National Cancer Institute Creates Fund to Assist Research into Orphan Cancers

[Guest guest]

(From a post on Karl's nhl-info list. This sounds like a great

idea. Hope those against using public funds for private companies

don't object too much to this idea.)

EXCLUSIVE REPORTS

St. Jude scientist eyes business for cancer research

Shepard

Stephan isn't an entrepreneur -- yet -- but he

intends to be

one by copying the strategy of GTx, Inc., launching his own

biotech

business and licensing his own research creations.

First, though, is counting on a brand new type of

grant from

the federal government designed to encourage academic

research

centers to collaborate with private companies.

In the lab, already has made significant

breakthroughs in

treating a form of cancer known as anaplastic lymphoma

kinase, but

when he contacted large drug companies with his results,

they

weren't interested because ALK has such a tiny potential

market. ALK

gets its name from the kinase, or protein, that's involved

in the

disease.

" There are only 1,000 new cases a year, " says. " When

your

child has ALK, it's 100% of cancer, but that's not what Big

Pharma

does. As soon as they heard the market size, I heard a

click on the

phone. "

So his research seems tailor-made for a new granting idea

from the

National Cancer Institute, part of the National Institutes

of

Health. After other efforts to encourage collaboration have

failed,

NCI has created the Academic Public Private Partnership

Program --

or the AP4. It's patterned after another granting program

administered by the National Science Foundation, which was

created

in 1973 to link industry with academic research centers

that focus

on engineering and materials development.

That earlier grant program sped the development of

thousands of new

products, with most modern plastics and polymers stemming

from the

project.

The NCI wants scientists to concentrate on orphan cancers

like ALK,

which otherwise cannot get much attention.

" My group works on the molecular genetics of leukemia and

lymphoma, "

says. " We identified the six genes that are mutated

in this

cancer, and the natural extension of that is to try and

develop

therapies to interfere with these genes. "

is a medical oncologist, and for 15 years has been

with the

Department of Pathology and Hematology-Oncology at St.

Jude. '

team developed compounds that block the target genes. By

testing the

same compounds on healthy mice, they also found that the

genes are

not essential to health or longevity, so blocking them

doesn't have

any detrimental side effects. It means has an

effective

treatment, but nobody wanted to listen.

" It's really frustrating for someone like me who has an

orphan

malignancy, " he says. " There are less than 20,000 new cases

of

pediatric cancer each year, so all of them are orphans.

After a lot

of frustration, we decided to do it ourselves. "

He still needed someone on the outside who could handle the

medicinal chemistry, that is, produce the compounds at

pharmaceutical grade, and be able to make compounds with

strict

quality assurance. is collaborating with San

Diego-based

ChemBridge Corp., which specializes in making compounds.

Among its

clients ChemBridge counts Memorial Sloan Kettering Cancer

Center and

GlaxoKline.

" We're rich and strong in chemistry, so we're looking for

academic

partners that are strong in biology to complement us, " says

Tom

Webb, vice president of research and development for

ChemBridge, and

its sister company, ChemBridge Research Laboratories. " We

look at

someone's reputation, their history of accomplishments and

contributions to science and medicine. "

In collaborating with St. Jude, Webb says, it was also

important

that a specific scientist recognize the value of chemistry,

even

though he is oriented toward biology. The two disciplines

are

merging into what's called chemogenomics and chemical

genetics.

In the relationship, and his team handle the

biologics, such

as animals, cultures and drug screening, while ChemBridge

focuses on

building the effectiveness and the potency.

Should be awarded the AP4 grant, St. Jude would be

designated

as an AP4 center, and for the first three years NCI will

match

private contributions at 150%. So if ChemBridge puts in

$100,000,

NCI will add $150,000. Beyond that, NCI's goal is to wean

projects

off the public dole and finance others the same way.

NCI, says, is also becoming entrepreneurial and

wants to spur

self-sustaining businesses, so the proposals most likely to

be

funded on the front end are the ones with the best

commercialization

potential.

included the promising work of four other

scientists, and

envisions forming a business in Memphis that would

licensing these

technologies from St. Jude. It's the same strategy used by

urologist

Mitch Steiner, who started GTx using his own inventions

created at

the University of Tennessee Health Science Center. GTx

holds the

licenses on eight UT patents, and is now preparing for a

$86 million

initial public offering.

In the last two years, has found that the same

kinase

involved in ALK may also be a culprit in other forms of

cancer,

including breast cancer and prostate cancer. While

pediatric cancers

are straightforward, adult cancers are much more complex,

involving

a number of mutations. His compound could be part of the

treatment

cocktail of the future, addressing just one of the

mutations.

It means the potential for his future business is also much

greater.

" NCI wants to pick winners, " says. " They want you to

become

profitable. "

© 2003 American City Business Journals Inc.