Researchers find new method to overcome major challenge of stem cell therapy

January 25, 2018

Researchers have discovered a new technique that overcomes one of the major challenges of stem cell therapy.

A drug, co-created by UBC researchers, might overcome one of the major challenges of stem cell therapy – their tendency to differentiate, becoming specific tissue cells too early and too quickly. If the drug works as well as it did in lab mice, it could help bring new stem cell treatments closer to reality.

UBC and Stanford University researchers were interested in using stem cells to help regenerate muscle tissue for the treatment of muscular dystrophy, a genetic disease in which muscles get damaged over time and weaken. Stem cells hold promise for such diseases because of their ability to differentiate or produce new cells that can form into specialized tissues in the human body. In theory, stem cells could replace damaged tissues with new tissue, unaffected by disease.

However, scientists have had a hard time developing successful treatments, especially for muscle, because once the stem cells are isolated in a lab dish, they stop simply being stem cells and begin to differentiate. In this case, the muscle stem cells stop dividing and become muscle fibers, which do not transplant well.

“Stem cells are like flour – they can be baked into any number of things like pies, cookies or bread – but once that happens, there is no going back to flour,” said Dr. Fabio Rossi, professor of medical genetics at UBC’s biomedical research centre and school of biomedical engineering, and senior author of the paper. “The problem was that all our stem cells were turning into ‘bread’ and we really needed them to stay as ‘flour’ so they could continue to replicate, creating enough cells so that we can transplant and regenerate the tissue effectively.”


Grow your own: the race to create body parts in the lab

5th December 2017

From replacement skin to entire new organs, regenerative medicine is finally leaving its early scandals – and the controversial ‘earmouse’ – behind. Could it one day provide a cure for birth defects, blindness, and diabetes?

Two years ago, Hassan’s father was faced with questions that he had no good answers for. “Why do I have this disease?” his seven-year-old son asked him. “Why do I have to live this life?”

Hassan was born with a rare genetic skin condition, called epidermolysis bullosa, that causes fragile, blistering skin. His first blister appeared when he was a week old, but soon after his family fled their native Syria and arrived as refugees in Germany, things got much worse. By June 2015, Hassan was admitted to a hospital, critically ill, having lost the skin from almost the entire surface of his body. “Except for his face, hands, and feet, he didn’t have any skin left,” his father recalls.

Having run out of conventional treatments, his doctors were preparing to start palliative care. But, as a last resort, they contacted an Italian scientist, Michele de Luca, who had carried out genetically modified skin transplants – but on nothing approaching this scale.

De Luca’s team went on to grow an entire replacement skin for Hassan. It was grafted on, like a patchwork quilt, and after spending months bandaged from head to toe, Hassan emerged effectively cured of his devastating illness. Two years on, he is well, his skin no longer blisters, he needs no medication or ointments, he plays football and, when he gets a cut, he heals normally.

“It felt like a dream for us,” the boy’s father says.

De Luca says that witnessing the recovery produced “one of the strongest emotions in my whole life … For a scientist working in this field, having these results justifies an entire career.”

It also marked a rare and long-awaited clinical success for the field of regenerative medicine, which has faced criticism for delivering just a handful of therapies after years of hype.

Scientists grow replacement skin for boy suffering devastating genetic disorder


Dec 2014 Isolation of esophageal stem cells with potential for therapy


Long gap esophageal atresia represents a significant challenge for pediatric surgeons and current surgical approaches are associated with significant morbidity. A tissue-engineered esophagus, comprising cells seeded onto a scaffold, represents a therapeutic alternative. In this study, we aimed to determine the optimal techniques for isolation and culture of mouse esophageal epithelial cells and to isolate CD34-positive esophageal epithelial stem cells from cadaveric mouse specimens. Cont…….


A team from UCL (University College London), Great Ormond Street Hospital (GOSH) and Royal Free Hospital are collaborating with the Cell Therapy Catapult on the development of a tissue repair product for babies in whom the esophagus has not developed properly. The project, funded by the UK Stem Cell Foundation and supported by UCL Business PLC, builds on work of UCL, GOSH, Royal Free Hospital and University College London Hospital. It is expected to result in the first clinical trial of a tissue-engineered oesophageal replacement in infants, providing effective swallowing as they grow.

Infants born with oesophageal atresia lack a complete esophagus, resulting in an inability to feed, long-term health issues and often requiring full-time care. Around 250 babies are born yearly with this condition in the UK, and as a leader in child health, GOSH treats a large number of these patients. With current surgical repair approaches generally ineffective, oesophageal atresia is an area of clear unmet medical need. The market for this condition is thought to be worth more than £70m pa, and a successful product would also have potential in oesophageal repair or replacement following trauma or disease in adults and children.

Using the expertise developed in previous studies, the project team will develop its oesophageal repair approach using stem and other cells taken from amniotic fluid in pregnancy, which is then grown on a donor ‘scaffold’ to create a new organ. Development and analysis of the prototype is expected to lead to a clinical trial in neonates in 2016. The Cell Therapy Catapult will be responsible for project management, as well as providing input around non-clinical, clinical and regulatory matters.

Dr Paolo De Coppi, a consultant pediatric surgeon at GOSH & clinical senior lecturer at UCL Institute of Child Health, said: ‘At Great Ormond Street we treat some of the most severe cases of oesophageal atresia, witnessing the difficult long-term health problems these children experience. This funding from the UKSCF, for which we are very grateful, will help us develop this technology, ultimately providing patients with an entirely new esophagus rather than trying to repair an inadequate one. This is part of our wider research into regenerative medicine, in which we are aiming to engineer rejection-free organs and tissues for transplant.’

Keith Thompson, CEO of the Cell Therapy Catapult, said, ‘The Cell Therapy Catapult is involved with several innovative scaffold-based tissue repair projects, and it’s clear that this is an important source of new products. We’re delighted that our first joint project with UKSCF is in this area of high unmet medical need, where our expertise can bring real patient benefit.’

Lil Shortland, CEO of UKSCF, said, ‘The UK Stem Cell Foundation is pleased to fund this important and ground-breaking research in the area of oesophageal atresia. With our focus on progressing the development of stem cell-based therapies for devastating conditions, this project is fully aligned with our objectives.’

Other links on this Site regarding stem cells


Cincinnati Children’s Launches Center for Stem Cell and Organoid Medicine

Nov 15, 2017

Mission is to Generate Human Organs to Study, Cure Disease.

CINCINNATINov. 15, 2017 /PRNewswire-USNewswire/ — Seven years ago Cincinnati Children’s scientists first used pluripotent stem cells to mimic natural human development and grow working human intestine in a lab. Today medical center doctors can bioengineer the gastrointestinal tissues of sick children to find clues about a child’s disease and how to treat it.

To watch the Video and read more about this click on the link below.


Sheikha Fatima’s donation brought tears to my eyes

July 17, 2014

On Wednesday, it was announced that HH Sheikha Fatima, “the Mother of the Nation”, had donated the amazing sum of £60 million (around Dh 384 million) towards the cost of a £90 million center for research into pediatric diseases at London’s Great Ormond Street Hospital for Sick Children, or GOSH.

I will confess that when I read the news, tears came to my eyes.

The Great Ormond Street Hospital, as many UAE residents will know, is one of the best hospitals for children anywhere in the world. It’s an institution that I am personally familiar with, having been directly involved with it twice in my life.

Once, 20 or so years ago, I and a group of British expatriates in Abu Dhabi, some of whom are still here, got together to raise a few tens of thousands of pounds for the hospital. That was a tough challenge but with a lot of effort and support from both Emiratis and expatriates, we reached our target and then had the pleasure of handing over a cheque for the amount raised to the late Diana, Princess of Wales on one of her visits to the UAE.


Gene, stem and cellular therapies at GOSH London

Nov 2017

Deputy Theme Lead: Professor Waseem Qasim

This theme aims to develop and refine our state-of-the-art infrastructure to enable us to deliver new gene and cell therapies into the NHS. These therapies use molecular tools to specifically edit faulty cells or genetic material and replace them with functioning versions. These techniques have previously been used at GOSH to successfully treat infants with childhood cancers such as leukemia.

During the new funding term, we aim to build on these successes and develop similar approaches for a wider range of conditions including areas of unmet need such as skin and muscular diseases.

To achieve this we will be investing in our infrastructure and regulatory processes, as well as working closely with industry partners.


Boost for stem cell research at GOSH

21 November 2017

A state-of-the-art stem cell research facility is now open at Great Ormond Street Hospital (GOSH), increasing our capacity for cutting-edge research into regenerative medicine.

Stem cells have the potential to develop into any type of cell in the human body and therefore hold great promise for regenerative medicine. They are increasingly used to help understand diseases, and to develop new personalised therapies.

GOSH’s new stem cell research facility, which is located in the UCL Great Ormond Street Institute of Child Health (ICH), specialises in induced pluripotent stem cells (iPSCs) – a type of stem cell that can be created from other tissues, including blood and skin samples donated by patients. The advantage of iPSCs is that there is a lower risk of rejection when the cells are transferred into the patient.

The facility was funded by Great Ormond Street Hospital Children’s Charity, and its running is supported by the National Institute for Health Research (NIHR) GOSH Biomedical Research Centre (BRC). The cutting-edge laboratory consists of sterile facilities and specialised equipment to grow and investigate stem cells. Projects already underway range from studies of childhood conditions affecting the muscle and liver, to the brain and the eyes. To celebrate the opening of the facility, the first iPSC Symposium was held in November where more than 140 researchers came to share their pioneering work on stem cells. Professor Jane Sowden, who set up the facility, said:

“The new laboratory will allow us to expand our research into stem cells for regenerative medicine, grow new tissues for transplantation and help us better understand how childhood diseases progress, paving the way for the development of new treatments to slow or stop complex childhood conditions.”

Using stem cells to restore vision

Dr. Elisa Cuevas and Professor Jane Sowden are using the new facility to grow stem cells which differentiate into retinal tissue – a layer of cells at the back of the eye essential for vision. Their goal is to develop light-sensitive photoreceptor cells so they can be transplanted into eyes of children with degenerative conditions such as Usher syndrome, to help improve their vision.

                                                Stem Cell – Autism trial                                                                      

Started in May 2017, this new study will determine the safety of intravenous infusions of human umbilical cord tissue-derived mesenchymal stem cells (MSC) in children with autism spectrum disorder.
Children participating in this study will receive up to three separate infusions 2 months apart at Duke University, North Carolina I am told the details will be released in 2019

Duke University, has a long established record using cord blood to treat to neurological conditions, such as autism, cerebral palsy and stroke, however, this is the first time an academic center in the USA has registered a trial of this nature using stem cells from cord tissue.

The New York Stem Cell Foundation (NYSCF) is uniquely positioned to make significant strides in improving human health.

The mission of The New York Stem Cell Foundation is to cure the major diseases of our time through stem cell research.

In addition to conducting innovative stem cell research in our own safe haven laboratories, NYSCF also supports and convenes global stem cell thought leaders and the broader stem cell community – all with the focus on getting to patients. Since our founding in 2005, NYSCF has been able to achieve outsized results in a relatively short period of time thanks to private philanthropy.

The NYSCF Research Institute, an independent laboratory, is a non-profit accelerator that serves to bridge the ongoing gap between research institutions and pharmaceutical and biotech companies by reducing the cost, time, and risk that historically inhibit the development of new treatments and cures. In just over one decade, NYSCF has created not only foundational tools for using stem cells in basic research but also pioneering technologies that will allow stem cells to be used therapeutically.

By pursuing and supporting only the most promising stem cell research and technologies, in our own laboratories and in partnership with leading institutions around the world, NYSCF serves as a catalyst in this transformational field, advancing our mission to accelerate cures for the major diseases of our time.

What Are Stem Cells?


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