Eric Weinbrenner is in the fight of his life as he and thousands of advocates seek to bring compassion to the data-driven scientific process of approving drugs meanwhile ALS patients die daily waiting for solutions to be found.
Imagine having worked hard to build a good life for the young family you’ve created; a beautiful home, small children --- the whole nine yards.
Then, suddenly, one sunny Arizona day just like any other, you’re told that due to a deadly disease, which has no cure, you won’t live to see the fruits of your labor blossom.
Life as you know it has been robbed in the blink of an eye.
The likelihood of seeing your two babies, a newborn and a 2-year-old, graduate high school and college is gone. Growing old with your wife is only a dream now.
This heartbreaking scenario is reality for Town of Paradise Valley resident Eric Weinbrenner, who was told he has amyotrophic lateral sclerosis --- also known as ALS or Lou Gehrig’s disease --- the day before Thanksgiving in 2019.
ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. In this disease, nerve cells break down, which reduces functionality in the muscles they supply. The cause is unknown.
People with ALS lose their functions to walk, talk, and eventually breathe. Data shows about 16,000 people across the United States have ALS at any one time. Half of all people affected with ALS live at least three or more years after diagnosis; 20% live five years or more; and up to 10% will live more than 10 years, according to the ALS Association.
Mr. Weinbrenner says the diagnosis is much more grim.
As his day-to-day life changes rapidly, Mr. Weinbrenner is racing the clock to help not only himself but the thousands of others who are diagnosed with ALS.
While creating a nonprofit organization to raise awareness and funds to ease the financial burden of those diagnosed with ALS, Mr. Weinbrenner joins others in a fight to see drugs with positive results fast-tracked to prolong lives.
NurOwn is a stem cell drug at the forefront of this fight.
Proponents believe NurOwn has the ability to slowdown the toll of ALS and prolong life for those with the disease. However, the process for a drug to receive approval from the US Food and Drug Administration is a long road, which for some, is not moving fast enough.
Clinical trials follow a typical series from early, small-scale, Phase 1 studies to late-stage, large-scale, Phase 3 studies. NurOwn is in Phase 3, and has been going through the FDA’s trial phases for upwards of a decade, according to Mr. Weinbrenner and his wife, Jen.
“The big problem is there is a ‘Right to Try’ Act that the government implemented; however, there is a hold up from the drug companies that cannot mass produce a drug in Phase 3 trial for all the ALS patients that want the ‘Right to Try,’” Mrs. Weinbrenner explained. “Specifically, NurOwn by BrainStorm is currently handmade in a lab for every trial patient. Until it is approved by the FDA, they will not be able to support the cost to get this product to all the ALS patients that need the treatment.”
While drugs related to the coronavirus are getting fast-tracked through the FDA process this spring, the Weinbrenners and others are pleading with federal officials for NurOwn to get the attention it deserves as 100% of the people diagnosed with ALS will die --- a statistic much higher than people infected with coronavirus.
“Once data is released end of this year from Phase 3 we don’t have another six months or a year for the FDA to take their sweet time to approve it,” Mrs. Weinbrenner said. “There needs to be legal authority over the FDA, which maybe Congress could pass for fast-tracking treatments in a 100% fatal diseases. We need to re-look at accelerated approval, fast-tracked drugs for ALS patients through the FDA. ALS patients are going to die, lose walking capabilities, speaking capabilities, breathing, and eating abilities while the FDA does not push this drug through.”
The FDA declined to be a part of this story, citing no available representatives for an interview.
Since being diagnosed last November, Mr. Weinbrenner has not sat idly by.
He started Paint for a Cure, a nonprofit organization who seeks to raise funds for other patients faced with the financial burden of ALS. Mr. Weinbrenner, who has a background in real estate and residential development, hopes to build a house for patients to live in.
Paint for a Cure was born out of Mr. Weinbrenner learning that painting helped his symptoms of ALS.
“The average life span is 30 months,” Mr. Weinbrenner said of the ALS diagnosis.
Through the help of a nutritionist who’s set him on a path of healthy eating and the use of vitamins, Mr. Weinbrenner is feeling better and seeing results.
“I’m helping to prolong this because there are treatments out there, such as NurOwn, which is a stem cell treatment. Fortunately, Trump passed a bill to fast-track drugs, but in this particular case NurOwn is such a small company, it cannot afford to get those treatments to people like myself.”
The Right to Try Act was signed into law May 30, 2018. The law is another way for patients who have been diagnosed with life-threatening diseases or conditions who have tried all approved treatment options and who are unable to participate in a clinical trial to access certain unapproved treatments.
The Weinbrenners point to the fatality rate of coronavirus --- less than 6%, according to data provided by Johns Hopkins University & Medicine --- compared to that of ALS.
“ALS is 100% and we’re still sitting on our asses, basically, waiting for them to fast-track something that’s in Phase 3 trial. And literally, 50% of the people on NurOwn are getting up out of wheelchairs, they’re able to write again with their dominant hand,” Mrs. Weinbrenner said. “We’ve talked with people and there’s a lot --- one guy literally ran around the FDA showing them that they can actually run again. He was not able to walk, and he was on the NurOwn trial.”
Mrs. Weinbrenner says for people like her husband, within one year he could lose his voice --- pointing out how his speech has already changed in five months since diagnosis, saying he sounds impaired.
“It’s important and imperative to get a treatment such as stem cells as quick as you can before those neurons start dying,” she said. “It’s hard to get those back, and stem cells are important to those.”
The Weinbrenners have found a lot of support through the ALS community, and specifically a Facebook group called “No More Excuses! FDA and ALSA Protest” with nearly 5,500 members.
Members of the Facebook group just recently traveled to Washington DC in May to publicly advocate for ALS treatment drugs.
Mike Henson, one of the five administrators of the Facebook group, is an ALS patient himself. He says ALS is basically his full-time job now, and the Weinbrenners specifically named him as being a great source of knowledge and support during this time.
“We need this stuff now,” Mr. Henson said of NurOwn. “This drug has been in development for over a decade now. This is its 11th year --- it’s come all this way, it’s clearly safe. What we’re seeing now is Dr. Fauci and the government do everything for COVID-19 that we need right now for ALS. “
Mr. Henson says his group is trying to force the FDA’s hand somehow to provide access to this drug for people.
“So far, we just have not been able to break through, and it’s really frustrating because we’re going to lose a lot of people waiting on this trial to end. A lot of people are going to die without the chance to ever try this drug, and that is going to be tragic,” he said.
While FDA trial processes do take a long time in general, ALS is a very challenging disease, Mr. Henson explained, noting that it’s considered a small disease.
“The reason it takes so long is because it’s a very difficult disease to treat. Historically, there hasn’t been anything that’s worked,” he said.
Mr. Henson says there are two FDA-approved drugs for ALS and in his opinion, neither one has made a dent in the disease.
“NurOwn is the first drug that has stopped the disease and even reversed it in some cases,” he said. “It’s not a cure, I want to be clear about that, but it does slow it down.”
ALS does not have biomarkers --- such as how cancer can be measured by the size of a tumor, for example.
“There is no biomarker in ALS so when you run these trials, there is no end-point. In other words, the only measurable end-point is a rating scale we use to see how our physical capabilities remain,” Mr. Henson said.
“That is the primary end point in all ALS trials because it’s the only one we’ve got. There is no PSA-score, there is no tumor size. There’s nothing to measure in ALS except whether or not a person is getting worse or not. It’s hard to prove whether or not a drug works for ALS.”
Mr. Henson says the FDA is holding the disease to a very high standard because there’s not a measurable endpoint.
“What the FDA has never understood is that we may never have that for ALS. So the question now is watching all these people getting better or stopping getting worse, how is that not enough?” Mr. Henson asked.
Dr. Jeremy Shefner, senior vice president of Barrow Neurological Institute, says while it may be frustrating for patients waiting for a drug to be approved, the process is not for nothing.
“It’s a frustration when you have an ultimately fatal disease and the drugs that may be useful are not readily available. And you know, that’s a frustration for anyone with cancer or heart disease, or ALS as well,” Dr. Shefner said.
“Specifically with NurOwn, that’s a stem cell study, and although I understand the frustration it’s being tested in a very efficient way, and we’ll find the information as fast as we can.”
Dr. Shefner says the trouble is that if you have a disease, which on the average, leads to death in somewhere between three and five years, a drug can’t be looked at for only a couple of months.
“You need a significant amount of time,” he said. “Another issue is that ALS patients are relatively rare so you need to recruit in the hundreds of patients to definitively determine if the drug works or not.”
Dr. Shefner, who has been working with ALS patients for more than three decades, says he can get frustrated as well that no cure or type of significant solution has been found.
“It’s not that people aren’t working as efficiently as possible, it’s that ALS is a hard one to treat,” he said. “We’re in much better shape than we were 10-15 years ago in terms of what we know about the disease and how focused the drugs are.”
The Barrow Neurological Institute is a major clinical site, Dr. Shefner says, noting how the medical facility is involved in most trials, and runs some as well.
For ALS, there are eight new drugs that will soon begin trials, Dr. Shefner said.
Today’s research will assist in people --- much like the Weinbrenners --- not to be robbed of their lives in the future.
“You never know what’s going to happen with a clinical trial,” he said. “I think the fact that we have so many experimental drugs on the verge of being tested, and currently being tested, makes this a really hopeful time.”
BrainStorm, the company behind NurOwn, expects to have results at the end of the year for its Phase 3 trial.
Chaim Lebovits, CEO of BrainStorm Cell Therapeutics says the drug is an investigational therapy, and BrainStorm has not made any claims about the effectiveness of the drug in ALS patients.
NurOwn is a double-blind, placebo-controlled trial at six top ALS centers in the United States.
“The successful Phase 2 trial led to the pivotal Phase 3 trial that is now fully enrolled as of Oct. 11, 2019,” Mr. Lebovits said in an emailed response to questions.
“We all eagerly await the results of the Phase 3 trial.”
The outcome of the Phase 3 trial will determine the next steps. If the trial is positive, it would be at that time BrainStorm submits to the FDA for review.
From a technical aspect, the cell therapy drug takes mesenchymal stem cells, which are then converted into a cell-type --- called MSC-NTFs --- that produce neurotrophic factors, AKA molecules, which promote nerve tissue growth and survival.
Mr. Lebovits says the specific type of stem cells are nature’s own drug delivery system, and the hope is to potentially stop or slow the progression of ALS in some people.
“The Phase 3 trial is designed to answer this question and we will have the results at the end of this year,” Mr. Lebovits said.
When asked if BrainStorm is aware of the groundswell of support its receiving from the ALS community, Mr. Lebovits says their email inboxes are full and phones ring frequently.
“Every week, we respond to hundreds of emails and phone calls from the ALS community, members of Congress and policy makers,” he said. “We are motivated to act with urgency. This mindset is shared by everyone at BrainStorm Cell Therapeutics who are working diligently in the hopes that one day soon we can make a difference in the lives of people and their loved one living with ALS.”
Arizona Senator Kyrsten Sinema is one of the several law makers the Weinbrenners contacted about their fight for ALS treatment.
In a written statement to the Independent, the Sinema office says this about the Paradise Valley resident:
“Mr. Weinbrenner and his family wrote our office with his concerns, and we encourage all Arizonans in need to write, call, or email our office so we may be able to assist them.
Kyrsten is a founding member of the Senate ALS Caucus, a bipartisan caucus of Senators working collaboratively to advocate on behalf of ALS patients and their families. Kyrsten has a long history of supporting breakthrough scientific and medical research. She has consistently supported increased funding for research at NIH, CDC, and the Defense Department, including increased ALS research in Congressional appropriations.
Kyrsten was also a champion and original cosponsor of the Right to Try law that passed Congress in May 2018. Right to Try allows patients with terminal illnesses who are out of medical options to receive investigational drugs, and was inspired by an Arizona state law.
Kyrsten was also supportive of the 21st Century Cures Act, now law, to streamline the development of new medical treatments and accelerate the drug approval process to bring treatments to market more quickly, while maintaining important protections for patients.”