A leading US genetic researcher has predicted that individualized molecular therapies to cure genetic disorders will be the next innovation in the field of medicine.
According to Dr. Eric Hoffman of the Children’s Research Institute at Children’s National Medical Center, such therapies will have the unprecedented ability to treat muscular dystrophies and other disorders.
Hoffman says that a small clinical trial that involves a new treatment for Duchenne muscular dystrophy provides a proof-of-principle for personalized molecular medicine.
Basically, the experiments involved the application of a nucleic acid drug called PRO051. It showed some success in restoring the expression of the specific protein, dystrophin, which is linked to healthy muscle tissue.
The treatment was shown to reactivate dystrophin protein production in small areas of muscle tissue at the injection site of muscular dystrophy patients.
Dr. Hoffman says, “Dozens of specific sequences will be required for effectively treating the majority of patients with Duchenne muscular dystrophy.”
However, he says that future approaches will require advances in systematic administration of large amounts of customized DNA-line drugs and proof that long-term delivery of it is not at all toxic.
But in order to realize the promise of personalized molecular medicine in muscular dystrophies and, ultimately, other disorders, it will be important to re-evaluate current measures of toxicity, efficacy, and marketing that ensure both safety for the patient, as well as rapid development and distribution of life-saving drugs,” he says.