Trial results create a WIN for all!

So sorry for the silence! I’ve been working on many topics and issues, and you will hear about them soon. I also forgot to share this guest blog post I did back in March 2017.

The Clinical Trials Arena post, Trial Results Create a Win for Patients and Companies, covers the reasons trial summaries need to make sense to real people. Health literacy is different than reading literacy and frankly, even people with MDs and PhDs (& other alphabet parts) behind their names have trouble understanding complex clinical trial information!

This post was an update to some my other posts, including:

Since that time, the European Medicines Agency (EMA) has finalized their Summaries of Clinical Trial Results for Laypersons recommendations. Unfortunately, there is still no final date on when public summaries must be posted on their website – it’s beginning to look like early 2019.

An article called Layperson Summary in the EU explains some of the history involved. By the way, PLEEEZ do NOT use the term “layperson” when referring to public research summaries! It is a ghastly term to use when you want to engage patients, and the Webster dictionary does not include “non-scientist” in any of its 14 definitions for the word “lay.” Truly, it sets up an elitist barrier that absolutely negates the intent of this initiative.

I am also now helping Health Literacy Media (HLM) develop their services for clinical trial sponsors. More on that soon – a new website is coming and will be announced!

Watch for updates about this and other initiatives related to sharing clinical trial results with patients and the public. It’s high time, and we’re all learning how to do this together!

Answers for DCIS are coming

Great news for those confused about Ductal Carcinoma In Situ (DCIS). That includes just about everyone, from doctors and researchers, to patients and their families!

Four new projects and resources are available: COMET logo

  1. A new study called COMET just opened that will look at whether women with low-risk DCIS will do just as well with active monitoring (also called Active Surveillance) as those who choose surgery, radiation and/or hormonal therapy. Watch the video.

    “The aim of this work is not to try and determine what’s ‘better,’ but rather to quantify the tradeoffs associated with these two approaches to DCIS treatment.”
    – Dr. Shelley Hwang, Principal Investigator

  2. A new website for DCIS also opened this week to help the over 50,000 women per year who are diagnosed with DCIS each year in the U.S. Of course, the site is also available for women worldwide.
  3. SHARE is sponsoring a webinar called “DCIS: What You Need to Know” that features
    SHARE DCIS webinaryours truly on March 22 at noon Eastern Time (US). We’ll explain what DCIS is, how to think about it, and what is needed to make rational decisions when faced with a diagnosis.
  4.  A new international research project called “Preventing Unnecessary Breast Cancer Treatment” was recently announced to learn how to find DCIS that will not turn into breast cancer so women won’t have to deal with treatment issues.CRUK DCIS graphic

Together, these projects can tell us how to deal with DCIS, what risk factors may cause approximately 1 in 10 women to develop a later invasive breast cancer, and hopefully, that Active Surveillance works just as well as invasive treatments.

By the way, about 90% of women with DCIS won’t get invasive breast cancer!

If you can’t wait to find out more about DCIS, check out this post or get the DCIS Dilemmas ebook. Stay tuned for more about these projects and other findings about DCIS!

COMET study team

Some members of the COMET Study team

A Place to Teach Old Drugs New Tricks

Due to popular demand, former blog guests Bruce Bloom, and Clare Thibodeaux from Cures Within Reach have returned with another post. This time, they explain how they bring researchers, older drugs, and new funders together to come up with new solutions for patients. Disclosure: I am a member of their Advisory Board, and think this concept is brilliant!
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Patients Want Their Data Shared

Patients also want to be:

  • Respected for their contributions to science and medical advances. Those contributions include samples from their bodies (biospecimens), information (data), experiences (input), and sometimes their very lives.
  • Protected from harm and misuse of sensitive data about themselves, their family, and/or their culture or ethnicity.

As long as we are respected and protected, we will participate in clinical research and learning healthcare systems so researchers can find better ways to treat and prevent diseases and medical conditions.

What & Who

This post focuses on data from past “legacy” cancer clinical trials. The Patient AdvocateProtectionCommittee in the Alliance for Clinical Trials in Oncology drafted a resolution to add the patient voice to the groundswell of support for data sharing.

Why?

  1. People who join cancer clinical trials are often asked to donate their tissue and data for future research.
  2. When patients give consent for research, they expect their information to be shared.
  3. Unfortunately, many hospitals, clinics, academic centers, or research groups will not release information to other researchers, even though patients gave their consent.
  4. This does not honor or respect patients who want to contribute to research.

Your Call to Action

  • Please read the resolution, sign on, and share it with your networks and organizations. While this resolution deals with cancer, its intent is to help all medical conditions.
  • Include patients in data sharing activities.

“Today, oncologists and cancer researchers realize that they can’t [advance cancer progress] alone… What’s required today extends beyond any individual or any individual discipline, beyond medicine itself… It requires somewhat of a change in mindset. It requires a lot more openness – open data, open collaboration and above all, open minds.

– Vice President Joe Biden, ASCO Speech

Thank you!

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.