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Medical Foods for Alzheimer's: Palatable Therapy or Snake Oil?

( 2 Articles Available, 0 Articles Pending )

In the absence of truly effective treatments, and in the presence of a rapidly growing, dementia-prone population of elders, it's perhaps no surprise that people are increasingly open to products claiming even the slightest hint of promise, however untested those claims may be. The FDA category of "Medical Food"—not really a food, not really a drug—has seen some growth recently, but many people do not know that the nation's drug agency does not check whether these products really work. Do they? 

Medical Foods—Fallback Option for Elusive AD Drug Status?

Two years ago, iffy results from a massive Phase 3 trial led a Canadian drug company to scrap development of its anti-amyloid therapy for Alzheimer disease. Today, seniors can purchase the very same compound, rebranded as a “science-based natural health product,” right off the shelves of Canadian drug stores or from their home computers via the Internet. The field at large could now be witnessing variations of this theme, played out more subtly through the appearance of AD’s first “medical foods.” Earlier this spring, Ketasyn (aka AC-1202)—a ketogenic drink powder that targets metabolic deficiencies associated with AD—hit the market under the trade name AxonaTM based on Phase 2 data reported initially in 2007 (see ARF related conference story) and published last month (Henderson et al., 2009). Meanwhile, recruitment proceeds apace for U.S. and European Phase 3 trials of the multi-nutrient drink SouvenaidTM (aka FortasynTM Connect in powder form), which purportedly boosts the brain’s supply of synaptic membrane constituents (see ARF related conference story).

“This is going to be in the limelight,” said Maria Carrillo of the Alzheimer’s Association. “People are going to be looking at dietary supplements and medical foods more and more, especially with the potential epidemic we're facing with AD.” An estimated 35 million people are living with dementia worldwide, and if present trends continue, the number with AD is projected to nearly double every two decades, reaching 65 million by 2030, according to a new report released last month (see ARF related news story).

Defined by the U.S. Food and Drug Administration in 1988, medical foods are a special class of therapeutic agent that fall somewhere in between unregulated food supplements and FDA-approved drugs (see Morgan and Baggott, 2006). Unlike dietary supplements, which are sold over the counter and intended for healthy people, medical foods target people with a specific disease and generally require a doctor’s prescription. However, a key difference from drugs is that medical foods do not undergo pre-market FDA review or approval, and do not have to be registered with the agency (see FDA Guidance for Industry).

With no FDA efficacy claim, how do consumers know that a medical food will do them any good? “The short answer is that consumers don’t know,” FDA supervisory nutritionist Sue Anderson wrote in an e-mail to ARF (see Q&A below). “Manufacturers should have information to substantiate that claims on their labels are truthful and not misleading. However, the claims are not reviewed by the FDA.”

Regulation Loophole
Therein lies the catch—or lure—depending on one’s perspective. Because medical foods consist of ingredients “generally recognized as safe” (GRAS), they escape much of the safety monitoring normally required for drugs. This, combined with the lack of clear standards for demonstrating efficacy, make the “medical food” designation appealing to investors looking to send their companies’ products on a quicker, cheaper route to market.

Take, for example, AC-1202, a form of medium-chain triglycerides being developed for AD by Accera, a biotech company in Broomfield, Colorado. The compound gets converted by the liver into ketone bodies, which enter the brain to provide an alternative energy source for neurons that no longer use glucose properly. “For the investors, one of the big appeals of our approach to AD is that we’re basically using a food ingredient to generate ketosis,” Sam Henderson, Accera’s executive director of research, told ARF. “You don’t have to go through the normal long drug development process. You can very quickly come to market with something like a medical food.”

In a small proof-of-concept study involving 20 people with mild to moderate AD, those who drank a milkshake containing emulsified AC-1202 had higher blood levels of ketone bodies compared with participants on an isocaloric placebo drink. The AC-1202 patients also had better ADAS-Cog scores several hours after drinking the milkshake, but only if they lacked the ApoE4 allele (Reger et al., 2004). That trial, published with Suzanne Craft at the University of Washington, Seattle, was done on angel financing, Henderson said. The company did secure some funding from venture capitalists, but “they didn’t have the monies to do the long-term development process,” Henderson said. “Their real motivation was rapid development of the product since our main ingredient is already recognized as a safe food ingredient.”

Though similar on their safety profiles, medical foods have one up on food supplements in the area of labeling. Whereas supplements are restricted to structure/function claims (e.g., “protects memory function”), medical food labels can mention specific diseases. According to the company’s website, AxonaTM (trade name for the AC-1202-containing drink) “is a specially formulated medical food intended for the clinical dietary management of the metabolic processes associated with mild to moderate AD.”

What’s in a Label?
This description raises eyebrows—mostly for what it does not claim. While the label indicates that the product alters metabolic problems associated with AD, “it doesn’t actually say whether it benefits AD patients or improves memory or anything like that,” said Douglas Galasko of the University of California, San Diego, in an interview with ARF. “In addition to changing some metabolic problem, there has to be a clinically meaningful outcome.”

Thus far, though, the field may lack scientific evidence to make this a reasonable expectation. It’s the classic question of association versus causation, Galasko said, noting weight loss as an example. “Most people with AD lose weight,” he said. “But they’re not developing dementia because they lose weight.” Likewise, Galasko questions whether addressing a metabolic deficiency associated with AD would actually slow the course of disease. Positron emission tomography (PET) studies have long established glucose hypometabolism in AD-affected brain areas. However, “Is that because the brain has a glucose problem or because synapses aren’t working properly and in fact there’s an appropriate amount of glucose metabolism per functional synapse?” he asked.

Craft, who ran Accera’s initial pilot study suggesting that Axona improved cognition in E4 non-carriers, does not find this chicken-or-egg issue so critical. “Even if energy issues were secondary, that doesn’t mean that supplying energy couldn’t be helpful,” she told ARF. She found the recent data promising (Henderson et al., 2009). “The fact that it was consistent with the pilot study (Reger et al., 2004) suggests, to me, that this is a real effect. It looked like there was some measurable benefit for patients who didn’t have the E4 allele. Those folks showed a three- to four-point ADAS-Cog benefit relative to placebo,” Craft said.

Nevertheless, the study failed to meet its primary endpoint—improved ADAS-Cog scores at day 90, relative to placebo, in treated participants at large. There was a meaningful ADAS-Cog signal compared to placebo at 45 days, but this effect largely dissipated by 90 days. “That’s worrisome,” Galasko said. “The FDA would not license a drug based on 45-day effects that were no longer present at 90 days.” In a phone interview with ARF, Henderson said the company had never filed an IND (Investigative New Drug application) and was “always planning on something like a medical food.” However, Steve Orndorff, Accera’s founder and CEO, spoke of different intentions for Axona in another story.

Silver Lining in ApoE4 Effects?
Craft agrees that the company still “has not done the definitive Phase 3 study” and thinks Axona’s apparent E4 differential benefit deserves further research. “It’s a fairly low-risk product. There’s GI upset (i.e., diarrhea in 24.4 percent of the treated participants versus in 13.6 percent of the placebo group), and of course that’s always a concern when used in a prolonged fashion in an older population. But other than that, there aren’t any identified serious adverse events associated with it. It seems they did show fairly robust benefit for a subgroup of folks,” Craft said. “I’d just like to see them test it one more time in a study that was designed and powered to specifically test this E4 differential response.” PET studies led by Eric Reiman, who heads the Arizona Alzheimer’s Consortium and directs the state’s Banner Alzheimer’s Institute in Phoenix, have shown that ApoE4 gene dose correlates with lower glucose metabolism (Reiman et al., 2005) and more amyloid-β (Reiman et al., 2009 and ARF related news story) in AD-affected brain areas.

Possible E4 effects were also buried within pre-planned exploratory analysis of data from the Alzheimer’s Disease Cooperative Study’s (ADCS) trial of docosahexaenoic acid (DHA). In this study, 402 mild to moderate AD patients at 51 U.S. sites took two grams of DHA daily for 18 months. This treatment had no effect on participants’ scores, relative to placebo, on the ADAS-Cog or the Clinical Dementia Scale Sum of Boxes (CDR-SOB), principal investigator Joseph Quinn, Oregon Health and Sciences University, Portland, reported in July at the International Conference on Alzheimer’s Disease (ICAD) in Vienna. However, when the data were re-analyzed in participant subsets defined by ApoE genotype, the researchers found that E4 non-carriers who received DHA declined more slowly on the ADAS-Cog and showed a similar trend on the Mini-Mental State Examination (MMSE). DHA treatment did not help people who had the E4 gene.

DHA supplements did seem to bring modest cognitive benefit in a six-month Memory Improvement with DHA Study (MIDAS) by Martek Biosciences Corporation, Columbia, Maryland, the primary supplier of algal DHA for supplementation. However, unlike the ADCS trial, which involved early AD patients, the MIDAS study enrolled healthy seniors with mild memory complaints. Relative to the placebo group, those who took 900 mg daily DHA capsules made fewer errors on the CANTAB Paired Associate Learning (PAL), an episodic memory test, compared to when they began the study. Some may interpret these findings to suggest that DHA could be more effective as a preventive therapy rather than a treatment for AD. “I wouldn’t jump to that one just yet,” Quinn told ARF. “The effects in the MIDAS study were pretty modest. It’s hard to know the clinical significance of those modest effects. The participants were not selected to represent a prodromal population.”

Discussion is underway for a future ADCS trial in people with mild cognitive impairment (MCI). “We want to look at a group of people who are at really high risk for AD, and look for evidence that DHA intervention could have an effect on outcomes that are more clinically meaningful,” Quinn said. Besides ADAS-Cog, these could include measures that more reliably capture the transition to dementia. The trial would also include stratification for ApoE genotype. “We probably won't exclude E4-positive subjects. But we will be more careful to stratify enrollment and make sure there are enough in each category,” Quinn said.

The MIDAS trial did not obtain participants’ ApoE genotypes, Karin Yurko-Mauro of Martek told Alzforum after her ICAD press briefing. However, Paul Aisen, University of California, San Diego, who heads the ADCS, said, “You have to assume that most people in that trial are E4-negative just based on ApoE’s population distribution and the fact that they were cognitively normal (i.e., did not have AD) at this age.” In the ADCS DHA trial, nearly 60 percent of participants were E4-positive. While ApoE effects are viewed with interest across the field these days, they have not so far panned out in trials designed to follow up on initial Phase 2 ApoE3 differential effects. A recent example is GlaxoSmithKline’s Phase 3 pharmacogenomic trial of the diabetes drug rosiglitazone, which grew out of an unexpected ApoE signal in Phase 2 (see ARF related news story). At this year’s ICAD in Vienna, the company reported that the compound had no impact on any of the ApoE-stratified subgroups (see Doctor's Guide news story). GSK’s Michael Gold noted that rosiglitazone testing for Alzheimer’s had ended (see also page 10 of press release [.pdf]).

Confirming ApoE effects in medical foods could present yet additional complications. For example, if DHA were to show benefit in ApoE4-negative MCI patients, one could imagine the troublesome scenario in which people would need genetic testing to be prescribed a medical food. This could mean that potential consumers who test as E4-negative would be eligible to take something lacking an FDA efficacy statement, while those who come up E4-positive would get hit twice—with news of elevated AD risk, and without another potential treatment option.—Esther Landhuis.

This is Part 1 of a two-part series. See Part 2

Q&A With Sue Anderson, FDA supervisory nutritionist. Questions by Esther Landhuis.

Q: What is a "medical food"?
A: In simple terms, a medical food is a product that is specially made because in no way can a regular diet be modified to meet the specific and distinctive nutritional needs of a disease or condition. It is not a product that is merely provided to sick people, although it may have a beneficial effect. It is a formulated product that contains ingredients shown to be directly linked biochemically or metabolically to the disease or condition in humans.

This website contains the statutory definition that has existed since 1988. There have not been any changes or updates to the definition.

Q: What key features distinguish a medical food from a drug?
A: Basically, intent of use and the provision of nutrition support by a medical food. There are specific requirements for pre-market safety and efficacy studies for new drugs and FDA approval of new drug products that are not required for medical foods.

Q: How is a medical food different from a nutraceutical?
A: There is no regulatory definition or framework for “nutraceuticals.”

Q: When was the "medical foods" designation created? Why?
A: The actual words “medical foods” began to be used in 1975. However, the statutory definition was not created until 1988. The most likely reason for the designation stemmed from the change in regulatory practices for such products. Products for inborn errors of metabolism used to be regulated as drugs, but were reclassified as foods and regulated under food law in 1972 so that the products would not have to undergo the pre-market testing and approval required for drugs.

Q: Can you give examples of where the "medical foods" designation has been granted in other disease areas?
A: No “granting” of a designation or classification is made given that there is no pre-market review or approval for medical foods. [Editor’s note: A review (Morgan and Baggott, 2006) mentions Lofenalac (a powder mixed into formula for babies and children with phenylketonuria) as an early example. Newer medical foods target pancreatic and renal insufficiencies (Ultrase MT and Renax), elevated homocysteine (Folgard RX 2.2), and other conditions including vascular dementia and AD (CerefolinTM)].

Q: Do insurance companies generally reimburse for medical foods? Are they expensive like drugs, or more like a food?
A: FDA does not regulate insurance coverage. The internal policies and practices of individual insurance companies differ. Medical foods are regulated under existing conventional food law and are not drugs. Some insurance companies may provide coverage and some others may not for varying reasons. Medical foods can range in cost from reasonable and affordable to thousands of dollars per year (i.e., medical foods used to manage inborn errors of metabolism).

Q: With no efficacy claim by the FDA, how do consumers know that a medical food will do them any good? Are medical foods required to meet any efficacy standards?
A: The short answer is that consumers don’t know. Manufacturers should have information to substantiate that claims on their labels are truthful and not misleading. However, the claims are not reviewed by FDA.

References

News Citations

  1. Washington: Shaking Up AD Treatment with Ketone Bodies
  2. Chicago: Phase 2 News—Therapeutic Breakfast Food?
  3. World Alzheimer’s Report Estimates 35 Million Cases
  4. More ApoE4 Means More Amyloid in Brains of Middle-Aged
  5. Madrid: Highs and Lows of The Insulin Connection
  6. Medical Foods—Food for Thought, But Think Twice

Paper Citations

  1. Henderson ST, Vogel JL, Barr LJ, Garvin F, Jones JJ, Costantini LC. Study of the ketogenic agent AC-1202 in mild to moderate Alzheimer's disease: a randomized, double-blind, placebo-controlled, multicenter trial. Nutr Metab (Lond). 2009;6:31. PubMed.
  2. Morgan SL, Baggott JE. Medical foods: products for the management of chronic diseases. Nutr Rev. 2006 Nov;64(11):495-501. PubMed.
  3. Reger MA, Henderson ST, Hale C, Cholerton B, Baker LD, Watson GS, Hyde K, Chapman D, Craft S. Effects of beta-hydroxybutyrate on cognition in memory-impaired adults. Neurobiol Aging. 2004 Mar;25(3):311-4. PubMed.
  4. Reiman EM, Chen K, Alexander GE, Caselli RJ, Bandy D, Osborne D, Saunders AM, Hardy J. Correlations between apolipoprotein E epsilon4 gene dose and brain-imaging measurements of regional hypometabolism. Proc Natl Acad Sci U S A. 2005 Jun 7;102(23):8299-302. PubMed.
  5. Reiman EM, Chen K, Liu X, Bandy D, Yu M, Lee W, Ayutyanont N, Keppler J, Reeder SA, Langbaum JB, Alexander GE, Klunk WE, Mathis CA, Price JC, Aizenstein HJ, Dekosky ST, Caselli RJ. Fibrillar amyloid-beta burden in cognitively normal people at 3 levels of genetic risk for Alzheimer's disease. Proc Natl Acad Sci U S A. 2009 Apr 21;106(16):6820-5. PubMed.

Other Citations

  1. see Q&A below

External Citations

  1. FDA Guidance for Industry
  2. another story
  3. Doctor's Guide news story
  4. page 10 of press release

Further Reading

Papers

  1. Henderson ST, Vogel JL, Barr LJ, Garvin F, Jones JJ, Costantini LC. Study of the ketogenic agent AC-1202 in mild to moderate Alzheimer's disease: a randomized, double-blind, placebo-controlled, multicenter trial. Nutr Metab (Lond). 2009;6:31. PubMed.
  2. Morgan SL, Baggott JE. Medical foods: products for the management of chronic diseases. Nutr Rev. 2006 Nov;64(11):495-501. PubMed.

Medical Foods—Food for Thought, But Think Twice

Updated on 23 October 2009.

While the Alzheimer disease field awaits a bona fide drug that goes beyond temporary relief of symptoms to actually slow disease progression, patients and caregivers may be increasingly open to products claiming even the slightest hint of promise—however untested those claims may be. It appears that some investors are banking on this very attitude. Hoping for a cheaper and quicker route to market, they have prodded several companies to veer off the AD drug development course and instead sell their products as nutraceuticals or medical foods. Items with these designations do not undergo pre-market review by the U.S. Food and Drug Administration and, as such, bypass the gauntlet of safety and efficacy trials typically required for drug approval (see Part 1 of this series).

“I believe the current situation is unfortunate,” said Paul Aisen, University of California, San Diego, who heads the Alzheimer’s Disease Cooperative Study. “It’s very difficult for consumers to even tell that medical foods are marketed without any basis for efficacy.”

Aisen led the Phase 3 trial for tramiprosate (Alzhemed), a compound once being developed by Neurochem of Laval, Québec, as an amyloid inhibitor for treatment for AD. Results of that trial, which involved 1,052 AD patients at 67 North American sites, were ruled inconclusive by the FDA in 2007 (see ARF related news story). The following year, the company changed its name to Bellus Health and began selling the same compound (also known as 3-amino-1-propanesulfonic acid or homotaurine, an amino acid found naturally in seaweed) under the brand name VivimindTM in Canadian drug stores and over the Internet (see ARF related conference story and ARF related news story).

With terms cleverly chosen to target healthy baby boomers, the company website describes VivimindTM as a “patented, science-based natural health product shown to protect memory function.” The site draws from post-hoc analysis of Phase 3 magnetic resonance imaging (MRI) data to claim that the compound works by slowing shrinkage of the hippocampus. These findings were published several months ago (Gauthier et al., 2009).

These claims distract from the bottom line, Aisen said. “There may be a number of factors that contribute to the negative result, including methodological issues, but the fact remains that the trial was negative,” he told ARF. “I do not think there is a basis for recommending tramiprosate to people with AD.” Regarding the possibility that the compound slows hippocampal atrophy, Aisen said, “It’s interesting...but until you have confirmed it in an independent trial, it’s just exploratory and entirely inconclusive because you’re playing with the data after the fact.”

Nevertheless, VivimindTM sells for CAD$45 per 60-tablet box, and by some measure, people are buying it. Gross sales totaled $82,000, though marketing and selling expenses from commercialization of VivimindTM amounted to 560,000 over the same time period, according to a fiscal report from the company’s website. Over the summer, the company announced that it received permission to sell VivimindTM as a food supplement in Italy (see news release). In the same report, company officials said they have filed a pre-market notification of a New Dietary Ingredient for homotaurine with the FDA, and are “pursuing mandatory associated regulatory activities to obtain marketing approval” of the compound as a dietary supplement.

Risks for Consumers and Investors
With medical foods, the regulatory loophole can be harder to recognize. Medical foods generally require a prescription, make claims for specific diseases, and must meet certain safety and manufacturing standards—making them appear almost like drugs to the lay consumer. In marketing literature, manufacturers couch their food products in drug language, with implicit assumptions of efficacy and FDA review. For example, Pan American Laboratories (PAMLAB) L.L.C. of Covington, Louisiana, is described on the company website as “a fully integrated pharmaceutical company...specializing in prescription medical foods.” A product website describes one of these food products, Cerefolin, as caplets “indicated for the distinct nutritional requirements of individuals under a physician's treatment for neurovascular oxidative stress and/or hyperhomocysteinemia, with particular emphasis for those individuals diagnosed with or at risk for mild to moderate cognitive impairment, vascular dementia, Alzheimer's disease, and/or recurrent or ischemic stroke.” The website for AxonaTM, the ketogenic drink mix that targets metabolic deficiencies associated with AD, assures lay readers that “claims for both medical foods and drugs must be supported by solid laboratory and clinical data.”

However, the FDA’s definition does not specify efficacy requirements for medical foods. “Because we understand that medical foods don't undergo the FDA's stringent review like treatment options, we cannot be assured that they are actually going to show effectiveness,” said Maria Carrillo of the Alzheimer’s Association, which issued a statement [.pdf] on medical foods earlier this spring. “If (companies) are making claims for treatment effects that improve cognition and AD, we really do need to see the large Phase 3 trial.”

Even though medical foods are formulated with ingredients “generally regarded as safe” (GRAS), safety concerns do not disappear altogether. “It's important for people to realize that though these are nutritional supplements, they are chemicals,” Carrillo said. “They can counteract other medications or even other concomitant factors, e.g., heart disease, high blood pressure. They could exacerbate some things while helping others.” With medical foods, there also is no post-approval safety monitoring as there is with drugs.

Furthermore, it’s not clear how well medical foods would do in the market if insurance plans do not cover them. AxonaTM hit the market in March, and costs about $3.70 per daily packet. “We don’t have any firm numbers on how many scripts are covered by insurance,” said Sam Henderson, executive director of research at Accera, Broomfield, Colorado, which develops AxonaTM. “Anecdotally, we know it has been covered in some cases, but my guess is that most people are paying cash.” Henderson told ARF the company prefers not to share sales data at this time.

Duel of the Breakfast Foods
Another therapeutic drink may be poised to challenge AxonaTM in the AD medical food arena several years later. This one, SouvenaidTM, is a once-a-day milkshake loaded with ingredients (including DHA, omega-3 fatty acids, and antioxidants) said to help build neuronal membranes and synapses. It is developed not by a pharma or biotech company, but by Nutricia, a medical nutrition company under Groupe Danone, Paris (Dannon to Americans). In a trial of 212 early AD patients at 28 U.S. and European sites, consuming the drink once a day for 12 weeks moved scores on one of the primary outcome measures—a delayed verbal memory task (WMS-r)—but did nothing for the other (an extended ADAS-Cog battery called ADAS-Cog-13). Lead investigator Philip Scheltens of Vrije University Medical Center in Amsterdam, The Netherlands, reported these 12-week data at the 2008 International Conference for Alzheimer’s Disease in Chicago (see ARF related conference story). A manuscript describing the findings is under review at the journal Alzheimer’s & Dementia, Scheltens told ARF via e-mail.

Because it is not a unique chemical entity but rather a patented combination of basic ingredients, SouvenaidTM is not a drug, Scheltens noted. It falls under Foods for Special Medical Purposes (FSMP), an E.U. designation similar to medical foods in the U.S. in that products are subject to food legislation on labeling and manufacturing, but require a doctor’s prescription (see FSMP guidelines). The regulatory path for FSMPs differs from country to country, as do reimbursement guidelines.

SouvenaidTM is not yet available anywhere in the world, even though it could be licensed in Europe as an FSMP based on available data, according to Scheltens. “As PI of the trial program, I insisted on gathering more evidence first,” he wrote to ARF. Enrollment is ongoing for three larger trials: a six-month U.S. trial that tests SouvenaidTM as an add-on to cholinesterase inhibitors in people with mild to moderate AD, a six-month E.U. trial in drug-naïve, mild to very mild patients, and a 24-month trial of prodromal AD patients (defined as per Dubois et al., 2007). The six-month U.S. trial will use 24-week change in ADAS-Cog as its primary outcome measure, while the six-month E.U. trial will use the Neuropsychological Test Battery (NTB). Both are sponsored by Danone Research B.V. in Wageningen, The Netherlands. A modified version of the NTB (Harrison et al., 2007) will serve as the primary endpoint for the 24-month prodromal AD study. This trial is sponsored by the European LipiDiDiet project, which focuses on using lipids in therapeutic and preventive approaches to aging and dementia.

The Way Forward
One might presume that the design of the SouvenaidTM program was influenced by the fate of Alzhemed—an AD compound that shifted from putative drug to dietary supplement after a negative Phase 3 trial (see ARF related conference story and ARF related news story). However, Scheltens said his team was “committed to the Phase 2 trial and the classification of Souvenaid as a medical food one to two years before (they) knew the direction Alzhemed was going.”

With three Phase 3 trials in the works, it appears that SouvenaidTM is being held to the same efficacy standards required for approved drugs. “We have applied pharma standards to this medical food product,” Scheltens wrote. “This is highly appropriate and, in my view, the only way forward. When it comes to efficacy requirements, my understanding is that medical foods should deliver proven benefits to patients, just as drugs do.”

If the Accera and Neurochem experiences are any guide, some suspect this trial strategy could be a win-win for Nutricia, because with positive results Souvenaid could be sold with stronger claims, and without such results, perhaps as a food supplement with lesser claims. Accera, the company that moved its product to market as a medical food after it missed its primary end point in phase 2, has among its investors Inventages Venture Capital Investment Inc. This group is the private equity and venture capital arm of Nestle SA, the world's largest food company.

Meanwhile, as the field gets a better handle on the utility of various biomarker and imaging modalities (see ARF ADNI update), trials will be designed in ways to improve chances of demonstrating efficacy. One element that will figure prominently in newer AD trials is the shift toward analysis of milder patients, as one of the Souvenaid trials is already doing (see ARF related conference story). “It’s very clear that the disease modification needs to happen in advance of damage to the brain,” said Barry Greenberg in an interview with ARF. Greenberg joined the University Health Network in Toronto, Ontario, after leaving Neurochem. Regarding Alzhemed and other compounds that have been tested in established disease, “there is a possibility they might have shown more of a signal if they had been started in identifiable pre-symptomatic patients,” Greenberg said. “Five years from now, I think we’ll be doing trials differently from how we do them today. We’re going to be able to identify patients who can be treated before their brains are too damaged to be repaired.”—Esther Landhuis.

This is Part 2 of a two-part series. See Part 1.

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References

News Citations

  1. Medical Foods—Fallback Option for Elusive AD Drug Status?
  2. FDA Deems U.S. Alzhemed Trial Results Inconclusive
  3. Philadelphia: European Trial of Alzhemed Ends, Marketing Morphs to Supplement
  4. Experts Slam Marketing of Tramiprosate (Alzhemed) as Nutraceutical
  5. Chicago: Phase 2 News—Therapeutic Breakfast Food?
  6. ADNI: One-year Data Narrow Field of MRI, FDG-PET Approaches
  7. Chicago: Trial Design Bedevils Search for New AD Drugs, Part 1

Paper Citations

  1. Gauthier S, Aisen PS, Ferris SH, Saumier D, Duong A, Haine D, Garceau D, Suhy J, Oh J, Lau W, Sampalis J. Effect of tramiprosate in patients with mild-to-moderate Alzheimer's disease: exploratory analyses of the MRI sub-group of the Alphase study. J Nutr Health Aging. 2009 Jun;13(6):550-7. PubMed.
  2. Dubois B, Feldman HH, Jacova C, Dekosky ST, Barberger-Gateau P, Cummings J, Delacourte A, Galasko D, Gauthier S, Jicha G, Meguro K, O'brien J, Pasquier F, Robert P, Rossor M, Salloway S, Stern Y, Visser PJ, Scheltens P. Research criteria for the diagnosis of Alzheimer's disease: revising the NINCDS-ADRDA criteria. Lancet Neurol. 2007 Aug;6(8):734-46. PubMed.
  3. Harrison J, Minassian SL, Jenkins L, Black RS, Koller M, Grundman M. A neuropsychological test battery for use in Alzheimer disease clinical trials. Arch Neurol. 2007 Sep;64(9):1323-9. PubMed.

External Citations

  1. fiscal report
  2. news release
  3. company website
  4. product website
  5. website
  6. statement
  7. FSMP guidelines
  8. six-month U.S. trial
  9. six-month E.U. trial
  10. 24-month trial
  11. LipiDiDiet
  12. Inventages Venture Capital Investment Inc

Further Reading

Papers

  1. Gauthier S, Aisen PS, Ferris SH, Saumier D, Duong A, Haine D, Garceau D, Suhy J, Oh J, Lau W, Sampalis J. Effect of tramiprosate in patients with mild-to-moderate Alzheimer's disease: exploratory analyses of the MRI sub-group of the Alphase study. J Nutr Health Aging. 2009 Jun;13(6):550-7. PubMed.