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

 
 
 

29 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

7 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 (NewCo) and slashing the workforce of 20,000 by 30 pe ...
Source: Business Times Singapore   Full article at: Business Times Singapore
 

 
Update
1

7 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 d ...
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

Spanish nurse cured of Ebola
Madrid, Oct 21 (Xinhua) Spanish nursing auxiliary Teresa Romero was pronounced cured of the Ebola virus disease according to World Health Organisation (WHO) guidelines after the results of a second test confirmed Tuesday that she no longer had the virus in her blood.
 
 
Clinical trials of Ebola vaccine start Nov 1 in Switzerland
Geneva, Oct 21 (IANS/EFE) The World Health Organisation (WHO) announced Tuesday that it will begin clinical trials Nov 1 in Switzerland of one of the two anti-Ebola vaccines currently under development.
 
 
Colombia to launch world's first floating gas liquefaction plant
Bogota, Oct 21 (IANS/EFE) The world's first floating gas liquefaction plant is due to begin operating next year off the coast of northern Colombia under a pact between Canada-based Pacific Rubiales Energy and Belgian shipbuilder Exmar.
 
 
Paralysed man walks again after pioneering cell therapy
London, Oct 21 (IANS/EFE) A paralysed man has been able to walk again after therapy that involved transplanting cells from his nasal cavity into his spinal cord, the BBC reported Tuesday.
 
 
 

Latest from the Network

Keira Knightley was 'a goody two-shoes'
Keira Knightley was ''a goody two-shoes'' when she was younger. The 29-year-old actress claims she was a ''boring'' well-behaved child and never skipped school because she didn't want to upset her parents or teachers....
Read more on Celebrity Balla
 
Kat Dennings is in love with Kim Kardashian
Kat Dennings thinks Kim Kardashian's butt is ''amazing.'' The '2 Broke Girls' actress was ''very nervous'' when the 'Keeping Up with the Kardashians' star filmed a cameo for the show's 80th episode and was too shy to...
Read more on Celebrity Balla
 
Taylor Swift doesn't 'need some guy' for inspiration
Taylor Swift is ''figuring out how to cope'' with life on her own. The 'Shake It Off' hitmaker hopes her upcoming album, '1989,' will prove she doesn't ''need some guy'' to help her ''make a great record'' or to feel...
Read more on Celebrity Balla
 
Sarah Hyland is 'great'
Sarah Hyland says she is ''great'' after obtaining a restraining order against her ex-boyfriend. The 'Modern Family' star is happy to be focusing on her career again after a judge recently granted her a three-year...
Read more on Celebrity Balla
 
India re-elected to UNHRC for two years
United Nations, Oct 21 (IANS) India was Tuesday re-elected to the United Nations Human Rights Council (UNHRC) for the term 2015-2017 in a keenly contested election in the UN General Assembly in New York. Other...
Read more on Politics Balla
 
Spanish nurse cured of Ebola
Madrid, Oct 21 (Xinhua) Spanish nursing auxiliary Teresa Romero was pronounced cured of the Ebola virus disease according to World Health Organisation (WHO) guidelines after the results of a second test confirmed...
Read more on Business Balla
 
Gurdwara closed in Belgium
Brussels, Oct 21 (IANS) The mayor of the town of Vilvoorde in Belgium, Hans Bonte, has ordered the closure of the local gurdwara, Guru Nanak Sahib, for one month. Belgian media Tuesday charged that the Sikh temple "...
Read more on Politics Balla
 
Air strikes kill 28 militants in Pakistan
Islamabad, Oct 21 (IANS) At least 28 militants were killed when the Pakistani army launched an air strike in the country's northwest tribal town of Datta Khel Tuesday, military said. In a statement, Inter Services...
Read more on Politics Balla
 
Report reveals large-scale torture in China's 'Black Jails'
Beijing, Oct 21 (IANS/EFE) The use of hotels and factories as clandestine prisons where thousands of people -- mostly women -- are tortured to prevent their complaints against the government from coming to light has...
Read more on Politics Balla
 
Clinical trials of Ebola vaccine start Nov 1 in Switzerland
Geneva, Oct 21 (IANS/EFE) The World Health Organisation (WHO) announced Tuesday that it will begin clinical trials Nov 1 in Switzerland of one of the two anti-Ebola vaccines currently under development. WHO Assistant...
Read more on Business Balla