play therapy with in the pool (mayopathy)

Research News :-Protein degradation a new drug target for Duchene muscular dystrophy?

Muscular dystrophy campaign

61 southwark street

London SE10HL 

3rd august 2012

  In muscle cells, dystrophin forms part of a group of proteins which acts as a scaffold to link the inside and outside of the cell. When dystrophin is not in the group (for instance in Duchene muscular dystrophy) the cell degrades the rest of proteins. Now, scientists have shows in mouse model that blocking this degradation can increase levels of the other proteins and help to stabilize the scaffold leading to reduced muscle damage.

What did the research show?

The dystrophin glycoprotein complex (DGC) is a group of several protein, including dystrophin, which works to stabilize muscle structure. The complex acts as a scaffold, linking the internal skeleton of the muscle to structures outside the cell.

In healthy muscle cells, the dystrophin protein hides a "signal" that tells the cell to degrade the DGC. The lack of dystrophin in Duchene muscular dystrophy can uncover this "signal" and lead to the loss of the DGC resulting in a weaker structural scaffold inside the muscle cell. This means the muscle is more likely to be damaged when it contracts and eventually leads to the muscle weakness and wasting seen in Duchene muscular dystrophy.

A team of researchers led by Prof Steve Winder at the University of Sheffield has tested whether blocking the "signal" for degradation could prevent the DGC being degraded in the absence of dystrophin. They created modified mdx mice (an animal  model of Duchenne muscular dystrophy) that were missing the signal and found that this prevented the loss of the DGC. They found that preventing the loss of the DGC led to an improvement in the muscle structure, and a reduction in muscle damage.

When the researchers examined the proteins that were present in the DGC of the modified mdx mice, they found that a protein called plectin was doing the job that dystrophin would normally do. Plectin is protein that plays a similar role to dystrophin, acting as a piece of scaffold to give cells stability and structure. The researchers do not know why plectin, rather than one of the other scaffold proteins in the cell, was taking the place of dystrophin in the DGC and this will need further investigation.

What does this mean for patients?

This research has increased our understanding of how a lack of dystrophin, leads to muscle damage. The researchers demonstrated that when dystrophin is missing, a "signal" is uncovered which tells the cell to degrade the rest of the DGC. The lack of a DGC means that the muscle has less protection from damage caused by the muscle contracting. If the "signal" is blocked, or switched off, then the DGC is not degraded by the cell and plectin can partially compensate for the lack of dystrophin, reducing muscle damage. This "signal" represents a new target for potential drugs for Duchenne muscular dystrophy and, like potential drugs which aim to increase utrophin production, chemicals that target this pathway may have the potential to treat all people with Duchene muscular dystrophy, regardless of their mutation.

Although this work is promising, it is still at a very early stage and has been carried out in an animal model. The techniques used to block the "signal" in mice could not be used in a clinical setting and so further research will be needed to identify candidate drugs which could specifically block the "signal" to degrade the DGC in the muscles of people with Duchene muscular dystrophy.

Research News :- New drug :- a potential treatment for duchenne muscular dystrophy

New drug :-  a potential treatment for duchenne muscular dystrophy

Muscular dystrophy campaign

61 southwark street

London SE10HL 

5^th April 2012

An international group of scientists, led by prof Gordon lynch at the university of Melbourne, Australia, have shown that increasing the amount of protein called heat shock protein 72(Hsp72) can have a beneficial effect in a mouse model of DMD. Giving amice drug called BGP-15(BGP-15 increasing Hsp72) improve the function of the limb muscles and more importantly, the breathing muscle as well as increasing the lifespan of the mice. Further work will have to be done before it can be confirmed that this approach will be beneficial for boys with DMD.

What did this research show?

The lack of dystropin in DMD leads to a series of events that cause the muscle to weaken and waste. One these events is a large increase in the amount of calcium inside the muscle .small tears in the muscle membrane that arise due to the lack of dystropin allow calcium into the muscle from the outside environment. High levels of calcium are damaging and lead to chronic inflammation and breakdown of the muscle.

 

The researchers decided to investigate proteins that are known to help regulate the amount of calcium inside the muscle. They wanted to find out if controlling the amount  of calcium in the muscle could prevent muscle damage occurring. They focused on a protein called heat shock protein 72 (Hsp72) known to be able to help cells regulate their calcium level.

Prof  lynch and his team used mdx mice (the mouse model for DMD) that were specially bred to also produce high level of Hsp72 in their muscles. When compared to  mdx mice that produce normal level of Hsp72, these mice had less muscle breakdown, improved muscle strength and importantly less degeneration of the diaphragm (breathing muscle). The researcher showed that this was due to the fact that the muscle were being repaired more efficiently.  

 Since these initial results were promising, the team decided to test whether a drug,called BGP-15, that is known to increase levels of Hsp72 in humans might show similar results. BGP-15 is currently in clinical trial to treat diabetes. They looked at two mouse models of DMD-the mdx mouse and the very severely affected double knock out  (dko) mouse. Dko mice are missing not only the dystrophin protein, but also a related protein called utropien. Utrophin is known to be able to compensate for a lack of dystrophin and if both proteins are missing, symptoms in mice are more severe and are very like those seen in boys with DMD. BGP-15 treated mice had decreased muscle breakdown and greater muscle strength than untreated mice. They also had less degeneration in the diaphragm which points to an improvement in their ability to breathe as well as an increase in lifespan.

Prof Dame key Davies, a muscular dystrophy campaign grantee and one of the team who working on the project said;

It is exciting to see that increased levels of the protein Hsp72, boosted by a novel drug called BGP-15, extends the life of these mice substantially. This makes it much likely that this drug will benefit patients not only with DMD, but with other muscle disorders too.

Dr marita pohlschmidt, director of research at the muscular dystrophy campaign said;

 The result are encouraging news for the development of a treatment that could delay the progression of DMD and therefore could improve the live expectancy of the boys. In addition, the suggested drug could be used in conjunction with one of the promising potential therapies that are currently tested in clinical trial, should any of them show a clinical benefit.

The main objective of our foundation

  Our foundation is keenly watching the global problems and their solutions. After a careful study and analysis, we had zeroed in the agonies of the children suffering from Muscular Dystrophy, which has been so far neglected by the Philanthropists/Organizations and till date no cure is available.

The main objective of our foundation is to build confidence in the children suffering from Muscular Dystrophy through physiotherapy, hydrotherapy, occupational therapy, play therapy etc, which will help to prolong the life span of the children. It throws the light in the Medical field through the constant & consistent research in exploring causes and solution for the prevention and cure of Muscular Dystrophy first time in India.

Services offered inMayopathy Institute of Muscular Dystrophy & Research Center

• Physiotherapy
• Occupational therapy
• Medical intervention
• Play therapy
• Hydrotherapy
• Yoga therapy
• Guidance’s and counseling
• Diet and nutrient
• Periodic monitoring and evaluation of client
• Undertaking research about severity of condition

further details can be viewed at our website www.jeevanfoundation.com. Activities of the Institute can also be checked in youtube link “Mayopathy English version” http://www.youtube.com/watch?v=je5H-CbhcIc

Mayopathy Institute of Muscular Dystrophy & Research Center

     Mayopathy Institute of Muscular Dystrophy & Research Center, an exclusive and first-of-kind institution, is located at a serene, pollution-free and nature-rich environment in Veeravanallur, a small hamlet in Tirunelveli district of Tamil Nadu, India. It is one of the units of Jeevan Foundation which is established in 2006, as a non-profit organization registered under Tamil Nadu Trust Act.

   The main objective of this institution is to provide Medical and holistic rehabilitation service by a multidisciplinary team for the persons suffering from muscular dystrophy and help them lead their lives with confidence and determination. It throws light in medical field through the constant and consistent research in exploring the causes and solution for the prevention and cure of Muscular Dystrophy.

Mayopathy Institute of Muscular Dystrophy & Research Centre is an exclusive and one of a kind institution, It’s located at a serene, pollution-free and nature-rich environment