A Dying Mom, A YouTube Plea & Genentech

In a video posted on YouTube two weeks ago, Diane Gant, 46, says that she is dying of Stage 4 breast cancer. Much like a scene from a film called 'My Life Without Me,' in which a dying mom tape records messages for her young daughters to offer advice and hope for each of their successive birthdays, Gant holds up letters to her young son in order to touch base with him when he graduates high school and college, and marries. She does not expect to be there in person.

And Gant also laments that a potentially life-saving Genentech drug is not yet available. "The most unfortunate part is that there is a very promising drug for me, pertuzumab, that the FDA is just sitting on since December and does not plan on releasing until June 2012. I am not expected to make it to June, my liver is engulfed with tumors and I am wasting away," she writes in a note accompanying the video.

The FDA, however, was not the issue. "Patients and healthcare providers seeking expanded access to unapproved drugs need to understand that drug companies must agree to provide these drugs to patients," Richard Pazdur, who heads the FDA Office of Oncology Drug Products, wrote in an FDA blog last week. The stumbling block was actually Genentech. And so, a few days and several thousand views later, the Roche unit blinked and agreed to make pertuzumab available to Gant based on compassionate use.

"Genentech is committed to a fair and impartial evaluation of each request for access to our investigational medicines, and takes these requests very seriously," a spokeswoman tells ABC News. "Appropriate decisions regarding potential access to investigational or unapproved medicines can only be made after in-depth discussions between Genentech clinical teams and the person's qualified treating doctor."

The episode underscores the debate over experimental treatments and the willingness of some patients to assume risks. Drugmakers "don't want a death attributed to their new drug," University of Pennsylvania bioethics professor Art Caplan tells ABC. "They may not have any idea how much drug to use and they often have a very limited supply on hand, which they hope to use in testing the drug not for one person's last desperate gasp at a bit more life."

"Every case is a huge maelstrom of hope, begging, corporate self-interest, slow bureaucratic due process, media spotlighting and public frustration," he continues. "It is simply very hard to solve access issues with a single policy given the many interests besides the patients that are in play in these cases. Each winds up being unique."

The Gant video, however, also illustrates how social media can change the equation. In years past, patients had to rely on a letter-writing campaign, a petition or, perhaps, a coveted connection with someone who had influence at a drugmaker in order to achieve the same result. By posting her video, though, Gant succeeded in breaking through barriers. Of course, there is also the possibility that such videos will become so commonplace that drugmakers will be unwilling to respond to each entreaty.

9 Comments

Apr 24, 2012 - 2:13pm
I wonder if the inevitable failure of pertuzumab to be the "miracle cure" this patient is hoping for will get as wide a distribution as the plea for the drug?
Andrea Apr 24, 2012 - 2:49pm
Doubtful that we shall ever hear the outcome...hopeful that she can be at peace in her final days with some quality of life left with her family
The novel monoclonal antibody pertuzumab functions by preventing dimerization of HER-2 (the target of Herceptin) with the other members of the human epidermal growth factor family HER-1, HER-3 and HER-4. In so doing, the cross talk between receptors is abrogated and downstream signaling is squelched. One of the most interesting aspect of the reports on this drug is it reflects the downstream pathways it targets. Pertuzumab inhibits signaling at the PI3K pathway, upstream from mTOR.

This, in the truest sense, is a breakthrough in metabolomics. There may be many reasons why cancer drugs remain out of reach for patients like this, but several changes could be implemented immediately to increase the rate of success of these new drugs. One of them is the need to redouble the efforts in the study of basic metabolism and the growing field of metabolomics (the metabolome). For personalizing cancer therapy for patients, metabolic profiles are essential. She should have received "active" drugs, the first time around, and doctors should be allowed to give them, using techniques that match patients to "active" therapies.

Apr 24, 2012 - 6:05pm
"Of course, there is also the possibility that such videos will become so commonplace that drugmakers will be unwilling to respond to each entreaty."

Ed, I repeated this last statement for emphasis. I'll tell flat out you the kind of entreaties that wil get pharma's attention. I happened to be at my desk at Abbott on the day in 1984 when my department received a personal phone call from President Ronald Reagan. Mr Reagan was calling to request an emergency shipment of TRH to the late Sen Jacob Javits. Mr Javits was dying from ALS and Abbott was studying TRH for potential efficacy in ALS at that time. The shipment was prepared and shipped the same day.

I'm not saying you have to be the President of the United States to get our attention, but a homemade youtube clip from a regular citizen probably won't cut it.

Apr 25, 2012 - 12:45am
Two corrections:

1. Her name is Darlene, not Diane. 2. She posted her video on YouTube on April 16, not two weeks ago.

Apr 25, 2012 - 8:52am
At the risk of sounding hard-hearted, these individual pleas for last ditch therapies get kind of old. Stage 4 of any type of cancer is incurable and her oncologist should be a little more realistic about this. Part of dealing with the terminally ill is preparing them for the inevitable, not suggesting "we could try this" therapies. These are commonly referred to as "home-brew" regimens, and do little for the patient.

Original Insider: How did that THR therapy work out for Sen. Javits? I'm not aware of any useful drugs for ALS even today.

Apr 25, 2012 - 10:29am
Devilcat, Sen Javits lived another two years with ALS after we shipped the TRH. He died in 1986. Difficult to say whether the TRH prolonged his life and I don't know whether it was studied further. I left Abbott in 1985.
Apr 27, 2012 - 12:18am
@ Devilcat, regardless of Darlene's prognosis, I would hardly call a medication set to be released in June 2012 a "home brew remedy." Your tone leaves me to believe your motives are not pure, but, perhaps, financial. As in, fear of financing potential help to the terminally ill.
May 3, 2012 - 8:47pm
Pam told me about your video and how your cancer has progressed. Darlene you are a wonderful person and I do not want to say good bye so I will pray for you and your family. I am so sorry that you have had to go through this. It's not fair for anyone. You are a very compassionate person and I have faith that you will get the help you need to have more time with your family. You are an amazing women and I am so glad to have met you and look forward to seeing you again at a marketing meeting soon. Love ya Darlene