FDA BLA

This topic describes FDA licensing, which is only required of public cord blood banks, ie. those that collect donations for use by patients outside the donor's family. The BLA requirement became effective in Oct. 2011, but 7 years later not all public banks in the US had completed the difficult and expensive licensure process. Those public banks which are not yet licensed are being allowed to continue operations if they are "moving towards" licensure.

We have a very good news article reviewing this topic by Mahendra Rao, MD PhD, dated January 2015.

Many people who see news reports about the FDA licensure of cord blood banks are confused, and for good reason. Cord blood has been used in stem cell transplants since 1988, and by 2017 there had been over 35,000 cord blood transplants worldwide. From the perspective of hematologists and oncologists, cord blood transplants have become a standard therapy for patients. Yet, despite this long track record, the FDA had never officially approved cord blood transplants as a standard therapy or established a licensing standard for public cord blood banks. As a result, hospitals that provided cord blood transplants had to operate under clinical trial protocols, and some patient's insurance providers would not pay for cord blood transplants because they were labeled "experimental". Hopefully, the establishment of the FDA BLA license for cord blood will encourage more health insurance plans cover it and more oncology treatment centers provide it, so that patients will have more access to this therapy.

The FDA licensing requirements are not intuitive: The FDA operates on the philosophy that human cells are a "biologic product", or in other words a drug, whenever they are given to a person other than the donor. The Biologics License Application "is a submission that contains specific information on the manufacturing processes, chemistry, pharmacology, clinical pharmacology and the medical affects of the biologic product. If the information provided meets FDA requirements, the application is approved and a license is issued allowing the firm to market the product." (from the FDA glossary)

Hence, in plain language, the FDA BLA license requires that the public cord blood banks must operate their laboratories as if they are manufacturing drugs. Some of the requirements are sensible, like detailed requirements for laboratory sterility and for monitoring of batches on the production line (except here every single cord blood collection is a separate batch). But in some ways the drug analogy is not really applicable to cord blood, and that has caused a great deal of grief for cord blood bankers. For example, the FDA requires that each cord blood unit must be labeled with an expiration date. But cells that are cryogenically frozen remain viable for decades, and no expiration date has been scientifically established. Moreover, we do not have the technology to put a label on a unit that is already cryogenically frozen.

The FDA BLA license creates distinctions between cord blood that was stored before versus after the bank's BLA approval. The licensure is not retroactive, so when a public cord blood bank gets a license, it only covers their incoming cord blood collections, and their older cord blood inventory is not licensed. Nonetheless, if a patient needs a non-licensed cord blood unit (for instance if it is the best match to the patient), then it can be shipped for treatment if the hospital has an FDA-approved clinical trial (one that has IND) or if the cord blood was requested via the NMDP, which has a global IND for all their banks.

Another important feature of the FDA BLA license is that it only covers approved indications: a list of diagnoses for which the FDA recognizes cord blood transplants as effective therapy. If physicians want to use cord blood from public banks for treatments that are not on the FDA BLA list, they must do so under a clinical trial IND label, regardless of whether or not the cord blood itself was banked under an FDA BLA license.

Finally, no innovation in cell therapy comes without press releases and marketing. Some (but not all) of the cord blood banks that have obtained their BLA license have given a name to their licensed cord blood "product". This leads laypeople to think that something new has become available, when it is really just a new label for the same cord blood transplant units that these labs produced before.

The public cord blood banks that have achieved FDA BLA status:

  1. National Cord Blood Program at NY Blood Center
    (Nov. 2011 product HEMACORD™)
  2. ClinImmune Labs at the University of Colorado
    (May 2012 product HPC, Cord Blood)
  3. Carolinas Cord Blood Bank at Duke University Medical Center
    (Oct. 2012 product DUCORD™)
  4. St. Louis Cord Blood Bank at SSM Cardinal Glennon Children's Medical Center
    (June 2013 product ALLOCORD)
  5. LifeCord Cord Blood Bank at LifeSouth Community Blood Centers
    (June 2013 product HPC, Cord Blood)
  6. BloodworksNW (laboratory for EvercordTM family bank)
    (Jan. 2016 product HPC, Cord Blood)
  7. Cleveland Cord Blood Center
    (Sept. 2016 product CLEVECORD)