FDA’s Historic Gene Therapy Approvals Could Be Beginning of the End of Blood Cancer

FDA’s Historic Gene Therapy Approvals Could Be Beginning of the End of Blood Cancer

In his inaugural address this June as chairman of the world’s largest biotechnology industry group, CEO John Maraganore of Alnylam Pharmaceuticals dropped more than a few jaws with his prediction that we can “expect that all hematological malignancies will be cured in the next decade.”

It was a “wow” moment: envisioning an end to blood cancer that soon.

On Aug. 30, biotechnology began to transform our industry’s vast potential into reality when the U.S. Food and Drug Administration (FDA) approved Novartis’ “living drug” called CAR-T therapy that genetically modifies a patient’s own blood cells to attack an untreatable form of leukemia in children and young adults. The FDA called that approval “historic.”

Following the announcement, I echoed the optimism of many in the biosciences when I speculated that “the first gene therapy ever approved for commercial use in our country won’t be the last. Today, CAR-T therapy is seen as a route to healing for young people tragically affected by a rare leukemia unresponsive to available treatments. Tomorrow, who knows? Already, there are investigations underway to determine if this method can be used to treat other kinds of cancer in different patient populations.”

Well, “tomorrow” came exactly seven weeks later.

Yesterday, the FDA made the thrilling announcement that it has now sanctioned the second-ever gene therapy for sale in the United States. Gilead Sciences (through its recent acquisition of Kite Pharma) soon will offer a new CAR-T therapy with the potential to send cancer into remission for thousands of patients with untreatable lymphoma.

The new immunotherapy was approved for adults who’ve undergone two unsuccessful rounds of chemotherapy or bone marrow transplants for the most common type of aggressive non-Hodgkin lymphoma — diffuse large B-cell lymphoma — and other forms of lymphomas, including primary mediastinal B-cell lymphoma, high-grade B-cell lymphoma and transformed follicular lymphoma.

In the pre-approval study, 52 percent of patients who received the treatment went into full remission and 21 percent went into partial remission.

One of the lives saved in the clinical trial belonged to Dimas Padilla, a 43-year old sales rep from Kissimmee, Florida. When he returned to the hospital after undergoing unsuccessful chemotherapy and bone marrow transplantation, he says his lymph nodes “were so bad that they moved my vocal cords to the side and I was without my voice for almost three months,” he said.  “Once they infused … the cells in my body, within two to three days all my lymph nodes started melting like ice cubes.”

Today, Padilla is in full remission.

FDA Commissioner Scott Gottlieb said yesterday’s approval was “another milestone in the development of a whole new scientific paradigm for the treatment of serious diseases.” He noted, “In just several decades, gene therapy has gone from being a promising concept to a practical solution to deadly and largely untreatable forms of cancer.”

We in the biotech community have talked for years about the promise of personalized medicine and gene modification. Gilead’s CAR-T approval for lymphoma and Novartis’ breakthrough for a rare, aggressive form of leukemia have delivered on that promise in a big way in 2017.  For the FDA, it’s a testament to the agency’s commitment to keep up with breaking science and provide the regulatory flexibility to get miracle cures to patients as quickly as possible

The biotechnology industry has had many disappointing and costly failures over the years trying to bring gene therapies to market, but researchers never gave up. Personalized cancer treatment is here at last, and the end of blood cancer as we know it is no longer a fanciful dream.

Greenwood, who represented Pennsylvania’s 8th district in the U.S. House of Representatives from 1993 to 2005, is the CEO of BIO.

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