When Lisa Wittenberg got a van in April that could accommodate her wheelchair, she got her nails done, ate at In-N-Out Burger and shopped at Target.
Three things she loved to do before ALS.
“That van is the biggest piece of (expletive) we’ve ever bought, but it’s been a life-changer,” she said with a grin, lying on a bed at UCI Medical Center on a recent morning.
The 46-year-old mother of two was diagnosed in August 2017 with Amyotrophic lateral sclerosis, or ALS. Also known as Lou Gehrig’s disease, ALS is a disorder of the nervous system that affects nerve cells and voluntary muscle movement.
Given two to five years to live, Wittenberg, mother of two boys — 17 and 13 — signed up soon after she was diagnosed to participate in a clinical trial called Brainstorm.
It’s a treatment that involves extracting patients’ stem cells, treating them with NurOwn, an experimental cell therapy, and injecting them back into the patient’s body.
It’s also a “random, double-blind and placebo-controlled,” which means half of the patients will not get the actual treatment, but a placebo — a substance that would have no therapeutic effect.
“Lisa’s Squad”
Each patient enrolled in the study gets three injections over 28 weeks and Wittenberg was at the hospital to receive her second injection April 24.
Darlene Van Dusen, Wittenberg’s mother, drives from Indio to Orange County every Wednesday to care for her daughter. And she is counting on this trial to help her daughter be there a little longer for her two grandsons.
At the hospital on the morning of her daughter’s second treatment, Van Dusen wore a pink t-shirt with the words “Lisa’s Squad” on it written around a sunflower, the official symbol of hope for ALS patients.
“Lisa’s always been a jokester, from the time she was little,” Van Dusen said. “This year, after daylight savings time, she joked about wanting that hour back. This is always how she’s been.”
“People like me shouldn’t have to lose an hour,” Wittenberg told her mom. “It’s not fair.”
Her daughter’s diagnosis caught her completely off guard, Van Dusen said. She didn’t know anyone who had ALS. She had to look it up online.
Van Dusen choked up as she remembered her daughter’s life until about a year ago when the downward slide of ALS upended her world.
Before she was stricken with ALS, Wittenberg was “active and fit,” hitting the gym and boot camps regularly, going on hikes and camping with her family, her mother said.
Van Dusen is pinning her hopes on this clinical trial, which is in phase three. That’s one step before it gets a stamp of approval from the Food and Drug Administration, after which it could become widely available.
“This has been the most promising trial so far,” she said.
“This trial means everything to us…to me.”
Is the treatment working?
When Wittenberg visited Dr. Namita Goyal, UCI Health neurologist and ALS specialist on April 2, after her first injection, she felt good. She was able to kick out her right foot, which she hadn’t been able to do before.
But the signs of improvement Wittenberg had been hoping for have faded since. She now believes her ALS symptoms are progressing faster.
Wittenberg said she can’t do some of the things she was able to when the clinical trial began.
She now struggles to hold a glass of water in her left hand — something she was able to do a month ago. At restaurants she asks for a plastic kids’ cup or a to-go cup.
Her breathing has declined. Her voice is feebler and the words come out much slower than before.
When she came down with a cold recently, she was unable to cough because the muscles of her diaphragm had considerably weakened.
“I’m 99 percent sure that I’m on a placebo,” Wittenberg said.
And she probably wouldn’t even know until after the trial ends in July 2019 as to whether she got a placebo or the real thing.
The idea she might be getting a drug that just might not do the job has been a source of significant frustration and anxiety for Wittenberg.
In May, she attended a three-day ALS Association Advocacy Conference in Washington D.C., where she got an opportunity to speak up about right-to-try laws, which allows terminally ill patients access to potentially lifesaving drugs before they are approved by the FDA.
President Trump on May 30 signed a bill into law allowing this option for terminally ill patients amid protests from opponents that such a law would curtail the FDA’s regulatory authority and leave patients open to financial exploitation.
During her trip, Wittenberg also spoke about the use of placebos in clinical trials.
“Why should there be a placebo?” she said. “I know I might be in a group that has gotten a placebo and I’ve come to terms with that. But what I’m hoping is that I have the right to try this drug after I’m done with this trial and after I’ve fulfilled my obligation.”
Anxiety and frustration
Participation in clinical trials offers hope for patients particularly those with ALS, said Debbie Wilson of Porterville whose brother Mark Harrison is also participating in Brainstorm at UCI Health.
But, she added, it can be a rough, emotional and devastating journey.
Wilson says ALS runs in her family. Her mother, aunt (mother’s sister) and grandmother all had ALS. Now, she is watching her brother suffer.
Things started well for Harrison. An ABC 30 video last month showed Harrison standing up and walking days after getting his first stem cell injection. It filled other patients, including Wittenberg, with hope and awe.
But Wilson now believes her brother could have been experiencing the “placebo effect.” Since then, she says, her brother’s condition has deteriorated.
“When he got on this trial, he walked in to UCI with a cane,” she said. “When he started the treatments, he was in a wheelchair. Now he is bedridden and needs assistance to breathe.
“He can barely talk.”
The family is trying to stay positive, but this has been a devastating few weeks, Wilson said.
“We don’t know if he’s getting the stem cell or the placebo and that’s extremely frustrating because you’re still going through all the inconvenience — the trips to the hospital, the spinal taps, the pain, the discomfort,” she said.
Like Wittenberg, Wilson wonders why ALS patients can’t get the real thing if a drug is in phase three, which means it’s already been deemed safe and effective.
“The thing is,” she said, “people with ALS don’t have time.”
Bound by regulations
Goyal, who is not just Wittenberg’s doctor but also a lead researcher for the clinical trial, says she understands her patients’ frustrations. But, she also cautions them about arriving at premature conclusions.
“The most important point to remember is that in the phase two study, while patients may not have noticed striking improvement in strength, the disease progression had slowed down when compared to the placebo group,” she said.
Wittenberg may be losing function, but the rate of loss might be slower, Goyal said.
“We just don’t know until the study is completed,” she said. “Maybe this treatment is not going to be a cure. But finding an effective therapy that significantly slows down decline would still be a remarkable win.”
While Wittenberg won’t qualify for this trial again, she will have an opportunity to participate in one of several other drug trials, which are just about to start up at UC Irvine, Goyal said.
Then again, almost all ALS trials and most clinical trials are required by the FDA to be placebo-controlled, she said.
So, for ALS patients like Wittenberg or Harrison, the feeling is akin to getting off one rollercoaster and getting on another.
But, in the world of clinical trials, placebos are sacred because researchers believe they help them understand the effect of the drug that is being studied on a particular disease.
“Trials are designed based on the best way to find out if the drug works,” said FDA spokeswoman Sandy Walsh. “Every clinical trial is carefully designed to answer certain research questions. Products are often tested to see how they compare with standard treatments, or to no treatment.”
Clinical trials also try to show difference between treatments, she said.
“The likelihood that a difference will be seen between treatment and control is greatest with a placebo treatment,” Walsh said. “Placebo-controlled trials also provide the maximum ability to distinguish adverse effects caused by a drug from those resulting from underlying disease.”
Holding on to hope
For now, Wittenberg takes a lot of comfort in her support group of family members and friends — particularly her best friend Marie Van Raes who cares for her once a week.
On a recent Tuesday, Van Raes was helping straighten her friend’s hair.
“What makes it harder for me is Lisa is so positive and bubbly all the time,” Van Raes said. “I try not to think too much about the future and enjoy this time with my friend.”
When Wittenberg was first diagnosed, she got multiple offers from friends who wanted to help. While she appreciated those offers, Wittenberg says most people don’t fully comprehend what it takes to assist someone with ALS.
“They have to be able to take me to the restroom because I absolutely cannot move on my own.”
The only way Wittenberg can get around in her home is with the Hoyer lift, a hydraulic lift with a pad attached to chains that can carry patients and place them where they need to be placed.
So, Wittenberg only relies on a close circle of trusted friends and family members.
They also help her get through difficult times such as right now — when she is waiting to find out what next. She has already discussed several options with Goyal.
It’ll be another clinical trial. And, she could get the real thing, or the placebo.
“But, I don’t have a choice, I have to do it,” Wittenberg said. “In six months, I won’t even qualify to be on a trial. Time’s running out and this is my only hope.”
This is the second story in an occasional series that will follow Lisa Wittenberg as she participates in an ALS clinical trial at UCI Health.
About the clinical trial
Brainstorm is still looking for ALS patients who might qualify for this clinical trial, which is expected to end in July 2019. Information: 949-824-3990, email stemcell@uci.edu or visit brainstorm-cell.com.
