Medical Research

Over the last century, medical research has arguably advanced human health more than all the previous 5,000 years combined. One statistic puts the change into perspective: Americans born in 1900 could expect to live an average of 49 years, but those born today will live an average of 77 years.

Much of the credit for that longer, healthier life span goes to medical discoveries such as insulin, antibiotics, blood pressure medications, and advanced surgery. Scourges such as polio and measles have been virtually eliminated in the U.S., and we have vastly increased our arsenal of treatments to fight AIDS and cancer.

Now deciphering the human genetic code promises a dramatic era of understanding, treating and preventing inherited diseases such as hemophilia and muscular dystrophy. But at the same time, advances in genetic research force us to address the social implications of being able to fundamentally alter our inner composition. Should researchers be allowed to tinker with our genetic codes, or create copies of human beings? Could we somehow be harming future generations by aiding sick people today?

As our knowledge of medicine reshapes society, it also presents new public policy challenges, such as the level of government funding for research, to what extent companies should be allowed to patent and control advances, and the implications of public-private collaborations.

The price of health

To build upon any of these new medical discoveries--from cancer screenings to Alzheimer's drugs to genetic repairs--will require money. How much money do we want to invest in pursuing such knowledge? Medical research requires hundreds of billions of dollars--a substantial portion of which comes from taxes--and scientists could easily justify spending more to create better diagnostic tests and more effective treatments. In fiscal year 2000, the federal government spent $17 billion on the National Institutes of Health, the agency primarily responsible for medical research. President Bush is seeking to double that figure by 2003.

Groups devoted to fighting specific diseases regularly compete for a greater share of existing federal dollars. Those who rally for more money for AIDS research, breast cancer or diabetes are vying for a greater share of the same pie. Meanwhile, research into diseases that are no less debilitating may suffer because they affect too few people to be economically viable. Other scientists argue that as government responds to the lobbying for disease research, it is neglecting basic scientific study of other vital problems, like the environment.

And with government funding comes the question of what research is appropriate to fund or even permit to go forward at all, particularly in genetics and stem cell research.

DNA and destiny

In the summer of 2000, scientists announced they had cracked the code of the human genome, finding about 30,000 individual genes that guide human development. Already, medical researchers have identified hundreds of defects in the sequence of these genes that are "markers" for diseases.

More than 4,000 diseases have genetic causes. (Click here to read about some inherited diseases). Unraveling the genome has heralded a new era for discoveries about how the body works and potentially how to make it work better. It allows patients to know whether they are at risk for certain diseases so they can take preventative measures. Eventually, doctors may actually be able to remove or alter specific genes to treat disorders or fix problems before they occur.

But what are the implications of knowing precisely who may develop a genetic disorder? Specifically, knowing a patient's risk may help save their life but could cost them their job or health coverage if their employer or insurance company finds out. In several European countries, insurers are banned from requiring genetic tests and 33 U.S. states either ban or restrict such testing. But if a patient decides to be tested, an insurer may be able to demand the results. In Britain, for example, insurers may ask for results if someone chooses to be tested for the fatal Huntington's Disease.

Designer genes

Now that science can identify the genes responsible for disease, the next frontier is manipulating genes for purposes of gene therapy, cloning and obtaining stem cells.

Gene therapy, where doctors attempt to repair someone's genetic code, is still in its infancy. Most of the experiments have so far been limited to replacing a defective gene with a healthy gene, which cannot be passed on to the patient's offspring. But scientists have also been actively discussing reprogramming human egg and sperm cells, not only treating the patient but passing on the changes to future generations.

Some scientists caution that fiddling with genes could have unintended consequences. For example, sickle cells, which cause sickle cell anemia, block certain kinds of malaria, so eliminating the cells might make some people less resistant to malaria. And no one knows what effect genetic tinkering will have on the gene pool a hundred generations from now. Others fear that parents might try to "design" their children to succeed or perhaps just to fit the fashion. Already there have been calls to ban such genetic manipulation, but supporters say that trying to impede one line of research could inadvertently hamper another.

But for the public and scientists both, the most dramatic advance in genetic research came from a Scottish sheep pen. An embryologist named Ian Wilmut stunned the world in 1997 when he introduced the first mammal cloned from an adult, Dolly the sheep, an exact genetic replica born of an experiment to create more productive farm animals. (Click here to learn how cloning works.)

Scientists in the United States, Italy and South Korea, among others, have publicly announced plans to produce the first human clone. Some of those most eager to explore human cloning are infertility specialists, who view it as another avenue to help their patients. A cloned baby would be an identical twin of one of the parents, only born years later. Still, many scientists around the globe (including Wilmut) have condemned even attempting a human clone as abhorrent, largely because 95 percent of animal cloning attempts fail, and most of those that come to term have severe deformities and die in a week or two. It took Ian Wilmut 277 failed attempts before Dolly was born. Many outside the scientific community have condemned the practice as unethical, and at least 24 nations as well as six U.S. states have banned human cloning.

For many scientists, the promise of medical technology lies with stem cells --
"master cells" capable of replicating indefinitely. That could give doctors the ability to create any body tissue, liberating people from the helplessness of waiting for an organ transplant and the risk of organ rejection. Stem cells are also highly promising for the treatment of ailments like Parkinson's or diabetes. (Click here for a definition of stem cells.)

But stem cells are controversial because of their source. Scientists believe the most promising stem cells come from week-old human embryos, but extracting the cells destroys the embryos. Congress has banned federally funded embryo research since 1995. But the NIH issued new rules in 2000 allowing federal funds to pay for research into the uses of stem cells, so long as no federal money is used to actually remove cells from the embryos. President Bush has gone a step further, announcing he would only fund research into stem cells already extracted from embryos as of August 2001 because he couldn't condone destroying any new embryos. But the compromise has not satisfied either side: Scientists doubt whether the 64 existing stem cell lines worldwide are adequate or healthy enough to be useful, and religious conservatives say any research that relies on embryos is morally corrupt.

Government decisions could have another impact on this research: Some scientists fear a federal ban on cloning would hamper stem cell research because once a useful stem-cell treatment is found, doctors would have to clone the stem cells to create enough of them to treat patients. (Click here for more on the theological debate.) Attempts to pass laws on cloning have stalled in Congress largely because of the debate between those who want a total ban on human cloning and those who want to ban cloning for reproduction but allow the procedure for treating disease.

Public money, private partnerships

Prior to World War II, support for medical research was a largely private undertaking. During the war, however, the federal government for the first time mobilized scientists not only to develop weapons but also to fight diseases threatening troops in a global war. The success of the collaboration persuaded the federal government to fund science in peacetime, resulting in the creation of the National Institutes of Health and the National Science Foundation.

In 1980, the federal government began encouraging academic-industry relationships by creating a uniform federal patent policy, allowing federal grantees to collaborate with commercial interests to promote inventions, and permitting universities to retain the title on inventions developed through government funding.

As a result, as many as 90 percent of life-science companies now have a financial relationship with academia. Corporate licensing of academic inventions account for more than $20 billion of the universities' annual revenue. In most cases, the researchers making the discoveries get some portion of the money.

The financial ties between academia and the corporate world have led some scientists to question whether researchers have -- intentionally or not -- compromised their work, either by overlooking data that may show a drug doesn't work, for example, or rushing research to get a drug patented before a competitor. Others question whether the partnerships that encourage commercial development also discourage the free sharing of information considered vital to science.

Another recent byproduct of the increased privatization of research has been the right of companies under patent law to refuse to allow foreign competitors to create generic -- and often significantly cheaper -- treatments for life-threatening diseases. Poorer countries in Africa, particularly, say they need the less-expensive generic drugs to fight AIDS, leaving the federal government in the awkward position of equivocating between protection of U.S. corporate interests and global humanitarian interests.

The public's viewpoint

The general public, while having a generally favorable view of science and medical research, is unfamiliar with many of the terms and concepts surrounding these issues. Public debate on gene therapy, stem cell research and human cloning, like the research itself, is in its infancy. Some surveys suggest that many people want to distinguish between using gene alteration to treat disease and creating "designer babies" with specific traits. The distinctions extend to human cloning as well. Most Americans say cloning individual organs and body parts is a good idea, but the overwhelming majority say scientists should not be allowed to clone human beings.

Surveys show that more than half of Americans think science and technology make our way of life change too fast. Yet few Americans say they would decrease spending for medical research and most believe they have benefited from it.

Choicework

For more detail on how society could address this issue, visit our Discussion Guide which sets out three alternative approaches to genetic research.

The points of view are drawn both from what the experts say about an issue and from what the public thinks about it, based on surveys and focus groups. We call this section "Choicework." Each point of view comes with the arguments for and against, along with some potential costs and tradeoffs.

• One perspective says that science should be free to pursue genetic research, which could have enormous benefits to humanity.
• A second perspective says that genetic research's potential for treating disease is too great to ignore, but that researchers should draw the line at changes that would be passed on to future generations. Such genetic engineering could have unforeseen and dangerous consequences.
• A third perspective argues that human life is sacred, and by trying to change our genetic makeup scientists are undermining the basic laws of nature and God.