From Controversy to Cure

Let's give it up for this amazing crowd tonight. What's up, Boston? Woo-hoo!

[CHEERING]

(SINGING) Do what I want 'cause I can, if I don't, because I wanna.

[MUSIC PLAYING]

We have four bands competing tonight. They each get a 15-minute set, and then at the end of the night, the judging panel comes together and decides on a winner.

Good job. Sounded great.

That winner gets 50% of the charity proceeds for their charity of choice.

(SINGING) We will, we will rock you.

[MUSIC PLAYING]

The idea behind the Battle of the Biotech Bands has to do with doing what we can for children and their families who are fighting diseases, here and around the world.

(SINGING) Don't you forget about me.

We know there are patients out there who are in dire need of serious treatment for serious diseases. And so any little bit that we can do, whether it's through our work or through music, passions that we have, we just want to come out and rock it.

(SINGING) Aaaaah, ah, ah-ah-ah-ah, yeah.

The creativity here tonight is an example of the creativity that goes on in the workplace and in those laboratories.

If you guys do your science as well as you do your music, we've got nothing to worry about here.

[CHEERING AND APPLAUSE]

In the Boston area, biotechnology literally takes center stage. And you don't have to look far to see why. That's because the region is home to the biotechnology capital of the world, Kendall Square in Cambridge, Massachusetts. Here, in less than one square mile, clustered around the Massachusetts Institute of Technology, are more than 100 biotechnology companies, nearly all of the world's top pharmaceutical firms, and millions of square feet of lab space. Groundbreaking drugs developed here have already touched the lives of 2 billion people worldwide, and more treatments are in the pipeline.

Kendall Square is simply the densest collection of life scientists in the known universe.

We're making big inroads in cancer. We'll make big inroads in Alzheimer's. We'll make big inroads in ALS, and many other diseases.

But just a few decades ago, this industry and the science underlying it didn't exist. Kendall Square was a desolate landscape of shuttered manufacturing plants and vacant lots.

When I arrived here in 1960, Kendall Square looked a bit like a war zone.

It was an area that was deserted. And beyond getting on and off at the train station, people typically didn't go there.

And now, when you walk down the street, there are young, excited people who are racing to get someplace, busy at some piece of inventing the future.

It's dynamic. It's energetic. It's vibrant. It's innovative. It's dense. You bump into people a lot, which I think is the beauty of the place.

So what brought about this dramatic change? How did this industry that affects so many lives and drives the economy of an entire region arise in such a short period of time? And why here, in such an unlikely-seeming area?

I hesitate to say that there was a logic to it.

It was not a deliberate path that led us to where we are.

There was a lot of courage. People really were willing to follow the science, take risks, to dare to do things that otherwise are completely unthinkable.

[MUSIC PLAYING]

They drove me to Rockefeller on this day in July 1962. Eventually, the person living in the next room introduced himself. He was a hippie. He had long hair, white clothes, sandals, unshaven. My mother peeked into the room, and it was a bit of a mess. And her parting words to me were, Harvey, he's not our kind, I don't want you to have anything to do with him. However, 13 years later, when David Baltimore won the Nobel Prize, it was all over Cleveland, my son's best friend just won the Nobel Prize.

When I came here in 1960, we were just starting molecular biology we had no reputation as a school of biology. No one even knew that MIT did biology. Today, biology permeates every part of MIT.

[MUSIC PLAYING]

Biology meant everything. It meant the kingdoms and the phyla and it meant physiology and strange creatures.

[INSECTS CHIRPING]

Until that time, biology was a collection of unrelated facts and observations, rather than a discipline governed by a small set of unifying principles.

Watson and Crick discovered the structure of DNA in 1953 with the data from Rosalind Franklin. MIT's leadership recognized that this was the unifying concept for biology.

MIT said, we just can't do everything. We're going to focus just on molecular biology.

Molecular biology was a view of biology as an information science. And the information was encoded in DNA.

It's just about then that Salvador Luria, a refugee from Italy, came to MIT.

Salvador Luria, who the next year won the Nobel Prize, was really the heart and soul of the MIT biology department.

Luria had a vision that all of biology was going to be transformed by a molecular approach to questions about living systems.

And what happened in the early 1960s was this brilliant flash of insight on the part of many that one could really understand everything if one really understood the details of how cells were organized, how they turned on and off different genes and proteins.

The year was 1963. The genetic code was still being cracked. Jim Watson would come to class talking about DNA and this revolution which is called molecular biology. And I'm listening, and at the end of the hour, I'm a different person. My life has been redirected. And it was clear that he was literally telling us the secret of life, and how everything biological gets made and why it works the way it does.

With this scheme of DNA makes RNA makes proteins, one could explain the entire biosphere with a very simple conceptual structure.

What molecular biology had given us was the ability to study the molecules that determine the properties of life. I thought one day, people will be able to work on cancer at the molecular level. They'll be able to understand how cancer comes about by studying genes.

More people each year die of cancer in the United States than all the Americans who lost their lives in World War II.

Richard Nixon was elected president in 1968. And he's very concerned about being re-elected in 1972. And so Nixon says he will declare a war on cancer, and push for legislation in 1971 as a way of preempting this as an issue in the presidential election.

This funding by Nixon was critically important because Luria correctly perceived that the war on cancer would offer him some largesse in building up the MIT Cancer Center and in recruiting faculty.

We built the Cancer Center in an old candy factory, which MIT owned. It was a very audacious idea that molecular biology was going to have an impact on cancer.

I was recruited to a faculty position. And I thought, wow, I hadn't thought about that, because that was something that women just didn't do, really, in my generation. When I wanted to go into work on cancer, my friends said, goodbye, we'll never see you again, because you could bring molecular biology to this problem, but it didn't mean it was going to work, that you were going to get anywhere.

The assumption at the time was, if we understood the causes of cancer, we would surely be able to treat the disease.

We had a very intense research group on the fifth floor of the Center for Cancer Research at MIT.

Oncogenes were discovered, and the functions of cancer genes were discovered, and the fact that some of these genes were active in human tumors was discovered, and how viruses cause cancer was discovered. And it was one discovery after another.

The legend on the campus was the lights never went out on the fifth floor of the cancer center. There was always somebody in the laboratory working, 24 hours a day.

The MIT Cancer Center ultimately had four Nobel laureates-- Baltimore, Luria, Susumu Tonegawa, and then Phil Sharp. It was an extraordinary powerhouse, and represented the wave of the future.

Luria brings his team in. They renovate an old candy factory. They're about to get started. And then there's a crisis.

[MUSIC PLAYING]

Scientists, including many at MIT, developed technologies for isolating individual genes, learning how to take these genes, put them in cells, and then make a lot of the protein that was coded by the gene.

For the first time, man had the ability to make a biological organism that may not have ever appeared before in the biological kingdom. And that was recombinant DNA.

It was an exciting advance because it really opened up new ways to be able to study human genetics.

This was new technology at a world historic level, like making steel or petrochemical industry. It wasn't just a little technical advance, it was going to be a whole new industry.

This represented the first time one could splice together genes from, for example, humans with the genes of bacteria, a very unnatural juxtaposition.

That led to a lot of debate about how this should be done. Should it even be done? What could be the consequences? What powers would come with this new technology?

And this conjured up all kinds of horrible scenarios that might spawn different kinds of plague.

[MUSIC PLAYING]

Doctor, you were the one who got this particular kind of research started, and then you spoke out about the possible dangers of it. What happened that caused you to do that?

Well, we could foresee that there was going to be great interest in this technique, and that many people were excited about the opportunities that was created by it. We could also see that some of the experiments that might be done might perhaps create some dangerous situation.

I started getting telephone calls calling me a monster for actually creating molecules that could possibly be very deleterious to humans.

Any new capability carries with it a potential danger, but we weren't actually sure what the range of danger was. In fact, some people thought there really wasn't much danger, and that natural processes would counter the synthetic processes that we had developed.

So you have all these other agendas that begin to pile on top of this-- people who are frightened that this might have risks to humans, and other people who are saying this is an industrial realm that we must not surrender.

Science had been moving incredibly fast, beyond the ability of the general public to assess it, to understand it, become comfortable with it. Many of us felt that we needed to take some kind of action. And so the first thing was a moratorium on those experiments.

So we called for our scientists throughout the world to pause, to temporarily defer certain experiments until we could meet and discuss the nature of the possible hazards and the kinds of experiments that could or should not be done.

The moratorium was remarkably respected all around the world.

Never in history had a group of scientists come together and said, we will not do x.

We called for a conference. And that was the quite famous Asilomar meeting that was held in a conference center in California.

That was going to be an international conclave to consider the risks and to try to set forth some principles for regulating research.

There were approximately 150 scientists from around the world.

And we also invited some lawyers, some ethicists. We invited members of the press.

They were every bit as much involved in the conference as were the scientists.

Every evening, we had big beer parties. And so there was people mixing and talking.

We wanted this to be open. We didn't want anybody to think we were some secret cabal.

It was an extraordinarily passionate and even contentious meeting.

Everybody was sort of pointing fingers. It's not my work that's dangerous. How could that be dangerous? It's your work that's dangerous. Don't tell me what I can do, can't do. So there was a lot of that back-and-forth.

The ethicists and the lawyers made it very clear that there were very severe personal risks. Everybody who came of the so-called experts thought the risks were very small, but they're not zero. If it's not zero and there is some potential risk, and you can't really quantify it or identify it, the best thing to do is go slow.

The scientists stop talking about if they should do the research. They start talking about how to do the research.

In essence, the proposal was called on the NIH to develop a series of guidelines that would instruct people on how to do these experiments safely.

The final statement of that conference did serve as a basis for the National Institutes of Health to promulgate some regulations of the field, which they did in late June of 1976.

By and large, recombinant DNA became doable. If you followed the guidelines, there was no problem.

What they hadn't necessarily counted on is that individual communities might become concerned. And that's where Cambridge City Council says we don't trust the federal government to make decisions about work that's being done within the bounds of the city of Cambridge, which really sets up one of the most dramatic cases of scientists and the concerned public confronting each other over how to regulate this technology anywhere in the nation.

My research involved recombinant DNA technology. I contributed to that technology while I was at MIT. I had some very interesting experiments I wanted to do. And then there was a continuation of the moratorium here in Cambridge.

But let me assure you that every city in the United States of America and throughout the world is now discussing the DNA experimentation in their cities and towns and villages and hamlets.

The hall was full of citizens of Cambridge and scientists from MIT and Harvard.

I'm standing in the balcony, because the hall was totally full, watching Mayor Vellucci here hold forth.

Most of us in this room, including myself, are lay people. We don't understand your alphabet. So you will spell it out for us so that we know exactly what you're talking about.

The debate itself came out of academia originally, really as questions raised by some biologists about the work of other biologists.

My name is Jonathan King. I'm associate professor of biology at MIT. I have been concerned with this issue for a number of years.

They essentially went to city council and said, we don't know the answers to these uncertainties, and we think that it's a community issue.

It was said that there was no danger of escape. I'd like to go down on record as saying that's a patently false statement. In fact, it is absolutely certain that they will escape.

We wanted regulation, as existed for many other hazardous materials in American society.

The risks in this case are purely hypothetical. Not only has no known dangerous organism ever been produced, but I believe it to be the opinion of the overwhelming majority of microbiologists that there is, in fact, no significant risk involved in experiments authorized to be done by the federal guidelines.

There was a reasonably wide held belief in Cambridge that federal agencies may not always have the best interests of the community in mind.

You've got to see it in the shadow of the atomic bomb, because that was the singular event in which the public suddenly became aware that science had done things that they never conceived of. And they kept worrying that there was an atomic bomb hidden away in modern biology.

The mayor of Cambridge became quite alarmed by the rhetoric - visions of viruses and germs or cloned creatures taking over the streets or infecting us or worse.

I have made references to Frankenstein over the past week, and some people think this is all a big joke. That was my way of describing what happens when genes are put together in a new way. This is a deadly serious matter. If worse comes to worse, we could have a major disaster on our hands.

So it was very dramatic. You know, in that environment, you know, you say things that, you know, maybe you wish you hadn't said.

I don't think those guidelines were written by a group of people who represented the public and all the interests. Those guidelines are like having the tobacco industry write guidelines for tobacco safety.

I'm kind of proud of it, even though many of my colleagues wouldn't talk to me.

This country, this community has not decided that we should go ahead with this research. It is very dangerous. It's very questionable.

Be it resolved that the Cambridge City Council insists that no experimentation involving recombinant DNA should be done within the City of Cambridge for at least two years.

[APPLAUSE]

[BOOS]

Here at the moment, we could unleash a whole new powerful tool to answer our questions. No.

[MUSIC PLAYING]

Had there been something that happened, an organism created that disseminated itself through Cambridge and maybe the world, we would have been blamed for that, and it would have brought a halt to the direction of modern biology, modern science. So there was a tremendous amount at stake.

Powerful forces were at play. The venture capitalists were already there in the background watching and waiting.

The mayor appointed a commission of ordinary citizens to answer one simple question-- are these scientists being honest with us? Is the process that was put in place at Asilomar a process that is going to safeguard the community?

That was a novel reality for the academic community. They were not accustomed to citizens perceiving themselves as equal partners in the development of science.

In the end, they decided that they thought the processes that were being developed at the National Institutes of Health were appropriate and that this technology was the future of biology and should be freed.

It took about a year to go from the first city hall meeting to the position in which we could really begin to plan to do those experiments in Cambridge.

The ban on using recombinant DNA was lifted, and that resulted in an explosion of biomedical research, because it was such a useful technological innovation.

The then-mayor of Cambridge, in a reaction to this activism, led the creation of the nation's very first zoning rules for a biotech-related research development, leading this as a way of controlling the growth of biotech in Cambridge. In fact, it had the opposite result, which was creating predictability for the industry. Where there was no predictability in zoning in development across the United States of America, there was in Cambridge. There was in Kendall Square.

And that's one of the reasons why Cambridge got out ahead of so many other areas, because we had a blueprint that set out what could be done in the city.

It liberated Harvard and MIT to move forward and to become centers of the new biology.

When Biogen came to Cambridge and applied for the first recombinant DNA license in a commercial organization in Cambridge, Cambridge said, we've gone through this debate. People in Cambridge know what the issues are. We have these guidelines. If you adhere to these guidelines, like the universities, we're fine.

And Mayor Vellucci showed up for the opening of the Biogen's first laboratory in Cambridge and cut the ribbon and said, you know, I like recombinant DNA experiments, particularly when they pay taxes. And that led to human insulin. That led to human growth hormone. That led to interferons for infectious disease and multiple sclerosis, and it's led to treatments of cancer. And it's just an extraordinary, extraordinary breakthrough.

So what we had, then, were a few small companies that really provided the nucleus for a biotech industry in Kendall Square.

The early days, people were guessing as to whether it would take 10 years or 2 years to be able to make a particular protein in pure form, and the FDA didn't even have guidelines as to how they would regulate new products. And those guidelines kept changing, and so it was kind of a real wild west moment. It was really the birth of an industry.

[MUSIC PLAYING]

From these roots in recombinant DNA, a lot of interesting things happened.

One is the cures. For a long time, the industry was very hesitant about using the "cure" word, but we've gotten to the point where if we're not eradicating, we're substantially and dramatically improving the lives of individuals with rare disease. So that's a real visceral impact.

I began working on the intellectual property, the patents mostly, for the fledgling Biogen, who, at that time, had no employees. They were basically seven or eight scientists in their labs around Europe and the US.

Biogen started by two venture capitalists.

They had a fantastic idea. Get the best scientists from all over the world and start a company.

A biotechnology firm that starts could probably expect to take 10 years before its product came to market. That's 10 years before any profit, and it's 10 years of needing an immense amount of money to sustain this operation.

And the probability of success, historically, has been about 5%, so when you have these incredibly risky investments, you're going to have a challenge in being able to raise the proper kind of financing to fund them.

Biotech was the first example where Wall Street was willing to fund new technology over a long period of not being profitable to own part of the company when it became profitable.

What really put Biogen on the map was the cloning of interferon. It really was this holy grail of biotechnology.

Interferon is a natural compound produced by the body when it is infected by a virus.

It seemed to kill viruses, kill cancers. It was amazing. But you couldn't get enough of it.

And because it was so unavailable, there grew up a bunch of myths about how potent it was going to be, and it's going to be a cure for cancer.

And Biogen set that as one of their projects to try to clone it and produce it.

We came to an agreement with Charles Weissman, one of the founders of molecular biology, that he would develop a research program to clone the gene for alpha interferon.

We didn't have a lab, and those of us who undertook projects undertook to do them in their university labs.

On Christmas Day, 1979, Charles is up in Davos, where he skis over the holidays with friends, and back in his lab is Nagata, who's a postdoc.

Nagata was extremely hard-working, and within a few months, he was then successful in actually cloning the cDNA of interferon alpha.

Nagata calls Charles and says he thinks he's seen a positive out of one or two of the bacteria colonies he was assaying.

When Charles cloned and expressed interferon, it was a world-shaking event, and we thought it will cure everything.

I flew to Switzerland immediately and spent the next two weeks writing the patent application, because we knew we're in a race. We knew other companies were trying to clone interferon as well.

We, as a scientific advisory board, had talked the company into having our January meeting in the Caribbean. And the first day of the meeting, Charles gets up and explains what he's done and the evidence that this is alpha interferon that he has the gene for. And we were just astounded.

The board, at the time, wanted to publicize this, because it would boost the share price of Biogen. And I didn't want to have this publicized before I had a scientific paper describing these results.

He agreed to hold the press conference, if he had a chance to present the data to scientists, who could judge it and critique it. And I said, we can get a group of scientists at MIT for a seminar on Monday. And so that became the plan. We flew off. Charles has got a typewriter, which he bought in the hotel. And he's pounding out this manuscript while he's flying, because he wants to be able to give people a description of what the data is.

And Charles came to MIT, gave a seminar, and then gave a press conference.

Charles gets up and starts talking about cloning the gene, and it's all DNA and joining and putting in bacteria and assaying interferon.

I thought it was going to be a big event-- you know, cloning interferon is something people had been trying to do for so long. But what ended up was there were a lot of critical questions, you know, what is an academic scientist doing working for a company?

And the reporters aren't getting it, so I get up, and I take the microphone from Charles, and I tell the reporters why cloning alpha interferon is a great thing.

Then the next day on the front page of the New York Times, it was, you know, Biogen cures cancer. And so that really set Biogen on the map and got people really thinking about the possibilities of recombinant DNA.

Scientists in Boston today claimed a major medical breakthrough. They said they have used bacteria to produce a substance called interferon. Interferon fights many viruses, including those believed to cause many forms of cancer.

It was, at the time, quite unusual that a biologist would be linked to some commercial interest.

There was the ethic that applying one's work to common everyday uses in clinical applications was dirty, would somehow sully the academic basic biomedical research agenda.

Some of my colleagues thought it was an awful thing to have done, you know. They actually called me names for having succumbed to this financial influence.

But MIT was different. It derived from a decades-long ethos at MIT, where one had encouraged electrical engineers and computer engineers to apply what they had learned to everyday practicalities that yielded many kinds of benefit for humanity at large.

The concept of, should a university be working with industry, will the grubby fingerprints of industry soil the ivory tower of academia-- MIT didn't have to go through that, because they weren't set up as an ivory tower. MIT started off with the charter of bringing science to industry and agriculture. So the faculty were working with industry probably since MIT opened its doors in 1865. They were starting companies all along.

Interferon doesn't really have much effect on lots of cancers, but it does have an effect, and it became a more than a billion dollar a year drug. It was sold for a particular type of leukemia, but more importantly, for hepatitis C. And for years, it was the only, really, treatment for hepatitis C.

After this discovery, scientists all around the world who were working in the field began to think about the commercial aspects of it rather than just the academic aspects of it.

In a sense, the entire industry starts from one or two companies-- Genentech on the west coast, Biogen here. You know, there are now 2,000, 3,000 companies. It's infection. The growth of the industry, in large part, is by infection. People see it's possible.

It was not planned. Most of the biotech companies, virtually all, were started by faculty entrepreneurs. The company that I helped start, Genzyme, focused on making recombinant enzymes that could be used to treat certain genetic disorders, such as Gaucher disease. I have seven grandchildren, and one of them has Gaucher and has been treated with the drug his grandfather helped develop.

My name is Andrew Steinert. I'm 16 years old, and I attend Belmont High School.

Gaucher disease is a disease in which the body cannot degrade a particular glycolipid, a particular fatty substance. It affects the liver and the spleen and the bones and things like that. He used to have to go to the hospital every three weeks to get an infusion. He now gets it at home, and he simply sits and does his homework while they put the line in and transfuse in the medicine.

Every ethnic group has its own genetic diseases, because one person develops a genetic disease, and then unknowingly, various descendants of that person have a child together, and you get two bad copies of the gene, and you have a disease. That's what happened with Gaucher.

We were very fortunate to have the Genzyme drug in place, clearly. And it also started me working on trying to develop solutions for other rare diseases.

Biotechnology can enable him to leave a really symptom-free, carefree life despite having Gaucher's.

He's never been sick. He's very healthy, very energetic.

This summer, I plan to bike across the country from Savannah, Georgia to Los Angeles, California over six weeks. It's about 85 miles a day. It sounds crazy, but I look forward to doing it.

[CHEERING]

The exciting thing is once you make an advance of that nature in medicine, it's an advance forever.

It's that culture, the culture of faculty entrepreneurs, that's the key. And that's what really drove Kendall Square.

Kendall Square actually has a history that dates back into the post-industrial revolution. It was once a geography that was deeply involved in manufacturing.

In the 1950s and 1960s, New England as a whole experiences an industrial slump. Many of the businesses that made East Cambridge such a vibrant neighborhood either close or relocate.

What was left behind in Kendall Square was a barren wasteland of former manufacturing facilities.

NASA was in a very expansive mood in this early period. It had just been created in 1958, and building some new research centers. And one of those that they felt was essential was a research center to study electronics. It was proposed that Cambridge be the site.

There were over 100 takings by the government to consolidate parcels, raze them, and prepare them for a competitive bid that Massachusetts was in the middle of, which was for the location of NASA.

People thought for many years that this is what the future of Cambridge would look like-- a hub of aerospace, research, and technology for years to come.

But then that plan never comes to fruition.

President Richard Nixon, concerned by the deficits and the costs associated with the Vietnam War, decided to make significant cuts in the national budget. Among those cuts was the electronics research center.

It left behind the cleared land and a lot of parking lots. By the late 1970s, MIT is not only sitting inside of a city, but it's also sitting next to an area of land that is effectively vacant. And that is the land where a lot of the first biotech companies that want to locate next to MIT move into, and they find office space. They find buildings that have strong floors and high ceilings, so they can sort of do flexible lab configurations in. And we just got lucky here in many respects.

If they hadn't torn down all those factories, if the land hadn't been vacant, would it have developed the same way?

At the time, people thought that was an enormous loss. But in retrospect, it was a silver lining behind a cloud, because it made an awful lot of space available in the subsequent decades for the building of, for example, the Whitehead Institute.

Jack Whitehead had developed a company to make scientific equipment, and so he wanted to build a research institute that would be his gift to the research community.

There were people who were suspicious that the White Institute was somehow a vehicle for enabling Jack Whitehead to fund his own biotech investment ventures.

This is a business guy, an industrialist who made a fortune out of innovation. He's going to look to skim off all the innovation that comes out of this research institute.

The Boston Globe published a series of articles that sort of inflamed the Boston area.

This was a bit of out of control paranoia, and, in fact, Whitehead was accused of all manner of things.

Here he was trying to do something totally philanthropic, and he couldn't convince people that it was totally philanthropic.

Academic Council discussed it, and then voted it down, and they voted it down by a huge margin, by 85%.

As Whitehead observed later, making money was much easier than giving it away.

He was giving us the opportunity to move a little closer to medicine without having a medical school. And so I had to convince my colleagues that that's what was going to happen, and not what the Boston Globe said was going to happen.

And after it had had a full hearing for a year and a half of discussion on the ground, it then went back to Academic Council and was voted in approval by 85%.

And in the end, we got a piece of land right next to the MIT campus, and we built it.

We were the pioneer building there, yes.

By 1990, the Whitehead Institute is perceived and acknowledged as, perhaps, the world's leading research center in molecular biology. It was that fast.

The Whitehead Institute's excellence is due to the vision of David Baltimore. He could sense talent, and when people like Eric Lander came along, he saw Lander to be a very intelligent person.

Here was a guy who had no formal training in genetics, in biology, in medicine.

He was not really a biologist. He was a mathematician.

And Eric decided that he was going to leave behind much of his mathematical career and his business career and become a geneticist just at the time when there were the first discussions of sequencing the human genome.

This heady idea that when we could barely sequence a couple hundred letters of DNA after a long day's work, we might read out the entire human genetic code captured the imagination of a young new generation of which I was part. And it just changed all our lives.

Very quickly, we formed the Whitehead MIT Center for Genome Research. That becomes one of the key enterprises in the larger human genome sequencing project. Somewhere in the neighborhood of 1/3 of the human genome was sequenced here at MIT.

Suddenly, it meant instead of wandering forever trying to look for one gene that's causing a disease, you had an inventory of all 20,000 human genes. You could see their locations. You could map their inheritance. You could begin to collect not just one genome, but by using it as a scaffold, all the variation in the human population. It sort of turned on the lights. We could look around, and we could see everything. That genome center became a magnet for smart, young, PhDs and MDs who all knew that this was an important part of the future of biology, and thus was born the idea of creating the Broad Institute.

And as a result of all this, what ended up within a radius of 150 yards-- four extraordinary institutions-- the core MIT Biology Department, the Koch Institute, the Broad Institute, and the Whitehead Institute.

And, of course, up the street is the McGovern Institute focused essentially on the brain and nervous system, and also the Picower Institute. All of these benefit, I think, from the initial gamble that was taken with the Whitehead.

In the 20th century, we treated diseases based on their symptoms. What's happened in the 21st century is all of medicine is getting rethought in terms of the fundamental causes of disease-- the genes that are mutated, the specific cellular mechanisms that are deranged. And it means that targeted therapies based on a real mechanistic understanding have become the norm. And because of that, hundreds and hundreds of new kinds of drugs are being developed. Thousands and thousands of clinical trials are being run. And we're beginning to see truly remarkable responses.

[MUSIC PLAYING]

I think the most remarkable to me is the idea that we can do gene therapy and cure certain genetic diseases, that we can do genome editing and take on things like sickle cell anemia or blindness.

Within 8, 10, 15 years, you saw lots of these companies springing up to develop therapeutics for a whole host of human diseases that we couldn't even think about treating even just a few years before.

If you go back 30 years, I don't think there was a single large pharmaceutical company in Massachusetts. And now, of course, there's everybody here.

The large companies started setting up offices and research labs in Boston to observe, partner, and, in some cases, buy these small biotech companies to add to their stable of pharmaceuticals.

Novartis was the first pharmaceutical company to invest in the Cambridge ecosystem, long before it was the mega cluster that it is today. The architecture of our research headquarters started to grow, first in the Necco Wafer factory that is now the headquarters of our cancer drug hunting, culminating in 2016, where we opened these two new buildings, completing more than a million square feet of lab space. Our pipeline has 340 drug discovery projects, 90 clinical stage molecules, and more than 500 clinical trials.

Most pharmaceutical companies now have deeply embedded collaborations, but also physical footprints here. And the biotech ecosystem is just absolutely thriving.

Pfizer moves into Cambridge. Sanofi buys Genzyme, and Millennium starts on human genomics. Vertex comes out of Harvard, and now we're seeing Google and Microsoft and Amazon, so Kendall Square has now taken on the patina of being high tech, strong biological, IT, big data center.

And some of them have been unbelievably successful. I mean I would think the market capitalization must be on the order of a trillion dollars, maybe more.

It's a little like a chemical reaction. Two reactants may react a little bit, but if you have a lot of them, all of a sudden, the concentration drives the reaction.

Kendall Square has now become one of the very most expensive places, if you think about real estate costs, per square foot rents. I think it's on par with Times Square in Manhattan. That's because everybody wants to be here.

LabCentral is a really interesting innovation in and of itself. There was a recognition that these tech incubators are fantastic, but what the life sciences really needs is wet lab incubators, a completely different animal.

There's often what's referred to as the valley of death between research in a university and a successful company. And the reason is the cost of building a lab can be millions of dollars. So what we discovered was that if we shared the resources, and you brought all the entrepreneurs together, not only could they get a very efficient access to the physical infrastructure that they needed to kind of get through that valley of death at a lower cost and hopefully not die, but they also found that they were around other people doing the same thing.

The people that traverse the valley of death are the ones that sort of, like, hold hands and get across through advice or connections. That's actually what Kendall Square is really good at is sort of like holding hands across that valley.

The vast majority of biotech companies never are profitable from selling any kind of drugs. That's not their goal. Their goal is to take a piece of intellectual property, a patent or an idea for a new therapy, and develop it to the point where a big pharma company will look at it and say, gee, this has promise. We will buy it up or license the technology, and we will take it through all the way to approval.

[MUSIC PLAYING]

Today is our Amgen Pitch It for a Golden Ticket Event. Once a year, we award two companies a golden ticket which gives them free bench space at LabCentral for one year. And so we had 44 companies applied. We selected six to do the pitch, and we'll actually hand out the two winners of the golden ticket this evening.

We are standing on the quite literal precipice of a true revolution in the way we treat cancer.

Our first product is a biodegradable minimally invasive implantable patch that we can embed with the powerful toxic agents.

These onco-selective messenger RNAs are delivered into cancer cells and healthy cells with lipid nanoparticles, just like a cancer vaccine.

Just giving these guys the first step on the ladder is very critical. Sometimes that can be the hardest part.

It's a non-viral transfection technology in which cells are flowing through a microchannel.

[APPLAUSE]

And now for the fun part, it's time for us to vote. You can choose which company that pitched tonight that you think should win one of Amgen's next golden tickets.

We're looking for an exciting innovation. We're here in Kendall Square, which is the innovation capital of the world. But we're also very interested in looking at technologies, approaches that would align with Amgen's overall broader strategy. And so that allows us to partner up our scientists with our Golden Ticket winners to help mentor them, to help them navigate some of the key questions that they have in the early phase.

So without further ado, I'm going to introduce the first Golden Ticket is being awarded to PanTher Therapeutics.

Yay!

[MUSIC PLAYING]

We are a spinoff of MIT. We are fundraising right now and getting the validation and the approval from big companies like Amgen, it really makes the difference in the way investors and the community can see our technology, because it's pre-vetted by big players in the space.

[INAUDIBLE].

One thing that's an accelerator about this community is that there's a lot of shared experience and shared wisdom. So when I have an idea for a new company, I don't have to learn everything from scratch. I can go across the hall, and I can say, is this a good idea? Has this been tried? What's the path forward? Do you know an investor I can talk to? Will you be on my board? How much money do you think I need? Is this the right moment? You know, all of those questions.

[MUSIC PLAYING]

One of the impacts of Kendall Square that I've seen is that kids here of all backgrounds will have opportunities to be exposed to this kind of research and get involved in it.

The Rindge School of Technical Arts is the technical arm of this comprehensive high school. You could take anything from biotechnology, engineering, computer science to culinary, carpentry, business. The biotech program here is building a pipeline of students who then pursue life sciences and become workers in this very dynamic and exciting field of biotechnology in Cambridge.

What happens in industry when there's an issue with, say, food poisoning, right?

So we'll transfer 15 of the B into the Bx.

Today, we were trying to find foods that were contaminated with the pathogenic E. coli bacteria, and the way we're able to do that is that we used polymerase chain reaction, PCR, and restriction enzymes in order to find a certain amount of base pairs that we could use to find where the pathogen was.

See how he's anchoring and stabilizing? That's a good practice.

When we run it in the gels, we could see, based on the marks that it leaves behind, which food sample does contain the pathogenic E. coli.

So let's capture that image and put this in your notebooks, so you can write this up.

A lot of the techniques, things like PCR, Bradford assays, I think a lot of those skills are skills I can take to an agricultural job that I want to do focusing on the sciences in particular.

I've really become confident in moving around and working in the lab. I've gotten to meet, like, a lot of people who are in the scientific careers who are willing to help and mentor me.

When students have this training in these basic lab skills, they are very attractive candidates for companies and research institutes that offer opportunities for high schoolers.

Oh, my god. That is gorgeous.

[MUSIC PLAYING]

Pretty much steadily since the 1950s on, molecular biology has continued to grow in its power exponentially. Every time you think, oh, it's gotta be topping off, you wake up, and something else has fired another booster rocket to get it into even higher orbit.

The most transformational technologies of the 20th century are digital technologies, and I believe that the convergence of biology with engineering will be the technology story of the 21st century.

The advances in quality of life were the result of convergence between engineering and the sciences. Now we're adding life sciences to the mix, and that is creating an explosive benefit to society in particular to the medical domain and health care.

I'm a chemical engineer, and I work in polymer science. We design large molecules, which are able to interact with a number of natural materials like proteins and DNA. We're building a staged nanoparticle attack. It is a one-two punch that we can deliver on a tumor cell to disable it, and then kill it.

The next 10, 20, 30 years is going to be a massive shift based on what machine learning and AI can do, even more than genomes, even more than all the other things that have happened. This is something that can really, really change the game.

Biotechnology has only had a modicum of the impact that it's going to have. Qualitative changes in medicine are going to come with the use of genes for therapy, and that's just beginning.

It's inconceivable that people could figure out how to manipulate the immune system to kill cancer. It's Everest. I mean-- and the fact is that many of the companies in Kendall Square, there are people climbing Everest every day.

The real impact is that we can cure diseases we couldn't cure before. I mean things that were death sentences aren't anymore.

I think it's pretty unlimited what's going to happen in Kendall in terms of biotech, genetic therapies, various RNA therapies, and CRISPR cellular therapies, tissue engineering, digital medicine, artificial intelligence, nanotechnology. I think it's pretty unlimited.