Gene Therapy: Effectiveness on Rare Brain Disease in Boys

Gene therapy was studied on a range of genetic diseases and were followed up later in life. One of those, a rare brain disease “Cerebral adrenoleukodystrophy,” results in a loss of neurological function and early death which happens in children in “the prime of their childhood development.” There were 77 boys which received “eli-cel” (elivaldogene autotemcel) therapy six years ago. The therapy places a healthy ABCD1 gene onto a Lenti-D lentivirus which replaces the malfunctioning ABCD1 gene. Today most of the boys are free of disability and show no decline in brain function. In another study, 6 of 35 similar patients developed blood cancers associated with the therapy but is considered a better situation compared to the imminent death most of the boys faced before the therapy. The researchers are trying to figure out where and how this problem arises in their therapy process to hopefully further improve its effectiveness.

Other studies show similar effectiveness in eli-cel gene therapy. 94% of 32, 3-13 year old boys, showed no loss of neurological function and 80% were disability free. In another trial containing 35 similarly aged boys, 6 of the boys ended up with a blood cancer again. 5 of them have myelodysplastic syndrome (MDS), where blood cells are improperly formed, and 1 has AML, where too many white blood cells are produced.

These studies are great because they show that gene therapy is increasing in its effectiveness and can be used more often to treat these various genetic diseases. I believe that receiving a cancer is better than living with severe disability and imminent death. I hope that these studies continue, and the researchers can discover a way to either improve or adjust the therapy in a way which the chances of side effects like cancer are severely reduced. Maybe they can get it to the point where there are 0 side effects and expand the therapy to other types of genetic diseases.

Links

https://www.usnews.com/news/health-news/articles/2024-10-10/boys-treated-with-gene-therapy-for-rare-brain-disease-doing-well-6-years-later

https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/what-gene-therapy