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Menlo Park resident Carmen Caricchio is on a quest to cure childhood inherited retinal diseases, a group of rare conditions that cause the degeneration of the retina and can lead to vision impairment and blindness.
“It’s really sad, because when parents first find out about it, it’s usually around kindergarten to age 12, and it’s usually because the kids aren’t seeing well,” Caricchio said. “So a lot of times they go undiscovered, and then unfortunately, once you know you have it, there’s nothing you can do.”
Caricchio founded RareSight almost two years ago to address IRDs, which typically do not receive the same level of resources as other eye diseases.
Although she is not a doctor, Caricchio has worked in the business side of ophthalmology for 25 years.
“All along, I think it bothered me that I was hearing about these problems that kids were having, and I was always told no one will invest in that. ‘There’s just not enough money to invest in pediatric ophthalmology.’ And I was really annoyed by that,” Caricchio said.
She said pediatric treatments are often seen as less profitable because most children have normal eyesight and the potential customer base is smaller.
“Kids get the leftovers, they get pushed to the end of the line. I thought it’s backwards. Kids should get everything first,” Caricchio said.
Treating IRDs can be especially difficult because therapies must reach the back of the eye, where the retina is located.
“You can stop the disease from progressing, or you can modify the disease or slow the disease with these drugs that aren’t approved for these diseases but they may be approved for something else,” Caricchio said. “Why aren’t they already out there? Well, the reason is that it can’t really get to the back of your eye very well. So the amount of drug that you would have to pour on your eye would be like gallons because it’s pouring off your face and it’s not getting where it needs to be.”
“And if you take a systemic drug, which goes all the way through your whole body just to get to the back of your eye, you’re kind of poisoning your entire body,” Caricchio added.
However, there is one approved treatment for one type of inherited retinal disease. It uses gene therapy to replace a defective gene in the retina, but only a small number of patients qualify and the procedure is extremely costly.
“There is currently no approved treatment for any inherited retinal disease except for one gene therapy treatment for retinitis pigmentosa. It’s been out for 10 years but very few patients qualify, and it’s about an $800,000 procedure. It’s really early in development. We have many companies running after gene therapy as the big promise, and wouldn’t that be great, but nothing’s made it,” Caricchio said.
RareSight has a different approach: using implants to deliver existing medications to children’s eyes.
“We have a lot of new intravitreal treatments. They’re already used in clinics for older people, for things like age-related macular degeneration and diabetic macular edema,” Caricchio said. “In clinical trials today, they are taking these liquid treatments and turning them into little implants… And so that’s really far along for adults, but nothing for kids.”
Caricchio partnered with Australian company Polyactiva, which developed a drug-delivery system called PREZIA for eye treatments. PREZIA uses covalent bonds to attach a therapeutic agent to a polymer backbone and has already shown success in clinical trials with medications for older adults.
The challenge is that the drugs RareSight wants to use for IRD have very small molecules that tend to be metabolized quickly. Caricchio believes PREZIA can hold the medication and release it at the correct time.
Because the treatment uses existing medications and a delivery system that is already producing promising results, she hopes it will be fast-tracked by the Food and Drug Administration.
“We’re planning to be in humans in two years, which sounds like a long time, but in biotech and life sciences, it’s actually really fast. The reason we can do that is because we have decades of data already. We don’t have to prove to the FDA what these things are. We’re changing the administration from a pill or a drop to an implant,” Caricchio said.
IRD affects more than children’s eyesight; it can cause feelings of isolation. As a kid, Caricchio had a lazy eye that was corrected with glasses.
“I just have a little tiny sliver of an understanding of what it might be like for these kids,” Caricchio said. “As a kid, I hated swimming because the minute I took my glasses off, people would see my eye. I always felt uncomfortable at sleepovers and had to keep my glasses close.”
She said some kids with IRD feel dread about going to the doctor for fear of learning their condition is worsening.
“It must be really scary for them to hear that their eyes have gotten worse or are getting worse. They don’t usually hear that they are getting better… The anxiety, the feelings about your eyes, the feeling of looking different, having to sit at the front of the room — it really resonates with me,” Caricchio said. “If there’s a way that we can help them, it should be the priority.”
