None — BACKGROUND: According to the emedtv.com, males are four-times more likely to have autism than are females. Research shows about one out of every 110 children has some form of autism. About 500,000 children under the age of 21 have autism. According to the Mayo Clinic, children with autism show symptoms in three major areas in terms of development: language development, behavior, and social interaction. Examples of social skill challenges are: poor eye contact, resists cuddling and holding, doesn't respond to his or her name, and retreats to time alone as opposed to time with others. A few symptoms of language development include: starting to talk later than 2 years of age, doesn't know how to put together words and sentences, and an inability to start a conversation or hold onto one. Lastly, behavioral concerns consist of: becoming perturbed when daily or routine things change, constant movement, and unusual sensitivity to light. Although there is no cure for autism, there are medications that can be taken in order to keep irritability, tantrums, and aggression at bay.
TRIALS SEARCHING FOR AN ANSWER: Although many trials and studies have been conducted looking for answers and cures to autism, three recent studies were conducted that may present some more answers. In one trial, doctors targeted the child's digestive system. Research showed that certain proteins can get through the child's digestive system that should be digested first. This affects the nervous system because the proteins are not being broken down. A second trial involved the investigation of whether or not cholesterol affects autism. Children with low cholesterol are at higher risk of developing autism. Furthermore, some doctors believe certain children are missing a key enzyme that produces cholesterol. By lacking this enzyme, brain development is affected, and autism may be the result. A third and final trial looked at a drug called Namenda. Currently, Namenda is used to help treat Alzheimer's, but some researchers believe this may also be an effective drug for improving motor skills and language in autistic children. Some side effects of Namenda include dizziness, headaches and pain. Allergic reactions such as rash, hives, and itching may occur as well.
IN-DEPTH INTERVIEW WITH L. EUGENE ARNOLD M.D.
L. Eugene Arnold, M.D., from the Ohio State University, tells us how a new drug may help doctors tackle autism in children.
Is there any medication now for children with autism?
Dr. Arnold: There's no medication for core autism symptoms. There are two drugs that are FDA approved for irritability, tantrums, self-injury and aggression in children with autism but nothing for the core symptoms of social interaction impairment, communication impairment or repetitive restrictive patterns of interest and activities.
What is this drug targeting?
Dr. Arnold: That's what we're hoping. Now we are fairly confident it would help any gastrointestinal problems that the youngster may have, but we're hopeful that it would also help the core autism symptoms.
What's the name of this drug?
Dr. Arnold: It's CMAT. It's a mix of digestive enzymes.
How does that mix of digestive enzymes have an effect on behavior?
Dr. Arnold: There are lots of theories, a lot of hypotheses about it. The fact of the matter is we don't really know for sure. One of the possibilities is that undigested proteins may have somehow upset the gastrointestinal function in such a way that it irritates the gut and proteins can get through that. Now that's just a hypothesis, it's not proven, and there are other hypotheses about it also.
Is this drug using that hypothesis?
Dr. Arnold: No, it's not based on that. It's based on the observations of Dr. Joan Fallon who noticed in her practice with children with autism that if she could help their gastrointestinal function, their autism symptoms also improved. She developed this investigational treatment. We're doing the study to find out if it works. We don't know yet for sure.
How is the study set up and how is it working?
Dr. Arnold: First we test the stool of the children to see if they have a low level of chymotrypsin. That's a pancreatic enzyme that prompts the gut to release other enzymes that then carry on digestion. If the level of chymotrypsin is low in the stool, then the youngster is taken into the study for a trial of the digestive enzyme supplement.
Do all children with autism have a low level of chymotrypsin in them?
Dr. Arnold: No. That's why we test that first because there's no reason to believe that this medication would help them if they don't have a low level. If they have a normal level, we just tell them that, and that's the end of it, they go no further in the study.
Does that mean if this study is proven correct and everything comes out, will there be different types of autism then?
Dr. Arnold: We already know there are different types of autism.
Are there different levels of Autism?
Dr. Arnold: Currently we know that not all children with autism have it for the same reason. For example, maybe 5% or so have Fragile X syndrome, but that's not the reason for the majority. That may be the case and that's why we're doing this study. At the same time, another small group may have autism for a different reason, maybe some sort of environmental toxin. There may be another group that has autism because of this lack of digestive enzymes, and if so, that's the group we're looking for. If there is such a group, then theoretically correcting that should help their autism and that's why we're doing this study.
How does the study work?
Dr. Arnold: There's a 12-week double blind part where people get either the real digestive enzyme or a placebo, a fake medicine that looks and tastes and feels the same. Then after those 12 weeks, everybody gets a chance to try the real enzyme for a year and a half or a little over a year.
How long has this study been going on so far?
Dr. Arnold: About five months.
Have you seen any differences? When would you start to see differences?
Dr. Arnold: We don't break the blind until everybody has gone through. We just don't have the answers yet.
Where could this lead then in the future?
Dr. Arnold: It would be one thing for which we would have a biological test that would direct treatment in autism. We need more of those. Right now, the only thing I can think of offhand is Fragile X, but we hope to add to the list.
Would this be something where you could have a test when you're an infant and be diagnosed right away?
Dr. Arnold: Conceivably, yes you could. If the test became cheap enough and if the percent of children who have autism for this reason would be high enough, then perceivably you could test the stool of infants and catch them very early. But that's speculation at this point.
What's exciting to you in this field? Is it this study?
Dr. Arnold: This is one of the exciting areas. Another one is the possibility that there may be a subgroup that has autism because of low cholesterol and we have a study looking into that also.
How would cholesterol affect symptoms of autism?
Dr Arnold: Cholesterol is necessary for normal brain development, and there's a disorder called Smith-Lemli-Opitz in which people are missing an enzyme for making cholesterol. Many of those develop autism if that problem is not corrected by diet. Ordinarily, we think of cholesterol as a bad thing and we promote low cholesterol foods, but there may be some individuals who should actually deliberately take cholesterol and that's why we're doing that study.
Is that why some children say they're cured of autism because they can take certain vitamins, or they change their gluten free diet? Would that be part of that impact?
Dr. Arnold: This would all be part of the same idea that there are different reasons for different people having autism, and if you can find one that's causing it for a given person and if you can correct then you should be able to help the autism. But all of this is in the very early stage of investigation right now.
Do you think that in the future we will be able to distinguish between different types of autism?
At this point, diagnoses are not made based on etiology on the cause. They're phenomenological. It's what you can see, and you make the diagnosis based on that. Now there are very specific operational criteria. For example, in autism there are 12 symptoms, and you have to have at least six of those, and at least two of them have to be in the social impairment areas. There are three different clusters, four symptoms each. That may change with the new diagnostic manual somewhat, but it's still a diagnosis that's made based on what you see. It's a surface diagnosis. It's not based on the cause. It can be many different causes of the same disorder. The goal, the Holy Grail, would be to be able to give some sort of test and say this person has these symptoms for this reason and base the diagnosis on the cause as well as on the symptoms. Some other areas of medicine have achieved that, some others are still working on it as we are in the field of autism.
Would you be able to give an infant medication so they would never experience the signs of autism?
Dr. Arnold: That would be ideal. Again, we're at a very early stage, and this is all speculation, but that's what we might hope for in the future.
Is there any other study that you're excited about?
Dr. Arnold: Yes, we also have a study of memantine, the Alzheimer's drug. Some preliminary work has suggested it might be useful and I mentioned a little while ago one that we're just applying for funding for which would test a vitamin mineral supplement for the symptoms of irritability.
What would the drug for the Alzheimer's disease tackle?
Dr. Arnold: There's some suggestion that cholinergic neurotransmission is deficient in autism and the Alzheimer's like drug. The memantine is a cholinesterase inhibitor, which means that it stops the breakdown of acetylcholine so that each molecule of acetylcholine lasts longer in the synapse. Therefore, you get more mileage out of each molecule. This will increase the cholinergic neurotransmission, which is theorized or hypothesized to be deficient in those cases.
Is the drug orally taken? How is that taken? Is it every day?
Dr. Arnold: Yes, it's orally. It's a powder that's mixed in the food and it's every day, yes.
Would that be something that you'd have to take for your lifetime?
Dr. Arnold: Probably. If it helps it would probably be needed for a lifetime. If a person has a deficiency of the enzymes, then probably it's going to be a lifetime thing. But it would be no worse than say, taking insulin for diabetes and there are many people who have this and if you replace then the person can carry on a fairly normal life. Now again, we don't know that's going to be the case in autism for this digestive enzyme. That's our hope, but there is an analogy where having to take something lifelong wouldn't be such a bad thing.
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