1. Professional Guidelines for Stem Cell Translation ISSCR George Q. Daley Director, Stem Cell Transplantation Center Children’s Hospital/ Harvard Medical School ISSCR President 2007-2008

2. Transplants CIBMTR HSC Transplant activity worldwide 1980-2009 '80'81'82'83'84'85'86'87'88'89'90'91'92'93'94'95'96'97'98'99'00'01'02'03'04'05'06'07'08'09 Allogeneic 20,000 25,000 35,000 30,000 15,000 10,000 5,000 0 Autologous

3. International Society for Stem Cell Research Promote safe, effective, innovative clinical translation : –Educate scientists and medical practitioners world-wide –Establish professional standards –scientific, medical, ethical –Help inform, formulate regulatory pathway –Train translational physician scientists –Educate patients and their families –Spotlight clinics marketing unproven treatments

4. Why are stem cells different from drugs? Stem cell products are entirely novel Stem cells persist in the patient Animal models only partially predictive

5. –Task force: 30 members, 14 countries –Scientists, physicians/surgeons, bioethicists, attorneys –Patients/ patient advocates 2008 –Bedrock principles –High standards of pre-clinical evidence, peer-reviewed –Rigorous quality control around cell manufacture –Scrupulous review of clinical protocol by IRB –Ensuring informed consent, patient autonomy –Rigorous long-term follow-up, reporting of negative results –Deliverable: Patient Handbook

6. Medical innovation (outside formal clinical trial) Few, seriously ill patients Written plan -- Compelling rationale; evidence of efficacy and safety -- Characterization, QC of cells; Description of mode of treatment; -- Rigorous, voluntary informed consent -- Considerations of cost, adverse events, clinical follow-up -- Institutional accountability Review by experts Imminent initiation of a clinical trials process

7. Declaration of Helsinki (10/19/13) http://jama.jamanetwork.com/article.aspx? articleid=1760318&utm_source=silverchair +information+systems&utm_medium=emai l&utm_campaign=jama%3aonlinefirst10%2f 19%2f2013

9. Promoting ethical, effective care Community and Professional standards –Reinforced thru continuing education, practice guidelines, professional societies, peer consultation –Professional accreditation/ certifications –Practice standards and legal liabilities –Tort law and medical malpractice Regulatory oversight –Legal, governmental, institutional Market forces Need to address patient, family needs