Weekly reads: stem cell therapy for eyes research, DMD, cheating death, cancer sensors

Many avenues are being pursued to get stem cell therapy to the clinic for eye conditions. These different approaches employ a variety of stem cell types. In some cases, such as with iPS cells, the stem cells themselves are not transplanted. Instead, the iPS cells are used to make differentiated eye cells, such as RPE cells, which are then introduced.

Stem cell therapy for eyes research

stem cell therapy, stem cell therapy for eyes
Stem cells being validated prior to use in diseased eyes. Fig. 1E. Jurkunas, et al. Science Advances. “(E) Confocal microscopy showing cell morphology at low, mid, and high density using vinculin (green) and actin F (red) staining during P1 expansion. The nuclei were stained with 4′,6-diamidino-2-phenylindole (DAPI) (blue).”

In other cases stem cells themselves are directly transplanted. An interesting question arises. Should a cell therapy that does not directly use actual stem cells but instead uses differentiated cells made from stem cells still be called a “stem cell therapy.”

I think so. What do you think?

Of course, another issue is when non-stem cells or mixed populations of cells are called “stem cells”. This happens pretty regularly with MSCs even though many researchers are rightly being more cautious and calling them “mesenchymal stem/stromal cells” to reflect their mixed populations or even just calling them, “mesenchymal cells”.

The new stem cell therapy clinical trial article

Using stem cells, doctors restored vision to people with devastating eye injuries, NBC News.  As is often the case, the headline here is a problem in that it implies things are much further along than they really are, but there is some concrete hope from this work.

Here’s the original research article on a small Phase 1 study in Science Advances: Cultivated autologous limbal epithelial cell (CALEC) transplantation: Development of manufacturing process and clinical evaluation of feasibility and safety. 

The team is working on developing a cell therapy treatment for a common cause of vision loss, limbal stem cell (LSC) deficiency, starting with autologous limbal epithelial cells. It’s too soon to know from this small safety study whether limbal cell therapy might be effective for this cause of blindness.

Participants have not shown any major adverse events so that’s a big first step. Also, there were hints of possible efficacy in some participants. However, from many years in the cell therapy field I’ve seen so many similar things pop up in early Phase 1 studies only to not hold up later. So, as always, one needs to be somewhat cautious and evaluate more data as they are released.

DMD gene therapy
“The Rodriguez family and Craig McDonald on Andrew’s infusion day at UC Davis Medical Center” UC Davis Photo.

Other reads

UC Davis first in the state to offer life-changing therapy for Duchenne Muscular Dystrophy, UC Davis. This is great news about clinical gene therapy work from my colleagues.

Engineered bacteria detect tumor DNA, Science. Cancer detection, especially very early on, is hot right now. This approach is quite interesting.

For This Venture Capitalist, Research on Aging Is Personal; ‘Bob Has a Big Fear of Death’, WSJ. Some of the measures taken here remind me of the extreme health regimens of Bryan Johnson and David SinclairThis WSJ piece has a lot about Altos Labs.

Intellia plays down concerns about accidental germline transmission in CRISPR trial, STAT+Just to be clear, because the STAT headline isn’t, no accidental germline transmission actually appears to have happened in human trials. It was evident in some animals in earlier studies.

Stem cell patch for treating macular degeneration to enter phase 2b clinical trial, News Medical Life Sciences. Some interesting work from USC. Note that this concept of a stem cell patch should not be confused with stem cell patches that are marketed to increase one’s endogenous stem cells.

Cell therapy trial for COVID

Intravenous Administration of Umbilical Cord-Derived Mesenchymal Stem Cells (UC-MSC) for Acute Respiratory Distress Syndrome Due to COVID-19 Infection, Cureus.  This is another very early study of cell therapy for COVID. I’d say it’s largely inconclusive in part because of its small size and design. The authors rightly point out the limitations of their study: “However, our study has several limitations. These include a small sample size, lack of a control group, potential selection bias due to single-site recruitment, and the presence of multiple comorbidities and medications, which complicates interpretation. Additionally, a short follow-up period, possible subjectivity in CT scans interpretation, lack of blinding, and unaddressed confounding factors also limit the study’s reliability. Moreover, we largely relied on short-term clinical improvement, which may not necessarily correspond to full recovery or long-term survival.”

See my recent broader update on cell therapy for COVID.

By the way, related to the early discussion about what we call “stem cell therapy”, it’s not clear to me from this article that these MSCs are actual stem cells.

1 thought on “Weekly reads: stem cell therapy for eyes research, DMD, cheating death, cancer sensors”

  1. I had a vision exam on line with Dr. Lewis and have followed protocol he prescribed with disappointing. I would like to move forward with stem cell therapy being offered.
    I would appreciate more information.

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