David Brafman: Engineering Human Pluripotent Stem Cells for the Study and Treatment of Neurological Disorders and Diseases: History, Opportunities, and Challenges

Oh, good — a tutorial about stem cells.

What makes a cell a stem cell? One, it can make more of itself, and two, it can differentiate into other kinds of cells.

What are the basic types of stem cell? There are multipotent, which can only turn into a few other kinds of cells, and the other is totipotent, which can turn into anything.

That’s the basic info.

Now we get a quick history . . . 1860s: Ernest Haeckel came up with the ideal. In 1890s, a little progress . . . then nothing until 1963, when we got the beginnings of bone marrow transplants. Idea-to-therapy took about 80 years.

There are lots of stem cells in our bodies — the brain has neural stem cells, there are lung progenitor cells, and satellite cells . . . in humans, what we really want is pluripotent stem cells (that can become anything at all), which have two sources. One is the blastocyst, which makes human embryonic stem cells. The other what they call induced pluripotent stem cells, which work just like embryonic stem cells but don’t come from blastocysts — they come from skin cells that have been re-engineered back to the “origin state.”

The source of blastocysts is leftover embryos from in vitro fertilization. The process of fertilizing a woman’s eggs to aid a couple in getting pregnant almost always results in more viable embryos than the couple is able to bring into the world . . . which means that many of those embryos end up being destroyed. When couples choose to donate them to science instead of having them disposed of, the blastocysts become the source of stem cells.

The other option for pluripotent stem cells is to use a process called induced pluripotency. It’s literally turning the clock back. Every cell in every human begins as pluripotent cell, and it goes through a series of changes to get sorted into lung, heart, blood, skin, whatever. Inducing pluripotency means walking it back down that path, all the way to the original stem cell, where it’s just like the ones in the blastocyst.

(There’s also something called direct reprogramming, which takes one kind of adult cell and turns it directly into another kind of adult cell. We heard about that this morning from Jan-Eric Ahlfors — he’s going to speak again tomorrow about his data gathered after using these cells clinically.)

In David’s lab, he’s been working with induced pluripotent cells in dishes to see if it might be possible to use them to help people with neurodegenerative diseases and conditions. Like schizophrenia.

Slide title: Stem Cell Therapies of Snake Oil?

There are people who are preying on the unsuspecting public, promising that they have a stem cell therapy that can cure anything and everything. This is happening not just overseas but here in the USA. One thing the clinics are selling is based on taking fat, processing it, and injecting it into patients. The number of clinics keeps rising; they’re not selling FDA-approved products; they’re charging between $600 and $20,000 for their “treatments.”

How do you know when you’re looking at snake oil? Claims will be something like, it works for every kind of illness, or the process will involve minimal manipulation to get the cells into shape for you. There are cases all over the world and in the USA of people dying after these treatments. But don’t these companies have lots of testimonials from former patients? They do, but they don’t have any followup from independent reviewers.

Can’t the government stop them? It’s difficult because they’re popping up quickly. So how can a person tell what’s legit and what’s not? Look for this:

  • Claims based on patient testimonials
  • Same cells for multiple diseases
  • Source of the cells not clearly documented
  • Treatment not documented with protocol
  • Claims of “no risk”
  • High cost

Look at A closer look at stem cells for more info.


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