Phase II of the NIH Center for Regenerative Medicine: The Common Fund is supporting the cutting edge work of Dr. Kapil Bharti in the National Eye Institute (NEI) to develop induced pluripotent stem cell (iPSC)-based therapies for advanced macular degeneration. At the same time, the NIH National Center for Advancing Translational Sciences (NCATS) has been working with stem cell experts to move translational methods forward via a centralized effort.
NCATS is establishing a new facility that will have three major goals:
- Establish detailed Quality Control (QC) standards to define differentiated cell types and pluripotency
- Develop methods to assess heterogeneity in cultured cells derived from iPSCs
- Develop standardized methods to produce mature cells meeting the QC standards above
Importantly, scientists at NCATS will engage extramural experts in stem cell biology so that all of these efforts will be carried out in a collaborative manner with leading laboratories in the US and abroad.
Details are being finalized, with launch of the facility expected later this year. Details will be made available on the NIH Common Fund website on this page: http://commonfund.nih.gov/stemcells/index.
Update on NIH CRM
The NIH Center for Regenerative Medicine (NIH CRM) is at a transition point. The goal of NIH CRM is to work through hurdles to the development of induced pluripotent stem cell (iPSC) therapies. NIH CRM plans to do this in two ways:
- A Therapeutic Challenge award to Dr. Kapil Bharti (National Eye Institute) will advance efforts to develop an iPSC therapy for age-related macular degeneration (AMD), a leading cause of blindness in the elderly (Read more about Dr. Bharti’s research here). Importantly, Dr. Bharti’s research will not only work towards the goal of a Phase 1 clinical trial for using patient-derived iPSCs in AMD, but will navigate through methodological and regulatory challenges that may be relevant to the broader iPSC community. Knowledge gained through this research will ultimately pave the way for other researchers aiming to move iPSC therapy to the clinic for the treatment of a wide variety of diseases.
- NIH CRM is working with the National Center for Advancing Translational Sciences (NCATS) to identify, prioritize, and address the most pressing challenges facing the extramural iPSC research community. These challenges will be identified via analyses of relevant current research supported by NIH and others, as well as discussions with academic and industry investigators actively pursuing translational projects.
NIH hosted a virtual workshop in May 2014 to gather input from scientific experts on iPSC research with the ultimate goal of translational therapies (Read the workshop summary here). This workshop extended from several sets of recommendations produced by the stem cell community over the past two years (see below) and discussed where strategic investments on the part of NIH to overcome these barriers could have the greatest impact within 4 years. The workshop helped to prioritize the needs voiced by the iPSC research community, and these recommendations will be instrumental in charting a future course for NIH CRM. Objectives stemming from this workshop and an outline of planned NIH CRM-NCATS activities will be developed and posted on this website this fall.
Recommendation documents that served as a starting point for the recent CRM workshop are listed below.
- Stem Cells: Future Scientific and Medical Opportunities, a report by the ASCB Stem Cell Task Force, November 2013. http://www.ascb.org/stemcellrevolution
- UK Strategy for Regenerative Medicine. http://www.mrc.ac.uk/news-events/publications/regenerative-medicine-strategypdf/
- Key Tools and Technology Hurdles in Advancing Stem-Cell Therapies, a White Paper by California Institute for Regenerative Medicine, Alliance for Regenerative Medicine, Cell Therapy Catapult, June 2013. http://www.cirm.ca.gov/sites/default/files/files/funding_page/Key-Tools-Tech-Hurdles-in-Advancing-Stem-Cell-Therapies.pdf
- Stem Cell Research: Trends and Perspectives on the Evolving International Landscape. Jointly prepared by EuroStemCell, Kyoto University’s Institute for Integrated Cell-Material Sciences (WPI-iCeMS), and Elsevier. http://www.elsevier.com/online-tools/research-intelligence/resource-library/resources/stem-cell-research-trends-and-perspectives-on-the-evolving-international-landscape
In 2010, the NIH Common Fund launched the NIH Center for Regenerative Medicine (NIH CRM) to serve as a national resource for stem cell science to accelerate the development of new medical applications and cell-based therapies. NIH CRM focuses on induced pluripotent stem cells (iPSCs), which are generated by coaxing adult cells into reverting back to an embryonic stem cell-like state, which then can generate a multitude of different cell types for use in therapies or screening. These cells offer the potential of therapeutic strategies using a patient’s own cells, thereby avoiding immune rejection complications typically associated with transplanted cells from other sources. From Fiscal Year 2010 to Fiscal Year 2013, the NIH CRM supported pilot projects within the NIH Intramural Research Program to bring unique resources at the NIH to bear to help translate iPSC research into the clinic. In addition, the NIH CRM has developed multiple stem cell lines, set up contracts for stem cell services and storage, developed standard consent forms, compiled the supporting protocols and standard operating procedures used to derive, culture, and differentiate stem cells into different cell types, and developed training courses. In 2014, the NIH CRM awarded a Therapeutic Challenge Award to Dr. Kapil Bharti (NEI) to move his work beyond the pilot stage toward the clinic. Dr. Bharti’s research focuses on age-related macular degeneration (AMD), a leading cause of blindness in the elderly. Dr. Bharti’s Therapeutic Challenge project will use iPSC derived retinal pigment epithelium (RPE) cells, the type of cell damaged in AMD, in preclinical efficacy and safety studies. While Dr. Barti will work towards the goal of a Phase 1 clinical trial using patient-derived cells, the methodological and regulatory challenges that must be overcome will be relevant to the broader community.