Hydatidiform Mole

Researchers have designed a new genetic therapy that could alleviate debilitating giant moles in a rare skin condition. Researchers at the Francis Crick Institute, UCL Great Ormond Street Institute for Child Health and Great Ormond Street Hospital for Children (GOSH) have designed a new genetic therapy that could alleviate debilitating giant moles in a rare skin condition. In the future, the treatment could potentially be used to reverse the giant moles, and therefore reduce the risk of affected children and adults from developing cancer. It could also potentially reverse other more common types of at-risk moles as an alternative to surgery. Small skin moles are common in the population, but in congenital melanocytic naevus syndrome (CMN), children are born with up to 80% of their body covered in big, painful or itchy moles, caused by mutations acquired in the womb. These moles can sometimes develop into a severe type of cancer called melanoma. Published today in the Journal of Investigate Dermatology, the researchers silenced a gene called NRAS, which is mutated in the cells in these moles, in cells in a dish and in mice. NRAS belongs to a group of genes (RAS genes) that, when mutated, can cause moles, and can predispose to cancer. The team used a genetic therapy called silencing RNA, which silences the mutated NRAS in mole skin cells. The therapy was delivered in special particles directly to mole cells. They gave injections containing the therapy to mice with CMN, which reduced the silenced the NRAS gene after just 48 hours. They also tested it in cells and whole skin sections from children with CMN. Silencing the gene triggered the mole cells to self-destruct. Veronica Kinsler, Principal Group Leader of the Mosaicism and Precision Medicine Laboratory at the Crick, Professor of Paediatric Dermatology and Dermatogenetics at GOSH/UCL, and NIHR Research Professor, said: "CMN is physically and mentally challenging for children and adults living with this condition and for their families. These results are very exciting, as not only does the genetic therapy trigger self-destruction of the mole cells in the lab, but we have managed to deliver it into the skin in mice. This results suggest that the treatment in future could potentially reverse moles in people, however more testing will be needed before we can give it to patients. "We are very grateful to our patients at Great Ormond Street Hospital, who have been actively participating over many years to help us produce this new potential therapy. After more studies, we hope the therapy can soon enter clinical trials in people." This research was funded by the National Institute for Health and Care Research (NIHR), Caring Matters Now Charity and Patient Support Group, LifeArc and the NIHR Great Ormond Street Hospital Biomedical Research Centre. Jodi Whitehouse, CEO of Caring Matters Now, who helped to fund the research, said: "This breakthrough in finding a treatment for CMN could transform the lives of the families we support living with CMN. As someone who was born with CMN covering 70% of my body and having undergone 30+ operations in my childhood to try and remove the CMN because of the fear of melanoma, with no success, this news is awe-inspiring and exciting. It brings real hope to the lives of those living with CMN." Catriona Crombie, Head of Rare Disease at LifeArc, said: "This work is part of our commitment to improving the lives of people living with rare diseases, by investing in promising research and helping scientists to overcome translational research barriers. If successful, we hope to see human clinical trials for this therapy within the next few years." The researchers have been working closely with the Crick's Translation team to develop the technology towards patient benefit. This has included securing translational funds from LifeArc, to carry out more research in mice to understand how the treatment works over a longer period.

end of relations(special f/x,made from my images) Kuzma/Getty Images/iStockphoto Under terms of the deal, Aurobindo would pay $900 million upfront with an additional $100 million in milestone payments. However, in a filing with the Securities and Exchange Commission, the company announced it had terminated that agreement. Generic drugmaker Aurobindo Pharma has called off its $1 billion acquisition plans for multiple assets owned by Sandoz after failing to secure approval from the Federal Trade Commission. India-based Aurobindo struck a deal with Sandoz, a division of Swiss pharma giant Novartis, in 2018 for 300 dermatology and generic drug assets. Under terms of the deal, Aurobindo would pay $900 million upfront with an additional $100 million in milestone payments. However, in a filing with the Securities and Exchange Commission this week, the company announced it had terminated that agreement. “Aurobindo today announced the mutual agreement with Sandoz Inc. to terminate the agreement to buy the Sandoz US generic oral solids and dermatology businesses from Sandoz Inc. This decision was taken as approval from the US Federal Trade Commission for the transaction was not obtained within anticipated timelines,” the company said in a filing on Thursday, according to LiveMint . Aurobindo was the only company based in India that bid on the Sandoz products. At the time the deal was announced, N. Govindarajan, managing director of Aurobindo, said the acquisition of the products was in line with the company’s strategy to “grow and diversify” its business in the United States. In addition to the products, Aurobindo was set to acquire manufacturing facilities in Wilson, North Carolina and Hicksville and Melville, New York. If the deal had gone through, Aurobindo said at the time the proposition was struck, it would have become the second-largest generic drug company in the U.S. based on the number of prescriptions, LiveMint reported. The deal would have also propelled Aurobindo to become the second-largest dermatology company in the United States for both generic and branded dermatology products. In February, Aurobindo won approval from the U.S. Food and Drug Administration for its Abbreviated New Drug Application for multiple doses of Trazodone Hydrochloride tablets, a treatment for major depressive disorder. Also, the company won approval from the FDA for Oxycodone Hydrochloride Oral Solution, an opioid agonist indicated for the management of pain severe enough to require an opioid analgesic, as well as methotrexate tablets, which are used for multiple indications, including severe, active, rheumatoid arthritis and the treatment of gestational choriocarcinoma, chorio-adenoma destruens and hydatidiform mole. The sale was orchestrated as Novartis and Sandoz were reprioritizing their businesses. As a result of the termination of the agreement with Aurobindo, Sandoz will continue to operate those assets as part of its U.S. business, Novartis said in a brief statement .