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Hope in a handful of ‘magic’ cells

Lupus expert explains beneficial effect of cells with potential life-changing implications 

Lupus patient and friends
Lashanda Wiggins, center, took part in phase 1 of a clinical trial testing a possible treatment for lupus. Photo provided
Andrew Lipke | adamshel@musc.edu | August 15, 2018

“Was there ever a time I thought ‘I’ll never get better?” Lashanda Wiggins asked pensively. “Like this is the rest of my life? Yes. Every day before April 27, 2017. Every single day. But this treatment, Dr. Gilkeson and MUSC gave me my life back.” 

There are times when life is overshadowed by elements outside our control. For Wiggins, that included a devastating diagnosis of lupus. But thanks to a clinical trial led by MUSC’s Gary Gilkeson, M.D., and Diane Kamen, M.D., there is hope on the horizon — not just for Wiggins but perhaps lupus patients everywhere. 
 
For many mothers, taking a newborn home from the hospital is exciting and feels like the final step of a 10-month journey. But for Wiggins, it signaled the beginning of a new road she would traverse — one so difficult, in fact, it would last for more than three years. 
 
It all began with back pain one morning in October of 2013. 
 
“The pain was so excruciating, I kind of laid on the floor and told my other son to watch the baby,” she explained. “He called his dad, he was only 3, but we had pictures in the phone. Then he called 911. They took me to the hospital by ambulance.” 
 
Dr. Gilkeson 
Dr. Gary Gilkeson 
After three successive visits to various hospitals in a period of 48 hours, Wiggins continued to experience immense discomfort. She was diagnosed with a bladder infection and sent home. Unfortunately, that wasn’t the problem. On her third trip to the hospital, a doctor discovered something disturbing. 
 
“They did MRIs, and they found lesions on my brain and on my spine. The doctor there said they weren’t equipped to handle it, so they brought me to MUSC, where doctors ended up diagnosing me.” 
 
Wiggins, a wife in her early 20s and mother of three, was told she had neuromyelitis optica, a manifestation of lupus. “Forty-eight hours later, I was paralyzed from the waist down, and I was blind 24 hours after that – all from lupus.”
 
Wiggins was terrified and began to fear not only for her life but that she might never be a real mother to her children again. 
 
“My nerves were kind of crazy,” she said with a laugh, “because my husband was at home with this new small baby who came home at 4 pounds, 4 ounces. My son was born prematurely at 34 weeks. So I’m scared, because he was scared to hold the baby in the hospital. I was just worried about my baby more than anything.”
 
Paralyzed from the waist down for six months and blind for three, she remained bedridden for more than six months while she slowly recovered her vision, then her mobility. MUSC became her home for three of those months. 
 
While her family continued to support her over a period of almost three years, she suffered nearly a dozen lupus flares, where her symptoms overwhelmingly increased. Systemic lupus is an autoimmune disease that causes the body’s immune cells — the defense system that usually protects against disease — to attack healthy organs and tissue. Lupus is not only more prevalent but also more severe in African-American and Hispanic women.
 
Wiggins' family searched desperately for any possible solution. 
 
Dr. Kamen 
Dr. Diane Kamen 
“My mom took a leave of absence from work for the three months while I was at MUSC. My dad worked especially hard on researching clinical trials. We even thought about going to Chicago to enroll in a bone marrow transplant trial, but when we learned that program has like a 15 percent mortality rate, we backed out really quick.”
 
After suffering through years of lupus flares, Wiggins was exhausted. Finally, in early 2017, she and her family learned of a study led by Chinese scientist Lingyun Sun, M.D., Ph.D., who used stem cell infusions to treat lupus flare-ups. Even more remarkably, a similar study was taking place at MUSC. Gilkeson, a renowned rheumatologist and lupus expert, and Kamen, a highly regarded lupus clinical trials expert, were using mesenchymal stem cells harvested from umbilical cords to treat the disease. Wiggins’ rheumatologist, Jim Oates, M.D., contacted Gilkeson and Kamen, the co-principal investigators of the study, and referred her. She was the first patient enrolled in the study, and in late April of 2017, she became part of a group of six patients being treated in the phase 1 trial to determine the safety of the treatment.

Magic cells

Gilkeson was intrigued by Sun’s work after visiting the Affiliated Drum Tower Hospital of Nanjing University Medical School in China in 2010. There he heard Sun present his work about the groundbreaking use of mesenchymal stem cells (MSCs) to treat lupus. That visit paid off in spades. The techniques for stem cell derivation were transferred to MUSC. 
 
After preclinical work at MUSC, funded by the Lupus Foundation of America (LFA), which demonstrated potential efficacy of the cells, the necessary FDA regulatory requirements for performing a phase I trial were completed. The results of the phase 1 trial, also funded by the LFA, are considered promising, with none of the patients having a serious adverse reaction and five of the six patients treated experiencing significant improvement in their lupus symptoms after receiving the therapy. 
 
Gilkeson and Kamen recently were awarded a $3.8 million grant from the Lupus Foundation of America and $4 million grant from the National Institutes of Health to perform a five-year phase 2 multicenter placebo-controlled trial, which will allow them and their team to determine the efficacy of MSCs in patients with lupus resistant to treatment. MUSC, through the office of the provost and College of Medicine dean’s office, is providing an additional half-million dollars for the trial.
 
Gilkeson described the potential functions of the stem cells used in phase I of the trial. “Their job is, if somebody breaks a bone or gets a cut or any kind of injury, then these cells are mobilized out of the bone marrow, and they go to the place of injury. But it was recognized a decade or so ago that they also have potential immune effects.” 
 
While MSCs may be derived from bone marrow, his team uses cells that are derived from the umbilical cords of women who have delivered via caesarean sections at MUSC and consented to helping with the study. The cells are processed at MUSC’s Center for Cellular Therapy, which houses an FDA-registered cGMC facility. There are only 15 such clean-cell facilities in the United States. MUSC will provide the MSCs for all seven medical facilities involved with the second phase of the  trial.
 
In phase 1, all six patients knew they were receiving the cells. 
 
“In our phase 1 trial, five out of the six patients met improvement goals, but again, we all knew they were getting ‘magic’ cells. They knew they were getting magic cells. The nurses knew they were getting magic cells. So everyone was hoping they’d improve.” 
 
He explained the placebo effect: a phenomenon where patients improve by sheer belief and optimism, not necessarily due to the therapeutic value of the treatments. The placebo effect was as high as 30 percent in other lupus clinical trials. In studies of the patients’ immune systems during the trial, Gilkeson and colleagues at MUSC and Emory were able to show significant changes that paralleled their clinical improvement. 
 
Lashanda Wiggins and her family 
Lashanda Wiggins, far right, has graduated from cosmetology school 
Phase 1 revealed that the treatment is safe and promising, but Gilkeson said phase 2 will define efficacy, and possibly show how the cells work, comparing patients that receive the cells to patients that receive a placebo infusion.  
 
Gilkeson described their ambitious plans for phase 2. 
 
“Everyone is going to continue their standard of care medications. We will enroll 81 participants in the trial; 54 of them will get the cells in one of two doses and 27 will get placebo.” 
 
Phase 2 of the project differs from the Chinese trial and phase 1, in two ways. The first is that this project is a double-blind study, meaning that all participants including the patients, nurses and physicians will be unaware who is receiving the cells versus who is receiving placebo. The second difference is the dose of the cells given. 
 
“In phase 1 and in the first cohort of patients for phase 2, we administered or will administer 1 million stem cells per kilo of body weight, but we’re going to be using 5 million cells per kilo of the patient’s weight for phase 2’s second cohort.” This dose escalation is likely to determine if dosage level affects the effectiveness of the treatment. 
 
Phase 2 will also delve more into how this treatment works, Gilkeson said. “It appears that the mesenchymal stem cells activate T-regulatory cells that serve to modulate the immune system and occur in lower amounts in some lupus patients. It also seems to decrease double negative B cells, a type of white blood cell that is increased in most lupus patients, to get them to a normal level.” 
 
Wiggins said regardless of exactly how the cells work, phase 1 yielded miraculous results for her. 
 
“It took about four weeks, and I noticed I had a lot more energy. I wasn’t as fatigued. Before I would take naps all throughout the day.” There was no doubt in her mind that the lupus she had long suffered with had been dealt a severe blow. “I got the infusion April 27, 2017, and I had my last flare in March of 2017. I haven’t had a flare since.”
 
She’s enthusiastic about the phase 2 trial, which will start enrolling patients in the next four weeks and is expected to last through June 2022, with six additional academic centers involved under the leadership of Gilkeson and Kamen.  
 
Kamen said the study recently took a large step forward with getting final approval from the NIH for the study protocol. “I’m involved in the protocol training. Actually, today was the day we trained the other six institutions participating in the trial.” 
 
With the groundwork well underway, they hope phase 2 will begin enrollment in August or early September.
 
For Wiggins, hope abounds, as she remains flare free for over a year. She recently graduated from cosmetology school and works part time at a salon and loves it. What is even more incredible, she said, is to be given back the gift of motherhood. 
 
“I can go outside and play with my kids with just sunscreen, and I’m fine. Before I’d just get dizzy and sick. I can’t express what it’s like to have my life back.” 
 
With her lupus under control, she expresses her gratitude to everyone involved in the study and MUSC. “MUSC is amazing. Any time my kids or I get sick, we always come here first. I feel that’s because MUSC is always studying and researching. They know a lot about a lot. Dr. Gilkeson gave me the floor to ask any questions I had and made me feel included. Honestly, he and his entire staff were amazing.”

MUSC researcher devotes career to study of lupus (MUSC Catalyst News, July 2, 2018)

MUSC leads research on potential lupus treatment (MUSC Catalyst News, Nov. 16, 2017) 

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