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posted by cmn32480 on Thursday March 09 2017, @12:14PM   Printer-friendly
from the about-bloody-time dept.

Dr. Lowe, from In the Pipeline, writes about an apparent cure for sickle-cell disease and the challenges of expanding expensive cures to developing countries:

News came recently of an apparent cure, via gene therapy, of sickle-cell disease in a young patient (whose condition was refractory to hydroxyurea and the other standards of care). Blood-cell diseases are naturally one of the main proving grounds for things like this, since their stem cell populations are in easily localizable tissues and the techniques for doing a hard reset/retransplantation on them are (in some cases) well worked out.

This is an important result, but all such approaches face a possible disconnect as they move forward. As it stands, such gene therapy is a rather expensive and labor-intensive process. Patients are carefully identified and handled one at a time, and there are a limited number of medical centers in the entire world that can operate at this level. The problem is, none of them are particularly close to the great majority of people who actually have sickle cell disease.

[...] Is there any hope that gene therapy and cell replacement could get to the point that you could carry it out at a useful rate in some of the places where it would be needed the most? That's going to to hard, but this article at Technology Review by Antonio Regalado shows some progress:

In October, (Jennifer) Adair demonstrated a new technology she thinks could democratize access to gene therapy. Tweaking a cell-processing device sold by German instrument maker Miltenyi, she mostly automated the process of preparing blood cells with a gene therapy for HIV that her center is also testing. Cells dripped in one end came out the other 30 hours later with little oversight needed. She even added wheels. Adair calls the mobile lab "gene therapy in a box."

[...] The many companies that are working on such therapies seem to be paying attention to this sort of work, because it's not only a possible path to getting clinical trials run (and eventually patients treated) in the regions where most such patients are to be found. Companies are going to be selling such things first to people in the wealthier developed countries, but that's only the beginning of the story (as it has been with antiretroviral drugs).

http://blogs.sciencemag.org/pipeline/archives/2017/03/08/gene-therapy-needs-machines
https://www.technologyreview.com/s/603762/this-lab-in-a-box-could-make-gene-therapy-affordable/
http://www.nejm.org/doi/full/10.1056/NEJMoa1609677
https://en.wikipedia.org/wiki/Sickle-cell_disease
https://en.wikipedia.org/wiki/Gene_therapy


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  • (Score: 2) by nobu_the_bard on Thursday March 09 2017, @03:10PM (4 children)

    by nobu_the_bard (6373) on Thursday March 09 2017, @03:10PM (#476961)

    We'll get there eventually. It may take 5 years or 50 or 500, but eventually we'll get there, and everyone will have it.

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    Total Score:   2  
  • (Score: 2) by Rivenaleem on Thursday March 09 2017, @04:49PM (3 children)

    by Rivenaleem (3400) on Thursday March 09 2017, @04:49PM (#477012)

    I wonder whether Aspirin was expensive when it first came to market? I do know that it was (adjusted for inflation) $16.39 a bottle (24 tablets) back in 1932, over 40 years after it was released (disclaimer: this price might be influenced by the great depression). So many things influence the price of something, and it takes a very long time before the price is solely influenced by "how much it costs to make and administer" and not "how do we recoup the cost of inventing it and the cost of the things we failed to invent".

    • (Score: 0) by Anonymous Coward on Thursday March 09 2017, @08:10PM (2 children)

      by Anonymous Coward on Thursday March 09 2017, @08:10PM (#477105)

      I think the price, particularly in pharmacological developments, never tends to come down to 'price to make and administer'. It's just an endless game of maximizing the sales x price curve with complete disregard for any and everything else. Aspirin is an amusing example. For those that don't know, the active ingredient in aspirin literally grows on trees, common ones - willow. Cultures for centuries have chewed on the bark, brewed tea from the leaves/bark, etc to create what is for all intents and purposes - aspirin. Buying a batch of off brand aspirin today is still going to be nicely marked up at $0.01 a pill.

      Medicine and capitalism just don't mix well. Competition doesn't work because the expensive to develop, near free to produce nature of medicine means some sort of government granted monopoly (eg - patents) is generally required. And when somebody is sick their price elasticity is basically 0. For potentially terminal illnesses set the price at "everything you have and then some" and people would still buy the products since the alternative is generally seen as less pleasant.

      Though maybe the bigger question is why there is no Elon Musk of medicine or most industries in general - somebody driven by motives beyond just the accumulation of wealth.

      • (Score: 2) by Grishnakh on Thursday March 09 2017, @08:49PM

        by Grishnakh (2831) on Thursday March 09 2017, @08:49PM (#477122)

        For those that don't know, the active ingredient in aspirin literally grows on trees, common ones - willow. Cultures for centuries have chewed on the bark, brewed tea from the leaves/bark, etc to create what is for all intents and purposes - aspirin.

        Not exactly. Willow bark has salicylic acid, and goes back to ancient Sumer and Egypt (more than just "centuries"). Aspirin is a derivative of this, acetylsalicylic acid, and is supposed to be less irritating.

        https://en.wikipedia.org/wiki/Aspirin#History [wikipedia.org]

      • (Score: 0) by Anonymous Coward on Thursday March 09 2017, @09:13PM

        by Anonymous Coward on Thursday March 09 2017, @09:13PM (#477136)

        somebody driven by motives beyond just the accumulation of wealth

        There are plenty but human health is a incredibly complex. It is difficult to develop prototypes, hard to isolate variables to experimentally test, difficult to optimize, basically impossible to extrapolate results to other populations (or even individuals), carries a high risk of harm for volunteers, requires access to healthcare facilities and expertise, ... I could go on for a while and get more specific but I'm sure you get the point.