ALEX MAISH
Nowadays, it was said, peers, not parents,
set the standards
for normal.
BY ALEX MAISH
In June, I had the opportunity to attend the CF Worldwide meeting held in Belfast, Northern Ireland, which was covered in the last CF Roundtable issue, and then I was able to stay for the 26th European CF Medical Conference held for the next two-and-a-half days. Having a technical background, I am always interested in the details of the latest medical research on CF and am awed by how much researchers have been able to learn in the past two or three decades about the complex functioning of our bodies.
It is truly amazing how much we know about the physical and chemical processes that occur in each of our cells. Research on CF, spearheaded by the CF Foundation’s direction and innovative concepts, employs and promotes some of the most advanced biotechnology techniques available. I looked forward to the opportunity to get a first-hand update on the state of CF medical research. With up to six concurrent sessions, it was impossible to attend more than a smattering of the talks presented. There were talks on all level of research, from cell membrane transport, the CFTR protein’s defects, Pseudomonas aeruginosa and other infectious bacteria, and anti-inflammatory therapy, including Zithromax. Much of it was very detailed, thorough, and careful.
Current CF medical research is largely focused in two directions, correcting or bypassing the basic defect, and reducing the damage caused by infection and the body’s inflammatory response to it. While researchers haven’t completely given up on curing CF by inserting the correct gene into lung cells, the mainstream research has moved on to the process downstream as the gene gets expressed as a working protein. The over 1000 different defect variations in the CF gene have been categorized into 5 classes depending on what stage this process gets interrupted, and work is progressing on methods to correct or bypass the process for each class.
Class I gets interrupted right at the start due to translation errors going from the DNA gene to the RNA working copy. Aminoglycosides, which include tobramycin, are being looked at to partly reverse this. Class II defects, which include the common delta-F508 defect, don’t make it all the way to the cell membrane. Chaperone molecules, such as MPB-07 and CPX, protect the mutant protein during processing allowing it to make it to the cell wall, and other drugs stimulate it to work at least partially. Classes III-V make it to the cell wall, but don’t correctly regulate ion transportation for various reasons.
Approaches that include stimulation of alternative channels, such as those active pre-birth, are also being investigated. There is much research ongoing into understanding P. aeruginosa and B. cepacia, mainly learning the structure and chemistry of the biofilm colonies they form including how they attach themselves to airways and how they protect themselves. Disrupting these mushroom-shaped mucous-covered film structures, which include internal nutrient rivers and complex chemical communication, is important in eliminating the infection. The hot infection therapy research is focused on macrolides, which include Zithromax, and how they may affect these biofilm structures and reduce the body’s inflammatory response.
While my technical side enjoys learning about the complexities of cell biology, as a CF patient, I find myself impatient to get to the results that had impact on patient therapies. I quickly found myself drawn to the psycho-social presentations, in part because it was mainly new territory for me and in part because it seemed to have so much to say towards achieving present benefits.
Several talks were geared to helping parents and caregivers deal effectively with kids and teens growing up with CF. Others dealt with CF psychotherapy, end-of-life decisions, theories of health behaviors, and behavior change therapy. I am impressed by the breadth of strategies available that make sense, by how much research and knowledge is available, and equally by how little of it is trickling down to the people that can use that knowledge; the parents, adults, and the CF center staff.
In my experience there is an incredible need for such psycho-social support in the CF community. In New Mexico where I live, it has been a real struggle just to maintain minimal physician care, to say nothing of nursing or psycho-social support, in this age of increasingly tight medical budgets. Until recently our adult program didn’t even have a social worker. There has been no pro-active effort to identify the needs of CF adults, and no resources to provide psycho-social support. At best, we have had some newsletters and Family Day gatherings, as the risk of cross-infection has restricted gatherings that could provide much needed interaction with other CF adults and families.
Most of the CF adults that I know don’t know or communicate with anyone else who has CF! Doctors don’t have the time to work with patients to integrate their care into their lives, and the rest of the staff is severely overloaded as well. Patients are left to fend for themselves.
What can be done in the present situation to promote better psycho-social support for CF adults? A program in England, funded by their lottery, employs a dozen CF adults as CF Patient Advocates who work closely with the CF teams in the numerous clinics to enact changes. They meet with parents and partners, but not directly with adults with CF, and use the phone, e-mail, video conferences, message board, surveys, letters, and web sites to elicit views and concerns. They then work with health authority contacts, politicians, hospital management, and clinicians to enact changes. While this approach doesn’t provide psycho-social support, perhaps it could be used in the U.S. to push for more such support for CF families.
There was too much information given at the talks I attended to relay it all, but I do want to mention a few concepts that were presented. Some of the talks about kids and adolescents contained ideas of value to adults as well. Communication is important. Enabling people to ask questions, and providing knowledge before it is needed can help not only for daily therapy, such as providing details on drugs, dosages, etc., but also as patients approach transplants or end-of-life. It was mentioned that sometimes the hang-up is with the physician or therapist rather than the patient being uncomfortable with the subjects.
One talk focused on the power of using peers in CF care.1 Nowadays, it was said, peers, not parents, set the standards for normal. Using them as a positive force in care can be very powerful for adolescents, and I would argue for adults as well. Often, it was reported, illness drives people closer to family rather than allowing the normal breaking away. Taking peers to clinic visits and therapy sessions in the status of a family member can provide a great source of support, companionship, and acceptance. Peers were also included at meals and during exercise. In nearly all cases peers were found to encourage adherence to therapy and to provide a positive influence.
The problem of non-adherence to therapy was highlighted by another talk that stated that adherence averaged only 50%. This broke down to 90% for pills, 50% for nebs, and only 30% for diet. Fully 38% of patients did no chest physiotherapy! All our advanced medicine does little good if it doesn’t reach the patient. Potential problems with using peers in CF care exist if the friendship ends, due to the knowledge held by the peer, and in putting pressure on the peer.
A talk on CF psychotherapy, directed at therapists, stated that most therapy is skill teaching and psycho-pathology directed, but it questioned whether CF adults actually lacked skills or if often they were just trying to deal with life questions and existential issues (the meaning of life). It asked if there should be more emphasis on considering core values in life and providing an unbiased assessment of coping strategies. Strategies it mentioned were identifying traps and dilemmas (false - either/or choices, such as should I go out and get hurt, or stay home and be lonely), snags (in my family we don’t talk negatively of others) and false beliefs (People love me because I’m active, cheerful and social. When I’m not healthy and need help, I’m not lovable.) It stated that 10% of 250 CF patients studied got counseling and supportive guidance.
Another presentation discussed Motivational Intervention (MI), a counseling style for therapists to elicit behavior change that is powerful because it uses the person’s own intrinsic motivation and resources for change.2 The principles involve expressing empathy (using reflective listening, where you repeat back what is said, and acceptance to facilitate change), developing discrepancy, rolling with resistance (not pushing), and supporting self-efficacy. The person, not the counselor, should present arguments for change and should be motivated by a perceived discrepancy between present behavior and important personal goals or values. Resistance to change isn’t directly opposed, and the counselor’s and person’s own belief in their ability to change sets the stage for it to happen. MI builds a positive relationship with the patients, and it respects their values and goals. Research has been done on using MI for diet, physical activity, and adherence to therapy. In the Belfast CF clinic, an attempt was made to conduct some skills training and try the method, but it was limited and difficult to fit in to the clinic schedule.
While medical research is impressive and important, it can be frustratingly slow. Over a decade ago CF was cured in the test tube, in vitro, by introducing CFTR into cells, but translating that into a cure in a person, in vivo, has been thwarted so far by the incredibly complex cellular defense mechanisms that living organisms have developed. Often the advances that have the most impact are found by accident rather than the careful advances of rigorous research, such as happened with Zithromax. Researchers still aren’t sure why it is so effective in some people.
While I believe the detailed medical research approach will have major benefits in the long run, I feel it is important not to overlook the immediate benefits we can achieve with psycho-social support and good old-fashioned care. With the medical care meltdown and cost-cutting that is occurring in this country, these programs are often the ones that don’t get supported. But they are things we can do today, and they can have a major impact on quality of life, which the research community often overlooks.
In one of the talks at the conference, about getting children’s consent to surgery, I was struck by the point that “children’s lives are worthwhile now, not for what they will develop into.” Likewise, we need to keep that same thought in mind about CF research and care. While there is a place for careful, advanced CF research that may one day give us a big breakthrough, there is much we can do today, through psycho-social support, to improve the quality of life of those with CF.