NKT Therapeutics Aims For Severe Asthma, Targeting Natural Killer T Cells

Xconomy Boston — 

Even though many venture firms are scrimping, there’s hope for biotech startups in search of capital to fund bold new areas of biological research. NKT Therapeutics, which revealed its $8 million first-round financing in March, has rallied venture capitalists to back its efforts to develop drugs that target lesser-known immune cells that potentially play key roles in asthma, cancer, and a bevy of other major illnesses. Robert Mashal, CEO of the Newton, MA-based startup, filled me on how the firm is channeling its funds into the novel science.

NKT is developing biological treatments to interact with natural killer T cells (or NKT cells), a class of white blood cells, which have a growing following in both scientific and industry circles for their potential to lead to the discovery of new drugs. Though there are drugs in testing that hope to stimulate NKT cells as part of a multi-pronged immune response, no drug that exclusively targets NKT immune cells is in clinical trials or has a track record with the FDA, Mashal says. Thus, NKT Therapeutics has embarked on a biotech journey with both great challenges and great possibilities.

Despite the hurdles ahead, the startup has a deep understanding of NKT cell biology that comes from its founding researchers—Steve Balk, of Beth Israel Deaconess Medical Center, Mark Exley, of Harvard Medical School, and Brian Wilson, at Massachusetts General Hospital—who are experts in the field (they’ve also been research collaborators for years prior to founding the startup in early 2008.) Specifically, the startup has an antibody that is designed to home in on a cellular receptor—a type of protein marker on the surface of cells—it believes is unique to many NKT cells.

The firm plans to initially develop the antibody to block the function of NKT cells in patients with asthma. Research has found that removing NKT cells from genetically engineered mice quells their asthma, Mashal says, and mice or monkeys do have asthmatic responses when NKT cells are activated in their lungs. Yet the firm is about a year and a half to two years from testing the antibody in human clinical trials, he adds.

“If this therapeutic does in humans what it does in animals, then we’ve got a good drug,” Mashal says. “I think that’s the biggest ‘if’ in the industry, and until you do the trials to test that, you never really know.”

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One response to “NKT Therapeutics Aims For Severe Asthma, Targeting Natural Killer T Cells”

  1. Leif Kring says:

    Researchers at Karolinska Intitute in Stockholm have linked NKT cells to auto immune diseases, i.e. a lack of them make the B-cells attack body tissue. This clearly indicates that removing the NKT cells will increase the risk of getting an auto immune disease! See ”Invariant NKT cells limit activation of autoreactive CD1d-positive B cells”, Fredrik Wermeling, Sara M. Lind, Emilie Domange Jordö, Susanna L. Cardell & Mikael C.I. Karlsson, Journal of Experimental Medicine, online 3 May 2010, doi: 10.1084/jem.20091314, paper issue 10 May 2010, Vol. 207, No. 5.