‘Bubble Boy’ Disease Cured With Stem Cells

‘Bubble Boy’ Disease Cured With Stem Cells
Alice Park
Nov. 19, 2014

Researchers have treated more than two dozen patients with a treatment made from their own bone marrow cells

Alysia Padilla-Vaccaro and Christian Vaccaro owe their daughter’s life to stem cells. Evangelina, now two, is alive today because she saved herself with her own bone marrow cells.

Evangelina, a twin, was born with a severe immune disorder caused by a genetic aberration that makes her vulnerable to any and all bacteria and viruses; even a simple cold could be fatal. But doctors at University of California Los Angeles (UCLA) Broad Stem Cell Research Center gave her a new treatment, using her own stem cells, that has essentially cured her disease. She’s one of 18 children who have been treated with the cutting-edge therapy, and the study’s leader, Dr. Donald Kohn, says that the strategy could also be used to treat other gene-based disorders such as sickle cell anemia.

I really, really, really hope this “pans out” in the long term! For this condition and for others that might respond to similar treatments!


Wow! What a heartwarming story. Thanks for posting it, Pete. So nice to have good news for a change.

This is just short of miraculous, and gives tremendous argument in favor of grants for “the good sciences” that doctors and researches need in order to find the cures they do.
Hopefully, this will work for Sickle Cell, as well. Heaven only knows maybe what else.

It’s great when people can use their own bone marrow cells for stem cell transplant. My great-niece couldn’t use her own, because they were the problem to begin with. And she didn’t have to have an exact match (just very close) because the chemo to destroy her old bone marrow also resulted in destroying her immune system, so she wound up with a whole new immune system.

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I took care of a Bubble Boy many many years ago however, he was no longer in a bubble. His father wrote a book about him, I believe it was called, “Jared’s Story.”

The problem with stem cells (thus far) is two fold. The stem cells don’t always go to where we want them. The stem cells will often go where they are needed more than where they were intended.

The other problem (and eventually science will work out these issues) is that sometimes they don’t stop working. Europeans are doing a lot of work with total knees and stem cells. The problem they are finding is that they just don’t stop growing more bone tissue. When cells don’t know when to stop growing, we tend to call that “cancer” and that’s not great. If someone already has something such as liver cancer the stem cells will feed the liver tumors and make them grow like crazy. You really have to know ahead of the game that there are no unwanted tumors throughout the body.

Stem cells in Type I and Type II diabetics, that is nothing short of amazing. People born being a Type I diabetic are going off all insulin, the stem cells are repairing a faulty pancreas. For me, anyway, that is shocking. The only down side is that people often need to be retreated every couple of years or so but that doesn’t really seem to high of a price to pay for the end result.

Stem cells, this is a topic that is of great interest in Mexico. They are leading the way in comparison to the US. The US has the FDA and that can stall progress by decades. Many US doctors (I hear) have moved to Mexico so they can advance their research before they retire. There is a Mexican version of the FDA but they seem a bit more realistic in this case.