Lecture : Progress in Human Genome Therapy (Theodore Friedmann)

Happened to notice an announcement for a lecture today on human genome therapy in the Salk lecture series mailing for this week. Theodore Friedmann, a professor of medicine at UCSD and a pioneer in gene therapy at Salk back in the early 70s, was the speaker. Interesting timing, as I am just completing a paper on the Human Genome project. Well, completing may be too strong a word. But I have been reading quite a bit this quarter on the subject. This lecture affirmed my recent realization that the roots of the HGP are deep within the War on Cancer.

Friedmann observe the when gene therapy first emerged, as with the genome project, advocates had what he called a naïve model for genetic disease – basically, the central dogma. Bad gene –> bad protein –> disease. Indeed, it is probably inaccurate to suggest that there were two different camps that shared a erroneous view. Gene therapy and the genome project branched off from a common root in the 70s and early 80s where they shared a common paradigm and similarly over-optomistic expectation.

As research expanded, scientists started discovering all that complicated stuff about gene function and progress with gene therapy stalled. I guess things are moving forward, but there are still challenges with basic elements of therapy such as the delivery vectors being used to transfer genes. Jesse Gelsinger’s death it seems was not caused by anything associated with the genetic disorder itself that was the object of his treatment but was a result of a fatal massive immune response triggered by a protein associated with the vector transporting the genes.

Incidentally, Friedmann noted that there has been one successful study with gene therapy with SCID patients (bubble boys – it’s x-linked) in France. This was completed after Gelsinger’s death. The first success in over 600 clinical studies. Another medical school professor asked if the slow progress was because of shortage of funds. Friedmann said no, not really – it’s just a difficult process. He thinks that more successes are around the corner. He had a good slide at the end of his presentation showing Athena’s birth, fully formed, from Zeus’s head as a reminder that advances won’t happen all at once but will be more incremental. He was frank in conceding scientists’ responsibility for generating this sort of expectation.

I wonder if gene therapy won’t end up getting superseded by prenatal genetic screening – which I imagine will be much simpler and more cost-effective and more appealing to prospective parents (even as everyone wrings their hands over designer babies.) An ounce of prevention… I guess it will depend in part on how fast the respective technologies develop. That's my operating paradigm in any case: technology will dictate the ethics or values on this matter, not the other way around.