The $2 Drug That Millions of Patients Aren’t Being Told About

What people living with age-related macular degeneration (AMD) need to know

Black-and-white ketch of an eye with long lashes; tiny blue pills on the iris and a drug molecule on the pupil are the only source of color.
Illustration by Christi Williams for Elemental

“I couldn’t open or close my eyes without pain. It was like salt or shards of glass… all I could do was go to bed,” said 71-year-old Beth, who was diagnosed with age-related Macular Degeneration (AMD) in both eyes last year.

Beth is one of 11 million Americans — a number expected to double by 2050 — suffering from this leading cause of vision loss in the developed world. Once a month, every month, she undergoes the procedure recommended by physicians across the nation: a $3,000 Lucentis injection directly into each eyeball to prevent fluid buildup at the back of her eyes.

Ranibizumab (sold under the brand name Lucentis) is the leading treatment prescribed for Beth’s form of AMD along with several other eye diseases. It works by shutting down the growth of new blood vessels at the back of the eye, but it needs to be administered directly into the eyeball to do so. Beth doesn’t know that there’s an alternative treatment available — a pill used for Parkinson’s disease that costs just a few dollars a month and has little to no side effects. Her doctor, like most others with patients like Beth, hasn’t told her about it.

Why? One could argue because, in America, we face a harrowing reality where profits trump progress and science is silenced.

When any one of us visits our physician with a health concern and needs treatment, we reasonably expect that our well-being will be prioritized, but in a private health care system where significant earnings are to be had, how can we be sure that we’re receiving the best option for our physical, emotional, and financial health? Unless we’re closely following the emerging scientific literature, the answer, quite simply, is: we can’t.

When science isn’t profitable

University of Arizona professor of ophthalmology, vision science, and physiology Brian McKay, PhD, has been a pioneer in his field for the last 20 years. When he discovered that a Parkinson’s medication, levodopa, could effectively treat and prevent AMD, he had hoped that this gentler alternative to the injections would be welcomed with open arms by the world of medicine. Unfortunately, that has not been the case.

“Pharmaceutical companies have an agenda that doesn’t always meet science per se,” McKay said. “Scientists have an issue when they find something that isn’t profitable. They run into problems.”

McKay described experiences presenting his research at national scientific conferences and encountering visibly angry clinicians and pharmaceutical representatives that make millions of dollars from U.S. taxpayer dollars each year from these costly injections. Scientists like McKay are problem-solvers, but the solutions they provide don’t always mesh with corporate interests.

There are two types of AMD: wet and dry. Dry is caused by a loss of cells in the retina and this accounts for approximately 85% of AMD cases (about 9 million people.) There’s currently no available treatment for dry AMD, one of the reasons being that scientific trials for this type can take years to reach an end point. “We’re talking about eight to 10 years as opposed to four to six months for wet AMD treatments,” McKay said, though he’s confident that the research will catch up eventually.

In the meantime, vitamins C and E are often recommended.

Dry AMD can advance to the wet variety, like Beth has, which is characterized by leaky blood vessels in the back of the eye that accumulate fluid behind the retina. Though only 15% of AMD cases are wet, it accounts for 90% of the blindness caused.

Approximately 30% of Americans over the age of 70 are fighting against the clock to keep their vision. There’s a strong genetic component to developing AMD, though environmental triggers such as smoking and diet may also play a role.

Levodopa has been around as a Parkinson’s treatment since the 1960s and while long-term use in patients can result in some side effects such as movement disorders, there have been little to no side effects observed in AMD treatment populations.

The National Eye Institute found in 2010 that 89% of cases in the U.S. were found in white individuals, and 65% of cases in females. McKay began his research into AMD while on the faculty at Duke University before moving to the University of Arizona in 2002. While studying albinism, known to cause vision problems as it impairs retinal development, McKay found that levodopa played a key role in some of the mechanisms in the eye that allow us to see. From there, things only got more interesting.

A simpler solution?

Once at the University of Arizona and working alongside genetics researcher, Murray Brilliant, PhD, McKay began questioning whether older adults already taking levodopa for Parkinson’s disease were suffering from the same vision loss otherwise seen in the population at large.

Analysis of 87 million patients’ medical records revealed that AMD occurred significantly later in patients taking levodopa (age 79.4 as opposed to age 71.2 years). Furthermore, the chance of developing AMD was also significantly reduced. This major study was published in the American Journal of Medicine in 2016. “We looked at about a quarter of the country,” McKay explained, “and it was unbiased… perhaps the only bias coming from those who had access to ophthalmology care.”

Levodopa has been around as a Parkinson’s treatment since the 1960s and while long-term use in patients can result in some side effects such as movement disorders, there have been little to no side effects observed in AMD treatment populations.

McKay co-authored a double-blind study (meaning that neither the subjects nor the researchers knew who was receiving the treatment or placebo) published in 2014 that looked at the potential benefits of levodopa for improving vision in 45 albino participants. While there was no statistically significant improvement, the most promising finding was how few side effects were reported.

Approximately 10% of subjects reported headaches, while a handful experienced dry mouth or thirst over the 20-week study period. It’s important to mention that the trial included many children (the average age of the subjects was 14.5 years) and there was an even split of males and females.

Eyeball injections

The Lucentis treatment procedure, though completed in a matter of minutes, is far from ideal. Patients undergoing the injection can expect to first have their eyes cleaned upon arrival at the clinic, numbed via drops or an initial injection, and held open with a special device before finally the treatment is administered. Patients undergoing these injections typically report sensitivity in the form of aching or pressure in the eye for about 24 hours following the dose.

“That’s a big loss in productivity and lifestyle that has to occur every month,” McKay said.

The problems don’t end there; anxiety is fairly common with these injections. Studies have shown that even experienced patients still worry at the prospect of the treatment each month, while other findings link these injections to an increase in blood pressure.

Infection is also a big risk. The eye is avascular, meaning that the blood circulation we rely upon to shift pathogenic bacteria that might invade other parts of the body isn’t an option here.

Lastly, retinal detachment can occur if the needle used touches the back of the eye during an injection. When this happens, multiple surgeries are needed to repair the damage. If only a small part of the retina becomes detached, patients may not experience symptoms, but if treatment isn’t initiated right away, there can be a risk of permanent vision loss or blindness.

“It’s been six months and I’m not seeing any improvement. I feel like things are just getting worse,” said Beth, who is desperately clinging to her crafting hobbies that require keen sight among other things.

Beth lives in Portland and fears the day she’ll no longer be able to enjoy the beauty of the Pacific Northwest. “I’ve been told that I’ll be legally blind in about five years,” she said.

There was no mention of alternatives to Lucentis when Beth was initially diagnosed. Recalling the first time she had an injection, she explained that she had to sit in her car for two hours before she could see well enough to drive home safely.

When she returned a month later for her second treatment, the lasting effects were even worse. A lubricant is typically applied following the injection to reduce any gritty sensation or irritation. Inadequate application of this left Beth’s eyes burning. She spent the rest of the day in bed, unable to cope with the cutting feeling that accompanied each blink.

Beth has persevered for the last nine months despite being convinced that her vision is getting worse. She described being in the backseat of her daughter’s car and looking out the window at the ocean at dusk, convinced she saw fire coming from the water.

“I’m really struggling in the dark,” she said. One evening, she experienced a particularly frightening drive home to safety. “All I could do was focus on the white line on the road,” she said.

Whether it’s trying to navigate her neighborhood after dusk, or deciphering the text on her television screen in the evening, Beth’s declining vision begs the question: Why hasn’t she been told about levodopa by a specialist physician who should be up to date on the research in her field?

A doctor’s duty

“These decisions are being made financially and not for the care of the patient. I think we should try to remove that from the equation somehow,” McKay said. He supports the use of a once-a-day pill that costs just $0.08 a dose (that’s $2.40 a month). Factor in the cost of a doctor to prescribe it — which is often covered by a co-pay — and quarterly or six-monthly eye checks and the total bill is still leagues away from the $2,000–$3,000 a month that Lucentis costs.

A significant portion of this price tag is pocketed by the doctor and clinic while the majority goes to the manufacturer, Genentech, who first brought the medication onto the market 15 years ago. Medicare pays for most of the cost, though patients are typically left with a couple hundred dollars out of pocket each month.

Retinal surgeon and assistant professor of ophthalmology at the University of Massachusetts, Andrew Lam, wrote in a 2015 article in the Washington Post about the alarming trend among doctors to dose patients with Lucentis despite data showing that a $50 alternative, Avastin, works just as effectively.

Avastin (bevacizumab) was first approved by the FDA in 2004 as a treatment for colon cancer, though it is routinely administered intravitreally (into the eyeball) to AMD patients as an “off-label” drug (meaning that it is used for a purpose other than that for which it was originally approved). While it is almost on par with Lucentis in terms of its effectiveness, some studies have shown that it can cause a marginally higher number of adverse effects and may require slightly more frequent doses than Lucentis.

Lam explained that this might be why doctors opt for Lucentis, feeling a moral obligation to avoid the possibility of more side effects despite the staggering price difference.

How can doctors do no harm while also considering the impact of their decisions on the health care system as a whole? Is that even their job?

McKay explained that the annual cost to Medicare for AMD is approximately $8.2 billion. This is covered by Part B, which pays for doctors’ charges and professionally administered prescription drugs. U.S. taxpayers fund this and switching to levodopa as an alternative treatment has the power to redistribute billions of dollars that are needed elsewhere.

Getting the word out

What we’re seeing with AMD is a symptom of a problem that stretches far beyond eye health alone. As long as private doctors continue to place profits above progress while allowing critical scientific research to go under the public radar, patients will continue to be taken advantage of and denied the best treatments available. That’s where scientists themselves must step up communication to fill the void.

McKay was taught early on in his career that it’s a scientist’s job to engage the public. Asking the right questions and spending time at the bench gets the job done, but the findings are only relevant if there’s a population able to benefit from them.

As long as AMD patients continue to be kept in the dark, McKay will be out there, encouraging them to ask their doctors about levodopa as a possible “off-label” treatment for them. In the meantime, he hopes to investigate the racial bias that is so well-documented in this disease. “If there is indeed a racial basis, it should be confirmed,” he said.

In a study that’s the first of its kind, McKay will work with collaborators to compare eyeball donations from Black and white individuals to determine if there are any differences and what can be learned if so.

While the research moves into interesting landscapes, Beth and millions of others are being left behind with no choice but to trust their doctors’ costly decisions. Her diagnosis dictates that she’s got five years left to enjoy her sight. With a rapid decline in quality of vision this last year alone, she doesn’t know what lies ahead, but she’s sitting tight and hoping for an off-label prescription that offers more than just a monthly stab in the dark.

Arizona-based PhD student from Britain | Popularising science and writing about important things as well as silly things

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