|Gene Therapy in China, Promising|
Source: Cancer decidions
This is the first in a multi-part article on a new treatment that is available only in China.
Chinese doctors, some of whom trained at top institutions in the United States, have opened an innovative cancer treatment center at Haidian Hospital in Beijing. The centerpiece of their treatment strategy is a form of gene therapy called Gendicine, a treatment unavailable in any other country in the world. (Company officials have informed me that it is now also being given to foreign patients at a facility in Shanghai, China.)
To date, the Haidian physicians have treated around 200 Chinese nationals and about 70 foreign patients, most of whom came from the US, Canada, and Europe. Although Gendicine has only been formally approved by the Chinese authorities for use in head and neck squamous cell carcinoma (HNSCC), it has also been used experimentally, in the clinical trial setting, to treat cancers of the digestive tract (esophageal, gastric, colon, liver, pancreas, gallbladder, rectum), lung cancer, sarcoma, thyroid gland cancer, breast cancer, cervical cancer, and ovarian cancer. In addition, advanced cancer patients with no other feasible avenues of treatment are being allowed, on a case-by-case basis, to receive the new drug.
The development of Gendicine, and the willingness to provide it to non-Chinese patients, caught most foreign observers by surprise. "I had no idea that this was going on in China," said Alan Kingsman, CEO of Oxford BioMedica, a British firm that makes a competing product. "Initially I didn't understand how this could have happened first in China and not in the US. But as one looks at the story, it's hard to find anything wrong."
"This is a wake-up call to America," said Mark Kay, president of the American Society of Gene Therapy (ASGT) and director of Stanford University School of Medicine's gene therapy program. "We need to look at some of the regulatory hurdles, and the funding issues: right now, funding for biomedical research in the US is really hurting, and it's short-sighted to think this doesn't hurt our economy in the long run."
Because of Gendicine and other innovative treatments, China is quickly becoming a destination for international patients seeking innovative and unconventional treatments for cancer that are not yet available in the famous medical facilities of the West. "Even in these countries, oncologists and cancer centers can't do anything more for them," said Li Dinggang, MD, the surgical oncologist who heads the Haidian center, referring to patients traveling to Beijing for treatment. "That's the point at which patients contact me," The Haidian hospital uses conventional radiation and chemotherapy as well as innovative treatments such as hyperthermia and high intensity focused ultrasound (HIFU). But the primary source of excitement is clearly the new gene therapy treatment, Gendicine.
This treatment has received increased publicity in recent months, including a highly positive article in Business Week (March 6, 2006). This article will probably lead to an increase in the number of cancer patients who will flock to China for last-ditch treatment. Dr. Li has called Gendicine a "milestone on the order of penicillin" in the treatment of cancer. Used in connection with chemotherapy and radiation, he claims that Gendicine has added years to the lives of patients and simultaneously improved their quality of life.
Such enthusiastic endorsements from medical practitioners are not uncommon, and they don't always stand the test of scientific scrutiny. But is it possible that in this case the enthusiasm is warranted? Perhaps. Let us therefore look more closely at gene therapy and Gendicine.
Gene Therapy Coming of Age
Gene therapy is a type of treatment that is aimed at manipulating specific genes within a patient's cells. In some forms of cancer, for example, a particular tumor suppressor gene called p53 is often thought to be defective. During gene therapy, engineered forms of the normal gene are reintroduced into the patient's tumor cells, with the goal of correcting the deficiency and restoring cellular function to normal. In about half of all human tumors, this crucial tumor suppressor gene is mutated and cannot carry out its normal activity of restraining cancerous growth.
Gendicine is made up of two components:
1) Normally functioning p53 genes
2) An adenovirus carrier or "vector" to transport this p53 into cancer cells.
Dr. Zhaohui Peng, whose company, the Shenzhen SiBiono GeneTech Co., produces the drug, has defined Gendicine in more technical terms as "a replication incompetent, recombinant, human adenovirus of serotype 5 engineered to contain the human wild-type p53 tumor suppressor gene" (BioPharm International 2004). In 2003, SiBono made history when China's State Food & Drug Administration (or SFDA) approved Gendicine for use in human cancer patients. Gendicine thus became the world's first commercially available gene-therapy drug, years ahead of any competitor.
In essence, there is nothing terribly sophisticated about the idea behind Gendicine. Doctors are injecting it directly into tumors with the aim of restoring normal p53 restraints on growth, thereby rendering cancer cells less virulent.
Opening of Clinic
The cost for patients coming from abroad for the two-month gene therapy treatment in Haidian is USD $20,000 and since this is an experimental treatment, it is generally not covered by US health insurance. This is expensive, but no more so than some of the standard 'targeted' drug therapies now being offered in Western hospitals. SiBono claims that more than 130 hospitals in China are now performing Gendicine treatment. One can theoretically inquire about it at virtually any of the major hospitals in China. However, treating foreigners can be a daunting task (not least because of language problems) and so most non-Chinese patients will probably want to go to Haidian or one of the few other clinics that are really set up to handle English-speaking clients.
The burgeoning of Chinese clinics that welcome foreign patients is an interesting development in what has become known as "medical tourism." The treatment offered at Haidian Hospital, for example, straddles the divide between conventional and alternative cancer approaches. On the one hand, the treatment is conventional in that it is government approved and the personnel involved are for the most part conventionally trained. The chairman and CEO of SiBiono, Dr. Peng Zhaohui, studied gene therapy in Japan and the US. Business Week has called him "the father of gene therapy in China." He also trained at the University of California, Los Angeles (UCLA), and has chaired a biological chemistry lab in Guangzhou. He established SiBiono in Shenzhen in 1998.
The director of the Gene Therapy Center at Haidian Hospital, Li Dinggang, MD, is an oncologist who in the 1990s spent five years as a research fellow at Johns Hopkins University, Baltimore. These people are certainly not your typical practitioners of complementary and alternative medicine (CAM). On the other hand, the program they are offering is clearly designed to attract foreign patients; the drugs and techniques they are using are largely unavailable (and unapproved) in Europe and the US, and the price they are charging for the protocol is fairly steep and must be paid for out-of-pocket by the patient. Their clinical trials have been conducted without the participation of better known facilities in Europe, Japan or the US. And they make what, at first sight at least, seem like premature and exaggerated claims of benefit.