Posted on 12/27/2004 8:12:01 PM PST by neverdem
LAST RESORTS
Everything literally changed in my life in one brief moment," said Stephen R. Neiley III. "There's nothing anyone can do about it. Nobody knows why."
Ten years ago, he was perfectly healthy, with a successful career and a family, when suddenly, for the first time in his life, he had a seizure. It was the beginning of a long and relentless illness, an intractable form of epilepsy that led him to give up his career, contributed to the demise of his marriage and left him, at 51, living on a farm in northwestern Pennsylvania with his parents.
Mr. Neiley was the first of five people whose lives, abruptly transformed by illness, were described in The New York Times this year in the series "Last Resorts." The patients had one element in common: they, or their parents in the case of two infants, pinned their hopes on experimental or unproven treatments because standard treatments either did not work or did not exist, or because doctors were genuinely unsure of the best way to help them.
In most cases the families were guided by their own doctors to enter studies or try experimental treatments. But one family found their own way by searching the Internet, a tool that is increasingly putting patients in direct contact with all that medicine has to offer.
In recent interviews, the patients or their family members discussed their decisions to venture into the uncertain world of medical experiments, and what had happened to them since. None expressed regrets about the paths they had chosen, though all had suffered from difficult treatments, side effects or complications, and the parents had agonized over decisions that led to painful procedures for their babies. Like Mr. Neiley, all said the illnesses had suddenly changed their lives and the lives of their families forever.
"You're not in the same world as anybody else," one of the parents said.
Last spring, Mr. Neiley had a pair of electrodes implanted in his brain in hopes of stopping or decreasing his seizures. The surgery, at New York Weill Cornell Center, was part of an experiment intended to test a pacemaker-type device that inhibits nerve impulses in a brain region that is too active in people with epilepsy.
In mid-December, Mr. Neiley said he thought the device was "somewhat working."
His course since the surgery has been uneven. Initially, he noticed a decrease in small seizures, the kind that cause twitching or jerking but not unconsciousness. More recently, though, they have increased. And from one to three times a week he still has severe grand mal seizures - but overall those seem to have decreased, he said.
Other problems have surfaced, including what he described as "spacious, dizzy feelings." He said he hoped the doctors would adjust the brain stimulator to deliver stronger shocks.
"I'm really curious to know what they plan on doing," he said. "They won't tell me because it's all part of the secrecy of the whole experiment. In the meantime I'm going day by day. There's not much else I can do."
All in all, Mr. Neiley said, he was "ecstatic" about the device because it seemed to be helping. But, he added, "After this thing's over with, I've already signed up for another experiment."
"I'm willing to do anything I can to find a cure or at least a stabilization of seizures," he said.
Ric Ray, who at age 52 had end-stage heart failure, had bypass surgery last summer to restore blood flow to his heart, and an additional operation to reshape its main pumping chamber and cut away scar tissue, to help it pump more efficiently. His operation was also part of a study, which was intended to identify the best treatment for patients like him with scarred, enlarged and weakened hearts. Other treatments in the study included heart medicines instead of surgery, and medicines combined with bypass surgery.
Mr. Ray had considered a transplant, but chose the study as a less drastic option that might, at the least, help him postpone a transplant.
Recently, he and his wife, Monika, said they believed the surgery had helped. Before it, he could barely move about the house without getting winded. But in the weeks and months after the surgery, he became able to take walks and ride an exercise bike. Tests showed that his heart function had improved. He had a problem a few months ago, some bleeding from the patch surgeons had sewn onto his heart - possibly brought on by his having lifted something too heavy - but it resolved.
"Ric is doing great," his wife said. "The doctors said he's doing more than they expected. We're just elated. He looks good, his color is good. He takes only about one nap a day. He used to take a lot of naps."
Mr. Ray said: "I'm doing O.K. There's times where you're not as well as you think you should be."
But, he said, given the extensive damage from his heart attack in 1987, he knew he could not expect to feel as strong as someone with a healthy heart. He likened himself to a person climbing a mountain but settling for the view halfway up instead of trying to get all the way to the peak.
"As far as breathing, getting out of breath, I have no problem with that at all," he said.
He said he was glad he had the surgery. Before it, he said, he saw no future. "Now, I don't see any problem if things stay the way they are," he said. "Hopefully, it will be years and years and years."
Sister Mary Andrew Matesich, 65, began entering clinical trials about six years ago, when breast cancer began spreading to her lungs. She has a doctorate in chemistry, and she was then the president of Ohio Dominican University.
Experimental treatments appealed to her for several reasons, she said: the scientist in her was intrigued by the chance to take part in research, her religious side liked the idea of joining studies that might help other women, and as a patient, she hoped there was a chance, however slim, that a new therapy might buy her some extra time.
She has participated in four studies. In some, her tumors shrank, but eventually they returned. And she has suffered side effects, including a severe lung disorder and nerve damage.
In mid-December she said, "I'm doing pretty O.K., but the disease is certainly advancing." There are tumors in her liver as well as her lungs.
In the fall, she said, she had a "little setback": tests showed that cancer had spread to one of her hipbones, and had eaten away 35 percent of the narrow neck of the femur. Doctors proposed inserting a titanium rod into the femur, from hip to knee, to keep it from breaking.
Sister Mary Andrew told them she was not inclined to have the surgery if she would be at "end-stage disease" in six months, she said. Her oncologist said he did not think she had reached that point, so she went ahead with the operation.
"It went amazingly well," she said. "It's remarkable how they do this. They said I would have surgery on Day 1, bear weight on Day 2 and go home on Day 3, and that's exactly what happened."
She also had radiation for the bone cancer, and said she was willing to consider still more clinical trials, including Phase 1 studies, which are generally the most risky because they are the first tests in humans and are meant to look for toxicity and to find out how high a dose patients can tolerate.
"I might be able to do Phase 1, something really way out of left field," she said. "But there's a very small nodule in my brain, and they don't know if it's cancer yet. You can't get into Phase 1 if you have brain metastases."
For the moment, she said, she was forgetting it all and heading to Washington to spend the holidays with her sister.
By the time Miles Coulson was 3 months old, his heart was failing - possibly the result of a viral illness - and was so weak that doctors feared he would not live long enough to receive a transplant. Though mechanical pumps to assist the heart are used to keep older patients alive in such situations, none small enough for Miles was approved in the United States.
In a desperate effort to save him, doctors at Lucile Packard Children's Hospital, at Stanford University, arranged to have a miniature device, the Berlin Heart, flown in from Germany. Miles had surgery on July 13 to connect his heart to the pump, and it kept him alive long enough to receive a transplant on Sept. 6.
Recently, he and his parents were able to return home to Dixon, Calif. Miles is nearly 9 months old, and he likes to play with his older brother and watch cartoons, his mother, Leigh Bills, said.
During a telephone interview on Dec. 23, she said: "Miles is doing great. Right now he's sitting in his chair smiling at me, playing with a stuffed bear. We're really lucky, really blessed. He's chubby and very happy."
He laughs often, she said, adding that he sounds like a "little old man," possibly because he is still hoarse from having had so many tubes in his throat. "It's a very grown-up laugh," she said.
Looking back, Ms. Bills said, Miles's course was so arduous and painful that sometimes she and her husband wondered if they were doing the right thing in continuing to allow drastic and invasive procedures to try to save him.
"The decisions we made, we couldn't be sure they were right for Miles, but they were right for us," she said. "Some did prolong Miles's suffering - with the hope of this exact situation. We're lucky for everyone's sake it went this way."
For the parents of another infant, 1-year-old Hannah Stimell, it is too soon to tell whether the radical course of therapy they chose for her will save her life or prevent brain damage from a fatal genetic disorder, Type A Niemann-Pick disease. In July, Hannah's parents, Scott and Jill Stimell, traveled from their home in Long Beach, N.Y., to Fairview-University Children's Hospital, at the University of Minnesota, where doctors treated Hannah with high doses of chemotherapy followed by a transplant of umbilical cord blood.
They, too, struggled over whether they should put her through such a difficult procedure. "Do we want to do this to her, or just enjoy the time we have with her?" Mrs. Stimell asked. Ultimately, they decided to treat her; it was her only chance.
Now, new cells from the transplant are providing normal levels of an enzyme that Hannah needs to break down deposits of a fatty substance that damages the brain and other organs. Her doctors say she is developing neurologically, at a time when she would be expected to regress. But it is not yet known whether the enzyme is getting through to her brain. The disease has not been successfully treated before. Time will be the test.
"I never thought we would get this far, even," said Mrs. Stimell. "I'm cautiously optimistic."
Putting a Weakened Heart in Experimental Hands
Heeding a Call to Test Breast Cancer Treatments
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