We're making big strides towards a band-aid sized device implanted under the skin of people with diabetes that would release insulin as needed, potentially eliminating the need to check blood sugars, count carbs and dose for insulin through injections or a pump!
Yes, on Friday news broke that San Diego-based biotech company ViaCyte filed an Investigational New Drug (IND) application with the FDA to move forward with Phase I/II clinical trials in people with type 1 diabetes. The trial would evaluate the safety and efficacy of a new stem cell-derived encapsulated cell replacement therapy, known officially as VC-O1. Basically, that product uses pancreas endoderm cells derived from embryonic stem cells and would be put into the body using something called the Encaptra delivery system.
You may remember from our past coverage that Encaptra is a flat device about the width of a credit card. It would be implanted in under the skin (possibly in the upper back) through a simple outpatient surgical procedure and designed to last at least a year, possibly up to five years, before it would need to be replaced.
This device would be loaded with insulin-producing cells before implantation, and contains pores that allow glucose and insulin to be transferred through, but not antibodies -- meaning insulin would be released as needed in response to the varying glucose levels, but no immuno-suppression drugs will be necessary because the device is protected from autoimmune attack by the sheet's membrane. Very cool!
This has been a long time coming... you might remember, this is the company formerly known as NovoCell. And JDRF has been working with them (and others) on this encapsulation tech for years, announcing a partnership with ViaCyte in December 2011. Since then, JDRF has funneled $6 million into this ViaCyte research to get to this point. And in February, JDRF announced it would provide as much as $7 million more to ViaCyte once this FDA filing was made, in order to help bring the encapsulation device from the clinical trials phase to development.
News of this regulatory milestone has been on the radar for months, with many in our D-Community and JDRF itching to share the latest developments. Indeed, the Encaptra prototype has been on display and being shown off at various diabetes events and conferences, such as JDRF Advocacy Day in March, and there have been several photos posted online of JDRF CEO Jeffrey Brewer, a D-Dad himself, smiling as he holds the band-aid sized encapsulation device. As part of this news, ViaCyte submitted to the FDA what's known as a Medical Device Master File (MAF) to get the ball rolling on that device's regulatory approval.
In a news release about the ViaCyte regulatory filing, Brewer said this:
"Today's announcement is a significant milestone and provides momentum on the path to conducting the first clinical study of this potentially transformative therapy for people with type 1 diabetes (T1D). We are excited to continue our collaboration with ViaCyte and believe beta cell encapsulation therapy may one day virtually eliminate the daily management burden for those living with T1D. The ability to encapsulate and thereby protect implanted insulin-producing cells has been a focus for JDRF and a product candidate like VC-01 could potentially play a key role in helping us achieve our vision of creating a world without type 1 diabetes."
**Related News:Word began circulating Sunday evening that on the same day this ViaCyte announcement went public, the JDRF Board contacted Jeffrey Brewer about its desire for a "change in leadership direction." So, he will be stepping down as CEO and the JDRF has named another D-Dad, Derek Rapp, to succeed him. Stay tuned for more updates on that...)
On the ViaCyte development, we inquired about the timing of the new trials, but of course it's all still TBD and dependent on how long the FDA takes to review the request.
In an interview last year, ViaCyte's CEO Paul Laikind told us that animal studies have proven effective and the company has been working to get investor funding for the human clinical trials. He said it could also potentially be a treatment option for insulin-using type 2s. During that interview, Laikind told us that in the first clinical trial, he anticipates the age inclusion range will be from 18-55 years. Once clinical trials start, information on the location of clinical study sites will be available online at the U.S. government online hub, clinicaltrials.gov.
Of course, this isn't the only encapsulation project underway and others are making progress, too. The ViaCyte research is being featured in The Human Trial film project, along with the Hanuman Medical Foundation's work in California on the Islet Sheet and the Diabetes Research Institute's BioHub (the latter of which recently got FDA approval to move ahead with a pilot human clinical trial). There's also Canada-based Sernova, already conducting clinical trials on an Islet Cell Pouch. And Living Cell Technologies in New Zealand is working on encapsulated pig islet cells with its Diabecell solution, that uses a micro-encapsulation technique.
There's a lot on the horizon for encapsulation research, and with all of these many players making progress, we're excited about the fast pace of research, to inform us all just how effective these kinds of therapies really are!