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Category Archives: Muscular Dystrophy Treatment

Santhera’s Raxone Receives First Positive EAMS Scientific … – GlobeNewswire (press release)

Posted: June 25, 2017 at 9:44 pm

June 22, 2017 01:23 ET | Source: Santhera Pharmaceuticals Holding Ltd

multilang-release

Liestal, Switzerland, June22, 2017 – Santhera Pharmaceuticals (SIX:SANN) announces that the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted Raxone (idebenone) a positive scientific opinion through the Early Access to Medicines Scheme (EAMS) for patients with respiratory function decline not taking glucocorticoids in Duchenne Muscular Dystrophy (DMD).

The aim of the EAMS is to provide patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorization when there is a clear unmet medical need.1 The MHRA decision allows patients with DMD, who meet criteria defined under this scheme, to gain access to Raxone, an investigational medicinal product currently under review for DMD for Marketing Authorization by the European Medicines Agency (EMA).

Under the EAMS, and as shown in the public assessment report,2 Raxone is indicated for slowing the decline of respiratory function in patients with DMD from the age of 10 years who are currently not taking glucocorticoids. The decline of respiratory function must be confirmed by repeated measurements prior to initiation of treatment. Raxone can be used in patients previously treated with glucocorticoids or in patients in whom glucocorticoid treatment is not tolerated or is considered inadvisable.

“We’re proud to receive the positive EAMS scientific opinion for Raxone in the UK and to have our lead compound designated as a promising innovative medicine, the first for a drug intended for the treatment of DMD,” said Thomas Meier, PhD, CEO of Santhera. “This decision allows patients with DMD to receive treatment for respiratory function decline who otherwise would not have access to such treatment options.”

“This is excellent news for patients with respiratory decline in Duchenne muscular dystrophy,” said Janet Bloor, Chair of the Board of Trustees at Action Duchenne. “The need for new treatments in DMD is very great and the EAMS can help to accelerate access for patients. Action Duchenne was pleased to provide advice during the development of the EAMS program and we are delighted to see this first positive opinion in DMD.”

About the UK Early Access to Medicines Scheme (EAMS) The UK’s industry-sponsored EAMS aims to give patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorization when there is a clear unmet medical need. Under the scheme, the MHRA provides a scientific opinion on the benefit/risk balance of the medicine, based on the data available when the EAMS submission was made. The opinion lasts for a year and can be renewed. The scheme is voluntary and the opinion from MHRA does not replace the normal licensing procedures for medicines.

About Santhera Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative pharmaceutical products for the treatment of orphan mitochondrial and neuromuscular diseases. Santhera’s lead product Raxone (idebenone) is authorized in the European Union, Norway, Iceland and Liechtenstein for the treatment of Leber’s hereditary optic neuropathy (LHON). For Duchenne muscular dystrophy (DMD), Santhera has filed a Marketing Authorization Application in the European Union and Switzerland for DMD patients with respiratory function decline who are not taking glucocorticoids. In collaboration with the U.S. National Institute of Neurological Disorders and Stroke (NINDS) Santhera is developing Raxone in a third indication, primary progressive multiple sclerosis (PPMS), and omigapil for congenital muscular dystrophy (CMD), all areas of high unmet medical need. For further information, please visit the Company’s website http://www.santhera.com.

Raxone is a trademark of Santhera Pharmaceuticals.

For further information, contact: Thomas Meier, PhD, Chief Executive Officer Christoph Rentsch, Chief Financial Officer Phone +41 61 906 89 64 Phone +41 61 906 89 65 [email protected] [email protected]

Sue Schneidhorst, Head Group Communications Phone +41 61 906 89 26 [email protected]

US investor contact US Public Relations contact Hans Vitzthum, LifeSci Advisors, LLC Chris Welsh, Consilium Strategic Communications Phone +1 212 915 2568 Phone +1 646 586 2113 [email protected] [email protected]

European Public Relations contact Amber Fennell, Consilium Strategic Communications Phone +44 20 3709 5700 [email protected]

Disclaimer / Forward-looking statements This communication does not constitute an offer or invitation to subscribe for or purchase any securities of Santhera Pharmaceuticals Holding AG. This publication may contain certain forward-looking statements concerning the Company and its business. Such statements involve certain risks, uncertainties and other factors which could cause the actual results, financial condition, performance or achievements of the Company to be materially different from those expressed or implied by such statements. Readers should therefore not place undue reliance on these statements, particularly not in connection with any contract or investment decision. The Company disclaims any obligation to update these forward-looking statements.

References 1 Medicines and Healthcare products Regulatory Agency Patient safety and Marketing authorisations, variations and licensing guidance. Available at: https://www.gov.uk/guidance/apply-for-the-early-access-to-medicines-scheme-eams (June 2017) 2 Public assessment report. Available at https://www.gov.uk/government/publications/early-access-to-medicines-scheme-eams-scientific-opinion-raxone-to-treat-the-decline-of-respiratory-function-in-patients-with-duchenne-muscular-dys

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Santhera’s Raxone Receives First Positive EAMS Scientific … – GlobeNewswire (press release)

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Potential leukemia drug better aimed at muscular dystrophy, NIH says – Patient Daily

Posted: at 9:44 pm

A drug formerly meant for cancer treatment may be better used to treat muscular dystrophy, according to researchers from the National Institutes of Health (NIH) and the University of Nevada, Reno School of Medicine.

Known as SU9516, the drug was meant to be used to battle leukemia until researchers discovered that it ramped up production of a protein missing from patients with muscular dystrophy, a disease that causes muscles to deteriorate.

Although the drugs positive results have only been confirmed in animal testing, scientists are hoping the discovery will provide the missing link for better muscular dystrophy treatment.

The studys chief author, pharmacology professor Dean Burkin of the University of Nevada, said he thinks SU9516 and other medicines could also treat illnesses other than muscular dystrophy.

Integrin stabilizes muscle structure, and helps stimulate muscle repair and regeneration, Burkin said in the release. If we can artificially increase its production with drugs, we think it can help protect muscle cells from damage.

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Altavista community raises money for local firefighter battling form of … – WSET

Posted: at 4:45 am

David Cox pictured with his three kids when he was working for the Lynchburg Fire Department.

ALTAVISTA, Va. (WSET) — David Cox has been fighting fires and helping save lives for 15 years with the Lynchburg Fire Department.

Cox says it’s been a life-long dream and has always been his passion.

But his wife Bess Cox said a of couple months ago, he received the worst news of his life.

“As of right now he has been diagnosed with Myasthenia Gravis, which is a form of Muscular Dystrophy,” She said.

So now instead of fighting fires, he is fighting for his own life.

Calvary Baptist Church wanted to step in and help the Cox family once word got around their town of Altavista about what he was going through.

Cox and his wife has three kids.

Pastor Seth Peterson decided to put together a basketball tournament and barbecue to raise money for Cox’s medical bills.

He said their goal was to raise $5,000, but they surpassed that goal and raised around $6,500.

Calvary Baptist Church is not even the Cox’s home church, but have a few friends that are members. Pastor Peterson said their church just wanted to help a family that was in need.

“I’ve spent my life giving to everybody else,” Cox said. “I just feel so humble and you know like I just don’t deserve this kind of treatment.”

Truman Cox, who has no relation, decided to come out to the fundraiser because David Cox saved his wife’s life two years ago while on on duty.

“Well he gave to me and I want to pay back,” He said. “So I came out here today to do what I could do to help him and his family.”

“I’m Gonna battle and I will be back to my normal strength… it’s just gonna take me some time, but I have all you people that are rallying behind me and that just give me more momentum to come back stronger,” Cox said.

The Cox family said they are appreciative of everyone that has been helping them in this time of need.

If you you were unable to attend the fundraiser, you can still help the Cox family out by donating to their gofundme page.

All of the money raised will go towards Cox’s medical bills.

“That money has helped us focus on his healthcare and not worry about the bills because you know, he has been without a salary for some time now,” Bess Cox said. “That money has been a blessing because I haven’t had to worry about the house payment… I mean that’s kept us going.”

Although Cox had to quit working for the Lynchburg Fire Department, he is still running calls with the Altavista Volunteer Fire Department.

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New Analyses of Translarna (ataluren) Data from ACT DMD Presented at the 12th Annual European Pediatric … – PR Newswire (press release)

Posted: June 22, 2017 at 2:47 pm

About Duchenne Muscular Dystrophy Primarily affecting males, Duchenne muscular dystrophy (DMD) is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-twenties due to heart and respiratory failure. It is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of skeletal, diaphragm, and heart muscles. Patients with DMD can lose the ability to walk as early as age ten, followed by loss of the use of their arms. DMD patients subsequently experience life-threatening lung complications, requiring the need for ventilation support, and heart complications in their late teens and twenties. It is estimated that a nonsense mutation is the cause of DMD in approximately 13 percent of patients.

About ataluren (Translarna)Ataluren, discovered and developed by PTC Therapeutics, Inc., is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne muscular dystrophy. Translarna, tradename of ataluren, is licensed in the European Economic Area for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older. Ataluren is an investigational new drug in the United States. The development of ataluren has been supported by grants from the Muscular Dystrophy Association; FDA’s Office of Orphan Products Development; National Center for Research Resources; National Heart, Lung, and Blood Institute; and Parent Project Muscular Dystrophy.

About PTC TherapeuticsPTC is a global biopharmaceutical company focused on the discovery, development, and commercialization of novel medicines using our expertise in RNA biology. PTC’s internally discovered pipeline addresses multiple therapeutic areas, including rare disorders and oncology. PTC has discovered all of its compounds currently under development using its proprietary technologies. Since its founding nearly 20 years ago, PTC’s mission has focused on developing treatments to fundamentally change the lives of patients living with rare genetic disorders. The company was founded in 1998 and is headquartered in South Plainfield, New Jersey. For more information on the company, please visit our website http://www.ptcbio.com.

Forward Looking Statements:All statements, other than those of historical fact, contained in this release are forward-looking statements, including statements regarding the future expectations, plans and prospects for PTC; the clinical utility and potential advantages of Translarna (ataluren); and the objectives of management. Other forward-looking statements may be identified by the words “look forward”, “plan,” “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “target,” “potential,” “will,” “would,” “could,” “should,” “continue,” and similar expressions.

PTC’s actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including PTC’s ability to resolve the matters set forth in the Refuse to File letter it received from the FDA in connection with its NDA for Translarna for the treatment of nmDMD, including whether PTC’s filing of the NDA over protest with the FDA will result in a timely or successful review of the NDA, and whether PTC will be required to perform additional clinical and non-clinical trials or analyses at significant cost, which, if successful, could potentially support the approval of the NDA filed over protest or a new NDA submission; the recommendation the advisory committee provides to the FDA for Translarna for the treatment of nmDMD; delays in PTC’s projected regulatory timeline for the NDA; PTC’s ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in the European Economic Area, including whether the European Medicines Agency (EMA) determines in future annual renewal cycles that the benefit-risk balance of Translarna authorization supports renewal of such authorization; PTC’s ability to enroll, fund, complete and timely submit to the EMA the results of Study 041, a randomized, 18-month, placebo-controlled clinical trial of Translarna for the treatment of nmDMD followed by an 18-month open label extension; the eligible patient base and commercial potential of Translarna and PTC’s other product candidates; PTC’s ability to commercialize and commercially manufacture Translarna in general and specifically as a treatment for nmDMD; the outcome of pricing and reimbursement negotiations in those territories in which PTC is authorized to sell Translarna for the treatment of nmDMD; the outcome of ongoing or future clinical studies in Translarna; expectations for regulatory approvals; PTC’s ability to meet existing or future regulatory standards with respect to Translarna; and the factors discussed in the “Risk Factors” section of PTC’s most recent Quarterly Report on Form 10-Q as well as any updates to these risk factors filed from time to time in PTC’s other filings with the SEC. You are urged to carefully consider all such factors.

The forward-looking statements contained herein represent PTC’s views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law.

For More Information:

Investors:Emily Hill+ 1 (908) [email protected]

Media:Jane Baj+1 (908) [email protected]

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/new-analyses-of-translarna-ataluren-data-from-act-dmd-presented-at-the-12th-annual-european-pediatric-neurology-society-congress-300478112.html

SOURCE PTC Therapeutics, Inc.

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New Analyses of Translarna (ataluren) Data from ACT DMD Presented at the 12th Annual European Pediatric … – PR Newswire (press release)

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Sarepta signs another Duchenne gene therapy pact as it aims for wider treatment – FierceBiotech

Posted: at 2:47 pm

Sarepta Therapeutics has penned its second DMD gene therapy pact this year as it announces a tie-up with Frances Genethon, a nonprofit R&D org.

The research collaborationwill see the Franco-American pair jointly develop treatments for Duchenne muscular dystrophy and comes after Sareptas first FDA approval for DMD with its controversial med Exondys 51 (eteplirsen).

RELATED: FDA expert lashes out at ‘worrisome’ Sarepta approval in JAMA

Sarepta is looking to tap into Genethons preclinical microdystrophin gene therapy approach, which can target the majority of patients with DMD. Its current med can only treat certain patients, namely those with the mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13% of the population with DMD.

It is hoping that with new tie-ups, it could produce a gene therapy that could treat many more, if not all, patients with the disease, although this is still some years off. DMD is a rare genetic disorder characterized by progressive muscle deterioration and weakness. The disease primarily affects young boysand occurs in about one out of every 3,600 male infants worldwide.

This builds on the pacts announced at the start of the year at the JPM conference, which saw it sign a deal with the Nationwide Childrens Hospital, which also focuses on the microdystrophin gene therapy program, as well as another form of gene therapy.

An initial phase 1/2a trial for the microdystrophin gene therapy is slated to begin at the end of the year and will be done at Nationwide Childrens. It also penned an exclusive license agreement with Nationwide for their Galgt2 gene therapy program, originally developed by researcher Paul Martin. This early-stage program aims to research a potential surrogate gene therapy approach to DMD, whereby the gene therapy looks to induce genes that make proteins that can perform a similar function as dystrophin. The goal will be to produce a muscle cell that can function normally even when dystrophin is absent, Sarepta said at the time.

Under the terms of its latest collaboration, Genethon will be responsible for the early development work. Sarepta has the option to co-develop Genethons microdystrophin program, which includes exclusive U.S. commercial rights. Financial terms, as is becoming more common with these pacts, have not been disclosed.

RELATED: With Exondys 51 approved, Sarepta chief Ed Kaye to bow out

Our agreement with Genethon strengthens our ongoing commitment to patients and is aligned with our strategy of building the industrys most comprehensive franchise in DMD, said Ed Kaye, Sareptas outgoing chief. This partnership brings together our collective experience in Duchenne drug development and Genethons particular expertise in gene therapy for rare diseases. We look forward to working with Genethon given their knowledge, large infrastructure and state-of the-art manufacturing capabilities to advance next generation therapies for DMD.

Frederic Revah, CEO of Genethon, added: Microdystrophin-based gene therapy is a very promising approach with potential application to a large majority of Duchenne patients. In order to accelerate the development of a treatment, we are very pleased to partner with Sarepta Therapeutics, which has demonstrated commitment and success for innovative therapies for Duchenne muscular dystrophy. This partnership brings together the highly complementary and synergistic expertises of Sarepta and Genethon, to the benefit of the patients.

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Researchers Aim to Repurpose Failed Leukemia Drug to Treat … – Managed Care magazine

Posted: June 17, 2017 at 10:44 am


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Researchers at the National Institutes of Health's National Center for Advancing Translational Sciences (NCATS) and at the University of Nevada Reno School …
A Drug Unsuccessful at Treating Cancer May Successfully Treat DMDAJMC.com Managed Markets Network
Duchenne MD Mice Improve Muscle Function with Former Cancer …Muscular Dystrophy News

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RASRx Receives Orphan Drug Designation from FDA – Business Wire (press release)

Posted: at 10:44 am

NEWPORT BEACH, Calif.–(BUSINESS WIRE)–RASRx announced today that the United States Food and Drug Administration (FDA) has granted an Orphan Drug Designation (ODD) for its compound RASRx1902 for the treatment of Duchenne Muscular Dystrophy. RASRx1902 is an oral therapy that has shown positive effects on muscle function in animal models of Duchenne. In these models, the RASRx1902 has improved muscle strength and regeneration while decreasing muscle inflammation, degeneration, and necrosis.

Through this ODD, RASRx is eligible for financial incentives that can stimulate investment in this program and expedite its regulatory timeline. In concert with CureDuchenne, the company is now transitioning to toxicology studies to advance RASRx1902 towards human clinical trials for the treatment of Duchenne.

RASRxs founders worked with the USC Stevens Center for Innovation, the technology transfer office for the University of Southern California, to exclusively license RASRx1902. Funding by CureDuchenne Ventures and a collaborative U.S. Department of Defense research grant with USC has accelerated the preclinical development of this program. Innovation is a core part of USCs culture and we are proud to support Dr. Kathleen Rodgers and RASRx to facilitate the technology transfer of RASRx1902 for DMD, said Michael Arciero, J.D. Director of Technology Commercialization and New Venture, USC Stevens Center for Innovation.

Duchenne is a fatal genetic disease that causes muscles to degenerate. It impacts approximately 1 in 3,500 boys. Those with Duchenne are usually diagnosed by age 5, lose their ability to walk by 12 and most dont survive their mid-20s. There are limited approved treatments and no cure for Duchenne.

We are passionate about making an impact in areas of unmet needs like Duchenne Muscular Dystrophy, said Kathleen Rodgers, a co-founder of RASRx. This lead program at RASRx takes a mutation-independent approach to halting, and possibly reversing, the degenerative progression of Duchenne. Through this approach, we hope to bring relief to patients with Duchenne, as well as other muscular dystrophies. We have been benefitted immensely from CureDuchennes knowledge of the disease and familiarity with the needs of the patients. This collaboration has been invaluable as we move forward in the development of this promising treatment.

About RASRx

RASRx is developing and commercializing technology licensed from the University of Southern California. The founders of the company are USC researchers with extensive drug development expertise having taken projects through late clinical stage development. RASRx has innovated a leading-edge, systems biology approach to treating the complex muscle pathology of Duchenne muscular dystrophy.

About CureDuchenne

CureDuchenne is the leading nonprofit focused on funding research to find a cure for Duchenne muscular dystrophy, a disease that affects more than 300,000 boys worldwide. CureDuchennes mission is to extend and improve the lives of those affected by Duchenne. With support from CureDuchenne, nine research projects have advanced to human clinical trials. CureDuchenne provides the Duchenne community with resources on the best standard of care through its CureDuchenne Cares program. For more information, please visit CureDuchenne.org and follow us on Facebook, Twitter, Instagram and YouTube.

About the USC Stevens Center for Innovation

The USC Stevens Center for Innovation is the technology transfer office for the University of Southern California and a university-wide resource for USC innovators in the Office of the Provost. The mission at the USC Stevens Center for Innovation is to maximize the translation of USC research into products for public benefit through licensing, collaborations, and the promotion of entrepreneurship and innovation. For more information, visit http://stevens.usc.edu.

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RASRx Receives Orphan Drug Designation from FDA – Business Wire (press release)

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Researchers aim to repurpose former experimental cancer therapy to treat muscular dystrophy – ScienceBlog.com (blog)

Posted: at 10:44 am

Researchers at the National Institutes of Healths National Center for Advancing Translational Sciences (NCATS) and the University of Nevada, Reno School of Medicine (UNR Med) have demonstrated that a drug originally targeted unsuccessfully to treat cancer may have new life as a potential treatment for Duchenne muscular dystrophy (DMD).

The candidate drug, SU9516, represents a different kind of approach for treating DMD, a degenerative muscle disease that usually begins in childhood and has no known cure. It is caused by a faulty gene that leads to progressive muscle weakness, with death often occurring around age 25. Rather than trying to fix or replace the broken gene, SU9516 ramps up the muscle repair process, helping reinforce muscle structure.

NCATS Chemical Genomics Center Acting Branch Chief Juan Marugan, Ph.D., and UNR Med Professor of Pharmacology Dean Burkin, Ph.D., led a team that screened more than 350,000 compounds to find SU9516, which had been previously developed as a treatment for leukemia. The research demonstrated that this compound improved muscle function in both laboratory and animal DMD models. The results, published recently in Molecular Therapy, may provide a promising approach against the disorder and other muscle-wasting conditions.

Those with DMD lack dystrophin, a protein akin to a molecular shock-absorber that helps keep muscle cells intact. Without dystrophin, muscles are fragile and easily injured. Individuals lose muscle strength and the ability to repair damaged muscle tissue. Most die from heart or respiratory problems.

Our findings open the door to develop new drug treatments for DMD, Marugan said.

In earlier research, Burkin, who is senior author of the current study, and his co-workers showed that boosting the levels of a cell structural protein, 71 integrin, in affected muscle cells could alleviate DMD symptoms in a mouse model. In addition, increased amounts of the protein slowed the diseases progress.

Burkin and his UNR Med colleagues collaborated with NCATS researchers, including co-team leaders Marc Ferrer, Ph.D., and Noel Southall, Ph.D., to screen a large collection of compounds for molecules that could increase 71 integrin production in mouse muscle cells grown in the laboratory. The screen revealed that SU9516 raised integrin production and promoted the formation of muscle cells and fibers from DMD muscle stem cells, another important indication of its potential as a drug.

In a series of pre-clinical experiments, the researchers showed that SU9516 increased the production of 71 integrin in human and mouse DMD muscle cells. Subsequent tests found SU9516 improved muscle function and slowed indicators of disease progression.

Burkin suggests that such a drug could be used alone, or in combination, with other therapies yet to be developed. There might be wide ranging applications to other muscle-damaging conditions, like cachexia, a wasting syndrome characterized by weight loss and muscle atrophy that is often seen in the late stages of cancers, and the effects of aging and injury, he noted.

Integrin stabilizes muscle structure, and helps stimulate muscle repair and regeneration, Burkin said. If we can artificially increase its production with drugs, we think it can help protect muscle cells from damage.

The NCATS-UNR Med team plans to work with medicinal chemists to make the molecule more specific for DMD, while also removing the toxic anticancer components, creating a safer version with a goal of future testing in patients.

The work was supported by NCATS through its intramural research program, the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) grants R01AR053697, R01AR064338 and R41AR067014, Cure CMD and Struggle Against Muscular Dystrophy.

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Muscle growth finding may assist with cancer treatment – Medical Xpress

Posted: June 14, 2017 at 9:49 am

June 13, 2017 Dr Craig Harrison and Dr Kelly Walton from the Monash BDI. Credit: Monash University

Monash University’s Biomedicine Discovery Institute (BDI) researchers have collaboratively developed a therapeutic approach that dramatically promotes the growth of muscle mass, which could potentially prevent muscle wasting in diseases including muscular dystrophy and cancer.

The approach, jointly developed with Baker Heart and Diabetes Institute scientists, combines – for the first time – molecules that inhibit three proteins which in turn repress muscle growth.

Published this week in the journal Proceedings of the National Academy of Sciences, the scientists found that inhibiting activin A, activin B and myostatin resulted in skeletal muscle mass increase by as much as 150 per cent in preclinical models.

Myostatin has long been recognised as the body’s major negative regulator of skeletal muscle mass, helping to maintain muscle homeostasis in the body, but creating molecules to target all three related proteins together was a novel approach.

“As a result of the study we can now more precisely regulate – and increase – muscle mass in the setting of disease,” co-lead author from Monash BDI, Dr Craig Harrison, said.

Dr Harrison said the study, the culmination of many years of research with the Baker Institute’s Dr Paul Gregorevic, was aimed mostly at developing a way of preventing muscle loss in the wasting condition cachexia, in cancer.

Dr Harrison said cachexia, observed in the end stages of cancer, was thought to contribute or directly cause 20 to 30 per cent of all cancer-related deaths. Palliative care is currently the only treatment for cancer cachexia. The condition is also seen in other diseases including diabetes, AIDS, and in heart and kidney failure.

The paper showed that the combination treatment could prevent muscle wasting in a cancer cachexia model as well as in muscular dystrophy. It could also potentially be used after clinical development in healthy and in ageing individuals undergoing a slow wasting of muscles, Dr Harrison said.

Activins and myostatin belong to the transforming growth factor- (TGF-) family of proteins, which both researchers have been investigating for a number of years.

Further pre-clinical research is proceeding.

Explore further: One step closer to an ‘exercise pill’

More information: Justin L. Chen et al. Specific targeting of TGF-? family ligands demonstrates distinct roles in the regulation of muscle mass in health and disease, Proceedings of the National Academy of Sciences (2017). DOI: 10.1073/pnas.1620013114

Suppressing production of the protein myostatin enhances muscle mass and leads to significant improvements in markers of heart and kidney health, according to a study conducted in mice. Joshua T. Butcher, PhD, a postdoctoral …

A discovery by Washington State University scientist Dan Rodgers and collaborator Paul Gregorevic could save millions of people suffering from muscle wasting disease.

A new study reveals that tumors release factors into the bloodstream that inhibit the repair of damaged muscle fibers, and that this contributes to muscle loss during cancer wasting. The condition, also called cancer cachexia, …

It is estimated that half of all cancer patients suffer from a muscle wasting syndrome called cachexia. Cancer cachexia impairs quality of life and response to therapy, which increases morbidity and mortality of cancer patients. …

(Medical Xpress) — Cachexia, a syndrome characterized by rapid weight loss and muscle deterioration, is a major cause of death among patients suffering from diseases like cancer, AIDS and chronic infection. In fact, 30 per …

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As a molecular biologist, Kaori Noridomi gets an up-close view of the targets of her investigations. But when she began studying the molecular structures of a rarely diagnosed autoimmune disorder, myasthenia gravis, she decided …

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Researchers at the National Institutes of Health’s National Center for Advancing Translational Sciences (NCATS) and the University of Nevada, Reno School of Medicine (UNR Med) have demonstrated that a drug originally targeted …

Monash University’s Biomedicine Discovery Institute (BDI) researchers have collaboratively developed a therapeutic approach that dramatically promotes the growth of muscle mass, which could potentially prevent muscle wasting …

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Researchers aim to repurpose former experimental cancer therapy to treat muscular dystrophy – National Institutes of Health (press release)

Posted: at 9:49 am


National Institutes of Health (press release)
Researchers aim to repurpose former experimental cancer therapy to treat muscular dystrophy
National Institutes of Health (press release)
… Sciences (NCATS) and the University of Nevada, Reno School of Medicine (UNR Med) have demonstrated that a drug originally targeted unsuccessfully to treat cancer may have new life as a potential treatment for Duchenne muscular dystrophy (DMD).

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Researchers aim to repurpose former experimental cancer therapy to treat muscular dystrophy – National Institutes of Health (press release)

Posted in Muscular Dystrophy Treatment | Comments Off on Researchers aim to repurpose former experimental cancer therapy to treat muscular dystrophy – National Institutes of Health (press release)

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