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Orphan Diseases Market Key Players analysis … – Digital Journal – Digital Journal

Gene Therapy Clinics | Posted by admin
Aug 25 2017

"Global Orphan Diseases Market- Global Forecast To 2022"

Global Orphan diseases Market information, by Type of Diseases (autoimmune disorders, genetic disorders, blood disorders, cancer, growth disorder, cardiovascular diseases, neurological disorders, respiratory disorders, digestive disorders, eye disorders and Others), by Type of Treatment (gene therapy, cell therapy, drug therapy and others), by End user (hospital and clinics, research laboratory and others) - Forecast to 2022

Market Synopsis of Global Orphan diseases Market:

Market Scenario:

Global orphan diseases market also known as rare disease is growing rapidly. It affects a very small percentage of the global population. Most of the orphan diseases are genetic and is remains throughout the life of the patient. There are no exact number of diseases available but approximately there are about 7000 different rare diseases and disorders throughout the globe. Global orphan diseases market is expected to grow at the average CAGR of 24.9% constantly throughout this period 2015-2022. It is also expected that this market which was US$ 121.6 billion in 2015 will grow to US$ 576.9 billion by 2022. . However due to lack of awareness, correct diagnosis, correct treatments and availability of healthcare facilities are inhibiting the growth of the global orphan diseases market.

Key Players for Global Orphan diseases Market:

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Global orphan diseases market has been segmented

On the basis of types of diseases which includes autoimmune disorders, genetic disorders, blood disorders, cancer, growth disorder, cardiovascular diseases, neurological disorders, respiratory disorders, digestive disorders, eye disorders and others.

On the basis of treatment type it segmented into gene therapy, cell therapy, drug therapy and others.

On the basis of end user the market is segmented into hospital and clinics, research laboratory and others.

Intended Audience

Taste the market data and market information presented through more than 50 market data tables and figures spread in 110 numbers of pages of the project report. Avail the in-depth table of content TOC & market synopsis on Global Orphan Diseases Market- Global Forecast To 2022

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Table of Content

1 Report Prologue 2 Market Introduction 2.1 Definition 2.2 Scope Of The Study 2.2.1 Research Objective 2.2.2 Assumptions 2.2.3 Limitations 2.3 Market Structure 3 Research Methodology 3.1 Research Process 3.2 Primary Research 3.3 Secondary Research 3.4 Market Size Estimation 3.5 Forecast Model 4 Market Dynamics 4.1 Drivers 4.2 Restraints 4.3 Opportunities 4.4 Mega Trends 4.5 Macroeconomic Indicators 4.6 Technology Trends & Assessment 5 Market Factor Analysis


The report gives the clear picture of current market scenario which includes historical and projected market size in terms of value, technological advancement, macro economical and governing factors in the market. The report provides details information and strategies of the top key players in the industry. The report also gives a broad study of the different market segments and regions.

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Orphan Diseases Market Key Players analysis ... - Digital Journal - Digital Journal

Gene Editing in Human Embryos Leaps ForwardHere’s the Science – Singularity Hub

Gene Therapy Clinics | Posted by admin
Aug 22 2017

Imagine walking down the street with a ticking time bomb in your chest, never knowing when your heart may explode.

Or going through five decades of life, having kids, and always wondering when your mind will finally slip away from you. Or worse yet, knowing that one day the same will inevitably happen to your children and your grandchildren.

If there were a curea way to irreversibly correct the faulty biology in yourself and your offspringwould you do it? And knowing that there might be risks, would you be comfortable making that decision for generations to follow?

Last week, a remarkable study published in Nature brought these and other questions back into public discourse. For the first time, an international team led by US scientists used CRISPR, a genetic editing tool, to correct a mutation that leads to heart failure in viable human embryos.

This isnt the first time scientists have tinkered with human embryos. But it is the first that shows that certain off-target effectspreviously thought immensely challengingcan be dealt with in a relatively straightforward way.

In other words, the new technology just brought us one step closer to correcting genetic deficits in humans. And what can be used to right a disease can also be used to enhance a healthy babyartificially altering their intelligence or physical appearance.

To be clear, this study is a long way off from the complicated changes required to make designer babies. This isnt Brave New World or Gattaca.

This is for [the] sake of saving children from horrible diseases, says lead author Dr. Shoukhrat Mitalipov at the Oregon Health and Science University, who previously worked on Dolly the sheep and three-parent babies.

And with this milestone, says Dr. George Church at Harvard University, were one step closer.

The human body runs on the tens of thousands of genes that form the code of life. Sometimes, just a single faulty gene can have devastating consequences, such as Huntingtons disease or hypertrophic cardiomyopathya condition that often leads to heart failure.

For decades, scientists have tried hacking lifes code to cure these genetic diseases at the DNA level. The process seems straightforward: like programmers decoding a bug, scientists would read through the bodys encyclopedia of genes, identify the faulty member, cut and paste the correct code into the original spotand voil, fixed!

The promise of genetic cures seemed easily within reach when a technology called CRISPR came onto the scene in 2012. CRISPR itself isnt a cure. Rather, its a pair of molecular scissors that scientists can direct to almost any point on the human genome and make a precise cut.

The cut triggers a cell to activate a DNA repair program. Almost all cells do this, but embryos go about it slightly differently. If provided with a normal copy of the gene, embryos will use the blueprint gene to reconstruct the broken piece, essentially overwriting the mistaken code. In theory, this leads to fewer mistakes than a normal cells stitch it up repair program, which doesnt use templates.

In practice, however, embryonic DNAs been hard to hack. Just two years ago, Chinese scientists reported giving up on correcting a genetic abnormality in human embryos due to off-target effects, saying that the CRISPR-based technology was too immature.

And for good reason. The safety and ethical barriers are enormous when editing embryosso-called germline editing. The reason is this: after sperm meets egg, the resulting single cell will develop into a persons entire body. This means that any changes to an embryo will (in theory) be present in every single cell in the grown human, including reproductive cells.

In other words, any changes to the embryo will not only affect the person it will become, but also his or her children, and their children and so on. If any unwanted mutation sneaks in during the procedure, the harm is multi-generational.

Then theres the problem of mosaicism. Oftentimes, an edited embryo can lead to a mosaic of genotypes in the resulting cellssome fixed, some not, and the individual still ends up with the disease.

The new study tackles both problems head-on.

Mitalipovs team decided to focus on hypertrophic cardiomyopathy, an inherited disease due to a gene called MYBPC3. People with the condition have two copies of the gene: one normal, one faulty. This means they have a 50-50 chance of passing the condition to their children.

Because the embryo already contains a normal copy of MYBPC3, explain the authors, it already has a blueprint the cell could use to repair the abnormal one. The team recruited a dozen healthy egg donors and one sperm donor that carried the faulty MYBPC3.

Normally, scientists encode all CRISPR components into an external bit of DNA called a plasmid, put that into a cell and rely on the cell to make the necessary proteins and molecules. Mitalipovs team took a more unusual route: using a tiny syringe, they directly injected the CRISPR machinery into either a fertilized embryo or into the egg cell right before fertilization.

In the first case, the CRISPR machinery sticks around for a long time. This increases the chance that it might go rogue and snip parts of the DNA it wasnt designed to cut.

By directly injecting the components, the CRISPR scissors are chewed up by the recipient cell after they do their work: less random snipping, more precision.

The tactic worked. When the team analyzed the resulting embryos at the four- or eight-cell stage, they found 72 percent contained only normal copies of the MYBPC3 gene, compared to roughly 50 percent found in non-edited controls.

Even though the yield of wild type/wild type embryos is still higher, its not 100 percent. We have room to improve, says Mitalipov.

Heres the kicker: using a variety of modern genetic sequencing techniques, the team scrutinized the embryos genomes for off-target effects. They couldnt find any. For all intents and purposes, the edited embryos looked completely healthy.

This doesnt necessarily mean the team avoided all unexpected mutations. It just means any genetic deletions or inserts didnt affect the embryos normal development.

Thats not all. The team also surprisingly found a way to minimize mosaicism. The key is to inject CRISPR components into the egg at the same time as they pumped in the sperm to fertilize it. This is much earlier in the developmental stage than anyone had previously attempted.

It worked. Out of the 58 treated eggs fertilized with the mutant sperm, 42 contained two normal copies of MYBPC3. Only one became a mosaic. In contrast, CRISPRing a fertilized embryo led to 13 out of 54 mosaics.

It makes previous work look pretty amateurish in terms of mosaicism and in terms of off-target effects, says Church.

Surprisingly, rather than bolstering a designer baby future, the study may have inadvertently doused a cold case of biological reality on the sci-fi idea.

Dr. Robin Lovell-Badge, a developmental biologist at the Francis Crick Institute in London, pointed out that the most unexpected result of the study is how the embryo chose to repair the gene.

In one experiment, the team tried introducing an artificial template of MYBPC3 in addition to the normal copy already present in the cell (from the healthy moms). But the cells completely ignored the researchers template, instead exclusively opting to use the maternal MYBPC3 to repair the mutation.

This suggests that you couldnt add anything that wasnt already there, says Lovell-Badge.

To Mitalipov, the crux of the conversation should be solidly based in therapy. My goal has always been to treat genetic diseases that have no cures, to save children, he says.

And there are still a lot of kinks that need ironing out before CRISPR could enter clinics. For one, scientists still hope to increase precision and accuracy. For another, IVF clinics already have solid screening protocols in place to weed out genetic abnormalities before implantation. While CRISPR can, in theory, boost the number of healthy embryos, it would have to work better to justify the cost.

To Dr. Richard Hynes, a cancer researcher at MIT who co-led a national committee that recently published a new guideline for editing embryos, the study is a big breakthrough.

What our report said was, once the technical hurdles are cleared, then there will be societal issues that have to be considered and discussions that are going to have to happen. Nows the time, he says.

Image Credit: University of Michigan via Flickr

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Gene Editing in Human Embryos Leaps ForwardHere's the Science - Singularity Hub

Global Cancer Biological Therapy Market 2017 Size, Development Status, Type and Application, Segmentation … – Digital Journal

Gene Therapy Clinics | Posted by admin
Aug 22 2017

""Cancer Biological Therapy Market"" adds Cancer Biological Therapy Market 2017 Global Analysis, Growth, Trends and Opportunities Research Report Forecasting to 2023reports to its database.

Cancer Biological Therapy Market:

Executive Summary

Biological therapy treatment is done with the help of living organisms, parts of living organisms or laboratory manufactured version of such content. There are various types of biological therapies, which inhibit specific molecules involved in development and growth of cancer tumor. Such therapies known as; cancer targeted therapies.

The global cancer biological therapy market is expected to reach USD 82,276.8 million by 2023 at a CAGR of 4.7% during the forecasted period.

The global cancer biological therapy market is segmented on the basis of phases, types, end users and regions. On the basis of phases, the market is segmented into phase I, phase II and phase III. In stage I & II the real impact of these therapies is seen and giving a success rate of 35% in Phase 1 and 20% in Phase II. The success rate of phase I is 35%.

On the basis on types, the global cancer biological therapy market is segmented into monoclonal antibodies, cancer growth blockers, interferons, interleukins, gene therapy, targeted drug delivery, colony stimulating factor, cancer vaccines and others. Monoclonal antibodies accounted for the largest market share of the global cancer biological therapy market. Colony stimulating factor is the fastest growing market at a CAGR of 5.2% during the forecasted period.

On the basis on end users, hospitals & clinics dominates the global cancer biological therapy market. Registering USD 26,790.6 million in 2016 and expected to reach at USD 38,471.9 million by 2023 at the rate of 4.4 % from 2016-2023.

On the basis of regions, the market is segmented into North America, Europe, Asia-Pacific and the Middle East & Africa. North America has the dominating market for cancer biological therapy. The cancer biological therapy market for North America is estimated at USD 19,481.2 million in 2016 and expected to reach by USD 29,516.9 million by 2023 at a fastest CAGR of 5.10%.

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Key Players

The leading market players in the global cancer biological therapy market include Merck Inc., F. Hoffmann-La Roche Ltd, Novartis AG, Amgen Inc., Bristol-Myers Squibb, Celgene, ELI Lilly and Company, EnGeneIC, and Pfizer

Study objectives

Target Audience

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Key Findings

The reports also covers regional analysis

o US

o Canada

o Germany

o France

o U.K.

o Italy

o Spain

o Rest of Europe

o Japan

o China

o India

o Republic of Korea

o Rest of Asia-Pacific

o Middle East

o Africa


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Traditional Therapy Clinics Ltd (TTC.AX) Money Flow Index Levels in Focus – Stock Daily Review

Gene Therapy Clinics | Posted by admin
Aug 22 2017

Traditional Therapy Clinics Ltd (TTC.AX) shares have seen theMoney Flow Indicator drop below 30, potentially spelling a near-term reversal if it crosses below the 20line. The Money Flow Indicatoris a unique indicator that combines momentum and volume with an RSI formula. Because of its incorporation of volume, the MFI is better suited to identify potential reversals using both overbought/oversold levels and bullish/bearish divergences. As with all indicators, the MFI should not be used by itself. A pure momentum oscillator, such as RSI, or pattern analysis can be combined with the MFI to increase signal accuracy.

The MFI was created by Gene Quong and Avrum Soudack and they believed a reading above 70-80 would signify Overbought territory where a reading below 20-10 would indicate that the conditions wereindicative of an Oversold price level.

Investors might be interested in taking a closer look at additional stock technical levels. After a recent check, Traditional Therapy Clinics Ltd (TTC.AX) has a 14-day ATR of 0.02. The average true range indicator was created by J. Welles Wilder in order to measure volatility. The ATR may help traders to determine the strength of a breakout or reversal in price. It is important to mention that the ATR was not designed to calculate price direction or to predict future prices.

Currently, the 14-day ADX for Traditional Therapy Clinics Ltd (TTC.AX) is sitting at 23.62. Generally speaking, an ADX value from 0-25 would indicate an absent or weak trend. A value of 25-50 would support a strong trend. A value of 50-75 would identify a very strong trend, and a value of 75-100 would lead to an extremely strong trend. ADX is used to gauge trend strength but not trend direction. Traders often add the Plus Directional Indicator (+DI) and Minus Directional Indicator (-DI) to identify the direction of a trend.

Checking in on some other technical levels, the 14-day RSI is currently at 32.37, the 7-day stands at 23.25, and the 3-day is sitting at 9.65. Many investors look to the Relative Strength Index (RSI) reading of a particular stock to help identify overbought/oversold conditions. The RSI was developed by J. Welles Wilder in the late 1970s. Wilder laid out the foundation for future technical analysts to further investigate the RSI and its relationship to underlying price movements. Since its inception, RSI has remained very popular with traders and investors. Other technical analysts have built upon the work of Wilder. The 14-day RSI is still a widely popular choice among technical stock analysts.

Investors may be watching other technical indicators such as the Williams Percent Range or Williams %R. The Williams %R is a momentum indicator that helps measure oversold and overbought levels. This indicator compares the closing price of a stock in relation to the highs and lows over a certain time period. A common look back period is 14 days. Traditional Therapy Clinics Ltd (TTC.AX)s Williams %R presently stands at -100.00. The Williams %R oscillates in a range from 0 to -100. A reading between 0 and -20 would indicate an overbought situation. A reading from -80 to -100 would indicate an oversold situation.

Taking a closer look from a technical standpoint, Traditional Therapy Clinics Ltd (TTC.AX) presently has a 14-day Commodity Channel Index (CCI) of -229.42. Typically, the CCI oscillates above and below a zero line. Normal oscillations tend to stay in the range of -100 to +100. A CCI reading of +100 may represent overbought conditions, while readings near -100 may indicate oversold territory. Although the CCI indicator was developed for commodities, it has become a popular tool for equity evaluation as well.

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Traditional Therapy Clinics Ltd (TTC.AX) Money Flow Index Levels in Focus - Stock Daily Review

Sodium Iodide Symporter for Nuclear Molecular Imaging and …

Gene Therapy Clinics | Posted by admin
Aug 13 2017

Theranostics 2012; 2(4):392-402. doi:10.7150/thno.3722


Byeong-Cheol Ahn

Department of Nuclear Medicine, Kyungpook National University School of Medicine and Hospital, Daegu, South Korea

Molecular imaging, defined as the visual representation, characterization and quantification of biological processes at the cellular and subcellular levels within intact living organisms, can be obtained by various imaging technologies, including nuclear imaging methods. Imaging of normal thyroid tissue and differentiated thyroid cancer, and treatment of thyroid cancer with radioiodine rely on the expression of the sodium iodide symporter (NIS) in these cells. NIS is an intrinsic membrane protein with 13 transmembrane domains and it takes up iodide into the cytosol from the extracellular fluid. By transferring NIS function to various cells via gene transfer, the cells can be visualized with gamma or positron emitting radioisotopes such as Tc-99m, I-123, I-131, I-124 and F-18 tetrafluoroborate, which are accumulated by NIS. They can also be treated with beta- or alpha-emitting radionuclides, such as I-131, Re-186, Re-188 and At-211, which are also accumulated by NIS. This article demonstrates the diagnostic and therapeutic applications of NIS as a radionuclide-based reporter gene for trafficking cells and a therapeutic gene for treating cancers.

Keywords: sodium iodide symporter, molecular imaging, radionuclide-based imaging, gene therapy, radionuclide.

The ability of the thyroid gland to concentrate iodide has long provided the basis for diagnosis and therapeutic management of benign thyroid diseases and thyroid cancer [1]. Thyroid scintigraphy with radioiodines or technetium-99m (Tc-99m) pertechnetate has played a key role in the evaluation of thyroid nodules with its ability of providing anatomical and functional information since the advent of modern endocrinology [2]. Radioiodine via an 'atomic cocktail' was first used medically for thyroid cancer treatment under the Atomic Energy Act since 1946 [3]. Thereafter, millions of patients with benign or malignant thyroid diseases have been given radioiodine for diagnostic and therapeutic purposes with successful outcomes. However, the uptake mechanism of radioiodine into thyroid tissue or thyroid cancers was not fully elucidated until 1996, when the sodium iodide symporter (NIS) was finally cloned [4]. This not only improved understanding of thyroid pathophysiology tremendously, but also offered promising molecular biological strategies of imaging and treatment. Clinical theranostic application of NIS function using radioiodine was projected to biologic preclinical experimental studies after the NIS cloning. NIS expression can be imaged feasibly with simple radiotracers, such as radioiodines or Tc-99m. By the easy imaginable characteristic of NIS, it has been used as an imaging reporter to monitor gene transfer.[5, 6] In addition to the potential as the imaging reporter gene, NIS has been used as a therapeutic gene to treat cancers through its ability to concentrate therapeutic doses of radionuclides in target cells [7-10].

This review is mainly focused on the theranostic application of NIS for radionuclide-based molecular imaging and radionuclide gene therapy in in vivo animal models.

NIS is an intrinsic plasma membrane glycoprotein with 13 transmembrane domains that actively mediates iodide transport into the thyroid follicular cells and several extrathyroidal tissues [11]. This protein plays an essential role in thyroid physiology by mediating iodide uptake into the thyroid follicular cells, a key step in thyroid hormone synthesis. NIS belongs to the sodium/solute symporter family or solute carrier family 5, which drives negatively-charged solutes into the cytoplasm using an electrochemical Na+ gradient [12]. The symporter co-transports two sodium ions (Na+) along with one iodide (I-), with the transmembrane sodium gradient serving as the driving force for iodide uptake; therefore, NIS functionality is dependent on the electrochemical sodium gradient that is maintained by the oubaine-sensitive Na+/K+ATPase pump (Fig. 1) [13].

NIS needs to be localized in the plasma membrane for efficient transportation of iodide into thyroid follicular cells. Poor iodide uptake in thyroid cancer cells compared to thyroid follicular cells is related to impaired targeting and retention of NIS at the membrane. Membrane localization of NIS requires thyroid stimulating hormone (TSH) stimulation; through TSH deprivation, NIS is not retained at the membrane, leading to a decrease in iodide uptake. Although TSH stimulation is essential for efficient NIS trafficking to plasma membrane of thyroid follicular cells, it is possible that TSH-independent mechanisms for the trafficking exist because non-thyroidal tissues also retain NIS at the membrane in the absence of TSH stimulation. One suggested mechanism of NIS targeting to the membrane is the phosphorylation of NIS at serine residues in the carboxy terminus. Protein-protein interaction is another suggested mechanism for the trafficking. NIS contains PDZ, dileucine and dipeptide motifs which might be associated with trafficking [1, 13]. Non-thyroidal cancer tissues also can express NIS; however, only 20-25% of NIS-positive tumors showed iodide uptake partly due to the intracytoplasmic location of NIS [14].

Although expression of NIS is also detectable in normal extrathyroidal tissues such as the salivary glands, gastric mucosa and lactating mammary glands, the expression is not regulated by TSH and is present at lower levels in these tissues than in thyroid tissue. Iodide organification is a particular and unique characteristic of the thyroid gland, and long-term retention of iodide does not occur in the extrathyroidal tissues expressing NIS [15].

Iodide uptake function of NIS. NIS transports 2 sodium ions and 1 iodide ion into the cytoplasm together. The electrochemical sodium gradient generated by the oubaine-sensitive Na+/K+ ATPase pump provides energy for this transfer.

NIS has marked advantages as an imaging reporter gene and as a therapeutic gene compared to other reporter or therapeutic genes due to the wide availability of radiopharmaceuticals and its well understood metabolism and clearance of these radiopharmaceuticals from the body [16].

NIS actively takes up radioiodine and Tc-99m; therefore, its function can be imaged with I-123, I-131, I-124 and Tc-99m [7, 15, 17]. No issues of labeling processes and stability arise when using these radiopharmaceuticals, whereas they may be a major concern of the radiolabeled ligands of other radionuclide-based reporter genes, such as the dopamine D2 receptor or herpes simplex virus thymidine kinase (HSV-tk) genes [16].

I-123 is produced in a cyclotron by proton irradiation of enriched xenon-124 (Xe-124) in a capsule, decays by electron capture to tellurium-123 (Te-123) with a half-life of 13.2 hours, and emits gamma rays with predominant energies of 159 keV (the gamma ray is primarily used for imaging) and 127 keV. I-123, mainly a gamma emitter, has a high counting rate compared with I-131 and provides a higher lesion-to-background signal, thereby improving sensitivity and imaging quality. Moreover, with the same administered activity, I-123 delivers an absorbed radiation dose that is approximately one-fifth that of I-131 to NIS-expressing tissues [18].

I-124 is a proton-rich isotope of iodine produced in a cyclotron by numerous nuclear reactions and decays to Te-124 with a half-life of 4.2 days. Its modes of decay are 74.4% electron capture and 25.6% positron emission. It emits gamma radiation with energies of 511 and 602 keV [19].

I-131 is produced in a nuclear reactor by neutron bombardment of natural Te-127, decays by beta emission with a half-life of 8.0 days to Xe-133, and emits gamma rays as well. It most often (89% of the time) expends its 971 keV of decay energy by transforming into the stable Xe-131 in two steps, with gamma decay following rapidly after beta decay. The primary emissions of I-131 decay are beta particles with a maximal energy of 606 keV (89% abundance, others, 248-807 keV) and 364 keV gamma rays (81% abundance, others 723 keV) [19]. As I-131 emits both beta and gamma rays, it can be used to image NIS gene expression; however, it is not recommended for imaging due to poor image quality (by high energy of the gamma rays) and the high radiation burden (by the beta rays) compared to I-123.

Tc-99m, a metastable nuclear isomer of Tc-99, has a half-life of 6.0 hours and emits 140 keV gamma rays which is an optimal energy for scintigraphic imaging. Tc-99m, the most commonly used radionuclides in routine nuclear medicine imaging, is usually extracted from Tc-99m generators which contain parent nuclide molybdenum-99 (Mo-99) [2].

Recently, F-18 tetrafluoroborate (F-18 TFB) was developed as a positron-emitting radiopharmaceutical that is actively taken up by NIS [20]. The rapid uptake and efflux of F-18 TFB in the rat thyroid cell line parallels the behavior of Tc-99m pertechnetate, which is known to be taken up in cells expressing NIS [20]. Uptake of F-18 TFB to thyroid follicular cells is stimulated by TSH and blocked by perchlorate. It was suggested that F-18 TFB transport occurs with little or no coupling to sodium transport, or that TFB occupies a binding site on NIS but is transported very inefficiently.

I-131, rhenium-188 (Re-188), Re-186 and astatine-211 (At-211), which emit particles from their nuclei, are used for radionuclide therapy on cells expressing NIS [1, 8-10, 13, 21]. Re-188 is an important therapeutic radionuclide, which is obtained on demand as a carrier-free sodium perrhenate by saline elution of the tungsten-188 (W-188)/Re-188 generator system. With a half-life of 17.0 hours and emission of a high-energy beta ray (maximal energy of 2.12 MeV) and gamma ray (155 keV, 15%) for imaging, Re-188 offers the prospect of cost-effective preparation of radiopharmaceuticals for cancer treatment [22]. Cyclotron-driven neutron activator may be an alternative for on-demand supply of Re-188 [23].

Currently, At-211 is the most promising alpha-emitter that has been studied for cancer therapy. It is the heaviest halogen, with no stable isotope. It decays via a double-branch pathway with a mean alpha-energy of 6.7 MeV (42% 5.9 MeV and 58% 7.5 MeV) and a half-life of 7.2 hours. As a consequence of its electron capture branching to its daughter polonium-211, X-rays of 77 to 92 keV in sufficient abundance are emitted, enabling external imaging (including single photon emission computed tomography [SPECT]) and gamma counting of blood samples as additional advantages. However, its widespread use in therapeutic doses is hindered as a result of limited availability of medium-energy cyclotrons with an alpha-particle beam for its production, which is currently feasible at only a few research centers [24]. Table 1 summarizes characteristics of radionuclides which can be used with NIS for diagnostic or therapeutic purposes.

Gamma camera imaging with radioiodine (I-131 or I-123) can visualize metastatic lesions in differentiated thyroid cancer patients who have undergone total thyroidectomy because the lesions are highly efficient at trapping circulating iodine by expression of NIS (Fig. 2) [25]. Radioiodine scintigraphy, once the mainstay of post-therapy imaging surveillance, has largely been replaced by neck ultrasonography as the modality of choice for long-term imaging surveillance, although it still may be used for the detection of occult or distant metastases, particularly in the setting of a newly elevated serum thyroglobulin level [26]. Routine use of radioiodine scintigraphy for surveillance is not recommended for low-risk patients. However, it is still used in patients with intermediate or high risk of recurrence, as well as to assess patients for evidence of recurrence in the setting of an elevated thyroglobulin level with a negative neck ultrasonography. Scintigraphy performed after empiric treatment with high doses of I-131 is more sensitive than the usual diagnostic I-131 scanning [26].

I-124 positron emission tomography (PET) has higher sensitivity for the detection of thyroid cancer lesions with NIS expression compared with I-131 whole body scintigraphy due to lower background noise and the higher resolution of PET imaging than gamma camera imaging. Additionally, PET images can be fused with CT and/or magnetic resonance imaging [27].

Detection and localization of metastatic thyroid cancer lesions by radioiodine scintigraphy or PET rely on the expression of NIS in the cancer cells which accumulate radioiodine [27].

Radionuclides used for diagnostic or therapeutic purposes associated with NIS.

T1/2: half-life, PET: positron emission tomography

A 21-year-old female who underwent total thyroidectomy due to papillary thyroid cancer. Chest simple radiography and CT did not demonstrate any metastatic lesion of the cancer in the neck and chest regions. However, a radioiodine whole body scan revealed lymph node metastases (white arrow) in the right supraclavicular area and diffuse lung metastases (black arrows).

Molecular imaging, defined as the visual representation, characterization and quantification of biological processes at the cellular and subcellular levels within intact living organisms, can be obtained by various imaging technologies, such as optical imaging, nuclear imaging, magnetic resonance imaging (MRI), ultrasound imaging and computed tomography (CT) [28]. Molecular imaging has the potential to provide unique information that will guarantee the safety and efficacy of biotherapies which utilize antibodies, bacteria or cells in humans, and also will contribute to the future development of novel biotherapies [15].

With the emergence of cell therapies in regenerative medicine, it is important to track cells injected into subjects. In this context, NIS has been used in preclinical studies. With transfer of the NIS gene into therapeutic cells such as cytotoxic T or natural killer cells, nuclear molecular imaging modalities can image the cells with a relevant radiotracer, such as I-123, I-124, I-131, Tc-99m or F-18 TFB. The NIS-expressing cells have been imaged with planar scintigraphy, SPECT or PET according to the administered radiotracers [15].

Nuclear imaging modalities, such as PET and SPECT, provide the 3-dimensional distribution of radiopharmaceuticals and have excellent sensitivity and high resolution with excellent tissue penetration depth [29]. These advantages permit these imaging techniques for use in translational research, from cell culture to preclinical animal models to clinical applications [28]. Both PET and SPECT give quantitative and non-invasive information on NIS gene expression or the number of NIS-expressing cells [15, 28].

As a gene reporter, NIS is able to be used for monitoring of gene and vector biodistribution and for trafficking of therapeutic cells [6, 15]. Contrary to the diagnostic application of radioiodine nuclear imaging using NIS gene expression for the detection of thyroid cancer recurrence or metastases, NIS gene transfer is a prerequisite for radionuclide-based molecular imaging (Fig. 3). Non-invasive imaging of NIS expressing nonthyroidal cells with a gamma camera or PET upon viral gene transfer has been demonstrated feasible and safe in experimental animals and humans as well (Fig. 4) [6, 8].

Cells without NIS gene expression obtain the function of iodine uptake with NIS gene transduction by viral or non-viral vector delivery. The cells can be imaged by radionuclide-based molecular imaging techniques using gamma ray or positron-emitting radiotracers and be cleared by beta or alpha particle-emitting radionuclides.

Visualization of macrophages expressing NIS with radionuclide-based molecular imaging. Inflammation at the right thigh (yellow arrow) was well visualized in F-18 FDG microPET imaging. Migration of microphages expressing NIS to the inflammation site (white arrow) was clearly visualized on I-124 microPET imaging [7].

Visualization of tumor cells expressing NIS with optical molecular imaging using I-124. Tumor xenografts of anaplastic thyroid cancer cells expressing NIS were well visualized on both microPET imaging (white arrows) and Cerenkov luminescence imaging (black arrows) after intravenous administration of I-124 [17].

Recently, I-131 and I-124, which are commonly used for thyroid imaging, were reported to have sufficient energy to result in Cerenkov radiation that can be visualized with sensitive optical imaging equipment and cells transfected with NIS gene were successfully imaged with the radioiodines using an optical imaging instrument in an in vivo animal model (Fig. 5) [17].

Radioiodine accumulation in NIS-expressing organs such as the thyroid is a deterrent to scintigraphic visualization of NIS-expressing cells in various animal models. To remove radioiodine uptake in the thyroid gland and better visualize NIS-expressing cells, the animal can be prepared with surgical total thyroidectomy or radioiodine ablation before administration of the NIS-expressing cells [30].

Molecular radionuclide-based therapy of differentiated NIS-expressing thyroid cancer with I-131 was the cornerstone on which nuclear medicine was built and it has been a very successful example of targeted therapy to reduce recurrence and mortality for almost 70 years (Fig. 6) [31, 32]. Therapeutic application of I-131 for hyperthyroidism and thyroid cancer was implemented in the early 1940s, and success of the applications resulted in the approval of medical radioisotope use and initiation of atomic medicine, later re-named nuclear medicine [31, 33].

Radioiodine therapy for thyroid diseases relies on the fact that thyroid follicular cells and differentiated thyroid cancer are efficient at trapping circulating radioiodine than other tissues [25]. I-131 treatment has been the most preferred therapeutic modality by physicians for hyperthyroidism in the United States and it has been one of the key treatment modalities for differentiated thyroid cancers worldwide [34, 35]. However, I-131 treatment is not very effective in de-differentiated thyroid cancer, which down-regulates NIS expression, and is meaningless in anaplastic thyroid and medullary thyroid cancers, which do not express NIS. One possible treatment option for de-differentiated thyroid cancer is the induction of re-differentiation with differentiating agents such as retinoic acid and thiazolidinedione [31, 36].

Expression of NIS is not uncommon in breast and stomach cancers, and some reports have shown visualization of primary or metastatic lesions of such cancers with radioiodine or Tc-99m scintigraphy [37-40]. The possibility of radioiodine treatment for cancers with sufficient NIS expression has been suggested; however, as far as the author knows, clinical reports on such treatment with successful outcome have yet to be published, likely due to insufficient NIS expression [16].

Although it has not been clinically attempted, anaplastic or medullary thyroid cancers lacking NIS expression can be treated with I-131 after NIS gene transfer to the tumors. Additionally, other tumor entities which do not express NIS can also be treated with the same strategy [31].

A 26-year-old female who underwent total thyroidectomy due to papillary thyroid cancer. (A) Chest simple radiograph did not demonstrate any observable metastatic lesions of the cancer. (B) CT scan of the chest demonstrated several metastatic lesions of the cancer in both lung fields (white arrows). TSH-stimulated serum thyroglobulin was 65.0 ng/mL. The patient was diagnosed with metastatic thyroid cancer of the lung. (C) A post initial high dose I-131 treatment (150 mCi) scan revealed numerous metastatic lung lesions. (D) A post 2nd high dose I-131 treatment (200 mCi) scan revealed fewer but still several metastatic lung lesions (black arrows). (E, F) A post 3rd high dose I-131 treatment (200 mCi) scan revealed no remarkable radioiodine uptake in both lung fields and chest CT showed only tiny lung nodules having no clinical significance. TSH-stimulated serum thyroglobulin was 1.4 ng/mL after the third treatment. The patient had achieved complete remission with three times of high dose I-131 treatment and her status still remains disease-free at 7 years follow-up.

In addition to its imaging potential, NIS can be used as a therapeutic gene through its ability to concentrate therapeutic doses of radionuclides in target cells [15]. Contrary to the therapeutic application of I-131 using NIS gene expression for treating thyroid cancer recurrence or metastases, NIS gene transfer is a prerequisite for tumors without NIS gene expression. After the transfer of the NIS gene into various cancer cells, they can be treated with beta or alpha particle-emitting radionuclides including I-131, Re-186, Re-188 and At-211, which are accumulated via NIS (Fig. 3) [31].

Right after NIS was cloned by Carrasco et al. in 1996, many researchers started to use the gene for therapeutic purposes with I-131, and in general, the results were effective. The effect of I-131 NIS gene therapy was enhanced with higher doses of I-131 and intervention with retinoic acid or dexamethasone, which increase radioiodine uptake [41]. Transcription factors such as Pax-8 and TTF-1 could induce or promote iodide uptake and specifically prolong iodide retention time in cancer cells [42, 43].

Re-188 and At-211 were also used as therapeutic radionuclides with NIS gene therapy to nonthyroidal tumors. Re-188 has advantages over I-131, as its beta ray energy is higher and has a shorter half-life, which makes it a more suitable radionuclide for NIS-expressing tumors. In addition, it is conveniently obtained from a W-188/Re-188 generator [44]. At-211, which emits extremely cytotoxic alpha-particles, is known to be taken up by NIS in thyroid tissue and has been used as a therapeutic radionuclide for NIS-expressing tumors in cell culture and animal experiments [21, 45, 46]. In addition to very effective tumoricidal effects, At-211 has the advantages of alpha-particle's short range and a short half-life, which allow for a minimal radiation burden to the surrounding environment, including people [46].

However, single radionuclide NIS gene therapy might have limited therapeutic effects and can produce serious adverse effects positively related to the amount of administered radionuclide dose. Reducing the radionuclide dose for NIS gene therapy is able to reduce the adverse effects, but might lead to limited effectiveness [47]. Combined treatment of radionuclide NIS gene therapy with other therapeutic approaches could be more efficient to improve therapeutic outcomes and can reduce adverse effects of radionuclide NIS gene therapy by reducing the radionuclide dose. Chemotherapy, genciclovir HSV-tk gene therapy, immunotherapy, external beam radiotherapy and siRNA therapy have been combined with radionuclide NIS gene therapy, and the results were almost always successful [8-10, 47].

Even though radionuclide NIS gene therapy has been shown to be effective in in vivo animal models, several issues must be resolved before this novel strategy can be useful clinically. First of all, vector systems having safe, effective and specific NIS gene delivery to the tumor are needed. The optimal time interval between NIS gene transfer and therapeutic radionuclide administration should be determined to obtain the most effective therapeutic results. Organs that normally express NIS, such as the thyroid gland and the salivary glands, are inevitably damaged by the therapeutic radionuclide; therefore, protecting or managing strategies for the organs need to be developed [13, 31, 48].

Even though radionuclide NIS gene therapy is only performed in the preclinical setting at the moment, clinical trials of the treatment are likely to happen in the not-too-distant future with advances in efficiency and safety of the therapy by close communication between these basic biological studies and clinical experiences of thyroid cancer treatment with I-131.

NIS provides an advantage of both as reporter and therapeutic genes and therefore, NIS gene transfer makes it possible to image, monitor and treat the tumor with appropriate radionuclides, just as in differentiated thyroid cancer. Another advantage of NIS is wide availability of appropriate diagnostic and therapeutic radiopharmaceuticals. Although NIS is one of the best theranostic genes, there are several pending questions that must be answered before its clinical use.

Tissues that normally express endogenous NIS such as the thyroid gland, salivary glands and stomach, are an obstacle for NIS-based imaging or treatment. Uptake of imaging radiotracers to the tissues conceals trafficking target cells expressing NIS which are located near the tissues. Uptake of therapeutic radionuclides to the normal tissues can damage the organs and may reduce tracer uptake to the target cells expressing exogenous NIS.

Retention time of radioiodine is generally short in NIS-transduced cells by rapid washout of the radioiodine, therefore absorbed dose and toxicity to the target cells might be limited and it precludes successful radioiodine NIS gene therapy. To prolong the retention time, drugs such as lithium carbonate, or co-transfer of the thyroid peroxidase gene was introduced, however, results were conflicting and not very effective [13]. Co-transfer of the thyroglobulin gene was also suggested to increase retention time [42]. Efflux of iodine from the cell is known to be related to pendrin, SLC5A8 and ClCn5, and even though not verified by experiments, down-regulation of these proteins can delay iodine efflux from the cell [42]. Ablation of the thyroid gland and low iodine diet are able to prolong the retention time in NIS transduced tumor cells, however applicability of this strategy is limited in a clinical situation. It can be feasibly applied only in thyroid cancer patients receiving previous thyroidectomy. Enhancement of radioiodine uptake by up-regulation of NIS expression has been tried with drugs such as retinoic acid or dexamethasone, troglitazone and external radiation [49, 50]. Histone deacetylase inhibitors (e.g. depsipeptide, trichosatin A and valproic acid) and demethylating agents (5-azacytidine) have been used to restore endogenous NIS expression [1]. In addition to increasing radiation dose to the NIS expressing cells, radiosensitization can enhance the biological effect of the same radiation dose. DNA damage repair inhibitors revealed a therapeutic benefit with radionuclide NIS gene therapy [51]. Further studies are needed for validation and optimization of the pharmacological approaches for prolonging the retention time, delaying iodine efflux, restoring/up-regulation of NIS expression and enhancing radiosensitization before practical use.

Several new diagnostic or therapeutic radiopharmaceuticals for NIS were recently studied. Cells expressing NIS can be imaged with F-18 FTB PET instead of radioiodine scintigraphy and be treated more effectively with Re-188, Re-186 or At-211 instead of I-131. Some of the radiopharmaceuticals are not suitable at present due to scarce availability and nontrivial safety issues related to their production and handling. Technical advancement of the production and handling skills for the radiopharmaceuticals is warranted.

With administration of I-131, the thyroid gland takes up I-131 and retains it within the gland for a long time by organification of the radioiodine. This will end in permanent hypothyroidism by radioablation of normal thyroid tissue. The salivary gland also accumulates the radionuclide and xerostomia can occur by radiation sialoadenitis related to uptake of the radionuclide. To maintain sufficient radioiodine uptake to the extrathyroidal cancer tissues expressing NIS, uptake of radioiodine to the thyroid gland can be suppressed by thyroid hormone replacement and antithyroidal drugs [52]. Stable iodine administration before administration of radioiodine can reduce radioiodine to the gland as well [13]. Radioiodine uptake in the salivary gland can be expelled by manual massage of the gland and may reduce incidence of xerostomia related to radiation-induced sialoadenitis [53]. Strategies for preventing or reducing side effects to normal tissues expressing NIS by radionuclides uptake must be developed and optimized before common clinical application of NIS-based radionuclide theranostics.

Although diagnostic and therapeutic use of the NIS gene began in clinics more than half a century ago, understanding of the biology of NIS has been advancing rapidly the last two decades. NIS-based molecular imaging and radionuclide gene therapy, cutting edge technologies in molecular imaging and gene therapy arenas, were born with imitation of diagnostic and therapeutic applications in the field of clinical thyroid practice. With fast advancement of molecular imaging and gene therapy with active research, these bench technologies are likely to be used in the clinical setting in the near future.

This study was supported by a grant (A102132) of the Korea Health Technology R&D Project, Ministry of Health & Welfare, Republic of Korea and the Ministry of Knowledge Economy (MKE), and a grant of the Korea Institute for Advancement of Technology (KIAT) and Daegyeong Leading Industry Office through the Leading Industry Development for Economic Region.

The authors have declared that no competing interest exists.

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Biological bypass shows promise in coronary artery disease – Medical Xpress

Gene Therapy Clinics | Posted by admin
Aug 12 2017

August 8, 2017

A new gene therapy that targets the heart and requires only one treatment session has been found safe for patients with coronary artery disease, according to a successful trial carried out in Finland. Enhancing circulation in the oxygen-deficient heart muscle, the effects were visible even one year after the treatment.

The randomised, blinded, placebo-controlled phase 1/2a trial was carried out in collaboration between the University of Eastern Finland, Kuopio University Hospital and Turku PET Centre as part of the Centre of Excellence in Cardiovascular and Metabolic Diseases of the Academy of Finland.

The biological bypass is based on gene transfer in which a natural human growth factor is injected into the heart muscle to enhance vascular growth. The trial was the first in the world to use a novel vascular growth factor that has several beneficial effects on circulation in the heart muscle. The trial also developed a novel and precise method for injecting the gene into the oxygen-deficient heart muscle area. A customised catheter is inserted via the patient's groin vessels to the left ventricle, after which the gene solution can be injected directly into the heart muscle. The method is as easy to perform as coronary balloon angioplasty, which means that it is also suitable for older patients and patients who are beyond a bypass surgery or other demanding surgical or arterial operations.

The biological bypass constitutes a significant step forward in the development of novel biological treatments for patients with severe coronary artery disease. A new blood test biomarker was also discovered, making it possible to identify patients who are most likely to benefit from the new treatment.

The biological bypass was developed by Academy Professor Seppo Yl-Herttuala's research group at the A.I. Virtanen Institute for Molecular Sciences of the University of Eastern Finland. At the Kuopio University Hospital Heart Centre, Professor Juha Hartikainen was responsible for the trial.

Securing six million euros of funding from the European Union, research into the biological bypass will continue, and a new phase 2b trial will start at Kuopio University Hospital in early 2018. This trial will also include five other cardiology clinics from Denmark, the UK, Austria, Spain and Poland. The multi-centre trial will be coordinated by the Kuopio University Hospital Heart Centre, and the gene therapy drug will be manufactured in the clean room facilities of FinVector Therapies Ltd. in Kuopio.

Explore further: Novel mapping technique targets gene therapy to hibernating heart muscle

More information: Juha Hartikainen et al. Adenoviral intramyocardial VEGF-DNC gene transfer increases myocardial perfusion reserve in refractory angina patients: a phase I/IIa study with 1-year follow-up, European Heart Journal (2017). DOI: 10.1093/eurheartj/ehx352

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Biological bypass shows promise in coronary artery disease - Medical Xpress

Cardiovascular disease cure? One session of THIS could help treat condition –

Gene Therapy Clinics | Posted by admin
Aug 09 2017

Coronary heart disease is the term that describes what happens when the heart's blood supply is blocked or interrupted by a build-up of fatty substances in the coronary arteries.

This is a process called atherosclerosis.

Coronary heart disease can't be cured yet but treatment can help manage the symptoms and reduce the chances of problems such as heart attacks.

However, now experts have found a new gene therapy which targets the heart and requires only one treatment session.


The treatment has been found safe for patients with coronary artery disease, according to a successful trial carried out in Finland.

It works by enhancing circulation in the oxygen-deficient heart muscle and experts said the effects were visible even one year after the treatment.

A trial was carried out in collaboration between the University of Eastern Finland, Kuopio University Hospital and Turku PET Centre as part of the Centre of Excellence in Cardiovascular and Metabolic Diseases of the Academy of Finland.

The biological bypass is based on gene transfer in which a natural human growth hormones - called a factor - is injected into the heart muscle to enhance vascular growth.



1 of 11

10 Step plan to eliminate your risk of heart disease

Cardiovascular disease could be treated with gene therapy

The trial was the first in the world to use a vascular growth factor which has several beneficial effects on circulation in the heart muscle.

Experts also developed a precise method for injecting the gene into the oxygen-deficient heart muscle area.

A customised catheter is inserted via the patients groin vessels to the left ventricle, after which the gene solution can be injected directly into the heart muscle.

The method is as easy to perform as coronary balloon angioplasty, which means that it is also suitable for older patients and patients who are beyond a bypass surgery or other demanding surgical or arterial operations.


Experts said the biological bypass constitutes a significant step forward in the development of novel biological treatments for patients with severe coronary artery disease.

A new blood test biomarker was also discovered, making it possible to identify patients who are most likely to benefit from the new treatment.

The biological bypass was developed by a research group at the University of Eastern Finland.

Experts said research into the biological bypass will continue with a new trial set to start in 2018.

This trial will also include five other cardiology clinics from Denmark, the UK, Austria, Spain and Poland.

This comes after it was revealed heart disease risk could be determined by your waist size.

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Cardiovascular disease cure? One session of THIS could help treat condition -

Understanding the muscle behind global duchenne muscular dystrophy market – WhaTech

Gene Therapy Clinics | Posted by admin
Aug 09 2017

Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by muscle degeneration and weakness. Duchenne muscular dystrophy (DMD) cause due to lack of protein known as dystrophin which causes muscles deterioration and break down, leads to difficulty in walking and general mobility.

DMD is a one of the most progressive childhood neuromuscular disorders. It affects mostly boys, but occasionally girls are affected.

DMD can be caused due to cardiac, neuromuscular, and orthopedic disorders.

Increasing research and development, introduction of novel disease therapies, rising demand for effective therapies among patients, and increasing disease prevalence are projected to fuel the growth of the global Duchenne muscular dystrophy market. According to the Centers for Disease Control and Prevention, in 2016, prevalence of Duchenne and Becker muscular dystrophy (DBMD) was 1 in every 7,250 males aged 5 to 24 years.

Rising prevalence of chronic diseases such as cardiovascular, neurovascular, and arthritis, and increasing health care insurance coverage are the other factors likely to accelerate the growth of the global Duchenne muscular dystrophy market. According to the World Health Organization, cardiovascular diseases accounted for 17.7 million deaths in 2015, representing 31% of all global deaths.

However, stricter regulation for product approvals and high product cost are likely to restrain the Duchenne muscular dystrophy market.

The Duchenne muscular dystrophy (DMD) market has been segmented based on treatment type, diagnosis, end-user, and region. In terms of treatment type, the market has been classified into drug therapy and novel therapy.

The drug therapy segment has been sub-segmented into corticosteroids and others. The novel therapy segment has been categorized into gene therapy, stem cell therapy, utrophin, and others.

In terms of diagnosis, the Duchenne muscular dystrophy market has been classified into blood tests, gene tests, and muscle biopsy. Based on end-user, the market has been classified into hospitals, specialty clinics, and ambulatory surgery centers.

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Geographically, the Duchenne muscular dystrophy market has been segmented into North America, Latin America, Europe, Asia Pacific, and Middle East & Africa. North America dominates the global Duchenne muscular dystrophy market due to new product innovation, high health care expenditure, and government awareness programs.

The United Parent Projects Muscular Dystrophy initiated World Duchenne Awareness Day. The aim of Duchenne Awareness Day is to raise awareness about Duchenne muscular dystrophy across the globe and September 7 has been declared as Duchenne Awareness Day.

Europe is the second largest market for Duchenne muscular dystrophy. The market in Asia Pacific is expected to grow at higher rate due to rapid rise in population, growing prevalence of chronic diseases, increasing health care coverage, and rising investment in research and development.

Emerging regions such as Latin America and Middle East & Africa will create a large opportunity in the global Duchenne muscular dystrophy market due to growing awareness among people, increasing public and private health care insurance coverage, etc.

Major players operating in the global Duchenne muscular dystrophy market include Pfizer, Inc., Eli Lilly and Company, Nobelpharma Co., Ltd., Sarepta Therapeutics, Inc., Tivorsan Pharmaceuticals, Acceleron Pharma, Inc., BioMarin Pharmaceutical, Inc., Asklepios Kliniken GmbH, FibroGen, Inc., and Santhera Pharmaceuticals Holding.

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 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)

Request a brochure of this report to know what opportunities will emerge in the rapidly evolving Duchenne Muscular Dystrophy (DMD) Market during 2017- 2025

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Understanding the muscle behind global duchenne muscular dystrophy market - WhaTech

Proton Therapy: How It Could Change The Outcome of Paediatric Cancer – HuffPost

Gene Therapy Clinics | Posted by admin
Aug 05 2017

Today, cancer is no longer on the list of incurable diseases, thanks to medical developments in the last few decades. Yet, it is hard to ignore the fact that cancer does alter the lives of patients. We know of many adults that have undergone not just physical but an emotional overhaul during their cancer journey - some of them returning to life with an altered perspective and unending empathy for people around them after having been through the trials and trauma of chemotherapy and radiation.

For children, it might be a totally different story. For many of them, the effects of cancer treatment do not begin until later in life resulting in other health concerns. In fact, studying these late effects is the research focus of many medical researchers in the field of pediatric cancer. Many of these effects are the result of how chemotherapy works - in order to kills the cancerous cells, it destroys a good number of healthy cells as collateral damage.

I recently spoke with Dr. Ramesh Rengan, medical director of Seattle Cancer Care Alliance (SCCA) Proton Therapy Center in Seattle, Washington, and a long-term researcher in the field of novel radiation approaches with immune therapies in the treatment of cancer. Dr. Rengan is an active voice for proton therapy emerging as an increasingly effective and less harmful way to treat specific types of cancers, particularly pediatric cancers and those that occur close to vital organs.

Here are a few excerpts from our interview:

The SCCA Proton center works on an alternative treatment method for cancer. Can you tell us more about it?

At the SCCA Proton Therapy Center, we use highly targeted proton beam radiation to treat cancer. By precisely focusing on the tumor, proton therapy minimizes radiation exposure to surrounding healthy tissue, thereby reducing the risk of short- and long-term radiation-related side effects. Our center is currently the only proton therapy center in the Pacific Northwest.

How does proton therapy work?

The fundamental concept underlying all cancer treatment is to deliver a lethally effective treatment to the cancer and only the cancer, be it chemotherapy, surgery or radiation. The challenge for the oncologist is to design a way to deliver this treatment with specificity to the tumor while minimizing the damage to the surrounding organs. What really dictates the long-term clinical outcome for the patient is not just the number of cancer cells that we kill but the balance between that and the number of healthy cells that are injured in the course of treatment.

Standard radiation uses X-ray beams that enter and pass through the patient to the tumor and exit the other side. However, because X-rays penetrate so well, all tissue that lies in front of and beyond the tumor is exposed to radiation, which can be potentially harmful to the patient.

Protons, due to their mass, stop within the patient and do not continue to pass through the body. We can therefore calibrate the proton beam to stop within the tumor itself. Additionally, due to their positive charge, protons deposit most of their radiation at the point where they stop, rather than near the point of entry into the patient as X-rays do. As a result of these properties, protons concentrate the radiation dose delivery to the tumor itself. This more targeted form of radiation is especially useful for pediatric cancers, where any excess radiation exposure to healthy, developing organs can be potentially harmful.

What are the benefits of proton therapy over other types of cancer treatments?

Protons allow radiation to be delivered to the tumor while significantly minimizing collateral radiation exposure to surrounding healthy tissue. That benefit pays dividends in the near term because it reduces exposure, and it drives additional future benefits as patients continue with their lives after treatment, such as fewer secondary cancers. The precision that protons allow is particularly beneficial for patients whose tumors are near critical organs or structures, such as the brain, bladder, rectum, heart or spinal cord; patients whose cancers have recurred after initial radiation treatment; and patients whose organs are particularly sensitive to radiation exposure, such as children and adults with certain genetic syndromes.

For what kind of cancers is the proton therapy most effective ?

Although protons can be used to treat most cancers that require radiation, this treatment has been best established for tumors that lie in close proximity to radiosensitive vital organs, such as central nervous system tumors and eye tumors. For pediatric patients, protons have become an indispensable tool, as children are particularly vulnerable to the negative effects of radiation. Therefore, proton treatment for many pediatric tumors is a well-established standard of care, despite the limited access to proton therapy centers worldwide. Today, we also treat tumors in the head and neck, lung, breast, esophagus and gastrointestinal system, as these tumors are often situated next to radiosensitive vital organs. As such, the role of protons is being actively investigated in ongoing clinical trials in these disease locations.

Are protons a stand-alone treatment, or is it best used in conjunction with other therapies?

Proton therapy can often be used in combination with chemotherapy and surgery, similar to standard radiation treatment.

How is proton therapy improving?

Access to clinics, lowered cost, optimized technology to treat a broader range of tumors and use of a combination of approaches to treatment are the biggest recent leaps we have made with proton treatment.

Its important to understand that protons initially were postulated in the treatment of cancer in a paper published in 1946. Protons are not new as a modality, having been around since the 1950s. It has taken a long time for the technology to become relatively cost-efficient and be feasibly delivered in a hospital-based or stand-alone outpatient clinical setting such as the Seattle Cancer Care Alliance Proton Therapy Center.

Growth has been deliberate and incremental: the first hospital-based proton center opened in the 90s. Today there are 20 proton centers in North America, but the real promise of proton treatment is being realized now. During the next 10 years, we expect to see that number triple or quadruple, as the cost of building a center has been rapidly decreasing while proton beam technology has been simultaneously improving.

Are protons the best choice for everyone?

Protons are just as effective as X-rays in killing cancer cells but generally require less exposure to surrounding healthy tissues in order to deliver this treatment to the cancer, so they are a potential option whenever radiation is called for in the curative treatment of cancer. However, protons are only a single tool in the ever-increasing arsenal against cancer. As such, protons are the best treatment for some patients, but not for others. The decision regarding whether protons are the right choice for a given patient is made by a proton-experienced radiation oncologist in conjunction with members of the multidisciplinary cancer care team and the patient.

What do you believe are the next big revolutions in cancer treatments?

The big revolution in cancer treatment will come from our increasing ability to deliver effective therapies to the tumor with absolute specificity. For example, that is the founding principle behind cancer immunotherapy, namely, to turn our immune system against the cancer. Similarly, the greater our ability to focus our radiation beam or our scalpels at purely the cancer and minimize the impact to the surrounding organs, the greater the benefit to our patients.

In the past few years we have made fundamental paradigm shifts in the way we treat cancer. Today we attack cancer by exploiting the unique genetic characteristics of the tumor to design drugs that attack the cancer by homing in on their gene signature. Even with these modern advancements, it must be mentioned that cancer is not just treated with one method alone; a combination of weapons is used to effectively cure most cancers. One might say that it takes a village to effectively treat a cancer patient.

Much of the early history of cancer treatment had focused on maximizing the lethality of our weapons against the cancer, either by increasing the radiation or chemotherapy dose or by performing larger and more radical surgery to remove the tumor. We now recognize that the impact of these treatments on healthy tissue can have an even greater negative effect on the clinical outcome than the positive benefit of killing the tumor. In short, we dont need a bigger gun or bomb, but rather a smarter bomb that equally emphasizes supporting the health of the patient and the destruction of the cancer. Protons are ideally suited to this evolution in cancer care.

Devishobhais the founder ofKidskintha,a happy place to jumpstart conversations around family and millennial parenting, living in India. You can find her voiceon the Huffington Post,LifeHack,,Addicted2Success,, Entrepreneur, Tiny Buddha,SivanaEast and others on a range of topics. You can get yourself equipped for happy parenting with one hack a week for an entire year (each one backed by science).

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Proton Therapy: How It Could Change The Outcome of Paediatric Cancer - HuffPost

Wilson’s Disease Market: Unmet Needs of Patient Population to Inspire Players for Improved Treatment Options – Edition Truth

Gene Therapy Clinics | Posted by admin
Aug 05 2017

The global Wilsons disease market is driven by the development of gene therapy as a potential cure for this disease. As per the European Association, approximately 6 to 12 percent of the total patient population suffering from liver failure is affected by Wilsons disease, and this in turn will drive a demand for their treatment. It has also been found that females with liver failure are more prone to Wilsons disease. In addition to this, the risk of Wilsons disease is high among patients with low hemoglobin, jaundice, and low cholinesterase.

As patients suffering from Wilsons Disease are unable to discharge copper at a normal rate from their liver on account of the mutation of the ATP7B gene, its treatment becomes essential. One of the key challenges of this market is the under-diagnosis of this disease. A lack of safety in the treatment offered is also another factor challenging the market. The current treatments show poor compliance and this is posing another challenge. However, the unmet need for efficient treatment for Wilsons disease can be viewed by market players as a scope for vigorous improvement and newer discoveries can be made by doubling their efforts in research and development.

The report segments the global Wilsons disease market on the basis of treatment, end-user, geography, and indication. By treatment, the market is segmented into penicillamine, zinc, and trientine. Of these, penicillamine continues to enjoy highest popularity as treatment option. On the basis of indication, the global Wilsons disease market can be segmented into psychiatric, hepatic, ophthalmic, and neurological. On the basis of end-user, the market can be segmented into specialty clinics, diagnostic laboratories, and hospitals.

On the basis of geography, the market is segmented into Asia Pacific, Europe, Latin America, North America, and the Middle East and Africa. Of these, North America is leading in the Wilsons disease market on account of growing patient awareness and increasing demand for various treatment options. Europe is anticipated to trail next. The increasing population and growing demand for quick diagnosis will boost the market in the region. The demand for improved and efficient treatment will also help the market players to strive for better discoveries and developments. The growing medical tourism in developing nations of Asia Pacific will also help the market to grow in the region.

Some of the leading players operating in the global Wilsons disease market are Meda Pharmaceuticals, Inc., Valeant Pharmaceuticals International, Inc., Teva Pharmaceuticals, Ipsen, Taj Pharmaceutical Limited, and Wilson Therapeutics.

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Wilson's Disease Market: Unmet Needs of Patient Population to Inspire Players for Improved Treatment Options - Edition Truth