Your DNA or Your Life?
Gottlieb, Scott
Your DNA or Your Life? Drug science goes digital BY SCOTT GOTTLIEB T hanks to the Human Genome Project's tsunami of data, a startling transformation in drug science is about to...
...Not so in other countries...
...Researchers won't be able to go back to ask new questions or acquire additional tissue samples as new clinical hypotheses arise...
...Digitizing the data brings the study of disease to an entirely new level, exploiting the power of computers and the mathematics of probability in a brand-new science called bioinformatics...
...Oncologists would have immediate access to clinical information on the treatment and prognosis of patients who have tumors matching their own patients...
...Anonymous patient data is a fixed set...
...Turns out, many Mormons are...
...To sleuth out the associations that cause disease, scientists will build better software and faster computers...
...When Myriad scientists thought they were closing in on a colon cancer gene, for example, they needed new samples obtainable only by colonoscopies...
...Researchers can get DNA from blood samples but, to understand how genes cause disease, they need tissue samples that reveal how that DNA sequence is expressed in bodily functions...
...Maybe lawless countries with a little technology and even less concern for privacy rights will make these data troves a lucrative export...
...But then you'll need a large DNA database to answer the question: Did everyone with that sequence go on to develop cancer or diabetes...
...Almost everyone who rolls into a hospital with heart failure gets Enalapril, a drug groups based solely on variations in DNA sequences and then to develop the targeted drugs most likely to work for them...
...And not only Mormons benefit...
...How many people are willing to do that for medical science...
...If the goal is to find the mutations coding for diseasetriggering gene products (like proteins or enzymes), then you want to compare genetically diverse populations in which at least a few people are bound to have a particular sequence...
...HE AMERICAN SPECTATOR 0 Summer Reading Issue 2001 Drug discovery is now a computer-based information science, driven by a handful of companies that have already amassed impressive databases...
...Poverty...
...Scientists scan these databases looking for DNA "markers" or random segments of DNA that vary slightly in sequence between different people...
...Thanks to the success of Celera Genomics and the Human Genome Project, drug discovery is now a computerbased information science, driven by a handful of companies that have already amassed impressive databases such as DoubleTwist, Incyte, Human Genome Sciences, Gemini Genomics PLC, and Celera...
...Researchers complain loudly in scientific journals that most Americans just aren't interested in parting with their DNA...
...And unlike most others, Myriad's database subjects are willing to participate in follow-up studies.This is no small thing...
...But even this valuable database is not the mother lode...
...Myriad Genetics thinks it has the answer: Mormons...
...Once you understand not only that this particular DNA sequence leads to disease, but how it does, you have a powerful new potential drug strategy: make a replacement for the mutated dysfunctional enzyme, for example, or create an antibody to neutralize a deadly protein...
...Iceland has licensed this information to DeCODE Genetics, a private biotechnology company...
...17 diseases...
...There's just one catch.Americans don't like divulging information about their health or their genes, especially if the information is going to lurk inside a big computer where employers or insurers might get it...
...The hope is that patients and their families will feel particularly inspired to provide tissue samples and fill out lengthy questionnaires to help cure their own or a loved one's disease.A group of biotechnology companies, dubbed the International Genomics Consortium (IGC), for example, links tumor gene profiles from malignant biopsies with anonymous clinical data on thousands of cancer patients, giving scientists a quick way to see if a gene implicated in one cancer is also active in others...
...The next step is to analyze tissue samples from patients to understand how that particular DNA sequence triggers disease...
...What kind of Americans will continuously donate DNA for medical science...
...DeCODE recently used its powerful database to isolate genes linked to diabetes and stroke...
...DNA sequences are chnically meaningless unless correlated with patient disease and lifestyle profiles...
...In a recent issue of the New England ournal of Medicine, researchers think they've spotted a medical answer...
...Lack of insurance...
...Why are black patients with heart failure more likely to die...
...Racism...
...Suppose you came upon a fragment of DNA that causes a particular cell to divide without the normal growth restraints (looks like cancer...
...Biochemical lab reactions can isolate what sequence of that DNA stimulates riotous cell division...
...One-drug-fits-all development strategies may soon look as outdated as one-color automobile manufacturing, as mass drugs give way to miracle drugs tailored to fit the individual patient's DNA profile, or that of the patient's family, or at the very least, his ethnic group...
...To do that, medical researchers must assemble DNA from thousands of people into powerful databases that can be mined with sophisticated software that correlates an individual's DNA patterns with particular THE AMERICAN SPECTATOR 0 Summer Reading Issue 2001 Medical researchers must assemble DNA from thousands of people into powerful databases that can be mined with sophisticated software...
...Mapping the human genome was only the first building block of a huge DNA edifice medical researchers now strive to erect...
...The Salt Lake City company has rights to the meticulous genealogy records of Utah Mormons, whose generations of recorded family health care histories help researchers spot potential genetic predisposition to disease...
...Myriad's powerful database allowed Salt Lake City researchers using DNA from Utah Mormons to uncover a breast cancer gene predominantly afflicting European Jews...
...Coupled with DNA samples from living descendants, the powerful database allowed Myriad Genetics to race ahead of other, betterfunded groups to uncover the gene linked to breast cancer, known as BRCA1...
...But Iceland's massive database is less than ideal...
...t 18 THE AMERICAN SPECTATOR 0 Summer Reading Issue 2001 Drug discovery is now a computer-based information science, driven by a handful of companies that have already amassed impressive databases...
...Bull's-eye...
...Iceland has a national government database that includes DNA descriptions from a majority of the nation's families, available in easily accessible electronic form, accompanied by a codified promise of donor privacy...
...Drug science goes digital BY SCOTT GOTTLIEB T hanks to the Human Genome Project's tsunami of data, a startling transformation in drug science is about to take place...
...The bottleneck won't be Americans' ability to use these advanced tools to manipulate DNA data, it will be our willingness to collect it...
...If those who inherit the markers always develop the disease, and the location of the marker in the DNA sequences is known, then you can safely infer the disease-causing gene must also be in the same spot...
...In America, scientists have all but given up on broad national DNA databases in favor of disease-specific ones...
...Icelanders display relatively little genetic variation...
...or a DNA sequence that produces a protein that retards insulin effectiveness (looks like diabetes...
...Similar national initiatives are being pursued in Estonia, Newfoundland, China, Singapore, and Tonga, among others...
Vol. 34 • July 2001 • No. 6
