Day Twenty-Six, Cushing’s Awareness Challenge

 

I have seen this image several places online and it never ceases to crack me up.  Sometimes, we really have strange things going on inside our bodies.

Usually, unlike Kermit, we ourselves know that something isn’t quite right, even before the doctors know.  Keep in touch with your own body so you’ll know, even before the MRI.

I asked doctors for several years – PCP, gynecologist, neurologist, podiatrist – all said the now-famous refrain. It’s too rare. You couldn’t have Cushing’s. I kept persisting in my reading, making copies of library texts even when I didn’t understand them, keeping notes. I just knew that someone, somewhere would “discover” that I had Cushing’s.

Finally, someone did.

These days, there’s no excuse to keep you from learning all you can about what’s going on with you.  There’s your computer and the internet.  Keep reading and learning all you can.  You have a vested interest in what’s going on inside, not your doctor.

 

 

 

Novartis drug Signifor approved in the EU as the first medication to treat patients with Cushing’s disease

* Reuters is not responsible for the content in this press release.

Wed Apr 25, 2012

Novartis International AG / Novartis drug SigniforR approved in the EU as the first medication to treat patients with Cushing’s disease . Processed and transmitted by Thomson Reuters ONE. The issuer is solely responsible for the content of this announcement.

Signifor is first targeted approach for Cushing’s disease,

  • a debilitating endocrine disorder caused by an underlying pituitary tumor that triggers excess cortisol[1],[2],[3]
  • Majority of patients in the Phase III clinical trial experienced a rapid and sustained decrease in mean cortisol levels with a subset of patients achieving normalization
  • With reduced cortisol levels, key clinical manifestations of the disease improved, including reductions in blood pressure, cholesterol, weight and body mass index[1]

Basel, April 25, 2012 – Novartis announced today that the European Commission has approved SigniforR (pasireotide) for the treatment of adult patients with Cushing’s disease for whom surgery is not an option or for whom surgery has failed[1]. Signifor is the first medicine to be approved in the European Union (EU) targeting Cushing’s disease.

The approval is based on data from the largest randomized Phase III study to evaluate a medical therapy in patients with Cushing’s disease, a disorder caused by excess cortisol in the body due to the presence of a non-cancerous pituitary tumor[1],[2],[3]. In the study, mean urinary-free cortisol (UFC) levels were normalized in 26.3% and 14.6% of the 162 patients randomized to receive Signifor 900µg and 600µg subcutaneous (sc) injection twice daily, respectively, at month six. The primary endpoint, the proportion of patients who achieved normalization of UFC after six months without dose up-titration relative to randomized dose, was met in patients treated with 900µg twice daily[4].

In addition, the study showed the majority of the patients remaining on the study at month six (91 out of 103 patients; 88%) had any reduction in their mean UFC[5]. The median reduction in mean UFC was 47.9% in both dose groups. Reductions in UFC were rapid and sustained through the end of the study, with the majority of patients experiencing a decrease within the first two months[4].

Overall reductions in the clinical manifestations of Cushing’s disease, including blood pressure, total cholesterol, weight and body mass index, were observed at months six and twelve in patients with both full and partial mean UFC control, with the greatest reductions observed in patients with normalized UFC levels[1],[4].

“As the first therapeutic option to specifically target Cushing’s disease, Signifor has the potential to redefine treatment of this debilitating disease,” said Hervé Hoppenot, President, Novartis Oncology. “By focusing research efforts on our understanding of this rare disease where there is significant unmet need, we have been able to successfully bring a novel treatment option to patients in the European Union.”

Cushing’s disease most commonly affects adults as young as 20 to 50 years and affects women three times more often than men. It may present with weight gain, central obesity, a round, red and full face, severe fatigue and weakness, striae (purple stretch marks), high blood pressure, depression and anxiety[2],[3],[6],[7].

“Patients with Cushing’s disease often struggle with a variety of debilitating health issues associated with the overproduction of cortisol and previously were faced with a treatment approach limited to surgery,” said Ellen van Veldhuizen, board member of the Dutch Adrenal Society. “The approval of pasireotide as a new treatment option that may help patients with Cushing’s disease is welcome news.”

The decision follows the positive opinion the Committee for Medicinal Products for Human Use (CHMP) adopted for Signifor in January 2012 for the treatment of Cushing’s disease and applies to all 27 EU member states, plus Iceland and Norway. Signifor has orphan drug designation for Cushing’s disease, a condition which affects no more than five in 10,000 people in the EU, the threshold for orphan designation[8],[9]. Additional regulatory submissions for pasireotide for the treatment of Cushing’s disease are under way worldwide.

About Cushing’s disease
Cushing’s syndrome is an endocrine disorder caused by excessive cortisol, a vital hormone that regulates metabolism, maintains cardiovascular function and helps the body respond to stress. Cushing’s disease is a form of Cushing’s syndrome, in which excess cortisol production is triggered by an adrenocorticotropic hormone (ACTH)-secreting pituitary adenoma. It is a rare but serious disease that affects approximately one to two patients per million per year. The first line and most common treatment approach for Cushing’s disease is surgical removal of the tumor[2],[3],[10].

About PASPORT-CUSHINGS
PASPORT-CUSHINGS (PASireotide clinical trial PORTfolio – CUSHING’S disease) is a prospective, randomized, double-blind, Phase III study conducted at 68 sites in 18 countries. The study evaluated the efficacy and safety of Signifor in 162 adult patients with persistent or recurrent Cushing’s disease and UFC levels greater than 1.5 times the upper limit of normal (ULN), as well as in patients with newly diagnosed Cushing’s disease who were not candidates for surgery[4].

Patients with primarily moderate to severe hypercortisolism were randomized to receive Signifor sc injection in doses of 900µg (n=80) or 600µg (n=82) twice daily. The primary endpoint was the proportion of patients who achieved normalization of UFC after six months without dose up-titration relative to randomized dose, which was met in patients treated with 900µg twice daily[4].

About Signifor (pasireotide)
Signifor (pasireotide) is approved in the European Union (EU) for the treatment of adult patients with Cushing’s disease for whom surgery is not an option or for whom surgery has failed. For the treatment of Cushing’s disease, Signifor has been studied as a twice-daily subcutaneous (sc) injection and is currently being evaluated as a long-acting release (LAR), once-monthly intramuscular (IM) injection as part of a global Phase III program. Signifor is a multireceptor targeting somatostatin analog (SSA) that binds with high affinity to four of the five somatostatin receptor subtypes (sst 1, 2, 3 and 5)[1],[3],[11].

Information about Novartis clinical trials for pasireotide can be obtained by healthcare professionals at www.pasporttrials.com.

Important Safety Information about Signifor
Signifor is contraindicated in patients with hypersensitivity to the active substances in Signifor or to any of the excipients and in patients with severe liver impairment.

Alterations in blood glucose levels have been frequently reported in healthy volunteers and patients treated with Signifor. Glycemic status should be assessed prior to starting treatment with Signifor. Patients need to be monitored for hyperglycemia; if hyperglycemia develops, the initiation or adjustment of antidiabetic treatment is recommended. Dose reduction or treatment discontinuation should be considered if uncontrolled hyperglycemia persists. After treatment discontinuation, glycemic monitoring (e.g. FPG or HbA1c) should be done according to clinical practice.

Monitoring of liver function is recommended prior to starting treatment with Signifor and after one, two, four, eight and twelve weeks during treatment and thereafter as clinically indicated. Therapy should be discontinued if the patient develops jaundice, other clinical signs of significant liver dysfunctions, sustained AST (aminotransferases) or ALT (alanine aminotransferase) increase five times the upper limit of normal (ULN) or greater, or if ALT or AST increase three times ULN with concurrent bilirubin elevation greater than two times ULN.

Patients with cardiac disease and/or risk factors for bradycardia need to be closely monitored. Caution is to be exercised in patients who have or may develop QT prolongation. Hypokalemia or hypomagnesemia must be corrected prior to initiating therapy and monitored thereafter. Electrocardiography should be performed prior to the start of Signifor therapy and as clinically indicated thereafter.

Treatment with Signifor leads to rapid suppression of adrenocorticotropic hormone (ACTH) secretion in Cushing’s disease patients. Patients need to be monitored and instructed how to monitor for signs and symptoms of hypocortisolism. Temporary exogenous steroid (glucocorticoid) replacement therapy and/or dose reduction or interruption of Signifor therapy may be necessary.

Monitoring of gallbladder and pituitary hormones is recommended prior to initiating treatment and periodically thereafter.

Signifor should not be used during pregnancy unless clearly necessary. Breast feeding should be discontinued during treatment with Signifor.

Signifor may affect the way other medicines work, and other medicines can affect how Signifor works. Caution is to be exercised with the concomitant use of drugs with low therapeutic index mainly metabolized by CYP3A4, bromocriptine, cyclosporine, anti-arrhythmic medicines or drugs that may lead to QT prolongation.

The most frequently reported adverse events (AE) (>10%) by investigators for Signifor were diarrhea, nausea, hyperglycemia, cholelithiasis, abdominal pain, diabetes mellitus, injection site reactions, fatigue and increased glycosylated hemoglobin (HbA1c), with most events being Grade 1-2. The tolerability profile of Signifor was similar to that of other somatostatin analogs with the exception of the greater degree of hyperglycemia[1].

Disclaimer
The foregoing release contains forward-looking statements that can be identified by terminology such as “potential,” “under way,” or similar expressions, or by express or implied discussions regarding potential future marketing approvals for Signifor or regarding potential future revenues from Signifor. You should not place undue reliance on these statements. Such forward-looking statements reflect the current views of management regarding future events, and involve known and unknown risks, uncertainties and other factors that may cause actual results with Signifor to be materially different from any future results, performance or achievements expressed or implied by such statements. There can be no guarantee that Signifor, or its LAR version, will be approved for sale, or for any additional indications, in any market, or at any particular time. Nor can there be any guarantee that Signifor will achieve any particular levels of revenue in the future. In particular, management’s expectations regarding Signifor could be affected by, among other things, unexpected regulatory actions or delays or government regulation generally; unexpected clinical trial results, including unexpected new clinical data and unexpected additional analysis of existing clinical data; government, industry and general public pricing pressures; the company’s ability to obtain or maintain patent or other proprietary intellectual property protection; competition in general; unexpected manufacturing issues; the impact that the foregoing factors could have on the values attributed to the Novartis Group’s assets and liabilities as recorded in the Group’s consolidated balance sheet, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those anticipated, believed, estimated or expected. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

About Novartis
Novartis provides innovative healthcare solutions that address the evolving needs of patients and societies. Headquartered in Basel, Switzerland, Novartis offers a diversified portfolio to best meet these needs: innovative medicines, eye care, cost-saving generic pharmaceuticals, preventive vaccines and diagnostic tools, over-the-counter and animal health products. Novartis is the only global company with leading positions in these areas. In 2011, the Group’s continuing operations achieved net sales of USD 58.6 billion, while approximately USD 9.6 billion (USD 9.2 billion excluding impairment and amortization charges) was invested in R&D throughout the Group. Novartis Group companies employ approximately 124,000 full-time-equivalent associates and operate in more than 140 countries around the world. For more information, please visit http://www.novartis.com.

Novartis is on Twitter. Sign up to follow @Novartis at http://twitter.com/novartis.

References
[1] SigniforR (pasireotide) Summary of Product Characteristics. Basel, Switzerland: Novartis; April 2012.
[2] National Endocrine and Metabolic Diseases Information Service. US National Institutes of Health. Cushing’s Syndrome. Available at:http://endocrine.niddk.nih.gov/pubs/cushings/Cushings_Syndrome_FS.pdf. Accessed March 2012.
[3] Pedroncelli, A. Medical Treatment of Cushing’s Disease: Somatostatin Analogues and Pasireotide. Neuroendocrinology. 2010;92(suppl1):120-124.
[4] Colao, A. A 12-Month Phase III Study of Pasireotide in Cushing’s Disease. New Engl J Med. 2012; 366:32-42.
[5] Tritos N., Biller, B. Advances in Medical Therapies for Cushing’s Syndrome. Discovery Medicine. 2012:13(69):171-179.
[6] Newell-Price, J., et al. The Diagnosis and Differential Diagnosis of Cushing’s Syndrome and Pseudo-Cushing’s States. Endocrine Reviews.1998;19(5):647-672.
[7] Bertanga, X., et al. Cushing’s Disease. Best Practice & Research Clinical Endocrinology & Metabolism. 2009;23:607-623.
[8] European Commission. The Centralised Procedure. Available at:http://ec.europa.eu/health/authorisation-procedures-centralised_en.htm. Accessed March 2012.
[9] European Medicines Agency. Committee for Orphan Medicinal Products. Public Summary of Positive Opinion for Orphan Designation of Pasireotide for the treatment of Cushing’s Disease. Available at:http://www.ema.europa.eu/docs/en_GB/document_library/
Orphan_designation/2009/10/WC500006176.pdf
. Accessed March 2012.
[10] Lindholm, J., et al. Incidence and Late Prognosis of Cushing’s Syndrome: A Population-Based Study. J Clin Endocrinol Metab. 2001;86(1):117-23.
[11] US National Institutes of Health. Efficacy and Safety of Pasireotide Administered Monthly in Patients With Cushing’s Disease. Available at: http://clinicaltrials.gov/ct2/show/NCT01374906. Accessed March 2012.

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Media release (PDF)

From http://www.reuters.com/article/2012/04/25/idUS52612+25-Apr-2012+HUG20120425

Day Twenty-Five, Cushing’s Awareness Challenge

In case you haven’t guessed it, my cause seems to be Cushing’s Awareness.  I never really decided to devote a good portion of my life to Cushing’s, it just fell into my lap, so to speak – or my laptop.

I had been going along, raising my son, keeping the home-fires burning,  trying to forget all about Cushing’s.  My surgery had been a success, I was in remission, some of the symptoms were still with me but they were more of an annoyance than anything.

I started being a little active online, especially on AOL.  At this time, I started going through real-menopause, not the fake one I had gone through with Cushing’s.  Surprisingly, AOL had a group for Cushing’s people but it wasn’t very active.

What was active, though, was a group called Power Surge (as in I’m not having a hot flash, I’m having a Power Surge).  I became more and more active in that group, helping out where I could, posting a few links here and there.

Around this time I decided to go back to college to get a degree in computer programming but I also wanted a basic website for my piano studio.  I filled out a form on Power Surge to request a quote for building one.  I was very surprised when Power Surge founder/webmaster  Alice AKA Dearest called me.  I was so nervous.  I’m not a good phone person under the best of circumstances and here she was, calling me!

I had to go to my computer class but I said I’d call when I got back.  Alice showed me how to do some basic web stuff and I was off.  As these things go, the O’Connor Music Studio page grew and grew…  And so did the friendship between Alice and me.  Alice turned out to be the sister I never had, most likely better than any sister I could have had.

In July of 2000, Alice and I were wondering why there weren’t many support groups online (OR off!) for Cushing’s. This thought percolated through my mind for a few hours and I realized that maybe this was my calling. Maybe I should be the one to start a network of support for other “Cushies” to help them empower themselves.

I wanted to educate others about the awful disease that took doctors years of my life to diagnose and treat – even after I gave them the information to diagnose me. I didn’t want anyone else to suffer for years like I did. I wanted doctors to pay more attention to Cushing’s disease.

The first website (http://www.cushings-help.com) went “live” July 21, 2000. It was just a single page of information. The message boards began September 30, 2000 with a simple message board which then led to a larger one, and a larger. Today, in 2012, we have over 8 thousand members. Some “rare disease”!

This is on the intro page of Cushing’s Help…

I would like to give abundant thanks Alice Lotto Stamm, founder of Power Surge, premier site for midlife women, for giving me the idea to start this site, encouraging me to learn HTML and web design, giving us the use of our first spiffy chatroom, as well as giving me the confidence that I could do this. Alice has helped so many women with Power Surge. I hope that I can emulate her to a smaller degree with this site.

Thanks so much for all your help and support, Alice!

Day Twenty-Four, Cushing’s Awareness Challenge

 

I first saw a similar image to this one with the saying Life. Be in it at a recreation center when my son was little.  At the time, it was “Duh, of course I’m in it”.

The original image was one a couple males, a couple females and a dog walking/running.  No folks in wheelchairs, no older folks and certainly no zebras.

It would be nice to have everyone out there walking or running but that’s not real life, at least in the Cushie world.  It’s been a long time since I’ve really been In My Life – maybe it’s time to get back.

A dear friend who has not one, but two forms of cancer is travelling throughout Europe for the first time after her husband’s death wrote:

Some final words before I turn in for the night. If there is a spark of desire within you to do something which is not contrary to God’s Holy Law, find a way to make it happen. All things are possible and blessings abound for those who love Him. Life is such an adventure. Don’t be a spectator – live every single moment for Him and with Him.

Somedays, it’s hard even getting up in the morning but I’m trying.  I’ve signed up for Water Aerobics for people with Arthritis and I’m actually going to class twice a week, I’ve applied for a new part-time job, bought plane tickets for a trip.

This is the one and only life I’ll ever have and I want to make the most of it!

 

The way it was.

Catherine doesn’t have Cushing’s, although she was tested for it.  Her blog post is part of the Cushing’s Awareness Challenge and it echoes how we often feel.  I actually posted about it in my Day Twenty-three post, as well.

Catherine said, in part:

So today I’d like to tell you about one of the very lowest moments in my struggle to figure out what was wrong with me.  Not because I particularly want to revisit it, but because I know that when I read of what other people (mainly women, mainly Cushing’s sufferers) had been through and survived, I felt inspired.  I felt that I was not alone and that the things I was experiencing could be overcome.

via The way it was..

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