Of the more than 300,000 Americans diagnosed with breast cancer each year, Joan Syron counts herself among the disease’s most privileged survivors.
In November 1995, a routine mammogram revealed a lump in her left breast, and a biopsy soon after showed it to be malignant. After surgery to remove the tumor in early 1996, Syron began a course of tamoxifen, a hormone therapy long seen as the gold standard for treating post-menopausal women who have undergone breast cancer surgery.
Tamoxifen has a proven track record of reducing the risk of breast cancer recurrence, but it is only considered effective for about five years, and so in February 2001, when her five-year stint on the drug was up, Syron was lucky enough to be asked to take part in a clinical trial to test letrozole — a then experimental hormone treatment designed to keep cancer at bay after five years of tamoxifen.
To say the trial was a success would be an understatement. Data collected after just over two years showed an impressive 43 percent reduction in risk of overall cancer recurrence and a significant reduction in the risk of the disease spreading to the opposite breast. Novartis, the Swiss drug that ran the trial, ended it early to allow patients on placebos to take advantage of the drug.
“I feel very fortunate that I was given the opportunity to take part in this trial,” said Syron, who is now 71 years old and in complete remission from her cancer. “The only thing I have to worry about is my yearly mammogram — whether it is five years or 25 years after your diagnosis, you’re always going to worry about that.”
New focus on oncology
These days, thanks to an increasingly refined understanding of how cancer develops in the body, Syron’s experience is not unique.
A new class of oncology drugs is emerging, offering a much-needed ray of hope to the millions of people who are diagnosed with cancer each year and potentially providing a source of strong sales growth for the biotechnology and pharmaceutical industries.
To date, many big drug firms have ignored the oncology market, preferring to concentrate on more lucrative areas of drug development, like cardiovascular diseases, or cholesterol treatments, but today just under half of all the drugs now in development are cancer-related, experts say.
At pharmaceutical giant Pfizer, oncology is the second-biggest area of drug development according to Bill Slichenmyer, vice president for Oncology Drug Development at Pfizer. “This is a large area of much need,” he told CNBC. “And we’re committed to transforming cancer into a chronic and manageable disease.”
Companies like Pfizer and Novartis are creating so-called “targeted” therapies that hone in on the molecular characteristics of cancer cells, leaving the healthy tissue that is often damaged by standard treatments, like radiation and chemotherapy, relatively unharmed.
The aim, experts say, is to find a cancer cell’s Achilles’ heel and devise treatments to exploit it. Some drugs target specific enzymes called "kinases" that control cell growth, while others are monoclonal antibodies that recognize markers on the surface of cancer cells and kill them. Some starve tumors by cutting off the blood vessels that keep them alive.
Gleevec — a wonder drug
One of the latest wonder drugs is Gleevec, a Novartis medication that was one of the first targeted therapies and approved by the U.S. Food and Drug Administration (FDA) in 2001 to treat chronic myeloid leukemia, a cancer of blood cells.
A conventional treatment for this rare form of cancer is a bone marrow transplant and chemotherapy, but Gleevec is orally administered and works by knocking out a cell protein called tyrosine kinase, essentially disabling a cancer cell’s ability to reproduce.
“After two years, 95 percent of the people are disease-free, so drugs like this are big news for us,” said David Epstein, president of Novartis Oncology, the arm of the pharmaceuticals firm that develops cancer therapies. Another benefit is the fact that the newer treatment causes side effects are much less unpleasant, he notes. “You might get some mild sickness, but nothing like the disabling effect of chemotherapy.”
With hot new cancer drugs like Gleevec, Zometa, Sandostatin LAR and Femara (Novartis' brand name for letrozole), the company now has the third biggest portfolio of oncology drugs in the world, and has about 10 or 12 new drugs in clinical trial at any one time, according to Epstein.
Epstein sees great potential in the oncology drug market, estimating that it is worth about one-tenth of the size of the worldwide pharmaceutical market, which is currently valued almost $500 billion in sales, according to IMS Health, a provider of information services to the pharmaceutical and healthcare industries.
“We have a rigorous process to identify where we think there is a need and we match that with areas where we think we have expertise,” Epstein said. Most pharmaceutical companies prefer to focus on producing drugs to treat high cholesterol, depression or hypertension, Epstein added — medicines with large demand that deliver a steady source of revenue.
“Oncology is relatively modest by comparison, but it’s growing rapidly,” he added. With the science getting to a place where people feel the drugs work well, and the prevalence of the disease growing as the population ages, many pharmaceutical companies are focusing on this area. And none of the current therapies are any good, so there is lots of room for improvement here.”
Dozens of other pharmaceutical and biotechnology companies, including market heavyweights like Genentech and Bristol-Myers Squibb, are making advances in oncology treatment. In 2003, there were about 400 new cancer drugs in development according to the latest data from the Pharmaceutical Research and Manufacturers of America, a trade group for the pharmaceutical and biotechnology industries.
At the same time, the FDA is working to get the new drugs approved and delivered to the patients who need them. A typical approval process for a drug can take anything between 12 months and a number of years to move from the initial drug discovery stage through the three stages of FDA approval — which involve testing the toxicity of a drug and its effectiveness on humans — to when it moves on to the market.
Costly Rx
But while there is a great deal of excitement about them, analysts stress that targeted cancer drugs are far from perfect. They are less toxic than chemotherapy, but they still have side effects, and they can sometimes cost as much as $100,000 a year for a standard therapy.
Analysts also stress that there are no miracle cures in the pipeline. They also say many of the latest treatments tend to work only in small subsets of cancer patients dealing with rare forms of the disease, which raises the question of market size and potential growth for companies developing the drugs, and the as yet unsolved problem of developing a test to match the right drug with the appropriate patient.
“The bad news [about these drugs] is they are very expensive and only work in a subset of patients, so the issue is can we figure out how you can define that subset,” said Robert C. Young, M.D., president of Philadelphia’s Fox Chase Cancer Center. “If we could, it would take the targeted treatment theory to the next level,” he added.
From an investment standpoint, some experts are cautiously optimistic about the prospects for the oncology drug market.
With cancer on its way to becoming the leading cause of death in the United States, and with big drug firms looking for new sources of revenue as multibillion-dollar drugs like Merck’s cholesterol remedy Zocor soon coming off patent, oncology is the new frontier for the pharmaceutical industry according to Peter Tollman, a consultant at the Boston Consulting Group who covers the biotechnology sector.
“Drug firms want to develop the next thing that’s going to make them money and one of the next big markets for drug discovery is the oncology market,” Tollman said.
Targeted therapies are the closest scientists have come to the holy grail of eradicating cancer altogether, but some researchers are daring to consider that possibility. They are working to develop "cancer vaccines" that do not interact with cancer cells, but instead teach the body’s immune system to stamp them out.
While he says it is very much in the experimental stage, Steven Heffner, an analyst at Kalorama Information, a life sciences market research firm, sees the market for cancer vaccines growing from $14.7 million in 2003 to $1.3 billion in 2008, with much of the growth coming in the cervical cancer market after the approval of Merck’s HPV vaccine. Other companies involved in the market include Avax in Australia, and U.S.-based firms Corixa and Aventis Pasteur.
“The real drivers of this market are the biotech companies — they are the ones developing the new treatments,” Heffner said, noting that there have already been some notable successes in treating melanoma and bladder cancer. “They are really looking for a cure here, and that’s what makes this so revolutionary.”