From:   [log in to unmask] (Parkinson's Action Network)
Reply-to:       [log in to unmask]
---------------------------------------------------------------
TESTIMONY OF JOAN I. SAMUELSON
PRESIDENT, PARKINSON'S ACTION NETWORK
Hearing of the House Appropriations Committee
Labor, Health and Human Services Subcommittee
February 4, 1998
I am one of a million Americans afflicted with Parkinson's disease and
related disorders.  I also am President of the Parkinson's Action
Network, which was created in 1991 to give a voice to our community in
the effort to speed research delivering breakthroughs and a cure for
this dreadful disorder.

I have the job today of focusing your attention on the particular needs
of my community, and  to convince you that the 1999 budget of the
Labor-HHS Appropriation must — yes, must — include a substantial
increase for Parkinson's research funding, pursuant to the $100 million
authorization in the Udall Parkinson's Research Act enacted in the last
Congress.

Why am I so emphatic?

· Because the current federal policy on Parkinson's wastes billions in
public and private dollars coping with its effects, when millions would
produce a therapy that would restore function, and bring us back into
the world.

· Because the disparity in funding attributable to variations,
invisibility or political clout cannot continue.

Parkinson's — the disorder:  Parkinson's is a movement disorder caused
by the degeneration of brain cells that produce dopamine, a
neurochemical controlling motor function.  By the time 80% of those
cells stop functioning, symptoms of stiffness, tremor and slowness of
movement begin to emerge.

The conventional treatment for Parkinson's is a 30-year-old drug
commonly known as "L-dopa"  which attempts to replace the missing
dopamine with an artificial substitute.  It usually restores function to
a certain extent and it may seem at first like a miracle drug.  But it
works inefficiently, it produces side-effects, and eventually it does
not work at all.  As the dopamine cell degeneration advances, it strips
away automatic movements needed to walk, talk, swallow, even move at
all.

Parkinson's — the impact:  Initially, we survive on a diet of desperate
hope, fed by L-dopa and related medications.  We attempt to keep work,
family and life moving smoothly, as the symptoms change in degree and
combination throughout the day, affected by diet, stress and fatigue.
Almost immediately, though, things dear to life are taken, such as, in
my case, the love of running and backpacking.  That begins a process of
loss that advances to strip away essential functions.

The impact on work:  Very soon it begins to affect working life, making
jobs dependent on motor skills impossible and jobs with any measure of
stress increasingly difficult.  In a 1988 study, a group of researchers
at the University of Rochester calculated that of the 44% of Parkinson's
patients in the first stages of the disease, 31% would lose their jobs
within one year as a result of Parkinson's.

Despite the common myth that Parkinson's only affects the oldest sector
of the country, in fact the average age of symptom onset is 57, with a
third of all victims' symptoms starting in their 20's, 30's and 40's.
As a result, Parkinson's-caused early retirements and forced disability
are the norm.  Some lose their jobs simply due to the stigma.  The
financial impact is enormous.

Every sort of work is affected.  People who must have reliable motor
movement to do their work — beauticians, house painters, typists — lose
their employment quickly.  For the rest of us, it is a somewhat slower
process, but at some point the tension of worrying about how to fit a
job's demands in the daily schedule of Parkinson's symptoms simply is
too much.

In my case, as a practicing lawyer and now running am advocacy
organization for our community, these are my daily struggles: worrying
about getting to a morning meeting and wondering when my first dose of
medication will "kick in," enabling me to function; needing to make a
phone call, but not being able to hold the telephone still with a
shaking hand; seeing others put off by my lurching gait, or my trembling
hand.

The impact on daily functioning:  At some point the symptoms become an
impossible hurdle, as the tiny number of dopamine neurons left
functioning just can't team up with the medication any more, and are
complicated by drug side-effects.  At that point, the swing between too
little and too much movement is just too much to manage in the outside
world.  We may continue living for a long time, but we drop out of
sight.

The nation — indeed, the world — has been riveted on the impact this
disorder has had on Muhammad Ali.  It is essential to remember the
unknown Americans who, like Ali, are losing the battle to live a normal
life.  They tell of family holiday dinners they can't attend, for fear
of knocking food off the table.  They talk of walking into the bathroom,
then suddenly freezing up and needing help to finish bathing or using
the toilet.

Every person afflicted with Parkinson's can describe the effort to
manage their medication so they are at their best when out of the
house.  And then, one day, that person starts disappearing, as the act
of coping becomes too much.  Perhaps if we died soon as a function of
Parkinson's its impact would appear more dramatic.  Instead, we slip out
of the functioning world and are forgotten.

The impact on our visibility: There is a common denominator in all these
cases: we start out courageously trying to power through our disability,
ignoring it, even hiding it, to maintain our normal lives.  We end up
silenced and imprisoned in our homes, our care facilities.  In either
case, there is an insidious ingredient: our suffering has been rendered
invisible to the outside world.

As a consequence, we have been neglected.  Attached is a chart, from
numbers provided by the NIH, showing direct Parkinson's research funding
by the NIH to be flat, at less than $30 million a year — that is, less
than $30 per patient — for a decade, and only slightly more now.
Estimated 1997 direct Parkinson’s spending totaled $34 million, or $34
per patient.  During a decade in which the American public and the
Congress have teamed up to attack a wide variety of killing and
disabling disorders, we have left out of the fight.

We are finally here, coming out of the closet in some cases, coming in
wheelchairs and walkers in others, and being represented by loved ones
when we can't get here at all.  And the Congress is beginning to
respond.  Last fall Congress enacted the Morris K. Udall Parkinson's
Research Act, which would authorize the long overdue expansion of
Parkinson's research, with annual research funding at $100 million a
year, which is the amount that Parkinson's researchers tell us they need
to maximize existing scientific potential and speed the breakthroughs we
need.  We are looking to this committee to fund it, with a $100 million
appropriation in the FY 1998 budget.

There is a terrible irony at work, however.  As we walk the halls of
Congress to drum up support for the Udall bill and the funding it will
need, we are told that no longer will Congress direct the NIH in its
funding needs, and that there is no more money to spare.  In effect, we
are told that our visibility comes too late.

There are two reasons we cannot accept this answer.  First, it is
grossly unfair to maintain a status quo funding disparity that, in
effect, continues to penalize us for our disease-caused invisibility.
As any review of NIH spending will show, technical elimination of
earmarking has by no means eliminated de facto earmarking: spending by
diseases continues at rates pre-ordained by earlier increases.  As our
"funding disparity chart" (attached) is just one such example.

The President’s recent announcement of a doubling of the Cancer
Institute budget over the next five years demonstrates this best.
Earmarking continues under other names.  Parkinson’s continues to be
excluded.

There is another important reason the Congress must increase Parkinson's
funding in 1999.  As federal taxpayers, we are owed a rational health
spending policy.  That requires spending money to cure us rather than
just care for us.

The cost to America: The cost of Parkinson's in America is massive.  In
testimony before the Senate Special Committee on Aging in 1995, Dr. Ole
Isacson of Harvard estimated the cost to be in excess of $25 billion.
The Network's surveys of the costs Parkinson's disability incurs on the
country -- in treatment, physical therapy, hospitalization, disability
payments, lost productivity, and assisted living -- indicate an equal or
greater amount, which translates into a massive burden on public sources
such as Medicare, Medicaid, and Social Security disability.

The cost is so high because we typically live in a disabled state for a
long time, and the battle against loss of function is ongoing, and
expensive.  Parkinson's medication alone is very expensive, probably
costing Americans well over a billion dollars.  The largest costs can be

due simply to losing the ability to work or care for oneself, which is
absorbed by the government through higher Social Security, Medicare and
Medicaid spending. This takes a huge toll on the American families hit
by Parkinson's, but it also burdens the society and hits the taxpayer.

This massive financial waste will rise steeply if Parkinson's is not
cured before my generation of "Baby Boomers" hits the years when
Parkinson's symptoms are most prevalent.  Imagine the additional burden
of lost tax revenue, medical care and disability from Baby Boomers with
Parkinson's.

The scientific promise: An examination of the scientific promise of this
disorder shows that an investment in Parkinson's research would return
many-fold.  The Dana Alliance for Brain Initiatives describes
Parkinson's as "one of the brightest spots in brain research."  There is
no doubt that huge, revolutionary breakthroughs are coming, and they
will drive breakthroughs for many other neurological and
non-neurological disorders — Huntington's, ALS, Alzheimers, spinal cord
injury, diabetes and more.  Consider:
· Neural growth factor, in particular one known as GDNF.  Animal studies
shows the growth factor revives the dormant cells and produces dramatic
symptomatic improvement.  Human clinical trials have begun.
· Neural cell transplantation, which has shown that symptomatic
improvement results from the flourishing of transplanted dopamine
neurons.  A few patients are now symptom-free without  medication.
· Advances in genetics and in links between Parkinson's and
environmental factors such as heavy metals, herbicides and pesticides.
· Steady increase in insights into the exact disease process, in which
the cells appear to self-destruct after assaults from one or more of
those causative factors.
· Rapid advances in the understanding of the role of genetics in
Parkinson’s, which also brings new clues about the disease process—and
its undoing.  A widely-cited 1997 discovery of the alpha-synuclein gene
did not produce a causal gene per se, but is a major clue in the matrix
of understanding.

But without question, those discoveries are coming in slow motion.
Every scientist describes immense frustration with the slow pace of
working on these breakthroughs because of the tiny research investment.
That translates directly into a breakthrough deferred into the future.
According to testimony before the Aging Committee last year by Dr. Ole
Isacson of Harvard, an additional $20-40 million per year spent to fund
100 of the most effective preclinical and basic research programs (@
$200,000-$400,000 each) will produce new Parkinson's treatments within
2-3 years, an effective therapy or cure within 5 years.  According to a
study by Dr. Roger Kurlan of the University of Rochester, even a 10%
slowing of progression will save $327 million per year.  That is ten
times more than the federal government is spending on Parkinson's
research.

We have been told there may not be enough money in the Labor-HHS
allocation to fund the Udall Act in full.  It is suggested that we are
forcing a reduction in funding for diseases that have done a better job
at telling their story, and been rewarded by higher research
investments.  Although we feel it is important to point out this
disparity, reducing productive research efforts in other biomedical
arenas is not the way to do it.

But the money must be found.  Perhaps Mo Udall is beyond saving.  But is
Muhammad Ali?  What about Billy Graham, or the Pope, Attorney General
Reno or Johnny Cash?  And what me, or about the unknown, invisible
Americans I represent today?  The 1999 Labor-HHS appropriation must
provide the $100 million authorized because this country literally
cannot afford to lose me or the many others in the same predicament.
With enough functioning dopamine neurons back in action, all of us could
be back to working, caring for others as well as ourselves, paying taxes
instead of applying for Social Security disability.

Conclusion:  The human suffering that results from Parkinson's is
immense and incalculable. That alone is a good reason to invest in a
cure.  The fiscal drain compels it.  The only reason we have been
neglected is our historical invisibility, caused by the ravages of
Parkinson's, the devastating stigma attached to it, and inevitable
silencing of us it causes.  We now have "come out," as it were, to tell
our story, only to be told it's too late, that earmarking funding on the
basis of need, or scientific promise, or fiscal sense, is
inappropriate.  That makes no sense.

We desperately want to hold on to our life dreams and our dignity.
That, given the scientific promise of therapeutic breakthroughs, should
be enough to justify the investment.  What we also know now is that it
is fiscally irresponsible not to.  The Udall Parkinson's Research Act’s
$100 million authorization must be funded in FY 1999.
 * * * * *
From: "Parkinson's Action Network" <[log in to unmask]>