Biotech’s Breakthrough Year

Medical advances are accelerating, and even the FDA approves.


Pay attention only to politics and you might think 2017 was a parade of horribles, yet most Americans saw their living standards rise and business innovations are happening apace. Consider the lifesaving medical breakthroughs greenlighted this year by the Food and Drug Administration.

Last week the FDA approved the novel gene therapy Luxturna to treat patients with hereditary retinal dystrophy, a degenerative disease that can lead to blindness. Luxturna, developed by Spark Therapeutics, is the first gene therapy approved in the U.S. that targets a disease caused by a specific gene mutation.

The retinal disorder can be caused by mutations in more than 220 genes, but scientists identified RPE65 as a culprit for between 1,000 and 2,000 U.S. patients. RPE65 provides the coding that programs an enzyme necessary to convert light in retinal cells. If there’s an error in RPE65, the enzyme isn’t produced.

Luxturna uses a virus as a vehicle to insert a normal copy of the gene directly into a patient’s retinal cells. According to the FDA, patients who received Luxturna in a trial “demonstrated significant improvements in their ability to complete [an] obstacle course at low light levels as compared to the control group.”

Gene therapy has shown promise in early trials for other disorders caused by monogenetic mutations such as hemophilia, sickle cell disease and Duchenne muscular dystrophy. It will be more difficult to treat maladies caused by multiple genetic mutations, but companies are investing billions in curative therapies.

A 2012 law encourages development of treatments for pediatric orphan diseases by allowing the FDA to award priority-review vouchers that pharmaceutical companies like Spark Therapeutics can later redeem for fast-track review of other drug treatments. The vouchers have boosted research in rare diseases.

Last month Sangamo Therapeutics used a novel technique that directly edits genes—i.e., without the use of a virus—in a California patient who suffered from a rare progressive metabolic disorder. The company is conducting trials using this precision genetic-engineering tool on patients with hemophilia B and Hurler syndrome.

Researchers are also making strides using cell-based gene therapy to treat persistent cancers. The FDA this year approved two CAR T-cell therapies for blood cancers, which in trials produced nearly miraculous results for patients who had failed to respond to other treatments. The therapy re-engineers a patient’s white blood cells by combining the defensive properties of B-cells with the offensive machinery of T-cells. Patients receive infusions of CAR T-cells, which target an antigen unique to cancer cells. As a bonus, CAR T-cell infusions produce less debilitating side effects than traditional treatments.

In a clinical trial of patients with large B-cell lymphomas, Yescarta by Kite Pharma (now owned by Gilead) produced an overall three-month response rate of 82% with 54% of patients experiencing complete remission. Fewer than 10% of non-Hodgkin lymphoma patients respond completely to traditional therapies. Likewise, 83% of pediatric patients with acute lymphoblastic leukemia responded within three months to Novartis’s Kymriah CAR T-cell therapy.

Although CAR T-cell treatments can cost several hundred thousand dollars, traditional treatments are often more expensive and aren’t cures. Prices will also likely drop as scientists develop tools to mechanize cell engineering, and the FDA approvals will encourage more CAR-T cell experimentation.

The FDA has a history of painfully slow drug approvals and setting arbitrary benchmarks to prove efficacy, which has slowed innovation and stalled investment. This is why it’s especially promising that the FDA in November approved for the first time a digital pill that can be used to monitor whether and when patients take their meds.

Otsuka Pharmaceutical embedded a sensor made of copper, magnesium and silicon into its antipsychotic drug Abilify. When ingested, the pill sends an electrical signal to a wearable patch that can transmit to a smartphone app. The digital Abilify hasn’t been shown to improve treatment compliance, but it was deemed safe and approved by the FDA. Its sensor technology could have broader pharmaceutical applications—such as reminding the elderly to take medications or helping doctors determine whether patients are abusing opioids.

Advances in biotechnology are likely to accelerate and compound as they did with computing—especially as corporate tax reform allows American companies to invest more in innovations that improve and save lives.

Appeared in the December 30, 2017, print edition.