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TagsNew treatment targets symptoms of disorder
BY BOB HOLT
NEWJERSEYNEWSROOM.COM
Latest developments in the battle against autism are happening right in New Brunswick, New Jersey.
The final testing of a new treatment for autism is taking place at Saint Peter's University Hospital.
"We are targeting the symptoms of autism," Dr. Joan Fallon told the Asbury Park Press. Fallon is founder of Curemark LLC, a drug-research and development company in Rye, N.Y.
The product is called "CM-AT," for "Curemark's autism treatment."
The testing being conducted at Saint Peter's involves 170 youngsters, hospital spokesman Phil Hartman said.
Fallon said the test pool covers children age 3 to 8. She said she expects results "sometime early next year."
Dr. Barbie Zimmerman-Bier of Saint Peter's, who is running the study, said: "Subjects heard about it and they got word from support groups. . . . There was a screening process to see if they were eligible.
Curemark told the Asbury Park Press in a prepared statement: "CM-AT, which has received fast-track status from the FDA, is based on Curemark's research that showed enzyme deficiencies in autistic children, resulting in an inability to digest protein. The inability to digest protein affects the availability of amino acids, the building blocks of chemicals essential for brain function.
The statement concluded, "If approved, CM-AT will be one of the first therapies to address the underlying physiology of autism."
Fallon said Saint Peter's was chosen because "they are a well-known center for autism," citing the work of Zimmerman-Bier, head of the developmental and behavioral pediatrics program at Saint Peter's Healthcare System.
Back in April during Autism Awareness Month, NJ.com reported that Caldwell College received a $550,000 federal grant to help ensure that more teachers learn how to recognize symptoms of autism in their students.
One in 94 children in New Jersey is diagnosed with autism — higher than the national average of 1 in 110 children, according to the Centers for Disease Control and Prevention.
The money can hopefully help to lead autistic children out of their isolation.
Autism New Jersey is a nonprofit agency providing information and advocacy, services, family and professional education, and consultation about autism. For more information or to make a donation, visit the Autism New Jersey web site.
But it would appear he is being hamstrung by Curemark, who is pumping up CM-AT before the phase III results are available. Their website currently links to a BusinessWeek article about the product:
"One of the most promising treatments in this category is a drug called CM-AT made by a startup called Curemark. Dr. Joan Fallon, the company's founder and CEO, observed that many autistics show a strong preference for foods high in carbohydrates and low in protein. A diagnostic test revealed that some autistic children lack enzymes that digest protein. As a result, these children produce fewer of the essential amino acids that are the building blocks for brain development and neuroreception. Fallon believes this deficiency is linked to the most severe symptoms of autism, and she says an early observational study of CM-AT, an orally ingested powder that delivers protein-digesting protease, showed "significant improvements." Curemark is enrolling patients in phase III clinical trials at 10 to 12 site the largest autism trial to date." (emphasis mine)
Where did she get these ideas? What evidence supports this hypothesis? Who is Dr. Joan Fallon?
First, Fallon is not a doctor; she is a chiropractor. She is not exactly widely published in the medical literature. In 2005 she put a piece in Medical Hypotheses (a controversial journal, to say the least) in which she "hypothesizes" that the antibiotic Augmentin may be responsible for autism. She applied for a patent to use the commonly available laxative lactulose to treat autism. And she's promoted the idea that autism is somehow related to the gut. None of her ideas really hang together. Is autism caused by a failure of protein digestion? By an antibiotic? By toxic ammonia levels? Whatever the cause, she seems to be looking for a profitable cure. I'm not sure that she can patent lactulose, but she apparently found a substance she can patent---CM-AT, a proteolytic enzyme in a novel delivery system.
There are a number of things I find disturbing about this study. While it appears to be well-run, it fails to conform to an important scientific and ethical standard: plausibility. According to the Helsinki Declaration protecting human research subjects:
12. Medical research involving human subjects must conform to generally accepted scientific principles, be based on a thorough knowledge of the scientific literature, other relevant sources of information, and adequate laboratory and, as appropriate, animal experimentation. The welfare of animals used for research must be respected.
Given the low plausibility of the hypothesis, even positive results, should they be found, must be interpreted with a high index of suspicion. The statistics underlying the study of implausible hypotheses tell us that positive results are often due to chance or problems with study design rather than the drug being tested. This is not an esoteric technical detail. This is a study being done on child subjects whose parents have been led to believe this treatment may help. There is no reason to believe this is so. Perhaps some day if there are convincing preliminary studies, the current one would be justified. At this point, though, it is simply another questionable study built on a foundation of bad science.
But it would appear he is being hamstrung by Curemark, who is pumping up CM-AT before the phase III results are available. Their website currently links to a BusinessWeek article about the product:
"One of the most promising treatments in this category is a drug called CM-AT made by a startup called Curemark. Dr. Joan Fallon, the company's founder and CEO, observed that many autistics show a strong preference for foods high in carbohydrates and low in protein. A diagnostic test revealed that some autistic children lack enzymes that digest protein. As a result, these children produce fewer of the essential amino acids that are the building blocks for brain development and neuroreception. Fallon believes this deficiency is linked to the most severe symptoms of autism, and she says an early observational study of CM-AT, an orally ingested powder that delivers protein-digesting protease, showed "significant improvements." Curemark is enrolling patients in phase III clinical trials at 10 to 12 site the largest autism trial to date." (emphasis mine)
Where did she get these ideas? What evidence supports this hypothesis? Who is Dr. Joan Fallon?
First, Fallon is not a doctor; she is a chiropractor. She is not exactly widely published in the medical literature. In 2005 she put a piece in Medical Hypotheses (a controversial journal, to say the least) in which she "hypothesizes" that the antibiotic Augmentin may be responsible for autism. She applied for a patent to use the commonly available laxative lactulose to treat autism. And she's promoted the idea that autism is somehow related to the gut. None of her ideas really hang together. Is autism caused by a failure of protein digestion? By an antibiotic? By toxic ammonia levels? Whatever the cause, she seems to be looking for a profitable cure. I'm not sure that she can patent lactulose, but she apparently found a substance she can patent---CM-AT, a proteolytic enzyme in a novel delivery system.
There are a number of things I find disturbing about this study. While it appears to be well-run, it fails to conform to an important scientific and ethical standard: plausibility. According to the Helsinki Declaration protecting human research subjects:
12. Medical research involving human subjects must conform to generally accepted scientific principles, be based on a thorough knowledge of the scientific literature, other relevant sources of information, and adequate laboratory and, as appropriate, animal experimentation. The welfare of animals used for research must be respected.
Given the low plausibility of the hypothesis, even positive results, should they be found, must be interpreted with a high index of suspicion. The statistics underlying the study of implausible hypotheses tell us that positive results are often due to chance or problems with study design rather than the drug being tested. This is not an esoteric technical detail. This is a study being done on child subjects whose parents have been led to believe this treatment may help. There is no reason to believe this is so. Perhaps some day if there are convincing preliminary studies, the current one would be justified. At this point, though, it is simply another questionable study built on a foundation of bad science.