ROAD TO THE CURE UPDATE APRIL 2018Category: Road to the Cure
Could Parkinson’s Be Next?
With the population living longer, neurodegenerative diseases such as Alzheimer’s (AD) and Parkinson’s (PD) diseases have become an increasing economic threat to the welfare of the world community, not to speak of the emotional toll it has taken on the patients and loved ones. According to some estimates, Alzheimer’s has more than 55 million individuals afflicted with the disease. US alone has 5.1 million Alzheimer’s patients, which does not take account for the millions that are yet to be diagnosed and those that are misdiagnosed. AD is the sixth-leading cause of death in the United States. It is the fifth-leading cause of death among those ages 65 and older and a leading cause of disability and poor health. With no cure on the horizon, the FDA has begun to rethink its strategy for the approval of new drugs for treating early stage (stage 1) Alzheimer’s disease. The following paragraph from the FDA is being distributed for comments purposes only, not for implementation.
Endpoints for Early AD Trials in Stage 1 Patients “Because it is highly desirable to intervene as early as possible in AD, it follows that patients with characteristic pathophysiologic changes of AD but no subjective complaint, functional impairment, or detectable abnormalities on sensitive neuropsychological measures (Stage 1 patients) are an important target for clinical trials. A clinically meaningful benefit cannot be measured in these patients because there is no clinical impairment to assess (assuming that the duration of a trial is not sufficient to observe and assess the development of clinical impairment during the conduct of the trial). In Stage 1 patients, an effect on the characteristic pathophysiologic changes of AD, as demonstrated by an effect on various biomarkers, may be measured. Such an effect, analyzed as a primary efficacy measure, may, in principle, serve as the basis for an accelerated approval (i.e., the biomarker effects would be found to be reasonably likely to predict clinical benefit, with a post-approval requirement for a study to confirm the predicted clinical benefit). As with the use of neuropsychological tests, a pattern of treatment effects seen across multiple individual biomarker measures would increase the persuasiveness of the putative effect.” [Source: Early Alzheimer’s Disease: Developing Drugs for Treatment Guidance for Industry, February 2018] In essences: The End Point simply means how to determine if the drug is working. In the case of Alzheimer’s, clearance or reduction of the amyloid-plaque, a biomarker, would be an indication that the drug is working.
Because clinically meaning benefit cannot be measured in early stage AD patients, the FDA seems to suggest that if a drug can bring “functional” change in the biomarkers of the disease the drug may serve as the basis for accelerated approval. This is an encouraging news for Companies like ICB International (ICBII) as it can potentially accelerate market penetration of its SMART Molecules for Alzheimer’s as these drugs have already shown significant reduction of amyloid-plaque (biomarker) in Alzheimer’s mouse models. The same may be true for Parkinson’s disease down the road, which will facilitate market entrance of ICBII’s drug, α-Syn-SM.