By Dr. Gary Deel, Ph.D., JD
Faculty Director, School of Business, American Public University
In several recent articles, I told the story of my autistic son Carl and how I came to be involved in autism research. Autism is a psychological and behavioral impairment that appears to affect children — mostly boys — worldwide at an increasing rate. Yet the causes and dynamics of autism are still not well-understood.
Autism encompasses a range of mental conditions that primarily hinder the ability to communicate and navigate social interactions and relationships. The financial and emotional costs of autism on diagnosed individuals and their families can be crippling.
A study in JAMA Pediatrics found that the lifetime cost for an individual on the autism spectrum was $2.4 million on average when an intellectual disability is involved. Yet we still have not discovered a cure for this condition.
One Potential Treatment Receiving Increased Attention Is Stem Cell Medicine
But one potential avenue of treatment that has been receiving increased attention in recent years is stem cell medicine. Before getting into the modern science of stem cell research, it’s worth taking a moment to review some common misunderstandings and misconceptions regarding the use of stem cells.
About two decades ago, a controversial public debate began about the ethics of using embryonic stem cells — stem cells derived from aborted or miscarried human fetuses — for medical research. The controversy centered on the fear that creating a medical industry demand for embryonic stem cells would encourage abortions. There were also religious arguments about abortion and the irreverence and impiety of harvesting cells from human fetuses.
As a result, embryonic stem cell research in the U.S. was all but abandoned. Researchers later learned that embryonic stem cells tend to grow uncontrollably and cause tumors, which of course are not helpful in pursuit of new medical treatments.
Stacey Kram of APU’s School of Health Sciences notes, “However, the use of ‘induced pluripotent stem cells’ or iPSCs, which are adult cells genetically reprogrammed to act as embryonic stem cells, may have a solid place in modeling human diseases for research and drug development. Imagine being able to remove cells from someone with ASD, reboot them to the embryonic stem cell state, and then observe in a laboratory as they develop to gain further understanding into possible genetic causes of the disease.”
Research and Treatment Today Primarily Centers around Other Types of Stem Cells
Instead, research and treatment today primarily centers around other types of stem cells, which can be harvested using noncontroversial methods. For example, stem cells can be extracted from adipose (fatty) tissue and from bone marrow in living persons without any serious harm to the donor. Stem cells can also be collected from umbilical cord blood and tissue following a live birth. Because these materials would normally be thrown away after birth, there are no ethical concerns about their collection and use.
In 2008, a stem cell clinic in Panama City, Panama, began researching these different types of stem cells and using them to treat patients with a variety of conditions including arthritis, cerebral palsy, severe spinal cord injuries, heart failure, multiple sclerosis, and of course, autism.
The clinic was founded by Neil Riordan, an American doctor and researcher. It is still at work today. Riordan decided to work in Panama because U.S. laws and public policies made the environment here too restrictive and risky, especially following the stigma surrounding stem cells from the earlier embryonic stem cell debate.
I am not a medical doctor, so for specific details on the medical wisdom behind stem cell medicine I would refer readers to Dr. Riordan’s book, Stem Cell Therapy: A Rising Tide.
Some Types of Stem Cells Are Capable of Identifying and Traveling to Damaged Areas
However, the basic theory behind stem cell treatment is that, after intravenous injection, some types of stem cells are capable of identifying and traveling to damaged areas of the body. The stem cells then somehow direct a repair function in the cellular routines in order to heal the damage and normalize the area.
In his book, Riordan explains that his clinic has tested various kinds of stem cells, but he has found that the most effective cells are umbilical cord tissue stem cells, also known as mesenchymal stem cells. Fortuitously, these cells are also the easiest to harvest and use because they are not bio-markered, meaning there is no need to match blood or HLA protein types between donors and patients. Cord blood stem cells, on the other hand, must be donor-matched for use.
The exact mechanics of stem cell effects are not clearly understood. But Riordan shares numerous stories of patients with near-miraculous results. Quadriplegic patients regaining the ability to walk. Arthritic patients in debilitating pain experiencing near complete recovery. Severely autistic children with dramatic improvements in symptoms.
It’s Believed Some Kind of Inflammation or Miswiring of the Brain’s Connections Causes Autism
It is believed that some kind of inflammation or miswiring of the brain’s connections causes autism and its symptoms. So if stem cells are indeed able to repair these abnormalities, that would explain the connection between treatment and effect.
A few years ago, Duke University began to study stem cell treatment for children with cerebral palsy. These trials used autologous umbilical cord blood stem cells that had been frozen by families at the time of their children’s birth, in hopes that the cells might be of some medical benefit to their children in the future.
The cerebral palsy studies showed some positive effects. Some participants experienced a reduction in symptom severity. However, interestingly, some of the children in these early trials had also been diagnosed with autism, and in addition to helping with cerebral palsy, the researchers noted that the treatments improved autism symptoms as well.
So in 2016, Duke began a concurrent set of studies to test the effects of cord blood stem cells on children diagnosed only with autism. In 2017, the research team published its Phase I results indicating that the treatment was completely safe; in other words, there were no serious adverse effects associated with the stem cell injections. Furthermore, although the sample size was very small (just 25 children), some of them showed improvement in autism symptoms.
Following the Phase I results, the Food and Drug Administration (FDA) gave Duke approval to continue with Phase II trials to test efficacy more thoroughly. They also gave the university permission to begin what is called an extended access protocol (EAP). That allows families with autistic children, who have preserved a portion of their child’s umbilical cord blood, to visit Duke and receive the same treatment outside the scope of the clinical trials.
In the summer of 2018, my wife and I took our son Carl to Duke for the treatment. I’m often asked, “Did it work?” My response admittedly is a bit complex. What I can say for sure is that my son is making good progress. He continues to improve every day. But he is also receiving daily Applied Behavior Analysis (ABA) therapy and other treatments.
So is his progress the result of the stem cell injections or of other factors? With a sample size of n=1, we’ll never know.
What I do know is that stem cell therapy is an area of worthwhile research in autism treatment. But it needs public recognition and support in order to grow and produce real answers. It is my hope that readers will support these efforts at the Duke Center for Autism and Brain Development and other relevant organizations, including Autism Speaks.
About the Author
Dr. Gary Deel is a Faculty Director with the School of Business at American Public University. He holds a JD in Law and a Ph.D. in Hospitality/Business Management. He teaches human resources and employment law classes for American Public University, the University of Central Florida, Colorado State University and others.