Denver Man Gets Gene Therapy to Restore Hearing

[Markku]

GenVec To Present At 2015 Stem Cell Meeting On The Mesa

GenVec, Inc. (NASDAQ: GNVC) announced that Douglas E. Brough, Ph.D., the company's chief scientific officer, will provide a brief review of GenVec's technology platform at the 2015 Stem Cell Meeting on the Mesa. This presentation will include highlights of the differentiated performance characteristics seen with GenVec's gorilla and monkey adenovectors, which offer advantages for multiple disease-fighting strategies such as nucleic acid delivery, genome editing and stem cell therapy.

The presentation is scheduled for Wednesday, October 7, beginning at 5:15 p.m. PDT/8:15 p.m. EDT. A live video webcast of all company presentations at the Stem Cell Meeting will be available at: http://stemcellmeetingonthemesa.com/webcast during the proceedings. In addition, GenVec will post a video replay of Dr. Brough's talk on the company website when it becomes available after the meeting concludes at www.genvec.com in the Investors & Media presentations collection.​

There's also a new presentation available (which is attached as a PDF to this post). The presentation includes topics such as the hearing loss trial design and clinical endpoints.

Credit to @attheedgeofscience for finding this.

 

[markoana]

GenVec To Present At 2015 Stem Cell Meeting On The Mesa

GenVec, Inc. (NASDAQ: GNVC) announced that Douglas E. Brough, Ph.D., the company's chief scientific officer, will provide a brief review of GenVec's technology platform at the 2015 Stem Cell Meeting on the Mesa. This presentation will include highlights of the differentiated performance characteristics seen with GenVec's gorilla and monkey adenovectors, which offer advantages for multiple disease-fighting strategies such as nucleic acid delivery, genome editing and stem cell therapy.

The presentation is scheduled for Wednesday, October 7, beginning at 5:15 p.m. PDT/8:15 p.m. EDT. A live video webcast of all company presentations at the Stem Cell Meeting will be available at: http://stemcellmeetingonthemesa.com/webcast during the proceedings. In addition, GenVec will post a video replay of Dr. Brough's talk on the company website when it becomes available after the meeting concludes at www.genvec.com in the Investors & Media presentations collection.​

There's also a new presentation available (which is attached as a PDF to this post). The presentation includes topics such as the hearing loss trial design and clinical endpoints.

View attachment 7928

Credit to @attheedgeofscience for finding this.

Does anyone @Markku @attheedgeofscience know why researches are not done more often on monkeys? They are far more similar to humans, brain, hearing etc.
I understand that working with mice is cheaper. But are there other impediments beside costs, some kind of animal rights or?

I was always thinking that researches would be at least 3 x faster if primates are used for experiments.

BTW. I love animals but it is about health, not cosmetics industry...

 

[Nucleo]

Does anyone @Markku @attheedgeofscience know why researches are not done more often on monkeys? They are far more similar to humans, brain, hearing etc.
I understand that working with mice is cheaper. But are there other impediments beside costs, some kind of animal rights or?

I was always thinking that researches would be at least 3 x faster if primates are used for experiments.

BTW. I love animals but it is about health, not cosmetics industry...

Ethical reasons.

https://en.wikipedia.org/wiki/Animal_testing_on_non-human_primates

 

[markoana]

...unless it is believed that the actions are essential to preserve the species or in relation to an unexpected outbreak of a life-threatening or debilitating clinical condition in human beings. EU Directive 2010/63/EU

Eighty-two percent of primate procedures in the UK in 2006 were in applied studies, which the Home Office defines as research conducted for the purpose of developing or testing commercial products.[38] Toxicology testing is the largest use, which includes legislatively required testing of drugs.[39] The second largest category of research using primates is "protection of man, animals, or environment", accounting for 8.9% of all procedures in 2006. The third largest category is "fundamental biological research,", accounting for 4.9% of all UK primate procedures in 2006. This includes neuroscientific study of the visual system, cognition, and diseases such as Parkinson's,[40] involving techniques such as inserting electrodes to record from or stimulate the brain, and temporary or permanent inactivation of areas of tissue.

Humans are unbalanced species. From non animal ethic and rights TO too restrictive-protective animal rights.

T. is definitely condition that should be tested od primates. Phase II would be far more predictable after Phase I done on primates

 

[Rob111]

Link to Dr. Staecker's recent talk on drug delivery methods to the inner ear:

http://media.corporate-ir.net/media...Drug Delivery To The Inner Ear - Staecker.mp4

 

[newarrior]

I just registered with Pioneer AND filled out the pre screen study form !

 

[newarrior]

Regardless of whether or not it works; the surgery was a success. They drilled a tiny hole using a laser in his stapes and then injected the drug through the oval window. The treatment method alone is ground breaking and we now have a safe way to access the cochlea directly.

AWESOME

 

[Zechariah]

Perhaps, but when Novartis is saying some people are hearing again(which has never happened before) then it is probable that the clinical trial is looking very good. Once improvements in hearing can be demonstrated, it will not be long before a treatment is made available.

I know it's easier to hope and think that "it won't be long" but the fact is that even in the best case scenario the treatment won't be available to general public in 5 years. If 5-10 years is "not long" for you - well then, carry on

 

[jeff W]

I know it's easier to hope and think that "it won't be long" but the fact is that even in the best case scenario the treatment won't be available to general public in 5 years. If 5-10 years is "not long" for you - well then, carry on

Yes, I think its wise to start saving a "t" fund.

 

[c0ldsun]

German news magazine Focus 49/2015 brought a title story about ear diseases and they also mentioned the Genvtec trail with Rob Gerk. But nothing that we already knew was reported: there are 45 ppl in the trail, Rob can hear new sounds and they expect results in 2 years.
However there was one interesting experiment mentioned from Hey-Kyoung Lee at
Johns-Hopkins-University: blind ppl compensate their blindness with better hearing, so the brain activicty will focus on hearing. They made an experiment with mices that they put them for one week in a dark room. Afterwards the hearing improved. They want to show that the brain is still teachable and can adapt to new circumstances. They plan to make a similar study with humans. Interesting approach.

 

[jeff W]

German news magazine Focus 49/2015 brought a title story about ear diseases and they also mentioned the Genvtec trail with Rob Gerk. But nothing that we already knew was reported: there are 45 ppl in the trail, Rob can hear new sounds and they expect results in 2 years.
However there was one interesting experiment mentioned from Hey-Kyoung Lee at
Johns-Hopkins-University: blind ppl compensate their blindness with better hearing, so the brain activicty will focus on hearing. They made an experiment with mices that they put them for one week in a dark room. Afterwards the hearing improved. They want to show that the brain is still teachable and can adapt to new circumstances. They plan to make a similar study with humans. Interesting approach.

 

[PRCRebel]

Some random new guy here...

Saw this little tidbit from the quarterly report three weeks ago:

"Dosing of the third cohort in the Phase 1/2 trial of CGF166 in patients with severe to profound hearing loss is underway and we are encouraged with the progress being made in the trial," said Doug Swirsky, GenVec's president and CEO.

http://www.prnewswire.com/news-rele...quarter-2015-financial-results-300174406.html

"Cohort" can be interpreted as a friend/companion or a group of people so I can only guess they've finished either the third individual or collaboration of people at this time.

 

[RB2014]

from what I remember reading, they were breaking up the testing in 3 groups. Each group gets a higher dosage. I think the third group or "cohort" was the group that got the highest dosage assuming the first two groups didnt have any adverse side effects.
This is good news though and he seems pretty positive about the results. I would like to think that while they are performing these trials, they are further perfecting the formula back at home so maybe more tests are to come.

Jeff, thats an interesting point. I wonder what they mean by better hearing though. I bet they mean better understanding. I don't think their actual hearing improves, just their ability to analyze speech. if they can stop their hearing from deteriorating though that is something cool. I wonder what mechanism triggers that and maybe we can reproduce it somehow.

 

[rsmcfar]

The niece of one of the patients has updated her blog about the clinical trial. He provides an interesting description of what is going on in his inner ear.

http://2030something.me/

 

[attheedgeofscience]

Latest corporate overview from GenVec attached (December 2015).

 

[Nucleo]

Latest corporate overview from GenVec attached (December 2015).

Interesting that they test hearing sensivity up to 16 khz (slide 19). Almost no audiological facilities can/will do such test.

I'm also wondering if they will test otoacoustic emissions. It was not mentioned in the presentation.

 

[Reinier]

I am surprised that testing up to 16 kHz is not standard procedure nowadays. Especially if the patient complains about T.
I am certain that after my acoustic trauma, frequencies higher than the tested 8 kHz are also damaged. The explosion I was exposed to sounded really high pitched.
Also I read that the cochlea is more sensitive for damage in frequencies higher than 8 kHz because the haircells for these frequencies are the first haircells in the cochlea.
I read in documents that testing above 8 kHz is done more all the time.
Also I wonder if in this clinical test scientists want to know if the injected drug is reaching all sections of the cochlea?

 

[Reinier]

Friday a test was done for a CR neuromodulator. The test was up to 16 kHz.
And for sure my damaged ear where I experience T most is also damaged above 8 kHz. Surprise surprise.
The audiologist told me one reason is that test equipment for the higher frequencies is much more expensive??

 

[spingee]

http://lifescienceinstruments.com/103-thickbox_default/infographic-cochlear-place-frequency-map.jpg
As you can see 8khz+ is in like 5% of cochlea lenght, its not that significant.
Anyway what would it help you if you know that it is damaged? There is still no way to repair it.

 

[jeff W]

http://lifescienceinstruments.com/103-thickbox_default/infographic-cochlear-place-frequency-map.jpg
As you can see 8khz+ is in like 5% of cochlea lenght, its not that significant.
Anyway what would it help you if you know that it is damaged? There is still no way to repair it.

Very on point that.

 

[Reinier]

As you can see 8khz+ is in like 5% of cochlea lenght, its not that significant.
Anyway what would it help you if you know that it is damaged? There is still no way to repair it.

I agree. Not able to repair. But this was just about understanding what could be going on regarding T and hearing damage in general. And science has got to start understanding what is damaged before they can try to device a way to repair it in future (repair we sincerely hope). Interesting "infograph" by the way.
As a teenager I used to hear slightly higher than 19 kHz. Oh.... I almost forgot. I used to be able to run longer distances (-;

 

[rsmcfar]

The trial has been paused for safety reasons. This is in an SEC filing by Genvec. No other details were provided.

 

[Reinier]

The trial has been paused for safety reasons. This is in an SEC filing by Genvec. No other details were provided.

To me that sounds like bad news. Or is this something that is not unusual during trials?
Hopefully nothing serious with one of the participants.

 

[Reinier]

I found this on the internet:

" On January 8, 2016, Novartis notified GenVec that it was pausing enrollment in the clinical trial of CGF166 to conduct a safety review of patient data, based on the recommendation of the trial's data safety monitoring board. The trial will continue to collect new safety and efficacy data on patients currently enrolled in the study."


I also found this: http://finance.yahoo.com/news/shareholder-alert-bronstein-gewirtz-grossman-175500268.html

I am completely new to this stock holders/financial world. But a shareholders alert and falling shares doesn't sound promising.

I am just curious if there is someone on this forum that is able to understand if this is happening all the time or whether this is unusual.

Perhaps I am opening a hornets nest?

 

[Nucleo]

GNVC started around 100$ in 2001. It is now a penny stock.

I don't know what any of this means though.

 

[Reinier]

This is a part from the article:
quote:
"This investigation concerns whether GenVec and certain of its officers and/or directors have violated the Federal Securities Laws under the Securities Exchange Act of 1934 (the "Exchange Act")."
End quote.

This has something to do with stock trading and not with the gen technology if I am correct.
After this news on Monday, stock price opened 35% lower than closing price on Friday.
Surely there is someone on this forum that can make more sense of the financial world/processes than I can.
I hope this does not mean that the trials with Novartis could stop. That would really be a disappointment since it is the first trial that is investigating hair cell regrowth in the cochlea as far as I am aware.

 

[jdjd09]

Can someone please clarify what is going on with this trials? It's been over a month. Is this still going on? I know a lot of people on here are in contact with researchers. What is going on with this?

 

[jeff W]

Can someone please clarify what is going on with this trials? It's been over a month. Is this still going on? I know a lot of people on here are in contact with researchers. What is going on with this?

They upped the dosage to look for increased effect in a new trial participant, there must have been a complication, ie headaches or dizziness or something (exactly what is not specified) Swirsky bought more shares before the safety pause, so its not like they are burning the shareholders, its a minor complication in the first ever trial delivering a target agent to the inner ear IN HISTORY. Planes fly smoothly now but at the beginning they were a little bumpy.

 

[RB2014]

To bad they can't ask the lab rats they previously tested how dizzy they were after each dosage.

 

[jeff W]

To bad they can't ask the lab rats they previously tested how dizzy they were after each dosage.

I think we should pool cash together on here and nominate a broker to invest it into cutting edge trials. we could choose the trials that push technology the furthest as opposed to ones with the lowest risk/best return