White House Chronicle

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Cancer ‘Moonshot’ Has Paltry Dollars, Losers

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By Llewellyn King

Whenever the government wants to be seen to be doing something huge, it invokes the Manhattan Project or the moon landing. So the new cancer initiative of the Obama administration is called the “moonshot.”

But it’s neither the equivalent of the Manhattan Project, which developed the atomic bomb during World War II, nor President Kennedy’s ambitious program to land a man on the moon, after the Russians appeared to have stolen a march with the launch of Sputnik, the first satellite in space.

Those programs succeeded because they were tremendous national commitments without regard to funding. The $1 billion in proposed funding for the “moonshot” cancer initiative is somewhere between modest and paltry. In the world of biomedical research, $1 billion simply doesn’t buy much.

The pharmaceutical industry estimates that it costs well over $1 billion to bring just one new drug to market. Cancer needs many drugs.

The lead agency in this new iteration of the war on cancer, declared in 1971, is the National Institutes of Health. It has an annual budget of $32 billion on which there are demands from many deserving fields of biomedical research besides cancer.

President Obama has asked Vice President Biden to lead the cancer moonshot effort. I’ve been with the vice president when he has talked about his commitment to the cause of cancer research and the death of his son, Beau, from brain cancer. His sincerity and his commitment to cancer research is palpable, but he won’t have the dollars to get the job done.

The biggest contribution to the research for a cancer cure may be the stimulation the moonshot will give to extant cancer efforts, but it’s not without a downside.

Many other diseases fear they may be undercut by the cancer initiative. In the world of biomedical research, there is finite funding and talent — and a new initiative tends to draw the best research minds. The top magnets for good biomedical researchers these days are cancer and AIDS, and many other deserving diseases lose out. Biomedical research requires stability, so that decades of a scientist’s life can be devoted to a single line of endeavor.

I follow one of the more obscure diseases, one that that has been pitiably starved of public and private funds: Myalgic Encephalomyelitis, also known as Chronic Fatigue Syndrome. Compared to any other disease affecting a large number of people (1 million victims of ME/CFS in the United States, according to the Centers For Disease Control), it has been funded so little by the government as to amount willful neglect. It receives a miniscule $5 million a year in funding.

Last year, after public and media pressure that has been applied for years, NIH Director Francis Collins announced that things would be rectified. But he didn’t mention a dollar figure; not when he made the announcement in October and not to date. No moonshot here, not even a Fourth of July firework.

Yet the suffering of those with ME/CFS is truly awful. I’ve been in the sick rooms and interviewed the few doctors who specialize in the disease, and the situation is one of unabated misery. Those who are the most affected can’t tolerate light or sound, and must pass their days in the silent dark. For years, one poor young man has had to take refuge from the disease in a modified closet. Others suffer from a world in which they’re punished for doing everyday things: A dinner with friends can mean days in bed for recovery.

There seems to be no light at the end of the victim’s physical pain and mental fog, despite decades of pleading from advocates and caregivers that some serious research be funded by NIH.

While we’ve been the world’s powerhouse in research in all sciences, biomedical is now being starved of research dollars. Recently America’s most revered virus hunter, Dr. Ian Lipkin, director of the Center for Infection and Immunity at Columbia University’s Mailman School of Public Health, has had to resort to crowdfunding. He and his deputy, Dr. Mady Hornig, can be found on YouTube eating red-hot chili peppers in an attempt to raise money for their ME/CFS research.

Dollars in across-the-board biomedical research are falling when they should be rising. Recently, NIH’s budgets have been 25 percent smaller in constant dollars than they were in 2003.

Research pays. Most of it doesn’t yield dramatic stuff, like a moonshot, but rather solid, incremental gain. In science, incremental gain is the equivalent of compound interest. But it needs sustained funding. Not rhetoric. — For InsideSources


A New Approach to Revolutionize Clinical Trials

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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