CBS News first interviewed the Frohnmayer family 12 years ago. Now CBS News Sunday Morning Correspondent Jerry Bowen reports that theres even a chance for a happy ending.
In many ways, last months gathering at a camp on Wisconsins Lake Geneva seemed like nothing out of the ordinary. Except it was.
The children in attendance have a rare genetic disorder called Fanconi anemia The body gradually stops producing oxygen-giving red blood cells and infection-fighting white blood cells and the platelets that control bleeding.
"We started this journey down the road of chronic illness around 15 years ago," says one father.
But Fanconi anemia is not only a blood disease. There is also a chromosome weakness that makes victims particularly susceptible to cancer.
While the average life expectancy for those with disease is 22 years, bone marrow transplants have helped improve the survival rate. Thats what 26-year-old Dani Sacks had, and she has the visible birth defects of some Fanconi anemia victims.
"I have the typical abnormalities of the hands," she says. "I have the four fingers. I have missing radial bones in both hands. Small head, small eyes. Short stature. But it doesnt bother me at all."
The guiding force at the Fanconi camp is the Frohnmeyer family.
Dave Frohnmayer, formerly Oregons attorney general, abandoned a run for governor to devote time to finding a way to save his two daughters, Kirsten and Katie. Both were found to have Fanconi anemia.
"You spend all that time tearing yourself up. But pretty soon you have to make a basic decision, or at least we decided that. You either become a victim yourself, or you fight back with everything at your disposal," Dave Frohnmayer says.
"Theres so much you can feel guilt about when you have a disorder like this," Lynn Frohnmayer explains. "Its a genetic disease. Weve given a genetic disease to our precious children. That doesnt feel very good."
Twelve years ago, the only known treatment was a bone marrow transplant. But there was no match in the family: not with the parents, brothers or even baby Amy.
So the Frohnmayers were forced to search the extreme branches of the family tree, 4,000 miles away in their ancestral homeland, the Canadian province of Nova Scotia. Dave Frohnmeyer went on the radio to appeal to strangers, who were, in fact, distant relatives.
The sisters, Kirsten and Katie Frohnmayer, then 14 and 8, held a news conference asking people to take a blood test to determine if they might be the perfect match.
"I would very much like to possibly find a suitable bone marrow match for myself or my sister," Kirsten said in an appeal.
More than 1,600 distant relatives were eventuall tracked down, but no match was found for the girls; there was no miracle in Nova Scotia.
"I dont really pity myself and I dont know why other people should," said Katie.
So the Frohnmayers returned home to Eugene, Ore., where Dave Frohnmayer is now president of the university. And they kept searching for a solution. But time ran out, and Katie died in 1991 at the age of 12.
Kirsten lived to go on to college and graduated from Stanford University. She died in 1997 at the age of 24. But that wasnt the end of it for the Frohnmayers.
Amy, who's now 12, has Fanconi anemia, too.
"I think its a little harder for me to deal with it than other FA patients, because Ive lost two of my sisters in this disease," says Amy. "Its making me scared about whats gonna happen to me."
The Frohnmayers' mission never stopped. They established a research foundation and created a Fanconi handbook sent to physicians across the country. With other parents, they've raised millions of dollars to fund scores of laboratory studies into the causes and cures of this rare disease.
And now, each summer at the camp the Frohnmayers started, doctors who review the very latest research join Fanconi families.
"Science has really advanced in a way that would have been thought to be almost science fiction at the time we were searching desperately in Nova Scotia for people who might be donors for bone marrow," says Dave Frohnmayer.
But this year there was encouraging news.
Advances in bone marrow transplantation made available a new drug enabling a transplant from an unrelated, only partially matched donor.
No longer science fiction, gene therapy would fix the bad Fanconi genes. A corrected gene is actually inserted into a damaged bone marrow cell so it starts making healthy blood.
But will it work in Lynn Mendenhall? At 45, she is among the oldest Fanconi patients alive, suffering now from mouth cancer, a consequence of the disease.
Mendenhall has been approved as the first patient when Dr. Chris Walsh of the University of North Carolina Gene Therapy Center starts his gene therapy clinical trials.
Scientists are also hopeful that studying the Fanconi genetic defect will help unlock some of the mysteries of cancer. Thats because abnormal Fanconi anemia cells behave like cancer cells.
"If we can pinpoint the gene and the mutation, which is abnormal in Fanconi patients, we will be able to extract and use it to understand something about lung and breast cancer in the general population," says a doctor at Boston's Dana-Farber Cancer Institute.
But that's still in the future. Meanwhile, back at Lake Geneva, the kids are dealing with things most kids don't have to.
"I talk to my parents a lot of the time," Amy continues. "They're really helpful. We just talk about a lot of hope."
"Doctors are finding hings every single day. They're learning new things about gene therapy and bone marrow transplants. Everythings looking really hopeful for the future," she says.
But is gene therapy Amy's best hope?
"I think were at the beginning of this process and I dont know when were going to feel that really is the answer to the problem," Lynn Frohnmayer says. "Ultimately it will be. But how long will that be and will it be in time for Amy? I dont know."