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Early success with CAR-T cell therapy offers encouragement for NJ biopharmas

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Dr. Alfred Gillio, director of the Children’s Cancer Institute at Hackensack Meridian Health’s Joseph M. Sanzari Children’s Hospital, with 9‑year‑old Jizaiah Ramos, the hospital’s first pediatric cancer patient to be successfully treated using CAR‑T cell therapy.
Dr. Alfred Gillio, director of the Children’s Cancer Institute at Hackensack Meridian Health’s Joseph M. Sanzari Children’s Hospital, with 9‑year‑old Jizaiah Ramos, the hospital’s first pediatric cancer patient to be successfully treated using CAR‑T cell therapy. - ()

In the eight months since Hackensack Meridian Health and Novartis Pharmaceuticals partnered to offer CAR-T cell therapy, HMH’s Joseph M. Sanzari Children’s Hospital in Hackensack reports it has successfully treated two pediatric cancer patients.

CAR-T cell therapy is a form of immunotherapy to treat B-cell lymphoma – a rare type of the disease that often proves to be a death sentence for those afflicted. The treatment previously involved a bone marrow transplant that frequently led to a relapse and weakened immune system. Essentially, it’s the body’s immune system that must identify and attack proteins in cancerous cells.

Soon after the partnership was formed, HMH became the first health system in New Jersey to offer the Novartis therapy, which is marketed as Kymriah.

The first patient at JMS Children’s Hospital to receive it was 9-year-old Jizaiah Ramos of Elizabeth, who has battled acute lymphoblastic leukemia, a rare type of bone cancer, since he was 4.

He previously was treated at another hospital with chemotherapy and radiation. That was followed by a bone marrow transplant at Hackensack University Medical Center. But a year after the transplant, his leukemia had returned. 

After undergoing the hospital’s immunotherapy program, his cancer is now in remission.

So too is 3-year-old Olivia Viscogliosi’s. Two months ago, she also was diagnosed with ALL. She underwent a bone marrow transplant, but two weeks later, her cancer returned. Her parents took her to JMS Children’s Hospital in a last-ditch effort to save her life, which the CAR-T therapy did.

These stories, of course, represent a triumph for the two children and their families. They also serve as encouragement for New Jersey-based life-sciences companies such as Novartis, Celgene Corp. and Johnson & Johnson, which have poured billions of dollars of research money into immunotherapy such as CAR-T in hopes the treatment represents the future of cancer care.

“These are children that we would have been sent home to hospice in the past. These are end-stage patients who really had no other options until the CAR-T therapy came along. It is a giant breakthrough for us, and to be able to treat these patients is so amazing.”

— Dr. Alfred Gillio, director of the Children’s Cancer Institute at Joseph M. Sanzari Children’s Hospital

Dr. Alfred Gillio, director of the Children’s Cancer Institute at JMS Children’s Hospital, said the two cases bring CAR-T cell therapy a step closer to becoming mainstream treatments.

“These are children that we would have been sent home to hospice in the past,” Gillio said. “These are end-stage patients who really had no other options until the CAR-T therapy came along. It is a giant breakthrough for us, and to be able to treat these patients is so amazing.”

For Celgene, these success stories could go a long way in helping to quell trepidation from investors who have questioned whether the company has overspent on immunotherapy.

In March, Celgene closed on a $9 billion acquisition of Juno Pharmaceuticals, a life-sciences company based in Washington state that specializes in immunotherapies. The company also committed to constructing a new, 135,000-square-foot CAR-T cell therapy research center on its Summit West Campus.

Geoffrey Porges, a biotech analyst at New York-based Sanford C. Bernstein & Co., pointed out that immunotherapies such as CAR-T are still years away from turning a profit.

“Celgene’s management are to be congratulated on the audacity of their deal-making, but we expect investors to bridle at the company’s increasingly aggressive front-end loading of their transactions,” Porges said shortly after the Juno acquisition. “This transaction amounts to prepaying much of the cost of a distance asset well in advance of the delivery of the asset.”

While Celgene CEO Mark Alles has too acknowledged profitability is not immediately on the horizon, he is confident the future of cancer treatment has arrived.

“The first thing that investors and the medical community ... [are] realizing is that this class of medicine of CAR-T therapy — whether its lymphoma, leukemia or myeloma — will replace existing chemotherapy,” he said in a prepared statement.

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Vince Calio

Vince Calio


Vince Calio covers health care and manufacturing for NJBIZ. You can contact him at vcalio@njbiz.com.

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