A first: All respond to CAR-T therapy in a blood cancer study – STAT

C

HICAGO Doctors are reporting unprecedented success from a new cell and gene therapy for multiple myeloma, a blood cancer thats on the rise. Although its early and the study is small 35 people every patient responded and all but two were in some level of remission within two months.

In a second study of nearly two dozen patients, everyone above a certain dose responded.

Experts at an American Society of Clinical Oncology conference in Chicago, where the results were announced Monday, say its a first for multiple myeloma and rare for any cancer treatment to have such success.

advertisement

Chemotherapy helps 10 to 30 percent of patients; immune system drugs, 35 to 40 percent at best, and some gene-targeting drugs, 70 to 80 percent, but you dont get to 100, said Dr. Len Lichtenfeld, deputy chief medical officer of the American Cancer Society.

These are impressive results, but time will tell if they last, he said.

Meet the biotech company trying to shake up how the FDA thinks about cancer drugs

Multiple myeloma affects plasma cells, which make antibodies to fight infection. More than 30,000 cases occur each year in the United States, and more than 115,000 worldwide. Its the second fastest growing cancer for men and the third for women, rising 2 to 3 percent per year, according to the National Cancer Institute. About 60,000 to 70,000 Americans have it now.

Nine new drugs have been approved for it since 2000 but theyre not cures; only about half of U.S. patients live five years after diagnosis.

With cell therapy, I cant say we may get a cure, but at least we bring hope of that possibility, said Dr. Frank Fan. He is chief scientific officer of Nanjing Legend Biotech, a Chinese company that tested the treatment with doctors at Xian Jiaotong University.

The treatment, called CAR-T therapy, involves filtering a patients blood to remove immune system soldiers called T cells. These are altered in a lab to contain a gene that targets cancer and then given back to the patient intravenously.

Doctors call it a living drug a one-time treatment to permanently alter cells that multiply in the body into an army to fight cancer. Its shown promise against some leukemias and lymphomas, but this is a new type being tried for multiple myeloma, in patients whose cancer worsened despite many other treatments.

In the Chinese study, 19 of 35 patients are long enough past treatment to judge whether they are in complete remission, and 14 are. The other five had at least a partial remission, with their cancer greatly diminished. Some are more than a year past treatment with no sign of disease.

Most patients had a group of side effects common with this treatment, including fever, low blood pressure, and trouble breathing. Only two cases were severe and all were treatable and temporary, doctors said.

The second study was done in the U.S. by Bluebird Bio and Celgene, using a cell treatment developed by the National Cancer Institute. It tested four different dose levels of cells in a total of 21 patients. Eighteen are long enough from treatment to judge effectiveness, and all 15 who got an adequate amount of cells had a response. Four have reached full remission so far, and some are more than a year past treatment.

The results are very remarkable, not just for how many responded but how well, said Dr. Kenneth Anderson of Dana-Farber Cancer Institute in Boston.

We need to be looking for how long these cells persist and keep the cancer under control, he said.

Dr. Carl June, a University of Pennsylvania researcher who received the conferences top science award for his early work on CAR-T therapy, said its very rare to see everyone respond to a treatment. His lab also had this happen all 22 children testing a new version of CAR-T for leukemia responded, his colleagues reported at the conference.

U.S. could save $825 million a year with a small change in immunotherapy dosing, study says

The first patients we treated in 2010 havent relapsed, June said.

Dr. Michael Sabel of the University of Michigan called the treatment revolutionary.

This is really the epitome of personalized medicine, extending immune therapy to more types of patients, he said.

Legend Biotech plans to continue the study in up to 100 people in China and plans a study in the U.S. early next year. The treatment is expected to cost $200,000 to $300,000, and whos going to pay for that is a big issue, Fan said.

The manufacturing process is very expensive and you cant scale up. Its individualized. You cannot make a batch as is done with a drug, he said.

Nick Leschly, Bluebirds chief executive, said the next phase of his companys study will test what seems the ideal dose in 20 more people.

Marilynn Marchione

Trending

How epigenetic changes prime pancreatic cancer cells to spread

How epigenetic changes prime pancreatic cancer cells to spread

Illumina says it can deliver a $100 genome

Illumina says it can deliver a $100 genome soon

Googles bold bid to transform medicine hits turbulence under

Googles bold bid to transform medicine hits turbulence under a divisive CEO

Recommended

Identifying another of cancers wily tricks: Masking pain so

Identifying another of cancers wily tricks: Masking pain so tumors can grow unnoticed

Even moderate drinking may speed brain decline

Even moderate drinking may speed brain decline

Study finds pregnancy seems safe for breast cancer survivors

Study finds pregnancy seems safe for breast cancer survivors

Read more:
A first: All respond to CAR-T therapy in a blood cancer study - STAT