Action plan 2011-2013

9 - Standard operating procedures Biobanks

AchievementsAchievements

1 -

Standard protocols and research tools for preclinical efficacy studies

Improve standardization of preclinical efficacy studies by implementing use of standard protocols and by developing additional research tools.

Raffaella Willmann
(Basel)

Achievements 2011-2013

Implementation of standardized protocols and guidelines for DMD and SMA

The TACT (TREAT-NMD advisory committee for therapeutics) requests the use of standardized procedures, or the rationale for not adopting these procedures, in all applications it receives and evaluates.

Standard protocols produced between 2007 and 2011 are followed up and regularly updated every 1.5-2 years. This task is funded by the Swiss Foundation for Research on Muscle Disease (FSRMM), member of the TREAT-NMD Alliance. The protocols were made available on the websites of two additional funding agencies (www.curecmd.org and www.fsrmm.ch), and reported at various international meetings by members of the TREAT-NMD Task Force. As of 2013, 16 protocols for assays on mdx mice, 4 protocols for the dog model GRMD, 11 protocols for SMA mouse models, 8 protocols for assays on the dyW mouse model, two recommendations on mouse handling and care and one on data handling are available online. Additionally, one review paper with recommendations for study design in the mdx mouse and an assessment of variability of outcomes in mammalian models for Duchenne muscular dystrophy were published.
The number of downloads for each SOP is being monitored since 2010. However, the monitoring system became reliable only after 2011. Data presented refer to 2011 and following years (data collection by Michael Hails, Newcastle University). Since new SOPs are added at irregular intervals to the website, we record the number of downloads per year and divide it by the average number of SOPs present on the web that year. As evidenced in the figure below, the scores recorded during 2011 remained stable in the following years. Most frequently viewed SOPs (with a total number of visits in the 4 years between 100 and 400) are histological protocols for mdx, followed by the one on data handling (recovery score) and by functional and behavioural protocols for mdx.

SOP Downloads 2011-2014

Average number of downloads per SOP per year

Expansion to other disease areas

Additional funding to start discussions on research quality in congenital muscular dystrophies (CMD) was secured by an AFM grant (nr. 16297, PI: Prof. Markus Rüegg, TREAT-NMD Task Force, partners: Prof. Volker Straub, TREAT-NMD Task Force and Dr. Anne Rutkowski, CureCMD). A working-workshop was organized in Washington, DC on 21 April 2013, where stakeholders from CMD-focused research, industry and patient organizations were invited. The workshop reached a consensus on the elaboration of standard protocols for isolation and immortalization of cell lines, zebrafish based drug screenings and preclinical efficacy testing in small and big mammals (mice and dogs) in a selected set of meaningful outcome measures. Four protocols are planned to be published with video-support in the Journal of Visualized Experiments -JoVE, under the coordination of Dr. Mike Lawlor (Children’s Hospital, Milwakee, USA).Additional protocols will be published online. From May to December 2013, leading labs for each selected technique or mouse/dog outcome measure were contacted and involved and there are three new protocols are available online.

References

1. van der Worp, H.B., et al., Can animal models of disease reliably inform human studies? PLoS Med, 2010. 7(3): p. e1000245.

2. Vesterinen, H.M., et al., Improving the translational hit of experimental treatments in multiple sclerosis. Mult Scler, 2010. 16(9): p. 1044-55.

3. Prinz, F., T. Schlange, and K. Asadullah, Believe it or not: how much can we rely on published data on potential drug targets? Nat Rev Drug Discov, 2011. 10(9): p. 712.

4. Benatar, M., Lost in translation: treatment trials in the SOD1 mouse and in human ALS. Neurobiol Dis, 2007. 26(1): p. 1-13.

5. Landis, S.C., et al., A call for transparent reporting to optimize the predictive value of preclinical research. Nature, 2012. 490(7419): p. 187-91.

6. Kilkenny, C., et al., Improving bioscience research reporting: the ARRIVE guidelines for reporting animal research. PLoS Biol, 2010. 8(6): p. e1000412.

7. Festing, M.F. and D.G. Altman, Guidelines for the design and statistical analysis of experiments using laboratory animals. ILAR J, 2002. 43(4): p. 244-58.

8. Grounds, M.D., et al., Towards developing standard operating procedures for pre-clinical testing in the mdx mouse model of Duchenne muscular dystrophy. Neurobiol Dis, 2008. 31(1): p. 1-19.

9. Shineman, D.W., et al., Accelerating drug discovery for Alzheimer's disease: best practices for preclinical animal studies. Alzheimers Res Ther, 2011. 3(5): p. 28.

10. Ludolph, A.C., et al., Guidelines for preclinical animal research in ALS/MND: A consensus meeting. Amyotroph Lateral Scler, 2010. 11(1-2): p. 38-45.

11. Nagaraju, K. and R. Willmann, Developing standard procedures for murine and canine efficacy studies of DMD therapeutics: report of two expert workshops on "Pre-clinical testing for Duchenne dystrophy": Washington DC, October 27th-28th 2007 and Zurich, June 30th-July 1st 2008. Neuromuscul Disord, 2009. 19(7): p. 502-6.

12. Willmann, R., J. Dubach, and K. Chen, Developing standard procedures for pre-clinical efficacy studies in mouse models of spinal muscular atrophy: report of the expert workshop "Pre-clinical testing for SMA", Zurich, March 29-30th 2010. Neuromuscul Disord, 2010. 21(1): p. 74-7.

13. Willmann, R., et al., Enhancing translation: guidelines for standard pre-clinical experiments in mdx mice. Neuromuscul Disord, 2011. 22(1): p. 43-9.

14. Willmann, R., et al., Mammalian animal models for Duchenne muscular dystrophy. Neuromuscul Disord, 2009. 19(4): p. 241-9.

 
12 Apr 2017