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FDA Fast Tracks BCMA-Targeted CAR T-Cell Therapy for R/R … – AJMC.com Managed Markets Network

By Stem Cell Medical Center

A version of this article was originally published on OncLive. This version has been lightly edited.

The FDA has granted a fast track designation to CB-011, a CRISPR-edited allogeneic chimeric antigen receptor (CAR) T-cell therapy developed by Caribou Biosciences, for the treatment of patients with relapsed/refractory multiple myeloma.1

CB-011 targets the B-cell maturation antigen (BCMA). This is the first allogeneic anti-BCMA CAR T-cell therapy engineered to improve duration of antitumor response through an immune cloaking, genome-editing approach that removes the B2M protein and inserts a B2MHLA-E fusion protein, according to the company.2

In November 2022, the FDA cleared an Investigational New Drug application for CB-011.2 Now, the product is under evaluation in the multicenter, open-label study CaMMouflage trial (NCT05722418).3,4

Fast Track designation for CB-011 allows us instrumental interactions with the FDA as we progress our clinical development and regulatory plans for CB-011, Syed Rizvi, MD, chief medical officer at Caribou Biosciences, stated in a press release.1 This designation could not be [timelier,] as we recently dosed our first patient in the phase 1 CaMMouflage trial.

The CaMMouflage trial will include patients who have received at least 3 prior lines of therapy. Eligible patients are also required to have an ECOG performance status of 0 or 1 and to have adequate hematologic, renal, hepatic, pulmonary, and cardiac function.3

Patients who have received a BCMA-targeted therapy within 3 months of enrollment and/or any prior CAR T-cell therapy are excluded. Those who received autologous stem cell transplant within 6 weeks of lymphodepletion or allogeneic stem cell transplant within 6 months of lymphodepletion, who have an active or prior history of central nervous system involvement, or who have received a live, attenuated vaccine with 4 weeks of lymphodepletion are not eligible.3

In the part A, the escalation stage of the trial, investigators will assess ascending doses of CB-011 in combination with cyclophosphamide and fludarabine to establish the recommended phase 2 dose (RP2D) of the regimen in a standard 3+3 dose-escalation design. For part B, the expansion stage, investigators will assess responses to the RP2D, or the maximum tolerated dose as measured by International Myeloma Working Group criteria.3

Recruitment for CaMMouflage is ongoing. Caribou shared plans to assign patients to a single administration of CB-011 at a dose of 50 x 106 CAR T cells this year.1

Cas12a CRISPR hybrid RNA-DNA (chRDNA) technology was utilized to make 4 gene edits in the creation of CB-011. In edits 1 and 2, investigators target cancer cells by site-specifically inserting a humanized anti-BCMA CAR into the TRAC gene. This mechanism knocks out T cell receptor expression, thereby reducing the risk for graft-vs-host disease (GVHD).2

In edits 3 and 4, investigators prevent recognition and rejection by patient T cells and blunt rejection by natural killer (NK) cells by site-specifically inserting a B2MHLA-E peptide fusion gene into the B2M gene of the CAR T cells. These edits disable endogenous B2M expression, which eliminates endogenous HLA class I presentation and reduces T cell-mediated rejection. At the same time, these changes facilitate expression of B2MHLA-E which reduces rejection caused by NK cells.2

Prior preclinical findings presented at the 2022 AACR Annual Meeting indicated that the CAR T cells expressing the B2M-HLA-E fusion induced a survival benefit compared with cells that do not express the fusion in the presence of NK cells in vitro.4 This result suggests that these cells may be able to fight off a recipients NK cells and circulate longer.

Furthermore, CB-011 induced long-term survival in mice carrying established orthotopically-engrafted multiple myeloma cells. Mice who received high doses of CB-011 also appeared to avoid GVHD.4

Our goal is to develop CB-011 as a readily available off-the-shelf treatment option for patients with relapsed or refractory multiple myeloma to overcome the need for apheresis or bridging therapy, variable quality and long manufacturing timelines, manufacturing failures, or the inability to bear the burden of treatments that require frequent dosing over several months, Rizvi added in the press release.1

CB-011 is Caribous second allogeneic CAR T-cell agent that is under exploration in hematologic malignancies.

Investigators are assessing CB-010, a CAR T-cell therapy with a PD-1 knockout, in patients with relapsed or refractory B cell non-Hodgkin lymphoma as part of the ongoing phase 1 ANTLER trial NCT04637763).2 One patient in that trial who received 8 prior lines of systemic therapy had an ongoing complete response through month 15.5

References

1. Caribou Biosciences announces FDA granted fast track designation to CB-011, an allogeneic CAR-T cell therapy for relapsed or refractory multiple myeloma. News release. Caribou Biosciences. April 4, 2023. Accessed April 6, 2023. https://investor.cariboubio.com/

2. Caribou Biosciences announces FDA clearance of IND application for CB-011, an allogeneic anti-BCMA CAR-T Cell therapy for the treatment of relapsed or refractory multiple myeloma. News release. Caribou Biosciences. November 21, 2022. Accessed April 6, 2023. https://investor.cariboubio.com/news-releases/

3. CRISPR-edited allogeneic anti-BCMA CAR-T cell therapy in patients with relapsed/refractory multiple myeloma (CaMMouflage). Updated March 2, 10, 2023. Accessed April 6, 2023. https://www.clinicaltrials.gov/ct2/show/NCT05722418

4. Garner E, Degagne E, Roy S, et al. A BCMA-specific allogeneic CAR-T cell therapy (CB-011) genome-engineered to express an HLA-E fusion transgene to prevent immune cell rejection. Cancer Res. 2022;82(suppl 12):LB009. doi:10.1158/1538-7445.AM2022-LB009

5. CRISPR-edited allogeneic anti-CD19 CAR-t cell therapy with PD-1 knockout induces prolonged complete response in relapsed/refractory follicular lymphoma patient: case report from CB-010 ANTLER trial. Presented at: the Lymphoma, Leukemia, & Myeloma Congress; New York, NY; October 18-22, 2022.

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FDA Fast Tracks BCMA-Targeted CAR T-Cell Therapy for R/R ... - AJMC.com Managed Markets Network

Bazedoxifene and cholecalciferol | DDDT – Dove Medical Press

By Stem Cell Medical Center

Introduction

Osteoporosis is a skeletal disorder characterized by low bone mass and an increased risk of bone fracture. Bone mass reaches its peak in young adulthood, and increased bone resorption relative to bone formation leads to the lowering of bone mass. According to the World Health Organization, osteoporosis is defined as hip or lumbar spine bone marrow density of more than 2.5 standard deviations below the mean found in the young-adult population.1 Osteoporosis is a significant public health concern among the elderly due to the increased morbidity and mortality associated with bone fractures. It affects both sexes and all races, and its prevalence is expected to rise as the global population continues to age.1

The pharmacologic agents for the prevention and treatment of osteoporosis include calcium, vitamin D, bisphosphonates, calcitonin, estrogen, selective estrogen receptor modulator (SERM), parathyroid hormone, and anti-receptor activator of nuclear factor-kappa B ligandantibody (denosumab).2 The therapeutic potential of new treatment approaches, such as exosomes derived from endothelial cells or mesenchymal stem cells, has been investigated recently in animal models.3,4

Bazedoxifene, a third-generation SERM, exhibits tissue-selective action, functioning as an agonist in skeletal tissue but as an antagonist in breast and uterine tissues.5,6 Bazedoxifene has been approved for the treatment of postmenopausal osteoporosis by the European Medicines Agency, and conjugated estrogens/bazedoxifene have been approved for the prevention of postmenopausal osteoporosis by the United States Food and Drug Administration.7,8 Cholecalciferol, commonly known as vitamin D3, plays an important role in bone metabolism. Cholecalciferol is converted to calcifediol (25-hydroxycholecalciferol) in the liver and then to calcitriol (1,25-dihydroxycholecalciferol), the active form of vitamin D, in the kidney. Although vitamin D can be obtained from dietary intake and synthesized in the skin after exposure to sunlight, its deficiency is common. The National Osteoporosis Foundation recommends 8001000 IU of vitamin D intake per day for individuals aged 50 and older.1

A fixed-dose combination formulation of bazedoxifene and cholecalciferol is a potentially promising therapeutic option for postmenopausal osteoporosis patients as it can improve treatment efficacy and medication compliance. Before developing a fixed-dose combination formulation, the pharmacokinetic (PK) interactions between bazedoxifene and cholecalciferol need to be assessed. This study aimed to examine the PK interactions between these drugs as well as their safety and tolerability when co-administered in healthy male subjects.

The study was designed as an open-label, randomized, three-period, three-treatment, six-sequence, crossover clinical trial. Healthy male volunteers aged between 19 and 40 years with body mass index (BMI) between 19 and 28 kg/m2 were eligible for inclusion. Subjects were screened based on past medical history, physical examination including vital signs (blood pressure, heart rate, and body temperature), urine drug screening, clinical laboratory tests, serology tests, and 12-lead electrocardiogram (ECG). Subjects with a history of venous thromboembolism, hypercalciuria, renal stone, hepatobiliary disease, galactose intolerance, drug abuse, or clinically significant hypersensitivity reaction were excluded.

A total of 30 subjects were randomly assigned to six sequences comprised of three treatments: one tablet of 20 mg bazedoxifene (Viviant; Pfizer Ltd., Seoul, Korea), two tablets of 800 IU cholecalciferol (HGP1501; Hanmi Pharmaceutical Co. Ltd., Seoul, Korea), or one tablet of 20 mg bazedoxifene along with two tablets of 800 IU cholecalciferol (Table 1). Outpatient visits were scheduled on day 10 of period 1 and day 9 of periods 2 and 3. At that time, subjects were provided with sunscreens (sun protection factor 50) and diaries to record their diet and activity. Subjects were asked to limit their exposure to sunlight by applying sunscreen and covering themselves with clothes and hats for outdoor activities. Subjects were required to refrain from taking dietary supplements and foods high in vitamin D content. Between two consecutive periods, there was a washout period of at least 14 days. These subjects were admitted to the Clinical Trial Center at Asan Medical Center (Seoul, Korea) the day before drug administration. The test drugs were administered orally under fasting conditions with 150 mL of water. After receiving the investigational drug(s), subjects were required to fast for four hours. Depending on the treatment, subjects were discharged on either day 2 (bazedoxifene monotherapy) or day 6 (cholecalciferol monotherapy or combined therapy). A follow-up visit was scheduled two weeks after the last dose.

Table 1 Overall Study Design

To determine the plasma bazedoxifene concentration, serial blood samples were collected at 0 (pre-dose), 1, 1.5, 2, 3, 4, 5, 6, 8, 10, 12, 14, 24, 48, 72, 96, and 120 hours after the dosing. To measure the baseline cholecalciferol level, blood samples were collected at 16, 12, 8, and 0 hours before the cholecalciferol dosing, and the average concentration for each individual was used as the baseline cholecalciferol concentration. Baseline-adjusted cholecalciferol concentrations were obtained by subtracting the baseline from the post-dose plasma cholecalciferol concentrations measured at 1, 1.5, 2, 3, 4, 5, 6, 8, 10, 12, 14, 16, 24, 48, 72, 96 and 120 hours after the treatment. If the baseline-adjusted cholecalciferol concentration had a negative value, zero was assigned.

Blood samples used for the drug concentration measurements were drawn into ethylenediaminetetraacetic acid K2 tubes. Plasma was separated by centrifugation at 1800 g for 8 minutes at 4 C and stored in Eppendorf tubes at 70 C until analysis. At the analytical laboratory (BioCore Co. Ltd., Seoul, Korea), samples were thawed at room temperature. The plasma concentrations of bazedoxifene and cholecalciferol were assayed using validated liquid chromatography with tandem mass spectrometry (LC-MS/MS). Bazedoxifene samples were prepared by liquid-liquid extraction, and cholecalciferol samples by protein precipitation and solid-phase extraction. Liquid chromatography was conducted using a Shimadzu UFLC system (Shimadzu Corp., Kyoto, Japan). For tandem mass spectrometry, a SCIEX TQ5500 mass spectrometer (AB Sciex LLC, MA, USA) was used. The calibration curves covered the ranges of 0.120 ng/mL for bazedoxifene and 0.110 ng/mL for cholecalciferol. For bazedoxifene, the accuracy of LC-MS/MS was 91.8113.0%, and the precision was 0.15.5%. For cholecalciferol, the accuracy of LC-MS/MS was 92.7105.6%, and the precision was 0.24.5%. The lower limit of quantification was 0.1 ng/mL for both compounds.

The study protocol was approved by the Institutional Review Board (IRB) of Asan Medical Center (IRB number: 20170227) and by the Korean Ministry of Food and Drug Safety. The study was registered at ClinicalTrials.gov (NCT03089112). All subjects provided written informed consent before receiving screening tests. All study procedures were conducted in accordance with the ethical principles stated in the Declaration of Helsinki and by the Good Clinical Practice Guidelines of the International Council for Harmonisation.9,10 The study was conducted from March 31, 2017 to June 16, 2017.

The PK parameters of bazedoxifene and cholecalciferol in each subject were analyzed by the non-compartmental method using Phoenix WinNonlin version 6.4 (Certara, NJ, USA). This analysis was based on the actual sampling time. The maximum plasma concentration (Cmax) and the time to reach Cmax (Tmax) were determined from the observed values. The terminal elimination rate constant (z) was estimated by linear regression analysis of the terminal portion of the semilogarithmic concentration-time curve. The terminal elimination half-life (t1/2) was calculated as the natural log of two divided by z. Demographic data and PK parameters were summarized using descriptive statistics. The Cmax and the values for the area under the plasma concentration-time curve from time zero to the last quantifiable concentration (AUClast) were log-transformed to compare the drug exposure between the combined therapy and monotherapy. The point estimate and 90% confidence interval (CI) for the geometric mean ratio (GMR) of the combined therapy to monotherapy were obtained for Cmax and AUClast.

Safety and tolerability were assessed throughout the study by physical examinations, laboratory tests (complete blood count, blood chemistry, and urinalysis), 12-lead ECG, and monitoring of adverse events (AEs). AEs were coded according to the Medical Dictionary for Regulatory Activities (MedDRA version 19.1) and recorded in terms of symptom/sign, onset, duration, severity, relationship to the investigational drug(s), action taken, and outcome.

Of the 30 study participants who received the investigational drug(s) at least once, 27 subjects completed the study (Figure 1). Two subjects withdrew their consent to participate, and one subject was excluded at the investigators discretion due to a medical need for concomitant medication that could potentially affect the PK data. Participant demographics, including age, weight, height, and BMI, are summarized in Table 2. The mean standard deviation (SD) values were 28.63 4.18 years for age, 71.53 6.99 kg for weight, 173.55 4.80 cm for height, and 23.75 2.09 kg/m2 for BMI. All subjects who received the investigational drug(s) at least once were included in the safety and tolerability assessment.

Table 2 Demographic Characteristics of the Participants (n=30)

Figure 1 Subject disposition.

The 27 subjects who completed the study were included in the PK analysis set, and the PK parameters are presented in Table 3. For bazedoxifene, the Cmax values (mean SD) for monotherapy and combined therapy were 3.30 0.92 ng/mL and 3.62 1.39 ng/mL, respectively. The AUClast values (mean SD) for monotherapy and combined therapy were 44.32 22.24 hng/mL and 50.78 24.98 hng/mL, respectively. The mean plasma concentration-time profiles of bazedoxifene for monotherapy and combined therapy nearly overlapped (Figure 2A). For bazedoxifene, the GMR (90% CI) of the combined therapy to monotherapy was 1.044 (0.92631.1765) for Cmax and 1.1329 (1.02321.2544) for AUClast (Table 4).

Table 3 Pharmacokinetic Parameters of Bazedoxifene and Baseline-Adjusted Cholecalciferol in Healthy Male Subjects (n=27)

Table 4 Geometric Mean Ratios of Combined Therapy to Monotherapy for AUClast and Cmax

Figure 2 Mean plasma concentration-time curves: (A) bazedoxifene (B) baseline-adjusted cholecalciferol. The error bars denote standard deviations.

For baseline-adjusted cholecalciferol, the Cmax values (mean SD) for monotherapy and combined therapy were 2.25 0.47 ng/mL and 1.95 0.54 ng/mL, respectively. The AUClast values (mean SD) for monotherapy and combined therapy were 59.43 22.95 hng/mL and 49.18 24.40 hng/mL, respectively. The mean plasma concentration-time profiles of baseline-adjusted cholecalciferol for monotherapy and combined therapy are shown in Figure 2B. For baseline-adjusted cholecalciferol, the GMR (90% CI) of combined therapy to monotherapy was 0.8543 (0.80050.9117) for Cmax and 0.8056 (0.74450.8717) for AUClast (Table 4).

Seventeen subjects showed a total of 26 AEs after drug administration (Table 5). No serious AE occurred throughout the entire study. All AEs were mild in severity and resolved without any sequelae. The most common AE was nasopharyngitis (five events in five subjects). Clinically significant findings from the physical examination were phlebitis of the arm and venipuncture site bruise. Clinically significant laboratory abnormalities included increased blood levels of creatine phosphokinase and triglyceride and decreased blood levels of neutrophil count. No clinically significant abnormalities were found with respect to vital signs and 12-lead ECG. Twenty-five AEs were considered to have no or unlikely relationship with the investigational drugs. One AE, fatigue observed after combined therapy, was considered to have a possible relationship with the administered drugs.

Table 5 Adverse Events in the Study Population After Drug Administration

This study investigated the PK characteristics of bazedoxifene and cholecalciferol, their PK interactions, and the safety and tolerability of combined therapy with these drugs in healthy male subjects.

The terminal elimination half-life of bazedoxifene was approximately 27 hours, and that of cholecalciferol was 13 hours in our study. The washout period of 14 days should therefore have been sufficient for both investigational drugs to be fully eliminated by the next period. The blood samples obtained up to 120 hours after dosing provided sufficient information for characterizing the exposures to bazedoxifene and cholecalciferol.

Previous studies on bazedoxifene pharmacokinetics have indicated a Tmax of 12 hours and a half-life of approximately 28 hours.11 As shown in Figure 2 and Table 3, bazedoxifene was rapidly absorbed when administered alone or in combination with cholecalciferol, showing median Tmax values of 2.00 h and 1.53 h, respectively. The Tmax of cholecalciferol is known to be approximately 15 hours. In our study, the median Tmax of cholecalciferol was 11.98 h when administered alone, similar to the 12.00 h value found for its co-administration with bazedoxifene.

In a recent study on bazedoxifene, the mean Cmax and AUClast in healthy male subjects after a single 20 mg oral dose were 3.191 ng/mL and 44.697 hng/mL, respectively.12 These numbers are consistent with the results of the current study (3.30 ng/mL and 44.32 hng/mL). The PK parameters Cmax and AUClast were used in our analyses to compare the combined therapy and monotherapy exposures. With bazedoxifene, the 90% CI for the GMR of the combined therapy to bazedoxifene monotherapy for Cmax fell within the 0.801.25 range, and for AUClast it was nearly within the above-mentioned range.

In the case of cholecalciferol, the AUClast was lower when this drug was co-administered with bazedoxifene, as indicated by the GMR of 0.8056 and associated 90% CI of 0.74450.8717. Bazedoxifene and cholecalciferol are drugs that exhibit wide therapeutic ranges.1315 Cholecalciferol is known to reach a sigmoidal dose-response curve plateau at a dose of 600 IU.15 Hence, based on the GMRs observed in our exploratory study, the difference in the AUClast for cholecalciferol is unlikely to have clinical significance in treatment efficacy at the dose level used in our protocol.

All of the AEs observed in our study population were mild, and the frequency of AEs in subjects who received combined therapy was not significantly different from that in subjects who received monotherapy. The most common AE observed in our present trial cohort was nasopharyngitis, which we considered unrelated to the investigational drugs.

One limitation of the present study was that the participants were all healthy men. The study was exploratory in nature, and male subjects were included to facilitate enrollment and minimize health risks to subjects. Drug-drug interaction studies are often conducted with male subjects, and the results are applied to women as well as men because gender does not seem to affect the pattern of drug-drug interaction for many drugs. Assuming that the PK interactions would follow the same pattern in men and women, this study was conducted in men instead of women. Additional studies in postmenopausal women with osteoporosis and patients with different pathophysiological conditions will help further characterize the PK interactions between bazedoxifene and cholecalciferol.

The combined therapy of 20 mg bazedoxifene (one tablet) and 1600 IU cholecalciferol (two tablets of 800 IU) was safe and well tolerated in healthy male subjects. In this present study, the oral co-administration of bazedoxifene with cholecalciferol tended to decrease the cholecalciferol exposure slightly. Considering that both drugs have wide therapeutic ranges, dose adjustment of cholecalciferol may not be necessary for its co-administration with bazedoxifene.

AE, adverse event; AUClast, area under the concentration-time curve from time zero to the last quantifiable concentration; BMI, body mass index; CI, confidence interval; Cmax, maximum plasma concentration; ECG, electrocardiogram; GMR, geometric mean ratio; IRB, Institutional Review Board; LC-MS/MS, liquid chromatography with tandem mass spectrometry; z, terminal elimination rate constant; PK, pharmacokinetic; SD, standard deviation; SERM, selective estrogen receptor modulator; Tmax, time to reach Cmax; t1/2, terminal elimination half-life.

The authors do not intend to share individual de-identified participant data of this study due to confidentiality.

This study was sponsored by Hanmi Pharmaceutical Co. Ltd.

Hyeong-Seok Lim has received grants from Hanmi Pharmaceutical Co. Ltd. for a range of research projects, including the study reported in this article. Jina Jung and Sung Hee Hong are employees of Hanmi Pharmaceutical Co. Ltd. The remaining authors declare no competing interests in relation to this article.

1. Cosman F, de Beur SJ, LeBoff MS, et al. National Osteoporosis Foundation. Clinicians guide to prevention and treatment of osteoporosis. Osteoporos Int. 2014;25(10):23592381. doi:10.1007/s00198-014-2794-2

2. Rejnmark L, Mosekilde L. New and emerging antiresorptive treatments in osteoporosis. Curr Drug Saf. 2011;6(2):7588. doi:10.2174/157488611795684686

3. Song H, Li X, Zhao Z, et al. Reversal of osteoporotic activity by endothelial cell-secreted bone targeting and biocompatible exosomes. Nano Lett. 2019;19(5):30403048. doi:10.1021/acs.nanolett.9b00287

4. Zeng ZL, Xie H. Mesenchymal stem cell-derived extracellular vesicles: a possible therapeutic strategy for orthopaedic diseases: a narrative review. Biomater Transl. 2022;3(3):175187. doi:10.12336/biomatertransl.2022.03.002

5. Komm BS, Kharode YP, Bodine PV, Harris HA, Miller CP, Lyttle CR. Bazedoxifene acetate: a selective estrogen receptor modulator with improved selectivity. Endocrinology. 2005;146(9):39994008. doi:10.1210/en.2005-0030

6. Stump AL, Kelley KW, Wensel TM. Bazedoxifene: a third-generation selective estrogen receptor modulator for treatment of postmenopausal osteoporosis. Ann Pharmacother. 2007;41(5):833839. doi:10.1345/aph.1H428

7. Conbriza. European medicines agency; 2009. Available from: https://www.ema.europa.eu/en/medicines/human/EPAR/conbriza. Accessed October 21, 2022.

8. DUAVEE. Drug Label (NDA 022247). Silver Spring, MD: US Food and Drug Administration; 2013.

9. World Medical Association. World medical association declaration of Helsinki: ethical principles for medical research involving human subjects. JAMA. 2013;310(20):21912194. doi:10.1001/jama.2013.281053

10. Guideline for Good Clinical Practice E6(R2). International council for harmonisation of technical requirements for pharmaceuticals for human use; 2016. Available from: https://database.ich.org/sites/default/files/E6_R2_Addendum.pdf. Accessed October 21, 2022.

11. Gatti D, Rossini M, Sblendorio I, Lello S. Pharmacokinetic evaluation of bazedoxifene for the treatment of osteoporosis. Expert Opin Drug Metab Toxicol. 2013;9(7):883892. doi:10.1517/17425255.2013.794221

12. Yeun JS, Kan HS, Lee M, et al. Pharmacokinetic comparison of two bazedoxifene acetate 20 mg tablet formulations in healthy Korean male volunteers. Transl Clin Pharmacol. 2020;28(2):102108. doi:10.12793/tcp.2020.28.e7

13. Archer DF, Pinkerton JV, Utian WH, et al. Bazedoxifene, a selective estrogen receptor modulator: effects on the endometrium, ovaries, and breast from a randomized controlled trial in osteoporotic postmenopausal women. Menopause. 2009;16(6):11091115. doi:10.1097/gme.0b013e3181a818db

14. Kawate H, Takayanagi R. Efficacy and safety of bazedoxifene for postmenopausal osteoporosis. Clin Interv Aging. 2011;6:151160. doi:10.2147/CIA.S15711

15. Institute of Medicine. Dietary Reference Intakes for Calcium and Vitamin D. Washington, DC: The National Academies Press; 2011.

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Bazedoxifene and cholecalciferol | DDDT - Dove Medical Press

Paterson Teacher Dies After Battling Brain Infection, Cancer – Daily Voice

By Stem Cell Medical Center

EmilySetteducato has died after battling cancer and a subsequent brain infection on Saturday, April 8, her obituary reads. She was 28 years old.

After battling leukemia, braving multiple rounds of chemotherapy, and receiving a life-saving stem cell transplant from an anonymous donor at just 27 years old, Emily thought her medical woes were over.

The Paterson elementary school teacher had left the hospital, returned to the classroom, celebrated a birthday, and was back to planning a future with her long-time boyfriend, Will.

The future was looking bright for Emily, until she contracted an infection known as the JC Virus, wreaking havoc on her brain.

Emily sister,Jackie Griffin launched a GoFundMe campaignlast January. While Emily was able to undergo treatment atCincinnati Children's Hospital, "recent test results show that the minor improvements were only temporary," Griffin said.

"Based on medical advice, our focus is now on making Emily as comfortable as possible through in-home care, which will require around the clock care for an unknown amount of time, possibly months."

Emily slipped away surrounded by her loved ones.

Emily's final weeks were spent showering her friends and family with love, her obituary reads, "lighting up whenever her life partner and 'her rock', Will, was nearby, making her family laugh with jokes and eyerolls, planning celebrations for her friends, and bragging about her new nephew to her many visitors," it reads.

Emily graduated Fair Lawn High School and William Paterson University, before beginning her career as a preschool teacher. She was a cherished member of the BJ Wilkerson Memorial Child Development Center and DA Quarles Early Childhood Center.

Services will be held at theVander Plaat Colonial Home. Click here for funeral details and here to donate to the GoFundMe page for Emily.

Click here to follow Daily Voice South Passaic and receive free news updates.

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Paterson Teacher Dies After Battling Brain Infection, Cancer - Daily Voice

Berlusconi says he believes he’ll recover ‘once again’ – The Associated Press

By Stem Cell Medical Center

ROME (AP) Former Italian Premier Silvio Berlusconi said he believes he will overcome the lung infection caused by a chronic leukemia that has hospitalized him in intensive care, Italian daily Il Giornale reported on Friday.

Its hard, but Ill make it once again, the 86-year-old media mogul said in a phone call with Il Giornales editor in chief, Augusto Minzolini.

I managed, also in delicate and difficult situations, to pull myself up, Berlusconi added, according to the report.

The media tycoon and three-time premier spent a second consecutive night in intensive care at Milans San Raffaele Hospital, where his doctors on Thursday signed off on a medical bulletin saying that he has had leukemia for some time, but that the cancer of the blood cells is in a persistent chronic phase.

The statement was the first official word from doctors since Berlusconi was admitted to San Raffaele on Wednesday.

The current treatment strategy envisions treatment of the pulmonary infection as well as specialized treatment aimed at limiting the negative effects of the chronic leukemia, the bulletin stated.

On Friday morning, Foreign Minister Antonio Tajani, quoting Berlusconis personal doctor Alberto Zangrillo, reported that Berlusconi was alert and was responding to the treatments. Tajani is the coordinator of Forza Italia, the political party that Berlusconi created some 30 years ago.

Berlusconi was admitted to an intensive care unit at San Raffaele Hospital on Wednesday for treatment of what aides indicated was a respiratory problem stemming from a previous infection.

The former premier and now senator had left the same hospital a week earlier after several days of tests.

Doctors said the kind of leukemia afflicting Berlusconi usually appears in people of advanced age and is characterized by an increase in white blood cells known as monocytes.

Treatment for older patients can involve a stem cell transplant. But that could be difficult, according to an Italian nonprofit association dedicated to combating leukemia. Another treatment would involve controlling the white blood cell count.

The last years have seen Berlusconi suffer numerous health problems, including heart ailments and COVID-19 in 2020, which saw him hospitalized then in critical condition with pneumonia.

He has had a pacemaker for years, underwent heart surgery to replace an aortic valve in 2016 and overcame prostate cancer decades ago.

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Berlusconi says he believes he'll recover 'once again' - The Associated Press

expert reaction to study looking at creating embryo-like structures … – Science Media Centre

By Embryonic Stem Cells

April 6, 2023

A study published in Cell Stem Cell looks at the generation of embryo-like structures from monkey embryonic stem cells.

Prof Magdalena Zernicka-Goetz, Bren Professor of Biology and Biological Engineering, California Institute of Technology; and Professor of Mammalian Development and Stem Cell Biology, University of Cambridge, said:

This is an exciting development building on work from our own and other labs showing the importance of establishing interactions between embryonic and extra-embryonic stem cells to establish models of the mammalian embryo at pre-and early post-implantation stages. The excitement of this study is not only that embryos generated from monkey stem cells provide a close model for human embryos, but monkeys are also experimentally tractable.

The authors follow approaches that have been previously used to direct embryonic stem cells into a naive state, and then use treatments that allow the nave monkey ES cells to form extra-embryonic cell types. Together these cells assemble into blastoids, structures resembling blastocysts, that are able to develop in vitro into structures with a striking resemblance to the embryonic disc at gastrulation, both in morphology and gene expression. The blastoids also appear to implant into foster monkey mothers but, in common with similar structures in the mouse, development appears restricted.

This study is a hugely encouraging development in the study of primate embryo models.

The paper is excellent and an important step forward but still the stem cell derived embryos have a limited developmental potential, as the authors state themselves. Nevertheless, it is an important step in the very exciting field of enormous potential for understanding how the embryo develops and why so many pregnancies fail.

Prof Roger Sturmey, Professor of Reproductive Medicine, Hull York Medical School, University of Hull, said:

The work by Li and colleagues is an impressive technical achievement that has demonstrated the possibility that embryonic stem cells from a primate can be persuaded to form structures that mirror many features of early embryos.

Similar achievements have already been reported in other species, however this work assesses the primate embryo-like structures in detail and gives new insights into how the cell lineages families of cells that constitute the early embryo can be generated from stem cells.

Remarkably, when cultured in a laboatory, the embryo-like structures are able to replicate a number of key developmental features, most notably the formation of cells that resemble the primordial germ cells the cells that can produce gametes as well as the formation of a structure similar to the so-called primitive streak. When transferred into a recipient macaque uterus, these embryo-like structures were able to generate components of a pregnancy response, but were unable to develop, indicating that while these structures do share many features with competent embryos, there are still aspects of early development that differ between competent embryos and stem-cell derived models, preventing full development.

The work by Li and colleagues will offer important new tools in our understanding of the earliest stages of embryo development, but also highlight the need for guidance in this area, something that scientists in the UK are actively working on.

Prof Alfonso Martinez Arias, ICREA Senior Research Professor, Department of Experimental and Health Sciences, Universitat Pompeu Fabra (UPF), said:

This is a timely study.

About half of human pregnancies fail during the proliferation of the zygote and the implantation of the blastocyst. Understanding the causes of this failure rate will impact human fertility and IVF success. In part to address this need, over the last few years, a number of Embryonic Stem (ES) cell models of early mammalian development have been created in the lab. Amidst these, mouse and human blastoids mimic mammalian blastocysts and as such can play an important role in understanding the process of implantation. Blastoids have been derived from mouse and human ES cells.

For these studies to go forward there is a need to develop a proper test for the function of the blastocyst: its implantation into the uterus. In the case of mouse blastoids this can be tested by implanting them into females. However, there is no such a test for human blastoids since, for obvious reasons, it is not possible to implant them into a human uterus. And yet there is a need to develop a system to study these structures in humans. Mouse reproductive biology and implantation are very different from human, which means that while an excellent system to find principles, the mouse is not useful for the specifics of this process; and this is what matters. It is this vacuum of a system to study human implantation and peri-implantation development that is addressed in the present study.

Following protocols established for human blastoids, macaque blastoids are made from nave stem cells and their potential is tested in two ways. One, by culturing them in vitro up to gastrulation stages and the other, by placing them in the uterus of a macaque foster mother. The idea behind this system is that it has reduced ethical barriers compared to human and therefore might provide an experimental system to test the potential of blastoids fully and, in the long term, to study infertility. The work is well conducted and the result is clear: although at the level of single cells macaque blastoids bear a strong resemblance to blastocysts, they do not behave as blastocysts. Although they implant and initiate gastrulation, they do not reach the end of this process. In vitro, blastoids cultured to form an epiblast and to undergo gastrulation, display progressive problems over time and, though they reach early stages of gastrulation, it is difficult to see in their data how faithful they are to an early gastrula. In one important experiment they implant some of these into female macaques and follow their progress with ultrasound. It appears as if they might perform well in the early stages of implantation, and the release of progesterone is a sign that something has gone well, but then, they disappear after about a week.

So, the important result of this work is that we are not close to generating blastoids that can be recognised as blastocysts by the mother. Definitely an important proof of principle but the lesson is that there is work to do.

An important difference between a blastoid and a blatocyst is their origin. The blastocyst in the egg, the blastoid in the ES cells. There might be elements in the oocyte that are important for the viability of the blastocysts and that will not be provided by the ES cells. Furthermore, if about 50% of conceptions fail at implantation, it is difficult to gauge whether the failure of the high level goal of the experiment (long term development in the womb) is due to defects in the blastoid system or whether the failure mirrors the natural situation; eight experimental subjects, the numbers of the experiment, are not sufficient to make a judgement. Only more experiments will decide and the one reported here, within well established ethical footprints, is definitely one to watch.

Dr Darius Widera, Associate Professor in Stem Cell Biology and Regenerative Medicine, University of Reading, said:

This is an interesting study that demonstrates the successful generation of embryo-like structures from monkey embryonic stem cells. These structures resembled natural early embryonic structures and could generate cell types of all three germ layers. Although similar studies have been conducted using human stem cells, this is the first report showing that (in this case, monkey) embryo-like structures can induce signs of pregnancy if transplanted into females. Therefore, the method could be used as a model of primate and human development and potentially provide new insights into certain factors that contribute to miscarriages in humans.

However, the study has some limitations. Only 3 out of 8 embryo-like structures were successfully implanted into female monkeys, and none of these persisted for more than one week. Thus, the structures do not have full developmental potential.

In addition, the ethical implications of embryonic stem cell research in monkeys are complex. Primates are intelligent, social animals with complex cognitive and emotional lives. Therefore, it is important to carefully consider both the potential benefits and the ethical impact of primate embryonic stem cell research.

Prof Robin Lovell-Badge FRS FMedSci, Group Leader, Francis Crick Institute, said:

The paper by Jie Li et al is another demonstration of the remarkable ability of pluripotent stem cells, in this case embryonic stem cells derived from early Macaque (non-human primate) embryos, to self-organise and begin a process of embryo formation in culture that mirrors that of normal Macaque embryos. However, the paper also shows that these stem cell-based embryo models are not entirely normal they could be implanted in female macaques, appear to initiate a pregnancy, but then fail soon after.

The authors were able to culture these stem cell-based embryo models, which they refer to as blastoids, through to gastrulation stages, equivalent to post-implantation embryos developing in a uterus, with good signs of development of all the main extraembryonic and embryonic tissues, where the latter included ectoderm, mesoderm and endoderm organised in a similar fashion to normal embryos. They could also demonstrate the presence of primordial germ cell-like cells and cells that are early progenitors of the blood system. These stages would be equivalent to those of human embryos at about 16 -18 days of development, beyond the 14 day limit (or the beginning of gastrulation) which is the maximum period normal human embryos are allowed to be cultured by law in the UK and some other countries.

It has been shown by others that human pluripotent stem cells can also be used to form blastoids, but to date such cultures have been stopped prior to gastrulation, but the paper by Li et al suggests that they could indeed be taken beyond this and provide valuable information about these early stages of human development that are otherwise very difficult to obtain. The data from the Macaque embryos and blastoid cultures may also help to understand aspects of human development, but without direct comparisons this will always be tentative, given how much mammalian embryos can vary at these stages.

These embryo models are referred to as integrated stem cell-based embryo models because they include extraembryonic tissues that normally give rise to the placenta and yolk sac that in a normal conceptus would permit implantation into the uterus and support the development of the embryo proper. So how much like a real embryo are these Macaque blastoids and could they implant and develop much further in a uterus? Although all the detailed comparisons presented in the paper of gene expression in the various cell types between normal Macaque embryos and the embryo models suggests that they can be very similar, the proportion of the blastoids reaching advanced stages was very low, indicating that most are not normal, and those that did still showed some differences. Moreover, while some could implant, begin to develop some complexity, and induce a typical response in the host uterus and lead to production of the typical pregnancy hormones, chorionic gonadotrophin and progesterone, the embryos all failed before gastrulation. This suggests that they failed to form fully functional extraembryonic tissues that could adequately support the embryo and that these could not give rise to a placenta, which would be essential for more complex development. It is likely that the same would be true for human integrated stem cell-based embryo models, although it would be unethical and illegal (in the UK) to attempt to implant these into a woman.

It seems likely that the culture methods for these integrated stem cell-based embryo models will be improved, and who knows it may eventually be possible to have them implant and develop normally, but the failure of this to happen as reported in this paper will give regulators some breathing space to develop appropriate rules for the culture of such human models, notably whether they can be taken beyond the equivalent of gastrulation stages, which would be of immense importance in helping to understand not just normal development of the human embryo, but what so often goes wrong and leads to embryo failure and congenital disorders.

Cynomolgus monkey embryo model captures gastrulation and early pregnancy by Jie Li et al. was published in Cell Stem Cell at 16:00 UK time on Thursday 6 April 2023.

DOI: 10.1016/j.stem.2023.03.009

Declared interests

Prof Magdalena Zernicka-Goetz: I have no conflict of interest to declare.

Prof Roger Sturmey: None.

Prof Alfonso Martinez Arias: I have no conflict of interests.

Dr Darius Widera: I have no conflict of interest to declare.

Prof Robin Lovell-Badge: I have no conflicts of interest to declare, except I do serve on the HFEAs Scientific and Clinical Advances Advisory Committee and I am a member of their Legislative Reform Advisory Group.

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expert reaction to study looking at creating embryo-like structures ... - Science Media Centre

NANOG (Part 1): Its Role In Aging And Cancer – Forbes

By Embryonic Stem Cells

eternal youth and the mythological island that NANOG protein was named after.Patrick Lynch, URL: https://www.irishcentral.com/roots/history/tir-na-nog-legend-eternal-youth

This is the first article in a series on the NANOG protein. Please see my companion series on klotho, another protein that may be involved in aging. You can read more about klotho in parts 1 and 2 of my series.

In Celtic mythology, Tr na ng is an island paradise and supernatural realm filled with everlasting youth, beauty, health, abundance, and joy. The inhabitants of Tr na ng consist of warriors and gods who are known for their poetry, music, and entertainment. By participating in the feast of Goibniu, the inhabitants of the island are granted immortality, giving Tr na ng its nickname The Land of Youth.

Now, studies report that a protein named after this Celtic taleNANOG may play a role in anti-aging and cancer prevention, but what is NANOG and why do researchers believe that it may prevent aging?

What is NANOG?

The key to NANOG and its biological function lies in our embryonic stem cells. Stem cells are a unique cell type, containing two characteristics that make them very powerful: pluripotency and the ability for self-renewal.

Pluripotency is the ability of stem cells to develop into many different tissues or cell types. While all embryonic stem cells are essentially the same basic cell, each stem cell has the potential to grow into nearly any tissue or cell type in the body. In other words, a single stem cell has multiple fates and depending on the environmental and chemical cues the stem cell is given, it can become anything from a brain cell to a muscle cell.

This becomes an especially important property when considering that stem cells have the ability to self-renew. Unlike many other cells, embryonic stem cells can simply divide to create multiples of themselves, allowing them to exist perpetually in the body so long as they can continue to self-renew.

What allows these stem cells to not only constantly regenerate, but to maintain their pluripotency? In other words, when new stem cells are created, what prevents them from spontaneously maturing into some form of adult cell?

Both pluripotency and self-renewal have been linked to a few, core transcription factors. Transcription factors are proteins in the body that control whether genes are turned on or off. Previous studies have found that the core transcription factors controlling stemness are Oct4, Sox2, Klf4, and NANOG.

How NANOG maintains pluripotency and self-renewal

An interesting aspect of NANOG is that it does not exist in adult cells. Instead, NANOG is only detected in stem cells that are still pluripotentthey have not begun to differentiate or develop into any particular cell type. Once a stem cell begins to differentiate, the NANOG gene is turned off and NANOG protein is no longer produced in the cell. Due to this observation, scientists suggest that NANOG may play a role in maintaining the pluripotency of stem cells, essentially preventing the stem cells from differentiating and maturing into adult cells.

There are three NANOG proteins that influence the pluripotency of human embryonic stem cells. These are Nanog1, Nanog2, and NanogP8. Researchers have found that when Nanog1 is abundant, it can prevent embryonic stem cells from differentiating. When the protein is scarce, embryonic stem cells tend to mature and differentiate into other cell types, losing their pluripotency.

NanogP8 is similar to Nanog1 except that it is mainly found in cancer cells, suggesting that NanogP8 may have larger anti-cancer properties than anti-aging properties.

How exactly could NANOG prevent stem cells from differentiating? While it is currently unclear what the biological mechanisms of NANOG are, two proteins that might offer a clue are leukemia inhibitory factor and STAT3. Both leukemia inhibitory factor proteins and STAT3 are widely known to prevent the differentiation of stem cells.

Leukemia inhibitory factor plays an important role in a wide array of biological processes including the growth of leukemia cancer cells and inflammation. The protein works primarily by controlling the activity of other transcription factors and proteins including STAT3. Researchers have found that when leukemia inhibitory factor levels drop and STAT3 activity decreases, stem cells begin to differentiate.

Interestingly, more recent experiments have shown that when NANOG is deficient, stem cells also differentiate. This occurs even in the presence of STAT3 and leukemia inhibitory factor, suggesting that NANOG may help maintain the pluripotency of stem cells by controlling the production and release of leukemia inhibitory factor and STAT3.

Additional studies focus on how NANOG may interact with other core transcription factors like Oct4 to prevent the differentiation of stem cells and maintain pluripotency. Researchers have suggested that NANOG and Oct4 may work together to control certain genes and proteins related to the pluripotency of stem cells. Oct4 is a transcription factor that is critically involved in the self-renewal of embryonic stem cells. Studies have shown that NANOG and Oct4 exhibit very similar patterns of behavior and often regulate similar genes in the body. This has led to the suspicion that NANOG and Oct4 may play a combined role in maintaining the pluripotency and self-renewal of embryonic stem cells.

Conclusion: NANOG in aging and cancer

While the biological mechanisms of how NANOG maintains the pluripotency of stem cells are unclear, it is evident that this small protein has an unusual ability to determine whether or not cells mature into different cell types. In the next installments of this short series, I will detail some of the more recent studies on NANOG, how the protein may prevent cellular aging, and how it could play a role in minimizing the growth of cancerous tumors.

I am a scientist, businessman, author, and philanthropist. For nearly two decades, I was a professor at Harvard Medical School and Harvard School of Public Health where I founded two academic research departments, the Division of Biochemical Pharmacology and the Division of Human Retrovirology. I am perhaps most well known for my work on cancer, HIV/AIDS, genomics and, today, on COVID-19. My autobiography, My Lifelong Fight Against Disease, publishes this October. I am chair and president of ACCESS Health International, a nonprofit organization I founded that fosters innovative solutions to the greatest health challenges of our day. Each of my articles at Forbes.com will focus on a specific healthcare challenge and offer best practices and innovative solutions to overcome those challenges for the benefit of all.

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NANOG (Part 1): Its Role In Aging And Cancer - Forbes

Stem Cell Therapy Market Size is Estimated to Increase USD 44.5 … – GlobeNewswire

By Embryonic Stem Cells

New York, April 11, 2023 (GLOBE NEWSWIRE) -- Market.us research reveals that The global stem cell therapy market size was estimated at USD 11.1 billion in 2022 and is expected to reach around USD 44.5 billion by 2032, poised to grow at a compound annual growth rate (CAGR) of 15.3% during the forecast period 2022 to 2030. Stem cell therapy is used to transplant stem cells; in this therapy, stem cells are changed by chemotherapy. Stem cell therapy contains types like Hematopoietic Stem Cells (HSCs), Mesenchymal Stem Cells (MSCs), Skin Stem Cells (SSCs), and Neural Stem Cells (NSCs). Chemotherapy act as an immune system to face disorders linked to blood or cancer, such as multiple myeloma, neuroblastoma, lymphoma, and leukemia. The cost of each stem cell therapy method can vary between 5,000$ and 50,000$.

Identify key trends, drivers, and challenges in the stem cell therapy market, which will help vendors improve their strategies to stay ahead of their competitors. View our PDF Sample Report - https://market.us/report/stem-cell-therapy-market/request-sample/

Key Takeaway:

The stem cell therapy market increased owing to a lot of investment in stem cell research, various clinical trials related to stem cell therapy, and an increasing number of cell therapy production facilities. Several cell therapy clinical trials were done during the epidemic due to the various benefits of stem cell therapies.

Factors affecting the growth of the Stem Cell Therapy industry?

There are several factors that can affect the growth of the neonatal intensive care respiratory devices industry. Some of these factors include:

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Top Trends in Global Stem Cell Therapy Market

The global cancer load is increasing. Hence, cancer therapies must be updated with regional and national priorities. Breast, lung, cervical, colorectal, and thyroid cancers are common among women. However, lung cancer and prostate cancer are the more frequent among men which, acquired a share of 33% of male cancer.

A stem cell transplant is a treatment for cancer types such as leukemia, neuroblastoma, multiple myeloma, or lymphoma types. Autologous transplants are favored in the cases such as leukemia, testicular cancer, multiple myeloma, lymphomas, and neuroblastoma. Autologous and allogeneic stem cell transplants can both be used for cancer treatments.

Regional Analysis

North America dominated the market share of the global stem cell therapy market, with 35% of this market in 2022. The most recent advanced technologies, considerable governmental & private investment, and economic expansion help to benefits the market region. The presence of the latest research infrastructure, rising R&D efforts to form chronic diseases therapeutic choices, and ordinary clinical trials review process for driving the market elements in the region. Key research organizations and market suppliers located in the United States receive large funding. Various projects in the region's pipeline are increasing consumer demand.

Due to an increase in the need for stem cell therapy and a rising prevalence of chronic and neurological diseases, Europe acquired the second-highest market share. The cell therapy in Asia-Pacific market growth is increasing at the highest growth rate because of its fast growth and several companies arrival in APAC. Moreover, the APAC region's increasing use of stem cell therapy and rising cancer dominance are projected to stimulate market expansion.

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Scope of the Report

Market Drivers

The efficient treatment demand is increasing due to the rising chronic disease rate. In the COVID-19 epidemic, chronic diseases such as heart disease, cardiovascular disorder, cancer, neurological disorder, stroke, and respiratory illnesses place a big stress on society. Four of the ten adults have greater than two chronic diseases, and six of ten adults have at least one chronic disease. These show how diseases are dominating as well as how nicely they are managed. Clinical trials are expected to raise stem cell demand during the forecast period.

Market Restraints

Although research on stem cells is very promising for the medical sector, its expansion is restrained due to ethical issues by a regulatory bodies. The devastation of the human embryonic stem cell is the main issue that might have foiled the development of stem cell research.

Market Opportunities

The banking of stem cells need is rising as the number of chronic disorders and genetic abnormalities has increased. An increasing firm has stepped into the stem cell banking sector due to rising public awareness of stem cell therapy. To boost sales and market for stem cell therapy, adult stem cells, which are genetically programmed, are using embryonic stem cells called pluripotent stem cells.

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Report Segmentation of the Global Stem Cell Therapy Market

Cell Source Insight

The largest market share was dominated by adipose tissue with 34% market share in 2022. Because source of this cell is used in adipose tissue, which is a specialized connective tissue containing lipid-rich cells known as adipocytes. These adipocytes are mainly discovered in the skin and deposit among the muscles, intestines membrane folds, inside the intestines, near the heart, and other places. Moreover, the global stem cell therapy market share depends on cell source types segmented into IPSCs, neural, PPSCs, embryo/cord, adipose tissue, and bone marrow.

Type Insight

The global stem cell therapy market is growing because the largest market share was occupied by the allogenic segment, with a 74% market share in 2022, owing to its increasing incidence of cancer and other related disorders. The global stem cell therapy market share is based on the type segregated into allogenic stem cell therapy and autologous stem cell therapy. According to the WHO, children of ages 0 to 19 are diagnosed with almost 300K new cases of cancer annually. In 2022, there will be 21 million new cases of cancer, according to estimates. This puts a strain on the global healthcare system and increases the demand for cancer treatments that are efficient anywhere in the world.

Application Insight

The largest market share was dominated by the diabetes segment, with a 28% market share in 2022. Because 450 million people are infected by diabetes across the globe, from WHO report. Diabetes is normal in several countries for individuals of low and medium incomes. The global stem cell therapy market is based on applications segmented into cardiovascular, diabetes, cancer, musculoskeletal disorders, and gastrointestinal diseases. Furthermore, the diabetes segment, also called diabetes mellitus of metabolic disease that is infested when blood glucose levels are too high. Over time period, diabetes disorder can cause a lot of harm to the kidneys, eyes, blood vessels, heart, and eyes.

End User Insight

The largest market share was dominated by the hospitals & clinics segment, with a 28% market share in 2022 because of the rising occurrence of skin conditions like acne. The global stem cell therapy market is based on end-user types segmented into hospitals & clinics, academic & research centers, ambulatory surgical centers, and other end users.

Drivers and challenges have an impact on market dynamics and can impact businesses. Find some insights from a sample report: https://market.us/report/stem-cell-therapy-market/request-sample/

Market Segmentation

By Cell Source

By Type

By Application

By End User

By Geography

Competitive Landscape

With the presence of many local and regional players, the market for global stem cell therapy market is fragmented. Market players have intense competition from prominent key players, mainly those with big recognition of the brand and high networks for distribution. Companies have started various expansion strategies to gain a top spot in the market, such as partnerships and product launches.

The competitive landscape of the market has also been examined in this report. Some of the major players include

Recent Development of the Stem Cell Therapy Market

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Stem Cell Therapy Market Size is Estimated to Increase USD 44.5 ... - GlobeNewswire

Regenerative Medicine Market Investments, Share and Revenue … – GlobeNewswire

By Embryonic Stem Cells

Jersey City, NJ, April 12, 2023 (GLOBE NEWSWIRE) -- InsightAce Analytic Pvt. Ltd. announces the release of a market assessment report on the "GlobalRegenerative Medicine Market Size, Share & Trends Analysis Report By Product (Therapeutics, Primary cell-based therapeutics, Stem Cell & Progenitor Cell-based therapeutics), By Therapeutic Category (Dermatology, Musculoskeletal, Immunology & Inflammation, and Oncology)- Market Outlook And Industry Analysis 2031"

The global Regenerative Medicine market is estimated to reach over USD 183.08 billion by 2031, exhibiting a CAGR of 15.02% during the forecast period.

In recent year, it has been determined that regenerative therapies can uniquely change the underlying pathological processes. Trial-stage regenerative medicines offer promising treatments for particular chronic diseases with unmet medical needs. Novartis announced the release of T-ChargeTM in December 2021, a next-generation CAR-T platform that would be used for cutting-edge investigational CAR-T cell treatments.

Free PDF Report Brochure @https://www.insightaceanalytic.com/request-sample/1687

The development of gene-based treatment, which uses targeted DNA delivery as a drug to combat numerous illnesses, results from significant effects in molecular therapeutics. With the restoration of gene function, gene therapy holds great promise for treating cancer and type 1 and type 2 diabetes. Gene-based medicines treat patients with conditions such as cancer, oncology, infectious diseases, cardiovascular disorders, monogenic diseases, genetic disorders, ophthalmological indications, and central nervous system illnesses. These elements have helped the market for regenerative medicine expand.

Recent Developments:

In April 2022, Obecabatagene autoleucel, a CD19-directed autologous chimeric antigen receptor T therapy being investigated in the ongoing FELIX Phase 2 study of leukaemia, has been given the Regenerative Medicine Advanced Therapy designation by the U.S. Food and Drug Administration (FDA). This was announced by Autolus Therapeutics plc.

List of Prominent Players in the Regenerative Medicine Market:

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Regenerative Medicine Market Report Scope:

Market Dynamics:

Drivers- The ability of adult stem cells to proliferate or self-renew forever and to develop all the cell kinds of the organ from which they originate has propelled research into these cells, with the potential to regenerate the complete organ from a few cells. No embryo must be destroyed in order to produce adult stem cells. Furthermore, medical research on stem cells has been thoroughly examined and attracted much attention. ExCellThera Inc. and Ossium Health recently announced a partnership to explore and advance opportunities to use adult stem cells from deceased donors from Ossium Health's first-ever bone marrow bank in combination with ExCellThera's ECT-001 cell expansion and rejuvenation technology. This collaboration will take place in April 2021. These kinds of developments are anticipated to accelerate market expansion.

Challenges:The market for regenerative medicine is projected to be hampered by a lack of information and moral considerations surrounding the usage of embryonic stem cells for research and development. Since cell therapy is a crucial component of regenerative medicine, it has a significant impact on the market growth rate. One of the leading market inhibitors may be the high cost of investment, which might be followed by problems with assay sensitivity, robustness, and reproducibility; the challenge of culture/propagation; and finally, the challenge of handling.

Regional Trends:Due to the presence of big players, the rapid advancement of technology, significant investments in stem cell and oncology research, and the presence of major players, North America is predicted to have the largest revenue share. The largest market in North America is the United States. In the U.S., numerous stem cell therapies are increasingly being used to treat a growing number of ailments like cancer and diabetes. According to the Heart Disease & Stroke Statistics Fact Sheet 2020, congenital heart abnormalities are predicted to affect at least 40,000 infants annually in the United States.

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Segmentation of Regenerative Medicine Market-

By Product-

By Therapeutic Category-

By Region-

North America-

Europe-

Asia-Pacific-

Latin America-

Middle East & Africa-

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Regenerative Medicine Market Investments, Share and Revenue ... - GlobeNewswire

Bourbonnais toddler beats leukemia with stem cell transplant – Kankakee Daily Journal

By Stem Cell Doctors

While watching 3-year-old Mads Pomranky excitedly play on a Tuesday afternoon with his 1-year-old sister, Valen, one would never guess the difficult health struggles hes overcome.

At 5 months old, Mads was diagnosed with juvenile myelomonocytic leukemia, a rare cancer of the blood that affects young children. The diagnosis eventually turned to acute myeloid leukemia and was treatable only through a transplant from a stem cell donor.

For parents Jennifer and Derrick Pomranky, of Bourbonnais, they made the immediate decision to approach the challenge with positivity, designating their son Mighty Mads.

Its pretty difficult to watch your little baby go through this, Jennifer said. I feel like right away, we both had the mentality of, Hes gonna get through this, were gonna get through this.

We got through it one day at a time, added Derrick.

As an infant, Mads was often sick with congestion or runny nose. It was a pediatrician, Dr. Josefina Batista at Riverside Healthcare, that noticed his spleen was enlarged. Further testing would show his red blood cell count being low while his white cell count was high. These red flags led to the eventual leukemia diagnosis.

His pediatrician caught it so fast that we had time on our side that they could pinpoint the diagnosis and take their time with the chemo and wean him into it a little bit, said Jennifer while flipping through a book the family made for Mads donor. The book details Mads journey with cancer and treatment.

The book documents that Mads had six rounds of chemo, 83 doses of chemo, 31 blood transfusions, 31 platelet transfusions, 11 bone marrow biopsies, five spinal taps with chemo, two skin biopsies and one cryo transfusion.

His parents noted they were lucky in the search for a donor, as Mads health care team found a 10 out of 10 match through the stem cell/bone marrow transplant registry, DKMS.

The donation was from a young woman, Kaelynn Speed, out of Cincinnati.

Weve been an advocate for [DKMS], Jennifer said, noting that the DKMS shirts her kids were wearing were given to the family when they first met Speed through a donor meeting facilitated by the donor registry.

By the time doctors were able to begin providing transplant care to Mads then 13 months old the world was in the throes of COVID-19. As a result, Speeds cell donation had to be frozen for two weeks before being administered to Mads.

Mom Jennifer said that the process took about an hour and was similar to a blood transfusion.

Now, Mads has to have bloodwork done every three months to make sure everything is on the up and up. Additionally, he does a survivorship follow up at Comer Childrens Hospital, where he had his treatments.

Hes doing great. Hes very rambunctious and a normal toddler, said Jennifer.

Born May 16, 2019, Mads now has double the annual celebrations first, his birthday, and second, what his parents have dubbed his rebirthday on June 17, which is when he had the transplant.

Before and after transplant, he was really sick, Derrick said. So that was a bad time.

When he was going through all of it, he missed a lot of his milestones. He was basically reborn again, added Jennifer.

LIFE AND DEATH

The family still keeps in touch with Speed and her family; they received her information on the one-year anniversary of Mads transplant and set up a Zoom meeting. The families met in person last summer.

Shes the nicest person, she was only 19 when she signed up [to donate], Jennifer said. Derrick added that shes studying nursing.

Between work and two toddlers, the Pomranky parents certainly have their hands full, but they always make time to encourage people to sign up to be stem cell and/or bone marrow donors.

You could potentially save a life, Jennifer said. Its a matter of life and death, literally.

Excerpt from:
Bourbonnais toddler beats leukemia with stem cell transplant - Kankakee Daily Journal

Invasive aspergillosis: Symptoms and more – Medical News Today

By Stem Cell Doctors

Aspergillosis is an infectious disease that occurs due to a common type of fungus, or mold, called Aspergillus. A type of aspergillosis called invasive aspergillosis particularly affects people with weakened immune systems.

Most individuals breathe Aspergillus spores daily and do not develop any health effects.

However, people with compromised immune systems, such as those who have had an organ transplant or are undergoing chemotherapy treatment, may be at greater risk of developing aspergillosis.

The disease usually affects the lungs but can spread to other areas of the body.

This article looks at invasive aspergillosis and its symptoms, diagnosis, outlook, and more.

Aspergillus mold is common indoors and outdoors and can cause infection in the sinuses and lungs of immunocompromised people.

If the infection spreads to the rest of the body, outcomes are very unfavorable and include death.

Experts do not know the extent of aspergillosis in the United States, as it is not a reportable infection. However, according to a database of insurance claims, there were almost 15,000 hospitalizations for this diagnosis in the U.S. in 2014.

That said, the number of people with aspergillosis has likely increased since then.

Invasive aspergillosis is less common than the milder, allergic forms of the disease. It is the most common fungal infection in people who have had stem cell treatment. It is also the second most common in those who have received solid organ transplants.

Invasive aspergillosis usually occurs in people with weakened immune systems who often already have other medical conditions. Therefore, it can be difficult to differentiate between the symptoms of the infection and those of the underlying illness.

The symptoms of invasive aspergillosis may include:

Types of invasive aspergillosis include:

Other types of aspergillosis include:

A person can develop aspergillosis by breathing in Aspergillus spores, which are common in indoor and outdoor environments.

Most people will not get sick from breathing the spores, but those with weakened immune systems are at greater risk of infection.

People at risk of invasive aspergillosis include individuals with weakened immune systems, such as those who have received:

Additionally, hospitalized patients with severe influenza and those with HIV are at risk of invasive aspergillosis.

To diagnose aspergillosis, a doctor may examine a persons medical history and risk factors and ask questions about their symptoms.

They may perform a physical examination and several laboratory tests. These may include:

Doctors may treat invasive aspergillosis with several different antifungal medications.

These may include:

Where possible, doctors may also decrease or discontinue immunosuppressive medications to help treat invasive aspergillosis.

Because Aspergillus spores are common in the environment, it is difficult for people to avoid breathing them in.

Experts recommend those with weakened immune systems take certain measures to protect against the fungus. However, these actions may not prevent aspergillosis.

Preventive measures may include:

The outlook for invasive aspergillosis is generally unfavorable.

The mortality rate of invasive pulmonary aspergillosis is about 20% six weeks after diagnosis, despite treatment with antifungal medications.

The mortality rate reaches close to 100% once the infection has spread to the central nervous system.

Most people safely breathe Aspergillus spores every day, as these are common in indoor and outdoor environments.

However, those with weakened immune systems may be at risk of developing health problems from breathing microscopic amounts of the fungus.

In immunocompromised people, such as those undergoing chemotherapy or those who have had stem cell or organ transplants, invasive aspergillosis may affect the lungs and sinuses. This can cause symptoms such as shortness of breath and coughing up blood.

The disease may spread to other areas of the body and is often fatal once it reaches the central nervous system.

Treatment for invasive aspergillosis typically includes the use of antifungal medication.

Doctors may advise high risk individuals who are immunocompromised to take measures that may help prevent aspergillosis. These approaches include wearing protective equipment and clothing, testing, and taking antifungal drugs.

See the article here:
Invasive aspergillosis: Symptoms and more - Medical News Today

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