Public release date: 14-Mar-2013 [ | E-mail | Share ]

Contact: Andrea Slesinski aslesinski@hematology.org American Society of Hematology

(WASHINGTON)-Transplants of blood-forming stem cells from umbilical cord blood may be an effective alternative to transplants of matched donor bone marrow stem cells to treat children with a rare, debilitating disease known as Hurler's syndrome (HS), according to results of a study published online today in Blood, the Journal of the American Society of Hematology (ASH).

HS is an inherited metabolic disease characterized by the lack of a critical metabolic enzyme (lysosomal -L-iduronidase) that breaks down long chains of sugar molecules in the body. In the absence of the enzyme, the sugar chains build up in the body and damage vital organs. Children with the disease, who can be diagnosed at birth or as late as age 8, typically have a poor prognosis; serious complications such as deafness, halted growth, joint disease, or heart valve problems can rapidly develop after diagnosis and can lead to death. Recently, hematopoietic cell transplants (HCT) have become a valuable treatment for HS, as transplanted cells can generate new, healthy, enzyme producing blood cells to replace the diseased cells. These donor cells will deliver enzymes to all organs, including the brain, and can prevent disease progression. However, clinicians remain challenged to determine the optimal cell source and appropriate conditioning regimens to prepare patients for transplant that will maximize survival and minimize transplant-related morbidity and mortality.

"Cord blood has been proposed as an alternative stem cell source for children with HS since it has been suggested that cord blood may increase their levels of lysosomal -L-iduronidase, which consequently may allow them to live longer with fewer complications," said lead study author Jaap Jan Boelens, MD, PhD, of the University Medical Center Utrecht, in Utrecht, Netherlands. "However, until now, no studies have compared the safety and efficacy of different stem cell and donor sources among a large group of patients to determine which may be the ideal transplant source."

With this aim, Dr. Boelens and a team of investigators conducted a retrospective analysis analyzing outcomes of HS patients treated with HCT in centers affiliated with the European Group for Blood and Marrow Transplantation (EBMT), Eurocord, and the Center for International Blood and Marrow Transplant Research (CIBMTR). A total of 258 infants and children with HS (ranging from 2 months to 18 years, with a median age of 16 months at transplant) who received an HCT between 1995 and 2007 were selected for analysis based upon certain criteria: a confirmed diagnosis of HS; a transplant of cells from a matched sibling donor (MSD), a matched or mismatched unrelated donor (UD), or cells from a single, unrelated cord blood (UCB) unit; and pre-transplant treatment containing a high dose of chemotherapy to help prevent their immune systems from rejecting donated cells.

Nearly half of the children evaluated in the study (45%) had received transplants of unrelated cord blood (81% of which was from mismatched donors) with the others receiving transplants of bone marrow stem cells from unrelated donors or matched sibling donors. All the patients received pre-transplant high-dose chemotherapy, and 19 percent had previously received intravenous enzyme replacement therapy with lysosomal -L-iduronidase. Taking into consideration stem cell source and pre-transplant conditioning, the team compared the rates of overall survival (OS), event-free survival (EFS), engraftment (the degree to which transplanted donor cells were able to reproduce into new cells) or graft failure, or death.

The team noted encouraging survival rates among the children with HS who had received an HCT, regardless of source, with 5-year estimated OS and EFS at 74 percent and 63 percent, respectively. Importantly, EFS rates were similarly high for children who had received donations of matched sibling cells or fully matched unrelated umbilical cord blood (MSD or UCB, 81%), compared to EFS rates for patients transplanted with cells from matched unrelated donors (66%) or unmatched unrelated cord blood donors (68%). EFS rates were highest among infants who received transplants at age 16 months or younger, indicating the importance of transplantation as early as possible after diagnosis for these children, when treatment may help prevent further disease burden or damage.

Within two months of undergoing transplants, nearly all patients (91%) showed evidence of donor cell recovery (as noted by increasing levels of white blood cells in the blood). Only 12 percent of children experienced secondary graft failure (when the body rejects donor cells).

As is common in patients receiving stem cell transplants, investigators observed reports of graft-versus-host disease (GVHD, which occurs when the donated immune cells attack the patient's cells as foreign tissue) among the patient sample. At 100 days post-transplant, roughly one-fourth of transplant recipients experienced moderate to severe acute GVHD, and only 16 percent experienced chronic GVHD (remaining at five years post-transplant).

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Cord blood effective alternative to matched donor stem cells for kids with rare disorder

PROTEINS, carbohydrates and vitamins are all on the menu for a breastfed baby. Now it seems you can add stem cells to that list. Evidence is piling up that both breast milk and breast tissue contain embryonic-like stem cells.

That might mean we will soon have access to a source of stem cells without destroying embryos. This would be a boon as stem cells can turn into any type of human tissue, making them useful for treating degenerative diseases like Alzheimer's or for regrowing damaged heart muscle.

In 2011 Foteini Hassiotou at the University of Western Australia in Crawley and colleagues found stem cells in lactating breast tissue and breast milk. When they grew the breast milk cells they turned into the three types of cells from which all tissues and organs develop just like human embryonic stem cells (hESCs) do.

Hassiotou has since found this "pluripotency" in many more breast milk samples and thinks that breast milk stem cells could one day replace those from embryos. In one study, her team looked at fresh breast milk from more than 70 healthy breastfeeding women. They found that BMSCs expressed several genes that are also found in hESCs and help them replicate. Cultured samples also grew into different tissues including bone, neuron, heart and pancreatic cells (Human Lactation, DOI: 10.1177/0890334413477242).

In some cases, the team found that 30 per cent of all cells in breast milk were stem cells. In studies with monkeys and mice the cells were shown to pass into the bloodstream.

"One can speculate wildly about what they do in the baby," says Hassiotou. But she thinks that breastfed infants could be getting a developmental head start, with stem cells from the mother contributing to organ development in the newborn.

However, BMSCs fail one widely accepted test for embryonic cells: when injected into mice, they don't form a type of tumour called a teratoma. For many this failure is a deal-breaker.

But BMSCs are not alone. Mari Dezawa at Tohuku University in Sendai, Japan, and colleagues have found pluripotent cells called MUSE cells in bone marrow and connective tissue that do not form teratomas.

"The best stem cells might not make tumours," says Hassiotou. Dezawa agrees, and says that MUSE and BMSCs could be superior if they turn into a wide variety of cells without the risk of forming tumours.

Thea Tlsty at the University of California in San Francisco says she can imagine that there are pluripotent cells that do not make tumours. Her team recently identified pluripotent stem cells in breast tissue from non-lactating women and men (PNAS, doi.org/krn). Although her cells do form tumours, she agrees that the standard test needs revisiting.

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Are breast milk stem cells the real deal for medicine?

Public release date: 15-Mar-2013 [ | E-mail | Share ]

Contact: Ivanka Moerkerken i.moerkerken@uroweb.org 31-026-389-0680 European Association of Urology

Arnhem, 11 March 2013 - Transplantation of mesenchymal stem cells cultivated on the surface of nanofibrous meshes could be a novel therapeutic strategy against post-prostatectomy erectile dysfunction (ED), conclude the authors of a study which is to be presented at the 28th Annual EAU Congress later this week.

The study was conducted by a group of Korean scientists and will be awarded 3rd prize for best abstract in non-oncology research on the opening day of the congress.

During their investigation, the group aimed to examine the differentiation of human mesenchymal stem cells cultivated on the surface of nanofibrous meshes (nano-hMSCs) into neuron-like cells and repair of erectile dysfunction using their transplantation around the injured cavernous nerve (CN) of rats.

"The objectives of the study reflect a very pertinent need in today's urology practice," said the lead author of the investigation Prof. Y.S. Song of Soonchunhyang University School of Medicine in South Korea. "Post-prostatectomy erectile dysfunction results from injury to the cavernous nerve that provides the autonomic input to erectile tissue. It is a common complication after radical prostatectomy which decreases the patient's quality of life".

"Although advances in equipment and surgical techniques reduce this complication, patients still experience erectile dysfunction after radical prostatectomy," he explained.

Treatment of phosphodiesterase 5 inhibitors shows insufficient effectiveness in the treatment of post-prostatectomy ED and it is believed that the transplantation of stem cells cultivated on the surface of nanofibrous meshes can promote cavernous neuronal regeneration and repair erectile dysfunction.

In the course of the study, the synthesised polymer was electrospun in a rotating drum to prepare nanofibrous meshes and hMSCs were prepared and confirmed. Eight week old male Sprague-Dawley rats were divided into 4 groups of 10 each, including sham operation (group 1), CN injury (group 2), hMSCs treatment after CN injury (group 3) and nano-hMSCs treatment after CN injury (group 4). Immediately after the CN injury in group 4, nano-hMSCs encircled the injured CN. Erectile response was assessed by CN stimulation at 2, 4 weeks. Thereafter, penile tissue samples were harvested and examined using morphological analysis and immuno-histochemical stain against nerves (nestin, tubulin III and map2), endothelium (CD31,vWF) and smooth muscle (smooth muscle actin).

The results of the study revealed that at 2, 4 weeks, transplantation of nano-hMSCs increased the expression levels of cavernous neuronal, endothelial and smooth muscle makers more than hMSCs alone.

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Stem cells transplantation technique has high potential as a novel therapeutic strategy for ED

Washington, March 13 (IANS) A new study has found that stem cells from the patient's own body fat could help seek out and destroy cancer cells in a type of brain tumour.

Johns Hopkins researchers say they have found the stem cells from body fat may have the potential to deliver new treatments directly into the brain after the surgical removal of a glioblastoma, the most common and aggressive form of brain tumour.

According to investigators, the mesenchymal stem cells (MSCs) have an unexplained ability to seek out damaged cells, such as those involved in cancer, and may provide clinicians a new tool for accessing difficult-to-reach parts of the brain where cancer cells can hide and proliferate anew.

The researchers say harvesting MSCs from fat is less invasive and less expensive than getting them from bone marrow, a more commonly studied method, reports Science Daily.

Results of the Johns Hopkins proof-of-principle study are described online in the journal PLOS ONE.

"The biggest challenge in brain cancer is the migration of cancer cells. Even when we remove the tumour, some of the cells have already slipped away and are causing damage somewhere else," says study leader Alfredo Quinones-Hinojosa, professor of neurosurgery, oncology and neuroscience at the Johns Hopkins University School of Medicine.

"Building off our findings, we may be able to find a way to arm a patient's own healthy cells with the treatment needed to chase down those cancer cells and destroy them. It's truly personalised medicine," adds Quinones-Hinojosa.

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Stem cells from body fat can destroy tumour cells : Study