Hope’s High Cost

IRVINE —  Marianne Whitmyer first noticed the changes in her fingers and her tongue. They looked the same but no longer worked the same, her fine motor control dissolving after two decades as a professional flutist.

The problem, doctors discovered early last year, is that Whitmyer’s brain is dying.

Whitmyer, principal flute and soloist with the Irvine Symphony, suffers from Huntington’s chorea, or Huntington’s disease, a progressive hereditary illness that leads to death. Researchers have found no cure, but recent advances have emboldened doctors to try experimental treatments aimed at stopping the brain-cell death that lies at the heart of the disease, whose most famous victim was folk singer Woody Guthrie.

Whitmyer, the divorced mother of a 12-year-old daughter, hopes to raise $48,000 for one such experiment at Good Samaritan Hospital--an operation she believes could reverse her deterioration and possibly save her life.

“I want to restore myself to my former self,” said Whitmyer, 45, whose insurance will not cover the experimental procedure.

How much the treatment, in which fetal tissue is implanted in the brain, will help Whitmyer remains uncertain. While many of the 22 patients who have undergone the procedure over the past year have improved radically and rapidly, even one of the doctors involved is uncertain about the results.

“I don’t know how long this improvement will last,” said Dr. Deane B. “Skip” Jacques of Good Samaritan Hospital in Los Angeles. “I’m a little skeptical myself. These [patients] have done fairly well, and I don’t know why. I don’t like things I can’t explain. But I think [the treatment] is worthwhile.”

The Good Samaritan program is one of at least three fetal-implant experiments underway around the world.

“We’ve talked to people who’ve had the procedures done at Good Samaritan Hospital who’ve been pleased with the results,” said Ellen Robinson of Santa Monica’s Hereditary Disease Foundation, which tracks research into Huntington’s disease. “But we’ve talked to others who’ve noticed no change. It’s a procedure that we neither endorse nor do we condemn.”

Jacques understands Robinson’s tepid response.

“I think they’re just being very cautious about it,” Jacques said. “You have to be that way from a scientific point of view.”

Within weeks of the procedure, patients have shown marked improvements, but the change comes before the graft of new tissue takes hold, Jacques said. Doctors haven’t been able to determine whether the grafts themselves are helping patients or whether patients are responding temporarily to the trauma of the operation.

Since it takes up to 15 years for Huntington’s disease to run its course, it will be several years before researchers know whether fetal-implant treatments work.

“So far, these people do seem to be getting better, or holding on, in terms of mental function,” Jacques said. “That is probably the most gratifying part of it. But I don’t want to give wrong impressions. I don’t think we’ve cured anybody.”

Whitmyer says she’s aware of the uncertainty, has talked with patients who have undergone the procedure and is convinced it’s the best chance she has to stave off the slow deterioration. At the worst, she said, the treatment could slow the course of the disease, buying time as researchers continue seeking a cure.

“There’s tremendous hope,” said Robinson of the Hereditary Diseases Foundation. “For the first time, [learning] the cause of cell death may be at hand.”

Huntington’s disease is an insidious killer, caused by a genetic malformation that leads a protein called huntingtin to build up abnormally in certain brain cells. Those cells eventually die, leading to loss of body control, personality changes and dementia, then death within 10 to 15 years.

The disease usually develops in people between the ages of 30 and 50, well after they have become parents themselves. Children of those with the gene have a 50% chance of carrying the gene themselves. Whitmyer said her daughter knows of her own potential to have the errant gene, which invariably leads to the disease, but she has not been tested.

Although the disease is genetic, Whitmyer says neither of her parents, who are still living, have the disease. She says she apparently developed the protein spontaneously, which Jacques described as a rare occurrence.

While Whitmyer’s condition was diagnosed 11 months ago, her symptoms began several years ago, said Peter S. Odegard, former conductor of the Irvine Symphony who was divorced from Whitmyer in 1991.

“It’s destroyed her life,” he said. “I don’t know how else to put it.”

He said the illness has robbed her of her musical abilities.

“Her sense of rhythm is affected,” he said. “She is just a shadow of her former self. She used to be a first-class flutist. It’s very sad.”

Whitmyer said beyond the physical problems, the disease has affected the way she processes information.

“Instead of being on time, I was 15 minutes late to everything, and not able to balance a checkbook or keep accurate records,” she said. “The fine details get lost for me.”

Drug therapy enabled Whitmyer to return to work recently after several months of not being able to perform, but she doesn’t know how long that will continue. Given her age--she’s 45--and the early stage of the disease, she’s optimistic about the treatment.

“There are no fears,” she said. “I have complete confidence in these people’s expertise.”

Donations in Whitmyer’s name are being accepted at the Neurosciences Institute, Good Samaritan Hospital, 637 S. Lucas Ave., Suite 501, Los Angeles, CA 90017. Information: (800) 762-1692.

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Hope for Huntington’s

Huntington’s disease, also called Huntington’s chorea, is a severe hereditary nervous system disorder. It causes degeneration of brain cells and is fatal. Through experimental surgery, Huntington’s (which killed singer Woody Guthrie) may be treated, and, in some cases, symptoms are alleviated. A look at the disease and its treatment:

SYMPTOMS

* Jerky, involuntary movements in face, arms, trunk

* No control of bodily functions

* Dementia in form of irritability, difficulty making decisions, apathy

* Progressive deterioration of memory and thought processes

* In children, disease is marked by loss of movement and muscle rigidity

WHO IT AFFECTS

* 25,000 Americans have Huntington’s disease

* Usually occurs between ages of 30 and 50

* An additional 60,000 carry the defective gene and will develop the disorder as they age

* Children of patients have 50% chance of developing Huntington’s

NEW TREATMENT

Defective brain tissue is replaced with cells from fetal tissue. Transplanted cells become integrated into the brain’s structure, restoring neural circuitry destroyed by the disease.

Sources: Encyclopedia Britannica, Stedman’s Medical Dictionary, Times reports; Researched by APRIL JACKSON / Los Angeles Times