Even the best ideas can fail without a lot of support. One of the things we pride ourselves on at the Stem Cell Agency is nurturing really promising ideas for new therapies through sustained funding, giving them the support they need to turn that promise into reality. So it’s very gratifying today to hear that one project we have supported for many years, ViaCyte’s VC-01™ implantable device for treating type 1 diabetes, just took a big step towards being tested in patients.
ViaCyte has submitted what’s called an Investigational New Drug application (“IND”) with the Food and Drug Administration (FDA) asking permission to start a phase 1/2 clinical trial in patients. If the FDA says yes then ViaCyte hopes to start testing their device in patients before the end of the year.
We have invested almost $40 million in nurturing the project through the early, most basic research to see if this approach could be made to work, and then through more rigorous advanced research and testing in animals to make sure it’s safe and that it is effective.
As our Chairman, Jonathan Thomas, says in a press release we sent out announcing the news:
“We have been strong supporters of Viacyte for many years and it’s great to see that they are well on the way to starting a First-in-Human trial, hopefully in the next few months. This therapy’s growth from an idea to a potential treatment highlights CIRM’s commitment to following promising science at all stages of development.”
The device is really quite ingenious. It is a thin plastic pouch that contains an immature form of pancreatic cells. When the device is implanted under the skin these cells become the different kinds of cells needed to regulate blood glucose levels. They are able to sense when blood glucose is high, and then secrete insulin to restore it to a healthy level. The truly impressive part is that the device has holes large enough to allow insulin to be pushed out, but too small to allow the body’s own immune system to get in and attack the device.
The goal of the first phase of this clinical trial, as with all phase 1 trials, is simply to show that the VC-01™ is safe. The second phase will also look at safety but also test it to see if it is helping patients, reducing their dependence on injected insulin. If the results from both those phases are encouraging, the next step is to test it in much larger numbers of patients to see just how effective it is.
But this first step, submitting an application to the FDA, is the starting point for all that. As our President and CEO C. Randal Mills said in our news release, getting to the starting line is often half the battle:
“This is good news for ViaCyte and is an encouraging sign of the progress they are making. Filing for an IND is a crucial step along the path to making a therapy available to patients and we’ll be working with them and supporting them every step of the way to try and make this happen as quickly, and as safely, as possible.”
You can read more about ViaCyte and our support for them on our website.