We’re not investing enough in autism research

Having cared for children in my practice for over 25 years, I have found a grand disparity between how we profess to safeguard our children’s future and how we actually care for them. Cliches like “our children are our future” abound, but the simple fact is that, as a society, we are failing children who are most in need.

As a scientist and practitioner who has committed my life’s work to improving the lives of children, I recognized early the imminent rise of autism in the U.S. Through my work with these children, I saw that some answers for them may lie in their overall physiology, rather than simply developing drugs to treat their “brains.” My work led to the discovery of an enzyme deficiency, and ultimately our company Curemark was formed to further research this discovery. Our hope is that it could potentially lead to a paradigm-shifting approach to autism—and a drug to help treat it.

The rise in autism

The increase in autism’s prevalence has received a great deal of press of late. When I began exploring this discovery, the condition was thought to occur in 1 in 150 children. Now the CDC puts prevalence at 1 in 36. California’s recent data says autism is present in 1 in 22 children.

Autism spectrum disorders have a significant impact on the family unit and communities, including the school systems that supply the majority of care for children with autism from ages 3 to 21. The disorder’s lifelong persistence, high level of associated impairment and morbidity, and the absence of effective treatment place a major economic burden on multiple aspects of our social structure. The considerable costs of adult care and loss of productivity from parents have serious implications for the financial future of the affected families and potentially society in general.

Our local and state systems, including education infrastructure, are hard-pressed for resources to care for children with severe forms of autism. Once childhood is over, their work, living situations, and ongoing care becomes either the purview of the government or of families willing to pay exorbitant sums of money to house and care for them.

Despite autism’s massive increase in prevalence and the absence of a standard of care for its treatment, programs to facilitate treatment for these children still face significant obstacles.

Is autism seriously debilitating?

A few years ago, Curemark applied to one of the overseas regulatory bodies for inclusion in a program for accelerated drug approval for patients who were “seriously debilitated.” After filing, we were asked to present our clinical trials findings in very young children with autism in person. When that meeting began, the first question from the chairperson was, “Do you think that children with autism are ‘seriously debilitated’?”

Really? I said to myself incredulously, thinking of the children I’d cared for in private practice and how even mild autism affected their lives, especially in learning.

Trying to hide my horror at the question posed, I said, “Absolutely.”

I then explained that the medical definition of seriously debilitated is “unable to perform tasks of daily living,” which accurately described many of the young children with autism who presented in my practice. I continued discussing the debilitating nature of autism—especially severe autism—to the committee and the need for new and additional attention to a disorder that disrupts lives and, often, children’s development.

The chairperson said they were actually looking for treatments for conditions such as cancer, which they deemed to fit the category of “seriously debilitating.” In their opinion, neurodevelopmental conditions did not fit their criteria, even though the only criteria stated in the regulations were that the condition be seriously debilitating.

At that moment, two things became clear: Children with autism need someone to advocate for them, and a broad education about autism was needed beyond the affected families and caregivers.

Children versus adults

Another thing I discovered beyond that meeting was that with disease or illness, the medical and research communities pay far more attention to adults who lose function (as in dementia or Parkinson’s) than to children who fail to develop. As a result, neurodegenerative diseases appear to garner more attention and resources than neurodevelopmental diseases and conditions. The immediacy of the condition garnered more attention than that which fails to develop over a long period of time

I set out to test that hypothesis, and one of things I looked at was the amount of money spent on research in these areas by the National Institutes of Health (NIH). While there are private foundations and other philanthropic organizations that fund research into various areas of health, the NIH budget is public and current.

Unfortunately, I found that the federal government’s eyes have been opened to the surge in autism—but not its wallet. While the U.S. government has passed the Autism Cares Act (which is up for re-approval), providing for treatment and care for children with autism and some funding for research, it is clearly nowhere close to what is necessary—or what is spent on neurodegenerative diseases. In 2023, the NIH funded $3.7 billion in research for Alzheimer’s disease and related dementias. Its funding for autism research was only $306 million during that same time period.

We can’t give up

At Curemark, we screened over 1,000 children for our FDA-clinical trials as we worked to develop a treatment for autism. In the process, we saw the range of off-label prescription drugs and unproven medical therapies that parents have employed to help their children. It’s breathtaking. The kids we saw have been given off-label drugs ranging from those that treat cancer to those that treat epilepsy—even in the absence of a prior seizure disorder. Children with autism need a treatment designed for them, not one that simply repurposes drugs intended to treat other medical conditions.

We can’t give up on children who have not developed on the same schedule as neurotypical children. At the same time, we need to gain acceptance for individuals with autism whose neurodiversity is a talent and not a challenge. This requires a willingness to think out of the box and a real commitment—to research, to advocacy, and to ensuring that society keeps its promise to all children.

Joan Fallon is founder and CEO of Curemark.