STATEMENT BY
TERRY R. PEEL
PRIVATE CITIZEN, BETHESDA, MARYLAND

BEFORE
SUBCOMMITTEE ON LABOR, HEALTH AND HUMAN SERVICES,
EDUCATION AND RELATED AGENCIES
COMMITTEE ON APPROPRIATIONS
U.S. HOUSE OF REPRESENTATIVES

CONCERNING
THE NEED TO ADDRESS THE DISEASE AMYLOIDOSIS

April 27, 2004 

 Mr. Chairman, I appreciate the opportunity to testify before the Committee on the issue of amyloidosis.  The disease, which is often fatal, leads to heart, kidney, liver and other organ failure.  Thousands die never knowing they even had the disease.  Thousands of others die because they found out too late to obtain effective treatment.  The small portion of those with amyloidosis who are lucky enough to obtain treatment face the risk of high dose chemotherapy and stem cell replacement.

 A year ago, like many Members on this Committee, I had never heard of amyloidosis. But then my wife Ann who is appearing with me today was diagnosed with Primary Amyloidosis.  It is a disease that is often misdiagnosed or diagnosed too late.  Less than one percent of the U. S. population has this disease (although that is probably vastly under diagnosed, especially in the African-American community).  Left untreated the disease has an average survival rate of about 15 months.  It literally can kill people before they know what has hit them.

The Disease and Treatment

  What is amyloidosis? It is a disease characterized by abnormally folded protein deposits in tissues. In the simplest of terms, cells malfunction and produce protein that settle on organs, such as the heart, kidney and liver.  These folded proteins clog the organs until they no longer are able to function—sometimes at a very rapid pace. In addition to primary amyloidosis, there are also cases of inherited or familial amyloidosis and secondary or reactive amyloidosis.

 There is no explanation for how or why amyloidosis develops.  There is no known cure.

 What can be done about it?  Until ten years ago there was little that could be done to effectively stop amyloidosis—only two percent of patients responded to low doses of oral chemotherapy treatment.  The average survival rate at that time was about a year.

 Although it is not a cure, new treatments have been found to be more effective.  Boston Medical Center and other Amyloid centers have found that large dose intravenous chemotherapy followed by stem cell replacement or rescue is an effective treatment in many patients.   Bone marrow is eliminated through high dose chemotherapy and the patient’s own extracted stem cells are replaced in order to improve the recovery process.

  The new procedure has increased the remittance and long term survival rate dramatically:  increasing from two percent to almost 50 percent of the number of patients with total remission.  Although this is certainly not an acceptable rate, it does give hope. 

 But this procedure is a treatment, not a cure.  Although the chemotherapy and stem cell rescue system has greatly improved the chance for survival, research needs to be done to find ways to either prevent the disease from occurring or to treat it through more effective and less risky methods.  A majority of the people with amyloidosis are not in good enough physical condition to undergo the rigors of the chemotherapy stem cell rescue process, and those who are qualified risk infection or other complications.

Diagnosis and Research

 Timely diagnosis is also of great concern to us.  Fortunately, Ann was diagnosed at an early stage by an alert doctor here in Washington. She was able to successfully undergo stem cell replacement last fall and will undergo a more limited treatment this July.  However, she is the exception.   Many people are diagnosed after the point that they are not able to undertake treatment.  Early treatment is the key to success. More needs to be done in this area to alert health professionals to identify this disease.

 Although there is some research being done on amyloidosis, there is not enough funding to do more.  We are fortunate to have at the hearing today, Dr. Martha Skinner, the Director of the Amyloid Treatment and Research Program at Boston University School of Medicine. Dr. Skinner has been the guiding light in amyloid research for more than 20 years.  This center was founded in 1960 and has been one of the primary research and treatment centers in the world. 

 We would like to thank Dr. Skinner for her work on behalf of the people who are struggling to overcome amyloidosis. She is finding ways to save thousands of lives every year.  

 The objective of the Boston research is to examine all aspects of amyloid disease and to develop new treatment approaches and ultimately a cure.  The investigators in the research program are from multiple disciplines and include biochemists, biophysicists, molecular biologists, pathologists, and immunologists. 

Steps To Be Taken

 Mr. Chairman, we ask that the Committee do three things to help address this deadly disease.  First, provide language in your report identifying amyloidosis as an important concern and encouraging more research to find a cure.  Second, encourage the Centers for Disease Control and the National Institutes for Health to educate the American public and medical profession on the need to diagnose this disease at an early stage.  Finally, provide fiscal year 2005 funds within the Health Resources and Services account for equipment and related research at the Boston University Medical School to help find improved methods for treating and curing this tragic disease.

 Mr. Chairman, I like to look to one of my favorite heroes, Helen Keller, for wisdom on how to be triumphant over adversity.  In 1921 she sent a letter to a friend stating, “The true test of character is to face hard conditions with the determination to make them better.”  This Subcommittee has met that test many times over the last quarter of a century—saving the lives of millions of Americans from devastating diseases.  I hope you will help my hero, Ann, and millions of others who suffer from this tragic disease. 

Thank you for your consideration.