Uplifting story: In Girl's Last Hope, Altered Immune Cells Beat Leukemia

[brittone2]

I thought this was a very uplifting story and thought I'd pass it along in case anyone else is interested in reading it. This little girl was brought back from the brink through the use of an experimental leukemia treatment out of CHOP.

http://www.nytimes.com/2012/12/10/health/a-breakthrough-against-leukemia-using-altered-t-cells.html?pagewanted=1&_r=0&hp

[Melbel]

Great story. Thanks for sharing!

[Momit]

Fascinating research and such a wonderful outcome for this little girl.

[chozen]

what a celebration!!

[mik8]

Truly indeed a breakthrough news! DH, a scientist, is aware of Carl Jone's approach for a while, but the reports of patient successes are coming in as pretty new news now. There have been few folks who have tried to do some of this but not really in patients yet because earlier genetic engineering (with retroviruses about 10-15 years ago to try to fix immunodeficiencies like SCID) didn’t go very well. Nice to see that Novartis has a significant buy-in too, and is committed to putting up a $20M research center there and are going to push this forward as a drug. This is wonderful, wonderful news....

[brittone2]

There have been few folks who have tried to do some of this but not really in patients yet because earlier genetic engineering (with retroviruses about 10-15 years ago to try to fix immunodeficiencies like SCID) didn’t go very well.

Yeah, i think that some of the issues with engineered retroviruses were actually at CHOP too, weren't they?

[WatchingThemGrow]

I know a little girl who went to CHOP in September to get this treatment!! DD made her some headband/scarves because she lived next door to us in the apartment we were in. I taught her brother a few years back and was overjoyed to see them, but sad to learn of her acute lymphoblastic leukemia.

Unfortunately the mom emailed me a few weeks ago to tell me her 11 year old girl has relapsed. I wonder if she's the one in the article. I've tried to call and email, but I'm certain the super sweet Korean mom is just beyond heartbroken. Is there something you guys can help me think of to do for the family? I SO wish this sweet girl was the success story in the article.

[mik8]

Yeah, i think that some of the issues with engineered retroviruses were actually at CHOP too, weren't they?

It was at Hopital Necker Enfants Malades in France, I think Paris, 10 kids and another 4 kids (boys) in London.

They used a different kind of retrovirus (this was over 10 years ago) to treat X-linked SCID in boys, and had a very impressive clinical response that cured them, but then 2 of those kids (the youngest ones who received the highest dose) came down with leukemia 30 months later, which was caused by the retrovirus inserting in their genome next to a gene important for hematopoiesis. That put a big chill into gene therapy for a long time.

I think what you are thinking of is Jesse Gelsinger who was the first person apparently who died in a clinical trial for gene therapy (at U. Penn in Philadelphia, dunno if he was at CHOP).

[mik8]

I SO wish this sweet girl was the success story in the article.

It is extremely hard on the family. Perhaps your DD came make something again homemade or write a personal note with a photo of herself. That's the thing about science. It is a trial and error world, but every minute progress is a huge step.

[brittone2]

]

I think what you are thinking of is Jesse Gelsinger who was the first person apparently who died in a clinical trial for gene therapy (at U. Penn in Philadelphia, dunno if he was at CHOP).
Yes, that's the case I was thinking of! Thanks for clearing that up.