Once a week, Hashmukh Patel, a 62-year-old retired semiconductor
engineer from Silicon Valley, travels with his wife, Bena, from their
Beijing hotel to Beijing-Haidian Hospital. They ride the crowded
elevator to the ninth floor, enter a pleasant, sun-filled ward with
private rooms, and Patel gets an injection that he hopes will save his
life. Suffering from late-stage cancer of the esophagus, he has come to
Beijing for a Chinese gene-therapy drug called Gendicine that's supposed
to kill tumor cells.
Patel tried just about everything before coming to China. He did three
months of traditional chemotherapy, flew to the Bahamas for treatment at
an alternative healing center, and tried to find clinical trials of
experimental drugs. Nothing panned out. By late 2005, his doctors said
that additional surgery or chemo could bring him only a few more months.
That wasn't good enough. "I'm not interested in buying time," says
Patel, sitting on a couch at Haidian and holding his wife's hand. He had
heard about Shenzhen SiBiono GeneTech Co., the producer of Gendicine,
which in 2003 became the world's first commercially available
gene-therapy drug. But the treatment, which is approved and available
only in China, costs $20,000 per two-month course and isn't covered by
U.S. health insurance.

SETBACKS IN THE U.S.
Patel is one of 70 foreign patients from 22 countries who have sought
gene-therapy treatment at Haidian from Dr. Li Dinggang in the past year
and a half. Li, the 50-year-old director of the Gene Therapy Center, is
an oncologist who spent five years as a research fellow at Johns Hopkins
University in the 1990s. Since 1997, he has worked closely with Dr. Peng
Zhaohui, the chairman and chief executive of SiBiono, seeking to make
China a leader in the field of gene therapy.
In the West, this experimental branch of biomedicine suffered major
setbacks following the death of one patient in a clinical trial in 1999.
Other patients later came down with cancer as a result of their added
genes, and the U.S. Food & Drug Administration halted a number of
trials.
That created an opportunity for Chinese researchers. Without the same
regulatory obstacles, they were able to take ideas that originated in
the U.S. but stagnated there. SiBiono's Gendicine, for instance, is
similar to a gene therapy treatment that was pioneered by Introgen
Therapeutics Inc. (INGN) in Austin, Tex., but has yet to win approval
from the FDA. As safety concerns forced the U.S. and Europe to apply the
brakes, "China speeded up," Li says.
The Chinese are determined to keep the momentum going, and believe they
can avoid the pitfalls that beset South Korea in stem cells. On Feb. 9,
China's State Council announced a big boost in research and development
spending to 2.5% of gross domestic product, from 1.3% now; biotech is a
top priority. Li estimates that two dozen Chinese companies are
developing gene-therapy drugs. "The Chinese are being very aggressive on
this," says Dr. Jack A. Roth, chair of thoracic and cardiovascular
surgery at the University of Texas M.D. Anderson Cancer Center in
Houston. "They see a window of opportunity."
As a result, China has attracted not just American patients but also
American researchers. James S. Norris, chair of microbiology and
immunology at the Medical University of South Carolina, is president of
the International Society for Cell & Gene Therapy of Cancer, which last
December had a conference in Shenzhen, China. "If I were making a
long-term investment in biotech, and particularly in gene therapy, I
would be making it in China, not here," Norris says. "They have figured
out how to get [gene therapy] approved." Norris is now trying to get
funding to test his own promising gene therapy approach in China.
The father of gene therapy in China is SiBiono's Peng. Born in 1952 in
the northern Chinese province of Shanxi, Peng has a joint PhD from a
university in Xian and from Japan's Chiba University. Like Haidian's Li,
he's a returnee from America, having worked at the medical school at the
University of California at Los Angeles and at a biotech startup in San
Diego in the mid-1990s. He launched SiBiono in 1998 with $300,000 in
seed money from the Shenzhen government. Since then, SiBiono has
received $5 million from private investors and more than $6 million in
government grants.
Gendicine itself combines a gene named p53, which suppresses tumor
formation, with a modified common virus. When the product is injected
into a tumor, the virus carries the gene into cancer cells. The gene
then prompts the tumor cells to commit suicide.
This basic approach was first developed by Introgen. The U.S. company's
CEO and president, David G. Nance, believes that his patents cover
SiBiono's treatment -- a view Peng disputes. Both companies have gotten
similar results in clinical trials. Used with radiation or other
treatments, the added gene causes more tumor shrinkage than the older
treatments used alone. It remains to be proven that the treatment
extends life, however, even though some patients have lived longer than
expected. Another problem: While the gene can be directly injected into
tumors that doctors can see, more clever delivery methods are needed to
get it into metastasized cancer cells.
Despite questions about the technology's ownership and effectiveness,
Peng has big plans for the privately held SiBiono. Construction is under
way on a $20 million facility on the outskirts of Shenzhen that will be
able to produce 1.5 million doses of Gendicine a year, compared with
just 180,000 now. Peng says the company is talking with foreigners about
potential partnerships. China is a logical place to work because more
than 2 million Chinese get cancer every year. Clinical trials are
inexpensive, at about one-tenth the cost in the U.S. And the regulatory
climate is favorable. "The Chinese government is more open to innovation
and new ideas, compared to the foreign counterparts such as the FDA in
America," says Peng.
"WE ARE ALL ANXIOUS"
Some people wonder if Chinese regulators should have required longer
trials before approving Gendicine. "Maybe they went into this
prematurely," says Dr. Bauer E. Sumpio, chief of vascular surgery at
Yale University School of Medicine. "It is hard to believe this would
pass muster through our own FDA." Adds Takeo Ohnishi, a professor at
Japan's Nara Medical University: "After the [problems] linked to gene
therapy in the U.S, nobody wants to have anything to do with such
treatments here."
Even in China, not everyone is sanguine. Cheng Jing, 42, is CEO of
CapitalBio Corp., a state-backed biotech company in Beijing that is
developing gene therapy. He fears that if Gendicine turns out to be
ineffective, people will lose faith in all gene therapy. He says that he
would feel more confident if there were test results from independent
doctors treating patients in the years since the commercial launch. "We
are all anxious," says Cheng.
Doctors close to SiBiono insist that China's first gene therapy drug is
both safe and effective. Haidian's Li admits there are risks, but argues
that's true for all other cancer treatments. "Surgery is risky," he
says. "Chemotherapy is risky. Radiotherapy is risky." Gene therapy, he
says, "can be used effectively to treat patients."
And thousands of patients, like Hashmukh Patel, are willing to take the
chance. His decision to travel to China was not hard, he says. "I didn't
think there was an alternative.'

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