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

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

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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 John J Hopwood Lysosomal Diseases Research Unit (LDRU) Womens

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ATSE Health Forum: Friday 22nd 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)

1978 - 2013 2013 -

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….. 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
  • Acknowledgements
  • LDRU success … ‘above the line outcomes’
  • South Australian Health & Medical Research Institute - SAHMRI

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

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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
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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 [?]
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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 22nd November 2013

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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 22nd November 2013

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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

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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 1999

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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

2011

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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

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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

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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
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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.

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How then to measure IP Commercialisation? ….. “a return without further exertion”

…Shaun Berg, October 2008 ….this is an ‘above the line’ outcome

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LDRU Successes & Incentives:

  • 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

“early diagnosis and effective therapy for LSD patients”

1988 1993 1998

2005

… ‘above the line outcomes’

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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 22nd November 2013

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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

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LDRU on L6 12 January 2012

Complete December 2013

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SAHMRI June 2013 to open 20 December 2013

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SAHMRI November 2013 to open 20 December 2013

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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

1988 1993 1998

“Thank you”

2005

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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
  • linked commercialisation, research/development & validation steps

“early diagnosis and effective therapy for LSD patients”

1988 1993 1998

2005 “early diagnosis and effective therapy for LSD patients”

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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

ATSE Health Forum: Friday 22nd November 2013

Our LDRU experience from: … "Doing Drugs: selling LSD to the US market”