Author Topic: Future/Novel drugs  (Read 6802 times)

ziad_okeil

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Future/Novel drugs
« on: March 16, 2015, 10:44:15 am »
Good morning everyone,

I believe Miriam will be the best/only person, who will be able to answer this question since she is a doctor and undoubtedly well informed about this issue.
 
My question concerns the new drugs that are currently under study around the world. I know there are the NEOD001 from “Prothena” in the USA and the SAP-CPHCP complex (Anti-SAP) drug from GSK in the UK:

- While there is much more information available on the NEOD001 drug than on the Anti-SAP one, I have the feeling they both work (technically/medically) in the same way, is that true?

- Are they stand-alone drugs that dissolve the Amyloidosis deposits from the organs/tissues without the need of chemotherapy?

- If yes, does this mean that conventional Chemotherapy might be off the table in the future treatments? Would they be enough to turn Amyloidosis into just a nuisance that one can live with?

On the trial’s official WebPages (of both of them), there is always an expected completion date mentioned at the bottom of the page. If we add to that date the fact that both drugs have supposedly been fast-tracked and/or exempted from certain bureaucratic processes (FDA/EMA) because they aim to meet an unmet medical need, could we be hopeful that they could be released soon? 2016/17?

I am well aware that not all information are allowed to be shared due to the confidentiality agreements between the different medical institutions and pharmaceutical companies but I wish some information could be shared to a certain extent.

Thanks
Regards,

Ziad
 
« Last Edit: March 17, 2015, 11:50:44 am by ziad_okeil »
Ziad

Miriam Vered

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Re: Future/Novel drugs
« Reply #1 on: May 03, 2015, 09:30:49 am »
Hi Ziad,


Sorry it's taken me a while to reply.
Here are the answers to your questions, from Professor Sir Mark Pepys:


While there is much more information available on the NEOD001 drug than on the Anti-SAP one, I have the feeling they both work (technically/medically) in the same way, is that true?

Both treatments use monoclonal antibodies.  NEOD001 is a monoclonal antibody that was created to bind to AA amyloid but it also binds to some AL amyloid deposits.  The CPHPC + anti-SAP antibody treatment, invented by Sir Mark Pepys at the UCL Wolfson Drug Discovery Unit in the Centre for Amyloidosis and Acute Phase Proteins, specifically targets the SAP protein that is present in all amyloid deposits of all types.  The anti-SAP drug therefore should be active in all forms of amyloidosis.  As far as we know, NEOD001 is currently being tested only in AL amyloidosis.
 
We know in great detail what the mechanism of action is for our treatment, have published the full experimental results, and are about to publish the first clinical results.  Nothing comparable has been published about NEOD001.  We know nothing more about NEOD001 than is available publicly on the internet therefore I’ll answer your other questions just for the CPHPC + anti SAP approach.
 
Our approach, which was licensed to GlaxoSmithKline in 2009, is not just a combination of two drugs but is an obligate therapeutic partnership.  Both components are essential and the CPHPC must be administered first in order for the anti-SAP antibody to given subsequently.  This treatment has been in its first in human clinical trial since autumn 2013 and the results so far are extremely encouraging.  The trial has proceeded slowly and very cautiously because the treatment is totally novel and unprecedented, so that safety considerations were crucial.  In the event it has proved to be well tolerated, with no severe adverse events.  The first results were presented publicly at the annual meeting of the Association of Physicians on 27 March 2015.  The abstract of the presentation, which is attached here, will be published in the Quarterly Journal of Medicine, and also available online.  The first full formal report of the results has also lately been submitted for publication.
 
Are they stand-alone drugs that dissolve the Amyloidosis deposits from the organs/tissues without the need of chemotherapy?

The CPHPC + anti SAP antibody treatment promotes the clearance of amyloid deposits from the tissues but it will not prevent the build up of new deposits.
 
AL amyloidosis will definitely still require chemotherapy to suppress or eliminate the cells that produce the amyloidogenic light chains.  It is hoped that by improving and restoring, if possible, the function of organs damaged by amyloid, patients will be healthier and able to tolerate chemotherapy better and indeed survive long enough after the initial diagnosis to experience the benefits of chemo.  Ultimately the treatment should help to prevent organ failure and thus avoid the need for interventions such as dialysis and organ transplantation.
 
In all other types of amyloidosis it will also always still be necessary to reduce production of the amyloidogenic protein, if at all possible, to prevent accumulation of amyloid.
 
However even if such treatments are either unavailable or unsuccessful in any type of amyloidosis, repeated courses of anti-SAP should helpfully reduce the amyloid load and, we hope, maintain vital organ function.
 
If yes, does this mean that conventional Chemotherapy might be off the table in the future treatments? Would they be enough to turn Amyloidosis into just a nuisance that one can live with?

Chemotherapy will always be necessary in patients with AL amyloidosis, to prevent further build up of amyloid deposits.  But if the CPHPC + anti SAP antibody partnership works as envisaged, and if it becomes a licensed treatment, then we hope that it will maintain and restore organ function and keep patients in good health in an unprecedented fashion.
 
On the trial’s official WebPages (of both of them), there is always an expected completion date mentioned at the bottom of the page. If we add to that date the fact that both drugs have supposedly been fast-tracked and/or exempted from certain bureaucratic processes (FDA/EMA) because they aim to meet an unmet medical need, could we be hopeful that they could be released soon? 2016/17?
 I am well aware that not all information are allowed to be shared due to the confidentiality agreements between the different medical institutions and pharmaceutical companies but I wish some information could be shared to a certain extent.


The legal framework that governs introduction of new drugs is infinitely complex and rigidly restrictive so that the pace of development is painfully slow.  Nevertheless, the very effective synergistic collaboration of the Wolfson Drug Discovery Unit and National Amyloidosis Centre teams with the drug development experts of GSK, one of the world’s largest and most effective pharmaceutical companies, is aimed very energetically at bring the new medicine into the widest possible availability at the earliest possible time.  Everyone involved is well aware of the unmet medical need and how keenly patients wish to know when the new treatment will become available.  However we are still at too early a stage to be able to disclose any useful information.  Apart from anything else, more patients must be treated so that we know more about mechanisms, effects and benefits.  The necessary studies are being planned and we will provide information as soon as we possibly can.
« Last Edit: May 04, 2015, 08:15:14 am by Miriam Vered »

patpinchin

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Re: Future/Novel drugs
« Reply #2 on: May 03, 2015, 12:34:07 pm »
A big thank-you to Ziad for your important questions and to Miriam for such informative answers ahead of official publication.

I am sure that forum members will be grateful for Miriam's clarification of the development of Professor Sir Mark Pepys' remarkable treatment which promises to significantly improve the lives of sufferers.

It is truly encouraging that the trial so far has achieved better than expected results and i hope that future studies will continue to succeed.

Undoubtedly the close collaborative nature of the Wolfson Drug Discovery Unit, the NAC and GSK cannot be underestimated. Whilst the availability to sufferers of this combination drug cannot come quickly enough for all who suffer, the fast tracking designation of Sir Mark's groundbreaking treatment is the result of brilliant scientific work together with dogged determination to overcome the complex necessary restrictive legislative procedures. We are in truly skilled hands and have much to be grateful for.  :)
« Last Edit: May 03, 2015, 04:21:22 pm by patpinchin »
Pat

ziad_okeil

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Re: Future/Novel drugs
« Reply #3 on: May 04, 2015, 08:01:19 am »
Dear Miriam,

Thank you very much for the detailed answers to my questions. I really appreciate it.
Please send my thanks and gratitude to Sir Mark Pepys for his time and effort.

NB: In the reply there was a mention of an attachment of a presentation that for some reason I cannot find.

Thanks again
Kind regards,
Ziad

Miriam Vered

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Re: Future/Novel drugs
« Reply #4 on: May 04, 2015, 08:16:07 am »
Hi Ziad,


I've added the attachment now. It's at the bottom of my previous post.
Sorry about that.

ziad_okeil

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Re: Future/Novel drugs
« Reply #5 on: May 04, 2015, 08:32:20 am »
Thanks a lot Miriam...

regards,
Ziad