Evangelina was diagnosed shortly after birth with severe combined immunodeficiency (SCID); it’s also known as “bubble baby” disease because in the past, children were kept in a sterile plastic bubble to protect them from getting sick. It’s a rare – and often deadly — genetic disorder that meant she had no functioning immune system, leaving her vulnerable to infections. Many children with this condition die within the first year of life.

Evangelina was cured as part of a clinical trial at UCLA run by Dr. Don Kohn. She underwent a stem cell transplant that took her own blood stem cells, genetically re-engineered them, and returned them to her body.  These re-engineered stem cells created a new blood and immune system.

Angelina can now play outside and attend parties with her twin sister Annabella. Her parents Alysia and Christian are overjoyed that both their children are healthy. “I get to look at my kids the way other parents should be looking at their children all the time, but until you are put in that position you never appreciate it, says Alysia. “We take things for granted. We all do. I know I do, particularly when the twins drive me crazy, but I also know how very precious this is.”

Alysia could not hold back her emotions at the December Board meeting and said through tears, “Thank you for keeping my family complete.”

An accident at a friend’s pool party on the eve of Jake’s high school graduation left him paralyzed from the chest down with no ability to use either his arms or hands.

On Thursday, July 7^th 2016, a few weeks later, Jake Javier became a member of a very select group. Jake underwent a stem cell transplant being tested in a CIRM-funded clinical trial sponsored by Asterias Biotherapeutics. He had 10 million stem cells transplanted into his neck at Santa Clara Valley Medical Center in San Jose. Since then Jake has regained use of his arms and is working hard to hopefully regain movement in other parts of his body.

“I’ve tried hand-cycling, using my hands and arms to power the bike rather than my feet. I liked that and might be interested in getting one of those bikes, could be useful for getting around. And I’ve been doing things I’ve never done before, like scuba diving. I got help putting on the gear and then went to the bottom of a 12 foot deep swimming pool. It was really cool. I liked that a lot.”

Even as he works hard on regaining the use of his arms and hands, he has an eye on the future: “Next fall I intend going back to school, to Cal Poly. I’m signed up for mechanical engineering. In the meantime I’m going to try and enroll in some online classes to help get back into the swing of things.”

In 2006, Karl was diagnosed with throat cancer. He had the tumor and his vocal cords removed and now relies on a voice box to speak. Several years later, doctors found the cancer had come back and this time had spread to his lungs. This is how Karl became patient #1 in a CIRM-funded clinical trial at Stanford.

“One day he (the doctor) said we have a new trial we’re going to start would you be interested. I said “sure”. I don’t believe I knew at the time that I was going to be the first one but I thought I’d give it a whirl. Fortunately, I lasted 13 months, 72 treatments with absolutely no side effects.”

The trial used a monoclonal antibody to target a specific protein, called CD47, that sits on the surface of cancer cells. This protein tricks the immune system into not attacking the cancer, earning CD47 the nickname from the Stanford researchers as the “don’t eat me” protein. The monoclonal antibody disables CD47, leaving the cancer vulnerable to being attacked by the patient’s own immune system. The therapy has stopped Karl’s cancer from growing, which has allowed him to spend more time with his wife, Vita, and family.