On Friday of last week I had my annual appointment at the IBM Research Clinic at the Centre for Neuromuscular Diseases, at Queens Square London.
As usual when I have a appointment at a hospital, the day started early, with Margaret my carer coming at 6.30 am to get me up, showered and dressed ready for the day.
My appointment was at 10.00 am, and I had been told to be ready for collection by the hospital transport two and a half hours before my appointment. The transport from this hospital is always late, but ever the optimist, I'm ready by 7.30 am, as requested.
The transport arrives only 45 minutes late, but this still gives us plenty of time to be at Queens Square on time for my appointment. After I'm strapped in, we set off, but have to pick up another patient, just a bit off our direct route.
The traffic isn't to bad and we arrive at the other patients. The driver goes and knocks on the door and waits for an answer. After a couple of minutes a lady comes down the road, walks past the driver, opens the door to the house, goes inside and shuts the door. A couple of minutes later the patient emerges from the house and starts to walk towards the ambulance. As he gets almost to the ambulance he remembers that he has left something behind, and has to go back indoors, shutting to front door, before coming out 5 minutes later. Despite always being told to be ready, so as not to delay the transport, this has taken over 15 minutes. The patient appears to be a regular on trips to the hospital, as he is known to the driver.
We finally get going, and the traffic has now built up quite a bit and it soon becomes apparent that we are going to be late for my appointment. One of my pet hates is being late for appointments - I'd rather be 20 minutes early than 20 seconds late. I wait until about 9.40 am, so that I can judge how much further we have to go, and then ring the clinic to say, I'm on my way but will be about 30 minutes late. They are very understanding, and tell me that its OK.
We eventually get to Queens Square about 25 minutes after my appointment time, and to save the driver trying to find a proper parking space, I tell him to drop me any where and I can then make my way to the clinic. He finds a space, right by a drop kerb, just round the corner from Queens Square - in the same street as Great Ormond Street Hospital. He gets the straps off my chair and I am ready to go.
I arrive just over 30 minutes late at the reception.
I am seeing one of the Clinical Research Fellows, called Dr Pedro Machado. Many of the people who see Pedro, particularly the ladies, refer to him as the "lovely" Pedro. This is actually very apt. He is a very nice Doctor, who is very enthusiastic about his work and he is always really interested in the patients he sees, and you get the real feeling that he really does care and want to help. Nothing is too much trouble for him.
Even though I am late and apologise, he says not to worry and we make our way through a labyrinth of corridors in the building to a consulting room.
When I was at the clinic last year, Liz Dewar from the Physiotherapy Department, sat in with us and she offered to see me, regarding the possibility of trying to stand. Pedro asked me about this and what had happened. I told him that we had had some success with the standing frame at the hospital, but that I had now purchased a standing up powered wheelchair, which I use every day at home to stand for between 15 and 20 minutes. He was really interested in this and how it worked, but when I started to tell him about the physical benefits I thought that standing on a daily basis was having, he was really delighted. I told him about the video that I had done of me standing in the chair, and that Liz had a copy of this, and he said that he would see her and get a copy.
We spoke briefly about how I was generally, and how I felt I am compared to last year when I had been at the clinic. One of the primary objectives of the clinic is to look at the progression of IBM over a long term, in a variety of patients, to try to build up a picture of how the condition progresses. Part of this is that each year we do a very extensive number of tests on the muscles to determine and measure the strength in the muscles. These are the normal tests that most myositis sufferers are familiar with, where a Doctor will, for example, ask you to raise your arm, and then push against you, to measure your resistance. However, the number of tests done by Pedro, are far more than those done by any other doctor.
The results from this years tests, are very similar to last years, which shows that my condition is pretty much stable. This is what I had thought, and hoped, and is a very good sign. I think this in part is down to the regular physiotherapy sessions I have about every 2 weeks at home, and to the standing.
Following this Pedro gives me an update on the drug trials, in connection with IBM. I have already posted about this on Facebook, but have included this details at the bottom of this blog, for anyone who has missed them.
Just before 11.30 am, we finish up, as Pedro has another patient coming in. We have had a very interesting and constructive hour.
As I am leaving, I meet John Robertson, a fellow IBM'er. who I have corresponded with for sometime through emails, Facebook, on the TMA forums, but this is first time we have meet. We have a nice chat before John is called in for his appointment.
I then make my way across Queens Square and into the National Hospital for Neurology and Neurosurgery, and firstly find a toilet, and then book in for the transport to take me home. After a wait of almost 2 hours, a lady finally comes an collects me. She drives one of the small vans, that they sometimes use to transport patients. Although the vehicle is clearly to small - my head is on the ceiling of the van, so I know I'll be banging my head every time we go over a pot hole or speed bump - by this time I just want to get home.
I finally get home about 3.45 pm, and I am exhausted.
Arimoclomol
A new funding grant application has just been presented to the FDA and it is hoped that they will have an answer on this in Mar/Apr 2014. If this is successful then the trial will still have some bureaucratic hurdles to get over and is therefore unlikely to start until late 2014.
The company that originally held the rights for this drug has sold these to a company in Denmark. This new company was looking to develop Arimoclomol for another condition, even rarer than IBM, and it was only when the team from UCLH went and did a presentation about the first part trial, that the company became aware of its potential application in the treatment of IBM.
This Danish company is a small pharmaceutical company. It does not have the budget/funds to be able to fund the drug trial itself. However, Pedro understands that the Board of this company are currently in discussions, to consider if they can approach their financial backers, with a view to them raising the funds required, if the FDA turns down the funding application.
The next part of the trial will involve about 200 patients. These will be split between the USA and UK, but there are discussions about other countries being involved. The criteria for the new trial, has not yet been set.
Novartis Bimagrumab/BYM338
The original trial was to test the safety of this drug, in humans. It was successfully shown to be safe, but there were also some encouraging results. Of the patients receiving the drug - and this was a single dose - under MRI, there was found to be on average an 7-8% increase in muscle. This doesn’t necessarily mean an increase in strength of function, but just an increase in muscle. However, one of the criteria for this trial was that the patient should be ambulatory and complete a 6 minute walk test. Of those patients who received the drug on the trial, there was an up to 10% improvement in the results of this test – this could have been the time to cover the same distance, or that they completed a further distance in the time.
In the UK, Pedro stated that they had been hoping to start the trial in January 2014, but there will be a delay whilst some further regulatory requirements are meet, but he is hopeful that the trial will now start in Apr/May 2014. He anticipates that the trial here in the UK will involve 30 patients, and that these will be split between 3 or 4 regional centres. One will be UCLH in London, and the others will be between Newcastle, Southampton and Manchester. He could not say what the numerical split of patients would be.
He thinks that patients for the trial will be randomly selected from the database of patients at each of these centres, and from any research patients. The patients must have a confirmed diagnosis of IBM, with a muscle biopsy that fully confirms this. To explain this a bit more, I have been diagnosed with IBM, and have a muscle biopsy that shows all the signs of IBM, but the biopsy isn’t in itself conclusive. Therefore it is possible that I might have another condition, very similar to IBM – which I have had DNA testing for – but they are unable to confirm another diagnosis. Therefore, I have a diagnosis of IBM, as they have nothing else to call it – in very simple terms.
Patients will also have to be taking less than 10 mg of Prednisolone daily, and some other immunosuppressant drugs will exclude patients, as these may interact with the BYM338 and affect results.
Patients will also need to be ambulatory, but can use any walking aids.
There may be other criteria beyond these but these will be the main ones.
The drug will be given over a 12 month period – one infusion each month. However if patient A receives their first infusion in May 2014, and the last patient doesn’t join the trial until Nov 2014, patient A will continue to receive the drug until the last recruited patient has had their full 12 months of treatment.
If a patient joins the trial and drops out at any point after the first infusion, for whatever reason, their results will still be included in the trial, and they will not be replaced.
There will be 4 levels with the trial. 25% of patients will receive a placebo, 25% will receive a low dose, 25% will receive an intermediate dose and 25% a high dose. It is not known at which level the drug will be most effective and it could be the case that at the low dose that there is no effect on the patient. Likewise, it could be that with the high dose, that there is an increased risk of side effects.
The drug is new and is being developed, and there is a potential risk to be included in the trial. Pedro reiterated some of the points raised by Dr Greenberg in his presentation to the TMA Annual Conference about the potential risk to the cardiac muscle.
My feeling is that Pedro is cautiously optimistic about both of these trials. He certainly gave me the impression that he is excited about these trials and the potential for both drugs.
A new funding grant application has just been presented to the FDA and it is hoped that they will have an answer on this in Mar/Apr 2014. If this is successful then the trial will still have some bureaucratic hurdles to get over and is therefore unlikely to start until late 2014.
The company that originally held the rights for this drug has sold these to a company in Denmark. This new company was looking to develop Arimoclomol for another condition, even rarer than IBM, and it was only when the team from UCLH went and did a presentation about the first part trial, that the company became aware of its potential application in the treatment of IBM.
This Danish company is a small pharmaceutical company. It does not have the budget/funds to be able to fund the drug trial itself. However, Pedro understands that the Board of this company are currently in discussions, to consider if they can approach their financial backers, with a view to them raising the funds required, if the FDA turns down the funding application.
The next part of the trial will involve about 200 patients. These will be split between the USA and UK, but there are discussions about other countries being involved. The criteria for the new trial, has not yet been set.
Novartis Bimagrumab/BYM338
The original trial was to test the safety of this drug, in humans. It was successfully shown to be safe, but there were also some encouraging results. Of the patients receiving the drug - and this was a single dose - under MRI, there was found to be on average an 7-8% increase in muscle. This doesn’t necessarily mean an increase in strength of function, but just an increase in muscle. However, one of the criteria for this trial was that the patient should be ambulatory and complete a 6 minute walk test. Of those patients who received the drug on the trial, there was an up to 10% improvement in the results of this test – this could have been the time to cover the same distance, or that they completed a further distance in the time.
In the UK, Pedro stated that they had been hoping to start the trial in January 2014, but there will be a delay whilst some further regulatory requirements are meet, but he is hopeful that the trial will now start in Apr/May 2014. He anticipates that the trial here in the UK will involve 30 patients, and that these will be split between 3 or 4 regional centres. One will be UCLH in London, and the others will be between Newcastle, Southampton and Manchester. He could not say what the numerical split of patients would be.
He thinks that patients for the trial will be randomly selected from the database of patients at each of these centres, and from any research patients. The patients must have a confirmed diagnosis of IBM, with a muscle biopsy that fully confirms this. To explain this a bit more, I have been diagnosed with IBM, and have a muscle biopsy that shows all the signs of IBM, but the biopsy isn’t in itself conclusive. Therefore it is possible that I might have another condition, very similar to IBM – which I have had DNA testing for – but they are unable to confirm another diagnosis. Therefore, I have a diagnosis of IBM, as they have nothing else to call it – in very simple terms.
Patients will also have to be taking less than 10 mg of Prednisolone daily, and some other immunosuppressant drugs will exclude patients, as these may interact with the BYM338 and affect results.
Patients will also need to be ambulatory, but can use any walking aids.
There may be other criteria beyond these but these will be the main ones.
The drug will be given over a 12 month period – one infusion each month. However if patient A receives their first infusion in May 2014, and the last patient doesn’t join the trial until Nov 2014, patient A will continue to receive the drug until the last recruited patient has had their full 12 months of treatment.
If a patient joins the trial and drops out at any point after the first infusion, for whatever reason, their results will still be included in the trial, and they will not be replaced.
There will be 4 levels with the trial. 25% of patients will receive a placebo, 25% will receive a low dose, 25% will receive an intermediate dose and 25% a high dose. It is not known at which level the drug will be most effective and it could be the case that at the low dose that there is no effect on the patient. Likewise, it could be that with the high dose, that there is an increased risk of side effects.
The drug is new and is being developed, and there is a potential risk to be included in the trial. Pedro reiterated some of the points raised by Dr Greenberg in his presentation to the TMA Annual Conference about the potential risk to the cardiac muscle.
My feeling is that Pedro is cautiously optimistic about both of these trials. He certainly gave me the impression that he is excited about these trials and the potential for both drugs.
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