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10-14-2002, 11:28 PM
"I just have to push and push and use all my strength to get any movement," she says.
Her struggle motivated her to form the Parkinson's Action Network from her home in Northern California. She lobbies in Washington and around the country for research funds to help scientists find a cure.
"If there isn't a cure for Parkinson's," Samuelson says, "I'll lose almost everything that I have."
One of the research efforts that Samuelson's Network supports is a promising approach with origins in a most unlikely source: HIV, the virus that causes AIDS.
A Delivery Aid
In a laboratory near San Francisco, researchers at Cell Genesys hope to perfect a therapeutic strategy that cashes in on the AIDS virus's ability to penetrate a cell's nucleus and deliver a huge genetic payload.
Cell Genesys is one of several labs that have been experimenting for the last few years with lentiviruses, a class of viruses that includes HIV. Peter Working, vice president of research and development at Cell Genesys, considers HIV the most potent virus of the lentivirus family.
"It can deliver a large amount of genetic material," he says, "which means that you can deliver big genes to fix big problems."
In most cases the genetic payload of HIV hijacks an immune cell's genes, forces it to make copies of the HIV's genes, and then sends those copies out to hijack other cells. The result cripples or shuts down the body's ability to defend itself.
Under lentivirus research, the genetic parts of HIV that make it dangerous are removed so that it can't attack immune cells or even reproduce to affect other cells. Instead, researchers replace the bad parts of HIV with therapeutic genetic material. In effect, the virus is turned into one big delivery truck that unloads genetic medicine.
"This is not a virus, a delivery vehicle that can divide," Working says. "It can't reproduce itself."
In diseases such as Parkinson's disease, Alzheimer's disease, and other disorders of the central nervous system, lentiviral therapy might be tailor-made to suppress or stop the disease.
Since the lentivirus can't reproduce itself, it can affect only the cell into which it penetrates. In disorders with a localized origin, such as Parkinson's disease, thousands or millions of converted HIV vectors would be sprinkled or infused onto disease spots to affect a genetic change.
Safety Concerns
The lentiviral approach offers an alternative to embryonic stem cell therapy, which triggers volatile ethical and religious debate. But converted HIV vectors may generate their own concerns.
"This is going to be the PR question going forward. It's HIV," says John McCamant, a Berkeley, California, biotech stock analyst.
McCamant, author of The Medical Technology Stock Letter, tracks all forms of biotech research, and he foresees promise in lentiviral therapy. But its origins in AIDS may obstruct or stall the pace of research, he says.
"The stakes have gone up tremendously," he says. "And this technology is gonna have to be beyond safe."
However, researchers at San Diego's Salk Institute, which helped pioneer lentiviral therapy, are driven by industry excitement that soars up to the Food and Drug Administration. Indeed, the FDA held a Washington seminar last October devoted largely to the promises of lentiviral vectors.
Cell Genesys' Working recognizes the risks and the potential.
"Really, of course, before we have to convince people it's safe, we have to convince the FDA that it's safe," he says.
The proof of safety may come sometime next year, when the first lentiviral vectors could go into a clinical human trial.
There's no need to convince Joan Samuelson. She's confident that if researchers develop an experimental Parkinson's disease drug from HIV, a long line of willing recruits will be ready and waiting to test it.
"We're scared already," she says. "AIDS doesn't scare us."
http://abcnews.go.com/sections/scitech/TechTV/techtv_HIVdelivery021010.html
Her struggle motivated her to form the Parkinson's Action Network from her home in Northern California. She lobbies in Washington and around the country for research funds to help scientists find a cure.
"If there isn't a cure for Parkinson's," Samuelson says, "I'll lose almost everything that I have."
One of the research efforts that Samuelson's Network supports is a promising approach with origins in a most unlikely source: HIV, the virus that causes AIDS.
A Delivery Aid
In a laboratory near San Francisco, researchers at Cell Genesys hope to perfect a therapeutic strategy that cashes in on the AIDS virus's ability to penetrate a cell's nucleus and deliver a huge genetic payload.
Cell Genesys is one of several labs that have been experimenting for the last few years with lentiviruses, a class of viruses that includes HIV. Peter Working, vice president of research and development at Cell Genesys, considers HIV the most potent virus of the lentivirus family.
"It can deliver a large amount of genetic material," he says, "which means that you can deliver big genes to fix big problems."
In most cases the genetic payload of HIV hijacks an immune cell's genes, forces it to make copies of the HIV's genes, and then sends those copies out to hijack other cells. The result cripples or shuts down the body's ability to defend itself.
Under lentivirus research, the genetic parts of HIV that make it dangerous are removed so that it can't attack immune cells or even reproduce to affect other cells. Instead, researchers replace the bad parts of HIV with therapeutic genetic material. In effect, the virus is turned into one big delivery truck that unloads genetic medicine.
"This is not a virus, a delivery vehicle that can divide," Working says. "It can't reproduce itself."
In diseases such as Parkinson's disease, Alzheimer's disease, and other disorders of the central nervous system, lentiviral therapy might be tailor-made to suppress or stop the disease.
Since the lentivirus can't reproduce itself, it can affect only the cell into which it penetrates. In disorders with a localized origin, such as Parkinson's disease, thousands or millions of converted HIV vectors would be sprinkled or infused onto disease spots to affect a genetic change.
Safety Concerns
The lentiviral approach offers an alternative to embryonic stem cell therapy, which triggers volatile ethical and religious debate. But converted HIV vectors may generate their own concerns.
"This is going to be the PR question going forward. It's HIV," says John McCamant, a Berkeley, California, biotech stock analyst.
McCamant, author of The Medical Technology Stock Letter, tracks all forms of biotech research, and he foresees promise in lentiviral therapy. But its origins in AIDS may obstruct or stall the pace of research, he says.
"The stakes have gone up tremendously," he says. "And this technology is gonna have to be beyond safe."
However, researchers at San Diego's Salk Institute, which helped pioneer lentiviral therapy, are driven by industry excitement that soars up to the Food and Drug Administration. Indeed, the FDA held a Washington seminar last October devoted largely to the promises of lentiviral vectors.
Cell Genesys' Working recognizes the risks and the potential.
"Really, of course, before we have to convince people it's safe, we have to convince the FDA that it's safe," he says.
The proof of safety may come sometime next year, when the first lentiviral vectors could go into a clinical human trial.
There's no need to convince Joan Samuelson. She's confident that if researchers develop an experimental Parkinson's disease drug from HIV, a long line of willing recruits will be ready and waiting to test it.
"We're scared already," she says. "AIDS doesn't scare us."
http://abcnews.go.com/sections/scitech/TechTV/techtv_HIVdelivery021010.html