Today’s New York Times ran a heartbreaking story by Amy Harmon about two cousins who developed melanoma. One was entered into a cancer clinical trial and received the investigational drug PLX4032. The other was ineligible for the trial, and therefore unable to access the experimental drug. Guess which cousin died? The article is one in… Continue reading Are Trials Necessary?
Tag: compassionate use
A Cure? "Compassionate Use" and Drug Regulation
Are government bureaucrats keeping dying patients from getting access to possibly life saving drugs? That’s one way to read Margaret Talbot’s story in the Sunday New York Times (“Fighting for a Last Chance at Life.” May 17, 2009). Talbot describes how mother Kathy Thompson sought access to an unlicensed therapy Iplex for her Amyotrophic Lateral Sclerosis… Continue reading A Cure? "Compassionate Use" and Drug Regulation
In Brugge / No Compassion (Part II)
Further to the therapeutic outlook on first-in-human studies at the Brugge meeting was Adrian Thrasher’s thoughtful presentation on his own X-SCID study at Great Ormand Street Hospital. Thrasher’s study was able to restore immune function in nearly all volunteers. Recently, however, his team reported a lymphoproliferative disorder like those seen in a very similar Paris… Continue reading In Brugge / No Compassion (Part II)
No Compassion
“They say compassion is a virtue….” so sang David Byrne of the Talking Heads. But what about “Compassionate Use?” This refers to the practice of giving terminally ill patients who are otherwise ineligible for early phase clinical trials access to investigational agents. At the Brugge ESGCT meeting, Finnish researcher Akseli Hemminki described providing 125 patients compassionate… Continue reading No Compassion