MZ, What do you think Amgen would feel about this? They are also working on Parkinson and Huntington's, but using the pump. I want to see a bidding war for ctii when amgen and genentech fight out for this little treasure.
Gains In Treatment Of Diabetes, Huntington's Seen Possible
Dow Jones News Service ~ March 26, 1997 ~ 9:38 pm EST By Laura Johannes Staff Reporter of The Wall Street Journal
One of the longstanding frustrations in biotechnology is that discovery of potential cures has outpaced the ability to deliver drugs into the body. The problem is particularly severe in the brain, where the body's natural defenses keep out drugs given by conventional oral or injection methods.
But two new research reports dealing with animal tests of drugs to fight diabetes and Huntington's disease have raised the chances of finding solutions, including the long-sought possibility of diabetics who use injections throwing away their needles and replacing them with insulin pills. The reports appear in today's issue of Nature.
One paper, by an international team of researchers that includes drug-delivery company CytoTherapeutics Inc., described how nerve-cell damage in monkeys -- designed to simulate the ravages of Huntington's disease -- was prevented by a therapeutic protein delivered through a plastic brain implant. The other, by researchers at Brown University, showed that another type of tiny capsule given to rats by mouth effectively delivered insulin and other drugs into their bloodstream.
"If my dream comes true, diabetics won't need to inject themselves dailymaybe just take a pill after meals," said Brown researcher Edith Mathiowitz. She cautioned that a marketable product could take 10 years to develop -- in part because of the danger that insulin could be potentially toxic if too much was released too quickly from the capsules.
Another option, perhaps closer to fruition, is research by W.R. Grace & Co. to develop an artificial pancreas. The drug-delivery device, about the size of a hockey puck, is implanted in the abdomen and filled with live pancreas cells that make insulin. It is now in early trials in humans, and the company says it hopes the device will help diabetics "lead more normal lives."
How to get relatively large protein drugs into the body has been a problem since the earliest forms of such drugs, which include insulin. Protein drugs cannot be taken by mouth, because unlike smaller chemical compounds, they are simply digested as if they were food. The problem has become more urgent with the recent approval of protein treatments for multiple sclerosis, while biotechnology, the science of gene-manipulation, is often aimed at producing protein therapies.
Research into ways to make orally-administered protein drugs is a very hot area. Emisphere Technologies Inc., of Hawthorne, N.Y., is working on technology that uses "carrier" molecules that bind to drugs and transport them across membranes. The small company recently signed an agreement with Eli Lilly & Co. to develop orally available drugs for growth disorders. Lilly makes human growth hormone, which is now given by injection to children who suffer from dwarfism.
But perhaps the biggest venue for alternate drug-delivery methods is the brain. Cures for many brain disorders have been stymied by the so-called blood-brain barrier, a semipermeable membrane that prevents large compounds in the blood from entering the fluid that surrounds brain cells. CytoTherapeutics, of Providence, R.I., has spent the past seven years trying to find ways to penetrate the barrier.
In the Nature paper, the company describes how it used an unusual device -- a tiny plastic capsule filled with genetically engineered cells that make a therapeutic protein called ciliary neurotrophic factorto deliver the drug directly into the brain. To test the system, researchers implanted the capsule in monkey brains then injected the brains with acid -- which kills the same nerve cells that die in Huntington's patients.
(END) DOW JONES NEWS 03-26-97
9 39 PM
Gains In -2-: Huntington's Strikes In Middle Age
The drug protected the cells -- lending new hope to the 30,000 people in the U.S. who suffer from Huntington's disease. Huntington's strikes in middle age. As nerve cells die, patients lose control of their bodies, moving in involuntary, jerky motions, and suffer gradual mental deterioration. The disease is fatal.
A clinical trial in humans, mainly to test safety of the CytoTherapeutics capsule, will begin later this year in Europe. CytoTherapeutics, collaborating with Genentech Inc., hopes to use the same device to deliver drugs for other central nervous system diseases, from Parkinson's to Lou Gehrig's disease.
"As our population is aging, we are faced with more and more disorders involving the process of natural degeneration," said Seth Rudnick, CytoTherapeutics' chief executive officer. "Our technology uses small devices that serve as miniature factories to make [therapeutic] proteins for years or months on end."
The Brown researchers found they could get rats to orally absorb two drugs, including insulin and an anticoagulant called dicumarol. The system involves a tiny plastic capsule that wedges between the cells that line the intestines and sticks there. Gradually, says Dr. Mathiowitz, the plastic coating comes off and the drug is released.
Her group also successfully delivered genetic material into rat liver cells using the oral capsules -- raising the possibility that such a capsule could eventually be used for gene therapy.
(END) DOW JONES NEWS 03-26-97
9 40 PM |