Health Rounds: Blindness treatment based on Nobel-winning research shows promise

(This is an excerpt of the Health Rounds newsletter, where we present latest medical studies on Tuesdays and Thursdays.)

Oct 9 (Reuters) - It's Nobel Prize season and today we feature promising early results of a novel technique for treating blindness that made Nobel laureates of ​three scientists this week. We also report on two studies comparing treatment options presented at the American Society for Radiation Oncology meeting.

NOBEL-WINNING THERAPY SHOWS EARLY PROMISE IN BLIND PATIENTS

Patients with blindness due to retinitis pigmentosa who received a treatment based on research that won the 2026 Nobel Prize in Physiology or Medicine had their ability to detect objects restored in a small trial that demonstrated ‌the potential of the novel approach.

So-called optogenetic therapy uses light to control brain cells. In the study, researchers combined gene therapy that made surviving retinal ganglion cells sensitive to light with delivery of light to stimulate these cells.

The 10 trial participants each received an injection into one eye ⁠carrying genetic instructions for ChrimsonR, a light-sensitive protein from GenSight Biologics.

Patients then wore goggles that captured visual information, ​converted it into patterns of light at wavelengths that activated ChrimsonR in the modified retinal cells, and ⁠sent the patterns of light back to the eye.

Seven patients became more sensitive to light, with six achieving clinically meaningful improvements, such as being able to detect when an object was present, determine where it was located and ‌reach toward it accurately.

One severe adverse event occurred immediately ‌after injection and resolved within minutes, researchers reported in The New England Journal of Medicine.

While larger studies are needed to definitively prove the usefulness of ChrimsonR and the goggles, ⁠the findings show that even at a very advanced stage of blindness, surviving retinal ganglion cells can be made responsive to light and ⁠transmit visual information to the brain, the researchers said.

"Even in people with profound vision loss, the visual system retains a remarkable capacity to process new information," study leader Dr. José-Alain Sahel of the University of Pittsburgh said in a statement.

RADIATION EFFECTIVE AS SURGERY FOR LOCALIZED PROSTATE CANCER

Radiotherapy kept localized prostate cancer under control just as well as surgery, with fewer side effects, researchers reported at the recent American Society for Radiation Oncology, or ASTRO, meeting in Boston.

After a median of eight years of following patients, survival without recurrence was statistically similar. Of those who received five sessions of stereotactic body radiation therapy delivered over two weeks, 91% were alive without cancer recurrence versus 84% after surgery.

SBRT delivers the radiation directly to the prostate while limiting exposure to nearby healthy tissue.

The trial involved 123 patients, most with low- or ‌intermediate-risk cancers who were monitored for reappearance of a tumor or a rise in prostate-specific antigen levels, a potential indicator of prostate cancer.

The trial's original goal ​was to compare quality of life after the interventions. Efforts to enroll nearly twice as many participants were thwarted by the COVID pandemic, leaving the study with too few patients to answer that question.

Because it was not designed to establish noninferiority for cancer control, the trial cannot prove the treatments are equivalent for this outcome.

"What we can say is that... we have mature data showing excellent cancer outcomes with five-treatment radiotherapy," study leader Dr. Nicholas van As of The Royal Marsden NHS Foundation Trust in the UK said in a statement.

A separate analysis presented at ASTRO found that patient-reported sexual-function scores declined in both groups but remained significantly better after the radiation therapy. Median erectile-function scores on a 25-point scale, where higher scores indicate better function, were 19 with SBRT and 5 for prostatectomy after five years.

Also at five years, 8.3% of SBRT patients were using urinary pads for leaks, compared with 48% of surgery patients.

AVOIDING WHOLE-BRAIN RADIATION IN RARE LUNG CANCER

Patients with an uncommon, aggressive form of lung cancer who develop brain metastases can benefit ​from a safer radiotherapy technique that is already standard for common lung tumors, according to data from a study reported at ASTRO.

Stereotactic radiosurgery uses high-dose radiation beams to destroy brain tumors without surgical incisions and with minimal damage to nearby brain tissues.

It is already the preferred treatment ‌for brain metastases ‌from most other tumors. Small cell lung cancer ⁠has been an exception for fear that treating only visible tumors could leave cancer cells behind.

As a result, patients undergo whole-brain radiation, increasing their risks for memory loss and other cognitive declines.

In the trial, 151 patients received either stereotactic radiosurgery or a modern form of whole-brain radiation.

Patients lived a median of 17 months after focused stereotactic radiosurgery versus nine months following whole-brain radiotherapy.

The trial was designed to assess the length of time before patients experienced cognitive decline from each treatment method. The researchers found no significant difference, possibly because of protection afforded by the newer form of whole-brain radiation, they said.

Comparisons of cognitive outcomes may also have been complicated by fewer whole-brain radiation patients alive for later ‌follow-up.

While prolonged survival was not the trial's primary goal and ​cannot be proved by this study, the results show how far both approaches have come, principal investigator Dr. Vinai Gondi of ‌the Northwestern Medicine West Region and Proton Center in Warrenville, ⁠Illinois, said in a statement.

"For patients with small ​cell lung cancer, we now have two modern radiation therapy strategies to consider and randomized evidence to help us understand how best to use them," Gondi said.

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(Reporting by Nancy ​Lapid; Editing by Bill Berkrot)

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