Thanks to member SammyJo for this info.


By Michelle Cortez
January 10, 2019

https://www.bloomberg.com/news/artic...-under-new-law

Right to Try law backed by Trump allows experimental drug use
Closely-held Belgian company providing brain-cancer vaccine

A California man with a deadly form of brain cancer is receiving an experimental therapy under the so-called Right to Try law, the first publicly known use of the controversial legislation that was signed into law in May 2018 by President Donald Trump.

The treatment, called Gliovac, is a vaccine crafted from the patient’s own cells and tissue taken from other patients with glioblastoma -- the same aggressive tumor that killed John McCain a year after his diagnosis. It’s being provided to the man, who has chosen to remain anonymous for now, by Epitopoietic Research Corp., or ERC, a closely-held Belgian company that is testing it in human trials.

The Right to Try law has been touted by Trump and other conservative backers as a way to cut out regulators at the Food and Drug Administration, which have typically overseen patients’ use of experimental drugs outside clinical trials. Advocates say that it will give dying patients options, while critics say it will expose them to unknown side effects of little-studied therapies, without knowing if they have a reasonable chance of benefiting.

The patient’s family went to ERC to request the therapy after he didn’t qualify for the experimental study of Gliovac or any other trial that was underway, said Daniela Bota, the medical director at the University of California, Irvine, School of Medicine’s brain tumor program. Bota is also the lead investigator for the official trial of the drug, which is in the second of three stages of study typically required by the FDA before approval.

‘Death Sentence’
“For many patients with recurrent glioblastoma, there is nothing,” said Joe Elliott, managing director of ERC’s subsidiary in the U.S. “It’s a death sentence. Time is of the essence to get them into the program. We just want to help patients in these situations where they don’t have any other treatment options.”

So far about 25 to 30 patients have been treated with Gliovac, mainly in clinical trials and a compassionate use program in Europe. Many people in the U.S. don’t qualify for the study now underway in Irvine because of the severity of their condition or other unapproved treatments they have tried, Elliott said.

Bota declined to give any additional information about the patient, whom she is still treating. There is no definitive proof that the approach will work or offer any benefit. Early results suggest some patients may respond to the therapy, though the results thus far are all subjective, Elliott said.

Right to Try
“Patients come from all over the country to our program, and in this day and age many come knowing what is in our clinical trial portfolio,” Bota said. “We try to offer a study to every patient who qualifies. But when they don’t qualify, that’s when they have the option for Right to Try. It’s an easier way to get the treatment directly to the patient.”

ERC has received a handful of requests for access to Gliovac since Right to Try was passed. The California patient, who began treatment in November, is the first to gain access, Elliott said.

The passage of Right to Try drew criticism from numerous sides, including some patient groups, drug companies and medical ethicists, who said efforts to supply an individual with an unapproved medicine could slow down development that could one day benefit many more people. The company said it wasn’t concerned about that.

There haven’t been any serious side effects from use of the vaccine, said Elliott. The FDA and other regulators will base approval decisions on the results of the clinical trial program that is currently underway and slated for an expansion, he said. It should be concluded in the next 18 months, he said.

‘No Options Left’
“For these patients, who really have no options left, therapeutically the risk to receive it is very low,” he said. “The potential benefit is higher but certainly no worse than if they didn’t receive the vaccine at all.”

Bota presented an initial analysis of her study at a medical meeting in November, showing patients given the vaccine in addition to Roche Holding AG’s targeted treatment Avastin had better overall survival than those given Avastin alone. The findings, involving only a handful of patients, must still be confirmed with larger studies.

While Elliott declined to comment on how much it costs to manufacture the therapy for each patient, he said it could run as high as other approved cancer medicines that cost between $373,000 and $475,000 per patient. The company isn’t charging the California patient.