Preclinical Data for ALD1613 at ENDO 2016

Posted on April 4, 2016 by MaryO

Alder BioPharmaceuticals, Inc. (“Alder”) (NASDAQ:ALDR), today announced that preclinical data on ALD1613, its anti-adrenocorticotropic hormone (ACTH) antibody for the treatment of congenital adrenal hyperplasia (CAH) and Cushing’s disease, were presented today by Andrew L. Feldhaus, Ph.D., in a poster presentation at ENDO 2016, the Endocrine Society’s 98th Annual Meeting in Boston, Mass. The presentation entitled “A Novel Anti-ACTH Antibody (ALD1613) Neutralizes ACTH Activity and Reduces Glucocorticoids in Rats and Nonhuman Primates” was presented as a late-breaking abstract.

Key Points:

  • In vitro, ALD1613 inhibits ACTH-induced cortisol secretion in a mouse adrenal cell line.
  • ALD1613 administration in rats with artificially elevated ACTH and corticosterone levels resulted in a rapid and durable reduction of plasma corticosterone levels.
  • In non-human primates, ALD1613 demonstrated stable and durable reductions in plasma cortisol levels by >50%.

Quote:

Randall C. Schatzman, Ph.D., President and Chief Executive Officer of Alder, said, “Existing therapeutic options for patients with congenital adrenal hyperplasia and Cushing’s disease comprise treatments that provide limited disease control and involve significant side effects. We believe these limitations indicate a clear need for new therapies such as ALD1613, which targets ACTH to diminish the overproduction of cortisol. The data presented today demonstrate the capacity of ALD1613 to reduce corticosteroid levels in preclinical settings. We intend to use these studies as part of an IND filing that we plan to submit to the FDA in the second half of 2016.”

From https://globenewswire.com/news-release/2016/04/03/825231/0/en/Alder-Presents-Preclinical-Data-for-ALD1613-at-ENDO-2016.html

Filed under: Clinical trials, Cushing's, Treatments | Tagged: , , , , , , , , , , , , | Leave a comment »

Woman Shaves Head Before Wedding to Support Best Friend

Posted on November 24, 2015 by MaryO

(IOWA) WCMH– An Iowa woman shaved her head three months before her wedding to support her best friend who was diagnosed with Cushing’s disease.

 

Alycia Kuberski didn’t think about how her wedding photos were going to look, but instead focused on her best friend Gina Gregoire Helton.

Helton was diagnosed with Cushing’s disease in 2014. It’s a rare illness that was caused by a cancerous carcinoid tumor in her left lung. In January of 2015, doctor’s removed Helton’s left lung and found the cancer had spread to her lymph nodes.

Four months later she went through chemotherapy and radiation. The treatments caused Helton to lose her hair in clumps. She decided to take control of her body and throw a head shaving party.

Helton did not expect her best friend to shave her head right before her wedding.

gina 1
Alycia with Gina on her wedding day.

“It meant so much to me that my friend would be willing to shave her head for me, especially 3 months before her wedding. I tried to talk her out of it, but she insisted, stating ‘It’s just hair, it is nothing compared to what you have been through,’ Gina Gregoire Helton said.

Helton said this brave and selfless move helped get the word out about Cushing’s disease.

“Her shaving her head did bring awareness because people would ask and she was able to share my story and bring awareness to Cushing’s disease, which is something I am very passionate about,” Helton said.

Helton has been cancer and Cushing’s disease-free since August.

gina 2
Best friends Alicia and Gina.
gina helton
 Alicia and Gina before shaving their heads.
gina2
Bold is Beautiful! Gina’s shaving head party.

From http://nbc4i.com/2015/11/23/woman-shaves-head-before-wedding-to-support-best-friend/

Filed under: Cancer, Cushing's, Treatments | Tagged: , , , , | Leave a comment »

Pituicytoma and Cushing’s Disease in a 7-Year-Old Girl: A Mere Coincidence?

Posted on November 9, 2015 by MaryO

Paola Cambiaso, Donato Amodio, Emidio Procaccini, Daniela Longo, Stefania Galassi, Francesca Diomedi Camassei, Marco Cappa

Download PDF

Abstract

Pituicytoma is a tumor extremely rare in childhood, with only 4 cases reported in literature. It is thought to arise from the specialized glial elements called “pituicytes.” The association of pituicytoma and Cushing’s disease (CD) has been described only once so far, in an adult patient.

A 7-year-old girl was referred for clinical signs of hypercortisolism, and a diagnosis of CD was made. MRI revealed 2 pathologic areas in the pituitary gland. The patient underwent surgery, with microscopic transsphenoidal approach, and a well-circumscribed area of pathologic tissue was identified and removed. Surprisingly, histologic and immunohistochemical study provided unequivocal evidence of pituicytoma. No pituitary adenoma could be identified.

For persistent hypercortisolism, the patient necessitated transsphenoidal endoscopic reintervention and 2 other lesions were removed. By immunohistological examination, these lesions were confirmed to be corticotropin-secreting adenoma. Unfortunately, there was no postoperative decrease in corticotropin and cortisol levels, and the patient underwent bilateral laparoscopic adrenalectomy.

Considering that we report a second case of association of pituicytoma and corticotropin-secreting adenoma, that CD is infrequent, and pituicytoma is extremely rare in childhood, the coexistence of these 2 tumors should not be considered a mere coincidence. To date, there is no conclusive evidence about the origin of these different subtypes of pituitary tumors. This case supports the hypothesis that these tumors share a common progenitor cell, which could be the folliculostellate cell.

View Full Text

Filed under: Cushing's, Rare Diseases, Treatments | Tagged: , , , , , , , , , , , , , , | Leave a comment »

Low Immediate Postoperative Serum-Cortisol Nadir Predicts The Short-Term, But Not Long-Term, Remission After Pituitary Surgery For Cushing’s Disease

Posted on October 26, 2015 by MaryO

Cushing’s disease is an ACTH-producing pituitary adenoma, and the primary treatment is microscopic or endoscopic transsphenoidal selective adenectomy. The aims of the present study were to evaluate whether the early postoperative S-cortisol level can serve as a prognostic marker for short- and long-term remission, and retrospectively review our own short and long term results after surgery for Cushing’s disease.

Methods: This single centre, retrospective study consists of 19 consecutive patients with Cushing’s disease who underwent transsphenoidal surgery.

S-cortisol was measured every 6 h after the operation without any glucocorticoid replacement. We have follow-up on all patients, with a mean follow-up of 68 months.

Results: At the three-month follow-up, 16 patients (84 %) were in remission; at 12 months, 18 (95 %) were in remission and at the final follow-up (mean 68 months), 13 (68 %) were in remission.

Five-years recurrence rate was 26 %. The mean postoperative S-cortisol nadir was significantly lower in the group of patients in remission than in the non-remission group at 3 months, but there was no difference between those in long-term remission compared to those in long-term non-remission.

The optimal cut-off value for classifying 3-month remission was 74 nmol/l.

Conclusion: We achieved a 95 % 1-year remission rate with transsphenoidal surgery for Cushing’s disease in this series of consecutive patients. However, the 5-year recurrence rate was 26 %, showing the need for regular clinical and biochemical controls in this patient group.

The mean postoperative serum-cortisol nadir was significantly lower in patients in remission at 3 months compared to patients not in remission at 3 months, but a low postoperative S-cortisol did not predict long-term remission.

Author: Jon Ramm-Pettersen Helene Halvorsen Johan EvangPål Rønning Per Hol Jens Bollers levJon Berg-Johnsen Eirik Helseth
Credits/Source: BMC Endocrine Disorders 2015, 15:62

Published on: 2015-10-26

Copyright by the authors listed above – made available via BioMedCentral (Open Access). Please make sure to read our disclaimer prior to contacting 7thSpace Interactive. To contact our editors, visit our online helpdesk. If you wish submit your own press release, click here.
From http://7thspace.com/headlines/519336/low_immediate_postoperative_serum_cortisol_nadir_predicts_the_short_term_but_not_long_term_remission_after_pituitary_surgery_for_cushings_disease.html

Filed under: Cushing's, Diagnostic Testing, pituitary, Treatments | Tagged: , , , , , , , , | Leave a comment »

Global Cushing’s Syndrome Market Size 2015

Posted on October 19, 2015 by MaryO

Cushing’s as money makers for drug companies 😦

~~~

Steroidogenesis inhibitors were responsible for approximately 28% of total drug sales in the 6MM in 2013, equating to around $50m. As a consequence of this trend, GlobalData expects overall revenues generated by this drug class to increase by approximately 390% to reach around $247m, encompassing 49% of total drug sales in the 6MM in 2018.

The expansion in this segment of the CS market is fuelled by the introduction of premium-priced pharmacological agents such as Novartis’ LCI699 and Cortendo AB’s NormoCort (COR-003) in the US, as well as the arrival of HRA Pharma’s Ketoconazole HRA (ketoconazole) to the European CS stage. One of the greatest unmet needs in this indication is a lack of effective drugs directed against the underlying cause of Cushing’s disease (the pituitary tumor).

Despite this demand, pharmaceutical companies are continuing to adopt a strategy that simply targets the adrenal glands. As a result, there is a vast amount of room for new or existing players to penetrate the market and capture considerable patient share.

Highlights

Key Questions Answered

Although the current standard of care (ketoconazole) is cheap and reasonably effective in most CS patients, it possesses worrying safety profiles, inconvenient dosing schedules, is difficult to obtain and can display waning efficacy over time. Newer medical treatments, for example, Novartis’ Signifor (pasireotide) and Corcept Therapeutics’ Korlym (mifepristone) address only some of these issues; yet, present their own limitations. The CS market is still marked by the existence of a multitude of unmet needs. What are the main unmet needs in this market? Will the drugs under development fulfil the unmet needs of the CS market?

The late-stage CS pipeline is sparsely populated; however, those drugs in development will be a strong driver of CS market growth. Which of these drugs will attain high sales revenues during 2013-2018? Which of these drugs will have the highest peak sales at the highest CAGR, and why?

Key Findings

One of the main drivers influencing growth in the Cushing’s syndrome market will be the introduction of second-generation steroidogenesis inhibitors, LCI699 and NormoCort (COR-003), in the US, which will rival existing standard of care medical treatments.

Another strong driver will be the arrival of Corcept Therapeutics’ Korlym (mifepristone) and HRA Pharma’s Ketoconazole HRA (ketoconazole) to the European CS market. Both drugs will stimulate significant growth here.

The launch of Novartis’ Signifor LAR (pasireotide) in the 6MM will equip physicians with a less frequently administered formulation of Signifor.

Reasons for inadequate CS treatment include poor physician awareness of the condition, delayed diagnosis, a lack of efficacious drugs for individuals suffering from severe hypersecretion, and a shortage of effective medicines targeting the source of Cushing’s disease.

Scope

Overview of Cushing’s syndrome, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and treatment guidelines.

Annualized Cushing’s syndrome therapeutics market revenues, annual cost of therapies and treatment usage pattern data from 2013 and forecast for five years to 2018.

Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the Cushing’s syndrome therapeutics market.

Pipeline analysis: comprehensive data split across different phases, emerging novel trends under development, and detailed analysis of late-stage pipeline drugs.

Analysis of the current and future market competition in the global Cushing’s syndrome therapeutics market. Insightful review of the key industry drivers, restraints and challenges. Each trend is independently researched to provide qualitative analysis of its implications.

Reasons to buy

Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline. Additionally a list of acquisition targets included in the pipeline product company list.

Develop business strategies by understanding the trends shaping and driving the Cushing’s syndrome therapeutics market.

Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the Cushing’s syndrome therapeutics market in the future.

Formulate effective sales and marketing strategies by understanding the competitive landscape and by analysing the performance of various competitors.

Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.

Track drug sales in the 6MM Cushing’s syndrome therapeutics market from 2013-2018.

Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.

From http://www.medgadget.com/2015/10/global-cushings-syndrome-market-size-2015-share-trend-analysis-price-research-report-forecast.html

Filed under: adrenal, Cushing's, podcast, Treatments | Tagged: , , , , , , , , , , , , , , , , , | Leave a comment »

« Previous Page — Next Page »