Three years ago, Nancy Klimas sat in an auditorium waiting to discuss her latest research progress. The audience was made up of the usual suspects at a scientific conference: doctors, scientists, and other academic colleagues. But this group was a bit different. The room was also packed to the brim with retired US veterans, all waiting to hear about any new developments over a “moonshot” idea that could be the closest attempt to a cure for Gulf War illness.
Klimas, who serves as the director for the Institute for Neuro-Immune Medicine at Nova Southeastern University in Florida, has been studying this debilitating condition for three decades. The strange sickness affects 175,000 to 250,000 soldiers who were deployed in the 1990-1991 Persian Gulf War. For those veterans, nearly half of who are pushing 50 or above, life has been an uphill battle. There is currently no cure for Gulf War illness, and because it involves a cluster of symptoms—fatigue, joint pain, diarrhea, memory loss—attempts to treat it have come up short. “These people served our country and put themselves in harm’s way,” says Klimas. “Now they’re sick with a chronic illness that ruined their quality of life and ability to work for more than 30 years.” Exhausting her options, Klimas came up with a rather unconventional idea: use a well-established abortion drug to reset the body’s overwhelming response to chronic illness.
Mifepristone, more widely known as the abortion pill, is capable of treating multiple illnesses. At low doses and when paired with another pill, misoprostol, the synthetic steroid binds to a protein in the uterus and stops the release of progesterone and other hormones needed to sustain pregnancy. But the drug has another effect, which Klimas is looking to tap. When taken at higher doses, mifepristone also blocks hormone receptors in the adrenal gland, which regulates the body’s stress response. The drug has already proven capable of doing this, and is currently approved as a treatment for the metabolic disorder Cushing’s syndrome.
Based on that evidence, Klimas wondered if the medication could temporarily block the adrenal gland and rebalance the hormone signals that are blunted with Gulf War illness. Repurposing the FDA-approved drug would also save the 10 to 15 years it would take to develop and test a brand-new drug. Klimas is halfway through her phase 1 trial testing the safety of the drug at different dosages on veterans, and is making plans for the second phase of the study.
The recent legal mess surrounding mifepristone access threw a wrench in Klimas’s plans, along with those of other researchers using mifepristone in their work. In early April, a federal circuit judge in Texas overturned the FDA’s 23-year-long approval of mifepristone, citing claims that the drug is unsafe for public use because abortion is now illegal in some states. And while the Supreme Court blocked the ruling that would have suspended mifepristone access across clinics, pharmacies, and mail orders, the future of the treatment remains uncertain in the US. “Obviously, we’re very concerned,” says Klimas, adding that mifepristone was already hard to get for research purposes. “Attempts to limit access to this drug has already had a splashback on the veteran population in these trials, as we’re delayed in rolling things out. How long will they have to wait for an effective therapy?”
Further constraints on mifepristone could impact medical progress on many other diseases and conditions as well. The medication is being studied as a potential treatment for diabetes in people without Cushing’s syndrome. It has also shown some potential in preventing weight gain caused by antipsychotic medication. Some ongoing clinical trials have found that mifepristone can be effective in slowing down the spread of breast cancer: The drug blocks progesterone receptors from releasing the hormone, which would normally stimulate tumor cell growth. And at different dosages, the pill can improve the quality of life of people dealing with painful uterine growths.
Banning mifepristone goes beyond stalling research—it puts any FDA-approved treatment at risk of being recalled. “You have a medication with an excellent track record of safety, efficacy, and high patient satisfaction,” says Carrie Cwiak, an OB-GYN at Emory Healthcare in Georgia. “The idea that the entire process for approving medication can be overturned [in court] is earth-shattering.” She says that restricting mifepristone opens a dangerous door to having people with legal power make treatment decisions based on their opinion and ideology rather than medical evidence.
If the courts decide to bar or limit mifepristone use down the line, it would discourage pharmaceutical companies from spending money on producing new drugs that appear controversial. That could include contraceptives, hormone blockers, or treatments completely unrelated to reproductive issues. “If you were a pharmaceutical company and it was going to cost you $20 million to move a pipeline drug all the way up through phase three [clinical trials], would you want to invest the money for it if it’s possible the bench could reverse the authority of the FDA?” Klimas asks.
Despite the setbacks on mifepristone access and potential legal battles, Klimas is optimistic that the research she is doing will help give veterans their long and overdue treatment. Her team is hoping to start their phase 2 trial soon and get as many results before politics interferes in science again.