Can Cord Blood and Tissue Stem Cells Treat Autism?

Autism Spectrum Disorder (ASD) is a complex neurological and developmental condition that is characterized by challenges with social interactions, speech and nonverbal communications, restricted interests and repetitive behaviors. Autism is considered to be a ‘spectrum’ disorder because the type and severity of symptoms can vary widely in people diagnosed with ASD.

Approximately 1 in 44 children have been identified with ASD. Boys (1 in 27) are four times more likely than girls (1 in 116) to be diagnosed with autism. Autism affects all ethnic and socio-economic groups.

Diagnosing ASD can often be difficult as there are no medical tests such as blood tests to diagnose the disorder. Doctors look at the child’s developmental history and behavior to make a diagnosis.

ASD can be detected as early as 18 months but by age 2, a diagnosis by an experienced professional can be considered reliable¹. Early diagnosis and intervention are the best chances to ensure that children receive the treatments and therapies they need to reach their full potential.

Developmental monitoring is an important tool in the diagnosis of ASD. This involves parents and/or child care providers observing whether the child meets the typical developmental milestones in an age appropriate manner.

The CDC has developed the Learn the Signs. Act Early. program to track and share milestones through various resources including their Milestone Tracker app. Developmental screening is performed by a pediatrician during routine well-child visits and the American Academy of Pediatrics recommends developmental and behavioral screening at 9, 18, and 30 months and ASD specific screens at 18 and 24 months.

While there is no cure for autism, there are treatments and therapies that can lessen the symptoms of ASD that can interfere with daily functioning and quality of life.² Because of the ‘spectrum’ nature of the disorder, there is no single treatment that will be more successful than any other treatment. Therefore, treatment plans will generally need to involve various healthcare professionals that are focused on the individual.

There are a number of ongoing and completed clinical trials for autism using stem cells derived from cord blood and/or cord tissue. However, it is important to understand that these clinical trials are not looking for a “cure” but are designed to relieve or lessen the effects of autism to a degree that other methods of treatment cannot achieve.

Study #1: Autologous Cord Blood Stem Cell Infusion

In a single-center, phase I, open-label trial, 25 children ages 2-6 with a confirmed diagnosis of ASD were enrolled in this study. Each child received an intravenous infusion of 10 to 50 million cells per kg body weight. The stem cell infusions were well tolerated with no reports of serious adverse events. Improvements in behavior were observed in the first 6 months after infusion and sustained at 12 months. It was noted that higher baseline nonverbal IQ was associated with a greater degree of improvement. This study also identified potential outcome measures that are feasible, sensitive enough to detect changes and developmentally appropriate for use with young children with ASD.

Key Takeaway: This study demonstrated that intravenous infusion of cord blood stem cells ranging from 10 to 50 million cells per kg body weight in children with ASD is safe and effective in a limited sample size. This study identified potential outcome measures suitable to detect changes in young children with ASD.

Study #2: Autologous Cord Blood Stem Cell Infusion

In a pilot randomized, double-blinded, placebo-controlled, cross-over trial, 29 children ages 2 to 6 with a confirmed diagnosis of ASD were enrolled in this study. The children were randomized to a treatment (minimum of 10 million cells per kg body weight) or placebo infusion group and at 24 weeks, the children received the opposite dose. There were no serious adverse events reported at the 12 and 24 week time points after stem cell infusion. The clinical researchers noted a trend towards improvement, particularly in socialization, but not statistically significant differences at the 12 and 24 week timepoints.

Key Takeaway: This study demonstrated that intravenous infusion of 10 million cells per kg body weight was safe with a trend towards improvement in socialization in a limited sample size.

Study #3: Autologous / Allogeneic Cord Blood Stem Cell Infusion

In a prospective, randomized, placebo-controlled, double blind study, 180 children ages 2 to 7 with a confirmed diagnosis of ASD were randomized to the autologous (n=56), allogeneic (n=63), or placebo (n=61) groups. The autologous and allogeneic groups received a single intravenous infusion of a minimum of 25 million cells per kg body weight. The stem cell infusion was found to be safe and well tolerated. However, the stem cell infusion did not meet the primary (improved socialization skills) or secondary endpoints (reduction in autism symptoms) for efficacy.

The clinical researchers performed a deeper dive into the study results to identify key challenges that may have impacted the study results. Some of the factors impacting study design included ages of the participants and their ability to be assessed, average dose of stem cells infused in the autologous vs allogeneic groups, time points for assessment and the assessment tools. However, the clinical researchers found that the stem cell infusions did demonstrate a benefit vs the placebo group for the subset of children who were 4 to 7 years of age without an intellectual disability. The threshold for intellectual disability is a non-verbal IQ score of 70.

Key Takeaway: This study demonstrated that autologous and allogeneic intravenous stem cell infusions were well tolerated. However, the stem cell infusions did not demonstrate an improvement in socialization skills or reduced autism symptoms as per the study hypothesis. Based on an analysis of a subset of children participating in this study, children ages 4 to 7 with a non-verbal IQ of 70 may benefit from stem cell infusion. Further clinical research is needed to provide more definitive evidence but this study shows a potential for benefit in a specific population of children.

Study #4: Allogeneic Umbilical Cord Tissue Mesenchymal Stem Cell Infusion

In an open-label, phase I study, 12 children ages 4 to 9 were treated with an infusion of cord tissue mesenchymal stem cells from an unrelated donor. The children received one, two or three doses of 2 million cells per kg body weight at 2 month intervals. The infusion of stem cells was well-tolerated in this limited sample size. Six of the 12 children showed improvements in at least two ASD-specific outcome measures. However, safety and efficacy will be more fully evaluated in an on-going phase II clinical trial (ClinicalTrials.gov NCT04089579).

Key Takeaway: The study demonstrated that cord tissue mesenchymal stem cells were well tolerated in this limited sample size. However, safety and efficacy will be more fully evaluated in an on-going phase II clinical trial.

References:

• Lord C, Risi S, DiLavore PS, Shulman C, Thurm A, Pickles A. Autism from 2 to 9 years of age. Arch Gen Psychiatry. 2006 Jun;63(6):694-701.
• Hyman, S.L., Levy, S.E., Myers, S.M., & AAP Council on Children with Disabilities, Section on developmental and behavioral pediatrics. (2020). Identification, evaluation, and management of children with autism spectrum disorder. Pediatrics, 145(1), e20193447.

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