BOX E-1Case Example of Successful Collaboration in Drug Discovery and Development

In 2002, the biotechnology company Sugen and the Salk Institute published the human kinome, a subset of the human genome (Manning et al., 2002). Kinases regulate proteins and, in turn, have multiple functions in cells in both the normal and the disease states. On the basis of that work, scientists now know that there are 518 kinases in humans. These findings have revolutionized the approach to the inhibition of these kinases by drugs used to treat cancer and other diseases and that are currently on the market.

Elsewhere, researchers knowledgeable about patients with severe combined immunodeficiency disease (popularly referred to as the “bubble boy syndrome”) had identified these patients as having mutations within the JAK-3 kinase, which suggested that a possible mechanism for affecting the deficiency of the immune system could be achieved through a JAK-3 inhibitor (Russell et al., 1995). This research was done at the National Institutes of Health.

Industry scientists at Pfizer spent several years discovering a compound that is active against JAK-3. The goal was to find a compound that would not block JAK-1 or JAK-2 kinase but that would be effective as an immunosuppressant by specifically and partially blocking JAK-3 without causing severe side effects.

Pfizer focused first on the drug’s role as a potential antirejection drug for patients who have received an organ transplant. It collaborated with the transplant center at Stanford University to conduct studies with primates, with promising results (see, e.g., Borie et al. [2004]). The drug is being tested with human transplant recipients. It is also being investigated as a treatment for rheumatoid arthritis (see, e.g., Changelian et al. [2008] and Stanczyk et al. [2008]).

From: E, The Pathway from Idea to Regulatory Approval: Examples for Drug Development

Cover of Conflict of Interest in Medical Research, Education, and Practice
Conflict of Interest in Medical Research, Education, and Practice.
Institute of Medicine (US) Committee on Conflict of Interest in Medical Research, Education, and Practice; Lo B, Field MJ, editors.
Washington (DC): National Academies Press (US); 2009.
Copyright © 2009, National Academy of Sciences.

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