E-drug: Forbes article on OneWorld Health
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Protecting the Orphan Drugs
Zina Moukheiber, forbes.com
Victoria Hale worked for the Food and Drug Administration in the
early 1990s reviewing drug proposals. She remembers seeing dozens
of experimental drugs that seemed to have great potential to cure.
Yet pharmaceutical companies didn't pursue them--the market wasn't
profitable enough to justify the cost. "It was frustrating," says Hale. "I
wanted to find a way to separate business decisions from clinical
decisions."
She founded the San Francisco-based Institute for OneWorld Health,
which bills itself as the first nonprofit pharmaceutical company. "It's
the ultimate oxymoron," says James McKerrow, a tropical diseases
expert and a OneWorld scientific adviser.
OneWorld is developing drugs that treat diseases the pharmaceutical
industry has abandoned--specifically parasitic illnesses that plague
the developing world.
Drugs need to cost less than $1 to make them affordable for poor
populations. At that price, drug companies can't recoup the more than
$100 million they typically spend to develop these so-called orphan
drugs. As a result, a flurry of partnerships among drugmakers,
charitable foundations and nonprofits have formed to fill the void,
including the Global Alliance for TB Drug Development and Medicines
for Malaria.
OneWorld Health has obtained all of its funding so far, $4.7 million,
from the Bill & Melinda Gates Foundation. It employs 15 scientists,
including chemists, microbiologists and biostatisticians. Hale's
husband, Ahvie Herskowitz, a cardiologist, serves as chief operating
officer, and oversees clinical trials as the chief medical officer.
A pharmaceutical chemist by training, the 42-year-old Hale set out to
build an organization in a methodical way. While at the FDA, she
made a checklist of six neglected diseases, from substance abuse to
rare orphan diseases, before deciding to pursue tropical illnesses.
Five years at the FDA gave her insight into regulations, but Hale
wanted a corporate stint too. She joined Genentech's drug
development team in 1994, where she played a part in such
successful drugs as Herceptin for breast cancer.
In 1998 she quit. At her first trip a year later to the World Health
Organization in Geneva to learn about its efforts in tropical diseases,
she got the cold shoulder. "The first question was 'You're an
American, why do you care about parasitic diseases?'" she recalls. "It
hurt my feelings."
By her third trip, Hale had overcome the skepticism. "This was a lady
worth talking to," says Winston Gutteridge, former head of R&D for
tropical diseases at WHO, and now a scientific advisor to OneWorld.
OneWorld's first drug was paromomycin, an injectable antibiotic, to
treat visceral leishmaniasis, a deadly parasitic infection that afflicts
1.5 million people primarily in India, and kills 200,000 people each
year. WHO had gotten the rights to the injectable form of
paromomycin from Pharmacia--now Pfizer. It had completed
mid-stage trials, but had shelved the drug due to a tight budget. Hale
got an agreement from WHO to take the drug through late-stage
trials, meaning OneWorld would design the trials, analyze the data
and submit it to the Indian government for approval.
WHO and OneWorld now jointly own the license to the injectable form
of paromomycin. Hale hopes to complete trials by year-end. If
successful, she could have paromomycin on the market by 2005. She
is talking to two Indian generic drug companies about manufacturing
the drug, and to a Dutch nonprofit drug distributor. Her aim is to sell
paromomycin for under $1 a day.
Last year OneWorld got an exclusive license for free from Celera
Genomics for a compound to treat Chagas disease, a bug that
causes heart failure and is prevalent in Latin America. Wayne
Montgomery, who heads intellectual property at Celera, admits the
drug would have gathered dust otherwise. Celera will not receive
royalties. OneWorld is testing the drug on lab animals.
Hale's biggest challenge is keeping her coffers full, since her drugs
are not likely to fund the nonprofit. Drug development remains costly,
and the risk of failure is high. She estimates it will cost her $7 million
to complete trials of paromomycin. She has gone back to the Gates
Foundation for more than $30 million to fund this and other projects.
What's not lacking are drugs to test. In May OneWorld signed a letter
of intent with PS Pharmaceuticals in San Francisco to develop a
pediatric diarrhea drug. In July, it announced it licensed another drug
candidate for Chagas disease from Yale University and the University
of Washington. Hale is also reviewing drugs for malaria, intestinal
worms and sleeping sickness. In all she's been e-mailed more than
120 proposals from scientists at pharmaceutical companies and in
academia.
Michael S. MacHarg
Associate, Development and Partnerships
Institute for OneWorld Health
580 California Street, Suite 900
San Francisco, CA 94104
USA
tel: +1-415-421.4700
fax: +1-415-421.4747
www.oneworldhealth.org
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