Expert interviews Dr. Gary Goldstein
President, Kennedy Krieger Institute
Clinical Scientific Advisor, Autism Speaks
Dr. Gary Goldstein discusses the current focus of autism research and the urgent need to conduct long-range clinical trials.
Gary Goldstein, M.D., is a child neurologist and president of Baltimore’s Kennedy Krieger Institute. Kennedy Krieger’s Center for Autism and Related Disorders offers one of the nation’s most comprehensive programs for the diagnosis, research, and treatment of autism spectrum disorders. Dr. Goldstein is the clinical scientific advisor to Autism Speaks.
Why is it so important to focus energy and resources on autism, especially now?
Autism diagnoses are increasing rapidly and the disorder now affects an overwhelming number of children and their families. Today, the CDC says that as many as 1 in 166 children have autism. Ten years ago, the estimate was 1 in 2,500. No one’s sure whether the increase stems from increasing incidence, better diagnostic methods, or a combination of the two, but in any case, it’s obvious that many people are interested in this issue now and want something done to improve understanding and treatments.
What’s the current focus of autism research?
There are actually three areas that different groups are emphasizing now: genetics, environmental triggers, and brain imaging technology. When it comes to the causes of autism, here are the facts: we know it runs strongly in families, although it’s not strictly inherited like muscular dystrophy or hemophilia. But if you have one child with autism it is much more likely than in unaffected families for a second child to have autism. With twins, if they are identical, the chance of the second twin also having autism is very high. With fraternal twins, the risk is no higher than with any other sibling, which suggests a strong genetic component. In terms of which genes are involved, searches have identified a number of sites, so it’s not a single gene. The NIH working with private foundations has committed $25 million to fund a search for genes that make someone more susceptible to autism. This project has the potential to learn a lot more about the mechanism of autism, and to suggest new medical treatments.
Another topic the CDC has discussed is the exposures that may provoke autism. Both prenatal and postnatal exposures like diet or chemicals. This is worthwhile because if we could identify an exposure, we could learn to avoid allowing the exposure to occur. And prevention is ALWAYS better than treatment. Another area of interest is technology. With tools like functional magnetic resonance imaging (fMRI), we can see what’s going on in the brain, which regions are involved. Once we understand the brain activity involved, we should be able to design more effective drugs to combat the symptoms of autism.
What do you see as the greatest obstacle facing researchers wanting to study autism?
The greatest vacuum I see right now is the ability to perform clinical trials, to test new treatments against the old, to add different permutations and eventually see better results. In order to do this properly, you need to recruit thousands of subjects first to understand how they’d progress with the best current treatment. We will need multiple sites throughout the nation to collaborate in these studies. To understand the importance of clinical trials, you just need to look at what’s happened with leukemia. Thirty years ago, 95% of children diagnosed with leukemia died. Today, 80% are cured. Here’s what happened: all children diagnosed with leukemia entered into clinical trials. New therapies were combined with old therapies, remissions were prolonged. The children got the best available care, which led to cures for many. With an autism trial, we’d need to understand the progression of the diseases, and break the children into groups. One cohort of newborn siblings, and another of 3-year-olds, another of school-age children, another for adolescent intervention. We could combine early intervention with the young children, and different educational packages in older children with a variety of medications.
Why have there been no long-range clinical trials as of yet?
Money has been the stumbling block. It takes an enormous amount of money-in the hundreds of millions of dollars-to run these clinical trials. To run an effective long-range study, we need to enroll thousands of children. And it costs $1,000 to $2,000 per child per year. We need to organize test centers, develop forms, and develop protocols. There needs to be ongoing reliability testing. And the data needs to be managed, which is incredibly difficult and challenging.
What is being done to generate money for autism research?
The autism foundations have done a great job in raising awareness about autism and in raising money for research. The autism community is very committed and has been very successful on a number of levels. But there needs to be a huge national campaign directed toward the general public. Again, to use the example of childhood leukemia, look at the effect an organization like St. Jude’s has had on promoting research in this area. Hundreds of millions of dollars each year. This is why leukemia is now a curable disease. This is what Autism Speaks is capable of doing. They have the seed money to launch a general campaign. Again, this requires a lot of money—you need advertising, media, public relations. If Autism Speaks can raise the kind of money I believe it can, it will have a huge effect. As I said before, getting these clinical trials going is incredibly expensive. But these clinical trials are imperative—only then will we be able to compare results and develop appropriate treatments. Only then will we be able to find the answers we need. Another effect will be the ability to get money from drug companies. And, just as important, once there is sufficient money going toward autism research, we will be able to attract some very brilliant people into the field. So you see there is a ripple effect. Raising the money is the important thing.
What’s another area that requires more resources?
It’s absolutely necessary to have more trained professionals who can work with autistic children. Money raised by Autism Speaks could set up training programs throughout the country and attract more people to the field. With autism, the core deficit is learning social interactions. Treatments need to reinforce and teach social interaction, to reward appropriate behavior, since people learn that way. That’s what most parents do with their children, but children with autism need more than their own family can provide; they need one-on-one support 30-40 hours a week. Because these therapies are so intense, we require a lot of people to make them available to all the kids who need them. That requires a lot of training and a lot of money. There are so few people in the country who are qualified to work with autistic children, especially in certain communities.
What other kinds of treatments do you believe will come to the forefront in the coming months and years?
As we learn more about the brain mechanisms and genes underlying autism we will have new drugs to test and combine with behavioral and educational interventions. If we can modify and make the brain more receptive with medication, then behavioral intervention should be more effective. The same principles should apply to older children and adults.
Do you believe we can find a cure for autism?
I think you can compare it to the progress that’s been made in cancer research and treatment. At one point there was no cure for cancer. Now, there are subtypes of cancer that are curable and some that, so far, are not. We will most likely find subtypes of autism that will respond to treatment, and others that will take longer. The brain is so mysterious, and the disease is so baffling. It will be a slow process. But will we get there? Absolutely.
More With Gary Goldstein
Autistic people have fought the inclusion of ABA in therapy for us since before Autism Speaks, and other non-Autistic-led autism organizations, started lobbying legislation to get it covered by insurances and Medicaid.
ABA is a myth originally sold to parents that it would keep their Autistic child out of an institution. Today, parents are told that with early intervention therapy their child will either be less Autistic or no longer Autistic by elementary school, and can be mainstreamed in typical education classes. ABA is very expensive to pay out of pocket. Essentially, Autism Speaks has justified the big price tag up front will offset the overall burden on resources for an Autistic’s lifetime. The recommendation for this therapy is 40 hours a week for children and toddlers.
The original study that showed the success rate of ABA to be at 50% has never been replicated. In fact, the study of ABA by United States Department of Defense was denounced as a failure. Not just once, but multiple times. Simply stated: ABA doesn’t work. In study after repeated study: ABA (conversion therapy) doesn’t work.
What more recent studies do show: Autistics who experienced ABA therapy are at high risk to develop PTSD and other lifelong trauma-related conditions. Historically, the autism organizations promoting ABA as a cure or solution have silenced Autistic advocates’ opposition. ABA is also known as gay conversion therapy.
The ‘cure’ for Autistics not born yet is the prevention of birth.
The ‘cure’ is a choice to terminate a pregnancy based on ‘autism risk.’ The cure is abortion. This is the same ‘cure’ society has for Down Syndrome.
This is eugenics 2021. Instead of killing Autistics and disabled children in gas chambers or ‘mercy killings’ like in Aktion T4, it’ll happen at the doctor’s office, quietly, one Autistic baby at a time. Different approaches yes, but still eugenics and the extinction of an entire minority group of people.
Fact: You can’t cure Autistics from being Autistic.
Fact: You can’t recover an Autistic from being Autistic.
Fact: You can groom an Autistic to mask and hide their traits. Somewhat. … however, this comes at the expense of the Autistic child, promotes Autistic Burnout (this should not be confused with typical burnout, Autistic Burnout can kill Autistics), and places the Autistic child at high risk for PTSD and other lifelong trauma-related conditions.
[Note: Autism is NOT a disease, but a neurodevelopmental difference and disability.]
Fact: Vaccines Do Not Cause Autism.