Here is the text of the information provided by the California stem cell agency on the individual patients featured at today's meeting of its governing board.
Brenden Whittaker
Diagnosed with X-linked chronic granulomatous disease (X-CGD), a rare disease that causes the immune system to malfunction leaving it unable to fight off certain bacterial and fungal infections which, over time, can be life-threatening. Hospitalized hundreds of times over the years Brenden almost died on a couple of occasions and had part of his lung and liver removed due to repeated infection.
Brenden was cured thanks to an experimental procedure, developed by Dr. Don Kohn, that removed some of his own blood stem cells; they were then genetically modified to remove the gene that causes the problem, and then re-infused to him. The modified blood stem cells created a new blood system and a healthy immune system capable of fighting off infections.
Evangelina Padilla-Vaccaro
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. 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.
(Evangelina’s parents are Alysia and Christian, her twin sister is Annabella)
Jake Javier
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 7th, Jake Javier became a member of a very select group. Jake underwent a stem cell transplant, getting 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 regain even more use.
Karl Trede
In 2006 Karl was diagnosed with cancer of the throat, he had the tumor and his vocal cords removed. Several years later they found the cancer had spread to his lungs so Karl became patient #1 in a clinical trial at Stanford.
The trial used a monoclonal antibody to target a specific protein on a cancer cell called CD47. 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 stopped Karl’s cancer from growing.
