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Source: Business Week

China, with lower regulatory hurdles, is racing to a lead in gene therapy

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.'




Last Updated ( Tuesday, 26 February 2008 18:32 )