Murrieta resident finds cancer treatment half a world away

By: JOSE CARVAJAL - Staff Writer | Sunday, November 27, 2005 9:14 PM PST ∞

MURRIETA ---- The constant hiccuping, at first, was nothing more than a minor inconvenience.

When it turned out that it was a symptom of something serious ---- esophageal cancer, one of the rarest forms of the disease ---- Carla Pechous wasn't sure what to make of it.

"I just thought it was weird," she said of her reaction on the day in July 2004 when doctors told her the cancer wasn't curable, only treatable. "I was completely shocked. It was not anything I ever thought I'd have. Sometimes, I feel like I'm living someone else's life."

Not only did the 68-year-old Murrieta resident have to come to grips with the fact that she has cancer, but, as it aggressively spread, she also had to figure out how she was going to deal with it.

After almost a year of trying several different treatments with little success, Pechous finally found one that she believes worked in an unexpected place ---- China.

The retiree and her husband, Don, recently returned from a six-week stay at Beijing Haidian Hospital, where she received regular doses of a gene-therapy treatment called Gendicine. The results so far have been positive, Pechous said, and since she had the treatment, her local doctors are saying that the cancer hasn't spread.

Through the therapy, doctors are able to isolate genes in a lab and insert them into cells that have defective copies of the gene or are missing them. Genes, which are made up of DNA, dictate the characteristics a person has. Defective or missing genes can lead to cells becoming cancerous.

Scientists believe that inserting normal genes into cells with bad copies of the genes is the key to treating all kinds of diseases and not just cancer.

Gendicine can be administered in a variety of ways, including intravenously and through chemotherapy. Pechous received the treatment through an IV.

Though Pechous' treatment has so far proven to be successful, the trip to Beijing cost upward of $20,000. Pechous says she's a little frustrated she had to go halfway around the world to find a successful treatment for her cancer.

Though research into gene therapy's potential to cure disease began in the United States more than 30 years ago, it still hasn't been approved as a treatment by the Food and Drug Administration.

At this point, only a limited number of participants in experimental studies are allowed to receive it and that leaves many who see the therapy as being the answer to curing their diseases little choice but to head out of the country to get it.

Gene therapy developed nearby

Researchers not too far from Southwest County working to unlock the disease-curing potential of gene therapy are urging patience. Though some countries have already approved certain gene-therapy treatments for widespread use and the United States has not, they say, the scientific community here is proceeding at an appropriate pace.

According to Dr. Theodore Friedmann, a professor of pediatrics and director of the human gene therapy program at the UC San Diego School of Medicine, the campus and its surrounding community have been pivotal in the advancements in gene-therapy research made over the last three decades.

"It is a field that was more or less born in this community," said Friedmann, who has been conducting gene therapy research for 33 years. "The concept and much of the technology has come from here."

He said that clinical research in the field only began about 15 years ago and that it often takes at least twice that time for researchers to fully and safely develop treatments once they get to that stage. Other cancer treatments, including chemotherapy, took almost 30 years of clinical research before they reached widescale application, he said.

In the case of Gendicine, Friedmann said, the Chinese government has decided that the treatment has been proven effective and safe in clinical research. He said it hasn't gotten to that stage yet in the United States, which has strict local and federal regulations when it comes to medicine.

"What has happened in China is they have moved more quickly in licensing the product," he said. "For many reasons, they have standards that may have been a bit more conducive to getting something licensed more quickly."

Also part of the process in the development of a treatment is the ethical discussion that accompanies the research. Because gene therapy deals with altering people's genetic makeup, Friedmann said, a consensus needs to be established as to under which circumstances that is acceptable.

While it might be acceptable to use the treatment in the case of cancer patients, he said, some might think it isn't acceptable in the case, for example, of elderly people who might want to use gene therapy to increase their failing muscle strength.

Everybody, not just the scientific community, needs to set the standards for proper use of gene-therapy treatment, Friedmann said.

"That's a societal process," he said. "That's not only a scientific process. There are lines that should be drawn and I don't know where they are."

But for Pechous, whose main concern has been to slow down the spread of her cancer, those ethical questions didn't matter much when time was running out.

"When you have cancer, you don't think about things like that," she said.

Early treatment the key

Dr. Dinggang Li is the director of the Gene Therapy and Surgical Oncology Center at Beijing Haidian Hospital. After getting a degree in medicine in China, Li began to learn about gene therapy while completing a doctoral fellowship at the Johns Hopkins University School of Medicine in Baltimore in the early 1990s.

He said via e-mail recently that, since the center was established in March 2004, it has treated more than 50 foreign patients like Pechous. They come from all over the world, he said, including countries such as Germany, Italy, Vietnam and Sri Lanka.

The gene-therapy treatment is part of a comprehensive attempt to treat the cancer, he said, which can include chemotherapy, radiation, heating therapy and herbal medicine.

As foreigners are heading to the hospital to seek the treatment, Li said, they aren't seeing the same levels of success that Chinese patients are seeing. Because gene-therapy treatment is readily available to Chinese citizens with cancer, he said, they are getting the treatment much sooner than foreign patients who are typically heading to the Chinese hospitals as a last resort.

Gendicine is only successful half the time in dramatically improving a foreign cancer patient's condition, Li said, while the success rate for Chinese patients is higher.

"The earlier the diagnosis and the treatment, the better the results will be," he said. "No doubt, cancer patients at the early stage will get better results from the comprehensive treatment."

China's State Food and Drug Administration approved Gendicine in October 2003 and in March of last year gave 100 hospitals in the country permission to use it, Li said.

Despite China being the first country to approve a gene-therapy treatment, Li said he believes that the United States will eventually become the world leader in creating such treatments. He points to the development of penicillin, which was initially approved in one form and then was produced on a wider scale.

"More and more gene therapy drugs will be produced after Gendicine," he said. "Then, the effect of gene therapy will be more obvious."

Taking control

Pechous and her husband were recently visited by another patient at the hospital in Beijing where Pechous received the gene therapy treatment. Ted Ticknor, 52, was given six to 12 months to live after doctors discovered he had prostate cancer that had spread to his bones.

Ticknor, who was visiting after receiving an experimental drug at Cedars-Sinai Medical Center in Los Angeles, said that he believed Gendicine had been successful in slowing the spread of his cancer. Ticknor lives in Vancouver and also had to go to China to get the treatment because Canada has not yet approved gene-therapy treatment.

He did have concerns about going to China to receive a treatment that hasn't been approved for use in North America, he said, but the staff at the hospital in Beijing proved to be capable and the treatment proved to be effective.

"What you try to do is satisfy, or try to satisfy yourself, that the people are credible and that this may hold some hope for you," he said.

For Pechous, going to China to get the treatment was empowering in certain respects. Having cancer, she said, can make one feel helpless and frustrated because the patient has little control.

Being proactive and trying a cutting edge treatment gave Pechous a sense that she had a certain amount of control in the situation. That's because she didn't have to rely on a doctor's recommendation after she had done her own research and decided on gene-therapy treatment, she said.

"You have to take a certain amount of responsibility for yourself," Pechous said. "That's the No. 1 thing ---- you have to take control of your own life."

Contact staff writer Jose Carvajal at (951) 676-4315, Ext. 2624, or jcarvajal@californian.com.

First name only. Comments including last names, contact addresses, e-mail addresses or phone numbers will be deleted. Attempts to misrepresent your identity or impersonate any person will not be approved. All comments are screened before they appear online, so please keep them brief. Comments reflect the views of those commenting and not necessarily those of the North County Times or its staff writers. Click here to view additional comment policies.