Now six years into its existence, the induced pluripotent stem (iPS) cell field is no longer in its infancy to put it mildly but what will it take to get IPS cells to patients?
Perhaps one might say that cutting edge new scientific fields advance in dog years.
In that way of thinking, the iPS cell field is really middle aged (42 years old).
Yet are iPS cells any closer to being used in actual patients to realize the dream of patient specific therapies?
I think the answer is “yes”, but even so we have a long way to go.
In a new review article, Key anticipated regulatory issues for clinical use of human induced pluripotent stem cells, in the journal Regenerative Medicine, I discuss some of the key issues from my perspective. I’m not citing my own paper for shameless self-promotion, but rather because I worked hard there to articulate the critical issues and I think you might find it an interesting and useful resource.
I wrote it after lengthy discussions with many experts in the field and the FDA, although they preferred not to be quoted.
The key issues in my mind for clinical use of iPS cells include the lessons from the regulatory pathways of human ES cells that established a ground work, potential unique issues for iPS cells such as genetic modification, possible future requirements for genomic QC, and concerns about iPS cell stability in bulk culture.
Despite these challenges, I’m optimistic that iPS cells can “jump the dish”, as I said in a previous review, to help patients in the future, perhaps in 10 years. Cost and timing are additional important issues that may make batch-prepared ES cell-based therapies more appropriate for certain ailments. I can’t wait to see what the future brings in this exciting, not quite so new field.
Love ‘jump the dish’ illustration. What would you see as ideal first use outside the dish?
I was diagnosed with Multiple Sclerosis in 2006 at the age of 48 with secondary progressive. The medications on the market do nothing to stop my progression so I have been reading your excellent blog and anything else on stem cells, hoping and praying for some sort of help. When I was first diagnosed I read it may be 10 to 12 years before stem cells would be available.Your comment of 10 years more makes me sad. I understand when people go to other countries with their life savings for treatment. I am patiently waitng!!! Lets get going!
Mary,
Thank you for your comment and for sharing your story.
I was just talking with a reporter about the timeline for treatments and my frustration with it, but at the same time the first rule of medicine is “do no harm”. I know the sense of urgency, but cutting edge new medical treatments that are largely untested are very risky. There is a reason for testing things in animals first, for example, even though it takes years.
From all that I know, I am deeply concerned that the stem cell treatments now being offered, particularly those outside the U.S., have a lot more potential to hurt patients than to help them. Even many offered in the U.S. are not tested and have not been FDA approved.
I understand your frustration with the slowness. The field is working hard. Beyond finding new ways to speed up the FDA approval process, the only other thing I can think of to step on the gas further to speed things up even more is to somehow get more funding for research, particularly research on new treatments.
Unfortunately things are going in the opposite direction! Not just for stem cells, but more broadly American investment in health-related research (i.e. NIH funding) has really been stagnant now for many years and I think we are all going to pay a price for that in slower development of new treatments.
I believe that stem cells will transform medicine, but the question is how long will that take?
Paul, thanks for this post. I agree research needs to move faster in order to get needed therapies to all patients.
The proposal I sent you regarding a collaborative research project addresses these issues of pace of progress, standardizations, access and a number of other concerns mentioned in the previous comments. A multi-state Texas and California collaboration would be awesome.