2010-04-15

American Society of Hematology (ASH) represents over 16,000 clinicians and scientists committed to the study and treatment of blood and blood-related diseases. Hematologists were instrumental in characterizing the first stem cell identified in the blood system and developing highly successful therapies from these cells that have resulted in a cure for some blood diseases and cancers. ASH supports all avenues of regenerative medicine research following appropriate standards and methodologies as well as public oversight.

Background

The field of regenerative medicine represents a multidisciplinary approach to treat diseases and disorders by enabling the body to repair, replace, restore and regenerate damaged or diseased cells, tissues and organs. The wealth of genomic information that became available from the successfully completed Human Genome Project has fostered the growth of the field of regenerative medicine and targeted cell therapies that can lead to the development of treatments and cures for many of the major medical issues of our time.

Current Challenges

The lack of coordination of research efforts, disjointed funding mechanisms and absence of harmonization of regulatory requirements of regenerative medicine research threaten to significantly delay the application of this research field into new therapies in the United States. In order to capitalize on the past decade's investments in biomedical and genomic research and significantly advance the field of regenerative medicine, there is a great need for:

1. Coordination of research efforts due to the unique nature of the field and its significant potential to develop treatment and cures for major diseases. Nearly 80 serious diseases have been treated by stem cell transplants, and some diseases such as leukemia, lymphoma and sickle cell disease can be cured by this application of regenerative medicine.
2. New research methodologies, including clinical trial design and standardizing methods for characterizing cell-based products, and the use of animal models for both efficacy and toxicity evaluation.
3. Safety standards and Good Manufacturing Process (GMP) that address the development of cell-based therapy products. 
4. Clinical database registries of recipients of cell-based therapies and tissue/DNA banking for future interrogations.

ASH Research and Clinical Trials Recommendations

ASH applauds NIH’s recent efforts in expanding opportunities for translational application of stem cell research in the institutes’ intramural program, especially the establishment of the Center of Excellence in iPS Cell Technology. However, ASH urges the NIH to identify major opportunities and gaps in biomedical research and funding mechanisms for the field of regenerative medicine as a whole, especially in the Institutes’ extramural program. These efforts should be addressed not by a single existing institute, but the NIH as a whole agency, in coordination with regulatory agencies such as FDA, in order to make the biggest impact on the progress of this field. ASH recommends that the NIH Office of Director should take the lead in establishing a regenerative medicine program or center at the NIH to facilitate resource utilization and coordination of NIH activities in this area. The establishment of the Center of Excellence in iPS Cell Technology is an important first step towards achieving this goal.

The NIH should review and improve its current funding mechanisms for regenerative medicine research to ensure that resources adequately meet the needs of basic discovery, translational and clinical applications of this evolving technology. Specifically, ASH recommends:

• Establish a special “Regenerative Medicine Translational Review Panel/Study Section” that will review P01/R01 grants. This study section should include broad expertise in basic and clinical investigations.
• Encourage additional pharmaceutical/biotechnology partnerships and collaborative grants.
• Develop Requests for Applications (RFAs) that focus on studying both the potential efficacy and the safety of these therapies, including patient-specific database registries for effectively tracking outcomes and adverse events of subjects receiving cellular reagents as well as developing a consensus around the utility of animals and animal models to test efficacy and safety of cellular products.

The NIH should coordinate efforts with the FDA and other regulatory agencies to re-examine the current clinical trials methodologies and determine if these designs are useful in the utilization of cell-based therapies. These joined efforts should be directed towards building a consensus for the design of clinical trials across multiple disease disciplines that optimizes the opportunity for data collection and dissemination. The following should be addressed in this effort:

• Assurance of adequate characterization of the cellular product to be used in human trials.
• A defined long-term follow-up plan for all trials utilizing stem cell derivative products.
• A defined plan to bank a portion of all cell products infused into human patients, or at a minimum, DNA from donor samples for future interrogation.
• Solicitation of input from multiple disciplines to maximize opportunities for data collection.

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