Newsletter - August 2013
The use of umbilical cord blood cells to treat neurodegenerative diseases offers hope for patients
A newly patented procedure paves the way
Millions of people worldwide suffer from neurological diseases of the brain and spinal cord, such as Alzheimer's disease, stroke, Lou Gehrig's disease (ALS), traumatic brain injury, and spinal cord injury; all devastating and deadly diseases that lack adequate treatment options.
For patients suffering from these diseases and conditions, there is hope on the horizon, however. That hope comes from blood collected from the umbilical cord of newly-born infants. In the past, umbilical cord blood was disposed of as medical waste. But now, we know that umbilical cord blood cells can possibly be used for the treatment of neurological conditions for which there are few therapeutic alternatives.
Human umbilical cord blood provides a rich source of multipotent stem cells that have the ability to become different types of cells, including neural cells. Currently, we are developing therapies for many different diseases, including those mentioned above, from cord blood-derived neural cells. In addition, cells obtained from umbilical cord blood could also potentially help repair damage from heart attacks and treat other cardiovascular diseases.
Finding ways to obtain and use umbilical cord blood cells to develop cell transplantation therapies for neurological disorders and diseases is a high priority here at Saneron.
Saneron is a biotechnology research and development company spun out from the University of South Florida (USF) and affiliated with Cryo-Cell International since 2001. In collaboration with USF researchers, we have recently received a patent for a new method of isolating stem cells from donated umbilical cord blood and preparing them for therapeutic transplantation.
These steps include: retrieving cord blood cells, separating the different types of cells by using a magnetic separator, and then "expanding" the cells, or incubating them in a "growth factor". Then, we use a "differentiation agent" to help the cells change into neural cells suitable for transplantation.
The umbilical cord blood cells can be provided by self-donors (autologous) or other-donors (allogeneic). Also, the umbilical cord blood sample may be fresh or frozen (cryopreserved). The cord blood produces a sufficient amount of stem cells so that a single sample can provide enough stem cells to treat a patient.
Saneron's umbilical cord blood research initially started with developing therapies for stroke, before expanding to Alzheimer's disease, Lou Gehrig's disease (ALS), spinal cord injury, traumatic brain injury, Sanfillipo syndrome, and myocardial infarction.
We have high hopes for our newly patented techniques for using umbilical cord blood cells therapeutically. These cells are great candidates for restorative stroke therapy because they are less mature than bone marrow cells. Their immaturity allows them to differentiate more readily into different kinds of cells. They are also easier to isolate and obtain than many other kinds of cells.
Our latest focus is partnering with USF's Center of Excellence for Aging and Brain Repair, to investigate the use of umbilical cord blood cells in treating patients with diabetes who have suffered strokes.
Because Every Kid Deserves to Feel Special:
A Profile of Hope for Henry Foundation
10,445 smiles and counting
Since 2003, Washington DC-based Hope for Henry has made life better for more than 10,000 kids bravely fighting cancer and life-threatening blood diseases. While undergoing bone marrow transplants, chemotherapy, and other life-saving procedures, 368 kids have had incredible in-hospital birthday parties; 1,036 kids were entertained and uplifted by gifts of brand new iPads or portable DVD players and gaming systems; 1,675 princesses, superheroes, witches and goblins trick-or-treated in the hospital wearing brand new costumes; 286 girls and moms looked and felt beautiful during their fashion photography session after having their makeup and nails done; and 2,041 kids have met Batman. This is just a small sample of Hope for Henry's entertaining program that produces smile after smile while distracting kids from painful medical procedures and the tedium of long hospital stays that are a necessary part of their treatment.
My husband Allen Goldberg and I founded Hope for Henry in 2003 on what would have been our son Henry's 8th birthday. During his too-short but magnificent life, Henry taught all of us to live well and laugh hard, even while he fought Fanconi anemia, a deadly blood disease.
Hope for Henry is the only organization in Washington DC whose singular focus is on improving the day-to-day experience of children with cancer and life-threatening blood disease year-round at Children's National Medical Center and MedStar Georgetown University Hospital. Every day, hospitalized kids are smiling and laughing because of Hope for Henry.
While the doctors and medical teams focus on curing physical illnesses, Hope for Henry is an Rx for their spirit, bringing joy, fun, magic and adventure into their lives despite their serious medical needs. Sadly, not everyone benefits from the admirable efforts to find cures – but every one of these courageous kids immediately benefits from Hope for Henry's ability to make them feel special and loved every day. In addition to the benefits of increased levels of hope and happiness, medical experts routinely observe that Hope for Henry's program contributes positively to patient recovery - through increased willingness to accept necessary medical care, decreased feelings of isolation, and reduced expressions of fear surrounding medical procedures.
Henry's legacy goes well beyond the work of the Foundation established in his name. In an effort to save his life, our family was the first in the world to use preimplantation genetic diagnosis (PGD) to try to get pregnant with a baby free of genetic disease and a perfect HLA match to Henry – the ideal stem cell donor - and hopefully his savior. We endured nine failed courses of the procedure between 1996-2000 before running out of time and resorting to a generous anonymous donor identified through the National Marrow Donor Program. Henry's story was featured in a New York Times Magazine cover story and twice on ABC News' Nightline. My book, Saving Henry (Hyperion, 2010) is the story of our family's search for a cure, and the long-lasting scientific impact of Henry's life.
Plastic Surgery with Adipose Stem Cells
Your own fat tissue is an abundant source of stem cells. Ever since Mesenchymal Stem Cells (MSCs) were first extracted from adipose tissue in the late 1990's, there has been an exponential growth in research and clinical trials using stem cells derived from adipose (fat) tissue.
The reason for this sudden interest is due to the inherent qualities of adipose tissue. First of all, adipose tissue is readily available and easily harvested using well established liposuction techniques. Secondly, there is a lot of adipose tissue available for harvest. Finally, the concentration of stem cells and other progenitor cells per gram of adipose tissue is more than 100 fold greater than the number of cells recovered from an equal amount of bone marrow.
The fact that adipose tissue is a prolific source of MSC's has important practical implications for therapy. Therapeutic doses of stem cells can be obtained without the need for culture expanding the cells in a lab. This shortens the cell processing time from several weeks (culture expanded) to one day (freshly isolated).
For example, in the cell processing station in my clinic we get mononuclear cell yields of 300,000 to 1.4 million cells per cc of tissue, depending on the patient's age and isolation techniques. If we harvest 200cc's (slightly less than a cup) of adipose tissue, the total cell yield can range from 60-280 million cells. These are therapeutic doses of cells that can then be re-administered to the patient in the same operating session. For this reason, adipose tissue is attracting the attention of many academic institutions, researchers, and clinicians.
After adipose tissue is extracted, the freshly isolated cells obtained with enzymatic digestion are referred to as the Stromal Vascular Fraction, or SVF. As implied by the name, the cells come from the stroma, or connective tissue matrix and blood vessels of the adipose tissue. The SVF is a mix of various cell types. Approximately 10% of the SVF are Mesenchymal Stem Cells (MSCs). The remaining cells are a mixture of precursor cells (pre-adipocytes, endothelial progenitor cells), immune cells (macrophages, lymphocytes), and a small amount of other cells including red blood cells. The beauty of this mixture is that the combination of cell types together represent a 'stem cell niche' where cells interact with differing degrees of 'stemness'.
The Stromal Vascular Fraction can be used immediately, stored for future use, or culture expanded to grow a repeat dose for use during a convalescent period. The US Armed Forces is exploring the potential of adipose tissue in wartime injuries and has forged alliances with several academic centers that have well established programs in adipose tissue research.
Another important milestone in the field of adipose stem cells is the development of relatively inexpensive modular medical devices that incorporate all the steps of extracting stem cells from a patient's adipose tissue in a closed environment that satisfies the FDA requirements of cGMP (cellular Good Manufacturing Practices). This means that any surgeon can have the capacity to extract, process, and reinject a patient's own stem cells for therapy in the same day.
Although the technology is available for cost effective deployment, regulatory hurdles remain to bring these devices to the marketplace. Even in the case of your own cells, called 'autologous cells', the FDA has not determined whether the SVF is considered a 'biologic drug'. Thus, any clinician contemplating use of autologous SVF must go through the process of obtaining an Institutional Review Board (IRB) permission for an experimental therapy and any clinical trial must be registered with the FDA. This can represent a significant challenge to many practitioners.
Despite these hurdles, adipose derived stem cell therapy is already being deployed in clinical trials around the world. In June 2012, Leeza Rodriguez began tracking the Adipose Stem Cell Clinical Trials that are registered at ClinicalTrials.gov database and posting the analysis on a page of my website. At that time, there were 54 registered clinical trials, and the list of disease indications blankets the entirety of the human body, including fistulas, breast reconstruction, cardiovascular disease, pulmonary, diabetes, auto immune, neurological, and others. Since June 2012, other indications such as traumatic brain injury and osteoarthritis have been added.
I am currently participating in a research study that uses autologous SVF to treat radiation damage in women following breast cancer treatments. Not only will we be treating radiation damage, but we will also be tracking the adipose stem cells to see where they migrate and how long they stay at the site of injury. This research study is funded by a grant from the Maryland Stem Cell Research Fund. Our team includes researchers at JHU, University of Maryland, Celsense, and CosmeticSurg.
Adipose stem cells will play an important role in the future of regenerative medicine. To learn more about adipose stem cells or to ask me questions, please visit my interactive blog. You can also follow Dr. Rodriguez on Google+.