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.

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.

Study compares ‘alternative’ donor source vs. traditional transplantation of blood stem cells from an adult, unrelated donor

Using stem cells derived from the umbilical cord, researchers have improved the heart muscle and function of heart failure patients, paving the way for noninvasive therapies.

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.

The Bank operates under strict guidelines and protocols established by state and federal health organizations for quality assurance. This includes NYSDOH, FDA, FACT, AABB, CLIA, and CAP.  Regular inspections and testing are done to ensure that our facility remains a safe haven for banked cord blood.  Above all, this makes it possible to work with the regional hospitals and providers to enable mothers in the Central New York area the opportunity to donate their cord blood to the public bank.