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Clues to Aging Through  Progeria Research <ul><li>Leslie  B.  Gordon, MD,   PhD </li></ul><ul><li>Medical Director,  </li>...
<ul><li>Premature “Aging Disease” </li></ul><ul><li>Rare: frequency 1/4-8 million </li></ul><ul><li>Onset  avg.  9 mo nths...
1998 - There Was No Hope… <ul><li>No Research Funding, No Non-profit Driving Research </li></ul><ul><li>Was this a genetic...
1999: The Progeria Research Foundation <ul><li>Mission </li></ul><ul><ul><li>Cause </li></ul></ul><ul><ul><li>Treatment </...
Addressing the Needs of Scientists, Physicians, Families Cell/Tissue Bank : 111 Search For The Gene Consortium Established...
Financial Profile: Income
Financial Profile: Expenses
Chapters, Individual Donors and Volunteers Partnerships create powerful forces for change! PreventionGenetics Private Foun...
 
Normal Aging Nuclear defects Progeria and normal aging: We all make progerin Progerin Premature Aging Progeria Coronary Ve...
Progerin  in  N ormal  A ging <ul><li>Progerin is expressed in normal fibroblast cultures at low levels compared to Proger...
FTIs Normalize Progeria Cells Normal  Fibroblast Progeria  Fibroblast Progeria  Fibroblast  + FTI , 72 hrs. Capell et al 2...
May, 2007 the First-ever Treatment Trial Begins at Children’s Hospital Boston
From Gene Finding to Clinical Trial <ul><li>A Home Base for Children/Families/Physicians  </li></ul><ul><li>Research-relat...
Avenues for Treatments and Cure  for Progeria   Drug Development Pathway Based Basic Biology Stem Cell Therapy Gene Therap...
CMV Ex 11 Ex 12 GFP sv40 dsRed STOP A high-throughput drug screen for progeria Tom Misteli, PhD aberrant correct
Key Points <ul><li>Model for translational medicine </li></ul><ul><li>Clinical Treatment Possibilities </li></ul><ul><ul><...
Thank  you! <ul><li>Together, we WILL find the cure. </li></ul>
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Progeria Research Foundation at Partnering for Cures

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The Progeria Research Foundation presents its research and development strategy at the 2009 Partnering for Cures conference in New York.

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Progeria Research Foundation at Partnering for Cures

  1. 1. Clues to Aging Through Progeria Research <ul><li>Leslie B. Gordon, MD, PhD </li></ul><ul><li>Medical Director, </li></ul><ul><li>The Progeria Research Foundation </li></ul><ul><li>Associate Professor, </li></ul><ul><li>Warren Alpert Medical School of Brown University and Hasbro Children’s Hospital , Providence, RI </li></ul><ul><li>Staff Scientist, </li></ul><ul><li>Children’s Hospital Boston and Harvard University, Boston, MA </li></ul>Audrey S . Gordon, Esq. President and Executive Director, The Progeria Research Foundation
  2. 2. <ul><li>Premature “Aging Disease” </li></ul><ul><li>Rare: frequency 1/4-8 million </li></ul><ul><li>Onset avg. 9 mo nths o ld </li></ul><ul><li>Uniformly Fatal </li></ul><ul><ul><li>CVD </li></ul></ul><ul><ul><li>average age 13 years </li></ul></ul>
  3. 3. 1998 - There Was No Hope… <ul><li>No Research Funding, No Non-profit Driving Research </li></ul><ul><li>Was this a genetic disease? We did not know. </li></ul><ul><li>No Central Source of Clinical Information </li></ul><ul><li>No Treatment Prospects </li></ul><ul><li>No Place for Families and Physicians to go for help </li></ul>
  4. 4. 1999: The Progeria Research Foundation <ul><li>Mission </li></ul><ul><ul><li>Cause </li></ul></ul><ul><ul><li>Treatment </li></ul></ul><ul><ul><li>Cure </li></ul></ul>www.progeriaresearch.org
  5. 5. Addressing the Needs of Scientists, Physicians, Families Cell/Tissue Bank : 111 Search For The Gene Consortium Established 2002 International Patient Registry/Medical Database: 79 HGPS Publications 2008 2000 1950 Research Funding : 27 Scientific Meetings: 5
  6. 6. Financial Profile: Income
  7. 7. Financial Profile: Expenses
  8. 8. Chapters, Individual Donors and Volunteers Partnerships create powerful forces for change! PreventionGenetics Private Foundations & Corporations Families
  9. 10. Normal Aging Nuclear defects Progeria and normal aging: We all make progerin Progerin Premature Aging Progeria Coronary Vessel 93-year-old Coronary Vessel
  10. 11. Progerin in N ormal A ging <ul><li>Progerin is expressed in normal fibroblast cultures at low levels compared to Progeria cultures (Scaffidi et al, 2005) </li></ul><ul><li>Progerin is expressed in normal skin biopsies, and biopsy microvasculature, and increases with age ( McClintock et al, 2007) </li></ul>
  11. 12. FTIs Normalize Progeria Cells Normal Fibroblast Progeria Fibroblast Progeria Fibroblast + FTI , 72 hrs. Capell et al 2005; Glynn et al, 2005; Toth et al, 2005; Young et al, 2005 FTIs Improved Disease in Progeria Mice Biology-based Treatment
  12. 13. May, 2007 the First-ever Treatment Trial Begins at Children’s Hospital Boston
  13. 14. From Gene Finding to Clinical Trial <ul><li>A Home Base for Children/Families/Physicians </li></ul><ul><li>Research-related Programs </li></ul><ul><li>Potential Treatment Based on Basic Biology </li></ul><ul><ul><li>In vitro and in vivo preclinical testing </li></ul></ul><ul><li>Expert Clinical Team </li></ul><ul><li>Drug Company Collaboration </li></ul><ul><li>Funding </li></ul>
  14. 15. Avenues for Treatments and Cure for Progeria Drug Development Pathway Based Basic Biology Stem Cell Therapy Gene Therapy Clinical Research
  15. 16. CMV Ex 11 Ex 12 GFP sv40 dsRed STOP A high-throughput drug screen for progeria Tom Misteli, PhD aberrant correct
  16. 17. Key Points <ul><li>Model for translational medicine </li></ul><ul><li>Clinical Treatment Possibilities </li></ul><ul><ul><li>Drug Development – </li></ul></ul><ul><ul><li>High Throughput Assay </li></ul></ul><ul><li>Implications for Aging and Atherosclerosis </li></ul><ul><li>Collaboration is Key </li></ul>
  17. 18. Thank you! <ul><li>Together, we WILL find the cure. </li></ul>

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