February 22, 1999 07:42 AM
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  The
Parkinson's Action Network (PAN) was founded in 1991 to provide a unified,
national voice for the Parkinson's community and
to promote a level of research support sufficient to produce effective treatment and
a cure before the end of the decade.
This has required: Working with Congress and the Clinton Administration, directly and through grassroots advocacy, to increase their awareness of the needs of the Parkinson's community and the impact of their decision-making on that community. MAJOR RETURN ON INVESTMENT IN RESEARCH IN PARKINSON'S DISEASE AND RELATED NEUROSCIENCE PARKINSON'S DISEASE: CURRENT COST BURDEN: Several preventive and restorative strategies such as neural growth factors, gene therapy techniques and surgical therapies show promise in animal studies or human clinical trials. § Important links to the cause (including genetic susceptibility and role of toxic agents) are becoming established. Leading scientists describe Parkinson's as the neurological disorder most likely to produce a break-through therapy and/or cure within this decade. STAGNANT CURRENT NIH INVESTMENT IN PARKINSON'S RESEARCH: RETURN EXPECTED FROM INVESTMENT IN PARKINSON'S RESEARCH: According to Dr. 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 treatments within 2-3 years, an effective therapy
or cure within 5 years. PARKINSON'S DISEASE WHAT IT IS ------WHAT IT COSTS ------------- WHAT ACTION IS NEEDED THE DISEASE In Parkinson's disease or Parkinsonism (PD), cells that produce the neurochemical dopamine inexplicably degenerate, causing tremor, muscle stiffness and loss of motor function. Although medication masks some symptoms for a limited period, generally four to eight years in most victims, they begin causing dose-limiting side-effects. Eventually the medications lose their effectiveness, leaving the victim unable to move, swallow or speak. PD is the biological opposite of Alzheimer's: While Alzheimer's destroys the mind, leaving the body intact and functioning, Parkinson's destroys the body's ability to function, taking away the physical abilities necessary to daily life and leaving the mind prisoner inside it. Although the cause is still uncertain, environmental toxins are a prime suspect. In the June 1993 Neurology, for example, researchers concluded that the "relative contribution of environmental agents" to young-onset Parkinson's "appears to be fairly large," and that their findings "add to the increasing weight of evidence that relates PD risk to exposure to pesticide-related products, mainly . . . insecticides or herbicides." THE COST The NIH estimates that between 500,000 and 1,500,000 Americans are afflicted with Parkinson's, with 50,000 more diagnosed each year. Approximately 40% are under the age of 60, effectively removing them from the productive work force. Unlike many other deadly ailments, Parkinson's victims remain alive but incapacitated for many years, sometimes decades, requiring a similar number of family members to be diverted from the work force by their role as caregivers. As a result, PD is estimated to cost the U.S. $25 billion a year in direct health-related expenses, indirect disability related costs and lost productivity. THE POTENTIAL FOR A BEAKTHROUGH Great advances in neurological research in the last few years have created the potential for major treatment breakthroughs, very possibly a cure -- in this decade. Such potential caused the Congress to declare the 90's the "Decade of the Brain." Among those scientific developments are: Neural Growth Factors: These "trophic" factors hold the potential for rejuvenating dormant neurons that have ceased to function in the Parkinson's-afflicted. Scientists are anxious to proceed to clinical research, since it appears these growth factors, once developed, may reverse symptoms and restore victims' ability to function. Fetal Tissue Implants: Scientists have produced remarkable results in animals and in the few humans able to participate in clinical trials during the six-year ban on federal support. While it is too early to predict its full potential, further clinical trials are now underway, and scientists expect to improve on these encouraging results. Genetically Engineered Cells: The creation of neural cells through genetic engineering is expected to add an additional means, beyond the use of fetal tissue, to replace dead brain cells. Scientists are working to develop this capability. THE NEED FOR A FAIR, ADEQUATE RESEARCH BUDGET History of Low Investment: Parkinson's has been very low on the funding priority list for years. Parkinson's research support from NIH in 1996 totaled $32 million. While federal funding of Parkinson's research in 1996 amounts to approximately $32
per patient, in comparison, research funding for most disabling or deadly diseases has
been substantially greater. The following chart details NIH research funding per patient
in 1994, for selected diseases.
This disparity exists because the Parkinson's community has been largely invisible: too crippled and too overwhelmed by their symptoms to function publicly, they disappear from society -- and Washington. In addition, bureaucratic roadblocks like the six-year ban for fetal tissue transplant research stalled significant research contributions. This unfortunate and inadequate level of funding must be rectified now, beginning with the 1996 federal budget. Investing in Parkinson's and other neurological research will not only save millions of Americans from immense suffering; it is also an economic tool that will help fight the deficit, pay for health care reform, and strengthen the U.S. economy -- in this decade. ROUTES TO A PARKINSON'S CURE . . . The symptoms of Parkinson's disease result from the degeneration of nerve cells in the mid-brain, and the corresponding loss of the neurotransmitting chemical dopamine produced by those cells. Conventional treatments revolve around pharmaceutical substitutes for dopamine (such as L-dopa) and drugs that temporarily enhance the cell's dopamine production. Such measures lose their effectiveness as more cells are lost; so a true Parkinson's cure requires finding ways of replacing damaged cells with healthy, viable ones...or nurturing those damaged cells back to life. GENETIC RESEARCH: Proponents in the research community maintain that identification and mapping of a Parkinson's gene is a very direct route to a cure...and that the gene could be identified within months. Such an advance would not only provide a means for identifying people at risk, but would potentially reveal the function of the gene -- how it triggers or effects a change in the nerve cells at risk. NEURAL TISSUE TRANSPLANTS: Researchers implant fetal neural tissues into the degenerate area of the brain, with the object that the new tissue will thrive and renew the production of dopamine. Dramatic results have been achieved in clinical studies, and a prototype therapy may be close at hand...however, increased funding is necessary to broaden the test population, increase cell survivability and determine precise implantation techniques. NEUROTROPHIC PROTEINS: Researchers are identifying a growing number of proteins that function to nurture nerve cells...and even appear to restore life to "dead" cells. Although "nerve growth factor" and similar proteins are relevant to many neurological diseases, at least one protein has been directly linked to the survivability of dopamine cells. Advances toward a neurotrophic cure will require novel ways of delivering a therapy inside the brain's largely impermeable blood-brain barrier. NEURO-PROTECTIVE AGENTS: On a different analytical plane, the damage done to nerve cells that result in Parkinson's and some other neurologic diseases is viewed as the work of "free radicals" -- molecular, metabolic by-products that can destroy healthy cells. Researchers are closing in on a naturally occurring enzymes that appear to deactivate free radicals in a healthy brain, and are testing antioxidant drugs that could mop up molecules before they do damage. GENETIC ENGINEERING: Scientists are modifying the genetic code of individual cells to obtain ways of supporting and extending these therapeutic technologies...for example, altering a patient's skin cell to become a dopamine-producing cell, one that could be implanted in the brain without rejection. Other researchers have fabricated an "adenovirus," a viral agent capable of invading a nerve cell and reprogramming the genetic code to produce dopamine. ENVIRONMENTAL LINKS: While some researchers are closing in on a Parkinson's gene, others are focusing on the increasing evidence that environmental toxins play some role...perhaps as a trigger in conjunction with a genetic susceptibility. FETAL TISSUE TRANSPLANTATION: THE SCIENCE, THE ETHICS, THE POLITICS BRIEF HISTORY OF THE SCIENCE AND POLITICS. For decades, researchers worldwide have been experimenting with the use of transplanted fetal neural tissue as a therapy for Parkinson's and other disorders including diabetes and various immune function disorders. In the mid-80's, success with laboratory and animal studies in curbing Parkinson's symptoms by substituting the fetal cells for degenerated dopamine cells led scientists to begin human clinical trials. When scientists at the federal National Institutes of Health sought to assist such work with intramural and extramural support, however, they were blocked by the imposition of a ban on federal support for such research by Presidents Reagan and Bush. In 1991 and 1992 the Congress passed legislation lifting the ban and adding a series of ethical guidelines to govern any federally-funded transplantation research using fetal tissue. The provision, known as the Research Freedom Act, was passed by the House (274-144, 7/25/91) and the Senate (87-10, 4/2/92). Despite support from strong opponents of abortion, including Senators Mark Hatfield, Robert Dole, John Danforth, and Strom Thurmond, the Act was vetoed by President Bush and a veto override failed in the House by 13 votes. The Act was again passed and signed by President Clinton in 1993. FETAL TISSUE TRANSPLANTATION IS NOT MENTIONED IN THE UDALL BILL. The Morris K. Udall Parkinson's Research, Education and Assistance Act, the first legislation to address the longstanding need for an expanded Parkinson's research program, authorizes $100 million in federal support. The bill contains no restrictions on what research should or should not be included. The intent is to advance research breakthroughs as fast as the current scientific opportunity permits. Nationwide experts in Parkinson's research and participants at a 1995 NIH-sponsored Parkinson's research planning conference have identified a comprehensive research agenda full of breakthrough potential that caused the Dana Alliance for Brain Research 1996 Report to name Parkinson's as "one of the brightest spots in brain research." Neural cell transplantation is only one component of this agenda. DESPITE THE OBSTACLE OF THE EIGHT-YEAR FEDERAL BAN, NEURAL CELL TRANSPLANTATION SHOWS GREAT PROMISE. Although the eight-year ban on federal support undoubtedly slowed the progress of such work, cell transplantation is showing great promise as an effective therapy for Parkinson's, Huntington's and other disorders. Results from human clinical trials in the U.S. and elsewhere show continued symptomatic mprovement as researchers refine their understanding of the process. Two Parkinson's patients (one in the U.S.) are reported off medication and symptom-free. THE ETHICAL GUIDELINES PRACTICED BY RESEARCHERS AND CODIFIED IN THE RESEARCH
FREEDOM ACT PREVENT THE PROMOTION OF ABORTION. A woman may not be approached for consent to donate the aborted tissue until after she has made the decision to have an abortion. The donor may not be paid for donation of the tissue. The donor may not designate who will be the recipient of the tissue, nor be informed of the recipient's identity. Violation of the restrictions is a federal felony punishable by 10 years in federal prison. There is no reported instance of any infringement of these guidelines, nor of any evidence that any woman has been encouraged to have an abortion as a result of transplantation research. TISSUE FROM SPONTANEOUS ABORTIONS OR ECTOPIC PREGNANCIES IS NO ALTERNATIVE. THE SUCCESS OF FETAL CELL RESEARCH WILL NOT DEVELOP A DEMAND FOR MORE ABORTIONS. If you have comments or suggestions, email me at: BobMartone@Kingwoodcable.com ---------------------------------------------------------------------------
TABLE OF CONTENTS
SECTION III: THE UDALL SOLUTION
SECTION IV: HOW CONGRESS WORKS
SECTION VII: LEAVE BEHINDS Parkinson's Disease - What is it? What does it cost? The need for fair adequate funding.
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