Amazing New Treatment Could Help Suffering Washington Babies
A remarkable medical breakthrough could be the path forward in helping people suffering from rare disorders. In a global first-of-it's-kind treatment, an infant with a rare, incurable, genetic disorder received a therapy that we thought only existed in the realm of science fiction.
The procedure was performed at the Children's Hospital of Philadelphia (CHOP) on an infant between six and seven months old. KJ was born with an inherited genetic deficiency (Carbamoyl phosphate synthetase I) that allows ammonia to build up in the blood. Too much ammonia build up is toxic so desperate action was needed to try and help KJ.
A team at CHOP and Penn Medicine worked on a gene editing therapy in hopes of helping little KJ. The therapy is called CRISPRs (Clustered regularly interspaced short palindromic repeats) and was customized to specifically address KJ's metabolic disorder.
KJs first treatment came in February of this year. As of April, when KJ was roughly nine months, he had received a total of three gene edited therapies. The most recently reported progress is that KJ has suffered no serious side effects. KJ will have to be closely watched for the rest of his life, so this is not being presented as a cure.
The study was published in The New England Journal of Medicine. One of the authors of the report, Dr. Kiran Musunuru, said in a news release about the promise of the therapy
The promise of gene therapy that we’ve heard about for decades is coming to fruition, and it’s going to utterly transform the way we approach medicine.
We can hope that Dr. Musunuru is right and that this is the beginning of a new avenue of treatment for millions suffering from different genetic afflictions.
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