CT researchers explore gene therapy that shows promise to slow aging, fight cancer
· Yahoo NewsGetting old goes much deeper than wrinkles and gray hair - many of the cells in your body are literally wearing out due to age. So what if your own immune system could be harnessed to sweep out aging cells and restore youth from the inside out?
That's one potential use of a class of gene therapies that are quickly moving from research labs at Jackson Laboratory in Farmington and clinical trials at Yale University to doctor's offices across the state.
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"Science fiction ideas" like curing cancer with the help of engineered viruses are now becoming standard treatments, said Dr. Derya Unutmaz, a professor of immunology and researcher at the Jackson Laboratory for Genomic Medicine in Farmington. In the last few years, AI has also jump-started innovation and brought the technology into new realms, he said.
"We're focused on curing aging, you know, making people young again," Unutmaz citing recent experiments in mice that have eliminated aged and damaged cells. "AI is facilitating at every level."
The technology used in the aging experiments, CAR T-cell therapy, has shown such promise in fighting cancer and autoimmune diseases that it has drawn the attention of Connecticut lawmakers this year.
"The challenge before us is no longer whether these therapies work, but whether every patient will be able to access them in time, regardless of where they live," said state Senator Saud Anwar, D-South Windsor, who introduced a new bill. Anwar, a doctor who specializes in lung diseases, said he drafted the legislation at the recommendation of a national scientific group in an effort to ensure access and affordability for the new treatments.
On Monday, the legislature's Public Health Committee is set to vote on the bill, which would set up a new advisory council on CAR T-cell therapy. The group would advise public health officials and other state agencies on how to expand access to the treatment, which has shown success in curing some forms of blood cancer.
"Gene therapy and CAR T therapy are no longer experimental ideas. They are already saving and transforming lives in rare and deadly diseases such as leukemia, sickle cell disease, spinal muscular atrophy and hemophilia," Anwar said.
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At a forum on rare diseases in Farmington on Tuesday, Jackson Laboratory for Genomic Medicine CEO Lon Cardon spoke of personalized gene therapy treatments that can move from the lab to the patient within months.
"That's not an incremental change, that is a sea change," Cardon said. At Jackson Laboratory alone, researchers are taking part in 300 different preclinical studies and 90 programs for different rare diseases, he said.
Immune cells altered to fight cancer
A type of immunotherapy, CAR T-cell therapy traces its roots to a doctor's observations in the 1920s that patients infected by tuberculosis were less likely to suffer from cancer, leading to the discovery that an immune system mobilized by bacterial infections can also block the growth of cancer cells. By the 1970s, vaccines made from bacteria that activated the immune system were standard treatment for types of bladder cancer.
Work on the biology of the AIDS virus in the 1990s opened up another path - engineering viruses to alter immune system T-cells, reprogramming them to kill cancer cells, said Unutmaz, who took part in some of the early research. That research led to CAR T-cell therapy, which has been proven highly effective against some types of cancers of the blood like lymphoma, myeloma and leukemia. Now the treatments are being tested for autoimmune conditions like lupus, caused when immune system components called B-cells go rogue.
"If you can kill those B-cells, you kind of reset the immune system," Unutmaz said. "And so these people are getting cured."
Another type of gene therapy based on muting defective genes was the focus of the keynote talk at the rare disease forum at Jackson Laboratory.
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Gene-editing technology like CRISPR has unlocked new approaches to treatment, said Dr. Timothy Yu, a researcher at Harvard and Boston Children's Hospital. The FDA in February outlined a streamlined approach to speed approvals for innovative gene therapies, sparking hope for the rapid introduction of new cures in coming years.
"We can now deliver therapies and now begin to develop these approaches to not just read out from the genome, but write back to the genome and influence the way that the genome works," Yu said.
More CT patients get gene therapy
With many uses of CAR T-cell and other gene therapies still in the clinical trial phase, most of the patients in Connecticut benefiting from the technology right now are patients with forms of blood cancer, said Dr. Micheal Hurwitz, a cancer researcher and associate professor of medicine at Yale. The therapy has also been approved for a rare type of sarcoma, or cancer of the soft tissue.
About 150 patients have been treated with CAR T-cell therapy at Yale in recent years and more are part of clinical trials run by the university, Hurwitz said. Intensive research is also being done to lessen the potentially serious side effects of treatment.
Newer trials are looking at combination therapies and the use of other immune system components to kill a wider range of cancers.
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"If we can get more advances like that, then that opens this up to many, many, many different cancers," Hurwitz said. "There will be different cells being used, using it for non-cancers, for immune issues… that's going to become huge, honestly."
Hartford HealthCare offers CAR T-cell therapy as part of its partnership with Memorial Sloan Kettering Cancer Center, said Dr. Peter Yu, physician-in-chief of the Hartford HealthCare Cancer Institute.
"Our processes allow rapid referral and patient appointments at MSK," Yu said. " We have a dedicated nurse navigator to assist with coordination of care at MSK and at HHC during the treatment and follow-up."
Cost limits access to innovations
Hurwitz said he appreciates the legislature's CAR T-cell advisory council bill as part of efforts to give more patients access to the therapy. Due to the treatment's cost and high demands of patients - like a 24-hour caregiver at home in the month after treatment - relatively few people can take advantage of CAR T-cell therapy right now, he said.
The engineered cells alone needed for CAR T-cell therapy cost up to $475,000 per patient due to the intensive lab work needed, according to the American Cancer Society. The price of hospital admission, tests, procedures and other expenses boosts total costs over $500,000 for most patients.
"While Medicare and many commercial insurances do cover some of the costs of CAR T-cell therapy, they usually don't cover the full amount," the cancer group said.
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The treatments are also currently limited to major medical centers, and patients need to stay nearby to be monitored.
"If you have the means, you get treatments that we know can be life-saving, and if you don't have the means, you just may not be able to get it," Hurtwitz said. "Government should know about this, and they should know about this huge disparity, and ideally, maybe they could do something about it."
Connecticut residents can also benefit from learning more about the treatments and advocating for more research into CAR T-cell and other advanced therapies in a time of federal cutbacks, Hurwitz said.
"Raising the profile of anything that's experimental is important so that people understand how important research is to getting treatments to cure people," Hurwitz said. "These are the kind of things that are going to eventually result ideally in people living a lot longer and better."
This article originally published at CT researchers explore gene therapy that shows promise to slow aging, fight cancer.