It’s easy to forward an email, “Like” a Facebook page, or support a petition. Tugging heart strings is one of the quickest ways to make a message go viral. But what’s the weight of these actions? Can they help effect meaningful change?
Mikaela Knapp is 25 years old and suffering from aggressive metastatic kidney cancer. She’s been turned down from four clinical trials for a new cancer drug and might not have much time left. The FDA has compassionate use provisions designed to help patients like Mikaela to get access to trial drugs, but compassion is a highly subjective response.
Keith Knapp, Mikaela’s husband, has turned to Change.org to gather support. He’s a quarter of the way to his goal of getting half a million people behind him. The million dollar question now, is how this type of activism impacts upon Bristol-Myers Squibb, Merck and Genentech – the companies running the trials.
Mikaela was diagnosed in October 2013, two years into her marriage and while working in public relations, her husband tells me. The doctors tried a variety of different drugs with little success. When they did find a treatment that worked, it was only effective for a month or two before the cancer started to spread again.
For the past week, Mikaela has been in a hospital in Folsom, California, Keith says. Her cancer is not being treated with anything but she returns periodically to the hospital when her pain becomes unmanageable. Doctors up her dosage of pain medication, before tapering it off to manageable level and sending her back home.
“No one can tell you for sure how much time she has,” says Keith, who works freelance and spends his days in Folsom – where the two grew up – alongside his sick wife.
New breakthroughs represent Mikaela’s only real chance at fighting her cancer, which has spread quickly to her brain and lungs. Major pharmaceutical companies have begun experimenting with ‘anti-PD-1’ drugs that may hold her last chance for combatting the disease.
In overly simplified terms, PD-1 cancers have a genetic cloak over them that hides them from the immune system and prevents the tumor from being attacked. Between 20 and 40 percent of cancer patients have PD-1 tumors. Merck’s early trials saw large and lasting tumor reductions in late-scale melanoma and lung cancers. If Mikaela is in this group, her husband says, it gives her a chance to extend her life and keep fighting.
The problem is that not everybody can get these drugs. Keith Knapp says that Mikaela has been rejected for four clinical trials. They had even paid a deposit and booked flights to Seattle for one when the doctors called to say she wasn’t suitable.
The aggressiveness and severity of Mikaela’s cancer makes doctors uneasy about treating her, Keith says. She has a rare subtype of kidney cancer. It spread quickly and she’s on steroids to manage swelling in her brain. But to qualify for a trial you need to be off steroids for eight weeks which Mikaela’s doctors don’t recommend.
The severity of the cancer is part of the horror of Mikaela’s condition, but also represents an opportunity. Her cancer cells are rapidly dying off and multiplying, which makes them easier to treat.
‘Compassionate use’ of trial drugs is granted at the discretion of the individual programs, working in conjunction with an oncologist and permission from the FDA. It can be granted for expanded access to a group of people in a similar situation, or on a single patient basis, essentially becoming a new clinical trial of one person.
“The patient must have a serious or immediately life-threatening disease or condition and no comparable or satisfactory therapeutic alternatives,” the FDA has declared. Reading the FDA’s rules and listening to Mikaela Knapp’s situation, she would seem to meet the requirements entirely. The Knapps are working with oncologists from UCSF, Stanford and UC Davis, who say they just need a drug company to play ball. Now they’re hoping to invoke the power of the crowd.
These things, sadly, can go either way. A seven-year old was granted access to drugs for compassionate use last week, through a Change.org petition with less than 20,000 supporters. Last November a father of two died waiting for access to drugs.
Keith Knapp understands why his wife hasn’t been picked for these trials. The drug companies want to find people that they have the best chance of helping.
“I understand about standardizing your data set,” Keith says. “But I tell them I’ll waive everything, I’ll just keep her memory to myself.”
A UCSF oncologist is trying to get Mikaela into another clinical trial this week. But Keith says they’re running down to the end of the list of options, with the petition looking more like a last stand.
“I’m just really scared that I’m going to lose my wife and then a year later these drugs will get approved,” Keith says. “I don’t understand why things have to move so slowly and people have to die.”
Keith Knapp knows that there are hundreds of other people in his same situation. Discretion is limited. Demand is high. He’s looking for preferential treatment, a miracle to allow him a chance to keep hoping for other miracles.
Mikaela’s online army sits 130,000 strong. Now it’s up to Bristol-Myers Squibb, Merck and Genentech to decide whether online advocacy can have real world impact.