Tuesday, May 31, 2011

Subject: Kindness Despite Being Ill Part 2

Dear APF Members,

I wanted to share a thought when we have bad days and we have pets, do we
ignore them, love them, and pay any attention to them?  Well I have three
dogs and usually I don't feel my best.  They each have their own
personalities, funny things they do but if we don't feel good we may at
times put them aside.  They can sense when we don't feel right, remember
that we never need to get upset with them because all they want is love and
affection when we don't feel right and our approval just as we seek it from
our family and friends. In return by showing this kindness it brings all of
us happiness and some measure of contentment.

Show kindness through loving animals and the living world. Loving animals
and caring for pets is kindness in action. Nothing compels you to care about
beings of another species, especially in a day and age where the tools of
human domination are so powerful. And yet, the very act of loving an animal
and respecting the animal for its own value is an expression of deep
kindness. As well, being kind to the world that sustains and nurtures us is
sensible as well as kind, ensuring that we don't poison the very elements
that assure us a healthy life.

o    Adopt or foster a pet. Your kindness will be rewarded by letting
another being into your life that will bring you joy and love.

o    Offer to pet-sit for a friend who is going away. Give your friend the
reassurance that someone loving and caring will be tending to her pet while
she's away.

o    Respect the species you're caring for. Humans don't "own" animals;
rather, we stand in a relationship of being responsible for their well-being
and care.

o    Take time to restore parts of your local environment with the local
community. Go for walks in nature with family, friends, alone, and commune
with the world that you're a part of. Share your love for nature with
others, to help reawaken their sense of connection with nature.

Practice the kindness effect

o    Practice kindness and generosity toward others. Being out of practice,
being shy, or not knowing how to reach out to others can only be overcome in
the doing, by continually trying until it becomes a natural impulse to be
kind and giving to others.

o    Ask for nothing in return. The greatest kindness expects nothing, comes
with no strings attached, and places no conditions on anything done or said.

o    Meditate to help spread kindness

Extend your kindness to people who are not "in your neighborhood"... Look at
all the wonderful things we have done with Porphyria Awareness Week Local,
National Media, we shared this with Dr's, family friends, neighbors, maybe
we went the extra mile got out of our comfort zone to spread the word.

"Be kind, for everyone you meet is fighting a hard battle". Attributed to
Plato, He was mentioned in this book I was just reading~ this saying is
recognition that everyone is undergoing some challenge or other in their
lives and that sometimes, it's all too easy for us to lose sight of that
when embroiled in our own problems or anger against them. Before committing
an action that might impact another person negatively, ask yourself a simple
question: "Is this kind?" If you cannot answer this in the affirmative, this
is a reminder to change your action and approach immediately.

o    Even where you're feeling at your very worst, remember that other
people are also feeling uncertainty, pain, hardship, sadness,
disappointment, and loss. In no way does this belittle your own feelings but
it does allow you to realize that people often react from their hurt and
pain rather than from their whole self, and kindness is the key to seeing
past the raging emotions and connecting with the real person inside.

o    Consider the adage "be cruel to be kind". Think about why this saying
is so popular. Do you think it is an appropriate way to view people's
situations? When you believe that someone truly needs to learn a lesson,
often one involving standing on their own two feet, one of the greatest
kindnesses that you can do for them is to withhold your judgment and to go
the extra mile to do things for them that will enable them to make the
changes or leaps of faith that they need to do, without actually trying to
make that change for them. We're all well aware that we cannot change
another human being. But kindness allows us to enable things to change
around them so that they can make the necessary changes for themselves.
Which means that we don't need to view our act as "cruel"; rather, it
becomes an act of "enabling".

o    If you're neglecting being kind to someone else just because you think
they can cope without your support or understanding, then you're practicing
selective kindness.

"Carry out a random act of kindness, with no expectation of reward, safe in
the knowledge that one day someone might do the same for you." These are the
words once said by Princess Diana. The practice of random acts of kindness
is alive and well as a conscious effort to spread more kindness, I know that
this reminder on kindness has helped me immensely in my battle with
Porphyria, how I speak with people how I view myself as a person and most am
I doing the Best that I can each and every day?  Do something today for
someone else your heart will be so happy!

From Amy Chapman AIP Patient

Tuesday, May 10, 2011


DAY AT THE FDA with Mike Kenworthy,Ph.D and Matt Johnson

I was part of the Afamelanotide trial last summer and was fortunate enough to have received the real thing. Unless you have EPP, I cannot explain what a miracle that was. But I did get the chance recently to do just that at the FDA Office of Orphan Diseases. Another fellow EPPer, Matt Johnson, joined me. In my 62 years I had never met anyone with EPP outside of my family (I have two cousins with EPP). That was an experience in itself. Those of us with EPP know that we can never explain to someone without EPP what it is like, but listening to Matt Johnson was like reading my own thoughts. Matt and I spoke a couple of times during telephone conferences before we actually met at the FDA in Silver Spring, Maryland. Although our session was scheduled for 10:30am, we were asked to come early to meet with Dr.Timothy Cote, the head of the Office of Orphan Diseases. I arrived just before 9am and Matt was close behind. Dr. Cote had just returned from Israel two day prior and was sick the day before, so he was not in good spirits, but after a few minutes listening to Matt and me, he was beaming. He told us that his staff, like all bureaucratd, tends to lose site of their mission and was delighted that we were there to remind them that we are out there.

Dr. Bob Desnick, a member of the APF Advisory Board, was also to attend, but his flight from New York delayed him an hour. By the time we were ushered into the conference room over 30 doctors and health professionals were waiting to hear what we had to say. For an hour Matt and I regaled them with the inexplicable world of EPP and the miracle of Afamelanotide during which time we were videotaped. We tried to tell them of the unrelenting pain, the isolation and self-doubt, and ultimate despair. Then we told them about our experience Last Summer and how it had changed our lives. We weren't supposed to directly discuss this, but it just couldn't stay untold - so we did. Dr. Desnick translated what we had to say into meaningful medical speak, but I believe that our words had more impact.

Like most families, we have gone to the beach almost every summer, and every summer after 15 minutes, I am banished to the house and sit with my feet in a lobster pot of ice water for the next 5 days “sipping” copious amounts of adult beverages. Needless to say this is hardly endearing to my wife and makes everyone wonder. But last summer, I spent hours on the beach with virtually no pain – a certified miracle if ever there was one. Like most EPPers, I have never told anyone outside of my immediate family about my condition. My colleagues at the office began referring to me as Mr. Tan without ever knowing why.

But the most compelling thing that day at FDA was a video that Matt had brought. It was a video of the episode that led to his diagnosis. He was 11 at the time and had gone skiing with his family. The video showed the progress of the effect of EPP over the period of about 10 days. As most of EPPers know, even though the pain has past, we are not a pretty site after 10 days. It had a profound effect on our audience.

Drs. Cote and Mueller thanked us for our time and promised that they would advocate our case to the extent they could, but could promise nothing. They explained that there is concern that the drug would be used for “off label” usage and that it would take time and effort to weigh the risks and benefits of a drug such as Afamelanotide. Dr. Mueller told us that she would share the video of our session with the FDA Center for Drug Evaluation and Research. I believe that this was a first and significant step in getting approval for the use of Afamelanotide, but I am not sure that the end is in sight. I live 45 minutes from the FDA campus and with the help of APF will continue to do whatever it takes to gain approval for use of Afamelanotide.

Friday, May 6, 2011


Since Panhematin has beeen on the news for the last month, I thought I would tell you my first expereince with the drug. When I took it so many years ago, it had not been FDA approved and subsequently becem the first ORPHAN DRUG.

That story will come at a later date.

. My doctor at NIH, Dr. Lamon , arrived at my room . I was wide-awake and ready for what I hoped would be good news. Dr. Lamon said he thought that I might benefit from treatments with infusions of hematin; however, the FDA had not yet approved heme therapy for the acute Porphyrias. Evidence from years of prior research indicated that hematin was effective and might enhance my recovery.
Since I had been very ill for quite some time, Dr. Lamon warned me that hematin was sometimes not as effective when given far along in an attack as I was then. Having never been one to stand and wait for anything, I certainly didn’t want to think more than that moment to tell him that I was willing to try the hematin as soon as possible. Participating as a research patient was also an intriguing proposal. I concluded that having the hematin infusions would provide me with the chance to benefit from the use of an experimental drug while being able to help someone else in the future. I liked that idea, too, so I was ready to begin the treatment.
I later learned that the rationale for administering heme therapy is to correct a heme deficiency in the liver and repress production of porphyrin precursors. Since the first report in 1971, numerous patients were given heme therapy and clinically benefited from the treatment. Although there have been no controlled studies, the reported results were impressive enough to advocate the early use of heme therapy for porphyric attacks. When heme therapy is given late in an attack, no substantial benefit may occur; but if given early, the heme therapy almost always normalizes ordinary porphyrin and porphyrin precursor values.
If a patient with signs and symptoms of a Porphyria attack is not stabilized within a day by glucose and other care, most specialists recommend the administration of heme therapy. Usually, a dose of 3-4 mg/kg, given once daily for four days, produces a highly beneficial effect in most patients. A decrease in pulse rate, blood pressure, abdominal pain and decreased levels of urinary porphobilinogen are commonly noted and can occur within a day. At the time, I didn’t know what would happen, but I was certainly ready to try.
The following morning Dr. Lamon walked into my room holding an extremely large syringe full of hematin for me to see. I gasped and remarked that the concoction looked like swamp water, a black/green color. Some patients have since told me that they thought that hematin looked like crankcase oil or a prop in a Frankenstein movie. Regardless, I was ready to receive the infusion. At that time, Dr. Lamon infused the remedy into a vein in my arm with a butterfly needle and a very large syringe. He repeated the process daily for five days.
The hematin may have looked like swamp water or crankcase oil, but it was 24K gold to me. After only a few infusions, I improved dramatically despite the fact that I had been ill for a number of weeks. Relief from my constant pain and nausea occurred quickly, but the extreme weakness remained for months thereafter. (Hematin was the first drug approved by the FDA for rare diseases under the Orphan Drug Act many years ago. It became available from Abbott Laboratories in Chicago under the trade name Panhematin. It has since been sold and is now available through Lundbeck, Inc , which purchased Ovation Pharmaceuticals ..)
My doctor now gives me Panhematin when I have an attack. . Since many Porphyria patients asked how my doctor arranged for the treatments, I have included the following description of the usual scenario of the infusion process. The IV infusion is much different now than it was when Dr. Lamon infused the hematin by hand with a large syringe and a butterfly needle.
Now when symptoms of an attack of AIP begin, my docotr immediately orders Panhematin through the hospital pharmacy. Some people take it at local infusions centers , in their doctors offices and soem who are on home care take it at home...Most of the time, the hospital pharmacy or whatever the source does not maintain Panhematin on hand. Therefore, it is ordered from Lundbeck and is shipped immediately from their emergency supply. In fact, it can arrive within a matter of 12 hours since they keep a supply neaar the airport in Chicago.

More to come .

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