Brynnlee and Archer Thomas, ages 4 and 2, didn’t hesitate on a recent morning when their grandmother, Shelly Hoover, called for help removing her socks. The two children charged in from their bedrooms and slid over to Hoover’s motorized wheelchair, each taking hold of one paralyzed leg and giggling furiously as they rolled down her bright purple knee-highs.
Beneath the thick fabric was a set of lavender toenails, painted to match the purple streak in the 51-year-old’s otherwise blonde hair. Since being diagnosed with Lou Gehrig’s disease in 2013, the Lincoln resident has made every effort to keep life vibrant even as she slowly loses control of her own muscles. This summer, she’ll join other California patients in a fight for experimental drugs that she hopes will give her a little more time to laugh with her loved ones.
Hoover, an educator and former administrator, plans to take advantage of California’s “right to try” law – a 2017 policy that allows patients with terminal illnesses to request experimental drugs that haven’t finished the Food and Drug Administration’s required trials. Critics say the California law, along with 33 similar laws nationwide, offers patients false hope and undermines the drug-approval process. Still, new federal legislation seeks to bolster the state policies.
With her neurologist’s help, Hoover plans to request an ALS medication when it completes its phase 1 trial this summer, she said.
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“There’s a lot in the pipeline; we have nothing that works, and we’re dying quickly,” Hoover said. “I’m extremely persistent, and I will keep asking until I get a yes. My alternative is to do nothing and die. It’s worth a fight for me to do it.”
“Right to try” laws allow patients to request drugs that have been proved safe for human consumption and passed a phase 1 clinical trial but have not undergone the next three phases, which test for effectiveness. Getting a treatment from its first clinical trial to full FDA approval can take up to eight years, according to Kenneth Kaitin, director of the Tufts Center for the Study of Drug Development.
Terminal patients have historically applied for unapproved treatments through the FDA’s “expanded access” program, which, like “right to try” laws, offers experimental drugs to patients who have an immediately life-threatening disease, have tried all existing approved treatments, are ineligible for clinical trials and have a written recommendation from a physician.
Between 2005 and 2014, the FDA received roughly 9,000 new applications for expanded access and approved 99.3 percent of them, according to a 2016 report from the agency’s Center for Drug Evaluation and Research.
“Right to try” laws cut out the FDA entirely, allowing patients to petition directly to drug companies. So far, manufacturers whose products are still in FDA trials have been reluctant to give those drugs out on the side, worried that an adverse effect in a “right to try” patient will prompt the FDA to block the drug’s development.
“From an industry and an FDA standpoint, it’s not a good idea,” Kaitin said. “There are a lot of vested interests in providing drugs by the standard mechanism. My sense is there will be a lot of fanfare each time there’s a new one of these bills that’s approved, but it will be done very rarely, in very circumscribed cases.”
Assemblyman Ian Calderon, D-Whittier, who wrote California’s “right to try” legislation, said the biggest holdup with the bill’s execution has been the lack of drug company involvement.
“If you’re a pharmaceutical company and you’re in the trial process, you could be incentivized to not give the drug for fear of losing your ability to stay in your trial,” Calderon said. “What we need is for the drug companies to be able to give the drugs to these patients without the fear of being kicked out of a clinical trial.”
Newly proposed federal legislation would do that by removing liability for physicians and manufacturers that aid “right to try” patients, and forbidding the FDA from using any patient data collected in “right to try” treatments against drug companies.
Both President Donald Trump and Vice President Mike Pence support federal legislation that would encourage manufacturers to release drugs outside of the FDA. The legislation could pass as a standalone or as part of the annually renewed Prescription Drug User Fee Act, said Starlee Coleman, spokeswoman at the Phoenix-based think tank Goldwater Institute, which has spearheaded “right to try” efforts nationally.
“There’s a tremendous amount of momentum this year that there wasn’t last year,” Coleman said. “We are optimistic. This is not going to happen quickly, we recognize that, but we’re hopeful that it will happen.”
Even if the federal protections for “right to try” pass, drug companies won’t be required to get on board. The industry group Pharmaceutical Research and Manufacturers of America said that when it comes to accessing experimental drugs, the FDA should stay involved.
“Because investigational medicines have not been approved by the FDA and determined to be safe and effective, FDA must approve expanded access to an investigational drug before a biopharmaceutical company can provide it to a patient,” said spokeswoman Caitlin Carroll in an email. “Any legislation should protect the integrity of clinical trials and the FDA oversight of expanded access to maintain the best interests of patients.”
There’s no estimate on how many Californians the new law will affect. It’s expected to be popular among ALS patients, who typically have two to five years to live post-diagnosis. Only one FDA-approved medication is available for ALS, which can slow but not stop the disease as it shuts down the motor neurons that communicate with muscles. The law could also help people with other disorders, including Duchenne muscular dystrophy and spinal muscular atrophy.
Multiple medical groups have spoken out against the legislation, arguing that it exposes patients to additional risk without any means of accountability. Don Nielsen, director of government relations for the California Nurses Association, said he’s concerned that patients will become “guinea pigs for the pharmaceutical industry.”
“The reason no one in the process wants the liability is because (the drugs) haven’t yet been tested,” Nielsen said. “It’s potentially a very unsafe situation for a patient. They’re being exploited by drug companies that could make a lot of money off the individual and not be held accountable.”
But Hoover said she doesn’t have time to wait for the FDA to find her the safest possible drug. She hasn’t been able to walk since 2013, and her arms have grown so weak that lifting them feels like raising 30-pound barbells, she said. Her spacious, one-story home is full of nicknamed medical equipment – her wheelchair, Ruby Tuesday, gets her from place to place while her lift, Lola, helps her transfer to her shower chair.
Soon she’ll welcome Obi, a robotic dinner plate with a mechanical arm to help her feed herself.
“You can see the desperation,” Hoover said. “Every month, there’s another thing I can’t do.”
Despite all that, Hoover manages to teach a course at nearby William Jessup University and tutor inmates at South Placer County Jail in Roseville. She hopes accessing a drug through “right to try” will buy her more time to do the things she loves.
“Where we have to approach it from is from a compassion perspective,” Hoover said. “What if it was your daughter? Or your mother? You would do anything you could to get access to a drug that might help you. So where do we find that balance between being compassionate to patients and also understanding liability?”