Do Clinical Trials Work?

As college professors, Alana and Morton Prescott could schedule their time around a daily ride in the countryside on their Draught horses, Cadillac and Caydence.

During a recent Friday ride, the couple reminisced about their cat, Matty, whom they had lost to leukemia just before her 16th birthday. About a year before she died, the Prestons had allowed their veterinarian, Dr. Pickens, to enroll Matty in a clinical trial of an FDA-approved human drug in an attempt to slow or stop the progression of her disease. Alana recalled that Matty had done well for about two months, but then everything went downhill. “We really were quite lucky,” sighed Morton, “because she died in her sleep and spared us a dreadful decision.” The couple rode the rest of the way home in silence, each lost in private memories of their beloved Burmese cat.

When they returned to their barn, Alana commented that Cadillac had not been his usual high-spirited self. After putting Cadillac in his stall, Morton checked his shoes. “I’m wondering if that new farrier had a problem putting on this front shoe, Morton wondered. “Didn’t it seem like he spent a lot of time with it?” Alana remembered that the farrier had mentioned that he thought Cadillac might be a bit lame in his right fetlock and that she should keep an eye on him. “Come to think of it,” Alana noted, “he did seem to stumble a bit on that muddy section at the bottom of the hill. Maybe we should call Dr. Pickens.”

As luck would have it, Dr. Pickens was not far from the Prescott’s farm when Morton reached him on his cell phone, and he offered to swing by on his way home. “I think you’re right, Alana,” Dr. Pickens said after taking him around the ring and then examining him. “He seems to have moderate pain during distal limb flexion. He’s also got prominent effusion in that right fetlock. Lameness was evident at the trot in a straight line and worsened considerably with flexion. There are a few ways we can treat it, but I’m really excited about a new clinical trial. Would you consider that?”

“Maybe but, after our experience with Matty, I’m not sure clinical trials work. Do you believe that clinical trials work?” asked Alana. Dr. Pickens rubbed his beard for a few moments and said, “Most clinical trials are successful, but not every participant outcome is successful.”

Dr. Pickens explained that he and other veterinarians often exhaust all available therapies in their efforts to arrest or cure a particular animal’s condition. At that point, the only remaining option may be an experimental therapy being tested in a clinical trial. “That was the situation with Matty,” Dr. Pickens reminded Alana. “Unfortunately for her, the study drug was not that effective. Incidentally, that drug never got approved.”

“Now, Cadillac is a horse of a different color, if you’ll pardon the pun.” Dr. Pickens then described the various options for treating Cadillac’s problem, and the likely timeframe and cost of each treatment. “If we enrolled Cadillac in the study, all of his treatment would be free. Plus, there’s an owner honorarium of $150 for participating.” Dr. Pickens then noted that study subjects were randomly assigned to treatment groups, so Cadillac could receive a placebo. That troubled Alana. “If he got the placebo, then what?” she asked. “If you’re not happy with his progress, you can remove him from the study at any time and switch to one of the current treatment options,” Dr. Pickens answered.

Alana still looked concerned. “What would you do if Cadillac were your horse? The poor baby’s only two. I never want him to be uncomfortable.”

“I’ve reviewed the safety profile of the study drug and the science behind it is sound. And, given what I’ve seen so far, I’d enroll him.” Alana looked at Morton for assurance. When he nodded, Alana said, “When can Cadillac start?” “I’ll come back on Monday morning with the consent form and other paperwork,” Dr. Pickens answered. “But, in the meantime, rest Cadillac for the weekend to decrease the chance of increased inflammation around that joint.”

This story has a happy ending because Cadillac was assigned to the active treatment group and the study drug was very effective at alleviating his lameness. Which means that Alana and Morton are back to their daily rides.

Clinical trials are not a guaranteed cure for every participant. However, they are the filter through which each new veterinary therapy must pass if it is to receive marketing approval. So, do clinical trials work? Absolutely!