Senior Officer

California Institute for Regenerative Medicine

DATE GOES

HERE

Considerations for the Use of Bio-

Specimens in Induced Pluripotent Stem

(iPS) Cell Research

Specimen Science: Ethics and Policy Implications

Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics

Cambridge MA

November 16, 2015

Geoffrey Lomax Dr. PH

Specimen Science: Considerations for

Induced Pluripotent Stem Cells (iPSCs)

• The value of iPSCs for addressing patients unmet

medical needs

• Considerations for the collection and management of

bio-specimens and derived cell lines:

o Donor re-contact / return of research finding

o Withdrawal from research

o Sensitive uses of cells

Our Mission

Accelerating stem cell

treatments to patients with

unmet medical needs.

MIS

SIO

N

iPSCs Tools for Addressing Patients

Unmet Medical Needs

Heart cells Brain cells

Gut cells Liver cells

Insulin-producing cells

Skin Cells

Reprogram

Blood

hiPSC

iPSCs Tools for Disease Modeling and

Drug Discovery

Sick brain cells Neurodegenerative

disease

Model disease

Clinical trial

Patients

OH

O

O

O

Drug development

hiPSC

Drug

Screen

Repository

Candidate drug

CIRM iPSC Banking Initiative

Seven Tissue Collection Programs Across Four Universities:

One hiPSC

Derivation Site:

Repository and Distribution:

Donor Re-contact / Return of Research

Findings

Criteria for return of results in genomic studies not satisfied:

• Genetic Instability: iPS cells have demonstrated significant genetic

variability upon reprogramming and subsequent culture.

• Validation Criteria: Protocols do not exist to harmonize results from

research laboratories utilizing iPS cell lines with clinical (Clinical

Laboratory Improvement Amendments (CLIA)-approved) laboratories

necessary to validate findings.

• Etiologic Complexity: Genetically complex conditions, such as

neurological and heart disease, result from a poorly understood

confluence of pleiotropic gene effects.

Donor Re-contact / Return of Research

Findings

But we do suggest:

In the future, we may want to contact you to (1) obtain additional

samples or updates on your health or (2) inform you about

significant new findings that may impact you, or (3) to get

your permission for research not covered in this consent form.

Donor consents to re-contact

Withdraw From Research

Donors are informed:

Primary specimens (skin blood) may be de-identified OR

destroyed

The code linking the iPSC line to the donor can be

removed

Once derived the iPSC line cannot be withdrawn

Sensitive Uses of iPSCs

Separate donor approval required for gamete or embryo

research

Educational Materials Supplement Donor

Consent

Use of Existing Bio specimens in iPSC

Derivation

Senior Officer

California Institute for Regenerative Medicine

DATE GOES

HERE

Considerations for the Use of Bio-

Specimens in Induced Pluripotent Stem

(iPS) Cell Research

Specimen Science: Ethics and Policy Implications

Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics

Cambridge MA

November 16, 2015

Geoffrey Lomax Dr. PH