Stories of Hope: Diabetes

This week on The Stem Cellar we feature some of our most inspiring patients and patient advocates as they share, in their own words, their Stories of Hope.

The last thing Maria Torres expected was to be diagnosed with type 2 diabetes. She exercised, ate well and kept her weight under control. There had to be some mistake. Maria asked her doctor to repeat the tests, but the results were the same. At 43, for reasons no one could fully explain, she had diabetes, and her life was going to change dramatically.

Maria Torres' diabetes diagnoses was frightening—but she is hopeful that stem cell therapies could one day change how doctors treat this devastating condition.

Maria Torres’ diabetes diagnoses was frightening—but she is hopeful that stem cell therapies could one day change how doctors treat this devastating condition.

“It really scared me,” says Maria. “I thought I was going to die soon.”

That Maria doubted her diagnosis is no surprise. Type 2 diabetes is often associated with obesity, and she didn’t fit the profile. Most likely, some undiscovered genetic component had made her susceptible to the disease.

Regardless, she now had to rework her life to manage the diabetes. Her cells had developed a condition called insulin resistance. Though her pancreas was producing insulin, which tells cells to take in blood sugar, the cells were not cooperating. As a result, glucose was accumulating in her blood, putting her at risk for heart disease, nerve damage, eye issues and a host of other problems.

To help her cells absorb glucose, she needs regular insulin injections. Maria injects the hormone five times a day and must often measure her blood sugar levels even more frequently.

Faithfully following this regimen has kept her alive for 20 years, but insulin is not a cure. Even with the regular injections, she faces dramatic mood swings and more serious complications as glucose levels rise and fall.

Working for a Cure
One of the most promising strategies to cure diabetes is to transplant beta cells, which sense blood sugar levels and produce insulin to reduce them. Patients with type 1 diabetes would benefit because new beta cells would replace the ones they’d lost to disease. Type 2 patients, like Maria, could increase their body’s ability to produce insulin, lowering blood sugar levels and alleviating the need for injections.

With almost $40 million in funding from CIRM, a San Diego-based company named ViaCyte is working on this solution. They have spent years developing new methods to turn human embryonic stem cells into insulin-producing beta cells. It hasn’t been easy. Stem cells are promising because they can form any tissue. However, to make a specific type of cell, researchers must replicate the exact signals that transform a stem cell into a beta cell, rather than a neuron or muscle cell.

In 2008, the company succeeded, but with a clever twist. They created progenitor cells, one step shy of mature beta cells, and allowed them to finish developing in the body. In animal studies, the hardier progenitor cells survived the transplant process and, once mature, began producing insulin. The project has another innovation up its sleeve: these progenitor cells are first placed in a porous capsule, about the size of a credit card, before transplantation under the skin. This device allows transfer of blood sugar, insulin, oxygen, and other molecules but keeps cells out, thus avoiding the possible attack and rejection by the patient’s own immune system.

ViaCyte’s goal is to start clinical trials for type 1 diabetes by the end of 2014. But the company eventually hopes to also help those with type 2. Maria Torres is eager for them to succeed, both for herself and her family.

“I have three kids, and I know they could have the same thing I have,” says Maria. “If they find a cure, for me, that’s peace of mind.”

For more information about CIRM-funded diabetes research, visit our Diabetes Fact Sheet. You can read more about Maria’s Story of Hope on our website.

Stem Cell Agency Funded Treatment for Type 1 Diabetes Takes a Big Step towards Clinical Trials

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.