What Are the Regulations for Research on Diseases like ALS?

[nautica]

Does anybody know the regulations concerning research.

For example, If I were to research on mice, what are the laws concerning controlled and non-controlled substances. Mostly concerned with schedule 2 and 3.

I am a student but I would have the funds to start a company if needed and could, also, have an M.D. with me if needed.

Thanks
Nautica

 

[iansmith]

bio lab research there something call the Good Laboratory Practice (GLP) Regulation. I think the FDA is responsible for these regulation.

Amazon.com sell book about it.
https://www.amazon.com/exec/obidos/...tory Practices&mode=blended&tag=pfamazon01-20

 

[Mike H]

I was going to say that chemical supply companies do sell DEA scheduled drugs and that if you happen to have a copy of the Sigma catalog they probably have the rules (or at least some references).

I imagine it is an enormous administrative hurdle, although I suspect it would scale with how many drugs you use and in what quantities.

 

[Monique]

If you are going to deal with research subjects or animals or radioisotopes there will be a lot of regulations. I wish I had something helpfull to say, but I really wouldn't know how to start up such a thing :(

Ian referenced some good books though :)
How about talking to some profs and see which guidelines they give.. and then get back to us to report how it works :)

 

[nautica]

The professors at my university are not involved in research. I have thought about applying for a position at a univeristy that is about an hour from my home. But, I would prefer to stay here and I would rather not work on someone elses project or on a project which I am not concerned with.

Also, the professors at my university have told me that I could use the facilities and that they would help if needed.

I will probably look into those books, mentioned above. I will let you know how things go.

Nautica

 

[Monique]

So what kind of research are thinking of and what kind of services are planning to supply? Just being curious :)

 

[nautica]

I have been doing research ALS for a couple of years. At least, reading others research. (I posted a couple of days ago, mainly to see if there was anything I missed)

But there are a couple of drugs that have been tested for different diseases and some for ALS, which I have interest in. I believe that until we find a cure that a "cocktail" of drugs need to be prescribed in order to effectively treat the disease.

Another drug which I am extremely intested in the the viral vector IGF-1. There is some research being done on ALS at the present with IGF-1, but it is not viral delivery, so I am not very confident that the results will be positive.

IMO, a drug must penetrate the blood/brain barrier in order to have any impact on a disease which targets the CNS. At this time, a viral delivery is the only method of achieving this.

Nautica

 

[Mike H]

Just checked my copy of the Sigma catalog since I am in lab.

If I'm understanding things correctly, both you and your institution need to be registered with the DEA. Or maybe it's just your institution but with a specific reference to you, it's somewhat unclear to me.

Apparently all orders for controlled substances must be filled out on an official DEA document.

The DEA's contact info from the catalog on this matter is:

Drug Enforcement Administration
400 Sixth Street, SW
Washington, D.C. 20023
(202) 307-7255.

From what little I know (my group may yet end up exploring the aspects of DEA licensure for our own research), the license/registration is not easy or quick. It's not unusual to get denied.

 

[nautica]

Great info. Does it help, if an M.D. or D.O. is involved??

thanks
nautica

Originally posted by Mike H
Just checked my copy of the Sigma catalog since I am in lab.

If I'm understanding things correctly, both you and your institution need to be registered with the DEA. Or maybe it's just your institution but with a specific reference to you, it's somewhat unclear to me.

Apparently all orders for controlled substances must be filled out on an official DEA document.

The DEA's contact info from the catalog on this matter is:

Drug Enforcement Administration
400 Sixth Street, SW
Washington, D.C. 20023
(202) 307-7255.

From what little I know (my group may yet end up exploring the aspects of DEA licensure for our own research), the license/registration is not easy or quick. It's not unusual to get denied.

 

[Moonbear]

To obtain controlled substances, you'll need DEA licensing. This is no small feat, because you will need to justify why you need access to those substances, that you are doing legitimate research, that you can restrict access to those substances, and will need to comply with a whole heap of paperwork and regulations.

Animal research requires following another whole set of rules. You either need an in-house Institutional Review Board or need to have oversight through an outside IRB (such as through a local university)...there are specific government regulations on the composition of this board, which includes at a minimum 5 members, 1 must be a scientist capable of evaluating the scientific merit of the proposed research, 1 must be a veterinarian, and 1 must be an unaffiliated member (someone who doesn't work for the institution or company).

Any federally funded research must follow the guidelines set out by the NIH Guide for the Care and Use of Animals, which covers all vertebrates. Even if the work is not federally funded, FDA policy also requires compliance with the NIH Guide as well as use of Good Laboratory Practices.

Any commercial use of animals is also regulated under the Animal Welfare Act, which is enforced by the USDA, and requires licensing and inspection of facilities twice per year. Mice and rodents aren't currently covered under the Animal Welfare Act, but Animal Rights activists are constantly lobbying to have them covered.

Even if your work is not federally funded, if you want to publish it, you'll need to follow all of the above guidelines anyway...scientific journals all require that any reports of research using animals meets those guidelines for appropriate animal welfare.

If you're thinking about using viral vectors in your research, you'll also need to address biosafety issues, which would also be covered by OSHA regulations in any workplace. Even the most innocuous viral vectors need to be handled under biosafety level 2 containment, and this will also tie into the animal welfare regulations and requirements. It's also not as straightforward as it sounds to just stick a gene into a virus and get it to the right cells.

If all you're planning on doing is testing a cocktail of already existing drugs, you're not going to have a profitable company because you can't make any money off of someone else's product...you'll run into heaps of trouble with patent infringement.

That you're not even aware of the regulations indicates to me you're very naive about how to conduct research. Everyone thinks they have great ideas while still an undergrad...write them down, hang onto that enthusiasm, and apply to a PhD program to learn how to do that work. Then, if those ideas still make sense, do the research with the experience under your belt that ensures you'll do it right.

 

[nautica]

Originally posted by Moonbear


If all you're planning on doing is testing a cocktail of already existing drugs, you're not going to have a profitable company because you can't make any money off of someone else's product...you'll run into heaps of trouble with patent infringement.

.

I am one of the few that is not interested in money. I was extremely successful in the Real Estate Business and have no need nor desire to make money in the medical industry.

I do however have a friend who is 3 years into ALS and with a 2 to 5 year life expactancy there is not much hope.

Thank you for the information, It is much appreciated.

One question, a drug like IGF-1 is not a controlled substance, in fact I am not even sure if it is recogized as a substance. What is the legality concerning substances like this?

Thanks
Nautica

 

[Moonbear]

Originally posted by nautica
I am one of the few that is not interested in money. I was extremely successful in the Real Estate Business and have no need nor desire to make money in the medical industry.

That's very admirable. However, I'm not sure you have a realistic idea of how expensive medical research really is. One thing smaller companies do when they need facilities they can't afford to build is to outsource the work to universities, either just by contracting the animal housing and procedure areas (this is usually limited by how much space a university has for their own researchers...if they have empty rooms, they are more than happy to have someone pay to use them, if they are full to the gills, they won't be very accomodating)...this also comes with regulatory oversight by the university, so will help you through all of that.

I do however have a friend who is 3 years into ALS and with a 2 to 5 year life expactancy there is not much hope.

I can see why this would be a strong motivating factor for you. You'll need to be realistic that with that short of a life expectancy, even if you're exactly right and all the experiments work perfectly the first time, which usually is not the case, testing in mice is a long way from testing in humans, and not likely to result in a cure or treatment in time to help your friend. I still would urge you to put the time into learning to do research properly so that you have the best chance of success...afterall, if the experiments aren't properly controlled or have a lot of technical problems, even if you are onto a cure, nobody will believe it.

Thank you for the information, It is much appreciated.

You're welcome. Something else you should consider is that if this is truly a viable approach, someone may already be working on it. One organization that will have members doing that sort of work is the Society for Neuroscience. I don't have their site bookmarked on the computer I'm using right now, but I think it's something simple like www.sfn.org.[/URL] They have abstracts of work presented at their annual meetings for the past several years available online. You can search that database with keywords...try things like ALS (or spell it out)or IGF-1. If what's important to you is just seeing the work done, and you have the means to financially support it, you may want to contact some of the people currently doing work in that area and see whether your ideas are viable and if they'd be willing to take on that work if you funded it...and perhaps they'd even agree to having you come into their labs to learn to do some of the work. Sometimes we don't see things published because they just don't work, so talking to people who know that specific field would help determine that. For example, maybe they know from their colleagues that someone tried it and it killed all the mice. Something like that would never get published.

[QUOTE][B]One question, a drug like IGF-1 is not a controlled substance, in fact I am not even sure if it is recogized as a substance. What is the legality concerning substances like this?

Thanks
Nautica [/B][/QUOTE]

It is of course a "substance", but I'm not sure if IGF-1 is under any regulatory control. I work at a university, so they may already have agreements with the vendors regarding purchasing of these sorts of substances. I have had to fill out some simple forms for some things that aren't really controlled, but are things companies don't want to just sell to the average person on the street, so they make you sign forms agreeing you're using it for legitimate research purposes, will keep it secured, etc. That's just covering their own liabilities.

However, you were talking about using viral vectors for IGF-1 delivery...in that case, you aren't looking to buy IGF-1, you're looking to find someone who has a cDNA clone for it. Those you don't just buy from a catalog, you either need to make it yourself or convince someone else they should provide it to you (in which case, you're still going to need to grow up more of it yourself). These things are not mass produced.

You mentioned trials are already underway using IGF-1. What is the effectiveness of delivering IGF-1 directly into the brain? That must be known or they wouldn't be taking it to clinical trials yet. Actually, that's usually the first step in testing something on a neurological disorder, to inject it directly into the brain and see if it does anything (in the appropriate animal model of course). I'm not sure if it is an issue for IGF-1 to cross the blood-brain barrier. I think it does, but I'd have to double check on that.

 

[Moonbear]

Okay, so I just did a quick search on PubMed just to see what's current. Your idea apparently is a good one, except it's already been done...and published in Science!

Kaspar BK, Llado J, Sherkat N, Rothstein JD, Gage FH. Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model.
Science. 2003 Aug 8;301(5634):839-42.

If this is something you're really interested in pursuing, I suggest you locate this article, look up the contact information for the corresponding author and talk to them about getting involved in the research in their lab in this area. If they've already published this, they are likely at least 3 steps ahead in their thinking already. Of course, keep in mind that when someone publishes in a high profile journal like Science something about a disease like that, they will have people crawling out of the woodworks asking them about cures for them, so you may not get a very quick response.

 

[nautica]

Thanks, I really appreaciate you taking the time to respond.

What really bothers me is that researh has become so "scientific" that it actually hurts the people with diseases like ALS, which, due to several factors, will be almost impossible to meet any of the guidelines. At least not in time to help anybody who has already contracted the disease.

thanks
nautica