Re: MED/NEURO: Human trials of spinal nerve regeneration in ~1 year?

Robert J. Bradbury (
Tue, 12 Oct 1999 11:57:06 -0700 (PDT)

On Tue, 12 Oct 1999, J. R. Molloy wrote:

> FDA suspends gene therapy studies
> Pennsylvania scientists had placed healthy genes in his liver to
> combat a rare metabolic disease.

Read this as - without treatment it is likely the patient would have died. Certainly their quality of life would have been very poor.

> Penn's study has been halted while
> scientists there, along with federal regulators, try to determine
> what went wrong.

Read this as - "accidents happen".

> The Food and Drug Administration decision late
> Friday halts two separate experiments by Schering-Plough to try gene
> therapy as a treatment for liver cancer and for colorectal cancer
> that spreads to the liver.

Read this as - we are going to delay this and see if we have a situation like we had a few years ago with the protein supplements (where the manufacturing process got contaminated and poisoned a few people).

Manufacturing gene therapy vectors is not an easy process due to the fact that you are dealing with highly complex structures and many steps in the process. There are things that can go wrong and ways contaminants can creep into the process (which is not to say this is what happened with the person cited).

However, you have a "damned if you do and damned if you don't" situation. If you don't use these technologies the patients *will* end up *dead* (though you could freeze them). If you do use the technologies the *might* end up dead sooner or they just might get better.

So press forward we must.