Some drugs work better, or only, in injectable form. Turn back the clock six years, to what was said then about beloranib. Science looked for a cure that would make the body produce less fat, and burn more of what it did make, for fuel. This result was earnestly desired, to ease the torture of insatiable hunger experienced by patients with Prader-Willi syndrome.
Reporting for Reuters, Esha Dey wrote,
Beloranib works by blocking an enzyme known as methionine aminopeptidase 2, or MetAP2, which plays a key role in the production and use of fatty acids.
[It] leads to a higher rate of fat burn and improves some key conditions related to heart safety, including reducing bad cholesterol and lowering inflammatory actions in the body.
The drug’s safety profile was, at that time, considered impressive. Were there side effects? Of course — mainly nausea, vomiting, and sleep disturbance. But on the up side, subjects given the highest dose lost an average 22 pounds in three months. An on the very far up side, the subjects “continued their normal food and exercise habits.”
If that aspect proved out, it would be an enormous selling point — perhaps even worth the daily needle sticks. Also, the injection concept might be an easier sell because it is subcutaneous, the kind where a hunk of skin is pinched up, rather than intramuscular.
One might have wondered, at the time, why go to so much trouble for Prader-Willi syndrome, a non-mainstream condition with a genetic basis? When a product affects only one out of around 13,000 people, can it possibly return even a fraction of the investment that was made in it?
A big corporation has money to experiment with, but why the interest in such a rare disease? Besides, the way things have been going, the condition might more easily be amenable to genetic manipulation, which would eliminate the need for medication.
On the other hand, the body’s fat metabolization is a subject of lasting interest to many populations. Years later, Derek Lowe wrote for Science Translational Medicine,
I feel sure that the way it was supposed to work was that beloranib was going to demonstrate its use in the Prader-Willi population, where it wouldn’t be so hard to demonstrate benefit and approval had much better chances. Then it (or a followup) could go for the larger diabetes market, using the rare disease revenue stream. This is a common strategy: get a foothold in the smaller, underserved patient population and then try to expand.
There is nothing sneaky about this tactic. It just happens to be the way these things work. Research funding is the pot of gold at the end of many exploratory rainbows, and sometimes the most direct path is not the path that leads there. For good or ill, human institutions can only do so much. Even the best efforts may be thwarted by the inexorable forces of nature.
(To be continued…)
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Source: “CORRECTED — New entrant in obesity drug race targets body, not the mind,” Reuters.com, 08/26/13
Source: “Zafgen’s Second Act,” ScienceMag.org, 05/08/17
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