A Breakdown of Duke Study Linking Autism and Stem Cell Therapy

Note from Team GR: Could stem cell therapy for autism become legal in the U.S. and Canada? A new Duke study investigates the safety and efficacy of stem cell treatment for patients with ASD. Our stem cell expert, Dr. Ernesto Gutierrez, breaks it down:

Written by Dr. Ernesto Gutierrez, M.D. 

I really enjoy being in the position where I’m currently at because I have a chance to share with others two things I’m very passionate about: health and regenerative medicine. Being able to share our experience directly with patients and parents of patients is tremendously rewarding and empowering for them which is probably why I like doing it so much!

Last month, the results from the Duke trial were published. They are very encouraging and optimistic about the future of stem cell therapeutics for the treatment of autism.

More than a few parents whom I’ve spoken to over the past few years have asked about the Duke trial. Some of them hoping to get included while others were waiting for the results to be published, well aware that it was only the first step in a series of studies which will have to take place before something like this is available for the general population.

But it’s really been in the last few weeks since the study was published that I started getting a lot of questions via our private Facebook group, our Facebook page , our contact forms , even Twitter and Instagram! Parents who contacted me want to know more about the study and the fact that they employed cord blood-derived stem cells.

Which is why I think this is a great time to share some of my views regarding this publication.

The researchers’ conclusion was:

“We demonstrated in an open label, phase I trial that intravenous infusion of autologous umbilical cord blood in young children with ASD is safe and feasible.”


So let’s break this down:

  • Open label: An open label trial refers to the type of trial where both the patient and the researchers know which treatment is being administered. This is in contrast to the single-blind (where the patient does not know whether he/she is receiving treatment or placebo) and double-blind trials (where neither the patient nor the researchers know who is being administered the treatment and who is receiving a placebo. This is the gold standard in clinical studies, but I doubt any parent would agree to submit their child for infusions of what could potentially be placebo.).
  • Phase I: This is the first type of trial a drug or treatment has to undergo and its primary goal is to assess efficacy and side effects. In other words, to make sure that it is safe to administer to people at the proposed dosage.
  • Intravenous infusion: the patients were treated via IV infusion alone.
  • Autologous umbilical cord blood: This is where it might get confusing. While the researchers employed stem cells from umbilical cord, they were autologous. Which means that each patient received their own banked stem cells; not cells from donors.

First of all, let me go ahead and say that I (as well as probably several of my peers) am super excited about the results of this study and what it means for the fields of regenerative medicine and of autism treatments.

The study they conducted was a Phase I study. Which means the researchers were evaluating SAFETY exclusively. Yes, they report clinical improvements as well but the study was DESIGNED to test the safety involved in administering iv stem cell infusions to patients within the spectrum.

Their results were very positive in this regard. And they will constitute the basis for future Phase II/III studies of stem cells and autism.

So now that I’ve broken down the basic results from the study, here’s my opinion on the matter: I think it’s great! Studies like this are laying the foundation for what has to be done in order for stem cell therapeutics to be available for everyone in the United States, Canada and Europe. Sure, we’re still many years away from seeing results of the Phase II/III trials and even more years away from the potential of receiving treatment in the US but it’s the right first step.

Unfortunately, for all of you reading this post, you probably need this to be available today, not in several years. Which is really the predicament most parents of autistic children currently have. Because of the way the FDA determines which treatments can and cannot be performed, stem cell therapeutics have been largely ignored in the United States and Canada for most of this century. However, this is not the case abroad.

In places like Mexico, the Caribbean, some countries in Europe and Asia stem cell therapeutics have continued to advance in many areas. In fact, most of the treatment centers abroad, –as is the case with our clinics– are backed by US-based preclinical research. Which means that clinics like ours do all of our lab-based research in the US (our laboratory is in Yorba Linda, California) and have treatment centers outside of the US (we currently have two clinics in Cancun and our group previously had another one in Guadalajara and in Ecuador) where we are able to serve the enormous number of patients who need treatment today.

So, in conclusion: I applaud the researchers behind the Duke study. This is the kind of research that needs to start happening for our industry to move the needle in the right direction. Cell-based therapies are the future of healthcare and we all need to demand progress.

What else would you like to know about this study and what it means for the autism community?

By the way, you can read the original publication here

About the Author: 

Ernesto Gutierrez’s training background includes an MD degree from Anáhuac University’s Faculty of Medicine –the most prestigious private medical school in Latin America– as well as training in the US and abroad achieving additional degrees in Age Management and Regenerative Medicine.

He currently serves as Chief Medical Officer for World Stem Cells Clinic and Rehealth, the premier destinations for autologous and allogeneic stem cell therapies in the world. His passion towards the development of novel therapeutic approaches to treat previously untreatable conditions has driven the clinic’s team to develop a unique, safe and highly effective Advanced Stem Cell Therapy for Autism which has already changed the lives of hundreds of autistic patients and their families.

Dr. Gutierrez is a Faculty Member for the Metabolic Medical Institute’s Stem Cell Fellowship as well as a lecturer at the American Academy of Antiaging Medicine (A4M), Generation Rescue, and the Medical Academy of Pediatric Special Needs (MAPS). He also regularly participates in peer-led interviews, podcasts, webinars and in person seminars aimed at patients and stem cell advocates.

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