Landmark Drug Delays Onset of Type 1

Teplizumab is an anti-CD3 drug that’s getting a “breakthrough” indication from the FDA to treat people at risk for getting type 1 diabetes.  This is nothing short of a big deal, so I wanted to spend some time trying to break this down and what it could mean for all of us.

So let’s start with the facts.

This drug works to try to dampen the autoimmune attack on the beta cells.  The study, which was published in the New England Journal, studied 76 people (55 kids, 21 adults) who were at high risk for getting type 1 as shown by having 2 or more autoantibodies (GAD, insulin, ICA, IA-2, or ZnT8) AND had some kind of dysglycemia.  That means they had elevated fasting or postprandial glucose levels.  So these people are essentially guaranteed to get type 1 at some point.  In other words, this was a very high risk population.  The group was then either treated with Teplizumab via an IV infusion once per day for 14 days or saline placebo for 14 days.  The drug was fairly well tolerated but people did have decreases in white blood cell counts and a rash was fairly common as well.

The bottom line, however, is that getting the drug delayed the onset of type 1 diabetes by about 2 years.  So it didn’t stop the process in its entirety, but hey, two years is two years.

Some other important caveats:

1. People had to be at least 8 years old
2. The majority of these people were found to be at risk because they had a sibling with T1D (as opposed to some other relative)
3. This is a very small cohort of patients (only 76 people)
4. This drug has already been tested in people who had recent onset of T1D and it did not really work. So it may work “better” as prevention than “treatment”

For anyone interested, a link to the study can be found here.

As far as I can tell, I don’t see the specifics of who this drug will be indicated for, but my guess is that it will follow along with the trial criteria.  Namely people will have to be > 8 years old, have multiple antibodies positive, and probably have abnormal glucose levels.  But we will see.

So what is really interesting to me, is what this will do for screening people.

Currently getting screened to be at risk is something conducted for research purposes only, but now that we have an approved drug, we will likely see this slowly move into the “mainstream”.  Meaning, it won’t be long before we are seeing patients to measure their antibodies and counsel them on therapy, prescribing this medication, etc.  It is also likely to give people a more compelling reason to get their family members tested.  You hear a lot from people (including myself) saying,  “I don’t know about screening my kids because there isn’t anything we can do about it.”  Well, now we might have that something.  So talking about getting family members tested might slowly move away from just cocktail party conversation to official recommendations and a public health movement.

I’m cautiously optimistic.

This is a small study, and I hope the results are “real”.  They are going to do another study in very recently diagnosed patients to see if we can use it at diagnosis and get similar results.  So stay tuned on that. In the meantime, having the first ever drug approved to delay the onset of type 1 diabetes is truly a landmark and should be one that we all celebrate.  NO AUTOIMMUNE DISEASE goes from nothing to a cure.  These small steps are meaningful and show that we are on the right track.

So don’t throw away your pump just yet, but maybe do a couple of fist pumps and like a soft “woo hoo!” while at work.