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Stem Cell Agency Helps Set the Stage for Revolutionary Medicine

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Newswise Scientists from UCLAs Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research have received new awards from the California Institute of Regenerative Medicine (CIRM), the state stem cell research agency, that will forward revolutionary stem cell science in medicine.

Recipients included Dr. Lili Yang, assistant professor of microbiology, immunology and molecular genetics who received $614,400 for her project to develop a novel system for studying how stem cells become rare immune cells; Dr. Denis Evseenko, assistant professor of orthopedic surgery, who received $1,146,468 for his project to identify the elements of the biological niche in which stem cells grow most efficiently into articular cartilage cells; Dr. Thomas Otis, professor and chair of neurobiology and Dr. Ben Novitch, assistant professor of neurobiology, who received $1,148,758 for their project using new light-based optigenetic techniques to study the communication between nerve and muscle cells in spinal muscular atrophy, an inherited degenerative neuromuscular disease in children; and Dr. Samantha Butler, assistant professor of neurobiology, received $598,367 for her project on discovering which molecular elements drive stem cells to become the neurons, or nerve cells, in charge of our sense of touch.

These basic biology grants form the foundation of the revolutionary advances we are seeing in stem cell science, said Dr. Owen Witte, professor and director of the Broad Stem Cell Research Center, and every cellular therapy that reaches patients must begin in the laboratory with ideas and experiments that will lead us to revolutionize medicine and ultimately improve human life. That makes these awards invaluable to our research effort.

The awards were part of CIRMs Basic Biology V grant program, carrying on the initiative to foster cutting-edge research on significant unresolved issues in human stem cell biology. The emphasis of this research is on unravelling the secrets of key mechanisms that determine how stem cells, which can become any cell in the body, differentiate, or decide which cell they become. By learning how these mechanisms work, scientists can then create therapies that drive the stem cells to regenerate or replace damaged or diseased tissue.

Using A New Method to Track Special Immune Cells All the different cells that make up the blood come from hematopoietic or blood stem cells. These include special white blood cells called T cells, which serve as the foot soldiers of the immune system, attacking bacteria, viruses and other invaders that cause diseases.

Among the T cells is a smaller group of cells called invariant natural killer T (iNKT) cells, which have a remarkable capacity to mount immediate and powerful responses to disease when activated, a small special forces unit among the foot soldiers, and are believed to be important to immune system regulation of infections, allergies, cancer and autoimmune diseases such as Type I diabetes and multiple sclerosis.

The iNKT cells develop in small numbers in the blood, usually less than 1 percent of all the blood cells, and can differ greatly in numbers between individuals. Very little is known about how the blood stem cells produce iNKT cells.

Dr. Lili Yangs project will develop a novel model system to genetically program human blood stem cells to become iNKT cells. Dr. Yang and her colleagues will track the differentiation of human blood stem cells into iNKT cells providing a pathway to answer many critical questions about iNKT cell development.

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Stem Cell Agency Helps Set the Stage for Revolutionary Medicine

Why I’m sure human stem cell trial will be safe

The new kind of stem cell announced yesterday may be the future of regenerative medicine, but Masayo Takahashi's pilot safety study using a type of stem cell to treat age-related blindness is at the cutting edge

Later this year, you will make history when you begin the first ever human trial of induced pluripotent stem cells. Why is this such a big deal? Stem cells have enormous medical potential because they can become any other type of cell. If we can use them to replace old or damaged cells, this could have huge implications for treating degenerative diseases.

Stem cells can be harvested from embryos, but this is ethically controversial. Despite this, there are several trials of these embryonic stem cells under way. Their use often requires drugs to stop the immune system from rejecting them, which can cause complications for elderly patients. Induced pluripotent stem (iPS) cells offer an alternative. These are made from a patient's own cells, removing the need for the immunosuppressant drugs. Plus there are no ethical issues.

How would treatment with iPS cells work? iPS cells are made by injecting several "reprogramming" genes into adult cells that have been removed from the body. This makes them rewind to an embryonic state. Then, we can make iPS cells differentiate into the cell type we need by injecting proteins that instruct embryonic stem cells to become liver, retina or any other type of cell. The idea is that these reprogrammed cells can then be inserted in the body to replace damaged cells. We are at least 20 years from any clinical treatments, but the potential is exciting.

Are there any potential pitfalls with iPS cell treatments? Yes, we have to be very careful because iPS cells multiply endlessly. This means that if any undifferentiated iPS cells were accidentally put into someone, they could cause tumours. That's why this study is so important. It is not a clinical trial, but a six-subject pilot study to confirm the safety of putting cells derived from iPS cells into humans.

Who are the participants in the study? The six people all have age-related macular degeneration in their eyes. This weakens the vision in the central field, eventually leaving people with only peripheral vision. In the type of degeneration we are working with, this is caused by the deterioration of the retinal pigment epithelium (RPE) the layer of cells that clears away extra-cellular debris that lands on the retina.

We aim to replace the damaged section of the RPE with cells created from skin taken from the patient's arm. The skin cells will be reprogrammed into iPS cells and then differentiated into RPE cells. It will take a year to grow enough RPE cells to introduce them to a damaged eye. Although I am excited to see if there is any improvement in sight, this study aims only to demonstrate the safety of RPE cells derived from IPS cells.

How confident are you that the pilot will be a success? Very confident. We have trialled this intervention on mice, rats and monkeys, and observed no tumours. I chose to work with RPE cells because of their characteristic brown pigment. This means we can avoid injecting tumour-causing iPS cells by selecting only the clumps of pure brown RPE cells. Of course, we do have to pick out around 50,000 RPE cells, so it can be a bit tough.

Another reason for optimism is that the retina is the safest place to try this out because we can watch the cells closely through the participant's dilated pupil.

What does the future hold for IPS cells? Right now it takes a lot of time, money and labour to reprogram cells. In our study, each intervention costs 20 million yen ($200,000) per eye and will take 10 people a year to complete. However, my research uses "auto-transplantation", in which the iPS cells come from the patient. The possibility of "allogeneic" treatment, in which iPS cells from one person could be used in many people, could reduce the cost tenfold. Shinya Yamanaka [who won a Nobel prize in 2012 with John Gurdon, for discovering iPS cells] plans to create an iPS cell bank to store a number of genetically average iPS cell cultures those that most easily integrate into people without immuno-rejection.

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Why I'm sure human stem cell trial will be safe

Failure to legislate ‘will hurt research’

Failure to legislate will hurt research

Tuesday, January 28, 2014

Even though stem cell manufacture has just been licensed in this country, the Governments ongoing failure to legislate in this area means pharmaceutical giants will still be wary of investing here, according to an expert in stem cell therapy.

CCMI General Manager Andrew Finnerty, CCMI Director Tim O'Brien, Minister Sean Sherlock and President of NUI Galway Dr. James Browne. Photograph by Aengus McMahon

Once the stem cells are harvested from the bone marrow of adult donors, they are grown in the Galway laboratory to generate sufficient quantities.

The first clinical trial using these stem cells is being funded by the Health Research Board and Science Foundation Ireland and will investigate the safety of using mesenchymal stem cells (MSCs) isolated from bone marrow for the treatment of critical limb ischemia, a complication associated with diabetes which can lead to limb amputation.

John ODea of the Irish Medical Devices Association (IMDA) said the centre was a key step.

I look forward to seeing its continued growth to assist in developing the skill sets and techniques that will be needed to embrace the new manufacturing opportunities that this exciting area will bring, he said.

The centre, one of a handful in Europe authorised for stem cell manufacture, has been developed by researchers at NUIGs regenerative medicine institute.

However, Dr Stephen Sullivan, chief scientific officer with the Irish Stem Cell Foundation warned all stem cell research operates at a pan-global level driven by big pharma and international equity firms and these players will only engage with researchers in countries where there is solid stem cell legislation in place. He welcomed the centre as a first step but said if Ireland is to compete at a top international standard, legislation remains necessary.

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Failure to legislate ‘will hurt research’

Q&A – Stem cells could offer treatment for a myriad of diseases

Q&A - Stem cells could offer treatment for a myriad of diseases

Tuesday, January 28, 2014

Q.What are stem cells?

Stem cells are different however as they are at an earlier stage in cell development and this means they can make more cells and transform into different cell types such as a skin stem cell can make all the different types of skin cells.

Q. And there are two types? A.Yes. There are two types of stem cells: embryonic stem cells and adult stem cells. Embryonic stem cells can generate all cells of the human body. Adult stem calls generate a more limited number of human cell types.

Q.Why are stem cells so important? A.For many years, adult stem cells have been used to treat rare blood and certain cancers.

However, adult stem cells cant generate all cell types. For example, scientists say there doesnt appear to be an adult stem cell that can make insulin- secreting cells of the pancreas. Embryonic stem cells can, however, as they can generate all cell types and the aim of scientists is to use these embryonic cells to generate healthy tissue to replace cells compromised by disease. This means that embryonic cells are more scientifically useful.

Q. And its also embryonic cells that are the more controversial, right? A.The use of embryonic stem cells is controversial here and in other countries as certain groups believe it is morally wrong to experiment on an embryo that could become a human. Embryonic stem cells are taken from embryos left over after assisted fertility treatments. According to the Irish Stem Cell Foundation, if they werent used for research into human disease, they would be discarded as medical waste. Embryos are not created purely for research purposes they say.

Q. Why are they so useful? A. Among the conditions which scientists believe may eventually be treated by stem cell therapy are Parkinsons disease, Alzheimers disease, heart disease, stroke, arthritis, diabetes, burns and spinal cord damage. Early trials are under way for treating forms of blindness. It is also hoped we can learn from embryonic stem cells how early body tissues develops and more about the pathway of diseases. This will enable us to make better and more effective drugs.

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Q&A - Stem cells could offer treatment for a myriad of diseases

Making Music Videos Helps Young Cancer Patients Cope With Treatment

January 28, 2014

Ranjini Raghunath for redOrbit.com Your Universe Online

Cancer treatment through chemotherapy, radiation or stem cell therapies can be physically and mentally exhausting for patients, especially younger ones. Many factors can help them feel positive about themselves and their treatment, including spiritual practices, supportive home environments and strong social connections with friends, family and physicians.

Now, a new study shows that making music videos and writing song lyrics may also help young cancer patients better cope with their treatment.

Researchers at the Indiana University School of Nursing, Indianapolis, carried out the music therapy intervention study, which was published online in the journal Cancer.

113 young patients aged 11-24 undergoing stem cell transplants for cancer were selected randomly for the study. Half of them were given audiobooks (the control group) and the other half were given three weeks to write down song lyrics, collect images and record music videos.

Patients in the second (test) group went through six training sessions each with a music therapist, who helped them identify and write about what was important to them, and guided them in creating the videos.

It really targeted them writing, having an opportunity to write about whats important to them, co-author of the study and music therapist, Sheri Robb, told Reuters. A lot of these kids as theyre going through treatment, they tend to not talk about these things.

The patients also had a chance to share the videos they created with family and friends. After about 100 days of treatment, patients in the test group reported that making those videos helped them better connect with their loved ones.

The intervention therapy helped the young patients feel stronger, more positive and helped improve their relationships with family and physicians, based on their responses to follow-up questionnaires, the researchers reported.

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Making Music Videos Helps Young Cancer Patients Cope With Treatment

Ireland’s first stem cell manufacturing centre approved at NUI Galway

Embryonic stem cells have been highly valued for their ability to turn into any type of cell in the body.

Stem cells can be manufactured for human use for the first time in Ireland, following Irish Medicines Board licensing of a new facility in Galway.

NUI Galways Centre for Cell Manufacturing Ireland aims to culture adult stem cells to tackle conditions such as arthritis, heart disease, diabetes and associated conditions.

The centre, which is one of less than half a dozen in Europe authorised for stem cell manufacture, has been developed by researchers at NUIGs regenerative medicine institute.

Stem cells serve as the bodys repair mechanism. They can be isolated from tissues such as bone marrow and fat, and cultured in laboratory settings.

More controversially, embryonic stem cells have been highly valued for their ability to turn into any type of cell in the body, but scientists can now use reprogrammed adult skin cells to create a stem cell that is very similar to embryonic versions.

The centre will be opened today by Minister of State for Research and Innovation Sen Sherlock, at a time when the Health Research Board and Science Foundation Ireland have approved funding there for clinical trials on using mesenchymal stem cells cells that can differentiate into a variety of types for treatment of critical limb ischemia, a condition associated with diabetes that can result in amputation.

The new centres director Prof Tim OBrien explained that the stem cells must be grown in the laboratory to generate sufficient quantities, following their isolation from the bone marrow of adult donors, and the facility will help Ireland to develop therapies for a broad range of clinical problems which do not have effective treatments today.

It will also allow us to translate discoveries from the basic stem cell research programme led by Prof Frank Barry at the Science Foundation Ireland-funded REMEDI to the clinic, and to be competitive for grant funding under the Horizon 2020 programme of the EU, he said.

Stem cell research in Ireland is in what scientists have described as a legislative lacuna, but this relates to use of embryonic stem cells and does not in any way inhibit the use of adult stem cells, Prof OBrien explained.

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Ireland’s first stem cell manufacturing centre approved at NUI Galway

Stem cell facility opens in Co Galway

Monday 27 January 2014 21.55

The first facility in Ireland licensed to manufacture human stem cells opened in NUI Galway this morning.

The development will mean some patients suffering from certain diseases will be able to access clinical trials of stem cell therapies.

Stem cells are human cells that can develop into many types of tissue.

As a result, scientists are increasingly studying their potential for regenerating diseased or damaged tissue in people suffering the effects of certain illnesses, such as heart attacks, stroke, arthritis or diabetes.

Before they can be used widely in humans such therapies must undergo extensive clinical trials.

The first Irish licence to manufacture stem cells for this purpose was granted to the Centre for Cell Manufacturing Ireland at NUIG.

The custom-built facility will take small samples of bone marrow from adult donors and culture them in a specially designed laboratory to make billions of stem cells.

The cells will then be injected back into the donor or other patients as part of clinical trials.

The first of those trials will investigate their use in the treatment of diabetes patients who are suffering reduced blood flow to lower limbs, a symptom that often results in amputations.

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Stem cell facility opens in Co Galway

Island’s first stem cell manufacturing centre approved at NUI Galway

Embryonic stem cells have been highly valued for their ability to turn into any type of cell in the body.

Stem cells can be manufactured for human use for the first time in Ireland, following Irish Medicines Board licensing of a new facility in Galway.

NUI Galways Centre for Cell Manufacturing Ireland aims to culture adult stem cells to tackle conditions such as arthritis, heart disease, diabetes and associated conditions.

The centre, which is one of less than half a dozen in Europe authorised for stem cell manufacture, has been developed by researchers at NUIGs regenerative medicine institute.

Stem cells serve as the bodys repair mechanism. They can be isolated from tissues such as bone marrow and fat, and cultured in laboratory settings.

More controversially, embryonic stem cells have been highly valued for their ability to turn into any type of cell in the body, but scientists can now use reprogrammed adult skin cells to create a stem cell that is very similar to embryonic versions.

The centre will be opened today by Minister of State for Research and Innovation Sen Sherlock, at a time when the Health Research Board and Science Foundation Ireland have approved funding there for clinical trials on using mesenchymal stem cells cells that can differentiate into a variety of types for treatment of critical limb ischemia, a condition associated with diabetes that can result in amputation.

The new centres director Prof Tim OBrien explained that the stem cells must be grown in the laboratory to generate sufficient quantities, following their isolation from the bone marrow of adult donors, and the facility will help Ireland to develop therapies for a broad range of clinical problems which do not have effective treatments today.

It will also allow us to translate discoveries from the basic stem cell research programme led by Prof Frank Barry at the Science Foundation Ireland-funded REMEDI to the clinic, and to be competitive for grant funding under the Horizon 2020 programme of the EU, he said.

Stem cell research in Ireland is in what scientists have described as a legislative lacuna, but this relates to use of embryonic stem cells and does not in any way inhibit the use of adult stem cells, Prof OBrien explained.

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Island’s first stem cell manufacturing centre approved at NUI Galway

Stem-cell therapy restores faith for arthritic pets

ELLSWORTH Visitors to the Bellaire pet crisis center With a Little help From My Friends get an official welcome from Moka.

The Labrador retriever was found behind a Bellaire restaurant in 2011 and now serves as the centers mascot.

Peforming her duties has been increasingly difficult for the dog, who suffers from severe arthritis in her hips. So recently the center turned to Ellsworth veterinarian Christian Randall of North Country Veterinary Services, the first in northern Michigan to offer in-clinic adipose stem cell therapy.

The procedure uses a pets own blood and tissue to produce plasma-rich platelets and stem cells that proliferate growth in damaged areas.

Dormant stem cells are separated from adipose -- fat tissue -- and activated with an LED technology that uses three different wave lengths of light. Then the cells are injected directly into the affected area or administered intravenously to help promote regeneration. The result is a decrease in pain and lameness and increased range of motion.

Its using the bodys own repair cells to repair damage, said Trey Smith, director of laboratory services for MediVet America, which developed the technology Randall uses.

The therapy is the first treatment to help heal and slow the progression of osteoarthritis and degenerative joint disease rather than just cope with the symptoms, said Randall, who saw the results while studying at Virginia Equine Imaging and now plans to use it on equine as well as canine and feline patients.

It concentrates, speeds up and amplifies the bodys own healing power, he said.

Stem cell therapy has been around for a while, but in-clinic availability of the technology is new. Only a handful of veterinarians in Ann Arbor and Grand Rapids offer the services, said Randall, who charges $1,800 to treat a dog or cat. Repeat injections are possible with banked plasma-rich platelets and stem cells.

Before the one-day procedure, veterinarians had to send blood and tissue to an outside lab for processing, a more costly three-day procedure that requires an animal's return visit to the vet for injection.

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Stem-cell therapy restores faith for arthritic pets