Producing insulin for people who can’t

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ViaCyte’s implantable stem cell pouch

One of the huge advantages of a stem cell agency like CIRM (not that there is anything out there quite like us, but anyway) is our ability to support projects as they progress from a great idea to a therapy actually being tested in people.

Exhibit A on that front came via a news release from ViaCyte, a company that is developing a new approach to helping people with severe Type 1 Diabetes (T1D).

Unlike type 2 diabetes, which is largely diet & lifestyle related and develops over time, T1D is an autoimmune condition where the person’s immune system attacks and destroys the insulin-producing cells in the pancreas. Without those cells and insulin the body is not able to regulate blood sugar levels and that can lead to damage to the heart, kidneys, eyes and nerves. In severe cases it can be fatal.

ViaCyte (which has been supported with more than $72 million from CIRM) has developed a pouch that can be implanted under the skin in the back. This pouch contains stem cells that over a period of a few months turn into insulin-producing pancreatic islet cells, the kind destroyed by T1D. The goal is for these cells to monitor blood flow and when they detect blood sugar or glucose levels are high, can secrete insulin to restore them to a safe level.

They tested this approach in 15 patients in a Phase 1 clinical trial in Canada. Their findings, published in the journals Cell Stem Cell and Cell Reports Medicine, show that six months after implantation, the cells had turned into insulin-producing islet cells. They also showed a rise in C-peptide levels after patients ate a meal. C-peptides are a sign your body is producing insulin so the rise in that number was a good indication the implanted cells were boosting insulin production.

As Dr. James Shapiro, the Chair of Canada Research and one of the lead authors of the study says, that’s no small achievement: “The data from these papers represent a significant scientific advance. It is the first reported evidence that differentiated stem cells implanted in patients can generate meal-regulated insulin secretion, offering real hope for the incredible potential of this treatment.”

And that wasn’t all. The researchers say that patients spent 13 percent more time in the target range for blood sugar levels than before the treatment, and some were even able to reduce the amount of insulin they injected.

Now this is only a Phase 1 clinical trial so the goal was to test the safety of the pouch, called PEC-Direct (VC-02), to see if the body would tolerate it being implanted and to see if it is effective. The beauty of this method is that the device is implanted under the skin so it can be removed easily if any problems emerge. So far none have.

Ultimately the hope is that this approach will help patients with T1D better regulate their blood sugar levels, improve their health outcomes, and one day even achieve independence from the burden of daily insulin injections.

Moving a great idea targeting diabetes out of the lab and into a company

Tejal Desai in her lab at UCSF: Photo courtesy Todd Dubnicoff

It’s always gratifying to see research you have helped support go from being an intriguing idea to something with promise to a product that is now the focus of a company. It’s all the more gratifying if the product in question might one day help millions of people battling diabetes.

That’s the case with a small pouch being developed by a company called Encellin. The pouch is the brainchild of Tejal Desai, Ph.D., a professor of bioengineering at UCSF and a CIRM grantee.

Encellin’s encapsulation device

“It’s a cell encapsulation device, so this material can essentially protect beta cells from the immune system while allowing them to function by secreting insulin. We are placing stem cell-derived beta cells into the pouch which is then implanted under the skin. The cells are then able to respond to changes in sugar or glucose levels in the blood by pumping out insulin.  By placing the device in a place that is accessible we can easily remove it if we have to, but also we can recharge it and put in new cells as well.”

While the pouch was developed in Dr. Desai’s lab, the idea to take it from a promising item and try to turn it into a real-world therapy came from one of Dr. Desai’s former students, Crystal Nyitray, Ph.D.

Crystal Nyitray: Photo courtesy FierceBiotech

After getting her PhD, Nyitray went to work for the pharmaceutical giant Sanofi. In an article in FierceBiotech she says that’s where she realized that the pouch she had been working on at UCSF had real potential.

“During that time, I started to realize we really had something, that everything that pharma or biotech was looking at was something we had been developing from the ground up with those specific questions in mind,”

So Dr. Nyitray went to work for QB3, the institute created by UC San Francisco to help startups develop their ideas and get funding. The experience she gained there gave her the confidence to be the co-founder and CEO of Encellin.

Dr. Desai is a scientific advisor to Encellin. She says trying to create a device that contains insulin-secreting cells is not new. Many previous attempts failed because once the device was placed in the body, the immune system responded by creating fibrosis or scarring around it which blocked the ability of the cells to get out.

But she thinks their approach has an advantage over previous attempts.

“This is not a new idea, the idea has been around for 40 or more years but getting it to work is hard. We have a convergence of getting the right cell types and combining that with our knowledge of immunology and then the material science where we can design materials at this scale to get the kind of function that we need.

Dr. Nyitray ““If we can reduce fibrosis, it really helps the cells get nutrients better, survive better and signal more effectively. It’s really critical to their success.”

Dr. Desai says the device is still in the early stages of being tested, but already it’s showing promise.

“We have done testing in animals. Where the company is taking this is now to see if we can take this to larger animals and then ultimately people.”

She says without CIRM’s support none of this would have happened.

“CIRM has been really instrumental in helping us refine the cell technology piece of it, to get really robust cells and also to support the development to push the materials, to understand the biology, to really understand what was happening with the cell material interface. We know we have a lot of challenges ahead, but we are really excited to see if this could work.”

We are excited too. We are looking forward to seeing what Encellin does in the coming years. It could change the lives of millions of people around the world.

No pressure.