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July 22, 1999

Conquering A Disease

A family's fund-raising efforts change the course of a rare neurological killer

http://www.jewishjournal.com/articles/item/conquering_a_disease_19990723

Los Angeles residents Pam and George Smith never expected to create a foundation that would raise more than $4 million for research. But then, they never expected their daughter to be born with a genetic disease so rare that it effects only between 500 to 1,000 Americans.

In the early 80s, Becca Smith seemed like any other happy, healthy infant. When it came time for her to advance from toddling to walking, however, Becca remained unusually wobbly. Medical tests initially indicated cerebral palsy. But eventually, a pediatric neurologist grew suspicious and decided to run additional tests. When the Smiths inquired about the doctor's concerns, he chillingly responded, "You don't want to know."

Becca was diagnosed with Ataxia Telangiectasia (A-T), a rare, progressive neurological disease. A-T effects the nervous and immune systems, causing loss of balance and increased frequency of infections. It has no known cure. When Becca was diagnosed with A-T in 1983 at the age of five, life expectancy was estimated at teens to early 20's.

The Smiths were determined to learn all that they could about this rare condition, but they found little information. At a time before the Internet, George and Pam Smith had to conduct their own fact finding mission, traveling across the county for information. What they uncovered was meager and discouraging. Speaking to scientists at the National Institutes of Health, the Smiths were told that, because of its rarity, A-T was an "orphan disease" and "not a funding priority."

Eventually, the Smiths discovered that a doctor in Los Angeles, Elena Boder, had originally diagnosed and named A-T. When they contacted Boder, she introduced the Smiths to Dr. Richard Gatti, a match that would change the course of the disease.

A Professor of Pathology and Laboratory Medicine at UCLA, Gatti was studying A-T as a model for understanding the relationship between immunology-how the body protects against disease and infection-and cancer. When Gatti described his research to the Smiths, George was sufficiently impressed to ask Gatti how much he would need to sustain his lab and concentrate on research. The answer was $100,000.

Smith responded, "You've got it. I'll do it."

Gatti also told the Smiths about a researcher named Yossi Shiloh, who was setting up a lab in Tel Aviv and needed $25,000 a year. Again, George responded, "You've got it."

The Smiths are no strangers to charitable activities. George, Chairman and CEO of a real estate financing company, has received the Man of the Year Award from the Jewish Federation's Real Estate Division and chairs the Israel bond appeal at Sinai Temple. Pam has earned the Federation's Lion of Judah designation, and raises funds for the Museum of Contemporary Art, where she serves as President of the Project Council.

But after committing funds to Gatti and Shiloh, says George, the couple realized, "We better start up a foundation and start raising money from our friends."

In 1984, the Smiths began the Ataxia-Telangiectasia Medical Research Foundation (A-TMRF), beginning what George calls a "sixteen year odyssey ... to slow the progress of the disease and ... someday find a cure."

To generate funds, Pam initiated an annual luncheon. Now in its 13th year, the event attracts about 200 women. Soon George instituted the annual "George Smith Partners Real Estate Luncheon," which attracted more than 1,600 professionals this year. He also began a program for senior real estate executives, offering an exclusive Executive Luncheon Series in exchange for a $2,500 membership. The fruits of these endeavors have enabled the A-TMRF to fund a permanent laboratory for Gatti at UCLA, develop Shiloh's lab in Tel Aviv, host three International Medical Conferences on A-T and award research grants to scientists throughout the globe.

When Gatti first met the Smiths, he had just petitioned the NIH for a grant to locate the chromosomal region of the A-T gene, but was told that the task he proposed was "Herculean" and "would probably never reach fruition." The Smith's funding enabled him to take the time needed-14 years-to pursue the task.

"Federal funding is notoriously fickle," explains Gatti, noting that the NIH likes to see substantial progress on research it has funded within three years. Without the A-TMRF, he says, the project would have "died on the vine."

In 1988, Gatti and his lab made history by locating the chromosome which carried the A-T gene. Gatti's work cleared the way for a second major breakthrough in 1995, when Dr. Yossi Shiloh's lab in Israel cloned the A-T gene. This meant that researchers could finally study the A-T gene instead of looking for it, turning their attention to developing ways to treat patients and slow the disease's progression.

Now, A-T has gone from a disease that Gatti says, "my colleagues couldn't pronounce" to a hot research area. Approximately 200 laboratories throughout the world are presently involved in research on A-T, compared to only a handful just a decade ago.

The NIH recently awarded Gatti and a colleague $2.4 million to further study A-T and its relationship to cancer.

But as research progresses, the clock continues to tick for Becca, now 21 years old. Scientific developments have meant improvements in diagnosis of the disease, but still no cure or treatments.

A-T has robbed Becca of her ability to walk without assistance. Her speech is slurred and she depends on others for assistance with writing and dressing. Although Gatti says life expectancy for A-T patients in the U.S. has climbed to the 30s and 40s, the Smiths can't help but wonder whether the research will progress quickly enough to help their daughter.

But Becca, whom Gatti says is "in good health," seems to live her life in the present. She graduated high school and is enrolled in Moorpark College in pursuit of an Associate of Arts degree. Four years ago, the Smiths moved from the westside to Calabasas so Becca could ride horses, an activity she began at the age of nine. Riding, swimming and working out with a physical therapist helps keep Becca strong.

Now, like her parents, Becca has gotten into the fund-raising game. In May, she and Pam opened Becca's Chic Boutique, a designer resale shop on Ventura Boulevard in Woodland Hills. "We have great clothing from many of the top designers with stores on Rodeo Drive," Becca boasts. Of course all proceeds benefit the A-TMRF.

George Smith says that the A-TMRF's luncheons and programs provide him with "a venue to strike at the consciousness of people." In all his various activities, says Smith, "My theme is: Go do something constructive with your life." The approach seems to run in the family. To find out more, call A-TMRF at (818) 704-8146.

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