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
Dec 24, 2015
Professional Guidelines for Stem Cell TranslationISSCR
George Q. DaleyDirector, Stem Cell Transplantation Center
Children’s Hospital/ Harvard Medical SchoolISSCR President 2007-2008
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
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
Why are stem cells different from drugs?
Stem cell products are entirely novel
Stem cells persist in the patient
Animal models only partially predictive
– 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
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
Declaration of Helsinki (10/19/13)http://jama.jamanetwork.com/article.aspx?articleid=1760318&utm_source=silverchair+information+systems&utm_medium=email&utm_campaign=jama%3aonlinefirst10%2f19%2f2013
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