Category Archives: Transhuman News

July 12, 2017 Killer T cells surround a cancer cell. Credit: NIH

Early phase Northwestern Medicine research has demonstrated a potential new therapeutic strategy for treating deadly glioblastoma brain tumors.

The strategy involves using lipid polymer based nanoparticles to deliver molecules to the tumors, where the molecules shut down key cancer drivers called brain tumor initiating cells (BTICs).

"BTICs are malignant brain tumor populations that underlie the therapy resistance, recurrence and unstoppable invasion commonly encountered by glioblastoma patients after the standard treatment regimen of surgical resection, radiation and chemotherapy," explained the study's first author, Dr. Dou Yu, research assistant professor of neurological surgery at Northwestern University Feinberg School of Medicine.

The findings were published in the journal Proceedings of the National Academy of Sciences.

Using mouse models of brain tumors implanted with BTICs derived from human patients, the scientists injected nanoparticles containing small interfering RNA (siRNA)short sequences of RNA molecules that reduce the expression of specific cancer promoting proteinsdirectly into the tumor. In the new study, the strategy stopped tumor growth and extended survival when the therapy was administered continuously through an implanted drug infusion pump.

"This major progress, although still at a conceptual stage, underscores a new direction in the pursuit of a cure for one of the most devastating medical conditions known to mankind," said Yu, who collaborated on the research with principal investigator Dr. Maciej Lesniak, Michael J. Marchese Professor of Neurosurgery and chair of neurological surgery.

Glioblastoma is particularly difficult to treat because its genetic makeup varies from patient to patient. This new therapeutic approach would make it possible to deliver siRNAs to target multiple cancer-causing gene products simultaneously in a particular patient's tumor.

In this study, the scientists tested siRNAs that target four transcription factors highly expressed in many glioblastoma tissuesbut not all. The therapy worked against classes of glioblastoma BTICs with high levels of those transcription factors, while other classes of the cancer did not respond.

"This paints a picture for personalized glioblastoma therapy regimens based on tumor profiling," Yu said. "Customized nanomedicine could target the unique genetic signatures in any specific patient and potentially lead to greater therapeutic benefits."

The strategy could also apply to other medical conditions related to the central nervous systemnot just brain tumors.

"Degenerative neurological diseases or even psychiatric conditions could potentially be the therapeutic candidates for this multiplexed delivery platform," Yu said.

Before scientists can translate this proof-of-concept research to humans, they will need to continue refining the nanomedicine platform and evaluating its long-term safety. Still, the findings from this new research provide insight for further investigation.

"Nanomedicine provides a unique opportunity to advance a therapeutic strategy for a disease without a cure. By effectively targeting brain tumor initiating stem cells responsible for cancer recurrence, this approach opens up novel translational approaches to malignant brain cancer," Lesniak summed up.

Explore further: Cold virus, stem cells tested to destroy deadly brain cancer

More information: Dou Yu et al, Multiplexed RNAi therapy against brain tumor-initiating cells via lipopolymeric nanoparticle infusion delays glioblastoma progression, Proceedings of the National Academy of Sciences (2017). DOI: 10.1073/pnas.1701911114

Early phase Northwestern Medicine research has demonstrated a potential new therapeutic strategy for treating deadly glioblastoma brain tumors.

To enhance the visibility of organs as they are scanned with magnetic resonance imaging (MRI), patients are usually injected with a compound known as a contrast agent before going into the scanner. The most commonly used ...

From checkout counters at supermarkets to light shows at concerts, lasers are everywhere, and they're a much more efficient light source than incandescent bulbs. But they're not cheap to produce.

A world-first non-destructive quality control method from the National Physical Laboratory (NPL) has enabled Oxford Instruments to commercialise wafer-scale fabrication technology for 2-D material MoS2.

Although scientists have for decades been able to synthesize nanoparticles in the lab, the process is mostly trial and error, and how the formation actually takes place is obscure. However, a study recently published in Nature ...

In today's increasingly powerful electronics, tiny materials are a must as manufacturers seek to increase performance without adding bulk.

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Nanomedicine opens door to precision medicine for brain tumors - Phys.Org

Kolon Life Science filed for a Biologics License Application (BLA) for Invossa-K Inj. with MFDS in August 2016 based on efficacy results from its Phase III clinical trials conducted at 12 major university hospitals in Korea. Invossa-K Inj. will be manufactured by Kolon Life Science, and marketed by Mundipharma and Kolon Pharmaceuticals for the Korean market.

"We are excited to launch the world's first cell and gene therapy for knee osteoarthritis and potentially the world's first disease-modifying osteoarthritis drug (DMOAD)," stated Mr. Woosok Lee, CEO of TissueGene. "This approval is the first critical step towards a global launch for this innovative, novel cell and gene therapy technology that will address one of the most pressing unmet medical need affecting millions of people suffering from osteoarthritis worldwide."

Invossa is a first-in-class cell and gene therapy drug designed to conveniently and effectively treat osteoarthritis of the knee through a single intra-articular injection. Clinical trials completed in Korea and on-going in the US have demonstrated pain relief, increased mobility, and potentially game-changing improvements in joint structure offering substantial relief and convenience for osteoarthritis patients who would otherwise be in need of surgery.

Through its national US Phase III clinical trials, TissueGene will be using the results to seek a DMOAD designation for Invossa from the US Food and Drug Administration (FDA), potentially making Invossa the first and only cell and gene therapy for osteoarthritis of the knee.

In November last year, Kolon Life Science signed a license agreement with Mitsubishi Tanabe Pharmaceutical Corporation, and Mitsubishi Tanabe Pharma is proceeding with the preparation of clinical trials through its exclusive development and commercialization rights in Japan.

TissueGene, Inc. TissueGene, Inc., is a Maryland-based regenerative medicine company specializing in cell and gene therapy. TissueGene's lead product is Invossa, an allogeneic, cell and gene therapy for osteoarthritis of the knee that is preparing for Phase III clinical trials in the US pursuant to a Special Protocol Assessment (SPA) agreement reached with the U.S. Food and Drug Administration. Information about the trials can be found at the NIH registry, ww.clinicaltrials.gov. For additional information about TissueGene, Inc., please visit http://www.tissuegene.com.

Kolon Life Science Kolon Life Science has been developing innovative cell and gene therapies including Invossa K Inj., the world's first cell-mediated gene therapy for osteoarthritis, since its founding in 2000. In addition to its biopharmaceuticals business, the company is also engaged in the business of providing active pharmaceuticals ingredients (API), eco-chemicals including antimicrobials for personal-care and industrial applications, as well as water-treatment solutions. For more information, please visit http://www.kolonls.co.kr/eng

View original content:http://www.prnewswire.com/news-releases/korea-approves-the-worlds-first-cell-and-gene-therapy-for-knee-osteoarthritis-300486969.html

SOURCE TissueGene, Inc.

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Korea Approves the World's First Cell and Gene Therapy for Knee Osteoarthritis - PR Newswire (press release)

Published Wednesday, Jul. 12, 2017, 7:35 am

Front Page Events New way to shut off genes speeds battle against genetic diseases

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Gene editing using the CRISPR system is transforming genetic research and could be poised to transform the treatment of genetic diseases such ashemophiliaandcystic fibrosis. Now aUniversity of Virginia School of Medicineresearcher has found a safer and highly efficient way to use the system to silence genes.

Mazhar Adli, PhD, and his team have developed a technique to prevent genes from carrying out their function without causing the extensive DNA damage the current approach requires. This is important because silencing genes allows scientists to understand what individual genes do and identify the ones that cause disease. Gene silencing also may one day let doctors better treat and even cure genetic diseases with abnormal gene activity.

Our genes the blueprints for life are segments of the long double strands of DNA in our cells. To silence genes using the current approach, the CRISPR system cuts both strands. Doing this too often causes the cells to die a major limitation for CRISPR. Even cuts that are not fatal to cells can have unintended effects that result from the bodys efforts to repair the broken DNA.

Adlis approach, on the other hand, avoids cutting the DNA altogether. Instead, it takes advantage of the fact that DNA is made of four main building blocks: cytosine, adenine, guanine and thymine. Adlis method lets scientists use CRISPR to convert one building block into another to artificially create what are called stop codons the off switches that naturally occur at the end of genes. Turn cytosine into thymine, for example, and the whole gene is silenced, meaning there is no protein production from that gene.

We found around 17,000 genes we can target this way, and, as you know, we have roughly 20,000 genes, Adli explained.So a very large fraction of the genome we can target with this CRISPR stop approach.

He noted the benefits of the new techniques genetic alchemy: Its about as efficient as what we used previously, he said. But more importantly, it is safer. It doesnt cause cell death. Further, the new approach is compatible with high-throughput screening that lets scientists do their research much more quickly. You can delete basically every single gene [of certain types] in a population of cells and then watch the entire population to see whats going to happen to them, he said. So with a single experiment you can interrogate the function of thousands and thousands of genes.

Adli, of UVAs Department of Biochemistry and Molecular Genetics, is making his technique available for free to scientists around the world. Any lab that uses CRISPR should have the capability to use it, he said. He expects it will initially be used for research in labs like his, but, with the first human tests of gene editing now beginning, that may change in the future.

Adlis team has outlined the new technique in an article in the scientific journalNature Methods. The article was written by Cem Kuscu, Mahmut Parlak, Turan Tufan, Jiekun Yang, Karol Szlachta, Xiaolong Wei, Rashad Mammadov and Adli.

The work was supported by a V Scholar award from the V Cancer Research Foundation and a pilot project award from the UVA Cancer Center.

Originally posted here:
New way to shut off genes speeds battle against genetic diseases - Augusta Free Press

Breathing in a New Gene Therapy to treat Pulmonary Hypertension

Newswise (New York, NY July 12, 2017) Mount Sinai has partnered with Theragene Pharmaceuticals, Inc. to advance a novel airway-delivered gene therapy for treating pulmonary hypertension (PH), a form of high blood pressure in blood vessels in the lungs that is linked to heart failure. If the therapy succeeds in human clinical trials, it will provide patients for the first time with a way to reverse the damage caused by PH.

This gene therapy technique comes from the research of Roger J. Hajjar, MD, Professor of Medicine and Director of the Cardiovascular Research Center at the Icahn School of Medicine at Mount Sinai, and has been proven effective in rodent and pig animal models. PH is a deadly disease that disproportionately affects young adults and women; 58 percent of cases are found in young adults and 72 percent are women. There is currently no effective cure for PH, and about 50 percent of people who are diagnosed will die from the disease within five years.

PH is a rare (15-50 cases per million people), rapidly progressing disease that occurs when blood pressure is too high in vessels leading from the heart to the lungs. The high pressure is caused by abnormal remodeling of the lung blood vessels, characterized by a proliferation of smooth muscle cells and a thickening and narrowing of these vessels, and can lead to failure of the right ventricle of the heart and premature death. Abnormalities in calcium cycling within the vascular cells play a key role in the pathophysiology of pulmonary hypertension, along with deficiencies in the sarcoplasmic reticulum calcium ATPase pump (SERCA2a) protein which regulates intracellular calcium within these vascular cells and prevents them from proliferating within the vessel wall. Downregulation of SERCA2a leads to the proliferative remodeling of the vasculature. This gene therapy, delivered via an inhaled aerosolized spray, aims to increase the expression of SERCA2a protein, and has been shown in rodents and pigs to improve heart and lung function, as well as reduce and even reverse cellular changes caused by PH.

This is a devastating disease, and our work in collaboration with many laboratories across the country has allowed us to identify a specific molecular target and use gene therapy to improve cardiovascular and lung parameters in experimental models of PH. We look forward to starting first-in-human studies using this approach in affected patients, said Dr. Hajjar, the senior author of the studies, highlighting that clinical trials will be underway in the next two years.It may take several years before a product is commercially available for PH patients.

We are excited about the potential for SERCA2a gene therapy as a new modality in treating this serious disease, said Jon Berglin, Chief Executive Officer of Theragene Pharmaceuticals, Inc. We look forward to develop and advance this promising product into the clinic.

This represents another critical advancement in a potentially transformative therapeutic breakthrough by Mount Sinai scientists, demonstrating our commitment to improving health outcomes. We are thrilled to be working with Theragene Pharmaceuticals, and continue to strengthen our expertise in partnering health care innovations with industry, said Erik Lium, PhD, Senior Vice President of Mount Sinai Innovation Partners, the commercialization arm of the Icahn School of Medicine at Mount Sinai.

About Mount Sinai Innovation Partners (MSIP)MSIP is responsible for driving the real-world application and commercialization of Mount Sinai discoveries and the development of research partnerships with industry. The aim is to translate these innovations into healthcare products and services that benefit patients and society. MSIP is responsible for the full spectrum of commercialization activities required to bring the Icahn School of Medicine and the Mount Sinai Health Systems inventions to life. These activities include evaluating, patenting, marketing and licensing new technologies, engaging commercial and non-profit relationships for sponsored research, material transfer and confidentiality, as well as fostering an ecosystem of entrepreneurship within our research and health system communities. For more information, visit http://www.ip.mountsinai.org.

About Theragene Pharmaceuticals, Inc.Theragene is a biopharmaceutical company developing cutting-edge science for the treatment of debilitating diseases. The Companys diverse portfolio consists of preclinical and clinical oncology and cardiology platforms utilizing next generation gene therapy and immunotherapy methods.

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Breathing in a New Gene Therapy to Treat Pulmonary Hypertension - Newswise (press release)