When the dark shadow of incurable disease settles across a life, it is brightened only by the hope that science is on the job: The cavalry will come.
Horribly the cavalry — researchers in the big pharmaceutical companies and the government-run National Institutes of Health and the Centers for Disease Control — may not even have mounted.
New drug development is a murky business governed by huge risks, inertia, bureaucracy and politics.
I've been looking at the role of biomedical research and the development of new therapies and drugs through the lens of one disease, Chronic Fatigue Syndrome (CFS), also known as Myalgic Encephalomyelitis. But it is symptomatic of the whole struggle for cures, which means funds. It is a peephole into a system in chaos; where good intentions, economic reality, public pressure, politics and bureaucratic apathy play a role in where the research dollars go.
I've been writing about CFS for several years now, so I understand the dilemmas those who are in charge of biomedical research in government and private industry face. It is a disease of the the immune system, like AIDS, but it is mostly a medical enigma. It is hard to diagnose because there are no normal markers in blood or urine. It prostrates its victims essentially for life. In its severest form, patients lie in bed in darkened rooms, often feeling that their bones are going to explode. It cries out for more research, as do many other little understood diseases.
A very small coterie of physicians — maybe not many more than 50 in the United States — specialize in CFS and have developed private clinics for research into alleviating therapies. None of them are set up to do major drug research in the way that pharmaceutical companies do.
Big Pharma — as the drug behemoths are known collectively — is at the heart of new drug development, aided by preceding biomedical research that takes place through government grants to researchers in universities, teaching hospitals and private clinics. It is a complex matrix.
A new drug can cost over $1.2 billion to develop. It is a very high-risk undertaking — maybe the riskiest investment decision made in the private sector is developing a new drug. It is also a tortuous undertaking.
First a target has to be selected where there is a large enough patient cohort to establish a market. Then the science begins. Diseases that are straightforward, in medical terms, edge out those where the causes may be multiple and the resolution may require a cocktail of drugs. Understandably, a rifle shot is more appealing than a shotgun blast. Eight out of 10 drugs fail and are abandoned at some point. The winners have to pay for the losers.
If, after years of research, a compound that may work is discovered, the laborious business of testing it on animals must precede human trials with control groups and years of analysis. Finally the drug must be approved by the Food and Drug Administration which looks for efficacy, safety, risk benefit and manufacturing stability.
Into this already difficult world of new drug development, enter the politicians.
Some believe private enterprise will shoulder all the risks and is the right place for research. Others don't understand the vital role that government research grants — administered by NIH and CDC — play in the development of biomedical knowledge: the essential precursor to new drugs and therapies. Its funding is on a see-saw; it was down under sequestration and funding is restored but not boosted under the new budget deals. It tops out at $29.9 billion, a decline of 25 percent since 2003, according to The Atlantic magazine.
Chronic Fatigue Syndrome — which has 1 million Americans suffering hopelessly every day — gets about $6 million a year from NIH. What's wrong with that largesse? Well, remember, it costs $1.2 billion to develop a new drug once the biomedical case is made. As they say, you do the math – and don't expect the cavalry to ride to the rescue anytime soon.
Across the board, researchers are dependent on government funds augmented by foundations and charitable giving. Yet biomedical research pays as a national investment. American drugs are an export commodity, the cost of healthcare is contained and, yes, the suffering is reduced even as life is extended. China, by the way, has said it will surpass the United States in actual biomedical research dollars in five years. — For the Hearst-New York Times Syndicate
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23andme.com was funded by goggle $$$ because of a founder parent illness. The evidence suggest that a similar funding for gut bacteria ( which is ten times the size of the human DNA) will lead to major breakthru for many of the auto immune conditions… Will bezos finally step up to donate for a major health initiative?
Dear Sir:
My thanks to you for another excellent article pertaining to ME/CFS. As a ten year patient, I have not met or read a person who seems to grasp the severity of this illness in the manner you do.
While I have suffered the misfortune of losing my career as an airline pilot, my wife and my home, I count myself truly fortunate among the ME/CFS population. With health care insurance and a private disability plan, I have been able to seek medical help and provide for myself despite not being able to venture outside of my apartment for months at a time, except when seeing a doctor or a trip to the grocery.
My main reason for writing to you after this particular article is that I wish to share with you a hypothesis for ME/CFS etiology. I have run this hypothesis past Dr. Kogelnik (whom you have interviewed) and he indicated that it may hold promise but we do not have the data. Thus going straight to your point about biomedical research and the money that drives it.
The hypothesis in a nutshell is a broken feedback loop between immune function and mitochondrial function. The concept is explained in detail by a set of four slides on my Google drive. The link to it is your field marked "Website".
By employing the principles of this hypothesis, my situation has improved substantially over the last six months. I am hoping for a full recovery at some point this year, and my greater hope is to help end the suffering that I know so many others are still experiencing.
My sincere thanks,
John Bochenek
My thanks again to Mr. King for his attention and empathy. Unfortunately, ME/CFS is far from alone in having no known cause or cure. Every so often I meet or read of a person who had an immune or autoimmune reaction that all but took them out. Case in point: a woman who appeared "nervous" to me who in fact had became partially paralyzed at age 26 after giving birth. Amazingly, she survived and mostly recovered from 8 years of steroid treatment, but had to relearn how to walk and talk. To this day, after a book-full of MRIs and blood tests, her doctors have no name for her condition or idea of the cause.
With allergies and such "autoimmune" phenomena on the rise, my hope is that Pharma will develop "bullets" to decrease inflammation and cytokine response, which in turn could get multiple-disease approval or at least off-label use for ME/CFS. With everything seemingly due now to "inflammation," you'd think the market would be big enough. Pharma researchers are certainly aware of this. The problems lie further up the chain with those who pick the safest, cheapest options to reward stockholders today, and the Feds' willingness to help organize and fund the necessary trials. I still hope to see some help in my lifetime, but I think it'll be by stroke of luck.
Letter to the Editor in The [Washington, Pa.] Observer Reporter
I disagree with what Llewellyn King said in his essay, “The shame of biomedical research across the U.S.,” which appeared on the Observer-Reporter’s commentary page Jan. 26. In the essay, King faults pharmaceutical companies and the National Institutes of Health for their failure in developing therapy for chronic fatigue syndrome. This reflects his lack of understanding for the potential and limits of biomedical research by pharmaceutical companies.
Dr. Janet Rowley, who died Dec. 17, discovered the specific crossover between chromosomes 9 and 22, resulting in the BCR-ABL1 fusion gene, also called the Philadelphia chromosome, which is responsible for chronic myeloid leukemia (CML). The subsequent development of tyrosine kinase inhibitors, such as Gleevec, has since rendered the once-dreaded CML into a chronic disease, with over 90 percent of patients enjoying a normal lifespan. This is a perfect example of how pharmaceutical companies reap what basic biomedical research sows.
Chronic fatigue syndrome is a totally different story. Experts have difficulty defining it. The Centers for Disease Control and Prevention (CDC) and the National Institute of Allergy and Infectious Diseases have both published and revised definitions several times. Theories of its causes abound, but there is no consensus.
King lamented the cutback of NIH funding for basic biomedical research, which, I agree, is shortsighted. This is not to say that the NIH shouldn’t fund research on chronic fatigue syndrome, but blaming Big Pharma at this point is missing the point. — Jer-Yuan Tsi, Waynesburg