[music] [applause] dawson: there’s a saying that if you’vemet one person with autism, you’ve met one person with autism. and that’s because autism is a spectrumdisorder; it affects people in many different ways. but in every case, that person has difficultiesunderstanding and navigating the social world. they have trouble making eye contact, readingfacial expressions and gestures, forming social relationships.
now, we know that autism begins in the prenatalperiod and we can actually diagnose autism when a child is 18 months of age. and yet the average age of diagnosis in theunited states is close to five years. and if you’re a child from an african-americanbackground, it’s going to be much older. and what that means is that we’re missingvery important years when the brain is rapidly developing, it’s plastic and it would bemost responsive to early intervention. now, we know that genes set the stage. they—they do contribute to autism in termsof its cause, but it’s actually experiences that shape the circuits in the brain.
a baby’s brain will have twice as many connectionsbetween brain cells as it will have when it’s adult. and when it interacts with the environment,those connections, those synapses that are used will be retained, and those that arenot used will die away. and in this way, our experiences actuallyshape the circuits in our brain. now, this synaptic pruning process is disruptedin autism. and that’s because a baby with autism ispaying attention to the world in a very different way. this is a picture of a one-year-old who laterdeveloped autism, and it’s at his first
birthday party. people are singing to him, they’re makingeye contact, they’re smiling, and yet that baby is paying attention to the cake and notthe people around him. compare this to a typical baby, the same scene,and this baby is noticing all of those people around him, and engaging, and what this meansis that the young infant with autism is not receiving the normal kind of stimulation tothe areas of the brain that are important for language development and social developmentduring this early period when the brain is developing. now at duke, our goal is to identify infantsthat are at risk for autism even before those
symptoms emerge. and we’re doing this by looking for whatwe call biomarkers. so these are biological changes that we canmeasure. some of them we’re measuring in the blood,such as changes related to the immune system. we look in—at patterns of expression ofgenes. we can also look at the structure of the brainthrough mri. or in this study, we used electrophysiologyto measure the dynamic activity of the brain, those neurons, and we found that by eightmonths of age, a baby who is going to go on to develop autism is already showing a differentpattern of functional connectivity between
different parts of the brain. particularly the frontal lobe. and we know that the frontal lobe is veryimportant for social and language development. now, we were interested in whether early interventioncould make a difference and change this early pattern of atypical brain development. so my colleague sally rogers and i developedan intervention called the early start denver model, where we taught therapists to bringthat baby’s attention back to an adult and to interact socially and to learn languageand learn how to play. and we’ve now conducted studies funded bythe national institute of health, randomized
clinical trials, where we’ve shown thatwhen you provide these early interventions that the impact on outcome is very significant. the average i.q. gain of a child who receivesthese interventions is 17 points. that’s over a standard deviation. now, in 2012, we published a study where weactually showed that early intervention not only affects behavioral development, but itaffects brain development. so we used electrophysiology to show thatwe actually had changed the patterns in the brain. and we showed that children who received earlyintervention now were showing normal patterns
of brain activity. and time magazine recognized this as one ofthe top 10 medical breakthroughs of 2012. and i think the reason why people were soexcited is because it said that the brain of a child with autism is very responsive,very plastic, and that early intervention can make a difference. now, this kind of work has been importantin our advocacy. because historically, people have not hadaccess to early intervention because there’s no insurance coverage that would pay for it. and so parents often would pay out of pocket.
they would get a second mortgage or not sendtheir other child to college in order to be able to pay for intervention. in fact, in north carolina, just last year,we finally passed a bill that will pay for or provide insurance coverage for early intervention. so in this—[applause] yes. and by the way, it took a lot of work andwhen—and when mccrory signed that bill, he acknowledged that duke was instrumentalin the passage of that bill. so we had to work for that. but in this picture, i’ve just testifiedto the u.s. senate on behalf of a bill that
would pay for insurance coverage for earlyintervention for our military families. and here i’m talking to senator kirstengillibrand, who has been a wonderful advocate for children’s health. now, in most parts of the world, uh, thereare no trained professionals to provide early intervention, and even if we could train them,they just—there’s not enough of them for the number of people that need them. and so, sally rogers and i decided to createa version of the early start denver model that could be delivered by parents. and we found that parents are very good atthis.
they can learn to use those strategies topromote language and social development. and now the early start denver model has beentranslated into 14 languages. it’s used worldwide. and in fact, in china, the ministry of healthis paying for a very large clinical trial where they’re teaching parents in chinato deliver these interventions with very positive outcomes. and more recently, lauren franz, who is ayoung psychiatrist at duke, received funding from the national institutes of health whereshe is going to be teaching families in the villages of south africa to be able to providethese interventions to their children.
so we’re really hopeful that these kindsof approaches will be able to begin to address the need, uh, for treatment on a global scale. now, as you can tell, i’m very excited aboutearly intervention and behavioral intervention does have a very big impact on the outcome. but unfortunately, there are many people thatdespite having received those intervention continue to struggle. about 25% of people with autism never learnto speak. and so our work is not done. and one of the reasons why i was very excitedto come to duke is because there are scientists
that are studying molecular or cellular therapiesthat are designed to enhance neuroplasticity and synaptic function, and could hopefullyhelp those individuals who were not as responsive to early intervention be able to have betteroutcomes. now currently, there are no fda-approved drugsthat address the core symptoms of autism, that are designed to improve social and languageand those core symptoms that we think of as autism itself. and yet at duke, we’re currently conductingfive clinical trials that are testing molecular or cellular therapies that are addressingcore symptoms and are designed to enhance neuroplasticity.
now, i’m going to end by just telling youabout one of those trials, but if you’re interested in the others, i’ll be, um, aroundduring the supper period and i’m happy to talk about the rest of them. but the work i’m going to tell you aboutis work that i’m conducting with a renowned pediatrician, joanne kurtzberg. and it’s such an honor to be collaboratingwith her. and she has been at the forefront in testingumbilical cord blood as a treatment for a wide range of conditions. all the way from conditions that affect infantsthrough now looking at adult stroke.
but these are all conditions that affect thebrain. and the rationale behind this approach isthat there’s increasing evidence that autism, at least in some cases, involves neuroinflammation. so in this slide that you see above me, it’sa pet scan. and you can see on the bottom that the scanof the person with autism has cells called microglia that are very activated. the microglia are the immune system in ourbrain, and compare that to the image of the brain from a typical person. now what dr. kurtzberg has shown is that cordblood, which contains stem cells, has the
effect of dampening that microglia, whichwe know interferes with synaptic functioning. and then it promotes brain development, andparticularly functional connectivity. and with a very generous gift from the marcusfoundation, we’ve now launched a program of research where we’re looking at cordblood treatment not only for autism but cerebral palsy and stroke. and we’ve just finished our first open-labelclinical trial for young children with autism. these are two- to six-year-olds. they come to—to duke. they have one infusion of cord blood and thenwe followed them for a year and we studied
not only their behavioral development butalso their brain development. and what we found was that about 60% of thosechildren actually did show significant improvements in their ability to socially interact andin their language skills. and these were correlated with changes thatwe could measure with mri. where we could see that the brain was becomingmore normally developed and functionally connected. now, this was an open-label trial. it means we didn’t have a control group. so we don’t know yet whether this actuallywas due to the cord blood treatment. but this summer, we will launch a very large,double-blind, randomized, placebo-controlled
trial, and at the end of that trial, we’llhave a better understanding of whether this might be an effective treatment for autism. so i’m going to end with this beautifulpainting and this is a painting that is by jesse park. she is a self-taught artist with autism. so thank you very much. [applause][music]
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