This is the second in a series of videos I’m doing where I answer questions about the stem cell field. You can see the Episode 1 video here.
Today I tackle more questions on the stem cell field. If you have more stem cell questions, let me know and I’ll try to answer them next time.
One reader asked me about stem cells for stroke and about one company, Athersys, in particular, but I haven’t had time to tackle that one in such a way as to do it justice so stay tuned on that good question.
I realized after the fact that regarding stem cell epigenetics I should have said broadly that this is a process of regulation of gene expression.
Hi Paul. Thanks for being accessible to answer questions from the general public. It would be great to hear you go into what some of the top challenges are for scientists to the goal of reaching certain milestones. I think I understand that the work is complicated in the way that researchers need to have a map of say cancer x’s genes. I suppose it’s a matter of then knowing what gene to delete, or modify? My obvious “real” question is when are we going to see the types of breakthroughs that make the general public take notice? I need you, or someone to dummy it down a bit. I am slowly, but surely learning the technical jargon, but boy…as a layperson trying to be an advocate, I, like the folks I am trying to get excited about this don’t really need or want to know what epigenetic chromatin looping engineering or whatever is. Or maybe we do? What, for a deeper understanding with the goals of advocating should I be studying? The sea of information is just that. I am trying to essentially do what you are doing, but in a way that is comprehensive, easy to understand, and highlights the results in order to convince. So at this point I can talk people’s ears off regarding the what is it questions. I’ve practically watched every dumb cartoon presentation available…arg! I can explain what the future looks like with all this in place, I can push down the knee jerk population crisis concern, etc. After all my study I still can’t tell people with certainty this IS going to happen. This IS a certain cure for this or that. We HAVE worked out the bugs with this technique or that, or we are close to it. Have we solved the delivery problem of Cas9 missing it’s target, for example? It seems Liz Parrish isn’t worried about that. Again, what are the hurdles we need to get over? How are researchers trying to overcome those problems and bottom line…How close do you think they are to nightly news worthy special reports? Thank you for your work and sorry for the length of my letter.
Hi Jon,
You ask great questions. I’ll try to address some here and some in the next video Q&A.
When will the public see a “wow, this is a game changer from stem cells” kind of stem cell development? I do think this kind of moment is coming, but most likely it’s going to be longer than we all hope. So many trials are ongoing that I think a 10 year time line overall for some “Eureka!” moment is not unrealistic. It could be sooner. It could be on vision. It might be on diabetes. Maybe something else. It’s coming.
The biggest hurdles to get over to one or more such discoveries whether it is stem cells or CRISPR? One of the toughest challenges is actually often overlooked — it’s actually extremely difficult to develop a therapy that actually is both effective and is safe. Most investigational therapies fail not because of the FDA, but because they lack the chops to start with. Of course we can’t always know in advance what’s going to end up being the real deal. That’s the tough part of science.
Another challenge is lack of funding these days.
For some things I would say we already had huge milestones. IPS cells already changed the world of biology and their disease modeling and drug testing potential is enormous. It’s a longer road to them being the proven basis for safe and effective cell therapies. Same kind of deal with CRISPR, which has already changed the world of biology. Clinical applications are coming, but to my knowledge there’s only 1 active CRISPR clinical trial. I cover the key CRISPR clinical challenges in what I hope is a very approachable way in my GMO Sapiens book better than I can here.
I’ll say more in the next video.
Thanks
Paul
My hope of all hopes is for scientists to have something to present to the public that will spur on faster research provided the monetary support. It’s sad and frustrating that money is such a big factor slowing the pace of the needed research. Thanks again. I look forward to your next blog.