Local family’s plight fails to bridge partisan divide - SanTan Sun News SanTan Sun News

Local family’s plight fails to bridge partisan divide

April 16th, 2022 SanTan Sun News
Local family’s plight fails to bridge partisan divide

By Paul Maryniak
Executive Editor

Twice in the last two months, Kendra Riley appeared before legislative committees to implore for the passage of a bill that she feels could ease the burden for parents who endure the heartbreaking ordeal that befell her family in March 2020.

The Ahwatukee mom had appeared on behalf of SB1163, – promoted by the Goldwater Institute as the “Right to Try 2.0.” It would give parents the right to obtain individualized experimental treatments in this country instead of forcing them to do what Kendra and her husband David had to do two years ago: Spend more than $500,000 to go to Italy to try to save two daughters’ lives.

But her plea fell on Senate Democrats’ deaf ears.

But her plea fell on deaf ears where Senate Democrats were concerned, except for Awatukee Sen. Sean Bowie and Yuma Sen. Lisa Otondo – who broke ranks with their caucus and was the only two members of their to vote in favor of the bill. One other Democrat, Tempe Sen. Juan Mendez didn’t vote – nor did Republican Sens. Kelly Townsend of Mesa and Michelle Rita-Ugenti of Scottsdale.

While SB1163 passed 16-11-3 on Valentines Day and two weeks later was recommended for approval by two House committes – where only Republicans again voted for it – it has yet to come up for a final floor vote.

The Rileys’ plight is the genesis for Right to Try 2 – which, if passed, would make Arizona the first state to go beyond the similar but more limited Right To Try law that Congress passed and President Trump signed in 2018. Arizona and 40 other states have adopted similar laws.

The Rileys’ ordeal began in March 2020 – just as the pandemic was starting to grip Arizona and the rest of the world.

The couple had far bigger worries than COVID-19: Their 2-year-old daughter Olivia had difficulty walking. An odd vibration developed in the iris of one eye.

Tests subsequently confirmed Olivia had metachromatic leukodystrophy (MLD), a rare genetic brain disease that has no cure as it destroys the protective fatty layer surrounding the nerves in the central nervous system, aggressively taking away motor function and other abilities.

Within 90 days of her first symptoms, Olivia could no longer walk or talk.

Subjected to moderate to severe pain on a daily basis, she is now in hospice care. Children with MLD typically aren’t expected to live past 6.

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The Rileys’ heartbreak only worsened after that.

Neither devastated parent knew they were carriers and so they had their two other daughters tested. And while their eldest daughter Eva is a carrier and not affected by the disease, the tests showed their 6-month old daughter Keira also had MLD.

The Rileys found that only one medical facility in the country, located in Iowa, offered a treatment program for Olivia. “It wouldn’t cure the disease” Kendra told the Senate Health and Human Services Committee Jan. 13 on behalf of a bill introduced by West Valley Sen. Nancy Barto.

“It would require weekly trips for us from Phoenix to Iowa,” Kendra continued. “So we were doing that every single week during the pandemic with her just to try and get as much time with her as we could with her.”

Meanwhile, an advocacy group offered the Rileys some hope for their baby: A medical facility in Milan, Italy, had developed a gene treatment that showed great success with children who had MLD but had not yet developed symptoms.

A team of doctors in Milan told Kendra that Keira could be the 31st kid in the world to have this treatment. “Kids that have had this treatment in the past – they’ve only been doing this for a decade – are now 11 and 12 and living completely normal lives. So they’re walking, they’re talking, they’re good playing soccer and going to school and you’d never know they have MLD.”

“We could have had everything done here, but it wasn’t in place, ” Kendra continued.

And so, she said, “We had to raise a half a million dollars. We had a month to do so. We had to get passports for the girls, visas for the girls. We had to move Olivia’s clinical trial to a site in Amsterdam. And we needed my parents to come with us.”

Thanks to the generosity of family, friends and especially the Ahwatukee-based Armer Foundation for Kids and the Desert Foothills Credit Union Foundation, the Rileys raised the money and stayed in Milan for six months as their infant received treatment at the San Raffaele-Telethon Institute for Gene Therapy.

“I’m very happy to say she’s advanced for her age. She’s walking, she’s talking, she’s running, she’s climbing,” Kendra said, her voice cracking with emotion. “It’s amazing. It’s things Olivia could never have the chance to do.”

“So I’m here today in support of this bill because if it were in place, and we were going through this right now, we would get this treatment for cure and we’d be here. We wouldn’t have to raise hundreds of 1000s of dollars, leave our jobs and our family and our friends and go halfway around the world during a global pandemic to save our daughter’s life.”

Now, a Minnesota clinic is providing the same kind of individualized treatment that has made Keira, now 2, able to do “all these things that we never got to see Livvy do.

“And Livvy is now enrolled in hospice and we just have to keep her as happy and comfortable as possible.”

About Right to Try 2

The 2018 federal Right to Try law gives families access to potentially lifesaving treatments for their children in the United States but under various conditions.

The biggest condition is that the treatments must have been approved by the U.S. Food and Drug Administration for large groups of patients.

Even a Minnesota clinic providing the same kind of treatment that Keira found in Milan must get special permission from the FDA on a case-by-case basis.

“Patients now have the right to seek treatments that are safe enough to be used in clinical trials but remain under clinical evaluation for final FDA approval,” Naomi Lopez, Goldwater Institute director of healthcare policy, told the House Health and Human Services Committee March 14.

She explained that even though such treatments might be considered “individualized,” the FDA requires they apply to large groups of people.

“Today this law needs to be modernized to account for the rapid advancements in medicine, such as gene therapy, which aren’t covered under the original Right to Try law,” Lopez said.

“That’s where Right to Try 2 comes in: Many of the medical innovations being pioneered today have made it impossible to take an individual’s genetic information and create a treatment for that individual person” she continued, explaining:

“The current clinical trial evaluation system – which was created more than half a century ago – isn’t keeping pace with these modern medical advances. And the end result is that individualized treatment is still subject to the same clinical trial process as single treatments that are intended for hundreds or even thousands of patients.

“And that doesn’t recognize how these new individualized treatments work. It’s a mismatch… Right to try for individualized treatments counts for new innovations and it helps get those innovations to the patients that need the most.”

SB1163 has a number of safeguards and limits. It does not require a clinic to provide individualize treatment, nor require insurers to pay for it. It prevents the state from blocking patients from receiving it.

For the most part, it exempts a provider of those treatments from lawsuits as long as “it complies with the law and has exercised reasonable care.” And it requires a very detailed explanation for the patient and their loved ones on the potential risks of the treatment.

“SB1163 is a game changer for families like mine and Kendra’s,” said Christine Hoffman, of the MLD Foundation who lost her daughter to the disease at age 8.

Testifying before the House committee, Hoffman said, “We have come so far in the last few years with gene therapy…The costs to take part in the treatment overseas are exorbitant and the logistics nearly impossible, especially if the family has other affected siblings that need to be taken care of at home.

“Expanding the bill to cover individuals that need life-saving treatment like our daughters means that the treatments can be done in the States. We can remain surrounded by our support systems, including medical and therapy providers, family and friends.

“Imagine being able to receive these potentially life changing treatments without having to leave our state, our kiddos and everyone with a rare disease deserve a fighting chance here at home. And this bill gives us that opportunity.”

There was no debate when the measure came to the Senate floor, but Demcoratic members of both the Senate and House Health and Human Services Committees gave different reasons why Kendra’s testimony failed to persuade them.

Sahuarita Sen. Rosanna Gabaldon said Kendra’s “very compelling testimony” resonated with her because she has a child with an autoimmune disorder,

“However, I still have questions. And I’m very torn with this bill because it’s something that could be really good, but there’s just not enough information,” Gabaldon said. “I’m gonna vote no today, but I’m gonna do my homework, I promise. And when it comes to the floor, my vote may change.”

Her vote didn’t change on Feb. 14.

Tucson Sen. Sally Ann Gonzales also thought Kendra and Hoffman gave “very compelling testimony,” but said, “We have had a lot of advances in the medical field. But sitting up here, we have to – we’re not doctors. And so it’s very hard to make these decisions up here.“

During the House Health and Human Services Committee hearing, Tucson Rep. Pam Powers Hannley asked Lopez how many patients have survived with experimental treatments under the 2018 Right to Try law.

Lopez said she had no idea because the Biden administration has not finalized rules for a law passed four years ago.

Lopez stressed both Right to Try measures “work in tandem with a clinical evaluation system. There is nothing being done that is random or that medical professionals would deem as unsafe.”

A few minutes later, Powers Hannely said, “A phase one trial determines whether or not the drug is going to kill you. It doesn’t determine whether it’s going to work or not in any case. So this bill is a Right to Try bill but it’s also a ‘buyer beware bill’ in my opinion.

“It will expand the types of experimental treatments that patients can try but it will also most likely increase the patient’s out-of-pocket medical costs because these treatments will not be covered by insurance for the most part. This is a consumer protection issue in my opinion. And with that, I vote no.”

Scottsdale Rep. Kelli Butler noted that her mother has a brain cancer “and it’s awful and I’m sure you feel desperate and would like to have anything that you think might help.

“I’m worried that this bill that we’re the first in the nation to contemplate an expansion like this. I’m worried that this is going to open a door to people who may not have the best intentions potentially, and that they will have the potential to really prey on people who are desperate.”

“We have a clinical trial process for a reason because it’s a careful process,” Butler continued. “It is not fast enough. It should be faster, but this bill worries me. Also worrying is that I’m looking at who’s signed in in support of this bill, the Goldwater Institute, the Republican Liberty Caucus, a bunch of individuals.

“But I don’t really see anybody associated or at least identified as being a medical person who is signed in in support of this. And I think it could open a troubling door to potentially take advantage of people and so I vote no.”

Bowie said he said no reason to vote against the bill.