A Laval family seeking nearly a million dollars for a last-ditch therapy offered in New York in an attempt to combat a young man’s aggressive blood cancer has raised over $310,000 in less than five days via crowdfunding. But who, in fact, recommended this treatment?
Touched by the story of Matthew Schreindorfer, 25, who is suffering from a deadly form of leukemia, donations began pouring in last Thursday within minutes of the family posting a “help save Matthew” campaign on YouCaring.com, a free online fundraising website.
The experimental gene therapy, called CART-19, is so new that it has been tested in only a handful of people in clinical trials at a few centres including at Memorial Sloan Kettering Cancer Center in New York. The treatment manipulates the patient’s own immune system to recognize and kill their own cancer cells.
Schreindorfer’s medical team in Montreal and New York say they cannot discuss the case, citing patient confidentiality.
“His oncologist said it would be a last resort treatment” to save his life, said Katia Luciani in a telephone interview from her husband’s bedside. Hours before the fundraising site went live, Schreindorfer was rushed to a Laval hospital emergency room with severe cramps, fever and pain.
He’s under an oxygen tent, taking morphine and is in no state to speak to anyone, Luciani said. “We’re not certain what the problem is. It’s overwhelming … crazy here and we’re trying to cope.”
Hematologist Jean-Sébastian Delisle, a principle investigator at Maisonneuve-Rosemont Hospital Research Centre, who is not involved in the case, said CART-19 is extremely promising, and perhaps “one of the most important discoveries of the century.”
“But that said, it’s very new and it’s hard to distinguish hype from hope” about success rates pegged at 90 per cent, said Delisle, an assistant professor of medicine at Université de Montréal.
“That’s too optimistic,” Delisle said, “there’s too few patient follow-up beyond one year, or one-and-a-half years. Success is assessed at five years. Most will relapse within two years. You can’t quote 90 per cent success until you’ve followed enough people for enough time.”
Because of intellectual property rights to the gene therapy, CART-19 is not available yet in Canada or Europe, however Novartis pharmaceuticals, which owns the rights, is planning international trials within two years.
Conventional therapy for leukemia is bone-marrow transplant. But patients must be in remission where the cancer is not detectable to go that route.
In the past six months, Schreindorfer tried several rounds of intensive chemotherapy and, when that failed, a local clinical trial — but nothing worked. Now the family is hoping the treatment in the New York will hold the key to his remission.
Clear that he is not discussing this case, Delisle said that patients who have exhausted conventional therapies at his hospital will often be referred to experimental trials held by colleagues in Montreal.
“For the very motivated, who ask frankly, ‘What are my best chances?’ we don’t hesitate to look elsewhere. In exceptional cases, we refer patients to the United States. And we help them with the process,” Deslisle said. But it’s based on a case by case evaluation. “It’s not a standard referral.”
“Often people are raising money for a slim chance at a cure and everyone must be aware of that.”
High school sweethearts, Schreindorfer and Luciani married last summer and had just returned from their honeymoon in June, ready to start their lives together in their new home, when illness struck. Schreindorfer came down with flu-like symptoms. Doctors initially believed he had caught a virus overseas, then mononucleosis, but further tests revealed something far more serious, acute lymphoblastic leukemia, an aggressive blood cancer. The family was in shock. Tears flowed.
“It’s been a real emotional roller coaster,” Luciani said. “He was always in shape, a hockey player and never sick. To hear it was cancer — it was just so shocking. But he took it like a speed bump on the road of life.”
But it would turn out to be the first bump of many. In October, Schreindorfer raised $40,000 for the Light the Night leukemia fundraiser. He was too sick to walk. Friends and family walked on his behalf.
Luciani said her husband’s only option now is Sloan Kettering, which requires a deposit of $675,000, and costs could go as high as $800,000. The price tag covers transportation, hospital stay, scans, blood tests and other medical costs associated with treatment in an American centre.
The family is on the hook for the entire amount as the provincial health insurance board does not cover costs of unrecognized or experimental therapies. The province will pay for special services covered under public health that are not available locally, providing two medical experts agree that the patient needs it, explained Caroline Dupont, spokesperson for the Régie de l’assurance maladie du Québec (RAMQ).
“We are sympathetic … but we can’t make an exception,” Dupont said.
Meanwhile, Schreindorfer will not be able to travel to New York until he is medically stable. Luciani says she’s trying to stay positive. Friends and family contributed their own money and the response to the online campaign has been overwhelming, she said.
“I didn’t think it would go that big,” she said. “It shows how he touched a lot of people, just being himself, even before he got sick.”
cfidelman@montrealgazette.com
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What is Acute Lymphoblastic Leukemia (ALL)?
Also called acute lymphocytic leukemia, it is an aggressive cancer of the blood and bone marrow, the spongy material where blood cells are made. ALL attacks the production of mature healthy blood cells. Red carry oxygen, white are crucial to fighting infection, and platelets play role in clotting and wound healing. A subset of diseased immature cells, called leukemia or B cells, in the bone marrow reproduce far too rapidly. As their numbers increase, they squeeze out normal, healthy blood cells, leading to infection, anemia and easy bleeding. Symptoms of the disease initially resemble flu or virus: fever, headache, fatigue, no appetite, frequent infections, and bone and joint pain. It is one of the most common form of childhood cancers, however up to 85 per cent survive; adults have a much poorer prognosis. A percentage of people remain resistant to intense treatment.
What is known about the CART-19 trial?
It is a United States government-funded experimental immunotherapy trial for advanced leukemia. It genetically modifies the patient’s own white blood cells in the laboratory with an artificial receptor (chimeric antigen receptor, or CAR). These re-engineered T-cells are then infused back into the patient where they recognize and attack CD19 protein, a hallmark of cancerous cells. In 2011, the Children’s Hospital of Philadelphia, Pa., began recruiting children with a deadly form of leukemia that failed to respond to existing medicines. Phase I of the trial had 22 children and five adults. According to the latest statistics available from 2013, 89 per cent showed remission where the disease is undetectable, but six of the people who initially responded well have since relapsed. Phase II of the trial was enlarged to include other sites, including New York and Maryland. It’s not known whether CART-19 is sufficient to get rid of the disease. Meanwhile, the treatment is considered a bridge to bone marrow transplant to replace a faulty immune system with a healthy donor’s.
Sources: The Leukemia & Lymphoma Society of Canada, Memorial Sloan Kettering Cancer Center, Leukemia & Lymphoma Society, U.S., Information Centre, National Cancer Institute at the National Institutes of Health.