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Margaret Anderson Headshot

Patients Need Discoveries Translated to New Therapies

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The current drug development paradigm is not working -- it takes an average of 13 years and $1 billion to develop a new drug. Despite our strong national commitment to medical research, and to the National Institutes of Health (NIH) in particular, the advances achieved in basic science are not being translated into new therapies, better prevention strategies and cures fast enough.

The opportunity we must seize now is contingent upon having systems in place that will allow these ideas and discoveries to be translated into effective products and therapies that will ultimately improve patients' health and quality of life. If indeed we are in an era where the scientific knowledge and possibilities are abundant, but they are getting stuck in the translational pathway, we have to get moving.

The proposed NIH National Center for Advancing Translational Sciences (NCATS) is one way to get moving. Creation of NCATS would speed the translation of basic discoveries to real world applications and ensure that we are focused on the end goal of our investment in research: treatments and cures.

NIH Director Francis Collins has described his vision for NCATS to the scientific community.

But what does NCATS mean to patients?

To patients, saving time means saving lives. NCATS would accomplish this by improving tools and making these widely available and creating efficiencies. This means the process of translational research will be improved, and patients will benefit.

This vision provides an opportunity for the patient and disease community to work together on addressing a problem that cuts across diseases.

And as a component of the NIH, NCATS would be able to leverage its extensive network of relationships and collaborations with academia, patient and disease organizations and industry.

For example, only 1 in 10,000 potential therapeutic compounds makes it through the drug development pipeline to the marketplace. However, in recent years, researchers have been learning that a compound that is a failure for one condition may turn out to be a godsend for people suffering from another disease.

NCATS would support creative ways to rescue drugs that have been abandoned by industry before FDA approval.

To date, there has been no effort to encourage this in a broad and systematic way. While most compounds prove to be safe in human clinical trials, many fall by the wayside relatively late in the development process because they do not effectively treat the condition for which they were intended. When that happens, biotechnology and pharmaceutical companies typically shelve the compound and all related data.

The result is countless compounds sitting "abandoned" in industry freezers. NCATS wants to change that. Given its status as a neutral third party, NCATS may be able to serve as an honest broker to match compounds abandoned by industry before approval with potential new applications.

One example of the power of drug rescue is azidothymidine (AZT), a compound that was abandoned in the 1960s after failing to show efficacy against cancer. More than two decades later, a collaborative effort between NIH and the private sector allowed NIH researchers to rescue AZT after they discovered that it was effective against an entirely different health threat: the human immunodeficiency virus, which causes AIDS. AZT went on to become the first drug approved to treat HIV/AIDS, providing much‐needed hope for people suffering from what was then a swiftly fatal disease.

All told, it took 25 months from the point when researchers learned AZT was active against HIV in the lab and FDA approval -- one of the shortest drug development timelines in recent history.

Another way to speed the drug development process and to reduce costs is to find new uses for drugs already approved by the FDA for a different condition. Many pharmaceutical companies have already started exploring repurposing FDA‐approved drugs. However, there have been only limited exchanges of information with other companies and with academic researchers working to find treatments for other diseases. NCATS would be a logical entity to facilitate the repurposing of FDA‐approved drugs.

We have already seen how this can work.

One noteworthy example of repurposing is thalidomide. Originally developed in the 1950s to treat morning sickness, it was pulled off the market because it caused severe birth defects. However, researchers later found that the drug could improve pain and skin inflammation in people suffering from leprosy. More recently, research teams supported by NIH found that thalidomide could inhibit the growth of certain cancers and, in 2006, the FDA approved the drug to treat multiple myeloma.

NCATS would also serve to strengthen the translational efforts underway at many NIH institutes.

Many institutes at NIH support both basic and translational research portfolios in an effort to move discoveries through the pipeline more quickly.

Mid-sized and smaller institutes that may not possess significant expertise in drug development would benefit greatly from having access to a central hub at NIH where such expertise exists.

Another specific way that NCATS would assist the institutes in enhancing their translational research efforts is in the design of projects; one institute director indicated that in a project they had just designed, if NCATS had existed as a resource approximately two years would have been saved because the expertise would have been in place to advise them to be more efficient.

All of these are essential to all the sectors, and the NIH has the ability to share the insights and system improvements coming out of this work across every entity in the medical research system. But the NIH can't go it alone. The entire medical research community must come together to ensure this proposed center does not become part of the road paved with good intentions. And we urge our congressional leaders to support the establishment of this important effort.

Patients need these improvements in translational research, and that means you and me and a whole lot of other people out there.

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