Posts Tagged ‘Translational Medicine’


EEG of brain; via childrenshospitalblog.org

This could be the Mama Bear coming out in me (gosh, it really must be because I HATE the term Mama Bear), but this so-called experiment and drug GRN-529 (article pressed below with link) that supposedly reduces autistic symptoms in mice rubs me completely the wrong way. The article is very careful to refrain from calling  GRN-529  a cure and to state that it’s treating…  no wait… REVERSING symptoms of autism, such as making the mice more sociable and minimizing obsessive-compulsive behaviors.  But tagging it as a drug that specifically reduces ASD symptoms… especially when those qualities are present in so many other different disorders and are so often simply personality quirks in people… it bothers me as a whole.

Of course there’s the whole “messing with the synapses and neurotransmitters of the brain” thing.  Considering these mice weren’t actually given autism, since that’s impossible, but that they were manually given symptoms that’s one of my reservations with this experiment.  I also have issues with the mice being inbred on top of their manufactured “symptoms.”  Maybe that’s a mountain out of a molehill, and I can’t quite articulate right now why that one bothers me so very much, but it does.  I think it’s also a bit disingenuous to claim that it’s not trying to cure ASD when it’s attempting to target the neurotransmitters the way that they would.

Don’t get me wrong here, I’m not against medications when they’re warranted.  I’m not against researching Autism Spectrum Disorders and all of the surrounding issues.  I think part of what’s bothering me is that the more I learn, the more it seems to me that ASD is a collection of neurological differences and disorders.  I believe that at its core, ASD is genetically based and ASD’s affect a wide array of neurological issues.

The behaviors can be so very difficult to deal with as a family, as a parent, and especially the child who becomes a teen and later an adult.  The destructive behaviors in high functioning individuals should be approached on an individual basis.  I worry, I think, that a drug like GRN-529 might end up being something that is “pushed” much the way ADHD medications are pushed for highly active and spirited children that aren’t actually ADHD.  Don’t like the symptom? Drug it up even if it’s not therapeutic.  Let’s make everyone else’s lives easier.  That might sound contradictory coming from me, since we have Gracie (my darling 3rd grader who has Autism) on a low dose of an ADD medication along with her seizure medication.  She’s able to concentrate and work at school.  She’s been better able to follow directions.  In her own way she has commented on her improved ability to focus, and that she likes that feeling.  She has less anxiety while at school because of it.  She has less anxiety about bed time than she used to (granted it’s still there, but not like before).  We’re also in the decision-making process for our eldest daughter regarding her ADHD.  It’s not easy making these decisions and figuring out if what we’re considering tips the scales in “we’re doing this for her and not for us” favor.

This particular bit of mouse research feels a bit misdirected.  I know that the research itself is much more complicated than the article is laying it out to be, but ASD is so much  more than that area of the brain.  It’s so much more than OCD and social awkwardness.  So maybe my feeling is that this bit of research is missing the point more than anything else.

Or maybe I’m over-reacting or misunderstanding or being overly critical or (gasp) all three.

Experimental drug reduces autism symptoms in mice, gov’t study shows – HealthPop – CBS News.

April 26, 2012 1:13 PM
Experimental drug reduces autism symptoms in mice, gov’t study shows

(CBS News) Autism affects one out of every 88 American children and while there are available treatments for early intervention, there is no cure. A new government-funded study has found an experimental treatment is effective at reversing symptoms of autism in mice.

For the study, published in the April 25 issue of Science and Translational Medicine, researchers from the National Institutes of Health bred a strain of mice to display autism-like behaviors. Similar to how children with autism have social deficits and engage in repetitive behaviors, these mice did not interact and communicate with each other and spent an inordinate amount of time engaging in repetitive behavior – in this case self-grooming.

Cue the experimental drug called GRN-529. The drug was designed to inhibit a type of brain cell receptor that receives the neurotransmitter glutamate. Glutamate is typically involved in learning and memory processes and stimulates other areas of the brain and nervous system.

When mice with the autism-like behaviors were injected with the experimental compound, they reduced the frequency of their repetitive self-grooming and spent more time around strange mice, even sniffing them nose to nose. When tested on a different strain of mice, the experimental compound stopped all repetitive jumping behavior.

“These new results in mice support NIMH-funded research in humans to create treatments for the core symptoms of autism,” Dr. Thomas R. Insel, director of the National Institute of Mental Health, said in a statement. “While autism has been often considered only as a disability in need of rehabilitation, we can now address autism as a disorder responding to biomedical treatments.”

The researchers said although most mouse brain findings often don’t translate to humans, the fact that these compounds are already being tested for an overlapping condition strengthens the case for the drug’s effectiveness. This class of compounds is currently being studied in patients with the genetic disease Fragile X syndrome, the most common inherited form of intellectual disability. About one third of patients with Fragile X syndrome also meet criteria for autism.

“These inbred strains of mice are similar, behaviorally, to individuals with autism for whom the responsible genetic factors are unknown, which accounts for about three fourths of people with the disorders,” noted study author Dr. Jacqueline Crawley of the NIMH. “Given the high costs – monetary and emotional – to families, schools, and health care systems, we are hopeful that this line of studies may help meet the need for medications that treat core symptoms.”

Some experts exercised caution with the new findings. In an accompanying editorial in the same journal issue, Baltazar Gomez-Mancilla, executive director of translational medicine neuroscience at Novartis, wrote, “It is too early to speculate as to whether or not autism spectrum disorders can be reversed by small molecules.”

Dr. Uta Frith, a professor of cognitive development at University College London, told BBC News that neurotransmitter problems have long been suspected as an origin of autism, “However, it will be a long time until these findings can be translated for human patients. Tampering with the synapse may well result in undesirable side effects,” he said.

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