By Juliette Goodrich & Molly McCrea
SAN FRANCISCO (CBS SF) -- Imagine being told you have just a few years left to live and a promising cutting-edge treatment exists that could extend your life, but no amount of money can buy it.
This scenario is not the plot of Hollywood movie. It is happening to several individuals who find themselves in this unfortunate predicament.
KPIX 5 was recently contacted by a woman who lives here in the Bay Area who is facing this dilemma.
Gwen Peterson moved to San Francisco 8 years ago from New England to begin a new job. Within weeks, she met the love of her life.
"They say we met the old-fashioned way - in a bar!" said Gwen, smiling.
Her husband Nate broke into laughter, nodding his head.
Gwen and Nate married in 2017. The young couple was looking forward to starting a family. But before they could celebrate their first wedding anniversary, Gwen started experiencing some unusual symptoms.
"My left foot was dragging and scuffing on the ground which led to dozens of falls, trips to the ER," noted Gwen.
Gwen would end up diagnosed with a progressive neurological disease called amyotrophic lateral sclerosis or ALS for short. It's commonly called Lou Gehrig's Disease.
"ALS is 100% fatal unfortunately. Patients usually live 2 to 5 years after their first symptom," said UCSF neurologist Dr. Catherine Lomen-Hoerth, who specializes in treating patients with ALS and is Gwen's physician.
ALS is a motor neuron disorder. It attacks nerve cells in the brain and spinal cord. It progressively robs people of their ability to move, speak, and ultimately breathe.
"The first thought is 'Well, what do we do about this? What's our action? How do we get treatment for this?', recollected Nate.
Nate and Gwen then heard how the treatment includes only 2 FDA approved drugs that show modest benefit.
"It's hard so being 32 years old, diagnosed with a disease that's still a death sentence and not want to fight. I immediately wanted to start fighting," remembered Gwen.
The clock was ticking. Gwen had to choose between getting pregnant or joining a clinical trial. If they qualify for a trial, ALS patients only have time to participate in one trial. When they progress too far, these trials are commonly out of reach for them.
"It was an extremely difficult decision," said Gwen.
She picked the trial and enrolled in a study that uses mesenchymal stem cells, harvested from the bone marrow taken from a hip bone in her body.
Mesenchymal stem cells are multipotent adult stem cells
These cells are cultured in a lab to produce a substance that may help nerves stay healthy. The experimental therapy is called NurOwn. It's developed by a biotech company called BrainStorm. The therapy is a biologic which in Gwen's case was delivered via lumbar puncture.
The hope is that the therapy would slow or prevent the progression of the disease. Gwen had a 50-50 chance of getting the actual unproven therapy. She may have also gotten a placebo. But she is certain she got the real thing.
Gwen told KPIX 5 that it worked for her.
"I have no doubt that I have stabilized," said Gwen.
Dr. Lomen-Hoerth was not involved in the study and told KPIX 5 that the trial has not yet made public who got the real agent and who got placebo. But she said without a doubt, her patients really need better treatments. She would also like patients to gain more access to promising unproven therapies.
"It'd be fantastic if patients could have more access to these drugs because the requirement s in order to enroll in a clinical trial are very strict," said the UCSF neurologist.
The trial ended. But the FDA stated the results do not help a majority of patients.
Those running the trial believe there was a benefit for a smaller group of those enrolled. The therapy remains unapproved.
At the end of the trial, Gwen was eligible to receive only 3 more doses of the experimental therapy. Her last treatment was in July. Now it is out of her reach.
"When an individual has this death sentence, I believe they should have access to drug that shows promise," said Representative Anna Eshoo, who represents California's 18th Congressional District that includes parts of Santa Clara, San Mateo, and Santa Cruz counties.
On July 29th, the Congresswoman recently chaired a subcommittee hearing on neurodegenerative diseases. Her office was contacted by another ALS patient named Jamie Berry from the South Bay.
"People often say to me, you don't seem to have ALS because it hasn't affected my speech or my breathing yet. but you know I'm in a wheelchair I can't walk, and my right hand is crippled," Jamie told KPIX 5.
Jamie Berry explains how people can help:
The panel heard from patients urging lawmakers to ensure the FDA act with greater speed and flexibility
They also want companies to make it easier for patients to gain access more quickly to promising unapproved therapies.
"When you are suffering and every single day your body is dying, then I think patients deserve dosages of hope," Congresswoman Eshoo told KPIX 5.
50 million Americans are affected by neurological disorders such as ALS, Alzheimer's Disease, Huntington's Disease, and Parkinson's Disease.
Eshoo told the panel how treatments for these conditions take on average 50% longer to reach approval. But these patients don't have that kind of time.
"We have more treatments in the pipeline than ever. However, we need access," said Gwen.
"Every day I see the challenges of this disease and I know it's going to get worse It is frightening. And so, anything I can do to help keep her with me and be my best friend is worth fighting for," added Nate.
One way they're fighting: these patients and their families have joined "I am ALS". The group was created by patients for patients. Members are seeking to accelerate the development of drugs and also to make promising therapies more quickly accessible.
There are two bills now in front of lawmakers and they've received bipartisan support. But during the pandemic, these laws may get lost in the shuffle.
These patients are urging everyone to take a moment and contact their federal representatives and urge them to act before October. If not, the bills may get passed to the next session, and these patients don't have that kind of time.
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