Duke University just concluded the first phase of their cord blood autism trial and the results look promising.
The researchers found significant improvements within the first 6 months following treatment. The improvements were also sustained at 12 months post treatment. The improvements were based on parent-reported measures as well as clinician assessments including the CGI-S, CGI-I, and EOWPVT, and objective eye gaze tracking measurements The greatest behavioral improvements were from children who had a higher nonverbal IQ improvement after the treatment.
The children were treated intravenously with their own (autologous) cord blood. All of the cord blood units were retrieved from private cord blood banks, except for one that came from a public cord blood bank. The one from a public cord blood bank is unusual, as there is no guarantee that a patient can ever retrieve the same (autologous) cord blood units that they donated to a public cord blood bank.
Why Did The Cord Blood Treatment Work?
A hypothesis is that the effector cells in the cord blood are having an effect on the immune modulation or regulation of neural connectivity in the brain via paracrine signaling, thereby altering brain connectivity and suppressing inflammation in the brain.
My own thought on what may be happening, is that the mesenchymal stem cells (MSCs) (although in small numbers in cord blood) are repairing parts of the brain that were damaged by an earlier immunological response.
Why Is Cord Blood Special?
Cord Blood is comprised of a large amount of hematopoietic stem cells (HSCs) and a small amount of mesenchymal stem cells (MSCs). MSCs are the stem cells that can turn into many different types of cells to repair tissues in the body by replacing damaged cells, including muscle, bone and brain cells as opposed to HSCs that can turn into all types of blood cells, including white blood cells, red blood cells, and platelets.
MSCs are the stem cells that all over the news now, due to their healing properties. MSCs in cord blood are exciting due to the fact that even small amount taken from cord blood can be grown into a much larger amount in the lab and then put back into the body to heal!
How Many Kids Were In The Trial?
The 25 children in the trial ranged from 2-5 years in age and all met the criteria for a clinical diagnosis of ASD based on the Diagnostic and Statistical Manual of Mental Disorders. Four of the children were female and 21 were male.
The upcoming Phase II will utilize allogenic (donor) as well as autologous (your own) cord blood. The allogenic cord blood could come exclusively from public cord blood banks and thereby will increase the size of the population that can benefit from its results.
The Phase I was conducted as an open label trial, meaning that the researchers and the participants knew that no placebo was being administered. An open label study can be highly biased, as everyone involved really wants the treatment to work. It is easy to understand how any parent would want to see positive results, especially after putting their young ones through the experience. The first phase was conducted as an open label, as the intent was to determine the safety of the treatment before moving onto the second phase.
Phase II will yield exciting, non-biased results as it will be double-blind and randomized, meaning that neither the researchers nor the participants will know who actually received the cord blood. Per the discussion section from the trial publication:
Accordingly, our next study will test the best available donor (autologous or allogeneic) versus placebo to lay the groundwork to extend access to this therapy for all affected children, if found to be effective.
It was disclosed by the team, that the lead author of the trial receives research funding from PerkinElmer, the corporate parent company of the ViaCord private blood bank.