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.
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
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:
- 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
- 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
- Safety standards and Good Manufacturing Process (GMP)
that address the development of cell-based therapy products.
- Clinical database registries of recipients of
cell-based therapies and tissue/DNA banking for future interrogations.
ASH Research and Clinical Trials
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.
of input from multiple disciplines to maximize opportunities for data
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