Newsletter - May 2013

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World Cord Blood Inventory 2012

World map with notes on regional cord blood inventory, both public and private

Frances Verter, PhD, Parent's Guide to Cord Blood Foundation

According to the World Marrow Donor Association (WMDA) 2012 annual report, as of 1 Jan. 2012 the world inventory of cord blood in public banks was 591 thousand. By comparison, the Parent's Guide to Cord Blood Foundation finds that as of 31 Dec. 2012, the world inventory of cord blood in family banks was over 2.47 million.

The Parent's Guide to Cord Blood Foundation inventory is based upon confidential interviews with the management of family cord blood banks in our world database. The Parent's Guide to Cord Blood Foundation also maintains an accompanying database of case histories of patients who received therapy with cord blood stored in family banks, and the total number of cases is now close to one thousand. Information from the bank and patient databases are only released in aggregate formats, to preserve both the confidentiality of the bank business information (unless it is already public knowledge) as well as the privacy of the patient medical histories. The only person who has ever seen the raw contents of either database is Dr. Frances Verter, and the databases are stored on encrypted hard-drives. A report on the bank and patient data was presented as poster #63 at the 2013 annual ISCT meeting.

The map above shows some breakdowns of cord blood storage by geographic region. Our database is accurate for the regions of USA and EU, but incomplete for the regions of South America and Asia. Cord blood banking started in the USA, where the world's largest public bank is the NY Blood Center (current inventory over 60,000) and the world's largest family bank is Cord Blood Registry (current inventory 425,000). In the WHO European region, the WMDA reports a net inventory of 207,385 among public cord blood banks, whereas the family bank trade association Cord Blood Europe reports a net inventory of 500,000 among its member family cord blood banks as of Oct. 2012. Whereas the WMDA finds the net inventory in public cord blood banks in the WHO Western Pacific region to be 151,700, by comparison the largest mixed public/family bank in Korea, Medipost, exceeds that with an inventory of 162,429 as of Feb. 2013. According to our database, Medipost is currently the world's 5th largest cord blood bank of any type, and the largest outside of the US and EU. The only country in the world that has slightly more cord blood in public banks than in family banks is Japan, where both inventories are close to 30 thousand.

Obviously, the slow growth of public cord blood banking for unrelated transplants, which is usually dependent on government and charitable funding, is completely dwarfed by the successful business model of family cord blood banking as a form of health insurance. It is in the best interests of public health, at a global scale, to bring the operating standards of all family banks up to the public bank level and harness more of their storage for therapeutic purposes.

It has often been suggested that public cord blood banks should adopt a "hybrid" business model, in which the same bank offers both public and family banking, as a path to economic viability. Yet most cord blood banks around the world continue to operate predominantly in either the public or family mode of banking. Instead of economic forces driving bank practices, a new trend is that new clinical applications of cord blood are pushing banks towards hybrid behavior.

Consider clinical trials for cerebral palsy and similar acquired neurological conditions as an illustration of shifts in cord blood banking practices: The use of cord blood as therapy for these conditions was pioneered by Dr. Kurtzberg at Duke University Medical Center in 2004, and was initially only available to children who had their own cord blood privately banked. Since then, about 300 clients from family banks have gone to Duke to receive their own cord blood for acquired neurological conditions (Ref: PGCB patient database). But recently over 60 children have entered clinical trials (NCT00593242, NCT01445041) at Duke by relying on the university's public cord blood bank to provide personal storage for regenerative therapy. Meanwhile, in Korea, a couple of recent clinical trials (NCT01193660, NCT01528436) have treated over 50 children for cerebral palsy with matched donated cord blood units. In each of these cases a public bank is providing cord blood not for transplants but for regenerative therapy, in one case autologous and the other allogeneic.

Another example of changing paradigms can be seen in India, a country that does not have a national network of public cord blood storage, but does have a robust private banking sector. In recent years dozens of children have received sibling cord blood transplants for thalassemia, a treatment option that would not have been available to them without family cord blood banks.

The common theme in all of these cases is that cord blood bank practices are moving away from the silo concept that public banks only provide transplants and family banks mainly supply regenerative therapies. We are seeing more public banks facilitating regenerative therapy, and in some parts of the world patients must rely on family banks for transplants.

This article and the accompanying graphic are copyright by Parent's Guide to Cord Blood Foundation.

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Brain Injury Association of America

BIAA logo

Every 18.5 seconds, someone in America suffers a brain injury. Across the US, 1.7 million people sustain a traumatic brain injury (TBI) each year. Another 795,000 individuals sustain an acquired brain injury (ABI) from non-traumatic causes each year. A brain injury can happen anytime, anywhere, to anyone – a brain injury does not discriminate. In the blink of an eye, a brain injury changes the way we think, talk, move and feel.

Brain injury is not an event or an outcome. It is the start of a misdiagnosed, misunderstood, under-funded neurological disease. Individuals who sustain brain injuries must have timely access to expert trauma care, specialized rehabilitation, lifelong disease management and individualized services and supports in order to live healthy, independent and satisfying lives. Currently more than 3.1 million children and adults in the US live with a lifelong disability as a result of TBI, and an estimated 1.1 million have a disability due to stroke.

The Brain Injury Association of America (BIAA), founded in 1980, is the country's oldest and largest nationwide brain injury advocacy organization. The mission of BIAA is to advance brain injury prevention, research, treatment and education, and to improve the quality of life for all individuals impacted by brain injury. BIAA is dedicated to increasing access to quality health care and raising awareness and understanding of brain injury. With a network of state affiliates, local chapters and support groups, BIAA is the voice of brain injury.

The 2013 Bowling for Brain Injury event is an inaugural event for the Brain Injury Association of America. Five pilot states: Illinois, Indiana, Missouri, Nebraska and New York will launch the event between March and June 2013. Teams of six will register. Each member of the team will create an individual webpage that can be used to email friends and family...asking for their support of your efforts. Bowling for Brain Injury is a fun and easy way to support a cause that impacts millions of Americans.

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Developing new treatments for Traumatic Brain Injury

Adrian Harel, PhD, MBA

Adrian Harel, PhD, MBA, Founder and President of MediCortex USA Ltd.

Traumatic brain injury (TBI) is considered to be a major cause of disability and death worldwide, especially in children (as a result of falls and playground injuries), soldiers (from blasts and accidents), and the elderly (falls and stroke). The incidence of TBI is 235 per 100,000, with a worldwide mortality of about 1.5 million per year, and in the USA more than 5 million people are coping with disabilities from TBI at a cost of $60 billion a year.

Symptoms of TBI are dependent on the type of TBI (diffuse or focal), the part of the brain that is affected, and the injury's severity. Briefly, the symptoms can include loss of consciousness, headache, vomiting, nausea, dizziness, balancing difficulties, blurred vision, ringing in the ears, bad taste in the mouth, fatigue or lethargy, and changes in sleep patterns. Cognitive and emotional symptoms include behavioral or mood changes, confusion, and trouble with memory, concentration, attention, or thinking.

In addition to the damage caused at the moment of and in the days following the injury, brain trauma causes delayed secondary events, including: initiation of an inflammatory cascade, blood-brain barrier disruption, degradation of lipids in the brain by oxidation, and degeneration of neurons. These series of events lead to the development of various neurological deficits.

The best treatment for TBI is prevention. Once brain injury does happen, the treatment depends on the recovery stage of the patient. In the acute stage, the primary aim is to stabilize the patient and focus on preventing further injury. Rehabilitation is then the main treatment for the sub-acute and chronic stages of recovery.

Currently, there is no actual designated treatment for TBI, or for preventing the progression of secondary degeneration from TBI. Despite promising pre-clinical studies, over the past decade, 30 phase 3 clinical trials have failed to show significant results at reducing secondary injury following TBI. Halting the secondary degeneration requires an intervention that simultaneously targets multiple factors which contribute to the progress of the neuro-degradation in the brain.

In recent years, stem cells have been embraced as a therapy for TBI. Stem cells can halt brain damage and promote healing by migrating to the site of injury where they reduce inflammation and have a tissue-protective effect. Pharmacological pre-clinical studies have demonstrated that treatment with autologous stem cells administered immediately after trauma may aid in reducing the immediate cognitive defects of TBI (1). Clinical studies in patients with TBI have demonstrated that neurologic function was improved at 6 months after cell therapy (2). Currently, there is an ongoing phase 1 clinical trial where children with TBI receive stem cells from cord blood.

schematic diagram of drug molecule components

At MediCortex USA Ltd., we are developing an alternative treatment that simultaneously targets multiple processes in TBI. The company has designed a family of chemically verified small molecules, called New Chemical Entities (NCEs) that aim to decrease levels of circulating toxic metal ions in the brain, as well as minimize oxidation by free radicals. The NCE’s, each with the ability to cross the brain-blood-barrier, have at least two more active sites that exert neuro-protective functions, such as free metal ion binding, anti-oxidation, anti-inflammation, and/or anti-bacterial (see the diagram). At present the molecules are being tested in pharmacological pre-clinical studies.

If successful, small molecules have many advantages over stem cells as emergency intervention for TBI patients. Only TBI patients whose parents stored their cord blood and cord tissue would have their own stem cells readily available for treatment. Otherwise, TBI patients pursuing stem cell therapy must undergo an invasive stem cell harvest or obtain donor stem cells. If an enhanced MSC population is desired, it takes about 2-3 weeks in the laboratory to culture the cells. It is possible that an off-the-shelf stem cell product could be developed based on donor MSC, but it would carry high production costs. By comparison, a TBI therapy that relies on small molecules, such as the NCEs, can be on the shelf for immediate treatment with no dose limitations, at a much lower cost of production, and with easy administration.

Since TBI is a complex multi-system condition, treatments are needed that target simultaneously various biochemical pathways that occur at different time points post the initial brain injury. A combination therapy is most likely to be successful at attenuating and even preventing secondary TBI-associated neuronal death and neurological dysfunction (3).

Adrian Harel has a PhD in neurobiology from the Weizmann Institute of Science and an MBA from the University of Haifa. He has been the CEO or manager of several companies that specialize in early-stage drug discovery for the treatment of acute neurodegenerative disorders. These include BrainStorm Cell Therapeutics (OTC: BCLI), Meditor Pharmaceuticals and Aminolab Technologies, Sepal Pharma, Molecular Cytomics, Heal-Or, and Proneuron Biotechnologies. From 2000 to 2002 he was the CEO of the Jerusalem Biotechnology Center. Dr. Harel is currently the CEO of MediCortex (www.medicortex.com), a privately held company that he founded in 2010.

References

  1. Nichols JE, Niles JA, Dewitt D, Prough D, Parsley M, Vega S, Cantu A, Lee E, Cortiella J. Neurogenic and neuro-protective potential of a novel subpopulation of peripheral blood-derived CD133+ ABCG2+CXCR4+ mesenchymal stem cells: development of autologous cell-based therapeutics for traumatic brain injury. Stem Cell Res Ther. 2013 4(1):3.
  2. Zhang ZX, Guan LX, Zhang K, Zhang Q, Dai LJ. A combined procedure to deliver autologous mesenchymal stromal cells to patients with traumatic brain injury. Cytotherapy. 2008;10(2):134-139.
  3. Harel A., New approach to brain trauma by chemical combination, 2nd international conference: Neurology & Therapeutics, 2013, Chicago, USA poster