Friday, March 16, 2018

Stem cell therapy for adrenoleukodystrophy


A 7-year-old boy traveled from Michigan to Minnesota to get his blood genetically modified, which helps him to battle a rare brain disease.

CBS Minnesota reports Landen Veneklase has Adrenoleukodystrophy (ALD).

ALD prevents Veneklase from going to school or playing outside — his immune system can't handle it.   

"We're here nonstop until we make our daily doctor appointments," said Veneklase's dad, Scot.

"Most hospitals don't even know what ALD is," his mother, Priscilla, added.

Priscilla says she didn't find out she was a carrier until her dad, cousin and several other boys in her family mysteriously died.

"Just happened to run into another doctor who put our family tree together, and we were losing so many boys in our family," she said.

Girls can be carriers of the disease, but the devastating symptoms only appear in boys.
So when Veneklase was born, his parents were able to be proactive. Every year, he had an MRI to watch for signs of ALD.

Those signs showed up this year.

"Once we found out he had a lesion on his MRI, we were already set up with the (University of Minnesota) to come here and get the ball rolling," Priscilla said.

The University of Minnesota Masonic Children's Hospital is one a few that not only knows ALD, but they have some of the top specialists.

Veneklase is one of eight kids involved in a clinical trial. His stem cells were sent to Texas to be genetically modified, which he calls "super hero blood." Once they're corrected, they come back to Veneklase.

"So they took out some of his stem cells and platelets," Scot said. "They shipped them off to get corrected."

Veneklase's parents say the pre-screening made all the difference.

Minnesota is one of few states that does ALD screening on newborns. But many other states, including Michigan, still do not.

The Veneklase family is working to change that.

https://www.cbsnews.com/news/landen-veneklase-super-hero-blood-adrenoleukodystrophy/

See:  http://childnervoussystem.blogspot.com/2017/11/hematopoietic-stem-cell-gene-therapy.html

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