 |
 |
Dear Friends, At the request of my father, Harold Jones, I am reposting the Testimony of Joan I. Samuelson & the Testimony of Muhammad Ali and Abraham Lieberman, M.D to the list because he has had requests for it in response to a message he sent to the list recently. Karen Bardo ******************************************************************************** ******************************** 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. ******************************************************************************* ******************************** National Parkinson Foundation, Inc. Written Testimony of Muhammad Ali and Abraham Lieberman, M.D. before the House of Representatives Commerce Committee Subcommittee on Health and Environment "New Developments in Medical Research" 2123 Rayburn House Office Building March 26, 1998 Mr. Chairman and distinguished members of this Subcommittee, my name is Abraham Lieberman. I am Medical Director of the National Parkinson Foundation and Executive Director of the Muhammad Ali Parkinson's Institute in Phoenix, Arizona. I am accompanied today by my friend and patient, Muhammad Ali who, of course, needs no introduction, and Mr. Nathan Slewett, volunteer Chairman of the Board of the National Parkinson Foundation, whose worldwide headquarters are located in Miami, Florida. We are here at the invitation of Chairman Bilirakis whom I want to personally thank and commend for holding these hearings on "New Developments in Medical Research." Muhammad has assured me that he would have preferred to speak directly to you today, but his Parkinson's disease has robbed him of what was one of his most prized assets....the ability to speak with resonance. I can assure you, however, that his thoughts and muted speech remain as eloquent as ever. We are here today for two principal reasons. First to enlighten this committee and thereby the United States Congress, on the pathology of Parkinson's disease, at least what we currently know about its causes, course and treatment. Also, we would be remiss if we failed to use this opportunity to demonstrate once again, why more research resources are needed NOW to take advantage of some of the fantastic scientific breakthroughs that are at our door step. Parkinson's disease (PD) affects more than a million Americans, and costs the United States more than five billion dollars a year in Medicare, Medicaid and nursing home outlays. In addition it costs each patient five thousand dollars a year for medications, doctor's visits and hospital stays. As a society we spend ten billion dollars a year for PD, without considering the indirect costs of lost income, missed opportunities and family disruptions. The ten billion dollars results in treatments that provide only temporary relief because there is no cure. No Parkinson's patient, no care giver, no doctor is happy, nor considers the ten billion dollars well spent. At the time of diagnosis, every patient had probably had Parkinson's disease for at least four years. Based on the time between disease onset and diagnosis, it's estimated there is at least one person who is undiagnosed, for each patient who is diagnosed. In other words, there are probably two million Americans with PD: 2% of the population over age 60, and 4% over age 70. The degeneration of nerve cells in Parkinson's disease is associated with a particular particle, called a Lewy body, in each dying cell. Based on the observation that the Lewy body is a marker for PD, and that 10% of the population over age 60 years have Lewy bodies without symptoms of PD, it's estimated that at least ten million Americans may develop PD, if they live long enough. Lest the young be sanguine, and relegate PD to senescence, 15% of all patients who develop PD are less than 50 years of age. And the disease appears in adolescence. Given the increased chance of developing PD with increased life, what is the point of conquering cancer, heart disease, stroke, and diabetes if the end result is a population, 10% of whom have PD and 10% of whom must care for the afflicted or live in dread of being afflicted? Public health, preventive medicine, and environmental safety are laudable goals only if the resultant increased life span is satisfying and free of the plague of Parkinson's disease. Parkinson's disease results from the death of pigmented nerve cells in the substantia nigra, part of the basal ganglia, the region of the brain between the hemispheres, the creators and initiators of thought and movement, and the brainstem and spinal cord, the executioners of movement. In the past ten years, as a result of developments in molecular biology, genetics, neurophysiology, and computer science, the mystery of cell death is being unraveled. It's known that some neurons are more susceptible to dying than others, that their death is associated with both external (to the cell) and an internal (within the cell) generation of free radicals and this, in turn, may be associated with the deposition of iron. Sadly, the end result of Parkinson's disease is that the human brain, the highest expression of creativity--rusts. Whether the cause of Parkinson's disease is an externally produced toxin, AN internally generated poison, a defect in the cell itself, or an abnormal gene is the now the subject of intense thought, debate, study, and experimentation. A debate that, as recently as ten years ago, was conceptually impossible and scientifically unprovable. Unraveling the mechanism of cell death in PD, will increase our understanding of the more complicated, disintegrative mechanisms in Alzheimer's disease and the aging process itself. Since the introduction of levodopa in 1967 it has been the mainstay of treatment for PD. Levodopa is combined with a dopa decarboxylase inhibitor, to facilitate its passage from the gut. There are several drugs including MAO-B inhibitors, COMT-inhibitors, and controlled-release levodopa preparations that enhance levodopa's activity. There are several other drugs, dopamine agonists, bromocriptine, pergolide, ropinerole and pramipexole, that activate the dopamine receptors. Recent discoveries have shown that nerve cells, heretofore considered incapable of regenerating themselves, are able to do so. This regeneration occurs under the influence of trophic, or growth factors, small molecules, poly-peptides, produced in the brain. There may be hundreds of trophic factors, each one specific for one or two nerve cells, so as to limit uncontrolled growth, and its resultant tumor induction. Several factors have been identified, characterized, engineered and tested in animal models of PD. One of them glial derived neurotrophic factor (GDNF) is undergoing clinical trials for Parkinson's disease. Knowledge of the mechanism of action of trophic factors will increase our understanding of cell death and aging and will lead to treatments heretofore thought possible only through immersion in the legendary and elusive, "Fountain of Youth." The challenge is to determine which trophic factors are relevant to which nerve cell, and which disease, to deliver the factors into the nervous system and target them to the relevant cells. These are complex engineering problems, but ones, as with the World War II Manhattan Project which produced the atomic bomb, are within the realm of being solved with our present technology. Within the past ten years neuroanatomists, neurophysiologists and neuropathologists have distinguished differences between the caudate nucleus and the putamen, the two components of the striatum. They have separated the ventral from the dorsal caudate, and ventral from the dorsal putamen, and have identified several cell types within the striatum. Meanwhile the neurochemists and molecular biologists have further characterized the striatum's nerve cells by co-localizing them with one or more of several neurotransmitters. The significance of the striatum's newly discovered external geography, and internal architecture is being scrutinized in PD and heretofore unknown relationships are emerging. While the external characteristics of the striatum were being studied, the more important and challenging task of understanding the internal biology of the individual cells has proceeded simultaneously. Specific proteins, and poly-peptides are produced in specific cells, and the ability of a cell to produce a specific protein defines the individuality of the cell. The ability to replicate, amplify or inhibit these interactions will have consequences for our health and our civilization as profound, and as monumental as splitting the atom. Thirty percent of Parkinson's disease patients develop a dementia which has many similarities to Alzheimer's disease. A smaller portion of PD patients develop a dementia, at an earlier age, called Lewy-body dementia. Whether the dementia of Parkinson's disease and Alzheimer's disease are different, or the same is now under intense investigation. Approximately 50% of Parkinson's disease patients become depressed during the course of the illness and require counseling and/or anti-depressant medication. In some patients the depression is a reaction to the PD, but in most, depression is an intrinsic component of PD. Unraveling Parkinson's disease will lead to a better understanding of the affective disorder. At the level of the human brain, as distinct from the human spirit, the formulation, initiation and execution of a thought, is conceptually similar to that of a movement. An idea recognized in the Bible by the sages who described the consequences of a stroke by saying, "If I forgot you, Jerusalem, let my right hand lose (not its strength or power) but its cunning." We are in the midst of a revolution in understanding the brain. The centerpiece of this is Parkinson's disease. With increased financial support from the government, foundations and private donors, the scientific community will unlock the mother-lode of information contained within the Parkinson brain that will banish these diseases and ameliorate the aging process itself. Mr. Chairman, as I am certain you have witnessed, the Parkinson's community has mounted one of the most visible and effective grassroots campaigns in history to secure additional needed funding for Parkinson's research. The culmination of this monumental effort was last November's passage of the Morris K. Udall Parkinson's Research and Education Act of 1997. When the Udall Bill was signed into law, both the President and Vice President specifically recognized its intent as a very essential part of the Labor/HHS appropriations Bill in which it was included as an amendment. Also recognized by the President was the bill's principal sponsor, and your colleague, Congressman Fred Upton who was on hand to witness its signing. It was truly a great moment. I am proud to say that the National Parkinson Foundation regarded the passage of the Udall bill as its highest legislative priority and lobbied aggressively for more than two years in favor of its enactment. Muhammad appeared last April on behalf of the NPF before the House Appropriations Committee to urge the Udall bill's passage and requested that that committee appropriate the funding called for in the Udall Bill after its enactment. That has not yet happened. The Morris K. Udall Parkinson's Research and Education Act is authorizing legislation. It does not provide for the funding that the entire Parkinson's community has worked tirelessly for, and fully expects will hasten the cure for this dreaded disease. Today, the Congress has before it the responsibility to fund the Udall legislation making this victory a full one rather than a hollow one. Some have said that the Udall legislation provides for up to $100 million for Parkinson's research. The National Institutes of Health have recently released numbers attributed to Parkinson's disease research that will soon exceed the $100 million provided for in the Udall legislation. This makes it appear that additional funding for Parkinson's research is not necessary. This conclusion is UNACCEPTABLE! Whatever the National Institutes of Health claims to be spending on Parkinson's disease is based upon an analysis that I am personally not in a position to challenge. However, the entire Parkinson's community believed that the Udall Bill would authorize at least $65 million additional dollars for Parkinson's disease research. This figure is based upon the funds leading Parkinson researchers tell us are needed to dramatically advance our finding a cure or significantly improved treatments for Parkinson's disease. The Parkinson's community has sought, and believes it has found a sympathetic and supportive ear in Congress. Parkinson's activists nationwide have sacrificed some of the best remaining years of their lives advocating a substantially increased federal investment in Parkinson research so that a cure may be found in their lifetime. To say to them now that there is no new money for Parkinson's disease is also unacceptable. If Congress is to honor the intent of the Udall Bill it must appropriate $100 million of additional funds for Parkinson's research for each of the next three fiscal years. The NPF recognizes that federal funding is one of several ways to support medical research. Therefore we have taken unprecedented steps to increase the role of the private sector in finding a cure for Parkinson's disease. The National Parkinson Foundation is the largest voluntary health agency representing the interests of people with Parkinson's disease. It supports 51 NPF Centers of Excellence worldwide. The research conducted by these centers is cutting edge and the grants it awards are subjected to a peer review process similar to that of the National Institutes of Health. It also provides a myriad of patient services including physical therapy, occupational therapy and speech therapy to persons suffering from Parkinson's disease. Furthermore, the NPF works closely with NIH in three ways. First, we provided needed additional funding to the National Center for Human Genone Research when they were looking for the gene that was believed to cause a form of Parkinson's disease. Within a short time, that gene was indeed located as highlighted in the President's State of the Union Address. Secondly, we are cooperating with NIH in providing "bridge" grants to those researchers whose grant applications were of high scientific merit, but fell just below the funding level. And finally, the National Parkinson Foundation has just designated the Parkinson intramural research facility at NIH as a NPF Center of Research Excellence. Thank you, Mr. Chairman and members of this distinguished committee for the opportunity to appear before to talk about Parkinson's disease. Muhammad and I have dedicated our lives to finding a cure for this disease and seek your help. I will be happy to answer any questions that you may have for either Muhammad or me.