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On the refrigerator of her Norwalk, Conn., home, Elizabeth Hart, 32, marks each of the daily squares of her calendar with an X, counting the days until her big event. With boundless hope and a case of the butterflies, she waits not for her wedding day or a due date, but for another occasion that will forever change her life-her brain surgery.

Hart was diagnosed with MS at the age of 22, just six months after she married Michael. What began as some puzzling numbness in her legs has deteriorated to a condition of extreme fatigue, muscle weakness, difficulty in walking, memory loss and depression. Worst of all for her are the tremors; her hands and head shake uncontrollably, making such everyday tasks as eating, drinking, reading or changing a light bulb exhaustingly difficult. Even holding her own children, ages 4 and 2, can be a nerve-racking experience. "I feel like I can handle everything else but the tremors," she says.

Now, for the first time in years, Hart has reason to hope. An FDA-approved surgical procedure called deep brain stimulation (DBS) may be capable of quieting her tremors. Used primarily on Parkinson's disease patients, the procedure involves implanting a "pacemaker" in the brain which emits finely tuned pulses of energy that relieve the patient's chaotic tremors without the cell destruction associated with traditional brain surgery.

Neurosurgeon Ali Rezai of the Cleveland Clinic Foundation, who has performed more than 400 such procedures, calls it one of the most important medical advances for Parkinson's disease in the past 30 years, Says Rezai. "Patients who could not brush their own teeth or feed themselves are now enjoying a full quality of life." It's All in the Genes

But while treatments such as DBS improve quality of life, they do not offer a cure. In this regard, much of the medical community is optimistic about the potential of gene therapy and stem-cell research. In this genetically engineered vision of the future, a diabetic may one day swallow an insulin gene that would provide the instructions for making insulin. A hemophiliac could have his genes altered to reduce the chances of spontaneous bleeding. A gene-therapy mouthwash laced with engineered viruses could battle oral cancer by fighting cancer-causing cells. All these treatments are already in the testing stages and may someday become reality. Still in its infancy, gene therapy might be used to treat, cure and perhaps even prevent disease by changing or replacing faulty genes with normal ones.

Already, a great deal of work is being done on inserting genes into brain cells to help with diseases such as Parkinson's and MS. Researchers in the United States and Switzerland, in fact, recently used a gene-therapy technique to reverse the damage of Parkinson's in monkeys.

And combining gene therapy with other treatments, such as radiation therapy in a cancer patient, could increase cure rates. In genetic radiotherapy, an experimental procedure at the Virginia Commonwealth University, cancer cells are infected with a virus that makes tumor cells more sensitive to-and more easily destroyed by-radiation. The combined treatment could increase cancer-cure rates up to 70 percent.

However, gene therapy is not without its challenges and risks. An experimental genetic therapy that seemed to cure a number of children born with life-threatening immune deficiency, sometimes referred to as "Bubble Boy" disease, has apparently caused another leukemia-like syndrome in one of those treated-a 3-year-old in France. And there are moral issues as well. Some worry gene therapy could be used to improve one's looks or intelligence.

The Promise of Stem Cells
Another area of great hope-and controversy-is stem-cell research. The master cells of the body, embryonic stem cells carry the most promise for medicine because they hold the ability to transform into any type of human cell: dopamine cells for a Parkinson's patient, for example, or neurons that could bring movement back to those paralyzed with a spinal-cord injury.

Like gene therapy, stem-cell research is still in its very early stages. It has been slowed by the ethical and political issues associated with use of human embryos, but its promise is unquestionable. Stem cells have already been successfully used for more than 20 years on leukemia patients to restore blood production after chemotherapy.

Parkinson's disease is another area in which stem-cell research holds great hope. In a recent study by Columbia University and University of Colorado researchers, for example, fetal dopamine-producing cells were implanted into the area of the brain that is deficient in Parkinson's patients. Symptoms such as stiffness and slowness of movement improved among some of the younger patients, though tremors generally continued. "The experiment was successful with some patients," says Columbia neurologist Stanley Fahn. "But we are concerned about possible side effects. How do we know the stem cells won't keep growing and form tumors, for instance?" Fahn believes that while the theories underlying stem-cell implantation are sound, more basic research is needed.

The Challenges Ahead
The challenge to find cures for deadly diseases goes beyond the medical and moral realm; there are huge financial hurdles as well. Take the case of cystic fibrosis, a genetic disorder in which the body's mucus becomes so thick and sticky it prevents a number of organs-particularly the lungs and pancreas-from functioning normally. Drug companies sometimes shy away from big investments into cystic fibrosis research because the market is considered too small; the disease, which lowers life expectancy to 33 years, affects just 30,000 people in the U.S.

Hope in such cases often comes in the form of private, patient-run organizations that have revved their search for a cure into high gear. The Cystic Fibrosis Foundation, for instance, has reinvented itself to spearhead a cure for its constituents through potent industry partnerships. Dr. Robert J. Beall, Ph.D., its president and CEO, runs the foundation completely atypical of most nonprofits, funding extensive research and forming powerful partnerships with innovative biotechnology firms such as Targeted Genetics and Inspire Pharmaceuticals.

"We differ from other not-for-profit organizations in that we take the responsibility on ourselves to find a cure," says Beall. In the past, the CF Foundation focused on understanding the basic defect in CF cells and on what can be done to correct it. To translate this knowledge from the test tubes to the patients, however, Beall created a business model more like that of a pharmaceutical company. "Using our own funds, we've discovered new compounds, created new networks to do trials and acted as our patients' pharmacy, distributing medication and ensuring the lowest costs."

Other times it takes celebrity status to help push the cause. Actor Michael J. Fox and boxing champion Muhammad Ali, both Parkinson's sufferers, recently testified before Congress for funding for the disease. Actor Christopher Reeve, paralyzed since his 1995 horse-riding accident, has asked Congress for more research dollars for spinal-cord injury.

Despite the enormous challenges, the search for cures goes on. Adds the Cleveland Clinic Foundation's Rezai, "Let us hope that as a civilized nation, we maintain the proper support-financial as well as academic-for the research efforts that are necessary to bring about a cure to any and all of the diseases and disorders that afflict humanity."

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