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Category Archives: Stem Cells

UCI researchers use stem cells as cancer-seeking missiles – 89.3 KPCC

Posted: August 2, 2017 at 11:44 am

A close-up of cell mutations that cause cancer. Steve Gschmeissner/Science Source

Chemotherapy is brutal a medicinal atomic bomb that destroys large swaths of cells, both cancerous and normal. And as a result, patients are often left physically devastated.

In a new study published in Science Translational Medicine, scientists at UC Irvine say they've come up with a way to use stem cells to help ameliorate those side effects. Think of it as a surgical strike with cancer-seeking missiles.

Professor Weian Zhao and his colleagues from UC Irvine modified stem cells so that they'd be attracted to enzymes released by breast cancer tumors. So, when injected into the body, the stem cells seek out the cells and bond with them.

The enzymes the scientists identified cause tissue to clump up into bundles of collagen and protein to create stiff tumors. The tumors become lumps that a patient can sometimes feel, and they act as a protective home for the cancerous cells.

The stem cells release an enzyme of their own, in turn, activating a type of chemotherapy that's been injected into the patient, which is inert until in comes in contact with the enzyme. The idea being that the chemotherapy only causes toxicity to a localized area, instead of destroying everything in its path.

"We can use a stem cells to specifically localize and produce the drugs only at the tumor site, so that we can spare the healthy tissue," said Zhao. "So, we can make the treatment more effective and less toxic to the patient."

"I think this is pretty unique in a way that it can target specific metastatic tissues with reduced toxicity overall," said Min Yu, assistant professor at the department of Stem Cell Biology and Regenerative Medicine at USC. "So, in that sense, I think it's very novel and very unique approach."

Yu, who was not involved in the research, complimented the UCI team's methods and results, especially how effective the treatment was on the particular cancer cell that they focused on. However, she said, from patient to patient and cancer to cancer, there are a myriad of different cells responsible, making treatment notoriously difficult to generalize. The therapy isn't a sure thing.

Zhao acknowledged that his team has a while to go before it can prove that the treatment is effective in people. So far, it's only been tested in mice. As a result, FDA approval and human trials could be years away.

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Stem cell brain implants could ‘slow ageing and extend life’, study shows – The Guardian

Posted: August 2, 2017 at 11:44 am

Scientists have slowed down the ageing process by implanting stem cells into the brains of animals, raising hopes for new strategies to combat age-related diseases and extend the human lifespan.

Implants of stem cells that make fresh neurons in the brain were found to put the brakes on ageing in older mice, keeping them more physically and mentally fit for months, and extending their lives by 10-15% compared to untreated animals.

The work, described as a tour de force and a breakthrough by one leading expert, suggests that ageing across the body is controlled by stem cells that are found in the hypothalamus region of the brain in youth, but which steadily die off until they are almost completely absent in middle age.

Researchers at Albert Einstein College of Medicine in New York hope to launch clinical trials of the procedure soon, but must first produce supplies of human neural stem cells in the lab which can be implanted into volunteers.

Of course humans are more complex, said Dongsheng Cai, who led the research. However, if the mechanism is fundamental, you might expect to see effects when an intervention is based on it.

Previous experiments had already hinted that the hypothalamus, an almond-sized part of the brain in humans, played some role in the ageing process, but what it was remained unclear. The latest investigation from the US team pinpoints which cells are important and how they might work.

In the first of a series of experiments in mice, Cai showed that neural stem cells, which are found in a handful of brain regions at birth, disappear from the hypothalamus over time. The stem cells are known to form fresh brain cells in youth, but the process slows down dramatically in adults. Though small, the hypothalamus forms a crucial connection between the bodys nervous and hormonal systems.

To test whether the decline in stem cells was causing ageing, and not itself a result of old age, the researchers injected mice with a toxin that wiped out 70% of their neural stem cells. The effect was striking. Over the next few months the mice aged more rapidly than usual, and performed much worse than control animals on a battery of tests of endurance, coordination, social behaviour and ability to recognise objects. Behaviourally mice aged faster when these cells were removed during early ageing, Cai told the Guardian. The animals died months earlier than healthy control animals.

Next, the scientists looked at what happened when aged mice received injections of fresh neural stem cells. This time the mice lived longer than controls, typically several months more, an increase of about 15%. If a similar extension was achieved in humans, a person with a life expectancy of 80 years could live to 92.

Having proved that it was neural stem cells that were important for ageing, the scientists ran further tests to work out what the cells were doing. They found that molecules called microRNAs, or miRNAs, that are released from neural stem cells were responsible for most of the ageing effects. When the molecules are produced in the hypothalamus, they flow into the clear fluid in the brain and spinal cord and affect how genes operate.

The mechanism is partially due to these cells secreting certain miRNAs which help maintain youth, and the loss of these leads to ageing said Cai, whose study is published in Nature. The next step is to create human neural stem cells in the lab for testing.

It is a tour de force, said David Sinclair at Harvard Medical School. Its a breakthrough. The brain controls how long we live. I can see a day when we are implanted with stem cells or treated with stem cell RNAs that improve our health and extend our lives. I would love to know which brain stem cell secretions extend a mouses lifespan and if human stem cells make them too.

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Stem cell brain implants could 'slow ageing and extend life', study shows - The Guardian

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Drive for stem cell donors in Langley – Surrey Now-Leader

Posted: August 2, 2017 at 11:44 am

Marie Grim of Langley is looking for 100 people between the ages of 17 and 35 who are willing to take a few moments to have their cheeks swabbed.

Theres no pain, no fuss, she said.

And you could save someones life, anywhere in the world.

You could match with someone in Japan.

The campaign to sign up more potential stem cell donors, people who are willing to allow DNA samples to taken using cotton swabs, was inspired by the experience of her sister-in-law.

Cloverdale resident Tania Grim, a mother of four was diagnosed with leukemia in January.

She had to wait several months before a compatible donor was found whose stem cells will be used to replace bone marrow and abnormal white blood cells eradicated by a combination of chemotherapy and radiation.

We have been on quite the journey, Marie said.

I have sat with her at appointments and heard others get news of their donor while she had not.

Now that Tania has her donor, Marie would like to improve the odds for other families.

She already has a location and tentative date to collect the swabs September 8 at Immanuel Christian Reformed Church in Langley if she can round up enough donors.

Tania, who is preparing for her stem cell procedure in September, urged prospective stem cell contributors to sign up.

I am so grateful that the word is being spread about the huge need for donors, Tania said.

It is a very simple thing to do that can save a life.

If you are the right age to be a donor, you can contact Marie at 604-530-1326 or by email at mariegrim@hotmail.com.

Interested donors can also contact Canadian Blood Services directly at https://blood.ca/en.

More than 390,000 Canadians have joined the OneMatch Stem Cell and Marrow Network registry maintained by Canadian Blood Services, volunteering to be stem cell donors for any patient in need of a transplant, anywhere in the world.

Right now, the agency says about 70 per cent of eligible donors on the registry are Caucasian, which means the odds of finding match for other ethnicities, such as Canadians with indigenous, Asian or African heritage, are not good.

The Canadian registry connects to an international network established by the World Marrow Donor Association (WMDA) that has access to over 28 million donors in over 53 countries.

Not everyone who registers will be matched to a patient and asked to donate, but each registrant provides hope for those waiting, a message posted to the agency website states.

A person could be a match within a few months of registering, a year later or even seven years later.

If a volunteer donor is found to be a match, they face a relatively minor surgical procedure and can expect to make a quick recovery.

The agency says over 80 diseases and disorders can be treated with a stem cell transplant.

There are hundreds of patients in Canada waiting for a match, but only half of them find a match.

Patients are more likely to find a matching donor from within their own ethnic group.

The odds of family members matching is extremely slight, the agency said, which is why it does not support donor drives targeting relatives.

RELATED STORY: Surrey teen rallies stem cell donors to help with desperate need for South Asians

dan.ferguson@langleytimes.com

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New NUH study to test stem cells as treatment for liver disease … – TODAYonline

Posted: July 12, 2017 at 1:47 pm

SINGAPORE The use of stem cell treatment to repair liver cirrhosis, or hardening of the liver, will be tested in a clinical trial here involving 46 patients and costing S$2.6 million.

The four-year study, which was launched yesterday, came amid a growing waiting list in Singapore for a liver transplant, which is currently the only cure for patients with end-stage liver cirrhosis.

Conducted by a multi-centre team from several restructured hospitals here, the study is led by the National University Hospital (NUH).

Liver failure is one of the top 20 causes of death in Singapore, but many patients are not suitable for a transplant due to factors such as age and surgical fitness.

Out of every five patients doctors see with end-stage liver disease, only one qualifies for a liver transplant, said Dr Dan Yock Young, principal investigator of the clinical trial and senior consultant at NUHs division of gastroenterology and hepatology.

(A liver transplant) is curative, but it is a complex procedure, and many patients are not suitable for it. For these patients, treatment is limited, but morbidity and mortality rates are high as high as 50 per cent in one year and this is probably worse than many (of the) other terminal illnesses we talk about today, he said.

Animal studies conducted over the last five years have shown that stem cells can reconstruct the micro-environment of a normal liver.

Like how branches are of critical importance in supporting the leaves and fruits of a tree, the endothelial (stem) cells contribute to supporting a nutritious environment for the hepatocyte (liver) cells, Dr Dan explained.

While similar stem-cell studies have been conducted in other centres in Asia, there has been no definitive evidence of the benefits of the treatment for liver patients.

The study will recruit 46 patients aged between 40 and 70 years old, and who are at the terminal stages of chronic liver disease, over three years. It is funded by the National Medical Research Council.

During the clinical trial, patients will be divided into a therapeutic group and a control group.

All patients will receive an injection to stimulate their bone marrow cells as part of the supportive treatment for their liver cirrhosis. However, only patients in the study group will have the stem cells from the bone marrow extracted and deposited directly into their liver for more targeted repair.

Using ones own stem cells will avoid the problem of cell rejection.

The liver tissue will be examined three months later, and an investigation to compare pre- and post-transplant results will be conducted after a year.

Since invasive surgery is not required for stem-cell therapy, the fatality risk is significantly lowered for the patient. However, other risks such as severe bleeding and infections still remain, given the patients weakened condition.

NUH also noted that the stem-cell therapy does not replace liver transplants, and the latter remains the best available treatment for liver cirrhosis.

It is very painful to turn patients away when we cannot offer them a liver transplant, said Dr Dan, adding that this stem cell therapy will serve as an alternative option.

We hope that this is a stepping stone to trials for stem cell candidates, he added.

MORE WAITING FOR A LIVER

The number of people on the waiting list for a liver transplant has been growing in recent years. In June last year, it was reported that there were 54 people on the list, more than double the 24 patients in 2011.

Chronic Hepatitis B remains the primary cause of non-alcoholic fatty liver disease, which refers to a range of liver conditions affecting people who drink little to no alcohol. However, obesity has become a contributing factor to the illness as well.

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UW-Madison scientists grow functional artery cells from stem cells – Madison.com

Posted: July 12, 2017 at 1:47 pm

In a step toward one of stem cell sciences chief goals, UW-Madison researchers have grown functional human artery cells that helped lab mice survive heart attacks.

The development, from the lab of stem cell pioneer James Thomson, could help scientists create arteries to use in bypass surgeries for cardiovascular disease, the nations top killer. Several challenges remain, however, and studies in people are years away.

This work provides valuable proof that we can eventually get a reliable source for functional arterial endothelial cells and make arteries that perform and behave like the real thing, Thomson said in a statement.

The research, reported Monday in the journal Proceedings of the National Academy of Sciences, is part of a federally funded effort at UW-Madison to create artery banks for cardiovascular surgery from universally compatible donors.

In a related project, other UW-Madison researchers are testing three-dimensional heart patches of heart muscle cells, grown from stem cells, in pigs. The goal is to replace diseased or damaged heart tissue in humans.

Since Thomson became the first scientist to successfully grow human embryonic stem cells in a lab in 1998, researchers around the world have been coaxing the universal cells into various cell types heart, pancreas, kidney, brain to develop therapies and better understand diseases.

Today, many researchers use cells reprogrammed to their embryonic state from mature cells known as induced pluri- potent stem, or iPS, cells as the raw material. Thomson helped discover iPS cells in 2007.

Many labs can convert embryonic stem cells or iPS cells into specific cell types, but developing specialized cell lines that are pure, functional and robust has been a challenge.

Thomson and his team set out to find a recipe for growing artery cells that would really function like arteries.

The researchers used two new techniques: single-cell RNA sequencing to identify genes highly expressed in cells that initiate artery development, and CRISPR-Cas9 gene editing to evaluate the function of the genes.

They found that five small molecules and growth factors are needed to encourage iPS cells to become functional artery cells. To their surprise, they discovered that insulin, a common growth factor that had been used before in trying to grow artery cells, actually inhibits such growth.

They used their recipe to make artery cells, and tested the cells in mice that had their left coronary arteries tied off to mimic heart attacks. Four weeks later, 83 percent of mice treated with the cells were alive, compared to 33 percent of mice that didnt get the cells.

We can use those cells to further create tissue-engineered arteries for bypass surgeries, said Jue Zhang, a scientist in Thomsons lab at the Morgridge Institute for Research and lead author of the study.

Developing off-the-shelf bypasses for surgery is the goal of an $8 million, seven-year grant UW-Madison received last year from the National Heart, Lung and Blood Institute to create universal artery banks.

The blood vessels of many cardiovascular disease patients arent suitable for use as bypasses, doctors say, and growing bypasses from individual patients stem cells would be timely and expensive. The hope is to use iPS cells from a rare population of genetically compatible donors to grow arteries anyone could use.

UW-Madison scientists, including engineers Tom Turng and Naomi Chesler and pathologist Igor Slukvin at the Wisconsin National Primate Research Center, plan to grow artery cells on scaffolds and test them in monkeys. If successful, the cells would be produced for human studies at the Waisman Biomanufacturing facility on campus.

The heart patches involve another $8.6 million, seven-year National Institutes of Health grant, shared with the University of Alabama-Birmingham and Duke University.

The patches involve three types of heart cells, derived from iPS cells, said Dr. Tim Kamp, a UW-Madison cardiologist and co-director of the universitys Stem Cell and Regenerative Medicine Center.

In studies in pigs, getting the patches to connect and survive when transplanted to pig hearts after heart attacks remains a challenge, Kamp said. Immune tolerance of the human grafts in pigs is another concern, he said.

But if such hurdles can be overcome, tests in humans could follow.

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Stem Cells Guided by Electric Fields May Offer New Therapies for … – Genetic Engineering & Biotechnology News (press release)

Posted: July 12, 2017 at 1:47 pm

Scientists at the University of California, Davis School of Medicine's Institute for Regenerative Cures report that electric fields can be used to guide neural stem cells transplanted into the brain toward a specific location. Their study (Electrical Guidance of Human Stem Cells in the Rat Brain), which appears in Stem Cell Reports, opens the door for potentially guiding stem cells to repair brain damage.

we report a strategy that mobilizes and guides migration of stem cells in the brain invivo. We developed a safe stimulation paradigm to deliver directional currents in the brain, write the investigators. Tracking cells expressing GFP [green fluorescent protein] demonstrated electrical mobilization and guidance of migration of human neural stem cells, even against co-existing intrinsic cues in the rostral migration stream.

Min Zhao, M.D., Ph.D., carries out research on how electric fields can guide wound healing. Damaged tissues generate weak electric fields, and Zhao's research has shown how these electric fields can attract cells into wounds to heal them.

"One unmet need in regenerative medicine is how to effectively and safely mobilize and guide stem cells to migrate to lesion sites for repair," Dr. Zhao said. "Inefficient migration of those cells to lesions is a significant roadblock to developing effective clinical applications."

Natural neural stem cells are found deep in the brain, in the subventricular zone and hippocampus. To repair damage to the cortex, they have to migrate some distance, especially in the large human brain. Transplanted stem cells might also have to migrate some way to find an area of damage.

Dr. Zhao, and his colleague, Junfeng Feng, M.D., a neurosurgeon at Ren Ji Hospital, Shanghai Jiao Tong University, and Shanghai Institute of Head Trauma, developed a model of stem cell transplants in rats. They placed human neural stem cells in the rostral migration stream, which is a pathway in the rat brain that carries cells toward the olfactory bulb. Cells move along this pathway, partly carried by the flow of cerebrospinal fluid and partly guided by chemical signals.

By applying an electric field within the rat's brain, the scientists found that they could get the transplanted stem cells to swim upstream against the fluid flow and natural cues and head for other locations within the brain.

The transplanted stem cells were still in their new locations weeks or months after treatment.

"Electrical mobilization and guidance of stem cells in the brain therefore provides a potential approach to facilitate stem cell therapies for brain diseases, stroke, and injuries," noted Dr. Zhao.

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Experimental stem cells could help dogs suffering from arthritis – FOX 46 Charlotte

Posted: July 12, 2017 at 1:47 pm

FOX 46 WJZY - By age 10 up to 80 percent of dogs will develop arthritis. It can make it difficult for them to walk, stand or even move, but the experimental use of stem cells from young dogs could helprejuvenatejoints in older animals.

Brian Cirillo is concerned about his four-year-old dog Cosby's health.

"He's always the last one to kind of get moving and if he's laying down for a long time he takes a long time to stand up," Cirillo said.

Initially acting as fosters, Brian adopted Cosby and his two siblings when they were just four weeks old, bringing the total number of rescue dogs at their home up to six.

"He's the only one that's so scared of everything and I'm starting to wonder now if it's because he's in pain, you know, and he doesn't want to have to get out of a situation or something," saidCirillo.

To diagnose Cosby's problem he's getting a physical exam, X-rays, and blood tests, but there's a possibility Cosby could qualify to get something else-- an injection of experimental stem cells into his joint.

"We're looking at taking the miraculous healing capabilities of the body, concentrating it, and then bringing it back to the body and we're not seeing a lot of side effects,"Cirillosaid.

Veterinarian Dr. Michael Amsberry owns the St. Francis Pet Care Center, one of several sites across the county taking part in a clinical trial testing whether specially grown stem cells made by animal cell therapies in San Diego will help arthritis symptoms in dogs.

"Specifically this study is knees, hips, elbows and shoulders but the most common in this study is hips," Dr. Amsberry said.

The cells are grown from umbilical cord blood.

"So what they've done is harvested little umbilical cords from c-sections from dogs and they isolate these cells. They grow them up, they can culture them up to hundreds of millions of cells so from one sample they can treat thousands of dogs," said Dr. Amsberry.

The treatment is free and Dr. Amsberry says so far he's injected eight dogs.

"Any downside, we just haven't seen any downsides period," he said.

It's an experimental option Brian hopes will work for Cosby.

"If he gets older and keeps getting worse that's never good so if we can try to get ahead of the problem with stem cells and actually cure the problem and he doesnt have to be on a lot of chemicals and medicines his whole life, that would be great," saidCirillo.

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Colon Cancer-Driven Stem Cells Linked to High-Fat Diet – Genetic Engineering & Biotechnology News

Posted: July 7, 2017 at 3:42 pm

Scientists in the U.S. have identified a molecular pathway that appears to play a key role in the link between a high-fat diet (HFD) and the development of colorectal cancer. The research, led by the Cleveland Clinics Sheerlarani Karunanithi, and Matthew Kalady, suggests that it may one day be possible to develop drugs that reduce tumor growth associated with obesity and a diet that is high in fat.

Their research is published today, in Stem Cell Reports, in a paper titled, RBP4-STRA6 Pathway Drives Cancer Stem Cell Maintenance and MediatesHigh-Fat Diet-Induced Colon Carcinogenesis.

The Cleveland Clinic teams review of published research indicated that high expression levels of two vitamin A signalling proteinsserum retinol binding protein (RPB4), stimulated by retinoic acid 6 (STRA6)in colorectal cancer tumors is associated with poor prognosis, increased tumor metastasis and recurrence, and resistance to cancer therapy. The RBP4-STRA6 pathway triggers the JAK2-STAT3 signaling cascade.

The researchers engineered STRA6- or RBP4-knockdown cancer cells to demonstrate that the RBP4-STRA6 pathway is important for promoting cancer cell proliferation and survival and for maintaining the expression of core stem cell transcription factors. They also found that the RBP4-STRA6 pathway plays a key role in maintaining colon cancer stem cells (CSCs), both in cell lines and in patient-derived xenografts.

The teams previous work had shown that knocking down STRA6 in a xenograft cancer model decreased tumor growth. In a new round of studies, they injected RBP4-knockdown cancer cells into experimental mice, and found that RBP4 deficiency resulted in the development of fewer tumors, and slower tumor growth and progression.

With evidence building for the role of RBPA4-STRA6 pathway in colorectal cancer development and progression, the team turned to look at diet-related cancer. A prior study had already suggested that HFDinduced obesity leads to increased intestinal stem cells and may impact colorectal cancer risk. This finding, combined with independent research establishing a role for the RBPA4-STRA6 pathway in diet-induced metabolic syndrome, prompted the Cleveland Clinic team to look at the relationship between HFD, cancer development, and the RBPA4-STRA6 pathway.

They injected either STRA6-deficient colorectal cancer cells or unmodified cancer cells into obesity-resistant mice fed either a normal diet or an HFD. HFD mice injected with unmodified cancer cells exhibited significantly increased tumor growth compared with mice fed a normal diet. In contrast, there was no relative increase in tumor growth among HFD animals receiving the STRA6-deficient tumor cells.

Our data clearly indicate that RBP4-STRA6 pathway is necessary for the optimal expression of stem cell markers such as NANOG, SOX2, and LGR5, and thereby for maintaining the colon CSC pool, the authors conclude in their published paper. "We have known the influence of diet on colorectal cancer, commented Matthew Kalady, M.D., colorectal surgeon, and co-director of the Cleveland Clinic Comprehensive Colorectal Cancer Program. However, these new findings are the first to show the connection between high-fat intake and colon cancer via a specific molecular pathway. We can now build upon this knowledge to develop new treatments aimed at blocking this pathway and reducing the negative impact of a high-fat diet on colon cancer risk."

The interesting finding here is that the high fat diet-induced effects appear to also involve the stem cell program, which is interesting for tumor growth and has implications on therapies, as tumor stem cells are also therapeutically resistant, the authors told GEN. In terms of treatments, what we might envision is targeting a new component of the signaling axis we identified to reduce cancer growth. The pathways we have identified are known to control many aspects of cell behavior, but the input to these signaling programs is new and may represent a possible target. In terms of next steps, one would be to see whether this can be applied to other obesity-driven tumors. Can lessons from colon cancer be leveraged to other tumor types? We also are interested in inhibiting this new signaling axis as well as trying to understand more about this signaling program, as we may be able to identify signaling nodes that can be efficiently targeted.

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Consider risks of unapproved stem cell treatment – Kamloops This Week

Posted: July 7, 2017 at 3:42 pm

Editor:

Re: (Stem cells to stem the wait? June 27):

There is a reason Health Canada has not approved certain stem-cell treatments: they have not been shown to work yet and even ones own minimally manipulated cells can be considered risky.

There is a great deal of research happening in Canada and globally and clinical trials are underway to test and improve the quality, safety and effectiveness of stem-cell therapies because scientists and industry believe they hold great promise.

As the KTW article noted, the federal government committed $20 million to the Centre for Commercialization of Regenerative Medicine in 2016, but it wasnt to establish a stem-cell therapy development facility in Toronto. Rather, the funding is to find better ways of manufacturing therapeutic cells, including stem cells, in the billions that are required for clinical use.

Not all stem cells are the same and it is crucial to ensure the purity of stem cells before they are injected into people.

There is a lot of support for stem-cell research and manufacturing in Canada and, if the public is patient, treatments will come.

For now, people seeking unapproved treatments should consider the risks (because they exist in the short-term and long-term) and be prepared to throw away their money if the treatment doesnt work.

Stacey Johnsondirector of communications and marketingCentre for Commercialization of Regenerative MedicineToronto

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Capricor yo-yos as J&J dumps stem cell partnership – FierceBiotech

Posted: July 7, 2017 at 3:42 pm

After three and a half years, Johnson & Johnson is ducking out of a partnership with Capricor Therapeutics focusedon the use of stem cells to treat cardiovascular disease.

The decision by J&J's Janssen unit not to pursue a license comes as little surprise. The therapy at the center of their end-2013 dealCAP-1002 (off-the-shelf cardiosphere-derived cells)has already been marked up as missing the target in a phase 1/2 study involving patients who had suffered a heart attack, although the data remains under wraps.

Added to that, in April, interim results from a trial in Duchenne muscular dystrophy revealed promising activity, and prompted Capricor to upgrade the importance of the new indicationwhich lies outside the scope of its license with J&J.

The California biotech has already said it plans to start a second trial in DMD in the latter half of the year. But while the back-up indication is a comfort to investors, there is no question that the company will miss the financial backing from J&J, which included $12.5 million upfront and up to $325 million in milestone payments, as well as the kudos of a big pharma partner in a sector that has failed so far to live up to early promise.

Weak or scarred heart muscle is a major cause of heart failure, so using stem cells to repair scar tissue appears to be a logical way of improving outcomes. Attempts to show a benefit have met with marginal success, however. Last year for example, Celyad's 271-patient trial of its C-Cure stem cell therapy revealed no improvement compared to a sham procedure.

Capricor's CEO Linda Marbn, Ph.D. accentuated the positive of claiming full rights to CAP-1002, including not only the DMD data but also work with Janssen on developing a commercial-scale manufacturing process for the cell therapy, to which it now has a "fully paid-up nonexclusive license."

She also said it settled "uncertainty concerning the scope of the license for CAP-1002" and frees the company to seek partners elsewhere.

"We discussed potential product registration strategies for this indication at our recent meeting with the U.S. FDA, and we look forward to providing an update on our clinical development plans in DMD very shortly," continued Marbn.

Capricor also announced in an SEC filing that it is filingfor resale of up to 1.2 million shares of its common stock but would not be receiving any proceeds from the transaction. It ended the first quarter with $2.75 million in cash.

Shares in the biotech fell after the announcement but had rebounded at the time of writing, though they are still in penny stock territory at $0.80.

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