Big Pharma’s focus on blockbuster cancer drugs squeezes out research into potential treatments that are more affordable. Says one researcher: “What is scientific and sexy is driven by what can be monetized.”
This story was co-published with The Daily Beast
Michael Retsky awoke from surgery to bad news. The tumor in his colon had spread to four of his lymph nodes and penetrated the bowel wall. When Retsky showed the pathology report to William Hrushesky, his treating oncologist, the doctor exclaimed, “Mamma mia.”
“Michael had a mean looking cancer,” Hrushesky remembers.
Retsky didn’t need anyone to tell him his prognosis. Although trained as a physicist, he had switched careers to cancer research in the early 1980s and spent more than a decade modeling the growth of breast cancer tumors. During his treatment, he joined the staff of one of the most prestigious cancer research labs in the country.
In the absence of chemotherapy, there was an 80 percent chance of relapse. Even with therapy, there was a 50 percent chance the cancer would return. The standard treatment was brutal. Six months of the highest dose of chemotherapy his body could withstand and, after that, nothing but hope.
Like many cancer patients, Retsky didn’t much like the odds. Unlike most cancer patients, however, he had the knowledge to question them. His own research had sown doubts that standard chemotherapy, as used the world over to treat colon and some breast cancers, was always the best approach. In collaboration with Hrushesky, the two devised an inexpensive, low-impact chemo treatment following surgery that dripped smaller doses of the drug into his body over a longer period of time.
Seventeen years later and cancer free, Retsky cannot be entirely sure the treatment cured him, but he believes it likely did. Numerous laboratory, animal and small human studies suggest that low-dose, continuous chemotherapy holds promise in shrinking tumors and preventing cancer’s recurrence. But the next step — testing what Retsky did in a large-scale clinical trial — is a longshot given the way cancer treatments are developed today.
Take Michelle Holmes, an associate professor of medicine at Harvard Medical School. She’s been trying for years to raise money for trials on the effects of aspirin on breast cancer. Animal studies, in vitro experiments and analysis of patient outcomes suggest that aspirin might help inhibit breast cancer from spreading. Yet even her peers on scientific advisory boards appear uninterested, she says.
“For some reason a drug that could be patented would get a randomized trial, but aspirin, which has amazing properties, goes unexplored because it’s 99 cents at CVS,” says Holmes.
Increasingly, Big Pharma is betting on new blockbuster cancer drugs that cost billions to develop and can be sold for thousands of dollars a dose. In 2010, each of the top 10 cancer drugs topped more than $1 billion in sales, according to Campbell Alliance, a health-care consulting firm. A decade earlier, only two of them did. Left behind are low-cost alternatives — therapies like Retsky’s or existing off-label medications, including generics — that have shown some merit but don’t have enough profit potential for drug companies to invest in researching them.
The newer drugs have in some cases shown dramatic life-extending results for patients. Yet cancer remains the second-most-common cause of death in the U.S. after heart disease, killing about 580,000 people a year. Worldwide, 60 percent of all cancer deaths occur in developing countries, where experts say the incidence of the disease is growing rapidly, as is a desperate need for affordable care. That has added urgency to an active debate about whether efforts to combat cancer — and where to put scarce research dollars — need to be rethought.
“If we are winning the war on cancer, we are not winning that fast,” says Vikas Sukhatme, Harvard faculty dean for academic programs at Beth Israel Deaconess Medical Center in Boston and the Victor J. Aresty Professor of Medicine at Harvard Medical School.
Sukhatme and his wife Vidula, an epidemiologist, are among those trying to do something about it. They have spearheaded a new nonprofit, Global Cures, to promote alternative treatments that are unlikely to attract commercial interest from drug companies.
Global Cures calls these forsaken therapies, ” financial orphans.” To help patients and their doctors, the nonprofit is producing reports that explain the science behind promising orphan therapies — those that have shown merit in animal studies and limited human data. And Global Cures also has set itself a more challenging goal — to find the money for clinical trials.
In one example, Retsky and a team of collaborators are exploring whether an inexpensive dose of a generic painkiller before breast cancer surgery might reduce lethal recurrences of the disease. If results in a small retrospective study of 327 mastectomy patients in Europe were to bear out, the anti-inflammatory drug ketorolac could save thousands of lives a year in the United States alone, Sukhatme has estimated.
The data behind the treatment are only suggestive, however, and more testing is required. Retsky and his colleagues have been unable to raise the millions of dollars a large-scale trial would need to make a real determination, in part because no drug company has the incentive to fund such a study, they say.
Without the confirmation of large-scale human trials, doctors are reluctant to approve patient use of orphan therapies, even in cases where there is little else to offer. It’s a challenging conversation when a patient suggests an alternative medication to a doctor, who despite having the ability to prescribe off-label, doesn’t want to risk making the situation worse. “It borders on crossing the line between good evidence-based medicine and simply trying to deal with the desperate hopes of desperate patients,” says Allen Lichter, chief executive officer of the American Society of Clinical Oncology. Nonetheless, Lichter acknowledges that there are financial orphans that do not get the review they deserve.
The financial orphan problem points to a deeper issue with the way cancer drugs are developed. Pharmaceutical companies exist to make a profit and cannot be expected to cover many important areas of research that go unexplored, according toLarry Norton,deputy physician-in-chief for Breast Cancer Programs at New York’s Memorial Sloan Kettering Cancer Center.It’s a gap in the system.
“The biggest challenge we have today is not necessarily the science,” Norton says, “it’s creating a business model that makes sense.”
In 1993, about a year before Retsky received his colon cancer diagnosis, he attended a breast cancer conference in Europe. An Italian scientist named Romano Demicheli presented data from a decades-long