Mapping the stem cell ecosystem: an interview with Paul Knoepfler

Written by RegMedNet

In this interview, Paul Knoepfler (UC Davis) discusses his recent Regenerative Medicine perspective on mapping and driving the stem cell ecosystem.

Read Paul Knoepfler’s paper here>>

Please introduce yourself and your institution

I’m a professor of Cell Biology and Human Anatomy at UC Davis School of Medicine (CA, USA) where I’ve been for a dozen years. My lab primarily works on the relationship between stem cells and cancer toward two big picture goals: safer regenerative medicine therapies and new, more effective treatments for what are now largely incurable childhood brain tumors.

I’ve also run The Niche stem cell blog for 8 years and do some policy research when I have time, mainly on unproven stem cell clinics. In addition, I’ve written two books including one on stem cells (Stem Cells: An Insider’s Guide) and more recently another (GMO Sapiens) on the potential use of CRISPR In humans. 

What inspired you to write this opinion?

I got interested in stem cells first as a graduate student at UCSD and then as a postdoc at The Fred Hutch Cancer Center in Seattle. Along the way in my earlier career, and now as a faculty member, it’s been interesting to watch the stem cell field evolve. As it has become dramatically more complex, it felt to me like not too many people were looking at the big picture of the entire area of stem cells.

A couple years ago I heard someone use the word “ecosystem” at a research talk and although the talk was somewhat focused elsewhere, it kind of clicked in my mind that we could view the entire stem cell arena as an ecosystem. The more I thought about it, the more I realized that this kind of model in a sense of the stem cell arena could be useful and might be helpful to the diverse group of people and organizations in the stem cell ecosystem. We could also track the ecosystem over time and I thought to myself that we could try to change its evolution.

Why has the stem cell and regenerative medicine ecosystem become so complicated?

On one level, the sharp increase in complexity of the ecosystem reflects some positive changes. For instance, today we have far more patient involvement and advocacy. We have exciting research ongoing on many different kinds of stem cells, whereas decades back it was almost entirely focused on hematopoietic stem cells. I believe the more types of stem cells that we work on, the more likely we are to make new discoveries and help patients. The ecosystem has more funders, more biotech companies and has led to more clinical trials.

On the other hand, a substantial part of the complexity of today’s stem cell ecosystem comes from what I see as negative sources including primarily the unproven stem cell clinic industry. The clinics collectively are at the center of what I see as a “constellation” of businesses primarily devoted to generating profit at the expense of patients. There is little evidence that adipose, bone marrow, and amniotic stem cells can be used safely and effectively for the diverse array of conditions marketed by for-profit clinics.

At the same time, we now know a lot more about how some patients have been harmed at clinics. It’s encouraging to see the FDA under Scott Gottlieb taking more action and now the FTC has taken some steps as well, but given the magnitude of the clinic constellation, the FDA and other agencies such as state medical boards, the FTC, and law enforcement agencies need to do much more.

How does the complexity of the stem cell ecosystem affect development and adoption of stem cell and regenerative medicines?

The complexity of the ecosystem in theory may allow for the development of a wider array of proven new therapies, but it’s such a jungle right now that the complexity can confuse things and potentially slow things down. Precious resources might be wasted as well. The unproven clinic part always muddies the waters and risks damaging the public’s perceptions of stem cell research and regenerative medicine. 

How could the number and identify of stakeholders evolve in the future?

One hopeful perspective is that within 5 years, and more definitely within 10 years, one or more new stem cell-based therapies will be approved and have positive impact in ways that both help patients and energize the field. This could shift the ecosystem to having a more prominent clinical pipeline reinforced by success.

Unfortunately, it’s also possible that the number of stem cell clinics just in the U.S. alone could continue to expand to 1,000 locations or more, negatively influencing the overall ecosystem even more. If that becomes the reality I would predict a lot more harm to patients too. This could potentially in turn lead to reduced funding of stem cell research including clinical trials. However, as bleak as this sounds, it’s not inevitable and today many more parties and organizations are working to counter the clinics.

Even if stem cell clinic numbers don’t increase but just stay the same in the next one to two years it’s still in a sense a victory and would likely point toward that industry shrinking in the future after that. 

Read Paul Knoepfler’s paper here>>