First Day of a ‘New Life’ for a Boy With Sickle Cell

WASHINGTON — There was supposed to be a special party for Kendric Cromer, 12, Wednesday, but it had to be postponed because he was too groggy to celebrate.

It was meant to mark the first day of his new life — the day he became one of the first children ever to be treated with a newly approved gene therapy that will free him from the sickle cell disease that has stolen his childhood.

On Wednesday, despite the excitement of the moment, Kendric was unable to keep his eyes open as he lay in his hospital bed at Children’s National Hospital in Washington because of the drugs he had been given in preparation for his treatment.

His life with the disease has been punctuated by episodes of excruciating pain, requiring days in the hospital as doctors tried to control it. Sickle cell eroded his hip bones. It prevented him from riding a bike or playing soccer or even going outside when the temperature was below 55 degrees Fahrenheit because cold often brought on intense pain.

Now he could see a future — in a month or so — without pain from sickle cell.

“I can’t wait to start my new life,” he told his mother, Deborah Cromer.

His disease is caused by an inherited genetic mutation that leads to blood cells that form crescent shapes — sickles — instead of discs. Trapped in blood vessels and organs, the cells cause damage and pain. Gene therapy fixes that problem by giving the patient a new, normal hemoglobin gene.

An estimated 100,000 people in the United States, most of them Black, have sickle cell disease.

Gene therapy dangles the prospect of normalcy for the estimated 20,000 people in the United States with the most severe forms of the disease — lives without constant pain and continuing damage to organs and bones and joints.

But all is not well in the world of sickle cell gene therapy.

In December, the Food and Drug Administration gave approval to two companies, Bluebird Bio of Somerville, Massachusetts, and Vertex Pharmaceuticals of Boston, to sell the first gene therapies approved for sickle cell disease. After nine months, Kendric remains the first Bluebird patient to progress this far, with at least a few others advancing toward his pace.

Doctors say that it is agonizingly slow to actually start treating patients.

The first step is for Vertex or Bluebird to approve a medical center to deliver the treatment — a process that involves negotiating complex contracts. Bluebird, which sells its therapy only in the United States, declined to disclose how many medical centers it had authorized. Vertex says it has more than 26 authorized centers in the United States and nine more in Europe, Britain and the Middle East.

“We all expected it to be much faster,” said Dr. Leo Wang of City of Hope Children’s Cancer Center in Los Angeles, which has so far sent cells from one patient to Vertex for the treatment and is in the final stages of getting authorization from Bluebird.

Another problem is capacity. The treatment is labor intensive, requiring patients to spend at least a month in the hospital. City of Hope can treat at least one patient a month, Wang said. Other large medical centers said they could treat only 10 or fewer per year, and some say they can treat just five or six.

Then there is insurance. Medical centers ask insurers for prior authorization. But even if they get it, hospital officials worry about how and when they will be paid.

“Authorization and reimbursement are not the same thing,” said Dr. Stephan Grupp at Children’s Hospital of Philadelphia, which has not yet started treating patients.

The hospital, he says, has to buy the treatment for $2.2 million per patient from Vertex or $3.1 million from Bluebird. It is reimbursed after the therapy is delivered to the patient. Hospitals get nervous, Grupp said, because they have to lay out a lot of money. “They want to see that reimbursement happen,” he said.

Some hospitals decided to treat one patient at a time, limiting how much they commit up front.

Making the process even more cumbersome, a hospital has to negotiate separately with each patient’s insurer, said Dr. Julie Kanter of the University of Alabama at Birmingham. Her medical center has not yet started treating patients.

Most hospitals, she said, “don’t want to approve the treatment until they know what the payment plan looks like.”

A key insurer in the state has not yet committed to paying, Kanter said.

“It’s unbelievably frustrating,” she said.

Kendric was lucky. His insurer was cooperative. He also rose to the top of the list because of the severity of his disease.

He has been through so much, as have his parents, Deborah Cromer, a real estate agent, and Keith Cromer, a federal employee. They stay in his room every time he is hospitalized. He sleeps on a chair and she on a narrow bench.

His treatment began in May when doctors removed his bone marrow stem cells. These immature cells are the source of all the body’s blood. Bluebird’s gene therapy adds a good hemoglobin gene to the cells.

After removing the stem cells, Kendric’s doctors shipped them to Bluebird’s facility in Allendale, New Jersey.

At that time, Bluebird estimated it would take three to six months before the treated cells would be ready.

The summer started well for Kendric. He volunteered as a counselor at an art camp. And he looked forward to visiting with relatives in South Carolina and spending time at the beach. The family was packed and ready to leave June 28, when Kendric called home from camp saying he had a terrible headache.

He turned out to have the bacterial infection MRSA, which is difficult to treat.

Although anyone can get such an infection, people with sickle cell are especially susceptible because their genetic disease damages the spleen, said Kendric’s doctor, Robert Nickel.

His parents unpacked their bags, canceled their vacation and moved into Kendric’s hospital room.

He was hospitalized for 10 days.

“We spent the Fourth of July here at the hospital,” Deborah Cromer said. He then spent six weeks at home on intravenous antibiotics.

Then the family got good news — Bluebird had completed its treatment of Kendric’s stem cells. The next step in his journey could begin.

On Sept. 5, he began four days of intense chemotherapy to clear his bone marrow, making way for his treated cells to repopulate his marrow.

The infusion began Wednesday. Kendric took Benadryl to forestall any possible allergic reaction to chemicals used to preserve his cells, but it made him so groggy he could hardly stay awake. He lay in bed with his Spider-Man blanket, a teddy bear and a little stuffed dog.

At 11 a.m. Kendric’s cells were wheeled up to the door of his room, 537 million of them in two small plastic bags of straw-colored liquid, frozen in liquid nitrogen. The hospital’s cellular therapy lab manager, Kathryn Bushnell, thawed the first bag in a pool of warm water. Then Kendric’s nurse, Rachel Purdie, started the infusion.

It took one minute, filling the room with a faint garlic smell from the preservative used to keep his cells viable. He slept through it. Then he got the second bag of cells, opening his eyes this time.

The cells will take up residence in his marrow and gradually divide and grow, re-creating his blood system. In a month, if all works as intended, he should be making blood cells that will not sickle — the cells will have a normal hemoglobin gene.

Kendric will stay in the hospital for a month. During that time he can expect to feel very ill with ulcerating sores in his mouth, throat and esophagus that will emerge about a week after his last infusion. Patients often are unable to eat and have to be fed with a nasogastric line. Children’s National has a laser wand patients can put in their mouth to help prevent the sores.

A big disappointment for Kendric is that he is missing school. He’s a straight-A student and says academics are his superpower. He will do his seventh-grade schoolwork remotely for a few months while he recovers from the chemotherapy and long hospitalization.

His parents are contemplating what life without sickle cell will be like.

“This is all we know,” Deborah Cromer said. “We will have to build a new way of life.”

“I have put all of my everything into caring for Kendric,” she added. “I’ve been thinking about how can I reinvent myself.”

As for Kendric, he wants to go to South Carolina for Thanksgiving to see his relatives. He wants to learn to ride a bike and to play basketball.

“I really just want to be a kid,” he said.

This article originally appeared in The New York Times .