collagen disease drug

as only you probably have seen me here introducing speakers in the past. today we are a ttle delayed and we will be back in about 10-15 minutes. so we're going to start with our first speaker dr. richard siegal who is the director of the
clinical program at the nins. and in between the two speakers -- he's here. you can do your introduction right now. >> we're just a little bit delayed. at any rate, so i want to begin this and read something to you.

so those of you will remember we talked with this with paul -- at the time when we discussed sexually transmitted diseases. great discovery of 606, magic drug to treat syphilis for many years until penicillin came1 around. but urlich was one of the great
founders of immunology and it's interesting that in the 18th century i wrote the following. we pointed out if the organism possesses certain contrivances where immunity reaction produced by all kinds of cells is prevented from acting against the organism's own elements so
as to give rise to auto toxins. if that happened we might be justified in speaking what he called the horror auto toxicas. of course it turns out he was right and the body can we made to self destruct under certain circumstances which are the subject of today's demystifying
medicine. so now we jump to the 21st century and 22 nobel prize later all for immunology and essential questions kind of remain although much has been learned along the way. that's also part of today's demystifying session.


really so much has been learned about autoimmunity that the basic question is raised what is atone immunity. it turns out as you'll hear to be more complex than just that the name implies. so how does autoimmunity occur? if you have some marker, can you
literally predict a disease many years before it's manifested. and are these markers cause or are they effect. are they prognostic, do they offer some approach to therapy. and even most recently like yesterday, what to me was an all new element, there's an article
in science that we'll put on the web side. so this is from march 1st science, a very interesting study in which the intestinal micro biome has been manipulated and in these studies the authors claim that it plays a role in driving the hormone dependence

immunity. this is on mice. based in general the autoimmunity diseases are frequently more common in women than in men. and the last one is not important. there's another recent
incriminatary -- that's reminiscent of the days when many of these autoimmune diseases what we now call them things like rheumatoid arthritis, all sorts of diseases that you'll hear about because nobody really knew what they were called.

it was a fashion when they were called psychosomatic diseases. there was another fashion related to focus of infection so people had their tonsils taken out and so forth to control their joint pains. there's this whole business of a search to understand the
mechanism and right in with it are therapeutic approaches much these diseases are extreme and serious. so we are very e to have two colleagues at nih to be with us today and discuss this topic. and i'll briefly introduce them both to you.
so the first speaker's going to be richard siegal who took his mdph degree at the university of pense and trained in medicine, rheumatology, came here and spent some time at michael lenardo who you may remember spoke earlier in this course, is an outstanding immunologist.
he's the director of nih ph.d. program and his official -- no, i don't have it. he's the clinical drucker of niams. there are certain populations of immune sites in the process and so or. our second speaker will be abner
notkins who graduate from yale, got his md at new york university, did clinical training at hopkins, came to nih and he's fundamentally a renaissance man. most specifically in virus infections and diabetes and the relationship between them.
in recent year has become really one of the leaders in thinking and doing research in this field of autoimmunity and he coined the concept of developing a battery of auto antigens that could be widely used in the auto antigen oem. oemics are both at six.
we're happy both these distinguished colleagues are here and will present this topic of autoimmunity. richard, i understand that ... good. >> thanks very much. thanks for inviting me. i'm happy to talk to you today

we have. have a patient in our lupus research program and has undergone some treatments here and i'll introduce her in a second. we'll talk for 15 minutes and i'll give a general talk about autoimmunity and dr. notkins

will hone in on auto antibodies if i understand your talk right. since we're going to put our guest on the spot a little bit telling us about lupus, maybe i can just put you on the spot for a second so i understand who i'm talking to. so i guess it's a pretty diverse
audience but just sort of show of hands who sees patients here medically trained? okay, some but not the majority. i'm going to talk in more generalities. when we have our patient talk we'll try to stay away from jargon.

our patient is more versed at medicine because she's been through a lot. this is our guest,ths aviles. anyway wire going to just have a talk about your disease. tell us how old you are now and when you first -- how old when you first were diagnosed with
lupus. >> 37 years old right now and i was 20 when i was diagnosed in 1996. >> so maybe just tell us a little bit about what was happening that you, what was happening to you that you thought you went to the doctor

and what you were diagnosed with. >> i had a rash on my legs. they were red and they used to get hot. and they hurt a lot. >> how long did that go on before you saw a physician. >> like two months.
>> were you feeling well or not well with that? was it just a rash and otherwise you were feeling well. >> i was very tired. >> a lot of fatigue. before 20, before that age, you had you think normal energy, normal childhood.

>> a lot of energy. >> right, okay. you brought me some pictures actually really looked like your whole body changed a lot with that. >> yes. >> and then so the rash was going on for about two months.
any other things happening along with the rash that you noticed? >> that was the only thing. >> and then at the time when you went to the doctor for that, did they diagnose you with lupus right away or did it take a long time. >> they diagnosed me right away

because my mom had lupus and she was diagnosed in 1978 and she passed in 1991. she was 38 years old. >> right away the doctors knew -- >> i had it in the background so they test me right away. >> because your mom had lupus
were you tested for that test before you had any symptoms. >> no, i don't think so. >> the reason i ask that is we know from studies of people in the military, very interesting, i'm not going to talk specifically about lupus today bepeople have these ant borders

in their blood 10 or 20 years before the onset of disease. there was a nice study of military recruits because everybody that goes to the military they get a blood test and they went back and found people who developed lupus maybe 20 years later and those same

auto antibodies were actually there before. we kind of know in people, you probably had that dna in your blood even before you got sick. so you have the rash been, you got the blood fess, it was positive at that time. you already, when they did more

tests on you were you already having other problems from the lupus at that time. >> no. i took ten years to get nephritis. >> so right at the beginning to treat the fatigue and the rash you took some medicine for that.

>> i took -- and prednisone. >> how did that do? did that seem to help. >> that helped with the rash and oh, i also had joint pain. my joints used to get swelled up and very painful. >> that's an important thing. lupus is our prototypical

disease that affects almost every organ in the body but it does affect the joints very often. that's why we as rheumatologists affects different organs. for ten years you had that and tell us what happened when you developed the nephritis.

>> in 2003, my blood pressure went really high and my ankles were swelled up. and i couldn't wear shoes or anything. i had a -- doctor and he gave me -- for six months. once a month. >> by vein.
it was once a month. they gave me -- and that didn't work either. they referred me here. >> so the kidney function we talked a little bit about this. her kidney junction which was normal before that started getting worse and despite these

two treatments have been kind of the standard treatments for lupus nephritis kidney inflammation things kept getting worse. you came here and you can tell us what kind of therapy. >> when i came here in 2006, i was in need of kidney transplant
on both kidneys. >> the kidney function was at a point. that is one of the most serious consequences of lupus and what we try to prevent with the sigh toxin. in your case it didn't work tell us what happened.

>> i got a stem cell transplant in may 16, 2006. >> that's the procedure. we're going to talk about it again where we did more intensive chemotherapy and gave back your own stem cells. it's an auto transplant not somebody else's and that's the

attempt to reset the immune system. we can separate things out. there are a lot of complications after that but then in terms of the lupus, tell us what happened with your kidneys and those kind of things that were failing before the transplant.

i don't need the kidney transplant. they're working fine right now. >> in terms of the lan es we really did reset and that went into remission. let's talk in terms of that. you have only one manifestation that we think was a new thing

that happened to you you were telling me when we talked before about your lupus. >> last year i had -- rash which i never had before even when i was diagnosed the first time. and it was after the stem cell. >> so that was very interesting to us.

this rash which we'll show a picture of, not yours but somebody else's, is a very characteristic and often the first manifestation of lupus, and that's actually what caused it to be named lupus. lupus is the latin word for wolf.

somebody thought this very red rash looked like wolf patches under their eyes. you got that in the wrong order you got it backwards but it did tell us that some element of your disease was still there even though very suppressed. and right now for lupus, do you

take any medicines. i just take ten milligrams of prednisone every day. >> so that's something that we sometimes use for that but pack nil you're not taking anymore. >> you can tell us a little bit, this was a success in terms of lupus but we won't go through

everything but you were in the hospital for quite a while. >> almost two years inpatient. >> there were a number of complications and i guess i'll sort of go through it briefly. there were some, you had a number of infections which needed some gastrointestinal

No comments:

Post a Comment