Thursday, September 25, 2014

VC-01 by ViaCyte Starts a Phase-I Clinical Trial

Some people are horrified at the idea of curing diseases by using embryonic stem cells.  If you are one of those people, stop reading now!  This posting is all about curing type-1 diabetes using human embryonic stem cells.  In the future, you should skip over all my blog postings with the tag VC-01 or ViaCyte.

ViaCyte (previously known as Novocell) has started a phase-I clinical trial for their encapsulated beta cell product, which is called VC-01.  This device is designed to cure type-1 diabetes.  The encapsulation coating allows blood sugar in, and insulin out, but does not allow the body's immune system to attack the beta cells. It also allows nutrients in and waste products out. This allows the beta cells to naturally grow and to react to the body's sugar by generating insulin which goes into the body's blood system. Meanwhile, the body's autoimmune attack can not target these beta cells, and you don't need to take any immunosuppressive drugs (as you would for a normal beta cell transplantation).  The cells inside the coating are human beta cells, grown from human embryonic stem cells.   Here is the company's official diagram:

This Trial

This trial will enroll 40 adults who have had type-1 diabetes for over 3 years.  There is no control group, but some people will get two implants while others will get 4 or 6 implants.  C-peptide will be measured after 6 months, and safety issues will be tracked for 2 years.  They hope to finish in August 2017.

Patients are being recruited now in San Diego, California, USA, and they plan to add more locations in the future.

Clinical Trial Record:
ViaCyte Page:
Twitter Traffic:

Discussion and Opinions

Encapsulated beta cells seem like a straight forward cure for type-1 diabetes, but companies have been working on them since the 1990s, without creating a cure.  There appear to be several difficult problems to solve, especially getting oxygen to the new cells.   Bottom line is this: while encapsulated beta cells sound like a "just needs engineering" cure for type-1 diabetes, decades of work has not led to a cure yet, so it is obviously harder than it looks.

Finally, ViaCyte is very well funded.  In the last few months, they have gotten over $16 million from CIRM, $20 million from Johnson and Johnson, $5 million in venture capital, and half a million from JDRF.

Similar Work

LCT's Diabcell is similar to ViaCyte's VC-01, in that they are both encapsulated beta cell devices. They do use different encapsulation coatings, and Diabcell uses pig beta cells, while VC-01 uses beta cells grown from human embryonic stem cells.  LCT has been tested in people for over 6 years, and is currently in phase-II trials.  (At one time it had approval to be sold in Russia, but it never was sold there.) There is also a device being tested at the University Clinical Hospital Saint-Luc in Belgium, which uses human beta cells (from cadavers) and a different encapsulation coating. 

Several organizations are doing animal tests on various encapsulated beta cell devices.  These include Cerco Medical, Beta-O2, DRI, and several more.

Finally, several organizations are doing human tests on beta cell devices which are not (yet) encapsulated, but they hope to encapsulate in the future.  DRI is doing work like this, as is Serova.  If beta cells are not encapsulated, then you must take immunosuppressives for the rest of your life, so I don't consider those a cure, yet. However, if they then progress to the point where immunosuppressives are not needed, then they would be a cure.

Terminology Note

Some of the news coverage refers to VC-01 as an "artificial pancreas", however I only use that term for electro-mechanical devices.  I use the term "encapsulated beta cells" for devices like VC-01.  You might also hear people refer to it as a "bioartificial pancreas".

If you care about the stem cell production method, here is the company's diagram:

Joshua Levy 
publicjoshualevy at gmail dot com 
All the views expressed here are those of Joshua Levy, and nothing here is official JDRF, JDCA, or Tidepool news, views, policies or opinions. My daughter has type-1 diabetes and participates in clinical trials, which might be discussed here. My blog contains a more complete non-conflict of interest statement. Thanks to everyone who helps with the blog.

1 comment:

U can succeed ! said...

Josh, I believe this is a Phase I/II clinical trial. Which means they can go right to phase III if this goes well.