World’s largest pharmaceutical company signs deal with ViaCyte supporting stem cell therapy for type 1 diabetes

It’s been a good week for ViaCyte, a good week for us here at the stem cell agency and potentially a great week for people with type 1 diabetes.

Earlier this week ViaCyte announced they have been given approval to start a clinical trial for their new approach to treating type 1 diabetes. Then today they announced that they have signed an agreement with Janssen Research & Development LLC and its affiliated investment fund, Johnson & Johnson Development Corporation (JJDC).

ViaCyte's President & CEO, Paul Laikind

ViaCyte’s President & CEO, Paul Laikind

Under this new agreement Janssen and JJDC will provide ViaCyte with $20 million with a future right to consider a longer-term transaction related to the product candidate that ViaCyte is developing for type 1 diabetes.

The agreement is a big deal because Janssen is a division of Johnson & Johnson, which just happens to be the largest pharmaceutical company in the world (they were also ranked the world’s most respected company by Barron’s Magazine in 2008, not a bad reputation to have). Companies like this have traditionally been shy about jumping into the stem cell arena, as they wanted to be sure that they had a good chance to see a return on any investment they made. Not surprising really. You don’t get to be as successful as they are by throwing your money away.

The fact that they have decided that ViaCyte is a good investment reflects on the quality of the company, the years of hard work the people at ViaCyte have put in developing their therapy, and the impressive pre-clinical evidence that it works. It also reflects the fact that we helped fund the project, investing almost $40 million in the program, and get it to this point

In a news release we issued about the announcement our President and CEO, C. Randal Mills, said:

“This is excellent news as it demonstrates that pharmaceutical companies are recognizing stem cell therapies hold tremendous promise and need to be part of their development portfolio,” says C. Randal Mills, Ph.D., President and CEO of the stem cell agency. “This kind of serious financial commitment from industry is vital in helping get promising therapies like this through all the phases of clinical trials and, most importantly, to the patients in need.”

What’s nice is that this is not just a one-off deal. This is the third time this year that a large company has stepped in to make a deal with a company that we are funding.

In January Capricor Therapeutics signed a deal with Janssen Biotech that could ultimately be worth almost $340 million for its work using stem cells to treat people who have had a heart attack. The same month Sangamo, who we are funding to develop a treatment for beta-thalassemia, signed a potential $320 million agreement with Biogen Idec.

As Randy Mills said:

“Our goal at CIRM is to do everything we can to accelerate the development of successful therapies for people in need,” says Mills. “These kinds of agreements and investments help us do that, not only by adding an extra layer of funding for development, but also by validating the scientific and commercial potential of regenerative medicine.”

It’s great news for ViaCyte. It’s confirmation for us that we have been investing our money well in a promising therapy. But most of all it’s encouraging for anyone with type 1 diabetes, giving them a sense of hope that a new treatment could be on the horizon.

Disease in a Dish – That’s a Mouthful: Using Human Stem Cells to Find ALS Treatments

Saying “let’s put some shrimp on the barbie” will whet an Australian’s appetite for barbequed prawns but for an American it conjures up an odd image of placing shrimp on a Barbie doll. This sort of word play confusion doesn’t just happen across continents but also between scientists and the public.

Take “disease in a dish” for example. To a stem cell scientist, this phrase right away describes a powerful way to study human disease in the lab using a Nobel Prize winning technique called induced pluripotent stem cells (iPSC). But to a non-scientist it sounds like a scene from some disgusting sci-fi horror cooking show.

Our latest video Disease in a Dish: That’s a Mouthful takes a lighthearted approach to help clear up any head scratching over this phrase. Although it’s injected with humor, the video focuses on a dreadful disease: amyotrophic lateral sclerosis (ALS). Also known as Lou Gehrig’s disease, it’s a disorder in which nerve cells that control muscle movement die. There are no effective treatments and it’s always fatal, usually within 3 to 5 years after diagnosis.

To explain disease in a dish, the video summarizes a Science Translation Medicine publication of CIRM-funded research reported by the laboratory of Robert Baloh, M.D., Ph.D., director of Cedars-Sinai’s multidisciplinary ALS Program. In the study, skin cells from patients with an inherited form of ALS were used to create nerve cells in a petri dish that exhibit the same genetic defects found in the neurons of ALS patients. With this disease in a dish, the team identified a possible cause of the disease: the cells overproduce molecules causing a toxic buildup that affects neuron function. The researchers devised a way to block the toxic buildup, which may point to a new therapeutic strategy.

In a press release, Clive Svendsen, Ph.D., a co-author on the publication and director of the Cedars-Sinai Regenerative Medicine Institute had this perspective on the results:

“ALS may be the cruelest, most severe neurological disease, but I believe the stem cell approach used in this collaborative effort holds the key to unlocking the mysteries of this and other devastating disorders.”

The video is the pilot episode of Stem Cells in Your Face, which we hope will be an ongoing informational series that helps explain the latest advances toward stem cell-based therapies.

For more information about CIRM-funded ALS research, visit our ALS fact sheet.