Category Archives: Diseases

Newswise UCLA stem cell researchers have found for the first time a surprising and unexpected plasticity in the embryonic endothelium, the place where blood stem cells are made in early development.

Scientists found that the lack of one transcription factor, a type of gene that controls cell fate by regulating other genes, allows the precursors that normally generate blood stem and progenitor cells in blood forming tissues to become something very unexpected - beating cardiomyocytes, or heart muscle cells.

The finding is important because it suggests that the endothelium can serve as a source of heart muscle cells. The finding may provide new understanding of how to make cardiac stem cells for use in regenerative medicine, said study senior author Dr. Hanna Mikkola, an associate professor of molecular, cell and developmental biology in Life Sciences and a researcher with the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.

It was absolutely unbelievable. These findings went beyond anything that we could have imagined, Mikkola said. The microenvironment in the embryonic vasculature that normally gives rise to blood cells can generate cardiac cells when only one factor, Scl, is removed, essentially converting a hematopoietic organ into a cardiogenic organ.

The two-year study is published Aug. 3, 2012 in the peer-reviewed journal Cell.

The findings were so surprising, in fact, that Mikkola and her team did not want to believe the results until all subsequent assays proved the finding to be true, said Amelie Montel-Hagen, study co-first author and a post-doctoral fellow.

To make sure we had not switched the samples between blood forming tissues and the heart we ran the experiments again and repeatedly got the same results, Montel-Hagen said. It turns out Scl acts as a conductor in the orchestra, telling the other genes in the endothelium who should be playing and who shouldnt be playing.

The team used microarray technology to determine which genes were playing in embryonic endothelium to generate blood stem and progenitor cells and found that in the absence of Scl, the genes required for making cardiomyocytes were activated instead, said study co-first author Ben Van Handel, a post-doctoral fellow.

The lone difference was that Scl was missing in the process that resulted in the fate switch between blood and heart.

Scl has a known role as a master regulator of blood development and when we removed it from the equation, no blood cells were made, Van Handel said. That the removal of Scl resulted in fully functional cardiomyocytes in blood forming tissues was unprecedented.

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Embryonic Blood Vessels that Make Blood Stem Cells can also Become Beating Heart Muscle Cells

NEWARK, Calif., Aug. 2, 2012 (GLOBE NEWSWIRE) -- StemCells, Inc. (STEM), a leading stem cell company developing and commercializing novel cell-based therapeutics and tools for use in stem cell-based research and drug discovery, today reported financial results for the second quarter ended June 30, 2012 and provided a business update.

"We continue to be encouraged by our progress in developing cell-based therapeutics for a broad array of disorders affecting the central nervous system," said Martin McGlynn, President and CEO of StemCells, Inc. "We have already reported top line results from our Phase I PMD trial and I am pleased to report that the manuscript with the complete PMD trial data is under peer review for publication by a top tier journal.

Our other clinical development efforts also continue to advance. We recently reported interim safety data from our chronic spinal cord injury trial, showing that our cells and the procedure have been well tolerated. We have also initiated a Phase I/II trial in dry AMD and look forward to enrolling our first patients in this study soon. Lastly, we recently reported preclinical data showing that our cells restored memory in two animal models relevant to Alzheimer's disease without having to reduce beta-amyloid or tau burden that are the pathological hallmarks of the disease. Results of this kind underscore the potential of our HuCNS-SC cells to potentially address a number of devastating CNS disorders.

Financially, we aim to do more with less and continue to carefully manage our burn rate. Last week's decision by CIRM to award us a $20 million disease team award is exciting and welcome. This award will not only provide additional resources, but is also a vote of confidence in our technology, our program and our people. Moving forward, we will continue to generate clinical data from our HuCNS-SC program in a thoughtful, cost effective manner, which is, we believe, the best pathway to grow shareholder value."

Second Quarter and Recent Business Highlights

Second Quarter Financial Results

Revenue from product sales increased 14% to $211,000 in the second quarter of 2012 compared to the same period of 2011 as our SC Proven media and reagents business continued to see increased unit volume. Total revenue in the second quarter of 2012 was $249,000, compared to $234,000 in the same period of 2011.

Our operating expenses decreased 24% to $5,535,000 in the second quarter of 2012 compared to the same quarter of 2011. Research and development expenses were 26% lower, and selling, general and administrative expenses were 16% lower, compared to the second quarter of 2011. The significant reduction in operating expenses was primarily attributable to continuing tight cost controls and a number of measures taken last year to reduce infrastructure and overhead costs, including a reduction in workforce implemented in May 2011.

Other income in the second quarter of 2012 was $6,184,000, compared to $3,055,000 in the second quarter of 2011. This increase was primarily due to a decrease in the estimated fair value of warrant liability. Our outstanding warrants are classified as a liability, with subsequent changes in the estimated fair value recorded as income or loss.

Loss from operations in the second quarter of 2012 was $5,350,000, a 25% decrease compared to the same period in 2011. Net income for the quarter was $834,000, or $0.03 per share, compared with a net loss of $4,035,000, or $(0.29) per share, for the second quarter of 2011.

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Stemcells, Inc. Reports Second Quarter 2012 Financial Results and Provides Business Update

MARLBOROUGH, Mass.--(BUSINESS WIRE)--

Advanced Cell Technology, Inc. (ACT; OTCBB: ACTC), a leader in the field of regenerative medicine, today announced treatment of the fourth patient, the first in the second patient cohort, in the companys Phase I/II clinical trial for dry age-related macular degeneration (dry AMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). The surgery was performed on Wednesday, Aug. 1 atWills Eye Institutein Philadelphia, by a surgical team lead by Carl D. Regillo, M.D., Chief of the Wills Eye Institute Retina Service, and professor of ophthalmology at Thomas Jefferson University. The patient was injected with 100,000 hESC-derived RPE cells and is recovering uneventfully.

We are very pleased to have the second dose cohort in both of our U.S. clinical trials underway, commented Gary Rabin, chairman and CEO of ACT. We are encouraged by our ongoing progress in all three of our clinical trials using our hESC-derived RPE cells to treat forms of macular degeneration. We have not observed any complications or side effects from the stem cell-derived RPE cells, and we will continue to monitor the patients for safety, tolerability and efficacy of this therapy.

The dry AMD trial is one of three clinical trials being carried out by the company in the U.S. and in Europe. Each trial will enroll 12 patients, with cohorts of three patients in an ascending dosage format. These trials are prospective, open-label studies, designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation into patients with dry AMD or Stargardt's macular dystrophy (SMD) at 12 months, the studys primary endpoint. Preliminary results from the two U.S. trials were reported in The Lancet earlier this year.

Doubling the cell dosage in both our U.S. trials is an important step forward in our clinical programs, said Robert Lanza, M.D., ACTs chief scientific officer. We anticipate continued progress and safety findings in both our U.S. trials as well as our concurrent European trial.

Dry AMD is the most common cause of vision loss in patients over 50 years and affects around 30 million people worldwide, said Dr. Regillo. Dry AMD is a form of macular degeneration with no approved drug treatment available to date. ACTs hESC-derived RPE cells could address the unmet medical need of combating dry AMD and other macular degenerations such as Stargardts disease. We are looking forward to analyzing the safety and efficacy data.

Further information about patient eligibility for ACTs dry AMD study and the concurrent studies in the U.S. and the E.U. for SMD is available atwww.clinicaltrials.gov,with the following Identifiers: NCT01344993 (dry AMD), NCT01345006 (U.S. SMD), and NCT01469832 (E.U. SMD).

About dry AMD

Degenerative diseases of the retina are among the most common causes of untreatable blindness in the world. Age-related macular degeneration (AMD) is the leading cause of blindness in people over age 60 in the United States, and the vast majority of cases of AMD are of the dry form, which is currently untreatable.

About Advanced Cell Technology, Inc.

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ACT Announces First Dry AMD Patient Treated with Higher Dosage of Embryonic Stem Cell-Derived RPE Cells

Editor's Choice Main Category: Stem Cell Research Article Date: 03 Aug 2012 - 14:00 PDT

Current ratings for: Stem Cell Therapy May Fix Defects From Injuries To Head And Mouth

4 (1 votes)

The clinical trial was a collaboration of researchers from the University of Michigan School of Dentistry and the Michigan Center for Oral Health Research together with Ann Arbor-based Aastrom Biosciences Inc. involving 24 patients who required jawbone reconstruction after tooth removal. The researchers divided the patients into two groups, with one group receiving experimental tissue repair cells (ixmyelocel-T) and the other group receiving traditional guided bone regeneration therapy. The tissue repair cells ixmyelocel-T are currently being development at Aastrom.

Leading investigator Darnell Kaigler, who is assistant professor at the U-M School of Dentistry said:

Kaigler stated that the treatment is best suited for large defects, like those resulting from trauma, diseases or birth defects, since these are very challenging to treat due to their complex nature of requiring various different tissue types, including bone, skin and gum tissue.

He continued saying that the key advantage of using stem cell therapy is that the patient's own cells are used to regenerate tissue instead of using man-made, foreign materials.

The study achieved promising results. Study participants in the cell therapy group received dental implants at 6 and 12 weeks after their experimental cell therapy and were noted to have a greater bone density and quicker bone repair compared with those who underwent traditional guided bone regeneration therapy. They also needed less secondary bone grafting when receiving their implants compared with the traditional bone regeneration group.

The team used cells extracted from the patient's hip bone marrow, which was subsequently processed using Aastrom's proprietary system. This allows the growths of many different cells, including stem cells, which were then relocated into different areas in the patient's mouth and jaw.

Kaigler concluded saying that stem cell therapies are still probably 5-10 years away from becoming a standard treatment for oral and facial injuries and defects and that more clinical trials need to be conducted, which include a larger number of patients with larger craniofacial defects.

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Stem Cell Therapy May Fix Defects From Injuries To Head And Mouth

American Cancer Society/Getty Images

File photo: Close up of cancer cells in the cervix. CIRM awarded UCLA $20 million for the university's study on the reprogramming of cancer cells.

This week, Californias public stem-cell agency awarded more than $150 million to advance research into eight diseases now under study by researchers at several California universities, one L.A. hospital and a biotech company.

The University of California Davis received the largest share of stem cell research grants from the California Institute of Regenerative Medicine.

The voter-approved agency, also known as CIRM, awarded nearly $50 million to three UC Davis research teams studying treatments for the bone disease osteoperosis, for the genetic brain disorder known as Huntingtons Disease and for Critical Limb Ischemia, a painful condition caused by severe blockage of arteries.

CIRM gave $20 million to a UCLA study on the reprogramming of cancer cells. And the agency awarded UC-Irvine and the biotech firm, StemCells, Inc. of Newark, California $20 million for their joint study into treatments for cervical spinal injuries.

Cedar Sinai Medical Center also made the list with an $18 million grant to speed their research into possible treatments for Lou Gehrigs disease, a neuromuscular condition that leads to paralysis and death.

And Stanford University won two awards of $20 million each to study cardiovascular disease at metastatic melanoma.

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CIRM awards $151 million in stem cell grants to 8 projects statewide

Imagine a man with a recent severe heart attack who has the muscle repaired with stem cells or a child with a severe bladder defect repaired with stem cells grown on a biodegradable scaffold. Sounds like science fiction but these are actual clinical studies in progress today. Stem cell therapies promise to be one of those scientific breakthroughs that will have an enormous impact on health care in the future. Stem cells will bring us closer to the goal of personalized medicine, just as genomics is doing. The course of a disease will change once we have the technology to develop and then insert stem cells into the human body to actually create a tissue. For example, a person with a heart attack will not go on to live the rest of his or her life with damaged heart muscle and resultant heart failure. Instead, stem cells will repopulate the heart muscle and make it whole again. Similarly, a person with Parkinsons disease will recover full faculties thanks to the ability of stem cells to regenerate the damaged area of the brain. The person with type I diabetes will be free of the disease because of the formation of new pancreatic islet cells. The athlete will play again because new cartilage will be created for the worn knee. This is the promise of regenerative medicine. I have written the above as though each will definitely happen, a promise that will be kept. They probably will, but it may be a long time before the science of stem cells is sufficiently developed that these types of incredible results will be commonplace. Adult stem cells are being used today for treatment of a few diseases and there are studies ongoing and planned for many additional possibilities. Lets consider a few of them. Each of our tissues has a population of cells that can divide as needed to keep the organ or tissue functional as cells die or are injured. We see this with our skin as it constantly lays down new cells which make their way to the surface as the dead cells on the surface are rubbed off in the shower. We also see it when we cut ourselves and yet in a few days the wound is completely healed that was stem cells at work. It appears that essentially every organ has its own pool of such cells. There are cells in the bone marrow that can become stem cells for many different tissues. These cells circulate in the blood and can be called to assist a tissue or organ to rebuild itself after injury or damage. So for example, if a surgeon takes one half of a fathers liver for transplantation into his son, we know that the fathers liver will grow back to normal size within about 6 to 8 weeks. Some of the stem cells will have been those already in the liver but some will have come from the blood stream to assist. Of course, the liver is the exception to the rule that if a portion of an organ is removed by trauma or surgery, it will not grow back. Cut off your finger and stem cells will help it to heal but not to grow back to its original state. Adult stem cells are the ones used for treating leukemia, myeloma and other cancers and for correcting certain childhood immune deficiencies. Most often is the use of allogeneic hematopoietic stem cell transplantation, meaning the use of stem cells obtained from a closely matched individual. An identical twin is ideal but few have such a potential donor. Only 25% of siblings will likely match completely. This leaves the use of the National Marrow Donor Registry to find as close a match as possible from unrelated individuals. The Registry has markedly improved the chances for a close match and thus for successful transplantation outcomes. Many parents are now having umbilical cord blood saved and frozen to have available in the unlikely event that their child requires a transplant many years later. Although these cells are identical they usually are not sufficient in numbers to lead to engraftment and often the white blood cells (neutrophils) recover only very slowly leaving a prolonged period of infection risk. Perhaps a technique will be found to get the umbilical stem cells to multiply in the laboratory so that a larger number would be available. Adult stem cells are being used in studies of myocardial infarction and heart failure. Current guidelines of immediate angioplasty and stent insertion as appropriate help protect the heart from permanent damage after an infarct. Still, about 400,000 new cases of heart failure are developing in the USA each year. Long term survival is limited once overt failure develops. Could the damaged heart muscle be fixed? The concept is to use stem cells to repopulate the muscle fibers and to have those cells divide over and over and differentiate into new muscle fibers or perhaps also the small vessels that carry blood to the muscle cells. So far there are some exciting animal studies and even some trials in patients that are encouraging enough to warrant further evaluations. For example, one study uses adult mesenchymal stem cells derived from the bone marrow and infused intravenously within 7 days after a heart attack. 42 centers are collaborating in this double blind, randomized trail in conjunction with Osiris Therapeutics. 220 patients will receive either the stem cells or a placebo and then be monitored with various imaging and functional studies. So, stay tuned. Another common albeit less lethal problem is loss of bladder control leading to incontinence. There are studies in progress to determine if stem cells placed into the bladders sphincter muscle will help it regain control. The adult stem cells are obtained from a leg muscle biopsy. Stem cells are isolated and allowed to grow in tissue culture. These are then injected into the weakened bladder sphincter muscle. Once again, these are studies just beginning but with intriguing early results. Here is another bladder repair concept. When the bladder muscle is weak or largely missing in children it may be possible to literally rebuild the bladder by tissue engineering. A biopsy of the bladder yields cells that can be grown in the laboratory to large numbers. They can then be placed on a biodegradable scaffold and grown further. In time they seem to create a new bladder muscle wall complete with blood vessels. This layer of cells can be implanted in the bladder of children with a defect. Once more I need to note that it is still early days in these studies but they do raise exciting possibilities. The message here is that adult stem cells are being used today for life threatening and life impairing diseases with excellent success and are being studied in other diseases with exciting prospects for the future.

Stephen C Schimpff, MD is an internist, professor of medicine and public policy, former CEO of the University of Maryland Medical Center and is chair of the advisory committee for Sanovas, Inc. and senior advisor to Sage Growth Partners. He is the author of The Future of Medicine Megatrends in Healthcare and The Future of Health Care Delivery- Why It Must Change and How It Will Affect You from which this post is partially adapted. Updates are available at http://medicalmegatrends.blogspot.com

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Medical Megatrends – Stem Cells – Part II of III