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Regenerative Medicine Program (RMP) Program Highlights

Current good manufacturing practices induced pluripotent stem cell line now available to enable development of new therapies and accelerate early-stage clinical research

Induced pluripotent stem cells (iPSCs) represent a powerful new avenue for potentially treating ailments ranging from Alzheimer's disease to spinal cord injury. Significant progress with stem cell therapy in mice is already underway; for instance, researchers have reversed diabetic conditions in mice using iPSC-generated insulin-producing beta-cells. Translating these studies into humans is the next challenge. The RMP supported, through a contract with Lonza Walkersville Inc., the development of a current good manufacturing practice (cGMP) clinical-grade iPSC line from human umbilical cord blood CD34+ cells. cGMP is a set of stringent regulations enforced by the US Food and Drug Administration that ensures each batch of cells produced will meet quality and safety standards required for potential clinical use. These cells were fully characterized for safety, sterility, plasmid clearance, endotoxin levels, karyotype and many other factors and are described in two publications by Behnam Baghbaderani and colleagues in Stem Cell Reports and Stem Cell Rev. The cGMP clinical-grade iPSC line – along with a parallel, identical, non-cGMP research grade iPSC line – is available to the research community through RUCDR Infinite Biologics at Rutgers University.

Use this link to request any iPSC line generated by the RMP
Read an NIH press release describing the these cells

 

Kapil Bharti’s Therapeutic Challenge Award to develop a stem-cell based therapy for macular degeneration – a two-year update

On September 18, 2015 Dr. Kapil Bharti of the National Eye Institute (NEI) and his research team presented an update to his Therapeutic Challenge award to develop a stem-cell based therapy to treat age-related macular degeneration (AMD). In AMD patients Retinal Pigment Epithelium (RPE) cells become damaged and lose function. Replacement of damaged RPE cells with functional stem-cell-derived RPE cells is a viable treatment believed to restore or improve vision. Inducible pluripotent stem cells (iPSCs) are stem cells that have been reprogramed from adult cells and can develop into any cell type in the body. For clinical applications iPSCs are to be manufactured using strict guidelines called current good manufacturing practice (cGMP) to ensure quality, purity, and safety of cells. Dr. Bharti’s team has developed a streamlined manufacturing process to generate transplant-ready cGMP-grade RPE cells from iPSCs and the entire manufacturing process takes slightly under 150 days. His team is currently performing pre-clinical animal toxicity, efficacy, and transplantation device compatibility studies in preparation for submitting a phase I Investigational New Drug (IND) application with the Food and Drug Administration in 2017. Learn more about Dr. Bharti’s research at NEI.

 

The Common Fund Issues New FY 2012 Awards in Regenerative Medicine

Thirteen NIH Intramural Research Program (IRP) laboratories have been funded on a competitive basis in fiscal year 2011 to conduct pilot projects in support of the NIH CRM mission.

View funded research…

 
NIH Center for Regenerative Medicine and NIH Clinical Center join forces in an effort to harmonize informed consent for iPS cell-based research and therapies

NIH Center for Regenerative Medicine and NIH Clinical Center join forces in an effort to harmonize informed consent for iPS cell-based research and therapies

The NIH Center for Regenerative Medicine has collaborated with the NIH Clinical Center Department of Bioethics on a manuscript that considers the challenges to obtaining informed consent to derive induced pluripotent stem (iPS) cells from donated tissue samples for research and therapeutic purposes. The manuscript provides concrete recommendations, along with a model consent form, in an effort to broadly harmonize informed consent, which currently varies considerably between tissue collection sites.

The promise of iPS cells in clinical therapies to replace damaged or diseased tissues, and as a resource for understanding a wide range of diseases and discovering candidate therapeutic drugs, is huge. However, to maximize their utility while safeguarding the donors that provide the starting material (e.g. skin) that the iPS cells are derived from, it is critical that informed consent is carefully considered now while the field is still nascent. This is particularly challenging when all the potential downstream uses of iPS cells are not yet know and can result in narrowly restricting the informed consent to a specific application at hand. Conversely, re-contacting donors/study participants indefinitely to re-consent to additional uses of the iPS cells, or to collect additional samples or information on their health, is not ideal either. This manuscript makes a cogent argument for a middle ground that balances the perspectives of a variety of stakeholders and arrives at a model consent form for the prospective collection of fresh specimens from which to derive iPS cells. Some important features include:

  • Research purposes are “open-ended” within the boundaries of all applicable laws and policies and may include transplanting cells or tissues made from iPS cells in to another patient to treat a disease
  • The iPS cells and participant’s medical information will be shared with other researchers to benefit medical research and society
  • Donors may be re-contacted for additional samples, medical information, or to consent to additional iPS cell uses not originally anticipated, but they do not need to oblige
  • A provision is included for donors to opt out of being re-contacted by researchers for any reason
  • Pediatric re-consent is incorporated to allow children to be informed upon reaching adulthood of the ability to review the previous consent and continue to participate in the study or not
  • Explicit language is included indicating that direct medical benefits for the donor are unlikely and no financial benefits will result from any commercial products developed from the iPS cells
  • Withdrawal at different stages of the research process is allowed. A request can be made to destroy any leftover original samples; however, any iPS cells already derived cannot be destroyed and any shared with other researchers cannot be retrieved, though the codes that link the sample to the donor can be removed

The implementation of a consistent approach to informed consent for iPS cell derivation and use across research/medical institutions in these early days as proposed in this manuscript could potentially be invaluable to providing stem cell researchers with crucial access to high-quality, thoroughly documented materials and resources.

Read more about the NIH CRM program

Reference:
Lowenthal J, Lipnick S, Rao M, Hull SC. Specimen collection for induced pluripotent stem cell research: Harmonizing the approach to informed consent. Stem Cells Translational Medicine. Published online May 8, 2012; doi: 10.5966/sctm.2012-0029.

 

 

 

The Common Fund Issues New FY2011 Awards to Spur Regenerative Medicine Research

Thirteen NIH Intramural Research Program (IRP) laboratories have been funded on a competitive basis in fiscal year 2011 to conduct pilot projects in support of the NIH CRM mission.

View funded research…

 

The Common Fund Issues First Awards in FY2010

Eleven laboratories in the NIH Intramural Research Program (IRP) are being supported to conduct pilot studies on the clinical applications of induced pluripotent stem cells (iPSCs).

View funded research…

 
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