The Food and Drug Administration issued new guides on drug development for neurological disorders. This sets the stage for possible treatments for Alzheimer's.
The disease-oriented development guide documents will provide details on how researchers can approach development for drugs for neurological disorders. FDA published the guide documents for Alzheimer's disease, Duchenne muscular dystrophy-related conditions such as migraine, epilepsy, and Amyotrophic Lateral Sclerosis on Feb. 15.
"These guidance documents will provide our current thinking and sound regulatory and scientific advice for product developers so that safe and effective treatments can ultimately be made available to patients," says FDA Commissioner Scott Gottlieb, M.D.
FDA is implementing the program under the leadership of FDA's Center for Drug Evaluation and Research Director Dr. Janet Woodcock.
Trials For Alzheimer's Treatment
The revised guideline will assist sponsors for the clinical development for the treatment of Alzheimer's even before the onset of dementia.
The approaches will include trials for patients who have not experienced visible impairments but may be identified using biomarkers such as the use of sensitive cognitive screening, imaging tests.
"Biomarkers may actually be able to detect who's on that Alzheimer's path," according to Maria Carrillo, chief science officer for the Alzheimer's Association.
This development, regarded as a major policy shift, will consider giving the go-signal for drugs for preclinical Alzheimers.
Based on the current guideline, Alzheimer's Disease: Developing Drugs for the Treatment of Early Stage Disease issued in February 2013, the FDA would only approve new drugs for Alzheimer's disease when clinical results are proven. Drugs for later stages of Alzheimer's must have clinical results before the FDA approves it.
Patients with stage 1 or preclinical Alzheimer's exhibit characteristic pathophysiologic changes but with no evidence of clinical impact.
Guide For Other Disorders
For migraine, manufacturers would only be required to conduct trials for pain reduction and effects of only the most bothersome symptoms.
For ALS, drug development can be targeted to an identified ALS patient subgroup and efficacy should be established by demonstration of a clinically meaningful effect on symptoms or function, or of a favorable effect on survival.
In relation to muscular dystrophy and related dystrophinopathies, the guideline stated that there is a need to understand the safety and efficacy of investigational drugs for dystrophinopathies across disease stages. The FDA said there is a strong rationale for treatment of patients at an early age to preserve the muscles.
Significant Policy Shift
The important policy shift, particularly in the development of the Alzheimer's drug, could give hope to the therapy of patients with early-onset Alzheimer's.
It would also open new opportunities for pharmaceutical companies and drug makers after failures from companies like Eli Lilly & Co., and Merck & Co. to develop drug treatments for the debilitating disease considered as an unbeatable enemy of drug makers. Merck & Co would stop the stage trial of verubecestat in early-Alzheimer's patients, studies determined that the drug was unlikely to work.
According to the Alzheimer's Association, in 2017, more than five million Americans have Alzheimer's dementia. Majority of the patients are individuals aged 65 years and older. Approximately 200,000 individuals have younger-onset Alzheimer's. It is the sixth-leading cause of the death in the United States.