Selectivity
of drugs has been the goal of pharmacology for over a
century, and the modern science of genomics makes it
seem tantalizingly closer than ever.
Drugs are not smart bombs that hit only the target
they're aimed at; many drugs are simply more toxic to
germs than to their human host. But the ultimate drug
would have no side effects at all. That ideal cannot be
reached through today's model of mass-market drugs,
because genetic differences cause people to respond to
drugs differently. Some people cannot tolerate
penicillin, for example; others require unusually low
doses of a given medicine because their liver enzymes
break it down so slowly.
What is wanted are custom-made smart bombs, designed
for each niche of the population and perhaps even
individual patients, according to the vagaries and
variance of their genes.
If this could be done, many drugs discarded because
they are good for some people but harmful to others
could be revived and prescribed selectively. Doctors
would not have to go through a lengthy process of trial
and error to find the right drug for their patients.
Overdoses would also be less likely, because dosage
would be based not only on age and weight but on genes
as well.
Such medical care would extend beyond designer drugs.
People could be genetically screened for their risk for
particular diseases. So one might learn that one was at
particular risk for, say, substance abuse, and decide
not to experiment with anything stronger than herbal tea.
The proof of concept has already been made. Children
with leukemia can be tested for a rare but fatal effect
of a common anticancer drug and steered to another drug.
Genetic differences linked to the body's breakdown of
certain drugs in the liver have also been used to
calibrate dosages. And recently, a genetic test was
developed that helps women at risk for a recurrence of
breast cancer decide whether or not to undergo chemotherapy.
Before customized medicine comes, though, we will need
to learn two things: how genes affect drug action in the
body and how different genes conspire with environmental
influences to give rise to disease in the first place.
The tools to do this have been developed, and
improvements are coming daily.
Because the human genome has now been sequenced, the
starting set of data is available. At this point,
microarrays, rapid DNA sequencers, protein biochips, and
other biotech tools must help scientists figure out what
it all means, and new technologies to provide useful and
speedy genetic profiles of patients must be developed, too.
Finally, and it will be a difficult step, the
pharmaceutical industry will have to reexamine its
strategy of wringing all its profit from a handful of
"one-size-fits-all" blockbusters. Custom drugs will fly
only if companies find ways to make money off niche
markets—maybe even a market of one.