Bacterial infection activates hematopoietic stem cells in the bone marrow and significantly reduces the ability to produce blood through induced proliferation. Credit: Professor Hitoshi Takizawa

It has been thought that only immune cells would act as the line of defense during bacterial infection. However, recent research has revealed that hematopoietic stem cells, cells that create all other blood cells throughout an individual's lifetime, are also able to respond to the infection. A collaboration between researchers from Japan and Switzerland found that bacterial infection activates hematopoietic stem cells in the bone marrow and significantly reduces their ability to produce blood by forcibly inducing proliferation. These findings indicate that bacterial infections might trigger dysregulation of blood formation, such as that found in anemia or leukemia. This information is important to consider in the development of prevention methods for blood diseases.

Background: Bacterial Infection and the Associated Immune Reaction

When a person becomes infected with a virus or bacteria, immune cells in the blood or lymph react to the infection. Some of these immune cells use "sensors" on their surfaces, called Toll-like receptors (TLR), to distinguish invading pathogens from molecules that are expressed by the host. By doing so, they can attack and ultimately destroy pathogens thereby protecting the body without attacking host cells.

Bone marrow contains hematopoietic stem cells which create blood cells, such as lymphocytes and erythrocytes, throughout life. When infection occurs, a large number of immune cells are activated and consumed. It therefore becomes necessary to replenish these immune cells by increasing blood production in bone marrow. Recent studies have revealed that immune cells are not the only cells that detect the danger signals associated with infection. Hematopoietic stem cells also identify these signals and use them to adjust blood production. However, little was known about how hematopoietic stem cells respond to bacterial infection or how it affected their function.

Proof: Hematopoietic Stem Cell Response to Bacterial Infection

Researchers from Kumamoto University and the University of Zurich analyzed the role of TLRs in hematopoietic stem cells upon bacterial infection, given that both immune cells and hematopoietic stem cells have TLRs. Lipopolysaccharide (LPS), one of the key molecules found in the outer membrane of gram negative bacteria and known to cause sepsis, was given to laboratory animals to generate a bacterial infection model. Furthermore, researchers analyzed the detailed role of TLRs in hematopoietic stem cell regulation by combining genetically modified animals that do not have TLR and related molecules, or agents that inhibit these molecules.

The results showed that LPSs spread throughout the body with some eventually reaching the bone marrow. This stimulated the TLR of the hematopoietic stem cells and induced them to proliferate. They also discovered that while the stimulus promoted proliferation, it also induced stress on the stem cells at the same time. In other words, although hematopoietic stem cells proliferate temporarily upon TLR stimulation, their ability to successfully self-replicate decreases, resulting in diminished blood production. Similar results were obtained after infection with E. coli bacteria.

Future Work

This study reveals that hematopoietic stem cells, while not in charge of immune reactions, are able to respond to bacterial infections resulting in a reduced ability to produce blood. This suggests that cell division of hematopoietic stem cells forced by bacterial infection induces stress and may further cause dysregulated hematopoiesis like that which occurs in anemia or leukemia. "Fortunately we were able to confirm that this molecular reaction can be inhibited by drugs," said one of the study leaders, Professor Hitoshi Takizawa of Kumamoto University's IRCMS. "The medication maintains the production of blood and immune cells without weakening the immune reaction against pathogenic bacteria. It might be possible to simultaneously prevent blood diseases and many bacterial infections in the future."

This finding was posted online in Cell Stem Cell on 21 July 2017, and an illustration from the research content was chosen as the cover of the issue.

Explore further: Innate reaction of hematopoietic stem cells to severe infections

More information: Hitoshi Takizawa et al, Pathogen-Induced TLR4-TRIF Innate Immune Signaling in Hematopoietic Stem Cells Promotes Proliferation but Reduces Competitive Fitness, Cell Stem Cell (2017). DOI: 10.1016/j.stem.2017.06.013

Journal reference: Cell Stem Cell

Provided by: Kumamoto University

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Bacterial infection stresses hematopoietic stem cells - Medical Xpress - Medical Xpress

Bone Marrow Stem Cells Comments Off on Bacterial infection stresses hematopoietic stem cells – Medical Xpress – Medical Xpress | August 25th, 2017

Erica Honoway is scheduled to speak at the annual "Run for Mandi" charity event in Saskatoon, after her son Lincoln was saved by a bone marrow transplant.Michael Bell / Regina Leader-Post

An annual run honouring a late Canadian hockey player is working with a bone marrow and stem cell registry group in hopes of helping more people in her name.

The Run for Mandi is named for Saskatchewan hockey player Mandi Schwartz, who was diagnosed with acute myeloid leukemia in 2008 while she was part of the Yale Bulldogs hockey team. She died in 2011.

The event kicks off Sunday afternoon at River Landing. The five-kilometre run and the one-kilometre family walk will start at two, and for the first time a bone marrow and stem cell registry group will be set up at the run.

Mandis mother, Carol Schwartz, said shes proud of the work being done by the Mandi Schwartz Foundation in her daughters name.

It just makes these events more meaningful lives are being saved, Schwartz said. Theres probably no greater gift than meeting someone who got a successful match.

The OneMatch Stem Cell and Marrow Network, a part of Canadian Blood Services, will accept registrations at Sundays event. Schwartz said theyve handed out information before, but this is the first time OneMatch will swab volunteers at the event to register them in the network.

Bobbylynn Stewart with Breck Construction, the title sponsor for the event, said she has a personal stake in helping organize the run because her mother also died of acute myeloid leukemia. Its a chance for the company and the community to help other families with similar struggles, she said.

When you have a blood cancer or disorder, often times you are relying on a stem cell match through the network, Stewart said. So growing that network is vital.

Alongside the run will be a charity silent auction and a barbecue. Mandis brothers, professional hockey players Jayden and Rylan Schwartz, are also expected to attend, along with NHL players Ryan Murray and JC Lipton, and AHL player Brandon Gormley.

Erica Honoway, scheduled to speak before the run, said she is haunted by how close her family came to sharing in the Schwartzs tragedy.

Her son Lincoln was diagnosed with aplastic anemia last year, but a bone marrow transplant helped save his life.

In all the registries in the world, they found two matches for Lincoln, Honoway said. Every single person who gets on is another chance for someone to have their life saved.

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Annual 'Run for Mandi' hosting bone marrow registry to combat cancer - Saskatoon StarPhoenix

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Madalayna and Tamara Ducharme, 33 days after Madalayna received her bone marrow transplant. Photo provided by Tamara Ducharme) By Adelle LoiselleAugust 25, 2017 5:10am

Six months ago, Windsor residents came out in droves to help baby Madalayna Ducharme find a bone marrow match.

Saturday, they can help again by taking part in a bowl-a-thon dedicated to supporting the families of those who still need a transplant.

The 12th annual Bowling for Bone Marrow Bowl-a-Thon takes place Saturday at Rose Bowl Lanes on Dougall Ave. in Windsor. Check-in is at noon, and the fundraiser gets underway at 1pm.

It is the Katelyn Bedard Bone Marrow Associations biggest fundraiser of the year, and this year it can count Madalayna among its success stories.

The baby girl, who celebrated her first birthday this week, likes to dance and can stand while holding her parents fingers. Her mother, Tamara Ducharme is grateful for every day.

We were unsure if we were going to make it there, to the first birthday, she says. Were hoping that shell be a healthy little girl.

However, the struggle is not over. Friday, the family is driving up Hwy. 401 for Madalaynas six-month post-transplant appointment at Sick Kids Hospital in Toronto.

Ducharme says her daughter has bi-weekly hospital visits to ensure her medication is up to date. Madalayna still uses a feeding tube, and even months later, there is still the question whether the bone marrow transplant from her brother is working.

Theyll probably do an x-ray, says Ducharme about the upcoming appointment. Shes had a little growth. If her bones show changes that means shes on the track of getting better. Now, if there is no change, I dont know what were going to do.

Life with a young child who has received a transplant can also be very isolating, and Ducharme admits it has not been easy.

Were bubbled. We really go anywhere. We dont really play with other kids, she says. Youve gotta take the proper steps to take care of your child. If she could catch anything and it could be really detrimental.

She says the association has been very good to her family and they are grateful for their, and the communitys support over a challenging chapter in their lives.

Bryan and Joanne Bedard understand the difficulties faced by families of children waiting for a donor. They lost their three-year-old daughter, Katelyn in 2005 at the age of 3 when they were unable to find one.

Since then, they have raised money for donor clinics and awareness of the OneMatch Stem Cell and Marrow Network which now has 6,500 registered donors. The Katelyn Bedard Bone Marrow Association has also donated $115,000 to stem cell and bone marrow transplant research at both the University of Windsor and the Universite de Montreal.

With files from Maureen Revait

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Bowl-A-Thon For Stem Cell Bone Marrow Transplant Recipients - BlackburnNews.com

Bone Marrow Stem Cells Comments Off on Bowl-A-Thon For Stem Cell Bone Marrow Transplant Recipients – BlackburnNews.com | August 25th, 2017

I didnt want it to affect my job as a BBC correspondent covering religious affairs. But in 2015 I went numb down one side of my body for several weeks. A scan showed multiple lesions, and a specialist confirmed I had relapsing-remitting MS. It felt like a life sentence. My family wasnt surprised wed long known it was a possibility. No tears were shed; my parents are of the stiff-upper-lip generation.

The next drug failed to stop another relapse. I wanted to keep reporting for as long as possible, but last September I finally stepped down frommy job, and my bosses helped me find other options involving less travel and with more predictable hours.

My MS seemed to be moving from relapsing-remitting to secondary progressive, as my body became less able to repair the brain damage caused by each relapse. So, after months of research, I decided to take a risk and have an autologous hematopoietic stem cell transplant (HSCT) a chemotherapy treatment that wipes out then regrows your immune system at a private clinic in Mexico.

HSCT has long been used to treat blood cancers, but its use in autoimmune diseases like MS is still undergoing trials. It destroys the malfunctioning cells thought to be responsible for damaging the myelin sheath, while stem cells harvested from your bone marrow are given back to shorten your time without a working immune system.

HSCT holds the most promise for people having regular MS attacks and its now offered to some MS patients at a few NHS hospitals in England under tight criteria. I didnt qualify, but I met several others who had been helped by it.

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'I approached my 50th birthday unable to balance or speak' - Telegraph.co.uk

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NBC-5 News anchor Rob Stafford will return to the air Monday, after months of grueling treatment for a rare blood disorder that gave him a harrowing look at "my own mortality."

"I thought we'd get this thing nipped in the bud," said Stafford, 58, who took a leave of absence in March after being diagnosed to be in the early stages of amyloidosis.

Instead, Stafford said, he spent much of the last six months too sick to eat, drink or walk while learning that the road back to health from serious illness is a process.

"You learn that everybody reacts to these drugs differently and there is no guarantee of any outcome," he said.

Amyloidosis occurs when abnormal protein called amyloid is produced in bone marrow and can be deposited in tissues and organs. There are more than 40 types of the disorder that affect the heart, kidneys, liver, spleen, nervous system and digestive tract. Stafford's type known as light chain amyloidosis is rare, according to Dr. Ronald Go, Stafford's hematologist at the Mayo Clinic in Rochester, Minn.

Doctors had planned to remove or "harvest" stem cells from Stafford's own bone marrow and freeze millions of healthy ones. After wiping out the unhealthy cells using chemotherapy, Stafford was to have the healthy stem cells transplanted back into his bone marrow, where they were to reproduce themselves, Go said in March.

Zbigniew Bzdak/Chicago Tribune

Rob Stafford, shown Aug. 24, 2017, is planning to return to the anchor desk at NBC-5 News on Aug. 28 after months battling amyloidosis.

Rob Stafford, shown Aug. 24, 2017, is planning to return to the anchor desk at NBC-5 News on Aug. 28 after months battling amyloidosis. (Zbigniew Bzdak/Chicago Tribune)

But Stafford ran into several complications immediately after the transplant process began that forced him to remain hospitalized for most of March.

"There were times in the hospital when I thought he might not make it," said his wife, Lisa Stafford, who would jog around the Rochester area to alleviate her stress.

"On the runs, I would stop at every church to pray and light a candle."

Stafford returned to his home in Hinsdale in early April, too weak sometimes to walk across the room, drink a milkshake or even stay awake for the news, he said.

In June, test results showed the bone marrow transplant did not work as they had planned, and Stafford would need a new course of action to fight the disease, he said.

It was a terrifying place to be, Stafford said.

"You think, 'What if nothing works?'" he said. "I have clearly thought about my own mortality."

Doctors at Rush University Medical Center started Stafford on a new regimen of weekly chemotherapy, which dramatically improved his health. While he has not yet reached the low amyloid measurements that define remission, doctors are optimistic about his recovery and have cleared Stafford to return to work, he said.

Stafford will return to the 10 p.m. news. Dick Johnson and Patrick Fazio will share anchoring duties with Allison Rosati at 5 p.m. and 6 p.m. until Stafford is ready to return to those newscasts, said Frank Whittaker, station manager and vice president of news for NBC Chicago.

"We are eagerly looking forward to Rob's return on Monday night," Whittaker said in an email. "He has inspired all of us with his courage and determination over the past six months. It will be great to have him back in our newsroom."

Stafford said he remains grateful for the support he and Lisa felt from viewers, who sent him a steady stream of Facebook messages, cards and personal stories.

"It's like running a marathon, and there are all these people along the side cheering you on," Stafford said. "It helps you get through it."

vortiz@chicagotribune.com

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After treatment for serious illness, NBC-5 anchor Rob Stafford returning to air - Chicago Tribune

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The parents of a Saskatchewan-born Yale University hockey player are trying to connect more people with a bone marrow and stem cell network that could save lives.

Rick and Carol Schwartz will be in Saskatoon on Sunday for the sixth annual Run for Mandi named after their daughter Mandi Schwartz, who was diagnosed with acute myeloid leukemia in December 2008 and died in April 2011.

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

Officials from the OneMatch Stem Cell and Marrow Network, part of Canadian Blood Services, will take swabs from volunteers in hopes of connecting donors with patients who need stem cell transplants.

Its the first time the event will have on-site registration for the network.

Fewer than 25 per cent of people find a stem cell donor in their family and only 50 per cent find a match in the international network of donors, according to Blood Services.

Mandi never found one.

It was frustrating to know that. Its almost like we let her down, Rick Schwartz said.

In 2010, his daughter penned a letter, stating her hope that doctors would find her a life-saving match. She also hoped to increase the donor registry to help others.

If someone else in Mandis family needed a stem cell transplant, she wouldve been the first person to help out, her mother said.

I just know she would be front and centre in leading a drive if she were with us today, Carol Schwartz said.

Another registration drive in Mandis name happens annually at Yale University. So far, more than 6,000 people have registered and 37 have resulted in stem cell matches.

Ideal candidates are between the ages of 17 and 35 and meet certain health criteria.

If a person registers and matches with a person in need, its usually as easy taking blood, according to Run for Mandi co-organizer Bobbylynn Stewart.

Fifteen per cent of the time, they do require your bone marrow, said Stewart, who lost her mother to acute myeloid leukemia.

They go in through your hip and draw it through there, so its under anesthesia. Its about an hour-long process.

READ MORE: Run for Mandi raises over $20K for memorial bursary funds

Sundays event lasts from 1 p.m. to 4 p.m. and running isnt required.

Lincoln Honoway, who was three when he was admitted to Regina General Hospital last year with dangerously low blood counts, will be in attendance.

After finding a stem cell transplant, Lincolns blood cell counts have started to rise and stabilize.

The run is planned for River Landing, with pro hockey players Ryan Murray, JC Lipon and Brandon Gormley expected to be there.

Mandis brother, Jaden Schwartz of the St. Louis Blues, will attend as well.

2017Global News, a division of Corus Entertainment Inc.

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Family of Mandi Schwartz connecting donors with stem cell network - Globalnews.ca

Bone Marrow Stem Cells Comments Off on Family of Mandi Schwartz connecting donors with stem cell network – Globalnews.ca | August 25th, 2017

TROY The second annual Buckeye Be The Match will build on its opening year by adding bikes to the event while raising awareness for bloodborne cancers this Saturday.

The Buckeye Be The Match will begin at 8 a.m. Aug. 26, at Treasure Island Park. New this year is a 15-mile and 50-mile bike route in addition to the 5K and 1K Fun Run in the park.

The event added the 15-mile family bike ride north to Piqua and back to Treasure Island as well as a more challenging 50-mile ride throughout the county to expand the use of the nearby bike paths to include cyclists of all levels. Bikers may begin to ride as early as 7:30 a.m. Saturday.

Online registration ends on Thursday, but registration in person will continue through 8 a.m. Saturday at the park. Opening events kick off at 9 a.m. All proceeds benefit the Be The Match organization, which helps build a national registry to find potential donors through a simple cheek swab.

The funding goes to Be The Match, which is dedicated to finding the bone marrow matches or stem cell matches for those with blood born cancers. It is very vital, said city council member Tom Kendall. If you dont want to run or be part of the bike ride, you do have the opportunity to save a life also. They will be taking swabs of those who would like to be put on the registry to be a potential donor for a person in need.

Kendall said Rum River Blend will provide entertainment as well as family-friendly activities through noon. There also will be a ceremony featuring the Be The Hero award, which nominates someone who has helped a survivor during their treatment and will be given out at the event.

Kendalls daughter, Lisa, was diagnosed at 28 with acute myeloid leukemia in 2011. She received a stem cell transplant that saved her life. Shes been a coordinator of the event and advocate for the Be The Match organization since it moved to Troy last year from Columbus.

Kendall said previous the Buckeye Be The Match raised more than $11,000 last year, exceeding its goal of $10,000.

For more information, visit http://www.bethematchfoundation.org.

Event to raise funds and awareness for bone marrow registry

Follow Melanie Yingst on Twitter @Troydailynews

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Buckeye Be The Match set for Saturday - Piqua Daily Call

Bone Marrow Stem Cells Comments Off on Buckeye Be The Match set for Saturday – Piqua Daily Call | August 24th, 2017

Vitamin C may prompt faulty stem cells in bone marrow to die off, rather than multiplying to spur blood cancers.

A new study has found that vitamin C may communicate to faulty stem cells within bone marrow that they should mature and perish in a normal manner rather than multiplying to spur blood cancers. This is the insight gleaned from a study spearheaded by NYU Langone Health Perlmutter Cancer Center researchers. Study details were recently published in Cell.

About the Findings

The authors of the study state specific genetic alterations are known to decrease the ability of an enzyme referred to as tet methylcytosine dioxygenase 2 (TET2) to promote stem cell maturation and death in patients who have specific types of leukemia. They determined vitamin C activates TET2 functionality in mice designed to lack the enzyme. It is possible that vitamin C will prove to be a safe and effective treatment for diseases spurred by leukemia stem cells deficient in TET2. It is likely that vitamin C will be used in combination with other targeted therapies.

Study Details

The researchers used genetically altered mice in which TET2 was turned off. These mice endured abnormal stem cell activity. Such changes were reversed when a genetic trick restored TET2 expression. Providing high doses of vitamin C functioned similarly to restoring TET2 functionality on a genetic level. Vitamin C's promotion of DNA demethylation caused stem cells to mature and limited the advancement of leukemia cancer stem cells from humans that were implanted in mice. Vitamin C treatment affected leukemic stem cells similar to damaged DNA. Vitamin C was used in combination with a PARP inhibitor to produce an enhanced effect on such stem cells, sending them from self-renewal to maturity and subsequent death.

TET2 and Cancer

Alterations in the genetic code that decrease TET2 functionality are found in 10% of those who have acute myeloid leukemia (AML). About one-third of patients with a form of preleukemia known as myelodysplastic syndrome and upwards of half of those with chronic myelomonocytic leukemia have such genetic code mutations. These cancers spur anemia, bleeding and infection risk as abnormal stem cells multiply within bone marrow until they block the production of blood cells. Recent tests show about 2.5% of cancer patients living in the United States might develop TET2 alterations. This includes some patients with solid tumors and lymphomas.

About Cell Death Switch

The results of the study center on the relationship between cytosine and TET2. Cytosine is one of the several letters of nucleic acidthat make up genes' DNA code. Each cell type has thesame genes yet each receives unique instructions to turn on only those required in a specific cellular context. Examples of such epigenetic mechanisms include DNA methylation. This is an attachment of a diminutive molecule to cytosine bases to put a halt to the action of a gene containing them. Gene expression within stem cells is fine-tuned when methyl groups are attached and removed. Stem cellexpressions can then mature and multiplyto form muscle, nerve, bone and other types of cells. The bone marrow holds stem cell pools as adulthood is reached until they can become replacement cells. Inpatients with leukemia, signals that typically tell blood stem cells to mature end up malfunctioning. This allows for endless multiplication and a self-renewing rather than the generation of regular white blood cells required to combat infection.

TET2 empowers an alteration in the molecular structure of methyl groups required for their removal from cytosines. Such demethylation activates genes that direct stem cells to mature and commence a countdown to self-destruction as a component of regular turnover. This functions as a means of combating cancer yet it is disrupted in blood cancer patients who have TET2 mutations.

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Vitamin C May Help Slay Blood Cancer Stem Cells - Anti Aging News

Bone Marrow Stem Cells Comments Off on Vitamin C May Help Slay Blood Cancer Stem Cells – Anti Aging News | August 22nd, 2017

The ABMDR team at the meeting with Archbishop Hovnan Derderian (center) and the Very Rev. Fr. Dajad Yardemian.

LOS ANGELESOn Sunday, August 20, during Holy Mass at St. Leon Cathedral in Burbank, Archbishop Hovnan Derderian, Primate of the Western Diocese, offered special prayers for patients of the Armenian Bone Marrow Donor Registry (ABMDR). In his sermon, the Archbishop praised the life-saving mission of ABMDR, and called on congregants to continue to support its work.

To raise public awareness of the ABMDR mission and encourage grassroots involvement in the organizations activities, the Western Diocese has observed a special Prayer Day in honor of ABMDR patients for the past several years. The Prayer Day is marked at St. Leon Cathedral as well as Armenian churches across Southern California.

In the course of his sermon on August 20, Archbishop Derderian stated that participating in the work of ABMDR is tantamount to praying and accomplishing a Godly mission. The Archbishop pledged the continuous support of the Diocese and appealed to all parishes to embrace the work of ABMDR, by joining its ranks as potential bone marrow stem cell donors, signing up as volunteers, and attending its public-benefit events such as the upcoming Match for Life, the ABMDRs 18th annual Gala, which will be held on Sunday, August 27, in Los Angeles.

The ABMDR team outside St. Leon Cathedral

Archbishop Derderian, who is one of ABMDRs most avid and longtime supporters, exemplifies the type of leadership that works tirelessly for the well-being of our community, said ABMDR president Dr. Frieda Jordan. We are honored and grateful for the Primates ongoing guidance and support.

Following the church service, numerous parishioners had the opportunity to become more familiar with the activities of ABMDR, as a team of Board members and volunteers from the organization answered questions and handed out information about becoming donors.

Subsequently Archbishop Derderian, along with the Very Rev. Fr. Dajad Yardemian, met with the ABMDR team at the Diocese. The discussion centered on ABMDRs most recent achievements as well as its plans for the immediate future. At the conclusion of the meeting, Archbishop Derderian presented scarves from Holy Echmiadzin to all members of the ABMDR team, as tokens of his appreciation.

Established in 1999, ABMDR, a nonprofit organization, helps Armenians and non-Armenians worldwide survive life-threatening blood-related illnesses by recruiting and matching donors to those requiring bone marrow stem cell transplants. To date, the registry has recruited over 29,000 donors in 42 countries across four continents, identified over 4,190 patients, and facilitated 30 bone marrow transplants. For more information, call (323) 663-3609 or visit abmdr.am.

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Archbishop Derderian Leads Prayers for ABMDR Patients at Diocese Churches - Asbarez Armenian News

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VistaGen Therapeutics has received a notice of allowance for a stem cell production patent, which the firm says could be used in autoimmune disorder and cancer treatments.

The US Patent and Trademark Office (USPTO) issued VistaStem a subsidiary of VistaGen the notice for patent no. 14/359,517, which covers methods for producing hematopoietic precursor stem cells usually found in red blood marrow.

These are stem cells that give rise to all of the blood cells and most of the bone marrow cells in the body, with potential to impact both direct and supportive therapy for autoimmune disorders and cancer, said VistaGen VP Mark McPartland.

With CAR-T cell applications and foundational technology, McPartland said he believed the technology will provide approaches for producing bone marrow stem cells for bone marrow transfusions.

Business opportunities

In December last year, VistaGen signed an exclusive sublicense agreement with stem cell research firm BlueRock Therapeutics, under which the latter paid VistaGen $1.25m (1.06m) upfront for its cardiac stem cell production technologies.

McPartland said he expects this recent notice of allowance to also create potential opportunities for additional regenerative medicine transactions.

IP portfolio growth

VistaGen told us it plans to secure IP protection in multiple domains and international jurisdictions.

We intend to grow our IP portfolio in a manner that emphasises platform protection and maximises opportunities for commercialisation and out-licensing, McPartland said.

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VistaGen's cell production methods receive US patent boost - BioPharma-Reporter.com

Bone Marrow Stem Cells Comments Off on VistaGen’s cell production methods receive US patent boost – BioPharma-Reporter.com | August 22nd, 2017

Howard University Hospital's Dr. Ermias Aytenfisu seeks to clear up misconceptions about marrow donation in the minority community.

WASHINGTON, D.C. (August 21, 2017) Elsa Nega is an Ethiopian-Canadian mother of two young children. She loves her children and wants to watch them grow. However, Nega has a rare form of blood cancer, leukemia, and needs a bone marrow transplant to survive.

Black patients like Nega are the least likely to find their suitable blood marrow match, according to Be The Match which is hosting a Stem Cell/Bone Marrow registry event at the Howard University College of Medicine on Wednesday, Aug. 30 between 11 a.m. and 3 p.m. The exact location for the registry drive is the lobby outside of room 1008 in the Numa P. Adams building.

Negas story began in February when she walked into her local ER and was rushed to intensive care. By the next morning Nega was diagnosed with Acute Lymphoblastic Leukemia (ALL) and started on chemo immediately. Unlike 90 percent of patients who go into remission after the first round of chemo, she did not.

Now, after three rounds of chemo, a bone marrow transplant is her only hope of recovery. Negas siblings were not a match and she is reaching out to the Washington region because of its large population of people of Ethiopian descent.

There are a lot of myths associated with marrow donation, said Amanda Holk, community engagement representative with the Be The Match in Washington, D.C. There is so much fear surrounding the process but most donors are back to work the next day.

ErmiasM. Aytenfisu, M.D., stroke medical director at Howard University Hospital said the most common way to donate bone marrow is through a procedure called peripheral stem cell donation. No surgery is involved. Donors receive medication to increase peripheral stem cells before the donation. On the day of donation, blood is removed through a needle on one arm and passed through a machine that separates out the blood-forming cells. Uncommonly marrow donation involves surgical techniques that use a special needle to take out blood forming cells. During the procedure, the patient is anesthetized and feels no pain.

Joining the bone marrow registry at the Howard University College of Medicine event involves a simple as a cheek swab and an application. A persons chance of being a match at that point is only 1 in 500. But, for a patient like Elsa, you could be the only one. Elsa does not have a single match on the registry although there are 30 million people signed up.

For more information, contact Amanda Holk via email AHolk@nmdp.org or 202-875-9987

For the Howard University registry drive, please note that you must be between the ages of 18 and 44 to join the registry since research has shown that the younger the cells, the better the patient outcomes. And the following conditions prevent you from joining:

Hepatitis B or C

HIV

Organ, marrow or stem cell transplant recipient

Stroke or TIA (transient ischemic attack)

Other upcoming local events to support Elsa Nega:

*Empower the community (The Helen Show)

Date: 08/26/2017 (Sat.)

Location: Washington Convention Center

*Ethiopian Day Festival

Date: 09/03/2017 (Sun.)

Location: Downtown Silver Spring

About Howard University Hospital

Over the course of its roughly 155-year history of providing the finest primary, secondary and tertiary health care services, Howard University Hospital (HUH) remains one of the most comprehensive health care facilities in the Washington, D.C. metropolitan area and designated a DC Level 1 Trauma Center. The hospital is the nation's only teaching hospital located on the campus of a historically Black university. For more information, visit huhealthcare.com

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Howard University Hosts 'Be The Match' Marrow Registry Drive - Howard Newsroom (press release)

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When Alex Shorheardthat he was a match for a stranger in Israel who would likely die without a stem cell transplant, he didn't think twice before saying "yes."

"If I today I help somebody, tomorrow I want somebody to help me too if I [am] sick," said Shor. "I don't think too much about it."

The request came from Ezer Mizion, an Israeli health service with the world's largest Jewish bone marrow registry, countingover 850,000 registrants worldwide. Shor said the representative told him the recipient would be a63-year-old man in Israel.

Shor, 41, had registered his DNA with the registry 10 years ago when he lived in Israel.

Shor and his family emigrated to Winnipeg nearly three years ago. In March, he got word that his stem cells were a match.

Stem cells are immature blood cells that can grow into healthy cells. They can make the difference between life and death for people with various forms of cancer, blood-related illnesses and metabolic disorders.

Shorwas agenetic match for the man based on the human leukocyte antigen (HLA) system, which codes the human immune system. The pair would have had to have 10 of the same HLA markers to be a viable match.

In May, Shorwent to a lab in Winnipeg to draw blood to send off to Israel to ensure hisblood would be compatible with the recipient's. Now, he plans to travel to Israel to donate his stem cells as soon as he hears from the physicians that the patient's condition has improved enough to tolerate the procedure.

Getting Shor's blood to Israel required a cooler, a courier and some creativity.

Vials of Shor's blood were transported to Israel in an ice-packed Thermos.

Dena Bensalmon, Canadian director of Israeli health service Ezer Mizion, put out a call on Facebook for a chaperone that could transport five vials of Shor's blood.

"Sixteen people came forward within about four minutes," she said.

One woman the perfect candidate was travelling from Winnipeg to Toronto, then on to Isreal. They packed the blood in ice in a Coleman thermos for the 12-hour journey.

"I met Dina in Toronto and then I switched the ice packs. They took the blood directly," she said.

Canada'sOneMatchregistry through Canadian Blood Serviceshas about 400,000 registrants.

But"if a person is Jewish, then the chances of them finding their match on a Jewish registry is far greater than them finding their match on a non-Jewish registry," saidBensalmon.

Canadian Blood Services has access to nearly 29 million volunteer donors and more than 720,000 cord blood units from dozens of countries around the world, as all the registries are connected under the umbrella of the World Marrow Donor Association, comprised of millions of people from across the world.

"I find the whole thing almost like watching a circle of life," said Bensalmon.

A volunteer brought vials of Shor's blood to Israel. She kept the thermos in her lap the whole 12-hour trip.

Shor said he just thought of his own father and how he would want someone to help him if he had a life-threatening illness. He encourages everyone to join a stem-cell registry.

"Tomorrow you may save somebody and tomorrow you don't know if you be sick and somebody save you," said Shor.

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Winnipeg man to donate stem cells to critically ill stranger in Israel - CBC.ca

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Home Cancer The power of vitamin C: Can it kill cancer stem cells?

Every three minutes, one person in the United States is diagnosed with blood cancer. Thankfully, there may be a new approach to helping these individuals fight it using vitamin C.

Researchers from Perlmutter Cancer Center at NYU Langone Health recently published a report in the journal Cell indicating that vitamin C may be able to tell faulty cells in bone marrow to mature and die instead of multiplying to cause blood cancers. They explained that specific genetic changes are able to reduce the ability of the enzyme known as TET2 to push stem cells to mature, which die in many people who suffer from leukemia. Experts discovered that vitamin C seemed to activate TET2 in mice that were engineered to be TET2 deficient. In simple terms, TET2 is a tumor suppressor that can prevent certain cells from growing uncontrollably.

Mutations that reduce TET2 function are present in about 10 percent of people with acute myeloid leukemia, 30 percent of patients with a pre-leukemia known as myelodysplastic syndrome, and close to 50 percent of people with chronic myelomonocytic leukemia. Tests indicate that about 2.5 percent of U.S. cancer patients develop TET2 mutations, including some with lymphomas.

The study focused on the relationship between TET2 and cytosine, which is one of four nucleic acid letters that make up the DNA codes in our genes. The attachment of a small molecule, referred to as a methyl group, to cytosine bases can shut down the actions of a gene. As the human body forms, the attachment and removal of methyl groups adjust gene expression in stem cells, which can mature and become muscle, bone, nerve, or other cell types. The bone marrow keeps stem cells in pools, ready to become replacement cells when and if needed. In the case of leukemia, the signals that are supposed to tell a blood stem cell to mature end up malfunctioning, leaving it to multiply instead of developing normal white cells, which are needed to help fight infection.

Medical scientists explain that TET2 allows for a change in methyl groups that are required to be removed from cytosine. This essentially turns on genes and directs stem cells to mature and eventually destroy themselves. Researchers say that this signals an anti-cancer mechanism, something that can help blood cancer patients with TET2 mutations.

The team of researchers genetically engineered mice to manipulate the TET2 gene. Techniques to turn off TET2 in mice lead to abnormal stem cell behavior. The changes were reversed when TET2 was restored. Since previous work indicated that vitamin C could stimulate TET2, the researchers theorized that high doses of vitamin C might reverse the effects of TET2 deficiency. It would be a case of turning up the action on the functional gene. As it turns out, high dose vitamin C treatment did induce stem cells to mature and also suppressed the growth of leukemia cancer cells implanted in mice.

As of now, the NYU team is working on identifying genetic changes that may contribute to the risk of leukemia in specific groups of patients. While this latest study provides some hope for blood cancer patients, the manipulation of TET2 is only a potential new treatment approach until further studies are conducted. Currently approved treatments for blood cancers include stem cell transplantation, chemotherapy, and radiation therapy.

Related: Combining antibiotics and vitamin C helps to combat cancer stem cells

Related Reading:

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http://www.cancercenter.com/terms/blood-cancers/https://ghr.nlm.nih.gov/gene/TET2

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The power of vitamin C: Can it kill cancer stem cells? - Bel Marra Health

Bone Marrow Stem Cells Comments Off on The power of vitamin C: Can it kill cancer stem cells? – Bel Marra Health | August 22nd, 2017

There is truth in the old proverb about apple consumption and medical appointments. Insufficient vitamin C can contribute to leukemia. This observed relationship has now been shown to operate through the regulatory role the vitamin plays in the operation of bone marrow stem cells.

These days messages touting a single ingredient as being capable of curing all ills are more likely to peddleturmeric or cannabis, but a few decades ago it was vitamin C that was hailedas preventing everything from theflu to cancer if you took enough. As exaggerated as most of these claims were, it's certainly true that ascorbate, as it is also known, is vital to our health, sometimes in ways that are still unexplained.

We have known for a while that people with lower levels of ascorbate (vitamin C) are at increased cancer risk, but we havent fully understood why, said Dr Sean Morrison of Childrens Medical Center Research Institute UT Southwestern. Stem cells clearly played a part, but are so rare in any individual tissue that it is impossible to collect the millions usually used for metabolic analysis. Moreover, most mammals make their own ascorbate, but humans cannot, impeding the use of animal models.

Morrison and his co-authors of a paper published in Nature had to develop new techniques to measure metabolite usage in populations as small as 10,000 stem cells to address the first problem. On applying these techniques the authors discovered each type of blood-forming cell has a distinctive signature to its metabolite consumption. They tackled the second problem using mice that lack ascorbate-producing enzymes.

When given a low vitamin C diet these mice had more, and more active, bone marrow stem cells, increasing blood cell production at the price of higher rates of leukemia. The vitamin C concentration was related to levels of the enzyme Tet2, which regulates blood production. Without enough Tet2, the stem cells behaved like an overheating engine, turning out blood cells at a great rate until they turned cancerous. Something similar is observed when mutations reduce Tet2 production.

The first clinical application of the discovery is for patients with clonal hematopoiesis, a condition that often involves reduced Tet2 production and leukemia. Our results suggest patients with clonal hematopoiesis and a Tet2 mutation should be particularly careful to get 100 percent of their daily vitamin C requirement, Morrison said. These patients... need to maximize the residual Tet2 tumor-suppressor activity to protect themselves from cancer.

Since stem cells are much sparser in the rest of the body than in bone marrow it will be even more challenging to extend the research to other cancers.

The ideal dose of vitamin C remains to be established, although a paper, coincidentally published last week, may indicate benefits beyond current recommendations.

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Vitamin C Can Suppress Leukemia Up To a Point | IFLScience - IFLScience

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Madalayna Ducharme is celebrating her first birthday, on Aug, 22, 2017. She's shown recently in a family pool.Courtesy of the Ducharme family / Windsor Star

Bowling for Bone Marrow fundraiser Saturday

To support the families of those who need astem cell/bone marrow transplant, head to this weekends 12th annual Bowling for Bone Marrow Throw a Strike for the Gift of Life.

The Katelyn Bedard Bone Marrow Association fundraiser is Saturday at Rose Bowl Lanes with a noon check-in time and bowling between 1 and 3 p.m. Walk-ins are welcome and the cost is $20 without a pledge form.Children under age 12 get in free and there will be a clown and childrens activities.

To register, call (519) 564-4119 or go online atwww.givemarrow.net/index.html.

Windsors warrior princess Madalayna Ducharme celebrates her first birthday Tuesday.

Were so happy and grateful that weve had her for a year, her mom, Tamara Ducharme, said Monday. I know back at the six-month mark we had a little celebration for her just in case we didnt have a year birthday.

On March 17, little Madalayna received a bone marrow transplant to save her from a rare genetic disorder.

Madalayna will get to try cake for the first time, even if its just for her tiny fingers to play in, and her mom plans to go live on Facebook in the late afternoon for the approximately 3,000 supportive followers on the Miracle for Madalaynasite.

I cant believe how many people love her and support us. It makes us so happy, Ducharme said. We could be going through this alone. I feel like there are 3,000 fighters in our corner.

Madalayna, dubbed the warrior princess, was just two months old when doctors noticed issues that a few months later would be diagnosed as malignant infantile osteopetrosis which leads toabnormal thickening of the bone. Without treatment, the one-in-200,000 genetic disorder would dramatically reduce the infants life expectancy.

The Windsor community rallied around the family, and there were efforts made to get more people to join the bone marrow registry. Ducharme said shes thankful for the support from the Katelyn Bedard Bone Marrow Association. She said getting swabbed for the registry wasnt just for Madalayna but to help all those waiting for a match.

Because of the genetic disorder, at first doctors werent looking to family members for a match but Madalaynas two-year-old brother Henrik proved a perfect match and doctors consulted in the United States and Europe agreed his bone marrow was the familys best option.

The family didnt get Madalayna home from Toronto and London hospitals until July, and the little warrior fought off a virus that is worrisome with transplant patients, her mom said. So far, blood tests are looking good but the family wont know until after more extensive tests later this week in Toronto whether the transplant is working.

Ducharme is asking for prayers for good news in Toronto. The transplant is as close to a cure as possible, she said. Madalayna may have hearing and sight issues from the disease, but if the bones look better and the transplant is working, it gives her a chance at a longer life. Ducharme has heard of a man who had the disease and a transplant as a baby and is now 25 years old.

Madalyna, who loves music and looks like a princess in her tutu and frilly dresses, is a bit delayed with all that shes been through, but a week ago she sat up for the first time and she likes to dance by bouncing and swaying to techno music. She still needs the tube in her nose and doesnt like drinking liquids and isnt eating properly. Shes improving but her mom doesnt know what her baby will think of birthday cake.

Were excited.

shill@postmedia.com

twitter.com/winstarhill

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First birthday for Windsor's 'warrior princess' after lifesaving transplant - Windsor Star

Bone Marrow Stem Cells Comments Off on First birthday for Windsor’s ‘warrior princess’ after lifesaving transplant – Windsor Star | August 22nd, 2017

Researchers at the Mayo Clinic and the University of Minnesota say theyre on the brink of a new era in cancer care one in which doctors extract a patients white blood cells, have them genetically engineered in a lab, and put them back to become personalized cancer-fighting machines.

The so-called CAR T cellular therapies are expected to receive federal approval this fall for certain rare blood cancers B-cell forms of lymphoma and leukemia. But scientists at the Minnesota institutions hope thats just the first step that will lead to better treatment of solid tumor cancers as well.

This is really the first approval of a genetically modified product for cancer therapy, said Dr. Jeffrey Miller, deputy director of the Masonic Cancer Center at the University of Minnesota. If the proof of concept works, he said, we might be on the right track to get away from all of that toxic chemotherapy that people hate.

Participating in industry-funded clinical trials, the Minnesota researchers hoped to determine if patients with leukemia or lymphoma would be more likely to survive if their own stem cells were extracted to grow cancer-fighting T-cells that were then infused back into their bodies.

One analysis, involving trials by Kite Pharmaceuticals at Mayo and other institutions, found a sevenfold increase in lymphoma patients whose cancers disappeared when they received CAR T instead of traditional chemo-based treatment.

I often tell patients that T-cells are like super robocops, said Dr. Yi Lin, a Mayo hematologist in Rochester. Were now directing those cells to really target cancer.

The U.S. Food and Drug Administration is widely expected this fall to approve CAR T products made by Kite and Novartis, which genetically engineer T-cells to target so-called CD19 proteins found on the surface of leukemia and lymphoma cells.

The side effects can be harsh, because the T-cell infusions trigger an immune system response that can produce fever, weakness, racing heart and kidney problems. Short-term memory and cognitive problems also have occurred. Brain swelling led to five deaths of cancer patients who took part in a CAR T trial by Juno Pharmaceuticals. The trial was shut down as a result.

Lin said brain swelling appeared mostly in adults with leukemia. For now, she expects Kites CAR T therapy to be approved for diffuse large B-cell lymphoma and the Novartis therapy to be approved for acute lymphoblastic leukemia in children. Federal regulations also might restrict CAR T for patients whose cancers survived traditional treatments.

Current practice to treat these cancers generally involves chemotherapy and radiation. Physicians then transplant stem cells, often from donor bone marrow, to regrow the patients immune systems, which are weakened in the process of treatment.

CAR T differs in that patients will receive infusions of their own T-cells, genetically modified, which their bodies will be less likely to reject.

Its individualized medicine, Lin said.

Im on my way

Before he tried CAR T at Mayo as part of a clinical trial, John Renze of Carroll, Iowa, had received two rounds of chemo, two rounds of radiation, and an experimental drug that did nothing to stop the spread of lymphoma.

After you fail about four times, you start to wonder if anything is going to work, the 58-year-old said.

At first, there was no room for him in the Mayo trial which has been a problem nationwide as desperate cancer patients have searched for treatment alternatives. But then he got the call one morning last summer while ordering coffee at his local cafe.

Can you get up here by one? the Mayo official asked.

Im on my way, Renze replied.

Even before federal approval comes through, researchers such as Miller are looking beyond the first-line CAR T therapies, and wondering if the approach can be used on solid tumors. Roughly 80,000 blood cancers occur each year in the U.S. that could be treated with CAR T, but the total number of cancers diagnosed each year is nearly 1.7 million.

The challenge is that solid tumors dont have the same protein targets as blood cancers. And T-cells would have to be more discriminating if infused to eliminate tumors in solid organs, Miller said. If you destroy normal lung tissue (along with lung cancer), thats not going to work, he said.

Mayo researchers are studying whether CAR T can work against multiple myeloma, a cancer of the bone marrow, while U researchers are exploring ways to better control the CAR T-cells after they are infused in cancer patients.

Researchers also are trying to understand whether CAR T produces memory in the immune system, so it knows to react if cancers resurface.

In addition, Miller is studying whether NK cells, which also play a role in the human immune system, can be genetically modified and infused instead of T-cells to target cancer. The body doesnt reject NK cells from donors as much, he said. So NK cells from donor bone marrow or umbilical cord blood could be collected and mass produced to potentially provide faster and cheaper treatments.

Like many breakthrough therapies, CAR T will be expensive, with a price likely to exceed $200,000 per patient. How insurers plan to cover it remains unclear. Blue Cross and Blue Shield of Minnesota is evaluating evidence regarding CAR Ts effectiveness, and will set a coverage policy after it receives FDA approval, said Dr. Glenn Pomerantz, Blue Cross chief medical officer.

A surge for Mayo?

Mayo expects a surge of hundreds of cancer patients per year if CAR T is approved, because it will initially be provided by large medical centers that have experience with the therapy and its side effects. The Rochester hospital is planning to add staff and space dedicated to CAR T.

Miller said the U is developing advice for referring doctors and hospitals statewide, so they know what to do if CAR T patients show up with complex symptoms.

They can be a bit delayed and you cant just keep people in the hospital to see if they develop these things, he said.

Renzes stem cells were taken last July, and his modified T-cells were put back a month later. He lost weight and felt sick for weeks, and had to drive three hours to Mayo for frequent checkups.

But as of last Aug. 31, the cancer had vanished.

Every three months, he returns to Mayo to make sure the cancer hasnt re-emerged. Then he returns to Carroll, where he owns farmland and car dealerships and dotes on his grandchildren.

For people like me that have already failed a bunch of times, youre happy to try anything, he said. I mean, what else would I have done?

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Mayo, U develop 'robocop' stem cells to fight cancer - StarTribune.com - Minneapolis Star Tribune

Bone Marrow Stem Cells Comments Off on Mayo, U develop ‘robocop’ stem cells to fight cancer – StarTribune.com – Minneapolis Star Tribune | August 20th, 2017

Representational image

Washington D.C. : A study has recently revealed that vitamin C may tell faulty stem cells in the bone marrow to mature and die normally, instead of multiplying to cause blood cancers.

According to researchers, certain genetic changes are known to reduce the ability of an enzyme called TET2 to encourage stem cells to become mature blood cells, which eventually die, in many patients with certain kinds of leukemia.

The new study found that vitamin C activated TET2 function in mice engineered to be deficient in the enzyme.

Corresponding study author Benjamin G. Neel said, "We're excited by the prospect that high-dose vitamin C might become a safe treatment for blood diseases caused by TET2-deficient leukemia stem cells, most likely in combination with other targeted therapies."

The results suggested that changes in the genetic code (mutations) that reduce TET2 function are found in 10 percent of patients with acute myeloid leukemia (AML), 30 percent of those with a form of pre-leukemia called myelodysplastic syndrome, and in nearly 50 percent of patients with chronic myelomonocytic leukemia.

The study results revolve around the relationship between TET2 and cytosine, one of the four nucleic acid "letters" that comprise the DNA code in genes.

To determine the effect of mutations that reduce TET2 function in abnormal stem cells, the team genetically engineered mice such that the scientists could switch the TET2 gene on or off.

The findings indicated that vitamin C did the same thing as restoring TET2 function genetically. By promoting DNA demethylation, high-dose vitamin C treatment induced stem cells to mature, and also suppressed the growth of leukemia cancer stem cells from human patients implanted in mice.

"Interestingly, we also found that vitamin C treatment had an effect on leukemic stem cells that resembled damage to their DNA," said first study author Luisa Cimmino.

"For this reason, we decided to combine vitamin C with a PARP inhibitor, a drug type known to cause cancer cell death by blocking the repair of DNA damage, and already approved for treating certain patients with ovarian cancer," Cimmino added.

The findings appear in journal Cell.

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Vitamin C may help genes to kill blood cancer stem cells - ETHealthworld.com

Bone Marrow Stem Cells Comments Off on Vitamin C may help genes to kill blood cancer stem cells – ETHealthworld.com | August 20th, 2017

Scientists recently used a gene-editing tool to fix a mutation in a human embryo. Around the world, researchers are chasing cures for other genetic diseases.

Now that the gene-editing genie is out of the bottle, what would you wish for first?

Babies with perfect eyes, over-the-top intelligence, and a touch of movie star charisma?

Or a world free of disease not just for your family, but for every family in the world?

Based on recent events, many scientists are working toward the latter.

Earlier this month, scientists from the Oregon Health & Science University used a gene editing tool to correct a disease-causing mutation in an embryo.

The technique, known as CRISPR-Cas9, fixed the mutation in the embryos nuclear DNA that causes hypertrophic cardiomyopathy, a common heart condition that can lead to heart failure or cardiac death.

This is the first time that this gene-editing tool has been tested on clinical-quality human eggs.

Had one of these embryos been implanted into a womans uterus and allowed to fully develop, the baby would have been free of the disease-causing variation of the gene.

This type of beneficial change would also have been passed down to future generations.

None of the embryos in this study were implanted or allowed to develop. But the success of the experiment offers a glimpse at the potential of CRISPR-Cas9.

Still, will we ever be able to gene-edit our world free of disease?

According to the Genetic Disease Foundation, there are more than 6,000 human genetic disorders.

Scientists could theoretically use CRISPR-Cas9 to correct any of these diseases in an embryo.

To do this, they would need an appropriate piece of RNA to target corresponding stretches of genetic material.

The Cas9 enzyme cuts DNA at that spot, which allows scientists to delete, repair, or replace a specific gene.

Some genetic diseases, though, may be easier to treat with this method than others.

Most people are focusing, at least initially, on diseases where there really is only one gene involved or a limited number of genes and theyre really well understood, Megan Hochstrasser, PhD, science communications manager at the Innovative Genomics Institute in California, told Healthline.

Diseases caused by a mutation in a single gene include sickle cell disease, cystic fibrosis, and Tay-Sachs disease. These affect millions of people worldwide.

These types of diseases, though, are far outnumbered by diseases like cardiovascular disease, diabetes, and cancer, which kill millions of people across the globe each year.

Genetics along with environmental factors also contribute to obesity, mental illness, and Alzheimers disease, although scientists are still working on understanding exactly how.

Right now, most CRISPR-Cas9 research focuses on simpler diseases.

There are a lot of things that have to be worked out with the technology for it to get to the place where we could ever apply it to one of those polygenic diseases, where multiple genes contribute or one gene has multiple effects, said Hochstrasser.

Although designer babies gain a lot of media attention, much CRISPR-Cas9 research is focused elsewhere.

Most people who are working on this are not working in human embryos, said Hochstrasser. Theyre trying to figure out how we can develop treatments for people that already have diseases.

These types of treatments would benefit children and adults who are already living with a genetic disease, as well as people who develop cancer.

This approach may also help the 25 million to 30 million Americans who have one of the more than 6,800 rare diseases.

Gene editing is a really powerful option for people with rare disease, said Hochstrasser. You could theoretically do a phase I clinical trial with all the people in the world that have a certain [rare] condition and cure them all if it worked.

Rare diseases affect fewer than 200,000 people in the United States at any given time, which means there is less incentive for pharmaceutical companies to develop treatments.

These less-common diseases include cystic fibrosis, Huntingtons disease, muscular dystrophies, and certain types of cancer.

Last year researchers at the University of California Berkeley made progress in developing an ex vivo therapy where you take cells out of a person, modify them, and put them back into the body.

This treatment was for sickle cell disease. In this condition, a genetic mutation causes hemoglobin molecules to stick together, which deforms red blood cells. This can lead to blockages in the blood vessels, anemia, pain, and organ failure.

Researchers used CRISPR-Cas9 to genetically engineer stem cells to fix the sickle cell disease mutation. They then injected these cells into mice.

The stem cells migrated to the bone marrow and developed into healthy red blood cells. Four months later, these cells could still be found in the mices blood.

This is not a cure for the disease, because the body would continue to make red blood cells that have the sickle cell disease mutation.

But researchers think that if enough healthy stem cells take root in the bone marrow, it could reduce the severity of disease symptoms.

More work is needed before researchers can test this treatment in people.

A group of Chinese researchers used a similar technique last year to treat people with an aggressive form of lung cancer the first clinical trial of its kind.

In this trial, researchers modified patients immune cells to disable a gene that is involved in stopping the cells immune response.

Researchers hope that, once injected into the body, the genetically edited immune cells will mount a stronger attack against the cancer cells.

These types of therapies might also work for other blood diseases, cancers, or immune problems.

But certain diseases will be more challenging to treat this way.

If you have a disorder of the brain, for example, you cant remove someones brain, do gene editing and then put it back in, said Hochstrasser. So we have to figure out how to get these reagents to the places they need to be in the body.

Not every human disease is caused by mutations in our genome.

Vector-borne diseases like malaria, yellow fever, dengue fever, and sleeping sickness kill more than 1 million people worldwide each year.

Many of these diseases are transmitted by mosquitoes, but also by ticks, flies, fleas, and freshwater snails.

Scientists are working on ways to use gene editing to reduce the toll of these diseases on the health of people around the world.

We could potentially get rid of malaria by engineering mosquitoes that cant transmit the parasite that causes malaria, said Hochstrasser. We could do this using the CRISPR-Cas9 technique to push this trait through the entire mosquito population very quickly.

Researchers are also using CRISPR-Cas9 to create designer foods.

DuPont recently used gene editing to produce a new variety of waxy corn that contains higher amounts of starch, which has uses in food and industry.

Modified crops may also help reduce deaths due to malnutrition, which is responsible for nearly half of all deaths worldwide in children under 5.

Scientists could potentially use CRISPR-Cas9 to create new varieties of food that are pest-resistant, drought-resistant, or contain more micronutrients.

One benefit of CRISPR-Cas9, compared to traditional plant breeding methods, is that it allows scientists to insert a single gene from a related wild plant into a domesticated variety, without other unwanted traits.

Gene editing in agriculture may also move more quickly than research in people because there is no need for years of lab, animal, and human clinical trials.

Even though plants grow pretty slowly, said Hochstrasser, it really is quicker to get [genetically engineered plants] out into the world than doing a clinical trial in people.

Safety and ethical concerns

CRISPR-Cas9 is a powerful tool, but it also raises several concerns.

Theres a lot of discussion right now about how best to detect so-called off-target effects, said Hochstrasser. This is what happens when the [Cas9] protein cuts somewhere similar to where you want it to cut.

Off-target cuts could lead to unexpected genetic problems that cause an embryo to die. An edit in the wrong gene could also create an entirely new genetic disease that would be passed onto future generations.

Even using CRISPR-Cas9 to modify mosquitoes and other insects raises safety concerns like what happens when you make large-scale changes to an ecosystem or a trait in a population that gets out of control.

There are also many ethical issues that come with modifying human embryos.

So will CRISPR-Cas9 help rid the world of disease?

Theres no doubt that it will make a sizeable dent in many diseases, but its unlikely to cure all of them any time soon.

We already have tools for avoiding genetic diseases like early genetic screening of fetuses and embryos but these are not universally used.

We still dont avoid tons of genetic diseases, because a lot of people dont know that they harbor mutations that can be inherited, said Hochstrasser.

Some genetic mutations also happen spontaneously. This is the case with many cancers that result from environmental factors such as UV rays, tobacco smoke, and certain chemicals.

People also make choices that increase their risk of heart disease, stroke, obesity, and diabetes.

So unless scientists can use CRISPR-Cas9 to find treatments for these lifestyle diseases or genetically engineer people to stop smoking and start biking to work these diseases will linger in human society.

Things like that are always going to need to be treated, said Hochstrasser. I dont think its realistic to think we would ever prevent every disease from happening in a human.

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Will Gene Editing Allow Us to Rid the World of Diseases? - Healthline

Bone Marrow Stem Cells Comments Off on Will Gene Editing Allow Us to Rid the World of Diseases? – Healthline | August 19th, 2017

ARZ050-051-059>061-070>073-LAZ001>006-010>014-017>022-TXZ097-151>153-165>167-192100-/O.NEW.KSHV.HT.Y.0009.170819T1500Z-170820T0000Z/Sevier-Howard-Little River-Hempstead-Nevada-Miller-Lafayette-Columbia-Union-Caddo-Bossier-Webster-Claiborne-Lincoln-De Soto-Red River-Bienville-Jackson-Ouachita-Sabine-Natchitoches-Winn-Grant-Caldwell-La Salle-Bowie-Panola-Nacogdoches-Shelby-Angelina-San Augustine-Including the cities of De Queen, Nashville, Mineral Springs, Dierks, Ashdown, Hope, Prescott, Texarkana, Stamps, Lewisville, Bradley, Magnolia, El Dorado, Shreveport, Bossier City, Minden, Springhill, Homer, Haynesville, Ruston, Farmerville, Bernice, Mansfield, Stonewall, Logansport, Coushatta, Martin, Arcadia, Ringgold, Gibsland, Jonesboro, Monroe, Many, Zwolle, Pleasant Hill, Natchitoches, Winnfield, Colfax, Montgomery, Dry Prong, Clarks, Grayson, Columbia, Jena, Midway, Olla, Carthage, Nacogdoches, Center, Lufkin, San Augustine, Hemphill, and Pineland242 AM CDT Sat Aug 19 2017...HEAT ADVISORY IN EFFECT FROM 10 AM THIS MORNING TO 7 PM CDTTHIS EVENING...The National Weather Service in Shreveport has issued a HeatAdvisory, which is in effect from 10 AM this morning to 7 PM CDTthis evening. * EVENT...High pressure across the area will allow for temperatures to climb into the mid 90s this afternoon. Hot temperatures combined with sufficient low-level moisture will allow for heat index values to climb to around 105 to 108 degrees across the advisory area.* TIMING...Heat index values will approach 105 degrees by late morning and persist through the afternoon into the early evening hours.* IMPACT...Precautions should be taken to prevent heat related illnesses, including limiting outdoor work activities to the late morning and early evening hours. PRECAUTIONARY/PREPAREDNESS ACTIONS...Take extra precautions if you work or spend time outside. Whenpossible, reschedule strenuous activities to early morning orevening. Know the signs and symptoms of heat exhaustion and heatstroke. Wear light weight and loose fitting clothing whenpossible and drink plenty of water. to reduce risk during outdoor work, the occupational safetyand health administration recommends scheduling frequent restbreaks in shaded or air conditioned environments. Anyone overcomeby heat should be moved to a cool and shaded location. Heatstroke is an emergency, call 9 1 1. a heat advisory means that a period of hot temperatures isexpected. The combination of hot temperatures and high humiditywill combine to create a situation in which heat illnesses arepossible. Drink plenty of fluids, stay in an air-conditionedroom, stay out of the sun, and check up on relatives andneighbors.&&$$

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Bone Marrow Transplant gives local cancer patient more time with his family - KTBS

Bone Marrow Stem Cells Comments Off on Bone Marrow Transplant gives local cancer patient more time with his family – KTBS | August 19th, 2017

WASHINGTON D.C: Good news! A study has recently revealed that vitamin C may tell faulty stem cells in the bone marrow to mature and die normally, instead of multiplying to cause blood cancers.

According to researchers, certain genetic changes are known to reduce the ability of an enzyme called TET2 to encourage stem cells to become mature blood cells, which eventually die, in many patients with certain kinds of leukemia.

The new study found that vitamin C activated TET2 function in mice engineered to be deficient in the enzyme.

Corresponding study author Benjamin G. Neel said, "We're excited by the prospect that high-dose vitamin C might become a safe treatment for blood diseases caused by TET2-deficient leukemia stem cells, most likely in combination with other targeted therapies."

The results suggested that changes in the genetic code (mutations) that reduce TET2 function are found in 10 percent of patients with acute myeloid leukemia (AML), 30 percent of those with a form of pre-leukemia called myelodysplastic syndrome, and in nearly 50 percent of patients with chronic myelomonocytic leukemia.

The study results revolve around the relationship between TET2 and cytosine, one of the four nucleic acid "letters" that comprise the DNA code in genes.

To determine the effect of mutations that reduce TET2 function in abnormal stem cells, the team genetically engineered mice such that the scientists could switch the TET2 gene on or off.

The findings indicated that vitamin C did the same thing as restoring TET2 function genetically. By promoting DNA demethylation, high-dose vitamin C treatment induced stem cells to mature, and also suppressed the growth of leukemia cancer stem cells from human patients implanted in mice.

"Interestingly, we also found that vitamin C treatment had an effect on leukemic stem cells that resembled damage to their DNA," said first study author Luisa Cimmino.

"For this reason, we decided to combine vitamin C with a PARP inhibitor, a drug type known to cause cancer cell death by blocking the repair of DNA damage, and already approved for treating certain patients with ovarian cancer," Cimmino added.

The findings appear in journal Cell.

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Vitamin C could help genes kill blood cancer stem cells - Economic Times

Bone Marrow Stem Cells Comments Off on Vitamin C could help genes kill blood cancer stem cells – Economic Times | August 19th, 2017