PRESENTATION DECK Moving science forward to recreate Human Organs Human Organ Project, Inc. www.humanorganproject.org.

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Presentation transcript:

PRESENTATION DECK Moving science forward to recreate Human Organs Human Organ Project, Inc.

Current realities facing organ failure patients Status of new emerging science based solutions Recent breakthroughs, including  Functional organ advances  Successful living human tissue transplants The goal: Create “de novo” human organs  New organs created from patient’s own cells outside the body, able to be transplanted back to the patient The path to get there  First, stimulate great foundational science  Then, translate that to broad clinical use in humans Overview of Presentation

Patients face unacceptable wait times for organs Health care costs for organ transplant are not supportable when expanded to all patients Lack of treatment results from the lack of organ availability Patients who receive an organ transplant face a lifetime of immunosuppression therapy and the resulting side effects  Risk of infection The Current Paradigm for Organ Transplant is Failing Humanity

Historically the number of patients on the transplant waiting list far exceeds the number of actual organs donated. Wait times often become an impactful critical factor (OPTN/SRTR 2010 Annual Report and 2012 website data) Current Shortage Realities Facing Patients Seeking an Organ Transplant US Waiting List (3/30/2012) ALLKidneyLiverHeartLung Candidates113,69391,69316,1083,1661,644 Additions per year 50,50033,58111,3023,3962,405 Actual transplants 28,66216,8996,2912,3331,770 Waiting time (most common duration) In each of these organ types, typical wait time is 1-2 years We can drive the science for de novo human organs, and save more lives

Organ and Survival TypeFollow-up Period 5 Years10 Years Tx Tx Kidney: Deceased Donor Patient Survival82.7%62.0% Kidney: Living Donor Patient Survival91.6%77.6% Liver: Deceased Donor Patient Survival73.6%60.4% Liver: Living Donor Patient Survival80.8%67.4% Heart Patient Survival75.0%56.4% Lung Patient Survival54.1%28.8% Unadjusted Patient Survival at 5 Years and 10 Years (%) Source: OPTN/SRTR Data as of October 1, Multi-organ transplants are excluded except where specified. Living donor transplants excluded unless explicitly listed We can drive the science for de novo human organs, and improve survival rates Survival Rates for Current Transplant Methods

Patients/Insurers also face significant, continually increasing costs associated with organ transplant. The average annual cost of immunosuppressive drugs is more than $11,000, and in some instances can be as much as $25,000 depending upon the specific medication regimen. (ASTS Fact Sheet) Current Cost Realities Facing Patients Seeking an Organ Transplant We can drive the science for de novo human organs, and lower health care costs

Most published studies report that the impact of transplantation on recipients’ Quality of Life post transplant vs. the actual pre-transplant organ failure period is, in general, associated with improvement. (From the Vanderbilt University Transplant Center, Nashville, Tennessee)  Objective assessment of functioning by clinical caregivers showed improvement  Subjective QOL improvement measured by the accepted surveys (SF-36 and the PAIS) as reported by recipients  Aggregate observed and reported incremental improvement continued for up to 5 years Human Organ Project believes that transplant of recreated organs from the patients own cells will result in a improved Quality of Life similar to that of their pre-transplant organ failure period. Current Quality of Life (QoL) Realities For Recipients of an Organ Transplant Despite the good Quality of Life improvements reported, post transplant Quality of Life when compared to the pre-transplant organ failure period and the normal healthy population seems less than expected. (2006 European Society for Organ Transplantation)  Still a deficit in the physical component compared with the normal population  Coping issues with symptoms and side effects of Immunosuppression  Perceived fear regarding Transplant Rejection  Coping issues with higher level of professional medical attention and expenses We can drive the science for de novo human organs, and improve quality of life

Scientists have developed early working rat hearts  Doris Taylor, Univ. of Minnesota Functional liver has been generated from animal liver scaffold  Shay Soker, Wake Forest Institute for Regenerative Medicine De novo human trachea has been transplanted into four patients  Paolo Macchiarini, Univ. of Barcelona De novo bladder has been transplanted into dozens of patients  Tony Atala, Wake Forest Institute for Regenerative Medicine 3D Bioprinted blood vessels and nerve grafts are in development  Gabor Forgacs, Univ. of Missouri Human Organ Project believes that the science of de novo organ creation is near a tipping point that can be triggered with additional research funding Tremendous Gains Have Been Made in the Early Science for Organ Tissue Engineering

We can stimulate faster progress Provide funding to top tier researchers Our strategy will be to drive:  Early research with best chance of moving quickly  Actual human clinical use of promising solutions Tremendous opportunities for impact exist today, evidenced by recent breakthroughs The Road Map to Breakthroughs Can Be Defined and Can Be Successful

Define Focus Areas Based on Donations Solicit Research Proposals Independent Review by Research Review Council (RRC) Comprised of Scientific Experts Scientific grants to most promising proposals Rigorous follow up to track project success Encourage collaborative projects between top academics and centers to speed innovation Human Organ Project Program Plan

Create de novo Liver, Lung, Kidney, Heart and other organs Eliminate organ transplant waiting lists  Increase the number of transplants by 100% over current practices with de novo organs  Target to maintain or improve current five year survival rates for these new patients (70%+) Remove need for donated and cadaver organs in transplant  After waiting lists are cleared, focus on reduction in costs  Achieve near 100% of transplants from de novo organs  Reduce health care costs and need for immunosuppression Human Organ Project Goals By achieving these targets, The transplant waiting list can be cleared completely.