highly recommended NYT article

[Lani]

http://www.nytimes.com/2013/11/03/magazine/how-dying-patients-get-access-to-experimental-drugs.html?pagewanted=4&pagewanted=all

[Andrea Barnett Budin]

Big WOW on so many levels. Multiple Lessons to be learned here.

I recently signed a petition for a woman dying and asking for compassionate access to a drug. She'd been denied repeatedly.

And then, the petition seemed to tip the scales.

Tenacity cannot be understated.

I sincerely thank you for this article, Lani!

[jaykay]

Another wow, thanks Lani. But it reiterates the old saying "it's not what you know, it's who you know".

[Becky]

And the major point is staying alive long enough for the next magic bullet that just might be the one magic bullet for you.

['lizbeth]

AUY922 is currently in a Phase II clinical trial for Her2 breast cancer:


Combination of AUY922 With Trastuzumab in HER2+ Advanced Breast Cancer Patients Previously Treated With Trastuzumab

Crizotinib has since received an accelerated approval for locally advanced or Metastatic Lung Cancer.

FDA Approval for Crizotinib

Brand name: Xalkori®


Approved for locally advanced (http://www.cancer.gov/Common/PopUps/popDefinition.aspx?id=CDR0000045955&version=Patient&language=English) or metastatic (http://www.cancer.gov/Common/PopUps/popDefinition.aspx?id=CDR0000044058&version=Patient&language=English) non-small cell lung cancer (NSCLC) that is anaplastic lymphoma kinase-positive as detected by an FDA-approved test.

Full prescribing information (http://www.accessdata.fda.gov/drugsatfda_docs/label/2011/202570s000lbl.pdf) is available, including clinical trial information, safety, dosing, drug-drug interactions and contraindications.
On August 26, 2011, the Food and Drug Administration (FDA) granted accelerated approval to crizotinib (Xalkori Capsules, made by Pfizer, Inc.) for the treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) that is ALK-positive as detected by an FDA-approved test. The FDA approved the Vysis ALK Break-Apart FISH Probe Kit (Abbott Molecular, Inc.) concurrently with the crizotinib approval. This companion diagnostic test is designed to detect rearrangements of the anaplastic lymphoma kinase (ALK) gene in NSCLC.
The approval was based on two single-arm trials, Study A (N = 136 patients) and Study B (N = 119 patients). Crizotinib, 250 mg, was administered orally twice daily to a total of 255 patients with locally advanced or metastatic ALK-positive NSCLC. Demographic analysis from the combined data of these trials noted that the median (http://www.cancer.gov/Common/PopUps/popDefinition.aspx?id=CDR0000044941&version=Patient&language=English) age was 52 years, 63 percent of patients were Caucasian, 30 percent were Asian, 48 percent were male and 84 percent had an ECOG performance status (http://www.cancer.gov/Common/PopUps/popDefinition.aspx?id=CDR0000044174&version=Patient&language=English) of 0 or 1. Fewer than 3 percent of patients were current smokers. Ninety-six percent had adenocarcinoma (http://www.cancer.gov/Common/PopUps/popDefinition.aspx?id=CDR0000046216&version=Patient&language=English), 95 percent had metastatic disease, and 94 percent had received prior systemic treatment (http://www.cancer.gov/Common/PopUps/popDefinition.aspx?id=CDR0000045922&version=Patient&language=English) for NSCLC.
The primary endpoint (http://www.cancer.gov/Common/PopUps/popDefinition.aspx?id=CDR0000044163&version=Patient&language=English) of both trials was objective response (http://www.cancer.gov/Common/PopUps/popDefinition.aspx?id=CDR0000044067&version=Patient&language=English) rate (ORR) as assessed by the investigator. In Study A, the ORR was 50 percent (95 percent CI: 42 percent, 59 percent) with a median response duration of 42 weeks. In Study B, the ORR was 61 percent (95 percent CI: 52 percent, 70 percent) with a median response duration of 48 weeks. Complete responses (http://www.cancer.gov/Common/PopUps/popDefinition.aspx?id=CDR0000045652&version=Patient&language=English) were observed in 1 percent of patients. No differences in ORR by performance status, number of prior chemotherapeutic regimens, or percentage of cells found to have the ALK gene rearrangement were noted.
The most common adverse reactions (at least 25 percent) observed in both studies were vision disorder, nausea, diarrhea, vomiting, edema, and constipation. Vision disorders included visual impairment, photopsia, vision blurred, vitreous floaters, photophobia (http://www.cancer.gov/Common/PopUps/popDefinition.aspx?id=CDR0000044077&version=Patient&language=English), and diplopia. Grade 3-4 adverse reactions in at least 4 percent of patients included increased ALT and neutropenia (http://www.cancer.gov/Common/PopUps/popDefinition.aspx?id=CDR0000045361&version=Patient&language=English). Crizotinib has been associated with severe, life-threatening, or fatal treatment-related pneumonitis (http://www.cancer.gov/Common/PopUps/popDefinition.aspx?id=CDR0000482345&version=Patient&language=English) with a frequency of 1.6 percent in clinical trials. All cases occurred within 2 months after the treatment initiation.
The recommended dose and schedule for crizotinib is 250 mg orally twice daily.

This summary was provided by Richard Pazdur, M.D., director of the FDA's Division of Oncology Drug Products.

[KsGal]

A big ole cyberhug for finding this and posting it. I really appreciate all the time and energy you put into research and postings here.
This was both inspiring and depressing at the same time. While I did take away from the article that attitude, faith and determination make a huge difference in outcomes, and that you don't have to give up when the doctor gives you a disheartening prognosis, I also took away that something like this is a one in a million situation. So that last part is pretty sad.
Very interesting to read, thank you again for sharing it with us.

[Andrea Barnett Budin]

https://scontent-a-mia.xx.fbcdn.net/hphotos-frc3/p480x480/1391803_172133426321529_93767887_n.jpg

TENACITY! I relate to this tree. Beyond stubborn. Persistence. Insistence. Determination.

Like a twig growing midst a concrete parking lot...

[suzan w]

That was an amazing article. I agree...with the 'it's who you know'...part, yet we are all such fighters here. Beating the odds, one day at a time!! Thank you for posting this, Lani!