Advancing Laboratory Treatments into Human Studies by Dr. Stephen Rose, Foundation Fighting Blindness

Head shot of Dr. Stephen Rose

Stephen Rose, Ph.D., is the Chief Research Officer of the Foundation Fighting Blindness, Inc.. The urgent mission of the Foundation is to drive the research that will provide preventions, treatments, and cures for people affected by retinitis pigmentosa, macular degeneration, Usher syndrome, and the entire spectrum of retinal degenerative diseases.

Dr. Rose’s essay, entitled “Found in Translation: Taking on the Challenge of Advancing Treatments into Human Studies,” appears in the Winter 2013 edition of In Focus, the Foundation’s quarterly newsletter. In clear language, Dr. Rose explains the process of – and the problems inherent in – moving a clinical trial from the laboratory to human subjects. It is reprinted with Dr. Rose’s, and the Foundation’s, permission.

Translating Research into Human Studies

Moving a potential vision-saving treatment out of the laboratory and into a clinical trial is a risky and costly proposition. While it can cost hundreds of thousands of dollars to demonstrate that a potential treatment works in an initial mouse or other model of retinal disease, it costs several millions to move, or “translate,” this into a human study. And even when the large investment is made, it may not yield the return of an approved therapy.

Why Is Translational Research Expensive?

Ensuring safety is a big reason. The therapy must be carefully evaluated in additional animal and/or cell-based models to show that it causes no adverse problems, such as starting an immune reaction or having a toxic effect. Also, because the human eye is much bigger than a mouse eye, researchers need to demonstrate that they can get a therapeutic dose of the treatment to the retina through eye drops or orally, which exposes the body to potential side effects.

In addition, the developer of the treatment must follow what are called “good manufacturing practices,” or GMP, to ensure that the treatment is sterile and safe, and that each dose consistently delivers the same concentration of drug or biological therapy.

Finally, for the clinical trial itself, there is the recruitment of patients, the selection of clinical researchers and trial sites and the determination of the actual protocols and outcome measures.

All of these translational efforts and decisions must be documented meticulously to gain authorization from a regulatory agency, such as the Food and Drug Administration for U.S. trials, to launch the human study.

No Guarantees

Perhaps the most sobering aspect of translational research is that even if all of the above are done thoroughly and correctly, there is no guarantee that the proposed therapy will ultimately save or restore vision in humans. In fact, most potential treatments won’t make it through the clinical trial process.

Because large pharmaceutical companies and therapy developers are often reluctant to take on the significant risk and expense of the translational process, many potential therapies stall in what is appropriately referred to as “The Valley of Death.”

The Translational Research Acceleration Program (TRAP)

Logo of the Foundation Fighting Blindness

While the reality of translational research can seem overwhelming, the Foundation Fighting Blindness is taking the challenge head on through its Translational Research Acceleration Program (TRAP). By advancing therapies into early-stage clinical trials, the program is also “de-risking” the treatment development process to attract for-profit and venture capital investments. The good news: Some TRAP projects are already doing just that.

TRAP was established by Gordon Gund, co-founder and chairman of the Foundation, along with other key research investors, who recognized that the focus on translation is imperative to get vision-saving therapies out to the millions who need them. Launched in 2008, the program is investing $20 million annually in moving promising gene therapies, stem cell treatments, and pharmaceuticals through late-stage lab studies and into clinical trials.

TRAP also supports projects for genetic testing, the discovery of new disease-causing genes and imaging studies to better understand retinal disease processes and treatment effectiveness. For details on the 15 projects currently funded by TRAP, visit

Additional Information about Clinical Trials from VisionAware

Most clinical trials are designated as Phase I, II, or III, based on the questions the study is seeking to answer:

  • In Phase I clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe and effective dosage range, and identify possible side effects.
  • In Phase II clinical trials, the study drug or treatment is given to a larger group of people (100-300) to determine if it is effective and to further evaluate its safety.
  • In Phase III studies, the study drug or treatment is given to even larger groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
  • In Phase IV studies, after the Food and Drug Administration (FDA) has approved the drug, continuing studies will determine additional information, such as the drug’s risks, side effects, benefits, and optimal use.

VisionAware will provide updates on Foundation Fighting Blindness research as they become available. You can learn more about clinical trials and ongoing eye and vision research at the VisionAware blog.