How to ask for help

When Gess is sick, several people ask "Is there anything I can do?" and my standard response is "Thanks, but we are fine." Mostly because I do feel "fine" and don't know what to ask for. But as things have changed, I know that I probably need more help, but I am not sure what to ask for. I am so used to doing this all by myself, it is just hard to envision having other people involved. We have started to have one couple more involved. They will take Gess to the hospital and they watch Beauty. But, there are other people that want to help and I honestly just don't know what I can even ask for. It is hard for me to ask for help, period. Even more so when I don't know what to say. I have the best friends and they really do want to help, and I know that I need to let them help. I just have to figure out how.

A better day

Today has been a better day. No more seizures thankfully. Last night we "slept" at our friends' house. Gess seemed to get a fair amount of sleep, but I had a hard time sleeping. I kept listening to him breathe. Every little twitch or movement jolted me awake. For a couple of hours I just sat there not even closing my eyes because all I could "see" was the seizure. I am not sure why it affected me so much, but it was one of the more disturbing things that I have seen. I think that part of it is the unknown and then also the non-responsive state that he was in. Physical things are one thing, but more "mental" things are another.

We came home around 10:00 ish this morning and I did a bit at home. I was exhausted so we took a nap. The weather has changed so we curled up and cuddled with Beauty. We were both able to get some sleep and stayed in bed for most of the day. When I woke up I felt a bit better, I think that the exhaustion has just been wearing on me.

This evening has been equally uneventful. Chinese delivery. DVR-ed TV. The three of us scrunched together on the couch. I did a little homework, a little shopping, facebook.

Gess's dad decided to come out next week. He is having some issues with the condo he is closing on soon.

So, here's hoping for a low key few days and then a good visit with Gess's dad.

Seizure

Gessner had a seizure tonight. Luckily he was in bed and I was right
there, but it was probably the scariest thing that I have ever
experienced. I was getting ready to leave for a CFF event and
wouldn't have been there if it had happened 10 minutes later. It
probably lasted about a minute, though it is hard to tell. The worst
part was when it was over his lips were blue and he was
nonresponsive. He just laid there with his eyes open but he would
respond to anything. I called 911 and then my friend Tasha. The fire
truck got there rather quickly and Gess finally started to be somewhat
responsive. He was really confused and kept asking me what happened.

They made sure he was stable, gave him o2 and called for an
ambulance. It seemed to take forever for them to get there and get us
to the ER.

The ER doctors think that it was caused by demoral. They also said
that meropenem (one of the abx he is currently on) can cause
seizures. They took blood and urine to measure the demoral
metabolites in his system, but we won't have those results for awhile.
The ER sent him home, though I would have prefered for them to admit
him. I did have the ER doctor consult with the pulmonary doctor on
call (who happens to be the director of the CF clinic) and she agreed
that it was fine for him to come home. We are staying at our friends'
house tonight so that I have back up in case something else happens.

I'm freaked out and scared that it is going to happen again, and worse
that I won't be there when it does. I called Gess's dad who
immediately booked a plane ticket and will be here at 3:00 pm
tomorrow. There really isn't anything that he can do, but I will be
glad to have him. Gess was mad that I told him to come, but I didn't
know what else to do.

I keep playing the last few days in my mind to see if there was
something that I missed or something that I should have done that
might have prevented this. I did not want him to get discharged from
the hospital yesterday. We actually got in an argument about it. I
just felt like he still needed to be there, but he wanted to come home.

I feel so much responsibility for watching over him and feel like
lately I have been failing.

Feeling Broken

Things are rough right now. Gess is in the hospital and I just feel like I am at the end of my rope. There isn't anything specific that is going on; in the grand scheme of "CF stuff" this seems rather minor, but it has just hit me. I think that it is just the combo of everything that is going on. I hate when I feel like this because I feel so utterly inadequate. The words "I hate myself" pop into my head too many times a day and they are getting harder to push away. Today I have done basically nothing. I just feel like I can't possibly move to do much. I have SO much to do and no excuse for not doing it. Yet, I just can't seem to make myself act. I am tired, no, I am exhausted. I am broken. Of course, Gess needs me and so I feel guilty for being such a wreck. I guess I will put on my big girl pants tomorrow, suck it up, and power through. Or at least I hope I will be able to.

In the hospital

Gess is in the hospital again. He hasn't been feeling well...sort of
fluish. It seemed to be getting worse instead of better so he decided
to go in. I was still on narcotics from my surgery, so a friend took
him in. They admitted him and put him on tobra and meripereom. They
also gave him fluids and pain meds.

They ran a bunch of tests, including for the swine flu. In fact I am
struggling to type this because my iPhone does not like the gloves I
am required to wear as contact precaution.

Gess is looking a lot better and feeling better too. He has only
vomited once today and that was coughing induced.

I believe that if the flu test comes back negative he will be able to
come home tomorrow.

Taking Care

My post about Compassion Fatigue has me thinking about things that I can do to take care of myself.  So, I am going to have a series of posts about this.  

One good way for me to relax is to read a good book.  I love books...get me in a bookstore (or on amazon.com) and you should hide my wallet!  When I was a kid, my siblings and I would go to the library every week during the summer to participate in their summer reading programs.  My sister and I would get huge stacks of books each week and then go home and devour them.  Some of them were silly books, but they were books nonetheless.  My love of reading continued through high school and college.  When I got to law school, however, I found that I stopped reading for pleasure. I think that it was because I was reading so much for class.  Occasionally I would get a "beach read" type book and more than once I found myself reading through the night instead of studying.

After law school I started to read again and it has been a good source of relaxation for me.  I generally have several books going at once and read a wide variety of book.  On top of this, I formed a book club.  That has been great.  We meet once a month (roughly) and rotate who hosts.  We have dinner, talk, laugh, and generally have a great time.

I also carry a book with me most places and that certainly comes in handy when we unexpectedly end up in the hospital!  I read all of Water for Elephants by Sara Gruen during one ER visit.

Compassion Fatigue

I was reading an article in Oprah magazine (there are good articles in it, I swear!) about Compassion Fatigue that I found very interesting.  Basically, it is the term that doctors have given to what happens to some people who are chronic caregivers.  It is different from burn out and has similar symptoms to PTSD.  In fact, another name for it is Secondary Traumatic Stress Disorder.  My reaction when I read this was "of course this happens."  I have experienced it myself (and think that I am finally coming out of a bout right now) and have seen other people experience it.  That being said, there was something that struck me about having a label to give to it and some sort of "official" recognition that this exists.

One thing that I have found many caregivers, including myself, experience is guilt.  I sometimes feel guilty because I am worn out, stressed, exhausted, etc.  As I type this, I realize how silly that sounds, but it is something that I do experience.  Much more frequently than I would like to admit.  

So, what can I do with this new "awareness"?  I guess it is just a reminder of what I already know--I need to make it a point to take care of myself too.  CF (and other stressors) are difficult.  There is no way of getting around that.  And in order to do my best at helping Gess through these things, I need to be healthy and sane.

The question is, how to do this?  As a perpetual "doer" I do find it difficult at times to identify my needs and know of things that I can do to "take care" of myself.  I used to be so bad that Gess would draw me a bath, light candles, and then literally lock me in the bathroom with instructions to relax until he let me out! (I had this habit of taking baths to relax, only to be ready to get out by the time the tub filled all the way!).

The Oprah article has the following recommendations:

• Mindfulness meditation: It's been shown to decrease depression and anxiety while boosting empathy.Oprah.com: Try these meditation exercises

• Keeping a journal: Research suggests that reflective writing helps prevent compassion fatigue.

• A daily act of self-centering: Set an alarm for noon and take four deep breaths; or when you wash your hands, sink into the experience, feeling the sensation of the water on your skin while noting, "I am worthy of my own time."

• Staying connected to the outside world with at least a phone call every day. Better yet, get outside, even just to take a walk.

• And don't be afraid to ask for help.

http://www.cnn.com/2009/LIVING/personal/08/28/o.have.compassion.fatigue/

I am going to make it a point to try to find new ways to take care of myself and to actually practice  the ones that I know work.  I also encourage all of you out there in blogger-land to do the same and let me know what you find that works!!

Oh, I also found a Compassion Fatigue Awareness Project--looks like a good resource, so I will check it out!

 

Gene Linked to Liver Disease in Cystic Fibrosis

Interesting article about liver disease and CF. Also, I think that Gess was one of the patients in the study (though I could be wrong, he did several at UNC)

http://www.drugs.com/news/gene-linked-liver-cystic-fibrosis-19746.html

Gene Linked to Liver Disease in Cystic Fibrosis

TUESDAY, Sept. 8 -- A variant of a particular gene in people with cystic fibrosis greatly increases their chances of developing severe liver disease, new research shows.

Cystic fibrosis is an inherited disorder that can lead to deadly lung infections and digestive problems.

About 3 percent to 5 percent of the 30,000 people in the United States with the condition will also develop a serious form of liver disease, including cirrhosis and portal hypertension, or high blood pressure caused by obstruction in the liver, according to the Cystic Fibrosis Foundation.

Researchers from University of North Carolina at Chapel Hill analyzed nine variants in five genes previously implicated in cystic fibrosis liver disease. The study included 124 patients with cystic fibrosis liver disease and 843 patients without liver disease. A second study looked at a different group of 136 patients with cystic fibrosis liver disease and 1,088 without liver disease.

The researchers found that people who had the "SERPINA1 Z allele," or gene variation, had a five times greater chance of developing liver disease. The other variants did not increase the risk of liver disease.

About 2.2 percent of people with cystic fibrosis carry the SERPINA1 Z allele, according to the study published in the Sept. 9 issue of the Journal of the American Medical Association..

Screening for the gene variation could help identify those at risk of developing the liver disease, the researchers wrote.

"The identification of the SERPINA1 Z allele as the first marker for the development of severe liver disease in CF [cystic fibrosis] illustrates the possibility of identifying CF risk factors early in life, conceptually as a secondary component of neonatal screening after the diagnosis of CF is confirmed," researchers wrote.

Updates

I went to Yakima to be with my dad on Monday evening. We got into town late, so didn't go to the hospital immediately. Instead, we went to the hotel and thanks to Ambien I slept pretty well. We went to the hospital early on Tuesday morning. Surprisingly, it went pretty well with seeing my dad. I guess I just left the "other" stuff at the door and dealt with the matter at hand. He actually looked pretty good and was in good spirits. His left arm is extremely swollen. He has a large blood clot in that arm. The doctors think that is was caused by the mass in his chest/lungs pushing against the vessel. They started him on a heprin drip to prevent more clots from forming and then started him on coumadin.

They did a bronchoscopy on Monday and took some samples from his enlarged lympnodes. And then we waited. And waited and waited. The pulmonologist told us that he is sure that the mass is cancer, but that he doesn't know what type. Based on the CT Scan it is unclear whether the mass is inside the lungs and jutting out or if it is on the outside near the lungs. His lung functions are pretty good, considering he has smoked for 60 years. His FEV1 was at 59%. His O2 saturation levels were low, so they kept him on 2 liters of 02 (on which he satted at 93-96%). I am not sure if this means that he will go home on supplemental 02.

I spend most of Tuesday and Wednesday at the hospital with him. We talked a lot. My aunt that I rarely see was also there, so we talked too. He told a lot of stories about when he was younger and when I was little. Many of those stories were hard to hear. They were stories about him being drunk and me being a little girl. Stories that are painful and that I would rather forget. So. I am carrying those with me right now and not quite sure what to do with them.

When we left the hospital on Wednesday evening the doctors thought that they would discharge him on Thursday, but subsequently they decided that they would keep him until they got the biopsy results back. Today (Friday) the doctor said that the sample taken during the bronch was not large enough, so they had to take another sample. I think that sample was taken today, but the results won't be back until Tuesday or Thursday, which means that he will be spending the long weekend in the hospital.

So far I am okay with everything...but am not sure what will come. I had pretty successfully shut out some of the issues with him from the past, but this is forcing them back into view again. At this point, I really do feel like I am "done" with those issues, as "done" as you can be. They happened, and there is nothing that can be done to change them. He is at least trying to change and be a different person, which I do respect. So, maybe that will be it and I will just deal with the medical issues at hand. I don't know for sure.

When we came home we brought my 18 year old brother home with us for a visit. There is a lot going on with him right now, and I don't feel like getting into the details. But suffice it to say that the last couple of days have been trying emotionally for me.

I am exhausted and am ready for a break and am going to try to get one this weekend even if it means kicking the boys out of the house!

Worried about seeing my father tomorrow

My father is in the hospital and is not doing well.  My sister sent me a text this evening and given the description, I knew that I needed to come.  Gess also decided to call and the first thing that he said after hanging up is "he's dying."  Gess was crying (I am still not sure why--he has probably seen my father 3 times ever).  Apparently my dad did ask for me to come, so I dropped what I was doing (which was making peach jam), and made the 3 hour trip.  Gess came with me (against my protests).

You'd think that going to the hospital would be nothing big for me now, specifically since we have practically lived at one this year, but I am worried about this.  For one, I only have bad memories associated with this hospital.  Basically, something "bad" happened with my mother here (and she doesn't know that I know about it) and so just being in the building triggers something with me.  Second, I haven't seen my dad in probably 7 years.  I have exchanged letters with him a bit, but haven't seen him face-t0-face since my last year of law school.  The reasoning behind this is pretty complex...or maybe not complex but difficult for me.  Long story short, my dad was not a great guy (abusive alcoholic) and left my mother (pregnant with three other children all under the age of 7) when I was 7.  I hardly ever saw him in the next 10 years.  I had assumed that he moved away, but found out that he in fact lived in the same little town that I lived in.  He just never cared to see us.  So, that hasn't sat well with me.  

He came back around just as I was graduating from high school and I had a semi-relationship with him during college (mostly at the urging of Gessner).  But he really just made excuses about what he had done, etc.).  So, finally I had enough and just stopped responding to his letters (letters have been our main form of communication).

About a year and a half ago, I wrote him again, worried that he would get sick and then I would regret not trying harder.  So, we again exchanged letters.  He seemed better this time.  I also found out that he was sick...diagnosed with COPD (which is no surprise since he has smoked since he was like 10).  There was an issue that came up because I didn't feel comfortable going to a family reunion with him, so I pretty much stopped communicating with him again.  I was too overwhelmed with what was going on with Gess and some other stuff and at the advice of my doctor and therapist, I really needed to cut out people that were "energy drainers" etc.  He was definitely on that list, so I really haven't had much contact with his for the past year of so.  

Today there was no question that I needed to come to see him even with all of this.  I am not sure why, but my gut told me  I needed to, so I followed.  I am a bit worried about the toll this is going to take on me.  My dad is old.  He will be 69 in just a few days.  He smokes and also drank for many years.  I am sure that his liver is bad, as well as his lungs (which rubs me the wrong way considering Gess's health at the age of 32 not because of stupid lifestyle choices, but because of CF...but that's for another day).  

He has a wife that I think I met once.  And a daughter that he adopted (which is a very touchy spot with me since he abandoned us and yet voluntarily took on someone else) and I imagine she might be there.  I haven't been around and I don't know if that was the right choice...it certain seemed like the right choice and I don't know where I will fit in tomorrow.  

I don't know exactly what is going on, though the explanation given by both my sister (to me) and my dad (to Gess) sounds like it could be a terminal situation.  

Not sure what tomorrow will bring, but I am going to try to keep my wits about me and just be there if he needs me (regardless of the past and all of those issues that cannot be changed).  At the same time, I will not accept anyone attempting to place blame or guilt on me for the past (not that I expect that to happen, but if it does, I will stand up for myself).  And above all, I have to try to keep myself from getting "sucked" into any drama that is just going to be stress causing.

For now, Ambien take me away....

Babies

Man, I feel like I am beating the proverbial dead horse, but I want to have kids. I am not sure why this is hitting me so much right now. It might be because I have been around kids a bit lately (I even had a 6 year old explain to me how to have kids!), but it also might be my birthday coming up (I will be 32 *gasp* in a couple of weeks).

I am planning to talk to Gess again about the issue, but those talks are always so emotionally draining (for both of us). He says that he wants kids, but feels like it would be irresponsible because of his CF. I do understand the argument and can agree with it on an intellectual level sometimes. But there is a part of me--a huge part of me--that just cannot accept that I "can't" have kids. I might feel differently if I were the one with CF. But I'm not, so here I am, wondering what to do. It seems so ironic that I have done everything "right" in terms of what society says you are "supposed" to do for starting a family: I went to college and graduated, and then married a great man that I love, and then went to law school and worked on my career so that I can have a good job and financially care for a family, waited until I was older and more mature, etc., etc., But here I am, only 32 and the possibility isn't looking good. I feel like an adolescent that just wants to stomp her feet and scream "It's not fair!" at the top of my lungs.

I wish there was a way that Gess and I could be on the same page about this, but really I don't think that it will happen. And that really hurts. I sometimes envision myself as a young-ish widow, all alone with no kids and no family (since my family is not supportive). That seems like such a bleak and lonely future.

I know that there isn't an answer. I also know that a lot of people have strong feelings about the whole CF-kids issue. So, I am not wanting to start any sort of debate. Just trying to get out my feelings, so hopefully I can move on to an "I'm okay" phase.

Physical Limitations

When I met Gess he was very active.  Really I think that he was so determined to not let CF define who he is, that he pushed himself a lot to be active.  He did martial arts as a kid and he still does demonstrations (which unfortunately ends up with broken hands sometimes).  We used to hike a lot and do a lot.  And I love to be active.  I have a long list of things that I want to do and a lot of them involve pretty intense physical activity.  

So...here is the dilemma--Gess has not been "up" for doing much physical activity right now.  I totally understand--he has been so sick and the bleeding, etc.  But I don't know how to deal with this on a day-to-day basis.  For now I have just been casually mentioning things and seeing how he responds.  Like a group of friends was going on a pretty easy 4-mile hike today and so I mentioned it and he said that he would like to go.  So last night I asked if he thought that he would still want to go and he said that he had bled that night, so he wasn't sure.  So...we didn't go.  It is fine that we didn't, I'm not complaining about that.  I am just not sure how we should deal with this on an ongoing basis.  We are supposed to go kayaking next weekend.  Gess set it up with a friend of his...but given his health and the recent track record, I don't know if we will be going or not. 

I guess what I really need to do is just have a talk with Gessner about this.  It will not be a fun conversation....but it is something that we need to do.  We just need to have a discussion about where he is and what he feels capable of doing.  Going on as if everything is the same as it used to be is not doing either of any favors.  It probably makes him feel badly when he has to cancel because he isn't up for it and it makes me frustrated that we are canceling plans, etc.  

I also don't know where is leaves me. I am an active person and want to continue to be.  If my shins ever get better I will be training for triathlons again.  But is this going to hurt Gess?  Is he going to feel like I am leaving him behind?  

Been a very bad blogger...

There hasn't been a lot going on lately.  Gess seems to be feeling okay.  He has had some bleeding, but nothing major.  No more sudafed or demoral, so no more loopy days.  I am struggling to write anything else about his health.  I guess it is because I am really tired of CF.  I have to deal with it (obviously), but I don't have to write about it :)  It is hard having a husband with CF and sometimes it is just exhausting. 

Vancouver and Chicago

I posted the details and photos from my trips on my other Blog.

Family Blues

I am so frustrated with family right now. Our society values family and commitment to family so much. So when you feel like breaking from that expectation, it feels like you are committing some cardinal sin. My family has been a continual source of stress and drama for me. I recently told my sister that I had to limit my contact with her and step back. This was really difficult to do, but was on the advice of my doctor and therapist. Basically I need to try to minimize the amount of interaction I have with those people that are "energy suckers." I am trying to recover from all of the stress. My body is telling me that if I don't do it now, something very bad is going to happen. Even knowing this, it is so hard to do. I can't even explain the knot in my stomach when I told my sister that I had to limit my contact. The other thing the really irks me is that my sister told me not to long ago that she knows how "easily" I get stressed out. WTF!! It's not like I am stressing over a a chipped nail! My life is full of big, stressful things. It pisses me off that my family would view me as just "easily stressed," particularly when I feel like I have done pretty well in the face of everything. I mean, maybe I am giving myself too much credit, but I think that I have done a pretty good job of keeping my sanity in the face of a lot of crap.

I have my niece with me this week (my sister's daughter), so the family situation has come up. I feel so badly for her. My sister is a mess (and I am not exaggerating) and I really worry about my niece. My sister's husband is a total loser. She recently left him after he hit her and my niece, but she is now back with him, insisting that he has changed. I seriously doubt it. I just hope that she can keep up in school and get the heck out of there as soon as she turns 18. Ughh...

On top of this, we are having major issues with Gess's family right now--particularly with his mother, which is just making things difficult. Why she needs to make an issue right now when Gess has been so sick, I don't know. Basically, over the years his mother has said many, many inappropriate things and has just not dealt with Gess very well at all. He has been pretty tolerant and forgiving, chalking it up to her just being out of touch or something. I had enough of it after the last comment that she made to me about being glad that we deal with the CF so that she can have her own life. Gess talked to her about it and she got mad at him. Seriously! Then things have just went downhill from there.

Apparently while I was out of town, Gess talked to his mom and these issues came up and he had a long, difficult talk with her. Basically he told her how the things that she did when he was a kid were so hurtful (like when he had to take care of all of his own medical stuff and she appeared like she didn't "care" about him being sick--his words). Gess was hopefully that it would move toward a better relationship, but it seems like it has done the opposite. Apparently he invited her to "talk" to him about the stuff via letters and she has done so; we get something in the mail from her almost every day. He kept them all and didn't open them at first. Instead, he brought them to his therapist's office to read them there, as a "safe" place. He didn't tell me much about what was in them, except that they were what he expected, and not in a good way. Also, in one she made a statement that "Lisa settled for you, even knowing you have CF." I cannot wrap my head around that statement. I can't think of any possible scenario in which a mother would tell her child that someone else "settled" by being with him. And I just can't imagine how that would feel.

As much as I am troubled by my own mother, there is still a part of me that wants acceptance and love from her. So, to have your mother make such a derogatory statement, in such a clear and direct manner must be devastating. It breaks my heart that he has to be dealing with this on top of everything else. I think that it shows amazing strength and character for him to try to communicate with her and I am so saddened that she responds like this. I really don't know what her issue is, but I just want to scream, and tell her to stop being so selfish and be a mother to her son. I know that she has went through a difficult time; I can't imagine what it would be like to have a son diagnosed with a disease such as CF. It would be utterly devastating and incredibly difficult. So a lot of leeway has to be given on how a person deals with that sort of thing; but that can't excuse this type of behavior and mindset. And really, what it is doing is ruining any chance that she has of a relationship with him. I really wish for his sake that this wasn't going on. I see how much it hurts him and how difficult it is. It makes me so angry that his emotional energy is being spent on this, instead of on getting healthy and fighting CF. I wish that she could see how her attitude affects him (and me) and how damaging it is. We deal with the CF alone, not because we choose to, but because that is the only choice. She isn't available or able to help deal with it, and from his perspective, she wasn't able/available long before I came into the picture. I wish that we had more help in handling this. It is exhausting and incredibly difficult to do on our own. And as it is now, involving "family" at these times seems more like an additional stressor than help. (There are some exceptions, this doesn't not apply to all of the family--for example, his Dad has been great in helping. He has been to the hospital with Gess many times and I know that he will always back me up in dealing with the doctors or even in talking with Gess when things are difficult, etc.).

I wish that it were easier to turn away from family. The concept of family is great. I love the idea of having a group of people that are there for you no matter what and who love you unconditionally. People that you call if something happens, and you know will be there. People that support you and carry you if you need it. I would love to have that sort of family. In generally that would be so great, but especially in dealing with a disease like CF. Unfortunately, with a few exceptions, that has not been the case for us and I hate to see the toll that is taking on Gess. Which of course makes me want to stand up and fight for him even more. I want to protect him from this hurt, but I can't. I wish that he could focus on his health and have more support. Also, I wish that I had his family as support. My family is completely worthless when it comes to any of this, so I wish that I had them as backup. I wish that I could talk to them about what is going on and have them a part of our life. I really just wish that I had a "family."

Dawn if you are reading this...

I keep trying to respond to your email but it keeps getting bounced back to me!  Email me :)

Common cold virus efficiently delivers corrected gene to cystic fibrosis cells

CHAPEL HILL -- Scientists have worked for 20 years to perfect gene therapy for the treatment of cystic fibrosis, which causes the body to produce dehydrated, thicker-than-normal mucus that clogs the lungs and leads to life threatening infections.

Now University of North Carolina at Chapel Hill School of Medicine scientists have found what may be the most efficient way to deliver a corrected gene to lung cells collected from cystic fibrosis patients. They also showed that it may take this high level of efficiency for cystic fibrosis (CF) patients to see any benefit from gene therapy.

Using parainfluenza virus, one of the viruses that causes common colds, the UNC scientists found that delivery of a corrected version of the CFTR gene to 25 percent of cells grown in a tissue culture model that resembles the lining of the human airways was sufficient to restore normal function back to the tissue.

"This is the first demonstration in which we've been able to execute delivery in an efficient manner," said Ray Pickles, Ph.D., associate professor of microbiology and immunology at the UNC Cystic Fibrosis Research and Treatment Center. "When you consider that in past gene therapy studies, the targeting efficiency has been somewhere around 0.1 percent of cells, you can see this is a giant leap forward."

"We discovered that if you take a virus that has evolved to infect the human airways, and you engineer a normal CFTR gene into it, you can use this virus to correct all of the hallmark CF features in the model system that we used," Pickles said. For instance, the experiment improved the cells' ability to hydrate and transport mucus secretions.

The resulting paper is published in the July 21 issue of the journal PLoS Biology.

Now the researchers must work to ensure the safety of the delivery system. In a pleasant surprise, simply adding the CFTR gene to the virus significantly attenuated it, potentially reducing its ability to cause inflammation. But the scientists may need to alter the virus further.

"We haven't generated a vector that we can go out and give to patients now," Pickles said, "but these studies continue to convince us that a gene replacement therapy for CF patients will some day be available in the future."

In addition to Pickles, UNC co-authors are Liqun Zhang Ph.D, research associate, CF Center; Brian Button Ph.D., assistant professor, CF Center; Sherif E. Gabriel Ph.D., associate professor, pediatrics); Susan Burkett, research analyst, CF Center; Yu Yan, research specialist, CF Center; Yan Li Dang, research specialist, CF Center; Tristan McKay Ph.D., postdoctoral fellow, CF Center; and Richard C. Boucher M.D., Kenan Professor of Medicine, director, CF Center.

Other co-authors are April Mengos of the Mayo Clinic College of Medicine, as well as Mario H. Skiadopoulos, Ph.D., Leatrice N. Vogel and Peter L. Collins Ph.D., all of the National Institute of Allergy and Infectious Diseases, National Institutes of Health.

The research was funded by the National Institutes of Health and the Cystic Fibrosis Foundation.

http://www.scienceblog.com/cms/common-cold-virus-efficiently-delivers-corrected-gene-cystic-fibrosis-cells-23222.html

New Drug with potential to treat both PA and B. Cepacia

Cystic Fibrosis - Liposomal Tobramycin Receives Second Orphan Drug Designation Within Weeks

Main Category: Cystic Fibrosis
Also Included In: Pharma Industry / Biotech Industry
Article Date: 17 Jul 2009 - 2:00 PDT

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An innovative treatment for infections of the respiratory tract in cystic fibrosis patients has received a second orphan drug designation in the US only weeks after a first designation was granted. The recent designation relates to Burkholderia cepacia pathogens that can cause lethal infections in cystic fibrosis patients. For Axentis Pharma AG of Zurich, Switzerland, both designations affirm the therapeutic potential of its product candidate Fluidosomes(TM)-tobramycin, whose unique microbiological profile sets it apart from other antibiotic formulations (including free tobramycin).

Axentis Pharma (Switzerland) announced that the Office of Orphan Products Development of the US Food and Drug Administration (FDA) has granted a second orphan drug designation to its lead product candidate Fluidosomes(TM)-tobramycin. This drug is a liposomal formulation of tobramycin and an innovative treatment for infections of the respiratory tract in patients with cystic fibrosis. Only three months ago, the FDA granted Fluidosomes(TM)-tobramycin orphan drug designation for the treatment of pulmonary infections caused by Pseudomonas aeruginosa. The newly granted second designation relates to pulmonary infections caused by Burkholderia cepacia (B. cepacia) pathogens.

Despite stringent infection control practices, B. cepacia infections still occur in cystic fibrosis patients and can lead to fatal sepsis. The cell envelopes of these especially virulent bacteria are impermeable to most antibiotics, which makes them particularly difficult to treat. Due to its unique mode of action, which allows the antibiotics to penetrate into the bacteria, Fluidosomes(TM)-tobramycin could become a particularly effective treatment for B. cepacia infections.

Prof. Dr. Miguel A Valvano, MD, Medical Advisor to Axentis Pharma, comments on the development: "Burkholderia cepacia is almost always multi-resistant to antibiotics and this, in conjunction with the poor prognosis of patients with B. cepacia infection, makes the treatment of these patients exceedingly complex. Tobramycin is in principle an effective antibiotic. The drug is however rather ineffective due to the impermeability of B. cepacia's cell envelope. In addition, B. cepacia - just like many other pathogens - has developed mechanisms to eliminate antibiotics once they have entered the cell. Fluidosomes(TM)-tobramycin seems to overcome these limitations by packing tobramycin into liposomes, which, by allowing effective penetration of the antibiotic into the bacterial cell, completely changes the microbiological profile of this antibiotic. Hence, Fluidosomes(TM)-tobramycin could be a totally new antibiotic formulation that addresses microbiological needs that no other antibiotic can."

What exactly happens when Fluidosomes(TM)-tobramycin encounters the bacterium is still not entirely clear, but pre-clinical data indicate a novel mode of action. Dr. Helmut Brunar, CEO of Axentis Pharma explains: "Once at the site of infection, tobramycin-containing liposomes seem to fuse with the cell membrane of the pathogen. In this way, the entire load of tobramycin contained in the Fluidosomes(TM) is released into the bacterial cell. Additionally, our data indicate that bacterial rescue mechanisms that pump tobramycin out of the cell are inhibited by the fusion process. The efficient delivery and maximum release of tobramycin into the bacterial cell together with inhibition of the clearance mechanism indicate that Fluidosomes(TM)-tobramycin has a highly efficient therapeutic effect."

About Axentis Pharma AG

Axentis Pharma is a respiratory specialty pharmaceutical company whose core competence is the combination of a fully patented, liposome-based drug delivery system with already established and well-characterized therapeutic agents. The company is using its platform delivery technology, named Fluidosomes(TM) technology, for the development of its lead product, an inhalable liposomal formulation of tobramycin. Axentis Pharma's lead product is designed to treat bacterial infections in the lungs.

About Fluidosomes(TM) technology

Axentis Pharma's Fluidosomes(TM) technology uses biocompatible lipids endogenous to the lung that are formulated into small liposomes. This nanocapsule platform offers wide-ranging potential for unmet medical needs, including chronic respiratory infections of the lung. In the case of Fluidosomes(TM)-tobramycin, the interaction between tobramycin and the microbial cell is triggered when the liposomes undergo a fusion process with the outer membrane of the bacterial cell wall. Tobramycin then penetrates into the inner cell compartment and triggers bacterial cell death.

Source: Axentis Pharma AG

http://www.medicalnewstoday.com/articles/157841.php

Cystic fibrosis treatments may have unseen long-term benefits

Cystic fibrosis medicines that help to break down mucus in the lungs may carry an unexpected long-term benefit, a study suggests

Cystic fibrosis medicines that help to break down mucus in the lungs may carry an unexpected long-term benefit, a study suggests.

The treatments not only help breathing in the short term - they may also make lung infections develop to be less harmful in the long run, research from the University of Edinburgh shows.

Scientists studied how bacteria which infect the lungs of cystic fibrosis patients gather nutrients from their surroundings. The work builds on the knowledge that most bacteria co-operate to scavenge what they need from their environment, but some bacteria do not actively hunt, instead stealing nutrients from neighbouring bacteria.

Scientists found that in a viscous environment, similar to thick mucus, the co-operating type of bacteria is most common. However, in a more liquid environment - similar to mucus having been broken down by medicine - the number of thieving bacteria increases, eventually outnumbering the scavenging type. In this environment, because the thieving bacteria are less adept at obtaining food, the bacterial growth slows down.

The results suggest that liquefying lung mucus would be expected to limit the impact of infection in cystic fibrosis.

Dr Rolf Kuemmerli, formerly a researcher at the University of Edinburgh, who led the study, said: "Treating cystic fibrosis patients with drugs that clear their lungs delivers short-term relief for the patient, but may have long-term health benefits too. We hope that our findings will underline the need for treatments that target mucus in the lungs."

Dr Freya Harrison of the University of Bath, who took part in the study, added: "Bacterial infections develop over time, and understanding how medical treatments affect this could be very important for managing long-term infections such as those found in cystic fibrosis."

Cystic fibrosis is an inherited condition that affects more than 8,000 people in the UK, according to the Cystic Fibrosis Trust. Thick mucus can clog the internal organs, especially the lungs and digestive system, making it hard to breathe and digest food.

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The study, carried out by researchers at the Universities of Edinburgh, Oxford and Bath, was published in Proceedings of the Royal Society B. Work was supported by the Royal Society and the Leverhulme Trust.

http://www.eurekalert.org/pub_releases/2009-07/uoe-cft071609.php

Second/Third Opinions

Several people commented about second/third opinions, so I thought that I would globally update on that :) I contacted both UNC and Denver. The nurse at UNC is going to talk to the doctors there and see if we can get IR to look at Gess's last procedure. That seems like the most logical first step. The IR folks at UNC are very familiar with CF patients and bleeding and Gess had his first 7 embolizations done there. The doctors rock, the nurse coordinator is AMAZING. UNC is a great clinic!

I also contacted Denver and actually spoke with Dr. Nick (the adult clinic director) about the issue. He didn't seem especially optimistic that they would be able to do anything differently for Gess regarding the bleeding. In fact, he told me that embolizations are really only good as life-saving measures. He said that they could look at Gess's overall treatment plan, etc., but said that it was likely that they would recommend anything different. So...we aren't going to plan a trip to Denver just for a clinic visit at this point. We will probably see the docs there the next time we are in Colorado (Gess's family lives there) just for another set of eyes, but it doesn't sound promising for our immediate concern.

So, right now it is UNC. The nurse should get back to me next week and then we will go from there.