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Stem Cell Therapy Saves Girl From Deadly Condition

Posted on June 29, 2018

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The life of a 5-year-old California girl has been saved by a treatment using her own stem cells, according to a news report by Fox News KTVU in Oakland, California.

Six years ago, the girl, Evie Padilla Vacarro, was born with an extremely rare disease known as severe combined immunodeficiency, also known as “bubble boy” disease.

The condition earned its moniker because for years children with the disease were kept in a sterile environment, like a “bubble,” to prevent them from becoming sick.

Severe combined immunodeficiency, also known as alymphocytosis, is a rare genetic disorder identified by a disruption in the development of T cells and B cells caused by genetic mutations.

T cells and B cells play a significant role in the body’s development of the adaptive immune system.

“The adaptive, or acquired, immune system develops as the body is exposed to germs,” said Dr. Joel Singer, a New York stem cell physician.

Severe combined immunodeficiency affects one in every 50,000 to 100,000 births, according to the United States National Library of Medicine Genetics Home Reference.

Left without a normal defense against germs, Evie was unable to fight off infections. Her condition was so deadly that she was at risk of dying if she contracted the common cold.

As a result, her parents were forced to wear masks to protect their baby from germs.

The usual lifespan for children with severe combined immunodeficiency is just two years.

Evie’s parents were desperate for a cure for their daughter. When she was still an infant, they took her to the University of California, Los Angeles to undergo a stem cell transplant as part of a groundbreaking treatment plan that had taken 30 years to develop.

The transplant involved taking Evie’s own stem cells from her blood and re-engineering them genetically. Once that process was complete, the re-engineered stem cells were returned to her body and began creating new blood cells and a new immune system.

Six weeks after her procedure, Evie’s T cell count began to increase, and her immune system began to develop. With her defense system being bolstered, Evie was able to fight off illness-causing bacteria and viruses.

She was also able to go into the outside world for the first time.

Four years later, Evie has had some other experiences she would never have been able to have before her treatment, such as flying on an airplane and going to public events with large crowds.

She will even begin kindergarten later this year.

Evie’s stem cell therapy procedure was made possible by the California Institute for Regenerative Medicine in Oakland. The organization has also supported stem cell treatment for 40 other sick children since its inception.

The California Institute for Regenerative Medicine was created after the passage of Proposition 71 in 2004 and works to help fund stem cell research and therapies to find cures for conditions such as severe combined immunodeficiency disease, Alzheimer’s disease, Parkinson’s disease and ALS, or amyotrophic lateral sclerosis, a fatal progressive neurodegenerative condition affecting the brain and spinal cord.

Evie’s story is just one of many stem cell regenerative medicine stories that highlight the body’s ability to heal itself, according to Singer.

“Stem cells are small but powerful healers. We see more and more applications for them and success in their use every day,” he said.

Using stem cells taken directly from a patient to use in their own body is ideal as there are no risks of rejection or reaction like with tissues transplanted from other people.

“There is little to no risk with this procedure, and many people see significant benefits after just one treatment for their condition,” Singer said.

 

Sources:

Seattle Cancer Care Alliance. SCID Facts. 2018.

Fox KTVU. 5-year-old California girl cured of rare disease using stem cells. 16 June 2018.

 

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