From discovery to delivery
Drug development at Dana-Farber
By Robert Levy
Illustration by Lee Whale
A Rip van Winkle awakening from a 20-year nap would blink in awe today at scientists' ability to identify the misbehaving genes and proteins at the root of cancer. But the process by which promising scientific leads are transformed into new therapies would be quite familiar.
Historically it has worked like this: An investigator in an academic research center such as Dana-Farber discovers a cell molecule that spurs cancer cell growth. In the laboratory, the researcher demonstrates that blocking the molecule with an antibody or other agent stops the growth. If the blocker can be tested in laboratory animals, the investigator will try to demonstrate that it halts tumors in them as well.
At that point, the researcher's home institution licenses a pharmaceutical or biotechnology company to develop a commercial drug to thwart the target molecule. The firm tries to fashion a compound that hits the target, lasts long enough in the body to reach the target, and crosses tissues that stand in the way. Ideally, the company finds a way to package the agent in pill form.
If the compound still seems promising, it is tested in animals – analyzing its ability to be absorbed, used, and eliminated by the body, and its potential hazards. With approval from the federal Food and Drug Administration, the company then contracts with a clinical research center such as Dana-Farber to study the drug's safety and effectiveness in patients. (One exception to this process involves monoclonal antibodies, specialized proteins that hunt cancer cells. Because they are natural substances, monoclonals often are developed into therapies largely within academic research centers.)
In several respects, this process has served cancer medicine well. It is responsible for many of the drugs available today and for many of those to come. The division of labor among basic scientists, pharmaceutical companies, and clinical researchers plays to the strengths of each. Laboratory "bench" scientists are experts in the molecular workings of disease and embody the academic tradition of free pursuit of knowledge. Pharmaceutical firms have the financial and technical means to rapidly test enormous numbers of agents against cancer, conduct the chemical manipulations that result in useful compounds, and produce them in large numbers. Clinical researchers specialize in carrying out the complicated protocols of testing new drugs in patients and measuring the risks and benefits.
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