Author Topic: New England Journal published results of Phase I trial of CPHPC+anti SAP  (Read 9360 times)

Miriam Vered

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The results of the phase 1 trial of CPHPC +anti SAP antibodies were published today in the prestigious New England Journal of Medicine-


http://www.nejm.org/doi/full/10.1056/NEJMoa1504942


I posted Professor Sir Mark Pepys' explanation of these results when these results were presented at the Annual Meeting of the Association of Physicians in March. See the topic titled "Results of Phase I trial of CPHPC +anti SAP antibodies.




patpinchin

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Thank-you for posting this Miriam. Very good to read this paper published in a prestigious US medical journal. It wil enable all forum members to see how very well regarded the outstanding work in drug development of Professor Sir Mark Pepys' and his team is beyond our shores.  Thank-you for making it available on our forum as to my knowledge there hasn't been anything in the UK press? Does this suggest that the opening of the phase 2 trial is on the way?
« Last Edit: July 16, 2015, 10:23:44 am by Miriam Vered »
Pat

Miriam Vered

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Pat, the New England Journal is internationally highly renowned, not just in the US.
The Financial Times had an article about it today, I'll post the link later.

Plans for the Phase II trial are in progress. I'll post here when I can give any further information about it.

georgeturner

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Miriam,

Thank you for making this article available on the forum. It's very encouraging to read in more detail about the results of the phase 1 following on from Sir Mark's explanation in your earlier post. Sir Mark and his team of developers have given us all much hope. To them and the patients who have volunteered to take part in this trial, many thanks.
Looking forward to more success with the phase 2.
Slainte Mhath,
George

patpinchin

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I read the very interesting article to which Miriam refers below, in yesterday's Financial Times but have found that the link will not open it more than once. It featured the outstanding and dedicated work of over 40 years, for amyloidosis of Professor Sir Mark Pepys leading to the development of his anti-body drug. There was a lovely photo too of Sir Mark with his charming smile and twinkly eyes. The headline of the article was disappointingly not about the progress of his drug for amyloidosis, but of it wider application as a possible new treatment for Alzeihmer's which is of greater public interest. It mentioned the possibility of a trial for 70 Alzeihmers' patients next year. This is wonderful news as Alzeihmer's is a global catastrophe and obviously it is an exciting development and must go ahead at the earliest opportunity. However if you are an amyloidosis patient with 13 years of chronic suffering and no symptom control, it feels rather frustrating that you are likely to to be ineligible for phase 2 of the amyloidosis trial.  :(
« Last Edit: July 17, 2015, 03:12:42 pm by patpinchin »
Pat

Miriam Vered

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The Financial Times piece is attached in the PDF here, or available online






patpinchin

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I have found the first link that Miriam posted opens in Chrome, iBooks and Kindle books. You can email it from the last two, to interested friends/famiy who are not forum members.

The second link opens the document for a few seconds then unfortunately goes to the suscription page and after that a différent article appears.
« Last Edit: July 24, 2015, 09:49:50 am by patpinchin »
Pat

patpinchin

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Hi Miriam,
I noticed a couple of details in the FT article which I would like you to confirm or correct please.

It said that phase 2 of the trial will open next year. This disappointed me as Professor Hawkins said at the Infoday in Sept 14 that it was hoped that phase 2 would open "next year". ie: 2015.

It also said there are 8,000 sufferers in the UK. I thought it was about 3,300? Perhaps 8,000 referred to the annual number of appointments at the NAC?
Pat

patpinchin

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Another very good article. Easy to understand. Link can be emailed.

http://medicalxpress.com/news/2015-07-treatment-amyloidosis.html
« Last Edit: July 24, 2015, 09:50:29 am by patpinchin »
Pat

Miriam Vered

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Hi Miriam,
I noticed a couple of details in the FT article which I would like you to confirm or correct please.

It said that phase 2 of the trial will open next year. This disappointed me as Professor Hawkins said at the Infoday in Sept 14 that it was hoped that phase 2 would open "next year". ie: 2015.

It also said there are 8,000 sufferers in the UK. I thought it was about 3,300? Perhaps 8,000 referred to the annual number of appointments at the NAC?


Hi Pat,
Here are Sir Mark's answers to your questions:

Planning and conduct of clinical trials of new drugs are extremely complex, challenging and costly activities. Predicting their timing and duration is therefore very difficult and is rarely precise. The phase I part B study is ongoing and continues to give encouraging results. A phase II study is being planned with the intention of starting early in 2016 and lasting about a year but until both the plans and all the essential preparations are complete there can be no certainty about these timings. Despite this unavoidable uncertainty I can assure you that both I and GSK are working as hard as possible to progress the development programme.


There is no definite information about the numbers of patients with systemic amyloidosis in the UK or elsewhere. There is also no way to obtain such information reliably.  Any numbers quoted by us or others are therefore only estimates based, at least in our case, on the best information and experience available. We believe there may be at least 3,000 patients with diagnosed systemic amyloidosis in the UK who are known to the NAC and perhaps as many again either unknown to us or not diagnosed. Others may have different estimates.




patpinchin

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Many thanks Miriam and Sir Mark for your detailed reply.

I am very pleased to hear that phase 1b is going well with continuing encouraging results. Thank- for the information for phase 11. It helps to be aware of ongoing plans. There is no doubt in my mind that you in partnership with GSK are trying your utmost to progress the development of your treatment. I am sure I write on behalf of many other sufferers who are all deeply appreciative of your personal efforts and those of many others.

Very interesting statistics. How fortunate are those of us referred, diagnosed, advised and monitored by the NAC. I had no idea there could be as many sufferers in the UK unknown to you.
Pat