Study: Brain cells of people with autism differ even before theyre born – Study Finds
By daniellenierenberg
PHILADELPHIA Autism is a neurodevelopmental disorder which creates difficulties with social interactions, communication, and repetitive behaviors. According to the Centers for Disease Control and Prevention, autism affects one in 54 children in the United States. Although autism typically isnt diagnosed until children are at least 18 months-old, a new study finds that abnormal brain cell development likely occurs much earlier.
Researchers say this even occurs while babies are still in the womb.
To study developing brain cells, a team from Kings College London and Cambridge University use a type of cell known as an induced pluripotent stem cell (or iPSC). Essentially, iPSCs are adult cells that scientists force to become immature embryonic-like stem cells. Scientists can program iPSCs to become one of many different cell types, including neurons. Since iPSCs are forced to restart their cellular development, they mimic the processes occurring in the womb. This allows them to serve as a useful means of studying early brain development.
Using iPSCs from hair samples is the most ethical way to study early brain development in autistic people, explains study author Dwaipayan Adhya in a media release. It bypasses the need for animal research, it is non-invasive, and it simply requires a single hair or skin sample from a person.
Adhya is a molecular biologist at the Autism Research Centre in Cambridge and Department of Basic and Clinical Neuroscience at Kings College London.
To create the iPSCs, the study analyzes hair samples from nine adults with autism and six neurotypical adults. The hair cells were then treated with growth factors (naturally occurring bodily substances that regulate cell division and survival). The authors looked at the cells appearance and genetic makeup at different phases of development.
The scientists results reveal iPSCs from neurotypical people look different from those participants with autism. At day nine, neurons from neurotypical people develop a characteristic pattern of neural rosettes, which have a dandelion-like shape. In contrast, cells from people with autism have smaller rosettes or dont form them at all. Cells from autistic individuals also express lower levels of important developmental genes.
The use of iPSCs allows us to examine more precisely the differences in cell fates and gene pathways that occur in neural cells from autistic and typical individuals, co-author Deepak Srivastava explains. These findings will hopefully contribute to our understanding of why there is such diversity in brain development.
The brain has been the ultimate black box. Here, the authors have used nerve cells derived from peripheral stem cells to peek inside this box. This important study suggests that this is possible and is deepening our understanding of autism, says John Krystal, Editor-in-Chief of the journal Biological Psychiatry.
The study is published in the June 22 edition of Biological Psychiatry.
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Study: Brain cells of people with autism differ even before theyre born - Study Finds
Im Optimistic That We Will Have a COVID-19 Vaccine Soon – The Atlantic
By daniellenierenberg
Read: The plan that could give us our lives back
The science is paying off. Novavax, a Maryland-based company working on this type of vaccine, recently reported the results of its Phase 1 trial. The levels of antibodies generated were stunning, about four times higher than those in individuals who are recovering from a COVID-19 infection.
Scientists are also using different strains of another virus, adenovirus, as a vector or a missile to deliver genes that code for these same spike proteins and that also provoke an immune response. The vector has been engineered in the lab to be replication-defective; that is, the vector is able to deliver the spike gene into humans but once its done its job, the vector cannot replicate any further. At least three groups are testing these vectors. A University of Oxford group, in partnership with AstraZeneca, has employed an adenovirus from chimpanzees and has already entered Phase 3 trials in humans. The Beth Israel Deaconess Medical Center group, in partnership with Janssen Pharmaceutica, is using Ad26, a human adenovirus, and the Chinese-based CanSino Biologics has begun Phase 3 trials with yet another human adenovirus, Ad5.
These examples are not just beautiful science (although they are beautiful science). By harnessing the increased power of the biological sciences, researchers are developing entirely new ways of rapidly developing vaccines.
My optimism doesnt stop with these early results, although they are key. Im also encouraged because at least five very different approaches (Ive walked through only three above) are being explored to make a vaccine. As we say in Canada, if you want to win, you have to take many shots on goal.
Equally important is the unprecedented global collaboration among scientists around the world, as well as the high degree of cooperation between scientists and clinicians, biopharmaceutical companies, government, philanthropic funders, and regulators. They are all working together toward the common goal of developing as quickly as possible a safe and effective vaccine against COVID-19.
I dont know which of the vaccine candidates undergoing clinical testing in humans will ultimately be shown to be safe and effective. They might all prove effective, albeit in different age groups or in people with different preexisting conditions. But the encouraging news is that all of the vaccine candidates that have entered trials in humans so far are safe and have elicited high levels of antibodies against COVID-19. Some have also been shown to activate the cellular arm of our immune system, another crucial component of our defenses against foreign pathogens.
The public-health imperative to obtain a safe and effective vaccine as quickly as possible goes hand in hand with the mandate that the approval process be above any political considerations and solely based on data from the clinical trials. Anything else risks losing the publics confidence in a vaccine or, in a worst-case scenario, might result in a vaccine that is less effective than those that might be approved later, or the widespread administration of a vaccine that turns out to have serious adverse side effects. That would be a public-health tragedy.
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Im Optimistic That We Will Have a COVID-19 Vaccine Soon - The Atlantic
What I learnt from checking in to the ‘Immunity Hotel’ – Telegraph.co.uk
By daniellenierenberg
There isnt a third person in our marriage but there is a rival. My husband is passionate about a family-run fasting clinic in Uberlingen, overlooking Lake Constance, Germany. He goes alone twice a year and was there at the beginning of lockdown. He happily remained there for six weeks unable to get home. Buchinger Wilhelmi was founded seventy years ago by Otto Buchinger, a medical officer in the navy who cured himself of paralysis caused by rheumatic fever by fasting for 19 days in 1918. Now one of the worlds leading therapeutic fasting centres, Buchinger is run by Ottos great-grandson, Leonard Wilhelmi.
The German government demanded that the clinic remain open during lockdown, concerned that hospital over-crowding would necessitate patients being moved there. This proved unnecessary. But after Easter, having taken the requisite health and hygiene precautions, they encouraged guests. They are currently full with a completely different clientele, according to owner, Raimund Wilhelmi (Ottos grandson.) Normally, two-thirds of our guests are repeat guests. Now, fifty percent have come for the first time and they are much younger. There is a significant difference in the demographic because people are waking up to a new awareness about health as Covid affects everybody. His son, Leonard, adds: The medical community agree that the main causes in violent reactions to Covid are diabetes, high blood pressure and being over-weight. We have been treating these conditions for decades and our goal is now to equip people with a better immune system to fight Covid.
The minute my husband heard about their new immune-boosting programme, he signed us up for atwo weeks' holiday in August. This was an extravagance at a cost of over two thousand pounds a person. Health is our most valuable commodity, he reassured me. After an hour in this sleek minimalist medical centre, my sixteen-year-old daughter burst into tears. All her boarding school issues were ignited. My inner rebel similarly baulked at the first 24 hours that we had to spend eating in our rooms, until the results of our Covid tests, taken on arrival, came through. (Thankfully they were negative or we would have been quarantined in our rooms for our entire stay.)
There was something convict-like about dining on trays in our monastically simple rooms. Thank goodness the clinic do not advise couples share rooms 80 per centof guests go alone so we each had our own room. As Andrew was doing the full ten-day fast of 250 liquid calories a day, which I couldnt stomach(I was on 800 solid calories a day and Daisy, 1800,) a shared room would have destroyed our marriage. Not because Andrew had an enema on the bed every other day but because his preternatural joy freaked us out. His pious enthusiasm for the regime initially made us hate it. Until we were fully institutionalised, (or as Daisy said indoctrinated) the stricture wore us out.
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What I learnt from checking in to the 'Immunity Hotel' - Telegraph.co.uk
First Woman May Be Cured of HIV Without a Bone Marrow Transplant – POZ
By daniellenierenberg
A California woman may be the first person to be cured of HIV without a bone marrow transplant, according to a recent report in Nature. More than 60 other so-called elite controllers, who have unusually potent immune responses to HIV, were found to have their virus sequestered in parts of their genome where it is unable to replicate.
The unusual case involves Loreen Willenberg, who acquired HIV in 1992. Her immune system has maintained control of the virus for decades without the use of antiretroviral treatment, and researchers have been unable to find any intact virus in more than 1.5 billion of her cells. Elite controllers are thought to make up less than half a percent of all people living with HIV.
I believe Loreen might indeed meet anyones definition of a cure, study coauthor Steven Deeks, MD, of the University of California at San Francisco, told POZ. Despite heroic efforts, we just could not find any virus that is able to replicate. Her immune system seems completely normal. Even her HIV antibodies levels are low, which is unprecedented in an untreated person.
Although antiretroviral therapy can keep HIV replication suppressed, the virus inserts its genetic material into the chromosomes of human cells, making it very difficult to eradicate. HIV can lie dormant in a reservoir of resting immune cells indefinitely, but when antiretrovirals are stopped and the cells become activated, they can start churning out new virus.
Previously, only two people were known to have been cured of HIV: Timothy Ray Brown, formerly know as the Berlin Patient, and a man in London. Both received bone marrow stem cell transplants from a donor with a rare genetic mutation that makes cells resistant to HIV entry. But this procedure is far too dangerous for people who dont need it to treat advanced cancer.
The new research suggests that Willenberg and some five dozen other people with long-term untreated HIV have their virus hidden away in their cells genomes in such as way that the viral genetic blueprint (known as a provirus) cant be used to produce new viral particles that can go on to infect other cells.
Xu Yu, MD, of the Ragon Institute of Massachusetts General Hospital, MIT and Harvard analyzed integrated HIV in millions of cells from 64 elite controllers and 41 typical HIV-positive people on antiretroviral therapy recruited at Mass General and San Francisco General Hospital.
In both groups, about 20% were women, the average age was approximately 56, they had been living with HIV for an average of 17 years and had undetectable virus according to standard tests for nine years. Overall, the elite controllers had a higher average CD4 count (about 900 versus 70, respectively).
The researchers used next-generation gene sequencing to characterize the participants viral blueprints, including where they were inserted into human chromosomes. They found that the elite controllers had fewer integrated proviruses, but a larger proportion of them were intact, or potentially capable of replicating. The virus in these individuals was highly consistent, without the wide variety of mutations seen in most people with HIV.
Whats more, their proviruses were integrated at distinct sites in the human genome, farther away from elements that enable viral replication. Specifically, the integrated DNA was not located near sites that switch on transcription or close to accessible chromatin, which contains histone proteins that package long DNA strands into a more compact form. The DNA must then be unwound from these proteins before it can be used to produce new virus.
These data suggest that a distinct configuration of the proviral reservoir represents a structural correlate of natural viral control, and that the quality, rather than the quantity, of viral reservoirs can be an important distinguishing feature for a functional cure of HIV-1 infection, Yu and colleagues wrote.
In a commentary accompanying the report, Nicolas Chomont, PhD, of the University of Montreal, characterized the proviruses in these elite controllers as being in a state of deep sleep compared with latent virus in typical people with HIV. This has only become apparent now because researchers have more sophisticated tools to pinpoint the location of proviruses within the genome.
It is unclear why this block and lock phenomenon happens in only a small proportion of people with HIV. Its possible that the virus ends up sequestered in these locations by chance. But the researchers think its more likely that the integrated proviruses at these sites are evolutionarily selected over time as the ones in locations more conducive to viral replication are eliminated by the immune system.
In Willenbergs case, the research team analyzed more than 1.5billion of her peripheral blood immune cells, including samples from gut tissue, where the virus often hides. Theycould not find any intact proviruses that could be used to produce new HIV. Given her absence of intact proviruses, the researcherswere unable to determine whether she ever fit the pattern of having latent HIV locked away in inaccessible locations.
Another 11 people, dubbed exceptional controllers, only had detectableprovirusesatremote sites in the genome where it could not replicate.Since this study, the researchers have discovered a couple more elite controllers who may qualify as additional cures, according to the New York Times.
This raises the possibility that a sterilizing cure of HIVmeaning complete eradicationmay be feasible in rare instances, the study authors suggested. A similar but less complete process may be at play in the subset of about 10% of people with HIV who maintain viral suppression after stopping antiretroviral therapy but who still have detectable proviruses (known as post-treatment controllers).
This research was first presented at the International AIDS Society Conference on HIV Science last summer, where Willenberg was referred to as the San Francisco Patient. Willenberg later went public with her status, and she and Yu discussed the study findings during a webinar with HIV cure advocates last November.
I broke out in tears when saw Dr. Yus final slide, said Willenberg, who over the years has participated in more than a dozen studies. I can only hope and pray that with continued dedication we can figure out how I have dumped the virus into the DNA junkyard.
The question now is whether its possible to develop treatments that could enable the millions of typical people with progressive HIV to become elite controllers like Willenberg. Chomont suggested that immune-based therapiesincluding CAR-T cellsmight be able to shrink the viral reservoir until it consists only of deeply latent proviruses that are unable to replicate.
The key question is how did her immune system achieve this remarkable state, Deeks said. We do not know. We need to find more people who are exceptional controllers like Loreen and get to work on figuring out the mechanism.
In this video fromamfAR, the Foundation for AIDS Research,Loreen Willenbergtalks about living as an elite controller of HIV.
Click here to read the Nature article.Click here for more news about HIV cure research.
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First Woman May Be Cured of HIV Without a Bone Marrow Transplant - POZ
Autologous Stem Cell And Non Stem Cell Based Therapies Market to Witness Widespread Expansion During 2018-2026 – Scientect
By daniellenierenberg
Autologous stem cell and non-stem cell based therapiesinvolve an individuals cell to be cultured and then re-introduced to the donors body. These therapies do not use foreign organism cells and are therefore free from HLA incompatibility, disease transmission, and immune reactions.Increasing demand for the new therapies in the field of regenerative medicine is directly facilitating the growth of autologous stem cell and non-stem cell based therapies market. Furthermore, since the risk to transplantation surgeries is significantly reduced in these therapies, they are increasingly being preferred for treatment of bone marrow diseases, aplastic anemia, multiple myeloma, non-Hodgkins lymphoma, Hodgkins lymphoma, Parkinsons disease, thalassemia, and diabetes.
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Moreover, rising incidents of cancer, diabetes and cardiovascular diseases along with growing geriatric population is another factor attributed for its high growth. However, side-effects of autologous stem cell and non-stem cell based therapies such as nausea, infection, hair loss, vomiting, diarrhea, etc. are expected to affect the market to an extent. High cost is another factor that can act as challenge to autologous stem cell and non-stem cell based therapies market. In spite of this, less risk post transplantation surgeries and favorable tax reimbursement policies are anticipated to reduce the impact of these limitation during the forecast period.Autologous stem cell and non-stem cell based therapies market can be segmented on the basis of application, end-user, and region.
In terms of application, the autologous stem cell and non-stem cell based therapies market can be segmented into blood pressure (BP) monitoring devices, intracranial pressure (ICP) monitoring devices, and pulmonary pressure monitoring devices. In terms of end-user, the market can be segmented into ambulatory surgical center and hospitals. By region, the market can be segmented into North America, Europe, Asia Pacific, Middle East and Africa and South America. Amongst all, Asia Pacific is anticipated to be the most attractive market owing to favorable reimbursement policies in the region.The players operating in autologous stem cell and non-stem cell based therapies market are limited. They are consistently involved in research and development activities for product development to keep up with the growing competition, thereby aiding the growth of autologous stem cell and non-stem cell based therapies market across the world.
The major players operating in autologous stem cell and non-stem cell based therapies market are Regennex, Antria(Cro), Bioheart, Orgenesis Inc., Virxys corporation , Dendreon Corporation, Tigenix, Georgia Health Sciences University, Neostem Inc, Genesis Biopharma, Brainstorm Cell Therapeutics, Tengion Inc., Fibrocell Science Inc., Opexa Therapeutics Inc, Regeneus Ltd, and Cytori Inc., among others.
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Autologous Stem Cell And Non Stem Cell Based Therapies Market to Witness Widespread Expansion During 2018-2026 - Scientect
bluebird bio Presents New Results from Clinical Development Program of elivaldogene autotemcel (eli-cel, Lenti-D) Gene Therapy for Cerebral…
By daniellenierenberg
DetailsCategory: DNA RNA and CellsPublished on Sunday, 30 August 2020 13:01Hits: 250
Long-term results from Phase 2/3 Starbeam study (ALD-102/LTF-304) suggest durability of response post eli-cel with all 20 patients who were free of major functional disabilities (MFDs) at two years (out of 23 evaluable patients) remaining MFD-free through last available follow-up, including all 10 patients who reached at least Year 5 follow-up visit
31 out of 32 patients in ALD-102 had stable Neurologic Function Scores following treatment with eli-cel, including 24 patients with a score of zero as of the last available visit
In clinical studies of eli-cel to date, there have been no reports of graft failure, graft rejection, graft-versus-host disease (GVHD), replication competent lentivirus, or insertional oncogenesis
Company on track to submit Marketing Authorization Application in EU by year-end 2020, and Biologics License Application in U.S. in mid-2021
CAMBRIDGE, MA, USA I August 29, 2020 I bluebird bio, Inc. (Nasdaq: BLUE) announced updated results from the clinical development program for its investigational elivaldogene autotemcel (eli-cel, Lenti-D) gene therapy in patients with cerebral adrenoleukodystrophy (CALD), including long-term results from the Phase 2/3 Starbeam study (ALD-102/LTF-304) and data from the Phase 3 ALD-104 study. These data were presented today at the 46th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT 2020), taking place virtually from August 29 - September 1, 2020.
CALD is a fatal neurodegenerative disease primarily affecting young boys. Currently, the only treatment available is allogeneic hematopoietic stem cell transplantation (allo-HSCT), which comes with associated, significant risks, including transplant-related mortality, graft failure or rejection, and graft-versus-host disease (GVHD), said David Davidson, M.D., chief medical officer, bluebird bio. Eighty-seven percent of patients in our Phase 2/3 Starbeam study of eli-cel are alive and free of major functional disabilities (MFDs) at 24 months or more of follow-up. Importantly, there were no reports of graft failure, graft rejection, or GVHD. It is gratifying to see the consistent outcomes with eli-cel and the durability of the treatment effect demonstrated in the children participating in our long-term follow-up study including 10 boys who have now reached at least their Year 5 follow-up visit.
Adrenoleukodystrophy (ALD) is a rare, X-linked metabolic disorder that is estimated to affect one in 21,000 male newborns worldwide. ALD is caused by mutations in the ABCD1 gene that affect the production of adrenoleukodystrophy protein (ALDP) and subsequently cause toxic accumulation of very long-chain fatty acids (VLCFAs) primarily in the adrenal cortex and white matter of the brain and spinal cord.
Approximately 40% of boys with adrenoleukodystrophy will develop CALD, the most severe form of ALD. CALD is a progressive neurodegenerative disease that involves breakdown of myelin, the protective sheath of the nerve cells in the brain that are responsible for thinking and muscle control. Symptoms of CALD usually occur in early childhood and progress rapidly, if untreated, leading to severe loss of neurologic function, and eventual death, in most patients. CALD is associated with six MFDs, which severely compromise a patients ability to function independently: loss of communication, cortical blindness, need for tube feeding, total incontinence, wheelchair dependence, and complete loss of voluntary movement. Nearly half of boys with CALD who do not receive treatment will die within five years of symptom onset.
Patients with CALD experience a rapid decrease in neurologic function after the initial onset of clinical symptoms, so early diagnosis and treatment is critical in order to stop the disease progression and preserve their neurological function. In the Phase 2/3 Starbeam study, 31 of 32 patients had a stable neurologic function score, suggesting that disease progression had stabilized and minimal neurological function was lost, following eli-cel infusion, said Dr. Jrn-Sven Khl, Department of Pediatric Oncology, Hematology and Hemostaseology, Center for Women's and Children's Medicine, University Hospital Leipzig. These results presented at EBMT 2020 are very encouraging and suggest treatment with eli-cel may prevent neurological decline in boys with CALD.
Eli-cel is a one-time investigational gene therapy designed to address the underlying genetic cause of CALD by adding functional copies of the ABCD1 gene into a patients own hematopoietic (blood) stem cells (HSCs) that have been transduced ex vivo with the Lenti-D lentiviral vector (LVV). The addition of a functional gene allows patients to produce the ALDP, which is thought to break down the toxic accumulation of VLCFAs in the brain. There is no need for donor HSCs from another person, as is required for allo-HSCT.
Starbeam Study (ALD-102)/Long-Term Follow-Up Study (LTF-304)
The ALD-102 study has completed enrollment. All reported data below are as of January 2020 and reflect a total population of 32 patients with a median follow-up time of 30.0 months (9.1 70.7 months).
Of the 32 patients who have received eli-cel as of January 2020, 20 have completed ALD-102 and enrolled in a long-term follow-up study (LTF-304). Nine additional patients continue to be followed in ALD-102 and have not reached 24 months post-treatment. As previously reported, two patients withdrew from the study at investigator discretion, and one experienced rapid disease progression early on-study resulting in MFDs and death. To date, 104.3 patient-years of follow-up have been reported for ALD-102 and LTF-304.
The primary efficacy endpoint in the study is the proportion of patients who are alive and free of MFDs at Month 24. Of those patients who have or would have reached Month 24, 87% have met the primary endpoint and continue to be alive and MFD-free at more than two years of follow-up (N=20/23). Fourteen patients have at least four years of follow-up, including 10 patients who have reached at least their Year 5 follow-up visit. The nine patients from ALD-102 that have not reached Month 24 have shown no evidence of MFDs.
Data on several secondary and exploratory efficacy outcomes are reported, including changes in neurologic function score (NFS), a 25-point score used to evaluate the severity of gross neurologic dysfunction across 15 symptoms in six categories; resolution of gadolinium enhancement (GdE), an indicator of active inflammation in the brain; and change in Loes score, an MRI measurement of white matter changes in CALD. Of the 32 patients treated, 31 had stable NFS following treatment with eli-cel, defined as NFS <4, without a change of >3 from baseline, and 24 patients maintained an NFS of 0. An NFS of 0 indicates that there are no concerns with the neurologic functions that are assessed on the 25-point scale. Loes scores generally stabilized within 12-24 months and GdE was no longer seen in most patients following eli-cel treatment.
The primary safety endpoint is the proportion of patients who experience acute (Grade 2) or chronic GvHD by Month 24. GvHD is a condition that may occur after an allo-HSCT, where the donated cells view the recipients body as foreign and attack the body. No events of acute or chronic GvHD have been reported post-eli-cel treatment. There have been no reports of graft failure or graft rejection.
In addition, there have been no cases of replication competent lentivirus or insertional oncogenesis to date. Integration site analysis (ISA) was conducted to determine the pattern of integration post-eli-cel infusion and assess whether dominant or expanding clones were present. In one patient, now enrolled in LTF-304 for long-term follow up, a case of benign clonal expansion was observed with three separate integrations in the DNA of the cell at ACER3, RFX3, and MECOM. As of the patients Month 62 visit in March 2020, the patient remained clinically stable. Bone marrow analyses showed no dysplasia (abnormal cell growth) or molecular abnormalities.
The treatment regimen, comprising mobilization/apheresis, conditioning, and eli-cel infusion, had a safety and tolerability profile primarily reflective of the known effects of mobilization/apheresis and conditioning. In ALD-102, as previously reported, three adverse events (AE) were considered possibly related to drug product and include one serious AE (SAE), BK viral cystitis (N=1, SAE, Grade 3), and two non-serious AEs, vomiting (N=2, Grade 1). All three AEs resolved using standard measures.
ALD-104 Study
bluebird bio is currently enrolling patients for ALD-104, a Phase 3 study designed to assess the efficacy and safety of eli-cel in patients with CALD after myeloablative conditioning using busulfan and fludarabine, a different chemotherapy conditioning regimen than what is used in ALD-102 (busulfan and cyclophosphamide). The primary efficacy endpoint is the proportion of patients who are alive and free of MFDs at Month 24, and the primary safety endpoint is the proportion of patients with neutrophil engraftment after eli-cel infusion. All reported data below are as of February 2020.
In ALD-104, the 13 patients currently on study have a median of 6.1 months of follow-up to date (min-max: 2.2 10.3 months). All 13 patients achieved neutrophil engraftment and 12/13 evaluable patients had platelet engraftment (platelet engraftment pending in one patient as of data cut date). Due to the limited duration of follow-up, only safety data are being presented.
No events of acute or chronic GvHD have been reported and there have been no reports of graft failure, graft rejection, cases of insertional oncogenesis, or replication competent lentivirus.
The treatment regimen, comprising mobilization/apheresis, conditioning, and eli-cel infusion had a safety and tolerability profile primarily reflective of the known effects of mobilization/apheresis and conditioning. In ALD-104, two AEs of pancytopenia were considered possibly related to eli-cel. These two ongoing AEs were deemed as suspected unexpected serious adverse reactions (SUSARs) by the principal investigator and were diagnosed approximately two months post-eli-cel infusion in two patients (one Grade 2 and one Grade 3). An additional AE was ongoing as of February 2020, a Grade 3 SAE of transverse myelitis that was diagnosed in the presence of viral infection (adenovirus and rhinovirus/enterovirus positivity) approximately six months after eli-cel infusion and deemed unrelated to eli-cel.
eli-cel Presentation at EBMT
Lenti-D hematopoietic stem cell gene therapy stabilizes neurologic function in boys with cerebral adrenoleukodystrophy
Presenting Author: Dr. Jrn-Sven Khl, Department of Pediatric Oncology, Hematology and Hemostaseology, Center for Women's and Children's Medicine, University Hospital Leipzig Poster Session & Number: Gene Therapy; ePoster O077
Presentations will be available for virtual viewing throughout the duration of the live meeting on the EBMT 2020 website and content will be accessible online following the close of the meeting until November 1, 2020.
About elivaldogene autotemcel (eli-cel, formerly Lenti-D)
In July 2020, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) granted an accelerated assessment to eli-cel gene therapy for cerebral adrenoleukodystrophy (CALD). bluebird bio is currently on track to submit the Marketing Authorization Application (MAA) in the EU for eli-cel for CALD by year-end 2020, and the Biologics License Application (BLA) in the U.S. in mid-2021.
bluebird bio is currently enrolling patients for a Phase 3 study (ALD-104) designed to assess the efficacy and safety of eli-cel after myeloablative conditioning using busulfan and fludarabine in patients with CALD. Contact This email address is being protected from spambots. You need JavaScript enabled to view it. for more information and a list of study sites.
Additionally, bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-304) for patients who have been treated with eli-cel for CALD and completed two years of follow-up in bluebird bio-sponsored studies.
The Phase 2/3 Starbeam study (ALD-102) has completed enrollment.
For more information about bluebird bio-sponsored studies visit: http://www.bluebirdbio.com/our-science/clinical-trialsor clinicaltrials.gov.
The European Medicines Agency (EMA) accepted eli-cel gene therapy for the treatment of CALD into its Priorities Medicines scheme (PRIME) in July 2018, and previously granted Orphan Medicinal Product designation to eli-cel.
The U.S. Food and Drug Administration (FDA) granted eli-cel Orphan Drug status, Rare Pediatric Disease designation, and Breakthrough Therapy designation for the treatment of CALD.
Eli-cel is not approved for any indication in any geography.
About CALD Early Diagnosis
Early diagnosis of CALD is important, as the outcome of available treatment varies with the clinical stage of the disease. Newborn screening for ALD is a critical enabler of early diagnosis and thus of successful treatment of ALD. Once a patient has been diagnosed with ALD, regular MRI scans are critical to detect white matter changes indicative of progression to CALD.
In the U.S., newborn screening for ALD was added to the Recommended Universal Screening Panel in February 2016 and is currently active in 17 states, accounting for > 58 percent of U.S. newborns. Outside the U.S., the Minister of Health in the Netherlands has approved the addition of ALD to their newborn screening program. Even though ALD newborn screening has not been implemented in most EU countries, efforts to begin pilot programs are slowly progressing.
About bluebird bio, Inc.
bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.
bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders including cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma, using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.
bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.
SOURCE: bluebird bio
Stem Cells Market is Expected to Thrive at Impressive CAGR by 2025 – Scientect
By daniellenierenberg
This report studies the Stem Cells market size (value and volume) by players, regions, product types and end industries, history data 2013-2017 and forecast data 2018-2025; This report also studies the global market competition landscape, market drivers and trends, opportunities and challenges, risks and entry barriers, sales channels, distributors and Porters Five Forces Analysis.
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Stem cells are a class of undifferentiated cells that are able to differentiate into specialized cell types. Commonly, stem cells come from two main sources: Embryos formed during the blastocyst phase of embryological development (embryonic stem cells) and Adult tissue (adult stem cells).
Both types are generally characterized by their potency, or potential to differentiate into different cell types (such as skin, muscle, bone, etc.).
Stem Cells market, by technology, is Cell Acquisition, Cell Production, Cryopreservation, Expansion, and Sub-Culture. Stem Cell Therapy in China is not mature, so in this report we mainly cover Stem Cell Banking market.
Stem Cells market, by technology, is Cell Acquisition, Cell Production, Cryopreservation, Expansion, and Sub-Culture. Stem Cell Therapy in China is not mature, so in this report we mainly cover Stem Cell Banking market.
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Geographically, this report is segmented into several key regions, with sales, revenue, market share and growth Rate of Stem Cells in these regions, from 2013 to 2025, covering
North America (United States, Canada and Mexico)
Europe (Germany, UK, France, Italy, Russia and Turkey etc.)
Asia-Pacific (China, Japan, Korea, India, Australia, Indonesia, Thailand, Philippines, Malaysia and Vietnam)
South America (Brazil etc.)
Middle East and Africa (Egypt and GCC Countries)
The various contributors involved in the value chain of the product include manufacturers, suppliers, distributors, intermediaries, and customers. The key manufacturers in this market include
CCBC
Vcanbio
Boyalife
Beikebiotech
By the product type, the market is primarily split into
Umbilical Cord Blood Stem Cell
Embryonic Stem Cell
Adult Stem Cell
Other
By the end users/application, this report covers the following segments
Diseases Therapy
Healthcare
We can also provide the customized separate regional or country-level reports, for the following regions:
North America
United States
Canada
Mexico
Asia-Pacific
China
India
Japan
South Korea
Australia
Indonesia
Singapore
Malaysia
Philippines
Thailand
Vietnam
Rest of Asia-Pacific
Europe
Germany
France
UK
Italy
Spain
Russia
Rest of Europe
Central & South America
Brazil
Rest of Central & South America
Middle East & Africa
GCC Countries
Turkey
Egypt
South Africa
Rest of Middle East & Africa
The study objectives of this report are:
To study and analyze the global Stem Cells market size (value & volume) by company, key regions/countries, products and application, history data from 2013 to 2017, and forecast to 2025.
To understand the structure of Stem Cells market by identifying its various subsegments.
To share detailed information about the key factors influencing the growth of the market (growth potential, opportunities, drivers, industry-specific challenges and risks).
Focuses on the key global Stem Cells manufacturers, to define, describe and analyze the sales volume, value, market share, market competition landscape, SWOT analysis and development plans in next few years.
To analyze the Stem Cells with respect to individual growth trends, future prospects, and their contribution to the total market.
To project the value and volume of Stem Cells submarkets, with respect to key regions (along with their respective key countries).
To analyze competitive developments such as expansions, agreements, new product launches, and acquisitions in the market.
To strategically profile the key players and comprehensively analyze their growth strategies.
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Stem Cells Market is Expected to Thrive at Impressive CAGR by 2025 - Scientect
Here are some must-try beauty products for $25 or less (and a couple splurges) – Commercial Appeal
By daniellenierenberg
Jean Chen Smith, Correspondent for Memphis Commercial Appeal Published 6:01 a.m. CT Aug. 28, 2020 | Updated 11:26 a.m. CT Aug. 28, 2020
Lily Lolo's Lip Gloss Set offers two colors perfect for day or night.(Photo: Lily Lolo)
You dont have to spend a ton of money on beauty products that are safe, effective and easy to use.With so many options in the marketplace today, it's easy to get lost among the offerings.
Here are someof our favoritebeauty finds under $25.
Beauty Society, an American skincare and cosmetics company that specializes in affordable products, was named one of 2019s top eco-friendly skincare companies because of itsrefilling and biodegradable packaging practices. Its matte liquid lipsticks ($22) are smudge free and long lasting. The unique formula prevents color from bleeding, which makes for fewer applications.
Details: beautysociety.com
Actsyl-3 ($25)is a fast-absorbing, non-greasy, formulated serum designed to improve follicle thickness and stimulate hair stem cells. The Actsyl-3 serum contains Redensyl (3%) and Capixyl (2%) two ingredients proven to help regrow hair. Key peptides also help to strengthen hair roots and improve scalp health all without affecting hormones.
Details: actsyl.com
Trinny Londons Eye2Eye / Eyeshade ($24) is a collection of flattering shades that function as both eyeshadows and eyeliners. Rich in pigment, moisturizing and easy to use, these glide on smoothly, allowing you to smudge, smoke or line your eyes. The company also carries a full line of accessories such as The Elizabeth ($20), which is a bright yellow bag for storing your makeup essentials.Fifteen percent of the proceeds for every The Elizabeth sold go to The Princes Trust, which supports the Change A Girl's Life Campaign, an initiative of Women Supporting Women.
Details: trinnylondon.com
Wonderskin Wonder Blading Lip Gloss ($22.50) delivers draw-dropping shine that makes lips look plump without a sticky or tacky feel. The high-lacquer finish can last for hours and works great over any of the companys coordinating Peel & Reveal Lip Colors.
Details: wonderskin.com
Crabtree & Evelyn's Petal Power Lip Scrub ($18) helps to cleanse and hydrate your lips using shea butter and coconut oil.It is gentle enough to use daily and great for exfoliating the dead skin from dry, chapped lips.
Details: crabtree-evelyn.com
Facial Works Sea Mist Calming Cucumber Toning Mist ($24) is the perfect travel companion.This is a soothing and calming toner providing antioxidant benefits from CleanSea Complex and is packed with Sea Whip, an anti-inflammatory ingredient from the Caribbean Sea. This 1-ounce bottle is travel-sized, so you can bring it with you anywhere. A full-size bottle is also available.
Details: thefacialworks.com
Beautycounter Countersun Mineral Sunscreen Stick will keep you protected.(Photo: Beautycounter)
Refresh and renew with Beautycounters Citrus Mimosa Body Wash ($25), a gentle and moisturizing cleanser that energizes with a citrus scent. Certified vegan and cruelty-free, this will leave your skin feeling soft, clean and hydrated.Dont forget the sunscreen!The Countersun Mineral Sunscreen Stick SPF 30C ($21) comes in a convenient stick form with plenty of protection. Water-resistant and formulated with non-nano zinc and California poppy, the SPF 30 formula provides an effective shield from UVA and UVB rays, while moisturizing with cocoa butter, without leaving a white residue on your skin. Best of all, it is reef-friendly and water-resistant for up to 80 minutes.
Details: beautycounter.com
These Purifying Facial Towelettes From Rooted Beauty will keep you refreshed.(Photo: Rooted Beauty)
On the Grove Collaborative website, you can find affordable beauty products that adhere to the highest standards.The site prioritizes products made with sustainable, plant-based ingredients that arent tested on animals. Each brand must meet strict guidelines regarding safety, ethics, environmental impact and animal welfare. Purifying Facial Towelettes ($6.95) from Rooted Beauty will keep you refreshed throughout the day. They remove dirt, oiland makeup with free-radical-fighting white tea, calming calendula and the R7 complex, which contains antioxidant-rich roots.Throw Lily Lolos Lip Gloss Set ($25) into your bag the colors are perfect for both day and night.
Details: grove.co/roven
Sjal Skincare's Balans is a gentle pore cleanser.(Photo: Sjal Skincare)
Sjal Skincare Balans ($75) is a lightweight deep pore cleanser that hydrates, detoxifies and protects the skin. This powerhouse cleaner contains potent ingredients such as pearl extract for brightening, African whitewood extract and fig leaf extract, a natural barrier to protect the skin against water loss.
Details: sjalskincare.com/products/balans
Westmore Beauty-Skin Conditioning Exfoliator ($39) is a creamy exfoliator packed with moisturizing ingredients like shea butter and squalene. The oil-free formula contains gentle exfoliating beads to instantly smooth away dirt and dry skin, making your skin feel soft and silky. Fragrance-free and made without the use of parabens, sulfates or phthalates, this is something you will want to put on your skin.
Details:westmorebeauty.com
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Here are some must-try beauty products for $25 or less (and a couple splurges) - Commercial Appeal
Cells Reach Out and Touch, Providing Evidence of Foresight and Design – Discovery Institute
By daniellenierenberg
Photo: Cichlid fish, by Russell D. Fernald and Sabrina S. Burmeister / CC BY (https://creativecommons.org/licenses/by/2.5).
Anarticle yesterdayforEvolution Newsabout allostery showed how an individual protein or RNA can send information to its distant domains. Information sharing can also occur between chains of molecules arranged in a signaling cascade, where each one triggers action in the next. This is a bit more like the Rube Goldberg technique, except that in cells, it is much more logical and reliable. Here are new examples ofmechanosensing(the ability to sense a touch) andmechanotransduction(the ability to pass on touch information). A paper onbioRxivexplains, Cells sense the physical properties of their environment, translate them into biochemical signals and adapt their behaviour accordingly.
One such system is the MAPK/ERK pathway that all eukaryotic cells use to get information from the cell surface into the nucleus. A diagram onWikipedias page makes it clear that many individual factors take part. Once the EGFR receptor triggers ERK on the cells exterior membrane, a signaling cascade begins with at least 16 cofactors and proteins transporting the information to the cell nucleus, which responds by transcribing code proteins or enzymes. ERK signals can also spread throughout the cytoplasm, leading to a variety of responses depending on the nature of the triggering molecule.
Now, Japanese scientists have noticed a further response in neighboring cells. When one cells ERK pathway is triggered, that cell shrinks. Neighboring cells sense the change and respond by shrinking themselves, causing a chain reaction.Researchers at Kyoto Universitylikened this to how crowds do The Wave at sporting arenas, passing collective motion throughout the stadium.
Cells are tightly connected and packed together, so when one starts contracting from ERK activation, it pulls in its neighbors, elaborates [Tsuyoshi] Hirashima. This then caused surrounding cells to extend, activating their ERK, resulting in contractions thatlead to a kind of tug-of-war propagating into colony movement.[Emphasis added.]
The response involves both chemical and mechanical factors. Our work clearly shows that the ERK-mediatedmechano-chemical feedback systemgenerates complicated multicellular patterns, the lead author comments.
Another touch-sensitive mechanism is the so-called Hedgehog (Hh) pathway, so named because defects in its function cause fruit fly embryos to look like the spiny animals. Hedgehog pathways are often associated with the primary cilium, an organelle that sticks out like an antenna from the cell membrane and senses its environment. When triggered, it also causes a cascade of reactions inside the cell.
Craig Albertson, a researcher at theUniversity of Massachusetts, Amherstwas curious why cichlid fish can evolve so quickly to environmental changes, including changing the shapes and densities of bone in their jaws. This capacity for phenotypic plasticity is not evolution of a Darwinian kind, but rather a programmed response to environmental cues.
Albertson works with a system cichlid fishes known throughout the scientific world as champions of phenotypic plasticity thatcan alter, in a single season, jawbone hardness or shape to match feeding conditions.They are also well known for their rapid evolution and diversity in jaw shapes, which hasenabled cichlids to adapt to many different food sources, including algae, plankton, fish, snails and even the scales of other fishes.
Albertson speculated that this capacity for rapid response to environmental cues might be associated with the Hedgehog signaling pathway. By tuning the amount of Hh signal, his research team discovered that more bone was deposited, or vice versa.
Albertson, explains, Bone cells in these fish are innatelysensitive to differentmechanical environments. But we were able to play with this system using a single molecular switch you turn up the Hh signal and the cells become more sensitive to the environment, or you turn the molecular sensor down and the cells become almost deaf to the environment.
Like ERK, the Hedgehog signaling pathway involves numerous factors that interact in chain reactions. And it is triggered by a mechano-sensor on the cell, the primary cilium.
An important clue came as Albertson learned more about how this molecular pathway works. He explains, There isa well-known mechano-sensor on most cells, including those that make the skeleton, called the primary cilium. Cells that lack this organelle are unable to sense or respond to environmental input, includingmechanical load.It turns out that several key protein components of the Hedgehog pathway are physically associated with this structure, making it an obvious candidate for an environmentally sensitive signal.
The team believes this kind of response to environmental cues could be responsible for other kinds of rapid evolution in other animals. The Hh signal has also been shown to regulate plasticity inbeetle horns, so there may be something special that positions it to be anenvironmental sensor across tissues and animals, Albertson says. This is not Darwinism; it is pre-programmed response using molecular machines capable of sensing touch.
How does skin stretch when a body grows? The answers may rely on mechanosensitive factors.Nature News and Views said recently, Stretching the skin of mice reveals thatmechanical strain is communicated by a subpopulation of stem cellsthat proliferate and promote mechanical resistance,and so generate extra skin.
One of the most remarkable examples of touch communication was announced this month inNature. Researchers at theUniversity of Montrealconfirmed the existence of nanotubes that grow out of cell membranes and reach across comparatively large distances to touch other cells, affect their behaviors, and even share organelles with them. They found these nanotubes in the retinas of mice, and believe they are responsible for controlling blood flow in the capillaries.
For the first time, we have identifieda communication structure between cellsthat is required to coordinate blood supply in the living retina, said Dr. Adriana Di Polo, a neuroscience professor at Universit de Montral and holder of a Canada Research Chair in glaucoma and age-related neurodegeneration, who supervised the study.
We already knew that activated retinal areas receive more blood than non-activated ones, she said, butuntil now no one understood how this essential blood delivery was finely regulated.
These nanotubes tunnel through the mass of retinal cells to distant capillaries, where they contact pericytes, cells that have the ability to control the amount of blood passing through a single capillary simply by squeezing and releasing it. This touch communication allows a retinal cell to tell the capillary it needs more blood or less blood.
Using a microscopy technique to visualize vascular changes in living mice, we showed that pericytes project very thin tubes, calledinter-pericyte tunnelling nanotubes, tocommunicate with other pericytes located in distant capillaries, said Alarcon-Martinez. Through these nanotubes,the pericytes can talk to each other to deliver blood where it is most needed.
Video micrographs show that even mitochondria can be passed down these nanotubes. The paper inNaturesays:
Here we identify nanotube-like processes that connect two bona fide pericytes on separate capillary systems, forming a functional network in the mouse retina, which we named interpericyte tunnelling nanotubes (IP-TNTs). We provide evidence that these (i) have an open-ended proximal side and a closed-ended terminal (end-foot) that connects with distal pericyte processes via gap junctions, (ii)carry organelles including mitochondria, which can travelalong these processes, and (iii) serve as a conduit for intercellular Ca2+waves, thusmediating communication between pericytes.
The cells literally reach out and touch other pericytes bound to other capillaries, and hand off signals and organelles. This gives the retinal cells, dependent on a steady supply of oxygen and nutrients, a way to fine-tune their own blood supply. The gap junctions act like filters: Small particles, such as ions, can pass through this junction, but larger objects, such as organelles, cannot. Tunneling nanotubes had been noted between cells in a petri dish before, but a companion article onNature News and Viewscalls this the firstin vivoevidence for the existence of a type of TNT-like protrusion. Maybe it wont be the first for long. The research teams headline calls them, Nanotubes in the eye that help us see.
These are just some of the ways that cells respond to mechanical forces. The chains of reactions can be very elaborate and irreducibly complex. But first, they have to be triggered by well-designed mechanosensors that can feel a factor in the environment and then pass along that information to downstream processes that can do something about it. Undoubtedly many more examples of mechanosensing and mechanotransduction remain to be discovered. Its hard to conceive of any of these systems arising piecemeal by accumulated mistakes (mutations).
Instead, they appear as systems of coordinated parts that were planned to adapt to changes, providing robustness. It is exciting to ponder how such pre-programmed responses to environmental cues could trigger rapid adaptations, giving rise to some of the spectacular variations seen in birds, beetles, fish, and other organisms. Prematurely attributed to Darwinian processes, these examples of phenotypic plasticity actually show foresight and design.
Read more from the original source:
Cells Reach Out and Touch, Providing Evidence of Foresight and Design - Discovery Institute
Mike Tyson reveals all after doctors gave him blood injection that left him feeling weird during stem cell t – The Irish Sun
By daniellenierenberg
MIKE TYSON has revealed he was injected with nearly-translucent blood in his bid to make a comeback... and the former heavyweight champ said it made him feel "weird".
The 54-year-old - who initially retired from boxing in 2005 - will fight Roy Jones Jr in November in his eagerly-anticipated comeback fight.
2
His return to action has been aided by stem-cell research therapy, which has left him feeling like a "different person".
In May, Tyson claimed: "You know what I had done? I had stem-cell research therapy.
"I feel like a different person but I can't comprehend why I feel this way.
"It's really wild what scientists can do."
Stem-cell therapy is the use of stem cells to treat or prevent a disease or condition that usually takes the form of a bone marrow transplantation.
Tyson opened up on the effects the treatment has had on him in an recent interview with rapper LL Cool J on the Rock the Bells Radio show on SiriusXM, earlier in 2020.
Commenting on the mental aspect of training for a fight for the first time in 15 years, he said: "My mind wouldnt belong to me.
"My mind would belong to somebody that disliked me enough to break my soul, and I would give them my mind for that period of time.
"Six weeks of this and Id be in the best shape Ive ever dreamed of being in. As a matter of fact, Im going through that process right now. And you know what else I did, I did stem-cell research."
Tyson was then asked whether that meant if his white blood had been spun and then put back in, to which he replied: "Yes. As they took the blood it was red and when it came back it was almost transfluid (sic).
"I could almost see through the blood, and then they injected it in me.
"And Ive been weird ever since, Ive got to get balanced now."
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FUR SUREDubois tips Fury to beat Joshua having sparred with BOTH heavyweight champs
WHAT IS STEM CELL TREATMENT USED FOR?
Stem cell transplants are carried out when bone marrow is damaged or isnt able to produce healthy blood cells.
It can also be used to replace damaged blood cells as the result of intensive cancer treatment.
Here are conditions that stem cell transplants can be used to treat:
Iron Mike had been called out by former rival Evander Holyfield to complete their trilogy following their two meetings in 1990s.
But he has since looked elsewhere, taking on Jones Jr later this year - potentially in front of a packed house.
Tyson is looking in incredible condition, too as he continues this hard graft.
Continued here:
Mike Tyson reveals all after doctors gave him blood injection that left him feeling weird during stem cell t - The Irish Sun
Global Hematopoietic Stem Cell Transplantation (HSCT) Industry 2020 Market Research With Size, Growth, Manufacturers, Segments And 2026 Forecasts…
By daniellenierenberg
IndustryGrowthInsights (IGI), a prominent market research firm in its own industry, has published a detailed report on Global Hematopoietic Stem Cell Transplantation (HSCT) Market. This market research report provides comprehensive and in-depth analysis on the market which can possibly help an enterprise to identify lucrative opportunities and assist them with fabricating creative business strategies. The market report provides information about the current market scenario regarding the global supply and demand, key market trends and opportunities in the market, and challenges and threats faced by the industry players.
The Hematopoietic Stem Cell Transplantation (HSCT) market report talks about the competitive scenario among the industry players and imparts aspiring and emerging industry players with the future market insights in a detailed manner. This market report includes crucial data and figures which are structured out in a concise yet understandable manner. The research report covers the updates on the government regulations and policies which illustrates key opportunities and challenges of the market. IndustryGrowthInsights (IGI) has been monitoring the market since few years and collaborated with eminent players of the industry to give better insights on the market. It has conducted vigorous research and implied robust methodology to provide accurate predictions about the market.
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Impacts of Advancements and COVID-19 on the market.
Amidst the COVID-19, few segments of the market have witnessed a disruption due to the gap in supply and demand which has impacted the growth of the Hematopoietic Stem Cell Transplantation (HSCT) market. Along with this, the latest advancements have changed the market dynamics of the market. This research report covers the wide-range analysis of the COVID-19 impact to the industry and gives out insights on the change in the market scenario due to the advancements.
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Market Segmentation
Some of the major companies that are covered in the report.
Regen Biopharma IncChina Cord Blood CorpCBR Systems IncEscape Therapeutics IncCryo-Save AGLonza Group LtdPluristem Therapeutics IncViaCord Inc
Note: Additional companies
Based on the type, the market is segmented into
AllogeneicAutologous
Based on the application, the market is segregated into
Peripheral Blood Stem Cells Transplant (PBSCT)Bone Marrow Transplant (BMT)Cord Blood Transplant (CBT)
Based on the geographical location, the market is segregated into
Asia Pacific: China, Japan, India, and Rest of Asia PacificEurope: Germany, the UK, France, and Rest of EuropeNorth America: The US, Mexico, and CanadaLatin America: Brazil and Rest of Latin AmericaMiddle East & Africa: GCC Countries and Rest of Middle East & Africa
IndustryGrowthInsights (IGI) provides yearly updates on the Hematopoietic Stem Cell Transplantation (HSCT) market that assist the clients to stay ahead in the competitive space.
Why one should buy this Hematopoietic Stem Cell Transplantation (HSCT) Report?
The market research report provides all valuable constituents of the market such as revenue growth, product pricing & analysis, growth potential, and guidelines to tackle the challenges in the market. The report covers all the crucial mergers & acquisitions, partnerships, and collaborations that created further created opportunities or in some cases, challenges for the industry players.
This report includes latest product news, advancements, and updates from the prominent player of the industry that has leveraged their position in the market. It also provides business strategies implemented by the key players and yardstick to arrive on informed business decisions. Moreover, it gives insights on the consumer behavior patterns that can help the enterprise to curate the business strategies accordingly.
IndustryGrowthInsights (IGI) bestows the clients with the specialized customized options related to the regional analysis, company analysis, and product analysis, among others.
Complete Table Content of the Market
Executive Summary
Assumptions and Acronyms Used
Research Methodology
Hematopoietic Stem Cell Transplantation (HSCT) Market Overview
Global Hematopoietic Stem Cell Transplantation (HSCT) Market Analysis and Forecast by Type
Global Hematopoietic Stem Cell Transplantation (HSCT) Market Analysis and Forecast by Application
Global Hematopoietic Stem Cell Transplantation (HSCT) Market Analysis and Forecast by Sales Channel
Global Hematopoietic Stem Cell Transplantation (HSCT) Market Analysis and Forecast by Region
North America Hematopoietic Stem Cell Transplantation (HSCT) Market Analysis and Forecast
Latin America Hematopoietic Stem Cell Transplantation (HSCT) Market Analysis and Forecast
Europe Hematopoietic Stem Cell Transplantation (HSCT) Market Analysis and Forecast
Asia Pacific Hematopoietic Stem Cell Transplantation (HSCT) Market Analysis and Forecast
Asia Pacific Hematopoietic Stem Cell Transplantation (HSCT) Market Size and Volume Forecast by Application
Middle East & Africa Hematopoietic Stem Cell Transplantation (HSCT) Market Analysis and Forecast
Competition Landscape
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Global Hematopoietic Stem Cell Transplantation (HSCT) Industry 2020 Market Research With Size, Growth, Manufacturers, Segments And 2026 Forecasts...
Genetic mutations may be linked to infertility, early menopause – Washington University School of Medicine in St. Louis
By daniellenierenberg
Visit the News Hub
Gene in fruit flies, worms, zebrafish, mice and people may help explain some fertility issues
Researchers at Washington University School of Medicine in St. Louis have identified a gene that plays an important role in fertility across multiple species. Pictured is a normal fruit fly ovary (left) and a fruit fly ovary with this gene dialed down (right). Male and female animals missing this gene had substantially defective reproductive organs. The study could have implications for understanding human infertility and early menopause.
A new study from Washington University School of Medicine in St. Louis identifies a specific genes previously unknown role in fertility. When the gene is missing in fruit flies, roundworms, zebrafish and mice, the animals are infertile or lose their fertility unusually early but appear otherwise healthy. Analyzing genetic data in people, the researchers found an association between mutations in this gene and early menopause.
The study appears Aug. 28 in the journal Science Advances.
The human gene called nuclear envelope membrane protein 1 (NEMP1) is not widely studied. In animals, mutations in the equivalent gene had been linked to impaired eye development in frogs.
The researchers who made the new discovery were not trying to study fertility at all. Rather, they were using genetic techniques to find genes involved with eye development in the early embryos of fruit flies.
We blocked some gene expression in fruit flies but found that their eyes were fine, said senior author Helen McNeill, PhD, the Larry J. Shapiro and Carol-Ann Uetake-Shapiro Professor and a BJC Investigator at the School of Medicine. So, we started trying to figure out what other problems these animals might have. They appeared healthy, but to our surprise, it turned out they were completely sterile. We found they had substantially defective reproductive organs.
Though it varied a bit by species, males and females both had fertility problems when missing this gene. And in females, the researchers found that the envelope that contains the eggs nucleus the vital compartment that holds half of an organisms chromosomes looked like a floppy balloon.
This gene is expressed throughout the body, but we didnt see this floppy balloon structure in the nuclei of any other cells, said McNeill, also a professor of developmental biology. That was a hint wed stumbled across a gene that has a specific role in fertility. We saw the impact first in flies, but we knew the proteins are shared across species. With a group of wonderful collaborators, we also knocked this gene out in worms, zebrafish and mice. Its so exciting to see that this protein that is present in many cells throughout the body has such a specific role in fertility. Its not a huge leap to suspect it has a role in people as well.
To study this floppy balloon-like nuclear envelope, the researchers used a technique called atomic force microscopy to poke a needle into the cells, first penetrating the outer membrane and then the nucleuss membrane. The amount of force required to penetrate the membranes gives scientists a measure of their stiffness. While the outer membrane was of normal stiffness, the nucleuss membrane was much softer.
Its interesting to ask whether stiffness of the nuclear envelope of the egg is also important for fertility in people, McNeill said. We know there are variants in this gene associated with early menopause. And when we studied this defect in mice, we see that their ovaries have lost the pool of egg cells that theyre born with, which determines fertility over the lifespan. So, this finding provides a potential explanation for why women with mutations in this gene might have early menopause. When you lose your stock of eggs, you go into menopause.
On the left is a normal fruit fly ovary with hundreds of developing eggs. On the right is a fruit fly ovary that is totally missing the NEMP gene. It is poorly developed and no eggs are visible.
McNeill and her colleagues suspect that the nuclear envelope has to find a balance between being pliant enough to allow the chromosomes to align as they should for reproductive purposes but stiff enough to protect them from the ovarys stressful environment. With age, ovaries develop strands of collagen with potential to create mechanical stress not present in embryonic ovaries.
If you have a softer nucleus, maybe it cant handle that environment, McNeill said. This could be the cue that triggers the death of eggs. We dont know yet, but were planning studies to address this question.
Over the course of these studies, McNeill said they found only one other problem with the mice missing this specific gene: They were anemic, meaning they lacked red blood cells.
Normal adult red blood cells lack a nucleus, McNeill said. Theres a stage when the nuclear envelope has to condense and get expelled from the young red blood cell as it develops in the bone marrow. The red blood cells in these mice arent doing this properly and die at this stage. With a floppy nuclear envelope, we think young red blood cells are not surviving in another mechanically stressful situation.
The researchers would like to investigate whether women with fertility problems have mutations in NEMP1. To help establish whether such a link is causal, they have developed human embryonic stem cells that, using CRISPR gene-editing technology, were given specific mutations in NEMP1 listed in genetic databases as associated with infertility.
We can direct these stem cells to become eggs and see what effect these mutations have on the nuclear envelope, McNeill said. Its possible there are perfectly healthy women walking around who lack the NEMP protein. If this proves to cause infertility, at the very least this knowledge could offer an explanation. If it turns out that women who lack NEMP are infertile, more research must be done before we could start asking if there are ways to fix these mutations restore NEMP, for example, or find some other way to support nuclear envelope stiffness.
This work was supported by the Canadian Institutes of Health, research grant numbers 143319, MOP-42462, PJT-148658, 153128, 156081, MOP-102546, MOP-130437, 143301, and 167279. This work also was supported, in part, by the Krembil Foundation; the Canada Research Chair program; the National Institutes of Health (NIH), grant number R01 GM100756; and NSERC Discovery grant; and the Medical Research Council, unit programme MC_UU_12015/2. Financial support also was provided by the Wellcome Senior Research Fellowship, number 095209; Core funding 092076 to the Wellcome Centre for Cell Biology; a Wellcome studentship; the Ontario Research FundsResearch Excellence Program. Proteomics work was performed at the Network Biology Collaborative Centre at the Lunenfeld-Tanenbaum Research Institute, a facility supported by Canada Foundation for Innovation funding, by the Ontarian Government, and by the Genome Canada and Ontario Genomics, grant numbers OGI-097 and OGI-139.
Tsatskis Y, et al. The NEMP family supports metazoan fertility and nuclear envelope stiffness. Science Advances. Aug. 28, 2020.
Washington University School of Medicines 1,500 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.
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Genetic mutations may be linked to infertility, early menopause - Washington University School of Medicine in St. Louis
‘We got the match’: Happy news for family fighting four-year-old’s Darcy Keeley’s leukaemia – 7NEWS.com.au
By daniellenierenberg
A familys wishes have come true after an inspirational search for a bone marrow donor for Darcy Keeley, four, uncovered a match.
The young Perth boy was diagnosed in June with acute myeloid leukaemia, needing a bone marrow transplant.
Finding a match proved difficult and sparked the campaign Do it for Darcy.
His family took to social media, relentless in their pursuit to help their little boy.
When the story of the boy dubbed the little lion started to spread, thousands of people came forward to try to lend a hand.
Recently one of our most loyal, passionate and enthusiastic supporters, Darcy Keeley, was diagnosed with Leukaemia, requiring 6 months of intensive chemotherapy followed by a bone marrow transplant, Hamersley Carine Footy Club posted on GoFundMe in July.
On their campaign Do it for Darcy, the family explained he was in isolation in Perth Childrens Hospital.
He needs a bone marrow donation and currently the national and international bone marrow registry is yet to find one, they wrote.
Thousands of people rallied around Darcy and his family, signing up to see if they were a potential match to be a donor.
On Thursday, his mum Casey announced in a heartfelt social media post they finally had a match.
IT HAPPENED... guys it actually happened!!! We got the match weve been waiting for! she said.
Darcys transplant doctor confirmed yesterday we have stem cells from some cord blood!
This is a huge relief and allows us to focus on the next step to recovery.
She said an employee at Lifeblood said literally thousands of people registered to try to help Darcy.
Casey also added that the campaign had subsequently led to many new donors.
There have been literally THOUSANDS of ABMDR [Australian Bone Marrow Donor Registry] registrations and new donors as a result of our Do it for Darcy campaign, she said.
The mum said this would help people all around the world, adding they were proud of the awareness the campaign had created.
It is impossible to express our gratitude for all of you who have rallied behind us and registered to be bone marrow donors, donated blood products and spread the word, she said.
As parents, there is nothing more heartwarming than people supporting your child, especially in a time of need.
And the love we have felt wrapped around us [has] helped us as a family immensely.
The loving mother conceded that while they are only at the beginning of fighting this illness, this win will help them move forward.
She also encouraged people to keep spreading awareness.
This is not the end of the fight, Casey said.
Its really just the beginning.
The transplant process will be challenging, but for right now lets focus on this win.
Continued here:
'We got the match': Happy news for family fighting four-year-old's Darcy Keeley's leukaemia - 7NEWS.com.au
Keio University gets OK for iPS-based heart cell transplant plan – The Japan Times
By daniellenierenberg
A health ministry panel on Thursday approved a Keio University clinical research project to transplant heart muscle cells made from induced pluripotent stem (iPS) cells into heart disease patients.
The research will be carried out by a team led by Prof. Keiichi Fukuda for three people between 20 and 74 suffering from dilated cardiomyopathy, which lowers the hearts power to pump blood. The first transplant will be conducted by the end of this year at the earliest.
The team will use iPS cells made by Kyoto University from the blood of a person who has a special immunological type with less risk of rejection.
The team will transform the iPS cells into heart muscle cells and inject about 50 million of them into the heart using a special syringe. Immunosuppressive drugs will be used for about half a year, and the team will spend a year checking to see whether the treatment leads to the development of tumors and irregular heartbeat or whether it restores heart function.
In January, Osaka University conducted the worlds first transplant of heart muscle cells made from iPS cells. The heart muscle cells were made into sheets and pasted on the surface of the patients heart so that a substance they emit can help regenerate the heart muscles. The cells themselves, however, disappear quickly.
Meanwhile, Keio University has confirmed in an experiment on monkeys that cells colonize after a transplant and heart function improves.
The university expects that transplanted cells will colonize over a long period also in the upcoming clinical research project.
According to the team, there are about 25,000 dilated cardiomyopathy patients in Japan.
A startup led by Fukuda is planning a clinical trial aimed at commercializing the iPS-derived cells, hoping they will also be used for the treatment of other cardiac diseases.
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Keio University gets OK for iPS-based heart cell transplant plan - The Japan Times
Global Stem Cell Reconstructive Market- Industry Analysis and Forecast (2020-2027) – Galus Australis
By daniellenierenberg
Global Stem Cell Reconstructive Marketwas valued US$ XX Mn in 2019 and is expected to reach US$ XX Mn by 2027, at a CAGR of 24.5% during a forecast period.
Market Dynamics
The Research Report gives an in-depth account of the drivers and restraints in the stem cell reconstructive market. Stem cell reconstructive surgery includes the treatment of injured or dented part of body. Stem cells are undifferentiated biological cells, which divide to produce more stem cells. Growing reconstructive surgeries led by the rising number of limbs elimination and implants and accidents are boosting the growth in the stem cell reconstructive market. Additionally, rising number of aged population, number of patients suffering from chronic diseases, and unceasing development in the technology, these are factors which promoting the growth of the stem cell reconstructive market. Stem cell reconstructive is a procedure containing the use of a patients own adipose tissue to rise the fat volume in the area of reconstruction and therefore helping 3Dimentional reconstruction in patients who have experienced a trauma or in a post-surgical event such as a mastectomy or lumpectomy, brain surgery, or reconstructive surgery as a result of an accident or injury. Stem cell reconstructive surgeries are also used in plastic or cosmetic surgeries as well. Stem cell and regenerative therapies gives many opportunities for development in the practice of medicine and the possibility of an array of novel treatment options for patients experiencing a variety of symptoms and conditions. Stem cell therapy, also recognised as regenerative medicine, promotes the repair response of diseased, dysfunctional or injured tissue using stem cells or their derivatives.
The common guarantee of all the undifferentiated embryonic stem cells (ESCs), foetal, amniotic, UCB, and adult stem cell types is their indefinite self-renewal capacity and high multilineage differentiation potential that confer them a primitive and dynamic role throughout the developmental process and the lifespan in adult mammal.However, the high expenditure of stem cell reconstructive surgeries and strict regulatory approvals are restraining the market growth.
The report study has analyzed revenue impact of covid-19 pandemic on the sales revenue of market leaders, market followers and disrupters in the report and same is reflected in our analysis.
Global Stem Cell Reconstructive Market Segment analysis
Based on Cell Type, the embryonic stem cells segment is expected to grow at a CAGR of XX% during the forecast period. Embryonic stem cells (ESCs), derived from the blastocyst stage of early mammalian embryos, are distinguished by their capability to distinguish into any embryonic cell type and by their ability to self-renew. Owing to their plasticity and potentially limitless capacity for self-renewal, embryonic stem cell therapies have been suggested for regenerative medicine and tissue replacement after injury or disease. Additionally, their potential in regenerative medicine, embryonic stem cells provide a possible another source of tissue/organs which serves as a possible solution to the donor shortage dilemma. Researchers have differentiated ESCs into dopamine-producing cells with the hope that these neurons could be used in the treatment of Parkinsons disease. Upsurge occurrence of cardiac and malignant diseases is promoting the segment growth. Rapid developments in this vertical contain protocols for directed differentiation, defined culture systems, demonstration of applications in drug screening, establishment of several disease models, and evaluation of therapeutic potential in treating incurable diseases.
Global Stem Cell Reconstructive Market Regional analysis
The North American region has dominated the market with US$ XX Mn. America accounts for the largest and fastest-growing market of stem cell reconstructive because of the huge patient population and well-built healthcare sector. Americas stem cell reconstructive market is segmented into two major regions such as North America and South America. More than 80% of the market is shared by North America due to the presence of the US and Canada.
Europe accounts for the second-largest market which is followed by the Asia Pacific. Germany and UK account for the major share in the European market due to government support for research and development, well-developed technology and high healthcare expenditure have fuelled the growth of the market. This growing occurrence of cancer and diabetes in America is the main boosting factor for the growth of this market.
The objective of the report is to present a comprehensive analysis of the Global Stem Cell Reconstructive Market including all the stakeholders of the industry. The past and current status of the industry with forecasted market size and trends are presented in the report with the analysis of complicated data in simple language. The report covers all the aspects of the industry with a dedicated study of key players that includes market leaders, followers and new entrants. PORTER, SVOR, PESTEL analysis with the potential impact of micro-economic factors of the market has been presented in the report. External as well as internal factors that are supposed to affect the business positively or negatively have been analysed, which will give a clear futuristic view of the industry to the decision-makers.
The report also helps in understanding Global Stem Cell Reconstructive Market dynamics, structure by analysing the market segments and projects the Global Stem Cell Reconstructive Market size. Clear representation of competitive analysis of key players by Application, price, financial position, Product portfolio, growth strategies, and regional presence in the Global Stem Cell Reconstructive Market make the report investors guide.Scope of the Global Stem Cell Reconstructive Market
Global Stem Cell Reconstructive Market, By Sources
Allogeneic Autologouso Bone Marrowo Adipose Tissueo Blood Syngeneic OtherGlobal Stem Cell Reconstructive Market, By Cell Type
Embryonic Stem Cell Adult Stem CellGlobal Stem Cell Reconstructive Market, By Application
Cancer Diabetes Traumatic Skin Defect Severe Burn OtherGlobal Stem Cell Reconstructive Market, By End-User
Hospitals Research Institute OthersGlobal Stem Cell Reconstructive Market, By Regions
North America Europe Asia-Pacific South America Middle East and Africa (MEA)Key Players operating the Global Stem Cell Reconstructive Market
Osiris Therapeutics NuVasives Cytori Therapeutics Takeda (TiGenix) Cynata Celyad Medi-post Anterogen Molmed Baxter Eleveflow Mesoblast Ltd. Micronit Microfluidics TAKARA BIO INC. Tigenix Capricor Therapeutics Astellas Pharma US, Inc. Pfizer Inc. STEMCELL Technologies Inc.
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Global Stem Cell Reconstructive Market- Industry Analysis and Forecast (2020-2027) - Galus Australis
YOUR HEALTH: When the body turns to crystals – WQAD.com
By daniellenierenberg
SAN DIEGO Cystinosis is a disease that slowly and aggressively attacks your organs, tissues, muscles, bones, eyes, even your brain.
It's a genetic disorder with no cure.
And right now, the only option for treatment is an army of pills to slow it down but missing even one dose can be devastating.
Now one man is "patient one", the first to try a new treatment that may save thousands of lives.
21-year old Jordan Janz is living with the rare, unrelenting disorder.
"I was diagnosed at eight months old and basically have been living with it my whole life."
In Jordan, cystine, an amino acid, gets trapped in his cells.
When cystine levels rise, crystals build up all over the body leaving a trail of damage... even causing him to vomit up to 13 times a day.
"It's not how strong you are physically," he said.
"I think it's how strong you are mentally when you come into this."
Traditional cystinosis treatments aim to slow the build up of cystine inside cells.
In order to do that Jordan takes 56 pills each day, but now he hopes to change that, Jordan is the first patient to test a unique gene therapy.
UC San Diego professor Stephanie Cherquie's took stem cells from Jordan's bone marrow, re-engineered the cells, introduced genes that will produce cystinosin, then reinfused Jordan with his own cystinosin-producing cells.
"So, then these cells become a source of healthy stem cells for the rest of the life of the patient," said Stephanie.
Jordan had to take chemo twice a day, but he hasn't let that scare him away.
"I'm doing this obviously for other cystinosis families, right?," said Jordan.
Hoping that many others after him will now get the chance at a better, longer life.
For those born with cystinosis who make it into adulthood, the average lifespan is around 28 years old.
We're told Jordan Janz is making a good recovery. though it is still too soon to tell his long-term prognosis.
If this story has impacted your life or prompted you or someone you know to seek or change treatments, please let us know by contacting Jim Mertens atjim.mertens@wqad.comor Marjorie Bekaert Thomas atmthomas@ivanhoe.com.
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YOUR HEALTH: When the body turns to crystals - WQAD.com
Method regrows cartilage to cushion bones – Futurity: Research News
By daniellenierenberg
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You are free to share this article under the Attribution 4.0 International license.
Researchers have found a way, in mice and human tissue, to regenerate the cartilage that eases movement between bones.
Loss of this slippery and shock-absorbing tissue layer, called articular cartilage, is responsible for many cases of joint pain and arthritis, which afflicts more than 55 million Americans.
The researchers can envision a time when people are able to avoid getting arthritis in the first place by rejuvenating their cartilage before it is badly degraded.
Nearly 1 in 4 adult Americans suffer from arthritis, and far more are burdened by joint pain and inflammation generally.
The researchers figured out how to regrow articular cartilage by first causing slight injury to the joint tissue, then using chemical signals to steer the growth of skeletal stem cells as the injuries heal.
Cartilage has practically zero regenerative potential in adulthood, so once its injured or gone, what we can do for patients has been very limited, says co-senior author Charles K.F. Chan, assistant professor of surgery at Stanford Universitys School of Medicine.
Its extremely gratifying to find a way to help the body regrow this important tissue, Chan says.
The work builds on previous research that resulted in isolation of the skeletal stem cell, a self-renewing cell that is also responsible for the production of bone, cartilage and a special type of cell that helps blood cells develop in bone marrow.
Articular cartilage is a complex and specialized tissue that provides a slick and bouncy cushion between bones at the joints. When this cartilage is damaged by trauma, disease, or simply thins with age, bones can rub directly against each other, causing pain and inflammation, which can eventually result in arthritis.
Damaged cartilage can be treated through a technique called microfracture, in which tiny holes are drilled in the surface of a joint. The microfracture technique prompts the body to create new tissue in the joint, but the new tissue is not much like cartilage.
I realized the only way to understand the process was to look at what stem cells are doing after microfracture.
Microfracture results in what is called fibrocartilage, which is really more like scar tissue than natural cartilage, says Chan. It covers the bone and is better than nothing, but it doesnt have the bounce and elasticity of natural cartilage, and it tends to degrade relatively quickly.
The most recent research arose, in part, through the work of surgeon and lead author Matthew Murphy, a visiting researcher at Stanford who is now at the University of Manchester.
I never felt anyone really understood how microfracture really worked, Murphy says. I realized the only way to understand the process was to look at what stem cells are doing after microfracture.
For a long time, Chan says, people assumed that adult cartilage did not regenerate after injury because the tissue did not have many skeletal stem cells that could be activated. Working in a mouse model, the team documented that microfracture did activate skeletal stem cells. Left to their own devices, however, those activated skeletal stem cells regenerated fibrocartilage in the joint.
But what if the healing process after microfracture could be steered toward development of cartilage and away from fibrocartilage?
The researchers knew that as bone develops, cells must first go through a cartilage stage before turning into bone. They had the idea that they might encourage the skeletal stem cells in the joint to start along a path toward becoming bone, but stop the process at the cartilage stage.
The researchers used a powerful molecule called bone morphogenetic protein 2 (BMP2) to initiate bone formation after microfracture, but then stopped the process midway with a molecule that blocked another signaling molecule important in bone formation, called vascular endothelial growth factor (VEGF).
What we ended up with was cartilage that is made of the same sort of cells as natural cartilage with comparable mechanical properties, unlike the fibrocartilage that we usually get, Chan says. It also restored mobility to osteoarthritic mice and significantly reduced their pain.
As a proof of principle that this might also work in humans, the researchers transferred human tissue into mice that were bred to not reject the tissue, and were able to show that human skeletal stem cells could be steered toward bone development but stopped at the cartilage stage.
The next stage of research is to conduct similar experiments in larger animals before starting human clinical trials. Murphy points out that because of the difficulty in working with very small mouse joints, there might be some improvements to the system they could make as they move into relatively larger joints.
The first human clinical trials might be for people who have arthritis in their fingers and toes. We might start with small joints, and if that works we would move up to larger joints like knees, Murphy says.
Right now, one of the most common surgeries for arthritis in the fingers is to have the bone at the base of the thumb taken out. In such cases we might try this to save the joint, and if it doesnt work we just take out the bone as we would have anyway. Theres a big potential for improvement, and the downside is that we would be back to where we were before.
One advantage of their discovery is that the main components of a potential therapy are approved as safe and effective by the FDA, says co-senior author Michael Longaker, professor of surgery.
BMP2 has already been approved for helping bone heal, and VEGF inhibitors are already used as anti-cancer therapies, he says. This would help speed the approval of any therapy we develop.
Joint replacement surgery has revolutionized how doctors treat arthritis and is very common: By age 80, 1 in 10 people will have a hip replacement and 1 in 20 will have a knee replaced. But such joint replacement is extremely invasive, has a limited lifespan and is performed only after arthritis hits and patients endure lasting pain.
The researchers say they can envision a time when people are able to avoid getting arthritis in the first place by rejuvenating their cartilage in their joints before it is badly degraded.
One idea is to follow a Jiffy Lube model of cartilage replenishment, Longaker says. You dont wait for damage to accumulateyou go in periodically and use this technique to boost your articular cartilage before you have a problem.
The work appears in the journal Nature Medicine.
Support for the research came from the National Institutes of Health, the California Institute for Regenerative Medicine, the Oak Foundation, the Pitch Johnson Fund, the Gunn/Olivier Research Fund, the Stinehart/Reed Foundation, The Siebel Foundation, the Howard Hughes Medical Institute, the German Research Foundation, the PSRF National Endowment, National Center for Research Resources, the Prostate Cancer Research Foundation, the American Federation of Aging Research, and the Arthritis National Research Foundation.
Source: Stanford University
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Method regrows cartilage to cushion bones - Futurity: Research News
Global Hematopoietic Stem Cell Transplantation (HSCT) Market Size, Analytical Overview, Growth Factors, Demand, Trends And Forecast To 2026 -…
By daniellenierenberg
DataIntelo has published a latest market research report on Global Hematopoietic Stem Cell Transplantation (HSCT) Market. The global report is prepared in collaboration with the leading industry experts and dedicated research analyst team to provide an enterprise with in-depth market insights and help them to take crucial business decisions. This report covers current market trends, opportunities, challenges, and detailed competitive analysis of the industry players in the market.
The published report explains about the current supply and demand scenario and presents the future outlook of the market in a detailed manner. DataIntelo has applied a robust market research methodology to bestow the new entrants and emerging players with 360 wide-view analysis on the latest advancements and their impacts on the market. It has congregated massive amount of data on the key segments of the market in an easy to understand format. The research report has laid out the numbers and figures in a comprehensive manner with the help of graphical and pictorial representation which embodies more clarity on the market.
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Report Covers Impacts of COVID-19 to the market.
The on-going pandemic has overhauled various facets of the market. This research report provides the financial impacts and market disturbance on the Hematopoietic Stem Cell Transplantation (HSCT) market. It also includes analysis on the potential lucrative opportunities and challenges in the foreseeable future. DataIntelo has interviewed various delegates of the industry and got involved in the primary and secondary research to confer the clients with information and strategies to fight against the market challenges amidst and after COVID-19 pandemic.
Market Segmentation:
Few of the companies that are covered in the report.
Regen Biopharma IncChina Cord Blood CorpCBR Systems IncEscape Therapeutics IncCryo-Save AGLonza Group LtdPluristem Therapeutics IncViaCord Inc
Note: Additional companies can be included in the list upon the request.
By Product Type:
AllogeneicAutologous
By Applications:
Peripheral Blood Stem Cells Transplant (PBSCT)Bone Marrow Transplant (BMT)Cord Blood Transplant (CBT)
By Geographical Location:Asia Pacific: China, Japan, India, and Rest of Asia PacificEurope: Germany, the UK, France, and Rest of EuropeNorth America: The US, Mexico, and CanadaLatin America: Brazil and Rest of Latin AmericaMiddle East & Africa: GCC Countries and Rest of Middle East & Africa
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Hematopoietic Stem Cell Transplantation (HSCT) Supply Chain Analysis
Hematopoietic Stem Cell Transplantation (HSCT) Pricing Analysis
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Global Hematopoietic Stem Cell Transplantation (HSCT) Market Size, Analytical Overview, Growth Factors, Demand, Trends And Forecast To 2026 -...
Demand for Medical Skin Care Products Market Driven by Shifting Consumer Perceptions and Growing Awareness – Scientect
By daniellenierenberg
Medical skin care products are used for beautifying or to address some other skin care problems. The cosmetic industry is booming and skin care forms a very huge part of this industry. The aesthetic appearance is so important that people spend a lot on skin care products and treatment. People being more technologically aware of the various new skin care products trending in the market. In addition to the aesthetic application, the medical skin care products are also used to address issues such as acne, pimples or scars.
Medical Skin Care Products Market: Drivers and Restraints
The medical skin care products is primarily driven by the need of natural based active ingredients products which are now trending in the market. Consumers demand medical skin care products which favor health and environment. Moreover, the consumers are updated with the trends so that various companies end up providing such products to satisfy the customers. For instance, a single product face mask has thousands of different variants. This offers consumers different options to select the product depending on the skin type. Moreover, the market players catering to the medical skin care products are offering products with advanced technologies. For instance, Santinov launched the CICABEL mask using stem cell material based on advanced technologies. The stem cells used in the skin care product helps to to protect and activate the cells and promote the proliferation of skin epidermal cells and the anagenesis of skin fibrosis.
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Medical Skin Care Products Market: Segmentation
On the basis of product type the medical skin care products market can be segmented as:
On the basis of application, the medical skin care products market can be segment as:
On the basis of distribution channel, the medical skin care products market can be segment as:
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Medical Skin Care Products Market: Overview
Medical skin care products are used to address basic skin problems ranging from acne to scars. There are various advancements in the ingredients used to offer skin care products to the consumers. For instance, the use of hyaluronic acid and retinoids is the latest development in the industry. The anti-aging creams are at the forefront as the help treating issues such as wrinkles, scars, acne, and sun damage. Another, product in demand is the probiotic skincare which include lactobacillus and bifidobacterium.
Medical Skin Care Products Market: Region-wise Outlook
In terms of geography, medical skin care products market has been divided into five regions including North- America, Asia- Pacific, Middle-East & Africa, Latin America and Europe. North America dominated the global medical skin care products market as international players are acquiring domestic companies to make their hold strong in the U.S. LOral is accelerating its U.S. market by signing a definitive agreement with Valeant Pharmaceuticals International Inc. to acquire CeraVe, AcneFree and Ambi skin-care brands for US$ 1.3 billion. The acquisition is expected LOreal to get hold of the brands in the price-accessible segment. Asia Pacific is expected to be the fastest growing region owing to the increasing disposable income and rising awareness towards the skin care products.
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Medical Skin Care Products Market: Key Market Participants
Some of the medical skin care products market participants are Avon Products Inc., Beiersdorf AG, Colgate-Palmolive Company, Kao Corporation, LOral S.A., Procter & Gamble, Shiseido Company, The Estee Lauder Companies Inc., Unilever PLC, Revlon, Clinique Laboratories, llc., Murad, LLC., SkinCeuticals, RMS Beauty, J.R. Watkins and 100% PURE.
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Demand for Medical Skin Care Products Market Driven by Shifting Consumer Perceptions and Growing Awareness - Scientect
Foetal cells are used to make the Oxford coronavirus vaccine. But they came from a foetus in 1973 – ABC News
By daniellenierenberg
Religious leaders have raised ethical doubts over one of Australia's primary coronavirus vaccine hopes because scientists have used foetal cells in its development.
Developers at Oxford University and pharmaceutical firm AstraZeneca are using cell lines from an electively aborted foetus in the vaccine candidate, with Anglican, Catholic and Greek Orthodox leaders questioning the practice.
But using foetal cells in vaccine development isn't new and the Catholic Church has previously expressed qualified support for the use of vaccines derived from these cells under certain circumstances.
We spoke to Bill Lott, a virologist at QUT's Institute of Health and biomedical innovation, to understand the role of foetal cells in vaccine development.
The foetal cells used in vaccine development are derived from a small number of foetuses which were legally terminated decades ago.
The Oxford vaccine uses HEK (human embryonic kidney) 293 cell lines, obtained from a female foetus in the Netherlands in 1973.
"We're using tissues that were from foetuses that were aborted 40, 50, 60 years ago," Dr Lott said.
"It doesn't require newly aborted foetuses."
While living human cells can only divide around 50 times, those foetal cells have been genetically modified so they can divide an infinite number of times.
"That's why we can use the cells that we harvested [decades ago] today," Dr Lott said.
"They're not the actual original cells, they've been immortalised and then propagated over the decades."
This means we'll never need to replace specimens used in development.
"Just by analogy, buying ivory is illegal [because] if you create a market for ivory, then it creates the demand to kill more elephants," Dr Lott said.
"In this case, that's not happening because these foetuses were aborted 60 years ago, 50 years ago, and using these immortalised tissues now is not going to create a need to go and get new ones."
In fact, scientists would prefer to keep using HEK 293 cell lines because they have been repeatedly tried and tested in a laboratory setting and found to be safe.
"When you're making a vaccine you require safety testing," Dr Lott said.
"If we went back and used a different cell type, you're throwing an unknown into the consideration.
"So that will severely slow down your ability to make these things.
"Using HEK 293, we've used it for decades and we know that it's safe."
This week, Australia's Deputy Chief Medical Officer Nick Coatsworth pointed out the use of foetal cells had been a "reality" in past vaccine development.
"The reality for vaccines is that they need cell cultures in order for us to grow them," he said.
"The human cell is a really important part of their development.
"There are strong ethical regulations surrounding the use of any type of human cell, particularly foetal human cells.
"This is a very professional, highly powered research unit at Oxford University.
"I think we can have every faith that the way they have manufactured the vaccine has been against the highest of ethical standards internationally."
Breaking down the latest news and research to understand how the world is living through an epidemic, this is the ABC's Coronacast podcast.
So, how do foetal cells help with vaccine development? Dr Lott explained they operate like a "vaccine factory".
First, scientists need to develop the vaccine candidate and then combine it with an adenovirus vector.
An adenovirus is a particular type of common virus that causes illnesses like bronchitis, pneumonia and a sore throat.
For instance, when you get a cold, you may be infected with an adenovirus, a coronavirus or a rhinovirus.
A vector is an organism that spreads infection by moving pathogens from one host to another.
So an adenovirus vector? "That's an adenovirus that has been sort of emptied out and then you put a different kind of genome in there to make protein," Dr Lott said.
The next step is to put the vaccine/adenovirus vector combination into a big vat of foetal cells.
"The viral vector infects these HEK 293 cells really, really efficiently," Dr Lott said.
"One reason why you use the HEK 293 is because you get essentially 100 per cent infection with the adenoviral vector.
"And what it does is it turns the HEK 293 cells into a vaccine factory."
What do we mean by "vaccine factory"? Dr Lott explains foetal cells begin producing "tons and tons of that modified adenovirus" which they then "spit out into the liquid bit of the cells" called the cell culture media.
"[The foetal cells] start cranking out this massive amount of modified adenovirus, and then you purify those things away from the cell tissue," he said.
"You pull the [cell] media off, and it's just going to be full of the vaccine and essentially no tissue.
"And that's what your vaccine is."
The foetal cells will operate as this "vaccine factory" regardless of whether the vaccine is effective or not so the next step generally involves animal and then human trials of varying scale.
Inherent in the whole process is stripping away the conditioned cell media, where the foetal cells are contained.
The head of the World Health Organization has warned we may never get a silver bullet for COVID-19. What could that future look like in Australia?
That means a successful vaccine developed using foetal cells will have no remnants of those cells in the final product.
"You purify the vaccine away from the cells that they were grown in, and then you destroy all the cells," Dr Lott said.
"So then you're going to take that liquid and you'll purify it some more, but there are not going to be any [foetal] cells in there.
"There's nothing left when it becomes the vaccine that gets delivered."
Foetal tissue has been used with innovative effect in various strands of medical research.
The difference is some of those processes require fresh foetal cells not the "immortalised" cells vaccine developers can use.
"The vaccine work is pretty straightforward," Dr Lott said.
"But cancer research, the research into the mechanisms of various things cystic fibrosis, haemophilia, rheumatoid arthritis that all required fresh foetal tissue."
Scientists studying Zika virus used foetal cells to discover that the virus crossed the placental membrane and caused brain damage in unborn foetuses.
"[That research] brought out a whole raft of therapies and protections for unborn foetuses [and] "saved a lot of lives, including [the lives of] unborn foetuses," Dr Lott said.
Foetal cell lines have been used in the development of various vaccines, including for chicken pox, Ebola, polio, rubella, shingles, Hepatitis A, and rabies.
Foetal tissue has also facilitated breakthroughs in the treatment of various medical issues including cystic fibrosis, haemophilia, IVF, Parkinson's and Alzheimer's diseases, AIDS, and spinal cord injuries.
Scientists have many different methodologies for developing vaccines and there are a variety of reasons why foetal cells aren't always used.
Billions are being poured into the race to find a coronavirus vaccine, with the winner owning a powerful political tool. During the last pandemic an Australian company got there first.
"Some of them don't use it because of ethical issues," Dr Lott said.
"Some of them don't use it because they're not using an adenovirus [vector], so they don't really need the HEK 293.
"And there are other [development] strategies.
"There's an mRNA strategy that's very popular.
"So some of them don't require it."
The development of a coronavirus vaccine was time critical because of the virus' devastating public health and economic impacts, Dr Lott said.
Therefore, it was important for scientists to diversify their methodologies in order to develop a vaccine as quickly as possible.
Both stem cells and foetal cells are critical to innovations in medical research but what's the difference between the two?
Dr Lott explains stem cells are basically the earliest iteration of a foetal cell before the cell differentiates itself into, for example, a hair cell, liver cell, eye cell or skin cells.
"A stem cell is simply a cell that can turn into a different cell types," Dr Lott said.
"That first embryonic stem cell can eventually turn into any kind of cell in your body.
"So you've got embryonic stem cells, and then you've got adult stem cells, and in between are the foetal stem cells [which] are partially differentiated.
"So foetal cells contain not only stem cells some of the foetal cells have already differentiated into their final cell type."
In 2005 and again in 2017, the Catholic Church expressed qualified support for the use of foetal-cell-derived vaccines but only if there was no available alternative.
A 2005 "moral reflection" issued by Pope Benedict XVI specifically addressed the issue.
"As regards the diseases against which there are no alternative vaccines which are available and ethically acceptable, it is right to abstain from using these vaccines if it can be done without causing children, and indirectly the population as a whole, to undergo significant risks to their health," the Pope wrote.
"However, if the latter are exposed to considerable dangers to their health, vaccines with moral problems pertaining to them may also be used on a temporary basis.
"We find a proportional reason, in order to accept the use of these vaccines in the presence of the danger of favouring the spread of the pathological agent."
In 2017, the life ethics arm of the Catholic Church issued a statement that: Catholic parents could vaccinate their children with a "clear conscience" that "the use of such vaccines does not signify some sort of cooperation in voluntary abortion".
Earlier this year and in the context of the coronavirus vaccine race, John Di Camillo, an ethicist with the National Catholic Bioethics Center, confirmed: "One is allowed to make use of [vaccine derived from foetal tissue] where there's a serious threat to the health or life of the individual, or of the greater population.
"This does not amount to a strictobligationto use it, but it certainly can be a legitimate choice in conscience if theres that serious reason, and there's no other reasonable alternative."