Navigation Links
New clinical trial results show how personalized medicine will alter treatment of genetic disorders
Date:12/26/2007

WASHINGTON, DC--One of the nations pre-eminent genetic researchers, Eric Hoffman, PhD, of Childrens Research Institute at Childrens National Medical Center, predicts that in relatively short order, medicines 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 patients 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 drugs overall chemistry can be shown to be safe," he says.


'/>"/>

Contact: Jennifer Leischer
jleische@cnmc.org
202-476-4500
Children's National Medical Center
Source:Eurekalert

Related biology news :

1. U of M begins nations first clinical trial using T-reg cells from cord blood in leukemia treatment
2. Clinical studies show REMICADE reduces incidence of bowel surgeries in ulcerative colitis patients
3. Phase 2 of Singapores Biomedical Sciences Initiative gains momentum for clinical research
4. Cleveland Clinic leading clinical program to improve early-stage lung cancer detection
5. Singulex Teams With Wyeth Pharmaceuticals to Translate Pre-Clinical Biomarker Research Into Clinical Study Design
6. Study of African traditional medicine will begin world-first clinical trial
7. New clinical data shows chromium picolinate improves cognitive function
8. Vitamin E trials fatally flawed
9. Research team says extraterrestrial impact to blame for Ice Age extinctions
10. Trial seeks genetic fingerprint for predicting drug effectiveness
11. The industrial space age
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:3/21/2016)... Massachusetts , March 22, 2016 ... facial recognition with passcodes for superior security   ... ), a leading provider of secure digital communications services, ... their biometric technology and offer enterprise customers, particularly those ... secure facial recognition and voice authentication within a mobile ...
(Date:3/15/2016)... , March 15, 2016 Yissum ... , the technology-transfer company of the Hebrew University, announced ... of remote sensing technology of various human biological indicators. ... raising $2.0 million from private investors. ... based on the detection of electromagnetic emissions from sweat ...
(Date:3/11/2016)... 11, 2016 --> ... report "Image Recognition Market by Technology (Pattern Recognition), by ... by Deployment Type (On-Premises and Cloud), by Industry Vertical ... by MarketsandMarkets, the global market is expected to grow ... Billion by 2020, at a CAGR of 19.1%. ...
Breaking Biology News(10 mins):
(Date:4/28/2016)... ... April 28, 2016 , ... ... Connecticut's innovative, growing companies, today announced the launch of VentureClash , a ... companies. , “VentureClash looks to attract the best early-stage companies here ...
(Date:4/28/2016)... ... April 28, 2016 , ... Morris Midwest ( http://www.morrismidwest.com ), ... manufacturers at its Maple Grove, Minnesota technical center, May 11-12. The event ... Almost 20 leading suppliers of tooling, accessories, software and other related technology ...
(Date:4/27/2016)... ... ... The Pittcon Organizing Committee is pleased to announce that Charles “Chuck” Gardner was ... since 1987. Since then, he has served in a number of key leadership positions ... the program and exposition committees. In his professional career, Dr. Gardner is the director ...
(Date:4/27/2016)... SILVER SPRING, Md. and RESEARCH ... -- United Therapeutics Corporation (NASDAQ: UTHR ) announced ... Co-Chief Executive Officer, of United Therapeutics will provide an ... Deutsche Bank 41 st Annual Health Care Conference. ... May 5, 2016, at 10:00 a.m. Eastern Time, and ...
Breaking Biology Technology: