Acthar For Myositis

Acthar, the only FDA-approved drug for myositis, has been available to treat myositis for years, but just recently has drawn attention because of Dr. Levine's small study resulting in the improvement of several patients who had not responded to other drugs.

Dr. Levine, who is on TMA's medical advisory board, is a member of Phoenix Neurological Associates. He is the founder and director of the Samaritan ALS clinic and co-director of the neurophysiology department at Banner Good Samaritan Medical Center.

Aisha Morrow, TMA:  Today we welcome Dr. Todd Levine, Dr. Levine, a neurologist with Phoenix Neurological Associates. He is the founder and director of the Samaritan ALS clinic and co-director of the neurophysiology department at Banner Good Samaritan Medical Center. Dr. Levine is on TMA's Medical Advisory Board and has presented on various topics at TMA's Annual Patient Conference. He completed a small study of Acthar, an older drug now being used in the treatment of difficult myositis cases, and will take questions today about the drug. Please check to see if your question has been answered previously before submitting it. Dr. Levine, thanks for being here today.

Dr. Levine:  I wanted to thank you for asking me here this afternoon. I will try to answer as many questions as possible.

TMA Member:  Is Acthor useful for IBM patients? Does this medication have any side effects?  Has it been tried on IBM patients? Do you recommend a 60 years old patient with IBM take this medication? Where I can get this medication?

Dr. Levine:  Acthar has not been studied in IBM, nor is it approved for patients with IBM. There is no way for me to know if it would benefit patients with IBM.

TMA Member:  Is Acthar a possible treatment for patient with DM/lupus,severe muscle wasting and weakness, ILD, and history of allergies to all previous immuno-suppressant drugs? Rituxan not advised due to allergies.

Dr. Levine:  Acthar is primarily being considered in patients who have failed multiple other therapies, so you could discuss it with your doctor.

TMA Member:  A recent article by Andrew Pollack in the New York Times said Acthar was "developed in the 1950s, by a division of Armour, the meatpacking company that once ruled the Union Stock Yards of Chicago. As in the 1950s Acthar is still extracted from the pituitary glands of slaughtered pigs---essentially a byproduct of the meatpacking industry. In 2007, it (Questcor) raised the price overnight, to more than $23,000 a vial." The current price is $28,000 for a 5-milliliter vial, according to the article. Has there been any trials or studies other than your recent small 5 patient study? 

Dr. Levine:  There are small studies underway to evaluate the utility of Acthar but there are no large studies planned that I am aware of. There is a large national registry that I am coordinating to see the effects of Acthar, so we hope in the next year or two to have a better understanding.

TMA Member:  Does Acthar or methotrexate work better for the skin symptoms of dermatomyositis?

Dr. Levine:  We do not know the answer to this question.

TMA Member:  Why would Acthar be prescribed instead of prednisone?

Dr. Levine:  Prednisone would be used first line in most cases, but if patients fail prednisone then your doctor could consider the use of Acthar.

TMA Member:  What does Acthar do that other medications used for DM or PM cannot do?

Dr. Levine:  Acthar binds to receptors called melanocortin receptors that are found on immune cells and may work to change the immune system differently than other medications and this could improve the immune response in PM/DM.

TMA Member:  If my insurance will not pay for use of Acthar, are there any options for me to have it be covered?

Dr. Levine:  The company does have patient assistance programs to help in these cases.

TMA Member:  Why has this treatment not been used before for myositis?

Dr. Levine:  I don't know..It has been FDA approved for over 50 years.

TMA Member:  What results have you seen in patients treated with Acthar?

Dr. Levine:  I have seen patients who have not responded to several medications do quite well with Acthar.

TMA Member:  If the FDA approves of Acthar, why would some insurers not be willing to cover it?

Dr. Levine:  Hopefully they will. It has been covered in all of my PM/DM patients so far as long as they have failed other therapies first.

TMA Member:  Are papers presented at the Rheumatology and Neurology Conferences and Symposiums on treatment for more severe cases of myositis with other overlap diseases? My concern is how many specialists with limited experience or even a lot of experience receive the most recent continuing medical education with myositis-related diseases.  I have been on about everything with no remission since 2007 and continue to get worse.

Dr. Levine:  This type of information is shared at conferences as well as journals. So we are trying to figure out better therapies all the time.

TMA Member:  Is there indication yet on how this drug will work for people with JO1 antibodies?

Dr. Levine:  None of the 5 patients I published had Jo-1 antibodies, so I do not have any experience with this subset. This is one of the questions we are trying to address with the registry I am running.

TMA Member:  I am 42 years old female. I have PM for 13 years. My CPK numbers are good and almost normal in the last 3 years but my muscles are weak that I can't walk stairs or get up from the floor. Do you think Acthar my help strengthen my muscles ?

Dr. Levine:  We do not know who the best candidates are for Acthar therapy. Since it is an FDA approved therapy it is reasonable to discuss it with your doctor.

TMA Member:  I am very sensitive to the side effects of prednisone.  Even 5 mg causes me irritability. Would Acthar be an effective replacement? Are there fewer side effects, and are there other side effects that prednisone does not have?

Dr. Levine:  Acthar causes your body to release its own steroids as opposed to taking outside steroids. The potential side effects are the same as steroids. In my published case series and longer-term follow up the side effects seem to be less than prednisone but we do not know if there is a difference, so you would have to watch for the same issues.

TMA Member:  I am a DM patient. Prednisone hasn't really helped me but my doctor believes it is keeping me from getting worse. Can I take Acthar with prednisone?

Dr. Levine:  I have treated patient with Acthar while they are on prednisone and this was published in my case series. One would have to watch for side effects and potential interactions but I did not see any in a small number of patients.

TMA Member:  My physician weaned me off prednisone because the mood swings were too unpleasant. Would Acthar be a better choice? I have PM.

Dr. Levine:  It is hard to know, but it may be worth discussing this with your doctor.

TMA Member:  I am a dermatomyositis patient. I have severe osteoporosis, presumably from long-term prednisone use. Would Acthar also cause osteoporosis?

Dr. Levine:  Acthar can still cause osteoporosis.

TMA Member:  Is Acthar something I can inject at home, or is it always done in the physician's office?

Dr. Levine:  Acthar is available as a self injection so all of my patients do it at their home themselves. The company offers some training as well free of charge.

TMA Member:  I was talking to my neurologist about Acthar and he said he was unfamiliar with it. I am wondering why he has never considered it in my care.

Dr. Levine:  The drug has been FDA approved and used to treat other autoimmune diseases like MS. There is not much data on its use in PM/DM. I think doctors will start to hear more about it and start to consider it more.

TMA Member:  I am on prednisone and it is working well and I am getting stronger. I have PM. However, I read that Acthar is better than prednisone because it stimulates your body's natural steroid production. Should I ask my rheumatologist to switch me to Acthar?

Dr. Levine:  You are correct about the difference between exogenous steroids, ones you take from pills or injections and Acthar which causes your body to produce its own. There have been no studies to say which is better.

TMA Member:  I have PM and my situation is getting worse. Prednisone did not help me. My physician tried Rituxan but I seemed to be prone to infections. She is now trying to decide between trying Acthar and IVIG. I still take methotrexate. What would be your recommendations?

Dr. Levine:  I am sorry but I can't comment on individual cases but it sounds like your doctor is considering good options. You have to discuss it with her.

TMA Member:  I have PM and now I have been diagnosed with ILD despite long-term steroids and cellcept, which haven't helped much. Would Acthar be a possibility for me?

Dr. Levine:  I have used Acthar in one patient with a history of ILD and she did well. However it has not been studied in large numbers. Again it would be worth talking to your doctor.

TMA Member:  As a woman with PM, I tried prednisone without success. I am currently still taking methotrexate. If I was trying Acthar, would it  be taken in conjunction with methotrexate, or have the studies found it works better by itself?

Dr. Levine:  The case series I published looked at patients who took Acthar along with other drugs. It was used for very refractory patients. The decision though would need to be made by your doctor.

TMA Member:  Would you recommend Acthar for someone with PM diagnosed 17 years ago who has been resistant  to medications and IVIG, experiencing restrictive lung disease and dropped head due to severely deteriorated para spinal muscles?

Dr. Levine:  I think most physicians who have been using Acthar for PM/DM are using it in their difficult refractory cases.

TMA Member:  What negative side effects were observed in your study?

Dr. Levine:  My case series treated patients for three months only and the only side effect was some taste disturbance. Longer term studies are needed to know the adverse effects. I am hoping the registry will help us understand this data better.

TMA Member:  If a patient is responding successfully to prednisone, what would be the advantage of switching to Acthar?

Dr. Levine:  It is very hard to mess with success. I usually don't change a patient who is doing well unless side effects become an issue.

TMA Member:  Are there plans to conduct an Achtar trial similar to the RIM trial for rituximab to find the most effective treatment for DM, PM patients?

Dr. Levine:  There is a large national registry underway to study how patients respond to Acthar but at the moment because of the time and expense of a study like RIM I am unaware of any plans for a similarly sized study with Acthar.

TMA Member:  I am 42 year old female. I have had PM 13 years. My CPK numbers are good and almost normal for the last 3 years but my muscles are so weak that I can't walk up stairs or get up from the floor. Do you think Acthar my help me strengthen my muscles ?

Dr. Levine:  I think when new drugs become available it tends to be used in the toughest cases that are refractory to standard drugs. So it may be worth talking to your doctor about Acthar.

TMA Member:  It appears that my insurance company does not want to pay for Acthar. Does the manufacturer have any programs to make the drug available? My physician believes it might be a good choice for me.

Dr. Levine:  There are patient assistance programs run by the company. When I last heard the company had gievn out over 150 million dollars in free drugs.

TMA Member:  How long does it take for Acthar to take effect in the body?

Dr. Levine:  In the case series I published most patients felt an improvement after three months but this was only 5 patients. So it would need to be something that you work through with your doctor.

TMA Member:  Can you discuss more recent patients results with acthar, especially since your prior study? Also, can you discuss if there is still a positive reception from insurers on the reimbursement front?

Dr. Levine:  I have prescribed it to about 5 other patients since the opriginal 5 and the majority have done well and felt that it helped. Insurance companies have approved all of my patients but that is because they have failed other therapies- in most cases many other therapies. The drug company has a very good patient program to help get the drug covered by insurance.

TMA Member: I have severe psoriatic arthritis, mild rheumatoid arthritis and mild myositis. I can not tolerate prednisone. Insurance has approved Acthar and I received the medication in the mail and am very excited. I do have anxiety as I am fearful of becoming cushinoid with too much cortisol. My rheumatologist said the day after my first Acthar injection I will do a 24-hr urinary cortisol test to ensure I am not producing too much cortisol. She wants a follow up in a week as she said it takes a few days to begin to work. I am taking 40 units twice a week. I am not sure if I should take the injection in the am or evening. Would one 24 urine cortisol test the day after first injection be sufficient to determine if I am taking a safe dose if it takes several days to get into my system? Would I not need to take the urine cortisol after the medicine is optimized? I am running out of medication options with the psoriatic arthritis and know I
have to try as I am unable to stand/walk for long & in a wheel chair. My quality of life is poor. Any advice you can share as to how to best ensure I am not producing too much cortisol would be greatly appreciated.

Dr. Levine:  The is no way for you to control how much cortisol you produce. The doctor is paying very close attention to this and can adjust the dose of the medication based on the cortisol results if she wishes. It sounds like you have a good doctor and you two are working as a team to figure out how best to use Acthar. Good luck.

TMA Member:  Where can we read your published cases that you spoke of earlier in this session?

Dr. Levine:  It was published in a journal called Drug Design development and therapy in June of 2012. It may be available on the myositis webpage if not you can google acthar and myositis and find it. 
Note:  Find Dr. Levine's Study at:

TMA Member:  ".....most physicians who have been using Acthar for PM/DM are using it in their difficult refractory cases" You said this earlier. About how many others doctors are using this drug in this type of situation? Is there any way to find out who these doctors are and where they treat?

Dr. Levine:  I know of dozens who have begun to use it in PM/DM. I don't know how you would find them in your areas though. Sorry.

TMA Member:  Is there a time of day that is better to take the injection, am or pm?

Dr. Levine:  Most take it in the morning.

TMA Member:  How many injections and what quantity per injection is usually prescribed with Acthar? At a cost of $28,000 per 5 ml vial, it appears to be cost prohibitive.

Dr. Levine:  It is very expensive, but so is IVIG. And most insurers pay for IVIG. The dosing is not set so the cost depends on how often and how many units your doctor prescribes.

TMA Member:  I am a 43 year old female diagnosed with PM. Just recently completed a 12 week Acthar session. Is there a timeframe between each session that is "normal" to wait before starting another one?

Dr. Levine:  I don't believe there is anything set in stone. I have had some patients that I have treated continuously now for up to nine months and other patients who I treat for 12 weeks and then they do well and I have chosen to stop and see what happens.It is all individualized. Like the disease is different in everyone, so is the therapy.

TMA Member:  Why did you become interested in trying Acthar for your patients when few other doctors seemed to be using it for myositis?

Dr. Levine:  It was a drug that was used by neurologists to treat multiple sclerosis patients who did not respond to steroids. So it seemed to make sense to try it in other autoimmune diseases where steroids had not worked. And it was FDA approved. And I guess for my really difficult to treat patients I was running out of other options.

TMA Member:  Are you or will you or any of the other doctors using Acthar, try to gather their information and results to publish or make available to other doctors not yet using it?

Dr. Levine:  Yes. The company has paid for and sponsired a registry that I initiated to try to do just this. If we can collect data on the patients who receive Acthar over the next two years we will know much more about it then we do know and we will also help make other doctors aware of whether it works.

TMA Member:  What criteria, tests and info do you gather to say that Acthar has been effective in your patients?

Dr. Levine:  I primarily follow their muscle strength, so I am looking to see if they get stronger. In some of the DM patients the rash has also improved.


TMA Member:  I am a male of 40, jo-1, on IVIG & imuran for last year.  CPK is 3000, feeling ok, sit most of the day. If I walk or stand for more than one hour, then muscles are painful by end of day. I am off methotrexate and prednisone for 6 months. I have fatty liver.  Would you recommend rituximab or Acthar?

Dr. Levine:  You would be considered a refractory patient who has tried many other therapies. Acthar could certainly be something to discuss with your doctor.


TMA Member:  My understanding that this drug has been FDA approved for a long time for other uses than DM/PM. Did the FDA have to approve it for DM/PM in recent years? If so, what did they use as their guidelines/ proof for use in DM/PM?

Dr. Levine:  In 2010 the FDA reexamined the label with all of the indications for Acthar and decided to leave PM/DM as an indicated disease. This was not based on any recent data or studies.


Aisha Morrow, TMA:  TMA would like to extend a special thanks to Dr. Levine for being with us and graciously spending the time to answer your questions. This concludes today's discussion. Thanks to all the members who participated today.

Dr. Levine:  Thank you all for your great questions today. I hope you found this helpful and I wanted to thank the TMA for arranging this.










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