Genomics promises to fundamentally change much of medical care.  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 (HGS) 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 will be 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 being hailed as an up and coming company and it stock price rose to over $100 per share. But the share price then plummeted along with those of other genomic-oriented companies. Fortunately for HGS, its CEO, Dr William Haseltine, made use of the high stock price to lock in a soon to be much needed cash stockpile. The company’s first drug candidate – to treat leg ulcers – met with failure and was dropped. A second drug – for hepatitis C- looked to be very promising and carried the hopes of staff and investors but a key trial was unsuccessful and HGS ultimately had to make the decision to pull the plug on this drug candidate as well – after expending many millions of dollars in the preclinical- and clinical testing phases. The stock price dropped to a low of 52 cents per share and the cash war chest was running lower and lower. Then – finally – came good news with the two belimumab patient studies and the stock price jumped immediately but to no where near the heights of  a decade before.

Belimumab looks to be a useful addition to the SLE treatment armamentarium. But there are questions and critics. SLE is much more common in African Americans but the two patient trials had relatively few blacks enrolled and there was some suggestion that perhaps they responded less well. The drug is also not without potential side effects including predisposing to serious infection. Further, some calculate that it will take about four (or more) patients treated to lead to one patient benefitted At $35,000 per patient per year, that means a total expenditure of about $140,000 or more to benefit that one patient.

My message is that genomics is and will be a transformational megatrend in medical care. But “targeted” therapy is not always all that narrowly targeted in a way that either is effective for all nor is the drug necessarily side effect free – and it may be quite expensive. The story of Human Genome Sciences demonstrates that the path from a genomic discovery in the laboratory to a marketed drug can be long, expensive and fraught with many disappointments along the way. But conversely, innovation as demonstrated here is and will be the lifeblood of continued future success and improved human health.

Stephen C. Schimpff is a retired CEO of the University of Maryland Medical Center in Baltimore and is the author of The Future of Medicine — Megatrends in Healthcare. He blogs at Medical Megatrends and the Future of Medicine.

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