Find out if this #LungCancer #Drug can save lives? - http://clapway.com/2015/08/21/new-genentech-trial-targets-multiple-cancers-by-attacking-a-single-gene-342/

In an interesting new trial funded by Genentech, vemurafenib, a skin cancer drug showed positive results in certain kinds of lung cancer and some other malignancies. The trial was based on a new concept of targeting gene mutations common to many cancers.

Current cancer therapies are usually specific to the type of cancer

Most clinical trials test the effect of the drug on a single type of cancer. Lately, advances in gene therapy made it possible to study cancer at its most fundamental level⎯genes. Scientists believe that although cancers may show organ heterogeneity, many of their molecular mechanisms have common genetic origins. For example, a genetic mutation called the BRAF V 600 is found to be present in many cancers.

BRAF is gene that makes the protein B-Raf, which when functioning properly prepares a cell for its death. An aberration in this mechanism sends the cell life-and-death cycle askew, leading to the development of cancer.

The Genentech clinical trial design

The clinical trial, also called the “basket” trial, attempted to test vemurafenib⎯a drug already approved for the treatment of melanomas showing the BRAF mutation⎯in other cancers with the same gene defect. Vemurafenib was developed by Plexxikon and marketed by Genentech under the name Zelboraf. A total of 122 patients with BRAFV 600 mutation-positive, non-melanoma cancers were enrolled in this open-label clinical trial. At the end of the study they were testes on the basis of response to cancer, survival without disease progression, and overall survival.

The clinical trial yielded mixed results

The study tested the drug effects on seven types of tumors. Among them, non-small-cell lung cancer showed a high response rate of 42%; the median survival time before progression of the disease was 7.3 months. Another group of cancer-like conditions⎯Erdheim–Chester disease or Langerhans’-cell histiocytosis⎯showed a 43% response rate and median treatment time of 5.9 months, with no one showing disease progression during this period. There were some anecdotal results with anaplastic thyroid cancer, salivary-duct cancer, ovarian cancer, and colorectal cancer; not much confidence can be placed on these results.

This study is a proof-of-concept that ushers in an era of molecular medicine. Though the results aren’t enough to yield an approval, this is a good starting point.

Here’s what you can do at home to lower your risk of cancer!

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New Genentech Trial Targets Multiple Cancers by Attacking a Single Gene

I would really love to know what other members of the deaf community think about gene therapy and how they feel about the possibility of hearing again. - http://clapway.com/2015/07/13/how-gene-therapy-might-restore-hearing-in-the-future/

Hearing loss affects five percent of the population. Five percent doesn’t sound like a lot, but in reality, it’s 360 million people worldwide. That’s a lot of people. As you know from my previous blogs, there’s a special place in my heart for the deaf community, so I find any related information interesting. When I saw the study done with gene therapy on deaf mice, I had to find out more.

The Deaf Culture

Many adults who are deaf were not born that way. I had a teacher when I was learning Sign Language explain to us that she was born hearing, but lost the ability as a child due to meningitis.

Medical technology was not as advanced and this is the outcome for several older deaf people. While deafness can be caused by various circumstances, it is not always the case that a person who is deaf would prefer the ability to hear.

Speaking from my experience in the community, I have known people who are perfectly happy and healthy being deaf and when asked if the opportunity ever arose to change their hearing status, they say they would decline. I know that part of the reason, for a few of these people, is that they don’t want to go through surgery to implant devices or have to mess with hearing aids all the time. They’ve lived their whole lives being deaf and don’t see it as a “disorder” or a “disability.” However, there are those that would accept a change in their hearing abilities. Each person will have their own opinion, and nothing is wrong with either side of it, but I’m very interested to see how this gene therapy study will impact the community.

What is Gene Therapy?

Gene therapy is a type of treatment still in the experimental phase. It involves taking genetic material and introducing it into a person’s cells to fight, or in some cases prevent, a disease. Research is being done with a number of diseases to see how well gene therapy will work in each scenario.

These diseases include immune deficiency disorders, Parkinson’s, some forms of cancer and even HIV. Various approaches are being taken in the testing process, such as replacing a mutated gene, taking out a mutated gene, or introducing a new gene into the body.

Restored Hearing in Mice with Genetic Deafness

While this study is still very experimental, researchers at Boston Children’s Hospital and Harvard Medical School have had successfully restored the hearing of mice with a genetic deafness in their test group. Improvements still need to be made, and while this type of therapy isn’t quite ready for clinical trials, the researchers are hopeful that they can be working with people in the near future.

As I mentioned before, the people I know are very torn on the subject. While I think it’s an enormous breakthrough for people who have lost their hearing later in life, I’m not sure that people who were born deaf would feel the same way.

I would really love to know what other members of the deaf community think about gene therapy and how they feel about the possibility of hearing again. Are you a part of the community? I’d love to hear your thoughts below in the comments section! 

How Gene Therapy Might Restore Hearing in the Future

Gene Therapy Could Be the Key to Deafness - http://clapway.com/2015/07/09/gene-therapy-could-be-the-key-to-deafness798/

A study published in the Journal Science Translation Medicine has discussed the successful restoration of hearing in mice with genetic forms of deafness by using gene therapy. The research offers hope for children born with genes that cause profound hearing loss.

70 DIFFERENT GENES CAN RESULT IN DEAFNESS

This has opened the doors for researchers to try the same on humans. More than 70 different defective genes are known to result in deafness. The scientists in this study focused on one gene called the TCM1. It is responsible for between 4-8 percent of cases of deafness and plays a central role in hearing by coding for important inner ear protein.

HARVARD MEDICAL SCHOOL’S GENE THERAPY PROTOCOL ISN’T READY

Boston Children’s Hospital and Harvard Medical School has said that their gene therapy protocol isn’t prepared for clinical trials. They need to tweak it a little in order to use it but there are sure that this could be used for therapeutic use in humans.

GENE THERAPY TESTED IN TWO TYPES OF MICE

The researchers tested gene therapy in two types of mutant mice. One type of mice had the TMC1 gene completely deleted and is the perfect model for the recessive TCM1 mutations in humans. Children who have two mutant copies of TCM1 have acute hearing loss from a young age, around two years old. This treatment was tested on two strains of mutant mice, representing different forms of TCM1-related deafness in humans. One mouse had no functioning TCM1 gene, and children with this kind of recessive genetic defect go deaf from a very young age.

The other strain, called Beethoven, had a less common form of TCM1 deafness caused by one copy of the paired genes not working. This “dominant” defect causes children to go deaf gradually from between the ages of 10 to 15.

In the recessive deafness, gene therapy with TCM1 restored the ability of sensory hairs to respond to sound producing a measurable electrical current and restored activity to the auditory part of the brainstem. In the dominant deafness model, gene therapy with a related gene, TCM2 was a success at the cellular and brain level and restored hearing partially, as proven in the startle test.

The researchers of this study have envisioned that deaf patients will have their genome sequenced and a precision medicine treatment injected into the ears to restore hearing. This form of therapy and restoration of hearing might be the replacement for the traditional cochlear implant.

Researchers restored the hearing of deaf mice by injecting a virus with the healthy gene into their inner ears. This gene therapy cure was successfully tested and could be offered to human patients in a little under five years.

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Gene Therapy Could Be the Key to Deafness

Forget smartphones? Here"s a s small, smart DNA sequencer. - http://clapway.com/2015/06/15/game-changer-dna-sequencer-the-size-of-a-smartphone-325/

Researchers in Canada and the U.K. have sequenced and assembled de novo, the first full genome of a living organism using a DNA sequencer that can fit in your pocket. If when you hear the word ‘minion’ you think of those small yellow creatures in the upcoming computer-animated comedy film ‘Minions’, you will be surprised by what you read next…

It could turn out to be a significant scientific leap forward: a team of scientists has used a mobile-phone-sized DNA sequencer called MinION to assemble a complete bacterial genome. Hard to imagine if we consider competing machines that can be as big as closets.

A REVOLUTION IN DNA-BASED DIAGNOSTICS

The cost-effective device, which has already been used by epidemiologists to read the genomes of Ebola viruses in West Africa and sequence patient DNA, will trigger what some have called a “democratization” of sequencing. Experts believe that with the combination of affordability, long-reads and portability, this USB thumb-drive genetic testing device, is primed to disrupt the way scientists do genomics.

Findings published today in the journal Nature Methods suggest that the DNA sequencer marketed by Oxford Nanopore allows room to scale up and sequence larger and more complex samples of genomes in organisms, eventually including human beings.

CHEWING GUM PACKET-SIZED DNA SEQUENCER

According to Dr. Jared Simpson, Principal Investigator at the Ontario Institute for Cancer Research, this chewing gum packet-sized DNA sequencer is designed to generate real-time data streaming and read long enough stretches of the genome allowing complete reconstructions off a tiny device.

Although some scientists don’t see the groundbreaking adoption of the device at a clinical level, the MinION definitely has an amazing ‘hip’ factor since it is so small and portable, and could be a game-changer due to its relatively affordable price. Traditional sequencers could cost as much as US $1 million, while researchers could use this DNA sequencer for a US$1,000 deposit.

Dr. Tom Hudson, President and Scientific Director of the Ontario Institute for Cancer Research believes that the technology has incredible potential. If scaled up, it could sequence tumour genomes and deliver tailor-made diagnosis and treatment to patients across the globe.

A shortcoming of the technology is that the single reads it produces are currently much less accurate than the reads produced by larger devices. Yet, with a jump in accuracy to match its competitors, this DNA sequencer has the potential to be used by scientists as an alternative or supplement to current options.

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Eventually we’ll neeed Urban Armor Gear for our smart, portable DNA sequencers (one hopes):

GameChanger: DNA Sequencer the Size of a Smartphone