Melanie Speaks to Congress as NCF Plans Federal Co-op

Speaking clearly in the hushed silence of a high-ceilinged Congressional hearing room, Downingtown teenager Melanie Stewart finally had her say on May 14, asking members of Congress to fund research into the kidney disease that has taken her two native kidneys and one transplanted from her father.

"There are thousands of people, mostly young, like me who would like a chance for a cure and a normal life," said Melanie, 15, who maintains a busy school and social life despite her battle with focal segmental glomerulosclerosis (FSGS).

"Over the last 8 years, I've spent most of my time in the hospital or hooked up to a dialysis machine while trying to keep up my schoolwork," she told the members of the U.S. House Appropriations Subcommittee on Labor, Health & Human Services, Education and Related Agencies.

Last year, Melanie could not appear before the committee because she was losing the kidney donated by her father, Brad, a NephCure Foundation (NCF) founding board member who spoke to the committee instead.

This year, Melanie and NCF President Lou Antosh appeared before the committee urging the panel to increase federal funding to the National Institutes of Health and to support, with more funds, a program of clinical trials into FSGS treatments planned to start this Fall.

Antosh, who sat at the hearing table with Melanie and his daughter, Christine, 18, said the disease was scarring Christine's kidneys and "she is one of thousands of young people in a race against time." He told the Members of Congress: "These young people look to you today for hope."

The planned clinical trials hold no hope for Melanie or Christine, who never responded to the medications expected to be used in the trials, said Antosh. But the federally-funded trials will collect body tissues and samples that scientists can use to research the molecular mechanism that causes FSGS, he said, asking that NIDDK fund such basic science.

Melanie's testimony ended the NCF allotted time before the committee in the Rayburn House Office Building hearing room, where movie star Julia Roberts testified a few days earlier. NCF legislative consultant Dale Dirks whispered to Congressional staffers: "We believe Julia Roberts was a great opening act for Melanie."

"Three years ago, FSGS destroyed both of my kidneys," the petite youngster explained to the committee. "On April 21, 1999, my dad gave me one of his kidneys. The year after the transplant was one of the hardest.

"Over that time, I had apheresis procedures done three times a week. As a result of the high doses of immune suppressant drugs, PTLD, a form of cancer, was found on my head. In November of 2000, I almost died because of a blood infection and a blood clot in my heart caused by the apheresis catheter.

"In March of last year, the day I was to testify before you, I had my donated kidney removed. I am now on dialysis again and am forced to start over again."

She looked up at subcommittee chairman U.S. Ralph Regula (R-OH) and concluded: "For everyone, I'm asking for your help to help me meet my goal of finding a cure."

The NCF testimony also disclosed that NCF is in discussions with the National Institute Of Diabetes and Digestive And Kidney Diseases (NIDDK) regarding a program in which NCF will provide matching funds for use in basic research related to the FSGS clinical trials.

Antosh also asked that the NIDDK give high priority to Nephrotic Syndrome and FSGS as the agency plans a new National Kidney Disease Education Project.

"Our cause includes something called Nephrotic Syndrome, which causes legs to swell and faces to distort," said Antosh.

"Some fortunate patients are treated with steroids and the syndrome disappears. But steroids and other drugs have been no help to Melanie or my daughter, who have progressed to a disease called FSGS.

"Our only hope at this point is you," he said.

Rep. Regula asked if both Melanie and Christine were on dialysis and was told that Melanie undergoes nightly dialysis, but Christine still has her native kidneys. However, a biopsy last summer showed the disease has caused scarring to her kidneys. This prompted an explanation by Antosh into the quest of scientists to determine how and why FSGS causes scarring of the nephron, the kidney filtering mechanism.

Chairman Regula expressed his support for the NCF cause and the efforts of NIH to undertake FSGS research.

In its testimony, NCF also asked that the National Center for Minority Health and Health Disparities initiate studies into the "troubling phenomenon" of FSGS incidence among African-Americans, who suffer the disease at disproportionate rates and have the worst prognosis.

Brad and Melanie Stewart and Chrissy and her parents (Lou and Joanne) spent the morning prior to the hearing in meetings at the offices of U.S. Sen. Arlen Specter (R-PA), U.S. Rep. Jim Saxton (R-NJ) and U.S. Rep. Rodney P. Frelinghuysen (R-NJ).

Brad and Melanie ended the day meeting with U.S. Rep. Joseph Pitts (R-PA), who posed with the pair for photos requested by a Pennsylvania newspaper which is preparing an article on Melanie.