Report from Hotel Washington, 1/2 block from White House
Day 2 -- 1995 PAN Public Policy Forum -- Tuesday 16 May
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>>>>>>>>          "Invisible No More"         <<<<<<<<<<
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      Summaries of most of today's presentations:
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CYNTHIA LEBOW, Justice Dept: Reviewed how other Federal
offices (President's, Justice) have an impact on
legislation. Need contacts in White House (Domestic Policy
Office, National Economic Council), and the Office of
Management and Budget.
        Also, tort reform legislation may impact PD issues.
How many and how companies can be held liable in a product
defect suit will affect what new products are introduced.
Some companies may back away from (PD) R&D.
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RICHARD KLEIN, FDA: Outlined the drug approval process and how it has recently
changed. Most of the changes were for
quicker use of AIDS drugs. (Need lottsa clout to do that.)
A current FDA buzz phrase is "moral persuasion" -- the FDA
tries to focus on what is morally right.
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BOB NEUMAN (worked for Mo Udall): Most concerned about what the "religious
right" is doing now with Congress. Several religious leaders are lobbying to
halt genetic related research and patents. Several bio-tech companies have PD
products in process, which may stop.
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DR. THOMAS CHASE, 25 years with NIH (National Institutes
of Health, the largest bio-med research facility in U.S.):
Today NIH is a completely different agency. At first they
had more than what was needed for the few research
projects. He worked on early l-dopa research and PD. Now,
they have an enormous amount of research ideas but limited
funding. Money decreased as ideas increased. The cost of
research has continued to rise.
        Years ago PD had lots of attention, now other
diseases get the attention and money. New PD neurological
hypothesis can't be funded. Neuro-protective trials need
many more subjects than before. Now need 6 mos. to 2 years
and 100s of patients to conduct experiments.
        Pharmaceutical companies are retreating from risk.
Research paths require large potential markets to justify
the large investments and probabilities of return on
investment to stockholders.
        NIH should focus on lab findings, let academic
research on experimental areas, and let private industry
fill in the gaps.
        He theorizes that a diminished-returns relationship
exists in disease research. The longer a disease is
investigated, the more each bit of new information costs.
PD is still in its infancy in terms of total discoveries
yet to be made. Therefore PD research should be funded
more because we have so much to gain.
        Much basic neuro-science can be translated to
possible treatments for PD. We have genetic and
environmental hypothesis. Perhaps a combination of causes
produces PD.
        L-DOPA helps almost all PD patients, but it produces
complications as time goes on. More research needed.
L-dopa hits all 5 sub-types of dopamine receptors. Could
a drug that targets the receptors (D1,D2,D3,D4,D5)
separately or in some combination be more effective?
        PALLIDOTOMY has the same effect as blocking the
glutamate system. A pill could replace this surgery.
 
NEW IDEAS: everyone will get PD if they live long enough
(125 years). What causes the rate of brain cell death to
increase in PD. Exitotoxicity (sp?) -- glutamate is one.
A glutamate blocker may slow exitotoxin stress. Oxidative
stress is another -- free-radical accumulation in brain.
Current NIH research with OPC-1147 and Eldepryl anti-
oxidants.
        CALCIUM accumulation: sick cells tend to let calcium
in. Don't know why calcium enters. Two kinds of drugs:
calcium channel blockers and calcium moppers.
        TROPHIC FACTORS: cause developmental cells to
multiply and differentiate. Getting trophic factor
(proteins) into the brain is difficult. They may have a
preventative and restorative function on damaged nerve
cells. Brain damage is already 60-70% in PD -- extensive.
 
Pitch these messages to Congress: Emphasize the
humanitarian and human suffering, and cost. PD strikes
during working years and takes one out of work force and
onto disability. PD research results carries over to other
diseases.
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DR. IRA SHOULSON (Univ Rochester) trained at NIH, mentor:
Dr. Chase, works with FDA in approving PD drugs.
 
FACT 1: 1 million affected, 60,000 new case/year.
        Occurrence in minorities is equal to general pop.,
        but prevalence is less, therefore onset happens
        later in life.
FACT 2: PD is a major disability over age 40
FACT 3: PD is under-funded relative to other diseases
FACT 4: No major breakthru since l-dopa, 30 years ago.
        Major limitations in current l-dopa therapy.
 
PROSPECTS FOR FUTURE: At NIH have 2 kinds research:
1. basic (fundamental)
2. patient research
   a. basic patient research.
   b. clinical trials (most expensive, necessary)
 
Important clinical areas: identifying who is at risk and
who is already losing brain cells but not yet expressing
PD symptoms. Need to ID vulnerable population and try to
prevent the progress of the disease. Use neuro-imaging to
ID people at risk. Checking marker tissue.
        Improve l-dopa therapy. Much done in private sector.
I head 160 PD study groups in US and Canada (40 research
centers). When does one begin PD therapy? When people need
it. NIH doesn't think this is an important research area.
But if patients (PAN) want it explored, lobby NIH.
        Surgery therapies point research to areas that may be
helped with less drastic therapies.
 
PUBLIC POLICY ADVICE:
- need increased federal funding for all diseases.
- limit on discretionary funds, decrease defense
- increase neuro-degenerative disorders research
- more partnering between government and private sector.
- importance of animal research in neurology
- train more honest competent scientists
- speak with unified voice for PD research. There are 5-6
  organizations. PAN is attempting to unify this voice.
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QUESTIONS:
Can pre-symptomatic PD people be identified early?
        The best early diagnosis is still by a skilled
neurologist. Using instruments, the most promising area is
SPECT scanning. Research is mostly in the private sector.
The firm DOPA-SCAN has promising work. It seems that
government help in the already ongoing work may accelerate
these processes. Note that from the firms' perspective, a
market of only a million people is small.
        Previously NIH didn't trust the private sector. Now
they realize that was counter-productive. Government and
private sector partnerships when well organized can
very effective -- each partner has a specific role in
research development and marketing.
 
UNIFIED VOICE: Must be part of long term plan.
 
DRUG ALTERNATIVE TO PALLIDOTOMY: Surgeons have said the
pallidotomy is boring to do. Pallidotomy is still
experimental. NIH feels that a pallidotomy drug may be
feasible. No drug company will develop it due to high
costs and risks. Drug development is highly regulated,
surgery is not.
 
        FUNDING SOURCES: Total bio-med research -- 45%
government vs. 55% from private sector. NIH gets $11.3
billion, drug companies provide $18 billion (includes some
marketing).
        Specifically for PD: $26 million at NIH; and $2-3
million by American voluntary organizations (APDA, PDF,
etc.). No accurate estimate for the total amount of
private investment in PD research was known, but could be
at least matching. However, it was felt that the private
sector has contributed more research benefit than NIH.
Little funding in Europe.
 
GENDER: The healthy female spouse is a better caregiver
than a male spouse.
 
GENETICS: Familial PD exists. But is not yet defined.
 
MODELS OF PD: MPTP model, and 6-hydroy-dopamine model
 
NIH needs more young parkys for research.
(When PAN is properly organized, we could help.)
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JOAN SAMUELSON: Why are we here?
1. $ for PD research, via Udall bill.
2. Work directly with NIH. For example when PAN suggested
that NIH look at environmental toxins (drinking water, air
borne pollutants, etc.) the N. Carolina NIH Center added
PD back to its research programs.
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SENATOR HATFIELD (R-OR):
 
1. WE NEED MORE CO-SPONSORS!!!!
   (A current list of Udall bill sponsors will follow.
    If your reps are not co-sponsors, please lobby them.)
 
2. The balanced budgets proposed are a tragedy for the
NIH, bio-medical research, and the Udall bill.
There are 3 current budget proposals for NIH:
 - President's -->  4.1% increase this year, then
                   10%   decrease by year 2000
 - Senate -------> 10%   decrease this year
 - House -------->  5%   decrease this year
(each exempts AIDS and the Centers for Disease Control)
 
Fear protects the budgets for contagious diseases.
In this Decade of the Brain, we will destroy the first
5 years of neurological infrastructure already built.
The Udall Bill will be in rubble. "By the time we get our
foot in the door, they come with a sledge hammer and smash
our toes."
 
We need to lobby for:
BUDGET PRIORITIES: Discretionary-non-military spending is
only 17% of the budget. Increases are planned for the rest
of the budget (discretionary-military and entitlements).
In this 17% is the NIH. Not only will research on the big-
5 diseases(heart, stroke, cancer, but not AIDS) get cut,
but the little diseases, like PD, will also get cut.
 
MORAL ISSUES: Is a $1 billion B-2 bomber more important
than $26 million for PD research? Which one must be cut.
Regarding fetal tissue transplant vs. abortion issues: we
must disconnect the tissue donor from the donee.
 
MEDICAL ECONOMICS: Our "seed corn" is that for every $1
invested in medical research, we reap $13 in benefits.
Payback is enormous, 13 to 1. That's the best way to cut
the budget -- avoid future spending in Medicare, SSI, etc.
 
TOBACCO TAX TRUST FUND: Politicking continues for a
tobacco tax to raise about $4 billion/year for all bio-med
research.
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NOTES:
1. Typed by A.J. Conovaloff on Margaret Monty's and Bob
   Martone's laptops, posted by Bob (Board member,
   Houston, TX, Area Parkinson's Society).
2. A colorful PD informative computer presentation/slide
   show for MS-DOS has been prepared by Jim Cordy
   using PAN and other data, and the program Lotus
   Freelance. We showed the program on Margaret's and
   Bob's color laptops. His presentation is excellent for
   support group education. It can be viewed on a color
   computer, as overhead transparencies, color slides,
   on paper, and, we hope, on the WWW.
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    Morris K. Udall Parkinson's Research,
       Assistance and Education Act of 1995
                 Senate Bill S.684
 
                    CO-SPONSORS
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       HOUSE             |       SENATE
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(D-HI) ABERCROMBIE Neil  |(D-CA) BOXER Barbara
(D-NJ) ANDREWS Robert    |(R-MS) COCHRAN Thad
(D-VA) BOUCHER Rick      |(D-KY) FORD Wendell
(D-PA) COYNE William     |(R-OR) HATFIELD Mark*
(D-FL) DEUTSCH Peter     |(D-HI) INOUYE Daniel K
(D-MI) DINGELL John      |(D-NE) KERREY Robert
(D-CA) DIXON Julian      |(D-NJ) LAUTENBERG Frank
(D-MA) FRANK Barney      |(D-MD) MIKULSKI Barbara
(R-PA) GREENWOOD Jim     |(D-IL) SIMON Paul
(D-NY) HINCHEY Maurice   |(R-WY) SIMPSON Alan K
(D-FL) JOHNSTON Harry    |(R-AK) STEVENS Ted
(D-WI) KLECZKA Gerald    |(D-MN) WELLSTONE Paul
(D-NY) LOWEY Nita        |( -  )
(D-MA) MARKEY Edward     |
(D-WA) McDERMOTT Jim     |
(D-CA) MILLER George     |
(D-HI) MINK Patsy        |
(R-MD) MORELLA Constance |
(D-MN) OBERSTAR James    |
(D-NJ) PALLONE Frank     |
(D-AZ) PASTOR Ed         |
(D-VA) SISISKY Norman    |
(D-MA) STUDDS Gerry E    |
(D-NY) TOWNS Edolphus    |
(D-CA) WAXMAN Henry*     |
(D-CA) WOOLSEY Lynn      |
(R-MI) UPTON Fred        |
( -..)                   |
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               * principal co-sponsors
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              House Senate  Count (%)
             ,------------.
    Democrat |  24 |   9  |  33 ( 83%)
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  Republican |   3 |   4  |   7 ( 17%)
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       Count    27    13     40 (100%)
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                             NEED OVER 100!
 
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