On the other side of an experiment
I’m three weeks into having type 1 diabetes (T1D) and very grateful that it’s 2017. If it were 1919, I’d have a painful year and a half to live while slowly dying from starvation. That’s a scary thought.
Today, my endo says I have a normal life expectancy and she wouldn’t expect me to ever need anything amputated. That’s a relief. She also says that within my life time there will be a cure. “I’m a pessimist, Jo. And even I think there will be a cure in the next ten years,” is how she phrased it last week.
Even without a cure, treatment with insulin has turned my meager year and a half life expectancy into one, that with proper management, should be just as long as the average American. That is all thanks to Frederick Banting. Banting discovered insulin in 1921, which led to the treatment of the first patient, Leonard Thompson, in 1922. His first insulin treatment took place at New York Presbyterian Hospital, across the street from where I am going for diabetes management. Thompson lived to be 27, which sounds disappointingly young, but the 13 years he lived after being diagnosed were seen as victories for the field of diabetes research, a marked increase from a year and a half. Insulin was mass produced by 1923 and in 2017 the same company, Eli Lilly, still mass produces insulin for people with T1D. Insulin was the miracle drug that turned a death sentence into a long term management issue overnight.
Miracle treatments are nice but what would be nicer is a miracle cure. It’s a strange sensation to be told in your mid-twenties that you have something that will never go away. Usually when we get sick we get used to being sick for a couple days, maybe a week. Being told you have something that lasts the rest of your life is something the human brain has difficulty comprehending. “Welcome to having a chronic condition,” is what Dr. Goland told me when I first sat down in her office. The idea of “chronic” is like the idea of marriage, global warming, and debt accumulation – we aren’t wired to think that long term. This might be why I started reading through ~200 pages of clinical trial listings on the clinicaltrial.gov website. I wanted to find something immediate, a possible cure I could try on the weeks-timescale, not the years-timescale.
That’s how I landed in Dr. Goland’s office and why on Thursday I’ll get screened for a clinical trial that may be a step toward a cure. The study has two parts. The first part involves collecting a skin biopsy that is used to make stem cells to generate a mini-Jo pancreas in vitro. The mini-Jo pancreas will be kept in a huge library of other mini-people pancreases to test whether different drug candidates work to prevent beta cell loss. Medicine of the future is crazy. We can already make parts of a human subject from stem cells to test things on before giving to that subject. My mini-Jo pancreas can give my endo an indication of which future drugs may or may not work well for me. Personalized medicine in the pipeline.
The second part of the study is looking at a particular drug candidate, a compound called TUDCA, to see if it can stop beta cell decline, and potentially regenerate beta cells, in people who are newly diagnosed with type 1. Beta cells produce insulin — the idea is that stopping decline and stimulating growth of beta cells can help type 1 diabetics regain endogenous insulin production. Using mini-Jo pancreas to compare to the results of real-Jo can indicate whether test tube versions of organs are predictive of how the drug will work in vivo. If you’re interested, the trial has a video on the hypothesis they are testing. More studies should put out videos.
The most interesting part of participating in a clinical trial is the totally different perspective you gain on experimental design. When you are on the scientist-side, it is easy to see why we want balanced designs and an even number of controls and treated subjects. On the subject-side, I really hope I don’t end up being a control. Fifty percent of subjects will get a placebo, fifty percent will get treated. When a drug has no known side effects and is already used safely for other chronic conditions such as liver disease, I don’t want to be the person that swallows the sugar pill instead of the potential cure. TUDCA is available at vitamin stores and supplement shops where someone like me could go to have a 100 percent chance of taking the compound rather than only a 50 percent chance. The dilemma is that the clinical trial also includes free care with some of the top diabetes experts in the world, free diabetes medications (which are $$) and the possibility that you get informed about future treatments that your stem-cell-generated pancreas responds to. The risk is that if you don’t get treated with TUDCA during the first 100 days of having T1D, you may not have any beta cells left by the time the trial is over. Even if you wanted to try TUDCA after being a control for 18 months, you may have missed the window of time over which it would work. The risk of TUDCA having an adverse reaction only in people with T1D and not in the trials that have already been done for people with cirrhosis seems unlikely.
When you’re in an experiment, suddenly having controls seems less rational than when you’re designing one. Should I take my 50 percent chance and hope to get lucky, or should I order TUDCA on Amazon for a 100 percent chance control-free trial? Sounds like a medical ethics topic to debate.
