The founding mission of MIT may seem like an unusual meal-time story for a child. But when Mark Bathe was growing up, it was a regular topic of conversation around the dinner table.

That is because Bathes father, mechanical engineer Klaus-Jrgen Bathe, was a long-standing, proud MIT faculty member, and regularly talked about MIT founder William Barton Rogers mission for the Institute.

Bathes father was a huge presence in his childhood, and his enthusiastic descriptions of MITs focus on fundamental yet hands-on science to benefit society made quite an impression on him. My father was the lens through which I saw the world, Bathe says.

So when Bathe was admitted to both MIT and another university as a senior in high school, there was little doubt in his mind as to where he would be enrolling.

Bathe joined MITs Department of Mechanical Engineering as an undergraduate, where he considers himself fortunate to have been trained in a broad and fundamental, yet problem-oriented, manner.

But with a longstanding desire to impact human health through medicine, Bathe moved on to graduate research in biomechanical engineering, in part under the stewardship of Alan Grodzinsky, a professor of biological, mechanical, and electrical engineering, and director of the MIT Center for Biomedical Engineering.

After receiving his PhD in 2004, Bathe decided to deepen his understanding of biomolecules by moving to the University of Munich in 2006, to carry out postdoctoral research in biological physics.

He then returned to MIT in 2009, joining the Department of Biological Engineering, where he established an interdisciplinary research group focused on using approaches from engineering, chemistry, physics, and computer science to understand and solve problems in applied biology.

I find the new emerging world of personalized medicine fascinating, Bathe says. In particular, the prospect of using gene-editing tools to correct disease-causing mutations that are either inherited or acquired, as well as the use of messenger RNAs to express specific proteins that are needed to alleviate disease.

Bathe, now an associate professor of biological engineering at MIT, creates a huge variety of programmed three-dimensional shapes out of single strands of synthetic DNA, a process known as DNA origami. These nanoparticles may ultimately be deployed as structural scaffolds to deliver vaccines, drugs, or even gene-editing tools such as CRISPR-Cas9 to specific parts of the body, he says.

Once delivered, the therapeutic payload could be released to edit the faulty genes that cause certain diseases.

It amazes me that with two therapeutic tools, namely CRISPR for gene editing and therapeutic messenger RNAs for protein production, we could, in principle, cure nearly any disease, potentially with minimal side-effects, but only if we can figure out how to successfully deliver these tools to act highly specifically in the target cells of interest, such as the gut, lungs, brain, or other organs, he says.

Tackling this problem can only be achieved through an interdisciplinary, long-term research effort, he believes.

Targeted therapeutic delivery is a highly interdisciplinary problem, involving everything from very applied, clinical medicine to basic macromolecular chemistry of nucleic acids and proteins, as well as the physics and engineering of macromolecular transport, Bathe says.

As a starting point, his laboratory, which includes engineers, chemists, computer scientists, and physicists, developed DAEDALUS (DNA Origami Sequence Design Algorithm for User-defined Structures), an algorithm designed to automate the process of assembling DNA nanoparticles. DAEDALUS, which takes a simple 3-D representation of the object and determines how this should be assembled from the DNA strands, can build any type of enclosed 3-D shape.

As a result, the algorithm, combined with new nucleic acid synthesis procedures, which were published in a paper in the journal Science last year, are allowing Bathe and his team to build the nanoparticles far more quickly and easily than was previously possible.

Despite decades of research into the delivery of nucleic acids and proteins, and the considerable potential for these therapeutics in clinical medicine, little progress has been made as measured by FDA-approved therapies, says Bathe. This is likely due in part to our poor understanding of macromolecular transport in the complex human anatomy, but also due to the lack of techniques available to engineer delivery tools, he says.

Were hopeful that fully synthetic, viral-like nucleic acid nanoparticles developed in our lab offer a new opportunity for the rational engineering of delivery tools for gene-centric therapies, he explains.

Working with with the Stanley Center for Psychiatric Research, Bathe and his team are also investigating novel methods of imaging patient-derived neuronal cells, in a bid to better understand how genes affect the signals sent between individual neurons in the brain.

He is also investigating the use of DNA and other molecules to store and process information, with density that is orders of magnitude higher than conventional silicon-based computing hardware.

When not in the classroom or his laboratory, Bathe takes part in a range of outdoor activities, including cycling, running, skiing, and hiking, as well as indoor swimming with MITs Masters Swim Team. He also greatly enjoys an occasional sprint triathlon on summer weekends.

My favorite weekend in the Boston area, however, is a ferry ride down to Marthas Vineyard for a bike ride around the island, ending with a swim and lobster roll by the seaside in Edgartown, he says. I cant recommend it highly enough!

Read this article:
Deploying therapeutic payloads to cells - MIT News

February 28, 2017 Credit: Cardiff University

Researchers at Cardiff University have discovered that genetic variation is the reason why some immune systems overreact to viruses.

Previous research had already revealed that a gene called Ifitm3 influences how sensitive people are to the influenza virus, with a variant form of the gene making cells more susceptible to viral infection. The new research reveals that Ifitm3 also plays an important role in controlling the extent of the inflammatory response triggered by virus infection.

The study suggests that individuals with deficiencies in Ifitm3 may have an overactive immune response to viral infection and may therefore be helped by a combination of anti-inflammatory drugs in addition to medicine that directly targets the virus.

World-wide the frequency of the variant Ifitm3 gene is 1 in 400, although it is much more common in certain ethnicities.

Dr Ian Humphreys from Cardiff University's School of Medicine said: "Now we know that genetic make-up influences how the immune system copes with infections, not only by influencing how the body controls an infection but also by controlling how strongly the body's immune system reacts, we can design therapeutic strategies for individuals who are seriously ill with infections, which are tailored to the individual based on their genetic profile."

The data were collected using immune cells from mice with and without the variant form of Ifitm3, to observe how the immune system responds to a virus called cytomegalovirus. The results could also be relevant for other viral infections such as influenza epidemics/pandemics.

Explore further: Genetics of flu susceptibility: Researchers find gene that can transform mild influenza to a life-threatening disease

More information: Maria A. Stacey et al. The antiviral restriction factor IFN-induced transmembrane protein 3 prevents cytokine-driven CMV pathogenesis, Journal of Clinical Investigation (2017). DOI: 10.1172/JCI84889

Researchers at Cardiff University have discovered that genetic variation is the reason why some immune systems overreact to viruses.

Scientists at Sanford Burnham Prebys Medical Discovery Institute (SBP) have identified a new regulator of the innate immune responsethe immediate, natural immune response to foreign invaders. The study, published recently ...

A new discovery by researchers at the Fred Hutchinson Cancer Research Center in Seattle makes an important step in identifying which specific T cells within the diverse army of a person's immune system are best suited to ...

As much as we try to avoid it, we are constantly sharing germs with those around us. But even when two people have the same infection, the resulting illnesses can be dramatically differentmild for one person, severe or ...

Scientists propose in Nature blocking a molecule that drives inflammation and organ damage in Gaucher and maybe other lysosomal storage diseases as a possible treatment with fewer risks and lower costs than current therapies.

If you've ever wondered how a vaccine given decades ago can still protect against infection, you have your plasma cells to thank. Plasma cells are long-lived B cells that reside in the bone marrow and churn out antibodies ...

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Genetic variant linked to overactive inflammatory response - Medical Xpress

1102 Bates Street, Suite 1670

Houston, Texas 77030

The GMP Facilities at the Center for Cell and Gene Therapy at Baylor College of Medicine are among the largest and most modern in the United States. Manufacturing of therapeutic biologics has existed at Baylor for more than twenty years, and is now centralized in 19,000 square feet on the 16th floor of the Feigin Center at Texas Childrens Hospital.

This facility opened in 2010 and contains 22 ISO 7 clean room suites supported by dedicated space for cell and product cold storage, flow cytometric analysis, quality control testing, data management and storage and quality assurance activities. The staff has extensive experience in GMP manufacturing of a wide variety of products and intermediates for cellular therapies and of viral and non-viral vectors, and master and working cell banks.

Manufacturing and testing supports more than 30 investigator-sponsored INDs conducted at the Texas Medical Center and institutions around the United States. Products have also been prepared under contract for researchers in Europe, Asia and Australia. The range of products that have been manufactured is shown under the Vector and Cell Therapy Facilitypages.

The facility is also accredited by the Foundation for the Accreditation of Cellular Therapy (FACT)and is CLIA registered for high complexity testing.

Center for Cell & Gene Therapy - cGMP Facilities

See the rest here:
Center For Cell & Gene Therapy - Cell Therapy

An orthopedist orders special testing to determine if an elder patient with right hip pain which limits walking and driving might need surgery to improve their quality of life.

Physicians are rigorously trained to make decisions in the best interest of their patients. Even after medical school and residency, doctors must follow the challenges of evidence-based medicine, standard of care, peer review, and muster the time for continuing medical education and certification.

Doctrors are not only held accountable by their peers, but also legally as they could be subject to lawsuits. Additionally, state licensing agencies overseeing medical professionals can discipline them should they not practice medicine up to the standards of quality medical decision-making.

However, what if the teens pediatrician feels hospitalization is acutely needed for mental illness, but it is denied by the insurance company? What if the Workers Comp physician orders an MRI for the powerline workers ailing right arm, but it is denied? Or, if special testing to evaluate grandmas worsening mobility and pain is turned down by the HMO? Who is held accountable?

To justify requests for specific patient care, physicians are forced to have Peer-to-Peer phone discussions with doctors employed by insurance companies, Workers Comp, and HMOs. Frequently, these conversations result in denial of further care without medical justification. A controversial question arises: Are denials by these company doctors considered a medical decision?

They are not. These decisions are considered utilization review. What does this mean? They are making decisions based on controlling costs, which is in the financial interest of the for-profit agencies they serve, but not necessarily in the best interest of the patient. Even though they are licensed doctors practicing medicine, their role in patient care is under the guise of utilization review, and therefore not under the scrutiny of state licensing agencies.

What if these physicians deny care because they are incentivized to enhance personal bonuses? More so, what if some are making decisions outside the realm of their medical expertise (e.g. a urologist on a diabetic)? Who holds these physicians accountable for moral transgressions, or lack of judgement?

In California, we have a Medical Board which oversees licensing for all state physicians. If you report a licensed physician for making substandard medical decisions, an investigation ensues. If though the doctor is employed by an insurance company, Workers Comp, or HMO and makes denial decisions on their behalf, it is considered utilization review and they are not held accountable.

I do not pretend to understand every law and rule governing the Medical Board. But these companies have created legal barriers protecting doctors who might make substandard medical decisions.

Many physicians continue to fight for patient care rights despite frustration and helplessness of ongoing phone calls and paperwork they face. Yet substandard medical care will hamper their efforts as laws are manipulated and oversight is negligible.

Making medical decisions has never been easy. Assuring accountability makes it even harder.

Gene Uzawa Dorio, M.D.

Read more from the original source:
Dr. Gene Dorio: Making Medical Decisions Without Accountability - KHTS Radio

A friend asked me about population structure, and methods to ferret it out and classify it. So here is a quick survey on the major methods Im familiar with/utilize now and then. Ill go roughly in chronological order.

First, you have trees. These are pretty popular from macroevolutionary relationships, but on the population genetic scale (intraspecific, microevolutionary) youre mostly talking about representing distances between groups in a tree format. You saw this in History and Geography of Genes, where genetic distances in the form of Fst values (proportion of genetic variation unique to between two groups) were used as distance inputs.

A problem with trees is that they dont model gene flow, a major dynamic on a microevolutionary scale. Also, complex relationships can get elided in tree frameworks, and as you add more and more populations you often end up with an incomprehensible fan-like topology.

Then you have principle component analyses (PCA) and related methods (e.g., multidimensional scaling, which is very different in the sausage-making but generates a similar output). Like trees, this is a visualization of the variation, in this case on a two dimensional plot (please dont bring up three dimensional PCA, theres no such thing until holograms show up).

The problem with PCA is that different types of dynamics can lead to the same result. For example, someone who is an F1 of two distinct groups occupies the same position as a population which happens to occupy a genetic position between two groups. Additionally, by constraining the variation into two dimensions, one can mislead in terms of relationships. There are many dimensions, but operationally you focus on on two at a time.

A paper of interest, Population Structure and Eigenanalysis.

Next you have model-based clustering introduced in Jonathan Pritchards Inference of Population Structure Using Multilocus Genotype Data. There are many flavors of this, but they operate under the same framework. You have a model of population dynamics, and see how the genotype data can be explained by parameters of the model. Of particular interest is assignment to one of K populations, which can be combined to explain the variation in the data.

Unlike PCA these model-based methods are rather good at identifying people who are first generation mixes, as opposed to those from stabilized groups along a cline. But, they also produce artifacts, because they are quite sensitive to the input data, and lend themselves to cherry-picking.

Earlier I said that one problem with the tree methods is that they dont model gene flow. Joe Pickrells TreeMix does so. Like the original tree methods, and unlike PCA or unsupervised model-based clustering, you specify a set of populations. Then you compare the populations in terms of their genetic distance, and fit them to a tree, but add migration parameters to that tree where the fit between the tree and the data is the most tenuous fit.

All visualizations are deformations of reality. TreeMix attempts to mitigate this somewhat by introducing another representation, that of migration.

Next we have local ancestry methods. By local ancestry, basically we mean methods which can assign ancestry to particular regions of the genome. While tree methods measure differences across pooled populations, PCA and model-based methods compare genotypes between individuals (this is a simplification, but bear with me). Local ancestry methods, like RFMix, compare regions of the genome with each other.

Related to, but not exactly the same, as local ancestry methods are haplotype based methods. In particular, Im thinking of the FineStructure and its related methods. These leverage variation across the genome in terms of haplotypes, rather than just looking at genotypes. They also tend to benefit from phasing, for obvious methods. FineStructure and its relatives tend to need more marker density than model-based methods, which require more marker density than PCA, which requires more marker density that tree based methods. These haplotype based methods allow for correction of and accounting for forces such as genetic drift, which tend to skew results in other methods.

Finally, there is the AdmixTools framework which is good for testing very explicit hypotheses. While many of the above methods, such as TreeMix and unsupervised model-based clustering, explore an almost open-ended space of structure possibilities, the methods in AdmixTools exists in large part to test narrow delimited models. This goes to the fact that many of these methods are complementary, and you should use them together to arrive at a robust result. For example, if you are assigning populations for TreeMix, you should use PCA and model-based clustering to make sure that the populations are clear and distinct, and outliers are removed.

Theres a lot I left out, but many of the other methods are just twists on the ones above.

See the original post here:
Razib Khan Blog Posts - The Unz Review

Track-1 Cell Therapy:

Cell therapyas performed by alternativemedicinepractitioners is very different from the controlled research done by conventionalstem cellmedical researchers. Alternative practitioners refer to their form of cell therapy by several other different names includingxenotransplanttherapy,glandular therapy, and fresh cell therapy. Proponents ofcell therapyclaim that it has been used successfully to rebuild damaged cartilage in joints, repair spinal cord injuries,strengthen a weakenedimmune system, treat autoimmune diseases such as AIDS, and help patients withneurological disorderssuch as Alzheimers disease,Parkinson's diseaseand epilepsy.

Related Conferences:

6th International Conference onTissue Engineering & Regenerative Medicine, Baltimore, USA, Aug 20-22, 2017; 8th World Congress and Expo onCell & Stem Cell Research,Orlando, USA, March 20-22, 2017; 15thWorld Congress on Biotechnology and Biotech Industries Meet,Rome, Italy,March 20-21,2017; 2nd International Conference onGenetic Counselling and Genomic Medicine ,Beijing, China,July 10-12, 2017; International Conference onClinical and Molecular Genetics, Las Vegas, USA, April 24-26, 2017.

Track-2 Gene therapy:

Gene therapyand cell therapy are overlapping fields of biomedical research with the goals of repairing the direct cause of genetic diseases in the DNA orcellularpopulation, respectively. The development of suitablegene therapytreatments for manygenetic diseasesand some acquired diseases has encountered many challenges and uncovered new insights into gene interactions and regulation. Further development often involves uncovering basic scientific knowledge of the affected tissues, cells, and genes, as well as redesigning vectors, formulations, and regulatory cassettes for the genes.Cell therapyis expanding its repertoire of cell types for administration.Cell therapytreatment strategies include isolation and transfer of specific stem cell populations, administration of effector cells, and induction of mature cells to becomepluripotent cells, and reprogramming of mature cells.

Related Conferences:

2nd International Conference onMolecular Biology , London, UK ,June 22-24, 2017; 3rd World Bio Summit & Expo, Abu Dhabi, UAE, June 19-21, 2017; 5th International Conference onIntegrative Biology, London, UK, June 19-21, 2017; 2nd World Congress on Human Genetics, Chicago, USA, July 24-26, 2017; 9th International Conference onGenomics and Pharmacogenomics, Chicago, USA, July 13-14, 2017.

Track-3 Cell and gene therapy products:

Articles containing or consisting ofhuman cellsor tissues that are intended for implantation,transplantation, infusion, or transfer to a human recipient.Gene therapiesare novel and complex products that can offer unique challenges in product development. Hence, ongoing communication between the FDA and stakeholders is essential to meet these challenges.Gene therapy productsare being developed around the world, the FDA is engaged in a number of international harmonization activities in this area.

Examples:Musculoskeletal tissue, skin, ocular tissue, human heart valves;vascular graft, dura mater, reproductive tissue/cells, Stem/progenitor cells,somatic cells, Cells transduced withgene therapyvectors , Combination products (e.g., cells or tissue + device)

Related Conferences:

7th International Conference onPlant Genomics, Bangkok, Thailand, July 03-05, 2017; 15th Euro Biotechnology Congress, Valencia, Spain, June 05-07, 2017; International Conference onIntegrative Medicine & Nutrition, Dubai, UAE, May11-13, 2017; 14th Asia-Pacific Biotech Congress, April 10-12, 2017; Beijing, China,15th Biotechnology Congress, Baltimore, USA, June 22-23, 2017.

Track-4 Cellular therapy:

Cellular therapy, also calledlive cell therapy, cellular suspensions, glandular therapy, fresh cell therapy, sick cell therapy,embryonic cell therapy, andorgan therapy- refers to various procedures in which processed tissue from animal embryos, foetuses or organs, is injected or taken orally. Products are obtained from specific organs or tissues said to correspond with the unhealthy organs or tissues of the recipient. Proponents claim that the recipient's body automatically transports the injected cells to thetarget organs, where they supposedly strengthen them and regenerate their structure. The organs and glands used in cell treatment include brain, pituitary,thyroid, adrenals, thymus, liver,kidney, pancreas, spleen, heart,ovary, testis, and parotid. Several different types of cell or cell extract can be given simultaneously - some practitioners routinely give up to 20 or more at once.

Related Conferences:

3rd International Conference onSynthetic Biology, Munich, Germany, July 20-21, 2017; 5th International Conference and Exhibition onCell and Gene Therapy,Madrid, Spain,Mar 2-3, 2017;International Conference onCell Signalling and Cancer Therapy,Paris, France,Aug 20-22, 2017; 7th Annual Conference on Stem Cell and Regenerative Medicine, Paris, France,Aug 04-05, 2016;3rd International Conference & Exhibition onTissue Preservation and Bio banking, Baltimore, USA,June 29-30, 2017.

Track-5 Cancer gene therapy:

Cancer therapiesare drugs or other substances that block the growth and spread ofcancerby interfering with specific molecules ("molecular targets") that are involved in the growth, progression, and spread ofcancer. Many cancer therapies have been approved by the Food and Drug Administration (FDA) to treat specific types of cancer. The development of targetedtherapiesrequires the identification of good targets that is, targets that play a key role in cancer cell growth and survival. One approach to identify potential targets is to compare the amounts of individualproteinsin cancer cells with those in normal cells.Proteinsthat are present in cancer cells but not normal cells or that are more abundant incancercells would be potential targets, especially if they are known to be involved incell growthor survival.

Related Conferences:

2nd Biotechnology World Convention,London, UK,May 25-27, 2017; International Conference on Animal and Human Cell Culture, Jackson Ville, USA, Sep 25-27, 2017; 9th International Conference onCancer Genomics, Chicago, USA, May 29-31, 2017; 6th International Conference onTissue Engineering & Regenerative Medicine, Baltimore, USA, Aug 20-22, 2017; 8th World Congress and Expo onCell & Stem Cell Research, Orlando, USA, March 20-22, 2017.

Track-6 Nano therapy:

Nano Therapymay be defined as the monitoring, repair, construction and control of human biological systems at themolecular level, using engineerednanodevicesand nanostructures. Basic nanostructured materials, engineeredenzymes, and the many products of biotechnology will be enormously useful in near-term medical applications. However, the full promise ofnanomedicineis unlikely to arrive until after the development of precisely controlled or programmable medical Nano machines andnanorobots.

Related Conferences:

15thWorld Congress on Biotechnology and Biotech Industries Meet ,Rome, Italy,March 20-21, 2017 ;2nd International Conference onGenetic Counselling and Genomic Medicine ,Beijing, China,July 10-12, 2017; International Conference onClinical and Molecular Genetics, Las Vegas, USA, April 24-26, 2017; 15th Euro Biotechnology Congress, Valencia, Spain, June 05-07, 2017; International Conference onIntegrative Medicine & Nutrition, Dubai, UAE, May11-13, 2017.

Track-7 Skin cell therapy:

Stem cellshave newly become a huge catchphrase in theskincarebiosphere. Skincare specialists are not usingembryonic stem cells; it is impossible to integrate live materials into a skincare product. Instead, scientists are creating products with specialized peptides andenzymesor plantstem cellswhich, when applied topically on the surface, help to protect the human skinstem cellsfrom damage and deterioration or stimulate the skins own stem cells. Currently, the technique is mainly used to save the lives of patients who have third degree burns over very large areas of their bodies.

Related Conferences:

5th International Conference and Exhibition onCell and Gene Therapy,Madrid,Spain,Mar 2-3, 2017;International Conference onCell Signalling and Cancer Therapy,Paris, France,Aug 20-22, 2017;2nd Biotechnology World Convention,London, UK,May 25-27, 2017; International Conference on Animal and Human Cell Culture, Jackson Ville, USA, Sep 25-27, 2017; 9th International Conference onCancer Genomics, Chicago, USA, May 29-31, 2017.

Track-8 HIV gene therapy:

Highly activeantiretroviral therapydramatically improves survival inHIV-infected patients. However, persistence of HIV in reservoirs has necessitated lifelong treatment that can be complicated bycumulative toxicities, incomplete immune restoration, and the emergence of drug-resistant escapemutants. Cell and gene therapies offer the promise of preventing progressiveHIV infectionby interfering with HIV replication in the absence of chronicantiviral therapy.

Related Conferences:

3rd International Conference onSynthetic Biology, Munich, Germany, July 20-21, 2017; International Conference onIntegrative Medicine & Nutrition, Dubai, UAE, May11-13, 2017; International Conference on Animal and Human Cell Culture, Jackson Ville, USA, Sep 25-27, 2017; International Conference onCell Signalling and Cancer Therapy,Paris, France,Aug 20-22, 2017;7th Annual Conference on Stem Cell and Regenerative Medicine,Paris,France,Aug 04-05, 2016.

Track-9 Diabetes for gene therapy:

Cell therapyapproaches for this disease are focused on developing the most efficient methods for the isolation ofpancreasbeta cells or appropriatestem cells, appropriate location forcell transplant, and improvement of their survival upon infusion. Alternatively, gene andcell therapyscientists are developing methods to reprogram some of the other cells of the pancreas to secreteinsulin. Currently ongoingclinical trialsusing these gene andcell therapystrategies hold promise for improved treatments of type I diabetes in the future. The firstgene therapyapproach to diabetes was put forward shortly after the cloning of theinsulingene. It was proposed that non-insulin producing cells could be made into insulin-producingcells using a suitable promoter and insulin gene construct, and that these substitute cells could restore insulin production in type 1 and some type 2 diabetics.

Related Conferences:

15thWorld Congress on Biotechnology and Biotech Industries Meet ,Rome, Italy,March 20-21, 2017;6th International Conference onTissue Engineering & Regenerative Medicine, Baltimore, USA, Aug 20-22, 2017; 8th World Congress and Expo onCell & Stem Cell Research, Orlando, USA, March 20-22, 2017; 14th Asia-Pacific Biotech Congress,Beijing, China,April 10-12, 2017;5th International Conference onIntegrative Biology, London, UK, June 19-21, 2017.

Track-10 Viral gene therapy:

Converting avirusinto a vector Theviral life cyclecan be divided into two temporally distinct phases: infection and replication. Forgene therapyto be successful, an appropriate amount of a therapeutic gene must be delivered into the target tissue without substantial toxicity. Eachviral vectorsystem is characterized by an inherent set of properties that affect its suitability for specific gene therapy applications. For some disorders, long-term expression from a relatively small proportion of cells would be sufficient (for example, genetic disorders), whereas otherpathologiesmight require high, but transient,gene expression. For example, gene therapies designed to interfere with a viral infectious process or inhibit the growth ofcancer cellsby reconstitution of inactivated tumour suppressor genes may require gene transfer into a large fraction of theabnormal cells.

Related Conferences:

3rd International Conference onSynthetic Biology, Munich, Germany, July 20-21, 2017;5th International Conference and Exhibition onCell and Gene Therapy,Madrid, Spain,Mar 2-3, 2017; International Conference on Animal and Human Cell Culture, Jackson Ville, USA, Sep 25-27, 2017; 9th International Conference onCancer Genomics, Chicago, USA, May 29-31, 2017; 14th Asia-Pacific Biotech Congress,Beijing, China,April 10-12, 2017.

Track-11 Stem cell therapies:

Stem cells have tremendous promise to help us understand and treat a range of diseases, injuries and other health-related conditions. Their potential is evident in the use ofblood stem cellsto treat diseases of the blood, a therapy that has saved the lives of thousands of children withleukaemia; and can be seen in the use ofstem cellsfor tissue grafts to treat diseases or injury to the bone, skin and surface of the eye. Some bone, skin andcorneal(eye) injuries and diseases can be treated bygraftingor implanting tissues, and the healing process relies on stem cells within thisimplanted tissue.

Related Conferences:

2nd World Congress on Human Genetics, Chicago, USA, July 24-26, 2017; 2nd International Conference onGenetic Counselling and Genomic Medicine ,Beijing, China,July 10-12, 2017; International Conference onClinical and Molecular Genetics, Las Vegas, USA, April 24-26, 2017; 2nd International Conference onMolecular Biology,London, UK,June 22-24, 2017; 15th Biotechnology Congress, Baltimore, USA, June 22-23, 2017.

Track-12 Stem cell preservation:

The ability to preserve the cells is critical to theirclinicalapplication. It improves patient access to therapies by increasing the genetic diversity of cells available. In addition, the ability to preserve cells improves the "manufacturability" of acell therapyproduct by permitting the cells to be stored until the patient is ready for administration of the therapy, permitting inventory control of products, and improving management of staffing atcell therapyfacilities. Finally, the ability to preservecell therapiesimproves the safety of cell therapy products by extending the shelf life of a product and permitting completion of safety and quality control testing before release of the product for use. preservation permits coordination between the manufacture of the therapy and patient care regimes.

Related Conferences:

7th Annual Conference on Stem Cell and Regenerative Medicine,Paris, France,Aug 04-05, 2016; 2nd Biotechnology World Convention,LONDON, UK,May 25-27, 2017; International Conference on Animal and Human Cell Culture, Jackson Ville, USA, Sep 25-27, 2017; 9th International Conference onCancer Genomics, Chicago, USA, May 29-31, 2017; 3rd International Conference onSynthetic Biology, Munich, Germany, July 20-21, 2017.

Track-13 Stem cell products:

The globalstemcell,Stem cell productsmarket will grow from about $5.6 billion in 2013 to nearly $10.6 billion in 2018, registering a compound annual growth rate (CAGR) of 13.6% from 2013 through 2018.This trackdiscusses the implications ofstemcellresearchand commercial trends in the context of the current size and growth of thepharmaceutical market, both in global terms and analysed by the most important national markets.

Related Conferences:

6th International Conference onTissue Engineering & Regenerative Medicine, Baltimore, USA, Aug 20-22, 2017; 8th World Congress and Expo onCell & Stem Cell Research, Orlando, USA, March 20-22, 2017; 15thWorld Congress on Biotechnology and Biotech Industries Meet,Rome, Italy,March 20-21, 2017; 2nd International Conference onGenetic Counselling and Genomic Medicine ,Beijing, China,July 10-12, 2017; International Conference onClinical and Molecular Genetics, las vegas, USA, April 24-26, 2017.

Track-14 Genetically inherited diseases:

Agenetic diseaseis any disease that is caused by an abnormality in an individual'sgenome, the person's entiregeneticmakeup. The abnormality can range from minuscule to major -- from a discrete mutation in a single base in the DNA of a single gene to a grosschromosome abnormalityinvolving the addition or subtraction of an entirechromosomeor set of chromosomes.Most genetic diseases are the direct result of a mutation in one gene. However, one of the most difficult problems ahead is to find out how genes contribute to diseases that have a complex pattern ofinheritance, such as in the cases of diabetes,asthma,cancerandmental illness. In all these cases, no one gene has the yes/no power to say whether a person has a disease or not. It is likely that more than one mutation is required before the disease is manifest, and a number of genes may each make a subtle contribution to a person's susceptibility to a disease; genes may also affect how a person reacts toenvironmental factors.

Related Conferences:

15th Biotechnology Congress, Baltimore, USA, June 22-23, 2017; 3rd International Conference onSynthetic Biology, Munich, Germany, July 20-21, 2017; 5th International Conference and Exhibition onCell and Gene Therapy,Madrid, Spain,Mar 2-3, 2017; International Conference onCell Signalling and Cancer Therapy,paris, France,Aug 20-22, 2017; International Conference on Animal and Human Cell Culture, Jackson Ville, USA, Sep 25-27, 2017.

Track-15 Plant stem cells:

Plantshave emerged as powerful production platforms for the expression of fully functional recombinantmammalian proteins. These expression systems have demonstrated the ability to produce complexglycoproteinsin a cost-efficient manner at large scale. The full realization of thetherapeuticpotential of stem cells has only recently come into the forefront ofregenerative medicine. Stem cells are unprogrammed cells that can differentiate into cells with specific functions.Regenerative therapiesare used to stimulate healing and might be used in the future to treat various kinds of diseases.Regenerative medicinewill result in an extended healthy life span. A fresh apple is a symbol for beautiful skin. Hair greying for example could be shown to result from the fact that themelanocyte stem cellsin the hair follicle have died off.

Related Conferences:

9th International Conference onGenomics and Pharmacogenomics, Chicago, USA, July 13-14, 2017; 7th International Conference onPlant Genomics, Bangkok, Thailand, July 03-05, 2017; 15th Euro Biotechnology Congress, Valencia, Spain, June 05-07, 2017; 5th International Conference and Exhibition onCell and Gene Therapy,Madrid, Spain,Mar 2-3, 2017; 3rd International Conference & Exhibition onTissue Preservation and Bio banking,Baltimore, USA,June 29-30, 2017.

Track-16 Plant stem cell rejuvenation:

Asplantscannot escape from danger by running or taking flight, they need a special mechanism to withstandenvironmental stress. What empowers them to withstand harsh attacks and preserve life is the stem cell. According to Wikipedia, plantstem cellsnever undergo theagingprocess but constantly create new specialized and unspecialized cells, and they have the potential to grow into any organ, tissue, or cell in the body. The everlasting life is due to the hormones auxin andgibberellin. British scientists found that plant stem cells were much more sensitive toDNAdamage than other cells. And once they sense damage, they trigger death of these cells.

Rejuvenate with Plant Stem Cells

Detoxifyand release toxins on a cellular level. Nourishyour body with vital nutrients. Regenerateyour cells and diminish the effects of aging.

Related Conferences:

International Conference on Animal and Human Cell Culture, Jackson Ville, USA, Sep 25-27, 2017; 14th Asia-Pacific Biotech Congress,Beijing, China,April 10-12, 2017; 15th Biotechnology Congress, Baltimore, USA, June 22-23, 2017; 3rd International Conference onSynthetic Biology, Munich, Germany,July 20-21, 2017; 5th International Conference and Exhibition on Cell and Gene Therapy,Madrid, Spain,Mar 2-3, 2017.

Track-17 Clinical trials in cell and gene therapy:

Aclinical trialis a research study that seeks to determine if a treatment is safe and effective. Advancing new cell andgene therapies(CGTs) from the laboratory into early-phaseclinical trialshas proven to be a complex task even for experienced investigators. Due to the wide variety ofCGTproducts and their potential applications, a case-by-case assessment is warranted for the design of each clinical trial.

Objectives:Determine thepharmacokineticsof this regimen by the persistence of modified T cells in the blood of these patients, Evaluate theimmunogenicityof murine sequences in chimeric anti-CEA Ig TCR, Assess immunologic parameters which correlate with the efficacy of this regimen in these patients, Evaluate, in a preliminary manner, the efficacy of this regimen in patients with CEA bearingtumours.

Related Conferences:

2nd Biotechnology World Convention,London, UK,May 25-27, 2017; International Conference on Animal and Human Cell Culture, Jackson Ville, USA, Sep 25-27, 2017; 9th International Conference onCancer Genomics, Chicago, USA, May 29-31, 2017; 8th World Congress and Expo onCell & Stem Cell Research, Orlando, USA, March 20-22, 2017; 15thWorld Congress on Biotechnology and Biotech Industries Meet,Rome, Italy,March 20-21, 2017.

Track-18 Molecular epigenetics:

Epigeneticsis the study of heritable changes in thephenotypeof a cell or organism that are not caused by its genotype. The molecular basis of anepigeneticprofile arises from covalent modifications of protein andDNAcomponents ofchromatin. The epigenetic profile of a cell often dictates cell fate, as well as mammalian development,agingand disease. Epigenetics has evolved to become the science that explains how the differences in the patterns ofgene expressionin diverse cells or tissues are executed and inherited.

Related Confderences:

5th International Conference onIntegrative Biology, London, UK, June 19-21, 2017; 2nd World Congress on Human Genetics, Chicago, USA, July 24-26, 2017; 9th International Conference onGenomics and Pharmacogenomics, Chicago, USA, July 13-14, 2017; International Conference onIntegrative Medicine & Nutrition, Dubai, UAE, May11-13, 2017; 14th Asia-Pacific Biotech Congress,Beijing, China,April 10-12, 2017.

Track-19 Bioengineering therapeutics:

The goals ofbioengineeringstrategies for targetedcancertherapies are (1) to deliver a high dose of an anticancer drug directly to a cancer tumour, (2) to enhance drug uptake by malignant cells, and (3) to minimize drug uptake by non-malignant cells. In ESRD micro electro mechanical systems andnanotechnologyto create components such as robust silicon Nano pore filters that mimic natural kidney structure for high-efficiency toxin clearance. It also usestissue engineeringto build a miniature bioreactor in which immune-isolated human-derived renal cells perform key functions, such as reabsorption of water and salts.In drug delivery for a leading cause ofblindness, photo-etching fabrication techniques from themicrochipindustry to create thin-film and planar micro devices (dimensions in millionths of meters) with protectivemedicationreservoirs andnanopores(measured in billionths of meters) for insertion in the back of the eye to deliver sustained doses of drug across protective retinalepithelial tissuesover the course of several months.

Related Conferences:

6th International Conference onTissue Engineering & Regenerative Medicine, Baltimore, USA, Aug 20-22, 2017; 8th World Congress and Expo onCell & Stem Cell Research, Orlando, USA, March 20-22, 2017; 15thWorld Congress on Biotechnology and Biotech Industries Meet,Rome, Italy,March 20-21, 2017; 2nd International Conference onGenetic Counselling and Genomic Medicine ,Beijing, China,July 10-12, 2017; International Conference onClinical and Molecular Genetics, Las Vegas, USA, April 24-26, 2017.

Track-20 Advanced gene therapy:

Advanced therapiesare different fromconventional medicines, which are made from chemicals or proteins.Gene-therapymedicines:these contain genes that lead to atherapeuticeffect. They work by inserting 'recombinant' genes into cells, usually to treat a variety of diseases, including genetic disorders, cancer or long-term diseases.Somatic-cell therapymedicines:these contain cells or tissues that have been manipulated to change their biological characteristics.Advanced Cell &Gene Therapyprovides guidanceinprocess development, GMP/GTP manufacturing,regulatory affairs, due diligence and strategy, specializing in cell therapy,gene therapy, and tissue-engineeredregenerative medicineproducts.

Related Conferences:

9th International Conference onGenomics and Pharmacogenomics, Chicago, USA, July 13-14, 2017; 7th International Conference onPlant Genomics, Bangkong,Thailand, July 03-05, 2017; International Conference onIntegrative Medicine & Nutrition, Dubai, UAE, May11-13, 2017; 14th Asia-Pacific Biotech Congress, Beijing,China,April 10-12, 2017; 2nd World Congress on Human Genetics, Chicago, USA, July 24-26, 2017.

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5th International Conference and Exhibition on

Ongoing Safety Initiatives

Pivotal clinical trials evaluate if a medicine is safe and effective enough to receive FDA approval for marketing. Even after approval, there is still a lot to learn about the medicine. For example, understanding the particular efficacy and safety profile of a medicine for pediatric uses or in the elderly.

In addition, doctors and patients may use our medicines in ways we have not already studied. So, we often conduct real world studies to assess the emerging risks and benefits of our medicine in larger or more diverse populations.

Medicines can have more than one single use. They may be used in different ways for the same disease or across diseases. A good example of this is cancer, where the biology often tells us that a medicine for treating one type of cancer could potentially work in another.

Approval initiates additional research by doctors worldwide. We may opt to partner with doctors at universities and hospitals to support these investigator-sponsored trials. And in doing so, understand the potential and limitation of a new medicine across may different diseases, dosing regimens and drug combinations.

Approval signals our ability to deliver our medicines to the patients who need it. But beyond the physical delivery, when one of our medicines is prescribed, we have a dedicated team of people who can help patients understand the range of support services we provide.

We offer coverage and co-pay support as well as patient assistance to make sure that healthcare coverage isnt a barrier to patients seeking our medicines. Whenever possible, we make sure that people can get the medicines they need, regardless of their ability to pay.

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Genentech : Making Medicine

Introduction

The Academies have provided leadership in the past on controversial new areas of genetic research, such as recombinant DNA technology, human embryonic stem cell research, human cloning, and gain-of-function research. In keeping with these past efforts, the National Academy of Sciences and the National Academy of Medicine have launched a new initiative to inform decision making related to recent advances in human gene-editing research. [Learn about related Academies studies and reports on genetic research]

The initiative includesan international summit to convene global experts to discuss the scientific, ethical, and governance issues associated with human gene-editing research, as well as a comprehensive studyby a multidisciplinary, international committee that will examine the scientific underpinnings and clinical, ethical, legal, and social implications of human gene editing. The committee will issue a report in 2016 with findings and recommendations for the responsible use of human gene-editing research.

Latest News

Study on Human Gene Editing Begins; First Data-Gathering Meeting Feb. 11-12 NAS and NAM are now moving forward with the second component of the Academies' Human Gene Editing Initiative, an in-depth, comprehensive review of the science and policy of human gene editing. Read Announcement

International Summit Concludes The U.S. National Academy of Sciences, U.S. National Academy of Medicine, Chinese Academy of Sciences, and the U.K.'s Royal Society co-hosted athree-day international summitwhere global experts discussed the scientific, ethical, and governance issues associated with these new and emerging human gene-editing technologies.

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About This Initiative

Powerful new gene-editing technologies, such as CRISPR-Cas9, hold great promise for advancing science and treating disease, but they also raise concerns and present complex challenges, particularly because of their potential to be used to make genetic changes that could be passed on to future generations, thereby modifying the human germline.

The National Academy of Sciences and the National Academy of Medicine's human gene-editing initiative will provide researchers, clinicians, policymakers, and societies around the world with a comprehensive understanding of human gene editing to help inform decision making about this research and its application.

Subscribe to our mailing list for updates by clicking on the button below.Questions about the initiative should be directed togeneediting@nas.edu.

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My Philosophy and Approach to Wellness... As an internist in the Rochester, New York area with 35 years of knowledge and experience, I strive to meet the personal needs of each of my patients through a preventive healthcare model, sometimes compared to concierge care.

Located in Pittsford, New York, my primary care practice serves patients in Monroe County, as well as locations between Buffalo and Syracuse and south of Rochester. I am affiliated with Rochester General Hospital. If hospitalized, I will visit you and coordinate your care with specialists at Strong Memorial, Highland and Unity hospitals. I regularly collaborate with the leading specialists in cardiology, neurology, psychiatry and geriatrics.

As a private internal medicine physician, my patients receive same-day or next-day visits in an unhurried, relaxed office where I can listen, diagnose and help you achieve your optimal health through wellness and prevention.I am never too busy to see you, and that includes house calls.I am just a phone call away, including evenings and weekends. I believe a very strong doctor-patient relationship is essential in achieving the best plan for healthy living and prevention of illness.

I believe that diet and exercise are essential components in achieving good health and I practice what I preach. I enjoy organic gardening and exercise regularly by swimming laps.My staff is very welcoming, efficient and will personally answer your calls and questions.I invite you to call my office and allow my staff to arrange a complimentary face-to-face office visit. Let me understand your health goals and show you how my proactive and personalized approach to healthcare will improve your life.

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Dr. Zito is one of those rare doctors that actually takes the time to LISTEN to his patients. He gets to the root of what works an...

Feel very comfortable with Dr. Zito and his expanations of my problems

Dr Zito has been our family primary care doctor ever since we moved to USA from UK, He and his staff has been are extremely caring...

I was in the hospital and the on call cardiologist said that I was fine to be discharged even after my complaining of severe pain ...

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Gene M. Zito, MD - Internal Medicine Doctor in Pittsford ...

Assistant Professor of Clinical Pathology and Dermatology Director of Dermatopathology

Dr. Kim is an assistant professor of dermatology and pathology at USC where he serves as the director of dermatopathology. He joined the Keck School of Medicine in July 2008.

Dr. Kim has lived and trained in many parts of the United States. Most recently, he completed a dermatopathology fellowship at Northwestern University in Chicago. Prior to that, he joined the faculty at Indiana University Department of Dermatology in Indianapolis.

Dr. Kim completed his dermatology residency at New York University in Manhattan where he also served as chief resident. He earned his undergraduate and medical degrees from Duke University and Indiana University, respectively.

Dr. Kim has earned numerous academic distinctions during his career. In addition to these distinctions, Dr. Kim has also won awards for community service leadership. Dr. Kim cares for patients with all types of dermatologic conditions. He is also available for dermatopathology consultations.

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Keck Medicine of USC - Gene H. Kim

Following on the heels of Angelina Jolie's widely celebrated decision to remove her breasts 'preventively,' few truly understand how important preventing environmental chemical exposures and incorporating cancer-preventing foods into their diet really is in reducing the risk of gene-mediated breast cancer.

There is so much fear and misinformation surrounding the so-called 'Breast Cancer Associated' genes, BRCA1 and BRCA2, that it should help to dispel some prevailing myths by looking at the crucial role that epigenetic factors play in their expression. Literally 'above' (epi) or 'beyond' the control of the genes, these factors include environmental chemical exposures, nutrition and stress, which profoundly affect cancer risk within us all, regardless of what variant ('mutated' or 'wild')* that we happen to carry within our genomes.

In 2012, a very important study was published in the Journal of Nutritional Biochemistry that looked at the role a natural compound called resveratrol may play in preventing the inactivation of the BRCA-1 gene. BRCA-1 is known as a "caretaker" gene because it is responsible for healing up double-strand breaks within our DNA. When the BRCA-1 gene is rendered dysfunctional or becomes inactivated, either through a congenital/germline inheritance of DNA defects ('mutation') or through chemical exposures, the result is the same: harm to the DNA repair mechanisms within the affected cells (particularly breast and ovary; possibly testicular), hence increasing the risk of cancer.

Ironically, while the prevalence of a "bad" inherited BRCA1 variation is actually quite low relative to the general population (A 2003 study found only 6.6% of breast cancer patients even have either a BRCA1 and BRCA2 germline mutation[1]), everyone's BRCA1 and BRCA2 genes are susceptible to damage from environmental chemical exposures, most particularly xenobiotic (non-natural) chemicals and radiation. This means that instead of looking to a set of "bad" genes as the primary cause of cancer, we should be looking to avoid exposing both our "bad" and "good" genes alike to preventable chemical exposures, as well as avoiding nutrient deficiencies and/or incompatibilities, which also play a vital role in enabling us to express or silence cancer-associated genes. [For more on why genes don't "cause" disease see: The Great DNA Data Deficit.]

The aforementioned resveratrol study is titled "BRCA-1 promoter hypermethylation and silencing induced by the aromatic hydrocarbon receptor-ligand TCDD are prevented by resveratrol in MCF-7 Cells."

Quite a mouthful.

Essentially, the BRCA-1 promoter is the gene sequence within the BRCA1 gene that drives the production of the protein that enables our cells to repair DNA damage, and when "silenced" (i.e. hypermethylated) via the receptor for aromatic hydrocarbons (which are primarily xenobiotic petrochemical compounds), it leads to chromosomal damage within those cells. This study looked at the role of resveratrol, a natural compound found in grapes, wine, chocolate, and peanuts, in preventing these chemically-induced changes in gene methylation, also known as 'gene silencing.'

According to the study:

"The aberrant hypermethylation of tumor suppressor genes has been recognized as a predisposing event in breast carcinogenesis [1]. For example, BRCA-1 promoter hypermethylation has been linked to loss or silencing of BRCA-1 expression in sporadic breast tumors [27] and the development of high-grade breast carcinomas [810]. Higher incidence (30%90%) of BRCA-1 hypermethylation has been reported in infiltrating tumors [2,1012], suggesting that epigenetic repression of BRCA-1 may accompany the transition to more invasive phenotypes. Moreover, BRCA-1 promoter methylation was found to be positively associated with increased mortality among women with breast cancer [13].

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Disclaimer: This article is not intended to provide medical advice, diagnosis or treatment. Views expressed here do not necessarily reflect those of GreenMedInfo or its staff.

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Home Asia Unhedged China banks on gene power firms for precision medicine

By Asia Unhedged on January 6, 2016 in

(From Caixin Online)

By staff reporter Wang Qionghui

The Chinese government is powering a homegrown precision medicine initiative aimed at improving patient treatment for chronic ailments such as cardiovascular disease, cancer and diabetes.

Human genome

Officials have declared precision medicine a customized form of health care based on genome-sequencing technology as one of the nations foremost science and technology projects under the 13th Five-Year Plan for the 2016-20 period.

A document published after a March meeting hosted by the Ministry of Science and Technology says the central government plans to spend 20 billion yuan to support precision medicine research by 2030, matching an anticipated 40 billion yuan in private investment. Moreover, the top public health authority, the National Health and Family Planning Commission, is drafting a strategic plan for promoting precision medicines development nationwide.

Companies that expect to benefit from the initiative include Shenzhen-based BGI Genomics Co., Hangzhous Berry Genomics Co. and Beijing Biomarker Technologies. Although young, the genetics services sector in the country is already diversifying, with firms staking claims in specialties such as prenatal care and niche services like disease and cancer detection through genetic testing.

BGI, the nations leader in genome sequencing, is a 16-year-old company that bought U.S. medical equipment maker Complete Genomics in 2012 and last October rolled out its first homegrown genome sequencing machine. Berry, established in 2010, is Chinas second-largest genome sequencer and the developer of non-invasive prenatal testing procedure thats been offered since 2011. Beijing Biomarker, founded in 2009, serves research institutions with genetic analyses and testing services.

The precision medicine movement has also won the attention of Internet and computer companies. In October, the U.S. chip maker Intel Corp. and Chinas e-commerce leader Alibaba Group Holding Ltd. announced a three-way partnership with BGI. The firms said they will collaborate to build a cloud-based online platform allowing clinics to access genetic data and other precision medicine services.

Precision medicine requires sharing an individuals genetic data and comparing it to huge amounts of data from similar patients, said Li Yingrui, chief executive of BGI Tech Solution Co., a subsidiary of BGI. Health specialists then use those comparisons to find differences and similarities to work out precise treatment regimes for individual patients. Read more

Categories: Asia Unhedged, China

Tags: BGI Genomics, Caixin Online, Chinese 13th five-year plan, Chinese government precision medicine efforts, Chinese human genome companies, Chinese human genome research

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China banks on gene power firms for precision medicine ...

MONDAY, June 5, 2017 (HealthDay News) -- Two drugs that target genetic flaws are giving people with specific types of advanced lung cancer a chance to live longer and better, a pair of new clinical trials finds.

A newly approved drug called alectinib (Alecensa) works twice as long as the current standard medication in halting cancer growth in patients with ALK-positive non-small cell lung cancer, results from a new global clinical trial show.

ALK is a gene that produces a protein that helps cancer cells grow and spread, according to the American Cancer Society (ACS).

In another study, an experimental drug called dacomitinib delayed cancer growth by about half in non-small cell lung cancer patients who had a mutation of the epidermal growth factor receptor (EGFR) that caused cancer cells to grow faster, a second trial reported. Non-small cell lung cancers comprise most lung cancer cases.

EGFR is a substance normally found on cells that helps them grow and divide, the ACS says.

The drugs, alectinib in particular, will let people live months or years longer just by taking a daily pill, said Dr. Bruce Johnson, chief clinical research officer at Dana-Farber Cancer Institute in Boston. Johnson is also incoming president of the American Society of Clinical Oncology (ASCO).

Alectinib works more than a year longer than crizotinib (Xalkori), which itself supplanted chemotherapy a few years back because it proved more effective with fewer side effects, Johnson said.

"This is kind of a game changer, because the drug itself works at least for two years, plus there are other treatments" that can be substituted when it ultimately becomes ineffective, Johnson said of alectinib. "We used to have to tell these patients 10 or 15 years ago that you've got eight months to a year. Now they most likely have years."

Both of these genetically driven forms of lung cancer are more common in nonsmokers, the ACS says.

The studies were both funded by the drug manufacturers. Hoffmann-La Roche funded the alectinib study. Pfizer and SFJ Pharmaceuticals Group funded the dacomitinib study.

The first clinical trial revealed that alectinib halts lung cancer growth for about 26 months on average. That compared to about 10 months on average for crizotinib, the drug now used as front-line treatment for ALK-positive patients.

Alectinib also works 84 percent better than crizotinib at preventing spread of advanced lung cancer to the brain, because it is better able to penetrate into the brain and kill cancer cells there, said lead researcher Dr. Alice Shaw, director of thoracic oncology at Massachusetts General Hospital Cancer Center in Boston.

About 5 percent of non-small cell lung cancer cases are ALK-positive. That means they have a genetically abnormal protein that fuels cancer growth. In the United States, about 12,500 people are diagnosed with ALK-positive non-small cell lung cancer each year, researchers said in background information.

Alectinib already is approved in the United States as a treatment for ALK-positive patients who no longer respond to crizotinib, Shaw said.

The results should "establish alectinib as the new standard of care" for ALK-positive lung cancer patients, rather than crizotinib, Shaw said.

ASCO expert Dr. John Heymach agreed, calling the clinical trial a "watershed moment."

Not only did the drug work better and longer, but it also produced fewer side effects in patients, noted Heymach, chair of thoracic/head and neck oncology for the University of Texas MD Anderson Cancer Center in Houston.

The most common side effects for alectinib were fatigue, constipation, muscle aches and swelling, while crizotinib patients most often suffered from gastrointestinal problems and liver enzyme abnormalities, according to the researchers.

The second clinical trial compared a new drug, dacomitinib, to the current standard targeted drug gefitinib (Iressa) in treating EGFR-positive lung cancer.

Each year about 15,000 people in the United States are diagnosed with EGFR-positive lung cancer, which involve mutations that increase the growth of cancer cells, researchers said in background notes.

Dacomitinib blocked EGFR mutations more effectively than first-generation drug gefitinib, providing a 41 percent lower chance of cancer progression or death, researchers found. On average, dacomitinib halted cancer growth for 14.7 months in patients, compared with 9.2 months with gefitinib.

"From the perspective of doctors who treat lung cancer daily, this is really a substantial advance," Heymach said, noting that the results put the drug "at the front of the pack in terms of efficacy."

However, dacomitinib also created more side effects, including acne in about 14 percent of patients and diarrhea in 8 percent of patients. Doctors wound up reducing the dosage in about 66 percent of patients as a result of side effects, said lead researcher Dr. Tony Mok, chair of clinical oncology at the Chinese University of Hong Kong.

Heymach said the side effects are "not life-threatening toxicities."

"These are toxicities that doctors who treat this for a living become accustomed to managing," Heymach said.

"At the end of the day, I think we now have one additional choice" in treating EGRF-positive non-small cell lung cancer, Mok concluded, adding that dacomitinib should be considered as a new first-line alternative treatment. The drug has not received FDA approval.

Neither of the tested drugs will be cheap. "Almost all these targeted drugs are thousands of dollars per month," Johnson said.

The results of both trials were scheduled to be presented Monday at ASCO's annual meeting, in Chicago. The findings were also being published June 6 in the New England Journal of Medicine.

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Gene-Targeted Drugs Fight Advanced Lung Cancers - Montana Standard

Feb. 12, 2020 13:00 UTC

BRISBANE, Calif.--(BUSINESS WIRE)-- Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced the appointment of Dr. John Markels, an accomplished pharmaceutical executive with three decades of general management, manufacturing and technology experience at Merck, to the Sangamo Board of Directors.

"We are very pleased to welcome John to our Board," said Sandy Macrae, Chief Executive Officer of Sangamo. Johns manufacturing expertise and global general management and therapeutic area leadership experience will greatly benefit Sangamo as we build out our own in-house gene and cell therapy manufacturing capabilities and advance our innovative pipeline of genomic medicine product candidates toward registration and eventual commercialization.

Dr. Markels has over 30 years of leadership experience in the pharmaceutical industry. He currently serves as President of Global Vaccines at Merck, a role in which he leads an integrated team dedicated to discovery and development, supply and access, and global marketing and long-term strategy for the vaccines portfolio. Earlier roles at Merck included President, Latin America, as well as a long career in senior leadership positions in major regions worldwide in manufacturing technology, operations and strategy, business development, alliance management, and supply chain. Dr. Markels received his Ph.D. in chemical engineering from the University of California, Berkeley and his B.S. in chemical engineering from the University of Delaware.

About Sangamo Therapeutics

Sangamo Therapeutics is committed to translating ground-breaking science into genomic medicines with the potential to transform patients lives using gene therapy, ex vivo gene-edited cell therapy, and in vivo genome editing and genome regulation. For more information about Sangamo, visit http://www.sangamo.com.

Forward Looking Statements

This press release contains or refers to forward-looking statements regarding Sangamo's current expectations. These forward-looking statements include, without limitation, statements regarding the potential benefits of cell therapy, the Company's ability to develop and commercialize product candidates to address genetic diseases with the Company's proprietary technologies and the timing of commencement or next stages of such programs and the anticipated benefits therefrom. These statements are not guarantees of future performance and are subject to certain risks, uncertainties and assumptions that are difficult to predict. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in Sangamo's operations and business environments. These risks and uncertainties are described more fully in Sangamo's Annual Report on Form 10-K for the year ended December 31, 2018 as filed with the Securities and Exchange Commission on March 1, 2019 and Sangamo's Quarterly Report on Form 10-Q for the quarter ended September 30, 2019 that it filed on November 6, 2019. Forward-looking statements contained in this announcement are made as of this date, and Sangamo undertakes no duty to update such information except as required under applicable law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200212005263/en/

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Sangamo Therapeutics Appoints John Markels to Its Board of Directors - BioSpace

Two gene editing firms unveil cancer drug pacts as ASCO comes to a close Boston Business Journal The research drew blowback from Intellia, which said that the claim about off-target effects lacked evidence and was based on a sample. In addition to Intellia and CRISPR Therapeutics, the other leading gene-editing firm is Cambridge-based Editas ... and more »

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Two gene editing firms unveil cancer drug pacts as ASCO comes to a close - Boston Business Journal

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In world-first breakthrough, French doctors use gene therapy to treat rare blood disease - RFI

Science Daily

Study parses influence of genes, environment in metabolic disease Science Daily By comparing two strains of mice -- one that becomes obese and diabetic on a high-fat diet and another resistant to a high-fat regimen -- researchers from the Perelman School of Medicine at the University of Pennsylvania identified genome-wide changes ...

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Study parses influence of genes, environment in metabolic disease - Science Daily

Potential Treatment

Gene therapy has been available for quite some time now. Advances in modern medical science, particularly in stem cell research, have made it possible to use DNA to compensate for malfunctioning genes in humans. The therapies haveeven proven effective fortreating rare forms of diseases. Now, a research team in France has shown that gene therapy may be used to cure one of the most common genetic diseases in the world.

The team, led by Marina Cavazzana at the Necker Childrens Hospital in Paris, conducted stem cell treatment on a teenage boy with sickle cell disease. The disease alters theblood through beta-globin mutations, which cause abnormalities in the blood proteinhemoglobin. These abnormalities cause the blood cells (which have an irregular shape, like a sickle, hence their name) to clump together. Patients with sickle cell disease usually need transfusions to clear the blockages their cells cause, and some are able to have bone marrow transplants. About 5 percent of the global population has sickle cell disease,according to the WHO. In the United States alone, the CDC reports that approximately 100,000 people have sickle cell disease.

The patient is now 15 years old and free of all previous medication, Cavazzana saidwhen discussing the outcome of their study. He has been free of pain from blood vessel blockages, and has given up taking opioid painkillers. Their research is published in the the New England Journal of Medicine.

The particular treatment given to the teenage boy at Necker Childrens Hospitalbegan when he was 13 years old. The team took bone marrow stem cells from the boy and added mutated versions of the gene that codes for beta-globin before putting these stem cells back into the boys body. The mutated genes were designed to stop hemoglobin from clumping together and blocking blood vessels the hallmark of sickle cell disease.

Two years later, the boys outcomelooks promising.All the tests we performed on his blood show that hes been cured, but more certainty can only come from long-term follow-up, Cavazzan said. Her team also treated seven other patients who also showed promising progress.

If the method shows success in larger scale clinical trials, it could be a game changer, saidDeborah Gill at the University of Oxford, The fact the team has a patient with real clinical benefit, and biological markers to prove it, is a very big deal.

Other research involving gene therapy is also showing similar promise. One which has already been approved by the FDA is a potential treatment for blindness. Others look at treating Parkinsons disease or evenprolonging human life. What these studies show is that gene therapyand stem cells may be able togive hope to patients with diseases that have long been considered incurable.

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Doctors Claim They've Cured a Boy of a Painful Blood Disorder Using Gene Therapy - Futurism

A pediatrician decides a struggling teen with mental illness needs hospitalization to neutralize psychologic demons impacting their personal and social life.

A workers compensation doctor requests a neck MRI in a powerline worker with growing right arm numbness and weakness to search for potential paralyzing nerve impingement.

An orthopedist orders special testing to determine if an elder patient with right hip pain which limits walking and driving might need surgery to improve her quality of life.

Physicians are rigorously trained to make decisions in the best interest of their patients. Even after medical school and residency, doctors must follow the challenges of evidence-based medicine, standard of care, peer review, and muster the time for continuing medical education and certification.

Doctors are not only held accountable by their peers, but also legally, as they could be subject to lawsuits. Additionally, state licensing agencies that oversee medical professionals can discipline them, should they not practice medicine up to the standards of quality medical decision-making.

However, what if the teens pediatrician feels hospitalization is acutely needed for mental illness, but it is denied by the insurance company? What if the workers comp physician orders an MRI for the powerline workers ailing right arm, but it is denied? Or, if special testing to evaluate grandmas worsening mobility and pain is turned down by the HMO? Who is held accountable?

To justify requests for specific patient care, physicians are forced to have peer-to-peer phone discussions with doctors employed by insurance companies, workers comp and HMOs. Frequently, these conversations result in denial of further care without medical justification.

A controversial question arises: Are denials by these company doctors considered medical decisions?

They are not. These decisions are considered utilization review. What does this mean? They are making decisions based on controlling costs, which is in the financial interest of the for-profit agencies they serve but not necessarily in the best interest of the patient.

Even though they are licensed doctors practicing medicine, their role in patient care is under the guise of utilization review and therefore not under the scrutiny of state licensing agencies.

What if these physicians deny care because they are incentivized to enhance personal bonuses? More so, what if some are making decisions outside the realm of their medical expertise (e.g., a urologist deciding about a diabetic)? Who holds these physicians accountable for moral transgressions or lack of judgement?

In California, we have a Medical Board that oversees licensing for all state physicians. If you report a licensed physician for making substandard medical decisions, an investigation ensues. If, though, the doctor is employed by an insurance company, workers comp or HMO and makes denial decisions on their behalf, it is considered utilization review, and they are not held accountable.

I do not pretend to understand every law and rule governing the Medical Board. But these companies have created legal barriers protecting doctors who might make substandard medical decisions.

Many physicians continue to fight for patient care rights despite frustration and helplessness of ongoing phone calls and paperwork they face. Yet substandard medical care will hamper their efforts as laws are manipulated and oversight is negligible.

Making medical decisions has never been easy. Assuring accountability makes it even harder.

Gene Uzawa Dorio, M.D., is a housecall geriatric physician and member of thePhysicians Organizing Committee atHenry Mayo Newhall Hospital. The views expressed in this column as his alone.

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Non-Medical Medical Decisions | Commentary by Dr. Gene Dorio - SCVNEWS.com