HERE’S WHERE WE SPEND THE MONEY WE RAISE

The money we raise and spend, on clinical trials, equipment to improve quality of life and the funding of clinical staff in 5 childrens hospitals in Australia, is crucial to the betterment and advancement of the lives of those living with Duchenne.
Please help.

HERE’S WHERE WE SPEND THE MONEY WE RAISE

The money we raise and spend, on clinical trials, equipment to improve quality of life and the funding of clinical staff in 5 childrens hospitals in Australia, is crucial to the betterment and advancement of the lives of those living with Duchenne.
Please help.

SOLID BIOSCIENCES – MEMBRANE STABILISATION THERAPY

Save Our Sons has granted funds to Solid Biosciences – a company which focuses solely on finding treatments for Duchenne – to develop a treatment approach which aims to stabilise the membranes that surround muscles. This approach may be able to protect the muscles of boys (and rare girls) with Duchenne and improve their function.

SOLID BIOSCIENCES – GENE THERAPY

Gene therapy is one of the most promising approaches being developed for Duchenne and in late 2017 three separate clinical trials started in boys with Duchenne. It is hoped that the therapy will be long lasting, but then it is expected that further doses will be needed. However, currently gene therapy can only be given once because the body develops an immune response to it. Save Our Sons is funding two projects looking at ways to allow re-administration of gene therapy.

REVERAGEN – VAMOLORONE (VBP15)

Save Our Sons Duchenne Foundation has been supporting ReveraGen’s development of vamorolone (previously known as VBP15) since 2009. This drug is a potential replacement for the corticosteroids that are commonly prescribed for Duchenne in the hope of maintaining muscle strength for longer. However, the steroids (such as prednisolone) come with a long list of worrying side effects such as stunting of growth, mood changes, weight gain, brittle bones and hormonal problems.

SOLID BIOSCIENCES – MEMBRANE STABILISATION THERAPY

Save Our Sons has granted funds to Solid Biosciences – a company which focuses solely on finding treatments for Duchenne – to develop a treatment approach which aims to stabilise the membranes that surround muscles. This approach may be able to protect the muscles of boys (and rare girls) with Duchenne and improve their function.

SOLID BIOSCIENCES – GENE THERAPY

Gene therapy is one of the most promising approaches being developed for Duchenne and in late 2017 three separate clinical trials started in boys with Duchenne. It is hoped that the therapy will be long lasting, but then it is expected that further doses will be needed. However, currently gene therapy can only be given once because the body develops an immune response to it. Save Our Sons is funding two projects looking at ways to allow re-administration of gene therapy.

REVERAGEN – VAMOLORONE (VBP15)

Save Our Sons Duchenne Foundation has been supporting ReveraGen’s development of vamorolone (previously known as VBP15) since 2009. This drug is a potential replacement for the corticosteroids that are commonly prescribed for Duchenne in the hope of maintaining muscle strength for longer. However, the steroids (such as prednisolone) come with a long list of worrying side effects such as stunting of growth, mood changes, weight gain, brittle bones and hormonal problems.

PHRIXUS – CARMASEAL-MD

Save Our Sons has supported research into Carmaseal-MD (also known as poloxamer 188)- a polymer that shows promise as a treatment for Duchenne due to its ability to stabilise muscle cell membranes.


In Duchenne, muscle cell membranes are fragile and tear easily, it is hoped that Carmaseal-MD will be able to seal these tears and protect the muscle. Preclinical research in animal models has shown that it may be particularly useful in protecting the heart and breathing muscles. Importantly, it could be used for all Duchenne patients no matter what their genetic mutation is.
Our  funding helped fund a small pilot trial in boys/young men with Duchenne to gather the initial evidence of safety and effectiveness prior to starting a larger clinical trial.


The trial involved 10 patients at Cincinnati Children’s Hospital who injected Carmaseal-MD subcutaneously (under the skin) once per day for six months. The participants were non-ambulatory; had early heart failure and impaired respiratory function. The study gathered essential data to further the research in to Phase 2B trials.  The next phase will add to the data on the efficacy of Carmaseal-MD and assessments of effectiveness will focus on heart, lung and upper limb function.

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MILO BIOTECHNOLOGY – FOLLISTATIN GENE THERAPY

Milo’s gene therapy is based on a protein called ‘follistatin’ which it was thought had the ability to increase muscle strength and prevent muscle wasting and fibrosis (formation of scar tissue). Follistatin is naturally made by the body and this gene therapy aimed to increase its production.

The therapy involved using a harmless virus called AAV to deliver the gene containing the instructions for the production of follistatin into the muscle cells.

After positive results were reported from a trial involving six men with Becker muscular dystrophy, funding was secured from the Duchenne Alliance for a further trial involving six boys with Duchenne in the USA.

UNIVERSITY OF LAVAL – OSTEOPROTEGERIN (OPG)

Save Our Sons Duchenne Foundation funded research into a potential treatment approach for Duchenne led by Dr Jerome Frenette, a world renowned scientist in Canada. The research centred around a protein called osteoprotegerin (OPG) which it was thought may be able to improve muscle function.


OPG is known to be important for bone health and drugs based on OPG, and molecules it interacts with, have already been developed and are currently used to treat osteoporosis (brittle bones).


Dr Frenette’s research group found that treating mice with Duchenne with OPG improved muscle function. This research project looked at whether OPG alone or in combination with other drugs – glucocorticoids (already used to treat Duchenne) and beta agonists (used to treat asthma) – could be an effective therapy for Duchenne. As a bonus, OPG may be able to strengthen the bones which become brittle when taking steroids (glucocorticoids) and offset negative effects that beta agonists may have on the heart.

PHRIXUS – CARMASEAL-MD

Save Our Sons has supported research into Carmaseal-MD (also known as poloxamer 188)- a polymer that showed promise as a treatment for Duchenne due to its ability to stabilise muscle cell membranes.


In Duchenne, the muscle cell membranes are fragile and tear easily, it is hoped that Carmaseal-MD will be able to seal these tears and protect the muscle. Preclinical research in animal models showed that it is particularly useful in protecting the heart and breathing muscles. Importantly, it could be used for all Duchenne patients no matter what their genetic mutation is.
Our funding went towards a small pilot trial in boys/young men with Duchenne to gather the initial evidence of safety and effectiveness prior to starting their larger clinical trial.


The trial involved 10 patients at Cincinnati Children’s Hospital who injected Carmaseal-MD subcutaneously (under the skin) once per day for six months. The participants were non-ambulatory; had early heart failure and impaired respiratory function. The study gathered further information on the safety of Carmaseal-MD and assessments of effectiveness focusing on heart, lung and upper limb function.

.

MILO BIOTECHNOLOGY – FOLLISTATIN GENE THERAPY

Milo’s gene therapy is based on a protein called ‘follistatin’ which has the ability to increase muscle strength and prevent muscle wasting and fibrosis (formation of scar tissue). Follistatin is naturally made by the body and this gene therapy aims to increase its production.

The therapy involves using a harmless virus called AAV to deliver the gene containing the instructions for the production of follistatin into the muscle cells.


After positive results were reported from a trial involving six men with Becker muscular dystrophy, funding was secured from the Duchenne Alliance to start another trial involving six boys with Duchenne in the USA.

UNIVERSITY OF LAVAL – OSTEOPROTEGERIN (OPG)

Save Our Sons Duchenne Foundation has funded research into a new potential treatment approach for Duchenne led by Dr Jerome Frenette, a world renowned scientist in Canada. The research centred around a protein called osteoprotegerin (OPG) which it is thought may be able to improve muscle function.


OPG is known to be important for bone health and drugs based on OPG, and molecules it interacts with, have already been developed and are currently used to treat osteoporosis (brittle bones).


Dr Frenette’s research group found that treating mice with Duchenne with OPG improves muscle function. Our funding of the research project looked at whether OPG alone or in combination with other drugs – glucocorticoids (already used to treat Duchenne) and beta agonists (used to treat asthma) – could be an effective therapy for Duchenne. As a bonus, OPG may be able to strengthen the bones which become brittle when taking steroids (glucocorticoids) and offset negative effects that beta agonists may have on the heart.

AKASHI – HT-100

Save Our Sons granted funding to Akashi Therapeutics for a clinical trial of HT-100 – a powerful anti-inflammatory and anti-fibrotic.

HT-100 is a substance taken orally that is designed to reduce fibrosis (formation of scar tissue) and inflammation and promote healthy muscle fibre regeneration. A phase 1b/2a clinical trial of HT-100 was started in 2013 in five hospitals in the USA.