The search for a diabetes cure is always a hot-button in our community, especially as it pertains to the research done by Dr. Denise Faustman in Massachusetts, whose focus on finding a cheap and easy vaccine stokes quite a bit of "hype vs. hope" emotion.

Even more controversy was kicked up surrounding Dr. Faustman's latest research update, presented at the American Diabetes Association's big annual Scientific Sessions conference this June in Orlando. The ADA and JDRF even went to the extent of issuing a joint public statement cautioning people to take her work with a grain of salt -- given the small sample size and the fact that others haven't been able to fully reproduce her results.

Furthermore, the ADA let it be known that it was investigating whether Dr. Faustman violated their embargo policy by doing a media blitz on her research several days prior to the start of the June conference. (Remember, embargoes are meant to keep the playing field even, so no one researcher steals the media spotlight pre-event). Word was out that if Dr. Faustman did breach the policy, she could be banned from the ADA annual meeting for a year or longer.

All this came up in June, and we've been curious to hear what the outcome would be.

We now know ADA leadership has made a determination, but just what has been decided remains a mystery. Despite all the fuss raised over the summer and the organization's public comments about possible missteps by Dr. Faustman's team, the ADA and JDRF are both now declining to say what the result of that investigation is. It certainly appears as though ADA is sweeping this under the rug without a public acknowledgment of whether it was correct or not in making those claims against Dr. Faustman earlier in the year.

Here's what we know...

 

Dr. Faustman's Diabetes Vaccine Research

First off, we've been following Dr. Faustman's research for more than a decade now. She's 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 is that boosting BCG could stop the pancreas from killing off the beta cells that make insulin and allow the affected cells to regenerate. Faustman made what was described as a groundbreaking discovery in mice in 2001, but wasn't initially able to replicate that, and her bold promotion of this research kicked off a firestorm of controversy among the medical community and research orgs who doubted her approach. In later years, Faustman points out that other mouse studies did replicate some of her initial findings, but that is up for debate depending on whom you ask.

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In her Phase I findings, her team at Massachusetts General Hospital found that in people with "long-term" or "advanced" type 1 diabetes, i.e. having the illness for at least 15-20 years, their vaccine introduced the beginning of pancreatic, insulin-producing cell regeneration.

She finished the first phase of her clinical trials in 2010. She applied for JDRF funding but did not receive a grant, presumably due to doubts about the validity of her work. Mostly because of having to fundraise independently, it took another several years to start the second phase of her research in 2015. That is ongoing and will likely take more years to complete (the estimated clinical trial completion time is 2023 at the moment).

The latest findings published on June 21 are a follow-up on the 9 (yes, nine!) participants enrolled in her original small study eight years ago. It looked at the PWDs' results over the course of three, five and eight years following the Phase I study.

While some mainstream media stories captured the latest on her research earlier in the summer, Dr. Faustman just recently in early October presented at the big EASD (European Association for the Study of Diabetes) in Berlin, Germany. That research showed the following: her BCG basically re-introduces germs back into the immune system, building it up and helping to rewire how the body responds so that it can essentially start boosting the body's BG-lowering capability. Essentially, the latest findings point to a lesser amount of germs in T1 PWDs as a reason for their immune system attack and inability to produce insulin, and by adding that back in through this vaccine it may be possible to start returning the system to normal.

Hmmm. Intriguing stuff, no matter how you slice it and whether it proves valid in clinical research phases over the next several years.

But many in the established medical and research world believe there is good reason to continue to doubt the validity of Dr. Faustman's work.

 

 

When Researchers Get Their Hands Slapped...?

Leading up to the ADA's Sci Sessions in June, Faustman's team did a media blitz releasing some of the newest findings in their research work on the BCG vaccine. Just days later, she presented at the ADA conference.

That set off a firestorm in the medical community, on two fronts:

First, prompted by long-term doubts about her work and enthusiastic self-promotion, the ADA and JDRF issued a joint statement cautioning the D-Community about taking the small study results too seriously. Specific limitations of the research were noted, and the statement concludes: "Overall, the findings prompt thought-provoking questions but not definitive answers, and do not provide enough clinical evidence to support any recommended change in therapy at this time." 

ADA noted that many doctors had reporting patients coming to them asking about this potential BCG vaccine, and the doctors were uncomfortable talking about it.

Secondly, following the orgs' joint statement, the ADA's Chief Science and Medical Officer Dr. William Cefalu said the organization was investigating whether Dr. Faustman had violated the event's embargo policy by discussing her research ahead of time.

We followed up in early September and were told by the ADA spokeswoman Michelle Kirkwood via email simply that, "ADA’s leadership team (has) made a decision and shared it directly with Dr. Faustman and her colleagues." She referred further questions to Faustman's team.

When we pressed for more, the ADA offered this statement: 

"All researchers found to be in violation of the ADA’s Embargo Policy have been notified of the ADA’s decision and the actions taken, as detailed in the policy. The ADA does not release the names of researchers who have been found to be in violation of the Embargo Policy; all decisions are shared directly with the research authors. The Embargo Policy is and must be applied uniformly to all researchers who present at the ADA’s Scientific Sessions."

Huh?! So wait a sec... you take the steps to criticize a researcher publicly, and make no secret of the fact that she could be banned from the Scientific Sessions, but then when the decision comes down, you refuse to share it with the public?

If this is an attempt to save face for the researcher, it's an odd one, given that she's already had her hands slapped publicly (metaphorically speaking). And what happened to transparency?

Of course we asked Dr. Faustman and team for details or a response, but they also declined to offer specifics. Instead, Dr. Faustman referred comments to Dr. Harry W. Orf, Senior VP for Research at Massachusetts General Hospital, who offered this statement:

"The referenced issue is between Massachusetts General Hospital and the ADA, and it is inadvisable for us to comment on this matter at this time. The hospital and Dr. Faustman’s lab remain focused on advancing the science and conducting the ongoing larger clinical trial with the hope that it will validate the positive results from the initial more limited patient cohorts, with the ultimate goal of benefiting patients with diabetes and their families. The MGH has reached out to the ADA to continue the conversations regarding the BCG clinical trial. We are not aware of issues related to Dr. Faustman attending ADA Scientific Sessions.”

 

What Happened to Transparency?

Why is it that ADA (and JDRF for that matter) took the bold step to issue a public joint statement cautioning our community to be wary of Dr. Faustman's results, and the ADA's Dr. Cefalu also publicly discussed the investigation into possible infractions on her part... yet they're slamming the door on sharing the outcomes of all this?

To be clear, we're not debating the merits of Dr. Faustman's research here, but rather exploring fairness and transparency -- not only for the researchers and medical community, but also for all the PWDs who have an interest in this cure research and witnessed the public back-and-forth in June.

Clamping down on this seems especially odd given our recent interview with the ADA's new CEO Tracey Brown, who specifically pointed to the organization's need to better connect with patients and build trust among the community.

It's certainly not the end of the line for Dr. Faustman if she is not present at the next ADA conference, even though it is the world's biggest such gathering, convening over 14,000 diabetes professionals. Her work can clearly go on, as usual.

But if there is a policy in place prohibiting the outcome of an investigation into a researcher from being made public, that should have been acknowledged at the outset. Or perhaps the policy should be re-examined, in a day and age when information is ubiquitous and transparency is key to building trust -- especially for advocacy organizations

Somehow it seems that situations like this should be part of the Patient Advocacy Transparency Act, introduced to Congress in June 2018. Because let's face it, doing the deals behind closed doors just isn't cutting it anymore.

We're just sayin'...