National Amyloidosis Centre Patient Forum

Amyloidosis => Research => Topic started by: Miriam Vered on April 05, 2015, 07:19:38 am

Title: Results of Phase I trial of CPHPC + anti-SAP antibodies
Post by: Miriam Vered on April 05, 2015, 07:19:38 am
Hi All,

The attached abstract was presented at the Annual Meeting of the Association of Physicians 27 March 2015.

Below is Professor Sir Mark Pepys' explanation:

The abstract summarises the findings of the initial part of the Phase I trial of the obligate therapeutic partnership of CPHPC plus anti-SAP antibodies in patients with systemic amyloidosis.

This was the first in human clinical trial of a first in class treatment that has never been used before.  The main aim was to show that the drug combination is safe and acceptably tolerated in patients.

Patients with amyloid in the heart were excluded from this first part of the trial for safety reasons.  We have substantial experience with CPHPC in amyloid patients and it has always been absolutely safe but anti-SAP antibodies have not previously been given.  The initial antibody doses were accordingly very small but in the absence of any ill effects, larger doses were given and were generally well tolerated.

Most of the patients were selected because they had amyloid affecting the liver as well as some other organs.  There are several different powerful methods for assessing both liver amyloid load and liver function.

The important results of the trial so far are as follows:

1.  The treatment is safe and well tolerated.

2.  After treatment with a sufficient antibody dose, there was evidence of reduction in amyloid load in almost all subjects.  This was associated with improved liver function in some subjects.

This finding is unprecedented.

There has never been any other treatment that directly targets amyloid deposits nor any intervention which produces such rapid clearance of amyloid from the tissues.

In some individuals the disappearance of amyloid was dramatic and in others, with much larger initial amyloid load, it was more subtle.  Crucially the dose level at which amyloid clearance was seen was much smaller than the optimal dose used in the mouse experiments on which this new clinical treatment was based.  Further experience with larger and repeated antibody doses, not yet reported, shows that additional clearance from the liver and other organs can be achieved.

The present trial is continuing and plans for a Phase II trial, aiming to confirm clinical efficacy and patient benefit are in progress.  However the results so far have shown convincingly that reduction in amyloid load in the tissues leads to improved organ function.
Title: Re: Results of Phase I trial of CPHPC + anti-SAP antibodies
Post by: Peter on April 05, 2015, 08:46:41 am
Miriam
The results look very encouraging, I heard about the drug when I was last at the NAC in November of last year, due up again early next month. I asked then no doubt like many others if I could be included on the trials but because of the heart involvement was told no as explained in your post.
What are the chances of this being extended seeing the positive results received, ever hopefull
Title: Re: Results of Phase I trial of CPHPC + anti-SAP antibodies
Post by: patpinchin on April 05, 2015, 09:12:51 am
Hurrah! Happy Easter everyone!  :) :) :)
Such wonderful news and so eagerly awaited by so many.
The best Easter Egg for Amyloidosis sufferers in the UK.

Congratulations and huge thanks to Professor Sir Mark Pepys and his team after so many years of hard work.   :)  :).  :)

Onwards and upwards with phase 2.

Thank-you SO much Miriam for posting this.  :) :) :)
Title: Re: Results of Phase I trial of CPHPC + anti-SAP antibodies
Post by: Annone Butler on April 05, 2015, 12:22:33 pm
Very good news. And just in time for Spring! Many Congratulations to all the team at the NAC and GSK.
Title: Re: Results of Phase I trial of CPHPC + anti-SAP antibodies
Post by: Elliei21@me.com on April 05, 2015, 05:12:13 pm
Thankyou Miriam for this most promising post on Easter Sunday, the results are very encouraging and hopeful for so many of us reading these results, I am at the NAC early May, I will definitely be asking about these trials whilst I'm there too.
I think we can all agree that there is a lot of hope for Amyloidosis sufferers when we are receiving trial results like that.
Great news !!!!! EllieX
Title: Re: Results of Phase I trial of CPHPC + anti-SAP antibodies
Post by: Helen33 on April 05, 2015, 05:39:40 pm
I do hope that I might be considered for  this trial drug.  Can someone inform me who I need to approach please.  It seems so much better option for me than more chemotherapy to try to prevent my kidneys going further into decline.  It is highly probable that the chemotherapy causes more damage. 

What a magnificent breakthrough. 
Title: Re: Results of Phase I trial of CPHPC + anti-SAP antibodies
Post by: patpinchin on April 05, 2015, 06:35:01 pm
I am among the small number of NAC patients awaiting this news for many years. Dr Gilmore first told me about CPHPC and its testing on mice models in 2006. It took several more years before the anti-SAP antibody was on the agenda. Eventually the reality of a drug and the leading drug company GSK to sponsor clinical trials costing many billions of pounds. Now 9 years later we have received today's amazing news from the wonderful great man who invented the drug. Proof of safety of the drug in humans, evidence of clearance of amyloid even at low dose at rates according to load and a phase 2 trial to prove efficacy in plan. In addition, one of the first new drugs designated by the European Medical Agency for fast tracking. Professor Pepys has devoted his life to Amyloidosis since he established the NAC all those years ago. Although sadly this drug is too late for some, current NAC patients can be hopeful of a better quality of life to come. We are indeed indebted to Sir Mark and his NAC team of researchers and Doctors.
Title: Re: Results of Phase I trial of CPHPC + anti-SAP antibodies
Post by: Miriam Vered on April 05, 2015, 08:19:23 pm

Helen asked:

Quote
I do hope that I might be considered for  this trial drug.  Can someone inform me who I need to approach please.


I'm sure that everyone on the forum is thinking something similar.


This is Professor Sir Mark Pepys' answer:


The entry criteria for the trial are extremely stringent for safety reasons. All patients under the care of the NAC are well known to those involved in selecting trial candidates and will be approached appropriately when the time is right. Please be assured that we are well aware of the desire of all patients to receive useful treatment as soon as possible and we are doing our best to ensure that this happens. Our goal is to create a safe new medicine that will benefit patients and everybody involved is working as hard as they can to make this happen as early as possible.


Title: Re: Results of Phase I trial of CPHPC + anti-SAP antibodies
Post by: Elizabeth on April 05, 2015, 09:13:41 pm
This sounds wonderful news and I'm sure gives all amyloidosis sufferers greater hope for the future. Many thanks for all involved in the research and development of this drug, as this could possibly transform the lives of many such as myself who suffer the consequences of this disease, and hopefully give us a far better prognosis.
Title: Re: Results of Phase I trial of CPHPC + anti-SAP antibodies
Post by: JellybabyJan on April 06, 2015, 05:11:37 pm
Hi Miriam

Great news.  Do you know if it will ever be available for patients with cardiac amyloidosis? I think it's worth the risk. Thanks.

Jan x
Title: Re: Results of Phase I trial of CPHPC + anti-SAP antibodies
Post by: Miriam Vered on April 06, 2015, 07:15:08 pm
Hi Jan,


The treatment is being developed with a goal of treating all types of amyloidosis affecting all organs, including cardiac amyloidosis. Unfortunately we don't yet know how effective it will be, and how long it will take until it is more widely available.
The information above is all that I can provide right now.
 I'll keep you all posted when further information is available.
 
Title: Re: Results of Phase I trial of CPHPC + anti-SAP antibodies
Post by: Lesley on April 06, 2015, 07:39:07 pm
If I had to give up one thing.... Mine wd be that pat Pinchin takes this drug trial before me. This wonderful courageous lady deserves  a hope. Please let pat be the one to trial this drug. My love to a very special brave lovely lady - pat Pinchin xxxx
Title: Re: Results of Phase I trial of CPHPC + anti-SAP antibodies
Post by: patpinchin on April 06, 2015, 07:54:07 pm
Please can you confirm Miriam, that the potential of the new drug is to clear amyloid deposits in tissues as well as organs? Organ involvement does not affect all AL sufferers, and is as you know very hard to treat when soft tissue is the area involved. Amyloid has thus far rarely regressed from soft tissue even with a full or very good haematological response.
Title: Re: Results of Phase I trial of CPHPC + anti-SAP antibodies
Post by: JellybabyJan on April 06, 2015, 07:56:45 pm
Hi Miriam

Thanks for the update, still very exciting

Jan
Title: Re: Results of Phase I trial of CPHPC + anti-SAP antibodies
Post by: Miriam Vered on April 07, 2015, 07:34:13 am
Pat asked:
Quote
Please can you confirm Miriam, that the potential of the new drug is to clear amyloid deposits in tissues as well as organs? Organ involvement does not affect all AL sufferers, and is as you know very hard to treat when soft tissue is the area involved. Amyloid has thus far rarely regressed from soft tissue even with a full or very good haematological response.


Hi Pat,


Professor Sir Mark Pepys' reply is as follows:


The aim of the treatment is to remove all systemic amyloid deposits from all tissues and organs. There are no animal models suited for investigation of this potential and only continuing clinical testing will eventually reveal the full effectiveness of the intervention. Initially the testing can only proceed in the context of a formal clinical trial with its unavoidably restrictive and onerous regulatory framework. Provided there are no safety issues and the results continue to be encouraging, it is possible that inclusion criteria may be broadened and that eventually there might be a possibility of compassionate use. However this is very uncertain and will be decided by GSK and not by the NAC, although we shall certainly suggest and advise.

Title: Re: Results of Phase I trial of CPHPC + anti-SAP antibodies
Post by: patpinchin on April 07, 2015, 10:02:37 am
Many thanks Miriam and Sir Mark. Your mind reading is spot on!  :)
Title: Re: Results of Phase I trial of CPHPC + anti-SAP antibodies
Post by: ziad_okeil on April 08, 2015, 11:49:31 am
Hello Miriam,

First of all, please allow me to express my admiration for what you are doing in/for this forum. It is a great job and you are handling it very well. My compliments!

I am actually willing to come for a check up/second opinion at NAC (Already contacted Prof. Hawkins via E-mail last year) but would this make me an NAC patient? Would this make me eligible to get a chance to be included in the trial? Or at least to eventually get the new drug whenever that will be available for NAC patients?
I know it is a long shot for me as I live in Austria and not a UK citizen.

Reminder:
I have SMM and AL AMY, Skin involvement, in my face/lips mainly. No internal organ involvement (so far). Except for the elevated Lambda FLC numbers in blood, all my other numbers seem to lie in normal range.
It is supposedly a very rare form of AL AMY (according to my doctors in Austria) especially that the AMY deposits are adjacent and not interlocking (whatever that means).

Thanks a lot

Kind regards,
Ziad
Title: Re: Results of Phase I trial of CPHPC + anti-SAP antibodies
Post by: Miriam Vered on April 08, 2015, 05:06:16 pm
Hi Ziad,


Thank you for your kind words.
When patients from abroad wish to be seen at the NAC, their own doctors should contact the NAC doctors to discuss the case.
I understand that you've already been in touch with Professor Hawkins. For anyone else reading this, the NAC doctors' contact information is available at:
https://www.ucl.ac.uk/amyloidosis/nac


Once it has been decided that a patient from abroad should be seen at the NAC, the Appointments and Office Manager Ramon Lamarca (contact details also at https://www.ucl.ac.uk/amyloidosis/nac) should be contacted to make the logistic arrangements.


Ziad, I recommend that you or your doctors should discuss your questions with Professor Hawkins.
Sorry that I can't be of more assistance.







Title: Re: Results of Phase I trial of CPHPC + anti-SAP antibodies
Post by: ziad_okeil on April 08, 2015, 05:33:36 pm
Thanks a lot Miriam for your quick reply.
I will certainly consider your recommendation.

Regards,
Title: Re: Results of Phase I trial of CPHPC + anti-SAP antibodies
Post by: alisonjayne on April 12, 2015, 08:52:55 pm
This is such positive uplifting news. I think of you all facing each day with a great deal of bravery just like David did. I sincerely hope this drug will be there for you all as soon as is possible.  Ali. X