Top AI companies join hands to discover novel drugs for DMD

Insilico Medicine a leader in artificial intelligence for drug discovery, biomarker development and aging research, announced a research collaboration agreement with A2A Pharmaceuticals, Inc. A2A is a biotechnology company headquartered in New York and focused on development of novel drugs for unmet needs in oncology, drug resistant bacterial infections, and other life threatening diseases.

The companies agreed to join forces to create a new company called "Consortium.AI" , which will apply the latest advances in AI to discovery of novel small molecules for the Duchenne Muscular Dystrophy (DMD) and other rare orphan diseases. Computationally pre-optimized new drug candidates have already been designed for targets validated through Insilico's artificial intelligence system A2A Pharmaceuticals will assume the management of the new company, provide the development expertise for the newly discovered compounds and should be contacted for the licensing of the compounds.

https://www.news-medical.net/news/20180720/Top-AI-companies-join-hands-to-discover-novel-drugs-for-DMD.aspx

A dietary supplement derived from glucose increases muscle-force production in the Duchenne muscular dystrophy (DMD) mouse model by 50% in ten days, according to a study conducted by researchers from Université Laval's Faculty of Medicine and Centre hospitalier universitaire (CHU) de Québec Research Centre-Université Laval. The results, which were recently published in the scientific periodical The FASEB Journal, pave the way for a clinical study to test the treatment's effectiveness on humans.

https://www.news-medical.net/news/20180630/Glucose-based-dietary-supplement-increases-muscle-force-in-Duchenne-muscular-dystrophy-mouse.aspx

MSU students develop exoskeleton app for patient with Duchenne muscular dystrophy

MSU students develop exoskeleton app for patient with Duchenne muscular dystrophy: "Zach Smith has Duchenne muscular dystrophy, a genetic disorder marked by progressive muscle degeneration. His lack of muscle control and being in a wheelchair made him a prime candidate for a computer-controlled exoskeleton arm.

Talem Technologies gave him an X-Ar exoskeleton that allows him to do many daily tasks, such as drink from a glass and turn on a light switch. Keeping everything level however, proved to be a challenge. That's where a team of Michigan State University students stepped in.

Working with the Talem Technologies and Urban Science, a Detroit-based consulting firm, a College of Engineering team of computer science students developed an app that allows Smith, who lives in Orlando, Florida, to maximize the use of his bionic-looking arms.

Mustafa Jebara, Dane Rosseter, Samantha Oldenburg, Alex Wuillame and Shun Yan created Mobile Maestro, available for iPhones and Android devices, that conveniently puts the exoskeleton's controls on the user's phone."



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Ezutromid Shows Potential in DMD | Medpage Today

Ezutromid Shows Potential in DMD | Medpage Today: "LOS ANGELES -- The investigative utrophin modulator ezutromid showed promise in treating Duchenne muscular dystrophy, according to a interim analysis of an open-label, phase II clinical trial.

Ezutromid appeared to have significantly deceased inflammation in the calf muscle of Duchenne patients after 24 weeks, reported Anne Heatherington, PhD, of Summit Therapeutics in Oxford, England, and colleagues at the American Academy of Neurology annual meeting."



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Research establishes use of chimeric cells as potential therapy for Duchenne muscular dystrophy

Research establishes use of chimeric cells as potential therapy for Duchenne muscular dystrophy: "Cells made by fusing a normal human muscle cell with a muscle cell from a person with Duchenne muscular dystrophy --a rare but fatal form of muscular dystrophy -- were able to significantly improve muscle function when implanted into the muscles of a mouse model of the disease. The findings are reported by researchers from the University of Illinois at Chicago in the Stem Cell Review and Reports."



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Summit’s DMD drug cuts muscle inflammation in POC trial | FierceBiotech

Summit’s DMD drug cuts muscle inflammation in POC trial | FierceBiotech: "Summit Therapeutics has posted further data from the ongoing phase 2 trial of ezutromid. The latest analysis links the utrophin modulator to a statistically significant decline in muscle inflammation in boys with Duchenne muscular dystrophy (DMD).

Oxford, U.K.-based Summit generated the data on muscle inflammation by measuring MRI transverse relaxation time T2 (MRS-T2). The technique quantifies the breakdown and inflammation of muscle, making it a useful tool for assessing the progression of DMD. MRS-T2 values rise as DMD progresses but can be brought down by steroids.

The Summit data suggest ezutromid may drive reductions in MRS-T2 beyond those achieved by the use of steroids alone. All 38 participants in the proof-of-concept trial were on stable steroid regimes. After 24 weeks of twice-daily ezutromid doses, MRS-T2 decreased by 0.861 milliseconds, on average."



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Cell therapy for heart disease caused by muscular dystrophy also improves limb strength, study shows

Cell therapy for heart disease caused by muscular dystrophy also improves limb strength, study shows: "Injections of cardiac progenitor cells help reverse the fatal heart disease caused by Duchenne muscular dystrophy and also lead to improved limb strength and movement ability, a new study shows.

The study, published today in Stem Cell Reports, showed that when researchers injected cardiosphere-derived cells (CDCs) into the hearts of laboratory mice with muscular dystrophy, heart function improved along with a marked increase in exercise capacity.

"We unexpectedly found that treating the heart made the whole body better," said Eduardo Marbán, MD, PhD, director of the Smidt Heart Institute and the investigator who developed the cardiosphere-derived cell technology used in the study. "These basic findings, which have already been translated to clinical trials, rationalize why treating the heart may also benefit skeletal muscle function in boys and young men with Duchenne.""



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Robotic arm developed for Duchenne patients -- ScienceDaily

Robotic arm developed for Duchenne patients -- ScienceDaily: "Researchers from the University of Twente's MIRA research centre, together with the VUmc, TU Delft and the Radboud umc, have developed the A-Gear: a robotic arm that can support the daily activities of people suffering the muscular disease Duchenne Muscular Dystrophy. They recently put the final touches to the first prototype for a discreet, body-connected support aid that can be worn under the clothing and that can support independent operation of the arm during important daily activities."



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New CRISPR gene-editing technique can correct majority of mutations that cause DMD

New CRISPR gene-editing technique can correct majority of mutations that cause DMD: "Scientists have developed a CRISPR gene-editing technique that can potentially correct a majority of the 3,000 mutations that cause Duchenne muscular dystrophy (DMD) by making a single cut at strategic points along the patient's DNA, according to a study from UT Southwestern Medical Center.

The method, successfully tested in heart muscle cells from patients, offers an efficient alternative to the daunting task of developing an individualized molecular treatment for each gene mutation that causes DMD. It also opens up possible new treatment approaches for other diseases that have thus far required more intrusive methods to correct single-gene mutations.

Scientists say the new strategy enhances the accuracy for surgical-like editing of the human genome, correcting mistakes in the DNA sequence that cause devastating diseases like DMD, a deadly condition caused by defects in the dystrophin gene. Normally, the dystrophin protein helps strengthen muscle fibers."



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Ezutromid Reduced Muscle Damage in DMD Patients, Interim Data Shows

Ezutromid Reduced Muscle Damage in DMD Patients, Interim Data Shows: "At 24-weeks, muscle damage and utrophin protein were measured through muscle biopsies. Muscle biopsies were taken before treatment, and 24 patients had their second biopsy after 24 weeks of treatment. The remaining patients will have their second one by the end of the study (48 weeks).

Muscle damage was significantly reduced after 24 weeks, based on a 23% decrease in the levels of developmental myosin, compared with baseline. The decrease of developmental myosin was observed in about 64% of patients (14 patients), with five of them showing more than 40% reduction."



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SLU scientists provide promising approach in designing new drugs for DMD

SLU scientists provide promising approach in designing new drugs for DMD: "In a paper published in the Nature journal Scientific Reports, Saint Louis University (SLU) researchers report that a new drug reduces fibrosis (scarring) and prevents loss of muscle function in an animal model of Duchenne muscular dystrophy (DMD), providing a promising approach in designing new medications for those suffering from DMD."



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New initiative aims to improve chances of medicines receiving positive HTA decisions

New initiative aims to improve chances of medicines receiving positive HTA decisions: "A unique initiative launched today by muscular dystrophy charity Duchenne UK is set to radically simplify the way necessary evidence is generated for submissions made to health technology appraisals (HTA) bodies such as the National Institute for Health and Care Excellence (NICE) and the Haute Autorité de Santé (HAS).

Launched today at the 8th World Orphan Drug Congress in Barcelona, this innovative collaboration – entitled HERCULES –  will focus on medicines used to treat Duchenne muscular dystrophy (DMD).  It will be the first time a group of pharmaceutical industry partners have ever collaborated to generate, share and align disease level data across an entire condition.

Although this new initiative focuses on DMD, it paves the way for similar approaches in other rare diseases, and has the potential to improve the chances of medicines in rare and orphan diseases receiving positive HTA decisions.  This unique collaborative approach has been welcomed by a range of industry health economists, as well as former NICE personnel."



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Summit completes initial dosing trials on Duchenne muscular dystrophy drug - Pharmaceutical Business Review

Summit completes initial dosing trials on Duchenne muscular dystrophy drug - Pharmaceutical Business Review: "Summit Therapeutics has completed the half-way stage of dosing of its Duchenne Muscular Dystrophy (DMD) candidate ezutromid in a 48-week phase 2 trial, dubbed PhaseOut DMD.

The UK-based Summit is likely to report results from the initial 24-week dosing period in the first quarter of 2018 which are anticipated to contain data from muscle biopsies, MRI and functional tests, and also safety data.

Summit Therapeutics chief operating officer and medical officer David Roblin said: "In the interim data readout, we aim to show a positive change following ezutromid treatment in muscle structure and health through the evaluation of muscle biopsies.

“This could potentially provide the first evidence of proof of mechanism for utrophin modulators in patients and give hope to those living with DMD.”"



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Clinical trial results could lead to treatment of heart disease in Duchenne muscular dystrophy patients

Clinical trial results could lead to treatment of heart disease in Duchenne muscular dystrophy patients: "After boys and young men with Duchenne muscular dystrophy received cardiac progenitor cell infusions, medical tests indicated that the patients' hearts appeared improved, results from a new study show. Patients in the study also scored higher on arm strength tests after receiving the cell infusions.

Results from the HOPE Duchenne randomized clinical trial of 25 patients were presented today at the American Heart Association Scientific Sessions in Anaheim. The cardiac progenitor cells administered to the patients were contained in CAP-1002, the lead investigational therapy under development at Capricor Therapeutics, Inc. (NASDAQ: CAPR)."



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DMD Study Shows Protein Utrophin Reduces Mitochondria Damage

DMD Study Shows Protein Utrophin Reduces Mitochondria Damage: "In recent years scientists have learned that the protein utrophin can substitute for the dystrophin protein that is missing in muscular dystrophy.

Researchers have now discovered that utrophin can reduce damage to energy-producing cell components known as mitochondria, whose deterioration is linked to MD. The reduction in damage improved the muscle health of mice with Duchenne muscular dystrophy.

Summit Therapeutics and collaborators from the University of Oxford presented the findings at the 15th Action Duchenne International Conference 2017 in Birmingham, England, Nov. 10-12."



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Duchenne MD Exon-skipping Therapy Is Now in Phase 1 Trial

Duchenne MD Exon-skipping Therapy Is Now in Phase 1 Trial: "Wave Life Sciences has started a Phase 1 clinical trial of a treatment for a gene malfunction linked to the most common form of Duchenne muscular dystrophy."



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Summit Highlights Utrophin Modulation as a Potential Universal Treatment Option in DMD at Action Duchenne Nasdaq:SMMT

Summit Highlights Utrophin Modulation as a Potential Universal Treatment Option in DMD at Action Duchenne Nasdaq:SMMT: "Summit Therapeutics plc (NASDAQ: SMMT, AIM: SUMM) the drug discovery and development company advancing therapies for Duchenne muscular dystrophy ('DMD') and Clostridium difficile infection, announces that the Company and collaborators from the University of Oxford will highlight the potential of utrophin modulation as a universal treatment for DMD in several presentations at the 15th Action Duchenne International Conference 2017, taking place November 10-12, 2017, in Birmingham, UK. The conference brings together families living with Duchenne and Becker muscular dystrophy, along with experts and industry members aiming to treat these diseases.

The Company's scientific adviser and co-founder, Professor Kay Davies from the University of Oxford, will present recently published preclinical data highlighting the benefits of utrophin modulation on muscle health in animal models of DMD. The data show that continuously expressing utrophin in a dystrophin-deficient background can reduce mitochondrial aberration and oxidative stress. Aberrant mitochondria drive, in part, oxidative stress, which is a contributing factor to muscle damage in DMD. In addition, Professor Davies is also expected to discuss previously published preclinical data of utrophin modulators, including ezutromid, that have shown these compounds' potential to prevent molecular disease, leading to functional improvements in mdx mice."



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DMD Therapy Exondys 51 Gets a Hand from Immune Cells, Study Reports

DMD Therapy Exondys 51 Gets a Hand from Immune Cells, Study Reports: "The newly approved Duchenne muscular dystrophy therapy Exondys 51 (eteplirsen) stays in immune cells several days after leaving the blood, continuing to help regenerate muscle fibers, according to a study in mice."



The newly approved Duchenne muscular dystrophy therapy Exondys 51 (eteplirsen) stays in immune cells several days after leaving the blood, continuing to help regenerate muscle fibers, according to a study in mice.

CRISPR-Gold corrects gene mutations in mice with Duchenne muscular dystrophy

CRISPR-Gold corrects gene mutations in mice with Duchenne muscular dystrophy: "Scientists at the University of California, Berkeley, have engineered a new way to deliver CRISPR-Cas9 gene-editing technology inside cells and have demonstrated in mice that the technology can repair the mutation that causes Duchenne muscular dystrophy, a severe muscle-wasting disease. A new study shows that a single injection of CRISPR-Gold, as the new delivery system is called, into mice with Duchenne muscular dystrophy led to an 18-times-higher correction rate and a two-fold increase in a strength and agility test compared to control groups."



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Summit Therapeutics PLC teams up with cTAP as it looks to accelerate development of its DMD drug

Summit Therapeutics PLC teams up with cTAP as it looks to accelerate development of its DMD drug: "Drug discoverer Summit Therapeutics PLC (LON:SUMM; NASDAQ:SMMT) has teamed up with the Collaborative Trajectory Analysis Project to try and accelerate the development of its lead Duchenne muscular dystrophy candidate.

Summit’s utrophin modulator for the treatment of DMD – ezutromid – is currently in a phase II clinical trial, with data from the 48-week study expected in the third quarter of 2018.

But the dual-listed firm hopes that by partnering with cTAP, it can potentially adapt and improve the overall clinical process."



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