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Gene therapy
Apr 21, 2006

Howdy Doc,

Since you work at UCLA, I think you're a good person to direct this question to. I read an article in the SF Chroincle that researchers at UCLA, Stanford and in Australia are conducting gene therapy research to halt the progression of HIV. They want to use stem cells to create a human white blood cell that is resistant to Hiv. The article made it seem like this was working.

My question it two-fold:

1) How likely is this to really be a breakthrough treatment for HIV? How much hope should we have?

2) In the article, the leading UCLA researcher(Mitsuyasu,I'm sure you know him) said he didn't believe gene therapy would ever replace anti-viral drugs.

Why would that be? The goal is to create a human antibody resistant to HIV, isn't it? If the new cell doesn't completely block HIV, then what is he doing this research for? Could this have been comment to appease drug companies?

Response from Dr. Daar

Howdy to you.

I am aware of the study you speak of and agree that it is very exciting. That being said, it is still preliminary and only time will tell how things will progress. Although I am generally a glass-half full kind of guy, I have been burned in this field too many times to get overly excited about advances in this early a stage of development.

I think Dr. Mitsuyasu's point was probably that current therapy has become so easy to take and well tolerated for most people that it is hard to imagine that it will be replaced in the near future by this or any other novel strategies in development. I suspect he was trying to provide important perspective so that people's hopes did not exceed the early scientific findings. He is also aware that there are many challenges that must be overcome before gene therapy will be highly efficacious. Research needs to start somewhere and small advances are important. It is difficult to predict at this time how many people might be helped by gene therapy even if it didn't completely replace antiretrovirals as first-line therapy.

After two paragraphs of speculation and opinion I can finally state something unequivocally. I do know Dr. Mitsuyasu, and I can assure you that he says nothing for the sole purpose of "appeasing drug companies." Furthermore, he is being realistic and there is nothing based on fact that he could say at this time that would discourage drug companies that are in the business of developing antiretroviral agents to treat HIV infection.

Best, Eric


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