CAR T-Cell Therapy: Genetically Programming the Immune System to Attack Malignant Cells

T cells and B cells are 2 primary cell types in our adaptive immune system, the source of immunological memory protecting us from subsequent pathogen exposure. B cells secrete pathogen-specific antibodies, which neutralize pathogens directly, or tag them for attack by other immune cells. T cells destroy pathogenic cells directly as well as secrete cytokines to attract additional immune cells.

Chimeric antigen receptor (CAR) T cells are patient derived T cells genetically manipulated to express an artificial transmembrane receptor. The artificial receptor is engineered from modular parts to bind to a surface protein (also called an antigen) on malignant cells and activate the T cell via engineered T cell signaling switches on the CAR.

Current FDA-approved CAR-T cell therapies express CARs recognizing CD19, which is expressed on the surface of almost all B cells, making these therapies specific for B-cell malignancies. Following binding with a CD19-expressing cell, the CAR T cell is activated to proliferate, eliminate the CD19-expressing cell, and persist within the patient.

Tisagenlecleucel (Kymriah, Novartis) is approved to treat pediatric and young adult patients (up to age 25) with relapsed or refractory (R/R) B-cell acute lymphoblastic leukemia (R/R ALL, ELIANA trial) and adult patients with R/R diffuse large B-cell lymphoma (R/R DLBCL, JULIET trial) after 2 or more lines of systemic therapy. Axicabtagene ciloleucel (Yescarta, Kite Pharma) is approved for adult patients with R/R large B-cell lymphoma (R/R DLBCL, ZUMA-1 trial, NCT02348216) after 2 or more lines of systemic therapy. There are approximately 700 new cases of pediatric and young adult R/R ALL annually in the United States. New cases of R/R DLBCL are approximately 7000 annually in the United States.

CARs can be engineered to recognize virtually any cell surface antigen and can be expressed in a variety of immune cells, suggesting that product development will result in many modular CAR units with vast application versatility. Many different antigen and cell type combinations are already currently in development to address several cancers, such as CAR T cells for pancreatic cancer (NCT03323944).

The first step in creating these personalized genemodified cell therapies is collecting patient lymphocytes via leukapheresis at a clinic or infusion center. Lymphocytes include T cells, B cells, and natural killer cells. The leukapheresis process lasts up to 4 hours and must be coordinated with the patients continuing care regimen to ensure sufficient T cells. The lymphocytes are cryopreserved and immediately shipped to a centralized manufacturing facility.

T cells are separated from the other cells in the leukapheresis product and genetically manipulated, typically using a lentiviral gene delivery method to carry DNA encoding the CAR protein, resulting in CAR T cells. The CAR T cells are cultured to a patient-specific appropriate dose. As this process is finishing, the manufacturer coordinates with the patients health care team to ensure the patient and team are prepared to infuse the CAR T cells. The manufacturing process from the apheresis process to the clinical CAR-T cell product varies widely from patient to patient, from 14 days up to a few months. The limiting step is typically reaching the appropriate CAR-T cell dose.

About a week before the scheduled CAR-T cell infusion, the patient receives multiple days of low-dose conditioning chemotherapy. This step serves to deplete lymphocytes before administration of the CAR T cells, improving the efficacy and persistence of therapy. The CAR T cells are then administered intravenously, and the patient is monitored for adverse events (AEs). The most common AEs with both currently approved products include cytokine release syndrome (CRS), neurological toxicity (NT), hypersensitivity reactions, serious infection, prolonged cytopenias, and hypogammaglobulinemia.3,4 Both products caution that therapy could cause hepatitis B viral reactivation.

The most severe reactions are CRS and NT, both of which can be life threatening. CRS, a common immune reaction following infusion of monoclonal antibodies and CAR T cells, is characterized by fever, nausea, chills, hypotension, tachycardia, asthenia, headache, rash, and dyspnea.5 Mild cases are easily managed, whereas severe cases require more aggressive and invasive therapy, such as mechanical ventilation and intravenous administration of tocilizumab. NT associated with CAR-T cell therapy is characterized by encephalopathy, headache, aphasia, delirium, insomnia, anxiety, tremor, dizziness, seizures, and peripheral neuropathy.

During the clinical trials of Kymriah and Yescarta, CRS and NT occurred in most patients with more than 10% experiencing severe CRS and more than 20% with severe NT.3,4 Initially, the 2 AEs appeared to be independent, but data are beginning to emerge suggesting a correlation. CRS might be a predictor of neurological events; however, neurological events do not predict CRS.6

Real-world evidence from patients treated with Kymriah, presented at the 2019 Society of Hematologic Oncology annual meeting, reported that slightly more than half experienced either of these conditions and less than 20% had severe cases.7 Two-year followup data regarding Yescarta reported severe CRS cases in 11% of patients and severe NT cases in 32%.8 Due to the high rate of occurrence and severity of CRS and NT, both Yescarta and Kymriah have restricted availability through Risk Evaluation and Mitigation Strategies.3,4 Both medications must be administered at a Risk Evaluation and Mitigation Strategiescertified health care facility with health care providers trained in the management and treatment of CRS and 2 doses of tocilizumab available for each patient before CART cell infusion.

Regardless of the potential for severe adverse events (AEs), the benefit of both Yescarta and Kymriah outweigh the risks, as they are highly effective singleadministration therapies. Despite the aggressive nature of the cancers treated with CAR T-cell therapy, meaningful clinical benefit can be achieved within 1 month. A summary of clinical trial primary response rates as well as 2-year data and published real-world data can be found in Table.

Despite differences between the 2 clinically available products, their safety and efficacy profiles in patients with R/R DLBCL are comparable. Differences between the therapies range from the molecular units comprising the CARs, manufacturing differences, lymphodepletion regimen, number of CAR T cells and volume infused, and whether the infusion and short-term patient monitoring occurs in an inpatient or outpatient setting.

Patient-specific genetically modified cell therapies can present many manufacturing challenges. One stark difference between the available therapies, relevant to the patient and clinician experience, has been the manufacturing time and failure rate. During ZUMA-1, of the 101 patients treated with Yescarta, the median time from leukapheresis to product delivery was 17 days (range, 14-51 days), and a 1% manufacture failure rate was reported.4 The ELIANA trial of Kymriah in patients with R/R ALL reported a 9% manufacture failure rate, whereas the JULIET R/R DLBCL trial reported a failure rate of 6.9%, and of the 106 patients receiving Kymriah, the median manufacture time was 113 days (range, 47-196 days).3

Commercial manufacture of Kymriah for DLBCL has struggled to meet specifications.11 While addressing the production issue, Novartis has initiated a safety study evaluating out-of-specification product (NCT04094311) and a managed-access program (NCT03601442).

Known causes of CAR-T cell therapy failure are T cell exhaustion and antigen escape. T cell exhaustion is characterized by a loss of responsive T cells due to changes in gene expression and can be prevented by immune checkpoint inhibitors PD-1, PD-L1, or CTLA- 4. Antigen escape describes a condition in which some cancer cells do not express the CAR-targeted antigen; therefore, they escape immune activation and survive within the patient. Engineering a secondary CAR to a different antigen, such as CD22 in the case of ALL, increases the likelihood of targeting all malignant cells. Solutions to both of these inhibitory mechanisms are currently under clinical trial investigation.12,13

The therapeutic success of CAR T cells ensures gene-modified immune cell therapy will be refined, optimized, and broadly applied until limits are reached. Many clinical groups are investigating biomarkers associated with severe AEs to provide an additional layer of precision care to the CAR-T cell therapy model.6,14 In addition, clinical trials are underway evaluating combination therapies to enhance the efficacy and improve the safety of CART cell therapy. Early-stage research is evaluating the possibility of off-the-shelf CAR-T cell therapy, not a patient-unique manufactured product, to reduce the time to treatment and achieve manufacturing efficiencies and consistencies.15,16 Gene-modified cell therapy, such as CAR T-cell therapy, is revolutionizing oncology, and this living drug model is breathing life into the hopes of patients with cancer and caregivers.

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CAR T-Cell Therapy: Genetically Programming the Immune System to Attack Malignant Cells - Pharmacy Times

IPS Cell Therapy Comments Off on CAR T-Cell Therapy: Genetically Programming the Immune System to Attack Malignant Cells – Pharmacy Times | February 25th, 2020

Medical ResearchThe belief that acute stress can turn hair gray is a popular one, but until now it hasnt been scientifically proven.

But findings that appeared in the publication Nature indicate that the belief may be more than a myth. The study, which used mice as models, was funded in part by the National Institutes of Healths National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and other NIH components.

Hair color is determined by cells called melanocytes, which produce the pigment melanin. New melanocytes are made from melanocyte stem cells that live within the hair follicle at the base of the hair strand. As we age, these stem cells gradually disappear. The hair that regrows from hair follicles that have lost melanocyte stem cells has less pigment and appears gray.

A research team, led by Dr. Ya-Chieh Hsu of Harvard University, used mice to examine stress and hair graying. The mice were exposed to three types of stress involving mild, short-term pain, psychological stress, and restricted movement. All caused noticeable loss of melanocyte stem cells and hair graying.

Having established a link between stress and graying, the scientists then explored several potential causes, including the role of the stress hormone corticosterone, but altering its levels didnt affect stress-related graying.

The researchers eventually turned to the neurotransmitter noradrenaline, which, along with corticosterone, was elevated in the stressed mice. They found that noradrenaline, also known as norepinephrine, was key to stress-induced hair graying. By injecting noradrenaline under the skin of unstressed mice, the researchers were able to cause melanocyte stem cell loss and hair graying.

Noradrenaline is produced mostly by the adrenal glands. However, mice without adrenal glands still showed stress-related graying. Noradrenaline is also the main neurotransmitter of the sympathetic nervous system, which is responsible for the fight-or-flight reaction in response to stress.

Ultimately, the team discovered that signaling from the sympathetic nervous system plays a critical role in stress-induced graying. Sympathetic nerves extend into each hair follicle and release noradrenaline in response to stress. Normally, the melanocyte stem cells in the follicle are dormant until a new hair is grown. Noradrenaline causes the stem cells to activate.

Using fluorescent labelling, the researchers observed the stem cells change to melanocytes and migrate away from their reserve in the hair follicle. With no remaining stem cells, no new pigment cells can be made, and any new hair becomes gray, then white.

When we started to study this, I expected that stress was bad for the body but the detrimental impact of stress that we discovered was beyond what I imagined, Hsu says. After just a few days, all of the melanocyte stem cells were lost. Once theyre gone, you cant regenerate pigments anymore. The damage is permanent.

The authors highlight the need to further study the interactions between the nervous system and stem cells in different tissues and organs. A news release from the NIH said that the knowledge gained in this work will be useful in future investigations into the impact of stress on the body and the development of new interventions.

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Stress and Gray Hair - ThirdAge

Skin Stem Cells Comments Off on Stress and Gray Hair – ThirdAge | February 24th, 2020

Patient Analyses and Safety Data Continue to Underscore Positive Impact of KD025 in cGVHD

Pre-NDA Meeting with FDA Planned for March 2020; Topline Results of Primary Analysis to be Announced in Q2 2020

NEW YORK, NY / ACCESSWIRE / February 23, 2020 / Kadmon Holdings, Inc. (NYSE:KDMN) today announced expanded results from the previously reported interim analysis of ROCKstar (KD025-213), its ongoing pivotal trial of KD025 in chronic graft-versus-host disease (cGVHD). The data were presented today in the oral latebreaker session at the 2020 Transplantation & Cellular Therapy (TCT) Meetings.

As announced in November 2019, KD025 met the primary endpoint of Overall Response Rate (ORR) at the study's planned interim analysis, two months after completion of enrollment. KD025 showed statistically significant and clinically meaningful ORRs of 64% with KD025 200 mg once daily (95% Confidence Interval (CI): 51%, 75%; p<0.0001) and 67% with KD025 200 mg twice daily (95% CI: 54%, 78%; p<0.0001). In the expanded KD025-213 dataset presented today, ORRs were consistent with the previously reported interim analysis across key subgroups, including in patients with four or more organs affected by cGVHD (n=69; 64%), patients who had prior treatment with ibrutinib (n=45; 62%) and patients who had prior treatment with ruxolitinib (n=37; 62%). Three patients achieved a Complete Response. Responses were observed in all affected organ systems, including in organs with fibrotic disease. KD025 has been well tolerated: adverse events were consistent overall with those expected to be observed in cGVHD patients receiving corticosteroids, and no apparent increased risk of infection was observed. Additional secondary endpoints, including duration of response, corticosteroid dose reductions, Failure-Free Survival, Overall Survival and Lee Symptom Scale reductions continue to mature and will be available later in 2020.

"KD025 has been well tolerated and has already demonstrated high response rates in patients with severe and complex cGVHD after a median of five months of follow-up," said Corey Cutler, MD, MPH, FRCPC, Associate Professor of Medicine, Harvard Medical School; Medical Director, Adult Stem Cell Transplantation Program, Dana-Farber Cancer Institute and a KD025-213 study investigator and Steering Committee member.

"We are extremely pleased with the interim outcomes of this pivotal trial of KD025 in cGVHD, which track closely our findings from our earlier Phase 2 study. KD025 achieved robust response rates across all subgroups of this difficult-to-treat patient population, who had a median of four prior lines of therapy, and 73% of whom had no response to their last line of treatment," said Harlan W. Waksal, M.D., President and CEO of Kadmon. "We plan to meet with the FDA for a pre-NDA meeting in March 2020 and to announce topline results from the primary analysis of this trial in Q2 2020."

At the TCT Meetings, Kadmon also presented long-term follow-up data from KD025-208, its ongoing Phase 2 study of KD025 in cGVHD (Abstract #15205). These data were recently presented at the 61st American Society of Hematology (ASH) Annual Meeting and Exposition in December 2019.

About the ROCKstar (KD025-213) Trial

KD025-213 is an ongoing open-label trial of KD025 in adults and adolescents with cGVHD who have received at least two prior lines of systemic therapy. Patients were randomized to receive KD025 200 mg once daily or KD025 200 mg twice daily, enrolling 66 patients per arm. Statistical significance is achieved if the lower bound of the 95% CI of ORR exceeds 30%.

While the ORR endpoint was met at the interim analysis, which was conducted as scheduled two months after completion of enrollment, topline data from the primary analysis of the KD025-213 study, six months after completion of enrollment, will be reported in Q2 2020. Full data from the primary analysis will be submitted for presentation at an upcoming scientific meeting.

About KD025

KD025 is a selective oral inhibitor of Rho-associated coiled-coil kinase 2 (ROCK2), a signaling pathway that modulates immune response as well as fibrotic pathways. In addition to cGVHD, KD025 is being studied in an ongoing Phase 2 clinical trial in adults with diffuse cutaneous systemic sclerosis (KD025-209). KD025 was granted Breakthrough Therapy Designation and Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of patients with cGVHD who have received at least two prior lines of systemic therapy.

About cGVHD

cGVHD is a common and often fatal complication following hematopoietic stem cell transplantation. In cGVHD, transplanted immune cells (graft) attack the patient's cells (host), leading to inflammation and fibrosis in multiple tissues, including skin, mouth, eye, joints, liver, lung, esophagus and gastrointestinal tract. Approximately 14,000 patients in the United States are currently living with cGVHD, and approximately 5,000 new patients are diagnosed with cGVHD per year.

About Kadmon

Kadmon is a clinical-stage biopharmaceutical company that discovers, develops and delivers transformative therapies for unmet medical needs. Our clinical pipeline includes treatments for immune and fibrotic diseases as well as immuno-oncology therapies.

Forward Looking Statements

This press release contains forward-looking statements. Such statements may be preceded by the words "may," "will," "should," "expects," "plans," "anticipates," "could," "intends," "targets," "projects," "contemplates," "believes," "estimates," "predicts," "potential" or "continue" or the negative of these terms or other similar expressions. Forward-looking statements involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. We believe that these factors include, but are not limited to, (i) the initiation, timing, progress and results of our preclinical studies and clinical trials, and our research and development programs; (ii) our ability to advance product candidates into, and successfully complete, clinical trials; (iii) our reliance on the success of our product candidates; (iv) the timing or likelihood of regulatory filings and approvals; (v) our ability to expand our sales and marketing capabilities; (vi) the commercialization of our product candidates, if approved; (vii) the pricing and reimbursement of our product candidates, if approved; (viii) the implementation of our business model, strategic plans for our business, product candidates and technology; (ix) the scope of protection we are able to establish and maintain for intellectual property rights covering our product candidates and technology; (x) our ability to operate our business without infringing the intellectual property rights and proprietary technology of third parties; (xi) costs associated with defending intellectual property infringement, product liability and other claims; (xii) regulatory developments in the United States, Europe, China, Japan and other jurisdictions; (xiii) estimates of our expenses, future revenues, capital requirements and our needs for additional financing; (xiv) the potential benefits of strategic collaboration agreements and our ability to enter into strategic arrangements; (xv) our ability to maintain and establish collaborations or obtain additional grant funding; (xvi) the rate and degree of market acceptance of our product candidates; (xvii) developments relating to our competitors and our industry, including competing therapies; (xviii) our ability to effectively manage our anticipated growth; (xix) our ability to attract and retain qualified employees and key personnel (xx) the potential benefits from any of our product candidates being granted orphan drug or breakthrough designation; (xxi) the future trading price of the shares of our common stock and impact of securities analysts' reports on these prices; and/or (xxii) other risks and uncertainties. More detailed information about Kadmon and the risk factors that may affect the realization of forward-looking statements is set forth in the Company's filings with the U.S. Securities and Exchange Commission (the "SEC"), including the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 2018 and subsequent Quarterly Reports on Form 10-Q. Investors and security holders are urged to read these documents free of charge on the SEC's website at http://www.sec.gov. The Company assumes no obligation to publicly update or revise its forward-looking statements as a result of new information, future events or otherwise.

Contact Information

Ellen Cavaleri, Investor Relations646.490.2989ellen.cavaleri@kadmon.com

SOURCE: Kadmon Holdings, Inc.

View source version on accesswire.com: https://www.accesswire.com/577466/Kadmon-Announces-Expanded-Results-of-Interim-Analysis-of-Pivotal-Trial-of-KD025-in-cGVHD

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Kadmon Announces Expanded Results of Interim Analysis of Pivotal Trial of KD025 in cGVHD - Benzinga

Skin Stem Cells Comments Off on Kadmon Announces Expanded Results of Interim Analysis of Pivotal Trial of KD025 in cGVHD – Benzinga | February 24th, 2020

Stress may play a key role in just how quickly hair goes from colored to ashen.

Scientists have long understood some link is possible between stress and grey hair.

But this new research more deeply probes the exact mechanisms at play.

The researchers initial tests looked closely at cortisol, the stress hormone that surges in the body when a person experiences a fight or flight response.

Its an important bodily function, but the long-term presence of heightened cortisol links to a host of negative health outcomes.

But the culprit ended up being a different part of the bodys fight or flight response the sympathetic nervous system.

These nerves are all over the body, including making inroads to each hair follicle, the researchers reported.

Chemicals released during the stress response causes pigment producing stem cells to activate prematurely, depleting the hairs reserves of color.

The detrimental impact of stress that we discovered was beyond what I imagined, a lead study author said.

After just a few days, all of the pigment-regenerating stem cells were lost.

Once theyre gone, you cant regenerate pigments anymore. The damage is permanent.

But stress isnt the only reason that most people get grey hair.

In most cases, its simple genetics.

Gray hair caused by loss of melanocytes (pigment cells) in the hair follicle.

This happens as we age and, unfortunately, there is no treatment that can restore these cells and the pigment they produce, melanin, a dermatologist told.

Genetic factors determine when you go grey.

There is nothing that can be done medically to prevent this from happening when it is genetically predetermined to happen.

That doesnt mean environmental factors such as stress dont play a role.

Smoking, for instance, is a known risk factor for premature graying.

So kick the habit if you want to keep that color a little longer.

Other contributing factors to premature graying include deficiencies in protein, vitamin B-12, copper, and iron as well as aging due in part to an accumulation of oxidative stress.

That stress prompted by an imbalance between free radicals and antioxidants in your body that can damage tissue, proteins, and DNA.

And some degree of oxidative stress is a natural part of life.

Changes you can pursue to delay premature grays include eating a diet high in omega-3 fatty acids such as walnuts and fatty fish.

It doesnt spend too much time in the skin-damaging and hair-damaging ultraviolet light of the sun, and taking vitamin B-12 and vitamin B-6 supplements.

That said, if you are going gray prematurely, it wouldnt hurt to go have a checkup just in case natural genetic factors arent the sole culprit.

Link:
Stress could be a major cause of grey hair - BOL News

Skin Stem Cells Comments Off on Stress could be a major cause of grey hair – BOL News | February 24th, 2020

A new proof-of-concept study has found a combination of two drugs, already approved by the FDA for other uses, may boost the release of stem cells from bone marrow and accelerate the healing of broken bones. Only demonstrated in animals at this stage, the researchers suggest clinical trials could progress rapidly considering the drugs have already been demonstrated as safe in humans.

"The body repairs itself all the time, says corresponding author on the study Sara Rankin. We know that when bones break they will heal, and this requires the activation of stem cells in the bone. However, when the damage is severe, there are limits to what the body can do of its own accord.

A great deal of current research is focusing on mesenchymal stem cell (MSC) therapies. MSCs are a type of adult stem cell that can grow into a variety of different cell types including muscle, fat or bone. Many current MSC treatments in development involve extracting a small number from a patient, growing them in laboratory conditions, then injecting them back into the patient.

The new research set out to investigate whether any currently approved drugs can function to mobilize the bodys natural ability in releasing MSCs, with a view on speeding up healing of bone fractures. A study published in the journal npj Regenerative Medicine, describes the testing of two already approved drugs in a rodent spinal injury model.

The two drugs tested were an immunostimulant called Plerixafor, used to stimulate the release of stem cells from bone marrow in cancer patients, and a beta-3 adrenergic agonist developed to help bladder control.

The results suggest the duo of drugs mobilize MSCs into the bloodstream and speed up the process of bone formation and healing by enhancing the binding of calcium to the injury site. Tariq Fellous, first author on the new study, suggests the next step is to investigate whether this drug combination enhances blood MSC levels in human subjects.

We first need to see if these medications release the stem cells in healthy volunteers, before we can then test them in patients with fractures, says Fellous. We have the drugs and know they are safe to use in humans - we just need the funding for the human trials.

The researchers say prior studies have identified circulating MSCs increase in volume following injuries such as burns, bone fractures, and even heart attack. The hypothesis is that the release of MSCs is a physiological process aiding general regeneration following injury, and if circulating numbers of MSCs could be pharmacologically enhanced then a variety of types of tissue regeneration could be accelerated.

It is important to note the current study only examined increases in circulating MSCs and the rate of spine injury healing compared to no drug treatment. The current research offers no indication whether the drug duo influences nerve healing or restores movement.

So, more work is certainly necessary to understand how clinically useful these results actually are. However, as the studys co-first author Andia Redpath notes, this re-purposing of existing medicines to boost stem cell activity is an easier, cheaper, and more efficient way to enhance healing compared to other, more complex and time-consuming, stem cell treatments in development.

Rather than devising new stem cell treatments from scratch that involve lengthy and expensive trials, our approach harnesses the power of the bodys own stem cells, using existing drugs, says Redpath. We already know the treatments in our study are safe, its now just a matter of exploring further if they help our bodies heal.

The new study was published in the journal npj Regenerative Medicine.

Source: Imperial College London

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Experimental study speeds up bone healing with 2 common medications - New Atlas

Bone Marrow Stem Cells Comments Off on Experimental study speeds up bone healing with 2 common medications – New Atlas | February 24th, 2020

A combination of two commonly available drugs may help the body heal spinal fractures.

Scientists have discovered a combination of two commonly available drugs that could help the body heal spinal fractures.

The early-stage research in rats, by a group of scientists led by Imperial College London, revealed two existing medications can boost the bodys own repair machinery, by triggering the release of stem cells from the bone marrow.

The scientists published their research in the journalnpj Regenerative Medicine.

The team says the two drugs (currently used for bone marrow transplants and bladder control) could be used for different types of bone fractures, including to the spine, hip, and leg, to aid healing after surgery or fractures.

When a person has a disease or an injury, the bone marrow (the spongy tissue within bone) mobilizes different types of stem cells to help repair and regenerate tissue.

This image shows repair of the spine three weeks after surgery. The image on the left is with no drug treatment, with the image on the right (b3+AMD3100) showing the effect of the two drug treatments. The red colour indicates calcium incorporating into the bone, which is associated with enhanced healing. Credit: Imperial College London/Beaumont Health

The new research, involving scientists from Beaumont Health in the U.S, suggests it may be possible to boost the bodys ability to repair itself and speed repair, by using new drug combinations to put the bone marrow into a state of red alert and send specific kinds of stem cells into action.

In the new study, funded by Wellcome, the researchers used drugs to trigger the bone marrow of healthy rats to release mesenchymal stem cells, a type of adult stem cell that can turn into bone, and help repair bone fractures.

Professor Sara Rankin, corresponding author of the study from the National Heart and Lung Institute at Imperial College London, said: The body repairs itself all the time. We know that when bones break they will heal, and this requires the activation of stem cells in the bone. However, when the damage is severe, there are limits to what the body can do of its own accord. We hope that by using these existing medications to mobilize stem cells, as we were able to do in rats in our new study, we could potentially call up extra numbers of these stem cells, in order to boost our bodies own ability to mend itself and accelerate the repair process. Further down the line, our work could lead to new treatments to repair all types of bone fracture.

The two treatments used in the research were a CXCR4 antagonist, used for bone marrow transplants, and a beta-3 adrenergic agonist, that is used for bladder control.

The rats were given a single treatment with the two drugs, which triggered enhanced binding of calcium to the site of bone injury, speeding bone formation and healing.

The researchers stress they did not analyze restoration of movement in the bone, or repair to additional tissue such as nerves.

One of the drugs used in the study was found to trigger fat cells in the bone marrow to release endocannabinoids, which suggests they may have a role in mobilizing the stem cells and thereby promoting healing. However, the researchers add that phytocannabinoids (such as cannabis) would not have the same effect, as they act on the brain rather than the bone marrow.

The researchers say the drug combinations now need to be tested in humans.

Dr. Tariq Fellous, first author of the research from Imperials National Heart and Lung Institute (NHLI) said: We first need to see if these medications release the stem cells in healthy volunteers, before we can then test them in patients with fractures. We have the drugs and know they are safe to use in humans we just need the funding for the human trials.

Dr. Andia Redpath, co-first author from the NHLI, added that repurposing existing medications that help the body heal itself so called Regenerative Pharmacology could have great potential as an efficient and cost-effective approach for a range of diseases. Rather than devising new stem cell treatments from scratch that involve lengthy and expensive trials, our approach harnesses the power of the bodys own stem cells, using existing drugs. We already know the treatments in our study are safe, its now just a matter of exploring further if they help our bodies heal.

This work was funded by Wellcome. Further support was provided by the National Heart and Lung Institute Foundation and the Lumbar Spine Research Society.

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New hope for the healing of spinal column injuries with generally available drugs - The Washington Newsday

Bone Marrow Stem Cells Comments Off on New hope for the healing of spinal column injuries with generally available drugs – The Washington Newsday | February 24th, 2020

DEFINITIONLeukemia is a Cancer of blood forming tissues including bone marrow and the lymphatic system.This type of cancer hinders the body's ability to fight infection, leukemia involves the white blood cells.

CAUSES OF LEUKEMIALeukemia can develop due to a problem with blood cell production.

Several factors have been identified which increase the risk of having the cancer:

A family history of leukemiaSmokingGenetic disorder such as Down syndromeExposure to chemicals such as benzeneExposure to high levels of radiationTYPES OF LEUKEMIALeukemia can be Acute or Chronic. In Acute leukemia, Cancer cell multiply quickly while in Chronic leukemia, the disease progresses slowly and early symptoms may be very mild.

Leukemia can also be classified according to types of cells which are myelogenous leukemia and lymphotic leukemia involving myeloid cells and lymphocytes respectively.

SYMPTOMS OF LEUKEMIAExcessive sweating most especially in the nightFatigue and weaknessUnintentional weight lossFever or chillsFrequent infectionsBleeding easily and bruising easilyEnlargement of the liver or spleenLEUKEMIA IN PREGNANCYLeukemia affects approximately 1 in 10000 pregnancies.Women with leukemia have non-specific symptoms and some of them could also be attributed to pregnancy.

The damage in the fetus is correlated with the time of exposition and the fetus is most vulnerable during organnogenesis phase, Although chemotherapy has effect in the fetus, there are reports of cases with successful pregnancy.

It is necessary to study the relation among chemotherapy, the leukemia and the fetus in long term studies where fetus has been exposed to chemotherapy agents and is reported with normal characteristics at birth.

ENZYMES DEFICIENT IN LEUKEMIABlast cells from 100 cases of Acute leukemia were evaluated for the presence of methylthioadenosine phosphorylase (MTAase), an enzyme important in polyamine metabolism.

Ten cases (10%) had undetectable levels of MTAase activity. A relatively high frequency (38%) of MTAase deficiency was seen in all of T-cell origin.

MTAase deficiency occurs in a wide variety of acute leukemia, that the lack of enzyme activity is specific in malignant cells. The absence of MTAase in some leukemia may be therapeutically exploitable.

TREATMENT OF LEUKEMIALeukemia is usually treated by a hematologist-oncologist. These are doctors who specialize in blood disorders and cancer.

The treatment depends on the type and stage of the cancer, the treatment includes the following:

Chemotherapy uses drugs to kill leukemia cells.Radiation therapy uses high energy radiation to damage leukemia cells and inhibit their growth.

Stem cell transplantation replaces diseased bone marrow with healthy bone marrow.

Biological or immune therapy uses treatments that help your immune system recognize and attack cancer cells.

Targeted therapy uses medication advantages of vulnerabilities in cancer cells.

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Leukemia: Cancer Of The Blood - Modern Ghana

Bone Marrow Stem Cells Comments Off on Leukemia: Cancer Of The Blood – Modern Ghana | February 24th, 2020

FLORENCE, Ala. (AP) Cole Kelley grinned widely and waved Saturday (Feb. 15) as his head kept swiveling from one direction to the other to take in the amazing view surrounding him.

Hundreds of family members, friends, Mars Hill School classmates and other members of the community lined both sides of the school's parking lot to welcome the 8-year-old home.

The Mars Hill student and his parents, John and Caroline Kelley, had been away from home for 15 months while he has battled a rare disease.

That included a 460-day stay at the National Institutes of Health facility in Bethesda, Maryland, which is one of the few facilities with experience treating the disease dada2, which is a deficiency of the adenosine deaminase 2 (ADA2) enzyme.

According to dada2.org, the disease causes "recurrent strokes, severe systemic inflammation, immune deficiency, and damage to many of the body's tissues and organs."

The community organized the homecoming parade, which included an escort from Florence's police and fire departments, as well as people dressed in superhero outfits.

Along with Cole's classmates, Mars Hill High School cheerleaders joined the crowd, as did the school's football players, who showed up in their jerseys. The long reception line spanned the length of the parking lot coming off Cox Creek Parkway.

The procession made two laps through the parking lot amid loud cheers and flowing tears from the congregation before the family, clearly emotional from the outpouring, drove home.

"It's been amazing," Cole's cousin, Sara Beth Searcy, said while wearing a "Best Day Ever" shirt she bought during a Disney World trip Cole and the family took before his long hospital stay.

"This whole community has supported us from the very beginning," she said. "We could not have gotten through this without this entire community wrapping their arms around us."

Cole classmate Rogan Willingham held a sign that read "God answered our prayers."

"He's prayed, I don't know how many times a day, for Cole," his mother, Ginger Willingham, said.

"I'm glad he's back," Rogan said, adding he looks forward to being able to "play with him and all kinds of stuff."

"I was praying for him a lot," he said.

There were numerous other signs with messages, such as "Sweet Home, Alabama," "Super Cole" and "Welcome home, Buddy."

Emily Stutts, a friend of the family who helped organized the welcome, and fellow member of Jackson Heights Church of Christ, said Cole has been sick since he was 2 months old.

He had a bone transplant at the National Institutes.

"He lived off of having blood transfusions because his body did not make red blood cells," Stutts said. "After a while that became risky because his body can't filter out all the iron. So a bone marrow transplant was the only answer."

He was enrolled in the "Be the Match" bone marrow registry and was a match with a young man from Australia, who had volunteered to join the registry.

"He was able to give the bone marrow and they flew it over to Maryland and did the transplant," Stutts said. "It didn't take initially, and Cole has had three stem-cell transplants, all from the one donation from the Australian man.

"The fact that he's coming home is a miracle, because he had some very, very scary times that they didn't think he would survive. He had zero immune until Christmas day. That day his immune response occurred."

She said John and Caroline often talk about the amazing support they have been receiving back home. A community spaghetti supper, T-shirt sales and a Christmas fundraising event are among methods supporters have used to provide financial assistance for the parents, who have not been able to work during their son's hospitalization.

"The people at the hospital there can't believe the support that they've been given from this community," Stutts said. "They told them they see people from all over the world, but they've never seen the outpouring of love like they saw for the Kelleys.

"They deserve it. They're such good people."

Florence company High Cotton Homes provided assistance in a major way, Stutts said.

"They had to have all new heating and air units and vents put in and a water purification system, all new flooring and all new duct work because of his immune system," she said. "High Cotton took that on, got donations and completely remodeled his room and made it so Cole would have his own bathroom to help with germ prevention."

Cole still is susceptible to sickness and will continue to have medical appointments, Stutts said.

"His liver was damaged from the transplant so he's going to go to Vanderbilt for them to check his liver," she said. "There's still things that need to be addressed, but as far as his bone marrow, that is doing OK."

Stutts said Cole's disease has not kept him down.

"He is full of life," she said. "He's a happy kid. Even though he's been sick, if you had seen him, you would have never know it. He's made a huge impact on the people at the National Institutes for Health because no child's been there that long. That became his family. They had a going-away party at one of the doctor's homes."

The emotions of Saturday's homecoming were obvious among the crowd, many of whom hugged one another and cried.

"The one thing I kept hearing from everybody is it was more emotional than they through it would be," said Ronnie Pannell, family minister at Jackson Heights. It's been a big show of love. Mars Hill loves Cole Kelley, there's no doubt about that.

Link:
Community give child a hero's homecoming - Thehour.com

Bone Marrow Stem Cells Comments Off on Community give child a hero’s homecoming – Thehour.com | February 24th, 2020

Hematology is the branch of medicine which deals with the study, diagnosis, and treatment of blood-related disorders. It diagnoses issues related to white blood cells, red blood cells, platelets, bone marrow, and lymph nodes. Hematology also deals with the liquid portion of blood known as plasma. Some blood-associated diseases are anemia, leukemia, myelofibrosis, blood transfusion, malignant lymphomas, and bone marrow stem cell transplantation. Hematology analyzers, coagulation analyzers, flow cytometers, and slide strainers are some of the hematologic instruments used to analyze blood and blood-related disorders. Hematology analyzers are highly specialized instruments used to count the number of white blood cells, red blood cells, and platelets.

Hematology analyzers are made up of multiple analytical modules with unique sample preparation processes. They assist in the diagnosis of various diseases, such as infections, anemia, viruses, diabetes, genetic problems, and cancer. It also regulates plasma drug level for both therapeutic and illicit drugs. Fully automated hematology analyzers and semi-automated analyzers are the two types of hematology analyzer available based on the type of automation. Fatigue, pale skin, and shortness of breath with exertion are some of the common symptoms of blood disorders. Clinical testing laboratories, hospitals, and research institutions are some of the users of hematology analyzers and reagents.

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Currently, North America dominates the global hematology analyzer & reagent market, followed by Europe. This is due to the regions high disposable income and increasing adoption of automated hematology instruments by diagnostics laboratories in these regions. Asia is expected to be the fastest growing market in the next five years. This is due to the developing healthcare infrastructure and increasing funding towards the development of hematology products in this region. Moreover, rising awareness about better healthcare and a large patient population base are also driving the growth of the hematology analyzer & reagent market in Asia.

Increasing adoption of automated hematology instruments, rising technological advancements, and integration of basic flow-cytometry techniques in modern hematology analyzers are some of the key factors driving the growth of the global hematology analyzer & reagent market. In addition, rising demand for high-throughput hematology analyzers and development of high-sensitivity point-of-care (POC) hematology testing are also fuelling the growth of the global hematology analyzer & reagent market. However, the high cost of hematology analyzers and intense competition among existing players are restraining the growth of the global hematology analyzers & reagent market. In addition, stringent and time-consuming regulatory policies for hematology instruments also impede the market growth.

Usage of microfluidics technology in hematology analyzers and introduction of digital imaging system in hematology laboratories could open up opportunities for new players in the global hematology analyzer & reagent market. In addition, increasing focus toward emerging markets, such as India and China, could also open up opportunities for new players in the global hematology analyzer & reagent market. Moreover, safety and quality of hematology analyzer could be a challenge for the growth of the global hematology analyzer & reagent market.

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Increasing instances of partnership among hematology instruments and consumables manufacturers is one of the recent trends in the global hematology analyzer & reagent market. The major companies operating in this market are

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Adoption of Hematology Analyzer Reagent Market to Soar Across Top Countries in the Globe Forecast to 2015 to 2021 - Lake Shore Gazette

Bone Marrow Stem Cells Comments Off on Adoption of Hematology Analyzer Reagent Market to Soar Across Top Countries in the Globe Forecast to 2015 to 2021 – Lake Shore Gazette | February 24th, 2020

Whenever Harry Wuest has a doctors appointment in northern Atlantas hospital cluster dubbed Pill Hill, he makes sure to stop by the office of Dr. Douglas Doug Murphy for a quick chat.

And Murphy, unless hes tied up in the operating room, always takes a few minutes to say hello to his former patient. Remember when ... ? is how the conversation typically starts, and its always tinged with laughter, often joyful, sometimes bittersweet.

Its a reunion of two men who shaped a piece of Georgias medical history.

Almost 35 years ago, Murphy opened the chest of Wuest and sewed in a new heart, giving him a second shot at life. Wuest was the third heart transplant patient at Emory University Hospital.

Tall, lanky, with short curly hair and a quiet demeanor, Wuest is the longest-surviving heart transplant recipient in Georgia and one of the longest-surviving in the world. The 75-year-old accountant still plays golf twice a week and only recently went from working full-time to part-time. My heart is doing just fine, he says.

Murphy is now the chief of cardiothoracic surgery at Emory Saint Josephs Hospital and still in the operating room almost every day. He has moved on to become the worlds leading expert in robotically assisted heart surgery.

Harry Wuest is originally from Long Island, New York. After a stint in the Air Force, he moved to Florida to work and go to school. He wanted to become a physical education teacher. Then, in 1973, he fell ill. It started with some pain on his left side. He didnt think much of it, but when he got increasingly winded and fatigued, he went to see a doctor.

Several months and numerous specialists later, he received the diagnosis: Cardiomyopathy, a disease of the heart muscle that can make the heart become enlarged, thick and rigid, preventing it from pumping enough blood through the body.

They didnt know how I got it, says Wuest, sitting back in a brown leather armchair in the dark, wood-paneled living room of his Stone Mountain home. Maybe it was a virus. And back then, there wasnt much they could do to treat it, except bed rest.

For the next 12 years, Wuest lived life as best as he could. He got a degree in accounting from the University of Central Florida and worked for a real estate developer. There were good days, but there were more bad days. He was often too weak to do anything, and his heart was getting bigger and bigger.

Emorys first transplant surgeon

The first successful human-to-human heart transplant was performed in Cape Town, South Africa, in 1967 a medical breakthrough that catapulted the surgeon, Dr. Christiaan Barnard, onto the cover of Life magazine and to overnight celebrity status.

This highly publicized event was followed by a brief surge in the procedure around the world, but overall, heart transplants had a rocky start. Most patients died shortly after the surgery, mainly due to organ rejection. Back then, immunosuppressive drugs, which can counteract rejection, were still in their infancy. Many hospitals stopped doing heart transplants in the 1970s.

That changed with the discovery of a highly effective immunosuppressive agent. Cyclosporine got FDA approval in 1983 and altered the world of organ transplants.

It was shortly thereafter when Emory University Hospital decided to launch a heart transplant program, but none of the senior surgeons wanted to do it. Even with the new drug, it was a risky surgery, and mortality was still high.

Its an all-or-nothing operation, Murphy says, as he sits down in his small office overlooking the grayish hospital compound. Hes wearing light blue scrubs from an early morning surgery. At 70, he still has boyish looks, with a lean build and an air of laid-back confidence. If you have a number of bad outcomes initially, it can be detrimental to your career as a surgeon, he says.

But Murphy didnt really have a choice. He remembers that during a meeting of Emorys cardiac surgeons in 1984, he was paged to check on a patient. When he returned, the physicians congratulated him on being appointed the head of the new heart transplant program. He was the youngest in the group and had been recruited from Harvards Massachusetts General Hospital just three years before.

Yeah, thats how I became Emorys first transplant surgeon, says Murphy.

He flew to California to shadow his colleagues at Stanford University Hospital, where most heart transplants were performed at the time. Back home at Emory, he put together a team and rigorously rehearsed the operation. The first transplant patient arrived in April 1985. The surgery was successful, as was the second operation less than a month later.

Around the same time, Harry Wuest wound up in a hospital in Orlando. He needed a transplant, but none of the medical centers in Florida offered the procedure. One of his doctors recommended Emory, and Wuest agreed. I knew I was dying. I could feel it. He was flown to Atlanta by air ambulance and spent several weeks in Emorys cardiac care unit until the evening of May 23, when Murphy walked into his room and said, Weve got a heart.

I could finally breathe again

The heart, as the patient later learned, came from a 19-year-old sophomore at Georgia Tech who had been killed in a car crash.

Organ transplants are a meticulously choreographed endeavor, where timing, coordination and logistics are key. While Murphy and his eight-member team were preparing for the surgery, Wuest was getting ready to say farewell to his family his wife and three teenage sons, and to thank the staff in the cardiac ward.

I was afraid, he recalls, especially of the anesthesia. It scared the heck out of me. He pauses during the reminiscence, choking briefly. I didnt know if I was going to wake up again.

The surgery took six hours. Transplants usually happen at night because the procurement team, the surgeons who retrieve different organs from the donor, only start working when regularly scheduled patients are out of the operating room.

Despite the cultural mystique surrounding the heart as the seat of life, Murphy says that during a transplant surgery, its not like the big spirit comes down to the operating room. Its very technical. As the team follows a precise routine, emotions are kept outside the door. We dont have time for that. Emotions come later.

Waking up from the anesthesia, Wuests first coherent memory was of Murphy entering the room and saying to a nurse, Lets turn on the TV, so Harry can watch some sports.

Wuest spent the next nine days in the ICU, and three more weeks in the hospital ward. In the beginning, he could barely stand up or walk, because he had been bedridden weeks before the surgery and had lost a lot of muscle. But his strength came back quickly. I could finally breathe again, he says. Before the surgery, he felt like he was sucking in air through a tiny straw. I cannot tell you what an amazing feeling that was to suddenly breathe so easily.

Joane Goodroe was the head nurse at Emorys cardiovascular post-op floor back then. When she first met Wuest before the surgery, she recalls him lying in bed and being very, very sick. When she and the other nurses finally saw him stand up and move around, he was a whole different person.

In the early days of Emorys heart transplant program, physicians, nurses and patients were a particularly close-knit group, remembers Goodroe, whos been a nurse for 42 years and now runs a health care consulting firm. There were a lot of firsts for all of us, and we all learned from each other, she said.

Wuest developed friendships with four other early transplant patients at Emory, and he has outlived them all.

When he left the hospital, equipped with a new heart and a fresh hunger for life, Wuest made some radical changes. He decided not to return to Florida but stay in Atlanta. Thats where he felt he got the best care, and where he had found a personal support network. And he got a divorce. Four months after the operation, he went back to working full-time: first in temporary jobs and eventually for a property management company.

After having been sick for 12 years, I was just so excited to be able to work for eight hours a day, he recalls. That was a big, big deal for me.

At 50, he went back to school to get his CPA license. He also found new love.

Martha was a head nurse in the open-heart unit and later ran the cardiac registry at Saint Josephs Hospital. Thats where Wuest received his follow-up care and where they met in 1987. Wuest says for him it was love at first sight, but it took another five years until she finally agreed to go out with him. Six months later, they were married.

Harry Wuest and his wife, Martha. She was a head nurse in the open-heart unit and later ran the cardiac registry at Saint Josephs Hospital. Thats where Wuest received his follow-up care and where they met in 1987. Wuest says for him it was love at first sight, but it took another five years until she finally agreed to go out with him. Six months later, they were married.

Having worked in the transplant office, I saw the good and the bad, Martha Wuest says. A petite woman with short, perfectly groomed silver hair, she sits up very straight on the couch, her small hands folded in her lap. Not every transplant patient did as well as Harry. And I had a lot of fear in the beginning. Now he may well outlive her, she says with a smile and a wink.

Wuests surgeon, meanwhile, went on to fight his own battles. Two and a half years into the program, Murphy was still the only transplant surgeon at Emory and on call to operate whenever a heart became available. Frustrated and exhausted, he quit his position at Emory and signed up with Saint Josephs (which at the time was not part of the Emory system) and started a heart transplant program there.

At St. Josephs, Murphy continued transplanting hearts until 2005. In total, he did more than 200 such surgeries.

Being a heart transplant surgeon is a grueling profession, he says, and very much a younger surgeons subspecialty.

He then shifted his focus and became a pioneer in robotically assisted heart surgery. He has done more than 3,000 operations with the robot, mostly mitral valve repairs and replacements more than any other cardiac surgeon in the world.

Heart transplants "remain the gold standard"

Since Murphy sewed a new heart into Wuest 35 years ago, there has been major progress in the field of heart transplants, but it has been uneven.

There is improved medication to prevent rejection of the donor heart, as well as new methods of preserving and transporting donor hearts.

Yet patients requiring late-stage heart failure therapy, including transplantation, still exceed the number of donor hearts available. In 2019, 3,551 hearts were transplanted in the United States, according to the national Organ Procurement and Transplantation Network. But 700,000 people suffer from advanced heart failure, says the American Heart Association.

New technologies and continued research are providing hope to many of these patients. There has been significant progress in the development of partial artificial hearts, known as Left Ventricular Assist Devices, or LVADs. They can be used as bridge devices, to keep patients alive until donor hearts are available, or as destination therapy, maintaining patients for the remainder of their lives.

Also, total artificial hearts have come a long way since the first artificial pump was implanted in a patient in 1969. The technology is promising, says Dr. Mani Daneshmand, the director of Emorys Heart & Lung Transplantation Program. But its not perfect.

Long-term research continues into xenotransplantation, which involves transplanting animal cells, tissues and organs into human recipients.

Regenerative stem cell therapy is an experimental concept where stem cell injections stimulate the heart to replace the rigid scar tissue with tissue that resumes contraction, allowing for the damaged heart to heal itself after a heart attack or other cardiac disease. Certain stem cell therapies have shown to reverse the damage to the heart by 30 to 50 percent, says Dr. Joshua Hare, a heart transplant surgeon and the director of the Interdisciplinary Stem Cell Institute at the University of Miamis Miller School of Medicine.

All of these ideas have potential, says Daneshmand. But none of them are ready to replace a human donor heart. A heart transplant remains the gold standard, because you cant accommodate the same success with a machine right now, he says.

Efforts around expanding the donor pool are really the best way to address this problem, while we wait for technology to catch up, he adds.

Besides Emory, other health care systems in Georgia that currently have a heart transplant program are Piedmont Healthcare, Childrens Healthcare of Atlanta and Augusta University Health.

Organ rejection remains a major issue, and long-term survival rates have not improved dramatically over the past 35 years. The 10-year survival is currently around 55 percent of patients, which makes long-term survivors like Harry Wuest rare in the world of heart transplants.

The United Network of Organ Sharing, or UNOS, which allocates donor hearts in the United States, doesnt have comprehensive data prior to 1987. An informal survey of the 20 highest-volume hospitals for heart transplants in the 1980s found only a scattering of long-term survivors.

In for the long haul

Being one of the longest-living heart transplant recipients is something that Wuest sees as a responsibility to other transplant patients, but also to the donors family, which hes never met. If you as a transplant recipient reject that heart, thats like a second loss for that family.

Part of this responsibility is living a full and active life. Both he and Martha have three children from their previous marriages and combined they have 15 grandchildren. Most of their families live in Florida, so they travel back and forth frequently. Wuest still works as a CPA during tax season, and he does advocacy for the Georgia Transplant Foundation. In addition to golf, he enjoys lifting weights and riding his bike.

Hes had some health scares over the years. In 2013, he was diagnosed with stage 1 kidney cancer, which is in remission. Also, he crossed paths with his former surgeon, and not just socially. In 2014, Murphy replaced a damaged tricuspid valve in Wuests new heart. That operation went well, too.

Murphy says there are several reasons why Wuest has survived so long. Obviously, his new heart was a very good match. But a patient can have the best heart and the best care and the best medicines and still die a few months or years after the transplantation, the surgeon says. Attitude plays a key role.

Wuest was psychologically stable and never suffered from depression or anxiety, Murphy says. Hes a numbers guy. He knew the transplant was his only chance, and he was set to pursue it.

Wuest attributes his longevity to a good strong heart from his donor; good genetics; great doctors and nurses; and a life that he loves. Im just happy to be here, he says.

Quoting his former surgeon and friend, he adds: Doug always said, Having a transplant is like running a marathon. And Im in for the long haul.

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34 years with a new heart and counting - MDJOnline.com

Cardiac Stem Cells Comments Off on 34 years with a new heart and counting – MDJOnline.com | February 24th, 2020

Houston, TX A variety of science investigations, along with supplies and equipment, launch to the International Space Station on the 20th SpaceX commercial resupply services mission.

The Dragon cargo spacecraft is scheduled to leave Earth March 2nd from Space Launch Complex 40 at Cape Canaveral Air Force Station in Florida. Its cargo includes research on particle foam manufacturing, water droplet formation, the human intestine and other cutting-edge investigations.

Airbus workers unpack the Bartolomeo platform at NASAs Kennedy Space Center in Florida in preparation for its launch to the International Space Station. The platform, manufactured by Airbus Defence and Space, hosts multiple external payloads in low-Earth orbit. (NASA)

The space station, now in its 20th year of continuous human presence, provides opportunities for research by government agencies, private industry, and academic and research institutions.

Such research supports Artemis, NASAs missions to the Moon and Mars, and leads to new technologies, medical treatments and products that improve life on Earth.

Particle foam molding is a manufacturing process that blows thousands of pellets into a mold where they fuse together. The shoe company Adidas uses this process to make performance midsoles, the layer between the sole of a shoe and the insole under your foot, for its products.

The BOOST Orbital Operations on Spheroid Tesellation (Adidas BOOST) investigation looks at how multiple types of pellets behave in this molding process. Using one type of pellet creates a foam with the same properties throughout the sole component. Using multiple pellet types can allow engineers to change mechanical properties and optimize shoe performance and comfort. Removing gravity from the process enables a closer look at pellet motion and location during the process.

Results of this investigation could demonstrate the benefits of microgravity research for manufacturing methods, contributing to increased commercial use of the space station. New processes for particle foam molding could benefit a variety of other industries, including packaging and cushioning materials.

The Bartolomeo facility, created by ESA (European Space Agency) and Airbus, attaches to the exterior of the European Columbus Module. Designed to provide new scientific opportunities on the outside of the space station for commercial and institutional users, the facility offers unobstructed views both toward Earth and into space.

Airbus is collaborating with the United Nations Office of Outer Space Affairs to offer UN Member States the opportunity to fly a payload on Bartolomeo. Developing countries are particularly encouraged to participate, and the mission is devoted to addressing the UNs Sustainable Development Goals. Bartolomeo is named for the younger brother of Christopher Columbus.

Droplet Formation Studies in Microgravity (Droplet Formation Study) evaluates water droplet formation and water flow of Delta Faucets H2Okinetic showerhead technology. Reduced flow rates in shower devices conserve water, but also can reduce their effectiveness.

That can cause people to take longer showers, undermining the goal of using less water. Gravitys full effects on the formation of water droplets are unknown, and research in microgravity could help improve the technology, creating better performance and improved user experience while conserving water and energy.

Insight gained from this investigation also has potential applications in various uses of fluids on spacecraft, from human consumption of liquids to waste management and use of fluids for cooling and as propellants.

Human intestine cells forming microvilli inside Emulates Intestine-Chip. (Emulate)

Organ-Chips as a Platform for Studying Effects of Space on Human Enteric Physiology (Gut on Chip) examines the effect of microgravity and other space-related stress factors on biotechnology company Emulates human innervated Intestine-Chip (hiIC). This Organ-Chip device enables the study of organ physiology and diseases in a laboratory setting. It allows for automated maintenance, including imaging, sampling, and storage on orbit and data downlink for molecular analysis on Earth.

A better understanding of how microgravity and other potential space travel stressors affect intestine immune cells and susceptibility to infection could help protect astronaut health on future long-term missions. It also could help identify the mechanisms that underlie development of intestinal diseases and possible targets for therapies to treat them on Earth.

Self-assembly and self-replication of materials and devices could enable 3D printing of replacement parts and repair facilities on future long-duration space voyages. Better design and assembly of structures in microgravity also could benefit a variety of fields on Earth, from medicine to electronics.

Called self-assembled colloidal structures, these are vital to the design of advanced optical materials, but control of particle density and behavior is especially important for their use in 3D printing. Microgravity provides insight into the relationships among particle shape, crystal symmetry, density and other characteristics.

Functional structures based on colloids could lead to new devices for chemical energy, communication, and photonics.

The Multi-use Variable-g Platform (MVP) used for the MVP Cell-03 experiment, shown with the MVP door removed and two carousels inside. (Techshot Inc.)

Generation of Cardiomyocytes From Human Induced Pluripotent Stem Cell-derived Cardiac Progenitors Expanded in Microgravity (MVP Cell-03) examines whether microgravity increases the production of heart cells from human-induced pluripotent stem cells (hiPSCs).

HiPSCs are adult cells genetically reprogrammed back into an embryonic-like pluripotent state, which means they can give rise to several different types of cells. This makes them capable of providing an unlimited source of human cells for research or therapeutic purposes.

For MVP Cell-03, scientists induce the stem cells to generate heart precursor cells, then culture those cells on the space station for analysis and comparison with cultures grown on Earth.

These heart cells or cardiomyocytes (CMs) could help treat cardiac abnormalities caused by spaceflight. In addition, scientists could use them to replenish cells damaged or lost due to cardiac disease on Earth and for cell therapy, disease modeling and drug development. Human cardiac tissues damaged by disease cannot repair themselves, and loss of CMs contributes to eventual heart failure and death.

These are just a few of the hundreds of investigations currently aboard the orbiting laboratory. For daily updates, follow @ISS_Research, Space Station Research and Technology News or our Facebook. Follow the ISS National Lab for information on its sponsored investigations. For opportunities to see the space station pass over your town, check out Spot the Station.

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NASA to send equipment to International Space Station to research Improving Shoes, Showers, 3D Printing - Clarksville Online

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February 23, 2020 by Michael Kuttner

Read on for article

As we hurtle towards round three in our general elections the frenetic canvassing of voters by desperate political parties is in stark contrast to the switched-off interest shown by those targeted.

Thankfully the same cannot be said for the daily announcements of further advances in good news whether it is scientific, medical or social spheres. Israeli ingenuity continues to be a light unto the nations.

QUICKER RESULTS EQUALS QUICKER DIAGNOSIS

The Israeli innovation can test 100 saliva samples in 15 minutes as opposed to one blood test that takes an hour to confirm coronavirus.

Quick diagnosis can help prevent the spread ofcoronavirus by slashing the timeit takes to decide that patients need to be quarantined and treated.

The technology is already in use for diagnosing the Zika virus and is used at Israels Tel Hashomer Hospital in Ramat Gan by the Ministry of Healths central virology laboratory.

ANOTHER ADVANCE IN CARDIAC CARE

Researchers succeeded in producing 3D engineered cardiac tissues from chamber-specific heart cells derived from human stem cells. This medical development opens the door for creating personalized medications for cardiac patients and advances in new cardiac drug developments.

This research model simulates the most common irregular heartbeat (arrhythmia), called atrial fibrillation. It opens the door for testing the success of various drugs on individual patients to prevent or stop arrhythmia.

Because they were able to separate atrial and ventricular tissue models, researchers can discover which drugs improve atrial cell function without damaging ventricular cell function.

DEFEATING CYBER HACKERS

Researchers from Ben-Gurion University (BGU) presented at the Cybertech Global Tel Aviv conference the first all-optical stealth encryption technology. The innovation uses fibre-optic light transmissions to secure cloud computing and data centre network transmission.

The technology uses standard optical equipment to send data in a manner that cannot beintercepted by hackers, unlike conventional digital methods. Another aspect of the system is that data gets destroyed if a hacker tries to decode it.

Because an eavesdropper can neither read the data nor even detect the existence of the transmitted signal, the optical stealth transmission provides thehighest level of privacy and securityfor sensitive data applications.

The patented technology has multiple applications, including high-speed communication and sensitive transmission of financial, medical or social media-related information. According to the Senior Vice President, Exact Sciences & Engineering, BGN Technologies, An eavesdropper will require years to break the encryption key.

TWO THOUSAND YEARS LATER

Long after the Romans departed archeological discoveries continue to be made. Two thousand years later the descendants of the Jews they tried to ethnically cleanse, now restored in their homeland, walk again in the very places they were once exiled from.

RECLAIMING A LOST HERITAGE

One of the miracles one witnesses by living in Israel is meeting Jews long lost to their heritage somehow finding their way back to their Faith and People.

Whether it is the Bnei Menashe from India, tribes from Uganda, individuals from Kaifeng, China or Jews from Ethiopia the common theme is of a return to Zion.

Often overlooked but now becoming a frequent occurrence is the discovery by descendants of Conversos, those driven underground or forcibly converted by the Spanish and Portuguese Inquisition five hundred years ago, of their Jewish heritage.

Watch this moving video of one of the latest such personal dramas. We truly are living in amazing times when lost Jews from the four corners of the world are returning.

Visit J-Wire's main page for all the latest breaking news, gossip and what's on in your community.

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On the other hand - J-Wire Jewish Australian News Service

Cardiac Stem Cells Comments Off on On the other hand – J-Wire Jewish Australian News Service | February 24th, 2020

Researchers from the group of Vlad Cojocaru together with colleagues the Max Planck Institute in Mnster (Germany) have revealed how an essential protein helps to activate genomic DNA during the conversion of regular adult human cells into stem cells.A cells identity is driven by which DNA is read or not read at any point in time. Signaling in the cell to start or stop reading DNA happens through proteins called transcription factors. Identity changes happen naturally during development as cells transition from an undesignated cell to a specific cell type. As it turns out, these transitions can also be reversed. In 2012, Japanese researchers were awarded the Nobel prize for being the first to push a regular skin cell backwards to a stem cell.A fuller understanding of molecular processes towards stem cell therapiesUntil now, it is unknown how the conversion of a skin cell into a stem cell happens exactly, on a molecular scale. Fully understanding the processes with atomic details is essential if we want to produce such cells for individual patients in the future in a reliable and efficient manner, says research leader Vlad Cojocaru of the Hubrecht Institute. It is believed that such engineered cell types may in the future be part of the solution to diseases like Alzheimers and Parkinsons, but the production process would have to become more efficient and predictable.Pioneer transcription factorOne of the main proteins involved in the stem cell generation is a transcription factor called Oct4. It induces gene expression, or activity, of the proteins that reset the adult cell into a stem cell. Those genes induced are inactive in the adult cells and reside in tightly packed, closed states of chromatin, the structure that stores the DNA in the cell nucleus. Oct4 contributes to the opening of chromatin to allow for the expression of the genes. For this, Oct4 is known as a pioneer transcription factor.

The data from Cojocaru and his PhD candidate and first author of the publication Jan Huertas show how Oct4 binds to DNA on the so-called nucleosomes, the repetitive nuclear structures in chromatin. Cojocaru: We modelled Oct4 in different configurations. The molecule consists of two domains, only one of which is able to bind to a specific DNA sequence on the nucleosome in this phase of the process. With our simulations, we discovered which of those configurations are stable and how the dynamics of nucleosomes influence Oct4 binding. The models were validated by experiments performed by our colleagues Caitlin MacCarthy and Hans Schler in Mnster.One step closer to engineered factorsThis is the first time computer simulations show how a pioneer transcription factor binds to nucleosomes to open chromatin and regulate gene expression. Our computational approach for obtaining the Oct4 models can also be used to screen other transcription factors and to find out how they bind to nucleosomes, Cojocaru says.

Moreover, Cojocaru wants to refine the current Oct4 models to propose a final structure for the Oct4-nucleosome complex. For already almost 15 years now, we know that Oct4 together with three other pioneer factors transforms adult cells into stem cells. However, we still do not know how they go about. Experimental structure determination for such a system is very costly and time consuming. We aim to obtain one final model for the binding of Oct4 to the nucleosome by combining computer simulations with different lab experiments. Hopefully, our final model will give us the opportunity to engineer pioneer transcription factors for efficient and reliable production of stem cells and other cells needed in regenerative medicine.ReferenceHuertas et al. (2020) Nucleosomal DNA Dynamics Mediate Oct4 Pioneer Factor Binding. Biophysical Journal. DOI: https://doi.org/10.1016/j.bpj.2019.12.038

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

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Visualizing the Conversion of Adult Cells to Stem Cells - Technology Networks

Skin Stem Cells Comments Off on Visualizing the Conversion of Adult Cells to Stem Cells – Technology Networks | February 22nd, 2020

LEXINGTON, Ky. (WKYT) - Firefighters in Lexington spent their Saturday morning supporting their own with a pancake breakfast raising money for a young woman with a rare disease.

Chris OBryan has worked as a firefighter in Kentucky for 23 years. This past summer his daughter began experiencing stomach pain. After months of testing, doctors diagnosed her PCH, a rare disease caused by a mutation in bone marrow stem cells.

Once other Lexington Firefighters heard about OBryans daughter, they decided to help however they can.

Its seven days that this came together, said firefighter William Tabor.

Tabor initiated the breakfast without ever meeting OBryan and his family before.

Both men say that goes to show the family community within the fire department. Within just more than an hour of beginning the breakfast at 8 Saturday, they raised $5,000.

The money thats raised here is twofold, started OBryan. Some of its for my daughter and some of its for a Louisville firefighter who was tragically killed in Saint Louis.

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Firefighter raise thousands within hours for one of their own - WKYT

Bone Marrow Stem Cells Comments Off on Firefighter raise thousands within hours for one of their own – WKYT | February 22nd, 2020

The first time Bill Cronin Googled his own cancer diagnosis in 2016, his heart sank. He had Szary syndrome, a rare and aggressive form of cutaneous T-cell lymphoma and staring back at him were countless articles predicting a negative prognosis.

However, after receiving a stem-cell transplant at Dana-Farber/Brigham and Womens Cancer Center, Cronin is returning to the life he enjoyed before cancer.

Im at a place I never thought Id get to, Cronin says.

In 2015, Cronin, then 60, started feeling incredibly itchy and developed an accompanying rash. He went to his dermatologist, who diagnosed him with eczema and told him to return in five months. The rash continued to grow, however, and at the five month mark, Cronins dermatologist encouraged him to undergo further testing at Dana-Farber.

A blood test revealed that Cronins T-cells a type ofwhite blood cells that make up part of the immune system had becomecancerous. In the case of Szary syndrome, lymphoma cells will circulatethrough the blood stream and deposit in different areas of the skin. This willgenerally lead to a full-body rash and intense itchiness.

Cronin would need a stem cell transplant to combat the disease, but before he could receive one, his care team had to get him into remission. Patients who do not achieve remission prior to transplant have a high chance of relapsing.

When they first told me everything, I was really scared, says Cronin. But I knew I was in one of the best places in the world to figure out and treat this rare disease.

Cronins pre-transplant care was spearheaded by oncologists David Fisher, MD, and Nicole LeBoeuf, MD, MPH, clinical director of Cutaneous Oncology at Dana-Farber, with his transplant conducted by Corey Cutler, MD, MPH, medical director of the Adult Stem Cell Transplantation Program at Dana-Farber. Initially, Cronins disease was incredibly resistant; for nearly three years, mainstay drugs including steroids, monoclonal antibodies, and enzyme blockers all failed to put his disease into remission.

Ultimately, it would take a new drug, mogamulizumab (a type of immunotherapy that directly kills T-cells involved with Sezary Syndrome) to get Cronins disease into remission.

In May 2019, Cronin was cleared to undergo an allogeneic transplant, a type of transplant that uses a donors stem cells, in this case, Cronins brother. Since his transplant Cronin has remained in remission.

We had to use all of our big guns to get him totransplant, but Im pleased with where we are now, says Cutler.

I know the situation can always change, but it was great tobe able to share some good news with my family and friends, adds Cronin.

Patients like Cronin serve as a reminder of how stem cell transplants have improved and continue to impact patient outcomes, Dana-Farber experts note. Initially offered to only an incredibly small patient population when first performed at Dana-Farber in the 1970s, research advancements have, and continue to, broaden who is eligible for a transplant. In 2019, Dana-Farber/Brigham and Womens Cancer Center (DF/BWCC) surpassed 10,000 total adult transplants.

This milestone indicates our success as a program and our volume has allowed us to do the research to help move the field forward rather impressively, says Joseph Antin, MD, chief emeritus of Adult Stem Cell Transplantation at DF/BWCC.

In 1996, Dana-Farber Cancer Institute and Brigham and Womens Hospital merged their then separate transplant centers. By pooling together physical and intellectual resources, the new combined program was able to more than double the number of transplants each hospital could perform individually.

We always felt collaboration was better than competition, explains Robert Soiffer, MD, vice chair of Medical Oncology for Hematological Malignancies and chief of the Division of Hematologic Malignancies, who oversaw the merger with Antin. Each side could learn from the other, and that helped to catapult us into the leadership position we have today.

The Stem Cell Transplantation Program is also bolstered by the Connell and OReilly Families Cell Manipulation Core Facility (CMCF), which was established in 1996. The state-of-the-art center, led by Jerome Ritz, MD, not only processes the stem cells for transplant; it also assists researchers in developing new cell-based therapies for patients.

Another key component to the programs success has been the creation of the Ted and Eileen Pasquarello Tissue Bank. The Pasquarello Tissue Bank receives, processes, banks, and distributes research samplesof blood, bone marrow, and other tissues. Through a database overseen by Vincent Ho, MD, the Institute is able to log, assess, and later review every patients disease, including all complications and mutations. This technology allows researchers to explore the genetic makeup of past donors and better understand why a transplant was or was not successful.

Were still learning from biological specimens we collected 20 years ago, and it will continue to impact care 20 years from now, Soiffer says.

Today, there is a continuous push to develop new and more precise therapies to complement and improve stem cell transplants. The hope is to bring new treatment options to patients like Cronin who are facing rare and difficult diseases.

Before his diagnosis, Bill, and Barbara Finney, his partner ofnearly 30 years, were avid English Country dancers. English Country dancingevolved from the court dances of Europe in the early 17th century, and Croninand Barbara have friends from all over the country who share their passion forit.

While Cronin isnt dancing just yet, as hes stillrecovering from his transplant, he says he couldnt have gotten through thiswithout his partner on the dance floor and in life.

Barbara has been amazing and has helped take care ofeverything I couldnt do, he adds. Ive been fortunate and privileged to notonly have her, but to have been able to come to Dana-Farber.

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Transplant for Szary Syndrome is Patient's First Step in Returning to the Dance Floor - Dana-Farber Cancer Institute

Bone Marrow Stem Cells Comments Off on Transplant for Szary Syndrome is Patient’s First Step in Returning to the Dance Floor – Dana-Farber Cancer Institute | February 22nd, 2020

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Dubai: It's an industry on the rise. And it's accelerating the speed by which Dubai is fast becoming the "new Beverly Hills of the Middle East."

Already, cosmetic surgery has topped the list in medical tourism in Dubai. According to a Dubai Health Authority (DHA) report, the emirate has the highest number of cosmetic surgeons per capita in the region about 50 specialists for a million people.

Globally, the cosmetic surgery market size is projected to reach $21.97 billion with 7.8% annual growth rate by 2023, according to a Medgadget report.

These are the top surgery options:

To put this in perspective, we talked to several people.

Myra J., a belly dancer working at a Dubai hotel. She was unhappy. Saddle bags on her thighs were making her performance less graceful. She honestly feared she would be jobless soon.

On a weekend, she checked into a leading aesthetic clinic and the surgeon trimmed the pockets of fat around her thighs, sculpting her body back into perfect shape.

It took her a week to recover and get back to work, but she is delighted with her shapely thighs and feels happier than ever before.

'Ageing gracefully'

Nina M., (52), always loved how beautiful her eyes were and how youthful her cheeks looked.

Last year, nowever, as she got busy with her sons marriage preparations, she started to experience bouts of anxiety at the sight of slowly encroaching bags under her eyes and a slight droop in her cheeks.

I wanted to look and feel my best and thought a filler and a round of botox were harmless indulgences. It took me just an hour at the clinic and the results were amazing. I am glad I was able to stop the onslaught of aging in time.

"Its beautiful to age gracefully, but we all do use creams and gels to delay the process. I think of these minor injectibles as tools in our make-up bag.

"I have used fillers and Botox twice since last year, combined with my beauty regimen. I feel it has given me not just confidence but also psychological boost. I would recommend it to everyone.

Anna M was a physical trainer but vexed with her body shape as she had a masculine build.

- Anna M, a physical trainer

This was affecting her work. Most women were intimidated by her personality and she was losing self-confidence.

Four years ago, she went in for breast implants and her life changed.

Addiction to surgery?

People talk about getting addicted to cosmetic surgery. But that is not so. Most people approach a cosmetic surgeon only when they cannot deal with a physical issue themselves and just like one needs medicine when one is ill, cosmetic surgery acts as a solution to boost confidence and self-esteem.

"Look at how people have reclaimed their health with gastric bypass!

- Dr Sanjay Parashar, chairman, Scientific of the Emirates Plastic Surgery Society

Changing the world one person at a time and providing them with an incredible burst of confidence, aesthetic and cosmetic surgeons in the UAE have built a practice of reliability that can take as little as one hour to a day to transform your personality.

Welcome to the multi-million dirham cosmetic surgery industry in the UAE.

It is the toast of medical tourism in the region, with a high footfall of Gulf and Asian medical tourists as well as resident expatriates.

The industry has accelerated at a speed that is making Dubai be hailed as the new Beverly Hills of the Middle East.

Cosmetic or Plastic Surgery?

These are two different concepts. Cosmetic surgery refers to aesthetic surgery and revolves around enhancement of physical features of an individual and is elective.

This includes procedures such as:

Plastic surgery, on the other hand, is a surgical speciality dealing with life-saving procedures of re-construction of the face and body owing to congenital defects, disfigurement due to accident, trauma, burns, tumour removal due to diseases such as cancer.

In most cases, plastic surgery is not elective.

Dh12b Medical tourism sales in 2018

Since the time pop stars began to inundate Instagram with images of their perfect bodies, dazzling smiles, flawless skin and enviable hair volume, elective procedures have become commonplace with teenagers as young as 13 who are going in for instant fixes.

While some procedures require a couple of days of hospital stay and being out of circulation for a while, many quick fixes are carried out during lunch breaks in one-hour durations.

Highest per capita cosmetic surgeons in UAE

Dr Sanjay Parashar, chairman, Scientific of the Emirates Plastic Surgery Society, told Gulf News: Cosmetic surgery tops the list in medical tourism in Dubai and according to a 2015 report of Dubai Health Authority (DHA), Dubai has the highest number of cosmetic surgeons per capita in the region about 50 specialists for a million people.

- Dr Zuhair Al Fardan, President of the Emirates Plastic Surgery

Dr Parashar added: The field has grown beyond expectations and much of the credit goes to the development of a world-class infrastructure in this field and the corresponding health regulations.

"Today, in Dubai, most Day Care Surgery centres where most of the plastic surgery procedures are carried out have the best international accreditations.

Dr Zuhair Al Fardan, President of the Emirates Plastic Surgery, said: Much of the advancement in plastic and cosmetic surgery is work in progress as surgeons are constantly upgrading themselves with techniques, technologies.

The UAE is keeping abreast of the best that is taking place in the world. In the last five years or so, there have been tremendous advancements in cosmetic and plastic surgery in the UAE.

"We have the top plastic surgeons of the world come here to do surgeries and the UAE hosts two major international plastic surgery conferences each year.

Soaring revenues

With greater acceptance and broadening of scope for the discipline, it is evident that plastic surgery is a major revenue earner and places UAE as one of the leading medical tourism destinations in the region.

From a price range of Dh150-250 for a filler to Dh40,000 for a detailed body sculpting procedure, these procedures are money-spinners.

Together, the plastic and cosmetic surgery is a multimillion dirham business in the UAE. While there are no exact figures available, safe estimates can be made, say surgeons.

MULTIMILLION BUSINESS

There are 30 hospitals in Dubai, of which 70 per cent are internationally accredited. The emirate aims to build 22 hospitals by 2020 18 private and 4 public hospitals.

In Dubai, at least 50 per cent of its 30 hospital offer cosmetic and plastic surgery options.

30 number of hospitals in Dubai, of which 70 per cent are internationally accredited

Besides that there are about 150 Day Care Surgery centres and 400 aesthetic clinics in Dubai.

All of them offer a bouquet of cosmetic surgery procedures and their average annual revenue is between Dh4-6 million a year.

400 Number of aesthetic clinics in Dubai

If one were to compute that with the numbers of facilities including hospitals, the annual revenue from cosmetic surgery would run into many millions of dirhams annually.

High on medical tourism

Currently, Dubai aims to attract 500,000 medical tourists a year by end 2020.

In a short priod of time, Dubai has managed to be ranked 17 among the top 25 global destinations for medical tourism and cosmetic surgery, along with fertility, orthopaedic, dental and wellness disciplines in the list of most-billed medical procedures.

40% percentage of tourists who come to Dubai come only for medical tourism. Medical tourism sales topped Dh12 billion in 2018, a 5.5% jump in the number of medical tourists

Based on official data, about 46 per cent of the current medical tourists in Dubai come from Asian countries. About a quarter (25 per cent), come from GCC and Arab countries and 13 per cent from African countries, and the remaining 16 per cent from other countries, mainly the UK and Commonwealth of Independent States (CIS) countries.

In fact, 40 per cent of tourists who come to Dubai come only for medical tourism.

- Dr Francis Conroy, consultant plastic, cosmetic and reconstructive surgeon at the American Hospital, Dubai

Medical tourism sales topped Dh12 billion in 2018, with a 5.5 per cent overall increase in medical tourists.

Medical tourists

Dubai attracted a total of 640,542 international and domestic medical tourists in 2018 (51 per cent were international patients).

European tourists consisting mostly of UK, French and Italian citizens, share 16 per cent of health and wellness tourists. A substantial medical tourism revenue, it is evident, is earned through plastic and cosmetic surgery.

Dr Francis Conroy, consultant plastic, cosmetic and reconstructive surgeon at the American Hospital, Dubai, remarked: American Hospital Dubai, is one of the few facilities offering both comprehensive reconstructive and cosmetic surgery services.

"Our plastic surgeons are fully trained in both reconstructive and cosmetic surgery so we see a wide-ranging case mix, from severe trauma cases to cancer cases and of course, those opting for cosmetic surgery."

Most popular cosmetic surgery

Typically, these patients would be female, who done with having children and raising them, now wish to address the changes in their body. I also see a large number of male patients who want to correct problems associated with their chest with the help of liposuction.

Source: Dr Francis Conroy, consultant plastic, cosmetic and reconstructive surgeon at the American Hospital, Dubai

Most popular cosmetic surgery

"Typically, these patients would be female, who done with having children and raising them, now wish to address the changes in their body. I also see a large number of male patients who want to correct problems associated with their chest with the help of liposuction, said Dr Conroy.

Non-surgical treatments (neuro-modulators, fillers, etc) are still very popular and I have seen a trend in that patients are starting with such treatments at an earlier age.

Given the prestigious reputation of the hospital and the Dubai governments plan to promote medical tourism, I have noticed a huge influx of patients from Africa, Nigeria and Ghana in particular.

"These patients come mainly for cosmetic surgery, knowing that they are in the hands of a highly qualified surgeon, in a safe, luxurious facility, with standards second to none, said Dr Conroy.

Top six cosmetic surgery procedures in town

The procedures can be divided into categories:

Does health insurance cover plastic surgery?

"Reconstructive surgery such as correction of birth deformities such as a tuberous breast, cleft lip, hand deformities, skin transplant following burns, road trauma and breast augmentation and reconstruction following a mastectomy and rebuilding after a tumour resection is all covered under all leading health insurances. There is also new kinds of stem cell therapy being used to regenerate tissues and nerves especially in diabetic patients.

Know the rules:

The DHA has made it mandatory for all Day Care Surgery Centres, most of who carry out aesthetic procedures, to have one leading international accreditation from Canada, US, UK or Australia.

These accreditations were earlier mandatory for hospitals only, but from 2020, all Day Care Surgery centres compulsorily must have an international accreditation.

This ensures that an independent, international medical body enforces global health standards to grant them certification and in case of a sentinel event, conducts its independent inquiry and downgrades these places in case of a serious lapse.

DHA on its own has issued a 25-page manual on quality and regulations that is to be followed at all centres.

A close examination of the Day Care Surgery centres indicates several layers of quality control.

Pre surgery quality

This involves free consultation, especially in case of a second opinion or a first time patient seeking to enquire about a procedure based on his/her requirement. When a patient uploads a request on the website of a centre from anywhere in the world, the centre has to provide a detailed consultation free of charge.

Services available to a patient

Once the patient is convinced and comes in person to consult the doctor, quality is upheld in the pre-diagnostic tests that the patient has to undergo.

Infrastructure quality

DHA has graded Day Care Centres into A, B and C categories based on the level of medical facilities that can be accessed by a patient. Anaesthesia methods such as oral, epidural and general also help classify centres. For instance, hair transplant procedures can only be carried out in B and above grade clinics. Day Care Centres that conduct surgeries under general anaesthesia much be equipped with the Advance Cardiac Life Support (ACLS) with their surgeons and registered nurses being certified as trained in administering ACLS to a patient.

Patient safety protocol

There are very specific guidelines for patient safety and the doctor/surgeon must explain the procedure in detail to the patient and his/her family and obtain a written consent to go ahead after ascertaining that all risks and side-effects have been clearly explained to the patient.

Post-operative regulation

There are specific protocols for discharge of patients undergoing cosmetic surgery.

Although ambulatory care means the patient has to be discharged within the same day, there is a specification about asking the patient to desist from long-distance travel, specific rehabilitative work to be carried out from the next day for which the patient has to be within Dubai and also specific instructions when an overseas patient is declared fit to fly out of the country.

When a patient flies out, he or she is provided with a proper review and notes, with instructions for overseas rehabilitation protocol with reference notes for the rehabilitation instructor and instructions for medication and periodic reviews.

Accountability

The law is clear, said Dr Al Fardan, The DHA regulations constitutes an accountability committee and holds an enquiry to fix the liability. If the centre is found guilty, its licence can be suspended or cancelled depending on the extent of guilt. If the surgeon, anaesthesiologists, nurse and technicians are found guilty, their license to practice is suspended or cancelled. If the crime is lighter, then both the centre and the team are let off with serious warnings.

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Dubai is the 'new Beverly Hills of the Middle East': Here's why - Gulf News

Cardiac Stem Cells Comments Off on Dubai is the ‘new Beverly Hills of the Middle East’: Here’s why – Gulf News | February 22nd, 2020

Alfie Commons and mum Lorna, 40, met Christin Bouvier, 34, for the first time on Wednesday after spending more than two years communicating via anonymous letters due to donation laws.

Alfie, from Toton, Notts, was diagnosed with acute lymphoblastic leukaemia (ALL) at seven months after he had a cold for six weeks.

After chemotherapy failed, Alfie was put on a trial immunotherapy drug which helped him to recover to be eligible for a stem cell transplant.

Unable to find a family match, his family found Christin on a worldwide register operated by blood cancer charity DKMS. She donated her cells in a one-hour op and they were flown to the UK and slowly passed into Alfies body in August 2016.

When the teacher, from Schwerin, Germany, was finally allowed to meet the family at Chiswick Town Hall in west London, the little boy gave a gift of Lacoste Pink perfume.

Mum-of-two Lorna, who works in HR, said: The meet-up was just amazing, it was everything we could have possibly dreamed of there were lots of hugs and tears.

It didnt feel like I was meeting her for the first time because wed been chatting for so long before.

As a family, we owe so much to Christin, words of thanks will never feel enough.

Christin just cried when she heard the recipient was a baby. She said: After I donated my bone marrow and the anaesthetic wore off, I called DKMS.

They told me that Alfie was a small baby and living in the UK but couldnt tell me any more due to the laws. When I found out Alfie was responding to treatment, so many tears of joy ran down my face. I still cant describe that moment.

Its a moment that is always with me. Whenever I feel a bit down, I think back to it as it always brings me so much happiness! She added that the meeting was so amazing. I was very nervous and shaking at first and when we finally met we cried a lot and hugged.

Alfie was shy at first but after a bit of time he became more comfortable and we played with some balloons and had a slice of cake.

After the transplant, Alfie developed a deadly immune condition, but this was controlled by medication. He was given the cancer all-clear in 2017 and has started school.

Lorna added: I just want more people to sign up to become donors theres a match for everyone.

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From Germany with love: Alfie, four, meets his stem cell saviour - Express

Bone Marrow Stem Cells Comments Off on From Germany with love: Alfie, four, meets his stem cell saviour – Express | February 21st, 2020

CHICAGO, Feb. 21, 2020 /PRNewswire/ -- According to the new market research report "Single-cell Analysis Marketby Cell Type (Human, Animal, Microbial), Product (Consumables, Instruments), Technique (Flow Cytometry, NGS, PCR, Mass Spectrometry, Microscopy), Application (Research, Medical Application), End User - Global Forecasts to 2025", published by MarketsandMarkets, the Single-cell Analysis Marketis projected to reach USD 5.6 billion by 2025 from USD 2.1 billion in 2019, at a CAGR of 17.8% during the forecast period.

Download PDF Brochure: https://www.marketsandmarkets.com/pdfdownloadNew.asp?id=171955254

The growth in this market is driven by technological advancements in single-cell analysis products, increasing government funding for cell-based research, growing biotechnology and biopharmaceutical industries, wide applications of single-cell analysis in cancer research, growing focus on personalized medicine, and the increasing incidence and prevalence of chronic and infectious diseases. However, the high cost of single-cell analysis products is expected to restrain the growth of this market to a certain extent during the forecast period.

The research applications segment accounted for the largest share of the market, by application, in 2018

Based on application, the Single-cell Analysis Market is segmented into research (cancer, immunology, neurology, stem cell, and other research applications) and medical applications (noninvasive prenatal diagnosis, in vitro fertilization, and circulating tumor cell detection). The research applications segment accounted for the largest share of the market in 2018. Increasing government initiatives in stem cell research and the wide usage of single-cell analysis in cancer research are the major factors driving the growth of the research applications segment.

Browsein-depth TOC on"Single-cell Analysis Market"

225 Tables 50 Figures252 Pages

The flow cytometry segment accounted for the largest market share in 2018

Based on technique, the market is segmented into flow cytometry, NGS, PCR, microscopy, mass spectrometry, and other techniques. The flow cytometry segment accounted for the largest market share in 2018. The large share of this segment is attributed to the wide usage of flow cytometry in detecting and measuring the physical and chemical characteristics of a population of cells or particles. However, the NGS segment is projected to register the highest growth rate during the forecast period. The high growth of the NGS segment is driven by the increasing application of single-cell analysis products in drug discovery for cancer and other chronic diseases.

Get 10% Customization on this Research Report: https://www.marketsandmarkets.com/requestCustomizationNew.asp?id=171955254

North America dominates the Single-cell Analysis Market

The global market is segmented into five major regions, namely, North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa. In 2018, North America accounted for the largest share of the market. The growth in this market can be attributed to the increasing drug development activities in the pharmaceutical and biotechnology industries, rising prevalence of chronic and infectious diseases, and an increase in stem cell research activities.

Prominent players in the Single-cell Analysis Market include Becton, Dickinson and Company (US), Danaher Corporation (US), Merck Millipore (US), QIAGEN (Netherlands), Thermo Fisher Scientific (US), General Electric Company (US), 10x Genomics (US), Promega Corporation (US), Illumina (US), Bio-Rad Laboratories (US), Fluidigm Corporation (US), Agilent Technologies (US), NanoString Technologies (US), Tecan Group (Switzerland), Sartorius AG (Germany), Luminex Corporation (US), Takara Bio (Japan), Fluxion Biosciences (US), Menarini Silicon Biosystems (Italy), and LumaCyte (US).

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Cell Counting Marketby Product (Instruments (Spectrophotometer, Cell Counter, Hemocytometer, Flow Cytometer, Hematology Analyzer), Consumables (Reagent, Assay Kits, Microplate)), End User (Pharmaceutical, Hospital, Research) - Global Forecast to 2023

Cell Isolation/Cell Separation Marketby Product (Reagents, Beads, Centrifuge), Cell Type (Human, Animal), Cell Source (Bone Marrow, Adipose), Technique (Filtration), Application (Cancer), End User (Hospitals, Biotechnology) - Global Forecast to 2024

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Single-cell Analysis Market Worth $5.6 Billion by 2025 - Exclusive Report by MarketsandMarkets - PRNewswire

Bone Marrow Stem Cells Comments Off on Single-cell Analysis Market Worth $5.6 Billion by 2025 – Exclusive Report by MarketsandMarkets – PRNewswire | February 21st, 2020

By keeping an eye on the market conditions and market trends, market research study is initiated depending on clients requirements to form this business document. This Cord Stem Cell Banking market report gives the details about market definition, market drivers, market restraints, market segmentation with respect to product usage and geographical conditions, key developments taking place in the market, competitor analysis, and the research methodology. One of the most noteworthy parts of this Cord Stem Cell Banking Market report is competitor analysis with which businesses can estimate or analyse the strengths and weaknesses of the competitors to gain benefits.

Global Cord stem cell banking market is estimated to reach USD 13.8 billion by 2026 registering a healthy CAGR of 22.4%. The increasing number of parents storing their childs cord blood, acceptance of stem cell therapeutics, high applicability of stem cells are key driver to the market.

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Few of the major market competitors currently working in the globalcord stem cell banking marketareCBR Systems, Inc., Cordlife, Cells4Life Group LLP, Cryo-Cell International, Inc., Cryo-Save AG, Lifecell, StemCyte India Therapeutics Pvt. Ltd, Viacord, SMART CELLS PLUS., Cryoviva India, Global Cord Blood Corporation, National Cord Blood Program, Vita 34, ReeLabs Pvt. Ltd., Regrow Biosciences Pvt. Ltd. , ACROBiosystems., Americord Registry LLC., New York Blood Center, Maze Cord Blood, GoodCell., AABB, Stem Cell Cryobank, New England Cryogenic Center, Inc. among others

Market Definition: Global Cord Stem Cell Banking Market

Cord stem cells banking is nothing but the storing of the cord blood cell contained in the umbilical cord and placenta of a newborn child. This cord blood contains the stem cells which can be used in future to treat disease such as leukemia, thalassemia, autoimmune diseases, and inherited metabolic disorders, and few others.

Segmentation: Global Cord Stem Cell Banking Market

Cord Stem Cell banking Market : By Storage Type

Cord Stem Cell banking Market : By Product Type

Cord Stem Cell banking Market : By Service Type

Cord Stem Cell banking Market : By Indication

Cord Stem Cell banking Market : By Source

Cord Stem Cell banking Market : By Geography

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Key Developments in the Cord Stem Cell banking Market:

Cord Stem Cell banking Market : Drivers

Cord Stem Cell banking Market : Restraint

Competitive Analysis: Global Cord Stem Cell Banking Market

Global cord stem cell banking market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions and others to increase their footprints in this market. The report includes market shares of cord stem cell banking market for Global, Europe, North America, Asia Pacific, South America and Middle East & Africa.

Scope of the Cord Stem Cell banking Market Report :

The report shields the development activities in the Cord Stem Cell banking Market which includes the status of marketing channels available, and an analysis of the regional export and import. It helps in making informed business decisions by having complete insights of market and by making in-depth analysis of market segments. This will benefit the reports users, that evaluates their position in Cord Stem Cell banking market as well as create effective strategies in the near future.

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Cord Stem Cell Banking Market 2020 to Witness Great Growth || Key Players Cryo-Save AG, Lifecell, StemCyte India Therapeutics Pvt. Ltd, Viacord,...

Bone Marrow Stem Cells Comments Off on Cord Stem Cell Banking Market 2020 to Witness Great Growth || Key Players Cryo-Save AG, Lifecell, StemCyte India Therapeutics Pvt. Ltd, Viacord,… | February 21st, 2020

NEW YORK, Feb. 21, 2020 /PRNewswire/ --Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium") announced today findings from the SIERRA trial that were presented at the 2020 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR (TCT) in Orlando, FL. Dr. Boglarka Gyurkocza, the principal investigator from Memorial Sloan Kettering Cancer Center, revealed that there were key differences in side effects reported in patients treated in the Iomab-B and control arms of the study with rates of febrile neutropenia, sepsis and mucositis being markedly lower in the Iomab-B arm.

The oral presentation also featured updated results from the fifty percent enrollment mid-point analysis including BMT access rates, engraftment and 100-day non-relapse transplant related mortality (TRM). After accounting for these factors, the results showed that, on an intent-to-treat or ITT basis, 78 percent of patients in the Iomab-B arm are potentially evaluable for the primary endpoint compared to 13 percent in the control arm. In addition, an important and recent protocol amendment was highlighted; the approximately 30 percent of patients who are expected to fail induction therapy with venetoclax plus hypomethylating agents1 are now eligible for enrollment in the SIERRA trial. The change is expected to increase the addressable patient population of the study given that this combination is now recommended as part of the NCCN or National Comprehensive Cancer Network guidelines, is widely used and expected to become the treatment of choice.

Key Interim Results:

The data tables that follow summarize key findings that were presented as part of the TCT proceedings. As highlighted in the table below, patients receiving Iomab-B showed lower rates of key adverse events relevant in the BMT setting in compared to patients randomized to receive physician's choice of salvage chemotherapy on the control arm. For example, patients receiving Iomab-B had a much lower incidence of sepsis of 3 percent compared to the 42 percent incidence in the control arm.

Adverse Event* N (%)

Randomized to Iomab-B andreceived BMT (N=31)

Randomized to Control Armand received BMT (N=7)

Febrile Neutropenia

8 (25.8)

3 (42.8)

Sepsis/Septic Shock

1 (3.2)

3 (42.8)

Stomatitis (mucositis)

3 (9.7)

2 (28.6)

Pneumonia/Lung Infection

4 (12.9)

1 (14.3)

Hypertension

61 (19.4)

1 (14.3)

Decreased Appetite

5 (16.1)

0 (0.0)

Device related infection

4 (12.9)

1 (14.3)

Hypophosphatemia

2 (6.5)

1 (14.3)

* All adverse events reported irrespective of attribution to protocol-directed procedures

1) 5 patients had hypertension considered unrelated to Iomab-B and 1 patient had hypertension possibly related to Iomab-B

"This detailed safety data from SIERRA is highly encouraging, particularly in this patient population, as neutropenia and sepsis are hallmark toxicities associated with chemotherapy-based conditioning regimens that next to relapse are leading causes of morbidity and mortality post-transplant," said Dr. Vijay Reddy, Vice President, Clinical Development and Head of BMT at Actinium. "Chemotherapy-based conditioning damages normal organs, such as the gastrointestinal tract, which allows gut bacteria to cause serious infections. The data showing less mucositis, febrile neutropenia, and sepsis are consistent with the targeted nature of Iomab-B, since a lack of damage to the gastrointestinal track would lead to a reduction in these adverse events. BMT is the only curable treatment option for older patients with active, relapsed or refractory AML. Yet these patients face restricted access and suboptimal outcomes due to reliance on chemotherapy-based conditioning regimens and perceptions in the hematologist community around safety and eligibility for BMT. With the SIERRA trial, our goals are to eliminate these barriers leading to more patients receiving BMT and with better patient outcomes. With this additional safety data in hand, we have even greater confidence in our ability to change the perceptions around BMT and are excited to update the transplant and hematology communities on Iomab-B's potential to positively impact patients through improved access to BMT and better outcomes."

The presentation highlighted that 100 percent (31/31) of patients receiving a therapeutic dose of Iomab-B achieved successful BMT engraftment with only a 6 percent (2/31) TRM rate compared to the control arm where 18 percent (7/38) achieved engraftment with a 29 percent (2/7) TRM rate. At the 100-day post BMT time point, on an ITT basis, there were 29 patients from the Iomab-B study arm potentially evaluable for the primary endpoint of durable Complete Remission (dCR) at 180 days compared to 5 patients in the control arm. By this measure, 78 percent of patients in the Iomab-B arm are potentially eligible for the dCR primary endpoint compared to 13 percent of patients in the control arm. The mid-point analysis and data presented at TCT can be viewed here.

Detailed engraftment data is presented in the table below:

BMT Feasibility andOutcome Data

Randomized toStudy Arm (N=37)

Randomized to Control Arm (N=38)

ReceivedTherapeutic Doseof Iomab-B andreceived BMT(N=31)1

Achieve CR andreceived standardBMT (N=7)

Did not Achieve CR(N=31/38)2

Crossed over from toIomab-B and receivedBMT (N=20)

BMT Engraftment Rate (%, N)

100% (31/31)

18% (7/38)

100% (20/20)

Median Bone Marrow Blasts% at randomization

(%, range)

29% (5-88)

26% (5-97)

At crossover: 31%

(6-87)

At randomization: 35%(5-75)

Median Days to BMT postrandomization (days, range)

30 (23-50)

67 (51-86)

64 (44-161)

Median Days to AbsoluteNeutrophil Count Engraftment(days, range)

15 (9-22)3

18 (13-82)4

14 (10-37)5

Median Day to PlateletEngraftment (days, range)

20 (4-39)3

22 (9-35)4

19 (13-38)5

100-day non-relapsetransplant related mortality(%, N)

6% (2/31)

29% (2/7)

10% (2/20)6

1) No therapeutic dose (6) due to: declining Karnofsky Performance Scale (PFS) (3), Infusion reaction (1), unfavorablebiodistribution (1), post-randomization eligibility (1)

2) Ineligible for crossover (9) due to: hospice care/progression (4), declined/ineligible for BMT (2), died pre-crossover (3),eligible for crossover (2) did not receive Iomab-B due to declining status

3) Absolute Neutrophil Count engraftment data not available (1), platelet engraftment data not available (4)

4) ANC and platelet engraftment data not available (1), engraftment failure (1)

5) ANC engraftment data not available (1) out of 20, platelet engraftment data not available (3)

6) 1 patient at 161 days had delayed transplant due to infection and respiratory failure, received Iomab-B and BMT when stable

Dr. Mark Berger, Actinium's Chief Medical Officer, stated, "As we approach critical enrollment milestones in the SIERRA trial, our focus turns to bringing Iomab-B to as many patients as possible that can benefit from this product candidate and as expeditiously as possible. Consequently, we made an important amendment to the SIERRA protocol to expand the potential patient pool by including in the eligibility criteria patients who fail induction therapy with venetoclax plus hypomethylating agents. As targeted agents such as venetoclax and others have gained approval, the acute myeloid leukemia treatment landscape has evolved with a significant percentage of patients being treated with these agents in frontline and relapsed settings. In fact, as of mid-2019, venetoclax plus hypomethylating agents have been included as part of the AML National Comprehensive Cancer Network guidelines and medical practice is embracing these regimens widely. However, these regimens are not curative, nor do they eliminate the need for BMT. Indeed, approximately thirty percent of patients fail to achieve a remission after two cycles of induction therapy with venetoclax and most patients ultimately relapse with a median duration of response of less than one year. This amendment has already had a positive impact on the trial that we expect to continue through the remaining portion of enrollment. Most importantly, if Iomab-B gains approval, this amendment will support its use for the significant and growing number of patients receiving and failing venetoclax as induction therapy instead of traditional 7+3 induction chemotherapy. We look forward to continuing to provide key updates as SIERRA reaches key milestones and completes enrollment in 2020."

Sources: 1) DiNardo et al. Venetoclax combined with decitabine or azacitidine in treatment-nave, elderly patients with acute myeloid leukemia. Blood 2019 133(1): 7-17 https://doi.org/10.1182/blood-2018-08-868752

About the SIERRA TrialThe SIERRA trial (Study ofIomab-B inElderlyRelapse/RefractoryAcute Myeloid Leukemia) is the only randomized Phase 3 trial that offers BMT (Bone Marrow Transplant) as an option for older patients with active, relapsed or refractory AML or acute myeloid leukemia. BMT is the only potentially curative treatment option for older patients with active relapsed or refractory AML and there is no standard of care for this indication other than salvage therapies. The SIERRA trial is a 150-patient, multicenter randomized trial that studying Iomab-B compared to physician's choice of salvage chemotherapy. The primary endpoint of the SIERRA trial is durable Complete Remission of 180 days and the secondary endpoint is 1-year overall survival. Iomab-B is an ARC or Antibody Radiation-Conjugate comprised of the anti-CD45 antibody apamistamab and the radioisotope I-131 (Iodine-131). The 20 active SIERRA trial sites in the U.S. and Canada represent many of the leading bone marrow transplant centers by volume. For more information, visit http://www.sierratrial.com.

About Transplantation & Cellular Therapy Meetings (TCT)

TCT, formerly known as the BMT Tandem Meetings, are the combined annual meetings of the American Society for Blood and Marrow Transplantation (ASBMT) and the Center for International Blood & Marrow Transplant Research (CIBMTR).Each year the conference brings together several thousand investigators, clinicians, researchers, nurses and other allied health professionals from over 500 transplant centers from over 50 countries around a full scientific program focused on bone marrow transplant and cellular therapies.

About Actinium Pharmaceuticals, Inc. (NYSE: ATNM)Actinium Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing ARCs or Antibody Radiation-Conjugates, which combine the targeting ability of antibodies with the cell killing ability of radiation. Actinium's lead application for our ARCs is targeted conditioning, which is intended to selectively deplete a patient's disease or cancer cells and certain immune cells prior to a BMT or Bone Marrow Transplant, Gene Therapy or Adoptive Cell Therapy (ACT) such as CAR-T to enable engraftment of these transplanted cells with minimal toxicities. With our ARC approach, we seek to improve patient outcomes and access to these potentially curative treatments by eliminating or reducing the non-targeted chemotherapy that is used for conditioning in standard practice currently. Our lead product candidate, I-131 apamistamab (Iomab-B) is being studied in the ongoing pivotal Phase 3Study ofIomab-B inElderlyRelapsed orRefractoryAcute Myeloid Leukemia (SIERRA) trial for BMT conditioning. The SIERRA trial is over fifty percent enrolled and positive single-agent, feasibility and safety data has been highlighted at ASH, TCT, ASCO and SOHO annual meetings. I-131 apamistamab will also be studied as a targeted conditioning agent in a Phase 1/2 anti-HIV stem cell gene therapy with UC Davis and is expected to be studied with a CAR-T therapy in 2020. In addition, we are developing a multi-disease, multi-target pipeline of clinical-stage ARCs targeting the antigens CD45 and CD33 for targeted conditioning and as a therapeutic either in combination with other therapeutic modalities or as a single agent for patients with a broad range of hematologic malignancies including acute myeloid leukemia, myelodysplastic syndrome and multiple myeloma. Ongoing combination trials include our CD33 alpha ARC, Actimab-A, in combination with the salvage chemotherapy CLAG-M and the Bcl-2 targeted therapy venetoclax. Underpinning our clinical programs is our proprietary AWE (Antibody Warhead Enabling) technology platform. This is where our intellectual property portfolio of over 100 patents, know-how, collective research and expertise in the field are being leveraged to construct and study novel ARCs and ARC combinations to bolster our pipeline for strategic purposes. Our AWE technology platform is currently being utilized in a collaborative research partnership with Astellas Pharma, Inc. Website: https://www.actiniumpharma.com/

Forward-Looking Statements for Actinium Pharmaceuticals, Inc.

This press release may contain projections or other "forward-looking statements" within the meaning of the "safe-harbor" provisions of the private securities litigation reform act of 1995 regarding future events or the future financial performance of the Company which the Company undertakes no obligation to update. These statements are based on management's current expectations and are subject to risks and uncertainties that may cause actual results to differ materially from the anticipated or estimated future results, including the risks and uncertainties associated with preliminary study results varying from final results, estimates of potential markets for drugs under development, clinical trials, actions by the FDA and other governmental agencies, regulatory clearances, responses to regulatory matters, the market demand for and acceptance of Actinium's products and services, performance of clinical research organizations and other risks detailed from time to time in Actinium's filings with the Securities and Exchange Commission (the "SEC"), including without limitation its most recent annual report on form 10-K, subsequent quarterly reports on Forms 10-Q and Forms 8-K, each as amended and supplemented from time to time.

Contacts:

Investors:Hans Vitzthum LifeSci Advisors, LLCHans@LifeSciAdvisors.com (617) 535-7743

Media:Alisa Steinberg, Director, IR & Corp Commsasteinberg@actiniumpharma.com(646) 237-4087

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SOURCE Actinium Pharmaceuticals, Inc.

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Actinium Presents Positive Findings from Mid-Point Analysis of Pivotal SIERRA Trial of Iomab-B at 2020 Transplantation & Cellular Therapy Meetings of...

Bone Marrow Stem Cells Comments Off on Actinium Presents Positive Findings from Mid-Point Analysis of Pivotal SIERRA Trial of Iomab-B at 2020 Transplantation & Cellular Therapy Meetings of… | February 21st, 2020