Great News: FDA Nod For ViaCyte IND of Diabetes Stem Cell-Based Product

VIACYTE, INC. LOGOType 1 Diabetes is a huge global problem.

Where are the solutions that would compliment or replace insulin therapy for diabetics?

There haven’t been too many that have gotten very far, which makes the news of a potential stem cell-based therapy moving along in the pipeline all the more exciting.

ViaCyte (see more posts here, here, here, here on the company) just got FDA approval for its investigational new drug (IND), paving the way to start a combined Phase I/II clinical trial. ViaCyte was one of four “good citizen” stem cell biotechs that I gave a shout out to in my book on stem cells.

The drug, VC-01, is an embryonic stem cell-derived product consisting of pancreatic progenitor cells (PEC-01 cells) delivered in a capsule (Encaptra device).

In mice, the product effectively treats a model of Diabetes and stabilizes blood sugar. Not too high and not too low. I really think there’s some hope here for human patients down the road with this and other similar kinds of approaches.

What Diabetic patients would be eligible for the trial? According to MarketWatch, about 40 patients with severe disease would be enrolled:

The Phase 1/2 clinical study will evaluate the VC-01 product candidate directly in patients with type 1 diabetes who have minimal to no insulin-producing beta cell function.  In addition to evaluating the safety of the product candidate in these patients, the study is designed to demonstrate the effectiveness of the VC-01 product candidate in replacing the lost endocrine function that is central to the disease.  In an open-label, dose-escalating format, ViaCyte expects to enroll approximately 40 patients in the study at multiple clinical sites.

You stock investors out there might be getting excited about ViaCyte, but unfortunately at present time it is a privately held company. This is one that even I, usually a very cautious investor, might consider buying the stock of if it goes public.

ViaCyte’s work has been funded by both CIRM and JDRF:

“We are pleased to have received FDA acceptance for our clinical trial protocol and look forward to initiating this study shortly” said Paul Laikind, Ph.D., President and Chief Executive Officer of ViaCyte.  “The commencement of this clinical trial marks a significant milestone for ViaCyte, as we begin studying the use of the VC-01 product candidate for the treatment of type 1 diabetes.  Instrumental in our ability to achieve this milestone are JDRF, the leading global organization focused on type 1 diabetes (T1D) research, and the California Institute for Regenerative Medicine, a leading organization focused on advancing stem cell research and regenerative medicine.  Both organizations have been strong supporters of the work we are doing at ViaCyte.”

This is a really big deal and a reason to cautiously have hope for the future of stem cell-based therapies for Type I Diabetes.

 

Blog readers investigate BioGatekeeper, the Yamanaka patent challenger

The readers of this blog never cease to amaze me. What an informed, energetic, bright group.

Within just days they may have collectively shed some light on an intriguing mystery in the stem cell field surrounding this question:

Who is trying, via the name BioGatekeeper, Inc., to nullify Yamanaka’s patent on cellular reprogramming to produce induced pluripotent stem (iPS) cells?

This past week the mysterious organization BioGatekeeper, Inc. filed a challenge to the Yamanaka Patent. The basis of the challenge is the assertion that cellular reprogramming was supposedly obvious based on pre-existing art, in this case meaning previous work and intellectual property (IP) on cellular reprogramming by others.

More specifically, BioGatekeeper focused on one other patent as the leverage for its argument to cancel the Yamanaka Patent: the Whitehead Institute Patent on reprogramming (aka The Whitehead Patent). The Whitehead Patent pre-dated Yamanaka’s. Like Yamanaka’s, the Whitehead Patent also focused on reprogramming of cells to pluripotency.

The Whitehead Institute has indicated that it has no involvement in BioGatekeeper.

A logical question then is why the people behind BioGatekeeper, whoever they might be, chose to focus on the Whitehead Patent as the driving force in their argument? That remains unclear at this time.

Who might be behind BioGatekeeper? The most logic candidates would be those who have been involved in cellular reprogramming over the years, particularly in the early days even before iPS cells. Read on on Page 2!

You’ve got to see these fun stem cell videos including contest winner

Zehner claymation stem cell video

For the last month I’ve been accepting entries to my blog’s stem cell video contest.

This video contest follows up on other fund and educational contests I’ve run with prizes including a stem cell essay contest. In addition I do an annual Stem Cell Person of the Year Award with a $1,000 prize, which I personally pay myself to the winner. These are all part of my way of giving back and promoting the stem cell field and good citizenship (read about my 2013 winner here). Stay tuned for our Stem Cell Person of the Year 2014 event, which will have a surprising new twist.

So how did our video contest turn out?

It has been a lot of fun.

We had numerous entries out of which I picked the top four to show below. It was an especially close call between the top two winners as to who would prevail.

Congrats to Robert Zehner who has won the grand $100 prize in the form of an Amazon gift card!

I found his claymation video (above in German with English subtitles) to be captivating and just downright fun. I love the outtakes/bloopers at the end too so stay tuned for those towards the last part of the video. Admittedly, his take on the stem cell world is extremely optimistic, but his enthusiasm is contagious.

CIRM’s Todd Dubnicoff and Kevin McCormack take second prize. They also provided a very well-done entry on using iPS cells to model ALS in a dish. Well done! Their video is above.

Third places goes to Bill Pfohl, who submitted a very cool stem cell rap video (below).

Emily Easto, Communications Coordinator, at the Centre for Commercialization of Regenerative Medicine (CCRM) also entered our stem cell video contest and finished fourth.

Her video, entitled CCRM Innovations: Cell Culture Bioreactors, also really wowed me (see below).

Emily described the video this way:

I created this video in collaboration with Dr. Elizabeth Csaszar, Development Scientist at CCRM, to show how cell culture with bioreactors is an essential step on the road towards commercializing regenerative medicine therapies. I believe this to be an accessible video that accurately and effectively explains the bioreactor’s role in the future of regenerative medicine and because there are no other comparable videos on cell culture with bioreactors, it is a useful education tool as well.

All these videos are great. Thanks for entering!

Congrats to all and especially our winner, Robert!

Stem Cell Person of the Year 2014 Award: Your Top Candidate?

Who has been the single most important, influential person in the world of stem cells this year? Who has made the biggest positive contribution in 2014?

On Monday, October 1, I will start taking formal nominations for the Stem Cell Person of the Year Award for 2014. This will be the third year of the award.stem cell person of the year 2014

Who’s your top candidate on your mind now? It’s time to start brainstorming. Post your ideas in the comments or email me.

Past winners received recognition for their outstanding contributions and a $1,000 cash prize.

Last year, the winner was Dr. Elena Cattaneo. I named her Stem Cell Person of the Year 2013 due to her courageous work on behalf of the goal of making the stem cell arena safer for patients and for protecting the reputation of the international stem cell field. In 2012, amazing patient advocate Roman Reed won the award because of his remarkably effective work advancing the cause of the stem cell field.

Who will win in 2014?

When the nominations open on Oct. 1 I will have a surprising announcement about this year’s award.

Challenge could cancel Yamanaka iPS cell patent

Yamanaka Patent challengeA new patent dispute has exploded in the stem cell field.

What’s going on?

In 2006, Shinya Yamanaka reported cellular reprogramming to create mouse induced pluripotent stem (iPS) cells in Cell and the next year multiple groups along with Yamanaka’s reported creating human iPS cells.

It’s no exaggeration to say this was a blockbuster development for the cell and developmental biology fields and Yamanaka later went on to win the Nobel Prize for this discovery.

Another element to the iPS cell story is the intellectual property (IP) related to iPS cells.

There have been numerous IP claims and patents related to cellular reprogramming and iPS cells, but the dominant patent has been U.S. Patent No. 8,058,065 to Yamanaka et al. that was issued on November 15, 2011, which has been commonly known as the “Yamanaka Patent”.

The common perception in the stem cell/regenerative medicine field has been that the cellular reprogramming sector would be mostly dominated at the IP level by the Yamanaka Patent despite there being other patents in this arena.

However, I just learned of a serious challenge to the Yamanaka Patent, which could really turn things upside down. I predicted an iPS cell patent war back in a post this May.

The iPS cell patent challenge was filed by a mysterious entity called BioGatekeeper, Inc.

What is BioGatekeeper? Read on on Page 2!