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AIDS Cure Research- Moving Into the Clinic

AIDS Cure Research- Moving Into the Clinic. Matt Sharp Long-term Survivor and AIDS Cure Activist San Francisco. Outline. Perspective Cure-related clinical trial issues Cure-related clinical enrolling Zinc finger nuclease approach My clinical experience Results SB-728 Issues

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AIDS Cure Research- Moving Into the Clinic

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  1. AIDS Cure Research-Moving Into the Clinic Matt Sharp Long-term Survivor and AIDS Cure Activist San Francisco

  2. Outline • Perspective • Cure-related clinical trial issues • Cure-related clinical enrolling • Zinc finger nuclease approach • My clinical experience • Results • SB-728 • Issues • Remaining questions

  3. Perspective • HIV cure-related research has progressed relatively quickly since the first clinic case report of Timothy Brown, who is considered now functionally cured after five years • Despite many unknowns the field is moving rapidly towards remarkable advances in HIV • Eradication/functional cure has become a new direction in HIV research and is already moving into the clinic requiring study volunteers • New drugs, vaccines, gene therapy, drug intensification trials and immune-based therapies are now being studied in humans

  4. Cure-related clinical trial issues • Recruitment in the age of effective ARV therapy • Are trials ethical? • What risks are reasonable? Protection from research injury • Informed consent • Challenging trial designs-ATI, invasive procedures • Reimbursement • Product continuation after trial completion • Grass-roots outreach and education • Community Advisory Boards to provide input into informed consents

  5. Cure-related clinical trials now enrolling • Trials now enrolling as of June 2012* • 6 immune-based or cell therapeutic • 8 therapeutic vaccine • 8 reservoir-related • Several others are being planned (ACTG, industry, etc.) • *clinicaltrials.gov • http://survivinghiv.blogspot.com/2012/05/hiv-cure-related-studies-currently.html

  6. Clinical background • Dx in 1988 with HIV w/ 409 CD4 • Sequential monotherapy throughout 90’s • Consented to be in dozens of clinical trials • Salvage patient developing multi-drug resistance • First reached undetectable 2007 w/ RAL+DRV/r yet T-cells never rebounded • Currently virally suppressed on stable HAART • Immunologic non-responder

  7. ZFN approach A kinder, gentler approach to making HIV-resistant Gene modification through zinc finger nuclease technology Disrupts the CCR5 gene in leukapheresised CD4 cells Cells modified with zinc finger nuclease introduced with adeno virus vector Modified CD4 cells; then expanded and frozen Infusion

  8. SB-728 phase 1 safety trial • First safety trial in virally suppressed immunologic non-responders • Screened and consented for SB-728 in June 09 • Apheresis procedure July 09 • “product” development-aprx. 6 weeks • Single infusion September 09 • Monthly blood draws, urine • 6 rectal biopsies-20 snips each procedure • One lymph node biopsy-off protocol

  9. My results • 6% of total cells were modified CCR5 cells • Modified cells found in gut • No clinical events (not analyzed) • No upper respiratory infections • Results after one year: • Baseline CD4 294; CD4% 14.7; CD4/CD8; • Month 12 CD4 458; CD4% 21.2; CD4/CD8; • Average annual CD4 488; CD4% 21.2; CD4/CD8; • Month 18 CD4 350; CD4%; CD4/CD8

  10. apheresis

  11. Issues • Rectal biopsies • Reimbursed $2000 over the course of one year • 5 protocol amendments w/new lengthy informed consents • No drug continuation for study volunteers yet • No safety issues • Immune system benefit?

  12. Science 13 May 2011: Vol. 332 no. 6031 pp. 784-789

  13. Remaining questions • Cure-related clinical trials will require many patients • Will healthy HIV+ people participate and enroll?If so, will participants understand informed consents? • Will product continuation be built into protocols if a benefit is found in the absence of a cure? • What is the best way to inform the community of complex and rapidly evolving scientific information? • On the road to the goal of a cure, will immunologic non-responders, long-term survivors and others be forsaken? • While there are risks there also may be benefits for INR

  14. Henrietta Lacks We must not see any person as an abstraction. Instead, we must see in every person a universe with its own secrets, with its own treasures, with its own sources of anguish, and with some measure of triumph. -Elie Wiesel The Nazi Doctors and the Nuremberg Code

  15. Imagine a World without AIDS

  16. mattsharpster@gmail.com

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