The Lipogems technology has great promise, but experts say itll take time to assess how successful the new procedure is.

What if you could put that little bit of body fat around your midsection to good use?

A procedure called Lipogems utilizes a persons body fat as a source of stem cells to help treat arthritis and joint conditions.

At least thats the promise.

Lipogems was approved for widespread use by the Food and Drug Administration (FDA) in November 2016, and its already garnering a lot of attention.

Rush University Medical Center recently became the first sports medicine specialists in the Midwest to offer treatment with the device.

The technology is ideal for patients with certain orthopedic conditions, such as painful joints including the knee, ankle, or shoulder with limited range of motion. Additionally, it can be used in soft tissue defects located in tendons, ligaments, and/or muscles to improve the biologic environment, said Dr. Brian Cole, professor of orthopedic surgery, and section head of the Rush Cartilage Restoration Center, in a press release.

Read more: Stem cell therapies offering hope for MS patients

Stem cells work by growing and differentiating themselves into different cells in the body based on the site of injection.

They are believed to help the natural regenerative processes in the body.

Hence they have earned the nickname as mini drug stores based on their ability to secrete a spectrum of bioactive molecules and support the natural regeneration of focal injuries.

Stem cells can be harvested from certain parts of the human body, most notably bone marrow and adipose tissue (fat).

Harvesting bone marrow stem cells is a significantly more invasive and time-consuming procedure that is performed using general anesthesia.

Lipogems offers a novel approach to orthopedic stem cell treatments by using a persons own fat.

The procedure uses a small incision into an area of subcutaneous fat, from which a quantity of fat tissue is harvested and processed by the Lipogems apparatus.

The technology itself, which really is the device that processes the fat, creates a concentration of fat that has been cleansed of all the extraneous things like red blood cells and fibrous tissues, Cole told Healthline.

The concentrated stem cells within that fat tissue are then applied to the problematic joint or bone area.

The procedure can be completed in under 30 minutes.

Read more: Stem cell therapy a possible treatment for rheumatoid arthritis

Lipogems offers a streamlined procedure for stem cell treatment, but there is nothing new about the science itself.

The use of stem cells to treat a variety of conditions has been ongoing for some time now.

As Healthline reported earlier this year, stem cells have been touted as a breakthrough treatment for some time, but real proof of efficacy is still being researched.

The same is true for Lipogems.

What were lacking is really good data at this point in the clinical setting, Cole said. There is substantial data in the laboratory suggesting that these cells may function in the way Ive described: reducing inflammation and so forth. But, we really dont have yet much in the way of good solid clinical data saying that definitively this is making a difference.

He further cautions individuals thinking that the new procedure, or that stem cells in general, are a panacea.

Read more: Unproven stem cell treatments offer hope but also risks

Instead, he would like those seeking orthopedic treatment to understand that Lipogems is just one part of a much larger and more complex suite of tools used by physicians.

It has to be taken into context of all the other possible treatment options, from simply icing down a swollen ankle, to changing your daily activity, to surgery.

The unfortunate thing is that people think, well this is the solution that can be used instead of, say, a joint replacement and no longer do we need to do surgery, said Cole.

Nothing could be further from the truth.

Nonetheless, Cole and his team are still excited about the possibilities of the Lipogems procedure.

Using a readily available and easily accessible substance like fat as a source of stem cells could have far-reaching implications for procedures in the future.

Were optimistic and intuitively there is a good argument to be made that this is as good or better than any other source of stem cells, said Cole.

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The BMT program at MedStar Georgetown is a joint effort with specialists from John Theurer Cancer Center and a key component of the Lombardi Comprehensive Cancer Center, the only cancer program in the Washington, D.C. region designated by the National Cancer Institute (NCI) as a comprehensive cancer center.

"Once considered experimental, BMT is today's established gold standard for treating patients with a number of malignant and other non-malignant diseases of the immune system, blood, and bone marrow, including multiple myeloma, lymphoma, and acute and chronic leukemia. For some conditions, blood stem cell transplant can provide a cure in patients who have failed conventional therapies," says Scott Rowley, M.D., chief of the BMT program at MedStar Georgetown as well as a member of the John Theurer Cancer Center's Blood and Marrow Stem Cell Transplantation. "For some conditions, it can actually be a cure; for others, it prolongs survival and improves quality of life. Having performed 100 BMTs at MedStar Georgetown including allogenic transplantation illustrates the strength and maturity of our program achieved in rather short time."

MedStar Georgetown's program is also the only comprehensive BMT center within Washington, D.C. and southern Maryland with accreditation from the Foundation for the Accreditation of Cellular Therapy (FACT) for adult autologous procedures, where the patient donates his or her own cells.

The BMT program at John Theurer Cancer Center is one of the top 10 transplant programs in the United States, with more than 400 transplants performed annually.

A BMT involves a two-step process: first, collecting bone marrow stem cells from the patient and storing them for future use. Then, a week or so later, patients receive high dose chemotherapy to eliminate their disease. The previously stored cells are reinfused back into the bloodstream, where after reaching the bone marrow, they begin repopulating and allow the patient to recover their blood counts over the following 2 weeks.

"Even though BMT is considered standard therapy for myeloma worldwide, in the United States fewer than 50 percent of the patients who could benefit from BMT are referred for evaluation," says David H. Vesole, M.D., Ph.D., co- chief and director of Research of John Theurer Cancer Center's Multiple Myeloma division and director of MedStar Georgetown's Multiple Myeloma Program.

"That's mostly due to physicians' concerns that a patient is too old or compromised from other health conditions like diabetes, cardiac disease or renal failure. But new techniques and better supportive care have improved both patient outcomes and the entire transplant process, extending BMT to more patients than ever before."

The MedStar Georgetown/Georgetown Lombardi Blood and Marrow Stem Cell Transplant Program is part of a collaborative cancer research agenda and multi-year plan to form an NCI-recognized cancer consortium. This recognition would support the scientific excellence of the two centers and highlight their capability to integrate multidisciplinary, collaborative research approaches to focus on all the aspects of cancer.

The research areas include expansion of clinical bone marrow transplant research; clinical study of "haplo" transplants use of half-matched stem cell donor cells; re-engineering the function and focus of key immune cells; and the investigation of "immune checkpoint" blocking antibodies that unleash a sustained immune response against cancer cells.

"In this partnership, we've combined John Theurer's strength in clinical care with Georgetown Lombardi's strong research base that significantly contributes to clinical excellence at MedStar Georgetown. By working together, we have broadened our cancer research to offer more effective treatment options for tomorrow's patients," says Andrew Pecora, M.D., FACP, CPE, president of the Physician Enterprise and chief innovations officer, Hackensack Meridian Health. "This is one of many clinical and research areas that have been enhanced by this affiliation."

"Our teams are pursuing specific joint research projects we feel are of the utmost importance and significance in oncology particularly around immuno-oncology as well as precision medicine," says Andr Goy, M.D., MS, chairman of the John Theurer Cancer Center and director of the division chief of Lymphoma; chief science officer and director of Research and Innovation, RCCA; professor of medicine, Georgetown University. "Together our institutions have a tremendous opportunity to transform the delivery of cancer care for our patient populations and beyond."

ABOUT THE JOHN THEURER CANCER CENTER AT HACKENSACK UNIVERSITY MEDICAL CENTER John Theurer Cancer Center at Hackensack University Medical Center is New Jersey's largest and most comprehensive center dedicated to the diagnosis, treatment, management, research, screenings, and preventive care as well as survivorship of patients with all types of cancers. The 14 specialized divisions covering the complete spectrum of cancer care have developed a close-knit team of medical, research, nursing, and support staff with specialized expertise that translates into more advanced, focused care for all patients. Each year, more people in the New Jersey/New York metropolitan area turn to the John Theurer Cancer Center for cancer care than to any other facility in New Jersey. Housed within a 775-bed not-for-profit teaching, tertiary care, and research hospital, the John Theurer Cancer Center provides state-of-the-art technological advances, compassionate care, research innovations, medical expertise, and a full range of aftercare services that distinguish the John Theurer Cancer Center from other facilities.www.jtcancercenter.org.

ABOUT MEDSTAR GEORGETOWN UNIVERSITY HOSPITAL MedStar Georgetown University Hospital is a not-for-profit, acute-care teaching and research hospital with 609 beds located in Northwest Washington, D.C. Founded in the Jesuit principle of cura personaliscaring for the whole personMedStar Georgetown is committed to offering a variety of innovative diagnostic and treatment options within a trusting and compassionate environment. MedStar Georgetown's centers of excellence include neurosciences, transplant, cancer and gastroenterology. Along with Magnet nurses, internationally recognized physicians, advanced research and cutting-edge technologies, MedStar Georgetown's healthcare professionals have a reputation for medical excellence and leadership. For more information please visit: medstargeorgetown.org/bmsct

ABOUT HACKENSACK MERIDIAN HEALTH HACKENSACK UNIVERSITY MEDICAL CENTER Hackensack Meridian Health Hackensack University Medical Center, a 775-bed nonprofit teaching and research hospital located in Bergen County, NJ, is the largest provider of inpatient and outpatient services in the state. Founded in 1888 as the county's first hospital, it is now part of one of the largest networks in the state comprised of 28,000 team members and more than 6,000 physicians. Hackensack University Medical Center was listed as the number one hospital in New Jersey in U.S. News & World Report's 2016-17 Best Hospital rankings - maintaining its place atop the NJ rankings since the rating system was introduced. It was also named one of the top four New York Metro Area hospitals. Hackensack University Medical Center is one of only five major academic medical centers in the nation to receive Healthgrades America's 50 Best Hospitals Award for five or more years in a row. Becker's Hospital Review recognized Hackensack University Medical Center as one of the 100 Great Hospitals in America 2017. The medical center is one of the top 25 green hospitals in the country according to Practice Greenhealth, and received 25 Gold Seals of Approval by The Joint Commission more than any other hospital in the country. It was the first hospital in New Jersey and second in the nation to become a Magnet recognized hospital for nursing excellence; receiving its fifth consecutive designation in 2014. Hackensack University Medical Center has created an entire campus of award-winning care, including: the John Theurer Cancer Center; the Heart & Vascular Hospital; and the Sarkis and Siran Gabrellian Women's and Children's Pavilion, which houses the Joseph M. Sanzari Children's Hospital and Donna A. Sanzari Women's Hospital, which was designed with The Deirdre Imus Environmental Health Center and listed on the Green Guide's list of Top 10 Green Hospitals in the U.S. Hackensack University Medical Center is the Hometown Hospital of the New York Giants and the New York Red Bulls and is Official Medical Services Provider to The Northern Trust PGA Golf Tournament. It remains committed to its community through fundraising and community events especially the Tackle Kids Cancer Campaign providing much needed research at the Children's Cancer Institute housed at the Joseph M. Sanzari Children's Hospital. To learn more, visit http://www.HackensackUMC.org.

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/john-theurer-cancer-center-and-medstar-georgetown-university-hospital-announce-100th-blood-stem-cell-transplant-300471445.html

SOURCE Hackensack Meridian Health

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John Theurer Cancer Center and MedStar Georgetown University Hospital Announce 100th Blood Stem Cell Transplant - PR Newswire (press release)

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Xconomy Boston

If youve donated blood, you probably received a follow-up message asking you to consider a platelet donation. Donation centers must constantly replenish their blood supplies, but the need for platelets is particularly acute. Compared to other components of blood, platelets are fragile and short lived, which puts high demand on the small supply of these cell fragments that are key to stopping bleeding.

Plasma separated from blood can be frozen and red blood cells can be refrigerated for up to 42 days. But platelets, which must be stored at room temperature, last just five days. After pathogen screening and transport, platelets have about two days to reach a patient, says Sven Karlsson, president and co-founder of Boston-based Platelet Biogenesis.

Karlssons startup aims to bring hospitals an alternative: platelets produced as needed from stem cells. Platelet BioGenesis, a spinout from Harvard University and Brigham and Womens Hospital, now has $10 million from a Series A round of funding to support preclinical testing of its regenerative medicine technology.

The body produces the components of blood in bone marrow. Platelets are made by a type of bone marrow cell called a megakaryocyte. Platelet Biogenesis produces its platelets in a two-step process. It first develops megakaryocytes from stem cells in culture, Karlsson says. Next, the megakaryocytes are fed into a device that the company developed that puts the cells through a process the company says is similar to what occurs in natural bone marrow physiology and results in the production of platelets.

Were making real, functional platelets, Karlsson says. Instead of mimicking the platelet, were mimicking the process of making a platelet.

Platelet transfusions are needed by patients whose bodies dont produce enough platelets, as well as those who have clotting problems. These transfusions are also used to treat patients who have cancer or are undergoing surgery. Since most platelet donation centers are in urban areas, the short shelf life of platelets makes supplies tight in rural areas. Donations dont entirely solve the problem, Karlsson says. While donating whole blood takes about 15 minutes, platelet donation takes 90 minutes, which makes it harder to get donors.

Researchers have tried to address the platelet shortage problem by developing synthetic platelets. But Karlsson says that the synthetic versions can cause side effects. The idea for replicating the bodys process of producing platelets stems from more than a decade of research. Jonathan Thon, the CEO and co-founder of Platelet BioGenesis, was pursuing ways to extend the shelf life of platelets. But Karlsson says Thon concluded a better approach might be developing a way to replace human donors. As a post-doctoral researcher at Harvard, he invented a microfluidic device that mimics a human bone marrow.

Before Platelet BioGenesis can bring its platelet-making technology to the market, it will need FDA approval. The regulator treats blood products as therapeutics, which means that the company will need to conduct clinical trials. Karlsson says the funding round will support preclinical research. Within three years, he expects the startup will be able to start Phase 1 safety studies. For those trials, Platelet BioGenesis will need to raise additional funds. The current financing round was led by Qiming US Healthcare Fund and included participation from Vivo Capital, VI Ventures, Adena Partners, eCoast Angels, as well as other unidentified investors.

If Platelet BioGenesis wins FDA approval, Karlsson says the company plans to become a manufacturer, selling its platelets in the existing blood supply chain. Its early to talk about pricing, but he says that Platelet Biogenesis products could be more affordable because they are produced from a pathogen-free source. They could also be produced on demand. While the startups platelets should have the same shelf-life as platelets from human donors, Karlsson says that the parent cells that produce platelets can be stored frozen. When platelets are needed, thawed cells can be placed in the companys microfluidic device, which produces platelets within hours.

Karlsson says Platelet BioGenesis might be able offer another advantage: beating the five-day shelf life of donor platelets. In the body, a platelet lasts 10 days. If all of Platelet BioGenesis platelets are born on the same day, they should theoretically have the same 10-day life, he says. Thats one of the things the company plans to test in upcoming trials. The company will also evaluate its platelets to make sure that they are comparable to donor platelets.

Imageby Wikimedia user Erhabor Osarovia a Creative Commons license.

Frank Vinluan is editor of Xconomy Raleigh-Durham, based in Research Triangle Park. You can reach him at fvinluan [at] xconomy.com

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Harvard Biotech Spinout Lands $10M to Make Platelets From Stem Cells - Xconomy

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Patients with rare blood cancer brought back to normal - The Hindu The Hindu Undergo bone marrow transplant at Apollo Cancer Institute. and more »

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This week alone in the news weve seen air strikes, suicide bombings and murders caused by hate. Violence over the hue of someones skin, the way they speak or how they dress. Hate-filled speech by neighbors at meetings and on Facebook. At dinner yesterday, my 8-year-old step-daughter asked her dad, Whats a bomb? My heart is heavy.

Its easy to forget that we are more alike than we are unalike. I offer to you a different perspective.

Six years ago my brother got the call he had Hodgkin Lymphoma, a cancer that starts in cells that are part of the bodys immune system. He was 28 years old. It started as a visible lump under his collar bone, and sometimes you wonder how can so much suffering be caused by such a little lump? And so my story begins.

About one year into his treatment, he reached remission, and from there he was required to undergo an autologous stem cell transplant (a transplant using his own stem cells) to replace his bone marrow and stem cells that were destroyed by chemotherapy and radiation. Fast-forward 10 or so months and my brothers cancer returned. This time the treatment plan had to change his body needed help actually fighting the cancer cells, rather than just a replenishment of normal blood cells. This time around, he required an allogeneic stem cell transplant (a transplant using the stem cells from a healthy donor) and as his sister, I needed to be tested to see if I was a tissue match.

This was all new to me and our family. You hear a lot about cancer. We all know someone who has it, if you dont have cancer yourself. But I knew nothing about stem cell transplants or what it meant to be a donor. First we had to find out if my brother and I were a match.

I received a kit in the mail and all I had to do was swab the inside of my cheek, place the swab inside a sealed bag, and mail it back to the hospital. A week or so later, my brother got the news from his doctor that changed our lives. I was, in fact, a match a near perfect match and we could move forward with his second stem cell transplant.

At this point in my story, youre probably thinking, Of course, youd be a match, youre his sister. I assumed so, as well. Read on.

On Aug. 12, 2016, my brother and I underwent our stem cell transplant at Dana Farber/Brigham and Womens Hospital in Boston. There are two different ways to donate stem cells peripheral blood stem cells (stem cells extracted from your blood after receiving five days of injections of a drug called filgrastim, used to increase the number of blood-forming cells in your bloodstream) and bone marrow (a surgical procedure where doctors use needles to withdraw liquid marrow from both sides of the back of your pelvic bone). Due to my brothers specific treatment plan, he required pure bone marrow, and my bone marrow was taken from my pelvis. Two liters worth of my bone marrow was processed at Dana Farber and then brought to my brother immediately, who received it via an IV drip.

So how does my story end? Why am I telling you all this?

My brother is thriving. My pelvis has healed. And we were absolutely blessed to find a match right within our family.

The reality is that fewer than 30 percent of patients with a blood cancer or blood disease will find a related-donor; the other 70 percent, thousands of patients with blood cancers like leukemia and lymphoma, sickle cell anemia or other life-threatening diseases, depend on the national bone marrow registry to find a match to save their life. Some day you or someone you love might depend on a complete stranger who might be a Muslim, a Republican, gay or straight. But it wont matter because from the inside, they will be the same.

I plead with you to remember that we are more alike than we are unalike, and to do something positive for humanity.

You can visit http://www.bethematch.org and join the Be The Match national bone marrow registry.

Or you can attend one of my in-person donor drives in Greenfield over the next few months. The first will be this Saturday, June 10, from 2 to 4 p.m. at the Pints in the Park event at the Greenfield Energy Park.

If you are between the ages of 18 and 44, patients especially need you. You could be someones cure.

I note the obvious differences

between each sort and type,

but we are more alike, my friends,

than we are unalike.

We are more alike, my friends,

than we are unalike.

From Human Family, a poem by Maya Angelou

Ashli Stempel is a Greenfield resident and a member of the Greenfield Town Council.

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June 8, 2017 One example of STAT1 GOF Mutation phenotype. Credit: Hiroshima University

Researchers report the first-ever study assessing how patients with "gain of function" mutation of the STAT1 gene respond to stem cell transplantation. It involved 15 young patients from nine different countries, each suffering a range of complications caused by the gene's mutation.

Of these, only six survived a regime of stem cell transplantationwith five completely cured and disease free by the study's conclusion.

The study was carried out by Dr. Satoshi Okada (Hiroshima University), Professor Jennifer Leiding (University of Florida), Professor Tomohiro Morio (Tokyo Medical and Dental University), and Professor Troy Torgerson (University of Washington).

Dr. Okada, who first discovered the STAT1 gain of function mutation in 2011, says, "Overall, this result is disappointing but the fact that five patients were cured proves that treatment with stem cells can work, and we now need to learn from these 15 individual cases."

The STAT1 gene plays a vital role in the body's immune system. Rare mutations can lead to STAT1's over-activation (GOF) and autoimmunity.

While the majority of patients afflicted typically show mild to moderate symptoms involving fungal (mostly Candida), bacterial, and viral infectionsabout 10 percent of cases are severe and life threatening.

Until now, developing suitable treatments has been challenging; e.g. anti-fungal drugs temporarily treat the symptoms but not the source mutation, and immunosuppressive therapies often do more harm than good by knocking out already overburdened immune systems.

With only one confirmed case prior to this study of a sufferer being successfully cured using stem cell transplantation, researchers are keen to build an understanding of best practices in order to offer real hope for the typically young sufferers of this condition.

The 15 selected patients were sourced via an international appeal to transplant centers and consortiums. Their ages ranged from 13 months to 33 years at the time of treatment. Screening by HU researchers confirmed that each had the STAT1-GOF mutation, and that the mutation was the source of their ailments.

Treatment was carried out independently by centers around the world. It used chemotherapy to eradicate the host's bone marrowthe source of the damaging STAT1 mutation in these patients. Healthy stem cell cultures sourced from donors were then transplanted into the subjects with the aim of reconstituting their bone marrow to a mutation-free, disease-fighting state.

The researchers suspect three reasons for the low 40 percent success rate:

In response, the researchers have made several proposals for improving this treatment. Due to most of the patients having mild to moderate ailments, only those suffering from severe symptoms should undergo this treatment. In addition, the chemotherapy dosage should be reduced. Those who received low-dose chemotherapy reacted better.

However, a balance must be struck. Low-dose chemotherapy may not eradicate host bone marrow to the extent required for its reconditioning the chance of transplant rejection is thus increased. With this in mind, support treatment may be required to neutralize host antibodies and prevent attacks of introduced stem cells.

Finally, due to the relative success seen in younger patients, stem cell transplantation should occur at as early an age as possible. Due to recent advancements in STAT1-GOF diagnosis, early detection is now a very real possibility hopefully leading to greater success rates, and less suffering for those carrying this potentially devastating mutation.

Explore further: 'Smart' genetic library makes disease diagnosis easier

More information: Jennifer W. Leiding et al. Hematopoietic stem cell transplantation in patients with Gain of Function STAT1 Mutation, Journal of Allergy and Clinical Immunology (2017). DOI: 10.1016/j.jaci.2017.03.049

Researchers at Hiroshima University have developed a smart genetic reference library for locating and weeding out disease-causing mutations in populations.

A single blood test and basic information about a patient's medical status can indicate which patients with myelodysplastic syndrome (MDS) are likely to benefit from a stem cell transplant, and the intensity of pre-transplant ...

UCLA researchers have developed a stem cell gene therapy cure for babies born with adenosine deaminase-deficient severe combined immunodeficiency, a rare and life-threatening condition that can be fatal within the first year ...

Physicians at the University of Illinois Hospital & Health Sciences System have cured 12 adult patients of sickle cell disease using a unique procedure for stem cell transplantation from healthy, tissue-matched siblings.

Using a technique that avoids the use of high-dose chemotherapy and radiation in preparation for a stem cell transplant, physicians at the University of Illinois Hospital & Health Sciences System have documented the first ...

A large, nationwide study published in the journal JAMA Oncology found that people who received transplants of cells collected from a donor's bone marrow the original source for blood stem cell transplants, developed decades ...

In news that may bring hope to asthma sufferers, scientists discover a mechanism that provides a possible new target for allergy treatments.

(HealthDay)Administration of allergen immunotherapy (AIT) in patients with allergic asthma leads to lower short-term symptom and medication scores, according to a review published online May 19 in Allergy.

A single treatment giving life-long protection from severe allergies such as asthma could be made possible by immunology research at The University of Queensland.

Malaria caused by Plasmodium parasites is a life-threatening infectious disease that kills at least half a million people annually while causing over 200 million new infections. In some cases, complications can quickly develop ...

Virtually the entire population of sub-Saharan Africa, and some 70% of African Americans, carry a gene variant (allele) which results in a trait referred to as Duffy-negative. It has long been known that carriers of this ...

Immunotherapy drugs to combat cancer have stimulated tremendous excitement among patients and physicians alike. They debuted in 2011, when the U.S. Food and Drug Administration approved ipilimumab (Yervoy) to treat metastatic ...

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Stem cell treatment for lethal STAT1 gene mutation produces mixed results - Medical Xpress

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June 6, 2017 Drs. Carlos Isales, Meghan McGee-Lawrence, William D. Hill and Mark Hamrick. Credit: Phil Jones

With age, the form and function of our bones and muscles drop off, putting us as increased risk for frailty and falls.

Now researchers at the Medical College of Georgia at Augusta University are dissecting just what happens to the stem cells that make the tissues, which help keep us upright, with an eye on improving our healthspan.

Osteoporosis already is a major public health problem affecting about 44 million Americans and costing billions annually. The world's older population is growing at an unprecedented rate with 8.5 percent of the worldwide population - 617 million people - age 65 and older, a proportion estimated to reach 17 percent by 2050, according to the National Institute on Aging.

"After age 65 you start losing about 1 percent of both muscle and bone per year," said Dr. Carlos Isales, endocrinologist, Regents' professor and vice chair for clinical affairs in the MCG Department of Neuroscience and Regenerative Medicine.

"Daily exercise decreases the slope of that decline. But what we are focusing on is trying to see if we can flatten the curve even further," said Isales, principal investigator on a new $9.3 million Program Project grant from the National Institutes of Health.

Time seems to alter the dynamic between the mesenchymal stem cells making bone and muscle and the amino acids that fuel them. The MCG scientists also have evidence it changes the signals stem cells send each other.

The bottom line: Our stem cell population gets reduced and the cells we have become less efficient at making bone and muscle, often opting for the easier task of making fat instead, Isales said.

The team, which includes principal investigators bone biologist Dr. Mark Hamrick, stem cell researcher Dr. William D. Hill and biomedical engineer Dr. Meghan McGee-Lawrence, wants to keep stem cells focused on making bone and muscle.

"We are looking at stem cells as a group and what is happening to them as we age," Hill noted. "This includes a loss of direction so they aren't as functional as they were before. The other thing we are looking at is their survival and their numbers."

"We are trying to figure out why the changes are happening and if we can target those cells to make them want to make bone again," McGee-Lawrence said.

Much as the function of bone and muscle is interwoven, so is their health and the factors that promote their loss or survival also are similar, said Hamrick.

A major culprit in their breakdown appears to be the metabolite kynurenine, a byproduct of the essential amino acid tryptophan. Tryptophan is among the nine amino acids our body can't make and we must consume in foods like turkey and soybeans so we can perform essentials like making protein. The researchers also think the fuel sends signals to cells, ones that aging stem cells apparently don't get.

The unhealthy metabolite is the result of a natural action called oxidation, which occurs anytime cells use oxygen. Particularly with age, the free radicals produced by oxidation can also damage cells. Kynurenine results when the enzyme, indoleamine 2,3 dioxygenase, or IDO, which a variety of tissues make to help moderate an immune response, oxidizes tryptophan. Over time, kynurenine piles up and appears to alter the dynamic of bone and muscle formation.

Again, somewhat ironically, the many functions of essential amino acids include working as antioxidants, so the researchers are putting together nutrient cocktails - minus tryptophan and with reduced protein content - that they hope can reverse age-related damage. Isales notes that they may find that other amino acids produce similar problems as tryptophan in the aged environment.

So they also are taking more direct approaches like whether an IDO inhibitor - which is already in clinical trials as a cancer fighter - can reverse changes and get stem cells to regain more youthful function.

In an effort to begin to see if what they have seen in laboratory mice holds up in humans, they are trying both approaches in human stem cells retrieved during the process of a knee or hip replacement by colleagues in the MCG Department of Orthopaedic Surgery.

They have laboratory evidence that in mice at least, high kynurenine levels impact the ability of cells in the bone marrow to make bone-forming cells called osteoblasts. In fact, even relatively young mice fed kynurenine experience bone loss, an increase in bone destruction by cells called osteoclasts and increased fat in their bone marrow. Conversely, mice with IDO knocked out maintain strong bone mass.

"You can make an old mouse young and you can make a young mouse old," Hill noted.

The team also has evidence that part of how age-related increases in kynurenine does damage is by altering microRNAs - small but powerful pieces of RNA that can control expression of hundreds of genes at the same time - as well as vesicles called exosomes that are hauling the microRNAs around. Stem cells secrete exosomes as one way to communicate, and apparently aging stem cells don't communicate well with each other.

"Exosomes are one mechanism of crosstalk between cells and also between different organs," said Hamrick. "Your liver is producing exosomes, fat produces exosomes, they will hit other organs and they carry, in some cases, positive messages and in some cases bad messages," said Hamrick, who is leading this project to restore positive messaging.

They have laboratory evidence that aging alters at least two microRNAs, miR-141 and miR-183, which prompts cells to make bone-eating instead of bone-forming cells. Again, they have shown that even young stem cells exposed to older exosomes will assume this bone-reducing stance. But they also have some evidence that some of the dietary interventions Isales is looking at could reverse the ill effects.

The team recently reported in the journal Tissue Engineering that exosomes from old and younger mice were similar in size and number and both had a lot of miRNAs. But aged exosomes had significantly and specifically more mi183, an miRNA already associated with cancer. In this case, high mi183 appears to decrease cell proliferation and the ability of immature cells to become bone cells and to support the general deterioration that comes with age, called senescence. Age-related increases of reactive oxygen species and oxidative stress help increase mi183 levels and these undesirable results. When researchers treat mesenchymal stem cells from young animals with exosomes from old mice, is suppresses formation of muscle-making genes; giving mi183 directly to bone and muscle producing cells makes them start acting old. Now they want to know more about how aging changes the secretion and cargo of exosomes by mesynchymal stem cells and how that in turn contributes to bone and muscle loss.

A third project, led by Hill, will focus on the cargo, the miRNAs, to learn more about exactly how they impact bone formation and turnover. "We think that the amino acids are controlling the expression of specific sets of microRNA," Hill said. That means they may want to target and even eliminate key or critical microRNAs, which could obviously affect expression of numerous genes as a result.

They also are exploring aging's impact on stromal cell derived factor 1, or SDF-1, which is critical to helping keep stem cells in the bone marrow and focused on making bone. Age-related changes appear to make SDF-1 instead encourage stem cells to wander. The researchers note that while these cells do often need to leave the bone marrow, to say help heal an injury, these age-related travels are random and often cells don't find their way back. A consistent goal is identifying intervention targets.

"The idea is if we can change the environment and change how they are signaling to themselves and to other cells, we can modify the stem cell directly that way," Hill said.

They are looking upstream as well for earlier points of intervention, including what is happening to histone deacetylase-3, or HDAC3. They have evidence that HDAC3, another pervasive regulator in the body that can turn gene expression up or down, is important in stem cells' age-related propensity to make fat instead of bone.

At least one reason is that reduced HDAC3 means less bone, which literally makes more room for fat, said McGee-Lawrence, who is leading these studies. Her previous studies have shown that when HDAC3 is deleted from the skeleton, bones are weaker, much like what occurs with aging.

Now they have evidence that mice treated with kynurenine, for example, have suppressed HDAC3 expression in the bone. They want to know more about just how HDAC3 gets suppressed as we age and exactly what that does to bone formation and fat storage besides just making room. The new grant is allowing them to put the pieces together better, looking further at just what suppresses HDAC3 and what suppression does to bone versus fat formation. The bottom line again is identifying early points of intervention and potentially nutrients to intervene.

"Something in the microenvironment of the bone is causing the cells, instead of wanting to make bone, they are storing a lot of fat," McGee-Lawrence said. "Some of these epigenetic factors, like HDAC3, some of the environmental factors like changes in the amino acids are causing the cells to dysfunction. We are hoping to figure out what that signal is and how to reverse it and to make those cells want to start making bone again."

Identical twin studies have shown that environmental factors definitely play a role, since the bone/muscle health of these twins often is not identical even though their genes are, Isales said. Rather than changing the genes themselves, environmental factors appear to have changed their expression: which ones are turned or on off. These epigenetic changes include factors from diet to stress to sleep patterns to age.

There are 20 amino acids, which are essential to protein production and a variety of other functions from giving cells structure to helping organs functions. Kyrurenine also is associated with the degeneration of our brain and immune system as we age. Mesynchymal stem cells also produce blood, cartilage and fat cells.

Isales also is vice chair of clinical and translational research in the MCG Department of Orthopaedics and a faculty member in the MCG Department of Medicine. Hamrick, Hill and McGee-Lawrence are all faculty members in the MCG Department of Cellular Biology and Anatomy. Other scientists helping support three core laboratories for the interrelated studies include the Administrative Core with Biostatistics, Maribeth Johnson and Dr. Jie Chen, MCG Department of Biostatics and Epidemiology; the Bone Biology Core, Dr. Mohammed Elsalanty, Department of Oral Biology, Dental College of Georgia at AU; and the Bone Stem Cell Core, Dr. Xingming Shi, MCG Department of Neuroscience and Regenerative Medicine.

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Major research initiative explores how our bones and muscles age, new ways to block their decline - Medical Xpress

Bone Marrow Stem Cells Comments Off on Major research initiative explores how our bones and muscles age, new ways to block their decline – Medical Xpress | June 7th, 2017

Shares of Shire (SHPG) were down $3.22 or 1.85% in early trading Wednesday to $170.88 after the company said it would transfer its U.S. investigational new drug application for for Graft-Versus-Host Disease candidate Alpha-1 Antitrypsin to Kamada (KMDA) . The treatment is aimed at addressing complications fromstem cell or bone marrow transplants.

Kamada is developing the drug in Europe. Kamada shares were down nearly 7% to $7.64.

Calithera Biosciences (CALA) was up to $16.10, a spike of 70 cents or 4.55%, after the FDA designated the company's lead product candidate, CB-839, in combination with Novartis' AFINITOR for Fast Track review for the treatment of metastatic renal cell carcinoma in patients who have received at least two prior lines of therapy.

Shares of Synergy Pharmaceutical (SGYP) rose 4.5% to $4.15 after the FDA accepted for review its supplemental New Drug Application for TRULANCE a candidate to treat irritable bowel syndrome with constipation. The FDA approved TRULANCE to treat chronic idiopathic constipation in January.

Over at Real Money, Bret Jensen looks at 4 Undervalued Biotech Stocks.

Also, Jim Cramer and the AAP team offer up stocks that will allow you to play it safe amid crazy politics.

In terms of volume, Johnson & Johnson (JNJ) and Sanofi (SNY) were among the most actively traded stocks midmorning but were both only down less than a percent.

Exact Sciences (EXAS) was trading at twice its daily volume and saw its shares fall about 6.6%, or $2.40, in early trading Wednesday to $34.19 apiece.

The Madison, Wisc.-basedmolecular diagnostics company said after markets closed on Tuesday that its underwriters, includingJefferies LLC, BofA Merrill Lynch and Robert W. Baird & Co. hadacuired7 million shares of commons stock with an option to buy about 1 million more.

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Biotech Movers: Shire Falls on Stem Cell Program Transfer; Calithera, Synergy Up on FDA Approvals - TheStreet.com

Bone Marrow Stem Cells Comments Off on Biotech Movers: Shire Falls on Stem Cell Program Transfer; Calithera, Synergy Up on FDA Approvals – TheStreet.com | June 7th, 2017

Dr Bishesh Poudyal of the Civil Service Hospital in Kathmandu is the doctor who carried out all 18 transplants. At Civil the cost per transplant is between Rs 400,000 to Rs 500,000. KATHMANDU, June 7:A total of 18 bone marrow transplants have been successfully carried out in Nepal by a single doctor in Kathmandu since 2012.

A bone marrow transplant is a medical procedure performed to replace bone marrow that has been damaged or destroyed by disease, viral infection, or chemotherapy. This procedure involves transplanting blood stem cells, which travel to the bone marrow where they produce new blood cells and promote growth of new marrow.

Bone marrow is the spongy, fatty tissue inside the bones. It creates the red blood cells that carry oxygen and nutrients throughout the body, white blood cells that fight infection, and platelets that are responsible for the formation of clots.

Dr Bishesh Poudyal of the Civil Service Hospital in Kathmandu is the doctor who carried out all 18 transplants. "I am going to carry out bone marrow transplants on another six patients in near future," said Poudyal, who was born at Jawalakhel of Lalitpur.

Dr Poudyal, who passed SLC 24 years ago from Adarsha Vidya Mandir, was inspired by his father to pursue studies in hematology and bone marrow transplant. After completing his MBBS from China and MD from India under government scholarships, he started working at the Bir Hospital. "I served there for two years at Bir Hospital as per the government rule for scholarship students," he said.

Then, Dr Poudyal left the Bir Hospital as he came to know that bone marrow transplant was not possible at Bir and joined Civil Service Hospital. He also practised at the Nobel Medical College Hospital at Sinamangal where he started bone marrow transplant in 2012. "As I came to know Nobel was charging patients between Rs 800,000 to Rs 1 million per transplant, I quit the hospital," he said.

At his initiation, the Civil Hospital started bone marrow transplant about a year ago. At Civil the cost per transplant is between Rs 400,000 to Rs 500,000. The transplant recepients ranged from 22 years old to 64 years. Two patients died after about nine months of transplant. "One died of tuberculosis infection and another died of disease complications," according to Dr Poudyal.

"Bone marrow is transplanted in cancer and other blood diseases. Bone marrow is transplanted in different ways-- by treating patients' bone marrow, using siblings' and parents' bone marrow and matched unrelated donor (MUD). "We have not transplanted bone marrow under MUD category," said Dr Poudyal. "MUD is a condition of matching gene with other persons. A person's genes match those of only one percent of the population of the entire world," he added.

There is no actual data of patients with bone marrow problems in the country. However, 400 to 600 patients visit Civil Service Hospital for treatment of acute lukemia and other blood cancer cases per year. "Forty to 50 percent patients of blood cancer recover fully while the recovery rate among bone marrow recepients is 70-80 percent," said Dr Poudyal.

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Nepal's sole bone marrow transplant doctor - Republica

Bone Marrow Stem Cells Comments Off on Nepal’s sole bone marrow transplant doctor – Republica | June 6th, 2017

Vancouver surgeon and UBC professorRonald Lett is appealing tothe public forhelp in finding a bone marrow transplant for his wife Elizabeth Nega, who has an aggressive form of leukemia.

Nega, better known as Elsa, discovered that she had acute lymphoblasticleukemia in February and urgently needs a bone marrow transplant.However, the Ethiopian Canadian wife and mother of two has been unable to find a match because of the low number of African donors.

Ronald and Elsa are now reaching out to people of African descent to register as bone marrowdonors. They've started a website, match4elsa.com, as well as Facebook and Twitter accounts, to find Elsa and other African-Canadians life saving transplants.

"I love to live. I want to be with my kids. I want to smile again. I want to play with them again. If you save my life, you will save my whole family," said Elsa Nega in her video appeal for a donor.

Lett is the founder and international director of the charity, Canadian Network for International Surgery(CNIS). He met Elsa in Ethiopia while he was there training local doctors to perform essential surgeries.

After dedicating his life to helping others, Lett says being unable to help his wife in her time of need has been difficult.

"I helplessly watch as the love of my life suffers terribly, has devastating complications from her treatmentbut has no promise of a cure," said Lett.

"Transplant, which only works half the time, is our only hopeand all the news concerning a match for Elsahas been bad too."

Elizabeth Nega, Ronald Lett and their two children are running out of time to find Elsa a bone marrow donor. (Helen Goddard)

Since discovering that she had leukemia, Elsahas beenput through several rounds of chemotherapy, but after failing to go into remission, obtaining stem cells from a bone marrow transplant has become her only hope of recovery.

Her brother and sister in Ethiopia were her best chance, but neither were a match.

The larger issue in finding a donor for Elsa is the lack of diversity in the donor registry.

Of the 405,000 Canadians on the stem cell registry, only 800 have an African background, and none are a match for Elsa, according toChrisvan Doornwith the One Match Program.

Even among the 29 million people on the international registry, no match has been found.

Lett and Elsa's children, Lana, 8, and Lawrence, 6, have contributed to the effort.

They're in a video reading a letter appealing to Ethiopians around the world, including Canadian-Ethiopian R & B singerThe Weeknd, asking for help to save their mom.

In the meantime, Elsa's health is declining, and she's hoping for a miracle, even if it's not for her.

"If they save somebody, that's like a lotteryor a big blessing, you know.It's a big chance to get somebody to match to you and save your life.You know many people can't do this." saidNega.

People interested in registering to be a bone marrow donor can register at blood.ca,must be between 17 and 35 years old and in good health.

The test involves a cheek swab at the nearest clinicor a kit can be mailed out.

Continue reading here:
Vancouver woman's family pleading for help finding a bone marrow donor - CBC.ca

Bone Marrow Stem Cells Comments Off on Vancouver woman’s family pleading for help finding a bone marrow donor – CBC.ca | June 6th, 2017

TAMPA Researchers at the University of South Florida show in a new study that bone marrow stem cell transplants helped improve motor functions and nervous system conditions in mice with the disease amyotrophic lateral sclerosis (ALS) by repairing damage to the blood-spinal cord barrier.

In a study recently published in the journal Scientific Reports, researchers in USFs Center of Excellence for Aging and Brain Repair say the results of their experiment are an early step in pursuing stem cells for potential repair of the blood-spinal cord barrier, which has been identified as key in the development of ALS.

USF Health Professor Svitlana Garbuzova-Davis, PhD, led the project.

Using stem cells harvested from human bone marrow, researchers transplanted cells into mice modeling ALS and already showing disease symptoms. The transplanted stem cells differentiated and attached to vascular walls of many capillaries, beginning the process of blood-spinal cord barrier repair.

The stem cell treatment delayed the progression of the disease and led to improved motor function in the mice, as well as increased motor neuron cell survival, the study reported.

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Mice with ALS improve with stem cell therapy - The Ledger

Bone Marrow Stem Cells Comments Off on Mice with ALS improve with stem cell therapy – The Ledger | June 6th, 2017

Paul Spagnuolo is working on creating a drug with an avocado compound that targets cancer cells. (Paul Spagnuolo)

A Guelph food science researcher is getting $100,000 from the Ontario Institute for Cancer Research to fund investigations into using an avocado compound as a possible treatment for leukemia.

Paul Spagnuolo discovered that Avocatin B, a compound mainly found in avocado pits can kill leukemia stem cells in 2015.

"Getting funds to do any type of research is a reason to celebrate," said Spagnuolo told CBC News.

The funding will further his research by allowing his lab to use better equipment and collaborate with cancer researchers from the University of Toronto, Princess Margaret Cancer Centre, Ottawa University and McMaster University.

Spagnuolo's lab tested more than 800 natural compounds for their ability to kill leukemia stem cells and discovered Avocatin B was the most potent and only targetedcancer cells.

Avocatin B kills leukemia stem cells by stopping fatty acid oxidation in the cells, a process necessary for the cancer cell to digest fat as a fuel source in order to live and grow.

"Our cells can utilize glucose primarily and some other parts, but leukemia cells are rewired so that if you inhibit the oxidation process, they will die," he said.

Spagnulo and his lab are now looking to develop a way to detect whether or not Avocatin B is circulating in the blood and bone marrow.

Leukemia cells live in the bloodstream or bone marrow, so it's important for the drug to make it to those parts to kill the cancer cells.

"We want to be able to detect our drug inside the blood so that we can understand how we can formulate products better to get our product into the blood," said Spagnuolo.

Moving forward, Spagnuolo's lab will have to report to OICR quarterly, it's a condition of the funding which is spread over two years and has the possibility of renewal for another two years.

"(It's) a lot more intense than I anticipated, but I think the key here is it's very results oriented," said Spagnuolo, "There's no complacency here."

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Pitting avocados against leukemia stem cells - CBC.ca

Bone Marrow Stem Cells Comments Off on Pitting avocados against leukemia stem cells – CBC.ca | June 6th, 2017

Multiple myeloma is cancer that involves our bone marrow with a specific cell called a plasma cell that patients can develop. Most patients will need a bone marrow transplant.

Patients needing bone marrow transplants dont have to travel far to receive this potentially life-saving transplant.

The actual day of the diagnosis was November 18th of 2015 and it was a diagnosis for multiple myeloma, said Steven Simpson.

Simpson was ready to fight from that day on. He learned from Dr. Kelly McCaul, the director of Avera Hematology Transplant Program, that he would need a bone marrow transplant.

There are many different types of transplant that we do. Theres basically an autologous transplant where patients would be their own donors for their stem cells and then theres allogenic transplant which are some sort of donor process. And so Steve has multiple myeloma. We would normally look at autologous transplant as the preferred pathway for patients with that disease, said Dr. McCaul.

Weve never had to leave anywhere other than here. This is it, said Simpson.

Simpson and his immediate family live no further than 20 minutes away from Avera McKenna so getting the transplant elsewhere was out of the question. But that didnt come without resistance from his insurance company.

Youre asking somebody to go three or four hours out of the way minimum for a period of time that could last anywhere from a week to whatever the process is. You lose your doctors. You lose the ability to have any local family support there as you need them and you dont really know what youre getting into. You just know what youre told, said Simpson.

Simpson and his insurance company worked together and was able to stay at Avera for his transplant.

I came in the day before scheduled for the transplant but left three hours after the transplant because I didnt have any reactions. Plus, we all knew that I had somebody available to watch me 24/7 for the period of time that we would have. The fact that you have your doctors here, your oncologist, your lab people, your nursing staff, everybodys here. They know who you are, said Simpson.

17 years ago when I first looked at this program one of the big things I looked at was the need in the community and it was felt from my perspective, and obviously Avera, that our need in the community was high. And it allows patients to stay within the community, close to family members, without having to drive four, five hours away, said Dr. McCaul.

Today, Simpson is well on his way to feeling like his old self, something he credits to staying close to home for his transplant.

For more information just call 877-AT-AVERA

Originally posted here:
Bone marrow transplant patient credits positive recovery to staying close to home - KSFY

Bone Marrow Stem Cells Comments Off on Bone marrow transplant patient credits positive recovery to staying close to home – KSFY | June 5th, 2017

As a third grade teacher at Ottawa Elementary School, Kelly Gianotti teaches students many important life lessons along with reading and math.

The most important lesson she has instructed was taught by example: how to save the life of a blood cancer patient.

Gianotti donated her stem cells in 2013 to help save the life of a blood cancer patient. The patient was in need of a bone marrow stem cell transplant and had no donor match in her family.

I had seen a flier at a local gym for a high school student who was looking for a match. That intrigued me. I went online to register, Gianotti said.

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A year later Gianotti learned she was a potential match, but not for the high school patient. She went through more testing and did the outpatient donation procedure.

Gianotti later found out her donation assisted MaryAnn Hastings, who lived near Boston, Mass. The two chatted via e-mail and were able to meet in 2016, when Gianotti traveled to Boston.

The lady I donated for died last February of a different type of cancer. I wanted to honor her and spread the word, Gianotti said, adding that Hastings family indicated she was able to give Hastings three extra years of life with her donation.

The donation experience motivated Gianotti to host the first DKMS bone marrow registration drive through Chippewa Valley Schools district. DKMS is an international nonprofit organization dedicated to the fight against blood cancer and blood disorders, according to its website.

The goal of the drive is to help register potential donors. It will be held Tuesday from 4 p.m. to 9 p.m. at Cheyenne Elementary School in Macomb Township. Gianotti said she hopes to register between 100 and 200 potential donors.

Requirements to join the bone marrow registry are that the donor be in good health and between the ages of 18 and 55. The process involves filling out a form, understanding the donation methods and swabbing the inside of each cheek for 30 seconds with a cotton swab. Donors swab their cheeks in a circular motion.

There is no cost to register, although donations are accepted. The donations assist DKMS in covering the $65 registration processing fee.

According to DKMS, 70 percent of people suffering from blood-related illnesses rely on donors other than their families.

If selected as a match for a patient, there are two different methods of donation, according to the DKMS website.

According to the DKMS website, a donation method used in about 25 percent of cases is a one or two hour surgical procedure performed under anesthesia to collect marrow cells from the back of the pelvic bone using a syringe.

To obtain more information about the drive or to make a monetary donation, visit fb.com/cvsgetsswabbed. Those who want to join the bone marrow registry but are unable to attend the June 6 drive can register at dkms.org.

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Chippewa Valley Schools hosts bone marrow registration drive - The Macomb Daily

Bone Marrow Stem Cells Comments Off on Chippewa Valley Schools hosts bone marrow registration drive – The Macomb Daily | June 5th, 2017

COUNTLESS lives across the world could be saved by an Oxfordshire familys appeal to find a bone marrow donor for their little boy.

Two-year-old Alastair Ally Kim has Chronic Granulomatous Disorder (CGD), a life-threatening condition.

He has now become the fourth person in the world to start an experimental gene therapy course at Great Ormond Street Hospital.

In the meantime, his parents have spearheaded 200 international donor drives to find their son a match, signing up 7,000 would-be donors in the process - some of whom have since been matched with other patients.

Father Andrew Kim, 37, of Hinton Waldrist near Longworth, said: We want to use whatever momentum Allys story has to help someone else. We know that matches have come through our drives for other people. Its awesome that someone will benefit from all this.

On Thursday, May 25 family friend Cathy Oliveira organised a drive at the Oxford Universitys Old Road research building, signing up 80 staff members in a day.

Ms Oliveira said: When everything happened with Ally I wanted to show support in any way we could; this is directly beneficial not just for Ally but for others.

Allys CGD means his immune system is compromised and the tiniest infection could leave him seriously ill.

His only chance of a permanent cure is a bone marrow stem cell donation, with a match likely to be of Korean or East Asian origin.

In April the youngster and mum Judy Kim, 36, an Oxford University researcher, travelled to London for him to begin a pioneering new gene therapy treatment.

After a week of chemotherapy to wipe out Allys immune system, cells taken from him are modified in a lab and re-introduced to correct the disorder.

Mr Kim said: Bone marrow would give him back 100 per cent functionality and gene therapy is 10 to 15 per cent; its enough to live in the real world, and not be scared he will die every time he gets an infection.

It has been a roller-coaster of a year, but theres nothing to do but move forward. We are really excited at the thought of him being able to come home this summer.

Blood cancer charity DKMS supported last weeks donor drive in Oxford.

Senior donor recruitment manager Joe Hallet said: Around 30 per cent of patients in need of a blood stem cell donor will find a matching donor within their own family.

The remaining 70 per cent, like Ally, will need to find an unrelated donor to have a second chance of life, so events like these are crucial.

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Oxford University staff join bone marrow stem cell donor drive for Oxford toddler Ally Kim - Witney Gazette

Bone Marrow Stem Cells Comments Off on Oxford University staff join bone marrow stem cell donor drive for Oxford toddler Ally Kim – Witney Gazette | June 3rd, 2017

(CNN) Here is some background information about stem cells.

Scientists believe that stem cell research can be used to treat medical conditions including Parkinsons disease, spinal cord injury, stroke, burns, heart disease, diabetes, osteoarthritis and rheumatoid arthritis.

About Stem Cells:Stem cell research focuses on embryonic stem cells and adult stem cells.

Stem cells have two characteristics that differentiate them from other types of cells:- Stem cells are unspecialized cells that replicate themselves for long periods through cell division.- Under certain physiologic or experimental conditions, stem cells can be induced to become mature cells with special functions such as the beating cells of the heart muscle or insulin-producing cells of the pancreas.

There are four classes of stem cells: totipotent, multipotent, pluripotent, and unipotent.- Totipotent stem cells that develop into cells that make up all the cells in an embryo and fetus. (Ex: The zygote/fertilized egg and the cells at the very early stages following fertilization are considered totipotent)- Multipotent stem cells can give rise to multiple types of cells, but all within a particular tissue, organ, or physiological system. (Ex: blood-forming stem cells/bone marrow cells, most often referred to as adult stem cells)- Pluripotent stem cells (ex: embryonic stem cells) can give rise to any type of cell in the body. These cells are like blank slates, and they have the potential to turn into any type of cell.- Unipotent stem cells can self-renew as well as give rise to a single mature cell type. (Ex: sperm producing cells)

Embryonic stem cells are harvested from four to six-day-old embryos. These embryos are either leftover embryos in fertility clinics or embryos created specifically for harvesting stem cells by therapeutic cloning. Only South Korean scientists claim to have successfully created human embryos via therapeutic cloning and have harvested stem cells from them.

Adult stem cells are already designated for a certain organ or tissue. Some adult stem cells can be coaxed into or be reprogrammed into turning into a different type of specialized cell within the tissue type for example, a heart stem cell can give rise to a functional heart muscle cell, but it is still unclear whether they can give rise to all different cell types of the body.

The primary role of adult stem cells is to maintain and repair the tissue in which they are found.

Uses of Stem Cell Research:Regenerative (reparative) medicine uses cell-based therapies to treat disease.

Scientists who research stem cells are trying to identify how undifferentiated stem cells become differentiated as serious medical conditions, such as cancer and birth defects, are due to abnormal cell division and differentiation.

Scientists believe stem cells can be used to generate cells and tissues that could be used for cell-based therapies as the need for donated organs and tissues outweighs the supply.

Stem cells, directed to differentiate into specific cell types, offer the possibility of a renewable source of replacement cells and tissues to treat diseases, including Parkinsons and Alzheimers diseases, spinal cord injury, stroke, burns, heart disease, diabetes, osteoarthritis, and rheumatoid arthritis.

Policy Debate:Cloning human embryos for stem cells is very controversial.

The goal of therapeutic cloning research is not to make babies, but to make embryonic stem cells, which can be harvested and used for cell-based therapies.

Using fertilized eggs left over at fertility clinics is also controversial because removing the stem cells destroys them.

Questions of ethics arise because embryos are destroyed as the cells are extracted, such as: When does human life begin? What is the moral status of the human embryo?

Timeline:1998 President Bill Clinton requests a National Bioethics Advisory Commission to study the question of stem cell research.

1999 The National Bioethics Advisory Commission recommends that the government allow federal funds to be used to support research on human embryonic stem cells.

2000 During his campaign, George W. Bush says he opposes any research that involves the destruction of embryos.

2000 The National Institutes of Health (NIH) issues guidelines for the use of embryonic stem cells in research, specifying that scientists receiving federal funds can use only extra embryos that would otherwise be discarded. President Clinton approves federal funding for stem cell research but Congress does not fund it.

August 9, 2001 President Bush announces he will allow federal funding for about 60 existing stem cell lines created before this date.

January 18, 2002 A panel of experts at the National Academy of Sciences (NAS) recommends a complete ban on human reproductive cloning, but supports so-called therapeutic cloning for medical purposes.

February 27, 2002 For the second time in two years, the House passes a ban on all cloning of human embryos.

July 11, 2002 The Presidents Council on Bioethics recommends a four-year ban on cloning for medical research to allow time for debate.

February 2005 South Korean scientist Hwang Woo Suk publishes a study in Science announcing he has successfully created stem cell lines using therapeutic cloning.

December 2005 Experts from Seoul National University Hwang of faking some of his research. Hwang asks to have his paper withdrawn while his work is being investigated and resigns his post.

January 10, 2006 An investigative panel from Seoul National University accuses Hwang of faking his research.

July 18, 2006 The Senate votes 63-37 to loosen President Bushs limits on federal funding for embryonic stem-cell research.

July 19, 2006 President Bush vetoes the embryonic stem-cell research bill passed by the Senate (the Stem Cell Research Enhancement Act of 2005), his first veto since taking office.

June 20, 2007 President Bush vetoes the Stem Cell Research Enhancement Act of 2007, his third veto of his presidency.

January 23, 2009 The FDA approves a request from Geron Corp. to test embryonic stem cells on eight to 10 patients with severe spinal cord injuries. This will be the worlds first test in humans of a therapy derived from human embryonic stem cells. The tests will use stem cells cultured from embryos left over in fertility clinics.

March 9, 2009 President Barack Obama signs an executive order overturning an order signed by President Bush in August 2001 that barred the NIH from funding research on embryonic stem cells beyond using 60 cell lines that existed at that time.

August 23, 2010 US District Judge Royce C. Lamberth issues a preliminary injunction that prohibits the federal funding of embryonic stem cell research.

September 9, 2010 A three-judge panel of the US Court of Appeals for the D.C. Circuit grants a request from the Justice Department to lift a temporary injunction that blocked federal funding of stem cell research.

September 28, 2010 The US Court of Appeals for the District of Columbia Circuit lifts an injunction imposed by a federal judge, thereby allowing federally funded embryonic stem-cell research to continue while the Obama Administration appeals the judges original ruling against use of public funds in such research.

October 8, 2010 The first human is injected with cells from human embryonic stem cells in a clinical trial sponsored by Geron Corp.

November 22, 2010 William Caldwell, CEO of Advanced Cell Technology, tells CNN that the FDA has granted approval for his company to start a clinical trial using cells grown from human embryonic stem cells. The treatment will be for an inherited degenerative eye disease.

April 29, 2011 The US Court of Appeals for the District of Columbia lifts an injunction, imposed last year by a federal judge, banning the Obama administration from funding embryonic stem-cell research.

May 11, 2011 Stem cell therapy in sports medicine is spotlighted after New York Yankee pitcher Bartolo Colon is revealed to have had fat and bone marrow stem cells injected into his injured elbow and shoulder while in the Dominican Republic.

July 27, 2011 Judge Lamberth dismisses a lawsuit that tried to block funding of stem cell research on human embryos.

February 13, 2012 Early research published by scientists at Cedars-Sinai Medical Center and Johns Hopkins University show that a patients own stem cells can be used to regenerate heart tissue and help undo damage caused by a heart attack. It is the first instance of therapeutic regeneration.

May 2013 Scientists make the first embryonic stem cell from human skin cells by reprogramming human skin cells back to their embryonic state, according to a study published in the journal, Cell.

April 2014 For the first time scientists are able to use cloning technologies to generate stem cells that are genetically matched to adult patients,according to a study published in the journal, Cell Stem Cell.

October 2014 Researchers say that human embryonic stem cells have restored the sight of several nearly blind patients and that their latest study shows the cells are safe to use long-term. According to a report published in The Lancet, the researchers transplanted stem cells into 18 patients with severe vision loss as a result of two types of macular degeneration.

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Stem Cells Fast Facts | KABC-AM - KABC

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Miami Herald

Want to save a life? Cuban American searches for bone marrow donor Miami Herald According to Gift of Life, a nonprofit, Boca Raton-based bone marrow and blood stem cell registry, 55 percent of Hispanic cancer patients and 75 percent of multiracial patients are never matched, some dying while waiting to get a transplant. The data ...

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Want to save a life? Cuban American searches for bone marrow donor - Miami Herald

Bone Marrow Stem Cells Comments Off on Want to save a life? Cuban American searches for bone marrow donor – Miami Herald | June 3rd, 2017

COUNTLESS lives across the world could be saved by an Oxfordshire familys appeal to find a bone marrow donor for their little boy.

Two-year-old Alastair Ally Kim has Chronic Granulomatous Disorder (CGD), a life-threatening condition.

He has now become the fourth person in the world to start an experimental gene therapy course at Great Ormond Street Hospital.

In the meantime, his parents have spearheaded 200 international donor drives to find their son a match, signing up 7,000 would-be donors in the process - some of whom have since been matched with other patients.

Father Andrew Kim, 37, of Hinton Waldrist near Longworth, said: We want to use whatever momentum Allys story has to help someone else. We know that matches have come through our drives for other people. Its awesome that someone will benefit from all this.

On Thursday, May 25 family friend Cathy Oliveira organised a drive at the Oxford Universitys Old Road research building, signing up 80 staff members in a day.

Ms Oliveira said: When everything happened with Ally I wanted to show support in any way we could; this is directly beneficial not just for Ally but for others.

Allys CGD means his immune system is compromised and the tiniest infection could leave him seriously ill.

His only chance of a permanent cure is a bone marrow stem cell donation, with a match likely to be of Korean or East Asian origin.

In April the youngster and mum Judy Kim, 36, an Oxford University researcher, travelled to London for him to begin a pioneering new gene therapy treatment.

After a week of chemotherapy to wipe out Allys immune system, cells taken from him are modified in a lab and re-introduced to correct the disorder.

Mr Kim said: Bone marrow would give him back 100 per cent functionality and gene therapy is 10 to 15 per cent; its enough to live in the real world, and not be scared he will die every time he gets an infection.

It has been a roller-coaster of a year, but theres nothing to do but move forward. We are really excited at the thought of him being able to come home this summer.

Blood cancer charity DKMS supported last weeks donor drive in Oxford.

Senior donor recruitment manager Joe Hallet said: Around 30 per cent of patients in need of a blood stem cell donor will find a matching donor within their own family.

The remaining 70 per cent, like Ally, will need to find an unrelated donor to have a second chance of life, so events like these are crucial.

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Oxford University staff join bone marrow stem cell donor drive for ... - Oxford Mail

Bone Marrow Stem Cells Comments Off on Oxford University staff join bone marrow stem cell donor drive for … – Oxford Mail | June 2nd, 2017

Blood stem cells are things of wonder: hidden inside each single cell is the power to reconstitute an entire blood system, like a sort of biological big bang.

Yet with great power comes greater vulnerability. Once these master cells are compromised, as in the case of leukemia and other blood disorders, treatment options are severely limited.

A bone marrow transplant is often the only chance for survival. The surgery takes a healthy donors marrowrich with blood stem cellsand reboots the patients blood system. Unfortunately, like organ transplants, finding a matching donor places a chokehold on the entire process.

According to Dr. George Daley at Harvard Medical School, a healthy sibling gives you a one in four chance. A stranger? One in a million.

For 20 years, scientists have been trying to find a way to beat the odds. Now, two studies published in Nature suggest they may be tantalizingly close to being able to make a limitless supply of blood stem cells, using the patients own healthy tissues.

"This step opens up an opportunity to take cells from patients with genetic blood disorders, use gene editing to correct their genetic defect and make functional blood cells," without depending on donors, says Dr. Ryohichi Sugimura at Boston Childrens Hospital, who authored one of the studies with Daley.

Using a magical mix of seven proteins called transcription factors, the team coaxed lab-made human stem cells into primordial blood cells that replenished themselves and all components of blood.

A second study led by Dr. Shahin Rafii, a stem cell scientist at Weill Cornell Medical College took a more direct route, turning mature cells from mice straight into genuine blood stem cells indiscernible from their natural counterparts.

This is the first time researchers have checked all the boxes and made blood stem cells, says Dr. Mick Bhatia at McMaster University, who was not involved in either study, That is the holy grail.

The life of a blood stem cell starts as a special cell nestled on the walls of a large blood vesselthe dorsal aorta.

Under the guidance of chemical signals, these cells metamorphose into immature baby blood stem cells, like caterpillars transforming into butterflies. The exact conditions that prompt this birthing process are still unclear and is one of the reasons why lab-grown blood stem cells have been so hard to make.

These baby blood stem cells dont yet have the full capacity to reboot blood systems. To fully mature, they have to learn to respond to all sorts of commands in their environment, like toddlers making sense of the world.

Some scientists liken this learning process to going to school, where different external cues act as textbooks to train baby blood stem cells to correctly respond to the body.

For example, when should they divide and multiply? When should they give up their stem-ness, instead transforming into oxygen-carrying red blood cells or white blood cells, the immune defenders?

Both new studies took aim at cracking the elusive curriculum.

In the first study, Daley and team started with human skin and other cells that have been transformed back into stem cells (dubbed iPSCs, or induced pluripotent stem cells). Although iPSCs theoretically have the ability to turn into any cell type, no one has previously managed to transform them into blood stem cells.

A lot of people have become jaded, saying that these cells dont exist in nature and you cant just push them into becoming anything else, says Bhatia.

All cells in an organism share the same genes. However, for any given cell only a subset of genes are turned into proteins. This process is what gives cells their identitiesmay it be a heart cell, liver cell, or blood stem cell.

Daley and team focused on a family of transcription factors. Similar to light switches, these proteins can flip genes on or off. By studying how blood vessels normally give birth to blood stem cells, they found seven factors that encouraged iPSCs to grow into immature blood stem cells.

Using a virus, the team inserted these factors into their iPSCs and injected the transformed cells into the bone marrow of mice. These mice had been irradiated to kill off their own blood stem cells to make room for the lab-grown human replacements.

In this way, Daley exposed the immature cells to signals in a blood stem cells normal environment. The bone marrow acts like a school, explains Drs. Carolina Guibentif and Berthold Gttgens at the University of Cambridge, who are not involved in the study.

It worked. In just twelve weeks, the lab-made blood stem cells had fully matured into master cells capable of making the entire range of cells normally found in human blood. Whats more, when scientists took these cells out and transplanted them into a second recipient, they retained their power.

This a major step forward compared with previous methods, says Guibentif.

In contrast, the second study took a more direct route. Rafii and team took cells lining a mouses vessels, based on the finding that these cells normally turn into blood stem cells during development.

With a set of four transcription factors, the team directly reprogrammed them into baby blood stem cells, bypassing the iPSC stage.

These factors act like a maternity ward, allowing the blood stem cells to be born, says Guibentif.

To grow them to adulthood, Rafii and team laid the cells onto a blanket of supporting cells that mimics the blood vessel nursery. Under the guidance of molecular cues secreted by these supporting cells, the blood stem cells multiplied and matured.

When transplanted into short-lived mice without a functional immune system, the cells sprung to action. In 20 weeks, the mice generated an active immune response when given a vaccine. Whats more, they went on to live a healthy 1.5 yearsroughly equivalent to 60 years old for a human.

Rafii is especially excited about using his system to finally crack the stem cell learning curriculum.

If we can figure out the factors that coax stem cells to divide and mature, we may be able to unravel the secrets of their longevity and make full-fledged blood stem cells in a dish, he says.

Calling both experiments a breakthrough, Guibentif says, this is something people have been trying to achieve for a long time.

However, she points out that both studies have caveats. A big one is cancer. The transcription factors that turn mature cells into stem cells endow them with the ability to multiply efficientlya hallmark of cancerous cells. Whats more, the virus used to insert the factors into cells may also inadvertently turn on cancer-causing genes.

That said, neither team found evidence of increased risk of blood cancers. Guibentif also acknowledges that future studies could use CRISPR in place of transcription factors to transform cells into blood stem cells on demand, further lowering the risk.

The techniques will also have to be made more efficient to make lab-grown blood stem cells cost efficient. Itll be years until human use, says Guibentif.

Even so, the studies deter even the most cynical of critics.

After 20 years, were finally tantalizingly close to generating bona fide human blood stem cells in a dish,"says Daley.

Image Credit: Pond5

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Limitless Lab-Grown Blood Is 'Tantalizingly Close' After 20 Years - Singularity Hub

Bone Marrow Stem Cells Comments Off on Limitless Lab-Grown Blood Is ‘Tantalizingly Close’ After 20 Years – Singularity Hub | June 2nd, 2017

Two separate research teams have succeeded in generating blood stem cells using completely different procedures. One team was led by stem cell biologist Dr. George Q. Daley of Harvard Medical School and Boston Childrens Hospital. The other team was spearheaded by Dr. Shahin Rafii of the Weill Cornell Medicines Ansary Stem Cell Institute in New York.

In both cases, reprogrammed blood stem cells were able to successfully produce blood cells when implanted into mice. And if either or both procedures turn out to be viable for humans, a future where blood donors will no longer be needed may soon be in the horizon because science has provided us with a way to produce unlimited blood supply.

Stem cells are specially programmed cells that are responsible for creating all of the bodys other cells. There are two types of stem cells embryonic and adult. Embryonic stem cells are located you guessed it in the embryo where they stay before they start to specialise. Adult stem cells are the ones used to repair and replace worn out or old cells.

Those are the natural types. Theres another type, though. Theyre called induced pluripotent stem cells (iPS cells for short). Unlike the first two types, iPS cells arent naturally present. Theyre actually adult stem cells that were converted back to their primitive state, which means they can be coaxed to turn into any type of cell.

Dr. Daley and his team chose to use both embryonic stem cells and iPS cells for their research. Using a combination of proteins, they coaxed the cells to turn into hemogenic endothelium a kind of embryonic tissue that eventually turns into blood stem cells. Next, they tested several transcription factors genes that tell other genes what to do until they came up with the combination (specifically: ERG, HOXA5, HOXA9, HOXA10, LCOR, RUNX1, and SPI1) that pushed the hemogenic endothelium into a blood-forming or blood stem cell state. They then injected those modified cells into the bone marrow of their mice subjects. After several weeks, portions of the mices blood and bone marrow developed different types of blood cells, including red blood cells, white blood cells, and even immune cells.

As Daley described the feat: Were tantalizingly close to generating bona fide human blood stem cells in a dish.

On the other hand, Rafii and his team chose a different route. They didnt make use of iPS cells. Instead, they created true blood stem cells, starting off by extracting stem cells from the blood vessel lining of mature mice. Next, they inserted transcription factors (Fosb, Gfi1, Runx1, and Spi1) into the genomes of the extracted cells, then kept these cells in Petri dishes designed to replicate the environment within human blood vessels.

Over time, the cells turned into blood stem cells and multiplied. They then injected those stem cells into mice treated with radiation (which meant most of their blood and immune cells were gone). The stem cells regenerated not just the blood, but the immune cells too. Consequently, the mice recovered and went on to live for over 1.5 years in the lab.

As described by Rafii, the procedure they used is similar to a direct aeroplane flight, while Daleys is like a flight that took a detour prior to reaching its ultimate destination. Doing away with the iPS part kind of makes Rafiis method slightly better than Daleys because it minimizes the threat of tumors forming or the body rejecting the stem cells, which is a typical reaction that iPS cells might cause. But if Daleys team is able to refine their process to eliminate this risk, then that will level the playing field, so to speak.

Whatever happens from here on, both procedures are nonetheless considered significant breakthroughs. And even though its not yet certain which method will turn out to be the better one for humans, whats clear is that both methods have the potential to be game-changers when it comes to any kind of treatment involving blood infusion and transfusion.

Both studies have been published in the journal Nature, with Daleys under the title Haematopoietic stem and progenitor cells from human pluripotent stem cells and Rafiis under the title Conversion of adult endothelium to immunocompetent haematopoietic stem cells.

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Scientists Close to Generating Unlimited Blood Supply from Stem Cells - Wall Street Pit

Bone Marrow Stem Cells Comments Off on Scientists Close to Generating Unlimited Blood Supply from Stem Cells – Wall Street Pit | May 31st, 2017