SAN JOSE WOMAN FIGHTING FOR CUTTING-EDGE ALS DRUG
Legislation: Jamie Berry, diagnosed with Lou Gehrig’s disease in July, is lobbying the House to pass a bill that would accelerate development and approval of new treatment
When Jamie Berry noticed a slight limp in her right foot in late May, she didn’t think much of it. Berry tends horses and drives tractors on her family’s 80-acre ranch in San Jose’s Almaden Valley, and she was also busy with an 8-yearold daughter, five dogs and an excavation business she runs with her husband. So Berry ignored what she assumed was a minor sprain.
But after Berry limped for several weeks, her leg became visibly swollen. A trip to the emergency room led to a visit with a neurologist.
“And then boom,” said Berry, 45. “They club you over the head with the worst diagnosis possible.”
On July 14, she was diagnosed with amyotrophic lateral sclerosis, or ALS. Often referred to as Lou Gehrig’s disease, it breaks down the nerve cells that control muscle movements. As the disease progresses, patients have trouble walking, speaking and breathing. Most die within two to five years.
Berry, however, quickly saw a ray of hope. Hours after receiving her diagnosis, she watched an online video of a West Virginia man with ALS who saw his symptoms disappear after taking part in a clinical trial for an experimental treatment. But Berry was heartbroken when she learned that despite strong evidence that the treatment is effective, it doesn’t yet have approval from the U. S. Food and Drug Administration and thus remains out of reach for suffering ALS patients.
That’s why Berry and many other patients with the disease are lobbying for a bipartisan bill in the House of Representatives that would give more than 16,000 Americans with ALS access to cutting-edge treatments.
In the months or even years it can take to get FDA approval of a new treatment, Berry said, “a lot of people who are fighting this fight won’t be here. If I could get access to it now, or even in the next couple of months, it could be lifesaving.”
Two medications are now commonly used to treat ALS, each of which can extend a patient’s life by only two or three months. In recent years, though, numerous clinical trials have offered hope for a more effective treatment.
One of the most promising is Nurown, a treatment developed by New York-based Brainstorm Cell Therapeutics. The treatment relies on stem cells, which are cells that haven’t become specialized in function. The cells are collected from an ALS patient’s bone marrow and cultured in a lab to produce high levels of chemicals that help nerve cells stay healthy. The cells are then injected into the patient’s spinal canal.
Results of a Phase II clinical trial indicated that six months after receiving a Nurown transfusion, patients saw a significant reduction in how quickly their symptoms worsened. In addition, their bodies showed fewer chemical signatures of the disease.
“Patients seem to really benefit,” said Abla Creasey, vice president of therapeutics and strategic infrastructure at the California Institute of Regenerative Medicine, a taxpayer- supported organization that funds the Nurown clinical trials in California.
In the most recent Phase III trial, about 200 patients were given three stem cell infusions over a period of six months. Thurman Maynard, the West Virginia man whose interview Berry found online, was one of the participants.
Maynard received his last treatment just over a year ago. Because of the experimental nature of the trial, however, he still hasn’t been told whether he received Nurown or a placebo.
But before the trial, he could hardly open a bottle of Gatorade, Maynard said in a phone interview. Today, he’s regained nearly all his strength.
“I know where I was, where I went, and where I’m at now,” he said. “I don’t want to go back to where I was.”
Maynard has used some of his newfound strength to pour concrete pads outside his house, making it accessible for the wheelchair he may need in the future.
All the data for the Phase III clinical trial has been collected, and Brain-Storm expects to publish the results by the end of the month, Mary Kay Turner, vice president of patient advocacy and government affairs, said in an email.
“If our Phase III trial supports an application” to the FDA she said, “Brainstorm will do everything it can to make the therapy available as quickly as possible.”
Depending on the results of the Phase III trial, Creasey said, the FDA could approve Nurown or offer it “conditional approval,” al-lowing it to be given to patients while additional trials are done.
Turner said Brainstorm doesn’t have an estimated timeline for approval. But Creasey said she expects an FDA decision by early 2021.
The uncertainty is aggravating for Berry. In the nearly four months since her diagnosis, her limping foot has developed into a severely weakened leg. She also gets winded easily walking around the ranch.
“You can feel (the disease) creeping in on you,” she said. “The frustrating thing is there’s an answer, but we can’t have it.”
In 2016, Berry organized a Gofundme page for a friend with cancer who had lost his home and dog in a wildfire. Now she’s found herself on the other side of fundraising, after her friends raised more than $100,000 to fund her medical treatment.
But right now, no amount of money will gain her access to the treatment she believes offers the best chance of improving her condition.
Berry initially thought she could access Nurown through the Right to Try Act. Signed by President Donald Trump in 2018, the law is aimed at making promising experimental drugs and treatments available before they’re approved by the FDA.
But the new treatment would cost hundreds of thousands of dollars per patient, Creasey said. So rather than making Nurown accessible only to ALS patients wealthy enough to afford it, the company decided not to offer it to Right to Try patients at all. Brain-Storm, however, is negotiating with health insurers to cover the treatment if and when it gets FDA approval.
For now, Berry is throwing her support behind HR 8662, a bill by Rep. Jeff Fortenberry, R-nebraska. The bill would create a center led by the FDA and the National Institutes of Health dedicated to accelerating the development and approval of ALS treatments.
The bill replaces similar legislation Fortenberry introduced in June. It gained 198 cosponsors.
The new bill, introduced Oct. 23, would allocate $100 million over five years to fund research and help companies such as Brainstorm provide experimental treatments to patients outside of clinical trials.
Berry is hopeful she and her fellow activists will rally enough bipartisan support for the bill to send it to the House floor either in the current congressional session or the one that begins in January.
These days, Berry hardly sleeps. Instead, she spends her nights firing off impassioned tweets and emails to politicians and strategizing with fellow ALS advocates.
A lthough she understands why the FDA’S process is necessary, she wryly summarized her own mindset as a terminally ill patient.
“What do we have to lose? If we get (the treatment) and we die, we were going to die anyway,” she said.
Two medications are now commonly used to treat ALS, each of which can extend a patient’s life by only two or three months. In recent years, though, numerous clinical trials have offered hope for a more effective treatment.