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Breakthroughs in Cancer Treatments Could Lie Just Around the Corner

Inder M. Verma used to take his dog for strolls in La Jolla. One day, they turned a bend, and the dog spotted a mouse.

“He did what dogs do and ate it,” recalled Verma, a professor in the laboratory of genetics and an American Cancer Society professor of molecular biology at the Salk Institute. “Then every day after that, he would stop at the same spot, but he never found that mouse again. That is how I feel. It is the excitement of discovery. Every day, I think I can do something new. Every day, I come to the lab, I still think, ‘Today will be the day of another discovery.’ I am as excited now as I was 30 or 40 years ago.”

And so it goes in the world of San Diego’s scientists who spend their days , and sometimes long nights , searching for breakthroughs that will prolong lives. It continues to be a daunting mission.

The American Cancer Society estimates that in 2007, close to 560,000 Americans are expected to die of cancer , more than 1,500 a day. The organization calls it the second most common cause of death in the United States, behind heart disease, accounting for one out of every four deaths.

But, in San Diego’s booming life sciences industries, much progress is being made.

Ivor Royston, founding managing partner of local venture capital firm Forward Ventures, is often credited as being the father of San Diego’s now thriving biotech industry.

“He was the miraculous spark that ignited the biotech revolution in San Diego and the U.S.A.,” said Dr. Albert Deisseroth, president and chief executive officer of the Sidney Kimmel Cancer Center in San Diego. “He is someone I respect very much.”

The founding president and former CEO of Kimmel, and a current member of its board of trustees, Royston was the co-founder of Hybritech Inc., San Diego’s first biotech startup. In 1986, Royston co-founded Idec Corp., which later merged with the Cambridge, Mass.-based Biogen.

“There are a lot of very exciting biotech companies in San Diego that are developing new cancer drugs, some very innovative,” Royston observed. “We are one of the top areas in the country for new cancer drugs.”

Royston credits the biotech boom to Rituxan, approved in 1997 for non-Hodgkin’s lymphoma and for rheumatoid arthritis in 2006. The drug was discovered by Biogen Idec and co-marketed with San Francisco-based Genentech Inc., a drug developer which maintains a state-of-the-art manufacturing facility on 60 acres in Oceanside. The drug now is being studied in late-stage clinical trials as a potential treatment for patients with chronic lymphocytic leukemia.

“Historically, it was the first antibody for treating cancer, and it stimulated other antibody treatments throughout the country,” said Royston. “We have a history of developing very innovative new cancer treatments. This is one of the top places in the world for the development of new cancer treatments.”

Pfizer La Jolla pharmaceutical company now is the center for oncology research for the company worldwide. Among the drugs it has developed is Sutent, approved by the Food and Drug Administration in 2006 for treatment of rare forms of stomach and kidney cancers. The company also has additional clinical trials under way for Sutent in treating lung, breast, colorectal, liver and prostate tumors.

According to development team leader, Zuleima Aguilar, senior director for oncology at Pfizer La Jolla, Sutent is the first new cancer drug to be granted approval by the FDA and the European Medicines Agency of London for two types of cancers simultaneously.

“I have been lucky to work in bringing a drug to market,” said Aguilar. “I pinch myself. I say, ‘I can’t be luckier than this.’ We are able to help so many people by developing these drugs that are effective. I love working in oncology for that reason. You know that you are doing something to help people. For years and years, all that was available was chemotherapy.”

But there is no one-size-fits-all drug for all cancers, said Aguilar.

“In the cancer population, tumors have different origins,” she explained. “One that creates colorectal is different from breast cancer. You have to test them separately and set up trials appropriate to the patients.”

Sidney Kimmel’s Deisseroth and his team have developed an experimental vaccine to prevent the recurrence of breast cancer, and have been cleared to use human volunteers in clinical testing at Sharp Hospital.

The vaccine will be tested in women who have already been diagnosed and treated for breast cancer, and are at risk for recurrence.

“A cancer vaccine has always been a dream that is hard to realize,” said Deisseroth. “Immune response tends to dwindle as we get older,” he said. “We designed around these defects. If we are successful in displaying a response pattern, and no toxicity in early relapse, we would move to treating individuals before they relapse. That is the ideal application of cancer vaccines.”

Genentech has developed a drug called Avastin, first approved by the FDA in 2004 in combination with chemotherapy for the treatment of metastatic colorectal cancer. In October, the FDA approved Avastin in combination with chemo for the most common type of advanced lung cancer.

“It is the first time a therapy has extended the life of advanced lung cancer patients beyond one year, and the first treatment advance in the disease in a decade,” said Kelli Wilder, a Genentech spokeswoman.

Avastin currently is being studied in more than 20 different types of cancer, she added.


The Personal Touch

Meanwhile, biopharmaceutical firm Favrille Inc. is focused on the development and commercialization of personalized therapies for the treatment of cancer and other diseases of the immune system.

The company has raised $45 million to continue developing and, eventually market, its centerpiece drug , FavId. Designed to follow Rituxan in the treatment of non-Hodgkin’s lymphoma, the drug is in phase-three clinical trials.

“We make FavId for every patient individually,” said John P. Longenecker, Favrille president and CEO. “It’s a novel approach, and is on the cutting edge of personalized medicine. Also, it is a cancer vaccine, and allows patients to participate in their own disease control.”

But Longenecker is quick to point out that this vaccine, rather than being preventive, is designed to be used as a treatment for the disease.

Personalized medication doesn’t come cheap.

“It’s different from manufacturing pills, where you pound them out,” said Longenecker, who declined to speculate on what the treatment is likely to cost.

But, he added, it’s all relative.

“These are patients that need to be treated for seven to 10 years with a variety of therapies, and those cumulative costs are very high,” he said. “If our product can induce long-term remission, there is a great cost savings here, even with its premium price.”

On the commercial end, Favrille is completing construction of an 80,000-square-foot manufacturing facility in Sorrento Mesa for the eventual launch of the product, said Longenecker.

Fast-tracked by the FDA, Longenecker said that he hopes to have the drug approved by the end of 2008. It’s been a long haul, starting with the company’s founding in 2000. Its investment in

FavId could end up being from $200 million to $300 million, said Longenecker, “depending on marketing activities, which can go through tens of millions of dollars.”


Paying The Price

Considering the many years of research, and millions of dollars invested in the development, and marketing, of cancer drugs, a question persists: How many people will be able to afford these miracle drugs?

Walter Eckhart, a professor and director of the Salk Institute Cancer Center in La Jolla and head of its molecular and cell biology laboratory, ponders the question, too.

“If you look at cancer treatment, it is pretty scary, the economics of it,” he observed. “It is something that our whole society must work on. The presidential election is a year from now, and already people are saying we need to know what your ideas are for the health care system. I think that there will be healthy debate about it.”

Salk’s Verma, considered a leading authority on the development of viruses for gene therapy, said that it is difficult to put a price on survival.

“This is something that society will have to decide, and pharmaceutical companies will have to decide,” he said. “Each drug is costing $800 million to $1 billion, so they can’t afford to sell at cheap prices either.”

Verma recalled that in the 1940s, the cost of penicillin was beyond the reach of many Americans, and the same was the case with treatments for HIV, the AIDS virus.

“It could be, that if a drug becomes universal, the industry will find a cheaper way to make it,” said Verma. “I suppose it’s the law of supply and demand.”

Equally perplexing is trying to determine who is best suited to receive the new therapies, he said.

“It’s a complex question,” said Verma. “It’s easy to say, ‘Is it worth $100,000 to prolong life for seven months?’ ” But what those seven months can bring to people is amazing. My dad is 93 and I will never see him shortchanged on anything because he is 93. You cannot think, when you are doing research, what it will cost, or you will be paralyzed.”

Nor does Verma place values on individual worth when it comes to treatment.

“When I have a leak, I want a plumber, not a professor,” he said.

Considering these deep philosophical questions, Verma joked, “It’s a lot easier to do an experiment. The data gives the answers, I don’t have to give the answers.”

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