A woman whose liver disease can’t be treated by conventional medicine, is receiving treatment at a California hospital using experimental “compound” grown from her own genes.
Doctors at UCSF Benioff Children’s Hospital Oakland performed a series of blood tests earlier this year and discovered that the 55-year-old woman was the first human ever to have a genetically engineered organ successfully implanted, according to Dr. Keith Ayoob, one of the hospital’s chief medical officers.
Ayoob, chief medical officer of the hospital, told The Washington Post in an email that the patient was at UCSF Benioff Children’s Hospital Oakland “for a highly curative process [stem cell transplant] but we tested a drug called TC-3700 that received a provisional approval from the FDA. This drug is a compound made from DNA in which the patient’s particular genetic disease is mutated” and is developed using cells taken from the patient.
He called it “a breakthrough in regenerative medicine.”
“Ultimately, the hope is that this process can be used broadly and almost uniformly as a way to treat a wide range of (genetically defined) diseases,” he added.
As of Wednesday afternoon, the patient’s name was not publicly available, or even identified as a woman in a press release issued by the hospital Wednesday, in which she was identified only as a 55-year-old patient with mesothelioma.
In the release, which urged women around the country to be tested for such illnesses, she said the drug had “turned my life around,” saving her ability to walk and “gain my independence.”
I am very grateful to all those involved in my care at UCSF Benioff Children’s Hospital Oakland. — Jennifer — YR — [No proper spelling]
David Maki, the patient’s friend, told The Post that she had no specific prognosis.
“It all depends on how well her stem cells do after transplant and whether her her repair mechanisms work,” Maki said. “She’s an unusual person for sure.”
As CNN reported, TC-3700, a small molecule, was put into the patient’s bloodstream following a series of blood tests that revealed an “alarming” tissue death in an organ that could not be transplanted.
“The drug does not alter the patient’s genes so it is not cancer related. But when cells die in tissue, it is cancer,” CNN reported, citing a publication from the American Society of Hematology.
“There are no specific treatments for these malignancies, and studies of gene therapy are ongoing,” according to an ABC News report from 2014.
Ayoob would not answer questions about the details of the process or the patient’s response, including when the test results indicated that the new drug was working.
However, he told CNN that a “couple of stem cell transplant events occurred” but declined to elaborate.
The patient, who was diagnosed with mesothelioma, is not alone.
Though Ayoob said the hospital has had other positive results with this process, he refused to say how many patients the hospital was treating, or whether the hospital has put the approach into use for a broader number of potential patients.
“That is a matter for privacy and safety,” he said in an email.
Ayoob said he could not say how successful the process had been, but that one of the patients the hospital had treated had a “very good experience,” leading doctors to proceed with “the proposed follow-up plan.”
NBC Bay Area, citing multiple media accounts, reported that the woman was living independently after the transplant. It also said the drug had been approved by the Food and Drug Administration for use in individuals with cancers related to the mesothelioma.
As well as the planned treatment of the woman’s own stem cells, the hospital said it has worked with the California Institute for Regenerative Medicine, which received approval from the state government to explore potential treatments and restore patient rights for the process.
“I am very grateful to all those involved in my care at UCSF Benioff Children’s Hospital Oakland,” the patient said in the hospital release. “As long as I am involved in this process, I will continue to exercise my right to be fully informed about how I am treated.”