Stem Cell Clinic

UCLA Researchers Unlock Protein Key to Harnessing Regenerative Power of Blood Stem Cells

By Stem Cell Medical Center

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Newswise In a study led by Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research member, Dr. John Chute, UCLA scientists have for the first time identified a unique protein that plays a key role in regulating blood stem cell replication in humans.

This discovery lays the groundwork for a better understanding of how this protein controls blood stem cell growth and regeneration, and could lead to the development of more effective therapies for a wide range of blood diseases and cancers.

The study was published online November 21, 2014 ahead of print in the Journal of Clinical Investigation.

Hematopoietic stem cells (HSCs) are the blood-forming cells that have the remarkable capacity to both self-renew and give rise to all of the differentiated cells (fully developed cells) of the blood system. HSC transplantation provides curative therapy for thousands of patients annually. However, little is known about the process through which transplanted HSCs replicate following their arrival in human bone marrow. In this study, the authors showed that a cell surface protein called protein tyrosine phosphatase-sigma (PTP-sigma) regulates the critical process called engraftment, meaning how HSCs start to grow and make health blood cells after transplantation.

Mamle Quarmyne, a graduate student the lab of Dr. Chute and first author of the study, demonstrated that PTP-sigma is produced (expressed) on a high percentage of mouse and human HSCs. She showed further that genetic deletion of PTP-sigma in mice markedly increased the ability of HSCs to engraft in transplanted mice.

In a complementary study, she demonstrated that selection of human blood HSCs which did not express PTP-sigma led to a 15-fold increase in HSC engraftment in transplanted immune-deficient mice. Taken together, these studies showed that PTP-sigma suppresses normal HSC engraftment capacity and targeted blockade of PTP-sigma can substantially improve mouse and human HSC engraftment after transplantation.

Chute and colleagues showed further that PTP-sigma regulates HSC function by suppressing a protein, RAC1, which is known to promote HSC engraftment after transplantation.

These findings have tremendous therapeutic potential since we have identified a new receptor on HSCs, PTP-sigma, which can be specifically targeted as a means to potently increase the engraftment of transplanted HSCs in patients, said Chute, senior author of the study and UCLA Professor of Hematology/Oncology and Radiation Oncology. This approach can also potentially accelerate hematologic recovery in cancer patients receiving chemotherapy and/or radiation, which also suppress the blood and immune systems.

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UCLA Researchers Unlock Protein Key to Harnessing Regenerative Power of Blood Stem Cells

Cambridge stem cell pioneer targets China partners

By Uncategorized

Cambridge stem cell pioneer DefiniGEN is in China this week showcasing technology that arguably gives the UK a world lead in countering liver and pancreatic cancer.

The young company is seeking Chinese partners to broaden the reach of the technology which holds a potentially significant payback in regenerative medicine.

With US global stem cell innovator Roger Pedersen among its technology founders, DefiniGEN was founded two years ago to commercialise a stem cell production platform developed at the University of Cambridge.

The platform generates human liver and pancreatic cell types using Nobel Prize winning human Induced Pluripotent Stem Cell (iPSC) technology.

DefiniGEN is visiting Shanghai and Beijing on a trade mission organised by UKTI East of England in partnership with the China-Britain Business Council.

The company is actively looking to partner with Life Science distributors and pharmaceutical drug discovery companies in China. CEO Dr Marcus Yeo and Dr Masashi Matsunaga business development manager for Asia Pacific - are spearheading the initiative.

The visit includes a range of medically-focused ventures from one to one meetings with key players to presentations at UK consulates.

DefiniGEN cells are provided to the drug discovery sector for use in lead optimisation and toxicity programmes.

The companys OptiDIFF platform produces validated libraries of disease-modelled human liver cells for a range of diseases. The phenotype (the composite of an organisms traits) and pathology of the diseases is pre-confirmed in the cells.

The technology provides pharmaceutical companies with more predictive in vitro cell products enabling the development of safer and more effective treatments.

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Cambridge stem cell pioneer targets China partners

Cell therapy trial offers new hope to liver disease patients

By Uncategorized

PUBLIC RELEASE DATE:

24-Nov-2014

Contact: Jen Middleton jen.middleton@ed.ac.uk 44-131-650-6514 University of Edinburgh @uniofedinburgh

Liver disease patients could be helped by a new cell therapy to treat the condition.

Researchers from the University of Edinburgh have received funding to start testing the therapy in patients within the next year.

It will be the world's first clinical trial of a new type of cell therapy to treat liver cirrhosis, a common disease where scar tissue forms in the organ as a result of long-term damage.

The Edinburgh team has received funding from the Medical Research Council and Innovate UK to investigate the disease, which claims 4000 lives in the UK each year.

The only successful treatment for end-stage liver cirrhosis at present is an organ transplant. The new therapy is based on a type of white blood cell called a macrophage, which is key to normal repair processes in the liver.

Macrophages reduce scar tissue and stimulate the liver's own stem cells to expand and form into healthy new liver cells.

Scientists will take cells from the blood of patients with liver cirrhosis and turn them into macrophages in the lab using chemical signals.

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Cell therapy trial offers new hope to liver disease patients

Okyanos Adopts WAL/ body-jet eco for Use in Cell Therapy

By Uncategorized

Freeport, Grand Bahama (PRWEB) November 24, 2014

Okyanos, the leader in cell therapy, announced the adoption of body-jet eco for use in the harvesting of adult stem cells for use in cell therapy. The Okyanos procedure begins with the extraction of a small amount of body fat, a process done using advanced water-jet assisted liposuction technology. The body-jet eco system is utilized during this procedure and allows a larger number of viable adult stem cells to be harvested. After separating the cells from fat tissue, the Okyanos medical doctor immediately injects these cells into and around the area needing treatment allowing targeting of the cells to repair damaged tissue.

According to Dr. Todd Malan, Chief Cell Therapy Officer and General Surgeon at Okyanos, who was involved in helping develop the appropriate settings of the body-jet eco use in adult stem cell harvesting, The body-jet eco was used during our first stem cell procedure at Okyanos. It performed flawlessly as expected and we feel it meets our tough standards. This is much gentler and more precise, making the overall procedure faster with less trauma to the surrounding tissue and less diversion of the adult stem cells from the intended area.

The body-jet eco is part of the water-jet assisted liposuction (WAL) family of devices, which detaches the fat gently from the tissue structure using a flat, fan-shaped water jet spray. The surrounding connective tissue, nerves and blood vessels remain in-tact which makes this procedure much gentler on the patient and leads to a quicker recovery with less pain medication required. The WAL process has a very high viability of fat cells and stem cells with a high take rate after fat grafting. The WAL family of devices is manufactured by human med AG with its headquarters in Schwerin, Germany, and distributed in North America by CAREstream America with its headquarters in Altamonte Springs, Florida.

Because the treatment is minimally invasive it requires that patients be under only moderate sedation. Post-procedural recovery consists of rest in a private suite for several hours that comfortably accommodates up to 3 family members.

Patients can contact Okyanos at http://www.okyanos.com or by calling toll free at 1-855-659-2667.

About CAREstream America: CAREstream America began in 2013 and is a division of CAREstream Medical Ltd, which has serviced Canadian customers respiratory and anesthesia needs for over 15 years. CAREstream America retains North American distribution rights to the full water-jet assisted human med AG product line. CAREstream America is the premier distributor of Aesthetic product lines ranging from water-jet assisted technology to vascular access imaging to nitrous oxide analgesia which help shape the body, showcase the veins and relieve the pain and anxiety of aesthetic procedures.

About Dr. Malan: Todd Malan, MD, serves as the Chief Cell Therapy Officer and General Surgeon at Okyanos Heart Institute, overseeing the liposuction and stem cell isolation step of the Okyanos cardiac cell therapy process. Known as an innovative cosmetic surgeon, Dr. Malans practices combine the most progressive and minimally-traumatic liposuction technologies available. A pioneer of fat-derived stem cell therapies, he became the first physician in the US to utilize stem cells from fat for soft tissue reconstruction in October, 2009, combining water-assisted liposuction, fat transfer and adult stem cell technologies.

About Okyanos: (Oh key AH nos) Based in Freeport, Grand Bahama, Okyanos brings a new standard of care and a better quality of life to patients with coronary artery disease, tissue ischemia, autoimmune diseases, and other chronic neurological and orthopedic conditions. Okyanos Cell Therapy utilizes a unique blend of stem and regenerative cells derived from patients own adipose (fat) tissue which helps improve blood flow, moderate destructive immune response and prevent further cell death. Okyanos is fully licensed under the Bahamas Stem Cell Therapy and Research Act and adheres to U.S. surgical center standards. The literary name Okyanos, the Greek god of the river Okyanos, symbolizes restoration of blood flow.

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Okyanos Adopts WAL/ body-jet eco for Use in Cell Therapy

New Stem Cell Treatment Found To Cure 'Bubble Baby' Disease

By Stem Cell Treatment

Chuck Bednar for redOrbit.com Your Universe Online

A new stem cell gene therapy developed by researchers at UCLA is set to begin clinical trials early next year after the technique reportedly cured 18 children who were born without working immune systems due to a condition known as ADA-deficient Severe Combined Immunodeficiency (SCID) or Bubble Baby disease.

The treatment was developed by Dr. Donald Kohn, a member of the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, and his colleagues, and according to the university, it is able to identify and correct faulty genes by using the DNA of the youngsters born with this life-threatening condition.

Left untreated, ADA-deficient SCID is often fatal within the first year of a childs life, reports Peter M. Bracke for UCLA. However, after more than three decades of research, Dr. Kohns team managed to develop a gene therapy that can safely restore the immune systems of children with the disease by using their own cells and with no noticeable side effects.

All of the children with SCID that I have treated in these stem cell clinical trials would have died in a year or less without this gene therapy, instead they are all thriving with fully functioning immune systems, Dr. Kohn, who is also a professor of pediatrics and of microbiology, immunology and molecular genetics, said in a recent statement.

Children born with SCID have to be isolated in a controlled environment for their own safety, because without an immune system, they are extremely vulnerable to illnesses and infections that could be deadly. While there are other treatments for ADA-deficient SCID, Dr. Kohn noted that they are not always optimal or feasible for many children. The new technique, however, provides them with a cure, and the chance to live a full healthy life.

SCID is an inherited immunodeficiency that is typically diagnosed about six months after birth, the researchers said, and children with the condition are so vulnerable to infectious diseases that even the common cold could prove fatal to them. This particular form of the condition causes cells to not create ADA, an enzyme essential for the production of the white blood cells which are a vital component of a healthy, normally-functioning immune system.

Approximately 15 percent of all SCID patients are ADA-deficient, according to the university, and these youngsters are typically treated by being injected twice per week with the required enzyme. This is a process that must continue throughout a patients entire life, and even then it doesnt always work to bring their immune systems to optimal levels. Alternately, they could undergo bone marrow transplants from matched siblings, but those matches are rare and the transplanted cells themselves are often rejected by the childs body.

Dr. Kohn and his colleagues tested two therapy regimens on 18 ADA-deficient SCID over the course of two multi-year clinical trials starting in 2009. During the trials, the blood stem cells of the patients were removed from their bone marrow and genetically modified in order to correct the defect. All 18 of the patients were cured.

The technique used a virus delivery system first developed in Dr. Kohns laboratory in the 1990s a technique which inserts the corrected gene that produces the ADA into the blood forming stem cells in the bone marrow. The genetically corrected blood-forming stem cells will then produce the T-cells required to combat infections.

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New Stem Cell Treatment Found To Cure 'Bubble Baby' Disease

Human embryonic stem cell-derived retinal pigment epithelium in patients with age-related macular degeneration and …

By Stem Cell Medicine

Background

Since they were first derived more than three decades ago, embryonic stem cells have been proposed as a source of replacement cells in regenerative medicine, but their plasticity and unlimited capacity for self-renewal raises concerns about their safety, including tumour formation ability, potential immune rejection, and the risk of differentiating into unwanted cell types. We report the medium-term to long-term safety of cells derived from human embryonic stem cells (hESC) transplanted into patients.

There was no evidence of adverse proliferation, rejection, or serious ocular or systemic safety issues related to the transplanted tissue. Adverse events were associated with vitreoretinal surgery and immunosuppression. 13 (72%) of 18 patients had patches of increasing subretinal pigmentation consistent with transplanted retinal pigment epithelium. Best-corrected visual acuity, monitored as part of the safety protocol, improved in ten eyes, improved or remained the same in seven eyes, and decreased by more than ten letters in one eye, whereas the untreated fellow eyes did not show similar improvements in visual acuity. Vision-related quality-of-life measures increased for general and peripheral vision, and near and distance activities, improving by 1625 points 312 months after transplantation in patients with atrophic age-related macular degeneration and 820 points in patients with Stargardt's macular dystrophy.

The results of this study provide the first evidence of the medium-term to long-term safety, graft survival, and possible biological activity of pluripotent stem cell progeny in individuals with any disease. Our results suggest that hESC-derived cells could provide a potentially safe new source of cells for the treatment of various unmet medical disorders requiring tissue repair or replacement.

Advanced Cell Technology.

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Human embryonic stem cell-derived retinal pigment epithelium in patients with age-related macular degeneration and ...

Stem cell trial to begin for children suffering from cerebral palsy

By Stem Cell Treatment

Nov. 23, 2014, 3:05 p.m.

It is hoped that a trial due to start next year, and involving about 20 Australian children with cerebral palsy, will show the benefits of using stem cells from their own umbilical cord blood to treat the condition.

About 20 Australian children with cerebral palsy will be infused with their own umbilical cord blood in a trial due to start next year, as physicians warn families against travelling overseas for experimental stem cell treatments.

The long-awaited Australian trial will provide some of the world's first evidence about the safety and effectiveness of using stem cells from umbilical cord blood to repair brain injury that leads to cerebral palsy.

Researchers are waiting on ethics approval for the trial which will provide treatment to families who have chosen to store their child's cord blood at private banks.

In some cases, children with cerebral palsy will be able to receive a sibling's cord blood if this is available.

Cerebral Palsy Alliance head of research Iona Novak said the study, led by the Murdoch Childrens Research Institute, will recruit children from around Australia who have access to privately banked cord blood.

Children aged one to 10 will receive infusions at private blood banks in Melbourne, Sydney and Brisbane, and will be assessed before and after the treatment to check for improvements.

Researchers will be unable to access cord blood from a public bank, which collects blood to treat blood disorders such as leukaemia and cannot be used for untested new therapies.

Associate Professor Novak said the trial was an important first step towards establishing whether stem cells could help repair the brain injury that leads to cerebral palsy, a series of disabilities associated with movement and posture.

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Stem cell trial to begin for children suffering from cerebral palsy

Stem cell failure 'led to children's deaths' at Great Ormond Street

By Stem Cell Doctors

preservation laboratory, where stem cells were kept for use in transplants in children whose bone marrow has been damaged during chemotherapy.

Concerns were first raised when Sophie Ryan-Palmer, 12, who had acute lymphoblastic leukaemia, failed to make progress after her transplant in June 2013, which involved using a donors stem cells rather than her own.

She had been diagnosed with leukaemia at the age of two and had undergone three previous transplants. She began fundraising for cancer charities when she was six.

By October last year the hospital had identified that a higher than usual proportion of eight children who had undergone stem cell transplantation between March and August had suffered what doctors call delayed engraftment. But by the time it stopped freezing stem cells on site at its base in Bloomsbury, central London, and launched an investigation, three of the eight had died.

Ryan Loughran, aged 13 months, from Bournemouth, died on July 10. Sophie, from Sunbury in Middlesex, followed on July 17. Katie Joyce, from Hertfordshire, died on October 6. A fourth patient, Muhanna al-Hayany, aged five, died in August this year. He had come from Kuwait to have the treatment. Following the deaths it was discovered that the method used to freeze the stem cells had inexplicably stopped working and that, although still alive, the cells were unable to mature properly.

At the inquest, Katie Beattie, the barrister representing Katie Joyces family, questioned whether the girls transplant in August should have been suspended, knowing Sophie and Ryan had died the previous month. Great Ormond Street went ahead even though there was plenty of time to stop it, she said.

Doctors from the hospital told St Pancras coroners court that they regretted not halting transplants sooner and Katies life might have been saved if they had. But they said they believed they were doing the right thing by continuing with the transplants because cancer doesnt wait.

Great Ormond Street said it has since overhauled its procedures to prevent further incidents, but is still investigating why the freezing process stopped working.

A spokesman said: Before giving our patients any frozen cells we carried out tests, which are standard across most laboratories in the UK, to ensure they were alive and viable. All of the samples passed these tests, so there was nothing to suggest there was a problem at this stage.

The coroner, Mary Hassell, is expected to deliver verdicts on all four deaths on Tuesday.

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Stem cell failure 'led to children's deaths' at Great Ormond Street

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