Statement Of
John F. Neylan, M.D.
President
American Society of Transplantation
April 15, 1999
Presented to House Appropriations Subcommittee For Labor, Health
and Human Services, Education, and Related Agencies
Mr. Chairman and Members of the
Subcommittee, thank you for the opportunity to present testimony
on behalf of the American Society of Transplantation (AST).
I am John F. Neylan, M.D., Medical
Director of Kidney Transplantation at Emory University and
I am President of the American Society of Transplantation
(AST). The AST, which has no governmental support, was established
in 1982. Our membership, now over 1,400 members strong, is
comprised of physicians, surgeons, and scientists actively
engaged in the research and practice of transplantation medicine
and immunobiology and represents the majority of professionals
in the field of transplantation in the United States.
A principle goal of the AST is
to serve as a forum for the exchange of scientific information
related to transplantation and immunology and to promote
and encourage research. One of the strategies for obtaining
this goal is our annual scientific meeting. Next month, our
18th annual scientific meeting will attract an
international attendance to the city of Chicago and will
feature the cutting edge science that is opening new frontiers
in transplantation medicine and immunobiology.
Today, my testimony will focus
on Fiscal Year 2000 appropriations for the National Institutes
of Health (NIH), with a special emphasis on the transplantation
research portfolios at the National Institute for Allergy
and Infectious Diseases (NIAID), National Institute for Diabetes
and Digestive and Kidney Diseases (NIDDK), and the National
Heart, Lung, and Blood Institute (NHLBI). I will highlight
those areas of research that need to receive additional emphasis
and funding in FY 2000.
During the next hour, four new
names will join those nearly 58,000 individuals in
this country waiting for a solid organ transplant. And by
the time I get home to Atlanta this evening, 10 individuals
will have died because the wait for a transplant was just
too long. It is unfortunate and absolutely unnecessary, but
the sad fact is that we as a nation are not living up to
our potential. Too many families are turning down the option
of organ donation.
But, Mr. Chairman with increased
funding for research there is hope.
Over the last
30 years, transplantation of solid organs has moved from
experimental to accepted therapy,
with approximately 20,000 performed in 1998 in the U.S. alone.
The success of this procedure has improved greatly over the
last few years with almost all solid organ recipients enjoying
an 83 - 97% survival rate at one year. Much of this success
can be attributed to research in immunosuppression that has
been funded by previous federal appropriations. Our better
understanding of the body's response to foreign proteins
has led to countless other breakthroughs in all areas of
medical science. However, this success has brought with it
new challenges.
More and more
individuals are agreeing to be placed on waiting lists for
an organ transplant,
and as a result the list has increased in size by 255 percent
in the last ten years. It is unfortunate and absolutely unnecessary
for those in need of a transplant to go without the "Gift-of-Life," but
this happens all too often because the supply of available
donors is far less that the demand. Each year, the AST identifies
the shortage of available donors as the number one problem
in the field of transplantation.
A year and half
ago, the Administration moved forward with a national organ
and tissue donation initiative
to encourage more families to discuss and understand there
loved ones' wishes in regard to donation. Most recently,
these activities have included a National Donor Day, and
this week, a National Donor Recognition Ceremony to honor
those individuals who have given the ultimate gift.the gift-of-life.
These important initiatives serve to raise national awareness
and
educate the public regarding the
importance and critical need for their participation in the
organ donation process. Such activities are helpful in the
reduction of family refusal, which is the number one cause
of loss of potential donors today. Therefore, the AST urges
this Subcommittee to provide the Department of Transplantation,
located in the Health Resources and Services Administration
with additional funds for FY 2000. This funding will help
to insure the success of these and other programs federally
initiated to enhance donor awareness and improve the public
trust in the process.
Research is central to all that
occurs in the transplantation process. The AST believes that
we are on the threshold of many important scientific breakthroughs
in areas of transplantation research, such as immune mechanisms
of rejection and tolerance as well as the response to animal
organs and tissues, so called xenografts. Because of this,
AST strongly urges this Subcommittee to continue its leadership
in the area of biomedical research and provide a 15 percent
increase in funding for the NIH in FY 2000. The AST supports
this level of increase for the NIAID, the NIDDK, and the
NHLBI, as well. By providing this increased level of funding,
this Subcommittee will achieve the ultimate goal (and the
second step) toward doubling the NIH budget by FY 2003. To
truly translate the promise of scientific discovery into
better health for all Americans -- the President, Congress
and the American people must continue the commitment to significant,
sustained growth in funding for the NIH. As a member of the
Ad Hoc Coalition for Biomedical Research and an organization
dedicated to the exchange of scientific information and the
promotion of research, AST urges the Subcommittee to continue
its strong leadership and commitment to improving the health
of our nation.
With this expanded authority,
clinical and basic transplantation funding at the NIH must
be increased. In particular, we recommend that Congress and
the NIH designate the following as high priority initiatives
at the NIAID, the NIDDK, and the NHLBI.
- The National Institute for
Allergy and Infectious Diseases (NIAID)
- Basic and clinical immunology,
stressing an understanding of immunologic mechanisms of
tolerance and autoimmunity, evaluation of chronic transplant
rejection, and immunosuppression in transplant patients.
- Basic immunology stressing
the response to xenotransplants and methods to overcome
the response.
- Research on identification
and treatment of infectious disease risks associated with
xenotransplantation.
- The National Institute for
Diabetes, Digestive, and Kidney Disease (NIDDK)
Recently, the AST participated
in the first Renal Research Retreat with NIDDK and the American
Society of Nephrology to examine progress in the area of
kidney transplantation and to identify critical research
priorities for the future. AST members are in the process
of editing the transplantation section of the final report.
NIDDK will be publishing this document shortly and we look
forward to sharing the findings of this report with you and
the other members of the Appropriations Subcommittee. Some
of the major priorities identified and discussed at this
forum included:
1) Mechanisms of immune recognition
and response to alloantigens and development of strategies
to induce donor specific tolerance;
2) Identifying and understanding
the processes and measures of graft dysfunction and failure;
senescence, injury and progression;
3) Development of immunologic
(surrogate) markers to predict graft outcomes (rejection,
dysfunction and acceptance);
4) Development of more selective,
more specific immunosuppressive strategies;
5) Optimizing immunosuppression
while minimizing the risk of complications (infections, malignancies
and toxicities);
6) Identification and management
of risk factors for patient morbidity and mortality (cardiovascular
disease and HTN, diabetes, bone disease, liver disease);
7) Expand donor pool and optimal
utility of available organs;
8) Overcoming the biological hurdles
of xenotransplantation (immunological, physiological and
infectious);
9) Improving quality of life:
Patient access to posttransplant care, rehabilitation, identification
and prevention of noncompliance; and
10) Biological alternatives to
whole organ transplantation (tissue engineering and cell
transplantation).
Great progress has been made in
the field of pancreas transplantation and now simultaneous
kidney/pancreas transplant offers greater survival at five
years than kidney alone, and the majority of patients no
longer need insulin. However, most insurance companies do
not cover this combined transplant because definitive prospective
studies have not been conducted. The NIDDK needs to sponsor
a trial to document the superior benefit of combined kidney/pancreas
transplant to convince third party payers and Medicare to
cover this procedure, and further fund research to assess
the comparative benefit of whole organ (pancreas) versus
islet cell transplantation.
- National Heart, Lung, and Blood
Institute (NHLBI)
- Clinical trials in immunosuppression
in heart transplantation.
- Research on chronic rejection,
applying promising new technology such as intravascular
ultrasound.
One new clinical advance is the
development of an intravascular ultrasound imaging technique
that allows measurement and detection of thickening of the
vessel wall of the transplanted organ and follows these changes
over time. This is currently being performed in heart transplant
recipients, but the test is not reimbursed. This is the
perfect opportunity to have NHLBI
partner with industry to conduct a trial of new therapies
to retard or prevent the development of this disease.
- Establish the creation of databases
for each stage heart disease to understand the disease
and plan future needs.
Heart failure is the number one
admitting volume diagnosis in the Medicare system, the longest
DRG, and it has the highest readmission rate. There are an
estimated one million people with this diagnosis which has
as much or more of an impact on the health care budget as
any other disease. In addition, the increasing average age
of the U.S. population indicates that this disease will only
increase.
The fruits of current transplant
research have engendered the important successes accomplished
in the field while offering continued and new challenges.
Ever more precise and powerful transplant immunosuppressive
drugs have greatly increased both patient and graft survivals.
However, despite our successes, almost half of renal grafts
are eventually lost to premature patient death with a working
transplant creating the challenge to understand the morbidity
in pre-existing and concomitant illnesses such as cardiovascular
disease, hypertension, infections, bone disease, diabetes,
and malignancies. Strategies to induce donor specific tolerance
in animal models hold promise. Strategies to hurdle xenogeneic
barriers using molecular genetic tools have begun. Expansion
of these efforts taken together with experiments characterized
above may bring the dream of successful replacement of a
failed organ for the natural lifetime of a patient to fruition.
Conclusion
We talk quite
a bit in the transplantation community about how receiving
a transplant can be the "Gift
of Life." You can't put a price tag in human terms of
such a gift. Yes, a transplant procedure and follow-up care
is expensive. But, relative to the lost productivity, the
impact on quality of life, and the cost of living with end
stage heart or renal disease, transplantation is very cost
effective. Also, it may be the only hope not just for improved
survival, but for a full and healthy life for many individuals
and their families. So, I end my remarks here today, by repeating
AST's request that this Subcommittee and Congress approve
a 15 percent increase for the NIH in FY 2000 - and thus allow
the high priorities and initiatives that I have discussed
to be funded and commence.
Thank you.
