Highlights
- A gene therapy called Kymriah developed for cancer is transformative in treating a certain type of cancer in children and young adults
- Kymriah gene therapy uses a patient's own immune cells to attack the cancer cells
- Kymriah will be available in only 16 states at certified treatment centers and costs about $475,000 for a one-time infusion
In Small Trial, Novel Gene Therapy for Cancer Pain Proves Successful
A novel gene therapy treatment successfully reduced pain symptoms in patients with cancer pain, researchers from the University of Michigan Department of Neurology have reported.
A novel gene therapy treatment successfully reduced pain symptoms in patients with cancer pain, researchers from the University of Michigan Department of Neurology have reported.
‘Chimeric antigen receptor (CAR) T cell treatment works by genetically reengineering a patient's T cells and white blood cells in the immune system, particularly in children and young adults.’
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However, this gene therapy called Kymriah (tisagenlecleucel), and also the ones similar to this raise difficult challenges for policy-makers. Karen Maschke, the first author, said that safety, access, and the cost has to be figured-out if everybody is going to benefit from the therapy.
Maschke, Michael Gusmano, and Hastings Center president Mildred Solomon describe the breakthrough, evaluate the challenges, and make recommendations in the October issue of Health Affairs.
Molecular Tumor Board Helpful in Advanced Cancer Cases
Proponents of personalized cancer treatments matched to a patient's DNA sequencing, say that physicians increasingly need help to maneuver through the genomes to find therapy options.
Proponents of personalized cancer treatments matched to a patient's DNA sequencing, say that physicians increasingly need help to maneuver through the genomes to find therapy options.
Chimeric antigen receptor (CAR) T cell treatment works by genetically reengineering a patient's T cells and white blood cells in the immune system. The T cells which are altered are later infused into the patient to attack the cancer cells.
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Gene Therapy Method Reverses Immune Response in Multiple Sclerosis
A unique gene therapy using the vector adeno-associated virus (AAV) immunotherapy has been successful in reversing immune responses in multiple sclerosis.
A unique gene therapy using the vector adeno-associated virus (AAV) immunotherapy has been successful in reversing immune responses in multiple sclerosis.
The clinical trials conducted by Novartis showed that 52 patients out of 63 were in remission for three months after they have received a one-time infusion.
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India�s Struggle With Thalassemia: Can Only Gene Therapy Offer Complete Cure?
The life and struggle of a person who suffers from beta thalassemia major, a rare genetic blood disorder, seen through the eyes of Gagandeep Singh Chandok
The life and struggle of a person who suffers from beta thalassemia major, a rare genetic blood disorder, seen through the eyes of Gagandeep Singh Chandok
"The FDA and Institutional Review Boards (IRBs) with oversight over human subjects research must carefully scrutinize [the risk-benefit profile] to determine whether the potential benefits of the investigational treatment justifies permitting cancer patients to enroll in a study," reveals the research team, as they continue to test the CAR T cell cancer treatments in clinical trials before obtaining FDA approval.
The research team also recommend the IRBs to mention all the possible adverse effects in the consent forms and later the FDA can conduct post-licensing monitoring of the adverse events, which the patients experience.
The agency requests Novartis to conduct a postmarketing study on the safety and efficacy of Kymriah.
"However, it is an open question whether the FDA, under the administration of President Donald Trump, will withdraw licensing approval from breakthrough therapy products whose clinical use over time indicates that they are ineffective or harmful to patients," they write.
Accessibility of Kymriah
The research team raises concern that most patients will not be able to access the therapy for a while even though they could benefit from it, it needs highly specialized skill to genetically reengineer the patients' T cells, administer the infusion, and treat severe side effects.
Kymriah will be available in only 16 states at certified treatment centers, all but four with just one treatment site.
The authors write, "Having fewer sites with highly trained clinical staff treating very sick patients is often optimal for ensuring high-quality delivery of sophisticated therapies, but at least at first there will be a severe access challenge."
Cost of Kymriah
The problem of availability can be resolved. However, the cost of treatment still poses important questions of justice, explains the article.
Kymriah will cost about $475,000 for a one-time infusion, and FDA approval of this gene therapy does not guarantee if the insurers would cover it.
The authors also write that if CAR T cell therapies are proven to be as safe and efficient as other recent breakthrough treatments, the benefits will be tremendous, and the demand will be high.
"However as has been the case with other recent beneficial but high-priced drugs, CAR T cell therapies may lead to higher health care costs, highly limited access, and even greater inequalities in access and health outcomes. For the public to benefit from breakthrough treatments without breaking Americans' health care system or pocketbooks, ethics and wise policymaking need to catch up with the science."
Reference
- Karen J. Maschke, Michael K. Gusmano and Mildred Z. Solomon. Breakthrough Cancer Treatments Raise Difficult Questions. Health Affairs, (2017).
Source-Medindia
