In one of the biggest advances against leukemia and other blood cancers in many years, doctors are reporting unprecedented success by using gene therapy to transform patients’ blood cells into soldiers that seek and destroy cancer.
A few patients with one type of leukemia were given this one-time, experimental therapy several years ago and some remain cancer-free today. Now, at least six research groups have treated more than 120 patients with many types of blood and bone marrow cancers, with stunning results.
“It’s really exciting,” said Janis Abkowitz, blood diseases chief at the University of Washington in Seattle and president of the American Society of Hematology. “You can take a cell that belongs to a patient and engineer it to be an attack cell.”
In one study, all five adults and 19 of 22 children with acute lymphocytic leukemia (ALL), had a complete remission, meaning no cancer could be found after treatment, although a few have relapsed since then.
These were gravely ill patients out of options. Some had tried multiple bone marrow transplants and up to 10 types of chemotherapy or other treatments.
Cancer was so advanced in eight-year-old Emily Whitehead of Philipsburg, Pennsylvania, that doctors said her major organs would fail within days. She was the first child given the gene therapy and shows no sign of cancer today, nearly two years later.
Results on other patients with myeloma, lymphoma and chronic lymphocytic leukemia (CLL), will be reported at the hematology group’s conference that started on Saturday in New Orleans.
Doctors say this has the potential to become the first gene therapy approved in the US and the first for cancer worldwide. Only one gene therapy is approved in Europe, for a rare metabolic disease.
The treatment involves filtering patients’ blood to remove millions of white blood cells called T-cells, altering them in the lab to contain a gene that targets cancer, and returning them to the patient in infusions over three days.
“What we are giving essentially is a living drug” — permanently altered cells that multiply in the body into an army to fight the cancer, said David Porter, a University of Pennsylvania scientist who led one study.
Several drug and biotech companies are developing these therapies. Penn has patented its method and licensed it to Switzerland-based Novartis AG. The company is building a research center on the Penn campus in Philadelphia and plans a clinical trial next year that could lead to federal approval of the treatment as soon as 2016.
Nearly 49,000 new cases of leukemia, 70,000 cases of non-Hodgkin lymphoma and 22,000 cases of myeloma are expected to be diagnosed in the US this year.
Many patients are successfully treated with chemotherapy or bone marrow or stem cell transplants, but transplants are risky and donors cannot always be found. So far, gene therapy has been tried on people who were in danger of dying because other treatments had failed.
The gene therapy must be made individually for each patient, and lab costs now are about US$25,000, without a profit margin. That is still less than many drugs to treat these diseases and far less than a transplant.
The treatment can cause severe flu-like symptoms and other side effects, but these have been reversible and temporary, doctors say.
Penn doctors have treated the most cases so far — 59. Of the first 14 patients with CLL, four had complete remissions, four had partial ones and the rest did not respond. However, some partial responders continue to see their cancer shrink a year after treatment.
“That’s very unique to this kind of therapy” and gives hope the treatment may still purge the cancer, Porter said.
Another 18 CLL patients were treated and half have responded so far.
Penn doctors also treated 27 ALL patients. All five adults and 19 of the 22 children had complete remissions, an “extraordinarily high” success rate, said Stephan Grupp at the Children’s Hospital of Philadelphia.
Six have since relapsed, though, and doctors are pondering a second gene therapy attempt.
At the National Cancer Institute, James Kochenderfer and others have treated 11 patients with lymphoma and four with CLL, starting roughly two years ago. Six had complete remissions, six had partial ones, one has stable disease and it is too soon to tell for the rest.
Ten other patients were given gene therapy to try to kill leukemia or lymphoma remaining after bone marrow transplants. These patients got infusions of gene-treated blood cells from their transplant donors instead of using their own blood cells. One had a complete remission and three others had significant reduction of their disease.
An endangered baby pygmy hippopotamus that shot to social media stardom in Thailand has become a lucrative source of income for her home zoo, quadrupling its ticket sales, the institution said Thursday. Moo Deng, whose name in Thai means “bouncy pork,” has drawn tens of thousands of visitors to Khao Kheow Open Zoo this month. The two-month-old pygmy hippo went viral on TikTok and Instagram for her cheeky antics, inspiring merchandise, memes and even craft tutorials on how to make crocheted or cake-based Moo Dengs at home. A zoo spokesperson said that ticket sales from the start of September to Wednesday reached almost
‘BARBAROUS ACTS’: The captain of the fishing vessel said that people in checkered clothes beat them with iron bars and that he fell unconscious for about an hour Ten Vietnamese fishers were violently robbed in the South China Sea, state media reported yesterday, with an official saying the attackers came from Chinese-flagged vessels. The men were reportedly beaten with iron bars and robbed of thousands of dollars of fish and equipment on Sunday off the Paracel Islands (Xisha Islands, 西沙群島), which Taiwan claims, as do Vietnam, China, Brunei, Malaysia and the Philippines. Vietnamese media did not identify the nationalities of the attackers, but Phung Ba Vuong, an official in central Quang Ngai province, told reporters: “They were Chinese, [the boats had] Chinese flags.” Four of the 10-man Vietnamese crew were rushed
CHINESE ICBM: The missile landed near the EEZ of French Polynesia, much to the surprise and concern of the president, who sent a letter of protest to Beijing Fijian President Ratu Wiliame Katonivere called for “respect for our region” and a stop to missile tests in the Pacific Ocean, after China launched an intercontinental ballistic missile (ICBM). In a speech to the UN General Assembly in New York on Thursday, Katonivere recalled the Pacific Ocean’s history as a nuclear weapons testing ground, and noted Wednesday’s rare launch by China of an ICBM. “There was a unilateral test firing of a ballistic missile into the Pacific Ocean. We urge respect for our region and call for cessation of such action,” he said. The ICBM, carrying a dummy warhead, was launched by the
As violence between Israel and Hezbollah escalates, Iran is walking a tightrope by supporting Hezbollah without being dragged into a full-blown conflict and playing into its enemy’s hands. With a focus on easing its isolation and reviving its battered economy, Iran is aware that war could complicate efforts to secure relief from crippling sanctions. Cross-border fire between Israel and Hezbollah, sparked by Hamas’ attack on Israel on Oct. 7 last year, has intensified, especially after last week’s sabotage on Hezbollah’s communications that killed 39 people. Israeli airstrikes on Hezbollah strongholds in Lebanon followed, killing hundreds. Hezbollah retaliated with rocket barrages. Despite the surge in
RSS