What The Headline About A Vaccine Against Type 1 Really Means

The headlines this morning are certainly encouraging: "Vaccine Promising Against Type 1 Diabetes." Sometimes they're using stronger language, such as "Is Vaccine a Cure For Type 1 Diabetes?"

But this is the modern media at work. It is especially common in cancer reporting, where we see words like "promising" and "potential" in headlines with extraordinary frequency, yet over the last forty years it's hard to assert, according to the data, that too many of those "promising" or "potential" breakthroughs ever amounted to anything. In fact, more often than not, these headlines are from pre-clinical trials, meaning they haven't yet investigated the treatment in living human beings.

It's not that the latest headline about a vaccine used against type 1 diabetes shouldn't give us all a little hope or encouragement that it may pan out. Rather, instead of encouraging passive hope, it should encourage action—namely, participation in a clinical trial.

The Study

The study, "Proof-of-concept, randomized, controlled clinical trial of Bacillus-Calmette-Guerin for treatment of long-term type 1 diabetes" appeared in the journal PLoS One and the most important term in the title is the first one: proof-of-concept.

Authored by researchers from Harvard, the study is a double-blind, placebo-controlled trial of adults with type1 diabetes who had had the disease for an average of 15.3 years. It involved a grand total of six subjects: meaning that six subjects were randomized to get either the vaccine or a placebo. So, with just three people receiving the vaccine, it is extremely difficult to draw any grand conclusions from a sample size of three.

The vaccine in question is a generic Bacillus Calmette-Guerin (BCG) vaccine. The researchers applied the immunotherapy to stimulate tumor necrosis factor (TNF). The TNF kills the autoimmune T lymphocytes that destroy type-1 diabetes causing, insulin-secreting pancreatic beta cells while apparently managing to leave healthy T-cells alone. It also improved insulin sensitivity (according to measured C-peptide levels).

In two of the three patients that received the vaccine—as well as one control patient who received a placebo but during the trial developed acute Epstein-Barr virus infection (a known TNF inducer)— tests "exclusively showed increases in dead insulin-autoreactive T cells … C-peptide levels (pmol/L) significantly rose transiently in two BCG-treated subjects" and to a lesser extent, in the EBV-infected subject compared to a group of diabetics the researchers used as reference subjects.

Study Conclusions

Here's the study's conclusion:

"We conclude that BCG treatment or EBV infection transiently modified the autoimmunity that underlies type 1 diabetes by stimulating the host innate immune response. This suggests that BCG or other stimulators of host innate immunity may have value in the treatment of long-term diabetes."

They also wrote that trials in the future should look into possibly higher doses of BCG or more frequent administration of the vaccine.

Action

Typically, studies that conclude with something to the effect of "further studies are needed to confirm these findings" are horribly frustrating. We can all say that about anything. It reads like a cop-out.

But in a proof-of-principle trial such as this one, these results should encourage people to participate in those "further studies." As it is, clinical trial participation among adults in the US is frighteningly low. It is also replete with mythology and misinformation.

Check out the Facts & Figures offered by the Center for Information & Study on Clinical Research Participation (CISCRP).

While participation doesn't automatically equal progress or breakthroughs, this much is true: Without participation, there is no progress and there are no breakthroughs.