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European Biopharmaceutical Review


More for Less

Conducting medical research for drug rediscovery is a growing challenge, as the amount that is spent on it is minimal. However, there are many advantages with this method that are being overlooked

Approximately $140 billion is spent annually in the US on medical research. Of that total, the amount spent on research for drug rediscovery is about one-tenth of one per cent – the rest goes towards new discovery research. Yet in comparison, drug rediscovery research delivers an exponentially higher return on investment and provides substantially greater advantages for quickly delivering safe affordable treatments.

Millions of patients and caregivers are suffering with thousands of different diseases that currently have no effective treatment. Many patients are struggling with a rare disorder, defined in the US as a disease affecting fewer than 200,000 people. A Food and Drug Administration (FDA) approved treatment is available for only five per cent of the 7,000 rare diseases and disorders. The for-profit research industry cannot afford to create a new treatment for most of these rare disease patients.

However, new treatment opportunities can affordably be unearthed from current scientific knowledge, existing medical treatments and clinical practice observations by reusing, reconfiguring and combining approved drugs and devices, modifying treatment protocols and testing clinical observations. That is the essence of drug rediscovery or repurposing, and the resulting benefits are many.

Drug Repurposing

Rediscovery research is relatively fast and economical. It typically takes from one to three years to impact patients, compared to an average of 12-19 years with new discovery research. And it's less costly. The price tag to repurpose an FDA-approved drug or medical device is often less than $500,000, compared to more than $1.5 billion for the traditional approach.

The probability for rediscovering a successful outcome is much better with the repurposing methodology. The drugs have already been tested and proven to be safe and effective at treating at least one medical condition. Both the primary effects and the sideeffects of an approved drug can potentially have a positive effect on other medical conditions. 10-30 per cent of rediscovery research will create a new treatment, versus one of 10,000 compounds tested via new discovery research. Plus, repurposing existing drugs that are approved by the FDA minimises the obstacles of establishing a safety profile for drug interactions, as well as the need to educate physicians about usage, and reduces healthcare costs.

Success Story

An example of successful philanthropic drug rediscovery is the first and only effective treatment for refractory autoimmune lymphoproliferative syndrome (ALPS), a rare childhood blood disease that causes anaemia and increased infections.

ALPS patients with uncontrolled disease might spend 5-10 days in the hospital monthly during childhood and they very seldom live beyond their twenties. Rediscovery research created a treatment for this disorder by repurposing rapamycin, a safe drug already on the market.

Children on rapamycin have significantly reduced disease symptoms and rarely require hospitalisation. Many no longer require other expensive medications with side-effects. Children with ALPS who are taking rapamycin are leading generally normal lives and their lifespan is projected to increase substantially. Plus, the cost of medical care for many patients has dropped from as much as $100,000 per year to less than $5,000 per year.

Growing Collaboration

Philanthropic drug rediscovery research is creating new collaboration among stakeholders, including philanthropists, research institutions, researchers, disease-specific research funding and patient advocacy groups, industry, media and lay leaders. There is beginning to be a shared commitment to raising the necessary funding, creating and administering a grant submission and review programmes, and helping to publicise rediscovery research results that make a positive near-term impact on patients. Focus must be placed on how rediscovery research delivers value to underserved patient populations through a process of developing safe treatments that are much faster, more efficient and less expensive.

Expanded funding for drug repurposing research would provide an incentive for more scientists and clinicians to create and complete pilot clinical trials that provide proof of a ‘new’ repurposed treatment through a grant programme. This can focus on any disease, use any therapy and impact any group of patients in order to create better treatment options, improve early diagnosis or lengthen disease remission.

The approach to achieve these results can take one of several courses. Often, as with the ALPS success story, research results in repurposing a generic drug with FDA approval, to deliver an effective ‘new’ treatment. The drug can be prescribed off-label since it is being used in a way that is different from that described in the FDA-approved drug label.

Rediscovery Strategies

Another approach to successful drug rediscovery can be through combining drugs. Sometimes an older drug, or several older drugs, are combined with a newer drug to increase the effectiveness of the newer drug; or a drug is coupled with a non-drug treatment option, such as radiation, to make the non-drug treatment work better. Alternatively, a non-drug treatment option could be partnered with an available drug to make the drug work better.

Occasionally, when there is more than one drug that is effective at treating one specific disease, drug rediscovery will test various combinations of the different drugs to determine if a particular combination of the drugs works better for treating that disease, compared to the efficacy of any one of the drugs taken independently. Many nutraceuticals, such as vitamins, supplements and botanicals, have strong biological impact and can also be repurposed to create treatments that would be available to physicians and their patients.

Modifying current treatment protocols is another rediscovery strategy to identify additional therapies to help more patients for longer periods of time. These alterations can include modifying the time of day for administering the drug and reducing drug doses, while increasing the frequency of drug administration to reduce side-effects. Potential opportunities can also be found in scientifically testing clinical observations, including those from integrative medicine or medical approaches that have been reported to be effective in other parts of the world.

Funding Issues

Big Pharma also undertakes drug repurposing. Their focus is on maximising profits by repurposing proprietary assets to create profitable new treatments. Unless rediscovery research is being conducted by a pharmaceutical company with the intent to market another FDA-approved use for their drug, there is not a large financial incentive for Big Pharma to undertake drug rediscovery. Therefore the bulk of the projects in drug rediscovery are initiated through non-profit medical research organisations.

Philanthropic funding is currently the core of many successful rediscovery research initiatives. Finding the rediscovery research ideas begins with the non-profit organisation distributing a request for Letters of Intent (LOI). The LOI identifies the disease to be researched, the cost and length of the project, and includes a brief research description. A science advisory board from the non-profit organisation reviews all LOIs against criteria established to determine the viability of the proposed project. Factors determining eligibility may include:

● Confirming alignment of the proposed project with the definition of rediscovery research

● Targeting a disease that has insufficient treatment options

● Focusing only on those projects that would not be of interest to a for-profit entity

● Designing research to create a relatively safe and inexpensive treatment

● Creating a treatment resulting in significant patient impact in the near-term (next two to four years)

● Projecting a reasonable cost for the research

After screening LOI submissions, those that are deemed to be acceptable as prospective opportunities for drug rediscovery research are invited to submit a full grant application. All grant applications are also reviewed by the organisation leading the process, ranked by viability to deliver successful results, and presented to both scientific and lay boards and funders for their input on whether or not to approve and proceed with the proposed research.

Research Momentum

The source of philanthropic funding for rediscovery research can come from an individual donor or group of donors, a non-profit disease specific organisation, a foundation or a corporation. Drug rediscovery pursued by a non-profit organisation expedites delivery of safe and effective treatments when minimal profit potential exists. Philanthropic drug repurposing is growing from an accidental occurrence in research to a purposeful programmatic approach.

With the growth of both for-profit and philanthropic rediscovery research, and the massive patient need, there is a great opportunity for philanthropy, industry and government to work together to create incentives to push for more drug rediscovery to provide more patients with safe, effective ‘new’ treatments that are more affordable.

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Bruce Bloom is the President and Chief Science Officer of Cures Within Reach, a medical research non-profit organisation that has been helping patients since 2005 by repurposing drugs and devices to quickly deliver safe and affordable treatments and cures for diseases and rare disorders. He holds a Juris Doctor degree from the Chicago Kent College of Law, a Doctor of Dental Surgery degree from the University of Illinois Medical Center, and a BSc degree in Biology from the University of Illinois. Bruce is a faculty member at Kendall College, an accomplished lecturer and serves as a board member for numerous organisations. Email:
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