[Congressional Record Volume 145, Number 73 (Wednesday, May 19, 1999)]
[Extensions of Remarks]
[Pages E1017-E1018]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]




                         A WORRIED GRANDFATHER

                                 ______
                                 

                            HON. FRED UPTON

                              of michigan

                    in the house of representatives

                        Wednesday, May 19, 1999

  Mr. UPTON. Mr. Speaker, I recently had the pleasure of introducing 
one of my constituents, Dr. Fred Mathews, at a hearing of the 
Subcommittee on Labor, Health and Human Services, and Education 
Appropriations. Dr. Mathews had been invited to speak on behalf of the 
Neurofibromatosis Foundation in support of increased funding for this 
often devastating disease.
  It is a privilege to know Dr. Mathews and count him as a friend. In 
addition to his 47 years of practicing optometry in Dowagiac, MI, he 
has devoted his talents and energy to improving the quality of life in 
his community and expanding education opportunities and excellence in 
our state. When he learned that his lovely young granddaughter, 
Allison, was afflicted with neruofibromatosis, he took on the most 
important fight of his life--the fight for a cure for this disease for 
Allison and for the at least 100,000 others who have this neurological 
disorder. His testimony before the subcommittee was eloquent, and I 
would like today to submit it to the Congressional Record so that 
others may see the urgency of the need to find a cure. Dr. Mathews' 
testimony follows:

                         A Worried Grandfather

       Thank you Congressman Upton and thank you Mr. Chairman and 
     members of the Committee for allowing me to testify. I am 
     Fred Mathews, a constituent of Congressman Upton from 
     Southwestern Michigan.
       I am here today because my beautiful granddaughter Allison 
     has Neurofibromatosis, a not so rare and devastating genetic 
     disorder. In 1994 Allison was four years old when I first 
     asked her parents about some spots on her skin. I had assumed 
     these were simple birthmarks. This was the first time her 
     parents shared with me that she had Neurofibromatosis, or 
     abbreviated called NF. Up until then we had been shielded 
     from the terrible truth.
       I am an optometrist in a small town in southwest Michigan. 
     I have practiced there for 47 years. Even though I am not a 
     medical doctor I have better than a layman's knowledge of 
     general medical problems. However, I had never heard of NF.
       Immediately I began to research NF. I called research 
     centers. I called the National Institutes of Health. I linked 
     up with the National Neurofibromatosis Foundation. My 
     testimony today has the blessing of that fine organization.
       There is no way to describe the despair and hopelessness 
     that families experience when faced with the fact that a 
     child or grandchild has an incurable disease. My research 
     left my wife and me panic-stricken. Here is a short version 
     of what my research revealed.
       NF is the most common neurological disorder caused by a 
     single gene. At least 100,000 Americans have NF. This makes 
     NF more prevalent than Cystic Fibrosis, hereditary muscular 
     dystrophy, Huntington's Disease and Tay Sachs combined.
       NF causes tumors to grow anywhere on or in the body. NF can 
     lead to disfigurement, blindness, deafness, skeletal 
     abnormalities, dermal, brain and spinal tumors, loss of 
     limbs, malignancies and learning disabilities. The terrible 
     disfigurement is why NF has erroneously been confused with 
     the so called ``elephant man'' disease.
       NF affects both genders, all races and ethnic groups 
     equally. NF research in 1994 (when I first learned of my 
     granddaughter's problem) had begun about 9 years earlier by 
     the National NF Foundation. The gene causing NF had just been 
     discovered.
       My personal research did reveal some good news for my 
     family and me. My granddaughter has the NF1 gene rather than 
     the NF2 gene. With the NF2 gene the tumors and other bizarre 
     disorders can start soon after birth. NF1 however, which my 
     granddaughter has, sometimes does not manifest serious 
     problems until puberty or beyond.
       I also learned from Peter Bellermann, President of the 
     National NF Foundation, and the world's greatest crusader to 
     find a cure for NF, that researchers were hopeful of finding 
     a cure in 10-15 years. Simple mathematics told me that this 
     might be too late for my granddaughter and thousands of kids 
     like her who were living with this time bomb.
       I also learned that researchers believed that the projected 
     time for a cure could possibly be cut in half if more 
     research dollars were available.
       I am grateful that this Committee and the Congress did 
     respond to our plea and did appropriate significant new funds 
     for NF research. In 1995 Chairman Porter also added language 
     to the Appropriations Bill which expressed to NIH the 
     commitment of this Committee for accelerated NF research.
       Because of this Committee, the Congress, the NIH, the 
     National NF Foundation and many dedicated researchers, our 
     Allison who is now 9 years old, has a chance to avoid the 
     ravages of NF. We are thankful and hopeful but still very 
     apprehensive. The time clock is still running rapidly. 
     Research has been extremely successful but has a long way to 
     go to find a cure.
       The National NF Foundation and I urge that the language 
     which has been in the Appropriations Bill for the past four 
     years, expressing this Committee's commitment to NF research, 
     be in the FY 2000 bill.
       I am grateful for the courtesy members of this committee 
     and other members of congress and their staffs have shown 
     Peter Bellermann and me these past few years. Some of you 
     have my granddaughter's picture in your office.
       In my opinion, no expenditure by the Federal Government is 
     more rewarding, more needed, and more appropriate than 
     research for dread diseases including NF. As a grandfather of 
     a little girl with one of these dread diseases, I feel 
     anxiety, frustration but also hope knowing that the timetable 
     for a cure of NF and other diseases is almost solely 
     dependent on the willingness of the Congress to recognize 
     medical research as its #1 priority. That is why Mr. Chairman 
     we strongly support a significant increase in funding for the 
     National Institutes of Health medical research. With the NIH 
     as the quarterback, the greatest hope we have for finding a 
     cure for NF and all other dread diseases, lies with this 
     Committee and the NIH.
       Since my allotted time is up Mr. Chairman, I respectfully 
     request permission to extend my remarks in the written 
     testimony I will leave with the Committee.
       Mr. Chairman and Members of the Committee, on behalf of the 
     National Neurofibromatosis Foundation, as well as the 
     thousands of children and adults with NF, I thank you and my 
     Allison thanks you.
                                  ____


               Extended Testimony of Dr. Fred L. Mathews


                             April 29, 1999

       I am pleased and proud that NF research has been pointed 
     out to be a model for ``Managing Science.'' It represents an 
     effective partnership between public agencies, most notably 
     the U.S. Congress and the National Institutes of Health, 
     private organizations and the National Neurofibromatosis 
     Foundation and scientists and clinical researchers in the 
     field who have achieved their progress by consensus and by 
     collaborating to a remarkable degree. To use the vernacular, 
     NF research has given a ``good bang for the buck'' to all who 
     have invested in it.
       NF research has significant potential for other very large 
     patient populations. Since the NF genes have been implicated 
     in the signaling process that determines cell growth and cell 
     differentiation, NF research also has great promise for the 
     tens of millions of Americans with malignancies.
       NF also causes learning disabilities at about five to six 
     times the frequency found in the general population. Work in 
     that aspect of NF research therefore has considerable 
     potential for the estimated 30 million Americans who are 
     learning disabled.
       Given the wide variety of symptoms of NF, I understand that 
     you Mr. Chairman and the

[[Page E1018]]

     Committee have urged those involved in NF research to foster 
     collaborative efforts between and among the various 
     initiatives at the NIH under whose purview these 
     manifestations fall. Peter Bellermann, President of the 
     National NF Foundation has informed me that these efforts are 
     taking place and that ``Cross-Institute'' activities are a 
     reality.
       NF has the attention at the highest level of the NIH 
     beginning with the Director Dr. Varmus. It extends to the 
     Institute heads, especially Dr. Gerald Fischbach at NINDS and 
     to Dr. Richard Klausner at the National Cancer Institute. 
     These progressive officers work at continuing the cross-
     institute efforts, participate in scientific meetings of NF, 
     and advise other funding agencies to avoid duplication of 
     funding.
       NF has been a success story for research for all who have 
     invested in it. True success will, however, come only when a 
     cure is found and real people like my granddaughter can look 
     forward to happy lives, free of NF's terrible consequences. 
     We now have to go the next hard miles. Researchers now stand 
     ready to translate basic scientific knowledge into clinical 
     application. The next agenda includes continued work in basic 
     research, starting comprehensive natural history studies for 
     NF and beginning the all important process of clinical trials 
     with innovative approaches. We all pray that this will lead 
     to an effective treatment for NF.
       In closing, I would be remiss if I did not thank my 
     Congressman Fred Upton and his staff person Jane Williams for 
     their very special help and support. We are also very 
     appreciative of the longtime support Congressman Murtha has 
     given NF funding. And a special thanks to you, Chairman 
     Porter, who in 1995 took time from your busy schedule to meet 
     personally with Peter Bellermann and me so that we could tell 
     you of the urgent need for accelerated NF funding. Your 
     ongoing support since then has been tremendously helpful. To 
     the members of this Committee who have supported us in this 
     critical effort, we also offer another thank you.

     

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