番茄社区

Do you have a patient with an inherited retinal disease (IRD) who is considering participation in a clinical trial? With voretigene neparvovec (Luxturna) being the only treatment approved for an IRD thus far, and many novel therapies currently being studied, some patients may be looking to other avenues for a chance at treatment.

Overall, "the earlier we can treat, the better," Christina Weng, MD, MBA, of Baylor College of Medicine and the Cullen Eye Institute in Houston, told 番茄社区. "These diseases are degenerative, and we still do not have reliable ways to restore vision once it is lost as a result of photoreceptor death or retinal atrophy; thus, intervening at a point before cell death or atrophy occurs is key."

"The first step is to make sure patients receive , since many clinical trials in this space are still mutation-specific," Weng noted. For patients whose mutation is undetermined, some treatments that are not mutation specific are also being investigated.

"Once you have their disease and mutation identified, search on websites such as to find trials that are actively enrolling; I have found that these patients are often willing to travel across state lines if there is a trial that suits them," said Weng, who also helps her patients connect with trials for which they may be eligible.

Patients should discuss with their own eye care provider, Ian MacDonald, MD, of the University of Alberta in Canada, told 番茄社区. In addition, "Fighting Blindness Canada maintains a registry of patients who wish to be linked to clinical trials."

Cynthia Qian, MD, of the University of Montreal, noted that patients may find navigating the ClinicalTrials.gov website confusing, "as anyone can list any study, and equal weight may be given to studies of different levels of evidence."

Patients should be encouraged to feel comfortable doing their own research and discussing it with their eye care provider, she added, and, in turn, clinicians should stay up to date, so as to be able to talk with their patients, or be prepared to refer patients to colleagues who are able to provide more information.

"From a counseling standpoint, I always try to set appropriate expectations for the patient, while giving them hope that with all the research that is currently ongoing, the chance of identifying an effective treatment is better than ever before. It's a delicate balance," said Weng.

Patients who participate in clinical trials should do it to advance the field, Jacque Duncan, MD, of the University of California San Francisco, told 番茄社区. "They are more likely than not to experience no benefits from the intervention, and participation requires a major commitment of time and possible discomfort. However, progress toward development of treatments is not possible unless people who are very altruistic participate in clinical trials."

A survey of oncology patients, published in the , identified the following concerns around clinical trial participation: lack of knowledge regarding trials (47%), fear of side effects (43%), frequent visits (25%), cost (20%), transportation issues (14%), wage loss (9%), and inability to determine own treatment (4%). Of note, the survey analysis showed that patients with an annual income greater than $25,000 were approximately twice as likely to be concerned about treatment costs and side effects.

To help with patient concerns, a National Cancer Institute-funded research team developed an approach in pediatric oncology that can be used to discuss possible with a broader range of patients for various disease areas, with a sequential approach:

1. Explain the disease
2. Describe current best-proven treatments
3. Discuss the possibility of a clinical trial

Clinicians should encourage questions, and ensure that the potential participant has a good understanding of each step before moving on to the next one.

Asked which of the seem most promising, MacDonald said that "the clinical trials of a modified RNA approach -- which uses an antisense oligonucleotide -- seem most promising to me."

"It's important to note that we need to be very careful in recognizing that these approaches are experiments and they are clinical trials," he cautioned. "Luxturna is the only [approved] treatment and its effect looks promising."

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