Target Health Proudly Supports the Arts in NYC


May 2013  —  Springtime in Central Park, New York City (Manhattan)



We believe that the Arts represent the soul of a city, and of a society. Driven by our founder and CEO Joyce Hays, and as part of corporate civic responsibility and to give back to our great city, Target Health is pleased to announce that it supports the following theatre and cultural groups in NYC (and one in Santa Fe, NM):


1. Manhattan Theater Club
2. Roundabout Theatre Club
3. Signature Theater Company
4. Atlantic Theatre Company
5. Metropolitan Opera
6. MCC Theater Company
7. New York Theater Workshop
8. PILOBOLUS Dance Company
9. SITE Santa Fe


For more information about Target Health contact Warren Pearlson (212-681-2100 ext. 104). For additional information about software tools for paperless clinical trials, please also feel free to contact Dr. Jules T. Mitchel or Ms. Joyce Hays. The Target Health software tools are designed to partner with both CROs and Sponsors. Please visit the Target Health Website at

Regenerative Medicine: Surgery for Girl Born Without Windpipe


Jim Carlson/OSF Saint Francis Medical Center
Hannah Warren with her parents, Darryl and Young-Mi Warren, a few days
before the operation at Children’s Hospital of Illinois.


A Synthetic Windpipe


Credit: Jim Carlson/OSF Saint Francis Medical Center — Hannah, who was born
without a trachea, no longer needs a tube to breathe, and after the surgery she
could put her lips together for the first time.


Credit: Jim Carlson/OSF Saint Francis Medical Center — Dr. Philipp Jungebluth,
left, and Dr. Paolo Macchiarini inspected a neotrachea seeded with stem cells.
The implant is designed to reduce the risk of rejection.



Using plastic fibers and human cells, doctors have built and implanted a windpipe in a 2-year-old girl – the youngest person ever to receive a bioengineered 1) ___. The surgery, which took place on April 9, 2013 at Children’s Hospital of Illinois and has been formally announced, is only the sixth of its kind in the world and the first to be performed in the United States. It was approved by the FDA 2) ___ – and ___ ___ under rules that allow experimental procedures when otherwise the patient has little hope of survival.


Dr. Paolo Macchiarini, a specialist in the field of 3) ___ medicine who developed the windpipe and led the complex nine-hour operation, said the treatment of the Korean-Canadian toddler, Hannah Warren, made him realize that this approach to building organs may work best with children, by harnessing their natural ability to grow and heal. “Hannah’s transplant has completely changed my thinking about regenerative medicine,” said Dr. Macchiarini, a surgeon at the Karolinska Institute in Stockholm. He said he would like to proceed with a 4) ___ clinical trial in the United States.


Hannah was born without a windpipe, or trachea – an extremely rare condition that is eventually fatal in 99% of cases – and had lived since birth in a newborn intensive 5) ___ care unit in a Korean hospital, breathing through a tube inserted in her mouth. Because of other developmental problems, she cannot eat normally and cannot speak. Nearly three weeks after the surgery, the girl is acting playfully with her doctors and nurses, at one point smiling and waving goodbye to a group of visitors. Dr. Mark Holterman, a pediatric surgeon at the hospital, said that Hannah was 6) ___ largely on her own, although through a hole in her neck, not through her mouth yet. Dr. Macchiarini described a look of befuddlement on the child’s face when she realized that the mouth 7) ___ was gone and she could put her lips together for the first time.


The goal of regenerative medicine, or tissue 8) ___, is to create or regrow tissues and organs to ease transplant shortages or treat conditions that do not have an effective cure. After years of scant progress, tissue engineers have begun to make advances as they have gained a better understanding of the role that stem cells – basic cells that can become tissue-specific ones – play in signaling the body to grow and repair itself. Still, only a few relatively simple organs have been made and 9) ___, and the science-fiction-inspired goal of ready-made hearts or other complex organs remains far off. Until now, the youngest recipient of a tissue-engineered organ was a 4-year-old spina bifida patient who received a bladder.


Dr. Macchiarini has performed the five other windpipe implants similar to Hannah’s. One patient, an American man who was operated on in Stockholm, has died. An Eritrean man has lived the longest so far, surviving for about 2.5 years since the 10) ___.


To make Hannah’s windpipe, Dr. Macchiarini’s team made a half-inch diameter tube out of plastic fibers, bathed it in a solution containing stem cells taken from the child’s bone 11) ___ and incubated it in a shoebox-size device called a bioreactor.


Doctors are not sure exactly what happens after implantation, but think that the stem cells signal the body to send other cells to the windpipe, which then sort out so the appropriate tissues grow on the inside and outside of the tube. Because the windpipe uses only the child’s own 12) ___, there is no need for drugs to suppress the patient’s immune system to avoid rejection of the implant. Dr. David Warburton, director of the regenerative medicine program at the Saban Research Institute in Los Angeles, who was not involved in the windpipe work, said that “guarded optimism with a major dash of skepticism is the watchword” for such experimental approaches. “The challenges will be making a wind pipe that functions better than a temporary fix,” he said.


While one-of-a-kind operations like this are ethically justified given the desire to save the life of the patient, some experts say that regenerative medicine would be better served by clinical trials that could, through analysis of more data, provide a better understanding of how effective these kinds of 13) ___ are and how they function. “No doubt large trials are critical,” said Martin Birchall, a professor at University College London who used to work with Dr. Macchiarini but has switched to using donor 14) ___ from cadavers, which he strips of their original cells. Dr. Macchiarini said he was now ready to go ahead with a clinical trial in the United States, and the Peoria hospital said it would like to arrange one if it could gain F.D.A. approval.


The toddler faces a long rehabilitation process as she breathes normally for the first time. Doctors hope that with additional operations she will be able to eat through her mouth and 15) ___. As the girl grows, she will also need a bigger windpipe. Dr. Macchiarini estimated that she might need a new one in four years, but said his team tried to delay a replacement for as long as possible by oversizing the implant and including some biodegradable 16) ___, which may allow it to stretch. The girl’s parents, Darryl and Young-Mi Warren, said that shortly after Hannah was born they had been told that there were some treatments for her condition, but that the odds of her living past age 6 were very slim. “We didn’t want Hannah for just another couple of years,” Mr. Warren said. “We wanted her for the rest of our lives.” Mr. Warren did some Internet research two years ago and discovered Dr. Macchiarini’s work. He shared it with Dr. Holterman, who happened to learn about Hannah while on a business trip to South Korea. Dr. Holterman and the hospital’s chief surgeon, Dr. Richard H. Pearl, persuaded Dr. Macchiarini to come to Peoria to do the procedure. Mr. Warren said he was amazed that it all worked out. “It actually is unbelievable,” he said. “The fact that they’re doing this, at this time, when Hannah needed it the most.” Source: NYTimes, May 1, 2013


ANSWERS: 1) organ; 2) Food and Drug Administration; 3) regenerative; 4) clinical; 5) care; 6) breathing; 7) tube; 8) engineering; 9) implanted; 10) surgery; 11) marrow; 12) cells; 13) implants; 14) tracheas; 15) speak; 16) plastic


Video About Other Regenerative Medical Issues

2013 Mayo Clinic: Center for Regenerative Medicine

Regenerative Medicine


Human Stem Cells


Regenerative medicine is the “process of replacing or regenerating human cells, tissues or organs to restore or establish normal function”. This field holds the promise of regenerating damaged tissues and organs in the body by replacing damaged tissue and/or by stimulating the body’s own repair mechanisms to heal previously irreparable tissues or organs. Regenerative medicine also empowers scientists to grow tissues and organs in the laboratory and safely implant them when the body cannot heal itself. Importantly, regenerative medicine has the potential to solve the problem of the shortage of organs available for donation compared to the number of patients that require life-saving organ transplantation. Depending on the source of cells, it can potentially solve the problem of organ transplant rejection if the organ’s cells are derived from the patient’s own tissue or cells.


Widely attributed to having first been coined by William Haseltine MD (founder of Human Genome Sciences), the term “Regenerative Medicine” was first found in a 1992 article on hospital administration by Leland Kaiser. Kaiser’s paper closes with a series of short paragraphs on future technologies that will impact hospitals. One such paragraph had ‘‘Regenerative Medicine’’ as a bold print title and went on to state, ‘‘A new branch of medicine will develop, that attempts to change the course of chronic disease and in many instances will regenerate tired and failing organ systems.’’


Regenerative Medicine refers to a group of biomedical approaches to clinical therapies that may involve the use of stem cells. Examples include the injection of stem cells or progenitor cells (cell therapies); the induction of regeneration by biologically active molecules administered alone or as a secretion by infused cells (immunomodulation therapy); and transplantation of in vitro grown organs and tissues (Tissue engineering).


A form of regenerative medicine that recently made it into clinical practice, is the use of heparan sulfate analogues on (chronic) wound healing. Heparan sulfate analogues replace degraded heparan sulfate at the wound site. They assist the damaged tissue to heal itself by repositioning growth factors and cytokines back into the damaged extracellular matrix. For example, in abdominal wall reconstruction (like inguinal hernia repair), biologic meshes are being used with some success.


At the Wake Forest Institute for Regenerative Medicine, in North Carolina, Dr. Anthony Atala and his colleagues have successfully extracted muscle and bladder cells from several patients’ bodies, cultivated these cells in petri dishes, and then layered the cells in three-dimensional molds that resembled the shapes of the bladders. Within weeks, the cells in the molds began functioning as regular bladders which were then implanted back into the patients’ bodies. The team is currently working on re-growing over 22 other different organs including the liver, heart, kidneys and testicles.


Dr. Stephen Badylak, a Research Professor in the Department of Surgery and director of Tissue Engineering at the McGowan Institute for Regenerative Medicine at the University of Pittsburgh, has developed a process which involves scraping cells from the lining of a pig’s bladder, decellularizing (removing cells to leave a clean extracellular structure) the tissue and then drying it to become a sheet or a powder. This cellular matrix powder was used to regrow the finger of Lee Spievak, who had severed half an inch of his finger after getting it caught in a propeller of a model plane.


As of 2011, this new technology is being employed by the military to U.S. war veterans in Texas, as well as to some civilian patients. Nicknamed “pixie-dust,” the powdered extracellular matrix is being used with great success to regenerate tissue lost and damaged due to traumatic injuries. For example, a 19-year old male patient who lost a significant amount of tissue on his heel after a fall from a cliff received two applications of the extracellular matrix powder at Kaiser Hospital in Roseville, California. His heel successfully regenerated, avoiding the necessity of bone and skin grafts, as well as other reconstructive surgery.


In June 2008, at the Hospital Clínic de Barcelona, Professor Paolo Macchiarini and his team, of the University of Barcelona, performed the first tissue engineered trachea (wind pipe) transplantation. Adult stem cells were extracted from the patient’s bone marrow, grown into a large population, and matured into cartilage cells, or chondrocytes, using an adaptive method originally devised for treating osteoarthritis. The team then seeded the newly grown chondrocytes, as well as epithileal cells, into a decellularized (free of donor cells) tracheal segment that was donated from a 51 year old transplant donor who had died of cerebral hemorrhage. After four days of seeding, the graft was used to replace the patient’s left main bronchus. After one month, a biopsy elicited local bleeding, indicating that the blood vessels had already grown back successfully.


In 2009 the SENS Foundation was launched, with its stated aim as “the application of regenerative medicine – defined to include the repair of living cells and extracellular material in situ – to the diseases and disabilities of ageing.” In 2012, Professor Paolo Macchiarini and his team improved upon the 2008 implant by transplanting a laboratory-made trachea seeded with the patient’s own cells.


Because a person’s own (autologous) cord blood stem cells can be safely infused back into that individual without being rejected by the body’s immune system – and because they have unique characteristics compared to other sources of stem cells – they are an increasing focus of regenerative medicine research. The use of cord blood stem cells in treating conditions such as brain injury and Type 1 Diabetes is already being studied in humans, and earlier stage research is being conducted for treatments of stroke, and hearing loss.


Current estimates indicate that approximately 1 in 3 Americans could benefit from regenerative medicine. With autologous (the person’s own) cells, there is no risk of the immune system rejecting the cells, so physicians and researchers are only performing these potential cord blood therapies on children who have their own stem cells available. Researchers are exploring the use of cord blood stem cells in regenerative medicine applications.


Research has demonstrated convincing evidence in animal models that cord blood stem cells injected intravenously have the ability to migrate to the area of brain injury, alleviating mobility related symptoms. Also, administration of human cord blood stem cells into animals with stroke was shown to significantly improve behavior by stimulating the creation of new blood vessels and neurons in the brain. This research also lends support for the pioneering clinical work at Duke University, focused on evaluating the impact of autologous cord blood infusions in children diagnosed with cerebral palsy and other forms of brain injury. This study is examining if an infusion of the child’s own cord blood stem cells facilitates repair of damaged brain tissue, including many with cerebral palsy. To date, more than 100 children have participated in the experimental treatment – many whose parents are reporting good progress. Another report published encouraging results in 2 toddlers with cerebral palsy where autologous cord blood infusion was combined with G-CSF.


As these clinical and pre-clinical studies demonstrate, cord blood stem cells will likely be an important resource as medicine advances toward harnessing the body’s own cells for treatment. The field of regenerative medicine can be expected to benefit greatly as additional cord blood stem cell applications are researched and more people have access to their own preserved cord blood.


On May 17, 2012, Osiris Therapeutics announced that Canadian health regulators approved Prochymal, a drug for acute graft-versus-host disease in children who have failed to respond to steroid treatment. Prochymal is the first stem cell drug to be approved anywhere in the world for a systemic disease. Graft-versus-host disease, a potentially fatal complication from bone marrow transplant, involves the newly implanted cells attacking the patient’s body.


The stem cells found in a newborn’s umbilical cord blood are holding great promise in cardiovascular repair. Umbilical cord blood is blood that remains in the placenta and in the attached umbilical cord after childbirth. Cord blood is collected because it contains stem cells, which can be used to treat hematopoietic and genetic disorders. Researchers are noting several positive observations in pre-clinical animal studies. Thus far, in animal models of myocardial infarction, cord blood stem cells have shown the ability to selectively migrate to injured cardiac tissue, improve vascular function and blood flow at the site of injury, and improve overall heart function.


In review, tissue engineering first took off in the U.S. about 20 years ago. Initially the interest was intensified by the shortage of transplantable organs – hearts, kidneys, lungs, livers, and other donor organs. Over the years, mechanical organs and synthetic body parts have been used, but they have their drawbacks: last a short time, cause blood clots, and promote infections. Seen as the answer, born was the global market for bioengineered tissues and other regenerative medicine products. According to federal estimates, more than $4.5 billion has been invested into tissue-engineering companies since 1990. To date, quite a few lab-made skin and cartilage products have been approved for sale. Many companies and research institutions are pursuing the goal, that organs and other body parts one day will be grown like so much farm produce, to rejuvenate patients suffering from a wide range of ailments. Much progress is being made. The variety of potential applications for lab-grown tissues remains seemingly limitless.


Stem cells allow us, for the first time, to model and study human disease as it actually occurs. Reliance on animal models and cultured cells, while valuable for their overarching insights into the course of disease, fail to fully recapitulate the underlying cellular mechanisms. The generation of stem cell lines from patient blood or skin samples grant an unprecedented means to functionalize the human genetics that, overlay our burgeoning understanding of genes to the natural course of disease.


Cord blood is a rich source of stem cells, which have been used in the treatment of over 75 diseases, including leukemia, lymphoma and anemia. Parents may choose to bank their newborn’s cord blood against the possibility that it will be useful in the future, should the child or a related family member fall victim to a disease that is treatable by cord blood stem cells.

In the United States, the Food and Drug Administration regulates cord blood under the category of “Human Cells, Tissues, and Cellular and Tissue Based-Products.” The Code of Federal Regulations under which the FDA regulates public and private cord blood banks is Title 21 Section 1271. Both public and private cord blood banks are eligible for voluntary accreditation. Other countries also have regulations pertaining to cord blood.


CBS feature first aired on TV March 23, 2008


2013, Wound healing & Tissue Engineering


2013, Ethical Issues in Regenerative Medicine


Regenerative Medicine


2013, Stanford Univ School of Medicine: Institute for Stem Cell Biology & Regenerative Medicine

Advances in Regenerative Medicine


The emerging field of regenerative medicine may one day revolutionize the treatment of heart disease and neurodegenerative disorders, solve the organ donor shortage problem, and completely restore damaged muscles, tendons and other tissues. The key challenge is to give the body a kind of starter kit – made of various proteins, fibers or cells – or to clone extra copies of the semispecialized stem cells that are already found in adult patients and to allow the body to take over from there. The extra help allows the body to regrow tissues of the type or in the amount that it normally could not do by itself. Already such self-healing treatments have somewhat rejuvenated a few patients’ ailing hearts and helped surgeons repair injured muscles. Below is a short update of progress in regenerative medicine.


Stem cells – progenitor cells that can give rise to a variety of tissues – play an important role in this endeavor. Researchers are now learning how to mix sugar molecules, proteins and fibers to create an environment in which the stem cells can develop into replacement tissue. As the following stories show, investigators have made strides in replacing damaged heart tissue and rebuilding muscle. They are also in the early stages of developing new nerve cells. Some of these advances could emerge from the lab as treatments in a few years, or they may take decades, or they may ultimately fail. Here are a few of the most promising ones.


Stem Cells May Transform Treatment for Heart Failure


HEART REPAIR: Harvesting semispecialized stem cells from an ailing heart, helping them to make millions of copies of themselves and injecting those cells into the heart enable the organ to break down scar tissue and grow new muscle. Image: Bryan Christie


In early 2009 Mike Jones bought a newspaper at a convenience store in Louisville, Ky., and read about a local doctor who wanted to try something unprecedented: healing an ailing heart by harvesting and multiplying its native stem cells – immature cells with regenerative powers. Jones, then 65, had congestive heart failure: his heart was no longer pumping blood efficiently. He contacted the doctor, Roberto Bolli of the University of Louisville, and in July of that year Jones became the first person in the world to receive an infusion of his own cardiac stem cells.


Wound Repair with Biological Scaffolding Material


BIOLOGICAL SCAFFOLDING: A cell (center) is embedded in a framework of tissue fibers. By removing the cells and implanting the remaining structure, surgeons may coax the body to grow its own replacement organs. Image: Bryan Christie


Regrowing muscles, tendons and even organs may be possible using nature’s own adhesive. For years biologists were so focused on the internal workings of cells that they pretty much ignored the “glue” that holds those cells together in a body, human or otherwise. And yet once researchers started looking deeper into the stuff between cells, known as the extracellular matrix, they began to realize just how dynamic the whole arrangement is. Not only does the matrix provide the biological scaffolding necessary to keep animal tissues and organs from dissolving into a gooey mess, but it also releases molecular signals that, among other things, help the body heal itself.


Use for 3-D Printers: Creating Internal Blood Vessels for Kidneys, Livers, Other Large Organs


Inner Anatomy


INNER ANATOMY: An ingenious use of sugar molds coated with cells may allow investigators to replicate the sturdy internal vessels that are needed to carry oxygen deep within larger organs, such as the kidneys (shown here), and to remove wastes. Image: Bryan Christie


To build large organs that work properly, researchers need to find a way to lace them with blood vessels


The audiences at TED talks are used to being wowed as they learn about advances in technology. Even by TED standards, however, the 2011 presentation by Anthony Atala of the Wake Forest Institute for Regenerative Medicine was amazing. Unseen by the audience at first, various vials and nozzles hummed with mysterious activity behind Atala while he was on the stage. About two thirds of the way through the talk, a camera zoomed in on the device’s internal armature and showed it weaving back and forth, depositing living cells grown in a laboratory culture layer by layer on a central platform, basing its activity on highly accurate three-dimensional digital renderings. The process, known as 3-D printing, resembles the operation of ink-jet printers but, in this case, instead of ink the printer uses a solution of living cells. In the end, Atala’s machine produced, layer by layer, a life-size kidney made of human cells, much as a personal 3-D printer can spit out, say, a plastic replacement part for a coffeemaker.


Neural Stem Cell Transplants May One Day Help Parkinson’s Patients, Others


BRAIN GROWTH: To replace brain cells lost to neurodegenerative disorders such as Parkinson’s disease, some researchers are experimenting with grafts of fetal brain tissue and injections of young neurons grown from stem cells in the lab. Image: Bryan Christie


Inside the human brain, branching neurons grow beside, around and on top of one another like trees in a dense forest. Scientists used to think that any neurons that wilted and died from injury or disease were gone forever because the brain had no way to replace those cells. By the 1990s, however, most neuroscientists had accepted that the adult brain cultivates small gardens of stem cells that can turn into mature neurons.


Neurodegenerative disorders devastate the brain, but doctors hope one day to replace lost cells. Imagine the scourge of Alzheimer’s Disease wiped out, when these lost cells can be replaced with new ones. Source: May 2013

2013 Printing a Human Kidney, Anthony Atala MD

2012 New Yorker sponsored MED Talks: Anthony Atala MD

Surgeon and Researcher, Anthony Atala 2012

(ignore the really dumb intro here)

Columbia University: Professor Jeremy Mao

Lab-Grown Genitals and Spray-on Skin


New kidneys, ears and finger bones are among the body parts being grown at Wake Forest University’s Institute for Regenerative Medicine


A few years ago, Dr. Anthony Atala’s lab at Wake Forest University got good at making ears. They were growing new ears on a scaffold using patient’s cells, because so many soldiers were losing their ears in explosions. Now the Department of Defense has a project that’s closer to Atala’s heart: making new genitals for soldiers who have stepped on bombs. Other labs are still moving forward with the ear project for the military. But Atala has special expertise dating back to his days as a pediatric urologist. He’s already grown bladders using a patient’s own cells, and he’s made penises that rabbits were able to put to their proper use, fathering litters of new little bunnies. He hopes to use this expertise to help rebuild the bodies of veterans wounded in Iraq and Afghanistan, as well as men and boys injured in car accidents.


Atala is one of the pioneers of regenerative medicine. But the field has taken off in a big way, attracting biotechnology companies, the Armed Forces Institute of Regenerative Medicine (AFIRM) and academic labs, which are working to literally make the blind see and the lame walk again. AFIRM currently funds around 50 research labs, including leaders such as the University of Pittsburgh Medical Center, Rutgers University, the Cleveland Clinic and Rice University. These labs are perfecting spray-on skin and aim to mass-produce new body parts using bioprinters based on the jet printers attached to your home computer.


All of this technology is years away with the most advanced treatments have just begun the very earliest stages of human testing. But all evidence points to the tantalizing prospect of grow-your-own organs and possibly even limbs within a decade or so, and some approaches, such as muscle transplants and spray-on skin, are helping a lucky few now.


Rebuilding the lower abdomen, the genitals, the pelvic area and the bladder are among the least talked-about injuries but among the most horrible affecting war veterans. The improvised explosive devices, or IEDs, planted by insurgents across Iraq and Afghanistan blow off feet, legs and arms, and they can especially damage the pelvic areas that are difficult to protect with body armor. Atala’s lab is also working to make kidneys, muscle implants, and even to find ways to get fingers to regenerate on their own. (It has to do with waking up some very powerful DNA that goes to sleep soon after a fetus develops).


Spray-on Skin


One area of intense competition – or collaboration – is in spraying on new skin. AFIRM is funding several projects testing a product that uses a patient’s own skin cells, so that rejection is not an issue. Old-fashioned skin grafts may close a wound or a burn, but they don’t heal prettily. ReCell is a product, more of a process really, that uses a small plug of a patient’s own skin, broken down into a soup using enzymes. Cells known as keratinocytes, which give skin its structure, and the melanocytes, which give color, are pulled out, mixed into a liquid suspension and then sprayed over the damaged area. It’s a thin layer but the cells quickly multiply and, if the process is done right, form an even layer of new skin within days. The result is much more natural-looking than a graft.


‘Happy to be alive’: Blast amputees confront uncertain road ahead


Nearly a dozen victims of the bomb blasts at the Boston Marathon, have undergone amputations, and even more may have lost or threatened limbs, hospital officials said. It will take sustained support to aid their recovery, including help from other amputees. Skin is easier to heal because it’s a relatively simple organ and on the surface of the body. Limbs are more complicated – they are made up of bone, muscle, nerves, connective tissue and also skin. Labs are taking a more traditional approach in trying to restore limbs, by transplanting them. But even there regenerative medicine can play a role. This is where stem cell research comes in. Stem cells are the body’s master cells, and there are several kinds. People have stem cells known as adult stem cells all through their bodies, and they are already partly “educated” to become blood, muscle, bone or nerve cells. These cells divide and multiply to produce muscle, bones and blood, and they also secrete compounds that help existing tissue and cells regenerate. Some of the projects on AFIRM’s wish list include calls for labs that can combine techniques used to build new body parts with the use of stem cells to help them generate and integrate with the rest of the body.


More powerful cells come from embryos that have barely begun to develop. An entire human body, the collection of muscle, bone, brain, blood, nerves and organs, all develops from the ball of just a few cells that forms days after fertilization. Each one of these cells, known as human embryonic stem cells, contains all the coding needed to make every cell type in the body.


Helping the Blind to See


In 2012, scientists at Advanced Cell Technology, based in Massachusetts, reported they had used some of these human embryonic stem cells to partially restore vision in two legally blind patients. First they “trained” the cells by incubating them in a nourishing soup of chemicals designed to make them differentiate into retinal cells. The stem cells, infused directly into the eye, regenerated cells known as retinal pigment epithelium cells. One patient said she can thread needles again and another has been able to resume shopping on her own. ACT has since gotten permission to treat more patients with higher doses of the cells, now that they have at least been shown not to cause any harm. They’re going after patients with degenerative eye diseases such as age-related macular degeneration and Stargardt disease. In both conditions the cells in the retina gradually die and patients go slowly and irreversibly blind.


Geron was the first company to test human embryonic stem cells in people. In 2010 it ran a clinical trial infusing the cells into a young man injured in a car accident, as well as three others. The hope was to regenerate their severed spinal cords. Again, these first patients were treated experimentally only to show the approach was safe. Unfortunately, Geron dropped its stem cell program in November 2011, saying it wanted to focus on cancer drugs instead.


‘I Was Afraid it Would Be a Dream’


Another patient, Ted Harada had a second infusion of stem cells in 2012. The 40-year-old former Fedex employee has Lou Gehrig’s disease, medically known as amyotrophic lateral sclerosis (ALS). ALS attacks nerves called motor neurons, gradually and inexorably paralyzing its victims. It’s always fatal as patients lose every bit of their ability to move, even to breathe. There’s no treatment and no cure. Harada is hopeful enough to have tried the highly experimental treatment not once but twice. It’s painful – surgeons have to cut open his spine and infuse the stem cells right into his spinal fluid. But the last time Harada was treated, he went from walking with a cane to running with his kids. However, the effects did gradually wear off so in August 2012, Harada got a second infusion of stem cells, which are made by a company called Neuralstem, this time in his neck. Time will tell how this turns out.


2013, How Europe can build on its leading position in regenerative medicine – Webinar

Pluristem’s PLX-PAD Cells’ Safety in Human Lung Models of Pulmonary Hypertension


Umbilical cord blood is blood that remains in the placenta and in the attached umbilical cord after childbirth. Cord blood is collected because it contains stem cells.


Pluristem Therapeutics Inc. a leading developer of placenta-based cell therapies, have announced that independent researchers from the Queensland Lung Transplant Service at the University of Queensland, Australia demonstrated that following infusion of Pluristem’s PLX (Placental eXpanded) cord cells in a human lung model of pulmonary arterial hypertension (PAH), blood flows were maintained and no adverse hemodynamic effects were noted. The findings were presented on April 25, 2013 at the 33rd Annual Meeting and Scientific Sessions of the International Society of Heart and Lung Transplantation (ISHLT) in Montreal, Canada. For the study, Daniel C. Chambers MD et. al. from Prince Charles Hospital and the University of Queensland, Australia induced pulmonary hypertension, ex vivo, in four human lungs that had been declined for use in transplantation. Supra-therapeutic doses of Pluristem’s PLX-PAD cells were then infused over 15 minutes directly into the pulmonary artery. Pulmonary vascular resistance stabilized during and for the hour post PLX-PAD infusion without adverse hemodynamic manifestations. The authors concluded they have demonstrated the acute hemodynamic safety of supra-therapeutic doses of PLX-PAD cells in an ex vivo model of pulmonary arterial hypertension.


Pluristem’s PLX cells are mesenchymal-like adherent stromal cells (ASCs) derived from full term human placentas. These cells are expanded in the company’s proprietary bioreactor system that creates a three-dimensional (3D) microenvironment. This 3D technology allows for the controlled, large-scale growth of cells implementing an optimized, standardized, scaled-up and “hands-off” operation. This allows PLX cells to be mass-produced with batch-to-batch consistency for a fraction of the cost of traditionally expanding cells using petri dishes or tissue flasks. Furthermore, Pluristem’s 3D expansion technology allows for the production of specific PLX cell products for each of the Company’s targeted indications.

TARGET HEALTH excels in Regulatory Affairs. Each week we highlight new information in this challenging area


FDA Approves Procysbi for Nephropathic Cystinosis, a Rare Genetic Condition


Cystinosis is a rare genetic condition that affects an estimated 500 patients in the United States and about 3,000 patients worldwide. Fatal if not treated in early childhood, cystinosis causes a protein building block called cystine to build up in every cell of the body. The buildup of cystine causes kidney problems, which can cause the body to lose too much sugar, proteins and salts through the urine. Cystinosis may lead to slow body growth and small stature, weak bones and developing and worsening kidney failure. There are three types of cystinosis, the most severe being nephropathic cystinosis, which severely damages the kidneys. Currently the FDA approved drugs used to treat cystinosis include Cystagon (cysteamine bitartrate), an immediate-release tablet that was approved in 1994, and Cystaran (cysteamine ophthalmic solution) eye drops, approved last year to treat corneal cystine crystal accumulation.


The FDA has approved Procysbi (cysteamine bitartrate) for the management of nephropathic cystinosis in children and adults. Procysbi is a delayed-release capsule intended for patients ages 6 years and older. While Cystagon is taken every six hours around the clock to control cystine levels, Procysbi is a long-acting formulation that is taken every 12 hours. Procysbi was granted orphan product designation because it is intended to treat a rare disease or condition.


“Procysbi is the only delayed-release product approved by FDA to treat nephropathic cystinosis, offering patients with this rare disease an important new treatment option,” said Andrew E. Mulberg, M.D., deputy director, Division of Gastroenterology and Inborn Errors Products, Center for Drug Evaluation and Research, FDA.


The major study supporting Procysbi’s safety and effectiveness involved 43 adult and pediatric patients with nephropathic cystinosis. Patients were randomly assigned to receive Cystagon or Procysbi for three weeks before being switched to the other product for an additional three weeks. Blood testing showed Procysbi was as effective as Cystagon in controlling cystine levels.


The most common side effects in patients treated with cysteamine products include nausea, bad breath, abdominal pain, constipation, indigestion or upset stomach, headache, drowsiness and dizziness. Other uncommon but serious side effects include ulcers or bleeding of the stomach or intestine, altered mental state, seizures, severe skin rashes and allergic reactions.


Procysbi is marketed by Novato, Calif.-based Raptor Pharmaceuticals.


Asparagus Gruyere With Eggs and Bacon Bits



We emphasize asparagus, because the tender stalks are in season right now.


If we let asparagus alone it would grow into four- to six-foot tall ferns. Instead we harvest the young sprouts to grace our spring tables. Asparagus takes a lot of space and time (an asparagus field takes three years to yield a harvest), which is how it got its rep as a luxury vegetable.


Asparagus is more widely available and less expensive than it ever used to be. But in the Spring it should get the royal treatment – served as its own course. We may choose to steam it, but pan roasting, grilling, stir-fry and just using asparagus raw, are all healthy ways of preparing it. We don’t recommend boiling veggies, as vitamins and minerals may be lost. In the Springtime, asparagus are wonderful served without cooking at all.




2  pounds thin asparagus, trimmed and washed

4 tablespoons extra virgin olive oil

Pinch of freshly ground black pepper (or grind to your taste)

Pinch freshly grated nutmeg

1/3 to 2/3 cup grated Gruyere (see note)

1/2 cup Bacon Bits (real cooked or artificial) or finely minced prosciutto

2 Tablespoons minced parsley

4 eggs, beaten

3 to 4 tablespoons freshly grated Mozzerella




1. Preheat the oven to 300 degrees.

2. When asparagus is Spring-tender, don’t cook.  Spring season in the East and Midwest is March through the end of June.  Simply wash, dry, break tough ends off, and cut into 1-to-1.5-inch lengths.

3. Put the cut asparagus in a bowl. Add the olive oil and season with the pepper and nutmeg. Stir, to cover the asparagus completely with the oil mixture.

4. Turn the asparagus and the oil mixture into a 9- or 10-inch pie plate. Arrange in an even layer. Sprinkle with the Gruyere, bacon bits or prosciutto and parsley. Pour the beaten eggs on top, gently shaking the pan to distribute.

5. Bake until the eggs are set into a custard and a slight crust forms on top, about 15-20 minutes.

6. Remove from oven and sprinkle the mozzarella over the top and return to oven for another 5-10 minutes, or until the cheese melts.  Serve hot or warm.  Serves 2 to 4.



Note: You may use Fontina in place of the Gruyere.


This quick and easy dish is perfect for weekend brunch or any lunch. If my husband is travelling it’s good for a solo dinner. If he’s at home I would serve this delicious dish for dinner, with a simple tossed salad, a smokey-oaky Sauvignon Blanc and a cheese and fruit platter.