NYSCF and the CMTA enter collaboration to advance neuropathies research

By adminDecember 11, 2014Stem Cell Medical Center

PUBLIC RELEASE DATE:

10-Dec-2014

Contact: David McKeon dmckeon@nyscf.org 212-365-7440 New York Stem Cell Foundation @nyscf

New York, NY (December 10, 2014) - The New York Stem Cell Foundation (NYSCF) Research Institute, a non-profit organization dedicated to accelerating cures through stem cell research, announced a collaboration today with the Charcot-Marie-Tooth Association (CMTA), a patient-led disease foundation with the mission to advance research on genetic neuropathies that leads to the development of new therapies. The immediate aim of the collaboration is to develop a bank of induced pluripotent stem cell (iPSC) lines for a variety of neuropathy disorders of known genetic causation and to eventually develop personalized drug therapies.

NYSCF will make stem cells lines from Charcot-Marie-Tooth patient materials that have been curated in a biobank assembled by Dr. Michael Shy at the University of Iowa, a member of the CMTA STAR consortium of sponsored investigators. Utilizing its automated technology, the NYSCF Global Stem Cell ArrayTM, NYSCF will systematically generate iPSC lines from tissue samples obtained from patients representing a number of disease states. These cell lines will then be used to develop methods for creating differentiated cells that mimic the myelin-producing Schwann cells that are defective in Type 1 Charcot-Marie-Tooth (CMT) disorders of peripheral nerve, as well as the motor and sensory neurons that are defective in Type 2 disorders. Members of the STAR consortium currently engaged in this CMTA-sponsored effort to differentiate iPSC lines include Dr. Robert Baloh, Cedar-Sinai Medical Center, and Dr. Gabsang Lee, Johns Hopkins University. The ultimate aim of this research is to create a personalized medicine approach to rapid testing of human drug responsiveness in a dish. The iPSC lines will also be expanded and banked by NYSCF and made available to the global scientific community to be used for research and the development of therapies.

Patrick Livney, CEO of the CMTA notes: "The Foundation has assembled the scientific and clinical key opinion leaders in CMT disorders, and the research tools necessary to validate therapeutic opportunities for their clinical potential. We have set out to engage drug makers to work together with the CMTA to advance new therapeutic approaches to our patients, and our STAR network that combines this world class research expertise with an operational capability has been highly enabling to the formation of collaborative alliances for this purpose. Currently, there are no therapies for the different CMT disorders to halt either the onset or progression of the disease. This NYSCF collaboration represents an exciting opportunity for the CMTA to place research on therapies for Charcot-Marie-Tooth disorders in a personalized, patient context at a very early stage.

"We are very exctied to partner with the Charcot-Marie-Tooth Association to develop resources that will enable the pursuit of new treatments and eventually cures for neruropathy disorders," said Susan L. Solomon, Co-Founder and CEO of NYSCF. "Partnering with CMTA provides us with the necessary community of scientists, patients, disease experts, as well as resources that allows us to move research forward. We believe that this type of interdisciplinary collaboration between various stakeholders is essential to to move research forward in the pursuit of cures."

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About Charcot-Marie-Tooth Association

The Charcot-Marie-Tooth Association (CMTA) is a registered 501c3 dedicated to serving an international patient community that suffers from rare and disabling neuropathies of genetic origin. The Foundation directly engages its STAR scientific and clinical research network in the identification, validation and clinical development of therapies for the different Charcot-Marie-Tooth disorders.

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NYSCF and the CMTA enter collaboration to advance neuropathies research

Microsoft billionaire takes on cell biology

By adminDecember 9, 2014Stem Cell Medical Center

Allen Institute

Paul Allens latest philanthropic endeavour will be modelled on his successful brain institute.

Billionaire businessman and philanthropist Paul Allen plans to pump US$100million into investigating the most basic unit of life the cell.

The Allen Institute for Cell Science, which was launched on 8 December, will be modelled on the Microsoft co-founders Allen Institute for Brain Science in Seattle, Washington, which since 2003 has spent hundreds of millions of dollars creating a series of brain atlases that have become go-to portals for neuroscientists interested in where particular genes are active or how distant neurons communicate.

As its first project, the latest Allen institute will develop an analogous cell observatory that will display how a cells working parts, such as ribosomes, microtubules and mitochondria, interact and operate over time, says executive director Rick Horwitz. He has shuttered his cell-biology laboratory at the University of Virginia in Charlottesville to lead the institute in Seattle, Washington. The 70 or so scientific staff who will join the institute will work together on the overall goals of the observatory to build a global view of the myriad activities inside cells rather than on their own interests. Its going to be much more like the Manhattan Project, Horwitz says.

Allen Institute

Rick Horwitz shut down his lab at the University of Virginia to lead the Allen Institute for Cell Science.

Mapping every little detail of every kind of cell is a tall order, even with the backing of the worlds 27th richest person. Our problem is that this thing could blow up on us. It could be very, very big, Horwitz says. Were going to make judicious decisions to try to contain it.

Some of those choices have already been made, after meetings this year with leading cell biologists. The institute will study human induced pluripotent stem cells (cells coaxed into an embryonic stem-cell-like state) as they differentiate in the lab into two cell types: heart-muscle cells called cardiomyocytes; and the epithelial cells that line body cavities. These tissues were chosen as much for their relevance to disease cardiomyocytes malfunction in heart disease and most cancers arise in epithelial tissues as for the ease with which they can be reproducibly generated and grown in the lab.

The institutes plan is to engineer many different cell lines and determine how different cellular components respond to stimuli such as infection or exposure to a drug. These data will then guide the construction of computer models aimed at predicting how cells operate under various conditions, and all the information gained will be made available online. The institute will also distribute its cell lines so that other scientists can build on its work.

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Microsoft billionaire takes on cell biology

New single-cell analysis reveals complex variations in stem cells

By adminDecember 4, 2014Stem Cell Medical Center

PUBLIC RELEASE DATE:

4-Dec-2014

Contact: Kat J. McAlpine katherine.mcalpine@wyss.harvard.edu 617-432-8266 Wyss Institute for Biologically Inspired Engineering at Harvard @wyssinstitute

(BOSTON) -- Stem cells offer great potential in biomedical engineering due to their pluripotency, which is the ability to multiply indefinitely and also to differentiate and develop into any kind of the hundreds of different cells and bodily tissues. But the precise complexity of how stem cell development is regulated throughout states of cellular change has been difficult to pinpoint until now.

By using powerful new single-cell genetic profiling techniques, scientists at the Wyss Institute for Biologically Inspired Engineering and Boston Children's Hospital have uncovered far more variation in pluripotent stem cells than was previously appreciated. The findings, reported today in Nature, bring researchers closer to a day when many different kinds of stem cells could be leveraged for disease therapy and regenerative treatments.

"Stem cell colonies contain much variability between individual cells. This has been considered somewhat problematic for developing predictive approaches in stem cell engineering," said the study's co-senior author James Collins, Ph.D., who is a Wyss Institute Core Faculty member, the Henri Termeer Professor of Medical Engineering & Science at MIT, and a Professor of Biological Engineering at MIT. "Now, we have discovered that what was previously considered problematic variability could actually be beneficial to our ability to precisely control stem cells."

The research team has learned that there are many small fluctuations in the state of a stem cell's pluripotency that can influence which developmental path it will follow.

It's a very fundamental study but it highlights the wide range of states of pluripotency," said George Daley, study co-senior author, Director of Stem Cell Transplantation at Boston Children's Hospital and a Professor of Biological Chemistry and Molecular Pharmacology at Harvard Medical School. "We've captured a detailed molecular profile of the different states of stem cells."

Taking this into account, researchers are now better equipped to manipulate and precisely control which cell and tissue types will develop from an individual pluripotent stem cell or stem cell colony.

"The study was made possible through the use of novel technologies for studying individual cells, which were developed in part by collaborating groups at the Broad Institute, giving our team an unprecedented view of stem cell heterogeneity at the individual cell level," said Patrick Cahan, co-lead author on the study and Postdoctoral Fellow at Boston Children's Hospital and Harvard Medical School.

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New single-cell analysis reveals complex variations in stem cells

Researchers recreate stem cells from deceased patients to study present-day illnesses

By adminDecember 3, 2014Stem Cell Medical Center

PUBLIC RELEASE DATE:

1-Dec-2014

Contact: Cara Martinez cara.martinez@cshs.org 310-423-7798 Cedars-Sinai Medical Center @cedarssinai

LOS ANGELES (Dec. 1, 2014) - Research scientists have developed a novel method to re-create brain and intestinal stem cells from patients who died decades ago, using DNA from stored blood samples to study the potential causes of debilitating illnesses such as inflammatory bowel disease.

The lab research, published in the journal STEM CELLS Translational Medicine, could yield new therapies for people who suffer from aggressive motor-neuron and gut-related conditions that proved fatal to the deceased patients who long-ago volunteered their blood samples.

"The potential implications of this research are vast," said Dhruv Sareen, PhD, the study's lead author, and assistant professor and director of the David and Janet Polak Foundation Stem Cell Core Laboratory in the Board of Governors Regenerative Medicine Institute.

By using a deceased patient's stored blood samples, Sareen and his colleagues found that they can develop stem cells known as iPSCs in a petri dish - essentially reanimating diseased cells from patients long after they have died.

This approach allows researchers to connect the dots between a deceased patient's symptoms, genetic information contained in DNA and the behavior of stem cells in the lab. This, in turn, enables investigators to study the biological mechanisms behind diseases and potentially design new therapies.

The technique also allows physicians to replace invasive biopsy procedures typically required of living patients to create iPSC cells.

"These novel developments allow us to create new lines of stem cells from literally millions of patient samples stored in large repositories," said Clive Svendsen, PhD, director of the Board of Governors Regenerative Medicine Institute. "Some of these deceased patients were diagnosed with rare and important diseases."

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Researchers recreate stem cells from deceased patients to study present-day illnesses

World Stem Cell Summit kicks off in SA with Public Education Day

By adminDecember 3, 2014Stem Cell Medical Center

NEWS

1200+ scientists, patient advocates from 40 countries in town for summit

Posted YESTERDAY, 6:04 PM Updated YESTERDAY, 6:33 PM

SAN ANTONIO - More than a thousand scientists, industry leaders and patient advocates from 40 countries are headed to San Antonio for the World Stem Cell Summit.

Organizers are calling it the center of the universe when it comes to stem cells and regenerative medicine.

On Tuesday the summit kicked off with Public Education Day, where some of the smartest scientists in the field broke the topic down into bite-sized pieces.

"To be able to replenish our cells that die within a tissue on a daily basis, in order for us to be able to heal wounds, we have to have stem cells," said Elaine Fuchs, an investigator for the Howard Hughes Medical Institute.

She started her research in the field in the 1970s with work on skin stem cells, and said she was fascinated with creating skin in a petri dish that could then be used for burn therapy.

Fuchs spoke at Public Education Day about the most basic biology of stem cells and said that knowledge is leading to a new world in medicine.

"The biology of stem cells is gong to be and is being extremely valuable in terms of developing new therapies and coming up with new drugs to treat various different devastating diseases," Fuchs said.

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World Stem Cell Summit kicks off in SA with Public Education Day

A Swedish Scientist is Among the Recipients of the Hamdan Medical Award

By adminDecember 1, 2014Stem Cell Medical Center

DUBAI, December 1, 2014 /PRNewswire/ --

The Karolinska Institutet Professor Olle Ringdn, who is also the medical director at the Center for Allogeneic Stem Cell Transplantation at the Huddinge University Hospital in Stockholm, is a co-winner of the Hamdan Award for Medical Research Excellence, in the topic of Cell Therapy, sharing the prestigious Award with the American Professor Carl June.

(Photo: http://photos.prnewswire.com/prnh/20141201/719718 )

At a grand ceremony, H.H. Sheikh Hamdan Bin Rashid Al Maktoum, Deputy Ruler of Dubai, UAE Minister of Finance and the Patron of the Award will honor the Swedish Professor Ringdn alongside with 18 other winners of various categories of this Award at its 8th term (2013-2014).

Prof. Ringden devoted his life to treating patients with various life-threatening disorders. By endless hard work during four decades, he has substantial discoveries in a variety of research fields that led to decreased transplantation-related mortality and improved survival.

He established a stem cell research laboratory, where he, amongst other achievements, pioneered allogenic hematopoetic stem cell transplantation research in Sweden.

He conducted various comprehensive studies of the immune system, thus uncovering the compartmentalization of this system. Subsequent to his work, spleen cells are routinely used for immunological testing of deceased organ donors for transplantation.

Prof. Ringdn developed several immunosuppressive therapies to facilitate peripheral blood stem cell transfer from unrelated donors. He highlighted the potential value of mesenchymal stem cells in various transplantation regimens, and this is one of his numerous achievements in the field of stem cell transplantation.

He established bone marrow and allogenetic hematopoietic stem cell transplantation (AHSCT) in Sweden, and designated a research laboratory for this purpose. This laboratory was the nucleus for what became the Centre for Allogeneic Stem Cell Transplantation (CAST) in 1999, which is unique for this specialty in Northern Europe.

Prof Olle Ringdn changed the way immunomodulatory therapy is applied; by using cells from the patient, or from appropriate donors, to re-focus immune responses, heal inflammation, restore and rebuild tissues. This concept is now well embraced and practiced worldwide; helping to save the lives of many patients.

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A Swedish Scientist is Among the Recipients of the Hamdan Medical Award

Researchers Recreate Pain-Sensing Neurons

By adminDecember 1, 2014Stem Cell Medical Center

Researchers at the Harvard Stem Cell Institute and the Stem Cell and Regenerative Biology Department have successfully developed pain-sensing neurons from mouse and human skin cells, according to a report published in Nature Neuroscience in late November.

The study specifically aimed to simulate primary afferent nociceptors, very specialized pain-sensing neurons.

According to head researcher Clifford J. Woolf, a professor of Neurology and Neurobiology at theMedical School, the findings may lead to development of more effective pain medications, better methods to evaluate who is at risk for developing chronic pain, and ways to combat pain complications resulting from cancer chemotherapy.

What weve been able to do with stem cell technology for the first time is recreate some of the key elements of the nervous system by taking one cell and turning it into a particular other cell, Woolf said. This enables us for the first time to really dissect how human neurons function in the nervous system.

For Woolf, the study is a culmination of many years of research, previously done only in mouse cells.

The fact that we can even create these cells with a human gives us the opportunity to study the mechanisms of the way human nervous systems work that havent been possible before, Woolf said.

Tony L. Yaksh, co-director of the Pain and Symptom Management Core of the University of California at San Diegos Regional Cancer Center, emphasized the studys role in raising new questions for further research.

I think this is highly innovative and an extremely well-done paper, Yaksh said. Id say this represents a very concerted, well-organized group effort to define this very critical issue. It leaves you with more questions than before you read the paper, but it starts the ball rolling in a very exciting way.

Yaksh also highlighted the importance of the study in streamlining gene-specific treatment.

The significance is [that] this allows us to define a mechanism that may be relevant to pain transmission in humans without having to euthanize a human being, Yaksh said.

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Researchers Recreate Pain-Sensing Neurons

Fear of unknown barrier to iPS cures, expert says

By adminDecember 1, 2014Stem Cell Medical Center

Masayo Takahashi, who led the worlds first operation to implant induced pluripotent stem (iPS) cells into a human body, says greater Japanese understanding of the risks and benefits of new medical procedures is key to achieving her goal of standardizing the procedure one day.

About two months after her team successfully transplanted retinal cells grown from iPS cells into a woman in her 70s with age-related macular degeneration, Takahashi, an ophthalmologist at the Riken Center for Developmental Biology in Kobe, said she wanted to make the treatment available and affordable in about 10 years.

My goal is to make the treatment a standard one, Takahashi said Wednesday at the Foreign Correspondents Club of Japan in Tokyo, adding that the pioneering operation in September was just a start.

The operation was primarily aimed at checking for any medical problems that might arise, including the possibility of cancer, after iPS-derived retinal cells were transplanted to the patient, whose form of the disease could lead to loss of vision.

It is expected to take around a year to assess the safety and side effects of the transplant operation.

The most difficult part of the surgery was removal of damaged tissue, Takahashi said. So we were very excited when the damaged tissue was removed safely.

The surgery, performed by a team of researchers at the Riken institute and the Institute of Biomedical Research and Innovation Hospital, was the first in which iPS-derived cells were introduced into a human body.

Developed by Nobel Prize-winning Kyoto University professor Shinya Yamanaka, iPS cells are a versatile type of stem cell that can grow into various types of human body tissue.

However, a potential obstacle to spreading the treatment is reluctance among Japanese to take risks when they encounter something new, including medical treatments, according to Takahashi.

In Japan, people dont accept risks at all, she said, adding it is necessary to accept a certain level of risk when receiving advanced medicine and it is important to understand the balance between risks and benefits.

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Fear of unknown barrier to iPS cures, expert says

Cardiac Stem Cells (CSCs) | University of Maryland Medical …

By adminNovember 30, 2014Stem Cell Medical Center

For immediate release: September 10, 2012

Baltimore, MD --Researchers at the University of Maryland School of Medicine, who are exploring novel ways to treat serious heart problems in children, have conducted the first direct comparison of the regenerative abilities of neonatal and adult-derived human cardiac stem cells. Among their findings: cardiac stem cells (CSCs) from newborns have a three-fold ability to restore heart function to nearly normal levels compared with adult CSCs. Further, in animal models of heart attack, hearts treated with neonatal stem cells pumped stronger than those given adult cells. The study is published in the September 11, 2012, issue of Circulation.

The surprising finding is that the cells from neonates are extremely regenerative and perform better than adult stem cells, says the study's senor author, Sunjay Kaushal, M.D., Ph.D., associate professor of surgery at the University of Maryland School of Medicine and director, pediatric cardiac surgery at the University of Maryland Medical Center. We are extremely excited and hopeful that this new cell-based therapy can play an important role in the treatment of children with congenital heart disease, many of whom don't have other options.

Dr. Kaushal envisions cellular therapy as either a stand-alone therapy for children with heart failure or an adjunct to medical and surgical treatments. While surgery can provide structural relief for some patients with congenital heart disease and medicine can boost heart function up to two percent, he says cellular therapy may improve heart function even more dramatically. We're looking at this type of therapy to improve heart function in children by 10, 12, or 15 percent. This will be a quantum leap in heart function improvement.

Heart failure in children, as in adults, has been on the rise in the past decade and the prognosis for patients hospitalized with heart failure remains poor. In contrast to adults, Dr. Kaushal says heart failure in children is typically the result of a constellation of problems: reduced cardiac blood flow; weakening and enlargement of the heart; and various congenital malformations. Recent research has shown that several types of cardiac stem cells can help the heart repair itself, essentially reversing the theory that a broken heart cannot be mended.

Stem cells are unspecialized cells that can become tissue- or organ-specific cells with a particular function. In a process called differentiation, cardiac stem cells may develop into rhythmically contracting muscle cells, smooth muscle cells or endothelial cells. Stem cells in the heart may also secrete growth factors conducive to forming heart muscle and keeping the muscle from dying.

To conduct the study, researchers obtained a small amount of heart tissue during normal cardiac surgery from 43 neonates and 13 adults. The cells were expanded in a growth medium yielding millions of cells. The researchers developed a consistent way to isolate and grow neonatal stem cells from as little as 20 milligrams of heart tissue. Adult and neonate stem cell activity was observed both in the laboratory and in animal models. In addition, the animal models were compared to controls that were not given the stem cells.

Dr. Kaushal says it is not clear why the neonatal stem cells performed so well. One explanation hinges on sheer numbers: there are many more stem cells in a baby's heart than in the adult heart. Another explanation: neonate-derived cells release more growth factors that trigger blood vessel development and/or preservation than adult cells.

This research provides an important link in our quest to understand how stem cells function and how they can best be applied to cure disease and correct medical deficiencies, says E. Albert Reece, M.D., Ph.D., M.B.A., vice president for medical affairs, University of Maryland; the John Z. and Akiko K. Bowers Distinguished Professor; and dean, University of Maryland School of Medicine. Sometimes simple science is the best science. In this case, a basic, comparative study has revealed in stark terms the powerful regenerative qualities of neonatal cardiac stem cells, heretofore unknown.

Insights gained through this research may provide new treatment options for a life-threatening congenital heart syndrome called hypoplastic left heart syndrome (HLHS). Dr. Kaushal and his team will soon begin the first clinical trial in the United States to determine whether the damage to hearts of babies with HLHS can be reversed with stem cell therapy. HLHS limits the heart's ability to pump blood from the left side of the heart to the body. Current treatment options include either a heart transplant or a series of reconstructive surgical procedures. Nevertheless, only 50-60 percent of children who have had those procedures survive to age five.

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November Tip Sheet from Cedars-Sinai Medical Center

By adminNovember 26, 2014Stem Cell Medical Center

Contact Information

Available for logged-in reporters only

Newswise Following is the November 2014 tip sheet of story ideas from Cedars-Sinai Medical Center. To arrange interviews, please contact the individual listed.

Familys Battle With Cancer Draws Nurse Into Run for Her Brenda Durand, RN, followed in the footsteps of her mother, Bonnie, and became a nurse. But the two women had more in common than their careers. In 2004, Bonnie was diagnosed with stage 3 ovarian cancer. Two years later, Brendas sister learned she had breast cancer, prompting the mother and her two daughters to undergo genetic testing. The results came back: All three women carried the BRCA 1 mutation, putting them at greater risk for breast and ovarian cancer. For the next six years, Brenda and her mom worked to raise funds for ovarian cancer research, with Brenda participating in Cedars-Sinais annual Run for Her 5K Run and Friendship Walk. In 2012, Bonnie lost her life to ovarian cancer. But the loss of her mother has strengthened Brendas resolve to fight harder against ovarian cancer, and on Nov. 9, she participated in the 10th annual Run for Her. She is available for interviews. CONTACT: Cara Martinez, 310-423-7798; Email cara.martinez@cshs.org

Lou Gehrigs Disease Study: Renewing Brains Aging Support Cells May Help Neurons Survive Lou Gehrigs disease, also known as amyotrophic lateral sclerosis, or ALS, attacks muscle-controlling nerve cells motor neurons in the brain, brainstem and spinal cord, leading to progressive weakness and eventual paralysis of muscles throughout the body. Patients typically survive only three to five years after diagnosis. Now, with publication of a study by investigators at the Cedars-Sinai Board of Governors Regenerative Medicine Institute, ALS researchers know the effects of the attack are worsened, at least in part, by the aging and failure of support cells called astrocytes, which normally provide nutrients, housekeeping, structure and other forms of assistance for neurons. CONTACT: Sandy Van, 808-526-1708; Email sandy@prpacific.com

Cardiac Stem Cell Therapy May Heal Heart Damage Caused by Duchenne Muscular Dystrophy Researchers at the Cedars-Sinai Heart Institute have found that injections of cardiac stem cells might help reverse heart damage caused by Duchenne muscular dystrophy, potentially resulting in a longer life expectancy for patients with the chronic muscle-wasting disease. CONTACT: Sally Stewart, 310-248-6566; Email sally.stewart@cshs.org

Cedars-Sinai Study of Lou Gehrigs Disease Shifts Origin Focus to Brains Motor Neurons Lou Gehrigs disease, also known as amyotrophic lateral sclerosis, or ALS, might damage muscle-controlling nerve cells in the brain earlier in the disease process than previously known, according to research from the Cedars-Sinai Board of Governors Regenerative Medicine Institute. The findings could shift researchers attention from the spinal cord to the brains motor cortex as the diseases initial point of dysfunction. CONTACT: Sandy Van, 808-526-1708; Email sandy@prpacific.com

New Alzheimers Program to Focus on Prevention, Intervention, Research and Support Seeking to stem the rapid increase of Alzheimers disease, Cedars-Sinai has launched a new Alzheimers Prevention Program to help identify patients at risk of developing the neurological disorder and to reduce the impact on those diagnosed with the slow-moving condition. The program represents a concerted effort by clinicians, researchers, patients, families, caregivers and community agencies to address an approaching tsunami of Alzheimers care. Medical authorities expect the number of cases nationally to triple by 2050, inundating the healthcare system with patients and costing more than $1 trillion. CONTACT: Sandy Van, 808-526-1708; Email sandy@prpacific.com

Sister to Sister, Nations First Organization Dedicated to Womens Heart Health, Donates Educational Content, Intellectual Property to Cedars-Sinai Heart Institute

After 15 years of educating women about heart disease and providing more than 100,000 free cardiovascular screenings, Sister to Sister: The Womens Heart Health Foundation founded by Irene Pollin, MSW, announced today that the organization will cease operations on Dec. 31. Pollin also announced that the pioneering organization is donating its educational content and intellectual property to the Barbra Streisand Womens Heart Center in the Cedars-Sinai Heart Institute. CONTACT: Sally Stewart, 310-248-6566; Email sally.stewart@cshs.org

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November Tip Sheet from Cedars-Sinai Medical Center