Introduction

44 BIOHYPE
a patient to make him better I wanted to try and purify glucocerebrosidase
[the missing enzyme in Gaucher disease] from some human source,
to reduce the possibility of humans rejecting it when it came in.” One
night, while out to dinner with the father of two children with Gaucher
disease, it came to him in a flash. Why not human placentas They were
fresh tissues and would likely have higher than normal concentrations of
the rare proteins. It took another half-decade to work out the procedure
for purifying the enzyme from the placentas. Brady and a team of scientists
spent nights and weekends liquefying placentas with a hand-cranked
grinder in the cold room next door to their lab. NIH would get a patent
for the process in 1975. 6
Two years before the patent was granted, Brady and his team had
enough enzyme to attempt their first clinical trial. But tests on several
patients had spotty results, largely because they didn’t have enough
enzyme to maintain large enough doses over a long enough period of
time. They also learned that something occurred to the enzyme during
purification that made it difficult to absorb. It wasn’t getting into the
cells that had built up excess levels of lipids. 7
Brady and his colleagues solved the first dilemma by seeking outside
help in purifying the enzyme. They contracted with Henry Blair, who had
worked at NIH with Brady but left to form the New England Enzyme
Center at Tufts University Medical School in Boston. Supported solely by
contracts from Brady’s lab, Blair set up a lab for large-scale purification
and began collecting fresh placentas. In 1981, with NIH getting ready to
move into larger clinical trials and biotechnology fever exploding all
around him, Blair privatized his venture. He launched Genzyme, with the
NIH contract as its major source of revenue.
But just as the company was getting off the ground, the new venture
stumbled. Animal experiments showed the enzyme wasn’t getting to the
cells where the excess lipids were stored. Brady assigned several
researchers to the problem, including Barranger and Scott Furbish, who
later went to work for Genzyme. They discovered that the purification
process stripped away the end of the enzyme that stuck to the lipidstoring
cells. So they developed a process for restoring the sticky end of
the purified enzyme and gave it to Genzyme. The company was back in
business. Over the next decade, Genzyme received nearly $10 million in
contracts to produce the enzyme for NIH, giving the start-up company a
major lift in its formative years. Blair eventually hired a young economist
named Henri Termeer to run the firm. A 1992 study by the Office of
Technology Assessment (OTA) estimated that at least a fifth of all the