The best talk today was the keynote address by Dr. George Daley focused mostly on iPS cells.
Dr. Daley is one of the world’s experts on iPS cells and pluripotent stem cells more generally.
He covered many interesting elements of the iPS cell field including the genetic mutations that seem to occur during the reprogramming procedure. Epigenomic changes. Ways to integrate gene therapy and cell therapy approaches that are very exciting.
However, he also asked a cautionary question:
How long will it take to bring stem cell therapy to the clinic? Not just the exciting early trials of ACT and Geron, but fully vetted safe and effective treatments.
His answer surprised me.
Decades.
Not a decade, but decades plural.
I think George Daley is a realist and I hope he is being too cautionary, but maybe he is right with this answer.
He said if the history of gene therapy is any lesson then we have a long road ahead. However, unlike gene therapy which in essence started from scratch, we already have effective cell therapies such as bone marrow transplantation that have been around for decades. This is a real, very powerful cell therapy. This foundation is likely to prove very valuable. There are millions of people who need help faster than decades.
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Whats fully vetted and safe?
Many drugs on the market are not absent harmful effects…why all the caution with stem cells?
I mean if they accomplish an objective (helping paralyzed walk,regain bodily functions) with the risk of complications down the line…so be it…I think the problem is this technology is so disruptive to the status quo that they fear its wake.
Lanza is like the Howard Hughes of stem cells…the guy takes risks and pushes the envelope.
MStudent,
That’s an excellent comment.
I love the idea of stages for the development of new therapies. I just might have to “borrow” that idea for a post of my own.
Besides, gene therapy was beset with early fiascos which resulted in getting a bad reputation among the public (and investors, I assume). In my opinion, this was unfair to some extent. As you have said in the past, all therapies (experimental or otherwise) carry risks, and what must be done about that is determine what those risks are, and minimize them as much as possible. Besides, the most important incident behind the scare -Jesse Gelsinger’s death- had many elements which corresponded more closely with human error than with the dangers of gene therapy, I think. Nonetheless, it hit the field hard. But nonetheless there have been reports of gene therapy clinical trials achieving very impressive things. I don’t think it’s record is that bad, all things considered. It is just being annoyingly slow to get to the clinic.
Last year I read an interview with a former teacher, in a newspaper, which had some lines which are significant on this topic, I think. “All new therapies pass through an initial enthusiasm stage because of the novelty, a second phase of partial disenchantment because nothing is easy, and a third phase of consolidation of the technique after having surpassed the problems and difficulties via a solid research work. Gene therapy is between the second and the third”. Maybe the same could be said of cell therapy?