Sunday, December 23, 2012

New Orphan Drug Approved for Cushing's


For Immediate Release: Dec. 14, 2012
Media Inquiries: Morgan Liscinsky, 
Consumer Inquiries: 888-INFO-FDA

FDA approves Signifor, a new orphan drug for Cushing's disease

The U.S. Food and Drug Administration today approved Signifor (pasireotide diaspartate) injection for the treatment of Cushing's disease patients who cannot be helped through surgery.

Cushing's disease is caused by over-production of cortisol, a hormone made by the adrenal glands. A tumor in the pituitary gland leads to overstimulation of the adrenal gland, which results in excess cortisol production. Cortisol regulates many important functions in the body, including response to stress and injury. Patients with Cushing's disease may have increased weight, glucose intolerance or diabetes, high blood pressure, easy bruising, and increased risk for infections.
"Although surgery tends to be first line therapy to treat Cushing's disease, Signifor is a new treatment option for patients when surgery hasn't worked or isn't an option," said Mary Parks, M.D., director of the Division of Metabolism and Endocrinology Products in the FDA's Center for Drug Evaluation and Research.

The safety and effectiveness of Signifor were evaluated in a clinical trial of 162 Cushing's disease patients. Trial participants were randomly chosen to receive one of two dose levels of Signifor over a six-month treatment period. Some patients who safely responded to the medication where allowed to continue treatment. Signifor resulted in decreased cortisol levels as measured in urine collected over a 24-hour period. This reduction was seen as early as one month after starting treatment. About 20 percent of patients in the clinical trial were able to reduce urine cortisol levels into the normal range.

Signifor caused increases in blood sugar levels, which could be detected as early as two weeks after starting treatment. Continued treatment caused or worsened diabetes in some patients; therefore, patients need to be carefully monitored for this side effect and be treated appropriately with anti-diabetic therapies, including insulin.

The FDA is requiring three postmarketing studies for Signifor: a clinical trial to assess high blood sugar (hyperglycemia) management; a long-term prospective observational cohort study (registry) of patients with Cushing's disease treated with Signifor; and focused safety monitoring for reports of serious hyperglycemia, acute liver injury, and adrenal insufficiency.

Signifor is administered under the skin (subcutaneously) twice daily, and will be dispensed with a Medication Guide, including instructions for patients and caregivers that describe the risks and adverse reactions people should be mindful of when using the product.

The most common adverse reactions observed in the clinical trial included hyperglycemia, diarrhea, nausea, abdominal pain, and gallstones.

Signifor is manufactured by Novartis Pharma Stein AG, Stein, Switzerland.

For more information:

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.



See for more information.

Friday, December 21, 2012

Spotlight on Cushing's: Lori on Dr Oz

Be sure to watch Lori's heartbreaking 
and informative segments 
on the Dr Oz show.

Thank you Dr Oz!

Out of Control Obesity: 
One Woman's Struggle with 
Cushing's Disease

Thursday, December 20, 2012

My Friend Lori Featured in News

Doctors, Patient From Ohio State Wexner Medical Center To Be Featured On 'Dr. Oz' Show

Wednesday December 19, 2012 5:39 PM

COLUMBUS, Ohio - An innovative operation conducted at The Ohio State University Wexner Medical Center will be featured on "The Doctor Oz Show."
Doctors used the operation to treat Cushing's Disease, an illness Lori Genser Burkhoff has suffered from for most of her life.

Cushing's Disease is caused by a tumor on the pituitary gland, the master gland of the body. Burkhoff was diagnosed when she was 14, when she gained 60lbs in a few months.

"The shape of my face was sort of as if somebody injected air into it. I was weak. I was losing my hair. I was just falling apart," Burkhoff said.

Doctors operated on her pituitary gland three times, but the problems persisted.

"Having a fourth pituitary surgery is sort of unheard of in the medical community, and there are very few doctors in the country who would even attempt to do this," Burkhoff said.
Then Burkhoff, a New York resident, heard of a new way to remove brain tumors, pioneered at the OSU Wexner Medical Center.

Instead of cutting open her head to reach the tumor and remove it, doctors went in through her nose. A brain surgeon worked through one nostril, while Dr. Ricardo Carrau, an ear nose and throat doctor, worked through the other.

"The advantage of doing is through the nose is you have an actual corridor. The nose has some air space. The sinuses have some air space," Carrau said. "So if you can connect the two, you can get to the pituitary gland."

Carrau said that avoiding incisions during surgery helps patients heal faster.

"They recover incredibly better than what they were doing before," Carrau said.

Burkhoff needed two surgeries. CBS' Dr. Oz was there to see for himself how it was done.

Now Burkhoff said she plans to come home to her husband and daughter.
"I'm hoping for a real start to 'Act Two,'" she said.

Doctors removed Lori's entire pituitary gland and part of the nearby bone to avoid a return of Cushing's Disease. Since the surgery, Lori has lost 30 pounds.

The Dr. Oz Show will have Lori's full story Thursday at 4 p.m. on 10TV.
©2012 by All rights reserved. 

Tuesday, December 18, 2012

Cushing's to be Featured in National News

In September 2012, Dr. Oz from the Dr Oz Show joined my dear friend Lori and her amazing surgical team for her 4th (and ultimately 5th) brain surgery in Columbus, Ohio.  Lori has been battling Cushing's for 20 years.  

Please tune in to/ DVR this network television show this Thursday, December 20 to learn more about Cushing's. Check your local listings.

(Show lists the topics as sextuplets and Marlo Thomas/ cancer).

From the Dr Oz Show:
"Can you imagine gaining so much weight, despite a healthy lifestyle, that it would eventually lead you to die from obesity?  It's a real illness, and it's called Cushing's Disease.  Dr. Oz met Lori, a woman desperate to survive this devastating and rare condition, and he joined her in the operating room as she underwent the dangerous surgery that could end her life or improve it forever. Dr. Oz shares his journey and he introduces Dr. Daniel Prevedello from Ohio State University Medical Center who performed Lori's ground-breaking procedure."

Lori has also created a web site to help share her story. 

I hope you will be able to watch. This is a big day in Cushie World.


Sunday, December 9, 2012

Australian Pituitary Foundation

Our friends in Hobart, Tasmania, Australia gathered to discuss the pituitary gland today.

Click here for more program details.

Images courtesy of Australian Pituitary Foundation. 
All rights reserved.

Sunday, December 2, 2012

Therapy of Adrenal Insufficiency

This new article from our friends in Italy is a comprehensive yet easy-to-read look at adrenal insufficiency. I find this information so valuable in understanding the mechanics of sufficient and timely cortisol replacement for Cushies post-op pituitary surgery or, for me, combined with nighttime ketaconazole as a medical therapy for persistent Cushing's disease. I applaud the authors for their wondrous graphs and tables highlighting the most important aspects of cortisol control. In particular, my heart sang when reading about the future of cortisol replacement section. Hope is on the way.


Therapy of adrenal insufficiency: an update


Adrenal insufficiency may be caused by the destruction or altered function of the adrenal gland with a primary deficit in cortisol secretion (primary adrenal insufficiency) or by hypothalamic-pituitary pathologies determining a deficit of ACTH (secondary adrenal insufficiency). The clinical picture is determined by the glucocorticoid deficit, which may in some conditions be accompanied by a deficit of mineralcorticoids and adrenal androgens. The substitutive treatment is aimed at reducing the signs and symptoms of the disease as well as at preventing the development of an addisonian crisis, a clinical emergency characterized by hypovolemic shock. The oral substitutive treatment should attempt at mimicking the normal circadian profile of cortisol secretion, by using the lower possible doses able to guarantee an adequate quality of life to patients. The currently available hydrocortisone or cortisone acetate preparations do not allow an accurate reproduction of the physiological secretion pattern of cortisol. A novel dual-release formulation of hydrocortisone, recently approved by EMEA, represents an advancement in the optimization of the clinical management of patients with adrenal insufficiency. Future clinical trials of immunomodulation or immunoprevention will test the possibility to delay (or prevent) the autoimmune destruction of the adrenal gland in autoimmune Addison's disease.

View the complete article in .pdf form here:

or view HTML: