Global Health Repurposing Awards 2017 Blog Series

July 13, 2017

By Jennifer Ball


Sylvia and Berish GHRADrs. Berish Rubin and Sylvia Anderson direct the Laboratory for Familial Dysautonomia (FD) Research at Fordham University, which is dedicated to finding treatments for FD. FD is a rare inherited disease that impacts the development and function of the autonomic nervous system due to loss of functional IKAP protein. Patients with FD suffer from a variety of symptoms that include loss of body temperature regulation, blood pressure swings, vomiting crises, cardiovascular instability and lack of overflow tears.

Cures Within Reach recently recognized Drs. Rubin and Anderson for their influential research in the FD field by awarding them the Janet Davison Rowley Patient Impact Research Award at the 2017 Global Health Repurposing Awards. “We are extremely honored to be a recipient of an award that is named after an incredible trailblazer who was an excellent clinician, an outstanding researcher and an exceptional human being,” the researchers said. Read the full post!

Global Health Repurposing Awards 2017 Blog Series

July 13, 2017

By Jennifer Ball

Tranfaglia GHRAIn 1992, Dr. Mike Tranfaglia’s son Andy was diagnosed with fragile X, a rare genetic disorder that causes mild to moderate intellectual disability. This diagnosis led Dr. Tranfaglia and his wife, Katie Clapp, to found FRAXA Research Foundation (, a nonprofit organization focusing on finding a cure for fragile X. As part of its research strategy, FRAXA has made a significant investment in repurposing research, which is why Cures Within Reach awarded FRAXA with the Golan Christie Taglia Patient Impact Philanthropy Award at the 2017 Global Health Repurposing Awards.

The mission of Cures Within Reach is to improve patient quality and length of life by leveraging the speed, safety, and cost-effectiveness of medical repurposing research to help drive treatments to patients. “This is a great honor for FRAXA, and great encouragement to us individually. We spend a lot of time working on a rare disease in relative obscurity, and so this recognition is important to us,” said Dr. Tranfaglia. Read full post!

Global Health Repurposing Awards 2017 Blog Series

July 13, 2017

By Jennifer Ball

Lieven GHRAWhen the weight loss drug fenfluramine, half of the “fen-phen” combination, was taken off the market, it meant the worst for families with children who have the rare epileptic disorder Dravet syndrome. Some of these children had been taking fenfluramine to treat their condition for several years. Now, parents no longer had a solution for reducing their children's seizures. Enter Dr. Lieven Lagae, Director of the Childhood Epilepsy Program at the KUL University Hospitals in Leuven, Belgium.

Recognizing that fenfluramine helped children with Dravet syndrome in his hospital's residential epilepsy center, he decided to fight to bring this treatment back to market. “Fenfluramine made a real difference in the lives of our patients with Dravet syndrome,” Dr. Lagae said. “Freedom from seizures is really not a typical progression of Dravet syndrome.”

Cures Within Reach, a nonprofit that supports repurposing drugs, devices and nutraceuticals to treat unsolved diseases, has awarded Dr. Lagae with the Cures Within Reach Industry Patient Impact Clinical Award. Read the full post!

Global Health Repurposing Awards 2017 Blog Series

July 13, 2017

By Jennifer Ball

Tim Walbert GHRATimothy P. Walbert, chairman, president and chief executive officer of Horizon Pharma plc, started Horizon in 2008 with a focus on improving patients’ lives. He has grown the company from a handful of founding staff to more than 1,000 people in less than ten years. The efforts of the company to help patients with rare diseases by finding new therapeutic uses for existing medication is quite amazing, and is the reason why Mr. Walbert is being awarded the Cures Within Reach (CWR) Patient Impact Legacy Award. He received this honor at the Global Health Repurposing Awards on June 27 at the Radisson Blu Aqua Hotel in Chicago. “Receiving this award is a tremendous honor because it validates Horizon’s undertaking to make a positive impact on the rare disease community, giving a voice to these patients in the pharma community and beyond,” Mr. Walbert said.

Mr. Walbert first heard about CWR four years ago, and was immediately drawn to its mission of catalyzing research that looks at finding new uses for existing medicine and science as a means to deliver treatments to patients in need. Read the full post!

Global Health Repurposing Awards 2017 Blog Series

February 28, 2017

By Jennifer Ball and Lauren Collalto, GHRA Marketing Committee

For Nicole, a young girl with a rare autoimmune disease, the repurposing of the drug Rapamycin was a miracle.
Nicole is one of thousands of children for whom repurposed drugs change outcomes. “[Being sick] involved
half of my life in the hospital,” Nicole said. To the people who helped get her repurposed treatment, she said,
“You saved my life.”

Stories like Nicole’s are why Cures Within Reach is hosting the 5th Annual Global Health Repurposing Awards
on June 27, 2017 at the Radisson Blu Aqua Hotel in Chicago, IL, where this non-profit will give out patient
impact repurposing awards in philanthropy, industry and research among guests from industry, academia,
clinical care, philanthropy, government, and patient advocacy. “The 2017 GHRA Awards will highlight the
impact that repurposing research can have on improving patient outcomes and reducing healthcare costs,” says
Robert Metz, Senior Vice President of Horizon Pharma, co-chair of the event. Read the full post

One Health of a Life: Deborah Collyar

September 27, 2016

Guest blog post by Dr. Bruce Bloom & Dr. Clare Thibodeaux

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!

Ways to repurpose existing drugs, devices and nutraceuticals can offer powerful “new” treatments for diseases that desperately need them. A recent blog post (“How to Solve Diseases with Existing Drugs”) talked about the power of repurposing research. So why doesn’t more research with available drugs get done?


Read full article here

One Health of a Life: Deborah Collyar

April 28, 2016

Guest Blog post by Dr. Bruce Bloom, Amy Conn and Dr. Clare Thibodeaux

Currently 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.

Click here to read more....

Stanford Social Innovation Review

Dr. Bruce Bloom, April 8, 2016

Social finance could provide much-needed incentives for the development of known generic drugs to treat new diseases.

Around 500 million people globally suffer from one of 7,000 diseases that lack a known, effective treatment. But new drugs developed by the pharmaceutical industry address only 10 to 30 additional diseases each year. At this rate, it would take between 250 and 700 years to find treatments for them all!

Fortunately, new drugs are not the only answer. We can also test the clinical benefits of drugs already approved for human use for one disease for the treatment of other diseases. In this way, we could quickly repurpose thousands of cheap and widely available generic drugs to create “new” treatments for diseases that currently lack remedies.
Repurposing has the potential to bring more treatments to more patients more quickly than discovering, testing safety and efficacy, and securing approval for new drugs; it also can significantly cut healthcare costs. Read the full article Here.

Global Genes RARECast with Daniel Levine, January 27, 2016

Bruce E. Bloom Informal HeadshotThe high cost and long time it takes to develop drugs has people looking for alternative strategies for finding new treatments. One such approach is repurposing—finding new uses for already approved drugs. This is particularly compelling for rare diseases where small patient populations can serve as a disincentive to drug developers and the need for therapeutics is largely unmet. We spoke to Bruce Bloom, president and chief science officer of Cures within Reach, which launched the crowdsourcing platform CureAccelerator to fund repurposing research for rare diseases.

Bloom discussed the benefits of repurposing, how the CureAccelerator will work, and why he believes the initiative could help to build a new approach to repurposing research and developing treatments for rare disease. Listen to the full episode here!

Clare Thibodeaux, PhD, January 11, 2016


Too many people suffer with a rare disease. The search for treatments is often too slow and prohibitively expensive. But it doesn’t always have to be this way. There is a growing interest in drug repurposing – an approach that can deliver safe and affordable solutions quickly.

Cures Within Reach (, a global nonprofit focused on drug repurposing, has partnered with the Canadian Institutes of Health Research for a new funding initiative. Together they are calling for proposals for research into repurposing drugs specifically for rare diseases indications. This program is designed to encourage collaboration from clinical sites located both in Canada and the United States. A minimum of 10 projects will be selected for funding through this program.

How We Get To Next 

Nick Sireau, September 29, 2014

NickSireauBruce Bloom is just one man, but he potentially knows a way to save millions of lives. Addressing a room full of pharma executives at a conference in Boston, he commands their full attention. He speaks with authority and passion.The topic is one that industry has shirked for too long. It’s about how to find new purposes for existing drugs when there’s no intellectual property left to protect. Most of us don’t realize that many of the drugs we rely upon regularly can be used for many different, often unrelated, illnesses...Read full article here 

Bruce E. Bloom Dr. Bruce Bloom, April 15th, 2014

Cures Within Reach started repurposing drugs, devices and nutraceuticals by accident.  We funded one many studies repurposing Thalidomide for multiple myeloma in 2000.  We selected that Rediscovery Research project for funding, not because it was a drug repurposing project, but because it was the most promising project to accelerate the search for a cure from among a group of projects, all the rest of which were New Discovery Research.  In 2000, there was almost nothing published in the news or scientific literature about repurposing.  Had we shouted "Rediscovery Research" in the Grand Canyon of the medical research world in 2000, there would have been no echo!


Marquette Intellectual Property Review, Volume 18, Issue 1 (2014)

Dr. Bruce Bloom

Bruce E. Bloom Informal HeadshotThe current medical solution industry, with amazing scientists and businesspeople working as hard as they can, is not creating treatments and cures for most of the world's diseases. Pharma in 2013 is using a fifty-year-old business model to leverage current knowledge and technology, but that is generating few new treatments, and each treatment increases healthcare costs.  Read More


Dr. Mitchell Seymour, February 17th, 2014

Dr. Mitchell Seymour The most common approach for the new use of an "old" drug is off-label prescribing by doctors, often considered "innovative clinical care". Doctors get their motivation to prescribe off-label from sources like evidence in medical literature, presentations at professional conferences, and medical professional word-of-mouth. One challenge of off-label use is that insurance companies may not reimburse, leaving the patient to carry the financial burden of this innovative clinical care. However, insurance companies periodically review the value of off-label use through guided review of the medical literature and through a detailed risk-benefit analysis. If the story is good – that the new use for an old drug reduces health care costs – then off-label use can be included in the insurer's drug formulary, and the prescription gets covered. 

Dr. Teresa McNally, January 16, 2014

The National Alzheimers Project Act (NAPA) calls for a concerted national plan for Alzheimer’s Disease and Related Dementia (ADRD) treatment and care.  This collaboration between multiple federal healthcare offices, industry and non-profits is tasked to (a) prevent and treat ADRD by 2025, (b) optimize the quality and efficiency of care, (c) expand support for patients and their families, (d) enhance awareness and engagement, (e) track progress of the plan and to improve the process.  The plan and its goal will also be mirrored by the G8 countries making this a global effort to develop treatments for ADRD. 

Dr. Bruce Bloom, January 6, 2014

Bruce E. Bloom

Medicine and health is always in an evolution, and these days it is often evolving in opposite directions at the same time.  Health declines or improves because of behavioral changes, depending on whether you have adopted a sedentary lifestyle or started eating a healthier diet-or both at the same time!  Health improves or declines because we change medical care.  Technology brings us earlier diagnoses and more powerful treatments, but we’ve lost some of the healing power that a physician can bring to a patient because of time and regulation constraints.   

Dr. Mitchell Seymour, January 2, 2014

Dr. Mitchell Seymour While much of drug repurposing is physician-initiated and occurs as part of innovative clinical care, clinical research is an important tool to objectively and systematically evaluate the safety and efficacy of an old drug for new uses. Before a clinical trial can begin, an Institutional Review Board (IRB) reviews and approves a proposed study; its goal is to protect human subjects and to support Good Clinical Practice. As such, IRB approval is a regulatory hurdle in repurposing research - but there may be another, like the Food and Drug Administration (FDA) or other regulatory agencies worldwide.  

Dr. Bruce Bloom, December 10, 2013

Bruce E. Bloom

Cures Within Reach is totally focused on finding new uses for existing science and medicine, what we call Rediscovery Research™.  It wasn’t always our focus.  We missed the power of repurposing for a long time!  We first funded repurposing medical research in 2000, when we supported a project at Mayo Clinic repurposing the leprosy drug Thalidomide for a new indication in the blood cancer in multiple myeloma.  We didn’t support it because it was repurposing-we supported it because of the quality of the research and researcher.   We supported several other Rediscovery Research projects between 2002 and 2008, also not purposefully repurposing-just picking good projects from good researchers. 

Dr. Teresa McNally, December 5, 2013

It is clear that the repurposing or repositioning of existing approved drugs to treat diseases of unmet medical need can be a rapid, cost effective and successful strategy.  This figure, published in 2004, shows the combination of steps used in traditional de novo drug discovery compared to a repositioning approach.

Dr. Mitchell Seymour, November 20, 2013

Seymour head shotWhen I mention off-label drug use to my non-clinical colleagues or to the public, I often face confusion, winces, or leery looks. “Isn't that dangerous?” “How can that be allowed?” Misperceptions exist that off-label use is risky and deceptive. One goal of Cures Within Reach is to educate on the prevalence, impact, and value of repurposing “old” or approved drugs for new uses. Our audience is broad, but we accept that challenge with both passion and purpose.

Amy Conn, Director of Advancement
November 11, 2013

My colleagues here at Cures Within Reach have attended the Milken Institute’s Fifth Annual Partnering for Cures meeting in New York before.  But for me, it was my first experience with “business speed dating” at the Grand Hyatt with many hundreds of people working to weave a functional cloth out of the tangled threads of the healthcare industry.

Dr. Mitchell Seymour, October 24th, 2013

Dr. Mitchell SeymourWhen current medical options are limited or insufficient, doctors often leverage their knowledge and professional experiences to think and act outside the box. Off-label use is a critical tool in this innovative clinical care. 

A recent article in the Washington Post provokes interesting considerations regarding drug repurposing. doctor prescription The article suggests that off-label prescribing is inherently suspect or dangerous, and further supports this through commentary from prominent bioethicist Alexander Capron.  Capron believes that when physician finds a helpful off-label use for a drug, he/she is ethically obliged to publish a research article on the findings.

Dr. Teresa McNally,  October 17, 2013

Hutchinson-Gilford Progeria (HGPS) is a rare genetic disease for which there is no treatment.    HGPS patients age approximately seven times more rapidly than the normal population and usually die from age related cardiovascular disease before their 30th birthday.  Recent clinical studies from NIH have shown that the anti-cancer drug, lonafarnib, can improve the cardiovascular health of HGPS patients.  These promising studies demonstrate, for the first time, that the symptoms of HGPS can be reduced by therapeutic intervention. They have paved the way for future clinical trials to increase lifespan in this patient population.

Dr. Bruce Bloom, October 8, 2013

Drug repurposing, reprofiling, repositioning is growing like crazy! Industry, academia and philanthropy are suddenly all looking to drug repurposing as way to create “new” treatments. I just attended my fifth international drug repurposing conference in the last 24 months, this time in London. All five conferences have been small and personal-no more than 60 people, often as few as 25. The attendees at this conference included two academics, ten from biotech, ten from pharma, a few consultants, one venture capitalist and one from philanthropy-me from Cures Within Reach. Three attendees were from the US and the rest were from Europe.

Dr. Teresa McNally, October 3, 2013

Detailed clinical trial data are considered to be valuable information by both the pharmaceutical industry and the public sector. This information is critical for drug repurposing efforts where knowledge of the efficacy, off-target and side effect profiles of existing therapies are key to identifying new therapeutic opportunities. If detailed clinical data for a drug is publicly available, the time and effort required for a successful repurposing strategy are much reduced.

Dr. Mitchell Seymour, September 26, 2013

Seymour head shotBefore a clinical trial can begin, an Institutional Review Board (IRB) reviews and approves a proposed study with the goal to protect human subjects and to support Good Clinical Practice.  Cures Within Reach supports research involving off-label use of Food and Drug Administration (FDA)-approved drugs, also known as “repurposing”. Repurposed drug studies can differ in many ways from the FDA-approved, “on-label” use of said pharmaceuticals. These differences require special scrutiny by IRBs during their review of the clinical protocol. Differences can include:

Patient population - gender, age group, concurrent disease or organ function

Drug dose or dosing schedule - drug dose and/or frequency, duration or schedule of treatment

Drug formulation - drug delivery method, different excipients, different release properties

Dr. Persis Edwin, September 24, 2013

The Stanley Medical Research Institute in Chicago has concentrated for the last decade on exploring options in treating Bipolar Disorder (BD) and Schizophrenia. Their focus has been on resolving residual symptoms when patients fail therapy or partially respond to treatment.  This "failure" is very frustrating for both the patient and the clinician.  In my exposure to psychiatric patient care patients often became non-compliant when they felt their therapy was not working. 

Dr. Brian Dayton, September 12, 2013

Kudos to the National Institutes of Health for putting some monetary teeth behind its commitment to drug repurposing.  In June of this year, NIH awarded $12.7 million to match nine academic research groups with a selection of existing compounds from pharmaceutical industry partners to explore new treatments for patients in eight disease areas, including Alzheimer's, Duchenne muscular dystrophy and schizophrenia.  Led by the National Center for Advancing Translational Sciences (NCATS), this pilot program, called Discovering New Therapeutic Uses for Existing Molecules, provides academic research groups with milestone-driven, cooperative agreement awards valid for two or three years.

Dr. Bruce Bloom, September 5, 2013

Drug and device repurposing in medicine and medical research is everywhere, but unless we look closely, we don’t see it.  Several recent studies show that 20% of all prescriptions are written off-label and for mental health drugs over 30%Almost 2/3 of all pediatricians say they prescribe drugs off-label to their patients  Off-label drug use is common in obstetrics since during the last five decades of activity the FDA had approved only two drugs for obstetrical indications.  Some drugs are used more frequently off-label than for their original, FDA-approved indications.  While this is drug repurposing in its most basic, much of this off-label prescribing is done without the benefit of scientific validation

Dr. Bruce Bloom, March 28, 2013Bruce E. Bloom Informal Headshot

Two of our favorite organizations in the Repurposing Revolution are getting together to drive drug repurposing to patients. DART Therapeutic, a biotechnology firm started by a group of Duchenne muscular dystrophy (DMD ) voluntary health organizations, and Biovista, a partner of ours and the leader drug repurposing opportunity discovery entered into a research collaboration to identify and develop drug repositioning candidates for DMD using Biovista's Clinical Outcome Search Space (COSS)™ technology. We've used the COSS technology to help us find some drug repositioning opportunities for leukemia, and are working with BioVista on several other diseases.  Read more in this recent press release.

In addition, one the DMD projects in our Rediscovery Research™ portfolio has just gotten funding approval. This project, proposed by Dr. Subha Raman of Ohio State University, will complete a randomized, controlled clinical trial repurposing a drug typically reserved for advanced heart failure to delay heart disease in DMD patients.  

Dr. Bruce Bloom, May 12, 2012

Bruce E. Bloom

Ashoka, the fellowship of Social Entrepreneurs, held their first Global Summit of the Ashoka Support Network in Miami, FL May 9-11. The Ashoka Support Network (ASN) is an international group of business leaders and philanthropists that volunteer to support the social enterprise work of the 3000+ Ashoka Fellows worldwide. I was elected to the Fellowship in 2010 for the work I do with Partnership for Cures.

Dr. Bruce Bloom, October 23, 2012 Bruce E. Bloom Informal Headshot

What's in a name? Shakespeare wrote something about a rose by any other name smelling just as sweet. Does that mean the name of something is unimportant, that it's just the thing itself that matters?

Maybe, though a name can change perception. Chilean sea bass, a favorite seafood dish in high end restaurants, used to be thrown back into the sea when it was known by its previous name, the Patagonian Toothfish. No human would want to eat THAT, even though the Patagonian Toothfish is wonderfully flavored and meaty. That name doesn't describe the thing in a way that attracts people to it.

Jonathan LethemGuest Post

Five years ago, in an essay in Harper's called The Ecstasy of Influence, I issued a call to artists and critics to embrace the reality of "repurposing" in the creative act -- the timeless wisdom of appropriation and reuse of existing materials in the realm of arts and culture. In one section, called "Undiscovered Public Knowledge" I wrote (or, actually, retyped, since my words in this instance were wholly appropriated themselves from Steve Fuller's book The Intellectual):

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