Page 12 - Envision January-February 2020

Cover Story

V ISION S CIENCE R E SE AR CH

NEW GENE THERAPY COULD REVOLUTIONIZE WET
AMD TREATMENT
Imagine an eye damaged by wet age-related
“ The goal macular degeneration (AMD) that makes Nineteen ocular adverse effects related to
its own medicine! A promising new gene
ocular inflammation potentially associated
is a potentially therapy for treating the condition could do with ADVM-022 administration were
just that by replacing regular intravitreal
one-and-done injections with a “one-and-done” treatment. observed in the cohort: 14 were classified
as mild and five as moderate. The
treatment. You may In the ongoing OPTIC phase 1 clinical trial inflammation responded well to topical
steroid drops.
occasionally need a for ADVM-022 (Adverum Biotechnologies),
six patients with wet AMD went 34 weeks
booster but this gene after receiving one intravitreal injection Patients maintained BCVA (best corrected
visual acuity) from baseline, experiencing
therapy could without needing rescue injections to a mean two-letter loss, and had a mean
control the disease. Patients with wet AMD
52.7 µm improvement in central subfield
theoretically last a typically require anti-VEGF treatment thickness.
“ “This is potentially paradigm-shifting,” “The follow-up, which included a median
every four to six weeks.
lifetime.
of 32 weeks, proved that the visual acuity
said lead researcher, Szilard Kiss, M.D.,
director of Clinical Research and Chief fluctuations are just fluctuations in a
small number of patients with long-term
of the Retina Service in the Department wet AMD,” said Dr. Kiss. “None are
of Ophthalmology at Weill Cornell attributable to any wet AMD disease
Medical College in New York City. “It’s not activity.”
science fiction—gene therapy for AMD is
becoming a clinical reality.” AMD is a degenerative eye disease that
occurs when part of the retina is damaged
Dr. Kiss’s research, which was presented at by leaky blood vessels that form behind
rd
the 123 annual meeting of the American the retina. This causes scarring and kills
Academy of Ophthalmology (AAO), is off retinal cells, leading to a loss of central
focused on developing a gene therapy that vision. AMD is the leading cause of vision
allows the eye to make its own anti-VEGF loss and blindness in North Americans
medicine. Ideally, it would be administered over the age of 50 and its incidence is
not through a surgical procedure in an expected to soar as the population ages.
operating room, but via an injection into
the eye in the doctor’s office. Most patients with AMD must visit their
ophthalmologist’s office every four to eight
Dr. Kiss and his colleagues developed a weeks for an anti-VEGF injection directly
next-generation vector that can insert into their eye (often both eyes). It can be
into the eye’s cells the genetic material that difficult to maintain this schedule for many
makes a molecule similar to aflibercept elderly patients, who must deal with other
(Regeneron Pharmaceuticals), which is maladies and rely on friends and family to
widely used in anti-VEGF medicine. Once take them to their appointments.
inside the cells, the DNA sequence begins
making the aflibercept protein. Dr. Kiss believes the new AMD therapy
could be available within three to
“Instead of taking a vial of aflibercept and five years, depending upon the results of
injecting it into the eye, your eye makes the further clinical studies and regulatory
aflibercept,” Dr. Kiss explained. “The goal approvals.
is a potentially one-and-done treatment.
You may occasionally need a booster but
this gene therapy could theoretically last a
lifetime.”

10 / JANUARY—FEBRUARY 2020 / www.ENVISIONMAGAZINE.ca /