Today, we’re excited to bring you fresh updates straight from Dr. Faustman herself: Finally, her Phase II studies are set to begin in the coming months! Here’s our recent telephone interview with her, including updates on the overall state of her research, that now seems to have more backing from the broader scientific community worldwide.

Fast forward to 2015 — it’s been five years since her first round of research and about three years since we published our last one-on-one interview with Dr. Faustman. A lot has been going on, even though she’s largely been in a holding pattern awaiting the start of the next phase of clinical studies, she tells us.

DiabetesMine has talked with Dr. Faustman over the years and kept tabs on her progress, from that href=”https://www.healthline.com/diabetesmine/a-talk-with-denise-faustman/” target=_blank”>initial chat in 2009 to over updates over the years.

For those not familiar with Dr. Faustman’s work at Massachusetts General Hospital, she has long been studying something called BCG (Bacillus Calmette Guerin), a generic vaccine that’s been around for almost a century and was originally designed to combat tuberculosis (TB). The idea: boosting BCG could stop the pancreas from killing off beta cells, allowing those affected by diabetes to regenerate these insulin-making cells. She made a groundbreaking discovery in mice in 2001, but wasn’t initially able to replicate that, and her research has kicked off a firestorm of controversy among the medical community and research orgs who doubted her approach.

Wow, it seems like eons since we last chatted with the influential Dr. Denise Faustman about her controversial research in creating a vaccine to cure diabetes.

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DM) For all of us non-science people out there, give us a primer on BCG and what your research is all about?

DF) Essentially, BCG is a close non-toxic relative of TB and it was first noticed in the early 1900s when consumption killed a lot of people. There was one group of people within a population that didn’t die or even get TB, and it turned out it was young girls milking cows. That’s how BCG was discovered, and over time we learned it was because of the cows and udders, and there was another form of BCG on farms. So, a vaccine could be developed.

Bringing this back to diabetes is interesting, as we didn’t go after it per se. What we knew from 20 years of science data (at the time), is that BCG doesn’t fall from the sky — even if I wish it did. People with type 1 diabetes, multiple sclerosis (MS), and other diseases had a relative deficiency in a hormone known as TNF, and with a natural vaccine boosting TNF, you could get rid of bad T-cells and boost T-regs, and the pancreas regenerates. We decided to use a safe, 100-year-old vaccine to make this happen and we’ve found that it works.

According to this timeline of BCG research, your Phase I human trial finished a whole five years ago. What were the results?

In early data, we showed that in long-term diabetics, indeed these T-regs were boosted and we could see the targeted death of bad T-cells. We also the beginnings of pancreas regeneration. Sure, no one was throwing insulin syringes away yet, because it was just the beginning… but it showed that this could be done. And importantly, this was in long-term type 1s with 15 to 20 years — that rattled a lot of people. This was a totally unique patient population, and not how most research was done in newly diagnosed individuals.

The people in the trial had diabetes for an average of 15 years, and this showed that we could restore insulin production, at least briefly, for people who had type 1 for many years. Phase I was in 2010, so we’ll soon be seeing the five-year followup — something we learned from BCG research on multiple sclerosis is important, and so we’ll be re-studying the patients with type 1 who went through this.

What’s been the holdup in getting Phase II started?

I’m glad you asked that. What we’ve been up to is proving to the world that there’s another reason we should be doing these trials in people who’ve had this disease for a long time. But also just working in conjunction with others, beyond diabetes. In the spirit of science, we’re sharing with other groups around the world who are studying BCG — whether it’s for celiac or MS or Sjogren’s Syndrome. They should be able to continue to learn from our research, without starting the same research over, and of course without compromising our own research.

The biggest roadblock we ran into was the short supply of BCG in the U.S., because it stopped being produced after the Big Pharma factory making it was shut down. BCG isn’t a high-tech product, so when you go to make more, it’s restricted on where it can be manufactured — kind of like a flu vaccine, you just can’t make it at any lab. Think of it like this: If you have a plant making french fries, you can’t suddenly have them start making hamburgers even though they’re both food products often served together. We had to get a contract to do this. We didn’t want to be in the manufacturing business, but we had to in order to continue this research.

As of two weeks ago, we have a new BCG strain and manufacturing process that’s gotten through the FDA. We are very proud of where we are.

That doesn’t sound cheap…

We have been raising money, and so far have raised $18.9 million. The NIH is now in on those trials and funding this for Sjogren’s Syndrome, and data from animal models shows that small doses of BCG in Sjogren’s has a similar effect as it does in diabetes: stopping the disease and regenerating the organ. So, that’s encouraging to see them invested. The JDRF isn’t on board. And Helmsley Charitable Trust is similar to JDRF, as they’re just interested in being part of the discussion on all of this. People vote with their dollars for research, and for this BCG research the big money is coming in from Europe, the NIH, the Lee Iacocca Family Foundation, and private donors.

So, what will Phase II look like and when will it get going?

We are going to look at how much BCG is needed, and how frequently. That’s the key, the secret: knowing how much to dose. With Phase II-a, I’ll try to perfectly match those results in Phase I, with longstanding type 1s who still make a little C-peptide. Then it will be Phase II-B where there isn’t C-peptide in longtime type 1s. And after each part, we need to follow these people for five more years.

We have gotten approval for Phase II and we’re all OK on the manufacturing front, so it will start shortly. Probably in the coming months. In our last research update from the Fall, we wrote that we’re planning for 120 people. We are always looking for more patients to be a part of this, so interested persons can email us at diabetestrial@partners.org.

But we won’t be seeing results anytime soon, since we’re talking another five-year study…

These aren’t fast trials, by any means. We have a five-year followup. But that’s important, because after more than two years, the effects become monumentally more significant. We know this is worth it, because data now shows from Europe that using BCG compared to the standard of care is most effective.

The medical community generally hasn’t backed you in the past. Do you feel there’s more acceptance and support of your work now?

It’s quite amazing what’s happened over the past few years. This is a cheap and generic drug that could be very effective, and we’ve been saying that message over and over ahead since the start. Now, it’s really taken off, especially outside the States where there’s no competition and pricing issues like we have here. There are more efforts on this, and the data tells an effective story.

We have worldwide collaborators taking these steps, and that’s pretty good validation to us, that others want to be a part of this story.

Tell us a bit more about how this research has gone global?

There are more than 7 institutions studying this on a number of different autoimmune conditions, and the early data is showing that the efficacy of BCG could be better than any drugs on the market now.

In Turkey, they decided to follow BCG on the prevention of diabetes, actually. That was in mouse studies… not that you can trust a mouse, but it reinforced what was already found in the other mouse studies. Kids with one vaccine, at age 12 and 14, had the same incidence as those in the general population; but if kids got three vaccinations, the incidence of T1D went way down. That was the first prevention trial using multi-doses, and that data was given to a group in London for re-analysis and has been validated.

In Denmark last year, they tested 5,000 newborns and restarted them on BCG and they’ll look at in 2-5 years to see about allergies and any biomarkers that surface as to the vaccine use.

As I mentioned earlier, the NIH has started trials on Sjogren’s, and there are more than 7 others around the world now studying this.

Now, there are about 20 papers around the world showing what everyone’s seeing — that what we told patients for decades about the honeymoon period was wrong. This opens up the eyes of endocrinologists and patients, about a new vision. That these people should be used for trials, and not just go on a pump because they’ve had diabetes for too long. We hope that concept catches on.

You also published a book on all this global collaboration last year, right?

That was based on a non-profit meeting in late 2013, and we invited about 12 groups to attend and share their research on BCG. That book, The Value of BCG and TNF in Autoimmunity, is a report on the meeting and what we discussed. One thing was how we learned from the MS research community that we needed to study people and the drug for five years, and that’s changed how we looked the upcoming Phase II trials. That was our first meeting, and we will have another in Italy this October with more groups invited.

How has the diabetes research changed, since you first got started?

Ten years ago, no one used the R word (regeneration), and we weren’t allowed to use that in our science papers. That’s changed over time, and now it’s a common concept that everyone is going after. We have come a long way, in thinking how the human pancreas does this very slowly, like in MS where it takes five years.

Despite sometimes using them yourself, you’re not a fan of mice research… what do you see changing as far as the science community’s reliance and skepticism about ‘curing’ mice?

I like to say that it’s a comfortable job studying mice, and just writing a few papers a year and not having to translate that into humans. It’s a good career move to study mice, and that’s a big problem. At the ADA Scientific Sessions last year, a researcher from Sweden stood up and said to everyone in the auditorium that they should be ashamed. Because we’ve failed at every type 1 trial in the past 10 years, since it’s based on mouse research and that doesn’t work. And that’s true — T1D trials are getting a bad name, because they all feel the same. People get frustrated because the mice are cured, but the people research fails. It’s so important to get that research into humans. We need to start saying to our researchers: don’t publish a mouse story saying you have something ‘new and effective’ unless you get blood samples from humans showing the same. If you really believe in your data, then you’d better march it forward into humans before you stand up and say how great this discovery is.

Lastly, how can people follow the latest development in your BCG research, Denise?

We have a newsletter you can sign up for, and people can reach out to us to get more information at Faustman Lab or by emailing at diabetestrial@partners.org.

Thanks for taking the time to chat again, Denise — looking forward to seeing the next phase get going,even if does take 5 years to see results. Keep us in the loop, of course!

*** June 2015 Update ***

The FDA announced it had officially approved Dr. Faustman’s Phase II trial, which runs through June 2023. The clinical trial information can be found here.