Sunday, March 2, 2014

Clinical Trial; Additional info on NIH

On Saturday morning, Dr. Kenneth Fischbeck from the National Institutes of Health was the guest for the KDA chat room. Dr. Fischbeck is a leading researcher, professor and educator on the subject of Kennedy's Disease. The chat focused on two current NIH research projects for finding a treatment for Kennedy’s Disease. Below are some excerpts from the chat. The entire chat can be found on the KDA website.

  • We are just starting an NIH trial with the drug from Novartis. Our trial is with a compound called BVS857 that Novartris has under development that activates the IGF-1 pathway. It fits with studies in our group at the NIH that have shown a benefit of IGF-1 in mouse versions of KD. It's pretty exciting for us to get to this stage, the result of several years of work with the mice in our lab and over a year and a half of working on it with the folks at Novartis. The BVS trial will also include several sites in Europe , in Italy, Germany, and Denmark.
  • BVS is a version of IGF-1 that should work on the muscles and also should help to protect motor neurons. IGF-1 (and we expect BVS) helps to stimulate muscle growth. It also should help by increasing the breakdown of the mutant androgen receptor protein. BVS is a new drug that has not yet been approved by the FDA.
  • We are starting with 2 KD patients from this area to make sure it's safe. The first is due to get the first dose on Tuesday. We'll be recruiting 6 more in July (if all goes well) and another 30 next January. The trial has 2 parts. Part A tests increasing doses for safety & tolerability. Part B tests the highest tolerated dose for efficacy over a 12-week period. The idea is that BVS would be given by injection, like insulin. We are planning on weekly injections (our best guess at present). We'll have to see how long the drug stays in the bloodstream in this trial. We may wind up giving it more or less often than weekly if it works.
  • In Part A the patients will get injections with gradually increasing doses every 2 weeks. We currently plan to give injections once a week for Part B. We will randomize Part B patients: 2:1, that is, 20 on drug and 10 on a placebo. The placebo is needed for comparison to test whether the drug is really working. Part B, with the 30 patients, will involve 12 weeks of treatment. It is our hope that we will see a benefit in 3 months. Our time frame is to finish Part A (which is just starting now) by the end of this year, and part B by the end of 2015.
  • Possible side effects are low blood sugar (like with insulin) at high doses. Also, IGF-1 (and BVS) at high doses have caused facial weakness (Bell's palsy). These are two things we are trying to be careful about by keeping the doses low and advancing slowly. Also, we are only giving the drug to people with low IGF-1 to begin with. Most KD patients have low IGF-1.
  • In Part B we will be using thigh muscle MRI as a way to confirm that the drug is increasing muscle growth. We'll also be testing muscle strength and function, and having patients fill out questionnaires to see how strong they feel.
  • For the BVS study, we are taking patients who have some weakness and are still able to walk for at least 2 minutes, with or without a cane. We decided to test BVS first in the range of patients who are weak but still able to walk, but the hope is that it would also help patients who are weaker and perhaps those who do not yet have muscle weakness (to slow or prevent the onset). Our hope is that BVS will also help patients who are wheelchair bound, but we'll have to see whether it works in patients who are able to walk first. For safety reasons, we are having the patients in the US come to the NIH weekly for the injections and follow-up (we cover the travel expenses). To see if you are eligible, you can contact Angela Kokkinis at the NIH: or by telephone: 1-301-451-8146. We have only selected the first two subjects for the BVS trial. There will be plenty of opportunity for patients to join in later this year and next, for those who are able to get to the NIH or one of the European sites on a weekly basis. If you live outside the US, the best bet is to contact one of the sites in Europe, which should be coming online in the next few months. For those interested in participating, but are currently taking Avodart (dutasteride): The rules of the trial require that the patients be off androgens & anti-androgens such as dutasteride for at least 3 months before starting. The other inclusion & exclusion criteria are listed on the website, or you can ask Angela.
  • If the current trial is successful, then there would probably be follow up studies in more places. There is a small chance the FDA would approve the drug if it has a strong effect in this study, but they usually like to have at least two positive studies for approval.
  • The trial has been posted publically on the website (you can search under "BVS857" or "Kennedy's disease") and on the NIH Clinical Center website. The company has not yet made public the details of the drugs composition, pending a patent application.
  • Reference other trials: Laura Bott and Cartlo Rinaldi in our lab have been working on ASC-JM17 and ASC-J9 in mice, and they have some interesting results. They were able to confirm that both drugs have a benefit in mice, although not as strong an effect as reported previously. They are working out the mechanism of action, which should help in developing a more potent drug.


  1. Hi Bruce! My name is Heather and I had a quick question for you and was wondering if you could email me when you have a free moment? I greatly appreciate it and hope to hear from you soon!! :-)

  2. Heather, I don't have your email address, so I cannot email you. You can email me at: I look forward to hearing from you.

  3. Hi Bruce, I had a look at the trials site but there seems to be little progress (other International sites not yet recruiting) so I was wondering whether you had heard anything further on the study yet, and if they're still expecting to finish by December 2015?

  4. Hi Bruce

    It seems that nothing is moving , any progress in other disease that this drug was planned for ?

  5. Well, we are all anxiously awaiting news. It is difficult to have patience when you have a progressive disorder.

    We have reached out to NIH for an update, but here is what we know. The trial is still in Phase A testing dosage, safety, etc. Should the results of testing with the current patients be positive, then they will move on to Phase B where there will be more patients involved. I wouldn't count on any dates, but I am hoping Phase B will start this spring or early summer.

    1. Hello - Is there latest trial on SBMA we can subscribe to ?. pls suggest

    2. Go to the Clinical Trial website ( and it will list current and previous trials. Also, visit the Kennedy's Disease Association website ( frequently to read about pending and concluded trials including the results of trials.


  6. Hi, I’m diagnosed with SBMA, I’m still walking, but my muscle weakness is growing. I really hope for the progress of clinical trials to treat this disease. Share information. I will be very grateful.


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