Drug tailored to one girl with brain disease paves way for similar customized treatments
- N1C Moderator
- Oct 8, 2019
- 1 min read
Updated: Aug 16, 2023
Jocelyn Kaiser (Science)
October 9, 2019
After their apparent success treating a young girl with a drug tailored to counteract a genetic mutation that had given her a usually fatal brain disease, the researchers behind the innovative strategy have this week laid out criteria for similarly helping more sick children. But the U.S. Food and Drug Administration (FDA) is cautioning that such one-off therapies need to be thoroughly considered before moving ahead and carefully evaluated, in part because desperate parents sometimes perceive improvements from a treatment that are not real.
In early 2017, after hearing of 6-year-old Mila Makovec, who had a condition called Batten disease that progressively damages brain cells and leads to death by adolescence, neurologist Timothy Yu of Boston Children's Hospital and co-workers offered to try to help. They quickly designed and had a company synthesize a strand of RNA intended to mask a mutation in a gene called CLN7, which over time was causing Mila's brain cells to accumulate waste and die. They first showed the potential drug, an antisense oligonucleotide that they dubbed "milasen," could correct the CLN7 defect in cells cultured from her skin. With FDA approval, in January 2018 they then began to infuse the RNA into her spinal fluid. The team soon saw improvements in Mila's condition, such as fewer and shorter seizures, Yu reported at a meeting 1 year ago.
KAISER, J. (n.d.). Drug tailored to one girl with brain disease paves way for similar ... https://www.science.org/content/article/drug-tailored-one-girl-brain-disease-paves-way-similar-customized-treatments
My husband was diagnosed with Creutzfeldt-Jakob Disease (CJD) four years ago. For over two years, we relied on prescription medications and therapies, but unfortunately, his symptoms continued to worsen. His memory, coordination, and overall strength declined, and everyday life became increasingly difficult.Last year, out of desperation and hope, we decided to try an herbal treatment program from NaturePath Herbal Clinic. Honestly, we were skeptical at first, but within a few months of starting the treatment, we began to notice real changes. His alertness improved, his movements became steadier, and he regained a surprising amount of energy and clarity.Incredibly, he also regained much of his independence and confidence. It’s been a life-changing experience he feels more like himself again, better than…