrain cells called astrocytes. (The brains support cells, astrocytes perform jobs like controlling blood flow.)The viruses also infected other kinds of cells, but their payload was designed to work only in astrocytes, and apparently caused no harm to the other cells.

The molecules, called transcription factors, reprogrammed some of the astrocytes to become dopamine neurons, which were first detected three weeks later in the mouse brains. The dopamine neurons were abundant 15 weeks later, an indication that after changing into dopamine neurons the astrocytes stayed changed.

Five weeks after receiving the injections, the mice, which used to have Parkinsons-like gait abnormalities, walked as well as healthy mice. That suggests that direct reprogramming [of brain cells] has the potential to become a novel therapeutic approach for Parkinsons, Arenas told STAT.

That could have value for preserving the brain circuitry destroyed by Parkinsons, said Colorados Freed.

A lot of hurdles need to be overcome before this becomes a Parkinsons treatment. The Trojan horse system for delivering the reprogramming molecules inside viruseswould need to turn more astrocytes into dopamine neurons and leave other kinds of cells alone: Although viruses getting into mouse brain cells apparently caused no harm, that might not be so in people. We will need to use virus with selective [attraction] for astrocytes, Arenas said.

The morphed cells would presumably be ravaged by whatever produced Parkinsons in the first place. But in other cell transplants, Arenas said, the disease catches up with transplanted cells in 15 to 20 years, buying patients a good period of time. He thinks it might be possible to give patients a single injection but hold off some of the reprogramming with a drug, turning it on when the brain again runs short of dopamine neurons.

The basic technology to develop such strategies currently exists, he said.

The Karolinska lab is working to make the techniquesafer and more effective, including by using viruses that would deliver reprogramming molecules only to astrocytes. We are open to collaborations aimed at human studies, Arenas said.

Would patients be willing to undergo brain injections? People with Parkinsons disease, Beck said, are willing to go through a lot for any hope of improvement.

Sharon Begley can be reached at sharon.begley@statnews.com Follow Sharon on Twitter @sxbegle

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Mighty morphed brain cells cure Parkinson's in mice, but human trials still far off - STAT

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Stem cell drug screen yields potential alternative to statins Science Daily Next, they generated induced pluripotent stem cells from these skin cells. Stem cells continually double their numbers while in culture. This meant that a sample of converted skin cells from a single patient with FH provided a renewable source of liver ...

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April 6, 2017 A mouse heart section showing human progenitor cells that formed functional human blood vessels. Purple color signifies human blood vessels, red staining signifies the blood vessels of the mouse that received the human cell implants. Credit: Jalees Rehman.

Researchers from the University of Illinois at Chicago have identified a molecular switch that converts skin cells into cells that make up blood vessels, which could ultimately be used to repair damaged vessels in patients with heart disease or to engineer new vasculature in the lab. The technique, which boosts levels of an enzyme that keeps cells young, may also circumvent the usual aging that cells undergo during the culturing process. Their findings are reported in the journal Circulation.

Scientists have many ways to convert one type of cell into another. One technique involves turning a mature cell into a "pluripotent" stem cellone that has the ability to become any type of celland then using chemical cocktails to coax it into maturing into the desired cell type. Other methods reprogram a cell so that it directly assumes a new identity, bypassing the stem-cell state.

In the last few years, scientists have begun to explore another method, a middle way, that can turn back the clock on skin cells so that they lose some of their mature cell identity and become more stem-like.

"They don't revert all the way back to a pluripotent stem cell, but instead turn into intermediate progenitor cells," says Dr. Jalees Rehman, associate professor of medicine and pharmacology at UIC, who led the team of researchers. Progenitor cells can be grown in large quantities sufficient for regenerative therapies. And unlike pluripotent stem cells, progenitor cells can only differentiate into a few different cell types. Rehman calls this method to produce new cells "partial de-differentiation."

Other groups have used this technique to produce progenitor cells that become blood vessel cells. But until now, researchers had not fully understood how the method worked, Rehman said.

"Without understanding the molecular processes, it is difficult for us to control or enhance the process in order to efficiently build new blood vessels," he said.

His group discovered that the progenitors could be converted into blood vessel cells or into red blood cells, depending on the level of a gene transcription factor called SOX17.

The researchers measured the levels of several genes important for blood vessel formation. They saw that as progenitor cells were differentiating into blood vessel cells, levels of the transcription factor SOX17 became elevated.

When they increased levels of SOX17 even more in the progenitor cells, they saw that differentiation into blood vessel cells was enhanced about five-fold. When they suppressed SOX17, the progenitor cells produced fewer endothelial cells and instead generated red blood cells.

"It makes a lot of sense that SOX17 is involved because it is abundant in developing embryos when blood vessels are forming," Rehman said.

When the researchers embedded the human progenitor cells into a gel and implanted the gels in mice, the cells organized into functional human blood vessels. Skin cells that had not undergone a conversion did not form blood vessels when similarly implanted.

When they implanted the progenitor cells into mice that had sustained heart damage from a heart attack, the implanted cells formed functional human blood vessels in the mouse heartsand even connected with existing mouse blood vessels to significantly improve heart function.

The human adult skin cells used by Rehman's team can easily be obtained by a simple skin biopsy.

"This means that one could generate patient-specific blood vessels or red blood cells for any individual person," Rehman said. Using such personalized cells reduces the risk of rejection, he said, because the implanted blood vessels would have the same genetic makeup as the recipient.

Rehman and his colleagues noticed something else about the progenitor cells - they had elevated levels of telomerase - the "anti-aging" enzyme that adds a cap, or telomere, to the ends of chromosomes. As the caps wear away a little bit each time a cell divides, they are believed to contribute to aging in cells, whether in the body or growing in culture in the laboratory.

"The increase in telomerase we see in the progenitor cells could be an added benefit of using this partial de-differentiation technique for the production of new blood vessels for patients with cardiac disease, especially for older patients," Rehman said. "Their cells may already have shortened telomeres due to their advanced age. The process of converting and expanding these cells in the lab could make them age even further and impair their long-term function. But if the cells have elevated levels of telomerase, the cells are at lower risk of premature aging."

While telomerase has benefits, the enzyme is also found in extremely high levels in cancer cells, where it keeps cell division in overdrive.

"We were concerned about the risk of tumor formation," Rehman said, but the researchers didn't observe any in these experiments. "But to truly determine the efficacy and safety of these cells for humans, one needs to study them over even longer time periods in larger animals."

Explore further: Adult stem cells help build human blood vessels in engineered tissues

More information: Lianghui Zhang et al, SOX17 Regulates Conversion of Human Fibroblasts into Endothelial Cells and Erythroblasts via De-Differentiation into CD34Progenitor Cells, Circulation (2017). DOI: 10.1161/CIRCULATIONAHA.116.025722

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Read more from the original source:
Turning skin cells into blood vessel cells while keeping them young - Medical Xpress

Scientists at the Medical University of South Carolina (MUSC) have found that a class of heart failure drugs might decrease low-density lipoprotein (LDL) cholesterol levels in patients who do not respond to statins. In a study appearing in the April 6, 2017 issue ofCell Stem Cell, cardiac glycosides reduced levels of a precursor of LDL in liver-like cells, and patients taking cardiac glycosides for heart failure had low LDL.

Not everyone with high LDL cholesterol responds to statins. Statins increase levels of a cell surface receptor that removes LDL cholesterol from the bloodstream. However, statins do not work in patients with familial hypercholesterolemia (FH), who have a rare mutation in that receptor. FH patients have very high cholesterol and die of cardiovascular disease by their forties. The existing drugs for FH can cause fatty liver disease, and the best treatment is a liver transplant.

Stephen A. Duncan, D. Phil., SmartStateTMChair of Regenerative Medicine at MUSC, and his colleagues, including Max A. Cayo, Ph.D., an MSTP student at the Medical College of Wisconsin, developed a drug screen to identify an alternative to statins. They focused on apolipoprotein B (ApoB), a molecule that liver cells use to make LDL and which is normal in patients with FH. Drugs that decrease ApoB could potentially lower cholesterol independently of the LDL receptor in FH patients and also in patients with other forms of high cholesterol.

FH was a perfect model for testing alternatives to statins. Yet the rarity of FH meant these liver cells were scarce. Duncan's group obtained skin cells from a patient with the rare disorder from the Next Generation Genetic Association Studies consortium of the National Heart, Lung, and Blood Institute, which studies genetic mutations linked to cardiovascular diseases. Next, they generated induced pluripotent stem cells from these skin cells. Stem cells continually double their numbers while in culture. This meant that a sample of converted skin cells from a single patient with FH provided a renewable source of liver-like cells that retained the mutation.

The team treated their liver-like cells with the SPECTRUM drug library, a collection of 2300 pharmaceuticals, many of which have reached clinical trials. In a surprising finding, all nine cardiac glycosides in the library, some once widely prescribed for heart failure, reduced ApoB levels in liver-like cells from the patient with FH, ranging from 29 percent (ouabain) to 38 percent (digoxin) to 73 percent (gitoxin). In further tests, they also lowered ApoB levels in human hepatocytes and reduced them by 30 percent in mice engineered to grow normal human livers without the FH mutation at doses eight times below their toxicity thresholds. Molecular tests revealed that glycosides shorten the lifetime of the ApoB molecule, in part by increasing how quickly it is degraded.

As everyone needs ApoB to make LDL cholesterol, this was proof that cardiac glycosides could potentially also work in patients with other forms of high cholesterol. To find out, the team combed through more than five thousand records of patients prescribed cardiac glycosides for heart failure who also had LDL cholesterol records. On average, LDL cholesterol levels were lower in those taking a cardiac glycoside (reduction of 9 mg/dL) or a statin (reduction of 14 mg/dL) than in those not taking any drug. No difference in LDL cholesterol levels was noted between those taking an angiotensin-converting enzyme inhibitor, another heart failure drug with no known role in cholesterol production, and those not taking any drug. Duncan's team also found patients who had LDL measurements recorded both before and after being prescribed a cardiac glycoside. LDL cholesterol dropped in 16 out of 21 patients and by an average of nearly 26 points, which was similar to the 32-point drop seen in a matching group of patients prescribed statins.

This study contains the first evidence to date that cardiac glycosides could potentially reduce LDL cholesterol independently of the LDL receptor, where statins act, by promoting ApoB degradation.

It is not clear from this study whether cardiac glycosides decrease LDL cholesterol in patients who do not have heart failure or at what dose they should be used. The cardiac glycosides have narrow ranges of efficacy for the treatment of heart failure, above which they can be toxic. However, they could offer inexpensive, life-saving options for patients with FH. Digoxin, the cardiac glycoside most commonly prescribed for heart failure, costs less than one dollar per day. Additionally, a cardiac glycoside in a low dose could conceivably provide an added benefit to patients already taking a statin. Finally, using stem cell-based screens of drugs that are already on the market is an innovative way to investigate treatments for rare liver diseases.

"There are so few livers available for transplant," says Duncan. "Having the stem cell model where we make liver cells in the culture dish opens up a possibility of using this not only to investigate a disease, but also as a way to discover drugs that could fix a disease."

Continued here:
Stem Cell Drug Screen Yields Potential Alternative to Statins - R & D Magazine

No two stem cells are identical, even if they are genetic clones. This stunning diversity is revealed today in an enormous publicly available online catalogue of 3D stem cell images. The visuals were produced using deep learning analyses and cell lines altered with the gene-editing tool CRISPR. And soon the portal will allow researchers to predict variations in cell layouts that may foreshadow cancer and other diseases.

The Allen Cell Explorer, produced by the Allen Institute for Cell Science in Seattle, Washington, includes a growing library of more than 6,000 pictures of induced pluripotent stem cells (iPS) key components of which glow thanks to fluorescent markers that highlight specific genes.

The Cell Explorer complements ongoing projects by several groups that chart the uniqueness of single cells at the level of DNA, RNA and proteins. Rick Horwitz, director of the Allen Institute for Cell Science, says that the institutes images may hasten progress in stem cell research, cancer research and drug development by revealing unexpected aspects of cellular structure. You cant predict the outcome of a football game if you know stats on all the players but have never watched a game.

The project began about a year ago with adult skin cells that had been reprogrammed into an embryonic-like, undifferentiated state. Horwitz and his team then used CRISPRCas9 to insert tags in genes to make structures within the cells glow. The genes included those that code for proteins that highlight actin filaments, which help cells to move and maintain their shape. It quickly became clear that the cells, which were all genetic clones from the same parent cell, varied in the placement, shape and number of their components, such as mitochondria and actin fibres.

Computer scientists analysed thousands of the images using deep learning programs and found relationships between the locations of cellular structures. They then used that information to predict where the structures might be when the program was given just a couple of clues, such as the position of the nucleus. The program learned by comparing its predictions to actual cells.

The deep learning algorithms are similar to those that companies use to predict peoples preferences, Horwitz says. If you buy a chainsaw at Amazon, it might then show you chain oil and plaid shirts.

The 3D interactive tool based on this deep learning capability should go live later this year. At the moment, the site shows a preview of how it will work using side-by-side comparisons of predicted and actual images.

Benjamin Freedman, a cell biologist at the University of Washington in Seattle, looks forward to playing with the Cell Explorers predictive function once the Allen Institute team has taught their algorithm to recognize more iPS cells that have been changed genetically or chemically. For example, Freedman says he could delete a gene related to kidney disease in one of the fluorescently tagged stem cells from the Allen Institute and see how the mutation affects the glowing structure. Then he could use the sites modelling tool to determine how other cellular components might be altered. Ultimately, Freedman says, we want to understand processes at the cellular level that cause disease in the kidney as a whole.

In the coming months, Allen Institute researchers will update the site with images of stem cells at different stages of cell division, and as they transform into distinct cell types, such as heart and kidney cells. Catching cells at different time points can be crucial to identifying fundamental processes, says Horwitz.

Allen Institute for Cell Science

Structural differences in the DNA (purple) and cellular membrane (blue) of genetically identical stem cells.

The Allen Institutes visual emphasis on stem cells dovetails with a number of efforts to catalogue other aspects of cells. For example, the London-based charity Cancer Research UK is creating interactive virtual-reality models of breast cancer cells in tumours. And an international effort called the Human Cell Atlas seeks to define all human cell types in terms of their molecular profiles, including DNA sequences, RNA transcripts and proteins.

Aviv Regev, a computational biologist at the Broad Institute in Cambridge, Massachusetts, who is working on the Human Cell Atlas, says that the Allen Cell Explorer complements her project by focusing on the look of cellular features as opposed to how genes, RNA and proteins interact within the cell. The community is accepting that there are a lot of differences between cells that we thought were the same until recently, she says, so now were taking an unbiased approach to learn about pieces in the puzzle we didnt know existed before.

Originally posted here:
Machine learning predicts the look of stem cells - Nature.com

Last week, a patient with blurry vision in his right eye walked into a doctors office and became the first person to receive reprogrammed stem cells from a donor to treat his age-related macular degeneration.

The patient a Japanese man in his 60s is not alone, as four other patients have been approved for the procedure by Japan's health ministry. The first medical case was reported on March 28 by Nature.

In a one-hour operation by surgeon Yasuo Kurimoto, the patient received skin cells from a human donor at Kobe City Medical Center General Hospital. The donors skin cells were reverse engineered into induced pluripotent stem (iPS) cells. These cells are often seen as a game-changer in the world of regenerative medicine as they have the ability to become almost any type of cell in the body.

In this case, the iPS cells were turned into retinal cells, which were then implanted into the retina of the patient, who has age-related macular degeneration. It is hoped the procedure will stop the progression of the disease, which can lead to blindness. The transplantis not being touted as a cure for the condition, merely a prevention methodfrom further damage.

During the procedure, the surgical team injected 50 microliters of liquid containing 250,000 retinal cells into the patients eye, according to the Japan Times. The real test, however, will be the next phase of monitoring.

What sets this transplant apart is also what makes the recovery process precarious. Doctors will need to keep a careful watch on the patient, as iPS cells from a donor are not a genetic match and could cause an immune rejection.

At this point, you might remember a similar case in 2014 with a Japanese woman at the same hospital. She also received retinal cells derived from iPS cells, however hers were taken from her own skin, not a donor's.

"A key challenge in this case is to control rejection," said Riken researcher Masayo Takahashi to the Japan Times. "We need to carefully continue treatment."

In an update, the team said the Japanese woman was doing well and her vision had not declined. They decided to change track and use donor cells for this study because it holds a more viable future for such transplants.

It's hoped, if all goes well here, that researchers can create a bank of donor stem cells. Such a future would cut down on costs and reduce wait times, as cultivating ones own cells can take several months. However, there's stillmuch to be done.

After the procedure, Takahashi told a press conference that the surgery went well. They will continue to monitor the situation and provide further updates in the future.

Read more:
Man Receives Reprogrammed" Stem Cells From Donor In Medical First - IFLScience

In what's reported to be a world-first, last Tuesday, a Japanese man received a pioneering retinal cell transplant grown from donor stem cells instead of his own.

Doctors took skin cells from a donor bank and reprogrammed them into induced pluripotent stem (iPS) cells, which can be coaxed to grow into most cell types in the body.

For this procedure, the physicians grew the iPS cells into atype of retinal cell, and then injected them into the retina of the patient's right eye.

The test subject was a man in his 60s who has been living with age-related macular degeneration-a currently incurable eye disease that slowly leads to loss of vision.

If this news sounds somewhat familiar, it's because the same team of Japanese doctors successfully performed a similar transplant in 2014. But in that case, the iPS cells came from the patient's own skin, not from a donor.

The 2014 treatment involved culturing a patient's cells into a thin sheet of retinal pigment epithelium cells, which they transplanted directly under her retina.

One year later, their results showed that the patient's disease had not progressedas it would have without any treatment, and she continues to do well.

But a second case study after the 2014 success never went ahead - the researchers found genetic abnormalities in the iPS cells they had derived from an additional patient's skin. To avoid complications, the doctors fromRIKENand Kobe City Medical Centre General Hospital decided to halt the trial and refine their approach.

Now they are back with a potentially safer technique that uses cells from a donor bank. The patient who received the transplant last week is the first of five approved for a study by Japan's health ministry in February this year. It's important to note that so far this is a safety study - a precursor to a clinical trial.

As team leader Masayo Takahashi from RIKEN told a press conference, we will have to wait and see for several years until we know for sure whether last week's transplant was a complete success - which is the whole point of doing a safety study like this.

"A key challenge in this case is to control rejection. We need to carefully continue treatment," she said.

The patient will be closely observed for a year, and then receive check-ups for three more years. The main things for the team to look out for are rejection of the new retinal cells, and the development of potential abnormalities.

An editorial in Nature praises the team's cautious approach, emphasising that this work with iPS cells could pave a smoother path for other trials in the emerging field of stem cell medicine.

If donor cells turn out to be a viable option in iPS cell procedures, it would be huge for creating more affordable stem cell treatments that anyone can benefit from.

Instead of having to induce stem cells out of each individual patient's samples, doctors could go down the cheaper and quicker route of simply picking a suitable match from a donor bank.

Stem cell treatments such as this new procedure are an extremely promising avenue in medicine, but scientists are right to remain cautious and proceed slowly. Just last month a devastating case report broke the news that three women lost their eyesight by participating in a dodgy stem cell trial.

On the other hand, in 2015, an experimental stem cell treatment showed promise in multiple sclerosis (MS) patients, and just last year, stem cell injections were used to help stroke patients in recovery.

With all these exciting developments, we'll definitely be keeping an eye on further reports from the Japanese team.

Read more from the original source:
A Japanese man just got another person's stem cells transplanted in ... - ScienceAlert

KAMLOOPS My curiosity was sparked when I read that a stem cell centre was opening in Kamloops (Kamloops This Week, March 21, 2017).

So I went to the location of the centre at 470 Columbia St only to find a parking lot. Thinking that the address might be wrong, I searched the directory of the medical building next door and found that no stem cell centre was listed.

The Stem Cell Centers website lists Kamloops as the only one in Canada. Dr. Richard Brownlee is named as the surgeon with more information coming soon.

Stem cell therapy, says the website, can help with orthopedic or pain management, ophthalmological conditions, cardiac or pulmonary conditions, neurological conditions, and auto-immune diseases, among many other conditions and disease that results in damaged tissue.

One of the ophthalmological conditions they treat is macular degeneration. If your vision is fading due to macular degeneration, you know its time to seek help. Our non-invasive Stem Cell Therapy treatment might be the solution for you.

I wanted get Dr. Brownlees reaction to news that an unproven stem cell treatment had resulted in blindness according to the New England Journal of Medicine as reported in the Globe and Mail, March 20, 2017.

This week, the New England Journal of Medicine (NEJM) reported on three individuals who went blind after receiving an unproven stem cell treatment at a Florida clinic. The patients paid thousands of dollars for what they thought was a clinical trial on the use of stem cells to treat macular degeneration.

The writer of the Globe and Mail article, Timothy Caulfield, Research Chair of the in Health Law and Policy at the University of Alberta, doesnt name the Florida clinic.

The Stem Cell Centers website refers optimistically to treatment for macular degeneration at a Florida clinic, although apparently not theirs since no Florida clinic appears on their list. It tells of how Doug Oliver suffered from macular degeneration before stem cells were extracted from his hip bone and injected them into his eyes. Almost immediately, Olivers eyesight started to improve. I began weeping, he said.

Caulfield encourages caution. Health science gets a lot of attention in the popular press. People love hearing about breakthroughs, paradigm shifts and emerging cures. The problem is, these stories are almost always misleading. It can also help to legitimize the marketing of unproven therapies.

Reports from the Stem Cell Centers own website are cautionary as well. It quotes an abstract from a study done by the Southern California College of Optometry on how stem cells might ultimately be used to restore the entire visual pathway.

The promise of stem cell research is phenomenal. Scientific American (Jan., 2017) reports that brains can be grown in a lab dish from stem cells taken from skin. These samples can be used to research brain disorders ranging from schizophrenia to Alzheimer's disease, and to explore why only some babies develop brain-shrinking microcephaly after exposure to the Zika virus.

However, Dr. George Daley, dean of Harvard Medical School, concludes that there are only a handful of clinical applications available and they are for skin and blood-related ailments.

Practice, it seems, has not yet matched the promise of stem cell research.

Go here to see the original:
Stem cell centre coming to Kamloops? | CFJC Today - CFJC Today Kamloops

KAMLOOPS My curiosity was sparked when I read that a stem cell centre was opening in Kamloops (Kamloops This Week, March 21, 2017).

So I went to the location of the centre at 470 Columbia St only to find a parking lot. Thinking that the address might be wrong, I searched the directory of the medical building next door and found that no stem cell centre was listed.

The Stem Cell Centers website lists Kamloops as the only one in Canada. Dr. Richard Brownlee is named as the surgeon with more information coming soon.

Stem cell therapy, says the website, can help with orthopedic or pain management, ophthalmological conditions, cardiac or pulmonary conditions, neurological conditions, and auto-immune diseases, among many other conditions and disease that results in damaged tissue.

One of the ophthalmological conditions they treat is macular degeneration. If your vision is fading due to macular degeneration, you know its time to seek help. Our non-invasive Stem Cell Therapy treatment might be the solution for you.

I wanted get Dr. Brownlees reaction to news that an unproven stem cell treatment had resulted in blindness according to the New England Journal of Medicine as reported in the Globe and Mail, March 20, 2017.

This week, the New England Journal of Medicine (NEJM) reported on three individuals who went blind after receiving an unproven stem cell treatment at a Florida clinic. The patients paid thousands of dollars for what they thought was a clinical trial on the use of stem cells to treat macular degeneration.

The writer of the Globe and Mail article, Timothy Caulfield, Research Chair of the in Health Law and Policy at the University of Alberta, doesnt name the Florida clinic.

The Stem Cell Centers website refers optimistically to treatment for macular degeneration at a Florida clinic, although apparently not theirs since no Florida clinic appears on their list. It tells of how Doug Oliver suffered from macular degeneration before stem cells were extracted from his hip bone and injected them into his eyes. Almost immediately, Olivers eyesight started to improve. I began weeping, he said.

Caulfield encourages caution. Health science gets a lot of attention in the popular press. People love hearing about breakthroughs, paradigm shifts and emerging cures. The problem is, these stories are almost always misleading. It can also help to legitimize the marketing of unproven therapies.

Reports from the Stem Cell Centers own website are cautionary as well. It quotes an abstract from a study done by the Southern California College of Optometry on how stem cells might ultimately be used to restore the entire visual pathway.

The promise of stem cell research is phenomenal. Scientific American (Jan., 2017) reports that brains can be grown in a lab dish from stem cells taken from skin. These samples can be used to research brain disorders ranging from schizophrenia to Alzheimer's disease, and to explore why only some babies develop brain-shrinking microcephaly after exposure to the Zika virus.

However, Dr. George Daley, dean of Harvard Medical School, concludes that there are only a handful of clinical applications available and they are for skin and blood-related ailments.

Practice, it seems, has not yet matched the promise of stem cell research.

See original here:
Stem cell centre coming to Kamloops? - CFJC Today Kamloops

On March 28, a Japanese man in his 60s became the first person to receive cells derived from induced pluripotent stem (iPS) cells that had been donated by another person.

The surgery is expected to set the path for more applications of iPS cell technology, which offers the versatility of embryonic stem cells without the latters ethical taint. Banks of iPS cells from diverse donors could make stem cell transplants more convenient to perform, while slashing costs.

iPS cells are created by removing mature cells from an individual (from their skin, for example), reprogramming these cells backto an embryonic state, and then coaxing them to become a cell type useful for treating a disease.

In the recent procedure, performed on a man from Hyogo prefecture, skin cells from an anonymous donor were reprogrammed and then turned into a type of retinal cell that was transplanted onto the retina of thepatient who suffers from age-related macular degeneration. Doctors hope the cells will stop progression of the disease, which can lead to blindness.

In a procedure performed in September 2014at the Kobe City Medical Center General Hospital, a Japanese woman received retinal cells derived from iPS cells. They were taken from her own skin, though, and then reprogrammed. Such cells prepared for a second patient were found to contain genetic abnormalities and never implanted.

The team decided to redesign the study based on new regulations, and no other participants were recruited to the clinical study. In February 2017, the team reported that the one patient had fared well. The introduced cells remained intactand vision had not declined as would usually be expected with macular degeneration.

In todays procedure performed at the same hospital and by the same surgeon Yasuo Kurimoto doctors used iPS cells that had been taken from a donors skin cells, reprogrammed and banked. Japans health ministry approved the study, which plansto enroll 5 patients, on 1 February.

Using a donor's iPS cells does not offer an exact genetic match, raising the prospect of immune rejection. But Shinya Yamanaka, the Nobel Prize-winning stem-cell scientist who pioneered iPS cells, has contended that banked cells should be a close enough match for most applications.

Yamanaka is establishing an iPS cell bank, which depends on matching donors to recipients via three genes that code for human leukocyte antigens (HLAs) proteins on the cell surface that are involved in triggering immune reactions. HisiPS Cell Stock for Regenerative Medicine currently has cell lines from just one donor. But by March 2018, they hope to create 5-10 HLA-characterized iPS cell lines, which should match 30%-50% of Japans population.

Use of these ready-made cells has advantages for offering stem cell transplants across an entire population, says Masayo Takahashi, an ophthalmologist at the RIKEN Center for Developmental Biology who devised the iPS cell protocol deployed in todays transplant. The cells are available immediately versus several months wait for a patients own cells and are much cheaper. Cells from patients, who tend to be elderly, might have also accumulated genetic defects that could increase the risk of the procedure.

At a press conference after the procedure, Takahashi said the surgery had gone well but that success could not be declaredwithout monitoring the fate of the introduced cells. She plans to make no further announcements about patient progress until all five procedures are finished. We are at the beginning, she says.

This article is reproduced with permission and wasfirst publishedon March 28, 2017.

Here is the original post:
Japanese Man Is First to Receive "Reprogrammed" Stem Cells from Another Person - Scientific American

Thomas Deerinck, NCMIR/SPL

In a medical first, a donor's iPS cells were transformed into retinal cells and transplanted into a patient.

On 28 March, a Japanese man in his 60s became the first person to receive cells derived from induced pluripotent stem (iPS) cells that had been donated by another person.

The surgery is expected to set the path for more applications of iPS cell technology, which offers the versatility of embryonic stem cells without the latters ethical taint. Banks of iPS cells from diverse donors could make stem cell transplants more convenient to perform, while slashing costs.

iPS cells are created by removing mature cells from an individual (from their skin, for example), reprogramming these cells backto an embryonic state, and then coaxing them to become a cell type useful for treating a disease.

In the recent procedure, performed on a man from Hyogo prefecture, skin cells from an anonymous donor were reprogrammed and then turned into a type of retinal cell that was transplanted onto the retina of thepatient who suffers from age-related macular degeneration. Doctors hope the cells will stop progression of the disease, which can lead to blindness.

In a procedure performed in September 2014at the Kobe City Medical Center General Hospital, a Japanese woman received retinal cells derived from iPS cells. They were taken from her own skin, though, and then reprogrammed. Such cells prepared for a second patient were found to contain genetic abnormalities and never implanted.

The team decided to redesign the study based on new regulations, and no other participants were recruited to the clinical study. In February 2017, the team reported that the one patient had fared well. The introduced cells remained intactand vision had not declined as would usually be expected with macular degeneration.

In todays procedure performed at the same hospital and by the same surgeon Yasuo Kurimoto doctors used iPS cells that had been taken from a donors skin cells, reprogrammed and banked. Japans health ministry approved the study, which plansto enroll 5 patients, on 1 February.

Using a donor's iPS cells does not offer an exact genetic match, raising the prospect of immune rejection. But Shinya Yamanaka, the Nobel Prize-winning stem-cell scientist who pioneered iPS cells, has contended that banked cells should be a close enough match for most applications.

Yamanaka is establishing an iPS cell bank, which depends on matching donors to recipients via three genes that code for human leukocyte antigens (HLAs) proteins on the cell surface that are involved in triggering immune reactions. HisiPS Cell Stock for Regenerative Medicine currently has cell lines from just one donor. But by March 2018, they hope to create 5-10 HLA-characterized iPS cell lines, which should match 30%-50% of Japans population.

Use of these ready-made cells has advantages for offering stem cell transplants across an entire population, says Masayo Takahashi, an ophthalmologist at the RIKEN Center for Developmental Biology who devised the iPS cell protocol deployed in todays transplant. The cells are available immediately versus several months wait for a patients own cells and are much cheaper. Cells from patients, who tend to be elderly, might have also accumulated genetic defects that could increase the risk of the procedure.

At a press conference after the procedure, Takahashi said the surgery had gone well but that success could not be declaredwithout monitoring the fate of the introduced cells. She plans to make no further announcements about patient progress until all five procedures are finished. We are at the beginning, she says.

Read more from the original source:
Japanese man is first to receive 'reprogrammed' stem cells from ... - Nature.com

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Skin stem cells gain traction for skin repair and regeneration ... FinancialsTrend Although a tremendous progress has been made, large full-thickness skin defects are still associated with mortality due to a low availability of donor skin areas. and more »

See more here:
Skin stem cells gain traction for skin repair and regeneration ... - FinancialsTrend