Creativity in a Government Health Service: the challenge and the triumph

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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

1 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)

2 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

3 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

4 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 [?]

5 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 /2007: Two FDA approved drugs, thousands of patients treated world-wide 2013: South Australian Health and Medical Research Institute (SAHMRI) [

6 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 /2007: Two FDA approved drugs, thousands of patients treated world-wide 2013: South Australian Health and Medical Research Institute (SAHMRI) [

7 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

8 Commercialisation of LDRU Intellectual Property 1991: Agreement with CSL Ltd (Australia) develop therapies for five LSDs 1992: Pilot production of MPS VI enzyme: rh4s : 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

9 2011 ERT for Bowen [age 7 to 17 years]: From looking for carers to looking for careers.. [father 2006] Obtained provisional driving license Gained University entrance to degree course in Politics/Arts

10 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

11 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

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

13 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.

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

15 LDRU Successes & Incentives: above the line outcomes early diagnosis and effective therapy for LSD patients Quality science for clinical outcomes 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

16 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 /2007: Two FDA approved drugs, thousands of patients treated world-wide 2013: South Australian Health and Medical Research Institute (SAHMRI) [

17 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

18 LDRU on L6 12 January 2012 Complete December 2013

19 SAHMRI June 2013 to open 20 December 2013

22 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

23 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 CSL; TKT, Shire; Genzyme; Pharming BV; BioMarin Thank you 2005

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

26 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

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