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3 Steps for Tackling a Devastating Disease

August 27, 2014
An estimated 24 million people worldwide have dementia, most from Alzheimer’s disease. That number is predicted to double by 2020 – just over five years from now – and triple by 2040.

The numbers are equally striking for the U.S., where more than a million people over the age of 65 are afflicted today – a number that is expected to triple by 2050.

The financial cost to the U.S. healthcare system of caring for individuals with the disease is estimated at over $200 million today and will rise to over $1 trillion by 2050.

The societal costs are inestimable. These predictions do not include individuals with early-onset of the disease, who are under the age of 65 at first onset or diagnosis.

Without intervention or prevention to delay or slow Alzheimer’s progression in patients, or the discovery of a cure, the disease will overwhelm the U.S. healthcare system within the next 25-30 years. Even incremental improvements in treatment, however, can generate substantial savings. The Alzheimer’s Association projects that a medicine that delays the onset of the disease by five years would decrease the number of American patients with Alzheimer’s by nearly half in 2050 and save $447 billion in related costs.

Something must be done.

I believe there are three key steps that will help expedite the development of cures and treatments:

  1. Collaborate by forming public-private partnerships and coordinating efforts with existing initiatives;

  2. Collect health care data, genomic data, and biospecimens to identify potential risk factors, causes, biomarkers and targets for intervention; and

  3. Communicate the outcomes of the study publicly to translate the results into treatments and, potentially, a cure.

There is broad agreement that advancements in biologic and drug development will best be accomplished through collaborations that bring together knowledge, skills, and expertise, as well as funding, from the public and private sectors to enhance what each sector is capable of and is doing on its own.

Such partnerships and consortia already are making progress in a number of areas with high public health impact, including Alzheimer’s.

Public and private entities worldwide are engaged in efforts to understand the disease, to determine how best to develop therapies, and to address the enormous challenges facing caregivers.

In the U.S., many such efforts also are under way, in part as a result of the mandate of the National Alzheimer’s Project Act and under the National Alzheimer’s Strategic Plan.

In addition, the International Genomics of Alzheimer’s Project combines the efforts of the Alzheimer’s Disease Genetics Consortium, the Cohorts for Heart and Aging Research in Genomic Epidemiology, the European Alzheimer Disease Initiative, and the Genetic and Environmental Research in Alzheimer Disease consortium. These groups are working together to identify Alzheimer’s genetic risk factors. Thus far, their cooperative efforts already have identified more genes than had been identified in the previous 20 years.

There is recognition that these multiple efforts need to be coordinated effectively for the greatest possibility of realizing a return on investment that expedites prevention of the impending Alzheimer’s crisis.

The lack of effective treatments is not for lack of desire to address the current and impending impacts of chronic disease in general and Alzheimer’s, specifically. Indeed, from the perspective of the biopharmaceutical industry, this is a top priority. However, the high failure rate of potential treatments, in particular, discourages R&D and investment.

It is well recognized that a first step toward a cure is to try to identify the cause of the disease, individuals who are at risk for the disease, and potential targets for intervention.

I propose the development and execution of a large-scale, longitudinal study that will include sequencing the genomes of 100,000 volunteers in age cohorts from those in their 20s through those in their 80s, as well as obtaining biospecimens and additional health care data from those individuals, to develop biological markers that may predict Alzheimer’s or other chronic diseases for which the cause is unknown or poorly understood. This large-scale, long-range study will not only yield data necessary to find ways to cure and prevent Alzheimer’s, it would help researchers find ways to treat hundreds of other diseases for which we still lack adequate therapies.

Our hopes for cures hinge on understanding how to intervene to halt the disease’s progress. Through this proposed study, we would identify precursors and early signs of disease or disease risk. This information could be made publicly available so drug and device developers would have defined targets and potentially could develop ways to prevent and treat the disease.

The urgency of finding a cure for Alzheimer’s cannot be overstated. It is difficult to find anyone whose life has not been affected by this devastating disease. Biotech holds the greatest promise for finding a cure. We must act now for the patients and their families who are counting on us.

NOTE: Jim will be participating in a 21st Century Cures Initiative Roundtable in Lancaster, Penn. tomorrow to discuss how to expedite cures and treatments for devastating diseases such as Alzheimer’s. For more information on the event, please visit this link.