Setting the Pace in Drug Development

 

Drug talk: Scott Bazemore (left), a research associate at Aronex; Dr. Geoffery F. Cox, chairman and CEO of Aronex; Dr. Gabriel Lopez-Berestein, chief of the Section of Immunobiology and Drug Carriers at M. D. Anderson; and Martin P. Sutter, a director of Aronex Pharmaceuticals, discuss the future of new drugs currently in development to treat cancer and its effects.

 

 

Researchers at M. D. Anderson are accelerating their pace of new drug development while reaping the increasing benefits of partnerships with several technology companies.

 

Recent technology transfer milestones include:

 

· Liposomal amphotericin-B, the first drug designed at M. D. Anderson, now is available worldwide and considered standard treatment for life-threatening fungal infections.

 

· The first royalties on the sale of that drug, Albelcet, totaled more than $1 million in 1997.

 

· $1 million was received in late 1997 as part of a sub-license fee for discovery of an important tumor suppressor gene.

 

· The cumulative licensing income for inventions at M. D. Anderson doubled from $2 million in 1996 to $4 million by early 1998.

 

· M. D. Anderson ranked first among U.S. cancer centers in number of patents issued - 118 during the last decade.

 

· Twelve companies created as spinoffs from M. D. Anderson-conducted research provide more than $10 million in equity portfolio.

 

· Ten new drugs in the institution's development pipeline involve anti-cancer agents for several types of cancers as well as leukemia.

 

"These and other exciting activities help demonstrate that M. D. Anderson affords virtually unlimited opportunities for new drug development," says William J. Doty, director of the Office of Technology Development.

 

Since that office was established in 1987, the growth in transferring biotechnology designed at M. D. Anderson to the private sector has soared. In the last fiscal year, a total of 78 patents were filed and 13 were issued. Over the past decade, 540 invention disclosures were made by faculty and staff members, resulting in 118 patents.

 

Doty and his staff utilize expert consultants in determining the commercial potential of inventions and in the complex process of filing patent applications. As a component of The University of Texas System, patents sought by M. D. Anderson are filed on behalf of the UT Board of Regents.

 

Royalties and licensing fees are major benefits for inventors and the institution. The recent successes in both categories have raised awareness of M. D. Anderson as one of the country's top centers fostering development of better drugs as well as techniques and devices to improve cancer treatment.

 

"The commercialization of Albelcet is a great example of how a new drug can progress from a scientist's initial idea to being used throughout the world," Doty says.

 

Albelcet is the brand name for liposomal amphotericin-B, which was developed by Dr. Gabriel Lopez-Berestein when he was a junior faculty member in the early 1980s. The drug has revolutionized treatment for patients with cancer and other major diseases that have impaired their immune systems and left them vulnerable to systemic fungal infections.

 

In 1984, M. D. Anderson filed a patent for Dr. Lopez' technique that incorporates the anti-fungal drug amphotericin-B in tiny fatty carriers known as liposomes. A patent was issued for the drug delivery method three years later.

 

"As I think back to the early days of laboratory and animal testing of liposomal amphotericin-B, I believe coming to M. D. Anderson for a fellowship in 1979 and joining the faculty in 1981 were the best decisions I ever made. The development of this drug is a prime example of the high priority here for translational research, which takes findings in the laboratory and expedites application to patients," notes Dr. Lopez, professor of medicine and chief of the Section of Immunobiology and Drug Carriers.

 

M. D. Anderson's first spinoff company started in 1987, when a group of investors formed The Macrophage Company to commercialize new anti-cancer compounds. The company's name was changed in 1989 to Argus Pharmaceuticals, Inc., and went public with a $12 million stock offering in 1992. At the time, Argus was among approximately 250 of an estimated 1,200 biotechnology companies in the nation to offer public stock.

 

The next milestone occurred in 1995, when Argus merged with two other companies and became Aronex Pharmaceuticals, Inc. Aronex recently moved into new headquarters in The Woodlands. Other promising drugs and delivery techniques from M. D. Anderson faculty are in clinical development and should be available soon for cancer patients.

 

Among drugs being assessed for commercialization at Aronex are:

 

· Nyotran, a liposomal formulation of the anti-fungal drug nystatin. It is being tested in clinical trials in the U.S. and Europe prior to seeking approval from the U.S. Food and Drug Administration for its broader use.

 

· Atragen, a liposomal form of trans-retinoic acid originally evaluated for patients with acute promyelocytic leukemia and now being studied for other types of cancer.

 

· Annamycin, a new anthracycline analog under review in clinical trials for advanced breast cancer patients at M. D. Anderson.

 

Additional novel compounds in the M. D. Anderson development pipeline are in various phases of preclinical testing at Aronex and other companies and in clinical trials to assess their safety and efficacy. This group includes drugs for patients with ovarian, breast, lung, pancreatic, head and neck, and kidney cancers, malignant melanoma and such serious complications as septic shock.

 

Doty believes the fall of 1997 will turn out to have been "the biggest turning point" in assuring accelerated drug development in the future at M. D. Anderson.

 

"In October, we received a total of $1,050,000 from The Liposome Company of Princeton, New Jersey, for royalty sales of Albelcet, which had been licensed originally to E. R. Squibb and Sons, Inc. Squibb (now Bristol-Myers Squibb Company of New York) entered into a collaboration with The Liposome Company to produce commercial quantities of the drug, and then in 1991, they assigned final development of Albelcet to The Liposome Company," Doty explains.

 

The second major event occurred in November, when $1 million was received from Myriad Genetics, Inc., of Seattle, as M. D. Anderson's share of the sub-licensing fee for a critical tumor suppressor gene identified by Dr. Peter Steck and Dr. Alfred Yung of the Department of Neuro-Oncology.

 

A recent showcase of emerging technologies was offered in mid-February when M. D. Anderson President Dr. John Mendelsohn hosted a seminar attended by 55 investment bankers and institutional analysts from across the nation and some other countries. Dr. Mendelsohn and four senior faculty members described their promising research projects.

 

The participants were in Houston for the Texas Biomedical Investment Conference to learn about investment opportunities. Among the 12 Texas-based companies sponsoring the conference were Aronex and Introgen Therapeutics, Inc., of Austin, a privately held company that is developing gene therapy products to treat cancer. Introgen was formed to commercialize gene therapy techniques designed at M. D. Anderson.

 

Looking ahead, Doty expects increasingly successful technology transfer. He predicts royalties and licensing fees will reach $20 million early in the 21st century.

 

"M. D. Anderson has achieved a critical mass in its translational research efforts to bring advances from our laboratories to better therapies for cancer patients," Doty says. "While we are plowing the money earned on these discoveries back into our research, we also are helping enhance the important biotechnology industry in Houston and throughout Texas," Doty says.

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