Few health problems are as pressing or as devastating as Alzheimer’s disease, which turns family members into strangers and practically ensures a long, painful demise. By 2050, the number of Americans suffering from the disease is expected to reach 14 million, more than double the current figure of 5.8 million. The good news is that a cure may finally be in sight, but only if officials at the Food and Drug Administration (FDA) keep an open mind and pursue science-based policy. With permissive policies, the agency can finally allow treatments that could bring the devastating and costly disease ravaging America’s seniors to an end.
Researchers have put in countless hours parsing through the trials-and-errors of treating the disease. They may have finally succeeded in creating medications that remove the brain plaque likely behind the illness. Developed by Biogen, aducanumab works as an antibody therapy that removes amyloid plaques thought to induce memory loss and reduced cognition. The medication has had some mixed results and early evidence wasn’t nearly good enough for the FDA to justify approval. In March, Biogen discontinued its development of the medication after study results painted a disappointing picture for Alzheimer’s patients.
At the time, the drug producer was only examining data from patients that had completed drug trials in December 2018. But there was a cohort of patients that continued taking the drug until March 2019 and a recent reanalysis of the data including these longer-term patients found promising results. In the “EMERGE” trial, patients randomly assigned aducanumab had a 23 percent improvement in cognitive functioning (via the Clinical Dementia Rating–Sum of Boxes test) compared to non-takers. These results reinforce an earlier, smaller study, which found that half of the Alzheimer’s patients taking the maximum dosage of aducanumab for a year no longer tested positive for plaque. Now, these results should be taken with not just a grain, but a mountain, of salt. In the twin trial that happened alongside “EMERGE,” there was no discernible, positive result for Alzheimer’s patients.
But for Alzheimer’s sufferers and their families, even conflicting results are far better than nothing at all. It’s been 16 years since the FDA has approved a medication for Alzheimer’s disease (which has a zero percent survival rate). One might think that, in these cases, the agency might approve the drug though perhaps warn doctors about the medication’s conflicting evidence. But due to the FDA’s track-record and statistical standards, industry analysts would call such sentiments naïve. Baird analyst Brian Skorney argues, “The bottom line is, the FDA standard of approval is substantial evidence of efficacy and the cumulative data for aducanumab falls really far of this standard.” Skorney has a point, but the conclusion speaks more to the FDA’s regulatory rigidity than the rational case for aducanumab.
The FDA cares less about results (per say) and more about whether the results matched exactly what the drug maker predicted. A drug (i.e. Provenge for prostate cancer treatment) could have a powerful impact in reducing mortality, but perhaps the medication works via a different mechanism than the manufacturer thought or takes longer to have an effect on patients than supposed. In these cases, the FDA will ask the manufacturer to go back to the drawing board and restart the application process.
Or, in the case of AstraZeneca’s rejected PT010 application for treating Chronic Obstructive Pulmonary Disease (COPD), new evidence emerged after the application was submitted to the agency. In these cases, the FDA could and should be more open-minded in evaluating the evidence. And the agency has even less of an excuse to reject medications for complete killer diseases such as Alzheimer’s.
Time is running out for millions of patients and experimental medications with promising (albeit mixed) results are currently their only hope. Alzheimer’s and countless diseases can go the way of polio, but only with an accommodating regulatory system.