Much is written about unintended consequences of policies that start from good intentions and often end up creating larger problems than they meant to solve. This is one of the better arguments in favor of a smaller, less activist government. Those who argue in favor of more government must somehow believe that government bureaucrats are uniquely able to discern and avoid such "unintended consequences.' In some cases, the unintended consequences are so predictable as to raise questions about the intelligence of the architects. A series of policies and proposals that are in the news have entirely predictable consequences which will become increasingly apparent as times goes on.
Biotech innovation is one of the great, impending growth fields for the near term. Biology is rapidly becoming an Information Technology and new treatments for all sorts of intractable medical conditions are right around the corner. The system works by balancing hope (or hype) with a risk averse regulatory environment. This means that new discoveries are hyped long before they offer any possibility of clinical efficacy. Compounding the inherent tendency to disappoint desperate people, the regulatory agency (FDA) in charge of medical technology requires negotiating a byzantine structure of rules and regulations which delay potentially life saving treatment and makes it far costlier than necessary. The predictable consequences include terribly painful outcomes:
Report: Fetal stem cells trigger tumors in ill boy
A family desperate to save a child from a lethal brain disease sought highly experimental injections of fetal stem cells—injections that triggered tumors in the boy's brain and spinal cord, Israeli scientists reported Tuesday.
Scientists are furiously trying to harness different types of stem cells—the building blocks for other cells in the body—to regrow damaged tissues and thus treat devastating diseases. But for all the promise, researchers have long warned that they must learn to control newly injected stem cells so they don't grow where they shouldn't, and small studies in people are only just beginning.
Tuesday's report in the journal PLoS Medicine is the first documented case of a human brain tumor—albeit a benign, slow-growing one—after fetal stem cell therapy, and hammers home the need for careful research. The journal is published by the Public Library of Science.
"Patients, please beware," said Dr. John Gearhart, a stem cell scientist at the University of Pennsylvania who wasn't involved in the Israeli boy's care but who sees similarly desperate U.S. patients head abroad to clinics that offer unproven stem cell injections.
"Cells are not drugs. They can misbehave in so many different ways, it just is going to take a good deal of time" to prove how best to pursue the potential therapy, Gearhart said.
The unidentified Israeli boy has a rare, fatal genetic disease with a tongue-twisting name—ataxia telangiectasia, or A-T. Degeneration of a certain brain region gradually robs these children of movement. Plus, a faulty immune system leads to frequent infections and cancers. Most die in their teens or early 20s.
Israeli doctors pieced together the child's history: When he was 9, the family traveled to Russia, to a Moscow clinic that provided injections of neural stem cells from fetuses—immature cells destined to grow into a main type of brain cells. The cells were injected into his brain and spinal cord twice more, at ages 10 and 12.
Back home in Israel at age 13, the boy's A-T was severe enough to require that he use a wheelchair when he also began complaining of headaches. Tests at Sheba Medical Center in Tel Aviv uncovered a growth pushing on his brain stem and a second on his spinal cord. Surgeons removed the spinal cord mass when the boy was 14, in 2006 and they say his general condition has remained stable since then.
But was the boy prone to tumors anyway or were the fetal stem cells to blame? A Tel Aviv University team extensively tested the tumor tissue and concluded it was the fetal cells. Among other evidence, some of the cells were female and had two normal copies of the gene that causes A-T—although that boy's underlying poor immune function could have allowed the growths to take hold.
Fetal, ie embryonic, Stem Cells (ESC) were shamelessly hyped over the last 8 years, in part because people who did not understand biology could use the issue to attack the Bush Administration. ESC will never be as clinically useful as Adult derived Stem Cells for a long list of reasons. At the moment and for the foreseeable future, research on ESC is and will remain much farther away from clinical applicability than similar research on partially differentiated adult stem cells (or adult stem cells that have been partially de-differentiated to a state of pluripotentiality.)
Balancing risks and potential benefits is extraordinarily difficult when considering interventions in the human organism. The extensive testing required by the FDA before a new treatment is approved is designed to minimize risk, yet even when the Medical Scientists believe they know the risks of particular treatments, surprises abound, and when surprises show up in medical testing, more often they are unpleasant surprises than pleasant surprises (like Minoxidil.)
The Thalidomide disaster of the 1960s led to such restrictive rules and risk aversion that many promising treatments are routinely delayed or eschewed altogether for fear of an inability to gain a return on investment (now >$1.4 Billion to bring a new drug to market) or fears of unlimited and endless liability for untoward outcomes. (See Killing the Golden Goose.) The fact that the Democrats (and their allies in the litigation-industrial complex) identified "Big Pharma" as a target during the last several years is likely to make these problems worse.
As I noted in Regulation & Innovation, you are likely to have more access to medicines and cutting edge technology, including some which have an excellent risk/reward ratio, if you are a dog rather than a person.
I do believe that Medical professionals have a better grasp of risks in Medicine but, especially in an environment where desperate consumers are willing and able to educate themselves about the risks, greater patient autonomy and choice can only be beneficial.
The Laetrile scam/fad/craze (whatever you prefer) in the 1970s showed that people are willing to do stupid things when they are desperate. No matter how paternalistic our government becomes, there will always be people ready, willing, and able to believe nonsense. Most people are not scientifically literate and human beings have a deep seated predisposition to believe in magic and nonsense.
Medical Science is in the early phase of its own Singularity; in other words, the data is accumulating far faster than our Research establishment can metabolize it. We are in the situation of sipping from a fire hose through a straw. In such a setting, in order to maximize our ability to explore all reasonable paths forward, a system that allows mature adults to decide on their own risk tolerance would be far superior to a system that treats every change as a danger to be defended against and only in the (near) guaranteed absence of risk (an impossibility) allows new treatments.
In New York we now see Alex Rodriguez admitting using a designer drug to enhance his performance. He rightly faces the disapprobation of baseball fans for having been caught cheating. In the next several years it is likely that a drug or drugs will enter the market for Diabetes based on the Sirtuin system which is enhanced by Resveratrol. There are already millions of people taking Resveratrol to slow aging. Once such a drug enters the market, there will be many millions of people taking it off-label as a naturalistic experiment. It is an inevitability.
We have the choice as a society of continuing to pretend that the government can protect us from all risks associated with medical technology (while allowing anything sold as a nutritional supplement to escape any regulation), which will retard medical advancement and subject the most desperate to dangerous and poorly managed risks, or making the system more rational, treating adults like adults, and allowing each of us to determine our own risk tolerance.
If our society continues on the path of allowing our rules to be determined by treating each of us as if we are the least capable, and empowering government to protect us all as potential helpless victims, we will be surrendering the future to those who are either more unscrupulous or more courageous.
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