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Notre Dame establishes professorships in adult stem cell research

By daniellenierenberg

Public release date: 22-Jun-2012 [ | E-mail | Share ]

Contact: William Gilroy gilroy.6@nd.edu 574-631-4127 University of Notre Dame

Alumnus Michael Gallagher and his wife, Elizabeth, have made a $5 million gift to establish the Elizabeth and Michael Gallagher Family Professorships in Adult Stem Cell Research at the University of Notre Dame.

Their gift, which will fund three new endowed professorships in adult and all forms of non-embryonic stem cell research, will strengthen Notre Dame's leadership in the field of stem cell research and enhance the University's effective dialogue between the biomedical research community and the Catholic Church on matters related to the use and application of stem cells and regenerative medicine.

"As a Catholic university, Notre Dame carries a mantle of responsibility to use our scholarship and resources to help alleviate human suffering, and, in this area of research in particular, to do so with deep respect for the sanctity of all human life," said Rev. John I. Jenkins, C.S.C., the University's president. "These new professorships will enable us to effectively build upon an already strong foundation in this critically important field. We are tremendously grateful to the Gallaghers for making this possible with their transformative gift."

Despite years of research, there are no known cures for a large number of degenerative diseases, such as Type 1 diabetes, Parkinson's disease, cardiovascular disease, macular degeneration and spinal cord injuries. Stem cell research has the potential to contribute to the discovery of new and successful treatments for these and other diseases because it holds the unique promise of regenerating damaged cells and tissues, fully restoring tissues and organs to their normal function.

Although this vital area of research could accelerate the ability to alleviate much human suffering, it has generated extensive ethical debate with the use of embryonic versus non-embryonic stem cells. The Catholic Church affirms the dignity of all human life at every stage and vigorously opposes the destruction of human embryos for the harvesting of stem cells. At the same time, the Church strongly endorses the use of adult and non-embryonic stem cell research as a potential therapy for individuals suffering from these debilitating diseases. Research has demonstrated that adult stem cells, including all forms of non-embryonic stem cells, such as induced pluripotent stem cells and umbilical cord stem cells, can be harvested and programmed to achieve pluripotency the same characteristic that enables embryonic stem cells to differentiate into any type of cell.

An urgent need exists to increase the number of faculty experts performing adult stem cell research at Notre Dame. Doing so will expand upon the strong foundation the College of Science holds in these areas and will help create an environment for excellence in which faculty and students can learn, grow, collaborate and ultimately affect human health.

"We are overwhelmed with gratitude at the generous gift from Mike and Liz Gallagher," said Gregory P. Crawford, dean of the College of Science. "The impact of this gift is truly beyond measure. It will play a crucial role in attracting three more of the best faculty in the field of adult stem cell research to Notre Dame. Furthermore, this gift will equip our existing talented group of adult stem cell researchers at Notre Dame to take the next great leap toward ultimately forming a premier center in adult stem cell research."

Michael Gallagher is a 1991 graduate of Notre Dame, and his wife, Elizabeth, is a 1992 graduate of Saint Mary's College. They have two sons, Brock and Jack, and currently live near Denver.

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Husband competes to raise awareness about bone-marrow registration

By daniellenierenberg

FOR THE PAST decade, two things have consumed large chunks of Malvern native Tom Kramer's time.

The first is his training regimen. Kramer, 46, is a practicing triathlete who will compete Saturday morning in the eighth annual Philadelphia Insurance Triathlon in Fairmount Park.

The second is the search for a bone-marrow match for his wife Pam, also a triathlete, who was diagnosed with a rare form of leukemia in 2000 and eventually willl need a bone marrow transplant.

At some point, Kramer made a creative decision to have those cumbersome obligations intersect. Desperate to spread the word about the importance of registering as a bone-marrow donor he estimates only 9 million people are registered Kramer embarked on a four-event quest over the span of 8 months to raise awareness.

"It was just me in the beginning," he said. "All I had was a banner and some testing kits."

Kramer completed a marathon, two Ironman half-triathlons and a full Ironman triathlon. Eventually his effort gained steam, finally culminating last year when the Kramers incorporated their hard work into the non-profit Racing to Register.

Using endurance sports as a platform, Racing to Register aims to enlarge the pool of potential donors for blood cancer patients in need of lifesaving bone marrow or stem cells.

"We think that the endurance part the reason we chose that platform is that you have to have a lot of endurance to go through that kind of treatment," Kramer said. "There is that marriage there if we can put ourselves through this, you can register."

Athletes that who join Team RTR complete the donor registration process and, in return, the program facilitates their endurance training through coaching, discounted gear and more.

While his wife's illness is what got him started, Kramer says the event has grown into something much bigger. With more than 2,100 registrants, RTR has produced four potential matches.

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Columbia Asia Referral Hospital, Yeshwantpur Announces a Comprehensive Bone Marrow Transplant Service

By JoanneRUSSELL25

BANGALORE, June 22, 2012 /PRNewswire/ --

Adding an impetus to the already existing image of Bangalore being a healthcare destination of India, Columbia Asia Referral Hospital, Yeshwanthpur (CARHY), announced comprehensive bone marrow transplant (stem cell transplant) service on Thursday. This facility will give hope to many cancer patientsin and around Bangalore as there are very few hospitals in South India providing allogeneic transplant, which involves using stem cellsfrom a donor with a similar genetic makeup.

The bone marrow transplant (BMT) service will have a team of medical experts including clinical hematologist, oncologist, and other qualified doctors from allied specialties like pediatrics, infectious disease specialist and trained nurses for stem cell transplant, state-of-the-art HEPA filtered room, ICU, 24 hrs blood bank services and radiology services for providing comprehensive care during stem cell transplant.

Addressing the media, Dr. Nandakumar Jairam, Chairman and Group Medical Director, Columbia Asia Hospitals,said, "We are happy to announce allogenic bone marrow transplant service at our hospital in Yeshwanthpur, over and above the existing autologous transplant service. This will enhance comprehensive bone marrow transplant treatment delivery; a dire need for the people of Karnataka and neighbouring states. This will also help many international patients who look for such a treatment in India."

"This facility is dedicated to providing end-to-end services including expert counsel from a clinical hematologist and an entire team of doctors and nurses providing the latest in medical advances to those suffering from blood cancer and some non-cancerous conditions affecting thebone marrow," said Dr. Satish, Consultant in Clinical Hematology, Columbia Asia Hospitals.

"Bone marrow transplant, also called hematopoietic stem cell transplant (HSCT), is a treatment optionfor certain cancers. With this launch, Columbia Asia Referral Hospital Yeshwanthpur becomes one among the very few centers in India to offer allogeneic bone marrow transplants. Till now, we were doing only autologous transplants which involved the usage of the patient's own stem cells. Now, we will be able to manage conditions like high risk leukemia's, myelomas and lymphomas," said Dr Satish.

"Some of the most effective treatments for cancer such as chemotherapy and radiation are toxic to the bone marrow.The marrow produces different cells that make up the blood such as red blood cells, white blood cells and platelets. The stem cells from the bone marrow are extracted before the administration of high dose chemotherapy and then reintroduced or transplanted to the patient so that blood cell production process is re-established in the bone marrow," addedDr Neelesh Reddy, Consultant Medical Oncology, Columbia Asia Hospitals.

In fact earlier stem cells were collected only from the bone marrow in the hip bones under general anesthesia. However with advanced technology and medical supervision stem cells can now be collected from peripheral blood after giving injections. Stem cells are then harvested by simple procedure called apheresis, (in the same way as dialysis is done) and the rest of the blood is returned to the person.

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Stem cell therapy gives dog new lease on life

By Sykes24Tracey

Stem cell therapy has gone to the dogs. The technology aimed at giving ailing pets a new lease on life has arrived in Hawaii.

13-year-old Kumba is still a bit dazed, coming out of general anesthesia. The veterinarian at Surf Paws Animal Hospital just extracted about two tablespoons of fat tissue from the dog. Stem cells from that fat tissue will then be used to help him with his arthritis.

"Once we get the stem cells then we do some extra processing steps to wake them up so that they're very active. At the end of that, the veterinarian will inject the stem cells into the areas of damage," says Carol Spangler Vaughn of Medivet America.

A company called MediVet America is bringing the technology to animal hospitals in Hawaii. This is a first for Oahu. The company says the procedure works on other animals with different types of ailments.

"So the nice thing about this we're not gonna give you a puppy back but we'll give you some nice quality time with your animal. You won't have to put them down because of their arthritis," Vaughn said.

Kumba's arthritis had gotten worse in the past five years, and his owners were wondering whether it was best to end his life to stop him from suffering.

'When we start saying things like oh we don't know how much longer, poor Kumba, he must be in a lot of pain. That kind of stuff really hits home especially since he's been with us for so long," said Rumi Hospodar Kumba's owner.

But with this new procedure, they're counting on Kumba to be pain free in a few weeks and are looking forward to get backdoing some of the things Kumba enjoyed, like swimming.

"He can't do that now since his joints are so bad, and he's getting so old so that's one of the many things I'm looking forward to," Kelsea Hopsodar, his other owner said.

The cost of the procedure runs from 24 to 28 hundred dollars, and it's covered by most pet insurance policies.

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Stem cell therapy in Hawaii going to the dogs

By JoanneRUSSELL25

HAWAII KAI (HawaiiNewsNow) -

Cutting-edge technology is helping Hawaii's pets live better lives for months, even years. We were there as a beloved dog named Kumba received one of the first-ever, in-clinic stem cell therapy surgeries in the islands.

13 year old Kumba doesn't know he's a guinea pig. The Rottweiler-Lab mix is one of the first in Hawaii to undergo the stem cell procedure at Surf Paws in Hawaii Kai.

Kumba suffers severe arthritis in his hips and knees, doesn't eat much, and is even a bit depressed. "It's an effort for him to get up off the floor, and when he gets up and crosses the room, you can see the stiffness," says his owner, Rumi Hospodar.

Kumba's kids learn some of details of his surgery. Then, he's moved to a table and nods off from anesthesia. Once he's prepped, the procedure begins. The vet removes about two tablespoons of fat tissue from Kumba's shoulder. From there, the stem cells are separated from the fat and activated. Then, they're injected back into the affected areas.

The entire process takes four hours, but the dog is actually only under for about 20 minutes. Surf Paws used to send the tissue to the mainland for processing, but with technology from Medi-Vet America, they can do it all here.

"The patient had to be, you know, go home and come back a few days later and the timing was a little bit difficult. Now, everything is same day," says Surf Paws veterinarian Dr. Cristina Miliaresis.

Cost depends on the size of animal but can run up to $2,800. It's mainly done on dogs, cats, and horses who suffer osteoarthritis, hip dysplasia, ligament and cartilage damage, and other degenerative diseases. Their quality of life can improve within a couple of weeks.

Dr. Miliaresis says, "Some people might say, 'Oh, the dog's 13. Why are you doing this for a 13 year old dog? But even 6 months, pain-free, after a very, it's not simple, but it's a pretty straightforward procedure, to me (would be) just amazing."

The techs move all 97 pounds of Kumba to post-op - while his anxious owner looks on.

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Stem Cells To Aid In Heart-Related Research

By NEVAGiles23

June 21, 2012

Connie K. Ho for redOrbit.com

Pumping vigorously night and day, the heart is clearly one of the most important organs in the human body. It is also one of the most delicate parts of the body. As such, news regarding heart-related diseases is beneficial to both doctors and patients. University of Michigan (UM) researchers recently reported the discovery of a new method that could produce cardiac muscle patches from stem cells.

The innovative process was created at UMs Center for Arrhythmia Research and effectively uses stem cells that can copy the hearts squeezing action. The cells showed activity that was like that of peoples resting heart rate. The rhythmic electrical impulse transmission of the engineered cells worked at a rate of 60 beats per minute and this rate was 10 times quicker than rates reported in other stem cell studies.

To date, the majority of studies using induced pluripotent stem cell-derived cardiac muscle cells have focused on single cell functional analysis, remarked senior author Dr. Todd J. Herron, an assistant research professor in the Departments of Internal Medicine and Molecular & Integrative Physiology at the U-M, in a prepared statement.

The researchers believe that the stem biology findings will be beneficial to those who suffer from common but life-threatening heart diseases. They hope that the use of stem cells will assist patients diagnosed with arrhythmia, which is found in approximately 2.5 million people. With arrhythmia, patients suffer an irregularity in the hearts electrical impulses and this can hinder the hearts ability to circulate blood.

For potential stem cell-based cardiac regeneration therapies for heart disease, however, it is critical to develop multi-cellular tissue like constructs that beat as a single unit, commented Herron in the statement.

Regarding the specifics of the project, the goal of the scientists was to use stem cells to develop skin biopsies. These biopsies could be used to produce large quantities of cardiac muscle cells, which could then help transmit uniform electrical impulses and work as a cohesive unit. In collaborating with researchers from the University of Oxford, Imperial College, and the University of Wisconsin, the team was able to design a fluorescent imaging platform. The platform used light emitting diode (LED) illumination to quantify the cells electrical activity.

Action potential and calcium wave impulse propagation trigger each normal heart beat, so it is imperative to record each parameter in bioengineered human cardiac patches, remarked Herron in the statement.

Overall, authors of the study believe that the velocity of the engineered cardiac cells is still slower than the velocity of cells found in the beating adult heart. However, the velocity of the engineered cardiac cells is quicker than those previously reported; it is also similar to the rate found in commonly used rodent cells. For future scientific research purposes, the investigators theorize that human cardiac patches could be utilized instead of rodent systems. The new method could be used in many cardiac research laboratories and allow cardiac stem cell patches to be utilized in disease research, new drug treatment testing, and therapies focused on repairing damaged heart muscles.

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Scientists grow tiny liver in mouse's head

By raymumme

Using stem cells from human skin, Japanese scientists have grown a small human liver inside the skull of a mouse.

Hideki Taniguchi and Takanori Takebe from Yokohama City University used stem cells generated from human skin cells and developed them into percussor liver cells, the New Scientist reports.

Then they added other cells from umbilical cord blood vessels. The combination of cells then "guided itself" to form a small structure similar to liver tissue, Takebe said.

"We mixed and graded the cells onto the culture dish and they moved to form a cluster," he said. "It was a surprising outcome from what was, to be honest, an accident."

They implanted the structure into the head of a mouse, which was suffering from a severe genetic immune system disorder that prevented it from having an immune reaction to the foreign tissues.

The increased blood flow in the mouse's skull allowed the tissue to keep growing.

Within 48 hours, human blood vessels and human proteins formed. Glycogen and amino acids levels were the same as those of a human liver.

"It's not yet a perfect liver," Takebe said. "Improvements need to be made, such as the reconstruction of a bile duct."

The study could be significant for the field of regenerative medicine, but the researchers aren't yet sure whether the organ is a fully functioning liver, or whether they will be able to scale it to human size.

The findings were presented at the at the International Society for Stem Cell Research's annual meeting in Yokohama.

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StemCells, Inc. Initiates Phase I/II Clinical Trial in Dry Age-Related Macular Degeneration

By daniellenierenberg

NEWARK, Calif., June 21, 2012 (GLOBE NEWSWIRE) -- StemCells, Inc. (STEM) today announced initiation of a Phase I/II clinical trial of the Company's proprietary HuCNS-SC(R) product candidate (purified human neural stem cells) in dry age-related macular degeneration (AMD) referred to as Geographic Atrophy. There are no approved treatments for dry AMD.

The trial is being conducted at the Retina Foundation of the Southwest's (RFSW) Anderson Vision Research Center in Dallas, Texas, one of the leading independent vision research centers in the United States. David G. Birch, Ph.D., Chief Scientific and Executive Officer of the RFSW and Director of the Rose-Silverthorne Retinal Degenerations Laboratory, is the principal investigator of the study.

"Dry AMD is the most common form of macular degeneration, and has a very debilitating effect on quality of life," said Dr. Birch. "Transplanting neural stem cells to protect photoreceptors in patients diagnosed with AMD is an innovative, but logical, approach, well supported by the Company's recently published preclinical data. We are very excited to be conducting this trial at RFSW."

A summary of the Company's preclinical data was featured in the February 2012 issue of the international peer-reviewed European Journal of Neuroscience (available online at http://onlinelibrary.wiley.com/doi/10.1111/j.1460-9568.2011.07970.x/abstract). The data demonstrated that HuCNS-SC cells protect host photoreceptors and preserve vision in the Royal College of Surgeons (RCS) rat, a well-established animal model of retinal disease which has been used extensively to evaluate potential cell therapies. Transplantation of HuCNS-SC cells significantly protects photoreceptors from degeneration. Moreover, the number of cone photoreceptors, which are responsible for central vision, remained constant over an extended period, consistent with the sustained visual acuity and light sensitivity observed in the study. In humans, degeneration of the cone photoreceptors accounts for the unique pattern of vision loss in dry AMD.

"Unlike others in the field, our clinical strategy is to preserve visual function before it is lost," said Stephen Huhn, MD, FACS, FAAP, Vice President and Head of the CNS Program at StemCells, Inc. "Our published preclinical data provides a strong rationale for this approach in dry AMD and we hope to replicate these results in this clinical trial. We are very pleased to be working with Dr. Birch and the Retina Foundation of the Southwest, who have the expertise and referral base to undertake this important study. We anticipate that we will be able to accrue the requisite number of patients for this trial in relatively short order."

About Age-Related Macular Degeneration

Age-related macular degeneration refers to a loss of photoreceptors (rods and cones) from the macula, the central part of the retina. AMD is a degenerative retinal disease that typically strikes adults in their 50s or early 60s, and progresses painlessly, gradually destroying central vision. According to the RFSW website, there are approximately 1.75 million Americans age 40 years and older with some form of age-related macular degeneration, and the disease continues to be the number one cause of irreversible vision loss among senior citizens in the US with more than seven million at risk of developing AMD.

About the Trial

The Phase I/II trial will evaluate the safety and preliminary efficacy of HuCNS-SC cells as a treatment for dry AMD. The trial will be an open-label, dose-escalation study, and is expected to enroll a total of 16 patients. The HuCNS-SC cells will be administered by a single injection into the space beneath the retina in the most affected eye. Patients' vision will be evaluated using both conventional and advanced state-of-the-art methods of ophthalmological assessment. Evaluations will be performed at predetermined intervals over a one-year period to assess safety and signs of visual benefit. Patients will then be followed for an additional four years in a separate observational study. Patients interested in participating in the clinical trial should contact the site at (214) 363 3911.

About HuCNS-SC Cells

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Scientists Can Now Grow Functioning Liver From Stem Cells [Medicine]

By Dr. Matthew Watson

Scientists have promised a lot of regenerative medicine will come from stem cells, but so far progress has been fairly slow: they can stimualte regrowth of heart tissue, make incredibly expesnive artifical blood, orat bestconstruct a short piece of vein. Now, though, scientists are claiming they can grow functional liver.

Nature reports that a team of scientists from Japan has presented its works at a conference, and it's incredible. In fact, George Daley, director of the stem-cell transplantation program at the Boston Children's Hospital in Massachusetts, told Nature that "it blew [his] mind." Wow.

The researchers used stem cells created from human skin cells, then placed the cells on growth plates in a specially designed culture medium. Over the course of nine days, the cells started producing chemicals that a typical liver cell, otherwise known as a hepatocyte, would produce. They then added endothelial and mesenchymal cellswhich form parts of blood vessels and other structural tissues within the bodyto the mix, in the hope that they would be incorporated and begin to help the cells develop a structure akin to the liver.

The result was amazing: two days later, the researchers found the cells assembled into a 5-millimeter-long, three-dimensional lump. That lump was almost identical to something known as a liver budan early stage of liver development. From Nature's report:

"The tissue lacks bile ducts, and the hepatocytes do not form neat plates as they do in a real liver. In that sense, while it does to some degree recapitulate embryonic growth, it does not match the process as faithfully as the optic cup recently reported by another Japanese researcher. But the tissue does have blood vessels that proved functional when it was transplanted under the skin of a mouse. Genetic tests show that the tissue expresses many of the genes expressed in real liver. And, when transferred to the mouse, the tissue was able to metabolize some drugs that human livers metabolize but mouse livers normally cannot. "

While it's not perfect, it's the first time anyone has successfully created part of a functional human organ from stem cells produced from human skin. If scientists hadn't quite managed to deliver on the promise of stem cells so far, they have now. [Nature]

Image by Spirit-Fire under Creative Commons license

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Makucell Unveils Renewnt™, a Revolutionary, Science-driven Skin Care Brand

By NEVAGiles23

SCOTTSDALE, Ariz. & LOS ANGELES--(BUSINESS WIRE)--Makucell, Inc., a pioneering regenerative biotechnology company dedicated to the development, manufacture and distribution of non-prescription products formulated to address the impact of aging and photo-damaged skin unveils the Renewnt (pronounced Re-new-int) brand, a revolutionary science-driven product line. Renewnts proprietary ingredient, Asymmtate, is a new approach to cellular aging, optimizing signals in the Wnt (pronounced wint) pathway to energize the skins stem cells, encouraging youthful cell behavior. The result is younger-looking skin which appears firmer and smoother. This molecular process is the key to our proprietary technology developed at USCs Keck School of Medicine and transferred to Makucells Renewnt skin care line.

We conducted standard industry safety tests, and the results were universally positive; Renewnt products were well tolerated with no adverse effects or safety issues. A combination of clinical trials and in vitro gene expression studies from treated biopsied skin continues to corroborate the aesthetic effects noted in the clinic.

The Makucell Science

The bodys signals govern skin stem cells, controlling the decision to remain dormant, divide or differentiate (become normal, active tissue cells). Signals flow in pathways and multiple paths converge into one the Wnt pathway. Makucells proprietary molecule Asymmtate encourages optimal signaling in the Wnt pathway. Optimal signaling stimulates the skin stem cells to begin the process leading to keratinocytes, fibroblasts and other dermal cells which produce collagen, elastic tissue and substances in the supporting skin matrix. This essential regenerative process is the key differentiator in Makucells Renewnt skin care products.

Michael Kahn, Ph.D. and his team of gifted research scientists at the Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research of the Keck School of Medicine at the University of Southern California developed the revolutionary ingredient, Asymmtate, Makucells core technology. Dr. Mark V. Dahl, Makucell Chief Medical Officer and former President of the American Academy of Dermatology as well as Professor Emeritus at the Mayo Clinic in Arizona, developed the formulations designed to target a specific skin type on a particular area of the body. Makucell has tested all the Renewnt products for safety and efficacy. The products:

Makucell is committed to supporting our claims with results from controlled, blinded studies, explains Dr. Dahl. We conducted standard industry safety tests, and the results were universally positive; Renewnt products were well tolerated with no adverse effects or safety issues. A combination of clinical trials and in vitro gene expression studies from treated biopsied skin continues to corroborate the aesthetic effects noted in the clinic.

Dr. Lawrence Rheins, President and Chief Executive Officer of Makucell, commented, Renewnt delivers extraordinary regenerative ability in a hydrating cream, providing an advanced anti-aging option. Asymmtate in Renewnt wakes-up the skins stem cells which have become sluggish with age, to begin rebuilding the underlying supporting skin matrix. As a result, skin looks plumper and has a rejuvenated, youthful appearance.

Makucells current Renewnt skin care product line includes:

Renewnt for Hydration, a day and night facial moisturizer for a more youthful-looking appearance.

Renewnt for Strength, for the dry, thinning skin on hands and forearms to seal in moisture, repair the signs of aging and restore the essential skin barrier.

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'Master molecule' may improve stem cell treatment of heart attacks

By Dr. Matthew Watson

ScienceDaily (June 20, 2012) Johns Hopkins researchers have discovered that a single protein molecule may hold the key to turning cardiac stem cells into blood vessels or muscle tissue, a finding that may lead to better ways to treat heart attack patients.

Human heart tissue does not heal well after a heart attack, instead forming debilitating scars. However, for reasons not completely understood, stem cells can assist in this repair process by turning into the cells that make up healthy heart tissue, including heart muscle and blood vessels. Recently, doctors elsewhere have reported promising early results in the use of cardiac stem cells to curb the formation of unhealthy scar tissue after a heart attack. But the discovery of a "master molecule" that guides the destiny of these stem cells could result in even more effective treatments for heart patients, the Johns Hopkins researchers say.

In a study published in the June 5 online edition of journal Science Signaling, the team reported that tinkering with a protein molecule called p190RhoGAP shaped the development of cardiac stem cells, prodding them to become the building blocks for either blood vessels or heart muscle. The team members said that by altering levels of this protein, they were able to affect the future of these stem cells.

"In biology, finding a central regulator like this is like finding a pot of gold," said Andre Levchenko, a biomedical engineering professor and member of the Johns Hopkins Institute for Cell Engineering, who supervised the research effort.

The lead author of the journal article, Kshitiz, a postdoctoral fellow who uses only his first name, said, "Our findings greatly enhance our understanding of stem cell biology and suggest innovative new ways to control the behavior of cardiac stem cells before and after they are transplanted into a patient. This discovery could significantly change the way stem cell therapy is administered in heart patients."

Earlier this year, a medical team at Cedars-Sinai Medical Center in Los Angeles reported initial success in reducing scar tissue in heart attack patients after harvesting some of the patient's own cardiac stem cells, growing more of these cells in a lab and transfusing them back into the patient. Using the stem cells from the patient's own heart prevented the rejection problems that often occur when tissue is transplanted from another person.

Levchenko's team has been trying to figure out what, at the molecular level, causes the stem cells to change into helpful heart tissue. If they could solve this mystery, the researchers hoped the cardiac stem cell technique used by the Los Angeles doctors could be altered to yield even better results.

During their research, the Johns Hopkins team members wondered whether changing the surface on which the harvested stem cells grew would affect the cells' development. The researchers were surprised to find that growing the cells on a surface whose rigidity resembled that of heart tissue caused the stem cells to grow faster and to form blood vessels. This cell population boom had occurred far less often in the stem cells grown in the glass or plastic dishes typically used in biology labs. This result also suggested why formation of cardiac scar tissue, a structure with very different rigidity, can inhibit stem cells naturally residing there from regenerating the heart.

Looking further into this stem cell differentiation, the Johns Hopkins researchers found that the increased cell growth occurred when there was a decrease in the presence of the protein p190RhoGAP. "It was the kind of master regulator of this process," Levchenko said. "And an even bigger surprise was that if we directly forced this molecule to disappear, we no longer needed the special heart-matched surfaces. When the master regulator was missing, the stem cells started to form blood vessels, even on glass."

A final surprise occurred when the team decided to increase the presence of p190RhoGAP, instead of making it disappear. "The stem cells started to turn into cardiac muscle tissue, instead of blood vessels," Levchenko said. "This told us that this amazing molecule was the master regulator not only of the blood vessel development, but that it also determined whether cardiac muscles and blood vessels would develop from the same cells, even though these types of tissue are quite different."

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Long-term pesticide exposure is harmful: STM study

By Dr. Matthew Watson

Kolkata, June 20 : Long-time exposure to pesticides via inhalation may cause moderate to severe blood toxicity and reduction in the total number of bone marrow cells, leading to several degenerative diseases like aplastic anaemia, researchers at the School of Tropical Medicine (STM) here say.

The researches arrived at the conclusion from procedures performed on mice.

"As a whole, exposure to pesticides reduced the total number of bone marrow cells or, in other words, suppressed them," Sujata Law, assistant professor (Stem Cell Biology) at STM's Department of Medical Biotechnology, told IANS.

Bone marrow is the soft, flexible tissue found in long bones such as the thigh bone and the hip bone that contain immature cells called stem cells.

Stem cells, particularly the haematopoeitic stem cells (HSC) or the blood-forming stem cells can develop into the following types - red blood cells that carry oxygen, white blood cells that fight infection and platelets that help to clot blood.

So, in effect, bone marrow is the birthplace of these important cells.

"Bone marrow suppression leads to a number of degenerative diseases like aplastic anaemia, where the deficiency in the number of cells in the circulating blood (peripheral cytopenia) is the main feature," Law said.

The exact underlying mechanism is unknown but it has been concluded from the research published in the Journal of Environmental Toxicology that the microenvironment of the stem cells, in which they develop, is somehow deranged and this prevents their development into the various types of cells.

"In order to prevent degenerative diseases related to pesticide exposure, it is of prime importance that those handling pesticides take precautions like wearing protective clothing, including masks and gloves," she said.

"Also pesticides should be stored in properly labelled containers, away from food, and kept out of reach of children and animals," Law said. (IANS)

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Understanding of spinal muscular atrophy improved with use of stem cells

By Dr. Matthew Watson

ScienceDaily (June 20, 2012) Cedars-Sinai's Regenerative Medicine Institute has pioneered research on how motor-neuron cell-death occurs in patients with spinal muscular atrophy, offering an important clue in identifying potential medicines to treat this leading genetic cause of death in infants and toddlers.

The study, published in the June 19 online issue of PLoS ONE, extends the institute's work to employ pluripotent stem cells to find a pharmaceutical treatment for spinal muscular atrophy or SMA, a genetic neuromuscular disease characterized by muscle atrophy and weakness.

"With this new understanding of how motor neurons die in spinal muscular atrophy patients, we are an important step closer to identifying drugs that may reverse or prevent that process," said Clive Svendsen, PhD, director of the Cedars-Sinai Regenerative Medicine Institute.

Svendsen and his team have investigated this disease for some time now. In 2009, Nature published a study by Svendsen and his colleagues detailing how skin cells taken from a patient with the disorder were used to generate neurons of the same genetic makeup and characteristics of those affected in the disorder; this created a "disease-in-a-dish" that could serve as a model for discovering new drugs.

As the disease is unique to humans, previous methods to employ this approach had been unreliable in predicting how it occurs in humans. In the research published in PLoS ONE, the team reproduced this model with skin cells from multiple patients, taking them back in time to a pluripotent stem cell state (iPS cells), and then driving them forward to study the diseased patient-specific motor neurons.

Children born with this disorder have a genetic mutation that doesn't allow their motor neurons to manufacture a critical protein necessary for them to survive. The study found these cells die through apoptosis -- the same form of cell death that occurs when the body eliminates old, unnecessary as well as unhealthy cells. As motor neuron cell death progresses, children with the disease experience increasing paralysis and eventually death. There is no effective treatment now for this disease. An estimated one in 35 to one in 60 people are carriers and about in 100,000 newborns have the condition.

"Now we are taking these motor neurons (from multiple children with the disease and in their pluripotent state) and screening compounds that can rescue these cells and create the protein necessary for them to survive," said Dhruv Sareen, director of Cedars-Sinai's Induced Pluripotent Stem Cell Core Facility and a primary author on the study. "This study is an important stepping stone to guide us toward the right kinds of compounds that we hope will be effective in the model -- and then be reproduced in clinical trials."

The study was funded in part by a $1.9 million Tools and Technology grant from the California Institute for Regenerative Medicine aimed at developing new tools and technologies to aid pharmaceutical discoveries for this disease.

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Cedars-Sinai researchers, with stem cells, advance understanding of spinal muscular atrophy

By daniellenierenberg

Public release date: 19-Jun-2012 [ | E-mail | Share ]

Contact: Nicole White nicole.white@cshs.org 310-423-5215 Cedars-Sinai Medical Center

LOS ANGELES (June 19, 2012) Cedars-Sinai's Regenerative Medicine Institute has pioneered research on how motor-neuron cell-death occurs in patients with spinal muscular atrophy, offering an important clue in identifying potential medicines to treat this leading genetic cause of death in infants and toddlers.

The study, published in the June 19 online issue of PLoS ONE, extends the institute's work to employ pluripotent stem cells to find a pharmaceutical treatment for spinal muscular atrophy or SMA, a genetic neuromuscular disease characterized by muscle atrophy and weakness.

"With this new understanding of how motor neurons die in spinal muscular atrophy patients, we are an important step closer to identifying drugs that may reverse or prevent that process," said Clive Svendsen, PhD, director of the Cedars-Sinai Regenerative Medicine Institute.

Svendsen and his team have investigated this disease for some time now. In 2009, Nature published a study by Svendsen and his colleagues detailing how skin cells taken from a patient with the disorder were used to generate neurons of the same genetic makeup and characteristics of those affected in the disorder; this created a "disease-in-a-dish" that could serve as a model for discovering new drugs.

As the disease is unique to humans, previous methods to employ this approach had been unreliable in predicting how it occurs in humans. In the research published in PLoS ONE, to the team reproduced this model with skin cells from multiple patients, taking them back in time to a pluripotent stem cell state (iPS cells), and then driving them forward to study the diseased patient-specific motor neurons.

Children born with this disorder have a genetic mutation that doesn't allow their motor neurons to manufacture a critical protein necessary for them to survive. The study found these cells die through apoptosis the same form of cell death that occurs when the body eliminates old, unnecessary as well as unhealthy cells. As motor neuron cell death progresses, children with the disease experience increasing paralysis and eventually death. There is no effective treatment now for this disease. An estimated one in 35 to one in 60 people are carriers and about in 100,000 newborns have the condition.

"Now we are taking these motor neurons (from multiple children with the disease and in their pluripotent state) and screening compounds that can rescue these cells and create the protein necessary for them to survive," said Dhruv Sareen, director of Cedars-Sinai's Induced Pluripotent Stem Cell Core Facility and a primary author on the study. "This study is an important stepping stone to guide us toward the right kinds of compounds that we hope will be effective in the model and then be reproduced in clinical trials."

###

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Researchers, with Stem Cells, Advance Understanding of Spinal Muscular Atrophy

By Sykes24Tracey

Newswise LOS ANGELES (June 19, 2012) Cedars-Sinais Regenerative Medicine Institute has pioneered research on how motor-neuron cell-death occurs in patients with spinal muscular atrophy, offering an important clue in identifying potential medicines to treat this leading genetic cause of death in infants and toddlers.

The study, published in the June 19 online issue of PLoS ONE, extends the institutes work to employ pluripotent stem cells to find a pharmaceutical treatment for spinal muscular atrophy or SMA, a genetic neuromuscular disease characterized by muscle atrophy and weakness.

With this new understanding of how motor neurons die in spinal muscular atrophy patients, we are an important step closer to identifying drugs that may reverse or prevent that process, said Clive Svendsen, PhD, director of the Cedars-Sinai Regenerative Medicine Institute.

Svendsen and his team have investigated this disease for some time now. In 2009, Nature published a study by Svendsen and his colleagues detailing how skin cells taken from a patient with the disorder were used to generate neurons of the same genetic makeup and characteristics of those affected in the disorder; this created a disease-in-a-dish that could serve as a model for discovering new drugs.

As the disease is unique to humans, previous methods to employ this approach had been unreliable in predicting how it occurs in humans. In the research published in PLoS ONE, to the team reproduced this model with skin cells from multiple patients, taking them back in time to a pluripotent stem cell state (iPS cells), and then driving them forward to study the diseased patient-specific motor neurons.

Children born with this disorder have a genetic mutation that doesnt allow their motor neurons to manufacture a critical protein necessary for them to survive. The study found these cells die through apoptosis the same form of cell death that occurs when the body eliminates old, unnecessary as well as unhealthy cells. As motor neuron cell death progresses, children with the disease experience increasing paralysis and eventually death. There is no effective treatment now for this disease. An estimated one in 35 to one in 60 people are carriers and about in 100,000 newborns have the condition.

Now we are taking these motor neurons (from multiple children with the disease and in their pluripotent state) and screening compounds that can rescue these cells and create the protein necessary for them to survive, said Dhruv Sareen, director of Cedars-Sinais Induced Pluripotent Stem Cell Core Facility and a primary author on the study. This study is an important stepping stone to guide us toward the right kinds of compounds that we hope will be effective in the model and then be reproduced in clinical trials.

The study was funded in part by a $1.9 million Tools and Technology grant from the California Institute for Regenerative Medicine aimed at developing new tools and technologies to aid pharmaceutical discoveries for this disease.

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‘Magical State' Of Embryonic Stem Cells May Help Overcome Hurdles To Therapeutics

By Dr. Matthew Watson

Salk researcher's findings suggest a potentially favorable time to harvest stem cells for therapy and may reveal genes crucial to tissue production

With their potential to treat a wide range of diseases and uncover fundamental processes that lead to those diseases, embryonic stem (ES) cells hold great promise for biomedical science. A number of hurdles, both scientific and non-scientific, however, have precluded scientists from reaching the holy grail of using these special cells to treat heart disease, diabetes, Alzheimer's and other diseases.

In a paper published June 13 in Nature, scientists at the Salk Institute for Biological Studies report discovering that ES cells cycle in and out of a "magical state" in the early stages of embryo development, during which a battery of genes essential for cell potency (the ability of a generic cell to differentiate, or develop, into a cell with specialized functions) is activated. This unique condition, called totipotency, gives ES cells their unique ability to turn into any cell type in the body, thus making them attractive therapeutic targets.

"These findings," says senior authorSamuel L. Pfaff, a professor in Salk'sGene Expression Laboratory, "give new insight into the network of genes important to the developmental potential of cells. We've identified a mechanism that resets embryonic stem cells to a more youthful state, where they are more plastic and therefore potentially more useful in therapeutics against disease, injury and aging."

ES cells are like silly putty that can be induced, under the right circumstances, to become specialized cells-for example, skin cells or pancreatic cells-in the body. In the initial stages of development, when an embryo contains as few as five to eight cells, the stem cells are totipotent and can develop into any cell type. After three to five days, the embryo develops into a ball of cells called a blastocyst. At this stage, the stem cells are pluripotent, meaning they can develop into almost any cell type. In order for cells to differentiate, specific genes within the cells must be turned on.

Pfaff and his colleagues performed RNA sequencing (a new technology derived from genome-sequencing to monitor what genes are active) on immature mouse egg cells, called oocytes, and two-cell-stage embryos to identify genes that are turned on just prior to and immediately following fertilization. Pfaff's team discovered a sequence of genes tied to this privileged state of totipotency and noticed that the genes were activated by retroviruses adjacent to the stem cells.

Nearly 8 percent of the human genome is made up of ancient relics of viral infections that occurred in our ancestors, which have been passed from generation to generation but are unable to produce infections. Pfaff and his collaborators found that cells have used some of these viruses as a tool to regulate the on-off switches for their own genes. "Evolution has said, 'We'll make lemonade out of lemons, and use these viruses to our advantage,'" Pfaff says. Using the remains of ancient viruses to turn on hundreds of genes at a specific moment of time in early embryo development gives cells the ability to turn into any type of tissue in the body.

From their observations, the Salk scientists say these viruses are very tightly controlled-they don't know why-and active only during a short window during embryonic development. The researchers identified ES cells in early embryogenesis and then further developed the embryos and cultured them in a laboratory dish. They found that a rare group of special ES cells activated the viral genes, distinguishing them from other ES cells in the dish. By using the retroviruses to their advantage, Pfaff says, these rare cells reverted to a more plastic, youthful state and thus had greater developmental potential.

Pfaff's team also discovered that nearly all ES cells cycle in and out of this privileged form, a feature of ES cells that has been underappreciated by the scientific community, says first author Todd S. Macfarlan, a former postdoctoral researcher in Pfaff's lab who recently accepted a faculty position at the Eunice Kennedy Shriver National Institute of Child Health and Human Development. "If this cycle is prevented from happening," he says, "the full range of cell potential seems to be limited."

It is too early to tell if this "magical state" is an opportune time to harvest ES cells for therapeutic purposes. But, Pfaff adds, by forcing cells into this privileged status, scientists might be able to identify genes to assist in expanding the types of tissue that can be produced.

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LIFE Focuses on Stem Cell Research – Analyst Blog

By LizaAVILA

Referenced Stocks: ILMN, LIFE, TMO

Given the recent flurry of activities, it seems that Life Technologies Corporation ( LIFE ) is focused on strengthening its foothold in the field of stem cell research. The company recently signed a non-exclusive agreement with iPS Academia of Japan for its induced pluripotent stem (iPS) cell patent portfolio. Based on this agreement, the company will be able to expand its portfolio for the iPS cell research community.

Besides, it is well placed to create iPS cells and differentiate them into various cell types to be used in drug discovery and pre-clinical research. The license also enables Life Technologies to provide creation, differentiation and screening services of iPS cell to scientists globally. We consider the agreement to be a significant achievement for the company in the field of stem cell research as iPS cells are gaining attention for use in the areas of drug discovery, disease research and other areas of biotechnology.

The agreement with iPS Academia of Japan comes on the heels of the partnership with Cellular Dynamics International, the world's largest producer of human cells derived from iPS cells. The partnership will aim at commercializing a set of three new products optimized to consistently develop and grow human iPS cells for both research and bioproduction.

These initiatives undertaken by Life Technologies should strengthen its Research Consumables segment. This segment includes molecular and cell biology reagents, endpoint PCR and other benchtop instruments and consumables. These products include RNAi, DNA synthesis, sample prep, transfection, cloning and protein expression profiling and protein analysis, cell culture media used in research, stem cells and related tools, cellular imaging products, antibodies and cell therapy related products. In the most recent quarter, this division recorded a 4% year-over-year increase in revenues to $420 million on the back of growth in cell culture workflow products, endpoint PCR products and molecular and cell biology consumables.

Life Technologies enjoys a strong position in the life sciences market, though management prefers to maintain a cautious but optimistic outlook for the remainder of the year. We are encouraged by the improvement in margins amidst the tight competitive scenario with the presence of players such as Thermo Fisher Scientific ( TMO ), Illumina ( ILMN ), among others.

We have a Neutral recommendation on Life Technologies. The stock retains a Zacks #3 Rank (hold) in the short term.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of The NASDAQ OMX Group, Inc.

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CBR – World's Largest Stem Cell Bank – Applies Two Decades of Experience to Advance Regenerative Medicine

By Dr. Matthew Watson

SAN BRUNO, Calif., June 20, 2012 /PRNewswire/ -- Twenty years ago this month, CBR (Cord Blood Registry) in partnership with the University of Arizona, processed the first cord blood stem cell sample in the world to be stored specifically for family use. Since 1992, the number of conditions treated with cord blood stem cells has greatly expanded, and so has CBR. Today, CBR is the largest family cord blood bank in the world with more than 425,000 samples in storage a population the size of a major city like Miami. What distinguishes the "city of individuals" with newborn stem cells banked at CBR is the exclusive opportunity to participate in a growing number of ground-breaking clinical trials.

(Photo: http://photos.prnewswire.com/prnh/20120620/SF27549-INFO)

(Logo: http://photos.prnewswire.com/prnh/20120216/AQ54476LOGO)

"As the leader and innovator in family banking, we believe every newborn deserves a healthy future and that we have a responsibility to lead the way," said Heather Brown, vice president of scientific & medical affairs at CBR. "Looking back, the creation of our bank allowed families for the first time to preserve a genetically-related source of newborn stem cells, ready and available if needed for a lifesaving transplant to regenerate a person's immune system after radiation or chemotherapy. As we look to the future, we are helping shape new areas of regenerative medicine. We are the only family bank actively pioneering clinical trials evaluating new therapeutic uses of cord blood stem cells for unexpected injuries and conditions with no current cure."

Expanding Areas of Clinical Research: Helping the Body Heal Injured Nerves Until very recently, the prevailing medical opinion in neurology has been that damage to the central nervous system caused by injuries like birth trauma, accidents or stroke is often permanent. Currently, intervention after injury focuses on stabilizing the patient to minimize damage. However, data from animal research in recent years has challenged this assumption, leading to cord blood stem cell clinical research to study whether these cells may stimulate neural cell and tissue repair to restore function and alleviate neurological impairments.

CBR is taking the lead in moving animal research rapidly into the clinic to investigate the ability for cord blood stem cells to trigger the body's own mechanisms to initiate nerve repair by establishing specific clinical trials at leading medical institutions across the country. By pairing researchers with children who have been diagnosed with chronic conditions like cerebral palsy, traumatic brain injury or hearing loss-- and who also have access to their own cord blood stem cells -- CBR is helping physicians move beyond surgery and drugs to evaluate how newborn stem cells may help the body repair itself.

Celebrating a History of Firsts Throughout its history, CBR has taken many of the first steps to create and advance the notion of preserving and ensuring access to high quality newborn stem cells that are viable for use. Among the company's contributions to stem cell medicine and science, CBR was:

"CBR continuously improves our systems and technology to maintain the highest published cell recovery rate in the industry of 99%, every single time. We treat every sample as if it belongs to our own child or grandchild," says Tom Moore, CEO and founder of CBR. "That care and precision is what we offer clinical researchers, who are partnering exclusively with CBR to evaluate the use of a child's own cord blood stem cells to help treat chronic diseases like cerebral palsy, hearing loss and traumatic brain injury."

About Cord Blood RegistryCBR (Cord Blood Registry) is the world's largest and most experienced cord blood bank.The company has consistently led the industry in technical innovations and safeguards more than 425,000 cord blood collections for individuals and their families. CBR was the first family bank accredited by AABB and the company's quality standards have been recognized through ISO 9001:2008 certificationthe global business standard for quality. CBR has also released more client cord blood units for specific therapeutic use than any other family cord blood bank. Our research and development efforts are focused on helping the world's leading clinical researchers advance regenerative medical therapies.For more information, visit http://www.cordblood.com.

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CBR - World's Largest Stem Cell Bank - Applies Two Decades of Experience to Advance Regenerative Medicine

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LIFE Focuses on Stem Cell Research

By NEVAGiles23

Given the recent flurry of activities, it seems that Life Technologies Corporation (LIFE) is focused on strengthening its foothold in the field of stem cell research. The company recently signed a non-exclusive agreement with iPS Academia of Japan for its induced pluripotent stem (iPS) cell patent portfolio. Based on this agreement, the company will be able to expand its portfolio for the iPS cell research community.

Besides, it is well placed to create iPS cells and differentiate them into various cell types to be used in drug discovery and pre-clinical research. The license also enables Life Technologies to provide creation, differentiation and screening services of iPS cell to scientists globally. We consider the agreement to be a significant achievement for the company in the field of stem cell research as iPS cells are gaining attention for use in the areas of drug discovery, disease research and other areas of biotechnology.

The agreement with iPS Academia of Japan comes on the heels of the partnership with Cellular Dynamics International, the world's largest producer of human cells derived from iPS cells. The partnership will aim at commercializing a set of three new products optimized to consistently develop and grow human iPS cells for both research and bioproduction.

These initiatives undertaken by Life Technologies should strengthen its Research Consumables segment. This segment includes molecular and cell biology reagents, endpoint PCR and other benchtop instruments and consumables. These products include RNAi, DNA synthesis, sample prep, transfection, cloning and protein expression profiling and protein analysis, cell culture media used in research, stem cells and related tools, cellular imaging products, antibodies and cell therapy related products. In the most recent quarter, this division recorded a 4% year-over-year increase in revenues to $420 million on the back of growth in cell culture workflow products, endpoint PCR products and molecular and cell biology consumables.

Life Technologies enjoys a strong position in the life sciences market, though management prefers to maintain a cautious but optimistic outlook for the remainder of the year. We are encouraged by the improvement in margins amidst the tight competitive scenario with the presence of players such as Thermo Fisher Scientific (TMO), Illumina (ILMN), among others.

We have a Neutral recommendation on Life Technologies. The stock retains a Zacks #3 Rank (hold) in the short term.

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Neuralstem Pioneering Efforts In ALS – Analyst Blog

By daniellenierenberg

By Jason Napodano, CFA

Neuralstem, Inc. (NYSE MKT: CUR ) has developed a technology that allows large-scale expansion of human neural stem cells ("hNSC") from all areas of the developing human brain and spinal cord. The company owns of has exclusive license to 25 patients and 29 patent applications pending worldwide in the field of regenerative medicine and cell therapy. Management is currently focusing the company's efforts on replacing damaged, malfunctioning, or dead neural cells with fully functional ones that may be useful in treating many central nervous system diseases and neurodegenerative disorders.

Neuralstem's lead development program is for Amyotrophic Lateral Sclerosis ("ALS"), also known as Lou Gehrig 's disease, named after the famous New York Yankee first baseman who was diagnosed with the disease in 1939, and passed in 1941 at the age of only 37.

ALS Background

ALS is a rapidly progressive neurodegenerative disease characterized by weakness, muscle atrophy and twitching, spasticity, dysarthria (difficulty speaking), dysphagia (difficulty swallowing), and respiratory compromise. The disease is almost always fatal, typically due to respiratory compromise or pneumonia, in two to four years. Initial symptoms of ALS include weakness and/or stiffness followed by muscle atrophy in the arms and legs. This is followed by slurred speech or difficulty swallowing, and loss of tongue mobility. Approximately a third of ALS patients also experience pseudobulbar affect (uncontrollable emotions). As the disease progresses, worsening dysphagia and respiratory failure leads to death. A small percentage of patients may also experience cognitive affects such as frontotemporal dementia and anxiety.

The vast majority (~95%) of cases are idiopathic, although there is a known hereditary factor that leads to familial ALS associated with a defect on the 21st chromosome that accounts for approximately 1.5% of all cases. There are also suspected environmental causative factors, including exposure to a dietary neurotoxin called BMAA and cyanobacteria, and use of pesticides. However, in all cases, the defining factor of ALS is rapid and progressive death of upper and lower motor neurons in the motor cortex of the brain, brain stem, and spinal cord. Prior to their destruction, motor neurons develop proteinaceous inclusions in their cell bodies and axons. This may be partly due to defects in protein degradation.

Treatment for ALS is limited, and as of today only riluzole, marketed by Sanofi-Aventis as Rilutek, has been found to improve survival to a modest extent (several months). Riluzole preferentially blocks TTX-sensitive sodium channels, which are associated with damaged neurons. This reduces influx of calcium ions and indirectly prevents stimulation of glutamate receptors. Together with direct glutamate receptor blockade, the effect of the neurotransmitter glutamate on motor neurons is greatly reduced. Riluzole does not reverse the damage already done to motor neurons, and people taking it must be monitored for liver damaged (about 10% incidence).

The remaining treatments for ALS are designed to relieve symptoms and improve quality of life. This supportive care includes a multidisciplinary approach that may include medications to reduce fatigue, control spasticity, reduce excess saliva and phlegm, limit sleep disturbances, reduce depression, and limit constipation. As noted above, median survival is two to four years. In the U.S., approximately 30,000 persons are currently living with ALS.

Neuralstem's Approach For ALS

Neuralstem is seeking to treat the symptoms of ALS via transplantation of its hNSCs directly into the gray matter of the patient's spinal cord. In ALS, motor neurons die, leading to paralysis. In preclinical animal work, Neuralstem cells both made synaptic contact with the host motor neurons and expressed neurotrophic growth factors, which are protective of cells.

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