Wednesday, July 15, 2015

Dear Dr. Collins: I’m Disabled. Can the N.I.H. Spare a Few Dimes?

Dear Dr. Collins: I'm Disabled. Can the N.I.H. Spare a Few Dimes?
By: Brian Vastag | July 14, 2015

Three years ago, a sudden fever struck veteran science writer Brian
Vastag on a blue-sky Wisconsin morning. He's been sick ever since. Now
cognitively and physically disabled, he lives on the island of Kauai.
On Brian's third "illiversary," he presents an opportunity to National
Institutes of Health Director Francis Collins.

Dear Dr. Collins,

You might recall the last time we spoke. It was January 2013, and I
was working as a science reporter at The Washington Post. Your people
arranged an early call for you to announce that the N.I.H. had decided
to retire most of its research chimpanzees. We spoke for about 20
minutes, and I typed up a 600-word story. It wasn't very good.

I was working from home that gray day because I had little choice. I
was mostly bedbound then, seven months after a sudden fever had
knocked me prostrate. My legs were so weak that climbing the stairs to
my home office required pulling myself up the railing hand-over-hand.
My brain was so sluggish I asked few questions of you. The ones I
managed to croak out were poor, no doubt.

Too sick to work, I did anyway. I loved my job at the Post – not an
easy gig to come by – and I was desperate to keep it. I had been
following the research chimp story for a while, and I liked that the
N.I.H. chose me to break your news.

I've long appreciated how the N.I.H. helps the world. My career began
there in 1998, when Paul van Nevel hired me for a science writing
fellowship at the National Cancer Institute. I count your
communications director John Burklow among my mentors, and I was
honored to write Paul's obituary. That first boost of professional
success propelled me to an exciting career.

Lately, though, my love for your august institution has been strained.
You see, I've been felled by the most forlorn of orphan illnesses. The
most accurate name for it is myalgic encephalomyelitis, which means
"painful inflammation of the brain and spine." (Yes, it is painful,
and yes, there's strong evidence of neuroinflammation.) At the N.I.H.
and elsewhere, it is instead called chronic fatigue syndrome. That's a
terribly vague and dismissive moniker for so serious an illness, and
one that needs to be retired. Fatigue is not the primary or most
troubling symptom for most people with M.E.

In the past, you've shown a soft spot for certain orphan diseases. Well, the history of M.E. is akin to locking an entire orphanage in a cellar and bulldozing the house.

On the list of illnesses the N.I.H. studies, M.E. (listed as "M.E./C.F.S.") is near the bottom in funding, ranked 231 out of 244. It received $5 million in 2014, less than hay fever, which cripples no one.
That's not enough money to equip a laboratory and run it for a
year. This abject neglect – or sustained prejudice, or maybe both –
stretches back a full three decades at the N.I.H. (For a detailed
history of how this sad state came to be, read Hillary Johnson's
deeply-reported book Osler's Web.)

No one has a good count of M.E. patients in the U.S. – the C.D.C. misspent funds earmarked for this purpose – but there's plausible evidence that several hundred thousand people are disabled by it. That's comparable to the burden of multiple sclerosis – the illness M.E. most closely resembles. N.I.H. funds $110 million in M.S. research each year, and a sustained government investment has been crucial in the development of a dozen F.D.A.-approved M.S. drugs. M.E.
patients have no approved treatments.

But M.E. is finally emerging from the basement. Brand name
institutions and big-time researchers now recognize the huge burden
M.E. places on society – tens of billions in medical expenses, lost
productivity, and missing tax revenue each year. Columbia University's
Ian Lipkin is searching for infectious triggers, and has reported
severe immune problems in patients. Columbia received $150 million in N.I.H. grants in 2015; Lipkin's operation gets a big chunk of that. But when the famous virus hunter applied for a trifling $1 million for M.E. research, the N.I.H. turned him down, twice. So spurned, Lipkin and colleague Mady Hornig recently resorted to eating habanero peppers to raise money.

Jarred Younger, at the University of Alabama-Birmingham, has a list of
off-the-shelf drugs and supplements that can reduce neuroinflammation.
They urgently need to be tested in patients. In Norway, a phase three
clinical trial of the cancer drug Rituximab in M.E. holds great
promise. In early trials, nearly two-thirds of patients improved after
repeated doses of Rituximab, and a quarter went into full remission.
Such promising results, if reported in cancer patients, would be
trumpeted as a breakthrough.
Additional studies to sort out which
patients might benefit from this drug are urgently needed; the N.I.H
Clinical Center would be a fantastic home for such trials. It's
unseemly to let a tiny Scandinavian country overtake the enormous U.S.
of A. in this, or any, realm of medical research.

At Stanford University, prominent geneticist Ron Davis is searching
for genetic risk factors. His investment is deeply personal, as his
adult son – formerly a world-traveling photographer – is severely ill
with M.E., and can no longer walk or talk.

When you peruse the recent M.E. literature, you'll see a mix of young
researchers and experienced lab leaders producing a string of insights
into how the illness damages the immune system and the brain.
Mutations in the gene MTHFR have been identified by the Open Medicine
Foundation as a risk factor. Diagnostic biomarkers await validation.
Promising treatments need to be tested in patients. And all of this
has happened with little support from the N.I.H.

Patient advocates have called for $250 million in M.E. research
funding, a figure commensurate with the burden of disease. This is a
huge ask, and in all likelihood politically infeasible, so let me make
a smaller one. A new N.I.H. program funded with as little as $10 million to $20 million per year would be absolutely transformative for the field – and for patients. Such a program would affirm the N.I.H.'s commitment to understanding the illness. It would draw more young researchers to studying M.E., and it would encourage further private funding. Such a commitment would also give patients – many housebound or bedbound for decades – hope that they'll be healthy again. I challenge you to find another illness where such a small investment could make such a huge difference.

You now have broad support from the medical community to make this
happen. Earlier this year, the Institute of Medicine made a strong
call for a robust M.E. research program. And just last month, an
N.I.H.-appointed panel urgently made the same recommendation. With the
bipartisan 21st Century Cures Act poised to pass Congress – giving
N.I.H. an extra $8.75 billion over five years – you could do so
without pulling money from existing programs. At the same time, you
could help things along by moving responsibility for M.E. from its
long-term parking spot at the Office of Research on Women's Health to
one of the institutes that, you know, funds disease research.

A year before I fell ill, I backpacked Rocky Mountain National Park.
My legs carried me up to the continental divide, where I sat on the
edge of a precipice marveling at the peaks around me. At nearly 13,000
feet above sea level, the thin air addled my thinking – a feeling I
now live with every moment, as if someone poured molasses into my ear,
gumming up all trillion synapses. It's a terrible way to go through
life, especially so for someone who not so long ago made a good living
with his brain.

Here in Hawaii, there's a smaller mountain behind my yard. It's called
Sleeping Giant, and the giant's forehead juts less than 500 feet above
my back patio. A well-trod trail carries people up there for sweeping
views down the volcanic slope and across the endless Pacific. Oh how I
would love to drink in that view. But I may as well be gazing up at
K2; a summit attempt would be supremely unwise, as a sophisticated
exercise test found that I suffer from severe metabolic, cardiac, and
pulmonary dysfunction. Exercise for M.E. patients is more damaging
than sugar is to a person with diabetes.

At 43, my productive life may well be over. There's a good chance I
have hiked my last trail. The nation's coffers lose some $25,000 in
tax revenue each year I remain disabled, and I will soon know if
Social Security Disability Insurance will start coming my way. I don't enjoy being a drain on society, and neither do any of the other M.E.
patients I know.
And yet, with the ever-growing research interest in
M.E., I have hope that someday I'll be able to stand for more than a
few minutes, walk for more than a block or two, maybe even resume my
career. (It took me four days, with frequent breaks, to write this
letter…that's a bit slow for newspaper work.)

The causes of M.E. will eventually be discovered, treatments will be
found, and patients will enjoy long-term remissions. As the leader of
our nation's medical research enterprise, you have a decision to make
– do you want the N.I.H. to be part of these solutions, or will the
nation's medical research agency continue to be part of the problem?
At the very least, you could ensure Dr. Lipkin doesn't have to scorch
his intestinal tract again just to drum up a few research dollars.


Brian Vastag

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