Three UCLA scientists receive grants totaling more than $18 million – UCLA Newsroom
By daniellenierenberg
Three researchers at theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLAhave received awards totaling more than $18 million from the California Institute for Regenerative Medicine, the states stem cell agency.
The recipients are Dr. Sophie Deng, professor of ophthalmology at the UCLA Stein Eye Institute;Yvonne Chen, a UCLA associate professor of microbiology, immunology and molecular genetics; and Dr. Caroline Kuo, a UCLA assistant clinical professor of pediatrics. The awards were announced at a CIRM meeting today.
Dengs four-year, $10.3 million award will fund a clinical trial for a blinding eye condition called limbal stem cell deficiency. Limbal stem cells are specialized stem cells in eye tissue that help maintain the health of the cornea. Because of genetic defects or injuries caused by infections, burns, surgeries or other factors, some people do not have enough limbal stem cells, which results in pain, corneal scarring and blindness.
The approach she is testing involves extracting a small number of limbal stem cells from a persons eye, multiplying them in a lab, and then transplanting them back into the eye, where they could regenerate the cornea and restore vision. The research will be conducted in collaboration with theUCLAUCI Alpha Stem Cell Clinic, a partnership between UCLA and UC Irvine.
The grants awarded to Chen and Kuo are for projects that are heading toward the FDAs investigational new drug application process, which is required by the agency before a phase 1 clinical trial the stage of testing that focuses on a treatments safety.
Chens two-year, $3.2 million award will fund efforts to create a more effectiveCAR T cell therapyfor multiple myeloma, a blood cancer that affects white blood cells. The research will evaluate a specialized form of CAR T therapy that simultaneously targets two markers, BCMA and CS1, commonly found on multiple myeloma cells. CAR T therapies that target BCMA alone have been effective in clinical trials, but the presence of BCMA on multiple myeloma cells is not uniform.
Previous research has shown that the marker CS1 is present in around 90% of multiple myeloma cells. A CAR T therapy that targets both markers could potentially help more patients and reduce the likelihood of a cancer relapse.
Kuos 2 1/2-year, $4.9 million award, will support the development of a stem cell gene therapy for a deadly immunodeficiency called X-linked hyper IgM syndrome, or XHIM.
The syndrome, which is caused by a mutation in the CD40LG gene, results in invasive infections of the liver, gastrointestinal tract and lungs. Currently, the only potential cure is a bone marrow transplant from a matched donor, which carries life-threatening risks and is often less effective for XHIM patients than patients with other forms of immune deficiency. Even with current treatments, only 30% of people with the syndrome live to age 30.
Kuo will evaluate a stem cell gene therapy that corrects the genetic mutation that causes XHIM. After removing blood-forming stem cells from a person with the syndrome, the therapy would use a genetic engineering technique called CRISPR to insert a correct copy of the affected gene into the DNA of the stem cells. The corrected blood-forming stem cells would be infused back into the patient, where they could regenerate a healthy immune system.
She will collaborate with Dr. Donald Kohn, a UCLA distinguished professor of microbiology, immunology and molecular genetics who has successfully treated two other immune deficiencies bubble baby disease and X-linked chronic granulomatous disease with a similar therapy.
Read more:
Three UCLA scientists receive grants totaling more than $18 million - UCLA Newsroom
BrainStorm Cell Therapeutics to Announce Third Quarter Financial Results and Provide a Comprehensive Corporate Update – Yahoo Finance
By daniellenierenberg
NEW YORK, Nov. 05, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics, Inc. (NASDAQ:BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, today announced that the Company will hold a conference call to update shareholders on financial results for the third quarter ended September 30, 2019, and provide a corporate update, at 8:00 a.m., Eastern Standard Time, on Thursday, November 14, 2019.
BrainStorms President & CEO, Chaim Lebovits, will present a corporate update, after which, participant questions will be answered. Joining Mr. Lebovits to answer investment community questions will be Ralph Kern, MD, MHSc, Chief Operating Officer and Chief Medical Officer, and Preetam Shah, PhD, Chief Financial Officer.
Participants are encouraged to submit their questions prior to the call by sending them to: q@brainstorm-cell.com; Questions should be submitted by 5:00 p.m., Eastern Standard Time, Tuesday, November 12.
The investment community may participate in the conference call by dialing the following numbers:
Those interested in listening to the conference call live via the internet may do so by visiting the Investors & Media page of BrainStorms website at http://www.ir.brainstorm-cell.com and clicking on the conference call link.
A webcast replay of the conference call will be available for 30 days on the Investors & Media page of BrainStorms website:
About NurOwnNurOwn (autologous MSC-NTF) cells represent a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. BrainStorm has fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also recently received U.S. FDA acceptance to initiate a Phase 2 open-label multicenter trial in progressive MS and enrollment began in March 2019.
About BrainStorm Cell Therapeutics Inc.BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six U.S. sites supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm also recently received U.S. FDA clearance to initiate a Phase 2 open-label multicenter trial in progressive Multiple Sclerosis. The Phase 2 study of autologous MSC-NTF cells in patients with progressive MS (NCT03799718) started enrollment in March 2019. For more information, visit the company's website at http://www.brainstorm-cell.com
Safe-Harbor Statement
Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.
Story continues
Read the rest here:
BrainStorm Cell Therapeutics to Announce Third Quarter Financial Results and Provide a Comprehensive Corporate Update - Yahoo Finance
19-28z CAR-T Therapy in Children and Young Adults With Relapsed/Refractory ALL: Promising Early Results – Cancer Therapy Advisor
By daniellenierenberg
According to results of a study published in Blood, children and young adults with relapsed/refractory B-cell acute lymphoblastic leukemia (ALL) who had minimal residual disease (MRD) prior to treatment and received high-dose preconditioning chemotherapy were most likely to respond to a second-generation CD-19 chimeric antigen receptor-T cell (CAR-T) therapy.1
Although it has been estimated that 90% or more of pediatric patients witha diagnosis of ALL will respond to multi-agent chemotherapy, the prognosis forthose with relapsed/refractory disease remains poor. One CD19-directed CAR-Ttherapy, tisagenlecleucel, is approved by the US Food and Drug Administration inpatients up to age 25 years with B-cell precursor ALL who either have refractorydisease or have experienced a second or later relapse.2
This open label, nonrandomized, phase 1 study (Clinical Trial Identifier: NCT01860937), evaluated the toxicity, feasibility, and response of 19-28z CAR-T therapy, a second-generation CD19-directed CAR-T therapy involving T cells expressing a chimeric receptor composed of an anti-CD19 antibody binding site and intracellular domains from the T-cell coactivating receptors, CD28 and the CD3-zeta chain3 in children and young adults up to 25 years of age with very high-risk ALL.1 Inclusion criteria included at least 2 relapses, early bone marrow relapse following complete response (CR), intermediate/late CR with poor response to re-induction therapy, or those with refractory disease, or ineligibility for allogeneic hematopoietic stem cell transplantation (allo-HSCT) or additional chemotherapy.1
The age range of the 25 patients treated with 19-28z CAR-T therapy onstudy was 1 to 22.5 years, with a median age of 13.5 years. Preconditioningchemotherapy involved high-dose cyclophosphamide (15 patients) and low-dosecyclophosphamide (8 patients), with 3 patients in each subgroup also receivingfludarabine.1
Regarding the feasibility of this approach, the prespecified CAR-Tcell dose was achieved for all patients for whom the 19-28z CAR-T therapyprocedure was undertaken.1
With respect to treatment toxicity,approximately one-third of patients experienced a grade 3/4 adverse event,including cytokine release syndrome (CRS) and neurotoxicity in 16% and 28% ofpatients, respectively. With the exception of 1 patient with grade 4 CRS andneurotoxicity who died following refractory Stenotrophomonas septic shock,these adverse events were reversible.1
Of the 24 patients includedin the response analysis, 75% achieved either a CR or a CR with incompletecount recovery (CRi). In the subsets of patients receiving preconditioningchemotherapy with either high- or low-dose cyclophosphamide, the CR/CRi rateswere 94% and 38%, respectively. Furthermore, treatment response was influencedby disease burden as evidenced by the considerably higher CR/CRi rate inpatients with baseline minimal residual disease (ie, less than 5% bone marrowblasts; 93%) compared with morphological evidence of disease at baseline (5% orhigher bone marrow blasts; 50%).1
The CR/CRi rate for thesubset of patients with pretreatment MRD treated with high-dose cytarabine was100%.1
Consolidation allo-HSCT was performed in 83% (15) of the patientsresponding to CAR-T therapy, with a median time from CAR-T infusion toallo-HSCT of 57 days. At a median follow-up of 28.6 months for respondingpatients, over half of these patients (8) were alive and had no evidence ofdisease.1
In their concluding remarks, the study authorscommented that thisanalysis has allowed us to determine the toxicity profile, confirm feasibility,evaluate response of this approach, and provide a direct comparison of the sameCD19-specific CAR T cell product that was previously published[3] inadult patients for the same indication.
The authors went on to highlight the findingof a reversible toxicity profile in the patients within their study as well asthe impact of preconditioning chemotherapy dose intensity and minimal pretreatmentdisease burden on response.
They further noted that within this cohort,the long-term persistence of response is encouraging, and in our primarilytransplant-naive patient population, the ability to proceed to allo-HSCT hasdemonstrated a favorable overall survival, manageable toxicity, and limitedincidence of relapse.
References
Read the original:
19-28z CAR-T Therapy in Children and Young Adults With Relapsed/Refractory ALL: Promising Early Results - Cancer Therapy Advisor
Dystrogen Therapeutics Announces That Treatment With Dystrophin Expressing Chimeric (DEC) Cells Improves Cardiac Function in Preclinical Duchenne’s…
By daniellenierenberg
CHICAGO, Nov.5, 2019 /PRNewswire/ -- Scientists from Dystrogen Therapeutics Corp. published data supporting cardioprotective effects of the Company's therapy for muscular dystrophy disorders. Cardiomyopathy is the most devastating cause of morbidity and mortality in Duchenne Muscular Dystrophy (DMD) patients and affects 30% of patients by 14years of age and 50% of patients by 18years of age. Heart failure in these patients is the result of cardiac myocyte death and fibrosis, leading to both diastolic and systolic dysfunction. Dystrogen Therapeutics Corp has developed an engineered chimeric cell therapy which has been previously shown to restore muscle function in pre-clinical studies. For Duchenne's muscular dystrophy, the company has developed dystrophin expressing chimeras "DECs." Using the company's proprietary technology, DECs are created by an ex vivo fusion of allogeneic human myoblast from a healthy donor with autologous human myoblast received from DMD patient. DECs have been shown to maintain the ability to express normal dystrophin protein in previously published pre-clinical studies. The new study published in the October 15th, 2019 online edition of the journal Stem Cell Reports and Reviewsconfirmed the protective effect of DEC on cardiac function after intraosseous delivery shown by increased values of both ejection fraction and fractional shortening, which at 90days revealed a rebound effect when compared to the vehicle injected controls and mice receiving not-chimeric cell therapy. Moreover, these functional improvements correlated with restoration of dystrophin expression in cardiac muscle at 90days post-DEC treatment.
"These findings are potentially significant for the treatment of DMD," said Dr. Maria Siemionow, MD, PhD Dystrogen Therapeutics Corp chief scientific officer and the therapy's inventor. "This study establishes DEC as a promising new option for cardiac protection and potential amelioration of DMD related cardiac pathology."
"These data add to the growing body of literature supporting the potential of our chimeric cell platform to restore systemic muscle function, with less potential side effects then gene therapy-based approaches," said Dr. Kris Siemionow, MD, PhD Dystrogen Therapeutics Corp CEO. "We are very pleased to have these data published in a highly relevant journal for the field and look forward to further exploring this opportunity."
About Dystrogen Therapeutics
Dystrogen Therapeutics is a clinical-stage life sciences company committed to developing personalized therapies for rare diseases. The company has developed a chimeric cell therapy platform. Dystrophin expressing chimeras "DEC" are based on ex vivo fusion of allogeneic human myoblast derived from donors with autologous human myoblast received from the DMD patient, where chimeric cells maintain the ability to express normal dystrophin protein. DEC cells increase the number/pool of normal myoblasts and reduce inflammation. DEC cells induce replacement of fibrotic tissue, thus significantly improving muscle strength and function in DMD pre-clinical studies. The therapy minimizes immune response effects and the need for immunosuppression. This new approach will be based on delivery and restoration of dystrophin in affected muscles preventing the premature loss of mobility and early mortality of DMD patients. The company is planning on enrolling patients for its DEC chimeric cell therapy Duchenne muscular dystrophy trial. This therapy offers a unique advantage and allows the patient's body and immune system to accept the chimeric cells without rejection. Pre-clinical results have demonstrated that increased dystrophin levels correlate with improved functional outcomes. First clinical results from DEC therapy are expected in late 2020.
Contact: info@dystrogen.com
SOURCE Dystrogen Therapeutics Corp
Future Outlook: Autologous Stem Cell Based Therapies Market Prediction and Analysis Offered By New Study 2019 2025: Leading Key Players: JCR…
By daniellenierenberg
A Qualitative Research Study done by Crystal Market Research on Global Autologous Stem Cell Based Therapies Market report provides current and future trends are outlined to determine the overall attractiveness and to single out profitable Autologous Stem Cell Based Therapies trends to gain a stronger position in Industry anticipated to reflect a positive growth trend in Upcoming years as well. Global Autologous Stem Cell Based Therapies market provide in depth coverage from various aspects and scenario to future trends and opportunities. This Autologous Stem Cell Based Therapies report provide latest customized and syndicated research along with consulting services.
Get FREE Research Sample Copy @https://www.crystalmarketresearch.com/report-sample/HC022437
The detailed study of global Autologous Stem Cell Based Therapies market report portraying the current landscape and important projections for market forecast, growth trend. This report is an whole guide for new aspirant to understand the trending values and Autologous Stem Cell Based Therapies future trend. The report arranged dependent on a top to bottom market examination with contributions from industry expert.
Continue
Get Discount of This Precious Report @ https://www.crystalmarketresearch.com/check-discount/HC022437
Autologous Stem Cell Based Therapies report studies market share and growth opportunity covering regions:
Get Customised Research Report: https://www.crystalmarketresearch.com/customization/HC022437
Contacts Us:
Judy | 304 South Jones Blvd, Suite 1896
Las Vegas NV 89107
United States
E-mail: sales@crystalmarketresearch.com | Ph: +1-888-213-4282
Head-To-Head Analysis: BioRestorative Therapies (OTCMKTS:BRTX) versus Livongo Health (OTCMKTS:LVGO) – Casper Courier
By daniellenierenberg
BioRestorative Therapies (OTCMKTS:BRTX) and Livongo Health (NASDAQ:LVGO) are both medical companies, but which is the better investment? We will contrast the two companies based on the strength of their dividends, risk, analyst recommendations, valuation, institutional ownership, profitability and earnings.
Profitability
This table compares BioRestorative Therapies and Livongo Healths net margins, return on equity and return on assets.
Analyst Recommendations
This is a breakdown of recent ratings for BioRestorative Therapies and Livongo Health, as provided by MarketBeat.
Livongo Health has a consensus price target of $44.20, suggesting a potential upside of 87.93%. Given Livongo Healths higher probable upside, analysts clearly believe Livongo Health is more favorable than BioRestorative Therapies.
Institutional & Insider Ownership
0.1% of Livongo Health shares are owned by institutional investors. 17.9% of BioRestorative Therapies shares are owned by insiders. Strong institutional ownership is an indication that large money managers, endowments and hedge funds believe a stock will outperform the market over the long term.
Valuation and Earnings
This table compares BioRestorative Therapies and Livongo Healths top-line revenue, earnings per share (EPS) and valuation.
BioRestorative Therapies has higher earnings, but lower revenue than Livongo Health.
Summary
Livongo Health beats BioRestorative Therapies on 7 of the 9 factors compared between the two stocks.
BioRestorative Therapies Company Profile
BioRestorative Therapies, Inc. develops therapeutic products and medical therapies using cell and tissue protocols, primarily involving adult stem cells for the treatment of disc/spine disease and metabolic disorders. The company's lead cell therapy candidate is the BRTX-100, which focuses on providing non-surgical treatment for protruding and bulging lumbar discs in patients suffering from chronic lumbar disc disease. It also develops the ThermoStem program, a pre-clinical program for the treatment of metabolic diseases, such as type 2 diabetes, obesity, hypertension, and other metabolic disorders, as well as cardiac deficiencies. In addition, the company provides curved needle device, a needle system with a curved inner cannula that allows access to difficult-to-locate regions for the delivery or removal of fluids and other substances. Further, it offers skin care products under the Stem Pearls brand name. BioRestorative Therapies, Inc. has a research and development agreement with Rohto Pharmaceutical Co., Ltd.; and a research agreement with Pfizer, Inc. and the University of Pennsylvania. The company was formerly known as Stem Cell Assurance, Inc. and changed its name to BioRestorative Therapies, Inc. in August 2011. BioRestorative Therapies, Inc. was incorporated in 1997 and is headquartered in Melville, New York.
Livongo Health Company Profile
Livongo Health, Inc. provides an integrated suite of solutions for the healthcare industry in North America. It solutions promote health behavior change based on real-time data capture supported by intuitive devices and insights driven by data science. The company offers a platform that provides cellular-connected devices, supplies, informed coaching, data science-enabled insights, and facilitates access to medications. Its products include Livongo for Diabetes, Livongo for Hypertension, Livongo for Prediabetes and Weight Management, and Livongo for Behavioral Health by myStrength. The company was formerly known as EosHealth, Inc. and changed its name to Livongo Health, Inc. in 2014. Livongo Health, Inc. was incorporated in 2008 and is headquartered in Mountain View, California.
Global Cryopreservation Equipments in Stem Cells Market to Witness Significant Revenue Growth During the Forecast Period, 20192023 – Health News…
By daniellenierenberg
The Global Cryopreservation Equipments in Stem Cells Market Report 2019 offers a profound analysis of the Cryopreservation Equipments in Stem Cells trade. It demonstrates a quick overview of trade knowledge and terminology of the market. The report highlights well-known performers from the global And Cryopreservation Equipments in Stem Cells along with their contribution to the marketplace to see their progress at intervals the calculable time. The global Cryopreservation Equipments in Stem Cells investigation report covers recent improvements whereas forecasting the expansion of most players along with their market shares.
Cryopreservation Equipments in Stem Cells report provides featured analysis that will drive your business to the next level. It overviews including growth factors, market demand, current trends, and forecast research by expert studies. This report estimate upcoming challenges and offers a real solution to the Cryopreservation Equipments in Stem Cells.
Click here for sample [emailprotected] https://www.acquiremarketresearch.com/sample-request/198391/
Following are some of the various segmentation of the market available in the Cryopreservation Equipments in Stem Cells Market report:
Leading players in the market: Thermo Fisher Scientific, Charter Medicals, Linde Gas Cryoservices, praxair
Differentiation of the market based on types of product: Liquid Phase, Vapor Phase
Differentiation of the market based on types of its application: Totipotent Stem Cell, Pluripotent Stem Cell
Geographical Classification of the market: North America (U.S., Canada, Mexico), Europe (Germany, U.K., France, Italy, Russia, Spain, etc.), Asia-Pacific (China, India, Japan, Southeast Asia, etc.), South America (Brazil, Argentina, etc.), Middle East & Africa (Saudi Arabia, South Africa, etc.)
Cryopreservation Equipments in Stem Cells Market
Click here to get [emailprotected] https://www.acquiremarketresearch.com/discount-request/198391/
Our report will address client queries: 1. What is the market share of each region and top countries present in these regions?2. Which countries will depict the highest growth potential in the coming years?3. At which rate the Cryopreservation Equipments in Stem Cells market is growing globally and what are the future trends of this industry?4. Which are top product types and applications holding good potential and growth opportunities?5. Which are top Cryopreservation Equipments in Stem Cells industry players and who is their market competitors?6. Which are market drivers and constraints at present and during the forecast period?7. Which are the traders, dealers, and distributors operating in Cryopreservation Equipments in Stem Cells Industry?
The report on the keyword market is based on a detailed evaluation of the market, with the inclusion of comprehensive primary and secondary research. A Detailed assessment of the keyword market in terms of competitive scenario is supported by individual-level analysis of various aspects related to the market.
For more details click [emailprotected] https://www.acquiremarketresearch.com/enquire-before/198391/
About Us: Acquire Market Research is part of the Acquire Market Research Consultants Pvt. Ltd. and offers premium progressive statistical surveying, market research reports, analysis & forecast data for industries and governments around the globe.
Contact Us at:555 Madison Avenue,5th Floor, Manhattan,New York, 10022 USAPhone No.: +1 (800) 663-5579
Phase 3 Trial of ALS Cell Therapy, NurOwn, Gets Thumbs Up in Safety Review by Monitoring Board – ALS News Today
By daniellenierenberg
The Phase 3 clinical trial testing BrainStorm Cell Therapeutics cell therapy candidateNurOwn inamyotrophic lateral sclerosis (ALS) patients is continuing as planned after a second safety assessment by the trials independent Data Safety Monitoring Board (DSMB) found no reasons to stop, the company announced.
The DSMBs recommendation comes after a pre-specified interim analysis of the first 106 ALS patients treated repeatedly with NurOwn in this randomized, placebo-controlled clinical trial.
After reviewing all of the safety data as of September 30th, the DSMB has recommended the study continue without any changes in the protocol. We did not identify any significant safety concerns, Carlayne Jackson, MD, a professor of Neurology and Otolaryngology UT Health San Antonioand the DSMB chairperson, said in a press release.
DSMBs consist of research experts who monitor the progress of a clinical trial and review safety and efficacy data while the study is ongoing. This panel can recommend that a trial be stopped early because of safety concerns or evidence a therapy is not working as intended, or if the trials main goals have already been reached.
NurOwn consists of mesenchymal stem cells (MSCs; stems cells able to generate various cell types) collected from a patients bone marrow. These MSCs are expanded and matured into a specific cell type called MSC-NTF by growing them under conditions that induce them to secrete high levels of neurotrophic factors (NTFs) that support the growth, survival, and maturation of nerve cells.
MSC-NTF cells also deliver immune system regulating cytokines, small proteins important in cell signaling or messaging, to sites of damage, BrainStorm reports. It is thought this will help to slow or stabilize disease progression.
The double-blind Phase 3 trial (NCT03280056),fully enrolledat its six U.S. sites, is investigating use of NurOwn in 200 ALS patients whose symptoms became evident within two years of the studys start. Patients are randomized 1:1 to either NurOwn or placebo, given via intrathecal (spinal canal) injection every two months.
The studysprimary measures of safety and efficacy are being determined using the ALS functional rating scale score (ALSFRS-R; a score of abilities like swallowing, speech, handwriting, walking, etc.) in patients after 28 weeks of treatment compared to placebo.
A secondary goal is assessing how biomarkers, such as cell-secreted neurothrophic factors, inflammatory agents, andcytokines, change in the blood and cerebrospinal fluid (the liquid surrounding the brain and spinal cord) after treatment with NurOwn.
BrainStorm is expecting to have topline trial data by the end of 2020, which will potentially support the submission of a Biologics License Application (BLA) to theU.S. Food and Drug Administration requesting approval.
We are very pleased with the DSMB recommendation that the Phase 3 clinical trial continue without any protocol modification. This represents an important clinical trial advancement for BrainStorm and for the development of NurOwn as an innovative cellular therapy approach for ALS patients, added Ralph Kern MD, BrainStorms chief operating officer and chief medical officer.
This clinical trial is being funded by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989), and other types of investment.
NurOwn was given orphan drug status by both theFDA and the European Medicines Agency (EMA) as apotential ALS treatment.
Iqra holds a MSc in Cellular and Molecular Medicine from the University of Ottawa in Ottawa, Canada. She also holds a BSc in Life Sciences from Queens University in Kingston, Canada. Currently, she is completing a PhD in Laboratory Medicine and Pathobiology from the University of Toronto in Toronto, Canada. Her research has ranged from across various disease areas including Alzheimers disease, myelodysplastic syndrome, bleeding disorders and rare pediatric brain tumors.
Total Posts: 5
Ins Martins holds a BSc in Cell and Molecular Biology from Universidade Nova de Lisboa and is currently finishing her PhD in Biomedical Sciences at Universidade de Lisboa. Her work has been focused on blood vessels and their role in both hematopoiesis and cancer development.
Follow this link:
Phase 3 Trial of ALS Cell Therapy, NurOwn, Gets Thumbs Up in Safety Review by Monitoring Board - ALS News Today
NeuBase Therapeutics Inc. (NBSE)’s Financial Results Comparing With BioTime Inc. (:) – FinanceMercury
By daniellenierenberg
NeuBase Therapeutics Inc. (NASDAQ:NBSE) and BioTime Inc. (:), both competing one another are Biotechnology companies. We will compare their analyst recommendations, profitability, risk, institutional ownership, dividends, earnings and valuation.
Valuation and Earnings
Table 1 shows the top-line revenue, earnings per share (EPS) and valuation for NeuBase Therapeutics Inc. and BioTime Inc.
Profitability
Table 2 shows us NeuBase Therapeutics Inc. and BioTime Inc.s return on equity, net margins and return on assets.
Volatility and Risk
A 0.89 beta indicates that NeuBase Therapeutics Inc. is 11.00% less volatile compared to Standard & Poors 500. Competitively, BioTime Inc.s 181.00% volatility makes it more volatile than Standard & Poors 500, because of the 2.81 beta.
Liquidity
The Current Ratio of NeuBase Therapeutics Inc. is 3.7 while its Quick Ratio stands at 3.7. The Current Ratio of rival BioTime Inc. is 3.5 and its Quick Ratio is has 3.5. NeuBase Therapeutics Inc. is better equipped to clear short and long-term obligations than BioTime Inc.
Analyst Ratings
NeuBase Therapeutics Inc. and BioTime Inc. Ratings and Recommendations are available in the next table.
The consensus target price of NeuBase Therapeutics Inc. is $14.5, with potential upside of 184.31%.
Institutional and Insider Ownership
The shares of both NeuBase Therapeutics Inc. and BioTime Inc. are owned by institutional investors at 3.1% and 43.7% respectively. Competitively, 3.9% are BioTime Inc.s share held by insiders.
Performance
In this table we show the Weekly, Monthly, Quarterly, Half Yearly, Yearly and YTD Performance of both pretenders.
For the past year NeuBase Therapeutics Inc.s stock price has bigger growth than BioTime Inc.
Summary
NeuBase Therapeutics Inc. beats on 6 of the 10 factors BioTime Inc.
BioTime, Inc., a clinical-stage biotechnology company, focuses on developing and commercializing products addressing degenerative diseases based on pluripotent stem cells and HyStem cell/drug delivery platform technologies. Its product candidates include Renevia, a facial aesthetics product that is in pivotal clinical trial for the treatment of HIV related facial lipoatrophy; OpRegen, which is in Phase I/IIa clinical trial for the treatment of the dry form of age-related macular degeneration; HyStem-BDNF, a preclinical development program for the delivery of recombinant human brain-derived neurotrophic factor (BDNF) directly into the stroke cavity of patients for aiding in tissue repair and functional recovery; and ReGlyde that is in preclinical development as a device for viscosupplementation and a combination product for drug delivery in osteoarthritis. The company also develops AST-OPC1, a therapy derived from pluripotent stem cells that is in a Phase I/IIa clinical trial for spinal cord injuries; AST-VAC1, a patient-specific cancer immunotherapy that is in Phase II clinical trial for acute myeloid leukemia; and AST-VAC2, a non-patient specific cancer immunotherapy, which is in Phase I/IIa clinical trial to treat non-small cell lung cancer. In addition, it offers liquid biopsy tests for diagnosis of cancer; bone grafting products to treat orthopedic disorders; and mobile health software products. Further, it markets GeneCards, a human gene database; LifeMap Discovery, a database of embryonic development, stem cell research, and regenerative medicine; MalaCards, a human disease database; VarElect, an application for prioritizing gene variants; and GeneAnalytics, a novel gene set analysis tool. Additionally, the company develops and markets Hextend, a blood plasma volume expander used for the treatment of hypovolemia. BioTime, Inc. was founded in 1990 and is based in Alameda, California.
Receive News & Ratings Via Email - Enter your email address below to receive a concise daily summary of the latest news and analysts' ratings with our FREE daily email newsletter.
Continue reading here:
NeuBase Therapeutics Inc. (NBSE)'s Financial Results Comparing With BioTime Inc. (:) - FinanceMercury
Lymphatic System Discovered To Play Key Role in Hair Regeneration – Technology Networks
By daniellenierenberg
Given the amount of wear and tear its subjected to on a daily basis, the skin has a phenomenal ability to replenish itself. Spread throughout it are small reservoirs of stem cells, nested within supportive microenvironments called niches, which keep a tight rein on this repair process. Too much tissue might cause problems like cancer, while too little might accelerate aging.
Until now, scientists were uncertain whether the stem cells themselves could instruct other stem cells to form new skin by reshaping their niche. But new research in Science, led by Elaine Fuchs, the Rebecca C. Lancefield Professor, indicates that stem cells can indeed influence tissue regeneration. The study identifies a molecular coordination tool used by stem cells to signal across niches.
The researchers also discovered a new component of the niche: a specialized type of vessel called lymphatic capillaries, which transport immune cells and drain excess fluids and toxins from tissues. These capillaries form an intimate network around the stem cell niche within each hair follicle, the study showed, thereby interconnecting all its niches.
By turning the skin completely transparent, says postdoctoral fellow Shiri Gur-Cohen, we were able to reveal the complex architecture of this network of tubes.
Hair-follicle stem cells control the behavior of lymphatic capillaries by secreting molecules that act as an on-off switch for drainage, the scientists found, enabling them to control the composition of fluids and cells in the surrounding locale and ultimately synchronize regeneration across the tissue.
The involvement of the lymphatic system in this process is a new concept, says Fuchs, and might potentially provide new therapeutic targets for lymph-related conditions such as wound-healing defects and hair loss.
Reference
Gur-Cohen et al. (2019) Stem celldriven lymphatic remodeling coordinates tissue regeneration. Science. DOI: https://doi.org/10.1126/science.aay4509
This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.
Visit link:
Lymphatic System Discovered To Play Key Role in Hair Regeneration - Technology Networks
Some of the deadliest skin cancers may start in your hair – Siliconrepublic.com
By daniellenierenberg
Cells responsible for our hair colour could also be the starting point for some of the deadliest skin cancers.
Scientists in the US have discovered an unexpected origin of deadly skin cancers. In a paper published to Nature Communications, a team from NYU showed that some of these cancers may originate in stem cells in the hair follicles that give it colour, rather than in skin layers.
Hair follicles exist within skin layers as complex organs, but the study has shown that immature pigment-making cells may develop cancer-causing genetic changes, exacerbated by exposure to normal hair growth signals.
Previous models of this disease had put forward the idea that ultraviolet radiation through sunlight was a major risk factor in melanoma. However, this latest study argued that triggers may always be there in hair follicles. Unlike normal follicles, newly cancerous pigment stem cells migrate up and out of the follicles to establish melanomas in nearby surface skin before spreading deeper, it said.
The teams focus was on stem cells that mature into melanocytes, cells that make the protein pigment melanin, which typically protect skin by absorbing the suns damaging rays. By absorbing some wavelengths of visible light but reflecting others, pigments create hair colour.
In testing, specially bred mice were used with an ability to edit genes in follicular melanocyte stem cells only, which could be easily tracked. This confirmed melanoma cells can arise from melanocyte stem cells, which abnormally migrate up and out of hair follicles to enter the outermost layer of the skin.
They continued to move deeper into the skin layer where they not only shed their follicular origins, but also acquired signatures similar to neurons and skin cells almost exactly like those seen in human melanoma tissue.
By knowing where to look for the original, cancer-causing event, the researchers temporarily eliminated signals one by one in the follicular environment to see if cancer still formed in their absence.
Our mouse model is the first to demonstrate that follicular oncogenic melanocyte stem cells can establish melanomas, which promises to make it useful in identifying new diagnostics and treatments for melanoma, said the studys first author Qi Sun.
While our findings will require confirmation in further human testing, they argue that melanoma can arise in pigment stem cells originating both in follicles and in skin layers, such that some melanomas have multiple stem cells of origin.
Read more:
Some of the deadliest skin cancers may start in your hair - Siliconrepublic.com
UM Today | UM Today the Magazine | Fall 2019 | After the Fire – UM Today
By daniellenierenberg
Twenty seconds. Give or take.
Spencer Beach cant say for sure just how long he was on fire.
Long enough to stumble to two exitstwiceand yank on door handles that wouldnt budge.
The flash fire that engulfed him in an Edmonton home was sucking in all the oxygen, creating a vacuum that sealed him inside. A flooring installer by trade, he had been removing linoleum in the laundry rooma rush job since the manufacturer mislabelled the boxes and the contractor didnt want to incur penalties for a delay. Beach and the rest of the crew were often told to use their boss homemade shortcut: douse the flooring in a contact thinner to reactivate the glue. That way, you could just peel it off.
He was on his hands and knees when he first heard a whistle as loud as a thousand kettles, followed by a boom as the furnace set the fumes alight.
Now the skin on his face felt like it was shrinking. His lips, like theyd been swarmed by bees.
The heat was inside of me. It wasnt like any burn youve ever felt, Beach says. It was everywhere.
With no way out, he curled up on the floor, interlocking his fingers behind his head. His mind flashed to his wife, Tina, blonde and four months pregnant with their first child. He had just left her a voice message: the job was nearly done, he was the only one left, hed be home soon. Tonight, hed plan his buddys stag party.
Beach got to his feet and tried the door one more time.
In his right hand, Dr. Sarvesh Logsetty holds a pair of scissors. In his left, a piece of paper grabbed from the many stacks in his office at Winnipegs Health Sciences Centre (HSC), where he is head of the burn unit.
The surgeon is stumped.
Hes trying to remember the strategic cuts to make for a trick he would do as a kid. Its a paper craft that turns a 4-by-5-inch sheet into an open shape large enough to walk through.
What am I doing wrong?
Logsetty wants to use the analogy to explain the skin graft meshing techniques he routinely does for patients whove suffered severe burns to large swaths of their body. The techniques also use strategic cutsto expand pieces of skin up to nine times their original size.
Every centimetre counts in these life-saving reconstructive surgeries where patches of unharmed skin are removed from one site to cover another, explains the University of Manitoba professor of surgery and psychiatry.
Ta da, he says, holding up the large paper circle. Damn, that was driving me insane.
Dr. Sarvesh Logsetty's curiosity about suturing goes back to age four, to Hyderabad, India, where he would hang around the medical clinic run by his great-aunt // PHOTO BY DAVID LIPNOWSKI [BA(HONS)/08]
Finding solutions is what the 51-year-old has spent decades doing, to alleviate pain and hardship for burn patients. Each one of my research projects, he says, reflects a patients journey.
Logsetty knows Beachs well, having operated on him more than a dozen times. He was working the day Beach was rushed to University of Alberta Hospital. The surgeon remembers how this 29-year-oldwith burns to more than 90 per cent of his bodywas more worried about how others would handle the news than he was his own chances for survival (about five per cent).
Even afterwards, hes always thought about how he can help other people and how he can use his experience to help them get through, says Logsetty, who brought his expertise to UM in 2007.
One persons recovery involves a slew of professionals.
Beyond nurses to do dressing changes, there are dietitians, physiotherapists, occupational therapists, psychologists, psychiatrists and social workers. On the burn ward at HSC, the team includes UM students across disciplines. The unit admits 100 to 200 patients a year, and treats another 250 to 400 (including cases of frostbite and flesh-eating disease). About 40 per cent of patients are children. Among the adults, the majority are men, who tend to engage in riskier behaviour than women, Logsetty notes.
When compared to other health conditions, theres an added layer. With any burn, theres a sense of guilt. Did I do something that caused this? Especially as a parent. That adds to the burden, says Logsetty. Its different than something spontaneous like cancer, where it isnt that they left the oil on the stove for too long or didnt check the temperature when they put their child in the bathtub.
With any burn, theres a sense of guilt. Did I do something that caused this? Especially as a parent. That adds to the burden. SARVESH LOGSETTY
Among 20- to 60-year-olds, burns are most often flame-related mishaps involving cooking oils catching fire, accelerants flashing back while burning grass, or house fires erupting in the night, Logsetty says. Burns from scalding are most common in kids and the elderly.
His research probes what social determinants increase your chances. A study released with UM psychiatrist Dr. Jitender Sareen [MD/95, BSc(Med)/95] last year showed people with a low income were as much as five times more likely to suffer burns. And the researchers mapped high-incidence areas in Winnipeg for policymakers to target prevention strategies.
Logsetty offers a window into the daily challenges on the burn unit. Within its dual-chamber isolation rooms, theres an ongoing battle against infection, down to the microscopic fibres of hospital curtains that may harbour antibiotic-resistant bacteria.
Burn patients face a far greater risk because their injuries arent straightforward open wounds. Theres dead skin sitting there, providing a perfect food source for bacteria. And because the skin is dead, theres no link to the bodys blood. No trigger to get infection-fighting white blood cells to activate and defend. So the bacteria can happily grow, getting stronger, says Logsetty. The risk of infection is really high.
The seeping wounds are kept covered and clean. Peeling off the dressings can be excruciatingly painful and take hours. No matter what we use, the dressings tend to stick, says Logsetty. This agony is what drives him and UM collaborator Song Liu, a medical microbiology and infectious diseases researcher, also in the Rady Faculty of Health Sciences, in their efforts to reinvent the burn bandage.
Together, theyre developing not only a less sticky coating but a dressing they hope will transform burn care. Theyre designing a fabric to detect infection, alert medical staff by changing colour, and then release antibiotics from withinall without having to remove it, Logsetty says.
Different colours would indicate different bugs. If the spot of colour were to grow bigger, it would tell nurses the infection wasnt getting any better. The bandage will be made of nanofibres, or straw-like chemical structures that break down when they come into contact with bacterial enzymes. The centres will be filled with either a liquid to release dye or a liquid containing the antibiotic. A bandage that both diagnoses and treats infection would be a first in the field. Logsetty says they could have a tangible product in as little as five years.
Liu also holds a patent for an antibiotic-resistant fabric, which could one day mean self-cleaning hospital curtains. He bonded a chlorine-like chemical to the curtain that kills bacteria on contact, one that not only wont wash out in the laundry but is reactivated by water. One of Logsettys recent studies showed that, within two weeks of being washed, five out of eight untreated hospital curtains tested positive for antibiotic-resistant MRSA.
PHOTOS BY AMBER BRACKEN
Leather hiking boots. Leather knee pads. A leather work belt. Beach didnt think twice about the gear he put on the morning of Aug. 24, 2003. (But he did contemplate calling in sickjust a feeling that he should, but one he ignored.)
The leather is what saved the less than 10 per cent of Beachs body surface that wasnt burned. His feet below the ankles. A patch in the middle of each knee. His waistline.
Theres a line around my waist where you could see where my belt was, he says.
Only once the vapours and air pressure subsided in the home was he able to open the door to the garage and escape. A neighbour then came running with a hose.
Since that day, Beach has undergone 38 surgeries.
His burns reveal the most severe form: third- and fourth-degree, which tear through the epidermis and dermis and extend into the muscle, fat and bones. At first, these sites (that appear white or charred-black) are the least painful for patients, since the flames have destroyed the nerve endings.
While lesser burns can heal on their ownas cells lining our sweat ducts and hair follicles automatically spread out to rebuildsevere burns require grafting.
Skin from Beachs feet is now on his face. As is skin from his knees and hip. Pieces of his foreskin form his delicate, upper eyelids while skin from his scrotum shapes his lower.
Logsetty can use meshing instruments that stretch and expand what small percentage of skin survivesthe graphs look like criss-crossed grids, almost translucentbut each time he does, the skin gets thinner, leaving more room for scar tissue to fill in the gaps. Its the scarring that leads to chronic pain.
[With Beach] I had to take three per cent and expand it into more than 90 per cent, somehow or another, says Logsetty. The key to advancing burn care, he says, is to develop ways to grow better skin.
[With Beach] I had to take three per cent and expand it into more than 90 per cent, somehow or another. SARVESH LOGSETTY
Beach received synthetic skin, as well as skin from cadavers, before new skin was cultured from his own cells. With Logsetty at the helm, Beach became the firstand is still the onlyCanadian to receive a unique, double-layer skin thats more resilient than anything thats come before. A sample of his skin, the size of a business card, was cultured in a lab in Cincinnati, OH, and then multiplied time and time again to eventually cover nearly half his body.
Experimental in the early 2000s, the technique is still innovative today, says Logsetty, but not yet widely available because the company has faced hurdles bringing it to market. From an overall standards of burn care, its a gamechanger, he says.
Even though there are still improvements to makethe skin doesnt contain pigment, hair follicles or sweat ducts (so on a hot summer day, Beach has to watch for heat stroke)it means less scarring and greater quality of life, says Logsetty.
Hes also in the process of developing a research project at UM that will explore new ways to use stem cells to create skin, and is collaborating with a Quebec company, Loex, on a similarly robust skin alternative.
Skin-grafting surgeries can be marathons of endurance and difficulty. An intensive operation can take 12 hours, but shorter is best since patients are already so unstable going in. The temperature of the room is kept at 29.9C to prevent patients from becoming hypothermic. Their whole body is exposed on the tablein order to graft different areasand without skin, theyre without a key organ that helps regulate body temperature. When our temperature drops, we bleed more, so theres also a greater risk of bleeding out on the table. Disposable warming blankets, inflated with warm air, help retain heat.
In the stifling environment, Logsetty and the team wear surgical gowns made of Gortex or other waterproof fabrics. (One of his research projects studies the effects on the medical team, including how much weight they lose from sweat during a procedure.)
On this ward, named after Manitoba firefighters, the mortality rate is less than three per cent, on par with the top burn centres across North America, notes Logsetty. Not many of their patients are firefighters nowadays, given how safety training and gear have evolved. But unfortunately, when they do become injured, he says, its usually devastating.
Nine months after the fire, Beach rolled over for the first time. He had lost 63 pounds and at six-foot-two was down to 112. The scar tissue had built up on his ligaments and tendons, and his muscles were wasting away with atrophy. The movement was small but it felt like a big win that came just in time. After the fire, he was angry, depressed, suicidal; now he wanted to see what else he could do for himself.
With progress comes greater survival rates, which mean more people living with the long-term consequences of burn injuries like disability, financial problems and chronic pain. Trauma survivors are at least four times more likely to take their own life, Logsetty and Sareen revealed in a 2014 study. Theyve since discovered theyre also twice as likely to have depression, anxiety or substance-abuse issues.
The standard of care I try to hold myself toand teach my studentsis What would you expect for you or your loved one? SARVESH LOGSETTY
Logsetty says patients often tell him they dont want to go on. He helps them reintegrate with the life they once had, as much as possible. Its not, I fixed your hernia, your sutures are out, you can call me if you have a problem. There is a continuity of care we dont see in most other surgery.
Thats why hes made this his lifes work. One patient describes Logsetty as the most caring and considerate doctor I have ever met; another says he created a place of love in the burn unit.
The only burn expert between Edmonton and Toronto, he makes himself available 24-7 to residents and nurses, even when not officially on call. The standard of care I try to hold myself toand teach my studentsis What would you expect for you or your loved one? says the father of two kids (under age seven), and husband to epidemiologist Rae Spiwak [BA(Adv)/00, MSc/04, PhD/17], who also studies mental-health issues in trauma patients. The biggest thing Ive learned is that life can change in an instant.
This summer, Logsetty spoke at Winnipegs inaugural Face Equality Awareness event for people living with facial differences. Its important, he adds, to help people understand that, although the outside of somebody might have changed, the inside is still the samepart of what our team does really well is help burn survivors come to that understanding themselves.
It was Beachs wife who held up the mirror for him the first time, only once hed consulted with a psychologist. He couldnt bring himself to look beyond his nose, with its missing lobes and exposed bridge. Gone was the dimpled grin of a guy who was always the life of the party.
Now, if kids stare at the grocery store, hell engage with a smile and a wave. Often, they think hes just really olda grandpa, not a father, to his kids, he says. When adults approach, which hes totally fine with, its always the same question: Can I ask what happened?
Beach doesnt have photos of what he used to look like up in his house, only because theyre not picture people. And no longer does he appear as his former self in his dreams.
Im extremely proud of who I am, Beach says.
Hes a motivational speaker who finds fulfillment in trying to create positive change in the workplacewhos spoken to Winnipeg workers about putting safety before money and supervisors demands. But his life isnt without ongoing challenges.
He has nerve damage and reduced mobility in his joints.
(He says he has the equivalent of seven-and-a-half fingers, since doctors had to amputate portions, up until they found blood flow.) And with some stubborn wounds that wont heal, he regularly gets blood infections20 in the last 10 years. Nonetheless, he renovated his basement and next, hell build a fence.
Im extremely proud of who I am. SPENCER BEACH
With burn survivors like Beach, Logsetty notes, The scar doesnt define them. They define themselves.
In a recent Facebook post, he signed off one tough son-of-a Beach.
You want to be the person you used to be, Beach says, but now you have a different body to do it with.
He returnedjust onceto the site where it happened. Where a new house now stands.
I had to see it.
Early in his career Dr. Sarvesh Logsetty saw how each burn unit across the country was labouring in its own bubble. We have some very good burn centres across Canadapeople are doing great work and researchbut we didnt really work together as a burn program in Canada as a community.
When he joined UM in 2007 he established the Advancement of Burn Care Network and made Winnipeg its base. And last year he launched the Canadian Burn Association and annual symposium further connecting the multidisciplinary players in burn care including firefighters and survivors to learn whats working whats not and what to try next.
He says research in burn treatments is grossly underfunded since there are fewer champions for the cause. Were still at the infancy of really understanding wound healing says Logsetty how to improve it how to avoid scars where we can and how we can make them better.
While the frequency of burns has dropped dramatically since the 1960swith greater safety awareness smoke detectors and legislation to safeguard water heaterstrauma as a whole is still the leading cause of death in Canadians 40 and under. It costs the system more money than just about any other health-care problem thats out there, yet we barely hear about traumatic injury says Logsetty who as a general surgeon is also tasked with removing knives from abdomens after weekend violence, or bowels burst in car collisions. In the last week alone hes removed two spleens ruptured in crashes.
What frustrates me from an academic perspective is that trauma doesnt have a home. There is no institute for traumait gets lumped in under muscoskeletal health and arthritis. That means, in terms of resources, were struggling with identification of the importance of trauma and struggling with helping people understand why we do the research we do and how it affects the people that it affects.
Go here to see the original:
UM Today | UM Today the Magazine | Fall 2019 | After the Fire - UM Today
Meet the biohackers seeking to turbocharge their bodies and minds – Euronews
By daniellenierenberg
Biohackers believe that we should be using all the technology available to make our bodies and minds work the best they can in everyday life. And they held a summit about it in Helsinki over the weekend
Most people who've heard of Biohacking think of electronic chips inserted under the skin - Cyborg stuff. But here they're promoting a wearable ring to measure the body.
People like Ramsey who's testing out a machine which steadily takes the body to air conditions you'd expect at high altitude...believe in using all the information and modern technology available to optimse human performance.
"I feel amazing - mentally and cognitively - like a stoic. I wake up every morning feeling like the Hulk," says Ramsey Morgan - Biohacker from Seattle, USA.
The movement is trying to make itself more mainstream and accessible.
"That can be like nutrition and diet, that can be taking a sauna, that can be just meditating, that can be injecting yourself with stem cells or something like that. All of these things are exapmles of Biohacking. You're changing your physiological state in order to achieve a certain goal," explains Siim Land - Estonian Biohacker.
And while most of us probably don't get enough sleep, the Biohackers say there's vibration technology to help.
"It affects to the nervous system by calming down the sypathetic side, the fight and fleet [flight] side. So basically, when you calm that down, the sleep comes naturally. You don't have to take any pills or anything," says Katja Nyman - Neurosonic.
One of the products here at the Biohackers summit is the Vielight Neuron, and our reporter, Jack Parrock tested it out.
"This a photobiomodulation device , so this applicator goes inside your nostril like that. And then the headpiece goes on top of your head," Gennady Lemud, VieLight Communications and Marketing Director tells our reporter.
The light rays being pumped onto my head and up my nose are intended to increase oxygenation in the blood and boost performance and happiness. But at well over 15 hundred euros, these devices aren't cheap.
Some Biohackers use blood tests to regularly check their liver function. One of the most controversial aspects of Biohacking is DNA testing. The medical community is still cautious and there are concerns about the data that's harvested by companies. They say there's nothing to worry about.
"We're looking at a few snips, a few genes...100...nothing. So we can't use that information for anything more than delivering information back to you as the consumer or the customer," says Chris Moore - Nordic Laboratories
It's not all so technical - getting in a sauna and a 4 degree celsius bath is enough for some Biohackers. But with the ever evolving technological world we live in - these guys think they're the future.
See the article here:
Meet the biohackers seeking to turbocharge their bodies and minds - Euronews
Light-activated pancreatic cells produce insulin on demand – New Atlas
By daniellenierenberg
Diabetes is one of the leading health problems in our modern world and requires the careful management of a patients insulin levels. New research from Tufts University may make that process a little easier. In mouse tests, the team implanted beta cells that produce more insulin on demand, when theyre activated by blue light.
At the heart of both types of diabetes is insulin, the hormone that regulates blood sugar levels, allowing cells in the body to properly use it as energy. In type I diabetes, beta cells in the pancreas dont produce enough insulin, sometimes because the immune system destroys those vital beta cells. In type II diabetes, a patients cells stop responding to insulin, or the pancreas cant keep up with demand, meaning blood glucose levels spike to dangerous highs.
Managing the condition requires constant monitoring of blood sugar levels and boosting insulin levels as needed, either by directly injecting the hormone or through drugs that amplify the beta cells production of it.
For the new study, the Tufts researchers engineered pancreatic beta cells that can produce insulin on demand in this case, that demand is pulses of blue light. The beta cells were engineered with a gene that creates an enzyme called photoactivatable adenylate cyclase (PAC) essentially, when these enzymes are activated by blue light, they produce a molecule called cyclic adenosine monophosphate (cAMP).
In turn, this molecule instructs the beta cell to produce more insulin, but interestingly, it will only do so when theres already a high level of glucose. That helps to prevent a common complication of diabetes treatments, where producing too much insulin can cause the body to consume the available glucose too quickly, resulting in low blood sugar.
To test the new technique, the Tufts team implanted their engineered pancreatic beta cells under the skin of diabetic mice. The researchers found that the cells produced between two and three times more insulin when triggered by blue light and high glucose levels. Importantly, when they fired up the blue light while glucose was low, there was no bump in insulin, indicating that the failsafe worked.
In this way, we can help in a diabetic context to better control and maintain appropriate levels of glucose without pharmacological intervention, says Emmanuel Tzanakakis, corresponding author of the study. The cells do the work of insulin production naturally and the regulatory circuits within them work the same; we just boost the amount of cAMP transiently in beta cells to get them to make more insulin only when its needed.
Similar studies have shown promise in managing diabetes with implanted beta cells either synthetic versions or natural ones produced from a patients own stem cells. Theres still plenty of work to do before this type of treatment makes it to human trials, but the researchers say that using light is a step in the right direction.
There are several advantages to using light to control treatment, says Fan Zhang, first author of the study. Obviously, the response is immediate; and despite the increased secretion of insulin, the amount of oxygen consumed by the cells does not change significantly as our study shows. Oxygen starvation is a common problem in studies involving transplanted pancreatic cells.
Ultimately, tiny sources of light could be embedded alongside the cells, allowing doctors to trigger them remotely when needed. Or they could be automatically activated by a glucose sensor, to fully close the loop.
The research was published in the journal ACS Synthetic Biology.
Source: Tufts University
Continue reading here:
Light-activated pancreatic cells produce insulin on demand - New Atlas
Woman Who Was Attacked By Cat Sues the Outrigger Canoe Club in Waikiki – Legal Reader
By daniellenierenberg
Leslie Mansfield, a California resident who enjoys running a Napa Valley winery and writing cookbooks filed a lawsuit against the Outrigger Canoe Club in Waikiki after her foot was allegedly attacked by a cat. Eventually, the bite marks caused a rare, incurable condition known as host versus graft disease, prompting Mansfield to file the suit.
The incident occurred in September 2015 when Leslie and her husband were visiting the Outrigger Canoe Club to celebrate the end of her leukemia treatments. In the middle of having lunch at the clubs Hau Terrace restaurant, a cat suddenly jumped from a nearby bush and attacked her foot. Mansfield said, all of a sudden I felt this unbelievable sharp, excruciating biteWithin a week it was worse and the bite marks were black and it was really frightening.
According to the lawsuit, the infection from the bite continued to worsen and eventually she began to develop lesions in her mouth, on her skin, and throughout her body. She said, the lesions in my mouth are so swollen around my tongue and cheeks I have deep crevasse-like cuts in the roof of my mouth.
How did a simple cat bite get so infected, though? Well, because Mansfield had recently undergone a stem cell transplant, the bite compromised her immune system. According to Mansfield, who had stem cells donated from her brother, doctors told her that when she got bit by the cat, those cells not only began attacking the pathogens introduced by the cat but they also started to attack her system.
As a result, Mansfield experiences regular painful flares that leave her exhausted and unable to do much of anything. Her quality of life has been diminished and she blames the Outrigger Canoe club that harbored the cat.
When commenting on the matter, attorney Jim Bickerton who is representing Mansfield said, the cat spent its entire existence on those premises. It wasnt a stray that lived somewhere else and came visiting. This was home for this cat. He added that under Hawaii law, the club is not only responsible for the cat bite but its also responsible for the subsequent damage to his clients immune system. He said, if someone has very brittle bones, for example, and they take a small fallYou or I might just fracture a bone or not even have a fracture but they have fractures in 20 places. The person who caused that fall owns all of the damage.
In response to the lawsuit, a spokesperson for the club said, The health, safety, and well-being of all of our members, guests and staff are of primary importance to the Outrigger.
The suit is expected to go to trial next August.
Lawsuit: Cat bite at Outrigger Canoe Club caused womans rare disease
GRAFT-VERSUS-HOST DISEASE
View original post here:
Woman Who Was Attacked By Cat Sues the Outrigger Canoe Club in Waikiki - Legal Reader
How Stem Cells Can Heal The Body – Version Weekly
By daniellenierenberg
Stem cells from the patients body when isolated and administered at an appropriate time and at the right place, with the right dose, is expected to help the patient in various ways
Stem Cells In The Body
All humans are born and develop from a small tiny structure called an egg. The cells in the egg have a tremendous potential to develop, multiply and form different cells that are functional in the body. These cells are called mother cells or in scientific terms, they are called stem cells. And all human beings have these stem cells preserved in the body. It is these cells that help us in every day wear and tear and also for tissue repair.
The Body Can Heal Itself
Most of the cells in our body have a definite lifespan that need to be replaced by new cells. The stem cell reserves in the body make up for this and it is done without our knowledge! In fact, any cut or injury, external or internal is healed by the bodys innate mechanism. Our intelligent body recognises the signal of injury and recruits the required stem cells. These stem cells transform themselves into the cells that are required for the repair of the injury and it is always many types of cells in various permutations and combinations.
Where Stem Cells Reside
Bone marrow can be considered as the manufacturing unit of stem cells as it is continuously making blood cells and keeps our circulatory system working perfect all the time. Circulating blood is another source of stem cells, because it works as a courier, carrying cells and other essential enzymes, hormones from one organ to the other in the body. The body converts all the extra material into fat which gets accumulated around the belly. This fatty tissue works like a fixed deposit of stem cells.
Stem cells either from the donor (allogenic) or from the patient (autologous) are being used for more than 50 years and especially for treatment. Blood cancers and other blood-related diseases can be cured using a perfect matched donor stem cells obtained from bone marrow. Patients suffering from organ cancers like breast cancer etc. are given autologous stem cells as a supportive treatment along with chemotherapy and/or radiation.
Protocols for these treatments are standardised globally and considered as standard-of-care. In recent years, umbilical cord blood derived stem cells are being used as an alternative to bone marrow, especially in the paediatric age group. People fall victim to numerous degenerative diseases which occur, as the repairing stem cell system from the body fails slowly with age. Stem cells from the patients body when isolated and administered at an appropriate time and at the right place, with the right dose, is expected to help the patient in various ways. It may also replace, rejuvenate or restore the damaged tissues.
Our body carnes its own repairing kit in the form of stem cells and the body tries its level best to make use of these stem cells to ward off diseases. However, it is possible that with age, the bodys power to recruit and make use of the stem cells diminishes slowly. This is when dreadful degenerative diseases like diabetes, arthritis, Parkinsons disease and heart problems, set in. Heres what the clinical applications of regenerative medicine have found novel mechanisms of:
It is increasingly observed that this kind of autologous therapy takes care of the root cause of disease and offers benefits to patients to whom there is no further solution in other modalities of treatment.
Since each tissue and organ of our body is made up of cells that are derived from the egg cell, any disease which is due to derangement or degeneration of cells can be cured using autologous cellular therapy. And though the list can be endless, here are some examples where there have been very promising results:
Read the original post:
How Stem Cells Can Heal The Body - Version Weekly
Interview With Chaim Lebovits, CEO Of Brainstorm Cell Therapeutics – Seeking Alpha
By daniellenierenberg
This level of disease stabilization has not been observed to this date in approved or investigational ALS therapies.
-Mr Chaim Lebovits, CEO, Brainstorm Cell Therapeutics
In May of this year, I published an article on Brainstorm Cell Therapeutics (BCLI). This small company is developing a Mesenchymal stem cell product called NurOwn, which is in late phase 3 trials targeting Amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease. My article was bearish, deploring not only the company's cash position, but also phase 2 trial data. The article can be read here.
That article received a lot of critical comments from the ALS community. That made me realize that a fair overview of the issues could be best addressed by going through the comments, as well as my own coverage, and by asking BCLI management, specifically its CEO, Chaim Lebovits, to clarify some of these issues. So that's what I did. I emailed a set of 11 questions to Mr Lebovits, and he was kind enough to respond to them in great detail. The entire interview, sans any edits, is available to Total Pharma Tracker members.
Mr Lebovits has been with BCLI for well over 12 years, joining in 2007 as president and also becoming the CEO in 2015. He has helped develop NurOwn through its preclinical stage to its current stage, and is therefore just the right person to talk to if we want to understand NurOwn and BCLI.
I began by asking him to locate NurOwn in the ALS therapy space and where it stands with respect to competitors. What's its mechanism of action, and how does that MOA distinguish it from competition?
Mr Lebovits said that there are "currently 4 products active in phase 3 ALS clinical trials (Brainstorm (NurOwn, autologous MSC-NTF cells secreting neurotrophic factors), Orion (levosimendan, muscle troponin calcium sensitizer), Orphazyme (arimochlomol, heat shock protein enhancer), and Biogen (SOD1, antisense oligonucleotide)." Top line data from these ALS phase 3 trials is expected in 2020 (Q4 2020 for Brainstorm) and Orion, 2021 (Orphazyme), and 2022 (Biogen). He discussed a number of earlier stage compounds, as well as various stem cell therapies. He said that what distinguishes NurOwn among ALS therapies is that it "confers both neuroprotection and immunomodulation by delivering neuronal survival factors and immune regulatory molecules, including microRNA directly to the CNS compartment at or near the site of disease, and therefore directly addresses two important ALS disease mechanisms."
Among stem cell therapies, Mr Lebovits said that NurOwn distinguishes itself by being autologous and because it can produce high levels of neurotrophic factors. Moreover, unlike most stem cell competitors, it's delivered directly into the spinal fluid through bimonthly lumber punctures, unlike others which need an invasive surgical procedure "that carries considerable morbidity."
This feature it shares with a competing product from Corestem. However, it's differentiated from Corestem because "NurOwn is more convenient than the Corestem product as a single bone marrow cell harvest due to validated cryopreservation, whereas the Corestem product requires repeat bone-marrow aspiration for each treatment."
My next question was a technical question about pharmacoresistance. I wanted to know how NurOwn is managing to cross the blood-spinal cord barrier despite the strong pharmacoresistance (body's resistance to drugs) seen in ALS, specifically for disease-modifying neurotrophic factors. What was it about NurOwn's delivery mechanism that the company thinks is overcoming this natural resistance. So I asked: "Talking about MOA, pharmacoresistance is a disease driving mechanism in ALS. Can you discuss NurOwns delivery mechanism vis-a-vis the inability of neurotrophic factors to effectively cross the blood-brain barrier, or, specifically, the blood-spinal cord barrier (BSCB)? Please correlate that discussion regarding the observed increase in CSF NTFs post treatment as seen in the phase 2 trial."
Mr Lebovits explained this with great clarity - for his entire response, take a look at the complete interview. Broadly, what he said was that NurOwn, being delivered through lumber puncture directly into the spinal fluid, has an advantage. Moreover, the cells secrete neuronal survival factors as well as molecules that regulate the immune system, so that they are able to survive and overcome the pharmacoresistance. Systemically administered NTFs are unable to do that.
As he said, "In the phase 2 trial, CSF biomarkers obtained just prior to treatment and two weeks afterwards demonstrated that MSC-NTF cell secreted neurotrophic factors were significantly increased post treatment and correlated with the reduction in inflammatory biomarkers, consistent with the proposed mechanism of action."
My third and fourth questions related to aspects of the phase 2 study. One, comparison of safety and efficacy data with competitors, and two, the relevance of the reported Caspace-3 reduction of 60% in responders versus 30% in non-responders.
Mr Lebovits said that although the phase 2 study was not powered for efficacy, it exhibited a "level of disease stabilization (that) has not been observed to this date in approved or investigational ALS therapies." About the ongoing phase 3 study, he said the following:
Those who read my original article will recall I was particularly puzzled by the increased occurrence of serious adverse events in active-treatment groups than in placebo groups. 8/36 or 22.2% patients in the treatment arm had an SAE compared to only one out of 12 placebo patients, or 8.3%. Most SAEs were related to the progression of the underlying ALS, most commonly dysphagia. No SAEs were related to study treatment. So I asked Mr Lebovits how this data could be interpreted in the most positive way.
According to him, this decline was not an effect of treatment itself and simply indicated the need for repeat dosing in this patient group. His exact response was as follows:
The MSC-NTF treated group had a slightly more rapid rate of decline compared to the placebo group in the three-month run-in period and most ALS disease progression in the treated group was seen toward the end of the clinical trial, long after a single transplantation. In fact, the bulbar subscale, that includes assessment of swallowing, was the subscale most improved after MSC-NTF treatment in rapid progressors, suggesting that the late decline in motor function was not an adverse effect of treatment per se. Hence the need for repeated dosing.
Last week, the DSMB recommended continuation of the phase 3 trial without any modification. This was major good news, so we asked him about this. Mr Lebovits said that this was a second interim safety review, and there was no significant safety concerns. Therefore, the DSMB recommended no modification in protocol, and no other interim analysis is planned. Phase 3 data will be available by mid-2020 according to this interviewer's reading of the press release.
Now we moved on to another critical aspect of our analysis - funds, or rather, the lack of it. Since this is an important issue, here's the exact exchange we had.
Dr. Ashok Dutta: How does the company plan to fund its operations through the next couple years until the lead development candidate is approved and commercialized? Given the weak financial position, does Brainstorm see the possibility for ATM operations, or thinks about selling rights in regions like China, Japan or Europe to increase the financial condition?
CEO Chaim Lebovits: As you are aware we do receive proceeds from the hospital exemption pathway and also receive grant funding from CIRM and IIA. These avenues have allowed to fund and continue with our trials over the years with non-dilutive financing. From a business standpoint as our ALS phase 3 trial is now fully enrolled, the management team continues to hold high level conversation with some of the leading global pharmaceutical and biotechnology companies. We are actively engaged in strategic partnering and collaboration discussions and although we cannot disclose the details of our conversations due to NDAs we signed with them... we are exploring several opportunities with key interested parties to advance the opportunities for NurOwn development and commercialization. As you have rightly pointed out, we have a $20mm ATM facility in place with Raymond James. We may activate the ATM as required and raise up to $20mm by selling our stock at the market only if the prices are attractive to us. So far as of end of Q319, we have not activated the ATM. If the need arises and the prices are attractive to us, we may employ this tool to raise capital.
This is reassuring that the company intends to focus on non-dilutive financing. The ATM facility, coupled with the grants, should ideally see them through the approval phase. We still wonder how they will manage marketing and sales. Perhaps those commercialization NDAs they have signed will help.
Next, we discussed market potential and a question about a recent patent grant. The CEO's detailed responses can be found in the complete interview material.
The strong involvement of the ALS community impressed us previously, so we now asked the CEO about the recent roundtable convention they had with ALS advocacy groups. Since this will be important for the ALS community as a whole, here's Mr Lebovits' entire response on the question:
Finally, we asked him what we ask everyone: Give us three simple and straightforward reasons why investors would be interested. Here's what he said:
Thanks to the ALS community for inspiring us to conduct this interview, and to Mr Chaim Lebovits, CEO of Brainstorm Cell Therapeutics, for answering our questions.
Thanks for reading. At the Total Pharma Tracker, we interview management of important small biotech doing disruptive work in healthcare. Our members are given exclusive access to these interviews, which helps them with additional primary resource in doing DD on their investments. Sometimes, extracts from these interviews may be published for everyone; but TPT members always get the exclusive view.
Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.
Additional disclosure: General Disclaimer - This is to confirm that Avisol Capital Partners has neither requested, nor been offered, any monetary compensation for conducting this interview, by any party other than Seeking Alpha.
Also to be noted, this was an emailed questionnaire, and certain editorial material is present in this version, which may or may not reflect BCLI or its CEO's position on the issues discussed.
Read more here:
Interview With Chaim Lebovits, CEO Of Brainstorm Cell Therapeutics - Seeking Alpha
Growing Human Organs In A Lab: As Scientists Develop Pathbreaking Three-Organ System, Heres All You Need To Know – Swarajya
By daniellenierenberg
In September end, a good news greeted the biomedical world when a team led by Takanori Takebe at Cincinnati Children's Hospital Medical Center succeeded at growing a connected set of three organs: the liver, pancreas and biliary ducts, in the lab, from human stem cells. The findings were published in journal Nature.
While human organoids already provide a sophisticated tool for research, the connected set of three organs, for the first time, allow scientists to study how human tissues work in concert. This was dubbed as a significant step forward.
In October, another news came. At the annual meeting of the Society for Neuroscience, researchers said that brain cell clusters prepared in the lab- a type of organoid- show abnormal behaviour as compared to the normal brain cells.
They said that the cells in these clumps had ambiguous identities and made more stress molecules than cells taken directly from human brains. However, these abnormalities were found to be alleviated a little bit when the implanted into a more hospitable environment - a mouses brain.
What are organoids?
With the available technology, scientists can grow a group of cells in laboratories into three-dimensional, miniature structures that mimic the cell arrangement of a fully-grown organ.
This is done using stem cells.
Stem cells are special human cells that have the ability to develop into many different cell types, from muscle cells to brain cells.
The embryonic stem cells that are derived from unused embryos (These are created from an in vitro fertilization procedure and used for scientific research) are pluripotent, meaning, they can turn into any type of cell.
On the other hand are adult stem cells. They are derived from fully developed tissues, like the brain, skin, and bone marrow. These cells often have capability of turning into only certain types of cells. For example, a stem cell derived from the liver will only generate more liver cells.
However, the adult stem cells can be manipualted in the laboratory to act like embryonic stem cells. These are called induced pluripotent stem cells. (The technique was developed in 2006). However, scientists are yet to find adult pluripotent stem cells that can develop every kind of cell and tissue.
When scientists create right environment in the laboratory for them, these stem cells follow their own genetic instructions to develop into tiny structures that resemble miniature organs composed of many cell types.
Using these, researchers have been able to produce organoids that resemble the brain, kidney, lung, intestine, stomach, and liver etc.
For example, in the three-organ research mentioned above, Dr Takebe started with stem cells from human skin cells and then guiding and prodding those stem cells to form two very early-stage "spheroids" of cells loosely termed the foregut and the midgut (In human embryos, these form late in the first month of gestation. Over time, they merge and morph into the organs that constitute the digestive tract).
The spheroids were first placed next to each other in a lab dish suspended in a gel used to support organoid growth, then placed on top of a thin membrane that covered a carefully mixed batch of growth medium.
From this point on, the cells knew what to do, and 70 days later, the mini organoids began processing bile acids as if they were digesting and filtering food.
Why are organoids important?
The technique to develop organoids was named by The Scientist as one of the biggest scientific advancements of 2013.
Organoids are an excellent tools to study biological processes like uptake of nutrients, drug transport, secretion of hormones and enzymes etc. This way, diseases related to malabsorption of nutrients, and metabolism-related diseases like obesity, diabetes, insulin resistance can be studied at the cellular-level.
Recently, scientists at the at Memorial Sloan Kettering created a tumor organoid to develop a more accurate rectal cancer model.
In the case of the human brain, organoids opens a window to understand some of the most complicated and hidden aspects of our own biology. They can be used to study neuropsychiatric or neurodevelopmental diseases like schizophrenia or autism spectrum disorder, which are uniquely human diseases that affect the whole human genome.
Organoids also provide a window into how cells interact with each other and their environment. They can be used to create cellular models of human disease, which can be studied in the laboratory to better understand the causes of disease and identify possible treatments. The effects of different drugs and be tested.
Scientists have even used gene editing techniques (CRISPR-Cas9) on the stem cells to to introduce targeted mutations in genes corresponding to two different kidney diseases. When these modified pluripotent cells grew into human kidney organoids, they exhibited the diseases.
Using such organoids relieves the scientific community from experimenting on human and animal subjects. Also, certain treatments that would be unethical to administer on the latter, can be tested on the organoids.
With organoids, researchers can produce a limitless supply of tissue from each patient. This will also be extremely useful for the study of rare diseases, where the number of patients on which to conduct research and test treatments is limited.
Organoids are also being used to develop personalised and precision medicine.
For example, it was found that repairing the CFTR protein could give relief to a patient suffering from non-cystic fibrosis, an inherited disease caused due to a gene mutation. Using the Intestinal organoids grown from a patients stem cells, the doctors could quantify the patients response to the CFTR modulating therapy.
Organoids can have significant therapeutic applications. For example, pluripotent stem cells derived from a diabetes patient could be transformed into insulin-producing beta-like cells.
Organoids also offer an incredible opportunity to study developmental biology. Using them, for example, we can learn more about how organs are formed in embryonic stages and associated disorders.
The rest is here:
Growing Human Organs In A Lab: As Scientists Develop Pathbreaking Three-Organ System, Heres All You Need To Know - Swarajya
Imago BioSciences Further Expands Executive Team with Appointment of James D. Watson as Chief Business Officer – Yahoo Finance
By daniellenierenberg
SAN FRANCISCO--(BUSINESS WIRE)--
Imago BioSciences, Inc., a clinical-stage biotechnology company developing innovative treatments for malignant and life-threatening diseases of the bone marrow, today announced the appointment of James D. Watson as Chief Business Officer.
James has a long history of success in biotech financing, business development, and commercial planning. That experience will guide the strategic direction and growth of Imago, said Hugh Young Rienhoff, Jr., M.D., Chief Executive Officer of Imago BioSciences. His experience in corporate development and financings will help ensure the advancement of bomedemstat (IMG-7289) through clinical development in myelofibrosis and other indications.
Mr. Watson most recently served as Chief Business Officer at Sigilon Therapeutics where he closed a $473 million strategic partnership with Eli Lilly for a treatment for Type 1 diabetes. Prior to Sigilon, Mr. Watson was Chief Business Officer for Alvine Pharmaceuticals and led strategy, corporate development, new product planning, and finance during which he closed a transaction giving AbbVie the right to acquire Alvine for $345 million. Previously, Mr. Watson was CEO of a San Francisco-based, boutique investment bank focused on mergers, acquisitions, partnering, and raising capital for life science companies.
I am excited to join Imago BioSciences at this important stage of growth, helping the company realize its full potential. Bomedemstat has great promise for the day-to-day management of myelofibrosis and it offers the additional possibility of altering the course of this disease. Furthermore, its broader myeloproliferative neoplasm platform and expertise in life-threatening diseases of the bone marrow represent an opportunity to address areas of high unmet clinical need and to build a valuable company. said Mr. Watson.
About Bomedemstat (IMG-7289)
Bomedemstat is a small molecule discovered by Imago BioSciences that inhibits lysine-specific demethylase 1 (LSD1 or KDM1A), an enzyme essential for production and normal function of megakaryocytes and for self-renewal of malignant hematopoietic stem or progenitor cells. Megakaryocytes are the primary producer of growth factors and cytokines that drive myelofibrosis pathogenesis.
In non-clinical studies, bomedemstat demonstrated robust in vivo efficacy as a single agent and in combination with other therapeutics across a range of myeloid malignancy models including the myeloproliferative neoplasms encompassing myelofibrosis, essential thrombocythemia and polycythemia vera. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to bomedemstat for the treatment of myelofibrosis which is currently being studied in an international Phase 2b study. Additional clinical studies in hematologic disorders will begin in 2020.
About Imago BioSciences
Imago BioSciences is a clinical-stage, private therapeutics company focused on malignant and life-threatening diseases of the bone marrow. The initial clinical focus is on myeloproliferative neoplasm (MPN) disorders including myelofibrosis, essential thrombocythemia and polycythemia vera. Investors in Imago include a fund managed by Blackstone Life Sciences, Frazier Healthcare Partners, Omega Funds, Amgen Ventures, MRL Ventures Fund, HighLight Capital, Pharmaron, Greenspring Associates and Xeraya Capital.
View source version on businesswire.com: https://www.businesswire.com/news/home/20191104005265/en/
Latest Released Report on Autologous Stem Cell Based Therapies Market to Witness the Highest Growth Globally in Coming Years: Osiris…
By daniellenierenberg
Crystal Market Research has recently updated its massive report catalog by adding a fresh study titled Global Autologous Stem Cell Based Therapies Market Report 2019. The Autologous Stem Cell Based Therapies market report presents an analytical study that is defined based on the various parameters and trends followed by the global Autologous Stem Cell Based Therapies market. The report contains the assessment of futuristic growth based on past growth models and currently accompanied by the market. Extensive information on factors entered and market growth forecasts are also included in the market.
Global Autologous Stem Cell Based Therapies Market report provides an in-depth study of industry size, share, trend, opportunities within the latest research report added by CMR. The report consists of market sizes and forecast for the period from 2019 to 2025, and compounded annual growth rate (CAGR%) measured for individual segments and regional markets, competitive landscape of main market players, vital analysis of market dynamics and profiling of key providers collaborating in the Autologous Stem Cell Based Therapies market.
Continue
Get overview of FREE sample report @ https://www.crystalmarketresearch.com/report-sample/HC022437
Read more or inquiry about Autologous Stem Cell Based Therapies research @ https://www.crystalmarketresearch.com/send-an-enquiry/HC022437
Access discount related details @ https://www.crystalmarketresearch.com/check-discount/HC022437
Contacts Us:
Judy | 304 South Jones Blvd, Suite 1896
Las Vegas NV 89107
United States
E-mail:
sales@crystalmarketresearch.com
| Ph: +1-888-213-4282