Navigation Links
Experimental cancer drugs counter muscle deterioration seen in muscular dystrophy

Muscle weakness and fiber deterioration seen in muscular dystrophy can be countered by a class of drugs currently under study for their effects against cancer, a Burnham Institute study has found.

The report shed light on the potential use of these drugs, called histone deacetylase inhibitors, in promoting regeneration and repair of dystrophic muscles, thereby countering the progression of the disease, in two different mouse models of muscular dystrophy. Led by Burnham Institute assistant professor Lorenzo Puri, M.D., Ph.D., in collaboration with the Dulbecco Telethon Institute (DTI) of Rome and other colleagues in Italy and at the National Institutes of Health, the study was made available to researchers worldwide by expedited publication at Nature Medicine's website on September 17, 2006.

Puri's team discovered that ongoing treatment with the deacetylase inhibitor Trichostatin A, currently under clinical study for breast cancer, restored skeletal muscle mass and prevented the impaired function characteristic of muscular dystrophies. Importantly, these restored muscles showed an increased resistance to contraction-coupled degeneration--the primary mechanism by which muscle function declines in Duchenne muscular dystrophy and related dystrophies.

Indeed, muscles examined from dystrophic mice treated with Trichostatin A for three months displayed normal tissue architecture, as compared to the muscles examined from untreated, dystrophic mice. Furthermore, dystrophic mice receiving treatment were able to perform physical exercise (e.g. running on a treadmill) similar to normal, non-dystrophic mice.

Muscular dystrophy is a group of more than 30 genetic diseases, characterized by progressive weakness and deterioration of skeletal muscles. All are inherited, caused by a mutation in one of a group of genes responsible for maintaining muscle integrity. Puri's team studied the disease's most common form, Duchenne muscular dystrophy, which a ffects one in 3,500 male births, according to the National Institute of Neurological Diseases and Stroke. Inheritance is linked to the X chromosome and recessive, so the disease primarily affects boys. Most children with Duchenne muscular dystrophy die in their late teens or early 20s. The disease currently has no cure.

"We have identified a new rationale for treating muscular dystrophy, aimed at correcting the devastating effects of a single flawed gene," said Puri. "This is a significant advance over the use of steroids--currently the only treatment available--which offers palliative relief, often with severe side effects."

"These exciting results, while encouraging, will require extensive investigation to determine whether the effectiveness of these drugs in dystrophic mice will translate into an effective treatment for individuals suffering this disease," cautions Puri, who has devoted over 10 years to the study of muscular dystrophy. "It is difficult to predict how long it will take before these studies will be translated into therapies for human patients."

"Our future studies will focus on understanding precisely how several existing deacetylase inhibitors effect muscle regeneration. We will use this information to identify new compounds with similar or even better efficacy in treating muscular dystrophies."

Puri's research on the effects of deacetylase inhibitors on muscle regeneration was inspired by his previous studies, which started 10 years ago, in collaboration with Dr. Vittorio Sartorelli at NIH, on the biochemical and molecular mechanism regulating the expression of genes that coordinate muscle regeneration. These studies led to the identification of different enzymes (called acetyltransferases and deacetylases) that promote or inhibit the expression of regeneration genes, and have the potential of influencing the efficiency of muscle regeneration.

Source:Burnham Institute

Related biology news :

1. NIAID Initiates Trial of Experimental Avian Flu Vaccine
2. Experimental shingles vaccine proves effective in nationwide study
3. Experimental vaccine protects nonhuman primates when given after exposure to Marburg virus
4. Experimental TB drug effective against resistant and latent mycobacterium tuberculosis
5. Experimental drug reverses key cognitive deficits, pathology in Alzheimers
6. Experimental RNA-based drug kills prostate cancer cells effectively and safely
7. Experimental vaccine protects lab animals against several strains of H5N1
8. Experimental vaccine protects mice against deadly 1918 flu virus
9. Experimental vaccine given during pregnancy reduces stillbirths from common virus
10. Viral DNA sequence a possible trigger for breast cancer
11. Enzyme, lost in most mammals, is shown to protect against UV-induced skin cancer

Post Your Comments:

(Date:6/14/2017)...  IBM (NYSE: IBM ) is introducing several innovative ... to developing collaboration between startups and global businesses, taking place ... the event, nine startups will showcase the solutions they have ... France is one ... a 30 percent increase in the number of startups created ...
(Date:5/16/2017)... 16, 2017   Bridge Patient Portal , ... MD EMR Systems , an electronic medical ... GE, have established a partnership to build an ... the GE Centricity™ products, including Centricity Practice Solution ... These new integrations will allow healthcare delivery ...
(Date:4/19/2017)... April 19, 2017 The global ... landscape is marked by the presence of several large ... held by five major players - 3M Cogent, NEC ... accounted for nearly 61% of the global military biometric ... in the global military biometrics market boast global presence, ...
Breaking Biology News(10 mins):
(Date:10/12/2017)... ... October 12, 2017 , ... ... Market with the addition of its newest module, US Hemostats & Sealants. , ... thrombin hemostats, absorbable hemostats, fibrin sealants, synthetic sealants and biologic sealants used in ...
(Date:10/11/2017)... ... ... eye wash is a basic first aid supply for any work environment, but most personal ... rinse first if a dangerous substance enters both eyes? It’s one less decision, and likely ... dual eye piece. , “Whether its dirt and debris, or an acid or alkali, getting ...
(Date:10/11/2017)... ... 2017 , ... Disappearing forests and increased emissions are the main causes of ... year. Especially those living in larger cities are affected by air pollution related diseases. ... most pollution-affected countries globally - decided to take action. , “I knew I had ...
(Date:10/10/2017)... Philadelphia, PA (PRWEB) , ... October 10, 2017 ... ... University City Science Center’s FirstHand program has won a US2020 STEM Mentoring Award. ... accept the award for Excellence in Volunteer Experience from US2020. , US2020’s mission ...
Breaking Biology Technology: