Genomics promises to fundamentally change much of medical care as described in the two prior posts on this subject. But the ultimate value of this new understanding of basic human biology will in many cases come with fits and starts. The saga of belimumab (Benlysta) and Human Genome Sciences is illustrative.
Systemic lupus erythematosus (SLE) occurs in somewhere between 300,000 and 4 million Americans according to the Centers for Disease Control. It is more common in women than men and more common in African Americans than Caucasians. SLE is an autoimmune disease of unknown etiology which can affect many body organs and systems and can lead to death. The disease can wax and wane and can flare with activity in the central nervous system and the kidneys. Genomic studies done by Human Genome Sciences (HGS) more than a decade ago showed that it might be possible to create a monoclonal antibody to react against the B lymphocyte stimulator, a cytokine that has been found to correlate with activity in SLE.
HGS produced the monoclonal antibody – belimumab – and then conducted the required preclinical studies in test tubes and animals to demonstrate its activity and toxicities. With FDA approval it then conducted phase 1 and 2 studies in humans to determine toxicities, side effects and early suggestions of activity in humans. It then proceeded to complete two double blind randomized controlled trials comparing standard treatment to standard treatment plus belimumab in 1684 patients. The results were sufficient to win a 13 to 2 recommendation vote from the FDA’s advisory panel in November, 2010. The FDA announced its approval and the required package labeling in March, 2011. The FDA and HGS have both noted that this is the first new drug for SLE in 50 years and the approval was lauded by the Lupus Foundation of America. The intravenously administered drug is available for about $35-40,000 per year.
It all sounds straight forward – good science led to a new drug that should benefit many patients who have had limited treatment choices until now. But for HGS it has been a rocky road at best over the years. Founded in 1992, the company set out to use genomic discoveries to create new pharmaceuticals. By 2000, it was...
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