BioSpectrum (Asia) - - BIO EDIT - Milind Kokje Chief Edi­tor milind.kokje@mmac­tiv.com

The world is now at the thresh­old of a rev­o­lu­tion in med­i­cal treat­ment in the form of cell and gene ther­a­pies which are fast emerg­ing as new ther­a­peu­tic modal­i­ties. Var­i­ous de­vel­op­ments sur­round­ing the cell and gene ther­a­pies are be­ing re­ported with sci­en­tists work­ing on solv­ing tech­ni­cal hur­dles in the treat­ment, suc­cesses in us­ing the ther­a­pies on dif­fer­ent dis­eases, and com­pa­nies es­tab­lish­ing man­u­fac­tur­ing plants.

A new study con­ducted by Stan­ford Univer­sity re­searchers hopes to solve a ma­jor prob­lem of gene ther­apy, an au­toim­mune at­tack. In an ex­per­i­ment on mouse they have shown that the gene ther­apy can be ef­fec­tive with­out caus­ing a dan­ger­ous side ef­fect of au­toim­mune re­ac­tion which the pa­tient’s im­mune sys­tem is en­coun­ter­ing for the first time. Sev­eral such hur­dles in the gene ther­apy de­vel­op­ment are be­ing elim­i­nated with re­search and in­no­va­tions.

There have been sev­eral re­ports in the last two years of the en­cour­ag­ing clin­i­cal re­sults where the ther­apy was used in var­i­ous dis­eases like haemophilia, sickle cell dis­ease, mul­ti­ple can­cers, sev­eral neu­rode­gen­er­a­tive dis­or­ders and ge­netic dis­eases. A teenage boy in France was cured in March of a ge­netic dis­or­der us­ing ex­per­i­men­tal gene ther­apy. GSK’s gene ther­apy Striv­elis is avail­able since 2016 for a rare dis­ease among the chil­dren. Spark Ther­a­peu­tics’s Lux­turna is ap­proved for cor­rect­ing ge­netic blind­ness. Three CAR-T cell ther­a­pies are al­ready ap­proved in US by the FDA to treat blood can­cers. They in­clude No­varit’s Kym­riah, use­ful for rare blood can­cer treat­ment, and Kite Pharma’s Yescarta.

Be­sides these, many more ther­a­pies are un­der clin­i­cal in­ves­ti­ga­tion. These de­vel­op­ments are also re­flect­ing in the stocks mar­ket. Sang­amo Ther­a­peu­tics’s shares in­creased by over 33 per cent pre­vi­ous month when it re­ported in­terim data for its haemophilia A ther­apy. As a re­sult of these de­vel­op­ments, MIT de­clared 2017 as the year of gene ther­apy break­throughs.

Sci­en­tists feel that cell and gene ther­apy is fun­da­men­tally dif­fer­ent from other reg­u­lar ther­a­pies since in­stead of treat­ing symp­toms, it cor­rects the ge­netic cause of a dis­ease. So far, these ther­a­pies have been in­tro­duced for rare dis­eases and hence the num­ber of pa­tients is small. But that will not be the case in the fu­ture. Suc­cesses in CART-cell ther­a­pies demon­strate the need for ca­pac­ity ex­pan­sion. Some de­vel­op­ments are tak­ing place on that front too.

Swiss biotech com­pany Lonza opened its cell and gene ther­apy man­u­fac­tur­ing plant in US in April. The 30,000 sq feet fa­cil­ity will be used for en­gi­neer­ing viruses and gene ther­apy vec­tors since its short­ages have cre­ated bot­tle­necks in de­vel­op­ment of the ther­apy. A month prior to that Su­mit­omo Dainip­pon Pharma com­pleted its man­u­fac­tur­ing plant for Re­gen­er­a­tive Medicine and Cell Ther­apy, which has been built to pro­duce cells for use in clin­i­cal tri­als.

Al­though var­i­ous pos­i­tive de­vel­op­ments are be­ing seen in cell & gene ther­a­pies, few chal­lenges need to be ad­dressed. De­vel­op­ing cell en­gi­neered prod­ucts is one such chal­lenge due to many as­pects in­clud­ing pro­duc­tion, de­liv­ery, reg­u­la­tory and test­ing.

As the field is still young, one of the early chal­lenges is the high price tag these ther­a­pies come with. uniQure’s Gly­bera, the first gene ther­apy, was with­drawn last year due to pric­ing is­sue. In or­der to over­come this hur­dle some com­pa­nies are of­fer­ing full money back in case of fail­ures or charg­ing only those pa­tients who are show­ing pos­i­tive re­sults in the first month. But it is a tem­po­rary so­lu­tion that can only be adopted in the ini­tial phases.

Ac­cord­ing to the ex­perts, an­other hur­dle re­lated to the pric­ing is re­im­burse­ment of the treat­ment cost from the in­sur­ance com­pany. Es­tab­lish­ing health in­sur­ance re­im­burse­ment for a new cat­e­gory of prod­ucts which do not fit the es­tab­lished health­care de­liv­ery or in­sur­ance model is chal­leng­ing and time con­sum­ing.

Once these ob­sta­cles and chal­lenges are ad­dressed, cell & gene ther­a­pies can be­come a com­mon prac­tice. It seems that a new rev­o­lu­tion of medicine is around the cor­ner.

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