In the mid-80s, I was diagnosed with HIV, the virus that causes AIDS. I was 28 years old and given 18 months to live.
At the beginning of the AIDS pandemic, there was not a single therapy or drug to stem the scourge of AIDS in this country. But in the 1960s, AZT was approved by the FDA to treat cancer and was soon shelved for failing to show efficacy. Twenty years later, a collaborative effort between the NIH and industry allowed the NIH to repurpose AZT because it proved effective against the immunodeficiency virus. AZT became the first drug approved to treat HIV/AIDS. All told, it took 25 months from the point when researchers learned AZT was active against HIV in the lab to FDA approval. It was one of the shortest development timelines in recent history.
I stared taking AZT in 1987 and while it wasn’t the ultimate answer to my illness, I believe that it gave me the critical time I needed for other interventions to be developed. By this time, I was enrolled in clinical trials at the NIH. In 1995 my participation in these trials resulted in me receiving the first approved protease inhibitor that saved my life. However, a drug rescued from obscurity 20 years ago is the reason I can proudly make the statement that AIDS-related complications will not be the cause of my death.
This week, I stood with Health and Human Services Secretary Kathleen Sebelius and Dr. Francis Collins, the Director of the National Institute for Health, as they announced an initiative called The National Center for Advancing Translational Science (NCATS). That’s a big name for a very important project — and it’s going to save lives. Basically, it will do for other patients what AZT did for me and so many others.
This is an indication that the Obama administration and the medical research enterprise are thinking out of the box. And, they need to. NCATS can and will bring cures and interventions to tens of millions of Americans across this country who are suffering and looking for leadership from the federal government.
Sebelius and Collins laid out the goals of NCATS on Thursday. From Science Insider:
The National Institutes of Health (NIH) today announced a new plan for boosting drug development: It has reached a deal with three major pharmaceutical companies to share abandoned experimental drugs with academic researchers so they can look for new uses. NIH is putting up $20 million for grants to study the
“The goal is simple: to see whether we can teach old drugs new tricks,” said Health and Human Services Secretary Kathleen Sebelius at a press conference today that included officials from Pfizer, AstraZeneca, and Eli Lilly. These companies will give researchers access to two dozen compounds that passed through safety studies but didn’t make it beyond mid-stage clinical trials. They shelved the drugs either because they didn’t work well enough on the disease for which they were developed or because a business decision sidelined them.
Often tens of millions of dollars and years of research have gone into these compounds, so they are already well along the drug development pipeline. The government program will allow academics to “crowdsource” ways to use them, said NIH Director Francis Collins.
The idea of finding new uses for existing drug compounds is not new. NCATS is an important first step in identifying thousands of other drugs currently not in use, like AZT in 1986 for AIDS. But, it’s not only going to address HIV and AIDS drugs. I used that example because it is personal to me and because it illustrates the outcome that can be achieved through a model such as NCATS.
I have spent my career working with patient advocates, caregivers and scientists at the NIH, in academia and in the industry, who have spent their lives searching for cures. There isn’t one person in this country who hasn’t been touched by ALS, Parkinson’s, MS, Alzheimer’s, spinal cord injury, cancer, diabetes and thousands of other diseases and conditions. And for every American who is suffering, there is potentially an already approved drug waiting to be rediscovered. The Washington Post explained the “big advantage” to NCATS:
A big advantage of the program is that the companies have agreed to use a “template agreement” outlining the financial arrangements, sparing long and expensive negotiations with researchers.
That matters because these are challenging times, both economically and politically. Everyone seems to have a hard time agreeing on anything.
But this is a step in the right direction and it is critical that industry collaborate with patient groups and their constituents. At the NCATS announcement, Dr. Collins stated that the federal government will not play a role in determining drug prices. This presents the industry with a great opportunity to engage patient groups throughout the drug development process and ensure that patients’ voices are heard. It would be tragic to come up with new applications for these compounds, just to have them end up being inaccessible because of high prices.
I can pretty much guarantee that everyone will at some point become a patient. Developing new therapies and cures is difficult. It will always be difficult. But if we all work together — patients (which means all of us), the federal government and industry — we will get to our shared goal faster. And, it’s important to note that the drugs being used by NCATS are not FDA-approved. But, the compounds have undergone significant research and development by industry, including safety testing in humans, providing a strong starting point for scientists and permitting the process to move more rapidly. That’s going to have a beneficial impact on all patients — meaning, at some point, everyone.
I have to salute the three pharmaceutical companies, Pfizer, AstraZeneca, and Eli Lilly, for having the foresight to be pioneers in this initiative. Their commitment to curing diseases will not be forgotten by the patients in this country. The same is true for Dr. Collins and his entire team at the NIH and the NCATS.
That I was able to stand at a podium on Thursday, with the Secretary of HHS and the director of NIH, supporting an initiative that brings the federal government and the industry together for the good of the patients in this country, was a remarkable thing.
My plea is that this truly be just a first step with many, many more steps to come, as we all go together down the road to cures. The National Center for Advancing Translational Science gives me, and patients like me across this country something that you can’t put a price tag on — hope.