How to Solve Diseases with Existing Drugs

My blog guests this week are Amy Conn, Bruce Bloom, and Clare Thibodeaux from Cures Within Reach. Disclosure: I am a member of their Advisory Board, and think that testing older drugs for rare diseases is brilliant!

The power of repurposing

What if the latest treatment for cancer, diabetes or thousands of other unsolved diseases was already available?

Although this might sound too good to be true, “repurposing” existing drugs could make this a reality. Repurposing takes any drug, device or nutriceutical, already approved in one disease, and tests it in a different disease to quickly and affordably improve more patients’ lives.

cures within reach infographicCurrently more than 500 million people worldwide suffer from diseases that lack effective treatments. As a result, global health care costs are growing and patients are suffering. Yet new drug discovery can take 10-15 years and can cost over $2 billion. This process is too long and costly for many patients who needs a solution today.

Instead, repurposing leverages prior investments by finding new uses for “old” drugs. This means repurposed treatments can reach patients in about 3 years and for less than $500,000.

Testing for repurposing sometimes means they are first tested in tissue samples or animal models in laboratories to find accurate dosing. If this pre-clinical research is done, the drug can quickly move into human trials. In some cases, it moves into human trials immediately.

How do repurposing ideas happen?

Repurposing is not a new concept. Doctors often prescribe medications ‘off-label’ when they think the patient may benefit. This happens when there is no approved treatment for a specific medical condition.

  • In fact, 1 in 5 prescriptions (21.3%) written in the United States are for off-label use. The range varies widely, based on the medical condition or patient group. For instance off-label use can be as high as 7 in 10 children for pediatric illnesses, because sponsors don’t typically focus on them. Here is a table with common off-label prescriptions.

Repurposing research can take off-label prescribing a step further by confirming clinical observations through a clinical trial. The data from these trials can then be published in scientific journals, and shared with other doctors to become part of standard treatment.

Other sources

  • Patients also contribute insights when they describe how a drug or supplement that they are taking for one issue impacts a different disease or diagnosis (i.e. “my asthma drug seems to be helping my eczema”).
  • Ideas also come from laboratory scientists, who connect the dots between newly discovered disease information and existing compounds.
  • And in our emerging world of big data, informatics engines can comb through scientific literature to find existing ideas and generate new ideas suitable for immediate testing.

Who pays for repurposing research?

Sometimes, pharmaceutical companies finance repurposing projects.

A case in point: ViagraViagra_in_Pack

Originally tested as a drug for angina, sildenafil failed. Instead, it was repositioned into the Pfizer blockbuster Viagra for erectile dysfunction after patients mentioned this side effect. Sildenafil was still under patent protection and the market was huge, making it a lucrative endeavor. This drug was later repurposed by Pfizer a second time in a different dosage under the name Revatio for pulmonary arterial hypertension.

Too many are orphans

Many repurposed opportunities are based on generic drugs that are often inexpensive and widely available. These characteristics make them ideal candidates for repurposing for patients, but industry doesn’t often pursue them, since there is no clear path to profit. This is where philanthropy plays an important role.

Cures Within Reach (CWR) is a global non-profit dedicated to repurposing research

CWR focuses on improving patient outcomes in any disease with repurposed treatments, while addressing larger needs around global collaboration, and the creation of alternative financial incentives for repurposing.

Successful repurposing

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Since 2010, 13 repurposing projects that Cures Within Reach supported have made a clinical impact. One such project involved repurposing sirolimus, a generic drug originally used to reduce organ transplant rejection, to treat a rare and deadly childhood disease called autoimmune lymphoproliferative syndrome (ALPS).

Children with ALPS carry a genetic mutation that causes some of their white blood cells to multiply and crowd out other types of blood cells. They suffer from enlarged lymph nodes and spleens, increased infections and anemia. Some patients can spend up to 10 days a month in the hospital receiving treatment, placing physical, emotional and financial burdens on patients and their family.

CWR funded research that helped prove that sirolimus produced a lasting positive response in patients, leading to fewer hospitalizations, greatly lowered medical costs and improved quality of life. This result happened in 3 years for about $250,000. Sirolimus has since been repurposed in 5 more childhood autoimmune diseases with similar results.

Ready for more successes with CureAcceleratorTM

Philanthropic funding was critical to this high-impact repurposing research. There are many more opportunities like this one, in which a small “investment” can create a life-saving repurposed treatment.

To find important repurposing opportunities CWR asks:cure accelerator logo

  • How can we help repurposing researchers connect with strong funding streams?
  • Are there financial models other than traditional philanthropy that can help scale this research?

To answer these questions, CWR created an online crowd-sourced platform in 2015 called CureAccelerator.™ Check us out, and watch for another post soon about CureAccelerator.

Repurposing is an important healthcare strategy, both in terms of patient impact and cost savings. As scientists and clinicians learn more about the mechanisms and molecular targets of diseases, repurposing will play an even larger role. The key stakeholders in repurposing, from funders to researchers to patient advocates to industry, all need to work together to drive new “old” treatments to patients.

Learn more about repurposing drugs here.

Patients ARE the (end)Point of Clinical Trials

CT Arena post on Feb conference 2016Back in February, I spoke at the 2016 Arena International Outsourcing Clinical Trials West Coast Conference on “Patient Centricity” – a topic they gave me, along with some rather ambiguous objectives. I changed it to “Patients ARE the Endpoint” because it is time to get real about why the clinical trial industry exists in the first place.

Yu Ping Yen from Dance Biopharm joined me in the 30 minute session, and detailed diabetes patient interactions after I introduced patient-focused concepts. You can see from the slides how tough it was to present so much information in a 30 minute time slot!

Arena International also asked me to pen a summary about the session, which now resides on the Clinical Trials Arena site. Somewhat chagrined that the title still includes the centricity word, though! Come on, what does that mean, really? Slide1

My hope is that all patient representatives (whether they speak of their own experience or broaden their talks to cover endemic patient issues) speak clearly and succinctly about what patients need and want, and why we need to be in the development process from beginning to end. Maybe then, the clinical trial industry will focus on patients instead of just products.

Quality Diet Research Needed

Welcome to the controversial world of dietary recommendations. Seriously, what do you do when you hear opposing stories about fat, cholesterol, carbs, supplements, etc.? Probably the same thing the experts do – expound on your own opinions and go get a snack!

Debate from All Sides

Guidelines Key Showing Guidance Rules Or PolicyCriticism ignited over recent U.S. Dietary Guidelines (USDG), which diluted evidence that disagrees with long-held dogma from the nutrition establishment. A really good CardioBrief article from Larry Husten (reprinted in MedPage Today) explains. Bottomline – it’s time for nutrition research to set and use standards. Hopefully, they will implement them more effectively than some fields (but that’s another story).

“…it is time to transition from the current evidence-free zone to an era where dietary recommendations are based on the same quality evidence that we demand in other fields of medicine.”
Steve Nissen, Cleveland Clinic in Annals of Internal Medicine

One crucial point to consider is the difference between a true cause vs. a related factor (correlation) to a health problem. By the way, this Los Angeles Times article explains “correlation is not causation.” Unfortunately, the USDG did not take this into account, according to this criticism:

“…in the 2015 DGAC [Dietary Guidelines Advisory Committee] report, the distinction between correlation and causation is either ignored or dismissed.”
– Edward ArcherGregory Pavela, and Carl J. Lavie in Mayo Clinical Proceedings

So What?

If this was an esoteric debate between scholars, no one else would care. But we have to care. Guidelines impact millions of people whose health may get worse, as evidenced with increasing levels of obesity and diabetes when people replace fat and cholesterol-laden foods with sugar and carbs. Experts are also worried about trends in coronary heart disease, some cancers, and other diseases.

Why Diet is Hard to Study

breakfast_MJS16UtuAnother excellent article in Vox from Julia Belluz outlines 6 major reasons why it’s hard to study nutrition, according to 8 health researchers. Please read the article for details – here are the reasons:

  1. It’s not practical to run randomized trials for most big nutrition questions.
  2. Instead, nutrition researchers have to rely on observational studies — which are rife with uncertainty.
  3. Another difficulty: Many nutrition studies rely on (wildly imprecise) food surveys.
  4. More complications: People and food are diverse.
  5. Conflict of interest is a huge problem in nutrition research.
  6. Even with all those faults, nutrition science isn’t futile.

Where Does This Leave Us?

Today’s “precision medicine” promise (notice I didn’t say reality?) shows that bodies handle food, drugs, and other things differently. All of our guidelines need to reflect this. Research standards can also account for observational challenges and research bias. Conflicts of interest should also have major repercussions for abuse, such as losing research and/or medical privileges. These are just a few of the changes needed in this important field of research. Please voice your opinions whenever and wherever possible.

All content © 2016 by Deborah Collyar unless otherwise specified. All rights reserved. Permission is granted to use short quotes provided a link back to this page and proper attribution is given to me as the original author.