At the time of writing this, the GMA website, which usually posts follow-ups, has nothing on their site, which I found a little strange. The mother also appeared not with another member of her family, but a lawyer, who was also a family friend. I wondered how these unusual, but small anomalies reflected on modern society’s schizoid views on drugs. I certainly do not approve of “drugs”, the term which brings to mind things like cocaine, methamphetamine, or marijuana. It should also however bring to mind things like coffee, chocolate, and aspirin. Marijuana is a Schedule I drug in the U.S., which, according to definition, means it has no medical use. This is interesting since there has recently been quite an effort to promote so-called medical marijuana. So really our question should be what is a medicine?

In theory a reasonable definition might have medicine as being some sort of foreign substance that effects treatment of disease. Autism is certainly a disease, but is there any evidence that marijuana can “cure” autism? Without doing any research, we should already be skeptical that the answer could possibly be yes.

For one thing, autism is really a spectrum disorder. That means that there may be many causes, both genetic and environmental that produce the constellation of symptoms we call autism. If a doctor were treating you for cancer, he would first have to know whether it was brain cancer or skin cancer to choose a proper treatment. There are certainly anecdotal sources like this GMA news story, but also other places on the Internet that will tell you that someone has cured their child with marijuana brownies, but do you really know that that child was diagnosed with autism? How can a parent know if the supposedly cured child has the same etiology (same cause of the disease) as their own child? Marijuana, or at least its active component, THC, certainly has a psychotropic affect, as it interacts with cannabinoid receptors in the brain. Can this mean that somewhere somehow marijuana can have some unrecognized benefits for autistic individuals? At this time, there simply is insufficient information to come to a conclusion.

You can certainly understand the desperation of any parent who wanted to get the best treatment possible for their child. While the American Pediatric Society continues to oppose medical marijuana, they are in favor of at further investigation into its applications as a drug. Until then, even so-called medical marijuana is still illegal under federal and most state laws. Hopefully however, if a clinical application for autism is found, stigmas about the idea of marijuana as a “drug,” will not hinder scientific debate and progress.

In the emerging field of pharmacogenetics, scientists study genome variations and correlate them with drug treatment response.  For example, variations (also called polymorphisms) in genes encoding enzymes involved in drug metabolism have been found to affect the activation, deactivation, and toxicity of drugs used to treat cancer, heart disease, and psychiatric disorders.  Recently, scientists found that DNA sequence can also be used to predict responsiveness to current Hepatitis C treatment (a 48-week course of peginterferon-α-2b combined with ribavarin).

The sequence at a single DNA position (single nucleotide polymorphism, or SNP) on chromosome 19, close to the gene encoding the interferon-λ-3 protein, has a significant effect on a patient’s ability to clear Hepatitis C infection with treatment.  Patients with a C nucleotide at the critical position on both copies of chromosome 19 (CC genotype) are two to three times more likely to respond to treatment than those patients with the T nucleotide at the same position (TT genotype).

In the case of Hepatitis C, the mechanism by which one sequence is more therapeutic than the other is not yet understood.  However, sequence information can still assist doctors in selecting appropriate treatments: as alternative Hepatitis C treatments become available, doctors may bypass the current treatment for those patients with the TT genotype.

As DNA sequencing becomes cheaper and easier, and genome information becomes elucidated, the personalization of medicine may become a reality.

Just recently, a group of researchers at the University of Maryland School of Medicine have identified a variant of a gene that is believed to play a major role in determining why people do not respond to a popular anti-clotting medication. This gene variant, carried by as many as a third of the general population can put patients at increased risk for subsequent heart attacks, strokes and other serious cardiovascular problems. The interesting thing is, that this increased risk is not due to patients genetic predisposition for these disorders, but because it renders their medication ineffective.

Medicines that we introduce into our bodies often require one or several important mechanisms to unfold their intended effects: they may have to be actively transported into our cells, biochemically altered and thereby activated, or they may require deactivation and/or removal in order to not do more harm then good. Any of these processes may involve proteins on one level or another and, therefore, depend on genes. Thus, as we have maps that indicate the loci associated with genetic disorders (visit Tour > genome spots in DNA Interactive), we will soon have maps that tell us where to look if we wish to know our predisposition to the medications we use to cure ailments: whether they will do us any good, are totally useless or, in a worst case scenario, can even harm us.