Forensic Science: Bodies of Evidence
Studying Disease Effects in Underserved Youths
Quality Management in Health-Care Settings
Changing the Cancer Treatment Paradigm
Unwrapping Secrets of the Ancient World
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Changing the Cancer Treatment Paradigm
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Natalie R. Sacks '86 |
Despite significant advances in biomedical science, the treatment of cancer is confined largely to three measures that have not changed much over the years: surgery, chemotherapy, and radiation. Each therapy has its downside. Surgery always entails some measure of risk, and recovery can be long and painful. Chemotherapy and radiation destroy healthy cells along with cancerous ones, leading to such deleterious side effects as hair loss, nausea, and fatigue.
Cell Genesys, a South San Francisco-based biotechnology company, is looking to change the cancer treatment paradigm, and Natalie R. Sacks '86, the firm's vice president of clinical development, is an integral part of those efforts.
"We're trying to develop a new modality for treatment of cancer," Sacks says. "People are familiar with the existing things that you can do—you can cut it out, poison it, etc.—but now we're moving into an era of greater understanding of tumors and the biologic basis for the disease. That's allowing us to develop therapies that have a more biologic basis. Within that broader area, Cell Genesys is very focused on immunotherapy. That's a fairly new area where the overall goal is to get the patient's own immune system to target the tumor."
New Product Development
Sacks is responsible for shepherding Cell Genesys's products through the clinical-trial pipeline to final approval by the Food and Drug Administration. It is a charge with numerous and varied components, from medical issues to regulatory affairs to business development. Integrating those aspects into a seamless process that provides evidence of a product's safety and efficacy, she says, "requires a lot of work studying hundreds, if not thousands, of patients to figure out how you should give a new product to someone, what the dose should be, what the schedule should be, whether it should combined with something, and who exactly should it be given to and for how long."
Cell Genesys's lead investigational product is GVAX immunotherapy for prostate cancer, which is now in Phase III clinical trials. Sacks worked with "great colleagues" to design the protocol for its clinical trials, and met with the FDA to get permission to move forward with testing.
"One of the compelling things about the field is that it's been a mystery for a long time as to why cancer is ignored by the immune system," Sacks says. "The immune system should generally be noticing if something's not supposed to be there, but cancer has several mechanisms whereby it evades that process. The successful development of cancer immunotherapy has depended on a lot of breakthrough research into understanding how to get the immune system to recognize danger signals that will allow it to target the tumor. We're trying to translate that to effective therapy so patients have more options."
Large-Scale Questions
Sacks came to Cell Genesys nearly six years ago after training in medicine and oncology at the University of Pennsylvania, and biostatistics at the Harvard School of Public Health. The chance to work for an innovative R&D company proved too compelling to pass up.
"The biotechnology area seemed to be a good environment to answer large-scale questions that can potentially lead to new treatments for patients," she says. "A lot of fantastic research goes on in academic environments, but emerging biotechnology companies have tremendous strength in being able to answer critical questions perhaps more rapidly than one can in academia."
The GVAX immunotherapy for prostate cancer Phase III trials began in 2004 and have enrolled hundreds of patients in the United States, Canada, and Europe. Data from these trials are expected in the second half of 2009. The size and duration of these trials, which are not at all unusual in cancer drug development, are necessary to demonstrate that GVAX can improve patient survival.
"The drug-development process can be frustrating," Sacks says. "It takes many millions of dollars and many years, but if you're going to demonstrate thoroughly that the drug is safe and has clinical efficacy, there are no shortcuts."
Among the unique facets of immunotherapy research is that its metrics are quite different from those used to assess more traditional cancer treatments. Effective cytotoxic therapies—those that kill cells—lead to measurable tumor shrinkage. In contrast, immunotherapy typically takes longer to work. A meaningful anti-tumor response by the body's T and B cells "can take weeks or months" to happen, Sacks says, so the area "requires good thinking about how to adapt traditional clinical research methods to a class of agents that work differently than cytotoxic ones."
"It was appealing to me to figure out how to get to the end of this process without a map," she adds.
As if dealing with physicians and regulators didn't keep her busy enough, Sacks spends considerable time on business development, a duty that sends her to other pharmaceutical companies for discussions on collaboration.
"It's not what you learn in medical school," she notes. "To have great science and great medicine, you need money. We need resources."
Sacks continues to see patients, which helps her stay focused despite the lengthy timelines of her work.
"Patients are suffering from a lack of options," she says. "Although we've made tremendous strides in oncology, most patients still die. You just remain focused on the fact that you're trying to develop a new option, and it takes a long time."
Tom Durso writes about science, health care, and business for a variety of publications, including the Philadelphia Business Journal and Family Business magazine.