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Identifying the Right Disease Targets to Develop Better Drugs, Faster

 NIH Director Francis S. Collins, M.D., Ph.D.

NIH Director Francis S. Collins, M.D., Ph.D., recently announced the Accelerating Medicines Partnership.
Photo Courtesy of NIH

The National Institutes of Health (NIH), 10 biopharmaceutical companies, and several nonprofit organizations have launched an unprecedented partnership to speed validation of disease targets so that new, more-targeted drugs can be developed faster than traditionally has been the case. Initially, the Accelerating Medicines Partnership (AMP) aims to identify the most likely targets, or biomarkers, of Alzheimer's disease, type 2 diabetes, and the autoimmune disorders rheumatoid arthritis and lupus erythematosus (lupus). NIH Director Francis S. Collins, M.D., Ph.D., recently discussed with NIH MedlinePlus how more than $230 million will be invested in this public-private effort over the next five years.

"Patients and their loved ones can't wait!"
—Dr. Francis S. Collins

Why such a project now?

There's never been a better time. Recent advances in genomics, proteomics, imaging, and other technologies are helping to uncover many, many promising biological changes as potential targets for new diagnostic and drug development. The challenge has been finding the meaningful biological targets.

What's been the problem?

If a drug is aimed at the wrong target, it won't work. This wastes untold time and precious resources. Patients and their loved ones can't wait.

Aren't there enough drugs already?

No. Drug development is a terribly difficult, costly business. The vast majority of drugs entering the drug development pipeline fall by the wayside—after years of work and millions of dollars.

Why is this happening?

One major reason is that we're not selecting the right biological changes to target from the start.

How will the new partnership change the situation?

After more than two years in the making, with hundreds of hours of intense discussions among creative minds who left their affiliations at the door, the AMP partners recognize you can't change the world—or in this case, drug development—overnight.

So that's why we are starting with three- to five-year pilot projects in Alzheimer's disease, type 2 diabetes, and the autoimmune disorders lupus erythematosus and rheumatoid arthritis. These will set the stage for broadening this effort to other diseases and conditions.

Has such an approach ever worked?

Yes. Recently, researchers identified a protein, called PCSK9, as a drug target. People with a rare gene variant that greatly reduces PCSK9 have very low levels of cholesterol, along with dramatically decreased risk of heart disease. Half-a-dozen pharmaceutical companies are now racing to develop drugs that lower cholesterol by blocking PCSK9.

Are there more opportunities like this?

I think there are a whole lot more PCSK9s out there. More than 1,000 new biological targets for drug development have emerged in the last five years from the study of human DNA. But no single entity can sift through and pick out the real home runs. We just need the right teams to help us find the right targets—and AMP has the right stuff to do that. Once the right targets are identified and validated, the companies can go after them with all their competitive juices to make something happen.

How is this different from other public-private partnerships?

The data generated from this partnership will be open access. The scientists from both sectors will sit at the same table, working together to make this happen in a fully open, accessible manner. This is not where everybody goes off and plays alone in the lab. We are very serious about making real progress.

Who is going to pay for the partnership?

It is a full 50/50, public/private collaboration, with about half of the initial $230 million invested by NIH through the Foundation for the NIH, and half coming from the 10 participating companies.

How might work from the pilot broaden to other diseases?

Rather than tackling every disease, the partners agreed to focus on an initial set of diseases that seemed ripe for this effort to test the model. Each of the pilots has clear milestones to measure success. If the model proves effective for these diseases, then other diseases will be added.


Accelerating Medicines Partnership (AMP)

Alzheimer's Disease

The Partners

Government
  • NIH
Industry
  • AbbVie
  • Biogen Idec
  • GlaxoSmithKline
  • Lilly
Non-Profit Organizations
  • Alzheimer's Association
  • Foundation for the NIH
  • Geoffrey Beene Foundation
  • USAgainst Alzheimers

Type 2 Diabetes

The Partners

Government
    NIH
Industry
  • Johnson & Johnson
  • Lilly
  • Merck
  • Pfizer
  • Sanofi
Non-Profit Organizations
  • American Diabetes Association
  • Foundation for the NIH

Rheumatoid Arthritis and Lupus

The Partners

Government
  • NIH
Industry
  • AbbVie
  • Bristol-Myers Squibb
  • Merck
  • Pfizer
  • Sanofi
  • Takeda
Non-Profit Organizations
  • Alliance for Lupus Research
  • Foundation for HIH
  • Lupus Research Institute
  • Rheumatology Research Foundation

Spring 2014 Issue: Volume 9 Number 1 Page 2-3