Personalized Molecular Therapies may Offer Cure for Genetic Disorders

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.

Dr. Eric Hoffman of Children’s Research Institute at Children’s National Medical Center believes that such therapies will have the unprecedented ability to treat muscular dystrophies and other disorders.

He says that a small clinical trial involving a new treatment for Duchenne muscular dystrophy provides a proof-of-principle for personalized molecular medicine.

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.

"Dozens of specific sequences will be required for effectively treating the majority of patients with Duchenne muscular dystrophy," Dr. Hoffman says.

He, however, says that future approaches will require advances in systemic administration of large amounts of customized DNA-like drugs, and proof that long-term delivery of is not 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.

Dr. Hoffman envisions that some parts of the drug approval process may be developed for DNA-like molecular medicine as a 'class' of drugs, rather than individual testing of hundreds of different sequences.

"The patients and their families are crossing their fingers that the drug’s overall chemistry can be shown to be safe," he says.

The new treatment for Duchenne muscular dystrophy has been reported in the New England Journal of Medicine.

Source-ANI
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