Rare Disease Day: I have Cushing's

Today, I am thankful for the friends I have met along the way. Cushing's has devastated our lives in unimaginable ways, but it has brought courageously delightful people into my friendsphere.  

I thank Trisha for her efforts to highlight the difficulty in diagnosing this rare disease.  Please consider taking just a few moments to watch this youtube video she put together for our whole community.

Cushing's Disease: A Numbers Game

CORT Does Well in After Hours Trading

I first saw the word Cushing's almost five years ago. Even at that time, there was talk in the Cushing's community of a far-away land where magical medicine brought cures when surgeries couldn't. We have waited a long time for this.

Many of us patients with Cushing's have been closely following Corcept's march towards FDA approval for the drug (Korlym, was Corlux).  It seems investors took the time they needed to research Corcept a little more over the long weekend, because folks are buying the stock and driving the price up. 

Could the market surge have been from all of us Cushies?  Maybe a portion, but not too much.  We spend all our money on medical bills!
  

I wanted to share the article Corcept Has Upside Following Drug Approval. I posted a comment under the article,  and I hope that it will be accepted and posted (hi Adam!!).  This is what I wrote:

I have persistent Cushing's disease, and I will be eligible to take Korlym. I am also a stockholder. Wanna know more about the devastating effects of Cushing's? Read more about it on my blog, http://cushingsmoxie.blogspot.com/.  Besides an investment, this knowledge may save the life of someone you know.

*****

Drugs

 Return to Article

Corcept Has Upside Following Drug Approval

Adam Feuerstein

02/21/12 - 12:54 PM EST

A foul serving of crow is what I deserve for not believing Corcept would get Korlym approved on its first attempt. More on what I got wrong later; first, let's look at Corcept's future with Korlym approved.
Corcept plans to launch Korlym on May 1. The company has yet to disclose pricing but Alan Leong of Biotech Stock Research estimates conservatively one year of therapy will cost $75,000. [Leong deserves kudos for correctly predicting Korlym's approval.]
Again being conservative, Leong estimates that about 2,000 Cushing's patients in the U.S. will be eligible for Korlym therapy, leading to $150 million in U.S. sales by 2016. Corcept is pursuing Korlym approval in Europe, which could ultimately generate another $75 million in sales or $225 million total.
Corcept intends to market Korlym on its own in the U.S. and will only need to hire a small sales force since most Cushing's patients are treated by a handful of specialists. This means Corcept should be able to maintain high profit margins.
The FDA stated that as many as 5,000 patients may be eligible for Korlym treatment, which implies a peak U.S. market opportunity of $375 million (assuming $75,000-per-year pricing.)
Corcept's current market cap of $370 million is not fully valuing Korlym's market potential. Why? Likely because some early investors in the company are taking profits now while others may wait to see how Korlym launches before jumping in.
Corcept is also studying Korlym as a treatment for psychotic depression and weight gain caused by antipyschotics.
Cushing's syndrome is a rare condition in which a tumor on an adrenal gland like the pituitary causes excess production of the hormone ACTH. Excess ACTH, in turn, causes the body to produce too much of the stress hormone cortisol. Patients with Cushing's suffer from severe cardiovascular and metabolic disease that can be fatal.
FDA approved Korlym to control high blood sugar levels in adults with Cushing's. The drug works by preventing cortisol from binding to its receptor thereby blunting cortisol's effects on the body. Korlym does not decrease levels of cortisol in the body.
Corcept also sought approval for Korlym to treat hypertension in Cushing's patients but FDA rejected that claim.
I previously predicted an FDA rejection for Korlym -- a call that obviously went wrong. My biggest mistake was probably under-estimating the significance of Cushing's being a rare, life-threatening disease with no current treatment options. Korlym causes significant side effects but the drug is also well understood by FDA (the active ingredient is mifepristone also known as the "abortion pill" RU-486) and the benefits in this vulnerable Cushing's patient population outweigh the risks. I didn't think through the risk-benefit equation for Korlym well enough.
I also placed too much emphasis on a change in Corcept's risk factors (disclosed in the company's SEC filings) that warned of FDA possibly requiring additional pre-approval requirements for Korlym in Cushing's. In the past, changes like this made to a company's SEC filings have proven to be reliable indicators of trouble, but not this time.
--Written by Adam Feuerstein in Boston.
>To contact the writer of this article, click here: Adam Feuerstein.

There is No Reasoning with the Unreasonable

My New Life as a Cushie Non Grata

Ever have one of those weeks that convinces you every planet must be in retrograde? Welcome to my world. This past week was a doozy for me.

With perspective being the name of my game in 2012, I remember how I vowed just six weeks ago to focus my energies.  I needed time and the New Years resolution to help me focus on myself, not others. I decided to focus my energies towards what I considered the most importants things in my own life -- my family, goals, wishes, desires, and of course, my health.  

Without detailing all the changes swirling around which are bringing me just what I want in 2012, I am proud to say that I have made progress on several fronts.

Then BOOM. The floor drops out from underneath me. Not once. Not twice. Three times. Big seismic shifts in my medical life.

Through the tears of hurt, misunderstanding, and devastation, I have done a lot of soul-searching.

I am reminded of one of my favorite cartoons about the internet, social media, blogs, and message boards -- any and all outlets that let us interact with those 'like' us or who share a common interest, philosophy, or way of being.

MediaTapper.com's brilliant piece analyzing the cartoon of the same name: Someone is Wrong on the Internet is so well written I won't attempt to resummarize.  

I will admit that I laugh every time I see this.  I nod in recognition.  I know that person in the desk chair. She lives occasionally in my home, too. She and I share a seat sometimes, late at night, as my cortisol surges and keeps me awake, surfing the internet, desperate to connect with those like me.

I see her spirit seize those I see through my computer screen much too often, as they become hell bent on setting all manners, dignity and self-respect aside in an attempt to prove that you are the wrong-doer. They confront for sport (or to win JERK points at the JERK store, for you Seinfeld fans), saying things I am pretty sure they would never muster the courage to say in a face-to-face encounter.

I am reminded too often that the internet brings out that sinister side of human nature that can't leave well enough along, that won't let up until they see you suffer. It's a very ugly, dark, nasty side.

When I started this blog in May 2008, I never dreamed I would say the following:

Since this is the only place where I can say what I want without fear of being deleted or outvoted by the committee of prevailing opinions, let me take a moment to be very clear.

I do not want to be that person in the desk chair.

I am no longer that person in the desk chair.

I will not let anyone goad me into being that person in the desk chair.  

I assure you.

I will not let them turn me into that person in that desk chair.
I won't let that be me.

I realized this online debating thing could be a problem for me a long time ago. Faced with fear and armed with odd symptoms, tears but always a little hope, I started communicating others who suffered with the same disease I thought I had almost five years ago.  Through the years, the community and many individual members have saved my life with compassion and knowledge. I will always be thankful to so many.  However, every third triumph was accompanied with a subtle stumbling block, something I had to "get over."  Over time, I noticed triumphs mixed with veiled sarcasm, well-masked ill intentions, words-as-daggers, and group consensus.

Undeterred, I fought though struggled to keep my place in a community of those like the 'new' me--the sick me. This always baffled my husband, whose spent many hours listening to my woes and fears of not belonging.

Why bother with all that? Stay here close to me, he said. We will figure it out. We will get through this together.

With this seed planted in my mind, our own Cushing's experiences provided footing in an unfamiliar world. Fears of belonging and falling were soon replaced with balance and moderation. Shhhh, don't tell my husband, but in secret, I would continue to daily struggle to belong.

Slowly but surely, I did more of what I didn't think I could do. I felt myself being reabsorbed into the family that I was trying to build and sustain despite Cushing's. It is my family in my home, I realized, that I needed to sustain me through this medical journey.

This week, my little engine that could life-modification project got a violent shove into priority slot # 1 this week. Without notice, without any chance to clarify, I would come to find out that 1) my services were no longer needed and 2) services are no longer available to me. My patient life, experience sharing, community of support -- all deleted.


Dumbfounded. Betrayed. Rejected. Misunderstood. Mischaracterized. Overwhelmed. Hurt. Disappointed. Powerless. And yet Relieved.

I have proudly moved away from the desk, back into my life, and when my cortisol is not too high at night, even back into bed. 

* * * * * * * * *

There is no reasoning with the unreasonable.

It may take a little bit of time to sink in through any fears of reputation-tainting. So it bears repeating. 

There is no reasoning with the unreasonable.

* * * * * * * * *

Social Media in Moderation, I welcome you!

I must accept the positives it brings into my life -- connecting with my family and friends all over the world and sharing information about Cushing's with you readers, old and new.  While I regret missing announcements (surgeries, falls, adrenal crises, reoccurences), I know I must be remain positive, move forward, and use the internet in a way that feeds my soul, not bleeds it. I can, more importantly, reject any of the negative that it brings my way.  

This works for me as a Cushie, and it makes sense for anyone who is trying to surround themselves with folks that lift them up, not tear them down. 

Grab those around you that you want to take on this journey called life. Embrace them and say, Listen buddy, you are coming with me! Make sure they always know you care.  Leave behind all the rest to gather dust in a closed chapter in the story of your life. Move on and build new memories.

It is my hope that you consider this as important of any experience I've shared with you over the years.

For we Cushies--determined, willful, courageous beautiful Cushies--should not reserve one modicum of space for unwarranted stress in our life.

May you stand strong when good decisions are made for you.  ~Melissa

FDA approves first medication for Cushing's patients

This is excellent news for all my fellow Cushies whose excess cortisol led to type 2 diabetes.  I remain uncured with persistent cushing's disease.
Other medications are expected in 2012, but each has great risks. Hopefully, with this many pharmaceutical companies working at it, something useful will stick. I don't know how long folks like me can wait. It has been five long years already.
-- melissa


FDA approves Korlym for patients with endogenous Cushing’s syndrome
Today, Korlym (mifepristone) was approved by the U.S. Food and Drug Administration to control high blood sugar levels (hyperglycemia) in adults with endogenous Cushing’s syndrome. This drug was approved for use in patients with endogenous Cushing’s syndrome who have type 2 diabetes or glucose intolerance and are not candidates for surgery or who have not responded to prior surgery. Korlym should never be used (contraindicated) by pregnant women.
Prior to FDA’s approval of Korlym, there were no approved medical therapies for the treatment of endogenous Cushing’s syndrome.
Endogenous Cushing’s syndrome is a serious, debilitating and rare multisystem disorder. It is caused by the overproduction of cortisol (a steroid hormone that increases blood sugar levels) by the adrenal glands. This syndrome most commonly affects adults between the ages of 25 and 40. About 5,000 patients will be eligible for Korlym treatment, which received an orphan drug designation by the FDA in 2007.
Korlym blocks the binding of cortisol to its receptor. It does not decrease cortisol production but reduces the effects of excess cortisol, such as high blood sugar levels.
The safety and efficacy of Korlym in patients with endogenous Cushing’s syndrome was evaluated in a clinical trial with 50 patients. A separate open-label extension of this trial is ongoing. Additional evidence supporting the agency’s approval included several safety pharmacology studies, drug-drug interaction studies and published scientific literature. Patients experienced significant improvement in blood sugar control during Korlym treatment, including some patients who had marked reductions in their insulin requirements. Improvements in clinical signs and symptoms were reported by some patients.
The most common side effects experienced by endogenous Cushing’s syndrome patients treated with Korlym in clinical trials were nausea, fatigue, headache, arthralgia, vomiting, swelling of the extremities, dizziness and decreased appetite. Other side effects of Korlym include adrenal insufficiency, low potassium levels, vaginal bleeding and a potential for heart conduction abnormalities. Certain drugs used in combination with Korlym may increase its drug level. Health care professionals must be aware of the potential for drug-drug interactions and adjust dosing or avoid using certain drugs with Korlym.
Korlym should never be used by pregnant women. Although pregnancy is an extremely rare occurrence in Cushing’s syndrome patients because of the suppressive effect of excess cortisol on female reproductive function, Korlym will carry a Boxed Warning advising health care professionals and patients that the therapy will terminate a pregnancy.
The FDA has determined that a Risk Evaluation and Mitigation Strategy (REMS) is not necessary for Korlym to ensure that the benefits outweigh the risks for patients with endogenous Cushing’s syndrome. Several factors were considered in this determination including the following:

• There are no other approved medical therapies for this debilitating form of Cushing’s syndrome and very sick patients would suffer if impediments to access were imposed.
• The number of Cushing’s syndrome patients who will require treatment with Korlym is small, with an estimated 5,000 patients being eligible for treatment.
• The number of health care professionals in the United States who would potentially prescribe Korlym is very small and highly specialized. They are familiar with the risks of Korlym treatment in the endogenous Cushing’s syndrome population and frequently monitor patient status.
• The risks of Korlym treatment in the intended population can be managed through physician and patient labeling. The risks associated with Korlym will be outlined in a medication guide for patients.

The company has voluntarily proposed distributing Korlym through a central pharmacy to ensure the timely, convenient and appropriate delivery of the drug to Cushing’s patients or to the health care institutions where this therapy may be initiated. Most retail pharmacies are unlikely to keep adequate supplies of the drug for this rare condition and central distribution will give patients with Cushing’s syndrome better access to Korlym.
Korlym is manufactured by Corcept Therapeutics of Menlo Park, Calif.
For more information:

• Approved Drugs: Questions and Answers

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm292462.htm

FDA NEWS RELEASE

For Immediate Release: Feb. 17, 2012Media Inquiries: Morgan Liscinsky, 301-796-0397; morgan.liscinsky@fda.hhs.gov

Consumer Inquiries: 888-INFO-FDA

Pituitary Surgery & Awesome Diagrams

I never tire of reading about pituitary surgery. Having done it twice, I am still fascinated with the process. Recently, fellow Cushie Joann brought a new page to my attention. 

Mayfield Clinic has created an outstanding, easy-to-understand document for pituitary patients. You can also download the pdf, too.

 

After reviewing the multiple diagrams of the pituitary smack dab in the middle of the skull, I am reminded of others who diminish the severity of pituitary surgery.  They claim that pituitary surgery is not really brain surgery. The debate swirls among Cushies in the Cushing's community.  For me, it is very clear.  I have always been pretty outspoken that it is brain surgery. 

Let's review some basic facts. 

First, the brain is protected by the cranium because the brain is so important to the body's function. In the same way, the sella turcica, a bony little saddle where the pituitary sits, protects its passenger in the same way, for the same reason. Do you see my point?  Yes, thank you. 

Second, the neurosurgeon must drill a hole through the sella turcica to gain access to the pituitary.  If anyone is drilling anything in your skull to remove a tumor, I call this brain surgery!

Third, the American Brain Tumor Association lets us pituitary patients in the club. So does the National Brain Tumor Foundation.  Why would anyone really begrudge us?

Lastly, did you see how central the pituitary is to the brain? It is smack dab in the middle:  halfway between the ears, halfway between the nose and back of the head.  

Folks, really.  If you can't see how this is all taking place in the brain, I really can't make you see it any clearly.  I am all for facts, but sometimes, there is just no helping someone see what is right there at the top of the nose (internally).  

~Moxie Melissa

Share your story. Shape the future of Cushing's research and treatment.

** Please contact Corcept no later than Wednesday, 2/8/12. **

People with persistent Cushing’s Syndrome are invited to participate in market research and give input to the creation of patient information, tools, and resources. 

People with Cushing’s Syndrome (Cushing’s Disease, ectopic Cushing’s and adrenal Cushing’s) are invited to participate in market research and give input into the creation of patient information, tools, and resources. 

Patients are not eligible to participate if: 

• you were diagnosed more than 10 years ago (memory fades with time!)
• you have had a bilateral adrenalectomy
• you are in remission 

This market research will be conducted in a confidential, one-on-one interview that can take place over the phone or in-person in a central location near you. If you are interested in a phone interview or in-person interview, please contact Clair Carmichael Johnstone at cushing@compasshc.com or call (800) 856-6706. More information (including cities and locations for in-person interviews) will be provided on the phone. Those who participate in an in-person interview will be compensated $250 and those who choose a phone interview will receive $125 for their time. Thank you in advance!

=====

I spoke with Clair earlier this week. She was very supportive, and she is a great listener. Contact her today!

Melissa