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New Gene Therapy for Cancer Brings Hope and Challenges

New Gene Therapy for Cancer Brings Hope and Challenges

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  • 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

The first ever gene therapy developed for cancer can transform the treatment of a certain type of cancer in children and young adults and has been approved by the Food Drug Administration in August, reveals a new study.

The gene therapy is transformative, as it uses a patient's immune cells to attack the cancer cells. This drug is the first of many other drugs, which is capable of utilizing a patient's immune system.


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.

Novartis developed this new therapy, which helps to treat children and young adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL).

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.

Safety of Kymriah

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.

However, about 76 percent of patients experienced life-threatening neurological and other side effects caused by this gene therapy, which increased the risk of patients developing secondary cancers.

"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."

  1. Karen J. Maschke, Michael K. Gusmano and Mildred Z. Solomon. Breakthrough Cancer Treatments Raise Difficult Questions. Health Affairs, (2017). DOI: 10.1377/hlthaff/2017.1032

Source: Medindia

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