FDA licenses first cord blood product

by Alexey Bersenev on November 11, 2011 · 4 comments

in cell product, cord blood

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Yesterday, FDA approved first cord blood product in US. This is significant historical event in cord blood banking and clinical transplantation. HEMACORD (TM) is the first licensed hematopoietic stem/ progenitor cells-cord cell therapy product, manufactured by New York Blood Center Inc.

Let’s look at history of this event:

In an effort to assist manufacturers in applying for licensure for certain cord blood units, FDA issued the 2009 guidance document entitled “Guidance for Industry: Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications.” FDA instituted a two-year phase-in period for HPC-C manufacturers to submit either a license application or an investigational new drug application. That phase-in period ended Oct. 20, 2011, and these manufacturers now must submit such applications.

What I like in this story is a public transparency of the process. FDA has created Cord Blood Licensure Workshop web-site where you can read some documents. Importantly, FDA CBER has recorded Advisory Committee Meeting held on September 22, 2011 and offered it as a webcast*. This is a snapshot of Advisory Committee Meeting, discussing the licensing of cord blood products:


This is more than 6-hour panel discussion with presentations, but it worth watching if you’re involved in “cord blood business”.

The licensure is aimed to increase a quality of cord blood units and therefore improve transplant outcome. License will be given for particular indications. Will FDA restrict the use of cord blood to only licensed products eventually? It will be very interesting to see the impact of this licensing on the industry and transplant centers in near future. Some thoughts and notes you can read in Cord Blood Banking Group in LinkedIn. Please tell us what do you think of potential impact in the comments.
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* A webcast is running on Microsoft SilverLight platform.

{ 4 comments… read them below or add one }

Santiago Luengo Román November 14, 2011 at 4:29 am

This is the only way “to ensure” and increase the quality of cord blood units. In Germany “to manufature” cord blood unit you are obliged to be a pharmaceutical lab and fulfill GMP´s rules. I expect eurpean authorities will follow this initiative. Unfortunatelly the “official statement” have strong economical interest and they will try to pospone or stop this kind of guidance.

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Frances Verter, PhD November 14, 2011 at 4:34 pm

Personally, I would characterize the cord blood with a BLA license as the same wine in a new bottle, with an FDA label. Cord blood has been used in transplants since 1988 and over 20,000 patients worldwide have received cord blood transplants. The FDA licensing adds nothing to this track record.

To the best of my knowledge, every public cord blood bank in the USA is working on a BLA license, and the NYBC is merely the first to complete the FDA process. Technically, a public bank is not required to have a BLA, because cord blood can be transplanted EITHER with a BLA, OR with an Investigational New Drug (IND) status from the FDA. All the old cord blood inventory in public banks that predates the BLA licensing will be used under IND. But now, possession of an FDA BLA has become a quality standard for public banks, like AABB accreditation, and every bank in the US market feels compelled to pass this bar. So as I said, all the public cord blood banks have been preparing their license applications for months now. No one met the Oct 20th FDA deadline, so they were all given extensions.

In many ways the FDA licensing has limitations, and transplant physicians have been complaining bitterly about them. The license is based on data in the FDA docket, and that data is about 2 years old, so that it does not cover current transplant indications. Notable example: the BLA does not cover unrelated cord blood transplants for sickle cell disease. The HHS/HRSA Blood Stem Cell Advisory Council appointed a working group last week to challenge that and get it covered. Another deep flaw in the BLA is that it is issued to an individual bank, based on the units they released for transplanting various diseases. It does not have a provision to cover cord blood transplants of rare diseases (such as metabolic disorders) based on multi-center data. At the FDA hearing about the NYBC BLA on 9/22/2011, Dr. Kurtzberg of Duke University brought 3 children who had been transplanted for metabolic disorders to personally testify about the importance of adding that indication of use.

In many ways, the “drug” model that the FDA has applied to the cord blood stem cell “product” does not really fit the transplant community. Patients seeking cutting edge treatments will have to rely on centers that have IND’s, because the FDA approvals will always be behind the latest trials.

Meanwhile, the necessary upgrades that cord blood banks have to make to their paperwork and procedures in order to get a BLA are costing them tens of thousands of dollars and will be reflected in the cost of cord blood units for transplant. The cost increase makes US transplant units less competitive compared to foreign cord blood banks with the same HLA types.

Is there anything whatsoever positive about the BLA license? In the long run, if it helps to convince third party payers that cord blood transplantation is a mainstream therapy, so that more health insurance plans cover it and more oncology treatment centers provide it, that will be a good outcome. In the short term it has been nothing but pain for public banks.

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William Ganely February 18, 2012 at 10:43 am

Dr. Verter called licensed cord blood “the same wine in a new bottle”, noted that NYBC was “merely” the first bank to get the license, and that no bank met the Oct 20 deadline. NYBC recevied the license on November 10th, a short few weeks after the deadline. We are still waiting for the next bank to pass the strict FDA requirements, and it seems that most of the other public banks in the US have not even been able to file. Possibly 2 or 3 total banks have even managed to file the application, and one had to withdraw it because it was not even complete enough for the FDA to accept it. The fact that a public entity can in fact achieve license for a stem cell based product is phenomenal, considering the very strict quality control guidelines. The fact is that Dr. Verner doesn’t realize that not all cord blood units are the same quality, and that quality has an impact on patient safety and the efficacy of the treatment. NYBC has the only product that the FDA allows to purport safety, purity, potency, and efficacy… all of the other cord blood units in the U.S. are not of the same quality. It is unfortunate, because as a physician, one would like to have the reasonable assurance that the cord blood stem cells they ordered through a National Registry would be in good shape when they arrived, have more or less the correct number of stem cells, and have the cells be alive (viable) when the unit is thawed. That’s just not always the case, because of the variability in the sources of the cord blood. There are voluntary accreditation agencies and US HHS contracts and other standards, but in the U.S., the FDA is the agency with the most experience in inspecting reviewing biologic products… and there is a difference. That is why there are no other banks that have a license yet. Now, there will be some additional banks soon, and there are a small group of very fine banks in the U.S that provide cord blood. But the level drops off quickly, and I would like to know what Dr. Verter will say in 5 years when there are maybe 3-4 banks with a license and 20 others who cannot pass muster. The final thought I have on the topic is that stem cell transplant has been historically considered the practice of medicine, especially when the source is bone marrow. Cord Blood is now classified as a biologic drug, mostly due to the fact that it is manufactured and stored for possibly years before it is used. It is beneficial to patient care because it is “off the shelf” but if you do not have significant investment in quality systems, you will not be able to guarantee the quality of the product, as NYBC is able to do.

As a side note – Dr. Verter must have missed the information on the indications… there is no limit on the indication for the HEMACORD product.

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Frances Verter February 18, 2012 at 8:07 pm

William Ganely, would you please identify yourself?

I am the founder and director of the Parent’s Guide to Cord Blood Foundation. I have been educating the public since 1998 that not all cord blood units are the same quality. I have also been educating the public that not all discriminators used by banks for marketing are actually meaningful. Your turn to tell about yourself.

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