Archive for September, 2019

We are pleased to announce Technology Networks Explores the CRISPR Revolution. Through a series of exclusive interviews with world-renowned scientists and bioethicists, Technology Networks Explores the CRISPR Revolution will investigate the ground-breaking research taking place in the CRISPR space, CRISPR "controversies" and whether the CRISPR technology looks set to fulfil its promise of revolutionizing science.

The series will feature interviews with researchers behind the discovery of the CRISPR mechanism, such as Professor Francisco Mojica, the scientist involved its development as a gene-editing tool, Professor Jennifer Doudna, and the "godfather" of human genome research, Professor George Church.

The series will also explore the latest technologies available in the CRISPR "toolbox" including industry perspectives, its application in agriculture and farming through a conversation with Professor Alison Van Eenennaam and insights into the global conversation surrounding its ethical implications from Professor Glenn Cohen.

Kicking off the series on Oct 14th is an interview with the humble and immensely influential microbiologist, Professor Francisco Mojica.

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Technology Networks Explores the CRISPR Revolution Coming Soon - Technology Networks

Duchenne muscular dystrophy (DMD) is a rare but devastating genetic disorder that causes muscle loss and physical impairments. Researchers at the University of Missouri School of Medicine have shown in a mouse study that the powerful gene editing technique known as CRISPR may provide the means for lifelong correction of the genetic mutation responsible for the disorder.

Children with DMD have a gene mutation that interrupts the production of a protein known as dystrophin. Without dystrophin, muscle cells become weaker and eventually die. Many children lose the ability to walk, and muscles essential for breathing and heart function ultimately stop working.

"Research has shown that CRISPR can be used to edit out the mutation that causes the early death of muscle cells in an animal model," said Dongsheng Duan, PhD, Margaret Proctor Mulligan Professor in Medical Research in the Department of Molecular Microbiology and Immunology at the MU School of Medicine and the senior author of the study. "However, there is a major concern of relapse because these gene-edited muscle cells wear out over time. If we can correct the mutation in muscle stem cells, then cells regenerated from the edited stem cells will no longer carry the mutation. A one-time treatment of the muscle stem cells with CRISPR could result in continuous dystrophin expression in regenerated muscle cells."

In collaboration with other MU colleagues and researchers from the National Center for Advancing Translational Sciences, Johns Hopkins School of Medicine and Duke University, Duan explored whether muscle stem cells from mice could be efficiently edited. The researchers first delivered the gene editing tools to normal mouse muscle through AAV9, a virus that was recently approved by the U.S. Food and Drug Administration to treat spinal muscular atrophy.

"We transplanted AAV9 treated muscle into an immune-deficient mouse," said Michael Nance, a MD-PhD program student in Duan's lab and the lead author of the paper. "The transplanted muscle died first then regenerated from its stem cells. If the stem cells were successfully edited, the regenerated muscle cells should also carry the edited gene."

The researchers' reasoning was correct, as they found abundant edited cells in the regenerated muscle. They then tested if muscle stem cells in a mouse model of DMD could be edited with CRISPR. Similar to what they found in normal muscle, the stem cells in the diseased muscle were also edited. Cells regenerated from these edited cells successfully produced dystrophin.

"This finding suggests that CRISPR gene editing may provide a method for lifelong correction of the genetic mutation in DMD and potentially other muscle diseases," Duan said. "Our research shows that CRISPR can be used to effectively edit the stem cells responsible for muscle regeneration. The ability to treat the stem cells that are responsible for maintaining muscle growth may pave the way for a one-time treatment that can provide a source of gene-edited cells throughout a patient's life."

With more study, the researchers hope this stem cell-targeted CRISPR approach may one day lead to long-lasting therapies for children with DMD.

Reference: Nance et al. 2019.AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice. Molecular Therapy.DOI: https://doi.org/10.1016/j.ymthe.2019.06.012.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Researchers Use CRISPR to Correct Mutation in Duchenne Muscular Dystrophy Model - Technology Networks

A team has used a lentiviral capsid-based bionanoparticle system to deliver CRISPR-Cas9 gene editing therapies, reducing undesired effects.

Researchers have developed an improved CRISPR delivery system for gene editing, through a lentiviral capsid system. The team say that their findings could be useful in research and clinical applications by improving safety and avoiding possible immune responses.

using a traditional lentiviral vector allows the bionanoparticle to efficiently and safely deliver CRISPR-Cas9

The team, from Wake Forest Institute of Regenerative Medicine (WFIRM), US, packaged the Cas9 protein and guide RNA together within a lentiviral capsid-based bionanoparticle system.

Previously, the two components had to be delivered separately which was not as convenient, said Dr Baisong Lu, assistant professor of regenerative medicine at WFIRM and one of the lead authors of the paper.

Conventional CRISPR-Cas9 is not completely accurate and could potentially cut unexpected locations, causing unwanted results.

However, the using a traditional lentiviral vector allows the bionanoparticle to efficiently and safely deliver CRISPR-Cas9. The researchers observed that it reduced off-target rates compared to regular CRISPR-Cas9.

A similar strategy should be translatable to other editor proteins for gene disruption, said Anthony Atala, MD, director of WFIRM and a co-author of the paper. We may be able to utilise this to package and deliver other RNPs into mammalian cells, which has been difficult to achieve so far.

The findings were published in Nucleic Acids Research.

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Researchers improve CRISPR-Cas9 delivery efficiency - Drug Target Review

UK-based Oxford Nanopore has obtained a licence to CRISPR-Cas9 IP for nanopore sequencing, a third-generation approach used in the sequencing of biopolymers.

Oxford Nanopore, which specialises in DNA/RNA sequencing technology, announced the non-exclusive licence agreement with biotech company Caribou Biosciences yesterday, September 19.

Caribou was founded by scientists from the University of California, Berkeley, including CRISPR pioneer Jennifer Doudna.

Gordon Sanghera, CEO of Oxford Nanopore, said: The Cas9 technique will enable users to select and isolate the regions of the genome they are most interested in, including those not available to existing methods, ready for rapid analysis using our long-read, real-time sequencing technology.

According to the company, Cas9 enrichment with Oxford Nanopore sequencing enables scientists to cost-effectively sequence targeted regions that were not accessible previously.

Sanghera added: The entire library preparation process takes less than two hours so if combined with our portable sequencer MinION, this has the potential to open up fast-turnaround, near-sample testing in new ways.

In October last year, Amgen invested 50 million ($66 million) in Oxford Nanopore, as part of Amgens focus on using human genetics to deliver new medicines to patients.

Earlier in 2018, Oxford Nanopore announced it had raised 100 million from global investors, to be used to support the companys next phase of commercial expansion, including a new high-tech manufacturing facility in Oxford.

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Oxford Nanopore, CRISPR-Cas9, Caribou Biosciences, Jennifer Doudna, gene-editing, genetics, nanopore, University of California,

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Oxford Nanopore signs CRISPR licence - Life Sciences Intellectual Property Review

At this time, there is no cure for Duchenne muscular dystrophy (DMD), although there is one treatment for a subgroup of the disease. That is Sarepta Therapeutics Exondys 51 for DMD patients with a confirmed mutation amenable to exon 51 skipping. Recently the U.S. Food and Drug Administration (FDA) rejected Sareptas golodirsen for DMD with a confirmed mutation appropriate for exon 53 skipping.

DMD is a muscle wasting disease caused by mutations in the dystrophin gene. It is a progressive disease that usually causes death in early adulthood, with serious complications that include heart or respiratory-related problems. It mostly affects boys, about 1 in every 3,500 or 5,000 male children.

There just might be, however, hope for an actual cure. Researchers at the University of Missouri-Columbia, utilized CRISPR gene editing in a mouse model, to edit out the gene mutation and transplant AAV9 treated muscle into the mice. The transplanted muscle cells carried the edited gene and successfully produced dystrophin, the protein that is not produced in sufficient quantities in DMD patients.

The dystrophin gene is the largest in the body, and codes for the dystrophin protein, which is involved in muscle development and activity. One of the reasons DMD has been a tough nut to crack is that because of the genes size, its too large to fit into the typical viral vectors used in gene therapies. Thats partially why Sareptas approach is to use a type of RNA splicing that forces cells to skip over the faulty section of genetic code. This results in a shortened (truncated) protein that is still functional.

Research has shown that CRISPR can be used to edit out the nutation that causes the early death of muscle cells in an animal model, said Dongsheng Duan, the Margaret Proctor Mulligan Professor in Medical Research in the Department of Molecular Microbiology and Immunology at the MU School of Medicine and senior author of the study.

However, Duan went on, there is a major concern of relapse because these gene-edited muscle cells wear out over time. If we can correct the mutation in muscle stem cells, then cells regenerated from the edited stem cells will no longer carry the mutation. A one-time treatment of the muscle stem cells with CRISPR could result in continuous dystrophin expression in regenerated muscle cells.

Duans research, in collaboration with others at MU as well as the National Center for Advancing Translational Sciences, Johns Hopkins School of Medicine and Duke University, looked at whether muscle stem cells in mice could be effectively edited. They used AAV9, an adeno-associated virus recently approved by the FDA to treat spinal muscular atrophy (SMA)Novartis Zolgensma, which is also the source of the controversy over the companys data manipulation scandal.

They started by delivering CRISPR to normal mouse muscle via AAV9.

We transplanted AAV9-treated muscle into an immune-deficient mouse, said Michael Nance, an MD-PhD program student in Duans lab and the lead author of the paper. The transplanted muscle died first then regenerated from its stem cells. If the stem cells were successfully edited, the regenerated muscle cells should also carry the edited gene.

That appeared to work. They then tested if the muscle stem cells in the mice of DMD could be edited with CRISPRthey were.

This finding suggests that CRISPR gene editing may provide a method for lifelong correction of the genetic mutation in DMD and potentially other muscle diseases, Duan said. Our research shows that CRISPR can be used to effectively edit the stem cells responsible for muscle regeneration. The ability to treat the stem cells that are responsible for maintaining muscle growth may pave the way for a one-time treatment that can provide a source of gene-edited cells throughout the patients life.

The research was published in the journal Molecular Therapy.

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CRISPR Research Might Lead to Cure for Duchenne Muscular Dystrophy - BioSpace

Just because a business does not make any money, does not mean that the stock will go down. For example, although software-as-a-service business Salesforce.com lost money for years while it grew recurring revenue, if you held shares since 2005, you'd have done very well indeed. Nonetheless, only a fool would ignore the risk that a loss making company burns through its cash too quickly.

Given this risk, we thought we'd take a look at whether CRISPR Therapeutics (NASDAQ:CRSP) shareholders should be worried about its cash burn. In this article, we define cash burn as its annual (negative) free cash flow, which is the amount of money a company spends each year to fund its growth. The first step is to compare its cash burn with its cash reserves, to give us its 'cash runway'.

Check out our latest analysis for CRISPR Therapeutics

You can calculate a company's cash runway by dividing the amount of cash it has by the rate at which it is spending that cash. In June 2019, CRISPR Therapeutics had US$428m in cash, and was debt-free. In the last year, its cash burn was US$133m. That means it had a cash runway of about 3.2 years as of June 2019. Importantly, though, analysts think that CRISPR Therapeutics will reach cashflow breakeven before then. If that happens, then the length of its cash runway, today, would become a moot point. Depicted below, you can see how its cash holdings have changed over time.

NasdaqGM:CRSP Historical Debt, September 21st 2019

CRISPR Therapeutics boosted investment sharply in the last year, with cash burn ramping by 61%. That's bad enough, but the operating revenue drop of 96% points to a period of uncertainty and, quite potentially, heightened risk for holders." In light of the above-mentioned, we're pretty wary of the trajectory the company seems to be on. While the past is always worth studying, it is the future that matters most of all. So you might want to take a peek at how much the company is expected to grow in the next few years.

Even though it seems like CRISPR Therapeutics is developing its business nicely, we still like to consider how easily it could raise more money to accelerate growth. Companies can raise capital through either debt or equity. Commonly, a business will sell new shares in itself to raise cash to drive growth. We can compare a company's cash burn to its market capitalisation to get a sense for how many new shares a company would have to issue to fund one year's operations.

CRISPR Therapeutics has a market capitalisation of US$2.6b and burnt through US$133m last year, which is 5.1% of the company's market value. That's a low proportion, so we figure the company would be able to raise more cash to fund growth, with a little dilution, or even to simply borrow some money.

It may already be apparent to you that we're relatively comfortable with the way CRISPR Therapeutics is burning through its cash. For example, we think its cash runway suggests that the company is on a good path. Although we do find its falling revenue to be a bit of a negative, once we consider the other metrics mentioned in this article together, the overall picture is one we are comfortable with. It's clearly very positive to see that analysts are forecasting the company will break even fairly soon After considering a range of factors in this article, we're pretty relaxed about its cash burn, since the company seems to be in a good position to continue to fund its growth. For us, it's always important to consider risks around cash burn rates. But investors should look at a whole range of factors when researching a new stock. For example, it could be interesting to see how much the CRISPR Therapeutics CEO receives in total remuneration.

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Of course CRISPR Therapeutics may not be the best stock to buy. So you may wish to see this freecollection of companies boasting high return on equity, or this list of stocks that insiders are buying.

We aim to bring you long-term focused research analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material.

If you spot an error that warrants correction, please contact the editor at editorial-team@simplywallst.com. This article by Simply Wall St is general in nature. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. Simply Wall St has no position in the stocks mentioned. Thank you for reading.

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CRISPR Therapeutics (NASDAQ:CRSP) Is In A Strong Position To Grow Its Business - Yahoo Finance

CRISPR is the buzzword of the moment in the drug discovery industry mainly due to its potential to correct disease-causing mutations. However, those using the technology need to be mindful that it is used responsibly, and possible risks are considered before use. Mark Behlke discusses the potential of CRISPR in R&D and the challenges that it presents for researchers.

CRISPR TECHNOLOGY has generated much excitement in the drug discovery realm for its ability to make precise, permanent changes to DNA in animals, as first demonstrated approximately six years ago. It is currently being evaluated in early phase clinical trials for several disorders. Diseases caused by a single gene mutation sickle cell disease (SCD), Huntingtons disease and cystic fibrosis are all prime targets for using CRISPR gene therapy to correct the disease-causing DNA mutations. CRISPR is also being investigated as a treatment for acquired immune deficiency syndrome (AIDS) and to improve anti-tumour immunotherapy.

While news about potential CRISPR therapeutics and the start of new clinical trials dominate headlines in the lay press, CRISPR has also become a leading research tool to help scientists better understand gene function and establish model systems of human diseases needed to translate basic scientific knowledge into new medical treatments.

Link:
The elegant parallel of using CRISPR to understand disease mechanisms - Drug Target Review

-New data demonstrate successful differentiation of CRISPR-edited human pluripotent stem cells to pancreatic precursor cells-

ZUG, Switzerland, CAMBRIDGE, Mass., and SAN DIEGO, Sept. 17, 2019 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (CRSP), and ViaCyte, Inc., a privately-held cell therapy company, today presented data from the Companies regenerative medicine program targeted towards type 1 diabetes (T1D) in an oral presentation at the 55th Annual Meeting of the European Association for the Study of Diabetes (EASD) in Barcelona, Spain. The data demonstrate that the CyT49 pluripotent stem cell line, which has been shown to be amenable to efficient scaling and differentiation, can be successfully edited with CRISPR. The CyT49 pluripotent stem cell line is currently being used to generate islet progenitors for clinical trials.

These data provide further evidence that the combination of regenerative medicine and gene editing has the potential to offer durable, curative therapies to patients in many different diseases, including common chronic disorders like insulin-requiring diabetes, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. We look forward to advancing our T1D program in partnership with ViaCyte.

We are pleased with the data presented at EASD, which bring us potentially one step closer to a transformational therapy for patients with insulin-requiring diabetes through the development of an immune-evasive gene-edited version of our technology, said Paul Laikind, Ph.D., Chief Executive Officer and President of ViaCyte. ViaCyte has led the field over the past decade, being the first group to demonstrate a number of essential milestones on the path to a broadly applicable cell replacement therapy for diabetes. Now, in partnership with CRISPR Therapeutics, we aim to achieve yet another first, the development of an immune-evasive cell replacement therapy as a potential cure for T1D. The work being presented at EASD is an important step along that path.

To protect pancreatic islet cells from immune rejection, researchers utilized CRISPR/Cas9 gene editing to generate CyT49 clones that lack the 2-microglobulin (B2M) gene, a required component of the major histocompatibility complex class I (MHC-I), and express a transgene encoding programmed death-ligand 1 (PD-L1) to further protect from T-cell attack. Edited clonal cells maintained karyotypic stability and showed in vitro protection against T-cell mediated cell lysis.

About the CRISPR-ViaCyte CollaborationCRISPR Therapeutics and ViaCyte entered into a strategic collaboration in 2018 focused on the discovery, development, and commercialization of novel regenerative medicines including gene-edited allogeneic stem cell-derived therapies for the treatment of diabetes. The Companies are currently evaluating a preclinical-stage therapeutic candidate for insulin-requiring diabetes including type 1 diabetes, for which the Companies will jointly assume responsibility for development and commercialization worldwide.

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer AG, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in London, United Kingdom. For more information, please visit http://www.crisprtx.com.

About ViaCyteViaCyte is a privately-held regenerative medicine company developing novel cell replacement therapies as potential long-term diabetes treatments to achieve glucose control targets and reduce the risk of hypoglycemia and diabetes-related complications. ViaCytes product candidates are based on the derivation of pancreatic isletprogenitor cells from pluripotent stem cells, which are then implanted in durable and retrievable cell delivery devices. Over a decade ago, ViaCyte scientists were the first to report on the production of pancreatic cells from a stem cell starting point and the first to demonstrate in an animal model of diabetes that, once implanted and matured, these cells secrete insulin and other pancreatic hormones in response to blood glucose levels. ViaCyte has two product candidates in clinical-stage development. The PEC-Direct product candidate delivers the pancreatic isletprogenitor cells in a non-immunoprotective device and is being developed for type 1 diabetes patients who have hypoglycemia unawareness, extreme glycemic lability, and/or recurrent severe hypoglycemic episodes. The PEC-Encap (also known as VC-01) product candidate delivers the same pancreatic isletprogenitor cells in an immunoprotective device and is being developed for all patients with diabetes, type 1 and type 2, who use insulin. ViaCyte is also developing immune-evasive stem cell lines, from its proprietary CyT49 cell line, which have the potential to further broaden the availability of cell therapy for diabetes and other potential indications. ViaCyte is headquartered in San Diego, California. ViaCyte is funded in part by the California Institute for Regenerative Medicine (CIRM) and JDRF. For more information, please visit http://www.viacyte.com.

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CRISPR Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the safety, efficacy and clinical progress of our various clinical programs including CTX001 and CTX110; (ii) the status of clinical trials (including, without limitation, the timing of filing of clinical trial applications and INDs, any approvals thereof and the timing of commencement of clinical trials), development timelines and discussions with regulatory authorities related to product candidates under development by CRISPR Therapeutics and its collaborators; (iii) the number of patients that will be evaluated, the anticipated date by which enrollment will be completed and the data that will be generated by ongoing and planned clinical trials, and the ability to use that data for the design and initiation of further clinical trials; (iv) the intellectual property coverage and positions of CRISPR Therapeutics, its licensors and third parties as well as the status and potential outcome of proceedings involving any such intellectual property; (v) the sufficiency of CRISPR Therapeutics cash resources; and (vi) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential for initial and preliminary data from any clinical trial (including CTX001 and CTX110) not to be indicative of final trial results; the risk that the initial data from a limited number of patients (as is the case with CTX001 at this time) may not be indicative of results from the full planned study population; the outcomes for each CRISPR Therapeutics planned clinical trials and studies may not be favorable; that one or more of CRISPR Therapeutics internal or external product candidate programs will not proceed as planned for technical, scientific or commercial reasons; that future competitive or other market factors may adversely affect the commercial potential for CRISPR Therapeutics product candidates; uncertainties inherent in the initiation and completion of preclinical studies for CRISPR Therapeutics product candidates; availability and timing of results from preclinical studies; whether results from a preclinical trial will be predictive of future results of the future trials; uncertainties about regulatory approvals to conduct trials or to market products; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

CRISPR Investor Contact:Susan Kim+1 617-307-7503susan.kim@crisprtx.com

CRISPR Media Contact:Jennifer PaganelliWCG on behalf of CRISPR+1 347-658-8290jpaganelli@wcgworld.com

ViaCyte Investor Contact: Matthew LaneGilmartin Group on behalf of ViaCyte, Inc. +1 617-901-7698matt@gilmartinir.com

ViaCyte Media Contact:Jessica Yingling, Ph.D. Little Dog Communications Inc. on behalf of ViaCyte, Inc. +1 858-344-8091jessica@litldog.com

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CRISPR Therapeutics and ViaCyte Present Positive In Vitro Data Towards a Potential Immune-Evasive Cell Replacement Therapy for Diabetes at EASD 2019 -...

CRISPR may be a cure, but clinical trials may lack volunteers because of black patients' mistrust of biased and unethical medical practices.

"It's like something stabbing you in your bone just like repeatedly. And you can't stop it. And... it's something that makes you really tense, you just can't move... It's been a part of my life since I was six months old."

Twenty-three-year-old Maiya Washington has been living with sickle cell, a life-long "invisible" disease. It's a disorder with no visibly detectable signs and not often talked about.

"Although it is a disease that is invisible, so to speak, we go through a lot, you know, and we go through a lot to be normal to live life to get jobs to go to school. And it's hard. It's really hard," says Washington.

Sickle Cell Disease impacts about100,000 Americans,mostly African Americans. It's a genetic defect that affects red blood cells turning normal round red blood cells into sickle shapes. That shape can cause clumping andblock oxygen and blood flow,which can then lead to a wide range of health issues likestrokes, kidney problems,and organ failure.

"It's really debilitating, honestly," says Washington. "And it's hard because... you need somebody there with you. It's not like you can take yourself to the hospital. Or if you're at home and you're trying to manage the pain by yourself, you still need somebody there with you, because you're not able to do the simple things like go to the restroom by yourself, get yourself some water, fix yourself something to eat"

Currently, the only option to cure sickle cell disease is a bone marrow transplant, also called a stem cell transplant. But it requires a matching donor, which can be difficult to find. But now, the gene-editing tool CRISPR may bypass the need for a match and serve as a cure for most people. Dr. John Tisdale has worked on another Sickle Cell Disease gene therapy at the National Institute of Health.

"And it's all coming from red blood cells with a single misspelling, in their hemoglobin. So we should be able to fix that it's just one base," says Tisdale.

Related StoryIs The Machine That Can Snip And Swap Our DNA Awesome Or Ominous?

Here's how it works. First, stem cells are pulled from the patient's body. CRISPR is used to edit the DNA, and the new, edited stem cells are then re-inserted back into the body. The hope is that it will generate healthy red blood cells. Changes to the DNA won't be passed down to future children.

Could this development lead to a cure for sickle cell? A clinical trial using CRISPR can help determine that. So far, testing on 12 people has already begun. Biopharmaceutical company Vertex has partnered with CRISPR Therapeutics and they're looking for morevolunteers for this clinical trial.

Dr. Alexis Thompson, Head of Hematology, Lurie Childrens Hospital of Chicago says, "I certainly am very excited about it as a provider, is that patients will have choices. There is not a one size fits all for sickle cell disease, or really, really for in a community For the first time, there will be multiple, multiple clinical trials that are opening and hopefully those will lead to new treatments... because much of what we learn from sickle cell will be rapidly applicable to other conditions."

But as exciting as the potential for a cure for Sickle Cell Disease is, finding enough volunteers for testing is really difficult. Sure, CRISPR poses risks, including death. But one of the biggest barriers isblack patients' mistrust of the medical community,so much so that clinical trialstend to lack black patient enrollment.This is based on racist treatment both in the past and present.Science writer Usha Lee MacFarlingpoints to unethical medical practices of the past, like the use of Henrietta Lacks' cells without permission and leaving syphilis untreated in hundreds of black men in the Tuskegee experiment.

"I think there's just a huge awareness in the black community of these studies that were, you know, racist, that really treated black women of color and poor women as guinea pigs. It's a very sharp pain And it's definitely affecting people's reluctance, and inability to trust, the largely white medical establishment," says MacFarling.

And today, some black patients say that bias persists in medicine. Because sickle cell patientsvisit the emergency room an average of three times a year, they're often assumed to be addicts for seeking drugs to ease their pain.

Related StoryResearchers' Gene Technology Removes HIV From Mice For The First Time

"Doctors [have]...given me an inappropriate amount of medicine. That wasn't helping, that kind of basically looked at me as like, you know, drug-seeking, or just like faking it," says Washington.

In spite of all this, Maiya says she wants to participate in the trial because of how excruciating her pain is.

"I feel as though most people who deal with sickle cell or any kind of disability that alters their quality of life, they're going to be willing to figure out anything to get rid of what they had," says Washington.

In order to build trust between black sickle cell patients and CRISPR researchers, organizations like the American Society of Hematology and the Minority Coalition for Precision Medicine are doing community outreach. That includes even teaming up with churches.

Michael Friend, co-founder of Minority Coalition for Precision Medicine says: "It was kind of very easy to talk to faith-based leaders about sickle cell disease because it's a disease that primarily affects African Americans. And it's a disease that we found prevalent in most churches, and most pastors were familiar with the disease."

Related StoryScientists Concerned Over Program That Enlists Bugs To Spread Viruses

For now, every month Maiya's gets a blood transfusion to ease the pain. Her baby is lucky -- she doesn't have sickle cell, because her father doesn't have the gene.

"I wake up every day and she's there. She's my best friend and I love seeing her watching her grow so far, says Washington.

Most sickle cell patients don't live past their 40's and Maiya does worry about her future.

She says, "it does concern me because I want to be here as long as possible for her. And hoping that, you know, there's something that can come up that can be permanent, you know, as in terms of a cure or medication...just to help us have a longer lifespan and live a better quality of life. Because I do want to be able to see her grow up."

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CRISPR Cure For Sickle Cell May Be Slowed By Black Patients' Mistrust - Newsy

We are at war with the mosquito. A swarming and consuming army of 110tn enemy mosquitoes patrols every inch of the globe except for Antarctica, Iceland and a handful of French Polynesian micro-islands. The biting female warrior of this droning insect population is armed with at least 15 lethal and debilitating biological weapons, to be used against 7.7 billion humans deploying suspect and often self-detrimental defensive capabilities. In fact, our defence budget for personal shields, sprays and other means of deterring her unrelenting raids is $11bn (8.8bn) a year, and rising rapidly. And yet her deadly offensive campaigns and crimes against humanity continue with reckless abandon. While our counterattacks are reducing the number of casualties she perpetrates malaria deaths in particular are declining rapidly the mosquito remains the deadliest hunter of human beings on the planet.

Taking a broad range of estimates into account, since 2000, the average annual number of human deaths caused by the mosquito was around 2 million. Humans came in a distant second at 475,000, followed by snakes (50,000), dogs and sandflies (25,000 each), the tsetse fly, and the assassin or kissing bug (10,000 each). The fierce killers of lore and Hollywood celebrity were much further down our list. The crocodile was ranked 10th, with 1,000 annual deaths. Next on the list were hippos with 500, and elephants and lions with 100 fatalities each. The much-slandered shark and wolf shared 15th position, killing an average of 10 people per annum.

Yet the mosquito does not directly harm anyone. It is the toxic and highly evolved diseases she transmits that cause an endless barrage of desolation and death. Without her, however, these sinister pathogens could not be transferred or vectored to humans, nor could they continue their cyclical contagion. In fact, without her, these diseases would not exist at all.

Our immune systems are finely tuned to our local environments. Mosquitoes do not respect international borders. Marching armies, inquisitive explorers and land-hungry colonists brought new diseases to distant lands, but were also brought to their knees by micro-organisms in the foreign lands they intended to conquer. As the mosquito transformed the landscapes of civilisation, humans were unwittingly required to respond to her universal projection of power. After all, the truth is that, more than any other external participant, the mosquito, as our deadliest predator, drove the events of human history to create our present reality.

It has been one of the most universally recognisable and aggravating sounds on Earth for 190m years the whine of a mosquito. After a long day of walking while camping with your family or friends, you quickly shower, settle into your lawn chair, open an ice-cold beer and exhale a deep, contented sigh. Before you can enjoy your first satisfying swig, however, you hear that all-too-familiar sound, signalling the approach of your soon-to-be tormentors.

It is nearing dusk, her favourite time to feed. Although you heard her droning arrival, she gently lands on your ankle without detection, as she usually bites close to the ground. It is always a female, by the way. She conducts a tender, probing, 10-second reconnaissance, looking for a prime blood vessel. With her backside in the air, she steadies her crosshairs and zeros in with six sophisticated needles. She inserts two serrated mandible cutting blades (much like an electric carving knife, with two blades shifting back and forth), and saws into your skin, while two other retractors open a passage for the proboscis, a hypodermic syringe that emerges from its protective sheath. With this straw she starts to suck out 3-5 mg of your blood, immediately excreting its water while condensing its 20% protein content. All the while, a sixth needle is pumping in saliva that contains an anticoagulant, preventing your blood from clotting at the puncture site. This shortens her feeding time, lessening the likelihood that you feel her penetration and splat her across your ankle. The anticoagulant causes an allergic reaction, leaving an itchy bump as her parting gift. The mosquito bite is an intricate and innovative feeding ritual required for reproduction. She needs your blood to grow and mature her eggs.

Please dont feel singled out. She bites everyone. There is absolutely no truth to the persistent myths that mosquitoes fancy females over males, that they prefer blonds and redheads over those with darker hair, or that the darker or more leathery your skin, the safer you are from her bite. It is true, however, that she does play favourites and feasts on some more than others. Blood type O seems to be the vintage of choice over types A and B, or their blend. People with blood type O get bitten twice as often as those with type A, with type B falling somewhere in between. (Disney/Pixar must have done their homework when portraying a tipsy mosquito ordering a Bloody Mary, O-positive in the 1998 movie A Bugs Life.) Those who have higher natural levels of certain chemicals in their skin, particularly lactic acid, also seem to be more attractive. From these elements, she can analyse which blood type you are. These are the same chemicals that determine an individuals level of skin bacteria and unique body odour. While you may offend others and perhaps yourself, in this case, being pungently rancid is a good thing, for it increases bacterial levels on the skin, which makes you less alluring to mosquitoes except for stinky feet, which emit a bacterium that is a mosquito aphrodisiac. The mosquito is also enticed by deodorants, perfumes, soap and other applied fragrances.

She also has an affinity for beer drinkers. Wearing bright colours is also not a wise choice, since she hunts by both sight and smell the latter depending chiefly on the amount of carbon dioxide exhaled by the potential target. So all your thrashing and huffing and puffing only magnetises mosquitoes and puts you at greater risk. She can smell carbon dioxide from 200 feet away. When you exercise, you emit more carbon dioxide through frequency of breath and output. You also sweat, releasing those appetising chemicals, primarily lactic acid, that invite the mosquitos attention. Lastly, your body temperature rises an easily identifiable heat signature. On average, pregnant women suffer twice as many bites, as they respire 20% more carbon dioxide, and have a marginally elevated body temperature. This is bad news for the mother and the foetus when it comes to infection from Zika and malaria.

Unlike their female counterparts, male mosquitoes do not bite. Their world revolves around two things: nectar and sex. Like other flying insects, when they are ready to mate, male mosquitoes assemble over a prominent feature in the landscape from chimneys to antennas to trees to people. Many of us grumble and flail in frustration as that dogged cloud of bugs droning over our heads shadows us when we walk, refusing to disperse. Take it as a compliment. Male mosquitoes have graced you with the honour of being a swarm marker. Mosquito swarms have been photographed extending 1,000 feet into the air, resembling a tornado funnel cloud. With the cocksure males stubbornly assembled over your head, females will fly into their horde to find a suitable mate. While males will mate frequently in a lifetime, one dose of sperm is all the female needs to produce numerous batches of offspring. She stores the sperm and dispenses them piecemeal for each separate birthing of eggs. Her short moment of passion has provided one of the two necessary components for procreation. The only ingredient missing is your blood.

Back at the campsite, you have just finished your strenuous hike, and proceed to the shower, where you lather up with soap and shampoo. After drying off, you apply body spray and deodorant before finally putting on your bright red-and-blue beachwear.

It is nearing dusk dinnertime for the Anopheles mosquito. You have done everything in your power to lure a famished female of the species. Having just mated in a swarming frenzy of eager male suitors, she willingly takes the bait and makes off with a few drops of your blood a blood meal three times her own body weight. She quickly finds the nearest vertical surface and, with the aid of gravity, continues to evacuate the water from your blood. Using this concentrated blood, she will develop her eggs over the next few days. She then deposits roughly 200 floating eggs on the surface of a small pool of water that has collected on a crushed beer can that was overlooked during cleanup as you and your party headed home. She always lays her eggs in water, although she does not need much. From a pond or stream to a minuscule puddle in the bottom of an old container, used tire or backyard toy, any will suffice.

Our mosquito will continue to bite and lay eggs during her one-to-three-week lifespan. While she can fly up to two miles, she rarely ranges more than 400 metres from her birthplace. Although it takes a few days longer in cool weather, given the high temperatures, her eggs hatch into wiggling, water-bound worms within two or three days. Skimming the water for food, they quickly turn into upside-down, comma-shaped tumbling caterpillars who breathe through two trumpets protruding from their water-exposed buttocks. A few days later, a protective encasement splits and healthy adult mosquitoes take flight, with a new generation of succubus females ready to feed. This maturation to adulthood takes roughly one week.

Bacteria, viruses and parasites, along with worms and fungi, have triggered untold misery, and have commanded the course of human history. Why have these pathogens evolved to exterminate their hosts? If we can set aside our bias for a moment, we can see that these microbes have journeyed through the natural selection voyage just as we have. This is why they still make us sick and are so difficult to eradicate. You may be puzzled: it seems self-defeating and detrimental to kill your host. The disease kills us, yes, but the symptoms of the disease are ways in which the microbe conscripts us to help it spread and reproduce. It is dazzlingly clever, when you stop to think about it. Generally, germs guarantee their contagion and replication prior to killing their hosts. Some, like the salmonella food poisoning bacteria and various worms, wait to be ingested that is, one animal eating another animal.

There is a wide range of waterborne transmitters, including giardia, cholera, typhoid, dysentery and hepatitis. Others, including the common cold, the 24-hour flu and true influenza, are passed on through coughing and sneezing. Some, such as smallpox, are transferred directly or indirectly by lesions, open sores, contaminated objects or coughing. My personal favourites strictly from an evolutionary standpoint, of course are those that covertly ensure their reproduction while we intimately ensure our own. These include the full gamut of microbes that trigger sexually transmitted diseases. Many sinister pathogens are passed from mother to foetus in utero.

Others that germinate typhus, bubonic plague, Chagas and trypanosomiasis (African sleeping sickness) catch a free ride provided by a vector (an organism that transmits disease) such as fleas, mites, flies, ticks and mosquitoes. To maximise their chances of survival, many germs use a combination of more than one method. The diverse collection of symptoms, or modes of transference, assembled by micro-organisms helps them effectively procreate and ensures the existence of their species. These germs fight for their survival just as much as we do, and stay an evolutionary step ahead of us as they continue to morph and shape-shift to circumvent our best means of extermination.

To understand the stealthy, sprawling influence of the mosquito on history and humanity, it is first necessary to appreciate the animal itself, and the diseases it transmits. According to a quotation erroneously attributed to Charles Darwin: It is not the strongest of the species that survives, nor the most intelligent that survives. It is the one that is most adaptable to change. Regardless of the origin of this passage, the mosquito and its diseases most notably malaria parasites are the quintessential example of the point it is making. They are masters of evolutionary adaptation. Mosquitoes can evolve and adapt to their changing environments within a few generations. During the Blitz of 1940-41, for example, as German bombs rained down on London, isolated populations of Culex mosquitoes were confined to the air-raid tunnel shelters of the London Underground, along with the citys resilient citizens. These trapped mosquitoes quickly adapted to feed on mice, rats and humans instead of birds, and are now a species distinct from their above-ground parental ancestors.

What should have taken thousands of years of evolution was accomplished in less than 100. In another 100 years, jokes Richard Jones, former president of the British Entomological and Natural History Society, there may be separate Circle line, Metropolitan line and Jubilee line mosquito species in the tunnels below London.

While the mosquito is miraculously adaptable, it is also a purely narcissistic creature. Unlike other insects, it does not pollinate plants in any meaningful way, or aerate the soil, or ingest waste. Contrary to popular belief, the mosquito does not even serve as an indispensable food source for any other animal. She has no purpose other than to propagate her species, and perhaps to kill humans. As the apex predator throughout our odyssey, it appears that her role in our relationship is to act as a countermeasure against uncontrolled human population growth.

Throughout our existence, the mosquitos toxic twins of malaria and yellow fever have been the prevailing agents of death and historical change, playing the role of antagonists in the protracted chronological war between man and mosquito.

Following that fateful mosquito bite, the miscreant malaria parasite will mutate and reproduce inside your liver for one to two weeks, during which time you will show no symptoms. A toxic army of this new mutated form will then explode out of your liver and invade your bloodstream. The parasites attach to your red blood cells, penetrate the outer defences, and feast on the haemoglobin within. Inside the cell, they undergo another metamorphosis and reproductive cycle. Engorged blood cells eventually burst, spewing both a duplicate form, which marches forward to attack fresh red blood cells and also a new asexual form that relaxedly floats in your bloodstream, waiting for mosquito transportation.

The parasite is a shape-shifter, and it is precisely this genetic flexibility that makes it so difficult to eradicate or suppress with drugs or vaccines. You are now gravely ill with an orderly, clockwork progression of chills followed by a mercury-driving fever that may touch 41C. This full-blown cyclical malarial episode has you in its firm grip, and you are at the mercy of the parasite. Lying prostrate and agonisingly helpless on sweat-soaked sheets, you twitch and fumble, curse and moan. You look down and notice that your spleen and liver are visibly enlarged, your skin has the yellowing patina of jaundice and you vomit sporadically. Your fever will relapse at precise intervals with each new burst and invasion of the parasite from your blood cells. The fever then subsides while the parasite eats and reproduces inside new blood cells.

The parasite uses sophisticated signalling to synchronise its sequencing, and this entire cycle adheres to a very strict schedule. The new asexual form transmits a chemical bite me signal in our blood, significantly boosting the chances of being picked up by a mosquito from an infected human to complete the reproductive cycle. Inside the stomach of the mosquito, these cells mutate once more, into both male and female varieties. They quickly mate, producing threadlike offspring that make their way out of the gut and into the salivary glands of the mosquito. Within the saliva glands, the malaria parasite shrewdly manipulates the mosquito to bite more frequently by suppressing the production of her anticoagulant and thus minimising her blood intake during a single feeding. This forces her to bite more frequently to get her required fill. In doing so, the malaria parasite ensures that it maximises its rate and range of transfer, its procreation and its survival.

Temperature is an important element for both mosquito reproduction and the life cycle of malaria. Given their symbiotic relationship, they are also both climate-sensitive. In colder temperatures, it takes longer for mosquito eggs to mature and hatch. Mosquitoes are also cold-blooded and, unlike mammals, cannot regulate their own body temperatures. They simply cannot survive in environments below 10C. Mosquitoes are generally at their prime health and peak performance in temperatures above 23C. A direct heat of 40C degrees will boil mosquitoes to death. For temperate, non-tropical zones, this means that mosquitoes are seasonal creatures with breeding, hatching and biting taking place from spring through autumn. Although never seeing the outside world, malaria needs to contend with both the short lifespan of the mosquito and temperature conditions to ensure replication. The timeframe of malaria reproduction is dependent on the temperature of the cold-blooded mosquito, which itself is dependent on the temperature outside. The colder the mosquito, the more sluggish malaria reproduction becomes, eventually hitting a threshold. Between 15C and 21C (depending on the type of malaria), the reproductive cycle of the parasite can take up to a month, exceeding the average life span of the mosquito. By then, she is long dead, and brings malaria down with her.

Warmer climates can sustain year-round mosquito populations, promoting endemic circulation of her diseases. Abnormally high temperatures can cause seasonal epidemics of mosquito-borne diseases in regions where they are generally absent or fleeting. Global warming also allows the mosquito and her diseases to broaden their topographical range. As temperatures rise, disease-carrying species, usually confined to southern regions and lower altitudes, creep north and into higher elevations.

Since a breakthrough discovery by a team led by the biochemist Dr Jennifer Doudna at the University of California, Berkeley in 2012, the revolutionary gene-editing innovation known as Crispr has shocked the world and altered our preconceived notions about our planet and our place on it.

The pages of many widely read magazines and journals are currently consumed by the topic of Crispr and mosquitoes. First successfully used in 2013, Crispr is a procedure that snips out a section of DNA sequencing from a gene and replaces it with another desired one, permanently altering a genome, quickly, cheaply, and accurately.

The Bill and Melinda Gates Foundation has been funding research into mosquito-borne diseases since its creation in 2000. In 2016 it made investments in Crispr mosquito research totalling $75m. Our investments in mosquito control, said the foundation, include nontraditional biological and genetic approaches as well as new chemical interventions aimed at depleting or incapacitating disease-transmitting mosquito populations. These genetic approaches include the use of Crispr machinery to eradicate mosquito-borne diseases, most notably malaria.

The strategic goal of the Gates Foundation is the extermination of malaria and other mosquito-borne diseases; it is not to bring the mosquito which is harmless when flying solo, untethered from a hitchhiking micro-organism to the brink of extinction. Of the more than 3,500 mosquito species, only a few hundred are capable of vectoring disease. Prefabricated, genetically modified mosquitoes rendered incapable of harbouring the parasite (a hereditary trait passed down their bloodline) might just end the timeless scourge of malaria. But, as Doudna and the Gates Foundation are aware, gene-swapping technology also has the potential to unleash darker, more sinister genetic blueprints with dangerous possibilities. Crispr research is a global phenomenon, and neither Doudna nor the foundation has a monopoly on its limitless designs, its instruments of implementation or its operational execution.

It has been dubbed the extinction drive, as this is precisely what it can accomplish the extermination of mosquitoes by way of genetic sterilisation. This theory has been floating around the scientific community since the 1960s. Crispr can now put these principles into practice. To be fair, the mosquito altered our DNA in the form of sickle cell and other genetic malarial safeguards; perhaps it is time to return the favour. Male mosquitoes that have been genetically modified with domineering selfish genes using Crispr are released into mosquito zones to breed with females to produce stillborn, infertile or only male offspring. The mosquito would be extinct in one or two generations. With this war-winning weapon, humanity would never again have to fear the bite of a mosquito. We would awaken to a brave new world, one without mosquito-borne disease.

An alternative is simply to make mosquitos harmless, a strategy supported and funded by the Gates Foundation. With gene drive technology, Gates explained in October 2018, essentially, scientists could introduce a gene into a mosquito population that would either suppress the population or prevent it from spreading malaria. For decades, it was difficult to test this idea. But with the discovery of Crispr, the research became a lot easier. And just last month, a team from the research consortium Target Malaria announced that they had completed studies where mosquito populations were fully suppressed. To be clear: the test was only in a series of laboratory cages filled with 600 mosquitoes each. But it is a promising start.

Dr Anthony James, a molecular geneticist at the University of California, Irvine, Crisprd a species of Anopheles mosquito to make it incapable of spreading malaria, by eliminating the parasites as they are processed through the mosquitos salivary gland. We added a small package of genes, explains James, that allows the mosquitoes to function as they always have, except for one slight change they can no longer harbour the malaria parasite.

The Aedes breed is more difficult to tackle, since it transmits a handful of diseases that include yellow fever, Zika, West Nile, chikungunya, Mayaro, dengue and other encephalitides. What you need to do is engineer a gene drive that makes the insects sterile, James said of the Aedes breed. It doesnt make sense to build a mosquito resistant to Zika if it could still transmit dengue and other diseases.

We have valid, although yet unknown, reasons to be careful what we wish for. If we eradicate disease-vectoring mosquito species, would other mosquito species or insects simply fill the ecological niche? What effect would eliminating mosquitoes have on natures biological equilibrium? What would happen if we exterminate species that play an essential but unrecognised role in our ecosystem? We are just beginning to ask these morally fraught and biologically ambiguous questions, and for now, no one really knows the answers.

This is an edited extract from The Mosquito: A Human History of Our Deadliest Predator by Timothy Winegard, published by Text on 26 September and available at guardianbookshop.co.uk

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People v mosquitos: what to do about our biggest killer - The Guardian

The gene drive is one of the latest of advancements in genetic modification of living things. It may also be the most controversial, in a field that has seen more than its fair share of controversy. Traditionally, coverage of genetic modifications in food products has resembled war correspondence:

Side 1Gene drives are a valuable tool for controlling pests and perpetuating beneficial genes through agricultural productsSide 2Gene drives are a dangerous, untested and unnatural genetic changes created to deliberately drive a species to extinction.

Gene drive is a version of gene editinga newer, more precise way to change a DNA (or RNA) sequence, in this case by combining a guide RNA with an enzyme that can make a splice in the exact place where a sequence can be removed, another sequence inserted, or the existing sequence altered. Gene drive takes this to another level, making sure that a new or altered genetic sequence has a greater than 50 percent chance of being inherited. This can be done in a number of ways, some of which already exist in nature, some which are no different than traditional gene editing using CRISPR-Cas9, and others that have triggered a backlash from environmental activist groupsnon-governmental organizations (NGOs) that utilize fear for their own political ends.

So far, this debate has pitted NGOs like Friends of the Earth, Greenpeace and ETC Group (who are opposed to any genetic manipulations in food crops and animals) against scientists, some agricultural companies and even some government regulators (almost all of whom conclude that these products are no more dangerous than those developed through traditional breeding).

The activist effort is part of a long-standing campaign to conflate gene drives, gene editing and traditional transgenics (GMO) as the same technology, with the same scientific certainty (or uncertainty) and risks. In 2016, FOE and others asked for a worldwide moratorium on gene drives:

Gene drives, developed through new gene-editing techniques, are designed to force a particular genetically engineered trait to spread through an entire wild population potentially changing entire species or even causing deliberate extinctions. The statement urges governments to put in place an urgent, global moratorium on the development and release of the new technology, which poses serious and potentially irreversible threats to biodiversity, as well as national sovereignty, peace and food security.

Many times, these activist organizations have claimed the public shares their concerns. Citing survey data, the science community has retorted that most people embrace biotechnology when they recognize that it benefits them directly. But what has been missing from the battle between the pro- and anti-GMO positions is a scientific measure of public opinion on more recent techniques such as gene drive. In 2016, a comprehensive National Academies of Science (NAS) report called for not only continued research on the effectiveness and usefulness of gene drives, but also their ecological risks and engagement with the public. While institutions like FOE and ETC Group objected to the existence of gene drives, they did not represent the opinion of the public.

For the first time, that opinion was actually tested, by researchers at North Carolina State University and the University of Wisconsin. In a paper published in Science Advances, Zack Brown, assistant professor of resource economics at NC State and his colleagues surveyed 1,000 American adults on their opinions of gene drives. What they found, instead of opposition, was support for the technology, with a few caveats:

The survey results could be valuable in this early stage of gene drive (or gene editing, for that matter) development as research could possibly be directed toward designing drive strategies that could incorporate controlsnot an easy thing to do, Brown said in a press release.

This is the right timewhile the technology is still under development and before any release decisions have been madeto gain insights into what the public thinks, what types of information they prioritize from researchers, and who is trusted to carry out this sensitive research, said Michael Jones, a graduate student at NC State and co-author of the published survey results.

Another significant finding in the NC State/Wisconsin study was that Americans surveyed trusted universities and the US Department of Agriculture (USDA) (60 percent) over foreign universities, the US Department of Defense (18 percent) and private companies (16 percent) to research gene drive systems.

The survey did not ask respondents for their trust levels of NGOs like FOE and Greenpeace.

However, in another recently published survey, this one in Current Research in Biotechnology, 113 experts (scientists, government officials, agribusiness professionals) found that gene-edited crops posed little to no risk to society, the economy, human health or the environment. Less than five percent thought the techniques posed a high risk.

The experts, most of whom observed that NGOs generally opposed gene editing, gene drive and any other genetic modification, noted that this opposition is based on speculative risks, those that have no established theory or evidence data. The authors of the study, based at the University of Saskatchewan in Canada, warned that the problem with attempting to reconcile speculative risks with risks grounded in theory and evidence, is that speculative risks can be very fluid and dynamic, changing at will and [frequently] at the whim of eNGO political motives.

These new studies seem to support the idea that consumers are less wary of biotechnology when they know how its being deployed. While public opinion surveys show support with some caveats about taking precautions against accidents and outbreaks, by and large members of the public trust scientists, particularly those in academia and at relevant regulatory agencies to navigate this controversial but promising field of research.

Andrew Porterfield is a writer and editor, and has worked with numerous academic institutions, companies and non-profits in the life sciences.BIO. Follow him on Twitter@AMPorterfield

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Viewpoint: Public supports CRISPR, gene drives to battle infectious disease, plant pestsdespite activist opposition - Genetic Literacy Project

Scientists are reporting the first use of the gene-editing tool CRISPR to try to cure a patients HIV infection by providing blood cells that were altered to resist the AIDS virus.

The gene-editing tool has long been used in research labs, and a Chinese scientist was scorned last year when he revealed he used it on embryos that led to the birth of twin girls. Editing embryos is considered too risky, partly because the DNA changes can pass to future generations.

Wednesdays report in the New England Journal of Medicine, by different Chinese researchers, is the first published account of using CRISPR to treat a disease in an adult, where the DNA changes are confined to that person.

The attempt was successful in some ways but fell short of being an HIV cure.

Still, it shows that gene editing holds promise and seems precise and safe in this patient so far, said Dr. Carl June, a University of Pennsylvania genetics expert who wrote a commentary in the journal.

Thats really good for the field, June said.

Chinese government grants paid for the research, which was done openly with advance notice on a scientific registry and standard informed consent procedures. Some of those steps were missing or questioned in last years embryo work.

There are no ethical concerns on this one, June said.

Gene editing permanently alters DNA, the code of life. CRISPR is a relatively new tool scientists can use to cut DNA at a specific spot.

The new case involves a 27-year-old man with HIV who needed a blood stem cell transplant to treat cancer. Previously, two other men were apparently cured of both diseases by transplants from donors with natural resistance to HIV because they have a gene mutation that prevents HIV from entering cells.

Since donors like this are very rare, the Chinese scientists tried to create similar HIV resistance by editing that gene in blood cells in the lab to try to mimic the mutation.

The transplant put the mans cancer in remission, and the cells that were altered to resist HIV are still working 19 months later. But they comprise only 5 percent to 8 percent of such blood cells, so theyre outnumbered by ones that can still be infected.

They need to approach 90 percent or more, I think, to actually have a chance of curing HIV, June said.

Scientists are testing various ways to make the gene editing more efficient, and our results show the proof of principle for this approach, one study leader, Hongkui Deng of Peking University in Beijing, wrote in an email.

One very encouraging result: multiple tests show that the editing did not have unintended effects on other genes.

One of the concerns is that they could make a Frankenstein cell, that they would hit other genes instead of the intended target, so its good that this did not happen, June said.

China appears to be moving fast on such research and may get treatments approved sooner than the United States, June said. He has financial ties to some gene therapy companies and is leading a different study testing CRISPR to fight cancer in the U.S. Three patients have been treated so far and some results are expected by the end of this year.

Several other U.S. studies have been trying to control HIV by altering patients own blood cells using a different gene-editing tool called zinc finger nucleases. The first such test began a decade ago in the U.S.

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Gene-editing tool shows promise in fight against HIV - The Columbian

For the first time, scientists used the gene editing tool CRISPR-Cas9 in an attempt to treat an HIV patient in China. The patient showed improvements after the procedure and did not experience side effects.

The scientists at Peking University in Beijing utilized CRISPR to delete the gene called CCR5 from stem cells in a donated bone marrow. CCR5 is known for contributing to HIV infection.

A 27-year-old patient diagnosed with AIDS and acute lymphoblastic leukemia received the transplant. Doctors said the new and modified bone marrow should help treat his cancer and eliminate HIV.

"After being edited, the cells -- and the blood cells they produce -- have the ability to resist HIV infection," lead scientist Deng Hongkui told CNN.

The patient went under the knife for the bone marrow transplant in 2017. In early 2019, scientists said the man's acute lymphoblastic leukemia was in complete remission.

The stem cells with the editedCCR5 gene also stayed in his system 19 months after the procedure. The team published the results in The New England Journal of Medicine.

However, the new cells did not completely eliminatethe HIV virus. Scientists said the patient lacked enough amounts of stem cells to treat the disease.

Cells in the transplanted bone marrow carried only 5 percent to 8 percent of the edited CCR5. But it might not be a major problem for future experiments since enhancing the gene editing technique may improve outcomes, the scientists said.

"In the future, further improving the efficiency of gene-editing and optimizing the transplantation procedure should accelerate the transition to clinical applications," Deng said.

The initial study mainly aimed to test the safety and feasibility of using genetically-edited stem cells for AIDS treatment. Deng said one key finding is that the procedure did not cause any negative effect.

He added CRISPR has the potential to end blood-related diseases such as AIDS, sickle anemia, hemophilia and beta thalassemia.

Dengs team is not the first group to explore the use of CRISPR. China has been increasing its investment in the gene editing tool, which led to a number of first time experiments.

In 2016, the government announced biotechnology as part of its new Five-Year Plan. China is the first country to allow the use of CRISPR in humans and for the modification of nonviable human embryos.

CRISPR/Cas9 continues to provide scientists new ways to understand and fight previously untreatable diseases. Pixabay

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Scientists Use Gene Editing To Help HIV Patient Delete Disease In China - Medical Daily

In the quest for the next cancer cure, few researchers bother to look back at the graveyard of failed medicines to figure out what went wrong.

The number of failures is staggering: 97 percent of the time that a new drug is tested in a clinical trial for a particular type of cancer, it never makes it to the market. That means the humans (and animals) who participate in these experiments risk their lives on treatments that end up in the dustbin.

Now, a new study helps explain why the rate of failure is so high: In the case of targeted cancer therapies a relatively new class of oncology drugs the medicines may not actually hit the targets researchers intended.

Targeted therapies in cancer work differently from traditional treatments, like chemotherapy. Theyre supposed to be aimed at the specific genes, proteins, or tissues cancer cells rely on to thrive. (Chemo, on the other hand, generally works on all cells that are rapidly dividing, regardless of whether theyre healthy or cancerous.)

The new study, published in Science Translational Medicine, used CRISPR the latest and most precise gene-editing tool available to examine whether 10 different drugs worked as researchers projected. In every case, the researchers found that they didnt.

When the papers authors removed the genes from the genomes of cancer cells that were supposed to be essential for cancer growth, the cells continued to grow. And when they applied the medicines each targeting one of six genes to the newly removed genes, the drugs killed the cancer cells anyway. In other words, even when the supposed target of the therapies was fully deleted, the drugs worked.

This suggests its possible that a big driver of cancer-drug failures in clinical studies is that the drugs dont actually work as drug developers intended.

I hope this paper will help people see the need to raise to bar in terms of how we choose and validate cancer drug targets, said William George Kaelin, a Harvard University professor of medicine who was not involved in the study.

The study should also be a wake-up call for drug developers: [They] should make sure their drugs stop working if the target protein has been genetically removed, said Nathanael Gray, a Dana-Farber Cancer Institute cancer biologist, who was also independent of the research.

The finding is definitely fascinating. But so is the story of why the researchers decided to run the study in the first place and use the latest gene-editing technology to reanalyze, and possibly debunk, previous findings in cancer clinical studies.

A few years ago, one of the papers authors Jason Sheltzer, a research fellow in cancer biology at Cold Spring Harbor Laboratory and his colleagues became interested in a gene called MELK, which is supposed to serve as a biomarker for aggressive breast cancer in patients with a poor prognosis. In the US, some 270,000 new cases of invasive breast cancer will be diagnosed in 2019, and nearly 42,000 women are likely to die from the disease, according to the American Cancer Society.

The researchers started to tinker with the gene using CRISPR and found they couldnt reproduce many of the previous findings about MELK that had been uncovered using older gene-analyzing technologies, such as RNA interference. Namely, even when MELK was cut out, the breast cancer cells proliferated.

When a drug that was supposed to target MELK for breast cancer entered clinical studies, the researchers decided to use CRISPR again, this time to edit out the gene to see whether the drug still worked. We found the drug continued to kill breast cancer cells, regardless of whether the MELK it was targeting was present in the breast cancer genome, said Sheltzer.

This led Sheltzer and his colleagues to a big question: Had they just studied a uniquely bad cancer drug or did we stumble upon a bigger problem? he recalled. The extremely high failure rate [in cancer clinical trials] made us suspect there might be other instances of poorly designed drugs and poorly researched drug targets being tested in human patients.

Enter the new study. Sheltzer and his co-authors chose 10 drugs and drug targets that, like MELK, were at various stages of clinical development. They focused mainly on targets that had been discovered using RNA interference, again, a once-popular gene-analyzing technology that predated CRISPR. And they suspected that like MELK maybe itd been leading researchers down the wrong path.

In each case, they used CRISPR to cut out genes from the genomes of the cancer cells they were looking at genes thought to be essential for cancer growth. And they found that in every case, the drugs killed the cancer cells even though the gene that was supposed to be driving the cancer had been removed.

We wound up with 10 drugs that are potent anti-cancer agents. So we think that if we can figure out what these drugs actually do, we might be able to discover new cancer targets or we might be able to find patients who are more likely to respond, Sheltzer said.

Its also possible this kind of mistaken target helps explain why drugs fall short as they make their way through more and more rigorous stages of clinical studies.

But there could also be other explanations for the misfired targets. Sheltzer acknowledged that they chose medicines primarily discovered with RNA interference technology. And, Technology is always improving. So a lot of the drugs that are being tested in patients now were discovered and characterized five to 10 years ago. Its possible that targeted therapies, discovered more recently using newer genetic technologies, are more precise.

Both Kaelin and Gray shared a word of caution about the study: The researchers focused on targeted drugs that were already known to be problematic. As Kaelin put it, [They] picked drugs against targets where there was never, in my opinion, strong genetic data to support them. So, perhaps, cancer drugs with better-established targets would work as projected.

But Sheltzer says honing in on poor performers was part of the point of the study. A lot of cancer drugs get into clinical trials based on very weak genetic evidence, and when you carefully evaluate them, the rationale for targeting particular genes evaporates.

Either way, he and his colleagues hope the research inspires more analyses into why so many cancer drugs dont help patients. Research funding agencies are very interested in finding the next cure, Sheltzer argued, and they arent excited about this research into reproducibility and why some drugs fail. If we want to accelerate the quest for effective, new treatments, maybe they should be.

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Cancer treatment: drugs often fail in clinical studies. Heres a reason why. - Vox.com

Cancer Cells Resort to Cannibalism To Survive Chemo

By consuming neighboring cancer cells, some cells have found a way to obtain the energy they need to remain alive and induce relapse after a course of chemotherapy is completed.

Published in:Journal of Cell Biology

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Bone Marrow May Be the Missing Piece of the Fertility Puzzle

Study shows that when an egg is fertilized, stem cells leave the bone marrow and travel via the bloodstream to the uterus, where they help transform the uterine lining for implantation.

Published in: PLOS Biology

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Alzheimer's Risk Gene Targets the Brain's Immune Cells

The most prevalent genetic risk factor of Alzheimer's disease (AD), apolipoprotein E4, impairs the function of human brain immune cells, microglia.

Published in: Stem Cell Reports

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Researchers Use CRISPR to Correct Mutation in Duchenne Muscular Dystrophy Model

Duchenne muscular dystrophy (DMD) is a rare but devastating genetic disorder that causes muscle loss and physical impairments. Researchers at the University of Missouri School of Medicine have shown in a mouse study that the powerful gene editing technique known as CRISPR may provide the means for lifelong correction of the genetic mutation responsible for the disorder.

Published in: Molecular TherapyRead full story

Were the Neanderthals Wiped Out by a Common Childhood Illness?

The path to extinction for Neanderthals may well have been the most common and innocuous of childhood illnesses and the bane of every parent of young children chronic ear infections.

Published in:The Anatomical RecordRead full story

Failed drugs often are left on the shelf to gather dust. But sometimes, drugs can be dusted down and repurposed. In this article, we profile a new effort to bring drugs back from the dead and find solutions to conditions such as multiple sclerosis and chronic pain.

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A number of projects are underway to harness bioprinting to print functional human tissues, the first step to printing an entire organ. In this article, we take a closer look at three of these projects.

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TSE Explores Microplastics Detection Techniques

Astrocytes

Blood vessels and astrocytes in aging rat retina, confocal imaging, 40x. Blood vessels are shown in blue; astrocytes (supportive cells of the nervous system) are mostly in red. As organisms age, changes in astrocytes might contribute to disease and degeneration.

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7 Days in Science September 20, 2019 - Technology Networks

Genomes of living organism like that of humans can be edited with the technique of CRISPR. Things that could only be imagines earlier can be made possible with this technique, such as reversing congenital conditions or killing off viruses. CRISPR has now found another application in which it equips materials to change their properties when nearby there are specific DNA sequences.

The research behind this technology was done by a team of scientists from MIT and Harvard who also developed multiple types of devices using the technology. These include an electronic circuit that reacts to DNA cues, a microfluidic device with a DNA sensor that activates a valve to open and close and also gels that release drugs. The idea is to deliver therapies, perform diagnostics, and many impossible tasks up till now; by the interaction between human body and a whole set of new smart materials.

With the help of proteins known as Cas enzymes, DNA can be cut by scientists in specific locations by using CRISPR. A single- stranded DNA was used by the scientists in this new research as a structural component or a control mechanism. This gave smart biological functionality to whatever material it is in.

They developed a polyethylene glycol gel containing DNA bound to encapsulated drug. Acrylamide gel with the DNA was also created by the team. An electronic circuit with another gel was also created by the team with idea of advancing the technique where the result was conductive when the DNA strands within it are intact. One of the next things the team is working on is to find a way to use the technology to deliver engineered bacteria to help treat conditions that are gastrointestinal.

Gaurang Tayloris an MD/MBA candidate at the Johns Hopkins School of Medicine and Harvard Business School. He contributes regularly to CardioSource World News and Emergency Physicians Monthly. He is interested in developing scalable, tech-based solutions for medicine and education. He loves to share his knowledge and recent trends in the Healthcare Department by posting various articles. He has experience in medical device pathways and is passionate about understanding the human body.

Mail: gaurang.taylor@storyoffuture.com

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Biological Cues | CRISPR-Responsive Materials - Story of Future

DURHAM Biomedical engineers at Duke University have developed a method to address failures in a promising anti-cancer drug, bringing together tools from genome engineering, protein engineering and biomaterials science to improve the efficacy, accuracy and longevity of certain cancer therapies.

Using a combination of CRISPR-based targeting, a protein depot that allows for sustained release of the drug and a highly potent binding system, the team showed that their new strategy could overcome three critical problems that limit the efficacy of many cancer drugs their limited potency, their quick elimination from the body, and the ability of cancer cells to develop resistance to the drug.

The research appeared online Sept. 4 in the journal Science Advances.

More than 20 years ago, researchers discovered that the protein drug TRAIL, short for TNF-related apoptosis-inducing ligand, could effectively kill cancer cells without harming healthy cells at least, in the lab. TRAIL works by binding to specific protein receptors on cancer cells, ominously called death receptors, sending a signal that causes the cells to self-destruct. Although initial experiments showed the drug worked in a variety of cancer cell lines, including melanoma, lymphoma, pancreatic, prostate, lung, colon and breast cancer, TRAIL and similar drugs surprised researchers by showing limited success in clinical trials.

After more study, scientists pinpointed three reasons why the promising drug failed: TRAIL wasnt potent enough, the drug was being cleared from the body too quickly and some cancer cells were resistant to the therapy.

Duke engineers improve CRISPR genome editing with biomedical tails

Using a combination of three tools a highly potent protein drug, a depot that allows for sustained release of the drug, and CRISPR/Cas9 based gene editing to pinpoint the cause of resistance to the drug the Duke team, which included Mandana Manzari, a recent PhD graduate,Ashutosh Chilkoti, the chair of Duke biomedical engineering, andKris Wood, an assistant professor of pharmacology and cancer biology, demonstrated that their new strategy could provide a solution to these problems and give protein-based anti-cancer biologics like TRAIL that failed in the clinic a second chance.

The real significance of this research for me is the true cross-disciplinary nature of it, said Manzari, first author on the paper and now a post-doctoral researcher at the Memorial Sloan Kettering Cancer Center in New York. This is really the first example Ive seen where were bringing in pharmacology, drug delivery, and genomics to pinpoint the exact circumstances that cause a biologic to fail and then develop solutions.

The first step of the process involved addressing TRAILs limited potency. Typically, cells have multiple death receptors, but a specific receptor called death receptor 5 (DR5) is more prevalent in certain cancer cells. TRAIL, a three-part protein, binds to DR5 and links three death receptors together, sending a signal for cells to self-destruct. TRAIL can also bind to other death receptors and decoy receptors on normal cells. A more potent drug would be specific for a given death receptor, like DR5 that is present on cancer cells, and link together larger numbers of the receptor on a cell surface to send a stronger death signal to the cancer cell.

Manzari produced a highly potent, six-part death receptor agonist (DRA) that could bind six death receptors together and indude a much stronger self-destruct signal.

Next, the team examined how to prevent the super-potent death receptor agonist from being cleared from the body too quickly. They genetically fused the DRA to a temperature-responsive protein called elastin-like polypeptide (ELP), which forms a gel-like depot within a room-temperature solution. After the solution is injected under the skin, it dissolves, releasing the DRA over a longer period of time.

Duke researchers: Single CRISPR treatment provides long-term benefits in mice

Finally, Chilkoti and Manzari partnered with Kris Wood to better understand what caused certain cells to resist death by TRAIL or death receptor agonist (DRA). The team systematically disabled various genes in the cancer cells using CRISPR/Cas9 until they could deduce which were responsible for TRAIL or DRA resistance. Then they selected drugs to target the proteins produced by those genes and paired them with the DRA slow-release depot.

This work opens another exciting avenue for targeting a critical cell death pathway in cancer, an area of increasing interest in the translational cancer therapeutics community, Wood said.

When we figured out the genes that drive resistance, we were able to map them to commercially-available drugs that could specifically target the proteins that come from those genes, said Manzari. It basically gave us a platform to figure out what drugs we can combine with the DRA in cases where this drug or other protein drugs dont work well to nip that resistance in the bud.

With their triple-whammy tool, the team was able to effectively overcome intrinsic resistance, repress tumor growth and extend survival in mice that were implanted with colorectal cancers from human patients that are highly resistant to treatment with TRAIL.

Now, the researchers are considering how they could apply this method to other protein and small-molecule drugs that face similar barriers that limit their effectiveness.

I think the thing that really sets this approach apart is designing each piece of the platform rationally to address a specific problem and bringing them all together holistically to solve three critical problems that limit not just TRAIL, but many new cancer therapies, Chilkoti said.

Typically the protein engineering is one platform, the ELP strategy is one platform and the genomic screen strategy is its own platform, Manzari said. This is a good example of true synergy of engineering, pharmacology, genomics and materials. People always talk about bringing those together, and this is a clear example of that.

(C) Duke University

This research was funded by the National Institutes of Health (5R01EB007025-08, 5R01EB000188-12, 5R01GM061232-16, R01CA207083, and 5T32GM007105) and the Duke University BME/DCI Collaborative Grant.

CITATION: Genomically Informed Small Molecule Drugs Overcome Resistance to a Sustained Release Formulation of an Engineered Death Receptor Agonist in Patient-Derived Tumor Models, Mandana T. Manzari, Grace R. Anderson, Kevin H. Lin, Ryan S. Soderquist, Merve Cakir, Mitchell Zhang, Chandler E. Moore, Rachel N. Skelton, Mareva Fevre, Xinghai Li, Joseph J. Bellucci, Suzanne E. Wardell, Simone A. Costa, Kris C. Wood, Ashutosh Chilkoti. Science Advances, 2019. DOI 10.1126/sciadv.aaw9162

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Duke researchers utilize gene editing to improve cancer drugs' performance - WRAL Tech Wire

September 18, 2019 Last night the U.S. Food and Drug Administration (FDA) approved apalutamide (Erleada) for the treatment of metastatic hormone-sensitive (aka, castration-sensitive) prostate cancer (mHSPC). Apalutamide has previously received FDA-approval for the treatment of non-metastatic castration-resistant prostate cancer (nmCRPC).

PCF funded the initial synthesis of apalutamide at UCLA by chemist Michael Jung, PhD, in collaboration with prostate cancer physician-scientist Charles Sawyers, MD (now at Memorial Sloan Kettering Cancer Center).

mHSPC refers to men whose prostate cancer has spread to areas of the body outside of the prostate itself, and who are responsive to testosterone-lowering agents. This may refer to men who have had prior surgery or radiation and recurred, or men who were initially diagnosed with disease that was already metastatic (outside the prostate). Patients who are hormone-sensitive may have previously received androgen deprivation therapy (ADT) for a certain amount of time, but their cancer has not yet developed resistance to ADT.

This approval is based on results from the randomized phase 3 TITAN clinical trial, which was presented at the 2019 American Society of Clinical Oncology (ACSO) Annual Meeting, held in June, and published in the prestigious medical journal, The New England Journal of Medicine.

The TITAN trial, led by PCF-funded investigator Dr. Kim Chi, MD, of the Vancouver Prostate Centre, tested the addition of apalutamide versus placebo, to ADT in 1,052 men with mHSPC. Patients on this trial could have previously received ADT for no more than 6 months for mHSPC or no more than 3 years if used as adjuvant therapy for localized prostate cancer, and were not on ADT at the time of disease progression and trial enrollment. Patients could also have previously received docetaxel chemotherapy for no more than 6 cycles, and could not have progressed on that treatment.

Compared with a placebo, the addition of apalutamide to ADT significantly reduced the risk of death by 33%, and reduced the risk of radiographic disease progression (tumors growing on scans) or death (whichever came first) by 52%. Apalutamide also significantly delayed the average time to PSA progression, use of chemotherapy, and pain progression. Apalutamide was shown to prolong survival of patients with both low and high volume metastatic disease. The treatment combination of apalutamide and ADT was considered tolerable, and quality of life in patients receiving apalutamide was similar to those receiving placebo in addition to ADT. Adverse effects that were higher in patients receiving apalutamide vs placebo included rash (27% vs. 8.5% of patients), hypothyroidism (6.5% vs. 1.1% of patients), and fractures (6.3% vs. 4.6% of patients).

More information on this approval can be found here.

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BREAKING NEWS: FDA Approves Apalutamide (Erleada) for the treatment of metastatic hormone-sensitive prostate c - Prostate Cancer Foundation

New research was presented at ASTRO 2019, American Society for Radiation Oncology Annual Meeting, from September 15-18 in Chicago. The features below highlight some of the studies that emerged from the conference.

With survival of HPV-positive oropharyngeal cancer patients having improved remarkably over the past 2 decades, efforts are now being made to reduce long-term adverse effects of treatment in this population. Options include reducing chemotherapy, radiation, or both while hoping to maintain optimal outcomes. To test the validity of doing so, researchers assigned mostly non-smoking patients with low-risk human HPV-positive head and neck cancer to lower-dose intensity-modulated radiotherapy (IMRT) plus concurrent chemotherapy or IMRT alone. Rates of progression-free survival at 2 years were 90.5% in the combination group and 87.6% in the IMRT alone group. A pre-specified threshold for swallowing-related quality of life on the 100-point MD Anderson Dysphagia Inventory was surpassed by both groups, with scores of 85.3 in the combination group and 81.8 in the IMRT along group; these scores were down 5.6 and 6.2 points from baseline, respectively. Local recurrence rates at 2 years were 3.3% in the combination group and 9.5% in the IMRT group, while distant metastasis rates were 4.0% and 2.1%, respectively.

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Although the standard of care for early-stage favorable Hodgkins lymphoma (HL) includes radiotherapy, small studies suggests that metabolic response assessment after two cycles of chemotherapy using FDG-PET (PET-2) can predict the individual outcome and PET-2 negativity might allow reducing the overall treatment intensity. To assess whether omitting consolidating radiation therapy in patients with negative PET-2 is feasible without loss of efficacy as determined by progression-free survival (PFS), researcher randomized adults with newly diagnosed, early-stage favorable HL to standard combined modality treatment (CMT) or PET-guided treatment, whereby radiation therapy was restricted to those with a positive PET after CMT. The researchers found that omitting radiotherapy worsened disease control. Rates of 5-year PFS were 93.4% in standard therapy group and 86.1% in the de-escalation group. However, estimated 5-year overall survival rates were about the same in both groups. Among PET-2-negative and -positive patients, estimated 5-year PFS rates were 93.2% and 88.1%, respectively. In early-stage favorable HL, radiation therapy cannot be safely omitted from standard CMT without a clinically relevant loss of tumor control in patients with negative PET-2, conclude the study authors.

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Prior research has raised concerns about the cosmetic outcomes associated with partial breast irradiation (PBI), with one study finding adverse cosmetic rates to be higher in those treated with PBI than in those treated with whole breast irradiation (WBI). To compare outcomes with PBI versus WBI in women who underwent lumpectomy, researchers asked these patients and their physicians to rate the cosmetic outcomes of the procedure on the treated breast (vs the untreated breast) as excellent, good, fair, or poor at baseline, 12 months post-treatment, and 3 year post-treatment. At 3 years, reports of total satisfaction were 81% in PBI group and 86% in the WBI, while rates of somewhat dissatisfied were 1% and 2%, respectively and less than 1% in both groups were totally dissatisfied with their treatment. Treating physicians rated outcomes as comparable at 12 months but thought them to be better for WBI at 3 years, while physicians blinded to the therapy who reviewed photos of the patients breasts rated outcomes as equivalent at both 1 and 3 years.

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Prior studies suggest that the addition of high doses of radiation to immunotherapy in some cases results in a therapeutic synergy in which some tumors not targeted by radiation will shrinkthe abscopal effect. To determine if adding a high dose of radiation in just a few fractions to a single site of disease could reinvigorate the immune response in patients who progress on immunotherapy, study investigators looked for the abscopal effect in patients with non-small cell lung cancer (NSCLC) and at least two measurable tumors whose cancer progressed after pembrolizumab treatment. Among 50 patients who were immunotherapy-nave and began pembrolizumab when the trial started, 16 experienced disease progression and were treated with stereotactic body radiotherapy (SBRT) and six had progressed on anti-PD-1 therapy at the time of enrollment in the trial and were immediately treated with SBRT. Of these 22 patients, 9.52% achieved a partial response that was sustained beyond 1 year and 47.62% achieved stable disease. Patients with elevated tumor infiltrating lymphocytes scores (2 or 3 on a 0-3 scale) showed improved progression-free survival compared with patients who had lower scores (0-1), with a mean of 215 versus 59 days, respectively.

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Men should be offered hormone therapy while undergoing salvage radiotherapy (SRT) following prostate cancer surgery and subsequent biochemical recurrence, according to current treatment guideline recommendations. However, such therapy has been associated with adverse events but no overall survival benefit in those treated with early SRT. Thus, researchers conducted a secondary analysis of the study on which the guideline recommendation is based to determine if a patients pre-SRT PSA level could serve as a prognostic and predictive biomarker of benefit or harm from anti-androgen therapy. While overall survival was significantly improved in men with a PSA greater than 1.5 ng/mL following hormone therapy, those with a PSA level below 1.5 ng/mL experienced no significant overall survival benefit. In fact, men with PSA levels of 0.6 ng/mL were nearly twice as likely to die from non-cancer causes with the addition of hormone therapy, and between three and four time more likely to experience a combination of severe cardiac events and neurologic issues.

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Conference Highlights: ASTRO 2019 - Physician's Weekly

There's nothing more frustrating than waiting month after month for those two pink lines to pop up. Struggling with infertility can be a heartbreaking experience, but in many cases, the solution may be more simple than you realize.

One of the things that can affect your chances of getting pregnant is an imbalance of hormones. Now, you can take matters into your own hands by using an at-home hormone test to find out if your hormone levels are the source of the problem.

Here, experts give you a better understanding of the key hormones that can affect your fertility and explain how to have your levels tested.

While it's true that we have tons of different hormones coursing through our bodies, only a few come into play when we're trying to conceive. Below, we've listed some of the key fertility hormones you'll want to get familiar with if you suspect a hormone imbalance might be affecting your ability to get pregnant. The good news is, a hormone test will give you insight into these important hormones.

FSH is of the most important hormones for fertility, FSH or follicle-stimulating hormone is responsible for maintaining cycle regularity and producing healthy eggs.

LHor luteinizing hormone, may sound familiar; it's the hormone that's measured in at-home ovulation predictor kits (OPKs). LH is the hormone that tells your body to release an egg that's ready to be fertilized.

AMH, also called anti-Mullerian hormone, is responsible for maintaining the immature eggs your body has. If you come across a hormone test that measures your ovarian reserve, or how many eggs you have left, it's measuring AMH.

Progesteroneis a key player both in preparing the body for pregnancy and for helping a new pregnancy to continue. Often when a woman suffers repeated miscarriages, plummeting progesterone levels are the culprit.

Prolactin If you're thinking that this hormone is the one that handles milk production, you're correct! But it's also a key player in ensuring your cycle stays regular, which is essential when trying to conceive.

T3 and T4 Many women don't realize this, but these thyroid hormones have a major influence on the ability to get pregnant. In fact, Dr. Elena Villanueva with Modern Holistic Health, says, "The thyroid gland, female reproductive organs, and adrenal glands are intricately connected. If there is an issue with either the thyroid or the adrenals, becoming pregnant can be a challenge. The good news is a hormonal imbalance test for thyroid or adrenal dysfunction can reveal issues that can be easily fixed in most cases. Many women find that when their thyroid is brought back into healthy hormone range they easily become pregnant."

First, it's important to know that if your hormones are out of whack, it's not your fault, and you're not doing anything wrong.

"For better or worse, there are very few things we can do lifestyle-wise that negatively (or positively) impact hormone production and balance," says Dr. Emily Jungheim, a board certified reproductive endocrinologist and fertility expert at the Women & Infants Center in St. Louis, Missouri. "One lifestyle factor that can be important though is obesity. Extreme athletes can also have issues as low body fat can impact a woman's menstrual cycles and ovulation."

Medical conditions affecting the thyroid and adrenal glands can also affect hormones. In fact, the thyroid is so important to sustaining a healthy pregnancy that it's considered one of the biggest causes of hormone imbalance in women. Another is PCOS, which stands for Polycystic Ovary Syndrome. PCOS is a condition that affects between 6% and 12% of women of childbearing age, according to theCDC. It's characterized by irregular cycles, excess male-pattern hair growth, and infertility.

Other causes of hormone imbalance include the following:

If you've been trying to conceive for more than a few months and you're otherwise healthy, it may be time to consider your hormone levels. Below is a list of symptoms that may indicate a hormone imbalance, courtesy of Dr. Villanueva.

If you've realized by now that fertility isn't exactly a one-size-fits-all thing, you're right! Each woman is different, which can make it tough to determine when it's time to take action. But in general, Dr. Jungheim suggests that if you haven't conceived after 6-12 months of trying (it's fine it keep it to only 6 months if you're over 35), your cycles are totally irregular and unpredictable, or if you have symptoms of PCOS or a thyroid condition, it's wise to get checked.

Another scenario where hormone testing totally makes sense? If you're planning to have your eggs frozen for later use. "An AMH level can be helpful in understanding how many oocytes one might expect to get during a banking cycle or during IVF," she says.

There are two basic methods: you can have your doctor order the tests and have your blood drawn at a lab, or you can try an at-home hormone testing kit. More and more women are turning to at-home testing solutions as a way to kick-start their fertility journey.

It used to be that in order to get a hormone blood test, couples would first have to endure months of not getting pregnant, then schedule a visit to a reproductive endocrinologist. Now, at-home hormone tests for women are allowing couples to take back control and present their findings to their doctor as a first step in determining whether they're dealing with an easily solvable hormone imbalance or something more serious.

Hormones can be measured in blood, hair, urine or saliva, and each at-home hormone test functions a little differently. There are benefits and drawbacks to each method; while a hormone blood test is the traditional method, saliva hormone testing is becoming a widely accepted way of testing, since it gives a better average over time by collecting several different samples of spit in a single day (gross, we know).

If you decide to try an at-home test, it's a good idea to discuss your findings with your own doctor, especially if anything unusual pops up. Not only will they want to add the information to your medical history, but they may want to perform their own tests to confirm. Due to the nature of their profession, doctors are natural skeptics of any at-home test.

"There are a number of at-home hormone tests. Some are reliable and some are not, but all should be confirmed and interpreted with a medical provider if someone is concerned about an underlying medical condition or if someone is struggling with fertility," says Dr. Jungheim. That's why we recommend using at-home hormone tests in conjunction with your doctor's care rather than in lieu of it.

Here are a few of the top hormone level tests you can buy to measure your hormones in the comfort of your own home:

Proovis an at-home hormone test that uses urine to detect levels of progesterone in the bloodstream. In a typical cycle, progesterone is a hormone that's released immediately after ovulation; it's critical for a healthy pregnancy to occur.

Everlywell hormone test for women measures many of the fertility hormones mentioned above, like estradiol, FSH, AMH and LH with a simple finger prick in the comfort of your home. Results are promptly reviewed by a board-certified physician.

Modern Fertility uses a simple but effective private dashboard to deliver results, this hormone test measures different hormones based on whether and what type of birth control you use. Testing can be performed at home or in a designated lab.

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Could a Hormonal Imbalance Be Affecting Your Fertility? Here's What You Need to Know - Yahoo Lifestyle

Hormones are natural substances made by our glands in our body and the network of glands that make hormones is termed as endocrine systems. These hormones are carried through bloodstream and act as a messenger between one part to another part of our body. Hormone therapy is one of the major modalities of medical treatment for cancers which involves manipulation of the endocrine systems through exogenous administration of steroid hormones or drugs inhibiting or interrupting activities of specific hormones. Surgical removal of certain endocrine organs for instance oophorectomy can also be employed as a part of hormone therapy. In hormone therapy physician generally start with hormone receptor test that let caregivers to measure amount of cancer proteins or hormone receptors within a cancer tissue. By estimating the amount of hormones such as estrogen or progesterone the test either can be positive or negative. A positive test indicates growth of cancer cells with the help of hormones. In such cases physician divert the hormone therapy by blocking the interaction of hormones with the hormone receptor. Alternatively, in case of negative hormone receptor test which signifies null effect of hormones in growth and development of cancer cells other effective treatments can be rendered to cure cancer.

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A hormone therapy can be rendered either before or after a primary treatment. In case it is rendered before the primary treatment it is medically termed as neoadjuvant treatment which kills. Neoadjuvant treatments help to kill cancer cells and contribute to the effectiveness of the primary therapy. If hormone therapy is given after the primary cancer treatment, it is called adjuvant treatment. Adjuvant therapy is given to improve the chance of a cure. Now a day hormone therapy is widely used in treating breast and prostate cancer. In breast cancer the female hormone estrogen are primarily responsible for stimulating the growth and development of breast cancer cell in majority of cases. Recently in 2014, aromatase inhibitors such as Arimidex and Femara have been approved for treating breast cancers through hormone therapy. Apart from these FDA approved Zoladex Lupron can also be used in curing breast cancers through hormone therapy. In case of prostate cancer a variety of medications can be used as hormone therapy. Male hormones, such as testosterone, stimulate prostate cancer to grow. Hormone therapy is given to help stop hormone production and to block the activity of the male hormones. Some of the antiandrogens used as inhibitors of prostate cancer cell growth encompass flutamide, enzalutamide, bicalutamide, and nilutamide among others. some of the other cancers to which hormone therapy is gaining acceptance now a day include womb cancer, kidney cancer, ovarian cancer among others.

Major drivers to global cancer hormone therapy include rising incidences of cancer across globe. Statistically according to WHO cancer accounts for 8.2 million deaths in 2012 and it is estimated that annual cancer cases is expected to rise from 14 million in 2012 to 22 million by 2022. Rising awareness among physician and patients towards alternative cancer therapy processes such as target therapy, immunotherapy or hormone therapy is likely to uplift the market in forthcoming years. Side-effects associated with hormone therapy are major restraints to growth and acceptance of therapy. Some of the common side-effects associated with hormone therapy for cancer include nausea, vaginal spotting, irregular menstrual periods, skin rashes, loss of appetite, vaginal dryness, impotence and male breast enlargement among others.

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Prominent companies operating the global cancer hormone therapy market include AstraZeneca plc, Novartis International AG, Merck & co., QuatRx Pharmaceuticals and Pfizer, Inc. among others.This research report analyzes this market on the basis of its market segments, major geographies, and current market trends. This report provides comprehensive analysis of Market growth drivers Factors limiting market growth Current market trends Market structure Market projections for upcoming years

This report is a complete study of current trends in the market, industry growth drivers, and restraints. It provides market projections for the coming years. It includes analysis of recent developments in technology, Porters five force model analysis and detailed profiles of top industry players. The report also includes a review of micro and macro factors essential for the existing market players and new entrants along with detailed value chain analysis.

Reasons for Buying this Report This report provides pin-point analysis for changing competitive dynamics It provides a forward looking perspective on different factors driving or restraining market growth It provides a six-year forecast assessed on the basis of how the market is predicted to grow It helps in understanding the key product segments and their future It provides pin point analysis of changing competition dynamics and keeps you ahead of competitors It helps in making informed business decisions by having complete insights of market and by making in-depth analysis of market segments It provides distinctive graphics and exemplified SWOT analysis of major market segments

Note: Although care has been taken to maintain the highest levels of accuracy in TMRs reports, recent market/vendor-specific changes may take time to reflect in the analysis.

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Cancer Hormone Therapy Market 2019 By Demand Services, Developments, Advancements, Application, Platforms Types, Industry Growth Drivers and...

CHICAGO Women with breast cancer who want to take advantage of the more convenient course of partial breast irradiation (PBI) can now rest assured they are not going to have worse cosmetic outcomes by foregoing whole breast irradiation (WBI). This is the conclusion from the final report from the pivotal phase 3 trial (RTOG 0413) that compared the two approaches.

"I think this trial has confirmed the ASTRO consensus statement" that describes the patient population that is suitable for PBI, commented lead author Julia White, MD, professor of radiation oncology at the Ohio State University Comprehensive Cancer Center.

"A good population for PBI includes women over the age of 50 who have stage 1 breast cancer that is hormone sensitive, who take endocrine therapy for it, as well as ductal carcinoma in situ (DCIS) patients whose tumors are less than 2.5 cm in size," she said.

"These patients, who represent probably around 30,000 women a year, need to know that PBI is a good alternative for their cancer outcome and now, I don't think concern about cosmetic outcomes should be a barrier," she added.

White was speaking here at a press briefing at the American Society for Radiation Oncology (ASTRO) annual meeting, where she presented the final report from this trial.

The NRG Oncology/NSABP B39-RTOG 0413 study involved more than 4000 women with stage 0, I, or II breast cancer who had undergone a lumpectomy and were randomly assigned to either WBI or PBI.

WBI was administered at a dose of 50 Gy at 2.0 Gy/fraction or 50.4 Gy at 1.8 Gy/fraction to the whole breast, followed by an optional boost to 60 Gy for 5 to 6 weeks.

Women assigned to the PBI arm received 10 fractions, twice a day, for 5 to 8 days at a dose of 34 Gy in 3.4 Gy fractions. PBI was delivered via interstitial brachytherapy or by mammosite balloon catheter or in a dose of 38.5 Gy in 3.85 Gy delivered in 3-dimensional conformal radiotherapy.

Previously reported oncological outcomes from this trial showed that breast recurrence rates after PBI were within 1% of breast recurrence rates after WBI.

"The really good news was that 95% of all women had no recurrence out to 10 years," White added, "and the risk for distant metastases after each procedure was also pretty close at between 1% to 2% between the two approaches," she said.

At the ASTRO meeting, White presented results from a quality of life substudy that showed that cosmetic outcomes were equivalent between WBI and PBI as well.

In the substudy involving 900 women, breast cosmesis was assessed by the patients themselves, as well as by the accruing-site physician and by central review of digital photos by physicians blinded to the randomized treatment arm. Digital photos of the breasts were collected at baseline, and at 1 and 3 years after patients received either WBI or PBI.

The goal of the current substudy was to see if PBI induced changes in breast appearance that were equivalent to those induced by WBI as assessed by the 3 separate groups using the Global Cosmetic Score (GCS).

"Patients were stratified by chemotherapy use and no-chemotherapy use," the investigators noted. Just under half (47%) of the group had received chemotherapy.

Based on the patients' own assessment, changes in cosmetic outcomes at 36 months were equivalent for women who received WBI or PBI based on GCS scores in both chemotherapy recipients and nonchemotherapy recipients at 0.06 vs 0.04, respectively.

When the two groups were combined, change in cosmetic outcomes at 36 months were again equivalent at a GCS of 0.045, as White reported.

Judged by accruing-site physicians, change in the GCS again at 36 months was not quite equivalent in chemotherapy receipts who received PBI compared with WBI, and the same was true in nonchemotherapy recipients. In both groups, cosmetic outcomes were judged to be slightly worse following PBI compared with WBI, White reported.

However, when the two groups were combined, GCS changes were equivalent between PBI and WBI at a change in GCS score of 0.045, she observed.

When the digital photos were assessed by central reviewers, cosmetic outcomes were not equivalent among chemotherapy users, where PBI was deemed to be inferior to WBI.

However, the reverse was true for patients who did not undergo chemotherapy, among whom cosmetic outcomes in breasts treated with WBI were judged to be worse than they were for breasts treated with PBI.

Again, however, when the two groups were combined, cosmetic outcomes as assessed by central reviewers were felt to be equivalent for PBI and WBI, at a change in GCS of 0.028.

White also noted that there were no significant differences in mean changes in GCS as assessed by both patients and by digital photos reviewers at each time point that cosmesis was assessed during the study interval.

The only differences observed between mean changes in GCS at each time point of assessment were seen among accruing-site physicians who, at 36 months, rated changes in cosmetic outcomes at 0.43 for breasts treated with PBI vs 0.17 for breasts treated with WBI, which was statistically significant (P = .001).

On the other hand, patient satisfaction with cosmetic outcomes in those who had received chemotherapy, those who had not received chemotherapy, and the combination of the two groups were all equivalent between those who had undergone WBI and those who had received PBI.

"I think this is the link we were waiting for," White observed.

White noted that an earlier, similarly designed study referred to as the RAPID trial again demonstrated that the accelerated PBI regimen used in the trial was noninferior to WBI in preventing local recurrence, but there was an increase in late normal tissue toxicity and cosmetic outcomes were judged to be worse with PBI than with WBI.

"Thus, there was concern that women would have to sacrifice the appearance of their breast to undergo more convenient radiation," White said.

"But from our analysis, we feel pretty confident that from a patient perspective, there was no difference between treatment arms, and that one arm did not cause more change [in cosmetic outcomes] than the other," she concluded.

Commenting on the findings, Wendy Woodward, MD, professor of radiation oncology, MD Anderson Cancer Center in Houston, Texas, told the press briefing that the outstanding question at the time the RTOG 0413 trial was designed was whether there would be a similar detriment in cosmetic outcomes following PBI as was shown in the RAPID trial.

"Looking at the data very granularly, it is patient satisfaction and patient-reported outcomes that really matter and in this trial, [and these assessments] were completely equivalent," Woodward said.

"So I think this study is a cornerstone for highlighting that PBI is now standard of care for eligible patients and, ultimately, we will see these papers published and we will see practice change as PBI becomes a standard-of-care option," she concluded.

White disclosed travel expenses from IBA and Qfix. Woodward declared she has served as a consultant for Global Health International.

American Society for Radiation Oncology (ASTRO) 2019 Annual Meeting: Abstract #5. Presented September 16, 2019.

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Cosmetic Outcomes Equal After Partial vs Whole Breast Radiation - Medscape

A few weeks ago, a patient came to me complaining of nausea, muscle weakness and fatigue. Her urine was tea-colored despite drinking loads of water. A middle-aged woman, she seemed worried she had cancer or some deadly disease.

Her lab tests revealed significant liver dysfunction. But her symptoms were not due to liver cancer, hepatitis or other disease. It turned out she had liver toxicity from a green tea supplement that she'd heard was a "natural" way to lose weight.

When she stopped taking the supplement at my suggestion, her liver tests gradually normalized and she felt better over the course of a few weeks

I've seen the green tea issue in patients before and often witness the real-life pitfalls of eschewing traditional medicine, science and facts in favor of supplements, herbs and cleanses in the name of "natural" healing.

In an effort to be healthy, patients can easily become ensnared in the potential dangers of alternative medicine or homeopathy.

Let's be clear: Nature has a lot to offer patients.

The Greek physician Hippocrates is said to have reported on the use of St. Johnswort, a flowering plant, for mood disturbances in the 5th century BC Digoxin, a well-studied medicine used to treat heart failure, is derived from the foxglove plant.

Parkinson's patients are often commonly treated with the medication L-dopa, which comes from the plant Mucuna pruriens. Moreover, research repeatedly shows that consuming fruits and vegetables, getting adequate sleep and regular exercise, and spending time outdoors have myriad health benefits.

But nature isn't always so well-intended.

Spoiler alert: Arsenic, cyanide, asbestos and snake venom derive from nature. Refined sugar, a naturally occurring substance and one that lives in most Americans' pantries, is in large part responsible for our country's obesity epidemic. Simply because a substance comes from nature does not mean it is good for us.

An important key to health is using nature appropriately.

And in the case of my patient, she was able to lose weight when we made a clear plan to alter her basic human behaviors. Before she started taking the green tea extract, she was skipping breakfast, drinking the equivalent of two Venti coffees before noon, eating takeout meals for lunch, washing down her late-night dinner with two glasses of wine, sleeping restlessly, and spending too much time sitting and indoors.

Green tea extract was never going to be the quick fix that she - and other patients I have seen - had hoped. It may be attractive as a natural cure for extra body fat, but this promise has not been shown in any studies, according to the National Center for Complementary and Integrative Health at the National Institutes of Health.

The key to helping my patient was pretty basic: looking at her lifestyle, her stress, and creating some structure and accountability for important lifestyle changes.

While she wasn't able to eat like Gwyneth Paltrow would recommend (who can eat Pinterest-perfect meals like that as a mere mortal?), my patient took my advice to heart that she begin eating breakfast, packing healthy leftovers for lunch at work, cutting back the wine to weekends only, and getting more exercise on weekends.

As a result, she started sleeping better and feeling more energetic. Eventually, the weight started coming off, too.

Particular patients seem to be more susceptible to the lure of "naturopathic" medicine or homeopathy. Patients who have vague symptoms that do not fit tidily into a box, for example, are often the ones combing the Internet for answers to their health woes and spending hundreds of dollars on unproven and insufficiently regulated supplements and herbs.

According to the 2012 National Health Interview Survey (NHIS), which included a comprehensive poll on the use of complementary health approaches by Americans, 17.7 percent of American adults had used a dietary supplement other than vitamins and minerals in the past year.

That number is probably larger now: The total sales of herbal and dietary supplements in the United States were estimated to be more than US$8 billion in 2017, the 15th consecutive year of sales growth, according to a market research report. And women were more likely than men to use these products as well as people with more education.

Scientific data is often not the reason patients are drawn to herbal or "natural" supplements, Harvard School of Public Health researchers said. Of supplements users surveyed in 2001, 72 percent said they would continue using supplements despite a negative government scientific study. Patients reported getting much information about herbs from family, friends, advertisements and the Internet.

My patients often consider herbal remedies to be free of side effects, but many "natural" products can lead to toxicity and can dangerously interact with prescription medications.

Compounding the problem is that herbal and dietary supplements are not subject to the same strict regulatory standards as prescription drugs. On its website, NIH's Office of Dietary Supplements says the products "are not required to be reviewed by the FDA for their safety before they are marketed because they are presumed to be safe based on their history of use by humans."

Last year, another patient came in to see me complaining of fatigue, joint pains and abdominal bloating. She had seen a naturopath for these symptoms, who told her she had "chronic Lyme" disease and gave her multiple rounds of antibiotics and a bag full of daily herbal supplements. She said she didn't feel any better.

When we met, she told me she was certain she had Lyme disease that wasn't being adequately treated. In fact, the antibiotics she had been given had only worsened her abdominal issues and caused a new problem: an intestinal infection that causes bad diarrhea.

After 10 days of appropriate antibiotic treatment, her diarrhea was gone but she was back to her tired and achy self. At my recommendation, she stopped the supplements, and her fatigue abated somewhat.

When we discussed her situation further, she revealed to me she suffered from a love-hate relationship with sugar.

Like many of my patients, when she was stressed out she binged on sugar. For most people, ingesting sugar provides a quick hit of the pleasure hormone dopamine, and for some people that rush of dopamine and the accompanying instantaneous boost of energy can become addicting.

The problem is that a high sugar load causes a surge in the hormone insulin, which then results in a sudden drop in blood sugar - which can promote fatigue, weakness and irritability, among other symptoms.

If consumed in excess over time, such dietary sugar can cause abdominal distress, bloating and joint aches. This is what was probably causing my patient's symptoms.

So we made a plan for her to not only cut back on sugar but also fill her diet with healthy stuff to get ahead of hunger and avoid binges. I also recommended she work with a therapist to deal with stress-eating. Her joint aches went away and her energy improved after about two weeks, and she continues to see a therapist for stress-eating issues.

Food - and added support to use it properly - was the fix.

Symptoms such as fatigue, headaches, joint pains and irregular bowel movements are some of the most common complaints I see in my office. They can be challenging for physicians to figure out, largely because they require careful and attentive listening by the doctor.

And since more than 40 percent of patients do not tell their doctors about their use of complementary or alternative medicine (including 25 percent who takesupplements and/or herbs), physicians can be bewildered when trying to pin down a root cause for a patient's complaints.

Indeed, these patients are not easily diagnosed after a single lab test - and they are not easily fixed with a supplement.

Occasionally, it takes time with the patient, careful attention to the patient's story, and asking the right questions to get to the bottom of the problem. Often, the solution is right under our nose.

Nature is indeed wonderful, but it doesn't always come in a pill.

Lucy McBride is an internist based in the District.

2019 The Washington Post

This article was originally published by The Washington Post.

Original post:
A Doctor Explains The True Risk of 'Natural' Treatments Like Green Tea Supplements - ScienceAlert

How many times has your doctor said, we will get some blood tests and you are handed a piece of paper that looks like a foreign language or a menu from another country. Often we blindly take the paper order go to the lab and let them take tubes of blood out of us, with many concerned that all their blood will be gone.

I thought we would take a few minutes and discuss common blood tests, what to expect, what tests should you have, and what is new and exciting in blood testing.

First a little trivia for you, the average sized adult has about 1.5 gallons of blood in their body, that comes out to 12 pints of blood. Blood is made from within our bones, the kidney will actually secrete a hormone to tell the body it is time to make more blood. The average age of a blood cell is 120 days after which point it is removed from circulation by the spleen. The spleen acts a recycling plant by keeping the important parts of the red blood cell to be used again. For those of you who donate blood, the blood you donate is totally replaced in your body within 4- 8 weeks. For a simple blood draw of a few tubes of blood, that blood is replaced within days and makes no impact on your total blood volume.

When we say blood, it is often used as a generic word. Our blood actually is a lot more than red blood cells. It can be broken down to red blood cells, white blood cells, plasma and platelets. Each portion of our blood has important roles. Red blood cells carry oxygen to the body and return carbon dioxide to the lungs, White blood cells fight infections and act as a major portion of our immune system, platelets stop bleeding from advancing and plasma carries other factors needed to clot blood. When the blood is clotted we are left with serum which contains, lipids, electrolytes, hormones, proteins and antibodies to list a few.

You may notice when the tech is drawing your blood that they are using tubes with different color tops. The reason for this is that the tubes are treated with chemicals to make sure that the component you are measuring will be accurate. For example, if the tube has no chemicals in the tube, the blood will clot as it should, however if we are looking at components that allow the blood to clot we dont want them all used up, or if we want to count red blood cells we cant count them if they are all clotted and clumped together. So next time you have blood drawn watch as they change tubes and look for the different colors. You will now know why they do that.

So when you hear you are going to have blood tests it is important to understand exactly what they are testing for. The tests are done for many reasons and are specific for the indication being tested for. For example if we are concerned about an infection we will often do a White Blood cell count. We do this because when the body is under stress from an infection it will produce more white Blood cells to help fight the infection, so our WBC count will go up and give the doctor an idea as to how severe the bacterial infection is. What is unique is that viral infections actually cause the WBC count to drop in many cases as the virus impacts the production of WBCs for a short time.

Doctors will order blood tests that give them specific information to a condition that they are concerned about. What about when you are having a physical and the doctor orders basic tests, what are they looking for? The doctor at this time is looking at your body as a whole and not honing in on any specific disorder. In these cases he is essentially doing the same as your car dealer does with their multipoint inspection.

Lets go over the most common tests your doctor may order and what the doctor is checking with each.

1.Comprehensive metabolic Panel- this is a panel of tests that may include close to 30 different tests. These tests will give a snapshot of your kidney function, your liver function, your gall bladder function, your nutritional status, diabetes and your cholesterol. They can do all of these tests out of a simple tube of blood. They will ask you to fast for this as some portions of your cholesterol as well as your blood sugar will be impacted with any recent food intake.2.Complete blood count- this test will look at your White Blood cells, including the number of WBCs as well as what type of white blood cells. Red Blood cells are counted as well including both number of red blood cells but also sizes of the red blood cells. It will also look at how many platelets we have, which is important for blood clotting. These tests are not only signals of general health but also for leukemia, anemia and iron deficiency.3.Lipid Panel- this test looks at our cholesterol and takes it beyond just a total value for our cholesterol. The test breaks it down into our good cholesterol- HDL, our bad Cholesterol LDL and our very bad cholesterol- VLDL. HDL is good cholesterol as it removes harmful material from our blood. The bad cholesterols are bad because they need to blockage in our arteries.4.Hemoglobin A1c- we discussed how eating can impact your blood sugar level for a period of time while the body reacts to the intake. Patients with diabetes work to keep their blood sugar level in a tight range by checking their sugars throughout the day with finger sticks to check the glucose levels. This test will give an idea of a sugar range that your body has been in for the past 120 days as it measures how much sugar is in the Red Blood Cells which circulate for 120 days. This gives the doctor and the patient a better idea as to how well their blood sugar is doing for a long period of time5.Thyroid Function we dont discuss the thyroid much, that small gland that sits in our neck and is the powerhouse for our energy. If the thyroid is working well our energy level is where we need it. When it is not functioning at the right level, often low level, or hypo thyroid, we feel sluggish, fatigued and sometimes depressed. When it is working too much or hyperthyroid our heart may race, we may feel anxious and we may struggle to keep on weight.

These are just a few of the common tests that a physician may order as part of your checkup. It is important that you understand why a test is being done, when you will get the results back and who will explain them to you. Also you want to know how the results compare with your previous tests as slight changes may show a trend that needs more evaluation , intervention or possibly medication. It is good to have your own copy of your test results, with the electronic health record these are often available to you on your phone, in some cases as soon as the test is completed. For some individuals this is good as they know the goal for their diabetes or cholesterol, for others it is not great as many jump to conclusions after seeing a single blood test and then running off to Google the results. Abnormal results should be discussed with your physician or nursing staff, this should include a discussion of what the results mean, what you need to do, and what is the follow up on this abnormal result.

What is on the horizon for blood tests- some very exciting news

There are a few new blood tests worth discussing, three were reported recently.

ABBOTT Concussion blood test

2 weeks ago, Abbott reported the results of a study that showed their blood test for concussions could detect a concussion even when it was not noted on CT Scan. The blood test looks for proteins that are only found in the brain unless there is damage to the brain which allows it to be found in circulating blood stream. The test can be done in real time and takes about 15 minutes to get results, even on the sidelines of a football game or in the ER following some trauma. This test is very important as it may give a definitive answer to the question of a concussion. This information is very important as the current concussion testing is not completely accurate. An accurate diagnosis is key as removing the individual from activities that may result in another concussion is very necessary to minimize long term effects. This study showed that the test was much more accurate than any cognitive and physical exam screening and was also more exact than a CT scan. The test is not commercially available in the US yet but is expected to be soon.

This is important as a study out last week shows that repetitive mild impact to head is causing damage to the blood vessels in the brain. The study reports that repetitive impacts to the head not necessarily the big concussive blows are likely to changes to the micro vessels that deliver blood to our brain. If this blood test can identify those individuals as well we may see a much safer and accurate way to prevent the long term effects of concussions

CANCER SEEK

Cancer SEEK is a blood test that we have discussed in the past. It is a blood test that is being developed to identify 8 cancers from a single tube of blood. Essentially a liquid biopsy. The test will look for known DNA fragments from cancers that would be circulating in the blood stream. What is more interesting is the cancers this test may be able to identify. The current list includes Breast, lung, colon, ovarian, liver, stomach and pancreatic. As you look at this list, imagine that the test is perfected to a point where it can identify the cancer before traditional ways of testing, identify it earlier and more accurately.

The list includes some cancers that when identified are beyond the option for successful treatment. Lung cancer, ovarian cancer and pancreatic cancer comes to mind. Think about being able to eliminate the need for mammograms or colonoscopies to diagnose those two common cancers. The current stand is that this test will complement the current screening tools

Is this futuristic or is it something we will see soon? It may be here sooner than we think. It is currently in a large study and earlier studies have shown it to be quite good. The study that came out last year showed it to be quite accurate for ovarian, liver, pancreas, stomach and esophageal cancer, each of these very important as there are currently no screening test for each of these. The proposed price for this test is $500, which is a very appealing price for both patients and insurers. The ability to identify these cancers early will be lifesaving and game changing. Look for more information on this as well as the ability to potentially participate in a clinical trial, as they work to perfect this test.

LIFESPAN Blood Test

The other test is a bit more controversial, a group of researchers in Europe, are looking to design a blood test that could predict your lifespan as you age. The study is taking data from close to 45,000 individuals aged 18-109. During the follow up period 5,500 of the individuals had passed away. The researchers then looked at biomarkers in the blood and took that data to see if they could create a scoring system that would predict when a person might die. The researchers identified 14 different biomarkers in the blood, when some were found at low levels the chance of death was high, while other biomarkers when found at higher levels were associated with a lower chance of death. The data is being reviewed to see if levels could predict within a timeframe what the future life expectancy would be.

This obviously will be met with resistance from ethical view from a psychological view and from a healthcare utilization view. The concern about rationing health care would be raised quickly, along with utilization. If someone had this test and the results showed a predictive score that the patient would die within 5-10 years what would we do differently? Would we not do some surgery, would we not use expensive medications, would we give limited resources to an individual who had a better score. Would individuals make other life changing decisions based on the knowledge that they have a high chance of dying within the next 5 years. Many may argue that this is good information and that if we can predict our life span why wouldnt we want to know. It would allow us to make choices on work life balance, allow us to make financial decisions, and family and friend decisions.

Probably the biggest issue as this research is more publically available, would be can we change our lifestyle to change these biomarkers and improve or extend our lives. Would we start to track things we never thought were important, like our levels of leucine or valine. Would we find medications that could lower inflammation should be used as regularly as statin drugs are now.

There will be more to come on this study and how its results will be used in routine care in the future.

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Dr. Kevin Most: Blood Tests - WGN Radio - Chicago

Pregnancy test is an indicative marker test for determination of pregnancy, which measures the human chorionic gonadotropin (hCG) in urine samples. The hCG hormone is present in the human body only during pregnancy, which makes it possible for the test to provide accurate results. Various types of pregnancy tests are available in the market, the common one being the home pregnancy test (HPT), which offers benefits of pregnancy testing at home, without the need to visit a physician or gynecologist. Recent technological advancements in home pregnancy test have led to the emergence of digital pregnancy test kits. These test kits offer quick results in a digital format (pregnant / not pregnant) which eliminates the need for interpretation of results by the women, thereby improving accuracy of the test.

Rise in awareness among general population regarding pregnancy test kits and increase in pregnancy rates among women in emerging countries have boosted the adoption of pregnancy test kits globally. Conventional pregnancy test kits, including strip test kits and cup test kits, offer symbolic results of the test (lines or color code format), which need to be interpreted by the user. This increases chances of human error while interpretation of results. Digital pregnancy test kits have eliminated this possibility by offering a digital response (in yes or no format). These distinct benefits of digital pregnancy test kits over the conventional ones have propelled the adoption of digital pregnancy test kits in the global market, thereby driving the consumption and subsequent growth of the product in the global market during the forecast period. Moreover, incorporation of new technologies in the digital pregnancy test kits, such as indication of the pregnancy month of the woman, has been another major factor eliminating the undesired need to undergo ultrasound scans, thus reducing the health care cost of the patient and driving the adoption of these kits in the global market. However, higher price of digital pregnancy test kits than that of conventional ones, along with their unavailability in the emerging countries, is anticipated to restrain the growth of the global digital pregnancy test kits market in 2018.

The global digital pregnancy test kits market can be segmented into product type, distribution channel, and region. Based on product type, the global market can be divided into kits with week indicator and kits without week indicator. Based on distribution channel, the global digital pregnancy test kits market can be classified into institutional sales and retail sales. The institutional sales segment can further be categorized into maternity centers, gynecology clinics, and others. The retail sales segment can be bifurcated into hospital pharmacies, online pharmacies, and retail pharmacies & drug stores.

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In terms of region, the global digital pregnancy test kits market can be segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America dominated the digital pregnancy test kits market in 2017, and was closely followed by Europe, in terms of revenue. Rise in adoption of new and advanced test kits owing to higher awareness among people regarding pregnancy test kits and adequate reimbursement policies for digital pregnancy test kits in the U.S., Canada, and European countries are some of the major factors contributing toward the prominent share of these regions in the global market. Asia Pacific is projected to register a comparatively higher CAGR from 2018 to 2026, owing to higher pregnancy rate in emerging countries, such as China, India, and other Southeast Asian countries and rising adoption of digital pregnancy test kits in these countries.

Key players operating in the global digital pregnancy test kits market include SPD Swiss Precision Diagnostics GmbH, Church & Dwight Co., Inc., Gregory Pharmaceutical Holdings, Inc., and Sugentech, Inc.

The report offers a comprehensive evaluation of the market. It does so via in-depth qualitative insights, historical data, and verifiable projections about market size. The projections featured in the report have been derived using proven research methodologies and assumptions. By doing so, the research report serves as a repository of analysis and information for every facet of the market, including but not limited to: Regional markets, technology, types, and applications.

The study is a source of reliable data on: Market segments and sub-segments Market trends and dynamics Supply and demand Market size Current trends/opportunities/challenges Competitive landscape Technological breakthroughs Value chain and stakeholder analysis

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The regional analysis covers: North America (U.S. and Canada) Latin America (Mexico, Brazil, Peru, Chile, and others) Western Europe (Germany, U.K., France, Spain, Italy, Nordic countries, Belgium, Netherlands, and Luxembourg) Eastern Europe (Poland and Russia) Asia Pacific (China, India, Japan, ASEAN, Australia, and New Zealand) Middle East and Africa (GCC, Southern Africa, and North Africa)

The report has been compiled through extensive primary research (through interviews, surveys, and observations of seasoned analysts) and secondary research (which entails reputable paid sources, trade journals, and industry body databases). The report also features a complete qualitative and quantitative assessment by analyzing data gathered from industry analysts and market participants across key points in the industrys value chain.

A separate analysis of prevailing trends in the parent market, macro- and micro-economic indicators, and regulations and mandates is included under the purview of the study. By doing so, the report projects the attractiveness of each major segment over the forecast period.

Highlights of the report: A complete backdrop analysis, which includes an assessment of the parent market Important changes in market dynamics Market segmentation up to the second or third level Historical, current, and projected size of the market from the standpoint of both value and volume Reporting and evaluation of recent industry developments Market shares and strategies of key players Emerging niche segments and regional markets An objective assessment of the trajectory of the market Recommendations to companies for strengthening their foothold in the market

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Digital Pregnancy Test Kits Market: Strategic Analysis to Understand the Competitive Outlook of the Industry, 2026 - ScoopJunction