Arizona Becomes First State to Adopt Right to Try for Individualized Treatments

Arizona Becomes First State to Adopt Right to Try for Individualized Treatments

By Corinne Murdock |

On Wednesday, Arizona became the first state to allow patients to access individualized treatments otherwise blocked by the FDA. The bill was dubbed the “Right to Try for Individualized Treatments,” an expansion of original Right to Try law allowing patients with life-threatening illnesses to undergo clinical trials of the treatments of their choosing. 

Governor Doug Ducey signed the bill, SB1163, into law. The Phoenix-based Goldwater Institute, a conservative and libertarian public policy think tank, pioneered the original Right to Try law. That law also began in Arizona. It eventually received approval from Congress under former President Donald Trump. 

Goldwater Institute President and CEO Victor Riches said that the new law ensured protections for a fundamental right: the right to save one’s own life. 

“The right to try to save one’s own life is one of the most precious rights of all,” said Riches. “America doesn’t have to wait for the FDA to reform itself in order to put patients first. States can and should act now to protect all Americans’ fundamental right to try to save their own life.”

Goldwater Institute Executive Vice President Christina Sandefur said that the law provided new hope for Arizonans. Sandefur called the law “Right to Try 2.0.”

As AZ Free News reported, the bill was inspired by the Riley family in Phoenix. Two of their three daughters, Keira and Olivia, were born with metachromatic leukodystrophy: a sudden, fatal disease that attacks nerves in the brain and spinal cord. Due to FDA restrictions, the Rileys had to seek treatment outside of the country. They were forced to raise tens of thousands of dollars at the height of the pandemic in 2020 in order to relocate to Italy for the five months they needed to access treatment for Keira. The experimental gene therapy was their last hope to save her life.

Olivia couldn’t qualify for the treatment because she was already symptomatic. As of this report, she was transferred to hospice. The average life expectancy for metachromatic leukodystrophy patients is six years. 

Kendra Riley, the girls’ mother, spoke with conservative radio host James T. Harris about the bill’s enactment. Riley said that the next step would be to have the rest of the nation pass similar laws.

“You think if your child has one chance in the entire world to live a normal life, we should be able to have access to it in America,” said Riley. “The medicine is there but bureaucracy and insurance shouldn’t be holding it back. Everyone should have the right to a chance at a normal life. I truly hope this helps everyone.”

As they were rallying support for the bill, Sandefur characterized current law as immoral.

“It is unconscionable that an American patient has to travel to another country to Europe in order to be able to get access to a treatment that could save their lives,” said Sandefur.

Corinne Murdock is a reporter for AZ Free News. Follow her latest on Twitter, or email tips to corinne@azfreenews.com.

Phoenix Family Fights FDA Restrictions to Save Children’s Lives

Phoenix Family Fights FDA Restrictions to Save Children’s Lives

By Corinne Murdock |

One Phoenix family is fighting for their daughters’ lives: not only against the odds of the fatal genetic disease they both suffer from, but against the Federal Drug Administration (FDA) regulations that prohibit access to a potential cure. The life-saving treatment, a gene therapy, is only available in Milan, Italy — thousands of miles away at the cost of anywhere between $1,000 to $2,000 per round trip flight ticket. 

The two of three daughters in the Riley family, Keira and Olivia, were born with metachromatic leukodystrophy, a sudden-onset, fatal disease that causes a buildup of lipids on critical nerves in the brain and spinal cord, which in turn damages the myelin sheath that protects nerve cells. Medical journals identify this occurrence as a result of a deficiency in sulfatide enzymes, which break down lipids. The Riley family had to raise tens of thousands of dollars at the height of the pandemic in 2020 to relocate to Italy for five months so they could save Keira. 

Olivia didn’t qualify for the experimental gene therapy because she was already symptomatic. Within 90 days of showing her symptoms, Olivia lost the ability to walk and talk. 

“How do you even deal with that as a parent? Imagining two out of your three kids will soon be gone?” said their mother, Kendra. 

Due to cases like that of the Riley family, the Arizona legislature is considering a new law to expand on current laws enabling terminally ill patients to try treatments not yet approved by the FDA: SB1163, or the “Right to Try for Individualized Treatments.”

The Goldwater Institute pioneered the original Right to Try law that began in Arizona and eventually was approved by Congress under President Donald Trump, guaranteeing patients with life-threatening illnesses that exhausted all other options to participate in clinical trials of the treatments of their choosing. 

Christina Sandefur, Goldwater Institute Executive Vice President, dubbed SB1163 the “Right to Try 2.0” — an expanded upgrade to the original law tailored for more individualized needs. The State Senate passed the bill last month, and it’s awaiting a final vote before the House.

“It is unconscionable that an American patient has to travel to another country to Europe in order to be able to get access to a treatment that could save their lives,” said Sandefur. 

For the Riley family, it appears that the experimental gene therapy worked — since January 2021, Keira hasn’t exhibited any symptoms and has developed normally. In mid-January, the FDA granted the same treatment Keira received in Italy. The treatment was given approval, in part, because of results from the Milan, Italy trials.

Corinne Murdock is a reporter for AZ Free News. Follow her latest on Twitter, or email tips to corinne@azfreenews.com.