With two daughters dead from a blood disease and a third diagnosed, a father campaigns for gene therapy

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With two daughters dead from a blood disease and a third diagnosed, a father campaigns for gene therapy

Wednesday, October 6, 1999

By CAROL SMITH
SEATTLE POST-INTELLIGENCER REPORTER

Dave Frohnmayer has a more compelling reason than most to be watching the new gene therapy trial under way in Seattle.

Frohnmayer, president of the University of Oregon, has already lost two daughters to a rare genetic disease called Fanconi anemia that causes the bone marrow to fail. A third daughter, Amy, now 12, has the disease as well.

If the gene therapy trial against the blood disease leukocyte adherence deficiency proves successful, scientists may move quickly to test the same techniques against Fanconi anemia.

If so, the Frohnmayers hope Amy will be spared the fate of sisters Kirsten, who died at age 24 in 1997, and Katie, who died at age 12 in 1991.

Amy Frohnmayer gives her father David a picture for his new office at the University of Oregon. 1994 file photo. Chris Pietsch, The Register-Guard

Amy's blood counts are stable now, Frohnmayer said. But they're low and could dive to dangerous levels at any time, especially as she enters puberty when the body is in a state of rapid change.

"We watch her counts all the time," he said. "Our backs are against the wall."

Frohnmayer, a former Oregon attorney general who is on the board of directors at the Fred Hutchinson Cancer Research Center, has been raising money and awareness for gene therapy since Kirsten, his oldest daughter, was diagnosed in 1983.

The diagnosis was a shock. Kirsten was 10 years old when she suddenly began showing signs of unexplained fatigue, weakness and bruising. When tests revealed she had Fanconi anemia, the Frohnmayers also got the hard news that Katie, then 4, had the genetic trait as well.

The Frohnmayers have two sons without the disease, which strikes boys and girls equally.

In 1989, Frohnmayer and his wife, Lynn, started the Fanconi Anemia Research Fund, which has since raised more than $6 million for research into the genetic causes of the disease and possible gene-therapy treatments.

A decade ago, there were only about 18 researchers around the world working on Fanconi anemia, which affects about 1,500 patients in the United States. The genes that caused the disease were unknown.

Today, there are more than 100 scientists working on the disease and scientists have cloned three genes that account for about 90 percent of the cases.

"The fact that the genes have been cloned and isolated, makes it realistic that an experimental gene therapy is the next step," Frohnmayer said.

Groups at the University of Indiana, Harvard University, University of North Carolina in Chapel Hill and the Fred Hutchinson Cancer Research Center are now poised to begin clinical trials of various gene therapy protocols for Fanconi anemia. Each uses a slightly different approach.

"None of the trials are open for families yet," Frohnmayer said. "We're watching very carefully. We're hopeful it (a treatment) will be there in time for Amy."

The only other alternative for Amy is a bone marrow transplant.

But patients with Fanconi anemia are hypersensitive to radiation and chemotherapy, which makes bone marrow treatments extremely risky.

Fanconi anemia is a defect in the body's natural DNA repair mechanism. The genes associated with the disease are part of a suite of "caretaker" genes that protect the body against cancer. The failure of one of these genes causes bone marrow cells to die, but it also predisposes patients to cancer because the body can't repair cancerous mutations.

Better understanding and treatment for the disease that has decimated Frohnmayer's family could help researchers understand how to treat some forms of cancer.

"Over time, we're convinced this (gene therapy) will be the molecular medicine wave of the future," he said.

Until that happens, he'll keep fighting in memory of his two daughters and for the future of his third.

"We have to fight back," he said. "It's a horrible price we've already paid. You have to believe something more lasting and positive will come of it."

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