DORVAL, QC, Oct. 9, 2019 /CNW/ - Novartis Pharmaceuticals Canada Inc. is pleased to announce that sites in Quebec have been certified in accordance with applicable requirements to treat eligible patients with Kymriah (tisagenlecleucel), the first chimeric antigen receptor T cell (CAR-T) therapy that received regulatory approval in Canada. Patients with relapsed/refractory (r/r) pediatric and young adult B-cell acute lymphoblastic leukemia (ALL) and adult r/r diffuse large B-cell lymphoma (DLBCL) may be eligible to be treated with Kymriah at one of the initially certified Canadian treatment sites. This news coincides with the Quebec government announcement that Kymriah is now reimbursed for eligible patients under the Rgie de l'assurance maladie du Qubec (RAMQ)ii.

Eligible patients in Quebec are now able to access Kymriah from the Centre hospitalier universitaire (CHU) Sainte-Justine and Maisonneuve-Rosemont Hospital (HMR) in Montreal.

"Novartis feels it is important to acknowledge the collaborative effort by all stakeholders involved to ensure Canadians have access to the first approved CAR-T therapy for patients with B-cell ALL and DLBCL who historically have poor outcomes. With treatment centers certified in Quebec, this allows patients with these two life-threatening cancers the opportunity to be treated with CAR-T therapy," said Daniel Hbert, Medical Director, Novartis Pharmaceuticals Canada Inc. "Novartis is committed to bringing additional qualified treatment centers from other parts of the country into the network to give Canadians the opportunity to be treated closer to home."

Due to the sophisticated and individualized nature of Kymriah, treatment sites that are part of the network are required to be FACT-accredited (Foundation for the Accreditation of Cellular Therapy), qualified to perform intravenous infusion of stem cells collected from the bone marrow of a donor, also referred to as allogeneic hematopoietic stem cell transplantation (alloSCT) and have experience with cell therapies, leukemia and lymphoma to facilitate safe and seamless delivery of Kymriah to eligible patients.

"We are thrilled with this news because we will now be able to treat patients at our institution with the knowledge that their therapy will be publicly funded. We see this as a significant step forward. The young patients we see who have refractory or relapsed B-cell ALL are desperately in need of a new treatment option. Kymriah brings hope to patients who are literally in a fight for their life." said Dr. Henrique Bittencourt, hematologist at the CHU Sainte-Justine in Montreal and Associate Professor, Department of Pediatrics, Universit de Montral.

"The expertise at HMR has raised the profile of our organization, which is a major Quebec, Canadian and worldwide pole for health innovation. Thanks to the dedicated work of our care, research and teaching teams, patients can now access this new treatment with demonstrated effectiveness and impact on quality of life," said Sylvain Lemieux, President and CEO, Centre intgr universitaire de sant et de services sociaux (CIUSSS) de l'Est-de-l'le-de-Montral.

About Kymriah Kymriah (tisagenlecleucel), a CD19-directed genetically modified autologous T-cell immunocellular therapy, is approved to treat two life-threatening cancers that have limited treatment options and historically poor outcomes, demonstrating the critical need for new therapies for these patients.

Kymriah is approved by Health Canada for use in pediatric and young adult patients 3 to 25 years of age with B-cell acute lymphoblastic leukemia (ALL) who are refractory, have relapsed after allogenic stem cell transplant (SCT) or are otherwise ineligible for SCT, or have experienced second or later relapse; and for the treatment of adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, high grade B-cell lymphoma and DLBCL arising from follicular lymphomai.

Kymriah is a one-time treatment that uses a patient's own T cells to fight and kill cancer cells. Bringing this innovative therapy to Canadian patients requires collaboration among many health system stakeholders.

Kymriah (tisagenlecleucel) Important Safety InformationThe full prescribing information for Kymriah can be found at: http://www.novartis.ca

Novartis Leadership in Cell and Gene TherapyNovartis is at the forefront of investigational immunocellular therapy and was the first pharmaceutical company to significantly invest in CAR-T research, work with pioneers in CAR-T and initiate global CAR-T trials. Kymriah, the first approved CAR-T cell therapy in Canada, is the cornerstone of this strategy. Active research programs are underway targeting other hematologic malignancies and solid tumors, and include efforts focused on next generation CAR-Ts that involve simplified manufacturing schemes and gene edited cells.

About Novartis in CanadaNovartis Pharmaceuticals Canada Inc., a leader in the healthcare field, is committed to the discovery, development and marketing of innovative products to improve the well-being of all Canadians. In 2018, the company invested $52 million in research and development in Canada. Located in Dorval, Quebec, Novartis Pharmaceuticals Canada Inc. employs approximately 1,000 people in Canada and is an affiliate of Novartis AG, which provides innovative healthcare solutions that address the evolving needs of patients and societies. For further information, please consult http://www.novartis.ca.

About NovartisNovartis is reimagining medicine to improve and extend people's lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world's top companies investing in research and development. Novartis products reach more than 750 million people globally and we are finding innovative ways to expand access to our latest treatments. About 108,000 people of more than 140 nationalities work at Novartis around the world. Find out more at http://www.novartis.com.

Kymriah is a registered trademark.

References_____________________________________________i Novartis Pharmaceuticals Canada Inc., Kymriah Product Monograph. January 7, 2019.ii Quebec Ministry of Health and Social Services press release. October 8, 2019. Available at: https://www.newswire.ca/fr/news-releases/la-therapie-car-t-cell-maintenant-disponible-au-quebec-821953237.html

SOURCE Novartis Pharmaceuticals Canada Inc.

For further information: Novartis Media Relations, Daphne Weatherby, Novartis Corporate Communications, +1 514 633 7873, E-mail: camlph.communications@novartis.com

http://www.novartis.ca

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Novartis completes certification of initial sites in Quebec for first approved Canadian CAR-T therapy, Kymriah (tisagenlecleucel)(i) - Canada NewsWire

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Registration is now open for World Cord Blood Day 2019 (November 15th) including the official online conference (free event) which will feature numerous cord blood transplant doctors and cellular therapy researchers such as Dr. Joanne Kurtzberg, Dr. Karen Ballen, Dr. Elizabeth Shpall, Dr. Wise Young and Dr. Filippo Milano. In addition, free educational events will be held around the globe.

TUCSON, Ariz., Oct. 9, 2019 /PRNewswire/ -- World Cord Blood Day (November 15th, 2019) is a free event and open to the public. In addition to events worldwide, World Cord Blood Day will feature a free online conference. Renowned researchers and leading transplant doctors will give introductory presentations for the public as well as academic lectures specifically designed for healthcare professionals.

Attendees will learn about the 40,000+ cord blood transplants performed since 1988 to treat over 80 life-threatening diseases including sickle cell anemia, thalassemia, lymphoma and leukemia. In addition, attendees will learn about exciting advances in the emerging field of regenerative medicine to potentially treat autism, cerebral palsy, spinal cord injury and more.

As the host and organizer of World Cord Blood Day 2019, Save the Cord Foundation is proud to announce the following speakers for this year's program (in order of appearance): Dr. Joanne Kurtzberg (Duke Department of Pediatrics, Duke Center for Autism and Brain Development), Dr. Karen Ballen (University of Virginia), Dave Murphy and Monroe Burgess (Quick Specialized Healthcare Logistics), Dr. Wise Young (Rutgers University), Dr. Elizabeth Shpall (MD Anderson Cancer Center), Dr. Filippo Milano (Fred Hutchinson Cancer Research Center). In addition, attendees will hear from Dr. Alexes Harris who beat cancer thanks to a cord blood transplant from a donor and young Luke Fryer who was treated for cerebral palsy with his own cord blood in a clinical trial.

The morning session will focus on the success of cord blood transplants over the past 30 years and how transplant doctors use cord blood stem cells today, namely, to fight blood cancer. The afternoon session will look at new directions in cord blood research. Attendees will receive updates on several ground-breaking clinical trials using cord blood in regenerative medicine, cellular therapy and more. To view the full agenda, please visit: https://www.worldcordbloodday.org/online-medical-conference-agenda.html

Organized and hosted by Save the Cord Foundation (501c3 non-profit), this year's event is officially sponsored by Quick Specialized Healthcare Logistics. Inspiring Partners include the Cord Blood Association (CBA), Be the Match (NMDP), World Marrow Donor Association (WMDA-Netcord), AABB Center for Cellular Therapy and Foundation for the Accreditation of Cellular Therapy (FACT).

Story continues

Visit http://www.WorldCordBloodDay.org to learn how you can participate and/or host an event. Join us on social media using the hashtags: #WCBD19 and #WorldCordBloodDay.

About Save the Cord Foundation

Save the Cord Foundation (a 501c3 non-profit) was established to advance cord blood education. The Foundation provides non-commercial information to parents, health professionals and the public regarding methods for saving cord blood, as well as current applications using cord blood and the latest research. Learn more at http://www.SaveTheCordFoundation.org.

About Quick Specialized Healthcare Logistics

Quick is the trusted logistics leader serving the Healthcare and Life Science community for almost 40 years. Quick safely transports human organs and tissue for transplant or research, blood, blood products, cord blood, bone marrow, medical devices and personalized medicine, 24/7/365. Quick's specially trained experts work with hospitals, laboratories, blood banks and medical processing canters, and utilize the safest routes to ensure integrity, temperature control and chain of custody throughout the transportation process. Learn more at http://www.quickhealthcare.aero.

Media contact:Charis Ober225955@email4pr.com520-419-0269

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PENNY Lancaster was reduced to tears as she handed five-year-old Ronnie Musselwhite the Young Hero gong at The Suns Who Cares Wins health awards last night.

Spurs fan Ronnie bravely offered to give his older sister Ebonie a bone marrow transplant last year.

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Eight-year-old Ebonie had leukaemia and it was her only hope.

Mum Christine Jenkins, 40, said: Ronnies stem cells worked perfectly. They did what they were supposed to do but the leukaemia came back somewhere new.

Ebonie, of Crawley, West Sussex, nominated her younger brother before her death in June.

Rod Stewarts partner Penny chatted with Ronnie about his love of football and Spurs.

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She said: To say I am humbled to be here is an understatement. Sometimes you think life has turned a corner on you, but then someone else turns up to give you some inspiration.

"The courage this little man has shown is absolutely incredible. Hes only five years old, hes lost a sister.

"He was incredibly shy to stand up in front of everyone to collect his award, but he again was so brave.

Christine said: We want Ronnie to know that what he did still worked, was still brave, even though he lost his sister.

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PM Boris Johnson was also at the awards held at The Suns London HQ near The Shard and paid tribute to our NHS heroes.

He presented an award to a pair of quick-thinking hospital porters who saved the life of a seven-week-old baby boy.

Nick Evans, 48, and Ruth Lowe, 47, sprang into action after Logan Clifford stopped breathing.

His parents, Sarah and Mike were visiting a relative at the Princess Royal Hospital in Telford, Shrops, when they noticed Logans lips had turned blue.

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Sarahs screams alerted Ruth, who shouted for Nick. He grabbed Logan and performed CPR as he ran half a mile down the corridor to A&E.

Nick continued CPR until the resuscitation team took over and the porters stayed by Logans parents side until they knew he was going to be OK. Sarah, 30, has called the two porters my heroes.

As he handed the pair the Ultimate Lifesaver trophy, the PM said: The NHS is revered around the world, and in no small part due to the heroes working in it every day.

He added: My experience of the NHS is like everybody else in the NHS - one of admiration and love.

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"It is the most extraordinary institution in the world. If our country was an omelette then the NHS is the egg white that holds the great British cake together.

Virgin Radio DJ Chris Evans presented the Best Nurse gong to Liz Monaghan, 53. She set up the widely praised Purple Rose initiative, which aims to improve the care for patients and their loved ones in the last days of their life.

Liz, who works at the Florence Nightingale Hospice, based at the Stoke Mandeville Hospital in Aylesbury, Bucks, said: Im a little embarrassed to have won. Im a small part of a big team.

DJ Chris said: Youve got to prepare yourself for nights like this because otherwise they hit you like an express train.

Who Cares Wins Awards: The winners

BEST HEALTH CHARITY

Winner: Matt Hampson Foundation

Former English rugby union player Matt Hampson set up a charity to help others after being left paralysed in a scrum in 2005.

Other nominees: Superhero Foundation and Team Domenica

BEST NEONATAL SPECIALIST

Winner: Professor Kypros Nicolaides

Professor Nicolaides performed pioneering keyhole surgeon on Sherrie Sharps unborn son Jaxon. By extraordinary coincidence, as a young surgeon, he also operated on Sherries mother when she was in the womb.

Other nominees: Dr Vesna Pavasovic and Professor Massimo Caputo

UNSUNG HERO

Winner: Therapeutic Care Volunteers at South Tees NHS Foundation Trust

30 therapeutic care volunteers, who all have a learning or physical disability, give up their time to support patients with spinal injuries at The James Cook University Hospital in Middlesbrough. They include Ify Nwokoro.

Other nominees: Ben Slack and Rob Allen

GROUNDBREAKING PIONEER

Winner: Guys and St Thomas London Auditory Brainstem Implant (ABI) Service

Leia Armitage, eight, was born with a rare form of deafness and was never expected to speak. But she now can thanks to pioneering brain surgery and speech therapy carried out by Guys and St Thomas London Auditory Brainstem Implant (ABI) Service.

Other nominees: Dr Helen Spencer and Girish Vajramani

BEST DOCTOR

Winner: Dr Matthew Boulter

Dr Boulter served in Afghanistan, teaches wild trauma to army medics and his surgery became the first in Cornwall to be given veteran friendly accreditation.

Other nominees: Margaret France and Dr Bijay Sinha

BEST MIDWIFE

Winner: Jane Parke

Jane helped deliver the youngest surviving twin boys in Britain when they were born at 22 weeks last year. She flew 190 miles with their mum Jennie Powell to a specialist neonatal unit.

Other nominees: Charlotte Day and Nagmeh Teymourian

ULTIMATE LIFESAVER

Winner: Ruth Lowe and Nick Evans

Porters Ruth and Nick saved the life of Sarah and Mike Cliffords seven-week-old baby Logan. He stopped breathing as they walked through the main entrance of The Princess Royal Hospital in Telford to visit a sick relative.

Other nominees: Dr Mark Forrest and Mike Merrett

BEST NURSE

Winner: Liz Monaghan

Liz is the Matron of the Florence Nightingale Hospice in Aylesbury, Bucks, and came up with the idea for the widely praised Purple Rose initiative to improve the care for patients in the last days of their lives.

Other nominees: Margaret Ballard and Carlton DeCosta

MENTAL HEALTH HERO

Winner: Ben West

Ben lost his brother Sam, 15, to suicide last year and since his death, has campaigned tirelessly to raise awareness for mental health.

Other nominees: Beth Gregan and Catherine Benfield

YOUNG HERO

Winner: Ronnie Musselwhite

Ronnie offered to help his sister Ebonie by giving her a bone marrow transplant when she was diagnosed with a rare form of leukaemia. Ebonie nominated her brother for his bravery before she died in June.

Other nominees: Bella Field and Kaitlyn Wright

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I only walked ten metres into the room tonight and I already nearly burst into tears three times.

TV star Christine Lampard gave the Best Neonatal Specialist award to Prof Kypros Nicolaides, 66.

He was nominated by Sherrie Sharp, 29, of Horsham, West Sussex, for saving the life of her unborn baby son and her own.

After scans revealed Jaxson had spina bifida, Sherrie was offered a termination. But she contacted Prof Nicolaides, a surgeon at Kings College Hospital, London.

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He had saved her life 30 years earlier when she developed a rare blood disorder in her mums womb.

He agreed to perform ground-breaking surgery on Jaxson while he was in Sherries womb.

Prof Nicolaides said: I was delighted to be able to help. Sherrie said: He has saved so many generations of my family. Hes our guardian angel.

The Who Cares Wins Awards were set up in 2017 by The Sun to honour the nations heroic doctors, nurses, midwives, other NHS staff and volunteers.

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The Duchess of York presented an award to the parents of Natasha Ednan-Laperouse, 15, who died of an allergic reaction to a sandwich from Pret.

The duchess said: Can I just say to The Sun, I think youre incredible. Every minute Im sitting there and thinking Im so lucky. The NHS, The Sun and all of you, this is what makes Britain so great.

Lorraine Kelly, who presented the awards, said: Earlier on this year my dad was very sick and we honestly thought we were going to lose him.

"It was really difficult and it was only because of the efforts of the NHS hes still here. Its fantastic.

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Who Cares Wins Awards: The winners

BEST HEALTH CHARITY

Nominees: Superhero Foundation

Team Domenica

Winner: Matt Hampson Foundation

Former English rugby union player Matt Hampson set up a charity to help others after being left paralysed in a scrum in 2005.

BEST NEONATAL SPECIALIST

Nominees: Dr Vesna Pavasovic

Professor Massimo Caputo

Winner: Professor Kypros Nicolaides

Professor Nicolaides performed pioneering keyhole surgeon on Sherrie Sharps unborn son Jaxon. By extraordinary coincidence, as a young surgeon, he also operated on Sherries mother when she was in the womb.

UNSUNG HERO

Nominees: Ben Slack

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Penny Lancaster is reduced to tears as she gives bone marrow donor Ronnie Musselwhite, 5, The Suns Young H - The Scottish Sun

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This article, written byKatharine Sedivy-Haley, University of British Columbia, originally appeared on The Conversation and is republished here with permission:

When I was applying to graduate school in 2012, it felt like stem cells were about to revolutionize medicine.

Stem cells have the ability to renew themselves, and mature into specialized cells like heart or brain cells. This allows them to multiply and repair damage.

If stem cell genes are edited to fix defects causing diseases like anemia or immune deficiency, healthy cells can theoretically be reintroduced into a patient, thereby eliminating or preventing a disease. If these stem cells are taken or made from the patient themselves, they are a perfect genetic match for that individual, which means their body will not reject the tissue transplant.

Because of this potential, I was excited that my PhD project at the University of British Columbia gave me the opportunity to work with stem cells.

However, stem cell hype has led some to pay thousands of dollars on advertised stem cell treatments that promise to cure ailments from arthritis to Parkinsons disease. These treatments often dont help and may harm patients.

Despite the potential for stem cells to improve medicine, there are many challenges as they move from lab to clinic. In general, stem cell treatment requires we have a good understanding of stem cell types and how they mature. We also need stem cell culturing methods that will reliably produce large quantities of pure cells. And we need to figure out the correct cell dose and deliver it to the right part of the body.

Embryonic, 'induced and pluripotent

Stem cells come in multiple types. Embryonic stem cells come from embryos which makes them controversial to obtain.

A newly discovered stem cell type is the induced pluripotent stem cell. These cells are created by collecting adult cells, such as skin cells, and reprogramming them by inserting control genes which activate or induce a state similar to embryonic stem cells. This embryo-like state of having the versatile potential to turn into any adult cell type, is called being pluripotent.

However, induced pluripotent and embryonic stem cells can form tumours. Induced pluripotent stem cells carry a particularly high risk of harmful mutation and cancer because of their genetic instability and changes introduced during reprogramming.

Genetic damage could be avoided by using younger tissues such as umbilical cord blood, avoiding tissues that might contain pre-existing mutations (like sun-damaged skin cells), and using better methods for reprogramming.

Stem cells used to test drugs

For now, safety concerns mean pluripotent cells have barely made it to the clinic, but they have been used to test drugs.

For drug research, it is valuable yet often difficult to get research samples with specific disease-causing mutations; for example, brain cells from people with amyotrophic lateral sclerosis (ALS).

Researchers can, however, take a skin cell sample from a patient, create an induced pluripotent stem-cell line with their mutation and then make neurons out of those stem cells. This provides a renewable source of cells affected by the disease.

This approach could also be used for personalized medicine, testing how a particular patient will respond to different drugs for conditions like heart disease.

Vision loss from fat stem cells

Stem cells can also be found in adults. While embryonic stem cells can turn into any cell in the body, aside from rare newly discovered exceptions, adult stem cells mostly turn into a subset of mature adult cells.

For example, hematopoietic stem cells in blood and bone marrow can turn into any blood cell and are widely used in treating certain cancers and blood disorders.

A major challenge with adult stem cells is getting the right kind of stem cell in useful quantities. This is particularly difficult with eye and nerve cells. Most research is done with accessible stem cell types, like stem cells from fat.

Fat stem cells are also used in stem cell clinics without proper oversight or safety testing. Three patients experienced severe vision loss after having these cells injected into their eyes. There is little evidence that fat stem cells can turn into retinal cells.

Clinical complications

Currently, stem cell based treatments are still mostly experimental, and while some results are encouraging, several clinical trials have failed.

In the brain, despite progress in developing treatment for genetic disorders and spinal cord injury, treatments for stroke have been unsuccessful. Results might depend on method of stem cell delivery, timing of treatment and age and health of the patient. Frustratingly, older and sicker tissues may be more resistant to treatment.

For eye conditions, a treatment using adult stem cells to treat corneal injuries has recently been approved. A treatment for macular degeneration using cells derived from induced pluripotent stem cells is in progress, though it had to be redesigned due to concerns about cancer-causing mutations.

A path of cautious optimism

While scientists have good reason to be interested in stem cells, miracle cures are not right around the corner. There are many questions about how to implement treatments to provide benefit safely.

In some cases, advertised stem cell treatments may not actually use stem cells. Recent research suggests mesenchymal stem cells, which are commonly isolated from fat, are really a mixture of cells. These cells have regenerative properties, but may or may not include actual stem cells. Calling something a stem cell treatment is great marketing, but without regulation patients dont know what theyre getting.

Members of the public (and grad students) are advised to moderate their excitement in favour of cautious optimism.

Katharine Sedivy-Haley, PhD Candidate in Microbiology and Immunology, University of British Columbia

This article is republished from The Conversation under a Creative Commons license. Read the original article.

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BEYOND LOCAL: Expert recommends 'path of cautious optimism' about the future of stem cell treatment - ThoroldNews.com

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PITTSBURGH (KDKA) In a dish sits a human liver.

Not removed from a person, but created from scratch.

Its not like wahoo and the next morning you think, ah, Im gonna make a human liver,' says Dr. Alejandro Soto-Gutirrez of the Pittsburgh Liver Research Center.

It took five years of trial and error but using stem cells, genetic and tissue engineering, organ cultures and a team of experts in these areas, the researchers have come up with this.

Alexandra Collin de Lhortet, Ph.D. of the University of Pittsburgh School of Medicine explains the process.

A rat liver gets stripped of its cells so that only the connective tissue remains.

From a small piece of human skin, the scientists pluck out stem cells and coax them into becoming human liver cells and the cells are collected.

Then theyre injected into the chamber, called a bioreactor, where they take up residence in the empty rat liver.

The entire process from gathering the cells to make a liver, to get to this point, where you have an actual mini human liver in a bioreactor, takes several months.

It will stay alive, or viable, for only a few days.

But in that short time, the researchers can try different medicines to treat the diseased liver.

You could test any sort of therapeutic by simply injecting this chemical through the system, says Dr. Collin.

In the past, animal livers played a role in this kind of research but human livers didnt always respond in the same way.

With this system, the cells have had genetic modification to recreate diseases, for example, fatty liver, a growing problem in the United States.

This technology has the potential for personalized medicine. From your skin cells, they could grow your own mini liver to figure out which medicines would work for you.

I believe its a very good biological tool to screen treatments that are not otherwise being tested in humans themselves because its dangerous, says Dr. Soto.

As its designed, it would be a long stretch to create livers for transplantation.

If you mean how far we are to make actual livers for people, I think we are very far away. Were probably many years away. But this is a good step, Dr. Soto says.

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After 5 Years Of Trials, Doctors Create Human Liver From Scratch - CBS Pittsburgh

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Lab-grown meat has been successfully made in space for the first time.

Cells of a cow were taken to space where they were grown into small-scale muscle tissue using a 3D bioprinter.

Israeli food technology company, Aleph Farms grew the meat on the Russian segment of the International Space Station, 248 miles away from any natural resources.

The technique could be used in the future to provide meat for people living on the space station.

Aleph Farms said that the aim of the experiment was to advance its research into meat production and prove meat can be produced without natural resources.

"In space, we don't have 10,000 or 15,000 Litres of water available to produce one Kg (2.205 Pound) of beef," Aleph Farms said.

What is lab-grown meat?

This is the world's first lab-grown beef burger in 2013 made in a Petri dish

Lab-grown meat is meat made in a laboratory without killing animals.

Animals are made up of stem cells, which form special tissue like nerve or skin cells.

Scientists worked out how to take cells from an animal - like a cow- and multiply them in a special container called a Petri dish.

Eventually from one tiny muscle cell, tens of billions of cells can be grown. These join together to form muscle tissue.

Lots of strands of muscle tissue together can form 'meat'.

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Lab-grown meat made on International Space Station for the first time - CBBC Newsround

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Many of us rely on makeup as the secret to eternal youth. From covering up dark circles with concealer to adding a flush of colour with a rosy blush it's the perfect little pick-me-up.

But, of course, it's never that simple.

And if you're using the wrong products or the wrong techniques, you may not end up quite as fresh faced as you had hoped.

From using too much powder foundation to forgetting to use SPF, many of our go-to habits could actually be making us look older.

We speak to skin expert Paul Banwell to find out exactly what we should and shouldn't be doing.

To keep your skin hydrated, Paul says you can use simple methods to keep looking youthful.

He says: You can use a high intensity moisturiser, or use indigestible products like a liquid collagen drink - I recommend Skinade, the leading collagen drink which is carefully formulated, a mixture of vitamins and minerals which results in optimal skin health.

Wearing oil based products can clog pores and cause breakouts.

Paul says: The effects of clogged pores can be aided with medical facials like the photogenic facial from TBC Skin atelier, or microdermabrasion, followed by Dermalux LED treatments.

Alternatively, I'd recommend a hydroxyacid boost for pH equilibrium and chemical exfoliation - Rationale's Catalyst Range is best.

However, you can also make a difference by making small changes at home.

Paul says: Similarly, overusing fragranced and alcohol based products may dry out the skin, and in turn cause premature lines and wrinkles.

Try to use oil free products, and aim to use hydrating foundations and creams.

Pollution can be responsible for skin dryness, dullness, clogged pores and skin ageing.

Paul says: Some tips for shielding your skin from pollution are wearing sunscreen, using a good moisturiser to create a barrier between your skin and pollutants and double cleansing your skin - use a product like the Rationale Catalyst cleanser.

Sun exposure, both UV and infrared, can result in sunburn which also causes ageing issues for the skin as the years pass.

They're often credited with giving us a matte, flawless finish but powder foundations can be one of the worst culprits when it comes to ageing. Paul says: Avoid powders, as they tend to set into the fine lines of wrinkles which can make your skin look flaky.

For immune protection, and a product which can be used during a Sunday night ritual to make your skin look fresh and luminous for the week ahead which means you won't need to wear foundation, use the immunologist mask - to be performed weekly (the pot lasts 6 months).

It hydrates and reduces inflammation in problem/ sensitive skin.

While concealers are great for hiding flaws and imperfections, they can also draw attention to any unwanted lines and wrinkles.

Paul says: Concealers might be covering the dark circles, but they also accentuating fine lines, so make sure to only apply concealer to the inner half of your under eye.

Prepping the skin before wearing makeup is also key to a youthful glow. Paul says: Skin around the eyes is thinner that the rest of your face and shows age faster!

Eye creams and products that contain Retin A, a form of vitamin A, are most effective and promote the stimulation of collagen and elastin to tighten the skin.

Suncream shouldn't just be reserved for your annual holiday or trips to the beach. Rather, it should be part of your daily skincare regime.

Paul explains: UV exposure causes 90% of skin damage. Even people who already have signs of premature skin ageing can benefit from making lifestyle changes.

We should all be protecting our skin by using SPF 30 or higher which gives your skin a chance to repair some of the damage.

Fine lines and wrinkles absolutely have a part to play here too, and the best way to eradicate these is through protecting the skin against phototoxic damage and minimising loss of skin integrity.

Collagen peptides in a drink like Skinade will increase collagen turnover and are proven to minimise fine lines.

At the Banwell clinic, we offer Ultimate Sunscreen protection with Rationale B3-T, which will ensure skin is not affected as strongly when in sunlight.

After a long day, it can be tempting to just roll into bed without a second thought for your skin. But you may end up paying the price as a result.

Paul says: Sleeping in your makeup can result in the breakdown of healthy collagen which leads to premature skin ageing. Make sure to take your makeup off thoroughly, and Id recommend a Plasma Shower facial to boost cleansing of the skin, using stem cell technology.

Plasma showers alone help improve texture and quality of skin but can be boosted by various mesotherapy treatments including stem cells, hyaluronic acid and vitamins.

Essentially it encourages hydration, which is essential for optimum physiological functioning of the skin and to optimise all biological processes and immune protection.

Originally posted here:
Make-up mistakes that make you look older - and item that should go in the bin - Mirror Online

Skin Stem Cells Comments Off on Make-up mistakes that make you look older – and item that should go in the bin – Mirror Online | October 9th, 2019

Dr Madeline Lancaster has discovered how to turn stem cells into grey matter. It could lead to treatments for autism and spinal injury

My teenage daughter asks me where Im heading. I tell her Im off to Cambridge to interview a neuroscientist who grows mini-brains in her lab. Wow, my daughter replies. That is so cool.

I recount this to Dr Madeline Lancaster, the scientist in question. She beams and says she thinks its very cool too. In her labs at the Medical Research Councils state-of-the-art Laboratory of Molecular Biology building she cant conceal her enthusiasm as she shows me the mini-brains small curd-like blobs floating in dishes of pinkish fluid. She says wow a lot.

It is these brain organoids (as in organ-like, rather than organism-like) made of living cells that have made Lancaster, 37, a big name in science since she discovered how to create

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The scientist who grows tiny brains in her laboratory - The Times

Skin Stem Cells Comments Off on The scientist who grows tiny brains in her laboratory – The Times | October 9th, 2019

Josh LeVine and Oliver Walsh wanted to create a brand for men that wasnt just targeting the french male model on a motorbike. So, to bolster inclusivity and diversity for mens health, the duo built ASYSTEM, a startup which sells skincare and supplement products.

No one is really building in an aspirational, modern, diverse and inclusive way, which is the way we feel that brands should be built, Walsh said from the ASYSTEM beach house in Venice.

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ASYSTEM just raised a $4 million seed round from a crop of investors, including Firstminute Capital, S8 Capital, PLG Ventures. Board members include Kevin Datoo, former COO of Dollar Shave Club.

The betterment startup offers a subscription-based daily vitamin supplement package for $75 a month and a 3-step skincare package for $45 a month. While ASYSTEM wants to redefine mens wellness its hard to forget about the obvious, also venture-backed competition that has risen up recently.

Another company in the direct to consumer male wellness space is Hims, a San Francisco startup that works on men wellness and has raised $197 million in known venture capital to date, according to its Crunchbase profile.

LeVine said that Hims mainly focuses on Rogaine and Viagra through telemedicine. He pointed to two other brands, Roman, which sports healthy hair starting at $0.53 a day and Keeps, that also offers hair loss treatment and said they are not building products for traditional uses.

When you build a proper brand, you put the sticker on your car, he said. I dont know how many men would put a Hims sticker on their car; Id wear an Asystems on a t-shirt.

ASYSTEMs superhuman supplements promise optimization in focus, stamina, energy, mood, and sex drive, according to a press release. As a WIRED story pointed out this year, the blurry line between pharmaceuticals and supplements can be vague and potentially dangerous.

That said, Walsh said ASYSTEM worked with a range of individualsscientists and a qualified nutritionistto formulate the line.

The skincare uses fruit stem cells, avocado oils, and other ingredients, LeVine said. The entrepreneur started another fruit-based skin care product almost 15 years ago, which made its way into hotels and business airlines and used grape extracts from vineyards.

Along with building new products, the company will use the new funding to build out its experiential hub. Two weeks ago, along with the launch, ASYSTEM had a party in its beach house on Venice beach in California. Roughly 50 men from all ages showed up for a dinner, a beach workout, and guided meditation.

Walsh says the biggest surprise was that everyone was able to stay quiet for more than 10 minutes. Its proof, he says, that dialogue and camaraderie can come from this.

Illustration:Li-Anne Dias

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ASYSTEM Launches With $4M In Seed Funding To 'Redefine Male Wellness' - Crunchbase News

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DetailsCategory: DNA RNA and CellsPublished on Tuesday, 08 October 2019 19:53Hits: 337

Investigational Therapy Designed to Engineer Patients Own Stem Cells to Produce Essential Protein

CAMBRIDGE, MA, USA I October 08, 2019 IAVROBIO, Inc. (NASDAQ: AVRO) (the Company) today announced that the first patient has been dosed in the Companys AVR-RD-04 investigational gene therapy program for cystinosis, a devastating lysosomal storage disease, in an ongoing Phase 1/2 clinical trial sponsored by academic collaborators at the University of California San Diego. The gene therapy is derived from the patients own hematopoietic stem cells, which are genetically modified to produce functional cystinosin, a crucial protein that patients with cystinosis lack.

The trial will enroll up to six patients with cystinosis, a rare inherited disease caused by a defect in the gene that encodes for cystinosin. The cystinosin protein enables transport of the amino acid cystine out of lysosomes. When it is absent, cystine accumulates and crystalizes, causing progressive damage to the kidneys, liver, muscles, eyes and other organs and tissues. Cystinosis affects both children and adults; they face shortened life spans and often painful symptoms, including muscle wasting, difficulty breathing, blindness and kidney failure.

Cystinosis is a debilitating and progressive disease, and new treatment options are sorely needed. The current standard of care does not avert deterioration; at best, it can attenuate symptoms. Thats why gene therapy is particularly exciting: It has the potential to change the course of disease -- and the lives of patients -- by addressing the underlying cause of cystinosis, said Birgitte Volck, MD, PhD, President of Research and Development at AVROBIO. We believe we can engineer patients own stem cells so they sustainably produce the functional protein that is needed to prevent a toxic buildup of cystine and halt progression of the disease. We are so pleased that this investigational gene therapy is now in the clinic in collaboration with Dr. Stephanie Cherqui at UC San Diego.

The single-arm trial will enroll four adults and a potential follow-on cohort of two adults or adolescents at least 14 years of age who are currently being treated with cysteamine, the standard of care for cystinosis. If started at an early age and taken on a strict dosing schedule, cysteamine can delay kidney failure. However, the treatment regimen is highly burdensome, with side effects that can be severe and unpleasant, and many patients find it difficult to adhere to this treatment regimen. Even if compliance is high, cysteamine therapy cannot prevent kidney failure or avert other complications.

For people with cystinosis, there are no healthy days. They must take dozens of pills a day, around the clock, just to stay alive. It is a relentless disease and we urgently need new treatments, said Nancy J. Stack, President of the Cystinosis Research Foundation, which supported development of the gene therapy with more than $5.4 million in grants to Dr. Cherquis lab at UC San Diego. We believe that we are now an important step closer to the potential cure that our community has been working toward for many years.

The trials primary endpoints are safety and tolerability, assessed for up to two years after treatment, as well as efficacy, as assessed by cystine levels in white blood cells. Secondary endpoints to assess efficacy include changes in cystine levels in the blood, intestinal mucosa and skin and cystine crystal counts in the eye and skin. Efficacy will also be evaluated through clinical tests of kidney function, vision, muscle strength, pulmonary function and neurological and psychometric function, as well as through assessments of participants quality of life after treatment. The trial is funded by grants to UC San Diego from the California Institute for Regenerative Medicine (CIRM) as well as the Cystinosis Research Foundation.

This investigational gene therapy starts with the patients own stem cells, which are genetically modified so that their daughter cells can produce and deliver functional cystinosin to cells throughout the body. With this approach we aim to prevent the abnormal accumulation of cystine that causes so many devastating complications, said Stephanie Cherqui, PhD, an Associate Professor of Pediatrics at UC San Diego School of Medicine, and consultant to AVROBIO. We have been working toward this trial for years and we are grateful for all the support that brought us to this moment.

About AVR-RD-04

AVR-RD-04 is a lentiviral-based gene therapy designed to potentially halt the progression of cystinosis with a single dose of the patients own hematopoietic stem cells. The stem cells are genetically modified so they can produce functional cystinosin with the aim of substantially reducing levels of cystine in cells throughout the patients body. Before the infusion of the cells, patients undergo personalized conditioning with busulfan to enable the cells to permanently engraft. The Phase 1/2 clinical trial is being conducted under the name CTNS-RD-04 by AVROBIOs academic collaborators at the University of California, San Diego.

About Cystinosis

Cystinosis is a rare, inherited lysosomal storage disorder characterized by the accumulation of cystine in all the cells of the body, resulting in serious and potentially fatal damage to multiple organs and tissues and the shortening of patients life spans. The kidneys and eyes are especially vulnerable; more than 90% of untreated patients require a kidney transplant before age 20. An estimated 1 in 170,000 people are diagnosed with cystinosis.

About AVROBIO, Inc.

AVROBIO, Inc. is a leading, Phase 2 gene therapy company focused on the development of its investigational gene therapy, AVR-RD-01, in Fabry disease, as well as additional gene therapy programs in other lysosomal storage disorders including Gaucher disease, cystinosis and Pompe disease. The Companys plato platform includes a proprietary vector system, automated cell manufacturing solution and a personalized conditioning regimen deploying state-of-the-art precision dosing. AVROBIO is headquartered in Cambridge, MA and has offices in Toronto, ON. For additional information, visit http://www.avrobio.com.

SOURCE: AVROBIO

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AVROBIO Announces First Patient Dosed in Phase 1/2 Trial of Gene Therapy for Cystinosis | DNA RNA and Cells | News Channels - PipelineReview.com

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New York, Oct. 08, 2019 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "3D Bioprinting Market by Component, Material, Application, End user - Global Forecast to 2024" - https://www.reportlinker.com/p04680742/?utm_source=GNW The 3D bioprinting market is projected to reach USD 1,647 million by 2024 from USD 651 million in 2019, at a CAGR of 20.4% from 2019 to 2024. The growth in this market is mainly driven by technological advancements in 3D bioprinters and biomaterials, increasing use of 3D bioprinting in the pharmaceutical and cosmetology industries, and rising public and private funding to support bioprinting research activities. On the other hand, a shortage of skilled professionals and high development and production costs are hampering the growth of this market.

Microextrusion technology commanded the largest share of 3D bioprinters segment owing to technological advancements.The component segment of 3D bioprinting market is segmented into 3D bioprinters and bioinks.The 3D bioprinters market is further sub-segmented on the basis of technology into magnetic 3D bioprinting, laser-assisted bioprinting, inkjet 3D bioprinting, microextrusion bioprinting, and other technologies; whereas bioinks segment is further sub-segmented into natural, synthetic, and hybrid bioinks.

The microextrusion bioprinting technology has commanded the largest share of the market in 2019 due to technological advancements in the segment and the increasing research activities.

The drug discovery research application segment accounted for the largest share of the 3D bioprinting market in 2019.In terms of applications, the 3D bioprinting market is segmented into research applications and clinical applications.The demand for research applications is further sub-segmented into drug research, regenerative medicine, and 3D cell culture.

Among these, the drug research segment accounted for the largest share of the market in 2019, owing to the growing adoption of the 3D bioprinting technology by biopharmaceutical companies. While, in terms of clinical applications, the market is segmented into skin, bone & cartilage, blood vessels, and other clinical applications.

Based on material, living cells segment commanded the leading market share in 2019Based on material, the 3D bioprinting market is broadly segmented into hydrogels, extracellular matrices, living cells, and other biomaterials.Increasing R&D activities for the use of living cells in 3D bioprinting is driving the growth of the living cells segment.

Living cells have the ability to fabricate patient-specific tissues in a defined manner.With advances in 3D bioprinting, scientists and researchers are making use of living cells as a biomaterial in 3D bioprinting.

These cells can be used to print living tissues as well as organ structures for surgical implantations. However, ethical issues associated with the use of stem cells in 3D bioprinting might hamper growth of the segment.

The US 3D bioprinting market to hold prominent market share over the forecast period.On the basis of region, the 3D bioprinting market is segmented into North America, Europe, Asia Pacific, and Rest of the World (Latin America, and the Middle East and Africa).The US held the significant share of the global 3D bioprinting market in 2019.

Factors such as new product launches and technological advancements in 3D bioprinting technology and the presence of key players in the region are driving the growth of the 3D bioprinting market in the US. Moreover, extensive research activities and funding for 3D bioprinting will further fuel the market growth in the US.

Breakdown of supply-side primary interviews: By Company Type: Tier 1 - 45%, Tier 2 - 35%, and Tier 3 - 20% By Designation: C-level - 26%, Director-level - 30%, and Others - 44% By Region: North America- 34%, Europe - 26%, APAC - 23%, and RoW - 17%

The major players in the market include Organovo Holdings Inc. (US), CELLINK (Sweden), Allevi Inc. (US), Aspect Biosystems Ltd. (Canada), EnvisionTEC GmbH (Germany), Cyfuse Biomedical K.K. (Japan), Poietis (France), TeVido BioDevices (US), Nano3D Biosciences, Inc. (US), ROKIT Healthcare (South Korea), Digilab Inc. (US), regenHU (Switzerland), GeSiM (Germany), Advanced Solutions Life Sciences (US), and Regenovo Biotechnology Co., Ltd. (China) among others.

Research CoverageThis report studies the 3D bioprinting market based on component, application, material, end-user, and region.The report also studies factors such as drivers, restraints, opportunities, and challenges affecting market growth.

It also provides details of the competitive landscape for market leaders. Furthermore, the report analyzes micro markets concerning individual growth trends, and it also forecasts the revenue of the market segments for four main region.

Key Benefits of Buying the ReportThis report focuses on various levels of analysisindustry trends, market shares of top players, and company profiles, which together form basic views.It also analyzes the competitive landscape; and high-growth countries along with their respective drivers, restraints, challenges, and opportunities.

The report will help both established firms as well as new entrants/smaller firms to gauge the pulse of the market and garner greater market shares.Read the full report: https://www.reportlinker.com/p04680742/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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The 3D bioprinting market is projected to reach USD 1,647 million by 2024 from USD 651 million in 2019, at a CAGR of 20.4% from 2019 to 2024 -...

Skin Stem Cells Comments Off on The 3D bioprinting market is projected to reach USD 1,647 million by 2024 from USD 651 million in 2019, at a CAGR of 20.4% from 2019 to 2024 -… | October 9th, 2019

DUBLIN--(BUSINESS WIRE)--The "Cell Therapy - Technologies, Markets and Companies" report from Jain PharmaBiotech has been added to ResearchAndMarkets.com's offering.

This report describes and evaluates cell therapy technologies and methods, which have already started to play an important role in the practice of medicine. Hematopoietic stem cell transplantation is replacing the old fashioned bone marrow transplants. The role of cells in drug discovery is also described. Cell therapy is bound to become a part of medical practice.

Stem cells are discussed in detail in one chapter. Some light is thrown on the current controversy of embryonic sources of stem cells and comparison with adult sources. Other sources of stem cells such as the placenta, cord blood and fat removed by liposuction are also discussed. Stem cells can also be genetically modified prior to transplantation.

Cell therapy technologies overlap with those of gene therapy, cancer vaccines, drug delivery, tissue engineering and regenerative medicine. Pharmaceutical applications of stem cells including those in drug discovery are also described. Various types of cells used, methods of preparation and culture, encapsulation and genetic engineering of cells are discussed. Sources of cells, both human and animal (xenotransplantation) are discussed. Methods of delivery of cell therapy range from injections to surgical implantation using special devices.

Cell therapy has applications in a large number of disorders. The most important are diseases of the nervous system and cancer which are the topics for separate chapters. Other applications include cardiac disorders (myocardial infarction and heart failure), diabetes mellitus, diseases of bones and joints, genetic disorders, and wounds of the skin and soft tissues.

Regulatory and ethical issues involving cell therapy are important and are discussed. The current political debate on the use of stem cells from embryonic sources (hESCs) is also presented. Safety is an essential consideration of any new therapy and regulations for cell therapy are those for biological preparations.

The cell-based markets was analyzed for 2018 and projected to 2028. The markets are analyzed according to therapeutic categories, technologies, and geographical areas. The largest expansion will be in diseases of the central nervous system, cancer, and cardiovascular disorders. Skin and soft tissue repair, as well as diabetes mellitus, will be other major markets.

The report contains information on the following:

Key Topics Covered:

Part I: Technologies, Ethics & Regulations

Executive Summary

1. Introduction to Cell Therapy

2. Cell Therapy Technologies

3. Stem Cells

4. Clinical Applications of Cell Therapy

5. Cell Therapy for Cardiovascular Disorders

6. Cell Therapy for Cancer.

7. Cell Therapy for Neurological Disorders

8. Ethical, Legal and Political Aspects of Cell therapy

9. Safety and Regulatory Aspects of Cell Therapy

Part II: Markets, Companies & Academic Institutions

10. Markets and Future Prospects for Cell Therapy

11. Companies Involved in Cell Therapy

12. Academic Institutions

13. References

For more information about this report visit https://www.researchandmarkets.com/r/9q5tz1

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Global Cell Therapy Technologies, Companies & Markets During the Forecast Period, 2018-2028 - ResearchAndMarkets.com - Business Wire

Cardiac Stem Cells Comments Off on Global Cell Therapy Technologies, Companies & Markets During the Forecast Period, 2018-2028 – ResearchAndMarkets.com – Business Wire | October 9th, 2019

Mesenchymal Stem CellDerived Extracellular Vesicles as Therapeutics and as a Drug Delivery PlatformGyuhyeon Baek, et al., STEM CELLS Translational Medicine

The future of exosome therapeutics has great potential, but several challenges, as discussed in the present study, must be overcome before exosomebased therapy will become an important option as a nextgeneration drug delivery system.

Bmi1 Overexpression in Mesenchymal Stem Cells Exerts Antiaging and Antiosteoporosis Effects by Inactivating p16/p19 Signaling and Inhibiting Oxidative StressGuangpei Chen, et al., STEM CELLS

his study demonstrates that mesenchymal stem cell (MSC) overexpressing Bmi1 exerts antiaging and antiosteoporosis effects. These findings might provide a strategy to enhance the functionality of MSCs for use in therapeutic applications. The results suggest a clinical relevance of Bmi1 in MSCs, for example, upregulation of BMI1 expression in human MSCs by hypoxiccultures or treatment with sonic hedgehog activators, then using them for bone marrow concentrate therapy to enhance MSC potency in antiaging and antiosteoporosis.

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Stem Cells Portal

Cardiac Stem Cells Comments Off on Stem Cells Portal | October 6th, 2019

The new stem cells migrate to the cavities of the large bones and begin producing healthy, normal blood cells. The type of transplant you receive depends on your disease and the availability of a suitable donor.

Autologous (self-transplant): Your own cells are collected and frozen for later use. Autologous transplants are most commonly performed for lymphomas, multiple myeloma, testicular cancer and leukemia.

Syngeneic (identical twin transplant): Stem cells are donated by an identical twin, which is an ideal donor because of the matching genetic identity between donor and recipient.

Allogeneic (donor transplant): Stem cells are collected from a relative or an unrelated donor whose tissue type matches closely with that of the patient, or from umbilical cord blood. Allogeneic transplants are most commonly done for leukemias and bone marrow or immune system failure diseases.

At Winship, our Bone Marrow Transplant Center treats leukemia, lymphoma, multiple myeloma and plasma cell disorders; sickle cell anemia, testicular cancer and bone marrow failures.

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Bone Marrow and Stem Cell Transplant Center | Winship ...

Bone Marrow Stem Cells Comments Off on Bone Marrow and Stem Cell Transplant Center | Winship … | October 5th, 2019

CARLSBAD, Calif.--(BUSINESS WIRE)--

Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs, announced today that the United States Patent and Trademark Office (USPTO) has issued U.S. Patent No. 10,286,009, entitled Pluripotent stem cell-derived oligodendrocyte progenitor cells for the treatment of spinal cord injury covering methods for utilizing pluripotent stem cell-derived oligodendrocyte progenitor cells (OPCs) for the treatment of spinal cord injury (SCI). The claimed methods involve injecting OPCs derived from a pluripotent human stem cell line into the SCI site and covers both human embryonic and induced pluripotent stem cell-derived OPCs. The issued patent has a term that expires no earlier than 2036.

The issuance of this patent is an important milestone for the Company because the allowed claims provide valuable, long term protection for novel treatments employing off-the-shelf OPC1 cells designed to improve recovery outcomes following severe spinal cord injury, stated Brian M. Culley, Chief Executive Officer of Lineage. We believe we have one of the largest cell therapy intellectual property portfolios in the biotech industry and will continue to grow and defend our position as a leader in this exciting space.

OPC1 cells are produced by directing the developmental lineage of pluripotent cell lines to generate a proprietary and consistent population of oligodendritic cells. These cells are administered to the patient in an effort to confer post-injury regeneration, which is intended to provide greater motor recovery compared to the current standard of care. With encouraging data already generated from a 25-patient Phase I safety trial, the current focus for the OPC1 program is to introduce commercially-viable improvements to the manufacturing process and to initiate a comparative study later in 2020.

About OPC1

OPC1 is currently being tested in Phase I/IIa clinical trial known as SCiStar, for the treatment of acute spinal cord injuries. OPCs are naturally-occurring precursors to the cells which provide electrical insulation for nerve axons in the form of a myelin sheath. SCI occurs when the spinal cord is subjected to a severe crush or contusion injury and typically results in severe functional impairment, including limb paralysis, aberrant pain signaling, and loss of bladder control and other body functions. The clinical development of the OPC1 program has been partially funded by a $14.3 million grant from the California Institute for Regenerative Medicine. OPC1 has received Regenerative Medicine Advanced Therapy (RMAT) designation for the treatment of acute SCI and has been granted Orphan Drug designation from the U.S. Food and Drug Administration (FDA).

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineages programs are based on its proprietary cell-based therapy platform and associated development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally-differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed either to replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineages clinical assets include (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase I/IIa development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase I/IIa development for the treatment of acute spinal cord injuries; and (iii) VAC2, an allogeneic cancer immunotherapy of antigen-presenting dendritic cells currently in Phase I development for the treatment of non-small cell lung cancer. For more information, please visit http://www.lineagecell.com or follow the Company on Twitter @LineageCell.

Forward-Looking Statements

Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements, in some cases, can be identified by terms such as believe, may, will, estimate, continue, anticipate, design, intend, expect, could, plan, potential, predict, seek, should, would, contemplate, project, target, tend to, or the negative version of these words and similar expressions. Such statements include, but are not limited to, statements relating to changes in Lineages manufacturing process and the timing of future studies. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Lineages actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including risks and uncertainties inherent in Lineages business and other risks described in Lineages filings with the Securities and Exchange Commission (SEC). Lineages forward-looking statements are based upon its current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. Further information regarding these and other risks is included under the heading Risk Factors in Lineages periodic reports filed with the SEC, including Lineages Annual Report on Form 10-K filed with the SEC on March 14, 2019 and its other reports, which are available from the SECs website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Lineage undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

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Spinal Cord Stem Cells Comments Off on Lineage Cell Therapeutics Announces Issuance of U.S … | October 3rd, 2019

Inside your body, red blood cells are constantly on the move. They deliver oxygen to every tissue in every part of your body. These blood cells also cart away waste. So their work is crucial to your survival. But all that squeezing through tiny vessels is tough on red blood cells. Thats why they last only about four months.

Where do their replacements come from? Stem cells.

These are a very special family of cells. When most other cells divide, the daughter cells look and act exactly like their parents. For example, a skin cell cant make anything but another skin cell. The same is true for cells in the intestine or liver.

Not stem cells. Stem cells can become many different types. That is how an embryo grows from a single fertilized egg into a fetus with trillions of specialized cells. They need to specialize to make up tissues that function very differently, including those in the brain, skin, muscle and other organs. Later in life, stem cells also can replace worn-out or damaged cells including red blood cells.

The remarkable abilities of stem cells make them very exciting to scientists. One day, experts hope to use stem cells to repair or replace many different kinds of tissues, whether injured in accidents or damaged by diseases. Such stem cell therapy would allow the body to heal itself. Scientists have found a way to put specialized cells to work repairing damage, too. Together, these cell-based therapies might one day make permanent disabilities a thing of the past.

One unusual type of stem cell offers special promise for such therapeutic uses. For the recent development of this cell type, Shinya Yamanaka shared the 2012 Nobel Prize in medicine.

Meet the family

Blood stem cells live inside your bones, in what is called marrow. There, they divide over and over. Some of the new cells remain stem cells. Others form red blood cells. Still others morph into any of the five types of white blood cells that will fight infections. Although blood stem cells can become any one of these specialized blood cells, they cannot become muscle, nerve or other types of cells. They are too specialized to do that.

Another type of stem cell is more generalized. These can mature into any type of cell in the body. Such stem cells are called pluripotent (PLU ree PO tint). The word means having many possibilities. And its not hard to understand why these cells have captured the imaginations of many scientists.

Until recently, all pluripotent cells came from embryos. Thats why scientists called them embryonic stem cells. After an egg is fertilized, it divides in two. These two cells split again, to become four cells, and so on. In the first few days of this embryos development, each of its cells is identical to all the others. Yet each cell has the potential to develop into any specialized cell type.

When the human embryo reaches three to five days old, its cells start to realize their potential. They specialize. Some will develop into muscle cells or bone cells. Others will form lung cells or maybe the cells lining the stomach. Once cells specialize, their many possibilities suddenly become limited.

By birth, almost all of a babys cells will have specialized. Each cell type will have its own distinctive shape and function. For example, muscle cells will be long and able to contract, or shorten. Red blood cells will be small and plate-shaped, so they can slip through blood vessels with ease.

Hidden among all of these specialized cells are pockets of adult stem cells. (Yes, even newborns have adult stem cells.) Unlike embryonic stem cells, adult stem cells cannot transform into any and every cell type. However, adult stem cells can replace several different types of specialized cells as they wear out. One type of adult stem cell is found in your marrow, making new blood cells. More types are found in other tissues, including the brain, heart and gut.

Among naturally occurring stem cells, the embryonic type is the most useful. Adult stem cells just arent as flexible. The adult type also is relatively rare and can be difficult to separate from the tissues in which it is found. Although more versatile, embryonic stem cells are both difficult to obtain and controversial. Thats because harvesting them requires destroying an embryo.

Fortunately, recent discoveries in stem cell research now offer scientists a third and potentially better option.

The search for answers

In 2006, Shinya Yamanaka discovered that specialized cells like those in skin could be converted back into stem cells. Working at Kyoto University in Japan, this doctor and scientist induced or persuaded mature cells to become stem cells. He did this by inserting a specific set of genes into the cells. After several weeks, the cells behaved just like embryonic cells. His new type of stem cells are called induced pluripotent stem cells, or iP stem cells (and sometimes iPS cells).

Yamanakas discovery represented a huge leap forward. The iP stem cells offer several advantages over both embryonic and adult stem cells. First, iP stem cells are able to become any cell type, just as embryonic stem cells can. Second, they can be made from any starting cell type. That means they are easy to obtain. Third, in the future, doctors would be able to treat patients with stem cells created from their own tissues. Such cells would perfectly match the others, genetically. That means the patients immune system (including all of its white blood cells) would not attack the introduced cells. (The body often mounts a life-threatening attack against transplanted organs that come from other people because they dont offer such a perfect match. To the body, they seem foreign and a potentially dangerous invader.)

Scientists the world over learned of the technique developed by Yamanaka (who now works at the Gladstone Institutes which is affiliated with the University of California, San Francisco). Many of these researchers adopted Yamanakas procedure to create their own induced pluripotent stem cells. For the first time, researchers had a tool that could allow them to make stem cells from people with rare genetic diseases. This helps scientists learn what makes certain cell types die. Experts can also expose small batches of these diseased cells to different medicines. This allows them to test literally thousands of drugs to find out which works best.

And in the future, many experts hope induced stem cells will be used to replace adult stem cells and the cells of tissues that are damaged or dying.

Therapies take patients and patience

Among those experts is Anne Cherry, a graduate student at Harvard University. Cherry is using induced stem cells to learn more about a very rare genetic disease called Pearson syndrome. A syndrome is a group of symptoms that occur together. One symptom of Pearson syndrome is that stem cells in bone marrow cannot make normal red blood cells. This condition typically leads to an early death.

Cherry has begun to study why these stem cells fail.

She started by taking skin cells from a girl with the disease. She placed the cells in a test tube and added genes to turn them into stem cells. Over several weeks, the cells began to make proteins for which the inserted genes had provided instructions. Proteins do most of the work inside cells. These proteins turned off the genes that made the cells act like skin cells. Before long, the proteins turned on the genes to make these cells behave like embryonic stem cells.

After about three months, Cherry had a big batch of the new induced stem cells. Those cells now live in Petri dishes in her lab, where they are kept at body temperature (37 Celsius, or 98.6 Fahrenheit). The scientist is now trying to coax the induced stem cells into becoming blood cells. After that, Cherry wants to find out how Pearson syndrome kills them.

Meanwhile, the patient who donated the skin cells remains unable to make blood cells on her own. So doctors must give her regular transfusions of blood from a donor. Though life-saving, transfusions come with risks, particularly for someone with a serious disease.

Cherry hopes to one day turn the girls induced stem cells into healthy new blood stem cells and then return them to the girls body. Doing so could eliminate the need for further transfusions. And since the cells would be the girls own, there would be no risk of her immune system reacting to them as though they were foreign.

Sight for sore eyes

At University of Nebraska Medical Center in Omaha, Iqbal Ahmad is working on using stem cells to restore sight to the blind. A neuroscientist someone who studies the brain and nervous system Ahmad has been focusing on people who lost sight when nerve cells in the eyes retina died from a disease called glaucoma (glaw KOH muh).

Located inside the back of the eye, the retina converts incoming light into electrical signals that are then sent to the brain. Ahmad is studying how to replace dead retina cells with new ones formed from induced pluripotent stem cells.

The neuroscientist starts by removing adult stem cells from the cornea, or the clear tissue that covers the front of the eye. These stem cells normally replace cells lost through the wear and tear of blinking. They cannot become nerve cells at least not on their own. Ahmad, however, can transform these cells into iP stem cells. Then, with prodding, he turns them into nerve cells.

To make the transformation, Ahmad places the cornea cells on one side of a Petri dish. He then places embryonic stem cells on the other side. A meshlike membrane separates the two types of cells so they cant mix. But even though they cant touch, they do communicate.

Cells constantly send out chemical signals to which other cells respond. When the embryonic stem cells speak, the eye cells listen. Their chemical messages persuade the eye cells to turn off the genes that tell them to be cornea cells. Over time, the eye cells become stem cells that can give rise to different types of cells, including nerve cells.

When Ahmads team implanted the nerve cells into the eyes of laboratory mice and rats, they migrated to the retina. There, they began replacing the nerve cells that had died from glaucoma. One day, the same procedure may restore vision to people who have lost their sight.

Another approach

In using a bodys own cells to repair injury or to treat disease, stem cells arent always the answer. Although stem cells offer tremendous advances in regenerating lost tissue, some medical treatments may work better without them. Thats thanks to the chemical communication going on between all cells all of the time. In some situations, highly specialized cells can act as a conductor, directing other cells to change their tune.

In 2008, while working at the University of Cambridge in England, veterinary neurologist Nick Jeffery began a project that used cells taken from the back of the nose. But Jeffery and his team were not out to create stem cells. Instead, the scientists used those nasal cells to repair damaged connections in the spinal cord.

The spinal cord is basically a rope of nerve cells that ferry signals to and from the brain and other parts of the body. Injuring the spinal cord can lead to paralysis, or the loss of sensation and the inability to move muscles.

Like Ahmad, some researchers are using stem cells to replace damaged nerve cells. But Jeffery, now at Iowa State University in Ames, doesnt think such techniques are always necessary to aid recovery from spinal injuries. Stem cell transplantation, points out Jefferys colleague, neuroscientist Robin Franklin, is to replace a missing cell type. In a spinal injury, the nerve cells arent missing. Theyre just cut off.

Nerve cells contain long, wirelike projections called axons that relay signals to the next cell. When the spine is injured, these axons can become severed, or cut. Damaging an axon is like snipping a wire the signal stops flowing. So the Cambridge scientists set out to see if they could restore those signals.

Jeffery and his fellow scientists work with dogs that have experienced spinal injuries. Such problems are common in some breeds, including dachshunds. The team first surgically removed cells from the dogs sinuses or the hollow spaces in the skull behind the nose. These are not stem cells. These particular cells instead encourage nerve cells in the nose to grow new axons. These cells help the pooches maintain their healthy sense of smell.

The scientists grew these sinus cells in the lab until they had reproduced to large numbers. Then the researchers injected the cells into the spinal cords of two out of every three doggy patients. Each treated dog received an injection of its own cells. The other dogs got an injection of only the liquid broth used to feed the growing cells.

Over several months, the dogs owners repeatedly brought their pets back to the lab for testing on a treadmill. This allowed the scientists to evaluate how well the animals coordinated their front and hind feet while walking. Dogs that had received the nasal cells steadily improved over time. Dogs that received only the liquid did not.

This treatment did not result in a perfect cure. Nerve cells did reconnect several portions of the spinal cord. But nerve cells that once linked to the brain remained disconnected. Still, these dog data indicate that nasal cells can aid in recovering from a spinal cord injury.

Such new developments in cellular research suggest that even more remarkable medical advancements may be just a few years away. Yamanaka, Cherry, Ahmad, Jeffery, Franklin and many other scientists are steadily unlocking secrets to cellular change. And while you cant teach an old dog new tricks, scientists are finding out that the same just isnt true of cells anymore.

cornea The clear covering over the front of the eye.

embryo A vertebrate, or animal with a backbone, in its early stages of development.

gene A section of DNA that carries the genetic instructions for making a protein. Proteins do most of the work in cells.

glaucoma An eye disease that damages nerve cells carrying signals to the brain.

immune cell White blood cell that helps protect the body against germs.

molecule A collection of atoms.

neuron (or nerve cell) The basic working unit of the nervous system. These cells relay nerve signals.

neuroscientist A researcher who studies neurons and the nervous system.

paralysis Loss of feeling in some part of the body and an inability to move that part.

retina The light-sensitive lining at the back of the eye. It converts light into electrical impulses that relay information to the brain.

sinus An opening in the bone of the skull connected to the nostrils.

spinal cord The ropelike collection of neurons that connect the brain with nerves throughout the body.

tissue A large collection of related, similar cells that together work as a unit to perform a particular function in living organisms. Different organs of the human body, for instance, often are made from many different types of tissues. And brain tissue will be very different from bone or heart tissue.

transfusion The process of transferring blood into one person that had been collected from another.

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The prognosis for spinal cord injuries varies depending on the severity of the injury. There is always hope of recovering some function with spinal cord injuries. The completeness and location of the injury will determine the prognosis.

There are two levels of completeness in spinal cord injuries which impact the outlook:

Spinal cord injuries in which the patient has not experienced paralysis have the greatest chance of recovery. However, those patients who do experience paralysis still have a remarkable chance that is improving with research every day. The sooner treatments are implemented to strengthen muscles below the level of the spinal cord injury, the better the prognosis.

The first year of recovery is the hardest as the patient is just beginning to adjust to his or her condition. The use of physical and occupational therapy during this time is the key to recovery. The extent of the function fully returning is typically seen in the first two years after the initial injury.

Treatment options vary with each spinal cord injury, but typically include:

Mental health is a huge part of recovery for the spinal cord injury patient. Anxiety and depression are common in spinal cord injury patients. These patients will go through good days, and not so good days.

There may be days where the patient wants to give up completely on treatments, and will wonder if it is all worth it. Keeping up with the mental health of the spinal cord injury patient is incredibly important for the overall recovery. Mental health has been proven to directly relate to physical health.

Having a good support system is incredibly important to the overall outlook of a spinal cord injury patient. Spinal cord injury patients will need both physical and emotional support.

Caregivers should continually provide patients with:

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Hematopoietic stem cells (HSCs) are defined as stem cells that have a preference for becoming cells of the blood and immune system, such as white bloodcells, red bloodcells, and platelets. Found in the peripheral blood and bone marrow,hematopoietic stem cells are also present in plentiful supply within the umbilical cord blood of newborn babies.

For the past thirty years, cord blood has been used within transplant medicine, including for the treatment of leukemia and other blood diseases. For most conditions in which a bone marrow or peripheral blood stem cell transplant is an option, a cord blood transplant is a potential alternative.

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Hematopoietic stem cells(HSCs) are thestem cellsthat repopulate the blood and immune system within humans, via a process known ashaematopoiesis. For this reason, hematopoietic stem cell transplantation, better known as HSCT, can be a promising treatment approach for a wide range of conditions.

The use of human cord blood cells dates back as early as 1974, when it was first proposed that stem cell and progenitor cells were present in human cord blood.By 1983, the use of cord blood as an alternative to bone marrow had been proposed. Five years later in 1988, the first successful cord blood transplant to restore a patients blood and immune system cells took place in France.

In addition to a long history of use within transplant medicine, human cord blood cells are playing a growing role within regenerative medicine. Cord blood stem cells have been induced to develop into neural cells, suggesting that they may represent a potential treatment for neurological conditions, such as Alzheimers, Parkinsons, spinal cord injury, dementia, and related conditions.

Human cord blood cells can also develop into blood vessels, making them promising for the repair of tissues following stroke, coronary heart disease, rheumatic heart disease, congestive heart failure, and congenital heart conditions.

What Are the Benefits of Banking #CordBlood? The main benefit to banking cord blood is it allows parents to preserve stem cells for future medical use. Many parts of the body do not regenerate, so they are at risk of failing https://t.co/3oc4Ai4qef pic.twitter.com/kYy9Ds64ad

BioInformant (@StemCellMarket) July 23, 2018

It is also interesting to consider the common disease categories treatable with cord blood transplant, as shown in the table below.

There are more than 80 medical conditions for which transplantation of hematopoietic stem cells (including cord blood transplant) is a standard treatment option. Most of these therapies require allogeneic transplants, where the patient must use a genetically-matched cord blood donor. The only exceptions to this are patients who are transplanted for solid tumors or acquired anemias. In these situations, the patient may receive an autologous transplant.

Comprehensive lists of conditions treatable with hematopoietic stem cells are available here and here.

In addition, there is a range of disease categories for which cord blood transplant could represent a viable treatment method in the future. For these conditions, there are still unknown criteria that need to be determined before the cord blood stem cell transplant can become commonplace, such as patient criteria for optimal treatment effectiveness, optimum stem cell quantity for use in transplant, and preferred method of stem cell delivery into the patient, as shown below.

Download this infographic now and reference it later.

What do you think of the future of hematopoietic stem cell transplant? Share your thoughts in the comments below.

Hematopoietic Stem Cells: What Diseases Can these Stem Cells Treat?

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The Central Nervous System

The mature mammalian central nervous system (CNS) is composed of three major differentiated cell types: neurons, astrocytes and oligodendrocytes. Neurons transmit information through action potentials and neurotransmitters to other neurons, muscle cells or gland cells. Astrocytes and oligodendrocytes, collectively called glial cells, play important roles of their own, in addition to providing a critical support role for optimal neuronal functioning and survival. During mammalian embryogenesis, CNS development begins with the induction of the neuroectoderm, which forms the neural plate and then folds to give rise to the neural tube. Within these neural structures there exists a complex and heterogeneous population of neuroepithelial progenitor cells (NEPs), the earliest neural stem cell type to form.1,2 As CNS development proceeds, NEPs give rise to temporally and spatially distinct neural stem/progenitor populations. During the early stage of neural development, NEPs undergo symmetric divisions to expand neural stem cell (NSC) pools. In the later stage of neural development, NSCs switch to asymmetric division cycles and give rise to lineage-restricted progenitors. Intermediate neuronal progenitor cells are formed first, and these subsequently differentiate to generate to neurons. Following this neurogenic phase, NSCs undergo asymmetric divisions to produce glial-restricted progenitors, which generate astrocytes and oligodendrocytes. The later stage of CNS development involves a period of axonal pruning and neuronal apoptosis, which fine tunes the circuitry of the CNS. A previously long-held dogma maintained that neurogenesis in the adult mammalian CNS was complete, rendering it incapable of mitotic divisions to generate new neurons, and therefore lacking in the ability to repair damaged tissue caused by diseases (e.g. Parkinsons disease, multiple sclerosis) or injuries (e.g. spinal cord and brain ischemic injuries). However, there is now strong evidence that multipotent NSCs do exist, albeit only in specialized microenvironments, in the mature mammalian CNS. This discovery has fuelled a new era of research into understanding the tremendous potential that these cells hold for treatment of CNS diseases and injuries.

Neurobiologists routinely use various terms interchangeably to describe undifferentiated cells of the CNS. The most commonly used terms are stem cell, precursor cell and progenitor cell. The inappropriate use of these terms to identify undifferentiated cells in the CNS has led to confusion and misunderstandings in the field of NSC and neural progenitor cell research. However, these different types of undifferentiated cells in the CNS technically possess different characteristics and fates. For clarity, the terminology used here is:

Neural Stem Cell (NSCs): Multipotent cells which are able to self-renew and proliferate without limit, to produce progeny cells which terminally differentiate into neurons, astrocytes and oligodendrocytes. The non-stem cell progeny of NSCs are referred to as neural progenitor cells.

Neural Progenitor Cell: Neural progenitor cells have the capacity to proliferate and differentiate into more than one cell type. Neural progenitor cells can therefore be unipotent, bipotent or multipotent. A distinguishing feature of a neural progenitor cell is that, unlike a stem cell, it has a limited proliferative ability and does not exhibit self-renewal.

Neural Precursor Cells (NPCs): As used here, this refers to a mixed population of cells consisting of all undifferentiated progeny of neural stem cells, therefore including both neural progenitor cells and neural stem cells. The term neural precursor cells is commonly used to collectively describe the mixed population of NSCs and neural progenitor cells derived from embryonic stem cells and induced pluripotent stem cells.

Prior to 1992, numerous reports demonstrated evidence of neurogenesis and limited in vitro proliferation of neural progenitor cells isolated from embryonic tissue in the presence of growth factors.3-5 While several sub-populations of neural progenitor cells had been identified in the adult CNS, researchers were unable to demonstrate convincingly the characteristic features of a stem cell, namely self-renewal, extended proliferative capacity and retention of multi-lineage potential. In vivo studies supported the notion that proliferation occurred early in life, whereas the adultCNS was mitotically inactive, and unable to generate new cells following injury. Notable exceptions included several studies in the 1960s that clearly identified a region of the adult brain that exhibited proliferation (the forebrain subependyma)6 but this was believed to be species-specific and was not thought to exist in all mammals. In the early 1990s, cells that responded to specific growth factors and exhibited stem cell features in vitro were isolated from the embryonic and adult CNS.7-8 With these studies, Reynolds and Weiss demonstrated that a rare population of cells in the adult CNS exhibited the defining characteristics of a stem cell: self-renewal, capacity to produce a large number of progeny and multilineage potential. The location of stem cells in the adult brain was later identified to be within the striatum,9 and researchers began to show that cells isolated from this region, and the dorsolateral region of the lateral ventricle of the adult brain, were capable of differentiating into both neurons and glia.10

During mammalian CNS development, neural precursor cells arising from the neural tube produce pools of multipotent and more restricted neural progenitor cells, which then proliferate, migrate and further differentiate into neurons and glial cells. During embryogenesis, neural precursor cells are derived from the neuroectoderm and can first be detected during neural plate and neural tube formation. As the embryo develops, neural stem cells can be identified in nearly all regions of the embryonic mouse, rat and human CNS, including the septum, cortex, thalamus, ventral mesencephalon and spinal cord. NSCs isolated from these regions have a distinct spatial identity and differentiation potential. In contrast to the developing nervous system, where NSCs are fairly ubiquitous, cells with neural stem cell characteristics are localized primarily to two key regions of the mature CNS: the subventricular zone (SVZ), lining the lateral ventricles of the forebrain, and the subgranular layer of thedentate gyrus of the hippocampal formation (described later).11 In the adult mouse brain, the SVZ contains a heterogeneous population of proliferating cells. However, it is believed that the type B cells (activated GFAP+/PAX6+ astrocytes or astrogliallike NSCs) are the cells that exhibit stem cell properties, and these cells may be derived directly from radial glial cells, the predominant neural precursor population in the early developing brain. NPCs in this niche are relatively quiescent under normal physiological conditions, but can be induced to proliferate and to repopulate the SVZ following irradiation.10 SVZ NSCs maintain neurogenesis throughout adult life through the production of fast-dividing transit amplifying progenitors (TAPs or C cells), which then differentiate and give rise to neuroblasts. TAPs and neuroblasts migrate through the rostral migratory stream (RMS) and further differentiate into new interneurons in the olfactory bulb. This ongoing neurogenesis, which is supported by the NSCs in the SVZ, is essential for maintenance of the olfactory system, providing a source of new neurons for the olfactory bulb of rodents and the association cortex of non-human primates.12 Although the RMS in the adult human brain has been elusive, a similar migration of neuroblasts through the RMS has also been observed.13 Neurogenesis also persists in the subgranular zone of the hippocampus, a region important for learning and memory, where it leads to the production of new granule cells. Lineage tracing studies have mapped the neural progenitor cells to the dorsal region of the hippocampus, in a collapsed ventricle within the dentate gyrus.10 Studies have demonstrated that neurogenic cells from the subgranular layer may have a more limited proliferative potential than the SVZ NSCs and are more likely to be progenitor cells than true stem cells.14 Recent evidence also suggests that neurogenesis plays a different role in the hippocampus than in the olfactory bulb. Whereas the SVZ NSCs play a maintenance role, it is thought that hippocampal neurogenesis serves to increase the number of new neurons and contributes to hippocampal growth throughout adult life.12 Neural progenitor cells have also been identified in the spinal cord central canal ventricular zone and pial boundary15-16, and it is possible that additional regional progenitor populations will be identified in the future.

In vitro methodologies designed to isolate, expand and functionally characterize NSC populations have revolutionized our understanding of neural stem cell biology, and increased our knowledge of the genetic and epigenetic regulation of NSCs.17 Over the past several decades, a number of culture systems have been developed that attempt to recapitulate the distinct in vivo developmental stages of the nervous system, enabling theisolation and expansion of different NPC populations at different stages of development. Here, we outline the commonly used culture systems for generating NPCs from pluripotent stem cells (PSCs), and for isolating and expanding NSCs from the early embryonic, postnatal and adult CNS.

Neural induction and differentiation of pluripotent stem cells: Early NPCs can be derived from mouse and human PSCs, which include embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs), using appropriate neural induction conditions at the first stage of differentiation. While these neural differentiation protocols vary widely, a prominent feature in popular embryoid body-based protocols is the generation of neural rosettes, morphologically identifiable structures containing NPCs, which are believed to represent the neural tube. The NPCs present in the neural rosette structures are then isolated, and can be propagated to allow NPC expansion, while maintaining the potential to generate neurons and glial cells. More recently, studies have shown that neural induction of PSCs can also be achieved in a monolayer culture system, wherein human ESCs and iPSCs are plated onto a defined matrix, and exposed to inductive factors.18 A combination of specific cytokines or small molecules, believed to mimic the developmental cues for spatiotemporal patterning in the developing brain during embryogenesis, can be added to cultures at the neural induction stage to promote regionalization of NPCs. These patterned NPCs can then be differentiated into mature cell types with phenotypes representative of different regions of the brain.19-24 New protocols have been developed to generate cerebral organoids from PSC-derived neural progenitor cells. Cerebral organoids recapitulate features of human brain development, including the formation of discrete brain regions featuring characteristic laminar cellular organization.25

Neurosphere culture: The neurosphere culture system has been widely used since its development as a method to identify NSCs.26-29 A specific region of the CNS is microdissected, mechanically or enzymatically dissociated, and plated in adefined serum-free medium in the presence of a mitogenic factor, such as epidermal growth factor (EGF) and/or basic fibroblast growth factor (bFGF). In the neurosphere culture system, NSCs, as well as neural progenitor cells, begin to proliferate in response to these mitogens, forming small clusters of cells after 2 - 3 days. The clusters continue to grow in size, and by day 3 - 5, the majority of clusters detach from the culture surface and begin to grow in suspension. By approximately day seven, depending on the cell source, the cell clusters, called neurospheres, typically measure 100 - 200 m in diameter and are composed of approximately 10,000 - 100,000 cells. At this point, the neurospheres should be passaged to prevent the cell clusters from growing too large, which can lead to necrosis as a result of a lack of oxygen and nutrient exchange at the neurosphere center. To passage the cultures, neurospheres are individually, or as a population, mechanically or enzymatically dissociated into a single cell suspension and replated under the same conditions as the primary culture. NSCs and neural progenitor cells again begin to proliferate to form new cell clusters that are ready to be passaged approximately 5 - 7 days later. By repeating the above procedures for multiple passages, NSCs present in the culture will self-renew and produce a large number of progeny, resulting in a relatively consistent increase in total cell number over time. Neurospheres derived from embryonic mouse CNS tissue treated in this manner can be passaged for up to 10 weeks with no loss in their proliferative ability, resulting in a greater than 100- fold increase in total cell number. NSCs and neural progenitors can be induced to differentiate by removing the mitogens and plating either intact neurospheres or dissociated cells on an adhesive substrate, in the presence of a low serum-containing medium. After several days, virtually all of the NSCs and progeny will differentiate into the three main neural cell types found in the CNS: neurons, astrocytes and oligodendrocytes. While the culture medium, growth factor requirements and culture protocols may vary, the neurosphere culture system has been successfully used to isolate NSCs and progenitors from different regions of the embryonic and adult CNS of many species including mouse, rat and human.

Adherent monolayer culture: Alternatively, cells obtained from CNS tissues can be cultured as adherent cultures in a defined, serum-free medium supplemented with EGF and/or bFGF, in the presence of a substrate such as poly-L-ornithine, laminin, or fibronectin. When plated under these conditions, the neural stem and progenitor cells will attach to the substrate-coated cultureware, as opposed to each other, forming an adherent monolayer of cells, instead of neurospheres. The reported success of expanding NSCs in long-term adherent monolayer cultures is variable and may be due to differences in the substrates, serum-free media andgrowth factors used.17 Recently, protocols that have incorporated laminin as the substrate, along with an appropriate serum-free culture medium containing both EGF and bFGF have been able to support long-term cultures of neural precursors from mouse and human CNS tissues.30-32 These adherent cells proliferate and become confluent over the course of 5 - 10 days. To passage the cultures, cells are detached from the surface by enzymatic treatment and replated under the same conditions as the primary culture. It has been reported that NSCs cultured under adherent monolayer conditions undergo symmetric divisions in long-term culture.30,33 Similar to the neurosphere culture system, adherently cultured cells can be passaged multiple times and induced to differentiate into neurons, astrocytes and oligodendrocytes upon mitogen removal and exposure to a low serum-containing medium.

Several studies have suggested that culturing CNS cells in neurosphere cultures does not efficiently maintain NSCs and produces a heterogeneous cell population, whereas culturing cells under serum-free adherent culture conditions does maintain NSCs.17 While these reports did not directly compare neurosphere and adherent monolayer culture methods using the same medium, growth factors or extracellular matrix to evaluate NSC numbers, proliferation and differentiation potential, they emphasize that culture systems can influence the in vitro functional properties of NSCs and neural progenitors. It is important that in vitro methodologies for NSC research are designed with this caveat in mind, and with a clear understanding of what the methodologies are purported to measure.34-35

Immunomagnetic or immunofluorescent cell isolation strategies using antibodies directed against cell surface markers present on stem cells, progenitors and mature CNS cells have been applied to the study of NSCs. Similar to stem cells in other systems, the phenotype of CNS stem cells has not been completely determined. Expression, or lack of expression, of CD34, CD133 and CD45 antigens has been used as a strategy for the preliminary characterization of potential CNS stem cell subsets. A distinct subset of human fetal CNS cells with the phenotype CD133+ 5E12+ CD34- CD45- CD24-/lo has the ability to form neurospheres in culture, initiate secondary neurosphere formation, and differentiate into neurons and astrocytes.36 Using a similar approach, fluorescence-activated cell sorting (FACS)- based isolation of nestin+ PNA- CD24- cells from the adult mouse periventricular region enabled significant enrichment of NSCs(80% frequency in sorted population, representing a 100-fold increase from the unsorted population).37 However, the purity of the enriched NSC population was found to be lower when this strategy was reevaluated using the more rigorous Neural Colony-Forming Cell (NCFC) assay.38-39 NSC subsets detected at different stages of CNS development have been shown to express markers such as nestin, GFAP, CD15, Sox2, Musashi, CD133, EGFR, Pax6, FABP7 (BLBP) and GLAST40-45. However, none of these markers are uniquely expressed by NSCs; many are also expressed by neural progenitor cells and other nonneural cell types. Studies have demonstrated that stem cells in a variety of tissues, including bone marrow, skeletal muscle and fetal liver can be identified by their ability to efflux fluorescent dyes such as Hoechst 33342. Such a population, called the side population, or SP (based on its profile on a flow cytometer), has also been identified in both mouse primary CNS cells and cultured neurospheres.46 Other non-immunological methods have been used to identify populations of cells from normal and tumorigenic CNS tissues, based on some of the in vitro properties of stem cells, including FABP7 expression and high aldehyde dehydrogenase (ALDH) enzyme activity. ALDH-bright cells from embryonic rat and mouse CNS have been isolated and shown to have the ability to generate neurospheres, neurons, astrocytes and oligodendrocytes in vitro, as well as neurons in vivo, when transplanted into the adult mouse cerebral cortex.47-50 NeuroFluor CDr3 is a membrane-permeable fluorescent probe that binds to FABP7 and can be used to detect and isolate viable neural progenitor cells from multiple species.42-43

Multipotent neural stem-like cells, known as brain tumor stem cells (BTSCs) or cancer stem cells (CSCs), have been identified and isolated from different grades (low and high) and types of brain cancers, including gliomas and medulloblastomas.51-52 Similar to NSCs, these BTSCs exhibit self-renewal, high proliferative capacity and multi-lineage differentiation potential in vitro. They also initiate tumors that phenocopy the parent tumor in immunocompromised mice.53 No unique marker of BTSCs has been identified but recent work suggests that tumors contain a heterogenous population of cells with a subset of cells expressing the putative NSC marker CD133.53 CD133+ cells purified from primary tumor samples formed primary tumors, when injected into primary immunocompromised mice, and secondary tumors upon serial transplantation into secondary recipient mice.53 However, CD133 is also expressed by differentiated cells in different tissues and CD133- BTSCs can also initiate tumors in immunocompromised mice.54-55 Therefore, it remains to bedetermined if CD133 alone, or in combination with other markers, can be used to discriminate between tumor initiating cells and non-tumor initiating cells in different grades and types of brain tumors. Recently, FABP7 has gained traction as a CNS-specific marker of NSCs and BTSCs.42-43, 57

Both the neurosphere and adherent monolayer culture methods have been applied to the study of BTSCs. When culturing normal NSCs, the mitogen(s) EGF (and/or bFGF) are required to maintain NSC proliferation. However, there is some indication that these mitogens are not required when culturing BTSCs.57 Interestingly, the neurosphere assay may be a clinically relevant functional readout for the study of BTSCs, with emerging evidence suggesting that renewable neurosphere formation is a significant predictor of increased risk of patient death and rapid tumor progression in cultured human glioma samples.58-60 Furthermore, the adherent monolayer culture has been shown to enable pure populations of glioma-derived BTSCs to be expanded in vitro.61

Research in the field of NSC biology has made a significant leap forward over the past ~30 years. Contrary to the beliefs of the past century, the adult mammalian brain retains a small number of true NSCs located in specific CNS regions. The identification of CNS-resident NSCs and the discovery that adult somatic cells from mouse and human can be reprogrammed to a pluripotent state,62-68 and then directed to differentiate into neural cell types, has opened the door to new therapeutic avenues aimed at replacing lost or damaged CNS cells. This may include transplantation of neural progenitors derived from fetal or adult CNS tissue, or pluripotent stem cells. Recent research has shown that adult somatic cells can be directly reprogrammed to specific cell fates, such as neurons, using appropriate transcriptional factors, bypassing the need for an induced pluripotent stem cell intermediate.69 Astroglia from the early postnatal cerebracortex can be reprogrammed in vitro to neurons capable of action potential firing, by the forced expression of a single transcription factor, such as Pax6 or the pro-neural transcription factor neurogenin-2 (Neurog2).70 To develop cell therapies to treat CNS injuries and diseases, a greater understanding of the cellular and molecular properties of neural stem and progenitor cells is required. To facilitate this important research, STEMCELL Technologies has developed NeuroCult proliferation and differentiation kits for human, mouse and rat, including xenofree NeuroCult-XF. The NeuroCult NCFC Assay provides a simple and more accurate assay to enumerate NSCs compared to the neurosphere assay. These tools for NSC research are complemented by the NeuroCult SM Neuronal Culture Kits, specialized serum-free medium formulations for culturing primary neurons. Together, these reagents help to advance neuroscience research and assist in its transition from the experimental to the therapeutic phase.

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Swiss pharmaceutical giant Novartis made history as the first company to win FDA approval for a CAR-T therapy in the United States. Novartis announced that its genetically modified autologous (self-derived) immunocellular therapy, Kymriah, will cost $475,000 per treatment course. Shortly thereafter, Kite Pharma announced the approval of its CAR-T therapy, Yescarta, in the U.S. with a list price of $373,000. While these prices are expensive, they are far from trendsetting.

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Pricing of cell therapies is controversialbecause most cell therapy products are priced exponentially higher than traditional drugs. Unfortunately, most drugs can be manufactured and stockpiled in large quantities for off-the-shelf use, while cell therapies involve living cells that require a different approach to commercial-scale manufacturing, transit, stockpiling, and patient use.

To date, the highest priced treatment has not been a cell therapy, but a gene therapy (Glybera). At the time of its launch, Glybera was the first gene therapy approved in the Western world, launching for sale in Germany at a cost close to $1 million per treatment.[1] The record-breaking price tag got revealed in November 2014, when Uniqure and its marketing partner Chiesi, filed a pricing dossier with German authorities to launch Glybera. Unfortunately, Glybera was later withdrawn from the European market due to lack of sales.

Following the approval of Glybera, Kymriah, Yescarta, and more than a dozen other cell therapies, conversations surrounding pricing and reimbursement have become a focal point within the cell therapy industry.

In contrast to pharmaceutical drugs, cell therapies require a different pricing analysis. Below, price tags are shown for approved cell therapy products that have reached the market (prices in US$) and for which there is standardized market pricing.

Pricing of Approved Cell Therapy Products:

Apligrafby Organogenesis & Novartis AG in USA = $1,500-2,500 per use [2]Carticelby Genzyme in USA = $15,000 to $35,000 [3]Cartistemby MEDIPOST in S. Korea = $19,000-21,000 [4],[5]Cupistemby Anterogen in South Korea = $3,000-5,000 per treatment [6]ChondroCelectby Tigenix in EU = ~ $24,000 (20,000) [7]Dermagraftby Advanced Tissue Science in USA = $1,700 per application [8],[9]Epicelby Vericel in theUnited States = $6,000-10,000 per 1% of total body surface area [10]Hearticellgramby FCB-Pharmicell in South Korea = $19,000 [11]HeartSheetby Terumo in Japan = $56,000 (6,360,000) for HeartSheet A Kit; $15,000 (1,680,000) for HeartSheet B Kit (*Each administration uses one A Kit and 5 B Kits)[12]Holoclarby Chiesi Framaceutici in EU = Unknown (very small patient population)Kymriahby Novartis in USA = $425,000 per treatment[13]Osteocelby NuVasive in USA = $600 per cc [14],[15]Prochymalby Osiris Therapeutics and Mesoblast in Canada = ~ $200,000 [16]Provengeby Dendreon and Valeant Pharma in USA = $93,000 [17], [18]SpheroxbyCO.DON AG in EU = $9,500 $12,000 (8,000 10,000) per treatment[19]Strimvelisby GSK in EU = $665,000 (One of worlds most expensive therapies) [20],[21]Temcellby JCR Pharmaceuticals Co. Ltd. in Japan = $115,000-170,000 [22]*Pricing of TEMCELL is $7,600 (868,680 per bag), with one bag of 72m cells administered twice weekly and 2m cells/kg of body weight required per administration[23]Yescartaby Kite Pharma in USA =$373,000[24]

As shown in the list above, wound care products tend to have the lowest cell therapy pricing, typically costing $1,500 to $2,500 per use. For example, Apligrafis created from cells found in healthy human skin and is used to heal ulcers that do not heal after 3-4 weeks ($1,500-2,500 per use), and Dermagraftis a skin substitute that is placed on your ulcer to cover it and to help it heal ($1,700 per application).

Interestingly, Epicel is a treatment for deep dermal or full thickness burns comprising a total body surface area of greater than or equal to 30%. It has higher pricing of $6,000-10,000 per 1% of total body surface area, because it is not used to treat a single wound site, but rather used to treat a large surface area of the patients body.

Next, cartilage-based cell therapy products tend to have mid-range pricing of $10,000 to $35,000. For example, Carticelis a product that consists of autologous cartilage cells (pricing of $15,000 to $35,000), CARTISTEM is a regenerative treatment for knee cartilage (pricing of $19,000 to $21,000), and ChondroCelectis a suspension for implantation that contains cartilage cells (pricing of $24,000).In July 2017,the EMA in Europe also approved Spheroxas a product for articular cartilage defects of the knee with a pricing of$9,500 $12,000 (8,000 10,000) per treatment.

The next most expensive cell therapy products are the ones that are administered intravenously, which range in price from approximately $90,000 to $200,000. For example, Prochymal is an intravenously administered allogenic MSC therapy derived from the bone marrow of adult donors (pricing of $200,000), Provenge is an intravenously administered cancer immunotherapy for prostate cancer ($93,000), and Temcell is an intravenously administered autologous MSC product for the treatment of acute GVHD after an allogeneic bone marrow transplant (pricing of $115,000-170,000).

Finally, many of the worlds most expensive cell therapies are gene therapies, ranging in price from $500,000 to $1,000,000. For example, Kymriah is the first CAR-T cell therapy to be FDA approved in the United States (pricing of $475,00 per treatment course).Strimvelis isan ex-vivo stem cell gene therapy to treat patients with a very rare disease called ADA-SCID (pricing of $665,000).

Although these generalizations do not hold true for every cell therapy product, they explain the majority of cell therapy pricing and provide a valuable model for estimating cell therapy pricing and reimbursement. This information is summarized in the following table.

TABLE. Pricing Scale for Approved Cell Therapies

Another point of reference is also valuable. The RIKEN Institute launched the worlds first clinical trial involving an iPSC-derived product when it transplanted autologous iPSC-derived RPE cells into a human patient in 2014.While the trial was later suspended due to safety concerns, it resumed in 2016, this time using an allogeneic iPSC-derived cell product.

The research team indicated that by using stockpiled iPS cells, the time needed to prepare for a graft can be reduced from 11 months to as little as one month, and the cost, currently around 100 million ($889,100), can be cut to one-fifth or less.[25]

While many factors contribute to cell therapy pricing, key variables that can be used to predict market pricing include:

Another compounding factor is market size, because wound healing and cartilage replacement therapies have significant patient populations, while several of the more expensive therapies address smaller patient populations.[26]

To learn more about this rapidly expanding industry, view the Global Regenerative Medicine Industry Database Featuring 700+ Companies Worldwide.

What variable do you think influence the cost of cell therapies? Share your thoughts in the comments below.

BioInformant is the first and only market research firm to specialize in the stem cell industry. Our research has been cited by major news outlets that include the Wall Street Journal, Nature Biotechnology, Xconomy, and Vogue Magazine. Serving industry leaders that include GE Healthcare, Pfizer, Goldman Sachs, and Becton Dickinson. BioInformant is your global leader in stem cell industry data.

Footnotes[1] $1-Million Price Tag For Glybera Gene Therapy: Trade Secrets. Available at http://blogs.nature.com/tradesecrets/2015/03/03/1-million-price-tag-set-for-glybera-gene-therapy. Web. 21 Aug. 2017.[2] 2017 Apligraf Medicare Product and Related Procedure Payment, Organogenesis. Available at: http://www.apligraf.com/professional/pdf/PaymentRateSheetHospitalOutpatient.pdf. Web. 3 Mar. 2017.[3] CARTICEL (Autologous Chondrocyte Implantation, Or ACI). Available at: https://www.painscience.com/articles/cartilage-repair-with-carticel-review.php. Web. 3 Aug. 2017.[4] Cartistem?, What. What Is The Cost Of Cartistem? Available at: http://www.stemcellsfreak.com/2015/01/cartistem-price.html. N.p., 2017. Web. 3 Mar. 2017.[5] Cartistem. Kneeguru.co.uk. Available at: http://www.kneeguru.co.uk/KNEEtalk/index.php?topic=59438.0. Web. 3 Aug. 2017.[6] Stem Art, Stem Cell Therapy Pricing. Available at: http://www.stem-art.com/Library/Miscellaneous/SCT%20products%20%20Sheet%201.pdf. Web. 3 Mar. 2017.[7]Are Biosimilar Cell Therapy Products Possible? Presentation by Christopher A Bravery [PDF]. Available at: http://advbiols.com/documents/Bravery-AreBiosimilarCellTherapiesPossible.pdf. Web. 3 Aug. 2017.[8] Artificial Skin, Presentation by Nouaying Kue (BME 281). Available at: http://www.ele.uri.edu/Courses/bme281/F12/NouayingK_1.ppt. Web. 3 Mar. 2017.[9] Allenet, et al. Cost-effectiveness modeling of Dermagraft for the treatment of diabetic foot ulcers in the french context. Diabetic Metab. 2000 Apr;26(2):125-32.[10] Epicel Skin Grafts, Sarah Schlatter, Biomedical Engineering, University of Rhode Island. Available at: http://www.ele.uri.edu/Courses/bme281/F08/Sarah_1.pdf. Web. 31 July. 2017.[11] Nature. (2011). South Koreas stem cell approval. [online] Available at: http://www.nature.com/nbt/journal/v29/n10/full/nbt1011-857b.html. Web. 3 Sept. 2017.[12] Novick, Coline Lee. Translated version of the first two pages of Terumos Conditionally Approved HeartSheet NHI Reimbursement Price. [Twitter Post] Available at:goo.gl/YGCh6z. Web. 21 Sep. 2017.[13] Fortune.com. (2017). Is $475,000 Too High a Price for Novartiss Historic Cancer Gene Therapy? [online] Available at: http://fortune.com/2017/08/31/novartis-kymriah-car-t-cms-price/ Web. 8 Sept. 2017.[14] Skovrlj, Branko et al. Cellular Bone Matrices: Viable Stem Cell-Containing Bone Graft Substitutes. The Spine Journal 14.11 (2014): 2763-2772. Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4402977/. Web. April 12, 2017.[15] Hiltzik, Michael. Sky-High Price Of New Stem Cell Therapies Is A Growing Concern. Available at: http://www.latimes.com/business/hiltzik/la-fi-hiltzik-20151010-column.html. Web. 1 Sept. 2017.[16] Counting Coup: Is Osiris Losing Faith In Prochymal?, Busa Consulting LLC. Available at: http://busaconsultingllc.com/scsi/organelles/counting_coup_prochymal.php. Web. 3 Aug. 2017.[17] Dendreon Sets Provenge Price At $93,000, Says Only 2,000 People Will Get It In First Year | Xconomy. Available at: http://www.xconomy.com/seattle/2010/04/29/dendreon-sets-provenge-price-at-93000-says-only-2000-people-will-get-it-in-first-year/. Web. 3 Mar. 2017.[18] Dendreon: Provenge To Cost $93K For Full Course Of Treatment | Fiercebiotech. Available at: http://www.fiercebiotech.com/biotech/dendreon-provenge-to-cost-93k-for-full-course-of-treatment. Web. 3 Mar. 2017.[19]Warberg Research.CO.DON (CDAX, Health Care). Available at:http://www.codon.de/fileadmin/assets/pdf/03_Investor/Research_Report/2017_07_24_CO.DON_Note_Warburg_Research_englisch.pdf. Web. 21 Sept. 2017.[20] GSK Inks Money-Back Guarantee On $665K Strimvelis, Blazing A Trail For Gene-Therapy Pricing | Fiercepharma. Available at: http://www.fiercepharma.com/pharma/gsk-inks-money-back-guarantee-665k-strimvelis-blazing-a-trail-for-gene-therapy-pricing. Web. 3 Mar. 2017.[21] Strimvelis. Wikipedia.org. Available at: https://en.wikipedia.org/wiki/Strimvelis. Web. 13 Aug. 2017.[22] MesoblastS Japan Licensee Receives Pricing For TEMCELL HS Inj. For Treatment Of Acute Graft Versus Host Disease. Mesoblast Limited, GlobeNewswire News Room. Available at: https://globenewswire.com/news-release/2015/11/27/790909/0/en/Mesoblast-s-Japan-Licensee-Receives-Pricing-for-TEMCELL-HS-Inj-for-Treatment-of-Acute-Graft-Versus-Host-Disease.html. Web. 3 Mar. 2017.[23]TEMCELL HS Inj. Receives NHI Reimbursement Price Listing, JCR Pharmaceuticals Co., Ltd. News Release, November 26, 2015. Available at: http://www.jcrpharm.co.jp/wp2/wp-content/uploads/2016/01/ir_news_20151126.pdf. Web. 3 Mar. 2017.[24]Kites Yescarta (Axicabtagene Ciloleucel) Becomes First CAR T Therapy Approved by the FDA for the Treatment of Adult Patients With Relapsed or Refractory Large B-Cell Lymphoma After Two or More Lines of Systemic Therapy. Business Wire.Web. 19 Oct. 2017.[25]Riken-Linked Team Set To Test Transplanting Eye Cells Using Ips From Donor | The Japan Times. The Japan Times. N.p., 2017. Web. 23 July. 2017.[26]LinkedIn Comment, by David Caron. Available at: https://www.linkedin.com/feed/update/urn:li:activity:6316277496551665664/. Web. 21 Sept. 2017.

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