PBT2
Posted by CJ878
Does anyone know how promising this drug really is? I found this article [www.newshd.net] that states the drug can directly affect the HTT Protien. On a similar note I heard through a press relase that Sangamo will be attempting to file an IND application for research with Zinc Fingers and Huntington's by 2015. So that is positive news.
Thanks for opinions and knowledge on PBT2.
Thanks for opinions and knowledge on PBT2.
There is a press release on Prana's website dated 7/23/2013. Here are the details:
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Prana Completes Phase 2 Huntington Disease Trial with PBT2
Prana Biotechnology today announced the successful completion of Reach2HD, a phase 2 clinical trial in patients with early to mid-stage Huntington disease. The Company expects to announce the results arising from the trial in October.
Reach2HD is a randomised, double-blind, placebo-controlled Phase 2 trial testing the safety and efficacy of PBT2, the Company’s lead compound under development for both Huntington disease and Alzheimer’s disease.
“We have been extremely pleased with the conduct of the trial, at all levels including recruitment and patient retention,” said Dr Ray Dorsey, Principal Investigator of the Reach2HD study and Director of the Huntington Study Group Coordination Center.
Reach2HD had planned to recruit 100 patients with Huntington disease in 9 months. In fact, 109 participants were enrolled in the trial within that period. “The strong rate of recruitment reflects support for the Reach2HD trial within the Huntington disease clinical research community,” said Dr Dorsey. Of the 109 enrolled, 104 patients completed the trial, reflecting a retention rate of over 95%.
A Data Safety Monitoring Board met on five occasions throughout the trial and on each occasion recommended that no changes or modifications to the study protocol be made based on their review of the safety data.
The primary outcome of the trial is safety and tolerability. The trial also includes a number of secondary outcome measures from the cognitive, motor and behavioural domains affected in Huntington disease. A positive result of Reach2HD will identify signals of therapeutic benefit in one or more of the domains measured, which will inform the design of the next clinical trial.
Mr Geoffrey Kempler, Prana’s Chairman and CEO, said: “assuming we achieve the positive results we are hoping for in Reach2HD, we plan to meet with the US regulator, the Food and Drug Administration, and other regulatory agencies to discuss the next steps in the clinical development of PBT2 for the treatment of Huntington disease.”
“We plan to discuss the design of the next trial and agree on a set of clinical outcomes that, when achieved, will allow us to submit a New Drug Application for approval to start to market PBT2 for Huntington disease.”
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You can read the full release on their website: [www.pranabio.com]
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Prana Completes Phase 2 Huntington Disease Trial with PBT2
Prana Biotechnology today announced the successful completion of Reach2HD, a phase 2 clinical trial in patients with early to mid-stage Huntington disease. The Company expects to announce the results arising from the trial in October.
Reach2HD is a randomised, double-blind, placebo-controlled Phase 2 trial testing the safety and efficacy of PBT2, the Company’s lead compound under development for both Huntington disease and Alzheimer’s disease.
“We have been extremely pleased with the conduct of the trial, at all levels including recruitment and patient retention,” said Dr Ray Dorsey, Principal Investigator of the Reach2HD study and Director of the Huntington Study Group Coordination Center.
Reach2HD had planned to recruit 100 patients with Huntington disease in 9 months. In fact, 109 participants were enrolled in the trial within that period. “The strong rate of recruitment reflects support for the Reach2HD trial within the Huntington disease clinical research community,” said Dr Dorsey. Of the 109 enrolled, 104 patients completed the trial, reflecting a retention rate of over 95%.
A Data Safety Monitoring Board met on five occasions throughout the trial and on each occasion recommended that no changes or modifications to the study protocol be made based on their review of the safety data.
The primary outcome of the trial is safety and tolerability. The trial also includes a number of secondary outcome measures from the cognitive, motor and behavioural domains affected in Huntington disease. A positive result of Reach2HD will identify signals of therapeutic benefit in one or more of the domains measured, which will inform the design of the next clinical trial.
Mr Geoffrey Kempler, Prana’s Chairman and CEO, said: “assuming we achieve the positive results we are hoping for in Reach2HD, we plan to meet with the US regulator, the Food and Drug Administration, and other regulatory agencies to discuss the next steps in the clinical development of PBT2 for the treatment of Huntington disease.”
“We plan to discuss the design of the next trial and agree on a set of clinical outcomes that, when achieved, will allow us to submit a New Drug Application for approval to start to market PBT2 for Huntington disease.”
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You can read the full release on their website: [www.pranabio.com]
Update: trial results expected early next year.
From a Prana press release:
The Reach 2HD trial is a six month double-blind placebo controlled Phase 2 trial on 109 early-to-mid stage Huntington’s disease patients. The trial was successfully completed at the end of July 2013 with 95% of participants completing the entire six months of treatment. There has been a delay in finalising the database to achieve ‘database lock’, required before statistical analysis of the data may begin. The results, originally anticipated in the last quarter of 2013, are now expected to be reported early in 2014.
Source: [www.pranabio.com]
From a Prana press release:
The Reach 2HD trial is a six month double-blind placebo controlled Phase 2 trial on 109 early-to-mid stage Huntington’s disease patients. The trial was successfully completed at the end of July 2013 with 95% of participants completing the entire six months of treatment. There has been a delay in finalising the database to achieve ‘database lock’, required before statistical analysis of the data may begin. The results, originally anticipated in the last quarter of 2013, are now expected to be reported early in 2014.
Source: [www.pranabio.com]
This video is about a patient in the Alzheimer's trial of PBT2. There is some confusion about which trial is being described in the video but the patient has been confirmed by the company to be in the PBT2 trial. Their story is quite uplifting.
[www.youtube.com]
[www.youtube.com]
From what I had gathered, and I follow these trials based on the AD and dementia that many of my family relatives (aunts, uncles, grandparents) have suffered, the journalists pieced together comments that were more general - some applying to the SOLA trials, such as the 2000 participant trial under recruitment for SOLA, and the 30 percent reduction in rate of decline statistic also fits one sub-group only of the SOLA trial with other groups not showing improvement - so that statistic also should not be attributed to PBT2. There are actually six dementia related trials going on in that hospital the news piece related to, but it is one of the IMAGINE trial locations testing PBT2 in AD patients.
You are correct, as Prana put out a statement that they were not involved in this news piece or interview. In that public disclosure to the ASX and NASDAQ stock exchanges, they did confirm that the interviewed person was an IMAGINE trial participant testing PBT2, and of course, that results would not be known until all data is analyzed and unblended in March 2014.
The testimonial is very hopeful from my perspective, and I hope it provides benefit also for Huntington's Disease.
The other quite positive testimonial that I enjoyed specifically some time back regarding REACH2hd was the Dr. Jody Corey-Bloom 2012 Research update, where she relayed the enjoyment that their largest trial participant group there in San Deigo was having with the trial. I believe that likely involves some on this forum. On October 29 she just came out with another research update that also was an excellent 2 part video. It is amazing the progress they make in a year on the testing and evaluation front, and it was extremely educational for me on the differences between Huntington's and AD on both Symptoms, and areas affected by Huntington's Disease. Her latest research update makes no mention of the REACH2HD trial as the trial is over and results are being analyzed. It is likely an appropriate time not to give any indication either way.
I know not a lot has been said (rightly so) about the trial from those who were in the study, or a study partner, but I would like to say thanks to those who did participate.
In following the various AD trials, it is amazing the amount of independent scientific research that has come out in support of metals playing a key part in the pathology of both diseases, and I hope that PBT2 proves successful.
Just weeks ago a new study result came out, where different from prior tests on transgenic mice (AD or HD pathology), they tested PBT2 on normal aged mice (not having amyloid plaques) and the results were astounding, with one statement in the synopsis interpreted as providing a beneficial application in Huntington's Disease, which I attempted to Bold below.
There seems to be so much additional scientific research implicating metals in the pathology, that I still personally hold out a lot of hope, and I look forward to 2014 when results are finally known.
[finance.yahoo.com]
Synopsis of the Aging Cell publication
Typically mice live for 24 to 30 months, developing progressive cognitive impairment from 16 to 18 months. Age related cognitive decline is associated with measurable structural and biochemical changes in the brain, which were significantly improved by PBT2. In the study 22 month old mice were treated with PBT2 for a total of 12 days.
PBT2 restored learning and memory. The old mice treated with PBT2 performed learning and memory tasks to the same level exhibited by young mice and significantly better than untreated old mice (p < .01 or better).
PBT2 Increases markers of neurogenesis and neuron number:
a) Increased number of mature neurons by up to 27% in the hippocampus
b) Increased markers of cell proliferation by 67% and markers of numbers of immature neurons by 130% in the hippocampus.
c) Neuronal proliferation markers were elevated around the lateral ventricles by 214% (atrophy of peri-ventricular tissue is a feature of Huntington's disease)
PBT2 increases numbers of synapses in the hippocampus:
a) Synaptophysin levels increased by 38%
b) Dendritic spine density increased by 15%
PBT2 increases glutamate receptor levels in the hippocampus:
a) NMDA R2b levels increased by 88%
b) AMPA levels increased by 97%
PBT2 increases Protein Phosphotase 2a (PP2a) in the hippocampus:
a) PP2a increased by 22%
b) Phosphorylated Tau levels decreased by 81%
* Adlard et al, A Novel Approach To Rapidly Prevent Age-Related Cognitive Decline
You are correct, as Prana put out a statement that they were not involved in this news piece or interview. In that public disclosure to the ASX and NASDAQ stock exchanges, they did confirm that the interviewed person was an IMAGINE trial participant testing PBT2, and of course, that results would not be known until all data is analyzed and unblended in March 2014.
The testimonial is very hopeful from my perspective, and I hope it provides benefit also for Huntington's Disease.
The other quite positive testimonial that I enjoyed specifically some time back regarding REACH2hd was the Dr. Jody Corey-Bloom 2012 Research update, where she relayed the enjoyment that their largest trial participant group there in San Deigo was having with the trial. I believe that likely involves some on this forum. On October 29 she just came out with another research update that also was an excellent 2 part video. It is amazing the progress they make in a year on the testing and evaluation front, and it was extremely educational for me on the differences between Huntington's and AD on both Symptoms, and areas affected by Huntington's Disease. Her latest research update makes no mention of the REACH2HD trial as the trial is over and results are being analyzed. It is likely an appropriate time not to give any indication either way.
I know not a lot has been said (rightly so) about the trial from those who were in the study, or a study partner, but I would like to say thanks to those who did participate.
In following the various AD trials, it is amazing the amount of independent scientific research that has come out in support of metals playing a key part in the pathology of both diseases, and I hope that PBT2 proves successful.
Just weeks ago a new study result came out, where different from prior tests on transgenic mice (AD or HD pathology), they tested PBT2 on normal aged mice (not having amyloid plaques) and the results were astounding, with one statement in the synopsis interpreted as providing a beneficial application in Huntington's Disease, which I attempted to Bold below.
There seems to be so much additional scientific research implicating metals in the pathology, that I still personally hold out a lot of hope, and I look forward to 2014 when results are finally known.
[finance.yahoo.com]
Synopsis of the Aging Cell publication
Typically mice live for 24 to 30 months, developing progressive cognitive impairment from 16 to 18 months. Age related cognitive decline is associated with measurable structural and biochemical changes in the brain, which were significantly improved by PBT2. In the study 22 month old mice were treated with PBT2 for a total of 12 days.
PBT2 restored learning and memory. The old mice treated with PBT2 performed learning and memory tasks to the same level exhibited by young mice and significantly better than untreated old mice (p < .01 or better).
PBT2 Increases markers of neurogenesis and neuron number:
a) Increased number of mature neurons by up to 27% in the hippocampus
b) Increased markers of cell proliferation by 67% and markers of numbers of immature neurons by 130% in the hippocampus.
c) Neuronal proliferation markers were elevated around the lateral ventricles by 214% (atrophy of peri-ventricular tissue is a feature of Huntington's disease)
PBT2 increases numbers of synapses in the hippocampus:
a) Synaptophysin levels increased by 38%
b) Dendritic spine density increased by 15%
PBT2 increases glutamate receptor levels in the hippocampus:
a) NMDA R2b levels increased by 88%
b) AMPA levels increased by 97%
PBT2 increases Protein Phosphotase 2a (PP2a) in the hippocampus:
a) PP2a increased by 22%
b) Phosphorylated Tau levels decreased by 81%
* Adlard et al, A Novel Approach To Rapidly Prevent Age-Related Cognitive Decline
Today Prana Biotechnology announced that dosing has completed in the IMAGINE Alzheimer's trial for prodromal/mild AD achieving a 95% retention rate throughout this trial - about the same is in REACH2HD. I believe on average, phase II trials normally retain 69/70 percent.
It is the same compound (PBT2) that was used in the six month REACH2HD trial for Huntington's Disease, with the exception that it was a full one year trial at either placebo or the highest 250 Mg dose that was also a component of the REACH2HD trial (placebo, 100 Mg, 250 Mg).
Prana had previously announced that all but 5 participants elected to continue into the additional 1 Year open label extension to the IMAGINE trial announced in July, where all participants electing the extension (even prior placebo patients) will be dosed on PBT2 at the max 250 Mg dosage for an additional year.
In Prana's recent annual meeting they emphasized the extension was "physician requested". They also explained in a prior release that there was a five month lag between the end of the dosing an the announcement of the Open Label Extension for those five that elected not to continue, and it seems they have since then retained 100 percent of participants that have completed the IMAGINE trial with the ability to continue straight into the Open Label extension trial - that of this group all, or nearly all have elected to do so.
I am hopeful for a beneficial outcome in REACH2HD and IMAGINE trials.
It is the same compound (PBT2) that was used in the six month REACH2HD trial for Huntington's Disease, with the exception that it was a full one year trial at either placebo or the highest 250 Mg dose that was also a component of the REACH2HD trial (placebo, 100 Mg, 250 Mg).
Prana had previously announced that all but 5 participants elected to continue into the additional 1 Year open label extension to the IMAGINE trial announced in July, where all participants electing the extension (even prior placebo patients) will be dosed on PBT2 at the max 250 Mg dosage for an additional year.
In Prana's recent annual meeting they emphasized the extension was "physician requested". They also explained in a prior release that there was a five month lag between the end of the dosing an the announcement of the Open Label Extension for those five that elected not to continue, and it seems they have since then retained 100 percent of participants that have completed the IMAGINE trial with the ability to continue straight into the Open Label extension trial - that of this group all, or nearly all have elected to do so.
I am hopeful for a beneficial outcome in REACH2HD and IMAGINE trials.
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