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CCL7 as a novel inflammatory mediator in cardiovascular disease, diabetes mellitus, and kidney disease - Cardiovascular Diabetology - Cardiovascular...

Cardiac Stem Cells Comments Off on CCL7 as a novel inflammatory mediator in cardiovascular disease, diabetes mellitus, and kidney disease – Cardiovascular Diabetology – Cardiovascular… | September 19th, 2022

Positive Opinion Based on Landmark ZUMA-7 Study in Which 41% of Patients Demonstrated Event-Free Survival at Two Years versus 16% for Standard of Care -

SANTA MONICA, Calif.--(BUSINESS WIRE)--Kite, a Gilead Company (Nasdaq: GILD), today announces that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for Yescarta (axicabtagene ciloleucel) for adult patients with diffuse large B-cell lymphoma (DLBCL) and high-grade B-cell lymphoma (HGBL) that relapses within 12 months from completion of, or is refractory to, first-line chemoimmunotherapy. If approved, Yescarta will be the first Chimeric Antigen Receptor (CAR) T-cell therapy approved for patients in Europe who do not respond to first-line treatment. Although 60% of newly diagnosed LBCL patients will respond to their initial treatment, 40% will relapse or will not respond and need 2nd line treatment.

At Kite, we are committed to bringing the curative potential of cell therapy to the world, and changing the way cancer is treated, said Christi Shaw, CEO, Kite. Todays positive CHMP opinion brings us a step closer to utilizing cell therapy earlier in the treatment journey, potentially transforming the standard of care for the most common and aggressive form of non-Hodgkin lymphoma.

The European Commission will review the CHMP opinion, and a final decision on the marketing authorization is expected in the coming months.

For people with DLBCL and HGBL who do not respond to first-line treatment or have an early relapse, outcomes are often poor and there are limited curative treatment options for these patients, said Marie Jos Kersten, Professor of Hematology at Amsterdam University Medical Centers, Amsterdam. If approved, axicabtagene ciloleucel may offer a new standard of care for patients with relapsed or refractory DLBCL and HGBL. Importantly, in a randomized trial of axicabtagene ciloleucel versus the current standard of care, quality of life also showed greater improvement in the experimental arm.

The positive opinion for Yescarta is based on the primary results of the landmark Phase 3 ZUMA-7 study, the largest and longest trial of a CAR T-cell therapy versus standard of care (SOC) in second-line LBCL. Results demonstrated that at a median follow-up of two years, Yescarta-treated patients had a four-fold greater improvement in the primary endpoint of event-free survival (EFS; hazard ratio 0.40; 95% CI: 0.31-0.51, P<0.001) over the current SOC (8.3 months v 2.0 months). Additionally, Yescarta demonstrated a 2.5 fold increase in patients who were alive at two years without disease progression or need for additional cancer treatment vs SOC (41% v 16%). Improvements in EFS with Yescarta were consistent across key patient subgroups, including elderly patients (HR: 0.28 [95% CI: 0.16-0.46]), primary refractory patients (HR: 0.43 [95% CI: 0.32- 0.57]), high-grade B cell lymphoma including double-hit and triple-hit lymphoma patients (HGBL; HR: 0.28 [95% CI: 0.14-0.59]), and double expressor lymphoma patients (HR: 0.42 [95% CI: 0.27-0.67]).

In a separate, secondary analysis of Patient-Reported Outcomes (PROs) published in Blood patients receiving Yescarta and eligible for the PROs portion of the study (n=165) showed statistically significant improvements in Quality of Life (QoL) at Day 100 compared with those who received SOC (n=131), using a pre-specified analysis for three PRO-domains (EORTC QLQ-C30 Physical Functioning, EORTC QLQ-C30 Global Health Status/QOL, and EQ-5D-5L visual analog scale [VAS]). There was also a trend toward faster recovery to baseline QoL in the Yescarta arm versus SOC.

In the ZUMA-7 trial, Yescarta had a manageable safety profile that was consistent with previous studies. Among the 170 Yescarta-treated patients evaluable for safety, Grade 3 cytokine release syndrome (CRS) and neurologic events were observed in 6% and 21% of patients, respectively. No Grade 5 CRS or neurologic events occurred. In the SOC arm, 83% of patients had high-grade events, mostly cytopenias (low blood counts).

About ZUMA-7

ZUMA-7 is an ongoing, randomized, open-label, global, multicenter (US, Australia, Canada, Europe, Israel) Phase 3 study of 359 patients at 77 centers, evaluating the safety and efficacy of a single-infusion of Yescarta versus current SOC for second-line therapy (platinum-based salvage combination chemotherapy regimen followed by high-dose chemotherapy and autologous stem cell transplant in those who respond to salvage chemotherapy) in adult patients with relapsed or refractory LBCL within 12 months of first-line therapy. The primary endpoint is event free survival (EFS) as determined by blinded central review, and defined as the time from randomization to the earliest date of disease progression per Lugano Classification, commencement of new lymphoma therapy, or death from any cause. Key secondary endpoints include objective response rate (ORR) and overall survival (OS). Additional secondary endpoints include patient reported outcomes (PROs) and safety.

About Yescarta

Yescarta was first approved in Europe in 2018 and is currently indicated for three types of blood cancer: Diffuse Large B-Cell Lymphoma (DLBCL); Primary Mediastinal Large B-Cell Lymphoma (PMBCL); and Follicular Lymphoma (FL). For the full European Prescribing Information, please visit: https://www.ema.europa.eu/en/medicines/human/EPAR/yescarta

Please see full US Prescribing Information, including BOXED WARNING and Medication Guide.

YESCARTA is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of:

U.S. IMPORTANT SAFETY INFORMATION

BOXED WARNING: CYTOKINE RELEASE SYNDROME AND NEUROLOGIC TOXICITIES

CYTOKINE RELEASE SYNDROME (CRS)

CRS, including fatal or life-threatening reactions, occurred. CRS occurred in 90% (379/422) of patients with non-Hodgkin lymphoma (NHL), including Grade 3 in 9%. CRS occurred in 93% (256/276) of patients with large B-cell lymphoma (LBCL), including Grade 3 in 9%. Among patients with LBCL who died after receiving YESCARTA, 4 had ongoing CRS events at the time of death. For patients with LBCL in ZUMA-1, the median time to onset of CRS was 2 days following infusion (range: 1-12 days) and the median duration was 7 days (range: 2-58 days). For patients with LBCL in ZUMA-7, the median time to onset of CRS was 3 days following infusion (range: 1-10 days) and the median duration was 7 days (range: 2-43 days). CRS occurred in 84% (123/146) of patients with indolent non-Hodgkin lymphoma (iNHL) in ZUMA-5, including Grade 3 in 8%. Among patients with iNHL who died after receiving YESCARTA, 1 patient had an ongoing CRS event at the time of death. The median time to onset of CRS was 4 days (range: 1-20 days) and the median duration was 6 days (range: 1-27 days) for patients with iNHL.

Key manifestations of CRS ( 10%) in all patients combined included fever (85%), hypotension (40%), tachycardia (32%), chills (22%), hypoxia (20%), headache (15%), and fatigue (12%). Serious events that may be associated with CRS include cardiac arrhythmias (including atrial fibrillation and ventricular tachycardia), renal insufficiency, cardiac failure, respiratory failure, cardiac arrest, capillary leak syndrome, multi-organ failure, and hemophagocytic lymphohistiocytosis/macrophage activation syndrome.

The impact of tocilizumab and/or corticosteroids on the incidence and severity of CRS was assessed in 2 subsequent cohorts of LBCL patients in ZUMA-1. Among patients who received tocilizumab and/or corticosteroids for ongoing Grade 1 events, CRS occurred in 93% (38/41), including 2% (1/41) with Grade 3 CRS; no patients experienced a Grade 4 or 5 event. The median time to onset of CRS was 2 days (range: 1-8 days) and the median duration of CRS was 7 days (range: 2-16 days). Prophylactic treatment with corticosteroids was administered to a cohort of 39 patients for 3 days beginning on the day of infusion of YESCARTA. Thirty-one of the 39 patients (79%) developed CRS and were managed with tocilizumab and/or therapeutic doses of corticosteroids with no patients developing Grade 3 CRS. The median time to onset of CRS was 5 days (range: 1-15 days) and the median duration of CRS was 4 days (range: 1-10 days). Although there is no known mechanistic explanation, consider the risk and benefits of prophylactic corticosteroids in the context of pre-existing comorbidities for the individual patient and the potential for the risk of Grade 4 and prolonged neurologic toxicities.

Ensure that 2 doses of tocilizumab are available prior to YESCARTA infusion. Monitor patients for signs and symptoms of CRS at least daily for 7 days at the certified healthcare facility, and for 4 weeks thereafter. Counsel patients to seek immediate medical attention should signs or symptoms of CRS occur at any time. At the first sign of CRS, institute treatment with supportive care, tocilizumab, or tocilizumab and corticosteroids as indicated.

NEUROLOGIC TOXICITIES

Neurologic toxicities (including immune effector cell-associated neurotoxicity syndrome) that were fatal or life-threatening occurred. Neurologic toxicities occurred in 78% (330/422) of all patients with NHL receiving YESCARTA, including Grade 3 in 25%. Neurologic toxicities occurred in 87% (94/108) of patients with LBCL in ZUMA-1, including Grade 3 in 31% and in 74% (124/168) of patients in ZUMA-7 including Grade 3 in 25%. The median time to onset was 4 days (range: 1-43 days) and the median duration was 17 days for patients with LBCL in ZUMA-1. The median time to onset for neurologic toxicity was 5 days (range:1- 133 days) and the median duration was 15 days in patients with LBCL in ZUMA-7. Neurologic toxicities occurred in 77% (112/146) of patients with iNHL, including Grade 3 in 21%. The median time to onset was 6 days (range: 1-79 days) and the median duration was 16 days. Ninety-eight percent of all neurologic toxicities in patients with LBCL and 99% of all neurologic toxicities in patients with iNHL occurred within the first 8 weeks of YESCARTA infusion. Neurologic toxicities occurred within the first 7 days of infusion for 87% of affected patients with LBCL and 74% of affected patients with iNHL.

The most common neurologic toxicities ( 10%) in all patients combined included encephalopathy (50%), headache (43%), tremor (29%), dizziness (21%), aphasia (17%), delirium (15%), and insomnia (10%). Prolonged encephalopathy lasting up to 173 days was noted. Serious events, including aphasia, leukoencephalopathy, dysarthria, lethargy, and seizures occurred. Fatal and serious cases of cerebral edema and encephalopathy, including late-onset encephalopathy, have occurred.

The impact of tocilizumab and/or corticosteroids on the incidence and severity of neurologic toxicities was assessed in 2 subsequent cohorts of LBCL patients in ZUMA-1. Among patients who received corticosteroids at the onset of Grade 1 toxicities, neurologic toxicities occurred in 78% (32/41), and 20% (8/41) had Grade 3 neurologic toxicities; no patients experienced a Grade 4 or 5 event. The median time to onset of neurologic toxicities was 6 days (range: 1-93 days) with a median duration of 8 days (range: 1-144 days). Prophylactic treatment with corticosteroids was administered to a cohort of 39 patients for 3 days beginning on the day of infusion of YESCARTA. Of those patients, 85% (33/39) developed neurologic toxicities, 8% (3/39) developed Grade 3, and 5% (2/39) developed Grade 4 neurologic toxicities. The median time to onset of neurologic toxicities was 6 days (range: 1-274 days) with a median duration of 12 days (range: 1-107 days). Prophylactic corticosteroids for management of CRS and neurologic toxicities may result in a higher grade of neurologic toxicities or prolongation of neurologic toxicities, delay the onset of and decrease the duration of CRS.

Monitor patients for signs and symptoms of neurologic toxicities at least daily for 7 days at the certified healthcare facility, and for 4 weeks thereafter, and treat promptly.

REMS

Because of the risk of CRS and neurologic toxicities, YESCARTA is available only through a restricted program called the YESCARTA and TECARTUS REMS Program which requires that: Healthcare facilities that dispense and administer YESCARTA must be enrolled and comply with the REMS requirements and must have on-site, immediate access to a minimum of 2 doses of tocilizumab for each patient for infusion within 2 hours after YESCARTA infusion, if needed for treatment of CRS. Certified healthcare facilities must ensure that healthcare providers who prescribe, dispense, or administer YESCARTA are trained in the management of CRS and neurologic toxicities. Further information is available at http://www.YescartaTecartusREMS.com or 1-844-454-KITE (5483).

HYPERSENSITIVITY REACTIONS

Allergic reactions, including serious hypersensitivity reactions or anaphylaxis, may occur with the infusion of YESCARTA.

SERIOUS INFECTIONS

Severe or life-threatening infections occurred. Infections (all grades) occurred in 45% of patients with NHL; Grade 3 infections occurred in 17% of patients, including Grade 3 infections with an unspecified pathogen in 12%, bacterial infections in 5%, viral infections in 3%, and fungal infections in 1%. YESCARTA should not be administered to patients with clinically significant active systemic infections. Monitor patients for signs and symptoms of infection before and after infusion and treat appropriately. Administer prophylactic antimicrobials according to local guidelines.

Febrile neutropenia was observed in 36% of all patients with NHL and may be concurrent with CRS. In the event of febrile neutropenia, evaluate for infection and manage with broad-spectrum antibiotics, fluids, and other supportive care as medically indicated.

In immunosuppressed patients, including those who have received YESCARTA, life-threatening and fatal opportunistic infections including disseminated fungal infections (e.g., candida sepsis and aspergillus infections) and viral reactivation (e.g., human herpes virus-6 [HHV-6] encephalitis and JC virus progressive multifocal leukoencephalopathy [PML]) have been reported. The possibility of HHV-6 encephalitis and PML should be considered in immunosuppressed patients with neurologic events and appropriate diagnostic evaluations should be performed.

Hepatitis B virus (HBV) reactivation, in some cases resulting in fulminant hepatitis, hepatic failure, and death, can occur in patients treated with drugs directed against B cells, including YESCARTA. Perform screening for HBV, HCV, and HIV in accordance with clinical guidelines before collection of cells for manufacturing.

PROLONGED CYTOPENIAS

Patients may exhibit cytopenias for several weeks following lymphodepleting chemotherapy and YESCARTA infusion. Grade 3 cytopenias not resolved by Day 30 following YESCARTA infusion occurred in 39% of all patients with NHL and included neutropenia (33%), thrombocytopenia (13%), and anemia (8%). Monitor blood counts after infusion.

HYPOGAMMAGLOBULINEMIA

B-cell aplasia and hypogammaglobulinemia can occur. Hypogammaglobulinemia was reported as an adverse reaction in 14% of all patients with NHL. Monitor immunoglobulin levels after treatment and manage using infection precautions, antibiotic prophylaxis, and immunoglobulin replacement. The safety of immunization with live viral vaccines during or following YESCARTA treatment has not been studied. Vaccination with live virus vaccines is not recommended for at least 6 weeks prior to the start of lymphodepleting chemotherapy, during YESCARTA treatment, and until immune recovery following treatment.

SECONDARY MALIGNANCIES

Secondary malignancies may develop. Monitor life-long for secondary malignancies. In the event that one occurs, contact Kite at 1-844-454-KITE (5483) to obtain instructions on patient samples to collect for testing.

EFFECTS ON ABILITY TO DRIVE AND USE MACHINES

Due to the potential for neurologic events, including altered mental status or seizures, patients are at risk for altered or decreased consciousness or coordination in the 8 weeks following YESCARTA infusion. Advise patients to refrain from driving and engaging in hazardous occupations or activities, such as operating heavy or potentially dangerous machinery, during this initial period.

ADVERSE REACTIONS

The most common non-laboratory adverse reactions (incidence 20%) in patients with LBCL in ZUMA-7 included fever, CRS, fatigue, hypotension, encephalopathy, tachycardia, diarrhea, headache, musculoskeletal pain, nausea, febrile neutropenia, chills, cough, infection with an unspecified pathogen, dizziness, tremor, decreased appetite, edema, hypoxia, abdominal pain, aphasia, constipation, and vomiting.

The most common adverse reactions (incidence 20%) in patients with LBCL in ZUMA-1 included CRS, fever, hypotension, encephalopathy, tachycardia, fatigue, headache, decreased appetite, chills, diarrhea, febrile neutropenia, infections with an unspecified, nausea, hypoxia, tremor, cough, vomiting, dizziness, constipation, and cardiac arrhythmias.

The most common non-laboratory adverse reactions (incidence 20%) in patients with iNHL in ZUMA-5 included fever, CRS, hypotension, encephalopathy, fatigue, headache, infections with an unspecified, tachycardia, febrile neutropenia, musculoskeletal pain, nausea, tremor, chills, diarrhea, constipation, decreased appetite, cough, vomiting, hypoxia, arrhythmia, and dizziness.

About Kite

Kite, a Gilead Company, is a global biopharmaceutical company based in Santa Monica, California, with manufacturing operations in North America and Europe. Kites singular focus is cell therapy to treat and potentially cure cancer. As the cell therapy leader, Kite has more approved CAR T indications to help more patients than any other company. For more information on Kite, please visit http://www.kitepharma.com. Follow Kite on social media on Twitter (@KitePharma) and LinkedIn.

About Gilead Sciences

Gilead Sciences, Inc. is a biopharmaceutical company that has pursued and achieved breakthroughs in medicine for more than three decades, with the goal of creating a healthier world for all people. The company is committed to advancing innovative medicines to prevent and treat life-threatening diseases, including HIV, viral hepatitis and cancer. Gilead operates in more than 35 countries worldwide, with headquarters in Foster City, California.

Forward-Looking Statements

This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other factors, including the ability of Gilead and Kite to initiate, progress or complete clinical trials within currently anticipated timelines or at all, and the possibility of unfavorable results from ongoing and additional clinical trials, including those involving Yescarta; uncertainties relating to regulatory applications and related filing and approval timelines, including the risk that the European Commission may not grant marketing authorization for Yescarta for use in second-line DLBCL and HGBL in a timely manner or at all; the risk that any regulatory approvals, if granted, may be subject to significant limitations on use; the risk that physicians may not see the benefits of prescribing Yescarta for the treatment of LBCL; and any assumptions underlying any of the foregoing. These and other risks, uncertainties and other factors are described in detail in Gileads Quarterly Report on Form 10-Q for the quarter ended June 30, 2022 as filed with the U.S. Securities and Exchange Commission. These risks, uncertainties and other factors could cause actual results to differ materially from those referred to in the forward-looking statements. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. The reader is cautioned that any such forward-looking statements are not guarantees of future performance and involve risks and uncertainties and is cautioned not to place undue reliance on these forward-looking statements. All forward-looking statements are based on information currently available to Gilead and Kite, and Gilead and Kite assume no obligation and disclaim any intent to update any such forward-looking statements.

U.S. Prescribing Information for Yescarta including BOXED WARNING, is available at http://www.kitepharma.com and http://www.gilead.com .

Kite, the Kite logo, Yescarta and GILEAD are trademarks of Gilead Sciences, Inc. or its related companies .

View source version on businesswire.com: https://www.businesswire.com/news/home/20220916005209/en/

Jacquie Ross, Investorsinvestor_relations@gilead.com

Anna Padula, Mediaapadula@kitepharma.com

Source: Gilead Sciences, Inc.

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IMAC Holdings, Inc.

BRENTWOOD, Tenn., Sept. 09, 2022 (GLOBE NEWSWIRE) -- IMAC Holdings, Inc. (Nasdaq: BACK) (IMAC or the Company), today announces it has completed the third cohort of its Phase 1 clinical trial for its investigational compound utilizing umbilical cord-derived allogenic mesenchymal stem cells for the treatment of bradykinesia due to Parkinsons disease.

The third cohort consists of five patients with bradykinesia due to Parkinsons disease receiving an intravenous infusion of a high concentration stem cell treatment. The third and final cohort of the Phase 1 clinical trial was completed on Tuesday, September 6, 2022.

About IMACs Phase 1 Clinical Trial

The Phase 1 clinical trial, consisting of a 15-patient dose escalation safety and tolerability study, is being conducted at three of IMACs clinical centers in Chesterfield, Missouri, Paducah, Kentucky, and Brentwood, Tennessee. The trial is divided into three groups: 1) five patients with bradykinesia due to Parkinsons disease received a low concentration dose, intravenous infusion of stem cells, 2) five received a medium concentration intravenous dose, 3) and five received a high concentration intravenous dose. All groups will be subsequently tracked for 12 months. IMACs medical doctors and physical therapists at the clinical sites have been trained to administer the treatment and manage the therapy. Ricardo Knight, M.D., M.B.A., who is medical director of the IMAC Regeneration Center of Chicago, is the trials principal investigator.

The Institute of Regenerative and Cellular Medicine serves as the trials independent investigational review board, while Regenerative Outcomes provides management of the study. Further details of the trial can be found at clinicaltrials.gov.

About Bradykinesia Due to Parkinsons Disease

In addition to unusually slow movements and reflexes, bradykinesia may lead to limited ability to lift arms and legs, reduced facial expressions, rigid muscle tone, a shuffling walk, and difficulty with repetitive motion tasks, self-care, and daily activities. Parkinsons disease is the typical culprit of bradykinesia, and as it progresses through its stages, a persons ability to move and respond declines.

Story continues

According to Zion Market Research, the global Parkinsons disease therapeutics market was $2.61 billion in 2018 and is expected to grow to $5.28 billion by 2025. The Parkinsons Disease Foundation estimates that nearly 10 million people are suffering from Parkinsons disease, and almost 60,000 new cases are reported annually in the U.S.

About IMAC Holdings, Inc.

IMAC Holdingsowns and manages health and wellness centers that deliver sports medicine, orthopedic care, and restorative joint and tissue therapies for movement restricting pain and neurodegenerative diseases.IMACis comprised of three business segments: outpatient medical centers, The Back Space, and a clinical research division. With treatments to address both young and aging populations,IMAC Holdingsowns or manages outpatient medical clinics that deliver regenerative rehabilitation services as a minimally invasive approach to acute and chronic musculoskeletal and neurological health problems. IMACs The Back Company retail spinal health and wellness treatment centers deliver chiropractic care within Walmart locations. IMACs research division is currently conducting a Phase I clinical trial evaluating a mesenchymal stem cell therapy candidate for bradykinesia due to Parkinsons disease. For more information visitwww.imacholdings.com.

# # #

Safe Harbor Statement

This press release contains forward-looking statements. These forward-looking statements, and terms such as anticipate, expect, believe, may, will, should or other comparable terms, are based largely on IMAC's expectations and are subject to a number of risks and uncertainties, certain of which are beyond IMAC's control. Actual results could differ materially from these forward-looking statements as a result of, among other factors, risks and uncertainties associated with its ability to raise additional funding, its ability to maintain and grow its business, variability of operating results, its ability to maintain and enhance its brand, its development and introduction of new products and services, the successful integration of acquired companies, technologies and assets, marketing and other business development initiatives, competition in the industry, general government regulation, economic conditions, dependence on key personnel, the ability to attract, hire and retain personnel who possess the skills and experience necessary to meet customers requirements, and its ability to protect its intellectual property. IMAC encourages you to review other factors that may affect its future results in its registration statement and in its other filings with the Securities and Exchange Commission. In light of these risks and uncertainties, there can be no assurance that the forward-looking information contained in this press release will in fact occur.

IMAC Press Contact:

Laura Fristoe

lfristoe@imacrc.com

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IMAC Holdings, Inc. Announces Completion of Third Cohort of its Phase 1 Clinical Study of Umbilical Cord-Derived Mesenchymal Stem Cells for the...

Spinal Cord Stem Cells Comments Off on IMAC Holdings, Inc. Announces Completion of Third Cohort of its Phase 1 Clinical Study of Umbilical Cord-Derived Mesenchymal Stem Cells for the… | September 11th, 2022

A person with SMA may find it challenging to stand up, walk, control their head movements, and in some cases, even breathe and swallow

Spine. Image courtesy Pearson Scott Foresman/Wikimedia Commons

Spinal muscular atrophy (SMA) is a severe genetic condition that targets motor neurons in the central nervous system (CNS), resulting in progressive muscle atrophy, weakness, and paralysis. It is a group of genetic disorders in which a person cannot control the movement of their muscles due to a loss of nerve cells in the spinal cord and brain stem. A person with SMA may find it challenging to stand up, walk, control their head movements, and in some cases, even breathe and swallow. Some forms of SMA are present at birth, while others develop over time. Some have an impact on life expectancy.

SMA can be clinically divided into five subtypes. The most severe type is SMA type 0, appearbefore birth, can be fatal before or after birth within the first year of life. Type 1 SMA also called infantile-onset, is the most common type of SMA, accounting for 60% cases, which appears in infants and causes them to die or become dependent on a ventilator by the age of two. Children with SMA type 2 are sitters, while those with type 3 can walk on their own for a while before becoming wheelchair-bound. SMA type 4 develops in adults and causes later-life progressive weakness.

SMA is the most frequent cause of death in the infantile age group, occurring in one in 10,000 live births. However, the SMA carrier frequency was 1 in 38 in a recent Indian study. Children with SMA can currently receive supportive care in India that includes assisted ventilation, feeding, physiotherapy, orthotics, and spine stabilization.

What causes SMA?

SMA is caused by a very specific genetic mutation in a gene called theSMN1 gene. SMN is that protein that play a critical role in the survival of the nerve cells that control muscles. (SMN) protein keeps motor neurons healthy and functioning normally. The loss of motor neurons in the spinal cord caused by SMA patients, and insufficient levels of the SMN protein results in skeletal muscle weakness and wasting.

SMA patients gradually lose their ability to control their muscles movement and strength. The muscles closest to the torso and neck are frequently severely affected by the disease. Some SMA patients never sit, stand, or walk. Other signs of SMA include tongue fasciculation, a bell-shaped chest (caused by muscle weakness), weak cough, difficulty breathing , choking or trouble swallowing, weak sucking and labored breathing during feeding.

How is SMA diagnosed?

The diagnosis of spinal muscular atrophy depends on the type of SMA a person has and age of onset. The path to diagnosis for infants and children with more severe forms of SMA frequently starts when a parent or medical professional notices unusual muscle weakness (hypotonia). People with adult-onset SMA types, such as type 4, might begin the diagnosis process after observing minor symptoms like hand tremors.

Physical exam

A physical examination is required to identify the presence of symptoms like muscle weakness or a lack of reflexes in cases where a new-born is not screened for SMA at birth. A primary care physician or a neurologist could perform this.

Family medical history

As part of your or your childs physical examination, a thorough review of the patients family history is necessary to determine whether there have ever been any instances of neuromuscular disease in the family. If the physical examination and family history raise suspicion of SMA, genetic testing will likely be the next step.

Genetic testing

Through molecular genetic testing, which requires a blood sample, SMA is identified. A single gene is examined for mutations linked to a genetic disease in molecular genetic testing.

Importance of early diagnosis

A patient with SMA must first undergo a higher level of cognitive evaluation. The clinician should assess the patient for weakness before concentrating solely on SMA. A muscle biopsy could be the next step in the evaluation to more precisely distinguish between muscle weakness and nerve weakness. Finally, the clinician would probably identify this patients SMA based on the results of the combined muscle biopsy and electrode diagnostics.

If a diagnosis is made early, the individual has access to the tools and the resources that medical science has developed over the last number of years to assist optimal functioning.

The standard method for diagnosing SMA is molecular genetic testing. SMA should be given early consideration in any infant with weakness or hypotonia due to the effectiveness of molecular testing and high frequency of SMA in the hypotonic infant. All other infant causes of hypotonic weakness are included in the differential diagnosis of severe forms of SMA.

SMA is inherited in an autosomal recessive manner. Each pregnancy of a couple who have had a child with SMA has an approximately 25 per cent chance of producing an affected child. Carrier testing for at-risk relatives and prenatal testing for pregnancies at increased risk are possible if the diagnosis of SMA has been confirmed by molecular genetic testing in an affected family member.

Currently, there are several SMA treatments that have received FDA approval including Risdiplam (Evrysdi), Onasemnogene abeparvovec-xioi (Zolgensma) and Nusinersen (Spinraza). These targeted treatments may prevent the development or slow the progression of some features of SMA.

The severity of the disease varies depending on the type of SMA, with more severe subtypes needing more aggressive treatment. Proactive care and treatment decision-making by the multidisciplinary team and family are of paramount importance.

The author is MBBS, DCH, MRCPCH, Fellowship Pediatric Genetics, Consultant Clinical Geneticist, Salem Genetics Centre. Views are personal.

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Spinal Muscular Atrophy: Causes and importance of early diagnosis for proactive management - Firstpost

Spinal Cord Stem Cells Comments Off on Spinal Muscular Atrophy: Causes and importance of early diagnosis for proactive management – Firstpost | September 11th, 2022

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Clinical translation of stem cell therapy for spinal cord injury still premature: results from a single-arm meta-analysis based on 62 clinical trials...

IPS Cell Therapy Comments Off on Clinical translation of stem cell therapy for spinal cord injury still premature: results from a single-arm meta-analysis based on 62 clinical trials… | September 11th, 2022

DUBLIN--(BUSINESS WIRE)--The "Induced Pluripotent Stem Cells Market - Growth, Trends, Covid-19 Impact, and Forecasts (2022 - 2027)" report has been added to ResearchAndMarkets.com's offering.

The Induced Pluripotent Stem Cells Market is projected to register a CAGR of 8.4% during the forecast period (2022 to 2027).

Companies Mentioned

Key Market Trends

The Drug Development Segment is Expected to Hold a Major Market Share in the Induced Pluripotent Stem Cells Market.

By application, the drug development segment holds the major segment in the induced pluripotent stem cell market. Various research studies focusing on drug development studies with induced pluripotent stem cells have been on the rise in recent years.

For instance, an article titled "Drug Development and the Use of Induced Pluripotent Stem Cell-Derived Cardiomyocytes for Disease Modeling and Drug Toxicity Screening" published in the International Journal of Molecular Science in October 2020 discussed the broad use of iPSC derived cardiomyocytes for drug development in terms of adverse drug reactions, mechanisms of cardiotoxicity, and the need for efficient drug screening protocols.

Another article published in the Journal of Cells in December 2021 titled "Human Induced Pluripotent Stem Cell as a Disease Modeling and Drug Development Platform-A Cardiac Perspective" focused on methods to reprogram somatic cells into human induced pluripotent stem cells and the solutions to overcome the immaturity of the human induced pluripotent stem cells derived cardiomyocytes to mimic the structure and physiological properties of adult human cardiomyocytes to accurately model disease and test drug safety. Thus, this increase in the research of induced pluripotent stem cells for drug development and drug modeling is likely to propel the segment's growth over the study period.

Furthermore, as per an article titled "Advancements in Disease Modeling and Drug Discovery Using iPSC-Derived Hepatocyte-like Cells" published in the Multi-Disciplinary Publishing Institute journal of Cells in March 2022, preserved differentiation and physiological function, amenability to genetic manipulation via tools such as CRISPR/Cas9, and availability for high-throughput screening, make induced pluripotent stem cell systems increasingly attractive for both mechanistic studies of disease and the identification of novel therapeutics.

North America is Expected to Hold a Significant Share in the Market and Expected to do Same in the Forecast Period

The rise in the adoption of highly advanced technologies and systems in drug development, toxicity testing, and disease modeling coupled with the growing acceptance of stem cell therapies in the region are some of the major factors driving the market growth in North America.

The United States Food and Drug Administration in March 2022 discussed the development of strategies to improve cell therapy product characterization. The agency focused on the development of improved methods for testing stem cell products to ensure the safety and efficacy of such treatments when used as therapies.

Likewise, in March 2020, the Food and Drug Administration announced that ImStem drug IMS001, which uses AgeX's pluripotent stem cell technology, would be available for the treatment of multiple sclerosis. Similarly, REPROCELL introduced a customized iPSC generation service in December 2020, as well as a new B2C website to promote the "Personal iPS" service. This service prepares and stores an individual's iPSCs for future injury or disease regeneration treatment.

Thus, the increasing necessity for induced pluripotent stem cells coupled with increasing investment in the health care department is known to propel the growth of the market in this region.

Key Topics Covered:

1 INTRODUCTION

2 RESEARCH METHODOLOGY

3 EXECUTIVE SUMMARY

4 MARKET DYNAMICS

4.1 Market Overview

4.2 Market Drivers

4.2.1 Increase in Research and Development Activities in Stem Cells Therapies

4.2.2 Surge in Adoption of Personalized Medicine

4.3 Market Restraints

4.3.1 Lack of Awareness Regarding Stem Cell Therapies

4.3.2 High Cost of Treatment

4.4 Porter's Five Force Analysis

5 MARKET SEGMENTATION

5.1 By Derived Cell Type

5.2 Application

5.3 End User

5.4 Geography

6 COMPETITIVE LANDSCAPE

6.1 Company Profiles

7 MARKET OPPORTUNITIES AND FUTURE TRENDS

For more information about this report visit https://www.researchandmarkets.com/r/ylzwhr

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Global Induced Pluripotent Stem Cells Market (2022 to 2027) - Growth, Trends, Covid-19 Impact and Forecasts - ResearchAndMarkets.com - Business Wire

IPS Cell Therapy Comments Off on Global Induced Pluripotent Stem Cells Market (2022 to 2027) – Growth, Trends, Covid-19 Impact and Forecasts – ResearchAndMarkets.com – Business Wire | September 11th, 2022

MELBOURNE, Australia--(BUSINESS WIRE)--Propanc Biopharma, Inc. (OTC Pink: PPCB) (Propanc or the Company), a biopharmaceutical company developing novel cancer treatments for patients suffering from recurring and metastatic cancer, today announced significant effects of PRP against the tumor microenvironment and pre-metastatic niche has been reported by the Companys joint researcher, Mrs. Beln Toledo Cutillas MSc, at the laboratory of Professor Macarena Pern, PhD, University of Jan. Treatment with PRP was shown to have a favorable impact inhibiting, slowing, or reversing tumor development by acting as an anti-tumor agent, decreasing tumor cell proliferation, developing a non-malignant phenotype (observable characteristics) and promoting cell adhesion (sticking close to one another) and differentiation (cell specialization rather than stem cell like). It was concluded that PRP could have a significant impact on the tumor microenvironment as a potential clinical application. PRP is a combination of the two proenzymes trypsinogen and chymotrypsinogen.

Cancer remains one of the leading causes of death, globally. Despite recent advances in understanding its molecular and genetic basis, more than one third of those affected die each year from cancer. These alarming results are mainly attributed to current therapies not fully effective against cancer cells which may develop drug resistance, leading to recurrence and metastasis, causing more than 90% of cancer-related deaths. According to Mrs. Cutillas, This is why we need to find better and more effective therapeutic strategies. She explains that tumor formation is influenced by two factors, genetic changes in tumor cells and the rearrangement of components of the tumor microenvironment. In recent years, cancer research has focused on the tumor microenvironment.

Numerous assays, in vitro and in vivo studies, were conducted by Mrs. Cutillas confirming that PRP appears to have an anti-tumor effect and can act selectively against specific tumor elements, without affecting the non-tumor microenvironment and preventing its malignification (i.e., the process of making malignant).

Dr Julian Kenyon, MD, MB, ChB, Propancs Chief Scientific Officer said, The work undertaken by Mrs Cutillas highlights the significant potential applications of PRP in a clinical setting, specifically relating to drug resistance, and consequently recurrence and metastasis, which is the biggest cause of death for sufferers. The pioneering research being undertaken with our joint researchers at the Universities of Jan and Granada, continues to confirm our belief in the therapeutic potential of PRP, and may lead to exciting new ways to treat cancer patients suffering from solid tumors whilst reducing the threat of recurrence.

PRP is a mixture of two proenzymes, trypsinogen and chymotrypsinogen from bovine pancreas administered by intravenous injection. A synergistic ratio of 1:6 inhibits growth of most tumor cells. Examples include kidney, ovarian, breast, brain, prostate, colorectal, lung, liver, uterine and skin cancers.

About Propanc Biopharma, Inc.

Propanc Biopharma, Inc. (the Company) is developing a novel approach to prevent recurrence and metastasis of solid tumors by using pancreatic proenzymes that target and eradicate cancer stem cells in patients suffering from pancreatic, ovarian and colorectal cancers. For more information, please visit http://www.propanc.com.

The Companys novel proenzyme therapy is based on the science that enzymes stimulate biological reactions in the body, especially enzymes secreted by the pancreas. These pancreatic enzymes could represent the bodys primary defense against cancer.

To view the Companys Mechanism of Action video on its anti-cancer lead product candidate, PRP, please click on the following link: http://www.propanc.com/news-media/video

Forward-Looking Statements

All statements other than statements of historical facts contained in this press release are forward-looking statements, which may often, but not always, be identified by the use of such words as may, might, will, will likely result, would, should, estimate, plan, project, forecast, intend, expect, anticipate, believe, seek, continue, target or the negative of such terms or other similar expressions. These statements involve known and unknown risks, uncertainties and other factors, which may cause actual results, performance or achievements to differ materially from those expressed or implied by such statements. These factors include uncertainties as to the Companys ability to continue as a going concern absent new debt or equity financings; the Companys current reliance on substantial debt financing that it is unable to repay in cash; the Companys ability to successfully remediate material weaknesses in its internal controls; the Companys ability to reach research and development milestones as planned and within proposed budgets; the Companys ability to control costs; the Companys ability to obtain adequate new financing on reasonable terms; the Companys ability to successfully initiate and complete clinical trials and its ability to successful develop PRP, its lead product candidate; the Companys ability to obtain and maintain patent protection; the Companys ability to recruit employees and directors with accounting and finance expertise; the Companys dependence on third parties for services; the Companys dependence on key executives; the impact of government regulations, including FDA regulations; the impact of any future litigation; the availability of capital; changes in economic conditions, competition; and other risks, including, but not limited to, those described in the Companys periodic reports that are filed with the Securities and Exchange Commission and available on its website at http://www.sec.gov. These forward-looking statements speak only as of the date hereof and the Company disclaims any obligations to update these statements except as may be required by law.

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Propanc Biopharma Reports Significant Effects of PRP Against the Tumor Microenvironment - Business Wire

Skin Stem Cells Comments Off on Propanc Biopharma Reports Significant Effects of PRP Against the Tumor Microenvironment – Business Wire | September 11th, 2022

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Epidemiology of early infections and predictors of mortality after autologous hematopoietic stem-cell transplantation among multiple myeloma, Hodgkin,...

Bone Marrow Stem Cells Comments Off on Epidemiology of early infections and predictors of mortality after autologous hematopoietic stem-cell transplantation among multiple myeloma, Hodgkin,… | September 11th, 2022

September is observed as Blood Cancer Awareness month all over the world. During this month, activists and stakeholders work to raise awareness about the disease and the efforts being taken to fight blood cancers including leukemia, lymphoma, myeloma and Hodgkins disease.

The term blood cancer is a general description of various hematopoietic cancers. Our blood flows through blood vessels to supply all tissues in the body with nutrients. In the approximately 5 litres of blood circulating in our body there are billions of blood cells that carry out various vital functions. All blood cells originate from hematopoietic stem cells.

Haematopoietic stem cells are known as mother cells and do not yet have a specific function. They are able to renew and differentiate into cells with a specific function, thus replacing cells that die. In bone marrow, blood stem cells divide and develop into progenitor cells. Through further division, the progenitor cells mature and transform into different types of blood cells and then enter the bloodstream, says Dr Nitin Agarwal, HOD, Donor Request Management, DKMS BMST Foundation India.

Blood cancer is an abnormal proliferation (abnormal growth) of cells in the bone marrow especially white blood cells (WBCs). Cancer cells flood the bloodstream and drive out healthy cells. As a result, the blood can no longer perform its basic tasks, such as transporting oxygen and protecting the body from infection.

LeukemiaThis cancer is found in the bone marrow and the bloodstream. It is caused by abnormal rapid production of WBCs and high number of abnormal WBCs which cannot fight against infection, and they impair the bone marrows ability to produce red blood cells and platelets, says Dr Jimmy Mirani, Consultant Onco Surgeon, Wockhardt Hospital, Mumbai Central.

LymphomaA type of blood cancer which affects the lymphatic system, which removes the risk excess fluids from body and generates immune cells. Lymphocytes are blood cells which are used to fight against infections. These abnormal lymphocytes become lymphoma cells which multiply and get collected in the tissues, adds Dr Mirani.

There are two types of lymphoma, namely, Hodgkins lymphoma and non-Hodgkins lymphoma.

Non-Hodgkins lymphoma:It mainly impacts the B-cell or T-cell. This type of lymphoma occurs more commonly than Hodgkins lymphoma. Can vary clinically and diagnostically into slow-growing ones to very aggressive types, notes Dr. Amrita Chakrabarti, Consultant, Haemato-Oncology & Bone Marrow Transplant, Max Hospital, Shalimar Bagh.

Hodgkins lymphoma This type of lymphoma affects the B cells. Broadly divided into classical Hodgkins and nodular lymphocyte predominant types. Occurs in the adolescence or elderly age group.

MyelomaIt is the cancer of plasma cells; WBCs which produce disease and infection fighting anti-bodies. Myeloma cells prevent the functions and productions of these antibodies leaving a week immune system.

Multiple myelomaThis starts in the bone marrow when plasma cells begin to grow uncontrollably. As the cells grow, they compromise the immune system and impair the production and function of white and red blood cells causing bone disease, organ damage and anemia among other conditions, adds Dr Agarwal.

In most cases of blood cancer, the patient feels tired and weak. This happens because the number of red blood cells in the blood starts decreasing due to which there is a lack of blood in the person. Someof the commonsymptoms of blood cancers are fever, severe fatigue, bleeding from gums or skin, back ache, or bone pains, says Dr Pravas Mishra, Head Haematology/ Medical Oncology and BMT, Amrita Hospital, Faridabad.

Patients with myeloma might first present to an orthopaedical with a fracture originating from trivial trauma or to a nephrologist with a kidney dysfunction.Pain in bones and joints can be a symptom of not only arthritis but also blood cancer. Blood cancer is a disease in the bone marrow that is found in large amounts around the bones and joints.

Patientsmight present with nodes in the neck or axilla or groin or swelling in any part of the body. However most often a patientwith blood cancermight present with just a low haemoglobin. It is strongly advised not to ignore any anaemia, warns Dr Mishra.

A person suffering from blood cancer is prone to repeated infections. When leukemia cells develop in the body, then complaints of infection can be seen in the patients mouth, throat, skin, lungs, etc.

People who have cancer tend to have an abnormally low weight. If the body weight is reduced without any obvious cause, then it can be seen as the primary symptom of cancer.

The abnormal formation of leukemia cells in the body prevents the bone marrow from forming healthy blood cells such as platelets. Due to its deficiency, more bleeding problems can be seen from the nose of the patient, during menstruation and gums.

Blood cancer is diagnosed with the help of a wide range of diagnostic methods along clinical evaluation, such as blood tests, bone marrow tests, cytogenetic/karyotyping and molecular analysis, flow cytometry.

Myth: Blood cancer cannot be treated?

Fact: Once a patient is diagnosed with blood cancer, the first concern that comes to ones mind Is blood cancer curable?

Blood cancer is one type of cancer that has a high curability rate especially due to the advancement in the medical field, availability of newer, improved chemotherapy regimens, targeted therapy, and improved infection control measures. Timely diagnosis, especially early diagnosis, increases the chances of cure from blood cancer.Some of the other factors that impact the cure of blood cancer include the age of the patient, physical condition, presence of other comorbidities, stage of the disease, subtype of cancer, molecular factors, whether low grade/high grade, acute or chronic, the body parts that are affected and whether the disease is new onset or has come back after a previous cure.

You must understand that the cure or recovery from cancer is unpredictable, adds Dr. Chakrabarti.

There are cases when the patient has recovered even in the later stages of blood cancer. On the other hand, there are recorded cases where the patient couldnt recover even in the initial stages of blood cancer. So, its important to have realistic expectations and focus on following a healthy lifestyle with the advised treatment and measures. Early diagnosis and treatment play an important role in attaining cure.

Myth: All blood cancer patients need a bone marrow transplant

Fact: No, majority of patients suffering from blood cancers are treated without bone marrow transplant. A combination of chemotherapy, targeted therapy and immunotherapy is the best line of treatment.

Myth: Blood cancer occurs only in children?

Fact: No, blood cancers can occur in all age groups. All have a higher incidence in young children whereas Myeloid Leukaemia (MLL) is more frequently seen in senior citizens.

India is reeling under pressure of many misconceptions that exist amongst people about blood stem cell donation, its process and even its after-effects.

Myth: Once you donate blood stem cells, you will lose them forever.

Fact: Only a fraction of total stem cells is extracted during the process. Also, all the cells are naturally replenished within a few weeks

Myth: Donating stem cells is a really invasive and painful process

Fact: Blood stem cells are collected through peripheral blood stem cell collection (PBSC) which is completely safe and a non-surgical procedure. The process is similar to blood platelet donation that takes approximately three to four hours to complete and the donor can leave the collection center the same day.

Myth: Blood donation and a blood stem cell donation are same

Fact: Unlike blood collection for transfusion, blood stem cells are collected only when there is a match between the donor and patients human leukocyte antigen (HLA) combination (tissue type). So, you could be potentially the only match and life saver for a person with blood cancer in need of a transplant, adds Dr Nitin Agarwal. Blood stem cell donors donate only blood stem cells and the process is similar to a platelet donation.

Myth: Pregnant women cant register

Fact: This is untrue, a woman can register even during her pregnancy.

Myth: Stem cell donation leaves prolonged side-effects

Fact: No, there are no major side effects post blood stem cell donation. A person may only experience minor flu like symptoms because of the GCSF injections given to him/her before the donation, to mobilize blood stem cells in your blood stream.

Myth: Piercing and/or tattoo is a restricting factor

Fact: Piercing or a tattoo doesnt stop you from registering yourself to be a potential donor.

Myth: My blood stem cells can be stored

Fact: Your blood stem cells will not be stored. They last for around 72 hours and are delivered for the recipient straight to the hospital by a special courier. If the recipients body accepts them, the stem cells will start making healthy blood cells.

Myth: Joining a blood stem cell registry is no use. Most patients can find a stem cell donor within their own families

Fact: Per statistics, only 30% of blood disorder patients in need of a stem cell transplant are able to find a sibling match. About 70% of patients need an unrelated donor.

A registry like DKMS BMST Foundation India is a data bank of potential blood stem cell donors that houses details on thousands of committed blood stem cell donors. Any patient can benefit from this registry provided an HLA match.

Some of the blood cancer treatments include the following

Chemotherapy

This is the most important aspect of blood cancer treatment and involves using certain chemicals to kill the cancer-causing cells in the patients body. The prescribed drugs are given in a particular timeframe for the best possible improvement in the patients health. In some patients, a stem cell transplant is provided along with high dose chemotherapy.

Radiation therapyRadiation therapy helps to destroy cancer cells with the help of specific high-energy beams to kill cancer cells in precise areas of the body. This treatment is much beneficial for patients with lymphoma

Bone marrow transplantIn this procedure, healthy stem cells are utilized to replace the cells affected by cancer. This helps the patients recover in the best possible manner. Can be autologous (where stem cells are taken from the patients own body) or allogenic (when a healthy donor gives stem cells to the patient.)

Targeted Therapy

Usually in the form of oral medications or pills. They are given alongside chemotherapy/ or radiotherapy and affect specific cancer cells and help in destroying them.

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September is Blood Cancer Awareness Month: All You Need to Know - News18

Bone Marrow Stem Cells Comments Off on September is Blood Cancer Awareness Month: All You Need to Know – News18 | September 11th, 2022

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Brave Aubrey Austin, four, donated her own stem cells and saved her baby brother Carey's life on the day he turned one, after he was diagnosed with a rare type of blood cancer aged just eight months

Image: Supplied via Lucy Laing)

A brave little girl saved the life of her baby brother on his first birthday.

Carey Austin was diagnosed with a rare type of blood cancer when he was just eight months old.

His only hope of survival was a stem-cell transplant.

Against all odds, his sister Aubrey, four, was a perfect match.

Surgeons operated on Careys first birthday and six months later he is cancer-free thanks to his big sister.

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Their mum Naomi said: She absolutely adores Carey and when we explained to her about the transplant she wanted to do everything she could to save him.

Shes only four years old, yet she was only thinking of how she could help him. We felt so guilty putting her through an operation too, but it was Careys only chance of survival.

"She was so brave about it. She knew that her blood was going to save him.

During a two-hour procedure at Great Ormond Street Hospital, London, surgeons took out Aubreys stem cells and they were put into Careys body via a drip.

Naomi said: The fact that the transplant took place on Careys birthday was so significant that she was giving him a second chance at life on that special day.

The doctors and nurses said they had never seen anyone have a stem cell transplant on their birthday before.

Aubrey was very groggy and woozy when she came around from the operation, and she had puncture wounds on her back from where the stem cells had been taken out.

But she was still smiling through it all. She was so brave. She never complained about being in pain and she was just pleased to see how her little brother was afterwards.

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When the brother and sister saw each other for the first time after the operation, there was not a dry eye in the room.

Naomi said: It was so sweet when they were reunited.

We took Aubrey to see Carey and she gave him a cuddle. They were thrilled to see each other again.

After a two-day hospital stay for Aubrey and seven weeks for Carey, the family were able to settle back into life back home in Brighton, East Sussex.

Carey is now in remission, with no signs of the cancer cells in his body.

But his parents have been warned that the disease is so aggressive that until March next year there is a 40% chance of it returning. After that, the likelihood falls to just 5%.

Naomi added: Two other children lost their lives on the cancer ward while we were there, so we know how lucky Carey has been.

He and Aubrey have always been close but now their bond is stronger than ever.

"Shes a superstar and he couldnt have wanted anything more from a big sister. Hes doing so well now. He loves playing with his cars and hes just learning to walk too.

Aubrey is with him all the time she just adores him. She knows that she has saved his life and she loves being a big sister to him. They play cars together and hes learning to walk, so she stands with him encouraging him to take his steps.

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Carey fell ill last November but Naomi, a paediatric audiologist, and her husband Simon, a CPS lawyer, both 43, thought it was bronchitis because his sister had recently had the same thing.

A GP agreed but two days later he was rushed to hospital by ambulance with breathing difficulties.

Doctors at Great Ormond Street diagnosed juvenile myelomonocytic leukaemia, or JMML, which cannot be treated with chemotherapy. There are only 1.2 cases per million children in the UK each year.

Naomi said: I was hysterical. I kept trying to tell them that it wasnt cancer, it was bronchilitis. I couldnt accept what was happening.

Because parents are not suitable donors, Aubreys bone marrow was tested, a process that involves drawing a sample out using a needle.

Naomi said: There is only a 25% chance of any sibling being a match, so even with Aubrey we knew that the odds werent in our favour.

"If she hadnt been a match then we would have had to wait until doctors found an anonymous donor, but that may not have happened in time for Carey.

When the results came back to say that she was a perfect match for him, we couldnt believe it. We had been praying that she would save him, so to get the news that she was a match for him was just incredible.

When we heard I couldnt stop crying, it was so emotional. To think that Carey was going to have a chance of survival thanks to his big sister was the answer to our prayers.

The mum added: We did feel guilty about putting her through the procedure, but when we spoke to her about it, all she wanted to do was help. We were so proud of her.

The transplant was made even more special as it took place on March 15, which was Careys first birthday, giving the family a double celebration.

They are keen to raise awareness of the cancer symptoms and the charity Childhood Cancer and Leukaemia Group, which has helped them throughout their ordeal.

Naomi said: Having a child with cancer is one of the worst things that can happen to you. We didnt realise that it was leukaemia so we are thankful that it was spotted in time.

We received amazing support throughout from the hospital and from the CCLG.

We feel so lucky that Carey has come through it and it feels like a miracle to have him with us now.

Geoff Shenton, a childrens cancer specialist at Newcastle Upon Tyne Hospitals NHS Foundation, said: In a very small proportion of cases JMML can disappear on its own, but this is rare.

Most children will need a bone-marrow or stem-cell transplant. There is still a significant chance that the disease can relapse. There may be a possibility of a second transplant if this happens, but despite our best efforts, children still die from JMML.

For more information and support visit cclg.org.uk

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Girl, four, saves baby brother's life by donating her stem cells on his 1st birthday - The Mirror

Bone Marrow Stem Cells Comments Off on Girl, four, saves baby brother’s life by donating her stem cells on his 1st birthday – The Mirror | September 11th, 2022

This months top grants in regenerative medicine, sourced from Dimensions, includes projects on: a novel platform to enhance single cell interrogation of nervous system development, human endothelial cell regulation of ossification and the development of a dynamic double network hydrogel for generating pancreatic organoids from induced pluripotent stem cells.

This project aims to investigate a strategy, which utilizes novel spatial transcriptomics approaches, integrated multiplexed RNA/protein detection and visualization and computational algorithms to identify and map molecular markers of the preganglionic neurons in the ventral spinal cord and progenitor cell populations of the sympathetic ganglia. If successful, the approach could provide a foundation for basic research of peripheral nervous system birth defects and repair using stem cell-based therapies, as well as future studies of neuroblastoma initiation.

Funding amount:US$206,000

Funding period: 8 August 2022 31 July 2024

Funder:Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Research organization:Stowers Institute for Medical Research (MO, USA)

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Over one million patients undergo bone repair procedures in the USA annually, with autologous bone grafting remaining the preferred treatment for bone defects. The development of therapies that exploit the osteogenic potential of bone marrow-derived mesenchymal stem cells (bm-MSCs) has been limited due to limited understanding of the regulatory mechanisms of in vivo bm-MSC osteogenesis. Previous research from the group showed that the osteogenic potential of bm-MSCs is dependent on sustained proximity to endothelial cells. The goal of the present study is to elucidate the cellular and molecular mechanisms by which endothelial cells regulate the osteogenic differentiation of bm-MSCs and develop a foundation of knowledge upon which to build therapeutic strategies for bone regeneration utilizing autologous bm-MSCs.

Funding amount:US$442,000

Funding period: 10 August 2022 31 May 2027

Funder:National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

Research organization:Boston Childrens Hospital (MA, USA)

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Human induced pluripotent stem cells provide a valuable source of cells for basic research and translational applications. While there have been advances in lineage-specific differentiation of human induced pluripotent stem cells, there remains limited understanding on the impact of matrix stiffness, viscoelasticity and integrin ligand presentation on the multi-stage development of exocrine pancreatic organoids. This research aims to define the influence of matrix properties on the generation of exocrine pancreatic organoids by developing a viscoelastic dynamic double network hydrogel platform with controllable matrix mechanical properties and biochemical motifs. This will advance the application of chemically defined matrices as xeno-free artificial stem cell niches for organoid growth and tissue regeneration applications.

Funding amount:US$468,000

Funding period: 1 August 2022 31 July 2026

Funder:National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Research organization: Indiana University Purdue University Indianapolis (IA, USA)

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Fast-track status is granted for frontotemporal dementia, next to the existing designation for traumatic spinal cord injury

GELEEN, Netherlands, Sept. 8, 2022 /PRNewswire/ -- The European Union has grantedstem cell biotech Neuroplastan orphan medicinal product designation for the applicability of its stem cell technology platform to frontotemporal dementia (FTD), following a positive opinion from The European Medicines Agency (EMA). With the existing orphan disease designation (ODD) for traumatic spinal cord injury (TSCI), Neuro-Cells is now approved for a fast-track development pathway with market exclusivity for both a trauma-induced and a chronic degenerative central nervous system disorder. This marks an important milestone in the development roadmap of Neuroplast's Neuro-Cells platform, as a stepping stone to other chronic neurodegenerative diseases such as Alzheimer's, ALS and Parkinson's Disease. The potential width in therapeutic applicability of the Neuroplast technology gives perspective to millions of people suffering from neurodegenerative diseases that currently have no outlook on effective treatment.

One technology addresses underlying mechanisms of multiple acute and chronic neurological disorders

Several conditions of the central nervous system, even when they seem unrelated at first and may have distinctive causes, have similar underlying disease mechanisms in common. These include unprogrammed cell death boosted by inflammation. Neuro-Cells, an autologous, bone-marrow derived Advanced Therapy Medicinal Product, addresses that disease mechanism by moderating inflammation of damaged cells in the central nervous system, to limit further impairment. The treatment objective in acute disorders is to limit impact of sudden injury, where the treatment objective in chronic disorders is to limit progression of the disease.

Neuroplast is already running a fast-track development pathway for traumatic spinal cord injury (TSCI), with a Phase II clinical trial in progress. This designation for frontotemporal dementia illustrates the broader applicability of the same technology for acute as well as chronic neurodegenerative disorders, paving the way to explore further applicability to conditions such as ALS, Alzheimer's disease, traumatic brain injury, subarachnoid stroke and Parkinson's Disease.

Orphan disease designation for FTD awarded based on pre-clinical evidence

Orphan disease designations are restricted to products for rare conditions for which there are no satisfactory methods of treatment authorized. It allows for a faster market authorization pathway and ten-year market exclusivity.

Frontotemporal dementia (FTD) is a degenerative condition in the brain that affect approximately 3.8 people in 10,000 persons in the EU. Typical survival rate lies between three and fourteen years from symptom onset, dependent on the FTD variant at play.

For this approval, the European Union followed the positive opinion from the EMA after the EMA followed positive recommendations from the Committee for Orphan Medicinal Products (COMP). COMP partly based their conclusions on the availability of pre-clinical evidence in mice, that showed decrease in neuroinflammation markers and rescue of cognitive and social behavioral deficits. Examples include reduction of anxiety, depressive-like behavior and abnormal social behavior.

Neuroplast CEO Johannes de Munter states:

"This designation for frontotemporal dementia is an important milestone in expanding the Neuro-Cells development to a wider range of therapeutic areas. Using the same technology platform for traumatic spinal cord injury and frontotemporal dementia, illustrates an unusual range of acute and chronic neurological disorders that could potentially benefit from this."

Neuroplast is open to discuss investor opportunities to effectuate the clinical pathways to a wider scope of neurological conditions.

About Frontotemporal dementia

Frontotemporal dementia (FTD) is a degenerative condition in the brain that is characterized by behavioral and language impairments. Depending on the variant, patients experience changes in personality, emotion, speech or motor functions. Patients may first become indifferent or careless and have difficulty understanding sentences. While the condition progresses, patients may become language impaired, lack initiative and lose executive functions. The typical survival rate lies between three and fourteen years from symptom onset, dependent on the FTD variant at play.

FTD affects approximately 3.8 people in 10,000 persons in the EU, for whom there are no effective treatments available. Patients typically receive antipsychotics to limit behavioral symptoms.

About Neuro-Cells

Neuro-Cells is a transformative treatment under GMP. It contains non-substantially manipulated bone marrow-derived hematopoietic and mesenchymal stem cells, manufactured from a patient's own bone marrow (donor and receiver are the same person). Inflammatory inducing components and pathogens are removed during this process.

About Neuroplast

Neuroplast is a Dutch stem cell technology company focusing on fast-track development programs using autologous cell products for treatment of neurodegenerative diseases, with the aim of giving back perspective to people who suffer from those conditions.

The company was founded in August 2014 by physician Johannes de Munter and neurologist Erik Wolters. Current funders are Lumana Invest, Brightlands Venture Partners, LIOF and the Netherlands Enterprise Agency. Neuroplast is located at Brightlands Chemelot Campus in The Netherlands.

For more information, please visite http://www.neuroplast.com

About Lumana Invest

Investment company Lumana was established by entrepreneurs and unique due to not having a predetermined investment horizon. The Lumana founders showcase strong commitment to their portfolio companies by actively supporting management in strategic decision making.

About Brightlands Venture Partners

Brightlands Venture Partners (BVP) is the fund manager of Chemelot Ventures and is a so-called ecosystem investor. BVP invests in companies benefiting from and contributing to the Brightlands campuses in the south of The Netherlands. Other funds under management are BVP Fund IV, Brightlands Agrifood Fund and Limburg Ventures. The funds of BVP focus on sustainability and health; together the funds have made over 40 investments.

About LIOF

LIOF is the regional development agency for Limburg and supports innovative entrepreneurs with advice, network and financing. Together with entrepreneurs and partners, LIOF is working towards a smarter, more sustainable and healthier Limburg by focusing on the transitions of energy, circularity, health and digitalization.

About The Netherlands Enterprise Agency

The Netherlands Enterprise Agency operates under the auspices of the Dutch Ministry of Economic Affairs and Climate Policy. It facilitates entrepreneurship, improves collaborations, strengthens positions and helps realize national and international ambitions with funding, networking, know-how and compliance with laws and regulations.

Forward looking statements

All statements other than statements of historical facts, including the statements about the clinical and therapeutic potential and future clinical milestones of Neuro-Cells, the indications we intend to pursue and our possible clinical or other business strategies, and the timing of these events, are forward-looking statements. Forward-looking statements can be identified by terms such as "believes", "expects", "plans", "potential", "would" or similar expressions and the negative of those terms. These forward-looking statements are based on our management's current beliefs and assumptions about future events and on information currently available to management. Neuroplast B.V. does not make any representation or warranty, express or implied, as to the improper use of this article, accuracy, completeness or updated status of above-mentioned statements. Therefore, in no case whatsoever will Neuroplast B.V. be legally liable or liable to anyone for any decision made or action taken in conjunction with the information and/or statements in this press release or for any related damages.

In case of any further questions, please contact:

Neuroplast

Johannes de Munter, CEOT: +31 (0)85 076 1000E: [emailprotected]

LifeSpring LifeSciences Communication, Amsterdam

Leon MelensT: +31 6 538 16 427E: [emailprotected]

Logo: https://mma.prnewswire.com/media/1666795/Neuroplast_Logo.jpg

View original content:https://www.prnewswire.com/news-releases/neuroplast-receives-second-orphan-medicinal-product-designation-for-neuro-cells-paving-the-way-for-application-to-both-chronic-and-trauma-induced-neurodegenerative-diseases-301620123.html

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Neuroplast receives second orphan medicinal product designation for Neuro-Cells, paving the way for application to both chronic and trauma-induced...

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What we put in our bodiesfoodis regulated by the Food and Drug Administration and the U.S. Department of Agriculture.

What we put on itskin care and beauty productsis not.

That could be an issue.

In one 2021 report, scientists tested 231 popular makeup products from the U.S. and Canada and found that more than 100 had Per-and poly-fluoroalkyl substances (PFAS). These are chemicals that dont break down and build up in the body over time.

They include perfluorooctanoic acid, which may cause cancer, according to the American Cancer Society.

Clean skin goes beyond washing your face.

Your skin is a living, dynamic organ, says Nava Greenfield, M.D. of Schweiger Dermatology Group in New York City. Just like you consider carefully what you put into your mouth, you should take care in what you place on your skin.

Understanding whats in your products can help you achieve long-term health thats more than skin-deep.

Heres what the science says you should avoidand what to use instead.

The skin is our largest organ, notes Marianna Blyumin-Karasik, board-certified dermatologist, co-founder of Precision Skin Institute, and founder of Stamina Cosmetics.

The skin has high absorption, so skin care products that can be absorbed and enter our bloodstream can have detrimental effects on our overall health, Blyumin-Karasik says.

Some ingredients like synthetic or highly concentrated fragrances or chemicals in personal care products can trigger skin sensitivity, irritation, or a more intense allergy.

Symptoms can include:

Other ingredients have been linked to more serious problems, like:

For example, a 2018 review suggested that phthalates found in certain personal care products could adversely affect male fertility.

A 2021 study indicated that exposure to formaldehyde, sometimes used in keratin hair treatments, could lead to heart malformations in a developing fetus.

In 2020, California became the first state to issue a statewide ban on 24 chemicals, including methylene glycol and formaldehyde.

Other states dont have these bans, leaving consumers to analyze and interpret labels themselves.

Complicating things, some recommendations to avoid specific ingredients arent one-size-fits-all. Different people may have different (or no) reactions to certain ingredients, even if theyre common allergens.

Aside from real toxins and dangerous chemicals, a list like this will be different for each person, Greenfield says. Unfortunately, its not all black and white.

Having an idea of whats potentially toxic and whats more likely to cause skin irritation can help you make informed decisions about the products you choose.

From common allergens to potential carcinogens, here are the ingredients Blyumin-Karasik and Greenfield suggest avoiding:

Blyumin-Karasik and Greenfield warn that PEGs are a potential skin irritant.

Theyre most often found in lotions, creams, and hair products because they can act as skin conditioners and humectants, a common moisturizing agent.

A small 2021 case study examined six cases of acute sensitivity to PEGs. However, researchers also noted the allergy was rare.

Blyumin-Karasik notes that methyl and propyl parabens are preservatives with reputations for being hormone disruptors. However, research is mixed.

A 2017 study on gerbil prostates indicated that methylparaben could disrupt estrogenic and androgenic receptors that might affect the prostate.

Another 2017 study suggested parabens, including methyl and propyl parabens, posed little health risk. However, researchers noted that parabens could inhibit compounds with anti-estrogenic properties.

The FDA wrote in 2022 that it didnt have enough evidence to warn that parabens affect human health. The American Academy of Dermatology (AAD) reported similarly in 2019, noting that allergic reaction risks were low.

Found in some eye make-up products, lipsticks, and deodorants, aluminum can cause skin irritation, according to Greenfield.

Theres also been discussion as to whether aluminum is a carcinogen, or cancer-causing agent.

A 2015 study suggested aluminum can increase the migration of breast cancer cells and called for more research.

In 2013, the Cosmetic Ingredient Review said alumina and aluminum hydroxide was safe to put in cosmetics, noting that it doesnt get absorbed into the skin and less than 1 percent is absorbed orally.

This ingredient is a preservative commonly found in soaps and shampoos and may cause skin irritation or allergies, Blyumin-Karasik explains. Greenfield agrees with avoiding formaldehyde, saying its a common irritant.

A 13-year retrospective study of patch tests published in 2020 indicated that a polymer known as toluene-sulfonamide-formaldehyde resin (R-TSF or TSFR), often used in nail polish, was one of the most common allergens. Its also known as a carcinogen, but data shows its only hazardous if a person inhales a significant amount.

The American Cancer Society says formaldehyde in personal care products like shampoos typically contains formaldehyde levels that are far below hazardous to health. The organization says keratin hair smoothing treatments can raise indoor air concentration to hazardous levels.

Phthalates are typically used to make sure plastic doesnt break. They can also be used in fragrances in skin products. Blyumin-Karasik warns they may disrupt hormones.

A 2020 literature review indicated that phthalates could lead to:

A 2018 study suggested phthalates could lead to pregnancy loss and fertility issues.

However, its important to note that neither piece of research above was specific to phthalates in beauty products.

Key West and Hawaii recently banned oxybenozone, which is commonly found in sunscreen. Blyumin-Karasik says it can disrupt hormones and cause allergic reactions.

A 2020 review of 29 studies indicated no a link between fertility issues and oxybenzone and called for more research.

However, an older 2016 study indicated that men with higher levels of benzophenone-type ultraviolet (UV) filter concentrations had lower sperm concentrations.

Avoiding fragranced products and using a mineral-based sunscreen can help avoid harmful chemicals, Blyumin-Karasik says. Looking for preservative-free items can also cut down on risks of irritants and health hazards.

The main purpose of preservatives is to maintain the integrity of the personal care products, Blyumin-Karasik says. The natural alternatives may not attain as long of the shelf-life as the chemical ones, but theyre better for our well-being.

To clean up your beauty regimen, Blyumin-Karasik suggests looking for products that contain these safer ingredients instead.

Blyumin-Karasik suggests using tea tree oil, an essential oil found in shampoos, skin care items, hand sanitizers, and first aid products.

A 2021 study suggested tea tree oil could help disinfect hands when used in sanitizer.

Research from 2015 indicated it could aid wound healing, and a 12-week pilot study published in 2017 suggested it could reduce acne.

Instead of PEGs, opt for a humectant with fewer potential side effects. Blyumin-Karasik recommends glycerin.

One small 2017 study of women indicated that products with a mix of hyaluronic acid, glycerin, and Centella asiatica (gotu kola) could boost skin hydration for 24 hours.

A 2019 safety assessment suggested glycerin was safe to use in cosmetic practices.

Coconut oil, or Cocus nucifera, is extracted from the meaty part of a coconut fruit.

Blyumin-Karasik recommends it because its moisturizing and can reduce mold growth in skin care products.

A 2022 study indicated that a coconut oil-based serum combined with deer antler stem cell extract for two weeks could:

A 2019 study indicated virgin coconut oil had anti-inflammatory properties and supported its use in skin care products.

Blyumin-Karasik says elderberry, or Sambucus nigra extract, often found in serums, has versatile benefits for our skin.

She notes these benefits include antimicrobial effects and high levels of vitamin C.

Research on elderberry is limited, particularly in topical products. However, a 2019 study suggested it had anti-aging benefits when ingested as a supplement.

Blyumin-Karasik says willow bark, or Salix nigra extract, is an excellent source of skin preservation. She recommends it for its anti-inflammatory and antimicrobial properties.

Besides that, willow bark contains a potent salicin ingredient which has gentle exfoliating properties to cleanse pores and reduce skin surface oil, she says.

A 2019 study suggested willow bark total extract may have antioxidant and anti-inflammatory benefits.

An older 2010 study suggested that salicin, which is extracted from white willow bark, may have benefits when applied to the skin topically.

When shopping for personal care products, there are a few things youll want to keep in mind, depending on your age and any conditions you have.

Leave the layering for sweater weather, not skin care. Blyumin-Karasik says the biggest issues she sees in her clinic happen when people try to cake on too many products or ingredients.

Trying to be innovative or frugal, young individuals play with potentially hazardous ingredients such as baking soda or lemon juice which can lead to significant skin irritation, Blyumin-Karasik says. Older individuals try to layer too many products onto their skin such alpha hydroxy acids and potent retinoids and as a result, create skin allergy or irritation.

Blyumin-Karasik recommends working with a dermatologist to find the correct ingredients for your skin type and beauty goals.

A long ingredients list doesnt necessarily mean there are a ton of items working to boost your skins health. Sometimes, simple ingredient lists are most effective.

In general, if a skin care product has too many chemicals or fragrances, it can irritate the skin and cause skin rashes, and its best to avoid, Blyumin-Karasik says.

Individuals with sensitive skin, eczema, dermatitis, or rosacea will want to pay particular attention to product labels and the less is more mantra, Blyumin-Karasik says, as people with these conditions are more prone to irritation.

Theyre best served by using fragrance-free, sensitive skincare lines such as Avene and Bioderma, and definitely avoiding any of the above skin allergens, Blyumin-Karasik says.

Blyumin-Karasik advises acne-prone individuals to opt for products that wont clog pores. She suggests looking for words like oil-free and noncomedogenic and minimizing the use of occlusive moisturizers or make-up.

These can cause more breakouts and blemishes, Blyumin-Karasik warns.

When purchasing skin care products, youre making an investment in your bodys largest organ.

But some ingredients may not serve your skin or overall health.

Though research in some cases is minimal and others are mixed, Phthalates and some parabens are linked to hormonal disruption. Other ingredients are carcinogens or may cause irritation.

Speaking with a dermatologist can help you figure out the best and safest products and ingredients for your skin and overall health.

Beth Ann Mayer is a New York-based freelance writer and content strategist who specializes in health and parenting writing. Her work has been published in Parents, Shape, and Inside Lacrosse. She is a co-founder of digital content agency Lemonseed Creative and is a graduate of Syracuse University. You can connect with her on LinkedIn.

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6 Ingredients to Avoid Putting on Your Skin - Healthline

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Researchers from the Murdochs Children Research Institute (MCRI) are developing new treatments for congenital heart disease that could enable children born with birth defects can regenerate the damaged organ.

In 2011, Prof. Enzo Porrello, who is nowhead of the Heart Regeneration Laboratory at the MCRI,demonstrated the regenerative properties of newborn mouse hearts at the University of Texas Southwestern Medical Centre. Prior to this research, the capacity of mammalian hearts to regenerate was a debated topic.

This sort of changed our thinking of what was possible in terms of stimulating the human heart to regenerate itself following damage, such as a heart attack, Porrello said, reported theAustralian. And I guess this also fuelled my own interest in my subsequent career in the area of regenerative medicine.

After hearing about cases where newborns recovered from massive heart attacks, Porrello began to explore the regenerative properties of human newborn hearts.

In 2017, Porrello and Prof. James Hudson manufactured living and beating heart tissues from stem cells in a laboratory at the University of Queensland.

Porrello said that although other scientists had grown heart muscle cells from stem cells, nobody had grown the cells as miniature complex three-dimensional tissues. Additionally, they were not able to grow such tissues in a format compliant to drug development, he said.

And thats really the technological breakthrough that we were able to make.

According to the Australian Institute of Health and Welfare, approximately nine out of every 1,000 babies born around the world will be born with congenital heart disease. In Australia, it is estimated that 2,400 babies are born with congenital heart disease annually, while in America, nearly one percent of all babies born are estimatedby the Centre For Disease Control to have the condition.

Porrello said that, at the moment, if a child develops heart failure and doesnt respond to standard frontline therapies, a heart transplant is their only option. Children in this situation are put on a transplant waiting list, and whilst waiting for a heart to become available, they are put on mechanical support.

Heart transplantation is limited by organ donor availability, and its also limited by the need for lifelong immunosuppression in those patients, Porrello said.

And so if were able to develop these bioengineered heart tissues from stem cells, this could potentially prevent or delay the need for heart transplantation in these very unwell individuals with end-stage heart failure.

Porrello said that the ultimate goal of his research is to harness the self-repairing capacity of the newborn heart and to develop drugs that waken the hearts dormant regenerative abilities so that the organ may repair itself after damage.

I would say that based on recent studies in the field in the past 10 years since we first made our discovery in mice, we are certainly getting closer, he said.

There is sort of proof of concept that this is possible now, at least in mice, and the question is whether or not we can now make that a therapeutic reality in humans.

The first step in creating these complex heart tissues is attaching special molecules to stem cells; these molecules trigger the cells to morph into heart muscle tissue. The heart tissues are then developed in a plastic culture dish that consists of 96 tiny wells.

The geometry of the well is designed in such a way that the heart tissues spontaneously form when the heart muscle cells are inserted into the well, Porrello said.

He said that within each well of the device are tiny elastic micropillars; the pillars function as elastic cantilevers since they are attached to the dish at only one end and extend horizontally to the dish. The heart muscle cells condense around these cantilevers to produce tiny miniature beating heart tissues that contract around the micropillar; every time the tissue contracts, the micropillar within it deflects.

Porrello said that the device enables researchers to measure the force that the tissues are generating, allowing them to observe how fast the tissues are beating and whether they display any irregularities in their heartbeat. These capabilities are useful for treatment testing because the effect that medication or genetic manipulations of stem cells have on the tissues heartbeat can be seen.

And so it serves as a pretty powerful platform for looking at drug responses, but also modelling genetic forms of heart disease.

Were actually now scaling up these tissues and growing very, very large bioengineered heart tissue patches that can be implanted onto the heart.

In an email to The Epoch Times, Porrello said in the future that, bioengineered heart tissue patches could be used to treat adults with heart failure, and alternative approaches are already being trialled.

Our bioengineered heart tissues could also be used to support the failing heart in adults with underlying heart disease.

Further studies are required to confirm that our bioengineered heart tissue patches are safe and effective in animal models before progressing to human trials. These pre-clinical safety and efficacy studies are underway.

He noted that although significant advances and a better understanding of the hearts regenerative mechanisms have been made in recent years, using this knowledge to develop a safe and effective drug is a slow process.

It typically takes 10 years and around $1 billion dollars to develop a new heart failure drug and take it all the way through to clinical approval. We are at the beginning of that journey.

We need to gain a better understanding of the fundamental biology underlying heart regeneration before we can develop effective treatments.

Porello is now applying his discoveries in a clinical context at theMCRIto reach his goal of regenerating human hearts. The regeneration research at the institute has two branches, the first focuses on studying diseases using lab-grown models of the heart muscle. The models are made using blood and tissue samples collected from sick children at the Royal Childrens Hospital in Melbourne.

He said that this branch of the research enables the team to model the genetic basis of the disease in any individual.

Were using this technology to model childhood heart disease, trying to understand its causes, and then using those genetic models of heart disease to test and develop therapeutic approaches to treat those conditions, he said.

Porrello said that the second branch of the research performed at the MCRI explores the regenerative approach to growing the very, very large bioengineered heart tissue patches. The researchers plan is to eventuallyimplant the patches into a heart to function as a biological assistance device that supports the function of the heart.

If it works, it would be transformative, Porrello said.

Stem cells have been used in medicine for more than fifty years, with the most common stem cell procedure currently beingbone marrow transplantsalso known as hematopoietic stem cell transplantsused to treat patients with blood cancers such asleukemiaand blood disorders such assickle cell diseaseandthalassemia.

More recently, skin grown from stem cells has been used to treat extensive burns, and stem cells from fat (adipose tissue) have been used as tissue fillers.

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Regenerative Properties of the Newborn Heart Offers Hope for Those With Congenital Heart Disease - The Epoch Times

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After struggling with acne for years, White Orange founder Carishma Khubhani still had a hard time finding skin are products that worked for her and even when her acne cleared, she was worried that her skin may revert back to its old habits. I always wanted to have my own skin care line one day, said Khubani, who was a musician in Los Angeles before becoming a brand founder. [My dermatologist and esthetician] told me that the only things that have been proven to make a visible difference in your skin are vitamin C and retinol. Other vitamin C serums were expensive or unpleasant to use or simply didnt work which led her to create White Orange.

After three years of formulating, White Orange claims to bring on a new generation of vitamin C. Vitamin C is the king of skin care ingredients for good reason its proven to help with sun damage, dark spots, and even acne. But you might be surprised to learn that there are different types of vitamin C in the products you use. Most vitamin C products on the market (including the priciest products) use a form of vitamin C called L-ascorbic acid. Its a go-to because there have been so many clinical studies supporting its efficacy; however, the downside is that it can be irritating and unstable. (Stability ensures that the product retains its potency over time.) It's cheap and it's inexpensive and [brands who use it] want to maximize their profit margins to be able to pay all their overhead, Khubani says.

With this knowledge, Khubani chose to use a less-common form of vitamin C called tetra hexadecyl ascorbate, or BV-OSC. She claims its the most potent, yet stable form of vitamin C, and so far, the science looks promising: A study found that after an aqueous gel with 10% BV-OSC was applied to a group of patients over the span of two to 10 months, age spots, acne and skin redness all showed immense improvement.

In addition to tetrahexadecyl ascorbate, one of the most significant ingredients that influenced the name of the product is pith the white part of the orange (hence the brand name) which was included for its high concentration of vitamin C. Other ingredients include hyaluronic acid, ferulic acid, and vitamin E all superstar skin care ingredients proven to fight free radicals and help overall skin texture and brightness. White Orange also added orange stem cells, which feature their own exclusive proprietary complex, and a liposomal delivery system to help the ingredients penetrate more deeply into the skin.

Other products also use tetrahexadecyl ascorbate, like Sunday Riley C.E.O 15 Vitamin C Brightening Serum so what makes White Orange different? Its all in the delivery system. Many vitamin C products come in glass dropper bottles, so the product is exposed to light and air every time you use it, which allows the product to oxidize and become less effective. White Orange puts their product in a syringe-style bottle, so your product isnt exposed to air and you only pump out the amount you need, preserving the freshness of the serum. The formula is also vegan and cruelty-free.

Khubani recommends using the product before you apply your moisturizer and SPF and after you wash your face and potentially apply a toner. After washing my face with my CeraVe Hydrating facial cleanser, I used the White Orange serum and finished off with my trusty CeraVe moisturizer. The formula is very light and non-sticky and so far, the product seems to be very gentle and non-irritating (I have highly sensitive skin). I also really like the syringe bottle, which is travel-friendly and dispenses the perfect amount each time. I havent noticed any anti-aging or acne-preventing effects, but I would recommend trying this product if youre looking to add a gentle serum to a simple skincare routine and if you have sensitive skin, you can rest easy knowing this formula wont irritate.

Studies cited:

Telang P. S. (2013). Vitamin C in dermatology. Indian dermatology online journal, 4(2), 143146.

Al-Niaimi, F., & Chiang, N. (2017). Topical Vitamin C and the Skin: Mechanisms of Action and Clinical Applications. The Journal of clinical and aesthetic dermatology, 10(7), 1417.

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EARD2022 is over, but the research and events continue. Heres a summary of everything thats happened in August.

We are hiring! We are currently looking for a full-time chief of staff, a full-time data-driven Senior Marketing Manager, a part-time Youtube sponsorship/partnership acquisition lead, a social media intern, a part-time grant writer, and volunteers to support various programs. If you are interested in learning more about any of these positions, please contact us with your resume and salary expectations.

Announcing the Longevity Prize: The Longevity Prize is a series of prizes designed to honor the researchers who are helping to build a future in which age-related diseases are a thing of the past. This new initiative aims to accelerate progress in the rejuvenation biotechnology field and encourage innovation.

Stephanie Dainow to Present at the 9th ARDD Conference: On August 22, 2022, Lifespan.io Executive Director Stephanie Dainow participated in the Decentralized Science and Blockchain session as a part of the Emerging Tech Workshop at the worlds largest annual Aging Research and Drug Discovery conference (9th ARDD).

Longevity Camp: The Longevity Summer Camp is a four-day retreat featuring people from many longevity-related walks of life. Recently, somewhere between the former gold mining town of Nevada City and the infamous Donner Pass, a unique gathering took place.

Cells Return from Death: Cells, dead for an hour under warm conditions, have been revived. Questions about when life begins have been hot topics for awhile, but there is also debate about when life ends.

Rapamycin and Metformin: Rapamycin and metformin, two well-studied drugs in aging research, can be combined for synergistic effects in mice. Rapamycin and metformin are viewed by many as the two most promising anti-aging drugs, but now scientists have found that these drugs can work hand in hand.

Steve Horvath on the Present and Future of Epigenetic Clocks: Dr. Steve Horvath is the inventor of the epigenetic clock and, currently, principal investigator at Altos Labs. We talked about the recent developments in this immensely important field, including pan-mammalian clocks, two-species clocks, and single-cell clocks, along with the challenges the field faces.

Prof. Albert-Lszl Barabsi on Network Medicine: Albert-Lszl Barabsi is the Robert Gray Dodge Professor of Network Science at Northeastern University, and he also holds an appointment in the Department of Medicine at Harvard Medical School. We talked about a revolutionary network medicine approach that can greatly enhance our ability to understand biological processes and seek cures for disease.

Martin ODea Talks About the Longevity Summit: We recently had the opportunity to speak to Martin ODea about a new longevity-focused event happening in Irelands capital city on September 18th-20th. Martin holds an MBS and is a business lecturer at Dublin Business School in Dublin, Ireland. He is also the author of Beyond the Subjectivity Trap.

Dr. Aubrey de Grey Will Speak at the Longevity Summit Dublin: We recently caught up with Dr. Aubrey de Grey and talked to him about the upcoming Dublin Longevity Summit and how things are looking on the advocacy landscape.

Old Plasma Dilution Reduces Human Biological Age: The Journal Club has returned to our Facebook page with your host, Dr. Oliver Medvedik. This month, we have investigated a paper, Old plasma dilution reduces human biological age: a clinical study, in which Irina Conboy and her team investigated the effects of therapeutic plasma exchange on aging in people.

Vitamin D Fails to Improve Bone Health in Mega-Study: A high-quality, randomized, controlled trial found no effect of vitamin D supplementation or blood levels on the incidence of fractures in an aging population.

Hesperetin Upregulates Metabolism and Longevity in Mice: Researchers publishing in Journal of Biomedical Science have concluded that hesperetin, a compound found in various herbs, improves longevity in mice by promoting the expression of the pro-longevity gene Cisd2.

Caloric Restriction Improves Immune System Function: A new study published in Mechanisms of Aging and Development has shown that caloric restriction effectively restores T cell abundance in aged mice. Caloric restriction has become a well-known anti-aging intervention, as it can reverse several hallmarks of aging and extend lifespan in different animal models.

Ghrelin Is Associated with Worse Muscle Aging in Mice: A team of researchers publishing through Multidisciplinary Digital Publishing Institute has described an association between ghrelin and skeletal muscle aging in mice. Ghrelin is a peptide containing 28 amino acids. Its main function is to stimulate the appetite through receptors in the hypothalamus.

Sauna Combined with Exercise Improves Cardiovascular Health: In a randomized, controlled trial, scientists have shown that sauna and exercise, when taken together, might have a synergistic, beneficial effect on cardiovascular health and cholesterol levels. Sauna bathing has been credited with many health benefits, predominantly for the cardiovascular system.

Developing Nanobodies to Fight Parkinsons Disease: A team of researchers publishing in Nature Communications has described nanobodies that can destroy the -synuclein aggregates that characterize Lewy bodies, which are associated with dementia and Parkinsons disease. Traditional antibody therapies, while promising in some studies, are too large to enter cells in order to affect the aggregates there.

Scientists Move the Boundaries of Post-Mortem Recovery: Researchers have been able to achieve substantial recovery of cellular and organismal activity in pigs that had been dead for a full hour. Advances in resuscitation have already moved the boundaries of life and death, making it possible to revive a person several minutes after the heart stops beating.

An In-Depth Review of Skin Aging Genes: In a new systematic review published in Scientific Reports, multiple genes driving skin aging were identified. The authors start by explaining the intrinsic (genetic and chronological) and extrinsic (environmental) factors that drive skin aging.

Hypertension Is Associated with Brain Drainage Changes: Researchers publishing in Aging have found that enlarged perivascular spaces in the brain are correlated with vascular disorders. These spaces, which are part of the brains glymphatic system, allow for the drainage of potentially dangerous metabolites such as beta amyloid.

Rapamycin-Loaded Microneedles Reverse Hair Loss in Mice: Scientists have successfully regrown hair in a mouse model of hair loss using custom-made plastic microneedles loaded with rapamycin and epigallocatechin gallate (EGCG), an active ingredient in green tea.

Identifying Mitonuclear Genes for Longevity: Publishing in GeroScience, a team of researchers that included Nir Barzilai and Matt Kaeberlein examined genes that may affect both mitochondria and lifespan.

Dietary Restrictions Do Not Help Cognitive Function in Mice: A new study published in Neurobiology of Aging has shown that neither caloric restriction nor intermittent fasting improve late-life cognition in genetically diverse mice, but the effect depends on genetic composition.

Combining Senolytic Pathways Has Synergistic Effects: A team of researchers have explained in Aging how multiple compounds that target the BCL-2 protein family are considerably more effective against senescent cells than each compound by itself.

New Synthetic Molecule Alleviates Alzheimers in Mice: Scientists have synthesized a molecule that alleviates Alzheimers in a mouse model by targeting inflammation. Two of the most prominent and probably interconnected symptoms of Alzheimers disease are the accumulation of amyloid beta (A) and chronic neuroinflammation.

The Relationship Between Stroke and Inflammation: Publishing in Aging, a team of Chinese researchers has provided evidence showing a relationship between systemic inflammation and prognosis after a stroke. As the researchers point out, strokes are the leading cause of death in China.

Almost Half of Cancer Deaths Worldwide are Preventable: Researchers have shown that 44.4% of cancer deaths worldwide can be attributed to preventable risk factors, including behavioral and environmental ones. It is well known that many cancer cases occur due to behavioral and environmental and factors such as smoking and pollution, which makes them theoretically preventable.

Rapamycin and Metformin Show Synergy in Mice: Scientists have found that rapamycin and metformin work hand in hand in diabetes-prone mice, boosting each others effectiveness and blocking side effects. Both have been in use for various indications for decades and have decent safety profiles.

Plasma Dilution Appears to Rejuvenate Humans: Published in GeroScience, a groundbreaking study from the renowned Conboy lab has confirmed that plasma dilution leads to systemic rejuvenation against multiple proteomic aspects of aging in human beings. This paper takes the view that much of aging is driven by systemic molecular excess of signaling molecules, antibodies, and toxins.

Mitochondrial Drug Alleviates Atherosclerosis in Mice: Scientists have drastically improved various symptoms of atherosclerosis in mice by precisely targeting mitochondria with a plant-derived antioxidant. Atherosclerosis, the accumulation of plaques on arterial walls, is one of the deadliest age-related diseases.

Intravenous Stem Cells Alleviate Guinea Pig Osteoarthritis: Scientists have shown that intravenous delivery of mesenchymal stem cells, which has some advantages over the more conventional intra-articular injection, alleviates age-related osteoarthritis and decreases inflammation in guinea pigs. Osteoarthritis, a degenerative joint disease, is one of the most common causes of disability in old age.

Glycans as Biomarkers of Aging: In a new review published in Clinica Chimica Acta, researchers from the University of Zagreb discuss immunoglobulin G glycans, the changes that their composition undergoes with aging, and their potential as biomarkers of aging. One of the reviews co-authors is Prof. Gordan Lauc, who gave a presentation on them at EARD2022.

A wearable electrochemical biosensor for the monitoring of metabolites and nutrients: The monitoring of metabolites for the early identification of abnormal health conditions could facilitate applications in precision nutrition.

Epigenome-wide association study analysis of calorie restriction in humans, CALERIE TM Trial analysis: DNA methylation changes may contribute to caloric restrictions effects on aging.

Association of Leisure Time Physical Activity Types and Risks of All-Cause, Cardiovascular, and Cancer Mortality Among Older Adults: There were significant associations between participating in 7.5 to less than 15 MET hours per week of any activity and mortality risk.

Ginkgo biloba extract EGb 761 plus acetylcholinesterase inhibitors improved cognitive function in patients with mild cognitive impairment: These findings suggest that combined therapy with EGb 761 plus AChEI may provide added cognitive and functional benefits in patients with MCI.

Suppression of trimethylamine N-oxide with DMB mitigates vascular dysfunction, exercise intolerance, and frailty associated with a Western-style diet in mice: These therapies may be promising for mitigating the adverse effects of a Western diet on physiological function and thereby reducing the risk of chronic diseases.

Canagliflozin retards age-related lesions in heart, kidney, liver, and adrenal gland in genetically heterogenous male mice: Canagliflozin can be considered a drug that acts to slow aging and should be evaluated for potential protective effects against many other late-life conditions.

Fecal microbiota transplantation can improve cognition in patients with cognitive decline and Clostridioides difficile infection: This study revealed important interactions between the gut microbiome and cognitive function. Moreover, it suggested that FMT may effectively delay cognitive decline in patients with dementia.

Mitochondrial dynamics maintain muscle stem cell regenerative competence throughout adult life by regulating metabolism and mitophagy: As mitochondrial fission occurs less frequently in the satellite cells in older humans, these findings have implications for regeneration therapies in sarcopenia.

Long-lasting, dissociable improvements in working memory and long-term memory in older adults with repetitive neuromodulation: These findings demonstrate that the plasticity of the aging brain can be selectively and sustainably exploited using repetitive and highly focalized neuromodulation

Supplementing Glycine and N-Acetylcysteine (GlyNAC) in Older Adults Improves Aging Hallmarks: By combining the benefits of glycine, NAC and GSH, GlyNAC is an effective nutritional supplement that improves and reverses multiple age-associated abnormalities to promote health in aging humans.

VitaDAO Funds ApoptoSENS Project for $253,000: Preventing the dysfunction of natural killer cells may be a promising area to explore in the fight against cellular senescence. Researchers are hoping to define the correlation between the increase in senescent cells and the onset or worsening of disease in humans.

VitaDAO Backs Research into Chronic Oral Disease: Periodontal disease affects more than 47% of adults aged 30 and over. For people over 65 years of age, that number rises to over 70%, making periodontitis one of the most commonly observed age-related illnesses. Jonathan Ans lab seeks to research inflammation-targeting compounds that can help treat periodontal disease.

Researchers Propose Five New Hallmarks of Aging: Publishing in Aging five months after their panel discussion in Copenhagen, many well-known researchers have explained their reasons for wishing to add new hallmarks of aging to the existing paradigm.

SENS Research Foundation Announces Ending Aging Forum 2022: SENS Research Foundation has announced this years Ending Aging Forum, which will be held through a virtual conference platform with an immersive environment.

Longevity Investors Conference: Organized and sponsored by Maximon, the Longevity Investors Conference is focused on the investment aspects of longevity. The LIC welcomes everyone with an interest in the financial aspects of the longevity sector, including venture capitalists, asset managers, and managers of private equity funds and private banks.

Longevity Summit Dublin: This conference will feature two days of inspiring research developments along with top longevity entrepreneurs, biotech companies, longevity investors, and researchers from around the world.

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Rejuvenation Roundup August 2022 - Lifespan.io News

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FACT.MR

Over the coming years, the orthopedic navigation systems market is expected to experience significant growth due to rapid technological innovations, introduction of new orthopedic navigation products, rising cases of cardiovascular diseases, increased funding in R&D activities to improve orthopedic navigation product effectiveness, and rise in the prevalence of osteoarthritis.

United States, Rockville MD, Sept. 02, 2022 (GLOBE NEWSWIRE) -- Expanding at a high-value CAGR of 17%, the global demand for orthopedic navigation systems is projected to increase to a valuation of US$ 433.8 million by 2027, predicts Fact.MR, a market research and competitive intelligence provider.

By expressing three-dimensional computer images in comparative patient analysis, which is a feature of image-guided surgical systems, the orthopedic navigation system integrates information from pre-operative planning and intra-operative execution. These computer workstations for image-guided surgery include a surgical planning and display monitor, image-processing software, and a digitizing system.

As a result of bone spine damage to the spinal nerves, spinal cord, or neurological injury weakening, spinal injuries are the primary cause of mortality and morbidity. To reduce long-term functional disability, prompt medical and surgical care is essential, thereby driving the need for orthopedic navigation systems.

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Effective Results of Computer-assisted Navigation Systems

Other elements anticipated to influence the industry's revenue include associated benefits of computer-assisted surgeries (CAS), including low blood loss, shorter hospital stays, and simpler recovery.

Accurate implant alignment is made possible by CAS, which also enhances functioning, and quality-adjusted life years, and causes reduced discomfort, tissue damage, and problems.The aforementioned reasons are behind therising demand for minimally-invasive surgeries.

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Another factor that is anticipated to increase orthopedic navigation system demand is the development of technology in orthopedic surgical navigation procedures, as well as the rising prevalence of osteoarthritis, and increased investments in R&D.

Key Takeaways from Market Study

Demand for orthopedic navigation systems is expected to surge at a CAGR of 17% from 2022 to 2027.

Global orthopedic navigation system sales areanticipated to be driven by an increase in the use of minimally-invasive procedures and navigation software by doctors and surgeons due to the availability of affordable orthopedic navigationsolutions and greater awareness.

In terms of technology, optical navigation systems are superior to electromagnetic (EM) systems because they expose users to less radiation and provide greater accuracy during difficult operations, allowing surgeons to move accurately through the anatomy of a patient.

Sales of optical navigation systems are expected to balloon at a CAGR of 19% from 2022 to 2027.

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Winning Strategy

Top manufacturers of orthopedic navigation systems are concentrating on raising knowledge about these systems as well astheiruse and advantages among patients and medical professionals alike. By providing Continual Medical Education (CME) sessions, manufacturers of surgical navigation solutionsin developed nations have started to reach out to local communities.

As a result, more doctors and specialists are aware of the existence and application of orthopedic navigation systems. Furthermore, the 6- to 7-year warranty on commercially available orthopedic navigation devicesmakes the entire product sales cycle 7 years.

The market for orthopedic navigation systems is anticipated to expand rapidly over the forecast period due to increasing demand for technological assistance in orthopedic therapies.

Robotic-assisted surgical navigation robot NaoTrac was given CE mark clearance by Taiwan-based firm Brain Navi Biotechnology in November 2021. The company specialises in cutting-edge navigation robots.

Acuson Freestyle Elite ultrasound system, which can be used in conjunction with Artis angiography devices to provide quick and simple ultrasound guidance during interventional procedures, was introduced by Siemens Healthineers in March 2017.

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Segmentation of Orthopedic Navigation Systems Industry Research

By Technology :

Electromagnetic

Optical

Radiography

Others

By Application :

Knee

Spine

Hip

Joint Replacement

Others

By End User :

By Region :

North America

Latin America

Europe

East Asia

South Asia & Oceania

MEA

More Valuable Insights on Offer

Fact.MR, in its new offering, presents an unbiased analysis of the global orthopedic navigation systems market, presenting historical demand data (2017-2021) and forecast statistics for the period of 2022-2027.

The study divulges essential insights on the market on the basis of technology (electromagnetic, optical, radiography, others), application (knee, spine, hip, joint replacement, others), and end user (hospitals, clinics, ambulatory surgical centers, others), across five major regions of the world (North America, Europe, Asia Pacific, Latin America, and MEA).

Check out more related studies published by Fact.MR Research:

Orthopedic Braces and Support System Market:The global orthopedic braces and support system market was valued at aroundUS$ 3 Bnin 2020, which amounts to around11%share of the overall orthopedic devices market. Sales of orthopedic braces and support systems are slated to accelerate at a CAGR of6%to topUS$ 5.5 Bnby 2031. Demand for knee braces and supports is set to increase at a CAGR of5%across the assessment period of 2021 to 2031.

Orthopedic Power Tools Market:The global orthopedic power tools market is estimated atUSD 2.2 Billionin 2022 and is forecast to surpassUSD 3.5 Billionby 2032, growing at a CAGR of4.8%from 2022 to 2032.North America orthopedic power tools market accounts for the largest market share of24.8%.The escalating online presence of players with a strong distribution network coupled with well-established healthcare infrastructure is one of the key factors fueling the market growth.

Orthopedic Footwear Market:The global orthopedic footwear market is majorly driven by rise in the number of accidents, which is the major cause of orthopedic injury. In addition to this, increase in the availability as well as variability of orthopedic footwear in various applications also promotes the market growth. In context of this, about 6% of the U.S. population has foot injuries, bunions and flat feet or fallen arches each year. About 60% of U.S. population older than 17 are suffering from foot and ankle related injuries, sprains and strains of the ankle.

Bone Biopsy Systems Market:The global bone biopsy systems market is set to enjoy a valuation ofUS$ 227.6 millionin 2022 and expand at aCAGR of 6%to reachUS$ 408.9 millionby the end of 2032.Sales of bone biopsy systems accounted for more than30%of the global bone biopsy market at the end of 2021.Bone biopsy and bone marrow biopsy sampling have been one of the most painful experiences for patients. Efforts towards reducing this pain has led to the development of powered bone biopsy systems with increased efficiency.

Bone Marrow Processing Systems Market:A bone marrow processing system is a functionally closed, sterile system designed for automatically isolating and concentrating stem cells derived from donated bone marrow aspirate. Rising applications of bone marrow transplant procedures and bone marrow donation procedures used in the treatment of bone marrow cancers, such as acute leukemia, multiple myeloma, immune deficiency disorders, aplastic anemia, spinal fusions, lymphomas, non-union fractures, osteonecrosis and other rare genetic diseases of the bone marrow, is the primary driver in the market.

Bone Growth Stimulator Market:Bone growth stimulator market was nearly worthUS$ 1.8Bn in 2020 and is anticipated to expand1.6xover the forecast period, anticipated to reach a valuation ofUS$ 3Bn by 2031. In the short-run, bone growth stimulators revenue is likely to topUS$ 1.9Bn by 2022.The market for bone growth stimulators is dominated by North America. This is mostly due to the region's expanding elderly population and the growing burden of orthopedic illnesses. As of 2031, the U.S is expected to register a CAGR worth 5%.

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Increasing Road Accidents and Fall Injuries among Aged Population Primarily Driving Need for Orthopedic Navigation Systems: Fact.MR Analysis - Yahoo...

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How do bone marrow transplants work, and what conditions do they treat?

A bone marrow transplant is actually a misnomer, as these procedures transplant stem cells, not the actual bones. Specifically, these procedures use hematopoietic stem cells (HSC), also known as blood-forming stem cells, to potentially cure an ever-expanding number of diseases.

There are three main cell types found inside a persons blood based on their function:

red blood cells: these cells carry oxygen throughout the body

platelets: these cells help form clots to stop bleeding

white blood cells: these cells lead the charge in fighting infections (also known as the immune system)

Each of these cell types, despite their different functions, shapes, sizes and lifespan, arise from the same source the hematopoietic stem cell, which constantly replenish each cell type. HSCs reside almost exclusively deep inside our bones in the center of the hard, protective shelter of calcium and other minerals. So, the marrow (soft, middle portion of our bones) can be thought of as the factory that supply each person with the blood cells needed to overcome infections, trauma, and to live a healthy, long life.

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When we perform a transplant, we are actually either using a patients own stem cells (autologous transplant) or stem cells from another human (allogeneic transplant), leading to the more appropriate name of hematopoietic stem cell transplant (HSCT). These transplants are most commonly used to treat and cure cancer.

Autologous transplants are used in the treatment of many types of solid tumors (such as brain tumors, germ cell tumors, neuroblastoma), where the tumor can only be effectively destroyed by giving very high doses of chemotherapy that also damage the patients own HSCs. Before giving a patient those high doses of chemotherapy, we collect his or her own HSCs with a process very similar to dialysis (we remove stem cells from their blood), and then freeze and store them in a specialized lab.

After the patient receives that high dose of chemotherapy, the treatment team then thaws the stem cells and infuses them back into the patient via a specialized catheter placed in his or her veins. The stem cells quickly return home and find the bone marrow space, and within 10 to 21 days, they will start making new white blood cells and platelets, followed by red blood cells.

Allogeneic transplants are performed for many types of leukemias or bone marrow failure syndromes (such as aplastic anemia or Fanconi anemia) where the patients own stem cells are broken and need to be replaced by a healthy humans stem cells. However, many other non-malignant conditions (not cancer) can be effectively cured with this procedure, as conditions that result from defects of different blood cell types (red blood cells, white blood cells or platelets) are corrected when the factory is replaced with a healthy donors stem cells.

This is an exciting time in the field of transplant, as we are now able to offer cures for many childhood diseases that historically are chronic and/or life-threatening. HSCT is now being offered to patients with sickle cell disease/thalassemia (red blood cells are defective), along with many conditions that are now called inborn errors of immunity (white blood cells are defective).

Among the more than 500 different genetic conditions that damage white blood cells include severe combined immunodeficiency (SCID), hemophagocytic lymphohistiocytosis (HLH), chronic granulomatous disease (CGD), and severe congenital neutropenia (SCN). Not only are the numbers of conditions potentially cured with HSCT rapidly growing, but the success rates and ability to prevent and treat complications of this procedure are improving exponentially as well. We are looking forward to offering these procedures to more children here at UVa Childrens.

To learn more about Dr. Roehrs and the care he provides, visit uvahealth.com/findadoctor/profile/philip-a-roehrs.

Dr. Philip Roehrs is the clinical director for pediatric stem cell transplant and cellular therapy at UVa Childrens and UVa Health.

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Ask the Expert: How do bone marrow transplants work, and what conditions do they treat? - The Daily Progress

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City of Hope recently shared significant news at the 24th Annual AIDS Conference about a patient treated in 2019 whose HIV has been in remission. The man had been living with HIV for 31 years before coming to City of Hope with another grave diagnosisacute myeloid leukemia.One of the best hopes for long-term remission of acute myeloid leukemia (AML) is a stem cell transplant, and City of Hope has one of the nations leading transplant programs, having performed more than 17,000 transplants since 1976. In addition, the institution is at the forefront of using transplants to treat older adults with blood cancers, including increasing efficacy and safety in those over 60 and those with comorbidities, like the then 63-year-old City of Hope patient with HIV. The research was presented by Jana K. Dickter, M.D., City of Hope associate clinical professor in the Division of Infectious Diseases.

City of Hope hematologist Ahmed Aribi, M.D., assistant professor in the Division of Leukemia, prepared the patient for an allogeneic blood stem cell transplant with a chemotherapy-based, reduced-intensity regimen developed for treatment of older patients with blood cancers. Reduced-intensity chemotherapy makes the transplant more tolerable for older patients and reduces the potential for transplant-related complications from the procedure.

Aribi and his team worked with City of Hopes Unrelated Donor BMT Program directed by Monzr M. Al Malki, M.D. to find a donor who was a perfect match for the patient and had the rare genetic mutation, homozygous CCR5 Delta 32, which is found in just 1 to 2% of the general population.

People who have this mutation have a resistance to acquiring HIV. CCR5 is a receptor on CD4+ immune cells, and most strains of HIV use that receptor to enter and attack the immune system. But the CCR5 mutation blocks that pathway, which stops HIV from replicating.

After this successful transplant for both AML and HIV, the patient has been in remission for HIV since stopping ART in March 2021. While this outcome has happened in three other patients, the City of Hope patient was both the oldest to undergo a transplant with HIV and leukemia and go into remission for both. He had also lived with HIV the longest 31 years.

The City of Hope patient is another major advancement. It demonstrates that research and clinical care developed and led at City of Hope are changing the meaning of an HIV diagnosis for patients across the United States and the world, said John Zaia, M.D., director of City of Hopes Center for Gene Therapy, Aaron D. Miller and Edith Miller Chair for Gene Therapy and a leader in HIV research. City of Hope remains at the forefront of clinical research that changes peoples lives for the better.

When I was diagnosed with HIV in 1988, like many others, I thought it was a death sentence. I never thought I would live to see the day that I no longer have HIV. City of Hope made that possible, and I am beyond grateful. The City of Hope patient

The story above is one significant example of several important advances being made at City of Hope in the care of people with HIV. When many centers still treated patients with low-intensity, noncurative treatment approaches for HIV-related lymphoma, City of Hope challenged that paradigm by demonstrating that autologous transplantation could be used to cure patients who would otherwise die.

More recently, City of Hope is leveraging its leadership in CAR T cell therapya groundbreaking treatment currently used to rally the bodys natural defenses against cancer and exploring its potential in tandem with another advance, City of Hopes vaccine for cytomegalovirus (CMV).

In a proof-of-concept study, funded by theCalifornia Institute for Regenerative Medicine, lab models demonstrated that the combination therapy could recognize and eliminate HIV without serious toxicity to cells in the virus host. In cultured human cells, the CAR T cells killed cells tagged with the gp120 protein, and kept killing them, without significant signs of risking damage to healthy cells. In a mouse model for HIV/AIDS, high doses of the dual-action CAR T cells followed by the CMV vaccine were successful in controlling HIV, and even nestled into the bone marrow, indicating potential for treatment to keep working over the long term.

In addition to achieving breakthrough outcomes in cancer and HIV, City of Hope has been recognized as the seventh "Best Hospital" for cancer in the nation according to U.S. News & World Report's 2022-23 Best Hospitals: Specialty Ranking. This marks the first time the cancer treatment center has cracked the top 10 of the U.S. News & World Report annual rankings and the 16th consecutive year it has been distinguished as one of the nation's elite cancer hospitals. It was also rated as high performing in four cancer surgery specialties: lung, colon, prostate and ovarian cancers.

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From optimized stem cell transplants to CAR T cell therapy: Advancing options for cancer, HIV and more - City of Hope

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So far, Bcells havent gotten the same attentionindeed, genetically engineered versions have never been tested in a human. Thats partly because engineering B cells is not that easy, says Xin Luo, a professor at Virginia Tech who in 2009 demonstrated how to generate B cells that have an added gene.

That early work, carried out at Caltech, explored whether the cells could be directed to make antibodies against HIV, perhaps becoming a new form of vaccination.

While that idea didnt pan out, now biotech companies like Immusoft, Be Biopharma, and Walking Fish Therapeutics want to harness the cells as molecular factories to treat serious rare diseases. These cells are powerhouses for secreting protein, so thats something they want to take advantage of, says Luo.

Immusoft licensed the Caltech technology and got an early investment from Peter Thiels biotech fund, Breakout Labs. Company founder Matthew Scholz, a software developer, boldly predicted in 2015 that a trial could start immediately. However, the technology the company terms immune-system programming didnt turn out to be as straightforward as coding a computer.

Ainsworth says Immusoft had to first spend several years working out reliable ways to add genes to B cells. Instead of using viruses or gene editing to make genetic changes, the company now employs a transposona molecule that likes to cut and paste DNA segments.

It also took time to convince the FDA to allow the trial. Thats because its known that if added DNA ends up near cancer-promoting genes, it can sometimes turn them on.

The FDA is concerned if you are doing this in a B cell, could you develop a leukemia situation? That is something that they are going to watch pretty closely, says Paul Orchard, the doctor at the University of Minnesota who will be recruiting patients and carrying out the study.

The first human test could resolve some open questions about the technology. One is whether the enhanced cells will take up long-term residence inside peoples bone marrow, where B cells typically live. In theory, the cells could survive decadeseven the entire life of the patient. Another question is whether theyll make enough of the missing enzyme to help stall MPS, which is a progressive disease.

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A new gene therapy based on antibody cells is about to be tested in humans - MIT Technology Review

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