[Congressional Record (Bound Edition), Volume 147 (2001), Part 4]
[Extensions of Remarks]
[Pages 5833-5834]
[From the U.S. Government Publishing Office, www.gpo.gov]



                    TESTIMONY OF DR. IRVING SMOKLER

                                 ______
                                 

                          HON. LYNN N. RIVERS

                              of michigan

                    in the house of representatives

                        Wednesday, April 4, 2001

  Ms. RIVERS. Mr. Speaker, I would like to share with my colleagues, 
the testimony of Dr. Irving Smokler, presented to the House 
Appropriations Subcommittee on Labor, Health, and Human Services, 
Education and Related Agencies. Dr. Smokler is the president of the 
NephCure Foundation and testified regarding the need for increased 
funding for research and raising professional and public awareness on 
glomerular injury through the National Institute of Diabetes and 
Digestive and Kidney Diseases.

Testimony Regarding Fiscal Year 2002 Funding for National Institute of 
               Diabetes and Digestive and Kidney Diseases

     Presented by Irving Smokler, Ph.D., President of the NephCure 
  Foundation, Accompanied by Brad Stewart to the House Appropriations 
Subcommittee on Labor, Health and Human Services, Education and Related 
                   Agencies--March 20, 2001--10:00 AM


                   summary of fy 2002 recommendations

       1. Continue the effort to double funding for the National 
     Institutes of Health by providing an increase of 16.5%, to 
     $23.7 billion for FY02. Increase funding for the National 
     Institute of Diabetes and Digestive and Kidney Diseases 
     (NIDDK) by 16.5% to $1,518,443,525 for FY02.

[[Page 5834]]


       2. Prioritize glomerular injury research at NIDDK 
     (including clinical trials), raise professional and public 
     awareness about glomerular injury, and encourage more 
     aggressive scientific attention to all kidney diseases.
       3. Urge NIDDK to develop programs to attract talented 
     researchers to the field of glomerular injury.
       Mr. Chairman, and members of the subcommittee, I am pleased 
     to present testimony on behalf of the NephCure Foundation 
     (NCF).
       We are a relatively new, non-profit organization with a 
     mission of supporting research and public awareness on 
     glomerular injury, which is related to the filtering 
     mechanism of the kidney. I serve as president of the 
     foundation, and have a son, who has had a glomerular disease 
     since he was eleven months old. Although he is now 24 years 
     old and in remission, eighty percent of those in his 
     situation lose their kidneys or their life by the age of 
     five.

                       What is glomerular injury?

       Mr. Chairman, each kidney contains about one million tiny 
     filtering units called nephrons. Nephrons are the key to the 
     kidney's filtering function, processing a constant flow of 
     waste-laden blood, sorting out the vital fluids, from the 
     toxic and unnecessary elements.
       When someone suffers from a glomerular disease, this vital 
     process is impaired. In some instances, an individual will 
     lose protein and sometimes red blood cells in the urine, have 
     high cholesterol levels, and experience severe swelling in 
     the body from too much fluid. Incidence of this disruptive 
     Nephrotic Syndrome is increasing, and this perplexes 
     physicians who cannot identify the cause or cure.
       Sometimes damage occurs to the nephrons, specifically, 
     scarring of the glomeruli, which are microscopic capillaries 
     in the nephron. The severe form of this glomerular injury is 
     Focal Segmental Glomerularasclerosis (FSGS). Presently, there 
     is no treatment to reverse this damage. FSGS can lead to end 
     stage renal disease--total, or near total, permanent kidney 
     failure. Costly dialysis treatments become necessary and 
     kidney transplants may be required for severe cases.

                     The toll of glomerular injury

       Glomerular injury affects tens of thousands of patients in 
     the nation, most of them young. While it is unclear exactly 
     how many Americans are impacted, the incidence of glomerular 
     injury is on the rise. Severe forms of glomerular injury are 
     costly to diagnose and treat, and at this time the only 
     relief for these patients is with heavy medication, usually 
     steroids, which have strong and unpleasant side effects and 
     only work for about 30 percent of patients.
       Problems of misdiagnosis often occur with glomerular 
     injury. Most patients and parents have stories about the 
     unusual length of time between the first symptoms and 
     diagnosis. The early signs of glomerular injury, swollen 
     eyelids, are often mistaken for allergic reactions. Health 
     care professionals don't appear to be fully knowledgeable 
     about this disease.
       The physical changes, extreme swelling of the face and 
     body, can adversely affect all aspects of a young person's 
     life. With a stronger commitment to research and educational 
     awareness, suffering can be minimized and hopefully 
     eliminated.

               There is hope for scientific breakthroughs

       At a meeting co-sponsored by the NephCure Foundation, 
     preeminent scientists from around the world have shared their 
     findings about the podocyte, a major filtering cell, with 
     tentacle-like feet. The relationship between the podocyte and 
     the glomerulus may be a key to understanding glomerular 
     injury.
       Recently, researchers have discovered certain molecules 
     that are essential to the podocyte's function. As this 
     becomes better understood, scientists are hopeful of finding 
     better ways to treat glomeular diseases, and prevent their 
     progression to more grave conditions.
       This spring, NIDDK will begin to establish clinical trials, 
     which will test various treatments for hundreds of FSGS 
     patients. But there is a need for more funds to strengthen 
     the basic science behind these studies. Researchers need to 
     study tissue and fluids from those patients to advance their 
     knowledge of the molecular causes of FSGS.

                         What needs to be done?

       Respectfully, Mr. Chairman, the NephCure Foundation urges 
     this subcommittee to:
       1. Continue the support for doubling the National 
     Institutes of Health (NIH) and the National Institute of 
     Diabetes and Digestive and Kidney Diseases (NIDDK).
       2. Provide the funding and recommendations for the National 
     Institute of Diabetes and Digestive and Kidney Diseases to 
     aggressively pursue a scientific program which will advance 
     research into glomerular injury, conduct clinical trials, 
     raise public awareness, and recruit talented scientists to 
     this field of research.
       Thank you for the opportunity to appear before you today.
       Mr. Chairman, we hoped to have Melanie Stewart here to 
     testify today, but her health would not allow her to be here. 
     Her father, Brad Stewart, will read Melanie's statement.
       My name is Melanie Stewart. I'm 13 years old and have had 
     FSGS since I was six. Until a year ago I spent most of my 
     life in the hospital or hooked up to a dialysis machine for 8 
     hours every day. My kidneys finally died last year, so my dad 
     gave me one of his. I've done my best to keep it by taking 20 
     pills a day, fighting off infections, hemorrhages, and a 
     blood clot in my heart. The kidney my Dad gave me is failing.
       There are thousands of kids just like me who would like a 
     change at a normal life. For all of us, I'm asking for your 
     help in finding a cure for this disease.
       Thank you for listening.