Sunday, August 20, 2006

Gene Therapy and Arthritis

Arthritis consists of over 100 different conditions, ranging from minor conditions like 'tennis elbow' to very serious conditions. And arthritis can can affect both the young and the old. Here are some facts on arthritis from the Arthritis Foundation:

Number of Americans with arthritis or chronic joint symptoms:

--2005 – 66 million (nearly 1 in 3 adults)
-- 42.7 million have doctor-diagnosed arthritis and 23.2 million people live with chronic joint symptoms, but have not been diagnosed by a doctor.
--Arthritis is one of the most prevalent chronic health problems and the nation’s leading cause of disability among Americans over age 15.
--Arthritis is second only to heart disease as a cause of work disability.
--Arthritis limits everyday activities such as walking, dressing and bathing for more than 7 million Americans.
--Arthritis results in 39 million physician visits and more than a half million hospitalizations. Costs to the U.S. economy totals more than $86.2 billion annually.
--Arthritis affects people in all age groups including nearly 300,000 children. --Baby boomers are now at prime risk. More than half those affected are under age 65.
--Half of those Americans with arthritis don’t think anything can be done to help them.
--Arthritis refers to more than 100 different diseases that affect areas in or around joints.

The Boston Globe has this interesting article on gene therapy and arthritis. Here is a snippet from the article:

One effort, led by a Harvard Medical School researcher, is focusing on a simple idea: Inject into the diseased joint a gene that will continuously pump medicine right where it is needed. Another project , led by a Maryland company, will instead use genetically modified cells to prompt growth of damaged cartilage.

Some of the work will piggyback on gene therapy experiments in rheumatoid arthritis that are showing hints of effectiveness.

The arthritis studies are part of an expansion of gene therapy research to diseases that are neither purely genetic nor necessarily lethal. Seven years after the death of a healthy teenager [Jesse Gelsinger] in a flawed experiment stalled most gene therapy studies, research is booming in diseases ranging from Alzheimer's and angina to cancer and multiple sclerosis....

Paul Gelsinger, Jesse's father, warns potential volunteers to get involved in gene therapy studies only after asking lots of questions about safety. ``If it's not life-threatening, I would go for much more conventional treatment," he said.

Carlene Lauffer, however, said she would volunteer in a minute, although her arthritis is too advanced to qualify for Evans's research. Lauffer, 78, suffers from osteoarthritis and rheumatoid arthritis and has had finger joint replacements and a hip replacement. She takes medicine for rheumatoid arthritis, an auto-immune disorder in which the joint lining swells and produces a substance that destroys the joint's surface. She also takes pain pills for her osteoarthritis.

``I think there's a need for gene therapy" for arthritis, said Lauffer, of Weirton, W.Va. ``We can maybe lick this."

In 1996, Lauffer was the first patient to undergo gene therapy for rheumatoid arthritis in an experiment run by Evans and colleagues, then at the University of Pittsburgh, that was designed to test only the principle and safety. The researchers injected her knuckles with the gene therapy or a placebo, monitored it for a week, and then removed and replaced the diseased joints. In Lauffer and eight other volunteers, they found that the gene entered cells in the joint and pumped the same medicinal protein now being used in the osteoarthritis research. Five years later, none of the volunteers had any ill effects.

For rheumatoid arthritis, which affects more than 2 million Americans, Evans and at least four other groups of scientists are pursuing clinical trials in gene therapy. Targeted Genetics, a Seattle company, is furthest along, with preliminary results that show a 20 to 30 percent reduction in swelling and tenderness in some patients, said Pervin Anklesaria, vice president of therapeutic development. The results have not yet been published.