TELETHON TO HERALD RAPID RESEARCH PROGRESS

TUCSON, Ariz., Aug. 16, 1999 -- Human safety trials of gene therapy in one form of muscular dystrophy are expected to begin in September. A clinical trial to test a common antibiotic as a treatment for another form of muscular dystrophy is slated to start by year-end. As a result of these and other promising research initiatives, the 1999 Jerry Lewis MDA Telethon for the Muscular Dystrophy Association will celebrate rapid scientific progress.

"Our scientists have devised a protocol to test the safety of gene therapy in people with a form of limb-girdle muscular dystrophy," Robert Ross, MDA executive director and executive producer of the Telethon, said. "We're awaiting final National Institutes of Health review of the protocol and expect, in September, to begin the gene therapy trial already authorized by the Food and Drug Administration."

This gene therapy safety trial for limb-girdle muscular dystrophy is the first for a neuromuscular disease. Gene therapy involves replacement of disease-causing genes with correctly functioning ones. The approach may eventually lead to cures for many genetic diseases.

"Public support of the Telethon enabled MDA to commit $9.2 million to this pioneering gene therapy effort. It also enables MDA to fund more than 400 scientific teams each year, including the group that will soon begin a clinical trial of gentamicin for Duchenne muscular dystrophy," added Ross.

In July, MDA-funded investigators found that gentamicin, an antibiotic commonly used for bacterial infections, improved muscle function and reversed signs of muscular dystrophy in mice. The mice - like 15 percent of Duchenne patients - experience muscle wasting because a genetic flaw known as a "premature stop codon" instructs muscle cells to stop making the needed dystrophin protein.

In the latest fiscal year, MDA spent some $93.9 million in its programs of research, services and public health education. This year's Telethon, to be broadcast Sept. 5-6, will support MDA programs to benefit those affected by 40 neuromuscular diseases in the upcoming year.

"I'm deeply moved by the caring response millions give the Telethon each year. Their contributions make MDA a worldwide leader in research, and the source for the nation's most comprehensive medical services program of any voluntary health agency," said Jerry Lewis, MDA National Chairman.

Consistently ranked among leading national voluntary health agencies for the level of income expended on program services, almost all of MDA's income is derived from private voluntary contributions. MDA is the first nonprofit organization honored with the American Medical Association's Lifetime Achievement Award "for significant and lasting contributions to the health and welfare of humanity."

Research Progress

To expedite research seeking treatments and cures for neuromuscular diseases, MDA spends an average of $48 a minute year round. Since last year's Telethon, MDA-funded investigators have:

• Prepared for human trials of gene therapy for a form of limb-girdle muscular dystrophy and of gentamicin for a form of Duchenne muscular dystrophy.
• Delivered therapeutic genes to leg muscles of hamsters via the bloodstream, a technique that may one day make it possible to deliver genes to humans without multiple muscle injections.
• Continued efforts to design trials of gene therapy for Duchenne muscular dystrophy.
• Found a compound that may stimulate muscle cells grown in the laboratory to produce more utrophin. This protein might be able to stand in for dystrophin, the protein missing or faulty in the muscle cells of youngsters with Duchenne and Becker muscular dystrophies.
• Maintained 19 MDA/ALS research and clinical centers across the country, where continued human trials of potential drugs for amyotrophic lateral sclerosis (ALS, or Lou Gehrig's disease) are underway.
• Discovered that molecular "chaperones," proteins that protect some other proteins from harmful chemical reactions, may play a key role in whether nerve cells live or die in ALS.
• Identified the gene that, when flawed, causes nemaline myopathy, a disease involving muscle weakness and sometimes respiratory failure.
• Shown that the dietary supplement creatine prolonged life in mice with a form of ALS, and that it may increase muscle strength in people with muscular dystrophies and other myopathies.

Comprehensive Services

MDA allocates some $50 million in its services program to help adults and children affected by neuromuscular diseases. During the past year, MDA:

• Sent some 4,100 youngsters with neuromuscular diseases to more than 80 sessions of MDA summer camp, at a cost of some $400 per camper.
• Provided for tens of thousands of visits to MDA's 230 hospital-affiliated clinics for initial diagnosis and follow-up care.
• Gave tens of thousands of flu vaccinations to help prevent those with neuromuscular diseases from developing serious or life-threatening respiratory complications.
• Assisted with the purchase of more than 3,300 wheelchairs and leg braces, and with the repair of nearly 5,000 wheelchairs and sets of braces.
• Conducted meetings of more than 230 MDA-facilitated support groups for those with neuromuscular diseases and their families.

Vital Education

Telethon donations also help MDA educate professional and public audiences about neuromuscular diseases. The extensive program includes seminars, scientific conferences, workshops, media interviews, videos, publications, and exhibits of the MDA Art Collection. These outreach initiatives are augmented by MDA's toll-free information hotline, (800) 572-1717; by the Association's Web site, www.mda.org, where more than 200 leading clinicians and investigators offer authoritative replies to specific questions about neuromuscular diseases.

MDA's 1999 Jerry Lewis Telethon will originate from CBS Television City in Hollywood and be broadcast by some 200 television stations to an audience of some 75 million viewers. The Telethon also will be Webcast live in English, Spanish and Japanese at www.mda.org. The world's first multilingual programming on the Internet, the streaming video Webcast is being hosted by MDA national sponsors AT&T, RealNetworks and CyraCom International.