BEING nice gets you nowhere with politicians and big business. It鈥檚 financial
clout that counts or, failing that, kicking up a stink. It was the latter path
that a group of New York AIDS activists took in 1987. ACT UP stopped AIDS
conferences in full swing, disrupted speeches by politicians and marched on the
US National Institutes of Health. The group鈥檚 message was simple: drugs
companies and AIDS researchers were serving their own ends and ignoring the
needs of people with HIV.
As demonstrators blew whistles and chanted on the streets, other AIDS
activists got stuck into the science. They took on researchers and regulators.
They pushed for changes to clinical trials鈥攕o that they included women,
ethnic minorities and drug users, for example鈥攁nd for experimental drugs
against HIV to be speeded through the licensing system.
In the end, the activists got their way鈥攁 place at the table where
patients鈥� voices could be heard. And many AIDS researchers now admit that the
socially sensitive trials proposed by the activists improved the quality of
their science. People power worked by making a big stink.
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Fast forward 14 years to a group that鈥檚 taking the other tack. PXE
International was set up to support families of people who suffer a rare genetic
disorder called pseudoxanthoma elasticum. What sets the group apart is that it
has applied for patents that would give it control of a diagnostic test for PXE
and mouse models of the disease
(see 鈥淗ead to head鈥�).
With these in hand, it plans to lever support from companies and politicians.
PXE International is the brainchild of Americans Patrick and Sharon Terry,
whose children have the disease. In the 1990s, they set up blood and tissue
banks and convinced patients with PXE to donate samples. These, they offered to
scientists on the strict understanding that PXE International would receive a
stake in any intellectual property that emerged. Fortunately, enlightened
researchers and universities accepted their offer. Last year, when the gene for
PXE was discovered with help from the group鈥檚 samples, PXE International took
control of the patent rights.
What, you may ask, is the point? The disease affects only 1 in 100,000
people, and no company is going to invest heavily in developing drugs that will
sell to only a relatively few customers. Here, the Terrys have a plan. The PXE
gene is implicated in wrinkling of the skin and in heart disease, areas that
cosmetics and drugs companies are falling over themselves to research. By
offering licences to companies that want to work in these areas, the Terrys hope
to learn more about the disease process underlying PXE and, potentially, earn
money for PXE International.
The ultimate pay-off, though, is not financial but practical help for people
with the disease. They hope to get a diagnostic test and prenatal diagnosis out
to people as quickly as possible. They want to ensure that test results come
with genetic counselling, so that families understand the implications of the
tests. They also want to control the speed and direction of research into
treatments. PXE International has already helped academic researchers to attract
more than $4 million in NIH grants.
Contrast this with what support groups for other genetic diseases are doing.
Many have never even considered banking tissue samples or taking a stake in the
intellectual property that arises from research on the people they represent.
Others have taken up the long-term challenge of finding a cure for a disease
and, though successful in raising funds to support research, have neglected the
needs of people who live with the disease today.
In this sense, PXE International鈥檚 goals are closer to ACT UP鈥檚. But where
the AIDS activists worked noisily outside the system, PXE campaigners are
working within it. Mind you, the PXE group is now starting to make a noise. It
has given testimony to Congress on the need for more biomedical research and is
lobbying the US Food and Drug Administration to make the drug licensing system
cheaper. It is also adding its voice to those of other genetic interest groups
to present a global united front.
鈥淣ever doubt,鈥� said the American anthropologist Margaret Mead, 鈥渢hat a small
group of thoughtful committed citizens can change the world.鈥� Just as cancer and
diabetes groups learned from ACT UP how to use direct action to create change,
so PXE International is taking its method to other support groups. It deserves
to work. With luck, it will.
