Putting the muscle in muscle stem cells

By Heather Buschman, Ph.D.
October 8, 2010
66

Patients with muscular dystrophy suffer debilitating muscle loss that gets worse as they age. As the disease progresses, resident stem cells in a patient’s muscle tissue have to work extra hard trying to replace the diseased muscle. Over time, this special population of stem cells gets exhausted as they constantly proliferate (make more stem cells) and differentiate (specialize into new muscle cells).

Dr. Pier Lorenzo Puri, associate professor at Sanford-Burnham and Italy’s Dulbecco Telethon Institute, and colleagues are figuring out ways to keep the muscle stem cell pool fresh and ready to regenerate injured or diseased muscle. In a study published today in the journal Cell Stem Cell, they uncover the molecular messengers that translate inflammatory signals into the genetic changes that tell muscle stem cells to differentiate. These findings give the scientists a target to artificially dial the stem cell population up or down, a potential treatment that could boost muscle regeneration in muscular dystrophy patients.

“Our mission is to improve the lives of these patients and extend their lives until they can benefit from a cure 20 years from now,” says Dr. Puri,  a medical doctor who has worked with many muscular dystrophy patients throughout his career.

Diseased or injured muscle becomes inflamed as cells and molecules flood the area to control the damage and begin repairs. Dr. Puri’s latest findings center around an inflammatory molecule called tumor necrosis factor (TNF) and a messenger molecule it talks to, called p38 alpha MAPK. Essentially, p38 alpha MAPK responds to inflammation by determining whether stem cells loitering in adult muscle tissue keep refreshing the pool of stem cells, or differentiate into functioning muscle cells.

Armed with this information, the research team used antibodies directed against TNF to block p38 alpha MAPK activity specifically in stem cells, a move that produced more stem cells. Anti-TNF antibodies could be used to generate more muscle stem cells in muscular dystrophy patients.

Once more muscle stem cells are generated, they can be induced to differentiate into functioning muscle cells. According to Dr. Puri, “the effect of anti-TNF treatment is reversible, so withdrawing the drug could then force the expanded population of stem cells to repopulate muscle cells.”

What’s more, since anti-TNF is already FDA-approved to treat rheumatoid arthritis and other autoimmune diseases, Dr. Puri hopes it will be more likely to benefit the current generation of muscular dystrophy patients – people who can’t wait decades for new drugs to be developed and approved.

“There’s an emotional charge in my lab,” Dr. Puri says. “We know there are children waiting for us to do our jobs, and when we work we are targeting those faces, those smiles.”

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About Author

Heather Buschman, Ph.D.

Heather was an SBP Communications staff member.

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66 Comments

  1. Pingback: Tweets that mention Beaker » Blog Archive » Putting the Muscle in Muscle Stem Cells -- Topsy.com

  2. This is a wonderful discovery.I hope this will work for a host of muscular dystrophies .There are hundreds of patients young and old who are waiting for some wonder treatment to keep them on their feet.I am also one of them.If any adult is treated for LGMD successfully,I can also try my luck.Kudos to Mr Puri for his discovery.

  3. Heather Buschman on

    Hi Ricky –

    We’re working on it! Dr. Puri’s study and others will hopefully allow the current generation of muscular dystrophy patients to live longer and more comfortably until a cure is found. Our research is mainly focused on molecular pathways in cells. For human trials of therapies for muscular dystrophy and other diseases, please check out http://clinicaltrials.gov.

    Thanks for your support!
    Heather

  4. Dear Heather,

    Thanks for your posts. This article surely brings smile on all the MD patients including me. I’m looking forward to an alternative treatment if a proper cure isnt available at the moment. Salute to Dr Puri who has been trying to help the MD people with his sincere efforts. We all Indians are proud of him.

    Further, may I know when this drug will be available to patients all around the world?

    Thanks again

    RiSh

    • Heather Buschman on

      Dear RiSh,

      Thanks for the comment. We really appreciate hearing from the patients who might one day benefit from our research. I look forward to writing more about MD in the future.

      Take Care!
      Heather

  5. My second boy, aged 13, suffering from Spinal muscular atrophy-2 and hope Dr. Puri, and his team will have a break through in this and my boy is ready for any kind of research to be done on him for the sake of future generations

    Thank you

    venky

  6. Dear Heather,

    I have a son who is 6 years of age suffering from CMD, i sincerly hope for some cure for him, will Stem Cell treatment will help him, can we get an appointment with Dr Puri, is this treatment available in India, kindly help me as we are running from post to post on some remedy for him. Seek your reply

  7. My nephew is suffering from DMD, Is there a way to slow down the progression of disease, I heared the name of exon skipping, Is this a treatment viable ?

  8. Pingback: Beaker » Blog Archive » New Mouse Model for Muscular Dystrophy

  9. shamim bangash on

    sir basically i m a patient of backer type muscular dystrophy.sir if there is any specific treatment of this disease then plz contact with me.becoz day by day i m going to down ward and i want to improve myself quickly.i was done alot of treatment but they did not give me specific treatment.sir i m from pakistan and this is my contact number +923018831081.i m waiting for ur comment and suggestion.

  10. Hi,

    Are any of these trials avialable outside the US, such as in NZ? It is very disheartening reading about all these trials that one cannot be part of

    Cheers

    • We don’t have much information on trials in other countries. However, you may want to check with the university hospitals, particularly Otago and Auckland.

  11. dear sir!
    my son is 7.5 year old, suffering from DMD. could this treatment is helpful for this. how can i get treatment? please reply.
    thanks

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  13. Hi there,

    It sounds wonderful.. My cousin is suffering from muscle distrophy .. He is 17 nd below Shoulder there are no movements.. Doctor says This is his final stage of life … I dont know But I want him to live Few more years… If U can contact me on my email nd help me with something which can affect a life that would be appreciated. following is my email Add., Hoping forr a miracle. May god be pleased with everyone

    ashraf.ali@live.in

  14. Hi, sir
    iam patient of muscular dystrophy LGMD(UTROPHIN IS NEGATIVE IN ALL FIBERS) SIR
    iam from India is it true that from stem cell my disease will be stable pls tell me

  15. Hi,

    My son 14 year old is having LGMD. I want to know is there any specific treatment for LGMD? we are now giving him Heomeopathic medicines and ayurvedic medicines. he is on wheel chair.
    When can Dr Puri’s discovery tried on patients?
    Please guide.

  16. Hi Sir
    i am 35 yrs old and suffering from Myotonic MD.Is this treatment helpfull for this type of MD. Thank you for your efforts to help people with MD. You said of a anti. TNF. What is its trade name and is it available in India. I am eager to know more and also ready for clinical trials.

  17. Dear sir!

    My son is 7 year old, suffering from DMD. We are now giving him ayurvedic medicines.
    I am from India . We all Indians are awaiting for your medicine .
    when can we get the medicine.

    please reply.

    Thanks

  18. Hello…my name is Anna and i’m suffering from gamma sarcoglycanopathy…please if you can TRY to help me,let me know…thank you

  19. hello ……………my name is bharat and my age is 40 and i am suffering from muscular dystrophy and i am able to walk.pls tell me some about medicine and its cost…..thank you

  20. Dear Dr.
    I am 20 years old, female from India,suffering from Muscular Dystrophy.
    Now I am wheel chaired. My legs, hands etc have become totally weak. I can not even drive away a mosquito biting on my nose badly. My parents have to drive it away, if they are available nearby.
    Kindly advise me any possible treatment for my disease. My relatives are in US. They said they will assist in taking me to US for any possible treatment if available there.
    waiting for your kind reply.

  21. Shara and others,

    We are a basic biomedical research institute and do not provide clinical care. However, I recommend visiting clinicaltrials.gov, the U.S. government clinical trials database. The link is also on the bar on the right.

  22. Dr.Sarvjit Dudeja,Ph.D. on

    I think more funds should be available to resolve the problem of different types of muscular dystrophy.I worked for Ph.D. on muscular dystrophy,keen to persue further if position or fellwoship is available.

  23. Dear Dr. Puri,
    I am 21 yrs old, female, suffering from spinal muscular dystrophy. I am able to walk with a walker but cannot climb a single stair. I had an operation of scoliosis correction at the age of 13. what further treatment can i get so that i can lead a normal life? can the research on stem cell help me out? plz reply.
    Thank you.

  24. PUNEET AGGARWAL on

    my son ryan is suuferring from muscular dystrphy. He is 5 years old. we could be able to detect at the age of 3 years that his muscles are week. Now it has been found that his suffering from MD. Kindly tell how and what to do for the treatment.

  25. Dr. Lorenzo Puri on

    Thank you to everyone for your comments.

    Our studies are essentially addressing basic issues on how regeneration of dystrophic muscles can be implemented at the molecular levels. These studies are the foundation for future clinical applications, and in particular they are inspiring a number of pharmacological interventions toward implementing regeneration and blocking fibrosis and fat deposition in mouse models of Duchenne Muscular Dystrophy. The concept can be potentially exported to other forms of muscular dystrophy. We are in the process of experimenting pre-clinical studies (in animal models) that hopefully could be translated into clinical trials on human patients. We keep you informed through this blog. For sure we are working hard to contribute to win the battle against this terrible disease.

    — Dr. Lorenzo Puri

  26. NAIMISH RANA on

    I AM 19 YEARS OLD. I HAVE APROBLEM OF MUSCULAR DYSTROPHY. MY MOTHER’S FATHER HAD ALSO THIS PROBLEM HIS THREE BROTHER HAS ALSO PROBLEM. I CAN WALK NOW BUT I AM FACING TROUBLE TO WALK MY BROTHER HAS ALSO THIS PROBLEM BUT HE IS BETTER THAN ME. WE ARE TAKING TREATMENT OF HOMEOPATHIC DOCTOR WHOSE NAME IS KETAN BHAI THAKKAR IN INDIA,GUJRAT,SURAT.IF YOU HAVE ANY SOLUTION OF THIS PROBLEM PLEASE INFORM ME BECAUSE I WANT TO RUN AND PLAY LIKE NORMAL PEOPLE. I HAVE INTEREST IN CRICKET. WHEN I SEE THE PEOPLE WHO PLAY CRICKET I FEEL VERY SAD. SO PLEASE DO AS YOU CAN. I WILL WAITING FOR YOUR REPLAY PLEASE SEND ME THE REPLAY.

  27. One of my relatives son has ‘Muskular dystrophy’can it be treated with one marrow stem cells.
    if yes plz guide regarding that.

  28. talibhussain on

    Dear Dr. Puri,
    i would like to know the treatment for my nephew. He is 12 and half years old. four month ago he is facing problems to walk and with arms to move or catch some thing. Dr. diagnosis muscular dystrophy. Dr. further says that there no treatment for this. Now he is day by day getting weaken and problems to walk and almost going to be dropped sometime. Kindly let me know whether some treatment for this any where so that my nephew be able to at-least to be walking. he may be stable at this stage hardly walking. kindly reply me must. will wait for favourable response.

  29. Manjit Nanglu on

    Hi I am a women of 42 years old and suffer from DMD. I struggle with walking, stairs, etc. It is getting very difficult for me any small glimmer of hope is a step in the right direction. I am a very positive person and lead life to the full. I have a daughter who also is carrier of dmd. She is 9 years old as not got any symptons at the moment but I am hoping that there is a cure soon so that, if it is too late for me, maybe not for her.

    I would be interested in taking part in human trails if there are any.

  30. My brother in law is suffered by MD for the past 15 years.His parents tried hard by several means but nothing paid off.We are desperately looking for the treatment where atleast further degeneration can be reduced/stopped.I request Dr.Puri to put his valuable suggestion and the roadmap of his research.

  31. I am suffering from muscular dystrophy. My age is 60 years. I am from India. At present I cannot walk, but my upper limbs are working though they are weak. I would be thankful to know as and when the pre-clinical studies are translated into clinical trials.

  32. MY SON SUFFERING FROM SPINAL MUSCULAR ATROPHY AGED ABOUT 20 YEARS. PL. SUGGET WHERE WE CAN TAKE THE STEM CELLS.

  33. venkatesh samala on

    this is venkatesh, my younger brother is suffered from muscular dystrophy now his age is 25 years old, now present situation is very serious total body get weakened, now he is in position very difficult for breathing,we r fearing any thing happen later, is that available any medicine to help him to solve this present situation ,his body including legs, lungs including other body parts are too painful please suggest me any medicine i waiting for your responce.

  34. my son 18 years old is suffering with this disease at this time he can’t eat himself ,we have to sleep or sat him, can’t stand himself .At present his mind is in complete working position.there remains pain in his full body,he can’t take any pain killer please suggest any treatment if possible.and also tell me about his future life.

  35. subhash nagpal on

    my son is suffering from muscular dystrophy. he is 14 years old. he can’t walk. he is not able to lift his arm up.
    please tell any treatment if possible. i will be eagerly wating for your reply. please reply soon. my cotact no. is 09412159454, 07520115190

  36. Bhavin Parekh on

    Hi,

    my Aunt is below 40s and suffering from dystrophy myopathy and she is unable to move her lower body as well as hands properly.
    eventually she lost working of her lungs too and currently in hospital on vantilator in her last stage of life hoping for some miracles.
    The medical history of her says that she had this disease inherited from his Father’s family and there are many cases in her father’s family tree of the same disease.
    My aunt has two sons and they are also carrying the same genes as confirmed by the report.
    One of his son has the high count of this risk ( I don’t know exact name of this test and component).
    My cousin who shows this high count is of age around 16-17. But there yet no sign of disease started.
    Please Register my name in your research for studying data for future reference.
    Please keep on posting me every news on the research of the same so we can help many people suffering from dystrophy myopathy.
    Please contact me on 9819886103 (Bhavin parekh) or my uncle Harish Parekh – 9737897916 for any updates.
    I really wish and hope your research get suceess.
    Thanks to doctor Puri and Team for their noble efforts for dystrophy patients.

    Bhavin Parekh

  37. Shanthi Antony on

    Hi,
    My daughter and son are suffering from Severe Childhood Autosomal Resisive Muscular Dystrophy(SCARMD). Now my daughter is 19 years old and my son is 9 years old. My daughter and my son are affected by this disease from 7 years. They both can’t walk, run and can’t do anything by themselves. Is there any treatment for this disease? If Yes, please inform me through my mail id.

    Please Register my daughter and son names in your research for studying data for future reference.

    I really wish and hope your research get success.

    Thanks to doctor Puri and his Team for their noble efforts for dystrophy patients.
    Shanthi.

  38. RAJIV GUPTA on

    Hello Dr.puri my son yatharth is suffering from DMD he is 14years of age now he is unable to move his legs and hands he is my only child neither my entire family nor the family of my wife have any history of this disease time is running out for me is there any treatment so that it can atleast be prolonged I will be highly grateful to you if you keep me informed about latest treatment and breakthroughs my email is rajivgpt007@gmail.com wishing you all the success and thankyou rajiv

  39. dear Dr.puri,
    my friend’s(she) son has been suffering from muscular dystrophy.. he is at 6 now.. doctors said he ill alive upto 8.. i have two questions..

    1.ill he get recovery from this or extended life time?
    2.she has a sister..(unmarried).. ill it affect her offsprings too? is there any preventive measures for her sister?

    pls reply me, i am waiting for your positive reply..
    ………………….sri

  40. Hi
    I have a son that is 13 an he suffers from DMD he is loosing his grip on his hands and he cannot walk from the age of 10
    Is there any treatment that can stall the loss of the muscles degenerating while we pray for your team i know that would come with a solution very soon.

    VERY CONCERNED FATHER

  41. naeem shehzad on

    sir i have a becker muscular dystrophy since last five year. at present time sir i can not walk without support. i can not open my full arm. i cann,t lift my leg up. i want to know is there any treatment for bmd. if it is plz inform at my email or my cell no
    +923315386248
    thankyou

  42. I am suffering from Muscular dystrophy for the past 15-20 years. Now am feeling very difficult to walk.
    I dont know what type of MD I am having. I want to know what type of MD, and how I can find it? . plz inform me
    waiting for your positive reply at “lthiru2974@rediffmail.com” or my mobile no.9840225980.

    thank you

  43. gandhi prasadh on

    i am suffering from muscular dystrophy for the past 5years. now am feeling very difficult to walk and working with hands. i dont khow what type of MD. pls tell me about this and give any medicine for this MD?
    my cell no:8125659437
    and my mail id is
    “gandhi.mca02@gmail.com” ,
    i am form hyderabad

  44. Doctor,
    My sonis suffering from Ducchines Resisive Muscular Dystrophy(DMD). Now my is 9 years old. He is affected by this disease from 6 years. He can’t walk and can’t do anything by his own. Is there any treatment for this disease? If Yes, please inform me through my mail id.

  45. Hello Friends

    i m also suffering from muscular dystrophy but i didn’t give up and i m improving. you know we can do a lot of things for improvement and for enjoying life. i have specially created a website for helping people like me who are desperate to have a cure. visit http://www.helping-md-to-heal.com and give your suggestions. i just have started it and as i get time i will add more and more information to it.
    thanks
    rakesh

    • hello rakesh,
      I am anuradha singh..25yr old..I am suffering from becker muscular dystrophy..actually now a day I am facing some difficulty in walking..please help me if anything you know it..
      waiting for your reply
      regards,
      ANU..

  46. R/Sir, My sun is 13year’s old he can’t able to walk from last 5 years he is suffring by ‘Mascular Distrophy’. sir any suach remedy available on this disis & where kindly communicate. (Kindly send through E-Mail only – Do not publish.)

    • Im a tad bit confused by your uotseiqn but i’ll try anywayz. Stem cells already have live donors from abortions and etc. for you embryonic stem cells. I doubt blood banks or hospitals would be in charge since there are still too mnay factors that people don’t know about stem cells. And how stem cells and blood can be seperated well they’re 2 different things since stem cells frmo the bone marrow can be differentiated into blood cells. I hope this helps

  47. Raja Viswanatham on

    Hello Dr,
    This is Raja (29 yrs) from India,sir i m a patient of Progressive – LGMD. If there is any specific treatment of this disease or to control the disease to some extend.
    Is there stem cells therophy will help us to recover or control it.
    Day by day i m going to down ward and i want to improve myself quickly.i was done alot of treatment but they did not give me specific treatment.
    I m waiting for ur comment and suggestion
    My mail id is : Rajav2009@hotmail.com

  48. Hey Doc,

    Hope you get those smiles soon cause we too are suffering from the pain caused by this…… God bless all of you.

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