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 petition,, 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 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 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 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


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