Cord Blood Research & Clinical Trials
In the late 1980s, doctors began treating blood cancers and leukemia with hematopoietic stem cells found through cord blood research . Up until then, patients could only utilize bone marrow stem cells. There were many cases where bone marrow treatment failed or a bone marrow match could not be found. Researchers continue to explore new applications for the use of cord blood stem cells around the world. Today, over 80 diseases have been treated with cord blood stem cells because of ongoing cord blood research and clinical trials! The FDA regularly reviews the results of clinical trials for new treatments.
Researchers continue to conduct cutting-edge clinical trials in areas such as:
- Early hearing loss in infants
- Cerebral Palsy
- Cleft Pallets
- Congenital Pediatric Disorders
Featured Advanced Cell Therapy Trial: Expanded Access Cord Blood Therapy for Autism and Cerebral Palsy
Duke University Medical Center has received permission from the FDA to offer cord blood therapy for conditions like autism spectrum disorder and cerebral palsy under an expanded access clinical trial. This protocol establishes an umbrella clinical trial NCT03327467 registered on 31 Oct. 2017 which enables children who have these neurological disorders to receive therapy with their own cord blood or cord blood from a sibling, regardless of whether they qualify for a targeted clinical trial.
The registration of this clinical trial is a watershed moment, opening the door for many children who are afflicted with an acquired neurological disorder to travel to Duke University for cord blood therapy, provided they have a suffienctly matching cord blood unit in a family bank. Sibling therapy only requires a partial match, not a perfect match. This expanded access protocol is a triple win for patients, family cord blood banks, and Duke University Medical Center.
The FDA may grant permission for expedited access (aka expanded access) under one of four designation pathways. In order to be considered for the FDA's expedited access prorgram, the new therapy must treat a “serious condition” that that has substantial impact on day-to-day functioning. In addition, there must be an “unmet medical need” because the condition is not addressed adequately by therapeutic alternatives.
The Breakthrough Therapy designation for expedited access is based on the results of phase 2 clinical trials. Duke University has conducted multiple clinical trials investigating the use of both autologous and allogeneic umbilical cord blood (UCB) in the treatment of cerebral palsy (NCT01147653, NCT02599207) and autism spectrum disorder (NCT02847182). As stated in the new open access clinical trial, “The use of (UCB) in this fashion is based on safety and efficacy data from prior and ongoing clinical trials at Duke University Medical Center in over 700 patients with these diagnoses infused with autologous or sibling UCB over the past decade.”
In the United States, the Centers for Disease Control and Prevention reports that the prevalence of cerebral palsy is 1 in 323 children (0.3%) and the prevalence of autism spectrum disorder is 1 in 68 children (1.5%). However, in order to be eligible to participate in the new clinical trial, patients must have their own or a sibling’s cord blood preserved in a family bank. To date, the only study that has examined the prevalence of medical conditions among families with privately stored cord blood is a recent publication that surveyed clients of Cord Blood Registry® (CBR®). The authors found that, out of 94,803 respondent families, 4.23% reported at least one child with an indication for regenerative therapy with cord blood. For conditions similar to autism spectrum disorder and cerebral palsy, the combined prevalence was 2.18%.
Worldwide, there are projected to be tens of thousands of children who are eligible to take advantage of this new treatment pathway. In the United States alone, there are over a million cord blood units in family storage, so that if 2% of the inventory corresponds to children with eligible conditions, that potentially translates into 20,000 patients.
Breaking News: The Cord Blood Association (CBA) announces 13 Nov. 2017 they are seeking funds to convert this protocol into a multi-center clinical trial administered by CBA and Duke.
This article originally appeared on celltrials.org.
Breakthrough for babies with cleft palates as scientists claim umbilical cord blood could be used to repair the common facial defect
Researchers in Colombia have successfully trialled the surgery on nine children
They were able to grow new jaw bone after injecting stem cells into their faces
One girl, on whose case the report gives detail, managed to grow teeth normally
Blood from the umbilical cord could be used to repair cleft palates in babies, scientists claim. The new treatment - trialled on nine children in Colombia - could replace the need for bone grafts when children get older. Cleft palates, in which the skull has a gap on the face where the nose and mouth join up, affect around one in every 700 babies. Doctors now believe using stem cells taken from umbilical cord blood could reduce the number of operations affected babies need.
Cleft palates affect around one in 700 babies in the UK and are usually repaired with surgeries during childhood. Colombian researchers say they may be able to fix the deformity in a single procedure using stem cells from the baby's own umbilical cord. Researchers at the Hospital De San Jose in Bogota, Colombia, trialed the new surgery on nine children over the past 10 years. They said the operation showed good results in attempts to grow a new bone from scratch, to repair clefts. The authors wrote in the study: 'The potential regenerative power of the stem cell encouraged [us] to find new methods to be added to the classical surgical techniques and make possible better results [for] cleft patients.'
In a case study of one girl, they said she showed 'good thickness' of her jaw bone when she was followed up at the age of five after having the operation. The unnamed girl was diagnosed with the missing section of bone in an ultrasound scan while she was in the womb. Bone was missing from her upper jaw, where teeth should grow from when she got older. Immediately after her birth, blood was taken from the umbilical cord and frozen to be used later. Scientists took that specific blood because it is a rich source of stem cells – human cells which have the potential to grow into bone or other tissue. For the first few months of her life, the girl had the soft tissue in her jaw remoulded using a device similar to a dentist's retainer. When she was five months old, she had routine surgery to correct her cleft lip, which corrects the shape skin and flesh of the upper lip. At the same time, stem cells taken from the thawed umbilical cord blood were injected into the area where her jaw bone was missing. This was held in place with an absorbable pad and was left to allow the stem cells to grow. The procedure was successful and, in follow-up appointments, the girl had grown new bone where the stem cells were injected and had been left with normal teeth. Researchers said the operation's success meant the girl may not need any more surgery later in life – something they called a major advantage.
Current treatments for cleft palates can involve removing bone from elsewhere in the body, such as the hip, to then graft into the mouth. But bone graft surgery carries a risk of complications and it could be healthier for children to avoid it if they can, the doctors said. They called for more widespread studies on the stem cell treatment and said the patient would continue to be monitored in the future. Their findings were published in The Journal of Craniofacial Surgery.
Stem cells offer hope for autism
Gracie Gregory smiles beneath her brilliant blue eyes. She's sitting on her mother's lap, next to her older sister, Ryleigh, who boasts about Gracie being "very sweet and kind." It wasn't always so. Just a couple years ago, Ryleigh, 11, was scared of her sister when she'd throw tantrums and screaming fits. "She would've fought and kicked," Ryleigh says, noting that it wouldn't have been possible to sit like this next to Gracie. Why was she scared of her sister? "Because of the kicking." Gracie, 7, interrupts: "I don't even remember it." "We do," says her mother, Gina Gregory. Gracie has autism, a condition that affected nearly every aspect of her family's life after she was diagnosed at 2. But a new study is offering hope for the Gregorys and families like them. Gracie was one of 25 children who took part in the first-of-its-kind study at Duke University in Durham, North Carolina. The goal: to see whether a transfusion of their own umbilical cord blood containing rare stem cells could help treat their autism. The results were impressive: More than two-thirds of the children showed reported improvements.
A larger second trial is underway, one its researchers hope will lead to long-term treatment for children with autism. Skeptics say there are too many unanswered questions to get excited. Even Duke researchers acknowledge as much. The initial trial, published Wednesday in the journal Stem Cells Translational Medicine, was a safety study, not a controlled, double-blind study with definitive proof of positive results. This study was open-label, meaning everyone -- the doctors and the families -- knew that the therapy was being administered. But for the Gregorys, the change in their daughter has been monumental.
Updated 9:50 AM ET, Wed April 5, 2017
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Bringing opportunity to CNY!
At Upstate Cord Blood Bank we offer families the option to donate their cord blood for patients in need of a life-saving transplant. If the collected cord blood does not contain enough stem cells to meet the standards to be clinically useful to a patient, families can choose to donate the cord blood to researchers seeking to advance new treatments. Therefore, these invaluable stem cells will not go to waste! This is especially beneficial for studies seeking to advance new treatments for cancer and chronic diseases. This makes the Upstate Cord Blood Bank in Syracuse, New York a valuable resource for patients, physicians, transplant centers, and researchers across the US and around the world.