Steve Hunt’s untimely death shines light on rare, incurable disease – VVdailypress.com

In my wildest dreams, I never imagined I would be a widow at age 57. But here I am, alone and without my beloved husband Steve Hunt, former president, publisher and editor of the Victorville Daily Press. It all feels like a bad dream that I am going to wake up from at any minute.

How did this happen so fast? How did a man with robust health become fatally ill almost overnight with what doctors diagnosed as congestive heart failure (CHF)? Steve, 64, had no risk factors for this condition. He took great care of himself, too, always choosing nutritious foods and opting for natural supplements to cure his minor aches and pains rather than prescription meds.

About a month before Steve died, I came across some information about a disease called amyloidosis. Its extremely rare with only 4,500 cases diagnosed every year, according to the Amyloidosis Foundation.

Among the celebrities that suffer with amyloidosis are actor Michael York, NBA Hall of Famer Nate Tiny Archibald and Matt Millen, a four-time Super Bowl champion and broadcaster.

In a nutshell, the disease (which is not cancer) is caused by abnormal amyloid proteins which are produced in the bloodstream. The proteins build up in vital organs and essentially shut them down. So far, researchers have been unable to link a direct cause to the condition.

Symptoms include swelling of ankles and legs, fatigue and weakness, shortness of breath, irregular heartbeat and a number of other things.

Steve's symptoms were a near-perfect match to amyloidosis and suddenly, things began to make sense. But sadly, there is no cure for the condition and without treatment, the mortality rate is about one year from the onset of symptoms

The treatment plan for Steve included diuretics to get rid of the fluid buildup in his lungs, legs and feet, which is caused by the declining function of the heart. He also took potassium to keep his levels in check, as the diuretics often cause potassium to drop. His cardiologist opted to skip putting him on medication that would slow down CHF because the pills tend to lower your blood pressure and Steves BP was consistently running very low, sometimes registering a top number in the 70s.

Beyond low blood pressure, my husband also developed a host of other strange symptoms that seemed unrelated to CHF. He was losing fat and muscle mass rapidly, had a swollen tongue and was having trouble swallowing. Intermittently, red blotches appeared on his face and neck that looked like giant, broken blood vessels.

At that point, Steve could have undergone chemotherapy treatments or a stem cell transplant that likely would have prolonged his life.

By the time this came to our attention, his heart and other organs were damaged beyond repair. He also was much too weak to travel to one of the few hospitals in the country that treat the disease, such as the Mayo Clinic or Stanford University Medical Center.

A couple days before Steve died, he asked me to share his story hoping it would help others by bringing greater awareness about amyloidosis.

I pray that one day soon a cause will be identified, and a cure will be found for this dreadful malady.

Donations to the Amyloidosis Foundation can be made on Steves behalf at http://www.amyloidosis.org.

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Steve Hunt's untimely death shines light on rare, incurable disease - VVdailypress.com

Global Induced Pluripotent Stem Cells (iPSCs) Market 2019 Analysis & Forecast Report 2024 – Analytics News

According to this study, over the next five years the Induced Pluripotent Stem Cells (iPSCs) market will register a 12.7% CAGR in terms of revenue, the global market size will reach US$ 13910 million by 2024, from US$ 11320 million in 2019. In particular, this report presents the global market share (sales and revenue) of key companies in Induced Pluripotent Stem Cells (iPSCs) business, shared in Chapter 3.

This report presents a comprehensive overview, market shares, and growth opportunities of Induced Pluripotent Stem Cells (iPSCs) market by product type, application, key manufacturers and key regions and countries.

Research report is delivering the complete study of different factors for market growth and various factors are showing the significant responsibility in the growth of market during the forecast period. Hence, report is competently delivering the comprehensive study about the revenue considering share, production and price. Thus offering the outline of different segmentations about region, taking into the consideration the production details and revenue impacting the growth.

Request a sample report here: https://www.orbisresearch.com/contacts/request-sample/3685986

This study considers the Induced Pluripotent Stem Cells (iPSCs) value and volume generated from the sales of the following segments:

Segmentation by product type: breakdown data from 2014 to 2019, in Section 2.3; and forecast to 2024

Human iPSCsMouse iPSCs

Human iPSCs had a market share of 89% in 2018, followed by Mouse iPSCs.

Segmentation by application: breakdown data from 2014 to 2019, in Section 2.4; and forecast to 2024

Academic ResearchDrug Development and DiscoveryToxicity ScreeningRegenerative Medicine

Academic Research is the largest segment of Induced Pluripotent Stem Cells (iPSCs) application,with a share of 32% in 2018.

This report also splits the market by region: Breakdown data in Chapter 4, 5, 6, 7 and 8.

AmericasUnited StatesCanadaMexicoBrazilAPACChinaJapanKoreaSoutheast AsiaIndiaAustraliaEuropeGermanyFranceUKItalyRussiaSpainMiddle East & AfricaEgyptSouth AfricaIsraelTurkeyGCC Countries

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The report also presents the market competition landscape and a corresponding detailed analysis of the major vendor/manufacturers in the market. The key manufacturers covered in this report: Breakdown data in in Chapter 3.

Fujifilm Holding Corporation (CDI)NcardiaAstellas Pharma IncFate Therapeutics, IncSumitomo Dainippon PharmaReproCELLPluricell BiotechCell Inspire Biotechnology

In addition, this report discusses the key drivers influencing market growth, opportunities, the challenges and the risks faced by key manufacturers and the market as a whole. It also analyzes key emerging trends and their impact on present and future development.

Research objectives

To study and analyze the global Induced Pluripotent Stem Cells (iPSCs) consumption (value & volume) by key regions/countries, product type and application, history data from 2014 to 2018, and forecast to 2024.

To understand the structure of Induced Pluripotent Stem Cells (iPSCs) market by identifying its various subsegments.

Focuses on the key global Induced Pluripotent Stem Cells (iPSCs) manufacturers, to define, describe and analyze the sales volume, value, market share, market competition landscape, SWOT analysis and development plans in next few years.

To analyze the Induced Pluripotent Stem Cells (iPSCs) with respect to individual growth trends, future prospects, and their contribution to the total market.

To share detailed information about the key factors influencing the growth of the market (growth potential, opportunities, drivers, industry-specific challenges and risks).

To project the consumption of Induced Pluripotent Stem Cells (iPSCs) submarkets, with respect to key regions (along with their respective key countries).

To analyze competitive developments such as expansions, agreements, new product launches, and acquisitions in the market.

To strategically profile the key players and comprehensively analyze their growth strategies.

Table of Contents

1 Scope of the Report1.1 Market Introduction1.2 Research Objectives1.3 Years Considered1.4 Market Research Methodology1.5 Economic Indicators1.6 Currency ConsideredContinued

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Gene regulators work together for oversized impact on schizophrenia risk – National Institutes of Health

News Release

Monday, September 23, 2019

Modeled gene expression changes match those found in patients brains.

Researchers have discovered that gene expression regulators work together to raise an individuals risk of developing schizophrenia. Schizophrenia-like gene expression changes modeled in human neurons matched changes found in patients brains. The researchers, led by Kristen Brennand, of the Icahn School of Medicine at Mount Sinai, New York City, report on their findings in Nature Genetics. The work was funded by the National Institute of Mental Health (NIMH), part of the National Institutes of Health.

Genome-wide association studies have revealed at least 143 chromosomal sites associated with risk for schizophrenia. However, individually, each of these sites can explain only a small fraction of the risk. Even when the effects of disease-linked rare genetic variants are factored in, most of schizophrenias known high inheritance remains unexplained. One possible clue: more than 40% of the suspect chromosomal sites contain regulators, called expression quantitative trait loci, or eQTLs, that govern the expression of multiple genes.

Individually, these gene regulators have a modest effect on the brain. Working in concert, they exert different and more significant effects on the brain effects that boost schizophrenia risk, explained David Panchision, chief of the Developmental Neurobiology Program at NIMH. Learning more about the downstream cellular and molecular effects of such synergy holds hope for advances in precision psychiatry and more personalized medicine.

To explore the role of these regulators, Brennand and colleagues studied them in induced neurons using a molecular modeling technology. This induced pluripotent stem cell method makes it possible to grow a persons unique neurons in a petri dish using stem cells derived from their skin cells. The researchers used the model to take a closer look at the downstream molecular consequences of gene expression changes known to occur in schizophrenia, and compared them with changes seen in postmortem brains and similarly modeled neurons of people with the illness.

The researchers experimentally mimicked the interaction of multiple risk genes thought to contribute to schizophrenia. They used the gene editing tool CRISPR to simultaneously increase or decrease expression of four schizophrenia-implicated genes known to harbor eQTLs. The genes were selected because they were deemed most likely to confer disease risk by regulating gene expression. To trigger changes in the direction predicted to heighten risk for schizophrenia, expression was increased for three of the genes and decreased for one.

Manipulating expression of the four genes altered expression of 1,261 other genes 665 increased and 596 decreased. This was many more than would be expected if the genes had been merely acting individually, suggesting an underlying mechanism that is synergistic rather than additive.

This unexpected synergy between gene variants demonstrated how even subtle genetic variations can impact neuronal function, said Brennand. These interactions emphasize the importance of considering the complex nature of schizophrenia and other psychiatric disorders, where a combination of gene variants contributes to disease.

Many of the genes affected downstream contained variants that had been linked to autism spectrum disorder or bipolar disorder, in addition to schizophrenia consistent with other research suggesting genetic overlap across mental disorders.

The experimentally induced gene expression changes mirrored those seen in postmortem brains of people with the three mental illnesses. The same changes were also seen in induced pluripotent stem cell neurons from people with childhood-onset schizophrenia, a rare form of the illness thought to be more genetic in origin.

Notably, all of these gene changes resulted in loss-of-brain-function effects when screened one-at-a-time in a zebrafish model, said Brennand. We have added several of them to a list of genes worthy of further study for possible involvement in schizophrenia. There is an overwhelming need for future studies to similarly model such multi-gene interactions in complex cells and circuits.

GRANTS: MH101454,MH106056,MH109897

About the National Institute of Mental Health (NIMH):The mission of theNIMHis to transform the understanding and treatment of mental illnesses through basic and clinical research, paving the way for prevention, recovery and cure. For more information, visit theNIMH website.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

Schrode N, Ho S-M, Yamamuro K, Dobbyn A, Huckins L, Matos MR. Cheng E, Deans PJM, Flaherty E, Barretto N, Topol A, Alganem K, Abadali S, Gregory J, Hoelzli E, Phatnani H, Singh V, Girish D, Aronow B, Mccullumsmith R, Hoffman GE, Stahl EA, Morishita H, Sklar P, Brennand KJ. Synergistic effects of common schizophrenia risk variants. Nature Genetics, September 23, 2019.DOI: 10.1038/s41588-019-0497-5

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Gene regulators work together for oversized impact on schizophrenia risk - National Institutes of Health

Blood Pressure and Prostate Treatment May Prevent or Slow Parkinson’s, Early Study Suggests – Parkinson’s News Today

People taking Hytrin (terazosin)or similar medications to treat high blood pressure and benign prostatic hyperplasiamay be less likely to develop Parkinsons disease, an early study suggests.

Parkinsons patients who use these medications may also see their disease progress more slowly and with fewer complications, its researchers report.

The study, Enhancing glycolysis attenuates Parkinsons disease progression in models and clinical databases, was published in the Journal of Clinical Investigation.

Hytrin is indicated for the treatment of non-cancerous prostate enlargement and high blood pressure (hypertension). The medication, available as a generic (terazosin), acts on the alpha-1 adrenergic receptor to block adrenalines action, relaxing smooth muscle in both the blood vessels and the prostate, allowing blood and urine, respectively, to flow more easily.

Experts agree that energy metabolism plays a central role in the molecular mechanism of neurodegenerative diseases like Parkinsons. In fact, problems in energy metabolism and low levels of cellular energy are common features of this disorder.

Hytrin has been shown to enhance the activity of a protein called phosphoglycerate kinase 1 (PGK1), which is involved in a critical energy-producing process known as glycolysis, where the simple sugar glucose is broken down by cells to produce energy. Boosting PGK1 activity increases the number of energy molecules, also known as ATP, within a cell.

Because low levels of ATP have been observed in Parkinsons, increasing the breakdown of glucose and hence cellular energy, in theory, may slow down or prevent the neurodegenerative processes underlying this disease.

Researchers at Capital Medical University in Beijing, decided to test this hypothesis and investigate whether increasing PGK1 activity levels would change the course of Parkinsons disease.

Hytrin was found to increase brain ATP levels and slow or prevent nerve cell loss in several models of Parkinsons (MPTP, rotenone and 6-OHDA-induced or genetic models): mice, rats, flies, and induced pluripotent stem cells. MPTP, rotenone and 6-OHDA are all neurotoxins that induce death of dopamine-producing neurons and mimic Parkinsons symptoms.

Treatment with this medication increased brain dopamine levels the chemical messenger that is present in low levels in the brains of Parkinsons patients and partially restored motor function in both mice and flies.

Importantly, boosting PGK1 activity was beneficial even when treatment was initiated after the onset of neurodegeneration, suggesting the modulation of this proteins function could help to slow Parkinsons progression.

Because Hytrin is prescribed for other diseases, scientists also studied two human databases the Parkinsons Progression Markers Initiative and the IBM Watson/Truven database looking for a possible Hytrin effect in relation to Parkinsons disease.

These two data sets included a total of 4,072 individuals on Hytrin, doxazosin, or alfuzosin. The latter two medications produce effects similar to those of Hytrin.

A retrospective analysis on both databases revealed that use of Hytrin and related agents slowed Parkinsons disease progression, reduced the number of neurodegeneration-related complications, and lessened the risk of a Parkinsons diagnosis, compared to people not using these medications.

These findings identify a protein and a pathway that might be targeted to slow or prevent neurodegeneration in PD [Parkinsons disease] and potentially other neurodegenerative diseases with altered energy balance, the researchers concluded.

Terazosin, available only by prescription, can cause dizziness and fainting because it lowers blood pressure.

With over three years of experience in the medical communications business, Catarina holds a BSc. in Biomedical Sciences and a MSc. in Neurosciences. Apart from writing, she has been involved in patient-oriented translational and clinical research.

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Blood Pressure and Prostate Treatment May Prevent or Slow Parkinson's, Early Study Suggests - Parkinson's News Today

New Gene Editing Technique Shown to Correct COL7A1 Gene in RDEB Cells – Epidermolysis Bullosa News

A novel gene editing technique was able to correct mutations inCOL7A1 in cells taken from people with recessive dystrophic epidermolysis bullosa (RDEB), providing a proof of concept for using the editing technique in the disease.

The finding was published in theJournal of Investigative Dermatology in a study titled, Base editor correction of COL7A1 in recessive dystrophic epidermolysis bullosa patient-derived fibroblasts and iPSCs.

The idea behind gene editing is simple: If a disease (such as RDEB) is caused by a mutation in a gene (COL7A1), then changing the genetic code to remove the mutation should functionally cure the disease. Of course, rewriting genetic code in living cells much less in human beings is far from simple.

Most current lab techniques basically involve cutting out the section of DNA that has the mutation, then replacing it with a healthy version. The problem is that there are often small insertions or deletions in the code where the DNA is cut, which isnt desirable.

In the new study, researchers tested a novel editing technique that uses a lab-made protein called an adenine base editor (ABE) to change just one nucleotide (a letter in the DNA code) without the need to cut out a whole chunk of DNA.

The researchers tried using ABE to correct mutations in COL7A1in skin cells taken from two RDEB patients. Broadly, they were successful, correcting the mutation in the DNA in about 24% of the cells; these cells also expressed collagen type VII (C7), the protein encoded by COL7A1.

The researchers then used the corrected skin cells to generate induced pluripotent stem cells (iPSCs, a type of cell that will perpetually divide and that, through chemical cues, can be led to differentiate into many different cell types). These iPSCs still expressed C7, providing a proof of concept that could be helpful in translating these findings into patients.

The researchers also surveyed other spots in the cells genomes to see whether there were any places the ABE was making changes it wasnt intended to. They didnt find any, suggesting a fairly low off-target rate.

Collectively, our study shows the feasibility of autologous cellular engineering using base editing to correct COL7A1 mutations in cell populations currently employed clinically for RDEB, the researchers concluded.

Marisa holds an MS in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. She specializes in cancer biology, immunology, and genetics. Marisa began working with BioNews in 2018, and has written about science and health for SelfHacked and the Genetics Society of America. She also writes/composes musicals and coaches the University of Pittsburgh fencing club.

Total Posts: 12

Ins Martins holds a BSc in Cell and Molecular Biology from Universidade Nova de Lisboa and is currently finishing her PhD in Biomedical Sciences at Universidade de Lisboa. Her work has been focused on blood vessels and their role in both hematopoiesis and cancer development.

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Vaginitis Therapeutics Market Overview with Detailed Analysis, Competitive lands – News By ReportsGO

The ' Vaginitis Therapeutics market' research report added by Market Study Report, LLC, is an in-depth analysis of the latest trends persuading the business outlook. The report also offers a concise summary of statistics, market valuation, and profit forecast, along with elucidating paradigms of the evolving competitive environment and business strategies enforced by the behemoths of this industry.

The latest research study on the Vaginitis Therapeutics market is a pivotal collection of insights pertaining to this industry vertical, with respect to certain parameters. The research report focuses on providing an in-depth synopsis of this industry, specifically illuminating the market industry size and share, application bifurcation, product types, as well as novel opportunities in the business space.

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Efavirenz/Tenofovir/Emtricitabine Combination Drug Market Overview with Detailed – News by Intelligence Journal

The ' Efavirenz/Tenofovir/Emtricitabine Combination Drug market' research report added by Market Study Report, LLC, is an in-depth analysis of the latest trends persuading the business outlook. The report also offers a concise summary of statistics, market valuation, and profit forecast, along with elucidating paradigms of the evolving competitive environment and business strategies enforced by the behemoths of this industry.

The recent report about the Efavirenz/Tenofovir/Emtricitabine Combination Drug market is a detailed synopsis of the projections of this business space in tandem with an evaluation of the industry segmentation. The report depicts the Efavirenz/Tenofovir/Emtricitabine Combination Drug market to evolve as one of most profitable verticals, procuring substantial valuation by the end of the estimated duration, while simultaneously registering a profitable growth rate over the forecast timespan. The expansion opportunities that are prevalent in this business alongside the industrys geographical reach have also been stated in the report.

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Can Consciousness be Created? – University Observer Online

Scientists have produced mini brains that mimic preterm babies brains, have they created consciousness too? Jade Norton investigates.

It is difficultfor us to comprehend our own consciousness, let alone try to operationallydefine it in a scientific experiment. In the last centuryscientificadvancement has allowed for experiments to be conducted that cross the fieldsof possibility and allow humans to play their hand at creation. However, thereare many ethical considerations that come along with this. For example, whatresponsibilities would suddenly arise if a tissue on a bench could not onlyreact to experimental procedures but had an opinion on them? On the other hand,what if the creation of consciousness in the pursuit of understanding can leadto answers that would not have been answered otherwise? This raises thequestion of whether scientists should aim to create consciousness or is itsomething that should be left to the natural world without human interference?

One recentexperiment undertaken by scientists from the University of California, SanDiego used stem cell technology to create cortical organoids or minibrains that are capable of producing brain signals that mimic that ofpremature babies. The brains do not look like a typical human brain and areinstead a smooth pea-sized blob that is encased in a nutrient-rich medium. Theylack the folding seen in a human brain as they do not contain grey and whitematter but are more of a mass of neural tissue. An induced pluripotent stemcell, which is a cell capable of dividing into any cell in the human body withthe right instructions, was used to create the brain cells. These cells dividedand over the course of 10 months grew from base neuronal cells toneuroepithelium-like structures which are similar to that of human braintissue. These organoids were not capableof complex thought but were created with the idea of using them to studyneurological diseases.

Throughout thedevelopment of the tissue nested oscillatory network dynamics were measured, theseare networks of repetitive electrical activity produced by the human brain inresponse to stimuli.This electrical activity can be found in all livingneurological tissue but does not necessarily show consciousness as there is yetto be an electrical ping signalling life as we currently understand it.

The team in SanDiego measured oscillatory spikes from the minibrains weekly usingmicroelectrode arrays and found an increase in activity as the monthsprogressed. This implied that there was a neural network capable of newdevelopment contained in the tissue. The electrical activity spawned by theneural network of the cortical organoids produced in the lab was recorded andsaved. Then using an EEG (electroencephalograph) the scientists measured the neuralactivity of a premature baby. These neural patterns were compared to see ifthere was a substantial difference between the neural activity between them.The comparison used a subset of features from the EEG to offset variablefactors not found in the cortical organoid. The results of comparison using amachine-learning algorithm found that the development of each tissue had manysimilarities which were likely to have been part of a genetically programmedtimeline. However, these minibrains were unable to progress to furtherdevelopment than that of a premature baby and it is thought that this is due tothe lack of sensory input that would usually be felt through the womb by apremature baby.

The minibrains that were developed in this experiment did not have any evidence ofconsciousness and were almost one million times smaller than a human brain andwithout the multiple types of cerebral cells they didnt have the capability ofdeveloping the full neurological complexity that is needed to form consciousnessas we see it. The measurements of electrical activity were done withoutcomparison of physiological features which varied greatly between the twotissues and have an effect on the maturation of neurons essential fordevelopment. The reality of creating a sentient being similar to ourselves isstill resting in the world of science-fiction, but the ability to create atissue that mimics brain activity and can be used in medical research is adefinite possibility. Brains can now be grown on a petri dish, but as of yetthey havent voiced any complaints.

The ability togrow an organoid that has an extensive neural network that is similar to thatof a preterm baby raises the question of at what point does consciousnessarise? It depends on who you ask. The origin of consciousness has yet to have auniversal consensus and without it there are no clear ethical rules relating tothe growth and development of cerebral tissue. There is no indicator that willtell you that consciousness has been created so it is possible that there is oronce was a homegrown sentient lab tissue somewhere.

Along with theadvancements in organoid technology, ethical considerations will continue tocome into question. Without the ability to know when consciousness has dawned,how do you know if the organoid is feeling pain or is distressed? And oncesomething develops a consciousness it becomes a subject of an experiment ratherthan an object which entitles it to its own rights. Consequently, this wouldseem that it would give scientists the responsibility to uphold these rights,however, this is still a largely unexplored area.

With theknowledge of ethical responsibility in hand, the possibilities of opportunityfor advancement is huge with a literal minibrain to work with. Medical researchcan use these organoids to see the in vivo effects of certain mental illnessessuch as schizophrenia and epilepsy and see the progression of neurodegenerativediseases such as Parkinsons and Alzheimers without the invasive problem ofviewing it in a live person. The question of when consciousness begins issomething that can fuel a philosophers career but for a scientist the creationof consciousness is a possibility that has never before been so accessible,with increasing advancements we may soon have our own brain in a jar.

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Can Consciousness be Created? - University Observer Online

Lineage Cell Therapeutics to Present at 2019 Cell & Gene Meeting on the Mesa on October 3, 2019 – BioSpace

Organized by the Alliance for Regenerative Medicine, the Cell & Gene Meeting on the Mesa is a premier conference combining discussions between senior executives and top decision-makers in the industry. The program also includes presentations by the fields most promising companies in the areas of cell therapy, gene therapy, gene editing, tissue engineering, and broader regenerative medicine technologies and extensive one-on-one partnering capabilities.

A live video webcast of all company presentations will be available at: http://www.meetingonthemesa.com/webcast and will also be published on the conference website shortly after the event.

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. Lineage is also evaluating potential partnership opportunities for Renevia, a facial aesthetics product that was recently granted a Conformit Europenne (CE) Mark. For more information, please visit http://www.lineagecell.com or follow the Company on Twitter @LineageCell.

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Lineage Cell Therapeutics to Present at 2019 Cell & Gene Meeting on the Mesa on October 3, 2019 - BioSpace

Certain Anatomical Sites Appear to Respond Well to an Investigational T-Cell Therapy for HPV-Positive Epithelial Cancers – Cancer Therapy Advisor

There have been promising results in a few patients with human papillomavirus (HPV)-positive epithelial cancers who were treated with innovative T-cell therapies, according to a study published in the Journal of Clinical Oncology.1

The idea behind the treatmentis to target a constitutively expressed tumor antigen expressed only in tumorcells and not healthy tissue, said Christian Hinrichs, MD, investigator in theexperimental transplantation and immunology branch at the National CancerInstitute, NIH Lasker Clinical Research Scholar, and lead author of theresearch.

T cells are taken from thepatient and genetically engineered to express a T-cell receptor (TCR) targetingan HPV viral oncoprotein expressed on HPV-associated cancers. They are thenexpanded to huge numbers (up to 100 billion cells) and infused back into thepatient.

The main toxicities arerelated to the chemotherapy given in the conditioning regimen cyclophosphamideand fludarabine, said Dr Hinrichs, noting that these toxicities included bonemarrow toxicity, bleeding, and an increased risk of infection.

Any significant clinicalbenefit of engineered T cells so far has generally been limited to their use inhematological cancers. Why has progress been so slow for other cancer types?

This approach is similar to [chimericantigen receptor] CAR T-cell approaches, but the difference is that we dontuse a CAR that can only target antigens on the surface of the cell. We use aTCR that can target intracellular antigens. Many of the most attractiveantigens in oncology such as KRAS and BRAF are intracellular, hence, cant betargeted with CARs, said Dr Hinrichs.

Two patients enrolled in the trialwere described as having an objective response to the therapy, with 1 patient,in particular, having a remarkable response.

The best response was in awoman with metastatic anal cancer where 1 of her lesions went away completelyand another 2 shrank completely. We then operated to remove the residualdisease; shes now 4 years out with no disease. She had metastatic cancer; itsremarkable and [she] has gone years with no cancer, said Dr Hinrichs.

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Certain Anatomical Sites Appear to Respond Well to an Investigational T-Cell Therapy for HPV-Positive Epithelial Cancers - Cancer Therapy Advisor