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Archive for the ‘Bone Marrow Stem Cells’ Category

Peptide aimed at stem cell genesis debuts on supplement market – NutraIngredients-usa.com

A longtime product developer is bringing a peptide ingredient to the US market that has been researched for a unique property promoting the growth of bone marrow stem cells.

Called DH Stemogen, the product is the brainchild of Dr Marvin Heuer MD who has a history of product development with sports nutrition company MuscleTech. Dr. Heuer has a background in clinical research, having spent many years in drug development at Glaxo Smith Kline. He also runs a contract research firm, Heuer M.D. Research Inc. and is the CEO of omega-3 supplement manufacturer Blue Ocean Nutrascience.

The new product, called DH Stemogen is based on a Cyclo-{L-ALA-L-GLU(TRP-OH) peptide that was developed by a Russian biochemist.

Its a peptide that is a mimic of a naturally occurring thymic peptide,Dr. Heuer told NutraIngredients-USA. Heuer was promoting the launch of the product at the recent Expo West trade show in Anaheim, CA. At Heuer M.D. Research, as a company we are out looking for novel ingredientsto bring out, hopefully in the nutraceutical area.

We got interested in Prof. Vlad Deigins peptide research, Dr. Heuer said (Deigin is associated with the Institute of Bioorganic Chemistry at the Russian Academy of Sciences in Moscow.)We looked at this particular compound that he was launching as an ingredient in Russia about a year ago.

The peptide in DH Stemogen targets a particular type of stem cell hematopoietic cells (HSC). Stem cells in general are the building blocks of our bodies. These cells are able to transform themselves into almost any type of cell. There are various sources of stem cells in an adult body. One of the most important of them comprises the bone marrow, where the HSCs are produced. HSCs transform into all the main cell types in our blood, including red blood cells and white blood cells. Dr. Heuer said there is some evidence that those cells are able to reconstruct other body tissues by transforming into the specific tissue type cell such as liver, nervous tissue, kidney and skin.

These properties would seem to make Stemogen a natural for a healthy aging product positioning, Dr. Heuer said. But Deigins research, trending as it does over into disease endpoints, is a little problematical when it comes to supporting US-style structure function claims, he admitted. Other countries dont make the same hard and fast distinctions between dietary ingredients meant for supplement applications and active pharmaceutical agents meant for drugs, he said.

We are going to be very cautious about making structure/function claims,Dr. Heuer said. The product at the moment saysSupport your immune system and Support healthy levels of stem cells in your blood.

We are about to begin a whole profile of research in the U.S. and Canada, he added.

Dr. Heuer said one thing thats unique about the ingredient (and something that he says Deigin has patented) is a structural twist that improves the peptides stability. The criticism of some other novel peptides has been that interesting as their properties might be, once they hit the stomachs gastric fluid they blow apart into their constituent amino groups and all those novel properties are lost.

He has a patent on the way he makes this with a hex ring on the end that protects it in the GI tract and allows it to be absorbed,he said.

Bringing a synthetic analogue of a naturally occurring peptide to market as a dietary ingredient would seem to pose significant regulatory challenges. Dr. Heuer said hes confident there is a way through that thicket. The plan is to start first with a GRAS filing, and Dr. Heuer said he believes that the peptide would fall under the amino acid category in the DHSEA definitions of what constitutes a dietary ingredient.

Certainly there is a precedent of complex peptides being sold on the market, he said.

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Peptide aimed at stem cell genesis debuts on supplement market - NutraIngredients-usa.com

Adam Krief, Jewish father of 3 whose bone marrow search inspired … – Jewish Telegraphic Agency

Adam Krief, with his wife, Lia, had a rare form of blood cancer that proved to be fatal. (Facebook)

(JTA) Adam Krief, a Jewish cancer patient whose search for a bone marrow donor captured the attention of social media and celebrities including Kim Kardashian, Mayim Bialik and Jason Biggs, has died.

Krief, a father of three from Los Angeles, died Tuesday, a family friend confirmed to JTA. He was 31.

Krief was diagnosed with primary myelofibrosis, a rare form of blood cancer that is likely fatal if a stem celltransplant matchis notfound.To find anHLA, or gene complex matchfor Krief something more difficultto track downthan a blood type match drives were held around the world, including in North America,Israel, France and Mexico.

Kardashian posted about Krief on Facebook in September, saying he was a friend of a friend.

A bone-marrow donor was found last December seven matches were found, in fact, through the donor drives organized for him.

This is what cloud 9 looks like Im so grateful to let you all know that a donorhas been found, Krief wrote at the time, sharing a video with two of his children.

The Hope 4 Adam Facebook page on March 8 called for a Worldwide Unity Shabbat for March 11 and March 18 for the recovery of Krief, asking followers to Help us bring about a miracle.

On Monday, the Eretz Kabbalah Facebook page of the Los Angeles-based Eretz Cultural Center posted a call for followers to recite Tehillim, or psalms, on behalf of Krief.

After a long search for a bone-marrow match to save his life, he finally received one. However, after some complications, he is said to only have a few hours to live, the post said.

Krief is survived by his wife, Lia, and his young children.

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Jewish cancer patient finds bone marrow transplant following worldwide search, Kim Kardashians pitch

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Adam Krief, Jewish father of 3 whose bone marrow search inspired ... - Jewish Telegraphic Agency

March of the Men – The Hippocratic Post (blog)

March of the Men is a month-long campaign run by Anthony Nolan, the blood cancer charity, which seeks to recruit as many young men aged 16-30 to the register as possible.

AnthonyNolans aim to find a match for every person in need of a stem cell or bone marrow transplant. Young men aged 16-30 currently account for just 16% of the register, but provide over 50% of all potentially lifesaving transplants. This is why theMarch of the Men campaign is so important.

On average, people on the register have a 1 in 790 chance of being asked to donate stem cells or bone marrow in the next five years, whereas a man aged 16-30 has a 1 in 170 chance. As our research indicates that young men provide better outcomes for patients, its of vital importance that we encourage more of them to sign up as potential stem cell donors.

Marrow is the collective name given to AnthonyNolans student volunteer network. Operating in over 50 universities across the UK, Marrow groups campaign, fundraise and crucially recruit young students to join the stem cell register on our behalf. Incredibly, donors recruited at university by Marrow groups account for more than a quarter of all potentially lifesaving transplants that take place in the UK each year.

As the Marrow Volunteer Engagement Coordinator at AnthonyNolan, my job is to develop and deliver training workshops and presentations for our student volunteers. I also help to oversee the quality of our university recruitment events to make sure that procedures are being followed properly, and that were recruiting potential stem cell donors in the best possible way.

Recently, the AnthonyNolanResearch Institute (ANRI) carried out the largest-ever UK study into the factors that can make a stem cell transplant more successful. Early indications show the age of the donor can affect patient outcomes, with younger donors leading to better survival rates.

Marrow groups at universities have access not only to young people, but young people from a vast range of ethnic backgrounds, meaning they are of fundamental importance.

I recently had the pleasure of meeting a young man called Chris Lane at The London Clinic while he was donating his stem cells. Coincidentally, it turned out that it was me who had signed him up to the AnthonyNolanregister when I was a Marrow volunteer two years ago. He said: I had only briefly heard of AnthonyNolanbefore signing up. It wasnt until I saw a stand at university that my curiosity to find out more led me to want to be included on the register.

I had never thought about how I would feel or react to being told that I was required to donate but when it happened, I felt a sense of pride and required no second thought to follow through with whatever was required.

I think about the person who received my cells every day. Life is the best gift you could ever give someone so my fingers are always crossed for them.

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March of the Men - The Hippocratic Post (blog)

Veterinary Doctors Conduct Study Looking To Ease Arthritis Pain – CBS Philly

March 13, 2017 6:01 PM By Stephanie Stahl

PHILADELPHIA (CBS) Doctors at the University of Pennsylvania School of Veterinary Medicine are conducting a study to see if stem cell therapy will ease the pain of arthritis and the results of their research could benefit human patients as well.

Its Zoeys last check up,walking on a special mat called a forceplate to measure how much weight she puts on each leg.

It was just a year ago that putting weight on her front legs was painful.The 2-year-old Golden Retriever was diagnosed with elbow dysplasia, a condition that created arthritis in both elbows.

It is the most common cause of chronic pain in dogs, saidDr. Kimberly Agnello at Penn Vet.

Zoeys owner, Christine Brown, says she was a bundle of energy when she first got Zoey.

She was so sweet, said Brown. She was your typical energetic puppy.

But soon Brown knew her dog was hurting.

After coming back from a walk and taking a nap, she would get up and limp, said Brown. With her being a puppy it was devastating.

Zoey was enrolled in aPenn Vet trial to determine the benefits of stem cell therapy as a treatment to ease arthritic pain.

They are randomized into three groups, whether they receive an interarticular joint injection of hyaluronic acid or they geteither stem cells derived from their bone marrow or stem cells derived from fat, saidAgnello.

The stems cells from the dogs bone marrow are injected back into the elbow joint. Doctors hope it will relieve the arthritic pain.

We also remove a little fragment of bone that can be causing some more pain, saidAgnello.

The research isnt just about arthritis in dogs but humans as well.

The goals of this study are to look for different treatments to not only help our canine patientsbut also to help human patients with arthritis, saidAgnello.

For now results are promising.

Oh my gosh, she is not limping, she runs and jumps, and has a great time, said Brown.

The trial is ongoing so there is no hard data yet to show final results if stem cells are effective for treating arthritis, but Dr.Agnello says there are many dogs in the study and almost all of them have improved during the year-long research.

Stephanie Stahl, CBS 3 and The CW Philly 57s Emmy Award-winning health reporter, is featured daily on Eyewitness News. As one of the television industrys most respected medical reporters, Stephanie has been recognized by community and he...

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Veterinary Doctors Conduct Study Looking To Ease Arthritis Pain - CBS Philly

Stem Cell Therapy – Runner’s World


Runner's World
Stem Cell Therapy
Runner's World
This is why researchers and physicians think this therapy may help joint injuries caused by worn-out cartilage; in cell cultures, stem cells can grow new cartilage, and if this can happen in a joint, it may prevent the need for a joint replacement ...
Nutrients Boost Stem Cell FunctionProHealth
At 6th Annual Clinical Trial Supply New England 2017 Conference in Boston Asymmetrex Introduces A First Specific ...Benzinga
Research report explores the stem cell therapy market worth 145.8 million USD by 2021WhaTech

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Stem Cell Therapy - Runner's World

New Cardiac Stem Cell Therapy passes Phase I/II Trials – Labiotech.eu (blog)

TiGenix announces positiveone-year results forits phase I/II trial of donor-derived cardiac stem cell therapy in acute myocardial infarction (AMI).

The Belgian biotech TiGenixis developing allogeneic stem cell therapies. Now the companyhasannouncedthat its cardiac stem cell therapyAlloCSC-01 reached its primary endpoints in aphase I/IItrial.

In 2015, the companyacquired Coretherapixin a292M deal for its allogeneic cardiac stem cell pipeline, which is being developed for the treatment of AMI.The first-in-human trial was designed to test the safety and feasibility of an intracoronary infusion of donor-derivedexpanded cardiac stem cells (AlloCSCs)in patients with AMI and left ventricular dysfunction.

AlloCSC-01consists of adult allogeneic cardiac stem cells isolated from the heartof donors and expanded in vitro. In vivo studies suggest that these cellshave cardio-reparative potential by activating regenerative pathways and promoting the formation of new hearttissue.

Thecurrent phase II study demonstrated thesafety of these allogeneic stem cells. Initial results also revealed a larger reduction of infarct size in a subgroup of patients.

Myocardial infarction caused by blockade of coronary arteries

TiGenix is well known forChondroCellect, which was the first cell therapyto reach the European market for the repair of knee cartilage.After the companyrecently withdrew its market authorization for this product, due to a lack of reimbursement, the biotech is focusing on another stem cell therapy, Cx601, in addition to AlloCSC-01. Under development for Crohns disease, Cx601 is currently awaitingEMA approval and is in phase III trials in the US.

For a late-stage clinical company, TiGenix has a low market cap of191M. Even so, the company seems to be doing well these days with the progress of Cx601 and AlloCSC-01.

If AlloCSC-01 obtains market approval, it could treat the more than 1.9 millionpeople affected by AMI, a major cause of heart failure. So far, most treatments are palliative or restore myocardial function by angioplasty and insertion of a stent to support the vascular lumen.

Stem cell therapy of the heart is definitely not a new topic, but many trials have been conducted using the patients own stem cells derived from the bone marrow. A recent meta-analysisof such trials has suggested that these therapies are safe, but do not enhance cardiac function. TiGenixs approach using allogeneic heart-derived stem cells may offer a new and promisingopportunity in thefield.

Images via shutterstock.com / Liya Graphics andVeronika Zakharova

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New Cardiac Stem Cell Therapy passes Phase I/II Trials - Labiotech.eu (blog)

The Feds Are Spending Millions to Help You Survive Nuclear War – WIRED

Slide: 1 / of 1. Caption: Getty Images

Last week, as tens of thousands of US and South Korean soldiers gathered at a base in Iwakuni, Japan for an annual joint military exercise, North Korea fired four ballistic missiles from Pyongyang into the sea off Japans northwest coast. In a world where the US is headed by a Twigger-happy political neophyte and the risk of a Cold War reboot looms larger with each Wikileaks disclosure, this demonstration wasnt just an empty display of dictatorial propaganda. It was a reminder that the nuclear threat is still alive and well.

But even if youve taken a decades-long break from stocking your fallout shelter, the federal government hasnt. Over the last ten years the US has poured millions of dollars into technologies and treatments it hopes to never have to use, but could, in the event of a nuclear catastrophe. From assays that measure radiation exposure to cell therapies that restore dwindling blood cells to liquid spray skin grafts, government officials are now far better equipped to deal with diagnosing and treating people if the unthinkable were to happen. And the next generation of treatments are being funded right now.

In 2006, the Department of Health and Human Services established the Public Health Emergency Medical Countermeasures Enterprise to coordinate federal solutions to large-scale public health threats, including the nuclear one. Pretty much every agency you can think of is involvedCDC, NIH, FDA, DoD, DHS, USDA, VA, and OEM, among others. But in terms of nuclear countermeasures, three programs nested within HHS do the bulk of the heavy lifting.

The NIHs National Institute of Allergy and Infectious Disease is the first stop; it runs clinical and preclinical trials for promising technologies. Then theres the Biomedical Advanced Research and Development AuthorityBardawhich is basically a taxpayer-backed investment firm that develops these potential drugs, vaccines, treatments, and supplies and ushers them through FDA approval. Finally theres Project BioShield, which contracts with companies when their products are almost ready, ensuring a national market. To date, the project has acquired 12 products related to a nuclear blast or reactor meltdown, some FDA-approved, some still in late stage development, but all destined for the Strategic National Stockpile, the CDC-managed backup supply of drugs and medical supplies for use in a public health emergency. And each class of products addresses a different part of the threat.

The first is diagnosis. When a person is exposed to high levels of radiation, unpaired electrons careen around their cellular machinery, breaking DNA and causing damage to every organ, including the bone marrow. This means you cant generate new red blood cells, white blood cells, and platelets, so you cant fight off infections or coagulate your blood. People usually dont start feeling the effects of acute radiation syndrome for 24 to 48 hours, but damage to their cells DNA starts almost immediately. Which is why you need a reliable diagnostic device; following a nuclear event, people who feel well might actually be in danger, and people who werent exposed will want treatment just to be safe.

So Project BioShield acquired two diagnostic devices, known as biodosimeters, to tell the difference. One works by measuring gene expression, the other by visually analyzing cell nuclei. In the event of a nuclear event, the countermeasures weve procured will be precious resources, says Joe Larsen, acting director of Bardas division of chemical, biological, radiological, and nuclear medical countermeasures. Were going to end up with a lot of worried well demanding treatment, and we can only afford to treat people that need it.

That treatment, at least right now, consists of injections of immune-boosting cytokines, developed for cancer patients to restore depleted white blood cells lost during radiation treatments or chemotherapy. Project BioShield has acquired three such cytokine treatmentsbut, Larsen notes, they wont work for about 20 percent of people. For them, the only option will be bone marrow or cord blood transplants, which come with the extra obstacle of having to be matched with a donor. So Barda and Project BioShield are now looking for cellular therapies that dont require any donor matching to their portfolioa universal treatment. That could shore up gaps in our initial capability to treat radiation. And theyve got at a few promising options coming down the pipeline.

Barda recently signed a $188 million contract to developa stem cell therapy produced by California-based Cellerant Therapeutics, which restores white blood cells in leukemia patients whove had theirs taken outby chemotherapy. The cells are cryopreserved and shelf-stable, important features for a stockpile item. But the treatment is focused on white blood cells, and radiation exposure doesnt limit itself to the immune systems front-line fighters.

To that end, NIAID is funding clinical trials for a placenta-derived stem cell treatment developed by an Israeli company, Pluristem, that has shown the ability to restore all three blood cell linesred and white blood cells, as well as plateletsin animal models. Like Cellerants, the treatment comes cryogenically frozen along with a thawing device to deploy it easily in the field. The cells stay viable on liquid nitrogen inside their canisters, so you dont have to worry about losing them if the power goes out. From their injection site, the placental stem cells sense stress signals in bone marrow tissues, and send more than 20 signaling molecules to repair and restore their functions. The company isnt testing efficacy in humans, for obvious reasons. But Pluristem says their animal studies showed close to 100 percent survival rates with the treatment, compared to 30 percent without.

Arik Eisenkraft, who began working on an ARS application for Pluristems technology following the Fukushima disaster, isnt surprised that a potential solution to nuclear radiation would come out of a place like Israel. We live in a world of imminent threats, not theoretical ones, he said. Even though we dont have the same budgets and the same scope of institutes, what we do have is a real sense of urgency.

Neither Barda nor Pluristem could confirm whether or not a contract is somewhere in their future. But the agency did say it was looking at all the options. And with Bardas budget cut by $160 million last year and an uncertain future for disaster preparedness funds in a Trump administration, theres no time like the present for some urgency of their own.

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The Feds Are Spending Millions to Help You Survive Nuclear War - WIRED

Bone marrow recipient encouraging others to "Be the Match" – KXLY Spokane

SPOKANE, Wash. - A simple cheek swab and a few minutes of your time could save the life of someone in need of a bone marrow transplant.

In 2015, 34-year-old Danielle Vaughan had a stem cell transplant to save her life. For nearly a decade she suffered from mysterious symptoms and illnesses.

"So I had these spin and brain lesions that were very scary," said Danielle Vaughan of Spokane. "I had seizures. I was seeing doctors at Stanford, University of Washington and in Spokane, trying to figure out what was going on."

Eventually, doctors diagnosed Vaughan with Common Variable Immune Deficiency. Her sister, Dina Medin, also had the disorder and underwent a bone marrow transplant a few years ago. The sisters are two of six people in the world with CVID who have had transplants.

"All other treatment options had failed. There was nothing else, that was the only option," said Vaughan.

In September of 2015, Vaughan traveled to Seattle to prepare for the transplant. Vaughan's medical team turned to Be The Match, a national bone marrow donor registry to find her a match. Vaughan said 44 people came back as a perfect match. Her donor was a 27 year old man from the United States. She'd love to meet him one day.

"I would like to say thank you for really giving me a second chance at life. But mostly giving me the opportunity to watch my kids grow," said Vaughan.

Vaughan is now encouraging others to join Be The Match.

"You don't know who you could be helping and you are going to save that person's life," said Vaughan.

The Dairy Queen in Post Falls at 3560 E Seltice Way is hosting a Be The Match donor sign up on Thursday, March 16th from 1:45 p.m.- 4:00 p.m. To register all you need to do is fill out paperwork and swab the inside of your cheek with a special Q-tip.

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Bone marrow recipient encouraging others to "Be the Match" - KXLY Spokane

Be The Match registry seeking blood and bone marrow donors – Fox17

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A blood and marrow transplant replaces abnormal blood-forming stem cells with healthy cells. These are commonly used to treat blood cancers or other kinds of blood diseases that decrease the number of healthy blood cells in the body.

Dr. Stephanie Williams, specialist in blood and marrow transplant and Lindsay Syswerda, event coordinator talk about blood and marrow transplants, and some upcoming events around the topic.

People who receive a blood and marrow transplants are patients with life-threatening blood cancers, diseases that cause bone marrow failure, and other immune system or genetic diseases

However in order for transplants to take place, cells need to be donated from someone else. Transplants can be donated by either an adult donor, a cord blood unit, or a patient can use their own blood-forming cells.

Spectrum Health works closely with a registry for blood and bone marrow donors called Be The Match, which connects patients searching for a cure and life-saving bone marrow donors. As the largest and most diverse marrow registry, it helps patients get the transplant they need.

Anyone between the ages of 18 and 44 can join the registry, but there are guidelines that are in place to protect potential donors. When there's a possible match for a patient, Be The Match discusses the donor's health history and arranges a thorough physical exam.

If potential donors don't meet the medical requirements to join, there are other ways to help contribute to a cure.

One of the ways people can help with the cause is to join Spectrum Health and the Grand Rapids Griffins in their "Road to Recovery," which celebrates patients, families, and their healthcare team's journey at the Griffin's next home game. On March 31, at the Griffin's next home game, they'll be raising awareness for blood cancers and hope to educate others of the importance to join the Be The Match registry.

To learn more about Be The Match, visit BetheMatch.org and use the codeSHGRG.

If you have been diagnosed with cancer and would like a second opinion or consultation, call 1.855.SHCANCER (855.742.2623).

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Be The Match registry seeking blood and bone marrow donors - Fox17

Stem cell swabbing for cancer-stricken Edmonton boy so successful it ran out of kits – Globalnews.ca

A Thursday evening stem cell swab event aimed at finding a match for an eight-year-old Edmonton boy with leukemia was so successful, it ran out of kits.

Brady Mishio has acute myeloid leukemia, a cancer of the blood, and has undergone three rounds of chemotherapy since being diagnosed in November 2016. Doctors say he needs a bone marrow transplant in order to live.

Brady and his dad Terry Mishio.

The transplant needs to come from someone who is a match to Bradys stem cells. What Canadian Blood Services tells, and told me from the beginning, is that the odds are about 50/50 that Brady will find a match, said his father, Terry Mishio.

Matches are made via the Canadian Blood Services (CBS)OneMatch stem cell and marrow network. Its a database that pairs volunteer donors to patients who require stem cell transplants.

According to CBS, donors and patients are matched according to the compatibility of inherited genetic markers called human leukocyte antigens. A perfect stem cell match can be difficult to find, which is why the push is on to get more people registered.

The more people that are in this bank and they check it every 24 hours the more chances that he or some other Edmontonian kids are going to find that match, Mishio said.

READ MORE:Could you save his life? Edmonton boy needs to find stem cell match

Joining the bank is quick, simple and non-invasive: the potential donor just fills out some paperwork and swabs the inside of their mouth with a buccal swab (similar to a Q-Tip.)

A massive swabbing event to potential stem cell donors took place Thursday evening at the Holy Cross Ukrainian Catholic Parish in north Edmonton. Mishio is on leave from his job as a recruiter with the Edmonton Police Service, and several of his policing coworkers were expected to come out in support.

Organizers expected a big turnout, but the response was even greater than they hoped for.

A big crowd of potential stem cell donors getting swabbed at Holy Cross Ukrainian Catholic Parish in north Edmonton. March 9, 2017.

The event was scheduled from 4 p.m. to 8 p.m., but 100 people showed up before the doors were set to open. Mishios co-worker Trishia Comeau said by 7 p.m., 1,100 people had been swabbed and Canadian Blood Services had used up all of the swabbing kits in Edmonton.

Thanks people of Edmonton for helping, we are going to keep this momentum going, Comeau said early Friday morning.

WATCH ABOVE:Terry Mishio talks to Gord Steinke on Global News at 5 from Thursday evenings swabbing event.

If you couldnt make Thursdays event, visit Canadian Blood Servicesfor more information or to set up an appointment. You need to be between 17 and 35 to register.

2017Global News, a division of Corus Entertainment Inc.

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Stem cell swabbing for cancer-stricken Edmonton boy so successful it ran out of kits - Globalnews.ca

Success for Match4Tommy as bone marrow match found for … – Newham Recorder

PUBLISHED: 12:56 10 March 2017 | UPDATED: 13:06 10 March 2017

Sophie Morton

Tommy Simpson, five, was diagnosed with leukaemia last year

Nigel Simpson and Maxine Francis

The family of a five-year-old boy battling leukaemia have thanked prospective donors for coming forward after a stem cell match was found.

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Little Tommy Simpson was diagnosed with acute myeloid leukaemia in January 2016, with his parents launching an appeal to find a lifesaving stem cell donor.

His mixed race heritage - his Stratford-based firefighter dad Nigel Simpson is white British and his events manager mum Maxine Francis is black Carribean meant that the odds of finding a compatible donor were stacked against him.

The couple teamed up with the African Caribbean Leukaemia Trust (ACLT), who launched the #Match4Tommy campaign - and a year on, it has proved successful.

Maxine said: It means many things to Tommys dad and me, to know our son has a matched stem cell donor.

It means in the event of Tommy ever requiring a stem cell transplant; there would be no long agonising wait to find a match for our beautiful son.

It means we can move forward, knowing, we have done everything to make sure that whatever happens in the future, our son has all systems in place.

It means peace of mind.

Throughout the search, Tommy has been receiving treatment, to which he is responding well.

But should he relapse, having a stem cell donor already found would mean that he could be treated quickly, offering him a much better chance of survival.

Thousands of people signed up to the stem cell register during the course of the campaign, including at a donor drive held at Nigels workplace, Stratford fire station, which also raised 700 for ACLT.

Maxine, who lives in Barkingside, will be aiming to raise even more money for the charity when she takes on the London Marathon next month.

She said: ACLT supported my family and me throughout Tommys illness, and I want to raise as much funds as possible for them, to enable them to assist much more families in need with their lifesaving work.

Thank you to everyone who helped make this a reality for our small family. We couldnt have got this far without your help.

To sponsor her, visit uk.virginmoneygiving.com/MaxineFrancis. For details on how to join the register, visit aclt.org

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Success for Match4Tommy as bone marrow match found for ... - Newham Recorder

How ‘Cannibalism’ By Breast Cancer Cells Promotes Dormancy: A Possible Clue Into Cancer Recurrence – IFLScience

By studying the cannabilistic tendency of cancer cells, my research team has made some progress in finding out why.

The chances of recurrence and disease outcome vary with cancer subtype. About one-third of patients diagnosed with triple negative breast cancer, the most aggressive subtype, may experience a recurrence in another part of the body. This is called distant recurrence.

It has been difficult, if not impossible, to predict if and when the same cancer will recur and to stop it. Recurrent disease may arise from just a single cancer cell that survived the initial treatment and became dormant. The dormancy allowed it to hide somewhere in the body, not growing or causing harm for an unpredictable amount of time.

Determining what puts these dormant cells to sleep and what provokes them to wake up and begin multiplying uncontrollably could lead to important new treatments to prevent a demoralizing secondary cancer diagnosis.

Recently, my research team and I uncovered several clues that might explain what triggers these breast cancer cells to go dormant and then reawaken. We showed that cell cannibalism is linked to dormancy.

How do bone stem cells affect breast cancer?

Breast cancer can recur in the breast or in other organs, such as the lungs and bone. Where breast cancer decides to grow depends largely on the microenvironment. This refers to the cells that surround it, including immune cells, cells comprising blood vessels, fibroblasts and the select proteins they produce, among other factors.

Over a century ago, a surgeon named Stephen Paget famously compared the organ-specific prevalence of cancer metastasis to seeds and soil. Because breast cancer often relapses in bones, in this metaphor, which still holds forth today, the bone marrow provides a favorable microenvironment (the soil) for dormant breast cancer cells (the seeds) to thrive.

Just as seeds need soil to provide an environment for growth, cancer cells need an environment to grow. From http://www.shutterstock.com

Thus, a substantial amount of recent work has involved trying to determine the role in cancer dormancy of a special type of cell, called mesenchymal stem cells (MSCs). These are found in bone marrow.

MSCs in bone marrow are highly versatile. They are able to form bone, cartilage and fibrous tissue, as well as cells that support the immune system and formation of blood. They are also known to travel to sites of tissue injury and inflammation, where they aid in healing.

Breast cancer cells readily interact with MSCs if they meet in the bone marrow. They also readily interact if the breast cancer cells recruit them to the site of the primary tumor.

My research team and I recently focused on potential outcomes of these cellular interactions. We found an odd thing happens, which may provide insight into how these breast cancer cells hide for a long time.

In the laboratory setting, we produced breast tumor models containing MSCs. We also re-created the hostile conditions that naturally challenge developing tumors in patients, such as localized nutrient deficits caused by rapid growth of cancer cells and overcrowding.

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How 'Cannibalism' By Breast Cancer Cells Promotes Dormancy: A Possible Clue Into Cancer Recurrence - IFLScience

Kansas Stem Cell Center Close To First Clinical Trial – KMUW

An adult stem cell center established by the Kansas Legislature in 2013 is almost ready for its first clinical trial.

Buddhadeb Dawn, executive director of the Midwest Stem Cell Therapy Center, told legislators Tuesday that the trial will focus on treating graft-versus-host disease and will begin after final approvals from the U.S. Food and Drug Administration.

Our goal was to do this (trial) in January, but we got delayed because of different things, Dawn said during a hearing of the House Health and Human Services Committee. So we are now hoping to start it perhaps in summer.

Based at the University of Kansas Medical Center in Kansas City, the stem cell center has analyzed trials done elsewhere and hosted a clinical trial sponsored by a biotech company that uses modified stem cells from bone marrow to treat stroke.

But the graft-versus-host disease trial would be the first homegrown one.

Graft-versus-host disease is a potential complication when a patient receives a transplant of tissue, like an organ or bone marrow, from another person.

The disease occurs when transplanted tissue fights the patients natural immune system, potentially damaging the liver, skin or other areas. Its a rare illness, with about 20,000 cases in the United States each year.

Rep. Randy Powell, a Republican from Olathe, said the trial was a welcome and exciting development. He said his wife is at risk for the illness following treatment for leukemia.

I know that graft-versus-host is a big thing, Powell said. I think my wife still has an annual checkup where they keep their eye out (to make sure) thats not sticking its head up and causing issues.

Dawn said the center would like to take the next step and move into clinical trials using adult stem cells to treat things like joint ailments, diabetes and amyotrophic lateral sclerosis, also known as Lou Gehrigs disease.

But the regulatory process takes time.

Wed like to be able to offer a portfolio of different disease conditions that adult stem cells can benefit, Dawn said. Im hoping that within the next five years we would at least have some FDA approval for treatment with adult stem cells for other conditions.

Dawn said successful trials could lead to more private investment dollars so we are self-sustaining at some point in the future.

The centers reliance on state funds has been a point of contention for fiscally conservative legislators in the past. Most of the facilitys budget still comes from the states payment, which was reduced by about $28,000 to $754,500 last year.

Thats far less than what stem cell research facilities in other states receive.

Doug Girod, executive vice president of the KU medical center, said that given the budget, Dawn and his small team have done remarkable work.

We could be 10 times bigger than we are and doing 10 times as much if we had the resources, Girod said. But I think were maximizing every opportunity we can with what we have right now.

The center was spearheaded by socially conservative legislators, including Sen. Mary Pilcher-Cook, to showcase adult stem cell research as an alternative to using stem cells derived from human embryos.

About $56,000 of its annual budget goes to educating the public about the differences between embryonic stem cells and adult cells and hosting an annual conference about advances in adult stem cell treatment.

Rep. John Wilson, a Democrat from Lawrence, said he initially was skeptical about the facility because he thought the Legislature was inserting itself into a religious or philosophical fight. But he said his attitude has changed.

Im glad that despite my opposition to it the state has gone forward with funding some really terrific research, Wilson said. My concern now is how do we take it to the next level so all of this hasnt been for nothing.

Andy Marso is a reporter for KMUW's Kansas News Service, a collaboration of KMUW, Kansas Public Radio and KCUR covering health, education and politics in Kansas. You can reach him on Twitter@andymarso.

Go here to see the original:
Kansas Stem Cell Center Close To First Clinical Trial - KMUW

Neuroscientists pinpoint key gene controlling tumor growth in brain cancers – Science Daily

Neuroscientists pinpoint key gene controlling tumor growth in brain cancers
Science Daily
Cedars-Sinai investigators have identified a stem cell-regulating gene that affects tumor growth in patients with brain cancer and can strongly influence survival rates of patients. The findings, published in the online ... Led by Yu, investigators ...

and more »

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Neuroscientists pinpoint key gene controlling tumor growth in brain cancers - Science Daily

Lions LB Paul Worrilow gave stem cells to anonymous leukemia patient – Detroit Free Press

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Atlanta Falcons linebacker Paul Worrilow kisses his 15-month-old daughter, Julie, after the first day of training camp in Flowery Branch, Ga., on July 28, 2016.(Photo: Curtis Compton, Associated Press)

Height, weight: 6 feet, 230 pounds.

Joined the Lions:Worrilow, who turns 27 in May, agreed to a one-year contract with the Lions on Wednesday.

NFL career: He made the Atlanta Falcons in 2013 as an undrafted free agent after being a walk-on at Delaware. Worrilow was the Falcons' starting middle linebacker job in 2013-15. He led the team in tackles each of his first two seasons. Last season, the Falcons wanted to get faster at linebacker, so they drafted two, and Worrilow lost his job to rookie Deion Jones. Worrilow was relegated mostly tospecial teams in 2016 and played just four defensive snaps in the playoffs -- none in the Super Bowl.I know I can go and play good ball, Worrilow told the Atlanta Journal-Constitution. Whether if thats here or somewhere else.

Off the field:In 2011, he signed up for Delawares bone-marrow program. He underwent a six-hour procedure to donate peripheral blood stem cells to an anonymous 21-year-old leukemia patient.

Lions to make Ricky Wagner highest-paid RT; he's 'living his dream'

Contact Carlos Monarrez: cmonarrez@freepress.com. Follow him on Twitter @cmonarrez.

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Lions LB Paul Worrilow gave stem cells to anonymous leukemia patient - Detroit Free Press

Kansas Stem Cell Center Close To First Clinical Trial – KCUR

An adult stem cell center established by the Kansas Legislature in 2013 is almost ready for its first clinical trial.

Buddhadeb Dawn, executive director of the Midwest Stem Cell Therapy Center, told legislators Tuesday that the trial will focus on treating graft-versus-host disease and will begin after final approvals from the U.S. Food and Drug Administration.

Our goal was to do this (trial) in January, but we got delayed because of different things, Dawn said during a hearing of the House Health and Human Services Committee. So we are now hoping to start it perhaps in summer.

Based at the University of Kansas Medical Center in Kansas City, the stem cell center has analyzed trials done elsewhere and hosted a clinical trial sponsored by a biotech company that uses modified stem cells from bone marrow to treat stroke.

But the graft-versus-host disease trial would be the first homegrown one.

Download the Midwest Stem Cell Therapy Center annual update to legislators.

Graft-versus-host disease is a potential complication when a patient receives a transplant of tissue, like an organ or bone marrow, from another person.

The disease occurs when transplanted tissue fights the patients natural immune system, potentially damaging the liver, skin or other areas. Its a rare illness, with about 20,000 cases in the United States each year.

Rep. Randy Powell, a Republican from Olathe, said the trial was a welcome and exciting development. He said his wife is at risk for the illness following treatment for leukemia.

I know that graft-versus-host is a big thing, Powell said. I think my wife still has an annual checkup where they keep their eye out (to make sure) thats not sticking its head up and causing issues.

Dawn said the center would like to take the next step and move into clinical trials using adult stem cells to treat things like joint ailments, diabetes and amyotrophic lateral sclerosis, also known as Lou Gehrigs disease.

But the regulatory process takes time.

Wed like to be able to offer a portfolio of different disease conditions that adult stem cells can benefit, Dawn said. Im hoping that within the next five years we would at least have some FDA approval for treatment with adult stem cells for other conditions.

Dawn said successful trials could lead to more private investment dollars so we are self-sustaining at some point in the future.

The centers reliance on state funds has been a point of contention for fiscally conservative legislators in the past. Most of the facilitys budget still comes from the states payment, which was reduced by about $28,000 to $754,500 last year.

Were maximizing every opportunity we can with what we have right now.

Thats far less than what stem cell research facilities in other states receive.

Doug Girod, executive vice president of the KU medical center, said that given the budget, Dawn and his small team have done remarkable work.

We could be 10 times bigger than we are and doing 10 times as much if we had the resources, Girod said. But I think were maximizing every opportunity we can with what we have right now.

The center was spearheaded by socially conservative legislators, including Sen. Mary Pilcher-Cook, to showcase adult stem cell research as an alternative to using stem cells derived from human embryos.

About $56,000 of its annual budget goes to educating the public about the differences between embryonic stem cells and adult cells and hosting an annual conference about advances in adult stem cell treatment.

Rep. John Wilson, a Democrat from Lawrence, said he initially was skeptical about the facility because he thought the Legislature was inserting itself into a religious or philosophical fight. But he said his attitude has changed.

Im glad that despite my opposition to it the state has gone forward with funding some really terrific research, Wilson said. My concern now is how do we take it to the next level so all of this hasnt been for nothing.

Andy Marso is a reporter for the Kansas News Service, a collaboration of KCUR, Kansas Public Radio and KMUW covering health, education and politics in Kansas. You can reach him on Twitter@andymarso. Kansas News Service stories and photos may be republished at no cost with proper attribution and a link back to kcur.org.

See original here:
Kansas Stem Cell Center Close To First Clinical Trial - KCUR

How ‘cannibalism’ by breast cancer cells promotes dormancy: A … – Medical Xpress

March 8, 2017 by Thomas Bartosh, The Conversation Cancer cells, in red, cannibalize a type of stem cell, shown in green. The red cells with small specks of green are breast cancer cells that have eaten the stem cell.

Breast cancer death rates overall have steadily declined since 1989, leading to an increased number of survivors. But while breast cancer survivors are grateful their bodies show no trace of the disease, they still face anxiety. Breast cancer can and does return, sometimes with a vengeance, even after being in remission for several years.

By studying the "cannabilistic" tendency of cancer cells, my research team has made some progress in finding out why.

The chances of recurrence and disease outcome vary with cancer subtype. About one-third of patients diagnosed with triple negative breast cancer, the most aggressive subtype, may experience a recurrence in another part of the body. This is called distant recurrence.

It has been difficult, if not impossible, to predict if and when the same cancer will recur and to stop it. Recurrent disease may arise from just a single cancer cell that survived the initial treatment and became dormant. The dormancy allowed it to hide somewhere in the body, not growing or causing harm for an unpredictable amount of time.

Determining what puts these dormant cells to "sleep" and what provokes them to "wake up" and begin multiplying uncontrollably could lead to important new treatments to prevent a demoralizing secondary cancer diagnosis.

Recently, my research team and I uncovered several clues that might explain what triggers these breast cancer cells to go dormant and then "reawaken." We showed that cell cannibalism is linked to dormancy.

How do bone stem cells affect breast cancer?

Breast cancer can recur in the breast or in other organs, such as the lungs and bone. Where breast cancer decides to grow depends largely on the microenvironment. This refers to the cells that surround it, including immune cells, cells comprising blood vessels, fibroblasts and the select proteins they produce, among other factors.

Over a century ago, a surgeon named Stephen Paget famously compared the organ-specific prevalence of cancer metastasis to seeds and soil. Because breast cancer often relapses in bones, in this metaphor, which still holds forth today, the bone marrow provides a favorable microenvironment (the "soil") for dormant breast cancer cells (the "seeds") to thrive.

Thus, a substantial amount of recent work has involved trying to determine the role in cancer dormancy of a special type of cell, called mesenchymal stem cells (MSCs). These are found in bone marrow.

MSCs in bone marrow are highly versatile. They are able to form bone, cartilage and fibrous tissue, as well as cells that support the immune system and formation of blood. They are also known to travel to sites of tissue injury and inflammation, where they aid in healing.

Breast cancer cells readily interact with MSCs if they meet in the bone marrow. They also readily interact if the breast cancer cells recruit them to the site of the primary tumor.

My research team and I recently focused on potential outcomes of these cellular interactions. We found an odd thing happens, which may provide insight into how these breast cancer cells hide for a long time.

In the laboratory setting, we produced breast tumor models containing MSCs. We also re-created the hostile conditions that naturally challenge developing tumors in patients, such as localized nutrient deficits caused by rapid growth of cancer cells and overcrowding.

We discovered that cancer cells under this duress become dormant after eating, or "cannibalizing," the stem cells.

Our analysis provided compelling data demonstrating that the cannibalistic breast cancer cells did not form tumors as rapidly as other cancer cells, and sometimes not at all. At the same time, they became highly resistant to chemotherapy and stresses imposed by nutrient deprivation.

Dormant cells are widely linked to recurrence. We hypothesize that cannibalism thus is linked to recurrence.

What is cellular cannibalism, and why is it important in cancer?

Cellular cannibalism, in general, describes a distinct phenomenon in which one cell engulfs and eliminates neighboring, intact cells.

The percentage of cancer cells that show cannibalistic activity is relatively low, but it does appear to increase in more aggressive tumors.

There are several reasons breast cancer cells would want to eat other cells, including other cancer cells. It provides them with a way to feed when nutrients are in short supply. It also provides them a way to eliminate the very immune cells that naturally stop cancer growth. Cell cannibalism might also allow cancer cells to inherit new genetic information and, therefore, new and advantageous traits.

Notably, in our study, cannibalistic breast cancer cells that ate the stem cells and entered dormancy began to produce an array of specific proteins. Many of these proteins are also secreted by normal cells that have permanently stopped dividing, or senescent cells, and have been collectively termed the senescence-associated secretory phenotype (or SASP). Although cellular senescence is a part of aging, we are now realizing that it is also important for a variety of normal bodily processes, development of embryos and injury repair in adults.

This suggests that although dormant cancer cells do not multiply rapidly or form detectable tumors, they are not necessarily sleeping. Instead, at times they might be actively communicating with each other and their microenvironment through the numerous proteins they manufacture.

Overall, this might be a clever way for dormant cancer cells to "fly under the radar" and, at the same time, modify their microenvironment, making it more suitable for them to grow in the future.

Can cell cannibalism be exploited for diagnosis and treatment?

Although our results are promising, it's important to be cautious. While there appears to be a strong correlation between cell cannibalism and dormancy, for now we do not know if it is directly linked to cancer recurrence in patients. Studies are underway, however, to corroborate our findings.

Still, the fact that breast cancer cells cannibalize MSCs is intriguing. It provides an important foundation for developing new diagnostic tools and therapies. Indeed, we currently have several ways of applying our recent discoveries.

One exciting idea is to exploit the cannibalistic activity of cancer cells to feed them suicide genes or other toxic agents, using MSCs as a delivery vehicle, like a tumor-seeking missile.

Importantly, MSCs can be easily obtained from the body, expanded to large numbers in the laboratory, and put back into the patient. Indeed, they have already been used safely in clinical trials to treat a variety of diseases due to their ability to aid in tissue repair and regeneration.

A different avenue for drug development would involve keeping dormant cells in a harmless and nondividing state forever. It might also be possible to prevent cancer cells from eating the stem cells in the first place.

In our study, we were able to block cell cannibalism using a drug that targets a specific protein inside cancer cells. With this treatment approach, the cancer might essentially starve to death or be more easily killed by conventional therapies.

Explore further: A possible explanation for recurring breast cancer

This article was originally published on The Conversation. Read the original article.

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See the article here:
How 'cannibalism' by breast cancer cells promotes dormancy: A ... - Medical Xpress

How ‘cannibalism’ by breast cancer cells promotes dormancy: A possible clue into cancer recurrence – The Conversation US

Cancer cells, in red, cannibalize a type of stem cell, shown in green. The red cells with small specks of green are breast cancer cells that have eaten the stem cell.

Breast cancer death rates overall have steadily declined since 1989, leading to an increased number of survivors. But while breast cancer survivors are grateful their bodies show no trace of the disease, they still face anxiety. Breast cancer can and does return, sometimes with a vengeance, even after being in remission for several years.

By studying the cannabilistic tendency of cancer cells, my research team has made some progress in finding out why.

The chances of recurrence and disease outcome vary with cancer subtype. About one-third of patients diagnosed with triple negative breast cancer, the most aggressive subtype, may experience a recurrence in another part of the body. This is called distant recurrence.

It has been difficult, if not impossible, to predict if and when the same cancer will recur and to stop it. Recurrent disease may arise from just a single cancer cell that survived the initial treatment and became dormant. The dormancy allowed it to hide somewhere in the body, not growing or causing harm for an unpredictable amount of time.

Determining what puts these dormant cells to sleep and what provokes them to wake up and begin multiplying uncontrollably could lead to important new treatments to prevent a demoralizing secondary cancer diagnosis.

Recently, my research team and I uncovered several clues that might explain what triggers these breast cancer cells to go dormant and then reawaken. We showed that cell cannibalism is linked to dormancy.

Breast cancer can recur in the breast or in other organs, such as the lungs and bone. Where breast cancer decides to grow depends largely on the microenvironment. This refers to the cells that surround it, including immune cells, cells comprising blood vessels, fibroblasts and the select proteins they produce, among other factors.

Over a century ago, a surgeon named Stephen Paget famously compared the organ-specific prevalence of cancer metastasis to seeds and soil. Because breast cancer often relapses in bones, in this metaphor, which still holds forth today, the bone marrow provides a favorable microenvironment (the soil) for dormant breast cancer cells (the seeds) to thrive.

Thus, a substantial amount of recent work has involved trying to determine the role in cancer dormancy of a special type of cell, called mesenchymal stem cells (MSCs). These are found in bone marrow.

MSCs in bone marrow are highly versatile. They are able to form bone, cartilage and fibrous tissue, as well as cells that support the immune system and formation of blood. They are also known to travel to sites of tissue injury and inflammation, where they aid in healing.

Breast cancer cells readily interact with MSCs if they meet in the bone marrow. They also readily interact if the breast cancer cells recruit them to the site of the primary tumor.

My research team and I recently focused on potential outcomes of these cellular interactions. We found an odd thing happens, which may provide insight into how these breast cancer cells hide for a long time.

In the laboratory setting, we produced breast tumor models containing MSCs. We also re-created the hostile conditions that naturally challenge developing tumors in patients, such as localized nutrient deficits caused by rapid growth of cancer cells and overcrowding.

We discovered that cancer cells under this duress become dormant after eating, or cannibalizing, the stem cells.

Our analysis provided compelling data demonstrating that the cannibalistic breast cancer cells did not form tumors as rapidly as other cancer cells, and sometimes not at all. At the same time, they became highly resistant to chemotherapy and stresses imposed by nutrient deprivation.

Dormant cells are widely linked to recurrence. We hypothesize that cannibalism thus is linked to recurrence.

Cellular cannibalism, in general, describes a distinct phenomenon in which one cell engulfs and eliminates neighboring, intact cells.

The percentage of cancer cells that show cannibalistic activity is relatively low, but it does appear to increase in more aggressive tumors.

There are several reasons breast cancer cells would want to eat other cells, including other cancer cells. It provides them with a way to feed when nutrients are in short supply. It also provides them a way to eliminate the very immune cells that naturally stop cancer growth. Cell cannibalism might also allow cancer cells to inherit new genetic information and, therefore, new and advantageous traits.

Notably, in our study, cannibalistic breast cancer cells that ate the stem cells and entered dormancy began to produce an array of specific proteins. Many of these proteins are also secreted by normal cells that have permanently stopped dividing, or senescent cells, and have been collectively termed the senescence-associated secretory phenotype (or SASP). Although cellular senescence is a part of aging, we are now realizing that it is also important for a variety of normal bodily processes, development of embryos and injury repair in adults.

This suggests that although dormant cancer cells do not multiply rapidly or form detectable tumors, they are not necessarily sleeping. Instead, at times they might be actively communicating with each other and their microenvironment through the numerous proteins they manufacture.

Overall, this might be a clever way for dormant cancer cells to fly under the radar and, at the same time, modify their microenvironment, making it more suitable for them to grow in the future.

Although our results are promising, its important to be cautious. While there appears to be a strong correlation between cell cannibalism and dormancy, for now we do not know if it is directly linked to cancer recurrence in patients. Studies are underway, however, to corroborate our findings.

Still, the fact that breast cancer cells cannibalize MSCs is intriguing. It provides an important foundation for developing new diagnostic tools and therapies. Indeed, we currently have several ways of applying our recent discoveries.

One exciting idea is to exploit the cannibalistic activity of cancer cells to feed them suicide genes or other toxic agents, using MSCs as a delivery vehicle, like a tumor-seeking missile.

Importantly, MSCs can be easily obtained from the body, expanded to large numbers in the laboratory, and put back into the patient. Indeed, they have already been used safely in clinical trials to treat a variety of diseases due to their ability to aid in tissue repair and regeneration.

A different avenue for drug development would involve keeping dormant cells in a harmless and nondividing state forever. It might also be possible to prevent cancer cells from eating the stem cells in the first place.

In our study, we were able to block cell cannibalism using a drug that targets a specific protein inside cancer cells. With this treatment approach, the cancer might essentially starve to death or be more easily killed by conventional therapies.

Read more:
How 'cannibalism' by breast cancer cells promotes dormancy: A possible clue into cancer recurrence - The Conversation US

Did Gene Therapy Cure Sickle Cell Disease? – American Council on Science and Health

A number of recent headlines imply a case study just published in the New England Journal of Medicine proves that gene therapy has cured sickle cell diseasea genetic disorder that incurs tremendous pain, suffering and diminished life expectancy. Here, we will unpack the significance of the researchers findings.

First, lets address why this news could be so groundbreaking to those afflicted and their loved ones.

Sickle Cell Disease is an inherited condition that causes a mutated hemoglobinthe protein within red blood cells (RBCs) that carries oxygen for delivery to vital tissues. Oxygen feeds our organs so they can stay healthy and perform their respective jobs. This Hemoglobin S (aka Sickle Hemoglobin) polymerizes on deoxygenation and rids the RBCs of their malleability. As a result, these malformed sickled cells are stiff and clump together thereby occluding vessels which in turn prompts organ damage.

Roughly 90,000 Americans have Sickle Cell Disease. (1) The natural course of the illness involves a complex cascade of events intermingled with crises often triggered by infections. Anemia is commonplace (and often profound) given these faulty cells get readily destroyed, over consumed and dont last as long as healthy RBCs. Vasoocclusive Crises result from infarction and ischemiain infants the hands and feet swell, in particular. Basically, adequate blood flow is halted wherever the obstruction takes place. Aggressive pain management and rehydration is essential.

Prophylactic antibiotics are a mainstay in an effort to stave off infection which can routinely catapult patients into a life-threatening crisis. By early childhood, they develop a functional asplenia or ineffective spleen. So, they become especially susceptible to overwhelming infection by encapsulatedbacteriahence, why vaccination for pneumococcus and the like is so important. Sepsis can result. Parvovirus can cause an aplastic crisis.

Strokes. Pulmonary infarcts with subsequent hypoxia. Acute Chest Syndrome. Gallstones. Blood transfusions are frequent. Though the blood supply is well-tested for safety, recurrent transfusion can lead to issues like iron overload, for instance. This too must be treated. The list goes on of the challenges, battles and treatment complexities these patients endure. Because fetal hemoglobin has a higher oxygen carrying capacity, a disease-modifying drug like Hydroxyurea that increases its presence is used.

Allogeneic hematopoietic stem-cell transplantation represents the only cure, but less than 18% of those with severe disease have sibling donors who are a match. (2) This is also not without great risk, though those need to be weighed against how advanced the disease. Due to such limited progress in management of this condition, this team of researchers sought to examine whether therapeutic ex vivo gene transfer into autologous hematopoietic stem cells referred to as gene therapy, may provide a long-term and potentially curative treatment for sickle cell disease. (3)

What does this mean? They took samples from the bone marrow of a patient with severe disease. The cells here provide the origins of our blood components which includes our red blood cells. This is where the problem begins in generating the sickling. A cancer drug, busulfan, was used to condition the body expected adverse effects from this occurred which resolved with standard care (e.g. anemia, low platelets, neutropenia and so on). Using a lentiviral vector, they transferred an anti-sickling gene into the patients stem cells (retrieved from the bone marrow) which get put back into the patient in the hope they will multiply and replace the cells made with the defective gene.

In a study funded in part by Bluebird Bio whose product is LentiGlobin BB305 (the antisickling gene therapy subject of this publication), the team concludes their patient had complete clinical remission with correction of hemolysis and biologic hallmarks of the disease. Furthermore, after fifteen months the antisickling protein remained high at approximately 50% and the patient had no crises or hospitalizations. Before, the patient required regular transfusions. After, all medications were stopped, no pain ones were needed, and the patient returned to full activities at school. (4)

Ongoing research is underway in a U.S. multi center, phase 1/2 clinical study. The intention is to use this gene therapy to treat those with severe sickle cell disease and another condition called beta-thalessemia. So far, in the few patients who have participated, their results seemingly support this work. Clearly, longer term follow-up and larger populations are crucial to understanding the significance of this report. Additionally, stem cell transplantation is no minor feat.

That said, for a disease that disables at such a young age, this option could be quite an extraordinary one if the success persists. ACSHs Senior Fellow in Molecular Biology, Dr. Julianna LeMieux, puts the promise of gene therapy into even greater context for this and other disease entities:"This is an incredibly promising result, even with the obvious caveat that it is only one person. Sickle Cell is a disease that is ripe for genetic advances for a few reasons. First,the gene that is affected is known andcan be replaced by the healthy variant. Also, the cells that are needed to be alteredare easily accessible inthe bone marrow. In many diseases, this is not the case. But, this one success story is incredibly encouraging for the sickle cell community and for moving the field of curing diseases using genetic editing forward."

The team proved their concept. To know if "cure" is in this gene therapy's future, much more data needs to be acquired and study be implemented. Promising with cautious optimism might be the most apt description.

Source(s):

(1) (2) (3) (4) Jean-Antoine Ribeil, M.D., Ph.D. et al. Gene Therapy in a Patient with Sickle Cell Disease. N Engl J Med. 376;848-855. March 2, 2017.

Note(s):

To learn more about "Orphan Diseases" or rare ones that afflict less than 200,000 (but in total impact 25 million Americans) and drug discovery challenges, review: Did Pompe Disease Geta New Champion in President Trump? and Pompe Disease, Newborn Screening and Inborn Errors of Metabolism.

Follow this link:
Did Gene Therapy Cure Sickle Cell Disease? - American Council on Science and Health

Researchers report positive bone marrow transplant case | The Herald – The Herald

People with a certain gene have an adverse reaction to the antiretroviral efavirenz

Liz HighleymanCorrespondent An HIV-positive bone marrow transplant recipient at the Mayo Clinic experienced prolonged viral remission lasting nearly 10 months longer than the so-called Boston patients after interrupting antiretroviral therapy (ART), according to a report at the Conference on Retroviruses and Opportunistic Infections (CROI 2017) last month in Seattle.

Although his viral load eventually rebounded, his HIV reservoirs appeared to be reduced.

The only person known to be cured of HIV Timothy Ray Brown, known as the Berlin Patient stopped ART when he received a bone marrow transplant to treat leukaemia and has not had detectable virus for 10 years. Brown received a transplant from a donor with a double CCR5-delta-32 mutation, meaning they lack the CCR5 co-receptors most types of HIV use to enter T-cells.

It is unclear whether his sustained remission is attributable to the donors CCR5 mutation, the strong chemotherapy conditioning regimen used to kill off cancerous blood cells, a graft-versus-host reaction or multiple factors.

Bone marrow transplantation is apparently not sufficient to eradicate HIV.

A few years ago, Timothy Henrich reported on two HIV-positive bone marrow transplant patients in Boston who got stem cells from wild-type donors without the CCR5-delta-32 mutation, received a milder conditioning regimen and experienced acute graft-versus-host disease (GVHD). Both men maintained undetectable viral load longer than expected after interrupting ART, but eventually they experienced viral rebound at three and eight months after stopping HIV treatment.

The latest case, presented by Nathan Cummins of the Mayo Clinic in Rochester, Minnesota, and colleagues, involved a 55-year-old man who was diagnosed with HIV in 1990 and started combination ART in 1999 with a CD4 T-cell count of 300 cells/mm3. He stopped treatment between 2004 and 2009 for unexplained reasons, then restarted ART consisting of ritonavir-boosted atazanavir (Prezista) plus tenofovir disoproxil fumarate (DF) and emtricitabine (the drugs in Truvada).

In April 2013 the man was diagnosed with B-cell acute lymphoblastic leukaemia.

In anticipation of chemotherapy, his ART regimen was switched to raltegravir (Isentress), etravirine (Intelence), and tenofovir DF/emtricitabine. In October 2013 he underwent reduced intensity conditioning followed by an allogeneic stem cell transplant from a CCR5 wild-type donor.

At the time of transplantation the man had an HIV viral load of 25 copies/ml and a CD4 count of 288 cells/mm3, and he stayed on ART without interruption. After the transplant he developed opportunistic infections (E. coli septicaemia and pneumocystis pneumonia) and experienced GVHD at four months post-transplant.

The man continued on ART for more than two years after transplantation, mostly with detectable plasma viral load levels. HIV RNA was also undetectable in gut biopsy samples. HIV DNA in his peripheral blood cells became undetectable by day 56, and repeated leukapheresis procedures showed significant reductions in HIV RNA and DNA reservoir size.

In addition, that mans HIV antibody levels decreased, as indicated by weaker Western blot bands. However, single genome sequencing and phylogenetic analysis identified identical HIV clones at day 142, possibly due to homoeostatic proliferation, or replication of latently infected cells, while he had GVHD.

After having such low HIV levels for a prolonged period, the man underwent an analytic treatment interruption, or carefully monitored discontinuation of ART. His plasma HIV RNA levels were tested every two weeks for the first 12 weeks of ART interruption, then every four weeks.

At day 288 9,6 months after stopping ART he was found to have low-level viral rebound to 60 copies/ml. This rose to 1640 copies/ml by day 293, requiring that he restart HIV treatment.

The man had no evidence of drug resistance and his viral load was re-suppressed within a month.

Allogeneic peripheral blood stem cell transplantation in the setting of HIV is associated with significant reductions in HIV reservoir size by multiple measures, including prolonged combination ART-free remission, the researchers concluded.

They added that stem cell transplantation in the setting of suppressed viral replication may be associated with loss of HIV-specific immunity, and hypothesised that immune activation in the setting of GVHD without anti-HIV specific immunity may cause homoeostatic proliferation of latently infected cells, decreasing the chance of HIV eradication. http://www.aidsmap.com.

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Researchers report positive bone marrow transplant case | The Herald - The Herald

Stem Cell Therapy: Modern Solution to Joint Pain Relief – PR Newswire (press release)

PLAINVIEW, N.Y., March 6, 2017 /PRNewswire/ -- Board certified surgeon Dr. Andrew J. Rochman promotes the specialized treatment of Stem Cell Therapy in his private practice in Plainview, NY. The recent wave of positive response from its ability to target a growing number of health conditions has brought increased attention and demand from pain sufferers everywhere- including the Long Island area.

As of the fall of 2016, Dr. Rochman officially opened the Cell Therapy Center of NY (CTCNY) where he focuses on the treatment of osteoarthritis & rheumatoid arthritis, eroded cartilage and joint issues like tennis elbow, jumpers knee and golf elbow. He dedicated his practice to the large population suffering from musculoskeletal damage arising from sports injuries or the wear and tear from aging. "We are always seeking a safer and more effective alternative to surgery to battle physiological symptoms with the hopes of giving patients a pain-free life."

Dr. Rochman underwent extensive training from U.S. Stem Cell, Inc. (Sunrise, FLA) - a center for the development of effective cell technologies recognized for treating a variety of diseases and injuries. U.S. Stem Cell's discoveries include multiple cell therapies in various stages of development that repair damaged tissue due to injury or disease. Chief Science Officer Kristin C. Comella, expert in regenerative medicine with a focus on adipose derived stem cells, pioneered stem cell therapies derived from various sources including cord blood, bone marrow and muscle. "By harnessing the body's own healing potential, we may be able to reverse damaged tissue to normal function.... stem cells have the ability to form many types of tissues like bone, cartilage, muscle and even help to reverse some effects that have been caused by damaged tissue," states Ms. Comella.

Dr. Rochman's treatment center has recently seen an influx of patients from Long Island's large athletic and fitness community. President of the Wildwood Warriors triathlon team, John Graziano is one of the many joint and back pain sufferers from sports injuries seeking this alternative pain treatment. "In the world of triathlon, I train- I race- and I live with pain and lots of it!"

"The potential here is limitless," states Dr. Rochman. "It's actually a simple yet unique form of therapy with the possibilities of doing miraculous things. We found out within the past several years that human beings have stem cells in every tissue of their body and they actually live around the blood vessels." Today's stem cell therapy has been shown to manage and target a wide span of healing possibilities from blood cell diseases to cardiac disorders to autoimmune diseases. "So what we can do now is to extract fat cells from the belly or bone marrow cells and isolate the cells from those damaged tissues... perhaps in the future we can utilize this process to target neurological diseases, heart diseases... and we don't even know where it ends," says Dr. Rochman.

Dr. Andrew J. Rochman is a native New Yorker and a board-certified surgeon. He is a graduate of Colgate University and received his formal medical training from Nordestana University. He is an active member of the American Medical Association, the Medical Society of the State of New York, Nassau County Medical Society and the American College of Phlebology. Dr. Rochman manages several practices in specialized studies such as advanced vein therapy and gallbladder surgery. His current certification is with U.S. Stem Cell, Inc. specializing in cardiovascular treatment through stem cell technology. The Cell Therapy Center of NY is located at 700 Old Country Road, Suite 205 Plainview, NY. For more information, visit: http://www.celltherapycenterny.com or call 516-280-1333.

Media contact: Lennard Gettz 148804@email4pr.com 631-553-8748

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/stem-cell-therapy-modern-solution-to-joint-pain-relief-300417654.html

SOURCE Cell Therapy Center of NY

http://www.celltherapycenterny.com

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Stem Cell Therapy: Modern Solution to Joint Pain Relief - PR Newswire (press release)

Could you save his life? Edmonton boy needs to find stem cell match – Globalnews.ca

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Brady and his dad Terry Mishio.

Another stem cell swab event is taking place on Thursday with the hopes of finding a match for an eight-year-old Edmonton boy with leukemia.

In November, Brady Mishios dad took him to the hospital because he thought he had the flu.

Quickly they did some tests and then they did some more tests and X-rays and things and I knew at that point you know, a parent realizes something is awry there, Terry Mishio said.

WATCH:Edmonton boy with leukemia needs bone marrow transplant

That night, Bradywas diagnosed with acute myeloid leukemia, which is cancer of the blood.

I didnt know what the oncology unit meant, now I certainly do, Mishiosaid. Then, I think it was a nurse or someone who said, They think your son has cancer It turned out to be trueHe was diagnosed with cancer and he had leukemia.

Youre hoping its not true, Mishio said. Youre hoping they made a mistake.

Brady has already gone through three rounds of chemotherapy. Doctors say he needs a bone marrow transplant.

Now, the family is desperately searching for a stem cell match.

The world needs to know that this is what we need and this can save kids lives, Mishio said. Not just Bradys life theres other families in the Stollery Theyre waiting for their matches.

Its going to be a matter of minutes, he said of the testing process. Just to go in there, fill out the screen, the paper work, and then give a mouth swab and thats it. And then youre in the bank and you could save a life.

READ MORE:Alberta mother battling leukemia desperately searching for stem cell match

A massive swabbing event is takingplace on March 9 from 4 p.m. to 9 p.m. at the Holy Cross Ukrainian Catholic Parish at 9003 153 Avenue.

Organizers are expecting a big turnout, including Edmonton police officers and recruits, who will be getting swabs and registered as potential stem cell donors.

Basically, were looking for somebodys genetic twin, explained Robyn Henwood, with Canadian Blood Services.

In Canada alone we have about 1,000people at any given time looking for a match. Based on our international database, well find a match for about 50 per cent of them.

READ MORE:Young Edmonton boy searching for rare stem cell donor

If you cant make Thursdays event, visit Canadian Blood Services for more information or to set up an appointment. You need to be between 17 and 35 years old to register.

Its a little awkward to scrub the inside of your cheeks with Q-tips, Henwood said, but thats all it takes to register and potentially be the person that families are waiting for.

In 2013, 1,255 Canadians were diagnosed with acute myelogenous leukemia.

Eight-year-old Brady Mishio.

Eight-year-old Brady Mishio.

Eight-year-old Brady Mishio.

Brady and his dad Terry Mishio.

2017Global News, a division of Corus Entertainment Inc

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Could you save his life? Edmonton boy needs to find stem cell match - Globalnews.ca

‘Reset’ for the immune system – Burlington Times News

By Jessica Williams / Times-News

Shawna Hamlett will be born again June 8.

The 40-year-old Alamance native and single mother was diagnosed with relapsing remitting multiple sclerosis 13 years ago, and has dealt with episodes of numbness, sudden blindness, deafness, difficulty walking, pain and extreme fatigue.

But May 1, Hamlett will travel to Chicagos Northwestern Hospital to undergo a month-and-a-half-long experimental procedure called hematopoietic stemcell transplant, which uses stemcells from the patients own bone marrow, along with chemotherapy, essentially toreset the immune system.

By the time she receives the stemcell transplant June 8, she will have lost her hair and nearly all of the antibodies built up from vaccines that shes received since birth, but will no longer have multiple sclerosis.

This is mind-boggling to me, Hamlett said. My body wont remember that I had MS at all because my stemcells are going to be frozen [for] two weeks, and when they give them back, they dont know they had MS ever. The chemo completely reboots your immune system so much so that the vaccines you got when you were an infant your body no longer has.

She applied for the procedure in November and was, to her surprise, approved for it in late January. It couldnt come a moment too soon, Hamlett said, as the neurologist in Chicago told her during a consultation that shes one legion away from a catastrophic event maybe even paralysis, an even greater concern considering that she has two sons, 13 and 17.

I just wish I had done this years ago, when the doctor first started doing it, because I could have had their whole childhood without having it, and Ive had to struggle at ballgames. Ive been on the sidelines at soccer games with an IV in my hand because Ive had an IV at home and Im not going to miss their games, Hamlett said. But theyre going to grow up, and theyre going to get married and have kids, and I dont want to be not able to be around.

AS WITH ANY MEDICAL procedure, a heavy cost is involved. Along with spending the better part of nearly two months away from her kids, the longest shes ever been away from them, Hamlett will have to pay $25,000to $35,000 after insurance.

Shes enlisted help from community businesses, friends and family to help cover that expense, and has plans for seven fundraisers.

Hamlett says community support is essential for people with MS, and she would advise anyone just diagnosed to seek out others.

Talk to people who are in the same boat as you, she said. Theres a huge community of people on both Instagram and Facebook that suddenly become your family because nobody, we always say, nobody gets it until they get it. People who understand exactly what youre going through are out there, so youre not ever alone.

REGARDLESS OF HOW much money is raised, Hamletts life is about to change for the better. Shes nervous, but eager, and says its going to be a huge relief when shes fully-recovered.

The thing about MS is you never know from day to day how its going to be the next day, which is good and bad because, if its bad, then tomorrow could be a good day, but if its good, then tomorrow could be a bad day, she said. Id like to be on an even keel and just know that after the procedure there wont be any more unknowns.

Hamlet has set up a donation page through HelpHOPELive for donors to contribute to the cost of the procedure and hotel expenses for her time in Chicago. Visit https://helphopelive.org/campaign/12529 or search Shawna Hamlett HelpHopeLive to find out more.

All donations are tax-deductible, held by HelpHOPELive in the South Atlantic Stem Cell Transplant Fund, and administered by HelpHOPELive for transplant-related expenses only.

Reporter Jessica Williams can be reached at jessica.williams@thetimesnews.com or at 336-506-3046. Follow her on Twitter at @jessicawtn.

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'Reset' for the immune system - Burlington Times News

Scientists wage fight against aging bone marrow stem cell niche – Science Daily


Science Daily
Scientists wage fight against aging bone marrow stem cell niche
Science Daily
Red staining reveals the abundant presence of the protein osteopontin (OPN) in bone and endosteum of the marrow cavity, which is important to maintaining a healthy environment for blood-forming hematopoietic stem cells. Shown in blue are cell nuclei.

and more »

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Scientists wage fight against aging bone marrow stem cell niche - Science Daily

Opinion/Commentary: Seniors put at risk by outdated Medicare policies – The Daily Progress

Almost 30 years ago, the federal government helped make it easier for patients with leukemia and lymphoma to receive lifesaving stem cell transplants. Now, we need the federal governments help again to ensure that Medicare patients with these cancers and other serious blood disorders can access the care they need.

In 1987, Congress approved funding for a national database of patients willing to donate bone marrow or peripheral blood stem cells. That database is now known as the Be The Match Registry, operated by the National Marrow Donor Program/Be The Match. According to the NMDP/Be The Match, patients searching the registry have access to 27 million potential volunteer bone marrow and peripheral blood stem cell donors worldwide, along with more than 680,000 units of cord blood donated by mothers after giving birth.

Having access to such a large registry has made it easier for patients to find a match if they dont have a fully matched sibling donor, which is the case for about 70 percent of patients who receive a stem cell transplant. The registry has helped 80,000 patients receive bone marrow transplants, peripheral blood stem cell transplants, or cord blood transplants from an unrelated donor.

While the federal governments foresight and financial support have helped make adult stem cell and cord blood transplants the only cure available for these diseases possible for thousands of patients, Medicare coverage policies have not kept pace with this breakthrough treatment.

Medicare is more restrictive than private insurance companies in deciding for what indications stem cell transplants and cord blood transplants will be covered. With private insurance companies, we have the opportunity to talk with a medical director about the indication and provide literature to support the decision for a transplant. This opportunity is not available for our Medicare patients.

In most cases, Medicare doesnt decide whether to cover a stem cell or cord blood transplant until after the procedure is completed. This leaves most Medicare patients an impossible choice: Turn down their only chance at a cure or potentially face paying the significant cost of a transplant themselves. Even when Medicare does decide to reimburse for these transplants, according to the NMDP/Be The Match, it covers less than half the cost of the transplant.

Addressing this issue is especially important because seniors make up a large portion of the patients with the cancers and blood diseases that can be cured by a stem cell or cord blood transplant. For example, 24 of the 65 patients who received stem cell or cord blood transplants at University of Virginia Health System in 2016 had Medicare coverage.

So I am asking the Centers for Medicare & Medicaid Services to expand Medicare coverage for stem cell and cord blood transplants, along with paying for the search and procurement costs as they already do for solid organ transplants.

The federal government has helped save the lives of tens of thousands of patients through better access to stem cell and cord blood transplants. I hope now they will act to make sure all Medicare patients who need one of these transplants can receive it.

Tamila L. Kindwall-Keller, DO, MS is the associate clinical director of the Stem Cell Transplant Program at the University of Virginia Health System.

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Opinion/Commentary: Seniors put at risk by outdated Medicare policies - The Daily Progress

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