A ground-breaking study on a new experimental pill for people with Autism Spectrum Disorder (ASD) is currently underway in Toronto, but the community remains divided on its use.
Drug manufacturer Hoffmann-La Roche has commissioned an international research study for Balovaptan, a drug that could potentially help people on the spectrum manage everyday social and communication challenges better.
“It’s regulating hormones in our brain that have to do with how we perceive the social world, whether we understand social cues, how we relate to other people, and how we develop close bonds and relationships,” said Dr. Evdokia Anagnostou, the study’s lead researcher at Toronto’s Holland-Bloorview Kids Rehabilitation Hospital.
“The idea would be, people who have difficulty with everyday social functions, may have one more potential option for thinking of interventions, if they choose to use them.”
One of the Canadian sites participating in the study is the Autism Research Center at Holland-Bloorview, which launched the 40-week clinical trial to determine whether or not the drug can be effective. Dr. Anagnostou and her team began enrolling participants 18 years of age and older this past summer – half of them will be given the drug, while the other half will take a placebo.
Dr. Anagnostou, who is a Senior Clinician Scientist at the hospital, said participants in the study must undergo a process to determine whether or not they are the right fit. They speak with a physician to discuss their history, express their priorities, and get an understanding of the study, including the risks and benefits. Once approved, the patients take a daily dose of the medication, and are regularly monitored by staff.
“Although we know quite a bit about its safety, we don’t have a final decision on it, so we monitor people closely,” said Dr. Anagnostou. “They come and visit us every couple of weeks, they have blood work, they speak with the physician and psychologist.”
Once the participants have completed their six-month trial, they’ll have the option of continuing to take the drug for up to two years, in order to make the process more equitable, Dr. Anagnostou explains. That would allow researchers to collect more data in that time period, and allow an opportunity to try the medication for everyone who agreed to participate in the study.
Division in the community
The Autism community remains divided on the drug, as one disability rights group in Ontario has raised concerns around whether or not the pill is ethical.
Autistics 4 Autistics Ontario (A4A) is a self-advocacy group for adults with autism, calling for reform to autism funding and services at the federal and provincial levels. Anne Borden, a member of the province’s A4A executive board, tells CityNews the organization opposes the drug and the research, saying it could prey upon people’s hopes and dreams.
“Who is this benefiting?” Borden asks. “This kind of research represents a very old way of approaching autism, looking at autistic people like they’re a problem to be solved or sort of a broken version of normal, rather than taking a position of acceptance.”
Borden said developing a pill for this purpose speaks to the dignity of people with autism, and can negatively affect how they are treated and perceived. She called this a “profit-generating” pill that may put “people from a vulnerable population” at risk, adding that there are ethical questions rooted in its very existence.
“There’s a deep psychological impact when all the people in your life are constantly trying to fix you for who you are and, in this case, gives you a pill to make you act differently,” Borden said.
“It’s not looking at communication as a two-way street. Whereas other ideas like inclusion, accessibility, communication, dialogue, and research into the access needs of autistic people will do that.”
Dr. Anagnostou, who also works in the Department of Pediatrics at the University of Toronto, is aware of the criticism, but said there’s already a wide variety of work being done to understand the needs of adults, and how to produce an equitable environment for them. She notes that it’s important to give adults with ASD the same options as others who have neurological conditions.
In fact, not allowing these treatments to be developed, she argues, would be a questionably ethical decision.
“I would say that the ethical thing to do is to provide people with options about how to improve their well-being and their functioning, and autism isn’t a place where everybody has the same opinion of their condition,” she said.
“There are lots of people with autism who are actually asking for medications. Our position is that it is our responsibility to develop options and help patients make decisions that are appropriate to them, their families and their whole value systems.”
The research, she adds, has also been well-endorsed by families and advocacy groups. In recent years, an exercise funded by the Ontario Brain Institute led a study on what priorities were important for people living with neurodevelopmental disorders, as well as their families, caregivers, and health and education professionals.
Over 250 people participated in the surveys, identifying a list of 10 areas that they wanted researchers to focus on. Among the defined areas, respondents said they wanted researchers to develop interventions that would help improve their social skills, and develop and maintain social relationships.
“Autism Spectrum Disorder is defined by some difficulties in social functions and understanding of the social world. Not everyone experiences distress by it but a lot of people do,” Dr. Anagnostou said. “For people who do experience distress from not having the set of tools that are making navigating the social world easier, we want to make it easier.”
One of the groups that participated in the exercise is Autism Ontario, which represents families across the province. The group said these clinical trials are beneficial to advance research in the field and understanding what drugs and interventions are helpful are a defined priority for adults impacted by various neurodevelopmental conditions.
“The process that’s involved in conducting this clinical trial has gone through an ethics review,” said Margaret Spoelstra, the Executive Director of Autism Ontario. “When we do clinical trials, we can really pay attention to some of those variables that may have an impact on our consideration on future drugs.”
Borden also expresses concern that the drug will be used on people who don’t have the capacity to give informed consent and it would be left in the hands of their primary guardians, most likely their parents.
“Is it really going to help you connect with your child as they are for who they are, in a way where you really have a connection,” she said. “Or is it a magic pill that isn’t really going to bring you any closer or improve the life of your child. That’s the question I think all parents need to be asking themselves.”
Spoelstra, who supports the work Dr. Anagnostou and her team are doing at Holland-Bloorview, said questions around consent are important so that people can have access to details around how the drug would work and what risks it would come with.
“All of that information will need to be made available in a way that’s understandable by the person taking the drugs, by their families who support them and by any clinicians who are involved,” she said. “If there are any challenges, there’s a mechanism that those can be recorded.”
Hope for ground-breaking research
There are at least two streams of clinical trials currently underway in several countries, including in Canada.
The first is taking place at nearly a dozen international sites, involving children with ASD between the ages of two and four. The other focuses on adults with ASD at 55 study locations, including in British Columbia, Quebec and Ontario. There are 350 participants in the phase three clinical trial that is expected to be completed by March 31, 2023.
Over the years, concern has been raised over the lack of resources and funding available to adults with autism, and not many support for those who transition to adulthood. Dr. Anagnostou says this research is an opportunity to think about interventions for adults and developing solutions that are accessible for them.
“Adults are not big children. Their bodies and brains change with time and their priorities and wishes change with times,” Dr. Anagnostou said. “It’s important to think about interventions in adults, by thinking about what adults want and what their biologies look like.”
Though Dr. Anagnostou said they won’t have the results for another couple of years and adds the research itself is unique because there haven’t been any success stories of its kind, despite numerous efforts that would allow those who want it, to live “more meaningful lives in the way they choose to.”
“There has been no medication approved for the treatment or the assistance with social deficits in ASD,” said Dr. Anagnostou. “We haven’t been successful yet. This is part of a larger set of research programs that tries to find solutions for people who want to have help with social skills in adulthood.”
There are four phases to a clinical trial, as explained by Dr. Anagnostou, who also adds that this one compound has come the furthest among anything else they have. However, she states it’s important to acknowledge that this drug is years away from hitting the market. And there is a possibility that, at the end, results may indicate that it isn’t effective after all.
“My hope is that we create a suite of potential interventions, from medicines to psycho-social interventions, technologies, and environmental modifications to improve the lives of people with ASD,” she said. “That are accessible so people can get them when they need them and when they want them.”