[Extensions of Remarks]
[Page E586]
From the Congressional Record Online through the 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.
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

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.