Top 10 stem cell stories of the year 2015

liver organoidWhat were the top 10 stem cell stories of the year?

What were the biggest news and science headlines in the stem cell field in 2015, whether good or bad?

Below are mine. What are your top 10 stem cell stories of the past year? Let us know in the comments.

  • Organoids explode. Stem cell-based mini-organs (organoids) have exploded in terms of impact in the last couple years. 2015 had many examples of these super cool human organs in a dish made from stem cells. Organoid technology has huge potential for teaching us about human organ development and function as well as disease pathogenesis and treatment. I recommend this interview by Alexey with organoid pioneer Madeline Lancaster for more on this exciting trend.
  • Clinical trials pile up. More good news. The number of real, promising stem cell clinical trials continues to build. This is fantastic and shows the continuing maturation of the stem cell/regeneration medicine/cell therapy fields. Think about where things were 5-10 years ago with stem cell clinical trials and you see how much progress has been made.
  • Sale of CDI and Ocata to Japanese pharma. Japan continues to show leadership in commercializing stem cells and regenerative medicine, and this in part manifested in 2015 with the big-time purchases of two American stem cell biotechs by Japanese pharma companies. Does the nation of a biotech company matter in this day and age? Are pharma acquisitions of small stem cell biotech good news or bad or some combo of the two? We can expect more corporate acquisitions in the stem cell arena in the coming year. Stay tuned for my top predictions for the stem cell field in 2016.
  • Stopping of IPS cell clinical study in Japan with broader regulatory switch there to allogeneic IPS cell focus. The pioneering macular degeneration clinical study using IPS cells in Japan was stopped this year and now a second IPS cell study (Parkinson’s Disease) there has been delayed. Regulatory changes now mandate that IPS cell clinical work be done with allogeneic cells. This change still remains somewhat of a mystery given the big potential of IPS cells on the patient-specific front using an autologous approach. We’ll hear a lot more about this in 2016. Another regulatory change in Japan is the move to allow charging of patients for clinical trial participation. I’m not a fan of this change to put it mildly.
  • CRISPR. Of course CRISPR is not specific to stem cells, but it opens new doors to understanding stem cell biology and development, particularly in the human research sphere. The genetic research on human stem cells that has opened up because of CRISPR is very exciting.
  • Human genetic modification on the table. Related to the above, gene editing of human pluripotent stem and germ cells has big ethical and social implications. The potential future production of genetically modified human beings via primordial germ cell or pluripotent stem cell editing raises big, thorny questions. Whether we are talking about genetic modification of somatic cells for therapy or production of actual designer babies, this topic was a hot one in 2015. For more, check out my new book, GMO Sapiens.
  • Stem cell clinics continued to bloom. From Internet searches to patient contact, the data out there are consistent with a continued rapid growth of stem cell clinics pitching at best unapproved stem cell interventions to at worst stem cell snake oil.
  • FDA action absent still on predatory clinics. For two years the FDA has practically speaking done nothing to address the exploding problem of stem cell clinics (see above) selling unapproved stem cell “treatments” to tens of thousands of patients in the US. We cannot forget about stem cell tourism, but the stem cell clinic problem in the US is massive and growing, and only made worse by an FDA that appears to either have mixed feelings on it or simply be moving in slow motion. Something’s got to give on this and I thought that would happen in 2015. Maybe it will in 2016.
  • Celebrities as advertising for stem cell clinics. Also in the dubious sphere we saw in 2015 an acceleration of celebs being the top drivers of patient traffic to stem cell clinics. Gordie Howe made the biggest news, but then there was Bart Starr and many others. For every famous person who puts themselves at risk or who is put at risk by their families looking for hope from stem cells, how many ordinary people follow suit and how many people will lose precious savings and potentially be hurt?
  • CIRM 2.0 picks up pace. Ending with some good news, the new incarnation of The California Institute for Regenerative Medicine (CIRM) as CIRM 2.0 (a name I admittedly enjoy saying as I coined the term) continued to build momentum in 2015. CIRM 2.0 has a distinctly clinical focus. The agency is picking up the pace on issuing RFAs and new funding. 

Stem Cell Person of Year Award 2015 Top 12 Finalists

The voters have “spoken” and below is the list of the top 12 vote getters from the larger pool of nominees for Stem Cell Person of the Year in 2015. These are some amazing people.

Look for more information, such as mini-bios, soon on some of the top finalists.

There were nearly 4,700 votes in total.

Now I have the tough task of picking from this dozen just one winner, who will receive the recognition as the top stem cell outside the box innovator of 2015 and of course the $2,000 prize.Stem Cell Person of the Year 2015 Award Nominees

 

Fantastic organoid voyage: views from inside a mini-organ

Fantastic VoyageDid you ever see the classic sci-fi movie, Fantastic Voyage?

In it, the heroes travel inside of the human body in a craft, observing all kinds of awesome biology in an up close and personal kind of way on route into the brain with the goal to do microsurgery of a sorts.

Even though this movie came out a year before I was born, I saw it later as a kid and found it captivating.

“What if we could travel inside the body or even inside organs?” I thought back then. It seemed like we could learn amazing things first hand.

A new technology called organoids or mini-organs kind of makes this possible today.

In fact, organoids are extra exciting because this technology allows us to make miniature version of organs and then do science on them. The organoids can be differentiated and grown, depending on the type you want to make, from pluripotent or adult stem cells or other sources of tissue.

Even though we cannot literally climb inside to take a look, we can do the next best thing using histology and advanced microscopy even on “living” organoids. In a great piece of science writing, Cassandra Willyard, talks us through all the various new kinds of human organoids: liver, kidney, brain, pancreas, stomach, lung, breast, and the list goes on including “guts” as per the quote from Hans Clevers at right from Willyard’s article. I love this quote.Hans Clevers

If we could shrink ourselves down and literally climb inside a human organoid, what would we see? What amazing things might we report on from this voyage?

In mini-brains we’d see neurons, synapses, glia, oligodendrocytes, and fiber tracts. We even might be witness to electrical activity in this mini-brain that represents actual rudimentary thought of a kind. Imagine seeing that “in person” from the inside.

Cerebral organoidsIn a mini-kidney or liver organoid, we might see all different kinds of cellular and tissue activities. If we dropped the equivalent of a micro bottle of vodka or tiny firecracker inside as a model of injury, we might see the organs kick into action to repair themselves.

In a breast organoid we might see milk production from the inside or the first signs of breast cancer formation. In a mini-lung, we could possibly see lung cancer germinate too or hike around inside airways such as bronchi, bronchioles, and alveoli. A bio-spelunker.

Exploring inside a heart organoid you could feel what it is like to be inside of something very similar to a beating heart. Would you like the rhythm and beat or feel like there’s a constant earthquake?

Inside the organoids in the lab you don’t have to worry about some nasty immune cell trying to knock you off either.

Some of the labs focusing on organoid research have discovered important things about normal human development and disease from this work. The Madeline_Lancasterresearchers include teams from the labs of Drs. Hans Clevers, Jürgen Knoblich, Melissa Little, Takanori Takebe, and a growing number of others. The late Yoshiki Sasai did pioneering work in this area as well.

The postdocs and other trainees in these labs have done work that has changed our visions of what is possible in stem and developmental biology in a dish. For instance, Dr. Madeline Lancaster’s work on mini-brains has opened a lot of minds to all that is possible in brain neuroscience in a dish (see images above of a mini-brain and of Dr. Lancaster at right).

An organoid is not just a model system either, but also might have therapeutic potential. Tissues grown in 3-D that take on the form and function of real human organs even if in miniature form could form the basis of innovative therapies in the future as well.

I would say that so far in 2015 organoids are the most exciting development and some have argued they are most important new thing in the stem cell and developmental biology fields.

For past posts on this blog highlighting organoids you can read here.

If I only had a brain: scientists grow human mini-brain-like structures from pluripotent stem cells

Cerebral organoidsIn a first for the field, scientists have used human pluripotent stem cells to grow miniature brain-like structures in a dish in a lab (see beautiful image of one of these “mini-brains” at left from the paper).

This exciting, pioneering feat, accomplished by a team from the Austrian Academy of Science in Vienna led by Dr. Juergen Knoblich (see his lab page here), has drawn great attention in the mainstream media such as here at CNN.

The paper, which Lancaster, et al., was published in Nature and is entitled:

Cerebral organoids model human brain development and microcephaly

Below is a picture of the Knoblich lab. I like to show the people who did the science and not just the sciency images. I’m not sure which is Dr. Madeline Lancaster, who deserves congratulations for her outstanding first author paper.Knoblich lab

I believe that this paper is a major advance as it opens the door both to a better understanding of human fetal brain development and a new way to model disease states when brain development goes awry.

The team used this new technology to study one such case, a condition called microcephaly, where the human brain does not grow normally, leaving adults with brains that are most often too small to function normally. Interestingly, as a side note, some patients with microcephaly have entirely normal cognitive and behavioral function, a phenomenon that scientists do not entirely understand.

The cerebral organoids contained normal markers of many human brain cell types and the neurons produced exhibited evidence of electrical function.

It is important to stress that the team did not make actual human brains, miniature or otherwise. The cerebral organoids–about the size of a pea and grown from hESC or hIPSCs–only contained some brain regions and these were primitive in nature resembling fetal brain structures. Most notably, these brain-like structures lacked a cerebellum and almost entirely lacked a hippocampus.

Still, nothing is perfect and to me this seems like an impressive start to what is certainly going to be an important new line of stem cell research. It remains perhaps unlikely that this technology in the future will provide material for actual transplant of newly grown brain regions in human patients, but I wouldn’t rule anything out in the coming decades when it comes to applications of stem cell technology.