The May 1, 2007, Wall Street Journal reported the case of Penelope London, a four-year-old suffering from a rare cancer. Penelope was diagnosed when she was just sixteen months old as having an aggressive form of neuroblastoma. Doctors gave her only a 25 percent chance of being cured. Three years later, she has undergone not just multiple bouts of the standard cancer treatments--chemotherapy, radiation, bone-marrow transplants, and surgery--but also experimental therapies that have been in progressively earlier stages of human testing. These therapies have yielded varying results for her, but have managed to keep her alive.
In November 2006, Penelope's cancer had recurred and no treatment was working. That was when her father heard of Neotropix Inc., a company in Malvern, Pennsylvania, that is developing an experimental cancer drug. The drug contains a live virus that normally strikes pigs. Experiments show that, when introduced into the human body, it may attack certain kinds of cancer cells. However, the drug is still in the earliest phase of safety testing and has only been given to six humans. The first patient died--from the cancer, not the drug--but the Federal Drug Administration put the trial on hold to definitively determine this. Neotropix expects the resumed first-stage testing to last another eighteen months.
Yet Penelope is running out of time. The Wall Street Journal described her as dying and in great discomfort--she was admitted to New York University Medical Center because her pain medication wasn't working. The Londons, desperate to try anything that would help their daughter, asked Neotropix to make the experimental drug available to her. On April 18, after many board meetings and a consultation with an ethicist, Neotropix refused. Besides being nervous about the substantial risks to Penelope, chief executive Peter Lanciano was concerned that the FDA would put the drug's trial on hold again if she died after receiving it, despite some indications from the FDA that it would not. If the trial were again delayed, the drug would take longer to reach others who need it--in effect sacrificing future patients to help Penelope now.
Neotropix is also concerned about itself. The small, start-up business is only a few years old and has limited funds. The bad press if the treatment did more harm than good for Penelope could sink the company, meaning that no future patients would ever benefit from this drug or any others it might develop.
In the article, P. Sherrill Neff--managing partner in Quaker BioVentures, a venture-capital firm with a substantial investment in Neotropix--claimed that, in this situation, "There is no right answer." Nevertheless, the company had to reach a decision. Was it the right one? Or should Neotropix have given Penelope London their experimental drug?
By Peter C. Adamson
Despite advances in the treatment of childhood cancer over the past fifty years, therapies remain too toxic, and one of every five children still dies from the disease. Over the past decade, our ability to improve upon these cure rates has waned. For most children with cancer, if initial therapy fails and the cancer relapses, cure becomes very unlikely. The overall timeline for new drug discovery and development remains long, and in the increasingly risk-averse environment of drug development, often absent from the discussion is the possibility of a child dying from underlying cancer while clinical trials move forward and access to new experimental agents is limited. For the family of a child with recurrent cancer, these realities result in a fabric of hope, fear, and frustration.
The rapid advances we have made in our understanding of the biology of cancer have heightened expectations of more effective and less toxic treatments. But we must keep in mind that four of five new anticancer agents entering the clinical development pipeline fail to demonstrate meaningful benefit, and for the one in five ultimately approved by the FDA, survival benefit is usually measured in weeks or months. …