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

Key In Lock Showing Forbidden Information And Privacy

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.

Oops! Don’t Buy Chocolate Milk for Concussions

No, really. This is a real thing, created by money. Oops, I meant to say, created by researchers and institutions with MAJOR conflicts of interest. Why post it? Well, while this is the most blatant example we’ve seen in a long time, it happens every day, even to well-meaning researchers.

silhouette arrow to dollar signFACT: research is a money game. If your proposal piques the interest of a funder (e.g.  government (public), private foundations, commercial companies), you get to do your research. Researchers are eternally grateful because, otherwise, they have to find a new career. Many proposals are worthwhile and survive rigorous grant review (including serious peer review) to qualify as “good science.” This one didn’t…

Note: always be wary of overly-enthusiastic researchers

The chocolate milk story shows how urban legends are founded, and never die. This story came to light because it was PUBLICIZED without the necessary data from the Public Relations or Press Release (PR) office, not PUBLISHED (as in a scientific journal).

While I always hope for the best in research, skepticism is a good thing. Why are good scientists inherently skeptical? Because researchers can quickly lose their objectivity and tout success based on hope, not on facts. In the worst cases like this one, their ‘hope’ can turn to hype, fame and/or fortune. It takes constant vigilance by each scientist, and the whole research system, to make sure facts lead to relevant conclusions instead of ego.

Unfortunately, news aggregators, like prnewswire.com, also spread false hype without question. By the way, University of Maryland (U MD) has since removed the press release since the ‘scandal’ began.

“There are real consequences to PR spin of health research.”

Andrew Holtz, Yoni Freedhoff, & Kathlyn Stone, HealthNewsReview.org reviewers

In this case, consequences for people included mass purchases of this particular chocolate milk by school athletic departments, as reported by Quartz and others. Unfortunately, the sugar content is very high, leading to other issues involved in dietary studies (see the post Quality Diet Research Needed for more on this). Consequences for U MD aren’t done yet.

Concussion/milk study: fact or fiction? Cow - dairy

But I digress – let’s get back to the study. HealthNewsReview.org first commented on a U MD press release (PR), giving it a 1 out of 5 rating on January 5th, 2016 (that’s bad). They couldn’t get any study information (data) because it had not been published.

This was picked up by other news sites later in January, such as Stat News and NYMagazine Science of Us, claiming more bizarre issues with the study. The only reason Retraction Watch wasn’t involved – the study was never published.

“…a scandal that touches on vital issues of scientific ethics, the collision of money and research, and the lightning-quick pace at which pseudoscience can lead vulnerable people astray. And it all boils down to a simple question: How the hell could the University of Maryland have allowed this to happen?”

Jessie Singal, Science of Us

Then, on April 1st (no joke), CBS News reported that U MD “disavowed” the study and would give back the $228,910 to the company who sells the milk and sponsored the study. I learned about it from this Huffington Post sketch.

All of this adds to other examples of concussion research conflicts in which the National Football League (N.F.L.) is embroiled, as reported by The New York Times.

But Deb, You Don’t Do Concussions!

Web research

True, I usually cover research in cancers and infectious diseases. So? This deals with a universal research issue that impacts millions of patients and people. Seriously!

Research is often done well, by researchers who care and follow ethical principles. Even the good ones with whom I’ve served on countless committees and review panels, however, can let ego, opportunity, and fame trump true objectivity. Many are also led down conflicted paths by their institutions who smell money, and are willing to give undue influence to sponsors in return.

Frankly, we need set Standard Operating Procedures (SOPs) for these kinds of conflicts for ALL academic research institutions (after all, that’s what standard actually implies). For instance, don’t do it in the first place. But, when a conflict is discovered:

  1. Give the money back (in process).
  2. Recall the erroneous information (check).
  3. Fire the researchers involved (not yet).
  4. Publicize the error and steps taken to resolve the problem through the SAME CHANNELS + others (not yet).
  5. Share the data so others can learn from it (not yet). Doesn’t matter if it’s bogus or not – everyone can learn and take steps to make sure it doesn’t happen again.

Health/medical researchers and their institutions/companies HAVE to understand that their work impacts lives. They MUST start policing their own, or others will do it for them.Important Stamp Shows Critical Information Or Documents

The references I listed in this article aren’t hard to find. It takes some effort, but it should represent the least amount of effort we all put into study results to make sure the claims match the data. So, thanks HealthNewsReview.org and others!

Got research? Do it right, or suffer the consequences.

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.

Getting Real about Patients & Clinical Trials

I’ve been asked countless times to write about patients and clinical trials. Guess it’s time to share what I’ve learned, along with other patient advocates, as we work beside researchers in the proverbial trenches. Just so happens that this post coincides with April Fool’s Day – hope that’s not an omen!

Why this series?

This is the first in a series on Patients & Clinical Trials (CT). Why? Because clinical trials are really the only tried & true way we get new diagnostics, treatments, and preventive approaches to people. For all diseases and health conditions.

But the research system isn’t very good at it, even after 70+ years. It’s not for lack of trying – BILLIONs have been spent on tweaking existing structures, but perplexing problems persist.

Heads up on CT activities

Other themes will also emerge, based on over 20 years of changing research and medicine culture (or at least trying to). In the meantime, here are some of my activities in the CT world:

Event Inscription on Sign

Other activities:information-orange-circle_fkMzuUIu_L

  • The California Technology Assessment Forum (CTAF), part of ICER, released their recommendations from the February meeting for new drugs in diabetes and asthma.
  • CTTI just released materials from an expert meeting about the issues surrounding FDA streamlined approvals for new antibiotic drug development. A summary will be posted soon. You can also read a past post called Antibiotic Resistance – It’s Complicated!
  • Cures Within Reach focuses on ways that existing drugs can be used for other purposes. More from this group in a guest blog post here soon!
  • Two DCIS studies are being funded by PCORI to help answer treatment questions for this pre-cancer, and learn how women feel about them. You can also read a past post called When is ‘Carcinoma’ Not Cancer?
  • The Center for Medical Technology Policy (CMTP) is working on recommendations to allow patients to switch treatments in a clinical trial if their cancers keep growing.
  • The Metastatic Breast Cancer Alliance (MBCA) is creating a summary of their February meeting from the Research Task Force to Advance Progress.
  • The NC ProCESS project on prostate cancer has convened a patient advisory board to help communicate effectively with prostate survivors in their study.
  • Two groups I’m working with (MRCT and the NCI NCTN Biospecimen Banks) are discussing how to handle the challenges of giving individual research results to patients.

You can also read past CT posts about:

There are many other clinical trial happenings – these are just some of the ones in which I’m involved! Yes, it’s heavy on cancer, but remember that cancers cover the gamut from rare diseases to millions of people.

Updates will be posted regularly. Let me know what you’d like to hear about. TTFN.

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.

A Clinical Trial Failed Patients

Something horrible happened – a clinical trial failed, causing one unexpected death and seriously injuring 5 people. And instead of dealing with a dysfunctional research system, “experts” are spouting off on their own. The few articles written to date focus on the drug (aka the money), not on what people want to know.

My Initial Thoughts

high wire climbing in the wood

ClinicalTrialsArena asked me to comment, due to my unique expertise that straddles the patient and research worlds. After researching the topic,* here are my concerns – most of which have not been discussed yet.

This poses a much larger question – why am I the one bringing these up?

Fact 1: Something really bad happened – the first 6 people to get “repeated higher doses” died or were seriously damaged. This is a clear sign of system failure (e.g. approval, protocol, procedures, formulation, PharmcoDynamics).

Fact 2: A phase 1 clinical trial means it is the first time a drug is studied this way in people. 127 people in the trial is far larger than classic phase 1, and most don’t have placebos. Isn’t this more like a phase 2, or the new-fangled phase 1-2 trial? If so, what happened to the rules?

Fact 3: The drug showed activity in laboratory dishes and animals first. We don’t know what mouse-realtesting was done, or for how long. How realistic are the animal models, and how closely do they relate to humans?

Fact 4: Existing articles stress the need for more ‘transparency’ to share the drug’s molecular structure. Patients want honesty first, which in this case may mean, who screwed up?

Fact 5: I’m a big believer (and trail blazer) in presenting trial results, but that’s when we have results. NOT during an ongoing, active clinical trial. Why aren’t we calling for the protocol to be publicly published immediately?

medical_1000006456-120613intFact 6: Healthy volunteers joined this trial after reading an ‘informed consent’ form about its procedures and risks, and they were paid well. Why isn’t the consent form (for every trial) publicly available once the trial is approved?

Fact 7: In the U.S., the Institutional Review Board (IRB) would be shut down and investigated before any new trials could be opened.

Fact 8: A thorough investigation on all parts of this system failure is obvious. This, too, should be open and as public as soon as possible, and at all times.

Fact 9: Risk is an inherent part of clinical trials because risk is part of everyday life. Even when rules are followed, bad things can still happen. Patients aren’t stupid, so there is no need to shut down other clinical trials.

A Setback for Other Clinical Trials?

Amazing we even have to ask this question, right? Of course it will!

Only other researchers may think their trial will be ok because, oh let’s see, because that trial was different – yes, that’s it! That trial (take your pick):

  •  was in a different disease
  •  was in a different country
  •  was run by a company I don’t know
  •  had a different study design
  •  was a horrible accident that would never happen to me
  •  add your own excuse

But patients won’t care about any of that. They’ll steer away from danger or discomfort. And let’s face it, anything called a “clinical trial” isn’t comforting. And as far as whether the rule were (or weren’t) followed – that doesn’t matter either. The rules in the case of a clinical trial include the protocol, of course.

I am firmly in the patient/participant camp. Some may call this a bias, but I consider it the only worthwhile endpoint. I know patients want better answers NOW, and I know people in research deal daily with regulations and hubris.

There was clearly a system failure here, and people needlessly paid too high a price. Let’s find out why and make the pieces work together so people won’t worry about needlessly putting their lives on the line.

* Online information (as of 1/21/16) about the French clinical trial from a Portuguese company is located on Facebook, at #clinicaltrials on Twitter, and is listed in articles from:

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.

Full-Fledged Flu

‘Tis the season, folks. Fancy the opportunity to find flu facts while falling onto the sofa. I fear I may have infected a few as I felt the forceful signs form within. Of course, my bout came from fellow flyers during my fortnight of travel. For those flummoxed by my fanciful alliteration, I have an excuse – I’m under the influence of influenza. The Flu I.Q. widget is an interactive quiz to test your flu knowledge.

First, some flu facts, according to the US Center for Disease Control and Prevention (CDC):

  • Flu cases start in October/November, usually peak in February and can last until May.
  • Flu vaccines kick in about 2 weeks after you get them, so now is the time!
  • Flu strains (types) differ each year, hence the call for yearly flu vaccines.
  • Almost everyone over 6 months old should get a vaccine. That means you, since you can read.
  • The flu is caused by a virus, so antibiotics DON’T work!
  • People who are at higher risk of flu complications should get antiviral drugs once they have the flu.
    • This includes: kids under age 2, adults over age 65, pregnant women, people with some medical conditions, or those who are hospitalized with the flu.
  • Check out “What You Should Know About the 2015-2016 Influenza Season.”

Vaccines: To Take or Not To Take

The Flu I.Q. widget is an interactive quiz to test your flu knowledge.Some people refuse to take the flu vaccine, due to mistaken beliefs, irrational fears, or stubbornness. Suzanne Koven puts it this way in her STAT article:

[Blaming flu vaccines] is “like blaming umbrellas for the rain.”

Suzanne Koven, A doctor’s explanation of why so many patients say no to flu shots

Did I get a flu vaccine this year? No, too busy. Do I wish I had? Yes, so I could get all the things done I’m thinking about while having the flu!

More information about vaccines is listed hereYou can stay up to date on Twitter with @CDCFlu. Good information is also available here.

Here’s wishing you a happy, and HEALTHY holiday!

All content © 2015 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.