Groundbreaking Clinical Trials Study Cord Blood Stem Cells to Help Treat Brain Injury and Hearing Loss

SAN BRUNO, Calif., Feb. 16, 2012 /PRNewswire/ -- Cord Blood Registry (CBR) is the exclusive partner for a growing number of clinical researchers focusing on the use of a child's own cord blood stem cells to help treat pediatric brain injury and acquired hearing loss. To ensure consistency in cord blood stem cell processing, storage and release for infusion, three separate trials have included CBR in their FDA-authorized protocol—including two at the University of Texas Health Science Center at Houston (UTHealth) working in partnership with Children's Memorial Hermann Hospital, and a third at Georgia Health Sciences University, home of the Medical College of Georgia (MCG). This makes CBR the only family stem cell bank pairing researchers with prospective patients for these studies. 

(Logo: http://photos.prnewswire.com/prnh/20120216/AQ54476LOGO)

"Partnering with a series of specialists who want to research the use of a child's own newborn blood stem cells on a variety of disease states allows CBR to help advance medical research for regenerative therapies by connecting the child whose family banked with CBR to appropriate researchers," said Heather Brown, MS, CGC, Vice President of Scientific & Medical Affairs at Cord Blood Registry.  "The pediatric specialists from UTHealth, Children's Memorial Hermann Hospital, and Georgia Health Sciences University are at the forefront of stem cell research as they evaluate cord blood stem cells' ability to help facilitate the healing process after damage to nerves and tissue."

Hearing Loss and Traumatic Brain Injury Clinical Trials Break New Ground

Sensorineural hearing loss affects approximately 6 per 1,000 children by 18 years of age, with 9 percent resulting from acquired causes such as viral infection and head injury.(1,2,3)  The Principal Investigator of the hearing loss study is Samer Fakhri, M.D., surgeon at Memorial Hermann-Texas Medical Center and associate professor and program director in the Department of Otorhinolaryngology – Head & Neck Surgery at UTHealth.  He is joined by James Baumgartner, M.D., sponsor of the study and guest research collaborator for this first-of-its-kind FDA-regulated, Phase 1 safety study of the use of cord blood stem cells to treat children with acquired hearing loss. The trial follows evidence from published studies in animals that cord blood treatment can repair damaged organs in the inner ear. Clients of CBR who have sustained a post-birth hearing loss and are 6 weeks to 2 years old may be eligible for the year-long study. "The window of opportunity to foster normal language development is limited," said James Baumgartner, M.D.  "This is the first study of its kind with the potential to actually restore hearing in children and allow for more normal speech and language development."

Although the neurologic outcome for nearly all types of brain injury (with the exception of abuse) is better for children than adults,(4,5) trauma is the leading cause of death in children,(6) and the majority of the deaths are attributed to head injury.(7) Distinguished professor of pediatric surgery and pediatrics at UTHealth, Charles S. Cox, M.D. launched an innovative study building on a growing portfolio of research using stem cell-based therapies for neurological damage. The study will enroll 10 children ages 18 months to 17 years who have umbilical cord blood banked with CBR and have suffered a traumatic brain injury (TBI) and are enrolled in the study within 6-18 months of sustaining the injury. Read more about the trial here.

"The reason we have become interested in cord blood cells is because of the possibility of autologous therapy, meaning using your own cells. And the preclinical models have demonstrated some really fascinating neurological preservation effects to really support these Phase 1 trials," says Charles S. Cox, M.D., principle investigator of the trial. "There's anecdotal experience in other types of neurological injuries that reassures us in terms of the safety of the approach and there are some anecdotal hints at it being beneficial in certain types of brain injury."

Georgia Health Sciences University (GHSU) Focuses on Cerebral Palsy

At the GHSU in Augusta, Dr. James Carroll, professor and chief of pediatric neurology, embarked on the first FDA-regulated clinical trial to determine whether an infusion of stem cells from a child's own umbilical cord blood can improve the quality of life for children with cerebral palsy. The study will include 40 children whose parents have stored their cord blood at CBR and meet inclusion criteria. 

"Using a child's own stem cells as a possible treatment is the safest form of stem cell transplantation because it carries virtually no threat of immune system rejection," said Dr. Carroll. "Our focus on cerebral palsy breaks new ground in advancing therapies to change the course of these kinds of brain injury—a condition for which there is currently no cure."

Cerebral palsy, caused by a brain injury or lack of oxygen in the brain before birth or during the first few years of life, can impair movement, learning, hearing, vision and cognitive skills. Two to three children in 1,000 are affected by it, according to the Centers for Disease Control.(8)

Cord Blood Stem Cell Infusions Move From the Lab to the Clinic

These multi-year studies are a first step to move promising pre-clinical or animal research of cord blood stem cells into clinical trials in patients. Through the CBR Center for Regenerative Medicine, CBR will continue to partner with physicians who are interested in advancing cellular therapies in regenerative applications.

"The benefits of cord blood stem cells being very young, easy to obtain, unspecialized cells which have had limited exposure to environmental toxins or infectious diseases and easy to store for long terms without any loss of function, make them an attractive source for cellular therapy researchers today," adds Brown. "We are encouraged to see interest from such diverse researchers from neurosurgeons to endocrinologists and cardiac specialists."

About CBR

CBR® (Cord Blood Registry®) is the world's largest and most experienced cord blood bank.  The company has consistently led the industry in technical innovations and supporting clinical trials. It safeguards more than 400,000 cord blood collections for individuals and their families. CBR was the first family bank accredited by AABB and the company's quality standards have been recognized through ISO 9001:2008 certification—the global business standard for quality. CBR has also released more client cord blood units for specific therapeutic use than any other family cord blood bank. Our research and development efforts are focused on helping the world's leading clinical researchers advance regenerative medical therapies. For more information, visit http://www.cordblood.com.

(1)  Bergstrom L, Hemenway WG, Downs MP. A high risk registry to find congenital deafness. Otolaryngol Clin North Am. 1977;4:369-399.
(2)  Billings KR, Kenna MA. Causes of pediatric sensorineural hearing loss: yesterday and today. Arch Otolaryngol Head Neck Surg. 1999 May;125(5):517-21.
(3)  Smith RJ, Bale JF Jr, White KR. Sensorineural hearing loss in children. Lancet. 2005;365(9462):879-890.
(4)  Faul M, Xu L, Wald MM, Coronado VG. Traumatic brain injury in the United States: emergency department visits, hospitalizations, and deaths. Atlanta (GA): Centers for Disease Control and Prevention, National Center for Injury Prevention and Control; 2010.
(5)  Schnitzer, Patricia, PH.D., "Prevention of Unintentional Childhood Injuries", American Academy of Family Physicians, 2006.
(6)  Centers for Disease Control and Prevention, "10 Leading Causes of Death, United States, 1997-2007", WISQARS, National Center for Health Statistics (NCHS), National Vital Statistics System
(7)  Marquez de la Plata, Hart et al, National Institutes of Health, "Impact of Age on Long-term Recovery From Traumatic Brain Injury", Arch Phys Med Rehabilitation, May 2008.
(8)  Centers for Disease Control and Prevention, http://www.cdc.gov/Features/dsCerebralPalsy, accessed February 6, 2012

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Groundbreaking Clinical Trials Study Cord Blood Stem Cells to Help Treat Brain Injury and Hearing Loss

Synthetic protein amplifies genes needed for stem cells

Public release date: 16-Feb-2012
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Contact: Tara Womersley
tara.womersley@ed.ac.uk
44-131-650-9836
University of Edinburgh

Scientists have found a way to generate and maintain stem cells much more efficiently by amplifying the effect of an essential protein.

Researchers from Denmark, Scotland and the USA have created synthetic versions of a protein, which manipulates adult cells ? such as skin cells ? so that they can subsequently revert to an earlier, embryonic like state. These reverted cells have the potential to become any cell in the body.

As well as reverting adult cells to this state ? known as induced pluripotent stem cells , the protein also plays a key role in maintaining embryonic stem cells in a pure form. If the protein ? Oct4 ? is not present, the embryonic stem cells will start to differentiate into specific cells.

In order to reprogamme adult cells to have stem cell properties viruses need to be added to cell cultures to trigger production of significant quantities of Oct4.

Oct4 plays a powerful role in regulating stem cell genes. However, while large quantities of Oct4 are needed too much of it can ruin the properties of stem cells.

Scientists, whose work is published in the journal Cell Reports, were able to overcome this by producing a synthetic version of Oct4 that amplified the effect of the protein in its natural form.

The synthetic version of Oct4 was much more efficient in turning on genes that instruct cells on how to be stem cells and, as a result, the cells did not need as much Oct4 for either reprogramming or to remain as stem cells ? thereby eliminating problems caused by too much Oct4.

In fact, the synthetic Oct4 could support stem cells under conditions that they do not normally grow. These findings could also help scientists find new ways generate stem cells in the laboratory.

The study showed that Oct4 was mainly responsible for turning on genes that instruct cells on how to become stem cells, rather than turning off genes that encourage the cells to differentiate.

"Our discovery is an important step towards generating and maintaining stem cells much more effectively," says Professor Joshua Brickman, affiliated with both The Danish Stem Cell Center (DanStem), University of Copenhagen and Medical Research Council Centre for Regenerative Medicine at the University of Edinburgh.

"Embryonic stem cells are characterized, among other things, by their ability to perpetuate themselves indefinitely and differentiate into all the cell types in the body ? a trait called pluripotency. But to be able to use them medically, we need to be able to maintain them in a pure state, until they're needed. When we want to turn a stem cell into a specific cell, such as insulin producing beta cell, or a nerve cell in the brain, we'd like this process to occur accurately and efficiently. This will not be possible if we don't understand how to maintain stem cells as stem cells. As well as maintaining embryonic stem cells in their pure state more effectively, the artificially created Oct4 was also more effective at reprogramming adult cells into so-called induced Pluripotent Stem cells, which have many of the same traits and characteristics as embryonic stem cells but can derived from the patients to both help study degenerative disease and eventually treat them.."

Oct4 is a so-called transcription factor ? a protein that binds to specific DNA sequences, thereby controlling the flow (or transcription) of genetic information from DNA to mRNA. The synthetic version of Oct4 was created by using recombinant DNA technology whereby a gene was modified to produce new and more active protein. The modified gene was either introduced into stem cells or used to reprogram adult skin cells.

If scientists can exploit this programming of stem cell programs, it will improve the ability to generate stem cells directly from a patient. These cells could in turn potentially be used for individualised studies and for developing individualized therapies for degenerative diseases such as type 1 diabetes and neuro-degenerative diseases.

###

The paper "Transcriptional Activation by Oct4 Is Sufficient for the Maintenance and Induction of Pluripotency", is published in Cell Reports on February 16, 2012, 12:00 EST US time/18:00 Danish time/17:00 UK time. The study involved mouse embryonic stem cells, early embryonic progenitors cells in frogs as well as iPS cells from both mouse and human sources. The research was supported by grants from the Novo Nordisk Foundation (DK), the Medical Reseach Council and the Biotechnology and Biological Sciences Research Council (MRC and BBSRC, UK).

Contact: Tara Womersley, Press and PR Officer, University of Edinburgh, 44-131-650-9836 or 44-7791-355-804

Link to Cell Report: http://cellreports.cell.com/

Embargo: Until February 16 at 12:00 EST US time/18:00 Danish time/17:00 UK time

About DanStem

The Danish Stem Cell Center opened in the Summer 2011 as a hub for international basic, translational and early clinical stem cell research. Professor Brickman and his group joined DanStem in October 2011 to partake in the build-up the center.

DanStem address basic questions in stem cell and developmental biology, and develop novel stem cell based therapeutic approaches for diabetes and cancer. It is supported by two major grants from Novo Nordisk Foundation (DKK 350 million (? 47 million)) and the Danish Research Council for Strategic Research (DKK 64.8 million (? 8,7 million)), respectively. More information about DanStem at: http://danstem.ku.dk

About Medical Research Council Centre for Regenerative Medicine

The MRC Centre for Regenerative Medicine (CRM) is a world leading research centre based at the University of Edinburgh. Together we study stem cells, disease and tissue repair to advance human health. Our research is aimed at developing new treatments for major diseases including cancer, heart disease, diabetes, degenerative diseases such as multiple sclerosis and Parkinson's disease, and liver failure. http://www.crm.ed.ac.uk

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Synthetic protein amplifies genes needed for stem cells

Area woman’s disease puts focus on need for stem cell donations

Posted: Thursday, February 16, 2012 8:05 am | Updated: 2:27 pm, Thu Feb 16, 2012.

PITTSBURG — A brief cheek swab can wind up saving a life — and the life saved may be one of East Texas’ own.

Be The Match Registry will be camped out in the atrium of East Texas Medical Center in Pittsburg from 10 a.m. to 2 p.m. on Feb. 23 to take samples for the registry, which is just a sweep of the inside of a person’s cheek with a cotton swab. The organization nationally matches stem cell donors with those in need of a transplant.

Pittsburg resident Wanda Warrick has been diagnosed with Myelodysplastic Syndrome, a precursor to leukemia, and is in need of a stem cell transplant. She and her husband, Larry Warrick, joined up with Be The Match to help search for a donor.

He said the other way of looking at it would be if they wait too long, the disease could go further south and turn into leukemia, which is a cancer of the bone marrow.

Wanda Warrick was diagnosed five-and-a-half years ago with the disease and was told that within four or five years she would have to have a stem cell transplant. Larry Warrick said they’ve been lucky with the disease so far.

“Hers has been real slow to decline,” he said.

The couple went to visit a specialist in Seattle, where doctors started researching it before anyone else, he said.

“They do about 600 transplants a year,” Larry said. “(The doctor) goes, ‘10 years at the most, anybody would go with that. You’re one of the more fortunate ones.’ ”

Wanda Warrick is a former LVN, but Larry Warrick said the disease has taken that away from her, since one of the symptoms is a lessening of the body’s immune responses.

“She has a low platelet count,” he said. “If she’s cut, she bleeds easily. She gets sick easily. It took her out of the nursing field because she couldn’t make it through a full day’s shift.”

Now, Larry said, she can do some light housework in the morning, but fades around noon and has to nap.

The donor part of the transplant procedure is, thanks to recent technology, fairly simple — and free, thanks to Be The Match and the Warricks’ insurance. After the cheek swab, the donor is put on the list and checked against those in need of a transplant. After a hormone injection every day for five days, the donor is attached to a machine like a kidney dialysis machine, which circulates the blood through the machine, draws out the stem cells and then sends the blood back into the body through a needle in the other arm.

“Every once in a while, if they can’t get enough, they will do a bone marrow transplant,” Larry Warrick said. “Used to they had to do it from the bone marrow all the time.”

Wanda’s part of the transplant is harder. In order for her body to receive the new stem cells, the doctors have to kill off as much of her original bone marrow as possible. This is done through chemotherapy and radiation.

“There’s a 35 percent chance this could kill her,” Larry Warrick said. “It’s one of the most difficult decisions a person can make.”

By killing off all of the bone marrow, the body is defenseless against any disease, even the common cold, which could be fatal.

The new stem cells would then be injected into Wanda Warrick and allowed to take over her bone marrow. She would then be put on transplant medication anywhere from a few months to several years to the rest of her life, depending on how close the match is and how successful the transplant.

His wife has decided to go through with the surgery, he said, if a match can be found.

“She’s going to have it done in Dallas,” Larry Warrick said. “She’s going to wait until after next May so she can see at least one of her grandchildren graduate.”

He encourages everyone to come donate.

“They might be able to save someone’s life, maybe not Wanda’s, but someone’s, and that would make her happy,” he said.

Not a lot of people are aware of this, he said, but bone marrow or stem cell donation is racially specific.

“Whites donate for whites, Indians for Indians, Hispanics for Hispanics,” Warrick said. “There is a greater need for black, Hispanic and oriental, because they donate less than white people. That’s a need a lot of people don’t realize.”

He said he’s grateful that ETMC is letting Be The Match use their facility for the drive.

“I’d like to thank ETMC,” he said.

For those who cannot make the first registration drive, another registration will be held March 31 also at ETMC Pittsburg and will include Carter BloodCare taking blood donations as well.

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Area woman’s disease puts focus on need for stem cell donations