MONDAY, Aug. 22, 2022 (HealthDay News) -- The U.S. Food and Drug Administration has approved Zynteglo (betibeglogene autotemcel), the first cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions.

Zynteglo is a one-time, single-dose gene therapy product. Each dose of Zynteglo is customized and created using the patient's own bone marrow stem cells, which are genetically modified to produce functional beta-globin. The application was granted a rare pediatric disease voucher, as well as priority review, fast-track, breakthrough therapy, and orphan designations.

The approval was based on two multicenter clinical studies. Of 41 patients receiving Zynteglo, 89 percent achieved transfusion independence, defined as maintaining a predetermined level of hemoglobin without needing any red blood cell transfusions for at least 12 months. The most common adverse reactions seen with Zynteglo included reduced platelet and other blood cell levels, mucositis, febrile neutropenia, vomiting, fever, alopecia, nosebleed, abdominal pain, musculoskeletal pain, cough, headache, diarrhea, rash, constipation, nausea, decreased appetite, pigmentation disorder, and itch.

Given the potential risk for blood cancer associated with this treatment, patients receiving Zynteglo should have their blood monitored for at least 15 years for evidence of cancer. The FDA says patients should also be monitored for hypersensitivity reactions during Zynteglo administration and for thrombocytopenia and bleeding.

Approval of Zynteglo was granted to bluebird bio.

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FDA Approves First Cell-Based Gene Therapy for Beta-Thalassemia - HealthDay News

Clogging a proteins active site is a straightforward way to take it offline. Thats why the active site is often the first place drug designers look when designing new drugs, Reich explained. However, about eight years ago he decided to investigate compounds that could bind to other sites in an effort to avoid off-target effects.

As the group was investigating DNMT3A, they noticed something peculiar. While most of these epigenetic-related enzymes work on their own, DNMT3A always formed complexes, either with itself or with partner proteins. These complexes can involve more than 60 different partners, and interestingly, they act as homing devices to direct DNMT3A to control particular genes.

Early work in the Reich lab, led by former graduate student Celeste Holz-Schietinger, showed that disrupting the complex through mutations did not interfere with its ability to add chemical markers to the DNA. However, the DNMT3A behaved differently when it was on its own or in a simple pair; it wasnt to stay on the DNA and mark one site after another, which is essential for its normal cellular function.

Around the same time, the New England Journal of Medicine ran a deep dive into the mutations present in leukemia patients. The authors of that study discovered that the most frequent mutations in acute myeloid leukemia patients are in theDNMT3Agene. Surprisingly, Holz-Schietinger had studied the exact same mutations. The team now had a direct link between DNMT3A and the epigenetic changes leading to acute myeloid leukemia.

Reich and his group became interested in identifying drugs that could interfere with the formation of DNMT3A complexes that occur in cancer cells. They obtained a chemical library containing 1,500 previously studied drugs and identified two that disrupt DNMT3A interactions with partner proteins (protein-protein inhibitors, or PPIs).

Whats more, these two drugs do not bind to the proteins active site, so they dont affect the DNMT1 at work in all of the bodys other cells. This selectivity is exactly what I was hoping to discover with the students on this project, Reich said.

These drugs are more than merely a potential breakthrough in leukemia treatment. They are a completely new class of drugs: protein-protein inhibitors that target a part of the enzyme away from its active site. An allosteric PPI has never been done before, at least not for an epigenetic drug target, Reich said. It really put a smile on my face when we got the result.

This achievement is no mean feat. Developing small molecules that disrupt protein-protein interactions has proven challenging, noted lead authorJonathan Sandovalof UC San Francisco, a former doctoral student in Reichs lab. These are the first reported inhibitors of DNMT3A that disrupt protein-protein interactions.

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A new kind of chemo - University of California

When World Series champion Matt Szczur joined a bone marrow registry in 2007 as a freshman in college, he didnt think much of it. At the time, Szczur (pronounced like Caesar) was playing football for Villanova University. His coach, the legendary Andy Talley, encouraged him to get involved with an organization called Be The Match. As its name implies, the group matches bone marrow donors with recipients and raises money to help those suffering from life-threatening blood cancers, like leukemia and lymphoma, to provide necessary treatment.

I thought Id test [to be a match], and if I get a call, great, Szczur said in an interview with nft now. If not, it is what it is.

Fifteen years ago, the chances of matching a donor to a recipient were not what they are today. Bone marrow and cord blood unit donor-recipient pairing relies on matching human leukocyte antigen (HLA) types, something a patients ethnic background plays a prominent role in predicting. The more people in the registry, the larger the chances are of finding matches for people in need.

Szczur put it out of his mind until the fall of 2009 when he got a call from the Be The Match registry telling him they had found a recipient for his bone marrow type. It was right in the middle of playoffs, Szczur continued. I went and spoke with Talley, and he put his head down and said, Matt, I know youre going to do the right thing.

That match was a young girl named Anastasia Volkovskaya from Ukraine, whose doctors diagnosed her with leukemia just three months after being born.Doctors told us that she needs a transplant, explained Volkovskayas father Ivan in an ESPN video on Szczurs journey to becoming a donor produced for the sports-magazine broadcast series E:60. But in Ukraine, they dont do this. They said, Deal with it.

Volkovskayas parents kept searching for ways to save their daughter, eventually finding a clinic in Israel that could provide the transplant if they could find a matching donor. They found one in Szczur who was a 100 percent match. On May 4, 2010, Szczur donated his bone marrow in a peripheral blood stem cell (PBSC) procedure, a non-surgical operation in which doctors separate a patients blood-forming cells from the rest of the blood for bone marrow transplants.

It would take a while before Szczur learned precisely to whom he had donated. Confidentiality practices keep donors from learning the identity of recipients they match with for at least a year after the procedure. It deeply moved him when he finally learned Anatasias identity and got in contact with her and her family.

I remember like it was yesterday when I got the email from Be The Match about who she was and where shes from, Szczur said. When I was finally able to get in contact with her it was very emotional. I now have a son whos three. I understood the importance of [donating] then, but now, you know, Id do anything in the world to help my son, and Im sure thats how [ Volkovskayas parents] felt.

Volkovskayas parents appreciation for what Szczur did knows no bounds. This man gave life not just to one kid, he gave life to a whole family, said Anatasias mother Marina in ESPNs E:60 video.

Throughout his life, Szczur has engaged with art in one way or another. Whether sitting next to his father as a child and watching him make colorful bucktails (anglers lures traditionally made from deer hair) or experimenting with painting as a way to decompress from the stresses of being an athlete, its a part of life from which he never liked to stray very far.

After going on to play for Major League Baseball and winning the World Series with the Chicago Cubs in 2016, Szczur and his wife Natalie started a non-profit foundation dedicated to philanthropy and charity. To raise awareness for the foundation, Szczur decided to paint two self-portraits and auction them at an event the couple held in his hometown of Cape May, New Jersey. Both sold for $500. Cubs management later reached out to commission Szczur to make a painting of the teams World Series win, which the organization sold for $40,000.

Szczurs interest and involvement in NFTs would follow soon after. During the pandemic in 2020, fellow MLB player Micah Johnson reached out to Szczur to ask if he wanted to collaborate on an NFT project.I had read about the blockchain, but I had no idea what it was, Szczur said. I knew a little bit about Bitcoin, but Id never heard of Ethereum. I trusted Micah because he was a baseball player, and I knew he was a grinder. I respected him.

It doesnt get more real than someone who actually donated and saved the life of another human being.

Johnson suggested creating a portrait of George Floyd, and the two split the portrait in half, each depicting their side of Floyd in their own style. The limited edition piece sold out on Nifty Gateway in under six minutes, and the two used the funds raised from the painting to donate to initiatives dedicated to fighting injustices in the Black community.

Szczur has continued to expand into the world of crypto art, creating pieces that collectors can buy on OpenSea, Nifty Gateway, and SuperRare. His artwork frequently features bones and skeletons, which reflects the awareness hes trying to raise about bone marrow donations.

Having long wanted to work with Be The Match, Szczur found a golden opportunity to do so when a friend who had contacts at the bone marrow registry asked if he wanted to collaborate with the organization earlier this year.

Szczur and Be The Match are releasing two separate NFTs on Nifty Gateway in the coming days and weeks to raise awareness and money for the registry. The first is an open edition piece called Be The Match that drops on Friday, August 26, and will be available for sale for 48 hours. Editions start at $150 each. A second, limited-edition NFT drop will also be available through a 48-hour auction on Thursday, September 8, which Szczur says will be a different take on his previous skeleton and bone-themed NFT creations.

Half of all the proceeds from the NFT sales will go toward the Be The Match Foundation to help patients looking for a donor and add more potential donors to their registry.

This is Be The Matchs first experience in the NFT space, explained Alex Mensing, a Be The Match spokesperson in an interview with nft now. A partnership with Matt was a no-brainer. Hes been spreading awareness about bone marrow donations through his NFTs already, its something hes super passionate about. It doesnt get more real than someone who actually donated and saved the life of another human being. Thats our mission.

One of the reasons both Mensing and Szczur are excited to use NFTs as a way to spread awareness of the registry is because HLA types largely determine who can and cant match. The more diverse the registry, the greater the chances of those matches actually occurring.

I can say in all honesty that NFTs changed my life.

Right now, you see a disparity in the registry based on the ethnic diversity of the patient who is searching, elaborated Mensing. So today, a patient whos white or Caucasian has a 79 percent chance of finding a match on our registry. But Black or African American patient has only a 29 percent chance of finding a match. And so were doing a lot of recruitment to get more people to join the registry. And we have the most diverse registry in the world. Its just still not diverse enough to meet all of the patient need that exists.

Szczurs excitement for and dedication to the cause hes devoted the last decade to are admirable. Despite the maligned reputation people often attribute to NFTs and cryptocurrencies, hes adamant about their ability to do good in the world.

I can say in all honesty that NFTs changed my life, Szczur said. From top to bottom. I had no idea what I was going to do when I finished [playing] baseball. So, the transition from baseball to real life was helped so much by NFTs. I know people are skeptical about it, and they have the right to be. Cryptocurrency is so volatile. But they changed my life. Im grateful for the space.

The former MLB world champion is even more grateful for the chance to use the technology to spread awareness about a cause in which he firmly believes. His experience as a Be The Match donor is something he says will stick with him his entire life.

Theyre literally saving lives, Szczur emphasized. Everybody says theres no cure for cancer, but here we are doing this, and nobody really hears about it. So, I continue to push for this cure and for bone marrow awareness. I will continue to push this because I saw the impact that it had on my life and the impact it had on this familys life.

Those looking to support the foundations efforts and help save lives can add their names to Be The Matchs bone marrow registry here.

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A World Baseball Champion Is Using NFTs to Help Save Lives - nft now

Doctor with a cancer patient.

But, in order to defeat an enemy like cancer, you must first know more about it. Let's take a closer look at this dreadful disease.

Knowing the potential indicators of cancer is important regardless of your age or state of health. Symptoms are generally insufficient to diagnose the illness on their own. However, they may serve as hints for you and your physician to quickly identify and address the issue. Treatment for many types of cancer is most effective early on, when a tumor is tiny and the disease hasn't spread to other parts of the body.

The signs listed below are not exhaustive and do not necessarily indicate the presence of cancer. There are many common conditions that might share these symptoms. Visit your doctor as soon as possible so they can examine you more closely and take appropriate measures.

Cancer frequently exhibits the following symptoms in both men and women:

Artist's impression of cancer.

For men only, there are some sex-specific symptoms that could indicate the presence of cancer. For men, prostate, lung, and colorectal cancers are the most prevalent cancers that tend to develop.

Male cancer symptoms include:

Women, on the other hand, are more susceptible to some forms of cancer that are usually not expressed in males. Breast, lung, and colorectal cancer are some of the most common cancers in women, although men can also have these types of cancer. Cancers such as uterine, endometrial, cervical, vaginal, and vulvar cancers are particular to women.

Some of the warning signs for women include:

Every cancer starts in cells. More than a hundred billion billion (100,000,000,000,000) cells make up our bodies. Changes in one cell or a small number of cell can lead to the development of cancer.

Artistic impression of cancer cells.

When cells grow old or become damaged, they die, and new cells take their place. How much and how frequently cells divide is regulated by a number of factors, but sometimes this orderly process breaks down, and abnormal or damaged cells grow and multiply when they shouldn't. When cancer occurs, cells may begin to grow and multiply in an uncontrolled way, resulting in the formation of a mass known as a tumor, if any of these signals are damaged or absent.

The primary tumor is where cancer first appears. Some cancers, like leukemia, attack different parts of the body; in this case, the cancer originates in blood cells. With leukemia, however, tumors do not grow in a clump. Instead, cancer cells accumulate in the blood and, occasionally, the bone marrow.

However, any cell is technically vulnerable to becoming cancerous. While the functions of various cell types in the body vary, their basic functions are pretty much alike.

Every cell has a nucleus, which serves as its command center. Chromosomes, which contain thousands of genes, are located inside the nucleus. Long strands of DNA (deoxyribonucleic acid) are found in genes and serve as coded instructions for making proteins and other molecules.

Genes can be thought of as instructions for producing different products that the cells need. This may be a protein or regulatory molecules that help the cell assemble proteins. RNA. This process helps to control elements such as:

Genes ensure that cells develop and replicate (make copies) in an orderly and controlled manner, which is necessary to maintain the body's wellness.

When a cell divides, the genes can occasionally change. This is called a mutation. Ultimately, this means that a gene has been damaged or changed in some way.

A cell can randomly undergo a mutation while it is dividing. Some mutations result in the cell losing the ability to comprehend its own instructions. It might begin to outgrow control. One mutation is not generally enough to cause cancer. Usually, cancer occurs from multiple mutations over a period of time. That is one reason why cancer occurs more often in older people.

Gene mutations in a specific gene could signify that:

Cancer cells.

A damaged cell can take years to divide, grow, and develop into a tumor large enough to produce symptoms or be detected on a scan.

But what actually causes a cell's genes to mutate?

Since mutations can happen by chance when a cell is dividing, this can result in the cell becoming accidentally cancerous. But, mutations can also occur during the normal functions of cellular life.

Mutations can also be triggered by substances that enter the body from the outside, such as the substances in cigarette smoke. Sometimes, people also inherit genetic flaws that increase their risk of getting cancer.

Every day, some genes are damaged, but cells are quite good at fixing them. But the harm can worsen over time. Additionally, if cells develop too quickly, they are less able to repair the damaged genes and may be more prone to acquiring new mutations.

The first point to note is that not all cancers are fatal.

In England and Wales, for example, 50 out of every 100 (50%) people with cancer survive for ten years or more. In the UK, cancer survivorship is increasing and has doubled in the past 40 years. However, there is a huge variation in ten-year survival rates for different types of cancer, ranging from 98% for testicular cancer to just 1% for pancreatic cancer.

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These are some of the very worst cancers you could ever get - Interesting Engineering

AS one of the most common childhood cancers around, chances are you know what leukaemia is.

But can you actually name any of the symptoms associated with the deadly illness?

1

According to results of a recent survey, less than1 per cent of Britsare able to identify all four common symptoms of the cancer.

Leukaemia is a type of blood cancer that affects people of all ages, with 10,000 people getting diagnosed in the UK every year.

Overall survival rate for the disease stands at just over 50 per cent, making it one of the most deadly forms of cancer.

However, a survey conducted by two cancer charities found that over two-fifths (42 per cent) of people cannot recognise any of the four most widely reported symptoms of the disease.

It also revealed that 43 per cent people wrongly think leukaemia is most common in the under 24s.

Common leukaemia signs and symptoms include:

Leukaemia a type of blood cancer that affects cells in bone marrow and attacks the immune system.

The most common symptoms are:

Other symptoms of leukaemia include:

Fiona Hazell, chief executive ofLeukaemia UK-- one of the charities who published the survey-- believe this mistaken belief could cost lives.

"In reality, both incidence and mortality rates rise sharply after the age of 55," she explained.

"Raising awareness in this age group is critical in order to treat it early and effectively; and ultimately to improve survival rates overall," she said.

Like with all cancers, catching leukaemia early means treatment is more likely to be successful.

In response to the survey results, Leukaemia UKand Leukaemia Care have together launched a new campaign -- called #SpotLeukaemia -- to make people more aware of the symptoms.

Zack Pemberton-Whiteley, chief executive ofLeukaemia Care also said the results of the survey were "extremely worrying."

"It's crucial that if you think you have fatigue, bruising or bleeding or repeated infections that you contact your GP and ask for a blood test. It's as simple as that," he said.

There are four main types of leukaemia.

They are:

Acute Lymphocytic Leukaemia (ALL)-A rapidly progressing form of the disease.More common in children.

Acute Myeloid Leukaemia (AML) -Rapidly progressive. More common in adults.

Chronic Lymphocytic Leukaemia (CLL) -Slowly progressing form and more common in adults.

Chronic Myeloid Leukaemia (CML) -Progresses slowly and is more common in adults.

Cancer types, signs and symptoms

Everything you need to know about different types of Cancer

Currently there are five ways leukaemia can be treated.

They are:

Chemotherapy-These are cell-killing drugs which kill and/or stop them from dividing. Chemotherapy is often given in blocks or cycles of treatment.One cycle of treatment will consist of a series of doses of chemotherapy followed by a break for the healthy cells to recover.

Radiation therapy -This treatment uses high-energy radiation to kill cancer cells. It is not used to treat all types of leukaemia.

Targeted therapy -Drugs which specifically recognise and kill leukaemia cells.

Biological therapy -A treatment which uses the immune system to destroy leukaemia cells.

Stem cell transplant -Youngerpatients may be given a stem cell transplant (bone marrow transplant). This may be done using your own healthy stem cells or stem cells from a donor. This is most commonly done for acute leukaemia if chemotherapy does not cure the disease.

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Urgent warning as millions of Brits dont know the key signs of deadly cancer... - The US Sun

A type of blood cell cancer called angioimmunoblastic T-cell lymphoma (AITL) can develop as mutations accumulate with age in the stem cells from which the T cells in the blood develop.

However, the underlying mechanism by which AITL develops was unknown. Now, a team from the University of Tsukuba in Japan have shown that B cells, another type of blood cells, accumulate mutations in genes that control how the genetic material in the cell is packaged. These aberrant B cells then interact with T cells and lead to the development of AITL.

The paper, Clonal germinal center B cells function as a niche for T-cell lymphoma, was published in the journal Blood.

Blood cells such as B cells and T cells, involved in immunity, develop from stem cells in the bone marrow. Sometimes, mutations occur in individual stem cells that lead to the mutant stem cell giving an increased output of blood cells, all of which carry identical mutations.

The likelihood of this increases with age, known as age-related clonal hematopoiesis, or ACH. ACH is known to be linked to various cancers. AITL, a cancer of the T cells, is linked to ACH with mutations in a gene called TET2. The team used mouse models and human samples to show that theTET2 mutation needed to be present in all blood cells, not just the T cells, for a mouse model to develop AITL.

Using single-cell RNA sequencing, a technique that can show which genes are active within just one cell, they were able to profile the immune cells present in the samples. This revealed a significant increase in the number of aberrant germinal center B cells, a type of B cells with activating and proliferative capacities. These B cells showed recurrent mutations to genes for histone proteins, which organize the genetic material in the cell into higher structures. They also showed alterations to the pattern of a DNA modification called methylation, which affects the genes expressed in the cell.

B cells and T cells can interact through molecules on the cell surface known as CD40 and CD40 ligand.

Analysis of the single-cell sequencing data identified this interaction between CD40 and CD40 ligand as potentially essential for mediating crosstalk between the aberrant B cells and the tumor cells, said lead author Manabu Fujisawa.

Most importantly, the survival of the mice with AITL could be prolonged by treatment with an antibody designed to inhibit CD40 ligand, said main author Mamiko Sakata-Yanagimoto.

The genes expressed in the aberrant mouse GCB cells are also expressed in cells from human AITL withTET2 mutations.

This means that antibodies against CD40 ligand could potentially be a new therapeutic approach to human AITL.

The rest is here:
University of Tsukuba researchers understand the mechanism of AITL - Labiotech.eu

Certain patients with Wilm's tumor-expressing solid tumors are eligible to be treated with an investigational agent in a phase 1 clinical trial.

The first patient was dosed in a phase 1 dose escalation study (NCT05360680) which is evaluating CUE-102, of the interleukin 2 (IL-2) series, as monotherapy for the treatment of patients with Wilms Tumor 1 (WT1)-positive recurrent/metastatic cancers. The study will focus on colorectal, gastric, pancreatic, and ovarian cancers, according to a press release from Cue Biopharma.1

CUE-102 has the potential to activate the patients immune system against numerous WT1-expressing cancers, including solid tumors and hematologic malignancies, and has demonstrated selective and significant activation of WT1-specific T cells in preclinical studies. We believe that CUE-102 can play an important role in changing the treatment landscape for patients with WT1-positive cancers, by potentially delivering higher efficacy and lower toxicities than current available treatments, said Ken Pienta, MD, acting chief medical officer of Cue Biopharma, in a press release.

In a preclinical study of CUE-102 tested humans with peripheral blood mononuclear cells (PBMC) for cellular activity and specificity, while in vivo CUE-102 is testing in mice who are human leukocyte antigen HLA-A2 transgenic. Investigators found that CUE-102 both activates and expands WT1-specific CD8-positive T cells from PBMC of healthy donors. Similar to results seen with CUE-101, CUE-102 showed significant functional reduction of the IL-2 components.The in vivo study found CUE-102 expands polyfunctional WT1-specific CD8-positive T cells from mice who were nave and previously immunized, but the treatment did not alter the frequencies of other immune lineages.The in vivo study also found the WT1-specific CD8-positive T cells had a polyfunctional response to peptide-loaded target cells and selectively killed WT1-presenting target cells.These results, along with a tolerable safety profile, support the initiation of the phase 1 trial of CUE-102.2

This phase 1 study aims to find the dose-limiting toxicity and maximum-tolerated dose of CUE-102. The secondary end points include safety and tolerability, antitumor response rate, antitumor duration of response, antitumor clinical benefit rate, progression-free survival, and overall survival. The dose escalation portion of the trial will administer CUE-102 in intravenous doses of 1 mg/kg, 2 mg/kg, 4 mg/kg, 8 mg/kg, and an experimental recommended phase 2 dose every 3 weeks for up to 2 years.3

Eligible patients in this phase 1 study will include patients who have colorectal, gastric, pancreatic, or ovarian cancer who have an ECOG performance status of 0 or 1, a life expectancy of at least 12 weeks, have a human leukocyte antigen (HLA)-A*0201 genotype as determined by genomic testing, and tumors who are WT1-positive. Patients will be deemed ineligible if patients with central nervous system metastases have been treated and be asymptomatic, have a history of prior allogeneic bone marrow, stem cell, or solid organ transplant, treatment with radiation therapy within 14 days before the first dose of CUE-102, and a history of clinically significant cardiovascular disease.3

Initiating this phase 1 clinical study of CUE-102 at a starting dose of 1mg/kg, a clinically active dose in our Phase 1 CUE-101 clinical trial for HPV+ head and neck cancer, is an important step forward in demonstrating the modularity of our Immuno-STAT platform and the broader clinical potential of our CUE-100 series of biologics, said Dan Passeri, chief executive officer of Cue Biopharma, in the press release. We believe, given the preservation of the core molecular framework between CUE-102 and CUE-101 with the primary exception of the tumor-specific epitope, initiating the dose escalation trial at 1 mg/kg will result in reduced time and cost to evaluate tolerability at therapeutically active doses.

References

1. Cue biopharma doses first patient in phase 1 study of CUE-102 for Wilms Tumor 1 (WT1) - expressing cancers. Press release. Cue Biopharma; August 22, 2022. Accessed August 23, 2022. https://www.cuebiopharma.com/investors-media/news/

2. Zhang C, Girgis N, Merazga Z, et al. 720CUE-102 selectively activates and expands WT1-specific T cells for the treatment of patients with WT1+ malignancies. Journal for ImmunoTherapy of Cancer. 2021;9:doi: 10.1136/jitc-2021-SITC2021.720

3. A phase 1 in patients with HLA-A*0201+ and WT1+ recurrent/metastatic cancers. ClinicalTrials.gov. Updated July 7, 2022. Accessed August 23, 2022. https://clinicaltrials.gov/ct2/show/record/NCT05360680?term=NCT05360680&draw=2&rank=1&view=recor

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Phase 1 Trial of CUE-102 in WT1+ Solid Tumors is Now in Motion - Targeted Oncology

MIAMI, Aug. 25, 2022 (GLOBE NEWSWIRE) -- LongeveronInc. (NASDAQ: LGVN),a clinical stage biotechnology company developing cellular therapies for chronic, aging-related and life-threatening conditions, today announced that the European Patent Office (EPO) has issued a notice of its intent to grant the Company a patent (EP Application No. 15861319.0) related to methods to treat endothelial dysfunction and monitor the efficacy of allogeneic mesenchymal cell therapies, also known as medicinal signaling cells (MSCs). The cells are administered to patients with cardiovascular disease through the monitoring of a protein, Vascular Endothelial Growth Factor (VEGF), which is a signal protein produced by many cells that stimulates the formation of blood vessels.

We are extremely pleased to receive this notice from the European patent office, said Chris Min, M.D., Ph.D., Interim Chief Executive Officer and Chief Medical Officer at Longeveron. This patent will bolster our robust intellectual property portfolio and support our goal of delivering effective cell therapies for a range of aging-related and life-threatening conditions.

The patent is titled Methods for Monitoring Efficacy of Allogeneic Mesenchymal Stem Cell Therapy in a Subject. Longeverons lead investigational product is Lomecel-B, a cell therapy product derived from MSCs. Many of Longeverons clinical studies point to Lomecel-B exerting effects through pro-vascular functions and/or reducing endothelial dysfunction, a condition where the lining of blood vessels is abnormal leading to diminished health of blood vessels and blood flow regulation.

The Company is evaluating the use of MSCs to treat several indications, including Hypoplastic Left Heart Syndrome (HLHS), a rare and life-threatening congenital heart defect that affects approximately 1,000 babies per year. Longeveron received both a Rare Pediatric Disease Designation and Orphan Drug Designation from the United States Food and Drug Administration in 2021 for Lomecel-B for the treatment of infants with HLHS. Longeveron is currently evaluating Lomecel-B for HLHS in a Phase 2a trial.

Longeveron is also conducting a trial of Lomecel-B in patients with Alzheimers Disease in the US and for aging frailty in Japan.

Now that the European Patent Office has issued an Intention to Grant, Longeveron will await grant of the patent and then begin the process of registering the patent in a number of nation members of the European Patent Organization. In those jurisdictions where the patent is registered, the patent is expected to expire in November of 2035.

About Longeveron Inc.

Longeveron is a clinical stage biotechnology company developing cellular therapies for specific aging-related and life-threatening conditions. The Companys lead investigational product is the Lomecel-B cell-based therapy product, which is derived from culture-expanded medicinal signaling cells (MSCs) that are sourced from bone marrow of young, healthy adult donors. Longeveron believes that by using the same cells that promote tissue repair, organ maintenance, and immune system function, it can develop safe and effective therapies for some of the most difficult disorders associated with the aging process and other medical disorders. Longeveron is currently sponsoring Phase 1 and 2 clinical trials in the following indications: Alzheimers disease, hypoplastic left heart syndrome (HLHS), Aging Frailty, and Acute Respiratory Distress Syndrome (ARDS). Additional information about the Company is available at http://www.longeveron.com.

Cautionary Note Regarding Forward-Looking Statements

Certain statements in this press release that are not historical facts are forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, which reflect management's current expectations, assumptions, and estimates of future performance and economic conditions, and involve risks and uncertainties that could cause actual results to differ materially from those anticipated by the statements made herein. Forward-looking statements are generally identifiable by the use of forward-looking terminology such as "believe," "expects," "may," "looks to," "will," "should," "plan," "intend," "on condition," "target," "see," "potential," "estimates," "preliminary," or "anticipates" or the negative thereof or comparable terminology, or by discussion of strategy or goals or other future events, circumstances, or effects. Factors that could cause actual results to differ materially from those expressed or implied in any forward-looking statements in this release include, but are not limited to, statements about the ability of Longeverons clinical trials to demonstrate safety and efficacy of the Companys product candidates, and other positive results; the timing and focus of the Companys ongoing and future preclinical studies and clinical trials and the reporting of data from those studies and trials; the size of the market opportunity for the Companys product candidates, including its estimates of the number of patients who suffer from the diseases being targeted; the success of competing therapies that are or may become available; the beneficial characteristics, safety, efficacy and therapeutic effects of the Companys product candidates; the Companys ability to obtain and maintain regulatory approval of its product candidates; the Companys plans relating to the further development of its product candidates, including additional disease states or indications it may pursue; existing regulations and regulatory developments in the U.S., Japan and other jurisdictions; the Companys plans and ability to obtain or protect intellectual property rights, including extensions of existing patent terms where available and its ability to avoid infringing the intellectual property rights of others; the need to hire additional personnel and the Companys ability to attract and retain such personnel; the Companys estimates regarding expenses, future revenue, capital requirements and needs for additional financing; the Companys need to raise additional capital, and the difficulties it may face in obtaining access to capital, and the dilutive impact it may have on its investors; the Companys financial performance, and the period over which it estimates its existing cash and cash equivalents will be sufficient to fund its future operating expenses and capital expenditures requirements. Further information relating to factors that may impact the Company's results and forward-looking statements are disclosed in the Company's filings with the Securities and Exchange Commission, including Longeverons Annual Report on Form 10-K for the year ended December 31, 2021, filed with the SEC on March 11, 2022, and the Companys Quarterly Reports on Form 10-Q for the periods ended March 31, 2022, and June 30, 2022. The forward-looking statements contained in this press release are made as of the date of this press release, and the Company disclaims any intention or obligation, other than imposed by law, to update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise.

Investor Contact:

Elsie YauStern IR, Inc.212-698-8700elsie.yau@sternir.com

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New York, Aug. 23, 2022 (GLOBE NEWSWIRE) -- Kenneth Research has published a detailed market report on Global Cell Banking Outsourcing Market for the forecast period, i.e., 2022 2031, which includes the following factors:

Global Cell Banking Outsourcing Market Size:

The global cell banking outsourcing market generated the revenue of approximately USD 7200.1 million in the year 2021 and is expected to garner a significant revenue by the end of 2031, growing at a CAGR of ~18% over the forecast period, i.e., 2022 2031. The growth of the market can primarily be attributed to the development of advanced preservation techniques for cells, and increasing adoption of regenerative cell therapies for the treatment of chronic diseases such as cancer. Additionally, factors such as growing demand for gene therapy, and increasing worldwide prevalence of cancer are expected to drive the market growth. According to the World Health Organization, nearly 10 million people died of cancer across the globe in 2020. The most recurrent cases of deaths because of cancer were lung cancer which caused 1.80 million deaths, colon, and rectum cancer which caused 916 000 deaths, liver cancer which caused 830 000 deaths, stomach cancer which caused 769 000 deaths, and breast cancer which caused 685 000 deaths. Furthermore, it was noticed that about 30% of cancer cases in low and lower-middle income nations are caused by cancer-causing diseases such the human papillomavirus (HPV) and hepatitis.

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Global Cell Banking Outsourcing Market: Key Takeaways

Increasing Geriatric Population across the Globe to Boost Market Growth

Increasing demand for stem cell therapy, and increasing biopharmaceutical production are estimated to fuel the growth of the global cell banking outsourcing market. Among the geriatric population around the world, the demand of stem cell therapy is at quite a high rate. Hence, growing geriatric population across the globe is also expected be an important factor to influence the market growth. According to the data by World Health Organisation (WHO), the number and proportion of geriatric population, meaning the people aged 60 years and older in the population is rising. The number of people aged 60 years and older was 1 billion in 2019. This number is estimated to increase to 1.4 billion by 2030 and 2.1 billion by 2050.

In addition to this, increasing prevalence of chronic diseases, supportive initiatives by governments around the world, and growing awareness about stem cell banking are predicted to be major factors to propel the growth of the market. The growth of the global cell banking outsourcing market, over the forecast period, can be further ascribed to the rising investments in the R&D activities to continuously bring up more feasible solutions for medical procedures. According to research reports, since 2000, global research and development expenditure has more than tripled in real terms, rising from approximately USD 680 billion to over USD 2.5 trillion in 2019.

Browse to access In-depth research report on Global Cell Banking Outsourcing Market with detailed charts and figures: https://www.kennethresearch.com/report-details/cell-banking-outsourcing-market/10070777

Global Cell Banking Outsourcing Market: Regional Overview

The global cell banking outsourcing market is segmented into five major regions including North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa region.

Advanced Healthcare Facilities Drove Market in the North America Region

The market in the North America region held the largest market share in terms of revenue in the year 2021. The growth of the market in this region is majorly associated with the increasing number of pharmaceutical companies & manufacturers in the region, and increasing awareness for the use of stem cells as therapeutics. Increasing number of bone marrow and cord blood transplants throughout the region is also estimated to positively influence the market growth. It was noted that, 4,864 unrelated and 4,160 related bone marrow and cord blood transplants were performed in the United States in 2020.

Increasing Prevalence of Chronic Diseases to Influence Market Growth in the Asia Pacific Region

On the other hand, market in the Asia Pacific region is estimated to grow with the highest CAGR during the forecast period. The market in this region is driven by the increasing investment in biotechnology sector by government and private companies specifically in countries such as China, India, and Japan. Moreover, the increasing pool of patient with chronic diseases, such as cancer, and the ongoing research & development activities for cancer treatment is expected to propel the growth of the market. Further, increasing percentage of regional health expenditure contributing to the GDP is also estimated to be a significant factor to influence the growth of the cell banking outsourcing market in the Asia Pacific region. As per The World Bank, in the year 2019, share of global health expenditure in East Asia & Pacific region accounted to 6.67% of GDP.

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The study further incorporates Y-O-Y growth, demand & supply and forecast future opportunity in:

Middle East and Africa (Israel, GCC [Saudi Arabia, UAE, Bahrain, Kuwait, Qatar, Oman], North Africa, South Africa, Rest of Middle East and Africa).

Global Cell Banking Outsourcing Market, Segmentation by Bank Phase

The bank storage segment held the largest market share in the year 2021 and is expected to maintain its share by growing with a notable CAGR during the forecast period. The market growth is anticipated to be driven by the development of effective preservation technologies such as cryopreservation technique. Cryopreservation is a technique in which low temperature is used to preserve the living cells and tissue for a longer time. With the growing healthcare expenditure per capita across the world, demand for bank storage increasing notably. As sourced from The World Bank, in 2019, worldwide health expenditure per capita was USD 1121.97.

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Global Cell Banking Outsourcing Market, Segmentation by Product

The adult cell banking segment is estimated to hold a substantial market share in the global cell banking outsourcing market during the forecast period. The growth of this segment can be attributed to the significant prevalence of chronic diseases among the adults around the globe. For instance, according to the National Library of Medicine 71.8% of adult population suffered from cardiovascular diseases, 56% had diabetes, and 14.7% adults had arthritis as of 2020.

Global Cell Banking Outsourcing Market, Segmentation by Cell Type

Global Cell Banking Outsourcing Market, Segmentation by Bank Type

Few of the well-known market leaders in the global cell banking outsourcing market that are profiled by Kenneth Research are SGS SA, WuXi AppTec, LifeCell International Pvt. Ltd., BSL Bioservice, LUMITOS AG, Cryo-Cell International, Inc., REPROCELL Inc, CORDLIFE GROUP LIMITED, Reliance Life Sciences, and Clean Biologics and others.Enquiry before Buying This Report @ https://www.kennethresearch.com/sample-request-10070777

Recent Developments in the Global Cell Banking Outsourcing Market

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Global Cell Banking Outsourcing Market to Grow at a CAGR of ~18% during 2022-2031; Market to Expand Owing to the Development of Advanced Cell...

MONDAY, Aug. 22, 2022 (HealthDay News) -- The COVID-19 pandemic was associated with a decrease in transplantation in 2020, according to a study published in the July 1 issue of the American Journal of Surgery.

Alejandro Suarez-Pierre, M.D., from the University of Colorado School of Medicine in Aurora, and colleagues examined adult transplantation data as time series data in a population-based cohort study. Models of transplantation rates were developed using data from 1990 to 2019 to project the expected 2020 rates in a theoretical scenario in which the pandemic did not occur. Observed-to-expected (O/E) ratios were calculated for transplants.

The researchers found that 32,594 transplants were expected in 2020, but 30,566 occurred (O/E, 0.94; 95 percent confidence interval, 0.88 to 0.99). A total of 50,241 waitlist registrations occurred compared with 58,152 expected (O/E, 0.86; 95 percent confidence interval, 0.80 to 0.94). For kidney, liver, heart, and lung, the O/E ratios (95 percent confidence intervals) of transplants were 0.92 (0.86 to 0.98), 0.96 (0.89 to 1.04), 1.05 (0.91 to 1.23), and 0.92 (0.82 to 1.04), respectively. The corresponding O/E ratios (95 percent confidence intervals) of waitlist registrations were 0.84 (0.77 to 0.93), 0.95 (0.86 to 1.06), 0.99 (0.85 to 1.18), and 0.80 (0.70 to 0.94).

"The COVID-19 pandemic was associated with a significant deficit in solid organ transplantation, donation, and waitlist registrations in the United States in 2020. The impact was strongest in kidney transplantation and waitlist registration," the authors write. "While the pandemic persisted through 2020, the transplant system adapted remarkably well with a record number of transplantations performed."

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Drop Seen in Transplantation in 2020 With COVID-19 Pandemic - Consumer Health News | HealthDay - HealthDay News

SOMERVILLE, Mass.--(BUSINESS WIRE)--Cellarity, a life sciences company founded by Flagship Pioneering to transform the way medicines are created, announced today the release of a unique single-cell dataset to accelerate innovation in mapping multimodal genetic information across cell states and over time. This dataset will be used to power a competition hosted by Open Problems in Single-Cell Analysis.

Cells are among the most complex and dynamic systems and are regulated by the interplay of DNA, RNA, and proteins. Recent technological advances have made it possible to measure these cellular features and such data provide, for the first time, a direct and comprehensive view spanning the layers of gene regulation that drive biological systems and give rise to disease.

Advancements in single-cell technologies now make it possible to decode genetic regulation, and we are excited to generate another first-of-its-kind dataset to support Open Problems in Single Cell Analysis, said Fabrice Chouraqui, PharmD, CEO of Cellarity and a CEO-Partner at Flagship Pioneering. Developing new machine learning algorithms that can predict how a single-cell genome can drive a diversity of cellular states will provide new insights into how cells and tissues move from health to disease and support informed design of new medicines.

To drive innovation for such data, Cellarity generated a time course profiling in vitro differentiation of blood progenitors, a dataset designed in collaboration with scientists at Yale University, Chan Zuckerberg Biohub, and Helmholtz Munich. This time course will be used to power a competition to develop algorithms that learn the underlying relationships between DNA, RNA, and protein modalities across time. Solving this open problem will help elucidate complex regulatory processes that are the foundation for cell differentiation in health and disease.

While multimodal single-cell data is increasingly available, methods to analyze these data are still scarce and often treat cells as static snapshots without modeling the underlying dynamics of cell state, said Daniel Burkhardt, Ph.D., cofounder of Open Problems in Single-Cell Analysis and Machine Learning Scientist at Cellarity. New machine learning algorithms are needed to learn the rules that govern complex cell regulatory processes so we can predict how cell state changes over time. We hope these new algorithms can augment the value of existing or future single-modality datasets, which can be cost effectively generated at higher quality to streamline and accelerate research.

In 2021, Cellarity partnered with Open Problems collaborators to develop the first benchmark competition for multimodal single-cell data integration using a first-of-its-kind multi-omics benchmarking dataset (NeurIPS 2021). This dataset was the largest atlas of the human bone marrow measured across DNA, RNA, and proteins and was used to predict one modality from another and learn representations of multiple modalities measured in the same cells. The 2021 competition saw winning submissions from both computational biologists with deep single-cell expertise and machine learning practitioners for whom this competition marked their first foray into biology. This translation of knowledge across disciplines is expected to drive more powerful algorithms to learn fundamental rules of biology.

For 2022, Cellarity and Open Problems are extending the challenge to drive innovation in modeling temporal single-cell data measured in multiple modalities at multiple time points. For this years competition, Cellarity generated a 300,000-cell time course dataset of CD34+ hematopoietic stem and progenitor cells (HSPC) from four human donors at five time points. HSPCs are stem cells that give rise to all other cells in the blood throughout adult life, and a 10-day time course captures important biology in CD34+ HSPCs. Being able to solve the prediction problems over time is expected to yield new insights into how gene regulation influences differentiation.

Entries to the competition will be accepted until November 15, 2022. For more information, visit the competition page on Kaggle.

About Open Problems in Single Cell Analysis

Open Problems in Single-Cell Analysis was founded in 2020 bringing together academic, non-profit, and for-profit institutions to accelerate innovation in single-cell algorithm development. An explosion in single-cell analysis algorithms has resulted in more than 1,200 methods published in the last five years. However, few standard benchmarks exist for single-cell biology, both making it difficult to identify top performing algorithms and hindering collaboration with the machine learning community to accelerate single-cell science. Open Problems is a first-of-its-kind international consortium developing a centralized, open-source, and continuously updated framework for benchmarking single-cell algorithms to drive innovation and alignment in the field. For more information, visit https://openproblems.bio/.

About Cellarity

Cellaritys mission is to fundamentally transform the way medicines are created. Founded by Flagship Pioneering in 2017, Cellarity has developed unique capabilities combining high-resolution data, single cell technologies, and machine learning to encode biology, predict interventions, and purposefully design breakthrough medicines. By focusing on the cellular changes that underlie disease instead of a single target, Cellaritys approach uncovers new biology and treatments and is applicable to a vast array of disease areas. The company currently has programs underway in metabolic disease, hematology, immuno-oncology, and respiratory disease. For more info, visit http://www.cellarity.com.

About Flagship Pioneering

Flagship Pioneering conceives, creates, resources, and develops first-in-category bioplatform companies to transform human health and sustainability. Since its launch in 2000, the firm has, through its Flagship Labs unit, applied its unique hypothesis-driven innovation process to originate and foster more than 100 scientific ventures, resulting in more than $100 billion in aggregate value. To date, Flagship has deployed over $2.9 billion in capital toward the founding and growth of its pioneering companies alongside more than $19 billion of follow-on investments from other institutions. The current Flagship ecosystem comprises 41 transformative companies, including Denali Therapeutics (NASDAQ: DNLI), Evelo Biosciences (NASDAQ: EVLO), Foghorn Therapeutics (NASDAQ: FHTX), Moderna (NASDAQ: MRNA), Omega Therapeutics (NASDAQ: OMGA), Rubius Therapeutics (NASDAQ: RUBY), Sana Biotechnology (NASDAQ: SANA), and Seres Therapeutics (NASDAQ: MCRB).

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Cellarity Releases Novel, Open-Source, Single-Cell Dataset and Invites the Machine Learning and Computational Biology Communities to Develop New...

My gold standard would be the use of an orthobiologic joint treatment, such as autologous protein solution, as it would be the least invasive joint treatment for a pregnant mare as only one injection is recommended, while other regenerative products often recommend a series of treatments for best effects, she adds.

Orthobiologic treatments are processed using the horses own blood, often stallside, to isolate properties beneficial to the joint. These treatments can provide targeted pain relief without the potential negative physiological and metabolic side effects (e.g., spontaneous abortion, harm to the fetus) of steroid use. The downside is these treatments are significantly more expensive than steroids.

In most cases I would feel comfortable injecting a fetlock joint in a pregnant mare with a low dose of a steroid such as triamcinolone if I felt it was indicated for the quality of life of the mare and orthobiologic treatments were not an option financially, Crosby says. Treatment with polyacrylamide (hydro)gel (PAAG) could also be considered, although (manufacturers of) these formulations often recommend pretreatment with a steroid for best effects. Polyacrylamide gel works by reducing friction in a joint, which can be a very effective option for advanced arthritis.

Fitz is a 14-year-old Morgan gelding with early onset pituitary pars intermedia dysfunction (PPID, formerly equine Cushings disease). His condition is well-controlled on 1 milligram of pergolide (Prascend) daily, and his owner shows him regularly in saddle seat shows. Earlier this year Fitz was diagnosed with coffin joint osteoarthritis. He improved on a polysulfated glycosaminoglycan (Adequan) series but is still experiencing performance issues.

Bonny Henderson, DVM, IVCA, CVA, CREP, owner of Henderson Equine Clinic, in Avon, New York, is a huge proponent of adjunct therapies. Prior to injections she recommends her clients try a variety of nutraceuticals to provide building blocks for the healing process and to decrease systemic inflammation. She says shes had incredible results with this approach.

I like to combine Eastern and Western medicine for my patients, Henderson says. I try to get people to treat the whole horse, identify what is causing the lameness and why exactly the cause occurredin other words, the functional limitations predisposing the horse to the injury itself. Then I treat both the lameness and the underlying cause.

This case requires careful attention because of the horses endocrine disease. PPID horses can be more sensitive to steroids, and this can result in laminitis, says Henderson. Even if hes well-maintained, you have to take into account his body condition score and hoof capsule. If the hoof capsule contains external growth rings wider in the heel than the toe, youve likely had some prior clinical or subclinical (not showing obvious signs) laminitic episodes. There is a lot of concussion going through these horses feet; ground force reactions are much more pronounced due to the shoeing package and actions of the horse and have a greater impact on the hoof. You have to watch out for a subclinical condition of what we used to term road founder that would compound the metabolic issue of PPID.

In these complex cases Henderson says she reaches for an orthobiologic. I would first ultrasound this joint to visualize the health of the cartilage, she says. If there is cartilage present, I would start with an autologous protein solution. If the lameness is from an injury or if there is a lot of (inflammation in the joint fluid), I would recommend injecting -2 macroglobulin, followed by the autologous protein solution once the inflammation is resolved.

The -2 macroglobulin injections are relatively novel treatments in equine practice. This orthobiologic isolates the horses natural -2 macroglobulin, a potent anti-inflammatory with molecules typically too large to cross into the joint. The veterinarian can then inject the -2 macroglobulin into the joint to reduce the synovitis (joint inflammation) without the negative effects of corticosteroids.

Clover is a 22-year-old Thoroughbred mare. She is a retired racehorse-turned-jumper-turned-dressage horse. Her multitude of careers has left her with relatively severe carpal arthritis of her right forelimb, with osteochondral fragments and excessive bony changes. She has a very caring owner who has tried just about anything to keep the mare comfortable, including rounds of polysulfated glycosaminoglycan, intravenous hyaluronic acid, and systemic anti-inflammatories. Clover is still lame and resistant to flexion of the carpus. This joint is end-stage.

When osteophytes (bone spurs) are present in a joint, they are not usually the direct cause of a horses pain. In my experience, the greater pain comes from synovitis and the lack of cartilage. I would talk to the owner about -2 macroglobulin, because these cases often require a multiple-layered treatment plan, says Henderson. I would also recommend following the -2 macroglobulin with a 2.5% polyacrylamide hydrogel once the severe inflammation is controlled.

Researchers have shown that the 2.5% PAAG provides the synovial lining with structure and stability and facilitates increased production of joint fluid. The integration of the product into the membrane, thickening the structure, also provides shock absorption. It essentially increases joint lubrication and provides a cushion in these end-stage joints.

Often, horses with end-stage OA stop responding to medical management, at which point surgical fusion of the joint can offer long-term comfort.

Cole is an 8-year-old Warmblood stallion who competes in the hunter/jumper ring. His attending veterinarian has diagnosed him via radiographs and computed tomography with osteoarthritic changes in his distal cervical vertebrae, causing a left forelimb lameness.

Cervical pain and dysfunction in the horse has become increasingly recognized as a cause of poor performance and can be more involved than just pain originating from the joint proper, says Michael Caruso III, VMD, Dipl. ACVS-LA, owner of Reedsdale Equine Specialists, in Nashville, Tennessee, who specializes in equine lameness diagnosis and treatment.

While OA can affect any joint, the cervical vertebrae can be an insidious location. Osteoarthritis of the cervical articular process joints (is) obviously a disease of the cartilage surface and bone, but other structures are involved and intimately associated with the joint, including the joint capsule, synovium, subchondral bone (beneath the cartilage), and paraspinal muscles, Caruso explains.

Because cervical OA is so complex, vets must combine multiple methods to treat it. I believe that many horses with cervical facet joint pain/osteoarthritis require a multimodal approach to treatment depending upon the age of the horse and severity of the dysfunction, he says. We know that horses with neck arthritis can present with a wide range of issues, from poor performance and intermittent forelimb lameness to ataxia (incoordination).

Cervical joint OA can disrupt the adjacent spinal cord nerve roots, causing this neurologic manifestation.

Injection must be performed using ultrasound guidance, Caruso says. I would inject the articular process joints with a corticosteroid (betamethasone or triamcinolone), plus or minus hyaluronic acid, plus or minus (the antimicrobial) amikacin and, depending on the horses range of motion and muscle tension, might prescribe a muscle relaxant (methocarbamol) and/or perform mesotherapy and shock wave for any muscular/fascial pain adjacent to the affected joints.

Caruso says he would take any metabolic issues into account before injecting corticosteroids. In horses that have some sort of metabolic dysfunction, I will routinely utilize orthobiologics in the affected joints, he says, adding that his personal preferences are platelet-rich plasma (PRP) and autologous conditioned serum.

All horses treated for cervical pain are prescribed therapeutic rehabilitation, Caruso says. Dynamic exercises of not only the cervical region but also the whole body appear to positively affect muscle activation and strengthening. The exercise program aims to improve joint stability and range of motion by focusing on the deep paravertebral muscles.

Remington is a 6-year-old Warmblood gelding who competes in the jumpers. He becomes lame in the right hind, and his veterinarian isolates the lameness to the stifle. On imaging, the medial (toward the midline) meniscus looks enlarged and mottled. He is referred for an arthroscopy of his medial femorotibial joint. The surgeon suggests injecting it six weeks following the procedure.

With this case, Caruso says hed first recommend injecting mesenchymal stem cells (MSCs) into the medial femorotibial joint. While we have lots to learn about stem cells, in the stifleespecially postoperatively with meniscal damagestem cells have been shown to improve the long-term outcome for return to work in the horse, he says.

He cites one study (Ferris et al., 2014) on the outcome of horses undergoing stifle surgery plus bone-marrow-derived mesenchymal stem cell injections. The researchers found that of the horses treated for stifle injury with surgery and stem cells, 75% returned to some level of work postoperatively, which compares to previous reports of 60-63% with surgery alone.

The stifle is one joint that I will recommend injection with bone-marrow-derived MSCs following arthroscopic surgery as a first-line treatment, Caruso says. This is one joint that I believe stem cells have an advantage if finances allow.

Veterinarians typically harvest stem cells from the horse at the time of surgery, usually from the sternum, pelvis, or tibia. While they have potent healing factors, they are generally more expensive than the other orthobiologics mentioned. If an owner cant afford that price tag following stifle surgery, Caruso recommends PRP.

Clinically, I see a great response (from PRP) both in soft tissues and in joints, he says. I feel that MSC injection in stifle cases with proper rehabilitation following meniscal injury are more successful with less convalescent time.

While joint injection techniques are well-documented, the tricky part is what goes into the syringe. Gone are the days of simple corticosteroid injections as our only optionthough theyre still used and have a place in equine medicine. The insights from these veterinarians show we have several ways to approach a lameness, especially a complicated joint case.

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Equine Joint Injections: Case by Case The Horse - TheHorse.com

Tracing features in a large 3D electron microscopy dataset reveals a zebrafish blood stem cell (in green) and its surrounding niche support cells, a group photo method that will help researchers understand factors that contribute to blood stem cell health which could in turn help develop therapies for blood diseases and cancers. Image by Keunyoung Kim.

MADISON For the first time, researchers can get a high-resolution view of single blood stem cells thanks to a little help from microscopy and zebrafish.

Researchers at the University of WisconsinMadison and the University of California San Diego have developed a method for scientists to track a single blood stem cell in a live organism and then describe the ultrastructure, or architecture, of that same cell using electron microscopy. This new technique will aid researchers as they develop therapies for blood diseases and cancers.

Currently, we look at stem cells in tissues with a limited number of markers and at low resolution, but we are missing so much information, says Owen Tamplin, an assistant professor in UWMadisons Department of Cell & Regenerative Biology, a member of the Stem Cell & Regenerative Medicine Center, and a co-author on the new study, which was published Aug. 9 in eLife. Using our new techniques, we can now see not only the stem cell, but also all the surrounding niche cells that are in contact.

The niche is a microenvironment found within tissues like the bone marrow that contain the blood stem cells that support the blood system. The niche is where specialized interactions between blood stem cells and their neighboring cells occur every second, but these interactions are hard to track and not clearly understood.

As a part of the new study, Tamplin and his co-lead author, Mark Ellisman, a professor of neuroscience at UC San Diego, identified a way to integrate multiple types of microscopic imaging to investigate a cells niche. With the newly developed technique that uses confocal microscopy, X-ray microscopy, and serial block-face scanningelectron microscopy, researchers will now be able to track the once elusive cell-cell interactions occurring in this space.

This has allowed us to identify cell types in the microenvironment that we didnt even know interacted with stem cells, which is opening new research directions, Tamplin says.

As a part of this study, Tamplin, and his colleagues, including co-first authors Sobhika Agarwala and Keunyoung Kim, identified dopamine beta-hydroxylase positive ganglia cells, which were previously an uncharacterized cell type in the blood stem cell niche. This is crucial, as understanding the role of neurotransmitters like dopamine in regulating blood stem cells could lead to improved therapeutics.

Transplanted blood stem cells are used as a curative therapy for many blood diseases and cancers, but blood stem cells are very rare and difficult to locate in a living organism, Tamplin says. That makes it very challenging to characterize them and understand how they interact and connect with neighboring cells.

While blood stem cells are difficult to locate in most living organisms, the zebrafish larva, which is transparent, offers researchers a unique opportunity to view the inner workings of the blood stem cell niche more easily.

Thats the really nice thing about the zebrafish and being able to image the cells, Tamplin says of animals transparent quality. In mammals, blood stem cells develop in utero in the bone marrow, which makes it basically impossible to see those events happening in real time. But, with zebrafish you can actually watch the stem cell arrive through circulation, find the niche, attach to it, and then go in and lodge there.

While the zebrafish larva makes it easier to see blood stem cell development, specialized imaging is needed to find such small cells and then detail their ultrastructure. Tamplin and his colleagues spent over six years perfecting these imaging techniques. This allowed them to see and track the real-time development of a blood stem cell in the microenvironment of a live organism, then zoom in even further on the same cell using electron microscopy.

First, we identified single fluorescently labeledstem cells bylight sheet or confocal microscopy, Tamplin says. Next, we processed the same sample forserial block-face scanningelectron microscopy. We then aligned the 3D light and electron microscopy datasets. Byintersecting these different imaging techniques,we could see the ultrastructure of single rare cells deep inside a tissue. This also allowed us to find all the surrounding niche cellsthat contact a blood stem cell. We believe our approach will be broadly applicable for correlative light and electron microscopy in many systems.

Tamplin hopes that this approach can be used for many other types of stem cells, such as those in the gut, lung, and the tumor microenvironment, where rare cells need to be characterized at nanometer resolution. But, as a developmental biologist, Tamplin is especially excited to see how this work can improve researchers understanding of how the blood stem cell microenvironment forms.

I think this is really exciting because we generate all of our blood stem cells during embryonic development, and depending on what organism you are, a few hundred or maybe a few thousand of these stem cells will end up producing hundreds of billions of new blood cells every day throughout your life, Tamplin says. But we really dont know much about how stem cells first find their home in the niche where theyre going to be for the rest of the life of the organism. This research will really help us to understand how stem cells behave and function. A better understanding of stem cell behavior, and regulation by surrounding niche cells, could lead to improved stem cell-based therapies.

This research was supported by grants from the National Institutes of Health (R01HL142998, K01DK103908, 1U24NS120055-01, R24 GM137200) and the American Heart Association (19POST34380221).

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See-through zebrafish, new imaging method put blood stem cells in high-resolution spotlight - University of Wisconsin-Madison

A bone marrow transplant is a medical procedure that replacesthe bone marrow with healthy cells. Replacement cells might come from either ones own body or from a donor. A stem cell transplant, or more specifically, a hematopoietic stem cell transplant, is another name for a bone marrow transplant. Transplantation can be used to treat leukemia, myeloma, and lymphoma, as well as other blood and immune system illnesses that impact the bone marrow. Cancer and cancer treatment can damage the hematopoietic stem cells. Hematopoietic stem cells are blood-forming stem cells. Hematopoietic stem cells that are damaged may not develop into red blood cells, white blood cells, or platelets. These blood cells are vital, and each one serves a specific purpose. A bone marrow transplant can help the body regenerate the red blood cells, white blood cells, and platelets it requires.

The global Bone Marrow Transplant market is estimated to be valued at $10,356.1 Mn Mn in 2021 and is expected to exhibit a CAGR of 4.0% over the forecast period (2022-2028).

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The study provides data on the most exact revenue estimates for the complete market and its segments to aid industry leaders and new participants in this market. The purpose of this study is to help stakeholders better understand the competitive landscape and design suitable go-to-market strategies. The market size, features, and growth of theBone Marrow Transplantindustry are segmented by type, application, and consumption area in this study. Furthermore, key sections of the GlobalBone Marrow Transplantmarket are evaluated based on their performance, such as cost of production, dispatch, application, volume of usage, and arrangement.

Competitive Analysis: Global Bone Marrow Transplant Market

Detailed Segmentation:

By Type:

By Treatment Type:

:

: United States, Canada, and Mexico & : Argentina, Chile, Brazil and Others & : Saudi Arabia, UAE, Israel, Turkey, Egypt, South Africa & Rest of MEA. : UK, France, Italy, Germany, Spain, BeNeLux, Russia, NORDIC Nations and Rest of Europe. -: India, China, Japan, South Korea, Indonesia, Thailand, Singapore, Australia and Rest of APAC.

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Key Drivers & Barriers:

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Company profiles, revenue sharing, and SWOT analyses of the major players in theBone Marrow TransplantMarket are also included in the research. TheBone Marrow Transplantindustry research offers a thorough examination of the key aspects that are changing, allowing you to stay ahead of the competition. These market measuring methods assist in the identification of market drivers, constraints, weaknesses, opportunities, and threats in the global market.

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Increasing efforts to set up centers for Bone Marrow Transplant is expected to Boost the growth of the market, Top Key players | Lonza, Merck KgaA,...

Cell membrane-coated nanoparticles, applied in targeted drug delivery strategies, combine the intrinsic advantages of synthetic nanoparticles and cell membranes. Although stem cell-based delivery systems were highlighted for their targeting capability in tumor therapy, inappropriate stem cells may promote tumor growth.

Study:Stem cell membrane-camouflaged targeted delivery system in tumor. Image Credit:pinkeyes/Shutterstock.com

A review published in the journalMaterials Today Biosummarized the role of stem cell membrane-camouflaged targeted delivery system in tumor therapy and focused on the underlying mechanisms of stem cell homing toward target tumors. Nanoparticle-coated stem cell membranes have enhanced targetability, biocompatibility, and drug loading capacity.

Furthermore, the clinical applications of induced pluripotent stem cells (iPSCs) and mesenchymal stem cells (MSCs) were investigated as membrane-camouflaged targeted delivery systems for their anti-tumor therapies. In concurrence, the stem cell membrane-coated nanoparticles have immense prospects in tumor therapy.

Cell-based targeted delivery systems have low immunogenicity and toxicity, innate targeting capability, ability to integrate receptors, and long circulation time. Cells such as red blood cells, platelets, stem cells, tumor cells, immune cells, and even viral/bacterial cells can serve as effective natural vesicles.

MSCs derived from the umbilical cord (UC-MSCs), bone marrow (BM-MSCs), and adipose tissue (ATMSCs) are utilized in clinical applications. However, iPSCs are preferable over MSCs in clinical applications due to their easy fetch by transcription factor-based reprogramming of differentiation of somatic cells.

Stem cells (MSCs/ iPSCs) can be easily isolated and used as drug delivery systems for tumor therapy. Stem cell-based delivery systems have inflammation or tumor lesions targeting capacity. However, stem cells are often entrapped in the lung due to their size, resulting in microembolism.

Cell membrane-coated nanoparticles are applied in targeted delivery strategies. To this end, stem cell membrane-coated nanoparticles have tremendous prospects in biomedical applications. Although previous reports mentioned the role of cell membrane-coated nanocarriers in tumor therapy, delivery systems based on stem cell membranes have not been explored extensively.

Stem cell membrane-coated nanoparticles obtained from stem cells have complex functioning and can achieve biological interfacing. Consequently, stem cell membrane-coated nanoparticles served as novel drug delivery systems that could effectively target the tumor.

Previous reports mentioned the preparation of doxorubicin (DOX) loaded, poly (lactic-co-glycolic acid) (PLGA) coated MSC membrane-based nanovesicles, which showed higher cellular uptake than their PLGA uncoated counterparts. Similarly, the DOX-loaded MSC membrane-coated gelatin nanogels showed enhanced storage stability and sustained drug release.

Thus, the stem cell membrane-coated nanoparticles served as novel carriers for stem cells and facilitated the targeted delivery of the drugs at the tumor site. Since the stem cell membrane-coated nanoparticles had good targeting and penetration abilities, they enhanced the efficiency of chemotherapeutic agents in tumor therapy and minimized the side effects.

Reactive oxygen species (ROS) based photodynamic therapy (PDT) is mediated by photosensitizers with laser irradiations. Previous reports mentioned the development of MSC membrane-based mesoporous silica up-conversion ([emailprotected]2) nanoparticles that efficiently targeted the tumor due to their high affinity after being coated with MSC membrane.

These cell membrane-coated nanoparticles showed high cytocompatibility (with hepatocyte cells) and hemocompatibility (with blood). Moreover, the [emailprotected]2 nanoparticles-based PDT therapy under 980-nanometer laser irradiations could inhibit the tumors in vivo and in vitro. Consequently, the stem cell membrane-coated nanoparticles had circulation for an extended time and escaped the immune system, thereby increasing their accumulation at the tumor site.

Stem cell membrane-coated nanoparticles were also applied to deliver small interfering RNA (siRNA) via magnetic hyperthermia therapy and imaging. Previous reports mentioned the preparation of superparamagnetic iron oxide (SPIO) nanoparticles using an MSC membrane that reduced the immune response.

Additionally, the CD44 adhesion receptors were preserved on the surface of the MSC membrane during preparation. These prepared nanovesicles were unrecognized by macrophages, which enabled their stability in blood circulation. The nanosize and tumor homing capacity of MSCs helped the nanovesicles generate a dark contrast in T2-weight magnetic resonance imaging (MRI).

Cell membrane-coated nanoparticles helped fabricate various targeted delivery strategies. Especially, stem cell membrane-coated nanoparticles have the following advantages: stem cells are easy to isolate and expand in vitro. Thus, multilineage potential and phenotypes could be preserved for more than 50 population doublings in vitro.

Stem cell membrane-coated nanoparticles also have an intrinsic capacity to target inflammation or tumor lesions. Hence, these nanoparticles were established for tumor therapy, building a strong foundation for stem cell membrane-mediated delivery systems.

On the other hand, stem cell membrane-coated nanoparticles have the following drawbacks: Despite various sources for collecting MSCs (UC-MSCs/BM-MSCs/ATMSCs), the number of cells obtained is limited, although iPSCs are relatively easy to fetch by reprogramming differentiated somatic cells, the reprogramming is a high-cost step, restricting the clinical applications of iPSCs.

Zhang, W., Huang, X. (2022). Stem cell membrane-camouflaged targeted delivery system in tumor. Materials Today Bio.https://www.sciencedirect.com/science/article/pii/S2590006422001752

Disclaimer: The views expressed here are those of the author expressed in their private capacity and do not necessarily represent the views of AZoM.com Limited T/A AZoNetwork the owner and operator of this website. This disclaimer forms part of the Terms and conditions of use of this website.

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Stem Cell Membrane-Coated Nanoparticles in Tumor Therapy - AZoNano

TCR-CAR+ iPSC-derived CD8 T Cells Induced Complete and Durable Responses In Vivo in Systemic Leukemia Model

Cell-surface Markers, Gene Transcription Profile, and In Vivo Anti-tumor Activity of TCR-CAR+ iPSC-derived CD8 T Cells Compared Favorably with Healthy-donor Peripheral Blood CAR T Cells

Phase 1 Study Ongoing of First-ever iPSC-derived T-cell Product Candidate FT819 for Off-the-shelf Treatment of Patients with Relapsed / Refractory B-cell Malignancies

SAN DIEGO, Aug. 09, 2022 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. ( FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for patients with cancer, today announced the publication of preclinical study results demonstrating the successful generation, durable anti-tumor response, and functional persistence of TCR-CAR+ iPSC-derived CD8 T cells from induced pluripotent stem cells (iPSCs). The CD8 T cells were derived from a single engineered iPSC integrating a novel chimeric antigen receptor (CAR) transgene into the T-cell receptor alpha constant (TRAC) locus, ensuring complete bi-allelic disruption of T-cell receptor (TCR) expression and promoting uniform CAR expression. The discoveries were made under a multi-year research collaboration between the Company and Memorial Sloan Kettering Cancer Center (MSK) led by Michel Sadelain, M.D., Ph.D., Director, Center for Cell Engineering and Head, Gene Expression and Gene Transfer Laboratory, and were published this week in Nature Biomedical Engineering.

Scientists have previously differentiated induced pluripotent stem cells to form CAR T cells, however, it was observed that premature TCR or constitutive CAR expression resulted in the derivation of innate-like T cells that do not acquire the phenotype nor exhibit the function of conventional CD8 T cells, said Dr. Sadelain. Our published findings are the first to show the generation of iPSC-derived CD8 CAR T cells lacking a TCR, where timed and calibrated expression of the CAR in place of the TCR successfully drove T-cell maturation and promoted the acquisition of a transcriptional and functional profile more closely resembling that of natural CD8 T cells.

The mass production of TCR-CAR+ CD8 T cells from master engineered iPSC lines is a promising approach for development of off-the-shelf, cell-based cancer immunotherapies. Through a systematic assessment of factors that affect T-cell lineage commitment and induce adaptive T-cell formation, the researchers discovered that integrating the CAR construct into the TRAC locus delayed its expression and drove T-cell lineage commitment, and that regulation of CAR signaling strength promoted the generation of CD4+CD8+ double-positive cells mimicking thymic development in the absence of a TCR. Subsequent stimulation of the CAR matured the double-positive population into single-positive CD8 T cells with a phenotype highly correlated with peripheral blood CD8 effector T cells and distinct from T cells and natural killer cells. Preclinical studies showed that iPSC-derived TCR-CAR+ CD8 T cells were able to repeatedly lyse tumor cells in vitro and durably control leukemia in vivo, with persistence in the bone marrow, spleen, and blood, in a systemic NALM6 leukemia model.

These published findings continue to support our unique ability to generate TCR-CAR+ CD8 T cells from master engineered iPSC lines that exhibit a phenotypic profile and anti-tumor activity comparable to healthy donor-derived peripheral blood CAR T cells in preclinical model systems, said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. We believe our off-the-shelf, iPSC-derived CAR T cell programs overcome the numerous challenges associated with the manufacture, consistency, and reach of autologous and allogeneic CAR T cells, and we look forward to sharing initial clinical data from our landmark Phase 1 study of FT819 later this year.

The Company is conducting a multicenter Phase 1 study of FT819, the first T-cell therapy manufactured from a clonal master iPSC line to undergo clinical investigation. The product candidates clonal engineered master iPSC line is created from a single iPSC that has a novel CD19-targeted 1XX CAR construct integrated into the TRAC locus, ensuring complete bi-allelic disruption of TCR expression to prevent graft-versus-host disease and promoting uniform CAR expression for enhanced anti-tumor activity. Dose escalation is currently ongoing in single-dose and multi-dose escalation cohorts for relapsed / refractory B-cell malignancies.

Pursuant to a license agreement with MSK, Fate Therapeutics has an exclusive license for all human therapeutic use to U.S. Patent No. 10,370,452, which covers compositions and uses of effector T cells expressing a CAR, where such T cells are derived from a pluripotent stem cell including an iPSC. In addition to the patent rights licensed from MSK, the Company owns an extensive intellectual property portfolio that broadly covers compositions and methods for the genome editing of iPSCs using CRISPR and other nucleases, including the use of CRISPR to insert a CAR in the TRAC locus for endogenous transcriptional control.

Fate Therapeutics has licensed intellectual property from MSK on which Dr. Sadelain is an inventor. As a result of the licensing arrangement, MSK has financial interests related to Fate Therapeutics.

About Fate Therapeutics iPSC Product PlatformThe Companys proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that are designed to be administered with multiple doses to deliver more effective pharmacologic activity, including in combination with other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Companys first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single engineered iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf for patient treatment. As a result, the Companys platform is uniquely designed to overcome numerous limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is subject to batch-to-batch and cell-to-cell variability that can affect clinical safety and efficacy. Fate Therapeutics iPSC product platform is supported by an intellectual property portfolio of over 350 issued patents and 150 pending patent applications.

About FT819FT819 is an investigational, universal, off-the-shelf, T-cell receptor (TCR)-less CD19 chimeric antigen receptor (CAR) T-cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line, which is engineered with the following features designed to improve the safety and efficacy of CAR19 T-cell therapy: a novel 1XX CAR signaling domain, which has been shown to extend T-cell effector function without eliciting exhaustion; integration of the CAR19 transgene directly into the T-cell receptor alpha constant (TRAC) locus, which has been shown to promote uniform CAR19 expression and enhanced T-cell potency; and complete bi-allelic disruption of TCR expression for the prevention of graft-versus-host disease. FT819 demonstrated antigen-specific cytolytic activity in vitro against CD19-expressing leukemia and lymphoma cell lines comparable to that of primary CAR T cells, and persisted and maintained tumor clearance in the bone marrow in an in vivo disseminated xenograft model of lymphoblastic leukemia. FT819 is being investigated in a multicenter Phase 1 clinical trial for the treatment of relapsed / refractory B-cell malignancies, including B-cell lymphoma, chronic lymphocytic leukemia, and acute lymphoblastic leukemia (NCT04629729).

About Fate Therapeutics, Inc.Fate Therapeutics is a clinical-stage biopharmaceutical company dedicated to the development of first-in-class cellular immunotherapies for patients with cancer. The Company has established a leadership position in the clinical development and manufacture of universal, off-the-shelf cell products using its proprietary induced pluripotent stem cell (iPSC) product platform. The Companys immuno-oncology pipeline includes off-the-shelf, iPSC-derived natural killer (NK) cell and T-cell product candidates, which are designed to synergize with well-established cancer therapies, including immune checkpoint inhibitors and monoclonal antibodies, and to target tumor-associated antigens using chimeric antigen receptors (CARs). Fate Therapeutics is headquartered in San Diego, CA. For more information, please visit http://www.fatetherapeutics.com.

Forward-Looking StatementsThis release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 including statements regarding the advancement of and plans related to the Company's product candidates, clinical studies and preclinical research and development programs, the Companys progress, plans and timelines for the manufacture and clinical investigation of its product candidates, the Companys initiation and continuation of enrollment in its clinical trials including additional dose cohorts in ongoing clinical trials of its product candidates, the therapeutic and market potential of the Companys product candidates, and the Companys clinical development strategy, including for its product candidate FT819. These and any other forward-looking statements in this release are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that the Companys product candidates may not demonstrate the requisite safety or efficacy to warrant further development or to achieve regulatory approval, the risk that results observed in prior studies of the Companys product candidates, including preclinical studies and clinical trials, will not be observed in ongoing or future studies involving these product candidates, the risk of a delay or difficulties in the manufacturing of the Companys product candidates or in the initiation and conduct of, or enrollment of patients in, any clinical trials, the risk that the Company may cease or delay preclinical or clinical development of any of its product candidates for a variety of reasons (including requirements that may be imposed by regulatory authorities on the initiation or conduct of clinical trials, changes in the therapeutic, regulatory, or competitive landscape for which the Companys product candidates are being developed, the amount and type of data to be generated or otherwise to support regulatory approval, difficulties or delays in patient enrollment and continuation in the Companys ongoing and planned clinical trials, difficulties in manufacturing or supplying the Companys product candidates for clinical testing, and any adverse events or other negative results that may be observed during preclinical or clinical development), the risk that results observed in preclinical studies of FT819 may not be replicated in ongoing or future clinical trials, and the risk that FT819 may not produce therapeutic benefits or may cause other unanticipated adverse effects. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Companys actual results to differ from those contained in the forward-looking statements, see the risks and uncertainties detailed in the Companys periodic filings with the Securities and Exchange Commission, including but not limited to the Companys most recently filed periodic report, and from time to time in the Companys press releases and other investor communications. Fate Therapeutics is providing the information in this release as of this date and does not undertake any obligation to update any forward-looking statements contained in this release as a result of new information, future events or otherwise.

Contact:Christina TartagliaStern Investor Relations, Inc.212.362.1200[emailprotected]

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Fate Therapeutics Announces Preclinical Publication Highlighting Derivation of CD8 T Cells from TCR-CAR+ Induced Pluripotent Stem Cells -...

HAEMOGLOBIN IS a protein in your red blood cells. Your red blood cells carry oxygen throughout your body. If you have a condition that affects your bodys ability to make red blood cells, your haemoglobin levels may drop. Low haemoglobin levels may be a symptom of several conditions, including different kinds of anaemia and cancer.

If a disease or condition affects your bodys ability to produce red blood cells, your haemoglobin levels may drop. When your haemoglobin level is low, it means your body is not getting enough oxygen, making you feel very tired and weak.

Normal haemoglobin levels are different for men and women. For men, a normal level ranges between 14.0 grams per decilitre (gm/dL) and 17.5 gm/dL. For women, a normal level ranges between 12.3 gm/dL and 15.3 gm/dL. A severe low-haemoglobin level for men is 13.5 gm/dL or lower. For women, a severe low haemoglobin level is 12 gm/dL.

Your doctor diagnoses low haemoglobin by taking samples of your blood and measuring the amount of haemoglobin in it. This is a haemoglobin test. They may also analyse different types of haemoglobin in your red blood cells, or haemoglobin electrophoresis.

Several factors affect haemoglobin levels and the following situations may be among them:

Your body produces red blood cells and white blood cells in your bone marrow. Sometimes, conditions and diseases affect your bone marrows ability to produce or support enough red blood cells.

Your body produces enough red blood cells, but the cells are dying faster than your body can replace them.

You are losing blood from injury or illness. You lose iron any time you lose blood. Sometimes, women have low haemoglobin levels when they have their periods. You may also lose blood if you have internal bleeding, such as a bleeding ulcer.

Your body cannot absorb iron, which affects your bodys ability to develop red blood cells.

You are not getting enough essential nutrients like iron and vitamins B12 and B9.

Your bone marrow produces red blood cells. Diseases, conditions and other factors that affect red blood cell production include:

Lymphoma: This is a term for cancers in your lymphatic system. If you have lymphoma cells in your bone marrow, those cells can crowd out red blood cells, reducing the number of red blood cells.

Leukaemia: This is cancer of your blood and bone marrow. Leukaemia cells in your bone marrow can limit the number of red blood cells your bone marrow produces.

Anaemia: There are many kinds of anaemias involving low-haemoglobin levels. For example, if you have aplastic anaemia, the stem cells in your bone marrow dont create enough blood cells. In pernicious anaemia, an autoimmune disorder keeps your body from absorbing vitamin B12. Without enough B12, your body produces fewer red blood cells.

Multiple Myeloma: This causes your body to develop abnormal plasma cells that may displace red blood cells.

Chronic Kidney Disease: Your kidneys dont produce the hormone that signals to your bone marrow to make red blood cells. Chronic kidney disease affects this process.

Antiretroviral medications: These medications treat certain viruses. Sometimes these medications damage your bone marrow, affecting its ability to make enough red blood cells.

Chemotherapy: Chemotherapy may affect bone marrow cells, reducing the number of red blood cells your bone marrow produces.

Doctors treat low haemoglobin by diagnosing the underlying cause. For example, if your haemoglobin levels are low, your healthcare provider may do tests that reveal you have iron-deficiency anaemia. If that is your situation, they will treat your anaemia with supplements. They may recommend that you try to follow an iron-rich diet. In most cases, treating the underlying cause of anaemia will bring the haemoglobin level up.

Many things can cause low haemoglobin, and most of the time you cannot manage low haemoglobin on your own. But eating a vitamin-rich diet can help maintain your red blood cells. Generally, a balanced diet with a focus on important nutrients is the best way to maintain healthy red blood cells and haemoglobin.

keisha.hill@gleanerjm.comSOURCE: Centres for Disease Control and Prevention

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Factors that affect haemoglobin levels and how to detect when it's low - Jamaica Gleaner

The media continues the one-handed count of patients that seem to be cured of HIV as a man who has lived with the disease since the 1980s has been in remission for 17 months.

The story is always the samethey seem to be cured, and they get a cool nicknamein this case the City of Hope Patient, after Duarte, California, where he was treated.

The difference in this case was the treatmenta bone marrow transplant to treat blood cancer leukemia from a donor who was naturally resistant to the virus.

The most remarkable difference however, is that he is only patient cured of HIV by coincidence.

The man had developed leukemia, and took the bone marrow transplant for that reason. As it happened, the donor was resistant to HIV, and taught the mans body to create an immune response against the virus.

RELATED: Worlds Second Person Cured of HIV: 40-Year-old Man is Confirmed to Be 30 Months Virus-Free

This is also the first one who got it during the epidemic of HIV/AIDS that took so many lives.

When I was diagnosed with HIV in 1988, like many others, I thought it was a death sentence, said the City of Hope Patient. I never thought I would live to see the day that I no longer have HIV.

SIMILAR: Two Patients Make History After Essentially Being Cured of HIV Using Stem Cell Transplant

So far, only three people have been seemingly cured of human immunodeficiency virus (HIV) which weakens the bodys immune system and leads to the more severe AIDS (autoimmune deficiency syndrome) which can be lethal.

The man no longer takes antiretroviral drugs, the only treatment for HIV. A bone marrow transplant is not a likely future cure, do to it being a tricky and side-effectual procedure.

Nevertheless, all cure cases have been those where a patient is given a transplant of some kind, mostly stem cells, that contain the very rarely occurring natural immunity to the virus.

The case was reported at the AIDS 2022 conference in Montreal, Canada.

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Fourth Patient Seemingly Cured of HIV Through Wild Coincidence - Good News Network

Country

United States of AmericaUS Virgin IslandsUnited States Minor Outlying IslandsCanadaMexico, United Mexican StatesBahamas, Commonwealth of theCuba, Republic ofDominican RepublicHaiti, Republic ofJamaicaAfghanistanAlbania, People's Socialist Republic ofAlgeria, People's Democratic Republic ofAmerican SamoaAndorra, Principality ofAngola, Republic ofAnguillaAntarctica (the territory South of 60 deg S)Antigua and BarbudaArgentina, Argentine RepublicArmeniaArubaAustralia, Commonwealth ofAustria, Republic ofAzerbaijan, Republic ofBahrain, Kingdom ofBangladesh, People's Republic ofBarbadosBelarusBelgium, Kingdom ofBelizeBenin, People's Republic ofBermudaBhutan, Kingdom ofBolivia, Republic ofBosnia and HerzegovinaBotswana, Republic ofBouvet Island (Bouvetoya)Brazil, Federative Republic ofBritish Indian Ocean Territory (Chagos Archipelago)British Virgin IslandsBrunei DarussalamBulgaria, People's Republic ofBurkina FasoBurundi, Republic ofCambodia, Kingdom ofCameroon, United Republic ofCape Verde, Republic ofCayman IslandsCentral African RepublicChad, Republic ofChile, Republic ofChina, People's Republic ofChristmas IslandCocos (Keeling) IslandsColombia, Republic ofComoros, Union of theCongo, Democratic Republic ofCongo, People's Republic ofCook IslandsCosta Rica, Republic ofCote D'Ivoire, Ivory Coast, Republic of theCyprus, Republic ofCzech RepublicDenmark, Kingdom ofDjibouti, Republic ofDominica, Commonwealth ofEcuador, Republic ofEgypt, Arab Republic ofEl Salvador, Republic ofEquatorial Guinea, Republic ofEritreaEstoniaEthiopiaFaeroe IslandsFalkland Islands (Malvinas)Fiji, Republic of the Fiji IslandsFinland, Republic ofFrance, French RepublicFrench GuianaFrench PolynesiaFrench Southern TerritoriesGabon, Gabonese RepublicGambia, Republic of theGeorgiaGermanyGhana, Republic ofGibraltarGreece, Hellenic RepublicGreenlandGrenadaGuadaloupeGuamGuatemala, Republic ofGuinea, RevolutionaryPeople's Rep'c ofGuinea-Bissau, Republic ofGuyana, Republic ofHeard and McDonald IslandsHoly See (Vatican City State)Honduras, Republic ofHong Kong, Special Administrative Region of ChinaHrvatska (Croatia)Hungary, Hungarian People's RepublicIceland, Republic ofIndia, Republic ofIndonesia, Republic ofIran, Islamic Republic ofIraq, Republic ofIrelandIsrael, State ofItaly, Italian RepublicJapanJordan, Hashemite Kingdom ofKazakhstan, Republic ofKenya, Republic ofKiribati, Republic ofKorea, Democratic People's Republic ofKorea, Republic ofKuwait, State ofKyrgyz RepublicLao People's Democratic RepublicLatviaLebanon, Lebanese RepublicLesotho, Kingdom ofLiberia, Republic ofLibyan Arab JamahiriyaLiechtenstein, Principality ofLithuaniaLuxembourg, Grand Duchy ofMacao, Special Administrative Region of ChinaMacedonia, the former Yugoslav Republic ofMadagascar, Republic ofMalawi, Republic ofMalaysiaMaldives, Republic ofMali, Republic ofMalta, Republic ofMarshall IslandsMartiniqueMauritania, Islamic Republic ofMauritiusMayotteMicronesia, Federated States ofMoldova, Republic ofMonaco, Principality ofMongolia, Mongolian People's RepublicMontserratMorocco, Kingdom ofMozambique, People's Republic ofMyanmarNamibiaNauru, Republic ofNepal, Kingdom ofNetherlands AntillesNetherlands, Kingdom of theNew CaledoniaNew ZealandNicaragua, Republic ofNiger, Republic of theNigeria, Federal Republic ofNiue, Republic ofNorfolk IslandNorthern Mariana IslandsNorway, Kingdom ofOman, Sultanate ofPakistan, Islamic Republic ofPalauPalestinian Territory, OccupiedPanama, Republic ofPapua New GuineaParaguay, Republic ofPeru, Republic ofPhilippines, Republic of thePitcairn IslandPoland, Polish People's RepublicPortugal, Portuguese RepublicPuerto RicoQatar, State ofReunionRomania, Socialist Republic ofRussian FederationRwanda, Rwandese RepublicSamoa, Independent State ofSan Marino, Republic ofSao Tome and Principe, Democratic Republic ofSaudi Arabia, Kingdom ofSenegal, Republic ofSerbia and MontenegroSeychelles, Republic ofSierra Leone, Republic ofSingapore, Republic ofSlovakia (Slovak Republic)SloveniaSolomon IslandsSomalia, Somali RepublicSouth Africa, Republic ofSouth Georgia and the South Sandwich IslandsSpain, Spanish StateSri Lanka, Democratic Socialist Republic ofSt. HelenaSt. Kitts and NevisSt. LuciaSt. Pierre and MiquelonSt. Vincent and the GrenadinesSudan, Democratic Republic of theSuriname, Republic ofSvalbard & Jan Mayen IslandsSwaziland, Kingdom ofSweden, Kingdom ofSwitzerland, Swiss ConfederationSyrian Arab RepublicTaiwan, Province of ChinaTajikistanTanzania, United Republic ofThailand, Kingdom ofTimor-Leste, Democratic Republic ofTogo, Togolese RepublicTokelau (Tokelau Islands)Tonga, Kingdom ofTrinidad and Tobago, Republic ofTunisia, Republic ofTurkey, Republic ofTurkmenistanTurks and Caicos IslandsTuvaluUganda, Republic ofUkraineUnited Arab EmiratesUnited Kingdom of Great Britain & N. IrelandUruguay, Eastern Republic ofUzbekistanVanuatuVenezuela, Bolivarian Republic ofViet Nam, Socialist Republic ofWallis and Futuna IslandsWestern SaharaYemenZambia, Republic ofZimbabwe

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BUDDY SCOTT: Love stems from the Father | Brazos Living | thefacts.com - Brazosport Facts

MANY people suffer with back pain, whether that's from poor posture or a sporting injury.

Teenager Daniel Greer had been struggling with this - as well as neck pain.

2

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But rather than an injury or pull - a blood test confirmed a shocking diagnosis.

The 14-year-old from Northern Ireland was told he had acute myeloid leukaemia two months ago.

Now his family are racing to find a stem cell donor - as this is his only chance of survival.

Since his diagnosis, the music fanatic has been staying at the Royal Belfast Hospital for Sick Children and is being treated with aggressive chemotherapy.

Doctors say that a stem cell transplant will help repair his immune system - but only one in four people will find a match within their own family.

His older brother James, sadly isn't a match so Daniel will need a transplant from an unrelated donor.

Mum, Anne, is now speaking out in the hopes of getting more people to sign up to the stem cell register - with the possibility of finding her son a donor.

She said: "Daniel is an amazing, bright young man who lights up any room he walks into.

"His wicked sense of humour keeps our spirits up, even now while hes in hospital receiving chemotherapy.

"I know hes really proud that his story is inspiring people to sign up to the stem cell register.

"Those people will potentially help him, as well as many other people around the world who desperately need a stem cell transplant like Daniel."

When it comes to the stem cell register, young men make up just 18 per cent of those on it, blood cancer charity Anthony Nolan states.

However, this demographic also makes up more than half of all stem cell transplants for blood cancer and blood disorder patients.

Now the charity is helping with an international appeal to get Daniel a donor, dubbed the DoItForDaniel campaign.

Daniel, lives in Newry and so far local pharmacies have got behind the campaign - urging people to sign up to help save the lives of others.

What is leukaemia?

Leukaemia is a type of blood cancer that affects cells in bone marrow and attacks the immune system.

In most cases of leukaemia, there is no obvious cause. Little Azaylia had been diagnosed with Acute Myeloid Leukaemia (AML) , which is a rapidly progressive form of the illness.

Leukaemia is a cancer that leads to the body making too many abnormal white blood cells and means the body is less likely to be able to defend itself against infection.

These blood cells are not fully developed and are called leukaemia cells.

The disease is often classified as the type of cell affected (myeloid or lymphatic) and how it progresses (acute or chronic).

There are four main types of leukaemia.

Acute Lymphocytic Leukaemia (ALL)-A rapidly progressing form of the disease.More common in children.

Acute Myeloid Leukaemia (AML) -Rapidly progressive. More common in adults.

Chronic Lymphocytic Leukaemia (CLL) -Slowly progressing form and more common in adults.

Chronic Myeloid Leukaemia (CML) -Progresses slowly and is more common in adults

There has also been an awareness-raising drive about stem cell donation at Belfast International Airport.

It's hoped that the drive will allow the keen mountain biker and rugby player to continue to do the things he enjoys most.

Anthony Nolan chief executive Henny Braund said that finding a matching donor would mean everything to Daniel and his family.

"We are committed to supporting Daniel as he waits for news of the donor who could save his life.

Last year over 1,300 people around the world with blood cancer or a blood disorder were given a second chance of life because of the wonderful people that are signed up to the Anthony Nolan register.

But too many people, like Daniel, are told there is no matching donor for them.

Signing up to the register is quick and simple, and we urge anyone who is in good general health, especially young men aged 16-30, to come forward and potentially save the life of someone like Daniel.

Anyone aged 16-30 can sign up online through the Anthony Nolan website.

DONATING STEM CELLS & SIGNING THE REGISTER

When you join a stem cell registry you are on standby to be matched and potentially save a life although many people are never called up.

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My little boy is fighting for his life after complaining of back pain you could save him... - The Sun