They hold huge promise, but stem cell-based spinal cord treatments wont be clinically available in the near future

My three-year-old son was born with a very large spinal lipoma. He was considered quadriplegic. Through conventional physical and occupational therapies and surgery to remove some of the lipoma he has gained enough function to walk with a walker and use his arms. However, he is experiencing some regression as his nerves are dying.

I have saved the cord blood from his younger brother and sister. New research where mice are being paralyzed and then injected with stem cells looks very promising to us. The mice nerves that are sick or weak are being protected and strengthened. Our son needs his nerves protected from degeneration.

Conventional surgery is no longer an option because the nerve roots travel in and out of the lipoma and cannot be separated from the lipoma. Our only hope is to protect and strengthen what function he currently has.

My question is: How long before this type of stem cell therapy will be used on humans, more specifically children? And how do we get to be first in line? If it is 10 or 20 years away, there may be no way to save the function our son has worked so hard to gain. I havent read anything about risks or side effects. There have to be some, what are they? Also, are there other countries that are more aggressive in their use of stem cells on humans for treating paralysis resulting from spinal cord injury?

Barbara BourgeoisCentreville, Virginia, USA

There isnt an easy answer here, and Im not clear as to why function is being lost at this pointin particular, whether the lipoma is recurring. If this is the case, resolution of the lipoma is the main issue. In some instances, it is impossible to completely remove the tumor, severely limiting the potential benefits of secondary therapeutics (such as stem cells). However, on the topic of stem cells in particular, there are several issues to discuss.

First, there are many sources of stem cells, and this affects their potential clinical use. Cord blood-derived stem cells are probably the farthest away from potential clinical use for spinal cord injury at this point, because there has been less basic research done with them so far. Human embryonic and adult stem cell lines may be somewhat closer, but research on these in the laboratory has been somewhat mixedsome very promising results with regaining motor function, and some big potential concerns, such as causing tumor formation.

As a result, we are most likely still years away from testing these treatments in patients, even to establish safety. Some other kinds of cell treatments, such as ensheathing glial cells, are being tried in the clinic in China, Russia and Portugal based on previous laboratory research in the US. However, none of these overseas trials has been designed in accordance with US standards to rigorously test safety and efficacy, and it is very difficult to evaluate the patchy data coming out so far.

To sum up, as a researcher, I think stem cells hold a huge amount of promise, but we arent yet at a point where this work will be translated to the clinic in the immediate future.

Answered by Aileen J. Anderson ~ 1/22/2004

Posted on January 27th, 2004 in General SCI and Human Interest. Tagged: stem cells

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When Will Stem Cells Heal Spinal Cord Damage?

Spinal Cord Stem Cells Comments Off on When Will Stem Cells Heal Spinal Cord Damage? | May 13th, 2020

Bioscience Americas and the Global Institute of Stem Cell Therapy and Research would like to extend a special thank you to the Christopher and Dana Reeve Foundation for their support relating to our work at the University California Irvin and the Anderson Laboratory. We have made exciting progress using stem cells to treat cervical spinal cord injuries because of their generosity.

Now, based on the results of Dr. Andersons Phase I/II clinical trial, our research partners are conducting a Phase II proof of concept trial using HuCNS-SC in cervical spinal cord injury. In this study, research participants are being treated between 10 to 23 months post-injury.

Spinal Cord Injury (SCI) is damage to the human spinal cord into three different segments of the neural tissue leading to a severe form of motor and sensory loss. The kind of damage can be differentiated as:

In most of the reported cases of SCI, damage can be due to trauma or disease. Apart from the physical damage and complete dependency on caregivers, SCI can be emotionally damaging as well. Due to dependency even on basic mobility, negative attitudes of suffering trauma forever and frequent mood swings can lead suffers to remove themselves from social participation. Thus more than 30% of the reported cases of SCI showed significant signs of depression and negative impact on the functional improvement of overall health.

How prevalent is SCI?

Since SCI is associated with the loss of mobility, paralysis, and mortality due to other opportunistic infections, it is known as one of the most critical and disastrous medical conditions. Every year around 2 million to 5 million people are reported to suffer from spinal cord injury. On an average, middle-aged and young adult males are more susceptible to SCI mainly due to avoidable causes such as road accidents, injury, falls or violence. Mortality associated with SCI has been observed to be the highest immediately after the injury than in later years. The risk of mortality doubles with the severity level and is observed to be strongly influenced by the immediate availability of the best medical care. Preventable secondary opportunistic infections are also reported to be a major cause of death in many SCI patients, especially in the lower income groups.

About 90% of patients in the age group of 20-45 have been reported to face other complications such as limited employment, decreased quality of life, and severe depression.

Factors responsible for SCI.

In general, a spinal cord injury is a result of to the severe damage to different parts of the spinal cord such as the vertebral column, ligaments or the spinal disks. This typically originates from sudden trauma to the spinal cord such as fracturing, crushing or dislocating one or more vertebrae. Additional damage has been reported due to excessive bleeding, swelling, inflammation as well as other opportunistic infections. The most common reported causes of Spinal Cord Injury are:

Symptoms Associated with SCI

In general, the severity, as well as the area of injury, are the factors to be of concern in most of the cases of Spinal Cord Injury. On the basis of severity of injury, SCI is classified as:

What goes wrong in Spinal Cord Injury?

The human spinal cord is a fragile bridge connecting the brain to the other organs of the body. The spinal cord is encased in a protective covering of spinal vertebrae of the spinal column to prevent its damage from shock or injury. Our central nervous system, i.e., brain and spinal cord, is made up of millions of cells which coordinate and communicate to pass on the information from the brain to the other organs of the body via the spinal cord. This information is passed in the form of electrical signals which are then decoded by the specific organ.

Each neuron is made up of a cellular body with a long slender projection called the nerve fiber. These fibers are attached to other fibers to form a dense network of cells. In general, neurons carrying messages down the cord from the brain to other organs of the body are known as Motor Neurons. These neurons control the muscles of some of the important internal organs of the body such as heart, stomach, intestine, etc. The neurons traveling up the cord to the brain are known as Sensory Neurons, carrying sensory information from skin, joints, and muscles to control our ability to sense, touch and regularize temperature.

These neurons are insulated from the outer side by the coating of Oligodendrocytes and myelin sheath. These cells insulate the neuron to protect them from sudden damage and shock.

If any of the above types of cells are affected due to sudden damage such as shearing, laceration, stretching or shock, then the network of cells is disturbed due to which the passage of information from the brain to the spinal cord and vice versa is halted.

How Stem Cells treatment can help.

Stem cells are the mother cells that are responsible for developing an entire human body from tiny two-celled embryos. Due to their unlimited divisions and strong power to differentiate into all the cells of different lineage, the power of stem cells has been harnessed by our technology to isolate them outside the human body, concentrate in a clean environment, and implant back.

Thus stem cells treatment involves administration of concentrated cells in the targeted area, wherein they can colonize in the damaged area, adapt the properties of resident stem cells and initiate some of the lost functions that have been compromised by the disease or injury.

Thus with our standardized, broad-based and holistic approach, it is now possible to obtain noticeable improvements in SCI cases, in the symptoms as well as their functional abilities.

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Spinal Cord Injury Recovery Through Stem Cell Therapy

Spinal Cord Stem Cells Comments Off on Spinal Cord Injury Recovery Through Stem Cell Therapy | May 13th, 2020

Akin to cystic fibrosis (CF), scientists understand that certain mutations contribute to the development of the fatal neurological disorder amyotrophic lateral sclerosis (ALS). And much like CF drugmaker Vertex, a small Cambridge, Massachusetts-based biotech is forging a path to engineering precision therapies to treat the disease that killed visionary physicist Stephen Hawking.

The company, christened QurAlis, now has $42 million in its coffers with three preclinical programs and 5 employees (including senior management) to combat an illness that has long flummoxed researchers, resulting in a couple of approved therapies over the course of decades, neither of which attacks the underlying cause of the rare progressive condition that attacks nerve cells located in the brain and spinal cord responsible for controlling voluntary muscles.

ALS garnered international attention when New York Yankees player Lou Gehrig abruptly retired from baseball in 1939, after being diagnosed with the disease. In 2014, ALS returned to the spotlight with the Ice Bucket Challenge, which involved people pouring ice-cold water over their heads, posting a video on social media, and donating funds for research on the condition.

QurAlis chief Kasper Roet, whose interest in ALS was piqued while he was working on his PhD at the Netherlands Institute for Neuroscience focusing on a treatment for spinal cord paralysis and moonlighting at the Netherlands Brain Bank as an ad-hoc autopsy team coordinator, saw an opportunity to combat ALS when Harvard scientists Kevin Eggan and Clifford Woolf pioneered some new stem cell technology.

Essentially, they found a way to take skin cells from a patient, turn them into stem cells, and turn those into the nerve cells that are degenerating. Thats the missing link, Roet said. So now we can finally use patients own cells to both do target discovery and develop potential therapeutics.

So Roet packed up his things and shifted base to Boston to learn more, with plans to head back to Europe to start a company. He never left. QurAlis was born in 2016, working out of a co-working space called LabCentral after winning a spot via an Amgen-sponsored innovation competition. The company was carved out of a collaboration with Eggans startup Q-State Biosciences, which developed laser technology to examine cell behavior examining how a neuron fires was imperative in the drug discovery process for ALS.

QurAlis, which counts Vertexs founding scientist Manuel Navia as an advisor, now has three preclinical programs. The furthest along is a therapy designed to target a specific potassium channel that is implicated in certain ALS patients the plan is to take that small molecule into the clinic next year, Roet said.

It has become really clear that if you understand why a specific tumor is developing you can develop very specific targeted therapies, he explained in an interview drawing a parallel between ALS and oncology. Thats exactly the same strategy that we are following for ALS. The genetics have shown that over 25 genes are causing the (ALS) mutations. Some of them work together, some of them are very dominant and work alone what we are doing is trying to get those specific proteins that are tied to very specific ALS populations, where we know that that specific target plays a very important and crucial role in the development of the disease.

In 2018, QurAlis scored seed funding from Amgen, Alexandria, and MP Healthcare Venture Management. The Series A injection was led by LS Polaris Innovation Fund, lead seed investor Mission BioCapital, INKEF Capital and the Dementia Discovery Fund, and co-led by Droia Ventures. Additional new investors include Mitsui Global Investment and Dolby Family Ventures, and existing investors Amgen Ventures, MP Healthcare Venture Management, and Sanford Biosciences also chipped in.

Roet is not sure how long these funds will last, particularly given the uncertainty of the coronavirus pandemic. But some of the capital will be used in hiring, given that the QurAlis team is comprised of a mere five people, including Roet.

Weve been very productive, he said. But we can definitely use some extra hands.

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Precision therapy approach secures small biotech $42M haul to combat disease that inspired the Ice Bucket Challenge - Endpoints News

Spinal Cord Stem Cells Comments Off on Precision therapy approach secures small biotech $42M haul to combat disease that inspired the Ice Bucket Challenge – Endpoints News | May 13th, 2020

DUBLIN--(BUSINESS WIRE)--The "Global Stem Cell Therapy Market By Type (Allogeneic, Autologous, Syngeneic), By Source of Stem Cells (Adipose Tissue, Bone Marrow, Neural, Embryo/Cord Blood derived, iPSCs, Others), By Application, By End Users, By Region, Forecast & Opportunities, 2025" report has been added to ResearchAndMarkets.com's offering.

The Global Stem Cell Therapy Market is expected to grow at a formidable rate of around 12% during the forecast period. The industry is segmented based on type, source of stem cells, application, end-users, company and region.

The market is driven by the growing popularity and awareness pertaining to the use of stem cells for the prevention and cure of certain life threatening diseases. Additionally, increase in number of stem cell banks and growing investments by the government and private organizations for the development of stem cell preservation infrastructure is further propelling the market across the globe.

Based on type, the market can be categorized into allogeneic, autologous and syngeneic. The allogenic type segment is expected to register the highest growth during forecast period attributable to the rising commercialization of allogeneic stem cell therapy products, wider therapeutic applications of allogeneic stem cells, easy production scale-up process, growing number of clinical trials related to allogeneic stem cell therapies, among others.

Based on end-users, the market can be bifurcated into hospitals and clinics. The hospitals segment is expected to dominate the market during the forecast years. This can be accredited to the rising preference for stem cell therapies offered by hospitals proves beneficial for the business growth. Hospitals have affiliations with research laboratories and academic institutes that carry out research activities for developing stem cell therapies. On introduction and approval of any novel stem therapy, hospitals implement it immediately.

Regionally, the stem cell therapy market has been segmented into various regions namely Asia-Pacific, North America, South America, Europe, and Middle East & Africa. Among these regions, North America is expected to dominate the overall stem cell therapy market during the next five years on account of the increasing number of clinical trials for stem cell-based products and increasing public-private funding & research grants.

Major players operating in the Global Stem Cell Therapy Market include Osiris Therapeutics, Inc., MEDIPOST Co., Ltd., Anterogen Co., Ltd., Pharmicell Co., Ltd., Holostem Terapie Avanzate S.r.l., JCR Pharmaceuticals Co., Ltd., NuVasive, Inc., RTI Surgical, Inc., AlloSource, Thermo Fisher Scientific and others. The companies are developing advanced technologies and launching new services in order to stay competitive in the market.

Years considered for this report:

Objective of the Study

Key Topics Covered

1. Product Overview

2. Research Methodology

3. Executive Summary

4. Voice of Customer

5. Global Stem Cell Therapy Market Outlook

5.1. Market Size & Forecast

5.1.1. By Value

5.2. Market Share & Forecast

5.2.1. By Type (Allogeneic, Autologous, Syngeneic)

5.2.2. By Source of Stem Cells (Adipose Tissue, Bone Marrow, Neural, Embryo/Cord Blood Derived, iPSCs, Others)

5.2.3. By Application (Musculoskeletal, Wound & Injury, Cardiovascular Disease (CVD), Surgery, Acute Graft-Versus-Host Disease, Drug Discovery & Development, Others)

5.2.4. By End Users (Hospitals v/s Clinics)

5.2.5. By Company (2019)

5.2.6. By Region

5.3. Product Market Map

6. Asia-Pacific Stem Cell Therapy Market Outlook

7. Europe Stem Cell Therapy Market Outlook

8. North America Stem Cell Therapy Market Outlook

9. South America Stem Cell Therapy Market Outlook

10. Middle East and Africa Stem Cell Therapy Market Outlook

11. Market Dynamics

11.1. Drivers

11.2. Challenges

12. Market Trends & Developments

13. Competitive Landscape

13.1. Osiris Therapeutics, Inc.

13.2. MEDIPOST Co. Ltd.

13.3. Anterogen Co. Ltd.

13.4. Pharmicell Co. Ltd.

13.5. Holostem Terapie Avanzate S.r.l.

13.6. JCR Pharmaceuticals Co. Ltd.

13.7. NuVasive, Inc.

13.8. RTI Surgical, Inc.

13.9. AlloSource

13.10. Thermo Fisher Scientific

14. Strategic Recommendations

For more information about this report visit https://www.researchandmarkets.com/r/hmawq6

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Global Stem Cell Therapy Market Forecast & Opportunities, 2025 - ResearchAndMarkets.com - Business Wire

Bone Marrow Stem Cells Comments Off on Global Stem Cell Therapy Market Forecast & Opportunities, 2025 – ResearchAndMarkets.com – Business Wire | May 13th, 2020

A Scunthorpe mum who underwent 'brutal' stem cell treatment says the hardest part was not being able to see her daughters.

Joanne While has recently passed the six month anniversary of the treatment to wipe out and then regrow her immune system.

The mum-of-three was diagnosed with Multiple Sclerosis (MS) at the age of 31, and wasn't eligible for trial treatments in the UK.

The HSCTtreatment in Mexico saw her undergo chemotherapy and then have stem cells transplanted in the hopes of stopping the damage that the MS was causing. Some MS patients have also seen their symptoms be reversed from this.

"It was a very harsh, brutal treatment. I had to be kept in a special apartment where I protected from all germs. There was a lot of sickness and just getting out of bed some days was difficult," Joanne said.

"The hardest part was being away from my family. My ex-partner was very kind in taking a months unpaid leave to come to Mexico and help me through the treatment.

"At the end of the day, all of the treatment has been worth it.

"I had a brain MRI scan two months ago which showed that the MS lesions hadnt grown since last time. My body is still recovering, so time will tell exactly how good the news is."

The HSCT (haematopoietic stem cell transplantation) treatment cost the family 45,000 including flights and visas. They launched an online fundraising page last year to help cover the costs.

With her weak immune system still being rebuilt, Joanne has been shielding since before the coronavirus outbreak began.

Her family have adopted extremely strict hygiene measures at the Scunthorpe home to keep her healthy during this critical time.

HSCT is a chemotherapy-based medical procedure that wipes out the immune system and reboots it using a patient's own stem cells, which are harvested from their blood or bone marrow,

The aim is to reset the immune system to stop it attacking the rest of the body, therefore halting the progression of the MS.

It is the only medical procedure currently available that has halted the progression of the majority of patients undertaking it.

HSCT is currently available only on a trial basis in the UK, and only for individuals who have been unsuccessful with the range ofdisease modifying therapies. Each time it fails, irreparable damage is being done and the disease continues to progress.

"Before the outbreak, it had just gotten to the point where I was able to venture out for a coffee, but of course all of that has stopped now," Joanne said.

"I had to pull my daughters out of school early to minimise the risk of them bringing the virus home. Now we regularly sanitise the house and change clothes whenever we have to enter or leave in order to keep it as clean as possible.

"My eldest daughter, who is 24, has been wonderful as my carer. She has stopped work to prevent her from catching any infections.

"Im often tired and need a three hour nap in the afternoon, which can be difficult with a five-year-old in the house. Its been a balancing act, but Im so grateful to everyone who helped me during or since the treatment.

"Shielding can be frustrating, but its all about your mindset when you look at it. Its not that I cant go outside I get to be at home in my favourite place with my daughters."

Due to her compromised immune system, Joanne has had to start her vaccinations again and has just been given those that are usually given to babies.

Tests have also shown that her white blood count has recently decreased to the point it was in Mexico, although Joanne has hopeful it will recover.

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HSCT can initially cause mobility issues and stiffness in muscles, which Joanne is having physio to manage.

She is documenting her recovery on her Facebook page 'Jo's HSCT Journey'.

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Mum's brutal stem cell treatment has 'all been worth it' as she enjoys time with family - Grimsby Live

Bone Marrow Stem Cells Comments Off on Mum’s brutal stem cell treatment has ‘all been worth it’ as she enjoys time with family – Grimsby Live | May 13th, 2020

The Covid-19 (coronavirus) pandemic is impacting society and the overall economy across the world. The impact of this pandemic is growing day by day as well as affecting the supply chain. The COVID-19 crisis is creating uncertainty in the stock market, massive slowing of supply chain, falling business confidence, and increasing panic among the customer segments. The overall effect of the pandemic is impacting the production process of several industries including Life science Industry, and many more. Trade barriers are further restraining the demand- supply outlook. As government of different regions have already announced total lockdown and temporarily shutdown of industries, the overall production process being adversely affected; thus, hinder the overall Stem Cell Therapy market globally. This report on Stem Cell Therapy market provides the analysis on impact on Covid-19 on various business segments and country markets. The report also showcase market trends and forecast to 2027, factoring the impact of Covid -19 Situation.

To get sample Copy of the report, along with the TOC, Statistics, and Tables please visit @https://www.theinsightpartners.com/sample/TIPHE100000991/

Stem cell therapy is a technique which uses stem cells for the treatment of various disorders. Stem cell therapy is capable of curing broad spectrum of disorders ranging from simple to life threatening. These stem cells are obtained from different sources, such as, adipose tissue, bone marrow, embryonic stem cell and cord blood among others. Stem cell therapy is enables to treat more than 70 disorders, including degenerative as well as neuromuscular disorders. The ability of a stem cell to renew itself helps in replacing the damaged areas in the human body.

MARKET DYNAMICSIncrease in the number of stem cell banking facilities and rising awareness on the benefits of stem cell for curing various disorders are expected to drive the market during the forecast period. Rise in number of regulations to promote stem cell therapy and increase in number of funds for research in developing countries are expected to offer growth opportunities to the market during the coming years.

Key Players

The research provides answers to the following key questions:

The study conducts SWOT analysis to evaluate strengths and weaknesses of the key players in the Stem Cell Therapy market. Further, the report conducts an intricate examination of drivers and restraints operating in the market. The report also evaluates the trends observed in the parent market, along with the macro-economic indicators, prevailing factors, and market appeal according to different segments. The report also predicts the influence of different industry aspects on the Stem Cell Therapy market segments and regions.

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Our research and insights help our clients anticipate upcoming revenue compartments and growth ranges. This will help our clients invest or divest their assets.

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It is extremely vital to have an impartial understanding of market opinions for a strategy. Our insights provide a keen view on the market sentiment. We keep this reconnaissance by engaging with Key Opinion Leaders of a value chain of each industry we track.

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Our research ranks investment centers of market by considering their future demands, returns, and profit margins. Our clients can focus on most prominent investment centers by procuring our market research.

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Our research and insights help our clients identify compatible business partners.

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Stem Cell Therapy Market Segmented by Region/Country: North America, Europe, Asia Pacific, Middle East & Africa, and Central & South America

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COVID-19 Impact on STEM CELL THERAPY MARKET 2020 TO 2027-EXPANDING WORLDWIDE WITH TOP PLAYERS FUTURE BUSINESS SCOPE AND INVESTMENT ANALYSIS REPORT -...

Bone Marrow Stem Cells Comments Off on COVID-19 Impact on STEM CELL THERAPY MARKET 2020 TO 2027-EXPANDING WORLDWIDE WITH TOP PLAYERS FUTURE BUSINESS SCOPE AND INVESTMENT ANALYSIS REPORT -… | May 13th, 2020

As a skin-care brand, Augustinus Bader prides itself on releasing products not out of frivolity or trying to up its bottom line, but out of necessity. As Charles Rosier, cofounder and CEO of Augustinus Bader, puts it: "We want to only add product when we feel we are relevant," he tells Allure via phone from Paris, France, where he is currently self-isolating. "Our latest initiative was a reaction to something that we felt was needed by the consumer by everyone."

The brand's newest offering, The Hand Treatment, is meant to be just that. As it stands, given the current COVID-19 situation, we are all washing our hands (or should be) much more than we might otherwise normally do so. However, as you've also likely experienced dry skin and flaking cuticles, just as is the case with everywhere else on the body, overwashing the skin on our hands can bring on a host of issues all their own. Even if you forgo washing for an alcohol-based hand sanitizer, the effects can be just as profound if not more so.

"We are seeing more and more hand dermatitis," explains New York City-based board-certified dermatologist Dhaval Bhanusali, who is not affiliated with Augustinus Bader. "By overwashing, we strip the good oils from our skin and leave it dry and susceptible to breakdown."

Our skin's moisture barrier is responsible for keeping the good things in (like hydration and natural oils) and the bad things out (like allergens and bacteria). When the lipid barrier on the hands (or anywhere on the body) becomes too dried out, it can literally start to break down and lose functionality. Simply put, in order for our skin to function and remain healthy, its barrier must be intact. However, we're not saying that you should stop washing your hands just that it's equally important to replenish hydration after doing so.

That's where Augustinus Bader's new Hand Treatment comes into play. It's formulated with a blend of ultra-nourishing ingredients, including vitamin E, glycerin, and shea butter, as well as honey and white peony extract both of which also have natural antibacterial properties. However, as with all Bader products, the key ingredient is something called TFC8, which stands for its proprietary Trigger Factor Complex-8.

For those unfamiliar with the brand, TFC8 is not one singular ingredient but rather, a proprietary blend of "natural amino acids, high-grade vitamins, and synthesized molecules naturally found in skin," cosmetic chemist Ginger King (who is not affiliated with the brand) previously told Allure. In a nutshell, TFC8 provides an ideal environment in which our skin's stem cells can naturally regenerate it acts as a sort of guide, helping to set the skin's inherent repair system back on track.

"The TCF8 technology focuses on intrinsic repair processes based in intrinsic repair cells, which we call stem cells," Bader explains. "We wanted to develop a hand cream with our technology, which helps to protect and care for the skin."

The results of using TFC8 topically, according to Augustinus Bader himself, a German stem-cell scientist, and his many devotees (our own editor in chief among them), read like a laundry list of skin-care goals: Increased hydration, a strengthened skin barrier, faded fine lines, and dark spots. The proprietary ingredient is, of course, also formulated into the brand's other offerings: two creams for the face, one for the body, and most recently, a makeup primer collaboration with Victoria Beckham Beauty.

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Augustinus Bader Launches The Hand Treatment in Response to COVID-19 Pandemic Exclusive Details - Allure

Skin Stem Cells Comments Off on Augustinus Bader Launches The Hand Treatment in Response to COVID-19 Pandemic Exclusive Details – Allure | May 13th, 2020

CRANFORD, N.J., May 13, 2020 /PRNewswire/ -- Citius Pharmaceuticals, Inc. ("Citius" or the "Company") (Nasdaq: CTXR), a specialty pharmaceutical company focused on developing and commercializing critical care drug products, today announced that data on NoveCite MSCs will be presented this week at the American Society of Gene and Cell therapy (ASGCT) annual meeting. NC-MSCs are made by Novellus, Inc. ("Novellus"), a Cambridge-based biotechnology company, using its patented mRNA-based cell-reprogramming process. Earlier this year, Citius signed an exclusive option agreement to in-license NC-MSCs for acute respiratory distress syndrome (ARDS), including in COVID-19 patients, from Novellus.

The data to be presented show that NC-MSCs secrete higher levels of anti-inflammatory proteins compared to MSCs derived from bone marrow. From the abstract: "Comparative secretome analysis showed overexpression of multiple neuroprotective and anti-inflammatory factors, including CXCL1, VEGF-A, and CXCL5." In addition, NC-MSCs showed therapeutic benefit in an experimental autoimmune encephalomyelitis (EAE) mouse model, delaying disease progression and improving the clinical score compared to the control group, while bone marrow-derived MSCs showed no difference from the control.

"We are pleased to present these data at the annual meeting of the ASGCT," said Matt Angel, PhD, co-founder and Chief Science Officer at Novellus. "While conventional MSCs have shown promise in the treatment of inflammatory lung disease, protein secretion and manufacturability remain challenges for these approaches. The data that will be presented this week show that iPSC-derived MSCs secrete higher levels of anti-inflammatory proteins, and exhibit greater expansion potential than bone marrow-derived MSCs. We believe that these properties make iPSC-derived MSCs especially well-suited for an allogeneic cell therapy for ARDS."

"Last month Citius signed an exclusive option agreement with Novellus for worldwide development and commercial rights related to the use of these uniquely derived MSCs for the treatment of ARDS. The pre-clinical data that is being presented at the ASGCT annual meeting adds to our confidence in the higher potency of these MSCs, which we expect will result in better outcomes for patients with COVID-19 and ARDS," stated Myron Holubiak, CEO of Citius Pharmaceuticals. "We intend to study these cells in the clinic later this year to determine safety, efficacy, and the optimal dose of these cells in moderate to severe ARDS patients with COVID-19."

Citius has submitted a pre-IND meeting request and supporting briefing documents to the Center for Biologics Evaluation and Research ("CBER") of the FDA under the Coronavirus Treatment Acceleration Program (CTAP) for use of these MSCs for patients with Acute Respiratory Distress Syndrome (ARDS) due to SARS-CoV-2 disease.

Presentation Information:Title:Mesenchymal Stem Cells (MSCs) Generated Using mRNA Reprogramming Show Enhanced Growth Potential, Secretome, and Therapeutic Efficacy in a Demyelinating Disease ModelPresenter:Harris, Jasmine, Novellus, Inc.Date and Time: Wednesday, May 13 | 5:30 PM - 6:30 PM

About Acute Respiratory Distress Syndrome (ARDS)ARDS is a type of respiratory failure characterized by rapid onset of widespread inflammation in the lungs. ARDS is a rapidly progressive disease that occurs in critically ill patients most notably now in those diagnosed with COVID-19. ARDS affects approximately 200,000 patients per year in the U.S., exclusive of the current COVID-19 pandemic, and has a 30% to 50% mortality rate. ARDS is sometimes initially diagnosed as pneumonia or pulmonary edema (fluid in the lungs from heart disease). Symptoms of ARDS include shortness of breath, rapid breathing and heart rate, chest pain (particularly while inhaling), and bluish skin coloration. Among those who survive ARDS, a decreased quality of life is relatively common.

About Coronavirus Treatment Acceleration Program (CTAP)In response to the pandemic, the FDA has created an emergency program called the Coronavirus Treatment Acceleration Program (CTAP) to accelerate the development of treatments for COVID-19. By redeploying staff, the FDA is responding to COVID-19-related requests and reviewing protocols within 24 hours of receipt. The FDA said CTAP "uses every available method to move new treatments to patients as quickly as possible, while at the same time finding out whether they are helpful or harmful." In practice, that means developers of potential treatments for COVID-19 will benefit from an unusually faster track at the FDA to shorten wait times at multiple steps of the process.

About Citius Pharmaceuticals, Inc.Citius is a late-stage specialty pharmaceutical company dedicated to the development and commercialization of critical care products, with a focus on anti-infectives and cancer care. For more information, please visit http://www.citiuspharma.com.

About Novellus, Inc.Novellus is a pre-clinical stage biotechnology company developing engineered cellular medicines using its non-immunogenic mRNA, nucleic-acid delivery, gene editing, and cell reprogramming technologies. Novellus is privately held and is headquartered in Cambridge, MA. For more information, please visit http://www.novellus-inc.com.

Safe HarborThis press release may contain "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Such statements are made based on our expectations and beliefs concerning future events impacting Citius. You can identify these statements by the fact that they use words such as "will," "anticipate," "estimate," "expect," "should," and "may" and other words and terms of similar meaning or use of future dates. Forward-looking statements are based on management's current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition, and stock price. Factors that could cause actual results to differ materially from those currently anticipated are: the risk of successfully negotiating a license agreement with Novellus within the option period; our need for substantial additional funds; the ability to access the FDA's CTAP program for the MARCO trial; the estimated markets for our product candidates, including those for ARDS, and the acceptance thereof by any market; risks associated with conducting trials for our product candidates, including those expected to be required for any treatment for ARDS and our Phase III trial for Mino-Lok; risks relating to the results of research and development activities; risks associated with developing our product candidates, including any licensed from Novellus, including that preclinical results may not be predictive of clinical results and our ability to file an IND for such candidates; uncertainties relating to preclinical and clinical testing; the early stage of products under development; risks related to our growth strategy; our ability to obtain, perform under, and maintain financing and strategic agreements and relationships; our ability to identify, acquire, close, and integrate product candidates and companies successfully and on a timely basis; our ability to attract, integrate, and retain key personnel; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions, or circumstances on which any such statement is based, except as required by law.

Contact:Andrew ScottVice President, Corporate Development(O) 908-967-6677 x105 [emailprotected]

SOURCE Citius Pharmaceuticals, Inc.

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Citius Announces Data on NoveCite Mesenchymal Stem Cells (NC-MSCs) to be Presented at the American Society of Gene and Cell Therapy (ASGCT) Annual...

Skin Stem Cells Comments Off on Citius Announces Data on NoveCite Mesenchymal Stem Cells (NC-MSCs) to be Presented at the American Society of Gene and Cell Therapy (ASGCT) Annual… | May 13th, 2020

XconomyBoston

Few drugs exist that treat amyotrophic lateral sclerosis, a progressive disease that kills the nerve cells that allow patients to initiate and control muscle movement.

QurAlis, a Cambridge, MA-based startup, has an ambitious plan to develop a number of precision therapies for the disease based on forms of the condition identified by genetic mutation or a biomarker that CEO Kasper Roet (pictured) hopes to could one day, in combination, help most ALS patients.

Now the company has raised $42 million from investors in the US, Europe, and Japanmoney that will fund a move from the LabCentral incubator in Kendall Square to its own office, more than double the companys headcount by years end, and get at least one of its programs into human testing sometime next year.

The company is leveraging stem cell research from company co-founders Kevin Eggan and Clifford Woolf, Harvard University professors whoby harvesting normal skin cells from ALS patients and turning them into cells such as the motor neurons that damages as the disease progresseshave created models with the same DNA and gene mutations as those patients in an effort to identify new therapeutics for known ALS genes.

Mutations in more than 25 human genes have been implicated in ALS, the company says, and its strategy is to systematically investigate treatments targeting specific disease-causing mechanisms in patient subgroups. Some of those genes are also believed to cause frontotemporal dementia, a common cause of dementia that QurAlis also plans to treat.

One program QurAlis is advancing is intended for patients whose neurons are damaged and killed by the overactivation of certain receptors for glutamate, a key neurotransmitter, in a process known as excitotoxicity.

The company is also working on a treatment intended to return the autophagy process, through which cells recycle unwanted or damage components, to normal functioning. To do so, QurAlis is looking to target the enzyme TBK1, which plays a key role.

Roet, in an interview, said the company views its strategy as analogous to that pursued by Bostons Vertex Pharmaceuticals (NASDAQ: VRTX), which has developed multiple drugs for forms of cystic fibrosis (CF) caused by certain mutations, and late last year received approval for a combination of those drugs for about 90 percent of all CF patients.

We have identified ALS as a disease that we think we understand now, at least for specific subgroups of patients, he said. We understand what is driving the disease and we are able to develop very specific therapies for those patients.

Eggan, Woolf, Roet, and Jonathan Fleming launched QurAlis just over two years ago with seed funding from investors including MP Healthcare Venture Management, the investment arm of Mitsubishi Tanabe Pharma; the investment arm of Amgen (NASDAQ: AMGN); and Alexandria Venture Investments. Mitsubishi Tanabe markets edaravone (Radicava), one of four FDA-approved treatments for ALS. The FDAs 2017 nod for the drug made it the only ALS therapy OKd in the past 20 years.

The Cambridge, MA-based company said the new capital, a Series A financing round, brings the total it has raised to $50.5 million. The investment was led by LS Polaris Innovation Fund, Mission BioCapital, Dutch firm Inkef Capital, and the Dementia Discovery Fund. New investors including Droia Ventures, which operates from Luxembourg and Belgium, Mitsui Global Investment, and Dolby Family Ventures also participated, as did earlier investors including Amgen, MP Healthcare, and Sanford Biosciences.

As part of the deal, LS Polariss Amy Schulman, Inkef Capitals Roel Bulthuis, Dementia Discovery Funds Jonathan Behr, and Droia Ventures Luc Dochez join Mission BioCapitals Johannes Fruehauf on the QurAlis board.

Earlier this year some of the same investors, including Amgen and Dolby Family Ventures, backed a Series A financing for EnClear Therapies, a spinout of QurAlis. That company raised $10 million to advance the development of a dialysis-like medical device designed to filter out harmful proteins in the cerebral spinal fluid of patients with neurodegenerative diseases.

Sarah de Crescenzo is an Xconomy editor based in San Diego. You can reach her at sdecrescenzo@xconomy.com.

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QurAlis Hauls In $42M to Move New ALS Therapies Into Human Testing - Xconomy

Skin Stem Cells Comments Off on QurAlis Hauls In $42M to Move New ALS Therapies Into Human Testing – Xconomy | May 13th, 2020

Novadip Biosciences Reports Positive Interim Analysis of Phase I/II Bone Non-Union Study with NVD003

Mont-Saint Guibert, Belgium, 13 May, 2020: Novadip Biosciences (Novadip or the Company), a clinical-stage biopharmaceutical company leveraging its proprietary tissue regeneration technology platform, today announces positive data from the interim analysis of its Phase I/IIa clinical trial for autologous NVD-003 in adults with non-healing fracture of the lower limb.

NVD-003 is a novel autologous cell-based osteogenic (bone healing/[bone forming]) product that has been generated from Novadips proprietary 3M tissue regeneration platform. This platform is aimed at healing damaged tissues by restoring their natural physiology and consists of a 3-dimensional, scaffold-free extracellular matrix (ECM), utilizing differentiated adipose-derived stem cells (ASCs) to restore the physiology of natural healing. NVD003 presents as a scaffold-free 3D implant to fill critical-size bone defects where healing is compromised.

This phase I/II study is investigating in five European centers the potential of NVD-003 to promote bone union in nine adults with a non-healing fracture of the lower limb. There was 100% manufacturing success for NVD-003 and grafting surgery was completed successfully in all patients without deviating from standard medical practice. To date, with a median of 12 months post-treatment, no NVD003 related safety signal has been reported. Further exploratory analysis performed on data from the first five patients to complete a six month follow up showed a strong positive trend in radiological healing with confirmed bone formation for all patients and radiologically confirmed union for three of the patients.

Prof. Gunnar Anderson (MD, PhD), Professor and Chairman Emeritus of the Department of Orthopedic Surgery at Rush University Medical Center, Chicago, Illinois and Chairman of the Scientific Advisory Board commented: The early results of this study are remarkable both clinically and for the patients and we look forward to replicating these in a larger group in the future. It is hugely encouraging that we may potentially have a future solution for these patients with unmet needs.

Dr. Denis Dufrane (MD, PhD), Chief Executive Officer, Chief Scientific Officer commented: We are encouraged by the data from this interim analysis, which demonstrates the potential of our tissue regeneration 3M3 platform to restore natural healing processes in patients with reputedly difficult to treat bone defects. We look forward to further progressing NVD-003s clinical program in bone non-union and in patients with other similar conditions with no effective treatment option and hope to provide full study results in 2025.

NVD-003 is also in clinical stage for congenital pseudarthrosis of the tibia (CPT), a rare and disabling pediatric condition with very limited treatment options and has demonstrated clinical proof-of-concept in case studies.

Novadips tissue regeneration platform drives several new classes of product candidates with an initial focus on autologous cell therapies for critical size tissue reconstruction. Allogeneic therapeutics are in development for prevalent and complex tissue defects for bone and skin tissue and exosomes/miRNA-based therapeutics are being developed for immediate (off-the-shelf) clinical use.

- End -

Notes to editors

Novadip Biosciences

Novadip Biosciences is a clinical stage biopharmaceutical company leveraging its unique 3D tissue regeneration technology platform to generate multiple product candidates to address hard and soft tissue reconstruction for patients who have limited or no treatment options. The companys proprietary 3M3 platform is a 3-dimensional, extracellular matrix that utilizes adipose-derived stem cells to deliver highly-specific growth factors and miRNAs to mimic the physiology of natural healing and creates a range of products that address specific challenges in tissue regeneration. Novadips initial focus is on critical size bone reconstruction. The company is also applying its 3M3 platform to develop truly novel off-the-shelf/allogeneic therapies to address more prevalent tissue defects and miRNA/exosome products for broader indications. For more information, visit http://www.novadip.com .

For further information, please contact:

Novadip Biosciences

Denis Dufrane

Chief Executive Officer, Chief Scientific Officer

+32 (10) 779 220

info@novadip.com

For media enquiries:

Consilium Strategic Communications

Chris Gardner, Matthew Neal, Angela Gray

+44 (0) 20 3709 5700

novadip@consilium-comms.com

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Novadip Biosciences Reports Positive Interim Analysis of Phase I/II Bone Non-Union Study with NVD003 - Yahoo Finance UK

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Carlos R. Bachier, MD

Chimeric antigen receptor (CAR) T-cell therapies quickly burst into the spotlight of hematology-oncology disease management because of their potential to illicit deep and durable responses from patients whose disease is relapsed or refractory to multiple previous lines of therapy. Relevant professional meetings and oncology publications exploded with research and news about CAR T cells, and this cellular therapy strategy is now being explored across hematologic and solid malignancies.

CAR T cells are a scientific revolution, Tania Jain, MBBS, assistant professor of oncology at Johns Hopkins University in Baltimore, Maryland, said in an interview with Targeted Therapies in Oncology (TTO). They have brought about a paradigm shift in terms of how were treating patients.

The 2 currently FDA-approved CAR T-cell therapies, axicabtagene ciloleucel (Yescarta) and tisagenlecleucel (Kymriah), are both indicated for the treatment of adult patients with relapsed or refractory large B-cell lymphoma; additionally, tisagenlecleucel is approved for patients up to 25 years with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL).1-3 With a second wave of approvals likely on the horizon for therapies such as lisocabtagene maraleucel (liso-cel) and idecabtagene vicleucel (bb2121), CAR T is gaining traction and will likely play an increasingly prominent role in the future treatment paradigm in oncology.

CAR T-cell therapy administration is largely limited to the inpatient setting at both academic institutions and large accredited cancer centers, making such treatments unavailable to most patients. Other challenges with this type of therapy include its potential to cause serious toxicities resulting in organ damage and death.4

David G. Maloney, MD, PhD

Due to the promising efficacy of these agents, investigators have been working toward viable solutions to bring CAR T-cell therapies to more patients by alleviating difficulties associated with therapy delivery and patient care.

CAR T-cell therapies, both those currently approved and the many being explored in late-phase clinical trials, are produced from autologous T cells obtained from the patient receiving therapy. This personalization has led to tremendous success, yet it is a large part of why CAR T-cell therapy use remains limited to a select group of patients.

Time is an important consideration for patients who have experienced multiple relapses and may be too weakened by numerous lines of prior therapy to wait several weeks for the CAR T-cell manufacturing process. The effects of previous treatments or the disease itself can also present challenges, as manypatients are rendered lymphopenic and may be unable to produce enough T cells for harvesting. Roadblocks may remain for patients who are not limited by these factors; manufacturing success and effectiveness of the CAR T-cell product can be negatively influenced by disease-related dysfunctions of patients T cells.4

A new option, off-the-shelf CAR T-cell products, may help solve these problems. These premade products are manufactured using allogeneic donor cells (instead of autologous cells from the patient), and they present immediate advantages to clinicians, such as immediate availability, opportunity for product standardization, and decreased cost.5

The advantages [include] being able to access the cellular therapy in real time, as opposed to autologous products that havetobe manufactured,Craig S. Sauter, MD, clinical director of the Adult Bone Marrow Transplant Service at Memorial Sloan Kettering Cancer Center in New York, New York, explained in an interview withTTO. This is particularly important for patients who are not responding to therapy, which is a current requirement for treatment with CAR T cells, he added.

Findings from a phase I trial (NCT01430390) in patients with relapsed or refractory B-cell malignancies showed that patients with non-Hodgkin lymphoma (NHL) experienced durable responses with an Epstein-Barr virusspecific cytotoxic lymphocyte CAR product derived from cells harvested from third-party donors (rather than from their more precisely matched stem cell donors). All 4 patients with NHL and a single patient with chronic lymphocytic leukemia, who were treated with third-party cells, remained disease free and alive at the time of analysis, with a median follow-up of over 2 years.6

The advantages [of this type of therapy are] that it eliminates the need for apheresis [and] shipping cellular products back and forth. [Instead, clinicians] have a pharmaceutical product on the shelf for access, Sauter, who was an author on the trial, said. Another notable product being investigated in clinical trials is UCART19, an allogeneic engineered anti CD19CAR T-cell product, which is being evaluated in the phase I CALM trial in adult patients with relapsed or refractory B-cell ALL (NCT02746952) and in the phase I PALL trial of pediatric patients with relapsed or refractory CD19-positive B-cell ALL (NCT02808442). Other off-the-shelf agents are described in theTABLE.5

Issues with inpatient CAR T-cell therapy administrationinclude high demands on health care resources and strain on patients and their families. Moving treatment to the outpatient setting has the potential to reduce this strain; however,clinicians taking over care of patients receiving CAR T-cell therapy must be prepared with the proper resources to identify and manage adverse events associated with therapy.

One of the most notable risks to patients receiving CAR T-cell therapy is cytokine release syndrome (CRS), a systemic inflammatory response that is characterized by increased serum levels of inflammatory cytokines, fever, hypotension, hypoxia, and organ dysfunction.4 [CAR T] can also lead to neurological events and can cause confusion and, in some patients, seizures,Carlos R. Bachier, MD, Director of Cellular Research at Sarah Cannon Cancer Center in Nashville, Tennessee, explained in an interview with TTO.

Regardless of the infusion setting, patients require close monitoring in the hours and days following therapy administration. A review byLucrecia Yez, PhD, MS, and colleagues stated that key criteria for treating patients in the outpatient setting include an educated caregiver and necessary infrastructure allowing for outpatient visits plus adequate emergency and intensive care unit (ICU) access. Patients followed as outpatients must be given twice-daily temperature checks for a minimum of 14 days following treatment and preferably extending up to 3 to 4 weeks following infusion. Anysigns of back pain, skin rash, dizziness, chills, shortness of breath, chest pain, tachycardia, or neurological events that may indicate neurotoxicity or signs of CRS must be reported immediately so treatment can begin as quickly as possible.7

Because of the risk of CRS and neurotoxicity, both FDA-approved agents are restricted under the Risk Evaluation and Mitigation Strategy, an FDA-mandated program that builds in caution for use of agents with serious safety concerns.8,9 Therefore, 2 doses of tocilizumab (Actemra), an interleukin (IL)-6 receptor antagonistthat was approved in 2017 for management of CRS associated with CAR T-cell therapy,1,4 should be on hand for each patient before the infusion of CAR T cells. Steroids have also demonstrated efficacy against CRS, but concernssurrounding CAR T-cell suppression with these agents have established them as a second-line choice after tociluzumab.9

Immune effector cellassociated neurotoxicity syndrome (ICANS) is a group of neurologic symptoms associated with treatments such as CAR T-cell therapy. Predisposing factors include younger age, higher tumor burden, high levels of pretreatment inflammation, and history of early or high-grade CRS. Treatments for complications of ICANS vary. Some centers may prescribe prophylactic antiepileptic medications, such as levetiracetam, to prevent seizures in patients with grade 2 or higher neurologic events. AntiIL-6 therapy can be considered in patients with concurrent CRS, but corticosteroids are the preferred regimen in those with neurotoxicity alone.9

In February of this year, the investigational CAR T-cell product liso-cel was granted priority review by the FDA for the treatment of adult patients with relapsed or refractory large B-cell lymphoma who had undergone at least 2 prior therapies.10 Investigators believe that liso-cel therapy may have a place in a broad range of patients and in the outpatient setting.11

It turns out liso-cel has a low incidence of [CRS and ICANS], and they occurred relatively late compared with other products, said Bachier. Because of this low incidence, the strategy was to deliver liso-cel in an outpatient setting.

The feasibility of liso-cel administration on an outpatient basiswas evaluatedby Bachier and colleagues, and the results were presented at the 2020 Transplantation & Cellular Therapy Meetings of the American Society for Transplantation and Cellular Therapy and the Center for International Blood & Marrow Transplant Research, held February 19 to 23, 2020, in Orlando, Florida.12

The authors analyzed data from 3 clinical trials of liso-cel, with a focus on the subset of participants who were treated as outpatients. The included trials were the phase I TRANSCEND-NHL-001 (NCT02631044) and phase II OUTREACH (NCT03744676) trials in patientswhohadundergone at least 2 lines of prior treatment, as well as the PILOT study (NCT03483103), which examined liso-cel as second-line therapy in patients who were ineligible for autologous hematopoietic stem cell transplant because of age, organ function, or ECOG performance score. All 3 studies allowed outpatienttreatment, with some patients receiving their therapy in the nonuniversity setting.

This clinical trial included sites that were not a part of a university but had experience treating patients for stem cell transplant, Bachier said. Some of these sites that participated were notyour traditional university centers that had traditionally been involved in the development of these therapies.

Much caution was required in order to maximize patient safety and treatment efficacy. The approach of doing CAR T-cell therapy, in general, in the outpatient setting requires a robust clinical ability of the centers, said coauthor David G. Maloney, MD, PhD, medical director of Cellular Immunotherapy at the Immunotherapy Integrated Research Center of Fred Hutchinson Cancer Research Center in Seattle, Washington, in an interview. We were able to get people safely to the hospital, and it was rare that you would have to do escalation of care when people were admitted. Most of the time, patients could bemanagedand wereout of the ICU, withrare exceptions. But again, you still have to have the wherewithal to get patients to the ICU pot entially for aggressive care if needed.

Results of the analysis of outpatient data from the 3 trials showed that rates of toxicity and response were similar to those previously reported for the entire patient cohort (both inpatients and outpatients) of the TRANSCEND-NHL-001 trial.

Based on these results, the indication is that you can deliver [liso-cel] in the outpatient setting and the outcomes are good compared with those treated in the inpatient setting, said Bachier. Aside from that, it also showed that liso-cel could be safely administered outside of university programs and in more community-based programs, most of them being aligned [with] or part of stem cell and bone marrow transplant programs.

When planning or setting up a CAR T-cell therapy outpatient program, investigators anticipate possible barriers to successfultreatment. The greatest barrier, according to Bachier, is access to physicians and staff who are knowledgeable and trained to manage toxicities related to CART-cell therapy. These therapies still should not, in my opinion, be delivered [by clinicians in] community centers that do not have the expertise to deliver the therapies safely, he said.

Maloney added that centers should be required to have the ability to triage patients 24/7 and allow for patients to be directly admitted to the hospital if needed. In the case of the analysis of outpatient data from the 3 liso-cel trials however, he said, We found that around 30% to 40% of patients did not actually ever require hospitalization, whichis quite interesting. Most of the 60% to 70% of patients who were hospitalized were admitted for fever, he added.

In addition, sites must gain accreditation and approval, Jain pointed out.

Every center that intends to do CAR T-cell therapy is first approved by each of the companies [that manufacturethese agents], Jain said. The centers also have to be approved by FACT [Foundation for the Accreditation of Cellular Therapy], which is the same organization that approves centers for allogeneic stem cell transplant. These are some of the largest things that a center needs to go through, which takes care of things like developing standard practices and other guidelines to make sure that these [therapies] are used safely and appropriately.

As investigators and oncologists explore the feasibility of moving CAR T-cell therapy into more settings, 2 questions arise: What settings have on this therapy?

What type of training and skills do clinicians need? Like other clinicians, Sauter has concerns about new allogeneic cellular therapies,andhe hopes future research will focus on mitigating these challenges. The concern would be that these are not autologous products and there is the risk of rejection from the host immune system, he said. Strategies to circumnavigate that risk are at the forefront of investigationin off-the-shelf CAR T cells.

The research is not stopping with CAR T-cell therapy,though. Were seeing a lot of new molecules coming in that will be challenging the roles of CAR T cells, [such as] specific antibodies, which may even work in cases of CAR T-cell failure, Maloney said. We are still learning how to make those more effective and safer.

References:

1. FDA approves tisagenlecleucel for B-cell ALL and tocilizumab for cytokine releasesyndrome.FDAwebsite.PublishedAugust30,2017.AccessedApril14, 2020. bit.ly/2RC4eQ8

2. FDA approves axicabtagene ciloleucel for large B-cell lymphoma. FDA website. Published October 18, 2017. Accessed April 14, 2020. bit.ly/2yYIQOp

3. FDA approves tisagenlecleucel for adults with relapsed or refractory large B-cell lymphoma. FDA website. Published May 1, 2018. Accessed April 14, 2020. bit.ly/34zPoi8

4. Rafiq S, Hackett CS, Brentjens RJ. Engineering strategies to overcome the current roadblocks in CAR T cell therapy. Nat Rev Clin Oncol. 2020;17(3):147167. doi: 10.1038/s41571-019-0297-y

5. DepilS,DuchateauP,GruppSA,MuftiG,PoirotL.Off-the-shelfallogeneic CAR T cells: development and challenges. Nat Rev Drug Discov. 2020;19(3):185199. doi: 10.1038/s41573-019-0051-2

6. Curran KJ, Sauter CS, Kernan CS, et al. Durable remission following off-theshelf chimeric antigen receptor (CAR) T-cells in patients with relapse/refractory (R/R) B-cell malignancies. Presented at: 2020 Transplantation & Cellular Therapy Meetings; February 19-23, 2020; Orlando, FL. Abstract 120. bit.ly/2ufDYCu

7. Yez L, Snchez-Escamilla M, Perales MA. CAR T cell toxicity: current managementandfuturedirections. Hemasphere.2019;3(2):e186.doi:10.1097/ HS9.0000000000000186

8. Risk evaluation and mitigation strategies | REMS. FDA website. Updated August 8, 2019. Accessed April 14, 2020. bit.ly/2ykhLVt

9. JainT,BarM,KansagraAJ,etal.UseofchimericantigenreceptorTcell therapy in clinical practice for relapsed/refractory aggressive B cell non-Hodgkin lymphoma: an expert panel opinion from the American Society for Transplantation and Cellular Therapy. Biol Blood Marrow Transplant. 2019;25(12):2305-2321. doi: 10.1016/j.bbmt.2019.08.015

10. U.S. Food and Drug Administration (FDA) accepts for Priority Review Bristol-Myers Squibbs Biologics License Application (BLA) for lisocabtagene maraleucel (liso-cel) for adult patients with relapsed or refractory large B-cell lymphoma. News release. Bristol-Myers Squibb; February 12, 2020. Accessed April 15, 2020. bit.ly/37ruQbs

11. Helwick C. Strong activity shown for lisocabtagene maraleucel CAR T-cell therapy in aggressive large B-cell lymphoma. ASCO Post website. Published February 25, 2020. Accessed April 15, 2020. bit.ly/3eoD0pT

12. Bachier CR, Palomba ML, Abramson JA, et al. Outpatient treatment with lisocabtagene maraleucel (liso-cel) in 3 ongoing clinical studies in relapsed/refractory (R/R) large B cell non-Hodgkin lymphoma (NHL), including second-line transplant noneligible (TNE) patients: TRANSCEND NHL 001, OUTREACH, and PILOT. Presented at: 2020 Transplantation & Cellular Therapy Meetings; February 19-23, Orlando, FL. Abstract 29. bit.ly/37I7DC9

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Protocol Management, Off-the-Shelf Therapies Help Bring CAR T Into More Settings - Targeted Oncology

Skin Stem Cells Comments Off on Protocol Management, Off-the-Shelf Therapies Help Bring CAR T Into More Settings – Targeted Oncology | May 13th, 2020

Families stuck at home during lockdown are being warned about a common but dangerous garden weed which can leave children - and adults - with burns and blisters.

Giant Hogweed has been dubbed Britain's most dangerous plant because of the horrific burns it inflicts on anyone who touches it - especially children - its set to thrive in the weeks ahead thanks to recent weather conditions.

The plant grows wild as well as in gardens and is becoming common the in the UK, but when it comes into contact with skin it causes a painful blistering rash.

The recent warm weather and plenty of rain after a mild winter has created the perfect conditions for this hazardous plant to thrive.

The hogweed looks relatively attractive and is part of the carrot family, but contains toxic chemicals.

Giant hogweed, or Heracleum mantegazzianum, is a weed which has dangerous effects on human health.

Growing up to five metres tall, its sap contains toxic chemicals which react with light when in contact with human skin, causing blistering within 48 hours.

Effectively it prevents the skin from protecting itself from sunlight, which can lead to very bad sunburn and scarring.

It's actually pretty and looks a bit like cow parsley. It's got green stem spotted with dark red which varies from 38 cm in diameter. Each dark red spot on the stem surrounds a hair, and large, coarse white hairs occur at the base of the leaf stalk.

It produces white flowers clustered in an umbrella-shaped head that is up to 80 cm in diameter across its flat top.

Colette Jones, Chairwoman of Friends of Close Park where Giant Hogweed was spotted, told The Bolton News : "Children are drawn to them because they grow so tall. They break them off to use them as sticks not realising how dangerous they are."

Exposure can result in blisters, long-lasting scars, and - if it comes in contact with eyes - blindness.

The blisters will form within 48 hours - scars can last for years.

It can also cause cause long-term sunlight sensitivity in people who touch it.

Black or purplish scars may be left on your skin for years after.

Medical professionals say you should cover the affected area, and wash it with soap and water.

The blisters heal very slowly and can develop into phytophotodermatitis, a type of skin rash which flares up in sunlight.

If you feel unwell or have a severe reaction you are advised to see a doctor.

Giant hogweed was among the foreign plants introduced to Britain in the 19th century as an ornamental plant, but it's now widespread throughout the British Isles.

It's invasive, which means that it chokes off other plants and can reduce wildlife in an area.

The plant is native to the Caucasus region and Central Asia.

The Wildlife and Countryside Act 1981 made it illegal to plant or cause giant hogweed to grow in the wild.

It is found in most of the UK, along footpaths and riverbanks though it also grows in places like parks, cemeteries and wasteland.

The sap of giant hogweed has chemicals which are toxic to humans and cause photosensitivity. The sap is phototoxic and can cause phytophotodermatitis.

When they touch skin, they effectively remove any protection against the sunlight causing severe skin inflammations.

Children have been hospitalised and suffered third-degree burns to their skin before.

The severe reaction to the plant is caused by the presence of linear derivates of furanocomarin in the plant's leaves, seeds, flowers, stems and roots.

The chemicals enter the cells' nucleus forming bonds with DNA and cause cells to die.

The RHS advises caution when removing the plant - cover arms and legs, and ideally wear a face mask when working on it.

Cut plant debris, contaminated clothing and tools are potentially hazardous too.

Wash any skin that comes in contact with the plant immediately.

The Wildlife and Countryside Act 1981 made it illegal to plant or cause giant hogweed to grow in the wild. Giant hogweed clearances are carried out to remove the plant.

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Warning over common garden weed that causes horrific burns - Cambridgeshire Live

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Repeated setbacks aside, little Capricor has suggested it has generated some long-term data to support its pursuit to garner approval for its stem cell therapy for Duchenne muscular dystrophy, although some of the data appeared to underwhelmed investors.

The data from the small, placebo-controlled mid-stage study, HOPE-2, tracked the effects of the companys stem cell therapy CAP-1002, which is designed to temper the inflammation associated with DMD, in 8 boys and young men who are in advanced stages of DMD. The remaining 12 enrolled patients received the placebo.

The main goal of the study was a measure that evaluates shoulder, arm and hand strength in patients who are generally non-ambulant (performance of the upper limb (PUL) 2.0), as suggested by the FDA, Capricor said. It is one of several ways Capricor quantified skeletal muscle improvement in the trial.

The intravenous infusion of CAP-1002, given every 3 months, induced a statistically meaningful improvement of 2.4 points (p=0.05) versus the placebo group, in which patient declines were consistent with natural history data. However, on another measure of upper limb function, the trend was in favor of the Capricor drug, but did not hit statistical significance.

The companys shares $CAPR were down nearly 13% to $6.89 in morning trading.

Click on the image to see the full-sized version

Meanwhile, there were also some encouraging data on cardiac function the genetic condition is characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles.

As reflected above, CAP-1002 elicited an improvement across different measures of cardiac function, although the effect was not always statistically significant. In particular, the drug also caused a reduction in the levels of the biomarker CK-MB, an enzyme that is only released when there is cardiac muscle cell damage.

Armed with these data and an RMAT and orphan drug designation from the FDA, Capricor is now hoping to eke out a plan with the FDA for marketing approval.

LA-based Capricor initially set out to test the potential of technology that Eduardo Marbn, CEO Linda Marbns husband, developed at Johns Hopkins. But repeated setbacks clobbered the company, which in 2014 traded north of $14 a share. In 2017, J&J walked away from a collaboration on a stem cell therapy for damaged hearts after it flopped in the clinic.

In late 2018, the company voluntarily halted a DMD clinical trial, following a severe allergic reaction that occurred during infusion. In February 2019, the company said it is exploring strategic alternatives for one or more of its products and cutting 21 jobs to keep financially afloat, but had resumed dosing in its DMD trial.

The first batch of positive data on CAP-1002, which consists of progenitor cells derived from donor hearts and is designed to exude exosomes that initiate muscle repair by suppressing inflammation and driving immunomodulation, came last July when the company announced the drug had generated a positive effect at the interim analysis juncture of HOPE-2. Capricor is now working on to flexing its therapeutic muscle with CAP-1002 to fight the Covid-19 pandemic.

DMD is a rare muscle-wasting disease caused by the absence of dystrophin, a protein that helps keep muscle cells intact. It disproportionately affects boys and affects roughly 6,000 in the United States.

Patients are essentially treated with steroids. Sarepta Therapeutics now has two exon-skipping drugs designed to treat certain subsets of the disease, although the magnitude of their effect is controversial given that approvals were not based on placebo-controlled data. Meanwhile, Sarepta and others are also pursuing one-time cures in the form of gene therapies to replace the missing dystrophin gene in patients.

Social: Linda Marbn, Capricor CEO (Twitter)

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One year on, Capricor's stem cell therapy appears to help DMD patients in small study, but investors balk at the data - Endpoints News

Cardiac Stem Cells Comments Off on One year on, Capricor’s stem cell therapy appears to help DMD patients in small study, but investors balk at the data – Endpoints News | May 13th, 2020

The Cell and Gene Therapy Catapult (CGT Catapult) and Kyoto, Japan-based CiRA Foundation are launching a new collaborative research project focused on induced pluripotent stem (iPS) cell characterisation.

With the move, the groups are hoping to further the application of iPS cell technologies for the manufacture of regenerative medicine products.

The potential of distinct iPS cell lines for differentiation into specific cell types is usually biased towards some cell line-specificity which, the parties note, is very difficult to predict. As such, in order to select an appropriate iPS cell line for clinical trials it is necessary to differentiate several candidate cell lines, which is time-consuming.

CGT Catapult and CiRA plan to explore novel methods of evaluating cell differentiation and aim to establish reliable tests to predict the potential of iPS cell to differentiation bias, a capability that would help to advance the use of iPS cells for regenerative medicine products.

We are honoured to collaborate with CiRA Foundation, an organisation with world-leading capabilities in iPS cell technology, and to be the first group to utilise CiRAs innovative iPS cell lines outside of Japan, said CGT's chief executive Matthew Durdy

This is a truly exciting project to help further the application and manufacture of iPS cells into cell therapies. We look forward to progressing this promising research together, which has potential benefits for the global advanced therapies industry.

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Cell and Gene Therapy Catapult links with Japan's CiRA Foundation - PharmaTimes

IPS Cell Therapy Comments Off on Cell and Gene Therapy Catapult links with Japan’s CiRA Foundation – PharmaTimes | May 13th, 2020

Washington D.C: Already known for their shape-shifting abilities, stem cells can now add "death-defying" to their list of remarkable qualities, suggests a novel study.

The new study shows how stem cells, which can contribute to creating many parts of the body, not just one organ or body part, are able to postpone their own death in order to respond to an injury that needs their attention.

The study was done in planarians, which are tiny worms used as model organisms to study regeneration because of their ability to recover from any injury using stem cells.

"Planarian stem cells, even when challenged and under a lot of duress, will still respond to an injury by delaying death," said Divya Shiroor, first author and a graduate student in Dr Carolyn Adler's lab, in the College of Veterinary Medicine.

This could have important implications for cancer research and therapies, particularly when examining chemotherapy and surgery options for patients.

"By understanding how injury prompts planarian stem cells to withstand radiation. We hope to identify genes that, if shared with mammals, could perhaps help hone existing therapies," Shiroor said.

Planarians are commonly used in basic research because of their similarities to humans. Like humans, planarians have stem cells, similar organs and similar genes, but are much more adept at responding to injury, thanks to their higher volume of stem cells and lack of a developed immune system, which in humans complicates the healing process.

"This really simplifies the process of understanding the effects of both injury and radiation on stem cells, and allows us to study it directly without being hampered by parallel processes integral to wound healing, such as inflammation, that get simultaneously triggered in mammals," Shiroor said.

By uncovering the mechanisms that govern stem cells after wounding in a system like planarians, researchers could also apply this knowledge when engineering stem cells to respond similarly in the human body.

Labs have many ways to understand how planarians use stem cells to successfully recover and regenerate, but the Adler lab's combination of radiation and injury to identify a novel stem cell response is unique. The researchers plan on digging deeper to understand how the stressed stem cells know that there is an injury and what role other cells may play in their response.

"We have identified a key gene that is required for stem cell persistence after radiation and injury. and we plan on using this as a stepping stone for further exploration," Shiroor said.

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Researchers discover stem cells' 'death-defying' quality that aids healing - ETHealthworld.com

Skin Stem Cells Comments Off on Researchers discover stem cells’ ‘death-defying’ quality that aids healing – ETHealthworld.com | May 12th, 2020

A decade ago, scientists won the nobel prize for connecting the dots between telomeres and aging. Telomeres are the protective caps on the ends of the strands of DNA called chromosomes. Telomeres shorten every time a cell divides. When they become very short, they trigger cell crisis and cell death.

Since this discovery was made, researchers have found new insights into telomeres and their connection to wrinkled skin, as well as how their length might be extended.

Scientists have uncovered new information on telomeres that could help combat the effects of inflammation and aging. Researchers from the University of Pittsburg recently discovered how oxidative stress plays a critical in the link between telomeres and cancer. I dont want to blind you with science (telomeres are a complex area), but every breakthrough into telomeres will ultimately equal a breakthrough in understanding how we age and how we can keep cells including our skin cells healthy.

Telomeres are composed of repeated sequences of DNA. The results from this study suggest that the mechanism by which oxidative stress accelerates telomere shortening is by damaging the DNA precursor molecules, not the telomere itself. This will have a big impact on appreciating how to manage oxidative stress to prevent aging and diseases such as cancer.

At Stanford University School of Medicine, scientists claim they can now lengthen teleomeres. Skin cells with telomeres lengthened by the procedure were able to divide up to 40 more times than untreated cells (source).

However, longer teleomeres is not necessarily associated with a longer life (source). Indeed, those naysayers say that good diet and an exercise regimen will do that. Yet, it does seem that preventing further shortening of the telomeres could be beneficial, especially for aging skin. Telomeres are likened to the tips of shoelaces that stop them unraveling and so you really want to be thinking about how to stabilize your telomeres. Happily, advances in skin care can help you out. There are three approaches to consider: special ingredients that target telomeres, ingredients that prevent oxidative damage (known, of course, as antioxidants) and stem cells.

Target your telomeres

One very interesting ingredient is called astragalus, and although it is rare, we do have a couple of Truth In Aging finds with it. But first, what is it and how does it work? In 2008, a UCLA AIDS Institute study found that a chemical they called TAT2 from the astragalus root, which is frequently used in Chinese herbal therapy, can prevent or slow the progressive shortening of telomeres. It can be found in Prana Reishi Mushroom Shield ($42 in the shop) and ExPrtise Effective Anti-Aging Face Serum ($120 in the shop).

Another ingredient to look for is treprenone, also marketed under the name of Renovage. Its promise is to stabilize telomeres, so at the very least they won't shorten. Maintaining telomere length extends the Hayflick Limit (the rate at which cells turn over before conking out completely) by one third. Youll find treprenone (Renovage) in Your Best Face Correct ($150 in the shop) Your Best Face Boost ($65 in the shop.

Amp up with antioxidants

The research is clear: Preventing oxidative damage is the job of an antioxidant. Free radicals are charged chemical particles of oxygen that enter into destructive chemical bonds with organic substances such as proteins, as explained by Gerald Imber, MD. Antioxidants limit the production of free radicals and therefore help prevent oxidative stress. There are many sources in plants, and vitamins are also antioxidants.

There are 33 antioxidants in Skin 2 Skin Aging Intervention Cream ($73 in the shop), including the universal antioxidant alpha lipoic acid. The powerful antioxidants in Your Best Face Rescue ($145 in the shop) include spin trap, an advanced form of vitamin C and EGCG. Bee venom has some 18 active compounds include antioxidants, peptides with powerful anti-inflammatory actions, and enzymes. It is the key ingredient in LaCrme Beaut Luxurious Bee Venom Rich Face Treatment Cream ($202 in the shop).

Look for plant stem cells

Plant stem cells never age. British scientists found that plant stem cells were much more sensitive to DNA damage than other cells. Once they sense damage, they trigger death of these cells before it spreads and causes more. In addition, they have the ability to stimulate cell renewal and replace specific cells in need of repair.

Ao Skincare Raw Nourish AM Treatment ($65 in the shop) has the powerful antioxidant astaxanthin and plant stem cells.

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New Study Suggests the Consequences of Oxidative Stress on Telomeres - Truth In Aging

Skin Stem Cells Comments Off on New Study Suggests the Consequences of Oxidative Stress on Telomeres – Truth In Aging | May 12th, 2020

TEL AVIV, Israel, May 11, 2020 /PRNewswire/ -- Cellect Biotechnology Ltd. (NASDAQ: APOP), a developer of innovative technology which enables the functional selection of stem cells, today announced the publication of an article in Bone Marrow Transplantation, a peer-reviewed medical journal )member of the Nature publishing house) covering transplantation of bone marrow in humans and published monthly by the prestigious Nature Research, entitled 'Ex-vivo FAS-ligand to Improve Allograft Safety'. The article is co-authored by researchers at Cellect and its academic partners.

The paper highlights the pre-clinical research and demonstrates that engraftment is robust following transplantation of treated graft, and the graft retains its immune reconstitution and anti-leukemic effects. The Company has initiated a Phase 1/2 study in adults undergoing stem cell transplant for the treatment of hematological malignancies. The primary endpoint of the study is to evaluate the overall incidence, frequency, and severity of adverse events potentially related to ApoGraft at 180-days post-transplant. All patients transplanted through present time using the ApoGraft process were engrafted and time to engraftment was similar to the standard of care. To date, there have not been any safety and tolerability concerns during the study and patient enrollment is continuing. Both, the principal investigator (PI) and independent data safety monitoring board (DSMB) agree that no serious adverse events (SAEs) reported during the course of the study were related to the ApoGraft process.

The data from the pre-clinical research, and published in this paper, was included in the Company's Investigational New Drug (IND) application, which was approved by the U.S. Food and Drug Administration in late 2019. The Company has received all the necessary approvals to initiate the trial with its academic partner, Washington University, and plans to begin patient recruitment once the COVID-19 pandemic is mitigated and clinics can resume normal practices.

About Cellect Biotechnology Ltd.

Cellect Biotechnology (NASDAQ: APOP) has developed a breakthrough technology, for the selection of stem cells from any given tissue, that aims to improve a variety of stem cell-based therapies.

The Company's technology is expected to provide researchers, clinical community and pharma companies with the tools to rapidly isolate stem cells in quantity and quality allowing stem cell-based treatments and procedures in a wide variety of applications in regenerative medicine. The Company's current clinical trial is aimed at bone marrow transplantations in cancer treatment.

Forward Looking Statements

This press release contains forward-looking statements about the Company's expectations, beliefs and intentions. Forward-looking statements can be identified by the use of forward-looking words such as "believe", "expect", "intend", "plan", "may", "should", "could", "might", "seek", "target", "will", "project", "forecast", "continue" or "anticipate" or their negatives or variations of these words or other comparable words or by the fact that these statements do not relate strictly to historical matters. For example, forward-looking statements are used in this press release when we discuss Cellect's expectations regarding timing of the commencement of its planned U.S. clinical trial and its plan to reduce operating costs. These forward-looking statements and their implications are based on the current expectations of the management of the Company only and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. In addition, historical results or conclusions from scientific research and clinical studies do not guarantee that future results would suggest similar conclusions or that historical results referred to herein would be interpreted similarly in light of additional research or otherwise. The following factors, among others, could cause actual results to differ materially from those described in the forward-looking statements: the Company's history of losses and needs for additional capital to fund its operations and its inability to obtain additional capital on acceptable terms, or at all; the Company's ability to continue as a going concern; uncertainties of cash flows and inability to meet working capital needs; the Company's ability to obtain regulatory approvals; the Company's ability to obtain favorable pre-clinical and clinical trial results; the Company's technology may not be validated and its methods may not be accepted by the scientific community; difficulties enrolling patients in the Company's clinical trials; the ability to timely source adequate supply of FasL; risks resulting from unforeseen side effects; the Company's ability to establish and maintain strategic partnerships and other corporate collaborations; the scope of protection the Company is able to establish and maintain for intellectual property rights and its ability to operate its business without infringing the intellectual property rights of others; competitive companies, technologies and the Company's industry; unforeseen scientific difficulties may develop with the Company's technology; the Company's ability to retain or attract key employees whose knowledge is essential to the development of its products; and the Company's ability to pursue any strategic transaction or that any transaction, if pursued, will be completed. Any forward-looking statement in this press release speaks only as of the date of this press release. The Company undertakes no obligation to publicly update or review any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws. More detailed information about the risks and uncertainties affecting the Company is contained under the heading "Risk Factors" in Cellect Biotechnology Ltd.'s Annual Report on Form 20-F for the fiscal year ended December 31, 2019 filed with the U.S. Securities and Exchange Commission, or SEC, which is available on the SEC's website, http://www.sec.gov, and in the Company's periodic filings with the SEC.

Contact

Cellect Biotechnology Ltd.Eyal Leibovitz, Chief Financial Officerwww.cellect.co+972-9-974-1444

Or

EVC Group LLCMichael Polyviou+732-933-2754mpolyviou@evcgroup.com

View original content:http://www.prnewswire.com/news-releases/cellect-biotechnology-announces-positive-data-demonstrating-robust-engraftment-using-apograft-was-featured-in-bone-marrow-transplantation-primary-data-points-were-submitted-to-the-fda-during-investigational-new-drug-approval-proc-301056409.html

SOURCE Cellect Biotechnology Ltd.

Company Codes: NASDAQ-SMALL:APOP, TelAviv:APOP

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Cellect Biotechnology Announces Positive Data Demonstrating Robust Engraftment Using ApoGraft was Featured in Bone Marrow Transplantation; Primary...

Bone Marrow Stem Cells Comments Off on Cellect Biotechnology Announces Positive Data Demonstrating Robust Engraftment Using ApoGraft was Featured in Bone Marrow Transplantation; Primary… | May 12th, 2020

The healthcare industry has been focusing on excessive research and development in the last couple of decades to ensure that the need to address issues related to the availability of drugs and treatments for certain chronic diseases is effectively met. Healthcare researchers and scientists at the Li Ka Shing Faculty of Medicine of the Hong Kong University have successfully demonstrated the utilization of human induced pluripotent stem cells or hiPSCs from the skin cells of the patient for testing therapeutic drugs.

The success of this research suggests that scientists have crossed one more hurdle towards using stem cells in precision medicine for the treatment of patients suffering from sporadic hereditary diseases. iPSCs are the new generation approach towards the prevention and treatment of diseases that takes into account patients on an individual basis considering their genetic makeup, lifestyle, and environment. Along with the capacity to transform into different body cell types and same genetic composition of the donors, hiPSCs have surfaced as a promising cell source to screen and test drugs.

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In the present research, hiPSC was synthesized from patients suffering from a rare form of hereditary cardiomyopathy owing to the mutations in Lamin A/C related cardiomyopathy in their distinct families. The affected individuals suffer from sudden death, stroke, and heart failure at a very young age. As on date, there is no exact treatment available for this condition.

This team in Hong Kong tested a drug named PTC124 to suppress specific genetic mutations in other genetic diseases into the iPSC transformed heart muscle cells. While this technology is being considered as a breakthrough in clinical stem cell research, the team at Hong Kong University is collaborating with drug companies regarding its clinical application.

The unique properties of iPS cells provides extensive potential to several biopharmaceutical applications. iPSCs are also used in toxicology testing, high throughput, disease modeling, and target identification. This type of stem cell has the potential to transform drug discovery by offering physiologically relevant cells for tool discovery, compound identification, and target validation.

A new report by Persistence Market Research (PMR) states that the globalinduced pluripotent stem or iPS cell marketis expected to witness a strongCAGR of 7.0%from 2018 to 2026. In 2017, the market was worthUS$ 1,254.0 Mnand is expected to reachUS$ 2,299.5 Mnby the end of the forecast period in 2026.

Access Full Report @https://www.persistencemarketresearch.com/checkout/17968

Customization to be the Key Focus of Market Players

Due to the evolving needs of the research community, the demand for specialized cell lines have increased to a certain point where most vendors offering these products cannot depend solely on sales from catalog products. The quality of the products and lead time can determine the choices while requesting custom solutions at the same time. Companies usually focus on establishing a strong distribution network for enabling products to reach customers from the manufacturing units in a short time period.

Entry of Multiple Small Players to be Witnessed in the Coming Years

Several leading players have their presence in the global market; however, many specialized products and services are provided by small and regional vendors. By targeting their marketing strategies towards research institutes and small biotechnology companies, these new players have swiftly established their presence in the market.

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PMR Sales Revenue in the Induced Pluripotent Stem Cells Market to Witness Growth at Robust CAGR 7% During 2026 - Cole of Duty

Skin Stem Cells Comments Off on PMR Sales Revenue in the Induced Pluripotent Stem Cells Market to Witness Growth at Robust CAGR 7% During 2026 – Cole of Duty | May 11th, 2020

Megan Dunn takes a long look at the art in the Auckland Hospital collection and finds out that yes, there is art, even in intensive care but the price of seeing it is everything.

I got the pamphlet in the mail back in January. It says after someone close to you dies it is natural to:

The day the pamphlet arrived I splayed it open and looked at the photo inside: a network of hands holding other hands. Cropped close. Just linked hands held in support of one another. Beneath the photo: we grieve as deeply as we love.

I am unable to return to normal services.

Painting from collection donated by the Art Komiti of Aucklands Paremoremo Prison

I had noticed art in waiting rooms before, not art with a capital A, not the kind of art that an art writer such as myself would bother to write about, because art like so much else in our society has its hierarchy. Contemporary art is a high-stakes game; who you write about is as important as what you say. Who cares about the art in the waiting room? Lets abbreviate: who cares?

Me. Ive had cause to think about it because last year Mums cancer returned from outer space. Shed been in remission for 17 years, which might be a national record for multiple myeloma, a cancer of the blood. Ive never really understood what her cancer is, just that she beat it first time round, and, last July, the cancer cell count was up. Then she started chemo.

Im not coping, Im just not coping, Mum called after the first round, in tears.

Have you eaten anything?

No. I dont feel like eating.

It was the dex. The specialist, a blonde in a miniskirt and long black boots, said, Thatll be the dex. Short for dexamethasone, a steroid. Another bitter pill to swallow. At that point Mum had to take up to 10 pills a day. I looked at the specialist. I was angry. Mums not coping. I had flown to Auckland to join Mum for her next haematology appointment.

But what art could I prescribe? Optimism matters, but art isnt always soothing or kind.

Im lucky I have a chance of getting better, Mum said. True. Her prognosis was good.

We went upstairs for her second round of chemo. The nurse in blue gown, gloves and a shower cap wheeled it around in a mauve container, hooked it on a drip, and inserted the port into Mums vein.

Other patients stretched out on beds like La-Z-Boys, skin drained. They too were waiting, but looked like they didnt have a chance of getting better.

I stared into the little office next to Mums chair. Harried papers on the desk; a PC lit up like a bright face, ready to supply details, cheer, whatever counts most. On the wall, an original painting of a phutukawa tree. I call it original but the concept wasnt. The sea peeked out of the background, the sky bluer than the sea. The oils had been used with little mixing, but I didnt mind that painting of the beach in summer rudely lit up by the phutukawa tree, the needles burst open in the leaves, red as fireworks.

The nurses are very nice here, Mum said.

Niceness counts like stem cells it has a tally.

That was quick, I said.

It really doesnt take long.

I wondered how long the painting took the artist. Guessing from the paint application, not long, but longer than it takes a dose of chemo to run into your veins.

I wanted to ask the nurse: who bought that painting? Who is the artist? Who put that there? But I was too shy. Why stop this real raw moment, for an intellectual aside, a detour into the art we look at it when were waiting to get better or to see how much worse it can get?

The painting of the phutukawa tree in summer yields its bouquet. Just be.

***

Mum and I got lost en route to blood work. The hospital has an art collection in its corridors that must be maintained by someone, however irregularly. We passed a Claudia Pond Eyley print black lines of plants, bright colours, a Pacific infusion abandoned along a corridor. A trio of photographs of flowers. Where are we? Is it this the way? They need to give better directions. Then, in a gangway, a pair of large, textured, brown abstracts, neither good nor bad, just out of time, like photos of your parents when they were young, in the fashions of the day. Those sideburns. What chops! Mums perm. Dads flares. A big Pat Hanly painting called Vacation was by the escalator. I meant to come back, retrace my steps, and find all this art again. And I did come back, but by then everything had changed.

I dont want to write what comes next, because I dont want the wait to be over. Waiting is a comfort in its own sad way. Just wait and see.

The sea. Waves lap in and out. I see it whenever I attend my doctors surgery above Courtenay Place. Piha is by the Korean-born photographer Jae Hoon Lee. Its moved around the surgery over the years. Now its in the waiting room above a line of plastic chairs. Whoever bought it must have thought it was soothing and anaesthetic, a balm for a worried soul like mine.

What do you want to see when you go for a smear test, or hold a baby that wont go the fuck to sleep, or wait with your partner for test results, a prognosis, a new vaccine?

I thought Ill wait to write about the art at the doctors surgery or the hospital. Or the dentist, though I have not been to the dentist in ages, no cash, so Ill wait. How much art is in your life? How many fillings? Do you have art at home? If so, what? Where did you get it? At what price?

In the waiting room, I dont blame you if you dont want to look at something difficult and ugly or think about something hard. If youre just after a good view, Arent we all?

At my doctors surgery Pihahangs near a plastic container for pamphlets, ruffled in waves.

For every problem there is a pamphlet.

The pamphlet that arrived in the mail in January is titled Department of Critical Care Medicine Bereavement Follow-up Service.

Piha in situ at Courtenay Medical, Wellington

The waiting room isnt just literal, but it is literal too. I know because the last time I was there I got a hot chocolate from the machine. Warm, syrupy. No art, just a TV on the wall.

At the haematology department, I watched a dad sitting with his young daughter on his lap. Her mind looked far away. She waited with the patience of one who has waited before. Then her mum appeared in a turban. I looked at this young mother, I had no idea what cancer of the blood she had, but I really hoped she had a chance of getting better.

Two in 100 people die, Mum said. Back in December she was waiting for her stem-cell transplant. She would be in hospital for two to three weeks. The transplant would take her immunity to zero that was where the risk of infection crept in but then the white cells would ingraft and her count would go back up. Shed signed the forms, accepting the 2% chance she would die. If theres not a bed on Thursday, it will be next week.

I wondered: Will there be any art in her room? And if so, what will it say?

The phutukawa painting in the office of the haematology department says shush shush, that lulling noise of waves from the beach. Dont worry, relax. Its OK. Look at the view. But the Jae Hoon Lee photograph in my doctors surgery says to me your hurt is timeless, the sea will exist whether we do or not, release your grip, whatever happens next will be surgically safe, emotions are never still, time is an inlet, the sea runs in and out.

***

I always knew that waiting was part of the problem. What if you wait too long? Then you cant get around to what you were going to say because you are:

***

Outside Ward 82, ICU (acute surgery). On the intercom, the number one has been buzzed off, pushed too many times. All other numbers present and correct. My mother is in bed 17, I tell the intercom, after Ive pressed the number one that isnt there and waited. Then I walked through vaguely yellow corridors lined with three framed prints, each composition a rectangle yellow, blue, orange lined with holes down the middle. They reminded me of paeans to the common household sponge. I stop at the hand-gel pump and sanitised. The art at the hospital is sanitised too but Im beyond caring. Too much caring and you move though it and pass out on the other side somewhere in the vicinity of bed 17.

At the end of the corridor past the hand gel, the Chen family have donated a small square print, red with black scribble, in honour of the ICU team. I clocked the gold engraved plaque, their appreciation registered on the wall. The painting not unlike the size of a fire alarm, but there is no glass to break open, the call has already been raised.

I got the call from the registrar on December 22 to say that Mums stem-cell plant was not going as expected and she had been admitted to ICU.

Ive been thinking about what I will do when I get better, Mum said, the night I arrived. She sat propped up in bed, on oxygen. Her face flushed, swollen, but superficially OK.

I sat next to her bed. Oh yes, what do you think?

Im going to come to your book launch.

I smiled. My book of personal essays about art and life, already way behind schedule.

What else? I asked.

I might meet someone new. I might travel.

Where would you like to go? I asked.

She paused. Maybe Africa, she said. I could go on a safari.

I nodded. Mum found it hard to walk up the small pronounced hill to our house in Wellington, sweated easily, mopped her forehead. Then shed wait, slightly panting, for her breath to right itself again, restart.

That night, I slept in the hospital. They say you can stay in my room at the Motutapu Ward, Mum said. There was no art in her room, but a wall-to-ceiling poster of a forest, kauri trees, dense, shady, green.

In the morning I got buzzed back into ICU. Mums arms and legs, twitched, calling out; face red, body puffed up; trying to unpick the PICC line from her arm, in among the beeping and the rising heart monitor, the oxygen exhaling. She doesnt recognise me, is raving to the charge nurse, who held her hand and looked at me and explained, Im just going some gentle reorientation work.

I must have seemed stunned.

Do you want to speak outside the room? the charge nurse asked.

I nodded. In the room next door to bed 17, I wailed, What is happening? Oh, what is happening? The charge nurse held me in place and comforted me, when there was no comfort to give.

Next the intensivist arrived in her blue scrubs, removed her surgical mask, wiped her hands with gel, introduced herself as Kylie. She wanted a family meeting.

The whnau room: the painting was donated by the Art Komiti of Aucklands Paremoremo Prison

The whnau room at ICU contains another Claudia Pond Eyley print on one wall and a multi-panel painting of a New Zealand landscape on the other. The mountains, a lake, smooth and even and still. A perennial view of nature, so calm, so undisturbing to see. I cant blame the hospital for containing so many paintings of the view sky, sand, sea, soothing, stretching, somehow infinite. A vista of comfort, comprehended.

I sat down on one side of the big meeting table, Kylie on the other. What is your understanding of your mothers condition? she asked. I rattled off the facts while an accompanying nurse took notes.

Mum had come in for a stem-cell transplant. She had caught pneumonia at a point in the process when she had no natural immunity. She had developed delirium. Her pre-existing heart condition had been set off: arterial fibrillation. She needed oxygen support to breathe.

This is what the 2% of risk looks like? I asked.

Yes. Kylie explained that they couldnt provide sedatives like morphine as that would risk compromising Mums breathing further. They could put Mum on a ventilator if she deteriorated but that would not be a good sign and would come with an extra level of risk. What helped patients with delirium was familiar voices.

I asked her, Are you holding anything back?

Just that this is very life threatening. Her eyes, an expression, I can now only call grave.

Well, it seemed apt. I liked Kylie. The intensivist understood how much intensity was required.

The job title intensivist seemed funny to me because in high school I was always told I was too intense. We have to find a happy medium, a teenage girlfriend once told me. So I got into art because it seemed like a place where intensity went and didnt have to turn down a notch. But art isnt the only place for intensity. At the intensive care unit (acute) on the eighth floor of Auckland Hospital the intensivists are also at work.

All day the nurse at bed 17 and I spooned jelly or orange juice into her mouth, helped her not frantically unpick her PICC line, I just want to go for a walk! No, no dear, you cant get up. Mum, you just need to rest. Rest. A familiar voice on replay. Youll feel so much better if you rest.

In the night I noticed the print of a lone bugle boy down some New Orleans alley, presumably playing jazz. No plaque. Who bought it? What family? Who was lost?

In ICU the beeping was persistent, insistent. The constant sound of inhaling, exhaling. Poke your tongue out. Ahhhh. Good girl.

Were concerned about the delirium. In ICU, delirium can be intensified, especially in older people, by the strange sounds, lights, faces.

I forgot the ducks! I keep meaning to mention the duck painting, a watercolour, a good one too, of some ducks paddling around their pond, giving no quacks in ICU. The duck pond was donated by another family, with a brass plaque. I should have jotted down the name.

The eyeball is so moveable, up and over, it can even see things that arent there. Mum said her family prayer, over and over, eyes roving. We consecrate to Thee, O Jesus of Love My aunt clutched her hands and said the prayer with her.

One night in ICU I passed a large rectangular collage of brightly coloured red and pink buttons like some budget Damien Hirst pill painting. Never passed it again. Beep, beep.

Her oxygen levels are saturating nicely.

How much?

Fifty-five percent, then down to 48%.

Dont obsess about the blood count.

Look at the patient. Look at the face.

The face her not her.

My daughter in the car on the way to the hospital singing: Pop bang crack goes the Christmas cracker, pop bang, crack goes the Christmas cracker, we will pull it off POP.

Eyes popped, snapped.

Shes been restless.

You need to sleep.

Sleep.

I stop and start, keep typing the next line, then deleting it. I dont want to get to the end of this. I dont want to remember all the family meetings in the whnau room waiting. I dont want to chart the order of those disordered days.

What was I going to say about art?

***

I spent the first weekend after Mums stem-cell transplant in her room at the Motutapu Ward. Motutapu means sacred or sanctuary. Shed just had the big dose of chemo and was quiet, but not yet unwell. My aunt was going to join her on Monday Mums 69th birthday for what would be the worst week of the process. I sat on the bed, Mum on the La-Z-Boy. We said not a whole lot.

At one point, I think I can manage a walk.

We passed the two nurses stations. En route I pointed out the art. We stopped by a faded print of sunflowers, beneath glass, but the artist was no Van Gogh. The first time Id passed the sunflowers, I hadnt rated them at all. But that was before Id read that they were by Chris Corlett, a 17-year-old who died of acute lymphoblastic leukaemia 20 years ago. Sunflowers of Hope does look like it was painted by a teenage boy. Theres something gnarly about them. Large and abundant. Full of life. Except for the leaves Chris paid special attention to the leaves stippled with decay, a bit heavy metal.

Sunflowers of Hope, Chris Cortlett, Auckland Hospital, 2019.

We stood reading the accompanying framed text about the foundation Chris had started to build up a database of 100,000 bone-marrow donors.

Courage, charisma, strength of character, sincerity whatever it is that makes some people inspirational and very special, Chris Corlett had it.

Another Claudia Pond Eyley print near the kitchen and on the door a sign that read: Dont use if youre come from a red room. I was confused by it when Id made Mum a cup of tea earlier and had to ask the nurse, Is this a red room? (Mums room was not a red room.)

I felt the light weight of Mums hand on my arm as we looped around to reception. I showed her my favourite painting, tucked in a corner. Its colours were so bold that from the corner of my eye I first suspected Matisse. Then was embarrassed when I realised the artist is Harriet, aged six, who donated Flowers for the Leukaemia Ward in memory of her father, Ned.

Harriet may not be Matisse but for a six-year-old her vase of flowers is a masterpiece of colour and compression.

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Art in the waiting room - The Spinoff

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World Thalassemia Day is an annual observance day that occurs on May 8th. It is a world-wide campaign to raise awareness about thalassemia and its symptoms. This is done to help the patients living with this genetic disorder. World Thalassemia Day commemorates thalassemia victims and also aims at making thalassemia patients aware about the significance of medical consultation before marriage. This global observance day also tries to debunk myths and misconceptions surrounding the disease. The theme of World Thalassemia Day 2020 is Begin thalassemia prevention from young age, blood test before marriage will make the future generation safe. On this day, here we tell you all about the disease. Also Read - World Thalassemia Day 2019: How to Deal With Thalassemia

It is a genetic blood disorder that significantly reduces your haemoglobin count. Notably, haemoglobin is a protein molecule present in red blood cells. This protein helps RBCs in carrying oxygen and circulating it in the entire body. Also Read - World Thalassemia Day: Risk Factors, Types And Prevention Tips

The signs and symptoms of thalassemia depend on the type of thalassemia you have and its severity. Some common symptoms include fatigue, slow growth, weakness, abdominal swelling, pale skin, dark urine facial bone deformities etc. Usually, either a newborn shows thalassemia symptoms at the time of birth itself or develops it in the first two years of life. Also Read - World Thalassemia Day 2017: Importance of Blood donation and how it helps people with this fatal disease

Thalassemia occurs when the DNA of your body cells responsible for making haemoglobin, undergo mutation. This mutated DNA is passed on to the next generation.

A simple blood test can confirm the disease. Usually, if an expecting mother is known to be suffering from thalassemia, doctors perform a certain tests to find out if the fetus has also inherited the diseases and if yes, what is the severity of the genetic disease. To do that, chorionic villus sampling (testing a tiny sample of placenta) and amniocentesis (examining sample of fluid surrounding foetus) are performed.

In case, you have inherited a minimum number of mutated genes and suffering from mild thalassemia, you do not require treatment. However, in severe case, you may have to go through frequent blood transfusion, chelation therapy, or stem cell transplant.

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World Thalassemia Day 2020: Causes, Symptoms, Diagnosis And Treatment of The Disease - India.com

Skin Stem Cells Comments Off on World Thalassemia Day 2020: Causes, Symptoms, Diagnosis And Treatment of The Disease – India.com | May 9th, 2020