Day by day, Mike DeBartoli is losing control of his body.
Amyotrophic lateral sclerosis, or ALS, has claimed his ability to tie his shoes and button his clothes. The left hand he used to play basketball in college and then combat fires near Sacramento and Modesto for nearly three decades has withered and rests, immobilized, in a splint. He knows this is a prelude to an inevitable end – there is no cure – so he is willing to try anything.
“If my doctor told me today that ALS would be cured if you have a rock every day,” said DeBartoli, who is 53, “I’d swallow a rock every day.”
That determination brought DeBartoli from Tracy to Sacramento recently for a hearing on one of two bills offering a gleam of last-ditch hope to people like him. Part of a growing movement dubbed “right to try,” the legislation would allow Californians with fatal diseases to petition pharmaceutical companies directly for experimental drugs that have gone through some safety tests but have not yet garnered full approval from the Food and Drug Administration.
DeBartoli mentioned an ALS drug called GM604 currently being developed by Pasadena-based Genervon. An online petition asking the FDA to make the product widely available has garnered 750,000 signatures.
“If I’m able to take something that – who knows? – might help, I’m willing to do that because during that time I’m still around I’ll have that somewhat hope inside me,” DeBartoli said. “I’ll take that rather than not having hope at all.”
Arguments for Assembly Bill 159 and Senate Bill 149 echo the 1980s battles over HIV/AIDS medicine, when stricken patients implored the government to give them swifter access to not-yet-approved drugs. The newer right-to-try movement similarly decries the process of greenlighting new treatments as too slow to help those in need.
“Drug approval through the FDA can be quite a nightmare,” said Assemblyman Ian Calderon, D-Whittier. With his bill, he said, “there are no guarantees they’re going to get these drugs, but at least you know you live in a state where if you’re in that situation, you can try.”
The bills would circumvent the FDA entirely. Rather than seeking experimental drugs through the government, patients whose illnesses are sufficiently advanced could go straight to pharmaceutical companies developing the medications. The drugs would need to have passed the first phase of the development process, where researchers explore how the body processes the drugs.
Nationally, the idea is riding a wave of momentum. At least 36 states have taken up bills and 12 of them were signed into law, according to the National Conference of State Legislatures. Much of the credit for those advances comes from the nonprofit Goldwater Institute, which has helped introduce bills in numerous states and sponsored SB 149 in California.
“We’ve increased that scale as much as we could this year,” said Kurt Altman, national policy adviser for the Goldwater Institute. “It is a very common-sense piece of legislation that’s very bipartisan – this affects everybody.”
The California bills have drawn opposition from the California Medical Association and from groups representing nurses and oncologists. A group representing pharmaceutical manufacturers has sent a letter warning against skirting the FDA’s established channels.
“We have serious concerns with any approach to make investigational medicines available that seeks to bypass the oversight of the Food and Drug Administration and clinical trial process, which is not in the best interest of patients and public health,” a letter from Pharmaceutical Research and Manufacturers of America says.
Despite that formidable group of opponents, AB 159 cleared the Assembly floor with only two no votes. SB 149 advanced out of its first two committees without a single vote in opposition.
Skeptics warn that the drawn-out FDA approval process exists to protect people. They question whether the legislation would make a significant difference, particularly since pharmaceutical companies would still need to agree to make experimental drugs available – an uncertain proposition at best.
“We don’t think this bill actually delivers,” said Stephanie Roberson, a lobbyist for the California Nurses Association. “There has to be industry participation, and if the manufacturers aren’t willing to do anything, there’s nothing the doctors can do.”
Medical professionals acknowledge that the process of putting drugs through a series of tests and clinical trials can be frustratingly drawn out but say the wait is preferable to approving treatments that are dangerous or ineffective.
“The problem with a lot of drugs that have not yet been tested is that the patients are exposed to a significant amount of risk,” said Dr. Daniel Mirda, president of the Association of Northern California Oncologists, which opposes both bills. “Our concern is a drug that potentially could cause harm is used in a setting that then will interfere with, ultimately, its approval.”
A patient having an adverse reaction to an experimental drug could derail the process, Mirda said. If, for example, a patient with a history of heart issues takes a non-approved cancer drug and then suffers a heart attack, he said, “that will certainly derail that drug or put it on a serious pause mode.”
The FDA does allow some patients to try drugs short of full approval, through the so-called “compassionate use” option. The agency notes that it approves the vast majority of requests for the compassionate use program, although it is again up to drug companies to agree to offer the unfinished products. They are working to simplify an application process often criticized for its complexity and length, though they dispute that critique.
“I think that’s kind of a mythological thing,” said Richard Klein, director of the FDA’s patient liaison program. “They process pretty fast, because they are for people who are out of other therapies or don’t have anything that works for them.”
But right-to-try supporters say that the FDA’s route is simply too slow moving for people who are counting their remaining time in months and weeks.
“It’s a very onerous process,” said Sen. Jeff Stone, R-Temecula, SB 149’s author, adding that in his time working as a pharmacist, “I saw many of my patients that were stricken with very serious diseases have to fly to other countries to get treatments that were not authorized by the FDA in this country.”
The cancer attacking the kidneys of Keith Knapp’s wife had already entered an advanced phase when they learned of experimental drugs that might help. But Knapp said they were thwarted by drug companies reluctant to offer the product and doctors resistant to launching the lengthy compassionate use process of seeking it – pushback he traces to government hurdles.
“Both sides of that equation blamed the FDA for why they don’t want to be involved,” said Knapp. “I don’t see (the California bill) as something that solves all the problems,” Knapp added, but “it’s a step.”
Even if it is only a small step, and even if pharmaceutical companies decline to make the treatments available, Calderon said he is allied with lawmakers in other states who are together prodding the federal government to improve.
“I think the only way you can get change from the FDA,” Calderon said, “is pressure from the states.”
Editor’s Note: This story was corrected at 9:30 a.m. May 26, 2015 to say that it is Mike DeBartoli’s left hand in a splint.