It looked like 7-year-old Emily Whitehead had run out of hope. Ravaged by drug resistant leukemia, her doctors had exhausted all of the available FDA approved treatment options. So her family enrolled her in a small clinical trial at the Childrens Hospital of Philadelphia testing a new approach to fighting cancer – an immunotherapy that would genetically modify her bodys own T cells to hunt down and kill her cancer cells. In April 2012, she received her first treatment.
The results were stunning. Her body responded with an all-out attack on her cancer that melted her tumors away. But it also threatened to kill her, since the inflammation produced by her infection led to a raging fever. Her doctors diagnosed the source of the problem as cytokine release syndrome and prescribed another drug, used to treat rheumatoid arthritis, to bring her immune system under control. In August 2012, she started second grade, and remains healthy today.
Emilys story shows biopharmaceutical innovation at its best. The development of cancer immunotherapies has taken decades, and faced numerous failures along the way. But those risks have paid off, and were now seeing more successful immunotherapies reach patients that, many experts think, will redefine the battle against many different types of cancer.
For all of its fearsome reputation – still much deserved – oncology treatment is surging ahead scientifically, benefitting from decades of robust investment, relatively flexible regulatory tools, and advanced diagnostics (like whole genome sequencing) that are helping us to unravel the underlying mechanisms driving the disease. Even more powerful prevention and detection tools should help us catch the disease in its earliest, most vulnerable stages.
Other disease areas, however, are much farther behind oncology. In neurology, for instance, we still dont have a good understanding of the underlying mechanisms driving severe depression, schizophrenia, and autism. Collectively, these ailments strike Americans when they are young, robbing them of full potential and requiring years of expensive and intensive treatment. At the other end of the spectrum, Alzheimers threatens a tsunami of human and financial costs for an already overburdened health care system.
We need to shift our imagery of the “pipeline.” Its a useful metaphor for thinking of late stage products, but doesnt do justice to the complex “architecture” driving biomedical innovation. If you turn on your faucet, you expect a flow of water, and dont think about the sophisticated architecture – the dams, aquifers, purification and treatment plants, and massive array of pipes stringing hundreds of miles across your state and city and into your home. If one of those pieces of invisible architecture breaks down, the water flow can flow to a trickle.
Biopharmaceutical innovation – in the form of new medicines – is also the result of a complex architecture that is largely invisible to the patients and frontline physicians who utilize it. Investment in the NIH, training next generations of scientists in our schools and universities, and a financial environment that encourages and rewards investment into cutting-edge therapies, are all necessary for a pipeline to produce new cures.
That architecture is threatened today by many forces. A stagnant NIH budget, the potential of price controls for Medicare Part D, threats to the exclusivity granted to biotech medicines, and a decades-old regulatory system endanger a pipeline that holds more promise now than ever before.
So I would conclude with that thought. How many Emily Whiteheads are there in America today? And tomorrow you or someone you know and love can become the next Emily, desperate for hope.
While were applauding the pipeline, and everything it is capable of, lets not forget the pipeline is part of a vast and complex architecture driving medical innovation forward. So whatever we want from the pipeline tomorrow, we have to lay the investments for today. The lives of millions of Emilys depends on it.
Original Source: http://phrma.org/phrmapedia/conversations/patient-advocate-researcher-hope-biopharmaceutical-pipeline