SEATTLE, Feb. 05, 2021 (GLOBE NEWSWIRE) -- Neoleukin Therapeutics, Inc., “Neoleukin” (NASDAQ:NLTX), a biopharmaceutical company utilizing sophisticated computational methods to design de novo protein therapeutics, today announced that Jonathan Drachman, M.D., Chief Executive Officer, will participate in a fireside chat during the Guggenheim Healthcare Talks | 2021 Oncology Days on Friday, March 12, 2021 at 3 p.m. Eastern Time.

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Neoleukin Therapeutics to Participate in Guggenheim Healthcare Talks 2021 Oncology Days

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CAMBRIDGE, Mass., Feb. 05, 2021 (GLOBE NEWSWIRE) -- Spero Therapeutics, Inc. (Nasdaq: SPRO), a multi-asset clinical-stage biopharmaceutical company focused on identifying, developing and commercializing treatments in high unmet need areas involving multi-drug resistant bacterial infections and rare diseases, today announced that the United States Food and Drug Administration (FDA) informed Spero that a clinical hold has been placed on its Phase 2a clinical trial of SPR720, Spero’s investigational oral antimicrobial agent being evaluated in patients with nontuberculous mycobacterial pulmonary disease (NTM-PD).

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Spero Therapeutics Provides Update on SPR720 Phase 2a Clinical Trial

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Recent analysis of Phase 3 DESTINY PWS limited to data collected before the onset of the COVID-19 pandemic shows statistical significance in the primary and key secondary endpoints Recent analysis of Phase 3 DESTINY PWS limited to data collected before the onset of the COVID-19 pandemic shows statistical significance in the primary and key secondary endpoints

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Soleno Therapeutics Provides Recap of Key Opinion Leader Webinar on DCCR for Treatment of Prader-Willi Syndrome

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ZUG, Switzerland and CAMBRIDGE, Mass., Feb. 05, 2021 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced that members of its senior management team are scheduled to participate in the Guggenheim Healthcare Talks 2021 Oncology Day on Friday, February 12, 2021, at 3:30 p.m. ET.

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CRISPR Therapeutics to Participate in the Guggenheim Healthcare Talks 2021 Oncology Day

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SOUTH SAN FRANCISCO, Calif., Feb. 05, 2021 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced that on February 1, 2021, the compensation committee of GBT’s board of directors granted 14 new employees restricted stock units for an aggregate of 103,450 shares of the company’s common stock. These awards were made under GBT’s Amended and Restated 2017 Inducement Equity Plan (the Plan).

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GBT Announces New Employment Inducement Grants

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– Management to host webinar for analysts on Friday, Feb. 12, at 8:00 a.m. Eastern Time – – Management to host webinar for analysts on Friday, Feb. 12, at 8:00 a.m. Eastern Time –

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Denali Therapeutics to Present New Data on ETV:IDS (DNL310) for the Potential Treatment of Hunter Syndrome at WORLDSymposium™

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Bespoke Extracts Welcomes Trend Expert Carly Stojsic as New Director of Marketing

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Bagsværd, Denmark, 5 February 2021 — This company announcement discloses the data of the transaction(s) made in Novo Nordisk shares by the company’s board members, executives and their associated persons in accordance with Article 19 of Regulation No. 596/2014 on market abuse.

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Trading in Novo Nordisk shares by board members, executives and associated persons on 3-4 February 2021

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VANCOUVER, British Columbia, Feb. 05, 2021 (GLOBE NEWSWIRE) -- Bright Minds Biosciences (“Bright Minds” or the “Company”), a biosciences company focused on creating the next generation of psychedelic and related therapies for the treatment of mental health and central nervous system disorders, today announced that the Company’s common shares (the “Shares”) are expected to commence trading on the Canadian Securities Exchange (the “CSE”) on Monday, February 8, 2021, under the ticker symbol “DRUG.”

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Bright Minds Biosciences Announces Listing on Canadian Stock Exchange

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VALNEVA

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Valneva - Déclaration d’actions et de droits de vote – Janvier 2021

Global News Feed Comments Off on Valneva – Déclaration d’actions et de droits de vote – Janvier 2021 | February 6th, 2021

UKONIQ is approved for adult patients with relapsed or refractory marginal zone lymphoma after at least one prior anti-CD20 based regimen

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TG Therapeutics Announces FDA Accelerated Approval of UKONIQ™ (umbralisib)

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Please read the full announcement in PDF

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Amendment: Trading by management and close relations of management

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Regulated information

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argenx announces closing of global offering

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Every year, about 10,000 people in the U.S. need a stem cell transplant but cant find a donor.

The intense medical procedure, which can help those with leukemia, lymphoma, sickle cell anemia and other blood diseases, can save lives but securing a donor can be like finding a needle in a haystack.

Be The Match is a nonprofit, national registry where people can sign up to donate their stem cells. More than 35 million people around the world have volunteered yet only a small percentage of those donors are Americans, and even the registry admits most Americans dont know it exists.

The mother of a 12-year-old boy with leukemia has set out to change that.

Mandy Goldman is a hairdresser who lives with her husband and four children outside Boston. She remembers the devastating day five years ago when doctors told her the chemotherapy they gave her son Mateo Goldman, 9 years old at the time, didnt work.

They told us that our only option of curing Mateo was a bone marrow transplant, she says, a risky procedure that often involves a host of complications. But they had no other choice, she says.

The family got to work on the monumental task finding Mateo Goldman a close enough match.

Linda Matchan first reported the Goldman familys experience for The Boston Globe. In her research, she found very, very few people had any awareness of the need for bone marrow and stem cells donors. The awareness campaign around the subject is severely lacking compared to other campaigns like the importance of donating blood, she says.

For example, there's a little boy right now in North Carolina named Thor Forte, who's 10 and has sickle cell disease. And he has been waiting for literally half his life, five years, for a donor to be available, Matchan says. He's a tough match, but they finally did find somebody. And then when the time came for the procedure, the person backed out. So two years later, the boy is still waiting.

Fortunately, quickly after finding out Mateo Goldman didnt match with anyone in his family, he was paired with a donor on the registry from Germany. Mandy Goldman says Laura Stterlin of Frankfurt was ready to go and donate, ultimately saving her son.

Mateo Goldman wrote Stterlin, whose name he did not know at the time, a thank you note reading: Dear Donor, thank you for giving me the bone marrow. You feel like youre already part of my family, he says.

And unlike usual Make-A-Wish requests, Mateo Goldman asked to meet Stterlin in person halfway across the world. The trip to Germany was planned for summer of 2020 but has since been canceled due to the pandemic.

In 2019 when she was reporting this story, Matchan had a trip planned to Germany. She ended up meeting Stterlin and hearing the story of how she became a donor. Stterlin said she was at a sporting event with her husband when she got hungry and went on the hunt for some grub.

Dear Donor, thank you for giving me the bone marrow. You feel like youre already part of my family.

Germany has a robust public service campaign to get citizens to donate bone marrow, Matchan says. So it came to no surprise to Stterlin when she came across a kiosk to sign up.

Just three months later, she got a call and an email from the registry saying that there is somebody in the United States for whom she could be a match and was asked if she would donate, Matchan says. A couple of days later, she went into the hospital and did the donation.

Stterlins stem cells then crossed the Atlantic Ocean, making their way to America during a snowstorm.

The cells started working in Mateo Goldman right away but not without some difficulties, Mandy Goldman says. He battled total body stiffness from graft-versus-host disease, a complication of the transplant.

But, you know, Matteo's an amazing kid, she says, so through it all, he was smiling and making the best of it, even though he was suffering for a lot of the time.

Two years later, in July of 2020, the cancer came back. But since Mateo Goldmans first transplant, the science had evolved greatly.

So much so that his older brother, Leo Goldman, became a candidate to donate his cells for the second stem cell transplant.

I didn't realize how I could get my brother's cells, Mateo Goldman, now 12 years old, says. Once that sank in, I felt that it would connect me and my brother more.

Right before Christmas last year, the family got extraordinary news: Mateo Goldman had zero cancer in his bone marrow, Mandy Goldman says.

Now the mom of four is on a mission to raise awareness on stem cell donations and share the story of how it saved her sons life.

The amazing feeling Leo got from being able to be the person who saved his brother's life is something he's going to carry with him forever, she says. And even Laura [Stterlin], she gave him three and a half years of his life that we get to spend with him. I just really want to educate people about how empowering it is to do something so incredible for somebody else.

When she started talking to others to raise awareness, she was shocked to discover how fearful people were in committing to be a donor.

If people could see the trauma these patients go through her son had a drain placed in his stomach, total body radiation, chemotherapy that left him head-to-toe in a skin-burning rash she says then maybe they wouldnt be scared to dedicate a small action for someone whose only cure is through a stem cell transplant.

Once people are educated about how much of a difference it makes, she says, then I feel like they would do it.

Click here to learn more about the Be The Match Registry.

Tinku Rayproduced and edited this interview for broadcast.Serena McMahonadapted it for the web.

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After Bone Marrow Donation Saves 9-Year-Old Boy With Cancer, Boston Mom Fights To Raise Awareness - Here And Now

Bone Marrow Stem Cells Comments Off on After Bone Marrow Donation Saves 9-Year-Old Boy With Cancer, Boston Mom Fights To Raise Awareness – Here And Now | February 6th, 2021

Leukemia is a type of cancer that affects the blood. The two most common types in children are acute lymphoblastic leukemia and acute myelogenous leukemia.

In a person with leukemia, blood cells are released into the bloodstream before they are fully formed, so there are fewer healthy blood cells in the body.

Below, we describe the types of childhood leukemia, the symptoms, and the treatments. We then look at when to contact a doctor, what questions to ask, and where to find support.

Childhood leukemia is the most common form of cancer in children. It affects up to 3,800 children under the age of 15 in the United States each year.

Leukemia occurs when bone marrow releases new blood cells into the bloodstream before they are fully mature.

These immature blood cells do not function as they should, and eventually, the number of immature cells overtakes the number of healthy ones.

Leukemia can affect red and white blood cells and platelets.

The bone marrow produces stem cells. A blood stem cell can become a myeloid stem cell or a lymphoid stem cell.

Lymphoid stem cells become white blood cells. Myeloid stem cells can become:

Leukemia is typically acute or chronic, and chronic types are rare in children. They can include chronic myeloid leukemia or chronic lymphocytic leukemia.

Most childhood leukemias are acute, meaning that they progress quickly and need treatment as soon as possible.

Acute lymphoblastic leukemia (ALL) is the most common type in children, accounting for 75% of childhood leukemia cases.

It affects cells called lymphocytes, a type of white blood cell.

In a person with ALL, the bone marrow releases a large number of underdeveloped white blood cells called blast cells. As the number of these increases, the number of red blood cells and platelets decreases.

There are two subtypes of ALL: B-cell and T-cell.

In most childhood cases of ALL, the cancer develops in the early forms of B-cells. The other type, T-cell ALL, typically affects older children.

Research from 2020 reports that the majority of people diagnosed with ALL are under 18 and typically between 2 and 10 years old.

The American Cancer Society report that children under 5 years old have the highest risk of developing ALL and that this risk slowly declines until a person reaches their mid-20s.

The outlook for ALL depends on the subtype, the persons age, and factors specific to each person.

Myeloid leukemias account for approximately 20% of childhood leukemia cases, and most myeloid leukemias are acute.

Acute myelogenous leukemia (AML) affects white blood cells other than the lymphocytes. It may also affect red blood cells and platelets.

AML can begin in:

Juvenile myelomonocytic leukemia (JMML) accounts for approximately 12% of leukemia cases in children.

This rare type is neither acute nor chronic. JMML begins in the myeloid cells, and it typically affects children younger than 2 years.

Symptoms can include:

The symptoms of leukemia may be nonspecific similar to those of other common childhood illnesses.

A doctor will ask how long the child has been experiencing the symptoms, which can include:

Children may experience specific symptoms depending on the type of blood cell that the leukemia is affecting.

A low number of red blood cells can cause:

A low number of healthy white blood cells can cause infections or a fever with no other sign of an infection.

A low platelet count can cause:

Various factors can increase a childs risk of leukemia, and most are not preventable.

The following genetic conditions can increase the risk of leukemia:

Also, having a sibling with leukemia may increase the risk of developing it.

These can include exposure to:

If a child has symptoms that might indicate leukemia, a doctor may perform or request:

A bone marrow aspiration involves using a syringe to take a liquid sample of bone marrow cells. The doctor may give the child a drug that allows them to sleep through this test.

During the diagnostic process, a person might ask:

The doctor may recommend a variety of treatments for childhood leukemia, and the best option depends on a range of factors specific to each person.

The treatment usually consists of two phases. The first aims to kill the leukemia cells in the childs bone marrow, and the second aims to prevent the cancer from coming back.

The child may need:

Before or during treatment, a person might ask the doctor:

Questions to ask after the treatment might include:

Children who have undergone leukemia treatments require follow-up care, as the treatments often cause late effects.

These can develop in anyone who has received treatment for cancer, and they may not arise for months or years after the treatment has ended.

Treatments that can cause late effects include:

These complications may affect:

The late effects that may come can also depend on the type of treatment and the form of leukemia.

Because many leukemia symptoms can also indicate other issues, it can be hard to know when to contact a doctor.

Overall, it is best to seek medical advice if a child shows symptoms or behaviors that are not normal for them.

If a child has received a leukemia diagnosis, the effects can extend to parents, other family members, caregivers, and friends.

A person can find support and additional resources from:

The following organizations based in the United Kingdom also provide support and guidance:

Childhood leukemia can affect mental health, as well as physical health.

Learn more about mental health resources here.

According to the American Cancer Society, most children with leukemia have no known risk factors. There is no way to prevent leukemia from developing.

Because there are very few lifestyle-related or environmental causes of childhood leukemia, it is very unlikely that a caregiver can do anything to help prevent the disease.

A childs outlook depends on the type of leukemia. It is important to keep in mind that current estimates do not take into account recent advances in technology and medicine.

For example, the most recent 5-year survival rate estimates reflect the experiences of children who received their diagnoses and treatments more than 5 years ago.

The American Cancer Society report that the 5-year survival rate for children with ALL is 90%. The same rate for children with AML is 6570%.

Childhood leukemia is typically acute, which means that it develops quickly. As a result, a person should contact a doctor if they notice any of the symptoms.

The most common type of childhood leukemia is ALL, representing 3 out of 4 leukemia cases in children.

Treatment may include a combination of chemotherapy, targeted drugs, immunotherapy, stem cell transplants, surgery, and radiation.

The prognosis depends on the type of leukemia and the childs age.

This diagnosis can affect mental as well as physical health, and the effects can extend to caregivers, family members, and friends. Many different resources are available for support.

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Leukemia in children: Symptoms, causes, treatment, outlook, and more - Medical News Today

Bone Marrow Stem Cells Comments Off on Leukemia in children: Symptoms, causes, treatment, outlook, and more – Medical News Today | February 6th, 2021

Following stem cell transplant or treatment with CAR T-cell therapies, patients with hematologic malignancies and coronavirus disease 2019 (COVID-19) tend to have favorable outcomes, especially if they are diagnosed in complete remission (CR) and further out from their cell infusion, according to Miguel-Angel Perales, MD, underscoring that care should not be delayed despite the ongoing pandemic.

Delayed therapy results in patients with relapse or progression of disease who did not receive the intended cellular therapy; [weve seen this happen] in 34% of cases, Perales, chief of the Adult Bone Marrow Transplant Service at Memorial Sloan Kettering Cancer Center (MSKCC), said during a presentation delivered at the 2021 AACR Virtual Meeting on COVID-19 and Cancer.1 Given that we can avoid the risk of nosocomial transmission, I think this clearly indicates that we should be careful about how we manage these patients and not try to delay their care.

In his talk, Perales highlighted registry data detailing the impact of the pandemic on cellular treatment in patients with cancer, outcomes of patients who were infected with the virus and received hematopoietic cell transplantation, and the impact of virus-related delays in care.

Data reported to the ASH Research Collaborative COVID-19 Registry for Hematology, a global reference tool available to the public, showed that as of January 15, 2021, a total of 813 malignant and non-malignant cases of COVID-19 were reported, with just over 500 cases reported in the United States alone.2

When looking at cellular therapies received prior to a diagnosis with the virus, 10 patients had received CAR T-cell therapies (6 recovered, 4 died), 46 patients had undergone allogeneic stem cell transplantation (34 recovered, 7 died, 5 had unknown outcome), and the majority, or 78 patients, had undergone autologous stem cell transplantation (67 recovered, 7 died, 4 had unknown outcome).

An earlier analysis of data collected from this registry showed that among the first 250 patients for whom data were collected, the overall mortality rate was 28% (95% CI, 23%-34%).3 However, in patients with moderate to severe COVID-19 infection, the mortality rate was even higher, at 42% (95% CI, 34%-50%). This is a condition that has significantly impacted our patients with hematologic malignancies, noted Perales.

Another registry, of the Center for International Blood & Marrow Transplant Research (CIBMTR), requires the inclusion of outcomes of patients who have undergone transplantation or received CAR T cells.4 As of January 15, 2021, data for 1258 patients from 195 centers were reported to the registry and showed that 50.08% of patients had undergone allogeneic transplantation and 44.66% had undergone autologous transplantation. Only a small percentage of patients received cell therapy, according to Perales.

The age of patients at the time of infection ranged from less than 20 years to older than 70 years, with the majority of patients between the ages of 60 years and 69 years. When looking at infections by region, 29.35% of cases were reported in the Midwest, 23.44% were reported in the Northeast, and 22.73% were reported in the South. The majority of cases occurred within the first 2 years of their infusion. A total of 614 casesalmost half of all patientshad their infection resolve, while 58 experienced improvement; 187 patients had died.

In a subsequent paper, investigators examined risk factors associated with death from COVID-19 in recipients of allogeneic transplantation based on data from the CIBTR registry.5 Results from the multivariate analysis showed that age greater than 50 years (P = .016), male gender (P = .006), and COVID-19 infection in less than 12 months following transplantation (P = .019) were all significantly associated with increased risk of death.

Interestingly, race and ethnicity were not significant in this series, noted Perales. Similarly, when we look at patients [who have undergone] autologous transplant, the only factor that we saw was the diagnosis of lymphoma versus myeloma. Other factors were not significant.

In another analysis, investigators examined outcomes of patients following transplant who were infected with the virus at MSKCC. Of the first 77 patients diagnosed between March 15, 2020 and May 7, 2020, 37 had undergone autologous transplant, 35 had undergone allogeneic transplant, and 5 had received CAR T-cell therapy.6

The disease distribution was as expected, according to Perales. Thirty-eight percent of patients had plasma cell disease, 23% had acute leukemia, 23% had aggressive non-Hodgkin lymphoma (NHL), 5% had Hodgkin lymphoma, 4% had chronic myeloid leukemia, 4% had myelodysplastic syndrome, and 3% had indolent NHL.

When you look at day [of infection] post infusion, you see there was a significant range, said Perales. In fact, the number of patients were diagnosed with COVID-19 several months or even years after their cell therapy. These are the demographics of 77 patients, but this is representative of the patients that we transplant at our center.

Notably, 44% of patients did not have any comorbidities. Investigators also examined the home medications that patients were receiving at the time of their COVID-19 diagnosis. Here, 10 patients were receiving steroids, 18 were receiving immunomodulatory agents, 4 were receiving anticoagulation agents, and 14 were receiving immunosuppressive drugs.

Almost half, or 48%, of patients had mild COVID-19 infection, so they were not admitted to the hospital. Twenty-six percent of patients had moderate infection, and thus, were admitted to the hospital, while 22% had severe infection and were either admitted to the intensive care unit or died.

In that group, the majority of them actually had active malignancy, unlike the other 2 groups where the majority actually were in remission, said Perales. Patients who required high levels of oxygen [were often those who] had active malignancy.

Results from a univariate analysis looking at the predictors of disease severity revealed significant associations between the presence of comorbidities and infiltrates on imaging at the time of diagnosis. Overall, however, we were able to see favorable outcomes with patients after COVID-19 infection, said Perales. Two-thirds of patients actually had a resolution. We did see 14 deaths, which represented 18% of patients. This was 41% of patients who were admitted, but particularly those with an active malignancy.

Among patients who were admitted to the hospital but had a malignancy that was in remission, the mortality rate was 21%. This was due, in part, to the fact that in many cases, patients or their family members decided to forego aggressive medical care.

Additional data revealed that COVID-19 was linked with a drop in lymphocyte populations across the board, added Perales. Notably, lymphopenia with COVID-19 was not found to impair long-term immune reconstitution in patients who had undergone bone marrow transplant.

When looking at survival in patients after infection with COVID-19, overall outcomes were found to be favorable.

Investigators also examined the risk of nosocomial infections in patients who had undergone transplantation or received cellular treatment in light of the pandemic. They looked at a series of 44 cases.

In March 2020, 2 healthcare workers were exposed at MSKCC and 3 patients had documented COVID-19 infection. One patient was receiving treatment in the inpatient setting, but the patient did have frequent visits from family members, according to Perales. So, its unclear when or how the exposure occurred, Perales said. The patient ended up dying.

Two additional patients may have been exposed in the donor room while they were collecting the stem cell from the autologous transplant, added Perales. One patient eventually died from the virus.

Again, its unclear whether these patients were infected in the center or in the community, as COVID-19 was very prevalent at the time, said Perales. Importantly, we have not seen any additional cases of potential or definite COVID-19 nosocomial infection since March 2020 at our center.

When examining the impact of the pandemic on treatment delays, in March 2020, investigators started to prospectively collect data from patients whose transplant or cellular therapy was delayed as a result of the impact of the virus on resources at the hospital, particularly the capability of using intensive care unit beds.1

Results showed that 85 patients delayed treatment; of those patients, 29 have not received their intended cellular treatment. Sixteen were supposed to receive autologous transplant, 12 were supposed to undergo allogeneic transplant, and 1 was supposed to receive CAR T-cell therapy.

Of the 56 patients who eventually proceeded to treatment, 62% received autologous transplant, 67% received allogeneic transplant, and 86% received CAR T-cell therapy. The biggest reason for not proceeding to treatment with autologous transplant and CAR T-cell therapy was because they were deferred due to good disease control. Other reasons included was because of a new comorbidity (12%) or they died from the virus. The most prominent reason for not proceeding to allogeneic transplant during the pandemic was progression of disease (42%).

We conclude that patients who are recipients of allogeneic transplant, and particularly those with acute leukemia, as much as possible should proceed to their indicated therapy and not be delayed, concluded Perales.

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Perales Examines the Impact of COVID-19 on Recipients of Cellular Therapies for Cancer - OncLive

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A tech entrepreneur, who is determined to live for 180 years, claims his bizarre strategies will soon be as popular as cell phones. Dave Asprey firmly believes one of the keys to living longer is skipping breakfast. The American lifestyle guru has asserted that he can at least live to the year 2153 by using a variety of techniques, including a cold cryotherapy chamber sitting and intermittent-fasting. The 47-year-old millionaire devised the term 'Biohacking' to detail his methods of turning back the biological clock. In an effort to survive for as long, Dave has spent over $1,000,000 on hacks and techniques to improve the overall functioning of his body. He has parts of his bone marrow removed to re-inject his own stem cells back into his body.

Appearing on ITVs This Morning today, he joined host Holly Willoughby for a virtual conversation. Holly quizzed Dave why he wants to live so long, to which he replied that as a curious person, he feels there is a lot in the world that can be fixed and improved that he thinks he has not done yet. Dave confirmed that some of the things he wants to pioneer are expensive. However, there are other ways that are free of cost like fasting.

Dave also speculated that after applying his methods for longer life, people under 40 will be 'happy and highly functional' over a 100-years-old. He stated that he won't be the only one as his wife both is also racing to get to 180 years old. In comparison to others, Dave has set himself up for much less inflammation by controlling what he eats and how he sleeps, besides several other anti-ageing treatments. Explaining the benefits of reintroducing his own stem cells in his body, he said people heal when they are young. With age, the stem cells of the body get exhausted, so he opts for ways which gives him more stem cells.

Dave also follows cryotherapy, which is also known as cold therapy. It involves using low temperatures to treat a variety of tissue lesions. He is also taking cold showers for more than ten years. Dave uses another technique to live a long life known as intermittent fasting. This involves controlling the number of times that one eats meals to create periods of fasting over a certain period. According to Dave, it helps in disease prevention as fasting periods lets his body to 'repair itself' while not digesting food.

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US Man Who Wants to Live for 180 Years Re-injects His Own Stem Cells, Spends Rs 87 Lakh - News18

Bone Marrow Stem Cells Comments Off on US Man Who Wants to Live for 180 Years Re-injects His Own Stem Cells, Spends Rs 87 Lakh – News18 | February 6th, 2021

The reduced osteogenic differentiation of bone marrow mesenchymal stem cells (BMSCs) is the typical characteristics of pediatric aplastic anemia (AA) pathogenesis. Long non-coding RNA MEG3 is reported to promote osteogenic differentiation of BMSCs via inducing BMP4 expression.This study aims to investigate the mechanism of DNMT1/MEG3/BMP4 pathway in osteogenic differentiation of BMSCs in pediatric AA.BMSCs were isolated and purified from bone marrows of pediatric AA patients (n=5) and non-AA patients (n=5). The expression of DNMT1, MEG3, and BMP4 in isolated BMSCs was detected using quantitative real-time PCR and western blot analysis. Osteogenic differentiation was determined using Alizarin red staining. The methylation of MEG3 promoter and the interaction between DNMT1 and MEG3 promoter were detected using methylation-specific PCR and chromatin immunoprecipitation assay, respectively.Lowly expressed MEG3 and BMP4 and highly expressed DNMT1 were observed in BMSCs of pediatric AA patients. The overexpression of MEG3 promoted osteogenic differentiation of BMSCs. Luciferase reporter assay showed that MEG3 overexpression increased transcriptional activity of BMP4. The inhibitor of methylation, 5-azacytidine, suppressed DNMT1 expression and reduced methylation of MEG3 promoter. Overexpression of DNMT1 increased the binding between DNMT1 and MEG3 promoter. The simultaneous overexpression of DNMT1 and MEG3 restored the inhibition of osteogenic differentiation caused by DNMT1 overexpression alone.Our findings indicated that DNMT1 mediated the hypermethylation of MEG3 promoter in BMSCs, and DNMT1/MEG3/BMP4 pathway modulated osteogenic differentiation of BMSCs in pediatric AA.

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Hypermethylation-mediated downregulation of long non-coding RNA MEG3 inhibits osteogenic differentiation of bone marrow mesenchymal stem cells and...

Bone Marrow Stem Cells Comments Off on Hypermethylation-mediated downregulation of long non-coding RNA MEG3 inhibits osteogenic differentiation of bone marrow mesenchymal stem cells and… | February 6th, 2021

The mum of a Stockton toddler says time is running out to find a match for her son who is battling a rare blood cancer.

Little Mason Arrowsmith, one, was diagnosed with Juvenile Myelomonocytic Leukaemia (JMML) on Christmas Eve last year.

The condition is a rare type of slowly developing blood cancer that occurs in young children.

Although it was discovered at an early stage, doctors say Mason needs a stem cell or bone marrow transplant for the best chance of survival.

In a desperate attempted to help, his mum Katie Jordan will donate her own bone marrow later this month, but a full match is urgently needed.

"The hospital has been absolutely amazing, but unfortunately after searching worldwide there is no match for Mason due to his bone marrow being so rare," Katie said.

"We need to get him to transplant as soon as, the hospital is looking at the end of February, using me as his first donor in the hope that this will help him until we find a better match.

"I would give my life for Mason but unfortunately, I can only donate my bone marrow three times in a lifetime and Im not a full match for him.

"Children with JMML live around 12 months after diagnosis, we need to find mason a better match. Twelve months is not long enough for us to have with our boy, we are living the worst possible nightmare."

As Mason does not have a close enough match within his family - the only potential cure is through a stem cell transplant from an unrelated donor.

The family has now launched a 'Masons Mission' to encourage people to support blood cancer charity Anthony Nolan in raising urgent funds to add more donors to the stem cells register.

There is currently a blacklog of around 25,000 potential donors due to the impact of the coronavirus pandemic and the charity needs to raise up to an extra 500,000 to add people to the register, from ordering more swab packs to analysing completed swabs in its laboratory.

Any one of the 25,000 people who have applied to join the Anthony Nolan could be a match for Mason or one of the 2,300 patients in the UK, who need a stem cell transplant from a donor each year.

Katie added: "We have set our target at 10,000 which does seem a lot but this would help to cover 250 registrations and kits and allow us to continue our search for my baby."

Henny Braund, Chief Executive of Anthony Nolan said: "Were doing all we can to find a stem cell donor to give Mason a second chance of life.

"A perfect storm of the coronavirus pandemic, and a surge of 40,000 incredible people who have been inspired to join the Anthony Nolan register in the last month by patients, like Mason means that were in urgent need.

"The best thing people can do is support Anthony Nolans work financially. By giving anything, together we can add all potential lifesavers to the register, and give patients like Mason hope."

Anthony Nolan recruits people aged 16-30 to the stem cell register as research has shown younger people are more likely to be chosen to donate.

They also carry out ground-breaking research to save more lives and provide information and support to patients after a stem cell transplant, through its clinical nurse specialists and psychologists, who help guide patients through their recovery.

It costs 40 to recruit each potential donor to the register, so Anthony Nolan relies on financial support.

You can support Masons Mission here.

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