CHICAGO (USA Today) - The day before he turned 30, Marqus Valentine panicked. “I was so scared for midnight to come rolling around because subconsciously I was like, ‘This is it. Tomorrow's my last day on Earth,’” he said.

Valentine has sickle cell disease, an inherited blood disorder, and his doctors had warned him throughout his life that he was not likely to make it to 30.

That birthday passed without event, as have four more. Still, Valentine’s disease has left him severely disabled. “Instead of 34, I feel like I'm 68,” he said.

He uses a portable oxygen tank, needs a hip replacement and sleeps in a hospital bed on the first floor of his parents’ house — he is in too much pain to make it up the stairs to his bedroom. And an early death still looms: “I could wake up in the morning, brush my teeth, eat breakfast and by the time afternoon comes rolling around, I could possibly be dead.”

Like many people with sickle cell, Valentine has watched as one successful advocacy campaign after another brought attention and resources to other disorders, including breast cancer, HIV, cystic fibrosis and Lou Gehrig’s disease. But little is heard about sickle cell. With drug development and other treatments, outcomes for most diseases have improved over the past few decades, while life expectancy for sickle cell patients has declined.

To sickle cell patients and their families — mostly African-American — efforts to fight the disease appear slow, underfunded or ineffective, perpetuating century-old disparities.

"You feel like the baldheaded stepchild that no one cares about,” Valentine said from his bed at Edward Hospital in Naperville, Ill., while recovering from one of his bimonthly blood transfusions. “What about us? We're here too.”

Marqus and Francesca Valentine sit in their living room in Lisle, Ill. They are training their two dogs to be service animals to help Marqus with his disabilities caused by sickle cell disease.(Photo: Jenny Gold, Kaiser Health News)

Sickle cell disease affects an estimated 100,000 people in the U.S., causing chronic pain, multi-organ failure and stroke. With annual costs to treat the disease soaring past $1 billion, new efforts are afoot to improve the lot of patients. But each development faces obstacles:

►In July, the FDA approved the first new drug to treat sickle cell in two decades. Yet specialists say Endari, expected to hit the market this month, will likely have limited benefit. And insurers are balking at covering it. Several, including the Valentines’ Blue Cross Blue Shield’s Federal Employee Program, will cover Endari only if patients have “failed first” at other treatments, including blood transfusions and hydroxyurea (the only other drug available to treat sickle cell).

►Physicians associations are working to disseminate guidelines to improve care and reduce discrimination against sickle cell patients, often assumed to be drug addicts when they go to emergency rooms in severe pain, caused by their misshapen red blood cells clogging the vessels and cutting off oxygen to joints and organs. Still, a dearth of research on sickle cell makes it difficult to write evidence-based protocols.

►Legislation in Congress to fund research and treatment, stalled since 2009, is finally moving out of committee. But the bill would provide only $4 million — less than half its original funding level.

Most sickle cell patients struggle to access even the most basic care. ER doctors rarely understand how to treat them, and patients get misdiagnosed and inappropriately sent home, said Patricia Kavanagh, a pediatrician and ER physician at Boston Medical Center. “What we learn about sickle cell disease in med school is usually covered in 20 minutes.”

Nearly 40% of sickle cell patients have to be readmitted to the hospital within 30 days — more than for any other diagnosis, according to the federal government’s Agency for Healthcare Research and Quality.

"It's appalling. This country ought to be ashamed of itself,” said Valentine’s mother, Francesca Valentine, a registered nurse for over 35 years who has become an activist for sickle cell patients. "I am baffled that in 2017, we’re still not treating the disease based on science, and we still deal with racism and stigma and inaccurate information.”

In 2014, the National Heart, Lung, and Blood Institute, part of the National Institutes of Health, put out guidelines for physicians, and 11 national organizations endorsed the protocols. But protocols are only as good as the science behind them, and “the evidence for most of the guidelines is not strong,” because there have not been enough studies on the disease, said Rosalyn Stewart, a Johns Hopkins physician and an investigator on a federal grant to improve care for sickle cell patients.

Sickle cell funding pales in comparison to other diseases. Cystic fibrosis, which affects 30,000 people in the U.S., for example, gets seven to 11 times more funding per patient than sickle cell disease, according to a 2013 study in the journal Blood. The ALS challenge in 2014 raised $115 million for approximately 20,000 patients in the U.S.

Some new sickle cell studies are in the works, including eight funded by the Patient-Centered Outcomes Research Institute. But one major problem is that no national data registry exists for sickle cell disease, said Mary Hulihan, director of the sickle cell program at the Centers for Disease Control and Prevention.

“We don’t have even basic information like how many people in the U.S. have sickle cell disease or where they are getting their health care,” Hulihan said.

Despite the setbacks, experts and patients remain hopeful that the treatment of sickle cell disease can gain momentum and attention.

But if insurance doesn’t cover Endari for her son, Francesca Valentine said, she and her husband would both need second jobs to afford the drug. If necessary, she plans to challenge her insurer by writing letters explaining the science.

“It’s like this disease has to fight for everything. We should not have to fight for everything,” she said.

Kaiser Health News (KHN) is a non-profit news service covering health issues. It is an editorially independent program of the Kaiser Family Foundation that is not affiliated with Kaiser Permanente.