You might have seen a headline like one of these: Ohio man appears to be first in the world to be cured of Type 1 diabetes or this: A Cure for Type 1 Diabetes? For One Man, It Seems to Have Worked. That last one is from The New York Times.
Don't worry. You (or your kid) is not missing out on a cure.
So what did happen? Vertex corporation issued a press release hyping the fact that the first person who got a beta cell transplant as part of their clinical trial uses much less injected insulin in the 5 months since the transplant, than he used before the transplant. But there are three important facts to put this into context:
- The patient still takes insulin. His insulin requirements have dropped 91%, which is a huge drop, and I don't want to belittle it, but he is still injecting insulin; just much less.
- The patient now must take immune suppressive drugs for the rest of his life. Currently, he is taking a combination of 10 pills a day. These drugs have serious side effects (including risk of death). The very best that can be said of this treatment, as used on this patient, is that he traded high insulin therapy for low insulin therapy and immune suppression. Some people might consider this an improvement, but I would not.
- These results have been seen before, many times over the last 10 years. In fact, a JDCA report found that over 75% of people receiving Edmonton protocol transplants (first available in 2001) did not need to inject insulin for a year after the transplant:
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| Data from the Clinical Islet Transplantation Network Annual Report 2017 Graphic by the Juvenile Diabetes Cure Alliance | |
In short, the result which generated all the recent buzz is actually worse than transplant results over the last 20 years. All of these transplant "cures" (to date) require immune suppression and (in my opinion) are not cures at all.
Also, notice that by year five, 85% of the people who got a transplant were back to injecting insulin. These people still must take the immune suppression drugs for the rest of their life, even if transplant is not working any more.
Now, as a research milestone, this is solid. Vertex should be happy with these results, because it is the first time their product has been tested in a person, and they specifically started with a low dose. So the fact that the results are worse than other transplant results should be expected. As the research progresses, they will be able to use larger doses, and (hopefully) get better results. From a corporate point of view, this is very good news. If everyone in their phase-I trial has results this good, it will surely move to a phase-II trial.
Also, I don't want to minimize the impact this has had on this one person's life. His insulin usage has gone from 30-40 units a day down to about 3 units a day. His A1c has gone from 8.6% to 7.2%. Perhaps most importantly, his T1D was previously "brittle". He had seizures and ambulances had to be called for him many times. Now he reports much better control with vastly fewer seizures and emergencies in general. These are all important benefits from the transplant. There is no doubt that he is very happy with the outcome, and I'm sure there are others who would be very happy with these risks and results.
Additional Reading
Overview of islet transplantation with data on success rates and duration:
- http://thejdca.org/ict-survey-2021
- https://www.jdrf.org/blog/2021/11/29/progress-toward-cures-thoughts-from-jdrf-ceo-aaron-kowalski/
- http://thejdca.org/islet-transplanation-overview
Press Release from Vertex:
- https://news.vrtx.com/press-release/vertex-announces-positive-day-90-data-first-patient-phase-12-clinical-trial-dosed-vx
Just some of the news articles which reported on this "cure":
- https://spectrumnews1.com/oh/columbus/news/2021/12/09/ohio-man-cured-of-type-1-diabetes--at-least-temporarily-
- https://www.nytimes.com/2021/11/27/health/diabetes-cure-stem-cells.html
