50 whys to look for genes: 32. Production of usable amounts of medicinal biochemicals

From the early 1960s researchers looked for the hormones – that controlled white blood cell production.  By the early 1980s, having identified and purified the chemicals–Colony Stimulating Factors (CSFs), the problem was to extract enough pure CSFs from tissues to use clinically.

“To get enough material for one patient we would have had to work for 250 years,” Professor Metcalf of the Walter and Eliza Hall Institute said. “We had purified CSF, we had done elegant tissue culture experiments, but now we’re into logistics and were facing a big black hole.”  The team decided to try to use genetic cloning to produce CSFs for use in the lab… [By] 1986, they (and other groups) had cloned all four CSF genes from the mouse and human genomes, and had started producing them en masse. After 20 years of research, the hormone was finally ready to trial in animals. And it worked.  [By 2009] more than 20 million cancer patients ha[d] benefited from the therapy. (http://www.wehi.edu.au/about_us/achievements/professor_don_metcalf/)



Acute myelogenous leukemia (AML) is a fatal bone marrow cancer. Colony-stimulating factors (CSFs) are frequently administered during and after chemotherapy to reduce complications. [But CSFs] should not be given routinely to acute myelogenous leukemia patients post-chemotherapy since they do not affect overall survival or infectious parameters including the rate of bacteremias and invasive fungal infections. (Gurion et al. 2012)

2.  Cost-effectiveness.  CSFs are not cheap to provide so studies are undertaken to estimate the cost-effectiveness of CSF use in, for example, early-stage breast cancer when its use is directed to those most in need of the medication.  The relevance of defining “populations for which therapy is, or is not, cost-effective” (Silber et al. 1998) depends on national healthcare budget and policies (e.g., universal insurance) and an individual’s coverage.


(Introduction to this series of posts)


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