[Ip-health] New York Times: Billionaires With Big Ideas Are Privatizing American Science

Thiru Balasubramaniam thiru at keionline.org
Sat Mar 15 22:55:36 PDT 2014



A Focus on Disease

If the map of the world of private science has yet to be drawn, one thing
is clear: Much of the money is going into campaigns for a cure.

This private war on disease has resulted not only in significant advances
in treatment, but also in what experts describe as a major breakthrough in
how biomedical research is done. The method opens up blockages that have
traditionally kept basic discoveries from being turned into effective
treatments -- especially for rare diseases that drug companies avoid for
lack of potential profit.

"We think it's potentially transformative," said Maryann P. Feldman, a
professor of public policy at the University of North Carolina at Chapel
Hill who studies the approach.

The first success came with cystic fibrosis, which arises when a faulty
gene clogs the lungs and pancreas with a sticky mucus. People with cystic
fibrosis suffer from coughing, fatigue, poor digestion and slow growth, and
die relatively young.

Around 2000, a surge of wealthy donors began making large contributions to
the Cystic Fibrosis Foundation. Tom and Ginny Hughes of Greenwich, Conn.,
had two daughters with the disease, and gave millions of dollars. The
family also posed in
the foundation's "Milestones to a Cure" updates, and Mr. Hughes, a banker,
helped the charity develop strategies to expand its fund-raising.

Year after year, the foundation held galas, hikes, runs and golf
tournaments, eventually raising more than a quarter-billion dollars. With
great skill, it used the money to establish partnerships across industry
and academia, smashing through the walls that typically form around
research teams.

By early 2012, the financial surge produced the first
an underlying cause of cystic fibrosis. The drug counters a gene mutation
that accounts for 4 percent of the cases in the United States -- about 1,200
people. The medication thinned the deadly mucus, lessening symptoms and
drastically improving quality of life.

The success begot a global rush to turn basic discoveries into treatments,
a field now known as translational science. It also inspired rich donors to
shower new money on disease research.

Many of their efforts are rooted deep in personal or family trauma.
Sometimes, by sheer force of genetics and demographics, that impulse may
risk widening historical racial inequalities in health care and disease
research, disparities that decades of studies have shown to contribute to
higher rates of disease and death among blacks, Hispanics and other
minority groups.

A review of these campaigns finds that, as with cystic fibrosis -- which mainly
strikes people<http://www.lung.org/assets/documents/publications/solddc-chapters/cf.pdf>
Northern European descent -- a significant number are devoted to diseases
that disproportionately affect white people.

Ovarian cancer strikes<http://www.cdc.gov/cancer/ovarian/statistics/race.htm>
kills white women more often than minority women. In 2012, after his
sister-in-law died of the disease at age 44, Jonathan D. Gray, the head of
global real estate at the Blackstone Group, the private equity firm, gave
the University of
million to set up a center to study female cancers.

Melanoma, the deadliest of skin cancers,
strikes and kills whites preferentially. Debra Black, wife of the financier
Leon Black, survived a bad scare. Soon after, the couple teamed up with
Michael R. Milken, the former junk-bond financier, whose charity
FasterCures gives advice on how to accelerate research, to found the
Melanoma Research Alliance. It quickly became the world's largest private
sponsor of melanoma research, awarding more than $50 million for work at
Yale, Columbia and other universities.

Of course, the pervasiveness of most diseases means most philanthropists
give comfort and medical relief across the lines of race and ethnicity.
When Mr. Milken, for example, learned that he had prostate cancer, he set
up a foundation to fight it. The charity has raised more than half a
billion dollars, helping save not only him but also many black men, since
they develop the disease more frequently than white men do.

So, too, the techniques of translational science, inspired by philanthropy,
are now being applied in a federal effort against sickle cell anemia, a
blood disorder that mainly strikes black
people<http://www.cdc.gov/ncbddd/sicklecell/data.html> and
has long been something of a research orphan.

Scientists first described sickle cell anemia in 1910 and uncovered its
genetic basis in 1949. The discovery, by a team that included Linus
Pauling, a Nobel laureate twice over, was central to the creation of the
field of molecular medicine. Yet with little financing for sickle cell
research, either public or private, no drug has been developed that targets
the disease's underlying cause, even though it has crippled and killed
millions of people.

The government effort began with Dr. Collins, the N.I.H. director, who as a
biologist had helped uncover the cystic fibrosis gene. As the new cystic
fibrosis treatment emerged, he pressed the government to adopt the
breakthrough translational method, federal budget cuts notwithstanding.
Today, the N.I.H. translational science center <http://www.ncats.nih.gov/> has
an annual budget of more than $600 million and seeks new drugs for rare
diseases, which number in the thousands.

Dr. Collins, who works with many wealthy donors, said the government was
trying to level the playing field rather than rush off to where
"everybody's already piled up effort." An effective treatment for sickle
cell disease, he said, has "been a long time coming."

A candidate drug is undergoing clinical trials and looks promising. In
December, the company working with N.I.H. on the research effort announced
that a single dose<http://www.massbio.org/news/member_news/721-aesrx_presents_phase_1_2a_trial_data_of/industry_news_detail>
a "significant reduction" of pain for up to 24 hours.


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