Last week Novartis announced that, following a small clinical trial involving a few dozen patients, a new experimental drug had been found to bring about significant ‘improvements in the behaviors associated with retardation and autism in people with fragile X syndrome.’ According to the April 29th New York Times.
The Novartis trial, which began in 2008 in Europe with data analysis completed this year, was too brief to observe effects on basic intelligence. Instead, researchers measured a range of aberrant behaviors like hyperactivity, repetitive motions, social withdrawal and inappropriate speech. They gave one set of patients the drug and another a placebo, and after a few weeks switched treatments, with both doctors and patients unaware of which pill was which.
The results of the trial were something of a jumble until Novartis scientists noticed that patients who had a particular, undisclosed biological trait improved far more than others.
There have been a other reports of experimental drugs for treating Fragile X; the Novartis drug, it is reported, might also positively benefit autistic individuals who do not have Fragile X. (Go here for more information about autism and Fragile X Syndrome.) Said Katie Clapp, president and co-founder of the Fraxa Research Foundation, a nonprofit organization dedicated to financing fragile X research, and the mother of an 18-year-old son with Fragile X,
“This may be the key to solving the mystery of autism and other developmental disorders.”
The new drug, then, has the potential to stir up one of the thorniest and most divisive topics in discussions about autism, namely the question of a ‘cure for autism.’
Is a cure possible? Desirable?
Is autism something inborn and intrinsic to a person or is it something that a person can ‘grow out of’ or even ‘recover from’?
More and more autistic self-advocates, parents, and others speaking up about the strengths of being on the autusm’ spectrum. But others argue that, it’s fine for those who are ‘high-functioning‘ (a term that us itself the cause of much dispute) and who have Aspergers Syndrome to advocate for ‘neurodiversity‘ to not wish for a cure—but what a ‘cure’ about those who are (again, a disputed term) ‘low-functioning‘ and severely autistic?
Health and Human Services Secretary Kathleen Sebelius recently announced five new members of the Interagency Autism Coordinating Committee (IACC), a federal advisory committee that seeks to ‘accelerate progress in autism spectrum disorder (ASD) research and services.’ Among the new members are self-advocate Ari Ne’eman, the founding president of the Autistic Self Advocacy Network; the chief science officer for Autism Speaks, Geraldine Dawson, Ph.D.; the scientific director for the Simons Foundation, Gerald Fischbach, M.D.; the co-founder and board development chair of the Southwest Autism Research & Resource Center (SARRC) and the mother of an 18-year-old on the autism spectrum, Denise Resnik; and a member of the Faculty of the Medical Investigation of Neurological Disorders (MIND) Institute, Marjorie Solomon, Ph.D. The number of scientists and researchers appointed to the IACC outweighs the number of individuals who are themselves on the spectrum; as Autism Speaks’ mission statement says that it ‘dedicated to funding global biomedical research into the causes, prevention, treatments, and cure for autism,’ it seems that the question of a ‘cure’ might arise in some capacity.
I don’t know that there will ever be consensus about this issue. My own sense is that ‘cure’ isn’t really the right term to use. My son Charlie has certainly benefited from the numerous educational, therapeutic, and medical protocols that have been developed in the past several years. He has some speech (though it’s minimal, but he can talk); he’s learned to do a number of things—riding a bike, playing piano (at a very basic level)—that he might never have been able to; he is able to live with my husband Jim and me in our house in our community, despite my son having what I would refer to as ‘textbook autism nightmare without end” sorts of behavior challenges.
Certainly we always seek to find new ways to help him better communicate and learn, and to minimize his most difficult behaviors. But even if he could speak in full paragraphs, could read and write and do schoolwork at his grade level (8th grade), and had none of the serious behavior challenges that he does, Charlie would still not be what some refer to as ‘normal’ or ‘typical.’ He’d have (I would hope) fewer challenges at school and in general and his activities and opportunities might not have to be as limited as they are. But there’s something unique about Charlie to the core and whether or not he could be cured of that—-if he were, I’m not sure, would he still be Charlie?
Photo by Nancy Wombat.