360° Coverage : Husband Seeks Compassionate Use Of Anti-PD-1 Drug For Wife With...

2 Updates
Husband Seeks Compassionate Use Of Anti-PD-1 Drug For Wife With Rare Kidney Cancer
Photo Credit: Forbes Business

Husband Seeks Compassionate Use Of Anti-PD-1 Drug For Wife With Rare Kidney Cancer

Mar 26 2014, 12:29pm CDT | by

Mikaela Knapp is a 25-year-old woman from Calfornia’s Sacramento Valley suffering with a rare subtype of kidney cancer who has exhausted all possible, FDA-approved drugs to treat her metastatic...

Filed under: news

 
 
 

25 weeks ago

Husband Seeks Compassionate Use Of Anti-PD-1 Drug For Wife With Rare Kidney Cancer

Mar 26 2014, 12:29pm CDT | by

Mikaela Knapp is a 25-year-old woman from Calfornia’s Sacramento Valley suffering with a rare subtype of kidney cancer who has exhausted all possible, FDA-approved drugs to treat her metastatic disease. Her husband, Keith, and their friends and family have mounted what has become a viral campaign on social and traditional media to help her gain compassionate use access to one of several antibody-based drugs that unmask cancer cells hiding from the immune system.

What began a year ago as a painful lump on her rib was diagnosed last October as Stage IV metastatic Xp11 translocation renal cell carcinoma, a long name for a rare subtype of kidney cancer that is most often seen in children.

Keith told me yesterday that the cancer was originally thought to be a non-Hodgkin’s lymphoma. “Okay, we can do this,” he said, reflecting on that cancer’s relatively high cure rate.

“But when we learned what it really was after a biopsy, we read up and saw that all the numbers were turned around,” meaning that the high survival percentages for non-Hodgkin’s were now the same for poor outcomes with metastatic RCC. Indeed, Mikaela’s disease had spread not only to her bones but her brain as well.

Keith and Mikaela are high school sweethearts – they first met in sixth grade – and graduates of Stanford University who married in 2011. Photos of Mikaela posted on their website, She Got This, show a vibrant young woman and commenters who know her describe her as beautiful, inside and out. The couple applied this energy to her care and she spent six months at the University of California at San Francisco’s Medical Center.

Keith’s description of her treatment regimen would take me at least a lecture or two to cover in my cancer pharmacology classes: Pfizer’s sunitinib (Sutent®), the multi-tyrosine kinase inhibitor originally developed by Sugen, and a more recently-approved antiangiogenic drug targeting the tyrosine kinase activity of the three VEGF receptor subtypes, axitinib (Inlyta®); Novartis’ mTOR inhibitor, everolimus (Afinitor®) and Wyeth’s similarly-acting drug temsirolimus (Toricel®).

And that’s just the pharmacology.

Mikaela has also received gamma knife treatment for her brain metastases as well as whole brain radiation, plus spinal radiation.

They’ve pulled out all the stops yet Mikaela still has up to 20 distinct brain metastases that have raised bleeding concerns as well as bone and lung metastases, the latter of which has compromised her pulmonary function to the point that she’s really just operating on one lung.

Yet she remains remarkably positive and upbeat as in the YouTube video from Monday thanking those people supporting her Change.org petition, change.org/SaveMyWife, now sitting at over 193,000 signatures after gaining over 60,000 yesterday alone.

What are Mikaela’s choices now?

At the outset, I said that Keith has been aggressively pursuing both clinical trials and compassionate use requests for three respective PD-1-targeted antibody therapeutics that are in Phase II or Phase III trials: nivolumab from Bristol-Myers Squibb, MPDL3280A from Roche/Genentech, and lambrolizumab from Merck. GlaxoSmithKline also has another PD-1 antibody in development that they licensed from AmplImmune, AMP-224, currently in a Phase 1 trials that’s listed as active, but not recruiting, in the study’s ClinicalTrials.gov entry.

Enrolling in a clinical trial has been difficult because Mikaela’s past treatment and need for corticosteroids to control brain swelling are criteria from exclusion from most Phase 2 and Phase 3 trials. While they continue to explore Phase 1 trials that tend to take all comers, Keith thought their time and effort was better spent by pursuing the compassionate use track.

By the time I caught up with Keith yesterday afternoon, they had already received a solid “No” from both BMS and Genentech despite the fact that a Stanford oncologist has agreed to serve as their oncologist for the experimental therapy. Merck seems to be more open and is currently conferring with the oncologist. In his year-end 2013 report card for pharmaceutical companies, Forbes senior editor Matthew Herper described Merck’s drug as a potential “breakout product.”

The Knapps were also fortunate yesterday to gain the support of their Congressman Ami Bera, a UC-Irvine-trained M.D. who represents California’s 7th district in the House of Representatives./>/>

The campaign being mounted by Mikaela’s friends and family is multifaceted and is taking advanatage of all possible angles. In addition to the Change.org petition and direct appeals to pharmaceutical companies for compassionate use, Mikaela is fortunate to have already made a name for herself in the northern California public relations community. Their website is well-constructed, complete with a media kit, and her PR colleagues have been aggressively contacting print and broadcast journalists.

In my case, Mikaela’s colleague, Ashley Thompson of Sutherland and Gold, alerted me to this story because she had seen my other compassionate use coverage of Josh Hardy, the Virginia boy with a secondary adenovirus infection who had been seeking brincidofovir from the small, North Carolina company, Chimerix. Instead of a compassionate use approach, Chimerix launched a 20-patient, open-label trial of brincidofovir in which Josh could enroll. He has already received two doses of the drug and his adenoviral genome counts have plummeted by 250-fold.

Both Keith and Ashley have told me that what they need is any help in identifying sources at any of the three companies that might help with the compassionate use access to one of the anti-PD-1/PD-L1 antibodies and general support through signatures at their Change.org petition. They’re also asking for supporters nationwide to contact local media to gain wider coverage of this story.

As we discussed with the Josh Hardy case, Mikaela is fortunate to have an incredibly motivated husband and the support of public relations professionals who know how to pitch to traditional and social media outlets. True compassionate use reform will ultimately require that the FDA, companies, and patients, come to some manner of agreement over compassionate use access to experimental drugs that doesn’t require massive social media campaigns or doesn’t disproprotionately favor those with the means to mount such a campaign.

In the meantime, my view as a pharmacologist who trained in a medical oncology division is that Merck really has little to lose by offering lambrolizumab to Mikaela. If it provides some degree of durable response in a case as advanced as hers, that will garner terrific media coverage (although I don’t know how success in one high-profile patient would influence FDA NDA reviewers). In the unfortunate situation where the drug might not help Mikaela, Merck could always hedge that her case was so serious that no drug could help her.

We’ll continue to follow this case and, in general, the effect of social media on compassionate use requests.

Source: Forbes Business

 
Update
2

3 weeks ago

Khazanah throws MAS RM6b lifeline

Aug 29 2014 5:01pm CDT | Source: Business Times Singapore

August 30, 2014 1:15 AMKHAZANAH Nasional will inject RM6 billion (SS$2.4 billion) over three years to resuscitate loss-making Malaysia Airlines (MAS) under a recovery plan that includes even an Act of Parliament. Other key moves are migrating its operations, assets and liabilities to a new company (N ...
Source: Business Times Singapore   Full article at: Business Times Singapore
 

 
Update
1

3 weeks ago

MAS posts loss of RM307m for Q2

Aug 28 2014 5:00pm CDT | Source: Business Times Singapore

August 29, 2014 1:13 AMMALAYSIA Airlines (MAS) registered a loss of RM307 million (S$122 million) for the second quarter to end-June, but warned of worse to come in the second half when the "full financial impact of the double tragedies of MH370 and MH17 ...
Source: Business Times Singapore   Full article at: Business Times Singapore
 

 

Don't miss ...

 

<a href="/latest_stories/all/all/30" rel="author">Forbes</a>
Forbes is among the most trusted resources for the world's business and investment leaders, providing them the uncompromising commentary, concise analysis, relevant tools and real-time reporting they need to succeed at work, profit from investing and have fun with the rewards of winning.

 

blog comments powered by Disqus

Latest stories

OPEC daily basket price closes tad lower
Vienna, Sep 23 (IANS/WAM) The basket of 12 crude oils of the Organization of Petroleum Exporting Countries (OPEC) closed at $94.37 a barrel Monday, compared to $94.44 a barrel last Friday, the OPEC Secretariat said.
 
 
Social exclusion prompts employees to cheat
New York, Sep 23 (IANS) Managers must see that none of the employees feels excluded as even perception of social exclusion may lead to employees to engage in seedy activities, which may ultimately affect the organisation, suggests a study.
 
 
Healthy lifestyle key to cut heart attack risk in men
London, Sep 23 (IANS) If the very thought of heart attacks makes you squirm, making a few amends in your lifestyle can help you breathe easy.
 
 
How to help shy kids learn
New York, Sep 23 (IANS) If your kids are shy - anxious, fearful, socially withdrawn, and isolated - it is more important to engage them in learning activities than trying to change them, says a study.
 
 
 

Latest from the Network

OPEC daily basket price closes tad lower
Vienna, Sep 23 (IANS/WAM) The basket of 12 crude oils of the Organization of Petroleum Exporting Countries (OPEC) closed at $94.37 a barrel Monday, compared to $94.44 a barrel last Friday, the OPEC Secretariat said....
Read more on Business Balla
 
Asiad swimming: Indians fail to qualify for finals
Incheon, Sep 23 (IANS) Indian men failed to qualify for the finals of two Asian Games swimming events they participated in at the Munhak Park Tae-hwan Aquatics Center here Tuesday. Sajan Prakash and Saurabh Sangvekar...
Read more on Sport Balla
 
US, allies unleash airstrikes against IS in Syria
Washington, Sep 23 (IANS/EFE) The US and allied nations Tuesday launched air attacks against the Islamic State (IS) Sunni extremist group in Syria, the US Department of Defence announced. "Forces of the United States...
Read more on Politics Balla
 
Asiad basketball: India go down to Philippines
Incheon, Sep 23 (IANS) The Indian men's basketball team lost their opening preliminary round Group E encounter to Philippines 76-85 in the 17th Asian Games at the Hwaseong Sports Complex Gymnasium Tuesday. The Indian...
Read more on Sport Balla
 
Kanye West visits college
Kanye West made a surprise appearance at a Los Angeles college on Friday (09.19.14). The 'Yeezus' singer spent around two hours speaking to 40 students from the digital communications department at LA Trade Technical...
Read more on Celebrity Balla
 
Simon Cowell congratulates Terri Seymour on pregnancy
Simon Cowell has congratulated his ex-girlfriend Terri Seymour on her pregnancy. The 39-year-old TV presenter announced yesterday (22.09.14) she is expecting her first child with model boyfriend Clark Mallon, 28, and...
Read more on Celebrity Balla
 
Miley Cyrus burglars to face imprisonment
The two burglars who broke into Miley Cyrus' home in June are to be imprisoned. Tylor Scott and Naomi Charles broke into the 'Wrecking Ball' hitmaker's property earlier in the summer, stealing her Maserati, purses,...
Read more on Celebrity Balla
 
Israeli Army kills suspected murderers of Jewish teens
Jerusalem, Sep 23 (IANS) Two Palestinians suspected of having kidnapped and murdered three Jewish teenagers in June have been killed in an exchange of fire with Israeli security forces in the West Bank early Tuesday,...
Read more on Politics Balla
 
Separatist teacher jailed for life in China
Beijing, Sep 23 (IANS) Ilham Tohti, a teacher belonging to the Uygur ethnic group, was Tuesdaysentenced to life in prison for separatism in Urumqi, capital of northwest China's Xinjiang Uygur Autonomous Region,...
Read more on Politics Balla
 
US informed Syria in advance of strikes against IS
Damascus, Sep 23 (IANS) Washington had informed Syria of airstrikes against positions of the Islamic State (IS) in Syria before these strikes started, the Syrian foreign ministry said in a statement Tuesday. The...
Read more on Politics Balla