If approved by Legislature, Minnesotans could get easier access to experimental drugs, devices.
March 16, 2015 at 1:51PMLinda Griffiths talked about the future with her husband Jim as her ALS progresses rapidly while her treatment is limited to antidepressants and medications to keep incontinence at bay while her body deteriorates. (The Minnesota Star Tribune)
Linda Griffiths' ALS regularly makes itself known in the form of something else she no longer is able to do.
She accepts the new life the disease has forced her into, but she won't call it a death sentence. Not yet.
Less than a year ago, Griffiths, of Golden Valley, was diagnosed with amyotrophic lateral sclerosis, an incurable disease that attacks nerve cells in the brain and spinal cord, robbing patients of their ability to move and eventually, even to breathe. Most die within two to five years.
Griffiths' body is deteriorating rapidly, and standard treatments have been limited to muscle relaxers and antidepressants, along with stretching routines and massages from her husband, Jim.
An experimental drug called GM604 has showed positive results in a small sampling of test patients, but has not yet been approved by the Food and Drug Administration. Griffiths, 56, says she is running out of time and would like to try it: "If I'm going to die from this disease, my feeling is, give me a chance, at least."
Under the proposed Right to Try Act, patients like Griffiths who are diagnosed with terminal illnesses would gain easier access to drugs, procedures or medical devices that are still undergoing clinical trials. If the bill is approved by the Legislature, Minnesota would join five states in passing the Right to Try Act. An additional 26 states have introduced similar bills this year.
The movement started with the Goldwater Institute, a Phoenix-based conservative and libertarian think tank.
Under the bill, the medications still must pass the first and most exhaustive phase of FDA clinical trials, and patients must have a physician's signoff.
Manufacturers are not obligated to supply any drug, treatment or procedure.
"At its core, it is a personal freedom and choice effort to allow patients who have been told they've exhausted all of their options with a terminal disease to try investigational medications," said Craig Handzlik, state policy coordinator at the Goldwater Institute. "This allows patients to say 'I know there are risks here, but I really want to try it, and the only definite outcome if I don't try is death.' "
The government allows dying patients to take experimental drugs under what's called "expanded access," or "compassionate use." But Right to Try advocates say the bureaucratic red tape and the waiting period of months and sometimes years often is longer than the patient's life expectancy.
Not everyone favors greater access, even for terminal patients. Dr. Steven Miles, a bioethicist at the University of Minnesota, said the movement is merely an attempt at deregulation. The first phase of FDA testing, he said, is to determine whether the drug shows immediate toxicity. The second phase determines whether it actually works, while the third phase measures long-term harm and benefit. The end result could be patients desperate to try anything, regardless of whether it works. The same thing happened during the peak of the AIDS epidemic, Miles said.
"We can take down all the federal protections, but at what point do we want some oversight, or do we want this to become the Wild West of providers selling miracles?"
Although the bill defines terminal illness as "an advanced stage of a disease with a terminal prognosis and no known cure," Miles is concerned that it isn't more specific. Another key omission has him concerned.