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AABB CellSource - September 2013 

September 2013


CTSCC Elections — E-VOTING Closing Soon

Ballots for the six open positions on the Cellular Therapy Section Coordinating Committee (CTSCC) will close on Sept. 20. Members of the CT Section who have not yet voted are asked to cast their votes. The results will be announced at the CT Section Business Meeting on Oct. 14 during the AABB Annual Meeting & CTTXPO in Denver. Anyone experiencing problems accessing the electronic ballot should contact celltherapy@aabb.org.

Voices of AABB CCT Membership Extended to the Asia Pacific Region

AABB enjoys a broad membership located around the globe and recognizes that time differences make it difficult for some to join in subsection teleconferences. To address this challenge, the AABB Center for Cellular Therapies has organized a group within the Cord Blood Subsection called the Asia-Pacific Group, or APG. One of the principal goals of the APG is to meet monthly via an AABB teleconference at a time that accommodates the majority of individual members in this region. Led by a regional representative, the APG will address a variety of topics and relevant items such as best practices, cell processing challenges, addressing non-conformances, and quality issues. Interested members are encouraged to enroll in the Cord Blood Subsection. Meeting information will be sent to members located in the region.

New ISBT 128 Terminology Released

ISBT 128 terminology for cellular therapy products has been revised and posted. Major changes include the addition of the source of the cells in the name of the product, separated by a comma from the type of cells; deletion of the hyphenated class names; replacement of modifiers with attributes; and the movement of some attributes into new groups. Therapeutic cells, or TCs, also have been renamed. Interested professionals may review the rationale for these revisions. A focus group will make recommendations to the Cell Therapy Coding and Labeling Advisory Group regarding implementation timelines for standards-setting organizations, including AABB.

For U.S. facilities, the United States Consensus Standard for the Uniform Labeling of Cellular Therapy Products Using ISBT 128 has been updated and is available for comment until Oct. 25, 2013. In addition to the terminology changes, it is proposed that label text for indicating the volume of the product and anticoagulant be changed. This document may be viewed here.

Researchers Create 3-D System to Study Blood Vessel Growth

Researchers from Virginia Tech and Cornell University have established a 3-D microfluidic system to study a biological process known as endothelial sprouting. This process represents an early step in new blood vessel growth called angiogenesis. The results are described in a paper published in the "Journal of Biomedical Materials Research Part A" and highlight the importance of mechanical as well as biochemical factors.

Stem Cells in Urine Can Be Directed to Become Multiple Cell Types

Researchers at Wake Forest Baptist Medical Center's Institute for Regenerative Medicine have identified stem cells in urine that can be directed to become multiple cell types. The ability to use a patient's own stem cells for therapy is considered advantageous because the cells do not induce immune responses or rejection. However, because tissue-specific stem cells are a very small subpopulation of cells, they can be difficult to isolate from organs and tissues.

Growing a Complete Ear Is a Step Closer

Scientists at Massachusetts General Hospital in Boston say they have moved a step closer to growing a complete human ear from a patient's cells. In a new development in tissue engineering, they have grown a human-like ear from animal tissue. The ear has the flexibility of a real human ear.

Induced Pluripotent Stem Cell Therapy Clinical Trial for Eye Disease Starts in Japan

The RIKEN Center for Developmental Biology in Japan announced the launch of a pilot study to assess the safety and feasibility of the transplantation of retinal pigment epithelium, or RPE, cell sheets derived from autologous induced pluripotent stem cells, or iPSCs, in patients with exudative (wet-type) age-related macular degeneration. The protocol involves the establishment of autologous iPSCs from research participants. These iPSCs will be differentiated into RPE using a novel technology that allows the epithelial cells to be transplanted in monolayer cell sheets without the use of synthetic scaffolds or matrices.

Scientists Reprogram Cells on the Computer

Scientists at the University of Luxembourg have developed a computer model that makes predictions about changing differentiated cells into completely different cell types. They assert that this can be done entirely without stem cells. The theoretical model first queries databases where a vast amount of information on gene actions and their effects are stored and then identifies the genes that maintain the stability of differentiated cells. The model suggests which genes in the starting cells need to be switched on and off again, and at what time, in order to change them into a different cell type. The researchers still need to identify the growth factors that initiate the genetic activities.

3-D Printing Can Build New Bone

Researchers can generate bone tissue by using scaffolds created through 3-D printing and living stem cells to repair/replace damaged bone. To promote bone replacement, a 3-D bioprinter creates a scaffold in the shape of the bone and coats it with adult human stem cells, which are capable of developing into many different tissue types. The printer's "ink" consists of a polymer called polylactic acid, which provides the hard, mechanical strength of the bone, and a gel-like substance called alginate, which acts as a cushioning material for the cells. The printed product can be implanted in the body, where the scaffold will degrade and be replaced by new bone within about three months.

Stem Cell Transplants Clear HIV From Two U.S. Patients

Two patients with cancer who also were infected with HIV have no trace of the virus after receiving hematopoietic stem-cell transplants, suggesting they may have been cured of the AIDS-causing infection. In one patient, there was no detection of the virus 15 weeks after stopping HIV treatment, while in the other patient, HIV has not rebounded in seven weeks, according to results by researchers at Harvard Medical School and Brigham and Women's Hospital. The team of investigators, led by Timothy Henrich, said it is too early to conclude that the two men have been cured, as the virus may be lingering in their brains or gastrointestinal tracts. Their cases are similar to that of Timothy Brown, the so-called Berlin patient, who was the first person to be cured of HIV after getting a bone marrow transplant for leukemia in 2007. Brown was a keynote speaker at AABB's 2012 Annual Meeting & CTTXPO.

Stem Cell Research Yields Down Syndrome Breakthrough

Two University of Massachusetts Medical School scientists used a naturally occurring "off switch" to shut down the chromosomes that cause Down syndrome during laboratory experiments that used human stem cells, according to a press release from the school. According to the researchers, this discovery could lead to treatment and therapy based on the chromosome that causes the condition, rather than treatment based on its symptoms. The hope is that this proof of principle will make available multiple exciting new avenues for studying Down syndrome and may lead to future research concerning "chromosome therapy."


Mitochondrial Transfer From Cell Grafts: New Potential for Regenerative Therapies

Marie Csete MD, PhD

The cell's power plants, the mitochondria, likely started life as free-living bacteria. In a process called endosymbiosis, the bacterial ancestors of mitochondria were engulfed by another cell. Both cells benefited from the cohabitation, and together they evolved as a distinct lineage. Though mitochondria and other cell types teamed up in the distant past, their co-evolutionary story is reflected in the independence of mitochondrial biogenesis from cell proliferation in modern eukaryotic cells. Given this invasive bacterial origin for mitochondria, perhaps one should not be surprised at the retained phenomenon of mitochondrial transfer between cells, which is now emerging as an important consideration in regenerative medicine. Read more


AABB Updates Web Page With Summary of Regulations in India, Singapore

The AABB International Competent Authorities web page has been updated with summaries of cellular therapy-related regulations in India and Singapore. The summaries also include links to applicable guidelines, resources and regulations.

FDA Publishes Draft Guidance on Early-Phase Clinical Trials for Cellular Therapy Products

The Food and Drug Administration's Center for Biologics Evaluation and Research, or CBER, announced a draft guidance for industry titled "Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products," in a "Federal Register" notice dated July 2. The draft guidance provides recommendations to assist sponsors in designing phase 1 trials and some phase 2 trials that focus on the initial evaluations of the safety, tolerability or feasibility of investigational cellular and gene therapy products. The document highlights product-specific considerations for the key elements of a clinical study: trial objective(s), study population, use of a control group and blinding procedures, dose selection and escalation schedules, treatment plans, monitoring, and follow-up activities. When finalized, this guidance will apply only to cellular and gene therapy products regulated under Section 351 of the Public Health Service Act as biological products.

AABB will submit comments on the draft guidance by the Nov. 22 deadline. Those interested in sharing comments or concerns may contact the Regulatory Affairs department by October 25 with feedback for consideration, including any regulatory gaps in the draft guidance.

CBER's Cellular, Tissue and Gene Therapies Advisory Committee Set to Meet in October

The FDA's Center for Biologics Evaluation and Research, or CBER, posted information about the next Cellular, Tissue and Gene Therapies Advisory Committee meeting that will be held Oct. 22-23 in Silver Spring, Md. During this meeting, which is open to the public, the committee will discuss topics that include oocyte modification in assisted reproduction for the prevention of transmission of mitochondrial disease or for the treatment of infertility and updates on guidance documents from the Office of Cellular, Tissue and Gene Therapies, or OCTGT, CBER and the FDA. Specifically, the draft guidance "Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products," mentioned above, will be discussed on Oct. 23. The FDA website provides instructions for individuals interested in presenting either written or oral statements.

FDA Releases Guidance on IND Requirements for the Use of Fecal Microbiota

The FDA announced a guidance for industry titled "Enforcement Policy Regarding Investigational New Drug Requirements for Use of Fecal Microbiota for Transplantation to Treat C. difficile Infection Not Responsive to Standard Therapies" in a "Federal Register" notice dated July 18. The document relates to investigational new drug, or IND, requirements for the use of fecal microbiota for transplantation, or FMT, to treat Clostridium difficile. The guidance explains that the FDA will exercise enforcement discretion of the IND requirements when physicians seeking to use FMT to treat C. difficile — that is unresponsive to standard therapies — obtain an adequate letter of informed consent from the patient or the affected individual's legally authorized representative. At a minimum, the informed consent should state that this use of FMT products is investigational and describe the potential risks of the treatment. The guidance does not pertain to other uses of FMT and the policies it contains only are in effect while FDA continues to consider applicable IND requirements. This guidance should be implemented immediately.

The FDA's Center for Biologics Evaluation and Research held a public workshop May 2-3 titled "Fecal Microbiota for Transplantation" and afforded scientists and physicians the opportunity to discuss concerns about the FDA's IND requirements for FMT.

FDA Office of Cellular, Tissue and Gene Therapies Announces Meeting to Discuss Standards for Cellular Therapies and Regenerative Medicine

The Office of Cellular, Tissue and Gene Therapies, or OCTGT, in FDA's Center for Biologics Evaluation and Research, announced a public workshop titled "Synergizing Efforts in Standards Development for Cellular Therapies and Regenerative Medicine Products" that will be held on Oct. 7 in Silver Spring, Md. The workshop will focus on cellular and regenerative therapies, covering topics that include the types of standards and standards-setting organizations that currently exist, the development of standards, and regulatory application. The discussion also will include ways to minimize repetition and maximize collaboration when writing standards for both fields. AABB has been invited to present at this meeting. The workshop will allow time for discussion of high-interest subjects associated with standards development for cellular therapies and regenerative medicine. Individuals should register to attend by Sept. 23 as seating is limited; streaming of the public workshop webcast also is available.

AABB Submits Comments to FDA; Encourages Recognition of Differences Among Quality Agreements

In comments sent to the FDA on the draft guidance for industry titled "Contract Manufacturing Arrangements for Drugs: Quality Agreements," AABB expressed support for the use of written quality agreements. The association's approbation is based upon its use of a quality framework to develop standards, including "requirements for written agreements between facilities to define supplier and customer expectations, reviews of the agreements to incorporate changes and communication of such changes, and clear delineation of the responsibilities related to the requirements of the Standards." However, AABB also encouraged the FDA to recognize the variability in quality agreements that different facilities will use to describe contract manufacturing arrangements, noting that there is no single template that works for all facilities, even for those facilities that are contracting out the same operation.

FDA Issues Guidance for Industry on Clinical Study Monitoring

The FDA announced the availability of a guidance for industry titled "Oversight of Clinical Investigations — A Risk-Based Approach to Monitoring" in an Aug. 7 "Federal Register" notice. This guidance is designed to assist sponsors in developing risk-based monitoring strategies and plans for clinical investigations of drugs, biologics, medical devices and combinations of these interventions. It also is intended to improve protection of human subjects and the quality of clinical trial data by highlighting critical study parameters and strategies that rely on a combination of oversight activities. Additionally, the guidance highlights that sponsors may use a variety of approaches for monitoring clinical investigation conduct and makes specific recommendations for certain aspects of a clinical monitoring plan that include communication of monitoring results, management of noncompliance, ensuring quality monitoring and handling amendments to clinical monitoring plans.


Transplantation of Human Fetal-Derived Neural Stem Cells Improves Cognitive Function Following Cranial Irradiation

Researchers at the University of California Irvine showed that rats that received human neural stem cell transplants after brain irradiation improved hippocampal spatial memory as well as contextual fear conditioning performance, a brain function that relies on intact amygdala function. Both the amygdala and the hippocampus are parts of the brain involved with memory formation. The research demonstrated proof of principle for ameliorating cognitive dysfunction when the cells were injected two days after irradiation, a model that is similar to a potential clinical intervention given to treat brain cancer patients. Such irradiation frequently leads to serious and debilitating cognitive loss in patients, ranging from pediatric patients afflicted with medulloblastoma, to adults with glioblastoma multiforme. No current solutions exist to treat this medical condition. The key therapeutic mode of action by the transplanted cells in this model is to protect and preserve neurogenesis in the hippocampus. In clinical translation, this suggests that early intervention with neural stem cells could prevent cognitive complications due to radiation therapy.

Functional Assessment of Long-term Deficits in Rodent Models of Traumatic Brain Injury

Traumatic brain injury, or TBI, ranks as the leading cause of mortality and disability in the young population worldwide. The annual U.S. incidence of TBI in the general population is estimated at 1.7 million per year, with an estimated financial burden in excess of US$75 billion a year in the United States alone. Despite the prevalence and cost of TBI to individuals and society, no treatments have completed clinical trials to achieve clinical implementation. The rapid expansion of stem cell research and technology offers an alternative to traditional pharmacological approaches targeting acute neuroprotection. However, preclinical testing of these approaches depends on the selection and characterization of appropriate animal models. E.M. Gold et al. consider the underlying pathophysiology for the focal and diffuse TBI subtypes, discuss the existing preclinical TBI models and functional outcome tasks used for assessment of injury and recovery, identify criteria for preclinical animal models in which stem cell therapies can be tested, and review these criteria in the context of the existing TBI literature.

Wnt5a Regulates Hematopoietic Stem Cell Proliferation and Repopulation Through the Ryk Receptor

A study funded in part by AABB's National Blood Foundation characterizes a pathway that regulates proliferation of hematopoietic stem cells, or HSCs. Michael Nemeth and his colleague Benjamin Povinelli observed that HSCs expressing the Ryk receptor had lower rates of proliferation than cells not expressing this receptor. Their research shows Ryk interacts with the signaling molecule Wnt5a to suppress proliferation and promote quiescence, a resting state during which HSCs are more efficient at hematopoiesis. Therefore, increasing Ryk activity could promote blood stem cell regeneration. Defective regulation of HSC proliferation can cause bone marrow failure disorders and hematologic malignancies. These findings were published in the journal "Stem Cells."


Cryopreservation Strategies: Black Magic or Scientifically Sound?

It is not too late to register for the day and a half workshop on preserving cellular therapy products. This highly interactive and collaborative course will cover the unique challenge of cryopreserving cellular therapy products. Topics include global transportation issues and regulations, storage conditions, freezing parameters and repository design. Offered in tandem with the AABB Annual Meeting & CTTXPO on October 10-11, registration includes an evening reception on Thursday and meals on Friday. Attendees also receive an invitation to the cellular therapy networking reception on Friday evening.

CT Schedule at a Glance Available

The complete "Schedule at a Glance" for all cellular therapy events has been posted to the AABB Annual Meeting & CTTXPO Web page.

Click and Go!

The mobile app for the AABB Annual Meeting & CTTXPO was designed to enhance the experience of meeting attendees and provide valuable information in one place for all users of smartphones and similar mobile devices. Not only is this free, easy-to-use interactive portable tool useful during the actual meeting dates, it also allows one to stay connected before and after the meeting. The app will be accessible throughout the year, providing users with an industry reference tool in the palm of their hands.

Science on the Go: Additional Podcasts

A new podcast has been added to the AABB Center for Cellular Therapies audio podcast series. The second episode in the Standards Source Podcast Series for Cellular Therapies will provide an in-depth look into the new clinical activities that are now a part of the Standards for Cellular Therapy Services, 6th Edition. In the Cellular Therapies Educational Podcast Series, experts tackle several challenging questions associated with gray areas in patient and donor care in cellular therapy in an upcoming episode "Ethical Considerations in Cellular Therapy Standards and Regulations."

Starting From Scratch and Other Dilemmas Addressed

"Establishing a Cellular Therapy Program" — developed in cooperation with the International Society of Blood Transfusion, or ISBT — will be the focus of an upcoming webinar. The AABB Center for Cellular Therapies offers a variety of topics in its webinar series where experts share their knowledge and experience, answer questions from participants, and provide valuable resources. The 60-minute interactive webinars consist of 45-50 minutes of presentation, followed by a 10-15 minute period for questions and answers. CME credits are available for the live sessions. Upcoming webinars are:

9/24 Cellular Therapy Best Practices in Document and Record Management: Be Inspection Ready (#1312)

11/19 Establishing a Cellular Therapy Program (#1314)

12/3 Perspectives to Consider for Global CT Compliance (#1315)

Education on Demand

Past recordings of audioconferences on topics in cellular therapies are available through the Live Learning Center including Changes to the 6th Edition of Standards for Cellular Therapy Services, Understanding Cell Therapy Product In-Process Stability From Collection to Infusion, and Protocols for Supporting HPC Transplant Patients Prior to, During, and After HPC Transplants.


Did You Hear the Latest?

Members of the AABB Center for Cellular Therapies subsections participate in an assortment of interactive activities such as journal clubs and presentations on timely topics and challenges, and they work as teams to develop tools and reference materials. The materials or "projects" produced by the subsections are located on the AABB Center for Cellular Therapies website. Recent postings include:

Quality Operations Subsection: This subsection developed a reference tool useful in developing a Quality Assurance Manager or Director job description.

Cord Blood Subsection: This subsection developed "Points to Consider for Consumables and Equipment: Electronic Tracking."



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