The Purpose of H. R. 2852 and S. 1717
“Cord Blood Stem Cell Act of
Cord blood now promises to be one of this century’s medical miracles. Reports from many parts of the world demonstrate the rapidly growing utility of unrelated cord blood transplants for both children and adults. What used to be discarded at birth can now be collected, processed, stored and used as a source for therapeutic stem cells for a variety of devastating diseases. Unlike bone marrow, cord blood is a product on the shelf that is rapidly available for patient use. New collection and processing techniques as well as new treatment tactics such as multiple units in adults make clear that cord blood may eclipse bone marrow transplantation for hematopoetic rescue within a decade or less. The Japan Cord Blood Network has grown from 4% of total transplants when it began in 1999 to 43% in four years. Furthermore, because of its rich mix of multipotential stem cells, cord blood has an expanding range of therapeutic potential including hepatic, cardiac, neuronal and other organ system diseases.
Despite the promise of cord blood, availability for patient care has been limited because of the lack of investment resources to build inventories. Every year thousands of patients succumb to blood and immune system malignancies and genetic diseases because a bone marrow match is not found. Most of these patients might have been treatable with cord blood had the nation’s inventory been significantly larger. Moreover, the threat of terrorism and nuclear catastrophe further heightens our need for instant sources of stem cells to treat potential victims.
New York Blood Center, an institution which has been fortunate and has worked diligently to build a substantial inventory, two years ago began promoting legislation that would create short term federal funding to address this national need. Last year, legislation was introduced in the House (HR2852) by Representatives Smith, Burr, Davis, Towns, Doolittle, Toomey, Faleomavaega, Weldon, and Myrick. This was followed by a bill in the Senate (S1717) introduced by Senators Hatch, Brownback, Specter, Dodd, as well as Feinstein, and Jeffords.
These bills commit the Federal Government to establish funding through the Department of Health and Human Services (HHS) to qualified Cord Blood Banks in order to grow the nation’s cord blood inventory by 150,000 units. The essentials of the legislation represent the vision of the congressional sponsors and are as follows:
1. Funding will be sufficient to grow the nation’s supply of high quality, ethnically and geographically balanced, public cord bank blood units such that all persons may have access to a well-matched therapeutic unit if needed.
2. Cord blood banks from
diverse areas of the
3. Selection of banks will be made by an ad hoc panel appointed by Health Resources Service Administration (HRSA) with Food and Drug Administration (FDA) playing a role in assessment and assurance of quality standards.
4. The HHS Secretary will appoint an independent Board of Directors composed of representatives of the Banks and other constituents. This board will oversee compliance with regulatory exigencies, set policy to maximize access to patients and advise the Secretary on matters pertaining to the use of the funds.
5. Banks will provide cord blood units for peer-reviewed research.
6. The public will be educated on the availability of cord blood and its therapeutic value.
7. Funding is primarily aimed at building the inventory and would go directly to banks for their operations. Banks would be expected to be self-sufficient through operations within five years with continuing federal support ceasing at that time. Minor funds would be reserved for activities of the Board of Directors while it is in place.
A cornerstone of this legislative effort is
recognition that cord blood banking, no longer experimental, is now a fully
accountable activity that often has dire consequences when quality standards
are not recognized or met. To
assure product safety and efficacy, cord blood banking requires strict
adherence to cGMP with at least the rigor of FDA regulated blood banking. The bills require banks to obtain “all
applicable Federal and State license, certifications and registrations”,
including registration with the Food and Drug Administration (FDA). With the expansion of cord blood banking
activity and the diversity of experience and techniques used, anecdotal
reports of distribution of cord blood units that have little or no therapeutic
use have become disturbingly more common. When a patient is conditioned and treated
with such a unit the outcome is often fatal.
practice and potential standards have now been reviewed by FDA through their
The proposed legislation has already yielded important results including passage of a $10M Appropriation in FY2004 and $9.4M in the Presidents FY2005 budget. Senator Arlen Specter was instrumental in these appropriations. In the conference committee there was a difference of opinion over the “independent network concept”. Representative Bill Young, Chairman of the House Appropriations Committee, suggested setting aside funds for a study to be performed by the Institute of Medicine (IOM) in order to make recommendations on the optimal model for administering the funds and implementing the network. IOM will examine the merits of the intent of the legislation to have cord blood banks funded directly with administration through an independent Board of Directors versus assigning all responsibility and incorporating the network funding and administration into the National Marrow Donor Program (NMDP).
From our perspective, as initiators of these legislative efforts, the arguments for an independent network versus NMDP incorporation are as follows:
1. The bills introduced describe an enterprise model of independent cord blood banks without corporate connections or overriding operational bureaucracy. It is a lower cost model with no middleman adding costs, complexity or bureaucracy between the cord blood bank and the transplant center. This approach is more likely to drive innovation, efficiency, and improved patient outcomes - much as the nation’s independent blood centers now provide well over half of our blood supply. Cord blood/cell therapy is a rapidly evolving activity. It will progress most effectively by maximizing necessary resources and minimizing bureaucracy and large institutional imperatives.
2. As emphasized above, quality standards review and enforcement should be decentralized and placed under federal law, preferably FDA. Reliance on administration of standards, accountability and control through a large central bureaucracy potentially introduces political bias and inefficiency that is unlikely to benefit patients. There are well known unfortunate examples of how large bureaucratic organizations have been unable to conform to minimum standards for biologics that the nation should not reproduce in cord blood or cell therapy.
3. Modern distributed
informatics begs for a decentralized database, search and distribution system
for cord blood. Most existing banks already
have local databases that must be managed and updated in any case. Centralized search and distribution systems,
while perhaps necessary for bone marrow donors, are neither necessary nor
desirable for cord blood. A distributed
database search algorithm would bypass the need for a duplicative centralized
database. The use of message-oriented middleware would allow searches to be
performed efficiently at the bank level and all bank results collated into a
single report, if needed. Access to both cord blood units and marrow donors
could be accomplished simply through a collaborative website. Such a system capable of serving an entire
network of cord blood banks was recently demonstrated at
4. While the efforts of NMDP and other registries in bone marrow donor coordination are laudable, cord blood is a competing technology and should not be under the control of its competition. It is unwise for the nation to interrupt its evolution and to create a national monopoly for cord blood or cell therapy – particularly through an organization with little track record or previous interest. Cord blood evolves from a very different set of concepts and skill sets. Unlike the bone marrow donor program, donor recruitment and management are minor issues in cord blood. On the other hand, cord blood banks must manufacture with processing, storage and supply management driven by cGMP concepts, much as our nation’s blood center operations. These are not skill sets or core competencies essential or paramount to a bone marrow program.
5. The advantages of cord
blood banking that operates separately from the bone marrow program are well
illustrated in the Japanese system in which the bank network is independent of
the bone marrow organization. This
system has shown remarkable growth and success in saving lives while assuring
the best product – cord blood or marrow for each patient. It has now served over 1,300 patients (
This exciting new
therapeutic has only just begun to realize its potential for saving lives. It is cell therapy in its largest scale and most
important contribution thus far with much more to come. It is important to note that all the
progress made thus far has been accomplished outside of the constraints of a
large national organization.
We arrive at a critical
crossroads where the nation is poised to make the necessary investment to make
this therapeutic marvel available to all who need it. Many more lives will be saved, as bone
marrow transplantation is clearly limited while cord blood emerges as an
exciting and successful alternative. All
applaud the historic efforts of NMDP to match bone marrow donors with
transplant recipients – a difficult task at best. However, there is an important distinction
between the cord blood model proposed by the forward-looking congressional
leaders and sponsors of the legislation, and the model proposed by NMDP. The former allows the field to progress
unfettered from the resource constraints to build supply while also having the
flexibility and autonomy of locally controlled operations. The latter links cord blood banks in
business and programmatic relationships to a large centralized system designed
to perform quite a different function and without significant experience or
previous commitment to cord blood banking.
As pioneers in cord blood banking and a founding member of