Our Research Strategy

Since our beginning, Charley’s Fund has focused on rapidly directing funding to the most compelling science. We also focus on solving the problems that stand in its way.

To date, we have directed more than $37 million to research. Check out the concrete examples below to learn more about some of our projects and how we work to translate research promise into real-life results.

Total funding to date:

more than $37 million

chart

pie-chart

Driving New Solutions

Protein Therapeutics

Treatments that aim to replace dystrophin or increase utrophin. Dystrophin is the protein kids with Duchenne are missing. Utrophin is a similar protein that can compensate for lack of dystrophin.

Case Study

Summit Therapeutics: Summit is conducting clinical trials for a compound that aims to increase utrophin. Charley’s Fund has been supporting the company’s work since 2008. We have helped them screen a vast library of compounds to identify molecules that have the potential to increase utrophin. Together with three other nonprofit partners, we financed the reformulation of the company’s lead compound and the phase 1 clinical trial. That work was deemed a success and has led to phase 2 trials which are now ongoing.

Treatments For The Whole Disease:

Lack of dystrophin causes a cascade of pathological effects. We support the development of new medicines that target these issues including fibrosis, muscle wasting, calcium infiltration, cardiomyopathy, and respiratory malfunction.

Case Study

DT-200: A company called Galapagos was developing a SARM (selective androgen receptor modulator) to treat cachexia, cancer-induced muscle wasting. They reached out to Charley’s Fund because they thought the drug might also be beneficial for kids with DMD. Charley’s Fund supported several studies in the DMD mouse model, and the results were encouraging. Ultimately Galapagos discontinued development of the drug for business/strategic reasons, so Charley’s Fund arranged for the rights to be transferred to Akashi Therapeutics. Akashi has conducted additional preclinical work on the molecule and is scheduled to conduct a short phase 1 study in healthy adult volunteers in early 2017.

Making It All Go Faster:

In addition to supporting the development of individual therapies, we also support system-wide improvements that will speed the way for all new treatments. This category includes developing better research tools, fortifying the clinical trials infrastructure, and smoothing the regulatory path for all new medicines.

Case Study

The Race to Yes: In 2013, we launched The Race to Yes, an independent movement powered by the Duchenne community and concerned public to help bring about a faster, more efficient regulatory process for DMD treatments. The Race to Yes pursues a three-pronged approach to achieving this end: direct conversations with the FDA, engaging elected officials, and public awareness/activism. We first sprang into action when we learned that the FDA was not providing Sarepta with clear and timely regulatory guidance for the clinical development of eteplirsen and follow-on exon skipping drugs in the company’s pipeline. Over the past two years, we have shined a bright light on the regulatory process to help ensure that our government is acting in the best interests of the people it serves.

Summit

A promising therapy clutched from the notorious “valley of death” and given new life

Background
Summit Therapeutics is a UK-based company that Charley’s Fund has been supporting since 2007. The company made good progress on its “utrophin modulator,” and even struck a deal with a deep-pocketed pharma partner to conduct the costly human clinical trials that would be needed to determine if the drug is a safe and effective treatment for Duchenne.

Problem
The first clinical trial in humans didn’t go so well. The drug didn’t have great uptake in healthy adult volunteers. If a drug can’t get where it needs to go, of course it can’t do what it needs to do. The pharma partner gave up, and Summit did not have the funds to keep the program alive.

Solution + Status

Before we allowed this drug to succumb to the notorious Valley of Death, we wanted to make sure it truly was a dead end. We hired a preclinical research expert to evaluate the data. He advised that if Summit reformulated the drug, there was a good chance they could overcome the bioavailability problem. Our advisor had seen this work several times before in his role as director of preclinical research at a large pharmaceutical company for more than a decade.

Charley’s Fund — together with three nonprofit partners — provided the funds to reformulate the compound and try again in healthy adult volunteers. The change improved the drug’s bioavailability, and the company proceeded to the next step: testing the drug in boys with Duchenne. Meanwhile, Summit RAISED $XX IN FOLLOW ON FUNDING FROM AN IPO, which has enabled them to build out their pipeline of Duchenne drugs. Today, Summit has multiple clinical trials going on for boys with Duchenne in Europe and the US.

EIM

Using technology to help get clear, objective results from clinical trials

Background
Our co-founder Dr. Benjy Seckler has always said, “The only thing worse than not having an effective treatment is having a treatment that works but not being able to show it.” In order to determine which experimental drugs are actually benefitting patients we need reliable and objective outcome measures that can tell us relatively quickly whether a drug is inducing a positive change. The most common measures used today are far from ideal, and hence are slowing the development of new medicines.

Problem
It can take a very long time to prove that an experimental treatment extends or improves life. A drug may preserve or strengthen muscle mass, but it can take many months or even years to become evident through changes on outcome measures such as the six-minute walk test or 4-stair climb. Without biochemical markers that change in response to drug treatment, access to new drugs is dramatically slowed.

Solution and Status

Electrical Impedance Myography (EIM) is a machine that measures the integrity of muscle fibers using high-frequency, low-intensity electrical current via surface electrodes. This tool has been used in other disease such as ALS, but had never been applied to Duchenne muscular dystrophy. We commissioned a clinical trial to determine whether EIM can distinguish between healthy muscle and DMD-affected muscle and identify stage of disease. The encouraging results were published in a peer-reviewed journal. http://www.ncbi.nlm.nih.gov/pubmed/25702806

While it is an important step in the scientific process, a published paper is just that – a step toward our ultimate goal of matierally improving the lives of boys with Duchenne. We are committed to moving the ball down the field until it reaches the end zone. Now, we are convening a “meeting of the minds” for Fall 2016 to determine whether this machine has significant promise as a reliable, objective outcome measure in DMD and if so, what next steps must be taken to make this happen.