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A New Approach to Revolutionize Clinical Trials

December 4, 2015 by Llewellyn King Leave a Comment

By Llewellyn King
It may be the biggest thing in medicine in a long time. It’s not a new drug, or therapy or discovery. Instead it’s a new way of doing clinical trials which could have a dramatic effect, particularly on those diseases that get less attention.
 
Initially it is aimed at the deadliest brain cancer, glioblastoma multiforme, known as GBM. From diagnosis, it can kill in about six months.
 
Traditionally clinical trials test one drug or a therapy on a lot of patients, sometimes thousands, against a control group that is treated with a placebo, or not treated at all. Trials drag on for years.
 
The aim in clinical trials, which are certified by the Food and Drug Administration and reviewed by FDA-appointed panels of experts, is to make sure that a drug, medical device or procedure is safe and effective. This system costs a huge amount of money — hundreds of millions of dollars — and takes years. It produces no collateral knowledge; and it doesn’t alleviate the suffering.
 
Also, it’s an imperfect system. When a drug or medical device is certified for sale by the FDA, it may be less effective than the manufacturer claims, or not safe. Witness the recalls and lawsuits against drugs and medical devices — like the ones against Avandia, the diabetes drug, and transvaginal mesh.
 
This form of testing is totally inappropriate for virulent diseases, where the cohort being tested is unlikely to live long enough to find the results. 
 
That stark truth has driven a global fraternity of specialists in brain cancer to set up a new concept in clinical trials. These doctors, neurosurgeons, neuro-oncologists, biostatians and genetic researchers are out to make trials a present-tense matter.
 
Members of this fraternity of researchers and clinicians come from around the globe with a heavy emphasis on the United States, China and Australia. They gathered in Washington last month to launch their trial, known as GBM AGILE, an acronym for Adaptive Global Innovative Learning Environment.
 
They are driven by the knowledge, as Anna Barker, GBM AGILE project director, put it, that the present treatments for GBM “are unacceptable.”
 
She told me that there has been little progress over the years. Other doctors told me that the three treatments used at present — surgery, chemotherapy and radiotherapy  — aren’t saving patients. Few survive, or have their lives appreciably extended.
 
“It is not acceptable, not acceptable,” Barker said over and over again.
 
Barker is a professor at Arizona State University, a former deputy director for strategic initiatives at the National Cancer Institute, part of the National Institutes of Health, and a crusading force for improvement in brain cancer treatment, as well as how clinical trials are conducted.
 
Webster Cavenee, a professor at the University of California at San Diego, said the drug that is used for treating this aggressive cancer doesn’t work because the cancer, which doesn’t metastasize, becomes resistant to it in a short time, rendering the procedure useless. “We need a cocktail of drugs,” he said, “so that the cancer is deceived.” 
 
The FDA, often seen as a barrier to new treatments, is on board with GBM AGILE and has promised to approve new procedures within days, according to Barker.
 
The work to get the trials set up is already well underway. More than 100 cancer specialists, divided into 10 major committees, are working on the structure. 
 
As I understand it, GBM AGILE will establish a global pool of patients whose physicians will report on therapies, drugs and environments that succeed or fail, building a huge database of clinical knowledge. Patients will cycle out, hopefully because they are cured, but sadly, more likely, because they’ve succumbed. The organizers call it “crowd-sourcing knowledge.”
 
It is hoped that major funding will come from big foundations and charities. But initially the institutions that will participate have been defraying expenses, with an important assist from the small but energetic group, the National Foundation for Cancer Research, whose president, Sujuan Ba, has played a role in bringing in Chinese research institutions.
 
In the lay language of today, there won’t be any stovepiping, less repetitive prescribing of doubtful treatments, and a greater sharing of the patient experience.
 
Vice President Joe Biden is an enthusiastic supporter. His beloved son, Beau, died from GBM. Biden told the gathering of BMG AGILE founders in Washington that if Beau hadn’t contracted the disease, he would’ve run for the presidency. — For InsideSources

Filed Under: King's Commentaries Tagged With: Adaptive Global Innovative Learning Environment, AGILE, Anna Barker, Beau Biden, brain cancer, brain tumor, clinical trials, FDA, Food and Drug Administration, GBM, GBM AGILE, glioblastoma multiforme, National Foundation for Cancer Research, Sujuan Ba, University of Arizona, University of California at San Diego, Vice President Joe Biden, Webster Cavenee

The Shame of Biomedical Research in the U.S.

January 19, 2014 by White House Chronicle 4 Comments

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

Filed Under: King's Commentaries Tagged With: Big Pharma, biomedical research, CDC, China, Chronic Fatigue Syndrome, drug industry, drugs, FDA, myalgic encephalomyelitis, NIH

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