If you've been reading news recently you might have seen headlines like these:
Stem cells reverse woman’s diabetes — a world first
Stem Cell treatment reverses Type 1 Diabetes!
Chinese scientists have put a young woman’s type 1 diabetes into full remission using stem cell treatment.
And even if you didn't your friends and relatives might have seen them and asked you about them.
(And that first one is from the journal Nature, which is a major scientific publication.)
Unfortunately, those headlines are basically hype. There is some news, and it is good news, showing important progress, but it is no where near as good as the headlines make it sound.
What Was Reported
The researchers took some adult stem cells from a person with type 1 diabetes, and treated those stem cells with a specific recipe of drugs, which caused them to change into beta cells. They then implanted those cells back in the person who donated them. The results were spectacular. After about 2 months the person stopped injecting insulin, and then was followed for another 10 months without needing any injected insulin.
The woman was not your average person with T1D. She had been diagnosed 11 years prior, so was well established. However, she had previously had two liver transplants and one pancreas transplant (more on that below). At the end of a year, her A1c was 5% and her blood glucose time-in-range was 96%, both great numbers.
Not A Cure
Before getting this transplant, this person had already had several transplants, and was therefore on immunosuppressive drugs and would need to stay on those drugs for the rest of her life. Therefore, by my definition, she was not cured but rather traded one treatment (insulin for T1D) for another treatment (immune suppression for a transplant). I don't consider this a cure. Some people may prefer one drug regimen over another, but it is not clear to me that one is generally better than the other. My understanding is that people who have whole life immune suppression generally have shorter life spans than people with T1D.
But in any case, I don't consider this a cure. Of course, you may, and if you do, then you should look into existing transplantation surgeries, because there are some available now that have similar results to what is seen here.
Shows Progress
The big headline "no insulin injections for almost a year" is misleading because those results have been seen previously in transplants when whole life immune suppression has been used. The JDCA has published two great overviews in 2016 and 2022, which you can read here:
- https://www.thejdca.org/publications/report-library/archived-reports/2016-reports/islet-cell-transplantation-update-new-phase-iii-study-results-show-insulin-independence-unchanged.html
- https://www.thejdca.org/publications/report-library/archived-reports/2022-reports/pioneering-islet-cell-transplant-team-publishes-20-years-of-data.html
The bottom line is that no insulin injections for a year occurs in half the cases, and for two years in over 40% of the cases. So the headlines are hyping something we can already do. (If you are willing to suppress your immune system for the rest of your life.)
If fact, there is an FDA approved transplant protocol, where over 2/3s of the people did not have to inject insulin for more than a year, but it does require immune suppression:
However, the research reported here does show some specific progress, and some strong future possibilities. The key improvements seen here are:
1. More stem cell availability. Previous transplants have used beta cells from cadavers, pigs, occasionally live doners, and just recently from the patient themselves. This research sources beta cells from the patient, which means there will always be a strong supply. The treatment that they use to mature the stem cells into beta cells is new and unique and the researchers claim it is much better than previously available techniques. They think it gives them more control over the resultant beta cells and also is more effective. It is this technique that is the real progress.
2. Using a person's own stem cells. My memory is that this is not the first ever case where T1D transplantation used cells from the person being treated. However, this is a very recent and experimental technique. The success here is important. Most importantly, there is hope that these cells will not be automatically attacked by the person's immune system, because they are not foreign. This is a serious issue with other transplants. Because these cells are not foreign, this transplant may not require long term immune suppression. That would be a huge breakthrough.
Unfortunately, since this person is already using long term immune suppression, there is no way to know from this trial if this kind of immune suppression is needed or not.
Furthermore, even if this procedure becomes immune suppression free in the future, it is not clear what will happen to the new beta cells long term without it. Will the person's T1D immune system attack the new beta cells just like it attacked the original ones? We don't know. That is future research that must be done.
There are four hopeful possibilities here (lines of research which might prevent this attack). They are theories held by some researchers. The first two are active areas of research, with several research groups working on each one, and the second two are tested on occasion, but are less actively pursued:
- Several research groups are investigating encapsulation, so that the immune system cannot physically get to the new beta cells, which would protect them.
- Other groups are developing the process of converting stem cells to beta cells so that the new cells were invisible to the immune system and protected that way. The immune system tends to focus on very specific structures in the "skin" of the cell to identify what to attack. If those structures are missing, then maybe the immune system would ignore the new cells.
- The trigger of the autoimmune attack on beta cells might be time specific. It occurs at a specific time for a specific reason. So therefore, years later, when the new cells are transplanted, the trigger is no longer there and the cells will not be attacked.
- Finally, the immune attack on beta cells might be location specific. It occurs because of the situation in the pancreas specifically. Therefore beta cells not implanted in the pancreas won't trigger the immune response.
The researchers were waiting for results from the first person, but now that they have them, they will start up two more people. It is a little hard to tell, but I suspect these two people will also have prior transplants and therefore long term immune suppression.
The study is ongoing and they are recruiting more people with T1D in Tianjin, China. The study is described as "Phase 0", but I don't know what that means in China. It is similar to an American phase-1 "pilot" study: no control group, no blinding, three people who will be followed for two years (I think) after transplantation. Primary end point is A1c. Secondary end points are C-Peptide and insulin use.
Contact: Wang Shusen +86 136 1218 3907 shusen1976@126.com
Shen Zhongyang +86 138 0301 9898 zhongyangshen@vip.sina.com
More Information
This research was published in the journal Cell, which is a big name scientific journal:
Chinese Clinical Trial Registry: https://www.chictr.org.cn/showproj.html?proj=192835
WHO Clinical Trial Registry: https://trialsearch.who.int/Trial2.aspx?TrialID=ChiCTR2300072200
Joshua Levy
http://cureresearch4type1diabetes.blogspot.com
publicjoshualevy at gmail dot com