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AABB CellSource - July 2013 

JULY 2013


ALLOCORD (HPC, Cord Blood) Receives U.S. FDA Approval

SSM Cardinal Glennon Children's Medical Center, St. Louis, Mo., was granted approval by the U.S. Food and Drug Administration to market ALLOCORD, hematopoietic progenitor cells from cord blood, on May 30. Regulatory documents, including the approval letter, are available online. This is the fourth cord blood product to be licensed by the FDA. SSM Cardinal Glennon Children's Hospital is an AABB-accredited cord blood facility.

Japan to Offer Fast-Track Approval Path for Stem Cell Therapies

A retooling of Japan's drug authorization framework, on its way to becoming law, could produce the world's fastest approval process specifically designed for regenerative medicine. The proposed law would create a separate approval channel for regenerative medicine, accelerating translation to the bedside.

Epilepsy That Does Not Respond to Drugs May Be Treated With Key Type of Cell

Medial ganglionic eminence, or MGE, cells — a key type of human brain cell that inhibits signaling in overactive nerve circuits — have been shown to grow seamlessly when transplanted into the brains of mice, raising hope that the cells might one day be used to treat severe forms of human epilepsy. Following one-time transplantation, MGE cells were found to control seizures in epileptic mice. The findings, the first ever to include reports of stopping seizures in mouse models of adult human epilepsy, "are an encouraging step toward using inhibitory neurons for cell transplantation in adults with severe forms of [the condition]," said study leader Scott Baraban, PhD, in a recent article. Cell therapy has become an active focus of epilepsy research because current medications — even when effective — control only symptoms and not underlying causes of the disease, according to the researchers.

Adipose-Derived Stem Cell Product Clinical Trials for Osteoarthritis Set to Begin

RNL BIO has announced that it has filed an investigational new drug, or IND, application with the U.S. FDA to begin clinical trials with its adipose-derived stem cell product, RNL-JointStem, for the treatment of osteoarthritis. Assuming approval of the IND by FDA's Center for Biologics Evaluation and Research, RNL BIO — a stem cell biotechnology company dedicated to the commercialization of autologous cell therapy products for a variety of degenerative and ischemic indications — plans to initiate its double-blinded, randomized, positive-control Phase II clinical trial during the third quarter of 2013 in Sugarland, Texas. Phase I and Phase II clinical trials of the product already have been completed under the authority of the Korean FDA.

'Bioteeth' Could Revolutionize Teeth Replacement

Scientists are one step closer to developing a method to replace missing teeth with bioengineered teeth or "bioteeth" generated from a person's own cells. Researchers at the Dental Institute at King's College London cultured human gum tissue from patients and combined it with tooth-forming cells from mice. By transplanting this combination of cells into mice, the researchers were able to grow hybrid human/mouse teeth containing dentine and enamel, as well as viable roots. The next challenge is to find adult sources of human tooth-forming cells that can be combined with human gum cells. If successful, the new procedure will revolutionize tooth replacement methods.

Three-dimensional Human Heart Muscle Behaves Like Natural Tissue

Duke University biomedical engineers have made a breakthrough in cell-based therapies by growing a three-dimensional human heart muscle that behaves like natural tissue — reaching unprecedented levels of electrical and mechanical maturation. The generated tissue, known as a "heart patch," may become an important treatment option for patients who have had a heart attack or may serve as a platform for testing new heart disease medications. The advancement in growing the patch, in which human embryonic stem cells are prompted to transform into cardiomyocytes with the appropriate cellular signals, overcomes two major obstacles facing cell-based therapies for cardiac disease: The patch conducts electricity at about the same speed as natural heart cells, and it "squeezes" appropriately. The results of the research, supported by the National Heart, Lung and Blood Institute, were published online in the journal "Biomaterials."

ThermoGenesis Announces Registration Approval of AXP in China

ThermoGenesis Corp., a supplier of enabling technologies for the processing and storage of stem cells and other biological tissues, announced that it has received registration approval for its AXP(R) AutoXpress(R) (AXP) product from China's FDA. The product is used to concentrate stem cells from cord blood, and with this approval, the company will immediately initiate commercial distribution of the product in China, one of the world's fastest growing cord blood markets.

ISBT 128 Discussed in an Editorial in the World Health Organization Bulletin

The May edition of the "Bulletin of the World Health Organization" includes an editorial by Warwick et al. on globally consistent coding systems for medical products of human origin, or MPHOs, emphasizing the importance of the Information Standard for Blood and Transplant, or ISBT 128. The International Council for Commonality in Blood Bank Automation, or ICCBBA, was established in 1995 as a not-for-profit organization to manage ISBT 128. The labeling standard was extended to cell therapy and tissues in 2000 and now includes products used for regenerative therapies. Today, ISBT 128 is used by more than 4500 facilities in more than 60 countries. It is endorsed and strongly supported by many international professional and accrediting bodies, including AABB, and is expanding in scope to harmonize the nomenclature and coding of all MPHOs to facilitate global traceability.

U.S. FDA Approves LifeSouth Community Blood Centers' BLA for HPC, Cord Blood

The U.S. FDA's Center for Biologics Evaluation and Research announced its approval of LifeSouth Community Blood Centers Inc.'s biologics license application for HPC, Cord Blood. The approval, dated June 13, allows the company to manufacture the product at its Gainesville, Fla.-based facility. This is the fifth cord blood product licensed by the FDA.

Science on the Go

The AABB Center for Cellular Therapies has added audio podcasts to its education repertoire. Users can download the podcasts using a variety of electronic devices. The exciting new offerings include the Standards Source Podcast Series for Cellular Therapies, a member benefit based on the Standards for Cellular Therapy Services, 6th Edition. In addition AABB is creating a Cellular Therapies Educational Podcast Series, which will feature information openly available on a variety of interesting topics in cellular therapy and regenerative medicine. Listeners will be able to register for the series at no charge and tune in regularly for additions to the schedule.

Standards for Cellular Therapy Services, 6th Edition Now Available

AABB's Standards for Cellular Therapy Services, 6th Edition, is now available in the marketplace. The sixth edition includes expanded content that addresses clinical activities including, but not limited to, requirements for patient consent, patient care, product preparation for administration, and clinical outcomes reporting. The CT Standards went into effect July 1, 2013.


Stem Cell Therapies for Multiple Sclerosis — Strong Hints of Benefit but Hard Data Needed

Marie Csete MD, PhD

Multiple sclerosis, or MS, is a chronic central nervous system, or CNS, disease — the most common non-traumatic debilitating neurologic injury in young adults. Both genetic and environmental risks for MS have been identified. About 20 percent of the genetic risk is attributable to the HLA haplotype, and genome-wide association studies have identified other genetic variants that contribute to risk as well. Environmental risk factors are smoking, Epstein-Barr virus infection, and poor vitamin D nutrition, though clinical trials of high-dose vitamin D have yet to identify a regimen that significantly alters the disease course.

The major pathophysiologic agents of MS are autoreactive T cells, with damage to the blood-brain barrier facilitating CNS inflammation, leading to demyelination and axon damage. Clinically, the disease is very heterogeneous and differs in presentation, symptoms, and course among ethnic groups, making design of clinical trials especially challenging. Read more


U.S. FDA Releases Draft Guidance on Charging for Investigational Products Under an Investigational New Drug Application

The Food and Drug Administration announced a draft guidance for industry titled "Charging for Investigational Drugs Under an Investigational New Drug Application — Questions and Answers" in a "Federal Register" notice dated May 9. The guidance describes how FDA implements its regulation on charging for an investigational drug under an IND application. The document addresses the topics for which the FDA receives the most requests for additional information, including charging for investigational drugs made available under expanded access programs. In a second "Federal Register" notice released May 9, FDA posted a related draft guidance for industry titled "Expanded Access to Investigational Drugs for Treatment Use — Question and Answers." This document provides answers to the most frequently asked questions about FDA's implementation of its regulations on expanded access to investigational drugs for treatment use.

International Competent Authorities Web Page Updated; EU Summary Now Available

The AABB Web site has been updated to include cellular therapy-related regulations in the European Union. Brief summaries of and links to applicable directives and regulations have been provided.

AABB Seeks Member Input on IND and BLA Draft Guidances for HPC, Cord Blood

The U.S. FDA's Center for Biologics Evaluation and Research, or CBER, recently has announced the availability of two draft guidances. "Biologics License Applications for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic and Immunologic Reconstitution in Patients With Disorders Affecting the Hematopoietic System," announced June 17, 2013, provides recommendations to assist establishments in obtaining a license for HPC, Cord Blood. The draft document expands the indications for use of HPC, Cord Blood — which were previously limited to hematopoietic reconstitution for specific diseases — listed in an earlier guidance titled "Minimally Manipulated, Unrelated, Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications," dated October 2009. The 2013 draft guidance includes hematopoietic and immunologic reconstitution — in conjunction with an appropriate preparative regimen for HPC, Cord Blood transplantation — in patients with disorders affecting the hematopoietic system that are inherited, acquired or result from myeloablative treatment. FDA's "reexamination of the legacy docket data" and "consideration of the proceedings of the 2011 meeting of the Cellular, Tissue and Gene Therapies Advisory Committee" led to the revisions of the list of included indications. Additionally, the draft guidance clarifies the types of clinical information that should be submitted with a BLA and includes applicable references to guidance documents on process and methods validation. The term HPC-C also is replaced with HPC, Cord Blood, which is the proper name and International Standard for Blood and Transplant 128-compliant term.

CBER also announced a draft guidance for industry and FDA staff titled "Investigational New Drug Applications for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic and Immunologic Reconstitution in Patients With Disorders Affecting the Hematopoietic System." It is intended to assist establishments in submitting an investigational new drug application, or IND, for HPC, Cord Blood. It pertains to instances when HPC, Cord Blood units are unlicensed (i.e., they do not meet the chemistry, manufacturing and control requirements of the BLA guidance) and no satisfactory alternative treatment is available for the suitable leukocyte antigen-matched cord blood transplant necessary for hematopoietic and immunologic reconstitution in patients with hematopoietic system disorders — inherited, acquired or resulting from myeloablative treatment. Revisions incorporated into the draft BLA guidance also are made in the IND guidance — the term HPC, Cord Blood now is used and the list of indications for use is expanded. Additionally, this draft guidance requires that all IND applications, including single patient INDs, have a table of contents. When finalized, the document will supersede a guidance for industry and FDA staff titled "Investigational New Drug Applications (IND) for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications" dated June 2011.

When finalized, these guidance documents will supersede their similarly titled counterparts published in 2009 and 2011, respectively. AABB will submit comments on the draft guidance documents by the Sept. 16 deadline. Association members may contact AABB's Regulatory Affairs department with concerns, including any regulatory matters not addressed in the draft guidance.


Umbilical Cord Blood Transplantation: The First 25 Years and Beyond

This year marks the 25th anniversary of the first umbilical cord blood transplantation or UCBT. During this period, the field of UCB banking and transplantation has expanded substantially with more than 600,000 UCB units stored for transplantation worldwide and more than 30,000 UCBTs performed. In a review appearing in "Blood," Ballen et al. discuss the growth of the field over the last 25 years including the scientific basis of UCBT, details of the historic first UCBT, recent pediatric and adult UCBT outcome data, as well as strategies for future improvement and exploration.

Sterility Testing of Minimally Manipulated Cord Blood Products: Validation of Growth-Based Automated Culture Systems

An article appearing in "Transfusion" presents the validation approach followed by the St. Louis Cord Blood Bank to fulfill U.S. FDA requirements pertinent to sterility testing methods. The authors describe validation components and results to assist cord blood manufacturers in planning a site-specific method validation as well as provide their experience with the performance of the BACTEC-FX system and BACTEC culture media.

Methods of Freezing Cord Blood Hematopoietic Stem Cells

HPC, Cord Blood (CB) is a valuable source of hematopoietic stem cells or HSCs. Extended storage of CB units may be possible provided that validated cryopreservation procedures are used. In a study appearing in "Transfusion" Antoniewicz-Papis et al. compared two methods of cord blood freezing — two-step cryopreservation and controlled-rate freezing — and evaluated quality before freezing and immediately after thawing. The authors stated that neither the cryopreservation procedure nor the freezing of isolated HSCs affect product quality, which may indicate that various freezing methods can be used for cell banking provided they follow recommendations of good manufacturing practice and EU Directive 2004/33/EC.

The Potential of Stem Cells for the Restoration of Auditory Function in Humans

Hearing loss is one of the most common disabilities affecting approximately 10 percent of the population. Hair cells and spiral ganglion neurons usually are damaged in most cases of hearing loss. Currently, there virtually is no biological approach for replacing damaged hearing cells. For the current "Regenerative Medicine" journal, Hu and Ulfendahl reviewed developments in stem cell technology that provide strategies for the treatment of deafness: differentiation of endogenous stem cells into new hair cells and introduction of exogenous cells into the inner ear to substitute injured hearing neurons. Personalized stem cells may eventually provide a novel intervention for patients with deafness in future clinical research trials.


2013 AABB Annual Meeting & CTTXPO Registration Open

Registration and housing for the AABB Annual Meeting & CTTXPO, to be held October 12-15 in Denver, Colo., is open. Online registration will remain open until October 15. AABB members save on registration. Non-AABB members can save by becoming a member.

Approximately 6,500 individuals will attend 120 educational sessions, review approximately 700 abstracts and visit nearly 200 companies in the exhibit hall. As the premier networking and educational event, attendees will learn the latest in cellular therapies, explore state-of-the-art products and services, and network with colleagues. Please visit the AABB Annual Meeting & CTTXPO website for more details.

AABB, BioCoR Offer Preconference Workshop

The unique challenge of cryopreserving cellular therapy products is the focus of "The Preservation of Cellular Therapy Products: An Interactive Collaborative Workshop" to be held October 10-11, 2013, prior to AABB's Annual Meeting & CTTXPO in Denver, Colo. The interactive workshop is a joint effort by the AABB Center for Cellular Therapies and BioCoR, the Biopreservation Core Resource at the University of Minnesota. Workshop topics include global transport issues and regulations, freezing parameters and repository design. Registration includes a Thursday evening reception and breakfast, lunch and the cellular therapy networking reception on Friday. Continuing education credits are available. Click here to learn more about this workshop.

2013 Cellular Therapies Webinar Series: July Offerings

The AABB Center for Cellular Therapies offers a variety of topics in its new webinar series where experts share their knowledge and experience, answer questions from participants, and provide user tools. 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 include the July 16 session, "MSCs as an Alternative Treatment for Specific Clinical Conditions," developed in cooperation with the Brazilian Association of Hematology, Hemotherapy, and Cellular Therapy or ABHH.

AABB Assessment Tools for Standards for Cellular Therapy Services, 6th Edition, Available

Revised cellular therapy services assessment tools for facilities and assessors — that are associated with the sixth edition of Standards for Cellular Therapy Services, or CT Standards — are now available on the AABB website. Both resources include information specific to the patient-centered clinical elements that are new to this edition's standards, which became effective on July 1. The facilities assessment tool offers assistance to establishments preparing for AABB assessments based upon the sixth edition of the CT Standards. The tool for assessors provides questions applicable to evaluating a facility's compliance with the standards. For further information, email accreditation@aabb.org or call +1.301.215.6492.

Assessors Sought for New AABB Cellular Therapy Clinical Program

Active individual members with experience in hematopoietic stem cell transplant are invited to apply for the new AABB CT Clinical Program. Eligible candidates should be trained as medical doctors, physician's assistants, or nurse practitioners, or hold a Bachelor of Science degree in Nursing. Candidates must have spent a minimum of three years working in an established cellular therapy clinical program and have familiarity with research protocols. Working knowledge of investigational products is preferable. All cellular therapy clinical assessors will receive professional training before participating in facility assessments evaluating compliance with CT Standards. More information for those interested in becoming assessors is available here.

Auditing in the Cellular Therapy Laboratory: New Digital Download

The newly released publication "Auditing in the Cellular Therapy Laboratory" outlines how to design, perform, and follow-up on audits — a quality tool used to verify that systems function as intended and requirements are met. Included are more than two dozen forms, checklists, and templates specific to activities in the cellular therapy laboratory. The item is available for download through the AABB Marketplace.

Proposal Invitation for AABB Press Books

Professionals interested in contributing scholarly works relevant to the cellular therapy field are invited to submit book proposal ideas to AABB. Interested authors or editors are encouraged to contact AABB staff for assistance in preparing a proposal for review by the AABB Press Editorial Board. For more information contact Jennifer Boyer at jboyer@aabb.org.

AHCTA Releases New Resource for Developing Cellular Therapy Programs to Aid Compliance With AABB Standards

Essential Elements — Cells and Tissues for Administration is a resource developed by The Alliance for Harmonisation of Cellular Therapy Accreditation that provides clear examples of compliance with standards for cellular therapies and details to support basic quality system elements. Essential Elements does not describe all of the requirements of AABB Standards for Cellular Therapy Services, but is intended to be a resource for new or developing cellular therapy programs. AHCTA is composed of professional organizations — including AABB — and aims to create a global set of quality, safety and professional requirements for cellular therapy including hematopoietic stem cell transplantation.


Directory Highlights Cellular Therapy Training Opportunities

The AABB Center for Cellular Therapies has developed a directory of training opportunities. The educational programs listed focus on cellular therapy laboratory practice, quality and clinical activities, and patient care. Structured classroom courses are not included. Institutions with relevant training programs may complete a submission form in order to be considered for inclusion in the directory.

New Subsection Materials on AABB Center for Cellular Therapies Website

Members of the AABB Center for Cellular Therapies subsections participate in an assortment of educational activities such as interactive presentations on topical issues, journal clubs and the development of tools and materials. The materials or "projects" produced by the subsections are located on the AABB Center for Cellular Therapies website. Recent postings include:

CT Regulatory Affairs Subsection: Several speakers presented information on ISBT 128. Visit the website to view their presentations.



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