Doctors have reported unprecedented success from a new cell and gene therapy for multiple myeloma. The study however is small ̶ 35 people ̶ and although early, all patients responded and all except two were in some level of remission within two months.

A follow-up second study of nearly two dozen patients showed that everyone above a certain dose responded.

The study was revealed at the American Society of Clinical Oncology conference in Chicago and experts say it is a first for multiple myeloma and rare for any cancer treatment to have such success.

Chemotherapy helps 10 to 30% of patients; immune system drugs, 35 to 40% at best, and some gene-targeting drugs, 70 to 80%, “but you don’t 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 added.

First CAR-T therapy for multiple myeloma

The treatment, called CAR-T Therapy, filters a patient's blood to remove T cells before they are genetically altered to target cancer. The altered T cells are then returned to the patient intravenously.

Previous attempts have shown promise against some types of leukaemia and lymphomas, but this is the first time it is being tried for multiple myeloma.

The study by Chinese company Nanjing Legend Biotech done with doctors at Xi’an Jiaotong University, saw 19 of 35 patients who are long past treatment to judge whether they are in complete remission ̶ 14 are. Five have at least a partial remission, with their cancer greatly diminished. For some, there has been no sign of disease for more than a year past treatment.

Like other CAR-T therapies, most patients had side effects such as fever, low blood pressure and breathing troubles. Only two cases were severe and all were treatable and temporary, the doctors said.

The second study done by Bluebird Bio and Celgene in the US, used a cell treatment developed by the National Cancer Institute. It tested four different dose levels of cells in a total of 21 patients.

18 were long enough from treatment to judge effectiveness and all 15 who received an adequate amount of cells responded. Four have reached full remission with some more than a year past treatment.

Future plans to provide hope for a cure for multiple myeloma

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.

Legend Biotech plans to further study 100 people in China and conduct another study in the US early next year. However, the treatment is expected to cost USD200,000 to USD300,000 and "who's going to pay for that is a big issue," said Dr Frank Fan, chief scientific officer of Legend Biotech.

Another obstacle for the treatment is that the manufacturing process is very expensive as it is individualised and cannot be scaled up to make batches as is done with a drug.

But with the cell therapy, Dr Fan said, "I can't say we may get a cure, but at least we bring hope of that possibility."

Juno's second-chance CAR-T treatment shows positive results

Separately, Juno posted new data at ASCO showing positive data for its second-chance CAR-T treatment. Last year, its once-leading CAR-T treatment JCAR015 saw a series of deaths before it was halted at the beginning of this year.

Although a death and some neurotoxicity were still present in the current data set, JCAR017 saw an overall 86% response rate and a complete response rate of 59% ̶ 26 out of 44 patients.

Juno remains encouraged by the high rates of durable responses and early survival data in the patients.

"We are also encouraged by the early safety data ̶ a majority of patients treated experienced no cytokine release syndrome or neurotoxicity of any grade, which suggests the potential for outpatient administration," said Sunil Agarwal, Juno's president of research and development. MIMS

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