EDMONTON -- Thirteen months old with her health deteriorating in an orphanage in China, Hosanna Crowell was introduced to a Canadian couple, Greg and Cathy Crowell, who would prove to be game changers in her life.

"I remember when they first handed her to us in the orphanage and we looked at her and she had such determination and this frail, little body," remembers Cathy Crowell. "She was sucking her two little fingers, looking around and taking it all in. I said to my husband, she's a fighter."

Now 14, Hosanna has never stopped fighting. Born with a heredity condition called Beta Thalissemia Major, her bone marrow produces deformed blood cells, preventing oxygen from sticking to them. Without the blood of donors, her organs would be starved of oxygen. Every two weeks she visits the Stollery Children's Hospital where she receives her transfusions. To date, she's had 286. But with other people's blood, comes complications. Each night she's given intravenous drugs over 11 hours to keep her body working.

"Right now it's becoming a burden to me," says Hosanna Crowell. "I have to get poked so much my veins are becoming really scar tissued and it's starting to be really hard to find spots."

The only cure is a stem cell transplant. "In terms of any individual, a sibling will have a one-in-four chance of being a match for any individual," says pediatric hematologist Dr. Catherine Corriveau-Borque.

The journey to find a match has been years in the making. "It's like finding a needle on the bottom of the ocean. It's way harder than in a haystack," according to Crowell.

A post on the Chinese version of Facebook garnered a lot of attention, viewed more than 27 million times. "The process was quite something and then seeing the response from China with so many people and it going viral... wow," recounts Crowell from her Stony Plain home. "The kindness of strangers just so impacted us."

The posts reached Hosanna's biological family. Her mother and father as well as two siblings came forward, did the DNA testing and underwent a procedure to see if there was a match. "Yes," says Hosanna Crowell, "one of my siblings is a perfect match."

A stem cell transplant is now scheduled for late 2021. The cost to make this happen sits around $80,000 to cover incidentals such as travel visas, transportation, accommodation and COVID-19 testing. A GoFundMe campaign is a quarter of the way there.

"Really we're just trying to jump through all the little hoops to get them here," Crowell adds. "This is an amazing thing that's happened, we've been given a gift for our daughter and we're very grateful. I also feel for people who are waiting for a donor and so I just encourage people to go and get tested, it's a simple thing. You can change someone's life forever."

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'Like finding a needle on the bottom of the ocean': Local teen finds perfect bone marrow match - CTV News Edmonton

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SASKATOON -- An effort to increase stem cell donors within Black communities across Canada is being driven by a group of women whove had difficulty finding full genetic matches themselves.

Genetic matches are crucial for patients in need of stem cell transplants, such as those with leukemia and lymphoma, and matches are more commonly found within their own racial, ethnic and ancestral groups.

But the new Black Donors Save Lives campaign notes that fewer than two per cent of those in the Canadian Blood Services stem cell donor registry are Black.

And that decreases their chance of finding a match, campaign lead Sylvia Okonofua told CTVNews.ca in a phone interview. It becomes a numbers game for Black people on the stem cell waiting list, where its like finding a needle in a hay stack for them.

The recent University of Regina biochemistry graduate, with sights on becoming a hematologist, timed the virtual campaign to kick off during Black History Month.

It was overall frustrating to know that a patient from my community is so much less likely than other patients to be helped, she told CTVNews.ca. When you see that your people have a really, really low chance of being helped out, it takes you aback.

Okonofua noted part of the campaign uses TikToks, shareable infographics, and even an original song to get the message out and reach a wide audience.

And she said part of the outreach involves having Black stem cell recipients talk about their experiences with the health-care system and speak to the historical mistrust the Black community has towards the medical community.

She founded her campus chapter of Stem Cell Club, a non-profit organization with chapters across Canada which recruits Canadians as potential stem cell donors.

Registration for Black Donors Save Lives can be done online, where participants between the ages of 17 to 35 can fill out a questionnaire and have a swab kit mailed to their address. After they swab the inside of their cheeks and send the sample back, if there is a person in need, 90 per cent of donors will be asked to donate stem cells very similar to the way a person would be giving blood.

But a big difference is the donor is given a growth hormone a week before donation in order to increase the number of stem cells, as well as the process taking four to six hours.

Alternatively, one out of 10 donors will be asked if theyd like to donate stem cells via bone marrow surgery, which can take place over a day.

In 2017, Reve Agyepong experienced firsthand the lack of Black stem cell donors, to treat her sickle cell disease, which involve red blood cells becoming misshapen, which can block blood vessels and lead to damage to bones, brain, kidneys, and lungs, and can ultimately be fatal.

But Agyepong, who was born in Edmonton to Ghanaian parents, was fortunate to receive a stem cell transplant from her sister.

It is such a blessing to have a match within your own family because the percentages are just so low, she told CTVNews.ca by email. I am so fortunate to have found a match in my family or else transplant would have been off the table for me.

In fact, only one in four patients who need a stem cell transplant are able to find a matched donor within their family, with Black patients being less than half as likely as white patients to find a unrelated person they match with on a donor registry, according to the campaign.

For Jamaican-Canadian Dorothy Vernon-Brown, who helped inspire this months campaign, the current efforts are deeply personal. In 2013, she was diagnosed with acute myeloid leukaemia and was heartbroken to discover there were no stem cell matches in Canada's registry or internationally.

She ultimately received stem cells from her sister, who was a half-match, and has been spreading information to Black Canadians ever since, through her own advocacy group, Donor Drive for Dorothy.

Stem cell transplantation is a miracle for patients, and I wish people knew how easy it is to be a stem donor, she recounted on a Twitter thread for another stem cell awareness campaign. You could give someone an opportunity like my sister gave me, to be around and live the life I want. People want to live, so if that gift is in your hands, I appeal to you to see it as something significant to do in your life.

Okonofua and Vernon-Browns efforts are being aided by Dr. Warren Fingrut, a hematologist whos the director of the aforementioned Stem Cell Club.

He told CTVNews.ca in an email hes seen firsthand far too many patients from ethnic and racial minority groups in situations where they dont have fully-matched donors and are forced to seek other treatments.

I find this heart wrenching and I am very motivated to work to address this, Fingrut said.

That led to him founding his non-profit a decade ago, which has gone on to recruit more than 20,000 Canadians as stem cell donors, with more than 55 per cent being non-white. But in cases such as Vernon-Brown and others, those figures need to be much higher.

We started running national campaigns last year, focused on the recruitment of diverse peoples as donors, as well as males who are also preferred by transplant physicians (all else being equal) as they are associated with better outcomes for patients, Fingrut explained.

The campaign is also being done in partnership with several other groups, including the Katelyn Bedard Bone Marrow Association, Black Physicians of Canada, Black Medical Students Association of Canada and the National Black Law Students Association of Canada.

This campaign is one example of an initiative in the health-care sector, which seeks to address racial disparity impacting the care of Black patients, he wrote, noting Black people face many such disparities in access to care, and we want to see others in the health-care sector working with Black Canadians to tackle these issues and address them, in collaboration with Black communities.

Okonofua hopes next Black History Month, theyll be able to have in-person swabbing events in places of worship, community hubs, and cultural gatherings to show how easy it is.

Fingrut said this the first time his group has specifically engaged with one racial group and hopes to expand it to other ethnic and racial communities including South Asians, Indigenous peoples, and those of mixed ancestry in the near future.

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Meet the women hoping to recruit more stem cells donors from Black communities - CTV News

Bone Marrow Stem Cells Comments Off on Meet the women hoping to recruit more stem cells donors from Black communities – CTV News | February 19th, 2021

The life-changing phone call came at 4:30 in the morning on Christmas Eve.

All of a sudden, Ashley Peddle went from making final preparations for the holiday season to planning cancer treatments that had to start within days.

The East Greenwich resident, 37, had been experiencing fatigue, shortness of breath and headaches for some time, but when her husband Ryan encouraged her to get checked out, she chalked it up to being a busy mother of four kids 10 and younger. Finally, in the week leading up to Christmas, her symptoms became too much to bear as she could not even climb the steps in her house and was falling asleep on the couch around dinnertime.

She saw a doctor and later some troubling results of blood tests led to the early-morning phone call to get to the hospital immediately.

We rushed right to the emergency room and in about an hour or two, our world was kind of rocked with the diagnosis of leukemia, Ryan Peddle says. (Penn Medicine) was great and started her treatment right away because the type of leukemia she has is very aggressive, so the sooner she started, the better.

Two months and two rounds of chemotherapy after being told she has acute myeloid leukemia, Peddles prognosis is good, her husband said, but she is not out of the woods yet. She recently returned home after a six-week hospital stay but will soon go back to receive a stem cell transplant, also known as a bone marrow transplant, which will help restore her bone marrow, produce healthy blood cells and strengthen her immune system. In turn, she will be better suited to fighting the leukemia and preventing a reoccurrence down the line.

The best donors for stem cell transplants are usually a family member such as a sibling, but if they are not a match, an unrelated volunteer whose tissue type matches that of the patient may be used. Not only have Ashley and Ryan become educated about the process throughout their ordeal, but so have a group of Ashleys friends, who decided to take action when they learned how badly donors are needed.

Shawn Keating, also of East Greenwich, took the lead and helped organize a drive-thru bone marrow registration event in Peddles honor, which will be held this Saturday, Feb. 20, from 10 a.m. until 2 p.m., at Samuel Mickle School, 559 Kings Highway, in Mickleton. Sponsored by Be The Match which runs the largest and most diverse bone marrow registry in the world to help people battling blood cancers like leukemia and lymphoma the event will allow adults between the ages of 18 and 44 to join the national list of potential donors.

Ashley Peddle was diagnosed with leukemia and is in need of a bone marrow transplant. A registration event is being held Feb. 20 from 10 a.m. until 2 p.m. at Samuel Mickle School, 559 Kings Highway, in Mickleton.

In addition to supporting Peddle, who she has become friends with through their childrens sports and school activities, Keating had two other reasons for getting involved.

Her kids are the same age as mine and when you see this happen to somebody, you realize it could happen to anybody, she said. The other thing was, I went to sign myself up for Be The Match and I realized that at one point I had already requested the kit and in the busyness of things, I must have forgotten to send it back. So I reached out to them, got to talking and thought this was the best option, especially for busy people. Its as easy as driving through, getting swabbed and being done. I just thought it would be an easier way to get as many people on the registry as possible.

Bree Amborn works for an organization called The Icla da Silva Foundation, which is a recruitment center for Be The Match, and she will be running Saturdays event. She said people can expect a simple process to getting registered, as they just need to fill out some forms on their phone and do a swab in each cheek for 10 seconds without even leaving their car.

Those who cannot attend but still want to join Be The Match can text PeddleStrong to the number 61474 or visit join.bethematch.org/PeddleStrong and have a kit mailed to their home. Amborn added that it is especially important to increase the numbers of donors in the African-American community, as Black patients chances of finding a match are only 23%, compared to 77% for white patients.

The need is super, super high to add more people and to work in diverse communities and increase those populations on the registry as well, she said, so if a patient is a person of color they have the same chances of finding their match as if the patient is white.

Amborn will be able to answer any questions people have on Saturday. She was actually a donor herself while in college; after signing up she found out she was a match for a 50-year-old woman battling myelofibrosis.

It was an extremely easy process and Be The Match was awesome, she said. They pay for everything, they organize everything and they schedule everything, and as the donor you just show up. Youre literally able to change someones life.

I think people have a misconception of how the donation process works, but 80% of the time the donation is actually taken from your bloodstream. It looks very similar to donating platelets, where you have a needle in each arm, they take blood out of one arm, they separate your stem cells from your blood and give you your blood back in the other arm. Theres a couple more steps to the process but thats really the basics. Youre awake the whole time and its not a surgery.

Keating is hopeful that when people hear that, their fears will disappear and they will be eager to register. She doesnt know what to expect for Saturdays turnout, predicting, we could get 50 people or we could get 300. But already the response in East Greenwich has been impressive, with more than 50 volunteers slated to be in attendance.

Some, like Keating and Jacqueline DAngelis, are bringing their teenaged children to help as well.

Like I told my daughter, we cant cure cancer, but we can certainly help others by collecting these swabs, said DAngelis, a neighbor of the Peddles who has known them for seven years.

Theres going to be a lot of people from our community out there this weekend to support Ashley and support this mission to increase that number. Ashley is such a wonderful part of the community and its nice to see how everyone can band together and try to make something good out of this difficult and trying situation for her family. Its nice to see so many families parents and their teenagers coming out to volunteer in the cold in the middle of a pandemic to do something nice.

Although the Peddles have been quarantining since Ashley returned home because her immune system is compromised, Ryan plans on stopping by the event and showing his appreciation for the volunteers and those joining the list.

I didnt know anything about the registry beforehand or else Id have been on it, he said. Thats one of the reasons were pushing this drive, to get as many people on the registry as we can. They may not help Ashley but theyll help someone just like her.

He said his wifes spirits have remained high during her treatment. One of the toughest parts was when she was in the hospital and could not get in-person visits from their daughter and three sons, ages 10, 8, 7 and 4. They were able to FaceTime frequently, however, for much-needed emotional support.

The family is also grateful for the outpouring of assistance from those around them.

Were honestly overwhelmed by the support weve received, Ryan said. Our kids are pretty active in sports and other activities in the township, and through that weve made a lot of really good friendships and met a lot of really great people. So many of them are stepping up, not only for this drive but to give blood, to donate platelets, to cook meals, to drop off little things for the kids like Valentines. Its just been overwhelming and we couldnt be more proud of the town we live in.

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N.J. mom of 4 has leukemia. A bone marrow registry is being held Saturday. - nj.com

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In people with polycythemia vera (PV), the bone marrow produces too many blood cells. This overproduction can lead to complications, such as abnormal blood clotting, unusual bleeding, and an enlarged spleen.

In rare cases, scar tissue may replace the bone marrow. When this happens, the bone marrow can no longer produce enough healthy blood cells. Experts refer to this condition, which is a type of chronic leukemia, as myelofibrosis (MF). It can sometimes lead to acute myeloid leukemia, though this is rare.

People with PV have a shorter-than-average life expectancy. Some of the possible complications of the disease can be life threatening.

Getting treatment can help reduce the risk of certain complications from PV, including blood clots. As a result, a person will likely lead a longer and healthier life with this disease if they receive treatment.

According to an article in Blood Cancer Journal, the median survival time for people with PV is 14 years after diagnosis. The authors take this survival time from a study in which half of the participants were still alive 14 years after diagnosis.

Younger people tend to live for longer with the disease. Research suggests that the median survival time for those under 60 years of age is 24 years following diagnosis.

Multiple factors affect the outlook and life expectancy of people with PV, including:

Blood clots are the most common cause of death in people with PV. When blood clots form in blood vessels, they can block the flow of blood to vital organs. This can lead to life threatening complications, such as stroke, heart attack, and venous thrombosis.

Treatment for PV can help relieve symptoms and lower the risk of blood clots. In this way, it also reduces a persons risk of life threatening complications.

In most cases, healthcare providers prescribe regular blood draws to treat PV. Blood draws reduce the number of blood cells in the body, which may help improve blood flow.

Healthcare providers may also prescribe low dose aspirin to help prevent the formation of blood clots. Additionally, they may prescribe other medications, such as hydroxyurea (Hydrea) or busulfan (Myleran).

If a person develops MF as a complication of PV, their healthcare provider may prescribe one or more of the following treatments:

These treatments may help improve symptoms, increase life expectancy, or both.

For example, scientists have found that stem cell transplants may help improve long-term survival in people with MF. However, this treatment comes with a high risk of life threatening side effects. It is especially risky for older adults and people with other health conditions. As a result, healthcare providers often avoid prescribing this treatment.

Some studies have found that treatment with JAK inhibitors may also improve survival rates in people with MF. However, when scientists reviewed the available evidence on Jakafi and Inrebic, they found that the quality of evidence on survival rates is limited. More research is necessary to confirm how these treatments affect life expectancy.

Early research involving people with PV found that the median survival time for those who did not receive treatment was less than 2 years after diagnosis. This research took place before the medical community recognized blood draws as a treatment option, and it reflects the high risk of blood clots in people not receiving treatment.

People with PV who do not receive treatment are more likely to develop blood clots. According to the Leukemia & Lymphoma Society, 4060% of people with untreated PV may develop blood clots within 10 years of diagnosis.

Scientists have not yet developed a cure for PV. However, healthcare providers may prescribe blood draws, medications, or other treatments to help manage symptoms, reduce the risk of complications, and increase life expectancy in people with this disease.

Researchers are also continuing to develop and test potential new treatments for PV, such as the anticancer drug imatinib mesylate (Gleevec) and novel types of JAK inhibitors.

In some cases, a persons healthcare provider may encourage them to take part in a clinical trial. In this type of study, participants receive an experimental treatment. People interested in learning more about the potential benefits and risks of taking part in a clinical trial can talk with their healthcare provider or the researchers running the study.

When a person receives a diagnosis of PV, getting treatment is important. Treatment may help minimize symptoms, lower the risk of complications, and improve life expectancy.

A persons recommended treatment plan for PV will depend on many factors, including their age, overall health, and whether they have developed certain complications.

People with PV who wish to learn more about their treatment options and outlook should talk with their healthcare provider.

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Polycythemia vera life expectancy: With treatment and more - Medical News Today

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Novartis and the Bill & Melinda Gates Foundation have partnered on a single-dose, in vivo gene therapy for sickle cell disease (SCD). The Foundation will offer funding for development of the therapy.

Existing gene therapy approaches to sickle cell disease are difficult to deliver at scale and there are obstacles to reaching the vast majority of those affected by this debilitating disease, said Jay Bradner, a hematologist and president of the Novartis Institutes for BioMedical Research (NIBR). This is a challenge that calls for collective action, and we are thrilled to have the support of the Bill & Melinda Gates Foundation in addressing this global unmet medical need.

The announcement comes only a day after bluebird bio announced that it has placed its Phase I/II and Phase III trial of LentiGlobin gene therapy for sickle cell disease (SCD) on temporary suspension. The cause is a Suspected Unexpected Serious Adverse Reaction (SUSAR) of acute myeloid leukemia (AML).

HGB-206 is the companys ongoing Phase I/II trial of LentiGlobin for SCD. It includes three cohorts, A, B and C. In Group C, a refined manufacturing process designed to increase vector copy number was used.

Group C also received LentiGlobin for SCD manufactured from hematopoietic (blood) stem cells (HSCs) collected from peripheral blood after mobilization with plerixafor, instead of by way of bone marrow harvest, which was the method used in Groups A and B.

HGB-210 is their ongoing Phase III single-arm open-label trial. It is evaluating efficacy and safety of LentiGlobin for SCD in patients between two years and 50 years of age with sickle cell disease.

Which underlines that even though gene therapy is making headway, it is still a cutting-edge technology.

SCD is a hereditary blood disease that affects millions of people globally, with more than 300,000 born with it each year. It primarily affects people of African descent, and sub-Saharan Africa bears about 80% of the disease burden. It affects the structure of red blood cells, causing a distinct sickle shape, which decreases the ability of red blood cells to transport oxygen efficiently.

Gene therapies might help end the threat of diseases like sickle cell, but only if we can make them far more affordable and practical for low-resource settings, said Trevor Mundel, president of Global Health at the Gates Foundation. Whats exciting about this project is that it brings ambitious science to bear on that challenge. Its about treating the needs of people in lower-income countries as a driver of scientific and medical progress, not an afterthought. It also holds the promise of applying lessons learned to help develop potentially curative options for other debilitating diseases affecting low-income populations, such as HIV.

Novartis also announced today that the U.S. Food and Drug Administration (FDA) approved the expanded indication for Entresto (sacubitril/valsartan) to decrease the risk of cardiovascular death and hospitalization for heart failure in adults with chronic heart failure. The biggest benefit was for patients with left ventricular ejection fraction (LVEF) below normal.

The expansion was based on data in the PARAGON-HF Phase III trial.

This approval is a significant advancement, providing a treatment to many patients who were not eligible for treatment before because their ejection fraction was above the region we normally considered reduced, said Scott Solomon, professor of Medicine at Harvard Medical School and Brigham and Womens Hospital, and PARAGON-HF Executive Committee co-chair. We can now offer a treatment to a wider range of patients who have an LVEF below normal.

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Novartis and Gates Foundation Team Up To Deliver Affordable Sickle Cell Gene Therapy - BioSpace

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A desperate family is facing a nervous wait after a stem cell donor finally found to give their four-year-old daughter a new chance at life fell ill.

Little Adeline Davidson has been waiting more than two years for the procedure to treat a rare form of blood cancer, and several arrangements with donors have fallen through during that time.

The Alness youngster had been due to go under the knife in Glasgow this week after it appeared that the search for a match had eventually come to an end.

But the family encountered yet another setback as the procedure was cancelled when the stem cell donor fell ill.

Adelines parents Steph, 26, and Jordan, 28, say their daughters transplant now hangs in the balance as they face an agonising wait to find out what is wrong with the donor.

They say the plans could be thrown into disarray with top level talks and a possible world-first procedure required if tests show that the illness is Covid-related.

The family will only be able to find out the nature of the donors condition after he is operated on and the cells removed.

If the donor has coronavirus, a team of international surgeons will assemble to debate whether it would be safe for Adeline to undergo the transplant.

Mrs Davidson said: We have been waiting more than two whole years for our ill child to get a bone marrow transplant.

The hospital have told me that the donor has to donate, and then they release the information on the cause of his illness.

If it is Covid, that would mean they would have to ask international doctors and surgeons if they could go ahead.

They have never given a child thats Covid-negative marrow from someone who is Covid-positive.

If they decide not to proceed, we are back to looking for someone else to begin the search again, which is just a crazy, horrible thought. I dont even want to think about it.

Mrs Davidson added that she would consider going ahead even if the cells have come from someone with coronavirus.

She said: I think we have to go with the doctors word, but Id be so frightened.

We wouldnt have another choice though, unfortunately.

If they, as professionals, believe doing it would outweigh the risks, we would just have to believe that too.

Over the last two years, Adeline has endured around 85 blood transfusions, one anaphylactic shock and emergency helicopter and ambulance transfers to hospital.

Mrs Davidosn added: There is potential for even worse news but we just hope that it isnt Covid he has.

If it isnt Covid, then everything moves along as it was meant to be.

We are aware that on the register there was no-one else, so we were lucky this guy popped up.

If all goes well, Adeline will receive her transplant in four weeks.

The latest setback comes almost two years to the day since her transplant journey began.

In December, the family were dealt a devastating blow as health officials postponed her procedure, scheduled to take place in January, due to Brexit complications.

The four-year-old requires a specific type off marrow, processed by a centre outwith the UK, which is then brought to the country by road.

Life-saving transplant for Highland youngster postponed due to delays caused by Brexit

Early last year, the family were forced to turn to the register and launch a public appeal in search for multiple new donors due to an array of complications.

Mrs Davidson praised Adelines resilience but admits it breaks her heart to not be able to see her daughter progress onto school in August.

She said: The first year I was so positive lets get on with it, this needs done and I never thought why us?

I just thought we have so much to be grateful for and thankful for.

However, the whole of the second year, Im just thinking is someone messing with us because thats what it feels like.

She added: Adelines been so good. She hardly complains and I just think its because she has no idea whats shes missing, which is sort of a good thing but sad.

She is lucky thats shes an outgoing kid. She is behind, she has not socialised and although she is switched on, shes probably not as far on as her peers.

Even now, she should have been staring primary one in August this year but shes not even been to nursery. Its hellish.

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Heartbreak for family of cancer-stricken four-year-old as stem cell donor falls ill at last minute - Press and Journal

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This article was originally published here

Front Immunol. 2021 Jan 21;11:594572. doi: 10.3389/fimmu.2020.594572. eCollection 2020.

ABSTRACT

Mycobacterium tuberculosis (Mtb), the causative organism of pulmonary tuberculosis (PTB) now infects more than half of the world population. The efficient transmission strategy of the pathogen includes first remaining dormant inside the infected host, next undergoing reactivation to cause post-primary tuberculosis of the lungs (PPTBL) and then transmit via aerosol to the community. In this review, we are exploring recent findings on the role of bone marrow (BM) stem cell niche in Mtb dormancy and reactivation that may underlie the mechanisms of PPTBL development. We suggest that pathogens interaction with the stem cell niche may be relevant in potential inflammation induced PPTBL reactivation, which need significant research attention for the future development of novel preventive and therapeutic strategies for PPTBL, especially in a post COVID-19 pandemic world. Finally, we put forward potential animal models to study the stem cell basis of Mtb dormancy and reactivation.

PMID:33584661 | PMC:PMC7873989 | DOI:10.3389/fimmu.2020.594572

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Initiation of Post-Primary Tuberculosis of the Lungs: Exploring the Secret Role of Bone Marrow Derived Stem Cells - DocWire News

Bone Marrow Stem Cells Comments Off on Initiation of Post-Primary Tuberculosis of the Lungs: Exploring the Secret Role of Bone Marrow Derived Stem Cells – DocWire News | February 17th, 2021

REDWOOD CITY, Calif.--(BUSINESS WIRE)--Jasper Therapeutics, Inc., a biotechnology company focused on hematopoietic cell transplant therapies, today announced the launch of a Phase 1/2 clinical trial to evaluate JSP191, Jaspers first-in-class anti-CD117 monoclonal antibody, as a targeted, non-toxic conditioning regimen prior to allogeneic transplant for sickle cell disease (SCD). Jasper Therapeutics and the National Heart, Lung, and Blood Institute (NHLBI) have entered into a clinical trial agreement in which NHLBI will serve as the Investigational New Drug (IND) sponsor for this study.

SCD is a lifelong inherited blood disorder that affects hemoglobin, a protein in red blood cells that delivers oxygen to tissues and organs throughout the body. Approximately 300,000 infants are born with SCD annually worldwide, and the number of cases is expected to significantly increase. Currently, hematopoietic stem cell transplantation (HSCT) is the only cure available for SCD.

"This clinical trial agreement with the NHLBI expands the development of JSP191 for transplant conditioning and could bring curative transplants to more patients in need," said Kevin N. Heller, M.D., Executive Vice President, Research and Development, of Jasper Therapeutics. "We look forward to collaborating with the NHLBI and learning more about the potential for JSP191 in patients living with sickle cell disease."

About JSP191

JSP191 (formerly AMG 191) is a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from the bone marrow. JSP191 binds to human CD117, a receptor for stem cell factor (SCF) that is expressed on the surface of hematopoietic stem and progenitor cells. The interaction of SCF and CD117 is required for stem cells to survive. JSP191 blocks SCF from binding to CD117 and disrupts critical survival signals in stem cells leading to cell death. This creates space in the bone marrow for engraftment of donor or gene-corrected transplanted stem cells.

Preclinical studies have shown that JSP191, as a single agent, safely depletes normal and diseased hematopoietic stem cells, including in animal models of severe combined immunodeficiency (SCID), myelodysplastic syndromes (MDS), and sickle cell disease (SCD). Treatment with JSP191 creates the space needed for transplanted normal donor or gene-corrected hematopoietic stem cells to successfully engraft in the host bone marrow. To date, JSP191 has been evaluated in more than 90 healthy volunteers and patients.

JSP191 is currently being evaluated in two separate Jasper Therapeutics-sponsored clinical studies in hematopoietic cell transplant. The first clinical study is evaluating JSP191 as a sole conditioning agent in a Phase 1/2 dose-escalation and expansion trial to achieve donor stem cell engraftment in patients undergoing hematopoietic cell transplant for SCID. Blood stem cell transplantation offers the only potentially curative therapy for SCID. JSP191 is also being evaluated in combination with another conditioning regimen in a Phase 1 study in patients with MDS or acute myeloid leukemia (AML) who are receiving hematopoietic cell transplant. For more information about the design of these clinical trials, visit http://www.clinicaltrials.gov (NCT02963064 and NCT04429191).

Additional studies are planned to advance JSP191 as a conditioning agent for patients with other rare and ultra-rare monogenic disorders and autoimmune diseases.

About Jasper Therapeutics

Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell. The companys lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplant. This first-in-class conditioning antibody is designed to enable safer and more effective curative hematopoietic cell transplants and gene therapies. For more information, please visit us at jaspertherapeutics.com.

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Jasper Therapeutics Announces Launch of New Clinical Trial with National Heart, Lung, and Blood Institute to Evaluate JSP191 in Sickle Cell Disease -...

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Transparency Market Research (TMR) has published a new report titled, Cord Blood Banking Services Market Global Industry Analysis, Size, Share, Growth, Trends, and Forecast, 20192027. According to the report, the globalcord blood banking services marketwas valued atUS$ 25.8 Mnin2018and is projected to expand at a CAGR of10.9%from2019to2027.

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High Incidence of genetic disorders and rise in hematopoietic stem cell transplantation rates to Drive Market

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Private Cord Blood Bank Segment to Dominate Market

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North America to Dominate Global Market

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Cord Blood Banking Services Market projected to expand at a CAGR of 10.9% from 2019 to 2027 KSU | The Sentinel Newspaper - KSU | The Sentinel...

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Novartis and the Bill and Melinda Gates Foundation are joining forces to discover and develop a gene therapy to cure sickle cell disease with a one-step, one-time treatment that is affordable and simple enough to treat patients anywhere in the world, especially in sub-Saharan Africa where resources may be scarce but disease prevalence is high.

The three-year collaboration, announced Wednesday, has initial funding of $7.28 million.

Current gene therapy approaches being developed for sickle cell disease are complex, enormously expensive, and bespoke, crafting treatments for individual patients one at a time. The collaboration aims to instead create an off-the-shelf treatment that bypasses many of the steps of current approaches, in which cells are removed and processed outside the body before being returned to patients.

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Sickle cells cause is understood. The people it affects are known. But its cure has been elusive, Jay Bradner, president of the Novartis Institutes for BioMedical Research, told STAT.

We understand perfectly the disease pathway and the patient, but we dont know what it would take to have a single-administration, in vivo gene therapy for sickle cell disease that you could deploy in a low-resource setting with the requisite safety and data to support its use, he said. Im a hematologist and can assure you that in my experience in the clinic, it was extremely frustrating to understand a disease so perfectly but have so little to offer.

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Sickle cell disease is a life-threatening inherited blood disorder that affects millions around the world, with about 80% of affected people in sub-Saharan Africa and more than 100,000 in the U.S. The mutation that causes the disease emerged in Africa, where it protects against malaria. While most patients with sickle cell share African ancestry, those with ancestry from South America, Central America, and India, as well as Italy and Turkey, can also have the hereditary disease.

The genetic mutation does its damage by changing the structure of hemoglobin, hampering the ability of red blood cells to carry oxygen and damaging blood vessels when the misshapen cells get stuck and block blood flow. Patients frequently suffer painful crises that can be fatal if not promptly treated with fluids, medication, and oxygen. Longer term, organs starved of oxygen eventually give out. In the U.S., that pain and suffering is amplified when systemic and individual instances of racism deny Black people the care they need.

Delivering gene therapy for other diseases has been costly and difficult even in the best financed, most sophisticated medical settings. Challenges include removing patients cells so they can be altered in a lab, manufacturing the new cells in high volume, reinfusing them, and managing sometimes severe responses to the corrected cells. Patients also are given chemotherapy to clear space in their bone marrow for the new cells.

Ideally, many of those steps could be skipped if there were an off-the-shelf gene therapy. That means, among other challenges, inventing a way to eliminate the step where each patients cells are manipulated outside the body and given back the in vivo part of the plan to correct the genetic mutation.

Thats not the only obstacle. For a sickle cell therapy to be successful, Bradner said, it must be delivered only to its targets, which are blood stem cells. The genetic material carrying corrected DNA must be safely transferred so it does not become randomly inserted into the genome and create the risk of cancer, a possibility that halted a Bluebird Bio clinical trial on Tuesday. The payload itself mustnt cause such problems as the cytokine storm of immune overreaction. And the intended response has to be both durable and corrective.

In a way, the gene delivery is the easy part because we know that expressing a normal hemoglobin, correcting the mutated hemoglobin, or reengineering the switches that once turned off normal fetal hemoglobin to turn it back on, all can work, Bradner said. The payload is less a concern to me than the safe, specific, and durable delivery of that payload.

For each of these four challenges delivery, gene transfer, tolerability, durability there could be a bespoke technical solution, Bradner said. The goal is to create an ensemble form of gene therapy.

Novartis has an existing sickle-cell project using CRISPR with the genome-editing company Intellia, now in early human trials, whose lessons may inform this new project. CRISPR may not be the method used; all choices are still on the table, Bradner said.

Vertex Pharmaceuticals has seen encouraging early signs with its candidate therapy developed with CRISPR Therapeutics. Other companies, including Beam Therapeutics, have also embarked on gene therapy development.

The Novartis-Gates collaboration is different in its ambition to create a cure that does not rely on an expensive, complicated framework. Novartis has worked with the Gates Foundation on making malaria treatment accessible in Africa. And in October 2019, the Gates Foundation and the National Institutes of Health said together they would invest at least $200 million over the next four years to develop gene-based cures for sickle cell disease and HIV that would be affordable and available in the resource-poor countries hit hardest by the two diseases, particularly in Africa.

Gene therapies might help end the threat of diseases like sickle cell, but only if we can make them far more affordable and practical for low-resource settings, Trevor Mundel, president of global health at the Gates Foundation, said in a statement about the Novartis collaboration. Its about treating the needs of people in lower-income countries as a driver of scientific and medical progress, not an afterthought.

Asked which is the harder problem to solve: one-time, in vivo gene therapy, or making it accessible around the world, David Williams, chief of hematology/oncology at Boston Childrens Hospital, said: Both are going to be difficult to solve. The first will likely occur before the therapy is practically accessible to the large number of patients suffering the disease around the world.

Williams is also working with the Gates Foundation, as well as the Koch Institute for Integrative Cancer Research at MIT, Dana-Farber Cancer Institute, and Massachusetts General Hospital, on another approach in which a single injection of a reagent changes the DNA of blood stem cells. But there are obstacles to overcome there, too, that may be solved by advances in both the technology to modify genes and the biological understanding of blood cells.

Bradner expects further funding to come to reach patients around the world, once the science progresses more.

There is no plug-and-play solution for this project in the way that mRNA vaccines were perfectly set up for SARS-CoV-2. We have no such technology to immediately redeploy here, he said. Were going to have to reimagine what it means to be a gene therapy for this project.

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Novartis, Gates Foundation pursue a simpler gene therapy for sickle cell - STAT

Bone Marrow Stem Cells Comments Off on Novartis, Gates Foundation pursue a simpler gene therapy for sickle cell – STAT | February 17th, 2021

RESEARCH TRIANGLE PARK After 13 years as a clinical-stage oncology company,G1 Therapeuticsof Research Triangle Park transformed into a commercial-stage company overnight upon the approval of its first drug by the U.S. Food and Drug Administration.

The FDA on Feb. 12 approved G1s trilaciclib, to be marketed as Cosela, for protecting bone marrow from chemotherapy damage in adult patients with extensive-stage small cell lung cancer (ES-SCLC).

Cosela will help change the chemotherapy experience for people who are battling ES-SCLC, said Jack Bailey, the companys chief executive officer. G1 is proud to deliver Cosela to patients and their families as the first and only therapy to help protect against chemotherapy-induced myelosuppression.

Myelosuppression, or damage to the bone marrow, is the most serious and life-threatening side effect of chemotherapy. Chemotherapy-induced myelosuppression reduces the bodys essential supply of white blood cells, red blood cells and platelets, and can lead to increased risks of infection, severe anemia and bleeding.

RTP drug firm G1 secures FDA approval for treatment to prevent chemo damage to bone marrow

These complications impact patients quality of life and may also result in chemotherapy dose reductions and delays, said Jeffrey Crawford,M.D., Geller Professorfor Research in Cancer in theDepartment of MedicineandDuke Cancer Institute. In clinical trials, the addition of trilaciclib to extensive-stage small cell lung cancer chemotherapy treatment regimens reduced myelosuppression and improved clinical outcomes.The good news is that these benefits of trilaciclib will now be available for our patients in clinical practice.

Cosela is expected to be commercially available through G1s specialty distributor partner network in early March, the company said.

G1 is committed to helping patients with in theU.S.gain access to treatment with Cosela through access and affordability programs. Patients and healthcare can call the companys support center at 833-418-6663 for information.

Cosela is intended to be given as a 30-minute infusion four hours prior to chemotherapy treatments containing platinum/etoposide or topotecan. About 90 percent of all patients with ES-SCLC receive at least one of these chemotherapy regimens during their treatment, according to G1.

The approval of Cosela is based on data from three randomized, placebo-controlled trials. Data showed that patients receiving Cosela before the start of chemotherapy had less neutropenia, an abnormally low number of neutrophils, white blood cells that fight bacterial and fungal infection.

Data also showed a positive impact on red blood cell transfusions and other myeloprotective measures.

Chemotherapy is the most effective and widely used approach to treating people diagnosed with extensive-stage small cell lung cancer, Bailey said. However, standard-of-care chemotherapy regimens are highly myelosuppressive and can lead to costly hospitalizations and rescue interventions.

To date, oncologists have relied on rescue therapy, a mix of growth factor agents, antibiotics and red blood cell transfusions, to restore bone marrow after it has been damaged by chemotherapy.

By contrast, trilaciclib provides the first proactive approach to myelosuppression through a unique mechanism of action that helps protect the bone marrow from damage by chemotherapy, Crawford said.

Cosela helps protect bone marrow cells from chemotherapy damage by inhibiting cyclin- dependent kinase 4 and 6, two enzymes involved in cancer cell growth. Inhibiting these enzymes temporarily stops hematopoietic stem cells and progenitor cells in the bone marrow from dividing, making them resistant to damage from chemotherapy drugs that target dividing cells.

Bonnie J. Addario, lung cancer survivor, co-founder and board chair of theGo2 Foundation for Lung Cancer, said many people with extensive-stage small cell lung cancerrely on chemotherapy to extend their lives and alleviate their symptoms.

Unfortunately, the vast majority will experience chemotherapy-induced side effects, resulting in dose delays and reductions, and increased utilization of healthcare services, she said.

G1 shares our organizations goal to improve the quality of life of those diagnosed with lung cancer and to transform survivorship among people living with this insidious disease. We are thrilled to see new advancements that can help improve the lives of those living with small cell lung cancer.

About 30,000 small cell lung cancer patients are treated inthe United Statesannually. SCLC, one of the two main types of lung cancer, accounts for about 10 to 15 percent of all lung cancers but is the more aggressive disease, tending to grow and spread faster than the other type, non-small cell lung cancer.

InJune 2020, G1 announced a three-yearco-promotion agreementwithBoehringer Ingelheimfor Cosela in small cell lung cancer in theU.S.andPuerto Rico. G1 will lead marketing, market access and medical engagement initiatives for Cosela whileBoehringer Ingelheimsoncology commercial team will lead sales force engagement initiatives.

G1 will book revenue and retain development and commercialization rights to Cosela and payBoehringer Ingelheima promotional fee based on net sales.

The three-year agreement does not extend to additional indications that G1 is evaluating for trilaciclib: breast, colorectal, bladder and non-small cell lung cancers.

G1 is a 2008 spin-out of the University of North Carolina at Chapel Hill.

The company raised $108 million in an initial public offering of stock in 2017 after receiving more than $95 million in three rounds of venture capital funding. The North Carolina Biotechnology Center provided two early-stage loans totaling $500,000.

G1s stock is traded on the Nasdaq Global Select Market under the ticker symbol GTHX.

(C) N.C. Biotech Center

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After 13 years of trials and tribulations RTP firm G1 wins first FDA approval for cancer drug - WRAL Tech Wire

Bone Marrow Stem Cells Comments Off on After 13 years of trials and tribulations RTP firm G1 wins first FDA approval for cancer drug – WRAL Tech Wire | February 17th, 2021

Black patients in need of bone marrow or blood stem cell treatments have a decreased chance of matching with a donor. The Seattle branch hopes to change that.

Seattles Be The Match Collection Center opened up less than a year ago and is celebrating its 100th blood cell donation with an important message: More bone marrow donors of color are needed.

The nonprofit donation center is a part of the National Marrow Donor Program and increases the capacity to collect blood cells in the Pacific Northwest. Seattles Clinical Manager Hannah Erskine said this month is an important time to focus on the donation gap.

In the midst of Black History Month, its important to note that we frankly dont have enough Black and African American donors on the registry, said Erskin.

Only 4% of approximately 22 million donors on the registry are African American, lowering the chances that a Black patient can find a bone marrow donor who is a genetic match.

According to Be The Match data, the likelihood of finding a matched adult donor is only around 23% for an African American or Black patient, versus a 77% match rate for a white patient.

These matched bone marrow or blood stem cell transplants can help cure blood cancers like leukemia and lymphoma, as well as other blood conditions, such as sickle cell disease. Be The Match has coordinated more than 100,000 transplants.

Erskine said registering is a simple mouth swab that will be mailed to potential donors. They will be contacted if they are a match with a patient.

Being a matching blood stem cell donor can potentially save a life. The first step in changing the trend is to join the registry at http://www.bethematch.org.

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Be The Match encourages people of color to join bone marrow registry - KING5.com

Bone Marrow Stem Cells Comments Off on Be The Match encourages people of color to join bone marrow registry – KING5.com | February 14th, 2021

Leukemia is a type of cancer that affects the blood and bone marrow, where blood cells are formed. All types of leukemia cause rapid, uncontrolled growth of abnormal bone marrow and blood cells.

The main differences between the types include how fast the disease progresses and the types of cells it affects.

There are four main types of leukemia, which we describe in detail below:

Lymphocytic leukemia affects the lymphocytes, a type of white blood cell. Myeloid leukemia can affect the white blood cells, red blood cells, and platelets.

According to the National Cancer Institute, roughly 1.5% of people in the United States will receive a leukemia diagnosis at some point.

In this article, explore the four main types, their symptoms, the treatment options available, and the outlook.

The full name of this type of cancer is acute lymphocytic leukemia, and acute means that it grows quickly. Lymphocytic means that it forms in underdeveloped white blood cells called lymphocytes.

The disease starts in the bone marrow, which produces stem cells that develop into red and white blood cells and platelets.

In a healthy person, the bone marrow does not release these cells until they are fully developed. In someone with ALL, the bone marrow releases large quantities of underdeveloped white blood cells.

There are several subtypes of ALL, and the subtype may influence the best course of treatment and the prognosis.

One subtype is B-cell ALL. This begins in the B lymphocytes, and it is the most common form of ALL in children.

Another subtype is T-cell ALL. It can cause the thymus, a small organ at the front of the windpipe, to become enlarged, which can lead to breathing difficulties.

Overall, because ALL progresses quickly, swift medical intervention is key.

As research from 2020 acknowledges, healthcare providers still do not know what causes ALL. It may occur due to genetic factors or exposure to:

Although genetic factors may play a role, ALL is not a familial disease.

Learn more about ALL here.

ALL is the most common form of leukemia in children.

The risk of developing it is highest in children under 5 years old. The prevalence slowly rises again in adults over 50.

ALL symptoms can be nonspecific difficult to distinguish from those of other illnesses.

They may include:

In a person with AML, the bone marrow makes abnormal versions of platelets, red blood cells, and white blood cells called myeloblasts.

The full name of this disease is acute myeloid leukemia, and acute refers to the fact that it is fast-growing.

It forms in one of the following types of bone marrow cell:

Doctors classify AML by subtype, depending on:

AML can be difficult to treat and requires prompt medical attention.

Learn more about AML here.

The most common risk factor is myelodysplastic syndrome, a form of blood cancer that keeps the body from producing enough healthy blood cells.

Other factors that increase the risk of developing AML include:

Most people who develop AML are over 45. It is one of the most common types of leukemia in adults, though it is still rare, compared with other cancers.

It is also the second most common form of leukemia in children.

Symptoms of AML can vary and may include:

CLL is the most common form of leukemia among adults in the U.S. and other Western countries.

There are two types. One progresses slowly, and it causes the body to have high levels of characteristic lymphocytes, but only slightly low levels of healthy red blood cells, platelets, and neutrophils.

The other type progresses more quickly and causes a significant reduction in levels of all healthy blood cells.

In someone with CLL, the lymphocytes often look fully formed but are less able to fight infection than healthy white blood cells. The lymphocytes tend to build up very slowly, so a person might have CLL for a long time before experiencing symptoms.

Learn more about CLL here.

Genetic factors are the most likely cause. Others might include:

CLL is rare in children. It typically develops in adults aged 70 or over. However, it can affect people as young as 30.

CLL typically causes no early symptoms. When symptoms are present, they may include:

Also, 5090% of people with CLL have swollen lymph nodes.

CML is a slow-growing type of leukemia that develops in the bone marrow.

The full name of CML is chronic myeloid leukemia. As the American Cancer Society explain, a genetic change takes place in the early forms of the myeloid cells, and this eventually results in CML cells.

These leukemia cells then grow, divide, and enter the blood.

CML occurs due to a rearrangement of genetic material between the chromosomes 9 and 22.

This rearrangement fuses a part of the ABL1 gene from chromosome 9 with the BCR gene from chromosome 22, called the Philadelphia chromosome. The result of this fusion is called BCR-ABL1.

BCR-ABL1 produces a protein that promotes cell division and stops apoptosis, the process of cell death, which typically removes unneeded or damaged cells.

The cells keep dividing and do not self-destruct, resulting in an overproduction of abnormal cells and a lack of healthy blood cells.

This occurs during the persons lifetime and is not inherited.

CML typically affects adults. People aged 65 and older make up almost half of those who receive a CML diagnosis.

The symptoms of CML are unclear, but they may include:

The symptoms may vary, depending on the type of leukemia. Overall, a person should get in touch with a doctor if they experience:

Learn more about the symptoms of leukemia here.

Treatment for ALL typically involves three basic phases: induction, consolidation, and maintenance. We describe these in detail below.

Treatment for AML involves the first two phases. The induction phase may include treatment with the chemotherapy drugs cytarabine (Cytosar-U) and daunorubicin (Cerubidine) or idarubicin (Idamycin). The doctor may also recommend targeted drugs.

The goal of this phase is to kill the leukemia cells, causing the cancer to go into remission, using chemotherapy.

The doctor may recommend:

People having chemotherapy may need to see their doctors frequently and spend time in the hospital, due to the risk of serious infections and complications.

This phase of the treatment lasts for about 1 month.

Even if the treatment so far has led to remission, cancer cells may be hiding in the body, so more treatment is necessary.

The consolidation phase may involve taking high doses of chemotherapy. A doctor may also recommend targeted drugs or stem cell transplants.

This phase, consisting of ongoing chemotherapy treatments, usually lasts for 2 years.

Since CLL tends to progress slowly, and its treatment can have unpleasant side effects, some people with this condition go through a phase of watchful waiting before starting the treatment.

For a person with CML, the focus is often on providing the right treatment for the phase of the illness. To do this, a doctor considers how quickly the leukemia cells are building up and the extent of the symptoms. Stem cell transplants can be effective, but further treatment is necessary.

Overall, the initial treatment tends to include monoclonal antibodies, targeted drugs, and chemotherapy.

If the only concern is an enlarged spleen or swollen lymph nodes, the person may receive radiation or surgery.

If there are high numbers of CLL cells, the doctor may suggest leukapheresis, a treatment that lowers the persons blood count. This is only effective for a short time, but it allows the chemotherapy to start working.

For people with high-risk disease, doctors may recommend stem cell transplants.

A persons prognosis depends on the type of leukemia.

Learn more about survival rates for people with leukemia here.

About 8090% of adults with ALL experience complete remission for a while during treatment. And with treatment, most children recover from the disease.

Relapses are common in adults, so the overall cure rate is 40%. However, factors specific to each person play a role.

The older a person is when they receive an AML diagnosis, the more difficult it is to treat.

More than 25% of adults who achieve remission live for 3 years or more after treatment for AML.

A person may live for a long time with CLL.

Treatments can help keep the symptoms under control and prevent the disease from spreading. However, there is no cure.

Stem cell transplants can cure CML. However, this treatment is very invasive and is not suitable for most people with CML.

The United Kingdoms National Health Service estimate that 70% of males and 75% of females live for at least 5 years after receiving a CML diagnosis.

The earlier a person receives the diagnosis, the better their outlook.

Leukemia is a type of cancer that affects the blood and bone marrow. It can affect people of all ages.

There are four main types of leukemia. They differ based on how quickly they progress and the types of cells they affect.

Treatments for all types of leukemia continue to improve, helping people live longer and more fully with this condition.

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Types of leukemia: Prevalence, treatment options, and prognosis - Medical News Today

Bone Marrow Stem Cells Comments Off on Types of leukemia: Prevalence, treatment options, and prognosis – Medical News Today | February 14th, 2021

The global stem cell therapy market is expected to witness a CAGR of 10.6% during the forecast period 2019-2024, and is also anticipated to reach USD 214.5 million by 2024. Growing awareness related to the therapeutic potency of stem cells, development of infrastructure related to stem cell banking and processing, development of advanced genome-based cell analysis techniques, and increasing private-public investment for the development of stem cell therapies are driving the growth of the stem cell therapy market.

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Supportive regulations to drive the growth of the stem cell therapy market

Supporting regulations across developing countries, increasing prevalence of chronic diseases, technological advancement in healthcare, cellular therapies are the major advancements in transforming healthcare and identification of new stem cell lines are also fueling the growth of the stem cell therapy market.

Diseases such as osteoarthritis, multiple sclerosis, heart failure, hearing loss and cerebral palsy are some of the diseases that could be treated using stem cell therapies. For instance, according to the WHO by 2050, it is estimated 900 million people will have disabling hearing loss. Moreover, 60 percent of childhood hearing loss is due to preventable causes.

Allogenic stem cell therapy market to hold the larger share in the market

There are two types of stem cell therapy, allogeneic and autologous. Of both, allogenic segment account for the larger share and is also predicted to grow at the faster rate in the coming years in the market due to its extensive therapeutic applications, increasing commercialization of allogeneic products, easy production scale-up process, and growing number of clinical trials related to allogeneic therapies.

The stem cell therapy market has been segmented by therapeutic application into gastrointestinal diseases, musculoskeletal disorders, surgeries, cardiovascular diseases, and wound and injuries. Musculoskeletal disorders category contributed the largest revenue in the market due to increasing prevalence of musculoskeletal disorders and bone & joint diseases, increasing availability of stem cell-based products for the treatment of musculoskeletal disorders, and growing patient preference for effective & early treatment strategies.

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The global stem cell therapy market has also been segmented by cell source into adipose tissue-derived mesenchymal stem cell, cord blood cells and bone marrow-derived mesenchymal stem cells. Of all the categories, the bone marrow-derived mesenchymal stem cells are increasingly being used for therapeutic applications.

North America offers huge opportunities for stem cell therapy industry players

The North American stem cell therapy market will remain the largest during the forecast period. The region is further predicted to observe the fastest growth during the forecast period in the global market owing to technological upgradation and large capital invested in the research and development activities. Moreover, increasing number of clinical trials to evaluate therapeutic potential of products, increasing prevalence of chronic diseases, the growing patient base for target diseases, growing public awareness related to the therapeutic potency of therapy, and increasing public-private funding & research grants for developing safe and effective stem cell therapy products are also supporting the growth of the North American stem cell therapy market.

Investing in research and development is the key strategy adopted by the market players

Major players in the industry are investing in the development of innovative and new products, which is strengthening their position in the stem cell therapy market. In February 2018, MEDIPOST announced that FDA has approved its stem cell-based Alzheimers disease drug, NEUROSTEM for clinical trials. Similarly, in March 2017, Osiris Therapeutics launched Prestige Lyotechnology, a method for storage of living cells and tissues.

Some of the key players operating in the stem cell therapy industry are Osiris Therapeutics, Inc., RTI Surgical, Inc., MEDIPOST Co., Ltd., Nuvasive, Inc., Pharmicell Co., Ltd., Holostem Terapie Avanzate Srl, JCR Pharmaceuticals Co., Ltd., Anterogen Co., Ltd., and Allosource.

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Global Stem Cell Therapy Market Set to Reach USD 214.5 Million by 2024 - The Courier

Bone Marrow Stem Cells Comments Off on Global Stem Cell Therapy Market Set to Reach USD 214.5 Million by 2024 – The Courier | February 14th, 2021

One-year-old Max Gardner was diagnosed with aplastic anaemia, in October 2020, a serious condition in which the bone marrow and stem cells do not produce enough blood cells.

After Max developed significant bruises and a rash over his body, parents, Connor Gardner and Rachel Nicholson, from Hebburn, were referred to South Tyneside District Hospital, where their brave little boy underwent tests.

Doctors initially believed that Max had an immune disorder but after he was admitted to the Royal Victoria Infirmary (RVI) further tests helped to diagnose him with aplastic anaemia.

The family was told that the condition could be fatal if not treated properly.

Doctors said Max needed to have a bone marrow transplant, which has the potential to cure him.

Dad Connor, 29, and mum Rachel, 27, were both tested to see if they would be a bone marrow match and the pair were overjoyed when Rachel was found to be a 9/10 match.

Max started chemotherapy on January 7 at the RVI and mum Rachel donated stem cells on January 13 at Newcastles Freeman Hospital.

The following day, January 14, Max underwent the transplant at the RVI.

The family is now waiting for the results of a Chimerism Test which will tell them for definite whether the stem cells have worked but signs are already looking positive.

Delighted dad, Connor, said: "His neutrophils [a type of white blood cell that protect us from infections] have been more than 0.50 for three days in a row, which means that he is essentially engrafted, which means that his body is accepting the transplant.

"So it is working, but we still have to wait for the test results."

Doctors say there is no doubt that it has worked with the way the numbers have gone up but they have to officially do it like that to make sure, Connor continued.

"But there is no reason why it shouldnt have [doctors] say.

"He has done really well to get to this stage, he has absolutely sailed through it, everyone is surprised with how well he has done.

This the best outcome we could have hoped for.

But it hasnt been plain sailing for the family, who have also had to face additional challenges during the treatment.

Parents Connor and Rachael initially were not allowed to visit Max at the same time due to Covid rules, however the hospital has now eased the restriction in their case.

The family also became sick with Norovirus in the run-up to the transplant, causing concern over whether it would have to be pushed back.

Thankfully, the transplant went ahead as planned and the family made a good recovery, although Max still needs help with his eating.

Max will now have to remain in hospital for a while longer as he recovers from the transplant.

Connor added: We can feel that we are nearly at the end of it.

"His neutrophils are the highest they have ever been since he became poorly so we feel like we are coming to the end.

The family are sharing Maxs journey to health on Instagram under the name @maxinamillionaajourney and hope his story will encourage people to sign up to the Anthony Nolan register to become a potential donor and help others like Max.

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Bone marrow transplant shows signs of curing brave little boy with one in a million condition - Shields Gazette

Bone Marrow Stem Cells Comments Off on Bone marrow transplant shows signs of curing brave little boy with one in a million condition – Shields Gazette | February 14th, 2021

Gorgeous little Arlo McArthur looks the picture of health and happiness.

Loved and adored by his family this little lively three-year-old from Milngavie is spoiled rotten by his three big sisters and his ultimate day out is playing golf with his daddy.

But behind the cheeky grin lies a devastating truth - he's a "ticking-timebomb" and needs a stem cell transplant to save his life.

So today, we've joined with Arlo's mum Nicole, dad Ian and his three doting sisters Carys, Brooke and Holly in asking Glasgow Live readers to step up and help this brave little boy.

They need young men, between 16 and 30 to volunteer to be tested to see if they are a match for the toddler. There's not much to it, a simple swab test carried out at home is enough for the experts to determine if you're a match.

The more people who register to be tested the better chance there is of finding the ideal candidate willing to donate the bone marrow little Arlo desperately needs.

For this family your help could mean the difference between life and death.

They've lived with the knowledge since he was 10 weeks old that a rare genetic condition could rob their precious little boy of his future.

Diagnosed with Wiskott-Aldrich Syndrome, it means Arlo's immune system doesn't function properly and it's difficult for his bone marrow to produce platelets, making him prone to bleeding.

Its estimated there are between 1 and 10 cases per million males worldwide. Arlo was only the third case at Queen Elizabeth University Hospital.

Doctors say they cant take the risk with an older donor as he was lucky to survive a previous transplant which failed when he was a baby.

His back-up is his dad Ian, 31, but he's only a half-match.

Sadly little Arlo's story isn't unique, across the country 2,300 people a year need a stem cell transplant and charity Anthony Nolan coordinates the search and raises money to support their vital work.

Nicole, 37, dreams of seeing her little boy attend his first day at school next August and believes someone out there can help that dream come true.

She pleaded: "Were asking as many people as possible to register and help give Arlo the life he deserves.

"We want to love and enjoy having our little boy around for a long time. He should be able to live out his life of dreams.

"Put yourself in the shoes of a parent whose child is ill, or someone else who is about to lose a loved one. Youve just been told in a room that they wont make it without stem cells. How does it feel?

"Its not just our Arlo, there are plenty of Arlos out there who need your help."

"People don't realise how easy it is to do. It's not this big operation, just a few injections and a day at an out-patient clinic to save someone's life. I wish it was opt-out, like organ donation.

"We dont have much time but I know in my heart the right match is out there."

To find out how you can can join the register and help the fight to save little Arlo and others just like visit Anthony Nolan's website here.

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Arlo's Army needs stem cell donor as mum begs for help to save three-year-old's life - Glasgow Live

Bone Marrow Stem Cells Comments Off on Arlo’s Army needs stem cell donor as mum begs for help to save three-year-old’s life – Glasgow Live | February 14th, 2021

wA new study led by cancer researchers of Medical University of South Carolina (MUSC) found that a solitary strain of Bacteroides fragilis altogether diminished graft-versus-host disease (GVHD) by ensuring gut integrity.

The findings reported in JCI Insight shows that even though bone marrow transplant can be a lifesaving procedure for patients with blood cancers; however, GVHD is a potentially fatal side effect of transplantation, and it has limited treatment options. This proof-of-concept study demonstrates that better treatment options may be on the horizon for patients with GVHD.

Xue-Zhong Yu, M.D., associate director of Basic Science at Hollings Cancer Center, and lead author Hanief Sofi, Ph.D., realized that protecting the health of the gastrointestinal tract is a good target for reducing severe GVHD.

"If we can figure out how to keep a patient's intestinal tissue healthy before and after bone marrow transplant, then the patient's outcome will be much better. We know that restoring the microbiota diversity in the gut is an effective solution, but that comes with many challenges," said Yu.

Patients with blood cancers, such as leukemia, must undergo radiation and chemotherapy before they can get their new cancer-free immune system through bone marrow transplantation. The balance between the immune system and intestinal microbiota, communities of microorganisms that live in the gut, is especially important for proper intestinal health. Unfortunately, the radiation and chemotherapy radically throw off this balance, and the diversity of the microbiota is reduced 100- or even 1,000-fold. This leads to a condition called "leaky gut."

Clinical studies have shown that patients who recover microbiota diversity faster have better outcomes and less severe GVHD. Reduced microbiota diversity is associated with more severe GVHD.

Other studies have shown that fecal microbial transplantation (FMT) can be effective at reducing GVHD, but the challenge is how to get the right donor. Patients are heavily immune-deficient after bone marrow transplantation, and there is a great risk of bad infection if FMT is used in humans.

The Yu laboratory used two different strains of mice to establish a GVHD model that closely resembles the biology that occurs in humans after bone marrow transplantation. The mice developed acute GVHD. FMT significantly reduced acute GVHD in this model and reduced donor T cell proliferation in the organs, which is what triggers GVHD.

The researchers then used genetic sequencing to see which bacteria strains were most different between the fecal material of GVHD mice that received FMT and those that did not receive FMT.

Mice that had the best outcome, the lowest GVHD, had the highest levels of a bacteria called B. fragilis. Mice given this single bacterial strain had significantly reduced acute and chronic (long-term) GVHD compared to mice that did not get B. fragilis. In fact, B. fragilis alone was as good or even better than FMT.

Administration of B. fragilis increased overall gut microbial diversity, including increasing the amount of other beneficial bacteria strains. Surprisingly, GVHD was reduced in this model not only by live bacteria but also by bacteria that had been killed by short exposure to high heat.

The observation that B. fragilis was the main effective bacteria in the FMT process was not entirely new: B. fragilis also reduces autoimmunity in type 1 diabetes and colitis.

The current study by Yu and colleagues has two important findings. First, a molecule called polysaccharide A on the surface of B. fragilis appears to be critical for the GVHD-reducing functions of this bacteria. When the bacteria were modified to lack polysaccharide A, GVHD was not reduced compared to mice that did not receive any B. fragilis.

Secondly, the administration of B. fragilis did not reduce the graft-versus-leukemia or cancer-killing effect of the bone marrow transplantation, even though it did reduce donor T cell expansion in the gut. This is critical, since GVHD treatment options that reduce the graft-versus-leukemia effect would not be clinically significant.

"If this can be translated into the clinic, it would be a safer, easier and more effective treatment option," said Yu.

Further study in humans is needed to get this potential treatment into the clinic. Hematopoietic stem cells, given via bone marrow transplant, are classic immunotherapies for liquid tumors, but strategies to make the transplantation safer and more beneficial are sorely needed. Hollings Cancer Center researchers continue to search for the most effective therapies to improve patient outcomes and quality of life, he said.

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Ensuring gut integrity may improve results in blood cancer: Study - Hindustan Times

Bone Marrow Stem Cells Comments Off on Ensuring gut integrity may improve results in blood cancer: Study – Hindustan Times | February 14th, 2021

When Marie Fuesel was treated for leukemia eight years ago, she needed donated blood products more than 100 times.

Theyd give me my chemotherapy, Id stay in the hospital for a week, then Id go home, get really sick and have to come back in for blood and platelets, says Fuesel, 53, a retired insurance agent who lives in suburban Chicago. I spent over 100 days in the hospital over eight months. The disease and treatments (affect the bone marrow and production of red and white blood cells and platelets), so many transfusions were required to achieve remission.

After eight months of chemotherapy, followed by a year on the targeted drug Sprycel (dasatinib) as part of a clinical trial, Fuesel went into remission. She no longer needs transfusions, but she still appreciates the need for blood donors. I wouldnt be alive if the blood wasnt available when it was needed, she says.

Back then, blood shortages werent common, but they are now. The stay-at-home orders at the beginning of the COVID-19 pandemic forced the cancellation of numerous blood drives, and safety concerns arising from its spread have prompted some frequent donors to stay away from donation centers.

Thats been a source of worry for oncologists. Patients with cancer use nearly one-quarter of the nations blood supply, according to the American Red Cross, and donated blood is a vital resource in the treatment of hematologic cancers. Patients who receive stem cell transplants often need transfusions of oxygen-carrying red blood cells, infection-fighting white blood cells and platelets to control bleeding. Blood transfusions are common in the supportive care of patients undergoing chemotherapy that suppresses production of all the blood cells that results in anemia, because they relieve symptoms that ensue, such as fatigue and shortness of breath.

Between March and June 2020, 37,000 blood drives were canceled, according to the American Red Cross. The impact of the blood shortage varied across the nation but has hit some cities particularly hard. The New York Blood Center, for example, which supplies New York City

hospitals, reported in December 2020 that it had just three days of supply on hand, down from the five- to seven-day supply it normally has.

Ongoing shortages are forcing cancer centers to change some of their procedures for using donated blood. We all recognize that we are in the midst of a public health crisis and that we all have to do our part, says Dr. Mikkael Sekeres, chief of hematology at the University of Miami Miller School of Medicine and a physician liaison in hematology at Sylvester Comprehensive Cancer Center.

In response to COVID-related blood shortages, several cancer centers adjusted their policies for transfusing blood. Moffitt Cancer Center in Tampa, Florida, for example, developed a blood shortage action plan, according to Dr. Kaaron Benson, director of the blood bank at Moffitt. It basically meant dropping some of the thresholds we would normally use for transfusion, Benson says.

Moffitt has not needed to implement the plan yet, but if it does, Benson says, the change would most likely have the biggest effect on patients with leukemia and lymphoma who are given platelets as a preventive strategy. Provided theyre not bleeding or engaging in activities that increase the risk of bleeding, studies have shown you can allow the platelet threshold to drop from our standard of 10,000 per microliter to 5,000, she says.

The technique was first suggested in a 1991 journal article and has since been widely accepted as an appropriate change to make during blood shortages, Benson says.

In recent years, many oncologists have set lower thresholds for red blood cell transfusions another change that has eased the strain on blood supply. They used to routinely order transfusions for patients with hemoglobin levels below 10 grams per deciliter. That number dropped to between 7 and 8 grams per deciliter after a series of studies showed that infusing red blood cells at the higher threshold did not improve treatment outcomes.

During the pandemic, Moffitt and other cancer centers are also delaying some stem cell transplants and elective surgeries, so that blood used during those procedures can be kept on hand for patients who urgently need it, such as trauma patients or those needing emergent surgery. But those decisions are made on a case-by-case basis, so patients should maintain a frequent dialogue with their oncologists to determine the best plan for managing their symptoms during the pandemic.

Patients with multiple myeloma, for example, can benefit from stem cell transplants, but its usually not urgent, says Dr. Stephanie Lee, a hematologist and professor at the Fred Hutchinson Cancer Research Center in Seattle. We have very good treatments for multiple myeloma, so we can continue to give patients chemotherapy for weeks or months, Lee says.

However, she explains, patients with leukemia who need stem cell transplants may be advised to undergo the procedure as quickly as possible, even during the pandemic, because delaying it could cause the cancer to grow and become resistant to treatment.

And some patients with cancer who are simultaneously fighting other diseases should receive all the blood and platelet transfusions they need to manage their cancer, as well as to address any risks posed by chronic conditions. If you have heart disease, and your hemoglobin drops even further, youre more likely to get angina or suffer a heart attack, Sekeres says. So, for those people with serious comorbidities, we are more aggressive in transfusing blood products.

Growing the Donor Pool

Stephenie Perry, who works as the business operations coordinator for the American Red Cross of Northwest Georgia, knows firsthand the value of donated blood. Perry is a survivor of Hodgkin lymphoma who needed several transfusions during her treatment, which consisted of a round of chemotherapy and two stem cell transplants.

Perry, 31, has been in remission since February 2018, but sometimes her red blood cell count still runs low and she needs another blood transfusion. I feel sluggish, and when I stand up, I get really dizzy, says Perry, who lives in Rome, Georgia. When I get a transfusion, its like someone has just given me a shot of energy.

How can patients adapt when blood shortages mandate less frequent transfusions? Lifestyle changes can make a big difference, Sekeres says. If a patient is becoming progressively anemic, and its someone who usually goes for a 2-mile walk every day, maybe theyll reduce it to 1 mile or cut (exercise) altogether, he says.

Some patients may be eligible for iron infusions, which can relieve symptoms of fatigue and lengthen the period between infusions, says Abbey Fueger, clinical trial nurse navigator for the Leukemia & Lymphoma Society.

In addition, there are other small changes that can lessen the risk of anemia and improve symptoms. Some physicians are trying to limit blood draws for patients and recommending nutritional supplements that might help them feel better and lengthen the time between infusions, she says.

Meanwhile, an effort is underway to expand the pool of potential blood donors. In April, the Food and Drug Administration (FDA) addressed blood shortages brought on by COVID-19 by easing up on some of its restrictions on who can donate. For example, people who are at risk of contracting HIV, and those who have a recent tattoo or piercing or possible exposure to an infected individual no longer have to wait one year to give blood. The new waiting period is three months.

The FDA also dropped the waiting period for donors who have traveled to malaria-endemic countries from one year to three months. And it no longer recommends that blood centers turn away donors who lived in certain European countries during the era when Creutzfeldt-Jakob disease, a rare and fatal degenerative brain disorder, was thought to be spreading.

The hospital community is rallying around the cause, holding blood drives of their own and encouraging family members of patients to donate blood.

During the first few months of the pandemic, Fuesel helped put together five small blood drives in her town of Orland Park, Illinois. They were so successful the American Red Cross and a local news broadcaster asked her to help run the seventh annual Great Chicago Blood Drive. So, she did, and on Jan. 13, that event collected 330 units of blood at the Orland Park location and more than 2,000 units at other drives around the city.

For donors who might be nervous about giving blood during a pandemic, Fuesel has a message: Its safe and important. All the beds are spaced apart, and there are different stations when you walk in for getting your temperature checked and using hand sanitizer, Fuesel says. I know these are hard times, but it doesnt cost anything to give your blood. Its a way to help.

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Keep it Flowing: Combating COVID-19 Blood Shortages in Cancer Treatment - Curetoday.com

Bone Marrow Stem Cells Comments Off on Keep it Flowing: Combating COVID-19 Blood Shortages in Cancer Treatment – Curetoday.com | February 14th, 2021

BY Ariana Shah

Despite Canyon Lakes Bernadette Mycroft and her little family not able to catch a break, the five of them wrap their arms around each other and refuse to give in to adversity. Adversity that just keeps piling on.

Bernadette, a popular kindergarten teacher at Tuscany Hills Elementary, is a single mom doing her best to effectively raise her four children, Bryan, Scarlette, James and Julliette. Bryan was diagnosed last year with Myelodysplastic Syndrome, a rare blood cancer. Hes been in and out of ICU for months and has been extremely close to death on multiple occasions.

The goal for months has been for Bryan, a high school senior, to be strong enough to undergo a life-saving stem-cell transplant surgery. That day finally arrived, against all odds, last week. He is currently undergoing chemotherapy in the bone marrow transplant unit at Radys Children Hospital in San Diego.

Bryan has spent most of the last couple of months in Rady Childrens Hospital in San Diego to treat a rare form of blood cancer. After much adversity, a major stem cell transplant of his sisters marrow occurred last week. A Go Fund Me campaign has been set up to help the family.

The battle to get to the transplant is a story of resilience, faith and a family determined to stick together despite blow after blow being dealt them.

Bryan has been battling. Bryan has been fighting for his life for many months in and out of ICU. The treatment Bryan underwent late last year resulted in him losing over 40 pounds. Then, in early December, Bryan and his entire family tested positive for COVID-19.

Bryans immune system was severely compromised, Bernadette said. But he was able to battle through it, despite having no white blood cells. Im so grateful for Dr. John Bradley, who administered Bryan with a COVID-19 antibody infusion that saved him. Bryan is so incredibly strong.

Bryan then had two more emergency surgeries.

The abscess that the chemo caused needed to be drained, Bernadette said. He cant heal from any more invasive surgeries due to his lack of white blood cells. What a nightmare. He just couldnt catch a break. Hes just so incredibly strong.

Everybody in the family recovered from COVID-19 and a weakened Bryan was able to come back home and stay with his family for a couple of weeks at Christmas. New Years Eve, though, found Bryan back in the hospital for more emergency surgery.

Bryans sister, Scarlette, was identified as a match and courageously agreed to give her brother this life-saving bone marrow transplant. On Feb. 1, Scarlette underwent a major operation to donate 8 million stem cells to be transplanted to her brother.

They have both been very courageous, brave and kind, Bernadette said. I could not be more proud of them. Little brother James and sister Juliette, too.

The stem cells were successfully transplanted into Bryan, and Scarlette was able to return home after a three-day stay in the hospital.

Its taken a toll on me to continuously drive back and forth from San Diego to Canyon Lake, Bernadette said. But, of course, Im doing it.

Bryan is currently on medication that prevents graft-versus-host disease. This procedure and aftercare will require him to be hospitalized for approximately four to six more weeks. Bernadette, meanwhile, has been on unpaid leave from Tuscany Hills Elementary for months.

Friends and the Canyon Lake Junior Womens Club are doing their best to take the family under their wing.

To say the Mycroft family is in crisis is an understatement, friend Tiffani Paul said as she set up a Go Fund Me account to help the family. Overcome with stress, worry, medical debt, loss of income and extraordinary expenses, the unbelievably proud and strong Ms. Mycroft has reluctantly allowed us to post this Go Fund Me on her familys behalf.

Those who wish to help the Mycroft family can donate to their Go Fund Me at https://www.gofundme.com/f/help-bernadette-mycroft-help-her-son-fight-cancer.

If there are any other approaches to help the Mycroft family, call or text Sonja of the Canyon Lake Junior Womens Club at 909-230-2702.

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Family's resilience heartwarming - The Friday Flyer

Bone Marrow Stem Cells Comments Off on Family’s resilience heartwarming – The Friday Flyer | February 14th, 2021

Apamistamab (Iomab-B) conditioning treatment with targeted radioimmunotherapy to the bone marrow resulted in high rates of successful allogeneic hematopoietic stem cell transplants in patients with active, relapsed, or refractory acute myeloid leukemia (AML), according to interim results from the phase 3 SIERRA trial, which were presented virtually at the 2021 Transplant and Cellular Therapies Meetings.1

In these patients with relapsed or refractory AML, we observed high rates of allogeneic stem cell transplant with curative intent [in] 88% of patients on the Iomab-B arm, 18% of patients who were randomized to the conventional care arm achieved complete remission and received standard of care allo-transplant, and an overall rate of 79% of allo-transplant in all enrolled patients, Boglarka Gyurkocza, MD, said in a virtual presentation.

Investigators sought to prove with this study that targeted radiation to the marrow with apamistamab, a radioactive iodine (131I)labeled anti-CD45 antibody, could enable the successful engraftment of patients despite active disease in the marrow. Safety and robust efficacy had previously been demonstrated with the agent in 271 patients treated in 9 different phase 1 and 2 clinical trials.

The SIERRA trial is looking to enroll 150 patients, and the trial is already over 75% enrolled. Recently, an independent data monitoring committee recommended that the trial continue to the planned full enrollment based on a positive pre-planned ad-hoc analysis.2

In the study, patients with active, relapsed, refractory AML are randomized 1:1 to receive either apamistamab conditioning therapy and allogeneic HCT or conventional care. In the control arm, patients who do not achieve a complete remission (CR) by day 42 are allowed to cross over to receive Iomab-B, and those who do have a CR undergo HCT or receive standard-of-care therapy of the physicians choice.

Durable CR (dCR) rate is the primary end point of the study, characterized as complete response at 6 months after initial CR, and the secondary end point is overall survival (OS) rate at 1 year.

Patients are eligible for enrollment if they have marrow blast count 5% or the presence of peripheral blasts, age 55 years, a Karnofsky score 70, and related/unrelated donor matching at human leukocyte antigen (HLA)-A, HLA-B, HLA-C, and DRB-1. Active, relapsed, or refractory AML was defined for the sake of the trial as primary induction failure after 2 cycles of therapy including chemotherapy or 2 cycles of venetoclax (Venclexta) with a hypomethylating agent or low-dose cytarabine, first early relapse after first CR of less than 6 months, relapse refractory to salvage chemotherapy regimen, or second or subsequent relapse. Secondary or treatment-related AML was also allowed.

In the SIERRA trial, patient-specific dosimetry was used to generate an individualized therapeutic dose to target marrow and spare non-hematopoietic organs. Patients in the investigational arm received a dosimetric dose of apamistamab ( 20 mCi) approximately 19 days prior to HCT followed by a therapeutic dose of apamistamab, which is individually calculated for each patient based on an upper limit of 24 Gy to the liver. After, patients remain on radiation isolation for several days before receiving fludarabine conditioning therapy (30 mg/m2/day for 3 days) and finally low-dose total body irradiation (200 cGy) prior to HCT.

Among the first 75% of enrolled patients (n = 113), patients in the apamistamab arm (n = 56) had a median age of 63 years (range, 55-77), 35% had intermediate risk and 61% had adverse risk, the median

percent of marrow blasts at baseline was 29% (range, 4%-95%), and had received a median of 3 prior treatment regimens (range, 1-7). At randomization, 56% were in primary induction failure, 16% were in first early relapse, 15% had relapsed or refractory disease, and 13% were in their second or later relapse.

In the conventional care arm, the median age was 65 years (range, 55-77), 32% had intermediate risk and 63% had adverse risk, median marrow blasts was 20% (range, 5%-97%), and had received a median of 3 prior regimens (range, 1-6). At randomization, 49% were in primary induction failure, 21% were in first early relapse, 21% had relapsed or refractory disease, and 8.8% were in their second or later relapse. Patients who crossed over to receive apamistamab (n = 30) had similar baseline characteristics.

Forty-nine patients in the apamistamab-randomized arm were able to go on and undergo allogeneic HCT compared with 10 patients in the conventional care arm. In the investigational arm, a median of 646 mCi (range, 3541027) of apamistamab was infused at a dose of 14.7 Gy (range, 4.6-32) to the marrow. The median infused CD34-positive cell count was 5.6 x 106/Kg (range, 1.8-208). Forty-five patients received peripheral blood stem cells (PBSCs), 3 received marrow grafts, 17 had related donors, and 31 had unrelated.

Individualized therapy of Iomab-B provided myeloablative doses of radiation to the marrow, Gyurkocza, a medical oncologist at Memorial Sloan Kettering Cancer Center, commented.

These patients had a median of 30 days (range, 23-60) to HCT after randomization and 14 days (range, 9-22) to neutrophil engraftment, with no graft failure reported. Patients also had 18 days (range, 4-39) until platelet engraftment.

We also observed 100% neutrophil and platelet engraftment in patients who received Iomab-B conditioning, despite a heavy leukemia burden, Gyurkocza said.

In patients in the conventional arm who went on to HCT, conditioning regimens for HCT consisted of fludarabine/melphalan in 2, fludarabine/melphalan/total body irradiation in 1, busulfan/fludarabine in 1, cyclophosphamide/fludarabine/total body irradiation in 2, and 4 had no data on conditioning regimens available. Eight of these patients had PBSCs, 2 had marrow, 3 had related donors, 6 had unrelated, and 1 was unreported.

Median days to HCT was 67 (range, 52-104) with 17 days (range, 13-83) to neutrophil engraftment and 22 days (range, 8-35) to platelet engraftment. There was 1 graft failure.

Among the patients who crossed over to receive apamistamab before HCT, the median infused dose was 592 mCi (range, 313-1013) with 15.5 Gy (range, 6.3-42) to the marrow. The median infused CD34-positive cell count was 5.1 x 106/Kg (range, 1.8-16.1). Twenty-eight patients had PBSCs, 2 had marrow, 10 had related donors, and 20 had unrelated.

Patients had a median of 62 days (range, 36-100) to HCT, 14 days (range, 10-37) to neutrophil engraftment, and 19 days (range, 1-38) to platelet engraftment. No graft failure was reported in this group.

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Apamistamab Conditioning Treatment Induces High Rates of HCT Success in AML - OncLive

Bone Marrow Stem Cells Comments Off on Apamistamab Conditioning Treatment Induces High Rates of HCT Success in AML – OncLive | February 11th, 2021