Stem cells that could one day provide therapeutic options for muscle and bone disorders can be easily harvested from the umbilical cord, just as the blood that goes through it provides precursor cells to treat some blood disorders, said University of Pittsburgh School of Medicine researchers in the online version of the Journal of Biomedicine and Biotechnology.

Umbilical cord tissue cells can be expanded to greater number, are remarkably stable and might not trigger strong immune responses, said senior investigator Bridget M. Deasy, Ph.D., assistant professor in the Department of Orthopaedic Surgery, Pitt School of Medicine.

The cord could become an accessible source of a multitude of stem cells that overcomes many of the restrictions, such as limited quantity as well as donor age and donor sex issues, that come with other adult stem cell populations.

Dr. Deasy and her team analyzed sections of two-foot-long human umbilical cords that were donated for research, looking for cells in that displayed the characteristic protein markers found in stem cells derived from other sources. The researchers then sought to find the best way to isolate the stem cells from the cords, and tested them in the lab to confirm their ability to produce specialized cells, such as bone and cartilage, while retaining their invaluable ability to renew themselves.

To build on these findings, the team will test the umbilical cord stem cells in animal models of cartilage and bone repair, as well as muscle regeneration.

Co-authors of the paper include lead investigator Rebecca C. Schugar, of Pitt’s Stem Cell Research Center, Department of Orthopaedic Surgery, and the Center for Cardiovascular Research, Washington University School of Medicine; Steven M. Chirieleison, Yuko Askew, M.D., Ph.D., Jordan J. Nance, and Joshua M. Evron, all of the Pitt Stem Cell Research Center; Kristin E. Wescoe, Benjamin T. Schmidt, both of Pitt’s Department of Bioengineering; and Bruno Peault, Ph.D., of the University of California-Los Angeles and the McGowan Institute for Regenerative Medicine, a joint effort of Pitt and UPMC.

The research was supported by grants from the National Institute of Arthritis and Musculoskeletal Research and Children’s Hospital of Pittsburgh of UPMC.

A study initiated by the World Health Organization and published in the May Journal of the American Medical Association shows that stem cell transplantation has become an accepted therapy worldwide and that the majority of transplant patients received a source of their own (autologous) stem cells.

This global study evaluated the more than 50,000 stem cell transplants performed in a specific calendar year (2006) and found that in 57% of the cases,  the transplants used an autologous, or a patient’s own, source of stem cells for certain cancers and blood disorders. 

This is the first time that the current state of hematopoietic stem cell transplantation has been documented on a global level, say the authors, led by Alois Gratwohl, MD, from the University Hospital Basel in Switzerland. 

This information “has become a necessity for correct patient counseling and healthcare planning,” they add.

Hematopoietic stem cells collected from a newborn’s cord blood have been used successfully for more than 20 years in transplant medicine.  

They are recognized as having distinct advantages compared to other sources of stem cells, because they are younger, more adaptable and have limited exposure to viruses and other environmental factors that can alter cell function.

The authors also note that a matched sibling (within the family) might represent the most efficient way of therapy for a patient with a genetic disorder such as aplastic anemia, thalassemia or severe combined immunodeficiency.

The O’Neill family from Cambridge are the first New Zealanders to take part in a ground breaking medical study into cystic fibrosis, thanks to the generosity of CordBank.

The O’Neills decided to bank the cord blood of their second son, Lenny, in the hope that it may one day be able to help treat his five year old brother, Liam, who suffers from the disease. As a result, they stand to benefit from the study which aims to prove that umbilical cord blood can successfully treat cystic fibrosis.

Monique and Sean O’Neill said, “We were approached by The Cystic Fibrosis Association to see if we would be interested in taking part in the medical study which is supported by CordBank, who are providing complimentary cord blood banking to New Zealand families who have a cystic fibrosis child and want to bank a sibling’s cord blood”.

“We are so relieved that we made the decision to bank Lenny’s cord blood and are thankful to CordBank for all their help. We are very excited about the search for a cure using cord blood, and want to do everything we can to help our son Liam and other cystic fibrosis kids benefit in the future”.

 “Liam has a daily struggle with cystic fibrosis, and anything that can be done to help him and other young people with cystic fibrosis is an amazing thing as far as we are concerned.” 

The Cystic Fibrosis Association approached CordBank to see if the company would be interested in providing free cord blood collection for up to 3 families who had a cystic fibrosis child and wanted to bank a sibling’s cord blood. Once Professor Bob Williamson’s team at the University of Melbourne’s Faculty of Medicine has proved that umbilical cord blood can be used to regenerate lung cells for children with cystic fibrosis, the stored sibling cord blood will be used to treat the brother or sister suffering from the disease.

 “Most cystic fibrosis siblings will not have the mutation causing cystic fibrosis, and sibling cord blood has a one in four chance of being a perfect match that will not be rejected by the immune system of the child living with cystic fibrosis”, said Professor Williamson.

“However, the cord blood of cystic fibrosis siblings is too precious for us to use in our study, and we are able to do our work on samples donated by unrelated people who are not affected”.

“But thanks to CordBank in New Zealand, we are ensuring that affected families are banking precious sibling cord blood now so they are ready when a breakthrough comes.” 

Professor Williamson emphasised that the breakthrough could still be several years away, however even at this early stage, there are positive signs the project is going well, and some lung proteins have been expressed in the cord blood cells in the test tube, “Cord blood is a very exciting resource, full of different kinds of stem cells.  These have already been shown to form liver, brain and skin cells, and they represent a real hope for people with cystic fibrosis now that we can add lung cells to the list.  We already know that umbilical cord blood is known to be safe and effective when used to treat childhood cancers and leukaemia, and we hope that cord blood will provide cells with a potential to rebuild damaged lungs in people with cystic fibrosis.” 

Announcing CordBank’s involvement with the project, the Chief Executive of the New Zealand Cystic Fibrosis Association, Kate Russell, said CordBank’s support was very much appreciated.

“Cystic fibrosis is an expensive illness and families with a cystic fibrosis child can be placed under severe financial strain. People would be surprised at what the government does not fund in terms of the most basic medical equipment and support for these families”.

Cystic fibrosis is a life-shortening genetic condition, with no known cure at this time.  Many children with cystic fibrosis used to die in early childhood, but advances in treatment have extended life expectancy to 30 years and more.

The condition primarily affects the lungs and digestive system, causing a build up of thick sticky mucus in the body resulting in repeated lung infections and severely impaired digestion.  To keep healthy, people with cystic fibrosis must receive chest physiotherapy daily to keep their lungs free of mucus.  Regular antibiotic treatment, dozens of enzyme pills to aid digestion, and periods of hospitalisation are also a part of life with cystic fibrosis.

A foundation established by Julian Robertson, New Zealand’s first honorary knight and prominent philanthropist, has given $10.2 million to Duke University in the USA to advance the use of cord blood as a therapy.

Robertson, who is renowned for his philanthropy and investment in New Zealand, has over the last 15 years developed two luxury golf courses at Kauri Cliffs in Northland and Cape Kidnappers in Hawke’s Bay, and invested in several wineries, including Dry River in Wairarapa and Te Awa in Hawke’s Bay.

Duke University has announced that the donation by The Robertson Foundation will go to support the work of Dr Joanna Kurtzberg who will use some of the funding to move forward with the first placebo-controlled, randomized clinical trial in children with (cerebral palsy) that has been specifically designed to answer key questions about the efficacy of cord blood treatments in children with this condition.

To date over 200 children have been reinfused with their own cord blood by Dr Kurtzberg, including New Zealander Maia Friedlander who was successfully treated with her own cord blood at Duke in August 2008. Maia, who suffered oxygen deprivation at birth, has made significant progress. Maia’s cord blood had been stored with CordBank in New Zealand. To view her story go to www.cordbank.co.nz

Dr. Victor Dzau, Duke’s chancellor for health affairs and chief executive of the Duke University Health System, said the state-of-the-art Translational Cell Therapy Center would advance the university’s pioneering cell therapy research and treatment programs for children and adults with cancer, cerebral palsy, stroke and brain injuries suffered at birth. Dr. Joanne Kurtzberg and her research team have spent decades investigating the therapeutic use of umbilical cord blood stem cells, he said.

Umbilical cord blood stem cells, normally discarded after birth, have the ability to grow and develop into various types of cells throughout the body. They can be harvested after birth and stored for future transplant in patients with many types of blood disorders, and increasingly, other diseases as well.

“The emerging field of regenerative medicine has great promise, and this generous gift will accelerate the pace of Dr. Kurtzberg’s and other Duke scientists’ world-renowned, translational work in cell therapies,” Dzau said in a statement.

“The creation of the TCTC will support the work of many Duke researchers exploring various applications of cell-based therapies.”

 “Dr. Kurtzberg’s research reflects the kind of transformational science that has the potential to change the lives of thousands of people throughout the country and around the world,” Julian Robertson, of the Robertson Foundation, said in a statement.

In an interview on CNBC last year, Julian Robertson shared his excitement about the potential of cord blood stem cells, having heard about Maia Friedlander’s treatment at Duke from her parents.

Major news outlets recently reported research demonstrating that mesenchymal stem cells (MSCs), a specific type of stem cell with unique properties, restored transparency to the cloudy corneas of laboratory mice.  The data, presented by researchers during the American Society for Cell Biology Annual Meeting in December 2009, suggests that transplantation of umbilical MSCs could be a potential treatment regimen for corneal disease – whether present at birth or acquired.  This study provides further evidence supporting the potential of umbilical MSCs for a variety of diseases.

Based on a U.S. News and World Report article about the study, those with corneal diseases may stand to benefit most if/when such a therapy were to become clinically available.  They wouldn’t need to wait for a donated cornea, which, as the article states, are in short supply, so the prospect of an alternative therapy would be helpful.

More than 80 clinical trials are already underway using MSCs, and doctors are enthusiastic about the results reported for therapies addressing several conditions, including stroke, heart attack, bone injuries and autoimmune diseases like type 1 diabetes and multiple sclerosis. In addition, because MSCs serve as the foundation of connective tissue, applications in treating common joint and sports injuries may be another potential application with widespread use.  MSCs are found in bone marrow, fat tissue, and the umbilical cord.

A recent story filed by Agence France-Presse (AFP), a Paris-based news service, offered a very misleading perspective on the current state of cord blood banking as well as the legitimate medical use of cord blood stem cells. 

In essence, the reporter failed to recognize the difference between overseas “stem cell clinics” offering “unproven cures” and accredited cord blood banks in the U.S. that are facilitating FDA-approved research using a child’s own cord blood stem cells for new medical uses.

The story focused on comments made by one stem cell researcher, Dr. Irving Weissman, at the annual meeting of the American Association for the Advancement of Science (AAAS).  Dr. Weissman appropriately denounces the practices of non-accredited “stem cell clinics,” which are often based in foreign countries with poor medical regulations, that offer infusions of stem cells as definitive treatments for a wide range of conditions.  Several medical bodies and institutions have raised concerns about the inability of these clinics to substantiate their medical claims as well as the risk they pose to patient safety.

This is very different from the scientifically-valid and regulatory-approved research occurring in countries like the U.S. where pioneering physicians are studying the use of a child’s own cord blood stem cells as treatments for type 1 diabetes(1) and cerebral palsy(2).   In fact, just last week Medical College of Georgia announced a new FDA-approved clinical trial to evaluate the use of a child’s own cord blood stem cells to treat cerebral palsy.  It’s important to know that participants in these clinical trials were eligible because they had access to their own, genetically-unique cord blood stem cells because their parents made the decision to privately bank them at birth.

The protocols for these clinical trials require participants to have access to their own cord blood stem cells because they provide a guaranteed biological tissue match, do not require preparatory therapies like chemotherapy, do not pose the immune complications of donor cell infusions, and eliminate the need for anti-rejection drugs which can create complications.  In other words, using a child’s own cord blood stem cells allows researchers to explore the potential of these cells in a way that is safe for patients. 

It’s also important to note, that cord blood stem cells have been used for more than two decades to treat nearly 80 conditions, including certain cancers, blood disorders and immune diseases.  Furthermore, stem cell researchers and leading medical organizations recognize that cord blood stem cells have unique characteristics.  In fact, the American College of Obstetricians and Gynecologists states that cord blood stem cells “offers several distinct advantages” over other sources of stem cells when used in treatments for these types of conditions(3). 
 
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(1)Clincialtrials.gov. Umbilical Cord Blood Infusion to Treat Type 1 Diabetes. University of Florida. http://clinicaltrials.gov/ct2/show/NCT00305344?term=cord+blood+and+diabetes&rank=1. Accessed February 2010.
(2)Clinicaltrials.gov. Safety and Effectiveness of Cord Blood Stem Cell Infusion for the Treatment of Cerebral Palsy in Children. Medical College of Georgia. http://www.clinicaltrials.gov/ct2/show/NCT01072370?term=cerebral+palsy+and+cord+blood&rank=1. Accessed February 2010.
(3)ACOG Committee Opinion. Umbilical Cord Blood Banking. Obstetrics and Gynecology. 2008;111(2):475-477.

Francis Verter founded the Parent’s Guide to Cord Blood website  in 1998. Since its launch, it has provided expectant parents with unbiased information about cord blood stem cells and cord blood banks.

Review the list of diseases treated with stem cells.

In addition, Cord Blood Registry provides an overview of  stem cell therapy data and Viacord has an update on infusions for emerging treatments.

At Children’s Memorial Hermann, pediatric trauma expert Dr. Charles Cox is studying cord blood to see if it can regenerate damaged brain tissues.

“Cord blood is one piece of that puzzle,” Cox said. “There aren’t any good restorative therapies for brain injury, which is why we got into this years and years ago.”

So far, cord blood can treat 80 different diseases – a convincing statistic to the neo-natal staff. To read more

Dr Cox directs the Pediatric Surgical Translational Laboratories and Pediatric Program in Regenerative Medicine at The University of Texas Medical School at Houston, which address problems that originate with traumatic injury and the consequences of resuscitation and critical care. The Program focuses on progenitor cell based therapy (stem cells) for traumatic brain injury, and related neurological injuries (hypoxic-ischemic encephalopathy, stroke, spinal cord injury), recently completing the first acute, autologous cell therapy treatment Phase I study for traumatic brain injury in children. To read more about Dr Cox

From The Times

February 17, 2010

Mark Henderson, Science Editor

The medical potential of reprogrammed stem cells that do not require the destruction of embryos has been exaggerated, according to the head of one of the world’s leading regenerative medicine companies.

Thomas Okarma, the chief executive of Geron Corporation, told The Times that while so-called induced pluripotent stem (IPS) cells will be extremely useful in research, they are unlikely to be suitable for transplanting to patients to treat disease.

“IPS cells have been talked up as therapy by people with no experience of developing therapies,” Dr Okarma said. More>

Congratulations to our December promotion winners – Claire Boocock and Dougall Cameron – who are expecting their first baby next month. They entered the ‘register by 31 December’ draw and won a CordBank collection kit worth $750.

“We decided to register with CordBank due to Dougall’s history of Leukaemia. We wanted the security of knowing that should our baby experience any health issue in the future then we have provided the best option for cure with cord blood banking.

We have both known about banking cord blood for a few years now and after speaking with family and friends we know we’ve made the right decision for us and our baby.

The cost involved was no question when it comes to our baby’s health, but winning our $750 Collection Kit was a wonderful bonus. Now we’re just so excited to welcome our baby into our family!” commented Claire.