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

Mar 26 2014, 12:29pm CDT | by

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

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

 
 

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

Soyuz rocket carrying two Galileo satellites lifts off
Brussels, Aug 22 (IANS) A Soyuz rocket carrying two Galileo satellites, the fifth and sixth of Europe's Galileo global satellite navigation system, lifted off from the spaceport in French Guiana Friday, the European Space Agency (ESA) has announced.
 
 
Fresh cases of Ebola recorded in Nigeria: Official
Abuja, Aug 22 (IANS) Fresh cases of the Ebola virus disease have been recorded in Nigeria, where the highly contagious disease has already claimed five lives, Minister of Health Onyebuchi Chukwu said Friday.
 
 
Fuel cell technology gets a boost
Washington, Aug 22 (IANS) In an impetus for fuel cell technology to build "smart" cars, scientists at Stanford University have developed a low-cost, emissions-free device that uses an ordinary AAA battery to produce hydrogen by water electrolysis.
 
 
3D-printed technology to make drug delivery better
Washington, Aug 22 (IANS) The US researchers have developed an innovative method for using affordable, consumer-grade 3D printers and materials to fabricate custom medical implants that can contain antibacterial and chemotherapeutic compounds for targeted drug delivery.
 
 
 

Latest from the Network

Looking forward to working with Modi government: Singapore PM
Singapore, Aug 22 (IANS) Singapore Prime Minister Lee Hsien Loong Friday said his country hopes to work with India in areas of infrastructure and education, and expressed his desire to meet his Indian counterpart...
Read more on Politics Balla
 
India beat Middlesex by 95 runs
London, Aug 22 (IANS) India returned to winning ways albeit in a warm-up cricket match, registering a comfortable 95-run win over Middlesex at Lord's here Friday. India were dismantled 3-1 in the Test series,...
Read more on Sport Balla
 
Pakistan: Tensions ease as PTI agrees to resume talks (Roundup)
Islamabad, Aug 22 (IANS) Tensions in the Pakistani political landscape eased Friday with the Imran Khan-led Pakistan Tehreek-i-Insaf (PTI) party agreeing to return to the negotiating table as the Senate or the upper...
Read more on Politics Balla
 
Ukrainian rebels kill four soldiers
Kiev, Aug 22 (IANS) At least four security personnel have been killed and 23 wounded in clashes with rebels in eastern Ukraine, authorities said Friday. Four Ukrainian servicemen were killed Thursday overnight in...
Read more on Politics Balla
 
Miley Cyrus concert banned in Dominican Republic
Santo Domingo, Aug 22 (IANS/EFE) The Dominican Republic government has banned a concert where US pop star Miley Cyrus was scheduled to perform in the nation's capital Sep 13. In a notice sent to the SD Concerts and...
Read more on Celebrity Balla
 
George Clooney's ex Elisabetta Canalis engaged
George Clooney's ex Elisabetta Canalis has got engaged. The 35-year-old beauty is set to tie the knot with Brian Perri in Italy next month, as is George and his new fiancée Amal Alamuddin, after the orthopedic surgeon...
Read more on Celebrity Balla
 
Mariah Carey's jealously to blame for split?
Mariah Carey's jealousy is reportedly partly to blame for her and Nick Cannon's split. The 44-year-old singer is said to have trust issues with her spouse - with whom she has four-year-old twins Moroccan and Monroe -...
Read more on Celebrity Balla
 
Jessica Alba is 'fearless' after motherhood
Jessica Alba has become ''fearless'' since motherhood. The 33-year-old actress admits her whole perspective of her career has changed since welcoming her daughter Honor, six, and Haven, three, into the world with her...
Read more on Celebrity Balla
 
Daniel Radcliffe wants fans to forget Harry Potter
Daniel Radcliffe wants his fans to forget about 'Harry Potter'. The 25-year-old actor is currently starring in romantic comedy 'What If', and despite its success, the actor is resigned to the fact the comparisons to his...
Read more on Celebrity Balla
 
Hamas executes 18 alleged collaborators with Israel
Gaza, Aug 22 (IANS/EFE) Palestinian militias Friday executed 18 people accused of collaborating with Israel, 48 hours after three top military leaders of the Islamic Palestinian movement Hamas were killed in an Israeli...
Read more on Politics Balla