Dr. Alejandro Lloret in his office at Isis (photo by HDSA-San Diego)

Speaking with the scientists at Isis Pharmaceuticals, Inc., you get a sense of the crucial human ingredients going into the creation of what could be the very first treatment for Huntington’s disease.

The men and women on the Isis team clearly are highly talented, many of them with advanced degrees from some of the world’s leading universities. Isis recruits top scientists from around the world – just walk down its halls and you will meet people from Asia, Europe, Mexico, and different parts of the United States. They use a highly specialized scientific vocabulary but know how to make it understandable to the average person.

Isis people are focused, driven, enthusiastic, and dedicated to their mission to find treatments and cures for diseases.

History in the making

And they are imaginative, using a cutting-edge technology – antisense – in which few people or investors put stock but which could revolutionize the pharmaceutical industry.

You get the feeling at Isis that history is being made – quietly, but confidently and inexorably.

The Isis team knows it bears a huge responsibility. Their work is about improving – and saving – lives.

For all of this to work, compassion is necessary. 

A young HD researcher

You’ll find it in people like the young, empathetic, and optimistic Alejandro Lloret, who received his Ph.D. in biomedical science at the Universidad Nacional Autónoma de México in Mexico City, the largest university in Latin America, and conducted post-doctoral work on Huntington’s disease at the Massachusetts General Hospital/Harvard Medical School.

The Mexico City-raised Dr. Lloret, whose parents are both M.D.’s, became a scientist because of his interest in biology. (Click here to listen to Dr. Lloret’s message to the Spanish-speaking community about Huntington’s disease.) As an undergraduate, he studied the genetics of yeast and gradually worked his way up the chain of life to humans.

At Harvard, Dr. Lloret worked with other scientists in the search for so-called modifier genes for Huntington’s disease. Whereas the defective huntingtin gene, discovered in 1993, is the root cause of Huntington’s, researchers believe that the modifier genes could determine other aspects of the disease, for example, the age of onset. As with defective huntingtin, which Isis aims to block in its multi-million-dollar effort to find a Huntington’s antisense drug, the modifier genes could become targets for drugs.

Dr. Lloret spent four years on the Harvard project. It could still take several years before a modifier gene is found, he said.

A life-changing experience

The lab work on modifier genes was long and demanding and had no connection to the everyday realities of the disease. But then, after meeting a young woman at a Huntington’s disease meeting in England several years ago, Dr. Lloret had an experience that abruptly changed his life.

“We sat at the same table. She was smoking one cigarette after another. And I just kept wondering, ‘Why is this girl smoking so much?’” he recalled. “And I think she knew what I was thinking. She just looked me straight in the eyes and said, ‘If I have to choose between dying of Huntington’s disease and of cancer, I choose to die of cancer.’

“She was 18 years old. She had the mutation. She told me that she eventually will develop the disease. She was amazed to meet someone that was working on the disease that she will eventually develop. That for me was a shock. Working in a lab is not the same thing as meeting the patients.

“That’s when I decided to go back to the lab and redouble my efforts on my research, because I wanted to help her. I don’t want her to die of either of those diseases. That’s terrible. That was the turning point in my career.”

Meeting the patients

Since then, Dr. Lloret has had much contact with people suffering HD symptoms as well as asymptomatic, at-risk individuals. He has visited patients and given talks on the disease in Mexico City.

In November 2006 he was deeply moved by further contact with HD when he attended the annual Thanksgiving dinner at the Guthrie Center in Great Barrington, Mass. “They read a piece of a journal from a patient with Huntington’s disease,” Dr. Lloret recalled. “He said that every single day that passes, there was little bit of him that was dying – that each word that he was placing in that journal, was a word that he will not remember the next day.”