Stem Cell Clinic

Heard On Sundial: Reopening Miami-Dade And The Keys, And Stem Cell Treatment For Coronavirus – WLRN

On this Wednesday, May 6, episode of Sundial:

What would reopening look like in Miami-Dade County?

The results of Miami-Dade Countys reopening of parks, marinas and golf courses have been mixed.

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Over the weekend, hundreds attempted to access boat ramps across South Dade and many were turned away. Also, thousands of people were cited at Miami Beachs South Pointe Park for not wearing protective gear. Officials later closed the park because so few people were adhering to the guidelines.

Listen to today's full show.

"We need to continue testing and retesting and that is going to be crucial to the next stage, opening the county," says Miami-Dade County Commissioner Esteban Bovo, who represents parts of Hialeah and Miami Lakes. "If we don't police ourselves we're going to continue in this cycle."

He joined Sundial to talk with host Luis Hernandez about the possibility of incentivizing residents to take more precautions during the global pandemic.

The Florida Keys have reopened.

Certain businesses like retail shops and restaurants in the Florida Keys were granted permission to reopen on Monday, but to locals only. Monroe County is still closed to visitors.

"It's a cautious start and a good start. I think people are excited to have options," says Rep. State Holly Raschein, R-Key Largo.

The county's checkpoint will remain in place until further notice. It only allows vehicles carrying Keys residents, property owners, workers and deliveries. Screenings will also continue at the Key West International and Florida Keys Marathon International airports.

Read more: Checking In On Keys Checkpoint: Monroe Emergency Management Chief Says 'It's Working'

Raschein, also the chairwoman of the House Agriculture and Natural Resources Appropriations Subcommittee, joined Sundial to discuss the impact of COVID-19 on the Florida Keys economy.

Stem cell treatment for coronavirus using umbilical cords.

Doctors and researchers are working hard to develop antiviral medication amid the coronavirus pandemic. In South Florida, a new coronavirus treatment that uses stem cells from umbilical cords is being tested now.

Dr. Camillo Ricordi, the Director of the Diabetes Research Institute and the Cell Transplant Center at the University of Miami Miller School of Medicine, joined Luis Hernadez to talk about how this treatment may help those sick with coronavirus.

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Heard On Sundial: Reopening Miami-Dade And The Keys, And Stem Cell Treatment For Coronavirus - WLRN

Researchers Convert Astrocytes to Neurons In Vivo to Treat… : Neurology Today – LWW Journals

Article In Brief

A mouse study shows that select transcription factors to the striatum can effectively and safely convert astrocytes to neurons to treat Huntington's disease.

Delivering two transcription factors to the striatum in a mouse model of Huntington's disease can safely convert astrocytes into neurons with high efficiency, according to a new study in the February 27 issue of Nature Communications.

The neurons grow to and wire up with their targets in the globus pallidus and substantia nigra, and remaining astrocytes proliferate to replace those that have been converted. The treatment extends the lifespan and improves the motor behavior of the mice.

What is exciting about this study is that the authors have clearly made cells that do what they are supposed to do, namely replace dying neurons in existing circuits, said Roger Barker, PhD, professor of clinical neuroscience and honorary consultant in neurology at the University of Cambridge and at Addenbrooke's Hospital, who was not involved in the work. I think the challenge of scaling up this strategy to the human Huntington's disease brain is pretty substantial, but nonetheless, this is an important discovery.

The new study, led by Gong Chen, PhD, builds on discoveries beginning in the mid-2000s showing that a small number of exogenously applied transcription factors could transform skin fibroblasts into stem cells, which could then be further converted to become virtually any cell type. That discovery was quickly followed by advances in direct reprogramming, in which one cell type is directly converted into another, skipping the stem cell intermediate.

Most of that work has taken place in vitro, and most attempts to use the strategy therapeutically have depended on transplantation of stem cells or newly converted cells.

We tried stem cell transplants to the mouse brain 10 years ago, but we couldn't find a lot of functional neurons, said Dr. Chen, professor at Guangdong-Hong Kong-Macau Institute of CNS Regeneration of Jinan University in Guangzhou, China.

It was also clear that anything you do in vitro, you eventually have to transplant, and that didn't seem to be a very promising technology, so I said, Let's try this in vivo, and put transcriptions factors directly into the mouse brain.

Dr. Chen initially tried introducing the transcription factor neurogenin 2, but the efficiency of conversion of astrocytes to neurons was very low, so he turned to the transcription factor NeuroD1, which Dr. Chen's group had previously shown could convert astrocytes into excitatory glutamatergic neurons.

In the current study, in order to generate GABAergic neurons, the team combined NeuroD1 with another transcription factor, D1x2, based on previous work showing its importance for generating GABAergic neurons.

The team packed the genes for the transcription factors into a recombinant adeno-associated virus vector (rAAV 2/5) and used an astrocyte-specific promoter to drive the transgene expression so that it preferentially expresses in astrocytes. They first injected the vector into the normal mouse striatum.

Surprisingly, this strategy worked very well at high efficiency, Dr. Chen said. After seven days, all transfected cells expressed astrocyte markers, indicating a high level of specificity in the vector. Of those cells, 81 percent co-expressed the two transcription factors. By 30 days, 73 percent of the cells expressing the transcription factors now expressed neuronal, rather than astrocytic markers, and were primarily GABAergic in character.

Next, Dr. Chen asked whether the remaining astrocytes could repopulate to replace those lost to conversion. Using immunostaining for astrocytes and neurons, as well as other techniques, the team found that the neuron/astrocyte ratio was unchanged, and that some remaining astrocytes could be found at different stages of cell division, suggesting the process facilitated astrocyte proliferation.

Dr. Chen then turned to the R6/2 mouse, the most common mouse model of Huntington's disease. He treated mice at 2 months of age, just as they began to show motor symptoms

As in the wild-type mice, astrocytes were converted to GABAergic neurons at high efficiency without altering the neuron/astrocyte ratio. The researchers observed similar results in a less-severe HD mouse model as well. Treated mice had only about half the degree of striatal atrophy as untreated mice. The converted neurons still contained aggregated huntingtin protein, but less than in native neurons, and similar to the reduced amount found in astrocytes in the mouse brain.

The real test of any cell therapy in neurodegenerative disease is whether the new cells can link into the existing circuits and provide functional benefit, feats that have been hard to achieve with transplanted fetal cells or stem cells.

Examining striatal slices from the treated mice, Dr. Chen found that the converted neurons displayed electrical properties largely identical to those of normal neurons, including resting potential, action potential threshold, firing amplitude, and firing frequency. They integrated into local circuits and behaved similarly to the native neurons around them. By tracking a marker contained in the AAV gene construct, they showed that converted neurons projected axons to the two basal ganglia targets of medium spiny neurons in the striatum, the globus pallidus and the substantia nigra.

Finally, Dr. Chen found that stride length and travel distance were both significantly improved in treated mice, though still falling below those of wild-type mice, and lifespan was significantly extended.

There were no hints of tumors in the mice, Dr. Chen noted. He suggested that in situ conversion is likely intrinsically safer in this regard than using stem cell-derived neurons, since a proliferative astrocyte is being converted into a non-proliferative neuron, with no residual pool of unconverted and potentially tumorigenic stem cells. We are actually reducing the tumor risk, he said.

Why the converted neurons developed appropriate neuronal connections is an important unanswered question, Dr. Chen said. He suggested there were two important factorsfirst, the astrocytes from which they arose are likely developmentally related to neighboring neurons, and thus may express similar position markers that help guide them to the right targets, just like the native neurons. Second, those remaining neurons may also provide guide tracks for the newly growing axons.

This conversion technique is not limited to Huntington's disease, he stressed, noting that his team last year published a paper showing promise in ischemic stroke, and work is underway to test its potential in Alzheimer's disease, Parkinson's disease, spinal cord injury, and ALS. He is also moving on to testing in non-human primates, setting the stage for eventual human trials.

I think eventually we will want to correct the Huntington's mutation as well, Dr. Chen said, for instance by using CRISPR, but he pointed out that while that strategy can repair diseased neurons, it cannot make new ones, like astrocyte-to-neuron conversion can.

This study is really elegantly done, commented Veronica Garcia, PhD, who has studied astrocytes derived from induced pluripotent stem cells from Huntington's disease patients as a postdoctoral scientist working with Clive Svendsen, PhD, in the Regenerative Medicine Institute at Cedars-Sinai Medical Center in Los Angeles.

The conversion efficiency is similar between wild-type and disease models, suggesting that the disease process is not interfering with the conversion, she said.

Astrocyte depletion does not seem to be a problem, at least in the short term, but Dr. Garcia noted there is a limit on the number of divisions astrocytes appear able to undergo, after which they lose the ability to proliferate. That may be a problem for chronic treatment, she suggested. Nonetheless, these results really look promising for therapeutic development.

The concept of trying to reprogram cells in situ to take on the phenotype of the cells that are lost is not new, commented Dr. Barker, but being able to do it with any degree of efficiency, to make enough cells to make a significant difference, has been problematic. For that reason, and because the cells grow to their target sites and make connections, these results are surprising.

A major hurdle for clinical trials, he noted, will be scaling up to the human striatum, which has approximately 100 times the volume of that in the mouse. Delivering the vector to such a large volume will be a significant challenge, he said, along with determining whether this approach will really work in a disease that affects many different brain structures such as in HD.

Dr. Chen is co-founder of NeuExcell Therapeutics Inc, which will develop clinical trials in the future. Drs. Barker and Garcia disclosed no conflicts.

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Researchers Convert Astrocytes to Neurons In Vivo to Treat... : Neurology Today - LWW Journals

BrainStorm Leases a New Cleanroom Facility at The Tel Aviv Sourasky Medical Center to Manufacture NurOwn for The European Union – GlobeNewswire

NEW YORK, N.Y., and TEL AVIV, Israel, May 07, 2020 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, announced today a lease agreement with the Tel Aviv Sourasky Medical Center (Sourasky)in Tel Aviv, Israel, to produce NurOwn in three state-of-the-art cleanrooms. The new facility will significantly increase the Companys capacity to manufacture and ship its product into the European Union and the local Israeli market. The cleanroom facility is part of Souraskys Institute for Advanced Cellular Therapies.

"Sourasky Hospital is a leader in the advancement and manufacturing of cell and gene therapy products and is well-equipped to rapidly scale up and produce NurOwn," stated Prof. Ronni Gamzu, CEO of Tel Aviv Sourasky Medical Center. "We look forward to continuing our work with BrainStorm to bring NurOwn to ALS patients and help fulfill the clinical therapy demands for the Companys pipeline programs.

"Sourasky Hospital, known for introducing pioneering solutions into clinical practice and advancing patient care, has a first rate team with the proven experience to produce regenerative products in accordance to the highest standard of cGMP manufacturing," said Chaim Lebovits, CEO of BrainStorm. "This agreement will ensure that we can provide NurOwn to patients after regulatory approval, not only in Israel but we have secured capacity to rapidly scale up production as we advance our investigational treatment across the European Union. We are very pleased to be able to expand our ongoing collaboration with Sourasky Hospital, one of the worlds most innovative and respected medical centers."

About NurOwn NurOwn (autologous MSC-NTF) cells represent a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. BrainStorm has fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also recently received U.S. FDA acceptance to initiate a Phase 2 open-label multicenter trial in progressive MS and enrollment began in March 2019.

About BrainStorm Cell Therapeutics Inc. BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six U.S. sites supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm also recently received U.S. FDA clearance to initiate a Phase 2 open-label multicenter trial in progressive Multiple Sclerosis. The Phase 2 study of autologous MSC-NTF cells in patients with progressive MS (NCT03799718) started enrollment in March 2019. For more information, visit the company's website at http://www.brainstorm-cell.com

Safe-Harbor Statement Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

CONTACTS

Investor Relations:Preetam Shah, MBA, PhDChief Financial OfficerBrainStorm Cell Therapeutics Inc.Phone: + 1.862.397.1860pshah@brainstorm-cell.com

Media:Sean LeousWestwicke/ICR PRPhone: +1.646.677.1839sean.leous@icrinc.com

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BrainStorm Leases a New Cleanroom Facility at The Tel Aviv Sourasky Medical Center to Manufacture NurOwn for The European Union - GlobeNewswire

Results From Pivotal Phase 2/3 Study of Emapalumab in Patients With Primary HLH Published in New Eng – PharmiWeb.com

STOCKHOLM, May 7, 2020 /PRNewswire/ -- Sobi announced today that the results from the pivotal phase 2/3 study evaluating the efficacy and safety of emapalumab in patients with primary haemophagocytic lymphohistiocytosis (HLH) were published in the New England Journal of Medicine on 7 May 2020. Emapalumab is the first therapy approved by the US Food & Drug Administration (FDA) for primary HLH and is under review by the European Medicines Agency (EMA).

Primary HLH is a rare syndrome that typically presents in infancy but can also be seen in adults and is associated with high morbidity and mortality. This life-threatening disease is characterised by immune dysregulation and uncontrolled hyperinflammation. The treatment objective is to suppress the hyperinflammation and control the acute features of the disease in order to successfully bring patients to haematopoietic stem cell transplantation (HSCT).

"The publication of the results in this highly respected medical journal is a testament to the medical importance of the emapalumab findings for patients with primary HLH," says Milan Zdravkovic, Head of Research & Development and Chief Medical Officer at Sobi. "The results further advance our understanding of primary HLH and the role of interferon gamma in its pathogenesis. Our hope is to contribute to the improvement of care and treatment for patients suffering from this potentially fatal disease."

The results with emapalumab in primary HLH published in the New England Journal of Medicine highlight the overall response rate of 63 percent in previously treated patients at the end of up to 8 weeks of treatment (compared to the pre-specified null hypothesis of 40 percent (p=0.02)). In the previously treated group, 70 percent of patients were able to proceed to transplantation. The most commonly reported adverse reactions ( 20 per cent) were infections, hypertension, infusion-related reactions and fever.

Michael Jordan, Professor of Pediatrics at the Cincinnati Children's Hospital Medical Center in the US and coordinating Principal Investigator of the study (US), confirms the importance of making advances in finding new therapies for HLH and emphasises the importance of the publication of the results: "The findings from the study are encouraging for those affected by this devastating disease."

Professor Franco Locatelli, Head of the Department of Onco-Haematology, Bambino Ges Children's Hospital IRCCS, Sapienza University of Rome, Italy, and coordinating Principal Investigator (EU), adds: "Emapalumab represents a prototype model molecularly targeted therapy and an important step towards improving outcomes for this severe and life-threatening disease."

This pivotal clinical study is the first study in primary HLH to prospectively assess and report treatment responses using predefined comprehensive objective clinical and laboratory criteria. Preclinical data have shown the central role of interferon gamma (IFN) in the pathogenesis of this disease1. Emapalumab is a monoclonal antibody that binds to and neutralises IFN. It was approved by the US Food & Drug Administration (FDA) on the basis of this clinical study for the treatment of primary HLH in adult and paediatric (newborn and older) patients with refractory, recurrent or progressive disease, or intolerance to conventional HLH therapy, and received Breakthrough Designation prior to review.

About emapalumab

Emapalumab is a monoclonal antibody that binds to and neutralises interferon gamma (IFN). In the US, emapalumab is indicated for paediatric (newborn and older) and adult primary haemophagocytic lymphohistiocytosis (HLH) patients with refractory, recurrent or progressive disease, or intolerance to conventional HLH therapy. Emapalumab is the first and only medicine approved in the US for primary HLH, a rare syndrome of hyperinflammation that usually occurs within the first year of life and can rapidly become fatal unless diagnosed and treated. The FDA approval is based on data from the phase 2/3 studies (NCT01818492 and NCT02069899). Emapalumab is indicated for administration through intravenous infusion over one hour twice per week until haematopoietic stem cell transplantation (HSCT). For more information please see http://www.gamifant.com including the full US Prescribing Information. Emapalumab is under review for primary HLH by the European Medicines Agency (EMA).

About SobiTM

Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Sobi is providing sustainable access to innovative therapies in the areas of haematology, immunology and specialty indications. Today, Sobi employs approximately 1,400 people across Europe, North America, the Middle East, Russia and North Africa. In 2019, Sobi's revenues amounted to SEK 14.2 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. You can find more information about Sobi at sobi.com.

For more information please contactPaula Treutiger, Head of Communication & Investor Relations+ 46-733-666-599paula.treutiger@sobi.com

Linda Holmstrm, Corporate Communication & Investor Relations+46-708-734-095linda.holmstrom@sobi.com

1. Jordan et al. Blood 2004;104:735-43.

This information was brought to you by Cision http://news.cision.com

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Results From Pivotal Phase 2/3 Study of Emapalumab in Patients With Primary HLH Published in New Eng - PharmiWeb.com

Characterization and Immunomodulation of Canine Amniotic Membrane Stem | SCCAA – Dove Medical Press

Alessandra de Oliveira Pinheiro,1 Valria M Lara,1 Aline F Souza,1 Juliana B Casals,2 Fabiana F Bressan,1 Paulo Fantinato Neto,1 Vanessa C Oliveira,1 Daniele S Martins,1 Carlos E Ambrosio1

1Department of Veterinary Medicine, Faculty of Animal Science and Food Engineering, University of So Paulo, Pirassununga, So Paulo, Brazil; 2Private Veterinary Practice, Pirassununga, So Paulo, Brazil

Correspondence: Carlos E AmbrosioDepartment of Veterinary Medicine, Faculty of Animal Science and Food Engineering, University of So Paulo, FZEA- Av. Duque de Caxias Norte, 225, ZMV, Pirassununga 13635-900, So Paulo, BrazilTel +55 19 3565-4113 Email ceambrosio@usp.br

Purpose: Amniotic membrane stem cells have a high capacity of proliferation, cell expansion, and plasticity, as well as immunomodulatory properties that contribute to maternal-fetal tolerance. Owing to the lack of research on human amniotic membrane at different gestational stages, the canine model is considered ideal because of its genetic and physiological similarities. We aimed to characterize the canine amniotic membrane (CAM) cell lineage in different gestational stages and evaluate the expression of immunomodulatory genes.Materials and Methods: Twenty CAMs from early (20 30 days) (n=7), mid- (31 45 days) (n=7), and late gestation (46 63 days) (n=6) stages were studied. The cell features were assessed by cell viability tests, growth curve, colony-forming units, in vitro differentiation, cell labeling for different immunophenotypes, and pluripotent potential markers. The cells were subjected to RT-PCR and qPCR analysis to determine the expression of IDO, HGF, EGF, PGE2, and IL-10 genes.Results: CAM cells exhibited a fibroblastoid morphology and adherence to plastic with an average cell viability of 78.5%. The growth curve indicated a growth peak in the second passage and we obtained an average of 138.2 colonies. Osteogenic, chondrogenic, and adipogenic lineages were confirmed by in vitro differentiation assays. Cellular immunophenotyping experiments confirmed the presence of positive mesenchymal markers (CD90 and CD105) and the low or negative expression of hematopoietic markers (CD45 and CD34). Qualitative analysis of the immunomodulatory functions indicated the expression of the IDO, HGF, EGF5, and PGE2 genes. When stimulated by interferon-gamma, CAM cells exhibited higher IDO levels throughout gestation.Conclusion: The CAMs from different gestational stages presented features consistent with mesenchymal stem cell lineage; better results were observed during the late gestation stage. Therefore, the gestational stage is a key factor that may influence the functionality of therapies when using fetal membrane tissues from different periods of pregnancy.

Keywords: canine stem cells, immunomodulation, fetal annexes

This work is published by Dove Medical Press Limited, and licensed under a Creative Commons Attribution License.The full terms of the License are available at http://creativecommons.org/licenses/by/4.0/.The license permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.

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Characterization and Immunomodulation of Canine Amniotic Membrane Stem | SCCAA - Dove Medical Press

Navigating cancer as a young adult: ‘I’m trying to figure out who I am’ – Stanford Medical Center Report

When David Llano was diagnosed with leukemia in June 2014, the news came as a shock.

Then 17, Llano had just finished his junior year of high school, and he was looking forward to a summer of hanging out with friends. Instead, he was immediately hospitalized at Lucile Packard Children's Hospital Stanford.

For the next few months, he was extremely ill, as he received chemotherapy treatments and eventually a stem cell transplant.

I wrote about his experience in a feature story for Stanford Medicine magazine. As the article explains, teens and young adults with cancer face biological and psychosocial challenges distinct from those of other cancer patients, both in treatment and as they recover.

During my reporting, I spoke with Stanford child and adolescent psychologist Emily Ach, PhD, who frequently works with teens who have cancer.

"The rift between the patient's experience and what's happening with their peer group is a real challenge to figure out," she told me.

Llano said he felt this rift when he returned to high school four months after his diagnosis. From the story:

The first day back at school was harder -- weirder, really -- than he expected.

"People I didn't even know would go up to me and hug me, be really touchy with me," he said. "All the kids pitied me."

His experience isn't unusual, psychologist Ach said. "Maybe at college reentry, kids are more sensitive and appropriate, but not necessarily," she said. "In high school, they're pretty reliably not, and being different in any way is really hard."

When Ach is helping patients prepare to return to school, she reminds them that although many aspects of having cancer are outside of their control, they get to choose how much information about their illness to share.

"Kids vary widely in terms of how open they want to be," Ach told me. For instance, some teens are comfortable responding to a question about a scar by saying, "Oh, I was treated for leukemia. That's where I had a port for my chemo." Others might prefer to say, "I don't really want to talk about it. I was sick, and I had a medical procedure."

Regardless of the specifics, planning responses for awkward questions helps, Ach said. "For a lot of kids, coming up with that on the spot is really hard, and the potential to be caught off-guard is so anxiety-provoking," she said.

Like many young cancer patients, Llano initially had trouble seeing what his future would hold: "You don't really have an identity," he said. "You're like, 'I have cancer and I'm trying to figure out who I am.'"

He kept up a few close friendships from before his illness, but decided to complete his senior year of high school at the hospital school at Packard Children's. There, other students had their own challenging medical journeys and wouldn't be surprised by what he'd been through.

Llano also became active in peer advising at the hospital, helping other teens navigate the challenges of cancer.

He is now in good health and planning a career as a child-life specialist.

Photography, including image of cancer survivor David Llano at his Sunnyvale home, by Timothy Archibald

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Navigating cancer as a young adult: 'I'm trying to figure out who I am' - Stanford Medical Center Report

Lineage Cell Therapeutics Reports New Data With OpRegen for the Treatment of Dry AMD With Geographic Atrophy – BioSpace

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, today announced that updated results from a Phase I/IIa study of its lead product candidate, OpRegen, a retinal pigment epithelium (RPE) cell transplant therapy currently in development for the treatment of dry age-related macular degeneration (AMD), were published online via the ARVOLearn platform as part of the 2020 Association for Research in Vision and Ophthalmology (ARVO) Meeting. The presentation entitled, Phase I/IIa Clinical Trial of Human Embryonic Stem Cell (hESC)-Derived Retinal Pigmented Epithelium (RPE, OpRegen) Transplantation in Advanced Dry Form Age-Related Macular Degeneration (AMD): Interim Results (Abstract # 3363764), was presented by Christopher D. Riemann, M.D., Vitreoretinal Surgeon and Fellowship Director, Cincinnati Eye Institute (CEI) and University of Cincinnati School of Medicine. Dr. Riemanns presentation is available on the Media page of the Lineage website. Lineage will also host a live call with Dr. Riemann, on Monday, May 11, 2020 at 5:00 p.m. ET/2:00 p.m. PT to further discuss the results of treatment with OpRegen. Interested parties can access the call on the Events and Presentations section of Lineages website.

This update is significant as it builds on our earlier reports of gains in visual acuity and provides a more comprehensive picture of treatment with OpRegen for dry AMD, with meaningful improvements in the progression of geographic atrophy, visual acuity, and reading speed observed in our first Cohort 4 patient and first Orbit SDS with thaw-and-inject formulation dosed patient, stated Brian M. Culley, Lineage CEO. As dry AMD is a slow and progressive disease, it takes many months to observe changes to retinal anatomy or visual acuity. With the benefit of longer follow-up, we now can report that some OpRegen treated patients are able to see better, have less growth in their area of GA, and are able to read faster, all of which represent significant enhancements to vision and quality of life metrics. In addition to these individual results, the pooled data continues to suggest a treatment effect in both visual acuity and GA progression. Notably, we also are reporting additional evidence that OpRegen cells remain present for at least 4 years and hope that longer follow-up periods will reinforce a growing body of evidence that OpRegen is well-tolerated and can provide sustained and clinically meaningful benefits with a single dose of RPE cells. Our near-term objective is to treat and monitor the final four patients in Cohort 4 of the current study and utilize these data to direct our clinical, regulatory, and partnership discussions. Our goal is to combine the best cell line, the best production process, and the best delivery system, to position OpRegen as the front-runner in the race to address the unmet need in the potential billion-dollar dry AMD market.

As a principal investigator on the OpRegen clinical study, I am excited to present this most recent update, where all Cohort 4 patients treated with OpRegen had improved Best Corrected Visual Acuity up to one year or at their last visit, demonstrating a substantial treatment response, stated Christopher D. Riemann, M.D. The pooled Cohort 4 data demonstrate a significant, greater than 10-letter sustained visual acuity improvement over the entire followup period. Reading center assessments of GA also suggest a reduction in GA progression in the OpRegen treated eye when compared to fellow eye in Cohort 4. I am encouraged by the results observed in patients treated to date with OpRegen and I look forward to dosing patients in this study at CEI.

KOL Call Information and Webcast

Lineage will host a conference call with Dr. Riemann, on Monday, May 11, 2020 at 5:00 p.m. ET/2:00 p.m. PT to further discuss the results following treatment with OpRegen. A live webcast of the conference call will be available online in the Events and Presentations section of Lineages website. Interested parties may also access the conference call by dialing (866) 888-8633 from the U.S. and Canada and (636) 812-6629 from elsewhere outside the U.S. and Canada and should request the Lineage Cell Therapeutics Call. A replay of the webcast will be available on Lineages website for 30 days and a telephone replay will be available through May 19, 2020, by dialing (855) 859-2056 from the U.S. and Canada and (404) 537-3406 from elsewhere outside the U.S. and Canada and entering conference ID number 6597936.

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 robust proprietary cell-based therapy platform and associated in-house 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 to either 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 programs are in markets with billion dollar opportunities and include three allogeneic (off-the-shelf) product candidates: (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase 1/2a 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 1/2a development for the treatment of acute spinal cord injuries; and (iii) VAC2, a cancer immunotherapy of antigen-presenting dendritic cells in Phase 1 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 Lineages objectives with respect to OpRegen. 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 in Lineages filings with the Securities and Exchange Commission (the 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 with the SEC, including Lineages Annual Report on Form 10-K filed with the SEC on March 12, 2020 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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Lineage Cell Therapeutics Reports New Data With OpRegen for the Treatment of Dry AMD With Geographic Atrophy - BioSpace