Using stem cells to reverse vision loss

Should stem stells be regulated as drugs? The FDA will hold hearings this year to explore stem cell treatments it deems unproven.

“I went from legally blind to legal-to-drive in eight weeks,” says Doug Oliver, 54, a Nashville, Tenn., man whose vision loss was reversed by experimental stem-cell surgery in 2015. The stem cells used were extracted from his own bone marrow. Oliver was born with good eyesight, but due to a hereditary condition, over a period of 11 years he gradually lost much of his vision. Doctors eventually described his condition as “incurable.”

Incurable is a four-syllable word that changes lives. Oliver hopes to speak publicly about that word when the Federal Drug Administration holds hearings this year to explore stem cell treatments it deems unproven and untested. Oliver’s treatment and subsequent vision recovery could be defined as “unproven” by the FDA, although he believes he is living proof.

The agency has signaled it may begin to classify stem cells as “drugs.” That would give it the power to disallow clinics from advertising stem cell treatments for an array of medical conditions. Many clinics nationwide do offer costly stem cell therapies to treat a variety of conditions, and the FDA might start viewing these clinics somewhat like snake-oil peddlers of the past, supported by too little scientific evidence to merit the label “medicine.”

The FDA recently issued “draft guidelines” on the use of stem cells. A hearing was scheduled for April 13 to help finalize the agency’s policy going forward, and to decide if stem cells are “drugs.” FDA approval of drugs requires evidence they are safe and effective, something that historically has cost drug companies millions of dollars and many years of clinical trials to achieve.

However, due to an outpouring of concern by numerous parties the hearing has been delayed, according to an FDA announcement, until “later this year”:

“There has been considerable interest in the Public Hearing, ‘Request for Comments – Draft Guidances Relating to the Regulation of Human Cells, Tissues or Cellular or Tissue-Based Products,’ scheduled to take place on April 13, 2016. Due to this interest (the FDA) . . . has postponed the hearing, which will be rescheduled later this year at a date to be determined.”

It cannot have escaped notice that “later this year” coincides with a national election, and academic research on stem cells could be impacted by FDA requirements. Current medical research into stem cells in the U.S is privately funded or state-funded, as monies and research grants are not available from federal sources. Because the FDA has not approved stem cell treatment, there is no federal funding to study it.

A short, incomplete list of U.S. institutions using private donations, their individual states’ grants, or other sources to study stem cells does not lack gravitas: Harvard Stem Cell Institute; Yale Stem Cell Center; University of Michigan Medical Research Institute Consortium for Stem Cell Therapies; University of California Santa Barbara Center for Stem Cell Biology and Engineering; University of Minnesota Stem Cell Institute; University of Colorado Center for Regenerative Medicine and Stem Cell Biology; Stanford University School of Medicine/Institute for Cancer/Stem Cell Biology and Medicine; Tulane Center for Stem Cell Research and Regenerative Medicine; University of Miami School of Medicine Interdisciplinary Stem Cell Institute — the list goes on to include dozens more.

During his 2012 TED Talk, Dr. Dennis Clegg, director of the University of California Santa Barbara’s Center for Stem Cell Biology and Engineering, and Co-director of the California Project to Cure Blindness, discussed animals that can regrow parts of themselves. One is the newt, a lizard-like animal related to salamanders, and another is planaria, a type of water worm. If a newt’s leg or foot is cut off, the missing limb regrows. After awhile, the newt sports a new leg. Planaria worms can be cut into pieces and each piece will regrow itself into a new worm. Clegg points out that humans have never been able to do this — we cannot regrow a new body part if it’s cut off. Our skin can grow scars after wounding, but we cannot regrow a finger, a toe or an arm, nor can we regenerate cells inside our eyeballs after they fail.

However, modern research has shown that stem cells found inside our bodies — in bone marrow, for example — can and do adapt and grow into new cells. They can be extracted from bone marrow, inserted into another location in the body, adapt to what needs to be healed in the new location, and then regrow into new healthy cells. This is what seems to have happened when stem cells were extracted from Doug Oliver’s bone marrow and inserted inside his eyes.

Plant stems spawned the label “stem” cells. Although tiny, a stem holds within it the potential to sprout flowers, vegetables, leaves, branches, fruit, vines, thorns or huge trees. Likewise a microscopic human stem cell contains the potential to branch out and regenerate damaged cells, which then build different kinds of body parts.

Oliver’s own reversal of his 11-year vision loss cemented his perspective on research: He believes the FDA’s actions should not stop clinical trials. FDA curbs need not spill over nor seep into ongoing research or trial surgeries. Oliver sees no reason to limit experimental surgeries like his own. FDA approval can take years, meanwhile, people whose vision could be restored, like his was, live in darkness right now, day after day, unable to see their loved one or friends, to earn a living or to care for themselves.

The very first time he discovered, while exploring online, stories about surgery to reverse sight-shrinking macular degeneration like his, Oliver didn’t believe it would apply to him. “Every time I saw anything that might help treat macular degeneration, it was restricted to age-related macular degeneration. I have one of the rarest kinds in the country, not age-related, so I was always disqualified.”

His doctor had communicated to him in no uncertain terms: “There was no treatment or cure for my disease, and I could expect to become legally blind,” Oliver said. “He advised me to check in with every few months to see if anything came up. That was it.

“I remember feeling like I was physically alone in the room. But I returned home and for two years did what he had suggested.”

One day his patience paid off. He discovered the Stem Cell Ophthalmology Treatment Study, or SCOTS, which is registered with the U.S. National Institutes of Health as one of stem cells’ few experimental “clinical trials.” Participating SCOTS surgeons were treating inherited eye diseases. “I called my wife Ann over immediately and showed her,” Oliver says. “Our first response was apprehension about the cost, but we acted like we were unwrapping presents.”

SCOTS uses stem cells taken from patients’ own bone marrow. “Bone marrow is the most-actively dividing cell in the body,” SCOTS director Dr. Steven Levy explains. “More than any other (human) tissue, bone marrow has regenerative processes.” Bone marrow’s regular job is to continuously replicate. Its more familiar purpose is fueling the growth of things like platelets and red blood cells when the body needs more of them.

Although SCOTS is registered with the National Institutes of Health, insurance does not cover treatments that lack FDA approval. So SCOTS’ experimental trial surgeries are patient-funded, meaning patients pay to participate. Because they are patient-funded these trial surgeries have so far been free from FDA bureaucracy. Oliver raised money for his own surgery with a GoFundMe campaign.

Following his surgery, Oliver could clearly see his grandchildrens’ faces for the first time in years. Recently, he wrote a letter to one of his state’s senators, Sen. Lamar Alexander (R-TN), a ranking member of the Senate Health Committee, about why he wants to be registered to speak at this year’s FDA hearing. His letter to Alexander highlights his personal story:

  • “On August 15 of last year, I received bone-marrow (my own tissue) stem cell treatments for an incurable form of Macular Degeneration called Malattia Leventinese. This rare, inherited form of the disease has had no treatment or cure.”
  • “Before surgery, I was legally blind. My visual acuity was 20/2000 in my left eye, 20/400 in my right. Now I am 20/40 in my left eye and 20/30 in my right, and received my Tennessee driver’s license in December, eleven years after surrendering it to blindness.”
  • “I am writing to ask you if there would be any way I could speak to you or the Health Committee about what I believe the impact this stem cell treatment had on my life, and my feelings about FDA intentions to limit stem cell treatment…”
  • “(This) could become overreach by the FDA in regulating stem cell regenerative medicine practice. … I believe it would shut down a vitally important arm of clinical research in this country.”
  • “It would ‘sentence’ so many currently blind or near-blind people to an unnecessary, sightless future, when I am proof there is much still to be clinically discovered and treatments still to be clinically developed with patient self-determination and their informed consent.”
  • “Admittedly, targeted, reasonable regulation of shoddy, misleading stem cell practice is needed, but this expansive level of FDA power, that would suffocate ingenuity the nation needs, is not.”

One day after his August 2015 surgery, the unexpected twists in his journey began. “The first thing I noticed was while riding in the car the day after surgery. We were riding in the car and I was looking straight ahead. I saw Ann at the steering wheel lift her hand and rub her cheek. I was so startled, I jumped and shouted ‘Ann!’ She pulled over quickly and said ‘What? What's wrong?’ I replied ‘I just saw your hand touch your face!’ It was then I realized I could see peripherally out of my left eye. Something that I hadn't been able to do for about 10 years.

“The next day, my right side peripheral vision returned, and I could see depth. I came out of the doctor's office and saw the crisp sparkle of cars in the sun parked in the parking lot. And the leaves on the trees. I started counting them.”

This was not the first time that SCOTS’ director Levy had heard such stories. “We’ve seen regeneration during (post-surgical) exam of the retina,” Levy says of SCOTS’ clinical trial surgeries where patients regained vision. “We’ve seen reconstitution of the retina, thickening of the nerve. We see cellular regrowth.” Regrown cells in the eye change everything for a blind person.

Oliver notes that he’s had to go through the same emotional process he did when he was losing his vision 11 years ago. “Relationships change and have to be renegotiated,” he says. “Self-confidence changes. I've become a better person having been vision-impaired. I find I have to work to keep those traits. Mostly patience and gratitude.”

His intention to travel to, and hopefully speak publicly at, the FDA hearing is part of his current effort to bring other blind people out of the darkness.

The World Health Organization estimates 285 million people globally are vision-impaired. The future of stem cell treatments in the U.S. is unclear given the recent FDA actions. If, following this year’s hearing, the agency decides that stem cell treatments are “drugs” — which the FDA must approve before they can be sold — the NIH’s SCOTS program could be impacted and individual doctors might not be able to perform the surgery that so changed Oliver’s life.

Questions arise about the future involvement of giant pharmaceutical companies in testing and research: Would that put the cost out of reach for many blind patients? With regard to profits and methodology, the pharmaceutical industry does not have a problem-free track record. Hundreds of millions of vision-impaired people worldwide translates into potentially mountainous profit.

Doug Oliver, “incurable” for 11 years, has unclouded vision about that future: “I want these treatment trials to be available sooner and to more people who have no hope of treatment in the near future for blindness.” He makes that crystal clear.

© 2016 by Kathy Jean Schultz

Twitter: @kjschul