AT ONE-AND-A-HALF YEARS OLD , “ Timmy ” knew his colors , shapes and numbers in English , Spanish and Farsi . He could put together puzzles faster than anyone else his age . He was a joyous and sociable boy . But by three years old , Timmy had seemingly disappeared , diagnosed with autism .
By the time he was 11 , Timmy spoke only “ in short two- and three-word sentences ,” said his mother . “ He was prone to echoing other people ’ s words and getting hyper , running to and fro in any social setting .”
The story is a common one for children diagnosed with autism spectrum disorder ( ASD ), a condition estimated to affect 1 in 68 children . But what happened next with Timmy is uncommon and , perhaps , the promise of a better future . Earlier this year , UC San Diego School of Medicine researchers conducted a phase I / II safety trial investigating the effects on autism of a 100-year-old drug called suramin . In Timmy and four other boys ages 5-14 , a single low dose of suramin resulted in nearly immediate improvements in core symptoms of autism .
Timmy became calmer . He spoke in complete sentences and remembered his numbers . He began eating new foods and joked with his therapist . Yet while his cognitive and behavioral improvements expanded over several weeks , they gradually declined and disappeared as the suramin dose wore off . “ It shows that all of these kids are capable of doing great things and they are sort of locked into their own bodies . And this is a drug that can unlock that ,” said Timmy ’ s mother . “ That ’ s sort of where we are as parents . You know your child is in there .”
The study was a coup for Dr . Robert K . Naviaux , who has spent the last 10 years pursuing a new unifying theory for the cause and treatment of autism . Naviaux has long sought a drug that could test his hypothesis that ASD is fundamentally associated with a treatable metabolic syndrome .
“ Metabolism is the language that the brain , gut and immune system use to communicate ,” says Naviaux . “ These systems are linked . You can ’ t change one without changing the others . Each of these systems works differently in autism , but more specifically , the communication between these systems is changed .”
Naviaux suggests the key to autism lies within the cell danger response ( CDR ), a natural and universal cellular reaction to injury or metabolic stress . CDR prompts cells to barricade themselves from both perceived threats and neighbors . But CDR can get stuck , and if it happens during early childhood development , Naviaux believes autism is the result . “ When cells stop talking to each other ,” he says , “ children stop talking .”
SURAMIN APPEARS to temper the CDR , blocking alarm signals so that cells return to a normal state and resume normal functions and development . “ We have tried every new treatment out there for over 10 years ,” said the mother of a 14-year-old trial participant . “ Nothing has come close to all the changes in language and social interaction that we saw after suramin . We saw our son advance almost three years in development in just six weeks after suramin . We want to see now if a few doses of suramin over a few months might help him even more .”
That ’ s Naviaux ’ s plan . While the initial trial results were encouraging , longer and larger trials with multiple doses will be necessary to further prove his theory . Naviaux is seeking donors to fund that effort , as all of his suramin research thus far has been supported by philanthropy . “ Additional trials may prove suramin is not the answer ,” Naviaux says . “ Its effects might be limited or not self-sustaining , or side effects could emerge .” But Naviaux believes his work can open eyes and possibilities , not just that suramin might be effective , but that ASD is not immutable . “ Right now , developmental psychologists are taught that autism is permanent — our research suggests otherwise .”
Winning the Cellular War
To test his theory that autism is a metabolic and potentially treatable condition , Dr . Robert Naviaux with the School of Medicine conducted trials of suramin , a sort of " cellular peacekeeper . "
“ When cells are injured , stressed or perceive a threat , the cell danger response is triggered . Cells behave like countries at war ,” Naviaux explains . “ They harden their borders . They don ’ t trust their neighbors . But without constant communication with the outside , cells begin to function differently .”
These different cell behaviors include spewing a molecule called adenosine triphosphate or ATP . ATP outside a cell acts as a danger signal , telling other cells to shift resources toward defense , or calling them to war . An ATP imbalance can then cause cells ’ nuclei to alter gene expression , which can result in developmental dysfunction . When this occurs in childhood , Naviaux believes , that may mean autism .
Suramin tells cells to stop spewing ATP . “ It silences the siren ,” says Naviaux , “ signaling the cellular war is over , the danger has passed and cells can return to ‘ peacetime ’ jobs like normal neurodevelopment , growth and healing .”
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