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LGL Leukemia: Overview, Symptoms, and Treatment – Healthline

By daniellenierenberg

Large granular lymphocytic (LGL) leukemia is a kind of cancer that affects blood cells. The disease is rare: Only about 1,000 people per year are diagnosed with it. It affects men and women in roughly equal numbers, and most of those diagnosed are over 60 years old.

Heres what we know about this form of leukemia.

Your blood is made up of four different parts:

Some of your white blood cells are larger than the rest. These cells contain tiny granules that can be seen under a microscope.

In people with LGL leukemia, these large, granular white blood cells copy themselves until there are too many. The fact that the white blood cells (also called lymphocytes) replicate themselves is what makes this disorder a type of cancer.

Your blood contains two different types of lymphocytes: T-cells (T-LGL) and B-cells, which are also known as natural killer cells (NK-LGL). B-cells fight off invading bacteria and viruses. T-cells attack other cells in your body that have become harmful, like cancer cells.

When your T-cells are copying themselves too much, you have T-LGL leukemia. If your natural killer cells are replicating too much, you have NK-LGL leukemia.

Most cases of LGL leukemia are chronic and slow-growing, whether theyre NK-LGL or T-LGL. Only around 10 percent of all LGL cases are aggressive, fast-growing cells.

Researchers dont yet know what causes LGL leukemia. The disorder is associated with a genetic change or mutation, usually to the STAT3 and STAT5b genes.

Between 10 and 40 percent of people with LGL leukemia also have a history of autoimmune disorders. The immune disorder most often associated with LGL leukemia is rheumatoid arthritis (RA).

About 20 percent of those with LGL leukemia also have RA. So far, researchers have been unable to determine which disorder began first.

Most people who are diagnosed with LGL leukemia will experience some of these symptoms:

A healthcare professional may look for other symptoms, too, including:

You should contact your doctor and seek treatment if youre having recurring infections, especially if you have a fever that doesnt go away or you have other infection symptoms, such as swelling or sores, that arent getting better.

To find out if you have LGL leukemia, a healthcare professional will analyze a sample of your blood. Your doctor may also take a sample of your bone marrow, often from your hip area, to look for abnormal cells.

To determine which type of LGL leukemia you have, your doctor could use a laser technology called flow cytometry to identify whether T-cells or NK-cells are replicating too much.

Most cases of LGL leukemia are slow growing. Doctors sometimes take a wait-and-watch approach to treatment.

You may not start treatment until tests or symptoms show that the condition has reached a certain level.

If tests show that your neutrophil levels have dropped too much, your doctor may start treatment at that time. Around 45 percent of people with this condition needed immediate treatment.

When treatment for LGL leukemia begins, it may or may not follow the same intensive course as other cancer treatments.

Most people will eventually need some combination of chemotherapy and immune-suppressing drug therapy. Your medications could include:

In some cases, treatment for LGL leukemia involves a bone marrow or stem cell transplant. Its also possible that your treatment could include removing your spleen, an organ in your abdomen that filters your blood and helps maintain your immune system.

Two to three times a year, you may need to visit a healthcare professional to have bloodwork done to monitor your health and the activity of your white blood cells.

While theres no cure for LGL leukemia, most cases progress very slowly, unlike other forms of leukemia. One study that followed 1,150 people with the disease found that they lived an average of 9 years after their diagnosis.

The more aggressive form of LGL leukemia doesnt respond well to treatment. Life expectancy is likely much shorter for those with this very rare subtype of LGL leukemia.

LGL leukemia is a rare type of cancer where large white blood cells copy themselves too much, making your body prone to frequent infections.

Most cases of LGL leukemia are slow-growing, so treatment might not be necessary at first.

Eventually, people with this condition might need a combination of chemotherapy and immunosuppressing medications to slow the growth of cancer cells. Theres no cure yet for LGL leukemia.

A small percentage of cases are a faster-growing type of leukemia that doesnt respond well to treatments. Life expectancy for this subtype is shorter than the slow-growing type.

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BioRestorative Therapies Announces Notice of Allowance for a New Patent Application Related to its Off-the-Shelf ThermoStem Program – GlobeNewswire

By daniellenierenberg

MELVILLE, N.Y., March 31, 2021 (GLOBE NEWSWIRE) -- BioRestorative Therapies, Inc. (the Company) (OTC: BRTX), a life sciences company focused on stem cell-based therapies, today announced that the United States Patent Office has issued a notice of allowance for a patent application related to the Companys metabolic ThermoStem Program. The notice of allowance was issued on March 22, 2021 and is related to a new patent application not previously announced.

Claims granted under the new patent cover methodologies related to generating exosomes and brown adipocytes from human brown adipose-derived stem cells. Exosomes are small extracellular vesicles produced by cells that contain lipids, messenger-RNA, micro-RNA, cytokines and proteins. Therapeutic benefits of using exosomes have been demonstrated in various disease models and may provide a valuable therapeutic tool for treating disease.

We are pleased to see that we have been granted an additional patent by the USPTO for our ThermoStem Program, said Lance Alstodt, the Companys CEO. Our comprehensive portfolio of patents under our ThermoStem Program continues to expand as we develop and protect intellectual property related to large and growing markets where brown adipocyte therapeutics can be applied. Im very proud of our team, driving towards the achievement of our stated goals. The advancement of our technology is a core, fundamental value driver of our Company. Our family of intellectual property coupled with our financial reporting progress are critical factors contributing to our growth strategy.

It is expected that the exosome diagnostic and therapeutic market will reach $368 million by 2022 as the development of research, clinical tools and therapeutics continues to grow in this emerging technology.

About BioRestorative Therapies, Inc.

BioRestorative Therapies, Inc. (www.biorestorative.com) develops therapeutic products using cell and tissue protocols, primarily involving adult stem cells. Our two core programs, as described below, relate to the treatment of disc/spine disease and metabolic disorders:

Disc/Spine Program (brtxDISC): Our lead cell therapy candidate, BRTX-100, is a product formulated from autologous (or a persons own) cultured mesenchymal stem cells collected from the patients bone marrow. We intend that the product will be used for the non-surgical treatment of painful lumbosacral disc disorders or as a complementary therapeutic to a surgical procedure. The BRTX-100 production process utilizes proprietary technology and involves collecting a patients bone marrow, isolating and culturing stem cells from the bone marrow and cryopreserving the cells. In an outpatient procedure, BRTX-100 is to be injected by a physician into the patients damaged disc. The treatment is intended for patients whose pain has not been alleviated by non-invasive procedures and who potentially face the prospect of surgery. We have received authorization from the Food and Drug Administration to commence a Phase 2 clinical trial using BRTX-100 to treat chronic lower back pain arising from degenerative disc disease.

Metabolic Program (ThermoStem): We are developing a cell-based therapy candidate to target obesity and metabolic disorders using brown adipose (fat) derived stem cells to generate brown adipose tissue (BAT). BAT is intended to mimic naturally occurring brown adipose depots that regulate metabolic homeostasis in humans. Initial preclinical research indicates that increased amounts of brown fat in animals may be responsible for additional caloric burning as well as reduced glucose and lipid levels. Researchers have found that people with higher levels of brown fat may have a reduced risk for obesity and diabetes.

Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events or results to differ materially from those projected in the forward-looking statements as a result of various factors and other risks, including, without limitation, those set forth in the Company's latest Form 10-K filed with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and the Company undertakes no obligation to update such statements.

CONTACT:Email: ir@biorestorative.com

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All About Multiple Myeloma, Type of Blood Cancer Kirron Kher is Suffering From – India.com

By daniellenierenberg

New Delhi: People woke up to terrible news of actor-politician Kirron Kher suffering from multiple myeloma, a type of blood cancer. She is currently undergoing treatment in Mumbai. Chandigarh BJP President and Kirrons colleague, Arun Sood revealed this at a press conference on Wednesday after which Anupam Kher also confirmed the same in a long note on Thursday morning. Also Read - Confirmed: Kirron Kher Diagnosed With Blood Cancer, Anupam Kher Calls Her a 'Fighter'

She had suffered a broken left arm at her Chandigarh house on November 11 last year. After her medical tests at Post Graduate Institute of Medical Education and Research (PGIMER) in Chandigarh, she was diagnosed with multiple myeloma. The disease had spread to her left arm and right shoulder. For treatment she had to go to Mumbai on December 4, Sood said at the press conference. Also Read - Anupam Kher And Kirron Kher Wish Each Other on 35th Wedding Anniversary With This Romantic Picture

Fans and well-wishers of Kirron are devastated by the news with many fans not being able to comprehend the shocking news. Here are a few things to know about the disease Multiple Myeloma that Kirron has been diagnosed with: Also Read - Kirron Kher-Anupam Kher's Mushiness in THESE Throwback Pictures on 34th Anniversary is All Hearts

It is a type of cancer that forms in a type of white blood cells known as a plasma cell. Your healthy plasma cells will fight infections and make antibodies that will attack germs, as per Mayoclinic. The cancerous plasma cells accumulate in the bone marrow. In this case, the cancer cells start producing an abnormal protein which further leads to complications. In simpler words, Multiple Myeloma happens when plasma cells become cancerous and grow out of control.

The symptoms can vary depending on an individual. Few reports suggest that initial symptoms may not be noticeable. But after a while, when it starts getting aggressive, as per Healthline, 4 major symptoms are noticeable which are referred to by the acronym CRAB which means:

Body gets a high level of calcium which can cause:

The cause is still unknown. It initiates with abnormal plasma cells which multiply in the bone marrow. Myeloma cells do not multiple and die, they divide indefinitely, as per Healthline.

Till now there is no cure for multiple myeloma. There are multiple treatments available including chemotherapy, interventional radiology, radiation therapy, stem cell transplantation, targeted therapy. Treatments are used when the disease starts getting worse.

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Heres what happens when a beam of subatomic particles hits you in the face – The Next Web

By daniellenierenberg

What would happen if you stuck your body inside a particle accelerator? The scenario seems like the start of a bad Marvel comic, but it happens to shed light on our intuitions about radiation, the vulnerability of the human body, and the very nature of matter.

Particle accelerators allow physicists to study subatomic particles by speeding them up in powerful magnetic fields and then tracing the interactions that result from collisions. By delving into the mysteries of the Universe, colliders have entered the Zeitgeist and tapped the wonders and fears of our age.

As far back as 2008, the Large Hadron Collider (LHC), operated by the European Organization for Nuclear Research (CERN), was charged with creating microscopic black holes that would allow physicists to detect extra dimensions. To many, this sounds like the plot of a disastrous science-fiction movie.

It came as no surprise when two people filed a lawsuit to stop the LHC from operating, lest it produce a black hole powerful enough to destroy the world. But physicists argued that the idea was absurd and the lawsuit was rejected.

Then, in 2012, the LHC detected the long-sought Higgs boson, a particle needed to explain how particles acquire mass. With that major accomplishment, the LHC entered popular culture; it was featured on the album cover of Super Collider (2013) by the heavy metal band Megadeth, and was a plot point in the US television series The Flash (2014-).

Yet, despite its accomplishments and glamour, the world of particle physics is so abstract that few understand its implications, meaning, or use. Unlike a NASA probe sent to Mars, CERNs research doesnt produce stunning, tangible images.

Instead, the study of particle physics is best described by chalkboard equations and squiggly lines called Feynman diagrams. Aage Bohr, the Nobel laureate whose father Niels invented the Bohr model of the atom, and his colleague Ole Ulfbeck have even gone as far as to deny the physical existence of subatomic particles as anything more than mathematical models.

Which returns us to our original question: what happens when a beam of subatomic particles traveling at nearly the speed of light meets the flesh of the human body? Perhaps because the realms of particle physics and biology are conceptually so far removed, its not only laypeople who lack the intuition to answer this question, but also some professional physicists.

In a 2010 YouTube interview with members of the physics and astronomy faculty at the University of Nottingham, several academic experts admitted that they had little idea what would happen if one were to stick a hand inside the proton beam at the LHC. Professor Michael Merrifield put it succinctly: Thats a good question. I dont know is the answer. Probably be very bad for you.

Professor Laurence Eaves was also cautious about drawing conclusions. [B]y the scales of energy we notice, it wouldnt be that noticeable, he said, likely with a bit of British understatement. Would I put my hand in the beam? Im not sure about that.

Such thought experiments can be useful tools for exploring situations that cant be studied in the laboratory. Occasionally, however, unfortunate accidents yield case studies: opportunities for researchers to study scenarios that cant be experimentally induced for ethical reasons. Case studies have a sample size of one and no control group.

But, as the neuroscientist V S Ramachandran has pointed out in Phantoms in the Brain (1998), it takes only one talking pig to prove that pigs can talk. On 13 September 1848, for example, an iron rod pierced through the head of the US railway worker Phineas Gage and profoundly changed his personality, offering early evidence of a biological basis for personality.

And on 13 July 1978, a Soviet scientist named Anatoli Bugorski stuck his head in a particle accelerator. On that fateful day, Bugorski was checking malfunctioning equipment on the U-70 synchrotron the largest particle accelerator in the Soviet Union when a safety mechanism failed and a beam of protons traveling at nearly the speed of light passed straight through his head, Phineas Gage-style.

Its possible that, at that point in history, no other human being had ever experienced a focused beam of radiation at such high energy. Although proton therapy a cancer treatment that uses proton beams to destroy tumors was pioneered before Bugorskis accident, the energy of these beams is generally not above 250 million electron volts (a unit of energy used for small particles). Bugorski might have experienced the full wrath of a beam with more than 300 times this much energy, 76 billion electron volts.

Proton radiation is a rare beast indeed. Protons from the solar wind and cosmic rays are stopped by Earths atmosphere, and proton radiation is so rare in radioactive decay that it was not observed until 1970. More familiar threats, such as ultraviolet photons and alpha particles, do not penetrate the body past skin unless a radioactive source is ingested.

Russian dissident Alexander Litvinenko, for instance, was killed by alpha particles that do not so much as penetrate paper when he unknowingly ingested radioactive polonium-210 delivered by an assassin. But when Apollo astronauts protected by spacesuits were exposed to cosmic rays containing protons and even more exotic forms of radiation, they reported flashes of visual light, a harbinger of what would welcome Bugorski on the fateful day of his accident.

According to an interview in Wired magazine in 1997, Bugorski immediately saw an intense flash of light but felt no pain. The young scientist was taken to a clinic in Moscow with half his face swollen, and doctors expected the worst.

Ionizing radiation particles such as protons wreak havoc on the body by breaking chemical bonds in DNA. This assault on a cells genetic programming can kill the cell, stop it from dividing or induce a cancerous mutation. Cells that divide quickly, such as stem cells in bone marrow, suffer the most. Because blood cells are produced in bone marrow, for instance, many cases of radiation poisoning result in infection and anemia from losses of white blood cells and red blood cells, respectively.

But unique to Bugorskis case, radiation was concentrated along a narrow beam through the head, rather than being broadly distributed from nuclear fallout, as was the case for many victims of the Chernobyl disaster or the bombing of Hiroshima.

For Bugorski, particularly vulnerable tissues, such as bone marrow and the gastrointestinal track, might have been largely spared. But where the beam shot through Bugorskis head, it deposited an obscene amount of radiation energy, hundreds of times greater than a lethal dose by some estimates.

And yet, Bugorski is still alive today. Half his face is paralyzed, giving one hemisphere of his head a strangely young appearance. He is reported to be deaf in one ear. He suffered at least six generalized tonic-clonic seizures. Commonly known as grand mal seizures, these are the seizures most frequently depicted in film and television, involving convulsions and loss of consciousness.

Bugorskis epilepsy is likely a result of brain tissue-scarring left by the proton beam. It has also left him with petit mal or absence seizures, far less dramatic staring spells during which consciousness is briefly interrupted. There are no reports that Bugorski has ever been diagnosed with cancer, though that is often a long-term consequence of radiation exposure.

Despite having nothing less than a particle accelerator beam pass through his brain, Bugorskis intellect remained intact, and he successfully completed his doctorate after the accident. Bugorski survived his accident. And as frightening and awesome as the inside of a particle accelerator might be, humanity has thus far survived the nuclear age.

This article by Joel Frohlich was originally published at Aeon and has been republished under Creative Commons.

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Retracing the Lineage of Cancer Cells – Technology Networks

By daniellenierenberg

There is no stronger risk factor for cancer than age. At the time of diagnosis, the median age of patients across all cancers is 66. That moment, however, is the culmination of years of clandestine tumor growth, and the answer to an important question has thus far remained elusive: When does a cancer first arise?

At least in some cases, the original cancer-causing mutation could have appeared as long as 40 years ago, according to a new study by researchers at Harvard Medical School and the Dana-Farber Cancer Institute.

Reconstructing the lineage history of cancer cells in two individuals with a rare blood cancer, the team calculated when the genetic mutation that gave rise to the disease first appeared. In a 63-year-old patient, it occurred at around age 19; in a 34-year-old patient, at around age 9.

The findings, published in the March 4 issue ofCell Stem Cell, add to a growing body of evidence that cancers slowly develop over long periods of time before manifesting as a distinct disease. The results also present insights that could inform new approaches for early detection, prevention, or intervention.

"For both of these patients, it was almost like they had a childhood disease that just took decades and decades to manifest, which was extremely surprising," said co-corresponding study author Sahand Hormoz, HMS assistant professor of systems biology at Dana-Farber.

"I think our study compels us to ask, when does cancer begin, and when does being healthy stop?" Hormoz said. "It increasingly appears that it's a continuum with no clear boundary, which then raises another question: When should we be looking for cancer?"

In their study, Hormoz and colleagues focused on myeloproliferative neoplasms (MPNs), a rare type of blood cancer involving the aberrant overproduction of blood cells. The majority of MPNs are linked to a specific mutation in the gene JAK2. When the mutation occurs in bone marrow stem cells, the body's blood cell production factories, it can erroneously activate JAK2 and trigger overproduction.

To pinpoint the origins of an individual's cancer, the team collected bone marrow stem cells from two patients with MPN driven by the JAK2 mutation. The researchers isolated a number of stem cells that contained the mutation, as well normal stem cells, from each patient, and then sequenced the entire genome of each individual cell.

Over time and by chance, the genomes of cells randomly acquire so-called somatic mutations--nonheritable, spontaneous changes that are largely harmless. Two cells that recently divided from the same mother cell will have very similar somatic mutation fingerprints. But two distantly related cells that shared a common ancestor many generations ago will have fewer mutations in common because they had the time to accumulate mutations separately.Cell of origin

Analyzing these fingerprints, Hormoz and colleagues created a phylogenetic tree, which maps the relationships and common ancestors between cells, for the patients' stem cells--a process similar to studies of the relationships between chimpanzees and humans, for example.

"We can reconstruct the evolutionary history of these cancer cells, going back to that cell of origin, the common ancestor in which the first mutation occurred," Hormoz said.

Combined with calculations of the rate at which mutations accumulate, the team could estimate when the JAK2 mutation first occurred. In the patient who was first diagnosed with MPN at age 63, the team found that the mutation arose around 44 years prior, at the age of 19. In the patient diagnosed at age 34, it arose at age 9.

By looking at the relationships between cells, the researchers could also estimate the number of cells that carried the mutation over time, allowing them to reconstruct the history of disease progression.

"Initially, there's one cell that has the mutation. And for the next 10 years there's only something like 100 cancer cells," Hormoz said. "But over time, the number grows exponentially and becomes thousands and thousands. We've had the notion that cancer takes a very long time to become an overt disease, but no one has shown this so explicitly until now."

The team found that the JAK2 mutation conferred a certain fitness advantage that helped cancerous cells outcompete normal bone marrow stem cells over long periods of time. The magnitude of this selective advantage is one possible explanation for some individuals' faster disease progression, such as the patient who was diagnosed with MPN at age 34.

In additional experiments, the team carried out single-cell gene expression analyses in thousands of bone marrow stem cells from seven different MPN patients. These analyses revealed that the JAK2 mutation can push stem cells to preferentially produce certain blood cell types, insights that may help scientists better understand the differences between various MPN types.

Together, the results of the study offer insights that could motivate new diagnostics, such as technologies to identify the presence of rare cancer-causing mutations currently difficult to detect, according to the authors.

"To me, the most exciting thing is thinking about at what point can we detect these cancers," Hormoz said. "If patients are walking into the clinic 40 years after their mutation first developed, could we have caught it earlier? And could we prevent the development of cancer before a patient ever knows they have it, which would be the ultimate dream?"

The researchers are now further refining their approach to studying the history of cancers, with the aim of helping clinical decision-making in the future.

While their approach is generalizable to other types of cancer, Hormoz notes that MPN is driven by a single mutation in a very slow growing type of stem cell. Other cancers may be driven by multiple mutations, or in faster-growing cell types, and further studies are needed to better understand the differences in evolutionary history between cancers.

The team's current efforts include developing early detection technologies, reconstructing the histories of greater numbers of cancer cells, and investigating why some patients' mutations never progress into full-blown cancer, but others do.

"Even if we can detect cancer-causing mutations early, the challenge is to predict which patients are at risk of developing the disease, and which are not," Hormoz said. "Looking into the past can tell us something about the future, and I think historical analyses such as the ones we conducted can give us new insights into how we could be diagnosing and intervening."

Reference:Egeren DV, Escabi J, Nguyen M, et al. Reconstructing the lineage histories and differentiation trajectories of individual cancer cells in myeloproliferative neoplasms. Cell Stem Cell. 2021;28(3):514-523.e9. doi:10.1016/j.stem.2021.02.001

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Black people are three times less likely to find a bone marrow donor than white people – WLNS

By daniellenierenberg

LANSING, Mich. (WLNS) More than three-quarters of African Americans are unable to find a bone marrow or stem cell transplant who need one, according to the nations largest bone marrow registry, Be the Match.

There are more than 20 million people on the registry, a database of people who volunteer to donate their stem cells or bone marrow if theyre found to be a match to someone seeking a transplant, and oftentimes, a transplant can mean giving people a second chance at life.

In fact, a blood stem cell transplant can be a cure for more than 70 diseases including blood cancer (leukemia, lymphoma, etc), sickle cell anemia and aplastic anemia to name a few.

However, the likelihood for people of color and Black people specifically, is staggeringly low.

Every year, millions of people are diagnosed with a life-threatening blood disorder.

That means millions of people are looking for a donor, someone whose special protein cells, called HLA proteins, match their own.

Patients are most likely to match donors who share the same ethnic background, according to Be the Match, but this is not always the case.

There is a catch though more often than not, there are not enough donors available and willing to give blood.

As the coronavirus pandemic has struck the world and cut blood donations severely, the challenge of finding a blood donor match has become more difficult.

Charles Poldo is a 51-year-old Black man living in mid-Michigan. In 2012, Poldo was diagnosed with acute myeloid leukemia, a type of cancer of the blood and bone marrow with excess immature white blood cells.

Acute myeloid leukemia runs in his family too but Poldo said he was determined to beat the cancer.

My mom and her dad died from it so when I got diagnosed, I knew I was not going to die, Poldo said.

So, Poldo embarked on a 7 year-long search for a donor.

But doctors could not find anyone of the more than 20+ million people on the Be the Match Registry who were exactly compatible with Poldo.

Doctors decided to proceed with an imperfect solution and Poldo received a treatment that did not fully cure his AML.

As a result, Poldo went into remission and was re-diagnosed with AML again in Nov. 2019.

By this time, technology had advanced to where Poldo could receive an imperfect match.

Even so, Poldos scenario is not an ideal one, especially because of the adverse effects of an imperfect match namely, his own immune system attacking and rejecting the donors cells.

In the medical field, they refer to this adverse effect as a post-transplant complication or graft-versus-host (GVHD) disease.

In Feb. 2020 just before the pandemic hit, Poldo received an umbilical cord transplant, which uses stem cells from umbilical cords donated at the time of a persons birth for instances like these.

Fortunately, Poldos immune system did not reject his transplants. But thats not the case for everyone.

Poldo said the person whose umbilical cord he received is now 8 years old. While he did not have the opportunity to meet his donor face-to-face, he is thankful.

Poldo is one of many African Americans who are unable to find a donor whose proteins match up exactly.

Even though Poldos transplant was successful, others are not as lucky.

People of color are underrepresented in the registry for a number of reasons, although there is no clear evidence as to why.

According to CBS News, a couple of reasons why people of color are underrepresented include a history of medical abuse of Black people in healthcare research, a lack of multi-lingual resources and stigma or fear of donating out of health concerns.

The Black community is more likely to be skeptical to sign up to be volunteers for medical trials or even the registry due to their abuse and mistreatment by medical researchers in the past.

For example, the 1932 Tuskegee syphilis trials also known as: Tuskegee Study of Untreated Syphilis in the Negro Male purposely infected several hundred Black people with syphilis without their informed consent or cure of the disease.

The trial that was supposed to last six months ended up lasting 40 years.

In addition, language barriers and access to knowledge about donating blood and stem cells exist among minority communities including Asian, Hispanic and Black people.

Here are some facts that Be the Match has clarified for those on the fence about donating.

Many mistakenly believe that donating blood stem cells is painful, when in reality its not.

It is a common misconception that donating blood stem cells is dangerous. The truth is that there are few risks in donating blood stem cells.

Many people think that donating to a patient in need is expensive for them, but Be The Match covers every cost related to donation.

If you are interested in signing up for the national bone marrow registry, text cure29 to 61474Or join.bethematch.org/cure29

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Stem Cell Therapy Market expected to reach USD 16.51 Billion by 2025 KSU | The Sentinel Newspaper – KSU | The Sentinel Newspaper

By daniellenierenberg

Stem Cell Therapy Market is valued at USD 9.32 Billion in 2018 and expected to reach USD 16.51 Billion by 2025 with the CAGR of 8.5% over the forecast period.

In its latest report on Stem Cell Therapy Market provides a concise analysis of the recent market trends. The report further includes statistics, market forecasts and revenue estimations, which in addition highlights its status in the competitive domain as well as expansion trends adopted by major industry players.

Rising prevalence of chronic diseases, increasing spend on research & development and increasing collaboration between industry and academia driving the growth of stem cell therapy market.

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*The sample pages of this report is immediately accessible on-demand.**

Scope of Stem Cell TherapyMarket-

Stem cells therapy also known as regenerative medicine therapy, stem-cell therapy is the use of stem cells to prevent or treat the condition or disease. Stem cell are the special type of cells those differentiated from other type of cell into two defining characteristics including the ability to differentiate into a specialized adult cell type and perpetual self-renewal. Under the appropriate conditions in the body or a laboratory stem cells are capable to build every tissue called daughter cells in the human body; hence these cells have great potential for future therapeutic uses in tissue regeneration and repair. Among stem cell pluripotent are the type of cell that can become any cell in the adult body, and multipotent type of cell are restricted to becoming a more limited population of cells.

The stem cell therapy has been used to treat people with conditions including leukemia and lymphoma, however this is the only form of stem-cell therapy which is widely practiced. Prochymal are another stem-cell therapy was conditionally approved in Canada in 2012 for the treatment of acute graft-vs-host disease in children those are not responding to steroids. Nevertheless, hematopoietic stem cell transplantation is the only established therapy using stem cells. This therapy involves the bone marrow transplantation.

Stem cell therapy market report is segmented based on type, therapeutic application, cell source and by regional & country level. Based upon type, stem cell therapy market is classified into allogeneic stem cell therapy market and autologous market.

Based upon therapeutic application, stem cell therapy market is classified into musculoskeletal disorders, wounds and injuries, cardiovascular diseases, surgeries, gastrointestinal diseases and other applications. Based upon cell source, stem cell therapy market is classified into adipose tissue-derived mesenchymal stem cells, bone marrow-derived mesenchymal stem cells, cord blood/embryonic stem cells and other cell sources

The regions covered in this stem cell therapy market report are North America, Europe, Asia-Pacific and Rest of the World. On the basis of country level, market of stem cell therapy is sub divided into U.S., Mexico, Canada, U.K., France, Germany, Italy, China, Japan, India, South East Asia, GCC, Africa, etc.

Stem Cell TherapyCompanies:

Stem cell therapy market report covers prominent players like,

Osiris Therapeutics, Inc

MEDIPOST

Anterogen, Ltd.

Cynata

Pharmicell

Cytori Therapeutics

Holostem Terapie Avanzate S.r.l.

JCR Pharmaceuticals

NuVasive

RTI Surgical

STEMCELL Technologies

BIOTIME

Osiris Therapeutics

Human Longevity

Advanced Cell Technology

Promethera Biosciences

Mesoblast and AlloSource

others

Stem Cell TherapyMarket Dynamics

Rising spend on research and development activities in the research institutes and biotech industries driving the growth of the stem cell therapy market during the forecast period. For instance, in January 2010, U. S. based Augusta University initiated Phase I clinical trial to evaluate the safety and effectiveness of a single, autologous cord blood stem infusion for treatment of cerebral palsyin children. The study is estimated to complete in July 2020. Additionally, increasing prevalence of chronic diseases creating the demand of stem cell therapy. For instance, as per the international diabetes federation, in2019, around 463 million population across the world were living withdiabetes; by 2045 it is expected to rise around 700 million. Among all 79% of population withdiabeteswere living in low- and middle-income countries. These all factors are fuelling the growth of market over the forecast period. On the other flip, probabilities of getting success is less in the therapeutics by stem cell may restrain the growth of market. Nevertheless, Advancement of technologies and government initiative to encourage research in stem cell therapy expected to create lucrative opportunity in stem cell therapy market over the forecast period.

Stem Cell TherapyMarketRegional Analysis

North America is dominating the stem cell therapy market due increasing adoption rate of novel stem cell therapies fueling the growth of market in the region. Additionally, favorable government initiatives have encouraging the regional market growth. For instance, government of Canada has initiated Strategic Innovation Fund Program, in which gov will invests in research activities carried out for stem cell therapies. In addition, good reimbursing scheme in the region helping patient to spend more on health. Above mentioned factors are expected to drive the North America over the forecast period.

Asia Pacific is anticipated to grow at a highest CAGR over forecast period due to rising awareness of benefits of stem cell therapies among the population. In addition, increasing collaboration between industry-academia to initiate research and development in the stem cell therapy expected to create the huge growth over the forecast period. For instance, as per the report of Pharma Focus Asia, members of Asia-Pacific Economic Cooperation collaborated with Life Sciences Innovation Forum to involve professionals having expertise in stem cell therapies from academia and research centers to promote developments in stem cell research which will foster regional market growth.

Key Benefits for Stem Cell TherapyMarketReports

Global Market report covers in depth historical and forecast analysis.

Global Market research report provides detail information about Market Introduction, Market Summary, Global market Revenue (Revenue USD), Market Drivers, Market Restraints, Market opportunities, Competitive Analysis, Regional and Country Level.

Global Market report helps to identify opportunities in market place.

Global Market report covers extensive analysis of emerging trends and competitive landscape.

Stem Cell TherapyMarketSegmentation

By Type

By Therapeutic Application

By Cell Source

Regional & Country AnalysisNorth America, U.S., Mexico, Canada , Europe, UK, France, Germany, Italy , Asia Pacific, China, Japan, India, Southeast Asia, South America, Brazil, Argentina, Columbia, The Middle East and Africa, GCC, Africa, Rest of Middle East and Africa

Table of Content

1.1. Research Process

1.2. Primary Research

1.3. Secondary Research

1.4. Market Size Estimates

1.5. Data Triangulation

1.6. Forecast Model

1.7. USPs of Report

1.8. Report Description

2.1. Market Introduction

2.2. Executive Summary

2.3. Global Stem Cell Therapy Market Classification

2.4. Market Drivers

2.5. Market Restraints

2.6. Market Opportunity

2.7. Stem Cell Therapy Market: Trends

2.8. Porters Five Forces Analysis

2.9. Market Attractiveness Analysis

Continued

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Stem Cell Therapy Market expected to reach USD 16.51 Billion by 2025 KSU | The Sentinel Newspaper - KSU | The Sentinel Newspaper

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Bone Marrow-Derived Stem Cells (BMSCS) Market To Witness Relatively Significant Growth During Forecast to 2027 The Courier – The Courier

By daniellenierenberg

TheBone Marrow-Derived Stem Cells (BMSCS) Marketresearch report thoroughly explains each and every aspect related to the Global Bone Marrow-Derived Stem Cells (BMSCS) Market, which facilitates the reports reader to study and evaluate the upcoming market trend and execute the analytical data to promote the business.

Bone Marrow-Derived Stem Cells (BMSCS) Market Insight:

Bone marrow-derivedstem cells(BMSCS) market is expected to gain market growth in the forecast period of 2020 to 2027. Data Bridge Market Research analyses the market to growing at a CAGR of 10.4% in the above-mentioned forecast period. Increasing awareness regarding the benefits associates with the preservation of bone marrow derived stem cells will boost the growth of the market.

Avail Your Free Sample Copy of the Bone Marrow-Derived Stem Cells (BMSCS) Market Report (Including Full TOC, List of Tables & Figures, Chart)@ https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-bone-marrow-derived-stem-cells-bmscs-market#utm_source=KA

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The report also inspects the financial standing of the leading companies, which includes gross profit, revenue generation, sales volume, sales revenue, manufacturing cost, individual growth rate, and other financial ratios.

Prominent Key Players Covered in the report:

CBR Systems, Inc, Cordlife Sciences India Pvt. Ltd., Cryo-Cell International, Inc.ESPERITE N.V., LifeCell International Pvt. Ltd., StemCyte India Therapeutics Pvt. Ltd, PerkinElmer Inc, Global Cord Blood Corporation., Smart Cells International Ltd., Vita 34 among other domestic and global players.

Key Pointers Covered in the Bone Marrow-Derived Stem Cells (BMSCS) Market Industry Trends and Forecast

TheBone Marrow-Derived Stem Cells (BMSCS) marketreport provides successfully marked contemplated policy changes, favorable circumstances, industry news, developments, and trends. This information can help readers fortify their market position. It packs various parts of information gathered from secondary sources, including press releases, web, magazines, and journals as numbers, tables, pie-charts, and graphs. The information is verified and validated through primary interviews and questionnaires. The data on growth and trends focuses on new technologies, market capacities, raw materials, CAPEX cycle, and the dynamic structure of the Bone Marrow-Derived Stem Cells (BMSCS) market.

Major Regions as Follows:

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Table Of Contents: Bone Marrow-Derived Stem Cells (BMSCS) Market

Part 01:Executive Summary

Part 02:Scope of the Report

Part 03:Research Methodology

Part 04:Market Landscape

Part 05:Pipeline Analysis

Part 06:Market Sizing

Part 07:Five Forces Analysis

Part 08:Market Segmentation

Part 09:Customer Landscape

Part 10:Regional Landscape

Part 11:Decision Framework

Part 12:Drivers and Challenges

Part 13:Market Trends

Part 14:Vendor Landscape

Part 15:Vendor Analysis

Part 16:Appendix

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To summarize:

The global Bone Marrow-Derived Stem Cells (BMSCS) market report studies the contemporary market to forecast the growth prospects, challenges, opportunities, risks, threats, and the trends observed in the market that can either propel or curtail the growth rate of the industry. The market factors impacting the global sector also include provincial trade policies, international trade disputes, entry barriers, and other regulatory restrictions.

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Creative Medical Technology Holdings Publishes Efficacy in Pain Reduction and Mobility in Patients with Disc Degenerative Disc Using StemSpine…

By daniellenierenberg

PHOENIX, March 4, 2021 /PRNewswire/ --(OTC - CELZ)Creative Medical Technology Holdings announced today a publication in the pre-print server SSRN describing data from its first 15 patients treated in a clinical trial evaluation perispinal injection of bone marrow cells in patients with disc degenerative disease. Evaluation of patients at 30,60 90, 180, and 360 days revealed significant improvement in mobility and reduction in pain score . The mean pain changed from 8.9 at baseline to 4.3 at 30 days and sustained to 1.8 at 6 months and 1.3 at 12 months with a gradual reduction in overall pain medication utilization guided by their healthcare team. No serious adverse effects were noted with some short-term bruising in two patients at the harvest site and no long term adverse events where reported related to the procedure.

"This publication, which is "pre-peer review" describes what to our knowledge is the first demonstration of a signal of clinical efficacy by injecting stem cells in areas surrounding the disc." Said Dr Amit Patel, Board Member and Co-Founder of the Company. "While others have intra-disc injection may help disc pain, the current work regenerates the blood supple to the disc, allowing the disc to heal itself."

The autologous utilization of bone marrow falls under the "minimal manipulation exception" and can be commercialized rapidly, in the same manner that the Company commercialized Caverstem for treatment of erectile dysfunction.

Granted United States Patent #9,598,673 which is owned by the Company covers the use of any mesenchymal stem cells, both from the patient or from donors, for reduction of lower back pain when injected into the major muscles of the lower back.

"Disc degenerative disease represents a multi-billion dollar market for which current medical solutions do not address the underlying cause, while surgery is expensive and not applicable for a significant number of patients." Said Timothy Warbington, President and CEO of the Company. "We are excited to follow the path we did with CaverStem and initiate commercialization of this technology for American patients."

To view our Publication: https://papers.ssrn.com/sol3/papers.cfm?abstract_id=3797402

About Creative Medical Technology HoldingsCreative Medical Technology Holdings, Inc. is a commercial stage biotechnology company specializing in regenerative medicine/stem cell technology in the fields of immunotherapy, urology, neurology and orthopedics and is listed on the OTC under the ticker symbol CELZ. For further information about the company, please visitwww.creativemedicaltechnology.com.

Forward Looking StatementsOTC Markets has not reviewed and does not accept responsibility for the adequacy or accuracy of this release. This news release may contain forward-looking statements including but not limited to comments regarding the timing and content of upcoming clinical trials and laboratory results, marketing efforts, funding, etc. Forward-looking statements address future events and conditions and, therefore, involve inherent risks and uncertainties. Actual results may differ materially from those currently anticipated in such statements. See the periodic and other reports filed by Creative Medical Technology Holdings, Inc. with the Securities and Exchange Commission and available on the Commission's website atwww.sec.gov.

http://www.StemSpine.comwww.CaverStem.comwww.FemCelz.com

SOURCE Creative Medical Technology Holdings, Inc.

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An 8-year-olds search for bone marrow match in battle with leukemia comes to North Texas – The Dallas Morning News

By daniellenierenberg

Jakobe Kobe Washington is eight years old, loves baseball and is fighting an aggressive form of leukemia.

The Florida boy, who is known to pray for other kids in the hospital, needs life-saving blood stem cells or a bone marrow transplant. So far, his family has been unable to find a match.

On Saturday, Be The Match and the Icla da Silva Foundation will host a drive-through swab event at Irving Mall to try to find a match for Kobe, who has extended family in North Texas.

Its tough to see your kid fighting a fight, and you cant do anything but be there to support him, no control in it at all, Kobes father Jordan Washington, who is from Dallas, told the ABC affiliate in Tampa Bay, Fla.

Every year, more than 12,000 patients turn to Be The Match, a national marrow donation program, to search for blood stem cells or a bone marrow donor to help cure them of blood cancers, such as leukemia and lymphoma, according to a release about the event.

Roughly half of those patients are unable to find a match, with only 23% of Black patients like Kobe finding a match, compared to 77% of white patients, according to the Icla da Silva Foundation, which serves as a recruitment center for Be The Match and focuses on minority populations.

Thats because race and ethnicity play a key role in stem cells and marrow, and of the 22 million potential donors on the registry, only 4% are Black.

Potential donors ages 18 to 44 are encourages to go to the Irving Mall, 3880 Irving Mall, between 10 a.m. and 2 p.m. Saturday.

Participants will then register from their phones and take a swab of their inner cheek.

Those unable to attend can text 4Kobe to 61474 to complete the online registration and have a cheek swab kit sent to their home.

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An 8-year-olds search for bone marrow match in battle with leukemia comes to North Texas - The Dallas Morning News

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Magenta Therapeutics Reports Fourth Quarter and Full-Year 2020 Financial Results and Recent Program Highlights – Tullahoma News and Guardian

By daniellenierenberg

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Mar 3, 2021--

Magenta Therapeutics (Nasdaq: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of stem cell transplants to more patients, today reported financial results for the fourth quarter and full-year ended December 31, 2020 and recent program highlights.

Building on our momentum from 2020, we continue to advance our portfolio with now two active Phase 2 clinical trials evaluating MGTA-145 plus plerixafor in patients with blood cancers undergoing autologous and allogeneic stem cell transplant and an additional planned Phase 2 clinical trial evaluating stem cell mobilization and collection in patients with sickle cell disease in partnership with bluebird bio. We have also made additional progress in our preparations for an IND filing for our MGTA-117 targeted conditioning program based on communications with the FDA and the advancement of our IND-enabling studies, said Jason Gardner, D.Phil., President and Chief Executive Officer, Magenta Therapeutics. We very much look forward to generating clinical data during the course of 2021 in these multiple disease settings.

MGTA-145: Stem Cell Mobilization and Collection for Hematopoietic Stem Cell Transplantation and Gene Therapy

Magenta is developing MGTA-145 plus plerixafor to harness these agents complementary mechanisms to mobilize hematopoietic stem cells (HSCs) for collection and transplantation, including for use with gene therapies. The ability to provide rapid, reliable, predictable and safe mobilization and collection of HSCs in stem cell transplantation and gene therapy could position MGTA-145 plus plerixafor to be the preferred mobilization regimen across multiple diseases due to improved patient experience and collection outcomes.

MGTA-145 Current and Planned Activity:

MGTA-145 Recent and Upcoming Scientific Conference Presentations:

MGTA-117: Targeted Conditioning

Magenta is developing a platform of novel antibody-drug conjugates (ADCs) for conditioning, a step in the transplant process that currently relies on the use of systemic chemotherapy agents and radiation. Magentas targeted conditioning programs are designed to selectively eliminate stem cells and/or immune cells from a patient prior to transplant or gene therapy, and to reduce or potentially eliminate the need for high dose or high intensity chemotherapy-based regimens.

MGTA-117, Magentas most advanced conditioning program, is a CD117-targeted antibody conjugated to amanitin and intended for use in patients undergoing transplant. MGTA-117 is designed to deplete hematopoietic stem and progenitor cells to clear space in the bone marrow prior to transplant in support of long-term engraftment and improved disease outcomes in patients. MGTA-117 has shown high selectivity, potent efficacy and tolerability in multiple preclinical studies.

Targeted Conditioning Current and Planned Activity:

Targeted Conditioning Recent and Upcoming Scientific Conference Presentations:

Cash Position: Cash, cash equivalents and marketable securities as of December 31, 2020, were $148.8 million, compared to $145.7 million as of December 31, 2019. Magenta anticipates that its cash, cash equivalents and marketable securities will be sufficient to fund operations and capital expenditures into 2023.

Research and Development Expenses: Research and development expenses were $12.3 million in the fourth quarter of 2020, compared to $18.7 million in the fourth quarter of 2019. The decrease was driven primarily by decreased preclinical costs for manufacturing related to the conditioning programs, lower manufacturing and clinical trial costs due to the discontinuance of enrollment in the Phase 2 clinical trial of MGTA-456 in inherited metabolic diseases in June 2020 and lower clinical trial costs for the MGTA-145 Phase 1 clinical trial which was completed in the first quarter of 2020.

General and Administrative Expenses: General and administrative expenses were $6.8 million for the fourth quarter of 2020, compared to $5.9 million for the fourth quarter of 2019. The increase was primarily due to an increase in personnel costs, professional services and insurance costs associated with Magentas expanded clinical trial preparations.

Net Loss: Net loss was $18.2 million for the fourth quarter of 2020, compared to net loss of $23.2 million for the fourth quarter of 2019.

About Magenta Therapeutics

Magenta Therapeutics is a clinical-stage biotechnology company developing medicines to bring the curative power of immune system reset through stem cell transplant to more patients with blood cancers, genetic diseases and autoimmune diseases. Magenta is combining leadership in stem cell biology and biotherapeutics development with clinical and regulatory expertise, a unique business model and broad networks in the stem cell transplant community to revolutionize immune reset for more patients.

Magenta is based in Cambridge, Mass. For more information, please visit http://www.magentatx.com.

Follow Magenta on Twitter: @magentatx.

Forward-Looking Statement

This press release may contain forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws, including express or implied statements regarding Magentas future expectations, plans and prospects, including, without limitation, statements regarding expectations and plans for presenting pre-clinical and clinical data, projections regarding future revenues and financing performance, our long-term growth, cash, cash equivalents and marketable securities, the anticipated timing of our clinical trials and regulatory filings, the development of our product candidates and advancement of our preclinical programs, the timing, progress and success of our collaborations, as well as other statements containing the words anticipate, believe, continue, could, endeavor, estimate, expect, anticipate, intend, may, might, plan, potential, predict, project, seek, should, target, will or would and similar expressions that constitute forward-looking statements under the Private Securities Litigation Reform Act of 1995. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation: uncertainties inherent in clinical studies and in the availability and timing of data from ongoing clinical studies; whether interim results from a clinical trial will be predictive of the final results of the trial; whether results from preclinical studies or earlier clinical studies will be predictive of the results of future trials; the expected timing of submissions for regulatory approval or review by governmental authorities; regulatory approvals to conduct trials or to market products; whether Magenta's cash resources will be sufficient to fund Magenta's foreseeable and unforeseeable operating expenses and capital expenditure requirements; risks, uncertainties and assumptions regarding the impact of the continuing COVID-19 pandemic on Magentas business, operations, strategy, goals and anticipated timelines, Magentas ongoing and planned preclinical activities, Magentas ability to initiate, enroll, conduct or complete ongoing and planned clinical trials, Magentas timelines for regulatory submissions and Magentas financial position; and other risks concerning Magenta's programs and operations are described in additional detail in its Annual Report on Form 10-K expected to be filed on or about March 3, 2021, its Quarterly Reports on Form 10-Q and its other filings made with the Securities and Exchange Commission from time to time. Although Magenta's forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Magenta. As a result, you are cautioned not to rely on these forward-looking statements. Any forward-looking statement made in this press release speaks only as of the date on which it is made. Magenta undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future developments or otherwise.

2020

2019

2020

2019

12,256

18,714

50,615

59,208

6,809

5,923

28,087

23,761

19,065

24,637

78,702

82,969

(19,065

)

(24,637

)

(78,702

)

(82,969

)

897

1,400

3,766

6,200

$

(18,168

)

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Magenta Therapeutics Reports Fourth Quarter and Full-Year 2020 Financial Results and Recent Program Highlights - Tullahoma News and Guardian

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COSELA (trilaciclib) for the Treatment of Chemotherapy-Induced Myelosuppression – Clinical Trials Arena

By daniellenierenberg

COSELA is the first myeloprotection therapy indicated to reduce the incidence of chemotherapy-induced myelosuppression in adult patients. Credit: G1 Therapeutics. Small cell lung cancer represents nearly 10% to 15% of all lung cancer cases. Credit: BonD80 / Shutterstock. Trilaciclib is a competitive inhibitor of cyclin dependent kinases 4 and 6 (CDK4/6). Credit: StudioMolekuul / Shutterstock.

COSELA (trilaciclib) is the first approved myeloprotection therapy indicated to reduce the occurrence of chemotherapy-induced bone marrow suppression in adult patients.

Developed by G1 Therapeutics (GTHX), a US-based clinical-stage biopharmaceutical company, the drug is available in a single-dose vial as a sterile, preservative-free, yellow lyophilised cake in a 300mg dosage strength for intravenous administration.

In June 2020, G1 Therapeutics signed a three-year co-promotion agreement with German pharmaceutical firm Boehringer Ingelheim (BI) to jointly promote trilaciclib for the treatment of small cell lung cancer in the US and Puerto Rico.

Under the agreement, G1 Therapeutics will lead marketing, market access and medical engagement initiatives for COSELA while Boehringer Ingelheim will undertake salesforce engagements.

In August 2020, China-based Simcere Pharmaceutical Group was granted the development and commercialisation rights of the drug in all indications for Greater China.

The New Drug Application (NDA) for trilaciclib was submitted to the US Food and Drug Administration (FDA) in June 2020 and granted priority review in August 2020.

In February 2021, the FDA approved trilaciclib to reduce chemotherapy-induced myelosuppression in extensive-stage small cell lung cancer (ES-SCLC) patients, prior to chemotherapy treatments involving platinum-etoposide or topotecan options. The FDA also bestowed breakthrough therapy designation to trilaciclib in August 2019.

Myelosuppression, also known as bone marrow suppression, is a chemotherapy-induced bone marrow damage condition that lowers blood cell production.

Although chemotherapy drugs are used to destroy cancer cells, they can also cause damage to healthy cells in the bone marrow such as hematopoietic stem and progenitor cells (HSPCs), which produce white blood cells, red blood cells and platelets.

Myelosuppression is a side effect of chemotherapy and occurs when the hematopoietic stem and progenitor cells are damaged by chemotherapy treatment, thereby suppressing the ability of bone marrow to produce blood cells.

The common symptoms associated with myelosuppression include fatigue, shortness of breath, and dizziness. Myelosuppression can also lead to serious blood cell diseases such as anaemia, neutropenia and thrombocytopenia.

Trilaciclib is a transient and competitive inhibitor of cyclin-dependent kinases 4 and 6 (CDK4/6). The drug delivers a myeloprotective therapy against chemotherapy-induced bone marrow suppression by inhibiting CDK4/6 that regulates cell cycle.

By inhibiting CDK4/6, trilaciclib temporarily and reversibly induces G1 cell cycle arrest in hematopoietic stem and progenitor cells (HSPCs) and prevents transition to the synthesis phase (S phase) of cell cycle, thus protecting the HSPCs from the damaging effects of chemotherapy and maintaining the normal function of the bone marrow.

COSELAs FDA approval was based on the outcome of three randomised, double-blind, placebo-controlled clinical trials in patients with extensive-stage small cell lung cancer. The effectiveness of drug was evaluated in combination with carboplatin-etoposide, with or without atezolizumab and topotecan chemotherapy.

The studies randomly enrolled 245 patients to receive either intravenous (IV) trilaciclib or placebo prior to the start of chemotherapy.

The primary endpoints of the studies were the percentage of patients with severe neutropenia and its duration during the first chemotherapy cycle.

The trials demonstrated clinical reduction in the duration and severity of neutropenia among ES-SCLC patients who received trilaciclib before chemotherapy.

A positive impact on red blood cell transfusions and other myeloprotective measures was also observed.

The most frequent side effects observed in the patients during the clinical trials were fatigue, hypophosphatemia, hypocalcaemia, hypokalaemia, headache, high aspartate aminotransferase levels and pneumonia. More than 3% of the patients who received COSELA experienced serious adverse reactions, including respiratory failure, haemorrhage and thrombosis.

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U. Cancer Center pilot projects: investigating cancer connections – The Brown Daily Herald

By daniellenierenberg

Eight labs who were recipients of the University Cancer Centers funding in December for projects advancing cancer research will use the funds to delve into cancer biology, cancer therapeutics and population science.

Four of the eight projects are investigating immunotherapy for gastrointestinal cancers, the tumor environments impact on cancer cell growth, the potential application of an FDA-approved Parkinsons drug to treat glioma brain tumors and the ability of a novel drug to target cancer cells that exhibit heightened aggressiveness following immunotherapy, The Herald previously reported.

The Herald spoke with three of the four other principal investigators that received grants.

Assistant Professor of Medicine Hina Khans pilot project will study the effects of blocking the antibody for chitinase 3-like-1, or CHI3L1, in advanced non-small cell lung cancer. CHI3L1 is a protein that plays an important role in tissue repair, and elevated levels of the protein indicate poor outcomes in advanced stage cancer patients. The researchers will test whether blocking the antibody a molecule that binds CHI3L1 will prevent cell resistance to immune checkpoint inhibitors in this type of lung cancer.

Assistant Professor of Medicine Olin Liang is interested in exploring womens ability to fight off leukemia and other blood diseases later in life relative to men. While the effect of aging on blood cancer development has been well-studied, not much research has gone into studying sex differences, Liang said.

Past work from the Liang lab has shown that the bone marrow environment remains healthier longer in women, leading to better blood cell production and immune response. By transplanting bone marrow stem cells from young male mice into middle-aged male and female mice, the researchers were able to compare the expression of these cells amongst the two sexes. They found higher expression in female middle-aged mice, which is indicative of a healthier bone marrow environment. This observation was due to receptors molecules that can interact with hormones to produce a response in a cell on the surface of bone marrow stem cells that were uniquely responsive to sex hormones predominantly found in women.

We have narrowed it down to two sex hormone receptors that may play a role, Liang said, referring to the receptors for follicle-timulating hormone and androgen hormone. The lab plans to use the Cancer Center pilot project funds to further study the importance of these receptors.

Using gene editing technology, the researchers plan on removing genes that code for these hormone receptors from model organisms. This step will allow them to test the effect that the loss of one or both of the receptors has on female stem cell expression levels. If the elimination of the sex hormone receptor diminishes stem cell expression, that may indicate that the receptor plays a regulatory role.

The Liang lab believes that results from these experiments will not only offer greater insight to the development of blood cancers, but also help in the formulation of sex-specific treatments. Liang hopes this research leads to treatments that enhance the male (blood cell producing) system to reduce risk of age-related blood cancer, or even other diseases.

Assistant Professor of Molecular Biology, Cell Biology and Biochemistry Mamiko Yajima studies the expression of germline molecules, which are normally only expressed during development, and how they contribute to plasticity, or the cells adaptability. Her pilot project will focus on the specific germline factor DEAD-Box Helicase 4 (DDX4), which has been found to be abnormally expressed in the tumors of certain cancers, such as small cell lung cancer and melanoma.

Yajimas lab has previously studied the expression of DDX4 in cells and organisms like sea urchins and mice. She plans to test if (DDX4) actually contributes to plasticity in the context of cancer. Yajima believes that as a germline factor, DDX4 may increase cancer cells adaptability, allowing them to develop drug resistance and migrate throughout the body more frequently.

The Yajima lab plans on using the Cancer Center funding to partner with Director of Thoracic Oncology at Rhode Island Hospital Christopher G. Azzoli and Associate Professor of Pathology and Laboratory Medicine Maria L. Garcia-Moliner to analyze DDX4 expression in cancer patient samples.

I applied for this funding with the specific goal to have access to clinical samples, Yajima said. This next stage of the project will facilitate collaboration between me, a basic biologist, and physician scientists that have the expertise to help me answer the question I want to study in a clinical setting.

To identify whether DDX4 expression correlates with patient survival, the lab will also use the funds to conduct clinical data mining of patient gene expression using the Universitys supercomputer.

Associate Professor of Dermatology and Epidemiology Eunyoung Cho studies the role of dietary factors in the development of chronic diseases. Previous work from Chos lab found that eating foods containing high levels of citrus, such as grapefruits, oranges and figs, is associated with an increased risk of skin cancer. The Cho lab plans to use the Cancer Center pilot project funds to determine the component of citrus fruit responsible for the increased risk of melanoma, the most fatal type of skin cancer.

Cho believes that furanocoumarins, a class of compounds present in high levels in citrus fruits, are what lead to the higher rates of skin cancer. These compounds can absorb ultraviolet radiation from sunlight and become activated, damaging DNA and causing mutations that can result in cancer.

To test this hypothesis, Cho has partnered with Associate Professor of Medical Science Elena Oancea, who specializes in melanoma research at the molecular level. They plan on measuring whether melanin-forming skin cells show increased levels of DNA damage when exposed to furanocoumarins and UV light.

If their data supports that furanocoumarins increase risk of cancer, this could open the door to population-based studies. Cho described one potential future direction as assessing whether furanocoumarin levels in human urine samples are indicative of melanoma risk.

Its very interesting to think about citrus fruit is something you eat all the time, Cho said. People dont understand that when you eat grapefruit (and) then go into the sunlight, you may actually increase your chance of (getting) skin cancer.

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

By daniellenierenberg

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

By daniellenierenberg

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

By daniellenierenberg

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

By daniellenierenberg

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

By daniellenierenberg

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

By daniellenierenberg

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

By daniellenierenberg

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

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

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