Medical Gene Therapy Could be the Wave of the Future

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Tony Rook
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Medical Gene Therapy Could be the Wave of the Future

Despite the recent setup back in some of the clinical trials for medical gene therapy, I am convinced that this technology will be an extreme medical advancement for diseases that have very little alternative. I believe that the greatest threat to this therapy unfortunately could be the governments that regulate them.

omid
omid's picture
trook wrote:Despite the

trook wrote:

Despite the recent setup back in some of the clinical trials for medical gene therapy, I am convinced that this technology will be an extreme medical advancement for diseases that have very little alternative. I believe that the greatest threat to this therapy unfortunately could be the governments that regulate them.

I strongly believe that in other advanced countries gene therapy will progress while we are set back by our government regulations and we will suffer the consequences later. We might need their help and technologies in the future and pay a lot of money to be able to use them.

Tony Rook
Tony Rook's picture
omid wrote:trook wrote

omid wrote:

trook wrote:
Despite the recent setup back in some of the clinical trials for medical gene therapy, I am convinced that this technology will be an extreme medical advancement for diseases that have very little alternative. I believe that the greatest threat to this therapy unfortunately could be the governments that regulate them.

I strongly believe that in other advanced countries gene therapy will progress while we are set back by our government regulations and we will suffer the consequences later. We might need their help and technologies in the future and pay a lot of money to be able to use them.

The only "silver lining" that I am beginning to see regarding these setbacks is the court of public opinion. If the public begins to see the benefits, then the individual states can take matters into there own hands. This is evident in the move that California made by funding embyronic stem cell research through the state, thereby by-passing any negative federal regulations. Unfortunately, the downside to this is that in the United States if we move in that direction, then our greatest advantage is lost. That is, the cumulative assets of all state funding to support specific research is much larger than the majority of states can support (obviously there are exceptions, i.e. CA and NY).

Jason King
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I think you have to be

I think you have to be careful not to confuse gene therapy and stem cell therapy. Gene therapy will probably be able to offer a very customized solution to a few very wealthy patients, whilst stem cell therapy should allow more general medical applications, hopefully at a more affordable price.

Unfortunately, in the US you have a situation in which the people with the most political power have the lowest comprehension potential. I don't know whether the saying "The fish begins stinking from the head" is used in the US or not but I think it could be.

And if ALL life is to be preserved / protected (ie embryonic stem cells) why doesn't the US government show more compasion for individuals who have progressed past the embryo stage (prisoners on DEATH ROW, US citizens that can not afford health care, Iraqi civilians, most of Africa)? How can you be pro-capital punishment, support the NRA and still be anti-stem cell research?

jachmoody
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It seems to me you need to

It seems to me you need to get more informed about the US people and Government--it sounds like the left leaning media have affected your objectivity.

jim

Jason King
Jason King's picture
I was in the States just

I was in the States just before Iraq was invaded and had the "pleasure" of sampling some of your "left leaning media". If CNBC News is left leaning then I hope that never meet anyone with right wing tendencies.

swannnyy
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parvoman wrote:I think you

parvoman wrote:

I think you have to be careful not to confuse gene therapy and stem cell therapy. Gene therapy will probably be able to offer a very customized solution to a few very wealthy patients, whilst stem cell therapy should allow more general medical applications, hopefully at a more affordable price.

Curious, I always thought it was the other way around. Gene therapy is pushed as the proverbial "silver bullet" that will be able to fix genetic diseases once and for all, and would be administered just like any other treatment. Stem cell therapy, on the other hand, is something that will require very advanced (read expensive) medical/scientific facilities, and would therefore be out of reach for many people (in any case, I think it unwise to project the US health system's foibles onto the rest of the world, as it is different to pretty much every other country, rich or poor).

The other point to consider is exactly what we mean when we talk of gene therapy. Of the gene therapy projects started around the world, about 2/3 are for cancer treatment, and the others for genetic diseases. It seems to me that stem cell therapy won't work for the majority of genetic disorders such as CF etc), but gene therapy will work brilliantly, if some technical issues can be resolved.

The biggest problem (and therefore the biggest challenge) for non-cancer treatment gene therapy is that of making sure the new "corrective" DNA gets into the patient's genome safely, without causing other disorders, as happened in the French SCID trial. The correlative problem/challenge for cancer gene therapy is making sure only malignant cells are targetted.

Protoplast
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Swannny,

Swannny,
I have to say, I do agree with you. As far as I am aware, stem cell technology is highly personalised, as to be successful without the use of immunosupressants, you need healthy stem cells derived which are compatible with the person who is to be treated. Theoretically gene therapy can be across the board, cystic fibrosis for example has been put forward as a target for gene therapy. (Please correct me if I am wrong...) A mutation in a membrane bound transport protein leads to misfolding, and protein aggregation, so the cells do not produce functional membrane proteins. An idea was to create some viral vector with homolous regions for integration purposes to insert a functional gene which could be taken via inhalers so as to get the lungs, as the mutation in nearly all cases of CF is the same, the viral treatment would work across the board.
I will post some references and papers later.

Braisler
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I got this information from

I got this information from the CF Foundation Website: The comments are my own.

The first strategy to evaluate gene therapy in patients used a modified adenovirus (or cold virus) to deposit normal genes directly into damaged CF airway cells. In these experiments, researchers delivered the adenovirus as nose drops, or they drizzled the treatment down a bronchoscope (flexible tube) to reach CF cells lining the airways. Ideally, the healthy gene will be delivered via an inhaled aerosol or through a vein.

--In retrospect, the use of Adenovirus that would cause immune mediated inflammation in a patient that already had respiratory problems was ill advised. This brings to the forefront the idea that gene therapy trials need to have proper oversight from scientists who are trained in the field. It is no longer adequate to rely on the citizen members of an institutional review board to approve or deny a study of this nature. They simply cannot be well enough informed of the issues. They lack the scientific background to make informed choices about the proposed research.

In recent trials, researchers have been testing the safety and efficiency of the modified adeno-associated virus (AAV) delivery system -- a method that is less likely to trigger the body's immune response than the adenovirus. In addition to monitoring safety and efficiency, patients also are monitored to detect whether the normal CF gene has "turned on." If so, the CF gene should produce a normal protein that can transport ions (charged particles) across the cell membrane, a process that is vital to the health of cells lining the respiratory tract. The AAV trial showed temporary improvement in lung function in AAV-treated patients.

--The use of AAV presents an important advance since it is non-immunogenic as a vector. I don't know what promoter system they are using in this trial or the serotype of AAV, so I can't comment on that.

rajcog
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trook wrote:Despite the

trook wrote:

Despite the recent setup back in some of the clinical trials for medical gene therapy, I am convinced that this technology will be an extreme medical advancement for diseases that have very little alternative. I believe that the greatest threat to this therapy unfortunately could be the governments that regulate them.

what is said might be acceptable