Kadimastem to Present Interim Results of Cohort A of Its Phase 1/2a Clinical Trial in ALS at the 7th International Stem Cell Meeting, in Tel-Aviv,…
By daniellenierenberg
NESS ZIONA, Israel, Nov. 11, 2019 /PRNewswire/ --Kadimastem Ltd.(TASE: KDST),a clinical stage cell therapy company, today announced that it will present the interim results of Cohort A of its ongoing Phase 1/2a Clinical Trial in ALS (as published in Company's press release) at the 7th International Stem Cell Meeting, to be held on November 12-13 at the Dan Panorama Hotel in Tel Aviv, Israel.
The International Stem Cell Meeting, hosted by the Israel Stem Cell Society, is a highly reputed conference, participated by international world leaders in stem cell research.
Presentation Details:
Title: "FIRST IN HUMAN CLINICAL TRIALS WITH HUMAN ASTROCYTES AS A NOVEL CELL THERAPY FOR THE TREATMENT OF ALS"
Session:ONGOING CLINICAL TRIALS WITH CELL THERAPY
Presenter:Arik Hasson, PhD, Executive VP, Research and Development, Kadimastem
Date:Wednesday, November 13, 2019
Time:1:50 pm Israel
Location: Dan Panorama Hotel, Tel Aviv, Israel
Rami Epstein, CEO of Kadimastem, stated: "We are pleased to share these results with global leaders in the cell therapy and stem cells industry,demonstrating the potential of AstroRx, our astrocyte-based cell therapy product,to bring treatment to ALS patients, and possibly other neurodegenerative diseases. We look forward to further share data of this ongoing trial, with final results of cohort A expected by year-end 2019and results of cohort B expected in Q3, 2020."
About the Phase 1/2a ALS Clinical Trial
The Phase 1/2a trial is an open label, dose escalating clinical study to evaluate the safety, tolerability and preliminary efficacy of AstroRxcells in patients with ALS. The trial is expected to include 21 patients and is being conducted at the Hadassah Medical Center, Jerusalem, Israel. The primary endpoints of the trial are safety evaluation and tolerability of a single administration of allogeneic astrocytes derived from human Embryonic Stem Cells (hESC), administered in escalating low, medium and high doses (100x106, 250x106, and 500x106 cells, respectively). The medium dose will also be administered in 2 consecutive injections separated by an interval of ~60 days. Secondary end points include efficacy evaluation and measurements. Treatment is administered in addition to the appropriate standard-of-care.
About AstroRx
AstroRx is a clinical grade cell therapy product developed and manufactured by Kadimastem in its GMP-compliant facility, containing functional healthy astrocytes (nervous system support cells) derived from human Embryonic Stem Cells (hESC) that aim to protect diseased motor neurons through several mechanisms of action. The Company's technology enables the injection of AstroRxcells into the spinal cord fluid of patients suffering from Amyotrophic Lateral Sclerosis (ALS) with the goal of supporting the malfunctioning cells in the brain and spinal cord, in order to slow the progression of the disease and improve patients' quality of life and life expectancy. AstroRxhas been shown to be safe and effective in preclinical studies. AstroRxhas been granted orphan drug designation by the FDA.
About ALS
Amyotrophic Lateral Sclerosis (ALS) is a rapidly progressive fatal neurodegenerative disease causing disfunction in the upper and lower motor nerves that control muscle function. ALS leads to muscle weakness, loss of motor function, paralysis, breathing problems, and eventually death. The average life expectancy of ALS patients is 2-5 years. According to the ALS Therapy Development Institute, it is estimated that there are approximately 450,000 ALS patients worldwide of which 30,000 reside in the US. According to the ALS Foundation for Life, the annual average healthcare costs of an ALS patient in the US are estimated at US$ 200,000. Thus, the annual healthcare costs of ALS patients in the US alone amount to US$ 6 Billion.
About Kadimastem
Kadimastem is a clinical stage cell therapy company, developing and manufacturing "off-the-shelf" allogeneic proprietary cell products based on its platform technology for the expansion and differentiation of Human Embryonic Stem Cells (hESCs) into clinical grade functional cells. AstroRx, the Company's lead program, is a clinical-grade astrocyte cell therapy for the treatment of ALS, currently undergoing a Phase 1/2a clinical trial. In addition, preclinical trials are ongoing with the Company's IsletRx pancreatic functional islet cells for the treatment of insulin dependent diabetes. Kadimastem was founded by Prof. Michel Revel, CSO of the Companyand Professor Emeritus of Molecular Genetics at the Weizmann Institute of Science. Prof. Revel received the Israel Prize for the invention and development of Rebif, a multiple sclerosis blockbuster drug sold worldwide. Kadimastem is traded on the Tel Aviv Stock Exchange (TASE: KDST).
Company Contacts:Yossi Nizhar, CFOy.nizhar@kadimastem.com+972-73-797-1613
Investor and Media Contact:Meirav Gomeh-Bauermeirav@bauerg.com+972-54-476-4979
Global Media Contact:Dasy (Hadas) MandelDirector of Business Development, Kadimastemd.mandel@kadimastem.com+972-73-797-1613
SOURCE Kadimastem
Neuroscience Is Taking the Spotlight at Xconomy’s Bay Area Xchange – Xconomy
By daniellenierenberg
XconomySan Francisco
These are heady times for neuroscience research. Startups developing new approaches to brain disorders are raising money to advance their discoveries toward clinical trials. One failed neuro drug is getting another shot.
On Nov. 19 in San Francisco, well hold the latest in our Xchange event series. Whats Next in Neuroscience Therapies will take a look at new technologies that are changing how we understand brain diseases and spinal injuries, as well as novel approaches that companies are taking to treat these conditions. One such company, Alector (NASDAQ: ALEC), aims to treat neurodegeneration as an immune system problem. The South San Francisco biotech is developing antibody therapies that bolster immune cells that help the brain clear away proteins and debris associated with neurodegenerative disorders.
Earlier this year, Alector completed a $176 million IPO. The company is now deploying that cash in clinical trials: a drug candidate for frontotemporal dementia and two experimental therapies for Alzheimers disease. Stephanie Yonker, the companys vice president of legal, will talk about her companys approach to neurodegeneration at the upcoming event.
BlackThorn Therapeutics is deploying technologies such as artificial intelligence and brain imaging to help it discover new drugs and enroll the clinical trials to test them. The San Francisco companys focus is neurobehavioral disease. BlackThorn quietly emerged four years ago based on research from the Scripps Research Institute in San Diego. At our forum, Jane Tiller, BlackThorns chief medical officer, and Kristina Burow of ARCH Venture Partners will tell BlackThorns story from its Scripps origins to the present day as a clinical-stage company backed by $130 million in financing. BlackThorn has completed Phase 1 tests of it lead drug in depressionand is preparing for Phase 2; an experimental autism spectrum disorder drug is being readied to start tests in humans next year.
Spinal cord injury continues to pose obstacles to treatment. The California Institute for Regenerative Medicine is pursuing new therapies through grants awarded to universities and companies. Much of this research aims to develop ways to use stem cells to heal the injury. Abla Creasey, CIRMs vice president of therapeutics and strategic infrastructure, will discuss these efforts. Meanwhile, the Christopher & Dana Reeve Foundation is raising a venture philanthropy fund to support new research. Ethan Perlstein, the Reeve Foundations chief scientific officer, will talk about the foundations efforts to find new treatments and potential cures for spinal cord injury.
If youd like to hear more about these new efforts to treat brain disorders and spinal cord injury, join us at the Hyatt Regency San Francisco for Whats Next in Neuroscience Therapies. You can see the agenda for the event here. Additional information, including registration details, are here. We hope to see you on Nov. 19.
Photo by Depositphotos
Frank Vinluan is an Xconomy editor based in Research Triangle Park. You can reach him at fvinluan [[at]] xconomy.com.
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Neuroscience Is Taking the Spotlight at Xconomy's Bay Area Xchange - Xconomy
Novel Molecule Reduces the Aggressiveness of Pediatric Cancer – Technology Networks
By daniellenierenberg
In Brazil, scientists affiliated with the Human Genome and Stem Cell Research Center (HUG-CELL) at the University of So Paulo (USP) have identified a molecule capable of reducing the aggressiveness of embryonal central nervous system tumors. These are malignant tumors that start in fetal cells in the brain and mainly affect children up to four years old.
The results arepublishedin the journalMolecular Oncology.HUG-CELLis one of the Research, Innovation and Dissemination Centers (RIDCs) supported by So Paulo Research Foundation - FAPESP. Its principal investigator isMayana Zatz, Professor of Human and Medical Genetics at USP's Institute of Biosciences (IB).
The approach proposed by the group can be classified as a type of microRNA-based therapy. A microRNA is a small RNA molecule that does not encode protein but plays a regulatory role in the genome. In this study, researchers used a synthetic version of an inhibitor of microRNA-367 (miR-367) with anti-tumor activity.
"We demonstrated in an animal model of a central nervous system tumor that treatment with a microRNA inhibitor attenuates properties of tumor stem cells and prolongs survival," saidOswaldo Keith Okamoto, a professor at IB-USP and the principal investigator for the study.
Okamoto explained that embryonal central nervous system tumors such as medulloblastomas and atypical teratoid/rhabdoid tumors (AT/RTs) tend to contain cells with characteristics similar to those of stem cells, which boosts their tumorigenic potential and capacity to invade tissue while also making them more resistant to cell death.
These tumors are caused by genetic or epigenetic aberrations in stem cells and neural progenitors when the nervous system is being formed during embryonic development. The neural stem cells that undergo these alterations later give rise to tumor cells. They form aggressive, fast-growing tumors that may appear shortly after birth, in later childhood or in adolescence.
In a previous study, the group tested an approach that used the Zika virus to destroy tumor stem cells (read more atagencia.fapesp.br/27677).
Expression and inhibition
A more recent study was led byCarolini Kaid, a postdoctoral researcher at IB-USP with a scholarship fromFAPESP.
Previous research has already shown that OCT4A, one of the genes that encode pluripotency factors, is overexpressed in aggressive medulloblastomas and that this overexpression is associated with an unfavorable prognosis. During hermaster's research, Kaid detected the expression of miR-367, a gene that promotes stem-like traits in tumor cells, in parallel with overexpression of OCT4A (read more atagencia.fapesp.br/21959).
The researchers then tested a specific synthetic inhibitor of miR-367 containing minor chemical alterations that make it more stable in cells. A patent application has been filed for the invention.
After inducing the formation of central nervous system tumors in mice using three different strains of tumor cells, the researchers injected the miR-367 inhibitor into the brain's right lateral ventricle, a pathway to the cerebrospinal fluid that surrounds the brain and spinal cord. From there, the miR-367 inhibitor was able to access the tumor cells.
Tumor size was reduced considerably, and survival improved in all groups of mice. The results confirmed what had previously been observed in cell cultures.
In this model, the researchers noted that when the synthetic molecule interacted with miR-367 in tumor cells, it prevented this microRNA from affecting the levels of proteins it normally regulates, such as ITGAV and SUZ12.
The latter is known to be involved in silencing pluripotency-related genes in embryonic stem cells.
While the role of ITGAV in embryonal central nervous system tumors is not fully understood, ITGAV is known to participate in the renewal of both normal and tumor stem cells.
"When miR-367 is inhibited in cancer cells, it stops regulating several proteins. This molecular alteration eventually affects the properties of these cells, resulting in an attenuation of the tumor's aggressiveness. This is what makes the strategy interesting," Kaid said.
The researchers believe that in humans, the synthetic molecule alone may be capable of at least containing the development of these tumors and improving survival. Even so, they are testing combinations of the molecule with drugs currently used to treat the tumors. They want to find out whether the approaches could be combined using lower doses of chemotherapy drugs.
Before clinical trials can be performed, however, pharmacology and toxicity studies will be necessary, as will pharmacokinetic testing to show how the molecule is metabolized and how long it stays in the organism (its half-life).
When embryonal central nervous system tumors are conventionally treated with surgery, chemotherapy and/or radiotherapy, morbidity and mortality rates for these patients are high. These tumors correspond to 10% of all central nervous system cancer cases in children.
Even patients who survive longer than most may suffer from permanent treatment-related sequelae that impair their quality of life, such as problems with development, cognition, locomotion and speech.
Reference: Kaid et al. 2019.miR367 as a therapeutic target in stemlike cells from embryonal central nervous system tumors. Molecular Oncology. DOI: https://doi.org/10.1002/1878-0261.12562.
This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.
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Novel Molecule Reduces the Aggressiveness of Pediatric Cancer - Technology Networks
Sesen Bio Inc. (SESN) and BioTime Inc. (:) Contrasting side by side – FinanceMercury
By daniellenierenberg
As Biotechnology companies, Sesen Bio Inc. (NASDAQ:SESN) and BioTime Inc. (:) are our subject to contrast. And more specifically their risk, institutional ownership, analyst recommendations, profitability, dividends, earnings and valuation.
Earnings & Valuation
Table 1 shows top-line revenue, earnings per share and valuation of the two companies.
Profitability
Table 2 shows Sesen Bio Inc. and BioTime Inc.s return on assets, return on equity and net margins.
Volatility & Risk
Sesen Bio Inc. has a beta of 0.65 and its 35.00% less volatile than S&P 500. From a competition point of view, BioTime Inc. has a 2.81 beta which is 181.00% more volatile compared to S&P 500.
Liquidity
The Current Ratio and a Quick Ratio of Sesen Bio Inc. are 6.4 and 6.4. Competitively, BioTime Inc. has 3.5 and 3.5 for Current and Quick Ratio. Sesen Bio Inc.s better ability to pay short and long-term obligations than BioTime Inc.
Institutional and Insider Ownership
Institutional investors held 31.6% of Sesen Bio Inc. shares and 43.7% of BioTime Inc. shares. 6.56% are Sesen Bio Inc.s share held by insiders. Insiders Competitively, held 3.9% of BioTime Inc. shares.
Performance
In this table we provide the Weekly, Monthly, Quarterly, Half Yearly, Yearly and YTD Performance of both pretenders.
For the past year Sesen Bio Inc. has -13.38% weaker performance while BioTime Inc. has 20.48% stronger performance.
Summary
BioTime Inc. beats on 5 of the 9 factors Sesen Bio Inc.
Sesen Bio, Inc., a late-stage clinical company, develops next-generation antibody-drug conjugate therapies for patients with cancer. It develops its products based on its Targeted Protein Therapeutics (TPTs) platform. The company's lead product candidate is Vicinium, a fusion protein that is in Phase III clinical trial for the treatment of high-grade non-muscle invasive bladder cancer. It also develops Vicinium in combination with Durvalumab, which is in Phase I clinical trial for the treatment of high-grade non-muscle invasive bladder cancer; and Vicinium in combination with AstraZeneca's checkpoint inhibitor for the treatment of squamous cell carcinoma of the head and neck. In addition, the company is developing systemically-administered TPTs, including VB6-845d for the treatment of solid tumors. The company was formerly known as Eleven Biotherapeutics, Inc. and changed its name to Sesen Bio, Inc. in May 2018. Sesen Bio, Inc. was founded in 2008 and is based in Cambridge, Massachusetts.
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.
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Sesen Bio Inc. (SESN) and BioTime Inc. (:) Contrasting side by side - FinanceMercury
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.
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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.
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NeuBase Therapeutics Inc. (NBSE)'s Financial Results Comparing With BioTime Inc. (:) - FinanceMercury
Leukaemia: what is it, how to spot the warning signs and who is at risk? – The Telegraph
By daniellenierenberg
How isleukaemia treated?
The treatment of leukaemia varies depending on the patient and type of leukaemia they have.
Acute leukaemia (fast developing) is usually curable with standard treatments, such as chemotherapy.
Chronic leukaemia (slow developing), is often incurablebut treatable. For CLL (a form of chronic leukaemia) some patients are not given treatmentstraight away;however if they do require treatment it will often involve chemotherapy.
The main treatments for leukaemia are:
Chemotherapy: This treatment involves theuse ofdrugs.Chemotherapy drugs either kill cancerous cells or stop them from dividing; they can also kill normal blood cells as a side effect.The type of leukaemia you have will depend on the amount and strength of chemotherapy you are offered, along with other factors such as your age and fitness.
Radiation therapy:Similar to chemotherapy, radiation therapy can be used to destroy the cancerous cells but using radiation waves rather than drugs.Again, the type of leukaemia you have will determine what treatment you're offered. External beam radiation therapy (EBRT) is often used for CLL.It is a fast, painless procedure which usually lasts just a few minutes.
Targeted therapy:Drugs are used to block the growth of cancer cells by disturbing specific molecules in the cells. Targeted therapy can also kill cancer cells by stimulating the patient's immune system to recognise the cells as a threat and consequently kill them.
Biological therapy:This treatment does not target the cancer cells directly, but instead helps to stimulate the body's immune system to act against the cancer. It is also often referred to as "immunotherapy". It is often usedfor patients with CML.
Stem cell or bone marrow transplant: Transplants for stem cells or bone marrow are commonly carried out for patients withacute leukaemia,if chemotherapy does not prove effective.By undergoing a stem cell or bone marrow transplant it can help replenish the healthy bone marrow in patients, and stimulate new growth that restores the immune system. It is usually given to younger, or more healthy patients.
Leukaemia Care, which provides support to individuals and families affected by blood cancer, is one ofthree charities supported by this years Telegraph Christmas Charity Appeal. Our two other charities are Wooden Spoon, which works with Britains rugby community to raise money for sick, disabled and disadvantaged children; and The Silver Line, a 24-hour helpline and support service for lonely elderly people. To make a donation, visit telegraph.co.uk/charity or call 0151 284 1927
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Leukaemia: what is it, how to spot the warning signs and who is at risk? - The Telegraph
Neural Stem Cell Transplantation Crawls Toward the Clinic – The Scientist
By daniellenierenberg
Pelizaeus-Merzbacher disease is a genetic malady that leaves neurons without their myelin coating. This deficit has devastating consequences for the boysits X-linkedwho have it. These children have severe developmental delay, so they have inability to walk, inability to talk and perform self-care, says Nalin Gupta, a professor of neurological surgery and pediatrics at the University of California, San Francisco (UCSF). Their neurologic function typically does not improve, and usually they actually die during childhood.
About a decade ago, the biotech firm StemCells Inc. was looking for a neurosurgeon to try out an intervention that might finally offer some help for these children. Because Gupta had experience conducting surgical clinical trials in kids with disabilities, the company approached him to see if he could transplant neural stem cells into the brains of boys with Pelizaeus-Merzbacher disease (PMD)an approach that researchers had considered promising for a range of conditions, but which had yet to be proven effective in a clinical trial for any disease. He agreed.
In 2012, Gupta and colleagues reported that four boys with PMD who had received pluripotent neural stem cells in a Phase 1 clinical trial tolerated the procedure, and imaging techniques that indirectly detect myelin indicated they may have had myelination in their brains one year following the transplant. This August, the researchers reported the results of a long-term follow-up study of those patientsall four are still alive at ages 10, 11, 12, and 13. Patients like these who have symptoms of the disease starting at birth typically die in their teens.
Although the researchers could not directly examine myelinationthat would require autopsiesthe imaging evidence is promising. There were some clinical improvements, too, although with such a small number of patients and no control group in a trial designed to examine safety, its hard to know whether they are attributable to the transplant.
We dont actually have a product that we can use even if we wanted to do a Phase 2 study in this disease.
Nalin Gupta, UCSF
Guptas study is the latest report in a series of clinical trials on neural stem cell transplantation, in which pluripotent neural cells taken, in most cases, from the brains of aborted fetuses are expanded in the lab and then injected into the brains or spinal cords of patients with incurable neurological disorders. These include stroke, multiple sclerosis, ALS, spinal injury, and Parkinsons disease. But for all the effort that has gone in to testing these cells, none have been able to work themselves out of trials and into clinical practice.
When asked which of the human clinical trials have been most successful, Steven Goldman, a professor of neurology and neuroscience at the University of Rochester, replies, So far, none of them, right? To date, no Phase 2 trial to evaluate the efficacy of a neural stem cell treatment has been completed, he points out. And scientists, Gupta included, are less-than-ecstatic about the methods and outcomes of the clinical trials that have been done so far.
Goldman, who was not involved in the PMD experiment, calls it by far the most rigorous and well controlled. But that trial can go no further.
Gupta says he and his colleagues felt that there was sufficient evidence from the Phase 1 trial to justify a Phase 2. They cant proceed, however, because StemCells, the company that funded the research and provided the cells, closed in 2016. We dont actually have a product that we can use even if we wanted to do a Phase 2 study in this disease, he says.
Research on other potential uses for neural stem cells are also affected by a lack of momentum. Theres somewhat of a pause in what people are doing in terms of stem cell therapeutics, says Gupta. Treatments for conditions such as spinal cord injury and stroke hold the most interest for their potential societal benefit, he says, but the complexity of the changes that occur when the brain or spinal cord are injuredmeaning regions composed of multiple cell types and networks of connections are just wiped outmake for a challenging repair. Were probably a long way from being able to transplant a structure that will recapitulate the three-dimensional organization and structure of the brain and spinal cord, he says. Trials for diseases with more specific defects might be more successful, he adds, such as multiple sclerosis, which like PMD involves demyelination.
According to Evan Snyder, the director of the Center for Stem Cells and Regenerative Medicine at the Sanford Burnam Prebys Medical Discovery Institute in La Jolla, California, there havent been enough trials, and certainly not enough under ideal circumstances, to know whether neural stem cell transplantation can be an effective treatment in humans. I think the field is too young to know right now if theyre effective. I think the field can just say that theyre safe, says Snyder. To be able to know whether the cells are effective, you really need to be able to put them into an optimal setting where their mechanism of action is optimal, and that kind of trial has never been done yet, he adds.
The animal research that laid the foundation for the PMD study, and other studies on diseases involving the loss or absence of myelination, took place in a mouse model called shiverer. These animals have a mutation that prevents their oligodendrocytes from making myelin, such that their neurons are badly insulated and cannot efficiently conduct electrical signals. The shiverer mice have problems with motor functions and self-care along with seizures. They also have a tremor, hence their name.
In 1999, Snyders lab reported in PNAS that injecting mouse neural stem cells into the brains of shiverer mice led to the remyelination of neurons as well as some tremor reductionmeasured by dipping each mouses tail in ink and noting the size of the stain it left on a piece of graph paper.
Using the model, Goldmans team later transplanted human glial progenitor cellswhich are derived from neural stem cellsinto shiverer mice, generating chimeras in which mouse neurons became insulated with human myelin. The chimeric mice, as Goldman reported in Cell Stem Cell in 2008, survived longer and had improved neurological phenotypes, including fewer seizures, compared with untreated controls. In 2012, StemCells Inc., in collaboration with researchers at Oregon Health & Science University and elsewhere, reported in Science Translational Medicine that transplanting shiverer mice with human neural stem cells resulted in remyelination in the brain. Also in that issue, Gupta and StemCells described the one-year results from the PMD trial, which used the same cells for transplantation.
Neurons (green) and glia (red) differentiated from human neural stem cells in culture. Nuclei are stained blue.
Evan Snyder
The PMD study was not the first trial launched by StemCells Inc. In 2006, the company launched a Phase 1 trial of neural stem cell transplantation in children with Batten disease, a fatal condition in which children are missing a lysosomal storage enzyme. That study was the first study authorized by the FDA for transplantation with neural stem cells into the brain, says Stephen Huhn, a biotech consultant and the former chief medical officer of the company.
The trial, which was completed in 2009, revealed the treatment to be safe, the authors reported in Journal of Neurosurgery: Pediatrics. Autopies on the brains of several kids who died of the disease during the study suggested that in some patients donor cells had both survived and migrated away from the subcortical and ventricular injection sites and into the basal ganglia, among other locations, Huhn says.
The fact that we saw even glimmers of an effect was for us very promising that cellular therapy could well have a place in the treatment of some neurological disorders.
Stephen Huhn, formerly of StemCells Inc.
The stem cells used for this and other StemCells trials were isolated from the brain of a single aborted fetus, expanded as balls of cells called neurospheres, and frozen for later use. Before injection into patients, the cells were thawed, cultured for two weeks, and dissociated, so that what was injected was no longer a neurosphere but a cluster of cells, according to Huhn. Because the neural stem cells were donor-derived, patients were given immunosuppressant drugs for several months following the transplant to prevent rejection.
Using the same procedure and stock of cells, Gupta and colleagues transplanted neural stem cells into the brains of the four boys with PMD in a Phase 1 trial that began in 2009 and ran through 2012the same trial whose long-term follow-up results came out this summer. One year after transplantation, diffusion tensor imagingan MRI-based technique that lets researchers indirectly observe myelinated axonsof the boys brains suggested that myelination had occurred.
From 2012 to 2015, the company ran a Phase 1/2 trial of neural stem cell transplantation for age-related macular degeneration. The treatment proved safe, and there was also evidence of a treatment effecta slowing of the retinal damage called geographic atrophy and improvements in visual functionin some patients, says Huhn.
At the same time, the firm was engaged in a Phase 1/2 trial of stem cell transplantation for patients with injuries to the thoracic region of the spine. The treatment proved safe, and Huhn notes that several participants seemed to have sensory improvement below the level of injury, which would imply that the stem cells were having a treatment effect.
But the companys run of auspicious results did not last forever: Its Phase 2 trial of neural stem cells to treat cervical spinal cord injury, which began in 2014, terminated two years later after an independent review of the emerging data found that the study was unlikely to show a statistically significant treatment effect, Huhn says. For that same reason, a follow-up study on the same patients also ended in 2016, he adds.
At that point, StemCells Inc. shut down. STAT reported that the reason was disappointing results from the spinal cord study.
Despite stopping, the companys work was not in vain, says Huhn, as it demonstrated that the approach is safe and might be worth pursuing. These are challenging disorders, Huhn says, adding that the fact that we saw even glimmers of an effect was for us very promising that cellular therapy could well have a place in the treatment of some neurological disorders.
According to Snyder, who was not involved in the work, the PMD trial suffered from the limitations of the clinical trials system. The unfortunate thing is the way clinical trials are designed, you only get a patient who has failed every other intervention, is very deep into the disease, and almost has no chance of anything changing the course, he says. This problem is not unique to the PMD trial but applies to all neural stem cell clinical trials to date, Snyder says. In the Batten disease trial, for instance, the patients had little hope of recovery, Snyder notes. Three of the six participants had died of their disease by the time the researchers stopped collecting data. Where stem cells are going to be most useful, ultimately, is going to be the early stages of a disease where there are regions that can be rescued, and where the cells are placed in a position where they can distribute themselves throughout the region that needs to be fixed. And no clinical trial has ever met those [criteria].
Although the follow-up PMD study revealed some myelination, there was not a lot of it, notes Goldman, who was not involved in the work. Theres some evidence for local remyelination around the region of the transplants, but there was nothing that was dispersed or broad, and these patients need really widespread remyelination, he says.
Goldman says he believes that there was not more widespread and robust myelination in the PMD patients because of the cell type used. While neural stem cells can give rise to oligodendrocytes, astrocytes, and neurons, they not very efficient at making oligodendrocytes, he says. And, he adds, they do not migrate much, which is necessary for them to have widespread effects. In contrast, human glial progenitor cells, which are produced from neural stem cells and give rise to both oligodendrocytes and astrocytes, are more migratory, says Goldman, and for this reason, the field has shifted away from neural stem cells and toward glial progenitor cells for transplantation. Goldman has trials of his own in the works using a neural stem cell derivative to treat multiple sclerosis and PMD through a company he cofounded, Oscine Therapeutics.
Other trials are currently underway. Researchers at Emory University and the University of Michigan, with funding from the company Neuralstem, have completed a Phase 1 study of neural stem cells to treat ALS and, according to ClinicalTrials.gov, a Phase 2 clinical trial is ongoing. Theres a Phase 2/3 trial of nasally delivered neural stem cells to treat Parkinsons disease enrolling in China. And theres an active Phase 1 trial for Parkinsons disease in Australia using human parthenogenetic neural stem cells derived from unfertilized eggs, rather than fetal tissue.
This year, Snyder received a California Institute for Regenerative Medicine (CIRM) grant to do work leading up to cell-based therapies for babies who are at risk for developing cerebral palsy due to perinatal asphyxia, or oxygen and blood deprivation in the womb, he says. Within the first few days of life, the researchers plan to do brain imaging to identify babies with regions of the brain where cells are injured but not dead, he says, then transplant neural stem cells. The injurys still very fresh and cells are sort of teetering on a knife edge. They can either go on to die or they can go on to live, and the [transplanted] stem cells make factors that push them in the direction to live, Snyder says. If that happens, the prediction is the babies will do much better.
Theres only a short window, when cells are damaged but not dead, during which a neural stem cell transplant can work, he adds. Other trials in older patients with more advanced disease, he suggests, may have missed their optimal treatment windows. Snyder predicts that if the right patients are transplanted with the right neural stem cells at the right time, I think then, under those circumstances, now youre going to start seeing not just safety but real efficacy.
Ashley P. Taylor is a New Yorkbased freelance reporter. Follow her on Twitter@crenshawseedsand read her work atashleyptaylor.com.
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Neural Stem Cell Transplantation Crawls Toward the Clinic - The Scientist
Huntington’s Marked by Inflammation and Changes in Brain’s Striatum Before Symptoms, Study Finds – Huntington’s Disease News
By daniellenierenberg
Massive inflammation and other changes in the striatum, an area of the brain selectively destroyed in Huntingtons disease (HD), are already present before patients develop any symptoms, a study has found.
These findings could help understand how the disease unravels and why this brain region is particularly sensitive to degeneration in individuals with Huntingtons.
The study, The caudate nucleus undergoes dramatic and unique transcriptional changes in human prodromal Huntingtons disease brain, was published in the journal BMC Medical Genomics.
Huntingtons is a neurodegenerative disease caused by mutations in the huntingtin(HTT) gene and marked by trouble in controlling movement, a progressive loss of thinking ability, and psychiatric problems.
Symptoms, which typically begin when people reach their 30s and 40s, stem from a selective degeneration of certain brain regions, particularly two areas: the basal ganglia, a region deep in the brain thats responsible for functions including movement coordination; and the cortex, the outer and highly twisted layer of the brain which controls thought, behavior, and memory.
Within the basal ganglia, HD targets nerve cells (neurons) of the striatum, especially in two areas known as the caudate nuclei and putamen. These regions can shrink and suffer massive damage as a result of disease progression.
Very little is known about the active disease processes leading to such debilitating symptoms. Obtaining post-mortem brain samples from people with disease-causing mutations who have not yet developed symptoms in other words, still have a largely intact striatum is very rare.
Researchers atBoston University School of Medicine (BUSM) had the opportunity to analyze samples from the striatum more precisely, the caudate nucleus of two HD-positive individuals who had no symptoms at the time of their death.
To pinpoint early drivers of disease, the team compared the activity (expression) of genes those turned on and turned off in the caudate nucleus of these asymptomatic individuals to the prefrontal cortex of 26 symptomatic Huntingtons patients and 56 healthy controls.
Researchers used a high throughput sequencing technology called RNA-Seq to determine gene expression profiles, and a preformed bioinformatics analysis to understand which genes and biological processes were altered.
Our data suggest that the striatum experiences massive inflammation in HD even before symptoms appear, and exhibits a similar gene expression pattern to that observed in prefrontal cortex. Patterns unique to the striatum are also observed, Adam Labadorf, PhD, director of BUs Bioinformatics Nexusand the studys senior author,said in a news release.
In addition to extensive inflammatory processes, the data also suggested that over the diseases course, the striatum undergoes some form of neurogenesis, or the generation of new nerve cells.
While these are only trends that warrant further investigation, researchers propose that active production of neurons could be happening in the striatum during the prodromal phase (before symptom onset) to compensate for the nerve cell loss that precedes symptoms.
The idea that active neurogenesis occurs in the adult brain is controversial, but could lead to exciting discoveries into the innate regenerative capabilities of the central nervous system, Labadorf said.
According to researchers, these findings provide clear evidence that the caudate nucleus is strongly affected in people positive for Huntington disease, before the emergence of any symptoms.
This study presents the most detailed analysis to date of the active disease process in the primarily affected brain region of HD, and although these results do not directly suggest any novel therapies, a better understanding of these processes is likely to lead to them, Labadorfadded.
An important observation was that some genes, like HSPA6, were perturbed across all HD patient samples relative to healthy brains. This set of genes may provide an opportunity to develop prognostic tests for disease progression, the researchers noted.
A robust clinical test measuring disease progression will likely take the form of a panel of key inflammatory and possibly developmental genes measured in the blood or cerebrospinal fluid (the liquid surrounding the brain and spinal cord), they wrote.
Ana is a molecular biologist enthusiastic about innovation and communication. In her role as a science writer she wishes to bring the advances in medical science and technology closer to the public, particularly to those most in need of them. Ana holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she focused her research on molecular biology, epigenetics and infectious diseases.
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Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.
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Huntington's Marked by Inflammation and Changes in Brain's Striatum Before Symptoms, Study Finds - Huntington's Disease News
The global nerve repair and regeneration market size is expected to reach USD 17.8 billion by 2026 registering a CAGR of 10.7% – Yahoo Finance
By daniellenierenberg
Nerve Repair And Regeneration Market Size, Share & Trends Analysis Report By Surgery (Nerve Grafting, Neurorrhaphy), By Product (Biomaterials Neurostimulation & Neuromodulation Device), And Segment Forecasts, 2019 - 2026
New York, Oct. 24, 2019 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Nerve Repair And Regeneration Market Size, Share & Trends Analysis Report By Surgery, By Product And Segment Forecasts, 2019 - 2026" - https://www.reportlinker.com/p05807210/?utm_source=GNW
The global nerve repair and regeneration market size is expected to reach USD 17.8 billion by 2026 registering a CAGR of 10.7%. Demand for neurological disorder therapies owing to increasing incidence and rising awareness about the same will drive the market. Moreover, government funding and reimbursement policies and uninterrupted technological advances are also projected to help boost the market growth.
In January 2016, the EU Horizon 2020 program funded a research project Autostem, launched by the NUI Galways Regenerative Medicine Institute (REMEDI), costing about USD 6.73 million. This project was to develop a robotic stem cell production factory, having an edge over the old traditional techniques. This technique offers prospects of new therapies for a range of diseases, such as cancers, diabetes, and arthritis. Increased R&D and investments by key companies in emerging countries are also driving the market growth. In July 2018, the Stem Cells Australia (SCA) received USD 3 million for stem cell research from the Medical Research Future Fund (MRFF).
In addition, government and private funded organizations are conducting clinical trials to develop a safe and effective therapy for different neurological disorders, such as Stem Cells in Umbilical Blood Infusion for Cerebral Palsy (Phase II) and usage of Polyethylene glycol (PEG) drug (Phase I) to promote axonal fusion technique to repair peripheral nerve injuries in humans.
Furthermore, in October 2017, Stryker Corporation acquired VEXIM, a France-based medical device company.VEXIMs portfolio is complementary to Strykers Interventional Spine (IVS) portfolio.
With this acquisition, Stryker will strengthen its distribution channels in Eastern Europe, Middle East, Asia, and Latin America. In January 2018, Boston Scientific Corporation received U.S. FDA approval for the first and only Spectra WaveWriter spinal cord stimulator system. This system is used for paresthesia-based therapy.
Further key findings from the study suggest: In 2018, neuromodulation and neurostimulation devices segment led the market due to increased cases of Central Nervous System (CNS) disorders and awareness about mental disorders and available treatments Biomaterials is anticipated to expand at the fastest CAGR during the forecast period due to technological advancements and development of biodegradable polymers that can help enhance spinal stabilization, healing of fractures, and reduce hospitalization North America led the market in 2018 owing to technological advancements and advent of new devices. Government initiatives and funding and increased cases of injured CNS, such as injuries to the spinal cord and brain, were some of the major reasons responsible for the regions growth Asia Pacific is expected to be the fastest-growing market during the forecast period. Growing geriatric population, technological advancements, and many unmet medical needs are some of the factors driving the regions growth In February 2016, Indian scientists working for Revita Life Sciences were approved to conduct clinical trials in 20 clinically dead patients to bring specific parts of their CNS back to life Combination of therapies including cocktail of peptides, nerve stimulation techniques, injecting the brain with stem cells and other techniques that were successful in bringing patients out of coma were to be used Existing medical devices were combined with regenerative biological medicines with an objective to achieve such a complex initiative Some of the key companies include Boston Scientific, Inc.; Stryker Corporation; St. Jude Medical, Inc.; Medtronic plc.; Baxter International, Inc.; AxoGen, Inc.; Polyganics B.V.; Integra; Cyberonics, Inc.; and Lifesciences CorporationRead the full report: https://www.reportlinker.com/p05807210/?utm_source=GNW
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Speaking Multiple Sclerosis: A Glossary of Common Terms – Everyday Health
By daniellenierenberg
Whether youve been recently diagnosed with multiple sclerosis (MS) or have been living with the condition for a while, chances are youll sometimes hear terms from your healthcare team that are new to you.
The following is a quick, alphabetical guide to the terminology you may need to know as you manage your condition:
Ankle-Foot Orthosis (AFO) A brace designed to support the position of the foot and motion of the ankle to compensate for nerve damage and muscle weakness in the area caused by MS and other movement disorders. An AFO is typically used to stabilize weak limbs or to reposition a limb with contracted muscles into a more normal position.
Autoimmune Disease Your immune system plays a major part of your bodys defense against bacteria and viruses by sending out cells to attack them once they enter your body. However, if you have an autoimmune disease, your immune system mistakenly attacks healthy cells in your body, causing them to weaken or break down. MS is thought to be just one example of an autoimmune disease. It has been suggested that in MS, your immune system may mistakenly attack the cells in your central nervous system.
Axon Long threadlike structures of nerve cells that send impulses to other cells in your body. Research suggests that damage to or loss of these fibers in progressive MS may be linked to worsening disability and more severe progression.
Central Nervous System (CNS) The group of organs in your body that includes the brain, spinal cord, and optic nerves. If you have MS, your bodys immune system may be working against the CNS, producing neurological symptoms such as muscle weakness and vision problems.
Cerebrospinal Fluid (CSF) A clear, colorless liquid that surrounds the brain and spinal cord to protect the CNS and assist in the circulation of nutrients and removal of waste products. In MS, damage to the myelin sheath of nerve cells causes certain types of proteins to be released into the spinal fluid. The presence of these proteins in the CSF, but not in the blood, may point to a diagnosis of MS.
Clinically Isolated Syndrome (CIS) A first episode of neurologic symptoms that lasts at least 24 hours and is caused by inflammation or demyelination (loss of the myelin that covers the nerve cells) in the CNS. People who experience CIS may or may not go on to develop MS. However, when CIS is accompanied by magnetic resonance imaging (MRI)detected brain lesions similar to those found in MS, you have a 60 to 80 percent chance of a second neurologic event and diagnosis of MS within several years, according to the National MS Society.
Cog Fog A commonly used term that refers to the cognitive changes experienced by many people with MS. According to MS Australia, approximately 50 percent of people with the condition will develop some degree of cog fog, or inhibited ability to think, reason, concentrate, or remember. For some, cognitive problems will become severe enough to interfere in a significant way with daily activities.
Corticosteroids (or Steroids) Prescription medication used to treat relapses in relapsing-remitting MS. Your doctor may prescribe intravenous (IV) corticosteroids if the symptoms of your relapse are causing significant problems, like poor vision or difficulty walking. These drugs work by suppressing the immune system and reducing inflammation in the CNS, and they may help relapse symptoms resolve more quickly. But they wont affect your ultimate level of recovery from a relapse or the long-term course of your MS. Methylprednisolone is a commonly used corticosteroid in MS.
Diplopia (or Double Vision) An eye problem in which you see two images of a single object. It may be present when only one eye is open (monocular) or disappear when either eye is closed (binocular). Diplopia is a common symptom of MS, and it occurs because of damage to the optic nerve.
Disease-Modifying Therapies (DMTs) Drugs designed to reduce new relapses, delay progression of disability, and limit new CNS inflammation in people with MS. Although there are multiple DMTs that have been approved by the U.S. Food and Drug Administration (FDA) for use in MS, these drugs generally work by reducing inflammation in nerve cells in theCNS.
Dysarthria A speech disorder caused by neuromuscular impairment and resulting in disturbances in motor control of the muscles used in speech. Its believed the demyelinating lesions in MS may result in spasticity, weakness, slowness, or ataxic incoordination of the lips, tongue, mandible, soft palate, vocal cords, and diaphragm, causing this speech impairment.
Dysphagia (Difficulty Swallowing) A condition that may occur in people with MS, leading to difficulty in eating solid foods or liquids, frequent throat clearing during eating or drinking, a feeling that food is stuck in the throat, or coughing or a choking sensation when eating or drinking. Its the result of nerve damage within the muscles that control swallowing.
Epstein-Barr Virus (EBV) A virus believed to be a possible cause or trigger for MS. Although the exact cause of MS remains unknown, researchers suggest an infectious agent may be involved in its development. Studies have found that antibodies (immune proteins that indicate a person has been exposed to a given virus) to EBV are significantly higher in people who eventually develop MS than in those who dont. Other research has noted that people with a specific immune-related gene and high levels of antibodies to EBV in their blood are 9 times more likely to develop MS than others.
Evoked Potentials A test that measures the speed of nerve messages along sensory nerves to the brain, which can be detected on your scalp using electrodes attached with sticky pads. Its sometimes used in the diagnosis of MS, because nerve damage can slow down the transmission of nerve signals. Evoked potential tests can indicate nerve pathways that are damaged prior to the onset of MS symptoms.
Exacerbation An occurrence of new symptoms or the worsening of old symptoms that may also be referred to as a relapse, attack, or flare-up. Exacerbations can be very mild, or severe enough to interfere with a person's ability to perform day-to-day activities.
Expanded Disability Status Scale (EDSS) A scale used for measuring MS disability and monitoring changes in the level of disability over time. Developed by neurologist John Kurtzke, MD, in 1983, the EDSS scale ranges from 0 to 10 in 0.5-unit increments (scoring is based on a neurological exam) and relies on walking as its main measure of disability. People with an EDSS of 1 have no disability and minimal loss of function, while those with an EDSS of 9.5 are confined to bed and totally dependent on others for functions of daily living.
Foot Drop (or Drop Foot) A symptom of MS caused by weakness in the ankle or disruption in the nerve pathway between the legs and the brain, making it difficult to lift the front of the foot to the correct angle during walking. If you have foot drop, your foot hangs down and may catch or drag along the ground, resulting in trips and falls. Foot drop can be managed with an AFO or other treatments.
Hematopoietic Stem Cell Transplantation (HSCT) A procedure designed to reboot the immune system, the National MS Society says, using hematopoietic (blood cellproducing) stem cells derived from a persons own bone marrow or blood. If your doctor recommends HSCT, youll undergo a chemotherapy regimen before these cells are reintroduced to the body via IV injection, where they will migrate to your bone marrow to rebuild the immune system.
John Cunningham (JC) Virus A common infection completely unrelated to MS that is found in as many as 90 percent of people, according to the UK's MS Trust. JC virus has no symptoms and is normally controlled by the immune system. However, if your immune system is weakened, the JC virus can reactivate, causing potentially fatal inflammation and damage to the brain known as progressive multifocal leukoencephalopathy (PML). Certain MS disease-modifying therapies have been linked with increased risk for PML.
Lhermittes Sign An electric shock-like sensation experienced by some with MS when the neck is moved in a particular way. The sensation can travel down to the spine, arms, and legs.
Lesion (or Plaque) Refers to an area of damage or scarring (sclerosis) in the CNS caused by inflammation in MS. These lesions can be spotted on an MRI scan, with active lesions appearing as white patches. With regular MRIs, a neurologist can tell how active your MS is.
Lumbar Puncture (or Spinal Tap) A procedure used for the collection of cerebrospinal fluid (CSF), sometimes done to help diagnose MS. For this procedure, your doctor will ask you to lie on your side or bend forward while seated, before cleansing an area of your lower back and injecting a local anesthetic. He will then insert a hollow needle and extract a small amount of spinal fluid using a syringe.
Magnetic Resonance Imaging (MRI) The diagnostic tool that currently offers the most sensitive noninvasive way of imaging the brain, spinal cord, or other areas of the body, according to the National MS Society. Its the preferred imaging method for diagnosis of MS and to monitor the course of the disease. MRI uses magnetic fields and radio waves to measure the relative water content in tissues, which is notable in MS because the layer of myelin that protects nerve cell fibers is fatty and repels water. In areas where myelin has been damaged by MS, fat is stripped away and the tissue holds more water. This shows up on an MRI as a bright white spot or darkened area, depending on how the images are made.
McDonald Criteria A guidance used in the diagnosis of MS, authored by an international panel of experts on the condition, originally in 2010. The guidance was updated in 2017. Among the key changes: advising for the use of brain MRI as part of the diagnostic process.
MS Hug A common symptom of MS. If you experience the MS hug, you may feel like you have a tight band around your chest or ribs, or pressure on one side of your torso. Some people find that it is painful to breathe. The MS hug can last for seconds, minutes, hours, or even longer.
Myelin A substance rich in lipids (fatty substances) and proteins that helps form the myelin sheath. In MS, particularly relapsing-remitting MS, an abnormal immune response produces inflammation in the CNS, effectively attacking the myelin in the cells.
Myelin Sheath An insulating layer of fatty substances and proteins that forms around the nerves in body, including those in the CNS. It allows electrical impulses to transmit quickly and efficiently along the nerve cells, but these impulses can be slowed if the sheath is damaged, causing MS.
Neurodegeneration Refers to the process by which the myelin sheath of cells in the CNS is damaged in MS. Its believed to be a major contributor to neurological disability in the condition, and may be the reason immune modulation treatments (disease-modifying therapy) are generally less effective in the progressive MS than in the relapsing-remitting MS.
Neurologist The point person for monitoring your MS treatment and managing MS symptoms. This specialist typically focuses on conditions affecting the CNS.
Neuropathic Pain A type of pain common in MS that results from changes or damage to the myelin sheath and the axons, or nerve fibers, it normally covers. MS-caused neuropathic pain may be chronic, intermittent, or occur only in response to a stimulus.
Neuropsychologist A specialist you may be referred to who helps you manage the cognitive effects of MS. Neuropsychological testing (or testing of the functioning of your brain) involves identifying memory or learning difficulties associated with MS. Cognitive rehabilitation may improve functioning.
Nociceptive Pain Caused by damage to muscles and joints, it can be either acute or chronic, and may not result from MS itself, but be caused by changes in posture or walking or the overuse of assistive devices in those with the condition.
Nystagmus A common eye abnormality in MS, its characterized by involuntary, rhythmic, back-and-forth motion of the eyeball, either horizontally or vertically. For those with nystagmus, the perception of the rhythmic movement of the surrounding stationary world (oscillopsia) can be disorienting and disabling.
Oligoclonal Bands (OCBs) Immunoglobulins, or proteins, that collect in blood plasma or cerebrospinal fluid (CSF). Although not every person with MS has OCBs, their presence can support a diagnosis of MS. Having OCBs is generally associated with a younger age of MS onset and a poorer prognosis.
Optic Neuritis An inflammatory condition that damages the optic nerve, a bundle of nerve fibers that transmits visual information from your eye to your brain, causing pain and temporary vision loss in one eye. Its been linked with nerve damage resulting from MS, and may be among the first symptoms a person with the condition experiences.
Pseudobulbar Affect (PBA) A neurologic effect experienced by roughly 10 percent of people with MS as well as some with Parkinsons disease or amyotrophic lateral sclerosis (ALS), according to the Multiple Sclerosis Association of America (MSAA). Its characterized by sudden, uncontrollable expressions of laughter or crying without an obvious cause, which can be distressing as well as embarrassing to those who experience it. PBA is believed to be a mood disorder related to the disruption of nerve impulses in the CNS, but its different from depression, which is also common in MS.
Pseudoexacerbation A temporary worsening of symptoms without actual myelin inflammation or damage. It is often triggered by other illnesses or infection, exercise, a warm environment, depression, exhaustion, and stress. Urinary tract infection (UTI) is the most common type of infection to cause a pseudoexacerbation.
Sclerosis A general hardening of the body tissue. The term multiple sclerosis refers to the multiple areas of scar tissue often called lesions that develop along affected nerve fibers and that are visible in MRI scans.
Spasticity A symptom of MS that causes your muscles to feel stiff, heavy, or difficult to move. When a muscle spasms, youll experience a sudden stiffening that may cause a limb to jerk. This may be painful.
Trigeminal Neuralgia (or Tic Douloureux) A type of neuropathic pain that occurs on the face (usually on one side only). Its a known symptom of MS, and you may experience it in your cheek; upper or lower jaw; inside the mouth; or in the area around your eyes, ears, or forehead. In MS, its typically caused by damage to the myelin sheath around the trigeminal nerve, which among other functions controls the muscles used in chewing. The condition is triggered by everyday activities, like tensing facial muscles while shaving or when chewing.
Vertigo An intense sensation of the surrounding environment spinning around one. In MS, vertigo is typically caused by growth of an existing lesion or development of a new lesion on the brain stem or cerebellum, the area in the brain that controls balance. It can also be a symptom of a problem with the inner ear, or it can be side effect of medication used to treat MS or other health conditions you may have.
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Speaking Multiple Sclerosis: A Glossary of Common Terms - Everyday Health
Stem Cell Assay Market Analysis On Trends & Need 2025 – Health News Office
By daniellenierenberg
Stem Cell Assay Market: Snapshot
Stem cell assay refers to the procedure of measuring the potency of antineoplastic drugs, on the basis of their capability of retarding the growth of human tumor cells. The assay consists of qualitative or quantitative analysis or testing of affected tissues and tumors, wherein their toxicity, impurity, and other aspects are studied.
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With the growing number of successful stem cell therapy treatment cases, the global market for stem cell assays will gain substantial momentum. A number of research and development projects are lending a hand to the growth of the market. For instance, the University of Washingtons Institute for Stem Cell and Regenerative Medicine (ISCRM) has attempted to manipulate stem cells to heal eye, kidney, and heart injuries. A number of diseases such as Alzheimers, spinal cord injury, Parkinsons, diabetes, stroke, retinal disease, cancer, rheumatoid arthritis, and neurological diseases can be successfully treated via stem cell therapy. Therefore, stem cell assays will exhibit growing demand.
Another key development in the stem cell assay market is the development of innovative stem cell therapies. In April 2017, for instance, the first participant in an innovative clinical trial at the University of Wisconsin School of Medicine and Public Health was successfully treated with stem cell therapy. CardiAMP, the investigational therapy, has been designed to direct a large dose of the patients own bone-marrow cells to the point of cardiac injury, stimulating the natural healing response of the body.
Newer areas of application in medicine are being explored constantly. Consequently, stem cell assays are likely to play a key role in the formulation of treatments of a number of diseases.
Global Stem Cell Assay Market: Overview
The increasing investment in research and development of novel therapeutics owing to the rising incidence of chronic diseases has led to immense growth in the global stem cell assay market. In the next couple of years, the market is expected to spawn into a multi-billion dollar industry as healthcare sector and governments around the world increase their research spending.
The report analyzes the prevalent opportunities for the markets growth and those that companies should capitalize in the near future to strengthen their position in the market. It presents insights into the growth drivers and lists down the major restraints. Additionally, the report gauges the effect of Porters five forces on the overall stem cell assay market.
Global Stem Cell Assay Market: Key Market Segments
For the purpose of the study, the report segments the global stem cell assay market based on various parameters. For instance, in terms of assay type, the market can be segmented into isolation and purification, viability, cell identification, differentiation, proliferation, apoptosis, and function. By kit, the market can be bifurcated into human embryonic stem cell kits and adult stem cell kits. Based on instruments, flow cytometer, cell imaging systems, automated cell counter, and micro electrode arrays could be the key market segments.
In terms of application, the market can be segmented into drug discovery and development, clinical research, and regenerative medicine and therapy. The growth witnessed across the aforementioned application segments will be influenced by the increasing incidence of chronic ailments which will translate into the rising demand for regenerative medicines. Finally, based on end users, research institutes and industry research constitute the key market segments.
The report includes a detailed assessment of the various factors influencing the markets expansion across its key segments. The ones holding the most lucrative prospects are analyzed, and the factors restraining its trajectory across key segments are also discussed at length.
Global Stem Cell Assay Market: Regional Analysis
Regionally, the market is expected to witness heightened demand in the developed countries across Europe and North America. The increasing incidence of chronic ailments and the subsequently expanding patient population are the chief drivers of the stem cell assay market in North America. Besides this, the market is also expected to witness lucrative opportunities in Asia Pacific and Rest of the World.
Global Stem Cell Assay Market: Vendor Landscape
A major inclusion in the report is the detailed assessment of the markets vendor landscape. For the purpose of the study the report therefore profiles some of the leading players having influence on the overall market dynamics. It also conducts SWOT analysis to study the strengths and weaknesses of the companies profiled and identify threats and opportunities that these enterprises are forecast to witness over the course of the reports forecast period.
Some of the most prominent enterprises operating in the global stem cell assay market are Bio-Rad Laboratories, Inc (U.S.), Thermo Fisher Scientific Inc. (U.S.), GE Healthcare (U.K.), Hemogenix Inc. (U.S.), Promega Corporation (U.S.), Bio-Techne Corporation (U.S.), Merck KGaA (Germany), STEMCELL Technologies Inc. (CA), Cell Biolabs, Inc. (U.S.), and Cellular Dynamics International, Inc. (U.S.).
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Stem Cell Assay Market Analysis On Trends & Need 2025 - Health News Office
In a first, 26-year-old DMD patient in UP survives with stem cell therapy – India TV News
By daniellenierenberg
Image Source : PTI
Children, suffering from DMD, usually die of cardio-respiratory failure. Represtational image
Duchenne Muscular Dystrophy (DMD) is a deadly genetic disorder, 99.9 per cent people suffering from which, die between the age of 13 to 23 years. However, in a first, a 26-year-old patient from Lucknow has survived DMD by regularly taking stem cells for the last five years.
Children, suffering from DMD, usually die of cardio-respiratory failure. But with the stem cell therapy, this patient has not lost muscle power in last five years and heart and lung muscles and the upper half of the body are working well.
Dr. B.S Rajput, the surgeon who is treating this patient, said, "DMD is a type of muscular dystrophy and being a genetic disorder, it is very difficult to treat. Autologous (from your own body) bone marrow cell transplant or stem cell therapy in such cases was started in Mumbai about 10 years back.
Dr Rajput, who was recently appointed as visiting professor at GSVM Medical College, Kanpur, said he has treated several hundred DMD patients and recently this combination protocol was published in the international Journal of Embryology and stem cell research.
The patient's father is elated that his son has maintained well with this treatment and now has even started earning by working on computers.
According to Dr Rajput, this disease is endemic in eastern UP, especially Azamgarh, Jaunpur, Ballia and some of the adjoining districts of Bihar, and one out of every 3,500 male child, suffers from the disease.
Yet the disease is not given as much attention as it should be.
Dr Rajput, who is consultant bone cancer and stem cell transplant surgeon from Mumbai, said though patients in Uttar Pradesh and Bihar get financial support from the Chief Minister's Relief Funds, the treatment of autologous bone marrow cell transplant is not included in the package list of Ayushman Bharat scheme, which deprives many from getting the treatment.
The doctor further informed that efforts are being made to establish the department of regenerative medicine in the medical college, where bone marrow cell transplant and stem cell therapy would be done even for other intractable problems like spinal cord injury, arthritis knee and motor neurone disease.
ALSO READ |Fasting triggers regeneration of stem cells capacity: Study
ALSO READ |UK patient 'free' of HIV after stem cell treatment
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In a first, 26-year-old DMD patient in UP survives with stem cell therapy - India TV News
In a first, 26-yr-old DMD patient in UP survives with stem cell therapy – The Woke Journal
By daniellenierenberg
Reading Time: 2 minutesLucknow:
Duchenne Muscular Dystrophy (DMD) is a deadly genetic disorder, 99.9 per cent people suffering from which, die between the age of 13 to 23 years. However, in a first, a 26-year-old patient from Lucknow has survived DMD by regularly taking stem cells for the last five years.
Children, suffering from DMD, usually die of cardio-respiratory failure. But with the stem cell therapy, this patient has not lost muscle power in the last five years and heart and lung muscles and the upper half of the body are working well.
Dr B.S Rajput, the surgeon who is treating this patient, said, DMD is a type of muscular dystrophy and being a genetic disorder, it is very difficult to treat. Autologous (from your own body) bone marrow cell transplant or stem cell therapy in such cases was started in Mumbai about 10 years back.
Dr Rajput, who was recently appointed as visiting professor at GSVM Medical College, Kanpur, said he has treated several hundred DMD patients and recently this combination protocol was published in the International Journal of Embryology and stem cell research.
The patients father is elated that his son has maintained well with this treatment and now has even started earning by working on computers.
According to Dr Rajput, this disease is endemic in eastern UP, especially Azamgarh, Jaunpur, Ballia and some of the adjoining districts of Bihar, and one out of every 3,500 male child, suffers from the disease.
Yet the disease is not given as much attention as it should be.
Dr Rajput, who is consultant bone cancer and stem cell transplant surgeon from Mumbai, said though patients in Uttar Pradesh and Bihar get financial support from the Chief Ministers Relief Funds, the treatment of autologous bone marrow cell transplant is not included in the package list of Ayushman Bharat scheme, which deprives many from getting the treatment.
The doctor further informed that efforts are being made to establish the department of regenerative medicine in the medical college, where bone marrow cell transplant and stem cell therapy would be done even for other intractable problems like spinal cord injury, arthritis knee and motor neurone disease.
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In a first, 26-yr-old DMD patient in UP survives with stem cell therapy - The Woke Journal
$-0.05 EPS Expected for Lineage Cell Therapeutics, Inc. (LCTX) – The Lamp News
By daniellenierenberg
Analysts expect Lineage Cell Therapeutics, Inc. (NYSEAMERICAN:LCTX) to report $-0.05 EPS on November, 14.They anticipate $0.02 EPS change or 28.57 % from last quarters $-0.07 EPS. After having $-0.06 EPS previously, Lineage Cell Therapeutics, Inc.s analysts see -16.67 % EPS growth. The stock decreased 0.81% or $0.0079 during the last trading session, reaching $0.9699. About shares traded. Lineage Cell Therapeutics, Inc. (NYSEAMERICAN:LCTX) has 0.00% since October 22, 2018 and is . It has by 0.00% the S&P500.
Lineage Cell Therapeutics, Inc., a clinical-stage biotechnology company, focuses on developing and commercializing therapies for the treatment of degenerative diseases in the United States and internationally. The company has market cap of $145.14 million. The company's lead product candidates include OpRegen, a retinal pigment epithelium cell replacement therapy, which is in Phase I/IIa multicenter trial for the treatment of the dry age-related macular degeneration; OPC1, an oligodendrocyte progenitor cell therapy that is in Phase I/IIa multicenter clinical trial for the treatment of acute spinal cord injuries; and VAC2, an allogeneic cancer immunotherapy of antigen-presenting dendritic cells, which is in Phase I clinical trial to treat non-small cell lung cancer. It has a 4.18 P/E ratio. It also develops Renevia, a facial aesthetics product that is in pivotal clinical trial for the treatment of HIV-associated facial lipoatrophy; ReGlyde, a HyStem product in preclinical development as a device for viscosupplementation, and as a platform for intraarticular drug delivery in osteoarthritis; and Premvia, a Hystem Hydrogel product, as well as develop bone grafting products for the orthopedic diseases and injuries.
More notable recent Lineage Cell Therapeutics, Inc. (NYSEAMERICAN:LCTX) news were published by: Businesswire.com which released: Lineage Cell Therapeutics Conducts Sale of Shares in OncoCyte Corporation Business Wire on September 11, 2019, also Businesswire.com with their article: Lineage Cell Therapeutics Announces Issuance of U.S. Patent for Method of Treating Spinal Cord Injury With Stem Cell-Derived Oligodendrocyte Progenitor Cells Business Wire published on September 04, 2019, Finance.Yahoo.com published: Lineage Cell Therapeutics to Present at 2019 Cantor Global Healthcare Conference on October 4, 2019 Yahoo Finance on September 26, 2019. More interesting news about Lineage Cell Therapeutics, Inc. (NYSEAMERICAN:LCTX) were released by: Businesswire.com and their article: Lineage Cell Therapeutics to Present at H.C. Wainwright 21st Annual Global Investment Conference on September 9, 2019 Business Wire published on September 05, 2019 as well as Finance.Yahoo.coms news article titled: Lineage Cell Therapeutics to Present at 2019 Cell & Gene Meeting on the Mesa on October 3, 2019 Yahoo Finance with publication date: September 23, 2019.
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$-0.05 EPS Expected for Lineage Cell Therapeutics, Inc. (LCTX) - The Lamp News
In a first, 26-yr-old DMD patient in UP survives with stem cell therapy – Outlook India
By daniellenierenberg
In a first, 26-yr-old DMD patient in UP survives with stem cell therapy
Lucknow, Oct 22 (IANS) Duchenne Muscular Dystrophy (DMD) is a deadly genetic disorder, 99.9 per cent people suffering from which, die between the age of 13 to 23 years. However, in a first, a 26-year-old patient from Lucknow has survived DMD by regularly taking stem cells for the last five years.
Children, suffering from DMD, usually die of cardio-respiratory failure. But with the stem cell therapy, this patient has not lost muscle power in last five years and heart and lung muscles and the upper half of the body are working well.
Dr. B.S Rajput, the surgeon who is treating this patient, said, "DMD is a type of muscular dystrophy and being a genetic disorder, it is very difficult to treat. Autologous (from your own body) bone marrow cell transplant or stem cell therapy in such cases was started in Mumbai about 10 years back.
Dr Rajput, who was recently appointed as visiting professor at GSVM Medical College, Kanpur, said he has treated several hundred DMD patients and recently this combination protocol was published in the international Journal of Embryology and stem cell research.
The patient''s father is elated that his son has maintained well with this treatment and now has even started earning by working on computers.
According to Dr Rajput, this disease is endemic in eastern UP, especially Azamgarh, Jaunpur, Ballia and some of the adjoining districts of Bihar, and one out of every 3,500 male child, suffers from the disease.
Yet the disease is not given as much attention as it should be.
Dr Rajput, who is consultant bone cancer and stem cell transplant surgeon from Mumbai, said though patients in Uttar Pradesh and Bihar get financial support from the Chief Minister''s Relief Funds, the treatment of autologous bone marrow cell transplant is not included in the package list of Ayushman Bharat scheme, which deprives many from getting the treatment.
The doctor further informed that efforts are being made to establish the department of regenerative medicine in the medical college, where bone marrow cell transplant and stem cell therapy would be done even for other intractable problems like spinal cord injury, arthritis knee and motor neurone disease.
--IANS
amita/rtp
Disclaimer :- This story has not been edited by Outlook staff and is auto-generated from news agency feeds. Source: IANS
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In a first, 26-yr-old DMD patient in UP survives with stem cell therapy - Outlook India
Stem cells treatment gives hope in fighting Autism, blood disorders – OrissaPOST
By daniellenierenberg
Bhubaneswar: The advanced treatment of using stem cells for treating Autism and other neurological ailments have come as a ray of hope for the people living with some of these ailments. Medical experts working in the sector claim that the use of the technology improved the lives of many.
According to experts who practice stem cell therapy, the results have been overwhelming. Many of the patients have either been able to fight a deadly disease with the help of stem cells while many have been able to improve their quality of lives by using it. However, the technology is still not used widely in state hospitals.
Medical experts claim that stem cells could be used to treat neurological disorders like Autism, cerebral palsy, mental retardation, brain stroke, muscular dystrophy, spinal cord injury, head injury, cerebellar ataxia, dementia, motor neurone disease, multiple sclerosis while it has also been used to treat cancers like blood cancer with the help of bone marrow transplant when assisted by stem cell therapy.
However, treatment of Autism with stem cells is a new developing sector where visible changes are said to have been reported among children treated with this technology. However, the advanced technology which is now confined to only private sector is a bit expensive.
Autistic kids are usually treated with drugs for symptomatic relief, special education, occupational speech and behavioural therapies. In Autism, despite the best available medical and rehabilitative treatments satisfactory relief is still a far cry, said Dr Nandini Gokulchandran, Head Medical Services, NeuroGen Brain and Spine Institute, Mumbai.
Dr Gokulchandran claims that she has treated many cases of Autism in kids with stem cells which helped in overcoming their limited abilities. Under the treatment regime, an insertion procedure is undertaken followed by training to improve the skills and abilities of autistic kids.
Another neurologist, Dr Richa Bansod said that in India it has been reported that 1 in every 250 children have Autism and this number in increasing with better recognition and awareness of the condition. On the other hand, stem cells are now been used to fight deadly diseases.
Dr Joydeep Chakaborty, an oncologist and stem cell expert from HCG Cancer Hospital, Kolkata said, Stem cells and bone marrow transplants are now being used to cure blood cancer in many cases. It is also widely used to treat blood disorders like Thalassemia, Sickle Cell Anaemia and others.
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Stem cells treatment gives hope in fighting Autism, blood disorders - OrissaPOST
Cell Therapy Market outlook with industry review and forecasts – Technology Magazine
By daniellenierenberg
Cell therapy market size is estimated to grow significantly during the forecast period from 2019 to 2025. The cell therapy industry should witness substantial expansion over the forecast timeframe due to technological advancements coupled with increasing utilization of cell therapy along with gene therapy as treatment to several conditions.
Increasing funding from private organization as well as government to encourage cell therapy clinical trials, inclusion of varied cell types such as mesenchymal stem cells, skeletal muscle stem cells, lymphocytes, dendritic cells, hematopoietic stem cells (HSC) and pancreatic islet cells for cell therapy research will drive market size growth.
Bone marrow transplant or hematopoietic stem cell transplantation is most commonly performed cell therapy. Growing utilization of this therapy for treating conditions such as blood cancer and other hematologic conditions should drive industry growth. Potential application of cell therapy includes treatment for urinary problems, cancers, repairing spinal cord injuries, autoimmune disease, rebuilding damaged cartilage in joints, immune system improvement, neurological disorders and infectious disease. Such wide-ranging applications of cell therapy should drive business growth over the coming years.
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Increasing prevalence of chronic conditions, government assistance and numerous companies investing heavily in stem cell therapy research and development should stimulate industry growth. Proven effectiveness of cell therapy products coupled with rendering favorable guidelines pertaining to cell therapy manufacturing should positively impact industry growth. However, high cost and stringent regulations related to practicing of stem cell therapy will hamper industry growth to certain extent during the forecast period.
Based on therapy type, the industry is segmented into allogenic and autologous therapies. Autologous segment is anticipated to witness significant growth due to lower risk of fatal complications and graft failure resulting in high survival rate should drive segment growth. Moreover, significant investment in autologous cell therapy research by companies such as Vericel Corporation should positively impact growth.
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Applications of cell therapy include cardiovascular, ocular, musculoskeletal, gastrointestinal, oncology, neurological, dermatology, wounds and injuries, and others. Oncology segment is estimated to witness lucrative growth due to strong pipeline including Chimeric Antigen Receptor (CAR) T-cell therapy that has delivered promising results such as full recovery in end-stage patients suffering from acute lymphocytic leukemia during clinical trials.
Based on end-user, the industry is bifurcated into hospitals, diagnostic centers, clinics, research institutes, regenerative medicine centers and others. Stem cell is core component of regenerative medicine. Increasing research conducted by regenerative medicine researchers on variety of stem cell types such as multipotent adult stem cells (hematopoietic stem cells found in umbilical cord blood and mesenchymal stem cells [MSC] found in adipose tissue), along with pluripotent stem cells such as the bioengineered cells called induced pluripotent stem cells (iPSC) should drive segment growth.
North America cell therapy market will witness robust growth during the forecast period due to favorable regulatory framework to promote development of cellular therapy platform and presence of numerous companies engaged in cell therapy research. Rising awareness and growing healthcare expenditure should further propel industry growth.
Large patient population base suffering from chronic conditions along with development of healthcare infrastructure will drive Asia Pacific cell therapy market. Growing inclination towards advanced medicinal therapies will lead to growth of cell therapy market in the region.
Some notable industry players include, Vericel Corporation, Medipost, Cells for Cells, JCR Pharmaceuticals, Osiris Therapeutics, Kolon Tissuegene, NuVasive, Stemedica Cell Technologies, BioNTech IMFS, Anterogen, Pharmicell, Fibrocell Science, Novartis AG, Glaxosmithkline among others. Industry players are investing heavily in cell therapy research to develop innovative product and gain significant market share of lucrative market in future.
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Cell Therapy Market outlook with industry review and forecasts - Technology Magazine
Reviewing Aldeyra Therapeutics Inc. (ALDX)’s and Neuralstem Inc. (NASDAQ:CUR)’s results – MS Wkly
By daniellenierenberg
Aldeyra Therapeutics Inc. (NASDAQ:ALDX) and Neuralstem Inc. (NASDAQ:CUR) compete against each other in the Biotechnology sector. We will compare them and contrast their profitability, institutional ownership, analyst recommendations, risk, dividends, earnings and valuation.
Valuation and Earnings
Table 1 demonstrates Aldeyra Therapeutics Inc. and Neuralstem Inc.s top-line revenue, earnings per share and valuation.
Profitability
Table 2 hightlights the net margins, return on assets and return on equity of the two companies.
Risk and Volatility
Aldeyra Therapeutics Inc.s 0.5 beta indicates that its volatility is 50.00% less volatile than that of S&P 500. Neuralstem Inc.s 94.00% more volatile than S&P 500 which is a result of the 1.94 beta.
Liquidity
The Current Ratio and a Quick Ratio of Aldeyra Therapeutics Inc. are 9.2 and 9.2. Competitively, Neuralstem Inc. has 3.8 and 3.8 for Current and Quick Ratio. Aldeyra Therapeutics Inc.s better ability to pay short and long-term obligations than Neuralstem Inc.
Institutional and Insider Ownership
Institutional investors held 69.9% of Aldeyra Therapeutics Inc. shares and 4.9% of Neuralstem Inc. shares. Aldeyra Therapeutics Inc.s share held by insiders are 2.2%. Insiders Competitively, held 1% of Neuralstem Inc. shares.
Performance
Here are the Weekly, Monthly, Quarterly, Half Yearly, Yearly and YTD Performance of both pretenders.
For the past year Aldeyra Therapeutics Inc. was less bearish than Neuralstem Inc.
Summary
On 9 of the 9 factors Aldeyra Therapeutics Inc. beats Neuralstem Inc.
Aldeyra Therapeutics, Inc., a biotechnology company, focuses on the development of products for inflammation, inborn errors of metabolism, and other diseases in the United States and internationally. It is developing ADX-102, a small molecule designed to trap and allow for the degradation of aldehydes, as well as ADX-103 and ADX-104 novel candidates for the treatment of noninfectious anterior uveitis, allergic conjunctivitis, dry eye syndrome, sjgren-larsson syndrome, and succinic semi-aldehyde dehydrogenase deficiency. The company was formerly known as Aldexa Therapeutics, Inc. and changed its name to Aldeyra Therapeutics, Inc. in March 2014. Aldeyra Therapeutics, Inc. was founded in 2004 and is headquartered in Lexington, Massachusetts.
Neuralstem, Inc., a clinical stage biopharmaceutical company, focuses on the research and development of nervous system therapies based on its proprietary human neuronal stem cells and small molecule compounds. The companys stem cell based technology enables the isolation and expansion of human neural stem cells from various areas of the developing human brain and spinal cord enabling the generation of physiologically relevant human neurons of various types. It is developing products include NSI-189, a chemical entity, which is in Phase II clinical trial for the treatment of major depressive disorder, as well as is in preclinical programs for the MCAO stroke, type 1 and 2 diabetes related neuropathy, irradiation-induced cognition, long-term potentiation enhancement, and angelman syndrome. The company is also developing NSI-566, which has completed Phase II clinical trial for treating amyotrophic lateral sclerosis disease, as well as is in Phase I clinical trials for the treatment of chronic spinal cord injury and motor deficits due to ischemic stroke. Neuralstem, Inc. was founded in 1996 and is headquartered in Germantown, Maryland.
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Reviewing Aldeyra Therapeutics Inc. (ALDX)'s and Neuralstem Inc. (NASDAQ:CUR)'s results - MS Wkly
Comparing of Axcella Health Inc. (AXLA) and Neuralstem Inc. (NASDAQ:CUR) – MS Wkly
By daniellenierenberg
Axcella Health Inc. (NASDAQ:AXLA) and Neuralstem Inc. (NASDAQ:CUR) compete against each other in the Biotechnology sector. We will compare them and contrast their analyst recommendations, institutional ownership, profitability, risk, dividends, earnings and valuation.
Valuation & Earnings
Table 1 shows gross revenue, earnings per share and valuation of the two companies.
Profitability
Table 2 provides the net margins, return on equity and return on assets of the two firms.
Analyst Recommendations
In next table is delivered Axcella Health Inc. and Neuralstem Inc.s ratings and recommendations.
Axcella Health Inc.s average price target is $22.5, while its potential upside is 330.21%.
Institutional and Insider Ownership
Roughly 0% of Axcella Health Inc. shares are owned by institutional investors while 4.9% of Neuralstem Inc. are owned by institutional investors. Comparatively, insiders own roughly 1% of Neuralstem Inc.s shares.
Performance
Here are the Weekly, Monthly, Quarterly, Half Yearly, Yearly and YTD Performance of both pretenders.
For the past year Axcella Health Inc. has stronger performance than Neuralstem Inc.
Summary
Axcella Health Inc. beats on 8 of the 10 factors Neuralstem Inc.
Neuralstem, Inc., a clinical stage biopharmaceutical company, focuses on the research and development of nervous system therapies based on its proprietary human neuronal stem cells and small molecule compounds. The companys stem cell based technology enables the isolation and expansion of human neural stem cells from various areas of the developing human brain and spinal cord enabling the generation of physiologically relevant human neurons of various types. It is developing products include NSI-189, a chemical entity, which is in Phase II clinical trial for the treatment of major depressive disorder, as well as is in preclinical programs for the MCAO stroke, type 1 and 2 diabetes related neuropathy, irradiation-induced cognition, long-term potentiation enhancement, and angelman syndrome. The company is also developing NSI-566, which has completed Phase II clinical trial for treating amyotrophic lateral sclerosis disease, as well as is in Phase I clinical trials for the treatment of chronic spinal cord injury and motor deficits due to ischemic stroke. Neuralstem, Inc. was founded in 1996 and is headquartered in Germantown, Maryland.
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Comparing of Axcella Health Inc. (AXLA) and Neuralstem Inc. (NASDAQ:CUR) - MS Wkly