New Clinical Trial Results Show How Personalized Medicine Will Alter Treatment O

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New Clinical Trial Results Show How Personalized Medicine Will Alter

Treatment Of Genetic Disorders

http://www.medicalnewstoday.com/articles/92777.php

One of the nation's pre-eminent genetic researchers, Hoffman,

PhD, of Children's Research Institute at Children's National Medical

Center, predicts that in relatively short order, medicine's next

innovation--individualized molecular therapies--will have the

unprecedented ability to treat muscular dystrophies, and other

disorders.

In the latest edition of the New England Journal of Medicine, Dr.

Hoffman posits that the results of a small clinical trial involving

a new treatment for Duchenne muscular dystrophy provides a proof-of-

principle for personalized molecular medicine. Practical

implementation of the 'exon-skipping' approach described in the co-

published report of vanDeutekom et al. will require advances in

systemic administration of large amounts of customized DNA-like

drugs, and proof that long-term delivery is not toxic. However,

these advances are likely to come in short order, with the oversight

and regulations of the FDA critical for appropriate labeling and

marketing of such personalized molecular target drugs.

Though this particular treatment remains in its early stages, within

the foreseeable future the now-standard Phase I, II, and III pathway

to drug approvals may need to be re-evaluated.

How can DNA-like drugs specific to a single patient's mutation go

through the existing approval process " Are the current standards of

rodent and monkey toxicity studies relevant and appropriate for DNA-

like drugs, when the animals do not have the same DNA target (or off-

target) sequences as humans " These and other questions are certain

to pose exciting challenges to both the approval and marketing

processes of drugs.

The study featured in the latest edition of The New England Journal

of Medicine, involves application of a nucleic acid drug called

PRO051. It shows some success at restoring the expression of the

specific protein--dystrophin, that is linked to healthy muscle

tissue. This approach 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, "

writes Dr. Hoffman. " 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. "

Dr. Hoffman envisions that some parts of the 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.