Treatment with stem cells from the umbilical cord
Autism and Cerebral Palsy
Treatment of Cerebral Palsy
CP is an abbreviation of the Latin name cerebral palsy. Palsy means weakness or problems with using the muscles. Cerebral means having to do with the brain. CP is caused by abnormal brain development or damage to the developing brain that appear in early childhood and affects a person´s ability to control his or her muscles permanently. Worldwide, the frequency is 2-3 cases/1000 live births1.
Behaviour problems are also common in children with CP, including neuropsychiatric disorders such as ADHD and autism spectrum disorders (ASD). There is no known cure for CP, however supportive treatments, medications, and surgery may help many individuals to reduce symptoms and disabilities while also improving mobility. This may include i.e. physical therapy and speech therapy.
Autism Spectrum Disorder
Autism Spectrum Disorder (ASD) are a group of developmental disabilities that can cause significant social, communication and behavioural challenges. CP is the result of brain damage that occurs before, during or shortly after birth whereas the etiology and pathogenesis of ASD are largely unknown although it is widely accepted that environmental, epigenetic, and inflammatory factors all contribute to the development of ADS. There are no medications that can cure ASD or treat the core symptoms. However, there are medications that can help some people with ASD function better.
Recent studies show that stem cell therapy might effectively cure CP. These advances have inspired the use of stem cell therapy in ASD (which is much more common). The evidence base is increasing that treatment with autologous umbilical cord stem cells can significantly improve the function of children with CP injury, and other neurological diseases, writes Boruczkowski et al., in an article published in 2019 2. The article is based on publications from PubMed and clinical trials registered at ClinicalTrials.gov and the purpose is to compile current knowledge and clinical use of umbilical cord blood. Several clinical studies also confirm the safety and efficacy of the use of umbilical cord blood (autologous and also allogeneic), including a meta-analysis of five clinical trials.3.
Dr. Joanne Kurtzberg
Joanne Kurtzberg is one of the most prominent researchers performing clinical trials with umbilical cord blood stem cells for the treatment of diseases and syndromes that affect the central nervous system 4. In 2010, Dr Kurtzberg conducted her first trial on the safety and feasibility of cord blood to treat acquired neurological disorders like cerebral palsy 5. Subsequent randomized, double-blind, placebo-controlled phase II study by Dr. Kurtzberg examined the effect of a single dose of autologous umbilical cord blood. The study from 2017 followed 63 children between ages of one and six years who had cerebral palsy because of brain damage occurred before or in connection with birth, had similar positive study results 6.
The participants were given a range of doses, from 10 million cells to 50 million cells per kg of body weight. One year after treatment, all participants who received umbilical cord blood showed improvement in motor function and brain activity. Children who received a higher dose of umbilical cord blood of at least 25 million cells per kg of body weight, showed greater improvements compared to children treated with a lower dose or placebo. This result is supported by two other randomized clinical trials within cell therapy, where they evaluated the use of cord blood stem cell infusion for the treatment of cerebral palsy in children 7,8.
Expanded Access Program
The first expanded access program (EAP) of cord blood stem cell therapy for CP launched 2017. Dr Joanne Kurtzberg is a part of this program and the Cord Blood Association annual meeting in September 2019, Dr Kurtzberg stated that to date over 320 children have received treatment through this program.
The FamiCord group started in 2019 another “Expanded Access Program” for the treatment of neurological diseases, including CP damage with umbilical cord blood (autologous) (Ref 2). The FamiCord group collaborates with over 1300 hospitals around Europe.
The term “Expanded Access Program” comes from the FDA (Food and Drug Administration) in the United States, where it is used to describe a program that gives patients with a serious illness or disorder access to experimental drugs that have been proven to be safe, but where the effect is still being studied and where there is no comparable or satisfactory alternative treatment.
To read more about the Expanded Access Program click here:
https://parentsguidecordblood.org/en/news/cerebral-palsy-expanded-access-program-europe
Here you can find registered clinical trials with umbilical cord blood for the treatment of CP injuries, which recruit patients:
Recruiting Trials: Cord Blood (parentsguidecordblood.org)
If you want more information about clinical studies, click on the link with the registration number of the clinical study you are interested in and you will proceed to the registration on the website of the clinical study: Home – ClinicalTrials.gov. The contact information can be found in the description of the clinical study.
References:
1 Arneson, C. L. et al. Prevalence of cerebral palsy: Autism and Developmental Disabilities Monitoring Network, three sites, United States, 2004. Disabil Health J 2, 45-48, doi:10.1016/j.dhjo.2008.08.001 (2009).
2 Boruczkowski, D., Pujal, J. M. & Zdolinska-Malinowska, I. Autologous cord blood in children with cerebral palsy: a review. Int J Mol Sci 20, doi:10.3390/ijms20102433 (2019).
3 Novak, I. et al. Concise Review: Stem Cell Interventions for People With Cerebral Palsy: Systematic Review With Meta-Analysis. Stem Cells Transl Med 5, 1014-1025, doi:10.5966/sctm.2015-0372 (2016).
4 Sun, J. M. & Kurtzberg, J. Cell therapy for diverse central nervous system disorders: inherited metabolic diseases and autism. Pediatr Res 83, 364-371, doi:10.1038/pr.2017.254 (2018).
5 Sun, J. et al. Differences in quality between privately and publicly banked umbilical cord blood units: a pilot study of autologous cord blood infusion in children with acquired neurologic disorders. Transfusion 50, 1980-1987, doi:10.1111/j.1537-2995.2010.02720.x (2010).
6 Sun, J. M. et al. Effect of Autologous Cord Blood Infusion on Motor Function and Brain Connectivity in Young Children with Cerebral Palsy: A Randomized, Placebo-Controlled Trial. Stem Cells Transl Med 6, 2071-2078, doi:10.1002/sctm.17-0102 (2017).
7 Min, K. et al. Umbilical cord blood therapy potentiated with erythropoietin for children with cerebral palsy: a double-blind, randomized, placebo-controlled trial. Stem Cells 31, 581-591, doi:10.1002/stem.1304 (2013).
8 Kang, M. et al. Involvement of Immune Responses in the Efficacy of Cord Blood Cell Therapy for Cerebral Palsy. Stem Cells Dev 24, 2259-2268, doi:10.1089/scd.2015.0074 (2015).