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.

Pain Meds – How NOT to Get Hooked

Any body can have pain that needs to be eased. Unfortunately, US healthcare has created an epidemic of addicts instead. It’s time to STOP this madness. Some are trying, but we all need to pitch in. Don’t think you need this? Think again!

  • In 2010, 1 out of 20 people over age 12 said they used a prescription painkiller for another reason. That’s 5% of our US population, people!
  • 7,000 people go to the emergency room EVERY DAY for this.
  • 44 people DIE every day from an overdose of pain meds.
  • You (or your family) can become addicted with ONE prescription.

Which Pain Meds?

Most of the problems come from a family of drugs called opioids. The most common opioids include: Hydrocodone (e.g., Vicodin), Oxycodone (e.g., OxyContin), Oxymorphone (e.g., Opana), codeine, and Methadone (especially when prescribed for pain). Addictions can also start from benzodiazepines that include Alprazolam (e.g., Xanax), Diazepam (e.g., Valium), and Lorazepam (e.g., Ativan).

Who is at risk?

In a word, everyone. Older people, patients with chronic diseases or cancers, adults of all kinds, and kids (even newborns). BTW, pain meds affect women differently than men. I found out how pervasive these problems are at a CTAF meeting on migraines (disclosure: I’m an Advisory Board member). Their report urges health systems and societies to educate patients and healthcare providers (medical staff), and CTAF created guides to help.

“…migraine patients in emergency departments (EDs)…receive opioids over 50% of theCDC women pain addiction
time, even though…strong evidence shows that opioids offer no short term benefits compared to other treatment options and raise the long-term risk of exacerbating migraines and of contributing to opioid dependence.”

CTAF 2014 Controversies in Migraine Management report

What YOU Can Do

CDC when prescription is problemIf you or your loved one take prescription painkillers, ask questions and talk with your doctor(s) and family. Questions like:

  • What is the goal of taking this prescription?
  • How long should I take these drugs?
  • Are there any risks to me from these pills?
  • What do I do with extra pills?

Learn how to:

  1. Manage pain.
  2. Know possible risk factors that can lead to overuse.
  3. Stop taking pain meds as soon as your pain gets better.
  4. Use the Food & Drug Administration (FDA) guide on how to get rid of unused prescriptions.
  5. If you have or suspect a problem, check the signs of pain meds abuse. Contact 1-800-662-HELP. If you have questions about specific medicines, call1-800-222-1222.
  6. The US National Institute on Drug Abuse (NIDA) also discusses drug addiction treatment.


CDC rx-painkillers-sales-and-deaths-700wIf you treat/care for patients
, PLEASE look at this CDC information (presentation and summary). Then LIMIT prescriptions and use lowest doses, TALK with patients about how to stop/store/dispose of pain meds, and AVOID combination prescriptions.

“Patients in pain need help, not addictions.” Deborah Collyar

If you can influence government/healthcare systems (i.e. everyone), help develop coherent state policies and Prescription Drug Monitoring Programs (PDMPs) that don’t discriminate against patients who really need pain meds. Research on pain meds is being done by NIDA and other sources. Other research articles in this area are available here. The US Center for Disease Control and Prevention (CDC) has been working on this since at least 2006.

Mathematics of Pain Relief

Mathematics of Pain Relief

“Reduce abuse and overdose of opioids and other controlled prescription drugs while ensuring patients with pain are safely and effectively treated.”

CDC 2012 Goal

You only have to hear one of the many heartbreaking stories to get the message. Please spread the word. We can all help solve this problem.

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.