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Anecdotal stories can be dangerous. While I have heard some positive stories about this drug, at this time it is just that, stories. When the Dr's come out with empirical data that supports improvement then the stories become facts.

I will agree with the fact that the FDA does drag these things out and make it so difficult and such a long process to prove safety and effectiveness that it is very difficult for small or start up companies to survive the research and trial phase without going broke.
 
trfgev,

and the people on the website 'patiens like us'.

there are maybe 30 patiens talking about dex trial,are they all violating the agreement ( go the site and, lots of positive about the dex trail.

ikki,

I've seen it there. I give it no more credibility than I do any other gossip I read on the ’Net.

I will agree with the fact that the FDA does drag these things out and make it so difficult and such a long process to prove safety and effectiveness that it is very difficult for small or start up companies to survive the research and trial phase without going broke.

Given the number of drugs that make it through the current process only to be recalled later because they cause harm over the long term, I surely don't want to make it easier for those kinds of time bombs to make it through the system.

The solution for those small and startup companies is to partner with larger companies who have the resources to make it through the lengthy trial period. If you want to blame the FDA process, then you should consider blaming the greed of the folks running those companies who want the big personal payday at the end.
 
The FDA does allow fast tracking for drugs that have very promising phase 2 data and are for chronic debilitating illnesses.
The study I have just completed for MS has been " fast tracked" . This still takes years!
I agree with tfogey, god help us if drugs whizz through the process, people will be at the mercies of inscrutable companies.
Lamotrigine was going to be the next best thing for MND many years ago. It didn't work. Let the clinical trial run it's course. If it's positive, then would be the time to consider compassionate use. If they do an interim analysis as i hope they will, then it will give a good idea of the drugs efficacy.
Biogen is a company that is renowned for it's compassionate attitude to drug development.
 
Trfogey you make good points but I can not totally agree with you. Many big companies do not want to in invest in drugs for diseases like ALS because the ALS is so far away and since we are a small population there is not a lot of revenue to be generated. I will speak about the trial I am in. The Neuralstem CEO Richard Garr along with the Chief Scientific Officer Karl Johe over a decade ago because Richard's son had a brain tumor and while his son is still alive, Richard wanted more options for his son and treatment. Richard was a successful lawyer and Karl Johe's son was in Garr's son's class. They invested a lot of their money have since secured more investors and become a public company, I feel I can honestly say that greed is not behind their motivation to make this successful.
I will also say that for the trial I am involved in a independent safety review and monitoring board consisting of Dr's and researchers approved the trial to progress to patients 13 - 18 back in June. The FDA given the same information did not give their approval until the end of October and in August asked additional questions about the mice and pigs despite having that info prior to patient #1's surgery and despite the fact they now had data on 12 people.

I will also point to Geron a company who was running a stem cell trial on people with spinal cord injuries. Despite the fact that the Dr in charge of conducting the trial saying that that thus far the trial was a home run from a safety stand point they announced yesterday they were shutting the trial down. The reason they stopped it had become too expensive to fund and they decided to consolidate their funds to 2 cancer studies they were conducting which were farther along.

I am not saying that the FDA should be eliminated. They are a necessary evil. I say evil because the process is cumbersome. Certainly there are unscrupulous companies and Dr's that need to be policed. Yet the process is tedious and at times excessively long. I also understand that this is how our system for discovering new therapies “protects” us from unnecessary risk and harm. However I ask what good is protection if you protect people to death?
 
This is from Dr. Paul Knopfler's blog. He runs a stem cell lab for UC davis. While nothing earth shattering here, I think he frames both sides of the argument very well.
Is it fair to tell a patient to be patient? Can we develop cures at warp speed?
I talk to people about stem cell science and the timeline for turning data into treatments and cures, their reactions completely depend on whom they are.

Scientists are patient, perhaps too patient….perhaps too understanding of the many years that we are told that science takes to get something to the clinic.

Patients and patient advocates are understandably less patient.

I think on both sides, patients and scientists, we have things to teach each other about the importance of time.

As both a scientist and a cancer patient/survivor, I kind of have one foot in each boat as the old expression goes. I can see both sides.

From the scientist’s perspective, we think we have no choice but to be patient. The current reality dictates that pre-clinical studies and clinical trials together take many years or decades. We are taught that his reality and that to try to speed this up would inevitably mean taking shortcuts that compromise safety. Is that the only reality?

From a patient’s perspective, time is totally different. For many, time is not on their side and they cannot wait many years or decades. When I was first diagnosed with prostate cancer, everyone was grim based on the histology of the biopsy. Although I am doing great and in remission for almost 2 years now, I know that could change and it could come back. Especially in the first months after my diagnosis, my research found that there are essentially no treatments for advanced prostate cancer. I don’t have that form of the disease at this point (and hopefully never will) but that is a disturbing reality that no treatments exist and that making new ones could take longer time than I might have.

Scientists can help patients understand the temporal nature of science is measured in years and why.

Patients can help teach scientists the urgency needed to move as fast as we safely can and to balance risk with reward.

High risk or life altering medical conditions justify higher risk forms of advancing clinical trials more rapidly.

If a hypothetical patient has only about 1 year to live or even 5 years to live, that patient’s perspective time is radically different than everyone else including his/her physicians and the scientists working on research that might provide a treatment or cure. Their perception of risk is also very understandably different.

When I was a kid I was a big Star Trek fan and I loved it when the Enterprise went into warp speed, seemingly defying the laws of physics to go faster than the speed of light and explore space.

I would ask is there some we can warp the timeline of developing cures without compromising safety?

What do you think?

We need to think about such reality challenging questions, particularly in light of the coming explosion in stem cell tourism.
 
Bottom line Trfogey is you may be satisfied and defend the status quo but I think there is always room for transformation...
Another relevant blog from Dr. Knoepfler:

Big tent team science: new ideas about clinical trials

As a stem cell scientist I hear all the time about how the pipeline from the lab to the clinic is incredibly slow and terribly inefficient. Meanwhile millions of people have conditions that the current state-of-the-art medicine cannot effectively treat. Somehow we need to make a fundamental change to improve this process. I think one aspect of this change is a new kind of team effort whereby MDs, PhDs, funding agency officials, regulatory officials, and critically patients are brought together earlier in the process and follow it through each step. This is big tent team science.

From the time a scientist identifies a potential target or the basis for a new drug in the lab until a patient receives a therapy based on that finding, assuming success clinical trials, is often mentioned as 25 years. Further, we hear that >95% of the time potential development of such possible therapies fail. Often times promising drugs also are dropped from further investigation not for scientific reasons, but rather for financial ones.

These are roadblocks to making new therapies that are urgently needed, but how do we overcome them without compromising our standards for safety and efficacy? We can see how dangerous it is with stem cell tourism if the current system involving the FDA is bypassed by people trying to make money from patients. Ideally there should be a way to make clinical trials faster, but it is not clear how to speed up the process without in parallel increasing risk. One inherent obstacle to speeding up trials of drugs for human use is that they must be tested in people and the trials must allow for enough time to study safety and efficacy in a manner that has relevance to the timeline of potential future patients who would receive such drugs. Thus, the timeline for trials has to be measured in years, not in shorter increments such as months. I think it is crucial that patients and patient advocates play a role in helping scientists, doctors, and policy makers understand and balance the importance of risk, meaning the potential for harm from side effects to patients. For certain conditions, such as those that cause lifelong disability of have extremely high mortality rates, perhaps a higher level of risk that might accompany a faster clinical trial process is acceptable. To some extent this acceleration can happen even today, but not to the extent that many patients view as essential.

Beyond time, of course success rate is crucial. Speeding up the clinical trial process will do no good, if the failure rate is still in the 90+% range and it is even possible that a faster clinical trial process will increase the failure rate. That a key question is how can we use creative ways to boost our success rate at least into the double digits in terms of how often candidate drugs become realized therapies? Do we need to change our model system of how we develop candidates in the first place or somehow include a filtering process to make our pool of candidates stronger?

One potential way to help both speed the process and increase its efficiency is to take a somewhat of a novel team approach whereby we bring together MDs, PhDs, and patients along with funding agency officials and regulatory officials earlier on in the process. In particular I think there is right now an enormous distance between PhDs and patients that is harmful to the process. One reason for this distance is an illogical cultural aspect of science whereby most PhD scientists avoid interacting with people who are not scientists such as patients. We need to change that as patients and patient advocates have a tremendously powerful perspective that PhDs and also MDs to some extent cannot simply try to imagine in their own minds. In addition, PhDs have a unique perspective that is important as well that oftentimes MDs may not inherently bring to the table themselves. For example, PhDs view clinical trials from their own perspective as experiments and are not surprised when they fail relatively often because that’s simply the way it is with experiments.

We hear a lot of talk about team science these days, but the biomedical team I envision is not just a grouping of different scientists, but also including non-scientists. I call it the “big tent” approach to team translational and clinical science. Nothing is a panacea and improving the clinical trials process is no easy task, but I think working together we are more powerful than working apart.
 
First, TedH5, I'm not defending the status quo at the FDA. I have no doubt that there is the same bureaucratic shenanigans going on there as are going on in most government agencies. However, the place to change that is by sending letters to Congress, not to drug manufacturers, as was proposed in the original post of this thread. I'd appreciate it, therefore, if you would refrain from putting words in my mouth to use as strawmen for your argument.

Next, what the original poster was asking us to do was to attempt to pressure the manufacturer of the drug in question to apply for compassionate use exemptions for this drug, as the company has apparently chosen not to do so at present. There may be very good reasons that the company has chosen not to go this, not the least of which may be adding additional FDA red tape to an already cumbersome process. If that's the case, we should be cheering the company, not barking at it.

Finally, if you are going to quote material from a blog, it is simply good netiquette to provide a link to the material that you are quoting and to make clear what is quoted material and what is your writing. I'd appreciate it if you would provide those links for the material you've quoted in your two prior posts.
 
I've seen it there. I give it no more credibility than I do any other gossip I read on the ’Net.



Given the number of drugs that make it through the current process only to be recalled later because they cause harm over the long term, I surely don't want to make it easier for those kinds of time bombs to make it through the system.

The solution for those small and startup companies is to partner with larger companies who have the resources to make it through the lengthy trial period. If you want to blame the FDA process, then you should consider blaming the greed of the folks running those companies who want the big personal payday at the end.

OK Trfogey when I read your above quote it certainly seems to me that you are defending the current process. How am I putting words in your mouth when you come out and say "Given the number of drugs that make it through the current process only to be recalled later because they cause harm over the long term, I surely don't want to make it easier for those kinds of time bombs to make it through the system. " I think it was reasonable for me to infer from your quote that you are not interested in making the process easier.

Here are the links you requested. You will see they were quoted in their entirety and not taken out of context.

https://www.ipscell.com/2011/09/big-tent-team-science-new-ideas-about-clinical-trials/

https://www.ipscell.com/2011/10/is-...o-be-patient-can-develop-cures-at-warp-speed/
 
Biogen is providing the drug to those who completed phase 2, and intends to provide the drug to those of us in phase 3. They obviously went to the FDA and recieved permission for this compassionate use.
 
Every company I have done clinical trials for, has offered the drug to the trial participants after the trial completion, on compassionate grounds as long as the clinical trials have proved the drug to be safe and efficacious. Biogen is no exception.
Its different to offering everybody with the disorder, the drug on compassionate grounds. That would be the same as giving it all away. How can any company be expected to spend billions on drug development then give it away at the end?
 
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