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Cord Blood Connect 2024

Eliza Stroh / Stem Cell News

9/30/2024

The 2024 Cord Blood Connect International Congress took place September 6-8 in Miami, Florida. An annual international assembly focused on news, research, and emerging science in the field of newborn cells, physicians, researchers, and others from the stem cell community convened from nineteen countries to learn and share the latest in perinatal cell and tissue research. 

CBR® supports model for autologous cord tissue use in repairing alveolar clefts 

CBR’s Clinical Operations team presented a poster, “Feasibility of Harnessing Umbilical Cord Tissue for Alveolar Cleft Repair: A Collaboration between a Large Academic Institution and Private Family Cord Blood Bank.” The poster highlights previous research in collaboration with Children’s Hospital Los Angeles and the University of Southern California, in which human umbilical cord tissue was shown to promote bone fill in a mouse model of alveolar cleft.1 To facilitate future research whereby children with clefts may use their own cord tissue stem cells for cleft repair at ages 7-8, CBR provides no-cost storage of newborn cells for children with prenatal diagnoses of cleft lip and/or palate at Children’s Hospital Los Angeles. In an attempt to improve the current standard of care for repair of alveolar clefts, we aim to establish a pool of potential candidates for a future clinical trial through CBR’s Newborn Possibilities Program®

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Cord Blood Myth Busters led by CBR’s Peter Bawin 

CBR’s own Peter Bawin, Vice President of Global Marketing, led a panel discussion of experts who addressed some of the myths surrounding cord blood banking and utility. In response to the myth that many cord blood units lack enough cells to be used in treatments, Collen Delaney of Deverra Therapeutics pointed out that supplementation of cord blood units with bone marrow is now commonplace, and that hematopoietic stem cell expansion techniques are rapidly developing. Because we don’t know just what the future holds, families should understand the significance of the one-time opportunity to collect newborn cells. To combat the falsehood that cord blood treatments are declining, Roger Horton from Manchester, England’s Anthony Nolan Institute described the increase in cord blood usage in recent years in the United Kingdom. Fran Verter, PhD, founder of the Parent’s Guide to Cord Blood, reported that the number of ongoing and new clinical trials using perinatal cells and tissues continues to increase annually, and the number of advanced cell therapy trials has returned to pre-pandemic levels.2

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Increasing newborn screening for Krabbe disease 

Dr. Joanne Kurtzberg, Pediatric Bone Marrow Transplant Specialist at Duke University, and her team presented on efforts to extend newborn screening (NBS) for Krabbe disease to more states. Currently part of NBS in only 12 states, infantile Krabbe disease is a severe lysosomal storage disorder in which patients lack the galactocerebrosidase enzyme, leading to demyelination of neurons. Because allogeneic hematopoietic stem cell transplant within 45 days of life can extend the lifespan and quality of life for affected children through enzyme replacement, early diagnosis of Krabbe disease prior to onset of symptoms can be of critical importance. Through a 2-tier testing strategy that identifies decreased galactocerebrosidase activity and increased psychosine, Krabbe can be detected with both high sensitivity and few false positives.3 The Kurtzberg team continues to advocate for inclusion of Krabbe disease in increasing numbers of states’ NBS panels. 

Half-HLA-matched related donors yield successful transplant outcomes, younger donors are better 

Transplant physician Mahasweta Gooptu, MD, presented her team’s data from the Dana Farber Cancer Institute, comparing the results of high-risk allogeneic transplant recipients treated with bone marrow stem cells from haploidentical related donors vs. fully matched unrelated donors. While patients in each category who were treated with the same myeloablative preconditioning regimen and post-transplant graft-versus-host disease prophylaxis had similar rates of overall survival, a lower risk of relapse and better outcomes were correlated with younger donor age (i.e., less than 25 years old).4 This finding is significant not only because it demonstrates the success of transplants using haploidentical donors, but it highlights the importance of cord blood as a donor source given the very young age of donors. Future research will compare transplant outcomes between related haploidentical bone marrow donors vs. related haploidentical cord blood donors. 

Hematopoietic stem cell expansion techniques continue to improve: promising data from transplant recipients 

Multiple researchers presented progress in the field of hematopoietic stem cell expansion, a long-standing challenge whereby the cells are multiplied ex vivo prior to use in treatments. A key area of expansion research focuses on modification of epigenetic markers of stem cells, either by upregulating or degrading the molecules that turn key genes on and off. UM171, a hematopoietic stem cell agonist, has been shown to safely and effectively expand cord blood stem cells and to enhance multilineage blood cell reconstitution in patients with hematological malignancies.5 Furthermore, patients receiving transplants with UM171-expanded cord blood achieved long-term, durable remissions and lower nonrelapse mortality relative to recipients of unmanipulated cord blood units.6

Romania’s CORDUS study seeks to inform which patients with autism spectrum disorder are likely to benefit from cord blood infusions 

Felician Stancioiu, MD, from Medicover Hospital for Children in Bucharest presented his team’s data from the CORDUS study, in which 56 children with autism spectrum disorder (ASD) were treated with autologous cord blood between 2019-2023. Based on psychotherapist assessments, multiple standard ASD assessment scales, and parent interviews, cord blood infusions led to statistically significant improvements in nearly 79% of children between ages 3-7, but less so in children over 7 years old (data not yet published). Previous clinical trials investigating the use of cord blood as a potential therapy for ASD have not been successful at demonstrating clinically measurable improvements in all patients, which is likely due in part to the complex and multifactorial basis of non-genetic autism.7 While there is still great interest in the use of cell therapy as a possible treatment for ASD, researchers agree that we need better ways to assess which patients on the autism spectrum are more likely to respond to stem cell infusions.7 Toward this goal of better identifying these children, Dr. Stancioiu suggests that patients with lower levels of inflammatory markers and lower ferritin levels may be best suited to cell therapy.8 Future work will further define a model strategy for testing such markers as well as studying whether particular characteristics of cord blood units make them more likely for them to be effective. 

The 2024 Cord Blood Connect International Congress was a reminder of the importance of collaboration among the cord blood and cord tissue community. Advancements in cord blood and cord tissue therapies rely on a community dedicated to researching and spreading awareness of the amazing potential of newborn stem cells. To get involved in the community and become a cord blood advocate, join the CBR Healthcare Provider Network today. 

1. Stanton E, Feng J, Kondra K, et al. (2024). A Calvarial Defect Model to Investigate the Osteogenic Potential of Umbilical Cord Stem Cells in Bone Regeneration. Plast Reconstr Surg. Mar 1;153(3). 2. Verter F, Bersenev A, Couto P. (2024) 2024 Update: How Many Clinical Trials Employ Perinatal Sources of Stem Cells? Parent’s Guide to Cord Blood Banking. https://parentsguidecordblood.org/en/news/2024-update-how-many-clinical-trials-employ-perinatal-sources-stem-cells. Accessed September 13, 2024. 3. Matern D, Basheeruddin K, Klug TL, et al. (2024). Newborn Screening for Krabbe Disease: Status Quo and Recommendations for Improvements. Int J Neonatal Screen. Jan 28;10(1):10. 4. Shaffer BC, Gooptu M, DeFor TE, et al. (2024). Post-Transplant Cyclophosphamide-Based Graft-Versus-Host Disease Prophylaxis Attenuates Disparity in Outcomes Between Use of Matched or Mismatched Unrelated Donors. J Clin Oncol. Jul 17:JCO2400184. 5. Cohen S, Roy J, Lachance S, et al. (2020). Hematopoietic stem cell transplantation using single UM171-expanded cord blood: a single-arm, phase 1–2 safety and feasibility study. Lancet Haematol. 7(2):e134-e145. 6. Cohen S, Bambace N, Ahmad I, et al. (2023). Improved outcomes of UM171-expanded cord blood transplantation compared with other graft sources: real-world evidence. Blood Adv. Oct 10;7(19):5717-5726. 7. Sun, J.M. and Kurtzberg, J. (2021), Stem cell therapies in cerebral palsy and autism spectrum disorder. Dev Med Child Neurol, 63: 503-510. 8. Stancioiu F, Bogdan R, Dumitrescu R. (2023). Neuron-Specific Enolase (NSE) as a Biomarker for Autistic Spectrum Disease (ASD). Life (Basel). Aug 13;13(8):1736.  

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