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:2/8/2016)... 8, 2016   HighPoint Solutions today ... (NRG),s pharmacovigilance technology services division.  NRG is a ... an Oracle Argus Specialized partner, providing drug safety ... companies. --> --> ... sciences capabilities and provides a global scale and ...
(Date:2/8/2016)... part of a major growth and expansion initiative, Dune Medical ... Chmura as President of Dune Inc., its U.S. division. ... experience in the medical device space will play a large ... --> --> In her new ... and operational functions in the U.S. She is tasked with ...
(Date:2/8/2016)... Feb. 8, 2016 Velano Vascular, a medical ... hospitalized patients and their practitioners, announced today that the ... Velano will use the proceeds from this financing, an ... in January 2015, to support the development and commercialization ... pediatric populations. Philadelphia , ...
Breaking Medicine Technology:
(Date:2/8/2016)... ... February 08, 2016 , ... ... While a significant number of women and men with eating disorders report a ... that best predicts the development of an eating disorder. , At the ...
(Date:2/8/2016)... (PRWEB) , ... February 08, 2016 , ... ... excited to share this important news! AHCC and the Home Health and Hospice ... Clinic, CMS' designee for official ICD coding guidance and clarifications, to address concerns ...
(Date:2/8/2016)... ... February 08, 2016 , ... The schedule is ... this country. The AutismOne 2016 Conference, which is being held May 25-29 at the ... hear elsewhere about helpful interventions and causes of chronic illness in children. , Very ...
(Date:2/7/2016)... Dallas, Texas (PRWEB) , ... February 07, 2016 ... ... its new MyDecision™ program. MyDecision™ empowers employers and organizations with the tools and ... , MyDecision™ combines three elements to cut the cost of providing employee healthcare ...
(Date:2/6/2016)... ... February 06, 2016 , ... With the FCPX LUT: Summer ... grades to their footage. A LUT is a Lookup Table that contains a mathematical ... color indicated by the table. By manipulating each pixel, LUT's can change each color ...
Breaking Medicine News(10 mins):