Articolo di Matteo Scibilia preso da www.ccsviitalia.org
La Myelin Repair Foundation (MRF), di cui Scott Johnson - un malato di SM che ha deciso 9 anni fa di avviare questa Fondazione, con l'obiettivo di trovare una soluzione per la remielinizzazione - è CEO, presidente e fondatore, ha annunciato di aver raggiunto un accordo con National Institutes of Health per uno studio, che dovrà verificare l'utilizzo di un farmaco per la ipertensione - già in commercio - per la terapia della Sclerosi Multipla (SM).
Il nome del farmaco non è stato rivelato, e gli è stato per adesso attribuito il codice MRF-008.
Avevamo già parlato precedentemente di Lisinopril, un ace-inibitore utilizzato per l'ipertensione. Non sappiamo se si tratti dello stesso prodotto o di uno simile. La cosa importante è che, in questi casi, trattandosi di prodotto già in commercio da una ventina di anni e utilizzato da milioni di persone, si può passare velocemente agli studi sulla sua efficacia, essendo la parte relativa alla sicurezza già confermata.
I ricercatori ritengono che MRF-008 potrebbe fermare la degenerazione della mielina e promuovere la crescita di nuova mielina.
Questa prima fase potrebbe completarsi entro 15 mesi, per poter poi passare ad uno studio con un maggior coinvolgimento di pazienti.
Il lavoro della MRF è particolarmente importante perché la maggior parte della ricerca fino a un decennio fa si è concentrata sul controllo dell' infiammazione causata dal sistema immunitario. La fondazione, invece, si è concentrata su metodi per fermare l'erosione della mielina e potenzialmente ripristinare la mielina nei pazienti con SM.
"Se parlate con la maggior parte delle aziende impegnate nella SM, adesso esse vi diranno che la prossima generazione di trattamento riguarda la riparazione delle mielina", ha detto Johnson . "Abbiamo spostato il tiro così lontano in soli nove anni. E questo è un enorme risultato."
A generic high blood pressure drug could unlock a new treatment window for multiple sclerosis patients in a groundbreaking study championed by a Bay Area nonprofit.
The Myelin Repair Foundation of Saratoga in the next few months will launch a Phase I safety trial of the drug, approved two decades ago to treat hypertension, said President and CEO Scott Johnson.
But while the road to winning Food and Drug Administration approval as a low-cost MS treatment is as tricky as it would be for any other compound, the drug’s generic use complicates the economics. For-profit drug makers may hesitate to invest in costly clinical trials, since the generic version would likely undercut the price a company could charge.
That restriction on pricing, in turn, could slow the drug’s availability on a broad scale for the world’s roughly 2.3 million MS patients.
What’s more, insurers may balk at covering a generic heart drug’s unapproved use in MS, even if physicians prescribe it.
But if a company tweaks the generic drug’s formula to receive new patents covering MS, the company could charge a price that covers clinical trial costs and make a tidy profit.
For the Myelin Repair Foundation, those development and commercialization issues have been prominent over the past several months. Foundation leaders have been in discussions with advisers — including a representative of Cambridge, Mass.-based MS drug developer Biogen Idec Inc. (NASDAQ: BIIB) — academic researchers and federal regulatory officials.
Now, thanks to an arrangement with the National Institutes of Health announced Monday, the foundation hopes to raise $2.5 million to cover its study costs.
“We never imagined funding any part of clinical trials,” said Johnson, an MS patient who 10 years ago founded the MRF. “All we care about is patients and getting treatments to patients.”
Moving quickly
A couple decades of safety data from the drug’s use for high blood pressure could accelerate its development in MS. The Phase I study — which typically would look at the drug’s safety in healthy volunteers — will focus only on MS patients, and a smaller number of patients at that, Johnson said. That way, he said, researchers can see if any distinct side effects pop up in people with the disease.
Historically, the drug’s side effects include drowsiness and dry mouth.
The trial, Johnson said, could “set a standard for future myelin repair clinical trials.”
Multiple sclerosis is a chronic roller coaster of a disease, highlighted by recurring, increasingly debilitating flare-ups followed by extended recovery periods. It is a particularly difficult disease to treat because no two people have exactly the same experience.
About 2.3 million people worldwide have MS, according to the National Multiple Sclerosis Society.
As its name suggests, the Myelin Repair Foundation’s research has zeroed on the fatty myelin sheaths that wrap around and protect nerve cells in the central nervous system. The loss of myelin is akin to exposing wires in an electrical cord — in this case causing neurons to misfire and leading to difficulty walking, vision problems and other symptoms.
The Myelin Repair Foundation isn’t revealing the generic drug’s name for now but is calling it MRF-008.
Researchers believe MRF-008 could stop the degeneration of myelin as well as promote the growth of new myelin by modulating a pathway, known as the endoplasmic reticulum stress, or ER stress. That pathway is believed to go into overdrive in MS patients, but early research shows it is controlled by MRF-008.
The foundation’s Phase I study is one of the first — if not the first — undertaken with a cooperative research and development agreement between a nonprofit and the NIH, Johnson said. The deal splits clinical trial responsibilities — and, importantly, costs — between the foundation and the NIH.
The study could be completed within 15 months of its start and move quickly into a larger mid-stage trial, Johnson said.
The $2.5 million price tag is minute for for-profit drug developers, but is a substantial investment for a nonprofit.
Although the economics of drug development make it unlikely that a company would jump into the mix, Johnson said potential corporate partners could be attracted by progress in myelin repair research.
“If this works like we think and we hope it will, it will be demonstrating something that’s never been demonstrated before,” Johnson said. “That will embolden others to focus more on this (myelin repair) area and gives the industry much more comfort.”
New foundations
In all, the MRF has plowed more than $45 million into myelin repair research over the past decade.
Life sciences leaders have long talked about the role of disease foundations in helping companies complete enrollment in clinical trials or fund drug development through the so-called “valley of death,” the critical point early in the trial process where cash traditionally runs short.
Yet while the Myelin Repair Foundation funds individual researchers worldwide — gathered under the umbrella of what it calls an Accelerated Research Collaboration, or ARC — it also taps its own research staff in Sunnyvale.
It was through that collaboration that the generic drug came to the MRF’s attention. An ARC researcher at the University of Chicago more than 15 months ago read a paper, Johnson said, that mentioned the drug’s use in the ER stress pathway.
The foundation has funded research in that pathway for some six years.
The paper’s authors “weren’t making any connection between that pathway and MS or myelin repair,” Johnson said, but it clicked with the researcher. The foundation quickly lined up a supply of the compound and tested it in Sunnyvale as well as in the labs of four separate ARC researchers.
The foundation’s work is especially important because approved MS treatments, and most research until a decade ago, have focused on controlling inflammation caused by the immune system. The foundation, instead, has zeroed in on methods to stop the erosion of myelin and potentially restoring myelin in MS patients.
“If you talk to most companies in multiple sclerosis, they would now say myelin repair is the next generation of treatment,” Johnson said. “We’ve moved that far in only nine years. That’s huge.”
La Myelin Repair Foundation (MRF), di cui Scott Johnson - un malato di SM che ha deciso 9 anni fa di avviare questa Fondazione, con l'obiettivo di trovare una soluzione per la remielinizzazione - è CEO, presidente e fondatore, ha annunciato di aver raggiunto un accordo con National Institutes of Health per uno studio, che dovrà verificare l'utilizzo di un farmaco per la ipertensione - già in commercio - per la terapia della Sclerosi Multipla (SM).
Il nome del farmaco non è stato rivelato, e gli è stato per adesso attribuito il codice MRF-008.
Avevamo già parlato precedentemente di Lisinopril, un ace-inibitore utilizzato per l'ipertensione. Non sappiamo se si tratti dello stesso prodotto o di uno simile. La cosa importante è che, in questi casi, trattandosi di prodotto già in commercio da una ventina di anni e utilizzato da milioni di persone, si può passare velocemente agli studi sulla sua efficacia, essendo la parte relativa alla sicurezza già confermata.
I ricercatori ritengono che MRF-008 potrebbe fermare la degenerazione della mielina e promuovere la crescita di nuova mielina.
Questa prima fase potrebbe completarsi entro 15 mesi, per poter poi passare ad uno studio con un maggior coinvolgimento di pazienti.
Il lavoro della MRF è particolarmente importante perché la maggior parte della ricerca fino a un decennio fa si è concentrata sul controllo dell' infiammazione causata dal sistema immunitario. La fondazione, invece, si è concentrata su metodi per fermare l'erosione della mielina e potenzialmente ripristinare la mielina nei pazienti con SM.
"Se parlate con la maggior parte delle aziende impegnate nella SM, adesso esse vi diranno che la prossima generazione di trattamento riguarda la riparazione delle mielina", ha detto Johnson . "Abbiamo spostato il tiro così lontano in soli nove anni. E questo è un enorme risultato."
A generic high blood pressure drug could unlock a new treatment window for multiple sclerosis patients in a groundbreaking study championed by a Bay Area nonprofit.
The Myelin Repair Foundation of Saratoga in the next few months will launch a Phase I safety trial of the drug, approved two decades ago to treat hypertension, said President and CEO Scott Johnson.
But while the road to winning Food and Drug Administration approval as a low-cost MS treatment is as tricky as it would be for any other compound, the drug’s generic use complicates the economics. For-profit drug makers may hesitate to invest in costly clinical trials, since the generic version would likely undercut the price a company could charge.
That restriction on pricing, in turn, could slow the drug’s availability on a broad scale for the world’s roughly 2.3 million MS patients.
What’s more, insurers may balk at covering a generic heart drug’s unapproved use in MS, even if physicians prescribe it.
But if a company tweaks the generic drug’s formula to receive new patents covering MS, the company could charge a price that covers clinical trial costs and make a tidy profit.
For the Myelin Repair Foundation, those development and commercialization issues have been prominent over the past several months. Foundation leaders have been in discussions with advisers — including a representative of Cambridge, Mass.-based MS drug developer Biogen Idec Inc. (NASDAQ: BIIB) — academic researchers and federal regulatory officials.
Now, thanks to an arrangement with the National Institutes of Health announced Monday, the foundation hopes to raise $2.5 million to cover its study costs.
“We never imagined funding any part of clinical trials,” said Johnson, an MS patient who 10 years ago founded the MRF. “All we care about is patients and getting treatments to patients.”
Moving quickly
A couple decades of safety data from the drug’s use for high blood pressure could accelerate its development in MS. The Phase I study — which typically would look at the drug’s safety in healthy volunteers — will focus only on MS patients, and a smaller number of patients at that, Johnson said. That way, he said, researchers can see if any distinct side effects pop up in people with the disease.
Historically, the drug’s side effects include drowsiness and dry mouth.
The trial, Johnson said, could “set a standard for future myelin repair clinical trials.”
Multiple sclerosis is a chronic roller coaster of a disease, highlighted by recurring, increasingly debilitating flare-ups followed by extended recovery periods. It is a particularly difficult disease to treat because no two people have exactly the same experience.
About 2.3 million people worldwide have MS, according to the National Multiple Sclerosis Society.
As its name suggests, the Myelin Repair Foundation’s research has zeroed on the fatty myelin sheaths that wrap around and protect nerve cells in the central nervous system. The loss of myelin is akin to exposing wires in an electrical cord — in this case causing neurons to misfire and leading to difficulty walking, vision problems and other symptoms.
The Myelin Repair Foundation isn’t revealing the generic drug’s name for now but is calling it MRF-008.
Researchers believe MRF-008 could stop the degeneration of myelin as well as promote the growth of new myelin by modulating a pathway, known as the endoplasmic reticulum stress, or ER stress. That pathway is believed to go into overdrive in MS patients, but early research shows it is controlled by MRF-008.
The foundation’s Phase I study is one of the first — if not the first — undertaken with a cooperative research and development agreement between a nonprofit and the NIH, Johnson said. The deal splits clinical trial responsibilities — and, importantly, costs — between the foundation and the NIH.
The study could be completed within 15 months of its start and move quickly into a larger mid-stage trial, Johnson said.
The $2.5 million price tag is minute for for-profit drug developers, but is a substantial investment for a nonprofit.
Although the economics of drug development make it unlikely that a company would jump into the mix, Johnson said potential corporate partners could be attracted by progress in myelin repair research.
“If this works like we think and we hope it will, it will be demonstrating something that’s never been demonstrated before,” Johnson said. “That will embolden others to focus more on this (myelin repair) area and gives the industry much more comfort.”
New foundations
In all, the MRF has plowed more than $45 million into myelin repair research over the past decade.
Life sciences leaders have long talked about the role of disease foundations in helping companies complete enrollment in clinical trials or fund drug development through the so-called “valley of death,” the critical point early in the trial process where cash traditionally runs short.
Yet while the Myelin Repair Foundation funds individual researchers worldwide — gathered under the umbrella of what it calls an Accelerated Research Collaboration, or ARC — it also taps its own research staff in Sunnyvale.
It was through that collaboration that the generic drug came to the MRF’s attention. An ARC researcher at the University of Chicago more than 15 months ago read a paper, Johnson said, that mentioned the drug’s use in the ER stress pathway.
The foundation has funded research in that pathway for some six years.
The paper’s authors “weren’t making any connection between that pathway and MS or myelin repair,” Johnson said, but it clicked with the researcher. The foundation quickly lined up a supply of the compound and tested it in Sunnyvale as well as in the labs of four separate ARC researchers.
The foundation’s work is especially important because approved MS treatments, and most research until a decade ago, have focused on controlling inflammation caused by the immune system. The foundation, instead, has zeroed in on methods to stop the erosion of myelin and potentially restoring myelin in MS patients.
“If you talk to most companies in multiple sclerosis, they would now say myelin repair is the next generation of treatment,” Johnson said. “We’ve moved that far in only nine years. That’s huge.”
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