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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.