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