The famous and controversial diabetes cure researcher Dr. Denise Faustman has just published exciting new research results showing evidence that "insulin production may persist for decades after the onset of type 1 diabetes" and "beta cell functioning also appears to be preserved in some patients years after apparent loss of pancreatic function." Her study results appears in the March issue of the journal Diabetes Care.
"The traditional concept is that in type 1 diabetes, the pancreas is dead within two years. What this data now shows is that it's not an acute disease where the pancreas dies in that short interval. Rather, in the majority of people, it's a gradual, slow decline," explained Dr. Faustman, when I spoke to her on the phone last week.
"This opens the question of what is the window for people with long-stage diabetes to still have a chance of being rescued. The time course of the disease is altered by this data!"
Dr. Faustman is nothing if not enthusiastic. One could even describe her as bubbly — which may account in part for the passionate following she's amassed for her controversial approach to cure research, using a substance called BCG vaccine to kill off the cells that attack the insulin-producing cells in type 1 diabetics.
This latest data was almost a byproduct of that work, she explains. Traditional "intervention" research focuses on patients newly diagnosed with diabetes, usually within one year. But Faustman's group could not afford the millions of dollars that big Pharma invests in locating and recruiting new onsets.
"Everybody's jockeying to try to get these kids, but we can't compete at that level for a cheap generic drug (BCG), and also, we already had mouse data showing that BCG actually worked long-term," she says.
So her group recruited adult patients with long-term type 1 diabetes even though they believed they were dealing with dead pancreases, she explains. When samples from these patients were sent off to Sweden for an ultra-sensitive C-peptide test, one surprising result that came back was pancreas activity. "The average in our group was 15 years out from diagnosis, and they all had some residual pancreas function. Actually, it's functioning at low activity but it's alive!" Faustman giggles.
What this means practically is that going forward, more PWDs should be qualifying for clinical trials — as even us long-timers are now candidates for beta cell regeneration studies.
"It's kinda like renewed hope for patients. They're saying, 'you mean my pancreas isn't dead?' It's a new concept for people with type 1 diabetes that their pancreas lives on for a long time. This gives us a bigger window of treatment than we had thought we had for a long time," Faustman says.
Giving Credit, Finding Corroboration
Faustman is quick to point out that she's following in the footsteps of a certain Dr. Alan Foulis, a pathologist in the UK who for many years kept reporting that he saw islet cells in the cadavers of patients with type 1 diabetes. In the research world, "everybody poo-pooed it, because they...did the standard C-peptide test on living patients and didn't see any function." Faustman says her new data is "the first functional data showing that the pancreas is working for a long, long time making insulin."
A certain Dr. Bart Roep based in Leiden, Netherlands, might disagree there. He appears to have just published a study that discovers exactly the same thing. "His discovery negates earlier research which concluded that (insulin-producing) cells are completely absent in type 1 diabetes patients," according to an Expatica.com article with the unfortunate headline "Dutch Professor: Type 1 Diabetes Can Be Cured." That sensational headline has the ill effect of making Dr. Reop sound like an unrealistic idealist, instead of a serious researcher whose findings indicate, in his own words, that "if these cells can be reactivated the patient could be cured, even as long as 10 years after the original diagnosis was made."
The work of Drs. Foulis and Roep is good news for Dr. Faustman, in the sense that her findings are supported by the work of other researchers. The way I understand it, a lack of corroboration by her research peers is what makes Dr. Faustman's BCG work so controversial, and is also the reason she hasn't received funding from established sources, like the JDRF.
Naturally, I asked Dr. Faustman what this all meant for the theory of the "honeymoon phase" in type 1 diabetes. She says that previous thinking about the honeymoon lasting just 6 months or a year and then ending abruptly is probably false.
"The pancreas activity is gradually getting lower and lower — it's not a 6 months' decline, but at 10 or 20 years, the A1C's are getting worse. Everybody blames the patient, but it gets harder and harder to manage," she says. Amen to that.
Faustman's BCG Trials Update
So what's happening with Dr. Faustman's BCG vaccine trials? They hope to re-start Phase 2 pilot studies within the year, she says. Of their $25 million goal, her group has raised $10 million in philanthropic donations thus far, "so we have a ways to go — although this is cheap compared to other trials conducted by big Pharma," she says.
"We're now trying to sustain the vaccine in patients — to get rid of the 'bad T-cells' so the pancreas has a higher and higher chance of recovery."
They're working with the FDA on guidelines for the study, which Faustman says is a new concept for the FDA, because the agency's current guidelines focus on trials working to change the rate of decay of the pancreas. "Ours is the first data they've seen where the pancreas could be turned back on, so the guidelines don't quite fit. What should the end points be? To what level do we have to turn the pancreas back on (to be considered successful)? And for how long?"
They're also working on the Phase 2 trial design: pre-screening patients and defining the population of subjects who should be included. "We've got people booked out for four years from all over world who want to get pre-screened," she says. "Some just want their name on the list, some want to send us their clinical info, and some want to come visit and give us blood samples."
Despite the onslaught, Faustman's still encouraging new patients to get in touch with her Boston-based lab. If you're interested, visit www.faustmanlab.org. No matter how long you've had type 1 diabetes, you'll be happy to know you no longer fall in the "DEAD PANCREAS" file.