Celsius Therapeutics Signs Multiple Agreements with Academic Institutions to Access Samples for the Identification of Novel Targets in Immune…

Sept. 19, 2019 12:30 UTC

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Celsius Therapeutics, a company focused on bringing personalized medicine to patients with cancer, autoimmunity and other complex diseases, today announced the signing of collaboration agreements with the Parker Institute for Cancer Immunotherapy (San Francisco, USA), Institut Gustave Roussy (Paris, France) and the University Health Network (Toronto, Canada). Under these three agreements, Celsius will apply its proprietary single-cell genomics platform to tissue samples from patients receiving immune checkpoint inhibitor therapies for triple negative breast cancer, bladder cancer and kidney cancer, respectively. The goal of these collaborations is the discovery of novel molecular mechanisms and targets for drug discovery.

We are delighted to collaborate with these premier academic groups, given their disease expertise and their commitment to cutting-edge research and improving the lives of cancer patients, said Tariq Kassum, M.D., chief executive officer of Celsius. The heterogeneity of response in immunotherapy studies suggests that a deeper understanding of disease biology and patient subpopulations is needed to fully realize the potential of this approach. These new partnerships highlight our interest in broadly engaging the academic community to better elucidate the cellular ecosystem of cancer, with the ultimate goal of translating new insights into novel precision medicines for patients.

Under these agreements, Celsius will apply its platform approach to generate single-cell data from patient biopsy samples taken pre- and post-treatment with checkpoint inhibitors. In each case, Celsius retains the ability to integrate the clinical information and single-cell genomics data generated from the studies into its growing database. The company plans to utilize its machine learning algorithms and functional genomics capabilities to rapidly identify and prioritize targets for drug discovery.

Our integrated platform allows Celsius to obtain samples from anywhere in the world, process them in a highly industrialized manner and rapidly deploy a suite of machine learning algorithms to identify new drug targets in relevant cell types, said Christoph Lengauer, Ph.D., co-founder and chief scientific officer of Celsius. Across just these three collaborations, we expect to analyze more than 300 longitudinal samples from over 150 patients, a scale that is unprecedented for single-cell genomics. Combined with the associated clinical data that is being gathered from these studies, we are building a massive database that will be leveraged to develop novel precision medicines.

About Celsius Therapeutics

Celsius Therapeutics is charting a new course of target and drug discovery by applying a systematic approach to single-cell sequencing of patient tissue, combining massive datasets, complex algorithms, and machine learning to discover first-in-class precision therapies with a transformative impact on the lives of patients with autoimmune diseases and cancer. Celsius was launched in 2018, is backed by Third Rock Ventures and GV (formerly Google Ventures), and is based in Cambridge, Mass. For more information, please visit http://www.celsiustx.com.

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A compound may improve pancreatic cancer survival rate, scientists find – PhillyVoice.com

Scientists have discovered a compound could help slow down pancreatic cancer by targeting proteins that promote metastatic cells.

Researchers at Johns Hopkins Medicine found that the compound, 4-HAP, reduced tumors in mice and could improve survival for pancreatic cancer patients.

According to the National Cancer Institute, 73,554 people in the U.S. have pancreatic cancer and only 9.3 percent of those diagnosed survive five or more years.

The study, published in the journal Cancer Research, examined two types of proteins, mechanoresponsive proteins and non-mechanoresponsive proteins.

Scientists looked at a total of seven proteins, including nonmuscle myosin IIA, IIB and IIC; alpha-actinin 1 and 4; and filamin A and B. The mechanoresponsive proteins IIA, IIC, active actinin 4 and filamin B increased cancer tissue in the pancreas by over-producing the cancer cells.

Scientists found that even though the protein nonmuscle myosin IIC was found to be low in the cancer cells it had a profound impact on the cell's overall function. When they exposed these proteins to 4-HAP, it increased the cells' overall structure and stiffened the cells.

The group then tested 4-HAP as a treatment for pancreatic cancer by using a mouse model that had human pancreatic tissue implanted in the mouse's liver. They found that the tumors reduced by 50 percent.Researchers believe that treating the cells with the compound will allow scientists to target cancerous cells while protecting the healthy cells in the pancreas.

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Making Sense of Medicine: The meaning of life | Lifestyles – The Daily News of Newburyport

Ancient Greek philosopher Aristotle made what may be the first known attempt to define life. He said that something is alive if it grows, is animated and nourishes itself. A more recent definition suggests the important characteristics being the capacity for growth, reproduction, functional activity and continual change preceding death.

There are other attempts, but they all fail in some way. Both of these definitions, for example, would consider fire to be alive and mules, which are born sterile, not to be alive. All that said, most of us have an instinctive understanding of what it means to be alive. If you see a bug or a bird or even an amoeba, you are certain it possesses life.

None of this, however, tells us anything about how life first started or whence it came. Again, there are countless thoughts about this, such as life resulting from a stroke of lightning zapping just the right elements at the right time. Some believe radiation was the instigator, and some suggest that early life forms arrived on Earth aboard rocks from outer space. No one knows for sure.

However, there is research that shows the possibility of creating the basic building blocks of life, amino acids and proteins, from basic inorganic compounds by simulating the conditions that must have prevailed at Earths beginning. And research in cell biology has established some remarkably likely facts about how we became human.

Its all about cells?

A cell is the smallest functional unit of every living thing; we are made from trillions of them. The cell has a jelly-like liquid called cytoplasm enclosed in a membrane. Within the cytoplasm are structures with functions similar to our organs, and they are called organelles, or little organs, with names like mitochondria, lysosomes and others. With these, the cell can perform for itself all the same functions that we need for life: respiration, reproduction, waste removal, energy conversion and more.

Some cells have an organelle called the nucleus inside the membrane, as well. These are called eukaryotes, meaning to have a true (eu) nucleus (kary). A nucleus is important, as it contains most of the cells genes that are part of its DNA molecules; DNA is packaged in the form of chromosomes in order to fit into the nucleus. Other cells are called prokaryotes, meaning to exist before (pro) a nucleus occurs; bacteria are prokaryotes. They, too, have DNA, but its located directly in the cytoplasm.

The organelles are like computer programs, apps, in that they can perform specific functions, but they have to be started by a brain. The important fact is that cells are intelligent, and their brain is the cell membrane. The membrane includes tens of thousands of IMPs (integral membrane proteins) that receive and send signals from and to their environment, directing the organelles to do their thing and so create the complex behavior of a living cell.

A brief timeline

Earth was created about 41/2 billion years ago, but it wasnt until 3.8 billion years ago that single-cell prokaryotes appeared. That is, the bacteria have been here a seriously long time.

The eukaryotes evolved from prokaryotes, appearing about 21/2 billion years ago, and the kind of life forms we experience today are only about 600 million years old. There were no mammals until about 200 million years ago, and we, Homo sapiens, have been around for a mere 200,000 years. So, what happened to get from the first eukaryotes to us?

Community building

For the first 3 billion years that there was life on Earth, it consisted of independent single cells, monads. They were bacteria, they were algae, they were protozoans and some fungi. It was long thought that they were solitary.

Research has shown, however, that the signals they use to organize their own physiology can be released into their environment, creating a kind of long-distance communication. For example, amoebas consuming food release a particular molecule that informs other amoebas of a supply of food to which many are then drawn. Other signal molecules like hormones were in use by these single-cell critters, as well.

As time passed, monads learned to increase the number of IMPs in their membrane and started to assemble themselves into close-knit cellular communities for survival. As the size and complexity of these multicellular communities grew, they became highly organized and parceled out specific functions to specialized groups of cells: forming body tissues and organs, building nervous and immune systems, etc. And so there evolved the complex organisms we recognize as plants and animals.

As the more complex animals evolved, there were specialized cells that became responsible for monitoring and regulating the flow of signaling among other cells. This central information processor became the brain that controls the overall interaction and behavior of all the cells in the body. We commonly believe that our mind is the same as our brain and is located our head. However, neuroscientist and pharmacologist Candace Perts elegant experiments showed that the mind is distributed throughout the body.

We as humans, and, in fact, all mammals and some others, are an advanced stage of this kind of community organization. Of special interest is the development of a region of the brain called the prefrontal cortex. In this, we find the capacity for thinking, planning and decision-making, as well as the seat of self-consciousness.

And so ...

Cellular communities continue to evolve, giving new perspectives on what it means to be alive.

Bob Keller maintains a holistic practice in Newburyport. He offers medical massage therapy for pain relief, as well as psychological counseling, dream work and spiritual direction. Many patients call him Dr. Bob and accuse him of doing miracles, but he is not a medical doctor nor a divinity. His expertise is medical massage therapy, understanding this miracle we call the human being. He can be reached at 978-465-5111 or rk2name@gmail.com.

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#E#ECTRIMS2019 – In Progressive MS, ATA188 Showing Safety and Hints of Effectiveness in Phase 1 Trial – Multiple Sclerosis News Today

People with progressive forms of multiple sclerosis (MS) and past exposure to the Epstein-Barr virus are responding to a potentialimmunotherapy known as ATA188, tolerating the treatment well and with signs that suggest effectiveness, early updated data from an ongoing Phase 1 trial show.

The research, Preliminary safety and efficacy of ATA188, a pre-manufactured, unrelated donor (off-the-shelf, allogeneic) Epstein-Barr virus-targeted T-cell immunotherapy for patients with progressive forms of multiple sclerosis, was presented at the 35thCongress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS)held in Stockholm (Sept. 1113).

Infection with the Epstein-Barr virus (EBV)is linked to a greater susceptibility to MS. EBV promotes the expansion of immune B-cells, which can produce autoantibodies against myelin, the protective coating of nerve fibers that is progressively damaged in this disease.

People with MS are believed to be deficient in T-cells, which would normally kill EBV-infected B-cells.

ATA188, byAtara Biotherapeutics, is designed to overcome this deficiency by providing allogeneic immune T-cells that target EBV in B-cells. (Allogeneic cells are from donors with no family or genetic relationship to the recipient; an advantage is that these cells are available immediately, or off-the-shelf.)

The Phase 1 study (NCT03283826), taking place in the U.S. and Australia, is assessing the safety and efficacy of ATA188 in progressive MS patents here, a near equal mix of secondary progressive disease (SPMS) and primary progressive MS (PPMS) ages 18 to 66. Patients are assigned to four groups, each testing a different cell dose: 5 x 106, 1 x 107, 2 x 107, and 4 x 107.

Clinically recognized MS scales are assessing ATA188 effectiveness at baseline (study start), and at roughly 3, 6, and 12 months after initial treatment. These include the Expanded Disability Status Scale(EDSS), the Fatigue Severity Score, the MS Impact Scale, the Timed 25-Foot Walk (T25FW) where patients walk 25 feet as quickly and safely as possible the 9-Hole Peg Test of hand and finger skills, 12-Item MS Walking Scale, and tests of visual acuity.

Early data previously reportedshowed that treatment with ATA188 was well-tolerated across the four groups, with no evidence of cytokine release syndrome(a form of systemic inflammatory response), graft versus host disease(which refers to the attack of the hosts cells by transplanted stem cells), or dose-limiting toxicities.

Preliminary data now reported at ECTRIMS, collected through July 29, 2019, covered the four groups of six patients each (median age, 56).

Results found no dose-limiting toxicities. There were also no reports ofclinically significant laboratory abnormalities in any of the groups.

One patient in group 4 (using 4 x 107cells) dropped out of the study due to aMS relapse that took place in the setting of an ongoing URI [upper respiratory tract infection] and possible dental infection, the researchers reported at ECTRIMS.

Safety results showed that across the four planned dose cohorts, ATA188 was well tolerated in patients with progressive forms of MS, with no evidence of cytokine release syndrome, graft versus host disease, or dose-limiting toxicities, they added.

Efficacy data were available from the first two groups, and hints of benefit were seen.

Four of the six patients in group 1 the lowest dose group showed clinical decline at 6 and 12 months, defined as worsening in two or moreMS scales compared to baseline. The other two showed at least partial improvement in these scales at month six, with one of these showing stable disease at 12 months.

In group 2 (1 x 107),all six patients showed either clinical improvement (two people) or partial clinical improvement (four people) at six months, or improvements in two or more or more MS scales. Data for this group did not stretch out 12 months.

Better results in terms of lesser disability were seen in people in group 2, where only one person experienced clinical decline compared to four of the six patients in group 1.

These preliminary results support continuing the trial to identify the dose for both the OLE [open-label extension] and the randomized, double-blind, placebo-controlled portion (part 2) of the study, the researchers wrote.

I am encouraged by the well tolerated safety profile, as well as early findings of potential efficacy in the ongoing ATA188 Phase 1 study, Amit Bar-Or, MD, a principal investigator in the trial and chief of MS Divisionat thePerelman School of Medicine at the University of Pennsylvania, said in apress release.

He also favored this studys innovative approach, that of using multiple MS scales to measure benefit.

I look forward to advancing the study alongside my colleagues for progressive MS patients who have limited treatment options,and where continual clinical decline is expected, Bar-Or added.

These early data support the potential of a T-cell immunotherapy targeting EBV-infected B-cells in progressive MS, said AJ Joshi, MD, Ataras senior vice president and chief medical officer.

Our recent completion of enrollment in the fourth and final dose escalation cohort moves us closer to identifying the dose to be used in the studys placebo-controlled Phase 1b part, Joshi added.We are committed to advancing ATA188 for MS patients, and look forward to presenting additional efficacy and safety results from this study in 2020, including from cohorts 3 and 4.

Of note, eight of the studys 14 authors are employees and stockholders of Atara. One other is a member of the companys Neurology Clinical Advisory Panel.

Jos is a science news writer with a PhD in Neuroscience from Universidade of Porto, in Portugal. He has studied Biochemistry also at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario, in London, Ontario. His work ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimers disease.

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Patrcia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.

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Researchers Develop New Class of Lung Cancer Drugs – PharmaLive

Working with a new class of cancer drugs called G12C KRAS inhibitors, researchers at the Francis Crick Institute and The Institute of Cancer Research (ICR), London, developed drug combinations to stay ahead of drug resistant tumors. They published their research in the journal Science Translational Medicine.

Cancer cells often develop resistance to drug treatments. Many are initially susceptible to chemotherapy, but over time develop resistance, causing researchers to work to develop new drugs that attack the cancer by way of different mechanisms or to find combination therapies that simultaneously kill the cells in different ways.

Mutations in the KRAS gene are found in 14% of lung adenocarcinomas, which is the most common type of lung cancer. It is a particularly deadly form with few if any effective treatments for most patients. About eight out of 10 patients die within five years of diagnosis. In the UK alone, about 2,800 people are diagnosed with lung cancers with the G12C KRAS mutation.

Lung adenocarcinoma is a type of non-small cell lung cancer. NSCLCs make up 80% of lung cancers, and of these about 50% are adenocarcinomas. It is the most common lung cancer in women, Asians and individuals under the age of 45. It is also, unexpectedly, more common in non-smokers than people who currently smoke. Contributing factors are genetics, secondhand smoke, and radon exposure.

Its likely that tumors will develop resistance to the new drugs, so we need to stay one step ahead, said Julian Downward, senior author of the study who led the research. We found a three-drug combination that significantly shrank lung tumors in mice and human cancer cells.

Downward added, Tumors treated with the combination shrank and stayed small, whereas those treated with the G12C KRAS inhibitor alone tended to shrink at first but then start growing again after a couple of weeks. Our results suggest that it would be worth trying this combination in human trials in the coming years, to prevent or at least delay drug resistance.

The other two drugs in the cocktail block the mTOR and IGF1R pathways, which had previously been tested in cancer patients. There are mTOR inhibitors on the market, such as rapamycin and temsirolimus (Pfizers Torisel). Currently, IGF1R inhibitors are experimental drugs in the clinical trial stage.

The researchers utilize cancer cells from patients with the G12C KRAS mutation, which they then edited to block the activity of 16,019 different genes. They then treated the edited cells with drugs that KRAS mutational cancer typically respond to.

We found that cell lines without the MTOR gene were significantly more vulnerable to both KRAS and IGF1R inhibitors, said Miriam Molina-Arcas, senior laboratory research scientist at the Crick. When we blocked all three pathways, the mutant cancer cells were simply unable to survive. This makes it a promising avenue for human trials in the coming years, although this is still early research. Promising results in mice and cells can tell us whats worth trying, but its impossible to predict how patients will respond until we actually try.

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Factbox: Animal health sector aims to emulate Zoetis biotech leap – Reuters

FILE PHOTO: Moose, a six-year-old English Bulldog with B-cell lymphoma, receives trial medical treatment at the Tufts Veterinary Medical School by vet technicians Stephanie Pollen (L) and Michele Willett in North Grafton, Massachusetts, U.S., September 13, 2019. REUTERS/Katherine Taylor

(Reuters) - A commercial breakthrough in the field of complex therapeutic proteins for dogs by sector leader Zoetis (ZTS.N) has roused rivals to action in a hunt for biotechnology medicines to treat pets.

Previously reserved for humans, biotech drugs made from genetically engineered living cells are now seen as ripe for the pet health market. The following are some of the recent deals, launches and development projects in the space:

- British animal health company Dechra Pharmaceuticals Inc (DPH.L) agreed to a deal https://www.akstonbio.com in August to utilize development work by U.S. insulin specialist Akston Biosciences for a once weekly shot for diabetic dogs to replace daily injections, with a view to expanding the collaboration to cats. Dechra has said long-acting insulin, which is typically produced from genetically modified bacteria, should become its biggest product.

- U.S. animal health company Kindred Biosciences Inc (KIN.O), which repurposed a human antidepressant to stimulate appetite in underweight cats, has several genetically engineered biologic drugs under development kindredbio.com/pipeline, targeting diseases such as dermatitis in dogs and anemia in cats.

- German early-stage biotech research firm Adivo in April signed a global collaboration deal here with Bayer's (BAYGn.DE) animal health unit a little over a year after Adivo was spun out of human biotech firm Morphosys (MORG.DE) with a mission to develop antibodies for dogs.

- Privately-held German drugmaker Boehringer Ingelheim, the global No. 2 in animal health, in April launched here the first stem cell-based veterinary medicine to treat lameness in horses caused by inflamed joints, a common worry for both recreational and competitive riders.

- Industry leader Zoetis Inc (ZTS.N), which has a successful anti-itch biotech drug for dogs on the market, said here in August it was seeking approval for more antibodies, targeting osteoarthritic pain in cats with plans for a 2021 market launch, drawing on its 2017 acquisition of Nexvet Biopharma. A similar product for dogs is also in the pipeline.

- In June of 2018, Zoetis signed a deal here with human biotech firm Regeneron Pharmaceuticals Inc (REGN.O) to adopt its antibody technology for animals, targeting inflammatory disease and cancer.

Reporting by Ludwig Burger; Edited by Bill Berkrot

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Only hearts harvested from living babies can be used for research, expert tells Daleiden hearing – Lifesite

SAN FRANCISCO, California, September 19, 2019 (LifeSiteNews) An expert in adult stem cell research told a San Francisco court Wednesday that fetal hearts used in research are harvested from living babies.

Dr. Theresa Deisher testified on the last day of the criminal preliminary hearing of David Daleiden and Sandra Merritt that had stretched into three weeks.

The investigative journalists from the Center for Medical Progress (CMP) are charged with 14 felony counts of taping confidential information without consent in connection with the undercover videos CMP released in 2015 exposing Planned Parenthood trafficking in baby body parts.

Public outrage over the groundbreaking videos prompted congressional hearings and calls for the federal defunding of Planned Parenthood.

Daleiden and Merritt are claiming a Section 633.5 defense that allows covert taping when investigating violent crimes. Their lawyers are also arguing the law does not consider as confidential conversations that have a reasonable expectation of being overheard.

A focus of Daleidens early research, a key element in the defenses case and a source of public debate during the hearing, is a 2012 Stanford University study that used human baby hearts supplied by the fetal organ harvesting company StemExpress in a Langendorff apparatus.

Daleiden testified that Deisher was one of the experts he consulted in 2012 about whether the hearts used in the study were harvested from living babies, and Deisher told him that was the case.

She confirmed that in her testimony Wednesday as a defense expert witness.

Deisher, who holds a Ph.D. in Molecular and Cellular Physiology from Stanford University School of Medicine, told the court that a heart requires energy to relax after contracting, and if it contracts after it runs out of energy, it is useless for research.

Once the heart goes into contraction, you cant get it to come out of that position, she told the court.

To be harvested, a heart has to be beating and be arrested in a relaxed position by perfusing it with a potassium solution, she told the court.

The relaxed heart is then transported to the research site, where it is started again electrically, Deisher said.

So to be used in theLangendorff apparatus, the fetal heart would have to be alive when dissected from the fetus? asked Daleidens lawyer Brentford Ferreira.

It has to be beating and immediately go on the Langendorff, Deisher said, or be arrested in a relaxed state while it was still beating and subsequently put on a Langendorff.

Deisher testified that according to scientific protocol, the 2012 Stanford studys citing of StemExpress meant the company supplied the human hearts.

She said Daleiden first called her in 2010 when he learned about the harvesting and selling of aborted baby body parts.

Deisher said she told him then that the most horrific thing about the trafficking in baby body parts was that some of the babies had to have beating hearts when they were harvested.

Between 2010 and 2013, Daleiden called her several times. He wanted to know what a Langendorff was and the vocabulary of stem cell research, she said. I just taught him the science.

Deisher said she is the founder and CEO of a company in Seattle that is developing alternative research methods to eliminate human exploitation in medical research. Her website bio says she is the first person to discover adult stem cells.

She testified that its never necessary to use fetal organs for stem cell research, because there are induced pluripotent stem cells available, which are produced from adult stem cells and can be cultured into any type of body cell.

Deisher said convenience and pressure from animal rights activists motivate researchers to use human fetal organs.

Deputy Attorney General Johnette Jauron asked Deisher if she consulted with Daleiden about his undercover operation before he began the 30-month project.

Deisher said she hadnt heard of CMP until the videos were released and that she only watched them much later.

Judge Christopher Hite also questioned Deisher, asking her if she ever discussed Daleidens undercover investigations with him or any of the Does, if she had performed abortions or worked at a fetal tissue procurement company, or was familiar with abortion techniques. Deisher said no to all questions.

Daleiden and Ferreira lauded Deishers testimony.

I thought it was riveting, especially when she talked about the horror of babies being born alive and having their hearts being torn out of their little bodies,Ferreira said.

Deishers testimony shows why I and my colleagues like Sandra Merritt and others did our undercover work with the Center for Medical Progress, Daleiden told LifeSiteNews.

Because theres very compelling evidence that the most valuable organs and tissues from aborted children are harvested from aborted children while theyre still alive, he said.

Those kids are actually killed through vivisection and that is a human atrocity that has to be exposed.

Deisher reiterated later to LifeSiteNews that in order to obtain a heart for a Langendorff apparatus, the organ harvesters would cut open the babys chest and they would take the heart out beating and drop it in a buffer with potassium.

It would then be transported in a vial with pre-oxygenated buffers and other preservatives to the research site.

Anyone involved in the field would know this is the case.

Of course, if the heart isnt beating, they cant get any of these cells, its impossible, technologically impossible, she said. Nobody wants a stopped heart.

The whole process is beneath the dignity of a civilized society, to cannibalize the bodies of aborted babies, to harvest from a living baby. Theyre not even anesthetized. We dont even do that to mice and rats, Deisher told LifeSiteNews.

Theres no scientific or medical reason that we need to do that and anyone who claims it either does not understand the full science or theyre not telling the truth, she said.

She believes the charges against Daleiden and Merritt will be dropped.

If they dont dismiss the charges, then theyre not following the law, Deisher said.

He was investigating based on the fact that I told him that babies were being harvested alive. Thats a crime, and undercover investigation is, if I understand from what the lawyers told me, perfectly legal if you think theres a crime thats occurring.

Read all LifeSiteNews coverage of Daleiden and Merritts preliminary hearing here.

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CRISPR/Cas9 Potential in Advancing Parkinson’s Understanding and Treatment Focus of Review Study – Parkinson’s News Today

In a recent review, scientists highlight the potential of gene editing technologies like CRISPR/Cas9 to not only understand the molecular mechanisms behind Parkinsons disease, but also identify new targets for treatment.

The review study, Interrogating Parkinsons disease associated redox targets: Potential application of CRISPR editing, was published in the journal Free Radical Biology and Medicine.

One of the hallmarks of PD is the loss of dopamine-producing neurons in the substantia nigra a brain region involved in the control of voluntary movements, and one of the most affected in PD. This occurs due to the clustering of a protein called alpha-synuclein in structures commonly known as Lewy bodies inside neurons.

Parkinsons is complex and multifactorial disease, with both genetic and environmental factors playing a role in either triggering or exacerbating the disease.

Genetic causes can explain 10% of all cases of PD called familial PD , meaning that in the majority of the cases (sporadic PD) there is an interplay between genetics and environmental risk factors.

Researchers atSechenov Universityin Russia and theUniversity of Pittsburgh reviewed the role of metabolic pathways, especially problems with mitochondria cells powerhouses and iron accumulation, as well as mechanisms in cell death (called apoptosis and ferroptosis) in the development and progression of Parkinsons disease.

These processes were discussed in the context of genome editing technologies, namely CRISPR/Cas9 a technique that allows scientists to edit genomes, inserting or deleting DNA sequences, with precision, efficiency and flexibility.

CRISPR is a promising technology, a strategy to find new effective treatments to neurodegenerative diseases, Margarita Artyukhova, a student at the Institute for Regenerative Medicineat Sechenov and the study first author, said in a press release.

Mitochondria dont work as they should in people withPD, resulting in shortages of cellular energy that cause neurons to fail and ultimately die, particularlydopamine-producing neurons. Faulty mitochondria are also linked to the abnormal production of reactive oxygen species, leading to oxidative stressan imbalance between the production of free radicals and the ability of cells to detoxify them that also damages cells over time.

Because mitochondrial dysfunction is harmful, damaged mitochondria are usually eliminated (literally, consumed and expelled) in a process called mitophagy an important cleansing process in which two genes, called PINK1 and PRKN, play crucialroles. Harmful changes in mitophagy regulation is linked with neurodegeneration in Parkinsons.

Previous studies with animal models carrying mutations in the PINK1and PRKNgenes showed that these animals developed typical features of PD mitochondrial dysfunction, muscle degeneration, and a marked loss of dopamine-producing neurons.

PINK1codes for an enzyme that protects brain cells against oxidative stress, whilePRKNcodes for a protein called parkin. Both are essential for proper mitochondrial function and recycling by mitophagy. Mutations in both the PINK1 and PRKNgene have been linked with early-onset PD.

However, new research suggests that the role of PINK1 and PRKNin Parkinsons could be more complex and involve other genes likePARK7(DJ-1), SNCA (alpha-synuclein) andFBXO7 as well as a fat molecule called cardiolipin.

CRISPR/Cas9 genome editing technology may be used to help assess the role of different genetic players in Parkinsons disease, and to look for unknown genes associated with disease progression and development. Moreover, this technology can help generate animal and cellular models that might help scientists decipher the role of certain proteins in Parkinsons and discover potential new treatment targets.

Iron is another important metabolic cue in Parkinsons. While its essential for normal physiological functions, excessive levels of iron can be toxic and lead to the death of dopamine-producing neurons in the substantia nigra.

Iron may also interact with dopamine, promoting the production of toxic molecules that damage mitochondria and cause alpha-synuclein buildup within neurons.

CRISPR/Cas9 technology can be used to help dissect the role of proteins involved in iron transport inside neurons, which in turn may aid in designing therapies to restore iron levels to normal in the context of Parkinsons disease.

Finally, researchers summarized evidence related to the role of two cell death pathways ferroptosis and apoptosis in PD. Ferroptosis is an iron-dependent cell death mechanism by which iron changes fat (lipid) molecules, turning them toxic to neurons. This process has been implicated in cell death associated with degenerative diseases like Parkinsons, and drugs that work to inhibit ferroptosis have shown an ability to halt neurodegeneration in animal models of the disease.

Apoptosis refers to a programmed cell death mechanism, as opposed to cell death caused by injury. Both apoptosis and ferroptosis speed the death of dopaminergic neurons.

CRISPR/Cas9 may help to pinpoint the key players in cell death that promote the loss of dopaminergic neurons in Parkinsons disease, while understanding the array of proteins that are involved in these processes.

These insights into the mechanisms of PD pathology [disease mechanisms] may be used for the identification of new targets for therapeutic interventions and innovative approaches to genome editing, including CRISPR/Cas9, the researchers wrote.

Genome editing technology is currently being used in clinical trials to treat patients with late-stage cancers and inherited blood disorders, Artyukhova notes in the release.

These studies allow us to see vast potential of genome editing as a therapeutic strategy. Its hard not to be thrilled and excited when you understand that progress of genome editing technologies can completely change our understanding of treatment of Parkinsons disease and other neurodegenerative disorders, she adds.

Patricia holds a Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She has also served as a PhD student research assistant at the Department of Microbiology & Immunology, Columbia University, New York.

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Alzheimers Disease May Be Caused by Copper In The Brain – Science Times

Lysette Maurice N. SandovalSep 18, 2019 09:40 AM EDT

Tai-Yen Chen, a chemist from the University of Houston, is conducting a study to explore the link between a person's exposure to molecules of copper protein in the brain cells and the risks of suffering from a neurodegenerative disease like Alzheimer's.

"For 100 years, scientists have continued to search for answers to Alzheimer's disease and the many potential causes, but none of them have been proven successful. Up until today, no one really knows why some people suffer from Alzheimer's and others do not," Chen said. He was recently awarded by the National Institute of General Medical Science funding amounting to $1.9 million dollars to further develop his theory that the copper protein balance in every cell in the brain may be the culprit to people developing and suffering from Alzheimer's disease.

Copper is considered an essential nutrient in the growth of cells in the human brain. They help the neurons receive and deliver messages all throughout the body. Every healthy cells have a certain amount of copper that is strictly regulated to remain at a certain level. Scientists from long ago have already uncovered the fact that patients with Alzheimer's disease have an unusually high level of copper on brain cells, which basically became the signature of the degenerative disease.

"The findings were unusual and we wanted to learn more about it," Chen said. His study will be looking into the biological regulation of copper levels in brain cells to help doctors identify potential cases of Alzheimer's disease. The abnormal growth of copper in the cells will be a clear indicator that will establish the link between the two. There are several other degenerative diseases that are linked to the imbalance of copper in the human brain. Menkes disease, a nervous system disorder that is characterized by low levels of copper, while Wilson disease is a genetic disorder that is caused by having too much copper present in the head.

"We want to know how the cells are able to regulate the copper levels inside them to be able to achieve the optimum amount of copper level," said Chen. He will be working mostly cells from the brain and the liver. "The study will be employing a unique method of looking into individual cells to be able to identify its behavior. The process will be repeated in all the other cells that will become part of the study to get the information needed. The collective behavior of these cells will help us uncover the facts we need to better understand the relationship between copper content in cells."

When the study is able to figure out how the regulation of copper content in cells is done naturally, particularly between normal, healthy cells and diseased ones, the findings will truly shed light on the overall pathology of Alzheimer's disease.

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Alzheimers Disease May Be Caused by Copper In The Brain - Science Times

Toyobo to market RNA Analysis Kit based on RIKEN technology to help develop genomic medicine – BioSpace

OSAKA, Japan, Sept. 18, 2019 /PRNewswire/ -- Toyobo Co., Ltd. has productized a ribonucleic acid (RNA) analysis kit based on a novel system titled RamDA-seq*1,that allows full-length sequencing of the total RNA of a single cell. The Laboratory for Bioinformatics Research at the RIKEN Center for Biosystems Dynamics Research developed this method last year.

As national health insurance programs in Japan have covered genomic medicine since June 2019, medical care is progressing to be tailored to individual patients by selecting suitable treatment and drugs after analyzing a patient's genes. In recent years, genetic information in a single cell has proven to be effective in treating diseases such as cancer caused by gene mutations, because each cell has different genetic properties. However, conventional methods have been unable to reliably detect disease-causing mutations from an extremely small amount of RNA*2 derived from a single cell.

RIKEN, which is based in Wako City, Saitama Prefecture, is one of Japan's largest scientific research institutes. RIKEN has developed a novel method, RamDA-seq, which enables the amplification of RNA in low bias and the detection of an expression level of genes in a single cell. This method allows the comprehensive identification of RNA varieties and their amounts in a single cell. It drastically improved the analytical precision of a disease-causing gene expression level and mutations in various RNAs, including non-polyadenylated RNAs whose functions remain unclear due to the difficulty in their detection.

RNA Analysis Kit, which Toyobo productized under RIKEN's technological guidance, consists of reagents and a user manual necessary to conduct RamDA-seq. The kit makes it possible to prepare samples more smoothly and stably, as described in articles published by RIKEN.

The kit is expected to expand the use of RamDA-seq, to accelerate the development of various medical fields ranging from basic research to regenerative and genomic medicine. Toyobo will launch the product for research institutes including pharmaceutical companies developing drugs for cancer and other diseases on September 30, 2019. The company aims to achieve annual sales of JPY 1 billion in fiscal 2022.

RNA Analysis Kit to be marketed

Genome includes a complete set of genetic information of the cell, which is recorded as sequences of four types of nucleotide in a biological polymer called DNA. Genes are regions that record genetic information in genome. Genetic information is read out by synthesizing RNA using DNA as a template.The cells that constitute our bodies control its gene expression to perform various functions, while having almost the same genetic information. Since this gene expression is mediated by molecules called RNA, a comprehensive analysis of RNA is effective in understanding the individual characters of individual cells.

About Toyobo Co., Ltd.: Toyobo Co., Ltd (Toyobo) is a worldwide specialty chemical company, headquartered in Osaka, Japan. Since its foundation in 1882 as a textile company, Toyobo has expanded the boundaries of its business beyond textiles to specialty business segments such as industrial and packaging films, functional polymers, industrial materials and healthcare with its unique core technologies originated in textiles and chemical fibers.

For more information, contact: https://www.toyobo-global.com/

View original content:http://www.prnewswire.com/news-releases/toyobo-to-market-rna-analysis-kit-based-on-riken-technology-to-help-develop-genomic-medicine-300920735.html

SOURCE Toyobo Co., Ltd.

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Toyobo to market RNA Analysis Kit based on RIKEN technology to help develop genomic medicine - BioSpace