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JDRF and others are stepping up efforts to find a biological (cell-based) cure for type 1 diabetes.
  • JDRF is establishing a string of type 1 diabetes cure research "Centers of Excellence" around the country, with the first already launched in Northern California.
  • In the biggest cure-focused acquisition ever, Boston-based Vertex Pharmaceuticals has acquired nearby diabetes stem cell biotech startup Semma Therapeutics.
  • Researchers at Johns Hopkins have discovered mysterious "Hybrid X Cells" that may play a big role in the development of autoimmunity and possibly trigger type 1 diabetes.
  • New research shows that eating too much gluten in the first 18 months of life may also be a trigger to developing T1D.

Newsflash, Folks: We aren't expecting to see a cure for diabetes at any point soon. But there are dedicated researchers out there working non-stop to pave the path, and they've made some interesting headway recently.

Among the progress is JDRF's launch of a new model for a cure research center, a bio-startup working on beta cell replacement that was just acquired by a large established Pharma company, and new research results presented at the big EASD (European Association for the Study of Diabetes) conference in Spain last week. That event also produced some key new information about the impact of gluten on type 1 diabetes.

Here's a brief look at these diabetes cure topics making headlines at the moment:

 

JDRF launches first cure-focused 'Center of Excellence'

The JDRF announced Sept. 4 that it's opened the first "Center of Excellence" aimed at T1D cure research, and plans to establish more at already-existing university and other research spots around the country and world. The first one is a collaboration between Stanford University and the University of California at San Francisco (UCSF), based on their shared work in immune system, beta and stem cell research.

Specifically, researchers there will concentrate on "the interaction of immune cells with insulin-producing beta cells; generating islets and immune cells from stem cells for next-generation therapies; and developing ways to transplant insulin-producing cells into people with T1D without requiring immunosuppression."

Since insulin cell transplantation isn't widely available for a variety of reasons -- including organ and cell donation limitations, and immunnosuppresion drugs that must be taken afterward for life -- the JDRF Northern California Center of Excellence will try to address those obstacles through continued research in beta cell biology and immunology.

The new designation means the JDRF and California researchers will work together to make sure the best people and necessary funding are funneled to this particular center. The same rationale will apply to future centers the JDRF opens and whatever their particular focus is.

For its part, the JDRF says this new model will provide these Centers of Excellence with "the stability to drive longer-term projects, as well as the flexibility to be nimble as new science emerges. The innovative funding model promotes collaboration and provides longer-term stability to institutions that have demonstrated excellence in T1D research. Each will be initially funded for five years. Funding beyond year three will be confirmed after a review and evaluation."

The T1D organization also notes that these centers will serve as central pillars of JDRF's broader strategy on cure research, and they're sponsored through donor contributions. For this first Northern California center, the JDRF credits these individual donors: Karen and Jeff Jordan, Michelle Griffin and Tom Parker, and Karen and Joe Niehaus.

Of course, we're curious how this will transform JDRF's cure research and better focus the resources and efforts happening around the U.S. and globally, and what this will mean for scientists and entites already working in these areas. In the past, cure research efforts definitely seemed a bit scattered, with many dots nots effectively connected. Hopefully, this new model will eliminate duplication and help hone in research where it matters most.

 

Largest T1D cure research acquisition ever

Also announced at the start of September, a huge corporate acquisition made headlines in the arena of diabetes cure research. Boston-based company Vertex Pharmaceuticals, largely focused on cystric fibrosis to date, bought the diabetes stem cell biotech startup in Cambridge, MA, known as Semma Therapeutics. That company was started in 2014 by high-profile researcher and D-Dad Dr. Douglas Melton, who's been working for over a decade on the creation of new insulin-producing cells.

You may remember that Melton made huge news back in 2013 with what was largely hailed as a breakthrough, though a few years later his research was retracted and called into question. His startup Semma came about in 2015 and was interestingly one of the first funding projects for JDRF's venture philanthropy T1D Fund (see our recent coverage here) in 2017.

Vertex has now shelled out $950 million to tap into the work Semma's been doing. The JDRF describes this as likely the largest T1D cure-focused transaction to ever occur.

Semma’s approach has been two-pronged:

  • Working to create a new supply chain of beta cells from human-derived stem cells, with the aim of transplanting these new cells directly into the liver, where they can produce insulin to naturally regulate BG levels.
  • Creating a device that can be implanted with the new insulin-producing cells housed inside, protecting them from the immune system attack. (Others working on this include ViaCyte, Eli Lilly with Sigilon Therapuetics, and the Diabetes Research Institute with its BioHub).

Semma's work remains in early clinical trials involving animals at this time, and there's certainly no guarantee it will pan out. But it's a huge potential boost to have a company like Vertex now devoting energy and resources to the effort.

Melton says, “Semma was founded to dramatically improve the lives of patients with type 1 diabetes. Vertex is ideally suited to accelerate the achievement of this goal.”

Leaders at JDRF's T1D Fund seem to agree.

“This is a major milestone in our fight to cure type 1 diabetes, in two respects,” says the T1D Fund's Executive Chairman Sean Doherty. “First, a terrific company like Vertex has the resources and expertise to achieve Dr. Melton’s vision, which JDRF shared and supported for many years. Second, we think that investors and industry will take notice of such a substantial value placed on promising type 1 diabetes therapies and look for opportunities to invest in other T1 diabetes efforts in a new, developing market.”

 

Hunting down rogue cells that cause diabetes

Researchers at Johns Hopkins in Baltimore have apparently discovered a mysterious group of "previously unknown cells" lurking in the body that may play a big role in the development of autoimmunity and possibly trigger type 1 diabetes. They've named this enigmatic new entity "Immune Cell X" because of its ability to morph into two other types of cells.

Supposedly, scientists have long believed these hybrid cells could not exist, but if they did, then they were likely just a tiny population along the lines of 7 out of every 10,000 white blood cells. Per Dr. Abdel-Rahim A. Hamad, an associate professor of pathology at Johns Hopkins who co-authored this latest study. For whatever reason, these so-called "rogue cells" get confused and transition into another type that the body deems foreign, and that starts the immune attack that eventually leads to T1D.

Not everyone in the research community is convinced of this, though. Because while other environmental and genetic triggers for T1D may also be at play, it's also possible that the apparent hybrid X cells are actually some of the other "normal" cells and not rogue imposters at all; they may simply serve two functions.

What's clear is that more research is needed on this front, and no doubt that will take time.

Assassinating Rogue Cells

Whether these hybrid X cells are important or not, other new findings presented at the #EASD2019 conference in Barcelona present a way to combat whatever the true T1D-triggering culprit may be at the cellular level.

Belgian clincial-stage company Imcyse is developing immunotherapeutics that might help treat and prevent chronic conditions like T1D, by developing peptides that could be injected or implanted into the body in order to identify and kill off the cells that attack the immune system -- as in the case of T1D.

Early trial data suggests that Imcyse's does indeed increase the number of protective cells in the body. These results are now expected to buoy the company's efforts and help fund a next-phase of research in 2020.

 

The diabetes-gluten effect?

One more new study presented at EASD caught our eye -- on gluten and diabtes, more in the realm of prevention than cure, but important nonetheless.

The impact of gluten on T1D has been a long-explored topic. It goes along with cow's milk and other potential environmental triggers of type 1 diabetes (especially in children).

This newest study shows that a child's intake of gluten at 18 months old led to a whopping 46% increased risk of developing T1D for each extra 10g of gluten consumed per day. However, there was no link between the prospective mother's intake of gluten during pregnancy and type 1 in her child. This research came from Oslo University Hospital and the Norwegian Institute of Public Health in Norway.

The study authors note, "Our observations may motivate future interventional studies with reduced gluten intake to establish whether there is a true causal association between amount of gluten intake in the child's early diet and type 1 diabetes in susceptible individuals."

Why this gluten effect, you might ask?

The researchers suggest it could be based on gluten influencing the gut microbiota and inducing inflammation in a so-called 'leaky gut' fashion. It could also be that gluten sometimes works with other triggers or environmental factors at play -- including a virus or genetic predispostion in children -- to push a child toward type 1.

Interestingly, the study authors specifically say their findings are not enough to push people away from eating gluten, especially cereal and bread that are such common gluten sources. And of course, more research is necessary.

 

Bottom line

The headlines around "diabetes cure" never seem to stop. It's important to be realistic about the incremental nature of scienfitic discoveries and not inflate false hope.

But it's equally important to know just how much research is underway, and follow the progress being made. So much investment and effort is bound to lead us at least to some effective interventions and 'functional cures' in the near future.