Pediatric Recurrent Respiratory Papillomatosis: Fighting the Battle of a Rare But Serious Disease

While setting up for a prospective randomized controlled trial, the company that initially produced HspE7 and supported the open-label trial ran out of funds. Fortunately, the company was recently purchased and the new owners, Nventa, are working to start trials where the former company left off, although no launch date for the larger trial has been announced.

As demonstrated by the HspE7 and celecoxib trial delays, one of the biggest challenges facing RRP researchers is obtaining funding for work on a rare disease. It is an orphan disease, said Dr. Wiatrak. It is extremely difficult to get funding for large trials. Additionally, because the disease is rare, recruiting an adequate number of patients demands that studies be run at multiple institutions, which increases the cost of a trial. The problem with papillomas is that you can’t do a study, a good prospective trial, at one institution. There are just not enough patients. There may not be enough patients in the entire country. We have looked at the possibility of doing international studies to have enough patients to accrue into the study to have worthwhile data, he said.

The Big Hope: HPV Vaccine

Experts agree that the most important weapon in the fight against RRP is likely to be the newly approved vaccine that protects against HPV infection. (For more about HPV vaccines, see the editorial on page 3.) Dr. Derkay compares the potential impact of this vaccine to the dramatic drop in acute epiglottitis following the introduction of the haemophilus influenzae type B (HiB) vaccine. When I was a resident in the 1980s, one of the most common ENT emergencies was epiglottitis resulting in acute airway distress, with one or two cases each week in big medical centers, said Dr. Derkay. The introduction of vaccine virtually wiped it out. Residents in my program have not seen a case of this.

However, the vaccine approach is not without challenges. Both the Merck vaccine, Gardasil, which has been approved by the FDA, and the GlaxoSmithKline vaccine, which is still in clinical trials, appear to block HPV infection. Yet only Gardasil is intended to protect against the HPV types that cause RRP. Gardasil is a quadrivalent vaccine designed to protect against infection of HPV-16 and -18, which are associated with cervical cancer, as well as against HPV-6 and -11. By contrast, the GlaxoSmithKline vaccine protects only against the cancer-associated types.

Therefore, in addition to the challenges that come with any vaccine, such as making sure the people who need it get it, otolaryngologists have another task in front of them: making sure that the most widely used vaccine is the right one.