In Brief: The work of Dr. Joanne Kurtzberg and her colleagues at Duke University aims to bring novel cord blood and cord tissue-based cellular therapies to the clinic. Per the license agreement, additional patients will be provided access to cord blood and cord tissue treatments under Investigational New Drug Applications (INDs) currently held by Duke at a forthcoming Cryo-Cell stem cell infusion clinic, expected to open in early 2022. Additionally, Cryo-Cell has been granted exclusive commercial rights to Duke’s intellectual property assets, FDA regulatory data, clinical expertise, and manufacturing protocols associated with various applications of cord blood and cord tissue stem cells.
Access to Innovative Treatments
An exclusive collaborative license agreement with Duke University grants Cryo-Cell the rights to proprietary processes and regulatory data related to cord blood and cord tissue developed at The Marcus Center for Cellular Cures at Duke. Furthermore, Cryo-Cell has been granted exclusive commercial rights to Duke’s clinical expertise and manufacturing protocols associated with various applications of cord blood and cord tissue stem and progenitor cells. Under this agreement, Cryo-Cell plans to explore, test, and administer umbilical blood and tissue derived cell treatments to patients with conditions for which there are limited FDA-approved therapies, including cerebral palsy, autism, and other neurological conditions. These treatments utilize the unique immunomodulatory and potential regenerative properties derived from cord blood and cord tissue cells.
Expanded Access Program
Since 2017, Duke’s Expanded Access Program (EAP) has enabled hundreds of families who have stored their children’s cord blood to take advantage of investigational therapies offered at the Marcus Center for Cellular Cures at Duke University.1 Children with various brain disorders, such as autism, apraxia, cerebral palsy, congenital hydrocephalus, hypoxic brain injury, and stroke, are eligible to participate. Dr. Joanne Kurtzberg, Director of the Marcus Center for Cellular Cures at Duke University and Cryo-Cell’s Medical Director, reported over 400 patients had been treated as of December 2020. Additionally, there are a reported 600+ screening patients and another 1,600 patients awaiting follow-up contact.2 The demand for those therapies continues to overwhelm the limited capacity available, with a waitlist several months long. These experimental treatments call for stringent selection criteria, including the number of cells required for a child’s infusion per body weight. Since children grow quickly, it is critically important to provide access to therapy before the child outgrows the number of cells available for the treatment. Licensing of Duke’s EAP treatment protocols allows Cryo-Cell to offer the same high-quality care they come to expect from an FDA-sponsored program at one of our forthcoming clinic locations.
Over the last several years, Dr. Joanne Kurtzberg and her team have dedicated a significant amount of research concerning autism and the use of cord blood for treatment. An initial study involving the treatment of 25 children ages 2-6 determined the safety and efficacy of cord blood cells in treating Autism Spectrum Disorder (ASD). Then, a Phase II randomized, double-blind study (DukeACT) of a single intravenous autologous or allogeneic, unrelated cord blood (CB) infusion in children ages 2-7 years with ASD was performed among 180 participants. The results of both published studies revealed cognitive improvement in a number of children treated with autologous and allogeneic cord blood infusion.3,4 In addition to cord blood, Dr. Kurtzberg and her team are also studying the effects of umbilical cord tissue mesenchymal stem cells in treating autism. A Phase I study was completed in treating 12 children with a dose of 2-6 million cells/kg. 58% of patients showed improvement in at least 2 out of 3 tested measures of improvement.5 These results led to the current development of a Phase II study (IMPACT) among 164-300 children ages 4-11 years.6 This clinical trial is actively recruiting participants and is expected to take roughly two years to complete.
After demonstrated safety, a Phase II published study of 63 children ages 1-6 with cerebral palsy (CP) showed that an autologous cord blood dose of 25 million nucleated cells/kg provided treated children with a significant improvement in motor function one year after infusion and was accompanied by new motor track development in the brain. As Dr. Joanne Kurtzberg et al. report in the study, “Important relationships were also detected via whole brain connectome analysis of MRI/DTI data, an objective measure of whole-brain connectivity including the motor network, suggesting that improvements in motor function result from increased or new connectivity induced by paracrine signaling of autologous cord blood stem cells. [They]confirmed that increased total brain connectivity is correlated with increased motor improvement, as reported by their previous studies.” Furthermore, results also showed that compared with children who received a lower cell dose, children who received a higher cell dose demonstrated improvement in motor function with an increase in both normalized total brain connectivity and changes in the sensorimotor network.7 A similar study was done to test the safety and efficacy of treating children with CP using allogeneic umbilical cord blood sourced from a sibling for infusion. Clinical results demonstrated the same clinical improvement as the children treated with autologous cord blood.8
Dr. Kurtzberg and her colleagues have also conducted research in infants with hypoxic-ischemic encephalopathy (HIE), a neurological condition that can be fatal. These studies involved up to four intravenous infusions of autologous cord blood stem cells cord blood infusion coupled with the standard of care, which is cooling. A Phase I study indicated that, of the 23 enrolled infants, nearly twice as many infants survived with normal function at one year with autologous cord blood infusion and cooling compared to babies with cooling alone.9 Other neurological conditions such as hydrocephalus, speech apraxia, anoxia by near-drowning, and stroke are also treated under the Expanded Access Protocol at Duke.
Extended Benefits for Patients
Patients can benefit from access to treatment in clinical trials, which are currently under an FDA-approved IND to treat indications such as autism, cerebral palsy, and other brain injuries. The same access to treatments currently offered to patients under Duke’s FDA Expanded Access Program will also be available at one of our anticipated clinic locations. Cryo-Cell is dedicated to aiding the advancement of regenerative medicine through forward-thinking products and services. Dr. Joanne Kurtzberg and her scientific team at the Marcus Center for Cellular Cures at Duke University, which is internationally recognized as the leading research and clinical center using cord blood and tissue cells for immunomodulatory and regenerative medicine applications, is a pioneer in cell therapies based on umbilical cord blood. Through this exclusive license agreement, Cryo-Cell intends to benefit patients by expanding into a triad of core business units:
1.) its renowned cord blood bank,
2.) biopharmaceutical manufacturing (once BLA(s) or Emergency Use Authorization(s) are approved by the FDA), and
3.) infusion clinic(s) services, initially under Duke's IND utilizing the FDA's Expanded Access Program.
“This is a major step forward in our growth at Cryo-Cell, and we believe that it will be a gigantic leap forward for patients with unmet medical needs waiting for treatment. Since 2011, Cryo-Cell’s mission statement has included the goal of advancing regenerative medicine. Now, we will be able to truly fulfill our vision.”
- David Portnoy, Chairman of the Board and Co-CEO
2. Kurtzberg, Joanne. (2020). Online Presentation. Clinical Trial Update: Cord Blood for Autism, Cerebral Palsy, Stroke & Cord Tissue for MIS-C. World Cord Blood Day 2020 Presentation. Retrieved November 17, 2020, from https://www.worldcordbloodday.org/cordblood-questions-answers-2020.html
3. Dawson, G., Sun, J., Davlantis, K., Murias, M., Franz, L., Troy, J., . . . Kurtzberg, J. (2017, April 05). Autologous cord blood infusions are safe and feasible in young children with Autism Spectrum Disorder: Results of A Single Center Phase I Open Label Trial. Retrieved March 10, 2021, from https://stemcellsjournals.onlinelibrary.wiley.com/doi/pdf/10.1002/sctm.16-0474
5. Sun JM;Dawson G;Franz L;Howard J;McLaughlin C;Kistler B;Waters-Pick B;Meadows N;Troy J;Kurtzberg J;. (n.d.). Infusion of human umbilical cord tissue mesenchymal stromal cells in children with autism spectrum disorder. Retrieved March 10, 2021, from https://pubmed.ncbi.nlm.nih.gov/32531111/
7. Sun, J., Song, A., Case, L., Mikati, M., Gustafson, K., Simmons, R., . . . Kurtzberg, J. (2017, December). Effect of autologous cord blood infusion on motor function and brain connectivity in young children with cerebral palsy: A randomized, placebo-controlled trial. Retrieved March 10, 2021, from https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5702515/
9. Cotten CM;Murtha AP;Goldberg RN;Grotegut CA;Smith PB;Goldstein RF;Fisher KA;Gustafson KE;Waters-Pick B;Swamy GK;Rattray B;Tan S;Kurtzberg J;. (n.d.). Feasibility of autologous cord blood cells for infants with hypoxic-ischemic encephalopathy. Retrieved March 10, 2021, from https://pubmed.ncbi.nlm.nih.gov/24388332/