Navigation Links
Hope for Progeria - Successfully Validated on Mice, a Treatment Could Soon be Tested on Children
Date:6/30/2008

PARIS, June 30 /PRNewswire/ -- Five years after the identification of the gene responsible for progeria, a rare disease causing accelerated and premature ageing, a team of Spanish and French researchers (Carlos Lopez-Otin and coll. - University of Oviedo - and Nicolas Levy and coll. - Inserm/AP-HM, Marseille, France) have successfully demonstrated a treatment for the disease, using mice. This treatment, combining two existing pharmacological molecules, should slow down the progression of this disease which has hitherto remained untreatable.

Subject to the authorisation of health agencies, this treatment could very soon be tested on 15 children in Europe. This represents a major advance for the families affected by this disease and - more generally - for the understanding of the normal ageing process.

From identification of the gene to the development of treatment and the projected clinical protocol, this work - published in the review Nature Medicine - has received the financial backing of the Association Francaise contre les Myopathies (Muscular Dystrophy Association) thanks to French Telethon donations.

The treatment recently validated in mice is based on a combination of two existing pharmacological molecules: statins (indicated for the treatment and prevention of atherosclerosis and cardiovascular risks) and aminobisphosphonates (indicated for the treatment of osteoporosis). The Franco-Spanish researchers have demonstrated that this treatment attenuated the effects of the disease and significantly raised life expectancy.

As previously demonstrated by Nicolas Levy's team - which originally identified the gene in 2003 - progeria is due to the accumulation in the cells of a truncated protein, progerin, whose toxicity is linked to the presence of a fatty acid which remains fixed to the protein (whilst it is eliminated in normal cells). In order to inhibit or block the toxicity of progerin, the researchers explored the path of synthesis of this fatty acid. In fact, pharmacological molecules are known to block certain stages in this path of synthesis.

After several attempts they noted that a combination of a statin and an aminobisphosphonate could prevent the fixation of the fatty acid to the progerin, and thus reduce its toxicity. The progerin being less toxic, the disease develops more slowly.

Following these encouraging results, a clinical protocol based on this treatment piloted by Nicolas Levy in Marseille (France) is about to start up. It should last 3 years and will concern 15 of the 25 children affected by progeria in Europe. The aim of this protocol is to slow down the progression of the disease and, if possible, prolong the life expectancy - at present very limited - of the children affected.

Article published in Nature Medicine : http://dx.doi.org/10.1038/nm.1786


'/>"/>
SOURCE AFM-Association Francaise contre les Myopathies
Copyright©2008 PR Newswire.
All rights reserved

Related medicine technology :

1. Novagali Pharma Successfully Enrolls First Patient in U.S. Phase I Clinical Study with CORTIJECT(R) for Diabetic Macular Edema
2. Celator(R) Pharmaceuticals Successfully Uses Nanoparticles to Administer Hydrophobic Drug Combinations
3. NanoBio Corporation Successfully Completes Phase 2b Study in Herpes Labialis
4. New Study Shows LEVITRA Successfully Treats Erectile Dysfunction (ED) in Men with Dyslipidemia, Including High Cholesterol
5. YMed, Inc. Successfully Completes First Clinical Cases With Innovative VascuTraK(TM) II PTA Dilatation Catheter
6. Harvard Medical School-Partners HealthCare Center for Genetics and Genomics Successfully Completes Evaluation of AutoGenomics 2C9-VKORC1 Panel to Assess Warfarin Sensitivity
7. TaiGen Biotechnology Successfully Completes Patient Recruitment for Phase II Trial of Nemonoxacin for Treatment of Adult Community Acquired Pneumonia (CAP)
8. Harvard Medical School-Partners HealthCare Center for Genetics and Genomics Successfully Completes Evaluation of AutoGenomics 2C9-VKORC1 Panel to Assess Warfarin Sensitivity
9. Blood Pressure Lowering Effect of Hypertension Treatment Device Further Validated in New Studies
10. XTL Biopharmaceuticals Announces Completion of Patient Randomization Into the Bicifadine Phase 2b Clinical Trial for the Treatment of Diabetic Neuropathic Pain
11. ABC News NIGHTLINE Features HIFU with Sonablate(R) 500 as Non Invasive Treatment Option for Prostate Cancer
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:10/11/2017)... , Oct. 11, 2017  BioPharmX Corporation (NYSE MKT: ... that developed an innovative way to use nonlinear optical ... delivery of new drugs. ... Dermatology Conference will show how researchers from BioPharmX and ... Medical School used a suite of imaging techniques in ...
(Date:10/11/2017)... Texas , Oct. 11, 2017  True ... services, has amplified its effort during National Breast ... about hereditary cancer risks. ... of Clinical Oncology calculated that more than 10 ... have inherited mutations in BRCA1 or BRCA2 and have ...
(Date:10/10/2017)... 2017  NDS received FDA 510(k) clearance in May 2017 for ... stand specifically designed for endoscopy environments. An innovative secondary monitor solution, ... solution to support the improvement of patient outcomes, procedural efficiency, and ... ... ...
Breaking Medicine Technology:
(Date:10/13/2017)... ... October 13, 2017 , ... As health professionals work to improve their approach ... patient is doing more than filling out a survey; in many cases health professionals ... emphasis in health care and research on the importance of active engagement with patients ...
(Date:10/13/2017)... ... October 13, 2017 , ... The American Board of Family Medicine's (ABFM) ... and Chief Executive Officer, succeeding Dr. James C. Puffer upon his retirement. Dr. Newton ... until Dr. Puffer’s retirement at the end of 2018. Upon assuming the role of ...
(Date:10/13/2017)... ... October 13, 2017 , ... PurhealthRX , a leading Health and ... Applying the Purzorb™process to full spectrum CBD oil will revolutionize the rapidly growing CBD ... that can be easily incorporated into liquid products, while reducing costs to end users. ...
(Date:10/13/2017)... QUEENS, N.Y (PRWEB) , ... October 13, 2017 , ... ... recently became a member of ElderCounsel, a national organization of elder law and special ... constantly changing laws and rules. It also provides a forum to network with elder ...
(Date:10/13/2017)... , ... October 13, 2017 , ... Many families have ... many long-term care insurance companies have a waiver for care if the client has ... period, when the family pays for care, is often waived, so the benefits from ...
Breaking Medicine News(10 mins):