Wed, Jul 20, 2005 page12

• Twist of fate drives biotech pioneer

  • AP
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  Brad Margus was making a killing in the shrimp business and living happily in South Florida with his wife and three young boys. Then his two youngest were diagnosed with a fatal genetic brain disorder that's as rare as it is unpronounceable.

  It's called ataxia-telangiectasia, better known as "A-T," and it afflicts about 500 youngsters in the US.

  Since doctors told Margus in 1993 that his toddlers Jarrett and Quinn would soon be confined to wheelchairs and probably dead before they were old enough to vote, the Harvard Business School graduate has been on a mission to save the boys from their genetic fate.

  

  Brad Margus is seen in an office at his company Perlegen Sciences in California earlier this month. Margus was making millions in the shrimp business when his two toddlers were hit with a rare brain disorder that usually ends in premature death. So he sold his company and has embarked on a quest to save his sons from their genetic fate.

  PHOTO: AP

  In the process, he has become a self-taught genetics expert, a lobbying fixture in Washington, and head of his own startup biotech company, making him a learned colleague alongside the field's leading scientists.

  Margus has helped raise US$17 million for the nonprofit patient advocacy group he created called the A-T Children's Project, and he sits down for an occasional national television interview with the likes of Barbara Walters.

  "It really is like a Forrest Gump story in that this shrimp guy with no science background started to get to know a lot of people who today are luminaries," Margus said.

  Perhaps most important, his quest led him to sell his South Florida shrimp processing plant and launch Perlegen Sciences Inc in 2000. Margus is chief executive of the Mountain View biotechnology company, which is on the vanguard of the "personalized medicine" movement.

  Adherents of personalized medicine are attempting to create treatments tailored to individual genetic make-ups.

  Humans' DNA is comprised of 3 billion chemical bits called nucleotides. Each of the 25,000 or so human genes are typically comprised of about 1,000 nucleotides.

  Some 99.9 percent of the nucleotides are identically arranged in all of us. It's the 0.1 percent difference that makes each of us unique -- and causes disease in some.

  One vital gene in Margus, for example, is missing one nucleotide found in nearly every other human on the planet. His wife has a similar defect. Two of their children ended up with each parent's mutant gene -- and A-T. (A fourth son, born after the A-T diagnosis, dodged the genetic bullet and is healthy).

  Personalized medicine's goal is to find and exploit these genetic quirks to better develop drugs beyond the pharmaceutical industry's one-size-fits all system. This approach has been kicked about for decades, but the field remained a scientific backwater until former US president Bill Clinton announced in 2000 that every gene in the human body had finally been mapped.

  Almost overnight, dozens of companies backed by enthusiastic venture capitalists popped up, all promising to soon use the human gene map to make personalized medicine the norm.

  But the bottom fell out soon after. The dramatic breakthroughs didn't come as quickly as hoped and most of the startups are either gone or have changed their focus.

  That's because making tailored-made drugs isn't as simple as it initially seemed. Yet, five years of incremental advances have brought personalized medicine back to respectability. Company scientists have recently published 1.6 million genetic differences sussed from the DNA of 71 Americans of European, African or Chinese ancestry. Now the trick is to determine what those variations mean.

  Perlegen's persistence through the false starts and bad times has made it the sector's leader. Its investors have quietly poured US$206 million into the company over the last five years and they are expected to reap handsome profits if Perlegen goes public by next spring as Margus hopes.
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