Ice Bucket Challenge Fires Up ALS Research 5 Years Later

Damian McNamara

July 02, 2019

 

Nancy Frates, the mom who started the ice bucket challenge, with her son Pete, who was 27 years old when this photo was taken.

Pete was a healthy Division I athlete before he was diagnosed with amyotrophic lateral sclerosis (ALS). His first symptom was a baseball wrist injury that would not heal.

"We were very lucky with my son. Pete was at an orthopedist who very quickly identified there was a neurologic problem," his mother, Nancy Frates, explained. "And we're in Boston, where ALS is very much a part of the community, so he was diagnosed very early."

When Pete learned he has ALS, only riluzole (Rilutek) was approved by the US Food and Drug Administration, but "we were told, 'we're not sure it does anything'," Frates told Medscape Medical News.

In 2012, the message was, "no treatment, no cure, 2 to 5 years prognosis, 100% fatal," she said.

"The fact that only one gene had been discovered in a disease that had been around for 150 years was a surprise," she added.

This year also marks the 80th anniversary of Lou Gehrig's iconic speech at Yankee Stadium after he was diagnosed with ALS.

Gehrig spent 17 seasons in Major League Baseball playing for the New York Yankees. The talented hitter, who set several major-league records during his career, stunned players and fans when he voluntarily took himself out of the lineup after his performance on the field was hampered by ALS.

On July 4, 1939, Gehrig stepped in front of the crowd and said: "Fans, for the past 2 weeks, you have been reading about the bad break I got. Yet today I consider myself the luckiest man on the face of the earth. I have been in ballparks for 17 years and have never received anything but kindness and encouragement from you fans."

Just 2 years after he retired at the age of 36, Gehrig died.

Henry Louis Gehrig is fondly remembered for his iconic "luckiest man on the face of the earth" speech.

Frustrated with the lack of research progress and effective ALS therapies, the Frates family took action with their grass roots initiative that went viral on social media.

Each participant nominated for the ice bucket challenge was encouraged to have a bucket of ice water poured over his or her head, upload video evidence of the act to social media, make a donation, and then nominate someone else to do the challenge.

This summer marks the fifth anniversary of the ice bucket challenge.

In large part because of the $115 million raised for ALS research during an 8-week period in 2014, five additional ALS-related genes were discovered.

There are currently 86 ALS therapies in the drug-development pipeline. And a second ALS medication, edaravone (Radicava), was approved by the US Food and Drug Administration after clinical trials indicated it could slow progress in some ALS patients by 33%.

Some of the money raised went directly to research grants, but some was used as seed money for researchers who could then increase the money with matching grants.

The success of the challenge also reinvigorated ALS researchers who had been toiling away, in some cases for decades, with little to show for it. Plus, the increased awareness and momentum in the field attracted a completely new crop of researchers.

And new donors continue to come forward to support ALS research efforts.

Watching ALS progress in a loved one "is the most brutal, brutal thing you could ever imagine," Frates explained.

Since her son's diagnosis, Pete has lost almost every function in his body and has been on a ventilator for the past 4 and a half years.

"His brain is there, and he can hear. He can still open his eyes and we communicate with him by just looking into his soul," she added. "His heart is still as strong as ever and as loving as anyone's."

"He's very, very disabled — but he lives," she said.

He Lives

Nancy and Pete Frates are learning to live with ALS.

The unfaltering progression of the disease brought some unwelcome surprises.

"We know the big milestones with the disease," Frates said: the day people can no longer walk and they have to go into a wheelchair; the day they can no longer swallow and they need a feeding tube; and the day they can no longer talk because of the progression of ALS.

"But there are also the little ones," she noted, "like the day Pete couldn't text us anymore, or the day he dropped his keys on the table when he realized he couldn't drive anymore — at 28 years old."

And then there was "the day I knew he wasn't going to say 'Mom' any more," she said. "There are things like that that just tear at your heart."

In the years since her son's diagnosis, Frates continues to dedicate her time to advocating for families facing the many challenges of ALS. "My work in this field is cathartic," she said. Pete's motivation "to get to work," his resolve to make a difference for others, and his hope "that no other family has to go through this is what drives me every day."

During her keynote address at the recent Infusion Nurses Society 2019 Annual Conference in Baltimore, Frates delivered three main messages to clinicians.

Her first message was one of gratitude. "Nurses are our family now" and support Pete and his loved ones, not only medically but emotionally and mentally as well, she explained. Over the past 7 years, "if we didn't have our nurses, I'm not sure where we would be."

Her second message was a reminder to remain compassionate.

Frates described the extensive preparation, frustration, and sometimes angst that goes into getting Pete ready to leave home. "When we walk through the door for our 11 AM appointment, you see our smiling faces." However, the hours of preparation start much earlier in the day. "Dealing with the logistics can be very frustrating. It is mentally exhausting," she said.

Pete's nurses get his medications and equipment ready. The family — Pete, his wife, their 5-year-old daughter, Frates, and her husband live in the same house — decides who will go with Pete to each appointment. "We all have commitments," she pointed out.

Hard Work and Hope

Frates said she shares these challenges with nurses and other clinicians so they will understand the personal struggle.

"What I try to express to the medical community is that you made a choice to come into a field where you are treating people who are at their most vulnerable, so I don't think empathy is absent; I'm just here to remind you of the importance of that compassion," she said.

Her final message was to inspire nurses to keep encouraging patients and their families. "No matter what they are infusing — whether it's nutrition, pain medication, or chemotherapy — they are delivering one thing, and that is hope."

"You can ask any patient or any family; when you have hope, everything else falls into place," Frates said.

Unlike in 2012, when there was little hope that the natural course of the disease could be altered, today a drug might actually slow progression.

But that is just the start, as Frates sees it. "When you have the top doctors in this field, who have been working on this for 30 or 40 years, saying, 'We know there will be a treatment, and hopefully a cure, at some point,' it means effective treatments are on the horizon."

"I am extremely, extremely optimistic," she added. "That's not just my opinion, it's an educated opinion. I am in communication with the top people in this field, and they are the ones saying that."

Although the ice bucket challenge did a great deal to raise awareness, she said it is still not at the level she would like to see.

Early Recognition Still Needed

For physicians, particularly primary care doctors, keeping ALS in their differential diagnosis is important. "Every physician should be aware of ALS. Most will need to educate themselves, because we know in medical school, unless you're going into the field of neurology, you're getting a paragraph on the disease," Frates said.

Refer patients with suspected ALS to a neurologist for further evaluation as quickly as possible. "A lot of GPs will send patients to an orthopedist first because the onset of symptoms can be similar to Lyme disease or carpal tunnel syndrome," she added.

It is also important to stay up to date on advances in the field, she advised, "because with our momentum, we're going to be identifying more treatments."

And the future will likely bring options for people with ALS to participate in clinical trials, she emphasized, so neurologists and other physicians need to remain aware of these and other possibilities.

Frates said she is not only grateful to medical professionals, but to the groundswell of support from each member of the general public who gamely poured a bucket of ice water over their heads to raise awareness for this disease.

"We want them to know how grateful we are," she said. More than 17 million people uploaded ice bucket challenge videos to Facebook. "People took hold of this, and look what they did."

"And look at what progress has been made in 5 years," she pointed out.

Prior to the ice bucket challenge, people were resolved to dying from ALS. Now, "people have a new mindset — we're living with ALS."

Journalist Damian McNamara's grandmother, Anne Smith, had ALS. In 2014, McNamara participated in the ice bucket challenge and raised money in his nan's memory.

Infusion Nurses Society (INS) 2019 Annual Conference. Presented May 18, 2019.

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