Creativity in a Government Health Service: the challenge and the - PowerPoint PPT Presentation

ATSE Health Forum: Friday 22 nd November 2013 Creativity in a Government Health Service: the challenge and the triumph …… therapeutics for lysosomal storage disorder patients John J Hopwood Lysosomal Diseases Research Unit (LDRU) Women’s and Children’s Hospital (WCH) South Australian Health and Medical Research Institute (SAHMRI) 2013 - 1978 - 2013

“Creativity in a Government Health Service: the challenge and the triumph” ….. a brief Outline Headings:  What are lysosomal storage disorders (LSD)  Early research and applied science experiences  Translation of LDRU research and IP … ideas through to drugs  Measures of success  LDRU success … ‘above the line outcomes’  South Australian Health & Medical Research Institute - SAHMRI  Acknowledgements

What are Lysosomal Storage Disorders? Characteristics:  more than 60 different LSD types  all genetically transmitted  common biochemical & clinical properties  most LSD are pre-symptomatic at birth  progressive to mostly irreversible pathology

What are Lysosomal Storage Disorders? Characteristics:  more than 60 different LSD types  all genetically transmitted  common biochemical & clinical properties  most LSD are pre-symptomatic at birth  progressive to mostly irreversible pathology  classical LSD pathology: death often before 20years  broad & variable clinical outcomes within each type  total incidence: classical 1 in 5,000; non-classical 1 in 200 [?]

ATSE Health Forum: Friday 22 nd November 2013 LDRU applied science experiences Timing of research and its translation to outcomes:  1978: Lysosomal Diseases Research Unit established in WCH: …with a goal/focus: ‘early diagnosis and effective therapy’  1978/2013: Papers 436; PhDs 23; Research funding > $60m; Patents 48  2006/2007: Two FDA approved drugs, thousands of patients treated world-wide  2013: South Australian Health and Medical Research Institute (SAHMRI) … [www.sahmri.com]

ATSE Health Forum: Friday 22 nd November 2013 LDRU applied science experiences Timing of research and its translation to outcomes:  1978: Lysosomal Diseases Research Unit established in WCH: …with a goal/focus: ‘early diagnosis and effective therapy’  1978/2013: Papers 436; PhDs 23; Research funding > $60m; Patents 48  2006/2007: Two FDA approved drugs, thousands of patients treated world-wide  2013: South Australian Health and Medical Research Institute (SAHMRI) … [www.sahmri.com]

Commercialisation of LDRU Intellectual Property 1991: Agreement with CSL Ltd (Australia) develop therapies for five LSDs 1992: Pilot production of MPS VI enzyme: rh4S 1995: ERT efficacy/safety shown in MPS VI cats 1995: CSL withdraws – anticipated impact of gene therapy? 1998: Partnered with BioMarin (USA) for MPS VI 1999: Large scale production of enzyme achieved 2000: FDA-approve phase I/II trials to commence in MPS VI patients 2002: Phase II trials, 10 MPS VI patients in Adelaide/Oakland 2003: Phase III trials, 36 MPS VI patients in 6 international centres 2003: Natural history, 100 MPS VI patients: genotype/phenotype relationship 2004: Phase III trials completed 2005: FDA approves rh4S as ERT therapeutic drug for MPS VI

Commercialisation of LDRU Intellectual Property 1991: Agreement with CSL Ltd (Australia) develop therapies for five LSDs 1992: Pilot production of MPS VI enzyme: rh4S 1999 1995: ERT efficacy/safety shown in MPS VI cats 1995: CSL withdraws – anticipated impact of gene therapy? 1998: Partnered with BioMarin (USA) for MPS VI 1999: Large scale production of enzyme achieved 2000: FDA-approve phase I/II trials to commence in MPS VI patients 2002: Phase II trials, 10 MPS VI patients in Adelaide/Oakland 2003: Phase III trials, 36 MPS VI patients in 6 international centres 2003: Natural history, 100 MPS VI patients: genotype/phenotype relationship 2004: Phase III trials completed 2005: FDA approves rh4S as ERT therapeutic drug for MPS VI

2011 ERT for Bowen [age 7 to 17 years]:  From “ looking for carers to looking for careers ” .. [father 2006]  Obtained provisional driving license - 2011  Gained University entrance to degree course in Politics/Arts - 2011

Translation of other LDRU therapeutics Steps and outcomes for therapeutics: 1991: CSL Ltd (Australia) for MPS I, II, IIIA, IIIB, VI 1995: CSL withdraws 1996: Shire HGT (USA) for MPS I, II, IIIA, IIIB 1997: MPS II used as a model for non-CNS ERT 2002: MPS II Phase I/II ERT trial completed 2005: MPS II Phase III ERT trial completed 2006: FDA approves MPS II enzyme as therapeutic drug

Translation of other LDRU therapeutics Steps and outcomes for therapeutics: 1991: CSL Ltd (Australia) for MPS I, II, IIIA, IIIB, VI 1995: CSL withdraws 1996: Shire HGT (USA) for MPS I, II, IIIA, IIIB 1997: MPS II used as a model for non-CNS ERT 2002: MPS II Phase I/II ERT trial completed 2005: MPS II Phase III ERT trial completed 2006: FDA approves MPS II enzyme as therapeutic drug 2010: MPS IIIA phase I/II CNS ERT trials start MPS II phase I/II CNS ERT trials start

What measures IP Commercialisation outcome? Major reviews are undecided 1, 2, 3 1. KCA, “Commercialisation Metrics Survey 2007” Sept 2008 2. DEST, “National Survey of Research Commercialisation 2003/04” Aug 07 3. “Review of National Innovation System” Oct 2008

Outcome is not? measured by:  Research funds won  Concepts, IP & patents  External consultancies  Number of IP licensing & option agreements  Formation of spin-out companies  Technology-transfers  Joint ventures  These ‘below the line’ activities contribute to achieving a commercialisation outcome. They are not a measure of success.

How then to measure IP Commercialisation? ….. “a return without further exertion” …Shaun Berg, October 2008 ….this is an ‘above the line’ outcome

LDRU Successes & Incentives: … ‘above the line outcomes’  Quality science for clinical outcomes “early diagnosis and effective therapy for LSD patients”  Children receiving therapy  Royalty to the State: over $300m; returns continue into future  Two-thirds to Health and Medical Research in SA  One-third to > 20 individual contributors to LDRU technology  One-sixth re-invested into LDRU commercialisation activities 2005 1988 1993 1998

ATSE Health Forum: Friday 22 nd November 2013 LDRU applied science experiences Timing of research and its translation to outcomes:  1978: Lysosomal Diseases Research Unit established in WCH: …with a goal/focus: ‘early diagnosis and effective therapy’  1978/2013: Papers 436; PhDs 23; Research funding > $60m; Patents 48  2006/2007: Two FDA approved drugs, thousands of patients treated world-wide  2013: South Australian Health and Medical Research Institute (SAHMRI) … [www.sahmri.com]

SAHMRI Flagship Facility: Designed for a Purpose … to maximise innovation and creativity, with the purpose to bring together researchers and community for translation to health and medical outcomes with community impacts

Complete December 2013 LDRU on L6 12 January 2012

SAHMRI June 2013 to open 20 December 2013

SAHMRI November 2013 to open 20 December 2013

Acknowledgements LDRU major support National Health & Medical Research Council of Australia:  Project and Program Grants  WCH Research Foundation: Program and Project Grants  Australian Research Council: Project Grants  Wellcome Trust  Hunter ’ s Hope; Julia ’ s Hope; Isaac Foundation  Channel Seven Medical Research Fund  Sanfilippo Children ’ s Research Foundation  Sanfilippo Alliance; Swiss Sanfilippo Foundation Children ’ s Medical Research Foundation   MPS Societies of UK and US  TLH Research “ Thank you ”  CSL; TKT, Shire; Genzyme; Pharming BV; BioMarin 1988 1993 1998 2005

LDRU ‘ involvement ’ in all steps to achieve overall success…… Steps: [motivation, focus and interactions] “early diagnosis and effective therapy for LSD patients”  team work within a broad ‘ science ’ expertise  integration with diagnostic & clinical expertise  strategic collaborations outside the LDRU  drive/intensity maintained with focus/goal: “early diagnosis and effective therapy for LSD patients”  academic program with PhD students / postdoctoral scientists  relationships with patients/parents/societies  linked commercialisation, research/development & validation steps 2005 1988 1993 1998

ATSE Health Forum: Friday 22 nd November 2013 Translational Research A definition: Transfer of advancing knowledge to improve health and wellbeing Usual experience: … this transfer into practice is often an inappropriately slow and haphazard process Our LDRU experience from: … "Doing Drugs: selling LSD to the US market”