Implications of 35 Years of Human Genome Mapping
When the project to map the human genome got underway 35 years ago, scientists knew the results would have profound implications for medicine. It was universally assumed that although the human genome was surely far more complex than that of any other species, all human genomes would prove to be pretty similar. Once that was confirmed by the mapping project, researchers could start looking for the common genetic root of this or that debilitating disease and then develop the drug to cure it.
But the universal assumption was wrong. The human genome was found to be a lot less complex than expected — with fewer genes than some plants, in fact — and while all human genomes are indeed similar in big-picture ways, it is the tiny differences between one genome and another that turn out to be absolutely crucial. If we think of genes as packages of information — which they more or less are — it’s a little like the difference a single letter in a single word can make: Change the first letter in “read” and you get “lead” or “bead” or “dead,” each a totally different meaning. So while our genomes are not all that complex, the variation in the way genes are constructed — with millions of variants possible in a single gene — effectively meant that all bets were off for finding a single universal cure-all for debilitating diseases.
Genome Mapping Ignites Personalized Medicine
Instead, the findings of the genome mapping project ignited the revolution in what we today call personalized medicine, and researchers set their sights on immunotherapies — that is, therapies that could “adjust” an individual’s specific genetics and thereby weaponize the individual’s immune system against the disease afflicting him or her. The fruits of that revolution are just beginning to show up now, in the form of new therapies, procedures, and drugs coming onto the market. Investors in the U.S. capital markets and all of us here at Nasdaq, as the home to many innovative biotech and pharmaceutical companies, have a front-row seat on this revolution. It’s from that perch that we’ve seen the growing excitement about a developing immunotherapy that is my choice for 2016’s Big Idea. It is called CAR-T — chimeric antigen receptor T-cells — and it genetically modifies a patient’s T-cells, the front-line soldiers of the immune system, with specific receptors that can distinguish and destroy cancerous cells. To make it happen, the patient’s T-cells are removed, modified — in effect, that one letter in one gene is changed, and then injected back into the patient and deployed to find, target, and kill the cancer.
The Big Idea that Can Work
What is really big about this Big Idea is that it can work in cancer patients for whom all other therapies have failed. Early clinical trials among a range of desperately ill cancer patients have been rich with promise. And did you hear about Baby Layla in England? Diagnosed at three months with aggressive leukemia that responded to neither chemotherapy nor a bone marrow transplant, she was declared incurable and palliative care was prescribed before she was a year old. But her family decided to make Layla the first test worldwide of an experimental CAR-T therapy, and within months, all traces of the disease were gone. No wonder an article in Forbes last June quoted one oncologist’s assessment of the therapy as “spectacular” after it returned his very sick, wheelchair-bound patients to “normal lives.”
IPO Activity around CAR-T Development
It was the recent flurry of IPO activity for start-ups dedicated to CAR-T development — and the interest shown in them by Big Pharma — that alerted us at Nasdaq to its potential. Juno Therapeutics, now expanding CAR-T to solid tumors; Kite Pharma, currently conducting a trial for patients with non-Hodgkins lymphoma; Bellicum, developer of a molecular switch technology for modulating T-cells at reduced toxicity; Ziopharm, working on a new technology for introducing the CAR into the T-cells; Cellectis, whose UCART 19 therapy was what saved Baby Layla: There seemed a sudden eruption of CAR-T development on our radar screen, and in a field, biotech, in which IPOs proliferate, the enthusiasm surrounding these IPOs has been feverish. Right now, as multicenter CAR-T clinical trials move into their final and decisive phase, we’re seeing ongoing consolidation in the sector with no let-up in the excitement.
Investor interest is typically a bellwether of innovations that can benefit consumers or particular industries. Here on the wide-open frontier of a new kind of medicine is an innovation that can truly benefit mankind — and just might end a scourge that has probably left no family anywhere untouched. That’s a Big Idea at its best.
Adena T. Friedman currently serves as President and COO of Nasdaq. In this role, Ms. Friedman is responsible for overseeing the strategy and operations as well as having financial responsibility for the company’s Listing Services, Information Services (comprising Indexes and Data Products) and Technology Solutions (comprising Market Technology and Corporate Solutions) business segments.
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